Penetrium Bio announced that it has received approval from the U.S. Food and Drug Administration (FDA) for its Phase 2a Investigational New Drug (IND) application to evaluate the efficacy and safety of 'Penetrium' in combination with standard therapy in patients with relapsed or refractory progressive solid tumors.

▶ Trial at 3 US institutions with 18 subjects... open-label, randomized, multicenter study

This trial is a Phase 2a open-label, randomized, multicenter clinical trial conducted in patients with relapsed or refractory progressive solid tumors. It will be conducted at three institutions in the United States, with a target enrollment of 18 subjects. The IND registration number is 183600.

The primary objective of the trial is to evaluate the antitumor activity of Penetrium in combination with the standard of care (SoC) by tumor type. Secondary objectives include additional evaluation of clinical efficacy, assessment of the safety and tolerability of the Penetrium-plus-SoC combination regimen, and characterization of the pharmacokinetic properties of Penetrium and its metabolites when administered in combination.

▶ Primary endpoint: objective response rate (ORR)... descriptive statistical analysis without hypothesis testing

The primary endpoint is the objective response rate (ORR), defined as the proportion of patients achieving confirmed complete response (CR) or partial response (PR) according to RECIST v1.1. ORR will be evaluated within each cohort by tumor type.

The analysis will be conducted on the mITT population, presenting the ORR and two-sided 95% confidence intervals for each treatment group. No formal hypothesis testing will be performed; all analyses will be conducted using descriptive statistics.

The trial duration is approximately 24 months from the date of IND approval and may change depending on the pace of subject enrollment. The expected completion date is September 3, 2028.

▶ Receipt of 'Study May Proceed Letter'... approval probability noted at about 10%

The company stated that it confirmed the approval on September 3 (Korea time) upon receiving the FDA's 'Study May Proceed Letter' via email from its contract research organization (CRO).

Additionally, in its regulatory filing, the company noted as an investor caution that the probability of the investigational drug ultimately receiving approval as a medicine is statistically known to be around 10%, and that the clinical trial and product approval process may yield results that fall short of expectations. It added that it may therefore modify or abandon its commercialization plans.