Company Product Description Indication Status
Phase I
Leading Biosciences Inc., of Carlsbad, Calif. LB-1148 (tranexamic acid) Serine protease inhibitor Intra-abdominal adhesions GI surgery study showed no abdominal adhesions at follow-up surgery in 2 people previously treated with study drug
Medincell SA, of Montpellier, France Ivermectin Antiviral, long-acting injectable depot formulation COVID-19 infection Trial validated safety of study drug dosed daily in oral form to simulate continuous release of active substance by long-acting injection; pharmacokinetic data showed limited peak circulating plasma concentration in first 12 hours
Siteone Therapeutics Inc., of South San Francisco ST-2427 NaV1.7 inhibitor Moderate to severe pain First healthy adults enrolled in study to inform doses and design of phase II studies in people with acute pain
Sorrento Therapeutics Inc., of San Diego COVI-MSC Human allogeneic adipose-derived mesenchymal stem cells COVID-19 infection Phase Ib study met target enrollment of 10 participants with COVID-19-related acute respiratory failure and all were discharged from hospital within 3 days following final infusion; pivotal study planned to support EUA submission
Syndax Pharmaceuticals Inc., of Waltham, Mass. SNDX-5613 Leukemia Menin inhibitor In phase I portion of Augment-101 trial, overall response rate (ORR) for evaluable participants (n=31) was 48% (n=15), with 67% (n=10) of these achieving minimum residual disease-negative status and 4 proceeding to stem cell transplant; ORR in evaluable participants harboring mixed lineage leukemia rearrangement (n=24) was 54% (n=13) and in those harboring NPM1c mutation (n=7) was 29% (n=2)
Phase II
180 Life Sciences Corp., of Menlo Park, Calif. Repurposed adalimumab TNF inhibitor Dupuytren contracture Following delay due to COVID-19 pandemic, data from phase IIb trial in 181 participants collected and submitted for analysis and review and on track to report in fourth quarter of 2021
Bioarctic AB, of Stockholm, and Eisai Co. Ltd., of Tokyo Lecanemab (BAN-2401) Beta-amyloid antagonist Alzheimer's disease Alzheimer's Research & Therapy published data from phase IIb Study 201 that showed reduction of clinical decline across clinical and biomarker endpoints at highest dose, which is being assessed in pivotal phase III Clarity AD study
Ionis Pharmaceuticals Inc., of Carlsbad, Calif. ION-373 Glial fibrillary acidic protein inhibitor Alexander disease Pivotal phase II/III study initiated in up to 58 people who will receive study drug or placebo for 60 weeks, with all participants set to receive ION-373 for additional 60-week open-label period
Ripple Therapeutics Corp., of Toronto IBE-814 (dexamethasone 6-month intravitreal implant) Glucocorticoid receptor agonist Inflammatory retinal disease First of 50 participants with diabetic macular edema or macular edema due to retinal vein occlusion dosed in Ripple-1 trial; efficacy endpoints include improvement in macular edema and visual acuity
T3D Therapeutics Inc., of Research Triangle Park, N.C. T3D-959 Dual PPAR delta/gamma agonist Alzheimer's disease First of up to 256 participants with mild to moderate disease dosed in Pioneer study following pause and adaptations due to COVID-19 pandemic
Phase III
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Vutrisiran TTR gene inhibitor Transthyretin-mediated amyloidosis Previously reported findings from Helios-A, which met primary and secondary endpoints, presented at AAN annual meeting
Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. Baricitinib  JAK inhibitor Severe alopecia areata In the Brave-AA1 study, 35% of patients taking baricitinib 4 mg/day and 22% of patients treated with baricitinib 2 mg/day achieved 80% or more scalp hair coverage at week 36, compared to 5% of patients in the placebo group (p≤0.001 for both doses compared to placebo); in the Brave-AA2 study, 33% of the 4-mg group, 17% of the 2-mg group and 3% of the placebo group met the goal (p≤0.001 for both doses compared to placebo)
Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. Erleada (apalutamide) + Zytiga (abiraterone)  Androgen receptor antagonist + cytochrome P450 17 inhibitor Metastatic castration-resistant prostate cancer Company will not pursue regulatory submissions after Acis combination study with prednisone, which met primary endpoint of progression-free survival, showed no benefit over active control of Zytiga + prednisone in key secondary endpoints, including overall survival 
Jazz Pharmaceuticals plc, of Dublin Xywav (calcium, magnesium, potassium, and sodium oxybates) GABA B receptor agonist   Idiopathic hypersomnia After the open-label optimization of up to 14 weeks and the 2-week stable-dose period, Epworth Sleepiness Scale (ESS) score improved from a mean of 15.7 to 6.1; Idiopathic Hypersomnia Severity Scale (IHSS) score improved from a mean of 31.6 to 15.3; in the randomized withdraw period, ESS scores LS mean difference for those who continued Xywav treatment compared to those who were switched to placebo was −6.51 (p<0.0001) and the difference in IHSS scores was −12 (p<0.0001), both in favor of Xywav
Morphosys AG, of Planegg, Germany, and Incyte Corp., of Wilmington, Del. Tafasitamab CD19-targeting monoclonal antibody Follicular or marginal zone lymphoma First of 600+ adults dosed in Inmind combination study with lenalidomide + rituximab in people with relapsed/refractory disease; primary endpoint is progression-free survival in follicular lymphoma 
Steba Biotech SA, of Luxembourg Padeliporfin Impact Vascular-targeted photodynamic therapy   Low grade upper tract urothelial cancer First of 100 patients treated in the Enlighted study; primary efficacy endpoint is the absence of tumors in the entire ipsilateral calyces’ renal pelvis and ureter; data expected in 2022

Notes

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