Vertex to Present New Data on VX-880 Clinical Trial at 82nd American Diabetes Association Annual Conference

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced three abstracts, including two oral presentations and one poster, detailing data on type 1 diabetes (T1D) and its Phase 1/2 trial of VX-880, a stem cell‑derived, fully differentiated pancreatic islet cell replacement therapy, have been accepted for presentation during the American Diabetes Association 82nd Annual Scientific Sessions Conference held June 3-7, 2022 in New Orleans, Louisiana.

Abstract and publication #259-OR entitled “Stem Cell-Derived, Fully Differentiated Islet Cells for Type 1 Diabetes,” will be presented on Monday, June 6, 2022 at 9:00 a.m. CDT during the oral presentation session “Other Therapeutic Agents,” which runs from 8:00 to 10:00 a.m. CDT. The abstract posted today details results from the Phase 1/2 clinical trial, demonstrating VX‑880 can restore insulin production and glucose control in T1D. Data will be updated and additional information will be presented at the conference.

Abstract and publication #92‑OR entitled “Persistence of Impaired Awareness of Hypoglycemia, Severe Hypoglycemic Events and Suboptimal Glycemic Control Despite Advanced Diabetes Technologies,” to be presented Saturday, June 4, 2022 at 1:45 p.m. CDT during the oral presentation session “Glucose Monitoring and Sensing,” which runs from 1:45 to 3:45 p.m. CDT. The abstract posted today, describing an online survey of 2,044 adults with T1D in the T1D Exchange registry and online communities, shows that in patients with high rates of diabetes technology adoption, such as continuous glucose monitoring, insulin pumps and hybrid closed loop systems, impaired awareness of hypoglycemia (IAH) and severe hypoglycemic events (SHEs) persist, with a substantial proportion of patients not achieving target glycemic control, indicating the need for novel T1D treatments.

Abstract and poster #652-P entitled “Gaps Remain in Achieving Target T1D Glycemic Goals Despite Advanced Technologies,” will be available as part of the general poster session to be held Saturday, June 4, 2022 from 11:30 a.m. to 2:30 p.m. CDT. The abstract posted today highlights that, despite improvements in glycemic control with diabetes technologies, many patients with T1D are still unable to achieve clinical targets and experience severe hypoglycemia, emphasizing the need for novel T1D treatments.

The accepted abstracts are now available on the ADA website.

About VX-880

VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology. VX‑880 is being evaluated for patients who have 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 pancreatic islet cell function, including glucose‑responsive insulin production. VX-880 is delivered by an infusion into the hepatic portal vein and requires maintenance immunosuppressive therapy to protect the islet cells from immune rejection.

About the Phase 1/2 Clinical Trial

The clinical trial is a Phase 1/2, multi‑center, single-arm, open-label study in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. This study is designed as a sequential, multi-part clinical trial to evaluate the safety and efficacy of VX-880. In Part A, the first two patients received half the target dose. In Part B, five patients will receive the full target dose, after which concurrent dosing at the full target dose will occur in Part C. Approximately 17 patients will be enrolled in the clinical trial. The Phase 1/2 study is on clinical hold in the U.S. and is ongoing in Canada.

About Type 1 Diabetes

T1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels. High blood glucose can lead to diabetic ketoacidosis and over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage and even death.

Due to the limitations and complexities of insulin delivery systems, it can be difficult to achieve and maintain balance in glucose control in people with T1D. Hypoglycemia often results because of the difficulty in balancing the different factors that impact glucose levels, including insulin, diet and exercise. Hypoglycemia remains a critical limiting factor in glycemic management, and severe hypoglycemia can cause loss of consciousness, coma, seizures, injury and can be fatal. Over time, patients with T1D can develop impaired awareness of hypoglycemia, meaning they are no longer able to perceive the early signs of a hypoglycemic event, which can be dangerous and result in life-threatening events.

Current standards of care do not address the underlying causes of the disease, and there are limited treatment options beyond insulin for the management of T1D.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding (1) the potential benefits, safety, and efficacy of VX‑880, (2) our plans to present data about VX‑880 at the American Diabetes Association Annual Conference, including data from our VX‑880 clinical trial and additional scientific presentations regarding VX‑880, and (3) our plans to continue to progress our Phase 1/2 program for VX‑880. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only 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 differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's research and development programs may not support registration or further development of its compounds due to safety, efficacy, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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