James Li, JW Therapeutics CEO

Sis­ter to Juno's liso-cel, JW's rel­ma-cel lands sec­ond-ever CAR-T ap­proval in Chi­na

Juno may have stum­bled its way to a dis­tant third fin­ish in the CD19 CAR-T race, but its joint ven­ture in Chi­na is head­ing off to a thriv­ing start.

JW Ther­a­peu­tics, which took its name from co-founders Juno and WuXi AppTec, has scored the sec­ond-ever CAR-T ap­proval by Chi­na’s Na­tion­al Med­ical Prod­uct Ad­min­is­tra­tion just weeks af­ter Fo­s­un Kite, an­oth­er joint ven­ture, claimed first.

Now sanc­tioned to treat re­lapsed or re­frac­to­ry large B-cell lym­phoma pa­tients who’ve had two or more lines of sys­temic ther­a­py, rel­ma-cel was de­vel­oped on the same cell process plat­form that spawned Juno’s liso-cel — now Bris­tol My­ers Squibb’s Breyanzi — but a dif­fer­ent drug in that it’s tai­lored for Chi­nese needs.

Those tweaks al­so gave rise to a po­ten­tial­ly best-in-class pro­file, ac­cord­ing to JW — a claim that will be sure to stoke heat­ed de­bate among cell ther­a­py mak­ers.

It is the sixth ap­proved CAR-T treat­ment glob­al­ly, based on clin­i­cal da­ta from more than 100 pa­tients. The NM­PA had grant­ed pri­or­i­ty re­view to its LB­CL ap­pli­ca­tion and a break­through ther­a­py des­ig­na­tion for fol­lic­u­lar lym­phoma.

When Juno and WuXi first start­ed the com­pa­ny, the idea was for JW to lever­age Juno’s sci­ence and re­search ex­per­tise with the savvi­ness in process de­vel­op­ment, man­u­fac­tur­ing, qual­i­ty con­trol, reg­u­la­to­ry and clin­i­cal de­vel­op­ment, as well as com­mer­cial­iza­tion that WuXi, a CRO gi­ant, can of­fer.

“Our goal has been very clear,” CEO James Li pre­vi­ous­ly said in an in­ter­view with End­points News. “We want to build the best cell ther­a­py com­pa­ny in Chi­na.”

Li, though, wasn’t con­tent with re­ly­ing on the Amer­i­can part­ners for dis­cov­ery, even af­ter strik­ing a new part­ner­ship with Lyell. In the sum­mer of 2020, he swooped in to buy Syra­cuse — the Chi­nese arm of Bay Area-based Eu­re­ka Ther­a­peu­tics — be­fore flip­ping to a $300 mil­lion IPO on the HKEX.

He’s not too wor­ried about oth­ers catch­ing up, ei­ther.

“What peo­ple don’t re­al­ize is it takes a much longer time ac­tu­al­ly if you want to have a com­mer­cial­ly vi­able process, to have some­thing mean­ing­ful you can com­mer­cial­ize,” he said, adding: “I think it’s a great thing if peo­ple try to dif­fer­en­ti­ate in ear­ly stage. Then whether they can go to com­mer­cial or not, I think that’s a big ques­tion mark. I would put a big ques­tion mark there. And then in that way I do see more and more col­lab­o­ra­tions be­tween com­pa­nies. Even more M&A in the space. If some­body has a re­al­ly good tar­get and demon­strat­ed ear­ly on, I would be hap­py to work with them.”

Why it Works: Man­u­fac­tur­ing a Vac­cine in a Mul­ti-Prod­uct Fa­cil­i­ty.

COVID-19 launched the pharmaceutical industry to the frontline in the battle against the fast-spreading global pandemic. The goal: distribute a safe, effective vaccine as quickly as possible. Major players in the vaccine market needed to partner with contract development and manufacturing organizations (CDMOs) to achieve the goal of mass vaccine quantities under expedited timelines. With CDMOs stepping up to play a critical role in the vaccine manufacturing process, multi-product CDMO facilities took the spotlight. Partnerships quickly formed as the race to save lives and fight a pandemic was on.

Safe­ty fears force Pfiz­er to change piv­otal DMD gene ther­a­py tri­al pro­to­col

As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

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Merck CEO Rob Davis

Mer­ck emerges as lead bid­der in po­ten­tial Ac­celeron buy­out with deal pos­si­ble this week — re­port

With rumors swirling about a potential buyout of biotech Acceleron and its lead PAH drug sotatercept, market watchers have been keeping close tabs on industry movers and shakers due up for an expensive bolt-on. According to a new report, it appears Merck may be the one.

Merck is in “advanced talks” on a deal to acquire Cambridge, MA-based Acceleron in what previous reports pegged as a potential $11 billion buyout, the Wall Street Journal reported Monday. A deal could come as early as this week, according to the Journal.

Alexander Lefterov/Endpoints News

The coro­n­avirus vac­cine that the world for­got could still help save it

Back at the beginning of the pandemic — back when we still called the virus “novel” and a single case in Washington state could make headlines — there emerged the story of the coronavirus vaccine that the world forgot.

It was an allegory for our pandemic ill-preparedness. At a time when the world had been caught so flat-footed, there were a pair of scientists who had seen the crisis coming, lab-coated Cassandras with an antidote if only the world had listened sooner.

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Habib Dable, Acceleron CEO

Days of heat­ed ru­mors cul­mi­nate in a re­port that Ac­celeron is in ad­vanced buy­out talks

Days of frothy rumors about possible M&A discussions at Acceleron were capped late Friday with a Bloomberg report asserting that the biotech company is in advanced talks for an $11 billion buyout deal.

Bloomberg was unable to identify any bidders in the deal, but speculation has been running rampant that the surging value of Acceleron stock had to be the result of leaks around the auction of the company. As of early Monday morning, we’re still awaiting the final word.

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Peter Marks (Jim Lo Scalzo/Pool via AP Images)

Tur­moil at CBER: Pe­ter Marks grabs con­trol of FDA's Of­fice of Vac­cines ahead of 2 key ca­reer leader de­par­tures

FDA’s top vaccine official Peter Marks is pulling the plug on a months-long transition for two top career vaccine officials who abruptly called it quits in late August.

Marion Gruber, director of the FDA’s Office of Vaccines Research & Review and 32-year veteran of the agency, her deputy director Phil Krause, announced their departures and then raised concerns with Covid-19 booster shots ahead of and during a recent Covid-19 booster vaccine advisory committee.

Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Ciaran McCrickard)

Seiz­ing Aduhelm prece­dent, Bio­gen, Ei­sai get the ball rolling on an­oth­er ac­cel­er­at­ed Alzheimer's ap­proval

Biogen — together with partners at Eisai — will be the first to breeze down that Alzheimer’s trail it blazed with the FDA’s historic and controversial accelerated approval of Aduhelm.

Eisai disclosed it’s initiated a rolling BLA submission for lecanemab, or BAN2401, the anti-amyloid beta antibody that Biogen has long touted as a follow-on to Aduhelm (then aducanumab). Following the precedent that the agency has now set, the two companies are gunning for an OK based on data suggesting that the drug could lower amyloid beta plaques.

Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Ciaran McCrickard)

New ARIA cas­es dog Bio­gen's Aduhelm launch in an­oth­er po­ten­tial blow to block­buster hopes — an­a­lyst

Perhaps anticipating a walkover with an FDA approval in hand, Biogen has instead faced a wall of pushback over its controversial Alzheimer’s med Aduhelm. With payers and clinics largely staying away, Biogen’s drug is posting some troubling safety signals that could derail the launch even further.

In a note to clients Friday, analysts from Baird revealed new safety data from the FDA’s Adverse Event Reporting System (FAERS) showing a troubling recurrence of ARIA in a crop of patients who have been dosed with Aduhelm.

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Albert Bourla, Pfizer CEO (John Thys, Pool via AP Images)

Covid-19 roundup: Pfiz­er/BioN­Tech sub­mit vac­cine da­ta to FDA for younger chil­dren; Doc­tors kept pre­scrib­ing hy­drox­y­chloro­quine

Pfizer and BioNTech said Tuesday they submitted to FDA positive data from a Phase II/III trial of their Covid-19 vaccine in children aged 5 to less than 12 years old.

A formal EUA submission for the vaccine in these children is expected to follow “in the coming weeks,” the companies said in a statement.

The trial of 2,268 healthy participants aged 5 to less than 12 years old showed the vaccine was safe and elicited robust neutralizing antibody responses using a two-dose regimen of 10 μg doses, which is one-third the dose that’s administered to adults.