Just in time to as­sure a de­ci­sion be­fore CVR dead­line, Bris­tol My­ers Squibb files NDA for Cel­gene/blue­bird CAR-T

A new CAR-T ther­a­py may be com­ing. And maybe $9 per share for Cel­gene in­vestors, too.

Bris­tol My­ers Squibb an­nounced they sub­mit­ted an NDA for the mul­ti­ple myelo­ma “ide-cel” CAR-T ther­a­py Cel­gene de­vel­oped in part­ner­ship with blue­bird. The ther­a­py is one of the three that has to be ap­proved to un­lock the BMS-Cel­gene con­tin­gent val­ue agree­ment that would give share­hold­ers of the ab­sorbed NJ com­pa­ny $9 per share. The first, ozan­i­mod, was ap­proved last week. The sec­ond, a CAR-T treat­ment for non-Hodgkin’s lym­phoma called liso-cel, was sub­mit­ted to the FDA in De­cem­ber.

As Mizuho’s Sal­im Syed point­ed out in a note to in­vestors, the tim­ing is not ran­dom. The March 31 sub­mis­sion means that even un­der stan­dard re­view, the FDA will is­sue a de­ci­sion by March 31, 2021, the dead­line for ap­proval un­der the con­tin­gent val­ue agree­ment.

Strong num­bers bol­ster BMS’s sub­mis­sion. At ASH in De­cem­ber, they re­leased Phase II da­ta show­ing that in 140 pa­tients with re­lapsed and re­frac­to­ry mul­ti­ple myelo­ma, around three quar­ters re­spond­ed and around a third showed a com­plete re­sponse. In the most ef­fec­tive dos­ing arm, 81.5% re­spond­ed and 35.2% went in­to com­plete re­mis­sion. The me­di­an du­ra­tion was 11.3 months.

BMS isn’t alone in the hunt, al­though they are for now the fur­thest along. At the same con­fer­ence, J&J un­veiled a batch of ear­li­er stage da­ta. It was small­er — 29 pa­tients — but every pa­tient re­spond­ed by month 6. Both ther­a­pies tar­get BC­MA, or B-cell mat­u­ra­tion agent, a pro­tein ex­pressed al­most ex­clu­sive­ly on ma­lig­nant cells.

The ther­a­py, though, comes with the same po­tent side ef­fects as pre­vi­ous CAR-T treat­ments. Cy­tokine re­lease syn­drome, the po­ten­tial­ly dead­ly side ef­fect in which ac­ti­vat­ed white blood cells send the im­mune sys­tem in­to in­flam­ma­to­ry over­drive, oc­curred in 83% of pa­tients in the ide-cel tri­al. How­ev­er, on­ly 5.5% were clas­si­fied as “se­vere.”

Liso-cel and ide-cel would be the third and fourth CAR-T treat­ments ap­proved since No­var­tis’s Kym­ri­ah and Gilead/Kite Phar­ma’s Yescar­ta and were ap­proved in 2017, al­though a sec­ond CAR–T ther­a­py from Kite Phar­ma al­so cur­rent­ly sits be­fore the FDA.

Af­ter the ASH da­ta, an­a­lysts ex­pect both BMS CAR-Ts to clear the fin­ish line, al­though Syed raised ques­tions about whether, amid Covid-19, the com­pa­ny’s cell ther­a­py man­u­fac­tur­ing fa­cil­i­ty in Wash­ing­ton state had been and would be able to be FDA-in­spect­ed. But not­ing that liso-cel al­ready was sub­mit­ted in De­cem­ber and giv­en pri­or­i­ty re­view in Feb­ru­ary, he gave a 90% chance of ide-cel ap­proval and wrote that “we would ar­gue there is a de­cent chance here the plant has al­ready been in­spect­ed and the con­cerns may be overblown.”

How long the ther­a­py re­mains a top op­tion for mul­ti­ple myelo­ma pa­tients is a dif­fer­ent ques­tion. At ASH, BMS al­so showed da­ta for CC-93269, a BC­MA-tar­get­ing bis­pe­cif­ic an­ti­body that put up “crazy num­bers,” as Baird an­a­lyst Bri­an Sko­r­ney put it: 89% ORR, 33% CR. Re­gen­eron al­so showed off its BC­MA bis­pe­cif­ic. Safer and dra­mat­i­cal­ly sim­pler to ad­min­is­ter, these an­ti­bod­ies may ul­ti­mate­ly win out, Sko­r­ney said.

”We think the true com­mer­cial op­por­tu­ni­ty is re­al­ized by the most safe­ly com­bin­able as­set with gener­ic Revlim­id/Vel­cade,” he wrote. “As such, al­though fur­ther be­hind than BC­MA-CART, we see these drugs leap­ing ahead in the treat­ment par­a­digm, bar­ring ma­jor safe­ty is­sues.”

In a sec­ond big set­back for Covid-19 an­ti­body treat­ment hopes, Re­gen­eron halts en­roll­ment for more se­vere pa­tients

Regeneron has just delivered more bad news for the hope that neutralizing antibodies could be used to treat patients with more severe forms of Covid-19.

The New York biotech said today that an independent monitoring committee recommended halting enrollment of patients who need high-flow oxygen or mechanical ventilation in one of the trials on their antibody cocktail, after finding “a potential safety signal” and “an unfavorable risk/benefit profile.” The news comes a week after the NIH scrapped a trial of Eli Lilly’s Covid-19 antibody after finding it was having little effect on an initial cohort of hospitalized patients.

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Bris­tol My­er­s' Richard Har­g­reaves pays $70M to launch a neu­rode­gen­er­a­tion al­liance with a star play­er in the ma­chine learn­ing world

Bristol Myers Squibb is turning to one of the star upstarts in the machine learning world to go back to the drawing board and come up with the disease models needed to find drugs that can work against two of the toughest targets in the neuro world.

Daphne Koller’s well-funded insitro is getting $70 million in cash and near-term milestones to use their machine learning platform to create induced pluripotent stem cell-derived disease models for ALS and frontotemporal dementia.

CEO Kenji Yasukawa (Astellas)

In ear­ly blow to Ken­ji Ya­sukawa's R&D re­vamp, Astel­las drops out of the TIG­IT race, cit­ing PhI fail­ure

Just after AstraZeneca jumped into the TIGIT race, Astellas quietly disclosed that it was leaving, dropping out of a hunt for an immunotherapy approach that has shown tantalizing promise but remains largely unproven.

Astellas revealed in their second quarter earnings today that they’ve ended development of the anti-TIGIT antibody they acquired in their up to $400 million buyout of Potenza in 2018. The Japanese pharma had been testing it in combination with Keytruda in a 300-person Phase I study on patients with advanced solid tumors. A smaller study testing the antibody alone was completed, 2 years ahead of schedule, in July.

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George Golumbeski (L) and Faheem Hasnain

George Golumbes­ki and Fa­heem Has­nain team up with Ver­tex Ven­tures HC in man­ag­ing $320M of biotech cash

Two longtime biotech veterans are joining a multibillion dollar VC firm in order to help steer its latest fund.

George Golumbeski and Faheem Hasnain have signed on to Vertex Ventures HC as executive advisors, the company announced Thursday, and will assist with their depth of experience in managing $320 million of capital. Both have had previous working relationships with managing partners Carolyn Ng and Lori Hu, which evolved “organically” to get to this point, Ng said.

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Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Eli Lilly CEO David Ricks (Evan Vucci/AP Images)

A p-val­ue of 0.38? NE­JM re­sults raise new ques­tions for Eli Lil­ly's vaunt­ed Covid an­ti­body

Generally, a p-value of 0.38 means your drug failed and by a fair margin. Depending on the company, the compound and the trial, it might mean the end of the program. It could trigger layoffs.

For Eli Lilly, though, it was part of the key endpoint on a trial that landed them a $1.2 billion deal with the US government to supply up to nearly 1 million Covid-19 antibodies.

So what does one make of that? Was the endpoint not so important, as Lilly maintains? Or did the US government promise a princely sum for a pedestrian drug?

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CMO Merdad Parsey (Gilead)

Gilead hits the brakes on a tri­fec­ta of mid- and late-stage stud­ies for their trou­bled fil­go­tinib pro­gram. It's up to the FDA now

Gilead $GILD execs haven’t decided exactly what to do with filgotinib in the wake of the slapdown at the FDA on their rheumatoid arthritis application, but they’re taking a time out for a slate of studies until they can gain some clarity from the agency. And without encouraging guidance, this drug could clearly be axed from the pipeline.

In their Q3 report out Wednesday afternoon, the company says researchers have “paused” a Phase III study for psoriatic arthritis along with a pair of Phase II trials for ankylosing spondylitis and uveitis. Late-stage studies for ulcerative colitis and Crohn’s are continuing, but you can see for yourself how big a hole this leaves in the inflammatory disease pipeline, with obvious implications if the company abandons filgo altogether.

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As­traZeneca sells off heart fail­ure and hy­per­ten­sion drugs to Chep­lapharm for $400M

Out with the old and in with the new: AstraZeneca is selling off two heart failure and hypertension drugs to Germany-based Cheplapharm, bagging $400 million and making way for development in other areas.

Cheplapharm paid $200 million for the European rights to Atacand (candesartan cilexetil) and Atacand Plus (candesartan cilexetil and hydrochlorothiazide) back in 2018. They’re now doubling that amount for commercial control in more than 70 countries.

News brief­ing: Ax­o­vant faces months of de­lay on lead Parkin­son's gene ther­a­py; Chi­nese CAR-T biotech nabs $100M

One of Axovant’s top gene therapy prospects for its second act is hitting a roadblock that could push its clinical timelines back by almost a year.

In an update, the biotech said it was informed about delays in CMC data and third-part fill-finish issues around mid-October by its manufacturing partner, Oxford Biomedica. Axovant has been developing a suspension-based process for the Parkinson’s drug; with that taking longer than expected, it now believes “it is unlikely that its planned randomized, sham-controlled trial of AXO-Lenti-PD will enroll patients by the end of calendar year 2021.”