Collaborating with Clinicians to Advance Rare Disease Treatments
Amlexanox: Potential Therapy for Nonsense Mutations in Rare Genetic Diseases
Amlexanox is a FDA-approved oral drug originally approved in Japan as a chronic treatment for adults and children asthma in 1987. It worked because it reduced inflammation, and those taking it had good results and few side effects. Amlexanox is no longer manufactured in Japan, and is not readily available in any other jurisdiction.
Fortuity Pharma is resurrecting amlexanox because it has shown promise in pre-clinical research studies as a potential therapy for those affected by nonsense mutations leading to premature stop codons in rare genetic disorders such as WOREE syndrome, cystic fibrosis, Duchenne muscular dystrophy, Charcot-Marie-Tooth disease, aspartylglucosaminuria, epidermolysis bullosa, and other rare diseases.
Amlexanox works through two primary mechanisms:
Read-through of Premature Stop Codons (PTC)
Amlexanox helps ribosomes bypass premature stop codons caused by nonsense mutations, allowing the ribosome to produce full-length mRNA that can produce full-length proteins that could provide partial or full function. This happens because the drug has the right size and shape to mimic the function of aminoacyl-tRNA. Amelxanox crosses the blood-brain barrier so it can also help rare neurologic diseases.
Inhibition of Nonsense-Mediated Decay (NMD)
Amlexanox also interferes with the NMD process, which typically degrades mRNA that have errors caused by read through of PTCs. By inhibiting NMD, the drug helps preserve the full length mRNA, supporting its ability to generate full length proteins with therapeutic potential in nonsense mutation genotypes in a wide variety of rare genetic disorders.
Join Us in Finding New Uses for Existing Drugs
The Fortuity Pharma team believes clinicians are key partners in bringing novel therapies to the rare disease community. Our lead drug, amlexanox, has the potential to treat a variety of genetic conditions, and we need forward-thinking clinicians to help us evaluate its real-world impact through clinical trials, off-label use, and compassionate access programs.
Clinical Trial Opportunities
We are currently preparing for both community based observational trials and investigator-led proof-of-concept trials at one or more academic institutions, focusing on rare conditions with nonsense mutations leading to premature stop codons. As a clinician, you can contribute by referring eligible patients, participating in our trials, or leading research efforts in your institution. Your expertise is invaluable in determining the therapeutic potential of amlexanox and similar drugs.
Compassionate Use and Off-Label Access
If you are treating patients with rare genetic disorders caused by nonsense mutations leading to PTCs, you may be able to provide them with access to amlexanox through compassionate use or off-label programs. Contact us to learn how you can help your patients benefit from this potentially transformative therapy.
Contact Fortuity Pharma
To learn more about our therapies or explore partnership opportunities, please reach out to us.
We are eager to connect with patients, clinicians, researchers, funders and other potential collaborators.