September’s top news stories

9th October 2018 (Last Updated October 9th, 2018 00:00)

Emerald Health Pharmaceuticals (EHP) started patient enrolment for its Phase I clinical trial of EHP-101 for the treatment of patients with multiple sclerosis (MS) and scleroderma, and Ra Pharmaceuticals reported positive data from the Phase lb trial examining the pharmacokinetic (PK) of zilucoplan (RA101495 SC) to treat patients with renal impairment. Drugdevelopment-technology.com wraps up the key news stories from September 2018.

September’s top news stories
A type of localised scleroderma known as morphea. Credit: Leith C Jones at English Wikipedia.

EHP started enrolment in EHP-101 Phase I trial for MS and scleroderma

Emerald Health Pharmaceuticals (EHP) started patient enrolment for its Phase I clinical trial of EHP-101 for the treatment of patients with multiple sclerosis (MS) and scleroderma.

The randomised, double-blind, placebo-controlled trial is being carried out in Australia.

It will be conducted in two parts: a single-ascending dose phase (Part 1) and a multiple-ascending dose phase (Part 2).


Ra Pharma reported positive data from Phase lb trial of zilucoplan

Ra Pharmaceuticals reported positive data from the Phase lb trial examining the pharmacokinetic (PK) of zilucoplan (RA101495 SC) to treat patients with renal impairment.

The trial investigated zilucoplan in severe renal impairment patients as a lead-in to studying the drug in complement-mediated renal disorders.

The multi-centre, open-label trial included 16 participants, comprising eight patients with severe renal impairment and eight healthy control subjects with normal renal function.


Catabasis Pharmaceuticals began Phase lll DMD trial of edasalonexent

Catabasis Pharmaceuticals initiated a Phase lll PolarisDMD trial to evaluate the efficacy and safety of edasalonexent for the treatment of patients with Duchenne muscular dystrophy (DMD).

The one-year, randomised, double-blind, placebo-controlled trial is also expected to support an application for commercial registration of edasalonexent.

It intends to enrol around 125 patients between the ages of four and seven who have not been on steroids for at least six months, irrespective of mutation type.


Novavax started Phase ll trial of NanoFlu for seasonal influenza

Novavax began a Phase ll clinical trial to evaluate the safety and tolerability of NanoFlu in comparison with two unnamed US-licensed comparators for the treatment of seasonal influenza.

The randomised, observer-blinded, active-controlled trial aims to study different doses of NanoFlu, both adjuvanted with the company’s Matrix-M adjuvant and unadjuvanted, against the comparators.

It also intends to select a dose/formulation of NanoFlu for a future pivotal Phase lll immunogenicity clinical trial.


Aldeyra reported positive Phase llb results from reproxalap trial

Aldeyra Therapeutics reported positive results from its Phase llb clinical trial of topical ocular reproxalap for the treatment of patients with dry eye disease.

The trial was a randomised, vehicle-controlled, parallel-group, multi-centre, double-masked study that enrolled 300 patients.

It evaluated 0.1% and 0.25% concentrations of reproxalap topical ophthalmic solution in comparison with a vehicle in the enrolled patients who were randomised equally to receive the drug or vehicle for 12 weeks.


Daiichi Sankyo dosed first patients in Phase lll trials for breast cancer

Daiichi Sankyo dosed the first patients in two Phase lll DESTINY-Breast03 and DESTINY-Breast02 trials investigating the safety and efficacy of [fam-] trastuzumab deruxtecan (DS-8201) to treat patients with previously treated HER2 positive unresectable and/or metastatic breast cancer.

DESTINY-Breast03 will compare [fam-] trastuzumab deruxtecan with ado-trastuzumab emtansine (T-DM1), and DESTINY-Breast02 will evaluate [fam-] trastuzumab deruxtecan in patients previously treated with standard-of-care HER2 therapies, including T-DM1.

[Fam-] trastuzumab deruxtecan is being examined by Daiichi Sankyo as a HER2 targeting antibody drug conjugate (ADC).


Boston Therapeutics started new trial of BTI320 for Type 2 diabetes

Boston Therapeutics commenced a new confirmatory, proof of concept adaptive trial to investigate the potential of BTI320 for the treatment of patients with Type 2 diabetes.

Type 2 diabetes is a chronic condition that impacts the way an individual’s body metabolises sugar (glucose), an important source of fuel for the body.

The double-blind, placebo-controlled trial aims to study BTI320’s ability to support and possibly improve current anti-diabetic regimen of glycemic control in subjects with Type 2 diabetes.


Nightstar reported positive preliminary results from XIRIUS trial

Nightstar Therapeutics reported positive preliminary safety and efficacy results from the XIRIUS trial, a Phase l/ll study of NSR-RPGR for the treatment of X-Linked Retinitis Pigmentosa (XLRP) in patients with RPGR mutation.

The results are based on the dose-escalation phase of the XIRIUS trial, an open-label, dose-ranging, single-eye study, currently being conducted at sites in the US and the UK.

They include data from the one-month follow-up for the first five cohorts of patients.


Alector started Phase l trial of AL001 for frontotemporal dementia

Alector commenced a Phase l INFRONT trial to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL001 for the treatment of patients with Frontotemporal Dementia With Granulin Mutation (FTD-GRN).

The randomised, parallel assignment trial intends to enrol 60 participants, including both healthy subjects and FTD-GRN patients.

During the trial, subjects will receive up to five single-ascending doses of AL001. Each cohort will also receive saline solution as a single infusion in a ratio of six active and two placebo subjects.


Actelion reported positive results from Portico trial for PoPH

Actelion Pharmaceuticals reported positive outcomes from the Portico trial after meeting the primary endpoint of the study.

Portico is a Phase IV, prospective, randomised, placebo-controlled, double-blind, multicentre, parallel-group trial that examined the efficacy and safety of Opsumit (macitentan) against placebo for the treatment of patients with portopulmonary hypertension (PoPH).

During the trial, 85 patients were randomised in a 1:1 ratio into two treatment groups to receive 10mg of macitentan or placebo for a 12-week double-blind treatment period.