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Numerous pathophysiological changes that contribute to tissue degeneration at the lesions make ulcerative NL difficult to treat. However, PCS499 and its metabolites simultaneously affect various biological pathways that can cause tissue degeneration, making PCS499 a promising novel treatment solution for NL.
Before we acquired the rights to PCS499, Concert Pharmaceuticals completed many of the New Drug Application studies, thus enabling the expedited development of PCS499. Because NL is a serious orphan condition with no existing treatments, the FDA has stated that PCS499 approval may be possible with one pivotal trial if the statistical significance of the one trial has a p << 0.05.
Despite the lack of FDA-approved treatments for NL, high doses of PTX have seen off-label use to treat NL patients. However, this form of treatment is only successful in a small percentage of cases, as high doses are not well tolerated by most patients. As an analog of PTX, PCS499 and its metabolites deliver approximately twice as much of the functional compound as PTX and are better tolerated by patients.
According to data collected from clinical experience and published case studies, it is possible for patients who can tolerate high doses of PTX to see significant improvement in ulcers within one to nine months. However, increasing PTX beyond its maximum dose (1.2 gm/day) to achieve a higher response rate can lead to dose-limiting side effects, including nausea, vomiting, and headaches. The Phase 2A study of PCS499 demonstrated that 1.8 gm/day is well-tolerated by patients and closed the ulcers entirely in the only two patients with severe ulcerative NL.
PCS499 breaks down into numerous active compounds, which may be ideal for treating NL.
Necrobiosis Lipoidica (NL) is a chronic, disfiguring skin condition with no currently approved FDA treatments. This condition is more common in individuals with diabetes and women, with an average age of onset between 20 and 60 years. Patients with NL experience persistent rashes that develop into ulcerated lesions in about one-third of cases. Ulceration can cause severe complications, such as life-threatening infections and necrosis.
NL patients in the U.S.1
NL patients worldwide2
1. Source: Muller SA, et al. Arch Dermatol. 1966; Jockenhöfer F, et al, J Dtsch Dermatol Ges. 2016; Company