Company Spotlight: Spark Therapeutics

Shares of Spark Therapeutics Inc. (ONCE) have gained nearly 43% year-to-date compared to 21% rise in the iShares Nasdaq Biotechnology Index (ETF) during the same period.

Spark is focused on developing gene therapies for inherited retinal diseases, neurodegenerative diseases and for diseases that can be addressed by targeting the liver.

The Company’s lead drug candidate is Luxturna (Voretigene Neparvovec) for inherited retinal disease due to biallelic RPE65 mutations.

Biallelic RPE65 mutations are characterized by a continuous decline in visual function beginning in early childhood.

Spark’s Luxturna is under FDA review, with a decision date set for January 12, 2018.

SPK-7001, being developed for Choroideremia, is another clinical drug candidate in the Company’s pipeline of products for inherited retinal disease.

Choroideremia is a rare inherited disorder that causes progressive vision loss, ultimately leading to complete blindness, and it mostly affects males.

SPK-7001 is under a phase I/II trial, and the study initially had 10 participants with later-stage Choroideremia.

An interim analysis of 4 out of 10 participants showed nonstatistically significant indications of efficacy on one or more endpoints. According to the Company, the non-significance of results may be due to duration of follow-up and later-stage of disease in that cohort.

So, the Company is expanding the study to include 5 additional participants at an earlier-stage of disease. The enrollment of earlier-stage participants is planned for completion this quarter (3Q, 2017).

The other clinical programs of the Company are focused on liver-mediated diseases, and they are:

— SPK-9001, being developed in collaboration with Pfizer Inc. (PFE), for Hemophilia B, which is under a phase I/II trial.
— SPK-8011 for Hemophilia A, which is also under a phase I/II trial.

Hemophilia B, also called factor IX (FIX) deficiency or Christmas disease, is a genetic disorder that causes the blood to take a long time to clot because of a deficiency or dysfunction of factor IX, a clotting protein. The current standard of care requires recurrent intravenous infusions of either plasma-derived or recombinant factor IX to control and prevent bleeding episodes.

Earlier in the month, the Company announced encouraging interim data of 10 participants in its ongoing phase I/II clinical trial of SPK-9001.

The SPK-9001 data demonstrated a 99% reduction in annualized infusion rate (AIR) and a 96% reduction in annualized bleeding rate…

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