On October 26, 2022 NovAccess Global Inc. (OTCQB:XSNX), a biomedical company developing novel immunotherapies for brain tumor patients, reported the approval of its application with the U.S. Food and Drug Administration (FDA) for Orphan Drug Designation (ODD) for TLR-AD1, a vaccine immunotherapy for the treatment of aggressive brain cancers, including glioblastoma and other high-grade gliomas (Press release, NovAccess Global, OCT 26, 2022, View Source [SID1234622476]).

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"Orphan Drug Designation is yet another timely milestone achieved by NovAccess Global as we prepare an Investigative New Drug (IND) application for FDA approval to start human clinical trials. We expect to submit the IND in the first half of 2023," said the Company’s Chief Executive Officer Dr. Dwain K. Irvin. "We are very pleased to have received this approval, and we believe the promise of our platform technology is underscored by the FDA expanding the scope of our original submission. This has been a team effort and I would like to congratulate our team for its ambitious and comprehensive efforts. The designation represents a critical step forward as we address an important and unmet healthcare challenge in the treatment of brain cancers."

"The FDA approval of our Orphan Drug application for TLR-AD1 is an important step forward," said Dr. Christopher Wheeler, President of StemVax Therapeutics, a wholly owned division of NovAccess Global, and served as Company’s lead in interactions with the FDA. "With Orphan Drug Designation status granted by the FDA, TLR-AD1 serves as our vanguard technology in our portfolio in the fight against cancer. It is our first in a platform of novel immunotherapy and innovative technology solutions we intend to bring to market in the service of cancer patients and their families across the globe. The special status afforded to us through the Orphan Drug Designation will enable an acceleration of the development of our therapies for new treatment options to treat a wide range of glioblastoma patients."

Dr. Wheeler continued, "Our therapeutic path involves a transformational process where tumor-killing immune responses for malignant glioma (MG) cells are higher than that of previous immunotherapies. This unique process involves the addition of proprietary substances to create a "cocktail" for more personalized treatment that substantially increases clinical benefits for patients. We look forward to advancing this novel immunotherapy into the clinic."

Glioblastoma is a form of aggressive brain cancer that annually impacts approximately 250,000 people globally and is on the rise in many countries, according to NovAccess scientists and published reports. The market data is more alarming, with glioblastoma accounting for approximately 50% of all malignant brain cancers diagnosed in the United States each year, and more than 10,000 Americans dying from this tumor type annually. Less than 5% of people with this cancer live longer than five years after their diagnosis. The global glioblastoma treatment market was estimated to be valued in excess of $2 billion in 2020, with projections for a compounded annual growth rate of more than 8% throughout the remainder of the decade.

The FDA’s Office of Orphan Products Development grants orphan designation status to investigational drugs and therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation provides benefits to drug developers which may include assistance in the drug development process, financial incentives to support clinical development, tax credits for clinical costs, exemptions from certain FDA fees and the potential for seven years of post-approval marketing exclusivity.

Sponsors seeking orphan drug designation for a drug must submit a request for designation to the FDA. Orphan drug designation is a separate process from seeking commercial approval or licensing, and the receipt of orphan drug designation status does not change the regulatory requirements or process for obtaining marketing approval from the FDA.

About TLR-AD1
TLR-AD1 is designed to activate anti-tumor immune responses against these brain tumors using immune-activating dendritic cells combined with the patient’s own tumor proteins. The resulting dendritic cell vaccines are matured with a proprietary combination of Toll-like receptor (TLR) adjuvants to boost their immune-activating potency beyond current vaccine preparations.

NovAccess Global expects to submit an Investigational New Drug (IND) application to the FDA for TLR-AD1 in the first half of 2023. In advance of the IND filing, the Company expects to announce a partnership with a clinical manufacturing organization for vaccine testing and production readiness for phase I-II clinical trials of TLR-AD1.