IMX-110 is currently in a Phase 1b/2a clinical trial.
The FDA awards RPD status for serious and life-threatening illnesses that primarily affect children under the age of 18; and impact on less than 200,000 people in the US.
Rhabdomyosarcoma is a high-grade malignant neoplasm, the most common soft tissue sarcoma in pediatric and adolescent populations and rare in adults. The prevalence of rhabdomyosarcoma in the country is about 20,000 children of all ages: and a five-year survival rate ranges from 20% to 30% for children in the risk group where cancer is widely spread in the body.
IMX-110 is ImmixBio’s lead candidate and the first clinical-stage product to come from its SMARxT Tissue-Specific Platform, which produces tissue-specific therapies that accumulate at target therapeutic sites at three to five times the rate of conventional drugs the company said. .
The California-headquartered biotech recently shared clinical data on multiple soft tissue sarcoma subtypes in several heavily pretreated patients demonstrating a median progression-free survival (PFS) of four months with no drug-related serious adverse events and zero dose interruptions due to toxicity.
The Rare Pediatric Disease designation qualifies Immix for rapid assessment.
If a new drug application for IMX-110 is approved, ImmixBio may be eligible to receive a Priority Review Voucher (PRV) from the FDA, which can be exchanged for a priority review for a subsequent marketing application, or may be sold or transferred.
“We are pleased with the FDA’s recognition of the urgent need for a safe and effective treatment for children with this devastating disease,” said Ilya Rachman, MD, PhD, CEO of ImmixBio. “We are encouraged by our Phase 1b/2a clinical data in soft tissue sarcoma. IMX-110 is a tissue-specific therapeutic agent that simultaneously attacks all three components of the tumor microenvironment, breaking the critical lifelines between the tumor and its metabolic and structural support. We believe that our SMARxT platform that generates tissue-specific therapies represents a clear alternative to the traditional one-target, one-mutation development model.”
The FDA has already granted IMX-110 orphan drug designation (ODD) for the treatment of soft tissue sarcoma.
Meanwhile, ImmixBio’s collaborations with BeiGene and Novartis are also exploring solid tumor therapy.