No­var­tis silent­ly yanks a trou­bled biosim­i­lar ap­pli­ca­tion; Chi­nese an­ti­body de­vel­op­er draws $39M round

• No­var­tis nev­er has tried to ex­plain why the FDA has re­ject­ed its biosim­i­lar of Am­gen’s Neu­las­ta. And it’s be­ing equal­ly ret­i­cent about its de­ci­sion to yank its ap­pli­ca­tion in Eu­rope. The “ap­pli­ca­tion for a mar­ket­ing au­tho­riza­tion for Ziox­ten­zo (peg­fil­gras­tim) has been with­drawn,” the EMA not­ed to­day. “Ziox­ten­zo was de­vel­oped as a biosim­i­lar med­i­cine to treat neu­trope­nia in can­cer pa­tients.” No­var­tis loves to high­light its R&D progress, but set­backs are dealt with silent­ly. The EMA, though, did ex­plain that the biosim­i­lar ap­peared to be un­ap­prov­able.  “One con­cern was that study re­sults were not able to show that the con­cen­tra­tions of peg­fil­gras­tim in blood were the same af­ter tak­ing Ziox­ten­zo and Neu­las­ta. The oth­er con­cern was the lack of a cer­tifi­cate of Good Man­u­fac­tur­ing Prac­tice (GMP) for the med­i­cine’s man­u­fac­tur­ing site. An in­spec­tion of the site will there­fore be need­ed be­fore the med­i­cine can be ap­proved.”

• The GT­JA In­vest­ment Group has led a $39 mil­lion round for Mab­works Biotech, a Chi­nese com­pa­ny that’s been work­ing on a cock­tail for Ebo­la. Har­vest Cap­i­tal Man­age­ment, Me­fund Cap­i­tal and E-Town Bio­med­ical Park al­so par­tic­i­pat­ed in the round for the Bei­jing based an­ti­body de­vel­op­er.

• Pfiz­er says that its Phase II study of a new C diff vac­cine, passed muster in a Phase II tri­al, set­ting up a late-stage study set to launch in the first half of this year. “We are very en­cour­aged by these in­ter­im im­muno­genic­i­ty and safe­ty re­sults demon­strat­ing ro­bust in­creas­es in vac­cine-elicit­ed neu­tral­iz­ing an­ti­bod­ies to both tox­ins, that we be­lieve could pro­vide pro­tec­tion against C. dif­fi­cile dis­ease,” said Kathrin Jansen, Ph.D., se­nior vice pres­i­dent and head of Vac­cine Re­search and De­vel­op­ment for Pfiz­er.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; 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.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

89bio to net $275M from stock of­fer­ing; As­sem­bly Bio to pause work on one HBV in­hibitor pro­gram

San Francisco-based biotech 89bio announced on Friday that it expects to rake in $275 million on a stock offering. The raise comes after 89bio announced on Tuesday results of a Phase II study showing that its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH.

To run a Phase III study, 89bio CEO Rohan Palekar told Endpoints News that the biotech “would need to raise additional capital.” 89bio offered over 16 million shares of its common stock at $16.25 per share, and expects the offering closes on March 28.