Collaborators launch clinical trial for cystic fibrosis

 

AbbVie, which was spun off from Abbott Laboratories earlier this year, will pay $45 million up front for rights related to the alliance, while Galapagos will be eligible for as much as $360 million in milestone payments, the company announced Sept. 24.

 

The goal of the collaboration is to identify compounds that correct defects in expression of and/or increase the activity of the main mutations in the cystic fibrosis transmembrane regulator protein. The companies plan to initiate Phase I studies at the end of next year. Once a drug receives approval, AbbVie would be responsible for commercial activities, while Galapagos would retain exclusive rights in China and South Korea and co-promotion rights in Belgium, the Netherlands and Luxembourg.

 

There is no cure for cystic fibrosis, but close monitoring and early, aggressive intervention can ease symptoms. Managing cystic fibrosis is complex and consists of preventing and controlling lung infections, loosening and removing mucus from the lungs, preventing and treating intestinal blockage and providing adequate nutrition. The market for CF thus is quite competitive.

 

“Cystic fibrosis has become a Big Pharma field,” Freya Vervoort, investor relations officer at Galapagos, tells DDNews. “With the deal with AbbVie, we gained the power needed to compete, and we increase the chance of developing successful CF therapies and to come up with the best CF therapy. AbbVie has the disease knowledge, experience in CF, development expertise, commercial strength and global reach.”

 

Galapagos initiated its research in CF in 2005 as part of a collaboration with the Cystic Fibrosis Foundation.

 

“Finding new medicines to fight CF and ultimately cure this disease is the driving force behind the Cystic Fibrosis Foundation,” the foundation’s website states. “To achieve this goal, we aggressively invest in drug development research with the most successful and innovative biotech companies and scientists in the world. In keeping with this business model, Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the foundation’s nonprofit drug development affiliate, customarily enters into contractual agreements with pharmaceutical companies to receive royalties after the approval and/or sale of certain drugs that are developed as a result of CFFT funding. Any royalties CFFT receives are reinvested in support of our mission.”

 

In 2010, Galapagos decided to pursue CF as the first orphan disease in which the company is attempting to discover, develop and launch its own medicines. Since then, Galapagos has developed small-molecule therapies that can restore the function of the defective CF protein (CFTR).

 

Vertex Pharmaceuticals was the first company to make it to market with a CFTR-targeting treatment, but “that therapy can only treat the G551D mutation, which is present in around 4 percent of CF patients,” Vervoort says. “Our aim is to offer superior novel oral therapies to CF patients with a broad range of mutations, including G551D and F508del.”

 

 Both companies contributed funding and science, and AbbVie and Galapagos will closely collaborate in the discovery phase, she says. Galapagos will be responsible for the preclinical phase up to Phase II. Then AbbVie takes over, moving it through Phase III and handling the majority of the marketing and sales activities.

 

“Galapagos is very pleased to join forces with AbbVie in this exciting new area of CF,” Onno van de Stolpe, CEO of Galapagos, stated in a news release. “Our programs in CF show promise. Partnering with AbbVie allows us to ramp up our commitment significantly, share development risk and expertise and increase our chances of bringing best-in-class therapies to CF patients.”

 

Jim Sullivan, president of AbbVie, added, “We’re pleased to enhance our partnership with Galapagos to include research in cystic fibrosis, a debilitating disease with significant unmet medical need. Our knowledge of the patient experience, combined with innovative advances in the understanding of disease etiology, offers the potential for new transformational treatments.”