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Solid Biosciences' (SLDB) DMD Drug Gets Orphan Tag, Stock Up
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Solid Biosciences Inc. (SLDB - Free Report) announced that the FDA has granted the orphan drug designation to its next-generational investigational candidate, SGT-003, which is being developed as a gene therapy for Duchenne muscular dystrophy (DMD).
The FDA grants the orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 patients in the United States. The orphan drug designation will grant Solid Biosciences market exclusivity for SGT-003 in the treatment of DMD for a predefined time period, along with the exemption of FDA application fees and tax credits for qualified clinical studies, all subject to approval.
The company’s stock jumped 3.8% in the last trading session and 4.4% in the after-market hours on Tuesday as the investors cheered the encouraging regulatory update for the DMD candidate. Over the past year, shares of SLDB have gained 9.4% against the industry’s 12.2% decline.
Image Source: Zacks Investment Research
DMD is a progressive and degenerative disorder that leads to weakness and wasting of the body’s muscles. The candidate is being developed using SLDB’s proprietary and rationally designed capsid (AAV-SLB101).
The investigational gene therapy already enjoys the FDA’s fast track designation in the United States for the treatment of DMD.
We remind the investors that in November 2023, the FDA cleared Solid Biosciences’ investigational new drug (IND) application to begin clinical studies on SGT-003 to treat DMD. The IND clearance was based on encouraging data from a pre-clinical study of SGT-003.
The company is currently in the process of securing approvals from the institutional review boards (IRB) at the clinical study sites to begin the planned phase I/II study of SGT-003 for DMD. Patient screening for the early to mid-stage DMD will commence shortly after the IRB approvals are secured.
Following patient screening, Solid Biosciences anticipates beginning dosing in the phase I/II DMD study of SGT-003 in the mid-to-late first quarter of 2024.
Currently, Sarepta Therapeutics (SRPT - Free Report) 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 - Free Report) . 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.
In the past 30 days, the Zacks Consensus Estimate for Puma Biotech’s 2023 earnings per share (EPS) has increased from 72 cents to 73 cents. During the same time frame, the consensus estimate for Puma Biotech’s 2024 EPS has increased from 64 cents to 69 cents. Over the past three months, shares of PBYI have rallied 123%.
PBYI beat estimates in three of the last four quarters while missing on one occasion, delivering a four-quarter average earnings surprise of 76.55%.
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Solid Biosciences' (SLDB) DMD Drug Gets Orphan Tag, Stock Up
Solid Biosciences Inc. (SLDB - Free Report) announced that the FDA has granted the orphan drug designation to its next-generational investigational candidate, SGT-003, which is being developed as a gene therapy for Duchenne muscular dystrophy (DMD).
The FDA grants the orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 patients in the United States. The orphan drug designation will grant Solid Biosciences market exclusivity for SGT-003 in the treatment of DMD for a predefined time period, along with the exemption of FDA application fees and tax credits for qualified clinical studies, all subject to approval.
The company’s stock jumped 3.8% in the last trading session and 4.4% in the after-market hours on Tuesday as the investors cheered the encouraging regulatory update for the DMD candidate. Over the past year, shares of SLDB have gained 9.4% against the industry’s 12.2% decline.
Image Source: Zacks Investment Research
DMD is a progressive and degenerative disorder that leads to weakness and wasting of the body’s muscles. The candidate is being developed using SLDB’s proprietary and rationally designed capsid (AAV-SLB101).
The investigational gene therapy already enjoys the FDA’s fast track designation in the United States for the treatment of DMD.
We remind the investors that in November 2023, the FDA cleared Solid Biosciences’ investigational new drug (IND) application to begin clinical studies on SGT-003 to treat DMD. The IND clearance was based on encouraging data from a pre-clinical study of SGT-003.
The company is currently in the process of securing approvals from the institutional review boards (IRB) at the clinical study sites to begin the planned phase I/II study of SGT-003 for DMD. Patient screening for the early to mid-stage DMD will commence shortly after the IRB approvals are secured.
Following patient screening, Solid Biosciences anticipates beginning dosing in the phase I/II DMD study of SGT-003 in the mid-to-late first quarter of 2024.
Currently, Sarepta Therapeutics (SRPT - Free Report) 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 - Free Report) . 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.
Solid Biosciences Inc. Price and Consensus
Solid Biosciences Inc. price-consensus-chart | Solid Biosciences Inc. Quote
Zacks Rank and Other Stock to Consider
Solid Biosciences currently carries a Zacks Rank #2 (Buy).
Another top-ranked stock in the same industry is Puma Biotechnology, Inc. (PBYI - Free Report) , sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 30 days, the Zacks Consensus Estimate for Puma Biotech’s 2023 earnings per share (EPS) has increased from 72 cents to 73 cents. During the same time frame, the consensus estimate for Puma Biotech’s 2024 EPS has increased from 64 cents to 69 cents. Over the past three months, shares of PBYI have rallied 123%.
PBYI beat estimates in three of the last four quarters while missing on one occasion, delivering a four-quarter average earnings surprise of 76.55%.