Stealth BioTherapeutics Receives Orphan Drug Designation from the European Medicines Agency for Elamipretide for the Treatment of Myopathic Mitochondrial DNA Depletion Syndrome

Rhea-AI Summary

Stealth BioTherapeutics (Nasdaq: MITO) has received orphan drug designation from the European Medicines Agency (EMA) for elamipretide aimed at treating myopathic mitochondrial DNA depletion syndrome (M-MDS). This designation applies to patients with nuclear DNA mutations participating in the ongoing NuPOWER Phase 3 clinical trial across multiple sites in the US and Europe. The ODD status underscores the unmet medical need for effective therapies, providing incentives such as marketing exclusivity in the EU for 10 years.

Positive

• Orphan drug designation granted by EMA for elamipretide enhances regulatory prospects in Europe.
• Strengthens potential for partnership opportunities in European markets.
• Ongoing enrollment in the pivotal NuPOWER Phase 3 trial indicates active progression in research.

Negative

• None.

Orphan drug designation applies to patients with primary mitochondrial myopathy due to nuclear DNA mutations (nPMM) in the NuPower Phase 3 clinical trial

NuPOWER Phase 3 trial is currently enrolling patients with multiple clinical trial sites initiated in the United States and Europe

BOSTON, May 31, 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 the European Medicines Agency (EMA) has granted orphan drug designation (ODD) for elamipretide for the treatment of myopathic mitochondrial DNA depletion syndrome (M-MDS), a designation which applies to patients with nPMM eligible for enrollment in the NuPOWER Phase 3 clinical trial, which is currently enrolling patients at multiple clinical trial sites in the United States and Europe.

"We are pleased that EMA has recognized the high unmet need for innovative treatments for this rare genetic condition which significantly limits the quality of life of affected individuals," said Chief Executive Officer Reenie McCarthy. "Orphan drug designation for M-MDS marks an important step toward advancing our regulatory initiatives in Europe.  As we continue to enroll patients in this pivotal trial, this designation strengthens our ability to support partnership opportunities in the European markets"

ODD is granted to investigational therapies intended for the safe and effective treatment of rare diseases with an unmet medical need that affect fewer than 5 in 10,000 people in the European Union. This designation provides companies with certain benefits and incentives including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU Member States, and if approved, marketing exclusivity in the EU for 10 years.

About M-MDS and nPMM

M-MDS is caused by mutations in nuclear DNA genes encoding for enzymes responsible for maintenance and replication of mitochondrial DNA.  Mitochondrial DNA replication, which is critical to support muscular energy demands, can be impaired as a result of these mutations, leading to signs and symptoms of M-MDS.  M-MDS is characterized by  muscle weakness, exercise intolerance, peripheral neuropathy and, in some cases, progressive external ophthalmoplegia.  These patients are also considered to have primary mitochondrial myopathy, or PMM, for which the Company has previously received orphan drug designation from the US FDA.  There are currently no FDA- or EMA-approved therapies for patients with M-MDS.

About Stealth

We are a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction. Mitochondria, found in nearly every cell in the body, are the body's main source of energy production and are critical for normal organ function. Dysfunctional mitochondria characterize a number of rare genetic diseases and are involved in many common age-related diseases, typically involving organ systems with high energy demands such as the heart, the eye, and the brain. We believe our lead product candidate, elamipretide, has the potential to treat both rare metabolic cardiomyopathies, such as Barth, Duchenne muscular dystrophy and Friedreich's ataxia, rare mitochondrial diseases entailing nuclear DNA mutations, as well as ophthalmic diseases entailing mitochondrial dysfunction, such as dry age-related macular degeneration. We are evaluating our second-generation clinical-stage candidate, SBT-272, and our new series of small molecules, SBT-550, for rare neurological disease indications following promising preclinical data. We have optimized our discovery platform to identify novel mitochondria-targeted compounds which may be nominated as therapeutic product candidates or utilized as mitochondria-targeted vectors to deliver other compounds to mitochondria.

Forward-looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding Stealth BioTherapeutics' plans, strategies and expectations for the clinical advancement of its drug development programs, including its ongoing clinical trial of elamipretide in nPMM and its expectations regarding regulatory interactions and partnership opportunities. Statements that are not historical facts, including statements about Stealth BioTherapeutics' beliefs, plans and expectations, are forward-looking statements. The words "anticipate," "expect," "hope," "plan," "potential," "possible," "will," "believe," "estimate," "intend," "may," "predict," "project," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Stealth BioTherapeutics may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of known and unknown risks, uncertainties and other important factors, including: Stealth BioTherapeutics' ability to obtain additional funding and to continue as a going concern; the impact of the COVID-19 pandemic; the ability to successfully demonstrate the efficacy and safety of Stealth BioTherapeutics' product candidates and future product candidates; the preclinical and clinical results for Stealth BioTherapeutics' product candidates, which may not support further development and marketing approval; the potential advantages of Stealth BioTherapeutics' product candidates; the content and timing of decisions made by the FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, which may affect the initiation, timing and progress of preclinical studies and clinical trials of Stealth BioTherapeutics product candidates; Stealth BioTherapeutics' ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Stealth BioTherapeutics' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Stealth BioTherapeutics' most recent Annual Report on Form 20-F filed with the Securities and Exchange Commission ("SEC"), as well as in any future filings with the SEC.  Forward-looking statements represent management's current expectations and are inherently uncertain. 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.

Investor Relations
Kendall Investor Relations
Adam Bero
abero@kendallir.com

IR@StealthBT.com 

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SOURCE Stealth BioTherapeutics Inc.

FAQ

What is the significance of the orphan drug designation for MITO?

The orphan drug designation from the EMA for elamipretide is significant as it provides regulatory and commercial advantages for treating rare diseases, enhancing the likelihood of successful drug development.

When did MITO announce the orphan drug designation?

The announcement regarding the orphan drug designation was made on May 31, 2022.

Where is the NuPOWER Phase 3 trial taking place for MITO?

The NuPOWER Phase 3 trial is currently enrolling patients at multiple sites in the United States and Europe.

What condition is elamipretide intended to treat according to the MITO press release?

Elamipretide is intended to treat myopathic mitochondrial DNA depletion syndrome (M-MDS) in patients with nuclear DNA mutations.

What are the benefits of orphan drug designation for MITO?

The benefits of the orphan drug designation include clinical protocol assistance, access to a centralized marketing authorization procedure, and 10 years of marketing exclusivity in the EU if approved.