Ken Keller, the Global Head of Oncology Business and the President and CEO of Daiichi Sankyo, Inc., expressed that achieving the fifth indication in the US is a notable achievement. Eligible patients who have previously undergone treatment for metastatic HER2-positive solid tumors can now benefit from Enhertu. The FDA’s accelerated approval for this indication, irrespective of tumor type, reflects the significant effectiveness demonstrated by Enhertu across a range of metastatic cancers.
In the Phase II DESTINY-PanTumor02 trial, patients diagnosed with HER2-positive (IHC 3+) solid tumors, whether assessed centrally or locally, spanning biliary tract, bladder, cervical, endometrial, ovarian, pancreatic, or other tumor types, and treated with Enhertu, exhibited a confirmed Overall Response Rate (ORR) of 51.4% (with a 95% confidence interval [CI] of 41.7-61.0) and a median Duration of Response (DoR) ranging from 19.4 months (with a range of 1.3-27.9+ [the plus sign denotes ongoing responses at the data cutoff]).
In the DESTINY-Lung01 trial, among patients with centrally confirmed HER2-positive (IHC 3+) non-small cell lung cancer (NSCLC) who received Enhertu, a confirmed ORR of 52.9% (95% CI 27.8-77.0) and a median DoR range of 6.9 months (with a range of 4.0-11.7+) were observed. Furthermore, in the DESTINY-CRC02 trial, patients diagnosed with centrally confirmed HER2-positive (IHC 3+) colorectal cancer demonstrated a confirmed ORR of 46.9% (95% CI 34.3-59.8) and a median DoR range of 5.5 months (with a range of 1.3+-9.7+).
This approval was authorized through the FDA’s Real-Time Oncology Review initiative after obtaining Priority Review and Breakthrough Therapy Designation for Enhertu in this particular context within the United States. The submission to US regulators was assessed as part of Project Orbis, a system that allows for simultaneous submission and evaluation of oncology treatments among collaborating global partners. In line with Project Orbis, Enhertu is also undergoing regulatory assessment for the identical indication by regulatory bodies in Australia, Brazil, and Singapore.
FDA Approves Basilea’s ZEVTERA Antibiotic for Three New Indications
Basilea Pharmaceutica Ltd, based in Allschwil, has declared that the FDA has approved ZEVTERA® (ceftobiprole medocaril sodium for injection) for use in treating grown-up patients with Staphylococcus aureus bloodstream infections (bacteremia) (SAB), encompassing those with infective endocarditis on the right side, and grown-up patients with acute bacterial skin and skin structure infections (ABSSSI), as well as for both grown-up and pediatric patients (aged 3 months to under 18 years old) with community-acquired bacterial pneumonia (CABP).
David Veitch, the CEO of Basilea, expressed enthusiasm about the approval of ZEVTERA in the US. He sees the FDA’s positive decision as a significant step towards making ZEVTERA available to patients in the US. With 10 years of market exclusivity granted from the approval date, Veitch believes that the US market presents the most crucial global commercial opportunity for the brand.
The NDA was backed by clinical effectiveness and safety information obtained from phase III trials such as ERADICATE (SAB)1 and TARGET (ABSSSI),2 alongside a phase III study on CABP.3 ERADICATE stands out as the most extensive double-blind randomized registration study ever carried out for a new antibiotic treatment in SAB.
Basilea’s phase 3 program for ceftobiprole receives financial support from federal funds provided by the US Department of Health and Human Services (HHS), specifically the Administration for Strategic Preparedness and Response (ASPR) and the Biomedical Advanced Research and Development Authority (BARDA), under contract number HHSO100201600002C. This collaboration has granted Basilea around USD 112 million, covering about 75 percent of the expenses associated with the phase 3 studies for SAB and ABSSSI, regulatory tasks, and non-clinical efforts.
Genmab Expands Oncology Reach Through Acquisition of ProfoundBio
Genmab A/S and ProfoundBio, Inc. have revealed a firm arrangement where Genmab will purchase ProfoundBio through a complete cash deal. ProfoundBio, a privately held biotech firm, specializes in advanced ADCs and related technologies aimed at treating specific cancers such as ovarian cancer and other solid tumors expressing FRα. The acquisition, valued at USD 1.8 billion in cash, will be settled at the time of closure, factoring in ProfoundBio’s net debt and transaction costs.
The acquisition will expand Genmab’s mid to late-stage clinical pipeline and enhance its suite of proprietary technology platforms. This deal grants Genmab global rights to ProfoundBio’s portfolio of next-generation ADCs, comprising three clinical and multiple preclinical programs. Among these is Rina-S, a promising Topo1 ADC targeting FRα, currently undergoing Phase II trials for ovarian cancer and other solid tumors expressing FRα. Moreover, the integration of ProfoundBio’s innovative ADC technologies with Genmab’s proprietary antibody platforms may open avenues for creating and advancing new medicines, potentially revolutionizing cancer treatment and benefiting patients’ lives.
The inclusion of Rina-S in Genmab’s collection will allow the company to expand its presence in the field of gynecologic oncology and establish a strong foothold in treating solid tumors. Positioned as a potentially superior ADC, Rina-S aims to cater to a wider range of patients compared to earlier FRα-targeted ADCs. Drawing from data from the ongoing Phase I/II clinical trial, Genmab plans to broaden Rina-S’s development scope to encompass ovarian cancer and other solid tumors expressing FRα. In January 2024, the FDA granted Fast Track designation to Rina-S for treating patients with high-grade serous or endometrioid platinum-resistant ovarian cancer that express FRα.
European Medicines Agency Approves Rocket Pharmaceuticals’ RP-L102 Marketing Application for Fanconi Anemia
Rocket Pharmaceuticals, Inc. declared that the European Medicines Agency (EMA) has approved the Marketing Authorization Application (MAA) for RP-L102, its investigational gene therapy for Fanconi Anemia of complementation group A. Fanconi Anemia is a rare genetic condition caused by mutations in the FANCA gene, leading to issues with DNA repair. It is marked by bone marrow failure (BMF), cancer susceptibility, and congenital malformations. RP-L102 utilizes a lentiviral (LV) vector for treatment.
“The approval of the MAA for RP-L102 represents a significant advancement in our mission to offer this promising gene therapy to individuals affected by this severe childhood condition. At present, there are no existing treatments that may potentially prevent BMF for FA patients,” stated Kinnari Patel, Pharm.D., MBA, President, Head of R&D and Chief Operating Officer at Rocket Pharma. “We are grateful to the patients, their families, and the researchers whose contributions led to this important milestone and who continue to be part of the clinical development program. We eagerly anticipate collaborating closely with the EMA throughout the evaluation process to ensure RP-L102 becomes accessible to FA patients in need of new therapeutic options.”
The approval of MAA was granted due to the favorable findings from the earlier revealed data of the worldwide RP-L102 Phase I/II clinical study. RP-L102 exhibited continual genetic improvement, thorough correction of symptoms, and stability in blood-related issues. The safety record was notably positive, showing no major concerns, and the treatment, given without any harmful pre-treatment, was well received. There were no indications of abnormal bone marrow development, dominant clones, or genetic changes associated with RP-L102.
Alexion’s AAN 2024 Data Highlights Ultomiris and Soliris’ Promise for Rare Neurological Disorders
Alexion, the Rare Disease division of AstraZeneca, will unveil fresh clinical and real-world findings from its top-tier rare neurology collection at the American Academy of Neurology (AAN) Annual Meeting in Denver, CO, from April 13 to 18, 2024. The company plans to share 14 abstracts, including five oral presentations, covering both generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). These presentations will include novel long-term outcomes from the pivotal Phase III CHAMPION-MG and CHAMPION-NMOSD trials, along with real-world data, further bolstering the extensive evidence supporting the safety and effectiveness of Ultomiris (ravulizumab) and Soliris (eculizumab) in treating gMG and NMOSD.
Christophe Hotermans, Senior Vice President and Global Medical Affairs Head at Alexion, expressed that Ultomiris and Soliris represent a beacon of innovation and optimism for those affected by gMG and NMOSD. These medications provide treatment choices that could revolutionize the care of these challenging illnesses. The data presented at AAN will highlight the ongoing advantages of Ultomiris and Soliris in both clinical trials and real-world scenarios for these patient groups. The dedication to advancing care and pioneering solutions for individuals with these rare neurological disorders remains steadfast.
The ongoing worldwide CHAMPION-NMOSD trial, which is open-label, is expected to reveal the potential of Ultomiris in preventing relapses among individuals diagnosed with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) NMOSD over the long term. Data will indicate that there were no officially confirmed relapses during the study period among patients with AQP4 Ab+ NMOSD who received Ultomiris, with a median treatment duration of 138 weeks. Additionally, the final assessment from the global open-label CHAMPION-MG extension study will highlight the advantages of continuous Ultomiris treatment for patients with anti-acetylcholine receptor (AChR) Ab+ gMG. Over a period of up to 164 weeks, patients treated with Ultomiris maintained enhancements in functional activities and quality of life, as demonstrated by sustained Myasthenia Gravis-Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) total scores.
