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EMA Initiates Review of Sodium Oxybate for Alcohol Dependence Following Efficacy and Safety Concerns

  • The European Medicines Agency has launched a comprehensive review of sodium oxybate medicines used to treat alcohol withdrawal syndrome and support long-term abstinence following concerns raised by French regulators.
  • The review was triggered by effectiveness concerns based on data from three studies and potential abuse risks due to the drug's psychoactive properties affecting the brain.
  • Sodium oxybate works by targeting GABA receptors in the brain and spinal cord, providing a calming effect similar to alcohol's mechanism of action.
  • The Committee for Medicinal Products for Human Use will assess the overall benefit-risk balance and review current risk mitigation measures before making regulatory recommendations.

PharmAla Files C$50 Million Shelf Prospectus to Advance MDMA Drug Development

  • PharmAla Biotech filed a preliminary base shelf prospectus enabling the company to raise up to C$50 million over 25 months to support growth strategy and novel MDXX molecule development.
  • The biotechnology company is uniquely positioned as the only provider of clinical-grade MDMA for patient treatments outside of clinical trials, addressing global supply shortages.
  • PharmAla's research pipeline includes lead drug candidate ALA-002 and focuses on developing novel drugs in the MDXX class, including its proprietary LaNeo™ MDMA formulation.
  • The shelf prospectus provides flexibility for various financing options including at-the-market distributions, though the company has no immediate plans for securities issuance.

GLP-1 Diabetes Drug Liraglutide Reduces Migraine Days by Half in Pilot Study

  • A 12-week pilot study of 26 adults with obesity and chronic migraines found that liraglutide, a GLP-1 diabetes drug, reduced average monthly headache days from 20 to approximately 9.
  • Nearly half of patients experienced at least a 50% reduction in headache days, with migraine disability scores cut by more than half on standard impact scales.
  • Researchers hypothesize the benefit comes from lowering cerebrospinal fluid pressure in the brain rather than weight loss, as participants lost only minimal weight during the study.
  • The findings suggest GLP-1 drugs may target alternative pathways for migraine treatment, offering hope for patients with unmet medical needs beyond current therapies.

Regeneron Expands Patent Protection for Aflibercept with New Formulation Patent Against Amgen

  • Regeneron filed a new patent infringement complaint against Amgen on June 17, 2025, asserting U.S. Patent No. 12,331,099 covering a stable liquid ophthalmic formulation without buffer requirements.
  • The Patent Trial and Appeal Board denied inter partes review petitions from Samsung Bioepis and Formycon challenging Regeneron's earlier aflibercept patent, citing substantial investment in parallel district court proceedings.
  • Amgen's biosimilar PAVBLU launched commercially in November 2024 after the Federal Circuit denied Regeneron's preliminary injunction motion on a separate patent covering buffered formulations.

GH Research Submits IND Hold Response to FDA for Depression Treatment GH001 Ahead of Schedule

  • GH Research PLC submitted its complete response to the FDA's clinical hold on GH001 ahead of schedule in June 2025, addressing regulatory concerns with comprehensive data and completed toxicology studies.
  • The company's lead candidate GH001 is formulated for mebufotenin administration via proprietary inhalation for treatment-resistant depression (TRD).
  • Phase 2b trial results showed GH001 met its primary endpoint with a statistically significant MADRS reduction of -15.5 points compared to placebo on Day 8 (p<0.0001).
  • The company believes GH001 has potential to transform current TRD treatment approaches based on observed clinical activity.

Cereno Scientific Secures 100 MSEK Financing to Advance HDAC Inhibitor Pipeline for Rare Cardiovascular and Pulmonary Diseases

Rare Disease
  • Cereno Scientific secured 100 MSEK through loan financing agreements with existing and new investors to advance its HDAC inhibitor portfolio targeting rare cardiovascular and pulmonary diseases.
  • The funding will support Phase IIb trial initiation for CS1 in pulmonary arterial hypertension and Phase II development for CS014 in idiopathic pulmonary fibrosis.
  • The company's lead candidate CS1 demonstrated favorable safety profile and positive clinical efficacy parameters in a completed Phase IIa trial for PAH patients.
  • The financing structure includes a new 25 MSEK loan, 50 MSEK advance payment, and 25 MSEK convertible conversion, providing runway to reach key clinical milestones.

Terumo BCT Unveils Automated CAR-T Manufacturing Platform Delivering 12 Billion Cells in 7-8 Days

CAR-T
  • Terumo BCT has developed a new automated workflow that consolidates T cell activation, viral transduction, and expansion into a single platform, streamlining CAR-T manufacturing significantly.
  • The protocol demonstrates the ability to produce over 12 billion CAR-T cells in just 7 to 8 days with doubled transduction efficiency compared to manual methods.
  • The integrated 3-in-1 approach uses serum-free culture conditions and closed-system design to reduce contamination risks while supporting generation of crucial T cell subtypes including memory cells.
  • The workflow is currently under evaluation by cell therapy developers and is expected to shorten production timelines, lower costs, and expand patient access to CAR-T treatments.

Lomond Therapeutics Receives FDA Clearance for Phase 1 Trial of Lonitoclax in Relapsed/Refractory AML

Targeted Therapy
  • Lomond Therapeutics announced FDA clearance of its IND application for lonitoclax, a selective BCL-2 inhibitor designed to address safety limitations of venetoclax.
  • The Phase 1 multicenter study will evaluate lonitoclax in combination with azacitidine in up to 60 patients with relapsed/refractory acute myeloid leukemia.
  • Preclinical data demonstrate lonitoclax has minimal immunosuppressive activity and improved safety profile compared to venetoclax, with synergistic activity when combined with other AML therapies.
  • The trial is planned to initiate in the third quarter of 2025 across multiple investigative sites, marking the company's third U.S. IND clearance.

Bio-Thera Solutions Receives Positive CHMP Opinion for Ustekinumab Biosimilar USYMRO

Monoclonal Antibody
  • The European Medicines Agency's Committee for Medicinal Products for Human Use adopted a positive opinion for USYMRO (ustekinumab), Bio-Thera Solutions' biosimilar referencing Stelara.
  • The positive recommendation was based on comprehensive analytical, non-clinical and clinical data demonstrating biosimilarity through Phase 1 and Phase 3 studies in healthy volunteers and severe plaque psoriasis patients.
  • Bio-Thera partnered with Gedeon Richter in October 2024 for commercialization rights in the EU, UK and Switzerland following potential European Commission approval.
  • USYMRO targets IL-12 and IL-23 pathways involved in chronic inflammatory conditions including psoriasis, psoriatic arthritis, Crohn's disease and ulcerative colitis.

Moleculin Biotech Raises $5.9 Million to Advance Annamycin Through Phase 3 AML Trial

Oncology
  • Moleculin Biotech completed a $5.9 million public offering to fund advancement of its lead drug candidate Annamycin through clinical development.
  • The company is conducting the MIRACLE Phase 3 trial evaluating Annamycin combined with cytarabine for relapsed or refractory acute myeloid leukemia.
  • Annamycin is designed as a next-generation anthracycline that avoids multidrug resistance and lacks the cardiotoxicity of current anthracyclines.
  • The funding will also support development of the company's broader pipeline including WP1066 for brain tumors and WP1122 for viral infections.

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