Nan­jing Leg­end wows AS­CO re­searchers with ear­ly da­ta on a BC­MA-tar­get­ed CAR-T con­tender

Chi­na’s Nan­jing Leg­end Biotech is a vir­tu­al un­known in the CAR-T field. But it gained mar­quee sta­tus at AS­CO to­day with stel­lar ear­ly mul­ti­ple myelo­ma da­ta on a drug tar­get­ed at B-cell mat­u­ra­tion pro­tein, or BC­MA.

Among 35 re­lapsed, drug-re­sis­tant pa­tients, 33 — 94% — demon­strat­ed clin­i­cal re­mis­sion rang­ing from a com­plete to a par­tial re­sponse. There was a 100% ob­jec­tive re­sponse rate, ac­cord­ing to in­ves­ti­ga­tors.

The in­ves­ti­ga­tors al­so flagged some promis­ing signs of dura­bil­i­ty.

Of the first 19 pa­tients to reach the four-month mark spec­i­fied in the tri­al, a re­mark­able 14 achieved a “strin­gent” com­plete re­sponse (sCR), which is as­so­ci­at­ed with longer sur­vival times. And 5 pa­tients who hit the one-year mark were still in the sCR stage.

Xi­ao­hu (Frank) Fan

Look­ing past the CD19 bio­mark­er, BC­MA of­fers one of the most promis­ing tar­gets in CAR-T. And this is, to date, the largest pa­tient group yet re­port­ed on. There are, how­ev­er, some sig­nif­i­cant caveats to point out. Its BC­MA ri­val blue­bird bio points out that these pa­tients ev­i­dent­ly weren’t as sick as the ones they treat­ed, with few­er ear­li­er drug fail­ures.

But you can be sure that there will be plen­ty of an­a­lysts pour­ing over the da­ta to­day to start cal­cu­lat­ing how the lead­ers all line up.

The same safe­ty is­sues that have plagued in­ves­ti­ga­tors for years now were ev­i­dent in the study. 85% of the pa­tients in the study ex­pe­ri­enced cy­tokine re­lease syn­drome, with two ex­pe­ri­enc­ing a grade 3 case that re­quired Actem­ra to tamp down on the in­flam­ma­to­ry re­sponse. All those pa­tients re­cov­ered.

And no mat­ter who is do­ing the work, CAR-T ap­pears to be more promis­ing than ever for this group of pa­tients.

“While it’s still ear­ly, these da­ta are a strong sign that CAR T-cell ther­a­py can send mul­ti­ple myelo­ma in­to re­mis­sion,” said AS­CO Ex­pert Michael S. Sabel in a state­ment. “It’s rare to see such high re­sponse rates, es­pe­cial­ly for a hard-to-treat can­cer. This serves as proof that im­munother­a­py and pre­ci­sion med­i­cine re­search pays off. We hope that fu­ture re­search builds on this suc­cess in mul­ti­ple myelo­ma and oth­er can­cers.”


Pic­tured: Nan­jing, Chi­na on April 27th, 2017 / Shut­ter­stock

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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