Reported Q2 revenues of $720 million and adjusted EBITDA of $184 million, with confidence in raising 2025 guidance.
Emphasized diversified portfolio and multiple growth drivers including new product launches, complex medicines, biosimilars, and strategic partnerships.
Projected continued growth with a focus on innovative and affordable medicines, aiming to be America's #1 affordable medicines company.
FDA Label Expansion for EMPAVELI in C3G and Primary IC-MPGN
FDA approved a label expansion for EMPAVELI for patients 12 years and older with C3 myelopathy and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
This is the company's third FDA approval in 4 years, highlighting scientific strength and regulatory success.
The approval enables treatment with a C3-targeting therapy that achieves outcomes across proteinuria reduction, eGFR stabilization, and C3 deposit clearance.
Launch is underway, expanding into rare nephrology and underserved populations, including pediatric patients and post-transplant recurrence.
Successful U.S. Launch of AMVUTTRA for ATTR-CM and Early Market Penetration
Alnylam reported a rapid and broad uptake of AMVUTTRA in the U.S. within just the first full quarter post-launch, with approximately 1,400 patients treated by June 30, 2025.
The launch has exceeded initial expectations, with coverage and access achieved faster than anticipated across major health systems and payers.
Nearly all of the 170 priority health systems now have formulary access to AMVUTTRA, enabling treatment initiation in a majority of ATTR-CM patients.
Early utilization data shows a balanced mix of first-line and stabilizer-progressing patients, with prescriber base tripling quarter-over-quarter, indicating strong physician confidence.
Successful Launch and Early Adoption of YUTREPIA in 11 Weeks
Liquidia's new inhaled prostacyclin, YUTREPIA, achieved over 900 prescriptions and 550 patient starts within just 11 weeks of launch, indicating an unprecedented market response.
The launch was executed with high precision and purpose, leading to rapid adoption across both specialty centers and community practices.
The product's ease of use, tolerability, and dose escalation capabilities have been highly praised by physicians and patients, surpassing initial expectations.
Market access strategies, including co-pay assistance and free vouchers, contributed to a 75% script-to-start conversion rate in the first six weeks.
The early momentum was achieved despite typical market entry barriers, suggesting strong unmet needs and product differentiation.
Nektar reported highly compelling initial data for rezpegaldesleukin (REZPEG) in atopic dermatitis, establishing it as a potential first-in-class T regulatory cell (Treg) therapy.
The company is pursuing a novel approach to stimulate Tregs via the IL-2 pathway, using native IL-2 with PEGylation, differentiating from competitors focusing on muteins.
Fast Track designation from FDA for both atopic dermatitis and alopecia areata underscores the unmet need and regulatory support for this mechanism.
REZPEG's mechanism aims to restore immune homeostasis by balancing T effector and Treg cells, addressing autoimmune and inflammatory disorders.
The company plans to enter Phase III in 2026, with a broad label targeting both biologic-naive and experienced patients, aiming to capture a significant market share.
Disappointing Phase III Hyalofast Trial Results and Impact on FDA Submission Timeline
The U.S. pivotal Phase III trial for Hyalofast missed its primary endpoints, with statistical significance not achieved on co-primary measures of KOOS Pain and IKDC Function.
The trial faced challenges due to declining use of microfracture, higher dropout rates, missed visits during COVID, and reduced evaluable sample size.
Despite the miss on primary endpoints, secondary endpoints showed statistical significance, and international data with positive 15-year outcomes support the product's value.
The company plans to submit the third and final PMA module in the second half of 2025, extending the review timeline to 2027 to ensure thorough review and dialogue with the FDA.
Innovative Tissue-Selective Weight Loss Drug Demonstrates Promising Phase 2b Results
Veru's drug candidate Inovasaram has shown in Phase 2b that it can selectively target fat tissue while preserving lean mass in older patients, a significant advancement over existing GLP-1 therapies.
The Phase 2b trial demonstrated a 42% greater fat loss with the 6 mg dose of Inovasaram combined with semaglutide, with statistical significance (p=0.017).
Inovasaram preserved 100% of lean mass during the trial, reducing the risk of muscle loss associated with weight management in older adults.
Physical function, measured by stair climb test, was preserved in patients treated with Inovasaram, with a 59.8% reduction in physical decline compared to placebo.
Positive safety profile was confirmed in the maintenance extension study, with no significant adverse events related to muscle or bone health.
The company plans to develop a novel modified release oral formulation of Inovasaram, protected by patents through 2037, to improve chronic weight management.
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