On September 20, 2022 NanOlogy LLC, a clinical-stage interventional oncology drug company, reported that a review article of preclinical and clinical research supporting its investigational drug, large surface area microparticle docetaxel (LSAM-DTX), has been published in Drug Delivery and Translational Research (DDTR) (Press release, NanOlogy, SEP 20, 2022, View Source;utm_medium=rss&utm_campaign=nanology-publishes-review-article-of-preclinical-and-clinical-research-supporting-lsam-dtx [SID1234619689]).

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The review article entitled Local administration of large surface area microparticle docetaxel to solid carcinomas induces direct cytotoxicity and immune‑mediated tumoricidal effects: preclinical and clinical studies provides a review of preclinical research that supports tumor-directed LSAM-DTX as well as promising results from the high-risk nonmuscle invasive bladder cancer arm of a clinical trial of local LSAM-DTX in urothelial carcinoma, which were published earlier this year in The Journal of Urology. The articles also follows an earlier review of preclinical and clinical research supporting another NanOlogy investigational drug, large surface area microparticle paclitaxel (LSAM-PTX), published in DDTR in 2020.

Highlights from the current article:

LSAM-DTX is retained in the tumor as a depot for continuous drug release after intratumoral injection in a range of solid tumor preclinical models.
LSAM‑DTX in combination with an immune checkpoint inhibitor demonstrated synergy in a preclinical metastatic breast cancer model.
Evidence of efficacy is seen in preclinical xenograft models of bladder, prostate, and renal cancer as well as evidence of an abscopal effect in a preclinical syngeneic renal cell adenocarcinoma model.
In the high risk nonmuscle invasive bladder cancer clinical trial arm (n=19) of a dose escalation/expansion trial of intramural and intravesical LSAM-DTX in urothelial carcinoma, 100%/78%/50% of subjects in the high-dose cohort (n=9) achieved a complete response at the 3/6/12 month timepoints respectively. Moreover, evidence suggesting favorable immunomodulation was reported after analysis of suitable tissue samples collected pre/post administration of LSAM-DTX.
In addition to LSAM-DTX, NanOlogy clinical programs have advanced tumor-directed LSAM-PTX in pancreas, lung, peritoneal, ovarian, prostate, and dermal cancers.

The NanOlogy therapeutic platform is based on a proprietary supercritical precipitation technology that converts active ingredients into stable large surface area microparticles (LSAMs) of pure drug optimized for tumor-directed therapy and continuous drug release to maximize drug delivered to the tumor and minimize systemic toxicity.

Taxane particles are covered by composition of matter patents issued in the US (US 9,814,685, US 10,507,195, US 10,993,927, and US 11,123,322), Canada, Europe, Japan, China, Hong Kong, South Korea, Australia, Indonesia, and Russia valid through June 2036. The composition patents form the foundation of an extensive intellectual property portfolio protecting NanOlogy investigational drugs, formulations, methods, and technology.

On September 20, 2022 Scholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported that its Board of Directors appointed Jay Backstrom, M.D., M.P.H., as Chief Executive Officer-Elect, effective September 20, 2022 (Press release, Scholar Rock, SEP 20, 2022, View Source [SID1234619688]). Dr. Backstrom will work closely with Founding and Interim CEO Nagesh Mahanthappa, Ph.D., M.B.A., before officially assuming the role of President & CEO on October 20, 2022, at which time he will also join the company’s Board of Directors.

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Dr. Backstrom has a unique combination of clinical research and development, regulatory, and leadership experience, which spans 30 years across the biopharmaceutical industry. He has been instrumental in organizing and executing development strategies and has led programs in a broad range of therapeutic areas through regulatory approvals. He most recently served as Executive Vice President, Research and Development at Acceleron Pharma, which was acquired by Merck in 2021. Prior to Acceleron, Dr. Backstrom served as the Chief Medical Officer and Head of Global Regulatory Affairs for Celgene Corporation and spent more than a decade at the company during a time of significant pipeline expansion and revenue growth. Prior to Celgene, he was Vice President, Global Medical and Safety at Pharmion Corporation. Early in his career he held industry roles at Quintiles, Hoechst Marion Roussel, and Marion Merrell Dow.

"Jay is an exceptional executive leader with a track record of success that spans several decades in the biopharmaceutical industry. He is the ideal leader for Scholar Rock, as he has brought transformative therapies targeting the TGFβ signaling pathway through development and into commercialization, including REBLOZYL and sotatercept," said David Hallal, Scholar Rock’s Chairman. "On behalf of the entire Board of Directors, I am thrilled to welcome Jay as Scholar Rock’s next CEO, where his extensive leadership experience and expertise in R&D and regulatory strategy will be critical for the next phase of the company. I would like to thank Nagesh for his remarkable leadership and commitment to Scholar Rock during his 10-year tenure at the company. He has led Scholar Rock from its initial startup phase and built an innovative company with a broad, late-stage pipeline targeting the TGFβ signaling pathway to bring potentially transformative therapies to patients suffering with devastating and life-threatening diseases."

"I am both honored and humbled to have this opportunity to lead Scholar Rock at such a pivotal time, and I am excited about the potential of Scholar Rock’s spinal muscular atrophy and oncology programs, where we have near-term opportunities to develop transformative therapies for patients through a highly differentiated platform targeting growth factors like TGFβ," said Dr. Backstrom. "I look forward to working closely with Nagesh, the Board, the executive team, and Scholar Rock employees to advance our lead clinical assets, build our pipeline, increase our impact on the lives of patients, and grow Scholar Rock for its next phase of success."

During the transition period, Dr. Backstrom will fully engage with the company, working closely with Dr. Mahanthappa, members of the executive team, the R&D team, and other functions to advance the pivotal Phase 3 trial for apitegromab for patients with spinal muscular atrophy, the Phase 1 SRK-181 proof-of-concept study for patients with advanced cancer, and pre-clinical programs from the company’s innovative platform. Dr. Mahanthappa will remain interim CEO until October 20, following which he will step off the Board of Directors and serve as a strategic advisor to Scholar Rock.

Dr. Backstrom holds an M.D. from Temple University School of Medicine, received post-graduate training in Internal Medicine at Temple University Hospital, and holds an M.P.H. from St. Louis University School of Public Health. Dr. Backstrom currently sits on the board of directors of Autolus Therapeutics, Be Biopharma, Disc Medicine, and Lava Therapeutics.

On September 20, 2022 Elicio Therapeutics, a clinical-stage biotechnology company developing a pipeline of novel immunotherapies for the treatment of cancer and other diseases, reported that it has been awarded a $2.8 million grant from the Gastro-Intestinal Research Foundation (GIRF) in Chicago to fund research for two therapeutic cancer vaccines (Press release, Elicio Therapeutics, SEP 20, 2022, View Source [SID1234619687]). Both vaccines have been designed with Elicio’s proprietary lymph node-targeting Amphiphile (AMP) platform that "educates" T cells on how to target particular antigens, such as mutated proteins in cancer. ELI-007 is being developed to target the BRAF gene mutation, and ELI-008 is being developed to target hotspot mutations in p53 in solid tumors including colorectal cancer, melanoma and non-small cell lung cancer (NSCLC). BRAF V600E mutations are present in 40% of melanoma, 10% of colon cancer and 2% of lung cancer while mutations in p53 are found in approximately 60% of patients with solid tumors.

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"We are excited to receive the grant from GIRF and look forward to broadening our pipeline by developing ELI-007 and ELI-008 to target these key cancer mutations. p53 hotspot mutants and mutant BRAF are examples of public cancer neoantigens shared across many patients and tumor types with limited therapeutic options for providing durable therapeutic benefit," said Peter DeMuth, Ph.D., Chief Scientific Officer at Elicio. "By targeting vaccine components to the lymph nodes, the AMP strategy is ideally suited to enhance tumor-specific immunity with the potential to eradicate tumors and promote durable patient responses."

Previous research has shown that T cells can respond to the driver mutation V600E in BRAF and that transfer of tumor-infiltrating lymphocytes that recognize mutated BRAF resulted in a durable complete response in a case study. In addition, small molecule inhibitors generate initial responses in BRAF V600E-mutated melanoma, but these are not sustained because of resistance due to alternative growth signaling pathways, and few initial responses occur in BRAF-mutated colon cancer. The protein expression of BRAF V600E is maintained at high levels in these tumors suggesting that they would be susceptible to T cells specific for the mutated BRAF.

Christopher Haqq, M.D., Ph.D., Executive Vice President, Head of Research and Development and Chief Medical Officer, added, "These mutations are recognized by some patients’ T cells at low levels, and when T cells recognizing tumor-associated antigens like p53 were present, progression-free survival in NSCLC was prolonged. The development of ELI-007 and ELI-008 represents the next level for our current pipeline of lymph node-targeted assets that includes ELI-002, a therapeutic cancer vaccine, which is currently in Cohort 3 of a Phase 1/2 trial in patients with mKRAS tumors. We have prioritized application of our AMP platform to promote immunity against validated cancer targets like BRAF and p53, so the support from GIRF, which is at the forefront of research in the gastrointestinal space including within oncology, is a great opportunity to work together to improve patient outcomes."

Jackie Casey, J.D., Executive Director, Gastro-Intestinal Research Foundation, added, "Patients have been at the center of our work at GIRF from the very beginning, and this grant is reflective of our mission to transform lives through groundbreaking research. Elicio’s AMP platform has demonstrated the potential to amplify the number of T cells and expand their function with promising data. With directed funds from a generous donor, we are proud to support this innovative biotech company as it pushes boundaries in therapeutic cancer vaccine development."

About the Amphiphile Platform

Our proprietary Amphiphile, or AMP, platform delivers investigational immunotherapeutics directly to the "brain center" of the immune system – the lymph nodes. We believe this site-specific delivery of disease-specific antigens, adjuvants and other immunomodulators may efficiently educate, activate and amplify critical immune cells, potentially resulting in induction and persistence of potent adaptive immunity required to treat many diseases. In preclinical models, we have observed lymph node-specific engagement driving therapeutic immune responses of increased magnitude, function and durability. We believe our AMP lymph node-targeted approach will produce superior clinical benefits compared to immunotherapies that do not engage the lymph nodes.

Our AMP platform, originally developed at the Massachusetts Institute of Technology, or MIT, has broad potential across cancers, infectious diseases and other disease indications to advance a number of development initiatives through internal activities, in-licensing arrangements or development collaborations and partnerships.

The Amphiphile platform has been shown to deliver immunotherapeutics directly to the lymph nodes by latching on to the protein albumin, found in the bloodstream, as it travels to lymphatic tissue. In preclinical models, we have observed lymph node-specific engagement driving therapeutic immune responses of increased magnitude, function and durability.

On September 20, 2022 Cellistic, the cell therapy development and manufacturing business of Ncardia BV, and Celyad Oncology (Euronext & Nasdaq: CYAD), a clinical-stage biotechnology company focused on the discovery and development of chimeric antigen receptor T cell (CAR T) therapies for cancer, reported a transaction whereby Cellistic will acquire Celyad Oncology’s Good Manufacturing Practice (GMP) grade cell therapy manufacturing capability, including the existing facility and all related personnel (the "Manufacturing Business Unit") (Press release, Celyad, SEP 20, 2022, View Source [SID1234619686]).

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Under the terms of an asset purchase agreement between Celyad Oncology and Cellistic, Cellistic agreed to acquire Celyad Oncology’s Manufacturing Business Unit in Mont-Saint-Guibert, Belgium, for a total consideration of €6 million. Celyad Oncology’s experienced manufacturing team will join Cellistic. The transaction is subject to a number of customary conditions precedent and is anticipated to close in the fourth quarter of this year.

"We at Cellistic are incredibly excited to welcome this uniquely talented team into our organization," said Stefan Braam, founder and CEO of Cellistic. "We’re bringing aboard a group of people whose passion and capabilities align incredibly well with our vision for the future of cell therapy. As a joined force, we have the talent and resources to further accelerate work on our proprietary platforms and the capability to enable Cellistic’s partners to bring iPSC-based allogeneic cell therapies to patients faster."

Michel Lussier, co-founder and interim CEO of Celyad Oncology, said, "We have focused our efforts on an allogeneic approach for the past few years and our manufacturing facility and staff has been a key element to enable many of our past trials, but has been underutilized in recent years as we mainly used the facility for our autologous candidates. Our current allogeneic programs are better suited for outsourced manufacturing. Through existing materials manufactured at Celyad, we have ensured the means to continue our clinical programs with cryopreserved cells until 2024. Based on this strategy, we are confident that this decision to transfer our manufacturing facility and the staff to Cellistic, who is the perfect company for such an agreement, will allow us to further execute on our business goals in the future."

Cellistic will invest substantial capital into the newly acquired 11,000 square foot facility, which will be optimized for its iPSC-based allogeneic cell therapy platforms and processes creating the world’s first purpose built facility to support customers from cell reprogramming and master cell banking through clinical trial material manufacturing. A team of more than 30 manufacturing, quality and related personnel from Celyad Oncology, all with substantial cell therapy manufacturing and immune-oncology experience, will join Cellistic as part of this transaction.

Celyad Oncology will provide additional guidance on the future business strategy of the Company in the fourth quarter of this year.

On September 20, 2022 Artiva Biotherapeutics, Inc., a clinical stage company whose mission is to deliver highly effective, off-the-shelf, allogeneic natural killer (NK) cell-based therapies that are safe and accessible to cancer patients, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s investigational new drug (IND) application for AB-201 (Press release, Artiva Biotherapeutics, SEP 20, 2022, View Source [SID1234619685]). AB-201 is an allogeneic HER2-targeted chimeric antigen receptor NK (CAR-NK) cell therapy for the treatment of solid tumors in the outpatient setting with the option for repeat dosing. Starting in the first half of 2023, Artiva plans to conduct a clinical study of AB-201 in HER2-expressing cancer patients at multiple clinical sites in the U.S.

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"AB-201 is the first systemically administered, CAR-NK cell therapy candidate targeting the most prevalent HER2-positive cancer indications, including breast and gastric carcinomas," stated Fred Aslan, MD, CEO at Artiva. "Our Manufacturing-First strategy and highly scaled AlloNKTM platform has now demonstrated the ability to produce both non-engineered and CAR-NK product candidates for off-the-shelf use in the outpatient setting, with the potential to treat thousands of patients from single umbilical cord blood donors."

"There is a significant unmet need for effective therapies in the treatment of HER2-positive tumors, and AB-201 has demonstrated compelling preclinical anti-tumor activity," said Dr. Edith Perez, a member of Artiva’s Board of Directors.

HER2, also known as ErbB-2, is a member of the epidermal growth factor receptor (EGFR) family of receptors and an oncogene that is highly expressed on the surface of several types of cancers. HER2 status is a predictive factor in several cancer types, and multiple HER2-directed agents, including monoclonal antibodies, antibody-drug conjugates, and small molecule tyrosine kinase inhibitors have been developed for use in the treatment of HER2-positive cancer. However, most patients will eventually experience a relapse or progression of their disease, and once HER2-directed therapies have been exhausted, patients may be offered cytotoxic chemotherapy that provides only a modest benefit. The absence of safe and effective treatments for patients who have exhausted HER2-directed options represents an important unmet medical need.

AB-201 utilizes Artiva’s AlloNKTM manufacturing platform and proprietary CAR design. A novel, high affinity anti-HER2 antibody converted to scFv structure confers highly specific tumor targeting and is coupled with Artiva’s unique costimulatory structure and IL-15 expression for enhanced activity and persistence. AB-201 has demonstrated potent anti-tumor activity in multiple preclinical HER2-positive tumor model systems. The underlying NK cell is derived from umbilical cord blood donors preselected for advantageous attributes including the high affinity variant of the CD16 receptor and a KIR-B haplotype. AB-201 cell products maintain high expression of CD16, as well as other activating innate cell tumor engaging receptors, enabling the potential for dual targeting therapeutic approaches via monoclonal antibody combinations. The resulting CAR-NK is manufactured at very large scale and cryopreserved in infusion-ready media to enable repeat clinical administrations in the outpatient setting.