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])."

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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

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.

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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.

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.

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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.

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.

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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.

On September 21, 2021 ImmuneID, Inc., a precision immunology company employing a proprietary platform to identify and therapeutically target antibody interactions that drive immune diseases, reported the appointment of James S. Scibetta as Chief Executive Officer and a member of the Board of Directors (Press release, ImmuneID, SEP 21, 2021, View Source [SID1234590077]). Mr. Scibetta succeeds David Donabedian, Ph.D., Venture Partner of Longwood Fund, who had been serving as CEO and will serve in an advisory capacity to the Company.

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"Jim brings to ImmuneID a proven track record of developing advanced and promising science into therapeutic products that improve patient lives," said Longwood Fund’s Christoph Westphal, M.D., Ph.D., ImmuneID co-founder and Board Chair. "He has demonstrated leadership in corporate strategy and capital formation, along with the establishment of collaborative, high-functioning teams across R&D, bioinformatics, CMC, and commercialization. Jim will be extremely valuable to ImmuneID as it continues to leverage its platform for applications related to diseases that implicate the human immune system, a disease area that is extraordinarily scientifically complex and for which there remains a significant unmet medical need."

"On behalf of the entire board, I would like to thank David for his significant contributions to ImmuneID," continued Dr. Westphal. "Under David’s leadership, the Company has raised over $70 million since its Q4 2020 launch, built out the scientific team, and secured state of the art lab space. I look forward to working further with David on the next Longwood portfolio company."

"The human immune system is highly complex and is involved in a large percentage of human diseases," Mr. Scibetta said. "The ImmuneID platform, developed in the lab of Lasker Award winner and Harvard professor Dr. Steve Elledge, has the ability to generate an unprecedented quantity of biologically relevant human immune response data. These proprietary capabilities, coupled with the emergence of big data analytics driven by AI and machine learning, have positioned ImmuneID to generate the next generation of precision immunology therapeutics for patients with autoimmune, allergy, cancer and infectious diseases."

Mr. Scibetta joins ImmuneID after serving as CEO and a member of the Board of Directors of Maverick Therapeutics Inc., a biotechnology company pioneering conditionally active bispecific T-cell targeted immunotherapies that was acquired by Takeda in April 2021. In this role, he led Maverick in the development of the COBRA Platform, which co-opts proteolytic activity in the tumor microenvironment to generate localized t-cell activation and cell killing. Prior to Maverick, he held various executive leadership roles at Pacira Pharmaceuticals (Nasdaq: PCRX), including President, and previously CFO, overseeing global R&D, CMC, and capacity expansion activities for the development of their drug EXPAREL. He successfully led the company’s 2011 IPO and subsequent debt and equity financings. Previously, Mr. Scibetta served as CFO of Bioenvision Inc. (NASD: BVIN, acquired by Genzyme) and Merrimack Pharmaceuticals. Mr. Scibetta began his career in investment banking, spending over a decade sourcing and executing transactions for a range of public and private healthcare and life sciences companies. He received a B.S. in physics from Wake Forest University and an MBA from the University of Michigan.

About ImmuneID’s Platform and Technology

ImmuneID’s technology platform and the underlying technology is designed to provide insights into human immune response throughout the course of disease progression for the identification and validation of therapeutic targets. The technology was originally developed in the lab of Stephen Elledge, Ph.D., ImmuneID co-founder and Chair of ImmuneID SAB, Lasker Award winner and The Gregor Mendel Professor of Genetics and Medicine, Harvard Medical School.