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.

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