Immix Biopharma Inc. (NASDAQ: IMMX) has announced that the US FDA has designated a Rare Paediatric Disease status to its drug, IMX-110, for treatment of Rhabdomyosarcoma (“RMS”), a life-threatening pediatric cancer form in children. IMX-110 is an experimental drug that the company is currently evaluating in Phase 1b/2a clinical study.
RMS most common soft tissue sarcoma in children
The FDA designates serious and life-threatening disorders that mostly affect children aged 18 and younger and afflict fewer than 200,000 individuals in the United States as rare pediatric diseases. RMS is the most prevalent soft tissue sarcoma in children and adolescents, with an estimated 20,000 children of all ages diagnosed in the United States.
ImmixBio may be entitled to earn a Priority Review Voucher (PRV) from the US FDA if a New Drug Application for IMX-110 is authorized in the United States. The PRV can be used to achieve priority review for any future marketing application, or it can be sold or transferred.
ImmixBio’s Chief Executive Officer, Ilya Rachman, said, “We are pleased by FDA’s acknowledgment of the urgent need for a safe and effective treatment for children with this devastating disease. 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 three 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.”
IMX-110 has ODD for soft tissue sarcoma treatment
ImmixBio’s SMARxT Tissue-Specific Platform, which develops Tissue-Specific Therapeutics that aggregate at designated treatment sites at three to five times the rate of traditional medicines, has launched its first clinical-stage product, IMX-110. Already IMX-110 has an orphan drug designation from the FDA for soft tissue sarcoma treatment
ImmixBio has published clinical results from numerous strongly pretreated patients with different soft tissue sarcoma subtypes, showing a mean progression-free survival (PFS) of four months, zero drug-related serious side events, and no dose interruptions due to toxicity.