The clinical data were presented at the annual meeting of the American Academy of Neurology.
The presentation from Part 1, the Dose Finding section, of the ongoing FIREFISH study. The open-label study investigated RG7916, an oral survival motoneuron 2 (SMN2) splice modifier, in type 1 SMA patients.
The data from Part 1 were presented at the American Academy of Neurology 2018 Annual Meeting and showed that RG7916 was well tolerated at all doses and to date has no drug-related safety findings leading to withdrawal
In addition, no babies have required a tracheostomy or permanent ventilation since study initiation and no baby has lost the ability to swallow. The median age of first dose was 6.7 months and babies have received RG7916 for a duration of up to 14.8 months. Recruitment is ongoing globally for the pivotal second part of the FIREFISH study.
”RG7916 continues to demonstrate compelling results in SMA patients and we look forward to making further gains to combat this devastating, fatal disease,” said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. ”We are encouraged by this promising data as we continue our ongoing global recruitment efforts for the SMA programs.”
FIREFISH is an ongoing, global multi-center, open-label, seamless Phase 2 study evaluating the safety and efficacy of RG7916 in babies aged 1–7 months at enrollment with Type 1 SMA and two SMN2 gene copies. Data from 21 patients from the completed Part 1 portion of this study suggested that RG7916 produced a dose-dependent increase in SMN protein levels in babies with Type 1 SMA.
SMA Type 1 is a devastating disease in which babies rarely live past the second year of life without permanent nutritional and/or ventilatory support,” stated Dr. Giovanni Baranello, Fondazione Istituto Neurologico Carlo Besta in Milan, Italy. ”This early data on the infants’ ability to swallow and lack of need of permanent ventilatory support is very encouraging.”
The SMA program was initially developed by PTC Therapeutics in partnership with the SMA Foundation in 2006 to accelerate the development of a treatment for SMA. In November 2011, Roche gained an exclusive worldwide license to the PTC/SMA Foundation SMN2 alternative splicing program. The development of these compounds is being executed by Roche and overseen by a joint steering committee with members from PTC, Roche, and the SMA Foundation.
FIREFISH: An open-label, two-part clinical trial. Part 1 was a dose escalation study in 21 infants for a minimum of 4 weeks. The primary objective of Part 1 was to assess the safety profile of RG7916 in infants and determine the dose for Part 2. Part 2 is a single-arm study with the dose selected in Part 1 in approximately 40 infants with Type 1 SMA for 24 months, followed by an open-label extension. This study is recruiting globally.
SUNFISH: A double?blind, two?part, placebo?controlled trial. Part 1 enrolled patients with Type 2 or 3 SMA to evaluate safety, tolerability, and PK/PD of several RG7916 dose levels. The pivotal SUNFISH Part 2, in non?ambulant patients with Type 2 or 3 SMA, is evaluating safety and efficacy of the RG7916 dose level selected from Part 1. This study is recruiting globally.
JEWELFISH: An ongoing, exploratory, open-label study to establish the safety and tolerability of RG7916 in people who have previously participated in a study with another therapy targeting SMN2 splicing.