Company Product Description Indication Status
Phase I
Arena Pharmaceuticals Inc., of San Diego Etrasimod (controlled release) Sphingosine 1-phosphate receptor modulator Ulcerative colitis Top-line data showed >75% reduction in average heart rate effect of controlled release (CR) formulation during 4-hour monitoring period, with heart rate slowing by low single digits from baseline with no titration; at additional measurements over 24 hours, CR heart rate effect was reduced or similar vs. standard etrasimod formulation
Atara Biotherapeutics Inc., of South San Francisco ATA-188 Autologous cytotoxic T-lymphocyte therapy Multiple sclerosis Monitoring of enrolled participants and retreatment in open-label extension of phase Ia continue; screening and enrollment of phase Ib participants paused due to COVID-19 pandemic
Axial Biotherapeutics Inc., of Waltham, Mass. AB-2004 Microbiome modulator Autism spectrum disorder Phase Ib/IIa trial met primary endpoint of safety, tolerability and adherence with no drug-related adverse events; reductions in plasma and urinary levels of several key microbial metabolites observed over 8-week dosing period; improvements also seen in irritability, anxiety and social withdrawal scores, particularly in those with more severe baseline scores
Helix Biopharma Corp., of Richmond Hill, Ontario L-DOS47 Immunoconjugate drug Pancreatic cancer Phase Ib/II study (LDOS006) testing combination with doxorubicin continues to enroll and has dosed 2 patients; however, given the COVID-19 pandemic, company expects patient enrollment will be impacted
I-Mab Biopharma Co. Ltd., of Shanghai TJC-4 CD47 antagonist Acute myeloid leukemia; myelodysplastic syndrome  First participant dosed in phase I/IIa monotherapy trial in China in relapsed/refractory disease
Moleculin Biotech Inc., of Houston Annamycin Lipophilic anthracycline derivative Acute myeloid leukemia  210 mg/m2 cohort completed dosing in European single-arm phase I/II trial in relapsed/refractory disease; across 19 people dosed in U.S. and Europe, including 10 treated at or above FDA lifetime maximum anthracycline exposure, study drug was safe and free of cardiotoxicity
Momenta Pharmaceuticals Inc., of Cambridge, Mass. M-254 (hypersialylated immunoglobulin) Dendritic ICAM-3 grabbing nonintegrin 1 modulator Idiopathic thrombocytopenic purpura  Most sites suspended enrollment in ongoing phase I/II trial due to COVID-19 pandemic; interim data for part B may not report as planned in second quarter of 2020, which would delay initiation of parts C and D
Noxxon Pharma NV, of Berlin NOX-A12 (olaptesed pegol) CXCL12 inhibitor Brain cancer First dose cohort (n=3) in phase I/II study enrolled and received initial therapy of study drug plus external beam radiotherapy; recruitment of participants for higher dose cohorts continues
Oncolytics Biotech Inc., of San Diego Pelareorep Oncolytic retrovirus Colorectal cancer Findings in Molecular Cancer Therapeutics showed that, in dose-escalation study in 36 people with KRAS-mutated tumors, combination of FOLFIRI, bevacizumab and pelareorep was well-tolerated; of 6 who received recommended phase II dose, 50% overall response rate and median overall survival of 25.1 months were observed; among 30 evaluable patients, 6 had partial response and 22 patients had stable disease
Zymeworks Inc., of Vancouver, British Columbia ZW-25 ERBB2 tyrosine kinase receptor inhibitor Breast cancer; gastroesophageal adenocarcinoma Partner Beigene Ltd. dosed first participant in 2-arm phase Ib/II trial evaluating study drug in combination with chemotherapy in first-line metastatic HER2-positive breast cancer and in combination with chemotherapy and PD-1-targeted antibody tislelizumab (Beigene) in first-line metastatic HER2-positive GEA
Phase II
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia Ifenprodil NMDA receptor epsilon 2 subunit inhibitor COVID-19 infection Protocol finalized for physician-initiated study in South Korea; submission for approval expected shortly
Ansun Biopharma Inc., of San Diego DAS-181 Recombinant sialidase Severe COVID-19 pneumonia Initiated randomized, double-blind, placebo-controlled proof-of-concept trial; 2-stage trial will initially enroll about 20 patients 
Apeiron Biologics AG, of Vienna APN-01 Angiotensin converting enzyme 2 stimulator COVID-19 infection Regulatory approvals received in Austria, Germany and Denmark for trial expected to enroll 200 participants with severe infection, with initial dosing expected shortly; outcome measures include need for mechanical ventilation and evaluation of measurable biological biomarker changes
Athera Biotechnologies AB, of Stockholm ATH-3G10 (PC-Mab) CCT alpha inhibitor Myocardial infarction Trial completed enrollment of 82 individuals with STEMI and last participant dosed; top-line data expected by year-end 2020
Biogen Inc., of Cambridge, Mass. Spinraza (nusinersen) Antisense oligonucleotide Spinal muscular atrophy  First participant dosed in higher-dose phase II/III Devote study; pivotal cohort in 3-part study will receive 2 loading doses of 50 mg 14 days apart followed by maintenance dose of 28 mg every 4 months to assess safety and tolerability vs. currently approved dose
Fulcrum Therapeutics Inc., of Cambridge, Mass. Losmapimod p38 alpha/beta MAP kinase inhibitor Facioscapulohumeral muscular dystrophy Phase IIb Redux4 trial is fully enrolled but trial activity suspended at many sites due to COVID-19 pandemic; top-line data for primary endpoint of change from baseline in DUX4 activity may not be available by end of third quarter of 2020, as planned
Mateon Therapeutics Inc., of Agoura Hills, Calif. OT-101 Antisense targeting TGF-beta Recalcitrant/resistant anaplastic astrocytoma Data published in Cancer Reports and Reviews showed the drug produced a median overall survival of 1,136 days compared to 590 days for TMZ-treated patients (p=0.011)
Momenta Pharmaceuticals Inc., of Cambridge, Mass. Nipocalimab (M-281) IgG receptor FcRn large subunit p51 antagonist Hemolytic disease of fetus and newborn; warm autoimmune hemolytic anemia Although not officially paused, Unity study in HDFN expected to enroll at slower pace due to impact of COVID-19 pandemic; global site activation continues in adaptive phase II/III Energy study in wAIHA but enrollment temporarily suspended
Momenta Pharmaceuticals Inc., of Cambridge, Mass. M-254 (hypersialylated immunoglobulin) Dendritic ICAM-3 grabbing nonintegrin 1 modulator Chronic inflammatory demyelinating polyneuropathy Trial now expected to begin in 2021 due to COVID-19-related delays
Rhovac AB, of Stockholm RV-001 Antigen-based cancer therapy Prostate cancer Recruitment being delayed due to COVID-19; company expects existing financing will cover the expected delay
Valbiotis SA, of La Rochelle, France Totum-63 Plant-derived substance Type 2 diabetes Plans to launch the 600-patient Reverse-It study in the first half of 2020; primary endpoint is reduction in fasting blood glucose for 3 doses for a total of 5 g per day compared to placebo; study also contains an open-label arm testing 2 doses for a total of 5 g per day
Phase III
Dermavant Sciences Inc., of Long Beach Calif., a subsidiary of Basel, Switzerland-based Roivant Sciences GmbH Tapinarof Therapeutic aryl hydrocarbon receptor modulating agent Plaque psoriasis Completed enrollment in the Psoaring 1 and 2 studies; data expected in the second half of 2020
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. Lonafarnib   Farnesyl transferase inhibitor Hepatitis delta virus infection Completion of D-LIVR study enrollment shifted into 2021 due to COVID-19
Geron Corp., of Menlo Park, Calif. Imetelstat  Telomerase inhibitor Transfusion-dependent patients with low or intermediate-1 risk myelodysplastic syndrome Completion of IMerge study enrollment isn't expected by year-end 2020 as previously disclosed; company also doesn't plan to start proof-of-concept study in high-risk MDS and acute myeloid leukemia by the end of 2020
Merck & Co. Inc., of Kenilworth, N.J. Keytruda (pembrolizumab) Monoclonal antibody targeting PD-1 Microsatellite instability-high or mismatch repair deficient unresectable or metastatic colorectal cancer Interim analysis of Keynote-177 study found it met 1 of the dual primary endpoints of progression-free survival; study will continue to evaluate other primary endpoint of overall survival
VBL Therapeutics Ltd., of Tel Aviv, Israel, and Nanocarrier Co. Ltd., of Chiba, Japan VB-111 (ofranergene obadenovec) Dual CD95/TNF receptor modulator Platinum-resistant ovarian cancer Nanocarrier plans to extend the ongoing Oval study to Japan
Xbrane Biopharma AB, of Solna, Sweden Xlucane   Biosimilar of Lucentis (ranibizumab) Wet age-related macular degeneration Xplore study remains open with 355 of 580 patients enrolled, but COVID-19 makes forecasting future recruitment rate challenging

Notes

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