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With the sale of its migraine portfolio to Pfizer pending, Biohaven said troriluzole – a lead asset in the pipeline soon to be spun out in a new company – failed in a Phase III trial.
PTC CEO Stuart Peltz tells Scrip that cost watchdogs are likely to look favorably on the firm’s transformational gene therapy for a distressing rare brain disorder in children who could not even lift their heads before treatment.
Despite promising clinical data and solid regulatory plans, biotech firms hoping to address the unmet need in Sanfilippo Syndrome through gene therapy are facing financial struggles.
Oral or feeding tube administration of the cerebral neuroprotectant should offer flexible dosing options for amyotrophic lateral sclerosis patients, the firm says.
Emerging Company Profile: The British biotech, co-founded by experimental researcher Lady Amanda Feilding, is developing psychedelics therapeutics for a rare but debilitating headache disorder and mental health disorders.
Lecanemab is now filed for accelerated approval with the US FDA, but the wait for a Phase III readout could impact the hunt for Biogen’s next CEO.
Aduhelm commercialization was a bust, so Biogen’s next big hope for a potential blockbuster Alzheimer’s therapy is partner Eisai’s amyloid protofibril-targeting antibody lecanemab.
Pfizer’s $11.6bn acquisition of the company brings it full rights to the oral migraine drug Nurtec ODT and nasal spray zevegepant, with aims to build a $6bn migraine franchise.
In this week's podcast version of Five Must-Know Things: big changes at Biogen; Paxlovid still a work in progress; Farxiga goes stellar; Moderna hints at M&A; and a chat with Roche Pharma India’s CEO on rare diseases.
Investor Deerfield Management and genomic sequencing giant Illumina entered into a five-year partnership to identify novel targets and drug candidates that may be spun out into new companies.
The Ireland-headquartered firm will advance the candidate into US and EU narcolepsy studies as part of its pipeline diversification project as portfolio products are set to lose their patent exclusivity.
While spinal muscular atrophy therapies Evrysdi and Zolgensma have gained ground at the expense of pioneer Spinraza, emerging myostatin inhibitors promise a complementary mechanism that could enhance functional outcomes.
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