Company Product Description Indication Status
Phase I
Hookipa Pharma Inc., of New York HB-201 Human papillomavirus E6/E7 protein inhibitor HPV 16-positive cancer First of 100 participants with treatment-refractory disease dosed in open-label phase I/II trial evaluating safety, tolerability and preliminary efficacy based on antitumor activity defined by RECIST 1.1 and immunogenicity, both as monotherapy and in combination with immune checkpoint inhibitor
Lineage Cell Therapeutics Inc. (formerly Biotime Inc.), of Carlsbad, Calif. Opregen Human embryonic stem cell-derived retinal pigmented epithelial cell therapy Dry age-related macular degeneration First cohort 4 participant treated with subretinal delivery system and thaw-and-inject formulation gained 25 readable letters (5 lines) 6 months following administration, as assessed by Early Treatment Diabetic Retinopathy Scale, representing improved visual acuity from baseline of 20/250 to 20/100 in treated eye
Morphosys AG, of Planegg, Germany Tafasitamab (MOR-208) B-lymphocyte antigen CD19 modulator Diffuse large B-cell lymphoma First participant dosed in phase Ib study evaluating safety and preliminary efficacy of drug in addition to rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone (R-CHOP) and in combination with lenalidomide in addition to R-CHOP in adults with newly diagnosed disease
Wave Life Sciences Ltd., of Cambridge, Mass. WVE-120102 HTT gene inhibitor Huntington’s disease Top-line data from ongoing phase Ib/IIa Precision-HD2 trial showed statistically significant reduction of 12.4% (p<0.05) in mutant huntingtin (mHTT) protein in cerebrospinal fluid, with analysis to assess dose response across treatment groups (2, 4, 8 or 16 mg) suggesting statistically significant response in mHTT reduction at highest doses tested (p=0.03); however, no difference seen in total HTT compared to placebo
X4 Pharmaceuticals Inc., of Cambridge, Mass. Mavorixafor (X4P-001) CXCR4 chemokine antagonist Waldenström’s macroglobulinemia Initiated phase Ib trial, in combination with ibrutinib (Imbruvica, Pharmacyclics LLC/Janssen Biotech Inc.), expected to enroll 12 to 18 participants with acquired “gain of function” mutation in CXCR4 in addition to MYD88 mutation; trial also designed to measure changes from baseline in biomarkers serum immunoglobulin M and hemoglobin
Phase II
Jazz Pharmaceuticals plc, of Dublin JZP-458 Asparaginase stimulator Acute lymphoblastic leukemia; lymphoblastic leukemia First participant enrolled in pivotal phase II/III study to evaluate pediatric and adult patients with ALL or LBL who have silent inactivation or allergic reaction to E. coli-derived asparaginases; primary objective is efficacy measured by asparaginase activity
Phase III
Axsome Therapeutics Inc., of New York AXS-07 (meloxicam + rizatriptan) Dual 5-HT 1b/1d receptor agonist; cyclooxygenase 2 inhibitor Migraine Momentum trial achieved statistical significance on regulatory co-primary endpoints of pain freedom (p<0.001) and freedom from most bothersome symptom (p=0.002) at 2 hours, compared to placebo and showed superiority to rizatriptan active comparator on key secondary endpoint of sustained pain freedom 2 to 24 hours after dosing (p=0.038); study drug also showed greater and more sustained migraine pain relief (p=0.006) and reduced use of rescue medication (p<0.001), compared to rizatriptan
Bioxcel Therapeutics Inc., of New Haven, Conn. BXCL-501 Alpha 2A adrenoceptor agonist Agitation Serenity program initiated in individuals with schizophrenia and bipolar disorder; primary endpoint of 2 adaptive trials of up to 750 individuals is change in acute agitation measured by Positive and Negative Syndrome Scale, examining Excited Component change from baseline, compared to placebo; top-line data expected in mid-2020
Eli Lilly and Co., of Indianapolis Selpercatinib (LOXO-292) RET tyrosine kinase inhibitor Medullary thyroid cancer  Libretto-531 trial opened to assess drug vs. physician's choice of cabozantinib or vandetanib in 400 participants with advanced or metastatic treatment-naïve RET-mutant disease; efficacy endpoints are progression-free survival, treatment failure-free survival, overall survival, overall response rate and duration of response; crossover permitted at progression for those randomized to standard of care
Principia Biopharma Inc., of South San Francisco PRN-1008 Bruton tyrosine kinase inhibitor Pemphigus Final data expected in second half of 2021 rather than first half of 2022 due to accelerated enrollment time lines
Sichuan Clover Biopharmaceuticals Inc., of Chengdu, China SCB-808 (etanercept biosimilar) TNF antagonist/binding agent; type II TNF receptor modulator Ankylosing spondylitis First participant dosed in trial in China assessing efficacy, safety and pharmacokinetics of prefilled syringe formulation of drug via subcutaneous administration vs. originator drug


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