Shares of Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) were trading at $393.83, down $78.44, or 16%, on top-line results from the phase II dose-ranging study evaluating the safety and efficacy of its NaV1.8 pain signal inhibitor, VX-993, in tackling acute pain after bunionectomy. Treatment did not result in a statistically significant improvement on the primary endpoint of the time-weighted Sum of the Pain Intensity Difference from 0-48 hours compared to placebo. Vertex said work with the candidate as a monotherapy in pain will be dropped.

Agios: Patients’ deaths don’t alter Pyrukynd’s benefit-risk profile

Agios Pharmaceuticals Inc. said four patients’ deaths tied to anemia therapy Pyrukynd (mitapivat) that were announced in a securities analyst’s Aug. 4 note to investors hasn’t changed the drug’s already established benefit-risk profile included on the drug’s U.S. prescribing information. The data came from the U.S FDA’s Adverse Event Reporting System Database via a Freedom of Information Act request from the analyst. The company’s stock plunged more than 20% during trading on Aug. 4 but rallied to close about 3% upward for the day. At midday today, shares were flat at $35.93. Pyrukynd, a pyruvate kinase activator, was approved by the FDA in February 2022 for hemolytic anemia in adults with a deficiency of the enzyme.

CML heating up, Novartis reaching Scemblix goals

Novartis AG’s recently described progress with Scemblix (asciminib), a third-generation allosteric tyrosine kinase inhibitor first approved by the U.S. FDA in October 2021 for Philadelphia chromosome-positive chronic myeloid leukemia (CML), brought more attention to the space. Competitors include Enliven Therapeutics Inc. with ELVN-001 and Terns Pharmaceuticals Inc. with TERN-701. Scemblix “blazed the trail for third-generation allosteric TKIs” in CML, Mizuho analyst Graig Suvannavejh pointed out, and the Terns compound may be poised to take advantage.

US HRSA testing the waters on 340B discounts as rebates

After shutting down manufacturers’ efforts last year to offer the mandated 340B discounts on prescription drugs as a rebate rather than an up-front price, the U.S. Health Resources and Services Administration (HRSA) is now inching the rebate door open for drugs that were selected for the first round of Medicare price negotiations. HRSA, which oversees the drug discount program, unveiled a voluntary 340B Rebate Model Pilot Program that would be open to qualifying drug manufacturers and that would allow the discounts to be applied as a rebate to all covered entities, including eligible hospitals, clinics and contract pharmacies. Manufacturers have until Sept. 15 to submit a rebate plan to HRSA for inclusion in the pilot.

AI hacks disordered proteins

Deep learning tools for protein design can also be used to create molecules that bind to them. Certain peptides, such as intrinsically disordered proteins (IDPs), are challenging to target due to their variable nature. However, scientists from the lab of Nobel laureate David Baker have developed a method to generate binders for IDPs by searching the world’s largest protein database with their AI-powered tool RFdiffusion.

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AB, Alveolus, Artelo, Catalyst, Clarametyx, Dyne, Genentech, Hansa, Helix, Hillevax, Inmune, Lava, Medicus, Praxis, Prime Medicine, Shilpa, Vor, Xencor, Xoma