The need-to-know this morning

Eli Lilly said it is buying Ajax Therapeutics, a privately held developer of blood cancer drugs, for as much as $2.3 billion. Ajax's lead medicine is a next-generation JAK2 inhibitor currently being evaluated in a Phase 1 study in patients with myelofibrosis.
Ligand Pharmaceuticals said it is buying Xoma, an aggregator of drug royalties, for $739 million.
Compass Therapeutics said its drug for advanced biliary cancer delayed tumor progression but failed to prolong survival compared to chemotherapy in a clinical trial.
BridgeBio shares were higher this morning on reports from the weekend that Pfizer had settled two of three patent cases regarding Vyndamax, its medicine for the heart condition known as ATTR-CM. While terms of the settlement have not been disclosed, any extension of patent protection for Vyndamax is also positive for BridgeBio, which sells a competing ATTR-CM drug called Attruby.

gene editing

CRISPR therapy slashes rare swelling attacks

Intellia Therapeutics said this morning that its one-time CRISPR treatment lonvo-z cut attack rates by 87% in a Phase 3 trial of hereditary angioedema, STAT’s Jason Mast writes. With more than 60% of patients becoming attack-free and no serious safety signals reported, the therapy is being positioned as a functional cure — or at least a major upgrade over existing options.

The results put the company on track for what could be the first in vivo gene-editing approval. It’s also a key test of whether patients will opt for a permanent, DNA-altering intervention over highly effective but repeat treatments, especially given lingering safety concerns in the field and one prior death in a separate Intellia study.

gene therapy

Astellas retries XLMTM gene therapy after deaths

From STAT’s Jason Mast: Astellas said it had dosed the first patient with its next-generation gene therapy for the fatal rare muscle disease X-linked myotubular myopathy (XLMTM).

A prior gene therapy for XLMTM, purchased by Astellas as part of a $3 billion acquisition in 2019, showed remarkable results across more than 20 young boys. More than half were able to come off ventilators, six stood on their own and five could walk without assistance. But in 2020 and 2021, four different boys died of liver failure linked to the treatment.

The new therapy was licensed in 2023 from Kate Therapeutics, a Broad Institute spinout. It uses a gene-ferrying virus engineered to travel directly to muscle, allowing the company to use a dose that more than 100-fold smaller. If successful, the therapy could bode well not only for the XLMTM community but also other next-generation gene therapies designed to reach specific organs. In late 2024, Novartis purchased Kate and its pipeline of other neuromuscular gene therapies for up to $1.1 billion.

chronic disease

Oruka’s psoriasis therapy posts strong results

A long-acting injectable treatment for plaque psoriasis helped 63% of patients achieve complete skin clearance in a mid-stage clinical trial, its maker, Oruka Therapeutics, reported.

The company also said an updated analysis of blood exposure levels supported the potential for the drug, ORKA-001, to be injected just once per year, STAT's Adam Feuerstein reports.

Oruka still needs to conduct late-stage clinical trials, but the latest results bolster ORKA-001’s potential to become a new treatment for the autoimmune skin disease with remission rates equal to or greater than current commercial blockbusters Skyrizi and Bimzelx, while requiring fewer injections.

biotech

Hair loss pill succeeds in late-stage study

Veradermics said its oral hair loss drug VDPHL01 boosted hair growth in a late-stage trial, with treated men gaining roughly 30 to 33 hairs per square centimeter over six months versus about seven in the placebo group. The vast majority reported visible improvement, STAT’s Allison DeAngelis writes — a level of consistency the company’s CEO says has long been missing in dermatology.

The pill is a reworked version of minoxidil designed to last longer in the body than existing oral options, and it arrives amid rising investor interest in aesthetic conditions like androgenetic alopecia, which affects tens of millions of Americans but hasn’t seen a new treatment in over a decade.

With another Phase 3 readout expected later this year and parallel studies in women underway, the company is positioning the drug as a mass market blockbuster.

psychedelics

FDA will fast-track review of three psychedelics

The FDA is working to accelerate reviews of psychedelic drugs from Compass Pathways, Usona Institute, and Transcend Therapeutics, handing out priority review vouchers as part of a broader push by the Trump administration to expand access to the once-taboo treatments.

The move, tied to a recent executive order and fueled in part by advocacy from figures like Joe Rogan and military veterans, marks a notable shift in Republican attitudes toward psychedelics.

Compass, which has already completed two Phase 3 trials of its psilocybin therapy COMP360, could now see a decision this year. Meanwhile, Usona and Transcend are pushing forward late-stage programs of their own, including a non-hallucinogenic MDMA-like drug for PTSD.

"‘Psychedelics’ was a bad word,” Deborah Mash, who leads DemeRx, a company working on an ibogaine derivative that’s been greenlighted for human trials by the FDA, told STAT. “President Trump has really given us momentum. He’s opened the door, and the new leadership in the FDA is making changes that are going to speed up drug development for this class of compounds.”