Originally published on January 22, 2014.
PTC announces that its SMA drug program, partnered with Roche, begins Phase I Safety Trials in healthy, adult volunteers. This brings the total number of SMA programs in clinical development to 5.
PTC Therapeutics, the SMA Foundation, and Roche announced today that their joint research program in SMA has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers. SMA is a genetic neuromuscular disorder that leads to muscle weakness and mobility impairment and is considered the leading genetic cause of death in infants and toddlers.
Luca Santarelli, Head of Neuroscience, Ophthalmology and Rare Diseases at Roche commented: “The compelling science behind this project and the highly synergistic alliance among Roche, PTC Therapeutics and the SMA Foundation are the groundwork for this research program. Although still early in development, we consider every step towards a therapeutic option in SMA of high importance for patients and families affected by this devastating and currently untreatable condition.”
The orally available small molecule compounds in the program are designed to target the underlying cause of the disease by increasing SMN protein levels in the nervous system, muscles, and other tissues. It is estimated that approximately one in 10,000 children are born with this rare genetic disorder.
2014 SMA Drug Pipeline Update
With this important achievement, the total number of SMA programs in clinical development is now 5. Clear evidence of progress is being seen in SMA with:
-5 programs in clinical trials.
-15 programs total in drug development.
-12 companies involved in these drug programs
Cure SMA has funded almost two thirds of all the ongoing drug programs for SMA.
1) Programs are advancing into the clinical trial stage of testing. There are now 5 novel programs being testing in clinical trials for SMA with several more following behind.
2) There is broad diversity of approaches in development. Diversity is important in case a particular approach turns out not to have benefit in patients. Currently there are six drug strategies being advanced for SMA: a) Small molecule SMN enhancers, b) Neuroprotectants, c) ASOs to correct SMN2 splicing, d) Stem cell therapy, e) Gene therapy, and f) Regulators of muscle function.
3) The total number of programs is growing. There are now 15 novel SMA therapeutic programs in various stages of preclinical and clinical development, up from just one a decade ago. This volume is needed to balance the low probability of programs successfully advancing to final FDA approval.
4) Funding and resources from government and industry are increasing. There are now 12 companies actively investing in the SMA drug pipeline. Industry expertise and resources are essential to advance programs through the expensive and complicated clinical stages of development.