Vertex Presents Key Findings on Kalydeco, Orkambi & Experimental CF Therapies at NACFC

Vertex Presents Key Findings on Kalydeco, Orkambi & Experimental CF Therapies at NACFC

A series of new updates and future development plans were recently announced by Vertex regarding its approved and candidate drugs to treat Cystic fibrosis (CF). The announcements, made at the 29th Annual North American Cystic Fibrosis Conference (NACFC), highlighted the plans for two next-generation correctors, VX-152 and VX-440, as well as new updates and future clinical studies for its prescription medicines KALYDECO (ivacaftor) and ORKAMBI (lumacaftor/ivacaftor).

Vertex first announced the progression of two next-generation correctors, VX-152 and VX-440, into clinical development. Each of these compounds will be studied independently and in a triple combination with VX-661/ivacaftor. The phase 2 studies in CF patients will include CF patients with two copies of the F508del mutation, patients with just one copy of this mutation and one other mutation that results in a minimally functional CFTR protein. VX-152 and VX-440 were developed with the intent to improve the results observed with ivacaftor with only one corrector, namely the processing and transport to the surface of the CFTR protein. In vitro results in human bronchial epithelial cells with the three variations of  the mutation (two copies, one copy and another mutation resulting in minimal function) confirmed the potential of this triple combination approach with significantly increased CFTR function.

Vertex also presented new developments regarding two FDA-approved CF drugs: KALYDECO, a prescription drug recently approved for treatment of patients aged 2 years or older with one of ten specific CF gene mutations, and ORKAMBI, approved for CF treatment of patients 12 years or older with two copies of the F508del mutation.

Vertex announced that KALYDECO had been recommended by the EU Committee for Medicinal Products for Human Use (CHMP) for children ages 2 to 5 with one of the specific mutations. It also announced plans to extend patient eligibility. Now, the company will conduct clinical studies to study the effect of KALYDECO in children under 2 years of age. Other presentations on the drug included long-term safety data in which the ivacaftor group in a 5 year study presented significantly lower annual risk of death, hospitalization and pulmonary exacerbation. Results from an analysis of children aged 6 to 11 showed that ivacaftor treatment resulted in better growth.

ORKAMBI was also recommended by EU CHMP for treatment of people aged or older than 12 years with the mutation profile indicated for treatment. Vertex announced that currently there is an ongoing phase 3 trial to study the drug’s effect on children aged 6 to 11 years. Other presentations on the therapy highlighted that the treatment resulted in less pulmonary exacerbation episodes  in phase 3 studies.

Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex, commented on the results and on future work, “With continued expansion in the number of people eligible for KALYDECO, the recent FDA approval of ORKAMBI in the U.S. and the advancement of two next-generation correctors into clinical development, we are making significant progress toward our goal of treating the vast majority of people with CF. We are conducting studies with our investigational combination regimens in groups of CF patients with mutations that represent approximately 90 percent of all people with CF. While there is much work still to be done, we believe we are on the right path to further enhance the treatment of CF in the years ahead.”

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