SIMD

Travere Therapeutics Reports First Quarter 2024 Financial Results

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월요일, 5월 6, 2024
FDA, Total, Risk, Ligand Pharmaceuticals, European Commission, Research, UPCR, Webcast, HCU, IND, EGFR, PDUFA, SIMD, Civil service commission, Diagnosis, PMDA, GAAP, CMA, Congress, Table, PROTECT, Priority review, Food, Abstract, FSGS, Income tax, CSL Vifor, Quarter, Medication, Therapy, SAN, Plasma, Urine, International, Safety, Proteinuria, ANNA, Immunoglobulin A, Homocysteine, Investigational New Drug, Society, Shanda, New Drug Application, Genetic, European, Disease, Patient, Quality of life (healthcare), IPR, Bank statement, Growth, Pharmaceutical industry

Net product sales for the first quarter of 2024 were $40.0 million, compared to $24.2 million for the same period in 2023.

Key Points: 
  • Net product sales for the first quarter of 2024 were $40.0 million, compared to $24.2 million for the same period in 2023.
  • Travere Therapeutics will host a conference call and webcast today, Monday, May 6, 2024, at 4:30 p.m.
  • ET to discuss company updates as well as first quarter 2024 financial results.
  • The Company believes that these non-GAAP financial measures are helpful in understanding its past financial performance and potential future results.

Arcturus Therapeutics Announces First Quarter 2024 Financial Update and Pipeline Progress

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수요일, 5월 8, 2024
Biotechnology, Infectious Diseases, COVID-19, Health, Pharmaceutical, Society, COVID, Investment, Meiji, ARCT, Ornithine transcarbamylase deficiency, Safety, SARS-CoV-2, Travel, European Commission, Research, OTC, Cystic fibrosis, Lyme disease, RNA, MAA, Inborn errors of metabolism, Trial of the century, Vaccine, CDMO, Omicron, SRR, GMT, SAD, Liver, CSL, EMA, JPMorgan Chase, SIMD, Civil service commission, Infection, Messenger, Arcturus Therapeutics, Therapy, European Medicines Agency, Pharmaceutical industry

“Arcturus continues to make encouraging progress in both our vaccine and therapeutics pipeline,” said Joseph Payne, President & CEO of Arcturus.

Key Points: 
  • “Arcturus continues to make encouraging progress in both our vaccine and therapeutics pipeline,” said Joseph Payne, President & CEO of Arcturus.
  • To support this effort, Arcturus along with CDMO partners are on track to deliver the initial 4 million commercial doses of Kostaive in Q3.
  • The Company will share a progress update on the Phase 2 study on July 1, 2024.
  • The expected cash runway extends at least three years based on the current pipeline and programs through the first quarter of fiscal year 2027.

Jaya Biosciences Presents Updated Preclinical Data in Alzheimer’s Disease at the 45th SIMD Annual Meeting

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화요일, 4월 16, 2024
GUSB, Society, PPT1, IDUA, Haploinsufficiency, Gene dosage, Central Waqf Council, Animal, GALC, Research, AAV, IND, CNS, Disease, Science, NAGLU, Patient, DSM-IV codes, SIMD, Toxicology, Annual general meeting, Plaque, SAN, Gene, APP, Antibiotics

During a podium presentation, Jaya Biosciences’ scientific founder and science advisory board chair, Professor Mark Sands, reported on recently updated human genetic analyses suggesting that heterozygous loss-of-function mutations in lysosomal enzyme genes are enriched in Alzheimer’s disease (AD) patients.

Key Points: 
  • During a podium presentation, Jaya Biosciences’ scientific founder and science advisory board chair, Professor Mark Sands, reported on recently updated human genetic analyses suggesting that heterozygous loss-of-function mutations in lysosomal enzyme genes are enriched in Alzheimer’s disease (AD) patients.
  • This new analysis generated from a much larger whole genome sequence database confirmed their previous human genetic findings from a smaller whole exome database.
  • “The updated human genetic data confirmed that heterozygous deleterious mutations in a subset of lysosomal genes are enriched in patients with Alzheimer’s disease,” said Prof. Mark Sands.
  • For more information about the 45th SIMD Annual Meeting, please go to SIMD2024 Meeting .

Zevra Therapeutics Presents New Data on the Long-Term Safety and Efficacy of Arimoclomol as a Treatment for Niemann-Pick Disease Type C at the SIMD 45th Annual Meeting

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월요일, 4월 15, 2024
FDA, Society, Niemann–Pick disease, EAP, American Academy, Patient, Assistant professor, NDA, MD, Deficit spending, Dell Medical School, Disease, PDUFA, University, NPC, SIMD, Pediatrics, Diagnosis, Assistant, Annual general meeting, Therapy, Neuroscience, Pharmaceutical industry

CELEBRATION, Fla., April 15, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that new long-term real-world data from the arimoclomol expanded access program (EAP: NCT04316637) for the treatment of Niemann-Pick disease type C (NPC) was featured in an oral presentation during the Society for Inherited Metabolic Disorders (SIMD) 45th Annual Meeting taking place at the Sheraton/Le Meridien Charlotte Hotel Complex in Charlotte, NC, from April 14-17, 2024.

Key Points: 
  • “NPC presents a therapeutic challenge, lacking approved treatment options in the U.S. and invariably leading to progressive loss of independence due to physical and cognitive impairments.
  • Among these 26 adults, mean (standard deviation, SD) age at NPC diagnosis and arimoclomol initiation was 23.7 (9.0) and 28.5 (6.5) years, respectively.
  • Patients continued arimoclomol treatment for a mean of 21 months (range: 12-32) and 69% (18/26) had recorded miglustat use.
  • Dr. Patterson’s talk was entitled “Evaluation of the Long-Term Effect of Arimoclomol in NPC - 48 Months Data from CT-ORZY-NPC-002.”

Elastic Reports 8x Speed and 32x Efficiency Gains for Elasticsearch and Lucene Vector Database

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금요일, 4월 26, 2024
Professional Services, Data Management, Technology, Data Analytics, Software, Artificial Intelligence, SIMD, RAG, Memory, 32X, Seamless, Java, ESTC, AI, Online shopping

Elastic (NYSE: ESTC), the company behind Elasticsearch®, today announced new vector database performance gains with Elasticsearch and Apache Lucene, with up to 8x speed and 32x efficiency.

Key Points: 
  • Elastic (NYSE: ESTC), the company behind Elasticsearch®, today announced new vector database performance gains with Elasticsearch and Apache Lucene, with up to 8x speed and 32x efficiency.
  • These advancements provide developers with the most flexible and open tools needed to keep up with rapid generative AI innovation.
  • “Elastic’s contributions bring multifold performance gains in speed, scale, and efficiency, ensuring the Elasticsearch-Lucene integration continues to be a versatile vector database and search experience loved by developers and trusted by enterprises the world over.”
    Customers are building the next generation of AI enabled search applications with Elastic’s vector database and vector search technology.
  • Roboflow uses Elastic vector database to store and search billions of vector embeddings.

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

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목요일, 4월 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.

MulticoreWare launches VaLVe, a replacement for platform-specific intrinsics in SIMD Programming

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화요일, 3월 5, 2024
SAN, Architecture, Chemical oxygen demand, Computing, MulticoreWare, Valve, AVX, NEON, SIMD, RVV, SVE, Software, CPU, Video game

VaLVe reshapes the landscape of SIMD programming, which has been dominated by solutions with limited portability or tenuous performance.

Key Points: 
  • VaLVe reshapes the landscape of SIMD programming, which has been dominated by solutions with limited portability or tenuous performance.
  • Offering a versatile vector programming layer for application development, VaLVe provides the portability and ease of use of standard C/C++ with the efficiency of platform-specific intrinsics.
  • Performance-minded application developers have long experienced the pains of manually updating applications with legacy SIMD optimizations to a new set of vector extensions.
  • Vinod Kannan, VP & GM, Compute BU at MulticoreWare said,
    "VaLVe embodies our commitment to empowering developers with a dynamic toolset for efficient programming.

Syntiant Achieves 50% Acceleration of Large Language Models for Edge Devices

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월요일, 1월 8, 2024
Car, NLP, Human, M12, LLM, SIMD, CES, Intelligence

“We are focused on making edge AI a reality, bringing low power, highly accurate intelligence to edge devices,” said Kurt Busch, CEO at Syntiant.

Key Points: 
  • “We are focused on making edge AI a reality, bringing low power, highly accurate intelligence to edge devices,” said Kurt Busch, CEO at Syntiant.
  • “Our core model optimizations enable considerable processing time acceleration across a range of compute platforms.
  • Demoed live at CES 2024 in Las Vegas, Syntiant’s optimizations demonstrate an adjacent class of LLMs that run entirely at the edge.
  • Visit www.syntiant.com or contact [email protected] to schedule a demo of the company’s technology being deployed in smart homes, teleconferencing solutions and event detection devices, among other use cases.

Synlogic Provides Corporate Update and Outlook for 2024

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목요일, 1월 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.

Reneo Pharmaceuticals Reports First Quarter 2023 Financial Results

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목요일, 5월 11, 2023
Exercise, DNA, Society, SIMD, G&A, PMM, Trial of the century, Patient, Investment, Annual general meeting, A-DNA, Skeleton, R, Research, Three Months, OLE, Pharmaceutical industry, Nursing, Health insurance, STRIDE

Completed enrollment of the pivotal STRIDE study; topline results anticipated 4Q 2023

Key Points: 
  • Completed enrollment of the pivotal STRIDE study; topline results anticipated 4Q 2023
    IRVINE, Calif., May 11, 2023 (GLOBE NEWSWIRE) -- Reneo Pharmaceuticals, Inc. (Nasdaq: RPHM), a clinical-stage pharmaceutical company focused on the development and commercialization of therapies for patients with rare genetic mitochondrial diseases, today reported financial results for the quarter ended March 31, 2023 and provided a business update.
  • “In the first quarter we achieved a key milestone with the completion of enrollment of the pivotal STRIDE study.
  • Research and development (R&D) expenses were $11.0 million during the first quarter of 2023, compared to $9.3 million for the same period in 2022.
  • General and administrative (G&A) expenses were $5.1 million during the first quarter of 2023, compared to $3.7 million for the same period in 2022.