Shots:
The P-III (ADorable‑1) study data assessed Ebglyss vs PBO in 363 pediatric pts (≥6mos.) with mod. to sev. AD; Topical corticosteroids were required for 2 wks. pre-randomization through Wk. 16, with tapering or discontinuation allowed once pts achieved IGA ≤2
At Wk. 16, the trial met it 1EP & key 2EPs, with 63% vs…
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Sana has reported 14mos. follow-up data from a FIH study of UP421, a hypoimmune-engineered allogeneic islet cell therapy, transplanted into a patient with Type 1 Diabetes without immunosuppression
Results showed sustained survival & function of pancreatic β cells. At 14mos., fasting & MMTT C-peptide levels matched the first 6mos. & exceeded in mos. 9-12,…
Shots:
Bayer has reported the P-III (FIND-CKD) trial data assessing Kerendia (finerenone; 10 or 20mg) vs PBO, both in addition to SoC, in >1,500 adults with non-diabetic chronic kidney disease
Trial met its 1EP, showing a statistically significant improvement in eGFR slope, defined as the mean annual change in eGFR from baseline to Month 32; data…
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OLE study assessed repinatrabit (JNT-517) in adolescents with PKU. Early time-point data remain embargoed to preserve parent-study blinding, with additional randomized-period results to be presented later
First cohort receiving repinatrabit (75mg, PO, BID) showed a 67% mean reduction in Phe from baseline (Day 56/OLE Month 1), with responses seen in all pts, incl. prior…
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Ultragenyx has reported the P-III (Enh3ance) study data assessing DTX301 AAV8 gene therapy vs PBO for the treatment of ornithine transcarbamylase (OTC) deficiency
DTX301 reduced 24hr. plasma ammonia by 18% vs PBO at Wk. 36, with ammonia levels maintained in the normal range, despite a 27% mean decrease in scavenger drugs & ~13% increase…
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Aplagon has dosed the first patient with APAC (IV) in its P-IIa (HEALING) trial in Finland for peripheral arterial occlusive disease (PAOD), leading to chronic limb-threatening ischemia (CLTI)
Trial will evaluate APAC in ~42 CTLI pts across 4 cohorts to assess safety, efficacy, & effects on thrombo-inflammatory biomarkers. Additionally, an associated PET imaging study with…
Shots:
The P-II trial consists of 4 overlapping stages: Stage 1 (vs PBO) for tilrekimig & ompekimig; Stage 2 (dose-ranging) & Stage 3 (ongoing) evaluated tilrekimig, with Stage 3 involving biologic-experienced adults; Stage 4 is an ongoing ompekimig dose-ranging study
Trial met its 1EP with more pts achieving EASI-75 at Wk. 16; PBO-adjusted rates were…
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BMS has reported the P-II/III (SUCCESSOR-2) trial data assessing mezigdomide (PO) + carfilzomib & dexamethasone vs carfilzomib & dexamethasone alone in pts with r/r multiple myeloma
The P-III portion showed improved PFS (1EP), with safety consistent with the known profile of the regimen & mezigdomide. The trial will continue to follow pts for survival…
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Insilico Medicine & TaiGen have reported complete enrolment of the trial & dosing of the first patient with ISM4808 in the P-I trial to treat CKD-related anemia
The P-I study consists of both single-ascending-dose & multiple-ascending-dose cohorts, designed to evaluate ISM4808 in healthy adults against PBO
In Dec 2025, TaiGen gained exclusive rights to…
Shots:
The P-II trial assessed 5 doses of petrelintide (SC, QW) vs PBO for 42wks. in 493 overweight or obese pts (mean BMI of 37 kg/m²) with weight-related comorbidities
Trial met its 1EP, with Q4W dose-escalated injections delivering significant weight loss across 5 arms after 28wks. & sustaining ~10.7% vs 1.7% mean reduction through Wk.…

