Disc Medicine Presents Positive Clinical and Translational Data Across Portfolio at the 66th American Society of Hematology (ASH) Annual Meeting

•  Positive updates across all programs, including updates from ongoing clinical studies and new translational data in preclinical models supporting use in existing and additional indications
• Management hosted a conference call during the ASH meeting on Sunday, December 8 discussing highlights of the presented data and next steps for the company

WATERTOWN, Mass., Dec. 09, 2024 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today spotlights 8 posters presented at the ASH 2024 annual meeting in San Diego, CA. This year’s presentations included updates from the BEACON and AURORA trials of bitopertin in patients with erythropoietic protoporphyria (EPP) and the Phase 1 SAD/MAD trial of DISC-3405 in healthy volunteers. Additionally, Disc presented a real-world patient survey highlighting the disease burden of EPP and multiple preclinical models highlighting the potential benefits of bitopertin, DISC-0974, and DISC-3405 in existing and new indications. The collection of data supports Disc’s continued advancement of all three clinical candidates and provides evidence for expansion opportunities in new indications.

In addition to its poster presentations, Disc presented complete results from the Phase 1b trial of DISC-0974 in anemia of myelofibrosis (MF) yesterday, December 8, in an oral presentation. These results demonstrated positive impact on clinically meaningful measures of anemia across a broad range of patient types and support advancement of the program into a Phase 2 trial in MF anemia, which is now initiated. Management hosted a call on Sunday, December 8 to review highlights of data presented throughout the ASH meeting and plans for next steps in development. The archive of the call is accessible on the Events and Presentations page of Disc’s website (https://ir.discmedicine.com/).

Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide.

Summary of Poster Presentations

Bitopertin:

Disc is advancing development and registrational activities for bitopertin in EPP, with the potential for accelerated approval using PPIX as a surrogate endpoint.

AURORA:

The AURORA study is a randomized, double-blind, placebo-controlled Phase 2 clinical trial that enrolled 75 adult subjects with EPP. Subjects were randomized 1:1:1 to receive 20 mg of bitopertin (n=26), 60 mg of bitopertin (n=25), or placebo (n=24) orally once daily for 17 weeks.

• Updated analyses show that bitopertin reduced PPIX in all prespecified subgroups across demographic and baseline patient characteristics
• Previously presented analyses showed that reductions in PPIX were associated with improvements in multiple clinical outcomes, including measures of sunlight tolerance, reductions in phototoxic reactions, and patient-reported quality of life

BEACON:

The BEACON study is a Phase 2, randomized, open-label, multiple dose clinical trial that enrolled 22 adults and 4 adolescents with EPP. Subjects were randomized 1:1 to receive 20 mg of bitopertin (n=14) or 60 mg of bitopertin (n=12) orally once daily for 24 weeks.

• Updated analyses show that bitopertin significantly reduced protoporphyrin IX (PPIX) at low and high doses and in both adult and adolescent populations
• Bitopertin had a meaningful impact on light tolerance, with similar benefit shown across adult and adolescent populations
• Reductions in PPIX were associated with improvements in multiple measures of sunlight tolerance
• Bitopertin was generally well tolerated and showed a similar safety profile in adults and adolescents

EPP LIGHT Study:

The EPP LIGHT Study is a patient survey study seeking to comprehensively describe the burden of disease in adults and adolescents with EPP.

• Across adult (n=164) and adolescent (n=33) respondents, EPP symptoms impact all facets of life including ability to be out in the sun for prolonged periods of time, ability to undertake daily activities, deficits in emotional functioning, and absenteeism at work and school
  • 68% of adults and 45% of adolescents experienced pain from a phototoxic reaction after