Definium Therapeutics’ DT120 meets its primary endpoint in the Phase 3 Panorama study in generalised anxiety disorder, AstraZeneca’s camizestrant misses its primary endpoint in the Phase III SERENA-4 study in first-line breast cancer, Vera Therapeutics’ atacicept cuts kidney disease progression risk in the Phase 3 ORIGIN 3 final analysis in IgA nephropathy, Connect Biopharma’s rademikibart improves lung function but misses its primary endpoint in a Phase 2 asthma exacerbation study, and BioVie’s bezisterim shows no significant effect overall but signals in high burden subgroups in the Phase 2 ADDRESS-LC study in Long COVID: the most significant clinical trial results 18 September 2026 has to offer from across the pipeline.
This week’s results span psychiatry, oncology, nephrology, respiratory medicine and post-viral illness. The standout is a second positive Phase 3 result for a single-dose lysergide (LSD) tablet in generalised anxiety disorder. No classic psychedelic has yet been approved by the FDA, and lysergide remains a Schedule I controlled substance. Against that, a major pharmaceutical company’s first-line breast cancer trial fell short of its primary goal. A kidney disease study delivered two-year data behind an accelerated approval already in hand. Two mid-stage studies returned mixed verdicts. One missed its primary endpoint, and the other found effects only in subgroups.
DT120 Meets Its Primary Endpoint in the Phase 3 Panorama Study in Generalised Anxiety Disorder
Definium Therapeutics announced on 14 September 2026 that DT120 met the primary endpoint of its Phase 3 Panorama study. Panorama is the company’s second Phase 3 study in generalised anxiety disorder (GAD). DT120 is Definium’s orally disintegrating tablet formulation of lysergide. At week 12, Hamilton Anxiety Rating Scale (HAM-A) scores fell by 9.8 points on a single 100 microgram dose. Scores fell by 4.7 points on placebo. The placebo-adjusted difference of 5.1 points was significant (p<0.0001), with a standardised effect size of 0.64. All three key secondary endpoints were also met at p<0.0001. These covered Clinical Global Impression of Severity (CGI-S) scores at week 12 and day 2, and HAM-A at week 1. Response, meaning at least a 50 per cent HAM-A improvement, reached 32 per cent against 14 per cent on placebo. Remission, a HAM-A score of 7 or below, reached 15 per cent against 4 per cent. Both were other secondary endpoints, significant at p<0.05.
Panorama (MM120-301, NCT06809595) enrolled 245 adults across roughly 32 study centres. Participants were randomised 2:1:2 to 100 micrograms (n=96), 50 micrograms (n=52) or placebo (n=97). The 50 microgram arm was included to help mitigate functional unblinding and was not powered for comparison. Most treatment-emergent adverse events were mild to moderate, transient and concentrated on dosing day. Overall adverse event rates were 94.8 per cent on 100 micrograms and 62.9 per cent on placebo. Illusion, nausea and headache were the most common, and the company reported no new safety signals. Definium describes DT120 as a partial agonist at the serotonin 5-HT2A receptor. Definium also describes GAD as one of the most common psychiatric disorders, with no new drug approved since 2007. The company has scheduled a pre-NDA meeting with the FDA for the fourth quarter of 2026. It anticipates a filing in the first half of 2027, following the positive Voyage results in August. Any approved product would also need DEA rescheduling before it could be marketed in the US.
Camizestrant Misses Its Primary Endpoint in the Phase III SERENA-4 Study in First-Line Breast Cancer
AstraZeneca announced on 11 September 2026 that SERENA-4 did not meet its primary endpoint. The Phase III trial tested camizestrant (Etcamah) plus palbociclib in first-line advanced breast cancer. Patients had oestrogen receptor positive, HER2 negative disease and no prior systemic treatment for advanced disease. The comparator was anastrozole plus palbociclib. Investigator-assessed progression free survival (PFS) showed a numerical improvement for camizestrant, but it was not statistically significant. AstraZeneca disclosed no hazard ratio, median PFS or p-value, and said data will be shared in due course. The safety profile was consistent with each medicine’s known profile, with no new concerns identified.
SERENA-4 (NCT04711252) enrolled 1,371 patients. Camizestrant is an oral selective oestrogen receptor degrader that blocks and degrades the oestrogen receptor. It is already approved in the US, EU and Japan, based on the SERENA-6 trial. That indication covers patients whose tumours develop an ESR1 mutation during first-line endocrine-based therapy. The US approval on 4 September 2026 was an accelerated approval. Susan Galbraith, Executive Vice President, Oncology Haematology R&D, said AstraZeneca was disappointed by the outcome. She said the result sharpens its focus on patients who can benefit today based on SERENA-6. She also stressed the importance of ESR1 testing for patients on first-line therapy. The CAMBRIA-1 and CAMBRIA-2 Phase III trials, covering around 10,000 patients, continue in the adjuvant setting.
Atacicept Cuts Kidney Disease Progression Risk by 76 Per Cent in the ORIGIN 3 Final Analysis
Vera Therapeutics announced on 15 September 2026 that atacicept-vymj (Trutakna) met all prespecified ORIGIN 3 final analysis endpoints. The Phase 3 trial enrolled adults with primary IgA nephropathy at risk of progression. Estimated glomerular filtration rate (eGFR) fell by 0.1 mL/min/1.73m² over 52 weeks on atacicept. It fell by 5.7 mL/min/1.73m² on placebo. That is a treatment effect of 5.6 mL/min/1.73m² (p<0.0001). The annualised eGFR slope through 104 weeks showed a treatment effect of 5.0 mL/min/1.73m² per year (p<0.0001). Composite kidney disease progression events occurred in 11 patients on atacicept and 38 on placebo. That corresponds to a 76 per cent risk reduction (hazard ratio 0.24, p<0.0001).
ORIGIN 3 (NCT04716231) reported topline results from a final analysis set of 428 patients. Atacicept is a fusion protein that blocks BAFF and APRIL, two cytokines that activate B cells in the disease. Vera reported safety generally comparable to placebo, with similar rates of overall adverse events and infections. The drug already holds US accelerated approval to reduce proteinuria, based on an earlier interim analysis. Vera plans to submit a supplemental Biologics License Application for full approval in the fourth quarter of 2026. Founder and Chief Executive Marshall Fordyce said the company looks forward to potential full approval in 2027.
Rademikibart Improves Lung Function but Misses Its Primary Endpoint in Phase 2 Asthma Study
Connect Biopharma announced on 15 September 2026 preliminary topline results from its Phase 2 Seabreeze STAT Asthma study. The trial tested rademikibart as an add-on treatment for acute asthma exacerbations. The primary endpoint, treatment failure over 28 days, fell by about 66 per cent against placebo. That reduction was not statistically significant (p=0.153). The company attributed the miss to a lower than projected overall treatment failure rate. The key secondary endpoint, post-bronchodilator forced expiratory volume in one second (FEV1) at day 7, was met. FEV1 rose by 250 mL on rademikibart against 120 mL on placebo, a 130 mL difference (p=0.023).
Seabreeze STAT Asthma (NCT06940141) randomised 160 adults and adolescents with asthma and type 2 inflammation. Patients received a single 600 mg subcutaneous dose of rademikibart (n=79) or placebo (n=81), alongside standard of care. Rademikibart is a monoclonal antibody against the interleukin-4 receptor alpha subunit, the same target as dupilumab. Here it was tested as a single dose around an acute exacerbation, not as maintenance therapy. Emergency department or unscheduled visits for worsening asthma fell by 50 per cent, though no p-value was reported. One serious adverse event occurred on rademikibart, against three on placebo. The company proposes FEV1 at week 1 as the primary endpoint for Phase 3. It expects COPD data later in September 2026, after which it plans to seek FDA alignment on Phase 3.
Bezisterim Shows No Significant Effect Overall but Signals in Long COVID Subgroups
BioVie announced on 15 September 2026 topline results from its Phase 2 ADDRESS-LC trial of bezisterim in Long COVID. The exploratory trial assessed neurological symptoms across 22 clinical endpoints, with none pre-specified as primary. No endpoint reached statistical significance in the overall intent to treat population. The company said 21 of the 22 endpoints showed numerical trends favouring bezisterim. In pre-specified subgroup analyses, patients with high baseline symptom burden showed statistically significant improvements. Patients with the most severe fatigue improved on five endpoints. Those with high post-exertional malaise improved on malaise, fatigue and objective cognitive measures. Patients with substantial objective cognitive impairment improved on four cognitive endpoints.
ADDRESS-LC (NCT06847191) randomised 203 patients to twice daily oral bezisterim or placebo. Bezisterim is an investigational oral drug that BioVie says reduces inflammation and improves insulin sensitivity. BioVie itself describes the subgroup findings as exploratory and hypothesis-generating. It says they require confirmation in an adequately powered prospective study. Safety was similar to placebo, with no serious adverse events on bezisterim and one on placebo. BioVie said the subgroup approach will guide the design of a future Phase 3 trial. Until then, any benefit in a broader Long COVID population remains unproven.
Looking Ahead
This week’s clinical trial results 18 September 2026 show how much depends on the endpoint a trial is judged by. Panorama gives Definium a second positive Phase 3 result in GAD. A filing would still need FDA review and DEA rescheduling before any launch. AstraZeneca has not indicated any change to camizestrant’s SERENA-6 based approvals. SERENA-4 does, however, narrow the drug’s path in upfront first-line treatment. Vera’s two-year data now underpin its planned application for full approval. Rademikibart hit a key secondary endpoint while missing its primary measure. Bezisterim showed effects only in subgroups, which its sponsor calls exploratory. None of this week’s five studies is the final word on its drug. Life Science Daily News will continue to bring you accurate, timely coverage of the trial data that matter most across the global life sciences pipeline. Catch up on last week’s clinical trials roundup.
