Collaborative Medicinal Development Enrolls First Patient In a Randomized, Placebo-Controlled Clinical Trial of CuATSM For The Treatment of Amyotrophic Lateral Sclerosis

MILL VALLEY,Calif. and MELBOURNE,Australia,Oct. 15,2019 -- Collaborative Medicinal Development,LLC) ("CMD"),a clinical stage biopharmaceutical company with a portfolio of candidates for treatment of neurological and neuropsychiatric diseases,announced today that it enrolled the first patient in a multicenter,randomized,double-blinded,placebo-controlled clinical trial to evaluate the efficacy and safety of CuATSM in subjects with Amyotrophic Lateral Sclerosis ("ALS"). ALS is also known as Motor Neuron Disease ("MND") and Lou Gehrig's Disease. CMD expects to enroll 80 patients across four sites in Australia. The study will assess effects of 6-months treatment with CuATSM compared with placebo on disease severity measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) and on cognitive function measured by the Edinburgh Cognitive and Behavioral ALS Screen (ECAS). Additional details about the trial (NCT04082832) can be found at www.clinicaltrials.gov . Study results should be available in approximately 18 months.

Dominic Rowe,MD,Professor of Neurology at Macquarie University and Coordinating Principal Investigator,stated,"We are excited to have enrolled the first patient in this study. Working together with our patients and the CMD team,we are optimistic that this may be the first effective disease-modifying treatment for ALS."

Craig Rosenfeld,CMD CEO,commented,"CuATSM is the first product candidate from CMD's pipeline and highlights our commitment to developing innovative medicines for neurological diseases with high unmet need."

CuATSM has received orphan drug designation for the treatment of ALS from the FDA Office of Orphan Products Development.

About CuATSM

CuATSM is a first-in-class,orally bioavailable,brain-penetrate small molecule.

The potential of CuATSM for treatment of ALS was first demonstrated in five mouse models of ALS. Positive results in the most aggressive mouse model of ALS were replicated by three independent investigators. A phase 0 clinical trial using 62CuATSM demonstrated target engagement in the motor cortex in patients with ALS. A phase 1/2a open-label study in patients with both sporadic and familial ALS showed that 6-months treatment with CuATSM was associated with compelling results in disease severity assessed by ALSFRS-R score and cognition assessed by ECAS score.

The potential of CuATSM for treatment of Parkinson's disease ("PD") also has been demonstrated in four mouse models of PD A phase 0 clinical trial using 62CuATSM demonstrated target engagement in the substantia nigra in patients with PD. A recently completed phase 1/2a open-label study in patients with PD showed 6-months treatment with CuATSM was associated with marked improvements in disease severity measured by UPDRS score and quality of life measured by PDQ-39 score.

About Amyotrophic Lateral Sclerosis (ALS)

ALS,also known as Motor Neuron Disease ("MND") or Lou Gehrig's disease,is a progressive and fatal neurodegenerative disease characterized by muscle weakness resulting from degeneration of motor neurons. Patients usually die of respiratory failure within 2 to 3 years of symptoms onset. There is an urgent unmet medical need for disease-modifying therapies in ALS. ALS is a rare disease and considered an "Orphan Disease" by regulatory authorities. Further information about ALS is available at https://fightmnd.com/

About FightMND

CMD acknowledges the generous support that FightMND provided for the CuATSM trial in ALS. Rebecca Sheean,Ph.D.,Research Director at FightMND stated "FightMND is dedicated to finding an effective treatment or cure for MND,also referred to as ALS or Lou Gehrig's disease. FightMND's support for CMD to perform a randomized study of CuATSM is consistent with our goals. CMD has demonstrated the potential of CuATSM in the Phase 1-2a study." Jamie Howden,CEO at FightMND added "We are excited by CuATSM as a potential disease modifying therapeutic for patients with ALS. We are grateful for CMD's commitment to join our community in standing up against MND!" More information about FightMND is available at https://fightmnd.org.au/ .

About Collaborative Medicinal Development,LLC

CMD is a privately-held biopharmaceutical company developing innovative therapies for neurodegenerative diseases. The Company's lead drug,CuATSM,was licensed from the University of Melbourne and entered clinical trials in ALS and Parkinson's disease in 2017. For more information about CMD,visit the website at https://colmeddev.com/ .

Contact Information

For further Information

Rory Hill,Business Development

RoryH@colmeddev.com

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