Developing novel and improved therapeutics for patients living with rare diseases

Birch Therapeutics is a privately held pharmaceutical company focused on developing therapeutics for patients affected by rare diseases. Birch is building a drug development pipeline through the repurposing or reformulation of an approved therapeutics or prodrugs design, utilizing the 505(b)2 pathway to accelerate clinical development and reduce development risk.

The company’s lead drug candidate, BRH101, is a small molecule prodrug being developed for the treatment of nephropathic cystinosis, a rare genetic lysosomal storage disorder. We believe BRH101 can potentially address many of the limitations of the currently FDA-approved therapy, cysteamine bitartrate, at therapeutic doses, including anorexia, nausea, vomiting, stomach pain, body odor, halitosis, pill burden and short shelf life.