Kintara Therapeutics (Nasdaq: KTRA) gained nearly 30% during Thursday’s trading session on the heels of announcing that the company’s VAL-083 treatment had received orphan drug designation from the FDA.

The company’s VAL-083 is a small-molecule chemotherapeutic with a novel mechanism of action to treat diffuse intrinsic pontine glioma (DIPG), a rare and highly aggressive childhood brain cancer.

VAL-083 has shown promising clinical activity against various types of cancer, including brain cancer, ovarian cancer, and lung cancer.

Developed by Kintara, this small molecule has a novel mechanism of action that sets it apart from other cancer treatments. The National Cancer Institute has sponsored clinical trials for VAL-083 in the United States, further demonstrating its potential as an effective cancer treatment.

Kintara is now working to advance VAL-083 through the Global Coalition for Adaptive Research's registrational Phase 2/3 clinical trial, known as the GBM AGILE Study. This trial will aim to support the development and commercialization of VAL-083 for the treatment of glioblastoma (GBM), a particularly aggressive and difficult-to-treat form of brain cancer.

GBM is a devastating disease that has a poor prognosis, with most patients surviving only a few months after diagnosis. However, the clinical activity of VAL-083 observed in previous studies has given hope that it may be able to improve the outcomes for GBM patients.

Robert E. Hoffman, Kintara's President and CEO stated: "This is another important step for us in evaluating VAL-083 as a treatment for brain tumors in addition to our lead indication of glioblastoma. We will continue with our clinical advancement of the drug candidate with our objective of delivering a much-needed new treatment that can benefit patients.”

The FDA's orphan drug designation program provides orphan status to drugs that are defined as intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people.

The orphan drug designation provides sponsor companies of a drug with development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.

Shares of Kintara Therapeutics trade on the NASDAQ under the ticker symbol KRTA. For more information visit www.kintara.com.