Pharming buys rare disease drug from Novartis for $20m

Pharming has bought a late-stage pipeline drug aimed at a rare disease of the immune system from Novartis for $20 million up front, plus other payments further down the line.

CDZ173, or leniolisib, is a small molecule drug in development for activated phophoinositide 3-kinase delta syndrome – also known as APDS.

Pharming will pay $20 million up front for leniolisib, which is being studied in a phase 2/3 trial that could lead to approval, and is enrolling patients in the US and Europe. If approved the drug is expected to reach the market in the second half of 2021, or the first half 2022, Pharming said.

Novartis is eligible to receive regulatory and commercial milestones and will also earn tiered, double digit royalties on net sales.

Leniolisib is a primary immune deficiency caused by a mutation in the PIK3CD gene that increases activity of PI3K-delta, a promoter of activity in the immune system.

This aims to treat the biological cause of the disease result where cells involved in immune response can fail to be differentiated properly.

As a result patients with the disease frequently suffer a functional inability to fight off infections, as well as developing airway and other lesions and certain cancers.

Beginning in childhood, people with APDS develop recurrent infections, particularly in the lungs, sinuses, and ears.

Over time, recurrent respiratory tract infections can lead to a condition called bronchiectasis, which damages the passages leading from the windpipe to the lungs (bronchi) and can cause breathing problems.

The disease also causes abnormal clumping of white blood cells, which can form solid masses in the moist lining of the airways or intestines.

While these clumps are benign, they can increase the risk of developing the cancer B-cell lymphoma.

The ultra-rare disease has incidence rates across the world of around 1-2 per million, but there is a commercially available test identifying patients who will benefit from the drug.

Novartis has completed preclinical and clinical work to date and will continue to run the ongoing trial and an open label extension study.

Pharming will work alongside Novartis to complete enrolment of the ongoing trial, and Pharming will market leniolisib using its reps in US and Europe, and look for ways to make the drug available in other markets.

The Dutch biotech already has a presence in rare diseases as its Ruconest (C1 esterase inhibitor [recombinant]) is already approved in hereditary angioedema.