Play­ing catch-up, Re­gen­eron and Sanofi push ahead on piv­otal PD-1 can­cer stud­ies

Re­gen­eron $REGN is wast­ing no time in push­ing ahead with what it hopes is their first piv­otal study for the lat­est in what is be­com­ing a long line of PD-1 check­point drugs. In its earn­ings re­lease this morn­ing the big biotech says that it is re­cruit­ing pa­tients for a po­ten­tial reg­is­tra­tion Phase II study on ad­vanced cu­ta­neous squa­mous cell car­ci­no­ma.

Mark Schoenebaum

ISI an­a­lyst Mark Schoenebaum added in a note this morn­ing that he’s keep­ing a close eye on their Phase II for non-small cell lung can­cer, which al­so launch­es this year.

Sanofi $SNY com­mit­ted back in 2015 to pay half of the $650 mil­lion de­vel­op­ment tab on REGN2810, part of a $1.8 bil­lion pack­age for a slate of im­muno-on­col­o­gy drugs — in­clud­ing mon­o­clon­al an­ti­bod­ies as well as bis­pecifics — that the two are now part­nered on. Sanofi’s own ef­forts in can­cer R&D stum­bled bad­ly, forc­ing a re­or­ga­ni­za­tion of its R&D ef­forts in one of many signs that the phar­ma gi­ant can’t seem to find its own path on drug de­vel­op­ment.

An­a­lysts see lit­tle to dis­tin­guish this PD-1 from the rest of the check­point field. But the next step in I/O in­volves a slew of com­bi­na­tion ther­a­pies that will look to change the land­scape on front­line ther­a­pies. Mer­ck and Bris­tol-My­ers Squibb got out in the mar­ket first, fol­lowed by Roche’s PD-L1 drug ate­zolizum­ab. Now As­traZeneca is fer­vent­ly pray­ing for a piv­otal suc­cess on dur­val­um­ab/treme­li­mum­ab — a cru­cial pro­gram for this phar­ma gi­ant — while Pfiz­er and Mer­ck KGaA close in on their own pro­gram.

Re­gen­eron scored a key court vic­to­ry yes­ter­day when the US Court of Ap­peals ruled in its fa­vor on Pralu­ent, stay­ing a de­ci­sion in Jan­u­ary that the PC­SK9 cho­les­terol drug should be jerked from the mar­ket due to the patent vi­o­la­tions seen with ri­val Repatha. Re­gen­eron and Sanofi al­so say that they’re ready to re­file sar­ilum­ab af­ter Sanofi’s Le Trait fa­cil­i­ty gets a clean bill of health. And they’re look­ing for a po­ten­tial megablock­buster ad­vance with a de­ci­sion look­ing on dupilum­ab. Oth­er late-stage mile­stones in­clude a Phase III for REGN2222 (an RSV-F An­ti­body) and a late-stage test for fas­inum­ab, an NGF an­ti­body for pain.

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.

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.

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

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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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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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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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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

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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