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  Molecule Spotlight: Risdiplam, the First Approved Small Molecule Splicing Modulator for Spinal Muscular Atrophy

  Spinal Muscular Atrophy (SMA)—the leading genetic cause of childhood mortality—is a disease in which the gene SMN1 is deleted or mutated. The highly similar gene SMN2 can undergo alternative splicing to generate functional protein transcripts of the SMN1 gene product, although native levels of the protein are too low to overcome the pathology. Here we…

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  Ten Examples of Best-in-Class Drugs

  It’s best to be first, but drugs that are approved later with the same mechanism of action can also create significant value for patients and society by being best-in-class (BIC), or what clinicians refer to as “gold standard.” Recently we highlighted key factors that can make a follow-on drug be considered a best-in-class drug. This…

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  Why Did GSK Pay $1.9B for Momelotinib, an Oral Ph. III Myelofibrosis Candidate?

  Momelotinib, an oral JAK1/2 inhibitor for myelofibrosis, is going to GSK as part of a $1.9 billion acquisition of Sierra Oncology. Anyone that’s ever worked on a kinase program probably looked at this structure and raised their eyebrows. $1.9 billion for another JAK2 inhibitor? It’s probably not super selective either, you might think… and you’d…

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  Small Molecule of the Year – 2021

  We asked the global drug discovery community to nominate and vote on their favorite molecule from 2021, and the results are in. The 2021 choice for Drug Hunter’s Small Molecule of the Year is Pfizer’s CoV-2 Mpro Inhibitor, PF-07321332 (nirmatrelvir, Paxlovid). Published in a November 2021 Science article, the molecule was praised for its impressive…

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  A Gram-Daily Aldehyde for Anemia: Voxelotor

  On Feb. 16, 12:00PM PST, Dr. Mira Pochon, Staff Scientist and Head of Translational Biology at Global Blood Therapeutics, gave a PBSS/CLSA webinar on one of 2019’s drug approvals, voxelotor (GBT440).  Mira has been with GBT since it’s early days in 2011.  GBT’s chemistry team was led by their CSO Dr. Brian Metcalf, former head…

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  A Polarized C-H···O Interaction Against IRAK4

  The C-H — O hydrogen bond is so well established in structural biology today, that it’s been overlooked that the C-H — O hydrogen bond was first proposed by a woman, June Sutor, in a 1960’s Nature article.[1] Hydrogen bonds were then assumed to be linear (A-H···A’ between 150-180º), and the C-H — O hydrogen…