ViaCyte Reports Compelling Preliminary Clinical Data from Islet Cell Replacement Therapy for Patients with Type 1 Diabetes

  • PEC-Direct (VC-02) demonstrates first instance showing glucose-responsive insulin production, C-peptide of 0.8 ng/mL, from implanted pancreatic progenitor cells in humans
  • Therapeutic effect showing increase to 88% time in range with corresponding reduction of HbA1C from 7.4% to 6.6% 
  • Late-breaking results presented at the American Diabetes Association’s 81st Scientific Sessions 

SAN DIEGO, June 25, 2021 – ViaCyte, Inc., a clinical-stage regenerative medicine company focused on developing cell therapies that provide a functional cure for patients with insulin-requiring diabetes, announced today compelling preliminary clinical data from its stem cell-derived islet cell replacement therapy for patients with type 1 diabetes (T1D).  The results reported in a late-breaking ePoster[i] at the American Diabetes Association’s Virtual 81st Scientific Sessions showed PEC‑Direct (VC-02) demonstrates effective engraftment and production of C-peptide, a biomarker used to assess production of insulin by functional pancreatic beta cells.  These data represent the first exhibition of implanted pancreatic progenitor cells producing endogenous insulin in a patient as seen by clinically relevant increases in glucose-responsive C-peptide levels, increased time in range, and reduction of HbA1C.

PEC-Direct is an islet cell replacement therapy comprised of stem cell-derived pancreatic islet progenitor cells in a pouch that allows direct vascularization of the implanted cells, thus requiring a concurrent immunosuppressant regimen.  Preliminary data for one patient through 9 months study duration implanted with PEC-Direct showed stimulated C-peptide Cmax concentrations increased from 0.1 ng/mL (baseline) to 0.8 ng/mL (week 39), and area under the curve (AUC)0-4hr increased from 0.225 (baseline) to 2.55 ng x hr/mL (week 39) indicating the cells were producing and secreting insulin.  Histological evaluation of the sentinel device used to monitor the therapeutic implant further indicated successful engraftment.  Importantly, the patient also experienced an increased time in range from 54% to 88% at week 42 and a 0.8% reduction of HbA1C from 7.4% (baseline) to 6.6% (week 39), both results suggestive of improved control of blood glucose with the therapy.  There were no serious adverse events reported for the patient during this same time period.

“The preliminary C-peptide, time in range, and HbA1C results in the patient treated with PEC-Direct are unprecedented in the field of stem cell-derived cell replacement therapies for T1D,” said Manasi Sinha Jaiman, MD, MPH, Vice President, Clinical Development at ViaCyte.  “These results offer clinical proof-of-concept that our islet cell replacement therapy is a promising approach to effectively control blood glucose and, with optimization, has potential to fundamentally change the management of T1D.”

“Patients with T1D struggle with relentless highs and lows in glycemia.  Recent advances in insulin administration through injections have not eliminated the need for a physiologically regulated cell source of circulating insulin.  The ability of the PEC-Direct cell therapy to achieve clinically relevant C‑peptide levels accompanied by an increased time in range is a significant step in the development of a cure for patients with T1D,” said the Principal Investigator of this EU-supported trial, Professor Bart Keymeulen, MD, PhD, University Hospital Brussels.

The PEC-Direct Phase 2 study is currently enrolling additional patients to evaluate the safety and efficacy of PEC‑Direct implanted subcutaneously in patients with T1D who have hypoglycemia unawareness and/or extreme glycemic lability.  Study participants upon enrollment are required to be negative for C-peptide, which is not confounded by exogenous insulin.  The Company anticipates reporting additional patient data readouts from the PEC-Direct study in the first half of 2022.

“Patients with T1D are constantly working to improve their time in range, and ViaCyte’s cell replacement therapy has significant promise to allow patients to achieve better health outcomes without the requirement for continuous monitoring and daily insulin,” said Howard Foyt, MD, PhD, FACP, Chief Medical Officer at ViaCyte.  “To that end, we continue advancing our pipeline of novel clinical-stage treatments for T1D, as well as our innovative immune-evasive PEC‑QT program, which is expected to be in clinical studies later this year.  We are pleased to see the translation of PEC‑Direct from bench to clinic to meaningful therapeutic benefits observed in patients and the realization of progress toward our mission of a functional cure for T1D.”

The ePoster abstract is available on the journal Diabetes® website at the following link: https://diabetes.diabetesjournals.org/content/70/Supplement_1/196-LB.

About ViaCyte

ViaCyte is a privately held clinical-stage regenerative medicine company developing novel cell replacement therapies based on two major technological advances: cell replacement therapies derived from pluripotent stem cells and medical device systems for cell encapsulation and implantation.  ViaCyte has the opportunity to use these technologies to address critical human diseases and disorders that can potentially be treated by replacing lost or malfunctioning cells or proteins.  The Company’s first product candidates are being developed as potential long-term treatments for patients with type 1 diabetes to achieve glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications.  To accelerate and expand the Company’s efforts, ViaCyte has established collaborative partnerships with leading companies, including CRISPR Therapeutics and W.L. Gore & Associates.  ViaCyte is headquartered in San Diego, California. For more information, please visit www.viacyte.com and connect with ViaCyte on Twitter, Facebook, and LinkedIn.

About PEC-Direct (VC-02)

ViaCyte’s PEC-Direct (VC-02) product candidate is being developed for treatment of patients with type 1 diabetes who have hypoglycemia unawareness and/or extreme glycemic lability.  It is an investigational cell replacement therapy comprised of pancreatic islet progenitor cells in a pouch designed to allow blood vessels to enter the device, which permits direct vascularization of the implanted cells but will necessitate the use of immune suppression therapy because the implanted cells are not hidden from the immune system.  PEC-Direct is designed to enable production of both insulin and glucagon to modulate blood glucose, improve time in range, and ameliorate or prevent complications associated with type 1 diabetes.  Phase 2 clinical studies to evaluate the safety and efficacy of PEC-Direct are ongoing (clinical trial identifier: NCT03163511).

About the ADA’s Scientific Sessions

The American Diabetes Association’s (ADA) 81st Scientific Sessions, the world’s largest scientific meeting focused on diabetes research, prevention, and care, will be held virtually June 25-29, 2021.  Leading physicians, scientists, and health care professionals from around the world will unveil cutting-edge research, treatment recommendations and advances toward a cure for diabetes.  Attendees will receive exclusive access to all virtual content for 90-days after the event, with access ending September 29, 2021. For more information, visit https://professional.diabetes.org/scientific-sessions.

[i] Keymeulen B, Jacobs-Tulleneers-Thevissen D, Kroon E, Jaiman M, Daniels M, Wang R, Pipeleers D, D’Amour, K, Foyt H.  (2021)  Stem Cell-Derived Islet Replacement Therapy (VC-02) Demonstrates Production of C-Peptide in Patients with Type 1 Diabetes (T1D) and Hypoglycemia Unawareness.  American Diabetes Association’s Virtual 81st Scientific Sessions.  Poster 196-LB.  https://diabetes.diabetesjournals.org/content/70/Supplement_1/196-LB.