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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs covers a wide range of indications that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

PhRMA sues Trump gov­ern­ment over drug im­por­ta­tion rule — days be­fore it's set to be ef­fec­tive

Ever since President Donald Trump floated the idea of using state-sponsored importation to lower drug prices, PhRMA has made its opposition abundant. Not only is the proposal dangerous and futile,  but the trade group has also argued that it may even be illegal.

Now that the FDA has issued its final rule permitting states to bring certain drugs from Canada, PhRMA is taking the government to court — just a few days before the rule is slated to take effect.

Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

Overnight for­tunes are be­ing made in biotech these days — and it's both en­cour­ag­ing and more than a lit­tle bit scary

Just to complete the last leg of a running story I’ve been tracking for a few weeks, Olema $OLMA has come through its IPO from the Thursday night pricing at $19 a share with a market cap just north of $2 billion.

That leaves newly-named CEO Sean Bohen holding a batch of 1,110,896 shares with a strike price of $4.82. As of Tuesday morning, the stock is now trading at $53.40, giving him a portfolio value of $53.4 million. Not bad for someone who was hired in September.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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