HCU

Travere Therapeutics to Present Abstracts at the Society for Inherited Metabolic Disorders and Genetic Metabolic Dieticians International

Retrieved on: 
Thursday, April 4, 2024
Homocysteine, Homocystinuria, Caregiver, SAN, HCU, Society, Treatment, Therapy, Genetic, SIMD, Pharmaceutical industry

SAN DIEGO, April 04, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present eight posters in classical homocystinuria (HCU) at Society for Inherited Metabolic Disorders (SIMD) in Charlotte, North Carolina from April 14-17, 2024, and Genetic Metabolic Dieticians International (GMDI) in Charlotte, North Carolina from April 17-20, 2024.

Key Points: 
  • SAN DIEGO, April 04, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present eight posters in classical homocystinuria (HCU) at Society for Inherited Metabolic Disorders (SIMD) in Charlotte, North Carolina from April 14-17, 2024, and Genetic Metabolic Dieticians International (GMDI) in Charlotte, North Carolina from April 17-20, 2024.
  • At SIMD and GMDI, the Company will present the trial design of the pivotal Phase 3 HARMONY Study of pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of classical HCU.
  • Additionally, the Company will share insights on the development of an innovative tool used for dietary management and monitoring in the Phase 3 HARMONY Study and open-label extension ENSEMBLE Study, and the positive results from cohort 6 in the placebo-controlled Phase 1/2 COMPOSE Study of pegtibatinase in classical HCU.
  • “Classical HCU is an isolating and devastating rare disorder with very limited treatment options, including adherence to highly restrictive diets, leaving patients and their caregivers with immense challenges,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics.

OTC Markets Group Welcomes Horizon Copper Corp. to OTCQX

Retrieved on: 
Thursday, April 4, 2024
Copper, United, Growth, OTCQX, HCU, Security (finance)

NEW YORK, April 04, 2024 (GLOBE NEWSWIRE) -- OTC Markets Group Inc. (OTCQX: OTCM), operator of regulated markets for trading 12,000 U.S. and international securities, today announced Horizon Copper Corp. (“Horizon Copper” or “Horizon”) (TSX-V: HCU; OTCQX: HNCUF), a copper company with a portfolio of high-quality copper assets, has qualified to trade on the OTCQX® Best Market.

Key Points: 
  • NEW YORK, April 04, 2024 (GLOBE NEWSWIRE) -- OTC Markets Group Inc. (OTCQX: OTCM), operator of regulated markets for trading 12,000 U.S. and international securities, today announced Horizon Copper Corp. (“Horizon Copper” or “Horizon”) (TSX-V: HCU; OTCQX: HNCUF), a copper company with a portfolio of high-quality copper assets, has qualified to trade on the OTCQX® Best Market.
  • Horizon Copper begins trading today on OTCQX under the symbol “HNCUF.” U.S. investors can find current financial disclosure and Real-Time Level 2 quotes for the company on www.otcmarkets.com .
  • Upgrading to the OTCQX Market is an important step for companies seeking to provide transparent trading for their U.S. investors.
  • “2024 is expected to bring several growth catalysts for Horizon Copper,” commented Erfan Kazemi, Horizon’s President & CEO.

MCR SEO Limited Launches In-Depth Analysis on the Impact of Google's Helpful Content Update on Specialized Service Websites

Retrieved on: 
Thursday, February 22, 2024
Area studies, Content, Content creation, AI, HR, MCR, HCU

Manchester, United Kingdom--(Newsfile Corp. - February 22, 2024) - MCR SEO Limited, a pioneering force in the UK digital marketing landscape, is proud to announce the release of its latest analysis focusing on Google's "Helpful Content Update" (HCU) and its implications for websites offering specialized services.

Key Points: 
  • Manchester, United Kingdom--(Newsfile Corp. - February 22, 2024) - MCR SEO Limited, a pioneering force in the UK digital marketing landscape, is proud to announce the release of its latest analysis focusing on Google's "Helpful Content Update" (HCU) and its implications for websites offering specialized services.
  • As Google continues to prioritize high-quality, user-centric content, MCR SEO Limited's timely analysis offers vital insights into how specialized service websites can optimize their content to align with Google's evolving standards.
  • Key Highlights of the Analysis:
    Strategic Insights: Understand Google's "Helpful Content Update" and its emphasis on rewarding websites that provide valuable, user-focused content.
  • AI and Content Creation: Explore the impact of AI on content development and how to maintain authenticity in the age of automated material.

Travere Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Thursday, February 15, 2024
SPARTAN, Research, New Drug Application, Growth, Table, CSL Vifor, DSM-IV codes, Webcast, Conditional, HCU, FY, Medication, Diagnosis, Plasma, Bank statement, Committee, FSGS, UPCR, GAAP, Therapy, CHMP, CMA, International, SAN, Total, Shanda, Kidney disease, Safety, Clinical trial, Quarter, FDA, Food, Risk, Income tax, PMDA, Patient, Disease, Kidney, Pivotal, Proteinuria, Pharmaceutical industry

Net product sales for the fourth quarter of 2023 were $39.9 million, compared to $25.8 million for the same period in 2022.

Key Points: 
  • Net product sales for the fourth quarter of 2023 were $39.9 million, compared to $25.8 million for the same period in 2022.
  • Research and development (R&D) expenses for the fourth quarter of 2023 were $59.7 million, compared to $58.1 million for the same period in 2022.
  • Total other income, net, for the fourth quarter of 2023 was $5.7 million, compared to $1.1 million for the same period in 2022.
  • ET to discuss company updates as well as fourth quarter and full year 2023 financial results.

Synlogic Announces Publication of Preclinical and Clinical Data for SYNB1353 as a Potential Treatment for Classical Homocystinuria

Retrieved on: 
Friday, February 2, 2024
Nature, Manuscript, Met, Homocysteine, AbbVie, Trial of the century, Cell Host & Microbe, Absorption, Research, Therapy, Patient, Safety, Methionine, Extrapyramidal system, Plasma, HCU, Dietary supplement

CAMBRIDGE, Mass., Feb. 02, 2024 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a biopharmaceutical company advancing novel therapeutics to transform the care of serious diseases, announced today the publication of a manuscript detailing the development and activity of SYNB1353 in preclinical models and demonstrating safety, tolerability, and clinical proof of mechanism in healthy volunteers through the successful lowering of methionine (Met), a precursor to homocysteine, in a dietary model of classical homocystinuria (HCU).

Key Points: 
  • Among findings outlined in the manuscript, SYNB1353 efficiently degraded both dietary and entero-recirculating methionine to prevent its absorption and subsequent conversion to homocysteine in preclinical models, suggesting that SYNB1353 should result in lowering of plasma homocysteine levels in HCU patients.
  • In addition, in results from the clinical study in healthy volunteers, SYNB1353 was generally well-tolerated and adverse events were mild to moderate, transient, and predominantly gastrointestinal in nature.
  • The proportion of subjects reporting gastrointestinal events were similar for the SYNB1353 and placebo cohorts (36.4% and 37.5%, respectively).
  • “Importantly, the SYNB1353 program has built on learnings and progress from our other rare metabolic disease programs, including the design of the potential therapeutic, the preclinical research and the advancement in clinical development.”
    The publication, entitled “The Live Biotherapeutic SYNB1353 Decreases Plasma Methionine via Directed Degradation in Animal Models and Healthy Volunteers,” and published in the peer-reviewed journal, Cell Host & Microbe, is now available online at https://www.sciencedirect.com/science/article/pii/S193131282400009X .

Travere Therapeutics Provides Corporate Update and 2024 Outlook

Retrieved on: 
Monday, January 8, 2024
CMA, Risk, Shanda, FDA, Plasma, UPCR, European, FSGS, Disease, Education, Therapy, New Drug Application, Kidney disease, CSL Vifor, Proteinuria, SPARTAN, CHMP, HCU, Growth, Committee, Patient, Safety, Diagnosis, Conditional, Pharmaceutical industry

SAN DIEGO, Jan. 08, 2024 (GLOBE NEWSWIRE) --  Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that, based on preliminary and unaudited financial data, the Company expects net product sales from continuing operations for the fourth quarter of 2023 to be approximately $40 million. For the fiscal year 2023, the Company expects net product sales from continuing operations to be approximately $128 million. The Company ended 2023 with approximately $567 million in cash, cash equivalents, and marketable securities which is expected to support operations into 2028. The Company also provided an update on key corporate, clinical, and regulatory development initiatives, including anticipated milestones for 2024.

Key Points: 
  • For the fiscal year 2023, the Company expects net product sales from continuing operations to be approximately $128 million.
  • The Company also provided an update on key corporate, clinical, and regulatory development initiatives, including anticipated milestones for 2024.
  • "Our strong fourth quarter results reflect the growing demand for FILSPARI in IgAN driven by our field teams’ education and outreach efforts to healthcare providers,” said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics.
  • The Company expects to announce complete full year 2023 financial results and provide a corporate update in February.

Synlogic Provides Corporate Update and Outlook for 2024

Retrieved on: 
Thursday, January 4, 2024
Conference, Food, AbbVie, Met, Nature Metabolism, Homocysteine, FDA, Plasma, DMC, Annual general meeting, Diet, Ageing, Trial of the century, Society, GI, Data monitoring committee, Phenylalanine, Part, SIMD, PKU, HCU, Patient, Safety, Pharmaceutical industry

CAMBRIDGE, Mass., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology company advancing novel, oral, non-systemically absorbed biotherapeutics to transform the care of serious diseases, today summarized accomplishments for 2023 and anticipated key milestones for 2024.

Key Points: 
  • “We are pleased with the important corporate progress we achieved in 2023 - highlighted by the initiation of our global pivotal Synpheny-3 study evaluating our potentially transformative treatment option for PKU,” said Aoife Brennan, M.B.
  • Ch.B., Synlogic President and Chief Executive Officer.
  • “We expect 2024 to be similarly momentous, with the achievement of key milestones, including the upcoming Data Monitoring Committee review of initial study data.
  • More information on the Synpheny-3 study is available at www.clinicaltrials.gov, identifier NCT05764239 and also by visiting pkuresearchstudy.com.

Travere Therapeutics Initiates Pivotal Phase 3 Clinical Trial of Pegtibatinase for the Treatment of Classical Homocystinuria (HCU)

Retrieved on: 
Thursday, December 14, 2023
Patient, Food, Breakthrough therapy, Caregiver, Homocysteine, Research, FDA, Plasma, Intellectual, SAN, Therapy, Stroke, Orphan drug, CBS, Doctor of Philosophy, HCU, Diagnosis, Safety, Pharmaceutical industry

SAN DIEGO, Dec. 14, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced the Company has opened enrollment in the HARMONY Study, a global, randomized pivotal Phase 3 clinical trial of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU).

Key Points: 
  • SAN DIEGO, Dec. 14, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced the Company has opened enrollment in the HARMONY Study, a global, randomized pivotal Phase 3 clinical trial of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU).
  • Classical HCU is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS).
  • “Classical HCU is a devastating rare disease that often manifests in childhood and can lead to serious complications due to toxic levels of homocysteine.
  • Participants will be randomized 1:1 to receive 2.5 mg/kg of pegtibatinase or placebo, administered subcutaneously, for a 24-week blinded treatment duration.

Synlogic Announces Abstract Accepted for Poster Presentation at the International Conference on Microbiome Engineering 2023

Retrieved on: 
Thursday, December 7, 2023
HCU, Homocystinuria, Poster, Conference, AbbVie

CAMBRIDGE, Mass., Dec. 07, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology company advancing novel, oral, non-systemically absorbed biotherapeutics to transform the care of serious diseases, announced that an abstract outlining the SYNB1353 program as a potential treatment for homocystinuria (HCU), including findings from fermentation process improvements to increase activity of methionine degradation, has been accepted and will be presented at the International Conference on Microbiome Engineering 2023, held in Berkeley, California on December 8th to 10th.

Key Points: 
  • CAMBRIDGE, Mass., Dec. 07, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology company advancing novel, oral, non-systemically absorbed biotherapeutics to transform the care of serious diseases, announced that an abstract outlining the SYNB1353 program as a potential treatment for homocystinuria (HCU), including findings from fermentation process improvements to increase activity of methionine degradation, has been accepted and will be presented at the International Conference on Microbiome Engineering 2023, held in Berkeley, California on December 8th to 10th.
  • Poster presentation details are as follows:
    For more information, please visit the conference website .
  • The poster will be available following the conference on the Synlogic website.

Travere Completes Successful pre-NDA Meeting for FILSPARI in IgAN; Provides Regulatory Updates for both IgAN and FSGS

Retrieved on: 
Monday, December 4, 2023
Workforce, Depreciation, Food, Nephrology, Immunoglobulin A, Shanda, FDA, SAN, Therapy, Focal segmental glomerulosclerosis, New Drug Application, Proteinuria, HCU, EGFR, Pharmaceutical industry

SAN DIEGO, Dec. 04, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced the completion of a successful pre-NDA meeting with the U.S. Food and Drug Administration (FDA) for FILSPARI® (sparsentan) in IgA nephropathy (IgAN). The Company will submit a supplemental New Drug Application (sNDA) in the first quarter of 2024 for conversion of the existing U.S. accelerated approval of FILSPARI to full approval. The Company also completed regulatory engagement on focal segmental glomerulosclerosis (FSGS) in which the FDA communicated that the Phase 3 DUPLEX Study results alone are not sufficient to support an sNDA submission for an FSGS indication for sparsentan. As a result, the Company will be conducting additional analyses of FSGS data with plans to re-engage FDA in 2024, and is implementing a strategic reorganization in Q4 2023 to focus near-term resources on the ongoing FILSPARI launch in IgAN and the advancement of pegtibatinase in classical homocystinuria (HCU).

Key Points: 
  • The Company will submit a supplemental New Drug Application (sNDA) in the first quarter of 2024 for conversion of the existing U.S. accelerated approval of FILSPARI to full approval.
  • “Following a successful pre-NDA meeting, we are pleased to be moving forward with our planned sNDA submission for full approval of FILSPARI in IgA nephropathy.
  • “Unfortunately, there is uncertainty around a regulatory path forward for FSGS.
  • Together with $634.6 million in cash and cash equivalents as of September 30, 2023, the Company expects to have a cash runway into 2028.