Two people believed to be FDA inspectors leave the Juno facility in Bothell, MA, as captured by a spotter hired by an investor. (via Mizuho analyst Salim Syed)

Bris­tol My­ers hints at some prob­lems with an FDA in­spec­tion need­ed for the liso-cel OK, and the Cel­gene CVR in­vestors are pan­ick­ing

The nail-bit­ing among the in­vestors who held on to their con­tin­gent val­ue rights $BMYRT from the Cel­gene buy­out is giv­ing way to pan­ic to­day.

Dur­ing their Q3 call with an­a­lysts, ex­ecs said that the FDA had in­spect­ed the Both­ell, WA plant be­ing used for liso-cel (JCAR017), the CAR-T Bris­tol My­ers picked up in the deal. That’s a plus.

But there was a hitch.

They went on to say that no in­spec­tion had yet even been sched­uled for the Texas CMO in­volved. And with that, the slow trick­le of in­vestors seep­ing out of the CVR group turned in­to an in­stant rout. The stock plunged about 80% from the $3.40 still left for the CVR at the end of the day yes­ter­day. That took it down in­to pen­ny stock ter­ri­to­ry.

Why the stam­pede?

Sal­im Syed

Re­mem­ber that dur­ing last-minute buy­out ne­go­ti­a­tions, Bris­tol My­ers CEO Gio­van­ni Caforio in­sist­ed that they re­place cash on the ta­ble with a $9 CVR tied to the ap­proval of 3 drugs by cer­tain dates. Ozan­i­mod made the dead­line, but the fate of liso-cel (JCAR017) and ide-cel are hang­ing on FDA ac­tions. Liso-cel was de­layed and ide-cel got slapped with a refuse-to-file. That drug has un­til the end of Q1 next year to make good.

As Bris­tol My­ers notes in its Q3 pre­sen­ta­tion to­day, there’s a Nov. 16 PDU­FA date loom­ing for liso-cel, and with­out a clean bill of health on CMC, reg­u­la­tors aren’t like­ly to come through with an OK for a once close­ly-watched ther­a­py that earned break­through sta­tus at the FDA. That could spell an­oth­er sig­nif­i­cant de­lay.

Sal­im Syed at Mizuho, who’s been track­ing every tiny de­tail around the CMC work — in­clud­ing pri­vate eye re­ports com­plete with tele­pho­to lens­es on the Both­ell in­spec­tion — summed it up here:

The way it was couched on the call was that this is due to COVID-19 and the FDA want­i­ng to keep its em­ploy­ees safe. How­ev­er, in­vestors were ex­pect­ing, and what seemed to make most log­i­cal sense, is to in­spect the plants to­geth­er close­ly in se­quence, Both­ell and Texas.

Syed, though, is urg­ing calm in the melee. While the PDU­FA is dat­ed Nov. 16, the dead­line on the CVR is the end of this year — still some weeks out.

Re­mem­ber, the FDA on­ly needs here about ~3 weeks (by rea­son­able stan­dards and our KOL call) post-in­spec­tion to fin­ish up all the pa­per­work and get a drug to the fin­ish line.

Be­sides, he adds, this is a BTD pro­gram, so the FDA should be will­ing to hur­ry up. The ex­ecs at Bris­tol My­ers nev­er said if there could be a vir­tu­al in­spec­tion and be­sides, the com­pa­ny al­ready said that if they learned that they would miss the PDU­FA, they’d say so prompt­ly.

So far, so good on that score. The PDU­FA re­mains in place.

But with the dead­line draw­ing very, very close, a lot of the in­vestors are bolt­ing.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

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Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Covid-19 man­u­fac­tur­ing roundup: Mary­land looks to grow biotech ca­pac­i­ty with $400M check; Rus­sia lands sec­ond Sput­nik V part­ner this week

A Maryland real estate project has added three new biotech-focused manufacturing and research buildings to an office park to keep up with demand created by the pandemic, the Washington Business Journal reported.

The Milestone Business Park — located off of I-270 in Germantown, MD — will see the new buildings and a total of 532,000 square feet as the campus rebrands to Milestone Innovation Park.

Michel Vounatsos, Biogen CEO (Biogen via YouTube)

Damn the crit­ics, Bio­gen CEO Vounatsos or­ders full speed ahead on prep­ping a con­tro­ver­sial ad­u­canum­ab launch as FDA de­ci­sion looms

Right now one of the most interesting parlor games on Wall Street is offering odds on Biogen’s chances of getting an FDA OK on their controversial Alzheimer’s drug aducanumab.

For most objective players, it looks about like a coin toss, maybe a little worse than 50/50, as the Street balances the bull case of a full, mega blockbuster approval, a restricted approval or a disastrous order to go back to the clinic and mount a new Phase III. That last option was clearly the guidance most of the outside experts in the panel review offered the agency, as the industry is still puzzling out the question of whether or not the FDA is getting tougher in its oversight of drug development.

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