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Targeting Coenzyme A (CoA) genetic disorders

In Memory of Riley

CoA Therapeutics is targeting Coenzyme-A for patients with PKAN, organic acidurias and other diseases of CoA sequestration

About CoA and its Biosynthetic Pathway

Coenzyme A (CoA) plays a crucial role in energy metabolism and is implicated in a large number of disorders, from ultra-rare diseases like pantothenate kinase-associated neurodegeneration, or PKAN, and organic acidurias (OAs), to common diseases such as Type 2 diabetes.

CoA and its related derivatives, which are called thioesters, are used by approximately 4% of cellular enzymes. CoA thioesters regulate the crossroads of energy metabolism. They are involved in some of the most important pathways responsible for breaking down metabolites and building key molecules necessary for healthy physiology. These pathways include the Krebs cycle, fatty acid synthesis and degradation, ketogenesis and steroidogenesis.

CoA Biosynthetic Pathway

The CoA biosynthetic pathway begins with pantothenate (Vitamin B5), which is obtained through diet and the gastrointestinal microbiome. The sole biologic fate of pantothenate is CoA synthesis. Pantothenate is converted by pantothenate kinase (PanK) to 4’-phosphopantothenate, which is rapidly converted into CoA. CoA thioesters inhibit PanK function, providing a feedback mechanism for regulation of CoA levels.

About PKAN

Pantothenate kinase associated neurodegeneration, or PKAN, is the most common form of neurodegeneration with brain iron accumulation (NBIA). PKAN is a devastating disease that typically results in death in early adulthood. Individuals with PKAN typically develop symptoms before age 10 followed by a rapid deterioration in movement, speech and vision associated with neuronal degeneration. PKAN is estimated to affect up to 3 in every 1,000,000 people. There are currently no treatments approved for PKAN.

PKAN is an autosomal recessive genetic disorder caused by mutations in the pantothenate kinase 2 (PANK2) gene. The PanK2 enzyme plays a critical role in the synthesis of CoA in neurons; patients with PKAN either have a complete absence or a large deficiency of the enzyme activity, reducing neuronal CoA levels.

About organic acidurias

Organic acidurias (OAs), including propionic acidemia (PA) and methylmalonic acidemia (MMA), are autosomal recessive metabolic disorders in which normal amino acid metabolism is disrupted, causing a buildup of metabolic intermediates. OAs are associated with slow growth, lethargy, malnutrition, neurological damage and developmental delay. In different OAs, unique metabolic pathways are affected. It is estimated that organic acidurias affect five or six in every 100,000 births. The metabolic intermediates that accumulate in OAs can inhibit PanK enzymes, reduce cellular free CoA levels and thereby impact energy metabolism.

Our Targeted Approach

CoA Therapeutics is developing a novel small-molecule approach to modulate CoA levels by leveraging recent research about the Coenzyme A synthetic pathway. While PanK2 is inactivated in PKAN patients, other isoforms known as PanK1 and PanK3 are still active. This leads to decreased PanK activity and impaired CoA synthesis. Conversely, in organic acidurias, build-up of metabolic intermediates can inhibit the activity of all three isoforms of PanK and impair CoA synthesis. CoA Therapeutics’ small molecules can bind to all three PanK isoforms – although in PKAN, PanK2 is defective – prevent feedback inhibition, and thereby increase PanK activity and increase CoA synthesis.

PKANOrganic Acidurias
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About Us

CoA Therapeutics was founded in 2018 with the aim of developing novel therapies for difficult to treat and rare genetic disorders by harnessing advances in understanding of the CoA pathway.

CoA Therapeutics’ approach and lead compounds are based on breakthrough scientific developments led by Suzanne Jackowski, Charles Rock, Richard Lee and Stephen White at St. Jude Children’s Research Hospital in Memphis, Tennessee.

CoA Therapeutics is led by veteran biotechnology executives whose sole objective is to partner with patients and physicians to bring effective therapies for PKAN and other diseases to market at the soonest possible opportunity.

Management

CS

Christine Siu

CHIEF EXECUTIVE OFFICER, IN RESIDENCE

US

Uma Sinha, Ph.D.

CHIEF SCIENTIFIC OFFICER

AW

Anna Wade, Ph.D.

CHIEF OPERATING OFFICER

AJ

Agnieszka ‘AJ’ Jurecka, M.D., Ph.D.

VICE PRESIDENT, CLINICAL DEVELOPMENT

JP

JENNIFER PAVILLARD

VICE PRESIDENT, REGULATORY AFFAIRS

TF

Tessa Forbes, M.P.H.

SENIOR DIRECTOR, CLINICAL OPERATIONS

Board of Directors

NK

Neil Kumar, Ph.D.

CoA IS A MEMBER OF THE BRIDGEBIO FAMILY

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.

www.BridgeBio.com

News

BridgeBio Pharma Announces Dosing of First Patient in Phase 1 Trial of BBP-671, a Potential Best-In-Class Treatment for Propionic Acidemia (PA) and Methylmalonic Acidemia (MMA)


BridgeBio Pharma Presents Positive Phase 1 Data in Healthy Volunteers, Advancing Development of BBP-671 for Pantothenate Kinase-associated Neurodegeneration (PKAN) and Organic Acidemias


Biomarkers for Drug Development in Propionic and Methylmalonic Acidemias


Research Reveals New Treatment Strategy for Propionic Acidemia


Data Published in Nature Communications Demonstrates CoA Therapeutics’ Potential to Treat Genetically Driven Pantothenate Kinase-Associated Neurodegeneration (PKAN)


BridgeBio Pharma Launches CoA Therapeutics to Target Coenzyme-A for Rare Genetic Disorders

Contact

1800 Owens Street, Suite C-1200
San Francisco, CA 94158

Email
info@coatherapeutics.com

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