Alnylam's new pipeline star gets on FDA fast track; ARCH-backed biotech nabs orphan drug status
→ Just a day after Blackstone and Alnylam sketched out a R&D partnership as part of a sprawling $2 billion investment plan, the biotech said it’s obtained fast track designation for one of the drugs highlighted in the deal. The status was conferred to vutrisiran in the treatment of polyneuropathy of hATTR amyloidosis — an indication that Blackstone will help fund but not receive royalties on. Alnylam is now eligible to submit a rolling NDA for the drug, for which Phase III results are expected in early 2021.
→ Epirium, the ARCH-backed biotech that unveiled a $85 million round dedicated to its synthetic flavanol, has secured orphan drug designation for the drug as a treatment of Duchenne and Becker muscular dystrophy. “Our initial emphasis on Becker muscular dystrophy stems from our research demonstrating that EB 002 may stimulate mitochondrial biogenesis, leading to the restoration of tissue bioenergetics, while upregulating production of key proteins which may lead to improved muscle structure and function,” CMO Ransi Somaratne said. It’s unclear what stage the program is in.
→ Global health-focused VC Adjuvant Capital has led a $15 million Series A round for 54gene, helping bootstrap a research, services and development venture tapping into Africa’s genomic datasets. Launched in January 2019, the Nigeria-based company aims to address the lack of African representation in the data commonly used in genome-wide association studies (GWAS), with the goal of discovering insights of use in therapeutic development. Novartis, the Gates Foundation and other investors also participated.
→ Stanley Crooke has received another $500,000 donation for the n-Lorem Foundation, supporting his non-profit’s efforts to provide antisense treatments for patients with ultra-rare, N of 1 diseases.