business development

Strategic partnerships are an integral part of our model. We value collaboration with companies and institutions at the forefront of scientific innovation and we are focused on building robust relationships and alliances in the service of helping patients in need.

We seek academic and industry partners, both within the U.S. and globally, committed to making life-changing medicines as rapidly and safely as possible. Additionally, we are open to discussing portfolio divestments and licensing of pharmaceutical assets that are no longer being actively developed.

Patidegib topical gel

Topical HH Inhibitor (Patidegib) for Gorlin Syndrome

disease

Gorlin syndrome, Frequent basal cell carcinoma

genetic source

PTCH1 (patched-1, a smoothened inhibitor)

patients (us + eu)

120,000

modality

topical small molecule

Program summary

Patidegib topical gel is designed to selectively replace and/or augment the function of Patched (Ptc), a naturally occurring hedgehog signaling pathway inhibitor, by blocking the Smoothened (Smo) protein. Acting as a tumor suppressor, patidegib is designed to allow cells to function normally and limit the overproduction of tumors found in patients with Gorlin Syndrome and High Frequency Basal Cell Carcinoma. Because oral formulations for this treatment typically have serious systemic side effects which cause many patients to discontinue treatment, patidegib is formulated as a topical gel to reduce its side effect profile due to its minimal systemic exposure. BridgeBio is currently investigating the safety and efficacy of patidegib in patients with Gorlin Syndrome in a Phase 3 clinical trial.

Acoramidis

TTR stabilizer for transthyretin amyloidosis (ATTR)

disease

ATTR-CM

genetic source

TTR (transthyretin)

patients (us + eu)

>500,000 (U.S. & EU)

modality

small molecule

Program summary

Acoramidis (AG10) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin (TTR). Acoramidis was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a “rescue mutation” due to its ability to prevent or minimize ATTR in individuals carrying pathogenic, or disease-causing, mutations in the TTR gene. BridgeBio submitted its NDA to the U.S. FDA in 2023 with regulatory filings in additional markets to follow in 2024.