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Clinical Trial News

Kazia Therapeutics Secures $2 Million Private Placement to Advance Brain Cancer and Breast Cancer Drug Development

  • Kazia Therapeutics raised $2 million through a private placement priced at a 5% premium to market, demonstrating strong investor confidence in the company's oncology pipeline.
  • The funding will support continued clinical development of paxalisib, a brain-penetrant PI3K/mTOR inhibitor in trials for brain cancer and advanced triple-negative breast cancer.
  • The company is also advancing EVT801, a selective VEGFR3 inhibitor currently in Phase 1 trials for advanced solid tumors.

FDA Mandates Comprehensive Safety Label Changes for All Opioid Pain Medications

  • The FDA announced on July 31, 2025, that all opioid pain medications must update their safety labels to emphasize and explain the serious risks associated with long-term use, including addiction, misuse, and overdose.
  • The decision was based on data from two large FDA-required observational studies (PMR 3033-1 and 3033-2) that revealed serious side effects from long-term opioid use and identified a lack of adequate research on long-term effectiveness.
  • Drug manufacturers have 30 days to submit revised labels for FDA review, and the agency is also mandating new randomized controlled clinical trials to examine long-term opioid benefits and risks.
  • The labeling changes include clearer risk information, stronger dosage warnings, removal of language that could support indefinite use, and enhanced warnings about drug combinations and overdose reversal agents.

Sanofi Acquires Rights to Plozasiran for $395 Million in Greater China Deal with Arrowhead

Rare Disease
  • Sanofi has acquired rights to develop and commercialize plozasiran, a first-in-class RNAi therapeutic for familial chylomicronemia syndrome and severe hypertriglyceridemia, in Greater China for $130 million upfront plus up to $265 million in milestones.
  • Visirna Therapeutics completed a successful Phase 3 trial of plozasiran in Chinese FCS patients, meeting all primary and secondary endpoints, and submitted a New Drug Application to China's NMPA in January 2025.
  • Plozasiran targets apolipoprotein C-III (APOC3) to reduce triglyceride levels and has received Breakthrough Therapy Designation and Priority Review status from China's regulatory authority.
  • The drug is being investigated across multiple indications in the SUMMIT clinical program, including studies in severe hypertriglyceridemia and mixed hyperlipidemia beyond FCS.

A Phase 3 Study of VSA001 in Chinese Adults with Familial Chylomicronemia Syndrome

NCT05902598Active, Not RecruitingPhase 3
Visirna Therapeutics HK Limited
Posted 7/10/2023

Large Real-World Study Questions Effectiveness of Immunoglobulin Replacement Therapy in CLL Patients

Real World Evidence
  • A retrospective analysis of 6,217 CLL patients in Australia found that regular immunoglobulin replacement therapy was not associated with reduced risk of serious infections requiring hospitalization.
  • Despite increasing use of immunoglobulin therapy over the 14-year study period, serious infection rates doubled from 1.9% to 3.9%, with higher infection frequency during treatment periods compared to off-treatment intervals.
  • The study revealed that 45.9% of patients who started immunoglobulin therapy died during follow-up, with median survival of approximately six years from first treatment.
  • Researchers call for better clinical guidelines given the therapy's high cost, patient burden, and limited evidence of effectiveness in preventing serious infections.

DPIIT and Roche India Sign Strategic Partnership to Accelerate Healthcare Innovation in Critical Therapeutic Areas

  • The Department for Promotion of Industry and Internal Trade (DPIIT) and Roche Products (India) Pvt. Ltd. signed a Memorandum of Understanding to support DPIIT-recognized startups in oncology, neurology, ophthalmology, haematology, and rare diseases.
  • Under the partnership, Roche India will provide mentorship from global experts, support pilot and validation studies, and offer access to cutting-edge technologies and international platforms.
  • The collaboration emphasizes capacity-building in Health Economics and Outcomes Research (HEOR), Good Clinical Practices (GCP), and patient engagement to accelerate innovation-to-market deployment.
  • This public-private partnership aims to position India as a global hub for health innovation while ensuring patient-centric solutions tailored to India's healthcare needs.

Knight Therapeutics Submits MINJUVI Supplemental Application for Follicular Lymphoma in Brazil

Monoclonal AntibodyOncology
  • Knight Therapeutics' Brazilian affiliate has submitted a supplemental application to ANVISA seeking approval for MINJUVI (tafasitamab) in combination with rituximab and lenalidomide for previously treated follicular lymphoma patients.
  • The submission is based on the pivotal Phase 3 inMIND trial results, which demonstrated a 57% reduction in disease progression risk with median progression-free survival of 22.4 months versus 13.9 months for placebo.
  • Follicular lymphoma represents 20-25% of adult non-Hodgkin lymphoma cases in Brazil, with limited treatment options available for relapsed or refractory disease.
  • The application was selected for review under Project Orbis, with Knight planning additional regulatory submissions across Latin America in the coming months.

A Phase 3 Study to Assess Efficacy and Safety of Tafasitamab Plus Lenalidomide and Rituximab Compared to Placebo Plus Lenalidomide and Rituximab in Patients With Relapsed/Refractory (R/R) Follicular Lymphoma or Marginal Zone Lymphoma.

NCT04680052Active, Not RecruitingPhase 3
Incyte Corporation
Posted 4/15/2021

Certara Appoints AI Pioneer Dr. Chris Bouton as CTO to Advance Next-Generation Drug Development Platform

AI
  • Certara has appointed Dr. Christopher Bouton, founder of Vyasa Analytics, as Chief Technology Officer to lead development of an AI-powered model-informed drug development platform.
  • Dr. Bouton will spearhead technology strategy for integrating generative AI and biosimulation technology to accelerate drug discovery and development timelines.
  • The appointment strengthens Certara's position in the competitive biosimulation market, building on Bouton's track record of developing AI solutions including CoAuthor™ for regulatory writing.
  • Certara's next-generation platform aims to increase efficiency in discovering novel therapeutics by combining scientific expertise with advanced AI approaches.

Zenara Pharma Secures First Generic Sertraline Capsule Approval with 180-Day Market Exclusivity

Drug Approval
  • Zenara Pharma received FDA approval for the first generic version of Sertraline Hydrochloride Capsules in 150 mg and 200 mg strengths, marking a significant milestone in psychiatric medication accessibility.
  • The approval includes Competitive Generic Therapy designation, granting Zenara 180 days of exclusive marketing rights in the U.S. market.
  • The generic sertraline capsules are indicated for treating Major Depressive Disorder in adults and Obsessive-Compulsive Disorder in both adults and pediatric patients aged six years and older.
  • According to IQVIA sales data, the reference product generated approximately $35.5 million in U.S. sales for the 12-month period ending June 2025.

ChatGPT Reduces Clinical Trial Screening Time from 40 Minutes to Under 3 Minutes, But Human Oversight Still Required

AIOncology
  • ChatGPT-4 demonstrated superior performance over ChatGPT-3.5 in screening patients for clinical trials, achieving 84% accuracy with better sensitivity and specificity balance.
  • Screening times were dramatically reduced from over 40 minutes per patient to 1.4-3.0 minutes with GPT-3.5 and 7.9-12.4 minutes with GPT-4, though costs ranged from $0.02-$0.27 per patient.
  • Both AI models showed high specificity but low sensitivity in identifying eligible patients, with GPT-4 having only 16% median sensitivity despite 100% specificity.
  • Researchers concluded that large language models should complement rather than replace manual chart reviews due to difficulties in identifying patients who meet all eligibility criteria.

Breast Cancer Treatment De-escalation Advances Through Multi-institutional Clinical Trials

  • Multiple large-scale clinical trials are demonstrating that breast cancer treatment can be safely reduced without compromising outcomes, including shortened radiation schedules and elimination of radiation in select patients.
  • The I-SPY trial has enrolled over 4,000 patients since 2012, testing novel agents in the neoadjuvant setting that may achieve higher complete remission rates with less toxicity than standard chemotherapy.
  • Coordinated multidisciplinary care is essential for successful trial enrollment and treatment de-escalation, requiring precise timing and protocol adherence across surgical, medical, and radiation oncology teams.
  • Genomic profiling tools like Oncotype DX are enabling personalized treatment decisions, particularly for intermediate-risk ER-positive breast cancer patients where chemotherapy benefit remains uncertain.

I-SPY TRIAL: Neoadjuvant and Personalized Adaptive Novel Agents to Treat Breast Cancer

NCT01042379RecruitingPhase 2
QuantumLeap Healthcare Collaborative
Posted 3/1/2010

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