Whats going on here?

Sarepta Therapeutics' shares surged around 36% in premarket trading after the FDA expanded its approval of the gene therapy Elevidys.

What does this mean?

Sarepta received full FDA approval for Elevidys for ambulatory patients aged four and above and accelerated approval for those who cannot walk. This move significantly boosts the therapys market reach, with projections anticipating sales to hit $3 billion by 2025 and peak at $5 billion by 2027. In 2023, Elevidys generated $200.4 million in revenue, with expectations set to soar to $991.91 million this year. Given that Duchenne Muscular Dystrophy (DMD) affects roughly one in 3,500 male births worldwide, Elevidys' expanded approval now targets about 13,000 US patients covering nearly 90% of the US DMD market.

Why should I care?

For markets: The gene therapy gold rush.

In the evolving landscape of gene therapies for DMD, Sarepta's Elevidys is positioned to be the dominant player with no substantial competition until post-2027. RegenxBio is exploring early to mid-stage trials for a competing therapy, while Pfizer faced a setback with a recent late-stage trial failure. This dominance translates into significant market stability for Sarepta, backed by robust financial forecasts.

The bigger picture: The horizon looks bright.

With the global DMD treatment market projected to grow to $11.47 billion by 2034, the expanded use of Elevidys places Sarepta in a commanding position. The company's investor call scheduled for 08:30 am ET is expected to shed light on strategic plans moving forward. At $3.2 million per treatment, Elevidys might be one of the priciest therapies globally, but its value proposition and market penetration could redefine the treatment landscape for DMD.

Originally posted here:
Sarepta Therapeutics' Gene Therapy Gets Major FDA Approval - Finimize

The US Food and Drug Administration (FDA) has granted expanded approval to Sarepta Therapeutics Duchenne muscular dystrophy (DMD) gene therapy to treat patients aged four years and older with a confirmed mutation in the DMD gene.

Elevidys (delandistrogene moxeparvovec-rokl) was first authorised under the US regulators accelerated approval pathway in June last year for use in ambulatory patients aged between four and five years.

The one-time treatment has now been granted traditional approval to treat ambulatory patients aged four years and over, and accelerated approval to treat non-ambulatory patients in the same age group.

Estimated to affect one in every 3,500 male births worldwide, DMD is a severe genetic condition caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles.

Symptoms are usually first seen in early childhood and worsen over time, affecting patients ability to walk as well as their heart and respiratory muscles.

Administered as a single intravenous dose, Sareptas therapy is designed to address the underlying cause of DMD by delivering a gene into the body that leads to the production of Elevidys micro-dystrophin, a shortened protein that contains selected domains of the dystrophin protein present in normal muscle cells.

The FDAs latest decision on the therapy was supported by results from two double-blind, placebo-controlled studies and two open-label studies, which enrolled a total of 218 male patients with a confirmed disease-causing mutation in the DMD gene.

Doug Ingram, Sareptas president and chief executive officer, described the label expansion as a defining moment for the Duchenne community.

Sharing a similar sentiment, Jerry Mendell, co-inventor of Elevidys and senior advisor of medical affairs at Sarepta, said: The initial approval of Elevidys was a significant milestone, and the expanded indication means clinicians now have a treatment option for the great majority of boys and young men living with Duchenne.

This expansion speaks to the success of the science, the evidence and the improvements in the trajectory of the disease we have seen to date across studies.

Sarepta is responsible for regulatory approval, commercialisation and the manufacturing of Elevidys in the US, while Roche is responsible for regulatory approvals and bringing the therapy to patients across the rest of the world.

See the article here:
FDA expands indication for Sareptas Duchenne muscular dystrophy gene therapy Elevidys - PMLiVE

Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camdens rare, inherited immune deficiency. But its not available to him.

In 2022, London-based Orchard Therapeutics stopped investing in an experimental treatment for the condition, Wiskott-Aldrich syndrome. And there are no gene therapy studies he can join.

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Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate - goskagit.com

Pictured: A boy in a wheelchair over a muscle texture background/Taylor Tieden forBioSpace

In a much-anticipated regulatory decision on Thursday, the FDA significantly expanded the label and use of Elevidys, Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy (DMD). Most exciting, perhaps, is the expansion of Elevidys label to include older individuals as well as non-ambulatory ones, broadening the spectrum of patients eligible for this breakthrough therapy. While Elevidys was previously approved under accelerated approval for ambulatory individuals 4 through 5 years with a confirmed mutation in the DMD gene, Thursday's expanded approval includes ambulatory and non-ambulatory patients 4 years of age and older with a confirmed mutation in the DMD gene.

It was a year ago that the regulator granted accelerated approval for Elevidys as the first FDA-approved gene therapy for DMDdespite falling short of its primary efficacy endpoint in two studies. In making its decision, the FDA on Thursday said it considered the totality of the evidence, including the potential risks associated with the product, the life-threatening and debilitating nature of the disease and the urgent unmet medical need.

Under its previous label, Elevidys was limited to only about 3% of the total DMD population, according to Sarepta CEO Doug Ingram. However, BMO Capital Markets analyst Kostas Biliouris, in a note to investors, said that with Thursdays approval, the FDA has effectively opened Elevidys label to more than 90% of patients in the U.S.

Elevidys is now approved to treat a population that includes ~13,000 DMD patients (~90% of total prevalence), creating a significant commercial opportunity for [Sarepta], Biliouris wrote.

William Blair analyst Tim Lugo agreed, writing in an investor note that Thursdays FDA approval is the best-case scenario for Sarepta since many patients below age 4 are not yet diagnosed but will age into the label, opening the DMD market to all current and future patients who will be eligible for treatment. Lugo also highlighted the fact that Sarepta has an ongoing Phase II study for boys under the age of 4.

Its also worth mentioning that Elevidysdeveloped and commercialized in partnership with Rochehas a $3.2 million per patient price tag, making it one of the worlds most expensive medicines. Data analysis firm GlobalData said it expects Sarepta to pocket $5.7 billion in Elevidys sales by 2029.

Biliouris said in his investors note that Sarepta will have a monopoly in DMD for the next ~4+years. That 4+ years of monopoly is thanks in part to the stumble by Pfizers experimental DMD gene therapy, which was found not to improve motor function versus placebo in a Phase III trial last week.

Indeed, there is only one other company with a DMD candidate in Phase III: Capricor Therapeutics cell therapy CAP-1002. The company expects topline data from the ongoing trial by the end of this year. Other DMD treatments are all in earlier stages of development.

Jeff Chamberlain, president of the American Society for Gene and Cell Therapy and the McCaw Chair in Muscular Dystrophy at the University of Washington School of Medicine, told BioSpace last month that Sarepta has done a fantastic job on Elevidys, with some children treated with the gene therapy doing tremendously well, while others he said are not doing quite as well.

In the coming years, it is critical to overcome the challenges with the first generation of micro-dystrophin gene therapy products, according to Chamberlain. Just because we have an approved therapy doesnt mean you can stop and go home, Chamberlain said. We want something thats going to be more effective; thats going to work on all patients, not just some of them.

Greg Slabodkin is the news editor at BioSpace. You can reach him atgreg.slabodkin@biospace.com. Follow him onLinkedIn.

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Expanded Approval of Sareptas Elevidys Is Progress, But More Needed for DMD Patients - BioSpace

Shares of Sarepta (SRPT Quick QuoteSRPT - Free Report) rose nearly 34% in after-market trading on Thursday after it announced that the FDA approved the expanded use of its Duchenne muscular dystrophy (DMD) gene therapy Elevidys.

Elevidys is now approved to treat all DMD patients aged four years and older. While the FDA granted traditional approval for the therapy to treat ambulatory DMD patients (those who can still walk), it has granted accelerated approval for non-ambulatory patients.

Sareptas gene therapy was initially approved by the FDA last year under the accelerated pathway to treat ambulatory pediatric patients aged between four and five years with DMD. Following the FDA nod, Elevidys was the first-ever approved gene therapy for DMD.

The label expansion is mainly supported by data from the phase III EMBARK study, announced last October. Though the study failed to achieve its primary endpoint, it achieved statistical significance on all pre-specified key secondary endpoints, indicating that treatment with Elevidys modifies the course of DMD indication.

Management will still need to conduct a confirmatory study to convert the accelerated approval for non-ambulatory DMD patients to a full one. Sarepta is currently conducting the phase III ENVISION study to evaluate the safety and efficacy of gene therapy in non-ambulatory and ambulatory DMD patients. This study also satisfies the regulatory requirements for Elevidys approval outside the United States.

Elevidys is the only one-shot gene therapy for DMD in the United States. A progressive and degenerative disorder, DMD leads to weakness and wasting away of the bodys muscles. Despite being initially approved with a confined label, Sarepta recorded revenues of more than $200 million from Elevidys sales last year, which is an encouraging figure for a therapy that was commercially launched in the second half of 2023. This uptick in share price, following the label expansion announcement, is likely attributed to Elevidys blockbuster potential.

Year to date, Sareptas shares have inched up 28.1% against the industrys 7.9% fall.

Image Source: Zacks Investment Research

Sarepta developed Elevidys in collaboration with Roche (RHHBY Quick QuoteRHHBY - Free Report) . Sarepta and Roche entered into a licensing agreement in 2019 to develop and commercialize Elevidys. Per the agreement, Sarepta is responsible for marketing the therapy in the United States, while Roche is responsible for marketing the gene therapy outside the country. Sarepta is also eligible to receive collaboration revenues on the ex-U.S. sales made by Roche.

Sarepta is currently the market leader in DMD treatment. Apart from Elevidys, the company has three other therapies in its commercial portfolio targeting the DMD patient population, namely Exondys 51, Vyondys 53 and Amondys 45. Per management, these three drugs have the potential to address nearly a third of all patients with DMD in the United States.

However, competition in the DMD space is stiff as companies like Regenxbio (RGNX Quick QuoteRGNX - Free Report) and Solid Biosciences (SLDB Quick QuoteSLDB - Free Report) have been developing their respective gene therapy candidates for DMD.

Regenxbio is planning to hold an end-of-phase II (EOP2) meeting with the FDA early in the next quarter to discuss the study design of a pivotal late-stage study on RGX-202, its DMD gene therapy candidate. Based on the feedback, Regenxbio expects to start this pivotal study before 2024-end. If the outcome of this study is positive, it plans to seek accelerated approval for its therapy from the FDA.

Last year, the FDA cleared Solid Biosciences investigational new drug (IND) to start a phase I/II study on SGT-003 in pediatric patients with DMD. This study is expected to begin by the end of this month. SLDB expects to report initial data from this study by this years end. The FDA has granted fast-track, orphan drug and rare pediatric disease designations to Solid Biosciences gene therapy in DMD indication.

However, companies also have suffered major setbacks in the DMD space. Earlier this month, Pfizer (PFE Quick QuotePFE - Free Report) announced that a late-stage study evaluating its DMD gene therapy candidate failed to meet the primary endpoint and key secondary endpoints. Treatment with the Pfizer therapy failed to improve motor function among the DMD boys. Pfizer plans to share more detailed results from the study at an upcoming medical meeting.

Sarepta sports a Zacks Rank #3 (Hold). You can see the complete list of todays Zacks #1 Rank (Strong Buy) stocks here.

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FDA Expands Sarepta's (SRPT) DMD Gene Therapy Label - Zacks Investment Research

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Gene Therapy-Rare Disease | National | wataugademocrat.com - Watauga Democrat

Ascidian to receive $42 million in initial payment, and up to $1.8 billion in research, clinical and commercial milestone payments, as well as commercial royalties

Per-target agreement enables Ascidian to pursue additional internal and collaborative programs within neurology and other therapeutic areas

Combines RNA Exon Editors with next generation CNS delivery capabilities of Roche to develop novel medicines for difficult to treat neurological diseases

BOSTON, June 18, 2024 /PRNewswire/ --Ascidian Therapeutics, a biotechnology company seeking to treat human diseases by rewriting RNA, today announced a research collaboration and licensing agreement with Roche (SIX: RO, ROG;OTCQX: RHHBY) for the discovery and development of RNA exon editing therapeutics targeting neurological diseases.

Ascidian's RNA exon editing platform is designed to advance the therapeutic possibilities of RNA medicine and treat diseases not addressed by today's gene editing technologies. The company designs and develops RNA exon editing therapeutics that edit RNA exons at the kilobase scale.

Under the agreement, Ascidian will provide Roche exclusive, target-specific rights to Ascidian's RNA exon editing technology for undisclosed neurological targets. Ascidian will conduct discovery and certain preclinical activities in collaboration with Roche, and Roche will be responsible for certain preclinical activities, and further clinical development, manufacturing, and commercialization. Ascidian will receive an initial payment of $42 million and is eligible to receive up to $1.8 billion in research, clinical, and commercial milestone payments, as well as royalties on commercial sales worldwide. Based on the terms of the agreement, Ascidian is free to develop programs against other neurological targets internally or with other collaborators.

"Roche is known and respected worldwide for their expertise in complex neurological diseases, and I am proud of the scientific rigor and quality of the work done at Ascidian that has led to this partnership," saidMichael Ehlers,M.D., Ph.D., President and Chief Executive Officer of Ascidian Therapeutics. "The potential of treating disease by large-scale exon editing of RNA is vast. We look forward to working with the Roche team to develop first-in-class RNA exon editing medicines for multiple neurological diseases, with a mission and passion to relieve suffering and improve lives."

"Our partnership with Ascidian is an opportunity to harness advanced RNA exon editing technology, which has the potential to deliver transformative one-time therapeutics by editing multiple whole exons at the RNA level with a single treatment," said James Sabry, M.D., Ph.D., Global Head of Pharma Partnering at Roche.

Ascidian's platform enables targeting of large genes and genes with high mutational variance while maintaining native gene expression patterns and levels. By rewriting RNA, Ascidian's exon editing technology is designed to provide the durability of gene therapy, while sharply reducing risks associated with direct DNA editing and gene replacement.

About Ascidian Therapeutics

Ascidian Therapeutics, an ATP company, is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With discovery, preclinical, and clinical programs in retinal, neurological, neuromuscular, and genetically defined diseases, Ascidian's approach has the potential to treat patients with one dose of an RNA exon editor, opening new therapeutic possibilities for patients and their families who are seeking breakthroughs. Earlier this year, Ascidian announced U.S. FDA IND clearance for the first-ever RNA exon editing candidate, ACDN-01, which targets Stargardt disease and other ABCA4 retinopathies. Ascidian is currently executing the Phase 1/2 STELLAR clinical trial to evaluate the safety and efficacy of ACDN-01. For more information, visit http://www.ascidian.com.

SOURCE Ascidian Therapeutics

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Ascidian Therapeutics Enters Collaboration with Roche for Discovery and Development of RNA Exon Editing ... - PR Newswire