Newcastle University

University of Birmingham Pioneers Personalized Cancer Treatment Approaches with Novel Technologies

4 months ago

• University of Birmingham researchers are revolutionizing cancer diagnosis through innovative MRI and machine learning techniques that can identify pediatric brain tumor types in just ten minutes. • Birmingham launches Europe's first pancreatic cancer mRNA vaccine trial, developing personalized immunotherapy treatments tailored to individual patients' tumor profiles. • The shift from one-size-fits-all cancer treatment to precision medicine approaches marks a significant advancement in cancer care, incorporating genetic influences and patient-specific factors.

Akero's Efruxifermin Shows Promise in Reversing Cirrhosis Due to MASH in Phase 2b Trial

4 months ago

• Akero Therapeutics' Efruxifermin (EFX) demonstrated statistically significant cirrhosis reversal in patients with MASH in a Phase 2b trial. • In patients with baseline and week 96 biopsies, 39% treated with 50mg EFX showed cirrhosis reversal without worsening MASH, compared to 15% with placebo. • The SYMMETRY study underscores the benefit of longer EFX treatment, showing a doubling of effect size from weeks 36 to 96 in the 50mg group. • Akero is continuing to evaluate 50mg EFX in the Phase 3 SYNCHRONY Outcomes study for patients with compensated cirrhosis due to MASH.

MHRA Approves Amgen's Imdylltra (Tarlatamab) for Advanced Small Cell Lung Cancer

5 months ago

• The UK's MHRA has granted conditional marketing authorization to Amgen's Imdylltra (tarlatamab) for extensive-stage small cell lung cancer (ES-SCLC). • The approval is for adult patients who have progressed after at least two prior lines of therapy, including platinum-based chemotherapy. • Tarlatamab demonstrated a 41% objective response rate and a 9.7-month median duration of response in the Phase 2 DeLLphi-301 trial. • Imdylltra, a bispecific T-cell engager targeting DLL3, offers a new treatment option for ES-SCLC patients with limited alternatives.

Tharimmune's TH104 Advances to Phase 2 for Pruritus in Primary Biliary Cholangitis

5 months ago

• Tharimmune plans to initiate a Phase 2 clinical trial in 2025 to evaluate TH104 for moderate-to-severe pruritus associated with primary biliary cholangitis (PBC). • The Phase 2 trial will be a multicenter, randomized, double-blind, placebo-controlled study enrolling approximately 40 patients across the U.S., Europe, and the UK. • TH104 demonstrated a favorable pharmacokinetic profile and a mild side-effect profile in a Phase 1 clinical trial, with positive regulatory feedback from the FDA and EMA. • The company also reported corporate achievements, including licensing agreements and the appointment of Sanam Parikh to its Board of Directors.

Novartis and Apellis Advance C3G Therapies Towards FDA Submission

6 months ago

• Novartis' Fabhalta (iptacopan) demonstrated significant proteinuria reduction in the Phase III APPEAR-C3G trial, meeting its primary endpoint and supporting regulatory submissions. • Apellis' Empaveli (pegcetacoplan) showed a substantial reduction in proteinuria and C3c deposit clearance in the Phase III VALIANT trial, indicating early efficacy in C3G patients. • Nephrologists express a strong interest in new C3G therapies that can slow eGFR decline and reduce proteinuria, addressing the urgent need for innovative treatments. • Spherix data suggests treatment preferences may be influenced by administration route, with Fabhalta's oral administration potentially offering an advantage.

CervoMed's Neflamapimod Shows Promise in Dementia with Lewy Bodies (DLB) Treatment

7 months ago

• CervoMed's neflamapimod demonstrated a significant reduction in plasma GFAP levels, a key biomarker of neurodegenerative activity in DLB patients, in a Phase 2a trial. • The reduction in GFAP levels correlated with improved clinical outcomes, suggesting neflamapimod's therapeutic effect is mediated through the basal forebrain cholinergic system. • The RewinD-LB Phase 2b trial enrolled a DLB patient population without tau pathology, potentially increasing the study's statistical power to detect treatment effects. • Topline results from the RewinD-LB Phase 2b clinical trial of neflamapimod in DLB patients are expected in December, with high statistical power anticipated.

CervoMed's Neflamapimod Shows Promise in Dementia with Lewy Bodies by Targeting Neurodegenerative Activity

7 months ago

• CervoMed's neflamapimod demonstrates potential in treating Dementia with Lewy Bodies (DLB) by reducing neurodegenerative activity in the basal forebrain cholinergic system. • Phase 2a trial data indicates that neflamapimod significantly lowers plasma GFAP levels, a key biomarker, correlating with improved clinical outcomes in DLB patients. • The RewinD-LB Phase 2b study enrolled a DLB patient population optimized to exhibit neflamapimod's treatment effect, with topline data expected in December. • By focusing on patients without tau pathology, the RewinD-LB trial aims for enhanced statistical power to detect clinically meaningful differences between neflamapimod and placebo.

CervoMed's Neflamapimod Shows Promise in Dementia with Lewy Bodies Treatment

7 months ago

• CervoMed's neflamapimod demonstrated a significant reduction in plasma GFAP levels (p=0.015 vs placebo) in Phase 2a trials, suggesting reduced neurodegenerative activity in DLB. • The ongoing RewinD-LB Phase 2b study successfully enrolled DLB patients without tau pathology, optimizing the potential treatment effect of neflamapimod. • With 80 patients per arm, the RewinD-LB Phase 2b study boasts nearly 100% statistical power for its primary endpoint, with topline results expected in December. • Neflamapimod could become a first-in-class therapy for the 1.4 million DLB patients in the U.S. and EU, addressing a significant unmet medical need.

CervoMed's Neflamapimod Shows Promise in Dementia with Lewy Bodies by Targeting Neurodegenerative Activity

7 months ago

• CervoMed's neflamapimod demonstrated a significant reduction in plasma GFAP levels, a key biomarker of neurodegenerative activity in dementia with Lewy bodies (DLB). • The reduction in GFAP levels correlated with improved clinical outcomes, suggesting neflamapimod's potential to modify disease progression in DLB patients. • The RewinD-LB Phase 2b trial enrolled a DLB patient population optimized for treatment effect by excluding those with tau pathology, enhancing the study's statistical power. • Topline results from the RewinD-LB Phase 2b clinical trial of neflamapimod are expected in December, with increased confidence due to targeted patient selection.

Astex Pharmaceuticals Highlights Preclinical and Clinical Advances at EORTC-NCI-AACR Symposium

7 months ago

• Astex Pharmaceuticals is presenting preclinical data on ASTX528, a novel CBP/p300 HAT domain inhibitor, showcasing potent in vivo activity and a favorable safety profile. • The company will also present findings on ASTX295, a Phase II-ready MDM2 antagonist designed to be bone marrow-sparing, offering a potentially improved safety profile. • Biomarkers predictive of response to ASTX295 in solid tumors with wild-type p53 will be discussed, potentially refining patient selection for future clinical trials. • Further research on ASTX295 demonstrates that pulsatile induction of the p53 pathway enhances its therapeutic index in vivo, suggesting optimized dosing strategies.