Protoporphyria, Erythropoietic

MALIBU, Calif., March 04, 2024 (GLOBE NEWSWIRE) -- Leading biotech company CLINUVEL last week joined an intimate event bringing together California’s most prolific investors and philanthropists at the Malibu home of Lady Gaga and Michael Polansky. The event, hosted by Lady Gaga and Mr. Polansky, and Sean Parker and Alexandra Parker, shone a light on the groundbreaking work of CLINUVEL, a global biopharmaceutical firm whose cutting-edge melanocortin therapies transform the lives of patients at the highest risk of photodamage and skin cancer. Mr. Parker and Mr. Polansky have supported CLINUVEL for more than 15 years, finding alignment between the Company’s pioneering approach to medicine and the boundary-pushing work of the Parker Institute for Cancer Immunotherapy which works to revolutionise healthcare. Over a family-style dinner by chef Evan Funke of Los Angeles hotspot Felix Trattoria, and a performance by Grammy-winning Attacca Quartet, the hosts shared how CLINUVEL’s treatments herald a new era for the treatment of light-related conditions. The Group’s CEO Dr. Philippe Wolgen also unveiled what is next for the most exciting biopharmaceutical firm in the field of photomedicine today. The future of photomedicine For 25 years, CLINUVEL introduced a proof of concept by treating groups of patients whose needs had been overlooked by the pharmaceutical industry. These include the genetic disorders erythropoietic protoporphyria (EPP), xeroderma pigmentosum (XP) and the skin disorder vitiligo, when people lose their pigmentation. In 2014 CLINUVEL developed the only approved treatment in the world for EPP, accelerating CLINUVEL’s evolution into a profitable, self-sustaining and dynamic company. Now the Company is exploring the vast potential of its technology: how can CLINUVEL’s unique knowledge of melanocortins be scaled up to treat widespread health conditions that affect the skin, brain and nervous system? Beyond the world of pharmaceuticals, CLINUVEL’s technology has the power to disrupt the skincare market. After 43 years of photomedicine research, the Company is launching PhotoCosmetics, a range of advanced skincare products based on a group of peptides known as melanocortins. Revolutionising healthcare for those in needPhilanthropy is central to the work of Lady Gaga and Michael Polansky, Sean Parker and Alexandra Parker. CLINUVEL shares their passion for creating long-lasting change and radically improving patients’ lives. At the event in Malibu, CLINUVEL announced the Photomedicine Foundation, which will count Mr Polansky as a trustee. The initiative will help those most affected by debilitating conditions related to the sun. Through the Foundation, CLINUVEL is taking its world-leading expertise directly to communities who cannot afford care or treatment. The foundation’s mission is to support vulnerable patients at extreme risk of photodamage and skin cancer in Africa, Middle East and Latin America. Michael Polansky said: “It’s critical that investors strive to find companies whose missions extend beyond profitability, by supporting the development of technologies that can drive meaningful and sustained improvement to human life. CLINUVEL is an example of a company that aims to improve human health and I am delighted to become a trustee of the Photomedicine Foundation, which will play a vital role in improving the lives of individuals with light-related conditions who cannot access life-saving medical care.” Sean Parker said: “CLINUVEL is one of the most innovative Life Sciences companies that exists today with a management that has the patience to see through long development. Both its groundbreaking melanocortin technology and long-term business plan will disrupt the world of biopharmaceuticals and luxury skincare. The Photomedicine Foundation is a welcome addition to a company on a path to growth unlike any other.” CLINUVEL’s CEO, Dr. Philippe Wolgen said: “Sean Parker and Michael Polansky are unafraid to rip up the rulebook in their mission to advance science and improve global health. CLINUVEL is grateful to have their support as we translate our pharmaceutical technology into PhotoCosmetics and give back to underserved communities. A bright future looms for CLINUVEL.” About The CLINUVEL Group: CLINUVEL is a public biopharmaceutical company whose pioneering melanocortin technology has revolutionised the field of photomedicine. Over the last 25 years, CLINUVEL’s work has furthered scientific knowledge, delivered breakthroughs in medical technology and transformed patients’ lives. The Company has developed and commercialised SCENESSE® (afamelanotide), the only approved treatment in the world for the prevention of phototoxicity in adult patients with EPP. The treatment was approved for use by the US FDA in 2019 and is also approved for commercial distribution in Europe, Israel and Australia. CLINUVEL is conducting clinical trials to assess the efficacy and safety of its melanocortin technology in treating severe disorders including xeroderma pigmentosum (XP), vitiligo and arterial ischemic stroke. In 2023 it announced its expansion into skincare with the introduction of its first PhotoCosmetic product CYACÊLLE, a polychromatic solar screen. For more information visit www.clinuvel.comInstagram:@clinuvel_pharmaceuticals @clinuvelDNATwitter:@ClinuvelNewsFacebook:@Clinuvel

Updated data from BEACON continued to demonstrate significant, consistent reductions in protoporphyrin IX (PPIX) > 40% and improvements in sunlight toleranceRobust and consistent improvements across all measures of sunlight tolerance, including >3x improvement over historical control of precedented pivotal endpointBitopertin was generally well-tolerated with stable hemoglobin at both dose levelsEarlier today, Disc also announced initial positive data from the phase 1b study of DISC-0974 in myelofibrosis patients with anemia, demonstrating improvements in hemoglobin and reductions in transfusion burden WATERTOWN, Mass., Dec. 11, 2023 (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 presented updated results from the phase 2 open-label BEACON study of bitopertin, an orally administered glycine transporter 1 (GlyT1) inhibitor, in patients with erythropoietic protoporphyria (EPP) as an oral presentation at the 65th ASH Annual Meeting. The updated data are consistent with and confirm the initial positive results presented in June, demonstrating significant decreases in PPIX, robust and consistent improvements in sunlight tolerance across all study measures, including the precedented pivotal endpoint, and improvements in patient quality of life. “This has been a tremendous ASH meeting for Disc, as we presented data across our two most advanced programs. We are especially proud and excited to present the updated results from BEACON, which reflect a more robust open label data set and clearly indicate that reducing PPIX with bitopertin has the potential to result in dramatic benefits for patients with EPP. Importantly, this improvement was observed across every efficacy measure of the study, including our analysis of the precedented pivotal endpoint, cumulative time in light over 6 months, which we debuted at this meeting,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer. “With these results and the positive initial efficacy data from the DISC-0974 myelofibrosis study that we presented earlier today, Disc is preparing to enter its next stage of growth. We look forward to next year as we advance our full portfolio and obtain the readouts from AURORA and other studies.” The BEACON study (ACTRN12622000799752) is a randomized, open-label, parallel-arm study that enrolled 22 adult subjects with EPP or X-linked protoporphyria (XLP) in Australia, and has been expanded to include adolescents. This trial was designed to assess changes in levels of PPIX, as well as measures of photosensitivity, quality of life, and safety and tolerability. Subjects are randomized to receive either 20 mg or 60 mg of bitopertin once-daily for 24 weeks, after which patients have the option of continuing in an open-label extension of the trial for up to an additional 24 weeks. The updated data presented reflects results from all 22 adults, with a data cutoff of September 18, 2023 for PPIX data and October 20, 2023 for all other endpoints. The data are consistent with and confirm the initial positive results presented in June 2023. Protoporphyrin IX (PPIX) levels: Significant, dose-dependent, and sustained reductions in whole blood PPIX levels; mean reduction > 40% (p<0.001 versus baseline)Demonstrated substantial and consistent improvements in sunlight tolerance across all study measuresHighlights of the data presented: Average time to prodrome: Greater than 3x improvement vs. baseline (p<0.001)Increased proportion of days without symptoms: 78% vs. 33% (baseline)Increased proportion of sunlight challenges without prodromes: 54% vs. 7% (baseline)Phototoxic reactions: 92% reduction in patient-reported reactions while on treatment compared to baselineNearly all participants reported improvements in multiple quality-of-life measures at the end of study Mean cumulative total time in light on days without pain observed over the 6-month treatment period (precedented pivotal endpoint): 222.6 + 129.3 hours Bitopertin-treated participants had a >3x increase relative to historical control Bitopertin was generally well tolerated at both dose levels with no serious adverse events, stable mean hemoglobin levels, and no anemia adverse events (AEs) reported. The most common AEs were dizziness, lightheadedness, headache, and nausea. Earlier today, Disc also presented initial positive data from the ongoing phase 1b study of DISC-0974 in myelofibrosis (MF) patients with anemia. The data were presented as a poster during the ASH meeting and demonstrated suppression of hepcidin, increased iron levels and improvements in hematologic parameters, including increased hemoglobin and reduction in transfusion burden. The presentation was announced in a separate press release issued earlier today and will be reviewed again during the management call, as well as initial data from the first dose-escalation cohort of the ongoing phase 1b/2 study in non-dialysis dependent chronic kidney disease (NDD-CKD) patients with anemia. Bitopertin and DISC-0974 are investigational agents and are not approved for use as a therapy in any jurisdiction worldwide. Webcast Conference Call InformationManagement will host a call on Monday, December 11th at 9:30 pm ET / 6:30 pm PT to review data and operational plans. Please register for management’s webcast on the Events and Presentations page of Disc’s website (https://ir.discmedicine.com/). About Disc MedicineDisc 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, potentially 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. Disc Medicine Cautionary Statement Regarding Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, express or implied statements regarding Disc’s expectations, hopes, beliefs, intentions or strategies with respect to its AURORA Phase 2 and BEACON Phase 2 clinical studies of bitopertin and the results thereof, its Phase 1b/2 clinical studies of DISC-0974 in patients with MF and NDD-CKD patients with anemia, its Phase 1 clinical study of DISC-3405 in healthy volunteers, projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data and other clinical activities, Disc’s business plans and objectives, Disc’s analysis of market potential for patients with EPP, and Disc’s beliefs about operating expenses and that it will have capital to fund Disc well into 2026. The use of words such as, but not limited to, “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “future,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “seek,” “suggest,” “will,” or “would” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; the other risks and uncertainties described in the “Risk Factors” section of our Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, and other documents filed by Disc from time to time with the Securities and Exchange Commission (SEC), as well as discussions of potential risks, uncertainties, and other important factors in Disc’s subsequent filings with the SEC. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. Media Contact Peg RusconiVerge Scientific Communicationsprusconi@vergescientific.com Investor Relations Contact Christina TartagliaStern Investor Relationschristina.tartaglia@sternir.com

Substantial, dose-dependent reductions in serum hepcidin and increases in serum iron Hematologic response demonstrated by increased hemoglobin levels and reduction in transfusion burdenDISC-0974 was generally well-tolerated at all evaluated doses WATERTOWN, Mass., Dec. 11, 2023 (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 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. “We are thrilled to see this level of hematologic activity so early during dose escalation and in a range of patient types. Elevated hepcidin is an important driver of anemia in patients with MF and the initial data suggest that DISC-0974 has leading activity in suppressing hepcidin,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “This is the second program in the last six months where Disc has shown proof-of-concept data in patients, and we look forward to advancing DISC-0974 deeper into development and presenting updated data from both this MF and the ongoing CKD anemia study next year.” 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 patientsThese 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-0974One of two transfusion-dependent (TD) patients achieved transfusion independence by the Gale CriteriaHematologic activity was observed in MF patients, regardless of concomitant JAK inhibitor useTo 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. These data were presented at the 65th American Society of Hematology (ASH) Annual Meeting in San Diego, California and the poster is available on the ASH platform. Later today, the company will be presenting updated results from the phase 2 BEACON study of bitopertin in EPP as an oral presentation, December 11th at 5:30 pm PT / 8:30 pm ET. Management will host a call to review the presented data on Monday, December 11th at 6:30 pm PT / 9:30 pm ET. Please register for the event on the Events and Presentations page of Disc’s website (https://ir.discmedicine.com/). About 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 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. About Anemia of MyelofibrosisMyelofibrosis (MF) is a rare, chronic blood cancer that currently affects an estimated 16,000 to 18,500 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. About Disc MedicineDisc 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, potentially 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. Disc Medicine Cautionary Statement Regarding Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to its Phase 1b/2 clinical studies of DISC-0974 in patients with MF and NDD-CKD patients with anemia, projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; and Disc’s business plans and objectives. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the nature, strategy and focus of Disc; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of the availability of data from Disc’s clinical trials; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; the other risks and uncertainties described in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2022, Quarterly Reports on Form 10-Q for the quarters ended March 31, 2023, June 30, 2023, and September 30, 2023, and other documents filed by Disc from time to time with the SEC, as well as discussions of potential risks, uncertainties, and other important factors in Disc’s subsequent filings with the SEC. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. Media Contact Peg RusconiVerge Scientific Communicationsprusconi@vergescientific.com Investor Relations Contact Christina TartagliaStern Investor Relationschristina.tartaglia@sternir.com