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The P-II trial assessed UBT251 (2, 4 & 6 mg, QW, SC) vs PBO & semaglutide (1mg) in 211 Chinese T2D pts, which showed HbA1c reduction of 2.16% vs 1.77% (semaglutide) & 0.66% (PBO) at 24wks. from a baseline of 8.12%
Trial also showed weight loss of up to 9.8% vs 4.8% (semaglutide) &…
Apogee Therapeutics Reports Part A P-II (APEX) Maintenance Data on Zumilokibart in Atopic Dermatitis
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Apogee has reported maintenance data from Part A of the P-II (APEX) trial assessing zumilokibart (APG777; 360mg at Q3M or Q6M) in pts with mod. to sev. atopic dermatitis
Trial showed 75% (Q3M) & 85% (Q6M) of Wk. 16 zumilokibart-responders maintained EASI-75, while 86% (Q3M) & 78% (Q6M) sustained vIGA 0/1, with continued deepening…
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AL-S Pharma has reported the global P-II (AP-101-02) trial data assessing AP-101 (IV, Q3W) vs PBO in 73 pts with sporadic ALS (n=52) & SOD1 mutations (n=21) for 24wks., followed by 24wk. OLE phase, where all pts received AP-101, & 16wk. safety follow-up
Trial met its 1EP for safety & tolerability, with AP-101 showing…
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Ascendis Pharma has reported the P-II (New InsiGHTS) trial data assessing TransCon hGH (lonapegsomatropin, QW) vs somatropin (QD) in 49 prepubertal children (1-10yrs.) with Turner syndrome
At Wk. 52, TransCon hGH showed improved annualized height velocity (AHV) with LS mean AHV of 9.05 vs 9.04 cm/year, irrespective of starting dose, with mean dose of…
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Pfizer has reported P-II (FOURLIGHT-1) trial data assessing atirmociclib + fulvestrant vs fulvestrant or everolimus plus exemestane in 264 pts with HR+, HER2- advanced or metastatic breast cancer who had received prior CDK 4/6 inhibitor-based treatment
Trial met its 1EP, showing improved PFS as assessed by the investigator, with benefit consistent across all subgroups. OS…
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The US FDA has granted FTDto ATX-01 for the treatment of Myotonic Dystrophy Type 1 (DM1)
ATX-01 showed increased free MBNL, improved splicing abnormalities, & restored function in animal models, & is being evaluated in the P-I/IIa (ArthemiR) study
ATX-01 is an RNA-based therapeutic designed to inhibit miR-23b, which results in increased MBNL protein…
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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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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.…
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The P-II (COMPASSION-03/AK104-201) trial assessed cadonilimab in pts with recurrent or metastatic CC who have failed prior Pt-based CT, showing mOS of 17.5mos. in pts regardless of PD-L1 expression, with18 & 24mos. OS rates of 47.8% & 40.9%, respectively
Updated data with 26.5mos. mFU showed that among 99 efficacy-evaluable pts achieving CR, mOS & PFS…

