Drug Development
The mid-stage disappointment in Alzheimer’s disease delivers another blow to Neuphoria Therapeutics, which in November last year was forced to launch a strategic business review after a Phase 3 trial in social anxiety disorder failed.
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A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.
Recent approvals for Corcept Therapeutics and Merck have injected momentum into the space, where GSK, Allarity Therapeutics, OSE Immunotherapies and others are advancing their own candidates.
The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.
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A single 1-mg/kg dose of Eli Lilly’s base editor can lower LDL cholesterol levels by 62% in patients with a heritable form of hypercholesterolemia. The pharma acquired the asset last year in the $1.3 billion Verve Therapeutics buy.
Partners Summit Therapeutics and Akeso are expected to steal the show at the American Society of Clinical Oncology’s annual conference with data from their potential Keytruda rival, alongside Revolution Medicine’s groundbreaking pancreatic cancer candidate and other assets that could reshape patient care.
The tragic tale of TIGIT is well known. However, RIPK1, myc, STING and alpha-synuclein have also left a trail of failed clinical trials, canceled partnerships and sunk investments in their wake.
Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line.
Treatment with the TROP2 ADC sac-TMT led to a 70% objective response rate and progression-free survival was “significantly improved” as compared to placebo—the second positive readout for the asset this week.
Biogen and Denali’s Parkinson’s disease drug failed to significantly slow disease progression in a Phase 2b study, missing both primary and secondary endpoints.
Lilly met analysts’ sky-high expectations with 28.3% weight loss over 80 weeks for the triple hormone receptor agonist retatrutide in a highly anticipated readout on Thursday.
In September last year, a group of concerned stockholders raised alarm about Vaxart’s proposed reverse stock split, which the biotech was pushing for despite strong opposition from shareholders.
After being hit by safety issues and subpar results in another trial, BioMarin’s Phase 3 test of Voxzogo for a rare skeletal disorder called hypochondroplasia showed efficacy “solidly above” what the drug has shown for achondroplasia, which causes dwarfism.
In this episode of Denatured, as part of our series on the European life sciences investment ecosystem, you’ll be hearing from Regina Hodits, managing director at Angelini Ventures and Sofia Ioannidou, VC partner at Andera Life Sciences. They explore Germany’s biotech and life sciences ecosystem, including the science, infrastructure and policy changes needed to help European companies scale globally while staying rooted in Europe.