FDA Accepts License Application for Vertex Pharma’s and CRISPR Therapeutics’ Key Investigational Treatment

  Image: Vertex’s pipeline is impressive. We like its areas of focus and the progress it continues to make with key therapies in CRISPR and pain management. By Brian Nelson, CFA On June 8, it was announced that the Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) from two of our favorite speculative biotech entities Vertex Pharma (VRTX) and CRISPR Therapeutics (CRSP) have been accepted by the U.S. FDA. The submission received Priority Review for SCD, and exa-cel has the potential to be the first CRISPR gene-editing therapy to be approved. We continue to be huge fans of this technology and Vertex’s risk-adjusted exposure to the potential … Read more

Best Idea Vertex Pharma Outperforming in 2022

Image Shown: Shares of Vertex Pharmaceuticals Inc, an idea included in our Best Ideas Newsletter portfolio, have performed incredibly well year-to-date through October 2022. By Callum Turcan Through the end of normal trading hours on October 7, shares of Vertex Pharmaceuticals Inc (VRTX) are up ~34% year-to-date on a price-only basis. We include shares of Vertex Pharma as an idea in the simulated Best Ideas Newsletter portfolio and remain huge fans of the name. Vertex Pharma’s commercialized drug portfolio consists of various treatments for cystic fibrosis (‘CF’) and the cash flows generated from sales of these therapeutics are used to invest in the biotech company’s robust drug development pipeline. Earnings and Guidance Update On August 4, Vertex Pharma reported second … Read more

Our Favorite Biotech Vertex Pharma Powers Ahead, Leaps 6%+

Image: Vertex Pharma continues to soar toward our fair value estimate. By Brian Nelson, CFA  On Wednesday, January 26, Vertex Pharma (VRTX) reported solid fourth-quarter results that showed continued advancement across its clinical pipeline. For background reading on our thesis on the net cash rich, free cash flow generating biotech tied to established cystic fibrosis (CF) therapies and CRISPR technology, please read: “Best Idea Vertex Pharma Boosts Guidance (Again), Buying Back Stock While Awaiting Key Clinical Trials (December 2, 2021)” and “Best Idea Vertex Pharma Marching Forward with Innovative CRISPR Technology” (August 31, 2021).” Following fourth-quarter results, we’re reiterating our $250 per share fair value estimate at this time.   Image: Vertex finished an exciting 2021, and we look forward to … Read more

Best Idea Vertex Pharma Marching Forward with Innovative CRISPR Technology

Image Shown: An overview of Vertex Pharmaceuticals Inc’s commercialized drug portfolio and pipeline. Image Source: Vertex Pharmaceuticals Inc – Second Quarter of 2021 IR Earnings Presentation By Callum Turcan The world of medicine and therapeutics continues to evolve, with gene editing CRISPR technology offering the medical community an immense source of potential life-saving therapies over the years and decades to come. Our favorite way to gain exposure to this space is through Vertex Pharmaceuticals Inc (VRTX), an idea included in the Best Ideas Newsletter portfolio. What Is CRISPR? In short, CRISPR gene editing technology can identify a specified portion of DNA in a cell and when done properly (this is no easy task), CRISPR technology can alter that DNA, potentially … Read more

Best Idea Vertex Pharma Boosts Guidance (Again), Buying Back Stock While Awaiting Key Clinical Trials

Image Shown: Vertex Pharmaceuticals Inc is one of our favorite biotech ideas. Image Source: Vertex Pharmaceuticals Inc – Third Quarter of 2021 IR Earnings Presentation By Callum Turcan One of our favorite biotech plays out there is Vertex Pharmaceuticals Inc (VRTX), which is included as an idea in the Best Ideas Newsletter portfolio. The company’s commercial portfolio, meaning therapeutics that have already been approved by key health regulators, is centered on drugs that treat cystic fibrosis (‘CF’), including patients as young as four months old as well as older patients. Its branded commercialized CF treatments include TRIKAFTA/KAFTRIO, SYMDEKO/SYMKEVI, ORKAMBI, and KALYDECO. Vertex Pharma is currently generating substantial revenue and cash flows from these offerings. Guidance Update Sales of its CF … Read more