Sector News

Novartis sells phase 3 rare disease drug to Pharming

August 14, 2019
Life sciences

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.

“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.

By: Nick Paul Taylor

Source: Fierce Biotech

comments closed

Related News

October 2, 2022

GSK names Julie Brown, a 25-year AstraZeneca veteran, its first woman CFO

Life sciences

Five years ago, GSK made headlines when it hired Emma Walmsley to become the first woman to run a major pharmaceutical company. Now the Big Pharma has brought in another woman to control the company’s finances. Julie Brown will be GSK’s next chief financial officer. Brown, currently the chief operating and financial officer at fashion and beauty brand Burberry Group, is set to replace Iain Mackay.

October 2, 2022

Moderna creates new launch preparation role, poaches Novartis exec as manufacturing lead

Life sciences

Moderna created a new role responsible for “building out the company’s organization to support its growing pipeline.” Starting first thing 2023, Juan Andres, Moderna’s manufacturing head, will step into this new role under the title president of strategic partnerships and enterprise expansion, the company said Thursday.

October 2, 2022

Torrent Pharma to acquire Curatio for $245.16m

Life sciences

The latest takeover is anticipated to boost the presence of Torrent in the dermatology segment. Indian company Torrent Pharmaceuticals has signed a definitive agreement for the complete acquisition of Curatio Healthcare for $245.16m (Rs20bn).