Bitopertin

Disc Reports Topline Results from Phase 2 AURORA Study of Bitopertin in Patients with Erythropoietic Protoporphyria (EPP)

Retrieved on: 
Monday, April 1, 2024
PGIC, EPP, Patient, Light, Ruxolitinib, IRON, Sunlight, Bitopertin, Anemia, Pain, Medical imaging

On the key secondary endpoint of cumulative time in sunlight on days without pain, bitopertin patients had a positive response consistent with BEACON results, but the endpoint did not meet statistical significance due to strong placebo performance.

Key Points: 
  • On the key secondary endpoint of cumulative time in sunlight on days without pain, bitopertin patients had a positive response consistent with BEACON results, but the endpoint did not meet statistical significance due to strong placebo performance.
  • Subjects were randomized 1:1:1 to receive 20 mg of bitopertin, 60 mg of bitopertin, or placebo once daily for 17 weeks.
  • Two patients discontinued treatment due to treatment-emergent AEs, both in the 60 mg dose group: one due to dizziness and one due to a skin rash.
  • Please register for management’s webcast on the Events and Presentations page of Disc’s website ( https://ir.discmedicine.com/ ).

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.

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

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 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.

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.

Disc Medicine Expands Hematology Pipeline with Worldwide Licensing Agreement for Bitopertin, a First-in-Class Modulator of Heme Synthesis

Retrieved on: 
Thursday, May 27, 2021
Porphyrias, Chemical compounds, Branches of biology, Chemistry, Glycine reuptake inhibitors, Bitopertin, Heme, Erythropoietic porphyria, Sodium- and chloride-dependent glycine transporter 1, Cytochrome P450 reductase

CAMBRIDGE, Mass., May 27, 2021 /PRNewswire/ --Disc Medicine, Inc. announced today that it has entered into an exclusive worldwide licensing agreement withF.

Key Points: 
  • CAMBRIDGE, Mass., May 27, 2021 /PRNewswire/ --Disc Medicine, Inc. announced today that it has entered into an exclusive worldwide licensing agreement withF.
  • Hoffmann-La Roche Ltdfor the development and commercialization of bitopertin, an orally administered GlyT1 inhibitor with demonstrated effects on the heme biosynthesis pathway.
  • "Importantly, the well-established clinical safety profile of bitopertin will allow us to move rapidly into patient studies of several serious, hematologic diseases."
  • Erythropoietic porphyrias (EPs) are a family of ultra-rare, debilitating and potentially life-threatening diseases caused by mutations that affect the heme synthesis pathway.