Armed With Donation From Trial Participant's Family, Cognition (Nasdaq: CGTX) Initiates Expanded Access Program For Experimental DLB Drug

Cognition Therapeutics Inc. CGTX, a clinical-stage company developing drugs that treat neurodegenerative disorders including Dementia with Lewy Bodies (DLB), just received assistance in those efforts in the form of an anonymous philanthropic donation, which the company says will "substantially" fund an expanded access program for people who participated in the trial. An expanded access program enables people suffering from serious and/or life-threatening illnesses to access drugs in the development stage outside of a clinical trial.

When speaking of serious diseases, DLB certainly falls into that category. DLB is a form of dementia in which symptoms come on and progress quickly. DLB can quickly lead to a decline in thinking, reasoning and independent movements. It can also cause uncontrollable fluctuations in alertness, recurring hallucinations, sleep disruptions, tremors and slow movement. It impacts about 1.4 million people in the U.S. and is the costliest form of dementia. As it stands, there are no approved therapies for DLB, and treatments aimed at reducing symptoms for the debilitating form of dementia can be invasive and burdensome. Zervimesine (also known as CT1812) is Cognition Therapeutics’ experimental drug, which is an orally delivered small-molecule oligomer antagonist to treat this progressive form of dementia.

Protecting And Slowing Damage 

Cognition believes zervimesine shows promise in protecting neurons in the brain and retinal cells in the eye by binding to the sigma-2 receptor complex and restoring cellular damage response processes such as autophagy, cholesterol synthesis and protein trafficking. A large percentage of DLB patients have both Aβ and α-synuclein oligomers present in the brain. Zervimesine has the potential to slow the progression of both Alzheimer's disease and DLB by acting to prevent the buildup of both the toxic Aβ oligomers that drive Alzheimer's disease and the harmful α-synuclein oligomers responsible for DLB, reports Cognition. As a potential once-daily oral medication, Cognition said zervimesine represents a treatment paradigm shift from invasive and burdensome injections or infusions to convenient and comfortable therapy which can be taken at home. 

The donation comes from the family of a DLB patient who took part in Cognition's phase 2 SHIMMER study and was treated with zervimesine. With the open-label expanded access program or EAP, initially, 30 patients will take 100mg of zervimesine orally for one year. The EAP will be open to eligible SHIMMER participants who completed the phase 2 study, as well as additional patients with a diagnosis of mild-to-moderate DLB who meet the criteria for this program. 

The first of eight sites for the EAP to be activated is Banner Sun Health Research Institute in Arizona. Dr. David Shprecher will serve as the primary investigator at the site. Meanwhile, Dr. James E. Galvin will act as lead investigator for the multi-center, open-label EAP. Dr. Galvin is the director of the Comprehensive Center for Brain Health at the University of Miami Miller School of Medicine and was the study director and principal investigator on the SHIMMER study grant from the National Institute on Aging.

"At Cognition, our ultimate goal is to create a therapy that changes lives. We are moving as rapidly as possible to onboard participating sites so that we can begin providing zervimesine to eligible patients this month," said Lisa Ricciardi, president and CEO of Cognition Therapeutics. "Throughout the SHIMMER study, we have enjoyed a collaborative relationship with Drs. Galvin and Shprecher and their staff. Their commitment and that of the Cognition team has been instrumental in launching the EAP so rapidly. Cognition would like to extend our sincere thanks to the benefactor and all stakeholders who made this program a reality."

Study Results Hold Promise 

The drug’s phase 2 SHIMMER study resulted in positive topline results, and the company recently announced that zervimesine produced strong therapeutic responses across behavioral, functional, cognitive and movement measures in DLB patients.

The double-blind, placebo-controlled SHIMMER study had 130 adults enrolled who were evenly randomized to receive either a placebo or a daily dose of zervimesine for six months. Cognition Therapeutics reported the study met its primary endpoint of safety and tolerability. 

What's more, zervimesine-treated DLB patients scored an average of 86% better than placebo-treated patients on the neuropsychiatric inventory (NPI) A-L at the end of the study. This tool describes the frequency and severity of 12 separate behavioral symptoms. 

The SHIMMER trial patients receiving zervimesine also had fewer or less severe hallucinations and delusions and less anxiety and agitation than placebo-treated patients.

"As a physician, it's always rewarding when you are able to offer a medication to a patient that may make a meaningful impact on their health," said Glavin. "To have touched the anonymous donor's life so meaningfully that they felt compelled to support an expanded access program for so many people is humbling and rewarding. This program is a unique opportunity, and one that my colleagues and I are excited to be involved in."

DLB is a crushing disease with no FDA-approved treatments. Cognition Therapeutics wants to change that. Thanks to this new, anonymous donation, the company is much closer to expanding access to this potentially life-saving drug. 

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