Disc Medicine Opco, Inc.

WATERTOWN, Mass., Dec. 29, 2022 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (“Disc”), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, announced today that its previously-announced merger with Gemini Therapeutics, Inc. (“Gemini”) closed on December 29, 2022, following the approval of Gemini shareholders. The combined company, focused on advancing Disc’s pipeline of hematology programs, will operate under the name Disc Medicine, Inc. and its shares will commence trading on a 1-10 reverse split adjusted basis effective with the open of business on December 30, 2022 on the Nasdaq Global Market under the ticker symbol IRON. Concurrent with the closing of the merger, Disc completed a financing of $53.5 million from a syndicate of healthcare investors led by Access Biotechnology and including OrbiMed, Atlas Venture, 5AM Ventures, Novo Holdings A/S, Arix Bioscience, Rock Springs Capital, and Janus Henderson Investors. The projected cash and cash equivalents as of the close of the business combination are expected to be approximately $175 million, providing operating runway into 2025. “The completion of this merger and concurrent financing marks a transformative moment in Disc’s growth and ensures we are well-positioned to advance our portfolio of innovative, potentially first-in-class therapeutic candidates through key development milestones,” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. “We’re excited to enter the new year with multiple programs already in the clinic, a robust development pipeline and the financial strength from this merger. We look forward to maintaining this momentum and reporting on interim data read-outs from several patient studies in 2023.” The combined company will be led by John Quisel, J.D., Ph.D., the current CEO and president of Disc, and other members of the Disc management team. Disc will focus on advancing its development pipeline of investigational product candidates for hematologic diseases, including: The ongoing phase 2 BEACON and AURORA clinical trials of bitopertin, an investigational, orally administered inhibitor of glycine transporter 1 (GlyT1) that modulates heme biosynthesis, in patients with erythropoietic protoporphyria (EPP) The ongoing phase 1b/2 clinical trial of DISC-0974, a monoclonal antibody designed to suppress hepcidin by inhibiting the hemojuvelin (HJV) co-receptor, in myelofibrosis patients with anemia A planned phase 1b/2 clinical trial of DISC-0974 in patients with anemia of chronic kidney disease (CKD) who are non-dialysis dependent Preclinical studies of bitopertin and DISC-0974 to additional indications of interest and advancing several preclinical-stage programs in development designed to address hematologic diseases As part of the closing of the merger, Gemini effected a 1 for 10 reverse split of its common stock. Following the reverse stock split and closing of the merger, there will be approximately 17 million shares of the combined company’s common stock outstanding with prior Disc shareholders owning approximately 74% and prior Gemini shareholders owning 26%. The Board of Directors of the combined company will be composed of nine members, including eight Disc board members and one board member from Gemini. SVB Securities served as the exclusive financial advisor to Gemini and Wilmer Cutler Pickering Hale and Dorr LLP served as Gemini’s legal counsel. Morgan Stanley served as the lead financial advisor to Disc along with Wedbush PacGrow, and Goodwin Procter LLP served as Disc’s legal counsel. About Disc Disc Medicine is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit www.discmedicine.com.

WATERTOWN, Mass., Dec. 27, 2022 /PRNewswire/ -- Disc Medicine, Inc. ("Disc"), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, announced today that the U.S. Food and Drug Administration ("FDA") granted Orphan Drug Designation to bitopertin for the treatment of erythropoietic protoporphyria ("EPP"). Bitopertin is an investigational oral, selective inhibitor of glycine transporter 1 ("GlyT1") designed to modulate heme biosynthesis, and has been shown in preclinical studies to reduce accumulation of protoporphyrin IX ("PPIX"), the toxic metabolite that causes disease pathology in EPP patients. It is currently being studied in two ongoing Phase 2 studies in EPP, AURORA (NCT05308472) and BEACON (ACTRN12622000799752). "Receiving orphan drug designation for bitopertin is incredibly encouraging and validates our commitment to bring a potential new treatment to EPP patients," said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. "We are eagerly awaiting the results of our ongoing Phase 2 trials and look forward to collaborating with the FDA to progress bitopertin through clinical development." FDA Orphan Drug Designation may be granted to investigational drugs or biological products which show promise in treating rare medical diseases or conditions that affect fewer than 200,000 people in the United States. By receiving Orphan Drug Designation, bitopertin can benefit from certain development incentives and seven years of market exclusivity, subject to regulatory approval. About EPP EPP is a rare, debilitating and potentially life-threatening diseases caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate, PPIX. This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensations and potential blistering and disfigurement. PPIX 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). About Bitopertin Bitopertin is a clinical-stage, orally administered inhibitor of 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. The safety profile and effects of bitopertin on heme biosynthesis were previously established in a comprehensive clinical program comprising over 4,000 individuals across multiple clinical studies. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases beginning with EPP and X-linked protoporphyria ("XLP"). In preclinical models of EPP and XLP, bitopertin was shown to significantly decrease PPIX, a toxic intermediate of heme biosynthesis which is the underlying cause of the disease. Bitopertin is an experimental 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. About Disc Medicine, Inc. Disc is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders. We are building a portfolio of innovative, potential first-in-class therapeutic candidates that affect fundamental pathways of red blood cell biology. We are committed to developing treatments that empower and bring hope to the many patients who suffer from hematologic diseases. In August 2022, Disc announced it entered into a definitive merger agreement with Gemini Therapeutics, Inc. (NASDAQ:GMTX) ("Gemini"). For more information, please visit www.discmedicine.com.

WATERTOWN, Mass., Aug. 10, 2022 /PRNewswire/ -- Disc Medicine, a clinical-stage biotechnology company dedicated to the discovery and development of novel therapeutic candidates for the treatment of serious and debilitating hematologic diseases, announced today the initiation of BEACON, a phase 2 clinical study of bitopertin in patients with Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP). Bitopertin is an oral, selective inhibitor of glycine transporter 1 (GlyT1) designed to modulate heme biosynthesis, and has been shown in preclinical studies to reduce accumulation of protoporphyrin IX (PPIX), the toxic metabolite that causes disease pathology in EPP and XLP patients. "The initiation of the BEACON phase 2 study is an important milestone as it marks the first time bitopertin will be evaluated as a potential, disease-modifying therapy in patients with porphyria. It builds upon our previous studies in animal models of EPP and XLP, which demonstrated that bitopertin can significantly reduce protoporphyrin IX," said John Quisel, JD, PhD, Chief Executive Officer at Disc Medicine. "This is the second development program that Disc has successfully advanced into patient studies in the past few months. I want to thank our entire team for the tremendous effort that has brought us to this point and for their tireless dedication to patients." The BEACON Phase 2 study is a randomized, open-label, multiple dose clinical trial designed to evaluate the safety, tolerability, and efficacy of bitopertin in patients with EPP or XLP. It is designed to enroll approximately 20 patients at sites in Australia. The study will primarily assess changes in levels of PPIX as well as the pharmacokinetic profile, safety and tolerability of bitopertin in EPP or XLP patients. It will also include measures of photosensitivity, daylight tolerance, pain and exploratory biomarkers of hepatobiliary disease. Patients will receive orally-administered bitopertin for 24 weeks at doses of either 20 mg once-daily or 60 mg once-daily. Upon completion of the 24-week treatment period, patients may continue on bitopertin for an additional 24 weeks. "EPP and XLP are severe, debilitating and rare diseases that impact multiple dimensions of patients' lives, spanning severe and painful phototoxic reactions, complications of hepatobiliary disease, and a major toll on psychosocial development and quality of life. There is an immense need for novel therapies, particularly ones that address the underlying pathophysiology," said Robert Desnick, MD, Ph.D, Inaugural Chair Emeritus of the Department of Genetics and Genomics at Mount Sinai. "We are excited by the initiation of this clinical trial of bitopertin and its potential effects on PPIX, the molecular driver of these porphyrias and a major determinant of disease severity." About Bitopertin Bitopertin is a 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. The safety profile and effects of bitopertin on heme biosynthesis were previously established in a comprehensive clinical program comprising over 4,000 individuals across multiple clinical studies. Disc Medicine is planning to develop bitopertin as a potential treatment for a range of hematologic diseases beginning with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). In preclinical models of EPP and XLP, bitopertin was shown to significantly decrease PPIX, a toxic intermediate of heme biosynthesis and which is the underlying cause of the disease. Bitopertin is an experimental 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. About Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP) Erythropoietic protoporphyria (EPP) and X-linked Protoporphyria (XLP) are rare, debilitating and potentially life-threatening diseases 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. PPIX 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). About Disc Medicine Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders. We are building a portfolio of innovative, first-in-class therapeutic candidates that affect fundamental pathways of red blood cell biology. We are committed to developing treatments that empower and bring hope to the many patients who suffer from hematologic diseases. For more information, please visit www.discmedicine.com.