Company Product Description Indication Status
Phase I
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. ARQ-252 Janus kinase type 1 inhibitor Chronic hand eczema Enrolled first patient in study to test the safety, tolerability and pharmacokinetics of the cream for 2 weeks in 6 patients followed by a phase IIb portion testing the drug for 12 weeks; phase IIb scheduled to begin in the second half of 2020 with top-line data expected in the second half of 2021
Hookipa Biotech Inc., of New York HB-101 Cytomegalovirus  vaccine Cytomegalovirus  infection prophylaxis Data published in The Journal of Infectious Diseases showed the vaccine induced cytomegalovirus-specific poly-functional CD8 T-cell and neutralizing antibody responses in nearly all subjects; there were no vector-neutralizing antibodies, allowing for re-administration of the vaccine
Idera Pharmaceuticals Inc., of Exton, Pa. Tilsotolimod Toll-like receptor 9 agonist Anti-PD-1 refractory advanced melanoma In the Illuminate-204 study, median overall survival was 21.0 months in 49 evaluable patients treated at the 8-mg dose; overall response rate was 22.4%, including 2 complete responses
Phase II
Applied Therapeutics Inc., of New York AT-007 Aldose reductase inhibitor Galactosemia Drug dosed at 20 mg/kg reduced galactitol levels by approximately 50% within 6 days, which was sustained through the 27-day treatment period (p<0.01 vs. placebo); no increase in galactose observed; drug improved ventricular volume measured with MRI and improved N-acetyl-aspartate; decrease in brain galactitol levels observed by quantitative MR spectroscopy; testing the 40-mg/kg dose in healthy volunteers
Atyr Pharma Inc., of San Diego ATYR-1923 Immunomodulatory domain of histidyl tRNA synthetase fused to the FC region of a human antibody COVID-19 with severe respiratory complications FDA accepted the IND for a phase II study of 30 patients comparing two doses of drug to placebo
Catalyst Biosciences Inc., of South San Francisco SQ dalcinonacog alfa Factor IX Hemophilia B Completed dosing and the 30-day follow-up period; data expected during the second quarter of 2020
Phase III
Cara Therapeutics Inc., of Stamford, Conn., and Vifor Fresenius Medical Care Renal Pharma, of St. Gallen, Switzerland Korsuva (CR-845/difelikefalin)  KOR agonist Haemodialysis patients with moderate to severe chronic kidney disease-associated pruritus 54% of patients had a 3-point or greater improvement from baseline in the weekly mean of the daily 24-hour Worst Itching Intensity Numeric Rating Scale score at week 12 compared to 42% of patients on placebo (p= 0.02); 4-point improvement seen in 41% of patients on drug vs. 28% for patients on placebo (p= 0.01)
Erytech Pharma SA, of Lyon, France Eryaspase L-asparaginase encapsulated inside donor-derived red blood cells Second-line advanced metastatic pancreatic cancer More than 75% of the 500 patients enrolled; independent data monitoring committee found no safety issues in its third safety review; interim analysis expected around the end of 2020; final analysis expected in second half of 2021
Novartis AG, of Basel, Switzerland Mayzent (siponimod) Sphingosine 1-phosphate receptor modulator Secondary progressive multiple sclerosis 5-year data from the Expand study published in Neurology showed a 52% reduction in the annualized relapse rate for the Mayzent group compared to the placebo switch group (p<0.0001); risk of confirmed worsening of cognitive impairment (measured on the Symbol Digit Modalities Test) at 6 months reduced by 23% for the Mayzent group compared with placebo switch group (p=0.0014); benefits seen in the Mayzent group were sustained for up to five years
Oncoimmune Inc., of Rockville, Md.   CD24Fc Novel therapeutic Inflammation Initiated phase III trial testing the safety and efficacy of CD24Fc in hospitalized COVID-19 patients at the University of Maryland Baltimore Medical Center; at least 10 U.S. medical centers  will test the impact of CD24Fc in dampening inflammation caused by virus-induced cellar injuries, which is likely a major underlying cause of cytokine storm, immune dysfunction and pneumonia observed in severe COVID-19 patients
Synexis Inc., of Jersey City, N.J. Ibrexafungerp Broad-spectrum antifungal Vulvovaginal candidiasis In the Vanish-306 study, 63.3% of patients treated with drug had a clinical cure at day 10; 58.5% of patients had mycological eradication; 72.3% of patients were clinically improved; 73.9% had complete symptom resolution at day 25
Phase IV
CSL Behring, of King of Prussia, Pa., a unit of CSL Ltd. Hizentra (immune globulin subcutaneous) Subcutaneous immunoglobulin therapy Primary immunodeficiency  The Hilo study testing a new delivery method showed responder rates for patients treated with pump-assisted delivery were 86.7% for the 25 mL volume and 73.3% for the 50 mL and 50 mL volumes; responder rates were 77.8% (25 and 50 mL/hour), 66.7% (75 mL/hour) and 61.1% (100 mL/hour) by patient assigned to specific pump-assisted flow rates; mean serum immunoglobulin G trough levels were similar between day 1 and end of the study for the volume cohort and the flow rate cohort; responder rates for manual push were 100% (0.5 and 1 mL/minute) and 87.5% (2 mL/minute)


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