August’s top news stories

5th September 2019 (Last Updated September 5th, 2019 00:00)

Biopharmaceutical firm Retrophin reported that the Phase III clinical trial of fosmetpantotenate failed to meet endpoints in pantothenate kinase-associated neurodegeneration (PKAN) patients; and MyoKardia has commenced dosing of patients in a Phase I trial of small molecule MYK-224 for the treatment of hypertrophic cardiomyopathy (HCM). Clinicaltrialsarena.com wraps up the key headlines from August 2019.

August’s top news stories
PKAN is a rare, genetic neurological disease that causes progressive, debilitating symptoms. Credit: Birth Into Being.

Retrophin’s fosmetpantotenate fails in Phase III trial for PKAN

Biopharmaceutical firm Retrophin reported that the Phase III clinical trial of fosmetpantotenate failed to meet endpoints in pantothenate kinase-associated neurodegeneration (PKAN) patients.

PKAN is a rare, genetic neurological disease that causes progressive, debilitating symptoms such as movement disorders and visual impairment.

It develops due to the PANK2 gene mutation, which impacts the production of coenzyme A (CoA) needed for various biochemical reactions in the body.


MyoKardia starts dosing hypertrophic cardiomyopathy patients

MyoKardia commenced dosing of patients in a Phase I trial of small molecule MYK-224 for the treatment of hypertrophic cardiomyopathy (HCM).

HCM is characterised by excessive heart muscle contraction and decreased filling ability of the left ventricle. It results in debilitating symptoms and cardiac dysfunction.

MYK-224 is developed to specifically target the heart’s cardiac myosin motor protein to normalise contractility and filling. It is expected to offer some dosing advantages to select HCM patients.


Oxurion reports mixed results from Phase IIa DME study

Oxurion reported top-line results from the Phase IIa clinical trial of THR-317 in combination with ranibizumab to treat diabetic macular edema (DME) patients.

THR-317 is a humanised antibody targeting placental growth factor (PlGF), while ranibizumab is an anti-vascular endothelial growth factor (VEGF) antibody.

The randomised, single-masked, active-controlled, multi-centre Phase IIa trial assessed the safety and efficacy of three monthly intravitreal injections of the combination in a total of 70 subjects with centre-involved DME.


ViiV’s long-acting HIV regimen passes late-stage trial

ViiV Healthcare reported that the Phase III ATLAS-2M clinical trial of its long-acting, two-drug regimen developed to treat HIV infection met its primary endpoint.

The regimen comprises ViiV’s cabotegravir and Janssen’s rilpivirine.

ATLAS-2M compared the antiviral activity and safety of the long-acting regimen given every eight weeks to the regimen given every four weeks. The 48-week study enrolled HIV-1 patients with suppressed viral load and no resistance to either drug.


Fulcrum launches Phase IIb trial of losmapimod to treat FSHD

Fulcrum Therapeutics started a Phase IIb clinical trial of losmapimod in patients suffering from facioscapulohumeral muscular dystrophy (FSHD).

Losmapimod is a selective inhibitor of p38α/β mitogen-activated protein kinase (MAPK).

Fulcrum in-licensed the drug based on its finding that p38α/β inhibition decreases expression of the DUX4 gene in muscle cells extracted from FSHD patients.


Outlook Therapeutics closes enrolment in Phase III wet AMD study

Outlook Therapeutics concluded the enrolment of patients in a Phase III clinical trial of ONS-5010 for the treatment of patients with wet age-related macular degeneration (wet AMD).

An ophthalmic formulation, ONS-5010 is a bevacizumab product candidate developed to treat retinal diseases, including wet AMD.

Bevacizumab is a humanised antibody that blocks the vascular endothelial growth factor (VEGF) and related angiogenic activity.


Knopp begins study of dexpramipexole in eosinophilic asthma patients

Knopp Biosciences commenced patient enrolment in a Phase II clinical trial of dexpramipexole for the treatment of moderate-to-severe eosinophilic asthma.

Dexpramipexole is a small molecule immunologic developed to treat eosinophil-driven diseases such as asthma, hypereosinophilic syndromes (HES), and chronic rhinosinusitis with nasal polyps.

The randomised, double-blind, placebo-controlled, biomarker trial will assess the clinical effects of three different doses of oral dexpramipexole on peripheral blood eosinophil count.


Abivax starts enrolment in Phase IIb ulcerative colitis study

Abivax recruited the first patient for a Phase IIb clinical trial assessing oral, once-daily ABX464 to treat moderate to severe active ulcerative colitis (UC).

ABX464 is an anti-inflammatory molecule that binds to the cap-binding complex (CBC) and boosts its function in cellular RNA biogenesis.

The drug candidate is said to improve the selective splicing of a single long non-coding RNA to allow the production of an anti-inflammatory microRNA called miR-124.


Farxiga meets primary goal in Phase III heart failure trial

AstraZeneca’s Farxiga (dapagliflozin) met the primary endpoint in Phase III DAPA-HF study involving patients with reduced ejection fraction (HFrEF), with or without type 2 diabetes.

Compared to placebo, the drug showed a statistically-significant and clinically-meaningful decrease in cardiovascular death or the worsening of heart failure when given in addition to standard of care.

The safety profile of the drug was also observed to be consistent with its established profile.


Study finds majority of cancer drug trials lack racial diversity

A new 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.