#ASH17: The BC­MA-tar­get­ed CAR-T bb2121 from blue­bird and Cel­gene takes a star turn in the spot­light

A few days ago, Cel­gene $CELG launched the piv­otal Phase II/III study that will dic­tate the fu­ture of the BC­MA-tar­get­ing bb2121, the CAR-T in-li­censed from blue­bird bio $BLUE. And if they come close to the da­ta that blue­bird has on of­fer at ASH this week­end, this drug will go on to blaze a big trail in the mar­ket as the de­vel­op­ers move on to the next big fo­cus in the field of en­gi­neered T cells.

In­ves­ti­ga­tors in the Phase Ib study out­lined a 94% over­all re­sponse rate for bb2121, with a jaw-drop­ping 56% com­plete re­sponse rate among a small group of 18 pa­tients in heav­i­ly pre­treat­ed ac­tive dose co­horts. Three of those 10 CRs, though, were un­con­firmed at the time of the pre­sen­ta­tion in At­lanta.

The me­di­an pro­gres­sion-free re­sponse rate had yet to be reached. But the 6- and 9-month PFS marks were passed with 81% and 71% rates.

Nick Leschly

“This is what we all dream about,” says blue­bird CEO Nick Leschly, “this kind of da­ta for pa­tients that are in a re­al­ly, re­al­ly tough po­si­tion.”

Blue­bird’s stock jumped 24% in pre-mar­ket trad­ing Mon­day, with Cel­gene {$CELG) up close to 5%.

To be sure there was plen­ty of tox­i­c­i­ty on the dose rang­ing study, with an ad­vanced pa­tient with a pun­ish­ing tu­mor bur­den ex­pe­ri­enc­ing grade 4 neu­ro­tox­i­c­i­ty with cere­bral ede­ma and sub­arach­noid he­m­or­rhage af­ter get­ting the high dose: 450 x 106 CAR+ T cells. And there were a num­ber of cas­es of cy­tokine re­lease syn­drome, a hall­mark of these first gen­er­a­tion CAR-Ts go­ing through the clin­ic.

Cel­gene will now take 150 to 300 mil­lion cell dos­es in­to the land­mark study with about 80 pa­tients, look­ing to see large­ly if it can repli­cate any­thing sim­i­lar. If they are suc­cess­ful, the part­ners can look for­ward to a block­buster mar­ket for one of the most wide­ly an­tic­i­pat­ed late-stage stud­ies in on­col­o­gy.

David David­son

The da­ta un­der­score the po­ten­tial of BC­MA as a tar­get in the field, which is see­ing the first pi­o­neers hit the mar­ket by hit­ting CD19 with ma­jor ef­fect. The da­ta will cer­tain­ly bright­en the stage for blue­bird and Cel­gene, the clear lead­ers in the field, as a bevy of ri­vals fol­low up in hopes of catch­ing or leapfrog­ging the fron­trun­ners.

Juno, mak­ing a CAR-T come­back with JCAR017, just bagged a gam­ma sec­re­tase in­hibitor from Eli Lil­ly for its BM­CA ther­a­py, adding IP agree­ments with the Fred Hutch and On­co­Track­er on their com­bo ap­proach.

But blue­bird isn’t sit­ting still. It’s al­so snap­ping up new tech to keep its cut­ting edge, like the CAR-en­gi­neered gam­ma delta T cell pact it struck with Scot­land’s TC Bio­Pharm a few days ago.

“We’ve got all cylin­ders fir­ing,” says David David­son, the blue­bird chief med­ical of­fi­cer.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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Marshall Fordyce, Vera CEO

Gene ther­a­py play­er turned kid­ney spe­cial­ist Ve­ra drops a dud in lead­up to Nas­daq, pric­ing well be­low range

Vera Therapeutics took a big risk at the start of the year, pivoting away from its gene editing mission statement to chase a lead kidney drug instead — and they doubled down with an IPO just months later. But investors don’t seem impressed with Vera’s promise, and now the biotech is looking at a far more scaled-back offering.

On Friday, Vera priced its 4.35-million-share IPO at $11 per share, well below its targeted range of $14 to $16 and good for $47.58 million in proceeds. The biotech will start trading Monday under the ticker $VERA.

Darren Ji, Elpiscience CEO (Lilly Asia Ventures)

Kept an ocean away from its sci­en­tif­ic ad­vi­sors, Shang­hai's Elpi­science keeps up the clin­i­cal progress, re­fu­els for its I/O pipeline

When Elpiscience pooled $100 million for its Series B in late 2019, CEO Darren Ji promised to move what he described as one of the broadest immuno-oncology pipelines swiftly through the clinic in both the US and China.

Then a pandemic got in the way — but not by much. The Shanghai-based biotech managed to keep testing its 4-1BB/PD-L1 drug, get an OX40 agonist cleared for clinical trials (nabbing a collaboration with Junshi in the process), while in-licensing a Phase I bispecific from California’s TRIGR Therapeutics.

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