On January 3, 2021 Immix Biopharma, Inc. (Nasdaq: IMMX) ("ImmixBio" or the "Company"), a biopharmaceutical company pioneering Tissue-Specific Therapeutics (TSTx)TM targeting oncology and immuno-dysregulated diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for IMX-110 for the treatment of a life-threatening form of pediatric cancer in children, rhabdomyosarcoma. IMX-110, an investigational product, is currently being evaluated in a Phase 1b/2a clinical trial (Press release, Immix Biopharma, JAN 3, 2022, View Source [SID1234597956]).
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The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children aged 18 years or younger and impact fewer than 200,000 people in the United States.
If a New Drug Application in the United States for IMX-110 is approved, ImmixBio may be eligible to receive a Priority Review Voucher (PRV) from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application, or may be sold or transferred.
"We are pleased by FDA’s acknowledgment of the urgent need for a safe and effective treatment for children with this devastating disease," stated ImmixBio’s Chief Executive Officer Ilya Rachman, M.D., PhD. "We are encouraged by our Phase 1b/2a clinical data in soft tissue sarcoma. IMX-110 is a tissue-specific therapeutic that simultaneously attacks all 3 components of the tumor micro-environment, severing the critical lifelines between the tumor and its metabolic and structural support. We believe our SMARxT platform generating Tissue-Specific Therapeutics represents a distinct alternative to the traditional ‘single target, single mutation’ development model."
Rhabdomyosarcoma ("RMS") is a high-grade, malignant neoplasm, the most common soft tissue sarcoma in pediatric and adolescent populations and which rarely occurs in adults. The prevalence of RMS in the United States is approximately 20,000 children of all ages. The five-year survival rate ranges from 20% to 30% for children in the high-risk group where cancer spreads widely in the body.
IMX-110 is the first clinical-stage product of ImmixBio’s SMARxT Tissue-Specific Platform, which produces Tissue-Specific Therapeutics that accumulate at intended therapeutic sites at 3 to 5 times the rate of conventional medicines. The FDA has already granted orphan drug designation (ODD) to IMX-110 for the treatment of soft tissue sarcoma.
ImmixBio recently shared clinical data across multiple soft tissue sarcoma subtypes in several heavily pretreated patients demonstrating median progression-free survival (PFS) of 4 months with zero drug-related severe adverse events and zero dose interruptions due to toxicity. The data can be viewed in the Immix Biopharma Corporate Presentation at View Source