On Rare Disease Day 2024, Bright Path Laboratories shared an amazing step in our commitment to rare diseases. Bright Path successfully completed the development of Amlexanox as a potential treatment for WOREE Syndrome, a devastating rare disease that affects children causing significant disability and pre-mature death, usually before age 4.
WOREE Syndrome is a nano-rare disease, with only 170 patients on the registry and deemed “too rare for industry to care.” Although pharma companies knew the potential of Amlexanox in a variety of rare devastating diseases, it was not tested in these diseases and is not available anywhere in the world. Bright Path stepped in with their groundbreaking technology to solve the problem.
Amlexanox is a small molecule that can read through some nonsense genetic mutations to allow production of critical proteins that have created marked improvement in symptoms and function in a patient with WOREE syndrome, in addition to the drug’s many other applications. The use of Amlexanox in this rare disease was prompted by Johann Mentz, after his daughter, Lucia, was diagnosed with WOREE syndrome as an infant. Mentz, based in Australia, founded the WWOX Foundation and forged ahead with other parents to try to save their children.
After despair and discouragement from the medical community, who offered no viable treatment, Mentz scoured medical journals and uncovered Amlexanox as a potential treatment. He convinced Lucia’s neurologist to prescribe her the drug which showed incredible improvement in her symptoms and functioning. Sadly, after several months, Mentz couldn’t find the drug anywhere, since there were no U.S. manufacturers and the Japanese company manufacturing the drug had also discontinued its sales. Without Amlexanox, Lucia regressed dramatically. Together with his WWOX Foundation, co-founder, Seattle-based Stephen Pratt, who has two sons with WOREE Syndrome, Mentz pressed on to find a way to get Amlexanox to their desperately ill children.
With the assistance of Bruce Bloom, Chief Collaboration Officer at Healx, a Rare Disease AI Techbio Company based in Cambridge, England, the global search for a company to manufacture Amlexanox began. After rejections from some pharmaceutical companies and encountering high prices for the service and long timelines from others, Bright Path stepped in, developing the drug using its AI-enabled continuous flow platform.
The production of Amlexanox is the first step of many towards collaborations like this one bringing together Bright Path Labs, patient advocacy groups, and rare disease partners, such as Rare Remedy Therapeutics, in facilitating treatments and cures for rare diseases. This disease (like many other rare diseases) has a small window of opportunity, so speed of development is critical. Bright Path was able to develop Amlexanox in just two weeks, demonstrating that its advanced manufacturing technology is a fast, efficient, and environmentally safe process for making batches of drug and solving major obstacles in rare disease medicine.
Media Contact and for more information contact Jennifer Burgess at jenniferburgess@brightpathlabs.com.
