Rare Diseases
On the back of a stellar showing in Phase III, apitegromab is going to be filed in the US and Europe in the coming weeks for spinal muscular atrophy and CEO Jay Backstrom believes it will be a $2bn blockbuster.
The US biotech’s Duchenne muscular dystrophy drug is “undoubtedly the most successful gene therapy launch in all of history,” according to CEO Doug Ingram. “We have barely scratched the surface of the opportunity in front of us.”
Two treatments that target elF2B, a regulator of the integrated stress response that appears to be overactive in the progressive motor neurone disease, have missed their primary and secondary endpoints in a landmark platform trial.
From gene therapies to small molecule drugs, investors seemed to have eschewed the risks of biotech company investment propositions in 2024 but last year’s disappointments provided good lessons going forward.
Taiwan-based PharmaEssentia hopes to add essential thrombocytopenia to the label for its pegylated interferon product, already approved to treat polycythemia vera.
The Swiss-headquartered, NASDAQ-listed group has posted a Phase II win for OCS-05 in acute optic neuritis, which opens up its potential in additional, and much larger, neuro-ophthalmic indications, CEO Riad Sherif tells Scrip.
The Swiss giant’s blockbuster gene therapy is currently only administered intravenously into patients with spinal muscular atrophy under two years of age. Its intrathecal candidate, called OAV-101, has just passed a Phase III test which could expand its use in children and young adults.
UniQure said discussions with the FDA resulted in a path forward for seeking accelerated approval of its gene therapy AMT-130 in Huntington’s disease.
Primary biliary cholangitis or cirrhosis, PBC, is a lesser-known cousin of MASH but a silent killer, nevertheless. Scrip studies data from Citeline’s Pharmaprojects and separately Evaluate Pharma to reveal a promising pipeline and drug revenue forecasts, with APAC firms accounting for half the pipeline of drugs
Novartis will also pay up to $1.9bn for milestones and share US profits on the Phase II RNA-splicing candidate. PTC is discussing potential accelerated approval with the US FDA.
Deal Snapshot: The essential tremor space has not seen innovation in treatment for decades. Acadia believes Saniona's GABAA-α3 positive allosteric modulator could be the answer.
The Swiss pharma acquired Kate Therapeutics for up to $1.1bn, gaining preclinical gene therapies for neuromuscular conditions and novel platform technology.
But differences in trial design means it is hard to handicap GSK’s product against Mirum’s volixibat.
The company is trading below cash after admitting that an adverse event in a Rett syndrome trial left a patient critically ill.
The company has reached agreement with the US FDA on a pivotal trial for RGX-202 through an accelerated pathway.
With its data monitoring committee saying a Phase III trial of a drug-device combo product in pediatric myopia was not on track for success, Eyenovia will end the study and consider its options.
The MEK inhibitor, already approved for pediatric patients with the rare condition neurofibromatosis type 1, showed benefits for adults in a Phase III trial.
Despite a successful Phase III study of ALXN-1840 for the inherited disorder that prevents the liver from filtering copper out of the body, the UK major's rare disease division Alexion pulled the plug on the program last summer. However, Illinois-based biotech Monopar is confident in its potential.
Ocuphire will shift toward gene therapy for inherited retinal degeneration via a merger that will take Opus Genetics public. The firm will also be responsible for one of Ocuphire’s pipeline projects.
CEO of Sumitomo Pharma America unit, Adele M. Gulfo, draws from experiences working across companies including Pfizer, to advise on go-to-market strategies for a portfolio across primary care to rare disease.