Orotecan® (Oral irinotecan HCI) (VAL-413) Program
In a Phase 1/2a clinical study, plasma levels of Orotecan® were comparable to intravenous (IV) irinotecan. Orotecan® was well tolerated at doses up to 90 mg/m²/day, with no dose-limiting toxicities (DLTs) observed. Notably, some patients received treatment at home for up to 13 cycles (approximately 9 months). We believe the current trial results supports moving forward into a pivotal clinical study aimed at securing FDA approval.
Irinotecan in Pediatric Oncology
Irinotecan (IV) is a chemotherapy agent commonly used alone or in combination with other treatments for the management of adult and pediatric solid tumors. In pediatric cancers these include, but are not limited to:
- Ewing sarcoma
- Neuroblastoma
- Rhabdomyosarcoma
- Hepatoblastoma
- Medulloblastoma
IV administration via infusion, while effective, often requires protracted daily in-patient infusion treatments, contributing to patient burden and logistical challenges for families.
IV irinotecan given orally has been established as an effective alternative to intravenous treatment; however, poor palatability of the IV product has significantly limited clinical adoption of this practice.
Orotecan® (Oral Irinotecan HCl) A Patient-Centered Innovation
Orotecan® is a novel oral formulation of irinotecan hydrochloride developed to improve treatment experience for pediatric patients. It provides a more convenient, at-home alternative to traditional IV administration, aiming to minimize the physical, emotional, and logistical burden of frequent hospital visits.
Key Benefits:
- Reduced Hospital Time: IV regimens can consume 30–50% of treatment time in hospital settings.
- At-Home Convenience: Allows treatment continuity with fewer disruptions to daily routines.
- Improved Quality of Life: Minimizes emotional and physical toll associated with hospital-based care
- Enhanced Compliance & Cost Savings: Home-based care may improve adherence to therapy and reduce healthcare resource demands.
Clinical Development Lead-in-Trial
Orotecan® is currently undergoing a multicenter Phase 1/2a clinical trial to assess its safety, tolerability, and pharmacokinetics when administered alongside oral temozolomide. The study targets 20 pediatric patients aged 1 to 30 years with recurrent solid tumors, including Ewing sarcoma, neuroblastoma, rhabdomyosarcoma, hepatoblastoma, and medulloblastoma.
Study Endpoints:
- Characterize pharmacokinetics of Orotecan® vs. unformulated irinotecan (p.o.)
- Confirm improved tolerability
- Tumor response (RECIST v1.1)
- Safety and tolerability (CTCAE v5.0)
Orotecan® Clinical Trial
Orotecan® is currently being studied in a Phase 1-2 clinical trial enrolling at nine clinical sites in the United States. (ClinicalTrials.gov ID: NCT04337177)
- Duke University Children’s Hospital
- Cincinnati Children’s Hospital Medical Center
- Sarah Cannon Research Institute
- University of North Carolina
- UCSF Medical Center
- Atrium Health, Levine Children’s Hospital
- Children’s National Hospital
- Phoenix Children’s Hospital
- Riley Hospital for Children
Interim Study Results
Presented at the American Association for Cancer Research (AACR) annual meeting in April 2025 indicated that Orotecan® was well-tolerated, with no dose-limiting toxicities observed at the 90 mg/m²/day dose level. Pharmacokinetic analyses demonstrated that the oral formulation achieved plasma levels of irinotecan and its active metabolite SN-38 comparable to those observed with IV administration.
- Patients have been treated for up to 13 cycles (9months) at home.
- Well-tolerated up to 90 mg/m²/day with no dose-limiting toxicities
- Achieved plasma levels of irinotecan and SN-38 comparable to IV administration
- Some patients have received treatment at home for up to 13 cycles (9 months)
Clinical Development Strategy
- Orotecan® is positioned to enter pivotal registration-directed trials for recurrent pediatric cancers. The FDA has provided guidance that a single-arm trial with response endpoints would be suitable to support accelerated approval for Orotecan®.
Market Opportunity
- Pediatric cancers represent orphan cancer indications.
- Feedback from oncologists suggests strong potential for Orotecan® adoption in adult patients, indicating a substantial market opportunity
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