Seres has advanced preparation for the next stage of SER-155 development, including a Phase II protocol submission to the FDA.
The Phase Ib study showed a 77% relative risk reduction in bloodstream infections in allo-HSCT patients, supporting further development.
The upcoming Phase II study is designed to enroll approximately 248 patients with an adaptive design and interim analysis, with results expected within 12 months of initiation.
Positive interactions with the FDA and breakthrough therapy designation facilitate the study design and potential approval pathway.
The company aims to use interim data to inform next steps, including potential engagement with the FDA for a registrational study.
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.
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.
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.
Discontinuation of M6 Artificial Disc Product Lines and Its Impact
Orthofix announced the discontinuation of the M6 artificial cervical and lumbar disc product lines in February 2025.
Pro forma results exclude M6 impact, with a focus on margin improvements driven by this discontinuation.
The discontinuation has contributed to approximately 50% of the margin expansion in Q2 2025, indicating a significant shift in product portfolio and profitability strategy.
Progress in Hepatitis Delta ECLIPSE Registrational Program
All three ECLIPSE studies (1, 2, and 3) are now actively recruiting patients globally, with enrollment progressing well.
ECLIPSE 1's primary completion is expected by December 2026, with ongoing enrollment and no specific delays reported.
ECLIPSE 2 and 3 have recently enrolled their first patients; ECLIPSE 2 addresses patients unresponsive to bulevirtide, with a 24-week primary endpoint.
The program aims to demonstrate superior virologic response (target not detected) compared to existing treatments, with a goal of over 60% response rate in the combination therapy.
Transition to Clinical-Stage Biotech with First Clinical Trials
AbCellera achieved a major milestone by receiving Health Canada authorization to initiate its first two clinical trials for ABCL635 and ABCL575.
Dosing has begun in the Phase I trial for ABCL635, targeting moderate to severe vasomotor symptoms, marking a shift from platform to clinical-stage biotech.
A second Phase I trial for ABCL575 was also initiated, with dosing expected to start shortly, expanding the company's clinical pipeline.
The company added a third program, ABCL688, into IND-enabling studies, indicating ongoing pipeline expansion and transition into more advanced clinical development.
Regulatory and External Factors Influencing Growth and Development
The company discussed the impact of ACIP recommendations on expanding the hepatitis B market and the importance of policy changes, such as Medicare coverage updates, in supporting growth.
Dynavax's development programs, including shingles, influenza, Lyme disease, and plague vaccines, are aligned with regulatory pathways, with key data readouts expected in the coming months and years.
The company emphasizes its reliance on external collaborations and government partnerships, particularly with the U.S. Department of Defense, for advancing its vaccine pipeline.
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