Kiniksa Presents Preclinical Data on the GM-CSF Signaling Pathway in Temporal Arteries of GCA Patients

- June 12th, 2019

Kiniksa Pharmaceuticals (NASDAQ:KNSA) has announced preclinical data on its granulocyte macrophage colony stimulating factor (GM-CSF) showing pathways in temporal arteries in patients with giant cell arteritis (GCA). As quoted in the press release: “The preclinical data presented at EULAR underscore the mechanistic rationale of targeting GM-CSFRα in patients with GCA,” said Sanj K. Patel, Chief … Continued

Kiniksa Pharmaceuticals (NASDAQ:KNSA) has announced preclinical data on its granulocyte macrophage colony stimulating factor (GM-CSF) showing pathways in temporal arteries in patients with giant cell arteritis (GCA).

As quoted in the press release:

“The preclinical data presented at EULAR underscore the mechanistic rationale of targeting GM-CSFRα in patients with GCA,” said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. “GM-CSF, GM-CSFRα, and their associated signaling molecules were shown to be significantly upregulated in temporal arteries of GCA patients. Furthermore, mavrilimumab was shown to suppress the increased expression of genes associated with this inflammatory pathway. We are continuing to advance our global Phase 2 clinical trial of mavrilimumab in GCA and look forward to presenting top-line data in the second half of 2020.”

Dr. Maria C. Cidis a lead author of the poster presentation entitled GM-CSF Pathway Signature Identified in Temporal Artery Biopsies of Patients with GCA.

  • Data from this preclinical study comparing two independent sources of temporal artery biopsies showed the GM-CSF signaling pathway molecular signature was confirmed to be upregulated in GCA biopsies versus control at both the mRNA and protein level. GM-CSF and TH1 pathway signatures were demonstrated in GCA patient temporal arteries by independent analytical techniques. The data also demonstrated active GM-CSF signaling in diseased tissue is evidenced by increased expression of PU.1, a transcription factor downstream of GM-CSF signaling, in the vessel wall. Additionally, treatment of ex vivo cultures of GCA arteries with mavrilimumab suppressed expression of these genes, indicating the biological effect of mavrilimumab on genes relevant to GCA pathophysiology.

Click here to read the full press release.

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