Company Product Description Indication Phase III status


Aslan Pharmaceuticals Pte. Ltd., of Singapore Varlitinib Pan-HER inhibitor Biliary tract cancer Pivotal TreeTopp study missed co-primary endpoints; median progression-free survival was 2.83 months for varlitinib in combination with capecitabine vs. 2.79 for control; overall response rate was 9.4% for varlitinib vs. 4.8% for control
Aveo Oncology Inc., of Cambridge, Mass. Tivozanib VEGF tyrosine kinase inhibitor Refractory advanced renal cell carcinoma Updated data from Tivo-3 study showed, as of Aug. 15, 2019, data cutoff date, median overall survival was 16.4 months for tivozanib and 19.7 months for sorafenib; as of second data cutoff date, 20 patients remained progression-free on tivozanib arm vs. 2 on sorafenib, with median duration on study of 32.5 months
Bristol-Myers Squibb Co., of New York Yervoy (ipilimumab) Monoclonal antibody targeting CTLA4 Adjuvant treatment after complete surgical removal of stage IIIb/c/d or stage IV melanoma Study comparing Opdivo (nivolumab) plus Yervoy to Opdivo alone didn’t meet the primary endpoint of improved recurrence-free survival (RFS) in patients whose tumors expressed PD-L1 <1%; data monitoring committee recommended the study continue to asses the other co-primary endpoint of RFS in the all-comer population
Delta-Fly Pharma Inc., of Tokushima, Japan DFP-10917 Nucleoside analogue Acute myelogenous leukemia First participants registered in U.S. study enrolling individuals with relapsed/refractory disease
Diffusion Pharmaceuticals Inc., of Charlottesville, Va. TSC Trans sodium crocetinate Inoperable glioblastoma In the 19-patient lead-in portion of the Intact study, 6 of 7 patients who received the high dose TSC treatment are still alive; median survival currently 14.3 months
Epicentrx Inc., of La Jolla, Calif. RRx-001 Small-molecule immunotherapy targeting CD47–SIRP? Small-cell lung cancer Dosed first patient in Replatinum trial testing third-line and beyond SCLC; primary endpoint is progression-free survival
Erytech Pharma SA, of Lyon, France Eryaspase L-asparaginase encapsulated in donor-derived red blood cells Second-line pancreatic cancer Ongoing TRYbeCA1 study opened first U.S. site; review of first 150 patients by the independent data monitoring committee found no safety issues and committee recommended continuing the study as planned
Genentech Inc., of South San Francisco, a member of the Roche Group Tecentriq (atezolizumab) PD-L1 inhibitor Hepatocellular carcinoma Results from Imbrave 150 study in patients with unresectable disease who have not received prior systemic therapy showed combination with Avastin (bevacizumab) reduced risk of death by 42% (p=0.0006) and reduced risk of disease worsening or death by 41% (p=0.0001) vs. sorafenib
Morphosys AG, of Planegg, Germany Tafasitamab B-lymphocyte antigen CD19 modulator Diffuse large B-cell lymphoma B-Mind study of tafasitamab with rituximab plus bendamustine passed preplanned, event-driven interim analysis for futility, and independent data monitoring committee recommended increasing the number of participants with relapsed/refractory disease from 330 to 450
Myovant Sciences Ltd., of Basel, Switzerland Relugolix GnRH receptor antagonist Advanced prostate cancer Hero study met primary efficacy endpoint, with 96.7% of participants achieving sustained testosterone suppression to castrate levels (< 50 ng/dL) through 48 weeks; study achieved all 6 key secondary endpoints, including superiority to leuprolide acetate on rapid suppression of testosterone and prostate-specific antigen, all with "p" values < 0.0001
OSE Immunotherapeutics SA, of Nantes, France, and Haliodx SASU, of Marseille, France Tedopi (OSE-2101) Neoepitope combination Non-small-cell lung cancer In ongoing Atalante 1 trial, companies conducting translational investigation of immune biomarkers to define profile of responders
Polynoma LLC, of San Diego, unit of CK Life Sciences International (Holdings) Inc. Seviprotimut-L Polyvalent, shed melanoma antigen vaccine Melanoma Interim analysis of subgroups in ongoing adaptive Mavis study suggested enhanced recurrence-free survival (RFS) for study drug among those with American Joint Committee on Cancer stage IIB/IIC melanoma and those under age 60; drug did not significantly enhance RFS across full population
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Libtayo (cemiplimab) PD-1 inhibitor Advanced non-small-cell lung cancer Updated program, with first trial having enrolled 90% of planned 700 patients and independent data monitoring committee recommending trial continuation after overall survival interim analysis based on 34% of anticipated events; from first 361 patients, confirmed objective response is currently 42% for Libtayo and 22% for chemotherapy; for second trial, part 1 is fully enrolled, with part 2 at about 20% enrollment
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Ninlaro (ixazomib) Proteasome inhibitor Multiple myeloma Tourmaline-MM4 trial met primary endpoint of progression-free survival as first-line maintenance therapy vs. placebo in adults diagnosed with MM not treated with stem cell transplantation
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Alunbrig (brigatinib) ALK inhibitor Advanced anaplastic lymphoma kinase-positive non-small-cell lung cancer After more than 2 years of follow-up in the Alta-1L study, Alunbrig reduced the risk of disease progression or death by 76% compared to Xalkori (crizotinib, Pfizer Inc.) in newly diagnosed patients whose disease had spread to the brain at time of enrollment; Alunbrig reduced the risk of disease progression or death in all patients by 57%
Tocagen Inc., of San Diego Toca 511 & Toca FC Vocimagene amiretrorepvec and extended-release formulation of 5-fluorocytosine Recurrent high-grade glioma Study didn’t meet its primary or secondary endpoints; in a preplanned subgroup analysis of 60 patients with second recurrence, Toca 511 and Toca FC produced a 57% risk reduction for death compared to standard of care (lomustine, temozolomide or bevacizumab) with a median overall survival of 21.82 months compared to 11.14 months
Company Product Description Indication Phase III status


Amarin Corp. plc, of Dublin Vascepa (icosapent ethyl) Apolipoprotein B antagonist; phospholipase A2 inhibitor Cardiovascular disease 3,146 participants randomized in U.S. in global Reduce-IT cardiovascular outcomes trial showed 31% relative risk reduction and 6.5% absolute risk reduction in first occurrence of 5-point major adverse cardiovascular events; all prespecified hierarchical primary and secondary endpoints were reduced in U.S. subgroup, including myocardial infarction (8.8% to 6.7%, p=0.01), cardiovascular death (6.7% to 4.7%, p=0.007), stroke (4.1% to 2.6%, p=0.02) and all-cause mortality (9.8% to 7.2%, p=0.004)
Amarin Corp. plc, of Dublin Vascepa (icosapent ethyl) Apolipoprotein B antagonist; phospholipase A2 inhibitor Cardiovascular risk reduction Reduce-IT subgroup analysis showed risk reductions in U.S. patients treated with 4 g/day vs. placebo across prespecified composite and individual primary and secondary endpoints, including 31% relative risk reduction and 6.5% absolute risk reduction in first occurrence of 5-point major adverse cardiovascular events
Amarin Corp. plc, of Dublin Vascepa (icosapent ethyl) Apolipoprotein B antagonist; phospholipase A2 inhibitor Cardiovascular risk reduction At prespecified 9-month interim analysis, Evaporate study showed slowing of noncalcified plaque (sum of LAP, fibrofatty and fibrous plaque) vs. placebo (35% vs. 43%, p=0.010), total plaque (noncalcified + calcified plaque) (p=0.0004), fibrous plaque (15% vs. 26%, p=0.011) and calcified plaque (-1% vs. 9%, p=0.001), after adjustment by baseline plaque, age, sex, diabetes status, baseline triglyceride levels and statin use but no significant change in primary endpoint of low attenuation plaque vs. placebo (74% vs. 94%, p=0.469); investigator-initiated study continuing to full 18-month review
Astrazeneca plc, of Cambridge, U.K. Farxiga (dapagliflozin) SGLT2 inhibitor Reduced ejection fraction, with or without type 2 diabetes Analyses from Dapa-HF trial showed drug reduced risk of primary composite outcome of worsening heart failure, defined as hospitalization or an urgent visit, or death from cardiovascular causes vs. placebo, when added to standard of care
Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis Jardiance (empagliflozin) Sodium glucose transporter-2 inhibitor Cardiac failure Initiated 90-day Empulse superiority study in participants with and without type 2 diabetes to assess whether in-hospital administration of Jardiance 10 mg daily improves heart failure outcomes following stabilization in those hospitalized for any type of acute heart failure event; primary outcome is net clinical benefit (composite of all-cause mortality, number of heart failure events, time to first heart failure event and change from baseline in Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score) after 90 days of treatment
Caladrius Biosciences Inc., of Basking Ridge, N.J. CLBS-16 Autologous CD34+ cell therapy Coronary microvascular dysfunction Results from Escape-CMD trial
Merck & Co. Inc., of Kenilworth, N.J., and Bayer AG, of Leverkusen, Germany Vericiguat Soluble guanylate cyclase stimulator Chronic heart failure Victoria study to treat patients with worsening disease met the primary endpoint, reducing risk of the composite endpoint of heart failure hospitalization or cardiovascular death in patients with reduced ejection fraction vs. placebo when given in combination with available heart failure therapies
Mezzion Pharma Co. Ltd., of Seoul, South Korea Udenafil PDE5 inhibitor Congenital single ventricle heart disease In Fuel study that enrolled 400 adolescents with SVHD, those who took study drug over 6-month period showed improvement in exercise capacity measured by oxygen consumption at ventilatory anerobic threshold (VO2 at VAT); work rate at VAT and ventilatory equivalents of carbon dioxide at VAT also improved; peak VO2 improved but did not reach statistical significance
Novartis AG, of Basel, Switzerland Entresto (sacubitril/valsartan) Inhibits neprilysin and blocks angiotensin II type-I receptor Heart failure with preserved ejection fraction Subgroup analyses from Paragon-HF study show that, in specific subgroups, treatment may result in greater reductions in hospitalizations and cardiovascular death vs. valsartan; greater benefit seen in women with HFpEF and in HFpEF patients recently hospitalized for heart failure; in pooled analysis of Paragon-HF and Paradigm-HF trials, greater treatment benefit observed in patients with left ventricular ejection fraction below about 60%; data published in the Journal of the American College of Cardiology
Vifor Fresenius Medical Care Renal Pharma Ltd., of St. Gallen, Switzerland, and Chemocentryx Inc., of Mountain View, Calif. Avacopan Complement 5a receptor inhibitor Anti-neutrophil cytoplasmic antibody-associated vasculitis In 331-patient Advocate study, at week 26, 72.3% of patients treated with avacopan and 70.1% of patients treated with glucocorticoid standard of care were responders, defined as a Birmingham Vasculitis Activity Score of 0 and off glucocorticoid treatment for ANCA vasculitis for at least the preceding 4 weeks (p<0.0001 for noninferiority); at week 52, 65.7% of the avacopan-treated patients and 54.9% of glucocorticoid-treated patients had sustained remission (p=0.0066 for superiority of avacopan)
Company Product Description Indication Phase III status


Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. BCX-7353 (berotralstat dihydrochloride) Kallikrein inhibitor Hereditary angioedema 48-week results from APeX-S and APeX-2 trials showed participants who took 150 mg of oral, once-daily BCX-7353 achieved stable average attack rate of ? 1 per month; those switched from placebo to 150 mg of drug at week 24 saw mean attack rate drop to 0.5 per month at month 7 and to 0.4 attacks per month at month 12; those who completed 48 weeks on 150 mg (n=73) had median attack rate of 0 per month in 6 of 12 months, including month 12 (week 48)
Cara Therapeutics Inc., of Stamford, Conn. Difelikefalin (Korsuva) Opioid receptor kappa agonist Pruritus Results from pivotal Kalm-1 trial in individuals undergoing hemodialysis with moderate to severe chronic kidney disease-associated pruritus published in The New England Journal of Medicine; at week 12, 51.9% of those who received study drug achieved prespecified primary outcome of 3 point or > improvement in weekly mean of daily worst itch intensity numeric rating scale vs. 30.9% for placebo; additional analysis conducted for NEJM showed imputed proportion of patients who achieved primary outcome was 49.1% for difelikefalin compared to 27.9% for placebo (p<0.001) at week 12
Foamix Pharmaceuticals Ltd., Rehovot, Israel Amzeeq (minocycline) Tetracycline antibiotic topical foam Inflammatory lesions of non-nodular moderate to severe acne vulgaris Long-term safety data from 291 patients enrolled in studies 04 and 05 and treated for 52 weeks, published in the Journal of Clinical and Aesthetic Dermatology, showed application site adverse events occurred in less than 2% of patients during the 40-week extension; patient satisfaction was similar between week 12 and week 52
Revance Therapeutics Inc., of Newark, Calif. DaxibotulinumtoxinA Botulinum toxin Moderate and severe glabellar line Data published in Plastic and Reconstructive Surgery from the Sakura 1 and 2 studies showed half of the treated patients maintained none or only mild frown lines for at least 24 weeks; frown lines for half of the treated patients didn’t return to pre-treatment severity for at least 26-28 weeks
Takeda Pharmaceutical Co. Ltd., Takhzyro (lanadelumab-flyo)
Plasma kallikrein inhibitor
Hereditary angioedema Open-label extension of Help study in individuals 12 and older showed safety profile was consistent with original findings; drug, dosed 300 mg every 2 weeks, reduced mean attack rate by 87% overall compared with baseline (n=212); overall reduction of 92.6% observed in rate of attacks requiring acute treatment (n=212) and 83.6% in rate of moderate/severe attacks vs. baseline (n=212)
UCB SA, of Brussels Bimekizumab IgG1 antibody targeting IL-17A and IL-17F Moderate to severe plaque psoriasis Be Ready trial met co-primary endpoints of at least 90% improvement in Psoriasis Area and Severity Index (PASI 90) and Investigator Global Assessment (IGA) response of clear or almost clear (IGA 0/1) at week 16 vs. placebo; among key secondary endpoints, drug was statistically superior to placebo in achieving total skin clearance (PASI 100) at week 16, and was statistically superior to placebo in patient-reported reductions in itch, pain and scaling, as well as clear or almost clear scalp at week 16
Company Product Description Indication Phase III status


Acasti Pharma Inc., of Laval, Quebec Capre Omega-3 phospholipid derived from krill oil Hypertriglyceridemia Final participant completed final visit in pivotal Trilogy 1 trial, with top-line results expected as planned in December
Allena Pharmaceuticals Inc., of Newton, Mass. Reloxaliase Recombinant oxalate-degrading enzyme Enteric hyperoxaluria In the 115-patient Urirox-1 study, reloxaliase produced a mean reduction of 22.6% in average 24-hour urinary oxalate excretion measured during weeks 1-4, compared to 9.7% for placebo (p=0.004); 48.3% of patients taking reloxaliase had a reduction of 20% or more, compared to 31.6% of patients on placebo (p=0.06); in bariatric surgery patients, mean reduction was 21.2% for reloxaliase and 6% for placebo (p=0.01), and 50% of patients on reloxaliase met the 20% reduction goal compared to 28.9% of patients on placebo (p=0.036)
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. Lumasiran Hydroxyacid oxidase 1 modulator
Initiated Illuminate-C study expected to enroll about 16 individuals with documented diagnosis of primary hyperoxaluria type 1; primary endpoint is percentage change in plasma oxalate from baseline to 6 months, with data expected late next year
Daiichi Sankyo Europe GmbH, of Munich, Germany Bempedoic acid Once-daily ATP citrate lyase inhibitor Diabetes; hypercholesterolemia 2 pooled analyses from 4 studies showed bempedoic acid reduced HbA1c by 0.19% vs. placebo in in diabetes patients at 12 weeks; significantly lowered LDL-C in patients with hypercholesterolemia when added to maximally tolerated statin therapy, reducing by 18% vs. placebo, and by 25% vs. placebo in patients not on statin background therapy
Esperion Inc., of Ann Arbor, Mich. Bempedoic acid ATP citrate lyase inhibitor Elevated low-density lipoprotein cholesterol Results from Clear Wisdom study published in the Journal of the American Medical Association in patients with atherosclerotic cardiovascular disease and/or heterozygous familial hypercholesterolemia inadequately controlled with current lipid-modifying therapies showed significantly lowered LDL-C by 17% on background maximally tolerated statin therapy at 12 weeks; effect durable through 52-weeks; marketing applications under review in the U.S. and Europe
Hua Medicine Ltd., of Shanghai Dorzagliatin (HMS-5552) Dual-acting glucokinase activator Type 2 diabetes In the HMM0301 study, dorzagliatin produced a 1.07% HbA1c reduction from a baseline of 8.35% at 24 weeks compared to a reduction of 0.50% from a baseline of 8.37% for placebo (p<0.0001); 45.4% of patients taking dorzagliatin had an HbA1c of less than 7% compared to 21.5% of patients taking placebo (p<0.0001)
Poxel SA, of Lyon, France Imeglimin Targets mitochondrial bioenergetics Type 2 diabetes In 208-patient open-label extension of Times3 study, imeglimin and insulin reduced HbA1c by 0.64% vs. baseline over 52 weeks; patients who took placebo and insulin for the first 16 weeks and imeglimin and insulin for the final 36 weeks had a 0.54% reduction in HbA1c from baseline
Sanofi SA, of Paris Toujeo (insulin glargine) Long-acting insulin Type 1 diabetes Toujeo was noninferior compared to insulin glargine 100 units/mL (Gla-100)in children and adolescents, with both drugs producing a 0.4% mean reduction of HbA1c; drugs produced comparable number of hypoglycemia events; 6% of patients taking Toujeo had 1 or more severe hypoglycemia events compared to 8.8% of patients taking Gla-100; 8.2% of patients taking Toujeo had 1 or more hyperglycemia with ketosis events compared to 11.4% of patients taking Gla-100
The Medicines Co., of Parsippany, N.J. Inclisiran Cholesterol-lowering siRNA candidate Heterozygous familial hypercholesterolemia Results from Orion 9 met all primary and secondary efficacy endpoints; for primary endpoints, inclisiran delivered placebo-adjusted LDL-C reductions of 50% (71 mg/dL, p<0.0001) at day 510 and demonstrated time-averaged placebo-adjusted LDL-C reductions of 45% (63 mg/dL, p<0.0001) from days 90 through 540
Company Product Description Indication Phase III status


Cymabay Therapeutics Inc., of Newark, Calif. Seladelpar PPAR delta agonist Primary biliary cholangitis Achieved targeted 240 patient enrollment goal in the Enhance study; on track to complete full enrollment by the end of November
Everest Medicine Inc., of New York Etrasimod Oral sphingosine 1-phosphate receptor modulator Moderately to severely active ulcerative colitis Dosed first patient in trial assessing etrasimod for induction and maintenance therapy; about 330 patients will be enrolled in mainland China, South Korea and Taiwan
Genfit SA, of Lille, France Elafibranor Dual PPAR alpha/delta agonist Nonalcoholic steatohepatitis Data safety monitoring board recommended continuing Resolve-IT study without modification; top-line interim results expected in first quarter 2020
Intercept Pharmaceuticals Inc., of New York Obeticholic acid (OCA) Farnesoid X receptor agonist Liver fibrosis OCA-treated patients in the primary ITT group in ongoing Regenerate study showed time- and dose-dependent improvements vs. placebo across noninvasive tests, including blood tests of fibrosis, as early as 3 months after treatment initiation; vibration-controlled transient elastography, an imaging assessment of liver stiffness and surrogate of fibrosis, decreased from baseline in both OCA groups but increased with placebo at 18 months
Redhill Biopharma Ltd., of Tel Aviv, Israel RHB-102 (Bekinda) Oral, bimodal-release, once-daily pill formulation of ondansetron Acute gastroenteritis Data published in the Journal of the American Medical Association show Guard study met primary endpoint, prevention of further vomiting for at least 24 hours among adolescents and adults with gastroenteritis-related emesis without usage of intravenous hydration or rescue medication
Company Product Description Indication Phase III status

Genitourinary/sexual function

Exelixis Inc., of Alameda, Calif. Esaxerenone Mineralocorticoid receptor antagonist Diabetic nephropathy Partner Daiichi Sankyo Co. Ltd. reported that comparison study in 455 participants with incipient disease showed drug resulted in higher urinary albumin-to-creatinine ratio (UACR) remission rate vs. placebo (22.1% vs. 4%); treatment reduced UACR and was associated with reduction in progression from incipient to overt disease, both vs. placebo (-58.3% vs. +8.3% and 1.4% vs. 7.5%, respectively)
Janssen Pharmaceuticals Cos., of Beerse, Belgium, part of Johnson & Johnson Invokana (canagliflozin) SGLT2 inhibitor Kidney disease Analysis from Credence study found Invokana consistently reduced risk of renal and cardiovascular events in patients with various levels of kidney function, or estimated glomerular filtration rates (eGFR); greater renal absolute benefits were observed in those with most advanced renal insufficiency (eGFR <60 mL/min/1.73 m2)
Mallinckrodt plc, of Staines-Upon-Thames, U.K. Terlipressin Vasopressin analogue Hepatorenal syndrome type 1 Results from pivotal Confirm study show it met the primary endpoint of Verified HRS Reversal (VHRSR), defined as renal function improvement, avoidance of dialysis and short-term survival; 29.1% (58/199) in terlipressin-plus-albumin group achieved Verified HRS Reversal vs. 15.8% (16/101) on placebo plus albumin (p=0.012)
Obseva SA, of Geneva, Switzerland Nolasiban Oxytocin receptor antagonist Embryo transfer following in-vitro fertilization In the Implant 4 study, ongoing pregnancy rate at 10 weeks was 40.5% for nolasiban and 39.1% for placebo (p=0.745); company is discontinuing the in-vitro fertilization program
Reata Pharmaceuticals Inc., of Plano, Texas Bardoxolone Activator of Nrf2 Chronic kidney disease caused by Alport syndrome In the Cardinal study, after 48 weeks of treatment, bardoxolone improved mean estimated glomerular filtration rate (eGFR) by 9.50 mL/min/1.73 m2 compared to placebo (p<0.0001); following a 4-week withdrawal after the treatment, patients treated with bardoxolone had a mean retained eGFR of 5.14 mL/min/1.73 m2 compared to placebo (p=0.0012)
Company Product Description Indication Phase III status


Akebia Therapeutics Inc., of Cambridge, Mass. Vadadustat HIF prolyl hydroxylase inhibitor Anemia As reported by partner Mitsubishi Tanabe Pharma Corp., of Osaka, Japan, 52-week data from correction and conversion pivotal study in 304 Japanese individuals with nondialysis-dependent chronic kidney disease (CKD) showed mean hemoglobin (Hb) level of 11.51 g/dL for drug vs. 11.58 g/dL for those who received darbepoetin alfa; 52-week data from conversion study in 323 Japanese hemodialysis participants with anemia due to CKD showed mean Hb level of 10.39 g/dL for vadadustat vs. 10.62 g/dL for darbepoetin alfa
Astrazeneca plc, of Cambridge, U.K., and Fibrogen Inc., of San Francisco Roxadustat HIF prolyl
Anemia in chronic
kidney disease
Pooled analyses from phase III program showed, in nondialysis-dependent patients who received drug, risk of MACE, MACE+ and all-cause mortality was comparable to placebo; dialysis-dependent patients who received roxadustat had lower risk of MACE+ and no increased risk of MACE or all-cause mortality vs. epoetin alfa; in incident dialysis patients, those who received roxadustat had 30% lower risk of MACE and 34% lower risk of MACE+ vs. those who received epoetin alfa, with trend toward lower all-cause mortality for study drug
Fibrogen Inc., of San Francisco Roxadustat HIF prolyl hydroxylase inhibitor Anemia in chronic kidney disease Himalayas trial that enrolled 1,043 anemia in incident dialysis participants with CKD achieved co-primary endpoints of noninferiority in mean hemoglobin (Hb) change from baseline, with mean Hb increase from 8.4 g/dL to 11 g/dL in treatment arm vs. 8.4 g/dL to 10.8 g/dL for epoetin alfa, and proportion of patients achieving Hb response during first 24 weeks, at 88.2% vs. 84.4%
Fibrogen Inc., of San Francisco Roxadustat Oral inhibitor of HIF prolyl hydroxylase activity Anemia in chronic kidney disease Pooled analysis from 6 pivotal studies showed superiority to placebo in non-dialysis-dependent patients, showing improvement of 1.85 g/dL in Hb levels in baseline to average over 28-52 weeks s. 0.14 g/dL for placebo, for overall different of 1.72 g/dL (p<0.001); pooled analysis in dialysis patients showed superiority vs. epoetin alfa, with improvement of 1.22 g/dL in Hb levels from baseline to over 28-52 weeks vs. 0.99 g/dL, for overall treatment difference of 0.23 g/dL (p<0.0001); across both populations, effectively raised Hb levels regardless of iron depletion at baseline
Sierra Oncology Inc., of Vancouver, British Columbia Momelotinib JAK1, JAK2 and ACVR1 inhibitor Myelofibrosis Launched Momentum study in 180 patients who have been treated previously with a JAK inhibitor; primary endpoint is Total Symptom Score response rate of momelotinib compared to danazol at week 24; top-line data expected in fourth quarter of 2021
Company Product Description Indication Phase III status


ALK-Abello A/S, of Copenhagen Ragwitek Sublingual allergy immunotherapy Seasonal allergic rhinitis Study by Merck & Co. Inc. that randomized 1,025 children, ages 5 to 17, met primary endpoint of average total combined score (TCS) - sum of rhinoconjunctivitis daily symptom score (DSS) and rhinoconjunctivitis daily medication score (DMS) - over peak ragweed season and key secondary endpoint of average TCS during entire season, with relative improvements in TCS vs. placebo of ?38.3% (p<0.001) during peak season and ?32.4% (p<0.001) during entire season
Allergy Therapeutics plc, of Worthing, U.K.
TLR4 agonist Allergy Upcoming Grass study will take stepwise approach, with 2 stages covering both 2020/21 and 2021/22 pollen seasons rather than single large trial originally planned for 2020/21 season
Biogen Inc., of Cambridge, Mass. Vumerity (diroximel fumarate) Monomethyl fumarate prodrug Multiple sclerosis Evolve-MS-2 study found improved patient-assessed gastrointestinal tolerability of Vumerity vs. Tecfidera (dimethyl fumarate)
Eli Lilly and Co., of Indianapolis Taltz (ixekizumab) Monoclonal antibody targeting interleukin 17A Psoriatic arthritis In Spirit-H2H study of 566 biologic-naïve patients, at week 52, 39% of patients treated with Taltz achieved both a 50% or more reduction in American College of Rheumatology score and Psoriasis Area and Severity Index 100, compared with 26% of patients treated with Humira (adalimumab, Abbvie Inc.)
Gilead Sciences Inc., of Foster City, Calif., and Galapagos NV, of Mechelen, Belgium Filgotinib Oral JAK1 inhibitor Moderately to severely active rheumatoid arthritis Subgroup analysis from Finch 2 trial in patients who previously had inadequate response to biologic DMARD therapy (DMARD-IR) showed both doses improved clinical outcomes vs. placebo, regardless of number and mechanism of action of prior bDMARDs; after 12 weeks of treatment, 70.3% treated with 200 mg and 58.2% treated with 100 mg achieved ACR20 score vs. 25.7% on placebo; 68% of bDMARD-IR patients in 200-mg group and 51.2% in 100-mg group who had been treated with more than 1 biologic MOA achieved ACR20 response vs. placebo (27.3%)
Janssen Pharmaceuticals Cos., of Beerse, Belgium, part of Johnson & Johnson Tremfya (guselkumab) Monoclonal antibody against p19 subunit of IL-23 Active psoriatic arthritis 24-week data showed significant greater proportion of patients on Tremfya achieved at least 20% improvement in disease signs and symptoms (ACR20) vs. placebo; Discover-1 trial study showed 59% treated every 4 weeks (q4w) and 52% treated at weeks 0, 4 and every 8 weeks thereafter (q8w) achieved ACR20 vs. 22% on placebo (both p<0.001); Discover-2 showed 64% biologic-naïve patients receiving q4w or q8w achieved ACR vs. 33% on placebo (both p<0.001)
Marinomed Biotech AG, of Vienna Budesolv (budesonide) Synthetic steroid Hay fever In a 75-patient study, Budesolv reduced the total nasal symptom score by 1.2 points after 4 hours; Rhinocort Aqua showed no therapeutic effect at 4 hours; after 8 days drugs had comparable efficacy
Pfizer Inc., of New York Xeljanz (tofacitinib) Janus kinase inhibitor Juvenile idiopathic arthritis The 225-patient A3921104 study met its primary endpoint with the occurrence of disease flare in patients treated with tofacitinib being significantly lower than in patients treated with placebo at week 44
Company Product Description Indication Phase III status


Bavarian Nordic A/S, of Copenhagen MVA-BN smallpox vaccine Live attenuated modified vaccinia Ankara Monkeypox virus infection; Variola virus infection Completed enrollment of lot-consistency trial to support U.S. licensure of longer-lasting, freeze-dried formulation of MVA-BN smallpox vaccine, approved by FDA as Jynneos in liquid-frozen formulation; results expected in 2021
Bavarian Nordic A/S, of Copenhagen, Denmark Jynneos (MVA-BN) Smallpox vaccine Smallpox infection prophylaxis Data published in The New England Journal of Medicine showed Jynneos induced neutralizing antibody titers comparable with ACAM2000 at day 14; peak neutralizing antibodies induced by Jynneos were almost 2-fold higher on average than those stimulated by ACAM2000
Biondvax Pharmaceuticals Ltd., of Jerusalem M-001 Multimeric, multiepitope, single-protein, universal flu vaccine Influenza virus infection Full enrollment and randomization of 12,463 participants reached in pivotal trial, with results due by year-end 2020
Gilead Sciences Inc., of Foster City, Calif. Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg) HIV-1 integrase inhibitor; nucleoside reverse transcriptase inhibitor HIV-1 infection Data from 2 144-week studies showed noninferiority vs. dolutegravir/abacavir/lamivudine (50/600/300 mg) or dolutegravir + emtricitabine/tenofovir alafenamide (50/200/25 mg) was maintained from primary endpoint measurements at week 48; similar proportion of Biktarvy group achieved virologic suppression (82%; n=518/634) vs. the other regimens (84%; n=265/315 and 84%; n=273/325, respectively)
Gilead Sciences Inc., of Foster City, Calif. Descovy (emtricitabine 200 mg + tenofovir alafenamide 25 mg) Nucleoside reverse transcriptase inhibitor HIV pre-exposure prophylaxis At 96 weeks, global registration trial confirmed noninferiority of drug, dosed once daily, vs. Truvada (emtricitabine 200 mg + tenofovir disoproxil fumarate 300 mg, Gilead); lumbar spine bone mineral density increased by 0.95% in Descovy group and decreased by 1.39% in Truvada group (p<0.001)
Gilead Sciences Inc., of Foster City, Calif. Vemlidy (tenofovir alafenamide) Nucleoside reverse transcriptase inhibitor Hepatitis B virus infection Long-term analysis of 2 studies that enrolled 1,632 individuals showed hepatocellular carcinoma was observed in 21 patients, including 1% of Vemlidy cohort vs. 1.9% treated with tenofovir disoproxil fumarate (Viread) across 3 to 5 years of follow-up
Gilead Sciences Inc., of Foster City, Calif. Vemlidy (tenofovir alafenamide) Nucleoside reverse transcriptase inhibitor Hepatitis B virus infection In analysis from study of those with virally suppressed chronic disease, 243 previously treated with tenofovir disoproxil fumarate (TDF, Viread) for median of 4 years and switched to Vemlidy for 48 weeks showed improvement in bone and renal markers regardless of duration (<4 years vs ? 4 years) of prior TDF use
Iterum Therapeutics plc, of Dublin Sulopenem Antibiotic Complicated urinary tract infections Completed patient enrollment in the Sure study; top-line results expected in early 2020
Mycovia Pharmaceuticals Inc., of Durham, N.C. VT-1161 Antifungal Recurrent vulvovaginal candidiasis Completed enrollment for both Violet studies; top-line data expected in second half of 2020
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. REGN-EB3 (atoltivimab + odesivimab + maftivimab) Viral structural protein inhibitor Ebola virus infection The New England Journal of Medicine published results, reported in August, from Palm trial in 681 patients during ongoing outbreak in Democratic Republic of the Congo showing triple-antibody cocktail outperformed Zmapp (Mapp Biopharmaceutical Inc., Defryus Inc.) control arm across multiple measures, including primary endpoint of mortality at day 28 (33.5% vs. 51.3%, p=0.002) and secondary endpoint of reduction in days until virus was undetectable in bloodstream
Scynexis Inc., of Jersey City, N.J. Ibrexafungerp Oral antifungal Vulvovaginal candidiasis Top-line results from Vanish-303 study showed superiority vs. placebo at statistically significant level (p?0.001) for primary endpoint of clinical cure, defined as complete resolution (score of 0) of all signs and symptoms at day 10 test-of-cure visit(50.5% for Ibrexafungerp); study also met key study endpoints required for regulatory approval; NDA submission anticipated in second half of 2020
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan TAK-003 Vaccine Dengue Data from pivotal Tides trial published in The New England Journal of Medicine showed protection against virologically confirmed dengue (VCD), the primary endpoint, in children, 4 to 16; vaccine efficacy (VE) was 80.2% (p<0.001) in 12-month period after second dose, which was administered 3 months after first dose; similar degrees of protection seen in individuals who had and had not been previously infected with dengue based on planned exploratory analyses of secondary endpoints (VE: 82.2% vs. VE: 74.9%, respectively); other exploratory analyses showed 95.4% reduction in dengue-associated hospitalizations
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan TAK-003 Dengue vaccine Dengue prophylaxis Updated 18-month data from the Tides study showed overall vaccine efficacy was 73.3% compared to 80.2% in the primary endpoint analysis at 12 months
Company Product Description Indication Phase III status


Horizon Therapeutics plc, of Dublin Krystexxa (pegloticase injection Pegylated uric acid specific enzyme Chronic gout Patient case series results showed that adding methotrexate to course of Krystexxa could help patients with disease refractory to conventional therapies; in 10 adults with uncontrolled gout who received Krystexxa during or with treatment of methotrexate, 8 were complete responders at 24 weeks
Taiwan Liposome Co., of Taipei, Taiwan TLC-599 Non-opioid Bioseizer sustained-release version of dexamethasone sodium phosphate Osteoarthritis knee pain First patient enrolled in Excellence pivotal trial; study to test single and repeat dosing in about 500 patients; primary endpoint is magnitude of pain relief by WOMAC Pain score vs. placebo at weeks 16 and 40
Company Product Description Indication Phase III status


Eli Lilly and Co., of Indianapolis Taltz (ixekizumab) Monoclonal antibody targeting interleukin 17A Non-radiographic axial spondyloarthritis In Coast-X study of 303 patients who were biologic disease-modifying anti-rheumatic drug-naïve, at week 16, 35% and 40% of patients taking Taltz every 4 weeks or every 2 weeks, respectively, achieved Assessment of Spondyloarthritis International Society 40 (ASAS40) response, compared to 19% of patients treated with placebo; at week 52, ASAS40 response rates were 30%, 31% and 13% for Taltz every 4 weeks, every 2 weeks and placebo, respectively (p<0.01 for all Taltz to placebo comparisons)
Eli Lilly and Co., of Indianapolis Taltz (ixekizumab) IL-17 antagonist Axial spondylarthritis In Coast-X study in 303 adults with non-radiographic disease, at week 16 35% who received drug every 4 weeks and 40% who received it every 2 weeks achieved ASAS40 response vs. 19% who received placebo; at week 52, 30% treated with drug every 4 weeks and 31% treated every 2 weeks achieved ASAS40 response vs. 13% for placebo; Taltz also met major secondary endpoints at weeks 16 and 52
Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J. Edaravone (MT-1186) Oral suspension formulation of neuroprotective agent Amyotrophic lateral sclerosis Started study testing long-term safety and tolerability
Novartis AG, of Basel, Switzerland Cosentyx (secukinumab) IL-17 antagonist Psoriatic arthritis Narrowly missed statistical significance for superiority in ACR 20, the primary endpoint of the Exceed trial, while showing numerically higher results vs. Humira (adalimumab)
Novartis AG, of Basel, Switzerland Cosentyx (secukinumab) Monoclonal antibody targeting interleukin 17A Non-radiographic axial spondyloarthritis In the Prevent study, 42.2% of patients treated with Cosentyx achieved Assessment of Spondyloarthritis International Society 40 compared to 29.2% of patients taking placebo (p<0.05)
Santhera Pharmaceuticals AG, of Pratteln, Switzerland Idebenone Oxidoreductase inhibitor Duchenne muscular dystrophy Syros data showed long-term efficacy in slowing respiratory function loss in individuals with DMD under routine clinical care; published in Neuromuscular Disorders
Tonix Pharmaceuticals Holding Corp. TNX-102 SL Low-dose sublingual formulation of cyclobenzaprine hydrochloride Fibromyalgia Initiated Relief, a potential pivotal study of a 5.6-mg dose
Company Product Description Indication Phase III status


Acadia Pharmaceuticals Inc., of San Diego Pimavanserin (Nuplazid) 5-HT 2a receptor inverse agonist Schizophrenia In Advance study in 403 participants, drug showed statistically significant improvement on primary endpoint of change from baseline to week 26 on Negative Symptom Assessment-16 (NSA-16) total score vs. placebo (-10.4 vs. -8.5; p=0.043; effect size = 0.21); greater improvement in NSA-16 total score vs. placebo seen in 53.8% of those (n=107) who received highest dose of 34 mg (-11.6 vs. -8.5; unadjusted p=0.0065, effect size = 0.34); pimavanserin did not separate from placebo on key secondary endpoint of Personal and Social Performance scale; second pivotal study with 34-mg dose expected to begin in first half of 2020
Acelrx Pharmaceuticals Inc., of Redwood City, Calif. Sufentanil sublingual tablet 30 mcg (Dsuvia)
Opioid receptor mu agonist
Pain Pooled dosing and efficacy data among multiple demographic subgroups, published in Journal of PeriAnesthesia Nursing, showed 3.9 as mean number of doses administered from 0-12 hours, dosing was less frequent for those ?65 years compared to younger patients but similar among other subgroups; summed pain intensity difference to baseline over 12 hours was higher for study drug compared with placebo across subgroups
Akcea Therapeutics Inc., of Boston, and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. AKCEA-TTR-Lrx Antisense targeting transthyretin Polyneuropathy caused by hereditary TTR amyloidosis Started Neuro-TTRansform study of 140 patients; 20 patients will start on Tegsedi (inotersen) for 35 weeks; the co-primary efficacy endpoints at week 66, percent change from baseline in serum TTR concentration, change from baseline in the modified Neuropathy Impairment Score +7 and change from baseline in Norfolk Quality of Life Questionnaire-Diabetic Neuropathy, will be compared to placebo group from the NEURO-TTR study of Tegsedi
Allergan plc, of Dublin Ubrogepant Oral CGRP receptor antagonist Acute migraine Achieve II results published in The Journal of the American Medical Association showed treatment vs. placebo led to significantly greater rates of pain freedom at 2 hours with both 50-mg and 25-mg doses, and freedom from most bothersome migraine-associated symptom at 2 hours with 50-mg dose
Avadel Pharmaceuticals plc, of Dublin FT-218 Controlled-release sodium oxybate Excessive daytime sleepiness and cataplexy in patients with narcolepsy Reached target enrollment of 205 patients in the Rest-On study; top-line data expected in the second quarter of 2020
Chugai Pharmaceutical Co. Ltd., of Tokyo Satralizumab IL-6 receptor antagonist Neuromyelitis optica spectrum disorders Findings from SakuraSky study, reported in May, were published online in The New England Journal of Medicine
Flexion Therapeutics Inc., of Burlington, Mass. Zilretta (triamcinolone acetonide extended-release injectable suspension) Activates glucocorticoid receptor Moderate to severe osteoarthritis pain Results from sensitivity analysis testing single administration showed significant improvement (p<0.05) in Average Daily Pain (ADP) scores vs. immediate-release triamcinolone acetonide in crystalline suspension (TAcs) (weeks 5-19) and placebo (weeks 1-20); proportion of patients reporting no knee pain (ADP score=0) at week 12 was higher with Zilretta (~28%) vs. TAcs (~8%)
Genentech, of South San Francisco, part of the Roche Group, and PTC Therapeutics Inc., of South Plainfield, N.J. Risdiplam Survival motor neuron-2 splicing modifier Type 2 or 3 spinal muscular atrophy Pivotal part 2 of Sunfish trial in patients, ages 2-25, met primary endpoint of change from baseline in Motor Function Measure 32 scale after 1 year of treatment vs. placebo
Harmony Biosciences LLC, of Plymouth Meeting, Pa. Wakix (pitolisant) Histamine H3 receptor inverse agonist Narcolepsy; cataplexy Articles in Sleep describe long-term safety and efficacy of pitolisant on daytime sleepiness, cataplexy, hallucinations and sleep paralysis is confirmed and, separately, that pitolisant demonstrated significantly lower potential for abuse compared with phentermine and an overall profile similar to placebo
Neurocrine Biosciences Inc., of San Diego Ingrezza (valbenazine) Vesicular monoamine transporter 2 inhibitor Moderate or severe tardive dyskinesia Data from the Kinect 4 study published in the Journal of Clinical Psychopharmacology showed 90% and 89.2% of patients on 40 mg or 80 mg of Ingrezza, respectively, achieved ?50% improvement from baseline in the Abnormal Involuntary Movement Scale total score after 48 weeks of treatment; 89%-95% of patients achieved a Clinical Global Impression of Change-TD or Patient Global Impression of Change response of "much improved" or "very much improved"
Neurocrine Biosciences Inc., of San Diego Valbenazine Selective VMAT2 inhibitor Chorea associated with Huntington’s disease The Huntington Study Group initiated Kinect-HD study to enroll participants, ages 18 to 75
Pharmazz Inc., of Willowbrook, Ill. Sovateltide Endothelin-B receptor agonist Acute cerebral ischemic stroke Study has enrolled 7 of approximately 110 patients; top-line data expected by the middle of 2020
Revance Therapeutics Inc., of Newark, Calif. DaxibotulinumtoxinA for injection Stabilizing peptide excipient combined with highly purified botulinum toxin Isolated cervical dystonia Completed patient enrollment in pivotal Aspen-1 study; top-line data expected in second half of 2020
Supernus Pharmaceuticals Inc., of Rockville, Md. SPN-810 Dopamine D2 receptor antagonist Conduct disorder Parallel group trial for treatment of impulsive aggression in individuals 6 to 11 diagnosed with ADHD missed primary endpoint; drug, dosed at 36 mg, produced median reduction of 58.6% in average weekly frequency of impulsive aggression episodes from baseline, which was not statistically significant (p= 0.092) vs. placebo
Tonix Pharmaceuticals Holding Corp., of New York Tonmya (TNX-102 SL, cyclobenzaprine) Muscle relaxant Post traumatic stress disorder Following a meeting with the FDA, analysis of the primary endpoint for the Recovery study, the mean change from baseline in the Clinician-Administered PTSD Scale for DSM-5, was changed from week 4 to week 12; company plans to add an interim analysis that allows for a potential sample size adjustment or stopping for futility after 125 of the targeted 250 patients have completed or discontinued the 12-week treatment
Trevena Inc., of Chesterbrook, Pa. Oliceridine G protein biased mu-opioid receptor ligand Moderate to severe acute pain Data from the 768-patient, open-label Athena safety study published in The Journal of Pain Research showed adverse events (AEs) were mostly mild or moderate; most common AEs were nausea, vomiting, and constipation; 2% of patients discontinued treatment due to an AE
Company Product Description Indication Phase III status


Biogen Inc., of Cambridge, Mass. Timrepigene emparvovec NSR AAV-Rab-escort protein 1 gene therapy Choroideremia Enrolled last patient in STAR trial
Eyegate Pharmaceuticals Inc., of Waltham, Mass. Ocular bandage gel Cross-linked thiolated carboxymethyl hyaluronic acid Corneal wound repair Proved superior to standard of care in healing corneal wounds following photorefractive keratectomy surgery
Iveric Bio Inc., of New York Zimura (avacincaptad pegol) Complement C5 inhibitor Geographic atrophy secondary to dry age-related macular degeneration Starting second pivotal study with plans to begin enrollment in the first quarter of 2020
Omeros Corp., of Seattle Omidria (phenylephrine/ketorolac intraocular solution) Vasoconstrictor and a nonsteroidal anti-inflammatory drug Cataract surgery or intraocular lens replacement Data published in Clinical Ophthalmology showed 9.8% of 41 patients given Omidria required fentanyl compared to 42.1% of 19 patients given epinephrine (p=0.006); mean visual analogue scale (VAS) pain score was 2.3 for Omidria and 4.5 for epinephrine (p<0.0001); 85% of patients had a VAS score of 3 or less compared to 31.6% for epinephrine (p<0.0001)
Company Product Description Indication Phase III status


Gelesis Inc., of Boston Plenity (Gelesis100) Oral, nonsystemic, superabsorbent hydrogel Obesity Results of post-hoc analysis from Glow trial showed twice as many adults (11%) lost enough weight to achieve a BMI of 27 or less with Plenity vs. placebo (5%)
Rhythm Pharmaceuticals Inc., of Boston Setmelanotide MC4R agonist Pro-opiomelanocortin deficiency obesity and leptin receptor deficiency obesity Data showed treatment associated with reductions in BMI and BMI z-scores for patients with both POMC and LEPR deficiency obesity treated for over 1 year at therapeutic dose
Company Product Description Indication Phase III status


Restorbio Inc., of Boston RTB-101 Oral TORC1 inhibitor Prevention of clinically symptomatic respiratory illness Top-line data from Protector 1 study in adults, 65 and older, did not meet its primary endpoint; company stopped development in CSRI

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