INV043 Achieves 80% Tumor Control in Preclinical Anal Cancer Models
Invion Limited secures a pivotal FDA Orphan Drug Designation for its lead candidate INV043, targeting anal cancer with promising preclinical results and a fast-tracked development path.
- FDA grants Orphan Drug Designation to INV043 for anal cancer
- Seven-year US market exclusivity and financial incentives awarded
- Preclinical studies show ~80% tumor control with immune checkpoint inhibitors
- Planned clinical trial collaboration with Peter MacCallum Cancer Centre
- INV043 also showing promise in skin and prostate cancer trials
FDA Orphan Drug Designation – A Strategic Milestone
Invion Limited (ASX – IVX) has achieved a significant regulatory milestone with the US Food and Drug Administration granting Orphan Drug Designation to its lead cancer drug candidate, INV043, for the treatment of anal cancer. This designation is reserved for therapies addressing rare diseases and offers a suite of benefits designed to accelerate development and commercialisation.
Among the key advantages are seven years of exclusive marketing rights in the US post-approval, potential tax credits for clinical trial expenses, fee waivers, and a streamlined regulatory pathway that can include faster approvals and smaller clinical trials. For Invion, this status not only validates the potential of INV043 but also enhances its attractiveness to strategic partners amid a competitive oncology landscape.
Promising Preclinical Data and Clinical Plans
The FDA’s recognition follows encouraging preclinical data generated in collaboration with the Peter MacCallum Cancer Centre. INV043 demonstrated efficacy against six squamous cell carcinoma cell lines representing anal cancer and achieved approximately 80% tumour control in mouse models when combined with immune checkpoint inhibitors, an impressive improvement over the 12% control rate with immune checkpoint inhibitors alone.
Importantly, no adverse side effects were observed in these studies, underscoring the potential safety profile of the therapy. Building on this, Invion and Peter Mac are preparing to initiate an anogenital cancer clinical trial, which will explore INV043’s effectiveness in combination with immune checkpoint inhibitors across anal, vulvar, and penile cancers, areas with limited treatment options and significant unmet medical need.
Broader Oncology Potential and Market Context
INV043’s promise extends beyond anal cancer. Early results from ongoing Phase I/II trials in non-melanoma skin cancer indicate good tolerability and encouraging efficacy signals, while a Phase II prostate cancer trial reported a positive response rate of 40-44% without significant adverse events. These findings suggest that INV043 could become a versatile addition to cancer treatment regimens.
CEO Prof Thian Chew highlighted the strategic timing of this designation, noting the looming patent expirations of several blockbuster immune checkpoint inhibitors over the next five years. The orphan drug status could position Invion to leverage its complementary technology alongside these established therapies, potentially accelerating market entry and partnership opportunities.
Looking Ahead
Invion’s Photosoft™ technology, the platform behind INV043, represents a novel photodynamic therapy approach that selectively targets cancer cells while promoting an immune response. Its less invasive nature and minimal side effects offer a compelling alternative to conventional treatments like surgery, especially for challenging cancers such as those in the anogenital region.
As Invion advances its clinical programs, the orphan drug designation marks a critical step in transforming promising science into tangible patient benefits and commercial success.
Bottom Line?
INV043’s orphan drug status sets the stage for accelerated development and potential market leadership in rare cancer therapies.
Questions in the middle?
- When will Invion initiate and report results from the planned anogenital cancer clinical trial?
- How will INV043 perform in combination with immune checkpoint inhibitors in human patients?
- What strategic partnerships might Invion pursue to capitalise on the orphan drug designation?
