Bocidelpar

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Bocidelpar or ASP0367 or MA-0211 is an experimental drug that may improve mitochondria and endothelial function.[1] Bocidelpar is undergoing clinical trials under the name ASP0367 for Duchenne Muscular Dystrophy, ME/CFS, hypoxia, kidney failure and mitochondrial myopathies (which result in mitochondria dysfunction).[1][2][3] Bocidelpar was developed by Astrella Pharma's subsidiary Mitobridge and is being fast tracked by the FDA.[1][3]

Theory[edit | edit source]

Bocidelpar is investigated to determine whether it can turn on the Peroxisome Proliferator-Activated Receptor delta (PPAR-δ) pathway, which is the pathway that turns on different genes in order to regulate mitochondria.[1] The theory is that turning on this pathway will improve muscle function at a cellular level.[4]

Evidence[edit | edit source]

The first clinical trial for ME/CFS is trial NCT04855201, which has not yet published any results. The trial involves 40 adults aged between 18 and 60, and is randomized clinical trial with quadruple blinding.[2]

Clinicial trial results will look at oxygen uptake changes, exercise test values for aerobic exercise including measuring change in aerobic capacity and anaerobic threshold changes, with cardiopulmonary exercise testing being one of the tests carried out.[2]

Clinicians[edit | edit source]

David Systrom is helping conduct the current clinical trial of bocidelpar at Brigham and Women's Hospital, Boston, United States.[5]

Risks and safety[edit | edit source]

Unknown.

Costs and availability[edit | edit source]

See also[edit | edit source]

Learn more[edit | edit source]

References[edit | edit source]

  1. ↑ 1.0 1.1 1.2 1.3 Mitobridge (Astellas). "ASP0367 (MA-0211)". Parent Project Muscular Dystrophy. Retrieved April 25, 2022.
  2. ↑ 2.0 2.1 2.2 Astellas Pharma Global Development, Inc. (April 14, 2022). "A Phase 1b Multiple Oral Dose Study to Evaluate the Pharmacological Effect, Safety and Tolerability of ASP0367 in Participants With Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction".
  3. ↑ 3.0 3.1 "U.S. FDA Grants Fast Track Designation for ASP0367/MA-0211, a Selective PPARδ Modulator Being Developed for the Treatment of Primary Mitochondrial Myopathies" (PDF). Astrella Pharma. Retrieved April 25, 2022.
  4. ↑ "Duchenne Drug Development Pipeline". Parent Project Muscular Dystrophy. Retrieved April 25, 2022.
  5. ↑ Johnson, Cort (April 23, 2022). "$8 Million Clinical Trial of a Mitochondrial Booster Underway in ME/CFS". Health Rising. Retrieved April 25, 2022.

mitochondria Important parts of the biological cell, with each mitochondrion encased within a mitochondrial membrane. Mitochondria are best known for their role in energy production, earning them the nickname "the powerhouse of the cell". Mitochondria also participate in the detection of threats and the response to these threats. One of the responses to threats orchestrated by mitochondria is apoptosis, a cell suicide program used by cells when the threat can not be eliminated.

δ / Δ. Greek letter delta (a symbol used in science). Equivalent to a "d".

δ δ / Δ. Greek letter delta (a symbol used in science). Equivalent to a "d".

blinded trial A clinical trial is blinded if either the participants or the researchers don't know which treatment group they are allocated to until after the results are interpreted. (Learn more: www.nottingham.ac.uk)

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