FORZINITY™ (elamipretide) launch momentum continues one year after FDA approval

Pipeline advances across Barth syndrome, dry AMD, POLG, Parkinson’s and FAOD  

NEEDHAM, Mass., Oct. 14, 2026 /PRNewswire/ — Mighty Therapeutics Inc. (the “Company” or “Mighty”), a commercial-stage biotechnology company pioneering a new class of medicines that directly target mitochondrial dysfunction in rare and age-related diseases, today provided an update on key business, commercial, and pipeline milestones.

“We recently celebrated the first anniversary of the approval of FORZINITY, representing the first and only FDA-approved mitochondrial-targeted therapy, after a decade of close work with patients and advocacy groups to advance therapeutic options for diseases of mitochondrial dysfunction,” said Reenie McCarthy, Chief Executive Officer of Mighty. “One year later, we are gratified by ongoing commercial, clinical and regulatory progress in Barth syndrome, and we continue to break boundaries toward treatments for other rare and age-related diseases of mitochondrial dysfunction.”

Commercial Update
FORZINITY, which was approved by the FDA under accelerated approval on September 19, 2025, is currently commercially available for eligible U.S. patients who are living with Barth syndrome, a rare, serious, and life-threatening mitochondrial disease. FORZINITY is indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg.

From commercial soft launch in December 2025 through September 2026 Mighty reports:

• Gross Revenue of $17.5 million from December 2025, including $8.1 million for the third quarter of 2026;
• 60 patient enrollment forms received;
• 48 unique prescribers;
• Across the U.S. payer landscape, FORZINITY has received broad reimbursement access, with greater than 90% of lives covered across major national health plans;
• The Mito Assist™ patient support program continues to see broad adoption. This program provides education and access support and serves as a single point of coordination throughout the treatment journey.

Research and Development Update
Mighty continues to advance its development of elamipretide, bevemipretide and other mitochondria-targeted pipeline compounds for rare and age-related diseases.

• Dry AMD. The fully enrolled Phase 3 ReNEW clinical trial of elamipretide in dry age-related macular degeneration (AMD) remains on track for top-level data read-out in the second half of 2027. Mighty reached preliminary agreement with the FDA on endpoint selection and protocol design and plans to initiate its Phase 2 ReBUILD study of bevemipretide eye drops by year-end.
• Barth syndrome.
  • Following the first participant dosed, momentum continues in 4TAZPower, a global Phase 4 study to confirm the efficacy, safety, and pharmacokinetics of once daily elamipretide in individuals at least 5 years of age living with Barth syndrome. Study sites have been initiated in Canada, Australia, Ireland, and the United Kingdom.
  • The Company remains on track to initiate a one-month pharmacokinetic study this year to inform dosing of elamipretide for children weighing less than 30 kilograms. The study is intended to support a potential 2027 sNDA submission for children weighing at least 7 kg. The FDA will respond to the Company’s questions regarding the proposed sNDA submission contents via a Type C Written Response in the fourth quarter of 2026.
  • The Company continues to treat children weighing less than 7 kg through its expanded access program (EAP), typically entailing individual investigational new drug applications. The FDA has granted a meeting in the fourth quarter of 2026 to discuss an intermediate expanded access protocol intended to broaden access for these younger children. The Company has aligned with the FDA that data from this protocol may help inform future label expansion.
  • Mighty’s registry to collect long-term, real-world evidence from patients living with Barth syndrome, including patients taking FORZINITY or participating in EAP, will be initiated by year-end. The FDA encouraged Mighty’s registry efforts and has commented on the protocol, which may provide confirmatory evidence to support full approval in conjunction with the 4TAZPOWER data.
• POLG-related disorders. Mighty is requesting a meeting to align with FDA on a new pivotal trial evaluating elamipretide in primary mitochondrial disease due to nuclear polymerase gamma (POLG) mutations. The Company anticipates initiating a pivotal study in this indication in 2027.
• Preclinical pipeline.
  • Mighty continues to advance its broader pipeline of investigational mitochondria-targeted therapies, including systemic bevemipretide in Parkinson’s disease. The Company recently announced a collaboration with The POLG Foundation to develop a novel mouse-model of Parkinson’s to inform development efforts. Mighty is also progressing pipeline compound MYTX-589 in Leigh syndrome (through a collaboration supported by the UK Medical Research Council) and in Friedreich’s ataxia.
  • Mighty initiated IND-enabling studies for its third mitochondrial-targeted candidate, MYTX-255. Data presented at the 2026 INFORM meeting support the planned clinical development of MYTX-255 in fatty acid oxidation disorders (FAODs).
• Aging. A research team from Mighty and the University of Washington was named a Milestone 2 Awardee in the XPRIZE Healthspan competition. Work is underway to support a rigorous, placebo-controlled clinical trial of elamipretide in older adults, which will run through 2029.

Business Update
To support its ongoing pioneering work to develop a new class of mitochondrial-targeted therapeutics, Mighty Therapeutics secured up to $150 million in non-dilutive growth and equity capital through an agreement with K2 HealthVentures and additional equity financing from its founding investor. These agreements are expected to secure runway to projected cash flow positivity in early 2029.  

Recognition
Mighty was named a 2026 Industry Innovator in the National Organization for Rare Disorders (NORD®) Rare Impact Awards, which honor companies whose treatments have significantly improved outcomes for people living with rare diseases.

Chief Executive Officer Reenie McCarthy was also named one of 20 women changing the biopharma industry in Endpoints News’ 2026 Women in Biopharma special report, recognizing her leadership in advancing mitochondrial medicine for people living with rare diseases.

About FORZINITY™ (elamipretide) injection

INDICATION

FORZINITY™ is a mitochondrial cardiolipin binder indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg.

This indication is approved under accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

Contraindications
FORZINITY is contraindicated in patients with serious hypersensitivity to elamipretide or any of the excipients in FORZINITY.

Warnings and Precautions
Benzyl Alcohol Toxicity – Do Not Use in Neonates
Serious and fatal reactions (including metabolic acidosis progressing to neurotoxicity and gasping syndrome) have occurred in preterm and low–birth weight neonates receiving benzyl alcohol (BA)-containing drugs.
FORZINITY contains 20 mg BA/mL and is not approved for use in neonates or for IV administration.

Hypersensitivity
Reported reactions include rash, papular lesions, eczema/dermatitis, cough, and serious allergic reactions requiring emergency medical intervention.
Reactions may occur minutes to months after starting treatment. Monitor patients for signs and symptoms during treatment.
Discontinue FORZINITY permanently if a serious hypersensitivity reaction occurs.

Adverse Reactions
Adverse reactions occurring more commonly on FORZINITY than on placebo included injection site reactions such as injection site erythema, pain, induration, pruritus, bruising, and urticaria.

Eosinophilia
Modest elevations in eosinophil counts (peak ~90 days) occurred but were not associated with clinical symptoms.

To report SUSPECTED ADVERSE REACTIONS, contact FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Full Prescribing Information for FORZINITY.

About Mighty Therapeutics

Mighty is advancing novel therapies for people living with diseases involving mitochondrial dysfunction. Grounded in rigorous science and inspired by meaningful patient partnerships, the company is building a proprietary pipeline to directly address bioenergetic deficits at the source.

For more information, visit www.mightytx.com.

Media Contact
Ascent Strategic Communications
media@ascentcomms.com

Investor Contact
Precision AQ
Austin Murtagh
Austin.Murtagh@precisionaq.com

Patient Advocacy
PatientAdvocacy@mightytx.com

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SOURCE Mighty Therapeutics