Disc Medicine

Today we reported our third quarter financial results and shared recent operational progress across our portfolio. A key highlight is receiving the FDA Commissioner’s National Priority Voucher (CNPV), designed to shorten the NDA review process of #Bitopertin in #ErythropoieticProtoporphyria (#EPP) to 1-2 months from NDA acceptance. Looking ahead, we’ll be presenting initial data from our Phase 2 RALLY-MF trial of DISC-0974 in patients with #Anemia of #Myelofibrosis at #ASH25, and data from recently completed multiple-dose cohorts of our Phase 1b study of DISC-0974 in patients with anemia of non-dialysis-dependent chronic #KidneyDisease at ASN #KidneyWk. We also initiated a Phase 1b study of DISC-3405 in #SickleCellDisease in October, and we’re progressing an ongoing Phase 2 study of DISC-3405 in #PolycythemiaVera. Read more about our clinical programs in the latest press release: https://lnkd.in/e646xdK6

Disc will share two poster presentations, including initial data from the RALLY-MF Phase 2 trial of DISC-0974 in #Anemia of #Myelofibrosis and the design of our ongoing Phase 2 trial of DISC-3405 in #PolycythemiaVera, at the 2025 American Society of Hematology Annual Meeting on December 6-9. Disc will also host a call during #ASH25 to review highlights of the presented data and plans for next steps in development on Sunday, December 7, at 7:30am EST. Please register for the event on the Events and Presentations page of Disc’s website. Join us in Orlando this December to explore innovations pushing the boundaries of #Hematology. Read more: https://lnkd.in/ewZ9FgYU

BiotechTV recently featured CEO John Quisel in a segment on Disc’s receipt of a Commissioner’s National Priority Voucher from the U.S. Food and Drug Administration for #Bitopertin in #ErythropoieticProtoporphyria (#EPP), including #XLinkedProtoporphyria (#XLP). Watch the full video: https://lnkd.in/gaWhrynm

We are excited to announce we have received a Commissioner’s National Priority Voucher (CNPV) from the U.S. Food and Drug Administration for #Bitopertin in #ErythropoieticProtoporphyria (#EPP), including #XLinkedProtoporphyria (#XLP). The CNPV is designed to accelerate the development and review of certain drugs aligned with U.S. national health priorities by offering the opportunity to reduce drug application review times to 1-2 months. Read the full announcement here: https://lnkd.in/eNbAjPDD

We are excited to share the publication of new preclinical study with Boston Children's Hospital showing bitopertin’s potential to lower PPIX levels and prevent liver disease in #ErythropoieticProtoporphyria. Thank you to the team for advancing this important science. Read here: https://lnkd.in/e4CCbguW

We are excited to announce submission of a New Drug Application to the U.S. Food and Drug Administration for #Bitopertin in #ErythropoieticProtoporphyria (#EPP) and #XLinkedProtoporphyria (#XLP) under the accelerated approval pathway. This milestone showcases our teams’ unwavering commitment to advancing potentially innovative therapies for blood disorders with high unmet need. Thank you to the investigators, patients, families, and caregivers whose commitment and participation made these clinical trials possible. Read the full announcement here: https://lnkd.in/eqgGNXx9

Grateful to Patti who is living with #PolycythemiaVera (PV), and Dr. Raajit Rampal for speaking to our team today in honor of last week’s #MPNAwarenessDay on September 11. Their lived and clinical experiences offered invaluable insight into #MyeloproliferativeNeoplasms, complex chronic blood cancers like PV and #Myelofibrosis. At Disc, we remain committed to advancing awareness, education, and innovation for people affected by these conditions. Learn more about our approach at www.discmedicine.com

Today on #MPNAwarenessDay, we stand with the community to raise awareness of #MyeloproliferativeNeoplasms – chronic blood cancers such as #Myelofibrosis and #PolycythemiaVera. At Disc Medicine, we are committed to advancing new approaches for patients with myelofibrosis and polycythemia vera, where new treatment options are urgently needed. That’s why we’re proud to highlight the MPN Research Foundation’s newly launched “You and MPN: Animated Patient Guide for Myeloproliferative Neoplasms,” a resource offering guidance for patients and families. Explore the site here: https://lnkd.in/eKt3xmBt

Today we reported our second quarter financial results and shared recent operational progress across our portfolio.    We are on track to submit an NDA for #Bitopertin in #ErythropoieticProtoporphyria under the accelerated approval pathway in October, supported by a successful pre-NDA meeting. We also initiated a Phase 2 study of DISC-3405 in #PolycythemiaVera, with initial data expected in 2026. Looking ahead, we anticipate data readouts for DISC-0974 in Q4 2025, including initial data from the Phase 2 study in patients with #Anemia of #Myelofibrosis and multiple dose data from the Phase 1b study in patients with anemia of non-dialysis-dependent chronic #KidneyDisease. Read more about our clinical programs in the latest press release: https://lnkd.in/e2ksyN3u

Disc is proud to announce our new campaign to raise awareness for Erythropoietic Protoporphyria (#EPP), including X-Linked Protoporphyria (#XLP), a rare, often underdiagnosed genetic disorder driven by the buildup of Protoporphyrin IX (#PPIX). This accumulation is not only responsible for the hallmark symptom of painful light sensitivity, but can also lead to progressive liver damage, affect bone health, and drive disease-related impact on quality of life, mental health, and emotional wellbeing. We built a site specifically for people living with EPP and their caregivers. It is here to help with: • Understanding EPP • Tips for managing symptoms • Resources to help build your care plan • Links to support networks and resources We also launched an HCP-focused site offering dedicated resources to support clinical practice, including: • Disease education on EPP • Guidance on diagnosis and testing • Information on long-term management We’re committed to deepening understanding of these conditions. Visit our site and explore what’s available: • Patients: PPIXiswhy.com • HCPs: PPIXiswhyhcp.com Additional resources: • Facebook: https://lnkd.in/e3N4xwn6   • Instagram: https://lnkd.in/e523CcTd  #ErythropoieticProtoporphyria #XLinkedProtoporphyria #ProtoporphyrinIX #PPIXisWhy