Company Product Description Indication Status
Phase I
Allena Pharmaceuticals Inc., of Newton, Mass. ALLN-346 Urate-degrading enzyme Hyperuricemia in gout and advanced chronic kidney disease Dosed first of about 24 healthy volunteers; primary objective is to assess safety and tolerability over 28 days; data expected in fourth quarter of 2020
Celularity Inc., of Warren, N.J. CYNK-001 Human placental hematopoietic stem cell-derived NK cells COVID-19 Dosed first patient in phase I/II study
Compugen Ltd., of Holon, Israel COM-701 Anti-PVRIG antibody Advanced solid tumors First of about 100 participants dosed in phase I/II combination trial with Bristol Myers Squibb Co.'s PD-1 inhibitor Opdivo (nivolumab) and anti-TIGIT antibody, BMS-986207 
Cyclo Therapeutics Inc., of Gainesville, Fla. Trappsol Cyclo Cholesterol binding agent Niemann Pick disease type C1 With 3 additional participants completing 48-week phase I/II study, 6 of 7 met primary endpoint, achieving at least 1-point reduction in 2 or more of 17-domain NPC Clinical Severity Scale measures; 5 of 7 improved in co-primary outcome measure of Clinician's Global Impression of Improvement scale
Kamada Ltd., of Rehovot, Israel Plasma-derived anti-SARS-CoV-2 IgG Plasma-derived immunoglobulin therapy COVID-19 infection With phase I/II trial in hospitalized, non-ventilated people with pneumonia fully enrolled (n=12), interim data showed symptom improvement within 24 to 48 hours of treatment in 11, subsequently discharged within median hospital stay of 4.5 days of treatment; 1 who completed 14-day post-treatment follow-up subsequently required mechanical ventilation
Silence Therapeutics plc, of London SLN-124 TMPRSS6 gene inhibitor Beta-thalassemia  First of up to 24 healthy volunteers dosed
Transgene SA, of Strasbourg, France TG-6002 Oncolytic vaccinia virus therapy expressing FCU1  Advanced gastrointestinal tumors Independent safety review committee recommended trial be continued and that dose level be increased to a 3x109 pfu in the absence of dose-limiting toxicity at the 109 pfu dose; first translational data showed intravenous administration results in TG-66002 circulating transiently in bloodstream and inducing production of 5-FU at therapeutic doses
Translate Bio Inc., of Lexington, Mass. MRT-5005 CFTR gene modulator Cystic fibrosis Enrollment and dosing in phase I/II trial resumed following pause due to COVID-19 pandemic
Phase II
Athenex Inc., of Buffalo, N.Y. Encequidar + paclitaxel  P-Glycoprotein 1 inhibitor Breast cancer In conjunction with Quantum Leap Healthcare Collaborative, 2 arms added to I-Spy 2 trial to evaluate combination study drug + PD-1 inhibitor dostarlimab (Glaxosmithkline plc) in people with stage 2/3 HER2- cancer and + trastuzumab in those with HER2+ cancer; primary endpoint is objective response rate
Bellus Health Inc., of Laval, Quebec BLU-5937 P2X3 antagonist Refractory cough Phase IIb confirmatory trial, expected to initiate in fourth quarter of 2020 with interim analysis in mid-2021 and top-line data in second half of 2021, will evaluate 3 doses in 280 people; primary efficacy endpoint is placebo-adjusted change in 24-hour cough frequency using cough recorder
Clene Nanomedicine Inc., of Salt Lake City CNM-Au8 Gold nanocrystals Amyotrophic lateral sclerosis  Rescue-ALS trial in newly symptomatic people fully enrolled (n=8) ahead of schedule; top-line data expected in mid-2021
Goldfinch Bio Inc., of Cambridge, Mass. GFB-887 TRP cation channel C5 inhibitor Focal segmental glomerulosclerosis; diabetic nephropathy First of about 125 participants enrolled in Traction-2 study assessing activity of multiple doses over 12 weeks, measured by percentage change from baseline in urine protein to creatine ratio, urine albumin to creatinine ratio and 24-hour urine protein/albumin excretion
Insmed Inc., of Bridgewater, N.J. Brensocatib  Inhibitor of dipeptidyl peptidase 1 Non-cystic fibrosis bronchiectasis Data from the Willow study published in The New England Journal of Medicine showed both dose levels prolonged time to first pulmonary exacerbation compared to placebo (p=0.03 for the 10-mg group; p=0.04 for the 25-mg group); risk of exacerbation at any time during the trial was reduced by 42% for 10 mg vs. placebo (p=0.03) and by 38% for 25 mg vs. placebo (p=0.046)
Synairgen plc, of Southampton, U.K. SNG-001 Interferon-beta ligand Chronic obstructive pulmonary disease Interim analysis showed lung antiviral responses to viral infection were enhanced in those who received study drug vs. placebo, measured by increases in gene expression of interferon-beta-dependent antiviral biomarkers MX1 (p<0.001) and OAS1 (p=<0.001) in sputum cells; exacerbating patients who received study drug showed difference vs. placebo in change from baseline morning peak expiratory flow rate over days 2-15 of 25.5L/min (p=0.041)
Verona Pharma plc, of London Ensifentrine CFTR stimulator; PDE4 inhibitor; PDE3 inhibitor COVID-19 Started 45-patient pilot study to test drug delivered via pressurized metered-dose inhaler formulation in hospitalized patients; primary endpoint is proportion of patients recovered from COVID-19 and no longer hospitalized at day 29
Phase III
Albireo Pharma Inc., of Boston Odevixibat Bile acid transport inhibitor Progressive familial intrahepatic cholestasis  Pivotal Pedfic 1 trial met co-primary endpoints of reduction in pruritus (p=0.004) and serum bile acids (p=0.003) vs. placebo in children with PFIC1 or PFIC2 disease
Astrazeneca plc, of Cambridge, U.K. Breztri Aerosphere (budesonide/glycopyrronium/formoterol fumarate) Beta 2 adrenoceptor agonist; muscarinic receptor antagonist Chronic obstructive pulmonary disease Post-hoc analysis of Ethos trial showed benefit of study drug in reducing rate of moderate or severe disease exacerbations across all seasons vs. Bevespi Aerosphere (glycopyrronium/formoterol fumarate, Astrazeneca plc) in people with moderate to very severe disease
Biomarin Pharmaceutical Inc., of San Rafael, Calif. Vosoritide C-type natriuretic peptide ligand Achondroplasia Detailed data, published in The Lancet, showed statistically significant increase in annualized growth velocity over 52 weeks in treated children, with adjusted mean difference of 1.57 cm per year vs. placebo (p <0.0001)
Calliditas Therapeutics AB, of Stockholm Nefecon Oral formulation of the immunosuppressant budesonide Autoimmune renal disease IgA nephropathy First patient in China randomized in the Nefigard study
Corbus Pharmaceuticals Holdings Inc., of Norwood, Mass. Lenabasum Cannabinoid receptor type 2 agonist Diffuse cutaneous systemic sclerosis In the Resolve-1 study, median American College of Rheumatology Combined Response Index for Systemic Sclerosis scores at week 52 were 0.888 for patients taking lenabasum and 0.887 for placebo
HRA Pharma Rare Diseases SAS, of Paris Metyrapone Cortisol synthesis inhibitor Cushing's syndrome In Phase III/IV Prompt trial, study drug controlled mean of 3 urinary free cortisol (UFC) values in 80% of participants at week 12 with either normalization or at least 50% decrease
Merck & Co. Inc., of Kenilworth, N.J. Gefapixant (MK-7264) P2X3 receptor antagonist Refractory or unexplained chronic cough Gefapixant at the 45-mg dose reduced coughs per day by 18.45% relative to placebo at 12 weeks in the Cough-1 study (p=0.041) and by 14.64% relative to placebo at 24 weeks in the Cough-2 study (p=0.031); 15-mg dose did not meet the primary efficacy endpoint in either study
Novartis AG, of Basel, Switzerland Enerzair Breezhaler (indacaterol acetate, glycopyrronium bromide and mometasone furoate) Long acting beta2 agonist/long-acting muscarinic antagonist/inhaled corticosteroid  Asthma Post-hoc analysis of the Iridium study showed the high dose of Enerzair Breezhaler reduced both moderate or severe and severe asthma exacerbation rates in patients whose asthma is uncontrolled on medium- or high-dose long-acting beta2-agonist compared to the medium dose of Enerzair Breezhaler
Radius Health Inc., of Waltham, Mass. Abaloparatide patch Parathyroid hormone related peptide analogue Osteoporosis  Completed enrollment in the Wearable study comparing bone mineral density in patients using the abaloparatide patch compared to subcutaneous Tymlos (abaloparatide)
Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Dupixent (dupilumab) Monoclonal antibody that inhibits the signaling of interleukin-4 and interleukin-13  Moderate to severe asthma In the open-label extension, forced expiratory volume over 1 second improved by 13%-22% at 96 weeks compared to baseline for initial studies; average severe asthma attacks showed 0.31-0.35 events per year, compared to 2.09-2.17 events per year in the year prior to starting the studies
Savara Inc., of Austin, Texas Molgradex (molgramostim nebulizer solution) Granulocyte-macrophage colony-stimulating factor Autoimmune pulmonary alveolar proteinosis Data from the Impala study published in The New England Journal of Medicine showed the treatment didn't meet the primary endpoint of A-aDO2; post-hoc analysis with replacement (by imputation) of invalid A-aDO2 data for 4 severely affected patients who required continuous nasal oxygen therapy resulted in a change in A-aDO2 from baseline to week 24 that was greater in patients receiving continuous administration of Molgradex compared to placebo
Spero Therapeutics Inc., of Cambridge, Mass. Tebipenem HBr Carbapenem antibiotic of the beta-lactam class Complicated urinary tract infection and acute pyelonephritis In the Adapt-Po study, tebipenem HBr was noninferior to ertapenem with overall response rates of 58.8% and 61.6%, respectively
Strongbridge Biopharma plc, of Dublin Recorlev (levoketoconazole) Cortisol synthesis inhibitor Endogenous Cushing’s syndrome In the Logics study, 95.5% of patients withdrawn to placebo had a loss of mean urinary free cortisol response compared to 40.9% of patients who remained on Recorlev (p=0.0002)
Valneva SE, of Saint-Herblain, France VLA-1553 Live-attenuated chikungunya vaccine Chikungunya prophylaxis Started 4,000-participant study measuring immunogenicity 28 days after the single-shot vaccination
Phase IV
Bayer AG, of Leverkusen, Germany Adempas (riociguat) Stimulator of soluble guanylate cyclase Intermediate-risk adult patients with pulmonary arterial hypertension In the Replace study, 41% of patients taking Adempas had a clinical improvement in the absence of clinical worsening, compared with 20% of patients who remained on a phosphodiesterase-5 inhibitor (p=0.0007)


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