On March 2, 2023 Hepion Pharmaceuticals, Inc. (NASDAQ:HEPA), a clinical stage biopharmaceutical company focused on Artificial Intelligence ("AI")-driven therapeutic drug development for the treatment of non-alcoholic steatohepatitis ("NASH"), fibrotic diseases, hepatocellular carcinoma ("HCC"), and other chronic diseases, reported that the European Patent Office ("EPO") has granted European Patent No. EP 3886813, covering the innovative formulation of Hepion’s lead cyclophilin inhibitor, rencofilstat (Press release, Hepion Pharmaceuticals, MAR 2, 2023, View Source [SID1234628081]).

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The newly granted European patent also significantly extends the time period of Hepion’s patent exclusivity for rencofilstat. The Company’s original composition-of-matter patent estate, comprising 52 patents issued in every major market throughout the world, are expected to provide exclusivity to 2036. The newly granted rights, which will encompass 38 European countries, are expected to extend the drug candidate’s patent life by approximately eight years, to 2044. Additional filings are also underway to potentially extend composition-of-matter and method-of-manufacturing exclusivity until 2048. In addition to the indications covered in the original composition-of-matter patents (viral, cardiovascular, neurological, and inflammatory diseases), Hepion has also filed various method-of-use patent applications covering a variety of additional indications (fibrosis, cancer, and thrombosis).

Robert Foster, PharmD, PhD, Hepion’s Chief Executive Officer, stated, "Rencofilstat is very challenging to formulate, owing to the physical-chemical properties of the molecule. Nevertheless, as awareness of its potential to address a number of significant unmet medical needs across a variety of indications continues to grow amongst researchers and drug developers in global markets, it is important this opportunity is well protected. To that end, our expanding intellectual property estate, including this new European patent, should ensure that rencofilstat has long-term market exclusivity."

On March 2, 2023 Porton Advanced Solutions Ltd. (Porton Advanced) and DanausGT Biotechnology Co., Ltd. (DanausGT) reported on February 27, 2023, a strategic collaboration in gene and cell therapy pipelines to expedite the development of innovative therapeutics (Press release, Porton Advanced Solutions, MAR 2, 2023, View Source [SID1234628105]).

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As an end-to-end gene and cell therapy service provider, Porton Advanced CDMO offers comprehensive solutions covering plasmids, cell therapy, gene therapy, oncolytic virus, mRNA therapy, and bacterial therapy. On the other hand, DanausGT focuses on researching, developing, and producing new anti-cancer drugs and rare diseases with gene editing technology. DanausGT has established multiple R&D pipelines, and Porton Advanced will offer gene and cell therapy CDMO solutions to DanausGT, including plasmid, virus, and cell therapy products.

"We are excited to partner with DanausGT as their proprietary CRISPR/AAV technology is widely recognized. Their seven research pipelines are highly competitive, covering all the stages from pre-clinical to Clinical Phase I. Through our end-to-end gene and cell therapy CDMO, we hope to help DanausGT achieve IND approval for multiple pipelines and advance the development pipeline from domestic and overseas into the clinical stage. This collaboration will accelerate the development of innovative CGT therapeutics," said Dr. Wang Yangzhou, CEO of Porton Advanced.

"We are pleased to establish strategic cooperation with Porton Advanced. I believe this partnership will expedite the development of innovative gene cell therapy. We are looking forward to collaborating in more fields," said Dr. Wang Haifeng, founder, and CEO of DanausGT.

During the signing ceremony, Professor Jose Carlos Segovia, co-founder and CSO of DanausGT, presented a lecture entitled "Ex Vivo Gene Editing as a Definitive Cure for Hemolytic Anemias" and introduced the latest research progress of DanausGT. "We make gene editing technology and cell therapy to treat various diseases and work on therapy development for patient-centered care and clinical value. We look forward to collaborating with Porton Advanced to bring innovative biopharmaceuticals to patients earlier and deliver innovative values to society and the industry," Professor Segovia said.

On March 2, 2023 Pediatric reported that solid tumors make up approximately 40% of all childhood cancers (Press release, Children’s Hospital Los Angeles, MAR 2, 2023, View Source [SID1234628104]). While pediatric cancer is rare, children can develop a wide range of tumor types, located in different parts of the body, which can make the differential diagnosis challenging. Investigators at Children’s Hospital Los Angeles have developed a liquid biopsy for solid tumors that has the potential to aid in reaching a specific diagnosis when surgery or a tissue biopsy is not feasible. The study findings were published on February 23 in the journal npj Precision Oncology.

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"This is one of the first clinically validated liquid biopsy tests to be launched at a pediatric academic medical center," says Jaclyn Biegel, PhD, Chief of Genomic Medicine and Director of the Center for Personalized Medicine at CHLA.

"We created a test that may be helpful in making a diagnosis, determining prognosis, and potentially identifying an effective therapy for children with solid tumors," says Fariba Navid, MD, Medical Director of Clinical Research in the Cancer and Blood Disease Institute at CHLA. Dr. Navid and Dr. Biegel are co-senior authors of this study.

A specific test for pediatric tumors is required because the genetics of tumors that affect adults differ from those in children. Adult tumors tend to be caused by mutations—sequence-based changes in a gene—so most liquid biopsy tests have been developed specifically to identify these mutations. However, pediatric tumors arising from mutations are less common. In children, copy number changes—losing or having extra copies of one or more genes—or rearrangements of genes that result in gene fusions, are more characteristic. For their research study, the CHLA team combined a technique known as Low-Pass Whole Genome Sequencing (LP-WGS) with targeted sequencing of cell-free DNA from plasma to detect copy number changes, as well as mutations and gene fusions, that are characteristic of pediatric solid tumors. An important feature of the study was that it required a much smaller volume of sample than is required for liquid biopsy studies in adults. Since an infant or young child has a smaller blood volume, the assays needed to be scaled down to accommodate this difference.

To create the test, the researchers collaborated with clinical teams and research investigators at CHLA including Jesse Berry, MD, Director of Ocular Oncology and CHLA’s Retinoblastoma Program, as well as investigators involved in Oncology, Neurosurgery and Pathology and Laboratory Medicine. Leo Mascarenhas, MD, MS, Deputy Director of the Cancer and Blood Disease Institute at CHLA was also involved in the design and support of the project.

The first version of the test, launched in Nov. 2022, evaluates chromosomal copy number changes in blood samples, cerebrospinal fluid and the aqueous humor of the eye to aid in the clinical diagnosis for patients with solid tumors, brain tumors and retinoblastoma, respectively.

The next version of the clinical assay, available in about six months, will include detection of mutations and gene fusions.

The liquid biopsy-based genetic tests join the CHLA-developed OncoKids cancer panel, a next-generation sequencing-based assay designed to detect changes in DNA or RNA that are associated with pediatric leukemias, brain and solid tumors; the CHLA Cancer Predisposition Panel; RNAseq for cancer, a transcriptome-based assay using RNA sequencing; VMD4Kids, a panel for vascular and mosaic disorders; as well as methylation array-based profiling for pediatric brain tumors.

Eirini Christodoulou, PhD, and Venkata Yellapantula, PhD, both at CHLA, are co-lead authors on the study. Additional authors, all at CHLA, include: Jennifer Cotter MD, Xiaowu Gai PhD, Dejerianne Ostrow, PhD, and Moiz Bootwalla, MS, of the Department of Pathology and Laboratory Medicine; Katrina O’Halloran MD, James Amatruda MD, PhD, Anya Zdanowicz of the Cancer and Blood Disease Institute; and Liya Xu, PhD, of The Vision Center.

On March 2, 2023 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA testing, reported the Company’s first commercial coverage policies for its molecular residual disease test, Signatera, including its first pan-cancer coverage policy for adjuvant, recurrence monitoring, and treatment monitoring (Press release, Natera, MAR 2, 2023, View Source [SID1234628103]).

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Effective March 1, 2023, Blue Shield of California now provides commercial coverage of Signatera for plan members diagnosed with any solid tumors. Specifically, the policy describes tumor-informed ctDNA testing with Signatera as medically necessary for patients with stage I-IV cancer to provide information for (1) adjuvant or targeted therapy; and/or (2) monitoring for relapse or progression, including but not limited to the use of immunotherapy.

In addition, effective January 1, 2023, Blue Cross and Blue Shield of Louisiana is providing coverage of serial testing with Signatera for plan members diagnosed with colorectal and muscle invasive bladder cancer and for pan-cancer immunotherapy monitoring.

"Following the recent breast cancer coverage decision by Medicare, achieving our first commercial coverage policies for Signatera – including one that encompasses pan-cancer coverage – is another major milestone for Natera and the patients who will now have enhanced access to tumor-informed ctDNA testing," said John Fesko, chief business officer. "These developments underscore the medical necessity of Signatera to inform critical treatment decisions and detect recurrence earlier."

Data supporting the clinical validity and utility of Signatera has been published in approximately 40 peer-reviewed publications, including validation across multiple cancer types to detect recurrence earlier than standard diagnostic tools1,2 and to improve the assessment of treatment response in conjunction with imaging.3

About Signatera

Signatera is a custom-built circulating tumor DNA (ctDNA) test for treatment monitoring and molecular residual disease (MRD) assessment in patients previously diagnosed with cancer. The test is available for both clinical and research use, and has been granted three Breakthrough Device Designations by the FDA for multiple cancer types and indications. The Signatera test is personalized and tumor-informed, providing each individual with a customized blood test tailored to fit the unique signature of clonal mutations found in that individual’s tumor. Signatera is intended to detect and quantify cancer left in the body, at levels down to a single tumor molecule in a tube of blood, to identify recurrence earlier and to help optimize treatment decisions.

On March 2, 2023 Prostate Cancer Foundation reported that Immunotherapies have successfully treated many types of cancer with the exception of advanced prostate cancer that has become resistant to treatment, known as metastatic castration-resistant prostate cancer (mCRPC) (Press release, Prostate Cancer Foundation, MAR 2, 2023, View Source [SID1234628102]). New findings from a Prostate Cancer Foundation (PCF)-funded investigator reveal the mechanism by which prostate cancer cells reprogram the immune system to promote rather than suppress cancer, as well as a promising new combination therapy that could prevent them from developing this ability.

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The research led by 2016 Izzy Englander – PCF Challenge Award recipient Akash Patnaik, MD, PhD, MMSc, and his team at the University of Chicago was recently published in the peer-reviewed journal Clinical Cancer Research.

"These findings represent an exciting new opportunity to prevent prostate cancer cells from evading the immune system and a promising combination therapy for metastatic castration-resistant prostate cancer," said Howard R. Soule, PhD, Executive Vice President and Chief Science Officer of the Prostate Cancer Foundation. "PCF commends Dr. Patnaik and the research team on their achievement and proudly supports their work to bring us closer to our mission to eliminate death and suffering from prostate cancer."

Researchers found that by recruiting abnormal tumor-associated macrophages (TAM) that express PD-1 into the tumor microenvironment, the immune system promotes the growth of prostate cancer.

Androgen deprivation therapy (ADT) and chemotherapy are first-line treatment options for metastatic prostate cancer. However, patients often relapse and the disease advances. Nearly 35,000 men in the U.S. are projected to die of prostate cancer in 2023.

Approximately 50% to 75% of mCRPC patients have a mutation in the tumor suppressor gene PTEN which drives the PI3K tumor growth and survival pathway; in patients with PTEN-mutated tumors, the normal anti-tumor immune response is often suppressed. Alterations of the PTEN/PI3K pathway are known drivers of advanced prostate cancer. Clinical trials of single drugs to target this pathway and combinations of PI3K inhibitors and ADT have shown limited benefit in patients with mCRPC.

Patnaik and his team performed a series of experiments using PTEN-mutated mouse models. They found that following treatment with a combination of ADT and copanlisib, a PI3K inhibitor, PD-1-expressing immunosuppressive TAM blocked the anti-cancer immunity mediated by macrophages; the cancer cells forced the macrophages into tumor-promoting mode by binding to the protein PD-1 on their surface. The researchers hypothesized that adding a third drug to block PD-1 would unleash the ability of the macrophages to kill the cancer. The triple combination of PD-1 checkpoint immunotherapy plus ADT hormone therapy plus a PI3K inhibitor yielded a response rate 60% greater among treated mice than among untreated control mice. These data suggest that the three-drug approach is needed to overcome treatment resistance in PTEN-mutated prostate cancer.

As part of the PCF Challenge Award, a phase 1b clinical trial will be conducted to test the safety and efficacy of copanlisib in combination with an anti-PD-1 drug in patients with PTEN-deficient mCRPC. If successful, the research could result in a new precision immunotherapy regimen for these patients.