On June 3, 2021 Kintara Therapeutics, Inc. (Nasdaq: KTRA) ("Kintara" or the "Company"), a biopharmaceutical company focused on the development of new solid tumor cancer therapies, reported it has enrolled the final patient in the adjuvant arm of its ongoing Phase 2 clinical study of VAL-083 being conducted at the MD Anderson Cancer Center (MD Anderson) (Press release, Kintara Therapeutics, JUN 3, 2021, View Source [SID1234583453]). The adjuvant arm of the study investigates newly-diagnosed patients suffering from glioblastoma multiforme (GBM) receiving VAL-083 in place of standard of care temozolomide (TMZ) as adjuvant therapy following surgery and chemoradiation TMZ. The trial was designed to enroll up to 36 patients to determine whether treatment with VAL-083 improves overall survival.

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The Phase 2 trial is an open-label, two-arm, biomarker-driven study testing VAL-083 in GBM patients who have an unmethylated promoter of the methylguanine DNA-methyltransferase (MGMT) gene. Efficacy is being measured based on overall survival and progression-free survival. In February 2021, the Company announced the trial’s second arm (recurrent GBM) enrolled its final patient.

"Enrolling the final patient in the adjuvant arm of the Phase 2 study at MD Anderson is yet another important milestone for the Company as we continue to advance VAL-083 in multiple settings including the GCAR sponsored GBM AGILE study where we have already initiated patient recruitment at 15 sites as the only therapeutic agent currently being evaluated in this adaptive design registration study for all three GBM patient subtypes; newly-diagnosed methylated MGMT, newly-diagnosed unmethylated MGMT, and recurrent," commented Saiid Zarrabian, Kintara’s Chief Executive Officer.

VAL-083 is a small molecule bifunctional alkylating agent that crosses the blood-brain barrier. VAL-083 is independent of the MGMT resistance mechanism and has been assessed in over 40 Phase 1 and Phase 2 clinical trials in multiple indications sponsored by the U.S. National Cancer Institute (NCI). VAL-083 has been granted Orphan Drug Designation for GBM by the FDA and EMA and has also been granted Orphan Drug Designations for medulloblastoma and ovarian cancer by the FDA. In addition, the FDA has granted Fast Track Designation for VAL-083 in recurrent GBM. VAL-083 is approved as a cancer chemotherapeutic in China for the treatment of chronic myelogenous leukemia and lung cancer. VAL-083 has not been approved for any indications outside of China.

On June 3, 2021 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that three new strength tablets for XPOVIO, the Company’s first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) medicine, are now commercially available (Press release, Karyopharm, JUN 3, 2021, View Source [SID1234583452]). The availability of these additional strength tablets follows a U.S. Food & Drug Administration (FDA) approval on April 15, 2021. In addition to the original 20 mg strength tablets, XPOVIO is now available in 40 mg, 50 mg, and 60 mg strength tablets. XPOVIO tablets are available in seven different package sizes to help healthcare providers individualize the dosing and administration of XPOVIO based on patient needs. The additional dosage strength tablets may also increase patient compliance by simplifying their treatment regimen and reducing the pill burden experienced by some patients. With the introduction of these new tablet strengths and additional package sizes, there is no change to the efficacy and safety profile of XPOVIO.

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About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). XPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion. Karyopharm received accelerated U.S. Food and Drug Administration (FDA) approval of XPOVIO in July 2019 in combination with dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. NEXPOVIO (selinexor) has also been granted conditional marketing authorization for adult patients with heavily pretreated multiple myeloma by the European Commission. Karyopharm’s supplemental New Drug Application (sNDA) requesting an expansion of its indication to include the treatment for patients with multiple myeloma after at least one prior therapy was approved by the FDA on December 18, 2020. In June 2020, Karyopharm received accelerated FDA approval of XPOVIO for its second indication in adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple cancer indications, including as a potential backbone therapy in combination with approved myeloma therapies (STOMP) and in endometrial cancer (SIENDO), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with approved therapies in a variety of tumor types to further inform Karyopharm’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.

On June 3, 2021 Isofol reported that it will present an abstract at the annual ASCO (Free ASCO Whitepaper) meeting between June 4 – June 8 (Press release, Isofol Medical, JUN 3, 2021, View Source [SID1234583451]). Together with scientists from Sahlgrenska University Hospital and Precision for Medicine/QuartzBio (Geneva, Switzerland), we will present data about the regulation of 5-FU/folate-based treatment in colorectal cancer. MYC activation, a known transcriptional regulator of TYMS (the gene coding for TS), and one of the targets for the treatment, has been identified as a potentially relevant common upstream controller of a group of genes involved in 5-FU/folate efficacy. This relationship opens up for exploring further potential biomarker candidates as factors in 5-FU/Arfolitixorin efficacy and also combinations (including MYC-inhibitors).

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Information about the presentation

Title: MYC as a candidate upstream controller involved in TYMS gene expression and 5-FU/folate treatment efficacy in colorectal cancer

Authors: David A. Drubin, Anne-Katrin Hess, Natalie L. Catlett, Alessandro Di Cara, Yvonne Wettergren, Roger Tell

Abstract Number: e15512

Session title: Gastrointestinal Cancer—Colorectal and Anal

On June 3, 2021 Invitae Corporation (NYSE: NVTA), a leading medical genetics company, reported it has begun offering early access to its new Personalized Cancer Monitoring (PCM) platform as a laboratory-developed test performed at an Invitae central laboratory (Press release, Invitae, JUN 3, 2021, View Source [SID1234583450]). The service employs a novel combination of a tumor profile, blood tests and personalized assays based on a patient’s tumor with the goal of detecting circulating tumor DNA (ctDNA) before it is detectable by imaging or other conventional methods, offering earlier detection of cancer recurrence.

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

"Far too many patients who undergo treatment for cancer still relapse, and the cause is often the development of new cancer cell populations. One promising strategy for identifying recurrence early is to detect these cancer cells persisting in a patient after treatment, known as minimal residual disease (MRD), that cannot be detected with standard monitoring such as imaging," said Robert Nussbaum, M.D., chief medical officer of Invitae. "PCM has the potential to determine a therapy’s effectiveness much sooner than current monitoring methods, allowing clinicians to more efficiently refine and optimize treatment plans. In addition, patients whose cancer has been cured by tumor resection may be spared from unnecessary and potentially harmful adjuvant therapy, while those at risk of relapse can be diagnosed earlier and treated with the necessary therapies."

During the early access program, academic and pharmaceutical researchers will be able to utilize the pan-cancer, CAP-accredited and CLIA-certified PCM platform with testing and reporting completed by Invitae’s recently acquired laboratory in Iselin, New Jersey. Full commercial availability of PCM as a laboratory-developed test is expected later this year.

PCM is a pan-cancer, tumor-informed, liquid biopsy assay developed by Invitae to detect MRD and monitor for cancer recurrence. Clinical researchers may have the ability to have residual disease and/or cancer recurrence detected earlier than the current standard of care for most patients. By detecting residual disease/recurrence earlier, patients can be considered for therapy sooner, which may result in improved outcomes.

PCM includes three basic steps: 1. A patient’s surgically removed tumor or tumor biopsy and blood undergoes whole exome sequencing to create a patient specific tumor fingerprint. 2. Approximately 50 tumor-specific variants are selected for inclusion on a personalized ctDNA panel. 3. Patient-specific assays are created that can be used over time with minimally invasive blood draws to monitor for disease recurrence.

The platform is powered by Invitae’s Anchored Multiplex PCR (AMP) chemistry to perform error-corrected, next-generation sequencing. It is designed to identify traces of a patient’s original tumor DNA circulating in a patient’s blood, or ctDNA. Because ctDNA is a biomarker for MRD, AMP chemistry enables high sensitivity detection of MRD status. Detecting minute amounts of ctDNA while confidently determining MRD status may allow for earlier detection of relapse after treatment.

The high sensitivity and specificity of the PCM assay has been validated both in the laboratory and among NSCLC patients in the TRACERx study. Clinical validation studies will continue, as will the use of PCM to address research questions in support of meaningful clinical applications. Once more data become available in these important areas, PCM and other liquid biopsy approaches for monitoring MRD have the potential to become a mainstay in personalized oncology.

PCM could be applied in a variety of ways to help improve patient care and prolong survival outcomes. Its possible applications in the clinical setting include monitoring for recurrence, monitoring a patient’s response to therapy to guide treatment decisions, and improving clinical trial designs to help get new therapies to market sooner.

On June 3, 2021 -Imago BioSciences, Inc. ("Imago"), a clinical stage biopharmaceutical company discovering new medicines for the treatment of myeloproliferative neoplasms (MPNs), reported the appointment of Wan-Jen Hong, M.D., as chief medical officer (Press release, Imago BioSciences, JUN 3, 2021, View Source [SID1234583449]). Dr. Hong will be responsible for leading the clinical development of the Imago pipeline of therapies targeting LSD1 in various cancers and other bone marrow diseases. Dr. Hong joins Imago from Genentech, where she served as Group Medical Director in their late-stage clinical development group.

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"I am delighted to welcome Wan-Jen to our team, and we look forward to her leadership and guidance as we continue to advance our pipeline of therapies to treat diseases of the bone marrow," said Hugh Young Rienhoff, Jr., M.D., chief executive officer of Imago BioSciences. "Wan-Jen’s extensive background in hematology and oncology will be a strong asset for Imago as we complete our Phase 2 studies of bomedemstat for the treatment of myeloproliferative disorders, including essential thrombocythemia and myelofibrosis."

Dr. Hong is a physician scientist with more than 10 years of clinical and scientific expertise in hematology and oncology disorders. In her most recent role at Genentech, Dr. Hong worked on the development of several molecules across multiple indications in hematology and oncology, and played a leading role in the indication expansion of venetoclax (VENCLEXTA) in acute myeloid leukemia. Prior to Genentech, Dr. Hong was a clinical instructor in hematology at the Stanford University School of Medicine and is currently an adjunct clinical faculty member in its Division of Hematology. She earned a bachelor’s degree in chemical engineering and biology from Massachusetts Institute of Technology and completed both her medical doctorate and clinical training, including a hematology/oncology fellowship, at Stanford University.

"Imago has generated significant data on its lead asset bomedemstat in several indications," said Dr. Wan-Jen Hong, chief medical officer, Imago BioSciences. "Patients with myeloproliferative neoplasms and other hematological disorders continue to face significant challenges despite the current treatments. I am excited to work with the Imago team to develop bomedemstat as a new treatment option for patients with unmet medical needs."