Spark Therapeutics (ONCE) Gene Therapy Succeeds in Trial

Shares of the Spark Therapeutics Inc. (ONCE) were trading up +30.84 or +70.20 percent to $74.77 in Monday’s premarket after the company announced early this morning positive top line results for Spark’s SPK-RPE6, a genetic treatment for mediated inherited retinal dystrophies. Spark Therapeutics stock closed at $43.93, up +1.57 or +3.71 percent in Friday’s regular trading session.

Philadelphia, Pennsylvania based Spark Therapeutics was started in October of 2013 upon the acquisition of in licensed intellectual property as well as the development and commercial rights to preclinical and clinical programs started and advanced at the gene therapy center of the Children’s Hospital of Philadelphia and other institutions.
The company’s most advanced candidate is SPK-RPE65, a gene therapy treatment for rare blinding conditions caused by mutations in the RPE65 gene. The company had its initial public offering in January of 2015 securing more than $268 million in financing. Spark Therapeutics is collaborating with a number of other companies including Genable Technologies Ltd, Pfizer (PFE) and Clearside Biomedical.

This morning, Sparks announced it had obtained positive top line results from a Phase 3 pivotal trial for SPK-RPE65, its lead gene therapy product candidate used for the treatment of a type of inherited eye disorder. The pivotal trial met its endpoint and demonstrated improvements of functional vision in the group treated versus the control group as measured by the change in bilateral mobility testing over the period of one year.

The study was conducted on 31 patients with inherited retinal dystrophies, a wide range of hereditary disorders that affect the retina. SPK-RPE65 is a gene therapy treatment aimed at inserting corrective genes into cells that have malfunctioned. No serious adverse effects or harmful immune reactions were observed in the trial.

Subjects treated with SPK-RPI65 outperformed subjects in the control group in the first two secondary endpoints, which consist of full field light sensitivity threshold testing and the results of the mobility test change for the first eye injected. The third secondary endpoint did not show statistically significant evidence of benefits.

Kathy High, MD, co-founder, President and Chief Scientific Officer of Spark said in the company’s press release that, “This is an important moment for the field of gene therapy, and demonstrates Spark’s ability to carefully and precisely integrate technologies and approaches across a range of disciplines to move the concept of gene therapy toward a therapeutic reality for patients.”

Jeffrey D. Marrazzo, co-founder and Chief Executive Officer of Spark noted that, “These data further validate Spark’s platform which is being applied across a growing pipeline of clinical and preclinical programs in gene therapy. The impact of these results provides the strongest indication yet of the role that Spark is uniquely poised to play across a wide range of inherited diseases.”

Spark plans to submit the treatment for approval by the FDA in 2016. The market and investors are showing their enthusiasm for the results in this morning’s premarket, driving the stock up over 70 percent. This morning’s action could have the stock test its all time high of $79.50 made back in March.

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Published on Oct 5, 2015
By Jay Hawk
Jay Hawk
Jay Hawk enjoyed a 12-year professional financial markets career incorporating extensive first hand futures and options experience obtained by trading in the stock, commodity and forex markets on U.S. exchanges. Since retiring as a full-time financial market professional, he has been actively trading stock, commodities, forex and options for his own account and managing funds for others, as well as writing financial market commentary and educational articles.

Copyrighted 2016. Content published with author's permission.

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