FDA Selects Moderna's mRNA-3705 for START Pilot Program

Moderna, a leader in mRNA therapeutic development, has announced that the U.S. Food and Drug Administration (FDA) has chosen its investigational drug, mRNA-3705, for inclusion in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. This initiative is aimed at fast-tracking the development of treatments for rare diseases with significant unmet medical needs.

mRNA-3705 is being developed as a therapeutic for methylmalonic acidemia (MMA) caused by a deficiency in the mitochondrial enzyme methylmalonic-CoA mutase (MUT). This rare metabolic disorder can lead to severe health issues, including metabolic crises and multi-organ diseases, contributing to high mortality and morbidity rates. Currently, there are no approved treatments for MMA, with the standard care involving only dietary and palliative measures. In more severe cases, liver or combined liver and kidney transplants are the only effective treatment options available.

Dr. Kyle Holen, Moderna's Senior Vice President and Head of Development, Therapeutics and Oncology, expressed the company's enthusiasm about the FDA's decision. He highlighted that the selection of mRNA-3705 underscores the potential of Moderna's mRNA technology beyond vaccines. The inclusion in the START program is expected to enhance communication with the FDA, expediting the development process and paving the way for a pivotal study initiation in 2024.

The START pilot program was launched by the FDA in September 2023 to speed up the development of new treatments for rare diseases. It aims to select up to seven pioneering treatments, with three chosen by the Center for Drug Evaluation and Research (CDER) and four by the Center for Biologics Evaluation and Research (CBER). The program is designed to facilitate high-quality data generation to support marketing approvals. Selected manufacturers benefit from enhanced interactions with the FDA, allowing for more rapid and flexible guidance on clinical development aspects such as study design, patient population, and statistical methods.

mRNA-3705 is currently undergoing evaluation in a Phase 1/2 clinical trial, known as the "Landmark study." This adaptive, open-label study aims to assess the safety and tolerability of the drug when administered via intravenous infusion to patients aged one year and older with isolated MMA due to hMUT deficiency. The study is crucial for moving forward to more advanced stages of development.

Moderna’s investigational therapeutic, mRNA-3705, functions by instructing the body to produce the missing or dysfunctional proteins that cause MMA. It consists of mRNA encoding the human MUT enzyme, encapsulated within Moderna's proprietary lipid nanoparticle (LNP) delivery system. This same LNP formulation is utilized in Moderna's propionic acidemia program, mRNA-3927. The FDA has granted mRNA-3705 several designations, including Orphan Drug, Fast Track, and Rare Pediatric Disease, reflecting the urgent need for effective treatments for MMA.

Moderna is renowned for its groundbreaking work in mRNA technology, which has been instrumental in developing vaccines and therapeutics at unprecedented speeds. The company's mRNA platform has facilitated advancements across various medical fields, including infectious diseases, immuno-oncology, rare diseases, and autoimmune conditions. By operating at the intersection of science, technology, and health, Moderna aims to transform the treatment and prevention of diseases globally.

In summary, the FDA’s selection of mRNA-3705 for the START pilot program marks a significant milestone in the pursuit of effective treatments for MMA. With enhanced communication and support from the FDA, Moderna is well-positioned to advance mRNA-3705 through the clinical development pipeline, potentially offering new hope for patients with this life-threatening condition.

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