Novartis has acquired Kate Therapeutics for up to $1.1 billion, the companies said today, in a deal that expands the buyer’s presence in both neuroscience drug development and gene therapy.
Based in San Diego, KateTx focuses on developing adeno-associated virus (AAV)-based gene therapies to treat genetically defined neuromuscular diseases. Kate’s technology platforms integrate capsid and cargo technologies with the goal of delivering payloads to desired tissues, while potentially mitigating off-target effects to tissues such as the liver.
KateTx says its approach aims to improve both the efficacy and safety of gene therapies, opening potential possibilities for treating complex diseases previously difficult to address with current technologies, including inherited neuromuscular diseases.
Kate’s Directed Evolution of AAV Capsid Leveraging In Vivo Expression of Transgene RNA (DELIVER) platform combines diverse capsid library generation with transcript-based in vivo selection and machine learning to evolve functional capsid variants. By applying DELIVER, KateTx has developed a class of liver de-targeted muscle-tropic capsids designed to transduce both skeletal muscle and cardiac tissue with potency and selectivity in vivo, while avoiding the liver.
Kate’s all-preclinical pipeline features primary programs designed to avoid off-target tissues like the liver, supporting their potential to be one-time safe and effective gene therapies. The company last year licensed to Astellas Pharmaceuticals exclusive rights to develop and commercialize KT430, a treatment designed to deliver a functional copy of the MTM1 gene via a novel MyoAAV capsid to treat X-linked myotubular myopathy (XLMTM).
Other programs in KateTx’s pipeline:
- Duchenne muscular dystrophy (DMD): Kate’s KT809 is designed to deliver a shortened version of dystrophin (microdystrophin) to skeletal muscle and heart.
- Facioscapulohumeral dystrophy (FSHD): Kate’s FSHD program is designed to deliver a microRNA against DUX4 potently and uniformly to skeletal muscles throughout the body, knocking down DUX4 in skeletal muscles.
- Myotonic dystrophy type 1 (DM1): Kate’s DM1 program is in discovery phase.
“We have been highly impressed with the rigor and potential of Kate’s science, and we are confident this acquisition will further enhance our ability to bring forward new therapeutic options for patients living with neuromuscular diseases,” Fiona Marshall, President of Biomedical Research at Novartis, said in a statement.
Neuroscience is one of Novartis’ therapeutic areas, along with cardiovascular, renal, and metabolic disorders; immunology; oncology. Neuroscience is also among Novartis’ disease research areas, along with cardiovascular and metabolic diseases, diseases of aging and regenerative medicine, exploratory disease research, global health, immunology, and oncology.
KateTx was launched last year with a $51 million Series A round co-led by Westlake Village BioPartners and Versant Ventures, as well as the license with Astellas. The Japanese pharma made an undisclosed upfront payment to KateTx and has agreed to pay KateTx payments tied to achieving development, regulatory, and commercial milestone payments, plus royalties on worldwide sales.
Novartis has agreed to pay up to $1.1 billion for KateTx—a sum consisting of an unspecified cash payment that was paid at closing of the transaction and additional payments tied to achieving unspecified milestones.
“We are delighted to bring Kate Therapeutics’ platform technologies and programs together with Novartis scientific expertise and leadership in gene therapy and neuroscience innovation,” Marshall added.
