Noxopharm (ASX: NOX) will hopefully see an increase in value of their flagship drug following this morning’s announcement. The Company has been granted Orphan Drug Designation from the United States FDA for their proprietary drug, Veyonda, for the treatment of soft tissue sarcoma. 

Orphan Drug Designation is given to certain drugs and biologics that show promise as novel treatments to support the development of new treatments for rare diseases. The Designation allows for expedited regulatory processes, exemption from certain fees (estimated to be $2.9 million), tax credits for qualified clinical trials and the potential for seven years of market exclusivity following approval. 

Noxopharm is currently putting Veyonda through a Phase 1 dose escalation and expansion study. The drug is being given in conjunction with doxorubicin, a chemotherapeutic, to treat metastatic soft tissue sarcoma. Approximately 30 patients will be enrolled in the CEP-2 trial at major cancer sites in the US such as the City of Hope Cancer Centre in Los Angeles. 

Soft tissue sarcomas are a rare type of cancer that affects the tissues that connect and surround other structures. The five year survival rate for soft tissue sarcomas is about 65%. This decreases to 50% once the cancer has reached nearby organs or lymph nodes. Once the cancer has metastasised to other parts of the body, the five year survival rate drops further to 18%. Up to 50% of high-grade sarcoma patients develop these metastases and often do not survive past 12 months. 

Veyonda is Noxopharm’s lead asset. It is an oncotoxic and immuno-modulatory compound that works in conjunction with chemo and radiotherapy to increase the number of cancer cells killed and assists with activating the body’s own immune system. 

Chemotherapy has been the mainstay of cancer treatment since its invention in the 20th century. Today, the chemotherapy market is worth a huge USD $50 billion per annum. Seeing the opportunity to piggyback on this, Noxopharm developed Veyonda to hopefully improve the outcome of chemotherapy at lower doses, leading to fewer negative side effects. 

CEO of Noxopharm, Dr Gisela Mautner said: “It is pleasing that the Noxopharm application for Orphan Drug Designation was approved so quickly. Considering that out of approximately 360 approved ODDs last year, only four went to Australian companies, demonstrates the high bar that is being set by the FDA. The 7-year period of market exclusivity is commercially extremely valuable, as it means that the FDA will not approve a subsequent drug for the same use within this timeframe.

“The ODD 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 towards registration and approval for sale in the US. 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.”

Noxopharm rounded out the half year ended 31st December 2021 with a cash balance of $22.6 million, reporting a loss of -$8.6 million, up 28% on the year prior.