Progress and Expectations for ARCT-032 Cystic Fibrosis Program
The company is enrolling adult CF patients in a Phase II trial with inhaled ARCT-032, targeting non-responders to CFTR modulators.
Enrollment of the second cohort at 10-milligram dose is expected to complete in early September 2025, with interim data anticipated in September.
The trial focuses on a challenging patient population with severe unmet medical needs, emphasizing safety and lung function improvements.
Regulatory discussions with the FDA are planned for the first half of 2026 to align on pivotal trial design, including adolescent and pediatric enrollment.
The company highlights the potential for inhaled mRNA therapy to be a significant breakthrough for CF non-responders, differentiating from systemic small molecule modulators.
Dosing at 280 mg over 28 days is a novel approach, supported by proprietary delivery technology and drug purification methods.
Regulatory Progress and BLA Submission Strategy for INO-3107
Inovio remains on track to submit its BLA for INO-3107 in the second half of 2025, with a goal of file acceptance by year-end.
The company has completed the design verification testing of the CELLECTRA 5PSP device, a key regulatory milestone.
Inovio has requested a rolling submission of its BLA under breakthrough therapy designation, aiming for a 6-month review period and a potential PDUFA date around mid-2026.
The company successfully completed an FDA inspection of its clinical trial operations, an important step in regulatory compliance.
Differences in trial design and technology, such as DNA medicine versus viral vectors, distinguish INO-3107 from competitors' programs.
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.
Emi-Le Clinical Data Highlights and Tumor Response Rates
Mersana reported a 31% objective response rate (ORR) in evaluable patients with B7-H4 high tumor expression at ASCO 2025, with doses ranging from 38 to 67 mg/m².
In adenoid cystic carcinoma type 1 (ACC1), 4 confirmed responses and 1 unconfirmed response were observed among 9 evaluable patients, with a confirmed ORR of 56% after data cutoff.
The company emphasized Emi-Le's potential to address unmet needs in triple-negative breast cancer (TNBC) patients previously treated with topoisomerase 1 inhibitors, highlighting poor outcomes with current standard of care.
Data from the TNBC expansion cohort showed a 29% ORR in B7-H4 high patients, with median PFS of 16 weeks, suggesting promising efficacy beyond standard chemotherapy.
Mersana is progressing with dose expansion in TNBC, enrolling over 45 patients across two dosing regimens, with initial data expected in the second half of 2025.
The company believes the post-topo-1 TNBC opportunity is sizable, with Trodelvy expected to generate about $1 billion in 2025, and Emi-Le could serve as a subsequent line of therapy, especially as treatment landscapes evolve.
Strategic Acquisition of IllumiCare Enhances AI Capabilities
Premier announced the acquisition of IllumiCare in June, a move that significantly boosts its real-time insights and AI capabilities at the point of care.
IllumiCare is expected to generate $8-10 million in revenue in fiscal year 2026 with breakeven on EBITDA, adding a new growth engine.
The acquisition complements Premier's existing clinical decision support offerings and expands its addressable market amid mounting pressure on providers to improve clinical and financial performance.
Management highlighted the $100 savings per inpatient discharge as a key value proposition of IllumiCare, emphasizing its potential to deliver measurable impact.
Dafinchi