Company Product Description Indication Status
Phase I
Alpine Immune Sciences Inc., of Seattle ALPN-101 Dual CD28/ICOS co-stimulation antagonist Autoimmune and inflammatory diseases Updated results in healthy volunteers showed drug was well-tolerated with no serious adverse events; pharmacokinetics and pharmacogenomics exhibited desirable dose dependence, with increasing doses corresponding to increasing duration of complete or near-complete target saturation as well as inhibition of antibody responses to keyhole limpet hemocyanin immunization
Astrazeneca plc, of Cambridge, U.K. Exenatide once-weekly suspension GLP-1 receptor agonist Type 2 diabetes Study testing prefilled, single-dose autoinjector in Chinese patients stopped based on strategic considerations impacting clinical development of exenatide once-weekly suspension in China
Axovant Gene Therapies Ltd., of New York, and Basel, Switzerland AXO-AAV-GM1 Gene therapy Type II (late infantile and juvenile onset) GM1 gangliosidosis Completed enrollment in low-dose cohort in phase I/II study, at a dose of 1.5x1013 vg/kg delivered intravenously; study on track to report 6-month data from low-dose cohort by fourth quarter of 2020
Bavarian Nordic A/S, of Copenhagen MVA-BN WEV Prophylactic vaccine Equine encephalitis viruses (western, eastern and Venezuelan) Top-line results from healthy volunteers showed vaccine was well-tolerated and immunogenic across all dose groups; neutralizing antibody responses were observed in all dose groups, with peak levels reached after second vaccination; responses detected as early as 2 weeks after first vaccination in highest dose group
Blueprint Medicines Corp., of Cambridge, Mass. Avapritinib Kinase inhibitor Indolent systemic mastocytosis Updated data from part 1 of Pioneer trial showed treatment resulted in deepening improvements in overall disease symptoms, as measured by Indolent SM Symptom Assessment Form (ISM-SAF) total symptom score, and was well-tolerated through 24 weeks of follow-up; mean percent change from baseline was -35% in avapritinib 25 mg QD group (n=10) vs. -4% in placebo group; mean percent change from baseline in ISM-SAF skin domain score was -38%for avapritinib vs. +11% for placebo
Celldex Therapeutics Inc., of Hampton, N.J.  CDX-0159 KIT inhibitor Chronic urticaria Data from healthy subjects showed favorable safety profile as well as durable reductions of plasma tryptase, consistent with systemic mast cell suppression; a single dose induced dose-dependent tryptase reduction below the level of assay detection within days at doses as low as 1 mg/kg and maintained suppression for 2+ months at 3 mg/kg and above
Eli Lilly and Co., of Indianapolis LY-3381916 and LY-3300054 IDO-1 inhibitor and anti-PD-L1 checkpoint antibody Solid tumors Study terminated due to strategic business decision
Janssen Pharmaceuticals, a unit of New Brunswick, N.J.-based Johnson & Johnson Guselkumab Monoclonal antibody targeting IL-23 Hidradenitis suppurativa  Study withdrawn due to COVID-19
Junshi Biosciences Ltd., of Shanghai, and Eli Lilly and Co., of Indianapolis JS-016 SARS-CoV-2 neutralizing antibody COVID-19 Dosed first healthy volunteer in China study
Neurana Pharmaceuticals Inc., of San Diego Tolperisone Skeletal muscle relaxant Muscle spasms Enrolled first patient in study to test central nervous effects of drug in acute and painful muscle spasms without drowsiness or impact on cognitive function
Precision Biosciences Inc., of Durham, N.C. PBCAR-269A Allogeneic CAR T-cell therapy targeting BCMA Relapsed/refractory multiple myeloma Dosed first patient in phase I/IIa trial 
Sarepta Therapeutics Inc., of Cambridge, Mass. SRP-9003 Gene therapy  Limb-girdle muscular dystrophy type 2E Results from 3 participants in high-dose cohort measured at 60 days showed mean expression of 72.3% of transduced beta-SG, properly localized to the muscle sarcolemma, as measured by immunohistochemistry, exceeding predefined measure of success (50% positive fibers, which was previously achieved in cohort 1); all showed robust quantification of beta-SG, as measured by Western blot, with mean beta-SG of 62.1% of normal control, and reduction in serum creatine kinase levels (reduction of 89.1% from baseline); 1-year data from low-dose cohort showed all 3 participants continue to demonstrate improvements from baseline across all functional measures
Phase II
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. Berotralstat Oral antagonist of plasma kallikrein Hereditary angioedema New data from Apex-2 and Apex-S studies showed sustained decreases in attack frequency and improvements in quality of life scores over 48 weeks; in Apex-2, 31 patients on treatment had baseline attack rate of 2.9/month, which declined to 1.5/month after 1 month and to 1/month at 12 months; in Apex-S, patients had median attack rate of 0/month in 6 of 12 months, including month 12
CSL Behring, of Melbourne, Australia, a unit of CSL Ltd. Garadacimab (CSL-312) Factor XIIa-inhibitory monoclonal antibody Hereditary angioedema Data showed study met primary endpoint, demonstrating reduced number of attacks compared to placebo; mean percentage reductions were 88.68%, 98.94% and 90.50% in 3 garadacimab groups (75 mg, 200 mg and 600 mg subcutaneous doses) vs. placebo 
Fibrogen Inc., of San Francisco Pamrevlumab CTGF-targeting monoclonal antibody COVID-19 Started open-label phase II/III Borea study testing drug vs. standard of care in about 68 patients with hospitalized COVID-19 infection; primary objective is to assess the effect on blood oxygenation 
Gilead Sciences Inc., of Foster City, Calif., and Galapagos NV, of Mechelen, Belgium Filgotinib Oral JAK1 inhibitor Psoriatic arthritis Analyses from 2 studies, Equator and Equator-2, showed rapid sustained reductions in inflammatory biomarkers in patients with moderate to severe PsA; consistently demonstrated a statistically significant higher proportion of patients achieving ACR20 response vs. placebo across all subgroups; achieved a higher proportion of ACR50 response and Psoriatic Arthritis Disease Activity Score of low disease activity vs. placebo, reaching statistical significance in most subgroups; response patterns from open-label extension (OLE) study showed majority of patients who achieved minimal disease activity (MDA) and ACR50 response in original Equator trial maintained MDA and ACR50 at week 52 and a proportion of nonresponders in Equator achieved those responses in OLE study; in total, at week 52 of OLE, 33.6% of patients achieved MDA response and 55% achieved ACR50 response 
Neurocrine Biosciences Inc., of San Diego Crinecerfont (NBI-74788) Oral, nonsteroidal corticotropin-releasing factor type 1 receptor antagonist  Classic congenital adrenal hyperplasia Data demonstrated meaningful reductions in elevated adrenocorticotropic hormone and 17-hydroxyprogesterone levels (by 54% to 75%) at all doses studied, together with a dose-related decrease in androstenedione levels, ranging from 21% to 64%; at the highest dose, 75% of patients showed a response of at least 50% reduction from baseline for each of the 3 hormone markers at day 14 
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda Mavrilimumab Monoclonal antibody targeting granulocyte macrophage colony stimulating factor receptor alpha Severe COVID-19 pneumonia and hyperinflammation None of the 13 patients treated with mavrilimumab in phase II/III trial died compared to 27% of the 26 patients in the control group (p=0.086); 8% of mavrilimumab-treated patients progressed to mechanical ventilation compared to 35% of the control group (p=0.077); 100% of mavrilimumab-treated patients and 65% of control group patients attained clinical improvement (p=0.0001)
Relief Therapeutics Holding AG, of Geneva, and Neurorx Inc., of Radnor, Pa. RLF-100 (aviptadil) Vasoactive intestinal polypeptide Critical COVID-19 with respiratory failure Expanded study to include patients receiving high flow oxygen and noninvasive ventilation, in addition to those on ventilators
Phase III
Aimmune Therapeutics Inc., of Brisbane, Calif. Palforzia Peanut allergen Peanut allergy In the ARC004 study, after 2 years of daily treatment, more than 80% of patients had desensitization to 2,000 mg peanut protein; in the Artemis study, after 9 months, patients reported high mean total scale scores for effectiveness and satisfaction and moderate scores for convenience on the Treatment Satisfaction Questionnaire for Medication
Astellas Pharma Inc., of Tokyo Roxadustat Inhibitor of hypoxia-inducible factor prolyl-hydroxylase Anemia in nondialysis-dependent patients with stage 3–5 chronic kidney disease In the Dolomites study, roxadustat was noninferior to darbepoetin for a correction of hemoglobin levels during the first 24 weeks with 89.5% for roxadustat vs. 78% for darbepoetin; roxadustat was superior to darbepoetin for the decrease in low-density lipoprotein cholesterol with a least square mean difference of -0.403 mmol/L (p<0.01) and for time to first intravenous iron use with a hazard ratio of 0.45 (p=0.004)
Deciphera Pharmaceuticals LLC, of Waltham, Mass. Qinlock (ripretinib) Tyrosine kinase switch control inhibitor Fourth-line gastrointestinal stromal tumor Data from the Invictus study published in The Lancet Oncology showed Qinlock produced a progression-free survival of 6.3 months compared to 1 month for placebo (p<0.0001); median overall survival was 15.1 months for Qinlock compared to 6.6 months for placebo
Eli Lilly and Co., of Indianapolis LY-900014 Ultra-rapid formulation of insulin lispro Type 2 diabetes Enrollment in study comparing drug to Humalog suspended due to COVID-19
Eli Lilly and Co., of Indianapolis Taltz (ixekizumab) Monoclonal antibody targeting interleukin 17A Active psoriatic arthritis In the Spirit-Head-to-Head study, 49% of patients taking Taltz achieved minimal disease activity (MDA) compared to 33% of patients taking Humira (adalimumab); 47% of patients taking Taltz plus methotrexate achieved MDA compared to 47% of patients taking Humira plus methotrexate; 47% of patients taking Taltz plus a conventional synthetic disease-modifying antirheumatic drug (csDMARD) achieved MDA compared to 44% of patients taking Humira plus a csDMARD
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Takhzyro (lanadelumab) Monoclonal antibody targeting kallikrein Hereditary angioedema In the open-label extension of the Help study, mean monthly attack rates was 0.18 compared to 0.26 at the end of the original study
Phase IV
Alexion Pharmaceuticals Inc., of Boston Strensiq (asfotase alfa) Recombinant glycoprotein Hypophosphatasia Study withdrawn after portfolio review and in light of COVID-19 pandemic

Notes

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

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