NASDAQ: VRTX biotech behemoth Vertex Pharmaceuticals is unstoppable. The company developed a promising new product and provided positive phase 3 findings for prospective candidates in the past six months. Drugmaker won't quit. Vertex is developing another drug that might be enormous. Discover what it is and what it means for investors.
Straight from the playbook First, let's examine Vertex Pharmaceuticals' decade-long success. A rare lung illness termed cystic fibrosis was the biotech's main focus. No drugs addressed CF's causes before Vertex's achievements. The drugmaker is the only biotech company to create many medications. The company wants to launch treatments in unmet need.
Vertex targets APOL1-mediated kidney disease, a chronic kidney disease caused by gene abnormalities. Proteinuria, foot edema, and elevated blood pressure usually appear in advanced stages when the kidneys are near failure.
Vertex Pharmaceuticals began the phase 3 of its phase 2/3 clinical trial for inaxaplin, an APOL-1-mediated kidney disease therapy. This candidate had great phase 2a results, including a statistically significant proteinuria reduction. According to preliminary statistics, Vertex will include 10-year-olds in the trial.
A great forever stock Inaxaplin may assist 100,000 U.S. and European patients, according to Vertex Pharmaceuticals. APOL-1-mediated kidney disease has no recognized treatment for its etiology. About 92,000 CF patients live in the biotech's target regions. If Vertex passes late-stage clinical studies with inaxaplin, it might launch a new dominant franchise as successful as its CF portfolio.
Vertex's hopes don't depend on this candidate alone. Inaxaplin may fail late-stage trials. If it doesn't, other drugmakers may create rival medicines. Vertex Pharmaceuticals, which was successful in CF, would struggle with field competition. Vertex is a great biotech stock despite these dangers to inaxaplin's bid.
Due to its main product, Trikafta, the company continues to expand revenue and earnings. Vertex Pharmaceuticals' sales rose 11% to $9.87 billion and earnings per share rose 8.3% to $13.89. Vertex began receiving clearance for Casgevy, a CRISPR Therapeutics gene-editing medicine for two rare diseases, late last year.
At $2.2 million per treatment course in the U.S. and 35,000 patients in the nations where it will introduce Casgevy, Vertex Pharmaceuticals might make a fortune. The company also revealed positive phase 3 findings for acute pain and next-generation CF therapies. Both may be approved by regulators within a year, strengthening Vertex Pharmaceuticals' roster.
Vertex Pharmaceuticals' pipeline goes beyond inaxaplin, therefore its approved drugs should change in five years. Early-stage programs may pay off later. Vertex Pharmaceuticals is an innovative biotech that should continue to generate strong clinical and regulatory progress, financial success, and market-beating returns.
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