negotiating the high-stakes field of Next- ge Ne ratio N ca N ce rd rugs

Each year, researchers develop new drugs to fight an array of diseases and conditions. However, only a select minority progress from concept to candidate drug to patients via clinical trials.

One company that’s successfully run the gauntlet over the past 10 years is Canadian-Israeli immunotherapy biotech Vaxil Bio Ltd. ( TSX.V: VXL). It’s shepherding the development of ImMucin, a promising immunotherapy that assists a patient’s own immune system to recognize and attack cancer cells as it does other foreign cells. Vaxil’s ImMucin has successfully completed a clinical trial and was subsequently granted Orphan Drug Status by both the U.S. Food and Drug Administration (FDA) and European Medicines Agency. The company already possesses a portfolio of patents around the globe.

The U.S. Cancer Research Institute calls immunotherapy “the most promising cancer treatment of our time.” The FDA continues to approve immunotherapies for many types of cancers, and the slate of cancers being targeted for treatment continues to expand.

Chemotherapy, the mainstay of cancer treatment today, was developed after an accidental mustard gas spill in the Second World War demonstrated the improbable concept that toxic chemicals could treat cancer. However, the toxic nature of the chemicals used often induces significant side effects, and patients can relapse after remission.

“Like chemo, immunotherapy was originally considered a highly improbable approach,” says Isaac Maresky, Vaxil’s executive director of the board. “However, a recent wave of high-profile successful treatments — including those for previously untreatable cancers — has many believing immunotherapy will prove a more targeted, less toxic treatment. Analysts predict immunotherapy will be used to treat most cancers by 2030, although treatment will likely take a holistic approach, integrating multiple modalities. Vaxil’s invention offers a unique treatment mechanism, one that could even work synergistically in augmenting others.”

Vaxil researchers leverage the company’s proprietary VaxHit platform algorithm to search for promising peptides— chains of amino acids. They’re first concentrating on MUC1, a marker found in 90 per cent of all cancers. ImMucin is a synthetic injection containing copies of segments of the MUC1 cancer markers believed to be recognizable by T cells, which can then identify and destroy cancer cells.

Vaxil has already completed a Phase I/ II clinical trial for ImMucin. Using it to treat 15 patients with multiple myeloma, a rare blood cancer, researchers reported “a broad and robust immunological response in 100% of the patients.” Of those patients, 11 demonstrated either remission or stable disease for extended periods past completion of treatment. Yet even a highly promising drug requires capital support before it can reach patients commercially.

“It takes around 15 years to develop a new therapy, and Vaxil has already spent nearly a decade developing its novel thesis into a pipeline of promising drugs,” says Maresky. “With financing and continued results, it would not be unrealistic to expect Vaxil to develop a commercial cancer drug in under five years. Commercializing ImMucin as quickly as possible would result in financial independence and in turn fund additional Vaxil drugs already under development in our Israeli lab.”

In addition to ImMucin, Vaxil scientists have generated the first ever signal peptide-specific antibodies, which target the signal peptide portion of the MUC1 cancer marker. These antibody proteins are immune system watchdogs that bind and neutralize foreign invaders. The firm is currently awaiting a key patent decision on this platform.

Deeper in its pipeline, Vaxil has used its VaxHit algorithm to develop a potential tuberculosis vaccine. The World Health Organization estimates that 1.8 million people died from tuberculosis in 2015; that same year, 480,000 developed multidrug-resistant TB.

“Based on our laboratory data and in 21 patient samples, Vaxil vaccine candidates have produced superior results compared to current TB vaccines,” says Vaxil’s chairman, Dr. Saeid Babaei. “However, as a company, we must remain focused on cancer. Our strategy with our infectious disease platform is to partner with a large pharma possessing the infrastructure to move the program forward.”

While Dr. Babaei and Maresky focus on strategy and capital markets supporting its trials, researcher Dr. Riva Kovjazin continues R&D with Dr. Limor Chen, vice-president of operations and clinical affairs at Vaxil’s laboratory in the Weizmann Science Park, Israel. Pending regulatory approval and financing, Vaxil hopes to embark on its pivotal Phase II trial for ImMucin.

“With our MUC-1 based treatment, I believe we have a truly novel immunotherapy platform,” says Dr. Chen. “ImMucin’s FDA Orphan Drug Designation means the door is open for expedited pathways towards approval. We’ve already launched the manufacture of ImMucin for our planned clinical trial. After a decade of R&D, Vaxil’s next trial will certainly be our most important milestone, with the potential to catapult ImMucin into a registration trial, a major step towards seeking FDA approval.”