Direktnyheter • May 14
Disc Medicine Previews Positive Hematology Trial Data While Facing FDA and Legal Challenges Disc Medicine will present Phase 2 RALLY-MF trial data for DISC-0974 in myelofibrosis-related anemia at the EHA 2026 Congress, with the trial reporting an overall anemia response rate of 70% that was consistent with or without JAK inhibitor use.
The company will also share new data on bitopertin in erythropoietic protoporphyria at EHA 2026 and hold a corporate update call on June 15, 2026 to discuss clinical results and development plans.
Rosen Law Firm is investigating potential securities claims after the FDA issued a Complete Response Letter on February 13, 2026 declining approval of Disc Medicine’s bitopertin NDA, an event followed by a 22% drop in the stock and allegations of misleading information about the program.
Taken together, Disc Medicine sits at a crossroads where encouraging clinical signals for DISC-0974 and bitopertin are paired with legal and regulatory overhang from the bitopertin FDA setback.
You should weigh the potential value of upcoming data and management’s development plans against the risks from the ongoing investigation, stock volatility and the extra time and cost that further bitopertin evidence may require. Tillkännagivande • Apr 29
Disc Medicine, Inc., Annual General Meeting, Jun 18, 2026 Disc Medicine, Inc., Annual General Meeting, Jun 18, 2026. Tillkännagivande • Mar 28
Disc Medicine Announces Completion of Enrollment of Phase 3 Apollo Trial of Bitopertin in Erythropoietic Protoporphyria Disc Medicine, Inc. announced that the last participant has been randomized and dosed in the pivotal Phase 3 APOLLO trial of bitopertin in erythropoietic protoporphyria (EPP). Originally planned as an N=150 study, the study was expanded to 183 participants due to patient and physician demand. APOLLO is a double-blind, placebo-controlled Phase 3 study of bitopertin in patients ages 12 and above with EPP and X-linked protoporphyria (XLP), that includes sites in the US, Canada, Europe, and Australia. The co-primary endpoints are average monthly total time in sunlight without pain between 10:00 and 18:00 during the last month of the 6-month treatment period and percent change from baseline in whole blood metal-free PPIX after 6 months of treatment. Additional study design details, including powering assumptions, are provided in the corporate presentation on Disc’s website. Data from APOLLO is expected in Fourth Quarter 2026, after which a CRL response will be submitted with an FDA decision expected by mid-2027. Bitopertin is an investigational, clinical-stage, orally administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is developing bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Bitopertin has been studied in multiple clinical trials in patients with EPP, including the Phase 2 open-label BEACON trial, the Phase 2 double-blind, placebo-controlled AURORA trial, an open-label extension HELIOS trial, and the Phase 3 double-blind, placebo-controlled APOLLO trial. Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021. Tillkännagivande • Feb 14
Disc Medicine, Inc. Receives Complete Response Letter from FDA for Bitopertin for the Treatment of EPP Disc Medicine, Inc. announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the New Drug Application (NDA) for bitopertin as a treatment for patients with erythropoietic protoporphyria (EPP). Bitopertin has been under review for accelerated approval and as part of the Commissioner’s National Priority Voucher (CNPV) pilot program. Accelerated approval relies on (1) whether there is evidence of an effect on the proposed surrogate endpoint (% change in whole blood metal-free PPIX) and (2) whether the proposed surrogate endpoint, including the magnitude of change, is reasonably likely to predict a clinical benefit. On the first point, the FDA agreed that AURORA and BEACON provided sufficient evidence that bitopertin significantly lowers whole blood metal-free PPIX. On the second, based on review of AURORA and BEACON results, the FDA concluded that the trials did not show evidence of association between percent change in PPIX and sunlight exposure-based endpoints, as measured in the trials, despite the strong mechanistic and biological plausibility supporting the use of the PPIX biomarker in protoporphyria. The FDA indicated results of the APOLLO study could serve as evidence to support traditional approval. Disc believes the issue raised is readily addressable, given the APOLLO study is already well underway with topline data expected in fourth quarter. Disc plans to request a Type A meeting to review approach with the FDA. A blinded sample size re-estimation of the APOLLO study was conducted in January and no modifications to sample size were needed based on statistical analysis. There has been significant patient and physician enthusiasm around the APOLLO trial, allowing Disc to complete trial enrollment in March 2026, several months earlier than expected. Upon completion of APOLLO, Disc would then file a response to the CRL and expect an updated FDA decision by mid-2027. Disc has approximately $791 million at December 31, 2025 in unaudited cash, cash equivalents, and marketable securities and maintains guidance of providing runway into 2029. Tillkännagivande • Jan 13
Disc Medicine Inc Appoints Lisa Amaya Price as Chief Human Resources Officer Disc Medicine Inc. announced the appointment of Lisa Amaya Price, MBA as the company’s Chief Human Resources Officer. Ms. Amaya Price brings more than 25 years of experience in human resources and organizational leadership, including at multiple commercial-stage companies. Ms. Amaya Price joins Disc Medicine from Deciphera where she served as SVP and Chief HR Officer, helping lead the company’s integration into ONO Pharmaceuticals. Prior to this, she was the Chief People Officer at LGC Group, a private-equity global life science tools company with over 4,000 employees worldwide. Ms. Amaya Price was also a senior HR leader at Scholar Rock as well as Takeda Pharmaceuticals where she led organizational design and talent selection related to the acquisition of Shire Pharmaceuticals. Prior to Takeda, she spent 8 years at Biogen, serving in various positions of increasing responsibility, including most recently Senior Director, Human Resources of R&D. Earlier in her career, she held human resource leadership positions at Interpublic Group, Inc., Metro International S.A., and American Financial Systems, Inc. Ms. Amaya Price received a BA from Bard College, an MA from Simmons College, and an MBA from F.W. Olin Graduate School of Business at Babson College. Tillkännagivande • Dec 06
Disc Medicine, Inc. Presents Positive Initial Data from Rally-MF Phase 2 Trial in Patients with Myelofibrosis (MF) and Anemia at the 67th American Society of Hematology (ASH) Annual Meeting Disc Medicine, Inc. presented positive initial data from the RALLY-MF Phase 2 trial of DISC-0974 in anemia of MF at the ASH Annual Meeting in Orlando, FL. The data demonstrated that treatment with DISC-0974 resulted in substantial reductions in hepcidin and increases in iron levels translating to positive impact on clinically meaningful measures of anemia across a broad range of patient types. This ongoing Phase 2 open-label study had enrolled 47 adult patients with MF and anemia as of the data cutoff date of October 16, including 34 patients with sufficient follow up to be included in the responder analysis (non-transfusion dependent receiving no transfusions (nTD, n=24), transfusion dependent with low transfusion burden (TD Low, n=7) and transfusion dependent with high transfusion burden (TD High, n=3)). The trial was comprised of both patients receiving concomitant JAK inhibitor therapy (n=18) and not receiving JAK inhibitor therapy (n=16). DISC-0974 was administered subcutaneously at 50 mg every 4 weeks for up to 6 treatments. Initial results demonstrated:
Consistent, substantial decreases in hepcidin reaching >75% reduction from baseline and corresponding increases in serum iron. 63% of baseline nTD patients achieved a hemoglobin increase of =1 g/dL for =12 weeks (overall response) and 50% had an increase of =1.5 g/dL for =12 weeks (major response). 71% of TD Low patients achieved transfusion independence (TI, major response) over a 16-week period. 67% of TD High patients with at least 85 days on study achieved a =50% reduction in transfusion requirement (overall response). Initial data for additional n=3 TD High patients trending towards major response of TI >12 weeks. 50% of patients receiving concomitant JAK inhibitor therapy achieved a major hematologic response. Dosing with DISC-0974 was associated with improvements in FACIT-Fatigue scores in nTD and TD Low participants. DISC-0974 was generally well-tolerated. Diarrhea and urinary tract infections, neither considered serious, were the only adverse events (AE) that were considered related to DISC-0974 and reported in two or more subjects. The majority of AEs were not considered related to DISC-0974. Additional data to be shared in second half of 2026. Tillkännagivande • Oct 17
Disc Medicine, Inc. Announces Receipt of FDA Commissioner's National Priority Voucher (CNPV) for Bitopertin in Erythropoietic Protoporphyria Disc Medicine, Inc. announced that it received a Commissioner's National Priority Voucher (CNPV) from the U.S. Food and Drug Administration (FDA) for bitopertin in erythropoietic Protoporphyria (EPP), including X-linked protoporphyria (XLP). Disc submitted a New Drug Application (NDA) to the FDA for bitopertin for patients aged 12 years and older with EPP following the FDA's accelerated approval pathway in September 2025. Bitopertin has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA. The Commissioner's National Priority Vouchers program, announced in June 2025, is designed to accelerate the development and review of certain drugs aligned with US national health priorities by offering the opportunity to reduce drug application review times to 1-2 months. Additionally, companies selected for the program will be issued a voucher entitling them to benefits including enhanced communications and rolling review to allow for shortened review time. Erythropoietic protoporphyria is a rare, debilitating and potentially life-threatening disease caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate called protoporphyrin IX (PPIX). This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensations and potential blistering and disfigurement. PIX also accumulates in the hepatobiliary system and can result in complications including gallstones, cholestasis, and liver damage in 20-30% of patients and in extreme cases liver failure. Current standard of care involves extreme measures to avoid sunlight, including restricting outdoor activities to nighttime, use of protective clothing and opaque shields, and pain management. This has a significant impact on the psychosocial development, quality of life, and daily activities of patients, particularly in young children and families. Bitopertin is an investigational, clinical-stage, orally administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is developing bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Bitopertin has been studied in multiple clinical trials in patients with EPP, including the Phase 2 open-label BEACON trial, the Phase 2 double-blind, placebo-controlled AURORA trial, an open-label extension HELIOS trial, and the confirmatory Phase 3 double-blind, placebo- controlled APOLLO trial. Bitopertin is a investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021. Tillkännagivande • Oct 07
Disc Medicine, Inc. Announces Resignation of Mona Ashiya as Member of Board of Directors and Member of the Compensation Committee and the Nominating and Corporate Governance Committee, Effective October 3, 2025 On October 3, 2025, Mona Ashiya, Ph.D., a Class I member of the Board of Directors (the “Board”) of Disc Medicine, Inc. (the “Company”), notified the Company of her resignation from the Board, effective October 3, 2025. Dr. Ashiya was a member of the Compensation Committee and the Nominating and Corporate Governance Committee of the Board and, by resigning from the Board, also resigned from such committees. Dr. Ashiya's decision to resign was not the result of any disagreement with the Company on any matter relating to the Company’s operations, policies or practices. Effective upon Dr. Ashiya’s resignation, the size of the Board was reduced from nine members to eight. Tillkännagivande • Oct 02
Disc Medicine, Inc. Announces Submission of New Drug Application to US FDA for Accelerated Approval of Bitopertin for Patients with Erythropoietic Protoporphyria (Epp) Disc Medicine, Inc. announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bitopertin for patients aged 12 years and older with erythropoietic protoporphyria (EPP), including X-linked protoporphyria (XLP). Disc submitted the NDA under the FDA's accelerated approval pathway using reduction of protoporphyrin IX (PPIX) as a surrogate endpoint and requested a Priority Review based on bitopertin's potential to address the significant unmet need for EPP patients. Bitopertin has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA. The BEACON and AURORA studies demonstrated significant reductions in PPIX and improvements across key aspects of the disease, including improvements in light tolerance, reduction of phototoxic reactions, and improvements in quality of life. Disc is also studying bitopertin in a long-term extension study, HELIOS, and initiated the APOLLO confirmatory study in April 2025. The NDA includes a request for Priority Review, which, if granted, would accelerate the timing of the FDA's goal for review of the application to six months following the end of the 60-day filing review period rather than the standard 10-month review period. Priority Review status is designated for drugs that may offer a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious condition. Bitopertin is an investigational, clinical-stage, orally administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Bitopertin has been studied in multiple clinical trials in patients with EPP, including the Phase 2 open-label BEACON trial, the Phase 2 double-blind, placebo-controlled APOLLO trial. Bitopertin is a investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021. Erythropoietic protop orphyria (EPP),including X-linked Protoporphyria (XLP), is a rare, debilitating and potentially life-threatening disease caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate called protoporphyrin IX. This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensation and potential blistering and disfigurement.PPIX also accumulates in the most common common common disease. Tillkännagivande • Jul 22
Disc Medicine, Inc. Announces Positive Pre-Nda Meeting and Confirms Plans to Submit Nda for Bitopertin in Epp in October 2025 Disc Medicine, Inc. announced positive feedback from its pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) to discuss the planned NDA submission for bitopertin in EPP. The purpose of the meeting was to confirm the FDA's expectations for the proposed timing, format and content of the planned NDA submission. Based on feedback from the FDA, Disc plans to submit an NDA for bitopertin inEPP in October 2025 under the FDA's accelerated approval pathway based on Disc's existing data package. Bitopertin is an investigational, clinical-stage, orally administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases includingerythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Bitopertin has been studied in multiple clinical trials in patients with 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. Bitopertin is a investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensation and potential blistering and disfigurement. PIX also accumulates in the hepatobiliary system and can result in complications including gallstones, cholestasis, and liver damage in 20-30% of patients and in extreme cases liver failure. Current standard of care involves extreme measures to avoid sunlight, including restricting outdoor activities to nighttime, use of protective clothing and opaque shields, and pain management. This has a significant impact on the psychosocial development, quality of life, and daily activities of patients, particularly in young children and families. There is currently no cure for EPP and only one FDA-approved therapy, a surgically implanted synthetic hormone designed to stimulate melanin production called Scenesse®? (afamelanotide). Tillkännagivande • Jul 15
Disc Medicine, Inc. Appoints Nadim Ahmed to Its Board of Directors Disc Medicine, Inc. announced the appointment of Nadim Ahmed to its Board of Directors. Mr. Ahmed is currently the President and Chief Executive Officer of Cullinan Therapeutics. Mr. Ahmed joins the Disc Medicine Board with more than twenty-five years of leadership experience across a range of development and commercialization roles. He is currently the President and Chief Executive Officer at Cullinan Therapeutics, a biopharmaceutical company developing a pipeline of clinical-stage programs focused on oncology and autoimmune diseases. Prior to Cullinan Therapeutics, Mr. Ahmed worked at Bristol Myers Squibb, where he served as President, Hematology and at Celgene Corporation where he served as President, Global Hematology & Oncology. At Bristol Myers Squibb and Celgene, Mr. Ahmed oversaw multiple product launches and served as a member of both companies’ Leadership Teams. He holds a Master of Science degree from Loughborough University, UK and a Bachelor of Science degree from University College London, UK. Tillkännagivande • Jun 12
Disc Medicine Presents Positive Clinical Data Updates Across Portfolio At the European Hematology Association 2025 Annual Congress Disc Medicine, Inc. spotlights 5 posters presented at the EHA 2025 annual meeting in Milan, Italy. This year’s presentations included data from HELIOS, an ongoing open-label extension study of bitopertin in EPP, which showed favorable long-term efficacy and safety with sustained protoporphyrin IX (PPIX) reductions, improvement in quality of life, and improved liver biomarkers. Disc is advancing development and registrational activities for bitopertin in EPP, with plans to submit an NDA in H2 2025. The company has initiated APOLLO, a confirmatory clinical trial of bitopertin in adults and adolescents with EPP. Disc recently launched a campaign to raise awareness of EPP among physicians, patients, and caregivers with emphasis on the causative role of PPIX accumulation in the disease. Disc also presented longer term data from the continuation phase of its Phase 1b trial in MF anemia, showing sustained activity on key biomarkers and durable anemia response among major responders. Enrollment for the Phase 2 RALLY-MF trial of DISC-0974 is ongoing with the exploratory cohort for patients on concomitant momelotinib or pacritinib fully enrolled. The company expects to present initial RALLY-MF data in H2 2025. Additionally, Disc shared an update of the Phase 1 healthy volunteer trial of DISC-3405, initially presented at ASH 2024, which demonstrated deep, sustained reductions in serum iron and meaningful changes in hematologic parameters, establishing proof of mechanism. Based on these data, the company has initiated a Phase 2 trial of DISC-3405 in polycythemia vera (PV). Disc also presented an iron pulse study of DISC-3405 in healthy volunteers which showed inhibition of dietary iron uptake, further confirming the mechanism of action and supporting DISC-3405’s potential in diseases of iron overload. Tillkännagivande • Apr 26
Disc Medicine, Inc., Annual General Meeting, Jun 11, 2025 Disc Medicine, Inc., Annual General Meeting, Jun 11, 2025. Tillkännagivande • Mar 01
Disc Medicine, Inc. Announces That the Presentation of Full Results from Phase 2 Aurora and Beacon Studies Disc Medicine, Inc. announced that the Presentation of full results from Phase 2 AURORA and BEACON studies at 2024 ASH Annual Meeting demonstrating significant reductions in PPIX are associated with substantial improvements in time spent in sunlight, measures of quality of life, and reduction in phototoxic reactions. Positive end-of-phase 2 meeting with FDA providing a path toward potential accelerated approval for bitopertin in EPP with protoporphyrin IX (PPIX) reduction as a surrogate endpoint. Positive Type C meeting with FDA to achieve regulatory alignment on APOLLO post-marketing confirmatory trial design; on track to initiate trial by mid-year 2025. Planning to submit NDA under accelerated approval pathway in H2 2025 based on existing clinical data, including results from BEACON and AURORA Phase 2 trials. Presented positive data from the Phase 1b/2 study of DISC-0974 in anemia of myelofibrosis (MF), demonstrating robust and broad hematologic activity across patient segments Results showed substantial and durable improvements in hemoglobin, reductions in transfusion burden, and improvements in fatigue scores. Initiated the Phase 2 portion of the study in December 2024, enrolling a broad range of patients after positive discussions with FDA. Presented data from initial cohorts of ongoing Phase 1b study of DISC-0974 in patients with anemia of NDD-CKD, demonstrating hematologic activity following a single dose. Presented preclinical data at ASH 2024 demonstrating the potential of DISC-0974 to treat anemia of chronic inflammatory diseases such as IBD. The Company expects initial data from the ongoing Phase 2 MF anemia trial and multiple-dose data from the ongoing Phase 1b NDD-CKD trial in H2 2025. Presented complete data from the Phase 1 SAD/MAD trial in healthy volunteers, demonstrating proof-of-mechanism with substantial, dose-dependent increases in hepcidin and reductions in serum iron supportive of a once-monthly dosing regimen. Presented positive preclinical data in sickle cell disease highlighting the potential for DISC-3405 to provide therapeutic benefit in SCD by restricting iron. The Company plans to initiate a Phase 2 study in PV in H1 2025. Tillkännagivande • Jan 24
Disc Medicine, Inc. has completed a Follow-on Equity Offering in the amount of $225.499982 million. Disc Medicine, Inc. has completed a Follow-on Equity Offering in the amount of $225.499982 million.
Security Name: Common Stock
Security Type: Common Stock
Securities Offered: 3,918,182
Price\Range: $55
Discount Per Security: $3.3
Security Name: Pre-Funded Warrants
Security Type: Equity Warrant
Securities Offered: 181,818
Price\Range: $54.9999
Discount Per Security: $3.3 Tillkännagivande • Dec 09
Disc Medicine Presents Positive Updated Results from Phase 1B Trial in Patients with Myelofibrosis and Anemia in an Oral Presentation At the 66Th American Society of Hematology Annual Meeting Disc Medicine, Inc. presented positive updated results from a Phase 1b trial of DISC-0974 in patients with myelofibrosis and anemia. The data, presented in an oral session at the 2024 American Society of Hematology annual meeting in San Diego, CA, demonstrated that treatment with DISC-0974 results in substantial reductions in hepcidin and increases in iron levels translating to positive impact on clinically meaningful measures of anemia across a broad range of patient types. This Phase 1b multi-center, open-label study, enrolled 35 adult patients with MF and anemia, including patients who were: non-transfusion dependent receiving no transfusions (nTD, n=23), transfusion dependent with low transfusion burden (TD Low, n=5) and transfusion dependent with high transfusion burden (TD High, n=7). The trial was comprised of both patients receiving concomitant JAK inhibitor therapy (n=13) and not receiving JAK inhibitor therapy (n=22). DISC-0974 was administered subcutaneously at 14 mg (n=1), 28 mg (n=7), 50 mg (n=12), 75 mg (n=9), or 100 mg (n=6) every 4 weeks for up to 6 treatments. Results demonstrated: Consistent, substantial decreases in hepcidin reaching >75% reduction from baseline and corresponding increases in serum iron across patients, which translated to increased levels of reticulocyte hemoglobin and hemoglobin 68% of baseline nTD patients achieved a hemoglobin increase of =1.5 g/dL during study period and 50% had sustained increases for =12 weeks 100% of TD patients with 1-2 transfusions within a 12-week period at baseline (TD Low) achieved a =50% reduction in transfusion requirement 80% of TD Low patients achieved transfusion independence (TI) over a 16-week period 60% of TD patients with 3-12 transfusions within a 12-week period at baseline (TD High) achieved a =50% reduction in transfusion requirement 40% of TD High patients achieved transfusion independence over a 12-week period 54% of patients receiving concomitant JAK inhibitor therapy achieved a major hematologic response DISC-0974 was generally well-tolerated at all evaluated dose levels. Diarrhea was the only adverse event that was considered related to DISC-0974 and reported in two or more subjects. The majority of AEs were not considered related to DISC-0974. DISC-0974 is an investigational monoclonal antibody (mAb) targeting a BMP-signaling co-receptor called hemojuvelin (HJV) and is designed to suppress hepcidin production and increase serum iron levels in patients suffering from anemia of inflammation. DISC-0974 was in-licensed by Disc from AbbVie in 2019. Anemia of inflammation arises from abnormally elevated hepcidin and is the second most common form of anemia, affecting millions of patients in the US across numerous diseases such as chronic kidney disease, myelofibrosis, cancer, autoimmune diseases, and other conditions with an inflammatory component. Disc has established clinical proof-of-mechanism of DISC-0974 in a Phase 1 trial of healthy volunteers, completed a Phase 1b clinical trial in patients with myelofibrosis and anemia, and initiated a Phase 2 clinical trial of DISC-0974 in patients with MF anemia, as well as a Phase 1b/2a clinical trial of DISC-0974 in patients with chronic kidney disease and anemia who are not receiving dialysis. DISC-0974 is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Myelofibrosis (MF) is a rare, chronic blood cancer that currently affects an estimated 25,000 patients in the United States alone. Severe, progressive, and treatment resistant anemia is the primary clinical manifestation of MF. At diagnosis, over 80% of MF patients have anemia, which progressively worsens and ultimately renders the majority of patients dependent on chronic red blood cell transfusions. Recent studies have shown hepcidin to be a key molecular driver of anemia in myelofibrosis. Hepcidin is elevated by approximately 12-fold in MF patients, and is correlated with disease severity, anemia, and the need for red blood cell transfusions. Tillkännagivande • Oct 25
Disc Medicine, Inc. Presents Positive Data from SAD Cohorts of a Phase 1b Trial in Patients with Chronic Kidney Disease (CKD) and Anemia at the 2024 American Society of Nephrology (ASN) Kidney Week Disc Medicine, Inc. presented positive additional data from an ongoing Phase 1b study of DISC-0974 in patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) and anemia, including results from the 40 mg and 60 mg single ascending dose (SAD) cohorts. The data presented at the 2024 American Society of Nephrology (ASN) Kidney Week in San Diego, CA demonstrated that a single dose of DISC-0974 results in sustained suppression of hepcidin and mobilization of iron, and increased erythropoiesis and levels of hemoglobin in NDD-CKD patients with anemia. In the SAD portion of this study, participants with Stage 2-5 NDD-CKD were given a single dose of placebo (n=7) or DISC-0974 subcutaneously (SC) at 28 mg (n=9), 40 mg (n=6), or 60 mg (n=6). Dose escalation is ongoing in the SAD. This interim data set demonstrated: Substantial, durable, dose-dependent reduction in hepcidin from baseline compared to placebo across dose levels, with median reduction greater than 75% from baseline at highest dose level; Meaningful and sustained increase in transferrin saturation from baseline compared to placebo across dose levels, with median increase up to 3x from baseline at highest dose level; Early and sustained increase in mean reticulocyte hemoglobin from baseline across all dose groups through Day 22 and beyond: Maximal mean values through Day 22 of +1.14 pg at 28 mg, +1.49 pg at 40 mg, and +1.53 pg at 60 mg, compared with +0.21 pg on placebo. Increase in mean hemoglobin from baseline across dose groups over the study period: Change greater than placebo: +0.35 g/dL at 28 mg, +0.54 g/dL at 40 mg, and +0.55 g/dL at 60 mg. Mean maximal increase in hemoglobin of +0.8 g/dL at 40 mg and +0.7 g/dL at 60 mg compared with +0.2 g/dL on placebo: Maximal observed individual increase in hemoglobin up to +0.95 g/dL at 28 mg, +1.5 g/dL at 40 mg, and +1.8 g/dL at 60 mg. DISC-0974 demonstrated acceptable safety and tolerability at all evaluated dose levels: The majority of adverse events were deemed not related to DISC-0974 and all adverse events assessed as treatment-related were Grade 1 or 2. DISC-0974 is an investigational agent and is not approved for therapeutic use in any jurisdiction worldwide. Tillkännagivande • Sep 19
Disc Medicine, Inc. Announces Appointment of Steve Caffé as Chief Regulatory Officer Disc Medicine, Inc. announced the appointment of Steve Caffé, MD as the company’s Chief Regulatory Officer. Dr. Caffé is an experienced biotech executive with significant expertise in global regulatory leadership across a wide range of therapeutic areas, including hematology, oncology, and rare diseases. Dr. Caffé has more than 25 years of experience in global product development and regulatory affairs, having held senior leadership positions at several top biotechnology companies. Most recently, he served as Head of Regulatory Affairs at CRISPR Therapeutics where he was involved in the development and approval of Casgevy (exagamglogene autotemcel) for sickle cell disease and beta thalassemia. Prior to joining CRISPR, Dr. Caffé was the Senior Vice President leading Regulatory Affairs, Pharmacovigilance, Quality, and Patient Advocacy at Ra Pharmaceuticals. Before this, Dr. Caffé held senior-level regulatory positions at a number of other biopharmaceutical companies, including Sucampo Pharmaceuticals, AMAG Pharmaceuticals, MedImmune (Biologics Division of AstraZeneca), Baxter, Sanofi-Aventis and Merck. Across these experiences, Dr. Caffé has contributed to over 40 new drug approvals and major new indications worldwide in a wide range of therapeutic areas. Steve received his M.D. at the Université Pierre et Marie Curie in Paris, France. Tillkännagivande • May 03
Disc Medicine, Inc., Annual General Meeting, Jun 12, 2024 Disc Medicine, Inc., Annual General Meeting, Jun 12, 2024, at 09:00 US Eastern Standard Time. Agenda: To elect two Class I directors to our board of directors, to serve until the 2027 annual meeting of stockholders and until their successors have been duly elected and qualified, or until their earlier death, resignation or removal; to ratify the appointment of Ernst & Young LLP as our independent registered public accounting firm for the fiscal year ending December 31, 2024; and to transact any other business properly brought before the Annual Meeting or any adjournment or postponement of the Annual Meeting. Recent Insider Transactions Derivative • Apr 14
Independent Director notifies of intention to sell stock William White intends to sell 5k shares in the next 90 days after lodging an Intent To Sell Form on the 11th of April. If the sale is conducted around the recent share price of US$31.52, it would amount to US$155k. Since June 2023, William has not owned shares directly (This sale likely refers to shares that have not yet been received). Company insiders have collectively sold US$3.2m more than they bought, via options and on-market transactions in the last 12 months. Tillkännagivande • Apr 02
Disc Medicine, Inc. Reports Topline Results from Phase 2 AURORA Study of Bitopertin in Patients with Erythropoietic Porphyria (EPP) Disc Medicine, Inc. presented topline data from AURORA, a phase 2 study of bitopertin in patients with EPP. Treatment with bitopertin resulted in statistically significant reductions in PPIX, the primary endpoint, and significant improvements in the rate of phototoxic reactions with pain and the Patient Global Impression of Change (PGIC). On the key secondary endpoint of cumulative time in sunlight on days without pain, bitopertin patients had a positive response consistent with BEACON results, but the endpoint did not meet statistical significance due to strong placebo performance. The AURORA study is a randomized, double-blind, placebo-controlled phase 2 study that enrolled 75 adult subjects with EPP. Subjects were randomized 1:1:1 to receive 20 mg of bitopertin, 60 mg of bitopertin, or placebo once daily for 17 weeks. Summary of topline results: Primary endpoint: Bitopertin resulted in significant, dose-dependent, and sustained reductions in whole blood PPIX levels: -21.6% for 20 mg (p=0.003 vs placebo) and -40.7% for 60 mg (p<0.001 vs placebo); the placebo group had mean increases of +8.0%. Secondary endpoints. Cumulative total time in sunlight between 10 am and 6 pm on days without pain observed over the 4-month treatment period: bitopertin-treated patients recorded a mean of 175.1 hours at 20 mg and 153.1 hours at 60 mg, compared with 133.9 hours for placebo; results were not statistically significant compared to placebo. The magnitude of the improvement in the bitopertin-treated patients was comparable to that observed in the BEACON study, but the benefit in the placebo arm in the AURORA trial was greater than expected. Large improvements in light tolerance from baseline in 20 mg and 60 mg bitopertin treatment groups as measured by time to prodrome; results were not statistically significant relative to placebo. Substantial and dose-dependent reductions in phototoxic reactions with pain during the 4-month study period: 75% reduction in the incidence rate of new phototoxic reactions with pain at the 60 mg dose group compared to placebo (p=0.011); 60% reduction in the 20 mg dose group (p=0.109). Fewer bitopertin-treated patients reported a phototoxic event compared to placebo: 19% for 20 mg and 12% for 60 mg compared to 46% for placebo. Dose-dependent improvements in PGIC, which were statistically significant for the 60 mg dose: 77% of completers at 20 mg and 86% at 60 mg (p=0.022 vs placebo) reported that their EPP was “much better” compared with 50% for placebo. Bitopertin was generally well tolerated in both dose groups with no serious adverse events and stable hemoglobin levels. Two patients discontinued treatment due to treatment-emergent AEs, both in the 60 mg dose group: one due to dizziness and one due to a skin rash. The most common AE reported with bitopertin treatment was dizziness: n=4 in the 20 mg dose group, n=11 in the 60 mg dose group compared with n=4 in the placebo group (median durations of 4.5, 5, and 2 days, respectively). New Risk • Apr 01
New major risk - Revenue and earnings growth Earnings are forecast to decline by an average of 9.9% per year for the foreseeable future. This is considered a major risk. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. If profits are expected to decline, then in most cases the share price will decline over time as well. In addition, if the company pays dividends it will also likely need to reduce or cut them, striking a dual blow to total shareholder returns. Currently, the following risks have been identified for the company: Major Risks Earnings are forecast to decline by an average of 9.9% per year for the foreseeable future. Revenue is less than US$1m. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$174m net loss in 3 years). Share price has been volatile over the past 3 months (15% average weekly change). Shareholders have been diluted in the past year (30% increase in shares outstanding). Tillkännagivande • Feb 27
Disc Medicine, Inc. Appoints Pamela Stephenson as Chief Commercial Officer Disc Medicine, Inc. announced the appointment of Pamela Stephenson, MPH as the company’s Chief Commercial Officer. Ms. Stephenson is an experienced biotech executive with significant expertise in commercial and strategic leadership, particularly in therapies for rare diseases, and will be responsible for all aspects of Disc’s global commercialization strategy and execution. Ms. Stephenson has more than 25 years of biopharma commercial leadership experience across multiple functions and disease areas. Most recently, she served as the Chief Commercial Officer of Albireo, which was acquired by Ipsen, spearheading the successful launch of Bylvay for a rare cholestatic liver disease. Before joining Albireo, Ms. Stephenson served as Vice President, Global Market Access and Value, at Vertex Pharmaceuticals, where she led the global market access and pricing strategy for current and future products. Earlier in her tenure at Vertex, she led marketing and sales activities for the company’s hepatitis C and cystic fibrosis lines of business, and oversaw the U.S. launches of Incivek® (telaprevir) and Orkambi® (lumacaftor/ivacaftor). Prior to Vertex, Ms. Stephenson spent 10 years at Pfizer in marketing roles of increasing responsibility for brands such as Viagra® (sildenafil citrate), Lyrica® (pregabalin), and Aromasin® (exemestane). Ms. Stephenson served as board member of Zynerba Pharmaceuticals, a public biotech company focused on developing therapeutics for patients with rare neuropsychiatric conditions, which was acquired by Harmony Biosciences in October 2023. Ms. Stephenson holds a bachelor’s degree from Brown University and received her master’s degree in public health from Boston University School of Public Health. In connection with Ms. Stephenson’s appointment, Disc granted to Ms. Stephenson an inducement equity award outside of Disc’s Amended and Restated 2021 Stock Option and Incentive Plan in accordance with NASDAQ Listing Rule 5635(c)(4), comprised of (i) an option to purchase 55,000 shares of Disc’s common stock, at an exercise price equal to the closing price of the Common Stock on the date of grant, and (ii) a restricted stock unit award for 36,666 shares of Common Stock. The Option Award shall vest 25% on the first anniversary of Ms. Stephenson’s start date, with the remainder vesting in 36 equal monthly installments thereafter. The RSU Award shall vest in equal installments on each of the first, second, third, and fourth anniversaries of the vesting date set by Disc’s company vesting policy. The Inducement Award was approved by Disc’s Board of Directors (the “Board”), including a majority of the independent directors serving on the Board. Tillkännagivande • Feb 21
Disc Medicine Inc Receives FDA Fast Track Designation for DISC-0974 for the Treatment of Anemia in Non-Dialysis Dependent Chronic Kidney Disease Disc Medicine, Inc. announced that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to DISC-0974 for the treatment of patients with non-dialysis dependent chronic kidney disease (NDD-CKD) and anemia. Fast Track is a process designed by the FDA to facilitate the development and expedite the review of investigational drugs intended to treat serious conditions and for which nonclinical or clinical data demonstrate the potential to address unmet medical need. A therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the candidate’s development plan. Therapeutic candidates with Fast Track designation may also be eligible for priority review and accelerated approval if supported by clinical data. Tillkännagivande • Feb 10
Disc Medicine, Inc. Receives FDA Orphan Drug Designation for DISC-3405 for the Treatment of Polycythemia Vera Disc Medicine, Inc. announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to DISC-3405 for the treatment of patients with polycythemia vera (PV). FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity. DISC-3405 is an investigational, anti-TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) monoclonal antibody designed to increase hepcidin production and suppress serum iron. Disc in-licensed DISC-3405 from Mabwell Therapeutics in January 2023. Preclinical studies of DISC-3405 have demonstrated an increase in hepcidin production and suppression of serum iron levels in animal models of beta-thalassemia and polycythemia vera. Disc initiated a Phase 1 study of DISC-3405 in healthy volunteers in October 2023 and plans to develop DISC-3405 initially as a treatment for polycythemia vera as well as other hematologic conditions. DISC-3405 are an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Polycythemia vera is a chronic and rare myeloproliferative neoplasm characterized by the abnormal proliferation of red blood cells. PV affects approximately 150,000 patients in the U.S. and has a similar prevalence in Europe. Recent Insider Transactions Derivative • Feb 09
Key Executive notifies of intention to sell stock Brian MacDonald intends to sell 19k shares in the next 90 days after lodging an Intent To Sell Form on the 6th of February. If the sale is conducted around the recent share price of US$67.15, it would amount to US$1.2m. Since June 2023, Brian has owned 15.56k shares directly. Company insiders have collectively sold US$3.3m more than they bought, via options and on-market transactions in the last 12 months. Recent Insider Transactions Derivative • Jan 04
Key Executive notifies of intention to sell stock Brian MacDonald intends to sell 5k shares in the next 90 days after lodging an Intent To Sell Form on the 2nd of January. If the sale is conducted around the recent share price of US$57.76, it would amount to US$289k. Since June 2023, Brian has owned 15.56k shares directly. Company insiders have collectively sold US$3.3m more than they bought, via options and on-market transactions in the last 12 months. Tillkännagivande • Dec 22
Disc Medicine, Inc Announces Retirement of Brian MacDonald as Chief Innovation Officer and Appointment as Chair of Scientific Advisory Board Disc Medicine, Inc. announced that, after a distinguished 28-year career, including over 6 years at Disc, Brian MacDonald, MB, ChB, PhD, will be retiring from his role as Chief Innovation Officer. Dr. MacDonald plans to stay involved with Disc, transitioning from his current role to become Chair of Disc’s Scientific Advisory Board. Recent Insider Transactions Derivative • Dec 14
Independent Director notifies of intention to sell stock William White intends to sell 34k shares in the next 90 days after lodging an Intent To Sell Form on the 12th of December. If the sale is conducted around the recent share price of US$55.34, it would amount to US$1.9m. Since March 2023, William has not owned shares directly (This sale likely refers to shares that have not yet been received). Company insiders have collectively sold US$1.2m more than they bought, via options and on-market transactions in the last 12 months. Tillkännagivande • Dec 12
Disc Presents Initial Positive Data from Ongoing Phase 1B/2 Trial of Disc-0974 in Patients with Myelofibrosis (Mf) and Anemia At the 65Th American Society of Hematology (Ash) Annual Meeting Disc Medicine, Inc. announced data from initial dose cohorts of its ongoing phase 1b/2 study of DISC-0974, a monoclonal antibody designed to suppress hepcidin by inhibiting the hemojuvelin (HJV) co-receptor, in MF patients with anemia. The initial data demonstrated that treatment with DISC-0974 substantially decreased serum hepcidin, increased serum iron and resulted in improvements in hemoglobin or reduced transfusion burden across a broad range of MF patients. The phase 1b/2a multi-center, open-label, ascending-dose study (NCT05320198) is enrolling patients with MF and severe anemia, including both transfusion and non-transfusion dependent patients. The trial also includes patients who may or may not be receiving concomitant janus kinase (JAK) inhibitor therapy. Study assessments include safety and tolerability of DISC-0974, as well as markers of iron regulation, such as hepcidin and iron, and hematologic parameters. In the phase 1b dose-escalation phase, DISC-0974 is administered subcutaneously every 4 weeks for up to 6 treatments. Dose escalation is ongoing and the data presented reflect 11 evaluable subjects from the initial three dose levels (14 mg, 28 mg and 50 mg) as of the October 20, 2023 data cutoff. Key initial data presented: DISC-0974 dosing resulted in meaningful, dose-dependent decreases in hepcidin across all treated patients These reductions in hepcidin corresponded to dose-dependent increases in serum iron, Patients dosed with 28 mg of DISC-0974 had a >75% reduction in serum hepcidin and >75% increase in serum iron, Four of seven (57%) evaluable non-transfusion-dependent (NTD) patients at the 28 mg and 50 mg dose levels had a >1.5 g/dL hemoglobin increase from baseline after starting DISC-0974, One of two transfusion-dependent (TD) patients achieved transfusion independence by the Gale Criteria, Hematologic activity was observed in MF patients, regardless of concomitant JAK inhibitor use, To date, DISC-0974 has been generally well-tolerated. The adverse events (AEs) seen in two or more subjects were fatigue, anemia, diarrhea, and nausea. The majority of AEs were deemed not related to DISC-0974. Recent Insider Transactions Derivative • Nov 24
General Counsel notifies of intention to sell stock Rahul Khara intends to sell 10k shares in the next 90 days after lodging an Intent To Sell Form on the 22nd of November. If the sale is conducted around the recent share price of US$51.75, it would amount to US$518k. As of today, Rahul currently holds no shares directly (This sale likely refers to shares that have not yet been received). Company insiders have collectively sold US$803k more than they bought, via options and on-market transactions in the last 12 months. Recent Insider Transactions Derivative • Nov 17
General Counsel exercised options and sold US$176k worth of stock On the 14th of November, Rahul Khara exercised 5k options at a strike price of around US$14.69 and sold these shares for an average price of US$50.61 per share. This trade did not impact their existing holding. As of today, Rahul currently holds no shares directly. Company insiders have collectively sold US$803k more than they bought, via options and on-market transactions in the last 12 months. Recent Insider Transactions Derivative • Nov 06
General Counsel notifies of intention to sell stock Rahul Khara intends to sell 5k shares in the next 90 days after lodging an Intent To Sell Form on the 3rd of November. If the sale is conducted around the recent share price of US$45.90, it would amount to US$230k. As of today, Rahul currently holds no shares directly (This sale likely refers to shares that have not yet been received). Company insiders have collectively sold US$623k more than they bought, via options and on-market transactions in the last 12 months. Tillkännagivande • Oct 25
Disc Medicine Announces Departure of Joanne Bryce as Chief Financial Officer Disc Medicine, Inc. announced that Joanne Bryce, Chief Financial Officer, plans to depart the company. Disc has initiated a search to fill the position. To ensure an orderly transition, Ms. Bryce plans to remain with the company and oversee her current responsibilities until a successor has been identified. Tillkännagivande • Oct 05
Disc Medicine, Inc. Initiates a Phase 1 Study of DISC-3405 (anti-TMPRSS6 mAb) in Healthy Volunteers Disc Medicine, Inc. announced the initiation of a Phase 1 study of DISC-3405 (formerly MWTX-003) in healthy volunteers. DISC-3405 is a monoclonal antibody designed to targetTMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) to increase hepcidin and decrease iron. The study will be a randomized, double-blind, placebo-controlled, single- and multiple-ascending dose Phase 1 trial in healthy volunteers and will evaluate safety, tolerability, pharmacokinetics, and measures of pharmacodynamic activity, including markers of iron homeostasis and erythropoiesis. Following completion of this study, Disc plans to initiate a trial in polycythemia vera, for which DISC-3405 has received Fast Track Designation. Recent Insider Transactions Derivative • Aug 03
Chief Financial Officer notifies of intention to sell stock Joanne Bryce intends to sell 6k shares in the next 90 days after lodging an Intent To Sell Form on the 1st of August. If the sale is conducted around the recent share price of US$49.60, it would amount to US$274k. Since December 2022, Joanne's direct individual holding has decreased from 3.45k shares to 1.26k. There has only been one transaction (US$384k sale) from insiders over the last 12 months. Tillkännagivande • Jul 28
Disc Medicine Announces First Patient Enrolled in Phase 1/2 Clinical Trial of Bitopertin in Diamond-Blackfan Anemia Disc Medicine Inc. announced that the first patient has been enrolled in the National Institutes of Health-sponsored Phase 1/2 clinical trial of bitopertin in Diamond-Blackfan anemia (DBA). Bitopertin is an investigational, orally administered glycine transporter 1 (GlyT1) inhibitor designed to modulate heme biosynthesis. The Phase 1/2 study will be a single-arm, dose-escalation trial of bitopertin in DBA patients who either have steroid-refractory and/or relapsed disease or are unable to tolerate systemic corticosteroids. The study includes planned dose escalation within each participant to continually assess for hematologic response. Upon completion of the main treatment period, patients may continue on extended treatment within the trial. This study will be conducted and funded by the NIH under a Cooperative Research and Development Agreement (CRADA), under the direction of Dr. Cynthia Dunbar, M.D., the NIH Distinguished Investigator and Chief Translational Stem Cell Biology Branch, and Head, Molecular Hematopoiesis Section, NHLBI, with Dr. David Young, M.D., Ph.D., NHLBI Staff Clinician as Principal Investigator. The content of this research is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. Recent Insider Transactions Derivative • Jul 02
Chief Financial Officer notifies of intention to sell stock Joanne Bryce intends to sell 10k shares in the next 90 days after lodging an Intent To Sell Form on the 28th of June. If the sale is conducted around the recent share price of US$51.99, it would amount to US$524k. Since December 2022, Joanne has owned 3.45k shares directly. There has only been one transaction (US$384k sale) from insiders over the last 12 months. New Risk • Jun 27
New minor risk - Share price stability The company's share price has been volatile over the past 3 months. It is more volatile than 75% of American stocks, typically moving 10% a week. This is considered a minor risk. Share price volatility indicates the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. It also increases the risk of potential losses in the short term as the stock tends to have larger drops in price more frequently than other stocks. Currently, the following risks have been identified for the company: Major Risks Earnings are forecast to decline by an average of 15% per year for the foreseeable future. Revenue is less than US$1m. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$135m net loss in 3 years). Share price has been volatile over the past 3 months (10% average weekly change). Tillkännagivande • Jun 10
Disc Medicine, Inc. Presents Positive Initial Data from Phase 2 BEACON Trial of Bitopertin in Patients with Erythropoietic Protoporphyria at European Hematology Association (EHA) 2023 Congress Disc Medicine Inc. presented preliminary findings from its ongoing, Phase 2 open-label BEACON trial evaluating bitopertin, an orally administered glycine transporter 1 (GlyT1) inhibitor, in patients with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) at the European Hematology Association (EHA) 2023 Congress in Frankfurt, Germany. The initial trial data demonstrated consistent decreases in PPIX, significant increases in reported sunlight tolerance and improvements in measures of patient quality of life. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. There are currently two ongoing Phase 2 clinical trials of bitopertin in patients with erythropiietic porphyria, including an open-label trial called BEACON and a randomized, double-blind placebo-controlled trial called AURORA. Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. There is currently no cure for EPP and only one FDA-approved therapy, a surgically implanted synthetic hormone designed to stimulate melanin production called Scenesse(R) (afamelanotide). Tillkännagivande • May 12
Disc Medicine Announces Presentation of Initial Data from Phase 2 BEACON Trial in Patients with Erythropoietic Protoporphyria at European Hematology Association 2023 Congress Disc Medicine Inc. announced upcoming clinical data to be presented at the European Hematology Association (EHA) 2023 Congress in Frankfurt, Germany. The presentation will report initial results from BEACON, the Phase 2, randomized, open-label, parallel arm trial of bitopertin in Erythropoietic Protoporphyria (EPP) that is ongoing in Australia. This study was designed to assess changes in levels of protoporphyrin IX (PPIX), the toxic metabolite that causes disease pathology in EPP, as well as measures of photosensitivity, quality of life, and safety and tolerability. Additionally, an abstract outlining the study design of AURORA, a Phase 2, randomized double-blind, placebo-controlled trial of bitopertin in EPP will be published in the EHA Abstract Book. Both abstracts are now available on the EHA conference website. Tillkännagivande • Feb 17
Disc Medicine Initiates Phase 1b/2 Clinical Study of DISC-0974 in Adults with Non-Dialysis Dependent Chronic Kidney Disease (NDD-CKD) and Anemia Disc Medicine announced the initiation of a Phase 1b/2 clinical study of DISC-0974 in non-dialysis dependent chronic kidney disease (NDD-CKD) patients with anemia. DISC-0974 is a monoclonal antibody designed to suppress hepcidin by inhibiting the hemojuvelin (HJV) co-receptor, and thereby address anemia by enhancing the availability of iron for erythropoiesis. DISC-0974 is also currently being studied in a Phase 1b/2 clinical study in patients with myelofibrosis and anemia. The study will be a multi-center, Phase 1b/2 trial and will evaluate the safety, tolerability, and efficacy of DISC-0974 in NDD-CKD patients with anemia. The study endpoints will include hepcidin levels, serum iron and markers of iron mobilization and changes in hemoglobin. The study enrollment criteria will include patients with Stage II-V CKD not receiving dialysis, baseline Hb < 11.0 g/dL for males and Hb < 10.5 g/dL for females, and those not receiving concurrent treatment with erythropoietin-stimulating agents (ESAs). The study will be conducted in two parts: Phase 1b (Dose-Escalation): Randomized, double-blind, placebo-controlled study design; single, ascending doses of DISC-0974 will be administered subcutaneously at the following planned dose levels: 28 mg, 40 mg, 60 mg, 90 mg; safety, PK and hematologic effects will be assessed at each dose level; a dose for the expansion phase will be selected based on optimal increases in hemoglobin; Phase 2 (Expansion Stage): Open-label, single-arm study design; multiple doses of DISC-0974 administered subcutaneously, once-monthly at the dose level selected from the phase 1b portion of the study for three months. DISC-0974 is an investigational monoclonal antibody (mAb) targeting a BMP-signaling co-receptor called hemojuvelin (HJV) and is designed to suppress hepcidin production and increase serum iron levels in patients suffering from anemia of inflammation. DISC-0974 was in-licensed by Disc from AbbVie in 2019. Anemia of inflammation arises from abnormally elevated hepcidin and is the most common form of anemia, affecting millions of patients in the US across numerous diseases such as chronic kidney disease, myelofibrosis, cancer, autoimmune diseases, and other conditions with an inflammatory component. Disc has established clinical proof-of-mechanism of DISC-0974 in a Phase 1 trial of healthy volunteers and initiated a Phase 1b/2a clinical trial of DISC-0974 in patients with myelofibrosis and anemia, as well as a Phase 1b/2a clinical trial of DISC-0974 in patients with chronic kidney disease and anemia who are not receiving dialysis. DISC-0974 is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Board Change • Jan 03
No independent directors Following the recent departure of a director, there are no independent directors on the board. The company's board is composed of: No independent directors. 9 non-independent directors. Director Bill White was the last director to join the board, commencing their role in 2021. The company's lack of independent directors is a risk according to the Simply Wall St Risk Model.