iOnctura Expands Roginolisib Clinical Trials to US Market with Multi-Cancer Strategy
核心洞察
iOnctura (搜索) has expanded its roginolisib clinical trial program to the United States, with multiple cancer centers now enrolling patients in Phase II studies for metastatic uveal melanoma (搜索) and chronic lymphocytic leukemia (搜索).
The company's lead candidate roginolisib, an allosteric modulator of PI3Kδ (搜索), demonstrated promising early results including a doubling of overall survival compared to historical controls in uveal melanoma during Phase I trials.
Dana-Farber Cancer Institute received a substantial US Department of Defense grant to investigate roginolisib in combination with venetoclax and rituximab for relapsed/refractory chronic lymphocytic leukemia (搜索) patients.
iOnctura (搜索), a clinical-stage precision oncology company, has successfully expanded its roginolisib clinical trial program into the United States, marking a significant milestone in the company's global development strategy for treating neglected and hard-to-treat cancers. Multiple US trial sites are now actively enrolling patients in Phase II studies, with Dana-Farber Cancer Institute leading a new chronic lymphocytic leukemia (搜索) (CLL) trial backed by substantial US Department of Defense funding.
Multi-Indication Clinical Program Gains Momentum
The centerpiece of iOnctura (搜索)'s US expansion is the global Phase II randomized OCULE-01 study (NCT06717126), which began in March 2025 and is now enrolling patients with metastatic uveal melanoma (搜索) who have progressed on prior therapy. The study aims to assess whether roginolisib improves overall survival in this rare but aggressive form of eye cancer, with patients from both the US and Europe now participating in the trial.
Catherine Pickering, CEO and co-founder of iOnctura (搜索), emphasized the significance of this expansion: "We are proud to begin the next phase of iOnctura's global growth by making roginolisib available to US patients in our clinical studies. The enthusiastic response from US investigators, leading research centers, and patient communities highlights the significant opportunity to impact outcomes for individuals with limited treatment options."
Promising Early Clinical Signals
Roginolisib has demonstrated encouraging clinical activity in Phase I trials, particularly in uveal melanoma where the drug achieved a doubling of overall survival compared to historical controls. This rare eye cancer represents an area of significant unmet medical need, making the early positive signals particularly noteworthy for both patients and investigators.
The drug's unique mechanism as an allosteric modulator of PI3Kδ (搜索) distinguishes it from previous generations of PI3K inhibitors. This novel approach promises clinical activity without the detrimental tolerability issues that have limited earlier PI3Kδ inhibitors, potentially offering patients a more manageable treatment option.
Department of Defense Backs CLL Research
In a significant vote of confidence for roginolisib's therapeutic potential, the US Department of Defense recently awarded a substantial grant to Dr. Jennifer Brown at Dana-Farber Cancer Institute to explore the drug's application in relapsed/refractory CLL through a Phase I/II study (NCT06644183).
Dr. Jennifer R Brown, M.D. Ph.D., Director of the CLL Center of the Division of Hematologic Malignancies at Dana-Farber Cancer Institute and Principal Investigator on the study, expressed optimism about the combination approach: "We are enthusiastic about the potential of roginolisib in enhancing treatment outcomes for CLL patients who have relapsed after prior Bruton's tyrosine kinase inhibitor therapy. We feel roginolisib offers a promising avenue to improve remission depth and duration in combination with venetoclax and rituximab, whilst minimizing treatment-related side effects."
Expanding Across Multiple Cancer Types
Beyond uveal melanoma and CLL, iOnctura (搜索) is investigating roginolisib across several other malignancies where the PI3Kδ (搜索) pathway plays a critical role. The company has initiated studies in non-small cell lung cancer (搜索) (NSCLC) through the PULMO-01 study (NCT06879717), which began in May 2025, and myelofibrosis (搜索) (NCT06887803). In these diseases, targeting the PI3Kδ pathway in combination with standard therapies has shown potential to reverse treatment resistance.
The PI3K signaling pathway represents one of the most commonly dysregulated pathways across multiple cancer types, making roginolisib's broad applicability particularly valuable for addressing various malignancies with a single therapeutic approach.
Scientific Foundation and Future Development
Recent preclinical evidence published by Sasi, Tarantelli et al. in Haematologica supports the synergistic potential of roginolisib when combined with venetoclax in CLL treatment, providing scientific rationale for the combination studies now underway.
iOnctura (搜索) has strategically designed its clinical program to enable full development in uveal melanoma while simultaneously validating the approach in larger market indications. This parallel development strategy allows the company to address both rare diseases with high unmet need and more common cancers with significant commercial potential.
The company's approach represents a new archetype for precise PI3Kδ (搜索) inhibition through allosteric modulation, potentially overcoming the tolerability challenges that have limited previous drugs in this class while maintaining therapeutic efficacy across multiple cancer types.