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Axovant's Gene Therapy Gets Rare Pediatric Disease Status

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Axovant Gene Therapies Ltd. announced that the FDA has granted Rare Pediatric Disease (RPD) designation to its investigational gene therapy candidate, AXO-AAV-GM1, currently being developed in a phase I/II study for the treatment of GM1 gangliosidosis, a rare, inherited neurodegenerative lysosomal storage disorder.

Notably, the RPD tag is granted by the FDA in case of serious or life-threatening diseases, which usually affect less than 200,000 people in the United States, primarily individuals aged 18 years and below.

Following the above development, shares of Axovant were up 3.9% on Friday. However, the stock has declined 22.1% so far this year against the industry’s increase of 2.7%.


Per the company, AXO-AAV-GM1 is the only gene therapy candidate, which is being developed for both infantile (Type I) and juvenile (Type II) GM1 gangliosidosis. In November 2019, the FDA granted an Orphan Drug designation to AXO-AAV-GM1 for the given indication.

Axovant plans to announce six-month data (Stage 1) from the low dose juvenile cohort in the fourth quarter of 2020. The company also aims to begin a study in the second half of this year involving the high dose cohort, which will evaluate both infantile and juvenile patients. Currently, there are no FDA-approved treatments available for GM1 gangliosidosis.

We note that Axovant is developing another gene therapy candidate AXO-AAV-GM2 in a phase I/II study as a potential one-time treatment of GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease).

The company is also evaluating AXO-Lenti-PD, an investigational gene therapy, in a phase II study as a potential one-time treatment of Parkinson’s disease.

Zacks Rank & Stocks to Consider

Axovant currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include AVEO Pharmaceuticals, Inc. (AVEO - Free Report) , Immunomedics, Inc. and Amicus Therapeutics, Inc. (FOLD - Free Report) , all carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

AVEO’s loss per share estimates have narrowed 25.6% for 2020 and 28.6% for 2021 over the past 60 days. The stock has increased 4.7% year to date.

Immunomedics loss per share estimates have narrowed 0.9% for 2020 and 5.5% for 2021 over the past 60 days. The stock has skyrocketed 306.4% year to date.

Amicus’ loss per share estimates have narrowed 2.9% for 2020 and 7.5% for 2021 over the past 60 days. The stock has rallied 55.2% year to date.

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