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Disc Medicine's Bitopertin Gains Momentum with Positive Regulatory Feedback and Analyst Upgrades

• Disc Medicine received positive feedback from the FDA regarding bitopertin for erythropoietic protoporphyria (EPP), potentially allowing for accelerated approval based on PPIX reduction. • Analyst ratings for Disc Medicine have been upgraded to 'Strong Buy' by Raymond James, with a price target of $110, citing the accelerated timeline for bitopertin's launch. • The company is expected to submit a New Drug Application (NDA) for bitopertin in the second half of 2025, with potential approval and commercial launch anticipated in 2026. • Phase 1b study of DISC-0974 showed significant reduction in hepcidin levels and improvement in iron mobilization and hemoglobin levels in patients with NDD-CKD and anemia.

Raymond James has upgraded Disc Medicine (NASDAQ:IRON) shares to Strong Buy, setting a new price target of $110.00, up from $66.00, following positive feedback from the FDA regarding its drug candidate bitopertin for treating erythropoietic protoporphyrias (EPP).

Regulatory Path for Bitopertin

The FDA indicated it would consider an accelerated approval filing based on protoporphyrin IX (PPIX) reduction as a surrogate biomarker of efficacy for bitopertin in EPP. This allows Disc Medicine to proceed with existing data. The APOLLO study is designed to demonstrate the safety and efficacy of bitopertin, with significant reductions in PPIX levels already observed in earlier studies, crucial for improving patients’ light tolerance.
The APOLLO study’s co-primary endpoints focus on clinically meaningful outcomes, such as change in PPIX from baseline and duration of sunlight exposure without pain. The expected timeline aligns with FDA guidance.

Analyst Outlook

Analyst Rami Katkhuda from LifeSci Capital maintained a Buy rating on Disc Medicine's stock with an $85.00 price target, citing the company's clear regulatory path for bitopertin in treating EPP. The expected NDA submission for bitopertin is planned for the second half of 2025, with approval and commercial launch anticipated in 2026. Stifel Nicolaus also assigned a Buy rating to the stock with a $94.00 price target.

Clinical Advancements

Disc Medicine reported promising results from its Phase 1b study of DISC-0974, which showed significant reduction in hepcidin levels and improvement in iron mobilization and hemoglobin levels in patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) and anemia.

Financial and Strategic Moves

Disc Medicine announced a public stock offering of approximately $178 million to advance research and clinical development of its product candidates. The company also appointed Dr. Rahul Rajan Kaushik as Chief Technical Officer and Dr. Steve Caffé as Chief Regulatory Officer.
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Disc Medicine's bitopertin may receive accelerated FDA approval for erythropoietic protoporphyria (EPP) treatment based on existing clinical data.
The FDA has agreed to the design of Disc's Phase III APOLLO trial, which will serve as a confirmatory study for bitopertin.

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Reference News

[1]
Disc Medicine's Promising Path: Buy Rating Backed by Regulatory Milestones and Market Potential
markets.businessinsider.com · Jan 21, 2025

Analyst Rami Katkhuda maintains a Buy rating on Disc Medicine, citing its promising developments, including a clear regu...

[2]
Promising Growth for Disc Medicine: Buy Rating Backed by Strategic Advancements and Clinical Progress
markets.businessinsider.com · Jan 28, 2025
[3]
Raymond James increases Disc Medicine shares to Strong Buy on new drug - Investing.com
investing.com · Nov 4, 2024

Raymond James upgraded Disc Medicine's stock to Strong Buy with a $110.00 price target, following a successful FDA meeti...

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