AdComm Scheduled for Deramiocel on July 29th; PDUFA Date August 22nd

研报英文原文证据摘录

AdComm Scheduled for Deramiocel on July 29th; PDUFA Date August 22nd

Price Performance - 1 Year

was based on the positive Phase III HOPE-3 data, which randomized 106 ambulatory USD

and non-ambulatory DMD boys (1:1) to either 150 million deramiocel cells q3M (N=54) 40

or placebo (N=52). The majority (78%) of boys in HOPE-3 had cardiomyopathy at 35

baseline and ~90% were on cardiac medication. Deramiocel met the primary endpoint 30

showing a significant 1.2 point absolute difference or 54% slowing in PUL 2.0 of disease 25

vs. placebo. (p=0.029) Deramiocel also met the key cardiac endpoint by showing an 20

absolute 2.4% LVEF improvement, representing a 91% slowing of decline vs. placebo. 15

(p=0.041) In patients with cardiomyopathy at baseline, deramiocel improved LVEF by 10

3.3% vs. placebo, a >100% slowing of decline. Deramiocel was safe and well tolerated. 5

Jun-25 Aug-25 Oct-25 Dec-25 Feb-26 Apr-26 Jun-26

• Lawsuit Against Nippon Shinyaku. In May, Capricor filed a lawsuit against distribution Source: Bloomberg

partner Nippon Shinyaku and U.S. subsidiary NS Pharma in the Superior Court of

New Jersey, seeking to regain full U.S. distribution rights to deramiocel. Capricor and

NS have been unable to negotiate a reasonable transfer price, which NS would pay

Capricor before selling deramiocel. Capricor asserts that NS has not made meaningful

efforts to prepare for U.S. launch. NS has also sub-contracted other distributors without

written consent from Capricor. NS ultimately responded to Capricor's requests with

an amendment to the agreement that would require Capricor to surrender deramiocel

entirely. Refusing this, Capricor filed the lawsuit to regain full control of deramiocel.

We see upside if the lawsuit is successful as Capricor is currently eligible for 40% of