Company Product Description Indication Status
Phase I
9 Meters Biopharma Inc., of Raleigh, N.C. NM-002 GLP-1 agonist Short bowel syndrome First participant dosed in phase Ib/IIa trial in adults; change in daily urine output volume designated as primary endpoint as inversely related proxy for use of parenteral support
Akero Therapeutics Inc., of South San Francisco Efruxifermin (AKR-001) FGF21 analogue Metabolic diseases Data published in Cell Reports Medicine showed drug had sustained pharmacodynamic effects on insulin sensitivity and lipid metabolism in type 2 diabetes patients; demonstrated sufficiently prolonged half-life of 3-3.5 days to support weekly dosing; with variation in systemic concentration of only 2-fold when dosed weekly, treatment delivered larger and more consistent improvements in markers of insulin sensitivity and lipid metabolism than previously reported for FGF21 analogues
Araim Pharmaceuticals Inc., of Tarrytown, N.Y. Cibinetide Erythropoietin receptor agonist Diabetic macular edema In exploratory trial that enrolled 9 participants, study drug showed no significant effect on mean BCVA values but NEI-VFQ-25 composite scores improved and some individuals showed improvements in BCVA, retinal sensitivity, central retinal thickness, tear production and albuminuria as well as improved metabolic control
Cerecor Inc., of Rockville, Md. CERC-002  Tumor necrosis factor 14 ligand inhibitor COVID-19-induced acute respiratory distress syndrome First of about 82 hospitalized individuals enrolled in proof-of-concept study evaluating effect of single dose of study drug; primary outcome measures are respiratory failure and death vs. standard of care at 28 days; key secondary endpoints include ICU and hospital lengths of stay and oxygen saturation at end of study; top-line data expected in fourth quarter of 2020
Dragonfly Therapeutics Inc., of Waltham, Mass. DF-6002 Human IL-12/Fc fusion protein  Advanced solid tumors First participant enrolled and dosed in phase I/II trial 
Larimar Therapeutics Inc., of Bala Cynwyd, Pa. CTI-1601 Mitochondrial frataxin stimulator Friedreich’s ataxia Participants dosed in third cohort of trial evaluating single ascending doses, following pause due to impact of COVID-19 pandemic; top-line data expected in first half of 2021
Staten Biotechnology BV, of Nijmegen, the Netherlands STT-5058 Apolipoprotein C3 antagonist Hypertriglyceridemia Dosing initiated in first-in-human study enrolling healthy volunteers
Xylocor Therapeutics Inc., of Malvern, Pa. XC-001 VEGF gene stimulator Angina First 2 participants dosed in phase I/II Exact trial enrolling 12 individuals unresponsive to medication and unsuitable for coronary artery bypass graft or percutaneous coronary intervention who will receive 1 of 4 ascending intramyocardial doses, followed by expansion cohort of 17 participants at highest tolerated dose
Phase II
Anheart Therapeutics Co. Ltd., of Shanghai Taletrectinib ROS1/NTRK inhibitor  Non-small-cell lung cancer First of about 106 individuals with ROS1 fusion disease dosed in open-label, single-arm study in China; top-line data expected as early as fourth quarter of 2021
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist COVID-19 infection Safety data from 84 participants treated across 8 sites showed 34% (19 of 56) in leronlimab arm reported at least 1 serious adverse event (SAE) vs. 50% (14 of 28) in placebo arm; no SAEs in leronlimab arm deemed related to study drug
Phase III
Abbvie Inc., of North Chicago Rinvoq (upadacitinib) Oral, once-daily, selective, reversible JAK inhibitor Moderate to severe atopic dermatitis Monotherapy met both primary and all secondary endpoints in Measure Up 2 study; at week 16, 60% and 73% of patients receiving 15 mg and 30 mg, respectively, achieved at least 75% improvement in Eczema Area Severity Index vs. 13% in placebo group (p<0.001); of patients treated with 15 mg or 30 mg, 39% and 52%, respectively, achieved validated Investigator's Global Assessment for Atopic Dermatitis score of 0/1 vs. 5% on placebo (p<0.001); 42% and 60%, respectively experienced clinically meaningful reductions in itch vs. 9% on placebo (p<0.001)
Acadia Pharmaceuticals Inc., of San Diego Pimavanserin Selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors Major depressive disorder Top-line results showed Clarity study did not achieve statistical significance on primary endpoint, 17-item Hamilton Depression Rating Scale (HAMD-17) total score change from baseline to week 5; pimavanserin 34 mg, given once-daily as adjunctive treatment to standard antidepressant therapy was associated with mean reduction of 9 in HAMD-17 total score vs. 8.1 for placebo as adjunctive treatment (p=0.296)
Aimmune Therapeutics Inc., of Brisbane, Calif. Palforzia  Peanut (Arachis hypogaea) allergen powder-dnfp Peanut allergy Complete results from Artemis trial published in The Lancet Child & Adolescent Health showed study met all primary, secondary and safety endpoints, and demonstrated that treated participants experienced high degree of desensitization to peanut with manageable safety profile over 9 months; improvements in quality-of-life measures in treated participants were reported, most notably in how treatment affected perceived likelihood of future accidental exposure and risk of severe reactions
Avenue Therapeutics Inc., of New York I.V. tramadol Analgesic Bunionectomy Data published in Pain and Therapy showed 50-mg dose demonstrated statistically significant benefit (p<0.05) over placebo for primary and all key secondary efficacy endpoints, whereas 25 mg demonstrated intermediate results between the 50-mg and placebo arms
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Verdiperstat Myeloperoxidase inhibitor Multiple system atrophy Completed enrollment in M-Star study; top-line data expected by end of 2021
Blue Earth Diagnostics Inc., of Burlington, Mass. rhPSMA-7.3 PSMA-targeted radiohybrid PET imaging agent Prostate cancer detection Dosed first patients in Spotlight study evaluating safety and diagnostic performance in men with suspected prostate cancer recurrence based on elevated PSA following prior therapy
Ironwood Pharmaceuticals Inc., of Boston IW-3718 Gastric-retentive formulation of bile-acid sequestrant colesevelam Refractory gastroesophageal reflux disease Reached alignment with FDA on updates to phase III program, including changing primary endpoint from previous responder endpoint to a continuous endpoint; company plans to stop enrollment of study subjects and conduct blinded early assessment of efficacy, with outcome expected in fourth quarter of 2020
Lexicon Pharmaceuticals Inc., of The Woodlands, Texas Sotagliflozin SGLT1/2 inhibitor Type 2 diabetes Top-line data showed all 4 studies – Sota-Mono, Sota-SU, Sota-Glim and Sota-INS – met primary objectives of lowering A1C
Rafael Pharmaceuticals Inc., of Cranbury, N.J. CPI-613 (devimistat) Targets the mitochondrial tricarboxylic acid cycle Relapsed or refractory acute myeloid leukemia Crossed midpoint to first interim analysis in enrollment of Armada 2000 study testing combination of drug with high-dose cytarabine and mitoxantrone in older patients
Phase IV
Biogen Inc., of Cambridge, Mass. Spinraza (nusinersen) Antisense oligonucleotide Spinal muscular atrophy Plans to start Respond study to examine benefit and assess safety in infants and children with SMA who still have unmet clinical needs following treatment with gene therapy Zolgensma (onasemnogene abeparvovec, Novartis AG)


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