Pfiz­er in­vests in Ri­bon Ther­a­peu­tics; Ar­genx gets PDU­FA date ex­ten­sion

Mass­a­chu­setts-based biotech Ri­bon Ther­a­peu­tics has roped in a big name for some fi­nan­cial back­ing.

Ri­bon an­nounced on Mon­day that Pfiz­er has in­vest­ed $25 mil­lion in­to the biotech. Robert Rick­ert, Pfiz­er’s head of can­cer im­munol­o­gy dis­cov­ery, will be com­ing on­to Ri­bon’s sci­en­tif­ic ad­vi­so­ry board. The start­up will use the funds to sup­port the de­vel­op­ment of its pro­grams for on­col­o­gy and im­munol­o­gy.

Both com­pa­nies al­so reached an agree­ment to have Pfiz­er pro­vide ex­per­tise to sup­port mov­ing for­ward Ri­bon’s pipeline, but the biotech will keep the eco­nom­ic rights and con­trol of the de­vel­op­ment of all pro­grams. Ri­bon’s can­di­dates in­clude a PARP7 in­hibitor that tar­gets sol­id tu­mors and a PARP14 in­hibitor go­ing af­ter in­flam­ma­to­ry dis­eases.

“Pfiz­er’s cap­i­tal sup­port and ex­per­tise will be in­stru­men­tal as we work to ad­vance our lead pro­grams—RBN-2397 in can­cer and RBN-3143 in im­munol­o­gy—to key clin­i­cal mile­stones and en­able fur­ther drug dis­cov­ery and de­vel­op­ment from our pro­pri­etary BEA­CON+ plat­form,” said Ri­bon CEO Prakash Ra­man, in a re­lease.

Ar­genx’s PDU­FA date ex­tend­ed

The FDA has de­cid­ed to ex­tend the re­view for the Bel­gian-based com­pa­ny ar­genx.

Last week, ar­genx an­nounced US reg­u­la­tors had ex­tend­ed the re­view of the BLA for sub­cu­ta­neous ef­gar­tigi­mod for treat­ing adults with the au­toim­mune dis­ease gen­er­al­ized myas­the­nia gravis (gMG). The date has now been pushed back to the sum­mer, specif­i­cal­ly June 20.

The biotech added that the in­for­ma­tion sub­mit­ted for the drug re­view had a “ma­jor amend­ment” and need­ed an ex­ten­sion so it could be looked at. How­ev­er, no ad­di­tion­al da­ta or stud­ies will need to be done for now.

“We are con­fi­dent in the pro­file of SC ef­gar­tigi­mod and the strength of the ADAPT-SC dataset show­ing non­in­fe­ri­or­i­ty of our sub­cu­ta­neous prod­uct to VYV­GART. “We will con­tin­ue to work close­ly with the FDA as it com­pletes its re­view to bring this im­por­tant med­i­cine to peo­ple liv­ing with gMG,” said ar­genx CMO Luc Truyen, in a re­lease.

In No­vem­ber of last year, the com­pa­ny net­ted a pri­or­i­ty re­view vouch­er for $102 mil­lion from blue­bird bio, which planned to short­en the re­view pe­ri­od from 10 months to around six months.

Struc­ture Ther­a­peu­tics sets the terms for its IPO

An­oth­er biotech looks to be toss­ing its hat in­to the IPO ring.

Struc­ture Ther­a­peu­tics, a Cal­i­for­nia-based com­pa­ny, fo­cus­es on GPCRs (al­so the pro­posed tick­er sym­bol) and has can­di­dates tar­get­ing di­a­betes and id­io­path­ic pul­monary fi­bro­sis.

Ac­cord­ing to SEC doc­u­ments, Struc­ture is look­ing to en­ter Nas­daq, of­fer­ing up 8,950,000 shares, and ex­pect­ing the IPO to be priced be­tween $13 to $15.

Struc­ture plans to net around $111.5 mil­lion from the IPO, with around $90 mil­lion be­ing put to­ward the de­vel­op­ment of its GLP-1R pro­gram meant to treat di­a­betes and obe­si­ty.

The funds will be used to fin­ish a Phase Ib and Phase IIa study. Around $13 mil­lion will go to an­oth­er pro­gram, with the re­main­ing cash be­ing used for R&D and oth­er gen­er­al us­es. The IPO is meant to fund the com­pa­ny through 2025.

The share­hold­ers in­clude sev­er­al ven­ture cap­i­tal firms along with CEO Ray­mond Stevens and CFO Jun Yoon hold­ing over 3%, among oth­ers.

Ed­i­tor’s Note: A cor­rec­tion has been made to the spelling of Prakash Ra­man’s name.

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.

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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.

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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.

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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.

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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.

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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.

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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.