Bris­tol My­ers bags a long-await­ed FDA OK for ozan­i­mod — but the pan­dem­ic will keep it off the mar­ket

The FDA has come through with the long-awaited approval of ozanimod, the once top MS drug hopeful that was sidelined by the agency 2 years ago after Celgene was flagged for lame conduct in filing a drug application. But the global Covid-19 outbreak will keep it on the sidelines for awhile longer.

Now a new product for Bristol Myers Squibb in the wake of its $74 billion Celgene buyout, the drug will eventually be pitted against Novartis’ S1P1 rival Mayzent, which slipped ahead in the drug development race as Celgene stumbled and fell. J&J has its own rival in the field, ponesimod, which was recently filed for an OK of its own.

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5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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CEO Stephen Yoder (Pieris)

Pieris fi­nal­ly vaults FDA hold on next-gen sol­id tu­mor hunter, clear­ing the path for mid-stage tri­al

Finally freed from the restraints of a partial FDA clinical hold on its lead HER2-positive solid tumor candidate, Pieris Pharmaceuticals is now racing toward Phase II.

The FDA slapped a partial hold on Pieris’ PRS-343 back in July, restricting the biotech from enrolling new patients in a Phase I trial. While Pieris was allowed to continue dosing patients who were already enrolled, the agency requested they conduct an additional “in-use and compatibility study” before recruiting any more.

FDA hits the brakes on His­to­gen's knee car­ti­lage ther­a­py, ask­ing for more in­fo on man­u­fac­tur­ing process

A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Bris­tol My­ers Squibb gets re­view date for Op­di­vo com­bo in gas­tric can­cer, look­ing to over­turn Keytru­da's 3-year lead

The past two months have been tough for Bristol Myers Squibb and its checkpoint inhibitor Opdivo after setbacks in lung and brain cancers. But in the battle against Merck’s Keytruda, any success matters — and now Bristol could be looking at a quick approval for Opdivo in an unmatched indication.

The FDA will launch a speedy review of a combination of Bristol Myers Squibb’s Opdivo and chemotherapy to treat first-line patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer or esophageal adenocarcinoma, the drugmaker said Wednesday. The agency set an action date of May 25 for the application.

Cardiovascular chief Joerg Koglin (Merck)

Mer­ck makes good on big CV gam­ble with veri­ciguat nod — but com­pe­ti­tion will be fierce

The results for Merck’s leading heart drug fell short of what some cardiologists had hoped for, but they proved more than enough to convince the FDA, which handed down an approval Wednesday.

The agency OK’d vericiguat, now marketed as Verquvo, for patients with heart failure, offering one of the first new pharmaceutical tools to tackle a hard-to-treat group of patients at high risk for what remains the leading cause of death in the US and Europe. It’s also a notable win for Merck, who rolled the dice on the molecule in 2016, paying Bayer $1 billion for US rights to the then-experimental drug and ex-US rights for the approved CV drug Adempas.

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News brief­ing: Ve­rastem CMO ex­its two weeks af­ter join­ing com­pa­ny; Ther­mo Fish­er inks $550M M&A deal

Two weeks after joining Verastem Oncology as chief medical officer, Frank Neumann is leaving the company for another job.

Neumann had joined Verastem after leaving bluebird bio, which surprisingly split into two companies last week, one in oncology and one in rare diseases. It’s not yet clear to where Neumann is headed next, but he noted in a statement that Verastem’s data and strategy were “truly exciting.”

Covid-19 claims an­oth­er PDU­FA vic­tim as Glax­o­SmithK­line push­es back planned PD-1 roll­out

Bristol Myers Squibb isn’t the only pharma giant that’s been standing in the FDA’s waiting line for site inspections.

GlaxoSmithKline is telling us today that their H2 2020 PDUFA deadline for the PD-1 drug dostarlimab — picked up in its Tesaro buyout — was pushed back due to a delay in the manufacturing site inspection needed for a regulatory decision. And that is forcing the company to revise its timeline.

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