Takeda Pauses Narcolepsy Studies, Forges $1.1 Billion Gene Therapy Deal

Takeda_Tomohiro Ohsumi/Getty Images

Tomohiro Ohsumi/Getty Images

A safety concern has forced Takeda Pharmaceutical to suspend the dosing of patients in two Phase II studies of an experimental treatment for narcolepsy.

This morning, the company said it halted the studies of TAK-994, an investigational oral orexin agonist assessed for narcolepsy. While the company did not identify the safety concern in its brief announcement, Takeda said halting the trials early will provide the company time to interpret the benefit/risk profile of the experimental medication to determine the next steps in the developmental program.

Two years ago, the company presented proof of concept and Phase I data that showed the experimental treatment was well-tolerated at all dose levels.

Sarah Sheikh, head of Takeda’s Neuroscience Therapeutic Area Unit, said patient safety is of the utmost importance as the company develops treatments for neurologic and psychiatric diseases.

“Takeda is committed to bringing innovative, safe and effective treatments to patients with narcolepsy. We are working to quickly assess the totality of available data to inform the further development of TAK-994,” Sheikh said in a statement.

In addition to TAK-994, the company is also advancing TAK-861, another oral orexin agonist. That asset is assessed in a Phase I study.

For Takeda, the trial halt came one day after the company forged a gene therapy agreement with Selecta Biosciences valued at $1.1 billion. Selecta and Takeda partnered to develop gene therapies for two indications within the field of lysosomal storage disorders. The companies intend to leverage Selecta’s ImmTOR platform to enable the potential redosing of gene therapies.

Carsten Brunn, Ph. D., president and chief executive officer of Selecta, said the companies hope to overcome existing barriers with adeno-associated virus-driven gene therapies. They also aim to address immunogenicity constraints and unmet patient needs.

“Together, we look forward to overcoming barriers to current efforts in AAV-driven gene therapy, as well as striving to address immunogenicity constraints and unmet patient needs. This collaboration provides additional validation and further demonstrates the robust value of our ImmTOR platform, which may enable redosing of potentially life-saving gene therapies. We are excited to expand our growing pipeline with Takeda and build on the momentum of our rapidly advancing proprietary gene therapy programs,” Brunn said in a statement.

Selecta’s ImmTOR platform has the potential to amplify the efficacy of biologic therapies, including potential redosing of gene therapies. According to the company, the platform is designed to mitigate unwanted immune responses, allowing for multiple dosing and longer responses.

Madhu Natarajan, head of Takeda’s rare diseases drug discovery unit, expressed hope in the ImmTOR platform. The potential of ImmTOR could have “broad applicability across our gene therapy programs for a range of diseases,” Natarajan said in a statement.

Under terms of the collaboration, Takeda made an undisclosed upfront payment to Selecta. Over the course of the agreement, Selecta could earn up to $1.124 billion in developmental and commercial milestone payments. Selecta is also eligible for tiered royalties on future commercial sales.

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