Pfizer treats first patient in trial for investigational mini-dystrophin gene therapy for the treatment of Duchenne muscular dystrophy

Pfizer has stated that they have begun their Phase 1b clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in patients with Duchenne muscular dystrophy (DMD). The first patient received an infusion of the mini-dystrophin gene, administered under the supervision of principal investigator, Edward Smith, MD, Associate Professor of Pediatrics and Neurology at Duke University

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Trial results show immunotherapy & targeted drugs could stop melanomas returning

Some of the treatments currently used for certain advanced melanomas could be effective in those with an earlier stage of disease after surgery, according to results from two clinical trials. The studies (Checkmate 238 and COMBI-AD) examined an immunotherapy drug and a combination of targeted drugs respectively, the results showed that both types of treatment

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Scotland first UK nation to routinely offer HIV PrEP along with Psoriasis, MS and CLL treatments

Scotland is set to become the first UK nation to routinely offer HIV pre-exposure prophylaxis to eligible patients after the Scottish Medicines Consortium backed the use of Gilead’s Truvada along with four other new therapies to be funded by the NHS. In clinical trials Truvada (emtricitabine/tenofovir disoproxil) was revealed to cut the relative risk of

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GW Pharmaceuticals gains orphan status for rare epilepsy cannabinoid Epidiolex

UK-based GW Pharmaceuticals’ cannabinoid Epidiolex has been awarded Orphan status by European regulators as treatment for Lennox-Gastaut Syndrome (LGS), a rare and severe form of childhood-onset epilepsy. The approval follows data from two late-stage trials that showed when Epidiolex was added as an adjunct to patients’ current treatment, there was a significant reduction in the

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New Pfizer antibiotic, Zavicefta launched in UK

Zavicefta has now been launched in the UK to treat serious aerobic Gram-negative infections caused by antibiotic resistant bacteria. The Pfizer drug is a fixed combination of avibactam, a new beta-lactamase inhibitor, and ceftazidime, an antibiotic belonging to the class of third generation cephalosporins which is already approved for use in the EU.

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AstraZeneca expands their first line cancer immunotherapy programme

AstraZeneca has expanded a late-stage trial that assess a potential new immuno-oncology (IO) therapy combination for the first-line treatment of non-small cell lung cancer (NSCLC), to include extra endpoints for both the combo and monotherapy. The Phase III MYSTIC trial was originally designed to assess the benefit of durvalumab monotherapy and a combination of durvalumab

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Allergan increases pipeline with Parkinson’s deal

Allergan has stepped into the field of Parkinson’s disease with an option to acquire US-based neurodegenerative research group Lysosomal Therapeutics (LTI). LTI-291, LTI’s lead programme, is designed to stimulate the activity of glucocerebrosidase (GCase) in the brain, which is reduced in many lysosomal storage diseases because of mutations in the GBA1 gene.

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AZ initiate trials for using diabetes drug in kidney disease and heart failure

AstraZeneca has launched two new trials with its SGLT-2 inhibitor Forxiga with the aim of exploring its potential beyond the drug’s approved diabetes indication. AZ have initiated two randomised, placebo-controlled Phase IIIb outcome trials assessing Forxiga in the management of chronic kidney disease and chronic heart failure, within participants with and without type-2 diabetes. The

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