Vertex Pharmaceuticals Incorporated VRTX announced the initiation a clinical study on its investigational cell-based therapy, VX-880 for the treatment of type I diabetes (T1D). The phase I/II study will be conducted on approximately 17 T1D patients with severe hypoglycemia and impaired hypoglycemic awareness and will evaluate the safety and efficacy of different doses of VX-880.
Meanwhile, the FDA also assigned a Fast-Track Designation to VX-880 for TD1. The designation facilitates the development and expedites the review of drugs that treat serious conditions
Vertex had acquired VX-880 as part of its Semma Therapeutics’ acquisition in 2019 when it was in pre-clinical studies.
VX-880 is Vertex’s first of the two investigational programs for the transplant of functional islets into patients. VX-880, is for the transplantation of islet cells alone, using immunosuppression to protect the implanted cells. The second program will involve the implantation of the islet cells inside an immunoprotective device.
The latest phase I/II study on VX-880 involves an infusion of fully differentiated, functional islet cells, and chronic administration of concomitant immunosuppressive therapy, to protect the islet cells from immune rejection.
Vertex believes that VX-880,both alone and in combination with an implantable device has the potential to cure type I diabetes. It believes the candidate has the potential to restore body’s glucose levelsby restoring pancreatic islet cell function, including insulin production.
Vertex’s stock has declined 9.3% this year so far compared with a decrease of 0.6% for the industry.
While Vertex’s main focus is on the development and strengthening of its cystic fibrosis (CF) franchise, the company also has a rapidly advancing early-stage portfolio in seven other specialty disease areas like pain, alpha-1 antitrypsin deficiency (AAT), sickle cell disease (SCD), beta-thalassemia, APOL1-mediated kidney diseases and cell therapy for type I diabetes.
Vertex is co-developing a gene editing treatment, CTX001 in partnership with CRISPR Therapeutics CRSP for SCD and thalassemia in phase I/II studies. Meanwhile, phase II studies are ongoing for Vertex’s small molecule corrector for the AAT disease, VX-864, its oral small molecule medicine in APOL1-mediated focal segmental glomerulosclerosis, and its NaV1.8 inhibitor for pain, VX-150.
Zacks Rank and Stocks to Consider
Vertex currently carries a Zacks Rank #3 (Hold).You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Moderna’s earnings per share estimates have moved up from $12.72 to $21.52 per share for 2021 and from $11.45 to $15.22 for 2022 in the past 30 days. The stock has risen 24.2% so far this year.
Acorda’s stock is up 30.2% this year so far. Loss estimates for 2022 have narrowed from $5.08 per share to $4.59 per share in the past 30 days.
Breakout Biotech Stocks with Triple-Digit Profit Potential
The biotech sector is projected to surge beyond $775 billion by 2024 as scientists develop treatments for thousands of diseases. They’re also finding ways to edit the human genome to literally erase our vulnerability to these diseases.
Zacks has just released Century of Biology: 7 Biotech Stocks to Buy Right Now to help investors profit from 7 stocks poised for outperformance. Our recent biotech recommendations have produced gains of +50%, +83% and +164% in as little as 2 months. The stocks in this report could perform even better.
Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
Acorda Therapeutics, Inc. (ACOR): Free Stock Analysis Report
Moderna, Inc. (MRNA): Free Stock Analysis Report
Vertex Pharmaceuticals Incorporated (VRTX): Get Free Report
CRISPR Therapeutics AG (CRSP): Get Free Report
To read this article on Zacks.com click here.
The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.