HELIXMITH

Helixmith Submits Engensis DPN Phase 3 Protocol to US FDA

작성자 helixmith 작성일 20-10-12 10:29

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Helixmith Co., Ltd. announced on September 16th that the company has submitted VMDN 003-3 protocol for diabetic peripheral neuropathy (DPN) using its flagship gene therapy Engensis (VM202) to the US Food and Drug Administration (FDA).

The DPN phase 3 study, titled REGAiN-2A, aims to verify the efficacy of Engensis. The trial’s design is very similar to the recently launched REGAiN-1A DPN phase 3 trial. REGAiN-2A will be a 12-month study to collect data on long-term pain-relieving effects and safety.

The primary endpoint of the study is the change in the Average Daily Pain Score (ADPS) from the Brief Pain Inventory for DPN (BPI-DPN) from 7 days prior to the first injection visit to 7 days prior to the 6-month visit for Engensis compared to placebo. Secondary endpoints include: (1) proportion of patients with 50% reduction in the ADPS or more at 6, 9, and 12 months; (2) change in ADPS from the full BPI-DPN from 7 days prior to baseline compared to 7 days prior to the 9- and 12-month visits; and (3) change from baseline in the worst pain score from the BPI-DPN at 6, 9, and 12 months for Engensis compared to placebo.

REGAiN-2A is targeted at DPN patients not on gabapentinoids, such as Pregabalin and Gabapentin, which is the same patient population in REGAiN-1A. Also similar to REGAiN-1A, REGAiN-2A has an initial target enrollment of 152 and the maximum sample size is 250 participants based on an adaptive design analysis. The study will take place at 15 sites across the US.

Dr. Sunyoung Kim, CEO of Helixmith, commented, "The existing products indicated for painful DPN only relieve pain and do not target the disease itself. The current products are also often accompanied by serious side effects. Engensis for painful DPN is designed to target the root causes of neuropathy rather than the symptoms. We will do our best to ensure the successful completion of our two phase 3 trials.”


About DPN
Diabetic peripheral neuropathy (DPN) is one of the most common complications of diabetes. Of approximately 30 million US adults with diabetes, up to 50% may develop DPN, and disabling neuropathic pain (painful DPN or PDPN) may affect approximately 25% of diabetic patients (Shillo et al., 2019). Painful DPN typically manifests as burning or stabbing pain in the lower extremities. Currently approved medications for PDPN are palliative, limited to managing existing pain, and have the potential for serious side effects.

About Engensis
Helixmith's Engensis (VM202) is a gene therapy based on plasmid DNA. To date, more than 500 patients have been treated with Engensis across nine clinical trials in six different diseases and conditions. Data from previous clinical studies suggest that Engensis is well tolerated and has the potential to provide durable analgesic and/or symptomatic relief in a variety of disease settings. Beyond potentially alleviating pain, Engensis is designed to target the root underlying causes of neuropathy through its predicted angiogenic and neuroregenerative properties. The US FDA recognized the potential for Engensis to meet the unmet need for diabetic peripheral neuropathy in 2018 by designating it as a Regenerative Medicine Advanced Therapy (RMAT), making it the first RMAT-designated gene therapy for a prevalent disease with over one million patients. This designation grants all the benefits afforded by the fast track and breakthrough designations, including priority review, to Engensis.

About Helixmith
Helixmith is a gene therapy company headquartered in Seoul, Korea, developing new and innovative biopharmaceuticals to tackle previously untreated diseases, and is listed on KOSDAQ. The company has an extensive gene therapy pipeline, including a CAR-T program targeting several different types of solid tumors and an AAV vector program targeting neuromuscular diseases. Engensis (VM202), a the mostre advanced pipeline candidate, is a plasmid DNA therapy being studied for DPN, diabetic foodt ulcers, claudication, amyotrophic lateral sclerosis (Phase 2 beginning in late 2020), coronary artery disease and Charcot-Marie-Tooth disease.
Helixmith clinical development and manufacturing activities are based in San Diego, California, where the company co-owns a cGMP-ready DNA production facility, Genopis, Inc., an affiliated CDMO also in San Diego. Genopis serves both Helixmith and external customers in need of plasmid DNA for medical purposes.