Vertex Pharmaceuticals, Inc., a pharmaceutical company known for discovering, developing and commercializing novel medicines for serious diseases with largely unmet clinical needs such as cystic fibrosis (CF), recently revealed the company’s corporate outlook for the year 2015, prior to their scheduled presentation at the highly-anticipated 33rd J.P. Morgan Healthcare Conference in San Francisco. The company’s recent corporate updates center on their pipeline therapeutics aimed at addressing CF.
The Company’s corporate updates on solutions for CF in 2015 are as follows:
Progress on Kalydeco (ivacaftor)
The company did not disclose the results of the Phase II clinical trial for combined CF drug VX-661/Kalydeco, as the findings from this 12-week study are due towards the quarter’s end. However, plans to launch a Phase III project for the combined drug have been set forFebruary 2015. While their previous timeframe with regard to this study was originally pegged within the first half of the year, the company intends to accelerate the schedule after obtaining encouraging short-term findings on VX-661’s performance, and after consulting with the US Food and Drug Administration.
The upcoming Phase III program plans to treat CF patients carrying the 508del CFTR mutation with the VX-661/Kalydeco formulation, with subsequent clinical trials due to open come Q2 2015 for the 508del mutation and another undisclosed mutation. All mentioned trials will run for 8 to 12 weeks, with top-line results expected towards the year’s end.
US FDA Ruling on Kalydeco/Lumacaftor
Vertex is currently still awaiting the US FDA’s decision on Kalydeco/lumacaftor, another combined CF drug for patients carrying the 508del mutation, which is expected on July 5, 2015. There is a good chance the agency will release an early decision, as the combined drug has achieved accelerated review in Europe.
Vertex Pharma Finances
“Vertex enters a year where the potential approval and launch of the combination of lumacaftor and ivacaftor, and continued geographic and label expansion for KALYDECO, are expected to significantly increase the number of people treated with our medicines,” said the Company’s Chairman, President and Chief Executive Officer, Jeffrey Leiden, M.D., Ph.D. “We enter 2015 with a strong cash position of approximately $1.4 billion, which, when combined with continued revenue growth, will allow us to invest in key internal and external opportunities in CF and other diseases to bolster our pipeline and position the company to advance other transformative medicines in the coming years.”
The Company is expecting Kalydeco’s 2015 net sales to play around $560-580 million – numbers quite short of their original projection of $675 million.