Nerinetide is in clinical development for the treatment of acute ischaemic stroke. Ischaemic stroke is the most common type of stroke. It happens when a blood vessel is blocked by a blood clot, cutting off blood flow to part of the brain (ischaemia). Without blood supply, brain cells can be damaged or destroyed because they may not receive enough oxygen. Symptoms may include numbness or weakness on one side of the body and problems with balance, speech and swallowing. Symptoms may range from mild and resolve, through severe strokes that can lead to long-term disability, coma and death. Early treatment is critical to improve outcomes and aims to restore blood flow to the brain, prevent and possibly repair the damage. Current therapies address only the restoration of blood flow, but not protection of the brain by enhancing its resilience to ischaemia.
Nerinetide is an innovative new drug that protects the brain during an acute ischaemic stroke. As a neuroprotectant, nerinetide does not dissolve blood clots, but effectively pauses the toxic chemical reactions triggered by stroke. Treatment with nerinetide is being positioned to be initiated as soon as possible after the onset of symptoms. If licensed, nerinetide would provide critical time to patients with a stroke by stopping the loss of brain cells until further treatment can be administered.
Tofersen (BIIB067) is in clinical development for the treatment of amyotrophic lateral sclerosis (ALS – also known as motor neurone disease) caused by mutations in the SOD1 gene (SOD1-ALS). ALS is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. ALS is a debilitating and life-threatening disease. The gradual loss of neurons leads to a paralysing effect on muscles used for breathing, which usually leads to death from respiratory failure.