Gibson Oncology, NIH to begin Phase 2 trials of LMP744 for treatment of first-time recurrent glioblastoma

WEST LAFAYETTE, Ind. and MIAMI — Gibson Oncology, a clinical-stage private pharmaceutical company headquartered in Miami, announced it has entered Phase 2 clinical trials with LMP744 for the treatment of first-time recurrent glioblastoma patients.

The company is focused on advancing its novel small molecules with dual-action inhibition of topoisomerase 1 and reduction of overexpression of the cMYC oncogene.

 “The primary endpoint of the study is to evaluate tumor regression in patients with recurrent glioblastoma. Secondary endpoints will evaluate parameters such as progression-free survival, biological changes in glioblastoma tissues obtained pre- and post-treatment, self-reported quality of life and overall survival,” Gibson Oncology CEO Randy Riggs said.

During the Phase 2 trial, approximately 40 first-time recurrent glioblastoma patients will receive a once-a-day, one-hour infusion of LMP744 for five consecutive days, with biological analyses conducted on brain tissues obtained before and after this initial treatment period.

If biological results are favorable, treatment will continue for 12 cycles, with each cycle consisting of five consecutive days of treatment followed by 23 days of no treatment.

About glioblastoma and LMP744

Glioblastomas are malignant tumors that develop in the brain or spinal cord.

• Tumors grow rapidly and invade nearby tissue.
• Standard treatment includes surgery, radiation and chemotherapy.
• Life expectancy after diagnosis has been approximately 15-18 months for the past three decades.
• Recurrence of glioblastoma in these patients is likely with life expectancy of approximately six to nine months with limited treatment options.  

Upon receiving Food and Drug Administration approval of the Phase 2 protocol, the National Institutes of Health approved the Phase 2 trials following the successful completion of the Phase 1 study of LMP744. In two Phase 1 human trials involving over 40 heavily pretreated, advanced-stage cancer patients, LMP744 proved to be well tolerated and resulted in two patients whose tumors decreased at least 30% in size and 35% of patients whose tumors did not increase in size for a period of time lasting up to 18 months.

Gibson Oncology is also evaluating its clinical-stage compound LMP400 in collaboration with Dr. Matthew Waitkus at Duke University for the potential treatment of high-grade gliomas that are resistant to other therapies. LMP400 is an analogue of LMP744 with less susceptibility to being pumped out of the cancer cell, thus overcoming a potential drug resistance mechanism. LMP400 has also demonstrated dual action against topoisomerase 1 and cMYC overexpression.

Gibson Oncology has extensive intellectual property and orphan drug designations on both LMP744 and LMP400 and pediatric designation on LMP400.

Purdue University genesis of LMP744 and LMP400

LMP744 and LMP400 were designed and synthesized by a research group at Purdue University led by Mark Cushman, who serves as Gibson’s chief scientific officer, founder and board member. He is a Distinguished Professor Emeritus in the Borch Department of Medicinal Chemistry and Molecular Pharmacology, College of Pharmacy and a member emeritus of the Purdue Institute for Cancer Research and the Purdue Institute for Drug Discovery.

Cushman and his colleagues disclosed both drugs to the Purdue Innovates Office of Technology Commercialization, which received patents on the work from the United States Patent and Trademark Office.

About Purdue Innovates Office of Technology Commercialization

The Purdue Innovates Office of Technology Commercialization operates one of the most comprehensive technology transfer programs among leading research universities in the U.S. Services provided by this office support the economic development initiatives of Purdue University and benefit the university’s academic activities through commercializing, licensing and protecting Purdue intellectual property. In fiscal year 2025, the office reported 161 deals executed with 269 technologies licensed, 479 invention disclosures received and 267 U.S. and international patents received. The office is managed by the Purdue Research Foundation, a private, nonprofit foundation created to advance the mission of Purdue University. Contact otcip@prf.org for more information. 

About Purdue University

Purdue University is a public research university leading with excellence at scale. Ranked among top 10 public universities in the United States, Purdue discovers, disseminates and deploys knowledge with a quality and at a scale second to none. More than 106,000 students study at Purdue across multiple campuses, locations and modalities, including more than 57,000 at our main campus in West Lafayette and Indianapolis. Committed to affordability and accessibility, Purdue’s main campus has frozen tuition 14 years in a row. See how Purdue never stops in the persistent pursuit of the next giant leap — including its integrated, comprehensive Indianapolis urban expansion; the Mitch Daniels School of Business; Purdue Computes; and the One Health initiative — at https://www.purdue.edu/president/strategic-initiatives.