Solid Biosciences Inc. SLDB announced that the FDA has granted the Fast Track designation to its next-generation gene therapy candidate, SGT-003, for the treatment of Duchenne muscular dystrophy (DMD).
SGT-003 is being developed using the company’s proprietary and rationally designed capsid (AAV-SLB101).
The FDA’s Fast Track designations intend to expedite development and review timelines when preliminary nonclinical and clinical evidence indicates that the drug candidate may demonstrate substantial improvement over available therapies to address unmet medical needs for serious or life-threatening conditions. The Fast Track tag enables close communication between the FDA and sponsor in improving the efficiency of product development to get new therapeutics to patients faster.
Solid Biosciences’ stock rallied 68.4% in the last trading session as the investors cheered the encouraging regulatory update for SGT-003. Year to date, shares of SLDB gained 7.1% against the industry’s 20.6% fall.
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We would like to remind the investors that in November 2023, the company received the FDA’s nod to begin clinical studies on SGT-003 for the DMD indication, after getting the clearance of its investigational new drug (IND) application.
The IND clearance was based on encouraging data from a pre-clinical study of SGT-003. In the pre-clinical study, SGT-003 demonstrated increased biodistribution to cardiac and skeletal muscle, including the diaphragm compared with AAV9 in nonhuman primates.
The planned phase I/II SGT-003-101 study will evaluate the safety and tolerability of SGT-003 in pediatric patients with DMD at a dose of 1E14vg/kg. The candidate will be administered as a one-time intravenous injection to patients in two cohorts with a minimum of three patients each. Solid Biosciences is also keeping a provision for potential cohort expansion in the study if deemed necessary.
Per the company, cohort 1 of the early-mid-stage study will evaluate SGT-003 in patients aged 4 to < 6 years of age with DMD. The long-term safety and efficacy of the candidate will be evaluated for a total of five years following treatment.
DMD is a progressive and degenerative disorder that leads to weakness and wasting away of the body’s muscles.
Solid Biosciences Inc. Price and Consensus
Currently, Sarepta Therapeutics SRPT markets the only approved one-shot gene therapy drug in the United States. The FDA granted accelerated approval to SRPT’s Elevidys (delandistrogene moxeparvovec or SRP-9001), an adeno-associated virus-based gene therapy, to treat ambulatory pediatric patients aged between four and five years with DMD in June 2023.
Elevidys has been developed by Sarepta in collaboration with Roche RHHBY. Both Sarepta and Roche entered into a licensing agreement in 2019 to jointly develop and commercialize Elevidys. Per the agreement, RHHBY has exclusive rights to launch and commercialize the gene therapy in ex-U.S. markets.
Sarepta’s Elevidys is yet to gain full approval to treat DMD patients of all ages in the United States.
PTC Therapeutics PTCT currently markets Emflaza (deflazacort) in the United States for the treatment of DMD in patients aged five years and older. Emflaza is a non-gene therapy product. It was developed by Marathon Therapeutics and received FDA approval in 2017 as the first drug in the United States for DMD, regardless of genetic mutation.
In 2017, PTC Therapeutics acquired Emflaza from Marathon for an upfront payment of $140 million. PTCT remains liable to make tiered royalty payments to Marathon based on the annual net sales of Emflaza beginning in 2018.
PTCT’s Emflaza is a corticosteroid that demonstrates anti-inflammatory and immunosuppressant effects.
Solid Biosciences currently has a Zacks Rank #3 (Hold).
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