CELL AND GENE THERAPY
Rocket Pharmaceuticals, Inc. | September 21, 2022
Rocket Pharmaceuticals, Inc. a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, and Renovacor, Inc. a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases, today announced a definitive agreement under which Rocket will acquire Renovacor in an all-stock transaction for an implied value of approximately $2.60 per share, based on the volume weighted average trading price of Rocket shares of $15.51 for the 30 trading days through and including Monday, September 19, 2022. The boards of directors of both companies have unanimously approved the transaction, which is currently expected to close by the first quarter of 2023.
“The acquisition of Renovacor aligns with our strategy to expand our leadership position in AAV-based gene therapy for cardiac disease and gives us a perfect opportunity to continue on our mission to transform the lives of heart failure patients through the power of gene therapy. Building on our success in Danon Disease to date, I am particularly excited to expand our cardiology focus and capabilities and address a clear unmet medical need in BAG3-associated dilated cardiomyopathy. By combining Renovacor’s compelling preclinical work with our joint clinical, regulatory and CMC expertise, we believe we will be well-positioned to bring the highest impact gene therapy with the best chance for success to these patients in the most productive and efficient manner possible.”
Gaurav Shah, M.D., Chief Executive Officer of Rocket
Dr. Shah continued, “Given the positive pediatric safety data previously announced from our Phase 1 RP-A501 Danon Disease program, and the upcoming pediatric efficacy data and longer-term adult cohort data we anticipate presenting at the Heart Failure Society of America (HFSA) Scientific Meeting at the end of this month, this strategic acquisition gives us what we believe is the broadest platform in the field to address these devastating rare cardiac diseases. Furthermore, the acquisition will bring to Rocket key personnel, namely a team of leading cardiology drug development experts, critical capabilities, and valuable IP to support continued development of the BAG3 as well as other potential cardiac programs, including a gene therapy research collaboration for arrhythmogenic cardiomyopathy.”
Renovacor’s most advanced program, REN-001, is an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure. BAG3-DCM represents a significant unmet medical need in a patient population with rapidly progressive cardiac dysfunction in whom no treatments targeting the underlying mechanism of disease exist. Renovacor has deep technical expertise in the development of precision therapies that address genetically driven cardiac diseases. Further, Renovacor is supported by world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM. These assets and capabilities, all together, represent tremendous value and will enhance Rocket’s leading position in cardiac AAV-based gene therapy.
“Renovacor has made tremendous progress in advancing targeted gene therapies to address the high unmet medical needs of patients living with genetically driven forms of heart disease,” said Magdalene Cook, MD, Chief Executive Officer of Renovacor. “Our experienced team is excited to join Rocket in a shared vision of broadening patient access to precision medicines for cardiovascular disease and addressing common barriers jointly. We look forward to combining the considerable resources and expertise of Renovacor and Rocket in creating a category leader in the precision cardiology field. As a result of this combination, we will be suspending current guidance regarding preclinical and clinical timelines for our programs as we evaluate these items with the Rocket team.”
It is currently anticipated that the transaction will close by the first quarter of 2023, subject to approval by Renovacor and Rocket shareholders, receipt of any required customary regulatory approvals and the satisfaction of other customary closing conditions. RTW Investments, LP, a significant shareholder of both Rocket and Renovacor, has entered into a voting agreement with Renovacor, pursuant to which they have agreed, among other things, and subject to the terms and conditions of the agreement, to vote in favor of the Renovacor acquisition as a Rocket stockholder.
SVB Securities is serving as exclusive financial advisor and Goodwin Procter LLP is serving as legal counsel to Rocket. Wells Fargo Securities is serving as exclusive financial advisor and Troutman Pepper Hamilton Sanders LLP is serving as legal counsel to Renovacor.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
Renovacor is a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. The company’s lead program in BAG3-associated dilated cardiomyopathy (DCM) uses gene transfer technology to address the monogenic cause of this severe form of heart failure. Renovacor’s vision is to bring life-changing therapies to patients living with serious genetic cardiovascular and related diseases, by developing medicines that target the underlying cause of disease and provide a transformative benefit and significant improvement to quality of life.
CELL AND GENE THERAPY
Biocytogen | November 28, 2022
Biocytogen Pharmaceuticals Co., Ltd. announced that it has entered into an evaluation and option agreement with ADC Therapeutics SA. Biocytogen will grant ADC Therapeutics a license to evaluate Biocytogen’s proprietary antibodies against three tumor targets, with an option to license selected antibodies at a later date for global ADC development and commercialization. Biocytogen reserves all global rights for these antibodies beyond ADC development. Biocytogen will receive an upfront payment. For each option exercised, Biocytogen will be entitled to an option-exercise fee, and development and commercial milestone payments, which potentially total tens of millions of US dollars, as well as single-digit royalties on net sales.
Under the agreement, Biocytogen will provide ADC Therapeutics with monoclonal and/or biparatopic antibodies directed against three tumor targets, generated through Biocytogen’s Project Integrum. ADC Therapeutics will carry out feasibility studies and be responsible for further development and commercialization of the ADC products if the company exercises an option.
“ADC Therapeutics is a leader in the development and commercialization of ADCs. We believe this collaboration with ADC Therapeutics will underscore the value of Biocytogen’s proprietary antibody discovery and screening platforms. We’ve been dedicated to expanding the possibilities of antibody-based therapeutics in disease treatment, through the exploration of new types of targets and modalities through Project Integrum. The combination of Biocytogen’s strength in antibody development with ADC Therapeutics’ expertise and track record in payload and linker technology may lead to the generation of novel ADC medicines for unmet medical needs in the future.”
Dr. Yuelei Shen, President and CEO of Biocytogen
“We are pleased to enter this collaboration with Biocytogen as we continue expanding our toolbox to realize the full potential of our ADC platform,” said Patrick van Berkel, PhD, Chief Scientific Officer of ADC Therapeutics. “We look forward to evaluating the fully human antibodies from Biocytogen’s proprietary discovery platform, which have the potential to accelerate the development of our novel ADCs.”
Biocytogen is a global biotechnology company that drives the research and development of novel antibody-based drugs with innovative technologies. Using its proprietary RenMabTM /RenLite® mice platforms for fully human monoclonal and bispecific antibody development, Biocytogen has integrated its in vivo drug efficacy screening platforms and strong clinical development expertise to streamline the entire drug development process. Biocytogen is undertaking a large-scale project to develop first-in-class and/or best-in-class antibody drugs for more than 1000 targets, known as Project Integrum. This project has resulted in 28 drug co-development agreements and 16 RenMiceTM licensing agreements with companies around the world, including several partnerships with multinational pharmaceutical companies (MNCs). The company's pipeline includes 12 core products, among which two products are in phase II multi-regional clinical trials and two products are in phase I. Headquartered in Beijing, Biocytogen has branches in Haimen Jiangsu, Shanghai, Boston, USA and Heidelberg, Germany.
About ADC Therapeutics
ADC Therapeutics is a commercial-stage biotechnology company improving the lives of those affected by cancer with its next-generation, targeted antibody drug conjugates. The Company is advancing its proprietary PBD-based ADC technology to transform the treatment paradigm for patients with hematologic malignancies and solid tumors.
ADC Therapeutics’ CD19-directed ADC ZYNLONTA is approved by the FDA for the treatment of relapsed or refractory diffuse large b-cell lymphoma after two or more lines of systemic therapy. ZYNLONTA is also in development in combination with other agents. In addition to ZYNLONTA, ADC Therapeutics has multiple ADCs in ongoing clinical and preclinical development.
QIAGEN and Neuron23™ | September 15, 2022
QIAGEN and Neuron23 Inc., an early stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases, announced the signing of an agreement to develop a companion diagnostic for Neuron23’s brain penetrant leucine-rich repeat kinase inhibitor for Parkinson’s disease.
Under the new Master Collaboration Agreement, QIAGEN will develop and validate a clinical trial assay that will detect a combination of biomarkers discovered by Neuron23 that together predict the responsiveness of Parkinson’s disease to a LRRK2 inhibitor. The partnership will support the clinical development of Neuron23’s drug candidate that is currently in the late stages of preclinical development. Subject to further clinical development, the agreement also covers options for the future development of additional companion diagnostics.
Neuron23 joins a group of more than 25 leading pharmaceutical and biotechnology companies who have reached master collaboration agreements with QIAGEN to develop and commercialize companion diagnostic tests for their drug candidates – a deep pipeline of potential future products to advance precision medicine for the benefit of patients around the world.
The assay for this collaboration will be integrated into a next-generation sequencing workflow that leverages QIAGEN’s Sample to Insight capabilities, including instrumentation, IVD sample preparation, library preparation and bioinformatics. The workflow is planned to be developed using the NextSeqTM 500 System as part of the NGS strategic collaboration between QIAGEN and Illumina. Based on Neuron23’s artificial intelligence (AI)-enabled drug discovery and biomarker platform, it will target a complex signature of 50 single nucleic polymorphisms for U.S. and European populations and eventually additional SNPs that are prevalent in Asian populations.
“The collaboration with Neuron23 shows the rapid momentum precision medicine is gaining in disease areas outside oncology. Our expertise in blood- and NGS-based molecular testing from Sample to Insight will enable Neuron23 to run a clinical trial for a drug candidate that may have the potential to modify the course of an inexorable neurodegenerative disease in a genetically defined population.”
Jonathan Arnold, Vice President, Head of Oncology and Precision Diagnostics, at QIAGEN
“This collaboration combines the leading expertise of Neuron23 in drug discovery, data science, and machine learning with QIAGEN’s long-standing experience and global leadership in companion diagnostic development. QIAGEN’s blood-based test will help to identify patients with Parkinson’s disease who are likely to respond to Neuron23’s LRRK2 inhibitor. The development of a companion diagnostic identifying this sub-population of Parkinson’s disease patients will de-risk the clinical development of Neuron23’s LRRK2 inhibitor and help identify individuals who may benefit from this disease-modifying therapy. We are excited to be working with an industry leader on the first companion diagnostic developed for Parkinson’s disease,” said Nancy Stagliano, Ph.D., CEO of Neuron23.
No laboratory tests are currently available for the diagnosis of non-genetic cases for Parkinson’s disease. Usually, the disease is diagnosed based on medical history and neurological examination. Although no cure currently exists for Parkinson’s disease, therapies are used to alleviate some symptoms.
LRRK2 is a complex, multidomain protein found in neurons and many tissues and cell types throughout the body. Mutations in the LRRK2 gene are one of the most common causes of familial Parkinson’s disease and individuals who inherit gain of function mutations in LRRK2 are clearly at higher risk to develop the disease in later life. Additionally, there is emerging evidence that LRRK2 may play a role in a subset of the larger population of patients with non-familial Parkinson’s disease. Recent investigations have shown that small-molecule LRRK2 inhibitors can be neuroprotective, suggesting that therapies targeting LRRK2 could be beneficial in a larger population of patients.
QIAGEN is a pioneer in precision medicine and the global leader in collaborations with pharmaceutical and biotechnology companies to co-develop companion diagnostics, which detect clinically relevant genetic abnormalities to provide insights that guide clinical decision-making in diseases such as cancer. QIAGEN has an unmatched depth and breadth of technologies from NGS to polymerase chain reaction and digital PCR for companion diagnostic development. QIAGEN has ten PCR based companion diagnostic indications that are FDA approved, including therascreen EGFR for non-small cell lung cancer, therascreen KRAS for colorectal cancer and NSCLC, therascreen FGFR for urothelial cancer, therascreen PIK3CA for breast cancer based on tissue or plasma samples and the therascreen BRAF kit for colorectal cancer.
QIAGEN N.V., a Netherlands-based holding company, is the leading global provider of Sample to Insight solutions that enable customers to gain valuable molecular insights from samples containing the building blocks of life. Our sample technologies isolate and process DNA, RNA and proteins from blood, tissue and other materials. Assay technologies make these biomolecules visible and ready for analysis. Bioinformatics software and knowledge bases interpret data to report relevant, actionable insights. Automation solutions tie these together in seamless and cost-effective workflows. QIAGEN provides solutions to more than 500,000 customers around the world in Molecular Diagnostics, Applied Testing, Pharma and Academia. As of June 30, 2022, QIAGEN employed more than 6,100 people in over 35 locations worldwide.
Neuron23™ Inc. is an early stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases. Neuron23 combines recent advances in human genetics with a state-of-the-art artificial intelligence (AI)-enabled drug discovery and biomarker platform to advance therapeutics for devastating diseases. The Company’s focus areas are neurodegenerative diseases, neuroinflammatory diseases, and systemic autoimmune and inflammatory diseases. Founded in 2018, Neuron23 has assembled a world-class team of experts and entrepreneurs located in South San Francisco, CA.