Afamelanotide Acetate

WATERTOWN, Mass., July 21, 2025 (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 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. “At the pre-NDA meeting, we confirmed alignment with the agency on the expected timing and content of our NDA submission and, as a result, we are on track for a submission in October of this year,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “We appreciate the timely and constructive feedback from the agency as we work to bring bitopertin to the EPP community and prepare our organization for its first potential product launch.” 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 in EPP in October 2025 under the FDA’s accelerated approval pathway based on Disc’s existing data package. About Bitopertin 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 AURORA trial, and an open-label extension HELIOS 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. About Erythropoietic Protoporphyria (EPP) Erythropoietic protoporphyria (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 (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 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 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 the registrational pathway for bitopertin, including the timing of a potential NDA submission. 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 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; and the other risks and uncertainties described in Disc’s filings with the Securities and Exchange Commission, including in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2024, and in subsequent Quarterly Reports on Form 10-Q. 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 Rusconi Deerfield Group peg.rusconi@deerfieldgroup.com Investor Relations Contact Christina Tartaglia Precision AQ christina.tartaglia@precisionaq.com

First results of SCENESSE® study expected in 2H 2026Executive summary Recruitment target of 200 vitiligo patients (Fitzpatrick skin type III-VI) in CUV105 study achievedRandomised trial, 20-week treatment protocol plus 6-month follow upStudy sites in North America, Africa and EuropeFirst clinical observations with afamelanotide positive MELBOURNE, Australia, May 07, 2025 (GLOBE NEWSWIRE) -- CLINUVEL has met its recruitment target in its phase III trial (CUV105) of SCENESSE® (afamelanotide 16mg) in vitiligo, with more than 200 patients enrolled. The last patient to enter the study is scheduled to complete screening in May 2025. First results from the study are expected in the second half of 2026. CUV105: first SCENESSE® Phase III vitiligo trial SCENESSE® is being evaluated as a systemic repigmentation therapy for vitiligo patients, with a clinical focus on adolescent (12 years and above) and adult patients with darker skin types (Fitzpatrick III-VI).1 CUV105 is a randomised multi-centre Phase III trial being conducted in 37 study sites across three continents. The majority of patients (57%) have been enrolled in the United States. Patients receive treatment with either SCENESSE® every three weeks alongside adjuvant narrowband ultraviolet B (NB-UVB) phototherapy administered twice weekly, or NB-UVB monotherapy twice weekly, for 20 weeks. All patients are then followed up for six months. The vitiligo area scoring index (VASI) is being used to evaluate the primary endpoint, whereby the objective is to achieve a minimum of 50% repigmentation across the total body surface area (T-VASI50). Secondary endpoints include evaluations of repigmentation of face, neck and head (F-VASI) at week 20 and the maintenance of repigmentation following treatment completion. Patient Reported Outcomes deploy validated tools to assess patient perception of change (Patient Global Impression of Change in the face and the body and the Vitiligo Noticeability Scale) and quality of life (VitiQoL). More than 200 patients have enrolled in CUV105 and commenced the treatment protocol. Patients assigned to NB-UVB monotherapy are additionally eligible to receive SCENESSE® and adjuvant NB-UVB after completion of the follow-up period. CLINUVEL altered the CUV105 protocol at the end of last year to incorporate this extension period after requests from study sites and patients. CLINICAL OBSERVATIONS Clinical observations from CUV105 have been presented to global medical congresses, including the 2024 and 2025 American Academy of Dermatology Meetings, with further abstracts submitted for conferences later in 2025. Four previously released case studies – patients with skin type IV and varying disease duration – demonstrated repigmentation of vitiliginous lesions on the face or back after four weeks of commencing treatment with afamelanotide, and that some patients experience additional spontaneous repigmentation following the conclusion of the treatment protocol. A fifth case study shared today, demonstrates repigmentation of lesions on the arms and legs in a skin type V patient with a 20-year history of the vitiligo. All case study patients have reported satisfaction with the treatment results and that afamelanotide was well tolerated with adjunct NB-UVB. Above: the fifth case study to be released from CUV105, a 46-year-old male with skin type V treated with seven SCENESSE® implants and 40 NB-UVB sessions. The images demonstrate repigmentation of vitiliginous lesions on the patient’s right forearm (top) and lower legs (bottom). The patient was first diagnosed with vitiligo in 2004. The red outlines demonstrate the extent of the initially affected skin; the images are otherwise unaltered. COMMENTARY “We are thrilled with this enrolment milestone since we are essentially establishing a North American distribution network among dermatologists, anticipating the necessary infrastructure ahead of market entry of our breakthrough product,” CLINUVEL’s Director, Global Clinical Affairs, Dr Emilie Rodenburger said. “The first clinical observations of the systemic (total body) solution are encouraging, and now we will continue a regulatory discussion in Europe, Africa and North America, all in anticipation of CUV107, a second large trial evaluating the treatment effects of SCENESSE® in vitiligo.” ABOUT VITILIGO Vitiligo is an acquired depigmentation disorder affecting 1-2% of the global population. The disorder causes progressive loss of functional epidermal melanocytes (melanin producing skin cells), which leads to disfiguration and psychosocial distress. While the disease can have an impact on all patients, it is recognised that those with darker skin types are most severely affected. In North America, an estimated 820,000 individuals of darker skin types (IV-VI) are affected by vitiligo. The precise cause of the sudden start of this disorder remains unknown. There is only one pharmaceutical product, a topical immunosuppressant, approved for vitiligo in the United States and Europe. Vitiligo patients with less than 10% body surface area affected, who have previously failed transdermal formulations, are eligible for this treatment. With afamelanotide, a systemic non-immune suppressive solution would be offered to patients with darker skin complexions. Notes The first four case reports from the CUV105 clinical trial were released to the ASX on 20 January 2025 and can be accessed here. CLINUVEL thanks the patients and treating physicians for being able to share these case reports. 1 The Fitzpatrick Skin Type is a numerical classification of human skin colour, from type I skin that always burns, to type VI, dark skin that never burns. SCENESSE® for vitiligo patients: peer-reviewed research and conference presentations Elbuluk, N. (2024). What’s New in Vitiligo. 2024 Meeting of the American Academy of Dermatology. 11 March. San Diego, USA. Grimes, P. E., et al, (2013). The Efficacy of Afamelanotide and Narrowband UV-B Phototherapy for Repigmentation of Vitiligo. JAMA Dermatol, 149(1), 68–73. Grimes, P.E. (2024). What’s new and hot in pigmentary disorders. 2024 Meeting of the American Academy of Dermatology. 9 March. San Diego, USA. Kamangar, F. (2025) Afamelanotide: A Novel Promising Treatment for Vitiligo – Case Studies from the Randomized CUV105 Clinical Trial. Global Vitiligo Foundation Annual Scientific Symposium. 6 March. Orlando, USA. Lim, H. W., et al., (2015). Afamelanotide and Narrowband UV-B Phototherapy for the Treatment of Vitiligo: A Randomized Multicenter Trial. JAMA Dermatol, 151(1), 42. Lim, H.W. (2024). Clarence S. Livingood, MD Memorial Award and Lectureship: Photodermatology: Past, Present and Future. 2024 Meeting of the American Academy of Dermatology. 10 March. San Diego, USA. Toh, J. J. H., et al, (2020). Afamelanotide implants and narrow-band ultraviolet B phototherapy for the treatment of nonsegmental vitiligo in Asians. J Am Acad Dermatol, 82(6), 1517–1519. About CLINUVEL PHARMACEUTICALS LIMITED CLINUVEL (ASX: CUV; ADR LEVEL 1: CLVLY; Börse Frankfurt: UR9) is a global specialty pharmaceutical group focused on developing and commercialising treatments for patients with genetic, metabolic, systemic, and life-threatening, acute disorders, as well as healthcare solutions for specialised populations. As pioneers in photomedicine and the family of melanocortin peptides, CLINUVEL’s research and development has led to innovative treatments for patient populations with a clinical need for systemic photoprotection, assisted DNA repair, repigmentation and acute or life-threatening conditions who lack alternatives. CLINUVEL’s lead therapy, SCENESSE® (afamelanotide 16mg), is approved for commercial distribution in Europe, the USA, Israel, and Australia as the world’s first systemic photoprotective drug for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with erythropoietic protoporphyria (EPP). Headquartered in Melbourne, Australia, CLINUVEL has operations in Europe, Singapore, and the USA. For more information, please go to https://www.clinuvel.com. Authorised for ASX release by the Board of Directors of CLINUVEL PHARMACEUTICALS LTD. Head of Investor Relations Mr Malcolm Bull, CLINUVEL PHARMACEUTICALS LTD Investor Enquiries https://www.clinuvel.com/investors/contact-us Forward-Looking Statements This release contains forward-looking statements, which reflect the current beliefs and expectations of CLINUVEL’s management. Statements may involve a number of known and unknown risks that could cause our future results, performance, or achievements to differ significantly from those expressed or implied by such forward-looking statements. Important factors that could cause or contribute to such differences include risks relating to: our ability to develop and commercialise pharmaceutical products; the COVID-19 pandemic and/or other world, regional or national events affecting the supply chain for a protracted period of time, including our ability to develop, manufacture, market and sell biopharmaceutical and PhotoCosmetic products; competition for our products, especially SCENESSE® (afamelanotide 16mg), CYACÊLLE, PRÉNUMBRA®, NEURACTHEL® or products developed and characterised by us as PhotoCosmetics; our ability to achieve expected safety and efficacy results in a timely manner through our innovative R&D efforts; the effectiveness of our patents and other protections for innovative products, particularly in view of national and regional variations in patent laws; our potential exposure to product liability claims to the extent not covered by insurance; increased government scrutiny in either Australia, the U.S., Europe, the UK, Israel, China, Japan, and/or LATAM regions of our agreements with third parties and suppliers; our exposure to currency fluctuations and restrictions as well as credit risks; the effects of reforms in healthcare regulation and pharmaceutical pricing and reimbursement; that the Company may incur unexpected delays in the outsourced manufacturing of SCENESSE®, CYACÊLLE, PRÉNUMBRA®, NEURACTHEL® or products developed as PhotoCosmetics which may lead to the Company being unable to launch, supply or serve its commercial markets, special access programs and/or clinical trial programs; any failures to comply with any government payment system (i.e. Medicare, Medicaid, and U.S. Department of Veteran’s Affairs) reporting and payment obligations; uncertainties surrounding the legislative and regulatory pathways for the registration and approval of biotechnology, cosmetic and consumer based products; decisions by regulatory authorities regarding approval of our products as well as their decisions regarding label claims; our ability to retain or attract key personnel and managerial talent; the impact of broader change within the pharmaceutical industry, cosmetic industry and related industries; potential changes to tax liabilities or legislation; environmental risks; and other factors that have been discussed in our 2024 Annual Report. Forward-looking statements speak only as of the date on which they are made, and the Company undertakes no obligation, outside of those required under applicable laws or relevant listing rules of the Australian Securities Exchange, to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise. More information on preliminary and uncertain forecasts and estimates is available on request, whereby it is stated that past performance is not an indicator of future performance. Contact: Tel: +61 3 9660 4900 Fax: +61 3 9660 4909Email: mail@clinuvel.comAustralia (Head Office), Level 22, 535 Bourke Street, Melbourne, Victoria, 3000, Australia A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/db406f89-4bf4-4767-849c-269ffea46d9a