TOPEKA, IN — The Community Health Clinic, a clinic which specializes in rare diseases, recently dosed the first propionic acidemia patient with an investigational new drug for Propionic Acidemia (PA) and Methylmalonic Acidemia (MMA). The patient was successfully dosed in the second part of BridgeBio Pharma, Inc.’s Phase 1, first in-human clinical trial of BBP-671 in patients with PA and MMA. BBP-671 is an investigational oral therapy being developed as a potential treatment for diseases in which coenzyme A (CoA) metabolism is deficient, including PA and MMA. It is intended to increase coenzyme A (CoA) levels by allosterically modulating pantothenate kinases, key enzymes in the CoA biosynthesis pathway.
The first patient dosed in this portion of the trial has PA. PA is a rare metabolic disorder caused by mutations in genes that impact the development of enzymes that participate in amino acid metabolism, leading to life-threatening metabolic decompensations, as well as long-term complications involving multiple organ systems, including the heart, pancreas, kidney, liver, and brain. The current standard of care for patients with PA or MMA includes dietary restrictions, supplementation, and sometimes liver and/or kidney transplantation but the need to develop a treatment for these conditions remains high due to the long-term and life-threatening impact of these diseases.
“Improving the care of individuals affected by propionic acidemia has been a passion of mine for the last 12 years. We hope that this trial will provide valuable data and ultimately lead to a meaningful therapy for individuals affected by PA and MMA who currently have no approved treatment options. Being part of this clinical trial is key to translating medical innovation to community health care”, said Zineb Ammous, MD, Medical Director of The Community Health Clinic.
“It’s truly exciting for our small clinic in Topeka, Indiana to play a part in this trial, which will have lasting effects for our community, and everyone affected by PA,” said Jared Beasley, R.N., Executive Director of The Community Health Clinic. “We’d like to thank the patient family, Parkview LaGrange Hospital and Topeka Pharmacy for their collaboration.”
More information about the trial can be found on clinicaltrials.gov.
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About The Community Health Clinic The Community Health Clinic (CHC) is a 501(c)(3) non-profit healthcare facility located in Topeka, IN, that provides specialized genetics care consistent with Amish and Mennonite (Plain) values. Since their founding in 2008, The CHC has seen more than 4200 patients and 198 genetic disorders. For more information, please visit www.IndianaCHC.org.