Company Product Description Indication Status
Phase I
Cleave Therapeutics Inc., of San Francisco CB-5339 VCP/p97 inhibitor Relapsed/refractory acute myeloid leukemia or relapsed/refractory intermediate or high-risk myelodysplastic syndrome Treated first of 50-60 patients in the study designed to determine the maximum tolerated dose and/or recommended phase II dose of CB-5339
Cue Biopharma Inc., of Cambridge, Mass. CUE-101 Engager and activator of T cells  Human papillomavirus-positive recurrent/metastatic head and neck squamous cell carcinoma Following safety review committee assessment of cohort 4, first patient dosed in cohort 5
Ivy Brain Tumor Center at Barrow Neurological Institute, of Phoenix, and San Francisco-based Bridgebio Pharma Inc. Infigratinib  FGFR1-3 tyrosine kinase inhibitor Recurrent high-grade glioma driven by FGFR genetic alterations First patient treated in an investigator-initiated study; phase 0 will assess how well drug crosses the blood-brain barrier; phase II portion will measure progression-free survival rate at 6 months
Mind Medicine Inc., of New York 18-MC Non-hallucinogenic molecule based on the psychedelic substance ibogaine Opioid addiction Single ascending dose was well-tolerated; multiple ascending-dose study continues; plans to start a phase IIa study by the end of 2020
Miragen Therapeutics Inc., of Boulder, Colo. MRG-110  MiR-92 inhibitor Healthy volunteers (eventually heart failure and other ischemic diseases) Data published in Nucleic Acid Therapeutics showed the drug reduced miR-92a levels in whole blood, circulating endothelial cells and circulating extracellular vesicles; 2 miR-92a target genes were derepressed
Plus Therapeutics Inc., of Austin, Texas Rhenium nanoliposomes Radiotherapy Recurrent glioblastoma after standard surgical, radiation and chemotherapy treatment Completed fifth cohort of the Respect study; 1 cohort still planned; expanding study to another site
Phase II
American Brivision Holding Corp., of Fremont, Calif. ABV-1505 Inhibitor of the norepinephrine transporter Adult attention-deficit hyperactivity disorder Completed part 1 of the study; data expected in the fourth quarter of 2020
Arch Biopartners Inc., of Toronto Metablok Dipeptidase-1 peptide inhibitor Prevention of organ inflammation and acute kidney injury in COVID-19 Chose Broward Health Medical Center in Florida as the first U.S. clinical site for study; enrollment expected to start in August
Grifols SA, of Barcelona, Spain Albumin and immunoglobulin Plasma protein replacement therapy Mild or moderate Alzheimer’s disease Findings of phase II/III Ambar study, published in The Journal of the Alzheimer’s Association, demonstrate delay in cognitive and functional decline in AD patients when their plasma is replaced with albumin and immunoglobulin following the process of plasma extraction, using the plasmapheresis technique; results reveal positive impact in reducing progression of symptoms in patients treated over 14-month period vs. untreated patients
Iterion Therapeutics Inc., of Houston Tegavivint  Nuclear beta-catenin inhibitor Desmoid tumors Started enrollment of up to 25 patients in the phase IIa expansion study
Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany BNT-162b2 Nucleoside-modified mRNA vaccine COVID-19 Started global (except for China) phase II/III study testing candidate at 30-ug dose level in a 2-dose regimen; study to involve up to 30,000 participants
Reata Pharmaceuticals Inc., of Plano, Texas Bardoxolone  Activator of Nrf2  COVID-19 NYU Grossman School of Medicine initiated the Barcona investigator-sponsored study
Redhill Biopharma Ltd., of Tel Aviv, Israel RHB-102  Bimodal-release ondansetron Diarrhea-predominant irritable bowel syndrome Data from the 126-patient study published in The American Journal of Gastroenterology showed stool consistency of 56% for RHB-102 compared to 35.3% for placebo (p=0.036)
Spectrum Pharmaceuticals Inc., of Henderson, Nev. Poziotinib  EGFR tyrosine kinase inhibitor Previously treated non-small-cell lung cancer with HER2 exon 20 insertion mutations In cohort 2 of the Zenith20 study, drug produced an objective response rate of 27.8%; median duration of response was 5.1 months; median progression-free survival was 5.5 months
Phase III
Abbvie Inc., of North Chicago Rinvoq (upadacitinib) JAK inhibitor Atopic dermatitis In the AD Up study, Rinvoq at 15 mg and 30 mg plus topical corticosteroids (TCS) produced at least a 75% improvement in the Eczema Area Severity Index in 65% and 77% of patients, respectively, compared to 26% of patients taking TCS alone (p<0.001); 40% and 59% of patients taking 15 mg and 30 mg of Rinvoq plus TCS, respectively, achieved an Investigator's Global Assessment for Atopic Dermatitis score of 0 or 1, compared to 11% of patients taking TCS alone (p<0.001)
Astrazeneca, of Cambridge, U.K. Farxiga (dapagliflozin) SGLT2 inhibitor Chronic kidney disease DAPA-CKD study met its primary endpoint of a composite of worsening of renal function or risk of death, defined as a composite endpoint of ≥50% sustained decline in estimated glomerular filtration rate, onset of end-stage kidney disease or cardiovascular or renal death; study also met its secondary endpoints; data to be presented at a forthcoming medical meeting
Chiasma Inc., of Needham, Mass. Mycapssa (octreotide) Somatostatin analogue Acromegaly In the Chiasma Optimal open-label extension, mean IGF-1 levels were within normal limits at the end of the extension for 19 patients who took drug during the 36-week study and the 48-week extension; 93% of 14 patients who responded with IGF-1 within normal limits during the study maintained their response within the normal limits at the end of the extension
Corcept Therapeutics Inc., of Menlo Park, Calif. Relacorilant Nonsteroidal, selective modulator of glucocorticoid receptor Cushing’s syndrome Enrolled first patient in Gradient trial in patients with Cushing’s caused by adrenal adenomas or hyperplasia; about 130 patients will be enrolled; primary endpoints are improvement in glucose metabolism and hypertension
Orion Corp., of Espoo, Finland Levosimendan Calcium sensitizer Amyotrophic lateral sclerosis Refals trial did not reach prespecified endpoints, including statistically significant difference vs. placebo in slow vital capacity (SVC) in supine position at 12 weeks compared to baseline, and secondary endpoint of statistically significant difference vs. placebo in patient functionality measured by Revised Amyotrophic Lateral Sclerosis Functional Rating Scale in combination with survival at 48 weeks compared to baseline
Recordati Rare Diseases, of Puteaux, France, part of Recordati Group Isturisa (osilodrostat)  Oral inhibitor of 11-beta-hydroxylase Cushing’s syndrome The Lancet Diabetes & Endocrinology published results from Linc-3 study in adults with endogenous disease, showing rapid reduction in mean urinary free cortisol sustained alongside improvements in co-morbidities, clinical signs and quality of life over 48 weeks


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