Klotho Neurosciences Provides Shareholder Update Highlighting Recent Milestones as It Prepares to Address $8 Billion Neurodegenerative Disease Market

Rhea-AI Summary

Klotho Neurosciences (NASDAQ: KLTO) provided a strategic update highlighting significant milestones in their development of Klotho-based therapeutics for neurodegenerative diseases. The company is targeting an $8 billion market opportunity across ALS, Alzheimer's, and Parkinson's disease treatments.

Key achievements include receiving FDA Orphan Drug Designation for KLTO-202 for ALS treatment, establishing a manufacturing partnership with AAVnerGene, launching a research center in Okinawa, and securing $11 million in financing. The company has also regained Nasdaq compliance and retained full rights to its Klotho programs following the termination of its SkyBell collaboration.

Positive

• Received FDA Orphan Drug Designation for KLTO-202 in ALS treatment
• Secured $11 million in financing and eliminated all debt
• Regained full Nasdaq listing compliance
• Established strategic manufacturing partnership with AAVnerGene
• Retained full global rights to Klotho programs after SkyBell collaboration termination
• Expanded R&D capabilities with new research center in Okinawa

Negative

• Still in preclinical stage with no products in clinical trials yet
• Operating in highly competitive $8B market against established players

Insights

Biotechnology Investment Analyst

FDA orphan drug designation, manufacturing partnership, and $11M capital raise strengthen KLTO's position in targeting $8B neurodegenerative disease market.

Klotho Neurosciences has achieved several critical milestones that significantly enhance its competitive positioning in the neurodegenerative disease space. The FDA Orphan Drug Designation for KLTO-202 for ALS treatment represents a substantial regulatory advantage, providing tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity upon approval. This designation not only validates the therapeutic potential but also provides financial and competitive benefits that are particularly valuable for smaller biotechs.

The binding agreement with AAVnerGene for manufacturing KLTO-202 addresses one of the most challenging aspects of gene therapy development - production capabilities. This partnership eliminates the need for capital-intensive manufacturing infrastructure while leveraging specialized expertise, potentially accelerating time-to-clinic for their lead candidate.

Financially, the $11 million capital raise combined with debt elimination creates a cleaner balance sheet and extends runway for preclinical development. Regaining Nasdaq compliance removes a significant market overhang and ensures continued access to public markets. Most notably, the company has transitioned to full ownership of its intellectual property following the SkyBell collaboration termination, increasing potential future economics if programs succeed.

The company is targeting three major neurodegenerative conditions with substantial unmet needs: ALS, Alzheimer's, and Parkinson's. While still in preclinical stages with KLTO-202, the focus on accelerating IND-enabling studies indicates potential clinical entry within the next 12-18 months, a critical inflection point for valuation. The expanded research capabilities through the Okinawa center and internal team growth should help mitigate development risks during this crucial phase.

Neuroscience Drug Development Expert

KLTO's orphan designation for ALS therapy and AAVnerGene partnership accelerate development timeline for Klotho-based therapeutic with promising mechanism of action.

The FDA's Orphan Drug Designation for KLTO-202 represents significant regulatory validation for Klotho's approach to ALS treatment. Klotho protein has emerged as a fascinating therapeutic target because of its documented neuroprotective properties and involvement in multiple pathways relevant to neurodegeneration, including oxidative stress reduction, calcium homeostasis, and anti-inflammatory effects.

The partnership with AAVnerGene is particularly noteworthy from a scientific perspective. AAV (adeno-associated virus) vector delivery systems have proven highly effective for CNS gene therapies, with established blood-brain barrier penetration and long-term expression profiles. This collaboration suggests KLTO-202 likely employs an AAV-mediated approach to deliver the Klotho gene or a modified variant to neural tissues - a strategy that could provide sustained therapeutic protein expression following a single administration.

The establishment of a research center in Okinawa is strategically significant beyond geographic expansion. Japan has a rapidly aging population with high neurodegenerative disease prevalence and maintains a regulatory framework that can accelerate approval for therapies addressing age-related conditions. This positions Klotho for potential regional development advantages and access to specialized aging research expertise.

While still preclinical, the company's focus on biomarker development alongside therapeutic advancement reflects modern drug development best practices. Validated biomarkers for Klotho activity could significantly de-risk clinical trials by providing early signals of biological effect. The termination of the SkyBell collaboration, while potentially concerning from a partnership perspective, grants Klotho complete control over development timelines and strategic decisions - critical flexibility for navigating the complex regulatory landscape of neurodegenerative disease therapies.

Key partnerships, regulatory  progress, manufacturing advancements, along with significant capital infusion accelerate the development of Klotho-based therapeutics

NEW YORK, Aug. 18, 2025 /PRNewswire/ -- Klotho Neurosciences, Inc. (NASDAQ: KLTO) ("Klotho" or "the Company"), a biotechnology company advancing Klotho-based therapeutics for neurodegenerative diseases, provides a strategic update to shareholders highlighting the Company's key milestones and pipeline progress. These achievements strengthen Klotho's position as it advances toward the large and growing market for Amyotrophic Lateral Sclerosis (ALS), Alzheimer's disease, and Parkinson's disease therapies, which is estimated at over $8 billion annually by Grand View Research and others.

Over the course of this year, Klotho Neurosciences has achieved critical strategic and operational milestones:

• Pipeline Advancement and Regulatory Recognition: The company received Orphan Drug Designation from the FDA for KLTO-202 for the treatment of ALS, reinforcing the therapeutic potential of its Klotho-based programs to address significant unmet medical needs
• Strategic Partnerships: Klotho entered a binding agreement with AAVnerGene, Inc. to manufacture and develop its KLTO-202 gene therapy candidate using AAVnerGene's platform, accelerating preclinical studies and optimizing delivery of its therapeutic candidates.  The Company has since initiated manufacturing of KLTO-202
• R&D Expansion and Capabilities: The company launched a research center in Okinawa, Japan, and expanded its internal development team, strengthening preclinical research, biomarker development, and operational expertise
• Financial and Market Positioning: Klotho successfully raised over $11 million in financing to advance its programs, eliminating all debt and regaining full compliance with Nasdaq listing requirements, ensuring continued trading and investor confidence
• Independent Control of Pipeline: Following the mutual termination of its collaboration with SkyBell, Klotho retains full global rights to its Klotho programs, positioning the company to pursue development and commercialization on its own terms

Klotho CEO Dr. Joseph Sinkule commented, "2025 has been a transformative year for the Company. We are executing a focused and disciplined strategy to advance our Klotho-based therapeutics from preclinical development toward clinical evaluation, while simultaneously strengthening the Company's operational and scientific foundations and carefully managing our resources. Through strategic partnerships, expanded research capabilities, and the addition of key talent, we have enhanced our ability to drive program development efficiently and effectively. These accomplishments, together with regulatory progress and financial positioning, reinforce our capacity to maximize the potential of our pipeline, expand our technological footprint, and deliver sustained value creation for our shareholders as we continue to advance innovative solutions in neurodegenerative and age-related diseases."

Building on its 2025 progress, the Company is positioned to pursue the following key objectives:

• Accelerate preclinical and IND-enabling studies for KLTO-202.
• Evaluate complementary technologies and acquisitions to enhance pipeline breadth in brain health, organ function, and longevity.
• Continue expanding internal capabilities to strengthen R&D, manufacturing, and clinical readiness.

With these strategic initiatives, Klotho Neurosciences is building on its 2025 successes to advance its leadership in Klotho-based therapeutics and create meaningful long-term value for shareholders.

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 disease, 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. KLOTHO is managed by a team of individuals and advisors who are highly experienced in biopharmaceutical product development and commercialization.

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.

Investor Contact and Corporate Communications:

Jeffrey LeBlanc, CFO
ir@klothoneuro.com 

Website: www.klothoneuro.com

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SOURCE Klotho Neurosciences, Inc.

FAQ

What major milestones did Klotho Neurosciences (KLTO) achieve in 2025?

In 2025, KLTO received FDA Orphan Drug Designation for KLTO-202, secured $11 million in financing, established a manufacturing partnership with AAVnerGene, launched a research center in Okinawa, and regained Nasdaq compliance.

How much funding did Klotho Neurosciences (KLTO) raise in 2025?

Klotho Neurosciences raised over $11 million in financing to advance its programs and eliminated all debt, regaining full compliance with Nasdaq listing requirements.

What is the market size for Klotho Neurosciences' (KLTO) target diseases?

According to Grand View Research, KLTO is targeting an $8 billion annual market for treatments of Amyotrophic Lateral Sclerosis (ALS), Alzheimer's disease, and Parkinson's disease.

What is KLTO-202 and what FDA designation did it receive?

KLTO-202 is Klotho Neurosciences' gene therapy candidate for ALS treatment. It received Orphan Drug Designation from the FDA, highlighting its potential to address unmet medical needs.

What are Klotho Neurosciences' (KLTO) key objectives following the 2025 update?

KLTO aims to accelerate preclinical studies for KLTO-202, evaluate complementary technologies and acquisitions, and expand internal capabilities for R&D, manufacturing, and clinical readiness.