Pharming Group ends angioedema gene therapy collaboration with Orchard

Pharming Group has decided to terminate its gene therapy collaboration with Orchard Therapeutics, leading to the cessation of their preclinical hereditary angioedema (HAE) program. The Dutch company had earlier paid Orchard Therapeutics, based in the UK, an upfront amount of $17.5 million in cash and equity in 2021 to develop OTL-105, a gene therapy aimed at increasing C1 esterase inhibitor levels to prevent HAE attacks.

Three years ago, when the partnership was announced, Pharming's CEO Sijmen de Vries expressed strong confidence in the collaboration. He highlighted that Pharming had been dedicated to the HAE community for over two decades. The efficacy of Orchard’s approved hematopoietic stem cell therapies led Pharming to believe that this technology had a high potential for success in treating HAE, a rare and life-threatening disorder characterized by recurring swelling attacks in various parts of the body, including the face, throat, extremities, and abdomen. At the time, preclinical studies indicated promising results, showing high levels of SERPING1 gene expression through lentiviral-mediated transduction in multiple cell lines and primary human CD34+ HSCs.

However, Pharming has now decided to end the collaboration. In its first-quarter earnings report, the company announced its decision to terminate the research and licensing agreement with Orchard Therapeutics and halt the OTL-105 program. This termination means Orchard, which was acquired by Japan's Kyowa Kirin for $387.4 million in October 2023, will no longer be eligible for up to $189.5 million in potential development, regulatory, and sales milestones linked to the collaboration.

Pharming's decision aligns with its current strategic focus, particularly the clinical development expansion of leniolisib into additional primary immunodeficiency indications. The company acquired leniolisib from Novartis for $20 million in 2019. The kinase inhibitor, marketed under the name Joenja, has received FDA approval for patients with phosphoinositide 3-kinase delta syndrome. In the first quarter of 2024, Joenja generated $9.6 million in revenue, according to the latest earnings report. Furthermore, a phase 2 trial of leniolisib is underway, targeting primary immunodeficiency with immune dysregulation associated with PI3Kẟ signaling.

Pharming's shift in focus highlights its prioritization of expanding its clinical pipeline, particularly with leniolisib, which has already shown commercial success and regulatory approval. This strategic move away from the gene therapy collaboration with Orchard Therapeutics suggests that Pharming is concentrating its resources and efforts on areas with more immediate and tangible outcomes.

In conclusion, Pharming Group has ended its collaboration with Orchard Therapeutics, leading to the discontinuation of their preclinical HAE program. This decision underscores Pharming's strategic shift towards expanding the clinical development of leniolisib, a kinase inhibitor that has shown promising results and commercial potential in treating primary immunodeficiency conditions. The termination of the agreement also means that Orchard Therapeutics, now under Kyowa Kirin, will miss out on significant potential milestone payments.