Give me a ‘C’

CAMBRIDGE, Mass.—Vertex Pharmaceuticals Inc. has announcedthe establishment of a non-exclusive agreement with GlaxoSmithKline PLC (GSK)to conduct a Phase II proof-of-concept study of an all-oral regimen fortreating hepatitis C, consisting of Vertex's nucleotide analogue hepatitis Cvirus (HCV) polymerase inhibitor VX-135 and GSK's NS5A inhibitor GSK2336805.

Under the agreement, Vertex and GSK will split the costsassociated with the study, and no upfront payments or milestone payments areincluded. The study is intended to determine the safety, tolerability and viralcure rates of a 12-week combination of VX-135 and GSK2336805.

The Phase II proof-of-concept study will enrolltreatment-naïve people who have chronic non-cirrhotic genotype 1 hepatitis C.Vertex will conduct the study in the United States early next year, pendingdiscussions with regulatory authorities. The study will test multiple 12-weekcombination regimens of the two compounds, with and without ribavirin, anucleotide analogue and one of the standards of care in treating hepatitis C.The agreement does not cover further clinical development studies beyond thePhase II study.

"This agreement underscores our broad commitment to developall-oral treatment regimens for people with hepatitis C using medicines withinour own pipeline and by working in collaboration with other companies like GSKwho share our commitment to further improve treatment of this disease," Dr.Robert Kauffman, senior vice president and chief medical officer at Vertex,said in a press release. "We are focused on the development of new all-oraltreatments for hepatitis C that have the potential to provide a high cure ratewith only 12 weeks of treatment, and we look forward to the start of this studywith GSK."

VX-135 is an investigation uridine nucleotide analoguepro-drug designed for inhibition of HCV replication by acting on the NS5Bpolymerase. The compound has shown to have a high barrier to drug resistance inin-vitro studies, with demonstratedantiviral activity across all genotypes of HCV, including those that are moreprevalent outside of the United States. Based on its first seven-day viralkinetic data for the compound, gathered earlier this year, Vertex plans toinitiate several all-oral, Phase II proof-of-concept studies, which willincluding one study of VX-135 and ribavirin as well as one of VX-135 andtelaprevir, Vertex's approved protease inhibitor (marketed as INCIVEK forpatients with chronic genotype 1 hepatitis C).

GSK2336805 is an investigational NS5A replication complexinhibitor under development as a potential hepatitis C treatment.

Zachry Barber, director of corporate communications forVertex, says this is not the first time the two companies have worked together,citing a 2005 collaboration in which GSK and Vertex collaborated on thedevelopment of VX-409 for the treatment of pain, though the compound is nolonger in development. The company, he notes, believes that "we are wellpositioned to conduct multiple all-oral studies in the coming year, and togenerate a significant amount of data in 2013 to support advancing into pivotaldevelopment in 2014."

Vertex announced a similar agreement with JanssenPharmaceuticals Inc. at the same time as the GSK agreement. Under the agreementwith Janssen, Vertex and Janssen will conduct a Phase II proof-of-concept studyof an all-oral hepatitis C treatment regimen that combines VX-135 and Janssen'sprotease inhibitor simeprevir to evaluate safety, tolerability and viral curerates of a 12-week combination. The companies will share development costs, andno upfront or milestone payments apply.

Current World Health Organization (WHO) statistics place thenumber of hepatitis C sufferers at roughly 150 million worldwide, withapproximately three million of that number located in the United States,according to the Centers for Disease Control. An estimated 350,000 deaths areattributed to hepatitis C-related liver diseases annually, according to WHO.The Institute of Medicine of the National Academies noted that annual medicalcosts associated with hepatitis C are expected to total approximately $85billion in the United States by 2029.

GSK, Imagine Institute collaborate on rare skin disorder

PARIS—On Nov. 12, GlaxoSmithKline PLC (GSK) and the ImagineInstitute announced they willcollaborate on the development of medicines with the potential to treatNetherton Syndrome, a rare, genetically inherited, severe skin disorder.

Under the terms of the agreement, the alliance will seek tobuild on the work of Prof. Alain Hovnanian at the Necker Children Hospital inParis following significant breakthroughs in the understanding of the underlyingdisease mechanisms guided by the identification of the disease gene. Work onthe project will be carried out within GSK and both at the Genetics Departmentand Inserm Transfert unit U781 at Necker Hospital in Paris.

The University Hospital Institute Imagine and Inserm willreceive success-based financial support from GSK linked to reaching agreedmilestones, as well as an undisclosed upfront payment and royalties on salesfrom any product that is successfully commercialized out of the collaboration.Inserm Transfert will provide both parties with alliance management.

Netherton syndrome is one of the most severe genetic skindiseases of children and adults. No specific treatment is currently available,but recent progress made in the disease pathogenesis allow for the developmentof specific new drugs targeting key biological events.

"The partnership with GSK validates the role of the researchin the area of novel therapeutic targets and its underlying mechanisms incombination with highest industry standards in drug discovery. For thepatients, it also means the possibility of a novel treatment in an acceleratedtimeframe," said Hovnanian in a press release.