Imugene Limited (ASX: IMU), a clinical-stage immuno-oncology company, has announced a significant breakthrough with the US FDA granting Orphan Drug Designation (ODD) for its novel oncolytic virotherapy, CF33-hNIS, also known as VAXINIA. This latest achievement marks a vital step in the company’s quest to tackle cholangiocarcinoma, a rare and notoriously aggressive form of bile tract cancer.
Cholangiocarcinoma has long been a thorn in the side of oncologists, known for its poor prognosis and resistance to conventional treatments like chemotherapy. With few effective therapeutic options available, this cancer poses a high unmet clinical need. VAXINIA, Imugene’s brainchild, offers a glimmer of hope by employing an innovative approach that uses viruses to selectively target and destroy cancer cells, while also engaging the patient’s immune system to help wipe out tumours.
Why Orphan Drug Designation Matters
The FDA’s Orphan Drug Designation isn't just a badge of honour; it comes with a host of perks designed to foster the development of treatments for rare diseases. In Imugene’s case, the ODD means a range of incentives, including potential tax credits, access to grant funding, a waiver of certain regulatory fees, and a coveted seven-year market exclusivity period once VAXINIA clears the final FDA approval hurdle.
Leslie Chong, Managing Director and CEO of Imugene, hailed the FDA’s decision as a "major milestone," signalling the potential for VAXINIA to meet a desperate clinical need. “We are excited to continue advancing this program, which has already shown meaningful clinical responses in patients,” she said, referring to data from the company’s MAST (Metastatic Advanced Solid Tumours) trial.
Encouraging Early Results
Imugene’s push to secure FDA approval was bolstered by promising findings from its ongoing MAST trial, which is currently evaluating VAXINIA in patients with advanced solid tumours. So far, the trial has delivered results that warrant cautious optimism. One patient with cholangiocarcinoma achieved a complete response, while another stabilised after treatment, an encouraging outcome for a cancer that’s usually anything but.
In light of these positive signals, Imugene has launched a bile tract cancer expansion trial as part of the MAST program, aiming to enrol 10 more patients to further assess VAXINIA’s potential in treating this deadly disease. The trial is currently in the dose-escalation phase, where VAXINIA is being tested as both a standalone therapy and in combination with checkpoint inhibitors, a type of immunotherapy.
The Long Road Ahead
While the Orphan Drug Designation is undoubtedly a feather in Imugene’s cap, the road to FDA approval is long and filled with clinical and regulatory hurdles. For now, though, the Sydney-based biotech firm is riding high on this latest endorsement from the FDA.
Cholangiocarcinoma is a rare cancer, with fewer than 200,000 cases diagnosed in the US annually—just enough to qualify for the FDA’s orphan designation. However, its rarity doesn’t diminish its impact; for patients and their families, new treatment options like VAXINIA represent much-needed hope.
VAXINIA’s underlying technology, oncolytic virotherapy, is part of a growing field that seeks to weaponise viruses against cancer cells. By selectively infecting and killing cancer cells while leaving normal tissues unharmed, these therapies are designed to work in tandem with the body’s immune system. In some cases, as seen in Imugene’s trial, they can be combined with other forms of immunotherapy to amplify the therapeutic effect.
What's Next for Imugene?
Imugene has made no secret of its ambition to become a key player in the global immuno-oncology space. Beyond VAXINIA, the company’s pipeline includes multiple other immunotherapies aimed at harnessing the immune system’s natural ability to fight off tumours. The biotech firm is also developing an off-the-shelf CAR T-cell therapy, Azer-cel, for treating blood cancers, alongside a range of B-cell vaccine candidates targeting various cancer types.
With its deep pipeline of candidates and strong backing from international cancer experts, Imugene is positioning itself at the forefront of cancer immunotherapy innovation. But while the company is buoyed by today’s Orphan Drug news, investors will be keeping a close eye on how the MAST trial progresses and whether VAXINIA can eventually secure full regulatory approval.
For now, Imugene is focused on building on the momentum of the ODD grant, expanding its trials, and ultimately proving that VAXINIA can deliver for patients battling cholangiocarcinoma.
