Brain cancer

Sanofi and BMS paid big money for rare disease and cancer assets, while Regeneron got in the obesity game; AstraZeneca, Gilead and Amgen shone at ASCO; RFK Jr. and the CDC appeared to disagree over COVID-19 vaccine recommendations and several news outlets are questioning the validity of the White House’s Make America Healthy Again report.
The medium-sized biopharma is showing off new results from dordaviprone and Zepzelca, both of which were acquired through Jazz Pharmaceuticals’ dealmaking over the last five years.
Stifel analysts were bullish on the data, which showed a 16.5% drop in body-mass index among patients with damage to the hypothalamus taking Rhythm Pharmaceuticals’ Imcivree.
At the heart of the deal is the drug candidate dordaviprone, which is months away from a regulatory verdict for its use in H3 K27M-mutated diffuse glioma.
Madrigal Pharmaceuticals, X4 Pharmaceuticals and Day One Biopharmaceuticals secured their maiden approvals this year in metabolic dysfunction-associated steatohepatitis, WHIM syndrome and pediatric low-grade glioma. Geron Corporation and ImmunityBio also notched wins.
Emboldened by technological advances and a deeper knowledge of glioblastoma, Merck, Kazia Therapeutics, CorriXR Therapeutics and others are targeting the often-fatal brain tumor.
Six months after treatment with the radiopharmaceutical therapy, 77.8% of patients with meningioma were alive and had not experienced further disease progression, beating the 26% benchmark established in earlier studies.
Servier Pharmaceuticals’ vorasidenib on Tuesday secured the FDA’s green light for the treatment of patients with grade 2 gliomas carrying mutations in the IDH gene.
Kazia Therapeutics is eyeing an FDA accelerated approval pathway for its investigational glioblastoma drug paxalisib, with Wednesday’s release of secondary overall survival data from a Phase II/III trial.
FDA
The FDA on Tuesday approved Day One Biopharmaceuticals’ type II RAF inhibitor Ojemda, which is designed to penetrate the blood-brain barrier, for the treatment of relapsed or refractory pediatric low-grade glioma.
PRESS RELEASES