Vertex Pronounces FDA Clearance of Investigational New Drug (IND) Software for VX-880, a Novel Cell Remedy for the Therapy of Sort 1 Diabetes (T1D)

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BOSTON–(BUSINESS WIRE) – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the US Food and Drug Administration (FDA) has approved the IND to initiate a clinical trial for VX-880, an investigational study based on stem cells . fully differentiated pancreatic islet cell therapy for the treatment of T1D. Vertex plans to initiate a Phase 1/2 clinical trial in the first half of 2021 in patients with T1D with compromised hypoglycemic awareness and severe hypoglycemia.

“As we celebrate the 100th anniversary of the discovery of insulin this year, we are excited to bring to the clinic a world-class cell therapy that has the potential to make meaningful impact on people with T1D,” said Bastiano Sanna, Ph.D. ., Executive Vice President and Head of Cell and Gene Therapies at Vertex. “We look forward to embarking on our clinical program and testing our unique approach to replacing pancreatic islet cells destroyed in people with type 1 diabetes with our stem cell-derived, fully differentiated insulin-producing pancreatic islet cells.”

About VX-880

VX-880, formerly known as STx-02, is an allogeneic, human stem cell-derived, islet cell therapy being evaluated for patients with T1D with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 has the potential to restore the body’s ability to regulate glucose levels by restoring the function of pancreatic islet cells, including the production of insulin.

The VX-880 clinical trial includes the infusion of fully differentiated, functional islet cells and the chronic administration of concomitant immunosuppressive therapy to protect the islet cells from immune rejection.

About the clinical phase 1/2 study

The clinical trial is a phase 1/2, single-arm, open-label study in T1D patients with impaired hypoglycemic awareness and severe hypoglycemia. This will be a sequential, multi-part clinical trial to evaluate the safety and effectiveness of various doses of VX-880. Approximately 17 patients will be enrolled in the clinical trial.

About type 1 diabetes

T1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, resulting in a loss of insulin production and impaired blood sugar control. The lack of insulin creates abnormalities in the way the body processes nutrients, resulting in high blood sugar levels. High blood sugar can lead to diabetic ketoacidosis and, over time, complications such as kidney disease / failure, eye disease (including vision loss), heart disease, stroke, nerve damage, and even death. The limitations and complexities of insulin delivery systems can make it difficult to achieve and maintain a balance in glucose control in patients with T1D. Hypoglycemia remains a critical limiting factor in the management of blood sugar, and severe hypoglycemia can lead to loss of consciousness, coma, seizures, and injury and be fatal.

There are currently limited treatment options beyond insulin for the treatment of T1D.

About vertex

Vertex is a global biotechnology company that invests in scientific innovation to develop transformative drugs for people with serious illnesses. The company has several drugs approved to treat the underlying cause of cystic fibrosis (CF) – a rare, life-threatening genetic disease – and has several ongoing clinical and research programs for cystic fibrosis. In addition to CF, Vertex has a robust pipeline of small molecule investigational drugs for other serious diseases that provide deep insight into causal biology in humans, including pain, alpha-1 antitrypsin deficiency, and APOL1-mediated kidney disease. In addition, Vertex has a rapidly growing pipeline of cell and gene therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Vertex was founded in 1989 in Cambridge, Massachusetts. Vertex’s global headquarters are now in the Boston Innovation District and its international headquarters are in London. The company also has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the best places to work in the industry, including being on Science magazine’s Top Employer List for 11 years in a row and Best Place to Work for LGBTQ Equality through the human rights campaign. Visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram for company updates and to learn more about Vertex’s history of innovation.

Special note on forward-looking statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, without limitation, statements by Bastiano Sanna, Ph.D., in this press release, statements regarding developments, plans and expectations for our T1D pipeline program including our plans to initiate a Phase 1/2 clinical trial in people with T1D and the expected schedule of our clinical trials, statements about patient enrollment and dosing, statements about potential clinical trial outcomes, and expected benefits of VX-880; and our plans to provide further updates to our T1D pipeline program. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent only the beliefs of the company as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to be material differ from those expressed or implied in such forward-looking statements. These risks and uncertainties include, among other things, that the FDA may not approve our IND, that data from a limited number of patients may not indicate definitive clinical trial results, that data from the company’s development programs may not support registration or continue to develop Safety, efficacy or other reasons why the COVID-19 pandemic may affect the status or progress of our clinical trials, as well as other risks identified under the heading “Risk Factors” in Vertex’s most recent annual report and subsequent quarterly reports that have been filed with the Securities and Exchange Commission at www.sec.gov and available on the company’s website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)