April 7, 2025

The need-to-know this morning

Rhythm Pharmaceuticals said its drug setmelanotide, which is approved as Imcivree for genetic forms of obesity, succeeded in a Phase 3 trial of a type of obesity caused by damage to the hypothalamus. Rhythm plans to submit the data to regulatory agencies.
Caris Life Sciences, a closely held genomic sequencing and liquid biopsy firm, said it had raised $168 million from investors including Braidwell LP, Perceptive Advisors, Woodline, and Ghisallo. The firm has raised $1.86 billion since 2016.
GSK pledged $49 million up front and up to $2.6 billion in milestones to South Korea's ABL Biopharma for a technology to ferry drugs across the blood-brain barrier.

venture capital

Brash biotech VC speaks out on industry headwinds

Omega Funds founder Otello Stampacchia isn't shy about pointing out what he see as flaws in the biotech sector: The industry is flailing and dealing with inflated pipelines, he says, as well as weak governance and dwindling clinical success rates. Meanwhile, his venture firm has quietly delivered, STAT's Allison DeAngelis writes.

Since early 2022, Omega has seen eight IPOs and 14 acquisitions from its portfolio. But he warns that, as early research funding shrinks, fewer biotechs will be formed — and blames the 2019 bubble for inflated expectations of success these days.

"The later-stage stuff, I think, is getting screwed, and the super early-stage research is getting screwed," he said. "The current number of companies in biotech.… It's over-bloated because there's too many public companies who are potentially not going to be able to deliver clinical molecules."

vaccines

Funding for HIV vaccine trials suspended

The Trump administration's cuts to staff at the Centers for Disease Control and Prevention and to clinical trials are threatening to unwind progress that has been made in HIV prevention, affected researchers say.

Among other cuts, funds have been suspended for the HIV Prevention Trials Network and the HIV Vaccine Trials Network, the two most prominent networks for testing prevention drugs and vaccines, STAT's Jason Mast reports.

Funds have also been suspended for AIDS Clinical Trial Group, a nearly 40-year-old system that has been responsible for key breakthroughs dating back to the first effective AIDS drug, AZT. All three have been waiting on money that were supposed to be sent out in February.

REGULATION

FDA cracks down on CRO data fraud

In a rare but telling move, the FDA has rejected therapeutic equivalence studies from an undisclosed number of drugmakers after uncovering data falsification by India-based contract research organization Raptim Research. During a 2023 inspection of its Mumbai site, regulators found serious data integrity issues, especially in vitro studies used to support generics.

The agency deemed the data unreliable, forcing drug companies to either repeat studies or pull products entirely, STAT's Ed Silverman writes — though it doesn't expect shortages for now. The latest development adds to a troubling pattern of fraud at CROs in India, highlighting long-standing concerns about the integrity of the global pharma supply chain.

REGULATION

Reviving FDA voucher program could save more kids

A quietly powerful FDA initiative that saved thousands of children's lives by incentivizing drug development for rare diseases expired in December. And Congress needs to fix that, opines BridgeBio CEO Neil Kumar. The Priority Review Voucher program, which doesn't cost taxpayers anything and is funded fully by biopharma, gave companies a valuable tradable coupon in exchange for developing treatments for ultra-rare, life-threatening diseases.

These vouchers have spurred the approval of dozens of drugs that have collectively helped more than 200,000 children, he says.

"And so, we are asking our members of Congress: Please help us to help those children," Kumar writes. "Please give those kids a chance."

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