​Astellas Partners with Kate Therapeutics in Gene Therapy, After Four Patient Deaths in Previous Trials

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Pictured: Astellas sign/Courtesy, yu_photo, Adobe Stock

Thursday, Astellas Pharma announced its decision to license and advance a novel gene therapy from Kate Therapeutics for a debilitating muscle disorder. This move comes after four deaths and several severe adverse events occurred during clinical trials of a previous treatment from Astellas for the same condition.

Astellas and Kate both seek to treat a muscle disorder known as X-linked myotubular myopathy (XLMTM) using significantly lower doses of the viral vectors responsible for delivering genes into patients’ cells. Theoretically, this approach should reduce the likelihood of severe side effects and adverse events, Kate co-founder and CEO Kevin Forrest told BioSpace.

The safety issues surrounding Astellas’ AT132 gene therapy for XLMTM have hindered its progress, leading to a clinical hold from the FDA. Astellas did not state how licensing an alternative from Kate might affect the future of its existing candidate.

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.

KT430 will seek to address XLMTM, a rare and life-threatening neuromuscular disease leading to severe muscle weakness, affecting predominantly affects males, with an incidence rate of approximately 1 in 50,000 newborn boys globally, according to Boston Children’s Hospital.

Using a novel MyoAAV capsid, KT430 aims to deliver a functional copy of the MTM1 gene, targeting the extreme muscle weakness, respiratory failure and premature mortality that are hallmarks of this condition.

Kate Therapeutics Makes Its Debut

At the same time that 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

In addition to KT430, Kate’s initial pipeline also includes a program targeting myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy. DM1 is a progressive multisystem disorder that affects 1 in 15,000 adults in the U.S., according to Mount Sinai. FSHD affects between four and 10 people per 100,000 in the U.S., according to the National Organization for Rare Disorders.

Currently, there are no approved medicines for either DM1 or XLMTM.

Kate’s Series A was co-led by Westlake Village BioPartners and Versant Ventures, with additional participation from Osage University Partners and UF Innovate Ventures, according to the company. The company’s primary focus is the development of genetic medicines to treat muscle and heart diseases.

“There’s been some real successes in gene therapy ... as well as some CNS indications and musculoskeletal indications, but when it comes to heart and skeletal muscle, the field’s really at a standstill,” Forrest said.

Forrest said what co-founder and CSO Sharif Tabebordbar discovered while at the Broad Institute may be a pivotal technology for Kate.

“What Sharif developed at the Broad was a novel class of engineered AAV called MyoAAV, which can target skeletal and heart muscle at significantly lower doses than what’s currently available,” Forrest said. 

For Forrest, this collaboration with Astellas strongly validates Kate’s potential strength to produce novel therapies to save lives.

“This shows Astellas’ commitment to the space ... They know a lot about this patient population, their families and the KOLs [key opinion leaders],” he said. “It’s a great partner for us to test our AAV and engineered capsids in humans and potentially get some essential medicines to patients and their families.”

Meanwhile, Milan-based biotech firm AAVantgarde has also concluded a Series A funding round, with $65.4 million to propel the company’s work in AAV tech. The funds will be used to conduct proof-of-concept studies and further develop AAVantgarde’s two leading platforms, which have shown potential in ophthalmology applications, the company stated.

Lisa Munger is a senior editor at BioSpace. You can reach her at lisa.munger@biospace.com. Follow her on LinkedIn.​

Correction (June 8): This story has been updated from its original version to add facioscapulohumeral muscular dystrophy as a target in Kate's existing pipeline, to describe engineered capsids, not capsules, and to correct Forrest's comment regarding Astellas' knowledge of the patient population, their families and key opinion leaders in the space. BioSpace regrets the errors.

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