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Clinical Trial News

AstraZeneca Partners with CSPC for AI-Driven Drug Discovery Platform to Develop Novel Oral Therapies

  • AstraZeneca has entered into a strategic partnership with CSPC to leverage artificial intelligence-driven drug discovery capabilities for accelerated development of novel oral therapeutic candidates.
  • The collaboration targets multiple high-priority therapeutic areas with a specific focus on chronic indications, including preclinical development of small-molecule oral therapies for immunological diseases.
  • This partnership represents AstraZeneca's continued investment in AI-powered drug discovery platforms to enhance its pipeline development capabilities across various therapeutic areas.

Regeneron and Intellia Advance CRISPR Gene Therapy Trial for Hemophilia B Treatment

Rare DiseaseGene Editing
  • Regeneron Pharmaceuticals and Intellia Therapeutics are conducting a two-part clinical study evaluating REGV131-LNP1265, a CRISPR/Cas9-based gene insertion therapy for hemophilia B patients.
  • The experimental therapy aims to enable patients' bodies to naturally produce clotting factor IX, potentially reducing or eliminating the need for regular factor replacement therapy.
  • The ongoing study began in September 2024 and follows a dose escalation approach in adults before expanding to adolescents and children.
  • The gene therapy uses CRISPR/Cas9 technology to insert a functional gene into patients' DNA, representing a potential long-term solution for hemophilia B treatment.

MSBAI Secures $2M DoD Phase III Contract for GURU Generation 2 AI Platform

AI
  • MSBAI has been awarded a $2 million Phase III contract by the U.S. Department of Defense for GURU Generation 2, an AI-powered automation platform for engineering workflows.
  • The GURU platform reduces simulation failure rates from 88% to 2% and cuts setup times from hours or days to minutes for computational fluid dynamics simulations.
  • GURU Generation 2 will scale across key military supercomputers to support over 5,000 defense scientists and engineers in hypersonic vehicle development.
  • The contract elevates MSBAI to 'SBIR unicorn' status and positions the company for further DoD investments in AI-accelerated engineering innovation.

FDA Reviewers Flag "Discordant Results" for Rexulti-Sertraline PTSD Treatment Ahead of Advisory Committee Meeting

Drug Approval
  • FDA reviewers have identified "discordant results" in Otsuka Pharmaceutical's supplemental New Drug Application for brexpiprazole (Rexulti) combined with sertraline for PTSD treatment.
  • The fixed-dose study showed neither 2 mg nor 3 mg doses of Rexulti were superior to sertraline plus placebo, with reviewers calling it "clearly and convincingly a negative study."
  • This development represents another potential setback for PTSD treatment options, following the FDA's rejection of MDMA-assisted psychotherapy in 2024.
  • The FDA advisory committee will discuss these concerns on July 18, 2025, with approximately 13 million Americans currently struggling with PTSD and no new treatments approved in over 20 years.

Brexpiprazole as Combination Therapy With Sertraline in the Treatment of Adults With Post-traumatic Stress Disorder

NCT04174170CompletedPhase 3
Otsuka Pharmaceutical Development & Commercialization, Inc.
Posted 10/30/2019

A Study of Flexible Dose Brexpiprazole as Monotherapy or Combination Therapy in the Treatment of Adults With Post-traumatic Stress Disorder (PTSD)

NCT03033069CompletedPhase 2
Otsuka Pharmaceutical Development & Commercialization, Inc.
Posted 1/26/2017

Brexpiprazole as Combination Therapy With Sertraline in Treatment of Adults With PTSD

NCT04124614CompletedPhase 3
Otsuka Pharmaceutical Development & Commercialization, Inc.
Posted 10/17/2019

Thermo Fisher Scientific Acquires Sanofi's New Jersey Manufacturing Site to Expand U.S. Sterile Drug Production

  • Thermo Fisher Scientific will acquire Sanofi's sterile manufacturing facility in Ridgefield, New Jersey, expanding their strategic partnership to enhance U.S. drug product manufacturing capacity.
  • The acquisition includes a state-of-the-art sterile fill-finish and packaging facility with over 200 employees who will join Thermo Fisher upon transaction completion.
  • The deal strengthens Thermo Fisher's U.S. manufacturing network while ensuring continued production of Sanofi therapies and meeting growing demand from pharmaceutical and biotech customers.
  • The transaction is expected to close in the second half of 2025, with the facility becoming part of Thermo Fisher's Laboratory Products and Biopharma Services segment.

PolyPid Unveils 60-Day GLP-1 Delivery Platform to Transform Diabetes and Weight Management Treatment

  • PolyPid has unveiled a long-acting GLP-1 receptor agonist delivery platform that aims to provide therapeutic drug levels for approximately 60 days with a single subcutaneous administration.
  • The platform releases GLP-1 in a linear fashion, overcoming the burst release issues seen with current weekly injection regimens and potentially eliminating the need for frequent dosing.
  • The technology utilizes PolyPid's proprietary PLEX polymer-lipid matrix system, which has been clinically validated in over 1,000 patients across two Phase 3 trials with no major safety concerns.
  • This development targets the rapidly expanding diabetes and weight management market, which is projected to reach $100 billion by 2030 according to Goldman Sachs and J.P. Morgan Research.

Qualigen Therapeutics Secures Global Patent Protection for Cancer Drug QN-302 Across 25+ Countries

  • Qualigen Therapeutics has been granted new patents covering QN-302, a cancer drug candidate targeting pancreatic and gastrointestinal cancers, across more than 25 countries including over 20 European nations, India, China, and Russia.
  • The patents, titled "Substituted Naphthalene Diimides and Their Use," cover both the drug composition and manufacturing methods, with protection extending into 2040.
  • Company shares surged 20% following the announcement, with trading volume reaching over 44 million shares compared to the average of 23,000 shares.
  • The drug was originally developed by Professor Stephen Neidle and his team at University College London and is exclusively licensed to Qualigen Therapeutics.

MIPLYFFA Shows 5-Year Sustained Efficacy in Rare NPC Disease Extension Study

Rare DiseaseDrug Approval
  • Zevra Therapeutics published long-term data showing MIPLYFFA (arimoclomol) sustained disease progression reduction for at least 5 years in Niemann-Pick disease type C patients.
  • The 48-month open-label extension study included more than 270 patients worldwide, with some receiving treatment for up to seven years.
  • Results demonstrated no new safety concerns and aligned with the pivotal Phase 2/3 trial that showed MIPLYFFA halted disease progression compared to placebo.
  • MIPLYFFA is approved by the FDA for use in combination with miglustat for treating neurological manifestations of NPC in patients 2 years and older.

Voyager Therapeutics Advances Novel APOE4 Gene Therapy for Alzheimer's Disease Using IV-Delivered TRACER Platform

  • Voyager Therapeutics has launched a new gene therapy program targeting APOE4, the strongest genetic risk factor for Alzheimer's disease, using its proprietary TRACER capsid technology for intravenous delivery.
  • The bifunctional therapy simultaneously reduces harmful APOE4 expression while delivering protective APOE2 variant, achieving 90% reduction of APOE4 in key brain regions in preclinical studies.
  • The company's expanded Alzheimer's franchise now includes four wholly-owned assets targeting tau, amyloid, and APOE pathways, with first human data expected in late 2025.
  • Despite promising preclinical results, the stock has declined 67% year-to-date as investors weigh execution risks against the potential for breakthrough treatment in a market projected to exceed $50 billion by 2030.

Cystic Fibrosis Foundation Commits Additional $24 Million to Prime Medicine for Gene Editing Therapy Development

Gene Editing
  • The Cystic Fibrosis Foundation has committed up to $24 million in additional funding to Prime Medicine to advance prime editing gene therapy for cystic fibrosis patients.
  • The funding will focus on developing a prime editing therapy targeting the G542X nonsense mutation, one of the most prevalent CF-causing mutations with no available treatments.
  • Prime Medicine's prime editing technology has the potential to permanently correct CFTR gene mutations and could eventually treat the vast majority of people with cystic fibrosis.
  • The company is investigating multiple approaches including hotspot editing and PASSIGE technology to address various CF mutations through a single platform.

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