Here is your life science and drug development newsletter, sharing updates on clinical data and regulators decisions in the first 2 weeks of February 2025.
Fascinating Science: what after GLP-1 agonists?
Amid growing attention to the long-term efficacy and safety of GLP-1 agonists, a recent Nature article reviews future therapies aimed at reducing weight, preventing lean mass loss, and achieving lasting effects.
Pharmaceutical and biotech companies are exploring combining enteral peptides or targeting muscle energy use.
European Medicine Agency (EMA)
Andembry (garadacimab, CSL) was approved by the European Commission (EC) for preventing hereditary angioedema (HAE) attacks in adults and adolescents aged 12 and older. CSL press release.
Beyonttra (acoramidis, BridgeBio Pharma) was approved by the EC for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). BridgeBio press release.
US Food and Drug Administration (FDA)
The following approvals have been granted by the FDA within the past two weeks:
- Emblaveo (aztreonam and avibactam, AbbVie) was approved for adults with limited or no alternatives for treating complicated intra-abdominal infections (cIAI). AbbVie press release.
- Izervay (avacincaptad pegol intravitreal solution, Astellas) received a label expansion approval for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Astellas press release.
- Romvimza (vimseltinib, Ono Therapeutics) was approved for adult patients with symptomatic tenosynovial giant cell tumor (TGCT). FDA press release.
- Gomekli (mirdametinib, SpringWorks Therapeutics) was approved for the treatment of adult and pediatric patients 2 years and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. SpringWorks press release.
- Adcetris (brentuximab vedotin, Pfizer) in combination with lenalidomide and a rituximab product for the treatment of adult patients with relapsed or refractory large B-cell lymphoma (LBCL). Pfizer press release.
- Penmenvy (Meningococcal Groups A, B, C, W, and Y Vaccine, GSK) was approved for use in individuals aged 10 through 25 years. GSK press release.
- Evrysdi (risdiplam, Roche) tablet was approved for people living with spinal muscular atrophy (SMA). Roche press release.
New data from clinical trials (Phase 2, Phase 3)
Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between February 3 and 16, 2025:
- Following a safety committee recommendation, Pliant Therapeutics paused enrollment and dosing in the ongoing Phase 2b trial of bexotegrast in patients with idiopathic pulmonary fibrosis (IPF). Pliant press release.
- Bristol Myers Squibb announced the results of 2 studies: Phase 3 trial of Opdualag (nivolumab and relatlimab-rmbw) for the adjuvant treatment of patients with completely resected stage III-IV melanoma did not meet its primary endpoint. BMS press release. Phase 2 trial evaluating Breyanzi (lisocabtagene maraleucel) in adult patients with relapsed or refractory indolent B-cell non-Hodgkin lymphoma met its primary endpoint. BMS press release.
- Roche shared positive results from the Phase 3 trial of Gazyva/Gazyvaro (obinutuzumab) in people with active lupus nephritis (LN). Roche press release.
- Qlaris Bio reported positive results from two Phase 2 trials investigating QLS‑111 in patients with primary open angle glaucoma (POAG) and ocular hypertension (OHT). Qlaris Bio press release.
- Novo Nordisk announced positive results from the Phase 3 trial of Mim8 in children (aged 1-11 years old) with hemophilia A with and without inhibitors. Novo Nordisk press release.
- Regeneron shared positive data from the Phase 3 trial investigating Eylea HD (aflibercept) for the treatment of patients with macular edema. Regeneron press release.
- Spinogenix reported positive results from the Phase 2 study of its SPG601 therapy in adult men with Fragile X syndrome (FXS). Spinogenix press release.
- Alto Neuroscience announced positive results for the Phase 2b trial of ALTO-300 as an adjunctive treatment for patients with major depressive disorder (MDD). Alto Neuroscience press release.
- Johnson and Johnson announced the discontinuation of its Phase 3 study examining J&J and Sanofi’s investigational vaccine for extraintestinal pathogenic Escherichia coli after results were found to be underwhelming. J&J press release.
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