Disc Medicine to submit NDA for bitopertin in EPP in October

WATERTOWN, Mass. - Disc Medicine, Inc. (NASDAQ:IRON), a $2 billion market cap biotechnology company with strong financial health according to InvestingPro metrics, announced today it has received positive feedback from its pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration regarding bitopertin for the treatment of erythropoietic protoporphyria (EPP).

The company, which maintains more cash than debt on its balance sheet, confirmed it is on track to submit an NDA for bitopertin in October 2025 under the FDA’s accelerated approval pathway, based on its existing data package. The pre-NDA meeting established alignment with the agency on the expected timing and content of the submission. Investors following this development should note that IRON’s next earnings report is scheduled for August 8, 2025.

"At the pre-NDA meeting, we confirmed alignment with the agency on the expected content of our NDA submission and, as a result, we are on track for a submission in October of this year," said John Quisel, President and Chief Executive Officer of Disc Medicine, in a press release statement.

Bitopertin is an oral inhibitor of glycine transporter 1 designed to modulate heme biosynthesis. The drug has been studied in multiple clinical trials for EPP, including the Phase 2 open-label BEACON trial, the Phase 2 double-blind, placebo-controlled AURORA trial, and an open-label extension HELIOS trial.

EPP is a rare genetic disorder that causes severe reactions when patients are exposed to sunlight, including pain, edema, and burning sensations. The condition can also lead to liver complications in 20-30% of patients. Currently, there is only one FDA-approved therapy for EPP.

If approved, bitopertin could potentially become the first disease-modifying therapy for EPP, according to the company. Disc Medicine obtained global rights to bitopertin under a license agreement from Roche in May 2021. Analysts maintain a strong bullish outlook on IRON, with price targets ranging from $75 to $132. For deeper insights into IRON’s valuation and growth potential, access the comprehensive Pro Research Report available on InvestingPro, which includes 8 additional key insights about the company’s financial health and market position.

In other recent news, Disc Medicine has seen several notable developments. Morgan Stanley initiated coverage on the company with an Overweight rating and set a price target of $85.00, citing the potential for bitopertin to reach the market sooner than anticipated. This optimism is echoed by H.C. Wainwright, which reaffirmed its Buy rating with a $118.00 price target, highlighting the progress of bitopertin in treating Erythropoietic Protoporphyria (EPP) and the promising prospects of DISC-0974 for myelofibrosis. Meanwhile, Raymond James reiterated its Strong Buy rating and $89.00 price target, as it awaits further data on DISC-0974, which has shown clinical benefits in early trials.

Additionally, Disc Medicine has appointed Nadim Ahmed to its board of directors. Ahmed, who brings over 25 years of leadership experience, currently serves as President and CEO of Cullinan Therapeutics. His expertise in hematological diseases is expected to aid Disc Medicine as it advances bitopertin towards commercialization. The company is also preparing for several catalysts in the coming months, including a potential New Drug Application filing for bitopertin in EPP. These updates reflect ongoing strategic and operational efforts by Disc Medicine to strengthen its market position.

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