Company Product Description Indication Status
Phase I
Aravive Inc., of Houston AVB-500 Growth arrest specific protein 6 ligand inhibitor Ovarian cancer Data from first 31 participants with platinum-resistant recurrent disease treated at 10 mg/kg in ongoing phase Ib/II trial showed high serum drug levels were predictive of antitumor activity with statistically significant correlation to primary endpoint of progression-free survival (PFS; p=0.0066); those who met or exceeded minimal efficacious drug concentration showed greater than 4-fold increase in median PFS over those with low exposure (8.1 vs. 1.8 months; p=0.0016) and approximately 2-fold improvement in overall response rate (29% vs. 14%), including 1 complete response
Cstone Pharmaceuticals Co. Ltd., of Suzhou, China Tibsovo (ivosidenib) IDH1 gene inhibitor Acute myeloid leukemia First participant dosed in bridging registrational study in China designed to validate efficacy, safety and pharmacokinetics in patients with IDH1-mutant relapsed or refractory disease
Diurnal Group plc, of Cardiff, U.K. Ditest (native oral testosterone) Androgen receptor agonist Hypogonadism Primary endpoint, which compared rate and absorption of testosterone from single 120-mg dose with single dose of testosterone undecanoate 80 mg in fed state, was met in 24-patient study; Ditest achieved testosterone levels within healthy young male adult normal range after oral administration with less variability than testosterone undecanoate
Ideaya Biosciences Inc., of South San Francisco IDE-196 Protein kinase C inhibitor Metastatic uveal melanoma 27 participants enrolled in dose-escalation portion, including 12 in MUM dose-limiting toxicity (DLT) cohorts, 14 in MUM overflow cohort and 1 with GNA11 cutaneous melanoma in non-MUM GNAQ/11 cohort; all in DLT cohorts remained on therapy with duration of treatment ranging from 1.3 to 4 months as of Oct. 28 data cutoff with no reported DLTs or adverse events > grade 3
Phoenix Molecular Designs Ltd., of Vancouver, British Columbia PMD-026 Ribosomal protein S6 kinase-2 inhibitor Breast cancer First participant dosed in phase I/Ib study enrolling individuals with metastatic breast cancer in dose-escalation phase as well as those with triple-negative breast cancer in phase Ib; study includes certified companion diagnostic designed to detect RSK2 activation in breast tumors and correlate response
Seelos Therapeutics Inc., of New York SLS-002 (intranasal racemic ketamine) NMDA receptor antagonist Suicidal ideation First of expected 48 healthy volunteers dosed in drug-drug interaction (DDI) study in combination with venlafaxine ER or sertraline to evaluate pharmacokinetic profile, DDI and safety measures
Spring Bank Pharmaceuticals Inc., of Hopkinton, Mass. SB-11285 Stimulator of interferon gene agonist Advanced solid tumors First participants dosed in dose-escalation study with SB-11285 alone followed by combination with checkpoint inhibitor to determine recommended phase II dose; part 2 of trial will explore antitumor activity of intravenous drug in combination with checkpoint inhibitor
Trevena Inc., of Chesterbrook, Pa. Oliceridine Opioid receptor mu agonist Pain Pharmacokinetic studies of intravenous drug in individuals with end-stage renal disease and in those with hepatic impairment, published in Clinical Pharmacology in Drug Development, showed no dose adjustments needed in those populations
Vivus Inc., of Campbell, Calif. Qsymia (phentermine and topiramate ER) Adrenergic receptor agonist Binge-eating disorder; bulimia nervosa Findings from study that enrolled 22 participants, published in International Journal of Eating Disorders, showed mean objective binge-eating days declined over 4 weeks from 16.2 at baseline to 4.2 and 13.2 days for study drug and placebo, respectively (p<0.0001); abstinence rates were 63.6% with Qsymia and 9.1% with placebo (p<0.0001); Qsymia was associated with mean weight decrease of 5.8 kg compared with mean weight gain of 0.4 kg on placebo
Phase II
Brainstorm Cell Therapeutics Inc., of New York Nurown Autologous bone-marrow derived MSC-NTF cells Amyotrophic lateral sclerosis Findings in 48 participants, published in Neurology, showed rate of ALS disease progression (ALSFRS-R slope) was stabilized for up to 12 to 16 weeks in prespecified group with rapid progression (p<0.05) following single transplantation; higher proportion of Nurown-treated participants experienced ≥1.5-point/month improvement in ALSFRS-R slope at all post-treatment time points, statistically significant at 4 and 12 weeks post-transplant (p=0.004 and 0.046, respectively)
Heat Biologics Inc., of Durham, N.C. HS-110 (viagenpumatucel-L) Endoplasmin modulator Non-small-cell lung cancer Additional interim top-line data from cohort A in trial with Opdivo (nivolumab, Bristol-Myers Squibb Co.) showed median overall survival (mOS) of 16.9 months with 50% of patients censored, with median follow-up of 17 months, compared to 12.2 month mOS in Checkmate 057 study with nivolumab alone in similar population; objective response rate per iRECIST was 22% and disease control rate was 48%, with tumor shrinkage seen in 46% of participants
Medsenic SAS, of Strasbourg, France Arscimed  Arsenic trioxide Chronic graft-vs.-host disease Final participant enrolled in trial assessing optimal benefit/risk ratio for intravenous treatment with primary endpoint of partial and/or complete response rate 6 months after diagnosis; preliminary data expected midyear 2020; current observations confirm phase III development plan
Phase III
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 assess the other co-primary endpoint of RFS in the all-comer population
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
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
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

Notes

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