Barth syndrome

STEALTH BIOTHERAPEUTICS' BARTH SYNDROME NDA RECEIVES PRIORITY REVIEW DESIGNATION

Retrieved on: 
Tuesday, May 7, 2024
FDA, Syndrome, Elamipretide, Priority review, Mitochondrial myopathy, NDA, Food, Policy, New Drug Application, Agency, Ultra, Barth syndrome, Disease, Diagnosis, Death, Orphan drug, Pharmaceutical industry

NEEDHAM, Mass., May 7, 2024 /PRNewswire/ -- Stealth BioTherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration ("FDA") has now determined that its New Drug Application ("NDA") for elamipretide for the treatment of Barth syndrome is entitled to a Priority Review designation. Per FDA policy, the user fee goal date of January 29, 2025 is unchanged.

Key Points: 
  • The FDA grants Priority Review to applications for therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions.
  • Barth syndrome is an ultra-rare cardioskeletal disease which results in frequent pediatric deaths and imposes a significant disease burden on all affected individuals.
  • The FDA's decision to grant Priority Review designation follows its March 29, 2024 filing decision in which the FDA assigned a standard review designation.
  • Elamipretide has previously received Orphan Drug, Fast Track and Rare Pediatric Designations for elamipretide for the treatment of Barth syndrome.

STEALTH BIOTHERAPEUTICS ANNOUNCES FDA ACCEPTANCE OF NEW DRUG APPLICATION FOR ELAMIPRETIDE FOR THE TREATMENT OF BARTH SYNDROME

Retrieved on: 
Monday, April 8, 2024
Friends, New Drug Application, Elamipretide, Patient, Barth syndrome, NDA, Syndrome, Ultra, Quality of life, Safety, Muscle weakness, Stealth, Fatigue, FDA, Food, Medicine, Pharmaceutical industry

NEEDHAM, Mass., April 8, 2024 /PRNewswire/ -- Stealth BioTherapeutics, a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration ("FDA") has accepted for filing its New Drug Application ("NDA") for elamipretide for the treatment of Barth syndrome. The NDA is supported by the positive data from the SPIBA-001 Phase 3 Natural History Control Study and additional supporting efficacy and safety data from the TAZPOWER Part 2 baseline-controlled trial. Elamipretide received Fast Track Designation in 2017, Orphan Drug Designation in 2018 and Rare Pediatric Disease Designation in 2020. 

Key Points: 
  • Elamipretide received Fast Track Designation in 2017, Orphan Drug Designation in 2018 and Rare Pediatric Disease Designation in 2020.
  • The FDA indicated that it is currently planning to hold an advisory committee meeting to discuss the application.
  • The application was assigned a standard review designation, which the Company has asked FDA to reconsider.
  • Elamipretide is also in development for primary mitochondrial myopathy, with pivotal data from the fully-enrolled Phase 3 NuPOWER trial expected in late 2024.

The Barth Syndrome Foundation Delivers Petition to FDA Advocating for a Fair, Equitable and Appropriate Review of the Only Potential Treatment for Barth Syndrome

Retrieved on: 
Thursday, December 21, 2023
Patient, Elamipretide, Nephrology, Food, Genetic disorder, State, Division, Death, Infection, Friends, United States Congress, FDA, Hope, Fatigue, Disease, Exercise, NDA, Incidence, Muscle weakness, Congress, BSF, Education, Heart, Clinical trial, Life expectancy, Natural history, New Drug Application, Mutation, Barth syndrome, Military personnel, Medicine, Heart failure, DSM-IV codes, Child, Life, Pharmaceutical industry

BOSTON, Dec. 21, 2023 /PRNewswire/ -- The Barth Syndrome Foundation (BSF), the only patient advocacy organization dedicated to Barth syndrome and saving lives around the world through education, advances in treatment and finding a cure, today announced that it is petitioning the U.S. Food and Drug Administration (FDA) to review the New Drug Application (NDA) for elamipretide, the only potential treatment for Barth syndrome, in a fair, equitable and appropriate manner. Despite compelling evidence that elamipretide has been well-tolerated and has demonstrated clinical benefit in Barth syndrome, and despite ongoing efforts by the Barth Syndrome Foundation and other advocates to engage with regulators, the FDA has refused to review an NDA to date. The petition ran from mid-September to mid-November, and in just two months, garnered nearly 20,000 signatures from individuals across all 50 U.S. states, plus Washington, DC, Puerto Rico, the U.S. Virgin Islands and deployed military personnel.

Key Points: 
  • There are currently no FDA-approved treatments for Barth syndrome, and there are no other potential therapies in clinical development.
  • The Barth Syndrome Foundation and leading scientific thought leaders lobbied for the development of elamipretide for Barth syndrome beginning in 2014.
  • There simply aren't enough people with Barth syndrome, though, to meet the impossible statistical requirements of the FDA to give elamipretide a full drug review.
  • Barth syndrome is a rare, X-linked genetic disorder with an estimated incidence of only 1 in 1 million live births.

PHARMANOVIA ENTERS NEW CHEMICAL ENTITY LICENSING AGREEMENT WITH STEALTH BIOTHERAPEUTICS TO MARKET AND FURTHER DEVELOP A POTENTIAL NOVEL TREATMENT FOR BARTH SYNDROME

Retrieved on: 
Tuesday, May 30, 2023
NORTH, Barth syndrome, Partnership, AFRICA, Quality of life, AND, Muscle weakness, Patient, Cardiomyopathy, Cardiology, MENA, RARE, Safety, Pharmaceutical industry, EU

The agreement gives Pharmanovia the licensing rights to elamipretide for Barth syndrome in Europe and MENA.

Key Points: 
  • The agreement gives Pharmanovia the licensing rights to elamipretide for Barth syndrome in Europe and MENA.
  • Orphan Drug designations from the FDAiv and EMAv have been granted, demonstrating the unmet need in this condition.
  • It is estimated that Barth syndrome affects 1 in 300,000 to 400,000 births, making this an ultra-rare disease.
  • Partnering with Pharmanovia is a natural choice to achieve that goal given their experience in bringing medicines to patients across Europe and MENA and our mutual alignment on elamipretide’s potential to address the unmet medical needs of patients living with Barth syndrome.”

Stealth BioTherapeutics Enters into Exclusive Licensing Agreement with Pharmanovia to Commercialize Elamipretide for the Treatment of Barth Syndrome in Europe and Key Global Territories

Retrieved on: 
Tuesday, May 30, 2023
Barth syndrome, Stealth mode, Patient, European, Elamipretide, The Company, Rare, MENA, FDA, Treatment, United Arab Emirates, Pharmaceutical industry, EU, Stealth

NEEDHAM, Mass., May 30, 2023 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth BioTherapeutics"), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced their entry into an exclusive licensing agreement whereby Pharmanovia will commercialize elamipretide, Stealth's lead investigational product candidate, in Europe and other key global territories for the treatment of Barth syndrome, an ultra-rare life-threatening cardiac condition caused by mitochondrial cardiolipin deficiency for which there are currently no approved treatments. Elamipretide, an investigational mitochondrial protective agent which has been shown to improve mitochondrial structure and function through its interaction with cardiolipin, has completed Phase 3 development as a potential treatment for Barth syndrome.

Key Points: 
  • Elamipretide, an investigational mitochondrial protective agent which has been shown to improve mitochondrial structure and function through its interaction with cardiolipin, has completed Phase 3 development as a potential treatment for Barth syndrome.
  • In exchange, Stealth BioTherapeutics will receive upfront and future development, regulatory, and commercial milestone payments, plus tiered royalties on net sales of elamipretide in the licensed territories.
  • "While we work with FDA toward a potential US approval pathway, we remain committed to expanding access to elamipretide to individuals living with Barth syndrome in Europe and beyond" said Reenie McCarthy, Chief Executive Officer of Stealth.
  • James Burt, Chief Executive Officer of Pharmanovia, added "We are delighted to be Stealth BioTherapeutics' partner across EU, Switzerland, Norway, UK, Iceland and MENA.

The Barth Syndrome Foundation Recognizes First Annual Barth Syndrome Awareness Day

Retrieved on: 
Wednesday, April 5, 2023
Infection, Barth syndrome, Mayo Clinic, Heart failure, Education, BSF, Mutation, Research, Life expectancy, Muscle weakness, Death, Participation, Patient, Antisemitic Propaganda: an annotated bibliography and research guide, Diagnosis, FDA, DSM-IV codes, Disease, Pharmaceutical industry

BOSTON, April 5, 2023 /PRNewswire/ -- The Barth Syndrome Foundation (BSF), the only patient advocacy organization dedicated to Barth syndrome and saving lives around the world through education, advances in treatment and finding a cure, today recognizes the first annual Barth Syndrome Awareness Day .

Key Points: 
  • BOSTON, April 5, 2023 /PRNewswire/ -- The Barth Syndrome Foundation (BSF), the only patient advocacy organization dedicated to Barth syndrome and saving lives around the world through education, advances in treatment and finding a cure, today recognizes the first annual Barth Syndrome Awareness Day .
  • Every person with Barth syndrome or any other ultra-rare disease deserves a chance for a better, fuller, longer life.
  • "We are tremendously grateful to Representative Tonko, Representative Norman and Representative Bilirakis for introducing bipartisan legislation to recognize the first annual Barth Syndrome Awareness Day," said Emily Milligan, Executive Director of BSF.
  • If you have Barth syndrome or know someone who does, we encourage you to participate in Barth Syndrome Awareness Day in any of the following ways:
    Join our "Move 4.5" challenge to build a healthy habit and raise funds, or donate to make research and development and other programs possible
    Participate in Barth syndrome research through joining the Research GUID program, enrolling in the Barth syndrome registry or finding out if you qualify for a Mayo Clinic study to improve diagnosis

Stealth BioTherapeutics Announces Departure of Chief Financial Officer

Retrieved on: 
Friday, September 30, 2022
Degenerative disease, Risk, Safety, CFO, Stealth, COVID-19, Duchenne, Brain, FDA, Barth syndrome, Hope, Duchenne muscular dystrophy, Cardiomyopathy, Company, Investment, Forward-looking statement, Patient, Eye, U.S. Securities and Exchange Commission, CEO, Lists of diseases, Annual report, Nasdaq, Accounting, Board of directors, IgG4-related ophthalmic disease, Private Securities Litigation Reform Act, Amyotrophic lateral sclerosis, Board, Heart, Frontotemporal dementia, Marketing, Neuromuscular Disorders, Drug discovery, Patent, EMA, Mitochondrial myopathy, Pharmaceutical industry, Mitochondrion

BOSTON, Sept. 30, 2022 /PRNewswire/ -- Stealth BioTherapeutics Corp (Nasdaq:MITO), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that Robert Weiskopf, Stealth's chief financial officer, is leaving the company effective September 30, 2022.

Key Points: 
  • BOSTON, Sept. 30, 2022 /PRNewswire/ -- Stealth BioTherapeutics Corp (Nasdaq:MITO), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that Robert Weiskopf, Stealth's chief financial officer, is leaving the company effective September 30, 2022.
  • Mr. Weiskopf joined Stealth in 2019 following the company's initial public offering to spearhead its public financial reporting activities and grow the Company's financial team.
  • "Rob has played a key role in building the infrastructure to support Stealth as a public company and developing its accounting and reporting capabilities.
  • Rob leaves the company with a strong accounting and financial reporting foundation as well as a talented finance and accounting team," Ms. McCarthy said.

Stealth BioTherapeutics Announces Data from the Phase 2 ReCLAIM-2 Study of Elamipretide in Geographic Atrophy at the Clinical Trials at the Summit Meeting 2022

Retrieved on: 
Monday, May 23, 2022
Cardiomyopathy, U.S. Securities and Exchange Commission, Retina, Visual acuity, Lists of diseases, Macular degeneration, EMA, IgG4-related ophthalmic disease, Private Securities Litigation Reform Act, Clinical trial, Degenerative disease, SEC, Stealth, Risk, Annual report, Neuromuscular Disorders, Elamipretide, Quality of life, COVID-19, Summit, Marketing, Duchenne, Patent, Brain, Protalix BioTherapeutics, Death, Health, Severe cognitive impairment, Eye, Amyotrophic lateral sclerosis, Frontotemporal dementia, FDA, Program, Heart, Barth syndrome, Security (finance), Reclaim, Nasdaq, Patient, Duchenne muscular dystrophy, Hope, AMD, Neuron, Geographic atrophy, Forward-looking statement, Safety, Vance DeGeneres, Mitochondrial myopathy, EZ, CEO, Pharmaceutical industry, Medical imaging, Mitochondrion

BOSTON, May 23, 2022 /PRNewswire/ -- Stealth BioTherapeutics Corp (Nasdaq: MITO), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, announced new data from the ReCLAIM-2 study of elamipretide in geographic atrophy (GA) secondary to dry age-related macular degeneration (dry AMD) at the Clinical Trials at the Summit meeting in Lake Tahoe, Nevada.

Key Points: 
  • Additional analyses show that, consistent with prior findings from the ReCLAIM Phase 1 study, changes in LLVA correlated with baseline EZ attenuation in ReCLAIM-2.
  • Such forward-looking statements include those regarding Stealth BioTherapeutics' expectations for its clinical development of elamipretide for GA associated with dryAMDand other indications, the potential benefits of elamipretide and potential regulatory interactions.
  • Statements that are not historical facts, including statements about Stealth BioTherapeutics' beliefs, plans and expectations, are forward-looking statements.
  • Except as required by law, Stealth BioTherapeutics does not undertake any obligation to update forward-looking statements made by us to reflect subsequent events or circumstances.

Stealth BioTherapeutics Receives Orphan Drug Designation from FDA for Elamipretide for Treatment of Duchenne Muscular Dystrophy

Retrieved on: 
Thursday, May 12, 2022
Disease, Cardiomyopathy, Orphan Drug Act of 1983, US Foods, Cardiology, PMO, Neuromuscular Disorders, PMOS, U.S. Securities and Exchange Commission, Nasdaq, Heart, Patient, Duchenne muscular dystrophy, Private Securities Litigation Reform Act, Degenerative disease, DMD, COVID-19, Marketing, Eye, Lists of diseases, Amyotrophic lateral sclerosis, Duchenne, Orphan drug, Hope, FDA, AMD, Patent, Brain, Forward-looking statement, EMA, Safety, Elamipretide, Protalix BioTherapeutics, Orphan, Vance DeGeneres, IgG4-related ophthalmic disease, Mitochondrial myopathy, Nephrology, Person, SEC, Stealth, Barth syndrome, Security (finance), Risk, Annual report, Pharmaceutical industry, Mitochondrion

BOSTON, May 12, 2022 /PRNewswire/ -- Stealth BioTherapeutics Corp (Nasdaq: MITO), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, announced today that the US Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to elamipretide for the treatment of patients with Duchenne muscular dystrophy (DMD). 

Key Points: 
  • The company has previously presented data demonstrating that administration of elamipretide in combination with a PMO significantly improves dystrophin expression levels in the X-linked muscular dystrophy (mdx) mouse model.
  • "We are pleased that the FDA has recognized the high unmet need for innovative treatments for DMD," said Chief Executive Officer Reenie McCarthy.
  • Once granted, Orphan Drug Designation provides various development benefits for an investigational drug, including seven-year exclusivity after marketing approval is received.
  • Such forward-looking statements include those regardingStealth BioTherapeutics'expectations for elamipretide preclinical data and development efforts for Duchenne muscular dystrophy.

STEALTH BIOTHERAPEUTICS ANNOUNCES DATA FROM RECLAIM-2 PHASE 2 TRIAL OF ELAMIPRETIDE IN GEOGRAPHIC ATROPHY

Retrieved on: 
Monday, May 2, 2022
Vance DeGeneres, Amyotrophic lateral sclerosis, Lists of diseases, Safety, Eye, Degenerative disease, Risk, Annual report, OCT, CEO, GA, Investor, Northwest Biotherapeutics, Forward-looking statement, Brain, Company, Private Securities Litigation Reform Act, Patient, Stealth, FDA, Elamipretide, Disease, Heart, EMA, Retina, Geographic atrophy, IgG4-related ophthalmic disease, Mitochondrial myopathy, Barth syndrome, U.S. Securities and Exchange Commission, Neuromuscular Disorders, Hope, COVID-19, Nasdaq, Cardiomyopathy, Duchenne, Webcast, Duchenne muscular dystrophy, Patent, Marketing, Food and Drug Administration Amendments Act of 2007, AMD, Severe cognitive impairment, Medical imaging, Pharmaceutical industry, Mitochondrion

BOSTON, May 2, 2022 /PRNewswire/ -- Stealth BioTherapeutics (Nasdaq: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced top-line data from its Phase 2 ReCLAIM-2 trial evaluating elamipretide in patients with geographic atrophy (GA) secondary to dry age-related macular degeneration.  Although the trial did not meet its primary endpoints assessing mean changes in LLVA and GA, a key secondary endpoint showed however that elamipretide categorically improved visual function for patients with GA.  Additionally, elamipretide demonstrated proof of mechanism by reducing progressive ellipsoid zone loss, a key biomarker of retinal mitochondrial health which has been shown to be predictive of long-term GA growth and development.  Based on these data, the Company currently plans to progress development initiatives intended to better position this indication for potential partnering consistent with previous guidance.

Key Points: 
  • ET
    BOSTON, May 2, 2022 /PRNewswire/ -- Stealth BioTherapeutics (Nasdaq: MITO), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced top-line data from its Phase 2 ReCLAIM-2 trial evaluating elamipretide in patients with geographic atrophy (GA) secondary to dry age-related macular degeneration.
  • Administration of elamipretide resulted in a 2 or more line gain in LLVA for greater than 15% of patients completing the 48-week trial (p=0.04).
  • Stealth will host a conference call and webcast today at 8:30 am ET to discuss the ReCLAIM-2 trial.
  • Geographic atrophy (GA) is an advanced form of dry age-related macular degeneration (AMD), a leading cause of blindness.