PhaseV Secures $50M Series A to Transform Clinical Trials with AI Platform
• PhaseV has raised $50 million in Series A funding led by Accel and Insight Partners, bringing its total funding to $65 million to expand its AI-driven clinical development platform.
• The company's vertical AI platform has demonstrated impressive results across 30+ global pharmaceutical clients, including 50% reduction in trial costs, 40% decrease in enrollment size and trial duration, and 30% increase in trial success rates.
• PhaseV's technology spans four core applications supporting end-to-end clinical development, currently used across more than 20 therapeutic areas including neurology, oncology, and rare diseases.
Long-Term Disease Control Observed in NSCLC Patients After Immune Checkpoint Inhibitor Discontinuation
• A multi-institutional study found that NSCLC patients who discontinued immune checkpoint inhibitors due to adverse events still achieved median progression-free survival of 12.7 months and overall survival of 43.7 months.
• Longer treatment duration before discontinuation significantly improved outcomes, with patients treated for more than six months experiencing median overall survival of 86.9 months after stopping therapy.
• High PD-L1 expression, complete or partial response to treatment, and nonsquamous histology were identified as key predictors of prolonged disease control after treatment discontinuation.
Study Reveals Factors Influencing Immune Checkpoint Inhibitor Efficacy in Metastatic Colorectal Cancer
• Cleveland Clinic and Moffitt Cancer Center researchers analyzed data from nearly 19,000 metastatic colorectal cancer patients, confirming immune checkpoint inhibitors significantly improve survival in MSI-H tumors compared to chemotherapy alone.
• The study identified specific factors that may enhance immune checkpoint inhibitor efficacy in traditionally unresponsive MSS tumors, including high albumin levels and recent antibiotic use, potentially expanding treatment options.
• Approximately 12.3% of patients with MSS tumors achieved durable responses to immune checkpoint inhibitors, suggesting the need for further research to identify biomarkers that could predict treatment success in this population.
Merck's Enpatoran Shows Promise for Lupus Rash in Phase 2 Trial Despite Mixed Results
• Merck's oral TLR7/8 inhibitor enpatoran demonstrated clinically meaningful reduction in disease activity for patients with cutaneous lupus erythematosus and systemic lupus erythematosus with active lupus rash in the Phase 2 WILLOW study.
• The drug met its primary endpoint in Cohort A with up to 91.3% of patients achieving CLASI-50 response at Week 24, though it failed to meet the primary endpoint in the systemic lupus erythematosus cohort (Cohort B).
• As a potential first-in-class oral therapy targeting the TLR7/8 pathway, enpatoran could address significant unmet needs for lupus patients, with Merck now in discussions with health authorities regarding a global Phase 3 program.
BRAF Inhibitors Show Promise in Treatment of Relapsed/Refractory Hairy Cell Leukemia
• BRAF V600E mutation, present in 90% of hairy cell leukemia cases, has emerged as a key therapeutic target with BRAF inhibitors showing significant efficacy in relapsed/refractory disease.
• Clinical trials of vemurafenib and dabrafenib, alone or in combination with other agents, demonstrated high response rates with overall response rates ranging from 80-89% in heavily pretreated patients.
• The combination of dabrafenib plus trametinib showed particularly impressive results with a 89.1% overall response rate and 24-month duration of response rate of 97.7%, though managing side effects remains a challenge.
FDA Grants Priority Review to Boehringer's Zongertinib for HER2-Mutant Advanced Lung Cancer
• Boehringer Ingelheim's zongertinib could become the first oral targeted therapy for previously treated HER2-mutant advanced non-small cell lung cancer, with FDA decision expected in Q3 2025.
• Phase Ib trial demonstrated impressive 71% objective response rate in 75 previously treated NSCLC patients, with favorable safety profile and low treatment discontinuation rate.
• The drug addresses a significant unmet need in HER2-mutant NSCLC patients, who currently face poor prognosis with limited treatment options and less than 30% five-year survival rate.
FDA-Approved Fotivda Dose Shows Superior Efficacy in Renal Cell Carcinoma Treatment
• Analysis of three phase 3 trials demonstrates that the FDA-approved 1.34mg dose of Fotivda delivers superior antitumor activity compared to reduced 0.89mg dose in renal cell carcinoma patients.
• Higher Fotivda exposure correlates with improved progression-free survival, with patients in the highest exposure quartile achieving 9.7 months median PFS versus 5.6 months in the lowest quartile.
• Study reveals comparable hypertension risk between doses, while combination therapy with Opdivo at lower Fotivda dose showed no additional benefits over standard monotherapy.
Merck KGaA in Advanced Talks to Acquire SpringWorks Therapeutics; BridgeBio Secures EU Approval for ATTR Drug
• Merck KGaA confirms advanced acquisition discussions with SpringWorks Therapeutics, though critical conditions remain unmet and no binding agreement has been reached.
• BridgeBio Pharma's Beyonttra (acoramidis) receives European Commission approval for transthyretin amyloidosis with cardiomyopathy, triggering a $75 million milestone payment from Bayer.
• Biohaven's troriluzole receives FDA priority review for spinocerebellar ataxia, positioning it to potentially become the first approved treatment for this neurodegenerative disorder.
PADCEV-KEYTRUDA Combination Shows Sustained Survival Benefit in Advanced Urothelial Cancer Trial
• Phase 3 EV-302 trial demonstrates PADCEV plus KEYTRUDA reduces mortality risk by 49% compared to chemotherapy in advanced urothelial cancer patients, with median overall survival of 33.8 months versus 15.9 months.
• The combination therapy showed significant progression-free survival benefit of 12.5 months compared to 6.3 months with chemotherapy, representing a 52% reduction in disease progression risk.
• Extended 12-month follow-up data confirms sustained efficacy across all patient subgroups, including both cisplatin eligible and ineligible patients, with no new safety concerns identified.
Walden Biosciences Strengthens Board with Appointment of Rare Disease Expert Howard Mayer
• Walden Biosciences has appointed Howard Mayer, M.D., former Executive VP of R&D at Ipsen, to its Board of Directors as the company approaches key clinical milestones.
• Dr. Mayer brings extensive experience in clinical development and regulatory affairs, particularly in rare diseases, as Walden prepares for Phase 2 data readout of WAL0921 for glomerular kidney diseases.
• The appointment comes at a strategic time as Walden advances its pipeline, including the progression of WAL0623, a dynamin stabilizer, into clinical trials.
MiNK Therapeutics' Allo-iNKT Cell Therapy Shows Promise in Refractory Gastric Cancer
• MiNK Therapeutics' agenT-797, combined with botensilimab and balstilimab, demonstrates robust immune activation in refractory gastroesophageal cancer.
• The Phase 2 study reveals increased interferon-gamma levels and enhanced T-cell infiltration, suggesting improved clinical outcomes.
• Early administration of agenT-797 alongside checkpoint inhibitors before chemotherapy amplifies immune responses, optimizing T-cell priming.
• The off-the-shelf allogeneic iNKT platform offers a scalable and accessible treatment option for patients with hard-to-treat cancers.
Tolebrutinib Receives FDA Breakthrough Therapy Designation for Non-Relapsing Secondary Progressive Multiple Sclerosis
• The FDA has granted Breakthrough Therapy designation to tolebrutinib for adults with non-relapsing secondary progressive multiple sclerosis (nrSPMS).
• The designation is based on positive results from the HERCULES phase 3 study, which demonstrated a 31% delay in disability progression compared to placebo.
• Tolebrutinib is the first brain-penetrant BTK inhibitor to receive this designation for MS, addressing a critical unmet need in delaying disability progression.
• Regulatory submissions for tolebrutinib are being finalized in the US and EU, with ongoing studies for primary progressive MS anticipated in H2 2025.
Bionxt Solutions to Evaluate Sublingual Cladribine Formulation for Multiple Sclerosis in Clinical Trial
• Bionxt Solutions plans to initiate a clinical trial in the second half of 2025 to assess its sublingual cladribine formulation, BNT23001, for treating multiple sclerosis.
• The trial aims to compare the safety, efficacy, and bioavailability of BNT23001 with the oral formulation Mavenclad in patients with MS.
• BNT23001, a sublingual film, offers a potentially more convenient administration route, especially for patients with swallowing difficulties, and may have a faster onset of action.
• Bionxt is also pursuing patent protection for its sublingual thin-film formulation in key regions, including Europe, the U.S., and Canada.
FDA Approves Companion Diagnostic for Tepotinib in mNSCLC with MET Exon 14 Skipping Alterations
• The FDA approved FoundationOne Liquid CDx as a companion diagnostic for tepotinib, aiding in identifying mNSCLC patients with MET exon 14 skipping alterations.
• Tepotinib received regular approval in February 2024 for mNSCLC patients with MET exon 14 skipping alterations, following an earlier accelerated approval in 2021.
• Phase 2 VISION trial data supported tepotinib's approval, demonstrating overall response rates of 57% in treatment-naive and 45% in previously treated patients.
Standard Chemoradiation Shows Superior Outcomes in HPV-Associated Oropharyngeal Cancer
• A phase II/III trial (NRG Oncology HN005) revealed a 98% two-year progression-free survival rate with standard chemoradiation for HPV-associated oropharyngeal cancer, a record high.
• De-intensified treatments, involving lower radiation doses and immunotherapy (nivolumab) instead of chemotherapy, did not match the efficacy of standard chemoradiation.
• Experts emphasize that de-intensification strategies should remain experimental, requiring further research to minimize side effects while maintaining high cure rates.
• The findings set a new benchmark for progression-free survival expectations, challenging the validity of phase II trials using lower standards for this patient population.
Nivolumab Plus Chemotherapy Shows QOL Benefits and High Response in Urothelial Cancer Subsets
• The CheckMate 901 trial showed nivolumab plus chemotherapy maintained quality of life (QOL) compared to chemotherapy alone in urothelial carcinoma patients.
• A subset analysis of CheckMate 901 revealed a high complete response rate (63%) in patients with lymph node-only metastatic urothelial cancer treated with nivolumab plus chemotherapy.
• Early data on sacituzumab govitecan plus ipilimumab and nivolumab showed promising response rates but was halted due to unexpected immune myocarditis.
• The combination of antibody-drug conjugates and immune checkpoint inhibitors remains a promising strategy but requires careful management of toxicities.
Sutro Biopharma and Merck Advance Novel Cytokine Derivative into Phase 1 for Solid Tumors
• Sutro Biopharma announces the dosing of the first patient in a Phase 1 study for a novel cytokine derivative therapeutic developed in collaboration with Merck.
• The therapeutic is designed for the treatment of advanced or metastatic solid tumors, marking a significant step in the collaboration between the two companies.
• Sutro Biopharma will receive a $10 million payment from Merck as a result of achieving this clinical milestone, per the 2018 agreement.
• The collaboration leverages Sutro's XpressCF® and Xpress CF+® platforms for cytokine derivative development, with Merck holding exclusive worldwide rights to resulting therapeutic candidates.
Patient-Centric Approaches and Technology Reshape Clinical Trial Landscape, Industry Experts Report
• Only 39% of clinical trial sites achieve enrollment targets according to Tufts Center, highlighting critical recruitment challenges in drug development.
• Industry leaders from INC Research, DrugDev, and EMD Serono emphasize the importance of non-traditional partnerships and patient-focused technology solutions to improve trial participation.
• Experts advocate for incorporating patient feedback through advisory boards and focus groups, while cautioning that technology solutions must be tailored to specific patient populations' needs.
FDA Approves Bavencio: Merck KGaA and Pfizer's Breakthrough Immunotherapy for Rare Skin Cancer
• The FDA has granted accelerated approval to Bavencio (avelumab), the first approved therapy for metastatic Merkel cell carcinoma, developed through Merck KGaA and Pfizer's collaboration.
• Bavencio becomes the fourth checkpoint inhibitor to enter the market, joining established immunotherapies from Bristol-Myers Squibb, Merck & Co, and Roche in the oncology space.
• The drug's peak sales are projected to reach $4-6 billion, with development ongoing in multiple cancer indications including first-line lung cancer, gastric cancer, and ovarian cancer.
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