Business Wire

Vertex Announces Positive Day 90 Data for the First Patient in the Phase 1/2 Clinical Trial Dosed With VX-880, a Novel Investigational Stem Cell-Derived Therapy for the Treatment of Type 1 Diabetes

Share

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive Day 90 data for the first patient from the Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for people with type 1 diabetes (T1D). This is the first demonstration of a patient with T1D achieving robust restoration of islet cell function from such a cell therapy.

The patient was treated with a single infusion of VX-880 at half the target dose in conjunction with immunosuppressive therapy. The patient achieved successful engraftment and demonstrated rapid and robust improvements in multiple measures, including increases in fasting and stimulated C-peptide, improvements in glycemic control, including HbA1c, and decreases in exogenous insulin requirement. VX-880 was generally well tolerated.

“These results from the first patient treated with VX-880 are unprecedented. What makes these results truly remarkable is that they were achieved with treatment at half the target dose,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. “While still early, these results support the continued progression of our VX-880 clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression.”

“As a surgeon who has worked in the field of islet cell transplantation for decades, this approach, which obviates the need for an organ donor, could be a game changer,” said James Markmann, M.D., Ph.D., Professor of Surgery and Chief of the Division of Transplant Surgery at Massachusetts General Hospital. “We are excited to progress this unique and potentially transformative medicine through clinical trials and to patients.”

“More than a decade ago our lab had a vision for developing an islet cell replacement therapy to provide a functional cure to people suffering from T1D,” said Doug Melton, Ph.D., Xander University Professor at Harvard and an Investigator of the Howard Hughes Medical Institute. “These promising results bring great hope that stem cell-derived, fully differentiated islet cells could deliver a life-changing therapy for people who suffer from the relentless life-long burden of T1D.”

Efficacy Results

The patient was diagnosed with T1D approximately 40 years ago and has been dependent on exogenous insulin. In the one year prior to treatment, the patient experienced 5 severe, potentially life-threatening hypoglycemic episodes. Prior to treatment with VX-880, the patient’s insulin dose was 34 units per day and fasting and stimulated C-peptide levels were undetectable, indicating that the patient was not making their own insulin. Per the study protocol, the patient received half the target dose of VX-880 through a hepatic portal vein infusion in combination with a standard regimen of immunosuppressive agents.

Fasting C-peptide, HbA1c and 7-day average daily insulin dose were measured at various intervals after VX-880 treatment through Day 90. Fasting C-peptide was detected early after treatment with VX-880 and increased rapidly to Day 90. In parallel, HbA1c and daily insulin dose decreased over time.

Islet cell function was evaluated at baseline and at Day 90 using a Mixed Meal Tolerance Test (MMTT) with quantification of C-peptide levels, a direct marker for insulin production. At baseline prior to VX-880 treatment, fasting and stimulated C-peptide levels were undetectable, indicating no endogenous insulin production. At Day 90 after VX-880 treatment, fasting C-peptide was 280 pmol/L, reflecting restored basal insulin production and increased after MMTT stimulation to a peak of 560 pmol/L, indicating that VX-880 restored glucose-responsive insulin production. Also at Day 90, HbA1c improved from 8.6% at baseline to 7.2%, and daily insulin dose decreased from 34 units per day prior to treatment with VX-880 to an average dose of 2.9 units per day over a 7-day period at the Day 90 visit, reflecting a 91% decrease in daily exogenous insulin use.

Baseline and Day 90 Measures of Islet Cell Function for Patient 1

Baseline before

VX-880 infusion

Day 90 after

VX-880 infusion

Fasting C-peptide (pmol/L)

Undetectable*

280

Peak Stimulated C-peptide with MMTT (pmol/L)

Undetectable*

560

HbA1c (%)

8.6

7.2

Daily insulin dose (units/day)**

34

2.9

*The lower limit of quantitation of the C-peptide assay is 13 pmol/L.
**Daily insulin dose for baseline was measured on Day -3 prior to VX-880 infusion. For Day 90 post-infusion, average daily insulin dose was calculated over a 7-day period.

Safety Results

In this first patient, the safety of VX-880 was generally consistent with the immunosuppressive regimen used in this study. There were no serious adverse events (SAE) considered related to VX-880, and the majority of the adverse events were considered mild to moderate. The most common adverse events were severe hypoglycemic events, which were non-serious, not related to VX-880, and occurred in the perioperative period. Through Day 90, the patient had one SAE; this was a rash that was mild in severity, not related to VX-880, and resolved.

Next Steps

Based upon these data, Vertex plans to continue to progress the Phase 1/2 program for VX-880. There are multiple active sites in the U.S., and the Clinical Trial Application has been approved in Canada. Vertex is also progressing IND-enabling studies for its encapsulated islet cell program, which would potentially eliminate the requirement for immunosuppression, and plans to file an IND for this program in 2022.

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 chronic 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. The first two patients will be treated with half the target dose, followed by dose escalation to the target dose in the subsequent patients. Approximately 17 patients will be enrolled in the clinical trial. Enrollment is ongoing in this study.

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 patients 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 medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. 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, (i) statements by Bastiano Sanna, Ph.D., Dr. James Markmann, and Doug Melton, Ph.D. in this press release, (ii) our plans, expectations for, and the potential benefits of VX-880, (iii) our plans to continue to progress the Phase 1/2 program for VX-880 and IND-enabling studies for the encapsulated islet cell program, including anticipated regulatory filings in 2022, and (iv) our plans for dosing and enrollment of patients. 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 one patient 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)

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, +1 617-341-6108
or
Brenda Eustace, +1 617-341-6187
or
Manisha Pai, +1 617-429-6891

Media:
mediainfo@vrtx.com
or
U.S.: +1 617-341-6992
or
Heather Nichols: +1 617-839-3607
or
International: +44 20 3204 5275

About Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

KPMG Reports Global Revenues of $32.13 Billion for Fy21, Focus on Strategy Enables 10% Growth9.12.2021 01:01:00 CET | Press release

KPMG International announced today annual aggregated revenues for KPMG firms globally of $32.13 billion for the fiscal year ending 30 September 2021 (FY21). Reporting a 10% increase in US dollar revenues from FY20, this follows an extensive period of investment and focus on priority services and solutions addressing the challenges faced by businesses across the globe. Strong growth across functions: Advisory growing at 17%, Audit at 4% and Tax & Legal Services at 8%. During FY21, the global organization committed to a more than $1.5 billion investment to focus on a multi-year program to accelerate the delivery of its global environmental, societal and governance (ESG) plan and solutions. KPMG firms are focused on building an inclusive and diverse culture and have increased female diversity among leadership ranks to 27% of partners and directors. “2021 was a strong year for KPMG. We achieved outstanding growth in a challenging business climate, unveiled KPMG: Our Impact Plan to help dri

New Global Survey Finds Consumers Are Becoming More Selective About Where, When and How They Share Information with Brands9.12.2021 01:01:00 CET | Press release

The mobile app experience company Airship has today published a new report which highlights new consumer behaviour on mobile and their expectations from brands. Based on a survey of more than 9,000 consumers across seven countries, including the U.K, France, Germany, U.S, Australia, Singapore and India, the report found that 40% of global respondents are more likely to continue receiving brand communications if they are given controls over purpose, frequency and channel. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211208006131/en/ Information consumers are willing to share with brands for personalised interactions and special incentives (Graphic: Business Wire) The survey results show a strong and growing consumer preference for mobile apps, with 70% of UK respondents on average using apps across all industries more or about the same since the pandemic began — highlighting the opportunity to establish positive feedback lo

Seagen Announces Updated Results from Pivotal HER2CLIMB Trial Evaluating TUKYSA ® (tucatinib) in Patients with HER2-Positive Breast Cancer with Brain Metastases9.12.2021 00:00:00 CET | Press release

Seagen Inc. (Nasdaq:SGEN) today announced the presentation of new data from exploratory analyses from the pivotal HER2CLIMB trial showing that improvement in overall survival (OS) was maintained after an additional 15.6 months of follow-up when TUKYSA® (tucatinib) was combined with trastuzumab and capecitabine in patients with HER2-positive metastatic breast cancer (MBC) who had stable or active brain metastases. The data were featured today in a spotlight poster (Abstract #PD4-04) at the 2021 San Antonio Breast Cancer Symposium (SABCS). “The risk of breast cancer spreading to the brain is more pronounced for patients with aggressive subtypes of breast cancer, including HER2-positive breast cancer,” said Nancy U. Lin, M.D., Director of the Metastatic Breast Cancer Program in the Susan F. Smith Center for Women’s Cancers at Dana-Farber in Boston, MA. “These analyses provide a hopeful outcome for patients with HER2-positive metastatic breast cancer when cancer has spread to the brain as

IFF Declares Dividend for Fourth Quarter 20218.12.2021 22:15:00 CET | Press release

IFF (NYSE: IFF) announced that its Board of Directors has declared a regular quarterly cash dividend of $0.79 per share of its common stock, payable on January 7, 2022 to shareholders of record as of December 28, 2021. Welcome to IFF At IFF (NYSE: IFF), an industry leader in food, beverage, scent, health and biosciences, science and creativity meet to create essential solutions for a better world – from global icons to unexpected innovations and experiences. With the beauty of art and the precision of science, we are an international collective of thinkers who partners with customers to bring scents, tastes, experiences, ingredients and solutions for products the world craves. Together, we will do more good for people and planet. Learn more at iff.com, Twitter, Facebook, Instagram, and LinkedIn. View source version on businesswire.com: https://www.businesswire.com/news/home/20211208006055/en/Contact information Michael DeVeau Chief Investor Relations & Communications Officer 212.708.71

Walton Global Announces a $63.7 Million Distribution to Investors8.12.2021 21:00:00 CET | Press release

Walton Global, a real estate investment and land asset management company with US$3.4 billion under management is pleased to announce a total of CAD $63.7 million in distributions that will be made to investors within various corporations: WIGI Restructured Bond Corporation (WIGI RBC), McConachie Management Corporation and Roll-up Corporation (RUC). The distributions are scheduled throughout the month of December 2021. Roll-Up Corporation (RUC) A distribution of approximately CAD $55 million was approved on November 15, 2021, by the Walton-managed Roll-Up Corporation (RUC). The distribution was paid to shareholders of RUC on December 1, 2021. This is the third distribution made to investors in RUC, with this distribution representing $0.082 per share. RUC investors have received a total of CAD $104 million in distributions since the formation of the entity in 2018. RUC was formed through the amalgamation of 134 Canadian pre-development land entities and has more than 28,141 shareholder

KartRider: Drift Closed Beta Starts Today8.12.2021 20:58:00 CET | Press release

The next entry in Nexon’s phenomenally successful KartRider racing franchise is here! Cross-platform sensation, the free-to-play KartRider: Drift, will launch its Closed Beta at 4pm Pacific today, ending on December 15th at 5am Pacific. Access to the KartRider: Drift Closed Beta is available for PC on Nexon’s launcher and Steam, in addition to being available to console players on PlayStation®4 and Xbox One. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211208006024/en/ KARTRIDER: DRIFT CLOSED BETA STARTS TODAY - Closed Beta for Online Cross-Platform, Free-to-Play Party Racing Game Kicks Off (Graphic: Business Wire) Gearing up to deliver new thrills in the beloved Nexon racing franchise, KartRider: Drift offers unparalleled character and kart customization. Karts can be tailored as never before, allowing players to transform their rides to reflect their inner racing persona. To help make sure your ride represents who you ar

Rimini Street Wins Frost & Sullivan Best Practices Award for Third-Party Enterprise Software Support Services Company of the Year8.12.2021 19:00:00 CET | Press release

Rimini Street, Inc. (Nasdaq: RMNI), a global provider of enterprise software products and services, the leading third-party support provider for Oracle and SAP software products and a Salesforce partner, today announced it has been awarded the 2021 Asia Pacific Best Practices Award for Third-Party Enterprise Software Support Services Company of the Year by leading research and consulting firm Frost & Sullivan. The recognition recognizes Rimini Street's commitment to help clients maximize returns on their enterprise software and technology investments to better enable innovation to drive competitive advantage and growth. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211208005919/en/ Rimini Street Wins Frost & Sullivan Best Practices Award for Third-Party Enterprise Software Support Services Company of the Year (Graphic: Business Wire) Award Honors Innovation in Client Service Excellence The Frost & Sullivan Best Practices Aw