Shares of Solid Biosciences Inc. (SLDB - Free Report) surged 10.9% after the company announced that the FDA has lifted the clinical hold on its phase I/II trial, IGNITE DMD, for its experimental candidate, SGT-001.
SGT-001, Solid Biosciences’ lead candidate, is a microdystrophin gene transfer, which is being evaluated for the treatment of Duchenne muscular dystrophy (DMD).
The clinical hold was lifted as the FDA acknowledged that Solid Biosciences satisfactorily addressed all the issues raised.
The news gave investors much to cheer as SGT-001 is Solid Biosciences’ lead candidate. Solid Biosciences’ stock has gained 29.9% in the year so far, compared to the industry’s loss of 6.8%.
It has been a bumpy ride for Solid Biosciences so far, with the development of SGT-001.
We remind investors that the FDA had placed a clinical hold on IGNITE DMD, following the report of a serious adverse event (SAE) in the first patient dosed with SGT-001. The first patient dosed in the trial was a non-ambulatory adolescent, who received 5E13 vg/kg of SGT-001 on Feb 14, 2018.
However, the patient was hospitalized due to laboratory findings, which included a decrease in platelet count, followed by a reduction in red blood cell count and evidence of complement activation. The event was classified as a Suspected Unexpected Serious Adverse Reaction (SUSAR).
Earlier, Solid Biosciences filed an Investigational New Drug application (IND) for SGT-001 in September 2017 and initiated a phase I/II trial during the fourth quarter of 2017. In November 2017, the FDA notified Solid Biosciences that the company was not permitted to dose patients in the higher-dose group of IGNITE DMD, due to a partial clinical hold primarily related to the number of vials and manufacturing lots utilized per patient as well as manufacturing processes to support the higher-dose group. The issue was subsequently resolved.
Nevertheless, since the hold has been lifted, Solid Biosciences plans to resume the trial and reinitiate enrollment as quickly as possible after making the required changes.
We note that SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, in the United States, and Orphan Drug Designations in both the United States and the European Union.
Solid Biosciences plans to enroll and dose several children, prior to dosing additional adolescents. Moreover, Solid Biosciences can now also obtain the intermediate muscle biopsy at 45 days, post administration of SGT-001, to collect additional information about the time course of microdystrophin expression. Initial data from a pre-specified interim analysis of IGNITE DMD is expected in the second half of 2019.
Another company with a presence in the DMD market is Sarpeta Therapeutics (SRPT - Free Report) with Exondys 51, which is the first approved disease-modifying therapy for DMD in the United States.
Gene therapy has gained a lot of attention, of late, as quite a few companies are investing a lot of their R&D spend for developing candidates using gene therapy. Axovant Sciences (AXON - Free Report) recently licensed the exclusive worldwide rights to develop and commercialize AXO-LentiPD from Oxford BioMedica for Parkinsons’ disease.
uniQure (QURE - Free Report) is another gene therapy company, which is advancing a pipeline of proprietary and partnered gene therapies to treat patients with liver/metabolic, central nervous system and cardiovascular diseases.
Solid Biosciences carries a Zacks Rank #4 (Sell). You can see the complete list of today’s Zacks #1 Rank stocks here.
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