BEL-0218 (Small molecule pharmaceutical chaperone) – Type 3 Gaucher Disease.
Supported by years of safety data, BEL-0218 is a potential breatkthrough treatment for Type 3 Gaucher Disease. Type 3 Gaucher Disease is an orphan indication that is fatal to those suffering from the disease with high unmet need (i.e., BEL-0218 may be the first approved treatment for this fatal condition). Belrose acquired development rights to this program in 2014 and has developed a proprietary oral suspension dosage form.
Belrose’s proprietary suspension formulation of this oral treatment has been designed to improve therapeutic effectiveness and patient benefits. BEL-0218 is a new proprietary, microparticle-based dosage form of Ambroxol that allows patients to take a therapeutically effective dose without having to ingest large handfuls (more than 30 tablets per day) of a capsule or tablet formulation.
Belrose is currently working with the US FDA to confirm the requirements for pivotal clinical trials and other requirements to support approval of BEL-0218 for treatment of Type 3 Gaucher Disease.
BEL-0218 is protected by issued method-of-use patents and US Orphan Disease designation for Type 3 Gaucher Disease, which will provide BEL—0218 with seven years of exclusivity for this indication if granted. Belrose has also filed patent applications to protect BEL-0218’s novel microparticle suspension formulation.
Mechanism of Action / Advantages
BEL-0218 has been shown in pre-clinical studies to efficiently cross the blood-brain barrier and to restore the activity of glucocerebrosidase (“GCase”) in Gaucher disease.
Researchers believe that BEL-0218 acts as a “transporter” helping enzymes and other therapeutic agents to reach the tissue required to have an impact. There are also indications that BEL-0218 helps “re-fold” mis-formed proteins.
Proof of concept human trials have demonstrated therapeutic activity in Type 3 Gaucher Disease, where two out of ten patients treated with a combination of BEL-0218 and Cerezyme® (ERT) showed dramatic clinical improvement.