Novartis sells phase 3 rare disease drug to Pharming

Novartis headquarters
Novartis will work with Pharming to complete enrollment in the second part of the phase 2/3 trial of rare genetic disease treatment leniolisib. (Novartis)

Pharming has licensed late-phase rare genetic disease treatment leniolisib from Novartis for $20 million (€17.9 million) upfront. The immunomodulator could come to market in activated PI3K-delta syndrome (APDS) in the second half of 2021. 

Novartis was developing the selective PI3Kδ inhibitor, also known as CDZ173, in APDS in the belief it can increase the low white blood cell levels that characterize the rare genetic disease. However, the Swiss big pharma has decided to shift strategies in the middle of a phase 2/3 trial, offloading the rights to Pharming in return for a small upfront fee plus milestones and royalties.

Pharming’s interest in leniolisib relates to the overlap between the physicians that may prescribe the drug and its existing commercial asset, hereditary angioedema treatment Ruconest. Both drugs will be prescribed by immunologists, enabling Pharming to use existing infrastructure to push leniolisib.

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“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 said in a statement. “It is a perfect strategic fit for our existing medical and commercial infrastructure.” 

Novartis moved leniolisib into the open-label dose-escalation portion of a phase 2/3 trial in 2015 and presented long-term follow-up data on six patients late last year. The readout suggested leniolisib does not cause diarrhea or other side effects associated with mTOR or other PI3K inhibitors. Half of the patients stopped taking immunoglobulin following the normalization of B cell function.

Pharming hopes to build on these results to create a data set capable of persuading regulators to approved the drug, starting by working with Novartis to complete enrollment in the second part of the phase 2/3 trial.

GlaxoSmithKline also has a treatment for APDS in development. An inhaled formulation of that drug, another selective PI3Kδ inhibitor known as GSK2269557, is being tested in a 20-patient phase 2 trial.

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