On August 11, 2026 Gibson Oncology, a clinical-stage oncology company developing blood-brain-barrier-penetrant therapies for brain tumors and other difficult-to-treat cancers, reported that the first patient in the Phase II clinical trial of LMP744 has been enrolled and has completed the initial treatment cycle without complication. The trial is being conducted by investigators at the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health, and is evaluating LMP744 as a single agent in patients with recurrent glioblastoma (GBM). Gibson Oncology is providing partial funding and LMP744 to the trial under a Clinical Cooperative Research and Development Agreement (CRADA) with NINDS.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
LMP744 is a novel, dual-acting anticancer agent that works by reducing overexpression of the cMyc oncogene and by inhibiting topoisomerase 1 (TOPO1), two validated targets across a range of solid and hematological cancers.
Glioblastoma is the most common and aggressive primary brain tumor in adults. Approximately 13,000 new cases are diagnosed in the United States each year, median survival is generally 15 to 18 months, and the five-year survival rate is under 10 percent. Recurrent glioblastoma, for which there is no established standard of care, carries an especially poor prognosis.
In preclinical studies, LMP744 crossed the blood-brain barrier and achieved brain concentrations approximately 10 times the level needed to kill cancer cells, with a half-life in the brain exceeding 24 hours for each dose. Blood-brain-barrier penetration has long been one of the central obstacles in the treatment of brain tumors.
The Phase II trial is designed to enroll a total of 40 first-time recurrent GBM patients with a Karnofsky Performance Status (KPS) of 60 or better, with a primary endpoint of progression-free survival and a secondary endpoint of overall survival. LMP744 is being studied as a single therapeutic agent based on the strong anticancer effects observed in GBM patient-derived xenograft (PDX) animal models.
LMP744 has previously completed one Phase I clinical trial with 36 patients in heavily pretreated cancer patients, many of whom had failed as many as eight prior individual or combination treatments. Over 30% of the patients on single agent treatment with LMP744 experienced stable disease for up to 30 months and 2 patients were partial responders, even as many patients were sub-optimally dosed to establish the drug’s safety and tolerability before reaching the therapeutic dose level of 190 mg/m2.
"Completing the first treatment cycle in this trial is an important early milestone for LMP744 and, most of all, for the patients who face recurrent glioblastoma with limited therapeutic options, and one of the most difficult cancers in medicine," said Randall Riggs, President and Chief Executive Officer of Gibson Oncology. "We are grateful to the clinical team at NINDS and to the patients and families taking part in this study, and we look forward to advancing LMP744 as a potential breakthrough treatment in an area that has experienced little improvement in treatment options for decades."
Mr. Riggs continued, "The renewed excitement about LMP744 is two-fold. First, LMP744 was found to migrate rapidly into the brain at high concentrations, well beyond the levels needed to kill cancer, with each dose having a half-life of over 24 hours. Second, in PDX animal models implanted with patient-derived recurrent gliomas, just five doses of LMP744 produced tumor eradication. Together, these findings made a compelling case for initiating this Phase II study at the NINDS."
About LMP744
LMP744 is a novel, dual-acting indenoisoquinoline that reduces cMyc overexpression and inhibits topoisomerase 1 (TOPO1). In preclinical studies, it has demonstrated blood-brain-barrier penetration and sustained brain exposure. LMP744 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for all gliomas, including DIPG.
(Press release, Gibson Oncology, AUG 11, 2026, https://www.globenewswire.com/news-release/2026/08/11/3342662/0/en/gibson-oncology-announces-first-patient-completes-initial-treatment-cycle-in-phase-ii-trial-of-lmp744-for-recurrent-glioblastoma.html [SID1234669976])