AB Science Secures Chinese Patent for Masitinib in COVID-19 Treatment Until 2041
• AB Science has received a Chinese patent for its lead compound masitinib to treat COVID-19, providing intellectual property protection until April 2041.
• The patent is supported by preclinical data published in Science showing masitinib acts as a broad antiviral agent against SARS-CoV-2, with efficacy maintained against variants of concern.
• Recent positive Phase 2 study results confirm masitinib's anti-inflammatory activity in COVID-19 by controlling activated macrophages and mast cells, positioning it as a potential treatment for future pandemics.
CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder
• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.
• The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein.
• This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.
ClostraBio Secures INVENT Illinois Funding, Appoints New CSO, and Prepares for Microbiome Therapeutic Launch
• ClostraBio has secured new investment from the Illinois Innovation Venture Fund (INVENT) and NextGen Nutrition Investment Partners to advance its microbiome-focused therapeutics for gut health.
• The company has appointed Brian Meehan, PhD, as Chief Scientific Officer to lead the summer 2025 launch of CLB101™, a first-in-class probiotic that produces butyrate directly in the lower gut.
• University of Chicago spin-out ClostraBio is developing a dual-platform approach with next-generation probiotics and a polymeric prodrug delivery system to address unmet needs in microbiome-related gut health conditions.
Novel Chemo-Immunotherapy Approach Shows Promise for Advanced HPV-Negative Head and Neck Cancer
• A Phase 2 clinical trial at UChicago Medicine demonstrated that combining nivolumab immunotherapy with chemotherapy led to tumor shrinkage in 53% of advanced HPV-negative head and neck cancer patients.
• The innovative treatment approach, which included response-adaptive chemo-radiation therapy, showed improved survival outcomes and reduced toxic side effects, particularly in patients who responded well to initial treatment.
• Higher PD-L1 expression levels were associated with better treatment responses, suggesting its potential as a biomarker for predicting treatment outcomes in this patient population.
Bipartisan EPIC Act Seeks to Eliminate IRA's Small Molecule Drug Development Disincentive
• House legislators have reintroduced the bipartisan EPIC Act aimed at equalizing negotiation timelines between small molecule drugs and biologics under the Inflation Reduction Act.
• The current IRA framework creates a 'pill penalty' by allowing only 9 years before price negotiations for small molecules compared to 13 years for biologics, potentially reducing revenue by 50% during critical years.
• Industry experts argue the legislation is crucial for maintaining investment in small molecule drug development and ensuring patient access to future life-saving medicines.
Vafidemstat Phase IIa Results Published: Shows Promise in Treating Psychiatric Aggression
• Oryzon Genomics' Phase IIa REIMAGINE trial results demonstrate vafidemstat's effectiveness in reducing agitation and aggression across BPD, ADHD, and ASD patient populations.
• The groundbreaking basket trial establishes LSD1 inhibition as a novel therapeutic approach for managing psychiatric aggression, with findings published in Psychiatry and Clinical Neurosciences.
• Following successful Phase IIb results and FDA discussions, Oryzon is advancing to PORTICO-2 Phase III trial for vafidemstat in borderline personality disorder treatment.
FDA Clears Northwestern University's IND Application for Calidi's Novel Glioma Treatment CLD-101
• The FDA has cleared Northwestern University's IND application for Calidi Biotherapeutics' CLD-101, a stem cell-based platform delivering oncolytic viruses for treating high-grade glioma, with Phase 1b/2 trials starting late 2024.
• Previous Phase 1 trials demonstrated promising results with median progression-free survival of 9.05 months and overall survival of 18.4 months, showing no dose-limiting toxicity.
• The treatment addresses a critical unmet need in high-grade glioma patients, who currently face a challenging five-year survival rate of only 5-10% with conventional therapies.
STEP-2 Trial of Cervical Cancer Screening Interventions Joins the NIH Collaboratory
The NIH Pragmatic Trials Collaboratory welcomes the STEP-2 trial, focusing on cervical cancer screening through self-sampling kits in underserved populations, to its portfolio. This trial aims to address the low screening rates exacerbated by the COVID-19 pandemic by evaluating the effectiveness of different distribution methods of HPV self-sampling kits.
Anitocabtagene Autoleucel Shows Durable Efficacy in Advanced Multiple Myeloma
• Anitocabtagene autoleucel (anito-cel) demonstrates a high overall response rate of 97% in heavily pretreated, high-risk relapsed or refractory multiple myeloma patients.
• The therapy shows a manageable safety profile with no delayed neurotoxicities reported, making it a promising option for challenging-to-treat myeloma cases.
• Preliminary data indicates a 93.3% progression-free survival rate at 6 months and a 96.5% overall survival rate at both 6 and 12 months.
• A phase 3 trial (iMMagine-3) is underway to compare anito-cel with standard therapy, potentially leading to its approval as a new treatment option.
Amivantamab Plus Lazertinib Demonstrates Superior Overall Survival in EGFR-Mutated NSCLC
• The MARIPOSA trial demonstrated that amivantamab plus lazertinib significantly improved overall survival (OS) compared to osimertinib in EGFR-mutated non-small cell lung cancer (NSCLC).
• The combination therapy showed a 30% reduction in the risk of disease progression or death, with a median progression-free survival (PFS) of 23.7 months versus 16.6 months for osimertinib.
• Updated data from the MARIPOSA trial presented at the 2024 IASLC World Conference on Lung Cancer showed a sustained benefit in intracranial PFS with amivantamab plus lazertinib.
• Enhanced dermatologic management in the COCOON trial reduced the incidence of skin-related adverse events associated with amivantamab and lazertinib, improving patient quality of life.
AstraZeneca and Daiichi Sankyo Withdraw EU Application for Lung Cancer Drug Datopotamab Deruxtecan
• AstraZeneca and Daiichi Sankyo withdrew their EU marketing application for datopotamab deruxtecan (Dato-DXd) for advanced non-small cell lung cancer following EMA feedback.
• The decision was based on the Phase III TROPION-Lung01 trial results, where Dato-DXd showed improved PFS but not statistically significant overall survival.
• The companies remain committed to developing Dato-DXd for lung cancer, with ongoing trials exploring its potential in various settings.
• A separate application for Dato-DXd for HER2-negative metastatic breast cancer remains under review by the EMA.
Sana Biotechnology's HIP Technology Shows Promise in Type 1 Diabetes Islet Cell Transplant Study
• Sana Biotechnology's UP421 demonstrates survival and function of transplanted islet cells in a type 1 diabetes patient without immunosuppression.
• The first-in-human study shows HIP-engineered cells evade immune detection, producing insulin as indicated by increased C-peptide levels.
• MRI scans confirm graft survival 28 days post-transplantation, with no reported safety issues, suggesting potential for a curative T1D treatment.
• These results support the development of Sana's SC451, a stem cell-derived islet cell program, offering hope for scalable T1D therapies.
Experts Highlight Advances in Lung Cancer Treatment: Immunotherapy, ADCs, and Equity in Care
• Leading oncologists at the Chicago IVBM event discussed the transformation of lung cancer treatment, emphasizing the evolution of immunotherapy from second-line to first-line and early-stage treatments.
• Experts addressed emerging technologies including antibody-drug conjugates like trastuzumab deruxtecan, highlighting significant progression-free survival benefits in second-line settings.
• The panel identified critical challenges in healthcare equity, calling for standardized molecular testing protocols and enhanced collaboration between academic centers and community providers.
Eylea HD Shows Promise in Macular Edema Following Retinal Vein Occlusion
• Eylea HD (aflibercept 8 mg) demonstrated non-inferior visual acuity gains compared to Eylea (aflibercept 2 mg) in patients with macular edema following retinal vein occlusion.
• The Phase 3 QUASAR trial showed that Eylea HD, dosed every 8 weeks, achieved similar vision improvements to Eylea, dosed every 4 weeks.
• A significant portion of patients on Eylea HD were able to maintain an 8-week dosing regimen, potentially reducing the burden of frequent injections.
• Regeneron plans to submit these data to regulatory authorities, seeking approval for Eylea HD in this indication, offering a less frequent treatment option.
Anthracycline-Based Chemotherapy Improves Survival in High-Risk, Early-Stage Breast Cancer
• Adjuvant anthracycline- plus taxane-based chemotherapy (T-AC) improves survival outcomes in high-risk, early-stage, hormone receptor (HR)-positive, HER2-negative breast cancer patients.
• Patients with recurrence scores (RS) of 31 or greater, determined by the OncotypeDX test, experienced significant benefits from T-AC compared to taxane-based chemotherapy alone (TC).
• The addition of anthracyclines was associated with improved distant recurrence-free interval, distant recurrence-free survival, and overall survival at five years.
• While showing promise, the use of anthracyclines requires careful consideration of potential long-term risks, including cardiotoxicity and hematologic malignancies.
Rapcabtagene Autoleucel Shows High Remission Rates in Relapsed/Refractory DLBCL
• Rapcabtagene autoleucel, a CD19-directed CAR T-cell therapy, demonstrated an 88.3% overall response rate in patients with relapsed/refractory DLBCL.
• The therapy achieved a 65% complete remission rate, with durable responses observed in over half of the patients at 12 months.
• The CAR T-cell therapy utilizes the T-Charge platform, which allows for rapid manufacturing of cells in under 2 days.
• Treatment was well-tolerated, with manageable rates of cytokine release syndrome and neurotoxicity, suggesting a favorable safety profile.
Seth Lerner Honored with Huggins Medal for Contributions to Urologic Oncology
• Dr. Seth Lerner received the Huggins Medal at the 2024 SUO annual meeting for his contributions to urologic oncology, particularly in bladder cancer research.
• Lerner emphasized the importance of team science, citing the Bladder Cancer Research Consortium and The Cancer Genome Atlas (TCGA) project as key examples.
• His work has significantly advanced the understanding of bladder cancer molecular subtypes and their impact on treatment response and survival outcomes.
• Lerner also highlighted his involvement with SWOG and NCTN, emphasizing the value of collaborative clinical trials in improving patient care.
QuEra Computing-Powered Projects Advance in Wellcome Leap's Quantum for Bio Challenge
• Three research projects involving QuEra Computing have progressed to Phase Two of the Wellcome Leap's Quantum for Bio Challenge, focusing on healthcare revolution through quantum computing.
• These projects target quantum-enhanced drug discovery for myotonic dystrophy, scalable quantum simulation for virtual screening, and quantum chemistry techniques for Alzheimer's and Parkinson's-related proteins.
• QuEra's neutral-atom quantum computers, accessible via public cloud, provide advanced processing capabilities, supporting large-scale simulations and paving the way for quantum breakthroughs in healthcare.
CRB-701 Receives FDA Fast Track Designation for Relapsed/Refractory Metastatic Cervical Cancer
• The FDA has granted Fast Track designation to CRB-701 for the treatment of relapsed or refractory metastatic cervical cancer, aiming to expedite its development and review.
• CRB-701, a next-generation antibody-drug conjugate (ADC) targeting Nectin-4, is designed to improve targeted delivery and reduce toxic effects compared to existing therapies.
• Corbus Pharmaceuticals has completed enrollment in the dose-escalation phase of a Phase 1 clinical trial, with initial data expected in Q1 2025.
• Early data from the Phase 1 trial presented at ASCO 2024 showed promising antitumor activity and a manageable safety profile across multiple Nectin-4 positive tumor types.
University of Chicago's DFI to Commence Clinical Trials on Gut Microbiome Restoration
• The Duchossois Family Institute (DFI) at UChicago is set to begin clinical trials in January to investigate rebuilding the gut microbiome in compromised patients.
• The trials will use eight bacterial strains manufactured in-house at DFI's Good Manufacturing Practices (GMP) facility, with three strains already prepared for testing.
• Researchers aim to assess the impact of individual and combined bacterial strains on gut microbiomes, focusing on their retention and health-promoting metabolites.
• The initial phase will involve a small group of consenting patients from UChicago Medicine to ensure safety and controlled study parameters.
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