Daretabart

Renaissance Pharma Ltd, a wholly owned subsidiary of Essential Pharma and headquartered in the UK, announced that the US FDA has granted Fast Track Designation for Daretabart (hu1418K322A), a GD2-targeted monoclonal antibody, for the treatment of high-risk neuroblastoma (HRNB). The company simultaneously confirmed Investigational New Drug (IND) clearance, enabling US initiation of the SHINE Phase II/III trial in relapsed or refractory paediatric HRNB patients. Daretabart is developed under an exclusive licence from St. Jude Children’s Research Hospital. Fast Track Designation gives Renaissance Pharma more frequent interactions with the FDA throughout development and eligibility for rolling and accelerated review of any future marketing application. The designation does not itself confer approval or alter the evidentiary standard required. The clinical rationale for the designation draws on a Phase II study of the antibody — then designated hu14.18K322A — evaluating it as first-line and post-consolidation therapy in HRNB patients. Updated results, published in the Journal of Clinical Oncology in February 2022, reported a three-year event-free survival (EFS) rate of 73.7% and an overall survival (OS) rate of 86.0%. No data cutoff date or patient number was disclosed in the press release for this study. The SHINE trial, now cleared to enrol in the United States, targets a narrower population: children with relapsed or refractory HRNB. Daretabart binds GD2, a disialoganglioside cell-surface antigen expressed at high density on neuroblastoma cells. The antibody is designed to enhance immune-mediated tumour cell killing. Renaissance Pharma states that the molecule incorporates structural modifications intended to improve its tolerability profile relative to earlier-generation anti-GD2 antibodies, though no technical characterisation of those modifications — such as Fc engineering specifics or comparative binding data — was disclosed in the announcement. The company also confirmed that the first commercial-scale Good Manufacturing Practice (GMP) batch of Daretabart has been produced for use in the SHINE trial. Research context Neuroblastoma accounts for 7%-10% of all childhood cancers and is the most common extracranial solid tumour in children under one year of age. Approximately 800 new cases are diagnosed annually in the United States and more than 1,500 in Europe. Roughly 50% of all neuroblastoma patients present with high-risk disease, which carries a five-year overall survival rate of approximately 50% despite multimodal treatment — comprising induction chemotherapy, surgery, high-dose consolidation with autologous stem cell rescue, radiotherapy, and maintenance immunotherapy. The current standard of care in HRNB already includes anti-GD2 monoclonal antibody therapy. Dinutuximab (Unituxin, United Therapeutics) received FDA approval in 2015 for pediatric patients with HRNB who had achieved at least a partial response to prior first-line multiagent, multimodal therapy. Dinutuximab beta (Qarziba, EUSA Pharma) holds European approval. Both agents target GD2 and are associated with infusion-related pain, a consequence of GD2 expression on peripheral nerve fibres. Renaissance Pharma’s claim that Daretabart incorporates modifications to improve tolerability positions the antibody as a potential successor in the same mechanistic class, though head-to-head comparative data are not yet available. The relapsed or refractory HRNB setting targeted by the SHINE trial remains without a dedicated approved therapy, and response rates to salvage regimens in this population are low. The five-year survival for patients who relapse after standard therapy is estimated at below 10% in published literature, establishing the basis for the FDA’s recognition of unmet medical need in granting Fast Track status. Leave a Reply Cancel reply Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

Essential Pharma announces €900 million recapitalisation through a Gyrus Capital Continuation Vehicle led by AlpInvest and new strategic financing from Sixth Street Provides significant resources to build on diversified portfolio of established and rare disease medicines Egham, UK – 12 December 2024 – Essential Pharma (“Essential” or “the Company”), an international specialty pharma group focused on ensuring that patients have access to low volume, clinically differentiated, niche pharmaceutical products across key therapeutic areas, today announces the completion of a €900 million financing, through a Gyrus Capital single-asset Continuation Vehicle led by AlpInvest, a subsidiary of Carlyle Global Investment Solutions, and a new strategic financing facility with Sixth Street. Essential Pharma was acquired in 2019 by Gyrus Capital, who have supported the transformation of the Company into a global, high-growth specialty pharma group with significant development opportunities. The transaction will enable Essential to deploy additional capital to build on its diversified portfolio and late-stage pipeline across established and rare disease medicines. This will be achieved through its successful acquisition and development strategy under the leadership of CEO, Emma Johnson. This additional capital will support Essential’s mission to ensure access to essential medicines for patients across new and existing markets. Since her appointment in 2023, Emma has driven the Company’s expansion into rare diseases, with the addition of marketed product Colobreathe® and Hu14.18, an immunotherapy in late-stage clinical development for the treatment of high-risk neuroblastoma. Following the transaction, Lee Morley, appointed as Non-Executive Director to the Board in 2023, will assume the role of Chairman of Essential Pharma, succeeding Dr Patrick Vink. Lee has more than 30 years’ experience in the biopharmaceutical industry, with extensive knowledge of commercialisation, pricing, reimbursement and product launch strategy. He was a co-founder and CEO at EUSA Pharma before the company’s €750 million acquisition by Recordati (BIT: REC) in 2022. During his time at EUSA Pharma, he led the company’s transition from specialty pharma to oncology and rare disease, driving sales from €20 million to €150 million. Emma Johnson, CEO of Essential Pharma, commented: “This transaction provides us with substantial additional firepower, strengthening our ability to deliver essential medicines to patients who need them most. By moving into new geographical markets and building on our diversified portfolio and late-stage pipeline across our key therapeutic areas, we can expand our reach to help underserved patient populations across the globe. We are excited to continue our relationship with Gyrus Capital as we execute our strategy to become a leading, global specialty pharma group with a growing rare disease presence, where patient needs are often most acute. As we transition to our new ownership, I’d also like to thank Patrick for his significant contributions as Chairman and welcome Lee as he steps into the role.” Dr Robert Watson, Managing Partner at Gyrus Capital, said: “This is a landmark investment for Essential and for Gyrus Capital. We are delighted to partner with AlpInvest, Stepstone, Sixth Street and other significant private capital institutions, who believe in our shared vision of building an exceptional specialty pharma company, and bringing critical medical products to patients across the globe.” Sixth Street, a leading global investment firm with deep experience supporting biotech companies across all stages of growth, is providing a new strategic financing facility consisting of €300 million in structured equity and growth debt. Jeff Pootoolal, Partner at Sixth Street who will join the board of Essential Pharma, said: “Essential Pharma has assembled a leading team and platform in its pursuit of providing patients around the world with critical medicines. We are excited to support Emma and Essential’s leadership as they continue to execute and expand on their mission.” About Essential Pharma Essential Pharma is an international specialty pharmaceutical group dedicated to maintaining access to clinically differentiated, niche, branded medicines across multiple therapeutic areas. The group has been an important and valued partner to healthcare providers for over 20 years by giving underserved patient populations access to medicines that otherwise might not be available, and addressing clinical unmet needs. Essential Pharma operates globally in more than 70 countries, supplying a portfolio of products with a focus on the central nervous system (CNS), gastroenterology, ophthalmology, and rare disease. The group’s growth strategy is centred on portfolio optimisation and a targeted M&A approach to acquire commercial and late-clinical stage assets in the four therapeutic areas of focus. It is a trusted partner to multiple pharma companies of all sizes, with a proven history of integrating assets and managing complex technology transfers seamlessly while ensuring continuous supply to patients. For more information, please visit essentialpharmagroup.com About Gyrus Capital Gyrus Capital is a European investment firm dedicated to transformational investments in the healthcare and sustainability sectors. Based in Geneva, Switzerland, Gyrus invests in businesses that address the structural needs of society and the environment and are positioned for long-term sustainable growth. The firm focuses on complex transactions, particularly corporate carve-outs in the €50 million to €500 million range. A renowned group of experienced partners and industry experts supports Gyrus’s active investment and value-creation approach, working closely with entrepreneurs and managers. To learn more, please visit www.gyruscapital.com About Sixth Street Sixth Street is a global investment firm with over $80 billion in assets under management and committed capital. Sixth Street uses its long-term flexible capital, data-enabled capabilities, and One Team culture to develop themes and offer solutions to companies across all stages of growth. Sixth Street Healthcare and Life Sciences finances the development and commercialization of innovative therapeutics and invests in healthcare technology companies across all stages of growth. Investments in the sector include Apellis Pharmaceuticals, Arrowhead Pharmaceuticals, Arsenal Biosciences, Biohaven, Blueprint Medicines, Caris Life Sciences, Chroma Medicine, ConcertAI, Datavant, Immunogen, Ironwood, and Mammoth Biosciences, among many others. Founded in 2009, Sixth Street has more than 650 team members including over 250 investment professionals operating around the world. For more information, visit www.sixthstreet.com, or follow Sixth Street on LinkedIn. CONTACTS Essential Pharma Emma Johnson, CEO Tel: +44(0)1784 477 167 Email: info@essentialpharmaceuticals.com ICR Healthcare Tracy Cheung/Sukaina Virji/Isabelle Abdou Tel: +44 (0) 20 3709 5700 Email: Essentialpharma@icrhealthcare.com