ALS Treatment

Developing Treatments for Aggregated Misfolded Protein (AMP) Diseases

Amyotrophic lateral sclerosis (ALS), also called "Lou Gehrig's Disease," is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. The disease is characterized by a progressive degeneration of the motor neurons that link the brain to the spinal cord, and the spinal cord to the muscles in the body. Eventually the neurons die and, with that, the ability of the brain to initiate and control muscle movement is lost. ALS patients eventually lose voluntary muscle action and may become totally paralyzed in the later stages of the disease. There is currently no cure for ALS.

It is believed that, in ALS patients, aggregated SOD1 protein kills the nerves that activate the muscles. SOD1 has a “Jekyll-and-Hyde” nature as it normally plays an important protective role in detoxifying free radicals in the body but, when misfolded, can create lethal oxidative free radicals. Amorfix took on the challenge to develop an agent that only recognizes misfolded SOD1 and leaves the normal protein to do its normal activity.

Only a small fraction of normal SOD1 misfolds and so it should not take much drug to have a benefit. Amorfix started by predicting which parts of the SOD1 molecule would be exposed when it misfolds. The Company then created powerful and specific antibodies to "cap" only the misfolded SOD1. The body then removes the capped SOD1 before it has a chance to trigger misfolding and aggregation of neighboring molecules of normal SOD1. There is a long list of AMP diseases such as Alzheimer's disease, Parkinson's disease and cancer where we can apply this novel therapeutic approach. We are building a pipeline of misfolded protein targets.

Amorfix Dual Approach

Amorfix's technology targets misfolded SOD1 through two approaches, a passive infusion of manufactured monoclonal antibodies and an active immunization approach designed to elicit the production of similar antibodies by the patient's own body. Amorfix's technology is based on the premise that the misfolding and aggregation of SOD1 is a principal agent in the death of neurons that occurs in brain-wasting diseases. Amorfix believes that if misfolded SOD1 can be specifically recognized and its toxic activity neutralized by antibodies, brain-wasting diseases could be effectively treated.

Only Misfolded Proteins are Recognized

Only Misfolded Proteins are Recognized

  1. Normal SOD1 proteins
  2. Misfolded SOD1 proteins
  3. Therapeutic Antibodies
  4. Normal SOD1 protein unaffected and functioning normally

For more information on our ALS therapeutics program, please contact