Klotho Neurosciences Receives FDA Orphan Drug Designation for KLTO-202 in Treatment of ALS

July 10, 2025

DENVER, Colo., Jul 10, 2025 (247marketnews.com)- Klotho Neurosciences (NASDAQ:KLTO) reported that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for its investigational therapy KLTO-202 (s-KL-AAV.myo) for the treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease.

The designation applies to the company’s novel gene therapy platform utilizing a secreted-Klotho (s-KL) promoter and delivery system. Orphan Drug Designation is awarded to therapies intended for the safe and effective treatment of rare diseases affecting fewer than 200,000 individuals in the United States. It provides several development incentives, including tax credits for clinical trial costs, exemption from certain FDA fees, and seven years of market exclusivity upon approval, independent of any existing intellectual property protections.

ALS, also known as Motor Neuron Disease in parts of Europe, is a progressive neurodegenerative disorder that affects fewer than 200,000 Americans, with approximately 5,000 new diagnoses annually.

Dr. Joseph Sinkule, Klotho’s CEO, stated, “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.”

Klotho Neurosciences has completed proof-of-concept studies in two animal models of human ALS and is currently advancing manufacturing efforts for KLTO-202. The company plans to engage with both the U.S. FDA and the European Medicines Agency (EMA) to define the development pathway forward.

KLTO-202 is Klotho’s lead therapeutic candidate targeting motor neuron diseases and muscular dystrophies. It uses a muscle-specific promoter, “desmin,” to drive expression of the s-KL gene and protein, with targeted delivery to the neuromuscular junction—the critical interface between motor neurons and muscle tissue. KLTO-202 is currently an investigational product and has not been approved for use by any regulatory authority.

About 24/7 Market News
As a pioneer in digital financial market media, 24/7 Market News (24/7MN) is dedicated to the swift distribution of financial market news and information. 24/7 MN incorporates comprehensive corporate communications resources and tools to engage the investment community. Visit 24/7 Market News website here.

24/7 MARKET NEWS, INC Disclaimer
Please go to https://247marketnews.com/disclaimer/ for disclaimer information.

CONTACT:
24/7 Market News
Editor@247marketnews.com

Related news for (KLTO)

NASDAQ and NYSE quotes and data are delayed 15 minutes unless indicated otherwise. Market data and exchange information are provided for informational purposes only and is not intended for trading purposes. Neither 24/7 Market News Editors, 247 Market News, or data and content providers shall be liable for any errors or omissions, delays, misquotes or other market information relayed in any press materials. You should Use Realtime data to conduct due diligence before investing or trading, and trading in any stock is risky you could lose all your money.