According to a story from Market Screener, the biopharmaceutical company Theranexus and the Beyond Batten Disease Foundation (BBDF) have recently signed an agreement to work towards the continued development of a new treatment for Batten disease. The experimental drug will be called BBDF 101. The current agreement is an agreement “in principle” which will allow the company a six month period for the two organizations to reach a final partnership. BBDF has funded and directed years of research to get BBDF 101 where it is so far. Theranexus is committed to the development of therapies that modulate the activity between glial cells and neurons.
About Batten Disease
Batten disease is a nervous system disorder that tends to appear between the ages of five and ten years. This neurodegenerative disease is caused by mutations which affect the CLN3 gene. Girls tend to display symptoms later than boys, but their disease tends to progress more rapidly. Diagnosis is often difficult. Symptoms include seizures, vision problems, repetitive speech, learning regression or delays, scoliosis, decreased muscle and body fat, changes to personality and behavior, poor coordination, and speech loss. Symptoms generally progress over time. There is only one drug for Batten disease that can slow progression, but it is only effective in certain cases. Most treatment is supportive, and the disease is ultimately lethal. There is a dire need for more effective therapies to improve survival times and outcomes for patients with Batten disease. To learn more about Batten disease, click here.
Development and Partnership
Currently the foundation is preparing the submission of an application for an Investigational New Drug (IND) to the US Food and Drug Administration (FDA). The BBDF has also designed protocols for the trial which is currently being negotiated with the agency. If the experimental treatment is able to gain market approval, Theranexus would then take over implementing the marketing process.
BBDF 101 is a unique, proprietary combination of two different compounds that have already been approved that are expected to have a synergistic effect, in which the two drugs work more effectively together than alone. Since the drug contains already-approved components, this could allow it to be approved more quickly if it performs well in trials.
