Shares of Solid Biosciences (SLDB) are under pressure after the company announced preliminary findings from IGNITE DMD, its Phase I/II dose-ascending clinical trial evaluating the safety and efficacy of SGT-001 microdystrophin gene transfer for the treatment of Duchenne muscular dystrophy, or DMD. Commenting on the study results, H.C. Wainwright analyst Debjit Chattopadhyay told investors that the company's microdystrophin expression data "pales in comparison" to those from Sarepta Therapeutics (SRPT). Further, Chattopadhyay argued that if the trajectory of Solid's data holds, Sarepta's 12 to 18-month lead is "insurmountable."
DMD TRIAL RESULTS: Solid Biosciences announced preliminary findings from IGNITE DMD on Thursday. In the company's Phase I/II dose-ascending clinical trial evaluating the safety and efficacy of SGT-001 microdystrophin gene transfer for the treatment of DMD, initial three-month biopsy data showed low levels of microdystrophin protein expression. The company is currently engaging with the appropriate parties to dose escalate as planned and as soon as possible. Preliminary analyses are based on three-month biopsy data from the first three patients dosed with 5E13 vg/kg of SGT-001, the lowest dose outlined in the study protocol. In one patient, microdystrophin was detected via western blot below the five percent level of quantification of the assay and in approximately 10% of fibers via immunofluorescence. There were also signs of co-localization of neuronal nitric oxide synthase, nNOS, and beta-sarcoglycan associated with microdystrophin expression. In the second and third patients, microdystrophin was detected via immunofluorescence at very low levels, but it was undetectable via western blot. Six patients have been enrolled in IGNITE DMD, three to the active treatment group and three to the delayed treatment control group. The safety profile of SGT-001 remains unchanged and all patients continue to be followed per the study protocol.
'BAR MAY BE TOO HIGH' FOR PEERS: Commenting on Solid Biosciences' three-month biopsy data from the first three patients dosed, H.C. Wainwright's Chattopadhyay told investors that he believes microdystrophin expression data "pales in comparison to those from Sarepta." The analyst noted that Solid is advancing the program to the next higher dose and pointed out that preclinical data is suggestive of a dose-dependent increase in microdystrophin, which is a relevant consideration given that Sarepta's data reflects outcomes from patients dosed at 2x1014vg/kg versus the 5x1013vg/kg for Solid. Unfortunately, Solid's construct has elicited complement activation that necessitated the use of eculizumab, which might restrict the therapeutic window as the program advances, he contended. Overall, the analyst believes that if the trajectory of Solid's data holds, Sarepta's 12 to 18-month lead is "insurmountable." Sarepta is likely to be in a position to file for full approval with commercial supply on tap during late 2020, by which time a larger multicenter, and potentially multinational, registrational study is likely to be underway, he highlighted, adding that given the Food and Drug Administration’s position on functional endpoints, baring an unforeseen SAE-related program setback, it is "extremely unlikely for competition to leapfrog" Sarpeta. Further, given the commercial and technical challenges of bringing a microdystrophin gene therapy program to patients, the analyst would not be surprised if a competitor not leveraged to DMD potentially withdraws, "as microdystrophin gene therapy has the makings of a winner take all program." Chattopadhyay reiterated a Buy rating and $267 price target on Sarepta's shares.
PRICE ACTION: In morning trading, shares of Solid Biosciences have plunged about 67% to $7.46. Meanwhile, Sarepta's stock is fractionally down to $140.70.
Sarepta
-1.6 (-1.13%)
Solid Biosciences
-15.33 (-68.87%)