On September 20, 2022 Exscientia (Nasdaq: EXAI), ETH Zurich, the Medical University of Vienna, and the Center for Molecular Medicine (CeMM) reported a new publication in Blood Cancer Discovery, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), titled "Deep Morphology Learning Enhances Precision Medicine by Image-Based Ex Vivo Drug Testing" from the laboratory of Prof. Berend Snijder (Press release, Exscientia, SEP 20, 2022, View Source [SID1234619700]). This post-hoc analysis builds on the transformative work of the EXALT-1 trial, published in Cancer Discovery, by using deep learning algorithms to classify complex cell morphologies in patient cancer tissue samples into disease "morphotypes."

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EXALT-1 was the first prospective trial to demonstrate significantly improved outcomes for late-stage haematological cancer patients using an AI-supported precision medicine platform to guide personalised treatment recommendations as compared to physician’s choice of treatment. In EXALT-1, 40% of patients experienced exceptional responses lasting at least three times longer than expected for their respective disease. The post-hoc analysis published today in Blood Cancer Discovery shows that combining the technology as used in EXALT-1 with new deep learning advancements that take advantage of cell-specific features in high-content images revealed a potential to further increase these patient outcomes.

"Following results of the EXALT-1 study, these findings continue to validate that our AI-guided precision medicine platform has the ability to identify highly actionable clinical treatment recommendations for blood cancers, deepening our insights and enhancing the clinical predictive power of the platform to help patients," said Gregory Vladimer, Ph.D., VP Translational Research at Exscientia and co-inventor of the platform technology. "Cell morphology, or assessing the characteristics of cells, is fundamental to the diagnosis of cancer. Within this research, we were able to utilise deep learning within the platform to improve our ability to identify personalised cancer treatments, leading to improved clinical outcomes for patients. At Exscientia, we are excited to expand the platform’s applications in order to bring personalised medicine to broader populations."

"We believe performing drug screens directly in tumour tissues of cancer patients is a great step forward in understanding tumour complexity compared to traditional cell model systems. The fact that we can now harness the power of deep learning to turn these terabytes of images into actionable insights is very exciting indeed," added Prof. Berend Snijder, Principal Investigator at the Institute of Molecular Systems Biology of the ETH Zurich in Switzerland.

The impact of deep learning on the clinical predictive power of ex vivo drug screening was assessed in a post-hoc analysis of 66 patients over a period of three years in a combined data set of 1.3 billion patient cells across 136 ex vivo tested drugs across haematological diagnoses including acute myeloid leukaemia, T-cell lymphomas, diffuse large B-cell lymphomas, chronic lymphocytic leukaemia and multiple myeloma. Patients receiving treatments that were recommended by the platform’s immunofluorescence analysis or deep learning on cell morphologies showed an increased rate of achievement of exceptional clinical response, defined as a progression free survival period that lasted three times longer than expected for each patient’s respective disease. Post-hoc analyses confirmed that the clinical predictions became more accurate when also considering the drug toxicity on the healthy cells within the tested patient sample.

Exscientia’s precision medicine platform uses custom deep learning and computer vision techniques to extract meaningful single-cell data from high content images of individual patient tissue samples. This analysis generates clinically-relevant insights into which treatments will deliver the most benefit to an individual patient. Further evaluation of individual patient results through Exscientia’s genomics and transcriptomics capabilities may help Exscientia further understand which other patients may benefit from similar treatments. The underlying technology was developed by Dr. Gregory Vladimer and Prof. Berend Snijder while working in the laboratory of Giulio Superti-Furga at the CeMM Research Center for Molecular Medicine in Austria.

On September 20, 2022 ImmunoScape, a pre-clinical biotechnology company focused on the discovery and development of next-generation TCR-T-cell therapeutics reported that it has raised $14M in new financing (Press release, immunoSCAPE, SEP 20, 2022, View Source [SID1234619699]). Existing investor Anzu Partners led the round with participation from new investor Amgen Ventures and Singapore-based global investor EDBI.

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ImmunoScape’s differentiated Deep Immunomics platform utilizes the company’s proprietary combinatorial barcoding technology to enable the discovery and in-depth characterization of rare cancer-specific T-cells at high resolution. ImmunoScape’s platform is able to simultaneously evaluate tens of millions of T-cells in blood samples from hundreds of cancer patients to identify rare clinically relevant T-cell clones. The corresponding TCRs are then evaluated and prioritized to build a diverse portfolio of TCR-T-cell therapies.

By leveraging computational biology and machine learning, together with high throughput screening and evaluation of T-cell clones, ImmunoScape is able to efficiently identify novel T-cell therapy targets and TCR candidates. ImmunoScape has extensively validated its computational platform using virus-specific T-cells and is applying the same methods to build an extensive portfolio of cancer-specific TCRs.

"We have made significant strides in our discovery program and have identified several compelling clinical TCR candidates using our Deep Immunomics platform," said Choon Peng Ng, CEO, ImmunoScape. "The new funding will allow us to expedite our development efforts and help us to advance our therapeutic candidates toward the clinic. We are especially delighted that Amgen Ventures has become an investor and we look forward to working with their team to address important unmet medical needs with ImmunoScape’s technology."

"Amgen invests in promising new solutions to address healthcare’s biggest challenges, especially those that offer unique, value-based approaches that align with our mission to serve patients fighting serious illness," said Philip Tagari, vice president of research (therapeutic discovery), Amgen. "ImmunoScape’s Deep Immunomics and machine learning platforms have the potential to help uncover new treatments as we continue to develop the next generation of innovative medicines. We are excited to work with their team to unlock the full power of this technology."

"With research laboratories in both San Diego and Singapore, ImmunoScape is one of the global pioneers of TCR discovery. Their unique high-throughput TCR discovery and evaluation platform has an unprecedented capacity to test millions of human T-cells against hundreds of cancer antigens," said David Michael, managing partner at Anzu Partners. "From their origins at Singapore’s Agency for Science, Technology, and Research (A*Star), the company’s global team of immunologists are pursuing major breakthroughs in TCR cell therapy. We are delighted to work more closely with Amgen on these important efforts."

On September 20, 2022 Invenra, a company developing next generation therapeutic antibodies, reported that Chief Scientific Officer Daniel Pereira, Ph.D., will present preclinical data supporting the ongoing development of INV322, an antibody-based cancer therapeutic (Press release, Invenra, SEP 20, 2022, View Source [SID1234619698]). The preclinical research findings will be presented at the 13th Annual World Bispecific Summit, Thursday, September 22nd, 2022 and at the Immuno-Oncology Summit, Wednesday, October 12th, 2022, both in Boston.

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The presentation will highlight preclinical data related to the lead candidate INV322, a SNIPER Bispecific Antibody co-targeting CTLA-4 and CD25. In vitro studies have characterized avidity-mediated binding of INV322 to these tumor Treg antigens as well as an antibody dependent cell cytotoxicity (ADCC) mediated mode of action. In vivo studies using a murine surrogate also show potent anti-tumor responses after a single dose and selective regulatory T cell (Treg) depletion in syngeneic mice. Moreover, synergistic anti-tumor activity was also observed in combinations with anti-PD-1 and anti-PD-L1 agents.

"We are very pleased with the strong preclinical data that our research team has generated with INV322 using our proprietary multispecific B-Body Platform. INV322 also appears well tolerated in ongoing toxicology studies and thus we are also encouraged by its therapeutic potential as we progress our IND-enabling activities," said Pereira. "These efforts are also providing validation for both the SNIPER Bispecific antibody therapeutics approach, and our robust multispecific B-Body Platform."

Jonathan Davis, Ph.D., Invenra VP of Innovation and Strategy, will also be presenting on the Invenra B-Body Platform at the 13th Annual World Bispecific Summit. "Our platform has binding, biophysical, and manufacturing properties equivalent to the best conventional monoclonal antibodies, and far superior to the typical bispecific antibody platform," said Davis. "We are very excited to see the B-Body Platform and SNIPER Bispecific antibodies advancing in multiple indications and believe this will be a pivotal platform for bispecific antibody therapeutics."

About INV322

INV322 is a next generation anti-CTLA-4 x CD25 bispecific mAb developed using Invenra’s high-developability B-Body Platform. Using the SNIPER Bispecific approach, the antibody was designed with optimized affinities for both antigens, permitting avid antibody binding specifically when both antigens are present. The antibody was also optimized for an ADCC-mediated MOA and has exhibited excellent developability properties at scale.

On September 20, 2022 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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. (NYSE: RCOR), 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, reported 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 (Press release, Rocket Pharmaceuticals, SEP 20, 2022, View Source [SID1234619697]). The boards of directors of both companies have unanimously approved the transaction, which is currently expected to close by the first quarter of 2023.

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"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," said Gaurav Shah, M.D., Chief Executive Officer of Rocket. "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."

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."

Transaction Details

Under the terms of the definitive agreement, Renovacor shareholders will receive approximately 0.1676 shares of Rocket in exchange for each of their shares in Renovacor (subject to adjustment as described below) and are expected to own approximately 4.6% percent of Rocket equity on a fully diluted basis immediately following the closing of the transaction. The exchange ratio implies an equity deal value of approximately $53 million based on fully diluted shares outstanding and the acceleration and vesting of all earnout shares, or $2.60 per share of Renovacor, 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 exchange ratio is subject to adjustment based on Renovacor net cash at closing.

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.

Investor Webcast Information

Rocket management will discuss the transaction via webcast today, Sept. 20, 2022, at 8:00 a.m. ET. To access the webcast, please register online at: View Source Participants are requested to register a minimum of 15 minutes before the start of the call. The webcast replay will be available on the Rocket website upon completion of the event.

On September 20, 2022 NorthStar Medical Radioisotopes, LLC (‘NorthStar’), a global innovator in the development, production and commercialization of radiopharmaceuticals used for therapeutic applications and medical imaging, and IBA (Ion Beam Applications S.A., EURONEXT), the world leader in particle accelerator technology, reported a new agreement under which NorthStar will purchase two additional Rhodotron TT300 HE electron beam accelerators, and the associated beamlines, from IBA for the production of molybdenum-99 (Mo-99) (Press release, NorthStar Medical Radiostopes, SEP 20, 2022, View Source [SID1234619696]).

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The purchase marks a total of five Rhodotron accelerators that NorthStar has purchased from IBA to date. NorthStar previously purchased two electron beam accelerators from IBA in 2019 for the production of Mo-99, and purchased a third accelerator in 2021 for production of the therapeutic radioisotope actinium-225 (Ac-225). The additional accelerators will be used to further expand NorthStar’s commercial-scale radioisotope production capabilities at its Beloit, Wisconsin headquarters. All NorthStar production processes employ innovative, environmentally preferable technology and are non-uranium based.

NorthStar’s first Accelerator Production facility expansion in Beloit is nearing completion and moving to the final phase of activities required for licensure and FDA approval. Both accelerators are operating at full power and undergoing final testing. Equipment in the adjacent Isotope Processing facility has been installed and is undergoing final testing. Construction of NorthStar’s dedicated Actinium-225 Production facility is also well underway, with shipment of the third IBA Rhodotron accelerator expected by the end of 2022.

"NorthStar continues to invest in the future of nuclear medicine using innovative technology to advance environmentally sustainable radioisotope production, and the purchase of these additional accelerators marks another milestone of our highly productive relationship with IBA," said Stephen Merrick, Chief Executive Officer of NorthStar. "IBA has demonstrated extensive commercial expertise and excellent performance in delivering electron beam accelerators for our Mo-99 production expansion program, and in the design and custom-build of our Ac-225 accelerator. We are proud that IBA is a partner in helping to make these important diagnostic imaging and therapeutic radioisotope products available to advance patient health, and look forward to continuing to work with them."

Olivier Legrain, Chief Executive Officer of IBA commented, "We are delighted to sign this latest agreement with NorthStar Medical Radioisotopes and to continue to deliver innovative solutions for reliable radioisotope supply. IBA’s Rhodotron accelerators provide the most advanced electron accelerator technology in the world, enabling a non-uranium based and highly efficient method for producing medical radioisotopes such as Mo-99, Ac-225 and Cu-67. We look forward to continuing to work with NorthStar to advance research and help patients."