FDA sends small biotech and its re­for­mu­lat­ed pain med pack­ing, trig­ger­ing a col­lapse in the share price

A small-cap biotech found­ed by Fortress has run in­to a brick wall at the FDA.

Av­enue Ther­a­peu­tics $ATXI says the agency has hit it with a com­plete re­sponse let­ter for its pitch on IV tra­madol to treat post-op­er­a­tive pain. Ac­cord­ing to the biotech, reg­u­la­tors said that the IV ap­proach wasn’t safe, as any ad­di­tion­al need for quick pain re­lief would like­ly lead to “opi­oid stack­ing.”

The drug hasn’t re­ceived much at­ten­tion from an­a­lysts, but in­vestors clear­ly thought that the re­for­mu­la­tion was a shoo-in. Ahead of the bell this morn­ing, its shares were rout­ed, plung­ing 57%.

From Av­enue:

Specif­i­cal­ly, if a pa­tient re­quires an anal­gesic be­tween the first dose of IV tra­madol and the on­set of anal­ge­sia, a res­cue anal­gesic would be need­ed. The like­ly choice would be an­oth­er opi­oid, which would re­sult in opi­oid “stack­ing” and in­crease the like­li­hood of opi­oid-re­lat­ed ad­verse ef­fects. Oth­er than this po­ten­tial safe­ty con­cern, the FDA did not iden­ti­fy a safe­ty sig­nal in Av­enue’s clin­i­cal de­vel­op­ment pro­gram. In ad­di­tion, the CRL stat­ed that the FDA re­quires an ad­e­quate ter­mi­nal ster­il­iza­tion val­i­da­tion pri­or to NDA ap­proval, which is planned for lat­er this quar­ter.

Av­enue CEO Lucy Lu not­ed in a pre­pared state­ment that Av­enue is stick­ing with its pro­gram for re­for­mu­lat­ed Tra­madol — an old gener­ic first ap­proved 25 years ago — and will look to se­cure a meet­ing AS­AP with the FDA to see what it will take to get the ap­pli­ca­tion back on track.

Av­enue’s reg­u­la­to­ry strat­e­gy had rest­ed sig­nif­i­cant­ly on the idea of of­fer­ing pa­tients a non-opi­oid op­tion as they stepped through pain meds — fit­ting in­to the FDA’s en­cour­age­ment of non-opi­oids in the midst of wide­spread abuse. And in­ves­ti­ga­tors high­light­ed ev­i­dence of its ef­fi­ca­cy over a place­bo while al­so post­ing com­par­a­tive re­sults to 4 mg of IV mor­phine.

Av­enue is part of the Fortress sta­ble of biotech start­up com­pa­nies.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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Genen­tech to stop com­mer­cial man­u­fac­tur­ing at Cal­i­for­nia head­quar­ters

Genentech is halting commercial manufacturing at its California headquarters — and laying off several hundred employees.

The move is the result of a decision Genentech made in 2007 to relocate manufacturing operations from its South San Francisco headquarters location to other facilities or move the work to CDMOs, said Andi Goddard, Genentech’s SVP of quality and compliance for pharmaceutical technical operations, in an interview with Endpoints News. Genentech has made changes in capabilities and invested more in technology, so it doesn’t need as many large-scale manufacturing facilities as it did in the past, she said.

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In­cyte wins ac­cel­er­at­ed ap­proval for PD-1 in rare skin can­cer

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.

Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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FDA re­jects Ab­b­Vie's in­fu­sion ther­a­py for Parkin­son's, re­quests more in­fo on pump de­vice

The FDA rejected AbbVie’s 24-hour infusion therapy for Parkinson’s, saying it needs more information on a device used to administer the treatment before it can clear it.

The Chicago-area drugmaker said in a press release that the complete response letter from the agency didn’t include any requests for more efficacy or safety trials related to the drug, known as ABBV-951. The company said it aims to “resubmit the NDA as soon as possible.”

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FDA+ roundup: Leg­isla­tive asks for 2024 fo­cus on more au­thor­i­ties, gener­ic drug com­pe­ti­tion

The FDA’s legislative priorities for the next year highlight the agency’s focus on expanding generic drug competition, backstopping the supply chain and growing its current authorities.

On the new authorities front, FDA is seeking to expand its mandatory recall authority for all drugs, as the agency has been embroiled in a long process to remove some from the market. Covis Pharma refused to pull its preterm birth drug Makena, which won accelerated approval, for almost five years after failing its confirmatory trial. The company has since reversed course after a negative adcomm.

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FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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