ABBVIE | MedPath

REGENXBIO Secures $250 Million Royalty Monetization Deal to Advance Gene Therapy Pipeline

2 days ago

• REGENXBIO has closed a non-dilutive royalty bond agreement with Healthcare Royalty for up to $250 million, receiving $150 million upfront that extends its cash runway into early 2027. • The deal monetizes select royalties from ZOLGENSMA for SMA and payments from gene therapies for MPS disorders, while REGENXBIO retains other funding opportunities including a potential Priority Review Voucher. • This strategic financing supports REGENXBIO's late-stage pipeline development, including RGX-121 for MPS II, RGX-202 for Duchenne muscular dystrophy, and ABBV-RGX-314 for wet AMD.

Robust Pipeline of 50+ Therapies Reshaping Moderate to Severe Atopic Dermatitis Treatment Landscape

3 days ago

• The moderate to severe atopic dermatitis market is experiencing significant growth with 45+ companies developing over 50 pipeline therapies, driven by rising global prevalence and increased awareness of the condition. • Recent advancements include promising biologics like rezpegaldesleukin (granted FDA fast-track designation), JAK inhibitors such as VC005 and QY201, and novel mechanisms targeting IL-4/IL-13 pathways, offering new hope for patients with inadequate response to current treatments. • Key players including Nektar Therapeutics, Corvus Pharmaceuticals, and Apogee Therapeutics are advancing clinical trials with significant milestones expected in late 2025, potentially transforming the therapeutic landscape for this chronic inflammatory skin condition.

AbbVie and ADARx Form $335 Million Collaboration to Develop Next-Generation siRNA Therapies

8 days ago

• AbbVie has invested $335 million upfront in a strategic collaboration with ADARx Pharmaceuticals to develop small interfering RNA therapeutics across neuroscience, immunology, and oncology. • The partnership combines ADARx's proprietary RNA technology for precise mRNA silencing with AbbVie's expertise in antibody engineering, ADCs, and tissue delivery approaches to overcome siRNA targeting challenges. • Beyond the initial payment, ADARx is eligible to receive billions in additional contingent payments including milestone payments and tiered royalties as the collaboration advances.

Royalty Pharma Secures 99.9% Shareholder Approval for $1.1 Billion External Manager Acquisition

9 days ago

• Royalty Pharma shareholders have overwhelmingly approved the acquisition of its external manager, with 99.9% voting in favor at the company's 2025 Annual General Meeting. • The $1.1 billion transaction will transition Royalty Pharma from an external management model to an integrated corporate structure, generating projected cash savings exceeding $1.6 billion over ten years. • The internalization is expected to strengthen corporate governance, enhance transparency, and better align leadership with shareholder interests while ensuring management continuity.

Leap Therapeutics Halves Workforce and Refocuses Cancer Drug Development Amid Market Challenges

13 days ago

• Leap Therapeutics has announced a significant restructuring, reducing its workforce by approximately 50% and narrowing the development focus of its lead cancer drug candidate in response to challenging market conditions. • The strategic pivot aims to extend the company's cash runway while concentrating resources on the most promising clinical applications of its lead oncology asset, potentially improving its chances for regulatory success. • This move follows similar restructuring trends across the biotech sector, with companies like Arcturus, NGM Bio, and Erasca all recently announcing staff reductions and pipeline reprioritizations to navigate the difficult funding environment.

Balancing Efficacy and Cost Sustainability in Modern CLL Treatment: Insights from Dr. Pierluigi Porcu

a month ago

• Clinicians treating chronic lymphocytic leukemia (CLL) face growing challenges in balancing clinical efficacy with long-term cost sustainability, requiring a holistic approach to patient care. • Dr. Pierluigi Porcu emphasizes that effective CLL management requires understanding disease risk, patient comorbidities, and practice environment constraints including payer considerations. • Despite its importance, value-based care adoption remains insufficient in oncology, with challenges in defining and measuring value across patient outcomes, quality of life, and treatment costs.

Roche Expands Molecular Glue Portfolio with $2 Billion Monte Rosa Partnership

2 months ago

• Roche has signed a $2 billion partnership with Monte Rosa Therapeutics, including a $50 million upfront payment, to develop molecular glue therapies targeting previously "undruggable" proteins in cancer and neurological diseases. • This marks Roche's second major molecular glue deal in a month, following a similar $2 billion alliance with Orionis Biosciences, demonstrating the company's strategic push to establish leadership in this emerging therapeutic category. • Monte Rosa will lead discovery and preclinical activities using its QuEEN platform, with Roche taking over late-stage development of candidates that can potentially address the 80% of human proteins currently inaccessible to traditional drug development approaches.

Global Clinical Trials for Myelodysplastic Syndrome Show Significant Industry Investment in 2025

3 months ago

• A comprehensive review of global Myelodysplastic Syndrome clinical trials reveals extensive research activity across G7 and E7 countries, with major pharmaceutical companies leading development efforts. • The analysis highlights significant participation from industry leaders including Bristol-Myers Squibb, Novartis, and Sanofi, demonstrating strong commercial interest in MDS therapeutics. • The report indicates evolving clinical trial landscapes across multiple regions, with detailed tracking of trial phases, enrollment trends, and success rates over the past five years.

Phase 4 KINECT-PRO Trial Shows Ingrezza Significantly Improves Quality of Life in Tardive Dyskinesia Patients

3 months ago

• Neurocrine Biosciences' phase 4 KINECT-PRO study demonstrates sustained improvements in physical, social, and emotional impacts of tardive dyskinesia with Ingrezza treatment across all severity levels. • The 24-week trial showed significant patient-reported benefits starting as early as 4 weeks at the lowest dose (40mg), with 52 out of 59 enrolled patients completing the study. • Results revealed consistent efficacy regardless of underlying psychiatric conditions, with improvements measured across multiple validated clinical scales including AIMS and TD Impact Scale.

Novel Oral Psoriasis Therapies Show Promise: TYK2 Inhibitors Lead Breakthrough Advances

3 months ago

• Deucravacitinib demonstrates superior efficacy over apremilast in psoriasis treatment, with over 53% of patients achieving significant skin clearance at week 16. • Apremilast maintains its position as a key therapy for mild-to-moderate psoriasis, with 21% of bio-naïve patients achieving treatment goals in the ADVANCE trial. • Next-generation oral therapies, including JNJ-77242114 and zasocitinib, show promising results in clinical trials, potentially expanding treatment options for psoriasis patients.

FDA Approves Lilly's Omvoh (mirikizumab) for Crohn's Disease

4 months ago

• The FDA has approved Eli Lilly's Omvoh (mirikizumab-mrkz) for treating moderately to severely active Crohn's disease in adults, expanding its use for inflammatory bowel disease. • The approval was based on the VIVID-1 trial, which showed significant clinical remission and endoscopic response rates compared to placebo after one year of treatment. • Omvoh is the first biologic in over 15 years with Phase 3 two-year efficacy data at launch, demonstrating long-term disease control and visible healing of the intestinal lining. • Lilly has submitted marketing applications globally, aiming to provide a new treatment option for Crohn's patients and is working with insurers to enable patient access.

AbbVie's Telisotuzumab Vedotin Shows Promise in c-Met Overexpressing NSCLC

5 months ago

• AbbVie's telisotuzumab vedotin (Teliso-V) demonstrated a 35% overall response rate in patients with high c-Met expression in the LUMINOSITY trial. • The FDA granted breakthrough therapy designation to Teliso-V, highlighting its potential to significantly improve outcomes in NSCLC patients. • A phase 3 trial (TeliMET-NSCLC-01) is underway, comparing Teliso-V to docetaxel in c-Met-positive, non-squamous NSCLC patients. • Teliso-V targets c-Met, a protein involved in cancer progression and resistance to therapies like EGFR inhibitors, offering a novel approach.

FDA Approves Roche's Companion Diagnostic for HER2-Ultralow Metastatic Breast Cancer

5 months ago

• The FDA has approved Roche's PATHWAY HER2 (4B5) test to identify HER2-ultralow metastatic breast cancer patients for targeted treatment. • This approval expands treatment options for approximately 20-25% of HR-positive, HER2-negative breast cancer patients with HER2-ultralow status. • The DESTINY-Breast06 trial demonstrated that ENHERTU improved median progression-free survival compared to chemotherapy in HER2-low and HER2-ultralow patients. • Roche's diagnostic test standardizes HER2 assessment, reduces errors, and helps clinicians make informed treatment decisions for improved patient outcomes.

FDA Approves Tevimbra-Chemotherapy Combination for First-Line Treatment of Advanced Esophageal Squamous Cell Carcinoma

5 months ago

• The FDA has approved BeiGene's Tevimbra (tislelizumab-jsgr) in combination with platinum-containing chemotherapy for first-line treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma whose tumors express PD-L1. • In the pivotal RATIONALE-306 trial, patients treated with Tevimbra plus chemotherapy demonstrated a median overall survival of 16.8 months compared to 9.6 months with chemotherapy alone, representing a 34% reduction in risk of death. • This marks BeiGene's third FDA approval in less than a year, following previous approvals for Tevimbra in second-line ESCC and first-line gastric/gastroesophageal junction cancers, highlighting the company's expanding oncology portfolio.

Pharma Stock Roundup: Novo Nordisk's Obesity Study Disappoints, Lilly's Zepbound Approved for Sleep Apnea

5 months ago

• Novo Nordisk's CagriSema obesity treatment showed a 22.7% weight loss in Phase III, falling short of the anticipated 25% reduction, impacting stock prices. • Eli Lilly's Zepbound (tirzepatide) received FDA approval for moderate-to-severe obstructive sleep apnea (OSA), marking it as the first drug for obesity-related OSA. • AstraZeneca and Daiichi Sankyo withdrew their EU application for Dato-DXd in non-small cell lung cancer (NSCLC) but pursue U.S. approval for EGFR-mutated NSCLC. • Pfizer's Braftovi gained FDA approval in combination with Erbitux and mFOLFOX6 for first-line treatment of metastatic colorectal cancer with BRAF mutations.

Ubrelvy Shows Promise in Halting Migraines at Earliest Signs

9 months ago

• A clinical trial reveals that ubrogepant (Ubrelvy) may effectively stop migraines when taken at the first signs of an attack. • Participants taking ubrogepant were 73% more likely to report no disability and normal function compared to those on a placebo. • 65% of patients taking ubrogepant reported minimal migraine symptoms 24 hours later, versus 48% in the placebo group. • The drug targets CGRP, a protein instrumental in migraine processes, offering a proactive approach to migraine management.

CytomX Therapeutics Reports Promising Interim Phase 1 Data for CX-2051 Antibody-Drug Conjugate

a year ago

• CytomX Therapeutics has announced encouraging interim Phase 1 data for CX-2051, their novel antibody-drug conjugate targeting EpCAM-expressing solid tumors, showing early signs of clinical activity. • The dose-escalation study demonstrated a manageable safety profile with dose-limiting toxicities observed only at higher dose levels, supporting continued development of the therapeutic candidate. • Preliminary efficacy signals were observed across multiple tumor types, with several patients achieving stable disease, suggesting potential clinical benefit in difficult-to-treat EpCAM-positive malignancies.