Biodexa Licenses Phase 1-Ready GIST Drug MTX240 from Otsuka
Biodexa Pharmaceuticals has licensed MTX240, a Phase 1-ready molecular glue therapy for gastrointestinal stromal tumors from Otsuka. The drug demonstrated preclinical efficacy in resistant tumors and is eligible for orphan drug exclusivity, with clinical trials planned by year-end.
Biodexa Pharmaceuticals has licensed MTX240, a Phase 1-ready molecular glue therapy for gastrointestinal stromal tumors, from Otsuka Pharmaceutical. The drug candidate demonstrated dose-dependent preclinical efficacy in TKI-resistant and non-resistant GIST models and is eligible for U.S. and EU Orphan Drug designation, which would provide seven and ten years of market exclusivity respectively upon approval.
MTX240 is a novel molecular glue that works by forcing PDE3A and SLFN12, two proteins expressed in GIST cells, to bind together, triggering tumor cells to self-destruct through apoptosis. Unlike current tyrosine kinase inhibitors that frequently fail once tumor cells mutate, MTX240's unique mechanism of action is designed to work whether or not the GIST has acquired secondary mutations. The company plans to initiate a Phase 1b/2a study by year-end, focusing initially on patients with TKI-resistant GIST.
The global GIST market is currently valued at approximately $1.3 billion, with projected annual growth of around 6.6% through 2032. In the U.S. alone, between 4,000 and 6,000 people are diagnosed with GIST each year, while globally the number of new GIST patients annually is estimated at between 80,000 and 120,000. Once patients have cycled through available TKIs and acquired resistance to all of them, median time to tumor progression is 1.2 months and median overall survival is 9.8 months.
MTX240 has issued Composition of Matter patents extending through 2037. The drug candidate complements Biodexa's ongoing Phase 3 development of eRapa in Familial Adenomatous Polyposis, reinforcing the company's focus on gastrointestinal oncology. The strategic value of differentiated GIST assets was underscored by a recent approximately $1 billion acquisition of a Phase 1 candidate in the same therapeutic area.
Biodexa's pipeline strategy centers on advancing scientifically validated mechanisms with potential to address clear unmet medical needs, particularly in settings where existing therapies provide only transient benefit or have permanent life-altering impact on quality of life. The company holds exclusive global rights to MTX240 except for Japan, where rights are retained by Otsuka.