Pharming Adds CDZ173 To Its Rare Disease Pipeline, Foresees Launch by 2022
Executive Summary
The Dutch rare disease group says licensing CDZ173 from Novartis is Pharming’s first step towards building off the commercial success of its sole product, Ruconest, in HAE.
The Dutch rare disease company Pharming Group NV has embarked on expansion by acquiring an exclusive global license to CDZ173 (leniolisib), a late-stage small-molecule drug for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), a primary immune deficiency, in return for an upfront payment of $20m to Novartis AG as well as potential milestone and royalty payments.
CDZ173 is currently in a pivotal Phase II/III clinical trial as a treatment for APDS. 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 said it would work alongside Novartis to complete enrollment of the ongoing trial.
"The license of CDZ173 is our first step towards building off the commercial success of Ruconest in HAE to grow and diversify our portfolio." - Pharming CEO Sijmen de Vries
Announcing the exclusive licensing deal on 13 August, Pharming predicted a potential launch for CDZ173 by the second half of 2021 or first half of 2022, making it the Leiden, the Netherlands-based group's second marketed product behind Ruconest.
Pharming develops and produces therapeutic proteins through a bioreactor recombinant technology platform. Ruconest (conestat alfa) is a recombinant human C1 esterase inhibitor (rhC1INH) produced in rabbits which won European Medicines Agency approval in 2010 and US Food and Drug Administration approval in July 2014 as an intravenous treatment for hereditary angioedema (HAE).
Debilitating APDS
APDS is an ultra-rare, debilitating disease that impairs the immune system. There are no approved treatments available for the disease, which has incidence rates across the world of around one to two per million.
During a 13 August analyst call explaining the Novartis transaction, Pharming CEO Sijmen de Vries noted that there was a commercially available genetic test that could identify APDS patients who would benefit from CDZ173.
“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," de Vries told analysts.
Upon approval, Pharming would 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
“We are very excited about the profile of this drug, and about its strategic fit with our medical and commercial infrastructure… We are fairly close to where we can go to the regulators and hopefully get CDZ173 approved,” the CEO said.
On the issue of manufacturing, de Vries noted that because CDZ173 is a small molecule it is relatively straight forward, and that Novartis is in the process technology transfer to a CMO and that Pharming was likely to maintain the relationship, though that would be more fully assessed once the product was in their hands.
“We are pleased and honored that Novartis has selected us rather than other companies who were pitching for this compound and we look forward to working with them to finish development of this drug and bring it to the market,” the CEO added.
Under the deal’s terms Pharming, will pay Novartis a $20m upfront fee, as well as future but undisclosed regulatory and commercial milestones, along with tiered, double-digit royalties on net sales.