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EMA Opinion Bodes Well for Biopharma's Drug Getting Orphan Status
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The designation, opinion and asset are discussed in a ROTH Capital Partners report.

In a July 29 research note, ROTH Capital Partners analyst Scott Henry reported that Anavex Life Sciences Corp. (AVXL:NASDAQ) is pursuing orphan status from the European Medicines Agency (EMA) for ANAVEX 2-73 as a treatment for Rett syndrome.

A positive opinion from the EMA's Committee for Orphan Medicinal Products (COMP) was already issued, highlighted Henry. It means European regulators believe ANAVEX 2-73, a sigma-1 receptor agonist, for use in Rett syndrome patients meets the requirements for orphan drug designation. "This is a significant development milestone for the company," Henry commented.

Specifically, Rett syndrome is a rare, life threatening disease without satisfactory treatment. With fewer than 5 cases per 10,000 occurring in Europe, this form of autism has an associated life expectancy of 20–40 years, described Henry. There is no current treatment for the cause of Rett syndrome itself, only for comorbid conditions, for which drugs are the standard of care. The second criterion for orphan status is that ANAVEX 2-73 could potentially provide significant benefit to Rett patients.

The EMA COMP opinion bodes well for Anavex being granted the orphan drug designation in the European market, the analyst pointed out. In fact, ROTH expects it to happen within 30 days. (ANAVEX 2-73 already has orphan drug status in the United States, as of 2016.)

The benefits of the designation in the European Union (EU), Henry pointed out, include decreased regulatory fees for the company, help with protocol and document filing and, potentially, a faster path to regulatory approval in Europe. Anavex 2-73 also would have exclusivity in the EU for 10 years.

ANAVEX 2-73 is currently being evaluated in Rett syndrome in two Phase 2 clinical trials, one in the U.S., the other in Australia. Topline data are expected around year-end 2019 and early 2020, with results from the U.S. likely being released first. "Provided ANAVEX 2-73 will achieve both primary safety and quality positive signal from the secondary efficacy measures, these studies could provide a commercial path for ANAVEX 2-73 as the first to market for the treatment of Rett syndrome in the U.S. and Europe," Henry noted.

ROTH has a Buy rating and a $10 per share target price on Anavex, whose stock is currently trading at around $2.57 per share.


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Disclosures from ROTH Capital Partners, Anavex Life Sciences Corp., Flash Note, July 29, 2019

Regulation Analyst Certification ("Reg AC"): The research analyst primarily responsible for the content of this report certifies the following under Reg AC: I hereby certify that all views expressed in this report accurately reflect my personal views about the subject company or companies and its or their securities. I also certify that no part of my compensation was, is or will be, directly or indirectly, related to the specific recommendations or views expressed in this report.

ROTH makes a market in shares of Anavex Life Sciences Corp. and as such, buys and sells from customers on a principal basis.

Shares of Anavex Life Sciences Corp. may be subject to the Securities and Exchange Commission's Penny Stock Rules, which may set forth sales practice requirements for certain low-priced securities.

ROTH Capital Partners, LLC expects to receive or intends to seek compensation for investment banking or other business relationships with the covered companies mentioned in this report in the next three months.

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