An FDA advisory committee votes against Capricor Therapeutics’ deramiocel in Duchenne muscular dystrophy cardiomyopathy, Pfizer’s ritlecitinib meets its co-primary endpoints in two Phase 3 nonsegmental vitiligo trials, Karyopharm Therapeutics’ selinexor misses its primary endpoint in a Phase 3 endometrial cancer maintenance trial, Sanofi discontinues amlitelimab in moderate to severe atopic dermatitis, and Outlook Therapeutics’ Lytenava receives FDA approval as the first ophthalmic bevacizumab for wet age-related macular degeneration: the most significant clinical trial results 31 July 2026 has to offer from across the pipeline.
This week delivered a broad spread of readouts and regulatory decisions spanning rare neuromuscular disease, autoimmune dermatology, gynaecological oncology and retinal medicine. The defining story is an unusually public divergence between a sponsor and regulators over which version of a pivotal trial’s statistical analysis plan should govern interpretation, a dispute that played out in an advisory committee room and in one of medicine’s most selective journals on the same day. The remaining results run the full range of late-stage outcomes: a large dermatology programme meeting both co-primary measures, a maintenance strategy falling short of significance, a closely watched mechanism abandoned despite supportive long-term data, and an approval that gives two decades of off-label practice a regulated alternative. Here, Life Science Daily News brings you the most significant readouts and regulatory decisions of the week.
FDA Advisory Committee Votes Against Capricor’s Deramiocel in Duchenne Muscular Dystrophy Cardiomyopathy
Capricor Therapeutics announced on 30 July 2026 that the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee had voted that the available evidence did not support the effectiveness of deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). The committee voted 3 in favour, 9 against, with no abstentions, on whether the pivotal Phase 3 HOPE-3 study provides substantial evidence of effectiveness. The vote is non-binding. Capricor noted that the voting question addressed a narrower indication than the company had proposed and did not include a vote on deramiocel’s overall benefit-risk profile, and that in a separate discussion on upper limb function the committee’s feedback was directionally supportive. The application is a resubmission: the FDA issued a Complete Response Letter in July 2025 on the original filing, which rested on the Phase 2 HOPE-2 study and its open-label extension. There is no approved treatment for DMD-associated cardiomyopathy, the leading cause of death in the condition.
The dispute centres on which version of the trial’s statistical analysis plan should govern interpretation. In its briefing document released on 27 July, the FDA states that HOPE-3, which randomised 106 males aged 10 to 22, did not meet its pre-specified primary or key secondary endpoints. Under SAP version 1.1, on which the agency says the study was designed and powered, the difference in change from baseline to month 12 in PUL 2.0 total score was 0.66 points (95 per cent CI: -0.45, 1.77; p=0.24), and the difference in left ventricular ejection fraction was -0.04 percentage points (p=0.97). According to the briefing document, Capricor’s analyses instead rest on SAP version 3.0, dated 24 November 2025, one day before database lock and around five months after the randomised phase completed, which redefined the primary endpoint as percent change and produced a nominally significant result (p=0.029). That is the basis for the 54 per cent slowing of upper limb decline the company reports and which was published in The Lancet on 29 July. The FDA also states that SAP 3.0 was prepared by the applicant rather than the independent blinded biostatistics team named in the study’s approved blinding plan.
Capricor disputes that account. In a statement issued on 27 July, the company said its results are governed by SAP version 3.0, which it describes as the final analysis plan, finalised before unblinding, and characterised SAP version 1.1 as an unsigned, incomplete internal draft made obsolete by the addition of cohort B. Its own briefing document records SAP 3.0 as having been submitted to the FDA in response to agency feedback on an earlier version, and the FDA’s document carries a standard disclaimer that reviewer conclusions are not the agency’s final position. The committee was not persuaded. Janet Wittes of Wittes LLC, a clinical trials statistician on the panel, described the results as “very fragile” and said she had not seen enough evidence of benefit to vote yes, while FDA statistician Tingting Zhou told the meeting that the analytical models and missing-data handling were broadly similar across every plan version preceding the interim futility analysis. The panel’s patient representative dissented, criticising the agency for setting aside data on procedural grounds. The PDUFA target action date is 22 August 2026, one of several FDA drug approval decisions falling in this window.
Pfizer’s Ritlecitinib Meets Co-Primary Endpoints in Two Phase 3 Nonsegmental Vitiligo Trials
Pfizer announced on 30 July 2026 positive topline results from TRANQUILLO and TRANQUILLO 2, two pivotal Phase 3 trials evaluating once-daily Litfulo (ritlecitinib) in patients with active and stable nonsegmental vitiligo (NSV) across a broad range of disease severity. Across both studies, significantly more patients treated with ritlecitinib achieved at least a 75 per cent improvement in the Facial Vitiligo Area Scoring Index (F-VASI75) and at least a 50 per cent improvement in the Total Vitiligo Area Scoring Index (T-VASI50) from baseline at week 52 compared with placebo. In the United States both measures were co-primary endpoints; outside the United States, F-VASI75 at week 52 was the primary endpoint and T-VASI50 a key secondary endpoint. Pfizer reported that both the 50 mg and 100 mg doses delivered significant, clinically meaningful improvements over placebo.
TRANQUILLO evaluated 50 mg once daily in 607 adults and adolescents, while TRANQUILLO 2 evaluated 50 mg or 100 mg in 1,567 adults, with 50 mg comparisons in the latter exploratory and descriptive. Together the trials enrolled 2,174 patients across 271 sites, which Pfizer describes as the largest Phase 3 programme to date for an oral systemic therapy in NSV. Ritlecitinib, an oral inhibitor of Janus kinase 3 and the TEC family kinases, is already approved for severe alopecia areata in patients aged 12 and over. Pfizer reported a safety profile consistent with that established in alopecia areata, with no new signals; as a Janus kinase inhibitor, ritlecitinib carries a boxed warning covering serious infections, mortality, malignancy, major adverse cardiovascular events and thrombosis. The field changed shape in the same week: on 29 July the European Commission approved AbbVie’s Rinvoq (upadacitinib) for non-segmental vitiligo in adults and adolescents aged 12 and over, the first systemic medication approved for the condition in the European Union, and upadacitinib is also under FDA review. Incyte’s povorcitinib met its primary endpoint in two Phase 3 studies, with filings planned for the first half of 2027. Pfizer has so far said only that it intends to file, and has not released response rates.
Karyopharm’s Selinexor Misses Primary Endpoint in Phase 3 Endometrial Cancer Maintenance Trial
Karyopharm Therapeutics announced on 30 July 2026 topline results from the Phase 3 XPORT-EC-042 trial evaluating selinexor as a maintenance-only therapy compared with placebo in adult patients with TP53 wild-type advanced or recurrent endometrial cancer. The trial did not meet its primary endpoint of progression-free survival. In the modified intent-to-treat population (n=236), median progression-free survival was 12.75 months for selinexor compared with 7.43 months for placebo, but the difference did not reach statistical significance (hazard ratio 0.76; 95 per cent CI: 0.51, 1.12; one-sided p-value 0.0791). The safety and tolerability profile was consistent with selinexor’s established profile, with no new safety signals observed.
XPORT-EC-042 (ENGOT-EN20; GOG-3083; NCT05611931) was a global, randomised, double-blind, placebo-controlled study in which 257 patients were randomised 1:1 to 60 mg of oral selinexor once weekly or placebo until disease progression, following chemotherapy or chemotherapy plus a checkpoint inhibitor. Progression-free survival was tested sequentially across two populations, the first being the modified intent-to-treat population of 236 patients. Selinexor is an oral inhibitor of exportin 1 (XPO1), marketed as Xpovio and approved in the United States in multiple myeloma. Richard Paulson, President and Chief Executive Officer of Karyopharm, said the results fell short of expectations but do not diminish the company’s confidence in XPO1 inhibition. Karyopharm will reduce planned investment in endometrial cancer and prioritise its myelofibrosis and multiple myeloma programmes, and will present the full dataset at a future medical meeting.
Sanofi Discontinues Amlitelimab in Moderate to Severe Atopic Dermatitis
This story broke on 24 July and was not included in last week’s roundup. Given its clinical significance, we include it in the clinical trial results 31 July 2026 below..
Sanofi announced on 24 July 2026 the discontinuation of clinical development of amlitelimab, an OX40-ligand monoclonal antibody, in moderate to severe atopic dermatitis (AD), confirming it will not file for global regulatory review. The decision followed a strategic assessment of the pipeline, Sanofi concluding that the totality of efficacy and safety evidence does not support further development. The ESTUARY Phase 3 long-term extension study (NCT06407934) showed maintenance of clinical response without relapse in patients aged 12 and older, but Sanofi judged that amlitelimab would not represent a meaningful improvement to the standard of care. It had met its primary endpoint in two of three Phase 3 AD studies.
Amlitelimab (SAR445229, KY1005) is a fully human, non-T-cell-depleting antibody that blocks OX40L signalling early in an overactive immune response. The discontinuation leaves no OX40 or OX40-ligand directed candidate in late-stage AD development: Kyowa Kirin and Amgen discontinued rocatinlimab in March 2026 following emerging safety concerns. Sanofi will wind down ongoing AD studies with transition of care for enrolled patients and present additional results at a forthcoming medical meeting. A Phase 2 study in coeliac disease reads out in the second half of 2026, and full-year 2026 guidance is unchanged.
FDA Approves Outlook Therapeutics’ Lytenava as the First Ophthalmic Bevacizumab for Wet AMD
This story broke on 24 July and was not included in last week’s roundup. Given its clinical significance, we are covering it here.
Outlook Therapeutics announced on 24 July 2026 that the FDA has approved Lytenava (bevacizumab-vikg) for neovascular age-related macular degeneration (nAMD), or wet AMD, the first FDA-approved ophthalmic formulation of bevacizumab in the United States. Approval came ahead of a Prescription Drug User Fee Act date of 29 July following a Class 1 review of the resubmitted Biologics License Application. Bevacizumab has been used off label intravitreally for more than two decades in repackaged formulations of the intravenous oncology product, and the company anticipates 12 years of Reference Product Exclusivity under the Biologics Price Competition and Innovation Act. The approval rests on the NORSE programme, in which NORSE TWO (NCT03834753) compared bevacizumab-vikg 1.25 mg with ranibizumab 0.5 mg; the approved dose is 1.25 mg intravitreally once monthly.
Bevacizumab-vikg is a recombinant humanised IgG1 antibody that binds all isoforms of VEGF-A, suppressing the neovascularisation and vascular permeability that drive vision loss in wet AMD. The most common adverse reactions were conjunctival haemorrhage (4 per cent), eye pain (2 per cent) and vitreous floaters (2 per cent), and it is contraindicated in ocular or periocular infection and active intraocular inflammation. Outlook puts the United States retina market at approximately $8.5 billion annually and expects to make Lytenava available before year-end. The approval does not settle a long-running cost debate: repackaged intravenous bevacizumab has been available to retina clinics at a fraction of the price of branded anti-VEGF agents, and pricing has not been disclosed. The product already holds marketing authorisations in the European Union and the United Kingdom.
Looking Ahead
This week’s clinical trial results 31 July 2026 span rare neuromuscular disease, autoimmune dermatology, gynaecological oncology and retinal medicine, and together they underline how much of late-stage development now turns on interpretation as well as outcome. The deramiocel vote leaves little room for optimism ahead of the 22 August decision: the FDA usually follows its committees, and the panel’s objection was not to the size of the effect but to whether the analysis producing it can be relied on at all. If a sponsor can revise an analysis plan after the randomised phase closes and have the result treated as pre-specified, pre-specification stops meaning very much. In vitiligo the question is no longer whether an oral systemic reaches patients but which one and in what order, with upadacitinib holding the European approval and ritlecitinib the larger dataset but no submission. Selinexor’s five-month median gap is a reminder that a one-sided p-value of 0.08 in 236 patients is a hypothesis, not a finding. Amlitelimab’s exit removes the last OX40-directed candidate from the atopic dermatitis pipeline. And Lytenava’s approval brings an FDA-regulated product to twenty years of off-label practice, though whether it displaces cheaper repackaged bevacizumab will depend entirely on price, which Outlook has not disclosed. Life Science Daily News will continue to bring you accurate, timely coverage of the trial readouts and regulatory decisions that matter most across the global life sciences pipeline.
