Alnylam Pharmaceuticals has submitted their clinical trial authorization (CTA) application to the Medicines and Healthcare Products Regulatory Agency (MHRA) for their RNAi therapy, ALN-HSD, a treatment for nonalcoholic steatohepatitis (NASH). If approved, they will begin a phase 1 study in the United Kingdom.
Nonalcoholic steatohepatitis (NASH) is a disease that is characterized by a buildup of fat in the liver that causes inflammation and liver damage. This disease affects those who drink very little or not at all, but it mimics the liver disease of heavy drinkers. The liver damage has the potential to cause enough scar tissue that it prevents the liver from functioning. It affects up to one quarter of the people in the United States.
There is a debate about the cause of NASH. Some healthcare professionals believe that it is inherited, while others think it is triggered by environmental factors. The exact cause is unknown, but there are risk factors that are known to increase the chance of developing NASH. These include obesity, type 2 diabetes, high cholesterol, and metabolic syndrome. NASH is not always symptomatic, and it does not always progress. Symptoms can vary from person to person. Some of these symptoms include weakness, fatigue, weight loss, nausea, vomiting, loss of appetite, jaundice, itching, mental confusion, abdominal pain, swelling in the legs or abdomen, and spider-like blood vessels.
The first step in diagnosing this disease is a blood test. Other tests will follow in order to rule out other conditions. An ultrasound confirms the diagnosis. After a doctor confirms that it is NASH, treatment options include weight loss, lowering one’s cholesterol, avoiding alcohol, controlling one’s diabetes, a healthy diet, and exercise.
About the Application
Alnylam submitted the application to the MHRA in the beginning of August. If approved, they will begin the trial towards the end of the year and partner with Regeneron. It is for ALN-HSD, an RNA1 therapy that targets HSD17B13 in order to treat NASH.
This treatment is administered subcutaneously and utilizes Alnylam’s Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology. It has not yet been evaluated for safety by any agency.
Researchers are eagerly awaiting the MHRA’s decision, as they believe that this treatment has the potential to change disease course. Read more about it here.