Bris­tol-My­ers checks off an­oth­er mile­stone on the Cel­gene CVR, hus­tling Yescar­ta ri­val to the mar­ket

As Bris­tol-My­ers Squibb wrapped its $74 bil­lion ac­qui­si­tion of Cel­gene last No­vem­ber, in­vestors be­gan a 16-month count­down to March 2021 — the dead­line for the phar­ma gi­ant to gain ap­proval on three of its new pipeline fa­vorites in or­der for the $9 con­tin­gent val­ue rights to ma­te­ri­al­ize.

An­oth­er one of them is now firm­ly on track to hit the goal ear­ly.

The FDA has ac­cept­ed Bris­tol-My­ers’ BLA for liso-cel, the CD19-di­rect­ed CAR-T orig­i­nal­ly de­vel­oped by Juno, and grant­ed a pri­or­i­ty re­view that brings the PDU­FA date up to Au­gust 17, 2020.

You may re­mem­ber the drug as JCAR017, the fol­low-up drug at the cen­ter of Juno’s come­back fol­low­ing some lethal neu­ro­tox­i­c­i­ty is­sues with its first-gen JCAR015. Along with Juno’s BC­MA CAR-T pro­gram and the in­fringe­ment law­suit against Kite Phar­ma, it’s al­so part of what in­trigued Cel­gene enough to gob­ble up the whole op­er­a­tion with $9 bil­lion two years ago.

Bris­tol-My­ers has since tak­en over and pre­sent­ed an up­beat as­sess­ment of a 268-pa­tient piv­otal tri­al for re­lapsed/re­frac­to­ry large B-cell lym­phoma. In­ves­ti­ga­tors tracked a 73% re­sponse rate and 53% com­plete re­sponse rate among pa­tients who have un­der­gone at least 2 pri­or lines of treat­ment.

“Re­call, there was some de­bate in the mar­ket­place whether this drug would get Pri­or­i­ty Re­view giv­en it would be the third CAR-T on the mar­ket if ap­proved,” Mizuho an­a­lyst Sal­im Syed not­ed.

The news marks the “best case out­come,” he added, giv­en that the mile­stone dead­line for liso-cel is Dec 31, 2020.

“There re­mains a crit­i­cal need for ad­di­tion­al ther­a­pies in large B-cell lym­phoma, par­tic­u­lar­ly for re­lapsed or re­frac­to­ry pa­tients,” Stan­ley Frankel, Bris­tol My­ers’ SVP of cel­lu­lar ther­a­py de­vel­op­ment, said in a state­ment.

Gilead’s pi­o­neer­ing CAR-T Yescar­ta was ap­proved in the ex­act same in­di­ca­tion in Oc­to­ber 2017 based on sim­i­lar da­ta from a small­er tri­al. Bris­tol-My­ers’ hope is that a low­er rate of cy­tokine re­lease syn­drome — just 2% in TRAN­SCEND NHL 001 — can con­vince physi­cians and pay­ers to choose their ther­a­py, even as new ev­i­dence sug­gests CRS risks can be mit­i­gat­ed by ear­ly use of steroids.

“There is a po­ten­tial these pa­tients can be treat­ed on an out­pa­tient ba­sis,” Samit Hi­rawat, the chief med­ical of­fi­cer, pre­vi­ous­ly told End­points News.

The com­mer­cial prospect re­mains a whole oth­er ques­tion, as in­vestors ques­tion whether Yescar­ta sales have plateaued at rough­ly $120 mil­lion per quar­ter.

The oth­er two cat­a­lysts that for­mer Cel­gene share­hold­ers need to re­deem their CVR — now list­ed on the NYSE as $BMYRT — will be the ap­provals of mul­ti­ple scle­ro­sis drug ozan­i­mod and bb2121, the blue­bird-part­nered BC­MA CAR-T. Once spurned, ozan­i­mod now has a PDU­FA date of March 25. Mean­while, the bb2121 sub­mis­sion may not hap­pen un­til lat­er this year; it has the lat­est dead­line out of the triple crown, set at the end of Q1 2021.

So­cial im­age cred­it: AP

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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Mickey Kertesz, KidsandArtOrg via YouTube

Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

The Japanese bank has invested $165 million into Karius, a company that uses blood tests to diagnose infectious diseases, as part of its new Vision Fund 2. The full scope of the new fund has yet to be announced, but the first and newly-beleaguered Vision Fund poured $100 billion into technology companies, including the biotechs Vir Biotechnology and Roivant and the sequencing company 10x Genomics.

Methicillin-resistant Staph aureus (Shutterstock)

FDA grants ‘break­through’ sta­tus to an­tibi­ot­ic al­ter­na­tive as Con­tra­Fect rush­es to join fight against su­per­bug

An experimental drug that promises to be the first anti-infective agent to prove superior to vancomycin — an antibiotic approved in 1958 — has notched the FDA’s “breakthrough” status.

ContraFect said the designation was based on Phase II data in which exebacase was tested against a superbug known as methicillin-resistant Staph aureus, or MRSA. In a subgroup analysis, the clinical responder rate at day 14 was 42.8% higher than that among those treated with standard of care, the company said (p=0.010).

Zhong Nanshan, CGTN via YouTube

Har­vard joins coro­n­avirus fight with $115 mil­lion and a high-pro­file Chi­nese part­ner

For two months, as the novel coronavirus swelled from a few early cases tied to a Wuhan market to a global epidemic, most of the world’s focus and dollars have flowed toward emergency initiatives: building vaccines at a record pace, plucking experimental antivirals out of freezers to see what sticks and immunizing mice for new antibodies.

Now a new and well-funded collaboration between Harvard and a top Chinese research institute will play the long game. In a 5-year, $115 million initiative backed by China Evergrande Group, researchers from the Harvard Medical School, Harvard T.H. Chan School of Public Health and Guangzhou Institute for Respiratory Health will study the virus in an effort to develop therapies against infections by the novel coronavirus, known as SARS–CoV-2, and to prevent new ones.

No­var­tis gets a boost in block­buster mul­ti­ple scle­ro­sis race with Roche

In the first step of what’s likely to be a long and uphill battle for the drugmaker, the FDA has accepted Novartis’s BLA submission for a new multiple sclerosis drug and given it priority review. The PDUFA date for the potential blockbuster drug is in June.

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Juergen Horn

An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

Zoetis forked over $85 million in 2017 to acquire Nexvet Biopharma and its pipeline of monoclonal antibodies. Juergen Horn, Nexvet’s former chief product development officer, has now secured $15 million for his own biologic company for animals: Invetx.

Buoyed by emerging advances in gene therapies for humans, scientists have started looking at harnessing the technology for animals setting up companies such as Penn-partnered Scout Bio and George Church-founded Rejuvenate Bio. But akin to Nexvet, Invetx is working on leveraging the time-tested science of monoclonal antibodies to treat chronic diseases that afflict man’s best friend.

As coro­n­avirus out­break reach­es 'tip­ping point,' GSK lends ad­ju­vant tech to Chi­nese part­ner armed with pre­clin­i­cal vac­cine

As the coronavirus originating out of Wuhan spreads to South Korea, Italy and Iran, stoking already intense fears of a pandemic, GlaxoSmithKline has found another pair of trusted hands to place its adjuvant system. China’s Clover Biopharmaceuticals will add the adjuvant to its preclinical, protein-based vaccine candidate against SARS-CoV-2.

Clover, which is based in the inland city of Chengdu, boasts of a platform dubbed Trimer-Tag that produces covalently-trimerized fusion proteins. Its candidate, COVID-19 S-Trimer, resembles the viral spike (S)-protein found in the virus.