Vertex Stock Hits All-Time High on Phase 2 Study Results, Gene Editing Agreement

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  • Shares of Vertex Pharmaceuticals hit an all-time high after pain treatment study results and announcement of gene-editing technology deal.
  • Phase 2 trial of its drug VX-548 showed “statistically significant and clinically meaningful results” reduced pain in people with painful diabetic peripheral neuropathy.
  • A licensing agreement with Editas Medicine will give Vertex access to Editas' solutions. gene editing tool.

Shares of Vertex Pharmaceuticals (VRTX) took a record hit Wednesday after announcing promising results from a study of its experimental treatment to reduce pain in diabetics and reaching a deal on gene editing technology.

Vertex indicated that a trial of phase 2 of its drug VX-548 “resulted in a statistically significant and clinically meaningful reduction.” in weekly average daily pain intensity in people with painful diabetic peripheral neuropathy (DPN).

Dr Roy Freeman, Professor of Neurology and Director of the Center for Autonomic and Peripheral Nerve Disorders at Beth Israel Deaconess Medical Center, and a member of the Vertex Pain Steering Committee, said that based on the results, VX-548 “could offer the potential for a new class of medicine for the millions of patients suffering from neuropathic pain.

Vertex Medical and Executive Director Vice President of Global Drug Development and Medical Affairs, Dr. Carmen Bozic, said the company is “working with urgency” to move VX-548 into Phase 3 testing.

Separately, Editas Medicine (EDIT) announced that Vertex will pay $50 million upfront, with the possibility of an additional $50 million, under a non-exclusive licensing agreement for the Editas Cas9 gene editing tool. Editas noted that Vertex will use Cas9 for treatments targeting sickle cell disease and beta thalassemia. This includes Casgevy, developed by Vertex and CRISPR Therapeutics (CRSP), which was approved by the Food and Drug Administration last week.

Vertex Pharmaceuticals shares have ended up 13% higher on Wednesday after the announcement and have gained more than 41% so far this year.


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