•CDZ173 is in a registration-enabling study for the treatment of APDS, an ultra-rare, debilitating disease with no approved treatment
•APDS is treated by immunologists; the main physicians treating HAE and therefore already addressed by Pharming
•Upfront payment will be $20 million (€17.9 million)
•If approved, the drug is expected to reach the market in 2H 2021 or 1H 2022
•Pharming will hold a conference call at 15.00 CET/ 09.00 EST today: dial-in details can be found at the end of this announcement
Leiden, the Netherlands – 13 August 2019
Pharming Group N.V. (Euronext Amsterdam: PHARM) today announced it has entered into a development collaboration and license agreement with Novartis to develop and commercialize CDZ173, a small molecule phosphoinositide 3-kinase delta (PI3K?) inhibitor being developed by Novartis to treat patients with Activated Phosphoinositide 3-kinase Delta Syndrome (“APDS”).
APDS is a primary immune deficiency caused by a mutation in the PIK3CD gene that increases activity of PI3K?, a promoter of activity in the immune system. As a result of this 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. Patients frequently suffer a functional inability to fight off infections, as well as developing airway and other lesions and certain cancers. It is an ultra-rare disease with incidence rates across the world of approximately 1-2 per million. Importantly, there is a commercially available genetic test that can identify the patients who will benefit from CDZ173 making this program personalized medicine for these APDS patients and their family members who also have the mutation.
Novartis has completed all the preclinical and clinical work to date 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 US and Europe and look for ways to make the drug available in other markets worldwide.
Sijmen de Vries, Chief Executive Officer of Pharming, commented:
“This transaction represents a great milestone for Pharming. The license of CDZ173 is our first step towards building off the commercial success of Ruconest in HAE to grow and diversify our portfolio. 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 personalised treatment option for patients with no prospect of treatment. We are pleased to be joining Novartis in its commitment to these patients and we look forward to working alongside them to finish the development work on this drug.”
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