TAGRISSO Delivers Landmark Eight-Year Survival Outcomes in Early-Stage EGFR-Mutated Lung Cancer; Scholar Rock Receives FDA Approval for ISEMBYLD; Pharming’s Joenja Receives FDA Approval as First U.S. Treatment for Children With APDS; AbbVie Reports Positive Phase 3 Results for Atogepant in Menstrual Migraine; Rezera’s Ruvonoflast Delivers Positive Inflammation Results with Favorable Safety in RESOLVE-1

TAGRISSO Demonstrates Eight-Year Landmark Survival Benefit for Patients with Early-Stage EGFR-Mutated Lung Cancer

Updated exploratory findings from the Phase III ADAURA trial demonstrated that AstraZeneca’s Tagrisso (osimertinib) continued to provide a sustained and clinically meaningful overall survival (OS) benefit at eight years compared with placebo in the adjuvant treatment of patients with early-stage (IB, II, and IIIA) EGFRm NSCLC following complete tumour resection with curative intent.

The findings were presented during the Presidential Symposium at the IASLC 2026 World Conference on Lung Cancer (#WCLC26), organised by the International Association for the Study of Lung Cancer in Seoul, Republic of Korea (Abstract PL03.01). The results were also published simultaneously in the Journal of Thoracic Oncology.

After eight years of follow-up, Tagrisso maintained its OS advantage, lowering the risk of death by 47% versus placebo in the primary population of patients with Stage II-IIIA disease, based on a hazard ratio (HR) of 0.53 (95% confidence interval [CI], 0.38-0.75). Across the overall trial population comprising patients with Stage IB-IIIA disease, Tagrisso reduced the risk of death by 48% compared with placebo (HR 0.52; 95% CI, 0.39-0.71).

In the primary population, an estimated 74% of patients receiving Tagrisso remained alive at eight years, compared with 58% of those receiving placebo. In the overall trial population, estimated eight-year survival was 79% with Tagrisso versus 64% with placebo. Consistent with the planned final analysis, the OS benefit associated with Tagrisso over placebo was maintained across all predefined patient subgroups.

Supporting the clinical findings from ADAURA, real-world evidence presented at WCLC26 from a retrospective cohort study of US patients with early-stage (I-IIIA) EGFRm NSCLC showed that discontinuing Tagrisso before completing the recommended three-year treatment course more than doubled the risk of disease recurrence or death (Abstract P1.142). These findings highlight the importance of completing the full treatment duration established in ADAURA and emphasise the role of effective clinician-patient communication in supporting treatment persistence.

In the advanced disease setting, further findings from the Phase III FLAURA2 trial presented at WCLC26 reinforced the benefits of Tagrisso combined with platinum-pemetrexed chemotherapy as a first-line treatment for patients with advanced EGFRm NSCLC. A novel safety analysis (Abstract PT2.03.03) found that, following a median follow-up of 42.6 months, the safety and tolerability profile of Tagrisso plus platinum-pemetrexed remained consistent with the established safety profiles of the individual medicines. The analysis also showed a clear reduction in the emergence of new adverse events after the initial induction period.

Additionally, an exploratory analysis (Abstract P2.237) indicated that progression-free survival and OS hazard ratios numerically favoured Tagrisso in combination with platinum-pemetrexed over Tagrisso monotherapy, irrespective of patients’ baseline TP53 co-mutation status.

Scholar Rock’s ISEMBYLD Receives FDA Approval as a Muscle-Targeted Treatment for Spinal Muscular Atrophy

Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children aged two years and older who are receiving a survival motor neuron 2 (SMN2)-targeted therapy. SMA is a rare and serious neuromuscular disorder characterized by the irreversible loss of motor neurons and progressive muscle wasting. The disease leads to a continual decline in motor function over time and can significantly reduce independence in both children and adults.

ISEMBYLD is the first and only muscle-targeted therapy to demonstrate improved motor function in people with SMA who are receiving an SMN2-targeted treatment. In the Phase 3, randomized, placebo-controlled SAPPHIRE study, patients treated with ISEMBYLD achieved a robust and clinically meaningful improvement in motor function after one year, while those receiving an SMN2-targeted treatment alone experienced a decline in motor function.

“Today’s FDA approval of ISEMBYLD marks a defining moment for the SMA community as we now launch the world’s first-ever muscle-targeted treatment for children and adults living with SMA in the U.S.,” said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. “After decades of industry-wide efforts to unlock the potential of myostatin inhibition, Scholar Rock has delivered a therapeutic breakthrough with ISEMBYLD. Our U.S. commercial team is now working with physicians, SMA care teams, and payers to support the SMA community, while our Scholar Rock Supports™ team is prepared to provide comprehensive assistance to patients and caregivers. I would also like to extend my sincere appreciation to the clinical investigators, Cure SMA, and other patient advocacy organizations for their dedication and support throughout this journey. Most importantly, I am deeply grateful to the patients and families affected by SMA who participated in our clinical trials, placing their trust in Scholar Rock and demonstrating remarkable resilience throughout the process.”

Following the approval of ISEMBYLD, Scholar Rock was granted a Rare Pediatric Disease Priority Review Voucher, which can be used to obtain priority review for a future marketing application.

Pharming’s Joenja Becomes First FDA-Approved Treatment for Children With APDS

Pharming announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for 40 mg and 50 mg twice-daily doses of Joenja® (leniolisib). Joenja is an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor indicated for children aged 4 to 11 years who weigh at least 27 kg and have activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. The approval makes Joenja the first FDA-approved treatment for children aged 4 to 11 years with APDS in the U.S. The newly authorized doses are expected to become available to eligible pediatric patients in the U.S. in October through Pharming’s established specialty distribution network and patient-support infrastructure. On July 30, Pharming separately submitted an sNDA requesting approval of lower Joenja doses for pediatric patients with APDS aged 4 to 11 years who weigh between 13 kg and less than 27 kg.

APDS generally emerges during early childhood and is marked by substantial immune dysregulation and recurrent sinopulmonary infections. Over time, these infections may result in permanent lung damage and other severe complications. The impact of APDS on children and their families can extend beyond the physical manifestations of the disease. Frequent infections, medical visits, and ongoing treatment requirements can interfere with school attendance, education, and social development, further increasing the day-to-day burden of the condition.

“Today’s approval represents an important milestone in our efforts to broaden access to Joenja for more people living with APDS. We are grateful to the FDA, clinical trial participants, caregivers, investigators, healthcare professionals and the APDS community for their contributions to achieving this milestone,” said Leverne Marsh, Chief Commercial Officer of Pharming. “Our immediate focus is on helping physicians and families navigate the access process so that eligible patients can begin treatment as quickly and responsibly as possible.”

The FDA initially approved Joenja in March 2023 for the treatment of APDS in adult and pediatric patients aged 12 years and older. The latest approval is supported by findings from a multinational, open-label, single-arm Phase III study involving children aged 4 to 11 years with APDS. Over the 12-week treatment period, the study showed improvements in two clinically relevant characteristics of APDS, reduced lymphadenopathy and an increase in naïve B cells, indicating improvement in the underlying immune dysfunction. The safety profile remained consistent with previous clinical experience with Joenja, with all treatment-emergent adverse events reported as mild to moderate. No serious adverse events related to the drug were observed.

AbbVie’s Atogepant Shows Positive Phase 3 Results in Menstrual Migraine

AbbVie announced positive topline findings from the Phase 3 LUNA study evaluating atogepant, a calcitonin gene-related peptide (CGRP) receptor antagonist, for the preventive treatment of menstrual migraine in adults. The results further highlight AbbVie’s expertise in migraine research and its commitment to developing new treatment options for individuals affected by complex and disabling migraine attacks.

“These positive Phase 3 LUNA findings mark an important step forward for people living with menstrual migraine, as atogepant demonstrated superior efficacy compared with placebo across the primary endpoint and all eight ranked secondary endpoints,” said Primal Kaur, M.D., senior vice president, global development, immunology, neuroscience, eye care and specialty at AbbVie. “Menstrual migraine attacks can be severe, prolonged and highly disruptive to everyday life. These findings move us closer to our goal of providing a potential treatment option for women affected by this debilitating condition.”

In the study, participants received atogepant for seven consecutive days, beginning three days before the expected onset of menstruation, over three menstrual cycles. Atogepant achieved the primary endpoint by producing a greater reduction in migraine days during the perimenstrual period than placebo, averaged across the three menstrual cycles during the double-blind treatment period. Migraine days decreased by an average of 1.20 days with atogepant compared with 0.40 days with placebo, resulting in a reduction of 0.80 migraine days versus placebo (p<0.0001).

Atogepant also achieved statistically significant and clinically meaningful improvements compared with placebo across all eight ranked secondary endpoints (p<0.0001). These endpoints included assessments of headache burden, acute medication use, functional disability, cognitive function and treatment response. The safety findings were consistent with the established safety profile of atogepant, with no new safety signals identified.

Rezera’s Ruvonoflast Achieves Primary Inflammation Endpoint with Favorable Safety in Phase 2 RESOLVE-1 Trial

Rezera Inc. announced positive topline findings from RESOLVE-1, a Phase 2 randomized, double-blind, placebo-controlled trial assessing the efficacy and safety of ruvonoflast in participants both with and without type 2 diabetes.

The trial achieved its primary inflammation endpoint, demonstrating a change from baseline in high-sensitivity C-reactive protein (hsCRP) through week 24. Both the 150 mg once-daily and twice-daily ruvonoflast regimens evaluated in the study produced reductions in hsCRP compared with placebo, despite the trial not specifically enriching for participants with elevated baseline hsCRP levels. hsCRP reductions were observed among participants regardless of type 2 diabetes status, highlighting the potential applicability of ruvonoflast across the broader cardiometabolic disease spectrum.

The findings are consistent with previously reported evidence of ruvonoflast’s anti-inflammatory activity in individuals with elevated cardiovascular risk and a high inflammatory burden, where hsCRP levels declined by 82% after four weeks of treatment.¹ Taken together, the results demonstrate a clear dose-response relationship for ruvonoflast and establish a strong rationale for advancing into Phase 3 studies using doses of up to 450 mg/day.

“Results from RESOLVE-1 represent an important milestone in the clinical development of ruvonoflast, demonstrating dose-dependent clinical activity across a broad cardiometabolic population while also confirming a favourable safety profile that supports progression of this differentiated NLRP3 inhibitor into Phase 3,” said Dr. Jyothis George, MBBS, Ph.D., Chief Medical Officer at Rezera. “We are grateful to the participants and investigators who contributed to one of the largest and longest clinical studies of an NLRP3 inhibitor to date. These findings are aligned with our recently published peer-reviewed data in participants with elevated cardiovascular risk. We look forward to advancing ruvonoflast into a Phase 3 study in participants with PAD, an area characterized by significant unmet medical need.”

Beyond its effects on hsCRP, ruvonoflast treatment also produced consistent reductions in several thrombo-inflammatory biomarkers. The results indicate broad, dose-dependent engagement of the NLRP3 inflammatory pathway and support the potential of NLRP3 inhibition as an upstream approach to regulating chronic inflammation associated with atherosclerotic cardiovascular disease. Treatment had no effect on body weight.

Ruvonoflast demonstrated a generally favourable tolerability profile across all treatment groups. Safety analyses showed no evidence of ruvonoflast-related liver toxicity or increases in infections or serious infections. No serious adverse events were considered related to the study drug.

The RESOLVE-1 findings add to previously reported Rezera clinical data in participants with elevated cardiovascular risk and high inflammatory burden¹ as well as in individuals with neuroinflammation.² The Company’s plans to advance ruvonoflast into Phase 3 are supported by a clinical dataset involving more than 400 participants across five clinical trials of ruvonoflast. These studies included treatment durations of up to nine months and evaluated doses ranging from 150 mg/day to 450 mg/day.

Rezera has reached alignment with regulatory authorities regarding key components of the planned Phase 3 PAD study, including its endpoints, sample size, study duration, and safety considerations. The Company expects to initiate the Phase 3 PAD trial during the first half of 2027.