Helixmith submitted an initial protocol to the US FDA in March and has since pursued a detailed clinical site selection and operations program to prepare for the upcoming trial. Despite the COVID-19 pandemic, the company was able to complete selection of the first clinical sites, prepare key agreements related to the trial, and augment the protocol. The final protocol was submitted to the FDA on June 25, initiating the trial.
The clinical trial plan for REGAiN-1A has been registered at ClinicalTrials.gov, a site run by the US National Institutes of Health (NIH) to provide clinical information to patients in the US (https://www.clinicaltrials.gov/ct2/show/NCT04469270?term=NCT04469270&draw=2&rank=1).
REGAiN-1A has an initial target enrollment of 152 DPN patients at approximately 15 clinical sites across the US. The objective of the trial is to demonstrate the safety and efficacy of Engensis.
The primary efficacy endpoint compares the change in average daily pain scores from the 7 days prior to the first injection on Day 0 to the 7 days prior to the six-month visit in participants treated with Engensis versus placebo. Secondary endpoints include the statistical significance of (1) reduction in worst pain at the end of a six-month period versus placebo, (2) the ratio of patients with an average pain reduction of 50% or more at the end of the six-month period versus placebo, and (3) safety of Engensis versus placebo.
Helixmith carefully evaluated more than 100 potential clinical sites in the US and sought out high-performing commercial research sites and academic sites with expertise in DPN treatment. A major CRO with expertise in pain medicine will oversee clinical operations. In addition, Helixmith has engaged a clinical research consulting company that will provide periodic online expertise in identifying and mitigating random and systematic errors that may negatively impact clinical trial outcomes. Alongside the selection of top clinical sites and external clinical research partners, Helixmith has bolstered its internal clinical team to ensure high-quality clinical trial management.
Dr. Sunyoung Kim, CEO and Head of Global Clinical Development at Helixmith, commented, "We will do our utmost for this newly launched trial to expedite the commercialization of Engensis and maximize its value."
Helixmith's Engensis(VM202) is a gene therapy based on plasmid DNA. Beyond alleviating pain, Engensis is designed to target the root causes of neuropathy through its predicted angiogenic and neuroregenerative properties. The US FDA recognized the potential for Engensis to meet the unmet need for this condition 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.
The RMAT designation is a recent addition to the collection of programs mandated by the US Congress to accelerate the development and approval of novel regenerative therapies. The designation enables frequent discussions with the FDA on important issues in the development of innovative drugs. This recent addition to the agency’s expedited development programs grants all of the benefits afforded by the fast track and breakthrough designations, including priority review for Engensis.
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 patients with diabetes(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.