1. Camizestrant combination therapy improved the progression-free survival in HR+/HER- breast cancer patients with ESR1 mutation
On 26 February 2025, AstraZeneca announced results from a planned interim analysis of the SERENA-6 Phase III trial. The trial evaluated switching to the camizestrant combination versus continuing standard-of-care treatment with an aromatase inhibitor (AI) (anastrozole or letrozole) in combination with a CDK4/6 inhibitor in the 1st-line treatment of patients with hormone receptor (HR)-positive, HER2-negative advanced breast cancer whose tumours have an emergent ESR1 mutation. It was shown that camizestrant combination improved the primary endpoint of progression-free survival (PFS). Camizestrant is an investigational, potent, next-generation oral selective estrogen receptor degrader and complete ER antagonist. The trial evaluated switching to the camizestrant combination versus continuing standard-of-care treatment with an aromatase inhibitor in combination with CDK 4/6 inhibitor. The key secondary endpoints of time-to-second disease progression and overall survival were immature.
Link: https://www.astrazeneca.com/media-centre/press-releases/2025/camizestrant-improved-pfs-in-1l-hr-breast-cancer.html
2. Tezepelumab reduced the severity of chronic rhinosinusitis
On 1 March 2025, AstraZeneca and Amgen released full results from the Phase III WAYPOINT trial which evaluated the effects of Tezspire (tezepelumab) on patients with chronic rhinosinusitis with nasal polyps (CRSwNP) compared to placebo. Tezepelumab is a monoclonal antibody inhibiting a key epithelial cytokine called thymic stromal lymphopoietin (TSLP) to prevent epithelial-driven inflammation. Treatment with Tezspire significantly reduced nasal polyp severity, as measured by the co-primary endpoints’ nasal polyp score of -2.065 and nasal congestion score of -1.028 compared to the placebo at Week 52. Tezspire also significantly reduced the need for subsequent nasal polyp surgery by 98% and systemic corticosteroid treatment by 88% compared to placebo. The most common adverse events for Tezspire in the WAYPOINT trial were COVID-19, nasopharyngitis and upper respiratory tract infection.
Link: https://www.astrazeneca.com/media-centre/press-releases/2025/positive-results-from-the-tezspire-phase-iii-waypoint-trial-highlight-rapid-and-sustained-effect-in-chronic-rhinosinusitis-with-nasal-polyps.html
3. Twice-yearly depemokimab met co-primary endpoints in controlling chronic rhinosinusitis
On 01 March 2025, GSK announced positive results from the positive ANCHOR-1 and ANCHOR-2 phase III clinical trials assessing the efficacy and safety of depemokimab versus placebo in adults with chronic rhinosinusitis with nasal polyps (CRSwNP). Depemokimab is a twice-yearly administration targeting IL-5 which is a key driver for type 2 inflammation. Both trials met their co-primary endpoints with significant improvements in nasal polyp size and nasal obstruction.
Link: https://www.gsk.com/en-gb/media/press-releases/depemokimab-delivers-clinically-meaningful-and-statistically-significant-improvements-for-patients-with-chronic-rhinosinusitis-with-nasal-polyps-crswnp/
4. Gadoquatrane achieved non-inferior MRI diagnostic efficacy at half the standard GBCA dose
On February 26, 2025, Bayer announced positive results from the Phase III QUANTI CNS study evaluating investigational MRI contrast agent gadoquatrane in adults requiring MRI diagnosis on diseases related to the central nervous system. Contrast-enhanced MRI is one of the most powerful diagnostic tools in contemporary clinical medicine. The clinical study met all primary and secondary study objectives. The diagnostic efficacy was non-inferior to the comparator macrocyclic Gadolinium-based contrast agents (GBCAs) dosed at 0.1 mmol Gd/kg body weight. However, gadoquatrane only requires half of the dose compared to the comparator.
Link: https://www.bayer.com/media/en-us/positive-results-from-phase-iii-study-for-bayers-investigational-contrast-agent-gadoquatrane/
5. Approval of 2 mL pre-filled pen TAKHZYRO® in the EU
On February 24, 2025, Takeda announced that the European Medicines Agency (EMA) had approved an additional 2 mL pre-filled pen option for TAKHZYRO® (lanadelumab) for subcutaneous administration in patients 12 years old and above with hereditary angioedema. Hereditary angioedema is a condition characterised by rapid swelling of deeper layers of the skin and mucous membranes due to lack of C1 complement and uncontrolled kallikrein. While lanadelumab is able to bind and decrease plasma kallikrein to prevent recurrent inflammation. The drug has already been approved for different dosage forms inclyding150 mg solution for injection in pre-filled syringe, 300 mg solution for injection in a pre-filled syringe, and 300 mg solution for injection in vial.
Link: https://www.takeda.com/newsroom/newsreleases/2025/ema-patients-hae/
6. DB-OTO gene therapy improved hearing in children with OTOF-related hearing loss in a Phase 1/2 trial
On February 24, 2025, Regeneron announced updated data for the investigational gene therapy DB-OTO from the Phase 1/2 CHORD trial in children with genetic hearing loss due to variants of the otoferlin (OTOF) gene. Among the 11 participants with at least one post-treatment assessment, 10 demonstrated a notable response, with improved hearing at various decibel hearing levels (dBHL). Additionally, among five participants with 24-week assessments, three experienced improvements in average hearing thresholds to “nearly normal” (n=1; ≤40 dBHL) or normal (n=2; ≤25 dBHL) hearing levels. The therapy has previously received Orphan Drug, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the U.S. FDA and Orphan Drug Designation from the EMA.
Link: https://investor.regeneron.com/news-releases/news-release-details/latest-db-oto-results-demonstrate-clinically-meaningful-hearing
7. Announcements from FDA
Company | Drugs/Therapy | Indication | FDA decision | Clinical Data |
Merck | KEYTRUDA® (pembrolizumab) with Standard of Care as neoadjuvant treatment | Patients with resectable locally advanced head and neck squamous cell carcinoma (LA-HNSCC) | Acceptance for priority review of a new supplemental Biologics License Application (sBLA) | Phase 3 KEYNOTE-689 trial - Improvement in event-free survival (EFS) and major pathological response compared to adjuvant radiotherapy alone. |
BMS | Opdivo® (nivolumab) plus Yervoy® (ipilimumab) | First-line treatment option for adult and pediatric patients (12 years and older) with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (mCRC) | Acceptance for the review of supplemental biologics license application (sBLA) | Phase 3 CheckMate -8HW study - Meeting dual primary endpoints of progression free survival (PFS) compared to investigator’s choice of chemotherapy in the first-line setting and compared to Opdivo monotherapy |
Teva Pharmaceuticals and Medincell | UZEDY extended-release injectable suspension | Maintenance treatment of adults living with bipolar I disorder (BP-I) | Acceptance for the review of supplemental biologics license application (sBLA) | the RISE Study the SHINE Study |
Regeneron | odronextamab | Patients with relapsed/refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy | Acceptance for review of the resubmission a supplemental Biologics License Application (sBLA) | Phase 1 and pivotal Phase 2 trials (ELM-1 and ELM-2) - overall response rate of 80% (n=103), with 74% (n=95) achieving a complete response |
Bayer and BlueRock Therapeutics | OpCT-001 | Patients with primary photoreceptor diseases | Fast Track designation | / |
8. Recent CHMP recommendation
Company | Drug | Target | Type | Indication |
Roche | Columvi® (glofitamab) in combination with gemcitabine and oxaliplatin (GemOx) | CD20xCD3 Bispecific antibody | Combination | Adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) not otherwise specified who are ineligible for autologous stem cell transplant (ASCT). |
AbbVie | Upadacitinib | JAK inhibitor | Monotherapy | Adult patients with giant cell arteritis |
Novartis | Fabhalta® (iptacopan) | Factor B inhibitor | Monotherapy | Adults with C3 glomerulopathy |
J&J | DARZALEX® (daratumumab) subcutaneous (SC) formulation in combination with bortezomib, lenalidomide, and dexamethasone (daratumumab-VRd) | CD38 | Combination | Adult patients with newly diagnosed multiple myeloma |
AstraZeneca and Daiichi Sankyo | Trastuzumab deruxtecan (ADC) | HER2 | Monotherapy | Adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-low or HER2-ultralow breast cancer who have received at least one endocrine therapy in the metastatic setting and who are not considered suitable for endocrine therapy as the next line of treatment. |
Eli Lilly and Company | Jaypirca (pirtobrutinib) | Bruton's tyrosine kinase (BTK) inhibitor | Monotherapy | Adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) who have been previously treated with a BTK inhibitor. |
Regeneron | Linvoseltamab | BCMAxCD3 Bispecific antibody | Monotherapy | Adults with relapsed and refractory (R/R) multiple myeloma (MM). |
Eisai and Biogen | Lecanemab | Aβ | Monotherapy | Early Alzheimer’s Disease |
Link: https://www.roche.com/media/releases/med-cor-2025-02-28
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