Horizon Plans to Advance Oral dcSSc Therapy in Trials, Having Acquired Curzion

Horizon Plans to Advance Oral dcSSc Therapy in Trials, Having Acquired Curzion
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Horizon Therapeutics announced it plans to pursue the development of HZN-825, a potential therapy for diffuse cutaneous systemic sclerosis (dcSSc) that it gained in acquiring Curzion Pharmaceuticals.

Horizon plans to conduct a study this year to investigate the pharmacological profile of HZN-825, previously known as CZN001.

Company leaders also expect to soon meet with the U.S. Food and Drug Administration (FDA) to discuss a pivotal Phase 2b trial, which they hope to launch by June 2021. Pivotal trials are intended to provide results that support applications for regulatory approval.

“We look forward to meeting with regulatory authorities to define the development pathway for HZN-825 to potentially offer help to those who are impacted by this devastating disease,” Tim Walbert, the chairman, president and CEO of Horizon, said in a press release.

HZN-825 is an oral therapy intended to prevent the onset of skin fibrosis (scarring) by blocking a protein called lysophosphatidic acid 1 receptor (LPAR1).

Research in mice indicated that the onset of skin fibrosis is associated with binding of the lipid molecule lysophosphatidic acid (LPA, whose blood levels are elevated in scleroderma patients) to its receptor, LPAR1. This suggests that preventing LPA-LPAR1 interaction could potentially treat people with scleroderma.

HZN-825 was originally developed by Sanofi (then called SAR100842), and was later acquired by Curzion. Horizon paid $45 million upfront to acquire Curzion, obtaining rights to the investigational scleroderma treatment. Additional payments to Curzion and Sanofi are contingent on milestone achievements in the development process.

In five Phase 1 clinical studies conducted by Sanofi, the treatment was safe and well-tolerated. Sanofi has also conducted a short-term Phase 2a trial (NCT01651143), which suggested a potential benefit to dcSSc patients.

Results showed that eight weeks of treatment with HZN-825 slightly reduced skin thickness, with a greater effect in normalizing changed expression of LPA-associated genes compared to a placebo. This suggested that HZN-825 blocked LPAR1 function as intended.

Longer trials are necessary to confirm these early findings, Horizon said.

“This acquisition is one of several transactions we intend to make that directly aligns with our strategy to expand our development-stage pipeline, with a focus on our core therapeutic areas of rheumatology, nephrology, endocrinology and ophthalmology,” Walbert said.

David earned a PhD in Biological Sciences from Columbia University in New York, NY, where he studied how Drosophila ovarian adult stem cells respond to cell signaling pathway manipulations. This work helped to redefine the organizational principles underlying adult stem cell growth models. He is currently a Science Writer, as part of the BioNews Services writing team.
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David earned a PhD in Biological Sciences from Columbia University in New York, NY, where he studied how Drosophila ovarian adult stem cells respond to cell signaling pathway manipulations. This work helped to redefine the organizational principles underlying adult stem cell growth models. He is currently a Science Writer, as part of the BioNews Services writing team.
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