Ying Huang, Legend CEO

FDA push­es back de­ci­sion on J&J, Leg­end's BC­MA CAR-T

Bris­tol My­ers Squibb and blue­bird can hold on to their sta­tus as the mak­ers of the on­ly ap­proved BC­MA CAR-T for a bit longer.

The FDA — which was sched­uled to make a de­ci­sion on Leg­end and J&J’s cil­ta-cel on Nov. 29 — has de­layed that by three months and ex­tend­ed the PDU­FA date to Feb. 28.

Ac­cord­ing to the part­ners, the agency no­ti­fied J&J’s Janssen of the ex­ten­sion on Oct. 28, cit­ing the need for time to re­view re­cent­ly sub­mit­ted in­for­ma­tion “per­tain­ing to an up­dat­ed an­a­lyt­i­cal method fol­low­ing an FDA in­for­ma­tion re­quest.” Leg­end and Janssen then met with reg­u­la­tors on Mon­day. No ad­di­tion­al clin­i­cal da­ta are be­ing re­quest­ed, they added.

The three-month ex­ten­sion rep­re­sents a “stan­dard time for FDA re­view” and shouldn’t im­pact the BLA “ma­te­ri­al­ly,” Jef­feries an­a­lyst Kel­ly Shi con­clud­ed af­ter catch­ing up with Leg­end ex­ecs.

Mgmt stat­ed that the FDA in­for­ma­tion re­quest per­tains to the CMC sec­tion and the method val­i­da­tion for fi­nal prod­ucts. Im­por­tant­ly, the re­quest is not re­lat­ed to the man­u­fac­tur­ing process or the pre-ap­proval in­spec­tion. The three-month ex­ten­sion is the stan­dard time frame for FDA re­view af­ter re­ceiv­ing a re­sponse to an in­for­ma­tion re­quest. Mgmt com­ment­ed that the co and the FDA are com­mit­ted to de­liv­er the ther­a­py ear­li­est to pa­tients.

For­mer­ly known as LCAR-B38M, cil­ta-cel got its big break from ob­scu­ri­ty at the AS­CO meet­ing in 2017, as Leg­end CSO Frank Fan and his team stole the show with an im­pres­sive 100% over­all re­sponse rate among mul­ti­ple myelo­ma pa­tients. While many re­mained skep­ti­cal, Janssen jumped in with both feet by the end of that same year, of­fer­ing Leg­end $350 mil­lion cash in a deal that made it a glob­al play­er overnight and blazed a trail for oth­er Chi­nese biotech star­tups to fol­low.

Thanks large­ly to cil­ta-cel, whose ef­fi­ca­cy was con­firmed in sub­se­quent clin­i­cal tri­als con­duct­ed in the US, Leg­end has since spun out from its par­ent com­pa­ny, the Chi­nese CRO Gen­Script, gained a foothold on Nas­daq fol­low­ing a $424 mil­lion IPO and built out a glob­al R&D net­work with op­er­a­tions in Chi­na, Ire­land and the US.

It was sup­posed to join Abec­ma, co-pro­mot­ed by Bris­tol My­ers and blue­bird, on the US mar­ket. No­tably, a faulty CMC sec­tion ini­tial­ly led the FDA to is­sue a “refuse to file” let­ter in re­sponse to the part­ners’ first Abec­ma BLA and ask for ad­di­tion­al de­tails re­gard­ing val­i­da­tion and con­trol process­es used in lentivi­ral vec­tor and drug prod­uct man­u­fac­tur­ing. It was about three months be­fore the com­pa­nies re­filed.

“In com­par­i­son, we view the in­for­ma­tion re­quest to LEGN as a mi­nor set­back and be­lieve the three months ex­ten­sion is suf­fi­cient for FDA to com­plete the re­view process,” Jef­feries an­a­lysts wrote.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Lan Huang, BeyondSpring CEO

Months af­ter shock­ing in­vestors with lung can­cer win, Be­yond­Spring's lead drug hits road­block at the FDA

BeyondSpring shocked investors in early August after its once-marginal lead drug suddenly showed a lot of promise in a common form of lung cancer. With hopes high, the FDA has now slammed the door on that drug in another indication — does that spell bad news for BeyondSpring’s Cinderella story?

The FDA issued BeyondSpring a complete response letter for its plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia, effectively shutting down the drug’s immediate chances at a marketing approval, the biotech said Wednesday.

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Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi snaps up new vac­cine can­di­date and de­vis­es mR­NA game plan around it — but not for what you think

Paul Hudson has spotlighted vaccines, immunology and dermatology as some of the top R&D focuses at Sanofi. His latest deal brings all of them together.

The French pharma giant isn’t sharing any financial details about the buyout of Origimm, a low-profile, private Austrian biotech whose technology promises to identify antigens causing skin disease and build vaccines against them. Their lead candidate targets acne vulgaris.

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J&J and Sanofi's mul­ti­ple myelo­ma tit-for-tat con­tin­ues, as sub­cu­ta­neous Darza­lex wins com­bo ap­proval

J&J and Sanofi have gone back and forth in their multiple myeloma tug-of-war. Earlier this year, Sanofi notched an approval of Sarclisa in combination with Amgen’s Kyprolis to try to outflank the big conglomerate, but J&J is clapping back.

Wednesday afternoon, Amgen announced that the subcutaneous version of J&J’s blockbuster Darzalex is also now approved as a combo with Kyprolis and dexamethasone. The green light came through for adults with relapsed or refractory multiple myeloma who had progressed on one to three earlier lines of therapy.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.