Spark Therapeutics Announces $15-Million Milestone Payment

Spark Therapeutics recently announced it has earned a $15-million payment from Pfizer Inc. for achieving a pre-specified safety and efficacy profile development milestone in the ongoing hemophilia B Phase I/II trial of investigational SPK-9001, which has received breakthrough therapy and orphan product designations from the US FDA.

“We continue to make strong, tangible progress with our hemophilia pipeline, and achievement of this second milestone marks further advancement in the development of our investigational gene therapy for hemophilia B,” said Jeffrey D. Marrazzo, Chief Executive Officer of Spark Therapeutics. “We look forward to reporting additional data as we continue to document the clinical experience with SPK-9001.”

Under the terms of the license agreement with Pfizer, Spark Therapeutics received a $20-million upfront payment upon entering into the agreement in 2014, and a $15-million milestone payment in December 2015 for progress with the development program. The company is eligible to receive up to an additional $230 million in aggregate for achieving future development and commercial milestones, as well as royalties calculated as a low-teen percentage of net sales on any potential SPK-FIX products. Spark Therapeutics maintains responsibility for the clinical development of SPK-FIX product candidates through the completion of Phase I/II trials. Thereafter, Pfizer has responsibility for further clinical development, achieving regulatory approvals and potential global commercialization.

The SPK-FIX program leverages a more than two-decade long history of hemophilia gene therapy research and clinical development conducted by Spark Therapeutics and its founding scientific team. SPK-9001 is a novel bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimized, high-activity human factor IX variant enabling endogenous production of factor IX.

Spark Therapeutics, a fully integrated company, strives to challenge the inevitability of genetic disease by discovering, developing, and delivering gene therapies that address inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver. Its validated platform successfully has delivered proof-of-concept data with investigational gene therapies in the retina and liver. Its most advanced investigational candidate, voretigene neparvovec, in development for the treatment of RPE65-mediated IRD, has received orphan designations in the US and European Union, and breakthrough therapy designation in the US. The pipeline also includes SPK-7001, in a Phase I/II trial for choroideremia, and two hemophilia development programs: SPK-9001 (which also has received both breakthrough therapy and orphan product designations) in a Phase I/II trial for hemophilia B being developed in collaboration with Pfizer, and SPK-8011, in a Phase I/II trial for hemophilia A to which Spark Therapeutics retains global commercialization rights. For more information, visit www.sparktx.com.