Selecta's (SELB) Gene Therapy Gets Rare Pediatric Disease Tag
Selecta Biosciences, Inc. SELB along with North Carolina-based privately-held company Asklepios BioPharmaceutical, Inc. announced that the FDA has granted the Rare Pediatric Disease (RPD) designation to their investigational candidate MMA-101.
This gene therapy candidate is currently being developed in pre-clinical studies for the treatment of isolated methylmalonic acidemia (MMA), an inherited rare metabolic disorder caused by methylmalonyl-CoA mutase (MMUT) gene mutation.
Notably, the RPD tag is granted by the regulatory body in case of serious or life-threatening diseases, which usually affect less than 200,000 people in the United States, primarily children aged 18 years and below.
Additionally, the RPD status will allow a sponsor an opportunity to obtain a voucher from the FDA after the regulatory agency approves any rare pediatric disease. The company may use the voucher to obtain a priority review for a new drug application (NDA) or a biologics license application (BLA) submitted later.
The companies remain on track to initiate a phase I study on MMA-101 for treating patients with MMA in the first half of 2021 with preliminary data expected in the second half.
Shares of Selecta have lost 8.4% so far this year compared with the industry’s decrease of 1.9%.
We note that in June 2020, Selecta entered into a research and license option agreement with Sarepta Therapeutics SRPT. The deal granted the latter an option to license the rights to use the ImmTOR immune tolerance platform of the former for developing therapies to address neuromuscular diseases like Duchene muscular dystrophy and certain limb-girdle muscular dystrophies.
Also, during the same month, Selecta announced a strategic license agreement with Swedish Orphan Biovitrum AB (Sobi) to help advance the development and commercialization of the company’s lead pipeline candidate SEL-212. The candidate is being developed to treat patients with chronic refractory gout and resolve its debilitating symptoms including flares and gouty arthritis.
Zacks Rank & Stocks to Consider
Selecta currently carries a Zacks Rank #5 (Strong Sell).
Better-ranked stocks in the biotech sector include Allena Pharmaceuticals, Inc. ALNA and Eton Pharmaceuticals, Inc. ETON, both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Allena Pharmaceuticals’ loss per share estimates have narrowed 6.1% for 2020 and 9.7% for 2021 over the past 60 days.
Eton Pharmaceuticals’ loss per share estimates have narrowed 10.2% for 2020 and 2.2% for 2021 over the past 60 days. The stock has rallied 10% year to date.
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