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Beacon’s XLRP gene therapy surges toward FDA review
Beacon Therapeutics reported that its gene therapy laruparetigene zovaparvovec met the FDA-endorsed primary endpoint in its pivotal Phase II/III VISTA study in X-linked retinitis pigmentosa. The...
FDA/Advisory momentum for Grail’s multi-cancer blood test
Ahead of an FDA advisory committee meeting, Grail’s stock jumped as briefing materials posted by the agency suggested the regulator may take a favorable view of Galleri. The test is already...
FDA approval expands pediatric vascular anomaly care with safer dosing signals
A new study evaluated sirolimus dosing strategies in children with vascular anomalies and reported findings aimed at improving safety. With sirolimus now a key treatment in pediatric vascular...
Intrabio secures FDA nod for ataxia-telangiectasia treatment
Intrabio’s Aqneursa (levacetylleucine) won FDA approval as the first treatment for ataxia-telangiectasia, following its prior U.S. authorization in Niemann-Pick disease type C. The approval...
New FDA pilot program targets faster Phase I IND start-up
The FDA unveiled a pilot program designed to speed early clinical development by pairing sponsors with research sites prequalified for key steps in the IND process. Under the program, sponsors can...
Beacon’s earlier BLA staging after pivotal ocular gene therapy win
Beacon reported that it is preparing a biologics license application after positive results from its pivotal trial in X-linked retinitis pigmentosa. The company described the clinical readout as...
iNFixion’s NF1 strategy wins BIO startup recognition
iNFixion Bioscience won a top prize at the BIO International Convention’s Start-Up Stadium competition as it advances an NF1 treatment strategy centered on boosting function of the healthy NF1...
Stem Cell Network backs RejuvenRx with inaugural Canadian investment
Canada’s Stem Cell Network made its first direct company investment by awarding $1 million to Montreal-based RejuvenRx through a new Incubation Investment Award program. SCN said the initiative is...
Alkermes’ orexin agonist signals ADHD efficacy in early trial
Alkermes reported early randomized Phase 1 data for ALKS 7290, an orexin agonist, in adults with ADHD. The company said the drug was well tolerated and that results showed a signal of efficacy...
Grail cancer detection test heads into FDA advisory scrutiny
Shares in Grail surged after the FDA posted documents ahead of an advisory committee meeting on the company’s multi-cancer blood test, Galleri. The panel is scheduled for Wednesday, and the FDA is...
Beacon’s XLRP gene therapy clears pivotal hurdle
Beacon Therapeutics reported that its X-linked retinitis pigmentosa gene therapy, laruparetigene zovaparvovec (laru-zova), met the primary endpoint in the FDA-endorsed pivotal VISTA study. The...
Radiopharma consolidation accelerates with Telix–ITM mega-deal
Telix Pharmaceuticals agreed to acquire German isotope supplier ITM Isotope Technologies Munich in a stock deal valued at about $1.65 billion upfront, with potential back-end milestone payments....
FDA moves to streamline Phase I setup via sponsor-site IND prep
The FDA launched a pilot program that pairs drug sponsors with prequalified research institutions to expedite Phase I IND preparation and reduce delays to first-in-human studies. Under the...
NIH grant oversight proposals spark Capitol Hill backlash
Lawmakers and biomedical research advocates raised concerns about reported efforts to tighten political control over NIH grantmaking. United for Cures executive director Erica Sward warned that...
Alkermes orexin agonist posts early ADHD signal
Alkermes said its orexin agonist ALKS 7290 showed a signal of efficacy in a randomized Phase 1 study for ADHD, while reporting the therapy was well tolerated. The company presented early data in...
Cue pushes forward after Phase 2 chronic hives rebound
Cue Biopharma reported positive Phase 2 results for CUE-221 in chronic spontaneous urticaria (CSU), positioning the candidate for a Phase 2b/3 trial. Conducted in China, the study enrolled 145...
Chugai targets next-gen Hemlibra follow-on with NXT007
Chugai’s Lasker award-winning Hemlibra developers outlined efforts to make the company’s next-generation hemophilia A bispecific obsolete. The program centers on NXT007, designed to increase...
IntraBio gets FDA approval for ataxia-telangiectasia treatment
The FDA approved IntraBio’s Aqneursa (levacetylleucine), marking the first treatment for ataxia-telangiectasia, a rare neurodegenerative disorder. IntraBio previously received FDA approval for...
FDA updates nonanimal testing rules for preclinical programs
The FDA issued a final rule clarifying how nonanimal testing methods can be used in preclinical development when appropriate for assessing safety of drugs and biologics for human use. The agency...
Radiopharma M&A accelerates into scale and supply control
Telix Pharmaceuticals has agreed to acquire Isotope Technologies Munich (ITM) in a deal worth up to $1.65B upfront, potentially reaching about $2.35B with milestones, creating a more vertically...