How an immunological Trojan horse could treat disease

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Good morning, everyone. Damian here with some breathlessly anticipated news from Vertex Pharmaceuticals, an exit interview from an FDA lifer, and the debut of the $100 genome.

The need-to-know this morning

• Pfizer reported fourth-quarter and 2023 earnings, and reaffirmed 2024 guidance.
• Kura Oncology reported no cases of differentiation syndrome, a closely tracked side effect, in a new study that combines its experimental menin inhibitor, called ziftomenib, with chemotherapy in patients with acute myeloid leukemia. High rates of differentiation syndrome reported in previous ziftomenib monotherapy studies were a concern.
• Regeneron Pharmaceuticals is spending $5 million to acquire 2Seventy Bio’s pipeline of cell therapies for cancer and other diseases. A smaller 2Seventy will carry on, supporting the marketing and further development of Abecma, a CAR-T for multiple myeloma partnered with Bristol Myers Squibb.
• Gilead Sciences increased its ownership stake in Arcus Biosciences. The two companies are jointly developing an anti-TIGIT antibody for cancer.

Vertex’s pain drug hits in Phase 3

A novel pain medicine from Vertex Pharmaceuticals met its primary goals in three Phase 3 studies, setting the stage for an FDA application that could dramatically change the company’s fortunes.

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