Shares of Vertex Pharmaceuticals Incorporated (VRTX) jumped 9.7% on Wednesday to close at $205 after the biopharmaceutical company disclosed positive results from the open-label Phase 2 trial of VX-147.
The trial was designed to evaluate the efficacy, safety, and pharmacokinetics of VX-147 in patients with APOL1-mediated Focal Segmental Glomerulosclerosis (FSGS). Notably, VX-147 is the first-of-its-kind investigational therapy that targets the underlying cause of APOL1-mediated kidney disease.
The data demonstrated that VX-147 has a well-tolerated profile and led to a substantial 47.6% mean reduction in proteinuria at 13 weeks. These results also provide clinical evidence of treating kidney disease. (See Vertex stock charts on TipRanks)
Based on the outcome, Vertex has planned to advance VX-147 into pivotal development for APOL1-mediated kidney disease, including FSGS, in the first quarter of 2022.
The CMO at Vertex, Carmen Bozic, said, “We are working with urgency to advance this molecule into pivotal development with the goal of bringing this first-in-class therapy to the more than 100,000 patients in the U.S. and Europe living with APOL1-mediated kidney disease.”
The Chair of the Vertex APOL1 Program Steering Committee, Glenn Chertow, commented, “This approach has tremendous potential, as it targets the underlying genetic driver of kidney disease in these patients. These data demonstrate the potential of VX-147 as a targeted treatment for a patient population at unusually high risk of progression to kidney failure.”
Wall Street’s Take
Following the recent clinical update, Needham analyst Joseph Stringer reiterated a Hold rating on the stock.
Stringer said, “Overall, our takeaway is generally positive and we think these early results are encouraging. We await additional updates around the design of a Ph3 program. We continue to monitor pipeline progression beyond Cystic Fibrosis.”
The rest of the Street is cautiously optimistic about Vertex and has a Moderate Buy consensus rating based on 8 Buys, 5 Holds, and 2 Sells. The average Vertex price target of $246.08 implies 20.04% upside potential to current levels. Shares have fallen 9.6% over the past year.
According to the new TipRanks’ Risk Factors tool, Vertex stock is at risk mainly from three factors: Finance & Corporate, Legal & Authority, and Tech and Innovation, which contribute 27%, 23%, and 20%, respectively, to the total 44 risks identified for the stock.