Boehringer In­gel­heim buys CD38 drug; Mer­ck bets on ma­chine learn­ing in an­ti­body de­sign

CD38 may be best known as a tar­get for can­cer ther­a­pies — no­tably J&J’s Darza­lex and Sanofi’s Sar­clisa. But Boehringer In­gel­heim be­lieves it has po­ten­tial in im­muno­log­i­cal and fi­brot­ic dis­eases, too.

The Ger­man phar­ma gi­ant is ac­quir­ing a pre­clin­i­cal CD38 pro­gram from Ri­bon Ther­a­peu­tics, pay­ing an undis­closed up­front and promis­ing fu­ture mile­stones.

Un­like the ap­proved CD38 drugs, which are both an­ti­bod­ies, Ri­bon de­vel­oped a small mol­e­cule in­hibitor, which it be­lieves car­ries the ex­tra ad­van­tage of block­ing in­tra­cel­lu­lar CD38 ac­tiv­i­ty.

Ri­bon has at­tract­ed back­ing from a slate of VC as well as phar­ma in­vestors thanks to its plat­form elu­ci­dat­ing the fam­i­ly of NAD+-uti­liz­ing en­zymes. Its own lead pro­grams tar­get PARP7 and PARP14 to treat on­col­o­gy and in­flam­ma­to­ry dis­eases.

Ma­chine learn­ing in an­ti­body de­sign lands BigHat a deal with Mer­ck

Mer­ck is buy­ing in­to BigHat’s an­ti­body de­sign plat­form in a deal that can lead to three drug dis­cov­ery pro­grams.

San Ma­teo-based BigHat set out to pair wet lab ca­pa­bil­i­ties for syn­the­siz­ing, ex­press­ing, pu­ri­fy­ing and char­ac­ter­iz­ing mol­e­cules with ma­chine learn­ing tech­nolo­gies to en­gi­neer an­ti­bod­ies with “more com­plex func­tions and bet­ter bio­phys­i­cal prop­er­ties.” It’s al­so in a re­search col­lab­o­ra­tion with Am­gen to op­ti­mize and im­prove up­on one of Am­gen’s an­ti­bod­ies.

“This agree­ment with Mer­ck brings us a ma­jor step clos­er to our goal of 3-5 deep col­lab­o­ra­tions with lead­ing bio­phar­mas to com­ple­ment our in­ter­nal ther­a­peu­tic pipeline,” said Eliz­a­beth Schwarzbach, BigHat’s chief busi­ness of­fi­cer.

She added that Mer­ck and BigHat have al­ready start­ed work­ing on the first pro­gram.

Aldeyra files NDA for dry eye drug

It took per­haps longer than ex­pect­ed, but Aldeyra has fi­nal­ly filed its drug for dry eye dis­ease with the FDA.

Ad­min­is­tered as an eye drop, re­prox­alap would be the first mar­ket­ed re­ac­tive alde­hyde species) mod­u­la­tor if it’s ap­proved, ac­cord­ing to Aldeyra. The NDA sub­mis­sion car­ries da­ta from five clin­i­cal tri­als, the com­pa­ny added, which mea­sured ef­fi­ca­cy on a va­ri­ety of met­rics, in­clud­ing the oc­u­lar dry­ness symp­tom score, oc­u­lar red­ness, Schirmer test (a test of tear pro­duc­tion), and Schirmer test ≥10 mm re­spon­der analy­sis.

“The NDA sub­mis­sion for re­prox­alap is, to our knowl­edge, the most com­pre­hen­sive reg­u­la­to­ry pack­age ever for a dry eye dis­ease drug can­di­date,” pres­i­dent and CEO Todd Brady said in a state­ment. “With da­ta sug­gest­ing ac­tiv­i­ty with­in min­utes of ad­min­is­tra­tion, re­prox­alap could pro­vide an im­por­tant treat­ment op­tion for the mil­lions of dry eye pa­tients who gen­er­al­ly re­gard cur­rent­ly avail­able ther­a­pies as in­ad­e­quate.”

Aldeyra ran in­to a set­back back in late 2021 when re­prox­alap missed the pri­ma­ry end­point in a Phase III tri­al, but it charged ahead with an­oth­er late-stage tri­al with an amend­ed end­point — and the drug proved a suc­cess. Sub­se­quent da­ta, in­clud­ing from a crossover study, were al­so pos­i­tive.

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

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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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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.