Company Product Description Indication Status
Phase I
Aduro Biotech Inc., of Berkeley, Calif. BION-1301 Anti-APRIL monoclonal antibody IgA nephropathy Disclosed in first-quarter 2020 earnings that delays are expected in activating additional sites and enrolling patients in part 3 of study; as a result, data readout will likely be delayed until first half of 2021
Adverum Biotechnologies Inc., of Redwood City, Calif. ADVM-022  Intravitreal injection gene therapy Wet age-related macular degeneration New interim data from cohorts 1-3 in Optic trial in patients requiring frequent anti-VEGF injections showed long-term durability beyond 1 year from single IVT injection with 0 rescue injections in cohort 1; further evidence of dose response, with 6/6 patients rescue injection-free at 6x10^11 vg/eye dose and 8/11 patients rescue injection-free at 2x10^11 vg/eye; early evidence from cohort 3 suggested that 6-week prophylactic regimen of steroid eye drops results in fewer adverse events and less inflammation vs. 13-day prophylactic regimen of oral steroids as used in cohorts 1 and 2; early anatomic and vision improvements seen in cohort 3, with first 5 patients with 20 weeks follow-up showing mean CRT reduction (-137.8 mm) and mean BCVA5 gain (+6.8 letters)
Bicycle Therapeutics plc, of Cambridge, U.K., and Boston BT-5528 Bicyclic peptide Advanced solid tumors First patient dosed in combination part of trial with Opdivo (nivolumab, Bristol Myers Squibb Co.); monotherapy and combination study enrolling patients with solid tumor indications associated with EphA2 expression
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany BNT-162 mRNA vaccine COVID-19 First participants dosed in phase I/II trial; study designed to determine safety, immunogenicity and optimal dose of 4 mRNA vaccine candidates in single, continuous study
Phase II
Angion Biomedica Corp., of San Francisco ANG-3777 Small molecule designed to mimic hepatocyte growth factor  Transplant-associated acute kidney injury Results published in Transplantation demonstrated renal function improvement in patients at high risk for delayed graft function after kidney transplantation
Athersys Inc., of Cleveland Multistem Stem cell therapy COVID-19 First patients enrolled in pivotal phase II/III study in COVID-19-induced acute respiratory distress syndrome; Macovia trial to enroll about 400 subjects; primary efficacy endpoint will compare number of ventilator-free days through day 28 vs. placebo
Delmar Pharmaceuticals Inc., of San Diego VAL-083 (dianhydrogalactitol) Bifunctional DNA-targeting agent Glioblastoma multiforme Has enrolled 22 patients in adjuvant arm of ongoing study testing adjuvant treatment of MGMT-unmethylated GBM; in recurrent arm of study, 72 of planned 83 patients have been enrolled
Redhill Biopharma Ltd., of Tel Aviv, Israel Talicia (RHB-105) Fixed-dose oral capsule combination of 2 antibiotics (amoxicillin and rifabutin) and a proton pump inhibitor Helicobacter pylori infection Pivotal data published in the Annals of Internal Medicine showed 83.8% eradication of H. pylori with Talicia vs. 57.7% for amoxicillin/omeprazole (intent-to-treat analysis, p<0.0001), showing a 26.1% benefit; subjects confirmed adherent to therapy had response rates of 90.3% in Talicia arm vs. 64.7% in active comparator arm (p<0.0001)
Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Libtayo (cemiplimab) PD-1-targeting monoclonal antibody Basal cell carcinoma Top-line data for pivotal, open-label trial in advanced BCC patients who had progressed or were intolerant to prior hedgehog pathway inhibitor showed objective response rate (ORR) of 29% for patients with local disease, with estimated duration of response (DOR) exceeding 1 year in 85% of responders and durable disease control rate (DCR) of 60%; preliminary analysis in metastatic disease patients showed ORR of 21%, with estimated DOR exceeding 1 year in 83% of responders and durable DCR of 46%; regulatory submissions planned for 2020
Phase III
Akebia Therapeutics Inc., of Cambridge, Mass. Vadadustat Oral hypoxia-inducible factor prolyl hydroxylase inhibitor Anemia due to chronic kidney disease in adults on dialysis Top-line results showed Inno2vate cardiovascular outcomes trials hit primary and key secondary endpoints, demonstrating noninferiority to darbepoetin alfa as measured by mean change in hemoglobin between baseline and primary evaluation period (weeks 24 to 36) and secondary evaluation period (weeks 40 to 52); also achieved primary safety endpoint, defined as noninferiority vs. darbepoetin alfa in time to first occurrence of major adverse cardiovascular events
Baudax Bio Inc., of Malvern, Pa. Anjeso (meloxicam) Analgesic Pain management in colorectal surgery Economic substudy of phase IIIb study evaluating preoperative administration as part of a multimodal analgesic regimen vs. placebo in 55 patients who had undergone open or laparoscopic colorectal surgeries showed total mean costs of hospital stay were similar between both groups ($23,115 for Anjeso and $22,682 for placebo; p=0.3370), though removing outliers showed the Anjeso group numerically lower; mean hospital length of stay was numerically lower in Anjeso group (86.2 vs. 111.7 hours; p=0.0162); mean total opioid use significantly lower in Anjeso group from hour 0 through hospital discharge (29.22 mg vs. 45.17 mg; p<0.0339)
Corcept Therapeutics Inc., of Menlo Park, Calif. Relacorilant Selective glucocorticoid receptor antagonist Cushing’s syndrome  Disclosed in first-quarter earnings that start of Gradient study in Cushing’s disease caused by adrenal adenomas or hyperplasia delayed by one quarter due to COVID-19
Nabriva Therapeutics plc, of Dublin Xenleta (lefamulin) Pleuromutilin antibiotic Community-acquired bacterial pneumonia Results from post-hoc analysis from pivotal Leap 1 and 2 trials, published in Open Forum Infectious Diseases, demonstrated that time to clinical response was nearly identical to moxifloxacin, with a median time from treatment initiation to clinical response of 4 (3-4) days for lefamulin and 4 (3-5) days for moxifloxacin; median time from treatment initiation to clinical stability or clinical improvement was 3 (2-4) days in both groups
TG Therapeutics Inc., of New York Umbralisib plus ublituximab Dual inhibitor of PI3K-delta and CK1-epsilon plus CD20 antibody Relapsed/refractory chronic lymphocytic leukemia Top-line results showed Unity-CLL study testing combination compared to obinutuzumab plus chlorambucil in patients with previously untreated disease met primary endpoint at a prespecified interim analysis, showing statistically significant improvement in progression-free survival (p<.0001), and will be stopped early for superior efficacy; data from the study are intended to support a regulatory submission targeted by year-end 2020

Notes

For more information about individual companies and/or products, see Cortellis.

No Comments