Company Product Description Indication Status
Phase I
Amicus Therapeutics Inc., of Cranbury, N.J. AT-GTX-501 Ceroid lipofuscinosis protein gene stimulator CLN6 Batten disease Interim phase I/II data on Hamburg Motor & Language Aggregate Score showed, on combined scale of 0 to 6, mean rate of decline in children who reached 12-month time point (n=12) vs. natural history cohort (n=16) was 0.4 vs. 1.2 points, respectively; for those up to 24 months post-administration (n=8), decline was 0.6 vs 2.4 points vs. natural history cohort (n=16)
Basilea Pharmaceutica AG, of Basel, Switzerland Derazantinib FGFR inhibitor Intrahepatic cholangiocarcinoma Pooled data from 23 people in phase I and II studies, early access and compassionate use programs showed median progression-free survival of 7.2 months, with participants treated for median of 8.2 months
Kuur Therapeutics Inc., of Houston CAR-NKT cell therapy CAR-NKT cell therapy Relapsed/refractory neuroblastoma Interim findings published in Nature Medicine in children with high-risk disease showed that expressing the CAR with IL-15 enhanced tumor-fighting capabilities and in vivo persistence of autologous NKT cells; 2 of 3 patients studied showed tumor reduction following CAR-NKT infusion: 1 classified as stable disease and the other as partial response
Vedanta Biosciences Inc., of Cambridge, Mass. VE-202 Live biotherapeutic  Inflammatory bowel disease In 74 healthy volunteers dosed with 11-strain consortium of study therapy or placebo, live biotherapeutic colonized gut abundantly and above background strains detected by qPCR; colonization was most effective with vancomycin pre-treatment followed by multiple doses of consortium
Phase II
Amylyx Pharmaceuticals Inc., of Cambridge, Mass. AMX-0035 Sodium phenylbutyrate + tauroursodeoxycholic acid Amyotrophic lateral sclerosis Long-term safety data from phase II/III Centaur study showed no new safety concerns; most adverse events did not lead to study drug modification, interruption or withdrawal; using the ATLIS (Acute Test of Limb Isometric Strength) test to measure muscle strength, the mean rate of change per month in ATLIS scores trended toward AMX-0035 benefit, with the arm score reaching significance (p=0.04), though the leg muscle strength score did not reach statistical significance
Eli Lilly and Co., of Indianapolis Mirikizumab IL-23 antagonist Crohn's disease Serenity study in moderately to severely active disease achieved key secondary outcomes at week 52, with nearly 60% of participants showing endoscopic response (58.5% in randomized I.V. dosing group and 58.7% in subcutaneous group) and > 45% achieving patient-reported outcomes remission (46.3% in I.V. group and 45.6% in SC group); among subgroup with endoscopic remission at week 12, 50% and 64.3% in I.V. (n=6) and SC (n=14) groups, respectively, also had endoscopic remission at week 52
Gilead Sciences Inc., of Foster City, Calif., and Galapagos NV, of Mechelen, Belgium Filgotinib JAK1 inhibitor Ulcerative colitis Phase IIb/III Selection trial showed higher proportions of both biologic-naïve and biologic-experienced adults with moderately to severely active disease treated with study drug at 200 mg achieved clinical remission at week 10  vs. placebo (26.1% vs. 15.3%, p=0.0157, and 11.5% vs. 4.2%, p=0.0103, respectively); at week 58, 37.2% who received filgotinib 200 mg achieved clinical remission vs. 11.2% for placebo (p˂0.0001)
Gossamer Bio Inc., of San Diego GB-004 Hypoxia-inducible factor 1-alpha stabilizer Ulcerative colitis Screening initiated in Shift-UC study expected to enroll up to 195 people with active disease despite 5-aminosalicylate treatment; primary endpoint is clinical remission at week 12; participants will continue blinded treatment through week 36 with option to roll onto open-label extension
Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J. Tremfya (guselkumab) IL-23A inhibitor Crohn's disease Interim data from Galaxi 1 study in moderately to severely active disease showed, at week 12, greater reductions from baseline in Crohn's Disease Activity Index (CDAI) in each dose group (200, 600 or 1,200 mg I.V.) vs. placebo (least squares means: -154.1, -144.3, -149.5 vs. -36, respectively; all p<0.001); 54%, 56%, 50%, respectively, of those assigned to each dose achieved clinical remission vs. 15.7% for placebo (p<0.001); higher proportion treated with study drug achieved clinical response, patient-reported outcome remission, clinical-biomarker response and endoscopic response (all p<0.001) vs. placebo
Rhovac AB, of Lund, Sweden RV-001 RhoC GTPase modulator Prostate cancer Recruitment in phase IIb Bravac (RhoVac-002) study remained slower due to COVID-19 pandemic; additional sites opening and full enrollment expected in second quarter of 2021
Phase III
Astellas Pharma Inc., of Tokyo, and Seagen Inc., of Bothell, Wash. Padcev (enfortumab vedotin-ejfv) Nectin-4 modulator Urothelial cancer Second cohort in pivotal EV-201 trial in locally advanced or metastatic disease previously treated with PD-1/L1 inhibitor and ineligible for cisplatin showed 52% objective response rate per blinded independent review and median duration of response of 10.9 months
Astrazeneca plc, of Cambridge, U.K. AZD-7442 COVID-19 Spike glycoprotein inhibitor COVID-19 infection Long-acting antibody combination advancing into 2 trials in >6,000 participants; first will evaluate safety and efficacy to prevent infection for up to 12 months in about 5,000 participants and second will evaluate post-exposure prophylaxis and preemptive treatment in about 1,100 people
Bristol Myers Squibb Co., of New York Zeposia (ozanimod)  Sphingosine-1-phosphate receptor-1/5 modulator Ulcerative colitis True North study in adults with moderate to severe disease met both primary efficacy endpoints, showing statistically significant improvement vs. placebo in clinical remission at week 10 (18.4% vs. 6%; p<0.0001) and in maintenance at week 52 (37% vs. 18.5%; p<0.0001); study also met key secondary endpoints, including clinical response, endoscopic improvement and mucosal healing in induction at weeks 10 and 52
Celltrion Group, of Incheon, South Korea CT-P59 Monoclonal  antibody targeting SARS-CoV-2 COVID-19 prophylaxis in people who had contact with SARS-CoV-2-infected patients Launched the study of approximately 1,000 patients
Celltrion Healthcare, of Incheon, South Korea Remsima SC (CT-P13 SC) Biosimilar of infliximab Crohn’s disease and ulcerative colitis Of the 54 patients treated with CT-P13 PC, 77.8% achieved clinical remission at week 54
Janssen Pharmaceutical Co., of Spring House, Pa., a unit of Johnson & Johnson Stelara (ustekinumab) Monoclonal  antibody targeting interleukin-12 and IL-23 Crohn's disease In the Im-Uniti long-term extension study, 57% of patients maintained clinical response and 55% maintained remission through 5 years of treatment; 93% were steroid free
Seres Therapeutics Inc., of Cambridge, Mass.  SER-109 Microbiome therapeutic C. difficile infection In the Ecospor III study, drug produced a 30.2% absolute reduction of recurrence of infection compared to placebo at 8 weeks post-treatment; at 12-weeks, rate of recurrence was 16.7% for SER-109 and 47.8% for placebo p<0.001
Takeda Pharmaceutical Co. Ltd., of Osaka Japan Entyvio (vedolizumab) Monoclonal antibody targeting alpha4beta7 integrin Moderately to severely active ulcerative colitis In the Visible open-label extension study, 68.9% of patients had clinical remission and 70% had corticosteroid-free clinical remission at week 108

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