Last update 27 Jun 2024
Deramiocel
Last update 27 Jun 2024
Overview
Basic Info
Drug Type Stem cell therapy |
Synonyms Allogeneic adult stem cell therapy for myocardial infarction(Capricor), Allogeneic cardiosphere-derived cells(Capricor), Allogeneic CDCs - Capricor + [4] |
Target- |
Mechanism Cell replacements |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization |
Drug Highest PhasePhase 3 |
First Approval Date- |
RegulationOrphan Drug (US), Regenerative Medicine Advanced Therapy (US) |
Related
10
Clinical Trials associated with DeramiocelA Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.
Start Date22 Jun 2022 |
Sponsor / Collaborator |
A Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Intravenous Infusion of CAP-1002 in Patients With COVID-19 (INSPIRE)
This is a randomized, double-blind, placebo-controlled, Pilot, Phase 2 Exploratory study that will enroll subjects with a clinical diagnosis of COVID-19 confirmed by laboratory testing and who are in severe or critical condition as indicated by life-support measures.
Start Date15 Nov 2020 |
Sponsor / Collaborator |
Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial
This Phase 2, multi-center, open-label extension trial will provide CAP-1002 to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up. The trial will explore the safety and efficacy of sixteen intravenous administrations of CAP-1002, each separated by three months. Subjects will undergo a targeted screening during a 30-day screening period, eligible subjects will then undergo baseline safety and efficacy assessments on Day 1 prior to their first infusion of CAP-1002.
Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45 and 48. Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42 and 45 trial visits, unless otherwise indicated.
All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42 and 45. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.
Subjects will complete trial assessments at Screening; Day 1; Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45 and 48. Safety and efficacy assessments will be conducted prior to CAP-1002 administration at the Day 1, Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42 and 45 trial visits, unless otherwise indicated.
All CAP-1002 infusions will be conducted in an outpatient setting at the investigative site on Day 1 and Months 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42 and 45. Subjects will be observed in the outpatient setting for at least two hours post infusion and then discharged the same day, if medically cleared by the site Investigator.
Start Date20 Jul 2020 |
Sponsor / Collaborator |
100 Clinical Results associated with Deramiocel
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100 Translational Medicine associated with Deramiocel
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100 Patents (Medical) associated with Deramiocel
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5
Literatures (Medical) associated with Deramiocel02 Jan 2023·Expert opinion on biological therapy
Biological and genetic therapies for the treatment of Duchenne muscular dystrophy
Review
Author: Wilton-Clark, Harry ; Yokota, Toshifumi
INTRODUCTION:
Duchenne muscular dystrophy is a lethal genetic disease which currently has no cure, and poor standard treatment options largely focused on symptom relief. The development of multiple biological and genetic therapies is underway across various stages of clinical progress which could markedly affect how DMD patients are treated in the future.
AREAS COVERED:
The purpose of this review is to provide an introduction to the different therapeutic modalities currently being studied, as well as a brief description of their progress to date and relative advantages and disadvantages for the treatment of DMD. This review discusses exon skipping therapy, microdystrophin therapy, stop codon readthrough therapy, CRISPR-based gene editing, cell-based therapy, and utrophin upregulation. Secondary therapies addressing nonspecific symptoms of DMD were excluded.
EXPERT OPINION:
Despite the vast potential held by gene replacement therapy options such as microdystrophin production and utrophin upregulation, safety risks inherent to the adeno-associated virus delivery vector might hamper the clinical viability of these approaches until further improvements can be made. Of the mutation-specific therapies, exon skipping therapy remains the most extensively validated and explored option, and the cell-based CAP-1002 therapy may prove to be a suitable adjunct therapy filling the urgent need for cardiac-specific therapies.
01 Mar 2022·Lancet (London, England)Q1 · MEDICINE
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial
Q1 · MEDICINE
Article
Author: Collado, Jorge ; Johnson, Hannah ; Harmelink, Matthew M ; Connolly, Anne ; Muntoni, Francesco ; Furlong, Pat ; Anderson, Kristan ; Newton, Andrew ; Abedi, Mehrdad ; Dayan, Jonathan G. ; Pyzik, Erika ; Ruckdeschel Smith, Rachel ; Marbán, Linda ; Williams, Paula ; Hor, Kan N ; Varadhachary, Arun S ; Ascheim, Deborah D ; Jhawar, Sanjay ; Prasad, Poonam ; Chouteau, Wendy ; Finkel, Richard S ; Hendrix, Suzanne ; Kusmik, Kyle ; Janas, Joanne ; Wells, Julie ; Sarwary, Omaid ; Eagle, Michelle ; Brown, Michelle ; Surampudi, Prasanth ; Van Eyk, Jennifer ; Joyce, Nanette C. ; Giordano, Anthony F. ; Filar, Lauri ; Goude, Erica ; Rybalsky, Irina ; Butterfield, Russell ; McDonald, Craig M ; Anthonisen, Colleen ; Taylor, Michael D ; Rehborg, Rebecca ; Civitello, Matthew ; Tian, Cuixia ; Wezel, Germaine ; Duke, Julie ; Baek, Jayoon ; Marbán, Eduardo ; Henricson, Erik K ; Rogers, Russell G. ; Edmondson, Angie ; Vega, Melisa ; Nicorici, Alina ; Rodriguez, Aixa ; Mayer, Oscar H ; Evans, Maya ; Hogan, Nathaniel ; Rogy, Siegfried
BACKGROUND:
Cardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac muscle deterioration in experimental models of Duchenne muscular dystrophy. The HOPE-2 trial examined the safety and efficacy of sequential intravenous infusions of human allogeneic CDCs in late-stage Duchenne muscular dystrophy.
METHODS:
In this multicentre, randomised, double-blind, placebo-controlled, phase 2 trial, patients with Duchenne muscular dystrophy, aged 10 years or older with moderate upper limb impairment, were enrolled at seven centres in the USA. Patients were randomly assigned (1:1) using stratified permuted blocks to receive CAP-1002 (1·5 × 108 CDCs) or placebo intravenously every 3 months for a total of four infusions. Clinicians, caregivers, patients, and clinical operations personnel were fully masked to treatment groups. The primary outcome was the change in mid-level elbow Performance of Upper Limb version 1.2 (PUL 1.2) score at 12 months, assessed in the intention-to-treat population. Safety was assessed in all individuals who received an investigational product. This trial is registered with ClinicalTrials.gov, NCT03406780.
FINDINGS:
Between March 1, 2018, and March 31, 2020, 26 male patients with Duchenne muscular dystrophy were enrolled, of whom eight were randomly assigned to the CAP-1002 group and 12 to the placebo group (six were not randomised due to screening failure). In patients who had a post-treatment PUL 1.2 assessment (eight in the CAP-1002 group and 11 in the placebo group), the mean 12-month change from baseline in mid-level elbow PUL1.2 favoured CAP-1002 over placebo (percentile difference 36·2, 95% CI 12·7-59·7; difference of 2·6 points; p=0·014). Infusion-related hypersensitivity reactions without long-term sequelae were observed in three patients, with one patient discontinuing therapy due to a severe allergic reaction. No other major adverse reactions were noted, and no deaths occurred.
INTERPRETATION:
CAP-1002 cell therapy appears to be safe and effective in reducing deterioration of upper limb function in patients with late-stage Duchenne muscular dystrophy. Various measures of cardiac function and structure were also improved in the CAP-1002 group compared with the placebo group. Longer-term extension studies are needed to confirm the therapeutic durability and safety of CAP-1002 beyond 12 months for the treatment of skeletal myopathy and cardiomyopathy in Duchenne muscular dystrophy.
FUNDING:
Capricor Therapeutics.
01 Jul 2020·Basic research in cardiology
Allogeneic cardiosphere-derived cells (CAP-1002) in critically ill COVID-19 patients: compassionate-use case series
Article
Author: Friedman, Oren ; Chen, Peter ; Marbán, Linda ; Singh, Siddharth ; Akhmerov, Akbarshakh ; Zaman, Tanzira ; Falk, Jeremy ; Ebinger, Joseph E ; Marbán, Eduardo ; Gheorghiu, Mitch ; Makkar, Raj R ; Chakravarty, Tarun
Abstract:
There are no definitive therapies for patients with severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) infection. Therefore, new therapeutic strategies are needed to improve clinical outcomes, particularly in patients with severe disease. This case series explores the safety and effectiveness of intravenous allogeneic cardiosphere-derived cells (CDCs), formulated as CAP-1002, in critically ill patients with confirmed coronavirus disease 2019 (COVID-19). Adverse reactions to CAP-1002, clinical status on the World Health Organization (WHO) ordinal scale, and changes in pro-inflammatory biomarkers and leukocyte counts were analyzed. All patients (n = 6; age range 19–75 years, 1 female) required ventilatory support (invasive mechanical ventilation, n = 5) with PaO2/FiO2 ranging from 69 to 198. No adverse events related to CAP-1002 administration were observed. Four patients (67%) were weaned from respiratory support and discharged from the hospital. One patient remains mechanically ventilated as of April 28th, 2020; all survive. A contemporaneous control group of critically ill COVID-19 patients (n = 34) at our institution showed 18% overall mortality at a similar stage of hospitalization. Ferritin was elevated in all patients at baseline (range of all patients 605.43–2991.52 ng/ml) and decreased in 5/6 patients (range of all patients 252.89–1029.90 ng/ml). Absolute lymphocyte counts were low in 5/6 patients at baseline (range 0.26–0.82 × 103/µl) but had increased in three of these five patients at last follow-up (range 0.23–1.02 × 103/µl). In this series of six critically ill COVID-19 patients, intravenous infusion of CAP-1002 was well tolerated and associated with resolution of critical illness in 4 patients. This series demonstrates the apparent safety of CAP-1002 in COVID-19. While this initial experience is promising, efficacy will need to be further assessed in a randomized controlled trial.
74
News (Medical) associated with Deramiocel25 Jun 2024
SAN DIEGO, June 25, 2024 (GLOBE NEWSWIRE) -- In a release issued under the headline "Capricor Therapeutics Announces for the Treatment of Duchenne Muscular Dystrophy" on Tuesday, June 25th by Capricor Therapeutics, please note that the words "Pre-BLA Meeting with FDA for Deramiocel" were omitted from the headline. The corrected release follows. Capricor Therapeutics Announces Pre-BLA Meeting with FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD). Capricor’s goal for this meeting will be to finalize its BLA filing plans based on all currently available data as well as to work with the FDA to outline the rolling BLA submission timeline. “We continue to move rapidly towards potential approval of deramiocel for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “We now have formally scheduled our Pre-BLA meeting with the FDA, which will finalize our BLA filing plans and discuss available options to expedite the filing of our BLA. We recognize the FDA’s willingness to bring impactful therapies to market as quickly as possible due to the enormous unmet needs of patients with DMD. While we are delighted with the approval of gene therapy for DMD, we believe that it will take multiple therapies to combat DMD effectively. Based on the need to address the secondary consequences of DMD, we believe deramiocel can serve as a potential anchor therapy for DMD patients.” Additionally, Capricor will present its latest update of the HOPE-2 open label extension (OLE) 36-month data, both skeletal and cardiac, at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference being held June 27-29, 2024. About Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000 to 20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. Treatment options are limited and there is no cure. About Capricor Therapeutics Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter. Cautionary Note Regarding Forward-Looking Statements Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, as filed with the Securities and Exchange Commission on May 14, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements. Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation. For more information, please contact: Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com 858.727.1755
Phase 3Cell TherapyGene Therapy
25 Jun 2024
SAN DIEGO, June 25, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD). Capricor’s goal for this meeting will be to finalize its BLA filing plans based on all currently available data as well as to work with the FDA to outline the rolling BLA submission timeline.
“We continue to move rapidly towards potential approval of deramiocel for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “We now have formally scheduled our Pre-BLA meeting with the FDA, which will finalize our BLA filing plans and discuss available options to expedite the filing of our BLA. We recognize the FDA’s willingness to bring impactful therapies to market as quickly as possible due to the enormous unmet needs of patients with DMD. While we are delighted with the approval of gene therapy for DMD, we believe that it will take multiple therapies to combat DMD effectively. Based on the need to address the secondary consequences of DMD, we believe deramiocel can serve as a potential anchor therapy for DMD patients.”
Additionally, Capricor will present its latest update of the HOPE-2 open label extension (OLE) 36-month data, both skeletal and cardiac, at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference being held June 27-29, 2024.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000 to 20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. Treatment options are limited and there is no cure.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, as filed with the Securities and Exchange Commission on May 14, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.
Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation.
For more information, please contact:
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
Phase 3Cell TherapyGene Therapy
21 Jun 2024
Pictured: A boy in a wheelchair over a muscle texture background/Taylor Tieden for BioSpace
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 DMD—despite 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 Thursday’s 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 Thursday’s 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.
It’s also worth mentioning that Elevidys—developed and commercialized in partnership with Roche—has a $3.2 million per patient price tag, making it one of the world’s most expensive medicines. Data analysis firm GlobalData said it expects Sarepta to pocket $5.7 billion in Elevidys sales by 2029.
Sarepta’s Monopoly in DMD
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 Pfizer’s 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 doesn’t mean you can stop and go home,” Chamberlain said. “We want something that’s going to be more effective; that’s going to work on all patients, not just some of them.”
Greg Slabodkin is the news editor at BioSpace. You can reach him at greg.slabodkin@biospace.com. Follow him on LinkedIn.
Phase 2Phase 3Gene TherapyDrug Approval
100 Deals associated with Deramiocel
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R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Muscular Dystrophy, Duchenne | Phase 3 | US | 22 Jun 2022 | |
| COVID-19 | Phase 2 | US | 01 Nov 2020 | |
| Heart Failure | Phase 2 | US | 11 Sep 2016 | |
| Cardiomyopathies | Phase 2 | US | 19 Jul 2015 | |
| Myocardial Infarction | Phase 2 | US | 20 Apr 2014 | |
| Muscular Dystrophies | Discovery | US | - | |
| Muscular Dystrophies | Discovery | US | - |
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Clinical Result
Clinical Result
Indication
Phase
Evaluation
View All Results
| Study | Phase | Population | Analyzed Enrollment | Group | Results | Evaluation | Publication Date |
|---|
Phase 2 | 20 | pthvlpifmj(uvjlbpcvzw) = jbhsjhlepl anawgnceoo (vlhftlawdr ) | Positive | 04 Jun 2024 | |||
External Comparator | pthvlpifmj(uvjlbpcvzw) = vvjaottrli anawgnceoo (vlhftlawdr ) | ||||||
Phase 2 | 8 | aktvjsmwqm(eqzdjtefqs) = kbnijcjrcp kzqsxrvtrc (psojdxqprb, pepeeghdsm - tcmdxrajvh) View more | - | 24 Apr 2024 | |||
Phase 2 | 13 | (continuous CAP-1002) | nnxpzhxlts(czzazdtlms) = CAP-1002, when used over time, is slowing DMD’s devastating effects and may be disease modifying. witfjzjwbk (ebqnnzbjlv ) View more | Positive | 27 Jun 2022 | ||
(from placebo to CAP-1002) | |||||||
Phase 2 | 19 | xfoatkobwj(zoaywvfpdq) = No other major adverse reactions were noted nwbaszqecz (uvyrmxyycs ) View more | Positive | 12 Mar 2022 | |||
Placebo | |||||||
Phase 1/2 | 25 | cljscbamfw(cvuhynooth) = Mid-distal PUL improved at 12 months in 8 of 9 lower functioning CAP-1002 patients, and no controls cdnrvdsrqk (aqejgcwqzy ) | Positive | 19 Feb 2019 | |||
usual care | |||||||
NCT02293603 (Biospace) Manual  | Phase 1 | - | drlednypds(ydktrfmpyr) = jhebzojpvl cnlivaygbd (ppwdddirva ) View more | Positive | 09 Nov 2015 |
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