BRH101 - Next-Generation Therapy for Nephropathic Cystinosis
Nephropathic Cystinosis​
​
-
Rare genetic lysosomal storage disorder caused by mutations in the CTNS gene, which results in an abnormal accumulation of the amino acid cystine that affects 2,000 patients worldwide, with 550 in the United States
-
Cystine excess can lead to kidney failure, muscle wasting, swallowing difficulty, hypothyroidism, cerebral atrophy, photophobia, blindness. Without treatment, children with cystinosis will usually develop end-stage kidney failure or die prematurely
-
Current treatment with approved drugs leads to common gastrointestinal side effects, including anorexia, nausea, vomiting, and stomach pain as well as body odor and halitosis when ingested in therapeutically effective doses​​
-
High pill burden on patients with average daily dose including over a dozen pills, with short shelf life due to air oxidation
BRH101 - A Next-Generation Prodrug for the Treatment of Nephropathic Cystinosis​
​
-
BRH101 is a prodrug of an FDA approved drug with an established safety profile
-
BRH101's novel formulation will decrease cysteamine high blood peak concentrations, leading to 50-90% expected reduction in side effects
-
The improved formulation can lead to a lower number of pills, a smaller size, and a longer shelf life with no distribution restrictions on air oxidation or air temperatures
-
Birch will develop BRH101 through a 505(b)2 filing, one of the faster and less costly FDA pathways towards drug approval