Klotho Neurosciences Moves Forward with Manufacturing Gene Therapy for the Treatment of ALS

Rhea-AI Summary

Klotho Neurosciences (NASDAQ: KLTO) announced advancement in manufacturing and process development for KLTO-202, its investigational gene therapy treatment for amyotrophic lateral sclerosis (ALS). The company has licensed a unique RNA splice variant of the human alpha-Klotho gene from the Autonomous University of Barcelona for developing advanced gene therapies.

The company's research has demonstrated that overexpression of secreted alpha-Klotho (s-KL) using gene therapy has shown positive therapeutic outcomes in multiple animal studies, including mouse and non-human primate models. The timeline for development indicates approximately 8 months for manufacturing and 4-6 months for regulatory processes, with Phase I/II clinical trials expected to begin by Q3 2026.

KLTO plans to collaborate with contract research organizations (CROs) to manage manufacturing and clinical trials, maintaining operational efficiency without significant staff expansion. The company aims to use an AAV vector to deliver the s-KL gene directly to motor neurons affected by ALS, a disease that typically leads to paralysis and death within 2-3 years of diagnosis.

Positive

• None.

Negative

• Extended timeline to clinical trials (approximately 12-14 months)
• Complex manufacturing process requiring significant development time
• Multiple regulatory steps needed before trial initiation

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NEW YORK, June 30, 2025 /PRNewswire/ -- Klotho Neurosciences, Inc. (NASDAQ: KLTO) today announced that it is moving forward with manufacturing and process development work in preparation for clinical trials of KLTO-202, its investigational gene therapy for amyotrophic lateral sclerosis (ALS).

A unique RNA splice variant of the human gene called alpha-Klotho has been licensed by the Company from the Autonomous University of Barcelona ("UAB") including patents, patent applications, research, knowhow and other intellectual properties for use in the development of advanced gene and gene-engineered cell therapies.  The human alpha-Klotho gene is located in cells found throughout the human body. It is a five exon gene that produces two protein isoforms: a full-length protein found mainly in cells of the kidney called membrane-bound Klotho (or m-KL), which controls phosphate homeostasis, and a much smaller protein isoform called secreted alpha-Klotho (or s-KL). The s-KL RNA splice variant is found mainly in the brain and spinal cord neurons; the variant protein is neuroprotective by minimizing both oxidative stress and neuroinflammation. Animal studies supported by the Company over the past two years in mouse and non-human primate models of rapid aging, in models of human Alzheimer's disease, and in models of ALS have shown that over-expression and amplification of the tissue levels of s-KL using a gene therapy approach result in highly favorable therapeutic outcomes in every model tested. These results have been published in peer-reviewed scientific journals and now support the transition of KLTO-202 into the clinical development stage.

The Company expects that it will take approximately eight months to complete process development and manufacturing of KLTO-202, and about four to six months to conduct meetings with FDA, complete all FDA-mandated animal safety studies, file an investigational new drug application (IND), train and prepare clinical sites where the Phase I/II studies can be conducted, and then begin the single-dose gene therapy studies in ALS patients by the third quarter of next year. The Company will work with contract research organizations (CROs) to facilitate all activities including manufacturing and clinical trials without the need to hire several dozen staff members, which would significantly increase our operating overhead.

Dr. Joseph Sinkule, the Company's CEO and founder commented: "With our recent fundraising success, we're moving forward with manufacturing the s-KL transgene DNA for KLTO-202. We've identified a more efficient method of producing the AAV vector to deliver the s-KL gene directly to motor neurons—the cells most affected by ALS. Our goal is to increase local s-KL protein levels to protect these neurons from the damage that leads to voluntary and involuntary muscle paralysis and ultimately death."

ALS typically progresses rapidly, with most patients losing mobility, respiratory function, and life within just 2–3 years of diagnosis.

About Klotho Neurosciences, Inc. Klotho Neurosciences, Inc. (NASDAQ: KLTO), is a biogenetics company focused on the development of innovative, disease-modifying cell and gene therapies using a protein derived from a patented form of the "anti-aging" human Klotho gene (s-KL), and its novel delivery systems to transform and improve the treatment of neurodegenerative and age-related disorders such as ALS, Alzheimer's, and Parkinson's disease. The Company's current portfolio consists of its proprietary cell and gene therapy programs using DNA and RNA as therapeutics and genomics-based diagnostic assays. The Company is managed by a team of individuals and advisors who are highly experienced in biopharmaceutical product development and commercialization.

Investor Contact and Corporate Communications - Jeffrey LeBlanc, CFO
ir@klothoneuro.com

Website: www.klothoneuro.com

Forward-Looking Statements:

This press release contains forward-looking statements. These statements are made under the "safe harbor" provisions of the U.S. Private Securities Litigation Reform Act of 1995. These forward-looking statements generally are identified by the words "believe," "project," "expect," "anticipate," "estimate," "intend," "strategy," "future," "opportunity," "plan," "may," "should," "will," "would," "will be," "will continue," "will likely result," and similar expressions. Without limiting the generality of the foregoing, the forward-looking statements in this press release include descriptions of the Company's future commercial operations. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this press release, such as the Company's inability to implement its business plans, identify and realize additional opportunities, or meet or exceed its financial projections and changes in the regulatory or competitive environment in which the Company operates. You should carefully consider the foregoing factors and the other risks and uncertainties described in the documents filed or to be filed by the Company with the U.S. Securities and Exchange Commission (the "SEC") from time to time, which could cause actual events and results to differ materially from those contained in the forward-looking statements.  All information provided herein is as of the date of this press release, and the Company undertakes no obligation to update any forward-looking statement, except as required under applicable law.

 

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SOURCE Klotho Neurosciences, Inc.

FAQ

What is KLTO-202 and how does it work for ALS treatment?

KLTO-202 is a gene therapy that delivers the s-KL gene directly to motor neurons using an AAV vector. It aims to increase local s-KL protein levels to protect neurons from damage that causes muscle paralysis in ALS patients.

When will Klotho Neurosciences (KLTO) begin clinical trials for its ALS treatment?

The company expects to begin Phase I/II clinical trials by the third quarter of 2026, following approximately 8 months of manufacturing and 4-6 months of regulatory processes.

What are the key milestones for KLTO's ALS gene therapy development?

Key milestones include completing process development and manufacturing (8 months), FDA meetings, safety studies, IND filing, and clinical site preparation (4-6 months) before beginning Phase I/II trials.

How is Klotho Neurosciences (KLTO) managing the development of KLTO-202?

KLTO is partnering with contract research organizations (CROs) to handle manufacturing and clinical trials, avoiding the need to hire additional staff and keeping operational overhead low.

What research supports KLTO-202's potential effectiveness?

Animal studies in mouse and non-human primate models of rapid aging, Alzheimer's disease, and ALS have shown favorable therapeutic outcomes, with results published in peer-reviewed scientific journals.