Company Product Description Indication Status
Phase I
Glycostem Therapeutics BV, of Oss, the Netherlands Onkord Off-the-shelf allogeneic natural killer cellular immunotherapy Acute myeloid leukemia First patient has been dosed in the pivotal phase I/Iia trial with 33 patients at 8 clinical sites in 5 European countries
Kadimastem Ltd., of Ness Ziona, Israel Astrorx Cell therapy Amyotrophic lateral sclerosis All 5 patients in cohort B were treated and completed the 6-month follow-up period, exhibiting no treatment-related serious adverse events or dose-limiting toxicities; it also showed a statistically significant decline of 45% in the disease progression rate
Liminal Biosciences Inc., of Laval, Quebec, and Cambridge, U.K. Fezagepras Anti-inflammatory and antifibrotic small molecule; modulator of FFAR1 and PPAR alpha Idiopathic pulmonary fibrosis First subject has been dosed in multiple ascending-dose clinical trial
Nektar Therapeutics, of San Francisco NKTR-255 IL-15 pathway agonist Relapsed or refractory head and neck squamous cell carcinoma or colorectal cancer First patient has been dosed in the phase I/II trial evaluating NKTR-255 plus cetuximab in up to 80 patients
Plus Therapeutics Inc., of Austin, Texas Rhenium Nanoliposome A nanoliposome-encapsulated radionuclide Recurrent glioblastoma Completed the sixth dosing cohort in the Respect phase I trial; 18 patients have now been treated
Provention Bio Inc., of Red Bank, N.J. PRV-101 Polyvalent inactivated vaccine Coxsackievirus B Initiated the Provent study in healthy volunteers to test the vaccine in preventing the infection presumed to trigger type 1 diabetes and celiac disease
TFF Pharmaceuticals Inc., of Austin, Texas Voriconazole Inhalation Powder Next-generation, direct-to-lung, inhaled dry powder version of voriconazole Invasive pulmonary aspergillosis Dosed the first subjects in the phase Ib trial
Phase II
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia NP-120 (ifenprodil) An N-methyl-D-aspartate receptor antagonist COVID-19 Interim phase IIb data show a trend toward fewer patients requiring mechanical ventilation in the high-dose treatment arm compared with those in the untreated arm at day 15; the final dataset will be available in the first quarter of 2021 
Anavex Life Sciences Corp., of New York Anavex 2-73 (blarcamesine) Activates the sigma-1 receptor Rett syndrome Top-line data show it was well-tolerated and demonstrated dose-proportional pharmacokinetics and adverse events that were similar with placebo; all secondary efficacy endpoints showed statistically significant and clinically meaningful sustained improvements, including the Rett Syndrome Behavior Questionnaire (p=0.048) and the Clinical Global Impression Improvement Scale score (p=0.014) in the intent-to-treat population
Arca Biopharma Inc., of Westminster, Colo. Rnapc2 (AB-201) Small recombinant protein; a selective inhibitor of tissue factor COVID-19 First patient was enrolled in a phase IIb trial at the University of Colorado Hospital, with a total of 5 patients randomized to date; top-line data expected in the second quarter of 2021
Diamedica Therapeutics Inc., of Minneapolis DM-199 Insulin sensitizer; kallikrein 1 modulator End-stage renal disease; IgA nephropathy Last participant began dosing in diabetic kidney disease cohort of Redux study; enrollment reached 50% in IgA nephropathy and African Americans cohorts
Emalex Biosciences Inc., of Chicago Ecopipam Dopamine D1 receptor antagonist Stutter First adult participant dosed in Speak Freely study
FSD Pharma Inc., of Toronto FSD-201 (palmidrol + morphine, ultramicronized) Cannabinoid CB2 receptor modulator + opioid receptor mu agonist COVID-19 infection First of 352 participants dosed in phase IIa trial in people hospitalized with newly documented infection; primary outcome measure is improvement in clinical status in combination with standard of care
Harmony Biosciences Holdings Inc., of Plymouth Meeting, Pa. Wakix (pitolisant) Histamine H3 receptor inverse agonist Excessive daytime sleepiness First of up to 70 participants ages 6 to 65 with Prader-Willi syndrome enrolled; primary outcome measure is improvement in EDS measured by Multiple Sleep Latency Test, with top-line data expected in first half of 2022
Hightide Therapeutics Inc., of Shenzhen, China HTD-1801 Anti-inflammatory agent Nonalcoholic steatohepatitis Study in adults with co-morbid type 2 diabetes met primary endpoint, with those who received 1,000 mg twice daily (n=34) showing mean absolute liver fat content decrease from baseline of 4.83% (p=0.011 vs. placebo) and mean relative liver fat reduction of 24.14% (p=0.016 vs. placebo), measured by MRI-PDFF; treatment for 18 weeks also resulted in reduction in mean HbA1c level from 7.4 at baseline to 6.8 (p=0.004)
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda Vixarelimab Oncostatin M receptor subunit beta inhibitor Prurigo nodularis Dosing initiated in phase IIb trial expected to enroll about 180 people with moderate to severe disease to assess 3 once-monthly subcutaneous dose regimens vs. placebo; primary efficacy endpoint is percent change from baseline in weekly average Worst-Itch Numeric Rating Scale at week 16
Minoryx Therapeutics SL, of Barcelona, Spain Leriglitazone PPAR gamma agonist Friedreich ataxia Top-line results from Frames study showed changes in spinal cord area, the primary outcome, were inconclusive since no progression was seen in those on placebo; study drug prevented iron accumulation in the brain vs. placebo (ANCOVA p=0.050); Composite Cerebellar Function Severity scale showed decline in upper limb ataxia prevented vs. placebo (O’Brien test, Wilcoxon p=0.043)
Nicox SA, of Sophia Antipolis, France NCX-4251 (fluticasone) Glucocorticoid receptor agonist Blepharitis Initiated phase IIb Mississippi trial to evaluate once-daily dosing at 0.1% vs. placebo in 200 people with acute exacerbations; primary outcome measure is proportion who achieve complete cure in eyelid redness, debris and discomfort at day 15; top-line results expected in fourth quarter of 2021
Palatin Technologies Inc., of Cranbury, N.J.  PL-9643 Dual melanocortin MC1/MC5 receptor agonist Dry eye disease In subpopulation with moderate to severe disease (N=61), study achieved statistical significance (p<0.05 vs. vehicle) at weeks 2 and 12 for primary sign endpoint of inferior corneal staining and for superior and total corneal staining; temporal, nasal and total conjunctival staining; tear film break-up time and multiple ocular symptoms, including discomfort; additional sign and symptom measures trended toward significance (p<0.1 vs. vehicle); U.S. phase II/III trial expected to begin in mid-2021
Verastem Inc., of Boston VS-6766  RAF/MEK inhibitor Non-small-cell lung cancer Registration-directed adaptive Ramp 202 trial initiated as monotherapy and in combination with FAK inhibitor defactinib in people with KRAS-mutant disease; first part will determine optimal monotherapy or combination regimen in KRAS-G12V mutant disease, with exploratory arm to evaluate other KRAS mutations
Phase III
Erytech Pharma SA, of Lyon, France Eryaspase (L-asparaginase, erythrocyte-encapsulated) Asparaginase stimulator Pancreatic cancer Pivotal Trybeca-1 trial in second-line disease fully enrolled 510 participants, surpassing target enrollment of 482; results from interim superiority analysis expected in first quarter of 2021, with trial either continuing to final analysis in fourth quarter of 2021 or concluding early if sufficient improvement seen in overall survival
Mesoblast Ltd., of Melbourne, Australia Revascor (rexlemestrocel-L) Allogeneic cell therapy Chronic heart failure Dream-HF trial in 537 people with advanced disease missed primary endpoint, showing no reduction in recurrent non-fatal decompensated heart failure events; trial showed 60% reduction in incidence of nonfatal ischemic major adverse cardiac events due to myocardial infarction or stroke vs. controls (p=0.002) and 60% reduction in death from all cardiac causes in 206 heart failure patients with NYHA class II disease vs. controls (p=0.037)
Novan Inc., of Morrisville, N.C. SB-206 (berdazimer sodium) Antiviral Molluscum contagiosum Pivotal B-Simple4 trial 60% enrolled, with full enrollment of 850 participants expected in first quarter of 2021 and top-line results targeted for second quarter of 2021; primary endpoint is proportion with clearance of treatable lesions at week 12
Novartis AG, of Basel, Switzerland Beovu (brolucizumab) VEGF-A ligand inhibitor Diabetic macular edema Pivotal Kestrel study of Beovu 6 mg achieved primary endpoint of noninferiority to aflibercept 2 mg in change in BCVA at week 52 and key secondary endpoint of noninferiority in average change in BCVA to aflibercept over week 40 through week 52
Zealand Pharma A/S, of Copenhagen Dasiglucagon Glucagon analogue Congenital hyperinsulinism When added to standard of care in 32 children, study drug missed primary endpoint, failing to reduce rate of hypoglycemia vs. SOC alone assessed by intermittent self-measured plasma glucose; in exploratory analysis, hypoglycemia was reduced by 40% to 50% vs. SOC alone when assessed by blinded continuous glucose monitoring


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