Atlanta, GA, May 23, 2018 – On May 21 2018, the US Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to VM202 (donaperminogene seltoplasmid), a novel plasmid gene therapy by ViroMed Co., Ltd. dba VM BioPharma, a Korean biologics developer. It is the first time RMAT designation has been granted to a gene therapy targeting a prevalent disease with a large market potential considering, thus far, most of the RMAT grantees have been new drug candidates targeting life-threatening cancers, injury–induced indications, or hereditary illnesses.
“This is incredibly exciting news for patients with painful diabetic peripheral neuropathy,” said Professor Sunyoung Kim, DPhil, Chief Scientific Officer and Founder. “This designation, VM202’s innovative and pleiotropic mechanism of action as well as the positive safety and efficacy results to date, affirms the work of ViroMed scientists and clinical researchers within the United States who are passionate about bringing a new and novel breakthrough to millions of individuals around the world struggling daily with this serious and painful condition," said Dr. Jack A. Kessler, Professor of Neurology and Pharmacology, Feinberg School of Medicine, Northwestern University and lead investigator of VM202 Painful Diabetic Peripheral Neuropathy (PDPN) clinical development program.
The designation recognizes VM202’s potential as a regenerative medicine, differentiating it from currently available analgesics. With RMAT designation, the company will get to closely work with the FDA for guidance on generating the evidence needed to support approval of the drug in an efficient manner.
A VM BioPharma representative commented, “In its letter, the FDA determined that VM202 for the treatment of painful diabetic peripheral neuropathy meets the criteria for RMAT. This implies that, looking at our clinical data, the FDA recognizes VM202’s potential as a regenerative medicine for PDPN to meet unmet medical needs of patients.”
“To the best of our knowledge, ViroMed’s VM202 is both the only non-viral gene therapy and the only gene therapy for prevalent disease to receive the RMAT designation from the FDA and potentially the only regenerative medicine product for a pain indication,” said Dr. William Schmidt, Vice President of Global Clinical Development. He added, “If approved by the FDA when our clinical trial program has been completed, the potential benefits of this to patients, to their families, and to physicians, who treat this terrible progressive disease, cannot be underestimated. Our Phase 1 and Phase 2 data show that subjects with painful diabetic peripheral neuropathy may achieve substantial decreases in foot pain and improvement in normal sensory function that has an onset within only a few weeks after initial in-office treatment with VM202. In addition, data from Phase II studies suggest that VM202 provides functional improvements that could potentially last 9 months or longer after the first series of injections spaced two weeks apart.”
VM202 (donaperminogene seltoplasmid) is a first-in-class proprietary non-viral regenerative plasmid DNA gene therapy. VM202 is a novel genomic cDNA hybrid human hepatocyte growth factor (HGF) gene with a novel and proprietary coding sequence (HGF-X7) expressing two isoforms of HGF, HGF728 and HGF723- a configuration that closely mimics endogenous HGF productions that is needed for optimal therapeutic benefits. The key feature of the proprietary HGF-X7 is that it was designed by inserting part of intron sequences into certain sites of HGF cDNA so that both isoforms of HGF protein are expressed simultaneously and efficiently as in the human genome. Because there is no change in the coding region of the HGF gene, HGF proteins generated from VM202 are identical to wild-type human HGF proteins, a structure like that produced endogenously within a person’s own body.
When introduced into the body, VM202 produces hepatocyte growth factor (HGF) protein locally, which can potentially induce the formation of new blood vessels, modify levels of select pain mediators, support regeneration/repair of damaged peripheral nerves and ameliorate atrophic condition of skeletal muscle.
About VM BioPharma and ViroMed Co., Ltd.
VM BioPharma is the US division of ViroMed Co., Ltd., an R&D focused biopharmaceutical company based in Seoul, Korea, developing new and innovative biopharmaceuticals to meet unmet medical needs. Currently, the company is actively focusing on developing the proprietary plasmid DNA-based drug VM202 at various clinical stages in the U.S., Korea, and China, for cardiovascular and neurological diseases, including but not limited to, PDPN, Diabetic Foot Ulcers, amyotrophic lateral sclerosis (ALS) and ischemic heart disease (IHD).
Note: ViroMed Co., Ltd. is not in any way associated with ViroMed Laboratories.
This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions. Forward-looking statements are based on management's current plans, estimates, assumptions and projections, and speak only as of the date they are made. ViroMed undertakes no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond the control of the company. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are made public in more detail through the company’s corporate public announcement in compliance with the Financial Investment Services and Capital Markets Act of the Republic of Korea.