RARE Daily

FDA Grants Rare Pediatric Disease Designation to Immix Biopharma for IMX-110 for Life-Threatening Pediatric Cancer

January 3, 2022

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Immix Biopharma for IMX-110, its experimental therapy for the treatment of a life-threatening form of pediatric cancer in children, rhabdomyosarcoma.

Photo: Ilya Rachman, CEO of ImmixBio

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 percent to 30 percent 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. IMX-110, an investigational product, is currently being evaluated in a phase 1b/2a clinical trial.

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.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Mirum Pharmaceuticals sold its voucher for $110 million.

“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,” said Ilya Rachman, CEO of ImmixBio. “We believe our SMARxT platform generating Tissue-Specific Therapeutics represents a distinct alternative to the traditional ‘single target, single mutation’ development model.”

Author: Rare Daily Staff

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