Orphan Drug Status Granted to Gibson Oncology's New LMP744 Cancer Treatment

Amarex has announced a significant achievement for Gibson Oncology: the FDA has granted Orphan Drug Designation (ODD) for LMP744, a promising treatment for gliomas. This drug demonstrates exceptional capability by crossing the blood-brain barrier at ten times the necessary concentration to kill cancer cells and maintaining this level for over 24 hours after each dose.

“For decades, glioblastoma brain cancer patients have not seen improvements in survival rates with Temodar, the current standard treatment,” noted Mr. Randall Riggs, President & CEO of Gibson Oncology. “Our objective is to bring LMP400 and LMP744 to market as swiftly as possible to help cancer patients in need.”

Receiving ODD highlights the innovative nature of LMP744, which targets two critical cancer pathways: it inhibits TOPO 1 and reduces cMyc overexpression through strong binding to the G4 quadruplex of cMyc. Dr. Kush Dhody, President of Amarex, remarked, “This designation arrives at a crucial time for families battling this disease. It positions Gibson Oncology for future success and advances the commercialization of this life-saving treatment.”

A multi-year collaboration with cMyc expert Dr. Danzhou Yang from Purdue University led to the discovery that both LMP744 and LMP400 are potent inhibitors of TOPO 1 and the cMyc oncogene. LMP744 selectively targets these major cancer drivers, offering a unique mechanism of action that positions it effectively against cancers with high unmet medical needs.

Currently, LMP744 and LMP400 are set to enter Phase 2 human clinical trials for recurrent gliomas in partnership with the National Institutes of Health (NIH). Gibson Oncology aims to leverage the FDA’s ODD for both drugs to expedite their regulatory progression for all gliomas, including pediatric cases. This rapid advancement is crucial for delivering these treatments to cancer patients promptly.

Gibson Oncology, a private biotech firm, boasts a unique portfolio of five small molecules for cancer therapy discovered at the National Cancer Institute and Purdue University. These agents influence both TOPO 1 and cMyc activity and have undergone five clinical trials supported by the NCI in recurrent solid tumors and lymphomas. The lead compound, LMP744, is ready for a Phase 2 trial in patients with first recurrent glioblastoma, while LMP400 is set for a similar trial in glioma patients with PTEN deficiency.

Additionally, Gibson's innovative second-generation drugs, known as the 7-Azaindenoisoquinolines (AZAs), have shown combined TOPO 1 and cMyc activity. Discovered by Dr. Mark Cushman at Purdue University and exclusively licensed to Gibson, these new agents exhibit TOPO 1 effects without resorting to traditional mechanisms used by older drugs like Irinotecan and Topotecan. They act on cMyc inhibition epigenetically through the G-quadruplex.

Amarex Clinical Research, LLC, is a global Contract Research Organization offering extensive expertise in clinical research. Their services encompass project management for Phase I-IV trials, regulatory affairs including FDA applications, clinical operations, adaptive study designs, statistical analysis, data management, medical monitoring, and safety pharmacovigilance. Amarex’s comprehensive services support clients from regulatory approval strategies to conducting trials and preparing marketing applications.