Spinraza rival? With DMD flops in the rearview mirror, PTC Therapeutics posts strong SMA data
After years trying to push a thrice-failed Duchenne drug into the US market (and ultimately failing to do so), PTC Therapeutics is finally onto something interesting.
The New Jersey company’s stock $PTCT is soaring on news of a completely different program — one partnered with Roche — that’s just impressed analysts and investors with updated data from a Phase I trial.
This drug, called risdiplam or RG7916, is being tested in babies with spinal muscular atrophy, a genetic condition that robs people of physical strength by affecting the motor nerve cells in the spinal cord. It’s the number one genetic cause of death for infants. The disease is caused by a gene mutation that prevents the body from producing a muscle development protein called SMN, which leads to atrophy of the muscles, motor impairment, and paralysis. Babies born with the severest kind of SMA never learn to crawl, sit up, or walk, and many lose the ability to breathe. They often die within 18 months.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.