LA JOLLA, Calif., September 6, 2022 /PRNewswire/ — Helixmith today announced top results from a Phase 2A study in people with ALS (amyotrophic lateral sclerosis). ALS is a fatal neurodegenerative disease of the upper and lower motor neurons that causes progressive paralysis and eventually death from respiratory failure. This Phase 2A was a multicenter, double-blind, placebo-controlled study involving 5 sites (4 in the US and 1 in Korea) and 18 subjects randomized at a 2:1 Engensis/placebo ratio. In this Phase 2A study, three treatments of Engensis, a plasmid DNA encoding human HGF, or a placebo were injected into the upper and lower extremities at months 0, 2, and 4. One treatment consisted of two cycles of injections, at 2 week intervals. , 64 mg of Engensis or placebo in total. Therefore, a total of 192 mg of Engensis was administered to each subject over a period of 4 months. Additionally, there was a 6-month follow-up period after the first injection on day 0.
Study results demonstrated that Engensis was safe and well tolerated at this dosage regimen. There was no difference in the frequency of TEAEs (83% for each group) between the Engensis and placebo groups. One TEAE, bronchitis, was reported in the Engensis group, but was determined to be unrelated to the study drug. Injection site reactions were reported by 50% of the Engensis group and 66.7% of the placebo group. Most injection site reactions were grade 1 or 2 and resolved within a short time, and none of the study participants discontinued due to the number of injections. These data suggest that repeated high-dose treatments of Engensis were safe and well tolerated, providing great flexibility in designing dosing regimens for future clinical studies.
Since the primary endpoint of this study was to test safety and tolerability, efficacy was only measured as an exploratory endpoint. ALSFRS-R scores, muscle functions using portable dynamometry and ALSAQ-40 were among the measures collected. Because the study size was small and 4 subjects dropped out early, we were unable to compare efficacy between the Engensis and placebo groups.
An important part of this study was the collection of biopsy samples from participants’ injection sites. Engensis is a gene therapy administered intramuscularly. Most of the data collected to date, showing the effects of HGF expressed from Engensis, comes from animal models. Data collected from clinical trials have been limited so far. Thanks to our dedicated clinical trial participants, muscle biopsy samples were collected during the trial and will be subjected to histological and molecular biology analyzes using RNA-Seq.
Helixmith greatly appreciates the generous and enthusiastic participation of ALS patients. Data from these results should provide valuable insights into understanding the mechanisms of action of Engensis and its effects on human gene expression, which will greatly aid in the development of innovative drugs based on HGF/c- signaling. Met.
Helixmith will continue analyzing the Phase 2A data upon receipt of the full report and plans to present the results at a future conference and determine next steps for Engensis in ALS at that time.
Helixmith is a clinical-stage gene therapy company headquartered in Seoul, Korea, developing new and innovative biopharmaceuticals to treat previously untreated diseases, and is listed on KOSDAQ. The company has an extensive portfolio of gene therapies, including a CAR-T program targeting several types of solid tumors and an AAV vector program targeting neuromuscular diseases. Engensis (VM202), the most advanced pipeline candidate, is a plasmid DNA therapy studied for painful diabetic peripheral neuropathy, diabetic foot ulcers, claudication, amyotrophic lateral sclerosis, coronary artery disease and Charcot-Marie disease -Tooth.
SOURCE Helixsmith UNITED STATES Inc.