Collaborative Medicinal Development LLC ("CMD"), a privately-held biopharmaceutical company developing innovative therapies for neurodegenerative diseases, In a presentation, Dr. Craig Rosenfeld, CMD's CEO, reported the results of 24 weeks of treatment with CuATSM at the recommended phase 2 dose. Patients with idiopathic PD showed a marked improvement in disease severity evidenced by a decrease in total UPDRS score of 7 points over 24 weeks. Patients also showed a marked improvement in quality of life evidenced by a decrease in PDQ-39 score of 15 points over 24 weeks; a 5 point change in PDQ-39 is considered the threshold for a clinically meaningful change. In the natural history of PD, both UPDRS and PDQ-39 scores increase (worsen) in correlated fashion over time.
CuATSM is a small synthetic molecule that contains copper.
The metallo-protein Cu/Zn-superoxide dismutase (SOD1) is a ubiquitous enzyme responsible for scavenging superoxide radicals. Mutations in SOD1, which alter its metal binding capacity and can result in protein misfolding and aggregation, have been linked to familial amyotrophic lateral sclerosis (ALS). Cu-ATSM is an orally bioavailable, blood-brain barrier permeable complex that has traditionally been used in cellular imaging experiments to selectively label hypoxic tissue via its susceptibility to reduction by oxygen-depleted mitochondria.
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The starting dose was 12 mg/day, which has been shown to be well tolerated in an ongoing phase 1 pharmacokinetic and dose-finding study of Cu(II)ATSM in patients with ALS (ClinicalTrials.gov identifier NCT02870634).