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Clinical Trial News

Relaxed Trial Criteria Show Promise for AML and MDS Patients

  • A clinical trial demonstrated the feasibility of including acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) patients with comorbidities typically excluded from standard trials.
  • Azacitidine, with or without vorinostat, showed acceptable toxicity and clinical benefit in patients with organ dysfunction or poor performance status.
  • The study suggests that relaxing exclusion criteria could broaden clinical trial access and offer potential therapies to a wider range of patients with dismal prognoses.
  • Findings indicate that stringent exclusion criteria may protect the drug being studied rather than the patients, limiting the extrapolation of trial outcomes to those with unfavorable clinical features.

Oncolytic Viruses Gain Momentum in Cancer Treatment Following Merck's $394M Viralytics Acquisition

  • Merck & Co's $394 million acquisition of Viralytics signals growing interest in oncolytic viruses as potential enhancers for cancer immunotherapy treatments.
  • With 10 oncolytic viruses in clinical development and 40 in labs, these agents show promise in 'priming' tumors for checkpoint inhibitor therapy, potentially improving response rates.
  • Targovax emerges as a leading independent biotech in the oncolytic virus space, developing TG01 for pancreatic cancer and ONCOS-102 for mesothelioma in partnership with AstraZeneca's MedImmune.

AbbVie's Elagolix Demonstrates Significant Efficacy in Two Phase 3 Trials for Uterine Fibroids

  • AbbVie's investigational drug elagolix met primary endpoints in two pivotal Phase 3 studies, achieving clinical response rates of 68.5% and 76.2% compared to placebo rates of 8.7% and 10.1% respectively in women with uterine fibroids.
  • The oral GnRH receptor antagonist, combined with low-dose hormone add-back therapy, significantly reduced heavy menstrual bleeding as measured by the alkaline hematin method in premenopausal women aged 18-51.
  • Both ELARIS UF-I and UF-II studies met all ranked secondary endpoints, with safety profiles consistent with previous Phase 2 studies, though hypoestrogenic effects including hot flashes and bone mineral density reduction were observed.
  • The positive results support regulatory submissions for elagolix as a potential non-surgical treatment option for the millions of women affected by uterine fibroids, which impact up to 80% of women by age 50.

AI, Blockchain, and Big Data: New Technologies Promise to Slash Pharmaceutical R&D Costs

  • Pharmaceutical R&D currently consumes 20% of industry revenues, with each new drug development costing up to £1.2 billion and facing a mere 4.1% success rate.
  • Third-wave AI technologies are revolutionizing drug discovery by enabling faster analysis of complex datasets and more efficient clinical trial processes, with major companies like Johnson & Johnson and Merck & Co already investing.
  • Blockchain technology and big data analytics are improving clinical trial transparency and drug safety monitoring, potentially reducing development costs and improving success rates.

Former Eli Lilly Executive Alex Azar Sworn in as US Health Secretary, Pledges to Combat High Drug Prices

  • President Trump has appointed Alex Azar, former Eli Lilly US operations head, as the new Health and Human Services Secretary, prioritizing drug pricing reform and opioid crisis management.
  • Despite concerns over potential industry conflicts, Azar brings unique experience combining private sector pharmaceutical leadership with previous HHS service during the Bush administration.
  • Azar has committed to addressing prescription drug costs, with potential focus on value-based purchasing and Medicare reform, while Trump emphasizes tackling price disparities between US and other countries.

Clinical Trial Outsourcing Set to Reach 72% by 2020 as Industry Embraces CRO Partnerships

  • The pharmaceutical industry is witnessing a dramatic shift towards outsourcing clinical trials, with projections showing an increase from 23% in 2012 to 72% by 2020.
  • Strategic partnerships between sponsors and Contract Research Organizations (CROs) demonstrate potential for reducing costs, improving trial efficiency, and enhancing data quality.
  • Successful collaboration between sponsors and CROs requires transparent communication, shared decision-making, and aligned performance metrics to overcome operational challenges.

Roche's Ocrevus to Match Novartis's Gilenya Pricing in UK MS Market

  • Roche announces pricing strategy for Ocrevus in the UK multiple sclerosis market, aligning costs with Novartis's Gilenya at approximately £19,169 per patient annually.
  • Ocrevus becomes the first approved treatment for primary progressive MS (PPMS), though NICE approval for this indication may face longer review timelines.
  • The drug has demonstrated strong efficacy in relapsing MS with no direct links to PML, positioning it favorably against competitors like Biogen's Tysabri and Tecfidera.

Janssen to Address Key Challenges in Drug-Device Combination Product Development at Pre-Filled Syringes Conference

  • Janssen's Head of Combination Products Center of Excellence, Susan Neadle, will deliver a keynote presentation on drug-device combination product development challenges at the Pre-Filled Syringes East Coast Conference in Boston.
  • The conference will feature critical discussions on manufacturing processes, including high-concentration monoclonal antibody formulation filling and technical updates from industry leaders GSK and ZEON.
  • Interactive workshops focusing on design considerations and human factors for connected drug delivery systems will be led by experts from Battelle and Cambridge Consultants Ltd.

FDA Grants Breakthrough Therapy Designation to Lenvima-Keytruda Combination for Advanced Renal Cell Carcinoma

  • The FDA has granted Breakthrough Therapy Designation to Eisai's Lenvima (lenvatinib) combined with Merck's Keytruda (pembrolizumab) for treating patients with advanced and/or metastatic renal cell carcinoma.
  • The designation was based on results from Study 111, a multicenter Phase 1b/2 clinical trial evaluating the combination therapy in selected solid tumors including renal cell carcinoma.
  • This marks the second Breakthrough Therapy Designation for Lenvima and the twelfth for Keytruda, providing expedited development pathways and regulatory benefits.
  • The combination therapy is being jointly developed by Eisai and Merck as part of a broader strategic collaboration worth up to $5.76 billion.

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