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The market is not helping.

But this is also Option expiration on Friday with MAX PAIN at $15, but unlikely to get that low. The Option MM may want to push it there if he is short the "wrong" calls (under $17). Then again, there are other players involved too.

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

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We're very close to getting approval, and once that happens another leg of the shorts will be knocked out, and an additional revenue stream ramp will start.  That's certainly no guarantee of a higher stock price, but it should be supportive, and allow subsequent use off label. 

That said, the price is certainly frustrating.  The calls I sell help me a bit emotionally, if not that much financially.

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I feel like I am in the same boat, although I only sporadically sell PUTs. My $14s expire Friday.

Thing is... there are knowns and unknowns and ones that will be known at a predictable time.

It will be important that BAC and UBS upgrade their price targets and that JPM will initiate coverage.

If Greg and Dac can make good on the promise of the chains of ASC and boost Omidria to close to $30M in Q2, Omidria may have to be re-evaluated because with growth it becomes worth over $1B in a sale. 

When the price of narso comes out, we may see the shares rise, or fall.

Everyone needs to be warned that the coming milestone is not as simple as FDA approval, or not.
And there is always the wild card of the ISPY trial.

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

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True, but regardless of the narso pricing, actual approval reduces uncertainty, and presumably more that a potential negative pricing disappointment.  If nothing else it continues to derisk the investment.  I agree that Omidria should start to act as a floor on the stock price regardless of narso. 

I'd like to know how passing nopain or further clarification from CMS on permanent pass through would help as well.

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NOPAIN ensures 5 years of reimbursement no matter what CMS does.
CMS never awards separate reimbursement for a set period of time, except it is a limited time like the New Surgical Drug category, which was what Omdiria was in.

Normally, once approved for separate reimbursement because the drug qualifies under CMS rules, it does not lose it.
CMA approved Omidria under their anti-opioid rules and IMO Omidriaqualifies under the exparel precedent (tolong for me to explain it again).

Greg's position is that, the question of reimbursement is over. It has been granted. It would be a departure from CMS procedures to revoke it.

NOPAIN only convinces the skeptic that there is at least 5 years.

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

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Exactly, and OMER has a lot of skeptics one way or another, financially, scientifically re approval, market wise, etc;, and tearing them done one by one can only help.

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yup...although the biased tend to stay biased.
The solution is to attract more new buyers.

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

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This drug, with a recent EUA for Covid reduces mortality from 34.9% to 30.6%...not much.
But it may be a competitor for narsoplimab, because it can now be purchased and used and narso cannot.
==================
Coronavirus (COVID-19) Update: FDA Authorizes Drug for Treatment of COVID-19
June 24, 2021
Today, the U.S. Food and Drug Administration issued an emergency use authorization (EUA) for the drug Actemra (tocilizumab) for the treatment of hospitalized adults and pediatric patients (2 years of age and older) who are receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO). Actemra is not authorized for use in outpatients with COVID-19.

In clinical trials of hospitalized patients with COVID-19, Actemra in addition to the routine care patients receive for treatment of COVID-19, which included corticosteroid therapy, was shown to reduce the risk of death through 28 days of follow-up and decrease the amount of time patients remained hospitalized. The risk of patients being placed on ventilators or death through 28 days of follow-up was also decreased.

“Today’s action demonstrates the FDA’s commitment to making new therapies available through every stage of the global COVID-19 pandemic,” said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research. “Although vaccines have been successful in decreasing the number of patients with COVID-19 who require hospitalization, providing additional therapies for those who do become hospitalized is an important step in combating this pandemic.”

Actemra is a monoclonal antibody that reduces inflammation by blocking the interleukin-6 receptor. In the case of COVID-19 infection, the immune system can become hyperactive, which may result in worsening of disease. Actemra does not directly target SARS-COV-2. Actemra is a prescription medication given by intravenous infusion that is FDA-approved for multiple inflammatory diseases, including rheumatoid arthritis. Under today’s EUA, the FDA is authorizing the emergency use of Actemra for the treatment of certain hospitalized patients with COVID-19. Actemra is not approved as a treatment for COVID-19.

The issuance of an EUA is different than an FDA approval. In determining whether to issue an EUA, the FDA evaluates the totality of available scientific evidence and carefully balances any known or potential risks with any known or potential benefits of the product for use during an emergency. Based on the FDA’s review of the totality of the scientific evidence available, the agency has determined that it is reasonable to believe that Actemra may be effective in treating COVID-19 for the authorized population. And, when used to treat COVID-19 for the authorized population, the known and potential benefits of Actemra outweigh the known and potential risks for the drug. There are no adequate, approved and available alternative treatments to Actemra for the treatment of COVID-19 in hospitalized adults and pediatric patients (2 years of age or older) who are receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or ECMO.

The data supporting this EUA for Actemra are based on four clinical trials. These included one randomized, controlled, open-label, platform trial [Randomised Evaluation of COVID-19 Therapy (RECOVERY)] and three randomized, double-blind, placebo-controlled trials (EMPACTA, COVACTA and REMDACTA). While all four clinical trials contribute to the FDA’s understanding of Actemra for the treatment of COVID-19, the most important scientific evidence on the potential benefit of Actemra for its authorized use came from the RECOVERY and EMPACTA trials.

In the RECOVERY trial, 4,116 hospitalized patients with severe COVID-19 pneumonia were randomized to receive either Actemra in addition to usual care (2,022 patients) or usual care alone (2,094 patients). The primary endpoint evaluated death through 28 days of follow-up, and the results of the primary analysis were statistically significant. The probabilities of death by day 28 were estimated to be 30.7% for patients receiving Actemra and 34.9% for patients receiving usual care alone. The median time to hospital discharge was 19 days for patients receiving Actemra and more than 28 days for patients receiving usual care alone.

In the EMPACTA trial, 389 hospitalized patients with COVID-19 pneumonia were randomized to receive Actemra (249 patients) or placebo (128 patients). The primary endpoint evaluated the need for mechanical ventilation or death through 28 days of follow-up. For patients receiving Actemra, there was an observed reduction in progression to mechanical ventilation or death compared to patients who received placebo, with the primary analysis results being statistically significant. The proportion of patients who required mechanical ventilation or died by day 28 was estimated to be 12.0% for patients receiving Actemra and 19.3% for patients receiving placebo.

In the COVACTA trial, 452 hospitalized patients with severe COVID-19 pneumonia were randomized to receive Actemra (294 patients) or placebo (144 patients). The primary endpoint was clinical status through 28 days of follow-up assessed on a 7-category ordinal scale. While there was no statistically significant difference observed in clinical status on the 7-category ordinal scale at day 28 between treatment groups, the COVACTA trial contributed to the assessment of the safety for Actemra when used for the treatment of COVID-19.

In the REMDACTA trial, 649 hospitalized patients with severe COVID-19 pneumonia were randomized to receive Actemra in combination with remdesivir (430 patients) or placebo in combination with remdesivir (210 patients). The primary endpoint was time to hospital discharge or “ready for discharge” through 28 days of follow-up. Additionally, while there were no statistically significant differences observed between treatment groups with respect to time to hospital discharge or “ready for discharge” through 28 days of follow-up, the REMDACTA trial contributed to the assessment of the safety for Actemra when used for the treatment of COVID-19.

Under the EUA, fact sheets that provide important information about using Actemra in treating COVID-19 as authorized must be made available to health care providers and to patients, parents, and caregivers. These fact sheets include dosing instructions, potential side effects and drug interactions. Common side effects of Actemra observed in the COVID-19 trials include constipation, anxiety, diarrhea, insomnia, hypertension and nausea.

The EUA was issued to Genentech Inc.

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

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Waiting endlessly on narso's approval to treat serious covid cases and HSCT-TMA is NOT on my bucket list.

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You can just ignore it.
It is going to progress without our scrutiny.

On the other hand, I have modeled where we are with the Phase 2 trial, and we know that without those results, OMER will not apply for any kind of approval.

It will only be after Quantum Leap declares narsoplimab to work, based upon the Phase 2 ISPY trial that Omeros can apply for FDA approval. Knowing how slow they were for getting the full BLA submitted, I would hope none of us would be holding our breath once we hear of ISPY success.

In addition, OMER will not be approved to sell narsoplimab for CV (or anything else) unless/until adequate supplies of the drug are available to be sold. In the last presentation, at the annual meeting, Greg made a point to say that he had adequate amounts of narso for trials and HSCT-TMA commercialization, but NOT to treat CV19.

He is also not flush with cash and we have not been told whether the company is currently having more narso made.

OTOH Greg did imply that good ISPY results would hopefully lead to help (money &/or prior manufacturing & orders) that would solve this problem.

Of course, the FDA wants you to show them the cart full of narso, before they approve of you hitching up a horse.

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

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APLS has a market cap over $5 B before calculating the value of being up $1.80/share in the aftermarket.

They have no approved drugs and they just signed a deal to jointly co-develop drugs, rather than to license any existing drugs. 
***************************
Apellis, Beam to collaborate on therapies for complement-driven diseases
Jun. 30, 2021 4:42 PM ETBeam Therapeutics Inc. (BEAM), APLSBy: Jonathan M Block, SA News Editor1 Comment

Apellis Pharmaceuticals (NASDAQ:APLS) and Beam Therapeutics (NASDAQ:BEAM) will collaborate to develop therapies for complement-driven diseases.
The body's complement system is thought to play a role in immune system-related disorders, as well as diseases of the central nervous system.
The five-year agreement calls for the use of Beam's proprietary base editing technology. The companies will work on six research programs focused on several targets, including those in the eye, liver, and brain.
Base editing is a new type of precision genetic medicine that uses a chemical reaction create precise, predictable, and efficient single base changes at targeted genomic sequences without making changes in DNA strands.
Apellis has exclusive rights to license each of the six programs. Beam can enter a 50-50 U.S. co-development and co-commercialization agreement with Apellis for one of the programs.
Beam will receive a $50M payment when the agreement is signed, followed by a $25M payment one year later. The company is also eligible for milestone and royalty payments.
Apellis shares are up 1.3% to $64 and and Beam shares are up 0.6% to $129.50.
***********************************

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

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"Following talks with the FDA, ChemoCentryx on Monday filed an amendment to its application for avacopan as a treatment of anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis, resulting in a new PDUFA goal date of Oct. 7.

While the company did not explain what was contained in the amendment filing, its stock spiked by more than 10% on the news. How the agency will ultimately decide on the drug remains a mystery. The agency’s Arthritis Advisory Committee in May voted 9-9 on whether the efficacy data from a small Phase III trial support approval of avacopan, 10-8 in favor of the drug’s safety profile, and 10-8 that the benefit-risk profile is adequate to support approval.

There was a contentious debate at the meeting over whether the Phase III trial was enough to support the approval as the FDA raised concerns about the statistical analyses of the data in the trial and what effect the use of glucocorticoids on top of cyclophosphamide or rituximab in both treatment arms had on the efficacy of avacopan.

“We appreciate the opportunity to put additional data and information before the Agency, information which we believe addresses many of the issues raised at the Advisory Committee meeting,” ChemoCentryx CEO Thomas Schall said in a statement." — Zachary Brennan

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

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This is interesting for a number of reasons.
Supporters of the company were confident of success.
Some think the FDA is protecting those who overcharge for insulin.
But they got a CRL rejection at their PDUFA... something we do not want.

In addition, the last section of the PR discloses that they use their website to divulge new significant information. I find that significant because I have never seen that kind of notice before.
=======================
Provention Bio Receives Complete Response Letter (CRL) to Biologics License Application (BLA) for Teplizumab for the Delay of Clinical Type 1 Diabetes (T1D) in At-risk Individuals
Previously reported pharmacokinetic (PK) drug product comparability considerations remain outstanding

The CRL did not cite any clinical deficiencies related to the efficacy and safety data packages submitted to the BLA


(PRNewsfoto/Provention Bio, Inc.)
NEWS PROVIDED BY

Provention Bio, Inc.
Jul 06, 2021, 07:00 ET

SHARE THIS ARTICLE
     
RED BANK, N.J., July 6, 2021 /PRNewswire/ -- Provention Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to intercepting and preventing immune-mediated disease, today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the Company's Biologics License Application (BLA) for teplizumab for the delay of clinical type 1 diabetes (T1D) in at-risk individuals.

In the CRL, received late evening on July 2nd, 2021, the FDA stated that a single, low-dose pharmacokinetic/pharmacodynamic (PK/PD) bridging study in healthy volunteers to compare planned commercial product with drug product originating from drug substance manufactured for historic clinical trials had failed to show PK comparability. "As PK remains the primary endpoint for demonstration of comparability between the two products, you will need to establish PK comparability appropriately between the intended commercial product and the clinical trial product or provide other data that adequately justify why PK comparability is not necessary."

The Company expects relevant additional PK/PD data being, or to be, collected from a PK/PD substudy in patients receiving 12-days of therapy in the ongoing Phase 3 PROTECT trial in newly diagnosed T1D patients later this quarter. These data will be analyzed by independent, unblinded third-parties to maintain the integrity of this placebo-controlled trial. Upon review of the results from this substudy, the Company will determine whether to submit these data to the FDA for its review, along with any other relevant data and analyses based on our ongoing discussions with FDA, to support PK comparability or otherwise justify why PK comparability is not necessary.

In the CRL, the FDA cited several additional considerations related to product quality,  which the Company believes have either been addressed in amendments already submitted to the BLA or can be addressed in the short-term. The CRL acknowledged that the FDA had not reviewed several amendments already submitted by the Company in response to certain Chemistry, Manufacturing and Controls (CMC) information requests.

The FDA also stated that certain deficiencies conveyed during a recent general inspection, not specific to teplizumab, at a fill/finish manufacturing facility used by the Company will need to be resolved before approval.

The CRL did not cite any clinical deficiencies related to the efficacy and safety data packages submitted to the BLA and confirmed the acceptability of the proposed proprietary name for teplizumab. The FDA requested that the Company provide a safety update as part of its BLA resubmission. The CRL contained other comments and recommendations that do not impact approvability, as well as general guidance regarding the resubmission process.

"We want to recognize the patients, their families, study investigators, clinicians and T1D champions that have played such a crucial role in the development of teplizumab and thank our partners and our team of dedicated employees and consultants for their outstanding contributions. We also want to acknowledge the efforts of Drs. Yanoff and Unger and the review team at the FDA, who have worked so closely and transparently with us throughout the priority review of our BLA for this Breakthrough Therapy drug," said Ashleigh Palmer, co-founder and CEO of Provention Bio. "We know the T1D community is urgently awaiting therapeutic advancements to address their medical needs and believe our collective passion and committment will continue to drive us forward to meet this goal. We will continue to work collaboratively with the FDA to hopefully secure approval of teplizumab and bring the first disease-modifying therapy for T1D to at-risk patients as soon as possible."

The Unmet Need in Type 1 Diabetes (T1D):

Over 1.6 million Americans have T1D, an autoimmune disease caused by the destruction of beta cells. Diagnosis of T1D usually occurs in children and young adults, but it can happen at any age after symptoms appear when a person cannot make enough insulin. However, T1D starts in the body long before any symptoms and can be detected through a blood test. The psychological impact of T1D is hard to quantify, but a diagnosis is life-altering, and regular monitoring and maintenance can be extremely stressful. T1D typically takes more than a decade off a person's life, and life expectancy is reduced by 16 years on average for people diagnosed before the age of 10. Insulin therapy and glucose monitoring are currently the standard of care for treating clinical-stage T1D, and are necessary to keep T1D patients alive. The constant monitoring and administration of insulin represents a significant life-long burden for patients. No disease-modifying treatments for T1D are currently available.

About Teplizumab (PRV-031):

Teplizumab is an investigational anti-CD3 monoclonal antibody (mAb) being developed for the delay of clinical type 1 diabetes (T1D) in at-risk individuals. In the pivotal TN-10 Study, a single 14-day course of teplizumab delayed insulin-dependent, clinical-stage disease by a median of at least two years in presymptomatic patients with Stage 2 T1D compared to placebo. The observed adverse events were mechanism-based, transient, and predictable, including lymphopenia, transaminase elevations, rash, and cytokine release events.  These results were published in the New England Journal of Medicine and simultaneously presented at the American Diabetes Association meeting in 2019.  More than 800 patients have received teplizumab in multiple clinical studies involving more than 1,000 subjects. In previous studies of newly diagnosed patients, teplizumab consistently demonstrated the ability to preserve beta-cell function as shown by C-peptide, a measure of endogenous insulin production. It correspondingly reduced the need for insulin use. Teplizumab has been granted Breakthrough Therapy Designation by the FDA and PRIME designation by the European Medicines Administration. Provention is currently also evaluating teplizumab in patients with newly diagnosed insulin-dependent T1D (the Phase 3 PROTECT study).

About Provention Bio, Inc.:

Provention Bio, Inc. (Nasdaq: PRVB) is a biopharmaceutical company focused on advancing the development of investigational therapies that may intercept and prevent debilitating and life-threatening immune-mediated disease. The Company's pipeline includes clinical-stage product candidates that have demonstrated in pre-clinical or clinical studies proof-of-mechanism and/or proof-of-concept in autoimmune diseases, including type 1 diabetes, celiac disease and lupus.

Visit www.ProventionBio.com for more information and follow us on Twitter: @ProventionBio.

Internet Posting of Information:

Provention Bio, Inc. uses its website, www.proventionbio.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation F.D. Such disclosures will be included on the Company's website in the "News" section. Accordingly, investors should monitor this portion of the Company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

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

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Apellis Announces Agreements to Exchange Approximately $198.6 Million in Principal Amount of Its 3.500% Convertible Senior Notes Due 2026 for Common Stock

Jul. 07, 2021 10:06 PM ETGlobeNewswireApellis Pharmaceuticals, Inc. (APLS)
WALTHAM, Mass., July 07, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that it has entered into separate, privately negotiated exchange agreements with certain holders of its 3.500% Convertible Senior Notes due 2026 (the “Notes”). Under the terms of these exchange agreements, the holders have agreed to exchange with Apellis approximately $198.6 million in aggregate principal amount of Notes held by them for (i) 4,530,431 shares of Apellis’ common stock, which is equal to 22.8065 shares per $1,000 principal amount of Notes exchanged plus (ii) an additional number of shares of Apellis’ common stock per $1,000 principal amount of Notes exchanged equal to the quotient of (a) $449.64 divided by (b) the average of the daily volume-weighted average prices of Apellis’ common stock over the 10 consecutive trading days commencing on July 8, 2021. The exchange transactions are expected to close on July 23, 2021, subject to the satisfaction of customary closing conditions.

The shares of Apellis’ common stock issuable in the exchanges have not been registered under the Securities Act of 1933, as amended, or the securities laws of any state or other jurisdiction, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and such other jurisdictions.

This press release does not constitute an offer to sell or a solicitation to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful.

About Apellis

Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop transformative therapies for a broad range of debilitating diseases that are driven by excessive activation of the complement cascade, including those within hematology, ophthalmology, nephrology, and neurology

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

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https://www.kirkland.com/news/press-rel … g-ipo-of-k

Company IPOing may have a MASP2 drug

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

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July 14, 2021 07:16 AM EDT Deals
AstraZeneca's planned takeover of Alexion clears last regulatory review, deal to close next week
Max Gelman
Editor
The final regulatory hurdle for AstraZeneca’s mega-merger with Alexion Pharmaceuticals has been cleared, paving the way for the deal to close as soon as next week.

Britain’s financial watchdog, The UK Competition & Markets Authority, has rubber stamped AstraZeneca’s $39 billion takeover of the Boston-based rare disease biotech, the companies announced Wednesday morning. As a result, the transaction is expected to close on July 21, with Alexion shares $ALXN being converted to AstraZeneca stock $AZN and removed from Nasdaq the next day.

Shares will also be admitted to the London Stock Exchange and Nasdaq Stockholm, the two markets of AstraZeneca’s home countries of the UK and Sweden. Once the merger is wrapped up, Alexion will essentially become AstraZeneca’s entire rare disease unit with blockbuster Soliris and follow-up drug Ultomiris coming on board.


Marc Dunoyer
“We are very pleased to have secured this critical final clearance,” AstraZeneca CFO Marc Dunoyer said in a statement. “We look forward to the imminent closing of the transaction so that we may pursue our shared ambition to bring more innovative medicines to patients worldwide and begin AstraZeneca’s next chapter of growth.”

While questions surrounded the merger back when it was announced in December 2020, CEO Pascal Soriot has outlined a vision for AstraZeneca to pave a new path in rare diseases for the first half of the next decade. With a particular focus on immunology, he’s said he expects Alexion to help drive growth in the field to the tune of double digit revenue through 2025.

The rosy projections still have to perform, however, and many are likely to scrutinize the pipeline AstraZeneca is acquiring in the deal. Andexxa, one of Alexion’s approved medicines to treat acutely uncontrolled bleeding of Factor Xa inhibitors, returned disappointing sales numbers and proved pivotal in mounting activist pressure on CEO Ludwig Hantson in 2020.


Ludwig Hantson
Dunoyer, who will lead the new Alexion subsidiary when the deal is complete, told Endpoints News in an interview last month that AstraZeneca doesn’t plan to auction off Andexxa, and instead hopes to turn it around in a similar fashion to the drugmaker’s Brilinta drug.

Of course, it does help that AstraZeneca will immediately benefit from Soliris, which raked in more than $4 billion in sales in 2020, as well as Ultomiris, the planned successor for Soliris. Alexion has positioned Ultomiris to soak up most of the sales from the older drug once it hits its patent cliff later this decade, despite some new competition.

Wednesday’s greenlight from the UK was largely expected following the FTC signing off on the deal in April and the EU signaling its approval last week. The FTC review came despite President Joe Biden’s administration saying it would take a harsher stance on Big Pharma mergers in March, but none of the feared antitrust measures came to pass.

One reason may have been the lack of a pipeline overlap between the two companies, according to an analysis from Evaluate Pharma at the time. None of the companies’ marketed drugs have any crossover in indications, and the only pipeline candidate that could be seen as similar is Alexion’s cerdulatinib — a potential intersection with AstraZeneca’s blood cancer franchise.

And while the Alexion deal had been viewed by some as a potential bellwether for the industry, the FTC has been harsher on other companies so far this year. The commission has sued to block Illumina’s $8 billion buyout of Grail, expressing concerns over Illumina’s potential stranglehold on the DNA sequencing market.

The Illumina merger is running up against new headwinds as well, with the EU expected to launch a full-scale antitrust probe at the end of its review next week, Reuters reported Tuesday.

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

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https://api.wsj.net/api/kaavio/charts/big.chart?nosettings=1&symb=kdmn&uf=0&type=2&size=2&sid=23562650&style=320&freq=9&entitlementtoken=0c33378313484ba9b46b8e24ded87dd6&time=1&rand=4367068&compidx=NASDAQ&ma=0&maval=9&lf=1&lf2=0&lf3=0&height=335&width=579&mocktick=1

MAJOR AMENDMENT leads to PDUFA extended to August 30th but approval comes on July 16th, 45 days earlier than scheduled. See the two PRs below.

Kadmon Announces U.S. FDA Has Extended the Review Period for Belumosudil in Chronic Graft-Versus-Host Disease
March 10, 2021 at 4:14 PM EST
PDF Version
NEW YORK, NY / ACCESSWIRE / March 10, 2021 / Kadmon Holdings, Inc. (NASDAQ:KDMN) today announced that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for belumosudil for the treatment of chronic graft-versus-host disease (cGVHD). In a notice received from the FDA on March 9, 2021, the Company was informed that the Prescription Drug User Fee Act (PDUFA) goal date for its Priority Review of belumosudil has been extended to August 30, 2021.
The FDA extended the PDUFA date to allow time to review additional information submitted by Kadmon in response to a recent FDA information request. The submission of the additional information has been determined by the FDA to constitute a major amendment to the NDA, resulting in an extension of the PDUFA date by three months.

"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," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We are committed to bringing belumosudil to market, once approved, to help meet the needs of patients living with cGVHD."

About Belumosudil
Belumosudil (KD025) is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and pro-fibrotic processes. In November 2020, the U.S. Food and Drug Administration (FDA) accepted and granted Priority Review for the NDA for belumosudil for the treatment of patients with cGVHD. The NDA is being reviewed under the FDA's Real-Time Oncology Review (RTOR) and Project Orbis pilot programs. The FDA has granted Breakthrough Therapy Designation to belumosudil for the treatment of patients with cGVHD after failure of two or more lines of systemic therapy as well as Orphan Drug Designation to belumosudil for the treatment of cGVHD.

About cGVHD
cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD.

About Kadmon
Kadmon is a clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Kadmon's clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.
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U.S. FDA Grants Full Approval of REZUROCK(TM) (belumosudil) for the Treatment of Patients with Chronic Graft-Versus-Host Disease (cGVHD)
July 16, 2021 at 2:20 PM EDT
PDF Version
- REZUROCK is approved for the treatment of adult and pediatric patients 12 years and older with cGVHD after failure of at least two prior lines of systemic therapy -
- Kadmon to Host Conference Call on Monday, July 19, 2021 at 8:00 a.m. ET -

NEW YORK, NY / ACCESSWIRE / July 16, 2021 / Kadmon Holdings, Inc. (Nasdaq:KDMN) today announced that the U.S. Food and Drug Administration (FDA) has approved REZUROCK™ (belumosudil) 200 mg once daily (QD) for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy. The FDA granted Breakthrough Therapy designation and Priority Review for REZUROCK and reviewed the New Drug Application (NDA) under the Real-Time Oncology Review (RTOR) pilot program. The FDA approved this NDA six weeks ahead of the Prescription Drug User Fee Act (PDUFA) goal date of August 30, 2021. REZUROCK is the first and only FDA-approved small molecule inhibitor of ROCK2, a signaling pathway that modulates inflammatory responses and fibrotic processes.

"REZUROCK represents a new treatment paradigm for thousands of cGVHD patients, including those with difficult-to-treat manifestations like fibrosis," said Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine at Harvard Medical School and Medical Director, Adult Stem Cell Transplantation Program at the Dana-Farber Cancer Institute. "REZUROCK has shown robust and durable responses across the spectrum of cGVHD and is safe and well tolerated, allowing patients to stay on therapy and achieve meaningful benefit from treatment."

The FDA approval of REZUROCK is based on safety and efficacy results from ROCKstar (KD025-213), a randomized, open-label, multicenter pivotal trial of REZUROCK in patients with cGVHD who had received two to five prior lines of systemic therapy. There were 65 patients treated with REZUROCK 200 mg taken orally QD. The median time from cGVHD diagnosis was 25.3 months and 48% of patients had four or more organs involved. Patients had cycled through a median of 3 prior lines of systemic therapy and 78% were refractory to their last therapy. REZUROCK 200 mg QD achieved an Overall Response Rate (ORR) of 75% through Cycle 7 Day 1 of treatment (95% Confidence Interval (CI): 63, 85), with 6% achieving a complete response and 69% achieving a partial response. The median time to first response was 1.8 months. Sixty-two percent (62%) of responders did not require new systemic therapy for at least 12 months following response. The median duration of response, calculated from first response to progression, death, or new systemic therapies for chronic GVHD, was 1.9 months. ORR results were supported by clinically meaningful improvement from baseline in the Lee Symptom Scale (LSS) score, a chronic GVHD symptom measurement, in 52% of patients through Cycle 7 Day 1 of treatment.

"Patients receiving REZUROCK reported significant improvements in cGVHD symptoms, showing that not only did treatment result in organ responses, but it also made people feel better. This is so important for a chronic disease with a high symptom burden," said Stephanie Lee, MD, MPH, Professor at the Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine, and Research Director of the Long-Term Follow-Up Program at Fred Hutchinson.

REZUROCK has been well tolerated and adverse events have been consistent with those expected in patients with advanced cGVHD receiving corticosteroids and/or other immunosuppressants.

"We are proud to introduce REZUROCK as a new treatment that uniquely addresses the underlying inflammatory and fibrotic pathophysiology of chronic GVHD," said Harlan W. Waksal, MD, President and CEO of Kadmon. "Thank you to the patients, their families and caregivers, who are the center of our focus in achieving this significant milestone. We have built a hematology/oncology-experienced commercial team and we look forward to rapid adoption of REZUROCK for patients in need."

REZUROCK is expected to be available in the United States by late August 2021.

Kadmon is committed to helping patients with treatment access and support. Kadmon ASSIST™ is a program designed to help and support REZUROCK patients and their caregivers throughout their treatment journey. This program provides reimbursement assistance and savings programs for eligible patients. For more information, please call 1-844-KADMON1 (1-844-523-6661), Monday-Friday, 8:00 a.m.to 8:00 p.m. ET.

The NDA for REZUROCK is part of Project Orbis, an initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology drugs among participating international health authorities.

Conference Call and Webcast

Kadmon will host a conference call on Monday, July 19, 2021 at 8:00 a.m. ET to discuss the FDA approval of REZUROCK.

To participate in the conference call, please dial (866) 762-3021 (domestic) or +1 (703) 925-2661 (international) and reference the conference ID: 5399837.

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

118

Re: OMER Comparators, Competitors, VALUATION & potential partners

More companies with PDUFA dates scheduled that should be watched:

ETON Drug: Topiramate Date: 6 Aug 2021 PDUFA

SESN Drug: Vicinium  Date: 18 Aug 2021 PDUFA

VRCA Drug: VP-102   Date: 23 Sept 2021 PDUFA

One of these is up big in anticipation.
The others are acting crappy, like Omeros.
https://api.wsj.net/api/kaavio/charts/big.chart?nosettings=1&symb=OMER&uf=0&type=2&size=2&sid=3942233&style=320&freq=1&entitlementtoken=0c33378313484ba9b46b8e24ded87dd6&time=6&rand=1377090344&compidx=aaaaa%3a0&comp=KDMN%2c+ETON%2c+SESN%2c+VRCA&ma=0&maval=9&lf=1&lf2=0&lf3=0&height=335&width=579&mocktick=1

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

119

Re: OMER Comparators, Competitors, VALUATION & potential partners

BTD for narsoplimab in HSCT-TMA led to lots of discussions with FDA that led to Omeros able to convince FDA to accept a small N, no comparator, Phase 2 trial as a Pivotal trial. That is NOT always the case.
https://endpts.com/small-struggling-bio … collapses/
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July 26, 2021 07:10 AM EDT R&D
Small, struggling biotech winds up with a 3X loser as another PhIII of its lead drug collapses
John Carroll Editor & Founder
ENDPOINTS

Little Tonix Pharmaceuticals has run into another brick wall as its lead drug — a reformulated muscle relaxant originally approved 44 years ago — has failed another Phase III study, sending shares back into penny stock territory.

Three years after going down in their first Phase III trial of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) for symptoms of PTSD, the biotech — which had been encouraged by a breakthrough designation at the FDA — reported late Friday the drug also failed its second late-stage challenge for pain associated with fibromyalgia. Outside data monitors recommended the Phase III trial be halted for futility after deciding interim data made it unlikely the drug would pass muster.

Researchers will now halt enrollment and continue to follow patients already on the drug.

Tonix shares $TNXP, which earlier had to be rehabilitated to stay on Nasdaq, crumbled — dropping 35% and leaving shares at 64 cents at the start of trading Monday.

The failure follows Tonix’s initial reported success in its first Phase III fibromyalgia study.

The drug failed a late-stage trial for symptoms of PTSD in 2018, sending execs back to the drawing board with another Phase III PTSD trial, which also failed. They still have it in the pipeline for PTSD, shifting focus yet again to sleep disturbance.

The latest failure at Tonix underscores that breakthrough designations may make it easier to discuss things with regulators, but it’s no guarantee of success in a clinical trial.

“We are surprised and disappointed that the interim analysis did not support continued enrollment in this Phase 3 RALLY study, especially considering the previous Phase 3 RELIEF study, which had a similar design and achieved statistical significance on the primary endpoint. After the currently enrolled participants complete the study, we will proceed with a full analysis of the unblinded data from the study to determine the next steps in this program,” commented CEO Seth Lederman.

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