Ax­o­vant: That pos­i­tive p-val­ue we re­port­ed yes­ter­day? Um, we screwed that up too

Yes­ter­day Ax­o­vant ex­ecs watched their stock price $AX­ON col­lapse af­ter the biotech found­ed by the youth­ful for­mer hedge fund man­ag­er Vivek Ra­maswamy was forced to con­cede that its lead drug was worth­less.

David Hung

To­day, the com­pa­ny led by CEO David Hung fol­lowed up with the ex­tra­or­di­nar­i­ly em­bar­rass­ing ad­mis­sion that it man­aged to in­cor­rect­ly re­port the p val­ue for the one clear­ly pos­i­tive end­point they had tout­ed as a sign that the biotech’s sec­ond drug, nelotanserin, was wor­thy of a piv­otal tri­al.

In­stead of min­ing a pos­i­tive end­point from the da­ta on nelotanserin, post hoc, they had an­oth­er fail to ex­plain. And it was bad.

Tues­day morn­ing Ax­o­vant put out a re­lease that their post-hoc sub­set analy­sis of Parkin­son’s pa­tients with a base­line SAPS-PD score greater than 8.0 (n=19) showed that a 40 mg for two weeks fol­lowed by 80 mg for two weeks did in­deed re­sult in a 1.21 point im­prove­ment.

But…

That p=0.011 fig­ure they post­ed was wrong. In fact, it was an in­fi­nite­ly worse p=0.531. In­stead of find­ing some rea­son for hope, the da­ta in­di­cat­ed Ax­o­vant had even more rea­son to doubt suc­cess. And in­stead of go­ing to the FDA look­ing for guid­ance on next steps in in­ves­ti­gat­ing the drug for de­men­tia with Lewy bod­ies (DLB) pa­tients with mo­tor deficits as well as pa­tients with more se­vere base­line psy­chot­ic symp­toms, they’ll be stick­ing with DLB.

Vivek Ra­maswamy

This, in the biotech world, is what is tech­ni­cal­ly called a world-class mess up. Ex­cept they don’t use the word mess.

Ra­maswamy has been able to raise $2 bil­lion from in­vestors based on his claim that the can find as­sets on the shelves of bio­phar­ma com­pa­nies and quick­ly hus­tle them to ap­provals. To­day, the biotech ex­ec — who had been straight­for­ward in pre­sent­ing the first key fail­ure for Alzheimer’s — is keep­ing a low pro­file as the com­pa­ny tries to set the record straight on the num­bers.

It’s not a good week for the com­pa­ny.

Un­lock­ing ESG strate­gies for growth with Gilead Sci­ences

RBC Capital Markets explores what is material in ESG for biopharma companies with the ESG leads at Gilead Sciences. Gilead has long focused on sustainability but recognized a more robust framework was needed. Based on a materiality assessment, Gilead’s ESG strategy today focuses first on drug access and pricing, while also addressing D&I and climate change. Find out why Gilead’s board is “acutely aware” of the contribution that ESG makes to firm’s overall success.

What con­tro­ver­sy? Eli Lil­ly plots Alzheimer's BLA fil­ing lat­er this year as FDA taps more an­ti-amy­loid drugs as break­throughs

The FDA is keeping the good news coming for Alzheimer’s drug developers. And Eli Lilly is taking them up on it.

Amid continued controversy around whether Biogen’s new flagship drug, Aduhelm, should have been approved at all — and swelling, heated debates surrounding its $56,000 price tag — the agency had no issue handing them and their Japanese partner Eisai a breakthrough therapy designation for a second anti-amyloid beta antibody, lecanemab, late Wednesday.

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Hervé Hoppenot, Incyte CEO (Jeff Rumans)

ODAC echoes FDA con­cern over In­cyte PD-1, as Paz­dur sig­nals broad­er shift for ac­cel­er­at­ed ap­proval

After the FDA lambasted their PD-1 ahead of an adcomm earlier this week, Incyte ran into new trouble Thursday as ODAC panelists voted against an accelerated OK by a wide margin.

Members of the Oncologic Drugs Advisory Committee recommended with a 13-4 vote to defer a regulatory decision on Incyte’s retifanlimab until after more data can be collected from a placebo-controlled trial. The PD-1 therapy is due for a PDUFA date in late July after receiving priority review earlier this year.

Richard Pazdur (vis AACR)

FDA en­cour­ages in­clud­ing in­cur­able can­cer pa­tients in tri­als, re­gard­less of pri­or ther­a­pies

The FDA on Thursday called to include those with incurable cancers (when there is no potential for cure or for prolonged/near normal survival) in appropriate clinical trials, regardless of whether they have received existing alternative treatments.

Historically, many cancer clinical trials have required that participating patients previously received multiple therapies, according to Richard Pazdur, director of the FDA’s Oncology Center of Excellence.

New FDA doc­u­ments show in­ter­nal dis­sent on Aduhelm ap­proval

In a lengthy review document and a pair of memos from top officials, the FDA released on Tuesday night its most detailed argument yet for approving Biogen’s intensely controversial Alzheimer’s drug aducanumab.

The documents amount to an agency attempt to quench the firestorm their decision kindled, as outside advisors members resigned and experts warned that an unproven drug now could stretch Medicare’s budget to a breaking point. Ultimately, the documents show how CDER director Patrizia Cavazzoni and Office of New Drugs director Peter Stein both concurred with FDA neuroscience head Billy Dunn on the accelerated approval while the staff at FDA’s Office of Biostatistics did not think an approval was warranted.

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Karen Flynn, Catalent

Q&A: When the pan­dem­ic struck, Catal­en­t's CCO had just joined the team

Karen Flynn came aboard Catalent’s team just in time.

The company was going through a surge of changes, and she had been brought over from her role as CCO of West Pharmaceutical Services to serve in the same capacity for the New Jersey-based CDMO. Then a few months later, the pandemic was in full-force.

Since then, Catalent’s been in hyper-expansion mode. In early May, it acquired Promethera’s Hepatic Cell Therapy Support SA subsidiary and its 32,40-square-foot facility in Gosselies, Belgium. Prior to that, the company acquired Belgian CDMO Delphi Genetics, wrapped up the expansion of an already-existing site in Madison, WI and added an ultra-low temperature freezer partner in Sterling. As Emergent has botched millions of doses of AstraZeneca’s vaccine, the company has swooped in to move that production to its Maryland plant as well.

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Yuval Cohen, Corbus CEO (Corbus via YouTube)

An­oth­er Cor­bus pro­gram hits the skids af­ter late-stage flop, plum­met­ing the small biotech's shares

Corbus Pharmaceuticals’ plans to position lenabasum as a pipeline-in-a-product aren’t going so well.

After shelving a program in scleroderma, the Norwood, MA-based biotech has revealed that its lead candidate failed both the primary and secondary endpoints in another Phase III trial.

Lenabasum failed to show a statistically significant difference in total improvement compared with placebo in treating dermatomyositis, a rare disease that causes muscle inflammation and skin rash, the company said Thursday. The news sent Corbus’ $CRBP stock spiraling around 30% early Thursday morning.

On heels of Aduhelm ap­proval, Bris­tol My­ers jumps back in­to Alzheimer's race

Bristol Myers Squibb last put major resources behind an Alzheimer’s drug nearly a decade ago, when their own attempt at targeting amyloid flamed out in mid-stage studies. They invented another molecule, a Tau-targeted antibody, but jettisoned it to Biogen in 2017 as they dropped out of neuroscience altogether.

But on Thursday, the New York pharma announced they were getting back in the game. Bristol Myers exercised an $80 million option to bring a tau-targeted antibody from Prothena into a Phase I study. The opt-in, which Bristol Myers triggered ahead of analyst expectations, opens the door for another $1.7 billion in milestones down the road.

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Elizabeth Warren (Michael Brochstein/Sipa USA)(Sipa via AP Images)

Sen­a­tors call for hear­ing to ex­am­ine how Medicare will han­dle Bio­gen's new Alzheimer's drug

Two top Senate Finance committee senators on Thursday called for a hearing to examine the questions and challenges for Medicare arising from the FDA’s recent approval of Biogen’s Aduhelm, the controversial new drug approved to treat Alzheimer’s disease.

In a letter to Senate Finance chair Ron Wyden (D-OR) and ranking member Mike Crapo (R-ID), subcommittee chair Elizabeth Warren (D-MA) and Bill Cassidy (R-LA) hinted at making policy changes to enable Medicare to more directly connect prescription drug pricing to clinical effectiveness. They raised questions about the “dramatic implications for our health care system” from the approval, which they said “stretch well beyond the scope of FDA’s jurisdiction.”

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