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Nasdaq 100 Biotechnology: Beyond Pure Tech; Still Pure Innovation

While the pure technology sector is the engine of innovation for much of what we consider the highest technology advancements of our age, there is a host of other industries represented in the Nasdaq-100 that are trailblazing. These companies that lie outside the pure tech sphere are some of the most innovative companies in the world today.

Biotechnology is a space that is undergoing rapid innovation. From medical devices that can turn major surgeries into office-visit procedures to gene therapy that can make the difference between life and death, biotechnology knows no end in the ways it can improve our lives and shape our world.

A Look Deeper: Biotech

Cited as one of the most innovative companies in the market today for four years straight by Forbes, Alexion (ALXN) is helping patients with rare and often overlooked diseases find hope.

It filed applications for approval with U.S. and E.U. regulatory authorities for Soliris (Eculizumab). Soliris treats patients with Refractory Generalized Myasthenia Gravis (gGM), a debilitating disease that weakens the muscles and leave patients unable to move speak, or breathe properly. Late last year, it initiated Phase 3 clinical trials of ALXN1210 to treat Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disease that is difficult to diagnose and often proved fatal before treatments were developed. Alexion was also named a top employer in both 2015 and 2016 by Science magazine.

Another top innovative company cited for the past three consecutive years by Forbes is BioMarin (BRMN), a San Rafael, California-based developer of treatments for rare diseases that have very small patient populations. These are often fatal genetic disorders that affect children. It has led the fight in using gene therapy to combat Hemophilia, making advancements and announcing an update to its proof of concept earlier this year.

As of late January, BioMarin was leading the market as the only company to have reached clinical trials for treatment of hemophilia A, according to LabioTech. Last year it enrolled its first participant in Phase 3 trials of its drug Vosoritide for treating children with Achondroplasia, which is the most common form of human dwarfism.

Incyte (INCY) also focuses on developing pharmaceutical products to treat rare diseases, focusing on treating cancer. It has shared innovative company honors on the Forbes list as well. Its product Jakafi is currently approved in the U.S. for treatment of intermediate or high-risk myelofibrosis and is being developed as a potential treatment for other forms of cancer. Earlier this year it announced it was collaborating with Calithera Biosciences to develop and commercialize CB-1158, a small molecule arginase inhibitor. Arginase is an enzyme that suppresses autoimmune functions that would otherwise help fight tumor growth. Preclinical trials have shown arginase inhibitors to help limit the size of tumors.

Tarrytown, N.Y.-based Regeneron (REGN) is also in the fight against serious diseases, and has taken a strong stand in favor of innovation. The company’s CEO Leonard Schleifer has said that biopharmaceutical companies need to focus on innovation more and less on pricing for future growth and that the focus on price has diminished the industry in the eyes of consumers, patients and political leaders. Pharmaceutical companies, particularly those that acquire existing drugs rather than develop their own, have drawn ire for high prices over the past several years. Schleifer pointed out that pharmaceutical companies will be viewed more kindly if they generate more of their profits from innovation rather than through raising prices on products.

Regeneron also ranks as one of Forbes’ most innovative companies. It has developed four FDA- approved medicines that help fight diseases such as cancer, asthma, arthritis and others. It also developed the VelociSuite technologies, manufacturing technologies and one of the world’s largest human sequencing efforts.

Vertex Pharmaceuticals (VRTX) is on the front line of the fight against cancer. It recently forged a licensing agreement with Merck for the worldwide development and commercialization of four different research programs to treat cancer. In late 2016 the company announced it had positive results from its Phase 3 study of its product Orkambi in children with Cystic Fibrosis. It noted that the results support its submission to the European Medicines Agency in the first half of this year. Also late last year it announced it would move to initiate Phase 2 studies of Cystic Fibrosis treatments with plans to further test another related product. Collaboration is key for Vertex and the company notes that its network is broad geographically and intellectually. It has research and development facilities in the U.S., Canada and the U.K., corporate offices in Boston and London, and 11 international offices in Europe and North America.

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


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

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