Aspartic acid

FDA Approves Belzutifan as First Oral Treatment for Advanced Pheochromocytoma and Paraganglioma

8 days ago

• The FDA has approved belzutifan (Welireg) for adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma, based on promising phase 2 trial results. • As a selective HIF-2α inhibitor, belzutifan demonstrated a 26% overall response rate with a median duration of response of 20.4 months, offering a novel mechanism of action compared to historically used treatments. • This approval is particularly significant as previous treatment options were limited, with the only previously approved agent no longer commercially available and alternative therapies associated with concerning toxicity profiles.

Cancer Vaccines Emerge as Promising Frontier in Oncology with Multiple Approaches Showing Clinical Success

22 days ago

• Cancer vaccines are gaining momentum across multiple tumor types, with FDA-approved options like BCG, sipuleucel-T, and talimogene laherparepvec demonstrating clinical utility in bladder cancer, prostate cancer, and melanoma respectively. • Novel vaccine approaches including mRNA-based mRNA-4157, KRAS-targeted ELI-002, and viral vector-based aglatimagene besadenovec are showing promising results in clinical trials, with significant improvements in survival outcomes across various cancers. • Experts believe cancer vaccines hold particular promise in early-stage and high-risk disease settings by targeting micrometastatic disease, potentially increasing cure rates and transforming treatment paradigms when combined with existing immunotherapies.

Aulos Bioscience to Present Promising Phase 2 Data for Novel IL-2 Therapeutic in Melanoma Treatment

2 months ago

• Aulos Bioscience will present new Phase 2 data for AU-007, an AI-designed monoclonal antibody targeting IL-2, showing promising results in second-line melanoma treatment at the upcoming AACR Annual Meeting. • AU-007's unique mechanism prevents IL-2 from binding to regulatory T cells while allowing it to activate effector T cells and NK cells, potentially overcoming limitations of traditional IL-2 therapies. • The novel therapeutic may significantly reduce serious side effects associated with high-dose IL-2 therapy, including vascular leak syndrome and pulmonary edema, by preventing IL-2 binding to CD25-containing receptors.

AstraZeneca's Eneboparatide Meets Primary Endpoint in Phase III Trial for Hypoparathyroidism

2 months ago

• Eneboparatide, an investigational parathyroid hormone receptor agonist, successfully normalized serum calcium levels and eliminated the need for standard supplementation in adults with chronic hypoparathyroidism at 24 weeks. • The CALYPSO Phase III trial enrolled 202 patients and will continue to 52 weeks to further characterize the drug's long-term safety and efficacy profile, with full data to be shared with global health authorities. • Hypoparathyroidism affects over 200,000 people in the US and EU, with approximately 80% being women, representing a significant unmet medical need despite existing treatments.

Merck's Enpatoran Shows Promise for Lupus Rash in Phase 2 Trial Despite Mixed Results

3 months ago

• Merck's oral TLR7/8 inhibitor enpatoran demonstrated clinically meaningful reduction in disease activity for patients with cutaneous lupus erythematosus and systemic lupus erythematosus with active lupus rash in the Phase 2 WILLOW study. • The drug met its primary endpoint in Cohort A with up to 91.3% of patients achieving CLASI-50 response at Week 24, though it failed to meet the primary endpoint in the systemic lupus erythematosus cohort (Cohort B). • As a potential first-in-class oral therapy targeting the TLR7/8 pathway, enpatoran could address significant unmet needs for lupus patients, with Merck now in discussions with health authorities regarding a global Phase 3 program.

TG Therapeutics to Present New BRIUMVI Data in Multiple Sclerosis at ACTRIMS 2025 Forum

3 months ago

• TG Therapeutics will showcase data from ULTIMATE I & II Phase 3 trials and ENHANCE Phase 3b trial evaluating BRIUMVI in relapsing multiple sclerosis at the upcoming ACTRIMS forum in Florida. • Key presentations will focus on the safety and tolerability of 30-minute BRIUMVI infusions, real-world experience data, and distinct disease activity trajectories compared to teriflunomide. • The ULTIMATE trials enrolled 1,094 RMS patients across 10 countries, demonstrating BRIUMVI's potential as a novel CD20-targeting treatment option for multiple sclerosis patients.

Plasmid DNA Manufacturing Market Set to Reach $11.33 Billion by 2034 Amid Gene Therapy Boom

3 months ago

• The global plasmid DNA manufacturing market is projected to grow from $2.64 billion in 2025 to $11.33 billion by 2034, representing a robust CAGR of 17.92%. • North America leads the market with $960 million in 2024, driven by biopharmaceutical industry presence, high disposable income, and advanced healthcare infrastructure. • Artificial intelligence is revolutionizing plasmid DNA production through computational modeling, improving CRISPR/Cas systems, and developing novel vectors for gene therapy applications.

TROPION-Lung12: Phase 3 Trial of Datopotamab Deruxtecan in Early-Stage NSCLC Initiated

4 months ago

• The TROPION-Lung12 phase 3 trial has begun, assessing adjuvant DATROWAY® (datopotamab deruxtecan) with or without rilvegostomig in resected stage 1 NSCLC patients. • The trial targets patients with ctDNA-positive status or high-risk pathological features, aiming to improve outcomes after surgery for early-stage non-small cell lung cancer. • DATROWAY®, a TROP2-directed antibody-drug conjugate, is being evaluated against standard of care to prevent disease recurrence in this high-risk population. • This global, multicenter trial will enroll approximately 660 patients across Asia, Europe, North America, and South America to evaluate disease-free survival.

AI Reshaping Drug Discovery: Nobel-Winning AlphaFold Leads Revolution in Pharmaceutical R&D

4 months ago

• DeepMind's AlphaFold team wins 2024 Nobel Prize in Chemistry for breakthrough AI system that accurately predicts protein structures, marking a significant advancement in drug discovery technology. • AI adoption is expanding across pharmaceutical R&D, with applications ranging from target identification to clinical trial optimization, potentially reducing Phase I development costs from $100M to $70M. • Despite promising advances in virtual cell modeling and generative AI, challenges remain in data scarcity and biological complexity, highlighting the need for balanced integration of AI with traditional research methods.

Datopotamab Deruxtecan's Role in HR+/HER2- Breast Cancer Treatment: Sequencing and Safety Considerations

4 months ago

• Datopotamab deruxtecan (Dato-DXd) is poised to become a standard treatment for metastatic hormone receptor-positive, HER2-negative breast cancer after prior systemic therapy. • Optimal sequencing of antibody-drug conjugates (ADCs) like Dato-DXd, sacituzumab govitecan, and trastuzumab deruxtecan remains uncertain, especially considering overlapping toxicities. • Real-world toxicity profiles suggest sacituzumab govitecan may cause more cytopenias and diarrhea, while Dato-DXd and trastuzumab deruxtecan are linked to interstitial lung disease. • The lack of overall survival difference in the TROPION-Breast01 trial raises questions about efficacy issues or the impact of crossover between treatment arms.

FDA Grants Orphan Drug Designation to Zai Lab's ZL-1310 for Small Cell Lung Cancer

4 months ago

• The FDA has granted Orphan Drug Designation to Zai Lab's ZL-1310, a DLL3-targeted antibody-drug conjugate, for treating small cell lung cancer (SCLC). • This designation provides ZL-1310 with incentives like fee waivers, tax credits, and potential market exclusivity for seven years upon approval. • ZL-1310 is currently being evaluated in a Phase 1a/1b trial as a monotherapy and in combination with atezolizumab for extensive-stage SCLC. • Preliminary data from the trial shows promising objective response rates and a favorable safety profile in patients with recurrent SCLC.

Lerociclib Plus Fulvestrant Shows Promise in HR+/HER2- Advanced Breast Cancer

4 months ago

• The LEONARDA-1 phase 3 trial demonstrated that lerociclib plus fulvestrant significantly improved progression-free survival (PFS) compared to placebo plus fulvestrant in patients with HR+/HER2- advanced breast cancer. • The combination of lerociclib and fulvestrant showed a manageable safety profile, with hematological toxicities being the most common adverse events, effectively managed with dose adjustments. • Lerociclib plus fulvestrant exhibited efficacy across various patient subgroups, including those with primary endocrine therapy resistance and liver metastasis, addressing a critical unmet need. • The study supports lerociclib plus fulvestrant as a potential treatment option for patients with HR+/HER2- advanced breast cancer who have progressed on prior endocrine therapy.

Arvinas Advances Vepdegestrant into Phase 3 Trials for Breast Cancer and Updates Pipeline Milestones

4 months ago

• Arvinas plans to initiate two Phase 3 trials in 2025 for vepdegestrant in ER+/HER2- metastatic breast cancer, one in the first-line setting with atirmociclib and another in the second-line setting with a CDK4/6 inhibitor. • Topline data from the Phase 3 VERITAC-2 monotherapy trial of vepdegestrant in second-line-plus ER+/HER2- metastatic breast cancer is anticipated in the first quarter of 2025. • Arvinas is set to present initial data from the Phase 1 trial of ARV-393 in B-cell lymphomas and file an IND application for a novel PROTAC KRAS G12D degrader in 2025. • Phase 1 trial with PROTAC LRRK2 degrader ARV-102 in patients with Parkinson’s disease has been initiated, with data expected to be presented in the first half of 2025.

Azenosertib Shows Promise in Cyclin E1+ Platinum-Resistant Ovarian Cancer

4 months ago

• Zentalis Pharmaceuticals presented updated clinical data on azenosertib, highlighting its potential in treating Cyclin E1+ platinum-resistant ovarian cancer (PROC). • In the DENALI trial, azenosertib monotherapy demonstrated an objective response rate (ORR) of approximately 35% in heavily pretreated patients with Cyclin E1+ PROC. • The FDA has granted Fast Track Designation to azenosertib for Cyclin E1+ PROC, potentially expediting its development and regulatory review. • Zentalis plans to initiate enrollment for DENALI Part 2 in the first half of 2025, with topline data expected by the end of 2026, aiming for accelerated approval.

Axcynsis Therapeutics' AT03-65 Receives FDA IND Clearance for CLDN6-Targeting ADC

4 months ago

• Axcynsis Therapeutics received FDA clearance for its IND application for AT03-65, a differentiated CLDN6-targeting antibody-drug conjugate (ADC). • AT03-65 leverages Axcynsis' AxcynDOT™ technology for precise drug delivery to tumors expressing CLDN6, a target in various cancers. • This regulatory milestone enables Axcynsis to advance AT03-65 into clinical trials, marking a significant step in developing novel cancer therapies. • The ADC is designed to improve efficacy and reduce systemic toxicity compared to traditional chemotherapy, addressing unmet needs in oncology.

MedMira's Reveal® G4 HIV-1/2 Rapid Test Achieves 100% Specificity in Clinical Trials; Awaits CLIA Waiver

6 months ago

• MedMira's Reveal® G4 Rapid HIV-1/2 antibody test demonstrated 100% sensitivity and specificity in clinical trials, including reactivity in early HIV-2 infections. • The company is in the final phase of U.S. clinical trials to achieve CLIA waived approval for the Reveal® G4 HIV-1/2 rapid test, with an update expected in early 2025. • MedMira has submitted data to Health Canada for regulatory approval of its Reveal® TP (Syphilis) rapid test and two additional rapid tests. • Strategic partnerships have been established in the U.S. to distribute MedMira's current and future product lines, including the Reveal® G4 HIV-1/2 rapid test.

Elutia Inc. Prepares for Full Launch of EluPro Antibiotic-Eluting Envelope After Positive Q3 Results

6 months ago

• Elutia Inc. reported the first commercial use of EluPro, an FDA-cleared antibiotic-eluting biologic envelope for pacemakers and neurostimulators, with full launch slated for 2025. • Q3 financial results showed a slight decrease in overall net sales to $5.9 million, offset by a 19% increase in SimpliDerm sales and a net income improvement of $1.3 million. • The company is expanding its sales force and engaging with group purchasing organizations to broaden EluPro's market reach, supported by clinical studies and published data. • Elutia anticipates a favorable market reception for EluPro, aiming to redefine the BioEnvelope market and improve patient outcomes through strategic partnerships and national availability.

Vitamin D Supplementation Shows No Significant Benefit for COPD Management

8 months ago

• A Cochrane review of 10 trials found that vitamin D supplementation does not reduce the rate of moderate or severe COPD exacerbations requiring systemic corticosteroids or antibiotics. • Vitamin D supplementation likely has no impact on lung function, as measured by FEV1 (% predicted), in COPD patients. • The analysis suggests vitamin D has little to no effect on mortality or quality of life in individuals with COPD, based on low-certainty evidence. • Current evidence does not support routine vitamin D supplementation for managing COPD symptoms or preventing exacerbations in most patients.

Drug Development Updates