History of Encalaret and BridgeBio Pharma
Encalaret is a drug candidate that is being developed by BridgeBio Pharma, a biotechnology company that is focused on finding cures for genetic diseases. Encalaret is currently being studied as a potential treatment for rare endocrine disorders that affect calcium metabolism, including hypoparathyroidism.
The development of encalaret began with the discovery of a molecule called R568 in the late 1990s. R568 was found to bind to a protein called the calcium-sensing receptor (CaSR), which is involved in regulating calcium levels in the body. This discovery led to the development of a series of molecules that were designed to bind to the CaSR and either activate or inhibit its function.
One of these molecules, known as NPS-2143, was found to be a potent inhibitor of the CaSR. It was initially developed as a potential treatment for osteoporosis, but it was eventually discovered that the molecule had the potential to be used to treat other conditions that affect calcium metabolism, including hypoparathyroidism.
In 2014, BridgeBio Pharma was founded with the goal of developing therapies for genetic diseases. The company acquired the rights to NPS-2143 and began developing it as a potential treatment for hypoparathyroidism. The molecule was renamed encalaret, and it underwent extensive preclinical testing to evaluate its safety and effectiveness.
Encalaret was granted orphan drug designation by the US Food and Drug Administration (FDA) in 2018. Orphan drug designation is granted to drugs that are being developed to treat rare diseases, and it provides incentives for companies to develop drugs for these conditions. Encalaret has also been granted orphan drug designation by the European Medicines Agency (EMA).
In 2019, BridgeBio Pharma initiated a phase 2 clinical trial to evaluate the safety and efficacy of encalaret in patients with hypoparathyroidism. The trial is ongoing, and the company expects to report data from the trial in the second half of 2022.
Encalaret has the potential to be a game-changing therapy for patients with hypoparathyroidism. The condition is currently managed with calcium and vitamin D supplements, but these treatments can be ineffective and can cause side effects. Encalaret works by targeting the underlying cause of hypoparathyroidism, which is a deficiency of parathyroid hormone, and could offer patients a more effective and safer treatment option.
BridgeBio Pharma is committed to developing encalaret and other therapies for rare genetic diseases, and the company is working closely with patients, physicians, and advocacy groups to bring these treatments to market. By supporting BridgeBio Pharma and its efforts to develop encalaret, individuals can help promote improved treatment outcomes for patients with rare endocrine disorders.
