ASTELLAS PHARMA EUROPE LTD.

CytomX Doses First Patient in Combination Trial of CX-801 with Keytruda for Metastatic Melanoma

3 days ago

• CytomX Therapeutics has dosed the first patient in a Phase 1 trial combining its masked interferon alpha-2b (CX-801) with Merck's Keytruda in patients with metastatic melanoma. • The novel combination aims to address the high unmet need in PD-1 refractory melanoma by localizing interferon's potent immune-stimulating effects to tumors while minimizing systemic toxicities. • Initial Phase 1a translational and biomarker data from this study are expected in the second half of 2025, potentially advancing a new approach in combination immuno-oncology therapy.

Astellas to Present New Long-Term Survival Data for Cancer Therapies at ASCO 2025

3 days ago

• Astellas will showcase 16 abstracts at the 2025 ASCO Annual Meeting, including two oral presentations highlighting long-term survival data for its oncology portfolio. • New post-hoc analyses will feature five-year overall survival data for XTANDI (enzalutamide) in metastatic hormone-sensitive prostate cancer and exploratory analyses for PADCEV (enfortumab vedotin) in urothelial cancer. • The presentations underscore Astellas' commitment to delivering meaningful clinical outcomes for patients with advanced prostate and bladder cancers through innovative treatment approaches.

US Orphan Drug Market Set to Exceed $190 Billion by 2030 as FDA Designations Accelerate

8 days ago

• The US orphan drug market is projected to surpass $190 billion by 2030, with over 7,300 molecules receiving FDA Orphan Drug Designation to date, of which approximately 17.9% have gained approval. • Since 2020, more than half of all new drug approvals by the FDA's Center for Drug Evaluation and Research have been granted orphan status, highlighting the growing importance of rare disease treatments in pharmaceutical development. • Despite criticism over high pricing, exemplified by Abeona Therapeutics' Zevaskyn at $3.1 million per treatment, the orphan drug model has evolved into a sound business strategy offering fewer competitors, faster approvals, and seven-year market exclusivity.

GSK and iTeos Terminate TIGIT Cancer Drug Development After Phase 2 Failure

9 days ago

• GSK and iTeos Therapeutics have discontinued development of belrestotug, their TIGIT-targeting immunotherapy, after it failed to significantly delay tumor progression in non-small cell lung cancer patients. • The companies are terminating all ongoing trials, including a Phase 3 study, ending their four-year collaboration that began with GSK's $625 million upfront payment and potential $1.45 billion in milestone payments. • iTeos is now conducting a strategic review to preserve capital, as the failure eliminates its most advanced drug candidate and represents another setback for TIGIT-targeting therapies in oncology.

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.

CytomX's CX-2051 Shows Promising 28% Response Rate in Advanced Colorectal Cancer Phase 1 Trial

10 days ago

• CytomX's EpCAM-targeting antibody-drug conjugate CX-2051 demonstrated a 28% confirmed response rate in late-line colorectal cancer patients, significantly outperforming current standard therapies with single-digit response rates. • The novel PROBODY® masking technology appears to successfully target EpCAM, a previously "undruggable" tumor antigen, with manageable safety profile and no dose-limiting toxicities observed in the Phase 1 study. • Median progression-free survival reached 5.8 months with 43% response rate at the highest dose level (10 mg/kg), supporting plans to advance to Phase 2 trials in first half of 2026.

Boehringer Ingelheim Launches Phase II Trial of First-in-Class Oral Treatment for Geographic Atrophy

16 days ago

• Boehringer Ingelheim has initiated the JADE Phase II clinical study to evaluate BI 1584862, a potential first-in-class oral treatment for geographic atrophy, a severe form of age-related macular degeneration. • Geographic atrophy affects over 5 million people worldwide with more than 40% considered legally blind, creating a significant unmet need for treatments that can slow disease progression and preserve vision. • The company is simultaneously advancing a second compound, BI 771716, an antibody fragment designed to penetrate retinal layers, demonstrating Boehringer's dual-pathway approach to addressing this debilitating eye condition.

Dyno Therapeutics Unveils Three AI-Designed AAV Capsids for Enhanced Gene Therapy Delivery

23 days ago

• Dyno Therapeutics has launched three breakthrough AAV capsid vectors targeting the eye, muscle, and central nervous system, each demonstrating significant improvements over conventional AAV vectors in non-human primates. • The new capsids—Dyno-4z2, Dyno-3hv, and Dyno-ahq—show up to 280-fold increased delivery efficiency, enhanced target specificity, and improved manufacturability, potentially addressing key limitations in current gene therapy approaches. • These AI-designed capsids are now available for licensing to partners developing gene therapies for ophthalmological, neuromuscular, and neurological disorders, with data presented at the 28th ASGCT Annual Meeting.

Pfizer to Showcase Breakthrough Cancer Therapies at ASCO 2025 with Over 60 Presentations

a month ago

• Pfizer will present data from more than 60 abstracts at ASCO 2025, including 15 oral presentations highlighting advancements across breast, genitourinary, hematologic, thoracic, and colorectal cancers. • Two late-breaking presentations will be featured in ASCO's press program, including pivotal survival data for BRAFTOVI in colorectal cancer and progression-free survival results for vepdegestrant in metastatic breast cancer. • The company will showcase novel antibody-drug conjugate combinations with immunotherapy and present five-year survival data for XTANDI in prostate cancer, supporting Pfizer's goal of delivering eight breakthrough cancer medicines by 2030.

Five-Year Study Shows Aflibercept Treat-and-Extend Regimen Maintains Vision in Neovascular AMD Patients

2 months ago

• A multicenter Japanese study demonstrates that aflibercept using a treat-and-extend regimen maintained baseline visual acuity and significantly reduced central macular thickness in neovascular AMD patients over five years. • Researchers identified smaller greatest linear dimension (GLD) as the only independent risk factor associated with better vision outcomes after five years of treatment (p=0.0024). • The study revealed patients required fewer injections after the first year, though 33% of discontinued cases experienced recurrence, with higher recurrence rates in those who had received more total injections.

Biotech Deal Landscape: February-March 2025 Sees Surge in Partnerships Across Multiple Therapeutic Areas

2 months ago

• The first quarter of 2025 witnessed significant biotech partnership activity, with Eli Lilly, AstraZeneca, and Novo Nordisk emerging as top collaborators in deals worth billions across small molecules, antibodies, and RNA therapeutics. • February 2025 featured notable acquisitions including Novartis's $2.15 billion buyout of Anthos Therapeutics, while March saw AstraZeneca acquire Belgian biotech EsoBiotec and Bristol Myers Squibb purchase 2seventy bio for $286 million. • Obesity therapeutics gained significant traction in March 2025, with AbbVie entering the field through a $350 million upfront deal with Gubra for an amylin analog, while Roche partnered with Zealand Pharma on petrelintide in a deal worth up to $5.25 billion.

Merck Halts Final Phase 3 Keytruda Trial in Prostate Cancer After Failed Interim Analysis

3 months ago

• Merck discontinues KEYNOTE-641 trial evaluating Keytruda plus Xtandi in metastatic castration-resistant prostate cancer after showing no survival benefit at interim analysis. • The failure marks Merck's fourth unsuccessful late-stage trial of Keytruda in prostate cancer, highlighting the challenges of immunotherapy in this typically "cold" tumor type. • The company simultaneously reported an unexpected setback in KEYNOTE-789 studying Keytruda combination therapy in EGFR-mutated non-small cell lung cancer.

AstraZeneca Acquires FibroGen China for $160 Million, Strengthening Roxadustat Position

3 months ago

• FibroGen has agreed to sell its China subsidiary to AstraZeneca for approximately $160 million, comprising $85 million in enterprise value plus an estimated $75 million in net cash holdings. • The strategic sale will extend FibroGen's cash runway into 2027 and enable continued development of its oncology pipeline, including the Phase 2 trial of FG-3246 for metastatic prostate cancer. • Upon closing in mid-2025, AstraZeneca will acquire all rights to roxadustat in China, where it leads the market for chronic kidney disease anemia treatment.

Phase II Trial Shows Breakthrough 63.6% Response Rate for Novel Bladder Cancer Combination Therapy

3 months ago

• Disitamab Vedotin combined with Toripalimab achieved a 63.6% pathological complete response rate in muscle-invasive bladder cancer patients, nearly doubling traditional chemotherapy outcomes. • The clinical trial demonstrated strong efficacy across all HER2 expression levels, with HER2 IHC 3+ patients showing an exceptional 84.6% complete response rate. • The treatment showed favorable safety with only 27.7% grade 3 or higher adverse events, significantly lower than conventional chemotherapy's 40-50% rate.

FDA Approves Lumakras and Vectibix Combination for KRAS G12C-Mutated Metastatic Colorectal Cancer

4 months ago

• The FDA has approved Lumakras (sotorasib) combined with Vectibix (panitumumab) for treating KRAS G12C-mutated metastatic colorectal cancer in adults after prior chemotherapy. • CodeBreaK 300 trial data showed the combination significantly improved progression-free survival compared to standard of care in chemorefractory patients. • Patients on Lumakras and Vectibix had a median progression-free survival of 5.6 months, compared to 2 months on standard of care, with a 26% overall response rate. • This approval provides a new targeted treatment option for a subset of colorectal cancer patients with limited alternatives, emphasizing the importance of biomarker testing.

J&J Seeks FDA Approval for TAR-200 in BCG-Unresponsive Non-Muscle Invasive Bladder Cancer

4 months ago

• Johnson & Johnson has initiated a New Drug Application (NDA) submission to the FDA for TAR-200. • TAR-200 is intended for patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC). • Phase 2b SunRISe-1 study data showed an 83.5% complete response rate with TAR-200 monotherapy. • The FDA is reviewing the application under the Real-Time Oncology Review (RTOR) program to expedite potential approval.

Bayer's Elinzanetant Shows Promise in Reducing Hot Flashes for Breast Cancer Patients

4 months ago

• Elinzanetant met primary endpoints in the Phase III OASIS 4 trial, significantly reducing the frequency of moderate to severe vasomotor symptoms (VMS) in women undergoing endocrine therapy for breast cancer. • The study also achieved secondary endpoints, demonstrating a reduction in the severity of VMS, improvements in sleep disturbances, and enhanced menopause-related quality of life compared to placebo. • Elinzanetant is a dual neurokinin-1 and 3 receptor antagonist, representing a potential non-hormonal treatment option for VMS in breast cancer patients and women at high risk. • Bayer is advancing regulatory submissions for elinzanetant in the US, EU, and other global markets, with potential launch expected later this year, pending regulatory approvals.

EMA Begins Review of Xtandi for Hormone-Sensitive Prostate Cancer

5 months ago

The European Medicines Agency (EMA) has initiated a review of Pfizer and Astellas' prostate cancer drug, Xtandi, for a new indication that could expand its use to metastatic hormone-sensitive prostate cancer (mHSPC) patients, based on the phase 3 ARCHES and ENZAMET trials. This move could intensify competition with Johnson & Johnson's Zytiga, which was approved for mHSPC last year in the US.

Cytokinetics and Pyxis Oncology Announce Key Milestones for 2025; CytomX Prioritizes Pipeline

5 months ago

• Cytokinetics anticipates a pivotal year in 2025, focusing on the potential FDA approval and commercial launch of aficamten for obstructive hypertrophic cardiomyopathy (HCM) in the U.S. • Pyxis Oncology is prioritizing the advancement of PYX-201, a novel antibody-drug conjugate (ADC), with data readouts expected in the second half of 2025 and the first half of 2026. • CytomX Therapeutics is focusing on its lead program, CX-2051, an EpCAM-targeting PROBODY ADC for advanced metastatic colorectal cancer, with initial Phase 1a data expected in 1H 2025.

FDA Rejects Astellas' Label Update for Geographic Atrophy Drug Izervay

6 months ago

• The FDA issued a complete response letter for Astellas' Izervay label update, which included data from the Phase 3 GATHER2 trial, impacting its competitive positioning. • The FDA's rejection was based on a statistical matter related to the proposed labeling language, with no concerns raised regarding the drug's safety or efficacy data. • This setback follows Astellas' withdrawal of Izervay's marketing application in the EU, further complicating the drug's global market access. • Izervay, a complement C5 inhibitor, is currently approved in the US for geographic atrophy secondary to age-related macular degeneration.