Gilead’s TRODELVY Demonstrates Significant PFS Benefit in First-Line Metastatic TNBC in ASCENT-03 Trial
Gilead Sciences has announced positive topline results from the Phase III ASCENT-03 trial of TRODELVY (sacituzumab govitecan-hziy) in first-line metastatic triple-negative breast cancer (mTNBC) patients who are ineligible for PD-1/PD-L1 inhibitor therapies. The study met its primary endpoint by showing a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to chemotherapy. This is the first notable advance for this patient group in over two decades, underscoring TRODELVY’s potential as a new standard of care.
The safety profile of TRODELVY was consistent with previous studies, with no new safety signals reported. Overall survival, a secondary endpoint, was not mature at the time of analysis, though no negative impact was observed. Gilead plans further monitoring and will present detailed findings at a future medical meeting. Regulatory discussions are also planned as the company seeks potential approval for this new indication.
The ASCENT-03 results follow the recent success of the ASCENT-04 trial, which evaluated TRODELVY in combination with KEYTRUDA in PD-L1+ mTNBC. Together, the trials suggest TRODELVY may be an effective foundational treatment across the full spectrum of first-line mTNBC. With four positive Phase III trials in HER2-negative metastatic breast cancer, TRODELVY remains the only approved Trop-2-directed antibody-drug conjugate (ADC) to show survival benefits across both mTNBC and HR+/HER2- mBC.
“Almost half of the patients diagnosed with metastatic triple-negative breast cancer do not receive treatment beyond first-line, demonstrating an urgent need for innovative treatment options in this early setting,” said Dr. Javier Cortes, Head of the International Breast Cancer Center in Spain and principal investigator of the ASCENT-03 study. “Traditional chemotherapy has been the standard of care, and we know that therapeutic advances in this disease area serve a critical unmet need.”
“The ASCENT-03 outcome represents the first clinically meaningful advance for this patient population in over 20 years versus chemotherapy,” said Dr. Dietmar Berger, Chief Medical Officer, Gilead Sciences. “By addressing this aggressive and difficult-to-treat disease earlier, we can potentially improve treatment options for the high unmet need that patients with metastatic triple-negative breast cancer face.”
Otsuka’s Sibeprenlimab Receives FDA Priority Review for IgA Nephropathy
Otsuka Pharmaceutical and Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC) announced that the FDA has accepted for review the Biologics License Application (BLA) for sibeprenlimab, an investigational monoclonal antibody that selectively inhibits APRIL (A PRoliferation-Inducing Ligand) for adults with immunoglobulin A nephropathy (IgAN). The BLA has been granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2025.
“Over the past decade, Otsuka has consistently approached difficult-to-treat diseases in nephrology with scientific and clinical innovation, seeking to provide crucial advancements for underserved patients with complex conditions like IgA nephropathy,” said John Kraus, M.D., Ph.D., executive vice president and chief medical officer, Otsuka Pharmaceutical Development & Commercialization, Inc. “If approved, sibeprenlimab would enable individuals living with IgA nephropathy to self-inject once every 4 weeks. We are thankful to share a potential treatment that could offer important clinical benefits and convenience to those living with this disease.”
The BLA submission is supported by data from the Phase III VISIONARY trial (NCT05248646), which met its primary endpoint at the prespecified interim analysis, and the Phase II ENVISION trial (NCT04287985). In Phase III, sibeprenlimab showed a statistically significant and clinically meaningful reduction in 24-hour uPCR after nine months of treatment compared to placebo.
Sibeprenlimab is designed for self-administration as a single-dose prefilled syringe for subcutaneous injection every four weeks. The therapy also holds Breakthrough Therapy designation based on Phase II results and offers a novel approach by targeting APRIL, a key driver in the 4-hit pathogenesis of IgAN that can lead to end-stage kidney disease.
Liquidia Secures FDA Approval for YUTREPIA Inhalation Powder in PAH and PH-ILD
Liquidia Corporation announced that the FDA has approved YUTREPIA (treprostinil) inhalation powder for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) to improve exercise ability. YUTREPIA is the first and only dry-powder prostacyclin formulation enabled by Liquidia’s proprietary PRINT™ technology, engineered for deep-lung delivery using a portable, low-effort inhalation device.
“Today, we celebrate for the patients and physicians who will now have access to a potential best-in-class dry-powder form of treprostinil with exceptional portability, tolerability, titratability, and durability,” said Dr. Roger Jeffs, CEO of Liquidia. “Thank you to the clinical investigation team, our steering committee, and the members of the pulmonary hypertension patient communities who helped make this day a reality. With today’s milestone, our commercial team is prepared to launch YUTREPIA and bring meaningful change to the lives of patients in need, and we look forward to speaking with physicians and patients about the unique benefits of YUTREPIA in the days and weeks ahead.”
FDA approval was supported by results from the Phase III INSPIRE trial, which showed that YUTREPIA was safe and well-tolerated in both treprostinil-naïve patients and those transitioning from nebulized formulations. Findings from the study have been published in the Pulmonary Circulation and Vascular Pharmacology journals. The inhalation powder provides a promising option for over 105,000 patients in the U.S. living with PAH and PH-ILD.
“I am so pleased that patients with PAH and PH-ILD now have this newly introduced option for inhaled treprostinil,” said Dr. Nicholas Hill, Chief of Pulmonary, Critical Care & Sleep Division at Tufts University School of Medicine and Principal Investigator of the INSPIRE trial. “Having treated patients for more than six years in Liquidia’s INSPIRE and extension studies, I am confident in the safety, tolerability, and dosing that YUTREPIA offers. The low-effort inhalation device used to deliver YUTREPIA may make it easier to start and maintain patients on treatment, especially those with limited inspiratory flows or lung capacity.”
Matt Granato, President and CEO of the Pulmonary Hypertension Association, also welcomed the news. He highlighted the significant burden of these conditions and the need for diverse therapeutic tools for physicians and patients alike.
“PAH and PH-ILD impact more than 105,000 patients in the U.S. alone. These patient communities and the physicians who serve them need therapies that can lead to the improvement of quality of life,” said Granato. “We are always glad to see industry research leading to the development of drugs that expand options for the patient community.”
However, legal uncertainty clouds the commercial rollout. United Therapeutics Corporation (UTHR) filed a complaint on May 9, 2025, alleging that YUTREPIA infringes U.S. Patent No. 11,357,782 and requested a temporary restraining order and preliminary injunction to halt its commercialization. Oral arguments were heard on May 20, 2025, and the motion is currently pending in court.
GSK’s BLENREP Combinations Receive Positive CHMP Opinion for Relapsed/Refractory Multiple Myeloma
In May 2025, GSK plc announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the approval of BLENREP (belantamab mafodotin) for the treatment of adults with relapsed or refractory multiple myeloma. The recommendation includes BLENREP in combination with bortezomib plus dexamethasone (BVd) for patients who have received at least one prior therapy, and with pomalidomide plus dexamethasone (BPd) for those previously treated with lenalidomide. A final decision by the European Commission is expected in Q3 2025.
“Today’s positive CHMP opinion is an important milestone toward bringing the benefits of BLENREP combinations to patients with multiple myeloma in Europe,” said Hesham Abdullah, Senior Vice President, Global Head Oncology, R&D, GSK. “BLENREP is well-positioned to address the unmet needs of these patients while also providing the benefit of in-office administration in both academic and community treatment settings without complex pre-administration regimens or hospitalization.”
The CHMP opinion is backed by strong efficacy data from DREAMM-7 and DREAMM-8, two pivotal Phase III clinical trials. These studies demonstrated statistically significant and clinically meaningful improvements in progression-free survival (PFS) compared to standard of care. DREAMM-7 also showed overall survival (OS) benefit against a daratumumab-based triplet regimen. Safety and tolerability profiles of BLENREP combinations remained consistent with those of the individual agents.
There are around 50K new multiple myeloma cases diagnosed annually in Europe, and nearly all patients relapse after initial treatment. BLENREP is currently the only anti-BCMA antibody-drug conjugate (ADC) approved for this population, offering a differentiated mechanism of action. Its administration flexibility allows treatment across a wide range of oncology settings.
BLENREP-related eye side effects observed in the trials were manageable and reversible with dose adjustments and monitoring, contributing to low discontinuation rates (≤9%) in both studies. In DREAMM-7, common non-ocular adverse events included thrombocytopenia (87%) and diarrhoea (32%), while neutropenia (63%), thrombocytopenia (55%), and COVID-19 (37%) were reported in DREAMM-8.
BLENREP combinations are also under regulatory review in the U.S. (PDUFA date: July 23, 2025), China (with Breakthrough Therapy Designation and priority review), Canada, and Switzerland. If approved, these combinations could significantly expand treatment options for patients experiencing early relapse.
Relief Therapeutics Earns FDA Rare Pediatric Disease Designation for RLF-TD011
Relief Therapeutics Holding SA announced that the FDA has granted Rare Pediatric Disease (RPD) designation to its investigational therapy RLF-TD011 for the treatment of epidermolysis bullosa (EB), a rare genetic disorder causing fragile skin and chronic wounds. The FDA had previously granted Orphan Drug Designation to RLF-TD011 for the same indication.
“The FDA’s decision to grant Rare Pediatric Disease designation to RLF-TD011 underscores both the critical need for new options for patients living with EB and the potential of our investigational therapy,” said Giorgio Reiner, Chief Scientific Officer of Relief. “We look forward to continued engagement with the FDA and to sharing the next steps in our development plan following our upcoming pre-IND meeting.”
RLF-TD011 is a hypotonic acid-oxidizing solution containing hypochlorous acid, engineered to offer potent antimicrobial and anti-inflammatory properties while supporting wound healing by maintaining a healthy microenvironment. Previous clinical findings have shown RLF-TD011’s ability to reduce pathogenic colonization without harming beneficial bacteria, addressing key challenges in EB wound care.
The Rare Pediatric Disease designation is granted to treatments targeting serious or life-threatening conditions affecting fewer than 200,000 individuals under age 18 in the U.S. Therapies that receive marketing approval under this designation may qualify for a Priority Review Voucher (PRV), a valuable asset that can expedite FDA review for another product. Notably, in May 2025, a PRV linked to a different EB treatment was sold for $155 million.
