Company Product Description Indication Status
Phase I
Advaxis Inc., of Princeton, N.J. ADXS-503 Therapeutic vaccine Metastatic non-small-cell lung cancer First 2 patients treated in the combination arm who previously progressed on Keytruda (pembrolizumab, Merck & Co. Inc.) showed substantial tumor shrinkage; 1 patient achieved a partial response and the other stable disease, with 25% reduction in target lesion
Argenx NV, of Breda, the Netherlands Cusatuzumab (ARGX-110)  Simple Antibody targeting CD70 Acute myeloid leukemia Due to COVID-19, partner Janssen Pharmaceuticals paused enrollment in phase Ib trial testing drug in combination with venetoclax and azacitidine
Frequency Therapeutics Inc., of Woburn, Mass. FX-322 Activator of Lgr5-positive epithelial stem cell proliferation and differentiation Sensorineural hearing loss Top-line results of exploratory study showed measurable concentrations of drug in every patient and that anatomical factors did not prevent the active agents from reaching the cochlea
Krystal Biotech Inc., of Pittsburgh KB-105 TGM1 gene stimulator Lamellar ichthyosis Clearly detectable TGM-1 expression in all treated areas following administration; phenotypic evaluation of treated areas showed reduced reversion to ichthyotic scaling phenotype
LG Chem Life Sciences Innovation Center Inc., of Cambridge, Mass. LC51-0255 S1P1 modulator Ulcerative colitis Results from healthy volunteer study showed drug was well-tolerated using oral administration for up to 21 days; systemic exposure and absolute lymphocyte count reduction proved dose-proportional, with favorable pharmacokinetic profile
Phoenix Tissue Repair Inc., of Boston PTR-01 I.V.-administered recombinant collagen 7 protein replacement Recessive dystrophic epidermolysis bullosa Well-tolerated in first 3 cohorts of phase I/II trial, with dose-dependent increase in collagen 7 skin deposition seen after just 3 infusions over 28 days; planning 6-month open-label study to inform pivotal trial design
Tolero Pharmaceuticals Inc., of Salt Lake City Alvocidib CDK9 inhibitor Myelodysplastic syndrome First patient dosed in Zella 102 study, testing 30-minute bolus followed by intravenous infusion in combination with decitabine
Ultragenyx Pharmaceutical Inc., of Novato, Calif. DTX-301 AAV8-based gene therapy Ornithine transcarbamylase deficiency 6 of 9 patients in the study have responded to the gene therapy, including all 3 patients in cohort 3 who are now confirmed responders
Viela Bio Inc., of Gaithersburg, Md. VIB-7734 Anti-ILT7 therapy Cutaneous lupus erythematosus Interim data showed safety comparable to placebo, depletion of plasmacytoid dendritic cells in peripheral blood and skin biopsies and dose-dependent improvements in CLASI scores
Phase II
Abivax SA, of Paris ABX-464 Rev protein modulator SARS-CoV-2-related inflammation Phase IIb/III Mir-age study to assess 50 mg, once-daily oral dose of study drug vs. placebo on top of standard of care in 1,034 elderly or high-risk adults with COVID-19 infection for prevention of acute respiratory distress syndrome; primary endpoint is absence of high-flow oxygen use, assisted ventilation or death within 28 days
Argenx NV, of Breda, the Netherlands Cusatuzumab (ARGX-110)  Simple Antibody targeting CD70 Acute myeloid leukemia Partner Janssen Pharmaceuticals paused enrollment of pivotal Culminate trial, testing combination with azacitidine in newly diagnosed elderly patients unfit for intensive chemotherapy, due to COVID-19
Capricor Therapeutics Inc., of Los Angeles CAP-1002 Cardiosphere-derived allogenic stem cell therapy Duchenne muscular dystrophy Top-line results of HOPE-2 study found improved upper limb strength and global improvements in cardiac function and correlated cardiac functional stabilization with reduction of a biomarker of cell damage; company meeting with FDA to discuss path ahead
Diamedica Therapeutics Inc., of Minneapolis DM-199 Insulin sensitizer; kallikrein 1 modulator Ischemic stroke Met primary safety and tolerability endpoints in the Remedy study with no drug-linked serious adverse events; also demonstrated therapeutic effect in participants who received tPA prior to enrollment, but not those receiving mechanical thrombectomy
Hookipa Pharma Inc., of New York and Vienna HB-101 Vaxwave-based prophylactic cytomegalovirus vaccine  Cytomegalovirus Due to COVID-19 pandemic, nearly all ongoing trial sites have suspended patient enrollment; it remains unclear when kidney organ transplants will resume at any of the sites
Horizon Therapeutics plc, of Dublin Tepezza (teprotumumab-trbw) Insulin-like growth factor 1 receptor antagonist Thyroid eye disease  Population pharmacokinetic analysis performed on phase II and III studies showed drug had long elimination half-life, low systemic clearance and low volume of distribution, supporting dosing (initial infusion of 10 mg/kg followed by 7 infusions of 20 mg/kg once every 3 weeks) used in trials and on label
Kaleido Biosciences Inc., of Lexington, Mass. KB-109 Microbiome metabolic therapy COVID-19 infection Study of KB-109 added to supportive self-care initiated in about 350 individuals in outpatient setting who are positive for SARs-CoV-2 with mild to moderate COVID-19 infection; top-line data expected in fourth quarter of 2020
Morphosys AG, of Planegg, Germany, and Incyte Corp., of Wilmington, Del. Tafasitamab  Humanized Fc-engineered monoclonal antibody directed against CD19 Relapsed or refractory diffuse large B-cell lymphoma Updated results from ongoing L-Mind study testing combination with lenalidomide from 80 patients in long-term analysis showed objective response rate of 58.8% (47 of 80 patients) and complete response rate of 41.3% (33 of 80); median duration of response was 34.6 months, with median overall survival of 31.6 months and median progression-free survival of 16.2 months
Relief Therapeutics Holding AG, of Geneva, and Neurorx Inc., of Radnor, Pa. RLF-100 (aviptadil, inhaled) Vasoactive intestinal polypeptide receptor agonist COVID-19-associated acute respiratory distress syndrome Final manufacturing completed for phase IIb/III trial to assess intravenous formulation in individuals on mechanical ventilation; enrollment initiated at first U.S. site
X4 Pharmaceuticals Inc., of Cambridge, Mass. Mavorixafor CXCR4 chemokine antagonist WHIM syndrome Open-label extension (up to 28.6 months) showed sustained, dose-dependent increases in absolute neutrophil and lymphocyte counts; long-term hematological improvements correlated with fewer infections and improved cutaneous warts; yearly infection rates decreased by 48%, from 4.63 in 12 months prior to trial to 2.41, at 300 mg and by 54%, to 2.14, at 400 mg; participants with cutaneous warts on hands and/or feet at baseline showed >50% reduction in warts at last dermatological evaluation
Phase III
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-501 (dexmedetomidine, sublingual) Alpha-2a receptor agonist Agitation Pivotal Serenity trials evaluating study drug in individuals with schizophrenia and bipolar disorder fully enrolled; top-line data expected in July 2020
Galmed Pharmaceuticals Inc., of Tel Aviv, Israel Aramchol Liver-targeted SCDI modulator Nonalcoholic steatohepatitis and fibrosis Decided to temporarily halt screening of new patients for phase III/IV Armor study due to COVID-19, and aims to resume activity on a country by country, state by state and site by site basis as conditions improve
Pfizer Inc., of New York PF-06482077 (20vPnC) 20-valent pneumococcal polysaccharide conjugate vaccine Streptococcus pneumoniae infection Responses elicited by vaccine candidate for the 20 serotypes were equivalent across all 3 lots, meeting primary immunogenicity objective; safety profile was similar to Prevnar 13 (pneumococcal 13-valent conjugate vaccine [diphtheria CRM197 protein], Pfizer) control 
Regentree LLC, of Princeton, N.J. RGN-259 (timbetasin) Thymosin beta 4 ligand Neurotrophic keratopathy In Seer-1 trial, which recruited 18 instead of 46 participants and closed early, 6 of 10 in treated group and 1 of 8 in placebo group achieved complete corneal healing in 4 weeks, for statistical difference of >0.05 (p = 0.0656, Fisher's exact test) in primary endpoint of ratio of corneal wound healed patients after 4 weeks of administration; prespecified secondary endpoint of corneal epithelial healing at day 43 and durability of treatment showed statistical difference using the Fisher's exact test (p = 0.0359)


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

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