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.
FogPharma | November 22, 2022
FogPharma®, a biopharmaceutical company pioneering a new class of precision medicines that could ultimately prove applicable to the vast majority of therapeutic targets, including those previously considered “undruggable,” today announced a $178 Million Series D financing. The financing round includes new investors ARCH Venture Partners, Milky Way Investments and Fidelity Management & Research Company and existing investors VenBio Partners, Deerfield Management, GV, Cormorant Asset Management, funds and accounts advised by T. Rowe Price Associates, Inc., Invus, Farallon Capital Management, HBM Healthcare Investments, Casdin Capital, and PagsGroup, also participated.
Proceeds from the Series D financing will be used to advance and accelerate FogPharma’s growing pipeline of hyperstabilized α-helical polypeptide therapeutics, a proprietary new class of drugs designed to overcome the limitations of today’s precision medicines with broad applicability to the vast majority of disease targets and therapeutic areas. FogPharma’s lead Helicon polypeptide development candidate, FOG-001, a first-and-only-in-class direct TCF-blocking β-catenin inhibitor with potential applicability to significant cancer patient populations, is expected to enter clinical development in mid-2023. In addition, FogPharma is advancing other first-in-class programs against important, biologically validated cancer targets that have remained elusive to other approaches including TEAD, NRAS, Pan-KRAS, ERG and Cyclin E1.
“FogPharma continues to make rapid progress on our moonshot mission to achieve universal druggability – a world where no targets are off-limits to medicine. We believe that Helicon polypeptides, a compelling new therapeutic modality, represent the future of precision medicine. We are thrilled by the support of our investors and will continue to build our platform capabilities, product pipeline which aims to address a significant percentage of cancer patient populations, and our phenomenal team across all levels as we aim to create one of the most impactful new classes of drugs in history.”
Gregory Verdine, Ph.D., founder, chairman and chief executive officer of FogPharma
In connection with the Series D Financing, Rick Klausner, M.D., has been appointed to FogPharma’s board of directors. In addition, Dr. Verdine has been appointed as chairman of the board.
“The team at FogPharma is building an unprecedented new therapeutic modality and robust pipeline with the potential to make a meaningful difference in the lives of cancer patients,” said Dr. Klausner. “I am excited to join the board of directors and be part of something special – particularly at this important time as FogPharma continues to impressively scale its science, team, operations and infrastructure, with the goal of advancing its first Helicon polypeptide therapeutic into the clinic.”
Dr. Klausner is currently the founder and chief scientist of Altos Labs and founder and chairman of Lyell Immunopharma. Dr. Klausner was founder and director of Juno Therapeutics and founder and director of GRAIL. He is also the chairman of Sonoma Biotherapeutics and co-founder and chairman of Lifemine Therapeutics. Previously, Dr. Klausner served as senior vice president, chief medical officer and chief opportunity officer of Illumina Corporation and as executive director for global health for the Bill and Melinda Gates Foundation. Dr. Klausner was appointed by Presidents Clinton and Bush as the eleventh director of the U.S. National Cancer Institute (NCI) between 1995 and 2001. Dr. Klausner served as chief of the Cell Biology and Metabolism Branch of the National Institute of Child Health and Human Development as well as a past president of the American Society of Clinical Investigation. He has served in senior advisory roles to the U.S., Norwegian, Qatari and Indian governments.
Universal Druggability Platform and Helicon™ Polypeptide Therapeutics Existing drug classes are limited in both reach and applicability, with more than 80% of known human protein disease targets considered “undruggable” because they are beyond the reach of both antibodies and small molecules.
FogPharma’s Helicon peptide drug discovery engine integrates directed evolution, proprietary α-helix conformational hyperstabilization chemistry, highly multiplexed drug optimization technology, artificial intelligence including deep learning and machine learning, structure-based drug discovery, cancer genomics and biology, and multiscale manufacturing to rapidly discover Helicon polypeptide therapeutics. This novel therapeutic modality combines the targeting strength and specificity of antibodies with the broad tissue distribution, intracellular target engagement and oral dosing optionality of small molecules to address the limitations of today’s precision medicines and reach the most difficult targets – achieving universal druggability.
FogPharma’s lead Helicon polypeptide development candidate, FOG-001, a first-and-only-in-class direct TCF-blocking β-catenin inhibitor. Dysregulation of the Wnt/β-catenin signaling pathway has been shown to occur in at least 20% of all human cancers. In the U.S. alone, FOG-001 has the potential to become a new treatment option for >1 million patients suffering from a broad range of intractable cancers.
In biochemical and cellular studies, FOG-001 has been shown to potently, precisely and selectively disrupt the interaction of β-catenin with its obligate downstream transcription factor, TCF. Preclinical studies have demonstrated the ability of FOG-001 to cause tumor growth inhibition and regression by disrupting β-catenin-dependent signaling.
FOG-001 is the inaugural member of FogPharma’s TCF-Catenix family of direct-acting β-catenin antagonists and combines key features that distinguish it from previously reported Wnt/β-catenin pathway modulators: FOG-001 acts inside the cell, where it directly binds the key oncogenic driver β-catenin; and FOG-001 blocks TCF-β-catenin engagement at the most downstream node in the canonical Wnt pathway, thus abrogating the signal transmission mechanism by which most, if not all, known Wnt pathway mutations are believed to drive oncogenesis.
FogPharma is a biopharmaceutical company pioneering the discovery and development of Helicon™ polypeptides. Through this novel therapeutic modality, FogPharma aims to address the limitations of existing drug classes and achieve universal druggability – a world where no targets are off-limits to new medicines. Spun out of Harvard University by pioneering academic scientist and successful biotech company builder Dr. Gregory Verdine, FogPharma is advancing a broad pipeline of Helicon polypeptide therapeutics against important and biologically validated cancer targets that have remained elusive to other approaches, with the goal of providing new targeted treatment options for significant cancer patient populations. FogPharma is headquartered in Cambridge, Mass., and has raised more than $360 million to date from leading life sciences investors.
Bionaut Labs | November 29, 2022
Bionaut Labs, the company that uses microscale robots to revolutionize the treatment of central nervous system diseases and disorders, announced $43.2M in a Series B round of financing led by Khosla Ventures, bringing the company’s total financing raised to date to $63.2 million. Also participating in the round are new investors Deep Insight, OurCrowd, PSPRS, Sixty Degree Capital, Dolby Family Ventures, GISEV Family Ventures, What if Ventures, Tintah Grace and Gaingels, along with all existing investors - Upfront Ventures, BOLD Capital Partners, Revolution VC, and Compound.
Many diseases of the brain and central nervous system are hard to treat because it is difficult to deliver therapeutics beyond the blood-brain barrier and reach deep locations in the midbrain with precision. Through magnetic propulsion, Bionauts™ can navigate the depths of the human body to deliver drugs locally, generating efficacy and avoiding side effects and toxicity from systemically delivered drugs. By reaching the midbrain safely through novel routes, Bionaut Labs aims to develop solutions to treat the most debilitating conditions including Parkinson’s disease and Huntington's disease, malignant glioma and hydrocephalus.
Funds will be used to advance clinical development of the company’s lead programs against malignant glioma brain tumors and Dandy-Walker Syndrome (a rare pediatric neurological disorder). Funds will also support further development of its proprietary Bionaut™ treatment platform, allowing future expansion of clinical targets and progression through Bionaut’s two accelerated FDA designations. Bionaut Labs will release major pre-clinical data packages from IDE and IND enabling studies in 2023, with the goal of initiating human clinical trials in 2024.
“There has been a dearth of innovation around treatments for conditions that cause tremendous suffering, in large part because past failures have discouraged even the best of researchers. Bionaut Labs remains committed to finding new ways to treat these devastating diseases, which are long overdue for a breakthrough.”
Michael Shpigelmacher, co-founder and CEO, Bionaut Labs
Bionaut Labs is co-founded by two robotics entrepreneurs, Michael Shpigelmacher and Aviad Maizels, who previously co-founded PrimeSense, the company that developed the facial recognition tech behind iPhone’s FaceID. Its leadership and medical team consists of experts across robotics, neuroscience, biology and drug development.
“We are extremely excited about the transformative potential Bionaut presents in treating debilitating neurological disorders,” said Vinod Khosla, founder of Khosla Ventures. “Anatomically precise treatment will make traditionally-used methods seem archaic, and Bionaut is at the forefront of this movement.”
“Bionaut Labs tackles a complex pharmaceutical problem which many companies have failed to address in the past,” said Giammaria Giuliani, director of the GISEV Family Office. “As pioneers of micro-robotics for CNS treatment, the company enjoys a strong first-mover advantage and carries great promise.”
As neurodegeneration continues to grow in prevalence in the aging global population, Bionaut Labs offers unprecedented therapeutic access to the brain and other hard-to-reach locations in the body, diagnosing and treating diseases that were previously unreachable. Bionaut Labs will transform the way the biopharmaceutical industry develops treatments by offering a mechanism to engineer the therapeutic index for optimal efficacy and safety.
About Bionaut Labs
Bionaut Labs is a biotech company pioneering precision micro-technology with the deployment of microscale robots to remove the barriers of localized treatment and detection of diseases. Magnetic propulsion-controlled Bionauts navigate to deep locations in the human body and brain safely and precisely through non-linear 3D trajectories, making Bionaut Labs the first to access the midbrain through previously inaccessible anatomical routes. Bionauts can perform localized treatment, detection and precise medical procedures to deliver outcomes that were previously unattainable. The FDA has granted Bionaut Labs a Humanitarian Use Device designation for BNL-201, a micro-robot design to treat Dandy Walker Malformation, and an Orphan Drug Designation for BNL 101, a drug-device combination for treatment of malignant glioma.