PTC Therapeutics Announces New Phase 1a/2b Clinical Trial

PTC Therapeutics, Inc. announced today that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) has started a Phase 1b/2a study in adult and pediatric patients. The placebo-controlled, randomized, multiple-dose study will enroll approximately 48 patients with SMA.

Because of a mutation in the survival motor neuron gene 1 (SMN1), individuals with SMA don’t produce survival motor neuron protein (SMN protein) at high enough levels. This program is one of several underway that target SMN2, the SMA “back-up gene.” This drug is the first orally available SMN2 splicing modifier for SMA to be tested in patients.

Drug Development Milestones

2014 has been a landmark year for SMA drug development. We now have 17 ongoing drug programs, and six have reached clinical trials—both the highest numbers ever. We also have 12 companies working on SMA drugs—another record number—including many of the largest pharmaceutical companies in the world. PTC and Roche are two of these major partners.

We’re excited to see our SMA community continue to expand. The more partnerships we can build, the more quickly we can move toward a treatment and cure for SMA.

To learn more about the trial enrollment criteria and clinical sites, visit

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