Cognition Therapeutics Phase 2 Trials Of Zervimesine Show Promise In Treating Dry AMD, Alzheimer's Disease And Dementia With Lewy Bodies

Cognition Therapeutics Inc. CGTX, a clinical-stage company developing drugs that treat neurodegenerative disorders, can add a third indication, dry age-related macular degeneration or dry AMD, to the diseases that its experimental drug zervimesine (CT1812) may be able to treat. 

The company just reported positive topline results of its MAGNIFY Phase 2 clinical trial of zervimesine in adults with geographic atrophy (GA) secondary to dry AMD. 

GA is an advanced type of dry AMD that results in the death of cells in the back of the eye and vision loss that is irreversible. With GA, cells in areas of the retina die, which leads to blind spots and eventually blindness. GA impacts about 1 million people in the U.S. and 5 million globally, and accounts for 20% of all legal blindness worldwide. 

The disease typically presents around the age of 50 and is more prevalent the older people get. Lifestyle choices such as smoking, poor diet and diabetes, among other factors, can speed the progression of this disease. Some 30% of people with dry AMD (about 8 million in the USA) will see their disease eventually progress to GA. There aren't many treatments available, and the ones that are, require doctors to administer monthly or bi-monthly injections in the affected eye (intravitreal) in their clinics.

Orally Delivered Pill Could Hold The Answer 

Zervimesine is different. It's an experimental, orally delivered small molecule drug that is taken once a day. Cognition Therapeutics said topline results in the phase 2 MAGNIFY study showed zervimesine-treated participants had 28.6% slower GA lesion growth on average, and at 18 months, their lesions were 28.2% smaller compared to the placebo. By slowing the growth of GA lesions, zervimesine may prevent further vision loss in people living with dry AMD.

The MAGNIFY study (NCT05893537) was concluded after approximately 100 of the planned 246 participants were enrolled. The company announced in February that the study would conclude in order to redeploy capital into their neurodegeneration studies, and at that time, they indicated they would make the results available. 

According to CGTX executives, the topline results compare favorably to published data from approved injectable complement inhibitors currently sold by Astellas and Apellis. Of the trial participants, about two-thirds of patients completed 12 months of dosing and one-third completed 18 months of dosing. 

"These results in dry AMD represent yet another indication in which zervimesine has potential to slow the progression of disease with a once-daily oral pill," says Anthony O. Caggiano, MD, PhD, Cognition Therapeutics' chief medical officer and head of research and development. "Compared to current treatment options, which require regular clinic visits for intravitreal injections, an effective oral treatment that patients can take at home to protect both eyes, would be truly transformative."

Third Indication For Zervimesine 

The positive results of the trial for treating GA marks the third indication in which zervimesine appears promising. Topline results from the company's exploratory phase 2 SHIMMER study for people who have dementia with Lewy bodies (DLB) demonstrated strong therapeutic responses across behavioral, functional, cognitive and movement measures. 

DLB is a progressive form of dementia in which symptoms can fluctuate in severity. The condition leads to a decline in thinking, reasoning and independent movements. It can cause uncontrollable changes 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, based on average total Medicare cost of care per patient. 

The double-blind, placebo-controlled phase 2 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 patients receiving zervimesine had fewer or less severe hallucinations and delusions and less anxiety and agitation than placebo-treated patients. Zervimesine-treated patients also demonstrated an 86% improvement in these behavioral symptoms, as measured by the Neuropsychiatric Inventory (NPI) scale, compared to placebo. This improvement in patient symptoms was accompanied by a notable reduction in caregiver distress, with caregivers of zervimesine-treated patients reporting significantly less stress related to managing these challenging behaviors, the company reported. These symptoms are a hallmark of DLB and can be debilitating for patients. 

Alzheimer's Disease Also On The List 

Then there's Cognition Therapeutics' SHINE study, which tested zervimesine in 153 people with mild-to-moderate Alzheimer's disease. According to the company, the phase 2 clinical trial showed that participants with lower levels of a protein called p-Tau217 in their blood  experienced a robust treatment effect, with 95% slowing of cognitive decline on the ADAS-Cog11 scale compared to the placebo.

About 7.2 million Americans 65 and older have Alzheimer's, which is characterized by memory loss, cognitive decline and changes in behavior. As the population ages, the cases of Alzheimer's are projected to grow at a rapid rate. By 2050, if nothing is done, about 12.7 million people in the U.S. 65 and older may develop this disease. 

"Dry AMD is now the third indication in which we have shown efficacy signals with a once-daily oral drug," said Lisa Ricciardi, Cognition Therapeutics' president and CEO. "We believe zervimesine has the potential to be used as a monotherapy or in combination with existing medications. This would allow physicians the flexibility to tailor treatment regimens for their patients. Importantly, it would also allow patients who are not appropriate for injectables to have access to treatment. With the right partner and development plan, we believe zervimesine could be a treatment breakthrough in these large, underserved patient populations."

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