2014: The year's biggest Drug Development stories

GlaxoSmithKline (GSK) reported positive results from an Ebola vaccine Phase I trial, which showed that the drug was well-tolerated and produced an immunological response in each of the 20 healthy adults in the US.


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GSK and NIH's Ebola vaccine shows promise in first Phase I human trial

gskebola

GlaxoSmithKline (GSK) reported positive results from an Ebola vaccine Phase I trial, which showed that the drug was well-tolerated and produced an immunological response in each of the 20 healthy adults in the US.

The new vaccine candidate was co-developed by the National Institutes of Health' (NIH) National Institute of Allergy and Infectious Diseases (NIAID) and Okairos, a biotechnology firm acquired by GSK in 2013.

The trial was conducted by the NIH and results were published in the New England Journal of Medicine (NEJM).

Washington University-developed new breast cancer vaccine shows promise

Researchers at Washington University School of Medicine in St. Louis, US, reported preliminary data from a small clinical trial of a new breast cancer vaccine.

Led by Washington University breast cancer surgeon and senior author Dr William Gillanders, the trial showed the vaccine to be safe in patients with metastatic breast cancer, as well as helped slow cancer progression.

The new vaccine causes the body's immune system to focus on a protein called mammaglobin-A, found in breast tissue.

Novartis's Phase IV trial of Onbrez Breezhaler in COPD patients meets primary objective

novartis copd

Novartis reported positive results from the Phase IV INSTEAD switch trial of Onbrez Breezhaler, carried out in patients with chronic obstructive pulmonary disease (COPD).

The company said the trial met its primary objective and the once-daily Onbrez Breezhaler (indacaterol) 150mcg showed non-inferiority in lung function at week 12 to twice-daily Seretide (salmeterol/fluticasone propionate (SFC)) 50 / 500mcg in patients with moderate COPD who had experienced no exacerbations in the previous year.

The global, randomised, double-blind, parallel-group, 26-week INSTEAD trial also demonstrated similar symptomatic benefits in terms of shortness of breath and health status after 12 and 26 weeks in patients treated with Onbrez Breezhaler compared to those on SFC.

Novartis Pharmaceuticals global head of development Tim Wright said the positive results help inform the switch from salmeterol/fluticasone to Onbrez Breezhaler in patients with moderate COPD and who are at low-risk of exacerbations.

Mr Wright said: "This confirms that Onbrez Breezhaler provides an effective maintenance treatment option for these patients."

Pfizer and Merck team up on Phase Ib combination trial to treat lung cancer

lung cancer

Pfizer agreed to evaluate the combination of its lung cancer drug crizotinib (Xalkori) with Merck's investigational anti-PD-1 antibody pembrolizumab, in a Phase Ib clinical trial to treat patients with ALK-positive advanced or metastatic non-small cell lung cancer (NSCLC).

The multi-centre, open-label clinical trial will evaluate the safety and tolerability of the combination therapy, and is scheduled to be initiated in 2015 by Pfizer.

Xalkori is a kinase inhibitor indicated to treat patients with metastatic NSCLC whose tumours are anaplastic lymphoma kinase (ALK) positive as detected by an FDA-approved test, while pembrolizumab is designed to reactivate anti-tumour immunity.

First clinical trial of Ebola vaccine begins in Mali, West Africa

University of Maryland School of Medicine's (UM SOM) Centre for Vaccine Development (CVD) together with CVD-Mali and the Ministry of Health of Mali started a clinical trial of the Ebola vaccine on Malian health care workers.

The vaccine was developed by the Vaccine Research Centre (VRC) of the National Institute of Allergy and Infectious Diseases (NIAID) in Bethesda, Maryland.

It consists of an adenovirus (cold virus) that develops a single attachment protein of Ebola virus in humans without creating any harm to the human body.

GSK and MMV initiate Phase III programme for development of malaria drug

GlaxoSmithKline (GSK) and Medicines for Malaria Venture (MMV) initiated a Phase III global programme to assess the efficacy and safety of tafenoquine, an investigational medicine, being developed for the treatment and relapse prevention (radical cure) of Plasmodium vivax (P vivax) malaria.

The disease is caused by one of several species of Plasmodium parasites known to infect humans and it occurs mainly in South and South East Asia, Latin America and the horn of Africa.

The most serious consequences described to be caused by the infection are severe anaemia, malnutrition and respiratory distress.

Pfizer's lung cancer drug dacomitinib fails in two Phase III trials

Pfizer released top-line results from two randomised Phase III trials of the irreversible pan-HER kinase inhibitor dacomitinib for the treatment of patients with advanced non-small cell lung cancer (NSCLC).

Both trials, ARCHER 1009 and NCIC CTG BR.26, evaluated dacomitinib in populations of previously treated patients with advanced NSCLC.

The study did not show dacomitinib improved progression-free survival (PFS) in either of the co-primary populations compared to erlotinib.

Novartis reports positive Phase III trial results of pancreatic cancer drug Afinitor

Novartis reported final results from a Phase III trial of Afinitor (everolimus) tablets plus best supportive care (BSC) compared to placebo plus BSC in patients with well-differentiated advanced and progressive pancreatic neuroendocrine tumours (pNET).

The company noted the RADIANT-3 trial met the secondary endpoint of overall survival.

Results from the trial showed a median overall survival (OS) of 44.02 months in the everolimus treatment arm and 37.68 months in the placebo arm, while the 6.34 month difference between the two arms was not statistically significant.

Cerulean starts dosing in Phase II combination therapy trial for ovarian cancer treatment

US-based Cerulean Pharma dosed the first patient in a Phase II clinical trial of its lead candidate, CRLX101, in combination with Avastin (bevacizumab) for the treatment of relapsed ovarian cancer.

The open-label single-arm Phase II combination therapy trial is being carried out at Massachusetts General Hospital (MGH) and the affiliated Harvard teaching hospitals under the direction of principal investigator Dr Carolyn Krasner.

The trial is designed to investigate the rate of progression-free survival at six months (PFS6) using RECIST (Response Evaluation Criteria in Solid Tumors) version 1.1 criteria in relapsed ovarian cancer patients treated with a combination of Avastin and CRLX101.

Abbvie begins Phase III trial of Veliparib to treat breast cancer patients

abbvie

US-based biopharmaceutical firm AbbVie started a Phase III clinical trial assessing the safety and efficacy of its investigational compound, veliparib (ABT-888), in combination with carboplatin and paclitaxel, two chemotherapeutic medicines, in patients with advanced breast cancer.

Veliparib is an investigational oral poly (adenosine diphosphate [ADP]-ribose) polymerases (PARP) inhibitor currently being assessed in multiple tumour types.

In the trial, the combination of veliparib, carboplatin and paclitaxel will be compared with treatment with carboplatin, paclitaxel and placebo in these patients with human epidermal growth factor receptor 2-(HER2) negative metastatic or locally-advanced breast cancer, containing BRCA1 and/or BRCA2 gene mutations.

Merck begins Phase III letermovir trial for prevention of CMV infection

merck cmv

US-based pharmaceutical firm Merck has enrolled the first patient in a global Phase III clinical trial of letermovir (MK-8228), an investigational antiviral agent, for prevention of cytomegalovirus (CMV) infection in high-risk bone marrow transplant patients.

The trial will assess the efficacy and safety of letermovir for the prevention of clinically-significant CMV infection in adults aged over 18 CMV-seropositive recipients of allogeneic hematopoietic stem cell transplants.

Letermovir is being developed for the prevention of human CMV infection and secured orphan product designation from the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) for the prevention of CMV viremia.

Gilead's Phase II RSV challenge study of GS-5806 meets primary and secondary endpoints

US-based biopharmaceutical firm Gilead Sciences reported results from a placebo-controlled, Phase IIa challenge trial of its investigational oral RSV fusion inhibitor 'GS-5806' in healthy adult patients intranasally infected with respiratory syncytial virus (RSV).

The trial achieved its primary and secondary endpoints of lower viral load, improvements in total mucus weight and also symptom diary score compared to a placebo.

The double-blind, placebo-controlled challenge trial was designed to evaluate the effect of GS-5806 on AUC RSV viral load (primary endpoint), as well as on mucus weight and total symptom score (secondary endpoints).