- EFS: A 32% reduction in the risk of recurrence, progression, or death compared to chemotherapy alone (EFS hazard ratio of 0.68; 95% CI 0.53-0.88; p=0.003902).
- Pathologic Complete Response (pCR): 17.2% for the Imfinzi plus neoadjuvant chemotherapy group versus 4.3% for the chemotherapy alone group (difference in pCR 13.0%; 95% CI 8.7-17.6).
IMFINZI was generally well tolerated with no new safety signals observed. The addition of Imfinzi did not compromise patients’ ability to complete surgery.
John V. Heymach, MD, PhD, highlighted the potential of durvalumab to address recurrence in resectable lung cancer. Susan Galbraith emphasized the significant benefit of the Imfinzi-based regimen and AstraZeneca’s commitment to working with the FDA.
IMFINZI is approved in Switzerland and the UK for resectable NSCLC (Stage II and III without known EGFR mutations or ALK rearrangements) based on AEGEAN results. Regulatory applications are under review in the EU, China, and several other countries. Imfinzi is also the global standard of care for unresectable Stage III NSCLC in patients whose disease has not progressed after chemoradiotherapy, based on the PACIFIC Phase III trial.
Ipsen and Day One Sign an Exclusive Agreement to License and Market Tovorafenib Outside the US for the Treatment of the Most Frequently Occurring Childhood Brain Tumor
Ipsen has secured ex-U.S. regulatory and commercial rights to tovorafenib (OJEMDA) for pediatric low-grade glioma (pLGG) and any future indications. Tovorafenib is the first FDA-approved treatment for relapsed or refractory pLGG with BRAF fusion or rearrangement, or V600 mutation, following the pivotal Phase II FIREFLY-1 trial. Day One Biopharmaceuticals will receive approximately $111 million upfront and up to $350 million in milestone payments, along with double-digit tiered royalties.
The ongoing Phase III FIREFLY-2 trial is evaluating tovorafenib as a monotherapy for newly diagnosed children and young adults with RAF-altered low-grade glioma requiring first-line systemic therapy.
This partnership grants Ipsen global rights outside the U.S. while Day One retains global development and U.S. commercial rights. Tovorafenib received Orphan Drug Designation and FDA approval in April 2024 for patients six months and older with relapsed or refractory pLGG with BRAF alterations, which account for over half of pLGG cases worldwide.
David Loew, CEO of Ipsen, highlighted the potential impact of tovorafenib on children with cancer and the importance of this biomarker-driven strategy. Jeremy Bender, CEO of Day One, emphasized the shared commitment to bring novel therapeutics to patients with limited treatment options and Ipsen’s expertise in oncology.
Alzheimer’s Drug LEQUEMBI Turned Down by EU Regulators
European drug regulators rejected the Alzheimer’s treatment LEQEMBI from Biogen and Eisai, presenting a significant obstacle for the companies as they try to increase the therapy’s adoption in the U.S. The European Commission, the EU’s executive body, has the final authority on LEQEMBI’s approval, but it almost always aligns with the drug regulator’s recommendations.
Eisai expressed its disappointment in a statement, saying it is “extremely disappointed” by the negative recommendation and plans to request a reexamination of the decision. The news led to a more than 6% drop in Biogen’s shares on Friday, while Eisai’s stock remained relatively unchanged.
LEQEMBI, which the FDA approved last year, has faced a slow rollout due to various challenges, including diagnostic test requirements and the need for regular brain scans. Despite these issues, the drug has also received regulatory approvals in other countries, such as Japan, South Korea, China, and Israel.
Considered a breakthrough for Alzheimer’s disease, LEQEMBI is a monoclonal antibody that slows disease progression in patients at early stages. This is significant for a condition that has historically been difficult to treat.
Pfizer’s Gene Therapy DURVEQTIX Gets Green Light from European Commission for Hemophilia B
Pfizer Inc. announced that the European Commission has granted conditional marketing authorization for DURVEQTIX® (fidanacogene elaparvovec), a gene therapy for treating severe and moderately severe hemophilia B in adult patients without a history of factor IX inhibitors and without detectable antibodies to variant AAV serotype Rh74. DURVEQTIX enables patients to produce factor IX themselves with a one-time dose, eliminating the need for multiple intravenous FIX infusions weekly or biweekly.
Alexandre de Germay, Chief International Commercial Officer and Executive Vice President at Pfizer stated, “DURVEQTIX offers the potential for long-term bleed protection with a one-time dose, significantly impacting hemophilia B care in the European Union.”
Hemophilia B is a rare genetic bleeding disorder characterized by a deficiency in FIX, leading to frequent and prolonged bleeding. Standard care involves prophylactic FIX replacement therapy, which requires regular intravenous infusions. Despite this, many patients remain at risk for spontaneous bleeding episodes, placing a strain on healthcare systems. According to DelveInsight, the US had the highest diagnosed prevalent cases of hemophilia B among the 7MM in 2023, with about 4,000 cases. This number is expected to rise during the forecast period. Hemophilia B was most prevalent in the 19-44 age group, accounting for approximately 35% of the total cases.
The conditional marketing authorization is based on the Phase III BENEGENE-2 study (NCT03861273), which demonstrated DURVEQTIX’s efficacy and safety in adult male participants with moderately severe to severe hemophilia B. The study met its primary efficacy endpoint, showing a statistically significant decrease in annualized bleeding rate (ABR) post-DURVEQTIX infusion compared to a prophylaxis regimen with FIX. The efficacy remained stable from year two to year four post-treatment. DURVEQTIX was generally well-tolerated with a safety profile consistent with earlier phase results.
This authorization is valid in all 27 EU member states, as well as Iceland, Liechtenstein, and Norway. The EC approval follows recent approvals by the FDA and Health Canada, where it is marketed as BEQVEZTM.
This milestone reinforces Pfizer’s commitment to improving the lives of people with hemophilia. Pfizer is also developing a gene therapy for hemophilia A (giroctocogene fitelparvovec) and investigating marstacimab, an anti-tissue factor pathway inhibitor for hemophilia A and B. Regulatory applications for marstacimab are currently under review by the FDA and European Medicines Agency.
CHMP Approves New Indications for SPEVIGO Following Positive Review
Boehringer Ingelheim announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of a new indication for SPEVIGO (spesolimab) injection. This recommendation is for the prevention of generalized pustular psoriasis flares in adults and adolescents from 12 years of age. Additionally, the CHMP recommended extending the approved indication for SPEVIGO infusion to treat GPP flares in the same age group as monotherapy. SPEVIGO is a humanized selective IgG1 antibody that binds to the interleukin-36 receptor (IL-36R), a key component in the immune system’s pathway implicated in GPP.
The regulatory decision is based on the positive results from the EFFISAYIL 2 trial, a 48-week clinical study that demonstrated SPEVIGO significantly reduced the risk of GPP flares by 84% compared to placebo. The trial, which included 123 patients, observed no flares after week 4 of subcutaneous SPEVIGO treatment in the high-dose group. Adverse event rates were similar between the spesolimab and placebo groups.
Dr. Peter van der Kerkhof, Professor and former Chairman of the Department of Dermatology at Radboud University Nijmegen Medical Centre in the Netherlands, highlighted the diagnostic challenges of GPP, a rare and highly variable disease. He emphasized that spesolimab’s recent approvals, combined with the CHMP recommendation, offer the potential for continuous treatment, addressing a significant unmet need.
Generalized pustular psoriasis is distinct from plaque psoriasis and is characterized by chronic, heterogeneous, inflammatory neutrophilic disease with painful skin manifestations and systemic symptoms such as fever, pain, and fatigue. The disease varies widely among individuals, with symptoms affecting both the skin and internal systems. Uncontrolled generalized pustular psoriasis can lead to life-threatening complications like multi-organ failure and sepsis, significantly impacting the quality of life and causing fear and anxiety over the disease’s unpredictable course.
Carinne Brouillon, Member of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim remarked on the significance of the CHMP’s positive opinion. Alongside approvals in China and the US, this opinion marks a strong shift in treatment, offering patients the potential for significant improvement in their condition and quality of life. The ability to prevent GPP flare-ups gives patients the possibility to regain control over their lives.
Frida Dunger, Executive Director of the International Federation of Psoriasis Associations (IFPA), underscored the broad impact of generalized pustular psoriasis, describing it as a relentless and unpredictable disease affecting all aspects of a person’s life. She stressed the need for quick diagnosis and appropriate treatment for all individuals with generalized pustular psoriasis and expressed optimism that collaborative efforts among stakeholders are moving in the right direction to address this need.
