Company Product Description Indication Status
Phase I
Aslan Pharmaceuticals Ltd., of Singapore ASLAN-004 Monoclonal antibody targeting IL-13 receptor α1 subunit Moderate to severe atopic dermatitis Data monitoring committee completed planned safety data review of second dose cohort and recommended multiple ascending-dose study proceed as planned; data expected in early 2021
Daiichi Sankyo Co. Ltd., of Tokyo DS-1055 GARP-directed immuno-oncology therapy Advanced or metastatic solid tumors Dosed first patient in study testing drug in subjects who have progressed on standard treatments, including checkpoint inhibitors
Galera Therapeutics Inc., of Malvern, Pa. GC-4711 Small-molecule superoxide dismutase mimetic Non-small-cell lung cancer Dosed first patient in phase I/II Greco-1 trial in combination with stereotactic body radiation therapy
Immunitybio Inc., and Nantkwest Inc., both of Culver City, Calif. hAd5-COVID-19 COVID-19 vaccine targeting inner nucleocapsid and outer spike protein COVID-19 First patient dosed; study at the Hoag Hospital in Newport Beach, Calif., enrolling healthy adults up to age 55 to test safety and reactogenicity of 2 doses
Mesoblast Ltd., of Melbourne, Australia, and New York Remestemcel-L Mesenchymal stem cell-based therapy Refractory Crohn’s disease and ulcerative colitis Started phase I/II study at Cleveland Clinic to test therapy delivered by endoscope directly to areas of inflammation and tissue injury in up to 48 patients
Trippbio Inc., of Jacksonville, Fla. TD-213 Designed to block SARS-CoV-2 virus' replication and associated acute inflammatory response COVID-19 Completed enrollment 
Phase II
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Lumasiran Hydroxyacid oxidase 1 modulator Primary hyperoxaluria type 1 At Jan. 30, 2020, data cutoff, ongoing open-label extension with up to 22 months of exposure (11-22 months; median 15 months) showed sustained reductions in urinary oxalate excretion, resulting in 74% (35.7%-88.3%) mean maximal reduction in urinary oxalate relative to phase I/II baseline; 17/18 participants achieved normal or near-normal levels of urinary oxalate and mean eGFR levels remained stable
Ardelyx Inc., of Fremont, Calif. Tenapanor Sodium hydrogen exchanger 3 inhibitor Hyperphosphatemia Study in Japanese hemodialysis patients concludes drug significantly decreased serum phosphorous levels in dose-dependent manner; 30-mg twice-daily dosing groups produced statistically significant 2.6-mg/dL mean reduction (p<0.001) in serum phosphorous from baseline to end of 6-week treatment period vs. placebo
Ardelyx Inc., of Fremont, Calif. Tenapanor Sodium hydrogen exchanger 3 inhibitor Hyperphosphatemia When added to phosphate binders in refractory hyperphosphatemia in Japanese patients on dialysis, treatment with combo achieved statistically significant 2.1-mg/dL mean reduction (p<0.001) in serum phosphorus, with 87% of patients in tenapanor group achieving target phosphorus levels
Enlivex Therapeutics Ltd., of Nes Ziona, Israel Allocetra Autologous cell-based therapy Severe and critical COVID-19 Dosed first 2 patients; trial expected to recruit up to 24 patients and assess Allocetra in combination with standard of care
Medicinova Inc., of La Jolla, Calif. MN-166 (ibudilast) Oral small-molecule MIF inhibitor and PDE4/10 inhibitor Chemotherapy-induced peripheral neuropathy Plans to start multicenter, placebo-controlled, randomized phase IIb trial testing drug as potential treatment to reduce acute neurotoxicity severity and CIPN in patients with metastatic colorectal cancer
Phase III
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Lumasiran Hydroxyacid oxidase 1 modulator Primary hyperoxaluria type 1 6-month primary analysis of Illuminate-B pediatric study (n=18) showed 72% mean reduction in spot urinary oxalate:creatinine ratio from baseline, averaged across months 3 to 6 (primary endpoint); positive results also seen across secondary endpoints, including proportion of people (9/18) who achieved urinary oxalate levels at or below 1.5 times ULN
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Lumasiran Hydroxyacid oxidase 1 modulator Primary hyperoxaluria type 1 At data cutoff of May 1, 2020, extension period of Illuminate-A showed those randomized to study drug during double-blind period who continued through month 12 (n=24) maintained reduction in 24-hour urinary oxalate excretion, with 64% mean reduction compared to baseline, and 88% of these achieved levels at or below 1.5x ULN of urinary oxalate; those originally randomized to placebo who crossed over to lumasiran during extension (n=13) saw 57% mean reduction in 24-hour urinary oxalate excretion after 6 months and 77% achieved levels at or below 1.5 x ULN
Ardelyx Inc., of Fremont, Calif., and Kyowa Kirin Co. Ltd., of Tokyo Tenapanor Phosphate absorption inhibitor Hyperphosphatemia Additional analysis of long-term Phreedom study in people with CKD on dialysis showed decrease in mean serum phosphorus from 7.7 mg/dL at baseline to 5.1 mg/dL at end of randomized treatment; data from Block trial showed study drug reduced serum phosphorus as monotherapy in participants with CKD on dialysis; data from Amplify showed tenapanor + phosphate binders reduced serum phosphorus levels in difficult-to-control hyperphosphatemia
Dicerna Pharmaceuticals Inc., of Lexington, Mass. Nedosiran LDHA gene inhibitor Primary hyperoxaluria  Interim data showed all 13 participants (10 with PH1 and 3 with PH2) who received nedosiran, previously completed phase I Phyox1 trial and reached day 180 in ongoing Phyox3 trial achieved normal (n=12) or near-normal (n=1) urinary oxalate excretions at 1 or more time points; all 10 with PH1 and 2 of 3 with PH2 achieved normal excretions
Fibrogen Inc., of San Francisco Roxadustat HIF prolyl hydroxylase inhibitor Chronic kidney disease Pooled data from phase III development program in non-dialysis- and dialysis-dependent people with anemia of CKD showed major adverse cardiovascular events (MACE; all-cause mortality, myocardial infarction and stroke) and MACE+ (MACE + heart failure or unstable angina requiring hospitalization) rates were highest when hemoglobin (Hb) was <8 g/dL, decreased as Hb increased and were lowest when Hb levels were greater than or equal to 10 g/dL
Genmab A/S, of Copenhagen Darzalex (daratumumab) ADP ribosyl cyclase-1 inhibitor Multiple myeloma At preplanned interim analysis, part 2 of Cassiopeia study conducted by Intergroupe Francophone du Myelome, Dutch-Belgian Cooperative Trial Group for Hematology Oncology and Janssen Research & Development LLC met primary endpoint of improving progression-free survival, with 47% reduction in risk of progression or death (p<0.0001)
Moderna Inc., of Cambridge, Mass. mRNA-1273 COVID19 spike glycoprotein modulator COVID-19 infection Cove study completed enrollment with 30,000 participants
Omeros Corp., of Seattle Narsoplimab Mannan-binding lectin-associated serine protease-2 inhibitor Thrombotic microangiopathy Final data from pivotal trial in hematopoietic stem cell transplant-associated disease showed 61% complete response rate (CRR) in full analysis set (FAS, those who received at least 1 dose of study drug; p<0.0001 compared to 15% efficacy threshold agreed with FDA); 74% CRR in per-protocol (PP) population (those who received protocol-specified treatment for at least 4 weeks; p<0.0001 compared to the 15% threshold); 100-day survival was 68% in FAS, 83% in PP and 94% in complete responders; median overall survival was 274 days in FAS, 361 days in PP and not estimable for complete responders (>50% alive at last follow-up)
Soligenix Inc., of Princeton, N.J. SGX-301 Heat-shock protein inhibitor Cutaneous T-cell lymphoma Optional treatment across all lesions during compassionate use, safety portion of Flash trial (cycle 3), for total of 6 months in study, showed 49% who elected to receive study drug showed 50% or > reduction in combined Composite Assessment of Index Lesion Score vs. 40% who showed similar reduction after 12 weeks in cycle 2 (p=0.046); continued analysis of results from protocol mandated efficacy cycles 1 and 2 showed 12 weeks (cycle 2) with study drug was equally effective on patch (response 37%, p=0.0009) and plaque (response 42%, p<0.0001) lesions vs. cycle 1 placebo responses
Phase IV
Horizon Therapeutics plc, of Dublin Krystexxa (pegloticase)  Uricase stimulator Gout Initial data from ongoing Protect trial in people with uncontrolled disease who underwent kidney transplant (n=15) showed 5 participants completed treatment with sustained reductions in serum uric acid and 8 continue on therapy; 1 stopped per monitoring protocols and 1 terminated early over COVID-19 concerns


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