51

Re: Biotech Generally, including Regulatory

From Endpoints

Dutch agency says Zolgensma price should be cut in half
Novartis’ Zolgensma drug for spinal muscular atrophy is notorious for being the most expensive medicine in the world at about $2.1 million. But the Dutch are trying to change that.

The Zorginstituut, the national health care institute of The Netherlands, is pushing to slash Zolgensma’s price in half, per a statement released Monday. The institute noted that the price point does not reflect the drug’s effectiveness, saying there is too little evidence the drug works to cost its current price.

It has recommended that the Dutch government not cover the costs of the medicine unless this price cut is met.

“The Zorginstituut ensures that our care is and remains good and affordable, all this to only spend our money for healthcare on valuable treatments that are known to actually work,” the agency said in a statement. “We ultimately make these complicated but necessary choices for and on behalf of 17 million Dutch people, so that everyone continues to have access to good and affordable care in the future.”

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

52

Re: Biotech Generally, including Regulatory

Sounds like a short candidate (have not checked beyond this)
from STAT

The biotech stock outperforming Bitcoin

Fluctuations on the order of 1,800% are typically reserved for ironic cryptocurrencies, New Jersey diners, and scams. But Curis, a biotech company that has been trying and failing to invent drugs since 2000, has posted a one-year return that outpaces GameStop and Bitcoin.

The company, which traded for 88 cents a share 12 months ago, rose to about $16 yesterday on the news that its investigational treatment for blood cancer posted promising results in a small, open-label study. Curis’s drug, CA-4948, reduced the bone marrow cells that drive blood cancer for eight of nine evaluable patients, and there were four objective responses that included two complete remissions.

The results, presented at the European Hematology Association’s annual meeting, are still early. Six patients in the study are yet to be evaluated, and it’ll be key to track how many patients relapse over time. But as biotech turnarounds go, Curis’s one-year trip from being a penny stock to commanding a $1.4 billion valuation stands alone in 2021.

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

53

Re: Biotech Generally, including Regulatory

STAT

Pharma could have its own Colonial Pipeline moment

Nearly 10% of drug makers are highly susceptible to the kind of ransomware attack that shut down a major gas supplier earlier this month, according to a cybersecurity analysis.

As STAT’s Ed Silverman reports, the consultancy Black Kite took a look at pharmaceutical cybersecurity and found that 43% of the industry’s data management vendors were vulnerable to an attack, and half of the 200 drug companies it considered had more than 1,000 employees whose usernames and passwords were exposed on the deep web in the last 90 days.

The most at risk are mid-size companies, according to Black Kite, because they have enough revenue to be attractive targets but lack the security infrastructure found in major pharma.

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

54

Re: Biotech Generally, including Regulatory

2 articles
========
FDA’s inspection backlog: GAO raises concerns as delays mount
Posted 09 March 2021 | By Michael Mezher

FDA’s inspection backlog: GAO raises concerns as delays mount
Mary Denigan-Macauley, PhD

For decades, the Government Accountability Office (GAO) has raised concerns about the US Food and Drug Administration’s (FDA) inspections program. Now, a year after the agency halted most of its foreign and domestic inspections due to the COVID-19 pandemic, GAO warns that the ensuing backlog could take years to clear.

In a new report and testimony before the House Appropriations Subcommittee on Agriculture, Rural Development, Food and Drug Administration, and Related Agencies, GAO Health Care Director Mary Denigan-Macauley, PhD, said that the agency needs to come up with a plan to address the looming inspections backlog and tackle some of its longstanding issues.

FDA conducts three types of inspections: preapproval inspections, routine surveillance inspections and for-cause inspections. For routine surveillance inspections, FDA considers inspections of sites that have never been inspected and those that have not been inspected within five years to be mandatory and prioritizes its remaining resources on inspecting establishments based on risk.

Since declaring that it would halt all but mission-critical inspections, FDA has in part relied on alternative tools, such as inspection reports from foreign regulators, records requests, and product sampling to complement its oversight activities. The agency also has resumed some of its foreign and domestic inspections activities, albeit on a very limited basis.

“While these tools can provide useful information, most do not substitute for an inspection, and FDA will face a backlog as it was unable to complete more than 1,000 of its planned inspections for FY 2020,” Denigan-Macauley said. In January, GAO called on FDA to assess its alternative inspections tools and come up with a plan to deal with the backlog. (RELATED: Inspections: GAO calls on FDA to plan for backlog, review alternatives, Regulatory Focus 1 February 2021).

GAO’s report, submitted as testimony to the hearing, finds that “if inspections continue to be postponed, the backlog of mandatory inspections this will create could both extend the maximum interval between inspections beyond FDA’s five-year policy and reduce the resources available in fiscal year 2022 for inspecting the other highest priority establishments identified by its model.”

GAO_2.png

“It is a daunting job, and it’s not something that can just be deemed from a document review,” Denigan-Macauley said, noting that inspectors are able to make observations about cleanliness and compliance practices while on-site that would be difficult to glean from alternative tools.

“As it stands now, we remain very concerned that [FDA is] not going to have additional resources to be able to reduce that backlog, so perhaps they have to come up with a different way of looking at what they consider the most important ones to get done first,” they said.

Since making its recommendations in January, GAO notes that FDA has still not fully assessed how its alternative tools can be used to supplement or replace inspections going forward. FDA officials are in the process of conducting that assessment and are reportedly considering the use of remote video and other remote and live interactions with establishments and records to evaluate drug manufacturing operations.

While FDA has said the postponed inspections have not had a significant impact on approval decisions, Denigan-Macauley said that manufacturers are growing increasingly concerned as time goes on. “They said that while it’s true that the impact may not be felt immediately because the inspections for preapprovals are done several months prior, as the pandemic goes on it’s going to have more and more of an impact,” Denigan-Macauley said.

Despite FDA’s insistence, there have been several cases of companies receiving complete response letters (CRLs) or the agency deferring action on drugs and biologics in part due to its inability to conduct on-site inspections. (RELATED: FDA details review timelines as facility assessment-related CRLs pile up, Regulatory Focus 22 December 2020).

Vacancies

Aside from pandemic-related issues, Denigan-Macauley said that FDA’s biggest challenge is to hire and train investigators, something that has challenged the agency for years. (RELATED: Foreign Drug Inspections Decline as FDA Hiring Struggles Continue, Regulatory Focus 10 December 2019)

“Most importantly, they have got to figure out how to fill their vacancies. Even if you poured more money at them, I don’t think that would solve the problem because they’re having a challenge getting investigators to even do the work,” Denigan-Macauley said.

Despite FDA’s testimony last June that it would fill all investigator vacancies by the end of the year, there were still open investigator positions as of December, Subcommittee Chairman Sanford Bishop (D-GA) said, questioning whether hiring and training new investigators was a priority for FDA. (RELATED: FDA defends its oversight of foreign drugs amid Senate, GAO criticism, Regulatory Focus 3 June 2020).

“Something isn’t quite working and that’s what we’re still trying to figure out. Because there are still vacancies year after year,” Denigan-Macauley replied, adding that GAO has “serious concerns that the pandemic will exasperate … fulfilling those vacancies, particularly for those working in the foreign offices because a lot of individuals are now fearful of traveling and being stationed in India and China.”

While more money might not necessarily solve all of FDA’s inspection woes, the agency is set to get some additional funding for inspections that were delayed or cancelled due to the pandemic in the COVID-19 relief bill expected to pass in the House on Wednesday. (RELATED: House committee proposes $500M in COVID funding for FDA, Regulatory Focus 10 February 2021).

Resuming inspections

FDA faces significant challenges in conducting the inspections it has already postponed and the new inspections it will need to conduct to support product approvals and keep pace with its surveillance inspections policies.

The agency announced in July 2020 that it would resume domestic inspections using a rating system to determine whether it was safe to conduct inspections in a particular county. (RELATED: FDA looks to resume domestic inspections this month, Regulatory Focus 10 July 2020). “According to FDA’s area rating data, as of 3 December 2020, conditions were appropriate for conducting routine surveillance inspections in 49 US counties, with regulatory activity limited to mission critical inspections only in the more than 3,000 remaining counties,” according to the GAO report.

From March to 1 October 2020, the agency conducted just three foreign and 52 domestic inspections, compared to hundreds of foreign and domestic inspections it would typically conduct over the same period.

More recently, FDA restarted its inspections programs in China and India, though the agency “has not set a date for resuming routine foreign surveillance inspections in all countries.”

According to GAO, FDA conducted nine preapproval inspections in China from 26 October 2020 to 14 January 2021 but did not conduct any routine surveillance inspections during that time. In India, FDA conducted just two inspections through 25 February 2021, after resuming operations in January.

The GAO report also gives insight on the regulatory actions FDA has taken against foreign drug manufacturers based on the use of its alternative tools. Between March and December 2020, FDA placed 64 foreign establishments on import alert, one based on issued identified in a foreign inspection report, nine for refusing records requests or for deficiencies identified during records reviews and 54 based on product sampling.

In addition to the challenges posed by COVID-19, Denigan-Macauley told members of the subcommittee that FDA’s foreign inspections program faces “unique challenges” that have gone unaddressed for years.  Those challenges include the fact that the agency typically provides advanced notice of inspections to foreign establishments, compared to its practice of conducting unannounced inspections in the US, and the fact that the agency relies on the establishments themselves for translation services. During the COVID-19 pandemic, FDA has resorted to preannouncing all inspections as a safety measure.

“FDA continues to rely on the establishments for translations and still provides up to three months advance notice for most [foreign inspections], giving manufacturers the chance to fix problems before an investigator even arrives,” Denigan-Macauley said, adding that investigators have little flexibility to extend foreign inspections and are often tasked with inspecting massive facilities on their own.

Pressed by Subcommittee Ranking Member Jeff Fortenberry (R-NE) to give the agency a letter grade for its inspections program, Denigan-Macauley said, “It’s certainly not the failing grade that we would have given it two decades ago,” opting to give the agency a lackluster grade.

“The fact that inspections are going down that they have continued oversight problems overseas, I think at best we could say they are at a C,” Denigan-Macauley said.
===========

Gilead Declines 'Rare Disease' Status For Experimental Coronavirus Drug
March 25, 20203:48 PM ET
SYDNEY LUPKIN


After the Food and Drug Administration granted Gilead Sciences orphan drug status for its experimental drug remdesivir on Tuesday, Gilead asked that the agency rescind that status Wednesday.
Bloomberg/Bloomberg via Getty Images
In a surprising turnabout, drugmaker Gilead Sciences asked the Food and Drug administration on Wednesday to rescind orphan status for remdesivir, the company's experimental coronavirus treatment.

The agency granted this status to remdesivir on Monday, prompting a backlash from public health and consumer advocates. Orphan drug designation is intended to spur development of drugs for rare diseases by bestowing drugmakers with tax breaks, FDA fee waivers and seven years without generic competitors.

Although this isn't the first time a company has asked for an orphan designation to be rescinded, it's rare, says James Love, director of Knowledge Ecology International, a non-profit public interest group.

"Gilead must have been feeling the heat," Love says. "I think it's embarrassing to take something that's potentially the most widespread disease in the history of the pharmaceutical industry and claim it's a rare disease."

For its part, Gilead said it could proceed to develop the drug quickly without the special regulatory status.

"Gilead is confident that it can maintain an expedited timeline in seeking regulatory review of remdesivir, without the orphan drug designation," the company wrote in a statement. "Recent engagement with regulatory agencies has demonstrated that submissions and review relating to remdesivir for the treatment of COVID-19 are being expedited."

To get orphan status, a drug company must show its drug serves a population of fewer than 200,000 people in the United States, "at the time of the submission of the request for orphan-drug designation." Gilead's latest statement indicates that this was early March.

Domestic COVID-19 cases are expected to surpass that threshold. As of Wednesday afternoon, there were more than 60,000 confirmed cases of COVID-19 in the U.S.

Orphan drugs are among the most expensive in this country, in part because they are shielded from from generic competition for longer than other prescription medicines.

Knowledge Ecology International had been preparing to file a citizen petition to push the FDA to reverse its decision on remdesivir, Love says. Now, it won't have to.

"The facts and circumstances should have given the FDA the authority to have rejected the application, which they did not do," the says.

The FDA confirmed to NPR that it has "received and processed" Gilead's request.

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

55

Re: Biotech Generally, including Regulatory

May 25, 2021
7:58 PM -05
Healthcare & Pharmaceuticals
U.S. FDA may not review new COVID-19 vaccine EUA requests during pandemic
Reuters

The U.S. Food and Drug Administration said on Tuesday it may decline to review and process new emergency use authorization (EUA) requests for COVID-19 vaccines for the rest of the pandemic, if a company has not already begun discussions.

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

56

Re: Biotech Generally, including Regulatory

https://www.sec.gov/litigation/litrelea … r25099.htm

SEC Charges Medical Investigator with Insider Trading On Confidential Information from Clinical Trial
Litigation Release No. 25099 / May 27, 2021
Securities and Exchange Commission v. Mohammed A. Bari, No. 21-civ-00999 (S.D. Cal. filed May 26, 2021)
The Securities and Exchange Commission today announced settled insider trading charges against Dr. Mohammed A. Bari, of Rancho Santa Fe, California, for his trading in the stock of a drug company after learning that the company was on the brink of announcing positive clinical trial results.

According to the SEC's complaint, Bari was a medical investigator for the clinical trial of KarXT, a drug developed by Boston-based Karuna Therapeutics, Inc. for treating patients with schizophrenia. The complaint alleges that, in mid-November 2019, Karuna informed Bari that KarXT had proven safe and effective in the trial, and that Karuna planned to announce these results to the public within a few days. Karuna's management allegedly described the KarXT trial results to Bari as a significant milestone for the company. According to the complaint, Bari began placing orders to purchase Karuna common stock within hours of learning this information, and ultimately acquired more than 1,600 shares. The complaint alleges that Karuna publicly announced the positive results on November 18, 2019, and its stock price increased by more than 440 percent, earning Bari profits of nearly $120,000 by trading in advance of that announcement.

The SEC's complaint alleges that Bari violated the antifraud provisions of Section 10(b) of the Securities Exchange Act of 1934 and Rule 10b-5 thereunder. Without admitting or denying the allegations, Bari agreed to be permanently enjoined from violating these provisions of the securities laws and to pay a penalty of $238,434. The settlement is subject to court approval.

The SEC's investigation was conducted by Lance Jasper and supervised by Spencer Bendell in the Los Angeles Regional Office. The SEC acknowledges the assistance of the Financial Industry Regulatory Authority.

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

57

Re: Biotech Generally, including Regulatory

FDA has allowed ~1000 clinics in the USA to function and administering unapproved stem cell drug treatments?
Some causing blindness?

https://endpts.com/eye-injections-causi … or-action/

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

58

Re: Biotech Generally, including Regulatory

https://www.reuters.com/business/health … 021-05-31/

May 31, 2021
3:48 PM -05
Healthcare & Pharmaceuticals
Even after U.S. shift, opponents resist COVID-19 vaccine patent waiver
Emma Farge

A deal on an intellectual property waiver for COVID-19 vaccines at the World Trade Organization (WTO) was no closer to acceptance on Monday despite Washington's backing, due to expected scepticism about a new draft, sources close to the talks told Reuters.

Negotiations reopened at the WTO on Monday, focused on a highly anticipated revised draft submitted by India, South Africa and dozens of other developing countries last week.

A surprise U.S. shift earlier this month to support a patent waiver heaped pressure on remaining opponents like the European Union and Switzerland that are home to numerous drugmakers. But Monday's discussions - the 11th session since the initial waiver proposal in October - failed to achieve a breakthrough.

The waiver's main backers presented their new draft in Monday's private WTO meeting, allowing key players to give their first official feedback on its contents.

The meeting is critical because it will determine if the talks will advance to "text-based negotiations" as sought by director-general Ngozi Okonjo-Iweala.

A Geneva trade official said the proposal to start text-based discussions "gained traction" on Monday, including from the United States, which said it was open to discussion on any proposal that could boost vaccine production and delivery.

It did not openly support the revised text, but said it was analysing it.

Around 10 countries, including South Korea and Britain, continued to express doubts and asked for more time to study the new South Africa/India proposal.

Three sources close to the talks see problems with the text.

"There is an ocean between this waiver proposal and what was suggested by the U.S.," said a source involved in the talks who declined to be named. "There's definitely no quick resolution for this."

Two aspects of the waiver draft that may harden opposition are its scope and duration.

While U.S. Trade Representative Katherine Tai had previously said she is only focused on increasing vaccine access, the new draft also includes diagnostics, therapeutics and medical devices, among others.

"When you have a big bomb like the U.S. saying we will support the waiver, people were expecting the revised proposal would narrow the scope," said a Geneva-based trade source.

The draft also sets a time span for awaiver seen as temporary of "at least three years" and allows the WTO's 164 members to determinewhen it ends. Given they would need to do this by consensus, one country could repeatedly prolong it.

"If the proponents insist on it (the duration), there will almost certainly be no consensus agreement on the waiver," said Peter Ungphakorn, a former WTO staff member who now writes blogs on trade.

An EU diplomat told Reuters that the revised text would"likely call the U.S. bluff". The European Union is set to present an alternative plan for boosting production and availability of COVID-19 vaccines to the WTO in early June.

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

59

Re: Biotech Generally, including Regulatory

from STAT
FDA tea leaves have become illegible

For much of 2021, there’s been a creeping narrative that the FDA had suddenly taken a turn for conservatism, issuing surprise clinical holds and handing down shocking rejections, whether because of politics, internal strife, or three-dimensional chess, depending on whom you ask. But a recent run of on-time, drama-free drug approvals has complicated that theory.

Yesterday, Alkermes won FDA approval for an antipsychotic called Lybalvi. The decision came on the FDA’s promised date, and the label brought no curveballs or unexpected safety constraints. The week before, Biohaven won a similarly timely and uncomplicated approval for its oral migraine treatment. “While investors have become increasingly concerned about a less predictable FDA, we believe these two approvals should ease some of those concerns,” Mizuho analyst Vamil Divan wrote in a note to clients.

But the broader takeaway might be that the FDA has never been broadly predictable. The drug-approving arm of the agency is divided into various fiefdoms with their own regulatory ideologies, personnel changes, and internal dynamics. The past six months have provided ample evidence for divergent theories about the relative conservatism and flexibility of the FDA as a whole, but it might be wiser to conclude that it’s simply a land of contrasts.

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

60

Re: Biotech Generally, including Regulatory

Interesting ploy...
Sound like the company should be shorted, because they purposely distort...
from STAT
================
CureVac’s curious Twitter strategy gets results

On Friday, just after the markets closed for a three-day weekend, CureVac put out a press release noting that its ongoing Covid-19 vaccine trial had completed an interim analysis and would continue as planned. On the upside, that means independent monitors didn’t see any safety problems. On the downside, it means CureVac’s vaccine didn’t clear the roughly 85% efficacy threshold that would have stopped the trial early.

Quite a few people focused on the downside, which, combined with the timing of the press release, suggested that CureVac’s vaccine might be wending toward disappointment. As anonymous Twitter user @Biohazard3737 put it, CureVac “failed the first interim.” Taking an interesting social media strategy, the company used its corporate account to publicly dispute that characterization in a direct reply to @Biohazard3737, whose Twitter avatar is a sheep, and explain that, actually, this is good news.

And in the day since, CureVac’s share price has risen nearly 6%. The company’s valuation is now higher than it was before the whole interim analysis conversation began. It’s probably not advisable for publicly traded biotech companies to mix it up with anonymous alphanumeric accounts on Twitter, but in this one instance, it seems to have worked out just fine.

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

61

Re: Biotech Generally, including Regulatory

BIIB Alzheimer's drug has been approved by the FDA.
Biotech sector has jumped higher.

This scotches the rumor that the FDA is being hard on Drug companies (which was a stupid rumor anyway).
Really helps BIIB.
May help some people with Alzheimer's.

The doubt about approval was because the trial results were not robust.

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

62

Re: Biotech Generally, including Regulatory

Apparently they have now halted the stock so they can get order in there, I suppose... and move it higher.

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

63

Re: Biotech Generally, including Regulatory

BIIB is still halted.

Biotech indexes up big, WITHOUT the contribution from BIIB when it reopens.
IBB+6.8%
XBI+5.89%

Of course, the new drug may not sell all that much, but many families will push for the doctors to prescribe it to see if it works... and there will be a placebo effect in addition to whatever drug effects there really are.

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

64

Re: Biotech Generally, including Regulatory

BIIB is set to open in about 10 minutes.

This is a LOOONNNG halt.

It suggests that the Big holders have put pressure on this to be halted while the look for (presumably0 bidders.

It is good for the sector for the stock to rise a lot, but it then may attract a lot of shorts. It is only minimall effective according to 1 trial and not effective according to another.

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

65

Re: Biotech Generally, including Regulatory

Plus >$150 on BIIB

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

66

Re: Biotech Generally, including Regulatory

Good for OMEROS

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

67

Re: Biotech Generally, including Regulatory

An ENDPOINTS Editorial.
=================
June 7, 2021 05:02 PM EDTUpdated 3 hours ago Bioregnum
Just how sadly misguided is the FDA on aducanumab?
Bioregnum Opinion Column by John Carroll

I doubt very much that Biogen paid anyone at the FDA to get an approval on aducanumab with this generous (?) label. But if they had, I doubt that even the execs at Biogen would have asked for this much.

Cheating requires some caution, if you expect to get away with it. And this one goes way past a cautious manipulation of regulatory powers in accomplishing a megablockbuster company goal worth potentially tens of billions of dollars.

Where are the guard rails on this regulatory track? Nowhere to be seen.


Billy Dunn
The FDA gave the curiously accelerated approval based on a surrogate endpoint of eliminating amyloid plaque, a marker previously disavowed by none other than FDA neuroscience chief Billy Dunn. And it’s being allowed for literally all Alzheimer’s patients, no matter how advanced their disease is, based on what they call a reasonable assumption of efficacy for the most desperate patients.

We’ve addressed the data here before. There are no efficacy data to clearly demonstrate this drug helps patients.

In their reaction today, an unamused ICER just noted that:

The FDA “has failed in its responsibility to protect patients and families from unproven treatments with known harms.”

And how.


Michel Vounatsos
While opening up a motherlode of drug money, Biogen CEO Michel Vounatsos tells CNBC’s Meg Tirrell that they have up to 9 years to complete a validating study required by the FDA, confirmed here. Late-stage, post-approval studies do not require 9 years. It is a ridiculous amount of time.

Over that 9 years, Biogen can squeeze patients and payers for their $56,000 price. And how many will have to dig deep, selling homes and liquidating assets, to cover a drug that may well do little or nothing, with a serious threat of physical harm over that 9 years?

I’m not alone here. The FDA had to studiously ignore its expert panel and multiple voices opposed to an approval. But what we’re seeing in the local news today are docs talking up the first med approved for the disease, the big breakthrough that finally arrived. Try telling the patients and families who read those reports that the data don’t add up.

In the meantime, the FDA has seriously damaged real explorations in Alzheimer’s disease. Instead of pulling away from the amyloid theory, or going past it, they are now giving it a tremendous boost, despite a mountain of data that proves it’s the wrong path — at least on its own.

They’re creating precedent that can be followed over and over again, as the FDA did with Sarepta.

Ultimately, this is a disaster that will once again create a serious atmosphere of financial toxicity, for everyone in biopharma. With millions of patients suffering from Alzheimer’s and Biogen going to extremes in exploiting these patients, how long before the backlash takes hold in new legislation that threatens real advances for patients?

Make no mistake, this is a train wreck. We’ll be counting the bodies and assessing the damage for years to come.

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

68

Re: Biotech Generally, including Regulatory

And another story, this time about pricing.
How much do you charge for a drug that doesn't have proof it works, but everyone with relatives with Alzheimer's wants?

And I post it because a backlash against high priced drugs, is bad for all drug companies, even OMER, and even if the drugs work and saves the lives of patients.
https://endpts.com/analysts-hound-bioge … l-markets/
=============================
June 8, 2021 11:19 AM EDT Pharma
Analysts hound Biogen with questions over Aduhelm's $56K price point as news of Alzheimer's OK ripples through global markets
Max Gelman Associate Editor and Nicole DeFeudis Associate Editor

It’s been nearly 24 hours since the FDA handed down its controversial decision to approve Biogen’s Aduhelm drug for Alzheimer’s disease, and reactions from around the industry are pouring in as analysts try to size up the ramifications of Monday’s news.

Biogen hosted an investor call Tuesday morning, providing a forum for a number of topics that have been top of mind. The call came as the news of Aduhelm’s approval in the US rippled through global markets, while $BIIB stock soared to around $380 apiece (up from $286 on Friday), as Biogen’s partners at Eisai seek new approvals in Asia.

Chief among the discussion points today was the price of this new drug, which Biogen set at $56,000 annually — a figure that’s between seven and 22 times higher than ICER estimates of a fair price.

Evercore’s Umer Raffat kicked things off with a question on exactly that.

Saying there’s been a “disconnect” between how Biogen has presented its push for equitable pricing and access and Aduhelm’s cost, Raffat asked the slate of execs for their thought process behind the $56,000 figure, highlighting the lack of a primary outcome benefit for the drug. Raffat also questioned Biogen over how they planned on implementing a potential value-based contract, given the difficulty of doing so.

Biogen CEO Michel Vounatsos, however, largely sidestepped the meat of the question, responding with how he says Biogen tries to balance price with the value a drug might bring to patients. He agreed with Raffat over the challenges of value-based contracts, but said Biogen is open to them if the market provides an opportunity similar to the ones in multiple sclerosis.


Chirfi Guindo
Chirfi Guindo, Biogen’s head of global product strategy and commercialization, added some color on the topic, giving a broad view of the company’s pricing strategy as a whole. He noted that Biogen will not increase the price of Aduhelm for the next four years, a tacit acknowledgment that the cost would potentially be too burdensome.

But Guindo, too, avoided addressing the disconnect brought up by Raffat, saying only that Biogen considers the drug to be a specialty product, rather than a primary care drug. While admitting there are no good comparisons for Alzheimer’s drugs, Guindo attempted to justify the price by saying it costs “roughly a third” of new specialized cancer immunotherapies and about 25% below recently approved psoriasis treatments.

“We consider this to be a really responsible price, and we consider this to be a price that is sustainable for the system,” Guindo said.

Continuing with the pricing theme for the morning, SVB Leerink’s Marc Goodman followed up with a question about what Biogen will tell doctors about how long they should keep patients on the treatment. R&D chief Al Sandrock said this is an issue that Biogen needs to continue investigating, but pointed to long-term extension data from their Phase Ib trial indicating the possible reduction of amyloid plaque for “many years.”


Al Sandrock
Goodman pressed Sandrock further, pointing out the data from Biogen’s larger trials only measured amyloid plaque reduction out to 18 months. Sandrock, though, said he saw “no reason” to limit patients receiving Aduhelm to only that year-and-a-half period.

Analysts are further concerned about the impact the price will have on the US healthcare system, because most of the patients who will end up with Aduhelm prescriptions will be seniors on Medicare plans. JP Morgan’s Cory Kasimov questioned the team about this burden, pondering if a potential industry-wide backlash is in the cards.

Vounatsos said only that Biogen forecasts about 80% of Aduhelm patients will be on Medicare, with Guindo chiming in that yearly Medicare budgets are predictable enough so the company can adequately work with payers to ensure the system is sustainable.


Ronny Gal
Bernstein’s Ronny Gal, who didn’t ask a question on the call, also noted that if 1 million patients use Aduhelm at the current price point, total Medicare part B spending would double. That figure is probably out of range, as there are about 500,000 new Alzheimer’s diagnoses a year in the US. But a more realistic scenario, he said, is “equally mind-boggling”: If half of newly diagnosed patients start Aduhelm at the current price, the total cost to Medicare will be equal to the top five drugs in Medicare part B combined: Keytruda, Eylea, Opdivo, Rituxan and Prolia.

The total cost of Medicare part B drugs in 2019 was $37 billion. This is where Gal sees room for systemic reform, comparing the situation to Gilead’s HCV drug Sovaldi, which came on the market back in 2013 at $1,000 per pill, or about $84,000 per course. Gilead was vilified for the list price, and pressure from payers eventually brought down the average price. In 2017, the company announced it would price the drug in China at just $8,939.

Gal wrote:

As a reminder, when Gilead priced Sovaldi (an HCV cure) at $100K, PBMs went to employers and promised to rein in drug costs if given more power to control formularies. The powers they obtained then have led to pressure on multiple drug categories – e.g. Insulins, LABA/ICS… The pricing action of Biogen would likely be very negatively perceived by fiscal conservatives and in the context of FDA stretching to approve the drug based on borderline. This may be enough to shift the view within this critical narrow majority. The argument being that the industry can’t be trusted to police itself.

Biogen was also asked about a number of other topics, including the availability of CSF testing versus PET imaging to screen patients for amyloid plaque levels, the FDA’s broad label for the drug and what the commercial launch might look like in the coming weeks. One analyst from Goldman Sachs asked for details on the required confirmatory study, but Biogen said they couldn’t provide details on this topic just yet as they’re still talking with the FDA.

And the debates are taking off elsewhere. Cowen said Monday that they project Aduhelm to reach 8% of Americans with mild Alzheimer’s by 2025, yielding $7 billion in revenue, while Baird’s Brian Skorney estimates sales of about $7.5 billion over the same timeframe.

The research journal Health Affairs chimed in as well, saying the Centers for Medicare and Medicaid Services should protect consumers by ensuring access only for patients likely to see clinical improvement. If it’s not possible to see who might benefit, CMS should deny coverage until it’s safe to do so.


Haruo Naito
News of the approval reverberated around the world, as Biogen’s Tokyo-based partners at Eisai plan to roll out the drug in Asia. CEO Haruo Naito told Nikkei Asia on Tuesday that the drug would “become a substantial blockbuster,” with sales in Asia contributing significantly to the company’s earnings.

“Asia, including Japan and China, has an enormous number of [dementia] patients,” Naito told Nikkei.

Biogen submitted aducanumab for regulatory approval in Japan — which has a rapidly aging population with a median age just over 48 years old — back in December. It was the third market Biogen sought approval in, behind the US and Europe. Usually the timeline to a decision is about a year, according to Ivan Cheung, US chairman & global president of Eisai Neurology.

“As Japan has the oldest population in the world, it is anticipated that the social burden of Alzheimer’s disease will continue to grow,” Naito said in a statement at the time.

According to Cheung, the companies will co-promote and co-commercialize the drug around the world. In Japan and other Asian countries, excluding China and South Korea, Eisai will take the lead, and will receive an 80-20 profit split. In the US, the profit split is 55-45 between Biogen and Eisai, respectively. The companies have filed for approval in the EU, Switzerland, Canada, Japan and Australia, Cheung said.

“That, of course, our goal is to continue to file, as you can imagine, in many countries across Asia and other parts of the world,” he added.

Analysts are also talking about what the approval could mean for other companies. RBC’s Gregory Renza said it could be news for drugs like Acadia’s Nuplazid, which got a CRL back in April for the treatment of hallucinations and delusions associated with dementia-related psychosis. The agency cited a lack of statistical significance in some of the subgroups of dementia, and insufficient numbers of patients with certain less common dementia subtypes as lack of substantial evidence of effectiveness to support approval, according to Acadia.

“The news reads through most positively to ACAD, in our view, where Nuplazid/pimavanserin’s solid DRP data and filing package, despite the questions on the trial and the CRL, could now have a new boost, or more compelling case, to its path towards label expansion,” Renza said in a note to investors.

Yesterday, Nikos Robakis, a neuroscientist at Mt. Sinai Medical Center, told Endpoints News that he now fears companies will spend billions trying to get similar amyloid-clearing antibodies through clinical trials and past regulators, which could better be used funding research or alternative pathways.

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

69

Re: Biotech Generally, including Regulatory

Not really any NEW info in this article if you have been following the topic.
================
Controversial drug approval stokes concern about lack of a permanent FDA chief
Janet Woodcock did not release a statement with the approval — noteworthy considering it was the first new Alzheimer’s drug in nearly two decades.

Janet Woodcock, director of the Food and Drug Administration's Center for Drug Evaluation and Research testifies before the House Energy and Commerce Oversight and Investigations Subcommittee.
Acting FDA commissioner Janet Woodcock appeared to distance herself from Monday’s decision, despite a yearslong track record of championing quick approval of new medicines. | Photo by Win McNamee/Getty Images

By SARAH OWERMOHLE, ADAM CANCRYN and LAUREN GARDNER

06/11/2021 04:30 AM EDT

The Food and Drug Administration’s approval of a controversial new Alzheimer’s drug this week has hardened opposition to acting commissioner Janet Woodcock, who remains President Joe Biden’s apparent favorite to be the agency’s permanent leader.

The administration's search for a permanent FDA head has dragged on for months during an unprecedented public health crisis. Woodcock, a veteran regulator, has presided over major decisions on the pandemic response, tobacco and new medicines — including the $56,000-a-year Biogen Alzheimer's drug approved Monday despite thin evidence that it works.


Woodcock appeared to distance herself from Monday’s decision, despite a yearslong track record of championing quick approval of new medicines. She did not release a statement with the approval — noteworthy considering it was the first new Alzheimer’s drug in nearly two decades — or appear in the agency’s briefing with top officials after the announcement.


"It's an incredible loss of confidence in transparency around the agency, in particular the drug approval process” and “an abject failure of leadership” on Woodcock’s part that the drug was approved, said a former senior FDA official.

Critics for years have accused Woodcock of being too friendly with the industry she regulates, and that under her decadeslong tenure heading FDA’s drug division the agency approved new, expensive medicines without enough evidence they were effective or safe. Several, including a Duchenne muscular dystrophy medicine and certain cancer treatments, have yet to pan out and show clear clinical benefit. Another approval under Woodcock — for Dsuvia, a potent fentanyl pill — has drawn the ire of some in Congress and criticism from FDA’s outside advisors for worsening the addiction crisis.

“It isn't as black and white as people would like ... these are hard decisions and there's no really right answers,” Woodcock told POLITICO in April amid the agency’s last controversy over drug approvals, in that case involving cancer medicines that did not show benefit against certain tumors after several years on the market.

The White House did not respond to a request for comment for this story. An FDA spokesperson declined to comment.

A vial of the Johnson & Johnson Covid-19 vaccine is shown in Belgium.
THE VACCINE RACE

FDA panel endorses Johnson & Johnson vaccine in 22-0 vote
BY SARAH OWERMOHLE

Monday’s decision on the Alzheimer’s medicine, known as aducanumab or Aduhelm, threw the FDA into a fresh round of dissension and second-guessing from scientists, experts and even Wall Street analysts. Three members of FDA’s expert advisory panel — which voted against approving the drug in November — resigned in protest.



Longtime observers of the agency say it was highly unusual for Woodcock, as acting commissioner, to stay publicly silent about the aducanumab decision.

“It’s hard to imagine that she didn’t have a role in this,” said Diana Zuckerman, president of the National Institute for Health Research. Throughout the years, “she has been considered the person who was the most powerful at the agency, most of the time.”

Yet despite the blowback, there's little sign it has affected Woodcock's standing within the West Wing. Biden was pleased with the decision, according to two people familiar with his reaction, which comes as he's pushed for fresh investment in developing treatments for Alzheimer's and other diseases.

Biden is seeking $6.5 billion as part of his fiscal 2022 budget request to create a new research group within the National Institutes of Health aimed at accelerating work on Alzheimer's, diabetes and cancer.

Meanwhile, the White House has identified few viable alternatives to Woodcock for the top FDA job. The administration has vetted former Obama-era FDA official Michelle McMurry-Heath for the commissioner role, several people familiar with the process said.

But McMurry-Heath has now run the pharmaceutical industry group Biotech Innovation Organization for the past year, potentially making her a harder sell within a Democratic Party that's advocated cracking down on drug prices. BIO in May criticized Biden's decision to support waiving patents on Covid-19 vaccines and previously opposed Trump administration efforts to rein in drug prices.

Other former officials, including past commissioners, insist that Woodcock is still the best pick for the top role.

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

70

Re: Biotech Generally, including Regulatory

This article has an important tell for Omeros investors.
https://endpts.com/sen-manchin-to-biden … missioner/

Not because Manchin doesn't want Woodcock.
But because he is fanatical about being anti-opioid, given that WV has a large problem with abuse of this drug.
The Democrats and Republicans want to curry favor with Sen. Manchin because his is the centrist swing vote in the Senate.
This makes NOPAIN much more likely to pass, IMO.

Sen. Manchin to Biden: Don't nominate Woodcock for FDA commissioner
Zachary Brennan
Senior Editor
While Sen. Joe Manchin’s opposition to Janet Woodcock as Biden’s choice for FDA commissioner has been known for months, the West Virginia Democrat spelled out more detailed concerns in a new letter to President Biden on Thursday.

“Dr. Woodcock is not the right person to lead the FDA,” Manchin, an influential centrist, writes, while noting the agency has been without permanent leadership for about five months.

Manchin raises the example of the recent accelerated approval for Biogen’s Alzheimer’s drug, which Woodcock has since said she was not a part of, and how the agency has yet to explain why it went against its advisory committee’s near-unanimous recommendations to reject the drug, while noting that three of those adcomm members have since resigned.

Woodcock has remained mostly silent on the approval, although she told BioCentury recently that she thought the data on amyloid clearing “were quite solid.” The nonprofit watchdog Public Citizen has called for Woodcock to resign over the approval.

The opioid epidemic and Covid-19 are also top of mind for Manchin as he raises concerns that without a permanent, Senate-confirmed FDA commissioner, important decisions that impact the country’s response to Covid-19 “will be slowed and prevent additional work to combat the ongoing drug overdose epidemic.”

Manchin makes clear that the FDA has played a critical role in this overdose epidemic, but not in a good way. “By overseeing continuous approvals of stronger and more addictive opioids since the initial approval of OxyContin in 1995 – and Dr. Woodcock has been there for all of it. Dr. Woodcock has repeatedly ignored public health concerns and shown a dereliction of duty by not working to end this epidemic,” Manchin writes.

The decision for Manchin, who has deep ties to the biopharma industry as his daughter served as CEO of Mylan, to be more public about his concerns with Woodcock comes as at least three other Senate Democrats – Maggie Hassan of New Hampshire, Ed Markey of Massachusetts, and Catherine Cortez Masto of Nevada – have pre-empted Biden’s nomination and voiced their opposition to Woodcock, who by all indications is still the leading contender for the job.

Those Democrats voting against a potential Woodcock nomination may not end up mattering much because she may win over some Republican votes, not only because of the approval of Biogen’s Aduhelm, which several Republicans have since praised, but also because of her experience working for the Trump administration’s Operation Warp Speed as head of therapeutics.

When exactly Biden’s nomination may occur remains unknown. HHS Secretary Xavier Becerra told Bloomberg Law Wednesday that Biden will make the nomination once the busy president finds time.

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

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Re: Biotech Generally, including Regulatory

Note who wrote this Forbes article in 2018, see the profile at bottom.
===========================
Jun 8, 2018,09:14am EDT
An Unlikely Biotech Investor: The Government
Steve Brozak Contributor

This article is more than 3 years old.
Walking around the convention floor at BIO was a remarkable experience this week in Boston.  Not only were there an endless number of human health companies present, there were also animal health companies.  States like Georgia, New Jersey, Maryland, Hawaii were also present, and countries too, among them Estonia, Japan, Switzerland, and Canada was a major sponsor of this year’s conference.  The message at BIO was clear: The world needs biotech.

Not to be overlooked, nestled somewhere between the pavilions for Rhode Island and Amgen was an enclave of government booths showcasing research programs and demonstrating technologies sponsored by various military and civilian agencies of the United States Government.  It was an animated area on the conventional hall floor.  This year one of those agencies, the Biomedical Advanced Research and Development Authority (BARDA), took the opportunity at BIO to make a major program announcement regarding a new funding initiative called DRIVe (Division of Research, Innovation, and Ventures).  I attended BARDA’s DRIVe event on Wednesday morning hosted by BARDA's Director, Dr. Rick Bright, and the message was even clearer: BARDA is open for business in a very big way.

BARDA was formed in 2006 to fund the research, development and stockpiling of vaccines and other treatments to meet public health emergencies such as a chemical, biological, radiological, or nuclear attacks.  DRIVe’s mission will be to accelerate research, development, and availability of transformative countermeasures to protect Americans.  Unlike the current funding mechanisms the government uses, it seems that DRIVe will act more like a strategic investor in private and public companies in addition to being a grant maker.  This means that the new division may be able to make direct investments into companies BARDA would like to partner with and derive value by holding equity or equity-like instruments in the venture.  Investing in opportunities in this manner offers a pathway to renew funds to reinvest into other ventures deemed essential to the national interest.

DRIVe’s first public announcements have highlighted the need to control infections in the blood, or sepsis.   The standard-of-care for a patient with a life-threatening infection is to fill the patient with broad spectrum antibiotics in the hope that they will stop whatever is causing the infection, take a blood sample and, after 24 to 48 hours, get the results that indicate whether the disease is caused by bacteria or fungi and what pathogen is responsible.  A targeted therapy can at last be administered after the results of the blood test return.  All too often when an infection is finally diagnosed it has become a potentially fatal problem (usually when the patient has gone septic or has been unresponsive to previous anti-infectives for too long.)

The DRIVe initiative has just launched so it is just starting to get attention, but it should be considered an essential approach to stimulating development of new healthcare products that will prevent, diagnose and treat public health threats in a timely and effective manner.  Its value may not be perceived until the next global healthcare threat emerges, and even then its role in combating threats may be overlooked like many of BARDA’s major accomplishments that we take for granted as a society.

BARDA, Merck And The Fight To Beat Ebola

BARDA’s job isn’t as easy as funding research to make medical countermeasures materialize.  It has to work with companies to come to the table and work on initiatives that aren’t necessarily on the radar screens of big pharma and biotech.  For instance, between 2014 and 2016 Ebola took the lives of 11,000 people in a small region in Africa and was on the verge of becoming a worldwide threat.  The primary means for controlling Ebola then was isolating the sick and restricting travel outside the affected region.  With no widely available effective vaccine, one person on an airline flight could bring the disease to the U.S., Europe or Asia.  For the better part of a decade, BARDA has been working with pharmaceutical companies to enhance our ability to respond to threats like Ebola.

Today, there is another ongoing outbreak of Ebola in a different region of Africa, and this time, because of the efforts of BARDA and its work with Merck & Co., more than 4,000 doses of a novel vaccine are available to control and eliminate the disease threat.  BARDA and Merck have been working in concert to take the fight to Ebola, developing a single-shot vaccine that is being used to protect people who are at high risk of exposure to the virus.  Using a ring-vaccination protocol, Merck’s vaccine has already shown potential efficacy during testing in Guinea, West Africa.  The approach aims to stop the spread of a virus by vaccinating every person who came into contact with a patient and everyone who came into contact with the patient’s contacts.  BARDA is supporting late-stage activities in support of licensure and potential procurement of Merck’s Ebola vaccine into the Strategic National Stockpile, which could be up to $39 million in investment.

By working together and investing in Merck’s efforts, BARDA has not only helped to develop a countermeasure to a deadly virus.  BARDA has kept focus on our capability to respond to emerging biothreats, and ensured that our nation’s prowess to develop and manufacture new vaccines against diseases as they emerge stays intact.  Merck has shown its willingness to step up to the plate as a good corporate citizen and assist world governments tackle some of our toughest health challenges.

BARDA, Achaogen, And The Quest For New Antibiotics

The greatest looming threat to the public health of the U.S. is the emergence of bacterial diseases that are resistant or immune to many antibiotics now in use.  The CDC estimates that drug-resistant bacterial infections affect two million people and kill 23,000 in the U.S. each year at a financial cost of $20-35 billion. Those numbers continue to rise each year.

BARDA’s CARB-X program (Combating Antibacterial Resistance) establishes public/private partnerships to develop new and effective antibiotics.  Convincing companies to develop antibiotics and diagnostics for use in infectious disease is challenging for a few reasons.  First, there is the economic challenge.  Antibiotics and diagnostics are not as lucrative as drugs used to treat rare diseases and cancer.  Second, the regulatory pathway for antibiotics is incredibly risky.  Programs like CARB-X help to reduce some of the financing challenges, rewarding investors in infectious disease companies by providing non-dilutive cash to help propel the technology.  As much as it encourages development of new antibiotics, CARB-X also helps to bring new approaches to treating infectious disease to the forefront of discovery.

Achaogen, Inc., a small company with market value of a little more than one-half billion dollars, is a CARB-X company.  The company received two antibiotic incentive awards through the program for $23.4 million during a crucial stage in the development process.  The typical cost to bring a new antibiotic to market is well over one billion dollars.  Approval for a new antibiotic requires two successful large, phase 3 trials, unlike rare diseases, which only require smaller trials.  That is because antibiotics are so widely prescribed that even a small percentage of adverse events could cause a wide range of problems, from anaphylactic shock to liver damage or weakened and broken tendons, affecting the lives of thousands of people.

Achaogen’s drug, Plazomicin, has been supported by BARDA funding since 2010.  So far, BARDA has provided $136 million to support the development of Plazomicin, including helping to pay for two phase 3 trials.  Plazomicin belongs to the aminoglycoside class of antibiotics and provides a robust therapeutic option against serious bacterial infections that are multidrug resistant gram-negative bacteria, including carrbapenem-resistant Enterobacteriaceae (CRE).  The drug was tested in blood stream infections (BSI) and complicated urinary tract infections (cUTI).  In May an FDA Advisory panel was asked whether Achaogen’s drug showed “substantial evidence of safety and efficacy” in both BSI and cUTI.  The panel voted unanimously (15-0) that it did for cUTI, but only voted 4-11 that it did for BSI.  Regardless, the outcome is a huge success for BARDA as the company heads into a final decision on June 25th in two indications:  Septicemia/Bacteremia (BSI) and Urinary Tract Tract Infections.  Given the safety and clinical history of the drug to date, and the dire need for new drugs for doctors to turn to, we believe Plazomicin should be approved by the FDA.

I was able to catch up to Achaogen’s CEO, Blake Wise, after BIO, and he was unequivocal regarding the importance of BARDA to his company’s drug development process, saying, “The rise of antibiotic resistance poses a great threat to public health, and BARDA’s commitment to address this challenge has been welcomed by the infectious disease community.”  According to Wise, BARDA provided critical support to Plazomicin, and even other pipeline candidates, without which the pace of new drug development would have even more dangerously lagged the rise of antibiotic resistant infections.  Critical to the success of the partnership, management cited the creative approach BARDA has taken to forming innovative and flexible public/private partnerships to make sound investments in products to help fight antibiotic resistant bacteria.

Now with DRIVe joining BARDA's funding portfolio alongside BioShield and the CARB-X initiative, they have yet another tool in their war chest to spur discovery in the national interest.

WBB Securities Asset Management maintains a long position in Achaogen.

Steve Brozak

Steve Brozak is President of WBB Securities, LLC, an investment bank and research firm that specializes in the biotechnology, specialty pharmaceutical and medical device sectors. Steve is an award winning analyst whose research has been singled out from his peers for accuracy and performance year after year by the financial industry's most highly regarded rankings organizations. His research is currently monitored by the StarMine, FactSet and TipRanks Platforms. Steve has written articles and opinions for Forbes, CNN, ABC News, and Businessweek-Bloomberg on trends in the healthcare sector, and he appears regularly as a guest commentator on financial television outlets such as Bloomberg, BNN, CNN and CNBC. He has also authored pieces for Nature, The British Medical Journal and Brain Stimulation. Steve is an outspoken advocate for the absolute need for changes in how we research and finance our healthcare system. He retired as a Lieutenant Colonel from the US Marine Corps after several deployments. After retirement he completed a three-year appointment to the Navy and Marine Corps Retiree Council under the SECNAVs authority where he focused on healthcare, and other retiree issues. Steve received both his BA and MBA from Columbia University and completed his Doctorate in Medical Humanities, DMH from Drew University for the analysis of the American healthcare system. To learn more about WBB Securities, please visit www.wbbsec.com.

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

72

Re: Biotech Generally, including Regulatory

We will see if his $100 Omeros prediction comes true....But he seems to be the real deal, as opposed to other analysts we know.

73

Re: Biotech Generally, including Regulatory

Sen. Wyden releases 'principles for drug pricing reform'
Jun. 22, 2021 11:12 AM ETABBV, AMGN...By: Jonathan M Block, SA News Editor13 Comments
Senate Democratic Leadership Speaks To Media Ahead Of Impeachment Trial
Sarah Silbiger/Getty Images News
Sen. Ron Wyden (D-Ore.) has released a document titled "Principles for Drug Reform" that proposes giving Medicare the ability to negotiate prescription drug prices and lowering out-of-pocket costs for consumers at pharmacies.
He wants to give the HHS Secretary the power to negotiate Part D drug prices to create "a much needed mechanism to achieve fairer prices when the market has failed to do so."
Wyden adds that in a negotiation policy, Congress needs to establish clear criteria for market failure and for which drugs to negotiate, as well as define what constitutes a fair price.
He says he will propose legislation that will ensure consumers see savings on specific drugs, such as insulin, and that will also end rebate practices that "limit competition and patient access to affordable drugs."
Another principle is that drugmakers will need to provide rebates for drugs whose prices increase faster than inflation.
Wyden says he will also work to keep drug prices in check for those covered by employer and commercial health insurance plans.
Finally, Wyden believes drug pricing should reward scientific innovation. "Government funding for R&D should continue to receive strong support, while also protecting taxpayers, who shouldn't be paying twice for the research when a drug comes to market with high prices."

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

74

Re: Biotech Generally, including Regulatory

June 23, 2021 11:11 AM EDT Pharma
A Pfizer win in a heated anti-kickback case could lead to a ‘gold rush’ of biopharma companies subsidizing Medicare drugs, government says
Zachary Brennan
Senior Editor
Pfizer on Tuesday made the case for why it should be able to subsidize the cost of one of its rare disease drugs for some Medicare beneficiaries, but government lawyers warned of a “gold rush” of similar subsidies from biopharma companies if the court rules in Pfizer’s favor.

The District Court for the Southern District of New York on Tuesday heard oral arguments from both Pfizer and the government over whether the company can help poorer Medicare patients afford the company’s rare disease drug tafamidis, which comes with a $225,000 list price for a single year’s supply, of which Medicare beneficiaries have to pay about $13,000 annually.

HHS’ Office of the Inspector General previously rejected Pfizer’s plans to help the beneficiaries directly and another plan to set up a foundation that would provide the subsidies.

According to Pfizer, the company should be able to help all but the wealthiest Medicare beneficiaries, as the anti-kickback law does not deal with assistance that “merely enables patients to overcome a financial barrier to obtaining appropriately prescribed medication they desperately need.”

The government, however, called Pfizer’s suit “not only meritless, but also gratuitous. Rather than being a victim of adverse governmental action (e.g., an enforcement action) or improper action, Pfizer is simply unhappy it did not receive something from OIG to which it is not entitled.” The government also said that treating all eligible patients who have the rare, progressive, and fatal condition of Transthyretin Amyloid Cardiomyopathy with Pfizer’s tafamidis would increase health care spending in the US by over $30 billion a year.


Jennifer Michael
Jennifer Michael, former chief of the industry guidance branch at HHS and a member of the law firm Bass, Berry & Sims, told Endpoints News that the ruling on this case could have far-reaching implications, as the government showed toward the end of their oral argument what would happen if the court applied this Pfizer standard across the board. She said the government lawyers noted that it would lead to a “gold rush” across pharmaceutical companies to subsidize these payments, as the only check on pricing in Medicare Part D is this cost sharing requirement.

“If Pfizer moves forward, Congress will have to address this somehow,” Michael said, noting that the judge in the case said an opinion will be coming soon.

Michael also noted that the judge bluntly asked Pfizer why it doesn’t just lower the list price for tafamidis, rather than subsidize it. But Pfizer lawyers noted that no matter what the price is, there will always be a subset of patients that can’t afford the cost-sharing, and there’s still a legal question with respect to providing direct subsidies.

Since 2005, OIG has always been clear that where a drugmaker provides financial help, there’s a high risk for fraud and abuse, meaning the companies could end up paying people to take their drugs.

And Pfizer is no stranger to how that process works. Back in 2018, the company agreed to pay $23.85 million to settle allegations that the company violated the False Claims Act by paying kickbacks to Medicare patients through a purportedly independent charitable foundation.

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

75

Re: Biotech Generally, including Regulatory

Years ago on TIPS we used to follow this crappy oncology company. I seem to recall it was fishy then and it seems they are still busy misleading investors.
****************************
from STAT
The Cel-Sci saga may go on for years

Last week, after more than a decade of plodding clinical development, Cel-Sci admitted that its long-delayed treatment for head and neck cancer had no survival benefit. But thanks to some well-honed stalling tactics and a timely cash infusion, the company can likely keep this going for years to come.

As STAT’s Adam Feuerstein reports, Cel-Sci’s stated next step is to submit that treatment, Multikine, for FDA approval. But the fact that Multikine clearly failed in Phase 3 suggests the FDA will not be granting such an approval. Regulators might refuse to even consider Cel-Sci’s application.

But the company, which spent 13 months delaying the disclosure of its pivotal data, is surely capable of running out the clock. The company raised $32 million in a stock sale earlier this year and has about $47 million in cash.

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

76

Re: Biotech Generally, including Regulatory

July 9, 2021 07:47 AM EDT Pharma
Pelosi threatens to slide drug pricing reform into Biden's budget bill, rejects industry pushback that innovation would suffer
Josh Sullivan
Associate Editor
After a report released by a key House committee showed a projected $1 trillion in buybacks over the next 10 years by Big Pharma, Speaker Nancy Pelosi said she wants to attach drug pricing reform to the current budget bill, and slammed drug companies for suggesting innovation would be harmed as a result.

If passed, the bill would empower Medicare to negotiate directly with drug companies to lower prices, a longstanding goal for Democrats. The House report claims the policy shift could save taxpayers $456 billion in the next 10 years.

“We have an extraordinary opportunity to do this as we craft this reconciliation bill,” Pelosi said on a conference call with reporters.

The House Oversight Committee released a staff report that analyzed financial data from the 14 largest drug companies to evaluate the amount of money they invest in R&D, and the impact that has on Medicare price negotiations.

From 2016 to 2020, the companies spent $56 billion more on stock buybacks and dividends — $577 billion — than on R&D, the report found. The projected number of spending on buybacks from 2020 to 2029 is $1.15 trillion for the same 14 companies.

“The Committee previously released six staff reports showing that the pharmaceutical industry has targeted the United States for price increases for many years, while cutting prices in the rest of the world,” the report said. “The United States is particularly vulnerable to these pricing tactics because current law prohibits Medicare from negotiating directly with drug companies to lower drug prices.”

The 14 companies included the study are AbbVie, Amgen, AstraZeneca, Bristol Myers Squibb, Eli Lilly, Gilead, GSK, J&J, Merck, Novartis, Novo Nordisk, Pfizer, Roche and Sanofi. Between 2016 and 2020, the top executives for these companies brought home a combined salary of $3.2 billion, and eight of those companies spent less on R&D compared to buybacks and dividends, the report states. Amgen’s spending on buybacks was six-fold, compared to R&D costs.

“This report finds that the world’s leading drug companies have used price increases to boost payouts to investors and executives while spending less on research and development,” Rep. Carolyn Maloney (D-NY) said in a press release. “The report also shows that industry claims about the potential impact of pricing reforms are overblown.”

Industry lobbyists have played tight defense, arguing that it would deter the creation of new drugs, and hurt patients in the end. Brian Newell, a spokesperson for PhRMA, a biopharma lobbying group, told Bloomberg that his organization was committed to working with policymakers on commonsense solutions that address the “real challenges patients face.”

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

77

Re: Biotech Generally, including Regulatory

Janet Woodcock calls for independent investigation into Biogen and FDA relationships in unprecedented move

In the latest stunning chapter of the Biogen Aduhelm saga, acting FDA commissioner Janet Woodcock is calling for an investigation into the relationships between agency officials and Biogen executives.

Woodcock announced the move early Friday afternoon, posting a letter to the acting HHS Inspector General on Twitter.

The request comes as controversy continues to swirl around the approval, with prominent members of Congress launching probes and Biogen taking the extraordinary step this week to ask for a narrower drug label. Woodcock's call for a probe also comes less than two weeks after a report from STAT News highlighted a major undertaking from Biogen to get Aduhelm approved that began as early as 2019.

“We believe an independent assessment is the best manner in which to determine whether any interactions that occurred between the manufacturer and the agency’s review staff were inconsistent with FDA’s policies and procedures,” Woodcock wrote on Twitter.

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

78

Re: Biotech Generally, including Regulatory

July 19, 2021 10:09 AM EDTUpdated 10:43 AM PharmaFDA+
FDA’s domestic biopharma inspections return to normal as agency faces backlog of 8,000+
Zachary Brennan
Senior Editor
For the first time since March 2020, the FDA’s inspections of US-based biopharma sites have returned to normal, FDA acting commissioner Janet Woodcock said Monday at a small business regulatory event.

“I’m pleased to say that as of this month, we’ve begun transitioning back to standard operations for domestic inspections while continuing to prioritize mission-critical work for foreign inspections,” Woodcock said.

The return to normalcy will be welcomed by industry, which has grappled with a growing backlog of mostly surveillance inspections, although some of those delayed inspections have also delayed the approval of new drugs.

According to a report from FDA in May, an estimated 68 applications (including 48 for human drugs) have been delayed due to FDA’s inability to conduct pre-approval, pre-market, or pre-license inspections as of March.

The agency said it postponed nearly 8,000 non-mission-critical surveillance inspections in 2020 due to the pandemic. Surveillance inspections are routine inspections that monitor conformance to FDA requirements to identify quality problems and adverse trends, but unlike others, they’re planned in advance by applying established risk factors and statutory inspection frequency mandates.

“FDA estimates that roughly 14% of the 15,514 domestic surveillance inspections still to be conducted in FY21 will be achievable in the Base-Case scenario,” the report says, noting that about 3,229 of those 15,000+ inspections are related to human and animal drugs, while the rest are related to human and animal food.

And in terms of for-cause inspections, meaning inspections where there are consumer complaints or reports of adverse events, the FDA had to skip 8 of those inspections in FY 2020.

As far as how the agency is working around the rising cases of Covid-19 in certain hotspots across the country, FDA revealed earlier this month that it developed a rating system to assist in determining when and where it’s safest to conduct domestic inspections.

The rating system, which FDA said it may suspend through September as it resumes standard operations, uses real-time data to qualitatively assess the number of Covid-19 cases in a local area based on state and national data. FDA shared that data with state agencies that carry out inspections of FDA-regulated entities on the agency’s behalf under contract.

FDA also previously said that for the foreseeable future, prioritized domestic inspections will be pre-announced to FDA-regulated businesses. Woodcock did not announce any change to that policy.

Other Covid-related transitions
Following Woodcock’s comments, FDA center directors Jeff Shuren (devices), Patrizia Cavazzoni (drugs) and Peter Marks (biologics) also offered their perspectives on what Covid-related provisions may carry over to the post-Covid FDA.

Shuren stressed the need for faster guidance development, noting that some Covid-related guidance documents were developed in only days or weeks, and he’d like to see that expedited approach carry over beyond the pandemic. Marks and Cavazzoni concurred, and Cavazzoni mentioned the need to build off FDA’s accumulated experience with decentralized clinical trials during Covid-19, and the use of alternative approaches, such as remote evaluations, to inspect manufacturing and clinical trial facilities.

As far as when the EUA pathway may close for sponsors, Shuren said he didn’t think it would happen any time soon, and he noted that there will be an “ample transition period,” as well as guidance on that transition. Marks said the EUA transition period for vaccines may look complicated as there might be some pediatric indications still under EUAs for vaccines that have won full approvals. He stressed that the idea is to minimize anything that creates confusion, and decreases trust.

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

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Re: Biotech Generally, including Regulatory

Shares in Ardelyx Inc. (ARDX) plummeted more than 70% in after-hours trading Monday, following the biopharmaceutical company's announcement (https://ir.ardelyx.com/news-releases/news-release-details/ardelyx-provides-regulatory-update- new-drug-application) that the Food and Drug Administration appears unlikely to approve a drug for dialysis patients. Ardelyx revealed that it received a letter from the FDA stating that deficiencies in the information provided had been found that would preclude discussion of approvals Ardelyx has sought. When Ardelyx sought a meeting with the FDA to discuss the deficiencies, the request was denied, though Ardelyx stated that "the FDA noted that a key issue is the size of the treatment effect and its clinical relevance." "This is an extremely disheartening and disappointing communication from the FDA, particularly following the weeks of label discussions that occurred in early April, the fact that our NDA submission included three pivotal trials across 1,000 patients, all which met their primary and key secondary endpoints, as well as the additional data analyses we submitted in late April in response to the FDA's requests," Ardelyx Chief Executive Mike Raab said in a statement. Ardelyx shares, which closed at $7.70 in the regular session for a market capitalization of more than $700 million, saw shares dive to less than $2.50 in the after-hours trading period.

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