1 (edited by Alan Robert Ross 2020-11-17 09:13:24)

Topic: Biotech Generally, including Regulatory

Here's 2 cases of the dreaded CRL (complete response letter), where the FDA rejects a NDA or BLA. The FDA seems to be giving a lot of these and the OMER shorts are hoping Greg gets one.
==============================================
November 17, 2020 07:32 AM ESTUpdated 07:59 AM R&DRegulatory
UPDATED: Alkermes is handed a CRL for ‘3831, marking another setback on the R&D front
John Carroll
Editor & Founder
Alkermes $ALKS is going to have to wait some more before launching its anti-psychotic combo drug ALKS-3831. The FDA has handed the biotech a CRL, rejecting the application in the company’s latest setback in R&D.

After getting a majority of the outside experts that reviewed the drug for the FDA to provide a thumbs up on approval, the biotech reported today that their manufacturing operations didn’t pass muster during their remote inspection of records — an alternative to a formal survey the agency adopted last summer as a result of the pandemic.

Alkermes emphasized that regulators didn’t raise any questions about the combo itself, which is designed to provide the anti-psychotic therapy Zyprexa without the threat of severe weight gain. There are no new requirements for additional studies. But the manufacturing review unveiled a problem with the tablet coating process at their factory in Wilmington, OH.

advertisement
advertisement
According to Alkermes:

The observations noted in the CRL were specific to certain development batches of ALKS 3831. The company believes this issue has since been resolved and that sufficient data is available to address these observations. Alkermes is preparing those data for submission and plans to work closely with the Agency to resolve these items in a timely manner and complete labeling discussions for the application.

Alkermes has run through a series of frustrations on the R&D front in recent years. A key depression drug failed a late-stage study in the spring of last year. And even if they get an OK on ‘3831 soon, the company still faces low expectations on the sales front, with peak estimates in some cases hovering around $300 million.
=======================================
November 17, 2020 07:08 AM EST Regulatory
Penny stock player Adamis gets another CRL for high-dose naloxone as shares crater
Max Gelman
Associate Editor
The FDA has once again shut the door on micro cap biotech Adamis Pharmaceuticals’ high dose naloxone injection.

Regulators handed down the program’s second CRL in nearly 12 months, Adamis announced Monday, sending the penny stock spiraling. The new rejection came as a result of new chemistry, manufacturing and controls issues, though Adamis noted that none of the problems stemmed from the “extractables and leachables testing” problems that caused the first thumbs down.

“To me, it is very surprising to have new issues brought up this late in the review process,” CEO Dennis Carlo said in a statement. “We believe the comments and recommendations stated in the CRL can be addressed and overcome.”

advertisement
advertisement
Investors shunned shares at the news, sending Adamis $ADMP stock plunging roughly 41% in Monday trading.

The San Diego-based biotech works on specialty products, seeking to develop lower-cost alternatives to drugs already on the market. Adamis’ high-dose naloxone injection was aimed at treating opioid overdoses, as products like Narcan and Evzio generally use less than Adamis’ proposed 5 mg/0.5 mL dose.

Adamis re-submitted its application in May, about six months after first being turned away. Carlo said at the time that he felt the additional data addressed all the issues related to the FDA’s letter, and the company had entered into a distribution agreement for the program that would have, pending approval, totaled up to $26 million in upfront cash and milestone payments.

But regulators evidently saw something they didn’t like — again. Both CRLs for this candidate have now dealt with CMC concerns, and Adamis has requested a Type A meeting to resolve any outstanding questions.

This is not the first Adamis program to face rejection at the FDA. Back in February 2019, the agency handed back the company’s application for a lower-cost erectile dysfunction drug, saying Adamis did not provide enough data for a review. The experimental drug was a fast-disintegrating version of tadalafil, the same active ingredient in Eli Lilly’s Cialis.

Adamis’ main product is an epinephrine injectable called Symjepi, which is used to treat allergic reactions and anaphylactic shock similar to Mylan’s blockbuster Epipen. The plan was to use the same delivery system for their naloxone injection as Symjepi. The biotech is also developing cheaper alternatives in a range of acute respiratory diseases, such as Covid-19, influenza, asthma and COPD

(T)he issue here is not just related to a delay–it’s also a negative read on execution, with a potential follow-through onto stock sentiment, as this represents another regulatory setback for ALKS after the high profile rejection of ALKS5461 in MDD two years ago. These are obviously very different situations. But for ALKS, the pipeline is naturally a major driver of value creation and operating leverage, so this could increase pre-existing pressures to make a more concerted effort toward managing spend/investments.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

2

Re: Biotech Generally, including Regulatory

STAT
The SEC has noticed all those pre-planned stock sales

Below the unreservedly good vaccine news from Moderna and Pfizer is the slightly more complicated fact that company executives sold millions of shares as their stock prices soared. And with so much scrutiny on those pre-planned sales, the head of the SEC believes there should be a “cooling-off period” between trades.

Jay Clayton, speaking at a Senate hearing yesterday, advocated for a change to SEC rules that would force executives to wait a matter of months between setting up a trading plan and actually selling stock.

That might have saved Pfizer last week’s PR headache, which stemmed from CEO Albert Bourla’s planned sale of $5.6 million worth of shares. That was done under a plan set up in August, according to SEC records, meaning it might have been impossible under revised rules. But Clayton’s proposal wouldn’t appear to affect Tal Zaks, the Moderna chief medical officer who makes about $1 million a week selling company shares. His trading plan took effect in March, according to SEC filings.
************************
First criticism of the EUA process, and then reform

The FDA has been intensely criticized over some of the emergency use authorizations it has issued during the Covid-19 pandemic — including, most notably, the now-revoked EUA for hydroxychloroquine.

A government watchdog report issued yesterday found the agency has failed to “uniformly disclose its scientific review of safety and effectiveness data” for emergency use authorization as it does for medicines undergoing the normal review process. That's contributed to waning public confidence in the FDA itself, as STAT's Ed Silverman writes.

Coincidentally, the FDA yesterday pledged to start publicly sharing the reviews of scientific data and other information that they use to grant, revise, or revoke an EUA. Joshua Sharfstein, a former top FDA official, told STAT the move is a step toward transparency. "We’ll have to see what this means in practice, but I do think it’s the right principle," he says.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

3

Re: Biotech Generally, including Regulatory

Note the date of the announcement (Nov. 18th) and the decision target date in May 2021.
That suggests the NORMAL Priority Review timeline and suggests that an announcement of Priority Review may give us some information about when the decision date will be.

https://charts.stocktwits.com/production/original_258144065.png

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

4

Re: Biotech Generally, including Regulatory

Eiger BioPharmaceuticals Sells Priority Review Voucher for $95 Million

- Eiger and The Progeria Research Foundation will share proceeds 50/50

- Non-dilutive capital further strengthens Eiger's balance sheet
PALO ALTO, Calif., Nov. 23, 2020 /PRNewswire/ -- Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that it has entered into a definitive agreement to sell its Priority Review Voucher (PRV) for a lump sum payment of $95 million.  Eiger will retain fifty percent of the proceeds, or $47.5 million, under the terms of the Collaboration and Supply Agreement with The Progeria Research Foundation (PRF).

The PRV was granted in conjunction with the recent approval by the U.S. Food and Drug Administration of ZokinvyTM (lonafarnib) for treatment of Progeria and processing-deficient Progeroid Laminopathies.  The transaction remains subject to customary closing conditions, including anti-trust review.

"The sale of the PRV provides Eiger with an important source of non-dilutive capital and further strengthens our balance sheet.  The proceeds allow us to continue to ensure that all diagnosed children and young adults worldwide with Progeria and processing-deficient Progeroid Laminopathies have access to Zokinvy and to advance our late-stage pipeline that now includes three breakthrough therapy designated programs," said David Cory, President and CEO of Eiger.  "We are proud that Zokinvy is our first approved product and the first approved therapy for children and young adults with Progeria and processing-deficient Progeroid Laminopathies."

For full prescribing information, visit www.zokinvy.com.

About Zokinvy™ (lonafarnib)
Zokinvy blocks the accumulation of defective, farnesylated proteins which form tight associations with the nuclear envelope, leading to cellular instability and the process of premature aging in children and young adults with Progeria and processing-deficient Progeroid Laminopathies.

Eiger licensed exclusive worldwide rights to lonafarnib from Merck, known as MSD outside of the United States and Canada.  Merck will not receive any milestone payments for the development of lonafarnib for the treatment of Progeria, and has waived royalty obligations from Eiger for a specified quantity of lonafarnib.

INDICATION
ZOKINVY is indicated in adult and pediatric patients 12 months of age and older with a body surface area (BSA) of 0.39 m2 and above:

To reduce the risk of mortality in Hutchinson-Gilford Progeria Syndrome (HGPS)
For the treatment of processing-deficient Progeroid Laminopathies with either:
Heterozygous LMNA mutation with progerin-like protein accumulation
Homozygous or compound heterozygous ZMPSTE24 mutations

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

5

Re: Biotech Generally, including Regulatory

This could be quite significant .........

https://medicalxpress.com/news/2020-12- … cline.html


DECEMBER 1, 2020
Drug reverses age-related cognitive decline within days
by University of California, San Francisco

Just a few doses of an experimental drug can reverse age-related declines in memory and mental flexibility in mice, according to a new study by UC San Francisco scientists. The drug, called ISRIB, has already been shown in laboratory studies to restore memory function months after traumatic brain injury (TBI), reverse cognitive impairments in Down Syndrome , prevent noise-related hearing loss, fight certain types of prostate cancer , and even enhance cognition in healthy animals.
In the new study, published December 1, 2020 in the open-access journal eLife , researchers showed rapid restoration of youthful cognitive abilities in aged mice, accompanied by a rejuvenation of brain and immune cells that could help explain improvements in brain function.
"ISRIB's extremely rapid effects show for the first time that a significant component of age-related cognitive losses may be caused by a kind of reversible physiological "blockage" rather than more permanent degradation," said Susanna Rosi , Ph.D., Lewis and Ruth Cozen Chair II and professor in the departments of Neurological Surgery and of Physical Therapy and Rehabilitation Science.
"The data suggest that the aged brain has not permanently lost essential cognitive capacities, as was commonly assumed, but rather that these cognitive resources are still there but have been somehow blocked, trapped by a vicious cycle of cellular stress," added Peter Walter , Ph.D., a professor in the UCSF Department of Biochemistry and Biophysics and a Howard Hughes Medical Institute investigator. "Our work with ISRIB demonstrates a way to break that cycle and restore cognitive abilities that had become walled off over time."
Could Rebooting Cellular Protein Production Hold the Key to Aging and Other Diseases?
Walter has won numerous scientific awards, including the Breakthrough , Lasker and Shaw prizes, for his decades-long studies of cellular stress responses. ISRIB, discovered in 2013 in Walter's lab, works by rebooting cells' protein production machinery after it gets throttled by one of these stress responses—a cellular quality control mechanism called the integrated stress response (ISR; ISRIB stands for ISR InhiBitor).
The ISR normally detects problems with protein production in a cell—a potential sign of viral infection or cancer-promoting gene mutations—and responds by putting the brakes on cell's protein-synthesis machinery. This safety mechanism is critical for weeding out misbehaving cells, but if stuck in the on position in a tissue like the brain, it can lead to serious problems, as cells lose the ability to perform their normal activities, Walter and colleagues have found.
In particular, recent animal studies by Walter and Rosi, made possible by early philanthropic support from The Rogers Family Foundation, have implicated chronic ISR activation in the persistent cognitive and behavioral deficits seen in patients after TBI, by showing that, in mice, brief ISRIB treatment can reboot the ISR and restore normal brain function almost overnight.
The cognitive deficits in TBI patients are often likened to premature aging, which led Rosi and Walter to wonder if the ISR could also underlie purely age-related cognitive decline. Aging is well known to compromise cellular protein production across the body, as life's many insults pile up and stressors like chronic inflammation wear away at cells, potentially leading to widespread activation of the ISR.
"We've seen how ISRIB restores cognition in animals with traumatic brain injury, which in many ways is like a sped-up version of age-related cognitive decline," said Rosi, who is director of neurocognitive research in the UCSF Brain and Spinal Injury Center and a member of the UCSF Weill Institute for Neurosciences. "It may seem like a crazy idea, but asking whether the drug could reverse symptoms of aging itself was just a logical next step."
ISRIB Improves Cognition, Boosts Neuron and Immune Cell Function
In the new study, researchers led by Rosi lab postdoc Karen Krukowski , Ph.D., trained aged animals to escape from a watery maze by finding a hidden platform, a task that is typically hard for older animals to learn. But animals who received small daily doses of ISRIB during the three-day training process were able to accomplish the task as well as youthful mice, much better than animals of the same age who didn't receive the drug.
The researchers then tested how long this cognitive rejuvenation lasted and whether it could generalize to other cognitive skills. Several weeks after the initial ISRIB treatment, they trained the same mice to find their way out of a maze whose exit changed daily—a test of mental flexibility for aged mice who, like humans, tend to get increasingly stuck in their ways. The mice who had received brief ISRIB treatment three weeks before still performed at youthful levels, while untreated mice continued to struggle.
To understand how ISRIB might be improving brain function, the researchers studied the activity and anatomy of cells in the hippocampus, a brain region with a key role in learning and memory, just one day after giving animals a single dose of ISRIB. They found that common signatures of neuronal aging disappeared literally overnight: neurons' electrical activity became more sprightly and responsive to stimulation, and cells showed more robust connectivity with cells around them while also showing an ability to form stable connections with one another usually only seen in younger mice.
The researchers are continuing to study exactly how the ISR disrupts cognition in aging and other conditions and to understand how long ISRIB's cognitive benefits may last. Among other puzzles raised by the new findings is the discovery that ISRIB also alters the function of the immune system's T cells, which also are prone to age-related dysfunction. The findings suggest another path by which the drug could be improving cognition in aged animals, and could have implications for diseases from Alzheimer's to diabetes that have been linked to heightened inflammation caused by an aging immune system.
"This was very exciting to me because we know that aging has a profound and persistent effect on T cells and that these changes can affect brain function in the hippocampus," said Rosi. "At the moment, this is just an interesting observation, but it gives us a very exciting set of biological puzzles to solve.
ISRIB May Have Wide-Ranging Implications for Neurological Disease
It turns out that chronic ISR activation and resulting blockage of cellular protein production may play a role in a surprisingly wide array of neurological conditions. Below is a partial list of these conditions, based on a recent review by Walter and colleague Mauro Costa-Mattioli of Baylor College of Medicine, which could potentially be treated with an ISR-resetting agent like ISRIB:
Frontotemporal Dementia
Alzheimer's Disease
Amyotrophic Lateral Sclerosis (ALS)
Age-related Cognitive Decline
Multiple Sclerosis
Traumatic Brain Injury
Parkinson's Disease
Down Syndrome
Vanishing White Matter Disorder
Prion Disease
ISRIB has been licensed by Calico, a South San Francisco, Calif. company exploring the biology of aging, and the idea of targeting the ISR to treat disease has been picked up by other pharmaceutical companies, Walter says.
One might think that interfering with the ISR, a critical cellular safety mechanism, would be sure to have serious side effects, but so far in all their studies, the researchers have observed none. This is likely due to two factors, Walter says. First, it takes just a few doses of ISRIB to reset unhealthy, chronic ISR activation back to a healthier state, after which it can still respond normally to problems in individual cells. Second, ISRIB has virtually no effect when applied to cells actively employing the ISR in its most powerful form—against an aggressive viral infection, for example.
Naturally, both of these factors make the molecule much less likely to have negative side effects—and more attractive as a potential therapeutic. According to Walter: "It almost seems too good to be true, but with ISRIB we seem to have hit a sweet spot for manipulating the ISR with an ideal therapeutic window.
Explore further
Detailed structure illuminates brain-enhancing drug's action
More information: Karen Krukowski et al. Small molecule cognitive enhancer reverses age-related memory decline in mice, eLife (2020). DOI: 10.7554/eLife.62048
Journal information: eLife

Provided by University of California, San Francisco

6

Re: Biotech Generally, including Regulatory

Wow, sounds too good to be true.  Those will be luck mice that get it first smile

Hopefully they get it into some humans soon

7

Re: Biotech Generally, including Regulatory

Great Find!

It does sound amazing and too wide-ranging an effect, but hopefully it turns out to be even half as useful in humans.

I was in brain research, early on, and all this is new to me, although I can definitively say that mouse brain function is not the same as humans, in some respects. The hippocampus is one of the big differences.

Hippocampal damage from trauma or aging in humans, more than animals, controls turning short-term memory into long-term memories. This is why it is of significant interest in age-related cognitive change. It also leads to more perseveration, when damaged in animals and people. This means you continue to try things over and over even though the actions do not allow you to reach your goal.

Any drug that is supposed to affect the things this one appears to do, would logically affect this part of the limbic system.

Regrettably I do not think Calico is public and it will likely take quite a while to get the drug to market (and then cost an arm & a leg).

https://www.calicolabs.com/

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

8

Re: Biotech Generally, including Regulatory

I've been poking around on the Calico website and see there are no publications listed since 2018.

Also that they are much more an academic 'translational' and theoretical series of labs and NOT a drug company.

The have no source of revenue other than partnerships with funding organizations/companies and their finances are not publicly available. 

ISRIB has been licensed by Calico, a South San Francisco, Calif. company exploring the biology of aging, and the idea of targeting the ISR to treat disease has been picked up by other pharmaceutical companies, Walter says.

I presume this means Calico has purchased a license (although they are the early-stage research company that I'd expect to OUT-LICENSE ISRIB to a drug company set up to do clinical research).

Regrettably this underlines how preliminary these findings are. I see no evidence that Calico will take the drug into humans. For all we know their license is only for animal work.

On the other hand, one of the academics also has an appointment to Howard Hughes Medical Institute and they are likely to want to see the new technology used in humans.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

9

Re: Biotech Generally, including Regulatory

from STAT

Here’s why pharma should fear Biden’s HHS pick

While the drug industry has had a less than harmonious relationship with Health and Human Services Secretary Alex Azar in recent years, President-elect’s Biden choice to replace him could leave pharma longing for the simpler days of 2020.

As STAT’s Nicholas Florko reports, California Attorney General Xavier Becerra, Biden’s pick for HHS, has been a vocal advocate for what drug makers consider the nuclear option: seizing companies’ patent rights in the name of bringing down the cost of medicine. In August, Becerra led a bipartisan letter of attorneys general urging Azar to break Gilead Sciences’ patent on the Covid-19 treatment remdesivir, advocating the use of an obscure patent law called march-in rights.

If Becerra wins confirmation to HHS and invalidates Gilead’s patent, it would be a shot across the bow that could forever change industry behavior. “As soon as we sort of cross that red line, the world is different, and there’s no question about that,” said Ben Ippolito, a senior scholar at the American Enterprise Institute.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

10

Re: Biotech Generally, including Regulatory

Greenwich LifeSciences Ends up 1,000% on Positive Phase 2 Results
Shares remained active in after-hours trading Wednesday on a day the stock was halted over a dozen times after announcing positive results for its breast cancer treatment candidate.
TONY OWUSU4 HOURS AGO
Greenwich LifeSciences GLSI ended up almost 1,000% Wednesday and remained volatile in after-hours trading Wednesday after the clinical-stage biopharmaceutical company announced positive news from the Phase 2b trial of its breast cancer treatment candidate GP2.

Shares of the Stafford, Texas-based company opened the day at $16.07 after closing the previous session at $5.20 per share. They traded above $110 a share before settling back to end regular trading at $57.10, up 998%. In after-hours trading the stock rose $16.70, or 29%, to $73.80

The stock was halted more than a dozen times as the stock took off in afternoon trading.

The company announced the results of the final five-year follow-up on disease-free survival curves evaluating the reduction of breast cancer recurrences and "a potent immune response" in women.

They plan to move to Phase 3 trials in 2021, according to Greenwich LifeSciencesCEO Snehal Patel.

"The poster presented at the SABCS is important because the Kaplan Meier survival curves and demographic data further validate our promising HER2 3+ (breast cancer) Phase IIb data and support our plan to commence a Phase III trial," Patel said.

Recurring breast cancer affects 1 in 8 women, according to Greenwich.

About 50% of women with recurring breast cancer don't respond to Herceptin and Kadcyla, intravenous drugs that are taken as part of a chemotherapy regiment. This can result in metastatic breast cancer, which carries a much poorer prognosis. GP2 is supposed to prevent breast cancer recurrences in patients who have previously undergone surgery.

"By addressing this unmet need, GP2 may reach a potential market exceeding $5 billion," Patel said.

Investors traded more than 16 million shares of Greenwich Wednesday, nearly 600 times its normal trading volume of about 30,000 shares.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

11

Re: Biotech Generally, including Regulatory

Wow, this is great news.  Hopefully P3 goes well.

It's unbelievable the response to a P2 result.  I'm also surprised that a P2 has a 5 year follow-up component.

Good for those shareholders.  Hopefully Omer will do something similar in 21, although I doubt it would be in 1 day

12

Re: Biotech Generally, including Regulatory

from Stat

This press release was worth $1 billion

Yesterday, at 9:05 a.m., a little-known biotech company called Greenwich LifeSciences put out a press release saying that data on its investigational cancer treatment would be presented via poster at a medical conference. Greenwich’s market capitalization at the time was about $60 million.

Over the next few hours, traders would bid up its stock by nearly 3,000%, giving it a market value exceeding $1 billion by around 2:30 p.m. Greenwich closed the day up about 1,000%, and then traded up another 20% or so after hours.

It’s worth noting that the data in that aforementioned poster are not new. Greenwich disclosed identical results in the documents it filed before going public in September. Also worth noting: The company had just $6,835 in cash at the time of its IPO, and the proceeds of that offering are expected to last just 12 months. If Greenwich can’t raise more money, it “may be unable to continue as a going concern,” according to its filings with the SEC.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

13

Re: Biotech Generally, including Regulatory

December 9, 2020 09:50 AM EST DealsOutsourcing
Peter Thiel's Palantir scores contract with the FDA to speed up regulatory reviews as delays keep growing
Conner Mitchell
Associate Editor
Peter Thiel, the billionaire libertarian who quietly backed away in 2020 from his once-ardent support of President Donald Trump, is no stranger to government contracts. His company Palantir has secured dozens of contracts with the feds and now has another big fish on the hook.


Peter Thiel
Palantir, the Denver-based data and analytics company Thiel co-founded in 2003, entered into an agreement this week with the FDA as the government agency struggles to keep up with a surge of regulatory approval needs due to the Covid-19 pandemic.

As part of a three-year, $44.4 million contract, the FDA will use Palantir’s software to expedite data and analytics services through the Center for Drug Evaluation and Research with the goal of speeding up the review of potential new medicines and products such as hand sanitizers. Bloomberg first reported the existence of the contract.

Hamstrung by Covid-related lockdowns, the FDA in recent months has had to delay numerous approval processes for myriad drugs — mainly due to facility inspection requirements made more difficult by pandemic travel restrictions.

Recent publicized FDA delays have included Bristol Myers Squibb’s CAR-T liso-cel, Spectrum‘s neutropenia candidate Rolontis and daxibotulinumtoxinA, a frown-line injection from Revance. Beyond that, it’s unknown exactly how far behind the agency is on review processes, but the outsourcing of certain review processes to Palantir software could help officials clear out the backlog.

Palantir has long worked closely with the US government, a relationship which has only grown closer since the pandemic began. The company, Bloomberg also reported, has secured over 100 contracts with private companies and government health groups — including software that government officials have said is critical to the Operation Warp Speed mission to inoculate Americans with a COVID-19 vaccine.

Palantir went public on the Nasdaq at the end of September, with an initial direct listing of $10 per stock share — translating to a valuation of $22 billion. The announcement of the FDA contract alone boosted company shares 21% on Tuesday, and $PLTR is currently valued at $28.59 per share.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

14

Re: Biotech Generally, including Regulatory

This could be a problem for a lot of companies, including Omeros' attempt to get narso approved ASAP. Woodcock has long been berated for being too quick to rush drugs that may help people survive and labelled too industry-friendly. I always thought she was PATIENT-FRIENDLY.

If she is delaying approvals to get the job as Commissioner, I would no longer think she is Patient-friendly.
=========================
March 11, 2021 08:18 AM EST Regulatory
FDA delays Kadmon’s priority review as apparent clampdown continues
Jason Mast
Editor
Kadmon Holdings just lost their priority review.

The immunology biotech announced Wednesday that the FDA has pushed back the decision date for their graft-versus-host disease drug belumosudil by three months, after the agency requested additional information from the drugmaker. That will effectively turn their 6-month priority review into a 9-month one, just below the FDA’s standard decision-making deadline.

It’s not particularly unusual for the FDA to demand new information amid a review, but it’s also not particularly common, especially for drugs that receive priority review and breakthrough designation — honors meant to signal the agency thinks the drug is important and put drugmakers and regulators in close contact to ensure it has a smooth path to patients.

advertisement
advertisement
And the delay comes after two other drugmakers with applications before the FDA announced that they are facing unexpected hurdles after regulators demanded new information. FibroGen announced the agency was pushing back a March 20 decision date on a kidney drug after deciding to convene an advisory committee hearing. And Acadia claimed that after receiving little word from the agency for months and with an April deadline approaching, regulators sent them a notice that their application contained “deficiencies.”

The cluster of decisions has stirred speculation that acting FDA chief Janet Woodcock, often criticized for being too close to industry, is taking a tougher stance as she angles to be nominated as commissioner. In addition to the three delays, the FDA also gave Athenex a surprise rejection on their oral chemotherapy, demanding a new trial. And they issued a rare public letter about ALS developer Brainstorm Therapeutics, trying to set the record straight on the efficacy (or lack thereof) of a drug their CEO has been touting.

At the same time, regulators have clamped down on the accelerated approval process, asking big companies to pull drugs off the market after confirmatory studies failed.

Although it’s not clear what new information Kadmon submitted, their path ahead appears smoother than FibroGen’s or Acadia’s. FibroGen is facing a historically unfriendly advisory committee. Acadia, which had been hoping to turn the anti-psychotic Nuplazid into a blockbuster, is unable to discuss labeling and post-marketing requirements at all with regulators. They were not given a new PDUFA date.

Kadmon announced last year that their drug improved symptoms in more than two thirds of patients with graft-versus-host disease, the often chronic inflammatory condition that can arise after a bone marrow transplant.

If approved, the drug would make a major victory and turnaround for CEO Harlan Waksal and his brother Sam Waksal, the CEO of the company at the center of the infamous Martha Stewart insider trading scandal, vindicating investors who decided to bet on him despite his criminal record.

“We remain confident in the data supporting our application for belumosudil in cGVHD and look forward to continuing to work closely with the FDA during the remainder of the review process,” Harlan Waksal said in a statement. “We are committed to bringing belumosudil to market, once approved, to help meet the needs of patients living with cGVHD.”

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

15

Re: Biotech Generally, including Regulatory

Let's pretend I'm an optimist.  To date, as far as I know, Greg has done everything possible according to the book.  And we have read suggestive statements, or heard them, coming from FDA.  A delay similar to the above at this point in time would be a kick in the gut.

How would delaying approvals help this lady get the job as Commissioner?  Because she'd be seen as tough?  What about correct, accurate, approving what should be approved, not approving what should not be approved.....IOW competent.  We'll see.....

16 (edited by Alan Robert Ross 2021-03-11 15:22:11)

Re: Biotech Generally, including Regulatory

Avi... you are talking about anti-business pro-Govt. Progressive and who they'd want to be FDA Commissioner and how much pressure they put on Biden.

Many people, including Doctors, think a trial with less than 1000 people can't be good enough to establish facts.

The average person knows nothing about science and much more about.... ANGELS.

I would hope that Dr. Woodcock would not sacrifice people's lives to get a job. We do not know who Biden has appointed to lower positions and whether they were anti-Pharma Progressives.

There are good reasons to not live large drug companies, but  arbitrarily slowing approval of new, potentially life-saving, drugs should not be done because of a change in ideology.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

17

Re: Biotech Generally, including Regulatory

I understand.  People acting in their own interest even if, after achieving the immediate goal by changing their behavior, is nothing new.  That's just how it is, I suppose.  I just want this scenario to not negatively affect Omeros, is all.

I guess we're just going to have to wait until such time as we have some concrete evidence as to how much Biden pursues and accomplishes Far Left goals and programs, if he wants to or has to.

18

Re: Biotech Generally, including Regulatory

"Wanting" is not relevant here because we have no control.
My job is to keep everyone aware of the potential risks and rewards that I uncover, so that each of you can factor them into you investment decisions.

At some point the pros and or the cons result in decisions, even if that decision is to wait for developments. The trend of FDA decision making has been favorable up to the time of the pandemic and then FDA got busy and were handicapped in the ability to travel to do inspections. Now, a regime change has once again cause additional uncertainly that has been enhanced by recent FDA decisions.

No need for Omeros longs to voice concern because that goes with being long, as does our preference for a fast approval for narso.

As almost all of us are influenced by the share price and the direction of the trend, if Omeros continues to move higher, we longs will likely ignore the extra bit of risk represented by the FDA. If, instead, OMER price reverses and goes back down, we will start to see FDA delays or unpredictability as a bigger risk, because we are affected by the need for validation in ambiguous situations... as I have tried to pound into everyone's head, including my own.

We can only try our best to understand that the way we humans make decision are not optimum in all situations and try to factor that into our decisions.

Note that it tends to be easier to buy when the price is surging and sell when it is declining and that makes sense but is contrary to making a lot of money if you are correct that what you are buying will go up. Everything being equal, we make more money when we buy on weakness and sell on strength (preferably at or near the top).

Nobody does this perfectly if they are not a time-traveler.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

19

Re: Biotech Generally, including Regulatory

Pharma Giants Get Their Pennies Pinched on Drug Pricing;
Charley Grant
charles.grant@wsj.com

The vaunted ability of pharmaceutical companies to name their own price is vanishing as middlemen increasingly call the shots

The pharmaceutical industry's reputation as an omnipotent market force is increasingly out of date. Washington, D.C., hasn't yet caught on, but Mr. Market is in on the secret.
Drug companies do have wide freedom to set their own sticker prices for their products in the lucrative U.S. market. That hardly means they have carte blanche. To actually sell medication, a drugmaker needs to convince public and private health plans to place their product on the plan's formulary, which is a list of drugs the plan is willing to purchase. That means paying middlemen rebates and discounts to choose their drug over any other rival treatments. Failure to secure favorable formulary access could mean low sales even for a highly-effective and safe medication.
Those middlemen argue that the rebates and discounts, coupled with their bulk buying power, help keep insurance premiums low. Other providers of essential services, like drug wholesalers that deliver medication to pharmacies, hospitals and other healthcare sites, also get paid as a share of a drug's list price. As a result, a drug's sticker price doesn't typically reflect how much revenue the manufacturer gets when a prescription is filled. The net price that the manufacturer actually realizes as revenue is a carefully guarded trade secret. As for what a patient with insurance pays out of pocket, Americans already know that the differences can be big and baffling. The decision depends on the design of the insurance plan.
No one should worry about drugmakers being able to make ends meet, but their lives are getting harder. Data released last month by drugmakers themselves show that their ability to raise prices is waning. French pharmaceutical giant Sanofi said that its average U.S. gross price rose by just 0.2% in 2020 and that the average net price it realized fell 7.8%.
"To secure that formulary position costs us more and more every year," said Adam Gluck, Sanofi's chief of corporate affairs, in an interview. The company says that the list price for its Lantus insulin is up 141% since 2012 but that the net price is down 53% over that same period.
It isn't just Sanofi facing this dynamic. Merck & Co. said last month that its average U.S. sticker price rose 3.1% in 2020 even as its average net price fell slightly. That is a sea change from recent years: In both 2015 and 2016 Merck's average list price rose by about 10% while the net price realized by the drug giant rose by 5.5%. Nearly half of Merck's gross sales went out the door to third parties as discounts last year. A decade ago, that tally was around 27%. Other drugmakers like Bristol-Myers Squibb report similarly high spreads between gross and net sales.
The data should put the industry in relatively good standing with Washington—especially after the industry proved able to develop and manufacture Covid-19 vaccines in record time. After all, the high cost of healthcare is a perpetual issue in U.S. politics, and drugmakers have historically been a popular target. While President Biden has focused on other priorities early in his term, he vowed to address high prescription drug prices during his campaign.
SHARE YOUR THOUGHTS
What should be done about high drug prices? Join the conversation below.
Don't expect that to take the heat off the industry completely: Lawmakers may not have figured out that drugmakers' pricing power is on the wane, and politicians aren't ones for nuanced arguments in any case. Wall Street seems to have cracked the code, at least. The stock market as a whole is ebullient, but Sanofi, Merck and Bristol-Myers Squibb all have share prices well below their record highs these daysin an ebullient stock market. A broad index of pharmaceutical stocks has underperformed the S&P 500 by 6 percentage points so far this year.
No matter how lucrative a business might be, investors tend to focus on the future. While big drugmakers generate huge amounts of cash flow, it can be a challenge to grow sales and profits. Drug development is an expensive and difficult art and the vast majority of drugs under development fail to reach the market. Even the greatest successes eventually stop selling at high prices, either because of patent expirations or the arrival of a newer, better medication. In recent years, big drug companies have had to resort to expensive biotechnology acquisitions to build their pipelines. With more and more dollars needed to move the needle, the result has been an industry rife with slow-growing top lines and valuations stuck in neutral.
Now that picture has darkened further as big drugmakers' ability to name their price in their best and biggest market is fading away. At least their public image is on the mend.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

20

Re: Biotech Generally, including Regulatory

March 11, 2021 12:00 PM EST Regulatory
Trump’s HHS planted ‘ticking timebomb’ to sunset thousands of regs, lawsuit says
Zachary Brennan
Senior Editor
Between the time President Joe Biden won the election and took office, the outgoing Trump administration proposed and finalized a rule that would indiscriminately eliminate thousands of HHS and FDA regulations if the agencies don’t take action, setting the new administration up for a time-consuming, regulation by regulation, reversal.

A new lawsuit, though, is looking to overturn the rule far more quickly.

“The outgoing administration planted a ticking timebomb set to go off in five years unless HHS, beginning right now, devotes an enormous amount of resources to an unprecedented and infeasible task,” the National Association of Pediatric Nurse Practitioners, American Lung Association, the Center for Science in the Public Interest (CSPI) and several other plaintiffs allege in a complaint filed this week in the US District Court for the Northern District of California.

“There’s no way the agency could in a thoughtful way revisit all of their regulations that would be affected by this. It’s a recipe for chaos,” Peter Lurie, president of CSPI and a former associate commissioner at the FDA, told Endpoints News in a phone interview.

The final rule was billed as a way to cut through the red tape and bureaucracy of agencies like the FDA, but as many as 2,000 FDA regulations and many more for HHS could be cut by 2026 — unless the agency conducts and finalizes retrospective reviews for each regulation.

Erin Fuse Brown, professor of law at Georgia State University, told Endpoints News via email: “If it were to go into effect, the sunset rule would have far-reaching consequences for all the agencies under HHS, including the FDA. The FDA’s rules would be subject to the sunset rule, and while they would not expire immediately, the agency would have to spend considerable resources to determine which rules are subject to review, when they would expire without review, and initiate that process now to avoid triggering automatic expiration.

“This would likely draw valuable agency resources and time away from the important regulatory work they already do, such as approving new drugs and devices, and introduce considerable uncertainty to regulated industry that depends on the predictability of regulations to bring their products to market, get reimbursed, or monitor their safety,” she added.

The final rule even acknowledges that commenters on the initial proposal took issue with the potential elimination of crucial FDA regulations, like ones on current good manufacturing practices or others that protect people in clinical trials. But former president Trump’s HHS responded with a short explanation that the assessment and review of such regulations will be up to the Biden administration.

So what are some ways to stop the sunset rule if the lawsuit fails? The Biden administration could just withdraw the Trump-era rule, Lurie noted. But Fuse Brown explained that HHS would still need to go through a notice-and-comment procedure to do so because an agency must use the same rulemaking procedures to rescind a rule as to enact or amend it.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

21

Re: Biotech Generally, including Regulatory

biotech CAN BE DANGEROUS
IDRA Failed
DOWN 65% TODAY

Idera Pharmaceuticals Announces Results From ILLUMINATE-301 Trial of Tilsotolimod + Ipilimumab in anti-PD-1 Refractory Advanced Melanoma

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

22

Re: Biotech Generally, including Regulatory

March 19, 2021 07:30 AM EDT People
Founders of poop-testing uBiome, reportedly on the run, charged by SEC with defrauding investors
Max Gelman
Associate Editor
Following several high-profile hiccups a few years ago that included an FBI raid and ultimately led to a bankruptcy filing, uBiome saw its founders run into more trouble Thursday: securities fraud.


Jessica Richman
The SEC charged Jessica Richman and Zachary Apte, the co-founders of the poop-testing startup uBiome, with defrauding investors out of $60 million through misleading statements and false representations of the company’s prospects. According to the SEC complaint, the charges stem from a 2018 Series C round valuing the company that once appointed ex-Novartis CEO Joe Jimenez to its board at nearly $600 million.

Regulators submitted their filing in federal court in San Francisco, and are seeking to bar Richman and Apte from serving in future officer and director positions. Thursday’s indictment describes Richman and Apte as fugitives, per a Wall Street Journal report.

Founded way back in 2012, uBiome burst onto the scene with a pledge to crowdsource the sequencing and mapping of the human microbiome — the bacterial ecosystem inside the gut that some scientists say has wide-ranging impacts on one’s health. The company pitched itself as the next 23andMe, offering to analyze stool samples and return health-related advice.

It was an idea that captured the imagination of the early-2010s era Silicon Valley. By the time its Indiegogo funding page closed in February 2013, uBiome had raised over $350,000. The company also recruited several notable scientists over the years, including geneticist George Church to its scientific advisory board.

uBiome eventually expanded their products to include vaginal health and STDs women face. And to ostensibly beef up a drug R&D plan, the company added Jimenez to its board in 2018, despite a scandal where Jimenez paid former President Donald Trump’s personal attorney Michael Cohen a $1.2 million contract.

But the house of cards began to crumble when some researchers sounded alarms that the science behind the company didn’t all add up. They said that, like when 23andMe first started, there simply hadn’t been enough research into the microbiome to offer sweeping health advice based on a stool sample. 23andMe, after facing its own criticism, eventually gained FDA approval for several of its services.

Then in early 2019, uBiome cut 55 jobs from its 300-person workforce. That was quickly followed by reports that April of an impending FBI inquiry into uBiome’s business practices, which ultimately culminated in a raid of their offices. Officials were concerned the startup had allegedly hounded doctors to approve their tests with little oversight and overbilled customers.

The SEC brought those concerns to light in its complaint. Regulators claim that in order to generate revenue, Richman and Apte directed uBiome to fool doctors into ordering unnecessary tests and, despite several warnings from employees and their general counsel, ignored the need for the tests to meet certain health insurance company requirements.

Defendants ignored these warnings and adopted and approved several improper billing practices that they knew, or were reckless in not knowing, fell below insurer requirements and thus, once discovered, would prompt insurers to reject reimbursement claims for uBiome’s clinical tests. Defendants engaged in deceptive acts to conceal facts pertinent to uBiome’s practices from the company’s general counsel, the uBiome board, prescribing doctors, and insurers.

After the investigation became public, Richman and Apte left the company to make room for a new management team. That September, uBiome filed for Chapter 11 bankruptcy, shifting to Chapter 7 liquidation a month later in order to sell off its data and IP.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

23

Re: Biotech Generally, including Regulatory

Biotech has good new and it has bad news.
It is important to keep the downside in mind. In this case, the Management were at least honest and not going to keep things going to continue to earn salaries and bonuses.
=========================
March 22, 2021 08:34 AM EDTUpdated 08:50 AM R&D
Those NDA plans Odonate had for mid-'21? They're being scrapped — along with the entire biotech — in a sudden implosion
John Carroll
Editor & Founder
Last summer, San Diego-based Odonate $ODT CEO Kevin Tang enthusiastically sought to bolster investors’ enthusiasm for the company with what the biotech called “a potential important clinical advance for patients with metastatic breast cancer.” Their late-stage study had hit the primary endpoint, the company noted, and they were laying the groundwork for an NDA in mid-2021.

That message didn’t work so well, though, as investors trimmed the company’s market cap considerably from its earlier, high-flying ways. But it was still in the game with a market cap north of $730 million — until this morning. The stock collapsed, eviscerating 75% of its value ahead of the bell in a slide down to cash.

After reconfirming its NDA plans recently, the 8-year-old biotech — which had 153 staffers at the end of last year — now says that after they’ve met with FDA officials, there’s no hope for their drug, tesetaxel, an oral chemo in the taxanes class. Or the company.

advertisement
advertisement
Odonate now says it will transfer patients to other drugs and wind down operations after determining that their data package is “unlikely to support FDA approval.”

Tang’s final, brief sendoff:

We thank the investigators, study team personnel, and especially the patients and their caregivers for their endeavors to improve treatments for patients with breast cancer.

The stock, which ended Friday at $19.03 with a market cap of $753 million, was halted ahead of the announcement. Next comes the collapse and a bevy of angry investors looking for a better explanation of what just happened.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

24

Re: Biotech Generally, including Regulatory

Sounds like a fraud if they needed to meet with the FDA to determine that their drug is a total failure.

25

Re: Biotech Generally, including Regulatory

Frauds don't just go out of business. They take what money they have and come up with another drug candidate.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

26

Re: Biotech Generally, including Regulatory

As Ed pointed out, some of what's in this article could be the reasons people are selling, but these are knowns and so is the Democratic bias compared to the GOP bias. I don't know what triggered the correction, but at this point, people are likely selling because that is the new trend and the ETFication of the market accentuates that. IMO the individual company story needs to be much better than the average narrative to buck the trend, unless you are one of the stocks that are zooming higher attracting the momentum trading people and computers.
===============
March 24, 2021 07:30 AM EDTUpdated 08:08 AM PharmaRegulatory
Tougher regulatory environment for biopharma? Debate ensues
Zachary Brennan Senior Editor

Onlookers beware: trying to interpret how rigid or lax the current FDA drug approval landscape might be based on a couple of seemingly inter-related approvals or rejections can be a fool’s errand.

But what about when this seemingly tougher approval environment is met with an FTC ready to pounce on any biotech merger, and Congress is poised to actually do something on drug prices soon? Well, that may be a different story, according to SVB Leerink’s biotech analyst Geoffrey Porges.

Porges told investors this week that he believes the biopharma industry and its investors “will need to adjust to a different regulatory environment to the one they have enjoyed for the last 5 years.”

“For many recent investors in the industry this may come as a surprise, but for long term investors it should amount to a return to the long-term reality of risk in the industry,” he wrote. “That reality seems certain to feature more restrictions on drug pricing (and therefore low or even negative net price contributions to growth), more challenging drug reviews at the FDA (including more CRL’s, 483’s and other adverse outcomes), and greater scrutiny of M&A (and therefore fewer transactions and lower premiums).”


Geoffrey Porges
In another investor note last week, Porges explained some of these inter-related signs of a tougher FDA, noting “a surprise notice” from the FDA about deficiencies in Acadia’s application for a label expansion for its Parkinson’s drug Nuplazid, and an unexpected disclosure from FibroGen that the FDA would hold an advisory committee meeting to review its roxadustat application.

“While these two datapoints don’t establish a trend, and the drugs are under review by different divisions of the FDA, the overlap of these two cases with the transition to a new administration does raise questions about whether the FDA under acting commissioner Janet Woodcock, who is effectively auditioning for the permanent role, is adopting a more stringent approach to drug reviews, at least for the time being,” Porges wrote.

Another possible sign of a tougher FDA: the agency’s recent decision to hold an advisory committee to review whether different indications for Roche’s Tecentriq, Merck’s Keytruda and Bristol Myers Squibb’s Opdivo should remain on the market after they were approved under the agency’s accelerated pathway but failed to pass muster in confirmatory trials.

But for others watching the FDA closely — these recent changes may not be indicative of any wider changes.


Lowell Schiller
“I don’t know if I would call it a shift,” Lowell Schiller, chief legal and regulatory officer of Aetion and former principal associate commissioner for policy at FDA, told Endpoints News.

While noting the FDA’s renewed focus on the accelerated approval pathway, he said the agency is making good on an issue the agency has “long believed in.” He also said he didn’t think there were any shifts in the agency’s stringency with regard to approvals.

As former President Trump’s regulatory freeze thaws and more policy is released, Schiller said, “We’ll see what the priorities are, but the vast majority of policy from FDA is not partisan, and much of it’s made up of long-standing initiatives. It’s just a matter of when they’re completed,” he added.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

27 (edited by Alan Robert Ross 2021-03-27 01:57:00)

Re: Biotech Generally, including Regulatory

Biotech is getting whacked lately and to some extent it is because of talk of the Democratic government Nationalizing IP of drug patents.

I don't think that will happen in the US for a number of reasons, but there are legal reasons too. Here's an article helping to cause the Biotech sales:

And then some tweets about the legality of doing it.
===================
White House weighs temporarily lifting intellectual property shield on Covid-19 vaccines
PUBLISHED FRI, MAR 26 20215:06 PM EDTUPDATED FRI, MAR 26 20215:44 PM EDT
Kayla Tausche
The White House is weighing whether to temporarily lift intellectual property protections on Covid-19 vaccines and treatments.
The move would allow other countries to replicate existing vaccines.
Concerns have grown about the fact that the U.S. and a handful of other wealthy countries hold the right to a disproportionate amount of the global supply.

The White House is weighing whether to suspend intellectual property protections for Covid-19 vaccines and treatments, in response to pressure from developing nations and subsequent support from progressive lawmakers, according to three sources familiar with the matter.

A temporary suspension of intellectual property protections would apply to all medical technologies to treat or prevent Covid-19. South Africa and India made a formal request to the World Trade Organization to waive the protections until the pandemic is over, but the issue was tabled without a resolution.

The White House convened a meeting of deputy-level policymakers on March 22, a senior administration official said, but they reached no final decision.

The White House’s review comes in response to a letter sent in late March by House Speaker Nancy Pelosi, urging the administration to study the issue after several Democratic colleagues — including Reps. Earl Blumenauer of Oregon, Rosa DeLauro of Connecticut and Jan Schakowsky of Illinois — brought it to her attention. The letter has not been released to the public. But a senior aide said Pelosi supports the position of her members, who are in favor of issuing such a waiver, even on a temporary basis.

“The view is ‘We’re not safe until the world is safe,’” one of the sources said of the support from progressives on Capitol Hill.

The move would allow other countries to replicate existing vaccines. The United States has so far approved three vaccine shots: one developed by American company Pfizer and German-based BioNTech, another produced by U.S. firm Moderna and the third made by American company Johnson & Johnson.

Concerns have grown about the U.S. and a handful of other wealthy countries owning the rights to a disproportionate amount of the global vaccine supply, while other nations struggle to inoculate their people.

The Hill first reported the support for the move from progressive lawmakers.

The Office of the U.S. Trade Representative, which would be expected to deliver a final verdict to the World Trade Organization, said saving lives and ending the pandemic remains the “top priority of the United States.”

“As part of rebuilding our alliances, we are exploring every avenue to coordinate with our global partners and are evaluating the efficacy of this specific proposal by its true potential to save lives,” USTR spokesman Adam Hodge told CNBC.

The pharmaceutical industry has fiercely opposed waiving the patent protections. It worries doing so will undermine innovation to fight future diseases.

CNBC contacted Pfizer, Moderna and Johnson & Johnson for comment. 

Clete Willems, former deputy director of the National Economic Council, said lifting the protections would set a dangerous precedent of sharing technology.

“The administration needs to steer clear of this trap, which would undermine decades of U.S. policy against forced technology transfer to countries like China and won’t directly increase vaccine distribution,” Willems, now a partner at Akin Gump, told CNBC. “The model that they are pursuing with their Quad partners is much more promising.”

In advance of a meeting on March 12, the Quad — a group comprise of the U.S., India, Japan and Australia that seeks to counter the influence of China — announced a complex financing deal that would enhance manufacturing of vaccines in the Indo-Pacific, where there has been a shortage. The group set a goal of delivering up to 1 billion vaccines by 2022.

Nearly 19% of American adults, and about 15% of the total U.S. population, are fully vaccinated, according to Centers for Disease Control and Prevention data.
=================
NovaCapital2020

Yawn. Not happening. Here’s why:

Intellectual property is property (even if not physical). Only way gov’t can claim property is via eminent domain, which requires two considerations (among other things): fair value and what I’ll call proof of least intrusive option.

Fair value consideration in this example would require the government to give manufacturers $ equal to the fair value of IP given away (e.g., profit per dose).

“Proof of least intrusive option” would require gov’t to demonstrate there isn’t an easier or more efficient option outside of taking property. In this example, exporting doses or giving money to poor countries to buy vaccine doses would be a much less intrusive option.

The likely outcome here is US begins exporting vaccines once majority of domestic population has been vaccinated and also donates more money so poor countries can buy doses. Achieves US goal of helping the world without upending centuries of property law precedent.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

28

Re: Biotech Generally, including Regulatory

This is a sell the rumor type of deal going on right now.  I agree with your analysis Alan.

29

Re: Biotech Generally, including Regulatory

When does Govt. support of research justify a "pice of the action"?
When it directly results in new improved indications.
from STAT
=======
Despite millions in research funding, the U.S. has no patent claim on remdesivir

While the U.S. may have put $162 million toward the development of remdesivir, federal authorities aren’t entitled to patent rights on Gilead Sciences’ blockbuster treatment for Covid-19, according to a government report released yesterday.

As STAT’s Ed Silverman reports, a Government Accountability Office investigation concluded that the work of federally employed scientists did not result in any new uses of Gilead’s drug, and therefore the U.S. has no claim on the more than $3 billion the company has made on remdesivir.

The GAO’s report, which was requested by Congress, was meant to settle the contentious debate over the extent to which the federal government should benefit from its years-long contribution into researching uses for remdesivir.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

30

Re: Biotech Generally, including Regulatory

Bristol Myers faces $1B+ tax fight after accidental disclosure reveals alleged offshore patents scheme — report
Kyle Blankenship
Managing Editor
The IRS has battled Big Pharma for years over offshore patent transfer schemes, which it said have bilked the public interest by billions of dollars. Now, loose lips at the agency have revealed its pursuit of another drugmaker — Bristol Myers Squibb — that it says is on the hook for more than $1 billion in back taxes.

Bristol Myers could face nearly $1.4 billion in back taxes the IRS believes it evaded paying as part of a scheme to move its patent rights from the US to an Irish subsidiary and reap the income write-offs, the New York Times reports.

The dispute was revealed as part of an accidental disclosure from the agency last spring, the Times said, which was almost immediately pulled from public view.

advertisement
advertisement
At the center of the IRS’ claims is Bristol’s effort in 2012 to establish an Irish subsidiary as a vehicle to shuttle US drug patent rights over to a country with lower corporate tax rates. In a scheme known as amortization, companies can write off the value of an asset — patents, for instance — from their taxable income over a period of time. Bristol had already written off the value of its patents in the US, the IRS said, and set up a plan to move those rights to Ireland, where that value had not yet been written off.

The outcome? Around $1.38 billion in back taxes, the IRS said.

“Bristol Myers Squibb is in compliance with all applicable tax rules and regulations,” the company said in a statement. “We work with leading experts in this area and will continue to work cooperatively with the IRS to resolve this matter. Beyond that, we don’t comment on ongoing regulatory matters.”

The scheme was lucrative, the Times reported. The company’s effective corporate tax rate was -7% in 2012 compared with 25% in 2011. The difference was so stark that analysts reportedly quizzed executives on the disparity during a Q4 2012 earnings call — questions that Bristol refused to answer.

Meanwhile, Bristol Myers’ plan reportedly got a sign-off from two corporate auditors, PwC and White & Case, in late 2012. Both firms sent lengthy letters to Bristol essentially clearing the scheme — even after Merck had been cited by the IRS back in 2006 for a similar offshoring effort that saved it $1.5 billion in federal taxes over 10 years.

In that case, Merck offloaded its US patents to a Bermuda subsidiary, which it then turned and paid for access to those patents, the IRS said. The amortization scheme wasn’t even unique to Merck: Other big-name drugmakers such as J&J, Pfizer and Abbott have all been implicated in similar plans in the past.

In a 2018 report, Oxfam estimated that offshoring schemes were saving those four drugmakers around $2.3 billion per year in US federal taxes. In total across nine developing countries including the US, Oxfam estimated the firms were writing off around $3.7 billion per year.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

31

Re: Biotech Generally, including Regulatory

ACAD got an unexpected CRL (rejection) letter from the FDA and was own $4 to about $20. In March they were TWICE the current price, but plunged for a reason I do not know. yet.

Does anyone own this stock?

Here is ow ACAD explains the problem, saying the FDA went back on their promise.
https://ir.acadia-pharm.com/static-file … e5048e4153
I read a bit of this and concluded the FDA told them their stratified sample was inaccurate so ALL their data was not suitable in that statistical test of significance would be meaningless because it is on a sample of patients who were not chosen correctly.

I have not completed my study of this so my conclusion is tentative. They do quote the FDA in the link, but not in full.

I would have expected a larger decline on this news but perhaps the March decline let most of the air out of the balloon early.

Anyone follow this, please share what you know.
This is lso related to Omeros as I mentioned on Monday morning, because it is being blamed on the FDA being picky and looking for reasons to reject BLAs. It doesn't look like that to me, so far.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

32

Re: Biotech Generally, including Regulatory

It makes it seem that the likelihood or certainty of the CRL leaked into the market way ahead of time.  Just goes to show that some declines are based on information, while others are noise such as with games by shirts. How to tell one from the other is the question

33

Re: Biotech Generally, including Regulatory

enthusd wrote:

with games by shirts. How to tell one from the other is the question

the other team are skins.
and they have skin in the game.
smile

34

Re: Biotech Generally, including Regulatory

Haha, plenty of games to go around. Now if I could only avoid spelling mistakes on my phone.

35

Re: Biotech Generally, including Regulatory

Actually the FDA warned them they had a problem with their trial in March and THAT caused the initial meltdown.

IMO it was a faulty stratification of the sample of the variety of dementias.
Invalidates all the results.

I suppose they could add selected patients but that is usually not allowed (changing after the study is completed).

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

36

Re: Biotech Generally, including Regulatory

April 5, 2021 07:13 AM EDTUpdated 01:55 PM Regulatory
Acadia preps for battle as the FDA rejects a potential blockbuster application
John Carroll
Editor & Founder
The rolling argument about whether or not the FDA is getting tougher this year just got a fresh spur in favor of the affirmative.

Just a few weeks after the FDA signaled trouble for Acadia’s blockbuster pitch to expand the dementia franchise for Nuplazid, the biotech is back this morning with news that the FDA has handed over a CRL. And execs seem ready to throw down over the rejection with regulators as soon as possible, as a slapdown here would likely require at least one major trial to cure the FDA’s objections.


Billy Dunn
According to the biotech, regulators in the Division of Psychiatry supervised by Billy Dunn are reneging on their deal to analyze a broad patient population suffering from dementia-related psychosis, basing the rejection on failures in certain subgroups “and insufficient numbers of patients with certain less common dementia subtypes as lack of substantial evidence of effectiveness to support approval.”

No safety issues were raised but:

The Division also stated in the CRL that it considers the Phase 2 Alzheimer’s disease psychosis study -019, a supportive study in the sNDA filing, to not be adequate and well controlled, citing that it was a single center study with no type I error control of secondary endpoints in which certain protocol deviations occurred. The Company believes these observations impact neither the positive results on the study’s primary endpoint, nor the study’s overall conclusions of efficacy.

“Acadia stands behind the robustly positive results from the pivotal Phase 3 HARMONY study and the prospectively agreed trial design and criteria for establishing efficacy in DRP. Over the entire course of the review, the Division did not raise any concerns regarding the agreed upon study design, including the issues raised in the CRL,” said Steve Davis, CEO of Acadia. “We will immediately request a Type A meeting to work with the FDA to address the CRL and determine an expeditious path forward for the approval of pimavanserin in DRP.”

Acadia had been cruising along to what a number of analysts had concluded was an approval worth a couple billion dollars a year when execs stunned investors several weeks ago with the news that the FDA wasn’t willing to negotiate the label. That’s been cited among observers who feel that recent indications from the FDA signal a broader toughening on NDAs, though every move to support that argument is often followed by a thumbs-up on a critical issue from regulators.

This is the same FDA group that drew fire from critics for working hand in glove with Biogen on the aducanumab application, offering support right through a hostile panel review that provided near unanimous opposition.

Paul Matteis at Stifel offered a quick assessment, noting:

(P)imavanserin’s efficacy in the relaspe prevention trial was most robust in the Parkinson’s sub-population, and one could argue that this is the primary reason why the trial was stopped at the interim. We had figured this detail could be overlooked since the data for ADP was still pretty good (14 relapses placebo, 8 drug), but data in some of the other rarer subgroups is less consistent, and perhaps if one is not a believer in the “acute” ph2 trial the small numbers issue with the ph3 become more prominent. Bottom line: our view is that ACAD will likely need to run one or even two more trials here, and while pimavanserin has always seemed to be a very safe drug, it’s efficacy is at the low end of the range for atypical antipsychotics, which makes any future trial (especially one that needs to demonstrate clear acute efficacy in a placebo-controlled manner) fairly risky, in our opinion.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

37

Re: Biotech Generally, including Regulatory

More biotech stupidity/cheating hurts shareholders and gives the whole sector a bad name.
========================
UPDATED: FibroGen shares take a beating as CEO reveals safety data manipulation for its blockbuster contender roxadustat
John Carroll
Editor & Founder
FibroGen $FGEN backtracked on some of the key safety data used to boost hopes for their anemia drug roxadustat, eliminating its claim of superiority over ESAs for a large segment of the patient population. And investors quickly crushed its shares in retaliation for the surprise discrepancies.

According to CEO Enrique Conterno, FibroGen execs became “aware” that their analysis included post hoc changes to stratification factors used to assess the hazard ratio of their drug — essentially manipulating the data to enhance how much the therapy reduced key risks for patients and sharing that false profile with investors and regulators. Using pre-specified stratification factors raised the risk on key safety endpoints, which revolved primarily around MACE, a composite endpoint of all-cause mortality, stroke, and myocardial infarction.

“As members of senior management were preparing for the upcoming FDA Advisory Committee meeting, we became aware that the primary cardiovascular safety analyses included post-hoc changes to the stratification factors,” said Conterno in a statement. “While all of the analyses set forth below, including the differences in the stratification factors, were included in the NDA, we promptly decided to clarify this issue with the FDA and communicate with the scientific and investment communities.”

That presents a potentially huge problem along with the ethics scandal built into these events:

While these hazard ratios remain below 1.0, based on these analyses we cannot conclude that roxadustat reduces the risk of (or is superior to) MACE+ in dialysis, and MACE and MACE+ in incident dialysis compared to epoetin-alfa.

Incident dialysis patients make up a significant group in this field.

The company — which is partnered with AstraZeneca and Astellas — went on to say that the new, previously unreleased data “do not change the company’s assessment that roxadustat is comparable to placebo in non-dialysis dependent patients and to epoetin-alfa in dialysis dependent patients using MACE to measure cardiovascular safety.”

AstraZeneca did not immediately respond to a query from Endpoints News.

The idea that the company has incorrectly reported data for 2 years clearly didn’t sit well with analysts. Geoff Porges was among many wondering how this will likely play out now.

All these disclosures, and management’s apparent lack of information about the basis for the prior presentation before last week, will likely leave many investors wondering whether roxa has any chance of approval, and even whether the company’s partnership with AstraZeneca, and their respective management teams, will survive.

Michael Yee at Jefferies noted:

Bottom line is while overall the stats don’t have a huge change and the co believes conclusions still support an FDA approval – we believe the fact that Incident Dialysis is no longer “statistically” superior – is a material change to the profile and one of the key prior advantages for pts. This lowers peak sales and lowers Pos for approval (risk/benefit) and our PT. We believe new disclosures to the Street on statistics of the CVOT safety analyses do not give us or investors confidence about the rest of the filing (they said an internal review of all other data is underway…) and probability of approval.

Shares quickly plunged 30% on the news of the fiasco. That eviscerated about a billion dollars of its market cap.

That stock avalanche followed another crunch a few weeks ago when FibroGen execs revealed a surprise FDA adcomm meeting, which no one had expected.

As we reported at the time, FibroGen has long believed that mimicking the effect of high altitude could stimulate the production of red blood cells and increase hemoglobin level without triggering the cardiovascular side effects plaguing patients taking erythropoiesis-stimulating agents, the current standard of care. So any change in the safety profile could dramatically impact its future on the market, provided the company makes it that far.

“We’re taking this very seriously,” Conterno said on his Tuesday evening call with analysts. But there remain a number of important questions that have yet to be answered, or even asked. Who was responsible for the post hoc changes that amped up the safety profile, allowing FibroGen to make a case the actual data don’t back up? And what happens to those individuals now?

Data manipulation — changing data to make a drug look better than it is — is absolutely forbidden, as anyone at Novartis could tell you after the AveXis fiasco. And FibroGen CEO Conterno — named to his position 15 months ago following the sudden death of company founder Thomas Neff — still has considerable explaining to do.

Right after the data switch-up was revealed, the biotech reported that their surprise adcomm was slated for July 15. That will be closely watched as analysts search for more nasty surprises that could alter the profile of a drug many considered a likely blockbuster for years.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

38 (edited by Alan Robert Ross 2021-04-07 09:23:49)

Re: Biotech Generally, including Regulatory

Referring to the story above:
If the FDA was not being more careful and strict before, the latest flurry of bad experimental design and fudging data is likely to make the FDA double  check things and be more on the lookout for companies gaming the system.

That is NOT going to help Omeros get a faster approval.
If anything, it will slow it down.

The perps must be severely punished to deter the chance that new drugs without good data get approved because the FDA has been scammed.
==========
from STAT
Fibrogen has been touting false safety data for years

In a shocking development yesterday, Fibrogen said it had been touting false heart-safety data on its experimental anemia pill for at least two years, a revelation that further clouds the drug’s future in the eyes of the FDA.

As STAT’s Adam Feuerstein reports, Fibrogen made after-the-fact changes to how it analyzed cardiovascular safety data across six clinical trials of approximately 8,000 patients. In that analysis, which Fibrogen has repeatedly presented, the risk of cardiovascular trouble with Fibrogen’s drug was statistically comparable between roxadustat and either a placebo or a currently approved anemia injection used as a control. But under the study’s pre-specified analysis, disclosed for the first time yesterday, the results looked noticeably worse.

How could something like this happen? Who’s responsible for it? And how did the company only find out now? Fibrogen didn’t say.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

39

Re: Biotech Generally, including Regulatory

Who’s to blame for the Fibrogen disaster?
STAT
Now that the dust has somewhat settled on Fibrogen’s surprising revelation about false data, how do we apportion blame for a bizarre and damaging mistake?

The incorrect data, first presented in 2019, predates the majority of Fibrogen’s C-suite, including the CEO, CMO, and CSO. That creates a strange situation. For one, it’s perhaps a retroactive red flag that their predecessors all left the company in the run-up to an all-important FDA submission. And, more pertinent, it’s also arguably exculpatory for the new administration. Conducting a detailed review of already-presented data is arguably a micromanagerial endeavor for someone in the C-suite.

That being said, there’s a compelling theory of executive compensation stating that top executives get paid a lot of money to take the blame when the company does something wrong, regardless of the explanation. And to SVB Leerink analyst Geoffrey Porges, the whole scandal raises questions of “whether the company’s partnership with AstraZeneca, and their respective management teams, will survive.”

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

40

Re: Biotech Generally, including Regulatory

The more I read about AstraZeneca, the more I want to stay away from it.  My brother-in-law left employment there several years ago; I was surprised by the strong language he used to describe the company in negative terms.  He left for specific reasons.  He's loving it at Sanofi right now....

This Fibrogen affair is a disaster for the company, but OTOH the system has worked to prevent a drug from advancing, that did not deserve to advance.

41

Re: Biotech Generally, including Regulatory

Today's Horror story

April 8, 2021 05:19 PM EDTUpdated 3 hours ago Regulatory
Trouble brewing for Provention? Shares crater after FDA says their teplizumab drug profile doesn't match that from former owner Eli Lilly
Max Gelman Associate Editor
Endpoints

Provention Bio acquired the former Eli Lilly drug teplizumab a few years ago, hoping to bring the experimental drug back from the dead in type 1 diabetes after it flamed out in a pivotal diabetes study undertaken by the Big Pharma more than 10 years ago. But Provention is seeing troubled waters ahead following new feedback from the FDA last week.

The biotech revealed Thursday that regulators expressed concerns over the candidate’s pharmacokinetics data and comparability contained within Provention’s data package submitted to the FDA. Regulators are asserting the PK profiles of the drug manufactured by Lilly and by Provention are not the same, and asked for more data on April 2.

In an ominous sign, regulators have said that such concerns mean it is not ready to start post-marketing and label discussions, per Provention’s accounting of the feedback. Such comments are eerily similar to Acadia’s dementia program, which received a CRL just last week.

Provention responded to the feedback Thursday noting it is “willing to discuss these issues.” Regulators had scheduled a May 27 adcomm to discuss the experimental drug, which will continue as planned.

“We believe in the comparability of the drug product produced by our partner AGC biologics with [the] Eli Lilly manufactured product,” CEO Ashleigh Palmer said in a statement. “We look forward to working closely with the Agency to address its additional data requirement, so we can deliver teplizumab to patients as soon as possible.”

Investors responded sourly to the news after Thursday’s bell, with Provention $PRVB shares crashing almost 40%.

Lilly had developed the drug several years ago and Provention acquired it in 2018, taking over everything from patents to drug supply and cell lines. But when the FDA looked at the comparative PK data, they said they did not match.

The biotech noted that the teplizumab they tested was manufactured by the CDMO AGC Biologics. An investigational anti-CD3 monoclonal antibody, teplizumab is being evaluated for the delay or prevention of type 1 diabetes in at-risk individuals. The program received breakthrough status in 2019.

Teplizumab had a long history at Lilly, lingering in limbo for years after the pharma decided to punt following a pivotal failure in diabetes. But its scientific discoverer, biotech entrepreneur Jeffrey Bluestone, found new believers at Provention, who picked up rights to the drug and quickly made their way back to the FDA.

Provention ultimately used the program as its lead drug when it debuted with a $64 million IPO back in 2018.

Clarification: This story has been updated to clarify the nature of the data package Provention submitted to the FDA.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

42

Re: Biotech Generally, including Regulatory

Got to hand it to the FDA for having caught these discrepancies.  Same as what happened at the vaccine plant here, which made national news.  I wonder if Provention was trying to pull a fast one....

43

Re: Biotech Generally, including Regulatory

Exactly.

Shortsightedly foolish to want an incompetent FDA, especially for honest drug companies that want an FDA approval to convince regulators around the world that their drug does much more good than harm.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

44

Re: Biotech Generally, including Regulatory

I wonder how many people work for FDA doing this kind of work.  I suppose I could look it up.  I envision vast rooms full of people looking at computers, reading, reading, reading all day long, taking notes, referring to this and that.  Millions upon millions of pages of data, everything needs to be checked and double checked.  I wonder if they have computer programs that can "read" submissions and flag something that may not be correct - but still, the workload sounds to me as to be incredibly large.  And complex.  So much of it is statistics, which can be manipulated this way or that way.  It's no wonder that things take so long when it comes to drug development and approval....

45

Re: Biotech Generally, including Regulatory

STAT
The case for virtual inspections

FDA drug inspections are done, by and large, in person. But Covid-19 has made that approach infeasible in most cases over the past year. By September, 85% of inspections were halted in the U.S., and 99.5% were curbed abroad. Now, there’s a push toward making virtual inspections permanent.

Instead of visiting a manufacturing site, the thinking goes, inspectors could read up on the plant’s equipment and capabilities. “If inspectors decide to proceed, the manufacturer’s staff can carry a 360-degree camera around the plant as the inspector tells the producer where to go and when to move closer to something,” write two regulatory policy experts at George Mason University.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

46

Re: Biotech Generally, including Regulatory

Underperformance of healthcare sector masks its return to normalcy: WSJ
Apr. 19, 2021 11:15 AM ETUnitedHealth Group Incorporated (UNH)By: Dulan Lokuwithana, SA News Editor2 Comments
The S&P 500 Health Care index has returned ~6.9% so far this year lagging the ~11.4% gain in the overall index.
The underperformance ‘belies the reality’ that the sector which accounts for ~18% of the national gross domestic product, has mostly returned to normal after months of pandemic-related disruption, argues Charley Grant in an opinion piece on the Wall Street Journal.
With consensus beating Q1 financials from UnitedHealth (UNH -0.1%), the earnings season for the first quarter of 2021 is off to a solid start notes Grant who also points to the strength in the sector’s deal activity.
He cites the regulatory clearance for the merger between AstraZeneca (AZN +2.0%) and Alexion (ALXN +1.3%) and the acquisition of PPD (PPD -0.0%) by Thermo Fisher (TMO -0.6%).
Despite the uncertainty related to key factors such as insurance coverage and drug pricing, the healthcare industry continues to enjoy long-term tailwinds such as an aging demographic.
Highlighting the remarks from Pfizer CEO Alfred Bourla on the need for booster COVID-19 shots, Grant expects the virus impact to last even after the pandemic, benefiting the industry with an additional opportunity in testing, treatments, and vaccines.
The industry valuations are also ‘fairly reasonable’ argues Grant pointing to UnitedHealth and Pfizer which in terms of their 2021 earnings trade at ~21x and ~12x, respectively in line with their historical averages.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

47

Re: Biotech Generally, including Regulatory

Apr 19, 2021,09:34am EST|295,726 views
The Billionaire Scientist Behind The Pfizer-BioNTech Vaccine Has Not Sold A Single Share Of His Booming Stock
Nathan Vardi
Forbes Staff

As the pandemic began to rage a year ago, the biopharma industry responded in unprecedented fashion. Life sciences companies raced to find a solution to the nightmare virus, and promising vaccine efforts soon emerged. The stocks of the companies with the best vaccine candidates soared, and many of their executives rushed to sell shares with a fury that generated scrutiny.

There has been one huge exception. Uğur Şahin, the CEO and scientist behind the first Covid-19 vaccine authorized in the U.S., has not sold a single share of his company’s booming stock during the pandemic, Securities and Exchange Commission filings show.

Şahin’s decision to not sell any of his BioNTech shares stands in stark contrast to the large stock sales of some of the most prominent scientists and entrepreneurs whose biotechnology companies developed vaccines against the virus, particularly Moderna Therapeutics. It also reflects Şahin’s general approach to life and business. He is a CEO who lives in a modest apartment in the German town of Mainz, rides his bicycle to work and doesn’t own a car. He describes himself on his LinkedIn page first and foremost as a translational oncology professor at University Medical Center Mainz. Şahin accepts the financial edifice that surrounds biotechnology innovation—the venture fundraising, IPOs and merger deals. He reportedly learned the business aspects of biotechnology from online videos and reading a Business Plans for Dummies book. But in the end, Şahin is in it for the science and the patients. 

In its hour of greatest need, Şahin emerged from relative obscurity to provide the world with a game-changing vaccine that could save lives and tame the pandemic. As early as January 2020, Şahin became convinced Covid-19 would become a deadly pandemic and pivoted BioNTech to create a messenger RNA vaccine to combat it. He then partnered with U.S. pharmaceutical giant Pfizer PFE +0.3% to develop and supply 3 billion doses of the vaccine to the world by the end of 2021.

The Pfizer-BioNTech vaccine has radically changed Wall Street’s perception of BioNTech. In the months prior to the pandemic, Şahin had come to New York to sell investors on BioNTech’s stock as the company launched its initial public offering by listing on Nasdaq NDAQ +0.7%. At the time, BioNTech was a decade-old company that had yet to develop a single approved product from its mRNA and immunotherapy technologies. Şahin received a cool reception in the stock market, which initially valued BioNTech at $3.4 billion. With the development of the vaccine, however, BioNTech’s stock has risen by 900% since its 2019 IPO and today the company is valued at $37 billion. BioNTech now expects to generate $11.5 billion of revenue under deals for the vaccine that have already been struck. On paper, Şahin’s BioNTech stake is worth $6.1 billion. 

Filings made to the Securities & Exchange Commission in February and March show that Şahin controls 41.66 million BioNTech shares, a 17% stake, through Medine GmbH, a limited liability company he solely owns. Medine holds a relatively small number of shares for “a former colleague” and transferred 27,540 shares held for other colleagues to their beneficiaries under trust arrangements late last year. But amid this stock transfer, a securities filing in February made sure to point out that “Neither Medine GbmH nor Prof. Ugur Sahin, M.D. has sold any ordinary shares since February 13, 2020,” the eve of the pandemic. Such worded disclosures are not common in securities filings. Şahin, who declined to comment, appears to have wanted people to understand he wasn’t selling any BioNTech stock. 

Last year, executives and directors of companies like Moderna, Pfizer and Novavax NVAX -6% realized stock gains linked to investor enthusiasm around the vaccines by selling nearly $500 million of stock, the Wall Street Journal reported. The selling has continued this year. Moderna CEO Stephane Bancel has sold more than $150 million of Moderna stock since the start of the pandemic. He still owns just under 8% of Moderna. Tal Zaks has sold more than $100 million of Moderna stock, nearly all the shares he had accumulated since becoming Moderna’s chief medical officer in 2015.

Moderna has consistently explained that all executive stock sales are made through preset trading plans established under securities regulatory rule 10b5-1, which legally allows corporate insiders to periodically sell a predetermined number of shares, often tied to certain share price targets being attained.

In November, Pfizer CEO Albert Bourla sold about 60% of his stock in the company for about $5.6 million under a preset 10b5-1 plan. The sale came on the same day that Pfizer announced key clinical results showing that its Covid-19 vaccine was more than 90% effective. Bourla’s share sale had been authorized in February 2020 and updated in August. A few days later, Jay Clayton, then chairman of the SEC, suggested corporate executives not trade in their company’s stock immediately after preset trading plans are established. At a Senate hearing, Clayton called for a “cooling-off” period, but did not specify the length of such an interval. “Whether that’s four months so you cover a full quarter, or it’s six months—I can make arguments for either,” Clayton said. There was also some concern the insider stock sales by Moderna and Pfizer might undermine the public perception of their vaccines as crucial public health tools. 

For his part, Şahin has not sold any BioNTech stock in the last 18 months. The post-IPO lockup of his BioNTech shares expired around the start of the pandemic, securities filings show, and he was free to sell. As a result of hanging on to all the shares, ironically, he is for now much richer—at least on paper—given the continued surge of BioNTech’s stock price. He certainly believes the company’s cutting-edge technology will lead to the development of therapies and vaccines for other diseases.

“Our way of developing our technologies is not based on the idea of a single-trick pony,” Şahin told investors on a Wall Street call in March. “Rather, our goal from the very beginning was to build a novel industrial approach for precision pharmaceuticals that can address medical need in multiple disease areas.”

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

48

Re: Biotech Generally, including Regulatory

STAT

The FDA is still open for business

Amid all the sturm und drang about a suddenly conservative FDA, what would have been minor box-ticking news was worth $200 million in market value to a small biotech company.

Yesterday, Axsome Therapeutics said the FDA accepted the approval application for its depression treatment and agreed to a priority review, which shortens the process from 10 months to six months. In process terms, that’s not surprising. Axsome’s therapy met its goals in a Phase 3 study, and the bar for accepting an application is naturally lower than the one for approving a drug.

And yet Axsome’s share price rose more than 10% on the news. That’s likely a reflection of the widespread, if loosely founded, perception that the FDA has been cracking down on drug companies seeking new approvals. A series of surprising decisions have spooked investors and raised the perceived risk facing any company making its case before the agency. Whether those are isolated cases or signs of a trend is impossible to say, but Axsome’s comparatively predictable FDA experience suggests there hasn’t been a sea change.

********************************
Drug pricing might have fallen out of Biden’s agenda

Tomorrow, President Biden will give his first address to a joint session of Congress, laying out his sweeping policy proposals for infrastructure and economic recovery. And Democrats have no idea whether he’ll talk about drug pricing.

As STAT’s Rachel Cohrs reports, the 11th-hour confusion is reminiscent of the intra-party infighting that doomed drug-pricing measures under President Trump, who vocally supported them. This time, it’s the Democrats who can’t seem to get on the same page, with powerful lawmakers unsure whether the president’s stated commitment to lowering drug prices will make it into his early-term policy priorities.

That’s alarming to those advocating policies to lower prescription drug costs, who worry that putting drug prices on the back burner will waste a precious political moment in which Democrats have a chance to attach meaningful legislation to a large, fast-moving bill.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

49

Re: Biotech Generally, including Regulatory

This is a 2018 article.
===============
Biotech boom powers analysts to $4M-a-year salaries: WSJ
by Nick Paul Taylor | Aug 29, 2018 9:22am

The $3 million-plus pay packets set biotech apart from other industries, where around $1 million a year is typical.
The biotech boom is having a big effect on the pay packets of equity analysts, according to The Wall Street Journal. Driven by a desire to show they understand biotech, banks are reportedly paying analysts up to $4 million a year, several times more than the typical salary in other industries.

Analysts assess companies and write reports that help investors gauge their prospects and interpret news. Their work was separated from that of their colleagues in banking in the 2000s amid reports analysts were hyping companies to drum up business. Yet a perceived link between the prestige of an analyst and a company’s ability to win banking business survived the regulatory clampdown.

“To attract business on financing side, banks must have high-quality analysts respected by the buy side … who will be fair, credible and insightful,” Citigroup’s Yigal Nochomovitz told the WSJ.

Nochomovitz is reportedly among the analysts to benefit from those dynamics. Citigroup is said to have given Nochomovitz a big raise to keep him at the company. Matthew Harrison also reportedly secured a sizable pay bump from Morgan Stanley after fielding offers from rival firms.

Morgan Stanley knows there is a real risk an analyst will take up a better offer. The bank lost Andrew Berens to Leerink Partners earlier this year. Other recent moves include Alethia Young’s switch from Credit Suisse to Cantor and Michael Yee’s arrival at Jefferies. Yee reportedly left RBC Capital Markets after Jefferies offered $4 million a year. The other analysts are said to have received “lucrative” offers.

The $3 million-plus pay packets set biotech apart from other industries, where around $1 million a year is a typical salary. The potential for analysts to burnish the image of their employer in biotech circles and thereby help win business from the industry may account for some of the difference. Another possibility is that the complex science and unproven assets of biotechs make them harder to analyze than companies in other industries, which typically IPO as established businesses.

original content ©2020 to 2021 by Alan Robert Ross
Founder, Trust Intelligence
The foregoing is not investment advice.

50

Re: Biotech Generally, including Regulatory

BioNTech (BNTX) – The drug maker beat estimates on both the top and bottom lines for the first quarter, helping its stock surge by 8.7% in premarket action. BioNTech also said there’s no current evidence that points to the need to adapt its Covid-19 vaccine to emerging variants of the virus, although it is prepared to do so if necessary.