Zydus subsidiary supported drug data for Menkes disease set to be submitted to USFDA

New Delhi: Cyprium Therapeutics, a Fortress Biotech, subsidiary, with support from its licensing partner Sentynl Therapeutics, a wholly owned subsidiary of Zydus Lifesciences has announced positive data on CUTX-101, copper histidinate (CuHis), in patients with Menkes disease.

The data will be presented as a “Top-Rated Abstract” and Poster at the 2022 American College of Medical Genetics and Genomics (“ACMG”) Annual Clinical Genetics Meeting taking place March 22-26, 2022, virtually and at Music City Center in Nashville, TN.

The previously reported results are from an efficacy and safety analysis of data integrated from two completed pivotal studies in patients with Menkes disease treated with CUTX-101.

CUTX-101 has potential to be first FDA-approved treatment for Menkes disease; rolling submission of New Drug Application to FDA is ongoing and expected to be completed in mid-year 2022.

“The positive data that will be presented at the 2022 ACMG Annual Clinical Genetics meeting demonstrate the efficacy and safety of CUTX-101 and its potential to be the first treatment approved by the U.S. Food and Drug Administration (“FDA”) for patients with Menkes disease. We continue to make progress with our rolling submission of a new drug application (“NDA”) for UTX-101 which we anticipate to be completed in the middle of this year,” said Lung S. Yam, M.D., Ph.D., President and Chief Executive Officer of Cyprium.

In 2021, Cyprium partnered with Sentynl Therapeutics, Inc., a U.S.-based specialty pharmaceutical company owned by the Zydus Group, to bring CUTX-101 to market. Cyprium will retain development responsibility of CUTX-101 through approval of the NDA by the FDA, and Sentynl will be responsible for commercialization of CUTX-101 as well as progressing newborn screening activities.

Zydus’ wholly owned subsidiary Sentynl Therapeutics is focused on empowering patients
suffering from rare diseases. The positive data on CUTX-101, copper histidinate (CuHis) is a
positive step in this direction. The group company recently entered into an asset purchase
agreement for the purchase of BridgeBio’s NULIBRY™ (Fosdenopterin) for Injection to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra rare, life-threatening pediatric genetic disorder.

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

CUTX-101 is in clinical development to treat patients with Menkes disease. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under current good manufacturing practice (“cGMP”) and physiological pH.

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 (ET) with CUTX-101, compared to an untreated historical control cohort, with a nearly 80% reduction in the risk of death.