European Medicine Agency (EMA)
The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) will have their monthly meeting this week from June 10th to June 13th.
US Food and Drug Administration (FDA)
- Rytelo (imetelstat, Geron), the first telomerase inhibitor, was approved by the FDA for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia. Geron press release.
- The FDA approved a label extension for Arexvy (Respiratory Syncytial Virus (RSV) Vaccine, GSK) to include the prevention of RSV lower respiratory tract disease (LRTD) in adults aged 50 through 59 who are at increased risk. GSK press release.
- The FDA’s Psychopharmacologic Drugs Advisory Committee (PDAC) voted against Lykos Therapeutics’ application for the use of midomafetamine (MDMA, a non classic psychedelic, with psychological intervention ) for the treatment of post-traumatic stress disorder (PTSD) in adults, citing safety concerns.Lykos press release.
New data from clinical trials (Phase 2, Phase 3)
This week, a lot of good news from the reading of the clinical trials and only one negative.
Biomea Fusion saw their Phase 1 / 2 trials in diabetes put on clinical hold by the FDA. The regulator decision was based on potential drug-induced hepatotoxicity levels observed in dose escalation studies. Biomea press release.
On the other side, 13 studies reported positive data:
- BridgeBio shared positive results from the Phase 2 study of oral infigratinib in patients with achondroplasia. BridgeBio press release.
- Viking Therapeutics announced that its Phase 2b study of VK2809 in patients with biopsy-confirmed non-alcoholic steatohepatitis (NASH) met its primary endpoint. Viking press release.
- AbbVie announced positive results from the Phase 2 trial of Elahere (mirvetuximab soravtansine) monotherapy in heavily pre-treated patients with folate receptor-alpha (FRα) positive, platinum-sensitive ovarian cancer (PSOC). AbbVie press release.
- Moderna and Merck shared positive results from the Phase 2b study of mRNA-4157 (V940) plus Keytruda to treat high-risk melanoma (stage III/IV) following complete resection. Moderna press release.
- Takeda presented positive results from its Phase 2b trial of TAK-861 in narcolepsy type 1 (NT1). Takeda press release.
- BMS published data from three trials in which Breyanzi (lisocabtagene maraleucel; liso-cel) demonstrated clinically meaningful outcomes in a wide range of B-cell malignancies. BMS press release.
- GSK reported positive results from a Phase 2 study of Jemperli (dostarlimab) for locally advanced mismatch repair deficient (dMMR) rectal cancer. Jemperli was found to be 100% effective in treating all patients who were enrolled in the trial. GSK press release.
- Agios Pharmaceuticals announced that the Phase 3 trial of mitapivat met the primary endpoint and all key secondary endpoints in adults with transfusion-dependent alpha- or beta-thalassemia. Agios press release.
- Alligator Bioscience published positive data from their Phase 2 study of mitazalimab in first-line metastatic pancreatic cancer. Alligator press release.
- Annexon Biosciences reported positive results from its Phase 3 trial of ANX005 in treating Guillain-Barré syndrome (GBS). Annexon press release.
- Gilead Sciences shared promising interim data from their Phase 3 study of seladelpar in patients with primary biliary cholangitis (PBC). Gilead press release.
- Amgen announced that the Phase 3 trial of Uplizna (inebilizumab-cdon) to treat Immunoglobulin G4-related disease (IgG4-RD) met its primary endpoint. Amgen press release.
- Arrowhead Pharmaceuticals announced positive results from the Phase 3 study of plozasiran in patients with genetically confirmed or clinically diagnosed familial chylomicronemia syndrome (FCS). Arrowhead press release.
- MAIA Biotechnology reported positive efficacy data from its Phase 2 trial of THIO (6-thio-2′-deoxyguanosine) sequenced with checkpoint inhibitor cemiplimab (Libtayo) to treat advanced non-small cell lung cancer (NSCLC). MAIA press release.
