Clinical Trial News
Final 5-Year Results of the Phase 2 PACE Trial for CML and Ph+ ALL Patients
The PACE trial's final 5-year results demonstrate ponatinib's efficacy and safety in treating chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) patients who have undergone prior therapies. The study highlights significant response rates, durability of responses, and the impact of dose adjustments on maintaining efficacy while managing adverse events.
Highlighted Clinical Trials:
NICE Rejects Cenegermin for Rare Eye Disease, Citing Cost Concerns
• NICE has issued a draft rejection for Dompé's cenegermin, a novel eye drop therapy for neurotrophic keratitis, deeming its £14,500 per course cost exceeding NHS value thresholds.
• Clinical trials showed promising results with low recurrence rates of 0-5% at 56 weeks in the European REPARO study, though NICE noted concerns about long-term efficacy data.
• Without access to cenegermin, patients may need to resort to tarsorrhaphy, a disfiguring procedure where eyelids are partially sewn together to protect the cornea.
Late-Stage Clinical Trials Advance with Oxabact, Imeglimin, and Favipiravir
• Oxabact initiates a Phase III trial, marking a significant step in its development for treating bacterial infections, potentially addressing unmet needs in antibiotic therapy.
• Imeglimin progresses to Phase III, offering a novel mechanism of action for type 2 diabetes management and a potential alternative for patients inadequately controlled by current treatments.
• Favipiravir enters Phase III trials, aiming to provide an effective antiviral option for managing viral infections and potentially mitigating severe outcomes.
FDA Grants Breakthrough Device Status to 3i Diagnostics' Rapid Blood Infection Test
• 3i Diagnostics' Biospectrix technology receives FDA Breakthrough Device Designation for its ability to identify bacteria directly from blood samples in under 60 minutes, compared to traditional 2-6 day culture methods.
• The rapid diagnostic tool could significantly improve patient outcomes by enabling faster, targeted antibiotic treatment decisions, particularly crucial for preventing sepsis-related deaths.
• The technology addresses a critical healthcare need, with potential to help reduce the 44,000 annual sepsis deaths in the UK alone, of which 14,000 are considered preventable.
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.
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.