Biogen Acquiring Karyopharm’s Treatment for ALS, Other Neurodegenerative Diseases

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by Alice Melão |

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ALS therapy acquisition

Biogen is acquiring Karyopharm Therapeutics’ KPT-350 and other potential treatments for neurodegenerative diseases such as ALS.

“As a global innovative leader in neuroscience that brings world-class capabilities in developing and commercializing products targeting a broad range of neurological conditions, Biogen is well suited to further advance the development of KPT-350,” Michael G. Kauffman, Karyopharm’s CEO, said in a press release.

KPT-350 targets XPO1, a protein that regulates the transport of other proteins from a cell’s nucleus to the cytoplasm that surrounds it. By preventing proteins from leaving the nucleus, KPT-350 promotes the accumulation of inflammation inhibitors there. This leads to a potent, multifaceted inhibition of inflammation.

Karyopharm said KPT-350 crosses the blood brain barrier, which prevents many nerve cell therapies from reaching the brain, where they are needed. In addition, more KPT-350 crosses the barrier than similar therapies. These features make it an attractive option for treating central nervous system diseases, the company said.

In preclinical-trial studies, KPT-350 reduced inflammation and autoimmunity, or the immune system attacking healthy tissue instead of invaders. It also increased the body’s anti-inflammatory and neuroprotective responses.

Oral KPT-350 was not just effective in animal models of ALS, but also in models of multiple sclerosis, and other neurological and autoimmune disorders, Karyopharm said.

Research has shown that by keeping proteins in the nucleus, compounds like KPT-350 can suppress the neurodegeneration caused by mutations of the C9orf72 gene. These mutations are the most common cause of ALS and familial and sporadic frontotemporal dementia.

Studies have also shown that the neuroprotective effects of compounds like KPT-350 are similar to those offered by C9orf72 inhibitors. This further supports their use as treatments for neurodegenerative diseaeses, researchers have said.

Under their agreement, Karyopharm will receive an upfront payment of $10 million from Biogen. It will also be eligible for payments of up to $207 million based on the treatments it sold achieving development milestones. And it will receive royalties from Biogen on the sales of therapies that obtain regulatory approval.

“This transaction is part of our broader strategy of partnering our non-oncology [non-cancer-therapy] assets while we focus on our primary objective of executing the development of [the cancer therapy] oral selinexor,” Kauffman said.