Vertex Kalydeco Study Successful In Children Aged 1-2 Years

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced that a phase III study evaluating its key cystic fibrosis (CF) drug, Kalydeco in children (age 1-2 years) met its primary safety endpoint.

Kalydeco (ivacaftor) is presently marketed for the treatment of patients (two years and above) with CF who have one of the 38 mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The ARRIVAL study was conducted in young children less than two years of age who have one of 10 mutations in the CFTR gene. Not only did it reveal that Kalydeco was well tolerated in children as young as one year, the study also showed improvement across multiple endpoints, including measures of pancreatic function. Based on this data, Vertex plans to file regulatory applications for approval of Kalydeco in children aged 1 to 2 years in the first quarter of 2018. The study, however, continues in infants less than one year of age.

If approved for use in this additional patient population, sales of Kalydeco may improve further. Please note that Kalydeco’s sales rose 35% in 2015, 11% in 2016 and 12% in the first nine months of 2017.

Year to date, Vertex’s shares have gained 88.3%, outperforming the increase of 0.3% for the industry.

For Vertex, 2017 so far has been a tremendous year with positive data read-outs and regulatory approvals leading to an increase in eligible patient population for its approved CF medicines — Kalydeco & Orkambi. With a consistent expansion in patient population, Vertex’s CF product revenues have increased quarter to quarter throughout 2017.

While Vertex raised its guidance for Orkambi once, that for Kalydeco was upped twice this year. The company is optimistic that the positive trend of CF sales will continue in 2018 as it gains additional reimbursement approvals for Orkambi in ex-U.S. markets and gets approval to launch tezacaftor(VX-661) /ivacaftor combination medicine.

Meanwhile, the company has also made decent clinical progress across multiple CF studies this year with meaningful pipeline catalysts lying ahead.

Vertex carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biotech/genetics sector include Emergent BioSolutions Inc. (NYSE:EBS) , XOMA Corporation (NASDAQ:XOMA) and Myriad Genetics, Inc. (NASDAQ:MYGN) , all with a Zacks Rank #1 (Strong Buy).You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent BioSolutions’ earnings estimates have moved up by 1.4% for 2018 over the last 30 days. Share price of the company has risen 29.2% year to date.

Myriad Genetics’ shares have risen 98.4% year to date while 2018 earnings estimates have risen by 2.5% over the past 30 days.

XOMA shares have risen a massive 688.6% year to date while 2018 loss estimates have narrowed by almost 20% over the past 30 days.

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