Klotho Neurosciences Stock Jumps As FDA Grants Orphan Drug Tag To ALS Gene Therapy

The FDA’s Orphan Drug Designation offers incentives like seven years of U.S. market exclusivity, tax credits for clinical trials, and a waiver of the user fee for its ALS gene therapy.

Retail chatter around Klotho Neurosciences picked up late Thursday after the company said that the FDA had granted Orphan Drug Designation to its ALS gene therapy candidate, KLTO-202.

The designation is reserved for treatments targeting rare diseases and comes with several incentives to support development. 

These include seven years of market exclusivity, tax credits for clinical trials, and a waiver of the generic drug user fee amendments (GDUFA) user fee, independent of existing intellectual property rights.

At the close on Thursday, shares of Klotho Neurosciences rose 6.6% to $1.46, before slipping 6.9% to $1.36 in after-hours trading.

Last month, the company reported efforts to move forward with manufacturing and process development in preparation for clinical trials of KLTO-202. 

Klotho holds rights to a unique RNA splice variant of the human alpha‑Klotho gene, licensed from the Autonomous University of Barcelona. 

This variant produces the secreted form of Klotho protein (s‑KL), known for its neuroprotective properties in the brain and spinal cord.

Animal studies conducted in mouse and non-human primate models of accelerated aging, Alzheimer's disease, and ALS have demonstrated that boosting s‑KL through gene therapy yields therapeutic benefits across all tested models, according to peer-reviewed journals. 

These findings support KLTO-202’s progression into clinical development.

The company expects that process development and manufacturing will take about eight months, followed by four to six months of FDA meetings, mandated animal safety studies, an investigational new drug (IND) filing, and site preparations. 

A single-dose Phase I/II trial in ALS patients is planned for the third quarter of next year. 

In a statement, CEO Joseph Sinkule said that the therapy’s goal is to deliver therapeutic s‑KL concentrations to blood, brain, and muscle tissue.

KLTO-202, which uses a muscle-specific desmin promoter to drive s‑KL expression at the neuromuscular junction, remains unapproved for human use.

On Stocktwits, retail sentiment for Kloto was ‘bullish’ amid ‘high’ message volume.

Kloto’s stock has more than doubled in value so far in 2025.

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