- 100% of participants (10 of 10) with PH1
- 67% of participants (2 of 3) with PH2
- 70% of participants (7 of 10) with PH1
- 33% of participants (1 of 3) with PH2
Walter Klemp, Chairman and CEO of Moleculin, stated, “The trial in adults has been important in leading the way to establishing a safe and tolerable human dose level for what we believe is a first-in-class compound that crosses the blood-brain barrier and is being developed for the treatment of central nervous system malignancies. In animal models, WP1066 has been shown to stimulate immune responses that successfully modulate oncogenic transcriptional activity in tumor cells and repress their ability to drive tumor growth.”
According to Mr. Klemp: “In addition to providing valuable pharmacokinetic data, this trial also creates the foundation for future studies in WP1066, including another investigator-initiated study, which is currently being proposed to examine the combination of WP1066 with radiation for the treatment of GBM. This next clinical trial is being proposed as a result of recent discoveries presented in a peer-reviewed article published in Clinical Cancer Research (Clin Cancer Res June 30 2020 DOI:10.1158/1078-0432.CCR-19-4092), which reported findings that Moleculin’s STAT3 inhibitor, WP1066, used in combination with traditional whole brain radiation therapy, resulted in long-term survivors and enhanced median survival time relative to monotherapy in mice with implanted human brain tumors.”
The GEM-3 trial is a randomized, double-blind, intra patient placebo-controlled multicenter study designed to evaluate the efficacy and safety of B-VEC for patients suffering from both recessive and dominant forms of dystrophic epidermolysis bullosa. The trial aims to enroll approximately thirty (30) participants with DEB, aged 6 months or older at time of consent. Investigator identified wound pairs, up to three in each patient, will be treated once weekly for six months with either B-VEC or placebo.
The Primary Outcome Measure is complete wound healing determined by the Investigator, as compared to baseline in B-VEC treated wounds versus placebo treated at Weeks 20, 22 and 24.
Secondary endpoints to be evaluated in the study include complete wound healing at Weeks 8, 10 and 12; the mean change in pain severity (using either a VAS or FLACC-R Scale) per primary wound site associated with wound dressing; the proportion of primary wound sites with ≥75% healing assessed via Canfield photography. Additional exploratory measures include relative time to wound closure from baseline, duration of wound closure, mean reduction in wound surface area in B-VEC treated versus placebo treated wounds, mean change in Quality of Life in addition to Skindex score as compared to baseline at Week 24. Throughout the study, participants will complete questionnaires, have images captured of their study wounds, undergo physical exams, have vital signs and safety labs monitored.
The clinical trial INternational Study of Phase 3 IV RigosErtib, or INSPIRE, was finalized following guidance received from the U.S. FDA and European Medicines Agency (EMA). INSPIRE is a global, multi-center, randomized, controlled study to assess the efficacy and safety of IV rigosertib in higher-risk MDS (HR-MDS) patients who had progressed on, failed to respond to, or relapsed after previous treatment with a hypomethylating agent (HMA) within nine cycles over the course of one year after initiation of HMA treatment. This time frame optimizes the opportunity to respond to treatment with an HMA prior to declaring treatment failure, as per NCCN Guidelines. Patients are randomized at a 2:1 ratio into two study arms: IV rigosertib plus Best Supportive Care versus Physician’s Choice plus Best Supportive Care. The primary endpoint of INSPIRE is overall survival. The trial continued beyond the pre-specified interim analysis and is nearing its conclusion.
Patents covering oral and injectable rigosertib have been issued in the US and are expected to provide coverage until at least 2037.