SystImmune to Present Promising Data on Iza-bren for HER2-Negative Breast Cancer at ESMO Breast 2025
• SystImmune will present updated safety and efficacy data for izalontamab brengitecan (iza-bren), an EGFRxHER3 bispecific antibody-drug conjugate, in locally advanced or metastatic breast cancer at ESMO Breast 2025 on May 16.
• Early clinical results show encouraging efficacy across all HER2 levels in HER2-negative breast cancer patients, including HER2 0 patients, suggesting potential therapeutic benefits for patients with limited treatment options.
• Iza-bren, being jointly developed by SystImmune and Bristol Myers Squibb, works by blocking EGFR and HER3 signals to cancer cells while delivering a therapeutic payload that induces cancer cell death.
Prothena's Birtamimab Shows Promise in Phase 3 Trial for AL Amyloidosis with Focus on High-Risk Patients
• Prothena's Phase 3 AFFIRM-AL trial evaluates birtamimab in Mayo stage 4 AL amyloidosis patients, targeting a survival benefit with a p-value threshold of 0.10 per FDA agreement.
• Approximately 80% of trial participants are receiving daratumumab as standard of care, with researchers expecting mortality trends similar to previous VITAL trial observations.
• The company estimates 16,000 diagnosed and treated AL amyloidosis patients in the US, highlighting significant market potential for an anti-amyloid therapy that directly clears amyloid from vital organs.
Roche Launches SKYLINE: Four-Year Prevention Trial for Gantenerumab in Early-Stage Alzheimer's
• Roche initiates SKYLINE study, enrolling 1,250 participants aged 60-80 with early biological markers of Alzheimer's but no cognitive impairment, to evaluate gantenerumab's effectiveness in disease prevention.
• The study represents a significant investment in Alzheimer's research, featuring collaboration with prestigious institutions including Banner Alzheimer's Institute and Massachusetts General Hospital.
• Gantenerumab offers a potential advantage with its subcutaneous home administration option, distinguishing it from competitors requiring clinical infusions.
FDA Expands Evrysdi Approval to Include Infants Under Two Months with Spinal Muscular Atrophy
• Roche and PTC Therapeutics' oral SMA drug Evrysdi (risdiplam) receives FDA approval for use in infants younger than two months, enabling treatment across all age groups from newborns to adults.
• The RAINBOWFISH study demonstrated significant efficacy, with treated pre-symptomatic babies achieving key developmental milestones including sitting, standing, and walking within 12 months.
• The expanded approval positions Evrysdi to compete directly with Biogen's Spinraza and Novartis' Zolgensma, while offering the advantage of at-home oral administration.
Biopharma Industry Faces Pivotal Year: J.P. Morgan Conference Signals Key 2025 Challenges
• The 2024 J.P. Morgan Healthcare Conference kicked off with significant M&A activity, including Johnson & Johnson's $15 billion acquisition of Intra-Cellular Therapies.
• Despite notable deals from GSK and Eli Lilly with biotech companies, industry sentiment remains cautious amid regulatory uncertainty and questions about investment returns.
• Key challenges facing the sector include Trump administration's healthcare policies, investor confidence in biotech, success of immunology and obesity drug investments, and growing Chinese competition.
Prasinezumab Phase IIb Trial Fails Primary Endpoint, Shows Promise in Parkinson's Subgroup
• Genentech's prasinezumab Phase IIb PADOVA study in early-stage Parkinson's missed its primary endpoint of delaying motor progression.
• A pre-specified analysis showed a more pronounced effect in patients treated with levodopa (HR=0.79), suggesting a potential benefit in this subgroup.
• The investigational monoclonal antibody continues to be well-tolerated, with ongoing open-label extension studies to further explore observed effects.
• Roche plans to present full PADOVA study results at an upcoming medical meeting and will work with health authorities to determine next steps.
UCB's Minzasolmin Fails Phase 2 ORCHESTRA Trial for Early Parkinson's Disease
• UCB's minzasolmin, an oral alpha-synuclein misfolding inhibitor, did not meet primary or secondary endpoints in the Phase 2 ORCHESTRA trial for early Parkinson's.
• The ORCHESTRA study enrolled over 450 patients to assess minzasolmin's efficacy, safety, tolerability, and pharmacokinetics over 12-18 months.
• UCB will terminate the extension phase of the minzasolmin program and shift focus to glovadalen (UCB0022) and UCB7583 for Parkinson's treatment.
• Analysis of disease biomarker data from the ORCHESTRA study is ongoing, with findings to be submitted for publication in a peer-reviewed journal.
Parkinson's Disease Research Focuses on Disease-Modifying Therapies
• Research in the seven major markets (7MM) increasingly prioritizes disease-modifying therapies (DMTs) and treatments for non-motor symptoms of Parkinson's disease.
• 66% of the 93 products in Phase I-III development are prospective neuroprotective agents or DMTs targeting mechanisms like alpha-synuclein aggregation and neuroinflammation.
• 26% of pipeline DMTs target alpha-synuclein aggregation, though this approach has faced setbacks, highlighting the complexity of Parkinson's pathogenesis.
• There is also growing research into treatments for non-motor symptoms, with 3% of the pipeline specifically targeting Parkinson's disease dementia and psychosis.
Alzheimer's Drug Trials Under Scrutiny for Genetic Risk Disclosure
• Eisai and Eli Lilly face criticism for not fully disclosing genetic risks to participants in Leqembi and Kisunla Alzheimer's drug trials.
• Volunteers with the ApoE4 gene, particularly homozygotes, faced a higher risk of brain bleeding (ARIA) during the anti-amyloid treatments.
• Experts debate whether withholding genetic information undermined informed consent, balancing patient safety with scientific rigor.
• Current practice now favors upfront disclosure of ApoE4 status in Alzheimer's clinical trials to allow informed decision-making.
AbbVie to Acquire Cerevel, Novartis' Fabhalta Approved, and Other Pharma Updates
• AbbVie is set to acquire Cerevel Therapeutics for $8.7 billion, bolstering its neuroscience pipeline with candidates targeting schizophrenia, Parkinson's disease, and mood disorders.
• The FDA has approved Novartis' Fabhalta (iptacopan), the first oral monotherapy for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder.
• Pfizer halted Phase III development of its twice-daily obesity drug, danuglipron, due to significant gastrointestinal side effects observed in Phase II trials.
• Biogen and Eisai received full FDA approval for Leqembi, an Alzheimer's disease drug shown to slow cognitive decline, expanding Medicare coverage.
Alzheimer's Disease Treatment Advances: Lecanemab and Donanemab Offer New Hope
• Lecanemab (Leqembi) received full FDA approval in July 2023 and has shown success in slowing Alzheimer's progression, marking a significant milestone in treatment.
• Eli Lilly's donanemab demonstrated a 35% reduction in cognitive decline by targeting amyloid protein, offering another promising treatment option for Alzheimer's.
• These new drugs, while not cures, represent a crucial step forward, potentially leading to earlier diagnoses, increased awareness, and further research into Alzheimer's.
• Challenges remain in ensuring broad access, managing costs, and establishing necessary infrastructure for diagnosis and treatment, particularly in low- and middle-income countries.
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