Sodium phenylbutyrate/taurursodiol

Sodium phenylbutyrate/taurursodiol (development code AMX0035) is a combination drug being studied for the treatment of amyotrophic lateral sclerosis (ALS),[1] Alzheimer's disease,[2][3] and Wolfram syndrome.[4] It is a fixed-dose combination of two drugs, sodium phenylbutyrate and taurursodiol (tauroursodeoxycholic acid, TUDCA).

Sodium phenylbutyrate/taurursodiol
Combination of
Sodium phenylbutyratechemical chaperone
Taurursodiolbile acid
Clinical data
Other namesAMX0035
Legal status
Legal status
  • Investigational

AMX0035 acts by blocking cell death pathways in mitochondria and in the endoplasmic reticulum.[1] Sodium phenylbutyrate is a chemical chaperone that helps proteins maintain their normal conformation, preventing aggregation that may lead to cell death.[1] Taurursodiol improves mitochondrial energy production.[1]

In a phase 2/3 clinical trial, CENTAUR, for ALS AMX0035 showed increased survival times.[5][6][7][8][9] Based on the results, the FDA requested additional studies be performed before approval would be considered.[10] A phase 3 trial for ALS, PHOENIX, is planned.[11] A phase 2 trial for Alzheimer's disease, PEGASUS, is ongoing.[12]

References
  1. "AMX0035". alsnewstoday.com. Retrieved June 15, 2021.
  2. "AMX0035 to Treat Alzheimer's Disease". alzheimers.gov. Retrieved June 15, 2021.
  3. "AMX0035". alzforum.org. Retrieved June 15, 2021.
  4. "Amylyx Pipeline". amylyx.com. Retrieved June 15, 2021. We are currently exploring the potential of AMX0035 as a treatment option for ALS, Alzheimer’s disease and Wolfram syndrome
  5. Paganoni S, Macklin EA, Hendrix S, Berry JD, Elliott MA, Maiser S, et al. (September 2020). "Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis". The New England Journal of Medicine. 383 (10): 919–930. doi:10.1056/NEJMoa1916945. PMID 32877582.
  6. Paganoni S, Hendrix S, Dickson SP, Knowlton N, Macklin EA, Berry JD, et al. (January 2021). "Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis". Muscle & Nerve. 63 (1): 31–39. doi:10.1002/mus.27091. PMC 7820979. PMID 33063909.
  7. "Novel ALS Drug Continues to Show Survival Benefit". medpagetoday.com. 19 April 2021. Retrieved June 15, 2021.
  8. Paganoni S, Hendrix S, Dickson S, Knowlton N, Macklin E, Cohen J, et al. (13 April 2021). "Long-Term Survival of Participants in the CENTAUR Trial of AMX0035 for ALS (4550)". Neurology. 96 (15 Supplement).
  9. "Long-term survival outcomes of AMX0035 for ALS". 22 April 2021. Retrieved June 15, 2021.
  10. "FDA faces backlash after seeking more data on an experimental ALS therapy". biopharmadive.com. April 14, 2021. Retrieved June 15, 2021.
  11. "Study Design for Phase 3 PHOENIX Trial for AMX0035 in ALS Announced". May 19, 2021. Retrieved June 15, 2021.
  12. "Amylyx Pipeline". Retrieved June 15, 2021.
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