Moderna's Investigational Therapeutic for Methylmalonic Acidemia (mRNA-3705) Selected by U.S. Food & Drug Administration for START Pilot Program
Moderna's investigational therapeutic mRNA-3705 has been selected by the U.S. FDA for the START pilot program, which aims to accelerate the development of treatments for rare diseases. The FDA's Center for Biologics Evaluation and Research chose mRNA-3705 as one of four investigational medicines for this program. mRNA-3705 targets methylmalonic acidemia (MMA), a rare, life-threatening metabolic disorder. Inclusion in the START program will enhance communication with the FDA, potentially speeding up the pivotal study initiation scheduled for 2024. The Landmark study, evaluating the safety and tolerability of mRNA-3705 in patients with MMA, is currently in Phase 1/2.
- mRNA-3705 selected by U.S. FDA for START pilot program, enhancing its development focus.
- Increased FDA communication can accelerate pivotal study initiation for mRNA-3705 in 2024.
- mRNA-3705 selection emphasizes the potential of Moderna's mRNA platform beyond vaccines.
- START program supports development of treatments for unmet medical needs in rare diseases.
- FDA's selection highlights the innovative approach of mRNA-3705 in addressing serious MMA conditions.
- mRNA-3705 is still in early Phase 1/2 clinical trials, indicating a long path to market.
- Current standard of care for MMA includes only dietary and palliative measures, with no approved therapies, highlighting the burden of proof for mRNA-3705's efficacy.
- MMA is a rare disease, potentially limiting the market size and profitability of mRNA-3705.
Insights
Moderna's investigational therapeutic mRNA-3705 being selected for the FDA’s START pilot program is a significant development for both the company and the field of rare disease treatments. This program aims to expedite the development of therapies for conditions with high unmet needs. The selection of mRNA-3705, aimed at treating methylmalonic acidemia (MMA), underscores the FDA's recognition of the potential benefits of Moderna's mRNA technology beyond vaccines.
From a medical research perspective, this accelerates the path towards potential approval and availability of the treatment. mRNA-3705's accelerated development could result in faster access to a life-saving therapy for patients who currently have limited options, mainly dietary measures and transplants. This is particularly important for serious and life-threatening conditions like MMA, which have high mortality and morbidity rates.
The use of mRNA technology in treating MMA represents a novel approach that could pave the way for similar therapeutic applications. The FDA’s enhanced communication and support as part of the START program can significantly mitigate risks related to clinical trial design and execution, thereby increasing the likelihood of successful outcomes. This milestone-driven initiative also implies a more efficient path to pivotal clinical studies and ultimately, market approval.
This selection by the FDA for the START pilot program is a substantial boost for Moderna's stock potential. Firstly, it symbolizes a diversification of their mRNA platform, transitioning from a primary focus on vaccines to other therapeutics. The potential success of mRNA-3705 could demonstrate the versatility and robustness of their mRNA technology, positively influencing investor sentiment.
Moreover, the accelerated timeline for clinical development can lead to quicker commercialization if efficacy and safety are proven. This can translate into earlier revenue streams from a new product line, enhancing Moderna's financial outlook. The fact that this involves a rare disease with high unmet medical needs further adds value, as treatments for such conditions often attain higher market prices and favorable reimbursement scenarios.
However, investors should consider the inherent risks associated with biotech investments, particularly those in early-stage clinical trials. The success of mRNA-3705 is not guaranteed and the costs involved in its development are substantial. Despite these risks, the FDA’s selection under the START program provides a favorable risk-benefit profile for Moderna in the long term.
The endorsement of mRNA-3705 for the FDA’s START program provides a strategic market advantage for Moderna. By being one of only four treatments selected, it positions the company as a leader in the development of innovative solutions for rare diseases. This positions Moderna favorably within the biotechnology and pharmaceutical sectors, potentially leading to increased partnerships, collaborations and investor interest.
Given that there are currently no approved therapies for MMA, mRNA-3705 has the potential to be a first-in-class treatment, offering significant market exclusivity upon approval. This opens up a niche market that, despite its small patient population, can be highly lucrative due to the premium pricing of orphan drugs. The nature of the FDA's program also implies a more streamlined path to regulatory approval, reducing time-to-market and associated costs.
The strategic communication and collaboration with the FDA provided by the START program ensure that the development trajectory aligns closely with regulatory expectations, minimizing the risk of setbacks. This efficiency can provide a competitive edge in the marketplace, making Moderna a more attractive prospect for long-term investors.
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
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.
View the original press release on accesswire.com
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