Back to top

Image: Bigstock

Axovant's GM1 Gangliosidosis Candidate Gets Orphan Drug Tag

Read MoreHide Full Article

Axovant Gene Therapies Ltd. (AXGT - Free Report) announced that the FDA has granted an Orphan Drug designation to its investigational gene therapy candidate, AXO-AAV-GM1, being developed in a phase I/II study for the potential treatment of GM1 gangliosidosis, a rare, inherited neurodegenerative lysosomal storage disorder.

Notably, the Orphan Drug designation is granted to drugs capable of treating rare diseases that affect less than 200,000 people in the United States. This status also makes the company entitled to certain other benefits, including tax credits, clinical study expenses and a seven-year market exclusivity upon potential approval from the regulatory body.

Despite the positive news, shares of Axovant declined 4.9% on Monday. In fact, the stock has plunged 43.9% so far this year against the industry’s growth of 2.3%.



We remind investors that, in May, Axovant dosed the first patient with AXO-AAV-GM1 for the given indication. The company is looking to complete enrollment in part A of the AXO-AAV-GM1 registrational program, evaluating the safety and efficacy of the candidate, and plans to announce six-month data from Part A in mid-2020. Currently, there are no FDA-approved treatments available for GM1 gangliosidosis.

Meanwhile, Axovant is developing another gene therapy candidate, AXO-AAV-GM2, which is being developed 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 phase II study, as a potential one-time treatment of Parkinson’s disease.

Zacks Rank & Other Stock to Consider

Axovant currently carries a Zacks Rank #2 (Buy). A few other top-ranked stocks in the healthcare sector include Anika Therapeutics Inc. (ANIK - Free Report) , Idera Pharmaceuticals, Inc. (IDRA - Free Report) and Intellia Therapeutics, Inc. (NTLA - Free Report) , all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Anika’s earnings estimates have been revised 16% upward for 2019 and 19.1% for 2020 over the past 60 days. The stock has rallied 78.8% so far this year.

Idera’s loss per share estimates have narrowed 9.8% for 2019 and 17.2% for 2020 over the past 60 days.

Intellia’s loss per share estimates have narrowed 8.7% for 2019 and 15.2% for 2020 over the past 60 days.

Biggest Tech Breakthrough in a Generation

Be among the early investors in the new type of device that experts say could impact society as much as the discovery of electricity. Current technology will soon be outdated and replaced by these new devices. In the process, it’s expected to create 22 million jobs and generate $12.3 trillion in activity.

A select few stocks could skyrocket the most as rollout accelerates for this new tech. Early investors could see gains similar to buying Microsoft in the 1990s. Zacks’ just-released special report reveals 8 stocks to watch. The report is only available for a limited time.

See 8 breakthrough stocks now>>