ALS gene therapy candidate KLTO‑202 wins orphan drug status
Treatment delivers instructions for protein that may lower inflammation
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Klotho Neurosciences’ gene therapy candidate for amyotrophic lateral sclerosis (ALS).
The designation is intended to boost the development of potential treatments for rare diseases, or those that impact fewer than 200,000 people in the U.S. It offers a range of benefits and incentives, such as exemption from certain fees, tax credits for clinical trials, and seven years of market exclusivity upon approval.
“Receiving the orphan drug designation for [KLTO‑202] for the early treatment of ALS underscores the importance of bringing new treatment options to patients suffering from this rare, universally fatal disease,” Joseph Sinkule, Klotho’s CEO, said in a company press release.
KLTO‑202 is an experimental therapy that’s expected to protect nerve cells from damage by delivering the instructions for a protein known as secreted alpha-Klotho (s-KL) that’s found at lower levels in the muscles and spinal cord of ALS mouse models. The protein is believed to reduce inflammation and oxidative stress, that is, an imbalance between the production and clearance of harmful oxygen-containing molecules.
What is KLTO-202?
In KLTO‑202, the gene that codes for s-KL is packaged inside a modified and harmless viral vector called an adeno-associated virus (AAV). The gene is designed to be active only in neuromuscular junctions, the site where nerve cells and muscles communicate, where it’s expected to have myoregenerative properties, meaning it may help repair or rebuild muscle tissue that weakens as nerve cells die.
In animal models of ALS, a gene therapy delivering the gene that codes for s-KL was found to have many therapeutic effects, reducing inflammation and improving muscle strength and coordination. It also delayed the onset of symptoms, slowing their progression, and prolonging survival.
Klotho is finalizing studies that are testing KLTO‑202 in two animal models of human ALS. It’s also started manufacturing KLTO‑202, ahead of planned meetings with U.S. and European regulatory agencies to discuss its clinical development path. The company plans to launch a first-in-human, single-dose trial to evaluate KLTO‑202 in ALS patients by the third quarter of next year.
“My cousin Karen died from this horrific disease,” Sinkule said. “After the FDA’s review of the data leading to the orphan drug designation, we believe this … designation provides strong validation of our science and our approach to treat this disease.”
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