BAXFENDY Receives US Approval for Adult Hypertension as a First-in-Class Aldosterone Synthase Inhibitor; Regeneron Pharmaceuticals Shares New Developments in Phase 3 Melanoma Study of Fianlimab Combination; ENHERTU Gains Two New US Indications in HER2-positive Early Breast Cancer; REGENXBIO Reveals Positive Pivotal Trial Results for Duchenne Gene Therapy RGX-202; BeOne Medicines Gains FDA Approval for BEQALZI™ in R/R Mantle Cell Lymphoma

BAXFENDY Becomes the First FDA-Approved Aldosterone Synthase Inhibitor for Adults With Hypertension

AstraZeneca’s BAXFENDY (baxdrostat) has received US approval as the first aldosterone synthase inhibitor (ASI) for treating hypertension in adults whose blood pressure remains uncontrolled despite the use of other antihypertensive therapies.

Hypertension affects nearly 1.4 billion individuals globally. In the US, about half of hypertension patients already taking multiple blood pressure medications continue to experience uncontrolled hypertension, a major contributor to cardiovascular disease and early mortality. Hypertension remains the most common and impactful modifiable cardiovascular risk factor worldwide, responsible for substantial rates of death and disability.

BAXFENDY is a highly selective, first-in-class ASI developed to reduce blood pressure through targeted suppression of aldosterone production. Aldosterone is a hormone linked to elevated blood pressure and increased risks of cardiovascular and kidney complications.

The approval from the US Food and Drug Administration (FDA) was supported by positive Phase III BaxHTN trial data, in which BAXFENDY achieved statistically significant and clinically meaningful reductions in seated systolic blood pressure at both 1 mg and 2 mg doses among patients with uncontrolled or resistant hypertension receiving two or more background therapies. The treatment was generally well tolerated, with no unexpected safety concerns reported.

Regeneron Pharmaceuticals Shares Phase 3 Update on Fianlimab Combination in Frontline Advanced Melanoma

Regeneron Pharmaceuticals reported Phase III findings indicating that a first-line treatment regimen featuring its LAG-3 inhibitor fianlimab demonstrated numerically longer progression-free survival (PFS) compared with Merck & Co.’s KEYTRUDA in certain melanoma patients, although the improvement was not statistically significant.

The trial enrolled 1,546 patients with unresectable, locally advanced, or metastatic melanoma, who were randomised to receive either a high 1600-mg dose of fianlimab plus Regeneron’s PD-1 inhibitor LIBTAYO every three weeks, a lower 400-mg dose of fianlimab combined with LIBTAYO, placebo with LIBTAYO monotherapy, or Keytruda with placebo.

Regeneron announced that median PFS reached 11.5 months in the high-dose arm and 9.6 months in the low-dose arm, compared with 6.3 months for Keytruda monotherapy (p=0.0627 and p=0.4661, respectively). The high-dose regimen produced a hazard ratio (HR) of 0.845 versus Keytruda, while the low-dose regimen achieved an HR of 0.931.

Earlier Phase I results for the high-dose combination had shown a median PFS of 24 months, along with an objective response rate of 57% and a complete response rate of 25%. In the Phase III study, Regeneron stated that no new safety concerns were identified with the fianlimab combination and added that detailed findings will be presented at an upcoming medical conference.

US FDA Broadens ENHERTU Approval in HER2-positive Early Breast Cancer

AstraZeneca and Daiichi Sankyo announced that ENHERTU has received approval from the US Food and Drug Administration (FDA) for both neoadjuvant and adjuvant use in patients with HER2-positive early breast cancer. The approvals are supported by findings from the Phase III DESTINY-Breast11 and DESTINY-Breast05 studies, respectively.

For neoadjuvant therapy, ENHERTU in combination with subsequent taxane, trastuzumab, and pertuzumab (THP) is now approved for adult patients with HER2-positive Stage II or III breast cancer. In the adjuvant setting, ENHERTU is indicated for adult patients with HER2-positive breast cancer who continue to have residual invasive disease after trastuzumab-based (with or without pertuzumab) and taxane-containing treatment.

In the DESTINY-Breast11 trial, neoadjuvant treatment with ENHERTU followed by THP achieved a pathologic complete response (pCR) rate of 67.3%, compared with 56.3% for dose-dense doxorubicin and cyclophosphamide followed by THP (ddAC-THP), reflecting an 11.2% improvement (95% CI: 3.9–18.3; p=0.003). At the time of the pCR assessment, event-free survival (EFS) events had occurred in 4.5% of patients, while overall survival (OS) events were reported in 1.9% of patients. These findings were published in Annals of Oncology.

In the DESTINY-Breast05 study, adjuvant treatment with ENHERTU reduced the risk of invasive disease recurrence or death by 53% versus trastuzumab emtansine (T-DM1) in patients with HER2-positive breast cancer who had residual invasive disease following neoadjuvant therapy (HR 0.47; 95% CI: 0.34–0.66; p<0.0001). At the three-year mark, 92.4% of patients receiving ENHERTU remained alive and free from invasive disease, compared with 83.7% in the T-DM1 group. A total of 51 events (6%) were observed in the ENHERTU arm versus 102 events (12%) in the T-DM1 arm. The study results were published in The New England Journal of Medicine.

Results from both DESTINY-Breast11 and DESTINY-Breast05 were presented during the 2025 European Society for Medical Oncology Congress 2025. Following the DESTINY-Breast05 findings, trastuzumab deruxtecan (ENHERTU) has also been incorporated into the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) as a Category 1 recommended adjuvant therapy for patients with HER2-positive early breast cancer who have residual disease and are considered at high risk of recurrence after preoperative treatment.

REGENXBIO Shares Positive Phase III AFFINITY DUCHENNE® Topline Results for RGX-202

REGENXBIO announced positive topline and interim functional findings from the pivotal Phase III segment of the Phase I/II/III AFFINITY DUCHENNE® study evaluating RGX-202, its investigational gene therapy candidate for Duchenne Muscular Dystrophy. The study successfully achieved its primary endpoint with strong statistical significance (p<0.0001), as 93% of treated participants attained at least 10% microdystrophin expression at Week 12 (n=30). Interim analyses also showed a statistically significant association between microdystrophin expression and functional improvement.

RGX-202 is engineered to target the root cause of Duchenne by driving expression of a novel microdystrophin designed to closely resemble naturally occurring dystrophin. Notably, it is the only microdystrophin construct incorporating the C-Terminal domain, which is believed to help preserve and protect muscle function. The therapy’s differentiated design includes a proprietary construct, a proactive immunosuppression strategy, and a suspension-based manufacturing platform aimed at achieving high product purity. Collectively, these features are intended to enhance muscle function, durability, and safety outcomes for patients.

The pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial assessed RGX-202 at a dose of 2×10^14 GC/kg in 31 ambulatory boys aged 1 year and older. Interim topline data included safety outcomes (n=31), biomarker results (n=30), and functional assessments from patients older than 4 years who were evaluated 12 months after treatment (n=9).

In addition, more than 20 patients have already been enrolled in the confirmatory study of RGX-202 (target n=30), and the company anticipates completing dosing across all 60 patients enrolled in both the pivotal and confirmatory studies by mid-year.

BeOne Medicines’ BEQALZI Secures U.S. FDA Approval for R/R Mantle Cell Lymphoma

BeOne Medicines Ltd. announced that the FDA has granted accelerated approval to BEQALZI, a next-generation BCL2 inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL) who have received at least two prior lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor. BEQALZI was developed to deliver enhanced BCL2 inhibition with improved potency, selectivity, and a pharmacologic profile that may offer better efficacy, tolerability, and convenience compared to other therapies in its class.

The accelerated approval is based on efficacy and safety findings from the Phase 1/2 BGB-11417-201 study (NCT05471843), which were presented at the 67th American Society of Hematology Annual Meeting & Exposition. Independent review of the trial data showed:

• Overall response rate (ORR): 52% (95% CI, 42–62)
• Complete response (CR) rate: 16% (95% CI, 9.1–24.0)
• Median time to response (TTR): 1.9 months
• Median duration of response (DOR): 15.8 months (95% CI, 7.4 months–not estimable) at a median response follow-up of 11.9 months, with data still maturing
• Safety profile: sonrotoclax monotherapy was generally well tolerated

Continued approval for this indication will depend on verification of clinical benefit in the ongoing Phase 3 CELESTIAL-RRMCL trial (NCT06742996). The FDA has also granted sonrotoclax Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation for this indication.