FDA Advisory Committee Rejects New Indications for Genentech's Columvi and Pfizer's Talzenna Combination
• The FDA's Oncologic Drugs Advisory Committee voted 8-1 against approving Genentech's Columvi for a new indication, citing concerns about the pivotal trial's applicability to US patients.
• In a separate decision, the same committee unanimously rejected (8-0) Pfizer's application for Talzenna in combination with Xtandi for first-line treatment in adults with prostate cancer.
• These negative recommendations highlight the FDA's increasing scrutiny of oncology drug applications and may signal stricter requirements for clinical trial design and population representation.
FDA Advisory Committee Rejects Genentech's Columvi Expansion for Transplant-Ineligible DLBCL Patients
• The FDA's Oncologic Drugs Advisory Committee voted 8-1 against expanding Genentech's bispecific antibody Columvi for transplant-ineligible patients with relapsed/refractory diffuse large B-cell lymphoma.
• Committee members expressed concerns about the applicability of the Phase III STARGLO study data to the U.S. population, as half of the participants were Asian patients with only 25 enrolled from North America.
• Columvi, which targets CD20 and CD3 proteins, was initially approved in June 2023 for DLBCL patients who had undergone at least two prior lines of systemic therapy.
Glofitamab-GemOx Shows Survival Benefit in Phase 3 STARGLO Trial for R/R DLBCL, But FDA Advisory Committee Questions US Applicability
• The phase 3 STARGLO trial demonstrated that glofitamab combined with gemcitabine and oxaliplatin nearly doubled median overall survival to 25.5 months compared with 12.9 months for rituximab-GemOx in transplant-ineligible R/R DLBCL patients.
• Despite showing a 41% reduction in death risk and 63% reduction in disease progression, the FDA's Oncologic Drugs Advisory Committee voted 8-to-1 against the applicability of the trial data to US patients, citing regional outcome differences.
• Glofitamab, a CD20xCD3 bispecific antibody, is currently approved in over 30 countries and has accelerated approval in the US, with an FDA decision on full approval expected by July 20, 2025.
BATURA Trial: Airsupra Reduces Severe Asthma Exacerbation Risk by 47% Compared to Albuterol Alone
• AstraZeneca's Airsupra (albuterol/budesonide) demonstrated a 47% reduction in severe asthma exacerbation risk compared to albuterol alone in patients with mild asthma, according to the Phase IIIb BATURA trial.
• The anti-inflammatory rescue therapy reduced total systemic corticosteroid exposure by 63%, potentially decreasing risks associated with cumulative steroid use including diabetes, cardiovascular disease, and other adverse conditions.
• Results were so overwhelmingly positive that the Independent Data Monitoring Committee recommended early termination of the trial, suggesting a potential paradigm shift in asthma rescue treatment after 50 years of albuterol-only standard care.
Ten-Year APHINITY Data Shows Perjeta-Based Regimen Reduces Death Risk by 17% in HER2-Positive Early Breast Cancer
• Long-term follow-up data from the Phase III APHINITY trial demonstrates a statistically significant 17% reduction in risk of death when adding Perjeta (pertuzumab) to Herceptin (trastuzumab) and chemotherapy in early-stage HER2-positive breast cancer.
• The benefit was more pronounced in patients with lymph node-positive disease, showing a 21% reduction in death risk, reinforcing the regimen's value as a standard-of-care treatment in the curative setting.
• After ten years, 91.6% of patients receiving the Perjeta-based regimen were alive compared to 89.8% in the control group, with the previously reported invasive disease-free survival benefit maintained without new safety concerns.
Genentech to Invest $700 Million in New North Carolina Manufacturing Facility, Creating 400 High-Paying Jobs
• Genentech will establish a 700,000-square-foot high-volume fill-finish manufacturing plant in Holly Springs, North Carolina, representing a $700 million investment in the region's growing biotechnology sector.
• The facility will create approximately 400 new jobs with an average salary of $119,833—significantly higher than Wake County's average wage of $76,643—bringing an annual payroll impact exceeding $50 million.
• The project, supported by a Job Development Investment Grant, is expected to boost North Carolina's economy by more than $3 billion over 12 years, with a projected 230% return on investment of public dollars.
36-Month Results of OpRegen Cell Therapy for Geographic Atrophy to Be Presented at CTS 2025
• Long-term 36-month data from the Phase 1/2a trial of RG6501 (OpRegen) for geographic atrophy secondary to AMD will be presented at Clinical Trials at the Summit in June 2025.
• OpRegen, an allogeneic retinal pigment epithelial cell therapy developed by Lineage Cell Therapeutics in collaboration with Roche and Genentech, aims to counteract RPE cell loss in geographic atrophy.
• The therapy is currently advancing in a separate Phase 2a clinical study, potentially offering a novel treatment approach for geographic atrophy, a leading cause of adult blindness affecting over 5 million people globally.
ArkBio's AK3280 Shows Promising Results in Phase II IPF Trial with Significant Lung Function Improvement
• ArkBio's novel anti-fibrotic drug AK3280 demonstrated significant improvement in lung function during Phase II trials for idiopathic pulmonary fibrosis, with the highest dose group showing a 209.4 mL increase in FVC.
• Unlike current IPF treatments that merely slow disease progression, AK3280 achieved actual improvement in respiratory function while maintaining a favorable safety profile without the gastrointestinal issues associated with existing therapies.
• The multicenter, randomized study conducted across 31 clinical sites in China positions AK3280 as a potential breakthrough for IPF patients, with preparations now underway for pivotal Phase III clinical trials.
FDA Advisory Committee Discusses Columvi Combination for Relapsed or Refractory DLBCL
• Genentech's Columvi (glofitamab-gxbm) in combination with gemcitabine and oxaliplatin demonstrated a 41% reduction in risk of death for patients with relapsed or refractory diffuse large B-cell lymphoma in the Phase III STARGLO study.
• The FDA Oncologic Drugs Advisory Committee met to discuss the supplemental Biologics License Application for this combination therapy, focusing on the applicability of global trial results to U.S. patients.
• If approved, this first-of-its-kind bispecific antibody combination could provide a much-needed, off-the-shelf, fixed-duration treatment option for transplant-ineligible patients who currently face poor prognosis.
Balancing Efficacy and Cost Sustainability in Modern CLL Treatment: Insights from Dr. Pierluigi Porcu
• Clinicians treating chronic lymphocytic leukemia (CLL) face growing challenges in balancing clinical efficacy with long-term cost sustainability, requiring a holistic approach to patient care.
• Dr. Pierluigi Porcu emphasizes that effective CLL management requires understanding disease risk, patient comorbidities, and practice environment constraints including payer considerations.
• Despite its importance, value-based care adoption remains insufficient in oncology, with challenges in defining and measuring value across patient outcomes, quality of life, and treatment costs.
Roche Expands Molecular Glue Portfolio with $2 Billion Monte Rosa Partnership
• Roche has signed a $2 billion partnership with Monte Rosa Therapeutics, including a $50 million upfront payment, to develop molecular glue therapies targeting previously "undruggable" proteins in cancer and neurological diseases.
• This marks Roche's second major molecular glue deal in a month, following a similar $2 billion alliance with Orionis Biosciences, demonstrating the company's strategic push to establish leadership in this emerging therapeutic category.
• Monte Rosa will lead discovery and preclinical activities using its QuEEN platform, with Roche taking over late-stage development of candidates that can potentially address the 80% of human proteins currently inaccessible to traditional drug development approaches.
Xolair Shows Superior Efficacy Over Oral Immunotherapy in Landmark Food Allergy Trial
• Phase III OUtMATCH study demonstrates Xolair's superior efficacy with 36% of patients tolerating multiple food allergens compared to 19% in the oral immunotherapy group.
• Xolair treatment showed significantly better safety profile with zero serious adverse events, while oral immunotherapy group experienced 30.5% serious adverse events and higher treatment discontinuation rates.
• Follow-up data reveals promising results for post-Xolair food introduction, with 61-70% success rates for milk, egg, and wheat allergens, though lower rates for nuts.
Kazia Therapeutics Launches Phase 1b Trial of Paxalisib with Immunotherapy for Advanced Breast Cancer
• Kazia Therapeutics has initiated the ABC-Pax trial to evaluate paxalisib combined with immunotherapy in advanced breast cancer patients.
• The Phase 1b study will assess the safety and efficacy of paxalisib with pembrolizumab or olaparib in triple-negative breast cancer.
• The trial utilizes a novel liquid biopsy platform for real-time monitoring of cancer and immune cell behavior during treatment.
• This combination aims to reprogram dormant cancer cells, making them susceptible to immune attack, potentially improving patient outcomes.
Biopharma Industry Faces Continued Layoffs in 2025 Amid Strategic Realignments
• Biopharma companies continue to announce layoffs in 2025, driven by strategic realignments, pipeline reprioritizations, and challenging financial conditions.
• Several companies, including Intellia Therapeutics and IGM Biosciences, are cutting a significant portion of their workforce to focus on key programs and extend cash runways.
• These workforce reductions are often accompanied by discontinuation of certain drug development programs and a shift in strategic focus towards core assets.
• The layoffs reflect a broader trend in the industry as companies adapt to evolving market dynamics and prioritize efficiency and long-term sustainability.
Cytokinetics and Pyxis Oncology Announce Key Milestones for 2025; CytomX Prioritizes Pipeline
• Cytokinetics anticipates a pivotal year in 2025, focusing on the potential FDA approval and commercial launch of aficamten for obstructive hypertrophic cardiomyopathy (HCM) in the U.S.
• Pyxis Oncology is prioritizing the advancement of PYX-201, a novel antibody-drug conjugate (ADC), with data readouts expected in the second half of 2025 and the first half of 2026.
• CytomX Therapeutics is focusing on its lead program, CX-2051, an EpCAM-targeting PROBODY ADC for advanced metastatic colorectal cancer, with initial Phase 1a data expected in 1H 2025.
FDA Approves Roche's Companion Diagnostic for HER2-Ultralow Metastatic Breast Cancer
• The FDA has approved Roche's PATHWAY HER2 (4B5) test to identify HER2-ultralow metastatic breast cancer patients for targeted treatment.
• This approval expands treatment options for approximately 20-25% of HR-positive, HER2-negative breast cancer patients with HER2-ultralow status.
• The DESTINY-Breast06 trial demonstrated that ENHERTU improved median progression-free survival compared to chemotherapy in HER2-low and HER2-ultralow patients.
• Roche's diagnostic test standardizes HER2 assessment, reduces errors, and helps clinicians make informed treatment decisions for improved patient outcomes.
Durvalumab Gains Priority Review for Muscle-Invasive Bladder Cancer Treatment
• The FDA has granted priority review to durvalumab (Imfinzi) for muscle-invasive bladder cancer (MIBC), potentially expediting its approval.
• Phase 3 NIAGARA trial data supports the application, showing a 32% reduction in disease progression or recurrence with perioperative durvalumab.
• The Imfinzi regimen also demonstrated a 25% reduction in the risk of death, offering a significant survival benefit.
• Regulatory decisions are anticipated in the second quarter of 2025, with potential implications for MIBC treatment standards.
FDA Accepts sBLA for Glofitamab Plus Chemotherapy in Relapsed/Refractory DLBCL
• The FDA has accepted Roche's sBLA for glofitamab combined with gemcitabine and oxaliplatin (GemOx) for relapsed/refractory DLBCL patients ineligible for autologous stem cell transplant.
• The sBLA is based on the phase 3 STARGLO trial, which demonstrated a statistically significant and clinically meaningful improvement in overall survival compared to rituximab plus GemOx.
• The FDA is expected to make a decision on the approval of glofitamab in combination with GemOx by July 20, 2025, offering a potential new treatment option.
• The safety profile of glofitamab plus GemOx was consistent with the known safety profiles of the individual agents, with cytokine release syndrome being a common adverse event.
Roche and Lilly's Alzheimer's Blood Test Receives FDA Breakthrough Device Designation
• Roche and Lilly's Elecsys Amyloid Plasma Panel, a blood test for Alzheimer's, has been granted Breakthrough Device Designation by the FDA, expediting its development and review.
• The test detects phosphorylated tau proteins (pTau-217) in the blood, offering a less invasive alternative to PET scans and CSF analysis for early Alzheimer's diagnosis.
• The Elecsys test could help identify patients suitable for new amyloid-targeting drugs and accelerate recruitment into clinical trials, addressing the critical need for early diagnosis.
• With 75% of dementia cases undiagnosed globally, the pTau217 test promises to improve diagnostic rates and potentially alleviate strain on healthcare systems.
First RoActemra Biosimilar Tyenne Receives EU Approval, Marking Milestone for Fresenius Kabi
• The European Commission has granted approval for Tyenne, the first biosimilar version of Roche's blockbuster drug RoActemra (tocilizumab), developed by Fresenius Kabi for multiple inflammatory conditions and severe COVID-19.
• Tyenne is approved for all RoActemra indications, including rheumatoid arthritis, systemic juvenile idiopathic arthritis, and cytokine release syndrome, representing a significant advancement in accessible treatment options.
• This approval marks Fresenius Kabi's third biosimilar success and aligns with their Vision 2026 strategy to generate €1 billion in additional revenue every three years.
© Copyright 2025. All Rights Reserved by MedPath