Company Product Description Indication Status
Phase I
Forendo Pharma, a portfolio company of Stockholm-based Karolinska Development AB FOR-6219 HSD17B1 inhibitor Endometriosis The phase Ia part in healthy pre- and postmenopausal women showed pharmacokinetics of FOR-6219 were dose-proportional; steady state was reached within 3 days; half-life was 16-18 hours; in the phase Ib part in premenopausal healthy women treated for 14 days during the proliferative phase of the menstrual cycle, the women experienced a continued normal ovulatory menstrual cycle; plans to run a phase II study
Prometheus Biosciences Inc., of San Diego PRA-023 Humanized IgG1 monoclonal antibody targeting tumor necrosis factor-like ligand 1A Ulcerative colitis and Crohn's disease Started dosing of healthy volunteers in the multiple ascending-dose portion of the ongoing study testing the safety, tolerability, pharmacokinetics and pharmacodynamics of PRA-023
Phase II
Arena Pharmaceuticals Inc., of Park City, Utah Olorinab  Cannabinoid receptor 2 agonist Abdominal pain due to irritable bowel syndrome The phase IIb Captivate study didn't meet its primary endpoint of improvement in AAPS from baseline to week 12; in the prespecified subgroup of patients with AAPS scores of 6.5 or greater, the 50-mg dose produced reduction in AAPS of 1.64 points compared to placebo and 3.93 points from baseline at week 12 (p=0.01)
Azura Ophthalmics Ltd., of Tel Aviv, Israel  AZR-MD-001 Breaks bonds between abnormal keratin proteins Meibomian gland dysfunction Both the 0.5% and 1% dose groups reduced the Ocular Surface Disease Index (p<0.01) with the 0.5% dose producing improvements over control by month 3 (p<0.05); both doses increased the Meibomian Gland Score (p<0.01) with improvement at 1% dose over the control at month 2 (p<0.05) and month 3 (p<0.01); both doses increased the Meibomian Glands Yielding Liquid Secretion score (p<0.01) with improvement at 1% dose over the control group at month 1 (p<0.01) and month 3 (p<0.01)
Biomarin Pharmaceutical Inc., of San Rafael Vosoritide Analogue of C-type natriuretic peptide  Achondroplasia Completed enrollment in the study; top-line data expected in mid-2022
Longeveron Inc., of Miami Lomecel-B Bone marrow-derived medicinal signaling cell product Aging frailty Completed the phase IIb study; top-line results expected in the third quarter of 2021
Marker Therapeutics Inc., of Houston MT-401 MultiTAA-specific T-cell product Acute myeloid leukemia following an allogeneic stem cell transplant  Treated first of 160 patients in the study; primary objective is relapse-free survival for patients with adjuvant disease; primary objectives are complete response rate and duration of complete response for the patients with active disease; top-line data for the active disease group expected in the first quarter of 2022
Morphosys AG, of Planegg, Germany, and Glaxosmithkline plc, of London Otilimab (MOR-103/GSK-3196165) Monoclonal antibody targeting granulocyte-macrophage colony-stimulating factor Severe pulmonary COVID-19 related disease In the phase IIa Oscar study, treated first patient in the expanded cohort of patients 70 and older
Reveragen Biopharma Inc., of Rockville, Md., and Santhera Pharmaceuticals Holding AG, of Pratteln, Switzerland Vamorolone  Dissociative steroid Duchenne muscular dystrophy Last patient completed their last visit for the 6-month period of the phase IIb Vision-DMD study; top-line 6-month data expected in the second quarter of 2021
Spectrum Pharmaceuticals Inc., of Henderson, Nev. Poziotinib  EGFR tyrosine kinase inhibitor Previously treated non-small-cell lung cancer with EGFR or HER2 exon 20 insertion mutations In cohort 5d, the 8-mg twice-daily dose produced a partial response in 3 of 10 patients in the cohort with 2 patients still too early to assess; in cohort 5a, the 16-mg once-daily dose produced a partial response in 2 of 10 patients
Phase III
Amgen Inc., of Thousand Oaks, Calif. Blincyto (blinatumomab) CD19-directed CD3 T-cell bispecific T-cell engager High-risk first-relapse B-cell precursor acute lymphoblastic leukemia Data published in The Journal of the American Medical Association showed 69% of patients treated with Blincyto were alive and event-free compared with 43% of patients treated with chemotherapy; 93% of patients treated with Blincyto who had minimal residual disease (MRD) at baseline achieved MRD-negative remission compared with 24% of patients treated with chemotherapy; 36-month overall survival estimate was 81.1% for Blincyto compared to 55.8% in the chemotherapy group; median OS has not been met
Bharat Biotech International Ltd., of Hyderabad, India, and Ocugen Inc., of Malvern, Pa. Covaxin Virion inactivated COVID-19 vaccine COVID-19 prophylaxis At the first interim analysis, there were  36 cases of COVID-19 in the placebo group and 7 cases in the group who received Covaxin, resulting in a point estimate of vaccine efficacy of 80.6%
Eli Lilly and Co., of Indianapolis, and Incyte Corp., of Wilmington, Del. Baricitinib JAK1/JAK2 inhibitor Alopecia areata In Brave-AA2 study, once-daily 2-mg and 4-mg doses in adults with severe disease met primary efficacy endpoint at week 36, showing statistically significant improvement in scalp hair regrowth vs. placebo
Vir Biotechnology Inc., of San Francisco, and Glaxosmithkline plc, of London VIR-7831/GSK-4182136 Monoclonal antibody targeting SARS-CoV-2 Hospitalized COVID-19 The VIR-7831/GSK-4182136 arm of the ACTIV study met initial prespecified criteria to continue to the next phase, but sensitivity analyses of the available data raised concerns about the magnitude of potential benefit; independent data and safety monitoring board recommended the arm be closed to enrollment while the data mature

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