Klotho Neurosciences, Inc. Granted FDA Orphan Drug Designation for KLTO-202 for Treatment of Amyotrophic Lateral Sclerosis ("ALS" or "Lou Gehrig's Disease")

Rhea-AI Summary

Klotho Neurosciences (NASDAQ:KLTO) has received FDA Orphan Drug Designation for KLTO-202, its novel secreted-Klotho promoter gene therapy for treating Amyotrophic Lateral Sclerosis (ALS). This designation provides significant benefits including tax credits for clinical trials, GDUFA User Fee waivers, and 7 years of market exclusivity.

KLTO-202 targets motor neuron diseases using a muscle-specific promoter called "desmin" to express the s-KL gene and protein, specifically delivering therapy to the neuromuscular junction. The company has completed proof of concept studies in two animal models and is initiating manufacturing while planning regulatory meetings with the FDA and EMA.

Positive

• Received FDA Orphan Drug Designation providing 7 years of market exclusivity
• Successful completion of proof of concept studies in two animal models
• Tax credits and fee waivers granted through Orphan Drug Designation
• Targeting a rare disease market with 5,000 new cases annually in the US

Negative

• KLTO-202 is not yet approved for human use by any regulatory authority
• Early stage of development with clinical trials still pending
• Faces challenges in treating a universally fatal disease

Insights

Biotechnology Regulatory Expert

FDA's Orphan Drug Designation for KLTO-202 provides Klotho significant regulatory advantages for their ALS treatment candidate, boosting development prospects.

The FDA's grant of Orphan Drug Designation (ODD) to Klotho Neurosciences for KLTO-202 represents a significant regulatory milestone in the company's ALS treatment development program. This designation offers substantial benefits including tax credits for clinical trial costs, GDUFA user fee waivers, and critically, seven years of market exclusivity upon approval—independent of patent protection.

KLTO-202 (s-KL-AAV.myo) utilizes a novel approach combining a secreted-Klotho promoter with gene therapy delivered to the neuromuscular junction. The therapy aims to address both the neurological damage and offer neuroprotection through therapeutic concentrations of the s-KL protein in blood, brain, and muscle tissues.

The company has completed proof-of-concept studies in two animal models and is initiating manufacturing of the candidate. Their regulatory strategy includes upcoming consultations with both FDA and EMA to establish the development pathway.

For context, ALS affects fewer than 200,000 people in the US with approximately 5,000 new diagnoses annually, qualifying it as a rare disease. The ODD validates Klotho's scientific approach while providing regulatory advantages to accelerate development for this universally fatal condition that currently has limited treatment options.

NEW YORK, July 10, 2025 /PRNewswire/ -- Klotho Neurosciences, Inc. (Nasdaq: KLTO), a gene and cell therapy company focused on the treatment of neurodegenerative and other aging-related diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company's novel secreted-Klotho (s-KL) promoter, gene and delivery system (KLTO-202, or s-KL-AAV.myo) for the treatment of ALS.

The FDA grants Orphan Drug Designation to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. The Orphan Drug Designation provides certain incentives, such as tax credits, toward the cost of human clinical trials and a waiver for the payment of the GDUFA User Fee for market applications. Additionally, Orphan Drug Designation of the product provides the developers seven years of US market exclusivity and independent from the Company's intellectual property protection.

"Receiving the Orphan Drug Designation for s-KL-AAV.myo for the early treatment of ALS underscores the importance of bringing new treatment options to patients suffering from this rare, universally fatal disease" said Dr. Joseph Sinkule, Klotho's Chief Executive Officer. "My cousin Karen died from this horrific disease. We aim to deliver the first gene replacement therapy addressing the neurologic insult resulting in motor neuron damage and the potential neurologic protection induced by providing therapeutic blood, brain, and muscle concentrations of the s-KL protein. After the FDA's review of the data leading to the Orphan Drug Designation, we believe this ODD designation provides strong validation of our science and our approach to treat this disease" concludes Dr. Sinkule.

ALS is sometimes referred to as Lou Gehrig's disease. Lou Gehrig, who played for the New York Yankees for 17 years in the 1920s and 1930s, stunned players and fans by retiring from baseball at the age of 36 after being diagnosed with ALS. Prior to this diagnosis, Gehrig played in a record-breaking 2,130 consecutive games, was referred to as the "Iron Horse," and was considered one of the greatest baseball players of all time. Less than two years later, at the age of 37, Gehrig died of complications from ALS. ALS is also referred to as Motor Neuron Disease in the UK and elsewhere. ALS is considered a rare disease and affects fewer than 200,000 people in the US, with around 5,000 new cases diagnosed each year.

Klotho Neurosciences will have completed "proof of concept" studies in two animal models of human ALS and the Company is currently initiating manufacturing of the ALS-targeted product candidate, followed by meetings with the U.S. FDA and EMA in Europe to concur with the development path going forward.

KLTO-202, the company's lead product candidate targeting motor neuron diseases and muscular dystrophies, is composed of a muscle-specific promoter called "desmin," driving the expression of the s-KL gene transcript and s-KL protein, with targeted delivery of the gene therapy to the neuromuscular junction - the interface between the spinal cord and the muscles. At this time, KLTO-202 is not approved for human use by any regulatory authority.

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.

For more information, please visit the company's website at www.klothoneuro.com.

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

Cautionary Note Regarding 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. Copies of these documents are available on the SEC's website, www.sec.gov. 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 the significance of KLTO receiving Orphan Drug Designation for its ALS treatment?

The FDA Orphan Drug Designation grants KLTO tax credits for clinical trials, fee waivers, and 7 years of market exclusivity for KLTO-202, their ALS treatment candidate.

How does Klotho Neurosciences' KLTO-202 work in treating ALS?

KLTO-202 uses a muscle-specific promoter called desmin to express the s-KL gene and protein, delivering therapy to the neuromuscular junction between the spinal cord and muscles.

What is the current development stage of KLTO-202 for ALS treatment?

KLTO-202 has completed proof of concept studies in two animal models and is initiating manufacturing, with planned regulatory meetings with the FDA and EMA.

How many people are affected by ALS in the United States?

ALS affects fewer than 200,000 people in the US, with approximately 5,000 new cases diagnosed each year.

What regulatory approvals has KLTO-202 received so far?

KLTO-202 has received FDA Orphan Drug Designation but is not yet approved for human use by any regulatory authority.