Company Product Description Indication Status
Phase I
Atara Biotherapeutics Inc., of South San Francisco ATA-188  Allogeneic T-cell activated therapy Progressive multiple sclerosis Temporarily paused screening and enrollment of patients in phase Ib study due to COVID-19; company expects pause to be limited and plans to initiate enrollment in second or third quarter of 2020
Avrobio Inc., of Cambridge, Mass. AVR-RD-04 Gene therapy  Cystinosis Patient identification activities ongoing in phase I/II study, though certain data collection temporarily delayed due to COVID-19
Avrobio Inc., of Cambridge, Mass. AVR-RD-02 Gene therapy  Gaucher disease First patient enrolled and completed apheresis in phase I/II study, and dosing is anticipated for the second quarter but is dependent on when the clinical site allows; subsequent new enrollment timelines impacted by COVID-19
Rubius Therapeutics Inc., of Cambridge, Mass. RTX-240 Allogeneic cell therapy Solid tumor First patient dosed in phase I/II trial in relapsed/refractory or locally advanced solid tumors
Solid Biosciences Inc., of Cambridge, Mass. SGT-001 Gene transfer therapy Duchenne muscular dystrophy Ignite-DMD trial remains on clinical hold, per FDA, which requested further data and analyses relating to manufacturing; company expects to submit response by end of third quarter of 2020
X4 Pharmaceuticals, of Cambridge, Mass. Mavorixafor  CXCR4 chemokine antagonist  Waldenström’s macroglobulinemia Trial readout expected in second half of 2020
Phase II
Arca Biopharma Inc., of Westminster, Colo. Gencaro (bucindolol hydrochloride) Beta-blocker and mild vasodilator Atrial fibrillation in patients with heart failure In the 267-patient Genetic-AF study, Gencaro decreased cumulative atrial fibrillation (AF) burden by 26% compared to metoprolol succinate (p<0.001); compared to metoprolol, Gencaro decreased a composite endpoint of AF interventions and cardiovascular adverse events by 30% (p=0.008) and decreased AF interventions by 33% (p=0.009)
Avrobio Inc., of Cambridge, Mass. AVR-RD-01 Gene therapy Fabry disease Patient identification activities for FAB-201 trial continue for trial sites in Australia, Canada and the U.S.; dosing of new patients, which was paused as a result of COVID-19, is expected to resume as clinical sites allow; certain data collection has been temporarily delayed
Bicycle Therapeutics plc, of Cambridge, U.K. BT-1718 Peptide-toxin conjugate targeting MT1-MMP  Solid tumors Enrollment of new patients in phase IIa portion of trial at Cancer Research UK sites paused due to the COVID-19 pandemic
Calcimedica Inc., of La Jolla, Calif. CM-4620-IE Ca2+ release activated Ca2+ channel 1 inhibitor  Severe COVID-19 pneumonia Independent safety review committee recommended that the study continue without modification
Cidara Therapeutics Inc., of San Diego Rezafungin Echinocandin antifungal Invasive fungal disease Analysis of Strive study published in European Congress of Clinical Microbiology and Infectious Diseases meeting abstract showed single dose had a day 5 success rate of 62.3% vs. 55.7% for approved echinocandin comparator caspofungin dosed once daily; time to negative blood culture in rezafungin group was statistically faster than caspofungin group; analysis from parts A and B of Strive study showed treatment outcomes were comparable between North American and European patients
Enanta Pharmaceuticals Inc., of Watertown, Mass. EDP-305  Farnesoid X receptor agonist Primary biliary cholangitis In the Intrepid study, treatment with 1 mg and 2.5 mg produced a 20% reduction in alkaline phosphatase (ALP) at week 12 in 45% (n=14/31, p=0.106) and 46% (n=13/28, p=0.063) of patients, respectively, compared to 11% (n=1/9) of patients treated with placebo; absolute changes from baseline in ALP at week 12 for the 1-mg arm (p=0.017) and the 2.5-mg arm (p= 0.021) were statistically significant compared to the change from baseline in the placebo arm 
Enlivex Therapeutics Ltd., of Nes Ziona, Israel Allocetra Immunotherapy  COVID-19 associated lung dysfunction Started study measuring change in PaO2/FiO2 ratio number and severity of adverse events and serious adverse events as the co-primary endpoints
Hightide Therapeutics Inc., of Shenzhen, China HTD-1801 Ionic salt of 2 active moieties Nonalcoholic steatohepatitis and type 2 diabetes mellitus Phase IIa study met primary endpoint of an absolute liver fat reduction; secondary endpoints, including glycemic control and markers associated with liver injury, were also met; data to be presented at an upcoming medical meeting
Monopar Therapeutics Inc., of Chicago Validive Mucobuccal tablet of clonidine based on Lauriad mucoadhesive technology Severe oral mucositis In response to COVID-19, company has modified original adaptive design phase III trial to be a phase IIb/III trial to better fit the types of trials which can enroll patients in the current environment; primary endpoint of absolute incidence of SOM remains the same, but touch points with health care system have been minimized
Ovid Therapeutics Inc., of New York OV-101 (gaboxadol) Delta-selective GABAA receptor agonist Fragile X syndrome  In the Rocket study at week 12, the drug produced a 26.2% mean improvement in the Aberrant Behavior Checklist-Community for Fragile X syndrome total score from baseline (p=0.002), a 21.6% mean improvement in the Anxiety, Depression and Mood Scale total score from baseline (p=0.004) and a mean reduction of 0.4 in the Clinical Global Impressions Severity scale total score from baseline (p=0.002)
Oxurion NV, of Leuven, Belgium THR-687 Small-molecule pan-RGD integrin antagonist Diabetic macular edema Delayed start of planned study to test multiple doses of drug due to COVID-19 pandemic; trial to start as soon as conditions allow
Progenics Pharmaceuticals Inc., of New York 1095 Small-molecule radiotherapeutic targeting PSMA Metastatic castration-resistant prostate cancer Due to COVID-19, decided to place enrollment of new patients on hold in Arrow study testing combination with enzalutamide in chemotherapy-naïve patients
Rising Pharma Holdings Inc., of East Brunswick, N.J. Hydroxychloroquine  Inhibits virus entry COVID-19 prophylaxis in patients with cancer Memorial Sloan Kettering Cancer Center running the placebo-controlled study measuring likelihood of symptomatic COVID-19 infection as the primary endpoint; secondary endpoints include likelihood of severe COVID-19 and acute toxicity associated with administration of hydroxychloroquine during radiation or chemoradiation; drug donated by Rising Pharma
Swedish Orphan Biovitrum (SOBI) AB, of Stockholm Emapalumab  Monoclonal antibody targeting interferon gamma Primary hemophagocytic lymphohistiocytosis Data published in The New England Journal of Medicine showed the drug produced an overall response rate of 63% in previously treated patients; 70% of previously treated patients were able to proceed to transplantation
Theravance Biopharma Inc., of Dublin TD-1473 Gut-selective, oral pan-JAK inhibitor Ulcerative colitis Data from phase IIb portion of phase IIb/III Rhea maintenance study originally planned for late 2020 is now expected in 2021; delay due to 4-week suspension of new enrollment in response to COVID-19
Theravance Biopharma Inc., of Dublin TD-1473 Gut-selective, oral pan-JAK inhibitor Crohn’s disease Data from Dione study originally planned in late 2020 is now expected in 2021; delay due to 4-week suspension of new enrollment in response to COVID-19
Phase III
Aldeyra Therapeutics Inc., of Lexington, Mass. ADX-2191 Reactive aldehyde species inhibitor Proliferative vitreoretinopathy Patient enrollment in part 1 of adaptive Guard trial for prevention of PVR significantly delayed due to lack of clinical site availability and staffing resulting from COVID-19
Innovent Biologics Inc., of Suzhou, China, and Eli Lilly and Co., of Indianapolis Tyvyt (sintilimab) Monoclonal antibody targeting PD-1 First-line advanced or metastatic squamous non-small-cell lung cancer Orient-12 study testing Tyvyt plus Gemzar (gemcitabine, Lilly) and platinum chemotherapy met primary endpoint of progression-free survival; data to be presented at an upcoming medical conference
Ironwood Pharmaceuticals Inc., of Boston IW-3718 Gastric-retentive bile-acid sequestrant Refractory gastroesophageal reflux disease COVID-19 pandemic impacting enrollment in 2 trials, with most clinical sites no longer screening new patients; top-line data no longer expected in second half of 2020
Theravance Biopharma Inc., of Dublin Ampreloxetine (TD-9855) Norepinephrine reuptake inhibitor Symptomatic neurogenic orthostatic hypertension Data from 4-week Sequoia trial originally planned for late 2020 is now expected in 2021; delay due to 4-week suspension of new enrollment in response to COVID-19
X4 Pharmaceuticals Inc., of Cambridge, Mass. Mavorixafor  Antagonist of chemokine receptor CXCR4 WHIM syndrome Timing of top-line data release delayed from second half of 2021 to 2022 due to COVID-19
Phase IV
Wize Pharma Inc., of Hod Hasharon, Israel LO2A   Sodium hyaluronate  Dry eye syndrome in patients with Sjögren's syndrome Completed dosing of patients in the study; top-line data release may be delayed into third fiscal quarter of 2020 due to COVID-19


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

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