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Vertex Looks to Expand CF Pipeline with Concert's Deuterated Ivacaftor Candidate

Vertex Pharmaceuticals agreed a potentially $250 million deal to acquire global rights to Concert Pharmaceuticals’ mid-stage cystic fibrosis (CF) therapy candidate CTP-656. Under terms of the proposed deal, Vertex would pay Concert $160 million upfront and pot

Written by Peptide Therapy Guide Editorial Team
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Vertex Pharmaceuticals agreed a potentially $250 million deal to acquire global rights to Concert Pharmaceuticals’ mid-stage cystic fibrosis (CF) therapy candidate CTP-656. Under terms of the proposed deal, Vertex would pay Concert $160 million upfront and potentially another $90 million in milestones, dependent on regulatory approval of CTP-656 in the U.S. and reimbursement in the U.K., Germany, or France. Vertex would also acquire rights to Concert’s other CF research and preclinical programs. The proposed terms have been approved by Concert’s board but are still subject to approval by the firm’s shareholders,

Concert has used its DCE® deuterium chemistry platform to develop the cystic fibrosis transmembrane conductance regulator (CFTR) potentiator CTP-656 as a deuterated form of Vertex’s existing CF therapy, Kalydeco® (ivacaftor), which is approved in the U.S., Europe, Canada, and Australia for treating CF patients with specific CFTR gene mutations. Concert claims CTP-656 could potentially be used as part of once-daily combination CFTR modulator therapies.

“Our vision is to develop the most effective and convenient medicines for people with CF,” said Jeffrey Chodakewitz, M.D., evp and CMO at Vertex. “We look forward to exploring once-daily regimens that combine CTP-656 with other potential medicines from our broad CF pipeline that treat the underlying cause of the disease.”

Vertex’s approved CF therapies include Kalydeco and Orkambi® (lumacaftor/ivacaftor). Clinical development of Orkambi in children from aged 2 years and over is ongoing, and the firm has prevously said it expects to file a marketing approval application for the use of Orkambi in CF patients aged 6 to 11 years during the first half of 2017.

Vertex reported total CF product revenues of $1.68 billion in 2016, compared with $982.3 million for 2015. Net product revenues from sales of Orkambi were $979.6 million in 2016, compared with $350.7 million in 2015. Orkambi was launched in the U.S. in July 2015. Net product revenues from Kalydeco were $703.4 million in 2016, up from $631.7 million in 2015.

Vertex has five CF candidates in its clinical pipeline. An NDA filing for lead candidate tezacaftor (VX-661) is projected for later in 2017, subject to results from Phase III studies, which are expected during the first half of the year. A Phase III trial with tezacaftor in children aged 6 to 11 years is also ongoing.

In October 2016, Vertex expanded its long-term collaboration with Cystic Fibrosis Foundation Therapeutics, and in mid-2016 announced a potentially $315 million deal with Moderna, to develop CF treatments based on Moderna’s messenger Ribonucleic Acid (mRNA) Therapeutics™. In January 2017, Vertex reported the acquisition by Merck KGaA of two clinical, and two preclinical anticancer programs for $230 million upfront.

Concert says the Vertex deal would give it the financial resources to fully fund the development of its proprietary candidate CTP-543 into pivotal clinical trials for treating alopecia areata. CTP-543 is an oral JAK1/JAK2 inhibitor that Concert has developed using its deuterium chemistry as a modified form of Incyte’s Jakafi® (ruxolitinib). Jakafi is approved in the U.S. for treating myelofibrosis and polycthemia vera, and in June 2016 received breakthrough designation by FDA for treating acute graft-versus-host disease (GvHD). Concert says it projects starting a Phase IIa study with CTP-543 for the alopecia areata indication during the first quarter of 2017.

The firm has ongoing collaborations with Jazz Pharmaceuticals for development of the clinical candidate JZP-386 (deuterated sodium oxybate) as a treatment for narcolepsy and with Celgene for the development of CTP-730 (deuterated apremilast) for treating inflammatory diseases. Deuterated dextromethorphan (AVP-786) is being developed in partnership with Teva (previously Avanir), and is in Phase III development for the indication of Alzheimer’s disease agitation and in Phase II development for multiple neurologic/psychiatric indications.

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Related questions

01What you can do

You might want to take a friend or family member with you to the appointment to help you remember information. Before your appointment, make a list of: Symptoms and when they started. Include anything that makes symptoms worse or better. All medicines, vitamins, herbs and supplements that you or your child take. Include the doses. Family history, such as whether anyone in your family has cystic fibrosis. Treatment you or your child have had for CF, if any. Include what the treatment was and if it helped. Any other medical conditions and their treatments. Questions to ask your healthcare professional. Questions to ask may include: What is likely causing these symptoms? What kinds of tests are needed? What treatment do you recommend? I or my child have other health conditions. How will cystic fibrosis affect them? Are there any limits needed? Feel free to ask other questions during your appointment.

Source: www.mayoclinic.org ↗
02What Is Cystic Fibrosis?

Cystic fibrosis (CF) is a genetic disorder, which means you get it from your parents at birth. It affects your lungs, pancreas, and other organs. CF changes the way chloride (salt) moves through the cells of your body. This causes the mucus (which should be thin and slippery) in various organs to become thick and sticky. Over time, this thick mucus builds up inside your airways, making it hard to breathe. The mucus traps germs and leads to infections and inflammation. It can also cause severe, long-term damage to the lungs and lead to respiratory failure (inability to breathe normally) and death. In the pancreas, the thick mucus caused by CF prevents the release of digestive enzymes when you eat. This leads to malnutrition and poor growth. CF can also cause liver disease, reproductive problems, and cystic fibrosis-related diabetes (CFRD). More than 40,000 people in the U.S. live with CF. Doctors diagnose about 1,000 new cases each year. Today, more than half of the CF population is aged 18 or older, and new treatments have expanded the life expectancy by decades.

Source: www.webmd.com ↗
03When to see a doctor

If you or your child has symptoms of cystic fibrosis — or if someone in your family has CF — talk with your healthcare professional about testing for the condition. Make an appointment with a doctor who has skills and experience in treating CF. CF requires regular follow-up with your healthcare professional, at least every three months. Call your healthcare professional if you have new or worsening symptoms, such as more mucus than usual or a change in the mucus color, lack of energy, weight loss, or severe constipation. Get medical care right away if you're coughing up blood, have chest pain or trouble breathing, or have severe stomach pain and bloating. Call 911 or your local emergency number or go to the emergency department at a hospital if: You're having a hard time catching your breath or talking. Your lips or fingernails turn blue or gray. Others notice that you're not mentally alert.

Source: www.mayoclinic.org ↗
04How Strong Is the Evidence for Alyftrek?

Based on the current clinical studies, Alyftrek is a safe and effective treatment for people with cystic fibrosis. The Cystic Fibrosis Foundation published a CFTR modulator therapy care guideline in 2018. Alyftrek is not included in these guidelines since it was approved by the FDA after these guidelines were published.

Source: www.webmd.com ↗
05What is it used for?

A sweat test is used to diagnose cystic fibrosis (CF).

Source: medlineplus.gov ↗
Research context

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Research and Statistics: Who Has Cystic Fibrosis?

About 40,000 people are living with cystic fibrosis in the United States, and there are approximately 105,000 people with CF worldwide. (3) More than 75 percent of people with the disease are diagnosed by age 2, and more than half of all people living with cystic fibrosis are 18 or older. CF occurs predominantly in white populations, at a rate of 1 in 2,500 births. Between 2 and 5 percent of white people are carriers of the CFTR gene variant but have no overt clinical signs of disease. The disease is less common among African Americans, occurring at the much lower frequency of approximately 1 out of 17,000 births. (15) CF gene variants are most prevalent in persons of northern and central European ancestries or of Ashkenazi Jewish descent. They are rarely found in Native Americans, Asians, or native Africans. (16) CF is equally common among men and women, but women patients fare significantly worse than male patients with the disease. The median survival age for female CF patients is about three years younger than it is for men, but the reasons for the poorer survival rates among women are not completely understood. (17)

Source: everydayhealth.com ↗
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