Erythropoietic protoporphyria

Disc Medicine Announces Presentation of Initial Data from Phase 2 BEACON Trial in Patients with Erythropoietic Protoporphyria (EPP) at European Hematology Association (EHA) 2023 Congress

Retrieved on: 
Thursday, May 11, 2023
PPIX, Abstract, European Hematology Association, EPP, AURORA, Quality of life, Congress, IRON, Patient, Randomness, EHA, Bitopertin, Photosensitivity, Safety, Pharmaceutical industry, Erythropoietic protoporphyria

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.

Key Points: 
  • 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.
  • 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.
  • Management will host a call to review the presented data on Friday, June 9th at 7:30 am ET.
  • Title: Initial Data from the BEACON Trial: A Phase 2, Randomized, Open‐label Trial of Bitopertin in Erythropoietic Protoporphyria
    Title: Study Design of the AURORA Trial: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial of Bitopertin in Erythropoietic Protoporphyria

Disc Medicine to Host Disease Webinar with KOLs on Erythropoietic Protoporphyria (EPP)

Retrieved on: 
Thursday, April 13, 2023
San Francisco, KOL, University, EPP, University of California, San Francisco, AURORA, Research, IRON, Patient, Erythropoietic protoporphyria, Porphyria, Paris Diderot University, Epidemiology, Pharmaceutical industry, Gastroenterology

The event will also include an overview of Disc’s development plans of bitopertin for erythropoietic protoporphyria.

Key Points: 
  • The event will also include an overview of Disc’s development plans of bitopertin for erythropoietic protoporphyria.
  • Intended for investors and other interested audiences, the virtual event will feature presentations from leading porphyria experts, who will provide a disease overview and discuss epidemiology, pathophysiology, disease burden, and unmet need associated with EPP.
  • A live webcast of the event will be available in the Events and Presentations section of the Investor page of Disc’s website (https://ir.discmedicine.com/).
  • Please register for the event on the Events and Presentations page of Disc’s website or by clicking this link .

Disc Medicine Reports Full Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Friday, March 31, 2023
Diamond, Janus, Sodium- and chloride-dependent glycine transporter 1, OrbiMed, Diamond–Blackfan anemia, Bitopertin, MF, Dialysis, Congress, Development, Patient, Mab, TMPRSS6, EPP, Porphyria, Thalassemia, Growth, FDA, Polycythemia, IND, NIH, Chronic kidney disease, AURORA, IRON, Erythropoiesis, Glycine, Administration, Erythropoietic protoporphyria, Anemia, XLP, Research, Therapy, Ruxolitinib, JAK, SAD, Inflammation, Orphan, Novo Holdings A/S, CKD, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Society, Food, Generic drug, Pharmaceutical industry, Vaccine, Cryptocurrency, Greater China, Disc

In August 2022, Disc initiated BEACON, an open-label Phase 2 clinical study of bitopertin in patients with EPP and X-linked protoporphyria (XLP).

Key Points: 
  • In August 2022, Disc initiated BEACON, an open-label Phase 2 clinical study of bitopertin in patients with EPP and X-linked protoporphyria (XLP).
  • In October 2022, Disc initiated AURORA, a Phase 2 randomized, placebo-controlled clinical study of bitopertin in adults with EPP.
  • Disc completed a reverse merger with Gemini Therapeutics in December 2022, which resulted in Disc becoming publicly listed on NASDAQ, and raised approximately $90 million in operating capital.
  • Full Year 2022 Financial Results:
    Cash Position: Cash and cash equivalents were $194.6 million as of December 31, 2022 compared to $88.0 million as of December 31, 2021.

Disc Medicine Announces $62.5 Million Financing led by Bain Capital Life Sciences to Advance Portfolio of Novel Hematology Programs

Retrieved on: 
Tuesday, February 14, 2023
Diamond, Erythropoiesis, AURORA, DBA, Diamond–Blackfan anemia, Erythropoietic protoporphyria, Science, IRON, CKD, Patient, Clinical trial, Polycythemia, Form, Inflammation, Anemia, Trial master file, Sodium- and chloride-dependent glycine transporter 1, OrbiMed, IND, S-3, Dialysis, FDA, Chronic kidney disease, HJV, EPP, Ruxolitinib, Doctor of Philosophy, Pharmaceutical industry, Security (finance), Disc

The financing was led by Bain Capital Life Sciences, who contributed $50 million, with participation from existing investors, Access Biotechnology and OrbiMed.

Key Points: 
  • The financing was led by Bain Capital Life Sciences, who contributed $50 million, with participation from existing investors, Access Biotechnology and OrbiMed.
  • The offering is expected to close on or about February 15, 2023, subject to customary closing conditions.
  • The shares of common stock and pre-funded warrants were offered pursuant to a shelf registration statement on Form S-3 (File No.
  • “We are delighted to welcome Bain Capital Life Sciences as an investor during a transformational time for Disc.

Disc Medicine Announces Completion of Merger with Gemini Therapeutics

Retrieved on: 
Thursday, December 29, 2022
Janus, Erythropoietic protoporphyria, Wilmer Cutler Pickering Hale and Dorr, Anemia, Ruxolitinib, Morgan Stanley, Sodium- and chloride-dependent glycine transporter 1, EPP, IRON, Bitopertin, Novo Holdings A/S, Patient, Degenerative disease, AURORA, Pharmaceutical industry, Disc, HJV

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.

Key Points: 
  • 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.
  • 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.
  • Morgan Stanley served as the lead financial advisor to Disc along with Wedbush PacGrow, and Goodwin Procter LLP served as Disc’s legal counsel.

CLINUVEL progresses vitiligo treatment program

Retrieved on: 
Tuesday, May 10, 2022
Linda, Light therapy, JAMA Dermatology, COVID-19, Treatment, Randomness, American Academy of Dermatology, Immune system, Narrowband, Union, GLOBE, EPP, IRB, Peptide, Afamelanotide, Food, Physician, Safety, Patient, Institutional review board, Phototoxicity, Program, ADR, FDA, Adult, Anaphylaxis, Family, US Foods, DNA, History, Skin, Burns, Health, Quality of life, ASX, Erythropoietic protoporphyria, American Academy, Disease, Melanocortin, CUV, DNA repair, Mental health, Melanocyte, Research, Bourke Street, Melbourne, Mazda North American Operations, Pharmaceutical industry, Medical device, Dermatology, Vitiligo

The drug afamelanotide approved by US and European regulators for a rare light intolerance disorder is being evaluated by Australian company CLINUVEL as a treatment for vitiligo patients with darker skin types.

Key Points: 
  • The drug afamelanotide approved by US and European regulators for a rare light intolerance disorder is being evaluated by Australian company CLINUVEL as a treatment for vitiligo patients with darker skin types.
  • Treatment options remain limited, with no treatments for repigmentation of vitiligo currently approved by the US Food and Drug Administration (FDA).
  • Therefore we have chosen to focus on this patient group, with the highest unmet need, as our clinical program progresses, Dr Teng said.
  • Afamelanotide implants and narrow-band ultraviolet B phototherapy for the treatment of nonsegmental vitiligo in Asians.

Disc Medicine Announces Oral Presentation on Bitopertin at the 63rd American Society of Hematology Annual Meeting

Retrieved on: 
Thursday, November 11, 2021
Society, Degenerative disease, Heme, Sodium- and chloride-dependent glycine transporter 1, Boston Children's Hospital, Glycine, Hospital, Disease, Roche, ASH, Bitopertin, Hematologic disease, Lists of diseases, Treatment, Multimedia, PPIX, XLP, Exposition, Medicine, EPP, Erythropoiesis, Development, Erythropoietic protoporphyria, Hope, Patient, Pharmaceutical industry

Disc Medicine is planning to develop bitopertin as a potential treatment for patients with EPP and XLP as well as a range of other hematologic diseases.

Key Points: 
  • Disc Medicine is planning to develop bitopertin as a potential treatment for patients with EPP and XLP as well as a range of other hematologic diseases.
  • Bitopertin is an experimental agent and is not approved for use as a therapy in any jurisdiction worldwide.
  • Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders.
  • Disc Medicine is committed to developing treatments that empower and bring hope to the many patients who suffer from hematologic disease.

CLINUVEL Expands Pharmaceutical Portfolio

Retrieved on: 
Monday, November 8, 2021
Maintenance, Hormone, Photoprotection, ADR, CUV, Blood pressure, ACTH, European Commission, CGMP, NASDAQ, LTD, Technology, Appetite, Food, London Bioscience Innovation Centre, Degenerative disease, US Food Sovereignty Alliance, Burns, Bureau of Oceans and International Environmental and Scientific Affairs, Research, Afamelanotide, Phototoxicity, Adrenocorticotropic hormone, EPP, Company, GLOBE, Good manufacturing practice, Metabolism, Development, Light, Anaphylaxis, DNA repair, ASX, TMR, Royal commission, Central nervous system, Biology, Patient, Cell, DNA, Drug development, Erythropoietic protoporphyria, Adult, Pharmaceutical industry, Medical device

MELBOURNE, Australia, Nov. 08, 2021 (GLOBE NEWSWIRE) -- CLINUVEL has expanded its pharmaceutical development portfolio with NEURACTHEL, novel formulations of the melanocortin adrenocorticotropic hormone (ACTH).

Key Points: 
  • MELBOURNE, Australia, Nov. 08, 2021 (GLOBE NEWSWIRE) -- CLINUVEL has expanded its pharmaceutical development portfolio with NEURACTHEL, novel formulations of the melanocortin adrenocorticotropic hormone (ACTH).
  • We also know there are many more underserved patient groups who would benefit from NEURACTHEL treatment, and we will be addressing these unmet medical needs.
  • Headquartered in Melbourne, Australia, CLINUVEL has operations in Europe, Singapore and the USA.
  • SCENESSE , PRNUMBRA , and NEURACTHEL are registered trademarks of CLINUVEL PHARMACEUTICALS LTD.

Global Expanded Polypropylene Foam Market Trends, Forecast and Competitive Analysis, 2013-2018 & 2019-2024 - ResearchAndMarkets.com

Retrieved on: 
Friday, September 11, 2020
Chemicals, Plastics, Manufacturing, Materials, Goods economics), Physical sciences, Dielectrics, Plastics, Polypropylene, Thermoplastics, Epp, Foam, Erythropoietic protoporphyria, Mat, Polymeric foam

The study includes the expanded polypropylene (EPP) foam market size and forecast for the expanded polypropylene (EPP) foam market through 2024, segmented by density type, end use industry, and region as follows:

Key Points: 
  • The study includes the expanded polypropylene (EPP) foam market size and forecast for the expanded polypropylene (EPP) foam market through 2024, segmented by density type, end use industry, and region as follows:
    Some of the features of 'Expanded Polypropylene (EPP) Foam Market 2019-2024: Trends, Forecast, and Opportunity Analysis' include
    Market size estimates: Expanded polypropylene (EPP) foam market size estimation in terms of value ($M) shipment.
  • Segmentation analysis: Market size by density type, by end use industry, and region
    Regional analysis: Expanded polypropylene (EPP) foam market breakdown by North America, Europe, Asia Pacific, and the Rest of the World
    Growth opportunities: Analysis on growth opportunities in different applications and regions for expanded polypropylene (EPP) foam in the expanded polypropylene (EPP) foam market.
  • Strategic analysis: This includes M&A, new product development, and competitive landscape for, expanded polypropylene (EPP) foam in the expanded polypropylene (EPP) foam market.
  • Q.11 What M & A activities have taken place in the last five years in the expanded polypropylene foam market?

PRÉNUMBRA® - CLINUVEL's Second Afamelanotide Formulation

Retrieved on: 
Monday, July 13, 2020
Hormones, Branches of biology, Peptide hormones, Organ systems, Peptides, Afamelanotide, Melanocyte-stimulating hormone, Erythropoietic protoporphyria, Porphyria, Proopiomelanocortin, Melanocortin, Epp

MELBOURNE, Australia and SINGAPORE, July 13, 2020 (GLOBE NEWSWIRE) -- CLINUVEL PHARMACEUTICALS today revealed PRNUMBRA, the Companys second afamelanotide product in development.

Key Points: 
  • MELBOURNE, Australia and SINGAPORE, July 13, 2020 (GLOBE NEWSWIRE) -- CLINUVEL PHARMACEUTICALS today revealed PRNUMBRA, the Companys second afamelanotide product in development.
  • PRNUMBRA is a liquid (non-solid) formulation of afamelanotide to be evaluated by CLINUVEL in clinical trials as a treatment for acute and systemic diseases.
  • CLINUVEL commercially launched the first formulation of afamelanotide, SCENESSE (afamelanotide 16mg), for the treatment of the rare genetic disorder erythropoietic protoporphyria (EPP).1 SCENESSE, a solid-dose injectable controlled-release implant formulation, is approved in the European Economic Area and United States for adults with EPP.
  • Afamelanotide is a synthetic analogue of the human -melanocyte stimulating hormone which belongs to the family of melanocortins or proopiomelanocortins (POMC).