Clinical Trial Readouts
The late-stage trials behind the headlines: status, expected readouts, and the latest results, updated from the trial registry and company filings.
Updated 2026-10-20
| Drug | Company | Indication | Phase | Status | Readout expected | Latest result |
|---|---|---|---|---|---|---|
| rezpegaldesleukin (NKTR-358) | Nektar Therapeutics | Severe alopecia areata; atopic dermatitis (separate REZOLVE-AD study) | Phase 2b (REZOLVE-AA); Phase 3 ZENITH-AA planned to start in early 2027 | Completed | ZENITH-AA primary endpoint is the share of patients at a SALT score of 20 or below at week 52; the study is to start in early 2027. REZOLVE-AD off-treatment data are expected in the first quarter of 2027. | EADV 2026 (Oct 1, 2026): among REZOLVE-AA patients at a SALT score of 20 or below after 52 weeks of treatment, 75 percent (6 of 8) held the threshold four months off treatment and 63 percent (5 of 8) at six months, by non-responder imputation; SALT 10 responses deepened from 7 percent at week 52 to 19 percent six months off treatment. Nektar shares fell 22.4 percent to $46.99 on the release. |
| ontunisertib (AGMB-129) | Agomab Therapeutics | Fibrostenosing Crohn's disease | Phase 2a (STENOVA) complete; Phase 2b NOV-ERA planned | Completed | NOV-ERA Phase 2b, about 320 patients randomized across three doses and placebo; the primary endpoint is the share of patients whose index ileal stricture becomes endoscopically passable at week 24. Agomab has completed regulatory filings and plans to start the study in the coming months. | STENOVA open-label extension (Oct 1, 2026): one endoscopic balloon dilation and no surgeries across 49 patients treated with ontunisertib 200 mg twice daily for up to 60 weeks, an annualized event rate of 3 percent against the 23 to 35 percent the company cites for comparable patients on advanced therapies (cross-trial, disclaimed by the company). MRE suggested stricture stabilization, with placebo-arm patients who switched catching up; the optional week 48 endoscopy was too limited to interpret. |
| rademikibart (CBP-201) | Connect Biopharma | Acute COPD exacerbations; asthma (the same 28-day treatment-failure endpoint in both) | Phase 2 (Seabreeze STAT COPD, NCT06940154); a separate Phase 2 in asthma reported Sep 15, 2026 | Completed | Phase 3 design, endpoint and sample size to be settled with the FDA; the Sep 15 asthma release said the two readouts should help determine a potential Phase 3 endpoint and sample size requirements. | Seabreeze STAT Phase 2 (Sep 30, 2026): one 600 mg subcutaneous dose given during an acute exacerbation cut 28-day treatment failure to 2 of 78 (2.6 percent) from 11 of 81 (13.6 percent) on placebo, an 81 percent reduction (p=0.0122). Hospital or emergency department returns were 0 of 78 against 7 of 81 (p=0.0137) and new moderate-to-severe exacerbations 1 against 7 (p=0.0301); FEV1 improved about 70 mL over placebo at week 4 (p=0.1607, not significant). The asthma Phase 2 (Sep 15, 2026) missed the same 28-day failure endpoint with a 66 percent reduction (p=0.153) but improved FEV1 by 130 mL at day 7 (p=0.023). |
| z-basivarsen (DYNE-101) | Dyne Therapeutics | Myotonic dystrophy type 1 | Phase 1/2 (ACHIEVE, with a registrational expansion cohort); Phase 3 HARMONIA as confirmatory | Active, not recruiting | Registrational expansion cohort topline on video hand opening time at six months expected in the first quarter of 2027, carrying the accelerated approval filing; HARMONIA reports later on the five-times sit-to-stand test for traditional approval. | ACHIEVE one-year data (Sep 29, 2026): the pooled dose group of 25 to 26 participants improved video hand opening time by 3.2 seconds at six months against a 0.4-second worsening on placebo, and at 12 months improved 1.2 seconds on the five-times sit-to-stand test, 0.3 seconds on the ten-meter walk, 4.8 percent of predicted on quantitative muscle testing and grip strength, and 25.2 percent on the Myotonic Dystrophy Health Index, diverging from a propensity-matched natural history cohort of 41 to 46 participants from END-DM1; the patient-reported comparison used an unmatched cohort of 410. The 3.2-second vHOT improvement carried a standard deviation of 3.5 seconds, only 8 of 26 pooled participants received the registrational 6.8 mg/kg dose for the full 12 months, and no related serious treatment-emergent adverse events were reported to the April 20, 2026 cutoff. The 71-participant expansion cohort has a mean baseline vHOT of 8.3 seconds. |
| NXC-201 | Immix Biopharma | Relapsed or refractory AL amyloidosis | Phase 1/2 (NEXICART-2, registrational design) | Active, not recruiting | Final readout and BLA submission planned for mid-2027; Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations in hand, plus Orphan Drug from the FDA and the European Medicines Agency. | NEXICART-2 interim (Sep 29, 2026): 40 of 45 patients, 89 percent, in complete response by independent review, with all 25 of the most recently treated patients in complete response or MRD-negative; no relapses among complete responders or MRD-negative patients, and no neurotoxicity or enterocolitis to date. The May 2026 interim had reported 95 percent across 20 assessed patients, and the company projects the four pending MRD-negative patients will convert, lifting the rate to 98 percent. |
| VYD2311 | Invivyd | COVID-19 prevention | Phase 3 (LIBERTY and DECLARATION) | Active, not recruiting | DECLARATION safety and immunogenicity unblinding in October 2026, with clinical events kept blinded for a post-approval confirmatory cohort; a Biologics License Application under accelerated approval, resting on safety and antibody titers, is planned for 2027. | LIBERTY topline (Sep 29, 2026): among 210 healthy adults, treatment-emergent adverse events, injection site reactions or hypersensitivity reactions over six days were 56.5 percent on VYD2311 against 91.4 percent on Comirnaty, with no reaction above Grade 2 and no anaphylaxis; solicited systemic side effects were 44.1 percent against 68.1 percent (p=0.008), and 60.9 percent against 91.4 percent at 56 days (p below 0.0001). The study did not follow participants for COVID-19 infection. Adding VYD2311 to the vaccine raised neutralizing titers roughly 2.5-fold over the vaccine alone, with no blunting of the vaccine response. |
| Zenkuda (tarcocimab tedromer, KSI-301) | Kodiak Sciences | Wet age-related macular degeneration, treatment-naive; also diabetic retinopathy and macular edema following retinal vein occlusion | Phase 3 (DAYBREAK and DAYLIGHT in wAMD; GLOW1 and GLOW2 in DR; BEACON in RVO) | Active, not recruiting | Three-indication BLA planned for the fourth quarter of 2026, covering wAMD, DR and RVO; a separate asset, KSI-101, reports Phase 3 MESI topline data in December 2026 | DAYBREAK met its one-year primary endpoint of non-inferiority in best-corrected visual acuity against aflibercept, with a p-value of 0.0007. Kodiak reported that 54 percent of Zenkuda patients reached a 24-week dosing interval by year one under an AI-guided treat-to-dryness protocol after four monthly loading doses, with no intraocular inflammation and a cataract adverse event rate of 0.5 percent against 0.9 percent for aflibercept. CTOL Digital reported mean letter gains of 7.2 for Zenkuda against 7.6 for aflibercept. The comparator arm received aflibercept 2 mg dosed per label, an eight-week regimen; no head-to-head against Eylea HD or Vabysmo has been run. The same molecule missed the DAZZLE wet AMD endpoint in February 2022 with intraocular inflammation of 3.2 percent against zero, and missed the GLEAM and GLIMMER diabetic macular edema endpoints in July 2023 with cataracts of 19 percent against 9 percent, prompting a wind-down that was reversed that November. |
| tabirafusp-ted (KSI-501) | Kodiak Sciences | Wet age-related macular degeneration; diabetic macular edema | Phase 3 (DAYBREAK in wAMD; ALTO in DME) | Active, not recruiting | ALTO Phase 3 in diabetic macular edema is enrolling about 910 patients across three arms and tests superiority against aflibercept, a higher bar than the non-inferiority design used in DAYBREAK | DAYBREAK met the vision primary endpoint with a p-value of 0.0036 and the anatomical key secondary endpoint at less than 0.0001. Tabirafusp-ted was dosed on a fixed eight-week schedule with additional as-needed dosing up to monthly after four monthly loading doses, and reported an intraocular inflammation rate of 0.4 percent and no cataracts against 0.9 percent for aflibercept. The company is planning post-hoc analyses to identify which treatment-naive wet AMD subgroups benefit from IL-6 inhibition. |
| tavapadon (JUVMO) | AbbVie, from the Cerevel Therapeutics acquisition | Parkinson's disease, early stage and as add-on to levodopa | Phase 3 (TEMPO-1, TEMPO-2, TEMPO-3; TEMPO-4 open-label extension) | Completed | Approval granted Sep 28, 2026; US launch expected October 2026; TEMPO-4 open-label extension data reported through 85 weeks | FDA approved tavapadon on Sep 28, 2026 as the first selective D1/D5 agonist for Parkinson's disease. TEMPO-1 week 26 combined MDS-UPDRS Parts II and III: 1.8 points worse on placebo, 9.7 better at 5 mg, 10.2 better at 15 mg. TEMPO-2 flexible dose: 10.3 points better vs 1.2 on placebo. TEMPO-3 add-on: 1.7 more hours of on-time vs 0.6, and 1.9 fewer hours of off-time vs 0.9. TEMPO-2 discontinuations were 57 of 151 (38 percent) on the drug vs 23 of 153 (15 percent) on placebo, with adverse-event discontinuations of 36 (24 percent) vs 6 (4 percent). An independent meta-analysis rated the motor benefit at moderate certainty with no outcome reaching high certainty, and found no trial using an active comparator or running past 27 weeks. |
| sefaxersen (IONIS-FB-LRx) | Roche / Genentech, Ionis Pharmaceuticals | Primary IgA nephropathy, high risk of progression | Phase 3 (IMAgINATION) | Active, not recruiting | Interim proteinuria data presented at a medical congress; confirmatory eGFR slope at week 105, estimated study completion 2029-03 | IMAgINATION interim: met the primary endpoint, a statistically significant and clinically meaningful reduction in 24-hour urine protein-to-creatinine ratio vs placebo at week 37 in 459 patients, with no new safety signals. The magnitude of the reduction was not disclosed. Trial stays blinded to week 105 for the eGFR slope endpoint (Sep 22, 2026). |
| barzolvolimab | Celldex Therapeutics | Chronic spontaneous urticaria, including omalizumab-refractory | Phase 3 | Active, not recruiting | BLA planned 2027; 52-week data at a medical meeting | EMBARQ-CSU1/CSU2: met the primary and all key secondary endpoints in 1,939 patients. UAS7 improved 19.7 to 20.5 points vs 10.7 to 11.4 on placebo; complete response 42 to 46 percent vs 9.3 to 12.6 percent at week 12. Two Grade 4 anaphylaxis events in a dosed group (Sep 22, 2026). |
| VK2735 | Viking Therapeutics | Obesity / weight management, maintenance dosing | Phase 3 (VANQUISH); maintenance study Phase 2 | Active, not recruiting | Phase 3 primary completion 2027-07; oral maintenance part 2 pending | VK2735-102 maintenance: every-other-week arms retained 90 percent and monthly arms 85 percent of the weight lost over 21 weeks of weekly dosing, vs 61 percent on placebo over 12 weeks; GI events at or below placebo. Exploratory weight endpoints (Sep 22, 2026). |
| vosoritide | BioMarin | Hypochondroplasia | Phase 3 | Active, not recruiting | sNDA with the FDA; EMA submission on track | CANOPY-HCH-3 detailed results: +2.33 cm/year AGV difference (NEJM Evidence, Sep 9, 2026). |
| deucrictibant XR | Pharvaris | Hereditary angioedema prophylaxis (all three types) | Phase 3 | Active, not recruiting | NDA submission H1 2027 | CHAPTER-3 met: 83% reduction in monthly attacks versus placebo (Sep 8, 2026); 87% in type 1/2. |
| tarlatamab + durvalumab | Amgen / AstraZeneca | Extensive-stage small cell lung cancer, first-line maintenance | Phase 3 | Active, not recruiting | Detailed data at a medical congress, date undisclosed | DeLLphi-305 interim: OS, PFS and ORR all improved versus Imfinzi alone (Sep 8, 2026); no numbers disclosed. |
| del-desiran | Novartis (acquired with Avidity) | Myotonic dystrophy type 1 | Phase 3 | Completed | Full dataset evaluation; path discussed with health authorities | HARBOR missed its primary endpoint (vHOT, Sep 8, 2026); secondary signals reported but no numbers disclosed. |
| olpasiran | Amgen | Elevated Lp(a) / cardiovascular events | Phase 3 | Active, not recruiting | Primary completion 2028-03-31 (OCEAN(a) Outcomes) | No outcomes data yet. |
| lepodisiran | Eli Lilly | Elevated Lp(a) / cardiovascular events | Phase 3 | Active, not recruiting | Primary completion 2029-03 | No outcomes data yet. |
| muvalaplin | Eli Lilly (oral) | Elevated Lp(a) / cardiovascular events | Phase 3 | Recruiting | Primary completion 2031-03 | No outcomes data yet. |
| aficamten | Cytokinetics | Non-obstructive hypertrophic cardiomyopathy | Phase 3 | Active, not recruiting | Filing plans to follow | ACACIA-HCM met both primary endpoints (topline Aug 28, 2026). |
| lanifibranor | Inventiva | MASH | Phase 3 | Active, not recruiting | Topline expected Q4 2026 | NATiV3 last patient completed the final 72-week visit (Sep 3, 2026). |
| remlifanserin (ACP-204) | Acadia Pharmaceuticals | Alzheimer's disease psychosis | Phase 2/3 | Recruiting | Phase 3 primary completion 2028-01; detailed Phase 2 results at CTAD in November 2026 | RADIANT Phase 2 topline (Sep 24, 2026): the 60 mg dose reduced the primary symptom score by 12.6 points vs 10.4 on placebo at week 6, an effect size of 0.26 and a p-value of 0.0603, a miss; the key secondary clinician-rated severity scale was nominally significant at 0.37 (p=0.0077). The 30 mg arm is discontinued. Applying the refined enrollment criteria back to the Phase 2 dataset raised the primary effect size from 0.26 to 0.33. Adverse events, serious adverse events and discontinuations were similar to placebo, with no QT signal. |
What changed
- 2026-09-29: Zenkuda (Kodiak Sciences) and tabirafusp-ted (Kodiak Sciences) added: Phase 3 DAYBREAK met both primary endpoints in wet AMD, with Zenkuda holding 54 percent of patients on 24-week dosing at a 0 percent intraocular inflammation rate and a 0.5 percent cataract rate against 0.9 percent for aflibercept.
- 2026-09-29: Zenkuda (tarcocimab tedromer, KSI-301) (Kodiak Sciences): status Met primary endpoint -> Active, not recruiting
- 2026-09-29: tabirafusp-ted (KSI-501) (Kodiak Sciences): status Met primary endpoint -> Active, not recruiting
- 2026-09-30: z-basivarsen (Dyne Therapeutics) added: ACHIEVE one-year data against a matched natural history cohort, with the six-month video hand opening time endpoint carrying the accelerated filing; NXC-201 (Immix Biopharma) added: NEXICART-2 interim at 89 percent complete response; VYD2311 (Invivyd) added: LIBERTY tolerability against Comirnaty, with the approval case to rest on antibody titers. Three rows whose near-term events had all passed (zerlasiran, camizestrant, apitegromab) retired to hold the table at 20 rows.
- 2026-10-01: rademikibart (Connect Biopharma) added: Seabreeze STAT Phase 2 in acute COPD exacerbations cut 28-day treatment failure to 2 of 78 against 11 of 81 on placebo (p=0.0122), three weeks after the same endpoint missed in asthma (p=0.153); pelacarsen retired after Lp(a)HORIZON failed with no congress date set and three other Lp(a) assets still on the table.
- 2026-10-02: rezpegaldesleukin (Nektar) added: REZOLVE-AA off-treatment durability at EADV 2026, SALT 20 held in 6 of 8 patients at four months and 5 of 8 at six months off treatment. ontunisertib (Agomab) added: the STENOVA open-label extension reported one balloon dilation and no surgeries across 49 patients treated up to 60 weeks.
Compiled from public sources; every drug name links to its ClinicalTrials.gov record. This page is general information, not medical advice, and it is not a substitute for individual care.