Vertex optimistic about Casgevy launch as cell collections surpass bluebird’s Lyfgenia

Vertex Pharmaceuticals has made significant strides since receiving initial approvals for Casgevy (exagamglogene autotemcel) late last year. By mid-April, five patients had their stem cells collected in preparation for the gene therapy's production. Stuart Arbuckle, Vertex’s Chief Operating Officer, described this progress as "excellent," noting that many patients have already started treatment in regions where Casgevy is approved, such as the US, Europe, and the Middle East.

Casgevy employs CRISPR/Cas9 gene-editing technology and is priced at $2.2 million in the US. It is approved for patients with sickle-cell disease (SCD) and transfusion-dependent beta thalassemia. Arbuckle mentioned during a first-quarter earnings call that cell collections for Casgevy have occurred in all regions where the therapy is available.

In a similar vein, bluebird bio recently completed its first commercial cell collection for Lyfgenia (lovotibeglogene autotemcel). This gene therapy, priced at $3.1 million in the US, treats SCD patients who have experienced vaso-occlusive events. Both Casgevy and Lyfgenia received FDA approval last December.

Mizuho analysts observed that Casgevy appears to be progressing faster than Lyfgenia, especially in the US in terms of cell collections at treatment centers. Vertex and its co-development partner, CRISPR Therapeutics, have 18 active collection sites in the US, whereas bluebird operates more than 60 centers. Globally, Vertex has more than 25 authorized treatment centers (ATCs) for Casgevy and aims to bring 75 priority centers online. According to Morgan Stanley analysts, 80 patients are expected to receive Casgevy in 2024, with 54 of them in the US.

Arbuckle also stated that Vertex is making significant headway with payers. However, the company will only recognize revenue near the end of the patient's treatment journey, specifically at the point of infusion. Casgevy's revenue is factored into Vertex's annual sales forecast of $10.55 billion to $10.75 billion, with the gene therapy expected to contribute in the latter half of the year. Despite this, the majority of sales will still come from their cystic fibrosis (CF) treatments.

In the first quarter, Vertex's product revenue rose by 13% to $2.7 billion, surpassing estimates of $2.6 billion. This growth was driven by $2.5 billion in sales from their CF treatments, Trikafta and Kaftrio. Net income also saw an increase, reaching $1.1 billion, up from about $700 million the previous year.

David Song from Tema ETFs highlighted that over the next 12 to 18 months, investors are keen to see if Vertex can maintain its CF franchise and expand through internal developments and business acquisitions. Recently, Vertex agreed to acquire Alpine Immune Sciences for $4.9 billion, securing the experimental drug povetacicept for IgA nephropathy.

Additionally, Vertex is working to introduce a non-opioid pain medication to the market and has begun an FDA rolling submission for suzetrigine. Other pipeline projects include inaxaplin for autosomal dominant polycystic kidney disease and VX-880 for type 1 diabetes.