Cytokinetics’ aficamten meets dual primary endpoints in the pivotal Phase 3 ACACIA-HCM trial for non-obstructive hypertrophic cardiomyopathy, Axsome Therapeutics secures FDA approval of AUVELITY as the first non-antipsychotic treatment for Alzheimer’s disease agitation, Viridian Therapeutics’ elegrobart delivers a second positive Phase 3 result in chronic thyroid eye disease in the REVEAL-2 trial, and Mirum Pharmaceuticals’ volixibat meets its primary endpoint in the Phase 2b VISTAS study for primary sclerosing cholangitis, the most significant clinical trial results 8 May 2026 has to offer from across the pipeline.
This week delivered a strong set of readouts spanning cardiovascular disease, neurology, autoimmune conditions, and rare hepatology, a cross-section that reflects the breadth and maturity of the current global pipeline. The ACACIA-HCM result stands out as the most clinically significant: it is the first Phase 3 trial ever to demonstrate statistically significant improvements in both symptom burden and exercise capacity in patients with non-obstructive HCM, a population that has until now had no approved pharmacological options. Alongside it, a notable FDA approval, a second pivotal Phase 3 win in a competitive rare disease space, and a rare liver disease study that could pave the way for the first-ever targeted therapy in its indication make this one of the more consequential weeks of the year so far. Here, Life Science Daily News brings you the most significant clinical trial results 8 May 2026.
Cytokinetics’ Aficamten Meets Dual Primary Endpoints in Pivotal Phase 3 ACACIA-HCM Trial for Non-Obstructive HCM
Cytokinetics announced on 5 May 2026 positive topline results from ACACIA-HCM (Assessment Comparing Aficamten to Placebo on Cardiac Endpoints In Adults with Non-Obstructive HCM), the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (HCM). The trial met both dual primary endpoints, demonstrating statistically significant improvements from baseline to Week 36 compared to placebo.
The first primary endpoint, the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS), improved by a least squares mean (LSM) difference of 3.0 points in favour of aficamten (95% CI: 0.5–5.5; p=0.021). The second primary endpoint, maximal exercise performance as measured by peak oxygen uptake (pVO₂), improved by an LSM difference of 0.67 mL/kg/min in the aficamten group versus placebo (95% CI: 0.22–1.1; p=0.003). Statistically significant improvements were also observed across key secondary endpoints, including the proportion of participants achieving at least a one-class improvement in New York Heart Association (NYHA) Functional Class, a composite z-score of cardiopulmonary exercise parameters, and NT-proBNP.
ACACIA-HCM was a Phase 3, multicentre, randomised, double-blind, placebo-controlled trial randomising 516 participants (outside Japan) on a 1:1 basis. No new safety signals were identified. Left ventricular ejection fraction (LVEF) below 50% occurred in 10% of participants on aficamten compared with 1% on placebo, and two participants experienced a serious adverse event of heart failure associated with LVEF below 50%. The proportion of participants completing planned dosing was similar between the two arms (88.4% aficamten vs. 90.3% placebo).
Aficamten (MYQORZO®) is a cardiac myosin inhibitor already approved in the United States, Europe, and China for symptomatic obstructive HCM. Non-obstructive HCM, by contrast, currently has no approved pharmacologic therapies targeting the underlying hypercontractility of the disease. Cytokinetics has stated it intends to present the ACACIA-HCM data at an upcoming medical meeting and will discuss the results with the FDA and other global regulatory authorities with a view to seeking a label expansion.
FDA Approves AUVELITY as First Non-Antipsychotic Treatment for Agitation Associated with Alzheimer’s Disease
The FDA approved AUVELITY (dextromethorphan HBr and bupropion HCl) on 30 April 2026 for the treatment of agitation associated with dementia due to Alzheimer’s disease in adults, granting Axsome Therapeutics a significant label expansion for a drug previously approved for major depressive disorder in 2022. AUVELITY is the first FDA-approved treatment for this indication that is not an antipsychotic, representing a meaningful advance in a condition characterised by severe management challenges and significant caregiver burden.
The approval is supported by two randomised controlled studies. ADVANCE-1 was a five-week, double-blind, parallel-group study in which patients were randomised to receive AUVELITY (n=152) or placebo (n=156) twice daily. AUVELITY was statistically significantly superior to placebo on the primary endpoint of change from baseline in the Cohen-Mansfield Agitation Inventory (CMAI) total score at Week 5, with a 14.9-point reduction versus 11.6 points for placebo. On the key secondary endpoint, a statistically significantly greater proportion of AUVELITY-treated patients were rated by clinicians as at least minimally improved on the modified Alzheimer’s Disease Cooperative Study-Clinical Global Impression of Change. ACCORD-2 was a long-term, double-blind, randomised withdrawal study in which known responders to AUVELITY were randomised to either continue treatment or switch to placebo for up to six months. Patients who continued AUVELITY experienced a statistically significantly longer time to relapse of agitation symptoms compared with those switched to placebo (hazard ratio 0.276; p=0.001), with relapse observed in 8.4% of AUVELITY-treated patients versus 28.6% of those on placebo. The most common adverse reactions reported in the agitation trials, occurring in at least 5% of patients and more than twice as frequently as placebo, included dizziness, upset stomach, headache, diarrhoea, drowsiness, dry mouth, sexual dysfunction, and uncontrolled sweating.
AUVELITY is a combination of dextromethorphan, an uncompetitive NMDA receptor antagonist and sigma-1 receptor agonist, and bupropion, a CYP450 2D6 inhibitor included to enhance dextromethorphan exposure. The approval was supported by FDA Breakthrough Therapy designation and Priority Review. Axsome has confirmed that AUVELITY will be available for the Alzheimer’s agitation indication as early as June 2026, maintaining its current list price. The approval did not carry an additional black box warning beyond the existing suicidality warning already on the label from the MDD indication. Axsome enters a market where the only other approved therapy for this specific indication is Rexulti (brexpiprazole), an antipsychotic from Lundbeck and Otsuka that carries a black box warning regarding increased mortality in elderly patients with dementia-related psychosis — a warning AUVELITY does not share.
Viridian Therapeutics’ Elegrobart Delivers Second Positive Phase 3 Result in REVEAL-2 Trial for Chronic Thyroid Eye Disease
Viridian Therapeutics announced on 5 May 2026 positive topline data from the REVEAL-2 Phase 3 clinical trial of elegrobart in patients with chronic thyroid eye disease (TED). REVEAL-2 evaluated two dosing regimens of elegrobart, every four weeks (Q4W) and every eight weeks (Q8W), compared with placebo in patients with the chronic, or inactive, phase of TED.
The trial met its primary endpoint with a highly statistically significant treatment effect. Elegrobart Q4W achieved a proptosis responder rate (PRR) of 50% at Week 24 versus 15% for placebo, and elegrobart Q8W achieved a PRR of 54% versus 15% for placebo, both results being highly statistically significant. Elegrobart Q4W also achieved a statistically significant improvement in clinical activity score (CAS) responder rate as a key secondary endpoint. The safety and tolerability profile was consistent with prior elegrobart data.
Elegrobart is a subcutaneously delivered, half-life-extended monoclonal antibody targeting the insulin-like growth factor-1 receptor (IGF-1R). With positive results now in hand from both the REVEAL-1 trial (active TED) and REVEAL-2 (chronic TED), elegrobart is the only subcutaneous programme to have demonstrated positive Phase 3 data across both phases of the disease, a differentiated profile relative to competing programmes. Viridian’s separate intravenous anti-IGF-1R antibody, veligrotug, has a Biologics License Application (BLA) currently under Priority Review at the FDA, with a PDUFA target action date of 30 June 2026. Viridian has stated that a BLA submission for elegrobart is on track for Q1 2027, where it has the potential to become the first subcutaneous autoinjector treatment for TED.
Mirum Pharmaceuticals’ Volixibat Meets Primary Endpoint in Phase 2b VISTAS Study for Primary Sclerosing Cholangitis
Mirum Pharmaceuticals announced on 4 May 2026 that the primary endpoint was met in the VISTAS Phase 2b study evaluating volixibat, an investigational oral ileal bile acid transporter (IBAT) inhibitor, in patients with primary sclerosing cholangitis (PSC). PSC is a rare, progressive liver disease in which the bile ducts become chronically inflamed and scarred, for which there are currently no approved PSC-specific therapies in the United States.
The VISTAS study enrolled 158 patients with PSC, assigned to either a primary analysis cohort (moderate to severe itch; n=111) or a secondary analysis cohort (mild itch; n=47) based on baseline severity on the Adult Itch Reported Outcome (ItchRO) scale. The primary endpoint was evaluated in the primary analysis cohort. Volixibat 20 mg twice daily achieved a 2.72-point reduction from baseline in cholestatic pruritus versus 1.08 points for placebo, for a placebo-adjusted difference of 1.64 points (p<0.0001). Statistically significant itch reductions appeared within two weeks of treatment initiation and were also observed in patients with mild baseline itch in the secondary cohort. No new safety signals were observed; the safety profile was broadly consistent with the known effects of IBAT inhibition.
Volixibat is an oral, minimally absorbed agent that selectively inhibits the IBAT in the gut, reducing bile acid recycling and thereby lowering systemic and hepatic bile acid levels. Mirum has previously demonstrated positive Phase 2b results for volixibat in primary biliary cholangitis (PBC) via the VANTAGE study (2024). Full VISTAS results are set to be presented as a late-breaking oral presentation at the European Association for the Study of the Liver (EASL) International Liver Congress 2026. Mirum has scheduled a pre-NDA meeting with the FDA for volixibat in PSC in summer 2026 and plans to submit an NDA in the second half of 2026, which if successful would make volixibat the first approved treatment for cholestatic pruritus in patients with PSC.
Looking Ahead
This week’s clinical trial results 8 May 2026 illustrate a pipeline firing across multiple therapeutic fronts simultaneously. Cytokinetics’ ACACIA-HCM success opens a new chapter for aficamten and could substantially expand the drug’s commercial addressable population in HCM, a disease with growing diagnostic rates and currently limited treatment depth in the non-obstructive form. The FDA approval of AUVELITY for Alzheimer’s agitation offers clinicians and caregivers a first-in-class non-antipsychotic option in a population where safety concerns around existing agents have historically constrained prescribing. Viridian’s second Phase 3 success with elegrobart in TED reinforces the programme’s differentiated clinical profile across both active and chronic disease, setting the stage for a competitive BLA filing in 2027. And Mirum’s VISTAS results in PSC represent a meaningful step forward for a rare liver condition that has long lacked targeted pharmacologic options. Life Science Daily News will continue to bring you accurate, timely coverage of the clinical trial results 8 May 2026 that matter most across the global life sciences pipeline.
This clinical trials roundup is produced by the Life Science Daily News editorial team. All stories are selected and written independently.
