Celltrion Healthcare Co. Ltd., a unit of Celltrion Inc., of Incheon, South Korea

CT-P13 (biosimilar infliximab)

TNF alpha ligand inhibitor and binding agent

Inflammatory bowel disease

Pivotal study showed that subcutaneous (SC) formulation of CT-P13, marketed in U.S. as Inflectra and in EU as Remsima, was noninferior to intravenous (I.V.) formulation of the drug in pharmacokinetics at week 22, meeting primary endpoint; combined clinical remission rates for Crohn's disease and ulcerative colitis patients at week 30 also were comparable between I.V. and SC (66.7%, 44 of 66 in SC arm and 54.7%, 35 of 64 in I.V., p=0.1620)

Taiwan Liposome Co., of Taipei, Taiwan

TLC-590

Non-opioid, Bioseizer sustained-release formulation of ropivacaine

Postsurgical pain

Phase I/II data showed more immediate and long-lasting pain reduction vs. ropivacaine following inguinal hernia repair; each dose group reduced more pain than ropivacaine group, both with movement and at rest; pain reduction maintained through 168 hours; TLC-590 also reduced or eliminated use of opioids, with 58.3% of patients in 475-mg group not using any opioids throughout study; mean total opioid consumption was 54% less than ropivacaine group through 96 hours post-surgery

Axsome Therapeutics Inc., of New York

AXS-12

Norepinephrine reuptake inhibitor

Narcolepsy

Enrollment completed in Concert trial; top-line data due by year-end

Follicum AB, of Lund, Sweden

FOL-005

Osteopontin ligand modulator

Alopecia

Supplementary toxicology studies completed to form basis of phase IIa study with topical formulation of FOL-005, expected to begin early next year

Hemostemix Inc., of Calgary, Alberta

ACP-01

Blood-derived autologous stem cell therapy

Critical limb ischemia

Long-term follow-up from first 12 participants randomized 2-to-1 to ACP-01 or placebo and followed for at least 1 year following treatment showed healing of ulcers and resolution of ischemic rest pain in 10 of 12; outcomes maintained for up to 4.5 years; following recent review, data safety monitoring board recommended that trial, with 46 of planned 95 participants enrolled, continue as planned

Indalo Therapeutics Inc., of Cambridge, Mass.

IDL-2965

Integrin antagonist

Idiopathic pulmonary fibrosis

First participant dosed in third part of adaptive trial; following single and multiple ascending-dose (MAD) studies in healthy volunteers, MAD study in IPF patients is measuring effect on safety, pharmacokinetics and biomarkers over 28 days

Global Blood Therapeutics Inc., of South San Francisco

Voxelotor

Hemoglobin alpha subunit modulator

Sickle cell disease

Post-hoc analysis of data from 17 participants with SCD and leg ulcers enrolled in Hope trial showed all 4 treated with voxelotor 1,500 mg had their leg ulcers resolve or improve, and no participants who received study drug at 1,500 mg reported new leg ulcers after starting treatment; among 6 with leg ulcers at start of treatment with voxelotor 900 mg, 3 had leg ulcers resolve, 1 showed improvement from moderate to mild and 2 showed no change; 2 developed leg ulcers during the study; no participants treated with placebo showed improvement in their leg ulcers

Janssen Pharmaceutical Cos., unit of Johnson & Johnson, of New Brunswick, N.J.

Stelara (ustekinumab)

Dual IL-12/IL-23 receptor antagonist

Ulcerative colitis

2-year data from long-term extension of Unifi study showed that, of 399 participants in clinical response 8 weeks after receiving single intravenous (I.V.) induction dose and randomized to subcutaneous (SC) 90-mg injections every 8 or 12 weeks or to placebo who entered long-term extension, 83% to 91% of those who received Stelara SC every 8 weeks and 81% to 90% of those who received study drug every 12 weeks remained in symptomatic remission between weeks 44 to 92; among those who achieved clinical remission at maintenance baseline, 69% who received SC injections every 8 weeks and 80% every 12 weeks maintained symptomatic remission at both weeks 44 and 92

Myovant Sciences Inc., of Brisbane, Calif.

Relugolix

GNRH receptor antagonist

Endometriosis

Recruitment completed for Spirit 1 study evaluating combination therapy with estradiol 1 mg and norethindrone acetate 0.5 mg to treat endometriosis-associated pain; top-line data expected in second quarter of 2020

Odonate Therapeutics Inc., of San Diego

Tesetaxel

Taxane derivative

Metastatic breast cancer

Enrollment completed in multinational, randomized Contessa trial; top-line data expected in third quarter of 2020

Opko Health Inc., of Miami, and Pfizer Inc., of New York

Somatrogon

Growth hormone ligand

Growth hormone deficiency

Trial evaluating somatrogon dosed weekly in pre-pubertal children met primary endpoint of noninferiority to daily Genotropin (somatropin, Pfizer), measured by annual height velocity at 12 months

Roche Holding AG, of Basel, Switzerland

Tecentriq (atezolizumab)

PD-L1 binder

Unresectable hepatocellular carcinoma patients who have not received prior systemic therapy

When combined with Avastin (bevacizumab, Roche Holding AG), met both of co-primary endpoints, demonstrating statistically significant and clinically meaningful improvements in overall survival and progression-free survival compared with standard-of-care Nexavar (sorafenib, Bayer AG)

Seattle Genetics Inc., of Bothell, Wash.

Tucatinib

Tyrosine kinase inhibitor selective for HER2 without significant inhibition of EGFR

HER2-positive breast cancer

Met primary endpoint of progression-free survival, showing that the addition of tucatinib was superior to Herceptin (trastuzumab, Roche Holding AG) and capecitabine alone, with a 46% reduction in the risk of disease progression or death (p<0.00001)

Takeda Pharmaceutical Co. Ltd., of Osaka, Japan

Entyvio (vedolizumab)

Humanized monoclonal antibody alpha4beta7 integrin antagonist

Ulcerative colitis, Crohn's disease

Data from over 1,000 biologic-naïve patients receiving Entyvio or an anti-TNFα therapy were collected, with the incidence rate (per 100 person-years) for serious adverse events and serious infections estimated per cohort; the incidence rates for the first occurrence of a serious adverse event (vedolizumab: 4.6 [3.5-6.8]; anti-TNFα: 10.3 [9.5-14.9]) and serious infections (vedolizumab: 1.4 [0.8-2.5]; anti-TNFα: 2.6 [1.7-4.3]) were estimated to be lower with Entyvio treatment

Urovant Sciences Inc., of Irvine, Calif.

Vibegron

Beta 3 adrenergic receptor agonist

Overactive bladder

Part 2 of Courage study begun in men with benign prostatic hyperplasia

Actelion Pharmaceuticals Ltd., unit of Janssen Pharmaceutical Cos. of Johnson & Johnson, of New Brunswick, N.J.

Opsumit (macitentan)

Dual endothelin ET-A/ET-B receptor antagonist

Pulmonary arterial hypertension

Combination therapy with PDE5 inhibitor tadalafil (Adcirca, United Therapeutics Corp.) in Optima trial that enrolled 46 newly diagnosed participants showed 47% reduction in primary endpoint of mean pulmonary vascular resistance at week 16 and gains in functional parameters and risk profiles

Notes

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