Cardiomyopathy is a disease of the heart muscle that can impair the heart’s ability to pump blood effectively and may lead to heart failure, arrhythmias, or sudden cardiac death.
Several drugmakers saw shares fall after earnings missed expectations. Alnylam's Amvuttra sales disappointed despite growth; Neurocrine's guidance underwhelmed; AbbVie's Imbruvica and Roche's Vabysmo fell short of consensus.
Alnylam reported Q4 2025 adjusted EPS of $1.25, beating estimates, while revenue of $1.10 billion missed consensus despite 85% growth. Amvuttra sales surged 189% to $826.6 million. The stock fell 4.1% after the mixed results.
A clinical study explores whether echocardiography can detect heart amyloid as an early sign of Alzheimer’s, while laboratory research maps a biphasic gene expression cascade in the brain that identifies new therapeutic targets.
Phase 1 data in 17 adults with Friedreich's ataxia cardiomyopathy showed AAVrh.10hFXN (LX2006) was generally well tolerated and showed signs of biological activity. The gene therapy is being advanced into a larger trial.
Solid Biosciences has secured FDA alignment on a registration pathway for its Duchenne muscular dystrophy gene therapy SGT-003, with 36 patients dosed to date showing no drug-induced liver injury, myocarditis, or other serious adverse events reported with competing therapies.
Prothena reported Q4 2025 adjusted loss of 45 cents per share, meeting estimates, while ending the year with $308.4 million in cash. Roche and Novo Nordisk advanced partnered programs prasinezumab and coramitug into Phase III trials.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07753135 |
Association of Heart Failure Aggravation and Device-based Sensor Parameters |
ACTIVE_NOT_RECRUITING | |
| NCT07738107 |
ATR 1072 in Participants With PRKAG2 Syndrome |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT07674758 |
Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials |
RECRUITING | |
| NCT07617844 |
Multimodal mAgnetic Resonance imaGIng in Cardiovascular Disease |
RECRUITING | |
| NCT07591038 |
PET/CT Imaging in Carriers of TTR Mutations |
NOT_YET_RECRUITING | |
| NCT07574697 |
AIC Genotyping Study |
RECRUITING | |
| NCT07541833 |
Effectiveness and Treatment Patterns of Mavacamten in Patients With Obstructive Hypertrophic Cardiomyopathy in Japan (MANAGE-HCM) |
RECRUITING | |
| NCT07515235 |
DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies |
RECRUITING | |
| NCT07479641 |
A Bioequivalence Study of Two Formulations of HRS-1893 Tablets in Healthy Participants |
ACTIVE_NOT_RECRUITING | PHASE1 |
| NCT07428031 |
French Registry: Late Gadolinium Enhancement in Hypertrophic Cardiomyopathy |
NOT_YET_RECRUITING |
