Company Product Description Indication Status
Phase I
Adverum Biotechnologies Inc., of Redwood City, Calif. ADVM-022 Gene therapy expressing aflibercept Wet age-related macular degeneration  Interim data from the Optic study showed 7 of 9 patients in cohort 3 and 8 of 9 patients in cohort 4 were free of supplemental injections; patients in cohort 3 without supplement treatment had a mean improvement in best-corrected visual acuity (BCVA) of 4.1 letters and a mean change in central retinal thickness (CRT) of -132.7 μm; BCVA and CRT not measured in cohort 4 yet
Alligator Bioscience AB, of Lund, Sweden, and Aptevo Therapeutics Inc., of Seattle ALG.APV-527 Targets 5T4 and 4-1BB (CD137) Solid tumors expressing 5T4 Moving drug into clinical development; plan to file a CTA during the first half of 2021
Atara Biotherapeutics Inc., of South San Francisco ATA-188 Targets EBV-infected B cells and plasma cells Progressive forms of multiple sclerosis Of the 16 patients who entered the phase Ia open-label extension (OLE), 9 had sustained disability improvement (7 in the first 12 months and 2 during the OLE); in 7 out of the 9 patients, the disability improvement was driven by improvement in Expanded Disability Status Score; expanding enrollment to at least 64 patients and changing the primary endpoint to disability improvement
Biological E. Ltd., of Hyderabad, India, and Dynavax Technologies Corp., of Emeryville, Calif. COVID-19 subunit vaccine and CpG 1018 Receptor binding domain of the spike protein of SARS-CoV-2 and adjuvant COVID-19 prophylaxis Started the phase I/II study of 360 participants in India; results expected by February 2021
Calypso Biotech BV, of Amsterdam CALY-002 Humanized monoclonal antibody neutralizing IL-15 Healthy volunteers and patients with celiac disease and eosinophilic esophagitis Completed dosing of the first cohort of healthy subjects
Cerevance Inc., of Boston CVN-058 Antagonist of the type 3 receptor for serotonin Cognitive impairment associated with schizophrenia Drug produced a statistically significant improvement in mismatch negativity compared to placebo in phase Ib trial
Genenta Science Srl, of Milan, Italy Temferon  Hematopoietic stem/progenitor cells expressing Tie2 Glioblastoma multiforme Phase I/IIa trial showed T-cell immunorepertoire changes observed after treatment; evidence of clonal expansion, including tumor associated clones
Intravacc, of Bilthoven, the Netherlands SF2a-TT15 Shigella vaccine Shigella infection prophylaxis Data published in The Lancet Infectious Diseases from the 64-participant study showed the non-adjuvanted high dose induced a 25-fold increase in IgG geometric mean titer after 1 injection while the non-adjuvanted low dose induced a 5-fold increase, compared with baseline; alum adjuvant significantly enhanced the specific IgG response at both doses after the third injection
Precigen Inc., of Germantown, Md. PRGN-3005 UltraCAR-T cells Autologous CAR T-cell treatment targeting MUC16 Advanced ovarian cancer patients Dosed first patients using the Ultraporator system
Precigen Inc., of Germantown, Md. PRGN-3006 UltraCAR-T cells Autologous CAR T-cell treatment targeting CD33 Relapsed or refractory acute myeloid leukemia and higher-risk myelodysplastic syndrome Dosed first patients in phase Ia/Ib trial using the Ultraporator system
Rapt Therapeutics Inc., of South San Francisco FLX-475  CCR4 antagonist Multiple cancer indications In the phase I dose-escalation phase, 14 of 17 evaluable patients treated with FLX-475 had disease control, including an unconfirmed partial response; 13 of the 14 evaluable patients treated with FLX-475plus pembrolizumab had disease control, including 2 confirmed partial responses; in the phase II expansion cohorts, objective response rate was 50% in 2 patients with EBV+ lymphoma treated with the monotherapy; ORR was 60% in 5 nasopharyngeal cancer patients who crossed over from monotherapy to combination therapy; ORR was 33% in 6 patients with head and neck cancer who crossed over; ORR was 10% in 10 patients with head and neck cancer treated directly with the combination
Revelation Biosciences Inc., of Menlo Park, Calif. REVTx‑99 Intranasal formulation of phosphorylated hexa-acylated disaccharide, a synthetic version of monophosphoryl lipid A Healthy volunteers (eventually COVID‑19 patients at risk for hospitalization) Dosed 8 patients in the first cohort of the REVTx‑99; top-line data from the 4-cohort, 32-participant study expected in the first quarter of 2021
Rhovac AB, of Lund, Sweden RV-001 RhoC GTPase modulator  Prostate cancer  Phase I/II data published in the Journal for ImmunoTherapy of Cancer showed 18 of 21 evaluable developed a strong CD4 T-cell response against the vaccine, which lasted at least 10 months after the last vaccination
Phase II
Bioaegis Therapeutics Inc., of Morristown, N.J. rhu-pGSN Recombinant human plasma gelsolin Severe COVID-19 Expanding the clinical trial to additional sites in Europe; study has enrolled more than one-third of the target enrollment; independent data safety monitoring board recommended continuation of recruitment based on safety data
Kodiak Sciences Inc., of Palo Alto, Calif. KSI-301 Anti-VEGF antibody biopolymer conjugate Wet age-related macular degeneration Exceeded the target enrollment of 550 patients in phase IIb/III trial; data expected in early 2022
Obseva SA, of Geneva Ebopiprant  Prostaglandin F2alpha receptor antagonist Preterm labor In the Prolong phase IIb study, 12.5% of women who received ebopiprant delivered within 48 hours of starting treatment compared to 21.8% of patients who received placebo, a reduction of 55%
PTC Therapeutics Inc., of South Plainfield, N.J. PTC-299 Dihydroorotate dehydrogenase inhibitor COVID-19 Started phase II/III study in Australia; actively recruiting in the U.S., Brazil and Spain 
Redhill Biopharma Ltd., of Tel Aviv, Israel Yeliva (opaganib/ABC-294640) Sphingosine kinase-2 inhibitor Severe COVID-19 pneumonia Completed enrollment of 40 patients in the study; top-line data expected in the coming weeks
University of Virginia School of Medicine, of Charlottesville Fluvoxamine  Serotonin reuptake inhibitor COVID-19 None of the 80 patients who received fluvoxamine became seriously ill after 15 days, compared to 6 patients who received placebo
Phase III
Akebia Therapeutics Inc., of Cambridge, Mass. Vadadustat Hypoxia-inducible factor prolyl hydroxylase inhibitor  Anemia Study design and methodology for global Inno2vate program to treat anemia due to chronic kidney disease in adults on dialysis published in Nephrology Dialysis Transplantation, journal of European Renal Association-European Dialysis and Transplant Association
Beyondspring Inc., of New York Plinabulin Immune and stem cell modulator Neutropenia Registrational Protective-2 trial showed plinabulin in combination with pegfilgrastim vs. pegfilgrastim alone met primary endpoint of statistically significant improvement in rate of preventing grade 4 neutropenia in cycle 1 (31.5% vs. 13.6%, p=0.0015) and achieved statistical significance in key secondary endpoints, including duration of severe neutropenia and absolute neutrophil count nadir
Karyopharm Therapeutics Inc., of Newton, Mass. Xpovio (selinexor) Exportin 1 inhibitor Multiple myeloma Results of Boston (bortezomib, selinexor and dexamethasone) study in people with relapsed/refractory disease, reported in March 2020, published online in The Lancet 
Moderna Inc., of Cambridge, Mass. mRNA-1273 COVID-19 Spike glycoprotein modulator COVID-19 infection Cove study met statistical efficacy criteria prespecified in protocol; first interim analysis, based on 95 cases, showed 90 cases of COVID-19 in placebo group vs. 5 in treatment group, resulting in efficacy estimate of 94.5% (p<0.0001)
Medexus Pharmaceuticals Inc., of Montreal Ixinity (trenonacog alfa) Factor IX agonist Factor IX deficiency Study targeting label expansion for children under age 12 with hemophilia B reached 50% enrollment target; full enrollment expected by second quarter of 2021

Notes

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