Company Product Description Indication Status
Phase I
GT Biopharma Inc., of Beverly Hills, Calif. GTB-3550 Protein immune engager that binds to NK cells and targets them specifically to leukemia cells Relapsed/refractory acute myeloid leukemia  First patient dosed
TFF Pharmaceuticals Inc., of Austin, Texas Voriconazole inhalation powder Antifungal Invasive pulmonary aspergillosis  Completed all 4 dose-escalation cohorts of the single ascending-dose phase of the trial
Phase II
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia Ifenprodil NDMA receptor glutamate antagonist COVID-19 CRO Novotech identified physicians in South Korea who agreed to conduct investigator-initiated trial of repurposed agent, approved to treat certain neurological conditions; development of study protocol underway
Bellus Health Inc., of Laval, Quebec BLU-5937 P2X3 antagonist Chronic cough Dose-escalation, placebo-controlled Relief trial fully enrolled 68 participants, and 75% completed dosing; top-line data due in mid-2020
Cellphire Inc., of Rockville, Md. Thrombosomes Platelet-derived hemostatic agent Thrombocytopenia FDA approved start of trial assessing multiple doses in bleeding patients with thrombocytopenia due to primary bone marrow disorders or chemotherapy, immunotherapy radiation therapy and/or refractory response to fresh platelet concentrate transfusion, with or without splenomegaly
ISA Pharmaceuticals BV, of Leiden, the Netherlands ISA-101b HPV16-specific vaccine Cervical cancer CervISA combination study with carboplatin/paclitaxel in 77 people with late-stage, HPV16-positive disease, published in Science Translational Medicine, showed longer (p=0.012) overall survival (OS) in those with good immune response (median OS 16.8 months) vs. weak response (median OS 11.2 months); 11 of 14 alive at end of study showed strong vaccine-induced response, including 9 stage IVa-IVb individuals with mean OS of 3 years
Nantkwest Inc., of Culver City, Calif. CD16-targeted natural killer (haNK) cells Allogeneic NK cell therapy Merkel cell carcinoma  First of 33 participants with second- and third-line disease refractory to immune checkpoint inhibitors dosed in single-arm combination trial with super agonist N-803 (Immunitybio Inc.) and avelumab (Bavencio, EMD Serono Inc./Pfizer Inc.)
Phase III
Biondvax Pharmaceuticals Ltd., of Jerusalem M-001 (Multimeric-001) Non-flagellin-based, single-protein vaccine Influenza virus infection Ongoing pivotal European trial of universal flu candidate so far unaffected by coronavirus pandemic; CRO and statistician expect study to reach targeted number of swab samples, with results expected by year-end
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-501 (sublingual dexmedetomidine) Alpha 2A adrenoceptor agonist Agitation Enrollment on track in Serenity I and II trials assessing agitation associated with schizophrenia and bipolar disorder, respectively, with more than one-third of participants enrolled and treated; per previous guidance, trials expected to be completed by midyear 2020
Genentech Inc., unit of Roche Holding AG, of Basel, Switzerland Actemra (tocilizumab) IL-6 antagonist/modulator COVID-19  Pending FDA authorization, trial expected to begin in early April in collaboration with BARDA to assess study drug plus standard of care in hospitalized adults with severe COVID-19 pneumonia vs. placebo plus standard of care; global study expected to enroll 330 participants, with primary and secondary endpoints including clinical status, mortality, mechanical ventilation and ICU variables
Novartis AG, of Basel, Switzerland Inclisiran PCSK9 gene inhibitor Atherosclerotic cardiovascular disease Findings from pivotal Orion-10 and -11 trials, published online in The New England Journal of Medicine, showed study drug, at 17 months, resulted in placebo-adjusted LDL-C reductions of 52% and 50%, respectively, and time-adjusted reductions from months 3 through 18 of 54% and 49%, respectively
Novartis AG, of Basel, Switzerland Inclisiran PCSK9 gene inhibitor Heterozygous familial hypercholesterolemia  Findings from pivotal Orion-9 trial, published online in The New England Journal of Medicine, showed study drug, at 17 months, reduced LDL-C by 50% with time-adjusted reduction of 45% from months 3 through 18 vs. placebo
Pfizer Inc., of New York 20-valent pneumococcal conjugate vaccine (20vPnC, PF-06482077) Multivalent Streptococcus pneumoniae vaccine Streptococcus pneumoniae infection Top-line results in participants with no history of pneumococcal vaccination showed noninferiority objectives were met in those 60 and older for serotypes in common with Prevnar 13 (pneumococcal 13-valent conjugate vaccine [diphtheria CRM197 protein], Pfizer) at 1 month after vaccination and for 6 of 7 additional serotypes vs. pneumococcal polysaccharide vaccine PPSV-23 (Pneumovax 23, Merck & Co. Inc.); 1 new serotype missed noninferiority criteria by small margin; secondary immunogenicity objectives in 18- to 59-year-old participants compared to 60- to 64-year-olds met noninferiority for all serotypes
Pfizer Inc., of New York Abrocitinib Janus kinase 1 inhibitor Atopic dermatitis Jade Compare study met co-primary efficacy endpoints in treating moderate to severe disease; percentage who achieved each co-primary efficacy endpoint at week 12 was statistically superior with both 100 mg and 200 mg doses vs. placebo and superiority to placebo with both doses was maintained at week 16; active control dupilumab (Dupixent, Regeneron Pharmaceuticals Inc./Sanofi SA) was superior to placebo at weeks 12 and 16; key secondary endpoint, percentage with clinically significant reduction in itch by week 2 of treatment, was statistically superior for 200 mg abrocitinib vs. dupilumab and numerically higher but not statistically significantly higher for 100 mg vs. dupilumab
Phathom Pharmaceuticals Inc., of Florham Park, N.J. Vonoprazan H+ K+ ATPase inhibitor Erosive esophagitis Randomization in pivotal Phalcon-EE trial, initiated in fourth quarter of 2019, paused temporarily to cooperate with efforts to halt spread of COVID-19; top-line data expected in 2021
Phathom Pharmaceuticals Inc., of Florham Park, N.J. Vonoprazan H+ K+ ATPase inhibitor Helicobacter pylori infection Randomization in pivotal Phalcon-HP combination trial with amoxicillin (dual therapy) and with amoxicillin and clarithromycin (triple therapy), initiated in fourth quarter of 2019, paused temporarily to cooperate with efforts to halt spread of COVID-19; top-line data expected in 2021
Soligenix Inc., of Princeton, N.J. SGX-301 (synthetic hypericin) Heat-shock protein inhibitor Cutaneous T-cell lymphoma  Cycle 1 data from pivotal Flash trial that enrolled 169 participants randomized 2-to-1 to study drug or placebo showed statistically significant treatment response (p=0.04) in Composite Assessment of Index Lesion Score primary endpoint assessment at 8 weeks

Notes

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