CHICAGO, Aug. 19,
2024 /PRNewswire/ -- RNA Therapeutics, a
clinical-stage biotechnology company focused on innovative
applications of mRNA-based delivery systems for therapeutic
proteins and vaccines, announced that it has received a written
response to its Pre-Investigational New Drug Application (pre-IND)
questions submitted to the U.S. Food and Drug Administration (FDA)
regarding the development of its lead asset, RNAT-89 (BLA-761423),
an mRNA LNP formulation to express darbepoetin, as the first such
product presented to the FDA to seek advice on the naming of the
product, nonclinical and clinical testing and comparison with
Aranesp (Amgen) indicated for the treatment of anemia due to
chronic kidney disease (CKD) and non-myeloid malignancies where
anemia is due to the effect of concomitant myelosuppressive
chemotherapy. There is no biosimilar to Aranesp in the US. The
global market for erythropoietin (EPO) drugs, including
darbepoetin, was valued at $9,734.1
million in 2022 and is projected to reach $16,926.21 million by 2032, with a compound
annual growth rate (CAGR) of 5.7%. The current treatment cost with
darbepoetin is about $1,000, which
will be reduced to less than $50,
making it affordable globally.
The pre-IND meeting with the FDA clarified several issues, the
first being how the product would be named. There was no history of
naming such a product, and the FDA agreed with RNA Therapeutics'
suggestion to call it "Darbepoetin mRNA (xxxx) Injection." The FDA
has not approved any therapeutic protein product delivered by mRNA
technology, so this naming plan is significant information. It
should be noted that this naming will apply only if the mRNA
expresses the same protein, not its scFV or conjugated form for the
mRNA therapy developers. The FDA also agreed with RNA Therapeutics
composition, release specification, and nonclinical and clinical
testing protocols substantially reduced compared to a new BLA, even
though RNAT-89 is not considered a biosimilar.
"The completion of the pre-IND discussion with the FDA is a
significant milestone for RNA Therapeutics, and we believe it
validates our development strategy for RNAT-89," said Dr.
Sarfaraz K. Niazi, CEO of RNA
Therapeutics. "This the first mRNA-based therapeutic protein filed
with the FDA, and given its low cost of goods, we will be able to
sell our product at a fraction of the cost of any product
manufactured by the traditional recombinant process," said Dr.
Matthias Magoola, Chairman of RNA
Therapeutics. "This is also a revolution for biological drugs that
we project will be delivered through the body's bioreactor, the
ribosomes, making them safer and more effective, and at a minute
fraction of the cost of biological drugs that remain unaffordable
to 80% of patients worldwide; all this will go away once RNA
Therapeutics demonstrates this design that we hope is followed by
many," added Dr. Magoola.
About RNA Therapeutics
RNA Therapeutics is a clinical-stage biotechnology company
developing novel treatments and preventions using mRNA-based
delivery of therapeutic proteins and vaccines. Its pipeline
includes vaccines against Alzheimer's disease, diabetes type I, HIV
vaccine against active and latent viruses, antibody-transferrin
conjugates for neurodegenerative disorders, and cancer treatment.
RNA Therapeutics has over 100 US Patents on its technology. RNA
Therapeutics is fully funded to take all its products to the IND
approval stage when it licenses out to major pharmaceutical
companies.
For more information, please
visit https://www.therarna.com
Contact Information
Sarfaraz K. Niazi, Ph.D.
niazi@therarna.com
View original content to download
multimedia:https://www.prnewswire.com/news-releases/rna-therapeutics-receives-feedback-from-fda-for-the-first-in-class-mrna-based-delivery-of-therapeutic-protein-promising-affordable-biological-drugs-302224854.html
SOURCE RNA Therapeutics, Inc.