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Personal Genome Diagnostics and Cleveland Clinic Collaborate to Expand Utility of Liquid Biopsy Applications in Oncology Clinical Research

On September 21, 2021 Personal Genome Diagnostics Inc. (PGDx), a leader in cancer genomics, reported a collaboration with the Center for Immunotherapy and Precision Immuno-Oncology (CITI) and the Cleveland Clinic Lerner Research Institute, Cleveland, Ohio (Press release, Personal Genome Diagnostics, SEP 21, 2021, View Source [SID1234590084]).

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Both parties will collaborate to enhance capabilities within elioTM plasma complete reporting, as well as collaborate on the development of proprietary methods for complex biomarker detection and assay iterations to meet emerging liquid biopsy applications in solid tumors. This strategic collaboration combines Cleveland Clinic’s world-class research and commitment to innovation with the comprehensive PGDx portfolio and actionable genomic information. Both organizations are driven to elevate the standard of care for patients and increase utilization of precision diagnostics within the cancer care continuum.

"We are thrilled to collaborate with Cleveland Clinic, whose dedication to empowering the best care and research possible for patients with cancer, along with an immense track record of superior outcomes, makes them an ideal partner in this venture," said PGDx CEO Megan Bailey. "We look forward to expanding upon the capabilities of elioTM plasma complete and paving the way for a Center of Excellence enabling enhanced testing of ctDNA in cancer."

The CITI ctDNA team will work closely with the Cleveland Clinic Taussig Cancer Institute and the Pathology and Laboratory Medicine Institute to accelerate progress in ctDNA work in cancer patients. This effort is a partnership with Jame Abraham, M.D., Interim Chair, Cleveland Clinic Taussig Cancer Institute and Brian Rubin, M.D., Chairman of the Pathology and Laboratory Medicine Institute at Cleveland Clinic.

Timothy Chan, M.D., Ph.D., Chair of Cleveland Clinic’s Center for Immunotherapy & Precision Immuno-Oncology, said "This collaboration allows us to continue exploring new options to improve treatment strategies and patient outcomes in cancer. By investing in comprehensive testing solutions that utilize non-invasive sampling techniques, we intend to explore the clinical utility of high impact biomarkers such as blood TMB, clonal hematopoiesis, and others, with the aim to improve therapeutic modalities for human cancers."

PharmaCyte Biotech Releases More Positive Results From FDA-Required Biocompatibility Tests

On September 21, 2021 PharmaCyte Biotech, Inc. (NASDAQ: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported the results of a second U.S. Food and Drug Administration (FDA)-required test of biocompatibility of its CypCaps product for pancreatic cancer, which showed that the empty capsule material is "non-hemolytic (Press release, PharmaCyte Biotech, SEP 21, 2021, View Source [SID1234590083])."

The Chief Executive Officer of PharmaCyte Biotech, Kenneth L. Waggoner, said of the positive results, "We’re pleased to report that we have received positive results from the second of the biocompatibility tests performed under Good Laboratory Practices and required by the FDA in connection with PharmaCyte’s clinical hold. The data shows that, as expected, the capsule material does not cause blood cells to lyse either after direct or indirect contact with blood. Moreover, it confirms prior data that was observed previously in animal models and previous clinical trials."

The study, which was performed by a third-party Contract Research Organization in accordance with ISO 10993-4:2017 and ISO 10993-12:2021(E), was designed to determine if the device component of CypCaps (the empty capsule material) can cause the in vitro hemolysis (destruction) of red blood cells. Two different methods were used for the evaluation: (i) a direct contact method where the capsule material was mixed with rabbit blood; and (ii) an indirect method where the capsule material was extracted with saline and mixed with rabbit blood. The hemolytic index of both the empty capsules and the extraction material was such that the Contract Research Organization concluded that the test item in both the direct contact method and indirect contact method is considered as "non-hemolytic."

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, we encourage you to watch the company’s documentary video complete with medical animations at: View Source

Triumvira Announces First Patient with HER2-overexpressing Cancer Dosed with TAC-T Cell Therapy Using the Lonza Cocoon Platform

On September 21, 2021 Lonza and Triumvira Immunologics ("Triumvira") reported that the first patient has been dosed with Triumvira’s investigational T-cell antigen coupler (TAC-T cell) adoptive immunotherapy, TAC01-HER2, in development for the treatment of HER2-overexpressing cancers (Press release, Triumvira Immunologics, SEP 21, 2021, View Source [SID1234590081]). Triumvira’s TAC01-HER2, which harnesses natural T cell activation, was manufactured on Lonza’s proprietary Cocoon Platform at C3i Center Inc in Montréal, Canada. The Cocoon Platform is an automated and closed platform for patient-scale cell therapy manufacturing, designed to overcome manufacturing challenges associated with patient-scale personalized medicines.

This collaboration aims to bring novel immunotherapies to patients for the treatment of HER2-overexpressing cancers. The Cocoon Platform will enable manufacturing efficiencies, robustness, and cost reductions by decreasing the necessary manpower, time, and space requirements and ultimately allow the delivery of potentially curative cellular immunotherapies to more patients, at a higher quality and for a reduced cost. From the start of the collaboration, Triumvira’s TAC-T cell treatment was efficiently translated to Lonza’s Cocoon Platform, involving technology transfer to C3i and engineering runs, to support an investigational new drug (IND) submission in under a year.

Eytan Abraham, Vice President Personalized Medicine, Lonza, commented: "Treating the first HER2-overexpressing cancer patient at MD Anderson with Triumvira’s therapy demonstrates the potential of the Cocoon Platform to manufacture therapies at third party manufacturing sites using a decentralized model. The fact that the timeline for moving a fully automated manufacturing process to IND submission using the Cocoon Platform at a new manufacturing site was less than a year demonstrates the ability to truly accelerate timelines from lab to clinic using the Cocoon Platform. Our goal remains to enable partners to provide personalized immunotherapies at a lower cost, higher quality, and more quickly to critically ill patients."

Paul Lammers, President and CEO, Triumvira, added: "We’re proud to be the first company in the US to dose a patient with a cell therapy manufactured on the Cocoon Platform, which is an innovative and elegant solution that addresses challenges of manufacturing autologous cell therapies. Our proprietary TAC-T technology combined with Lonza’s manufacturing expertise enables us to deliver a high-quality and truly personalized cell therapy and manufacturing process that may one day be used at the point-of-care. We look forward to our continued partnership with Lonza and C3i as we move forward with the clinical trial of TAC01-HER2."

Louisa Petropoulos, CEO, C3i, commented: "C3i Center Inc. is founded on the principle that Canadian breakthroughs in cell and gene therapy for serious diseases like cancer should remain in Canada for the benefit of our health and economy. Experimental treatments like TAC01-HER2 won’t become a reality unless we can produce and refine cells quickly, reliably and affordably, and that is what an automated closed system like the Cocoon Platform brings to the table. We are thrilled to put the Cocoon Platform to the test as part of our suite of cutting-edge technologies as we support this critical clinical trial."

The TAC01-HER2 clinical trial is actively enrolling patients across numerous clinical study sites in the US. More information on Triumvira’s pipeline and programs can be found at www.triumvira.com and details on this clinical trial can be found at clinicaltrials.gov using ClinicalTrials.gov Identifier NCT04727151.

Biocept to Present Data at RAS-Targeted Drug Development Summit Showing Ability of Its Switch-BlockerTM Technology to Detect Rare Cancer Mutations

On September 21, 2021 Biocept (Nasdaq: BIOC), a leading provider of molecular diagnostic assays and services, reported that it will present data on its Target Selector assay formats for the ultra-sensitive detection of KRAS mutations using Switch-Blocker technology, which provides advantages for the assessment of therapeutic tumor response and is cost effective for serial monitoring (Press release, Biocept, SEP 21, 2021, View Source [SID1234590080]). Biocept’s presentation is on Sept. 23 at 2:00 p.m. EDT at the Third Annual RAS-Targeted Drug Development Summit, where the company will also host a virtual booth from Sept. 21-23, 2021.

The Summit brings together academic and biopharmaceutical leaders to share insights and data to advance the successful development of targeted monotherapies and combination strategies for RAS-driven cancers. RAS proteins are frequently mutated in cancers. In particular, KRAS mutations are present in approximately 25% of tumors, making them one of the most common gene mutations linked to cancer. They are drivers of some of the deadliest cancers, including lung, colorectal and pancreatic. As a result, there is significant interest among the biopharmaceutical and medical communities to develop and study new, highly targeted therapies to treat such cancers.

To support these efforts, Biocept offers flexible molecular testing solutions based on advanced technology, including its proprietary Switch-Blocker technology. The company’s Target Selector assays and kits, combined with its Switch-Blocker technology, enables the development of superior assays to detect and characterize genetic alterations in patients with cancer. Switch-Blockers enrich for oncogenic mutations while suppressing wild-type (normal) DNA, resulting in ultra-high sensitivity and specificity.

Biocept’s assays can be used to detect circulating tumor DNA (ctDNA) in tissue, blood and cerebrospinal fluid. For liquid biopsy applications, Switch-Blocker technology offers a 50- to 100-fold increase in mutant allele frequency of detection compared to conventional next-generation sequencing (NGS) and has been validated to 0.02% in blood. The technology offers similar analytical advantages in tissue, with the additional benefit of potentially reducing the Quantity Not Sufficient (QNS) rate because of the assay’s low sample input requirement compared to NGS-based assays.

Biocept offers an expanded KRAS assay to detect a variety of KRAS mutations, as well as assays for a wide range of other mutations that are clinically actionable based on NCCN guidelines. It also can develop custom assays with high sensitivity and specificity based on the unique clinical trial needs of biopharmaceutical companies. These cost-effective assays are significantly less expensive than NGS-based tests, an important consideration for companies conducting clinical drug trials.

"As this emerging area of therapeutic development rapidly grows, more companies are targeting rare and specific mutations with their drug candidates," said Michael Dugan, M.D., Biocept’s Senior Vice President, Chief Medical Officer and Medical Director. "Our patented Switch-Blocker technology identifies those mutations down to a very small mutant allele frequency. This capability can help companies more accurately identify patients who meet clinical trial inclusion criteria and better stratify patients, with the potential to positively impact patient selection, trial size and duration, costs and results."

"Biopharmaceutical companies often require molecular assays that are customized to meet the specific needs of their trials," said Michael Nall, President and CEO of Biocept. "Whether they are interested in one or multiple variants, we have the ability to quickly and cost-effectively build assays that are specific to their mutations of interest—which could then become companion diagnostics for their therapeutics. We look forward to working with companies to help ensure the success of their clinical trial programs, while expanding the market for our test offerings."

The presentation, titled "The Ultra-Sensitive Detection of KRAS Mutations Using Switch-Blocker to Aid Therapeutic Decisions and Monitoring," can be accessed here. To learn more about how Switch-Blockers in combination with Biocept’s array of liquid biopsy capabilities can help support both prognostic and predictive clinical trial enrichment, visit the virtual booth here.

Mirum Pharmaceuticals and Takeda Enter into Exclusive Licensing Agreement to Develop and Commercialize Maralixibat for Rare Pediatric Liver Diseases in Japan

On September 21, 2021 Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) reported that the companies have entered into an exclusive licensing agreement for the development and commercialization of maralixibat in Japan for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA) (Press release, Takeda, SEP 21, 2021, View Source [SID1234590079]). Maralixibat, an investigational, orally administered medication, is being evaluated globally in ALGS, PFIC, and BA.

Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of maralixibat in Japan. Takeda will also be responsible for development, including conducting clinical studies in cholestatic indications.

"Takeda is a leading global biopharmaceutical company with extensive experience in development and commercialization of novel therapies to treat rare diseases as well as gastroenterology and hepatology, making them an ideal partner as we look to accelerate the delivery of maralixibat to children living with rare liver diseases in Japan," said Chris Peetz, president and chief executive officer of Mirum. "As we approach potential commercialization in the United States and complete the recent filing for Alagille syndrome in Europe, our goal is to partner with top companies outside of North America and Europe to ensure global reach for patients with these terrible diseases. We are excited for Takeda to engage in the development of maralixibat and collaborate in our effort to advance this potentially life-changing therapy."

"There is a significant unmet medical need for a treatment to help patients with cholestatic diseases such as ALGS and PFIC in Japan and developing novel treatment for those patients suffering from rare liver diseases is a top priority for Takeda’s global R&D strategy," said Dr. Naoyoshi Hirota, general manager of Takeda development center Japan. "This agreement reinforces Takeda’s commitment to developing highly differentiated medicines to improve the health and quality of life of patients."

Mirum has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome. The NDA is currently under priority review with a PDUFA, or FDA decision date, of September 29, 2021. Mirum also recently submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients with ALGS.

About Maralixibat

Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated effects. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo. In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival. The U.S. Food and Drug Administration has granted maralixibat Breakthrough Therapy designation for the treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was shown to have a tolerable safety profile in the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Maralixibat has been studied extensively and its safety database represents the largest database for an ASBT inhibitor.

Until maralixibat is approved and available for prescribing, the medication is available to patients with ALGS through Mirum’s expanded access program. For more information, please visit ALGSEAP.com. For further information about maralixibat’s ongoing studies in pediatric liver disease, please visit the study websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study for biliary atresia.