In short
Surlorian (also known as ARM210 or S48168) is an investigational medicine being developed for autosomal dominant RYR1-related myopathy, a rare inherited condition that causes muscle weakness. It is a chemically synthesised substance given as a film-coated tablet by mouth. One phase 2 trial is currently authorised in the EU, planning to enrol 48 adults, and it is testing whether surlorian improves muscle strength and reduces fatigue compared with placebo.
Key points
- Surlorian is an investigational medicine for autosomal dominant RYR1-related myopathy.
- It is given as a film-coated tablet taken by mouth.
- The authorised trial is a phase 2 study comparing surlorian with placebo.
- The trial plans to enrol 48 adults with RYR1-related myopathy.
- The main measure is the 1-minute sit-to-stand test, which assesses muscle strength and fatigue.
- Surlorian has no marketing authorisation in the product records.
What is surlorian?
Surlorian, also known by its development codes ARM210 and S48168, is an investigational medicine being studied for adults with autosomal dominant RYR1-related myopathy. It is a chemically synthesised substance, meaning it is made through chemical processes rather than being derived from living cells. The product records list no marketing authorisation, so surlorian is not yet approved as a medicine in the EU. The company named in the product records is RyCarma Therapeutics, Inc.
How does surlorian work?
The trial registry data and the available sources do not describe the molecular mechanism of surlorian in detail. The condition it targets, RYR1-related myopathy, is caused by changes in the RYR1 gene, which provides instructions for a protein called the ryanodine receptor 1. This protein plays a role in muscle function. The trial is designed to assess whether surlorian improves muscle strength and decreases muscle fatigue in people with this condition.
Which conditions is it studied for?
- Autosomal dominant RYR1-related myopathy — a rare inherited muscle disease caused by changes in the RYR1 gene. People with this condition may be slower to walk or stand, have trouble walking and climbing stairs, and experience muscle weakness. In more serious cases, they may also have a curved spine (scoliosis), problems moving their eyes, and trouble breathing because their muscles are weak.
- Heart failure with reduced ejection fraction — a planned phase 2 trial listed in an external source would study surlorian in people with heart failure and RYR1-related myopathies.
How is surlorian given?
Surlorian is given as a film-coated tablet for oral use, meaning it is taken by mouth. The authorised trial uses a 300 mg dose taken orally.
What do the clinical trials show?
One phase 2 trial is authorised in the EU Clinical Trials Information System. It is a randomised, double-blind, placebo-controlled trial, meaning participants are assigned by chance to receive either surlorian or placebo, and neither they nor the researchers know which one they are receiving. The trial plans to enrol 48 adults with autosomal dominant RYR1-related myopathy.
The main measure is the change in the 1-minute sit-to-stand test (1-MSST) after approximately 28 days of dosing, comparing active treatment with placebo. This test measures how many times a person can stand up from a chair in one minute, reflecting muscle strength and fatigability.
The planned duration of the trial is approximately 16 weeks. It includes a screening period of up to 28 days, two treatment periods of 28 days each, a washout period of 21 days, and a follow-up visit 7 days after each treatment period. Participants have equal chances of receiving surlorian or placebo first. An optional open-label extension phase of up to 12 months is planned for participants who enrol into that phase.
What side effects are known?
The available sources do not report specific side effects of surlorian. The trial is designed to assess the safety and tolerability of surlorian in study participants, but no results on side effects are provided in the registry data or the supplementary sources.