PTC Therapeutics announced that RG7916, an additional SMN2 splicing modifier from
the company's joint development program with Roche and the SMA
Foundation in spinal muscular atrophy (SMA), has entered clinical
development. RG7916 is designed to shift SMN2 pre-mRNA splicing toward
the production of full length SMN mRNA. A Phase 1 study in healthy
volunteers has been initiated to investigate the safety, tolerability,
pharmacokinetics and pharmacodynamics of RG7916.
"We are pleased to initiate clinical development of a second candidate in our SMA collaboration," said Stuart W. Peltz,
Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "The goal of
this Phase 1 study is to understand more about the safety and activity
of RG7916 and be in a position to compare the profiles of each of our
development compounds to determine the best path forward for our SMA
program. SMA is the most common genetic cause of infant mortality and
one of the most common rare diseases. Currently there are no available
therapies to treat the underlying cause of the disease. PTC and our
collaboration partners are committed to advancing potential solutions
for SMA patients through our SMN2 alternative splicing program."
Two compounds are currently in
clinical development within the SMA program, RG7800 and RG7916. The most
advanced compound, RG7800, is the subject of a Phase 2 randomized,
double-blind, placebo-controlled trial called Moonfish in adult and
pediatric patients with SMA. Dosing in the Moonfish trial was suspended
in April 2015 and the trial was placed on clinical hold to investigate a non-clinical safety finding observed in a longer-term animal study.
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 RG7800 and RG7916
is being executed by Roche and overseen by a joint steering committee
with members from PTC, Roche, and the SMA Foundation.
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