CMS121 for Alzheimer's Disease

(CMS121 Trial)

Overseen By Brandon Lester

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Virogenics, Inc.

Must not be taking: CYP inducers, QT prolongers

Disqualifiers: Cardiovascular, Psychiatric, Renal, others

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, CMS121, to determine its safety and tolerability in healthy individuals, with the potential to eventually treat Alzheimer's disease. Participants will receive either the drug or a placebo (a pill with no active drug) in varying doses to assess how the body processes it. The trial also aims to understand how food affects the drug's absorption. Generally healthy, non-smoking individuals without major illnesses may be suitable candidates. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in humans.

Will I have to stop taking my current medications?

Yes, participants must stop taking any drugs, including prescription and non-prescription medications, herbal remedies, or vitamin supplements starting 14 days before the first dosing. Additionally, drugs that are significant inducers of certain enzymes must be stopped 28 days before the first dosing.

Is there any evidence suggesting that CMS121 is likely to be safe for humans?

A previous study showed that CMS121 had promising safety results, with no deaths or serious side effects reported. Although the study identified 149 treatment-related side effects, none were severe. The specific side effects were not listed, but the absence of severe or serious outcomes is a positive sign for safety.

CMS121 is under study for Alzheimer's disease due to its potential to address brain inflammation and other issues, as demonstrated in animal studies. While these results do not directly address safety, they suggest the drug functions as intended without causing major problems.

As this is an early phase study, researchers continue to test the treatment carefully to ensure its safety for humans. The results so far provide some reassurance about trying CMS121 in a clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard Alzheimer's treatments, which often focus on managing symptoms or altering neurotransmitter levels, CMS121 targets the disease at a cellular level by addressing oxidative stress, a key factor in neuronal damage. Researchers are excited about CMS121 because it is derived from a natural compound with antioxidant properties, potentially offering a new way to protect brain cells from damage and slow disease progression. Additionally, CMS121's unique mechanism of action could complement existing therapies, providing a more comprehensive approach to managing Alzheimer's disease.

What evidence suggests that CMS121 could be an effective treatment?

Research has shown that CMS121 yields promising results for conditions related to Alzheimer's disease. Studies have found that CMS121 can help reduce weight gain and improve blood sugar and cholesterol levels, which are important for overall health. It has also proven to be safe and well-tolerated in healthy adults, even at higher doses. Evidence suggests that CMS121 might help with memory and thinking problems linked to Alzheimer's, offering hope for its effectiveness. These findings indicate that CMS121 could be a helpful treatment for managing some symptoms of Alzheimer's.

In this trial, participants will receive CMS121 in various treatment arms, including single and multiple ascending doses, to further evaluate its effects and safety profile. ^{2 4 6 7 8}

Who Is on the Research Team?

Scott Rasmussen, MD

Principal Investigator

Celerion

Are You a Good Fit for This Trial?

Inclusion Criteria

Capable of understanding the written informed consent document; willingly provides valid, signed written informed consent; willing and able to comply with the study schedule, requirements, and restrictions.

Continuous non smoker who has not used nicotine containing products for at least 3 months prior to the first dosing.

Body mass index (BMI) ≥ 18.0 and ≤ 32.0 kg/m2 at the screening visit.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Single Ascending Dose (SAD)

Participants receive single escalating doses of CMS121 or placebo

36 days

Multiple visits for dosing and monitoring

Multiple Ascending Dose (MAD)

Participants receive multiple escalating doses of CMS121 or placebo

43 days

Multiple visits for dosing and monitoring

Open-label SAD Cross-over

Participants receive single doses of CMS121 in fed and fasted states to evaluate food effect

36 days

Multiple visits for dosing and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CMS121

How Is the Trial Designed?

Group I: Single ascending dose - CMS121 Experimental Treatment 1 Intervention

Subjects will receive a single oral dose of CMS121 under fed conditions.

Group II: Multiple ascending dose - Elderly cohort - CMS121 Experimental Treatment 1 Intervention

Subjects will receive multiple oral doses of CMS121 once daily (QD) for 7 days under fed conditions.

Group III: Multiple ascending dose - CMS121 Experimental Treatment 1 Intervention

Subjects will receive multiple oral doses of CMS121 once daily (QD) for 7 days under fed conditions.

Group IV: Food Effect - CMS121 Experimental Treatment 1 Intervention

On Day 1 of each of 2 treatment periods, a single oral dose of CMS121 will be administered following either a standard high fat/high calorie meal (Treatment A) or an overnight fast (Treatment B), as per each subject's assigned randomization sequence (AB or BA). There will be a washout of at least 7 days between doses.

Group V: Single ascending dose - placebo Placebo Group 1 Intervention

Subjects will receive a single oral dose of placebo under fed conditions.

Group VI: Multiple ascending dose - Elderly cohort - placebo Placebo Group 1 Intervention

Subjects will receive multiple oral doses of placebo once daily (QD) for 7 days under fed conditions.

Group VII: Multiple ascending dose - placebo Placebo Group 1 Intervention

Subjects will receive multiple oral doses of placebo once daily (QD) for 7 days under fed conditions.

Who Is Running the Clinical Trial?

Virogenics, Inc.

Lead Sponsor

Trials

1

Recruited

100+

National Institute on Aging (NIA)

Collaborator

Trials

1,841

Recruited

28,150,000+

Celerion

Industry Sponsor

Trials

85

Recruited

7,600+

Dr. Susan Thornton

Celerion

Chief Executive Officer since 2010

PhD in Molecular Biology from the University of Pennsylvania

Dr. Lorraine M. Rusch

Celerion

Chief Medical Officer since 2020

MD from an unspecified institution

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11006478/

CMS121: a novel approach to mitigate aging-related obesity ... Results: The CMS121 diet induced a 40% decrease in body weight gain and improved both glucose and lipid indexes. Lower levels of hepatic caspase ...

2. medrxiv.org medrxiv.org/content/10.1101/2025.02.28.25323123v1.full-text

Safety, Pharmacokinetics, and Cardiodynamics of CMS121 ... The effects of food were also evaluated in healthy young adult subjects. SD of up to 1800 mg or RD up to 900 mg/day for 7 days was generally ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05318040

Safety, Tolerability and Pharmacokinetics of CMS121, a ... This is a randomized, double-blind study of CMS121 or placebo given as single and multiple escalating doses in normal healthy subjects.

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40093210/

Safety, Pharmacokinetics, and Cardiodynamics of CMS121 ... The effects of food were also evaluated in healthy young adult subjects. SD of up to 1800 mg or RD up to 900 mg/day for 7 days was generally ...

5. medrxiv.org medrxiv.org/content/10.1101/2025.02.28.25323123v1.full.pdf

Safety, Pharmacokinetics, and Cardiodynamics of CMS121 ... Single ascending oral doses of CMS121 up to 1800 mg appeared to be safe and well tolerated by the healthy young adult subjects in this study.

6. cdn.caymanchem.com cdn.caymanchem.com/cdn/msds/27085m.pdf

Safety Data Sheet Hazards not otherwise classified. There are no adverse physical or health effects known that are not covered by the hazard classes of the.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7394765/

CMS121, a fatty acid synthase inhibitor, protects against ... Here we show that CMS121 alleviates cognitive loss, modulates lipid metabolism and reduces inflammation and lipid peroxidation in the brains of transgenic AD ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S2213231720308533

CMS121, a fatty acid synthase inhibitor, protects against ... Here we show that CMS121 alleviates cognitive loss, modulates lipid metabolism and reduces inflammation and lipid peroxidation in the brains of transgenic AD ...