Astellas retries XLMTM gene therapy after deaths

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New hair loss data for a sort of amped-up minoxidil is looking very plush. Also Astellas is reentering the clinic with a next-generation gene therapy for XLMTM and Intellia’s CRISPR therapy for hereditary angioedema impresses.

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.

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.

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