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.
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.
Revuforj's Rapid Adoption and Early Line Usage Trends
Since launch, over 500 patients have been treated with Revuforj, with approximately 90% of usage in KMT2A patients.
In just 7 months, Revuforj has reached 1/4 of the 2,000 diagnosed relapsed/refractory KMT2A AML patients annually.
Physicians are increasingly using Revuforj in earlier lines of therapy, with emerging claims data showing 70% of use in second and third-line settings.
The company estimates that about 1/3 of KMT2A patients treated with Revuforj have proceeded to transplant, with this percentage expected to grow.
Early indicators suggest a higher transplant rate in the commercial setting compared to pivotal trials, driven by earlier treatment.
Physicians plan to restart Revuforj post-transplant for 1 to 2 years, potentially extending treatment duration and improving outcomes.
Progress and Strategic Opportunities for SGR-1505 MALT1 Inhibitor
Initial Phase I data for SGR-1505 showed a well-tolerated profile with responses in heavily pretreated CLL and Waldenstrom's macroglobulinemia patients, with 3 of 17 CLL patients responding and all 5 Waldenstrom's patients responding.
The program has received FDA Fast Track designation, highlighting its potential as a promising therapeutic in refractory hematologic cancers.
Schrodinger is exploring strategic opportunities to accelerate clinical development and maximize the potential of SGR-1505, rather than pursuing independent late-stage trials.
The company plans to provide further updates on dose escalation, translational data, and regulatory feedback later this year.
The emerging profile of SGR-1505 suggests best-in-class potential, with early evidence supporting its use in refractory disease, especially in patients previously exposed to BTK and BCL2 inhibitors.
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.
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.
Progress and Pipeline Advancements with Notable Regulatory Approvals
AbbVie received FDA approval for EMRELIS in non-squamous non-small cell lung cancer and Rinvoq for GCA, marking significant milestones in oncology and rheumatology.
The company announced positive Phase III results for Rinvoq in alopecia areata, demonstrating superior efficacy with 54% of patients reaching 80% scalp coverage at 24 weeks.
Regulatory submission of TrenibotE, a first-in-class short-acting toxin for aesthetics, is underway, with potential to expand AbbVie's aesthetic portfolio.
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