Rare Daily Staff

Pharming Group said it has entered into a development collaboration and license agreement with Novartis to develop and commercialize CDZ173, an experimental drug to treat activated phosphoinositide 3-kinase delta syndrome (APDS), an ultra-rare immune disorder.

People with APDS often have low numbers of white blood cells, particularly B cells and T cells. Normally, these cells recognize and attack foreign invaders, such as viruses and bacteria, to prevent infection. 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 and can cause breathing problems. People with activated APDS may also have chronic active viral infections, commonly Epstein-Barr virus or cytomegalovirus infections.  Sufferers also frequently develop lymphomas and other cancers.

CDZ173 is a small molecule drug with immunomodulating and potentially anti-neoplastic activities.  CDZ173 inhibits a central regulator of the adaptive immune system, as well as the innate immune system.

CDZ173 is being studied in a registration-enabling phase 2/3 trial that is currently enrolling patients in clinical sites in the United States and Europe. To date, CDZ173 has proven to be safe and well-tolerated in healthy subjects as well as the APDS patients during the first in human clinical trial and the ongoing open-label extension trial.

Novartis has completed all of 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 United States and Europe and look for ways to make the drug available in other markets worldwide.

Under the terms of the agreement, Pharming will provide an upfront payment of $20 million.  Novartis is eligible to receive regulatory and commercial milestones and will also earn tiered, double-digit royalties on net sales.

“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,” said Sijmen de Vries, CEO of Pharming. “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.”

Photo: Sijmen de Vries, CEO of Pharming

X