Pharming Bolsters Immunology Pipeline with Novartis APDS Licensing Deal
Netherlands-based Pharming Group N.V. struck a licensing deal with Novartis to develop and commercialize CDZ173, a small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor for the treatment of patients with Activated Phosphoinositide 3-kinase Delta Syndrome (APDS).
Pharming paid Novartis an upfront fee of $20 million for the asset, a small molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K with immune-modulating and potentially anti-neoplastic activities. Novartis will be eligible for undisclosed regulatory and commercial milestone payments, as well as potential double-tiered royalties on sales.
Pharming Chief Executive Officer Sijmen de Vries called the deal with Novartis a “great milestone” for Pharming. He said the licensing of CDZ173 is the company’s first step to build off the commercial success of Ruconest, a treatment for hereditary angioedema (HAE).
“We are very excited by the profile of this drug and the match between its mechanism of action and the disease pathology, and it is a perfect strategic fit for our existing medical and commercial infrastructure. This is a great example of solid science and deep disease understanding coming together to create a real and personalized treatment option for patients with no prospect of treatment,” de Vries said in a brief statement.
Last year, Pharming suffered a temporary setback with Ruconest. In September 2018, the U.S. Food and Drug Administration issued a Complete Response Letter to the company as it sought to expand the drug’s indication to include prophylaxis in patients with HAE.
Novartis has completed all preclinical and clinical work to date on CDZ173 and will continue to run the ongoing registration-enabling trial and the ongoing open-label extension study. Pharming will work alongside Novartis to complete enrollment of the ongoing trial. Upon approval, Pharming will commercialize CDZ173 through its existing commercial infrastructure in the United States and Europe and look for ways to make the drug available in other markets worldwide. De Vries added that his company is pleased to work alongside Novartis in the development of CDZ173 for the treatment of APDS.
Activated Phosphoinositide 3-kinase Delta Syndrome is a rare immune deficiency caused by a mutation in the PIK3CD gene that increases activity of PI3Kẟ, a promoter of activity in the immune system. Due to the over-activity, the cells involved in immune response can fail to be differentiated properly, which means that sufferers are unable to react well to infections, and can suffer early cell death, Pharming said. Patients with APDS frequently suffer a functional inability to fight off infections, as well as developing airway and other lesions and certain cancers. Pharming noted that there is a commercially-available genetic test that can identify patients who will benefit from treatment with CDZ173. There are approximately one to two million people across the globe affected by APDS. There is currently no approved treatment for APDS.