Acadia, Neuren Headed To Phase III With Trofinetide In Rett Syndrome
Positive Phase II data in the unmet medical need could mean patients will have a therapy specific to their disease, rather than burdensome symptom-by-symptom treatment.
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The Italian firm has some talking to do with the US FDA before it can unblind Phase III data on sarizotan, its Rett syndrome therapy which could be "potentially transformative" for Newron.
Sarepta licenses gene therapy candidate from Lacerta, also makes equity investment. Neuren licenses North American rights to rare disease drug trofinetide to Acadia.
Moderna is pursuing a single-antigen strategy even though some of its mRNA vaccines have included multiple antigens; the SARS-CoV-2 spike protein likely provides the best antigen for developing immunity against the virus.