Amarex receives orphan drug status for novel cancer treatment LMP744 from Gibson Oncology

New name represents a milestone in glioblastoma treatment. Decades of development of a glioma treatment that crosses the blood-brain barrier at ten times the concentration required to kill cancer cells

GERMANTOWN, Md.–(BUSINESS WIRE)–Amarex today announced that its work on behalf of Gibson Oncology has resulted in an Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for LMP744.

LMP744 is designed to treat gliomas by crossing the blood-brain barrier (BBB) ​​at 10 times the concentration needed to kill cancer cells and maintaining high concentrations for 24 hours per dose.

“For decades, patients with glioblastoma brain tumors have not experienced any meaningful improvement in overall survival with Temodar, the current standard of care,” said Randall Riggs, President and CEO of Gibson Oncology. “Our goal is to bring both LMP400 and LMP744 to market using the shortest and most rapid route possible to help cancer patients in need.”

Obtaining ODD for LMP744 for all gliomas highlights the drug's promise and novel treatment for inhibiting two cancer targets: TOPO 1 and downregulating cMyc overexpression through the drug's strong binding to the G4 quadruplex of cMyc.

“This important award couldn't come at a more critical time for the families battling this disease,” said Dr. Kush Dhody, President of Amarex. “Achieving this milestone paves the way for future success for the company and, most importantly, paves the way for Gibson Oncology to commercialize this life-saving treatment.”

Through a multi-year collaboration with cMyc expert Dr. Danzhou Yang of Purdue University, Gibson Oncology discovered that LMP400, which received ODD earlier this year, and LMP744 are potent nM inhibitors of both the TOPO 1 and cMyc oncogenes. Together, the teams determined that LMP744, a small molecule, specifically targets two known drivers of human cancer: the cMyc oncogene and TOPO 1.

By discovering this compound's unique mechanism of action against cancer, Gibson Oncology was able to better position LMP744 and LMP400 for cancer targets with high unmet medical need.

Currently, LMP744 and LMP400 are poised to enter a Phase 2 human clinical trial in recurrent gliomas in collaboration with the National Institutes of Health (NIH).

Gibson Oncology believes it is important to leverage the orphan drug designation received from the FDA for both drugs for all gliomas, not just glioblastoma, which includes children with gliomas. Thanks to rapid regulatory progress, LMP400 and LMP744 have the shortest and fastest path to market, benefiting cancer patients in need.

About Gibson Pharmaceuticals
Gibson Oncology is a private biotechnology company with a unique portfolio of five small molecules for cancer therapy discovered at the National Cancer Institute and Purdue University. These compounds have both TOPO 1 and cMyc activity and have completed five fully NCI-supported clinical trials in recurrent solid tumors and lymphomas. Our lead compound, LMP744, is currently in Phase 2 trials in patients with first recurrent glioblastoma and LMP400 in patients with first recurrent glioma and PTEN deficiency.

Gibson has a second generation of newly invented and patented drugs that also have combined TOPO-1 and cMyc activity, known as 7-azaindeneoisoquinolines (AZAs). These compounds were discovered by Dr. Mark Cushman at Purdue University and exclusively licensed to Gibson. They also exhibit TOPO-1 effects that do not rely on the traditional camptothecin mechanisms found in older drugs such as irinotecan and topotecan. Gibson has demonstrated that the AZAs act epigenetically via the G-quadruplex to inhibit cMyc.

About Amarex Clinical Research, LLC
Amarex Clinical Research, LLC, is a full-service global contract research organization (CRO) with extensive experience in conducting clinical research. The combined experience of the leadership team includes the design and execution of several hundred clinical research projects in many therapeutic indications. Amarex offers services in project management: Phase I-IV, BE/BA, PK/PD; regulatory affairs: FDA submissions and meetings, submissions to international health authorities, GxP compliance audits; clinical operations; adaptive study designs; statistical analysis; data management; medical monitoring; safety and pharmacovigilance; and general consulting. Amarex can guide your product through the entire regulatory process, from creating the regulatory strategy to conducting studies and writing the regulatory application. Join our growing list of customers with approved products supported by high quality and cost-effective services. For more information, visit www.amarexcro.com.

Contacts

Media contact:
Kalli DeWeese
[email protected]