Aldeyra Therapeutics Inc., of Lexington, Mass.
Pleural malignant mesothelioma
In the Meso-2 study of 23 evaluable patients, ADX-1612 plus pemetrexed and platinum therapy produced stable disease or clinical response in 96% of patients; overall response rate was 61%, including 7 patients with a reduction of tumor burden of more than 50%; response rate in patients with epithelial histology was 76%
Checkpoint Therapeutics Inc., of New York
EGFR tyrosine kinase inhibitor
Advanced non-small-cell lung cancer
Drug produced an objective response rate of 75% in 8 treatment-naïve patients; disease control rate was 100% in 19 patients, including 84% who had target lesion reductions vs. baseline; 60% of the 5 patients with baseline brain metastases had intracranial disease response; plans to start a phase III study in 2019 in treatment-naïve EGFR mutation-positive patients
Pharmamar SA, of Madrid
RNA polymerase II inhibitor
Relapsed small-cell lung cancer
Treatment with lurbinectedin in combination with doxorubicin in patients with a chemotherapy-free interval (CTFI) of 30 days or longer produced overall survival of 10.2 months; OS was 11.5 months in platinum-sensitive patients with a CTFI of 90 days or better
TP Therapeutics Inc., of San Diego
ROS1 tyrosine kinaseinhibitor
ROS1 fusion-positive non-small-cell lung cancer
Of 10 tyrosine kinase inhibitor (TKI)-naïve evaluable patients in the Trident-1 study, 80% had an overall response, including a 100% response rate for the 3 patients with measurable CNS disease; overall clinical benefit rate was 100% in the 10 patients; of the 17 TKI-pretreated patients, the ORR was 18% and overall clinical benefit rate was 76%
VBI Vaccines Inc., of Cambridge, Mass.
Cytomegalovirus antigens, gB and pp65
Data and safety monitoring board recommended continuing the study without modification after completing its second safety assessment; company began enrolling patients in high-dose cohort; data from low- and intermediate-dose cohorts are expected in the fourth quarter of 2018
Bergenbio A/S, of Bergen, Norway
Non-small-cell lung cancer
In AXL-positive, previously-treated patients treated with bemcentinib and Keytruda (pembrolizumab), the overall response rate was 40%; in patients treated with bemcentinib and Tarceva (erlotinib), tumor shrinkage was seen in 6 of 9 patients; median progression-free survival has not been met for bemcentinib/Tarceva treatment but has exceeded 10 months for Tarceva monotherapy
Beyondspring Inc., of New York
Advanced or metastatic non-small-cell lung cancer
In Study 105, 0% of patients taking plinabulin had docetaxel-induced thrombocytopenia during cycle 1, compared to 45% of patients taking Neulasta
Intelgenx Corp., of Saint Laurent, Quebec
Leukotriene receptor antagonist
Mild to moderate Alzheimer's disease
Starting patient recruitment in the 70-patient study testing the safety, feasibility, tolerability and efficacy over 26 weeks of treatment
Marinus Pharmaceuticals Inc., of Radnor, Pa.
Positive allosteric modulator of GABAA
Completed enrollment in study testing the safety, pharmacokinetics and efficacy of the drug; data expected in the fourth quarter of 2018
Maxivax SA, of Geneva
Patient tumor antigens and an adjuvant GM-CSF
Head and neck squamous carcinoma, stage III/IV in recurrent or metastatic stage
Started an open-label study in 40 to 45 patients testing overall survival at 26 weeks
Mesoblast Ltd., of New York
Allogeneic mesenchymal precursor cell
Hypoplastic left heart syndrome
No cell-related safety concerns reported in trial to date
Novavax Inc., of Gaithersburg, Md.
Nanoparticle seasonal influenza vaccine
Enrolled first patients in study of 1,375 healthy older adults, comparing different doses and formulations of the vaccine with and without Matrix-M adjuvant to 2 comparator vaccines to determine the best dose/formulation to test in a phase III trial; top-line data expected in the first quarter of 2019
Reata Pharmaceuticals Inc., of Irving, Texas
Chronic kidney disease
In the IgA nephropathy cohort of the Phenix study, bardoxolone increased eGFR by 8 mL/min/1.73 m2 at week 12 compared to baseline (p<0.0001); pre-treatment decline was at an average annual rate of 1.2 mL/min/1.73 m2; in the cohort of patients with type 1 diabetic CKD, eGFR increased by 5.5 mL/min/1.73 m2 at week 12 compared to baseline (p=0.02); decline was at an average annual rate of 1.9 mL/min/1.73 m2 prior to study entry
Syndax Pharmaceuticals Inc., of Waltham, Mass.
Selective, oral, once-weekly inhibitor of class 1 HDACs
Non-small-cell lung cancer
Data from ongoing ENCORE 601 cohort of patients previously treated with chemotherapy and PD-(L)1 therapy showed 7 confirmed partial responses at time of data cut-off among 72 evaluable patients, for a 10% objective response rate, a median duration of response of 5.3 months and median progression-free survival of 2.8 months; results did not meet prespecified ORR endpoint
Astrazeneca plc, of Cambridge, U.K.
Unresectable stage III non-small-cell lung cancer
Data from PACIFIC trial in patients whose disease had not progressed following chemoradiation showed Imfinzi improved overall survival, the second primary endpoint, vs. placebo, regardless of PD-L1 expression, reducing the risk of death by 32% (p=0.0025)
Atox Bio Inc., of Chapel Hill, N.C.
Peptide binding to CD28 co-stimulatory receptor
Necrotizing soft tissue infections
Independent data monitoring committee completed pre-planned safety review of first 200 patients in ACCUTE trial and recommended the study continue without modification through completion; trial is expected to complete in 2019
Biocardia Inc., of San Carlos, Calif.
Stem cell therapy
9-month results from the 10-patient roll-in cohort of the pivotal study showed clinically meaningful improvements in symptoms, quality of life and exercise capacity, as measured by New York Heart Association class, Minnesota Living with Heart Failure Questionnaire and 6-minute walk distance, respectively
Boehringer Ingelheim GmbH, of Ingelheim, Germany
Triple angiokinase inhibitor
Phase III of LUME-Meso study found nintedanib combined with SOC pemetrexed/cisplatin does not impact PFS and OS for unresectable malignant pleural mesothelioma patients with epithelioid histology
Catabasis Pharmaceuticals Inc., of Cambridge, Mass.
Duchenne muscular dystrophy
Initiated PolarisDMD trial intended to support a registrational application; 1-year study is expected to enroll about 125 patients, ages 4 to 7, regardless of mutation type who have not been on steroids for at least 6 months; primary endpoint is a change in the North Star Ambulatory Assessment score, with secondary endpoints including timed function tests, 4-stair climb and 10-meter walk/run; top-line results expected in the second quarter of 2020
Daiichi Sankyo Co. Ltd., of Tokyo
[Fam-] trastuzumab deruxtecan (DS-8201)
HER2-targeting antibody-drug conjugate
Previously treated HER2-positive unresectable and/or metastatic breast cancer
Dosed the first patients in 2 global studies; 500-patient DESTINY-Breast03 is a head-to-head comparison vs. T-DM1 (ado-trastuzumab emtansine), with a primary endpoint of progression-free survival; 600-patient DESTINY-Breast02 will test the drug in patients previously treated with standard-of-care HER2 therapies, including T-DM1, with a primary endpoint of PFS
Genentech Inc., of South San Francisco
Small-cell lung cancer
IMpower133 study of Tecentriq plus carboplatin and etoposide for first-line treatment of previously untreated extensive-stage small-cell lung cancer showed combo helped people live longer compared with chemotherapy alone (OS = 12.3 months vs. 10.3 months; p=0.0069)
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan
Small-molecule inhibitor of ALK and EGFR
ALK-positive locally advanced or metastatic non-small-cell lung cancer
Results from ALTA-1L trial showed Alunbrig reduced the risk of disease progression or death by more than 50% vs. crizotinib in adults who had not received a prior ALK inhibitor
TG Therapeutics Inc., of New York
Ublituximab plus umbralisib
Glycoengineered monoclonal antibody targeting CD20 epitope plus oral PI3K-delta inhibitor
Chronic lymphocytic leukemia
Independent data safety monitoring board for the UNITY-CLL trial advised that the interim analysis of overall response rate could not be conducted at this time because data were not sufficiently mature; the company is now guiding that it will focus on the primary endpoint of progression-free survival and will no longer plan to seek accelerated approval based on ORR; the DSMB also recommended the trial continue without modification after reviewing safety data from more than 600 patients
For more information about individual companies and/or products, see Cortellis.