RARE Daily

Cyprium and Sentynl Sign Development and Purchase Agreement for Menkes Disease Therapy

February 24, 2021

Rare Daily Staff

Cyprium Therapeutics and Sentynl Therapeutics entered into an asset purchase agreement to commit development funding for and to acquire Cyprium’s proprietary rights to CUTX-101, its experimental therapy for the treatment of Menkes disease.

Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of copper transporter ATP7A. The minimum birth prevalence for Menkes disease is believed to be 1 anywhere between one in 8,664 and one in 34,810 live male births. Menkes is characterized by sparse and depigmented hair (kinky hair), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, failure to thrive, and neurodevelopmental delays. Mortality is high in untreated Menkes disease, with many patients dying before the age of three. Milder versions of ATP7A mutations are associated with other conditions, including occipital horn syndrome and ATP7A-related distal motor neuropathy. Currently, there is no FDA-approved treatment for Menkes disease and its variants.

CUTX-101 is a subcutaneous injectable formulation of copper histidinate in development to treat Menkes disease. In a phase 1/2 clinical trial, early treatment of patients with Menkes disease with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. In August 2020, Cyprium reported positive topline clinical efficacy results for CUTX-101, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment with CUTX-101, compared to an untreated historical control cohort, with a nearly 80 percent reduction in the risk of death. A Cyprium-sponsored expanded access protocol for patients with Menkes disease is ongoing at multiple U.S. medical centers.

Under the terms of the agreement, Sentynl, a subsidiary of the Zydus Group, will acquire CITX-101 from Cyprium, a Fortress Biotech partner company, for $20 million in upfront and regulatory milestone payments through NDA approval, as well as potential sales milestones plus royalties ranging from the mid-single digits up to the mid-twenties

Cyprium will retain development responsibility of CUTX-101 through regulatory approval, and Sentynl will be responsible for commercialization as well as progressing newborn screening activities. A Joint Steering Committee consisting of representatives from Cyprium and Sentynl will oversee its development. Cyprium will retain 100 percent ownership over any FDA priority review voucher that may be issued at NDA approval for CUTX-101.

“If approved, CUTX-101 will provide a major breakthrough and fill a significant unmet need for children suffering from this rare, fatal pediatric disease,” said Lung Yam, president and CEO of Cyprium.

Rolling submission of a New Drug Application to the U.S. Food and Drug Administration for CUTX-101 is on track to begin in the third quarter of 2021 and to be completed during the fourth quarter of 2021.


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