TMPRSS6

Agios Reports Business Highlights and Third Quarter 2023 Financial Results

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星期四, 十一月 2, 2023
Anti-transglutaminase antibodies, Research, RISE, PKU, Research and development, IND, AGIO, Rare, Sickle cell disease, MDS, Public expenditure, Enrollment, SG, Pyruvate kinase deficiency, R, Society, Phenylketonuria, ASH, Thalassemia, PK, Security, Abstract, Phenylalanine hydroxylase, TMPRSS6, Agios Pharmaceuticals, PAH, Agios, Patient, Administration, Nursing, Cryptocurrency, Pharmaceutical industry, Medical device, Video game, Alnylam Pharmaceuticals

CAMBRIDGE, Mass., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the third quarter ended September 30, 2023.

Key Points: 
  • Launch: Generated $7.4 million in U.S. net revenue for the third quarter of 2023, a 10 percent increase over the second quarter of 2023.
  • A total of 160 unique patients have completed prescription enrollment forms, representing an increase of 9 percent over the second quarter of 2023.
  • Net Loss: Net loss was $91.3 million for the third quarter of 2023 compared to $81.7 million for the third quarter of 2022.
  • ET to discuss third quarter 2023 financial results and recent business activities.

Disc Medicine Initiates a Phase 1 Study of DISC-3405 (anti-TMPRSS6 mAb) in Healthy Volunteers

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星期二, 十月 3, 2023
IRON, Pharmacokinetics, Erythropoiesis, Polycythemia, Patient, Fast track (FDA), Iron, TMPRSS6, Safety, Pharmaceutical industry, Disc

DISC-3405 is a monoclonal antibody designed to target TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) to increase hepcidin and decrease iron.

Key Points: 
  • DISC-3405 is a monoclonal antibody designed to target TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) to increase hepcidin and decrease iron.
  • “The initiation of this trial marks the third program that Disc has brought into the clinic and strengthens our position as leaders in the field of hepcidin biology and iron homeostasis.
  • We look forward to pursuing those indications upon the completion of this trial,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc.
  • Following completion of this study, Disc plans to initiate a trial in polycythemia vera, for which DISC-3405 has received Fast Track Designation.

Agios Announces Exclusive Worldwide License Agreement for Novel siRNA for the Potential Treatment of Polycythemia Vera

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星期四, 八月 3, 2023
PK, Rat, Hematologic disease, Therapy, Polycythemia vera, Hematocrit, PV, Small RNA, AGIO, Thrombosis, Agios, Phlebotomy, Patient, Standard of care, Iron, Risk, Hematology, RNA interference, Agios Pharmaceuticals, Blood volume, Death, IND, TMPRSS6, Pharmaceutical industry, Alnylam Pharmaceuticals

CAMBRIDGE, Mass., Aug. 03, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO) has entered into an exclusive worldwide license agreement with Alnylam Pharmaceuticals, Inc. under which Agios will acquire the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV). This agreement combines Agios’ deep scientific expertise and capabilities in rare hematologic diseases with Alnylam’s industry-leading siRNA platform and strong track record of success.

Key Points: 
  • CAMBRIDGE, Mass., Aug. 03, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO) has entered into an exclusive worldwide license agreement with Alnylam Pharmaceuticals, Inc. under which Agios will acquire the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV).
  • This agreement combines Agios’ deep scientific expertise and capabilities in rare hematologic diseases with Alnylam’s industry-leading siRNA platform and strong track record of success.
  • Under the terms of the agreement, Agios will make an upfront payment of $17.5 million to Alnylam for an exclusive global license to the TMPRSS6 siRNA program.
  • Alnylam will provide manufacturing support for Phase 1, after which Agios will assume full responsibility for manufacturing.

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 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.

Silence Therapeutics Announces Publication in Blood Demonstrating Role for Iron Regulation in Polycythemia Vera

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星期二, 四月 4, 2023
Research, Infectious Diseases, Genetics, Cardiology, Biotechnology, Other Health, Health, Other Science, Science, ASH, Population health, Beta thalassemia, GWAS, RNA, PV, Professor, WEHI, Patient, Risk, HFE, Liver, Therapy, Polycythemia, Phenotype, Society, TMPRSS6, Disease, Vaccine, Blood, Silence

The paper is authored by senior medical and scientific experts from Silence and leading population health and hematology researchers from WEHI (Walter and Eliza Hall Institute) in Melbourne, Australia as well as Cambride UK.

Key Points: 
  • The paper is authored by senior medical and scientific experts from Silence and leading population health and hematology researchers from WEHI (Walter and Eliza Hall Institute) in Melbourne, Australia as well as Cambride UK.
  • The study further demonstrated in a mouse model of PV that hepcidin, a master regulator of iron availability whose expression is influenced by HFE, governs the red blood cell (erythroid) phenotype in PV.
  • “The results we reported in Blood provide a genetic and biological rationale for treating PV with SLN124 by raising hepcidin to control systemic iron levels, thus reducing red blood cell count.
  • SLN124 works by silencing TMPRSS6, a gene that negatively regulates hepcidin expression, to increase production of hepcidin in the liver.

Disc Medicine Announces Exclusive Licensing Agreement with Mabwell Therapeutics for Novel Anti-TMPRSS6 Monoclonal Antibodies to Modulate Iron Homeostasis

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星期五, 一月 20, 2023
IND, Degenerative disease, Polycythemia, Thalassemia, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IRON, Erythropoiesis, FDA, Iron, Biology, Food, Patient, Investigational New Drug, TMPRSS6, Pharmaceutical industry, Disc

Disc plans to initiate a phase 1 trial in healthy volunteers in the second half of 2023.

Key Points: 
  • Disc plans to initiate a phase 1 trial in healthy volunteers in the second half of 2023.
  • MWTX-003 has the potential to address a wide range of hematologic disorders including polycythemia vera and beta-thalassemia by controlling iron homeostasis.
  • Genetic studies show that TMPRSS6 affects red blood cell formation by controlling the level of iron that is available for erythropoiesis.
  • The transaction is subject to customary closing conditions and approval by the shareholders of the parent company of Mabwell Therapeutics, Mabwell (Shanghai) Bioscience Co., Ltd.

Silence Therapeutics Announces Preliminary Single Dose Results from SLN124 Phase 1 Study in Patients with Thalassemia

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星期四, 九月 29, 2022
Other Manufacturing, Genetics, Transport, Clinical Trials, Manufacturing, Biotechnology, Health, General Health, Logistics, Supply Chain Management, Therapy, Thalassemia, Safety, RNA interference, Sirna, Mallinckrodt, FDA, Biomarker, Blood, TMPRSS6, U.S. Securities and Exchange Commission, Gene, Company, Low-density lipoprotein, GEMINI, Industry, Liver injury, Sirna Therapeutics, Patient, RNA, Polycythemia, AstraZeneca, Pathology, Nasdaq, Toxicity, Liver, Annual report, Private Securities Litigation Reform Act, Fast track (FDA), PV, Transferrin, Silence Therapeutics, Research, PD, Anemia, Pharmaceutical industry, MD, Silence

The primary objective of the single dose arm was to evaluate the safety and tolerability of SLN124 subcutaneous dosing (1.0, 3.0 and 6.0 mg/kg) in alpha/beta-thalassemia patients.

Key Points: 
  • The primary objective of the single dose arm was to evaluate the safety and tolerability of SLN124 subcutaneous dosing (1.0, 3.0 and 6.0 mg/kg) in alpha/beta-thalassemia patients.
  • Following a single dose, there were no serious adverse events, severe treatment emergent adverse events (TEAEs) that were SLN124 related or TEAEs leading to withdrawal.
  • SLN124 has rare pediatric disease and orphan drug designations for beta-thalassemia as well as orphan drug designation for PV.
  • SLN124 has demonstrated proof of concept in healthy volunteers and is being evaluated in a phase 1 study in patients with non-transfusion dependent thalassemia.

Silence Therapeutics Announces FDA Fast Track Designation for SLN124, a Novel Investigational siRNA Therapy for the Treatment of Polycythemia Vera

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星期四, 九月 8, 2022
Health, FDA, Genetics, Other Health, Clinical Trials, Research, Science, AstraZeneca, RNA interference, Polycythemia, RNA, Research, Lipoprotein(a), Gene, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Therapy, Liver, Myelodysplastic syndrome, Pathology, TMPRSS6, Food, Mallinckrodt, Hematologic disease, Thalassemia, Review, U.S. Securities and Exchange Commission, Sirna, Nasdaq, Company, FDA, PV, Silence Therapeutics, Sirna Therapeutics, Annual report, Private Securities Litigation Reform Act, Industry, Patient, Pharmaceutical industry

SLN124 demonstrated proof of mechanism and was well tolerated in a healthy volunteer study completed last year.

Key Points: 
  • SLN124 demonstrated proof of mechanism and was well tolerated in a healthy volunteer study completed last year.
  • "The granting of Fast Track Designation represents an important recognition by the FDA of SLN124s potential to address a significant unmet need in the treatment of PV," said Craig Tooman, President and Chief Executive Officer of Silence.
  • Fast Track designation aims to facilitate the development and accelerate the review of new therapeutics that are intended to treat serious or life-threatening conditions and that potentially address an unmet medical need.
  • Drugs that are granted this designation are given the opportunity for more frequent interactions with the FDA, as well as potential pathways for expedited approval.

Portfolio Company Disc Medicine Enters Definitive Merger Agreement with Gemini Therapeutics

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星期三, 八月 10, 2022
Diamond, Chronic kidney disease, Sickle cell disease, Sodium- and chloride-dependent glycine transporter 1, Hepcidin, Erythropoietic porphyria, Inflammation, Industry, DBA, AbbVie, Microdeletion syndrome, Genetic disorder, Cancer, Medicine, Diamond–Blackfan anemia, Hope, Patient, TMPRSS6, Biotechnology, Degenerative disease, Disc, Anemia, Polycythemia, Iron overload, Ruxolitinib, Hematologic disease, Thalassemia, Hereditary haemochromatosis, Pharmaceutical industry, HJV

Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders.

Key Points: 
  • Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders.
  • Disc is building a portfolio of innovative, first-in-class therapeutic candidates that affect fundamental pathways of red blood cell biology.
  • Disc Medicine is committed to developing treatments that empower and bring hope to the many patients who suffer from hematologic disease.
  • Disc has a clinical-stage development pipeline composed of investigational product candidates that affect heme biosynthesis and iron metabolism.