SIMD

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

Retrieved on: 
Thursday, April 4, 2024

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

Retrieved on: 
Tuesday, March 5, 2024

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

Retrieved on: 
Monday, January 8, 2024

“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

Retrieved on: 
Thursday, January 4, 2024

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

Retrieved on: 
Thursday, May 11, 2023

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.

Acer Therapeutics Announces OLPRUVA™ Commercial Launch Progressing Ahead of Schedule

Retrieved on: 
Monday, May 1, 2023

NEWTON, Mass., May 01, 2023 (GLOBE NEWSWIRE) -- Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today announced an update on progress in support of its commercial launch of OLPRUVA™ (sodium phenylbutyrate) for oral suspension, including drug availability now expected earlier than anticipated in mid-June 2023, subject to additional capital.

Key Points: 
  • “As a result of these efforts, I am pleased to report we are ahead of our anticipated launch schedule and now expect drug availability beginning in mid-June 2023.
  • We have also made significant progress in our ongoing discussions with payers regarding reimbursement, increased physician awareness and interest, and built out our patient support and fulfillment program.
  • Acer expects to publish OLPRUVA™’s list price, or wholesale acquisition cost (WAC), in mid-to-late May.
  • Acer has also made significant progress in support of its objective to raise awareness for OLPRUVA™ as a new, alternative treatment option for certain UCD patients.

Acer Therapeutics Reports Q4 and Full Year 2022 Financial Results and Provides Corporate Update

Retrieved on: 
Monday, March 27, 2023

OLPRUVA™ is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment.

Key Points: 
  • OLPRUVA™ is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment.
  • As a result, Acer is pausing the ACER-801 program until Acer has conducted a thorough review of the full data set
    Ended Q4 2022 with $2.3 million in cash and cash equivalents.
  • Cash and cash equivalents were $2.3 million as of December 31, 2022, compared to $12.7 million as of December 31, 2021.
  • For additional information, please see Acer’s Annual Report on Form 10-K filed today with the SEC.

Acer Therapeutics Announces Data Presented at Society for Inherited Metabolic Disorders Annual Meeting Identifying Preferred Urea Cycle Disorder Treatment Attributes

Retrieved on: 
Wednesday, March 22, 2023

The results of the survey showed that taste and odor were the most important attributes for both prescribing and for patient adherence and compliance .

Key Points: 
  • The results of the survey showed that taste and odor were the most important attributes for both prescribing and for patient adherence and compliance .
  • “OLPRUVA™5 leverages the well-established efficacy of sodium phenylbutyrate in an innovative dual-coating formulation designed for palatability,” said Adrian Quartel, MD, FFPM, CMO of Acer.
  • ACER-001 for the treatment of certain UCDs is now marketed in the U.S. as OLPRUVA™ (sodium phenylbutyrate) following FDA approval in December 2022.
  • New FDA-Approved UCD Treatment Option: OLPRUVA™
    ACER-001 (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and is marketed under the brand name, OLPRUVA™.

Cyclo Therapeutics Provides Business Update and Reports Full Year 2022 Financial Results

Retrieved on: 
Monday, March 20, 2023

Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with diseases, today reported its financial results for the full year 2022 and provided a business update.

Key Points: 
  • Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with diseases, today reported its financial results for the full year 2022 and provided a business update.
  • Further we continue to have encouraging strategic discussions across the industry,” commented N. Scott Fine, CEO of Cyclo Therapeutics.
  • Cyclo Therapeutics received Orphan Drug Designation for Trappsol® Cyclo™ to treat NPC1 in both the U.S. and EU and Fast Track and Rare Pediatric Disease Designations in the U.S.
  • Enrolled patients will be randomized across three study arms: 500 mg/kg or 1000 mg/kg of Trappsol® Cyclo™ and placebo.

Synlogic Announces Data Presentations at the Society for Inherited Metabolic Disorders (SIMD) 44th Annual Meeting

Retrieved on: 
Monday, March 20, 2023

In separate poster presentations, the company also presented clinical data and preclinical data related to its homocystinuria (HCU) program.

Key Points: 
  • In separate poster presentations, the company also presented clinical data and preclinical data related to its homocystinuria (HCU) program.
  • “We were delighted to review these encouraging findings from our two rare metabolic disease programs among the expert metabolic clinicians who attend the SIMD meeting,” said Dave Hava, Chief Scientific Officer and Head of Research and Development at Synlogic.
  • Clinical data presented included positive data from the Phase 1 study evaluating SYNB1353 in healthy volunteers using a dietary model of HCU.
  • Posters presented at the SIMD meeting are posted on the Publications page of the Synlogic website.