After completing enrollment in 5 dose cohorts, and with no dose-limiting safety signals observed to date by the independent DSMB, the Phase 1/2 trial of mRNA-3927 advances to the dose-expansion phase to determine a recommended dose

CAMBRIDGE, MA / ACCESSWIRE / May 3, 2023 / Moderna,Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced that an abstract reporting on interim data from the Phase 1/2 trial of mRNA-3927, an investigational mRNA therapy for propionic acidemia (PA) has been accepted for presentation at the 2023 American Society of Gene + Cell Therapy (ASGCT) Annual Meeting being held May 16-20, 2023 in Los Angeles, CA. The presentation details are as follows:

Title: mRNA-3927 Therapy for Propionic Acidemia: Interim Data from a Phase 1/2 Study
Session: Metabolic, Storage, Endocrine, Liver, and Gastrointestinal Diseases II
Date/Time: Thursday, May 18, 2023, 04:45 PM - 05:00 PM PT
Presenter: Gerald S. Lipshutz, MD, MS, David Geffen School of Medicine at University of California at Los Angeles

The ongoing global, Phase 1/2, open-label, multicenter, dose-optimization trial (ClinicalTrials.gov Identifier: NCT04159103) evaluates the safety, pharmacodynamics, and pharmacokinetics of mRNA-3927 in participants aged 1 year and up with genetically confirmed PA. The trial uses a dose-escalation approach to evaluate the intravenous administration of mRNA-3927. The initial dosing regimen was 0.3 mg/kg administered IV every 3 weeks; subsequent doses are administered every 2 weeks. Participants who complete the dose optimization trial (10 doses) are eligible to continue treatment in an open-label extension study (NCT05130437). At the ASGCT meeting, Moderna is presenting the latest results demonstrating mRNA-3927 is safe and advancing to the dose-expansion phase to further evaluate safety and efficacy and confirm the recommended dose for future clinical studies. This study includes a dose optimization stage (cohorts 1-5) followed by a dose expansion stage with progression dependent on the safety of the preceding cohort. Moderna's mRNA therapy for PA (mRNA-3927) encodes for two proteins that together can help the body make the enzyme that is deficient in PA patients.

"We are excited to see mRNA-3927 take these next steps in the development process and proud to work together with patients, families, and researchers to understand better the therapeutic potential of our mRNA candidate for propionic acidemia," said Dr. Kyle Holen, M.D., Moderna's Senior Vice President and Head of Development, Therapeutics and Oncology. "Advancing our PA clinical trial to dose expansion represents another important milestone for our rare disease portfolio. We are particularly encouraged by the reports we are hearing from patients and their caregivers on the impact mRNA-3927 has had on their families and are eager to proceed with further clinical testing."

About Propionic Acidemia (PA) and mRNA-3927

Propionic acidemia is a rare, serious, inherited metabolic disorder with significant morbidity and mortality, affecting 1 in 100,000-150,000 individuals worldwide. PA is caused by pathogenic variants in the propionyl-coenzyme A carboxylase (PCC) α or β subunits (PCCA and PCCB genes, respectively), leading to PCC deficiency and subsequent accumulation of toxic metabolites. PA is characterized by recurrent life-threatening metabolic decompensation events (MDEs) and multisystemic complications. Currently, there are no effective therapies for PA that target the underlying root cause of the disease.

mRNA-3927 is a novel mRNA-based therapeutic agent that is composed of two mRNAs encoding for normal human PCCA and PCCB subunits. Intravenous (IV) administration of mRNA-3927 is intended to restore functional PCC enzymes in patients with PA.

About Moderna

In over 10 years sinceits inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio and integrated manufacturing facilities that allow for rapid clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuitof both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna's capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID pandemic.

Moderna's mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Moderna has been named a top biopharmaceutical employer by Science for the past eight years. To learn more, visit www.modernatx.com.

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: Moderna's Phase 1/2 trial of mRNA-3927; Moderna's presentation at the 2023 American Society of Gene + Cell Therapy (ASGCT) Annual Meeting; the potential for mRNA-3927 to effectively treat patients with propionic acidemia; and the burden associated with propionic acidemia. In some cases, forward-looking statements can be identified by terminology such as "will," "may," "should," "could," "expects," "intends," "plans," "aims," "anticipates," "believes," "estimates," "predicts," "potential," "continue," or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. 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, 2022, filed with the U.S. Securities and Exchange Commission (SEC), and in subsequent filings made by Moderna with the SEC, 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.

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