Strand Therapeutics Awarded Phase I NIH SBIR Contract to Develop mRNA-based CAR-T Immunotherapy for Non-Hodgkin’s Lymphoma

On November 9, 2021 Strand Therapeutics, a privately held biotech company developing next-generation, programmable mRNA therapeutics beyond vaccines, reported that the company was awarded a Phase I Small Business Innovation Research (SBIR) contract from the National Cancer Institute (NCI) of the National Institutes of Health (NIH) to develop an off-the-shelf chimeric antigen receptor T cell (CAR-T) immunotherapy based on the company’s mRNA technology for the treatment of B-cell non-Hodgkin’s lymphoma (NHL) (Press release, Strand Therapeutics, NOV 9, 2021, View Source [SID1234594949]). The total funding amount awarded to Strand is approximately $400,000.

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As one of the most common cancers in the U.S., NHL accounts for approximately 4% of all cancers. According to the American Cancer Society, it is estimated that in 2021 over 80,000 people will be diagnosed with NHL. While CAR-T therapies have shown clinical benefit in patients, many individuals still suffer treatment-induced toxicities including cytokine release syndrome, neurologic complications and adverse effects from lymphodepletion. Furthermore, development of CAR therapies is costly, with manufacturing processes being difficult and time-consuming.

To address these challenges, Strand will use the funds from the contract to develop an in situ, off-the-shelf cell therapy based on the company’s proprietary self-replication, programmable mRNA platform to illicit a targeted CD19 CAR-T response. This novel approach has the capability of providing long-term, temporal and cell-type specific expression, potentially minimizing off-target effects and enabling redosing without lymphodepletion.

"Strand’s in situ CAR delivery approach and its capacity to provide long-term, programmable expression is the first such method that could offer life-saving therapeutics to all patients at a fraction of the cost of currently-available treatments," said Jake Becraft, PhD, co-founder and CEO of Strand. "This opportunity enables us to position ourselves in the field of mRNA-based CAR therapies and sets the stage to further develop our technology in areas of unmet medical need."

The company was recently awarded two Phase I NIH SBIR grants to advance its programmable, long-lasting mRNA therapeutics for melanoma and breast cancer, directed at enhancing the efficacy of anti-PD-1 immunotherapies.

Zymeworks Launches Global Phase 3 Zanidatamab Trial in First-Line HER2‑Positive Gastroesophageal Adenocarcinoma (GEA)

On November 9, 2021 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported, together with its partner BeiGene, the launch of HERIZON‑GEA‑01 (Press release, Zymeworks, NOV 9, 2021, View Source [SID1234594948]). This is a randomized, global Phase 3 study evaluating Zymeworks’ investigational HER2‑targeted bispecific antibody, zanidatamab, plus chemotherapy, with or without BeiGene’s anti-PD‑1‑targeted antibody tislelizumab, versus standard of care (trastuzumab plus chemotherapy), for the first-line treatment of metastatic HER2‑postive GEA.

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"We are incredibly excited to launch our second pivotal, and first Phase 3 clinical trial for zanidatamab, HERIZON‑GEA‑01," said Ali Tehrani, Ph.D., Zymeworks’ President & CEO. "Gastrointestinal cancers have significant unmet patient need and we have the opportunity to help a large and growing patient population. With two potential Biologics License Applications over the next 3 years, we believe zanidatamab has the potential to achieve blockbuster status and position Zymeworks as the leader in the treatment of HER2‑positive GI cancers."

The primary objective of the HERIZON‑GEA‑01 study is to evaluate the efficacy and safety of zanidatamab in combination with physician’s choice chemotherapy [CAPOX (capecitabine/oxaliplatin) or FP (5FU/cisplatin)] with or without tislelizumab compared to trastuzumab plus physician’s choice chemotherapy in subjects with advanced or metastatic HER2-positive GEA. Primary endpoints are progression-free survival by RECIST 1.1, assessed by blinded independent central review, and overall survival.

The HERIZON-GEA-01 study seeks to enroll approximately 700 patients at approximately 300 sites across 38 countries. BeiGene will oversee trial sites in Asia (excluding Japan), Australia and New Zealand, and Zymeworks will oversee trial sites in the rest of the world, including North and South America, Japan, Europe, Middle East, and Africa.

"We are pleased the HERIZON‑GEA‑01 study has begun enrollment and our aim is to establish zanidatamab as the foundational agent of a new standard of care with tislelizumab for the first‑line treatment of HER2‑positive GEA," said Neil Josephson, M.D., Zymeworks’ Interim Chief Medical Officer. "Based on the study design, we expect to have progression-free survival data as soon as 2024, which could enable submission of a supplemental Biologics License Application that same year."

GEA is the fifth most common cancer worldwide and approximately 20 percent of patients diagnosed with this form of cancer are HER2‑positive. HER2‑positive GEA has high morbidity and mortality and patients are urgently in need of new treatment options.

"The encouraging zanidatamab Phase 2 data support its further investigation with tislelizumab in this Phase 3 HERIZON-GEA-01 trial in first-line HER2-positive gastroesophageal adenocarcinomas," said Yong (Ben) Ben, M.D., Chief Medical Officer, Solid Tumors, at BeiGene. "We are excited to continue our collaboration with Zymeworks as we strive to address the unmet medical needs in this patient population by accelerating the development of zanidatamab."

Phase 2 Study Results

In September, Zymeworks presented data at the European Society for Medical Oncology Annual Meeting from a Phase 2 clinical study of 36 patients with HER2‑expressing GEA who received zanidatamab in combination with either CAPOX (n=14), FP (n=2), or mFOLFOX6 (5FU/leucovorin/oxaliplatin; n=20). None of the patients had received prior HER2‑targeted therapies.

In 28 response-evaluable patients with metastatic HER2‑positive GEA, zanidatamab plus chemotherapy resulted in a confirmed objective response rate (cORR) of 75% and disease control rate (DCR) of 89% overall, with a cORR of 93% and DCR of 100% in the proposed Phase 3 regimen of zanidatamab and CAPOX/FP. All patients except one experienced a decrease in their tumor size. Across all treatment regimens, the median duration of response is 16.4 months and the median progression-free survival is 12.0 months, with 61% of patients still on study at the time of data cutoff.

In addition, the data demonstrate that zanidatamab plus chemotherapy is generally well tolerated, with the majority of treatment-related adverse events (TRAEs) considered mild to moderate in severity (Grade 1 or 2). The most common grade ≥ 3 TRAE was diarrhea, which was manageable in the outpatient setting; introduction of prophylactic loperamide reduced the incidence in cycle 1 from 44% to 18%. No severe (grade ≥ 3) infusion-related reactions or cardiac events were observed.

Conference Call and Webcast

The company will host a conference call and webcast to discuss the HERIZON‑GEA‑01 Phase 3 trial design as well as the commercial strategy in HER2‑positive gastrointestinal cancers. The event will be led by Ali Tehrani, Ph.D., Zymeworks’ President and CEO, Neil Josephson, M.D., Zymeworks’ Interim Chief Medical Officer, and James Priour, MBA, Zymeworks’ Chief Commercial Officer. The speakers will be available to answer questions at the conclusion of the call.

Date: Tuesday, November 9th
Time: 4:15 pm ET (1:15 pm PT)

Interested parties can access the live webcast via the Zymeworks’ website at View Source A recorded replay will be accessible after the event through the Zymeworks website.

About Zanidatamab

Zanidatamab is a bispecific antibody, based on Zymeworks’ Azymetric platform, that can simultaneously bind two non-overlapping epitopes of HER2, known as biparatopic binding. Zanidatamab’s unique binding properties result in multiple mechanisms of action including HER2-receptor clustering, internalization, and downregulation; inhibition of growth factor-dependent and -independent tumor cell proliferation; antibody-dependent cellular cytotoxicity and phagocytosis; and complement-dependent cytotoxicity. Zymeworks is developing zanidatamab in multiple Phase 1, Phase 2, and pivotal clinical trials globally as a targeted treatment option for patients with solid tumors that express HER2. The FDA has granted Breakthrough Therapy designation for zanidatamab in patients with previously treated HER2 gene-amplified biliary tract cancer (BTC), and two Fast Track designations to zanidatamab, one as monotherapy for refractory BTC and one in combination with standard of care chemotherapy for first-line gastroesophageal adenocarcinoma (GEA). These designations mean zanidatamab is eligible for Accelerated Approval, Priority Review and Rolling Review, as well as intensive FDA guidance on an efficient drug development program. Zanidatamab has also received Orphan Drug designations from the FDA as well as the European Medicines Agency for the treatment of biliary tract and gastric cancers.

About the Zymeworks-BeiGene Collaboration

In November 2018, Zymeworks and BeiGene entered into license and collaboration agreements in which BeiGene was granted an exclusive license for the research, development, and commercialization of zanidatamab and ZW49 in Asia (excluding Japan), Australia, and New Zealand. The companies are collaborating on joint global development for selected indications, with the goal of developing zanidatamab and ZW49 worldwide across multiple HER2‑expressing cancers and lines of therapy.

Study Shows Biocept’s Switch-Blocker Technology Enhances Performance of Conventional PCR-Based Liquid Biopsy Assays in Detecting Rare Cancer Mutations

On November 9, 2021 Biocept, Inc. (Nasdaq: BIOC), a leading provider of molecular diagnostic assays, products and services, reported the publication of a study showing that the addition of Switch-Blocker technology to common PCR-based liquid biopsy assays significantly increased sensitivity in detecting rare cancer mutations (Press release, Biocept, NOV 9, 2021, View Source [SID1234594947]). The abstract was published in the November 2021 issue of the Journal of Molecular Diagnostics.

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Biocept’s proprietary Switch-Blocker technology enriches oncogenic mutations of interest while suppressing wild-type (normal) DNA, resulting in ultra-high sensitivity, specificity and accuracy. In this study, Switch-Blockers were combined with conventional real-time PCR and droplet digital PCR (ddPCR) assays.

"Our quantitative Switch-Blocker technology demonstrates an unprecedented ability to find and distinguish extremely rare genetic events—even in blood that contains mostly DNA from normal white blood cells," said Michael Dugan, Chief Medical Officer and Medical Director of Biocept. "This can greatly enhance the clinical sensitivity of our cell-free tumor DNA assays and has broad application in the continued development of highly sensitive and quantitative molecular diagnostic assays used to evaluate cerebrospinal fluid or blood from patients with cancer. Switch-Blocker-based assays can help detect cancer biomarkers that otherwise might be missed, improving treatment selection. They can also be used to evaluate treatment-related changes, find minimal residual disease or identify early disease recurrence."

Results showed that the addition of Switch-Blockers increased the sensitivity of allele-specific primer assays by more than 200 times, from about 1% minor allele frequency (MAF) to better than 0.01%. The sensitivity of multiplex competitive allele-specific TaqMan assays, commonly used with PCR amplification, were increased greater than 1,000 times, from about 10% MAF to 0.01% or better. The ability to significantly increase the sensitivity of conventional mutation assays using Switch-Blocker technology is critical for helping to find rare genetic events in a wide range of applications, including solid tumor cancers, where a majority of biomarkers in blood occur at less than 1% MAF.

The abstract (#TT33), titled "The Use of Switch-Blocker Probes for the Ultra-High Sensitivity of Detection of Rare Genetic Events Using Conventional Real-Time and Droplet Digital PCR Assays," can be accessed here.

About Switch-Blocker Technology

Biocept’s proprietary Switch-Blocker platform is the basis for the company’s Target Selector assays and can be used with tissue, blood and cerebrospinal fluid (CSF) samples. The technology enables industry-leading sensitivity for the detection of mutations/variants from circulating tumor DNA (ctDNA). It has been validated to 0.05% minor allele frequency in blood, which provides significant advantages for identifying actionable cancer biomarkers and assessing therapeutic tumor response. Switch-Blockers enhance the performance and specificity of the PCR method, the most widely used amplification approach for clinical diagnostic applications and can be customized to aid in biopharmaceutical research for the development of targeted therapies for cancer. Switch-Blocker technology also has been validated and found to be highly sensitive, quantitative and reproducible in detecting the presence of the SARS-CoV-2 virus that causes COVID-19 infections.

MAIA Biotechnology, Inc. Announces Poster Presentation at the Society for Immunotherapy of Cancer 36th Annual Meeting (SITC)

On November 9, 2021 MAIA Biotechnology, Inc., a targeted therapy, immuno-oncology company focused on developing potential first-in-class oncology drugs ("MAIA"), reported that the Company will present a scientific poster at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting (SITC) (Free SITC Whitepaper) which will be held November 10-14, 2021, in Washington, DC (Press release, MAIA Biotechnology, NOV 9, 2021, View Source [SID1234594946]).

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Poster Title:

Telomerase-Driven Telomeric DNA Modification in Cancer Cells Leads to Efficient Induction of cGAS-mediated Innate and Adoptive Immune Responses


Abstract ID:

697


Category:

Immune-stimulants and immune modulators


Date:

Friday, Nov. 12, 2021


Time:

7:00 am – 8:30 pm


Location:

Poster Hall, Walter E. Washington Convention Center

Accepted abstracts can be found in the Journal for ImmunoTherapy of Cancer (JITC).

EdiGene Enters Research Collaboration with the University of Wisconsin-Madison on in Vivo RNA Editing Therapies

On November 9, 2021 EdiGene, Inc., a global biotechnology company focused on translating gene-editing technologies into transformative therapies for patients with serious genetic diseases and cancer, reported that it has entered into a research collaboration with the University of Wisconsin–Madison as part of the company’s effort of translating proprietary LEAPER RNA editing technology into in vivo therapies (Press release, EdiGene, NOV 9, 2021, View Source [SID1234594945]).

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Under the partnership, EdiGene’s U.S.-based R&D Center will work with the David Gamm Laboratory at the University of Wisconsin-Madison to evaluate the pharmacological property of LEAPER RNA base editing candidates targeting specific genetic diseases.

"EdiGene’s U.S. R&D Center has built up its capabilities with an exceptional team of talented scientists who are advancing the company’s pipeline both within our own labs and through external partnerships," said Bo Zhang, Ph.D., Head of EdiGene’s U.S. subsidiary. "Dr. Gamm’s knowledge and insights are a tremendous asset in this effort."

"This collaboration allows us to leverage our expertise and experience to help translate EdiGene’s RNA base editing technology into transformative medicines that can benefit patients with genetic diseases who today have limited or no therapeutic options," said David Gamm, MD, Ph.D., Principal Investigator of the research, Professor of Ophthalmology and Visual Sciences, and Director of the McPherson Eye Research Institute at the University of Wisconsin–Madison.

LEAPER (Leveraging endogenous ADAR for programmable editing of RNA) employs short engineered ADAR-recruiting RNAs (arRNAs) to recruit native ADAR enzymes to change specific adenosine to inosine. LEAPER is a robust, precise, and efficient RNA editing technology uniquely suited for in vivo therapies with broad therapeutic applicability. It is developed by Professor Wensheng Wei’s lab at Peking University. Professor Wensheng Wei is the Scientific Founder of EdiGene.

"Earlier this year, we announced the first patient enrolled in multicenter Phase I clinical study of our investigational ex vivo gene-editing hematopoietic stem cell therapy ET-01. This collaboration furthers our efforts in RNA editing and in vivo gene-editing therapies," said Dong Wei, Ph.D., CEO of EdiGene. "Along with the expansion of our U.S. R&D Center, such effort will help accelerate the translation of our proprietary gene-editing technology for patients in need worldwide."

About the David Gamm Laboratory

The Gamm laboratory was established at the University of Wisconsin-Madison in 2003 to advance the use of human pluripotent stem cells (hPSCs) in the study and treatment of retinal degenerative diseases. To facilitate these efforts, the Gamm lab developed the first hPSC-based 3D retinal organoid culture method, which has since yielded key insights into mechanisms of early human retinal development. Moreover, their studies have established the authenticity of hPSC-derived retinal progeny, including photoreceptor cells (rods and cones), retinal pigmented epithelium (RPE) cells, and neural retinal tissue. Lastly, they were pioneers in the use of patient-specific and gene-modified iPSCs to model retinal disorders and to test therapeutic strategies, and have advanced efforts to adapt this technology for human use.