Porphyria

Disc Medicine Reports First Quarter 2024 Financial Results and Provides Business Update

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星期四, 五月 9, 2024
Pain, Iron, MF, Safety, Research, Hematology, Porphyria, IRON, EPP, Development, Administration, Ruxolitinib, Patient, Anemia, Society, Quality of life, Bitopertin, PPIX

These data confirmed that bitopertin significantly reduced toxic PPIX in EPP patients and led to reduced phototoxic pain reactions and improved quality of life.

Key Points: 
  • These data confirmed that bitopertin significantly reduced toxic PPIX in EPP patients and led to reduced phototoxic pain reactions and improved quality of life.
  • Research and Development Expenses: R&D expenses were $23.7 million for the quarter ended March 31, 2024, as compared to $20.2 million for the quarter ended March 31, 2023.
  • General and Administrative Expenses: G&A expenses were $7.8 million for the quarter ended March 31, 2024, as compared to $4.9 million for the quarter ended March 31, 2023.
  • Net Loss: Net loss was $26.9 million for the quarter ended March 31, 2024, as compared to $22.8 million for the quarter ended March 31, 2023.

FDA Approves New Treatment for Uncomplicated Urinary Tract Infections

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星期三, 四月 24, 2024
Urine, UTI, FDA, Public, Patient, Staphylococcus, Urinary tract infection, Pivmecillinam, Priority review, Safety, Woman, Cosmetics, Food, Dietary supplement, Heated tobacco product, Infection, Research, Human services, Bacteria, Nausea, Staphylococcus saprophyticus, Escherichia coli, Porphyria, Vaccine, History, Female, Therapy, System, Tablet, Diarrhea, Hypersensitivity, Multimedia, Animal, Proteus mirabilis, Clinical trial, Metabolic disorder, Division, Ibuprofen

SILVER SPRING, Md., April 24, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Pivya (pivmecillinam) tablets for the treatment of female adults with uncomplicated urinary tract infections (UTIs) caused by susceptible isolates of Escherichia coli, Proteus mirabilis and Staphylococcus saprophyticus.

Key Points: 
  • SILVER SPRING, Md., April 24, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Pivya (pivmecillinam) tablets for the treatment of female adults with uncomplicated urinary tract infections (UTIs) caused by susceptible isolates of Escherichia coli, Proteus mirabilis and Staphylococcus saprophyticus.
  • "The FDA is committed to fostering new antibiotic availability when they prove to be safe and effective, and Pivya will provide an additional treatment option for uncomplicated UTIs."
  • Uncomplicated UTIs are bacterial infections of the bladder in females with no structural abnormalities of their urinary tract.
  • Approximately one-half of all women experience at least one UTI in their lifetime.

Orphan designation: Adeno-associated viral vector containing porphobilinogen deaminase gene Treatment of acute intermittent porphyria, 29/04/2009 Withdrawn

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星期二, 四月 9, 2024
Community, Eurostat, Civil service commission, Depression, Diagnosis, Psychosis, Cell, Disease, Seizure, Nervous system, Patient, Committee, Knowledge, Light, Regulation, Anxiety, Liver transplantation, EMA, IRIS, AIP, Human, Urine, European, Skin, COMP, Oxygen, Porphyria, European Commission, Safety, Blood, Marketing, Liver, DSM-IV codes, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector containing porphobilinogen deaminase gene Treatment of acute intermittent porphyria, 29/04/2009 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector containing porphobilinogen deaminase gene Treatment of acute intermittent porphyria, 29/04/2009 Withdrawn

Disc Medicine Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

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星期四, 三月 21, 2024
Well, Research, Administration, Development, ASH, EPP, MF, Patient, NTD, Trial of the century, Ruxolitinib, IRON, Anemia, Porphyria, JAK

“The past year has been truly remarkable for Disc and marks another important chapter in our company’s story.

Key Points: 
  • “The past year has been truly remarkable for Disc and marks another important chapter in our company’s story.
  • During 2023, we provided the first evidence of the therapeutic potential of our programs, with positive read-outs in patients for both bitopertin and DISC-0974.
  • The increase was primarily due to the completion of a registered direct offering in February 2023 and a publicly marketed follow-on offering in June 2023.
  • The increase was primarily due to higher operating costs in the current period to support the continued advancement of our pipeline.

Human medicines European public assessment report (EPAR): Amglidia, glibenclamide, Date of authorisation: 24/05/2018, Revision: 8, Status: Authorised

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星期二, 一月 2, 2024
Liver, Medical Subject Headings, Diarrhea, Marketing, Diabetes, INN, Diabetic ketoacidosis, Allergy, ATC, International, Pancreas, Insulin, Disease, Language, Blood, Porphyrin, Mellitus, Glycated hemoglobin, Mouth, Blood sugar level, Infant, Abdominal pain, Porphyria, KATP, European Medicines Agency, ATP, Tablet, Chronic pain, Select, Cell, Patient, Time, Anatomy, Medicine, IIA, Neonatal diabetes, Hypoglycemia, Life, Medical device

Human medicines European public assessment report (EPAR): Amglidia, glibenclamide, Date of authorisation: 24/05/2018, Revision: 8, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Amglidia, glibenclamide, Date of authorisation: 24/05/2018, Revision: 8, Status: Authorised

Disc Medicine Reports Third Quarter 2023 Financial Results and Provides Business Update

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星期四, 十一月 9, 2023
FDA, Research, IRON, Glycine, Erythropoiesis, Food, EPP, PV, Sodium- and chloride-dependent glycine transporter 1, G&A, AURORA, Iron, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Ruxolitinib, Light, Porphyria, Inflammation, Poster, Sunlight, Society, ASH, Development, Photosensitivity, Hematology, Quality of life, Phase 10, Anemia, PPIX, Safety, MF, XLP, Pain, Patient, Administration, Polycythemia, Rare-earth element, Pharmaceutical industry, Vaccine, Disc

Third Quarter 2023 Financial Results:

Key Points: 
  • Third Quarter 2023 Financial Results:
    Cash Position: Cash and cash equivalents were $370.5 million as of September 30, 2023, which are expected to fund our operational plans well into 2026.
  • Research and Development Expenses: R&D expenses were $14.4 million for the quarter ended September 30, 2023, as compared to $7.9 million for the quarter ended September 30, 2022.
  • General and Administrative Expenses: G&A expenses were $4.5 million for the quarter ended September 30, 2023, as compared to $2.6 million for the same period in 2022.
  • Net Loss: Net loss was $14.1 million for the quarter ended September 30, 2023, as compared to $16.2 million for the third quarter of 2022.

Disc Medicine Reports Second Quarter 2023 Financial Results and Provides Business Update

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星期五, 八月 11, 2023
Sodium- and chloride-dependent glycine transporter 1, Ruxolitinib, Chronic kidney disease, European Hematology Association, EPP, AURORA, G&A, Porphyria, Inflammation, MF, Dialysis, Congress, IRON, Development, Erythropoiesis, Glycine, Patient, Administration, Anemia, EHA, Mab, XLP, Research, Polycythemia, Technology transfer, Safety, Pharmaceutical industry, Nursing, Cryptocurrency, Vaccine, Disc

Disc plans to initiate a Phase 1 study in healthy volunteers during the second half of 2023.

Key Points: 
  • Disc plans to initiate a Phase 1 study in healthy volunteers during the second half of 2023.
  • Second Quarter 2023 Financial Results:
    Cash Position: Cash and cash equivalents were $377.6 million as of June 30, 2023, which are expected to fund our operational plans well into 2026.
  • Research and Development Expenses: R&D expenses were $12.1 million for the quarter ended June 30, 2023, as compared to $7.7 million for the quarter ended June 30, 2022.
  • Net Loss: Net loss was $15.9 million for the quarter ended June 30, 2023, as compared to $9.6 million for the second quarter of 2022.

Disc Medicine Reports First Quarter 2023 Financial Results and Provides Business Update

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星期一, 五月 15, 2023
Diamond, Sodium- and chloride-dependent glycine transporter 1, Interim, Diamond–Blackfan anemia, Ruxolitinib, Chronic kidney disease, European Hematology Association, OrbiMed, AURORA, G&A, Porphyria, Inflammation, MF, Dialysis, Net, Congress, IRON, ATM, Erythropoiesis, Glycine, Development, Patient, Administration, Anemia, EHA, Mab, Research, FDA, Polycythemia, CKD, IND, NIH, TMPRSS6, Safety, Pharmaceutical industry, Generic drug, Vaccine, Cryptocurrency, Nursing, Disc, EPP, Greater China

Interim data will be presented at EHA Congress on June 9, 2023, with an accompanying management call at 7:30 am ET the same day.

Key Points: 
  • Interim data will be presented at EHA Congress on June 9, 2023, with an accompanying management call at 7:30 am ET the same day.
  • Announced a collaboration with NIH to study bitopertin in patients with Diamond-Blackfan Anemia in March 2023; the study is expected to initiate mid-year 2023.
  • First Quarter 2023 Financial Results:
    Cash Position: Cash and cash equivalents were $236.4 million as of March 31, 2023 compared to $194.6 million as of December 31, 2022.
  • Net Loss: The net loss was $22.8 million for the first quarter of 2023, as compared to $9.9 million for the first quarter of 2022.

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

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星期四, 四月 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

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星期五, 三月 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.