Early diagnosis and treatment is crucial for patients with primary hyperoxaluria type 1 (PH1). Unfortunately, due to a high prevalence of misdiagnosis, as well as poor therapeutic access in different parts of the world, there is still a high unmet need within this community. However, that may soon change. According to Kentucky Today, biopharmaceutical company BridgeBio Pharma, Inc. (“BridgeBio”) is working on developing BBP-711 for PH1. Recently, BridgeBio shared positive data from a Phase 1 clinical study in healthy volunteers.
If you’re interested in more in-depth data, take a look at the presentation given at the 54th Annual Scientific Meeting of the European Society for Pediatric Nephrology.
BBP-711: An Overview
Developed by BridgeBio, BBP-711 is a small molecule designed to inhibit glycolate oxidase. In addition to PH1, BridgeBio is developed BBP-711 for those with frequent kidney stones. According to BridgeBio:
targeting glycolate oxidase is a clinically validated approach to reduce urinary oxalate by lowering the concentration of glyoxylate.
Clinical Study Data on BBP-711
Altogether, 92 healthy participants enrolled within this Phase 1 clinical trial. Throughout the study, researchers have evaluated the safety, tolerability, pharmacokinetics, pharmacodynamics, and dose of BBP-711. The findings show that:
- BBP-711 was safe and well-tolerated, even in single doses of 3,000mg. While some side effects did occur, they were considered mild in nature.
- Researchers found that the body was able to rapidly absorb BBP-711. This means that patients would only require one dose daily.
- Finally, the treatment option helped reduce glycolate oxidase and increase plasma glycolate levels.
Moving forward, BridgeBio hopes to launch two clinical studies. The first, a Phase 2 trial, would further evaluate BBP-711 for those with frequent kidney stones, creating proof-of-concept. Next, a Phase 2/3 trial would understand the best therapeutic dose of BBP-711, as well as evaluate the efficacy, tolerability, and safety in patients with PH1.
About Primary Hyperoxaluria type 1 (PH1)
Primary hyperoxaluria type 1 (PH1) is a rare disorder which affects the kidneys. AGXT gene mutations cause PH1. Normally, this gene tells the body to make alanine-glyoxylate aminotransferase, a type of enzyme. However, the mutations prevent glyoxylate breakdown, leading to an overaccumulation of oxalate in the body. As oxalate and calcium come together, they can create kidney and bladder stones, as well as cause organ damage. Symptoms associated with PH1 can include:
- Anemia (low red blood cell count)
- Kidney and bladder stones
- Calcinosis (calcium buildup in soft tissues)
- Difficult or painful urination
- Stunted growth
- Skeletal abnormalities
- Inability to control urine / involuntary urination
- Frequent or recurrent urinary tract infections
- Kidney damage
- Renal failure
