Vertex upbeat on Casgevy launch as cell collections outdo bluebird’s Lyfgenia
并购基因疗法上市批准细胞疗法
Vertex Pharmaceuticals said that since gaining the first approvals late last year for Casgevy (exagamglogene autotemcel), five patients have had stem cells collected as of mid-April in preparation for manufacturing of the gene therapy. Stuart Arbuckle, chief operating officer, called the progress “excellent,” noting that “many patients have [also] begun the treatment journey” in the markets where Casgevy is approved, which include the US, Europe and the Middle East.
The therapy, which utilises CRISPR/Cas9 gene-editing technology and carries a list price of $2.2 million in the US, is authorised for use in patients with sickle-cell disease (SCD) and transfusion-dependent beta thalassaemia. Arbuckle, speaking on the company’s first-quarter earnings call, explained that the cell collections have occurred across all regions where Casgevy is available.
Meanwhile, bluebird bio recently said it completed the first commercial cell collection for its cell-based gene therapy Lyfgenia (lovotibeglogene autotemcel). The product is approved in the US, where it has a list price of $3.1 million and is used to treat SCD patients with a history of vaso-occlusive events. Both Casgevy and Lyfgenia gained clearance from the FDA last December.
Casgevy ‘ramping more quickly’
Mizhuo analysts noted that Casgevy “seems to be ramping more quickly” than Lyfgenia, particularly in the US, in terms of cell collections-to-treatment centres. According to the analysts, Vertex and co-development partner CRISPR Therapeutics have 18 active collection sites in the US, while bluebird has over 60 centres.
Globally, Vertex says it has activated more than 25 authorised treatment centres (ATCs) for Casgevy, with a target of bringing 75 priority centres online. Analysts at Morgan Stanley forecast that a total of 80 patients will receive Casgevy in 2024, including 54 in the US.
Revenue in second half
Arbuckle indicated that the drugmaker continues to “make great progress with payers,” although he reiterated that it will only “recognise revenue…near the end of the patient journey at infusion.” Vertex is including Casgevy revenue in its overall annual sales forecast of $10.55 billion to $10.75 billion, with the gene therapy contributing in the second half of the year. However, the company’s cystic fibrosis (CF) products are still set to generate the majority of sales.
Pipeline in focus
David Song of Tema ETFs remarked "over the next 12 to 18 months, investors are evaluating whether Vertex can sustain its CF franchise for several years and expand upon it through internal pipeline development, as well as business development." The company recently agreed to buy Alpine Immune Sciences for $4.9 billion, gaining the experimental drug povetacicept for IgA nephropathy.
Vertex is also hoping to bring a non-opioid pain drug to market, having initiated an FDA rolling submission for suzetrigine, while its other pipeline bets include inaxaplin in autosomal dominant polycystic kidney disease and VX-880 in type 1 diabetes. For related analysis, see Vital Signs: Dissecting dealmaking at Vertex and its Alpine takeout.
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