Vertex to End Phase II Study on AAT Deficiency Candidate

Vertex Pharmaceuticals Incorporated VRTX announced that it has stopped dosing and will discontinue the phase II study on its investigational oral small molecule corrector, VX-814, for the treatment of alpha-1 antitrypsin deficiency (AATD), an inherited condition that raises risk for lung and liver disease.

The company took the decision to stop dosing in the phase II study based on the safety and pharmacokinetic (PK) profile of VX-814 observed in the study to date. The study was being conducted to evaluate the ability of VX-814 to increase functional levels of alpha-1 antitrypsin, a protein mainly produced by the liver, over 28 days of dosing. However, elevated liver enzymes were observed in several patients, which were more than normal limits. Meanwhile, exposure levels were low as observed in the analysis of PK data from the study, which the company believes would make it impractical to safely reach targeted exposure levels and thus meaningful increases in AAT levels.

Vertex's shares were down almost 12% in pre-market trading on Thursday in response to the pipeline setback. Vertex’s stock has risen 24% this year so far compared with an increase of 3.1% for the industry.


Vertex has another AATD small molecule corrector in its pipeline, VX-864, for which a phase II study was initiated in July. This phase II study continues to enroll and dose patients. Data from the study are expected in the second half of next year.

The company’s main focus is on the development and marketing of its cystic fibrosis franchise. However, it also has a rapidly advancing early-stage portfolio in other specialty disease areas. Other than AATD, these disease areas include pain, sickle cell disease, beta-thalassemia and APOL1-mediated kidney diseases.

In sickle cell disease and thalassemia, Vertex is co-developing a gene-editing treatment, CTX001, in partnership with CRISPR Therapeutics CRSP. Meanwhile, enrollment is underway in a phase II study on VX-147 in APOL1-mediated focal segmental glomerulosclerosis (FSGS).

Zacks Rank and Other Stocks to Consider

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

Some better-ranked stocks from the biotech sector include Emergent Biosolutions EBS and Horizon Therapeutics HZNP. While Emergent has a Zacks Rank #1, Horizon Therapeutics has a Zacks Rank #2.

Emergent Biosolutions’ earnings per share estimates have moved up from $6.17 to $8.42 per share for 2021 in the past 30 days. The stock has risen 81.8% so far this year.

Horizon Therapeutics’ earnings per share estimates have increased from $2.83 to $2.87 per share for 2020 and from $4.28 to $4.38 for 2021 in the past 30 days. The stock has surged 121.8% so far this year.

The Hottest Tech Mega-Trend of All                

Last year, it generated $24 billion in global revenues. By 2020, it's predicted to blast through the roof to $77.6 billion. Famed investor Mark Cuban says it will produce ""the world's first trillionaires,"" but that should still leave plenty of money for regular investors who make the right trades early.

See Zacks' 3 Best Stocks to Play This Trend >>

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
Vertex Pharmaceuticals Incorporated (VRTX): Free Stock Analysis Report
Emergent Biosolutions Inc. (EBS): Free Stock Analysis Report
Horizon Therapeutics Public Limited Company (HZNP): Free Stock Analysis Report
CRISPR Therapeutics AG (CRSP): Free Stock Analysis Report
To read this article on click here.
Zacks Investment Research

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

More Related Articles

Sign up for Smart Investing to get the latest news, strategies and tips to help you invest smarter.