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Astellas Pharma Inc (OTC:ALPMF) (OTC:ALPMY) decided to license and advance a gene therapy from Kate Therapeutics for a debilitating muscle disorder known as X-linked myotubular myopathy (XLMTM).
Preclinical gene therapy candidate KT430 will seek to address XLMTM, a rare and life-threatening neuromuscular disease leading to severe muscle weakness, affecting predominantly males, with an incidence rate of approximately 1 in 40,000-50,000 newborn boys globally.
The move comes after four deaths and several severe adverse events occurred during clinical trials of a previous treatment from Astellas for the same condition.
The safety issues surrounding Astellas’ AT132 gene therapy for XLMTM have hindered its progress, leading to a clinical hold from the FDA.
Under the terms of the agreement, Astellas will make an undisclosed upfront payment to Kate, which is also eligible to receive milestone payments and royalties on worldwide sales. Astellas will receive an exclusive license to develop, manufacture and commercialize KT430.
At the same time the companies announced the new deal, Kate stated that the company is coming out of stealth mode with a Series A financing round of $51 million co-led by Westlake Village BioPartners and Versant Ventures.
