JP Morgan was een maand of 4 terug best enthousiast over Orchard. Ondertussen hebben ze de ontwikkeling van een eigenlijk ok-product gestopt, min of meer bij gebrek aan centjes. Het is eigenlijk nog een start up met iets van de helft van de market cap van Pharming.
"The last JPM stock pick we’ll look at is Orchard Therapeutics, a biopharma research company focused on the development of gene therapies for the treatment of rare diseases. The company’s goal is to create curative treatments from the genetic modification of blood stem cells – treatments which can reverse the causative factors of the target disease with a single dosing.
The company’s pipeline features two drug candidates that have received approval in the EU. The first, OTL-200, is a treatment for Metachromatic leukodystrophy (MLD), a serious metabolic disease leading to losses of sensory, motor, and cognitive functioning. Strimvelis, the second approved drug, is a gammaretroviral vector-based gene therapy, and the first such ex vivo autologous gene therapy to receive approve by the European Medicines Agency. It is a treatment for adenosine deaminase deficiency (ADA-SCID), when the patient has no available related stem cell donor.
In addition to these two EU-approved drugs, Orchard has ten other drug candidates in various stages of the pipeline process, from pre-clinical research to early-phase trials.
Anupam Rama, another of JPM’s 5-star analysts, took a deep dive into Orchard and was impressed with what he saw. In his coverage of the stock, he notes several key points: “Maturing data across various indications in rare genetic diseases continues to de-risk the broader ex vivo autologous gene therapy platform from both an efficacy / safety perspective… Key opportunities in MLD (including OTL-200 and other drug candidates) have sales potential each in the ~$200-400M range… Importantly, the overall benefit/risk profile of Orchard’s approach is viewed favorably in the eyes of physicians. At current levels, we believe ORTX shares under-reflect the risk-adjusted potential of the pipeline...”
The high sales potential here leads Rama to rate the stock as Outperform (Buy) and to set a $15 price target, implying a robust 122% upside potential in the next 12 months."