FDA Grants Orphan Drug Designation to Soft Tissue Sarcoma Treatment

The FDA has granted orphan drug designation to Veyonda, a novel proprietary formulation of idronoxil, for the treatment of patients with soft tissue sarcoma.

The agent’s active ingredient, idronoxil, is a first-in-class, dual-acting oncotoxic and immuno-oncology molecule found to have unique anti-inflammatory properties, according to developer Noxopharm Limited.

A clinical study indicated that Veyonda is able to directly kill cancer cells working with the immune system to eradicate tumors. Idronoxil has been found to block the production of inflammatory cytokines that can cause extreme inflammatory response and septic shock. Veyonda is being evaluated for use with radiation, immunotherapy, and chemotherapy acorss several cancers, according to Noxopharm.

“The orphan drug designation will significantly increase the value proposition of Veyonda to potential purchasers or licensees by both lowering current development costs and by providing future competitive and financial advantages as Veyonda progresses through the clinical trial stages toward registration and approval for sale in the United States,” said Gisela Mautner, MD, PhD, MPH, MBA, FACPE, chief executive officer of Noxopharm Limited, in a press release. “With the FDA orphan drug designation now secured for Veyonda, the Noxopharm team is excited to move our preclinical assets along the drug development process, while continuing to deliver on our clinical program plan.”

Veyonda is currently being evaluated in combination with doxorubicin as a first-line treatment for patients with metastatic soft tissue sarcoma, in an open-label, dose-escalation and -expansion, phase 1 CEP-2 trial.

The FDA allowed the study to proceed under the investigational new drug process after evidence indicates the agent may improve generally poor responses experienced with chemotherapy in patients with sarcoma.3 The trial builds upon the positive findings yielded from the multicenter, phase 1 CEP-1 trial, which had delivered the proof-of-concept of Veyonda in combination with chemotherapy.

CEP-2 is a phase 1, open-label, dose-escalation and dose-expansion study of Veyonda administered to patients groups treated with doxorubicin for metastatic soft tissue sarcoma. The study expects to enroll approximately 30 patients in the United States with a range of soft tissue sarcomas who will be administered Veyonda plus doxorubicin combination therapy as a first-line treatment.

Soft tissue sarcomas are frequently fatal, with up to 50% of patients with high-grade sarcoma developing metastases and dying within 12 months. The CEP-Program is based on pre-clinical and clinical data for Veyonda indicating it enhances the anti-cancer effect of several standard chemotherapeutic agents, according to Noxopharm.

Reference

Orphan drug designation granted to Noxopharm by US FDA. News release. Noxopharm Limited; March 22, 2022. Accessed March 29, 2022. https://bit.ly/3NHQMWr