Forzinity (elamipretide) / Stealth BioTherapeutics - LARVOL DELTA

SHAPE: Study of Healthy Aging and Physical Function With Elamipretide (clinicaltrials.gov) - P2 | N=30 | Recruiting | Sponsor: David Marcinek

New P2 trial

Elamipretide: First Approval. (PubMed, Drugs) - "Elamipretide (Forzinity™) is a mitochondrial cardiolipin binder being developed by Stealth BioTherapeutics for the treatment of a range of disorders featuring mitochondrial dysfunction...Elamipretide is also under phase III clinical development for use in the treatment of dry age-related macular degeneration and mitochondrial myopathies. This article summarizes the milestones in the development of elamipretide leading to this first approval for Barth syndrome."

Journal • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Genetic Disorders • Macular Degeneration • Metabolic Disorders • Myositis • Ophthalmology • Pediatrics • Retinal Disorders

Real-world disease burden and health care resource utilization for patients with Barth syndrome. (PubMed, J Med Econ) - "With a noteworthy absence of pharmacoeconomic evidence, utilization of combined clinical case report data from the medical literature, along with registry and claims datasets, demonstrate that BTHS is a costly disease associated with high disease burden and excessive HCRU. Forthcoming treatments (e.g. elamipretide) have the potential to reduce the high disease burden/HCRU."

HEOR • Journal • Real-world evidence • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Genetic Disorders • Heart Failure • Rare Diseases • Transplantation

Elamipretide: The first cardiolipin-directed mitochondrial therapeutic for Barth syndrome approved under accelerated approval. (PubMed, Drug Discov Ther) - "As a condition of accelerated approval, a confirmatory trial is required. Elamipretide represents a promising therapy addressing an unmet medical need in BTHS and provides a foundation for future mitochondria-targeted treatments."

Clinical • Journal • Cardiomyopathy • Cardiovascular • Fatigue • Hematological Disorders • Metabolic Disorders • Myositis • Neutropenia • TAFAZZIN

FORZINITY (Elamipritide). (PubMed, Clin Ther) - No abstract available

Journal

NuPower: Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) (clinicaltrials.gov) - P3 | N=102 | Completed | Sponsor: Stealth BioTherapeutics Inc. | Active, not recruiting ➔ Completed

Trial completion • Metabolic Disorders • Myositis • DNA2 • MGME1 • RRM2B

ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD) (clinicaltrials.gov) - P3 | N=313 | Active, not recruiting | Sponsor: Stealth BioTherapeutics Inc. | Recruiting ➔ Active, not recruiting | Trial primary completion date: Aug 2026 ➔ Aug 2027

Enrollment closed • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Stealth BioTherapeutics Announces FDA Accelerated Approval of FORZINITY (elamipretide HCl), the First Therapy for Progressive and Life-limiting Ultra-rare Genetic Disease Barth Syndrome (PRNewswire) - "The approval of FORZINITY is supported by the efficacy and safety data from the TAZPOWER clinical trial."

Accelerated approval • Genetic Disorders

STEALTH BIOTHERAPEUTICS RESUBMITS NEW DRUG APPLICATION FOR ELAMIPRETIDE FOR THE TREATMENT OF BARTH SYNDROME (PRNewswire) - "At FDA's request, the resubmission also contains additional post-marketing commitments regarding a proposed post-marketing trial to confirm the clinical benefit of elamipretide for the treatment of Barth syndrome."

FDA filing • Genetic Disorders

Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report. (PubMed, Mol Genet Metab) - "Here we describe a case of prenatally identified Barth syndrome-related severe left ventricle (LV) non-compaction cardiomyopathy, where ELAM was initiated shortly after birth for clinical heart failure and was associated with significant and sustained clinical improvement leading to an inactive status on the heart transplant list with eventual anticipated delisting. We provide a review of the current literature including the pathophysiology of Barth syndrome, the mechanism of action of ELAM, and its clinical applications."

Journal • Review • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Disorders • Metabolic Disorders • Myositis • Neutropenia • Transplantation

Cardiac Pathology in a Patient with a Novel Pathogenic Variant c.703del (p.Ile235SerfsTer4) of the TAFAZZIN Gene. (PubMed, Cardiovasc Pathol) - "We describe a case of Barth syndrome harboring a novel pathogenic variant of the TAFAZZIN gene exhibiting dilated cardiomyopathy, hypertrabeculation, endocardial fibroelastosis, and prominent mitochondrial abnormality. Elamipretide was well tolerated."

Journal • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Metabolic Disorders • Transplantation • TAFAZZIN

Contemporary insights into elamipretide's mitochondrial mechanism of action and therapeutic effects. (PubMed, Biomed Pharmacother) - "Elamipretide continues to show promise as a potential therapy for mitochondrial disorders. New basic science advances have improved understanding of elamipretide's MOA, enabling a better understanding of the molecular consequences of elamipretide-cardiolipin interactions."

Journal • Review • Age-related Macular Degeneration • Macular Degeneration • Metabolic Disorders • Myositis • Ophthalmology • Retinal Disorders

Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study. (PubMed, Orphanet J Rare Dis) - P2/3 | "Though the small sample size limits strong conclusions, this analysis suggests the BTHS-SA can produce reliable scores upon which valid inferences may be drawn. The BTHS-SA may be a useful tool to evaluate treatment benefits in this underserved population."

Clinical • Journal • P2 data • Fatigue • Myositis • TAFAZZIN

Significantly Improved Cardiac Function in Neonate with Barth Syndrome on Investigational Drug Elamipretide (ISHLT 2025) - "He was discharged home on oral heart failure medications and subcutaneous elamipretide...Barth syndrome is an X-linked recessive mitochondrial disorder arising from pathogenic variants in the TAFAZZIN gene, encoding a transacylase in the inner mitochondrial that catalyzes final processing and maturation of cardiolipin, which is important for high energy-requiring tissues such as cardiac muscle. Elamipretide is an investigational drug that stabilizes cardiolipin, resulting in mitochondrial membrane stabilization.Summary Our neonate with Barth syndrome associated LVNC cardiomyopathy showed remarkable improvement in cardiac function on elamipretide, demonstrating the potential of this medication for patients with this rare syndrome."

Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Metabolic Disorders • TAFAZZIN

Cardiac Pathology in a Patient with a Novel Likely Pathogenic Variant C.703del (p.Ile235SerfsTer4) of the TAZ Gene (ISHLT 2025) - "He continues to do well as an outpatient and remains on subcutaneous Elamipretide.Summary We describe a case of Barth syndrome harboring a novel likely pathogenic variant of the TAZ gene exhibiting dilated cardiomyopathy, endocardial fibroelastosis, and prominent mitochondrial abnormalities. Elamipretide is well tolerated and may have temporized the cardiac function to serve as a medical bridge to transplantation."

Clinical • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Metabolic Disorders • TAFAZZIN

Topical bevemipretide (SBT-272) eye drops in development for dry AMD: Phase 1 trial design informed by 13-week GLP study results (ARVO 2025) - "Conclusions Bevemipretide is a reimagined, next-generation small molecule in development for the treatment of dry AMD and follows the foundational studies and mechanism of action of subcutaneous elamipretide...Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

P1 data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology

Stealth BioTherapeutics Announces Achievement of 50% Enrollment Target in Phase 3 ReNEW Study of Elamipretide in Patients with Dry Age-Related Macular Degeneration (PRNewswire) - "Stealth BioTherapeutics Inc...today announced that it has achieved its 50 percent enrollment target in the global Phase 3 ReNEW study (NCT06373731) of elamipretide in patients with dry age-related macular degeneration (dry AMD). The total target enrollment is 360 participants. The ReNEW study is evaluating the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide – a first-in-class mitochondria-targeted investigational therapeutic – in participants with dry AMD...In addition to the ReNEW study, Stealth will initiate the ReGAIN study – a second global Phase 3 trial in dry AMD – in the coming months. Data from the ReNEW study is expected to be reported in 2026."

Enrollment status • New P3 trial • P3 data • Age-related Macular Degeneration

A Polytherapy Intervention in an Experimental Traumatic Optic Neuropathy Mouse Model. (PubMed, Ophthalmic Plast Reconstr Surg) - "Early combinatorial therapeutic intervention to address disparate molecular pathways following optic nerve trauma effectively halts retinal neurons' progressive structural and functional degeneration."

Journal • Preclinical • Inflammation • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain

Expanded-access use of elamipretide in a newborn with Barth syndrome: a case report. (PubMed, Eur Heart J Case Rep) - "He was weaned off oxygen completely on DOL49 and discharged home on DOL61 on daily subcutaneous elamipretide 0.5 mg/kg and oral heart failure medications...Our case suggests that elamipretide may have contributed to the improvement of LV function in this BTHS infant, supporting elamipretide's early use in BTHS. Our findings align with the previous studies in which elamipretide treatment demonstrated normalization of mitochondrial function and improvement in LV function."

Journal • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Genetic Disorders • Heart Failure • Metabolic Disorders

Expanded-access use of elamipretide in a critically ill patient with Barth syndrome. (PubMed, Genet Med Open) - No abstract available

Journal • Cardiomyopathy • Cardiovascular

ReCLAIM-2: A Randomized Phase II Clinical Trial Evaluating Elamipretide in Age-related Macular Degeneration, Geographic Atrophy Growth, Visual Function, and Ellipsoid Zone Preservation. (PubMed, Ophthalmol Sci) - P2 | "Administration of daily subcutaneous elamipretide 40 mg was investigated in subjects for 48 weeks followed by a 4-week follow-up period...The EZ attenuation/loss end point will serve as the regulatory approved primary end point in the elamipretide phase III clinical development program. Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article."

Journal • P2 data • Age-related Macular Degeneration • Dermatology • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Pain • Pruritus • Retinal Disorders

ELViS-FA: FRDA Investigator Initiated Study (IIS) With Elamipretide (clinicaltrials.gov) - P1/2 | N=20 | Completed | Sponsor: Children's Hospital of Philadelphia | Active, not recruiting ➔ Completed | Trial completion date: Dec 2024 ➔ Jul 2024

Metastases • Trial completion • Trial completion date • Ataxia • Friedreich ataxia • Movement Disorders

ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD) (clinicaltrials.gov) - P3 | N=360 | Recruiting | Sponsor: Stealth BioTherapeutics Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD) (clinicaltrials.gov) - P3 | N=360 | Not yet recruiting | Sponsor: Stealth BioTherapeutics Inc.

New P3 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Next-generation ocular topical SBT-272 demonstrates optimized retina tissue exposure: building upon elamipretide for treating dry AMD (ARVO 2024) - "Our in vivo results suggest that topical ocular SBT-272 has an advanced PK profile relative to SC elamipretide, with optimized delivery into retinal tissue resulting in increased retinal tissue concentrations while reducing systemic exposure. SBT-272 is a next-generation compound in development that follows the foundational studies and mechanism of action of SC elamipretide. Additional animal studies are ongoing, and the results will inform future development decisions."

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders