The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMXT 1501 to treat malignant gliomas, including a particularly aggressive childhood brain tumor called diffuse intrinsic pontine glioma (DIPG).
The FDA grants orphan drug designation to experimental therapies intended to treat conditions affecting fewer than 200,000 people in the U.S., offering extra incentives to companies that invest in treatments for rare diseases. The designation gives AMXT 1501’s developer, Aminex Therapeutics, perks such as tax credits and fee waivers, plus a guaranteed seven years of market exclusivity if the FDA ultimately approves the therapy is ultimately approved as a glioma treatment.
This is the second orphan drug designation the FDA has granted to AMXT 1501. The therapy also holds the designation as a potential treatment for neuroblastoma, another type of nervous system tumor marked by the uncontrolled growth of immature nerve cells.
“This second Orphan Drug Designation is a powerful validation of AMXT 1501’s potential to make a meaningful difference for patients facing some of the most devastating cancers,” Mark Burns, PhD, Aminex’s president and chief scientific officer, said in a company press release.
Combination treatment
The FDA’s orphan drug designation for glioma covers AMXT 1501’s use in combination with another anticancer agent, difluoromethylornithine (DFMO). According to Aminex, these two therapies are expected to work in concert to stop tumors from suppressing the immune system, allowing immune cells to attack and kill the cancer. DFMO is considered experimental in glioma, but it is approved as a treatment for some people with neuroblastoma under the name Iwilfin.
Gliomas are brain tumors that arise from the uncontrolled growth of glial cells, which normally support and protect nerve cells in the brain. DIPG is a particularly aggressive subtype of glioma that usually affects children ages 5 to 10. There is no cure, and the median survival time is less than a year after diagnosis.
“DIPG is heartbreaking — children diagnosed with this brain tumor have virtually no effective treatment options and very little time,” Burns said. “We are committed to changing that.”
A Phase 1/2 clinical trial (NCT06465199), sponsored by Milton S. Hershey Medical Center in Pennsylvania, is testing AMXT 1501 in combination with DFMO as a treatment for children and young adults up to age 21 who have DIPG, neuroblastoma, and certain other pediatric cancers.
In the Phase 1 part of the study, all participants will receive various dose regimens of AMXT 1501 plus DFMO, with the main goal of evaluating safety and determining the optimal dose for further testing. Those results will inform the second part of the study, in which participants will receive DFMO alone or in combination with AMXT 1501 at a set dose, to assess how adding AMXT 1501 affects disease progression. The trial is recruiting participants at six sites across the U.S., and the company said more sites are planned to open in the future.
Aminex is sponsoring a separate Phase 1/2 study (NCT07287917) testing AMXT 1501 plus DFMO in adults with inoperable, advanced breast cancer or melanoma. That study is also recruiting at sites in the U.S.
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