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H.C. Wainwright reiterated its Buy rating and $118.00 price target on Disc Medicine (NASDAQ:IRON) on Tuesday, falling within the broader analyst target range of $75-$132. The research firm cited positive data from the company’s HELIOS study, which evaluates bitopertin in erythropoietic protoporphyria (EPP) patients. According to InvestingPro, the company maintains a strong financial health score and holds more cash than debt on its balance sheet.
The HELIOS Phase 2 open-label extension study included 86 patients who received bitopertin at 60mg once daily. These patients were previously treated in the AURORA and BEACON studies, with data cut-off on February 4. With a market capitalization of $1.8 billion and a beta of 2.5, investors should note that IRON’s stock movements tend to be quite volatile.
H.C. Wainwright noted that bitopertin is "well positioned for accelerated approval, given consistent and durable efficacy," potentially becoming the first disease-modifying treatment for EPP. The firm also highlighted "early signs of liver function improvement" that could indicate long-term benefits beyond sunlight protection. For deeper insights into IRON’s valuation and 8 additional key ProTips, check out the comprehensive research report available on InvestingPro.
The positive HELIOS study results have strengthened H.C. Wainwright’s confidence in bitopertin’s potential to receive accelerated approval from regulators. The drug is being developed as a treatment for EPP, a rare genetic disorder that causes extreme sensitivity to sunlight.
Disc Medicine continues its clinical development program for bitopertin, building on data from its AURORA and BEACON studies that preceded the current HELIOS extension study.
In other recent news, Disc Medicine, Inc. presented promising data from its clinical programs at the European Hematology Association 2025 annual meeting in Milan. The company highlighted long-term data from the HELIOS open-label extension study of bitopertin, showing sustained reductions in protoporphyrin IX levels and improved quality of life measures for patients with erythropoietic protoporphyria. Disc Medicine plans to submit a New Drug Application for bitopertin in the second half of 2025. Additionally, the company reported positive results from its Phase 1b trial of DISC-0974 in myelofibrosis anemia, with 50% of non-transfusion dependent patients achieving a significant hemoglobin increase. The Phase 2 RALLY-MF trial is ongoing, with initial data expected later this year. Disc Medicine also shared findings from its Phase 1 study of DISC-3405, demonstrating significant reductions in serum iron levels, and has initiated a Phase 2 trial in polycythemia vera. In other developments, Disc Medicine held its Annual Meeting of Stockholders, where key proposals were voted upon, including the election of three Class II directors and the approval of executive compensation. Ernst & Young LLP was ratified as the company’s independent registered public accounting firm for the fiscal year ending December 31, 2025.
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