Update for February 23, 2022

Regulatory Update
 
The FDA approved mitapivat (Pyrukynd, Agios Pharmaceuticals), on 2/17/2022, for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
 
The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee will meet on 3/30/2022 to review AMX0035 for the treatment of ALS.
 
Announced Research Updates
 
Cytokinetics announced that in the 20-week, 276 patient, Phase II METEORIC-HF trial, treatment with omecamtiv mecarbi did not improve peak oxygen uptake during cardiopulmonary exercise testing compared to placebo in patients with HFrEF.
 
Sage and Biogen announced that in the 15-day, 440 patient, Phase III, CORAL trial, adding zuranolone to open-label standard of care antidepressant (ADT) resulted in an 8.9 point decrease in the HAMD-17 total score at day 3 compared to a 7 point decrease with ADT alone in patients with major depressive disorder. However, the difference between zuranolone and placebo was no longer significant by day 15.
 
BMS announced that in the 16-week, 100 patient, Phase III, VALOR-HCM trial, fewer patients treated with mavacamten elected to undergo septal reduction therapy compared to placebo in patients with symptomatic obstructive hypertrophic cardiomyopathy.
 
Gilead announced that patients who completed the 14-day blinded portion of the CAPELLA trial were enrolled in an open-label extension where they received lenacapavir subcutaneously every six months and an optimized background regimen. After 52 weeks, 83% (30/36) achieved an undetectable viral load (< 50 copies/mL).
 
GSK paused enrollment in three trials of its RSV vaccine candidate in pregnant patients, at the recommendation of the Independent Data Monitoring Committee, based on an observation from a routine safety assessment.
 
Published Research Updates
 
In the 92 patient, Phase III, REALITY trial, patients treated with apatinib achieved progression-free survival of 22.2 months compared to 4.5 months with placebo in Chinese patients with radioactive iodine–refractory differentiated thyroid cancer. A Phase II trial in adults 60 and older is not affected.
 
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