Company Product Description Indication Status
Phase I
Akston Biosciences Corp., of Beverly, Mass. AKS-452 COVID-19 spike glycoprotein modulator  COVID-19 infection First participants have been dosed in phase I/II open-label study set to evaluate 176 healthy volunteers ages 18 to 65; participants will receive 1 dose or 2 doses 28 days apart
Allogene Therapeutics Inc., of South San Francisco, and  Springworks Therapeutics Inc., of Stamford, Conn. ALLO-715 + nirogacestat Anti-B-cell maturation antigen AlloCAR T therapy + gamma-secretase inhibitor Multiple myeloma First participant with relapsed or refractory MM received ALLO-715 in combination with nirogacestat as part of ongoing open-label study
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. ALN-AGT Angiotensinogen ligand inhibitor Hypertension Treatment was associated with dose-dependent knockdown of serum angiotensinogen and reductions in blood pressure with durability supporting potential once-quarterly or biannual dosing regimen; reported to be safe and well-tolerated
Artelo Biosciences Inc., of La Jolla, Calif. ART-27.13 G protein-coupled receptor full agonist Appetite loss First patient dosed in CAReS trial for cancer-related anorexia; initial safety data expected from before end of 2021
Cardiff Oncology Inc., of San Diego Onvansertib  Polo-like kinase 1 inhibitor Metastatic colorectal cancer Interim update from ongoing phase Ib/II trial of patients treated with onvansertib + FOLFIRI/bevacizumab shows 7 of 18 evaluable patients achieved a partial response; 4 patients had a confirmed PR with 1 patient going on to curative surgery
Cardiol Therapeutics Inc., of Oakville, Ontario Cardiolrx (cannabidiol) THC-free nanoparticle formulation of cannabidiol Myocarditis Company plans to file a IND with FDA for a phase II international trial in acute myocarditis after randomized, placebo-controlled phase I study demonstrated that Cardiolrx was safe and generally well-tolerated at all dose levels
Rubius Therapeutics Inc.  RTX-321 Genetically engineered antigen presenting red blood cells  HPV 16-positive cancers First patient dosed in trial for the treatment of human papillomavirus 16-postive cancers
Turning Point Therapeutics Inc.  TPX-0131 ALK inhibitor Non-small-cell lung cancer Initiated phase I/II Forge-1 study enrolling patients with locally advanced or metastatic TKI-pretreated ALK-positive NSCLC; U.S. trial site starts to follow
VBI Vaccines Inc., of Cambridge, Mass. VBI-2601 Recombinant, protein-based virus-like particle vaccine Hepatitis B virus infection Data from phase Ib/IIa study in chronic HBV patients show robust HBV specific T-cell responses in ≥ 50% of evaluable patients across all study arms; well-tolerated with no safety signals observed at both low- and high-dose levels
Phase II
Antengene Corp. Ltd., of Shanghai ATG-008 (onatasertib) mTORC1/2 inhibitor Advanced solid tumors with NFE2L2, STK11, RICTOR or other specific genetic alterations First patient treated in the Bunch study testing the safety and efficacy of ATG-008
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda Mavrilimumab  Monoclonal antibody targeting granulocyte macrophage colony-stimulating factor receptor alpha Severe COVID-19 pneumonia and hyperinflammation In the cohort of patients who required supplemental oxygen in the phase II portion of the phase II/III study, 86.7% of the patients given mavrilimumab were alive and free of mechanical ventilation at day 29, compared to 74.4% of patients give placebo (p=0.1224); mavrilimumab produced a 65% reduction in the risk of requiring mechanical ventilation or death through day 29 (p=0.0175); mortality at day 29 was 8% for mavrilimumab and 20.5% for placebo (p=0.0718), translating into a 61% reduction in death (p=0.0726)
Lyra Therapeutics Inc., of Watertown, Mass. LYR-210 Mometasone furoate in bioresorbable polymeric matrices Chronic rhinosinusitis In the phase II Lantern study, after a single 7,500-µg dose, 100% of patients with or without polyps achieved the minimal clinically important difference of 8.9 points for SNOT-22 total score by week 24; the 7,500-µg dose improved bilateral ethmoid Zinreich scores at week 24 compared to the control (p=0.031); 1 patient in the 7,500-µg group and 2 patients in the 2,500-µg group required a rescue treatment compared to 7 patients in the control group; 7,500-µg dose improved nasal blockage, nasal discharge and facial pain composite score compared to the control at week 24 (p=0.003) and at earlier timepoints
Marinomed Biotech AG, of Korneuburg, Austria Tacrosolv Formulation of the immunomodulator tacrolimus Allergic rhinoconjunctivitis Completed treatment of patients; results expected by mid-2021
Sage Therapeutics Inc. and Biogen Inc., both of Cambridge, Mass. SAGE-324 Neuroactive steroid GABAA receptor positive allosteric modulator Essential tremor In the Kinetic study, SAGE-324 produced a 36% reduction in upper limb tremor amplitude, which is part of The Essential Tremor Rating Assessment Scale (TETRAS), from baseline to day 29 compared to a 21% reduction for placebo (p=0.049); SAGE-324 was numerically superior to placebo for TETRAS activities of daily living score at all time points
Phase III
Arcutis Biotherapeutics Inc., of West Lake Village, Calif. Roflumilast cream (ARQ-151) Phosphodiesterase type 4 inhibitor Mild to moderate atopic dermatitis Started the 650-patient Integument-Ped study in patients ages 2 to 5; primary endpoint is Investigator Global Assessment Success, defined as a Validated Investigator Global Assessment - Atopic Dermatitis score of ‘clear’ or ‘almost clear’ plus a 2-grade improvement from baseline at week 4; secondary endpoints include Worst Itch-Numerical Rating Scale and proportion of patients who attain at least a 75% reduction in the Eczema Area and Severity Index at week 4; data expected in the second half of 2022
Astrazeneca plc, of Cambridge, U.K. Farxiga (dapagliflozin) SGLT2 inhibitor Hospitalized COVID-19 with risk of developing serious complications The Dare-19 didn’t meet the primary endpoint of prevention of organ dysfunction and all-cause mortality or the primary endpoint of change in clinical status from early recovery to death, at 30 days; results will be presented at the American College of Cardiology Scientific Sessions in May 2021
Oxford University Inhaled budesonide Corticosteroid COVID-19 patients over 50 In the Principle platform study, estimated median time to self-reported recovery for inhaled budesonide was 1.134 days compared to 5.410 days for standard of care; 32% of those taking inhaled budesonide, compared to 22% of those receiving SOC recovered within the first 14 days; 8.5% of patients in the budesonide group were hospitalized with COVID-19 compared to 10.3% of the SOC group
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and F. Hoffmann-La Roche Ltd., of Basel, Switzerland Regen-Cov (casirivimab and imdevimab) Monoclonal antibody targeting SARS-CoV-2 Recently infected asymptomatic COVID-19 In the 2069B study, treatment reduced the overall risk of progressing to symptomatic COVID-19 by 31% through day 29 (p=0.0380) and by 76% after day 3 (p=0.0007); no patients treated with the antibodies had COVID-19-related hospitalizations or emergency room visit, compared to 6% of patients taking placebo (p=0.029)
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and F. Hoffmann-La Roche Ltd., of Basel, Switzerland Regen-Cov (casirivimab and imdevimab) Monoclonal antibody targeting SARS-CoV-2 COVID-19 prophylaxis among household contacts of SARS-CoV-2-infected individuals In the 2069A study, the treatment reduced the risk of symptomatic infections by 81% through day 29 (p=0.0001); patients who developed symptomatic infection resolved their symptoms on average within 1.2 weeks, compared to 3.2 weeks for placebo


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