Agios Pharmaceuticals Inc. said the 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 on the drug’s U.S. prescribing information.

Vertex Pharmaceuticals Inc.’s bad news from a phase II pain study and separately on the regulatory front caused shares (NASDAQ:VRTX) to close Aug. 5 at $393.83, down $78.44, or 16%. Vertex rolled out top-line results from the phase II dose-ranging study to test the safety and efficacy of its NaV1.8 pain signal inhibitor, VX-993, in tackling acute pain after bunionectomy.  

Novartis AG’s financial update included good news about Scemblix (asciminib), the first and only U.S. FDA-approved allosteric inhibitor for chronic myeloid leukemia (CML), which gained clearance in October 2021. The drug introduced a new mechanism of action by specifically targeting the ABL myristoyl pocket.

Subtyping is what made precision medicine in cancer a reality. And for successful drug discovery in all its stages, finding subtypes in Alzheimer’s disease is all but imperative. Prior to the approval of the modestly effective Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd.), Kisunla (donanemab, Eli Lilly and Co.), and the since-withdrawn Aduhelm (aducanumab, Biogen Inc./Eisai Co. Ltd.), more than a dozen failed phase III clinical trials were all that amyloid-targeting drugs had to show for themselves for decades of effort.

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.