Helixmith said that it has started its phase 3-2 trial for its gene therapy Engensis, also known as VM202-DPN, in treating diabetic peripheral neuropathy (DPN).
Since submitting the protocol for the trial to the U.S. Food and Drug Administration in March, Helixmith has been preparing plans for clinical site selection and clinical operation by selecting five sites to begin contract preparation, while also reinforcing its protocol.
With the FDA’s approval of the company's final protocol on Thursday, the company plans to confirm the safety and effectiveness of its drug in 152 U.S. patients at 15 medical institutions, including the Brigham and Women’s Hospital.
The company delayed the conclusion of its clinical phase 3-1b after reporting that there was a possibility of a mix between the placebo and Engensis treatment groups in the clinical phase 3-1 data in September last year.
In October, the company organized a research team in the U.S. and Korea and investigated the pharmacokinetic analysis anomalies in the clinical trial.
After completing its investigation, the company retracted its statement, saying that there had been no mix between the placebo and Engensis treatment groups. However, it admitted that the trial had failed to meet its primary endpoint.
While many industry watchers viewed the announcement as meaning the company had failed the trial, the company stressed that the trial's failure to meet the primary endpoint was not because of the lack of efficacy of the treatment but because of the specificity of the pain index and problems with a particular method of clinical operation.
The company claimed that in a phase 3-1B trial, supplemented with 101 patients, the treatment succeeded in achieving the goal in both the primary (safety) and secondary endpoint (effectiveness), reaffirming its plan to conduct phase 3-2 trials of the drug.
However, some industry experts remained skeptical about the further outcome of the most recent trial, as the company stopped short of adequately identifying the cause of the failure in its previous trial.
Helixmith said the company had put a lot of effort into making its trial work this time around, selecting top-notch contract research organization (CRO) firms to supervise the trial.
"The CRO that will oversee clinical operation is one of the top-five institutions worldwide," the company said. "Also, we have ushered in a new CEO specializing in the field of pain."
By selecting excellent CROs, the company said it has made every effort to ensure that the data related to clinical trials will be managed better than before.
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