FDA grants orphan drug status to novel brain cancer treatment
Numiera also gets investment from Colorado to develop glioma therapy
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to a novel brain cancer treatment — one designed for malignant gliomas such as glioblastoma — that’s being developed by Numiera Therapeutics.
Numiera also announced that Colorado’s Office of Economic Development and International Trade (OEDIT) will be investing in the company’s development program to help further advance this therapeutic approach.
“Our goal is clear: to move this science into the clinic and give hope to patients who currently have limited treatment options,” Josh Pan, PhD, head of business development and cofounder of Numiera, said in a company press release. Pan is also the chief business development officer for Swiss-based Landmark Bioventures.
“With the orphan drug designation and investment from the state of Colorado, we are well-positioned to take the next step toward clinical trials,” Pan said.
The FDA awards orphan drug designation to therapies being developed to treat rare diseases, specifically defined in the U.S. as conditions that affect fewer than 200,000 people. This status provides extra incentives for companies to invest in treatments for rare diseases — which, by definition, have a small market that can make it difficult for drug developers to turn a profit. Among other incentives, treatments granted orphan drug designation are guaranteed seven years of market exclusivity if they are ultimately approved by the FDA.
Numiera calls its brain cancer treatment strategy a ‘paradigm shift’
Gliomas are a type of brain tumor caused by the uncontrolled growth of glial cells — brain cells that help to support the activity of nerves. Numiera’s lead candidate, which the company did not name, aims to inhibit the growth of glial cells by interfering with how these cells generate energy.
According to Numiera, glioma cells get a lot of their energy by oxidizing, or burning, fat molecules called fatty acids. This is a notable difference from most healthy cells in the body, which mainly get energy by burning glucose, a type of sugar molecule.
By targeting how these cancer cells generate energy … we are not only addressing an unmet need for glioblastoma patients but also paving the way for broader applications in the field of oncology.
Numiera’s approach aims to inhibit the molecular machinery, called the carnitine palmitoyl transferase (CPT1) pathway, that glioma cells use to burn fatty acids. The company believes that blocking this pathway will help prevent the growth of cancer cells. To that end, Numiera is now involved in preclinical research aiming to advance this treatment strategy toward testing in people.
Elizabeth (Izi) Stoll, PhD, cofounder and CEO of Numiera, said the company’s brain cancer treatment strategy “represents a paradigm shift in neuro-oncology.”
“By targeting how these cancer cells generate energy through fatty acid oxidation, we are not only addressing an unmet need for glioblastoma patients but also paving the way for broader applications in the field of oncology,” Stoll said.
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