PTC Therapeutics (PTCT 47.16, +9.61, +25.59%) presented updated interim clinical
data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in
babies with Type 1 Spinal Muscular Atrophy (SMA) at the 22nd Annual SMA
Researcher Meeting in Dallas on June 16. The data demonstrated that at Day 182,
over 90% of the babies involved in the trial achieved a greater than 4-point
increase in CHOP-INTEND scores compared to baseline. The CHOP-INTEND data were
further supported by video footage presented by Dr. Giovanni Baranello
demonstrating achievement of antigravity movements, the ability to control
their head, roll, or sit, by babies participating in FIREFISH. Part 2 of the
pivotal FIREFISH study is ongoing. The SMA program is a collaboration dating
back to 2006 between PTC, the SMA Foundation, and Roche (RHHBY).
Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder that is the leading genetic cause of mortality in infants and toddlers. It is caused by a missing or defective survival of motor neuron 1 (SMN1) gene, which results in reduced levels of SMN protein. Insufficient levels of SMN protein are responsible for the loss of motor neurons within the spinal cord, which leads in its most severe form to muscle atrophy and death. An estimated 1 in every 11,000 children born are affected by this devastating disease.
Risdiplam is an investigational splicing modifier that targets the survival motor neuron 2 (SMN2) RNA to restore a functional transcript. Risdiplam is taken orally, crosses the blood brain barrier, and shows systemic distribution to the organs that are affected by low levels of SMN protein.
The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) is a test designed to measure motor milestone development of patients with SMA Type 1.
"We are delighted that up to 6.5-fold increase of protein production has translated into clinical impact for these babies in the FIREFISH study," commented Stuart W. Peltz, Ph.D. Chief Executive Officer of PTC Therapeutics, upon the results. "The survival data and CHOP-INTEND scores are very promising, since babies with Type 1 SMA typically do not experience functional motor milestone improvement based on natural history. We look forward to sharing updates for the programs as the data further develop at upcoming medical meetings."
"I am impressed by the clinical data and the changes reported by the patients' families," stated Dr. Baranello of the Fondazione Istituto Neurologico Carlo Besta in Milan, Italy. "Data on motor function seem more encouraging when we consider that we are seeing motor function improvements and milestones achievement at this early stage of the study, which was essentially a dose-finding study and most of the infants included have received their first dose after the age of 5 months. It is exciting to see evidence of clinical benefit from a systemic oral treatment for SMA."
PTCT opened steeply higher and lodged new three-year highs in early trading. Above $50/share, the company has a ~$2.3 bln valuation. Most of the rare-disease drug maker's revenue comes from Translarna, a treatment for Duchenne muscular dystrophy.
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