Biogen Ends Enrollment In Eye Disorder Study On Gene Therapy

Biogen Inc. (NASDAQ:BIIB) announced that it has completed enrollment in the global phase III STAR study on its gene therapy, timrepigene emparvovec, being developed to treat choroideremia (CHM). CHM is a rare, inherited retinal disorder that causes progressive loss of vision due to degeneration of the choroid and retina. This mainly affects males.

Timrepigene emparvovec (previously BIIB111) was added to Biogen’s portfolio in June as part of its acquisition of Nightstar Therapeutics.

Shares of the company have lost 4.2% year to date compared with the industry’s decline of 0.9%.

The STAR study enrolled 170 adult males with CHM. The primary endpoint of the study is the proportion of patients who will experience an improvement of at least 15 letters from baseline in best corrected visual acuity (BCVA) at 12 months post the treatment, as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity protocol.

The study was initiated based on positive proof-of-concept data from the phase I/II studies, which showed that at month 24, more than 90% of patients treated with timrepigene emparvovec via targeted subretinal injection maintained visual acuity instead of experiencing the natural decline in BCVA expected in this degenerative disease.

Notably, there are several companies, which are developing or have developed drugs to treat eye disease. Spark Therapeutics, Inc. (NASDAQ:ONCE) has Luxturna in its portfolio, which is the first directly administered gene therapy approved in the United States for a rare form of inherited vision loss. The treatment would help those with Leber congenital amaurosis, a rare genetic retinal disease.

We note that gene therapy helps mitigate the adverse effects of a malfunctioning disease-causing gene. The therapy intends to add a functional gene back in a person's system replacing the malfunctioning one to cure diseases.

Zacks Rank and Stocks to Consider

Biogen currently carries a Zacks Rank #3 (Hold).

A few better-ranked stocks in the biotech sector are Alkermes Plc. (NASDAQ:ALKS) and Anika Therapeutics Inc. (NASDAQ:ANIK) , both sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Alkermes’ earnings per share estimates have increased from 36 cents to 52 cents for 2019 in the past 60 days. The company delivered a positive earnings surprise in the trailing four quarters by 236.80%, on average.

Anika’s earnings per share estimates have increased from $1.75 to $2.03 for 2019 and from $1.36 to $1.62 for 2020 in the past 60 days. The company delivered a positive earnings surprise in the trailing four quarters by 53.31%, on average.

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