PTC To Test RG7800 in SMA

The investigational compound is designed to raise levels of the SMN protein, a lack of which is the underlying cause of spinal muscular atrophy (SMA)

Article Highlights:

  • PTC Therapeutics, a New Jesey-based biopharmaceutical company, will move its experimental, oral compound RG7800 from testing in healthy volunteers to testing in adults and children with spinal muscular atrophy (SMA).
  • The compound is designed to change the way cells interpret genetic information from the SMN2 gene and cause more full-length SMN protein production.
  • The new phase 1b-2a trial in SMA patients will test the safety and tolerability of RG7800, which have been demonstrated in healthy volunteers in an earlier study.
by Margaret Wahl on November 19, 2014 - 3:36pm

PTC Therapeutics, a South Plainfield, N.J., biopharmaceutical company, says it will soon open a trial to test the safety and tolerability of its investigational drug RG7800 in adults and children with spinal muscular atrophy (SMA). The announcement was made in a Nov. 19, 2014, press release.

The drug, now being developed by PTC in collaboration with corporate partner Roche and other entities,  is designed to change the way instructions from a gene called SMN2 are interpreted by cells, with the goal of raising levels of a protein called SMN (survival of motor neuron). A deficiency of full-length, fully functional SMN protein is the underlying cause of SMA.

MDA's support of foundational research on the genetics of SMA and the production of SMN protein have contributed to this and other SMA drug trials.

Stuart Peltz, CEO of PTC Therapeutics, said, "We are excited to initiate the next phase of this program and begin a trial in SMA patients with our partners. We believe that the encouraging clinical results and preclinical data from relevant disease models are promising indications that the orally administered small molecule RG7800 has the potential to modify the splicing of the SMN2 gene and generate more full-length SMN mRNA [final instructions for the SMN protein] in SMA patients."

An earlier, phase 1 study of the compound in healthy volunteers showed that all doses studied were safe, well tolerated and demonstrated a dose-related, beneficial effect on SMN2 gene processing.

The new study will be a phase 1b-2a study in approximately 48 SMA patients. Participants will be randomly assigned to receive RG7800 by mouth or an oral placebo for 12 weeks.

For details and updated information as it becomes available, see PTC Therapeutics; or enter RG7800 AND SMA in the search box at

For more about PTC's SMA drug development program, including a video, see PTC's Spinal Muscular Atrophy Program.

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