The Japanese drugmaker will obtain an exclusive global license to develop, manufacture and commercialise KT430, for an undisclosed upfront payment to KateTx, which will also receive additional milestone payments and royalties
Astellas, Leiden Bio Science Park. (Credit: Vysotsky/Wikimedia)
Japan-based Astellas Pharma has signed an exclusive license agreement with Kate Therapeutics (KateTx) to develop and commercialise KateTx’s investigational gene therapy, KT430.
KT430, which is in the preclinical stage, works to deliver a functional copy of the MTM1 gene, through a novel MyoAAV capsid, to treat X-linked myotubular myopathy (XLMTM).
Under the terms of the agreement, the Japanese drugmaker will obtain an exclusive global license to develop, manufacture and commercialise KT430.
Astellas will make an undisclosed upfront payment to KateTx, which will also receive additional development, regulatory and commercial milestone payments, and royalties.
Astellas chief strategy officer Adam Pearson said: “This agreement brings together Astellas and KateTx’s collective patient-focused missions, allowing us to evaluate how to advance this new potential therapy for people diagnosed with XLMTM.
“The combination of Kate’s unique scientific approach and Astellas’ depth of experience in developing gene therapies for XLMTM provides a solid foundation for the advancement of KT430 as it progresses towards the clinic.
“The addition of this new potential gene therapy treatment for XLMTM along with our current AT132 program further enhances our commitment to this patient community and dedication to delivering transformative medicines.”
XLMTM is a rare, serious, life-threatening neuromuscular disease characterised by extreme muscle weakness, respiratory failure, and early death.
It is caused by mutations in the MTM1 gene that affects myotubularin, a protein that plays an important role in the normal development, maturation and function of skeletal muscle cells.
The disease is associated with a high mortality rate of 50% in the first 18 months of life, and those who survive past infancy have additional 25% mortality by the age of 10.
Over 80% of patients require ventilator support, and most of them require a gastrostomy tube for nutritional support.
KateTx is a biotechnology company engaged in developing adeno-associated virus (AAV)-based gene therapies to treat genetically defined muscle and heart diseases.
KateTx president and CEO Kevin Forrest said: “We are very enthusiastic that Astellas has acquired worldwide rights to KT430 and what it means for the patients and families waiting for a potential new therapy for XLMTM.
“Our company uses novel technology platforms that directly address the key limitations of current gene therapies, including tissue-specific delivery and gene regulation, with the potential to improve efficacy and safety.
“We see tremendous opportunities to bring important new therapies to patients with very serious conditions like XLMTM, as well as for other genetic disorders that are the focus of our internal efforts.”