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OUR COMPANY

Amylyx is a Cambridge-based pharmaceutical company dedicated to the development of therapeutics for the treatment of neurogenerative disorders. Guided by our core values, we incorporate unconventional approaches through strong partnerships with industry leaders, scientists, doctors and organizations. We work collaboratively across everything we do to positively impact the lives of patients and their families.

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OUR STORY

“Why do neurons die?” Those four words, asked in a dorm room at Brown University in 2013, began the Amylyx journey to develop a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS). In September 2020, the New England Journal of Medicine (NEJM) published data demonstrating positive results for the company’s oral drug candidate for ALS, AMX0035, in the pivotal CENTAUR trial.

AMYOTROPHIC LATERAL SCLEROSIS (ALS): KEY FACTS

  • Relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord1,2
  • Most commonly diagnosed later in life (middle to late 50’s), but can be diagnosed in early adult years1
  • Many people with ALS will die from respiratory failure within 3 years of symptom onset3,4
  • Many of the pathways implicated in ALS converge on the mitochondria and endoplasmic reticulum5,6
  • Despite currently available therapies, ALS remains a rapidly progressive disease and leaves an unmet need for people with ALS1

REFERENCES: 1. Brown RH, Al-Chalabi A. Amyotrophic lateral sclerosis. N Engl J Med. 2017;377(2):162-172. 2. Al-Chalabi A, et al. Amyotrophic lateral sclerosis: moving towards a new classification system. Lancet Neurol. 2016;15(11):1182-1194. 3. Traxinger K, et al. Prognosis and epidemiology of amyotrophic lateral sclerosis: analysis of a clinic population, 1997-2011. Neurol Clin Pract. 2013;3(4):313-320. 4. Labra J, et al. Rate of disease progression: a prognostic biomarker in ALS. J Neurol Neurosurg Psychiatry. 2016;87(6):628-632. 5. Hardiman O, et al. Amyotrophic lateral sclerosis. Nat Rev Dis Primers. 2017; 3:17085. doi: 10.1038/nrdp.2017.85. 6. Manfredi G, Kawamata H. Mitochondria and endoplasmic reticulum crosstalk in amyotrophic lateral sclerosis. Neurobiol Dis. 2016;90:35-42.

INSIDE ALS: THE NEURONS BEHIND THE DISEASE

Learn how endoplasmic reticulum and mitochondrial dysfunctions contribute to ALS pathophysiology.

ALS: NEW PATHWAYS. NEW PROGRESS.

Learn more about ALS disease progression and severity, as well as the latest understanding of disease pathophysiology. This resource also reviews AMX0035 mechanism, CENTAUR trial design, and the importance of ALSFRS-R as an ALS endpoint.

ALSFRS-R: A CRITICAL ALS ASSESSMENT

ALSFRS-R (ALS functional rating scale-revised) is a critical measure of functional ability in people living with ALS, assessing 4 categories using a point-based scale: gross motor function, fine motor function, bulbar function, and respiratory function. Loss of a single point can reflect severe limitations to a person’s daily activities.

PIPELINE

Amyotrophic Lateral Sclerosis (ALS) PH. 2
Alzheimer’s Disease PH. 2
Wolfram Syndrome
 
AMX0035AMX0035
Preclinical ValidationPreclinical Validation
IND Enabling StudiesIND Enabling Studies
Phase 1Phase 1
Phase 2Phase 2
Phase 3Phase 3

ABOUT AMX0035

AMX0035 is our investigational product, a fixed dose co-formulation of two active compounds, sodium phenylbutyrate (PB) and Taurursodiol optimized to address both the toxic, unfolded proteins in the endoplasmic reticulum and the energy crisis in the mitochondria. We are currently exploring the potential of AMX0035 as a treatment option for patients with ALS and Alzheimer’s disease. Our aim is to expand upon this innovation to address other neurodegenerative diseases.

AMX0035 is an investigational product and its safety and efficacy have not been established by the FDA. The materials on this webpage are intended to provide scientific information regarding AMX0035 and the results of the CENTAUR trial.

SCIENCE

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