FDA Feedback on First-in-Class mRNA Therapeutic Protein Delivery

CHICAGO, Aug. 19, 2024 — RNA Therapeutics, a biotechnology company specializing in mRNA-based therapeutic delivery systems, announced the FDA's response to its Pre-Investigational New Drug Application (pre-IND) for its lead candidate, RNAT-89 (BLA-761423). This innovative product is a lipid nanoparticle (LNP) formulation designed to express darbepoetin, intended to treat anemia caused by chronic kidney disease (CKD) and chemotherapy-related non-myeloid malignancies. Notably, this is the first instance of the FDA providing guidance on the naming, nonclinical, and clinical testing of such a product.

The global market value for erythropoietin (EPO) drugs, including darbepoetin, was $9,734.1 million in 2022 and is expected to reach $16,926.21 million by 2032, growing at a CAGR of 5.7%. Current darbepoetin treatments cost about $1,000, but RNA Therapeutics aims to bring this down to less than $50, enhancing affordability worldwide.

FDA Pre-IND Meeting Insights

In the pre-IND meeting, the FDA and RNA Therapeutics addressed the unique challenge of naming the mRNA-delivered therapeutic protein. As there was no precedent, the FDA accepted the proposed name "Darbepoetin mRNA (xxxx) Injection." This naming convention is crucial as it sets the precedent for future mRNA protein therapies, clarifying that it applies if the mRNA expresses the same protein, not its scFV or conjugated forms.

The FDA also approved RNA Therapeutics' composition, release specification, and testing protocols. These protocols are significantly streamlined compared to a new Biologics License Application (BLA), even though RNAT-89 does not qualify as a biosimilar.

Significance for RNA Therapeutics

Dr. Sarfaraz K. Niazi, CEO of RNA Therapeutics, emphasized the importance of the pre-IND milestone, stating it validates their development strategy for RNAT-89. Dr. Matthias Magoola, Chairman of RNA Therapeutics, highlighted the potential economic impact, noting that their product can be sold at a fraction of the cost of traditional recombinant therapies. This cost-efficiency, he argued, could revolutionize the market for biological drugs by making them more accessible.

Dr. Magoola further elaborated on the broader implications of their technology, suggesting it could transform how biological drugs are delivered. By leveraging the body's ribosomes as bioreactors, their approach aims to enhance safety and efficacy while drastically reducing costs. This could make essential biological drugs affordable for the 80% of patients who currently cannot access them.

About RNA Therapeutics

RNA Therapeutics is a clinical-stage biotechnology firm developing treatments and vaccines using mRNA-based delivery systems. Their pipeline includes vaccines for Alzheimer's disease, type I diabetes, and HIV, as well as antibody-transferrin conjugates for neurodegenerative disorders and cancer treatments. The company holds over 100 US patents and is funded to advance its products to the IND approval stage, after which it plans to license them to major pharmaceutical companies.

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