Post-Makary FDA Signals Rare Disease Flexibility; Read-Through To Our Coverage

Research evidence excerpt

Post-Makary FDA Signals Rare Disease Flexibility; Read-Through To Our Coverage

I N D U S T R Y N O T E

1 8 J u n e 2 0 2 6

• CAMP4 Therapeutics (CAMP, Tenthoff) is pioneering the development of antisense

oligonucleotides (ASOs) to specifically inhibit regulatory RNAs (regRNAs) to upregulate

gene expression to treat genetic diseases. CAMP4 is developing CMP-002, an intrathecally

delivered ASO that targets a specific regRNA to upregulate SYNGAP1 gene expression,

restore protein levels and improve symptoms and cognition. SYNGAP1-related disorders

are a group of neurodevelopmental conditions that can cause severe childhood symptoms

including developmental delays and seizures. At ASGCT, CAMP4 presented that CMP-002

restored SYGNAP1 levels across brain regions in mice and NHPs, and reversed behavioral

and cognitive deficits in models of SYNGAP-related disorders. At TIDES, CAMP4 presented

new preclinical data showing CMP-002 reduced both incidence and severity of seizures in a

SYNGAP1 humanized mouse model. CAMP4 recently submitted the CTA for CMP-002 for

SYNGAP1-related disorders in Australia to start Phase I/II study in 2H:26.

• Capricor (CAPR, Tenthoff) is developing deramiocel, which is composed of cardiac-derived

cardiospheres (CDCs) to treat cardiomyopathy caused by Duchenne Muscular Dystrophy.

Capricor initially received a CRL, but subsequently reported positive Phase III HOPE-3 data,

in which deramiocel met the primary endpoint showing a significant 54% slowing of disease

in PUL 2.0 vs. placebo. (p=0.029) Deramiocel also met the key cardiac endpoint by showing

a 91% slowing of decline in LVEF vs. placebo. (p=0.041) In patients with cardiomyopathy

at baseline, deramiocel improved LVEF by 3.3% vs. placebo, a >100% slowing of decline.