Company

Product

Description

Indication

Status

Phase I

Alligator Bioscience AB, of Lund, Sweden

ATOR-1015

Next generation CTLA-4 bispecific antibody

Advanced solid tumors

First patient dosed in dose-escalation study in up to 53 patients; primary aim will test safety and tolerability and identify recommended phase II dose; results expected in the second half of 2020

Arqule Inc., of Burlington, Mass.

ARQ-531

Reversible dual inhibitor of wild-type and C481S-mutant BTK

Chronic lymphocytic leukemia

Company disclosed in its earnings call the first evaluable patient with C481S mutation dosed in cohort 7 (65 mg QD) achieved an 88% tumor burden reduction after the first scan

Boehringer Ingelheim GmbH, of Ingelheim, Germany

BI-655130

IL-36R-blocking monoclonal antibody

Generalized pustular psoriasis

Data published in The New England Journal of Medicine showed rapid improvement in symptoms in 7 patients experiencing acute moderate or severe disease flares; 5 of 7 patients in the 20-week study achieved clear or almost clear skin within the first week, following single-dose treatment, and all achieved that outcome after 4 weeks; average improvement in skin symptoms was close to 80% at week 4 and was maintained until end of study

Forendo Pharma Oy, of Turku, Finland

FOR-6219

HSD17B1 inhibitor

Endometriosis

Completed a phase Ia study showing single doses from 2 mg up to 175 mg and multiple doses up to 150 mg twice daily for 10 days were found to be safe and well-tolerated; data support initiation of phase Ib trial in healthy premenopausal women

Kezar Life Sciences Inc., of South San Francisco

KZR-616

Immuno-proteasome inhibitor

Lupus nephritis

Started randomized, double-blind, placebo-controlled single and multiple ascending-dose trial to test safety, tolerability, pharmacokinetics and target inhibition using simplified lyophilized formulation in up to 72 healthy female subjects

Phase II

Gilead Sciences Inc., of Foster City, Calif.

Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg)

HIV-1 integrase/nucleoside reverse transcriptase inhibitor

HIV-1

Through week 48 of phase II/III study, 98% (n=74/75) of virologically suppressed individuals ages 6 to 18 (n=100) living with HIV maintained undetectable viral load, and none developed treatment-emergent resistance

Opthea Ltd., of Melbourne, Australia

OPT-302

Soluble form of VEGFR3

Wet age-related macular degeneration

Independent data safety and monitoring board for ongoing phase IIb study reaffirmed for the second time its positive recommendation the trial continue without modification

Sanbio Group, of Mountain View, Calif.

SB-623

Cell therapy

Traumatic brain injury

Results from STEMTRA study showed primary endpoint was achieved, with the treatment group demonstrating statistically significant improvement in motor function vs. control group

Sound Pharmaceuticals Inc., of Seattle

SPI-1005

Mimics and induces glutathione peroxidase activity

Meniere's disease

Completed enrollment in phase IIb trial

Zynerba Pharmaceuticals Inc., of Devon, Pa.

Zygel (ZYN-002)

CBD gel

Autism spectrum disorder

Started 14-week, open-label BRIGHT study to assess safety, tolerability and efficacy in about 36 children and adolescents; top-line data expected in the first half of 2020

Phase III

Allergan plc, of Dublin

Rapastinel

Selective positive NMDA receptor modulator

Major depressive disorder

Top-line results from 3 pivotal studies as an adjunctive treatment showed rapastinel arms did not differentiate from placebo on primary and key secondary endpoints; an interim analysis of a separate relapse prevention study suggests primary and key secondary endpoints will not be met

Bioarctic AB, of Stockholm

BAN-2401

Amyloid beta-targeting monoclonal antibody

Early Alzheimer's disease

Partner Eisai Co. Ltd. said confirmatory phase study will be initiated in March 2019, with final readout of primary endpoint in 2022

Gilead Sciences Inc., of Foster City, Calif.

Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg)

HIV-1 integrase/nucleoside reverse transcriptase inhibitor (NRTI)

HIV-1

2 studies of resistance profile in virologically suppressed adults switching from dolutegravir/abacavir/lamivudine or boosted protease inhibitor found high rates of virologic suppression with Biktarvy in treatment-experienced adults (n=561/570; 98%), including those with pre-existing resistance to NRTIs (n=155/159; 97%); none developed treatment-emergent resistance during studies

Gilead Sciences Inc., of Foster City, Calif.

Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg tablets)

HIV-1 integrase/nucleoside reverse transcriptase inhibitor

HIV-1

Ongoing study evaluating 565 virologically suppressed adults who switched 1-to-1 from regimen of DTG+F/TAF or DTG+F/TDF to DTG+F/TAF or Biktarvy for 48 weeks found 99% (n=557/562) of those with a post-baseline visit and 99% (n=220/222) of those with resistance to any class of antiretroviral had undetectable viral load (HIV-1 RNA <50 copies/mL) with no emergent drug resistance

Helsinn Group, of Lugano, Switzerland

Anamorelin

Ghrelin receptor agonist

Weight loss, anorexia

First patient dosed in global program (2 24-week double-blind, randomized, placebo-controlled confirmatory trials) to evaluate efficacy and safety in patients with advanced non-small-cell lung cancer with cachexia; primary efficacy endpoint, measured at week 9, based on concomitant occurrence of clinically relevant improvement of body weight and 5-item Anorexia Symptoms Scale score in those alive at day 64

Viiv Healthcare Ltd., of London

Cabotegravir and rilpivirine

HIV-1 integrase strand transfer inhibitor and NNRTI

HIV-1

48-week data from ATLAS and FLAIR studies showed both met primary endpoints of noninferiority in maintaining viral suppression in adults at every-4-week injections vs. oral, daily, 3-drug regimen


Notes

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