Shire to develop mRNA technology with $15m cystic fibrosis collaboration

Shire is collaborating with Cystic Fibrosis Foundation Therapeutics (CFFT), the non-profit R&D arm of the Cystic Fibrosis Foundation, to support its Messenger RNA (mRNA) technology platform for CF.

CFFT has committed up to $15 million to the programme. In CF, mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene lead to a disruption of the normal regulation of fluids in the lung that cause the secretions to thicken, thereby restricting lung function and leading to recurrent lung infections.

Shire is investigating therapies to deliver mRNA to sites in the body where it can be used by the body’s own cellular mechanisms to produce normal working copies of the protein.

Shire’s goal is to deliver mRNA that codes for a fully functional (wild type) version of the CFTR protein to the lungs of CF patients. If high enough levels of functional CFTR protein can be produced, lung function may be improved, thereby reducing the frequency and severity of infections.

This collaboration comes on the back of other promising news for those with CF. In November, Vertex added to its CF armoury when it filed a two-drug combination, also targeting CFTR gene mutations, in Europe and the US. Its first-in-class CF drug Kalydeco (ivacaftor), launched in 2012, only works for a small number of patients, but the combination of this with another Vertex drug, lumacaftor, aims to treat the most common form of the disease – those patients with two copies of the F508del mutation.

Shire is also investigating other diseases for which its mRNA technology platform could bring benefits.

The company has a record 22 programmes in clinical development and anticipates a number of milestones over the next 18 months.

Philip Vickers, global head of R&D, said Shire aimed “to accelerate delivery of therapies to patients from a highly productive internal pipeline, complemented by the acquisition of external assets and innovative collaborations”. The company benefitted from the collapsed takeover deal by AbbVie in October to the tune of over $1.6 billion, bringing it substantial “firepower” to back its own acquisition plans in rare diseases. It intends to effectively double its revenue to $10 billion by 2020.

Highlights in its pipeline include Fast Track designation from the US FDA for SHP607, a protein replacement therapy for the prevention of Retinopathy of Prematurity (ROP), which is a potentially blinding proliferative retinopathy unique to prematurely born infants. It is currently in a phase II study, with topline data expected in the second half of 2015.

Shire expects to file a New Drug Application in the US for Lifitegrast (SHP606) for the treatment of signs and symptoms of Dry Eye Disease in Q1 2015.

It is also conducting rare disease trials in paediatric patients with Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC), and in adult patients with Primary Biliary Cirrhosis.

An Investigational New Drug application was submitted to the US FDA in November for SHP626 in patients with Non-Alcoholic Steatohepatitis (NASH) with the aim of starting a phase Ib multiple-dose study in Q1 2015.


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