Erythropoietic protoporphyria

Attention Disc Medicine, Inc. Investors: Company Investigated by the Portnoy Law Firm

Retrieved on: 
Monday, April 1, 2024
Patient, Placebo, Disc, IRON, Erythropoietic protoporphyria, EPP

LOS ANGELES, April 01, 2024 (GLOBE NEWSWIRE) -- The Portnoy Law Firm advises Disc Medicine, Inc. (“Disc” or the “Company”) (NASDAQ: IRON) investors that the law firm has initiated an investigation on behalf of investors that lost money on their Disc stock. Disc investors are encouraged to contact the firm to discuss their legal rights.

Key Points: 
  • LOS ANGELES, April 01, 2024 (GLOBE NEWSWIRE) -- The Portnoy Law Firm advises Disc Medicine, Inc. (“Disc” or the “Company”) (NASDAQ: IRON) investors that the law firm has initiated an investigation on behalf of investors that lost money on their Disc stock.
  • Disc investors are encouraged to contact the firm to discuss their legal rights.
  • The Portnoy Law Firm can provide a complimentary case evaluation and discuss investors’ options for pursuing claims to recover their losses.
  • The Portnoy Law Firm represents investors in pursuing claims caused by corporate wrongdoing.

Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Diamond, Community, MC1R, Civil service commission, Narcolepsy, Diagnosis, Pigment, Erythropoietic protoporphyria, Prevalence, Disease, EPP, Patient, Committee, EMA, IRIS, Agency, European, Skin, COMP, Sponsor, European Commission, Pain, Phototoxicity, PAH, Marketing, Phenylalanine hydroxylase, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Key Points: 


Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Orphan designation: Bitopertin Treatment of erythropoietic protoporphyria, 15/02/2023 Positive

Retrieved on: 
Saturday, January 20, 2024
COMP, Committee, Erythropoietic protoporphyria, European Commission, IRIS, Patient, EMA, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Bitopertin
    - Intended use
    - Treatment of erythropoietic protoporphyria
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2761
    - Date of designation
    - Sponsor
    Disc Medicine B.V.
  • Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Disc Presents Positive Updated Results from Phase 2 BEACON Study of Bitopertin and Other Programs at the 65th American Society of Hematology (ASH) Annual Meeting

Retrieved on: 
Tuesday, December 12, 2023
Quality of life, Anemia, Protoporphyrin IX, Bitopertin, Erythropoietic protoporphyria, IRON, Ruxolitinib, Disc, ASH, Photosensitivity, Light, Growth, XLP, Patient, Safety, EPP, Surveying

“This has been a tremendous ASH meeting for Disc, as we presented data across our two most advanced programs.

Key Points: 
  • “This has been a tremendous ASH meeting for Disc, as we presented data across our two most advanced programs.
  • “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.
  • Earlier today, Disc also presented initial positive data from the ongoing phase 1b study of DISC-0974 in myelofibrosis (MF) patients with anemia.
  • Bitopertin and DISC-0974 are investigational agents and are not approved for use as a therapy in any jurisdiction worldwide.

Disc Medicine Announces Grant of US Patent for Methods of Treating Erythropoietic Protoporphyrias with Bitopertin

Retrieved on: 
Tuesday, November 14, 2023
Bitopertin, Erythropoietic protoporphyria, ASH, EPP, Patient, IRON, XLP, Patent, Pharmaceutical industry, Disc

WATERTOWN, Mass., Nov. 14, 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 that the United States Patent and Trademark Office (USPTO) has issued patent No.

Key Points: 
  • WATERTOWN, Mass., Nov. 14, 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 that the United States Patent and Trademark Office (USPTO) has issued patent No.
  • 11,813,257 for methods of treating erythropoietic protoporphyrias (EPPs) with glycine transport inhibitors.
  • “We are pleased to have been issued this patent that further secures IP protection for bitopertin for the treatment of erythropoietic protoporphyria as we work to deliver a potentially transformative therapy to patients,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc.
  • Disc intends to continue to prosecute additional patent applications to support use of bitopertin in treating hematologic disorders.

Orphan designation: Dersimelagon Treatment of erythropoietic protoporphyria, 16/03/2022 Positive

Retrieved on: 
Monday, December 18, 2023
Medicine, COMP, Prevalence, Update, European Commission, Trick or Treatment?, Mitsubishi Tanabe Pharma, Phototoxicity, EPP, Disease, Narcolepsy, EMA, Civil service commission, Patient, Agency, European, MC1R, Marketing, Committee, Pigment, Community, Diagnosis, Pain, No Date, No Signature, Erythropoietic protoporphyria, Skin, EURORDIS, Heerdt, Pharmaceutical industry, Medical device, Fine chemical

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of erythropoietic protoporphyria in the European Union on 16 March 2022.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Disc Medicine Announces First Patient Enrolled in Phase 1/2 Clinical Trial of Bitopertin in Diamond-Blackfan Anemia (DBA)

Retrieved on: 
Thursday, July 27, 2023
Diamond, Sodium- and chloride-dependent glycine transporter 1, DBA, Head, EPP, Principal investigator, National Heart, Lung, and Blood Institute, CRADA, Research, Diamond–Blackfan anemia, Lung, Bangladesh Technical Education Board, Development, NHLBI, IRON, Cooperative research and development agreement, Patient, Erythropoietic protoporphyria, EHA, NIH, Disease, Pharmaceutical industry

Bitopertin is an investigational, orally administered glycine transporter 1 (GlyT1) inhibitor designed to modulate heme biosynthesis.

Key Points: 
  • Bitopertin is an investigational, orally administered glycine transporter 1 (GlyT1) inhibitor designed to modulate heme biosynthesis.
  • “The start of this trial represents another step toward demonstrating the potential of bitopertin to address a wide range of hematologic conditions.” said Will Savage, M.D., Ph.D., Chief Medical Officer of Disc.
  • 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.

Portfolio company Disc Medicine presents positive initial data from Phase 2 BEACON trial at EHA

Retrieved on: 
Saturday, June 10, 2023
PPIX, Sodium- and chloride-dependent glycine transporter 1, LSE, Disc, Biotechnology, Quality of life, Congress, Patient, Erythropoietic protoporphyria, EHA, XLP, Bitopertin, Photosensitivity, Safety, Pharmaceutical industry, EPP

The initial trial data from the BEACON study demonstrated consistent decreases in protoporphyrin IX (the disease-causing metabolite in EPP), significant increases in reported sunlight tolerance and improvements in measures of patient quality of life.

Key Points: 
  • The initial trial data from the BEACON study demonstrated consistent decreases in protoporphyrin IX (the disease-causing metabolite in EPP), significant increases in reported sunlight tolerance and improvements in measures of patient quality of life.
  • The BEACON trial is a randomised, open-label, parallel-arm trial enrolling up to 22 patients with EPP or XLP at trial sites in Australia.
  • Robert Lyne, CEO of Arix Bioscience, said: “We are very encouraged by the positive data from the BEACON trial.
  • We look forward to supporting Disc Medicine as they continue to advance the BEACON study through clinical development.

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

Retrieved on: 
Friday, June 9, 2023
EPIQ, Sodium- and chloride-dependent glycine transporter 1, Protoporphyrin IX, European Hematology Association, Metallicity, Disc, Week, EPP, Bitopertin, Quality of life, First grade, Pain, Congress, Sunlight, IRON, Patient, Erythropoietic protoporphyria, EHA, XLP, Photosensitivity, Poster, Safety, Disease, Pharmaceutical industry, Medical device, Sewage treatment, PPIX

The initial trial data demonstrated consistent decreases in PPIX, significant increases in reported sunlight tolerance and improvements in measures of patient quality of life.

Key Points: 
  • The initial trial data demonstrated consistent decreases in PPIX, significant increases in reported sunlight tolerance and improvements in measures of patient quality of life.
  • “We’re delighted to share these initial, positive data from BEACON, which provide the first clinical evidence supporting our therapeutic hypothesis of bitopertin in EPP.
  • Due to batch processing of samples, the data cutoff for PPIX data was April 7, 2023.
  • Management will host a call to review the presented data on Friday, June 9th at 7:30 am ET.

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