Kalshi will launch bets on biotech

Prediction market giant Kalshi announced this morning that it will now start taking bets on clinical trial outcomes and FDA approvals, STAT’s Elaine Chen writes. It’s starting with a small number of markets selected alongside partner AppliedXL, a tech company that already monitors and predicts clinical trial outcomes.

Kalshi believes prediction markets can give investors a more direct way to wager on drug development milestones while generating useful signals for biopharma decision-making. For now, it's sticking to late-stage trials and full approval decisions to reduce risks around insider trading and ambiguous outcomes. The company also says it has built in safeguards, including by barring trial participants and certain insiders from trading, though not everyone is convinced manipulation and subtle influences on trial conduct can be avoided.

“Any kind of adulteration that happens in [the clinical trial process] is ultimately threatening the well-being of people who are using that drug and the evidence about it,” said Jonathan Kimmelman, a bioethicist at McGill University.

cardiology

FDA approves Merck's oral PCSK9 drug, a first

From STAT's Matthew Herper: Merck said this morning that the FDA had approved Lipfendra, the first oral PCSK9 drug for lowering high cholesterol. The medicine is generically known as enlicitide.

PCSK9 inhibitors were once expected to be among the industry’s biggest sellers, but sales have remained modest. Amgen’s Repatha has annual sales of about $3 billion. Both it and Praluent, from Regeneron, have been shown to prevent heart attacks and strokes.

But both Repatha and Praluent were monoclonal antibodies that must be injected. Merck used a technology called macrocycles to make a PCSK9 inhibitor that could be swallowed. In one clinical trial, it lowered patients’ low-density lipoprotein (LDL), the “bad cholesterol,” by 59%.

Analysts polled by Visible Alpha expect Lipfendra sales of $315 million next year increasing to $1.3 billion in 2029 and $2.5 billion in 2031.

“This is a pivotal moment as we bring the first U.S. FDA-approved oral PCSK9 inhibitor to adults with high LDL-C, offering patients an important new option,” said Dean Li, president of Merck Research Laboratories, in a statement.

Merck said in December that enlicitide was granted a Commissioner National Priority Review Voucher, a controversial program that could enable a review in just a month or two. However, the FDA took about seven months reviewing the drug.

M&A

Eli Lilly buys a psychedelic medicine

Eli Lilly is acquiring AtaiBeckley, the developer of psychedelic treatments for mental health conditions, for up to $3.8 billion, to expand its portfolio of neuroscience medicines, the company said this morning.

The AtaiBeckley deal is just the latest in a string of acquisitions by the pharma giant, flush with cash from its booming GLP-1 business.

The centerpiece of the acquisition is AtaiBeckley’s experimental drug called BPL-003. It’s a synthetic form of the psychedelic 5-MeO-DMT administered intranasally for treatment-resistant depression.

multiple sclerosis

Study shows Epstein-Barr's role in multiple sclerosis

A new study helps explain how Epstein-Barr virus may trigger multiple sclerosis, STAT’s Lauren Chan writes.

In the study, scientists identified a specific immune pathway in which EBV-infected memory B cells activate CD4+ T cells that ultimately attack myelin, the protective coating around nerves. This builds on earlier evidence that links EBV infection to multiple sclerosis and suggests that targeting the virus directly — through future vaccines or antiviral drugs — could offer a more precise alternative to today's broadly immunosuppressive therapies.

Researchers also found that B cell-depleting drugs dampen the EBV-specific immune response, which offers clues for potential new drug targets.

“It’s very nice now to be able to understand more about the underlying mechanisms of how EBV likely causes MS,” lead author Kjetil Bjornevik, an epidemiologist at Harvard, told STAT.

conservation

Deadly venoms may be tomorrow's blockbusters

Some of today's most important medicines trace their origins to animal venoms, as researchers increasingly mine evolution's molecular toolkit for drugs that target human biology with remarkable precision. In an opinion piece for STAT, Steve Midway, an ecology professor at Louisiana State University, says one reason we must be mindful of conservation is to preserve a lucrative pipeline of future medicines.

ACE inhibitors grew out of Brazilian pit viper venom, the GLP-1 revolution began with a peptide from the Gila monster, and a cone snail toxin became a powerful non-opioid pain drug. Meanwhile, newer drug candidates include a scorpion-derived compound that makes brain tumors glow during surgery.

“Evolution has done most of the hard work for us. The peptides in venom were not assembled at random. They have been refined over millions of years to interact with precise biological targets,” he writes. “A chemist starting from scratch might spend a career just to create the venom, let alone the subsequent drug. Yet some of the most powerful medicines we will ever find are already here, carried in the fangs, stingers, and saliva of creatures we’ve long been taught to fear.”

Whether they’ll survive the Anthropocene is another question entirely.

rare disease

Amylyx approaches pivotal test for endocrine therapy

Amylyx Pharmaceuticals expects pivotal Phase 3 data later this quarter for avexitide, STAT’s Adam Feuerstein writes in his latest Biotech Scorecard newsletter. It’s a GLP-1 receptor antagonist designed to treat post-bariatric hypoglycemia, a debilitating condition in which patients experience dangerous blood sugar crashes after weight-loss surgery. A positive readout could put the company on track for an FDA filing and potential 2027 launch.

The study also marks a critical chapter in Amylyx's comeback after the it voluntarily withdrew its controversial ALS drug Relyvrio in 2024 when a confirmatory trial failed, a decision its co-founders say reflected a commitment to patients over commercial interests.

“Our principles have been pressure tested, maybe more than we wanted them to be, but I think the company that has emerged today is even stronger. We could not be more proud of our team’s resilience and focus,” Amylyx co-CEO Justin Klee said.

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