On December 16, 2021 Argobio and GeneCode reported their collaboration to develop small molecule Glial cell line – Derived Neurotrophic Factor (GDNF) mimetics for the treatment of Parkinson’s Disease and other indications (Press release, Argobio, DEC 16, 2021, View Source [SID1234597348]).

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In the venture studio model developed by Argobio, breakthrough technologies are embraced by a team of scientific experts with an entrepreneurial and industrial experience. Over the course of incubation, Argobio will fund the project development up to 3 ME and incorporate the start-up. After incubation, further venture funding will be provided by specialized VC investors.

"The launch of our first collaboration is a great achievement for Argobio. Argobio intends to incubate until series A at least five projects in the fields of rare diseases, neurosciences, and oncology over the next three years. With a committed capital of €50 million, and a team of pharma industry experts, Argobio expects to deliver strong projects, well-prepared for Series A funding," Thierry Laugel Argobio Chairman said.

GeneCode, a discovery-stage biotech company developing small molecule GDNF mimetics, in collaboration with the laboratory of Mart Saarma, Professor and Research Director of the Institute of Biotechnology, HI-LIFE, University of Helsinki, Finland, has identified and patented a series of small molecules activating the GDNF signalling receptor RET tyrosine kinase. Professor Saarma´s team has discovered GDNF receptors, clarified the GDNF-receptor structure and investigated the therapeutic potential of GDNF family members.

Professor Mart Saarma commented, "Argobio’s investment and participation in the development of effective GDNF mimetics is extremely encouraging news. GDNF mimetics can overcome the limitations of the GDNF protein and can open up a completely new page in the treatment of Parkinson’s disease."

GeneCode CEO Paavo Pilv added, "Our combined expertise will catapult this technology beyond what we would have been able to achieve alone. Considering the pivotal role of GDNF family ligands and RET signalling pathways for other groups of cells, applications of compounds developed by GeneCode are not limited only to Parkinson’s Disease but can be applied to treat other medical conditions."

"We have ambitious goals for the next two years," Argobio Entrepreneur in Residence Yves Ribeill, PhD said. "The whole is greater than the sum of the parts with the combined, diverse expertise of GeneCode, Argobio and Professor Saarma. I am confident that we will be able to demonstrate that the unique business model of funded partnerships initiated by Argobio in Europe will give new therapeutics a boost to succeed."

On December 16, 2021 Protocol First, a software solutions provider focused on accelerating clinical research,reported that it has joined Flatiron HealthⓇ as a wholly owned subsidiary, the two companies (Press release, Flatiron Health, DEC 16, 2021, https://www.businesswire.com/news/home/20211215006052/en/Protocol-First-Joins-Flatiron-Health [SID1234597346]).

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"This is a win-win for both companies," said Carolyn Starrett, Flatiron CEO. "Flatiron and Protocol First share a common vision in which technology and software increasingly bridge the gap between real-world care and clinical research."

Protocol First’s technology enhances research efficiency by breaking down silos between sites, study sponsors and contract research organizations (CROs). Effective Dec. 14, Protocol First’s employees and operations joined Flatiron’s Clinical Research business unit, which builds on Flatiron’s established leadership in real-world evidence with new technologies that better integrate research into everyday clinical care.

"Flatiron has deep expertise in unstructured-data processing, experience building software that integrates seamlessly into site workflows, and an engaged network of community and academic research sites," said Alex Deyle, General Manager, Clinical Research at Flatiron. "Protocol First’s technology expands and accelerates our ability to drive much-needed innovation in clinical research."

Protocol First aims to reduce the operational complexity of data collection and validation in clinical research by leveraging proven technologies and standards. The team, led by CEO Hugh Levaux and CTO Amit Shah, has developed several industry-defining software solutions that have been adopted by many leading CROs and biopharma companies. The technology is designed to handle the unique complexities of research in oncology as well as other disease areas. Plans are being developed to integrate the direct EHR-to-EDC connector application Clinical Pipe – already in use with major academic medical centers and health systems – with Flatiron’s OncoEMRⓇ, which is used by hundreds of community cancer centers across the U.S.

"We are very excited about this combination, which will take our technology to the next level, scaling and broadening its availability across the life sciences industry," said Levaux, who joins Flatiron as Vice President, Clinical Research. "The transition will be seamless for our customers, and the future belongs to the patients we will all serve with even better solutions."

On December 16, 2021 Menarini Silicon Biosystems (MSB), a pioneer of single cell technologies, reported its new FFPE Rescue Application, to enable molecular testing on formalin-fixed, paraffin-embedded (FFPE) samples with low tumor cell contents (Press release, Menarini Silicon Biosystems, DEC 16, 2021, View Source [SID1234597345]). Thanks to the sorting capabilities of the recently launched DEPArray PLUS instrument, it is possible to isolate a sufficient number of tumor cells to generate relevant molecular data, even from FFPE tissues that were previously discarded from conventional Next Generation Sequencing (NGS) processing. Thus, the DEPArray PLUS workflow can reduce the failure rates of FFPE molecular analyses and provide clinical researchers with valuable information about actionable mutations, potentially improving clinical trial recruitment and allowing more patients to access new targeted therapies.

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Formalin fixation and paraffin embedding remains the gold standard preservation method of human tissue for molecular diagnostics. NGS of FFPE tissue is becoming increasingly important to identify targetable mutations and has greatly enhanced the ability of physicians to optimize treatment strategies.

However, not all FFPE samples are suitable for molecular analyses. The failure rate for molecular testing of FFPE tissues ranges between 5 and 15%, with the primary driver being low tumor content. The precise digital sorting capability of DEPArray PLUS allows for the isolation of small pools of only a few hundred tumor cells that are then suitable for NGS library preparation and sequencing.

According to Dr Javier Hernández Losa, Director of the Molecular Biology Laboratory within the Pathology Department at the Vall d´Hebron University Hospital in Barcelona, Spain "the DEPArray platform allows us to conduct unambiguous genetic analyses on FFPE samples that failed the standard NGS procedure and identify potentially targetable mutations". MSB and the Vall d’Hebron University Hospital have recently engaged in a research collaboration to expand the types of complex samples that can be analyzed by DEPArray, such as cytologic samples. The Vall d’Hebron University Hospital Pathology lab provides molecular testing for many hospitals throughout Catalonia. It aims to establish a NGS testing service across the entire region to provide the information that will help physicians improve their decision-making and patient care.

Prof Lorenzo Leoncini, MD, Head of the Department of Medical Biotechnology and Director of the Pathological Anatomy Division at the University of Siena, Italy adds that: "the DEPArray technology helped to rescue tissue samples with less than a 5% tumor cell content to generate robust data on cancer mutations. We are now planning to use this technology in different new research projects. For example, we will use DEPArray to study tumor cells in classical Hodgkin Lymphoma tissue biopsies, a setting in which isolation of these rare cells is extremely challenging, as they are present in a very low percentage compared to normal cells".

"We are excited to be able to deliver such a unique technology for the analysis of FFPE biopsies with low tumor cellularity," said Fabio Piazzalunga, President and CEO of MSB. "Our DEPArray platform could help pathology labs reduce the significant burden, caused by FFPE biopsies that are rejected from molecular analysis and support pharma companies involved in clinical trials to expedite recruitment".

On December 16, 2021 Debiopharm (www.debiopharm.com), a Swiss biopharmaceutical company, reported the first patient treated in the newly launched open-label, phase I study evaluating Debio 0123, an oral, potent and highly selective WEE1 inhibitor, as monotherapy in patients with advanced solid tumors (NCT05109975). Part of an emerging new class of drugs working within the DNA damage response (DDR) pathway, the compound’s anti-tumor capacity has been evaluated in several preclinical studies along with the ongoing phase I study in combination with carboplatin-based chemotherapy (Press release, Debiopharm, DEC 16, 2021, View Source [SID1234597344]). This new trial’s primary objective is to identify the maximum tolerated dose and/or recommended phase II dose in adults with advanced solid tumors that have recurred or progressed after prior therapy and/or for whom no standard therapy of proven benefit is available.

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The development of the Debio 0123 program is rooted in the growing understanding of the DNA damage response of cancer cells. Research reveals that cancer cell survival relies on the tightly regulated cell cycle that pauses at certain points to allow the repair of damaged DNA so that tumor cells can continue to divide and grow. WEE1 is a catalyzing enzyme implicated in these "DNA repair stops" helping cancer to thrive. By inhibiting WEE1, the cell cycle checkpoints are compromised, driving cancer cells to start their replicating prematurely, or before the repair of detrimental DNA damage, ultimately leading to cell death. Furthermore, WEE1 inhibitors are suspected to selectively target tumor cells, inducing synthetic lethality without impacting survival of normal cells. The potential best-in class status of Debio 0123 relies on its highly selective inhibition against WEE1.

"We’re intrigued to learn more about the clinical benefits that WEE1 inhibition with Debio 0123 alone could offer cancer patients. We believe that this new modality can effectively exploit the genomic instability and malfunctioning of the DNA repair process in cancer cells in hopes that ultimately tumor progression is halted and patient survival is improved," Dr. Esteban Rodrigo Imedio, Senior Medical Director, Oncology Research & Development, Debiopharm. "As Debio 0123 is highly selective against WEE1, in time, ongoing clinical research could confirm Debio 0123’s potential best-in-class status."

Initially discovered by Almac Discovery before being in-licensed by Debiopharm in 2017, the evaluation of Debio 0123 as monotherapy could help to better characterize the safety and efficacy profile of the compound in a clinical setting and define the parameters for eventual phase II research. Pre-clinical research suggests potential activity for cancer patients, particularly in combination with DNA damaging agents such as chemo- and radiotherapy. WEE1 inhibitors are promising drug candidates as they inhibit DDR, offering the possibility to enhance the efficacy of these agents, frequently part of the standard-of-care of various cancer types. Debiopharm plans to advance the clinical program while simultaneously negotiating potential partnerships, such as during the upcoming JP Morgan 2022 conference, with larger pharmaceutical companies for eventual commercialization.

Dr. Stephen Barr, Managing Director & President, Almac Discovery commented, "Since the discovery of our highly selective WEE1 inhibitor, now known as Debio 0123, we have looked forward with anticipation to understanding its potential therapeutic benefit for cancer patients across the globe. We are therefore delighted that, in addition to the ongoing combination clinical study, Debio 0123 is also being evaluated as a monotherapy in the treatment of advanced solid tumors. We look forward to seeing further progress from this ongoing clinical research."

About Debio 0123

Debio 0123 is a WEE1 kinase inhibitor, a key regulator of the G2/M and S phase checkpoints, activated in response to DNA damage, allowing cells to repair their DNA before resuming their cell cycle. WEE1 inhibition, particularly in combination with DNA damaging agents, induces an overload of DNA breaks. In conjunction with abrogation of other checkpoints such as G1, the compound pushes the cells through cycle without DNA repair, promoting mitotic catastrophe and inducing apoptosis of cancer cells.

On December 16, 2021 Inceptor Bio, a Research Triangle Park-based next-generation cell and gene therapy biotechnology company, reported that it has executed an in-licensing agreement with the University of California, Santa Barbara (UCSB) for an investigational Chimeric Antigen Receptor Macrophage (CAR-M) therapy targeting difficult-to-treat tumors (Press release, Inceptor Bio, DEC 16, 2021, View Source [SID1234597343]).

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"This strategic alliance is an important step in advancing Inceptor Bio’s next-generation cell therapy platform focused on multiple novel mechanisms to address solid tumors," said Shailesh Maingi, Founder and CEO of Inceptor Bio. "Our aim for this cutting-edge technology is to transform how solid tumors are treated."

"CAR-M is a new frontier for harnessing immune cells to treat and cure cancer. We’re thrilled to partner with Dr. Denise Montell and UCSB on a potentially groundbreaking technology in the fight against difficult-to-treat cancers," added Mike Nicholson, Ph.D., Chief Scientific Officer at Inceptor Bio.

Macrophage cells naturally engulf viruses and bacteria through a process called phagocytosis, and when combined with a CAR construct to form a CAR-M, they can selectively target and engulf cancer cells and generate an immune response via modulation of the tumor microenvironment. Inceptor’s CAR-M technology builds upon this foundation and plans to optimize the effectiveness of this therapeutic approach.

This technology was invented by Dr. Denise Montell’s research lab at UCSB. Dr. Montell earned her B.S. in biochemistry and cell biology at the University of California, San Diego, and her Ph.D. in neuroscience at Stanford University. She joined the faculty at UCSB in 2012. Dr. Montell serves on the Board of Scientific Counselors of the National Cancer Institute and was elected into the National Academy of Sciences in 2021.

"I am excited to work with Inceptor to transform our fundamental cell biological discoveries into cancer immunotherapies," said Denise Montell, Ph.D., Duggan Professor, UCSB. "It is gratifying to see decades of our basic science research coming to fruition to help cancer patients. The partnership with Inceptor is making it possible."