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Vertex in Streamlined Clinical Trials for New Diabetes 1 Treatment

Vertex Boston, MA Office at Fan Pier.

With Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) presenting new data from its Phase 1/2 clinical trial of VX-880, a new stem cell based treatment for diabetes, it is natural to assume it to be a contender of Novo Nordisk’s (NYSE:NVO) blockbuster drug Ozempic.

Vertex, a global biotech firm specializing in small molecule treatments, ranging from Hep C and HIV treatments to cystic fibrosis (CF), are at the forefront of advancements in CF and have transformed the standard of care for patients with the disease. On the horizon is gene-editing in addition to its already established rational approach to pharmacology.

While quickly becoming an investor favorite and accumulating much needed momentum in its respective field, Vertex is still not setting out to usurp Novo Nordisk from the throne in the court of endocrinological disorders.

VX-880 is a cell therapy intended for use in people with type 1 diabetes, while Ozempic is intended for type 2. Type 2 diabetes accounts for 90% of diabetes cases.

Type 1 diabetes is an illness caused in part by autoimmune dysfunction that leads to reduced efficiency and premature death of islet cells, which are responsible for producing insulin to regulate a person’s blood sugar.

In a nutshell, the therapy’s concept is to take stem cells from healthy donors and force them to differentiate into islet cells, which typically reside in the pancreas. Then, those islet cells are infused into the patient, where they subsequently migrate to their normal location and engraft there.

Per this month’s press release “Nearly all participants (11 of 12) had a reduction or elimination of exogenous insulin use at their last visit.”

Vertex, with its new gene therapy Casgevy for treatment of sickle cell disease hitting the market recently, alongside other programs like it that may soon attain regulatory approval, has made waves in the pharmaceutical complex. In the near future they will even be providing a non-opioid candidate for treating acute pain. It’s clear that Vertex has a long runway for more growth and seems to be a conservative investment for the foreseeable future.