Moderna Reviews Clinical Trial Programs Across Portfolio at 2022 R&D Day
Interim data from Phase 1/2 propionic acidemia (PA) multi-dose Paramount trial shows mRNA-3927 was well-tolerated to date, with encouraging early signs of potential for clinical benefit
Interim data from Phase 1/2 glycogen storage disease 1a (GSD1a) single-dose Ba1ance trial shows mRNA-3745 was well tolerated to date, with encouraging early signs of potential for clinical benefit
Moderna announces a new development candidate, mRNA-3139, for ornithine transcarbamylase (OTC) deficiency, a rare genetic disorder
Phase 3 clinical trial of RSV vaccine, mRNA-1345, has enrolled more than 24,000 of the 34,000-participant target
Phase 3 immunogenicity and safety study of flu vaccine, mRNA-1010, is fully enrolled; Company to pursue accelerated approval pathway in 2023
Company is on track to report data from its Phase 2 personalized cancer vaccine (PCV) study in 4Q 2022
CAMBRIDGE, MA / ACCESSWIRE / September 8, 2022 / Moderna, Inc. (Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced advances across its portfolio of mRNA programs presented at the Company's annual R&D Day.
"We continue to make significant progress in accelerating our pipeline. Our Phase 3 flu and RSV clinical trials are rapidly advancing toward completion. We are seeing early clinical benefits in two rare disease programs and we expect to report on data from our personalized cancer vaccine trial later this year," said Stéphane Bancel, Chief Executive Officer of Moderna. "As a reflection of the Company's growth and progress, we are preparing for multiple product launches globally."
Updates and recent progress include:
Rare Diseases: Proof-of-Concept Studies
Propionic Acidemia: Propionic acidemia (PA) is a rare and severe metabolic disorder. PA is characterized by a deficiency of propionyl-CoA carboxylase, an enzyme involved in the breakdown of proteins, certain fatty acids, and other substances. This deficiency leads to the build-up of toxic compounds that can cause serious health problems like repeating episodes of life-threatening metabolic crises and damage to the brain, nervous system, and heart. There is no approved pharmacologic therapy for PA.
Glycogen Storage Disease 1a: Glycogen Storage Disease 1a (GSD1a) is a rare, inherited metabolic disorder. GSD1a is caused by a deficiency of an enzyme called glucose 6-phosphatase (G6Pase-α). G6Pase-α is critical for the release of glucose in the blood, and is needed to maintain normal blood sugar levels. Patients with GSD1a may have life-threatening events when their blood sugar levels are too low. They may also experience long-term liver complications, possibly including cancer. There are no approved pharmacologic therapies for GSD1a.
Methylmalonic acidemia: PA and methylmalonic acidemia (MMA) share similar biology and disease pathology (deficiency of an enzyme in the propionate pathway) with subsequent accumulation of toxic metabolites. MMA is a rare genetic disease with significant morbidity and mortality caused by a deficiency in an enzyme called methylmalonyl-CoA mutase (MUT). MMA mostly affects children, and there is no approved pharmacologic therapy. Long-term complications of MMA can include problems with neurocognitive function, slower growth, and heart, kidney, and blood problems.
Rare Disease: New Development Candidate
Leveraging data and learnings from the rare disease program, Moderna announces a new development candidate, mRNA-3139, for ornithine transcarbamylase (OTC) deficiency. mRNA-3139 uses the same lipid nanoparticles (LNP) as in Moderna's GSD1a program and is the Company's sixth rare disease candidate.
OTC deficiency is a rare genetic condition characterized by complete or partial lack of the OTC enzyme, resulting in ammonia build-up in the blood. The clinical presentation of OTC deficiency is very heterogeneous, with more severe symptoms including seizures, enlarged liver, and respiratory difficulty. For more severe or non-responsive cases that progress towards liver failure, treatment may include liver transplantation.
Late-Stage Respiratory Vaccines
Seasonal Influenza Vaccine Program: Influenza, which occurs seasonally, causes respiratory illnesses and places a substantial burden on healthcare systems. Worldwide, the influenza virus leads to 3-5 million cases of severe illness and 290,000-650,000 influenza-related respiratory deaths annually. About 8% of the US population experiences symptoms from influenza each year, with 140,000-710,000 hospitalizations and 12,000-52,000 deaths per year.
Respiratory Syncytial Virus Vaccine Program: Respiratory syncytial virus (RSV) causes a substantial disease burden in older adults aged 65 years and older. There are approximately 177,000 RSV-related hospitalizations in adults 65 and older in the U.S. each year and approximately 14,000 RSV-related deaths. Globally, there are more than 1.5 million episodes of acute respiratory tract infection related to RSV each year.
Combination Respiratory Vaccines
In addition to single-agent vaccines, the Company is progressing several combination respiratory vaccines, including a fully enrolled Phase 1/2 trial testing mRNA-1073 targeting SARS-CoV-2 and influenza. Clinical trials for mRNA-1230, targeting SARS-CoV-2, influenza, and RSV are expected to be initiated this year.
Late-Stage Latent Vaccine: CMV Trials
Cytomegalovirus Vaccine Program: Cytomegalovirus (CMV), a type of latent herpes virus, is the most common cause of infection that occurs before birth worldwide and is responsible for more than $1 billion in annual healthcare costs. CMV can cause long-term health problems in infected infants, such as hearing loss, vision impairment, cerebral palsy, learning disabilities, and decreased muscle strength and coordination.
Personalized Cancer Vaccine
In over 10 years since its 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 in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows 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 pursuit of 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-19 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 seven years. To learn more, visit www.modernatx.com.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: clinical trends in Moderna's Phase 1/2 PA trial; Moderna's pursuit of an accelerated approval pathway for mRNA-1010 and the timing of potential approval; the timing of data from Moderna's trials of its product candidates targeting PCV, MMA,GSD1a and RSV; the initiation of clinical trials for mRNA-1230, targeting SARS-CoV-2, influenza and RSV; Moderna's commercial priorities; the timing of future product launches; Moderna's manufacturing capabilities; and the timing of delivery of COVID-19 boosters. 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, 2021 and Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2022, each 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.
08/09/2022 Dissemination of a Financial Press Release, transmitted by EQS Group.