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Vertex (VRTX) Up on Kidney Disease Drug's Success in Phase II

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Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced positive data from a phase II study evaluating VX-147, its oral small molecule medicine for treating APOL1-mediated focal segmental glomerulosclerosis (FSGS), a severe kidney disorder.

Data from the study showed that treatment with VX-147 on top of standard of care led to a statistically significant, substantial and clinically meaningful mean reduction of 47.6% in proteinuria at 13 weeks compared to baseline. Proteinuria indicates the presence of abnormal quantities of protein in the urine and was measured by the urine protein to creatinine ratio (UPCR) in the study. High levels of proteinuria may indicate damage to the kidneys, which can quickly progress to kidney failure.  Vertex said the medicine was well tolerated in the study with no serious adverse events related to it.

The data provide the first clinical proof of concept that an oral small molecule APOL1 inhibitor can cause a reduction in proteinuria in patients with APOL1-mediated kidney disease. VX-147 targets the underlying genetic driver of kidney disease and has the potential to be an effective treatment for patients who are at an unusually high risk of progression to kidneyfailure

Vertex plans to push VX-147 into pivotal studies for APOL1-mediated kidney disease including FSGS in the first quarter of 2022.

Vertex’s shares were up almost 10% on Wednesday in response to the positive data. Vertex’s stock has declined 13.9% this year so far compared with a decrease of 16.1% for the industry.


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While Vertex’s main focus is on the development and strengthening of its CF franchise, the company also has a rapidly advancing early-stage portfolio in five additional diseases beyond CF. Other than APOL1-mediated kidney diseases discussed above, these areas are pain, sickle cell disease, beta-thalassemia and cell therapy for type I diabetes.

Vertex is co-developing a gene-editing treatment, CTX001, in partnership with CRISPR Therapeutics (CRSP - Free Report) in two devastating diseases — sickle cell disease and thalassemia. Phase I/II studies of CTX001 in adult transfusion-dependent b-thalassemia in Europe and sickle cell disease in the United States are ongoing.

Preliminary safety and efficacy data from the studies were positive. Vertex and CRISPR Therapeuticsplan to file regulatory applications for CTX001 for both indications by 2022 end.

VX-548, a selective NaV1.8 inhibitor, is being evaluated in two phase II acute pain studies, one following a bunionectomy surgery and the other following an abdominoplasty surgery. Data from the acute pain studies are expected in the first quarter of 2022.

A phase I/II study was initiated on VX-880 for the treatment of type I diabetes (T1D) in March. 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. Clinical development on the second program is expected to begin in 2022.

Vertex currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank stocks here.

Some better-ranked stocks in the biotech sector include Sarepta Therapeutics, Inc. (SRPT - Free Report) and IVERIC bio (ISEE - Free Report) , both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Estimates for Sarepta Therapeutics have narrowed from a loss of $6.95 per share to $4.99 per share for 2021 and from $4.83 per share to $3.61 per share for 2022 over the past 30 days.

Earnings of Sarepta Therapeutics surpassed estimates in two of the trailing four quarters and missed the same on the other two occasions. SRPT delivered an earnings surprise of 11.06%, on average.

Estimates for IVERIC bio for 2021 have narrowed from a loss of $1.18 to $1.12 per share in the past 30 days. The same for 2022 has narrowed from $1.17 to $1.10 in the past 30 days. Shares of IVERIC bio have gained 104.2% in the year so far.

IVERIC bio missed estimates in three of the last four quarters and surpassed expectations once, delivering a negative surprise of 5.58%, on average.