Pankaj Patel, Chairman Zydus Cadila (2)
Sentynl Therapeutics, US-based specialty pharmaceutical company owned by the Zydus Cadila group on February 24 announced it has entered into an agreement to buy Cyprium Therapeutics’ Copper Histidinate drug candidate CUTX-101 for the treatment of Menkes disease.
Under the terms of the agreement, Sentynl will make $20 million in upfront development and regulatory cash milestones through New Drug Application (NDA) approval in US. Royalties on CUTX-101 net sales ranging from the mid-single digits up to the mid-twenties are also payable. Cyprium will retain 100 percent ownership over any FDA priority review voucher that may be issued at NDA approval for CUTX-101.
In addition, Cyprium will retain development responsibility of CUTX-101 through approval of the NDA by USFDA, and Sentynl will be responsible for commercialisation of CUTX-101 as well as progressing newborn screening activities. Continued development of CUTX-101 will be overseen by a Joint Steering Committee consisting of representatives from Cyprium and Sentynl.
The rolling submission of NDA to USFDA for CUTX-101 on track to
begin in the third quarter of 2021 and to be completed during the fourth quarter of 2021, statement from Zydus said.
"The drug has demonstrated a compelling safety and efficacy profile in topline analysis for the treatment of Menkes disease," said Lung S. Yam, President and Chief Executive Officer of Cyprium.
"We look forward to working with Sentynl to begin the rolling submission of the NDA to the FDA this year. If approved, CUTX-101 will provide a major breakthrough and fill a significant unmet need for children suffering from this rare, fatal pediatric disease,” Yam said.
Cyprium is a partner company of Nasdaq listed Fortress Biotech.
“We are excited to collaborate with Cyprium in order to broaden our pediatric rare disease portfolio and commercialize CUTX-101, a potentially transformative product, for children with Menkes disease, an unmet healthcare need,” Matt Heck, Co-founder, President & Chief Executive Officer of Sentynl, said.
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 in 34,810 live male births, and potentially as high as 1 in 8,664 live male births, based on recent genome-based ascertainment The condition is characterized by distinctive clinical features, including 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 years old. Currently, there is no FDA-approved treatment for Menkes disease and its variants.