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WATERTOWN, Mass. - Disc Medicine, Inc. (NASDAQ:IRON), a $2.26 billion market cap biotech company trading near its 52-week high of $68.73, has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration for bitopertin, a treatment for patients aged 12 years and older with erythropoietic protoporphyria (EPP), including X-linked protoporphyria. According to InvestingPro analysis, the company maintains a strong balance sheet with more cash than debt.
The company is seeking approval through the FDA’s accelerated approval pathway, using reduction of protoporphyrin IX as a surrogate endpoint. Disc has also requested Priority Review status, which would shorten the review period from 10 months to six months following the 60-day filing review period.
The NDA submission is supported by results from the Phase 2 BEACON and AURORA studies, which demonstrated reductions in protoporphyrin IX levels and improvements in light tolerance, reduction of phototoxic reactions, and quality of life. The application also includes safety data from over 4,000 clinical trial participants from studies previously conducted by Roche.
Bitopertin is an oral inhibitor of glycine transporter 1 designed to modulate heme biosynthesis. The drug has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA.
EPP is a rare genetic disorder that causes severe reactions when patients are exposed to sunlight, including pain, burning sensations, and potential blistering. The condition can also lead to liver complications in 20-30% of patients.
"This NDA submission represents a pivotal moment not just for Disc but for the EPP community as we seek to provide patients with a treatment option that has the potential to address the underlying cause of disease," said John Quisel, Chief Executive Officer and President of Disc, according to the company’s press release.
The FDA is expected to make a decision on accepting and filing the NDA for review within 60 days of submission. While the company is not yet profitable, analysts tracked by InvestingPro are highly optimistic about its prospects, with price targets ranging from $85 to $132 per share. For deeper insights into Disc Medicine’s financial health and growth potential, investors can access the comprehensive Pro Research Report, available exclusively on InvestingPro along with 12 additional key ProTips about the company’s performance.
In other recent news, Disc Medicine, Inc. announced that it plans to submit a New Drug Application (NDA) for bitopertin, aimed at treating erythropoietic protoporphyria (EPP), to the U.S. Food and Drug Administration in October 2025. The company received positive feedback from its pre-NDA meeting with the FDA, aligning on the timing and content of the submission under the accelerated approval pathway. In analyst updates, Cantor Fitzgerald reiterated its Overweight rating with a $132 price target on Disc Medicine, expressing confidence in the company’s program. Similarly, Truist Securities initiated coverage with a Buy rating and an $86 price target, citing the company’s promising pipeline in hematological disorders. Raymond James also maintained its Strong Buy rating with an $89 price target, following Bristol Myers Squibb’s trial results, which highlighted improvements in red blood cell transfusion. Additionally, Disc Medicine appointed Nadim Ahmed to its board of directors, effective immediately, as a class I director until the 2027 annual meeting. These developments reflect ongoing strategic and operational progress within the company.
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