On June 17, 2021 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported it granted an exclusive option to Amasa Therapeutics, Inc. (Amasa), a privately-held biopharmaceutical company focused on the development of novel cell-based targeted biological therapeutics to treat cancer patients with unmet need, to acquire an exclusive, royalty-bearing license to use Lineage’s HyStem technology for the development and commercialization of therapies for local treatment of solid tumors under pre-negotiated terms (Press release, Lineage Cell Therapeutics, JUN 17, 2021, View Source [SID1234584170]). Under the option agreement, Amasa will purchase certain amounts of Lineage’s existing supply of clinical-grade HyStem biomaterial and has the right to purchase additional amounts in connection with its up to 12-month option to acquire the exclusive license. Lineage will receive an upfront cash payment and, if the option is exercised, would be entitled to additional payments, including event-specific payments, royalties on net sales and sublicense fees and royalties.

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"Lineage is a clinical-stage cell therapy company supported by a vast intellectual property portfolio. From this portfolio, we continue to find opportunities to unlock value from non-core assets through option and license agreements for assets such as HyStem"

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"Lineage is a clinical-stage cell therapy company supported by a vast intellectual property portfolio. From this portfolio, we continue to find opportunities to unlock value from non-core assets through option and license agreements for assets such as HyStem," stated Brian Culley, Lineage CEO. "Many tissue engineering and regenerative cell-based therapies will require the delivery of therapeutic cells in a matrix or scaffold for accurate anatomical placement, cell retention, and engraftment. This option agreement represents an opportunity to provide Amasa with access to our clinical-grade HyStem material for future development of oncology-related products delivered via HyStem and, alongside a previously announced deal with Advanced BioMatrix, is the second HyStem-related transaction we have entered into."

HyStem is a patented biomaterial that is made from and structurally mimics naturally occurring extracellular matrix, the structural network of molecules surrounding cells in organs and tissues that is essential to cellular function and tissue structure. The technology underlying the HyStem hydrogels is based on a unique thiol cross-linking strategy. Building upon this technology, the HyStem family of hydrogels are novel biomaterials that offer unique strategies for cell therapy and bioactive molecule delivery. A distinctive feature of the HyStem hydrogel is that it allows the mixture of cells with the matrix in a liquid form such that the cells and matrix can be injected easily through a small gauge syringe, and then the matrix can polymerize around the cells to create a three-dimensional tissue within the body. When implanted in HyStem hydrogels, cells remain attached and localized within the hydrogel and slowly degrade the implanted matrix and replace it with their natural extracellular matrices. Current research at leading medical institutions has shown that HyStem is compatible with a wide variety of cells and tissue types including brain, bone, skin, cartilage, vascular and heart tissues.

"We believe use of Lineage’s clinical-grade HyStem hydrogels will allow us to quickly move candidates into the clinic with our novel approach of using receptor-targeted cell therapies to address intractable solid tumors such as glioblastoma," stated Arthur Hiller, Amasa CEO. "The physiochemical properties of the HyStem hydrogel and its ability to provide a suitable extracellular matrix give our cell therapies the best opportunity to remain viable, be retained in the targeted tumor resection cavity, and eradicate residual tumor cells," stated Khalid Shah, founder of Amasa.

About HyStem

Lineage has developed a family of hyaluronan based hydrogels (HyStem) that mimics the natural extracellular matrix and has potential applications in 3-D cell culture, stem cell propagation and differentiation, tissue engineering, regenerative medicine, cell-based therapies, and as delivery vehicles for bioactive molecules. HyStem hydrogels were designed to recapitulate the minimal composition necessary to obtain a functional extracellular matrix (ECM). The individual components of the hydrogels are cross-linkable over time, and thus may be seeded with cells prior to in vivo injection, without compromising either the cells or the recipient tissues. HyStem hydrogels have been shown to support attachment and proliferation of a wide variety of cell types in both 2-D and 3-D cultures and provide timed release of proteins and other bioactive moieties. HyStem hydrogels exhibit a high degree of biocompatibility when implanted in vivo and are readily degraded in vitro and biodegrade in vivo through hydrolysis via naturally occurring enzymes. When implanted in HyStem hydrogels, cells remain attached and localized within the hydrogel and slowly degrade the implanted matrix and replace it with their natural ECMs. When used as a delivery vehicle, bioactive molecules are released by both diffusion as well as degradation of the hydrogel. The patented technology underlying Lineage’s HyStem hydrogel products in development, such as Renevia, has been exclusively licensed to Lineage for human therapeutic uses. Since the first published report in 2002, there have been over 300 academic scientific publications supporting the biocompatibility of thiol cross-linked hyaluronan-based hydrogels and their applications as medical devices and in cell culture, tissue engineering, and animal models of cell-based therapies. Due to the unique cross-linking chemistry, HyStem hydrogels have the ability to be formulated with cells and can be injected or applied as a liquid and form a gel in situ, which allows the hydrogel to conform to a cavity or space. This property of HyStem hydrogels is expected to offer several distinct advantages over other hydrogels, including the possibility of combining bioactive materials with the hydrogel at the point of use.

On June 17, 2021 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, reported the publication of data from the IMbark Phase 2 clinical trial in the Journal of Clinical Oncology in a paper entitled, "Randomized, Single-Blind, Multicenter Phase II Study of Two Doses of Imetelstat in Relapsed or Refractory Myelofibrosis (Press release, Geron, JUN 17, 2021, View Source [SID1234584165])." The publication highlights the clinical benefits observed in the study, including symptom response and overall survival, as well as the evidence of disease-modifying activity from biomarker and bone marrow fibrosis assessments.

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"We are pleased with the publication of our IMbark Phase 2 data in the high-impact Journal of Clinical Oncology. This highlights the importance of the study data in the advancement of treatment options for MF patients who no longer respond to currently approved JAKi therapies," said Aleksandra Rizo, M.D., Ph.D., Geron’s Chief Medical Officer. "Imetelstat is a novel telomerase inhibition approach that may alter the course of the disease in patients with myelofibrosis. We look forward to confirming these results in our ongoing IMpactMF Phase 3 clinical trial in refractory MF."

The publication reports efficacy, safety and biomarker results from the IMbark Phase 2 clinical trial and is available online. As stated in the paper, IMbark tested two imetelstat doses and the 9.4 mg/kg dose every three weeks demonstrated clinical benefits in symptom response rate, with an acceptable safety profile for this poor-risk JAKi relapsed/refractory MF patient population. Biomarker and bone marrow assessments suggested selective effects on the malignant clone.

"In these heavily pre-treated patients with high disease burden whose outcome is dismal, imetelstat treatment resulted in multiple clinical meaningful benefits, including symptom response and potential improvement in overall survival," said John Mascarenhas, M.D., Associate Professor of Medicine at the Icahn School of Medicine at Mount Sinai, and lead author of the paper. "In addition, the reductions in key driver mutations of the disease that were also correlated to clinical benefits suggest disease-modifying activity of imetelstat by targeting the underlying MF malignant clones, which differentiates imetelstat from other therapeutic agents currently in development for MF."

Ongoing IMpactMF Phase 3 Clinical Trial

IMpactMF is an open label, randomized, controlled Phase 3 clinical trial with registrational intent. The trial is planned to enroll approximately 320 patients with Intermediate-2 or High-risk myelofibrosis who are refractory to prior treatment with a JAK inhibitor, also referred to as refractory MF. Patients will be randomized to receive either imetelstat or best available therapy. The primary endpoint is overall survival (OS). Key secondary endpoints include symptom response, spleen response, progression free survival, complete response, partial response, clinical improvement, duration of response, safety, pharmacokinetics, and patient reported outcomes.

IMpactMF is currently enrolling patients. For further information about IMpactMF, including enrollment criteria, locations and current status, visit ClinicalTrials.gov/NCT04576156.

About Myelofibrosis (MF)

Myelofibrosis, a type of myeloproliferative neoplasm, is a chronic blood cancer in which abnormal or malignant precursor cells in the bone marrow proliferate rapidly, causing scar tissue, or fibrosis, to form. People with MF may have abnormally low or high numbers of circulating red blood cells, white blood cells or platelets, and abnormally high numbers of immature cells in the blood or bone marrow. MF patients can also suffer from debilitating constitutional symptoms, such as drenching night sweats, fatigue, severe itching, or pruritus, abdominal pain, fever and bone pain.

Approximately 70% of MF patients are classified as having Intermediate-2 or High-risk disease, as defined by the Dynamic International Prognostic Scoring System Plus. There are more than 35,000 patients worldwide and more than 13,000 patients in the U.S. living with Intermediate-2 or High-risk MF. The only drug therapies approved for treating these MF patients are JAK inhibitors (JAKi). Currently, MF patients who fail or no longer respond to JAKi treatment have no or limited options, resulting in shortened median overall survival.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Data from Phase 2 clinical trials provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in hematologic myeloid malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase inhibitor (JAKi) treatment.

On June 17, 2021 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company, reported in partnership with Seer, Inc. (NASDAQ: SEER), a life sciences company commercializing a disruptive new platform for proteomics, a webinar to present the newest trends in proteomics for biomarker discovery and translational research (Press release, Biodesix, JUN 17, 2021, View Source [SID1234584164]). The webinar entitled "A New Era in Precision Medicine – Uncovering the Depth & Breadth of the Plasma Proteome with Novel Proteomic Technologies" will be presented by Robert Georgantas, III, PhD, Senior Vice President, Research and Translational Science at Biodesix and Daniel Hornburg, PhD, Principal Scientist from Seer. The webinar is scheduled at 11:00 am ET, Tuesday June 22, 2021 and participants are encouraged to REGISTER HERE.

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Proteomics is the next frontier in biomarker and translational research with new emerging technologies that are enabling researchers to see deeper and broader into the plasma proteome than ever before. Biodesix is a recognized leader in profiling patients’ immune response to all cancer types using blood-based solutions that combine proteomic and genomic insights with a proprietary AI analytics platform.

The webinar will include an overview of Seer’s Proteograph Product Suite, which uses proprietary engineered nanoparticles to survey plasma proteins, allowing researchers to identify and quantitate proteins across the dynamic range of the proteome. Additionally, learnings will be shared on how the Seer Proteograph Product Suite will help define the biological underpinnings of tests created by Biodesix and how it is being integrated into the Biodesix mass spectrometry-based workflows for biopharmaceutical and academic research.

On June 17, 2021 KIYATEC, Inc. reported the publication of new peer-reviewed data that establishes clinically meaningful prediction of patient-specific responses to standard of care therapy, prior to treatment, in newly diagnosed glioblastoma (GBM) and other high-grade glioma (HGG) patients (Press release, KIYATEC, JUN 17, 2021, View Source [SID1234584163]). The results, the interim data analysis of the company’s 3D-PREDICT clinical study, were published June 16, 2021 in Neuro-Oncology Advances1, an open access clinical journal.

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A goal of the study, which continues to enroll, was for the test’s prospective, patient-specific response prediction to achieve statistical significance for predictive accuracy. The 3D-PREDICT study met this goal early, at its interim data analysis, an achievement that is uncommon for innovations in oncology. For clinicians and payors, the publication establishes the successful analytical validation and early clinical validation of KIYATEC’s 3D Predict Glioma assay.

The recent bipartisan resolution passed by the US Senate designating July 21, 2021 as Glioblastoma Awareness Day highlights the severity of this aggressive brain cancer. Fewer than 10% of patients survive longer than five years. Pharmaceutical and clinical efforts have only resulted in modest increases in overall survival since the disease was first described in the 1920s. Today, most newly diagnosed patients receive the same treatment regimen (radiation therapy and temozolomide), presenting an opportunity to improve care through shifting the paradigm toward individualized medicine for HGG treatment.

KIYATEC’s test results accurately identified the patients as future temozolomide responders or future non-responders prior to the initiation of drug treatment. The future responder group had a statistically significant 6-month comparative increase in overall survival. Since test results are available only seven days after surgery, this creates an opportunity to improve outcomes for each predicted non-responder by providing the possibility of patient-specific treatment strategies. In the future, KIYATEC’s results may also prove useful to improve outcomes for each predicted responder through patient-specific combination strategies.

Successful response-prediction for newly diagnosed patients follows the company’s previous success with predicting treatment response in recurrent high-grade glioma patients. In December 2020, KIYATEC announced a clinical case series demonstrating that use of their test doubled these patients’ median time to progression over what would be expected without use of the test. In addition, the earlier announcement demonstrated successful clinical use of the targeted agent dabrafenib in two patients that were not identified by genetic sequencing. By identifying successful response to drugs that would have been missed by today’s testing, KIYATEC’s results expanded the successful treatment options for these patients.

"Decision making in our framework is based on patient-specific evidence, embodying truly personalized medicine. Evidence of response before the first dose is administered creates options that were not previously available when it comes to treatment," said Matthew Gevaert, PhD, CEO of KIYATEC.

Versus other approaches, tests developed using KIYATEC’s 3D ex vivo cell culture platform demonstrate increased biological fidelity, which was first reported in 2019 in ovarian cancer. In newly diagnosed ovarian cancer patients, KIYATEC’s test prospectively and accurately predicted response to first-line chemotherapy with 89% accuracy. The new GBM results now establish comparable predictive accuracy in two solid tumors, with eight additional cancers in the company’s pipeline.

On June 17, 2021 Amneal Pharmaceuticals, Inc. (NYSE: AMRX) ("Amneal" or the "Company") reported the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for Bevacizumab, pursuant to Section 351(k) pathway of the Public Health Service Act, and with a standard review goal date in the second quarter of 2022 according to the BsUFA (Biosimilar User Fee Act) (Press release, Amneal Pharmaceuticals, JUN 17, 2021, View Source [SID1234584162]).

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The biosimilar was developed in collaboration with mAbxience, a Spain-based biotechnology company. Bevacizumab is the biosimilar version of Avastin and when approved will be marketed under the proprietary name AlymsysTM. Alymsys was approved by the European Medicines Agency (EMA) in February 2021. Amneal and mAbxience believe that the data supports the biosimilarity of its AlymsysTM product to Avastin.

"The FDA’s acceptance of our BLA for Bevacizumab is a significant milestone in our journey to become an important player in biosimilars," stated Chirag and Chintu Patel, Co-Chief Executive Officers. As previously announced, the Company expects its initial biosimilar portfolio will include Filgrastim (biosimilar for Neupogen), Pegfilgrastim (biosimilar for Neulasta) and Bevacizumab (biosimilar for Avastin).

Bevacizumab is a vascular endothelial growth factor inhibitor. Amneal is seeking approval for Bevacizumab for the treatment of metastatic colorectal cancer, in combination with intravenous fluorouracil-based chemotherapy for first- or second-line treatment and metastatic colorectal cancer, in combination with fluoropyrimidine-irinotecan- or fluoropyrimidine-oxaliplatin-based chemotherapy for second-line treatment in patients who have progressed on a first line Alymsys-containing regimen. Amneal also intends to seek approval for the remainder of the indications from the reference product label as soon as possible, subject to patent and regulatory exclusivities, by the biosimilarity pathway pursuant to Section 351(k).

According to IQVIA, a leading healthcare data and analytics provider, U.S. annual sales for Bevacizumab for the 12 months ended April 2021 were approximately $2.8 billion.