The need-to-know this morning

Novo Nordisk said its experimental oral drug, called etavopivat, reduced the risk of pain crises and improved hemoglobin response in patients with sickle cell disease, achieving the goals of a Phase 3 clinical trial.
Eli Lilly is reportedly nearing a deal to acquire Kelonia Therapeutics for more than $2 billion, according to the Wall Street Journal. Kelonia is a privately held developer of so-called in-vivo CAR-T therapies that make engineered cancer-killing cells inside the body. Its lead therapy is being investigated in an early stage study in multiple myeloma.
Helus Pharma, developer of psychedelics-based therapies for depression, said CEO Michael Cola has resigned "at the request of the board of directors." Chairman Eric So was appointed interim CEO.

CANCER

Finally cracking KRAS as a druggable target

When Revolution Medicines released results for its drug daraxonrasib in pancreatic cancer patients last week, it finally seemed like there was hope in a condition that has long proven intractable for researchers. But as STAT's Angus Chen reports, it was the product of years of effort — and could be just the start of a wave of new RAS inhibitors.

While Rev Med is now developing five different RAS inhibitors, there are over 60 drug candidates being tested in hundreds of clinical trials across multiple cancer types, including lung, colorectal, endometrial and more. Some, like daraxonrasib, are multi-RAS inhibitors, and others are specific to different RAS mutations like KRAS G12D or G12V.

“I see RAS inhibitors as a new anchor therapy for combination therapies. The ability to target the major oncogenic driver in so many cancers and allows for the addition of other agents and sequential use — that’s how I and most of the field are seeing where we are going,” said Andrew Aguirre, a medical oncologist and pancreatic cancer scientist at Dana Farber Cancer Institute.

And check out a Q&A with a pancreatic cancer expert from NYU Langone’s Perlmutter Cancer Center on why Rev Meds' drug is generating excitement, and what it could mean broadly for the field.

accolades

Breakthrough Prize honors gene therapy and ALS discovery

From STAT's Jason Mast: The Breakthrough Prize — the billionaire-backed prize fund that bills itself as “The Oscars of Science” — has awarded prizes to three groups of scientists responsible for pioneering genetic therapies and for uncovering the most common mutation behind genetic ALS. Each group will split a $3 million prize.

Kathy High, Jean Bennett, and Albert Maguire received the prize for developing Luxturna, the first approved gene therapy in the U.S., for a rare form of blindness. Stuart Orkin and Swee Lay Thein were honored for finding BCL11A, the genetic switch that Vertex and CRISPR Therapeutics exploited to develop Casgevy, the gene-editing treatment for sickle cell disease. And Rosa Rademakers and Bryan Traynor were recognized for identifying C9orf72, a gene that’s mutated in 20% to 30% of inherited ALS cases.

The C9orf72 discovery has yet to lead to treatments — one attempt failed four years ago — but has changed basic ALS research. Similarly gene therapies like Luxturna and Casgevy have yet to transform rare diseases, yet efforts are underway to change that, by developing similar treatments for other inherited eye disorders and a safer, more scalable gene-editing treatment for sickle cell.

Biotech

Extended use of Nektar drug shows promise in alopecia

Nektar Therapeutics said this morning that extended treatment with its experimental drug, called rezpeg, promoted greater hair growth in people with severe alopecia areata, an autoimmune condition that attacks hair follicles.

After one year, 27% of participants treated with either a low or high dose of rezpeg achieved a clinically meaningful hair-growth outcome, Nektar said. The response, formally known as SALT Score 20, is defined as 80% or more of the scalp covered by hair.

A Phase 3 study of the drug in alopecia areata will begin later this year, the company said.

policy

Trump executive order boosts psychedelics development

President Trump over the weekend boosted the development of psychedelic drugs in an executive order, directing the federal government to rush access to treatments and reevaluate their status as controlled substances.

The order directs the Food and Drug Administration to expedite some psychedelics as breakthrough drugs, as well as allowing them to be used through right-to-try legislation, which allow terminally ill patients to try experimental drugs outside of usual regulatory pathways.

Marty Makary, the commissioner of the FDA, said three priority review vouchers would be given to three serotonin 2a agonists this week. He said decisions from the agency could be expected later this summer.

drug pricing
PBM transparency push meets fierce industry resistance

The Trump administration’s attempt to force drug-pricing transparency is hitting a predictable wall with pharmaceutical benefit managers and insurers. They’re arguing against a proposed Department of Labor rule, saying that it’s illegal — and overly burdensome — to require the disclosure of rebates, fees, and other revenue streams.

Employers, unions, and outfits like the Mark Cuban Cost Plus Drug Company broadly support the effort — some even say it doesn’t go far enough. But biopharma companies are playing both sides a bit — backing scrutiny of PBMs but pushing to keep individual drug prices confidential, STAT’s Bob Herman writes.

“By requiring disclosure of PBM revenue streams derived from manufacturers, pharmacies, and other third parties, the proposed rule effectively regulates aspects of the pharmaceutical marketplace that exist outside the ERISA plan relationship,” a top lobbyist for PBMs said.