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Sarepta (SRPT) Down 11% on FDA Extending DMD Gene Therapy Review
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Shares of Sarepta (SRPT - Free Report) were down 10.6% on May 24, 2023 after management announced multiple updates on the company’s biologics license application (“BLA”) seeking accelerated approval for SRP-9001 to treat Duchenne muscular dystrophy (“DMD”). The gene therapy has been developed in collaboration with Roche (RHHBY - Free Report) .
Sarepta confirmed that based on discussions, the FDA intends to grant accelerated approval to SRP-9001 for use in Duchenne patients aged between four and five years. The agency stated that it will grant a non-age restricted expansion to SRP-9001 provided the phase III EMBARK study achieves its objectives. The EMBARK study is the proposed confirmatory study which will seek full approval for SRP-9001 in DMD indication. Top-line results from the EMBARK study is expected in fourth-quarter 2023.
The FDA also informed Sarepta that it requires modest additional time to complete the BLA review, including final label negotiations and post-marketing commitment discussions. As a result, the agency’s final decision on the BLA is now expected before Jun 22, 2023. The agency had previously planned to complete the SRP-9001 review by May 29.
This extension in review period by the FDA comes days after the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (“CTGTAC”) voted 8:6, narrowly recommending approval of the SRP-9001 BLA. This narrow voting was likely due to the FDA’s concerns on SRP-9001. Per the agency’s briefing documents issued earlier this month, the clinical studies conducted to date do not provide unambiguous evidence that SRP-9001 will benefit DMD patients.
Last month, a news article issued by a third-party claimed that the FDA was initially against approving Sarepta’s SRP-9001 BLA. Per this article, some of the FDA’s staff members reached a “non-binding conclusion” that SRP-9001 BLA should be rejected. However, the intervention of a top agency official Peter Marks, who also happens to be an advocate for faster gene-therapy approvals, shifted the FDA’s path toward discussing the BLA at the CTGTAC meeting. Per the FDA website, Marks is the current director of the FDA’s Center for Biologics Evaluation and Research (“CBER”).
In the year so far, Sarepta’s shares have moved up 1.4% against the industry’s 6.3% fall.
Image Source: Zacks Investment Research
The BLA is supported by data from multiple studies in a clinical development program evaluating SRP-9001 in DMD. Last year, Sarepta and Roche announced new functional data across these studies, demonstrating that treatment with SRP-9001 led to functional improvements in individuals suffering from DMD compared with a propensity-weighted external control group multiple time. The time points vary from one, two and four years post-treatment.
SRP-9001 has been developed by Sarepta in partnership with Roche, as part of a licensing agreement entered in 2019. Per the agreement, Roche has exclusive rights to launch and commercialize SRP-9001 in ex-U.S. markets.
Sarepta’s commercial portfolio consists of three RNA-based PMO therapies targeting DMD — Exondys 51, Vyondys 53 and Amondys 45. These drugs can potentially address nearly a third of all patients with DMD in the United States.
Other than SRP-9001, Sarepta is also developing SRP-5051 (vesleteplirsen), its next-generation exon-skipping pipeline candidate for treating DMD patients with skipping exon 51. This February, management also started an early-stage study (VOYAGENE) evaluating its other gene therapy candidate, SRP-9003, in patients with Limb-girdle muscular dystrophy (LGMD) type 2E/R4.
Sarepta currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the overall healthcare sector include ANI Pharmaceuticals (ANIP - Free Report) and Allogene Therapeutics (ALLO - Free Report) . While ANI Pharmaceuticals sports a Zacks Rank #1 (Strong Buy), Allogene carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 30 days, estimates for ANI Pharmaceuticals’ 2023 earnings per share have increased from $2.42 to $3.31. During the same period, the earnings estimates per share for 2024 have risen from $3.76 to $4.32. Shares of ANI Pharmaceuticals are up 16.0% in the year-to-date period.
Earnings of ANI Pharmaceuticals beat estimates in each of the last four quarters, delivering an average earnings surprise of 68.64%. In the last reported quarter, ANI Pharmaceuticals’ earnings beat estimates by 244.12%.
In the past 30 days, estimates for Allogene Therapeutics’ 2023 loss per share have improved from $2.44 to $2.32. During the same period, the loss estimates per share for 2024 have narrowed from $2.46 to $2.21. Shares of Allogene Therapeuticshave declined 9.5% in the year-to-date period.
Earnings of Allogene Therapeutics beat estimates in three of the last four quarters while missing the mark on one occasion. On average, the company’s earnings witnessed a surprise of 5.08%. In the last reported quarter, Allogene Therapeutics’ earnings beat estimates by 7.94%.
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Sarepta (SRPT) Down 11% on FDA Extending DMD Gene Therapy Review
Shares of Sarepta (SRPT - Free Report) were down 10.6% on May 24, 2023 after management announced multiple updates on the company’s biologics license application (“BLA”) seeking accelerated approval for SRP-9001 to treat Duchenne muscular dystrophy (“DMD”). The gene therapy has been developed in collaboration with Roche (RHHBY - Free Report) .
Sarepta confirmed that based on discussions, the FDA intends to grant accelerated approval to SRP-9001 for use in Duchenne patients aged between four and five years. The agency stated that it will grant a non-age restricted expansion to SRP-9001 provided the phase III EMBARK study achieves its objectives. The EMBARK study is the proposed confirmatory study which will seek full approval for SRP-9001 in DMD indication. Top-line results from the EMBARK study is expected in fourth-quarter 2023.
The FDA also informed Sarepta that it requires modest additional time to complete the BLA review, including final label negotiations and post-marketing commitment discussions. As a result, the agency’s final decision on the BLA is now expected before Jun 22, 2023. The agency had previously planned to complete the SRP-9001 review by May 29.
This extension in review period by the FDA comes days after the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (“CTGTAC”) voted 8:6, narrowly recommending approval of the SRP-9001 BLA. This narrow voting was likely due to the FDA’s concerns on SRP-9001. Per the agency’s briefing documents issued earlier this month, the clinical studies conducted to date do not provide unambiguous evidence that SRP-9001 will benefit DMD patients.
Last month, a news article issued by a third-party claimed that the FDA was initially against approving Sarepta’s SRP-9001 BLA. Per this article, some of the FDA’s staff members reached a “non-binding conclusion” that SRP-9001 BLA should be rejected. However, the intervention of a top agency official Peter Marks, who also happens to be an advocate for faster gene-therapy approvals, shifted the FDA’s path toward discussing the BLA at the CTGTAC meeting. Per the FDA website, Marks is the current director of the FDA’s Center for Biologics Evaluation and Research (“CBER”).
In the year so far, Sarepta’s shares have moved up 1.4% against the industry’s 6.3% fall.
Image Source: Zacks Investment Research
The BLA is supported by data from multiple studies in a clinical development program evaluating SRP-9001 in DMD. Last year, Sarepta and Roche announced new functional data across these studies, demonstrating that treatment with SRP-9001 led to functional improvements in individuals suffering from DMD compared with a propensity-weighted external control group multiple time. The time points vary from one, two and four years post-treatment.
SRP-9001 has been developed by Sarepta in partnership with Roche, as part of a licensing agreement entered in 2019. Per the agreement, Roche has exclusive rights to launch and commercialize SRP-9001 in ex-U.S. markets.
Sarepta’s commercial portfolio consists of three RNA-based PMO therapies targeting DMD — Exondys 51, Vyondys 53 and Amondys 45. These drugs can potentially address nearly a third of all patients with DMD in the United States.
Other than SRP-9001, Sarepta is also developing SRP-5051 (vesleteplirsen), its next-generation exon-skipping pipeline candidate for treating DMD patients with skipping exon 51. This February, management also started an early-stage study (VOYAGENE) evaluating its other gene therapy candidate, SRP-9003, in patients with Limb-girdle muscular dystrophy (LGMD) type 2E/R4.
Sarepta Therapeutics, Inc. Price
Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote
Zacks Rank & Stocks to Consider
Sarepta currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the overall healthcare sector include ANI Pharmaceuticals (ANIP - Free Report) and Allogene Therapeutics (ALLO - Free Report) . While ANI Pharmaceuticals sports a Zacks Rank #1 (Strong Buy), Allogene carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 30 days, estimates for ANI Pharmaceuticals’ 2023 earnings per share have increased from $2.42 to $3.31. During the same period, the earnings estimates per share for 2024 have risen from $3.76 to $4.32. Shares of ANI Pharmaceuticals are up 16.0% in the year-to-date period.
Earnings of ANI Pharmaceuticals beat estimates in each of the last four quarters, delivering an average earnings surprise of 68.64%. In the last reported quarter, ANI Pharmaceuticals’ earnings beat estimates by 244.12%.
In the past 30 days, estimates for Allogene Therapeutics’ 2023 loss per share have improved from $2.44 to $2.32. During the same period, the loss estimates per share for 2024 have narrowed from $2.46 to $2.21. Shares of Allogene Therapeuticshave declined 9.5% in the year-to-date period.
Earnings of Allogene Therapeutics beat estimates in three of the last four quarters while missing the mark on one occasion. On average, the company’s earnings witnessed a surprise of 5.08%. In the last reported quarter, Allogene Therapeutics’ earnings beat estimates by 7.94%.