Nxera Pharma Reports Positive Phase 3 Trial Results for Daridorexant in South Korea
Nxera Pharma announced top-line results from its pivotal Phase 3 clinical trial of daridorexant in South Korea, showing that the study successfully met both primary and secondary endpoints in adult and elderly patients with insomnia. Daridorexant is a dual orexin receptor antagonist, a class of drugs that dampens wake signals in the brain to improve sleep. The 50 mg once-daily dose was tested in a multicenter, randomized, double-blind, placebo-controlled study. In this trial, participants receiving daridorexant experienced statistically significant improvements in subjective total sleep time, reduction in subjective latency to sleep onset, and decreases in subjective wake after sleep onset compared with placebo, with strong p-values (p < 0.0001) across all key measures.
The safety profile of daridorexant was favorable: treatment-emergent adverse events were comparable between the daridorexant and placebo groups, indicating no unexpected safety concerns during the 28-day treatment period. Adverse event rates were similar between groups (about 13.4% in the daridorexant arm vs 14.8% in the placebo arm), suggesting that daridorexant was well-tolerated and aligned with previous global clinical experience.
Insomnia is a pervasive condition in South Korea, affecting 15 % to 25 % of adults, or roughly 6.5–11 million people, with significant impacts on quality of life, mood, daytime functioning, and overall health. The trial success marks a key regulatory milestone for Nxera Pharma, which plans to submit a marketing authorization application in South Korea in the first quarter of 2026, targeting approval and market entry by the first quarter of 2027.
Daridorexant is already marketed as QUVIVIQ in other regions: in Japan under a commercialization agreement with Shionogi, and in the United States, Canada, and parts of Europe under a licensing agreement with Idorsia. It is also marketed in China and Hong Kong by Simcere. This Phase 3 success in a new geography underscores the drug’s consistent clinical performance and supports Nxera’s strategy to expand its global footprint in sleep disorder therapeutics. The results provide South Korean regulators with compelling evidence on daridorexant’s efficacy and safety in local patients and bring the therapy closer to approval for insomnia treatment in that market.
AstraZeneca to Acquire Remaining China Rights to AbelZeta’s GPC3 Armored CAR-T Therapy
AbelZeta Pharma, Inc. announced that AstraZeneca has agreed to acquire AbelZeta’s remaining 50% share of the China development and commercialization rights to C-CAR031, an armored CAR-T cell therapy targeting glypican-3 (GPC3) for the treatment of hepatocellular carcinoma (HCC) and other solid tumors. Under the terms of the deal, AstraZeneca will gain sole global rights to develop, manufacture, and commercialize C-CAR031, consolidating control of the program worldwide. AbelZeta is set to receive up to $630 million, including upfront payment plus development, regulatory, and sales-based milestone payments tied to the China portion of the GPC3 program.
This transaction builds on a prior agreement in which AstraZeneca already owned C-CAR031 rights outside China, thereby simplifying strategic ownership and enabling AstraZeneca to lead the therapy’s global development and commercialization without regional fragmentation. AbelZeta remains entitled to additional milestone payments and potential royalties related to development and commercialization.
C-CAR031 is an autologous chimeric antigen receptor T-cell therapy designed to target GPC3, a cell surface protein that is highly expressed in many cases of HCC, the most common form of liver cancer. The construct includes AstraZeneca’s dominant negative transforming growth factor-beta receptor II armoring platform, an engineering strategy intended to help CAR-T cells resist immunosuppressive signals in the tumor microenvironment, a key challenge in solid tumor immunotherapy.
This armoring approach theoretically enhances CAR-T cell persistence and function in hostile tumor environments, potentially improving therapeutic efficacy in cancers that have historically been difficult to treat with cell therapies alone. HCC itself remains a major global health challenge; it is among the most prevalent and lethal cancers worldwide, often diagnosed at advanced stages where prognosis is poor. According to industry data, HCC accounts for a high number of new cases annually, with cases in China alone remaining significant, underscoring the urgency and strategic importance of developing effective new treatments. By taking full ownership of the C-CAR031 program, AstraZeneca reinforces its commitment to advancing innovative cell therapies in oncology while leveraging AbelZeta’s platform research and early clinical development efforts.
AbbVie Announces Topline Phase 3 Results for Epcoritamab in Relapsed/Refractory DLBCL
AbbVie, in collaboration with Genmab, released topline data from the Phase 3 EPCORE DLBCL-1 trial evaluating epcoritamab (DuoBody CD3×CD20) in adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who were ineligible for high-dose chemotherapy and autologous stem cell transplant. Epcoritamab is a bispecific antibody engineered to bind CD3 on T cells and CD20 on B cells, redirecting T cells to attack malignant B cells.
In a global, randomized, multicenter study enrolling 483 patients, epcoritamab demonstrated a significant improvement in progression-free survival (PFS) versus the investigator’s choice of chemoimmunotherapy, establishing the first Phase 3 evidence of a PFS benefit for a CD3×CD20 bispecific antibody in this setting. Secondary outcomes also showed improvements in complete response rates (CRR), duration of response (DoR), and time to next treatment.
Despite these positive signals, the trial did not achieve statistical significance in overall survival (OS). The hazard ratio for OS crossed the threshold for statistical significance, indicating that patients treated with epcoritamab tended to live longer than those on standard therapies, but the difference could not be conclusively attributed to the treatment at this stage. The safety results in the study were consistent with the known profile of epcoritamab, characterized by manageable adverse events typical of T-cell-engaging therapies.
These include risks such as cytokine release syndrome (CRS) and neurologic events, which are well-recognized in subcutaneous bispecific antibody treatment paradigms. The company plans to present detailed data at a future medical scientific conference and engage regulators to discuss next steps for potential registrations in this indication.
DLBCL is the most common type of non-Hodgkin lymphoma worldwide and is known for aggressive growth and poor outcomes after relapse or refractory disease. Epcoritamab has already secured regulatory approvals in multiple lymphoma indications in over 65 countries and continues to be studied in various combination regimens and earlier lines of therapy. While the absence of an OS benefit in this trial may adjust expectations for certain labels, the improvement in PFS and response measures suggests that epcoritamab retains clinical value, supporting its continued development and potential use in specific patient subsets of relapsed/refractory DLBCL.
ImmunityBio Announces Durable 15-Month Complete Response with CD19 CAR-NK Therapy in Waldenström Lymphoma
ImmunityBio announced updated results from its ongoing Phase 1/2 QUILT-106 trial testing an off-the-shelf allogeneic CD19 CAR-NK cell therapy in combination with rituximab for patients with Waldenström’s non-Hodgkin lymphoma (NHL) who have exhausted standard treatments. This regimen represents a chemotherapy-free, lymphodepletion-free immunotherapy that, unlike traditional CAR-T therapies, does not require cytotoxic conditioning to enable cell engraftment, simplifying administration and reducing treatment-associated toxicity. Four patients have now completed treatment in the QUILT-106 study, all in the outpatient setting, and early data show that the combination therapy achieved 100% disease control, with complete responses in all treated patients.
The most remarkable outcome is the duration of remission in evaluable participants: two patients have now sustained complete responses for up to 15 months and are continuing without additional therapy after receiving a total of 8 doses of the CAR-NK plus rituximab regimen. One patient had particularly extensive disease with nearly complete bone marrow infiltration (~95 % tumor presence), yet achieved complete morphological remission after four doses and remains in ongoing remission at 15 months.
The rapid onset of responses, often evident after just two treatment cycles, and the persistence of these complete responses long after therapy cessation underscore the potential for durable, immune-mediated tumor control without the need for prolonged treatment or hospitalization. Notably, the safety profile in the updated follow-up appears favorable, with no serious adverse events reported to date in this small cohort, supporting the feasibility of delivering potent cell therapy safely outside hospital settings.
The CAR-NK cell therapy combines engineered natural killer cells expressing a CD19-targeted CAR with rituximab, which targets CD20 on lymphoma cells. This dual targeting leverages both direct CAR-mediated cytotoxicity and rituximab-enhanced antibody-dependent cellular cytotoxicity (ADCC). The results represent an important proof-of-concept for next-generation off-the-shelf cell therapies in indolent lymphomas like Waldenström’s, where treatment options are limited and durable remissions are difficult to achieve with standard therapies. The company plans continued enrollment and longer follow-up to further characterize efficacy, safety, and durability of responses, with future studies designed to build on these promising early signals and explore further combinations.
Sobi Secures European Commission Approval for ASPAVELI® for C3G and Primary IC-MPGN
Swedish Orphan Biovitrum (Sobi) announced that the European Commission (EC) has approved ASPAVELI (pegcetacoplan) for the treatment of complement C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) in adult and adolescent patients aged ≥12 years. These two rare and serious kidney diseases are characterized by uncontrolled complement activation leading to progressive kidney inflammation, proteinuria, declining kidney function, and often progression to end-stage kidney failure, requiring dialysis or transplantation. Importantly, ASPAVELI is now the first approved therapy for both C3G and primary IC-MPGN in Europe, and the first specifically indicated for patients aged 12–17 years in these conditions.
The EC decision follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) and is based on robust clinical evidence from the Phase 3 VALIANT study, which demonstrated that pegcetacoplan provides significant clinical benefits across multiple key renal outcomes. In VALIANT, patients treated with pegcetacoplan showed reductions in proteinuria, stabilization of estimated glomerular filtration rate (eGFR), and clearance of pathological C3 deposits from the kidney, markers closely linked to disease activity and progression. These outcomes suggest that pegcetacoplan not only slows disease progression but also addresses the underlying complement dysregulation driving kidney damage.
C3G and primary IC-MPGN affect approximately 8,000 individuals in Europe, representing a population with a high unmet need, as more than half of patients historically progress to kidney failure within 5–10 years of diagnosis. The EC approval includes the use of ASPAVELI in combination with a renin-angiotensin system (RAS) inhibitor unless RAS treatment is contraindicated or not tolerated. The therapy works by inhibiting C3 and C3b, regulating excessive complement activation that contributes to renal injury.
The approval aligns with published VALIANT results showing clinical improvements and bolsters the therapeutic landscape for rare glomerular diseases, offering a targeted option where none previously existed. Sobi and its partner, Apellis Pharmaceuticals, hold global co-development rights to systemic pegcetacoplan, with Sobi responsible for ex-U.S. commercialization and Apellis holding U.S. rights. This milestone not only broadens patient access to ASPAVELI in Europe but also highlights the growing importance of complement inhibition as a strategy for treating rare kidney diseases.
