Briefing on muscular dystrophy treatment Elevidys: Quick Note

Research evidence excerpt

Briefing on muscular dystrophy treatment Elevidys: Quick Note

Global Markets Research

17 June 2026Chugai Pharmaceutical

4519.T 4519 JP / EQUITY: JAPAN PHARMACEUTICALS

Briefing on muscular dystrophy treatment Rating NeutralElevidys

Target price JPY 8,000Quick Note

Closing price

16 June 2026 JPY 7,523Gene therapy Elevidys aiming to tackle underlying condition while seeking to

ensure safety (Note: Quick Note reports are not a

Chugai Pharmaceutical held a briefing on 17 June to discuss Elevidys, its recombinant vehicle for changes to ratings, target

adeno-associated virus (rAAV) treatment for Duchenne muscular dystrophy (DMD). The prices, or earnings forecasts)

main takeaways were as follows.

(1) Elevidys promotes improvement in muscle function by forcing the expression of the

micro-dystrophin gene in skeletal and cardiac muscles via rAAV. It was granted

conditional three-year approval in May 2025, and was listed in Japan (at a cost of around Research Analysts

¥300mn per patient) on 20 February 2026. The company said it is currently administering Japan pharmaceuticals &

the drug to around 10 patients. healthcare

(2) According to Hirofumi Komaki, director general at the National Center of Neurology Hiroyuki Matsubara, Ph.D. - NSC

and Psychiatry, current treatment of DMD mainly focuses on suppressing symptom hiroyuki.matsubara@nomura.com

progression and managing complications, and there has been demand in clinical settings +81 3 6703 1240

for new drugs that improve abnormalities in the dystrophin gene that cause the condition.

Elevidys is a new drug that deals with this issue, and trials showed an improvement in

motor function versus placebo at 52 weeks. However, following approval in Japan, two

patients died of acute liver failure in US.