Where hypertrophic cardiomyopathy treatment is heading

Hypertrophic cardiomyopathy (HCM) is an inherited thickening of the heart muscle that affects about 1 in 500 people, though most are undiagnosed; tens of thousands are newly diagnosed in the US each year. It can cause breathlessness, chest pain, atrial fibrillation and, rarely, sudden death. Cardiac myosin inhibitors (mavacamten, aficamten) were the first drugs designed for the disease, and they are reducing the need for surgery. Gene therapies aimed at the underlying mutations are in early trials.

• 121studies open or running
• 12phase 3 trials
• 1,516papers published this year
• 40NIH-funded projects

1. Now

   Nov 2026

   A second myosin inhibitor approvedHappened 1.8 ml/kg/min exercise gain

   Aficamten raised peak oxygen uptake 1.8 ml/kg/min vs placebo; FDA approved December 2025.

   1.8: ml/kg/min gain in peak oxygen uptake, vs 0.0 with placebo.

   • SEQUOIA-HCM trial

   Better than a beta-blocker as first treatmentHappened 2.3 ml/kg/min vs metoprolol

   Aficamten beat metoprolol by 2.3 ml/kg/min in peak oxygen uptake.

   2.3: ml/kg/min better peak oxygen uptake than metoprolol.

   • MAPLE-HCM trial

   Fewer surgeriesHappened 15.7% still needed surgery

   With mavacamten, only 15.7% of people referred for septal reduction needed it or stayed eligible at 128 weeks.

   15.7%: went on to septal reduction or stayed eligible at 128 weeks.

   • VALOR-HCM, 128-week results

   Mixed results in nonobstructive HCMHappened 0.47 difference (mavacamten, not significant)

   Mavacamten failed in ODYSSEY-HCM, but aficamten met both goals in ACACIA-HCM.

   0.47: ml/kg/min difference in peak oxygen uptake (not significant).

   • ODYSSEY-HCM trial
   • ACACIA-HCM trial

2. 6 months

   by May 2027

   Filing for nonobstructive HCMLikely

   Cytokinetics plans to seek an added FDA indication for aficamten based on ACACIA-HCM.

   • ACACIA-HCM trial

3. 12 months

   by Nov 2027

   An SGLT2 drug for HCMPossible

   Lexicon's SONATA-HCM (500 people) tests sotagliflozin in obstructive HCM; due January 2027.

   • SONATA-HCM

   A metabolic drug for nonobstructive HCMPossible

   Imbria's ninerafaxstat phase 2 (165 people) is due December 2026.

   • Ninerafaxstat trial

4. 3 years

   by late 2029

   A different way to calm the heart musclePossible

   Edgewise's EDG-7500 aims to relax the heart without lowering ejection fraction; due December 2027.

   • CIRRUS-HCM

   Another myosin inhibitor vs metoprololPossible

   Braveheart's LIONHEART-HCM (210 people) is due October 2028.

   • LIONHEART-HCM

5. 5 years

   by late 2031

   Gene therapy for the causeLong shot

   Tenaya's TN-201 delivers a working MYBPC3 gene, the most commonly mutated gene in HCM.

   • TN-201 trial

   Preventing strokesLong shot

   LA-HCM (532 people) tests rivaroxaban for people with enlarged left atria; due 2032.

   • LA-HCM

The research paths

Borrowed from other conditions

Open to join now

• Obstructive HCM

  BHB-1893 vs metoprolol

  Phase 3 · 4 sites

  Locations and eligibility →

• HCM with enlarged left atrium

  Rivaroxaban

  Phase 3 · 17 sites

  Locations and eligibility →

• Nonobstructive HCM

  Ninerafaxstat

  Phase 2 · 76 sites

  Locations and eligibility →

• MYBPC3-related HCM

  TN-201 gene therapy

  Phase 1/2 · 10 sites

  Locations and eligibility →

See every recruiting HCM trial →

When the trials finish

Completion dates the trials registered. Results usually follow 3 to 12 months later.

What just happened

Standard treatment today

• No symptomsMonitoring; sudden-death risk assessment; family screening.
• Obstructive, symptomaticBeta-blocker or calcium-channel blocker; add mavacamten or aficamten; septal reduction if needed.
• Nonobstructive, symptomaticBeta-blockers, diuretics; aficamten under review.
• High sudden-death riskImplantable defibrillator.

See HCM trials recruiting now →