Company Product Description Indication Status
Phase I
ADC Therapeutics SA, of Lausanne, Switzerland ADCT-402 (loncastuximab tesirine) Antibody drug conjugate targeting CD19 Diffuse large B-cell, mantle cell or follicular lymphoma Terminated study testing combination with Imfinzi (durvalumab, Astrazeneca plc); in limited number of patients, no additional activity evident for combination vs. loncastuximab tesirine monotherapy
Bexion Pharmaceuticals Inc., of Covington, Ky. BXQ-350 Saposin C and phosphatidylserine Relapsed solid tumors Study in children and young adults terminated; company will not move forward with part 2 expansion portion in order to focus resources on further development of drug as earlier treatment measure in pediatric population
Biolinerx Ltd., of Tel Aviv, Israel Motixafortide CXCR4 inhibitor Acute respiratory distress syndrome secondary to COVID-19 and other respiratory viral infections Started an investigator-initiated phase Ib trial in up to 25 patients; primary endpoint is safety; respiratory parameters and inflammatory biomarkers will be assessed as exploratory endpoints; data from an interim analysis after 10 patients have completed initial treatment expected in the first half of 2021
Bioversys AG, of Basel, Switzerland BV-100  Antibiotic Healthy subjects (eventually serious infections caused by carbapenem-resistant Acinetobacter baumannii) Dosed first volunteer in the singe and multiple ascending-dose study testing the safety, tolerability and pharmacokinetics of BV-100
Cogent Biosciences Inc., of Cambridge, Mass. PLX-9486  Tyrosine kinase inhibitor of KIT exon 17 mutations Advanced gastrointestinal stromal tumors Confirmed objective response rate of 20% in 15 patients treated with PLX-9486 plus Sutent (sunitinib, Pfizer Inc.) in phase I/II trial; 27% of patients remained on therapy out to 27 months to 34 months
Enlivex Therapeutics Ltd., Nes Ziona, Israel Allocetra Autologous cell-based therapy Erosive osteoarthritis 1 patient treated with Allocetra in phase Ib trial had fluid drainage from the shoulder decline from 150–250 ml/day to <60 ml/day; C-reactive protein declined by 93%, from 7.34 pre-treatment, to 0.49 after treatment; at a 24-month follow-up, CRP level in the diseased shoulder remained within normal range
Genoscience Pharma SA, of Marseilles, France GNS-561 Solute carrier transporter inhibitor  Advanced or metastatic hepatocarcinoma or intrahepatic cholangiocarcinoma Disease stabilization observed in 2 of 7 evaluable hepatocellular carcinoma patients and 1 intrahepatic cholangiocarcinoma patient
Moderna Inc., of Cambridge, Mass. mRNA-3704 mRNA encoding human methylmalonyl-CoA mutase  Isolated methylmalonic acidemia Phase I/I study terminated before start of dosing due to business decision, not due to safety or efficacy reasons
Organicell Regenerative Medicine Inc., of Miami Zofin Perinatal-derived acellular biologic Moderate to severe acute respiratory syndrome related to COVID-19 Adding additional trial site for phase I/II trial; enrolled "several" patients to date
Pionyr Immunotherapeutics Inc., of South San Francisco PY-314 Targets TREM2 Solid tumors Starting study
Puretech Health plc, of Boston LYT-100 (deupirfenidone) Deuterated form of anti-inflammatory and antifibrotic pirfenidone Healthy subjects (eventually idiopathic pulmonary fibrosis) Drug had a favorable tolerability and pharmacokinetic profile in the multiple ascending-dose and food effect study; area under the cure was similar for treatment under fasted and fed conditions
Sernova Corp., of London, Ontario Cell Pouch System Insulin-producing islets Type 1 diabetes Patients had reduction in injectable insulin use, HbA1c levels and hypoglycemic episodes; C-peptide detected in bloodstream
VBI Vaccines Inc., of Cambridge, Mass. VBI-2601 (BRII-179) Recombinant, protein-based virus-like particle vaccine Chronic hepatitis B virus infections In the low-dose cohort of phase Ib/IIa study, 67% of 9 patients and 78% of 9 patients treated with VBI-2601 unadjuvanted and adjuvanted, respectively, had re-stimulation of T-cell responses to HBV surface antigens; hepatitis B surface antigen antibodies increased in 60% of 10 and 67% of 9 evaluable patients treated with VBI-2601 unadjuvanted and adjuvanted, respectively; data from the high-dose cohorts expected in the first quarter of 2021
Phase II
Afimmune Ltd., of Dublin DS-102 Synthetic prodrug of endogenous metabolite of eicosapentaenoic acid Acute alcoholic hepatitis Trial suspended per company decision
Boehringer Ingelheim GmbH, of Ingelheim, Germany BI-754091 PD-1 inhibitor Squamous cell carcinoma of the anal canal  Study testing combination with BI-836880 in patients with unresectable or metastatic disease withdrawn; sponsor decision, not due to safety reasons
Gilead Sciences Inc., of Foster City, Calif. Lenacapavir HIV-1 capsid inhibitor Treatment-experienced people with multidrug-resistant HIV-1 infection In the phase II/III Capella study, 88% of the 24 patients treated with lenacapavir experienced at least a 0.5 log10 reduction in HIV-1 viral load by the end of 14 days of functional monotherapy compared with 17% of the 12 patients who received placebo
Greenwich Lifesciences Inc., of Stafford, Texas GP-2 9 amino acid transmembrane peptide of the HER2/neu protein Breast cancer Disease-free survival rate at 5 years was 100% for GP-2 plus GM-CSF compared to 89.4% for patients taking GM-CSF alone (p=0.0338)
Hepion Pharmaceuticals Inc., of Edison, N.J. CRV-431  Hepatitis B structural protein inhibitor; peptidyl-prolyl cis-trans isomerase A inhibitor Nonalcoholic steatohepatitis After review of the safety and tolerability data for the 75-mg dose, the independent data safety monitoring board for the phase IIa Abition study recommended enrolling the 225-mg dose cohort; reductions in ALT and AST were observed at day 28
Kazia Therapeutics Ltd., of Sydney Paxalisib  PI3K pathway inhibitor Glioblastoma  Median progression-free survival was 8.4 months; overall survival was 17.5 months
Neurosense Therapeutics, of Herzliya, Israel PrimeC Antibiotic ciprofloxacin and nonsteroidal anti-inflammatory drug celecoxib  Amyotrophic lateral sclerosis  Preliminary trends seen at the interim analysis continue to show promise at 9 months; study set to complete in January 2021 after 12 months of dosing
Oncosec Medical Inc., of Pennington, N.J. Tavo (tavokinogene telseplasmid) DNA plasmid-based interleukin-12 Anti-PD-1 checkpoint-resistant metastatic melanoma In the phase IIb Keynote-695 study, Tavo plus Keytruda (pembrolizumab, Merck & Co. Inc.) produced a 30% overall response rate; response rate was 35% in M1c and M1d patients, who have organ or brain metastatic disease; response rate was 40% in patients who had disease progression after both anti-PD-1 and anti-CTLA4
Phase III
Aztherapies Inc., of Boston ALZT-OP1 Amyloid protein deposition inhibitor Alzheimer’s disease Completed last patient/last visit in Cognite trial, testing drug in patients with early stage disease; top-line data expected in first quarter of 2021
Chiasma Inc., of Needham, Mass. Mycapssa (oral octreotide capsules) Somatostatin receptor agonist Acromegaly Top-line data showed Mpowered noninferiority study met primary endpoint, with 91% of patients on Mycapssa maintaining IGF-1 response vs. 100% on injectable somatostatin analogues; Mycapssa also maintained mean IGF-1 within normal limits and was comparable to injectable therapy
Eli Lilly and Co., of Indianapolis, and Boehringer Ingelheim GmbH, of Ingelheim, Germany Jardiance (empagliflozin) SGLT2 inhibitor Type 2 diabetes and cardiovascular disease Post-hoc analysis of Empa-Reg Outcome trial showed reduced risk of total (first plus recurrent) cardiovascular events compared with placebo, when both were given on top of standard of care, in adults over 3 years of study; findings also published in The Lancet Diabetes & Endocrinology
Helixmith Co. Ltd., of Seoul, South Korea Engensis (VM-202) Gene therapy Diabetic peripheral neuropathy Enrolled first patient in Regain-1A trial in U.S.; primary endpoint compares change in average daily pain scores from the 7 days prior to first injection on day 0 to the 7 days prior to the 6-month visit vs. placebo
Milestone Pharmaceuticals Inc., of Montreal Etripamil nasal spray Short-acting calcium channel blocker Paroxysmal supraventricular tachycardia First patient enrolled in Rapid pivotal trial; data expected in late 2021/early 2022
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany BNT-162b2 mRNA-based vaccine COVID-19 Final analysis showed vaccine efficacy rate of 95% (p<0.0001) in participants without prior SARS-CoV-2 infection (first primary objective) and in participants with and without prior SARS-CoV-2 infection (second primary objective), in each case measured from 7 days after second dose; efficacy consistent across age, gender, race and ethnicity demographics; observed efficacy in adults over 65 was more than 94%
Sanofi SA, of Paris Rilzabrutinib Oral BTK inhibitor Immune thrombocytopenia  Initiated study 

Notes

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