WATERTOWN, Mass. - Disc Medicine Inc. (NASDAQ:IRON) announced today the topline results from its phase 2 study of bitopertin in patients with erythropoietic protoporphyria (EPP). The study met its primary endpoint, showing a dose-dependent, statistically significant reduction in protoporphyrin IX (PPIX) levels in both the 20 mg and 60 mg dose groups compared to placebo.
The AURORA study, a randomized, double-blind, placebo-controlled trial, enrolled 75 adult subjects with EPP, who were administered bitopertin or placebo once daily for 17 weeks. The results indicated that bitopertin led to a 21.6% reduction in PPIX levels for the 20 mg group and a 40.7% reduction for the 60 mg group. The placebo group, on the other hand, experienced an average increase of 8.0% in PPIX levels.
Secondary endpoints included cumulative total time in sunlight without pain and the rate of phototoxic reactions with pain. Although bitopertin-treated patients recorded more hours in sunlight without pain than those on placebo, the difference did not reach statistical significance. Still, there were significant improvements in the rate of phototoxic reactions with pain in the 60 mg dose group compared to placebo.
Patients also reported improvements in the Patient Global Impression of Change (PGIC), with the 60 mg dose group showing statistical significance. Bitopertin was generally well tolerated, with the most common adverse event reported being dizziness.
Despite the promising results, the company acknowledged that the key secondary endpoint of cumulative time in sunlight on days without pain did not meet statistical significance due to a strong placebo response. President and CEO John Quisel, J.D., Ph.D., noted that while additional analysis of the data is necessary, the company remains optimistic about the potential of bitopertin, particularly with upcoming readouts from other studies.
Bitopertin is a clinical-stage, orally-administered inhibitor of glycine transporter 1 (GlyT1) and is being developed as a potential treatment for a range of hematologic diseases, including erythropoietic porphyrias. EPP and X-linked Protoporphyria (XLP) are rare diseases that cause severe reactions to sunlight, leading to significant quality of life impairment.
The information in this article is based on a press release statement from Disc Medicine, Inc.
InvestingPro Insights
As Disc Medicine Inc. (NASDAQ:IRON) makes headlines with the success of its phase 2 study of bitopertin, investors are closely monitoring the financial health and market performance of the company. With a market capitalization of approximately $1.05 billion and a volatile price trajectory, IRON presents a complex picture.
InvestingPro data reveals a challenging financial situation, with a negative P/E ratio of -6.07 and an adjusted P/E ratio for the last twelve months as of Q4 2023 standing at -20.11. This indicates that the company is not currently profitable, a fact that is echoed by the negative operating income of -$91.12 million for the same period.
Despite these figures, IRON has experienced a significant one-year price total return of 193.82%, highlighting the potential that investors see in the company's future, particularly in light of recent clinical successes.
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