Rapid and Sustained Effect Demonstrated in Phase 3 TrialDr. Russell presented additional top-line analyses from the pivotal Phase 3 trial showing the rapid and sustained impact of SPK-RPE65 throughout the entire one-year study period. Significant differences emerged in both MT and FST by the first study visit, at 30 days. These effects were reproduced consistently at each subsequent study visit (at days 90, 180 and one year). Phase 3 Trial of SPK-RPE65: MT and FST Over Time (mITT) http://resource.globenewswire.com/Resource/Download/5331d5dc-30f7-4a6e-8dbf-e4d718cc08a3?size=o Durable Response Continuing through Three Years in Comparable Cohort from Earlier Phase 1 Study Dr. Russell also presented data on the durability of effect after three years as measured by MT and FST in a cohort of subjects that participated in the Phase 1 open-label 102 study, and would likely have met the eligibility criteria for the Phase 3 trial. All of these subjects continue to experience a durable improvement over three years from the time of administration to the contralateral, or second eye, with observation ongoing. These subjects received the same dose and volume of SPK-RPE65 that was used in the pivotal Phase 3 trial. The figures below reflect data from all subjects available for follow-up at each time point reported. Spark and the clinical investigators continue to follow study participants to evaluate the durability of response, and will provide further updates in the future through a series of peer-reviewed presentations and publications. Phase 1 Trial of SPK-RPE65: Durability of MT and FST Over Time (102-injected eye) http://resource.globenewswire.com/Resource/Download/9481e13e-e7df-4bbd-b6f1-7863ea5e11c5?size=o "We are pleased to provide these additional informative data, the totality of which highlight the rapid, sustained and durable effect associated with SPK-RPE65 across multiple functional and physiological parameters, at time points ranging from 30 days to three years," said Jeffrey D. Marrazzo, co-founder and chief executive officer of Spark. "We will continue to analyze the data from our groundbreaking pivotal Phase 3 trial in order to further elucidate the potential positive, meaningful impact that SPK-RPE65 can have on the lives of patients with RPE65-mediated blinding conditions." Principal Investigator Albert Maguire, MD, professor of ophthalmology at the Perelman School of Medicine of the University of Pennsylvania, will present additional data about SPK-RPE65 during the American Academy of Ophthalmology Retina Sub-specialty meeting on November 14 th in Las Vegas, NV. Pivotal, Phase 3 Trial Overview The pivotal Phase 3 trial of SPK-RPE65 is the first successful randomized, controlled Phase 3 trial ever completed in gene therapy for a genetic disease. The multicenter trial randomized 31 subjects with confirmed RPE65 gene mutations. The ITT population included 21 subjects in the intervention group and 10 in the control group. For the primary endpoint, subjects were evaluated at multiple time points over the course of one year for their performance in navigating a mobility course under a variety of specified light levels ranging from one lux (equivalent to a moonless summer night) to 400 lux (a brightly lit office) using the bilateral testing condition. Each attempt was recorded, and the videos were sent to independent, centralized, masked graders to assign a pass/fail score based on speed and accuracy with which the subjects navigated the course.
In addition to the primary endpoint, the statistical analysis plan included three secondary endpoints tested statistically in the following hierarchical order:
- FST (white light), which reflects underlying physiological function by measuring light sensitivity of the entire visual field.
- Change in MT score for the assigned first eye, which compares the MT performance between baseline and year one for the first eye injected for the intervention group and, for the control group during the control year, the first eye injected after they crossed over.
- Visual acuity (VA) testing, which measures changes in central vision by assessing the ability of the subject to read a standard eye chart.
|Primary outcome (ITT)|
|MT change score, bilateral||p = 0.001|
|Secondary outcomes (ITT)|
|FST, averaged over both eyes||p < 0.001|
|MT change score, first injected eye||p = 0.001|
|VA, averaged over both eyes||p = 0.17|
ContactsInvestor RelationsSpark Therapeutics, Inc.Stephen W. WebsterChief Financial Officer(855) SPARKTX (1-855-772-7589)Media Ten Bridge CommunicationsDan Quinn(781) email@example.comFinancial MediaTeneo StrategyAndy Maas(212) firstname.lastname@example.org