Moderna's Investigational Therapeutic for Methylmalonic Acidemia (mRNA-3705) Selected by U.S. Food & Drug Administration for START Pilot Program

Center for Biologics Evaluation and Research has chosen mRNA-3705 as one of four investigational medicines for accelerated development to address unmet medical needs for rare diseases

CAMBRIDGE, MA / ACCESSWIRE / June 6, 2024 / Moderna, Inc. (NASDAQ:MRNA) today announced that the U.S. Food and Drug Administration (FDA) has selected mRNA-3705 for the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. mRNA-3705 is an investigational therapeutic for methylmalonic acidemia (MMA) due to methylmalonic-CoA mutase (MUT) deficiency.

"We are excited about this opportunity and proud that our investigational mRNA therapeutic for MMA was selected by the U.S. FDA for the START pilot program. This selection highlights the promise of Moderna's innovative mRNA platform beyond vaccines and the potential this novel medicine may have in addressing the serious and unmet medical needs of MMA," said Kyle Holen, M.D., Moderna's Senior Vice President and Head of Development, Therapeutics and Oncology. "Selection for this program will enable enhanced communication with the U.S. FDA, resulting in acceleration of our development program as we prepare for pivotal study initiation for mRNA-3705 in 2024."

The START pilot program was initiated by the U.S. FDA in September 2023 to accelerate the development of novel treatments addressing unmet medical needs in rare diseases, with an initial selection of up to seven novel treatments, three by the Center for Drug Evaluation and Research (CDER) and four by the Center for Biologics Evaluation and Research (CBER). The milestone-driven initiative is intended to help the progression to pivotal clinical studies or pre-BLA/NDA meeting stages by enhancing communications between manufacturers and the U.S. FDA. Selected manufacturers will benefit from rapid, ad-hoc U.S. FDA interactions to support clinical development, such as study design, patient population, and statistical methods, beyond standard formal meetings. The program is designed to generate high-quality, reliable data to support marketing approvals, ensuring promising treatments advance efficiently through regulatory milestones.

MMA is a rare, life-threatening, inherited metabolic disorder that is most commonly (approximately 60% of cases) caused by a deficiency in the mitochondrial enzyme MUT. This deficiency can lead to metabolic crises due to a toxic buildup of acids in the body, progressing into multi-organ disease. As a result, MMA is associated with significant mortality and morbidity, and there are no approved therapies. Standard of care includes dietary and palliative measures. Currently, liver or combined liver and kidney transplants are the only effective treatments.

mRNA-3705 is being investigated in a Phase 1/2 study, called the "Landmark study," an adaptive, open-label study designed to evaluate the safety and tolerability of the investigational therapeutic administered via intravenous infusion in patients one year and older with isolated MMA due to methylmalonyl-CoA mutase (hMUT) deficiency.

About mRNA-3705

mRNA-3705 is designed to instruct the body to restore the missing or dysfunctional proteins that cause MMA and consists of mRNA encoding human MUT, the mitochondrial enzyme commonly deficient in MMA, encapsulated within Moderna's proprietary lipid nanoparticle (LNP). mRNA-3705 uses the same proprietary LNP formulation as the Company's propionic acidemia (mRNA-3927) program. mRNA-3705 has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease designation by the U.S. FDA.

About Moderna

Moderna is a leader in the creation of the field of mRNA medicine. Through the advancement of mRNA technology, Moderna is reimagining how medicines are made and transforming how we treat and prevent disease for everyone. By working at the intersection of science, technology and health for more than a decade, the company has developed medicines at unprecedented speed and efficiency, including one of the earliest and most effective COVID-19 vaccines.

Moderna's mRNA platform has enabled the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases and autoimmune diseases. With a unique culture and a global team driven by the Moderna values and mindsets to responsibly change the future of human health, Moderna strives to deliver the greatest possible impact to people through mRNA medicines. For more information about Moderna, please visit modernatx.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the selection of mRNA-3705 to participate in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program; the potential benefit of mRNA-3705 for the treatment of methylmalonic acidemia due to methylmalonic-CoA mutase deficiency; and the potential initiation of a pivotal study for mRNA-3705 in 2024. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna's control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others, those risks and uncertainties described under the heading "Risk Factors" in Moderna's Annual Report on Form 10-K for the fiscal year ended December 31, 2023 and in subsequent filings made by Moderna with the U.S. Securities and Exchange Commission, which are available on the SEC's website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna's current expectations and speak only as of the date of this press release.

Moderna Contacts

Media:
Luke Mircea-Willats
Sr. Director, International Communications
Luke.mirceawillats@modernatx.com

Investors:
Lavina Talukdar
Senior Vice President & Head of Investor Relations
+1 617-209-5834
Lavina.Talukdar@modernatx.com

SOURCE: Moderna, Inc.