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.
Strategic Focus on Phase III Readouts and Clinical Progress
The company is actively progressing towards multiple pivotal Phase III trials, including in myelofibrosis and endometrial cancer, with top-line data expected in 2026.
There is a strong emphasis on the potential of selinexor in combination with ruxolitinib to redefine the standard of care for myelofibrosis, with an estimated peak revenue potential of up to $1 billion annually in the U.S.
Management highlighted the significance of recent enrollment milestones, such as closing new patient screening for the SENTRY trial in myelofibrosis, which is a key step in their clinical development strategy.
The company is leveraging its clinical data to support regulatory and commercial ambitions, emphasizing the potential for selinexor to address unmet needs in diseases with limited treatment options.
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.
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.
Cash, cash equivalents, and restricted cash totaled $253.4 million as of June 30, 2025, with a cash runway extended into 2028.
General and administrative expenses decreased to $10.3 million in Q2 2025 from $12.3 million in Q2 2024, due to reduced share-based compensation and headcount.
Net loss for Q2 2025 was approximately $9.2 million or $0.34 per diluted share, improved from a net loss of $17.2 million or $0.64 per diluted share in Q2 2024.
Research and development expenses decreased to $29.6 million in Q2 2025 from $58.7 million in Q2 2024, reflecting lower manufacturing and clinical costs for COVID, flu, and OTC programs, partially offset by higher clinical costs for cystic fibrosis.
Revenue for Q2 2025 was $28 million, down $22 million year-over-year, primarily due to lower revenues from the CSL collaboration and amortization of upfront payments as CoStave progresses toward commercialization.
Total operating expenses for Q2 2025 were $40 million, down from $71 million in Q2 2024, driven by reduced manufacturing costs, clinical trial expenses, payroll, and employee benefits.
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.
Expenses aligned with expectations as the company fully transitioned into commercialization mode.
Liquidia closed Q2 2025 with $173 million in cash and cash equivalents, supporting ongoing commercialization and pipeline investments.
Q2 revenue totaled $8.8 million, including $6.5 million from YUTREPIA product sales and $2.3 million from treprostinil injection promotion services with Sandoz.
R&D expenses are expected to increase in the second half of 2025 due to ongoing label studies and initiation of the pivotal L-606 study.
SG&A expenses, excluding noncash and variable treprostinil costs, are expected to remain flat in upcoming quarters.
Strategic Acquisition of Solaris Health Accelerates Urology Expansion
Cardinal Health announced the acquisition of Solaris Health, the leading urology managed services organization, to expand its urology alliance physician network.
The Solaris Health acquisition adds over 750 providers across 14 states, significantly bolstering Cardinal's urology MSO platform.
This move positions Cardinal as a multi-specialty leader, with a focus on autoimmune, urology, and oncology therapeutic areas.
The acquisition complements other recent urology-focused acquisitions, creating a diversified revenue stream and strengthening market presence.
Management highlighted the strategic fit of Solaris within their broader urology and specialty care growth initiatives.
The deal is expected to be slightly accretive to EPS within the first 12 months post-close, with funding through a mix of cash and debt.
Dafinchi