2019 top news stories

3rd January 2020 (Last Updated January 3rd, 2020 15:28)

Athersys reports positive data from MultiStem cell therapy trial and CRISPR and Vertex treat first patient in gene-editing therapy trial. Clinicaltrialsarena.com wraps up the key headlines from 2019.

2019 top news stories
Chest x-ray of person with severe ARDS demonstrating widespread ‘ground-glass’ appearing opacities in both lungs. Credit: James Heilman, MD.

Athersys reports positive data from MultiStem cell therapy trial

Athersys reported positive results from a trial evaluating MultiStem cell therapy for the treatment of patients with acute respiratory distress syndrome (ARDS).

Initial data from the double-blind, randomised, placebo-controlled portion of the trial demonstrated that 25% of patients treated with MultiStem achieved a lower mortality rate than the 40% of patients treated with placebo.

It was also found that patients in the MultiStem treatment group achieved higher ventilator-free (VF) days and ICU-free days compared to the patients in the placebo group.


CRISPR and Vertex treat first patient in gene-editing therapy trial

Swiss gene-editing company CRISPR Therapeutics and US-based Vertex Pharmaceuticals started treating patients in a Phase I/II clinical trial of an investigational gene-editing therapy CTX001 for the treatment of transfusion-dependent beta thalassemia (TDT).

CTX001 is an autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy developed to help patients suffering from severe hemoglobinopathies.

The product is the first therapy under a research collaboration formed by CRISPR and Vertex in 2015 to use CRISPR/Cas9 for developing new treatments targeting underlying genetic causes of disease.


Biogen and Eisai end two Alzheimer’s drug trials

Biogen and Eisai scrapped the Phase III ENGAGE and EMERGE clinical trials evaluating aducanumab for the treatment of mild cognitive impairment due to Alzheimer’s disease, and mild Alzheimer’s dementia.

The move came after a futility analysis by an independent data monitoring committee revealed that the trials were not likely to meet their primary endpoint upon completion.

Biogen and Eisai noted that the decision was not based on safety concerns with the drug.


Gilead’s selonsertib fails in second Phase III trial for NASH

Gilead Sciences reported that selonsertib failed to meet the primary endpoint in the Phase III STELLAR-3 clinical trial performed in patients with bridging fibrosis (F3) caused by nonalcoholic steatohepatitis (NASH).

The announcement came two months after the apoptosis signal-regulating kinase 1 (ASK1) inhibitor failed to demonstrate positive outcomes in the Phase III STELLAR-4 study.

In the STELLAR-3 trial, the drug did not meet the primary endpoint of a ≥1-stage histologic improvement in fibrosis without worsening NASH at week 48.


BioMarin reports long-term data for haemophilia A gene therapy

BioMarin Pharmaceutical reported positive three-year data from an ongoing Phase I/II trial of its investigational gene therapy valoctocogene roxaparvovec.

Developed to treat adults with severe haemophilia A, the drug secured breakthrough therapy designation from the US Food and Drug Administration (FDA) and priority medicines (PRIME) access from the European Medicines Agency (EMA).

The therapeutic also received orphan drug designation from both regulators.


FDA lifts partial clinical hold on AbbVie’s trial of venetoclax

The US Food and Drug Administration (FDA) lifted the partial clinical hold placed on AbbVie’s CANOVA (M13-494), a Phase III trial evaluating venetoclax to treat relapsed / refractory multiple myeloma.

In March this year, the FDA placed a clinical hold on all trials evaluating venetoclax following a review of data from the Phase III BELLINI trial in relapsed / refractory multiple myeloma.

Venetoclax is being developed by AbbVie and Roche.

AbbVie said that a higher proportion of deaths were observed in the venetoclax arm of the study when compared to the control arm.


Study finds majority of cancer drug trials lack racial diversity

A study in the US found that less than 8% of oncology clinical trials over the past decade featured participants from the country’s major races.

The study was performed by the University of British Columbia, the Fred Hutchinson Cancer Center, the University of Texas MD Anderson Cancer Center, and Baylor University.

Data from cancer drug trials between 2008 and 2018, which comprised a total of 112,293 subjects across 230 studies, revealed a lack of racial and ethnic diversity.


Amgen’s KRAS-targeting cancer drug yields favourable results

Amgen reported additional results from the Phase I clinical trial of its investigational cancer drug AMG 510, which irreversibly targets the KRASG12C protein.

The open-label, multi-centre study involved patients with KRAS G12C-mutated solid tumours who previously received a minimum of two or more lines of therapy.

It assessed a once-daily, oral 180mg, 360mg, 720mg and 960mg dose of AMG 510.


GSK initiates Phase III programme of gepotidacin antibiotic

GlaxoSmithKline (GSK) initiated the Phase III EAGLE clinical programme of its investigational antibiotic, gepotidacin, for the treatment of uncomplicated urinary tract infection (uUTI, acute cystitis) and urogenital gonorrhoea (GC).

Gepotidacin belongs to the triazaacenaphthylene bacterial topoisomerase inhibitors antibiotic class. The drug specifically acts on the DNA gyrase and topoisomerase IV enzymes involved in bacterial replication.

The Phase III programme of gepotidacin comprises EAGLE-1 and EAGLE-2 trials. Dosing of patients in the programme has already been commenced.


Takeda reports positive long-term data for Alunbrig in ALK+ NSCLC

Takeda Pharmaceutical reported positive results from the Phase III ALTA-1L clinical trial of Alunbrig (brigatinib) in patients with anaplastic lymphoma kinase-positive (ALK+) non-small cell lung cancer (NSCLC).

Alunbrig is a selective tyrosine kinase inhibitor (TKI) of the ALK fusion protein in NSCLC. The ALTA-1L trial assessed the drug in 275 adult patients who did not receive previous treatment with an ALK inhibitor.

Risk of disease progression or death decreased by 76% with Takeda’s drug compared to crizotinib in newly diagnosed participants whose cancer had spread to the brain at baseline.