University of Kansas Medical Center

Springbok Analytics Pioneers AI-Driven MRI Analysis in Landmark Pediatric FSHD Study

2 months ago

• The NIH-funded MOVE Peds study represents the first large-scale clinical investigation of pediatric facioscapulohumeral muscular dystrophy (FSHD), addressing critical gaps in understanding disease progression in children. • Springbok Analytics is providing AI-powered muscle analysis technology that quantifies both fat infiltration and inflammation markers while minimizing scan time to under 45 minutes—crucial for pediatric patients. • Data from the multi-site study will establish essential natural history metrics, validate outcome measures, and potentially accelerate regulatory pathways for emerging FSHD therapies targeting children.

Regulus Therapeutics' Farabursen Shows Promise in ADPKD Phase 1b Trial and Gains FDA Agreement on Phase 3 Design

4 months ago

• Regulus Therapeutics announced positive topline results from an interim analysis of the Phase 1b MAD study of farabursen for ADPKD. • The study demonstrated a continued mechanistic dose response and a notable reduction in htTKV growth rate in ADPKD patients. • Regulus reached an agreement with the FDA on key components of a Phase 3 single pivotal trial for potential accelerated approval of farabursen. • Farabursen was well-tolerated in the Phase 1b study, reinforcing its potential as a safe treatment option for ADPKD.

NOURISH Trial Addresses Food Insecurity and Malnutrition in Blood Cancer Patients During Treatment

4 months ago

• A groundbreaking clinical trial, NOURISH, aims to combat food insecurity and malnutrition in blood cancer patients undergoing transplant or cellular therapy through structured food assistance programs. • Cancer patients experiencing malnutrition face increased risks of infection, longer hospital stays, reduced treatment tolerance, and higher mortality rates, according to Dr. Anna Arthur, the study's primary investigator. • The trial provides patients with bi-weekly food bags containing 2-3 days of balanced nutrition, along with recipes and cooking demonstrations to support optimal nutritional intake during treatment.

AskBio's AB-1005 Gene Therapy for Parkinson's Disease Gains FDA RMAT Designation and Enrolls First Phase II Participants

4 months ago

• AskBio's AB-1005, a gene therapy for Parkinson's disease, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, potentially expediting its development. • The RMAT designation was supported by positive 36-month Phase Ib data, demonstrating a favorable safety profile and encouraging trends in clinical outcome measures. • The Phase II REGENERATE-PD trial, evaluating AB-1005 in moderate-stage Parkinson's, has randomized its first participants across sites in the US, with plans to expand to Europe. • AB-1005 delivers glial cell line-derived neurotrophic factor (GDNF) to promote the survival of dopaminergic neurons, addressing a critical unmet need in Parkinson's treatment.

Nurix Therapeutics' BTK Degrader NX-5948 Shows Promise in B-Cell Malignancies and Autoimmune Diseases

4 months ago

• Nurix Therapeutics' NX-5948 demonstrates a 75.5% objective response rate in relapsed/refractory CLL/SLL patients in Phase 1 study. • NX-5948 receives PRIME designation from EMA for CLL and Fast Track designation from FDA for Waldenstrom's Macroglobulinemia. • Nurix plans to initiate pivotal trials for NX-5948 in CLL and expand development into autoimmune diseases in 2025. • The company's strong financial position, with $609.6 million in cash, supports aggressive development of its pipeline.

Real-World Data Reinforces Cilta-Cel Efficacy in Relapsed Multiple Myeloma, While Ide-Cel Shows Promise in CNS Involvement

5 months ago

• Real-world evidence confirms cilta-cel's effectiveness in relapsed/refractory multiple myeloma, mirroring clinical trial outcomes with high response rates and durable remission. • A significant portion of patients in the real-world study wouldn't have met clinical trial eligibility, highlighting cilta-cel's potential in a broader patient population. • Ide-cel demonstrates promising results in patients with CNS involvement of multiple myeloma, showing favorable responses and manageable safety profiles in a retrospective analysis. • Renal impairment does not significantly impact the efficacy of BCMA-targeting CAR T-cell therapies in relapsed/refractory multiple myeloma, though increased monitoring for ICANS and infections may be warranted.

Acalabrutinib Plus Venetoclax Significantly Improves PFS in Untreated CLL

5 months ago

• The phase 3 AMPLIFY trial demonstrated that acalabrutinib plus venetoclax, with or without obinutuzumab, significantly improved progression-free survival (PFS) in treatment-naive CLL patients. • The doublet and triplet regimens reduced the risk of disease progression or death by 35% and 58%, respectively, compared to chemoimmunotherapy. • The highest rates of undetectable minimal residual disease (uMRD) were observed in patients treated with the triplet regimen of acalabrutinib, venetoclax, and obinutuzumab. • The combination provides a chemotherapy-free, fixed-duration option for previously untreated CLL, offering improved outcomes and flexibility in managing the disease.

Nipocalimab Shows Promise in Phase II Trial for Sjögren's Syndrome

6 months ago

• Nipocalimab, an anti-IgG monoclonal antibody, demonstrated significant improvement in clinical ESSDAI scores in patients with primary Sjögren's syndrome. • A phase II trial revealed that patients receiving 15 mg/kg of nipocalimab every two weeks experienced a greater reduction in disease activity compared to placebo. • The higher dose of nipocalimab also led to improvements in Physician's Global Assessment and responder rates based on STAR and CRESS standards. • Janssen, the developer, is planning a phase III study to further evaluate the efficacy and safety of nipocalimab in treating Sjögren's syndrome.

Standardized Trial Design Proposed to Advance Bladder Cancer Trimodality Therapy

7 months ago

• Expert panel recommends pragmatic eligibility criteria for trimodality therapy (TMT) clinical trials in bladder cancer, including ECOG performance status 0-2 and specific tumor stages. • Bladder Intact Event-Free Survival (BIEFS) is proposed as a primary endpoint for registration trials comparing TMT interventions, focusing on clinically meaningful outcomes. • Overall survival is deemed an appropriate secondary or co-primary endpoint, acknowledging recent advancements in systemic therapy and their impact on long-term survival. • The panel emphasizes the importance of patient-reported quality of life assessments to support primary and secondary endpoints, ensuring patient-centricity in trial design.

EUS-Guided Celiac Plexus Block Shows Promise in Pancreatic Cancer Pain Management

7 months ago

• Endoscopic ultrasound-guided celiac plexus block (EUS-CPB) is being investigated for pain palliation in pancreatic cancer patients. • A multi-center, randomized controlled trial is underway to evaluate the efficacy of EUS-CPB compared to standard pain management. • The study aims to determine if EUS-CPB can reduce pain intensity and improve quality of life for patients with pancreatic cancer. • Preliminary results suggest EUS-CPB could offer a valuable alternative or adjunct to traditional pain control methods.

CAR T-cell Therapy Monitoring: Study Suggests Reduced REMS May Improve Accessibility

8 months ago

• A recent study suggests that the FDA's Risk Evaluation and Mitigation Strategy (REMS) for CAR T-cell therapy may be overly restrictive, impacting patient accessibility. • The study found that the incidence of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) is low after the first two weeks post-CAR T infusion. • Researchers propose a more flexible approach to monitoring, with a mandatory 2-week period near the treatment center, followed by individualized assessments. • Revisiting REMS mandates could reduce the physical and financial burden on patients, potentially improving access to CAR T-cell therapy, especially for underserved populations.

Gene and Cell Therapy Data Roundup: Updates in DLBCL, SMA, XLRP, and More

8 months ago

• REGENXBIO's RGX-121 shows sustained efficacy in MPSII, with significant reduction in heparan sulfate levels and reduced need for enzyme replacement therapy. • Biogen's nusinersen demonstrates superior efficacy in SMA at a higher dose compared to the FDA-approved dose, showing statistically significant improvement in CHOP-INTEND scores. • Allogeneic CAR-T Azer-Cel achieves complete responses in some DLBCL patients, with ongoing responses observed in cohort B of a phase 1/1b trial. • Beacon Therapeutics' AGTC-501 for XLRP maintains a favorable safety profile at 36 months, with most adverse events resolving within the first three months post-treatment.