Gadopiclenol: Reducing Gadolinium Retention Risks Through Coordination Chemistry

The FDA recently approved Agios' Pyrukynd (mitapivat, AG-348) , a first-in-class, allosteric activator of pyruvate kinase (PK), for treatment of hemolytic anemia in adults with the genetic condition, pyruvate kinase deficiency. The drug is given orally with a starting dose of 5 mg BID, titrating up to 50 mg BID depending on hemoglobin (Hb) [...]

Among the small molecule highlights in January’s news were a $100M+ Series A for a PARP1 + PI3Kα-focused company, clinical data with an SLC inhibitor for PKU, a 5-HT2C superagonist for seizures, and a NK1,3 dual antagonist for women’s health. A novel anti-obesity agent from phenotypic screening also made headlines, and the close of a major acquisition may bring relief to the industry. You can read about these notable scientific highlights and more below.

This October roundup features key clinical updates, including FDA approvals, significant trial results for major drug candidates, the initiation of new clinical trials, and updates on halted studies.

Our latest roundup of small molecules in the news includes two potential upcoming approvals: rezafungin Rezafungin (CD-101) is a once-weekly IV antifungal agent and next-generation echinocandin (glucan synthase inhibitor) that demonstrated non-inferiority vs. standard of care caspofungin (IV QD) (NCT03667690). An FDA AdCom voted 14 to 1 in [...]

Relyvrio, developed by Amylyx Pharmaceuticals, is a combination treatment of sodium phenylbutyrate and taurursodiol, approved by the FDA in 2022 for ALS (“Lou Gehrig’s disease”). The approval was controversial, with an Advisory Committee initially voting against approval based on Ph. II data. Now, Ph. III data have confirmed the drug is no better than placebo in ALS, turning this phenotypic drug discovery story from two undergrads into a post-mortem highlighting the importance of clearly defined molecular mechanisms of action. This article reviews what happened and what we can learn.