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Vertex Selects Triple Combo Regimen for Regulatory Submission

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Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced that it has selected the triple combination regimen of next-generation CFTR corrector VX-445 (elexacaftor), tezacaftor and Kalydeco (ivacaftor) for regulatory submissions globally. The company is developing the combination regimen for treating cystic fibrosis (CF) patients, aged 12 and older, with one F508del mutation and one minimal function mutation or with two F508del mutations.

The company is planning to file a new drug application in the third quarter of 2019, followed by a marketing authorization application in Europe in the fourth quarter of this year.

Investors cheered the news as shares of Vertex gained 2.2% on May 30. Vertex’s shares have increased 3.7% this year so far against the industry’s decrease of 0.2%.

Notably, Vertex was developing two triple combination regimens – either VX-445 or VX-659 in combination with tezacaftor and Kalydeco – simultaneously for a similar patient population with CF. Two 4-week and two 24-week phase III studies were evaluating the combination regimens separately.

Both regimens achieved unprecedented and statistically significant results in phase III studies in eligible patient population and had similar and positive benefit-risk profiles. However, Vertex chose the VX-445 triple combination regimen for regulatory submission based on detailed analysis of multiple factors, which showed that the regimen will be beneficial for larger patient population compared to the VX-665 combo.

Two phase III studies evaluating its VX-445 combo met the primary endpoint of significant improvement in lung function in patients suffering from CF. The improvement in lung function was measured as mean absolute improvements in percent predicted forced expiratory volume in one second or ppFEV1. Cystic fibrosis is a rare genetic disease that causes chronic lung infections and progressive lung damage.

In the 24-week study evaluating patients with one F508del mutation and one minimal function mutation, data showed that the triple combination regimen led to a mean absolute improvement in ppFEV1 of 13.8 percentage points from baseline through week four of treatment compared to placebo. The improvement was maintained over 24 weeks of treatment.

The 4-week study yielded a mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week four when VX-445 was added in patients already receiving tezacaftor and ivacaftor compared to placebo plus tezacaftor and ivacaftor.

Please note that many other companies like AbbVie (ABBV - Free Report) and Proteostasis Therapeutics, Inc. (PTI - Free Report) are also developing triple CFTR combinations for CF. Proteostasis Therapeutics is expected to report top-line data from its triple combo CF medicine in 2019.

Apart from its strong CF pipeline, Vertex is also developing other treatments targeting several indications and is focusing on gene therapies as well. Vertex has a partnership with CRISPR Therapeutics (CRSP - Free Report) to evaluate an investigational gene editing treatment, CTX001, for the two devastating diseases — sickle cell disease and thalassemia.

Zacks Rank

Vertex currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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