On October 13, 2020 Rappta Therapeutics ("Rappta"), focused on developing first-in-class anti-cancer drugs activating protein phosphatase 2A (PP2A), reported the closing of a EUR 9 million Series A financing co-led by Novartis Venture Fund ("NVF") and Novo Holdings with participation from Advent Life Sciences and a family office (Press release, Rappta Therapeutics, OCT 13, 2020, View Source [SID1234568443]). The company also announces it has successfully received funding from Business Finland, the Finnish innovation funding organization.

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PP2A is a critical enzyme regulating protein de-phosphorylation and a key tumor suppressor which to date has been very difficult to target pharmaceutically. Rappta has developed proprietary tools and a unique understanding of PP2A which allows it to therapeutically reactivate PP2A. As a result of PP2A’s central role in the regulation of protein de-phosphorylation, Rappta’s PP2A-reactivating technologies offer the potential to develop multiple lead compounds and build a platform for a new class of anti-cancer drugs.

Rappta has assembled a strong scientific, management, and commercial team based in Finland and the US. Rappta’s scientific team, led by CSO and co-founder, Professor Goutham Narla, Division Chief of Genetic Medicine at the University of Michigan, represents world-leading expertise in PP2A. The scientific team has published seminal papers on the structural, functional, and biological mechanisms of PP2A inactivation in human cancer. The team will be supported by the Scientific Advisory Board led by Dr. William Hahn, a Professor of Medicine at the Harvard Medical School and the Chief Scientific Officer at the Dana-Farber Cancer Institute.

Goutham Narla, Rappta’s CSO, board member and co-founder, commented: "I am thrilled to be working on this opportunity to build a new platform and a novel class of pharmaceuticals to treat cancer. We have a unique team whose deep understanding of the PP2A biochemistry, structural biology, biogenesis, medicinal chemistry and drug development represent the perfect combination of expertise to translate these discoveries to the clinic."

Mikko Mannerkoski, Rappta’s CEO, board member and co-founder, commented: "We are very pleased to attract such a strong syndicate of international investors which validates our approach to developing novel therapies to target the previously undruggable target protein PP2A. This funding will enable us to accelerate the development of our platform and advance the lead compounds towards clinical development."

Beat Steffen from NVF, Jeroen Bakker from Novo Seeds, and Raj Parekh from Advent Life Sciences, will join Mikko Mannerkoski and Goutham Narla on the Board of Directors with Beat Steffen serving as the chairperson.

On October 13, 2020 Nuvation Bio, Inc., an oncology company focused on revolutionizing cancer treatment by discovering, developing and delivering next-generation therapies that target the greatest needs in oncology, reported that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for NUV-422, an investigational CDK2/4/6 inhibitor (Press release, Nuvation Bio, OCT 13, 2020, View Source [SID1234568439]). The company plans to initiate enrollment in a Phase 1/2 study of NUV-422 in patients with high-grade gliomas by the first quarter of 2021.

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"The FDA’s acceptance of the IND for NUV-422 is an important milestone for Nuvation Bio. We look forward to initiating the Phase 1/2 study, our first-in-human trial of a compound derived from our deep pipeline, which includes six novel and mechanistically distinct early-stage oncology programs, each targeting multiple difficult-to-treat cancers," said David Hung, M.D., founder and chief executive officer of Nuvation Bio. "We have advanced NUV-422 through preclinical studies that have informed a robust clinical development plan to treat recurrent high-grade gliomas, including glioblastoma multiforme, which are known to carry significant alterations of CDK function. Based on this data, we believe that NUV-422 has the potential to offer significant clinical benefit to patients with high-grade gliomas, a devastating cancer for which treatment options are very limited."

Nuvation Bio’s open-label, Phase 1/2 dose-escalation and multiple expansion cohort study is expected to enroll approximately 80 adult patients with recurrent or refractory high-grade gliomas at trial sites in the United States. The Phase 1 dose-escalation part of the study will evaluate the safety, tolerability and pharmacokinetics of oral doses of NUV-422. The company anticipates reporting top-line data from that part of the study in 2022.

About NUV-422
NUV-422 is a selective small molecule resulting from Nuvation Bio’s cyclin-dependent kinase (CDK) inhibitor program. CDK4/6 inhibitors are known clinical entities with proven efficacy, but cancer cells can evade these treatments by increasing signaling through CDK2. Inhibition of CDK2 in addition to CDK4/6 cuts off the tumor’s natural escape route. NUV-422 is a potent inhibitor of CDK 2, 4 and 6. Preclinical studies have shown that NUV-422 has favorable blood-brain barrier penetration.

About High-Grade Gliomas
Primary tumors of the central nervous system (CNS) remain among the most difficult to treat, with a 5-year overall survival of approximately 35%. Gliomas, which begin in the glial or supportive tissue, represent 75% of malignant primary brain tumors in adults. Glioblastoma multiforme (GBM) accounts for 50% to 70% of all gliomas. More than 10,000 people in the United States each year are diagnosed with this aggressive, difficult-to-treat brain tumor.i No treatment advances have been made in GBM since 2009 when bevacizumab was approved by the FDA. Temozolomide and radiation are considered the current standard of care for newly diagnosed patients with glioblastoma.ii

On October 13, 2020 The Pancreatic Cancer Action Network (PanCAN) reported the launch of Precision PromiseSM, the first of its kind adaptive clinical trial platform for pancreatic cancer patients (Press release, PanCAN, OCT 13, 2020, View Source [SID1234568438]). PanCAN’s Precision Promise was envisioned with a nationwide team of leading clinicians, researchers, and diagnostic and drug developers to test novel treatment options for pancreatic cancer patients quicker and cheaper and get them to patients faster, transforming the way clinical research is done.

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PanCAN’s Precision Promise platform was designed to be smarter, faster and cheaper than traditional clinical trials.
PanCAN’s Precision Promise clinical trial is designed to develop new pancreatic cancer treatments more efficiently.

Every pancreatic cancer treatment available today was approved through a clinical trial; however, standard trials are slow, costly, and have only had a 10 percent success rate over the last 20 years. PanCAN’s novel clinical trial platform is designed to enable the development of new treatments more efficiently than standard pancreatic cancer trials by testing multiple experimental therapies at the same time. Through Precision Promise, metastatic pancreatic cancer patients may have the opportunity to receive both first- and second-line treatment options in one clinical trial. As a late-stage platform, the potential for new drug approvals by the FDA is built into the model, which can accelerate the drug development process by up to two years. The statistical design of Precision Promise was led by world-renowned statistician Dr. Donald Berry (Berry Consultants), who designed the I-SPY breast cancer trials and has over 400 peer-reviewed publications.

Pancreatic cancer is currently the third leading cause of cancer-related death in the U.S., with an overall five-year survival rate of just 10 percent. Roughly 63 percent of patients die within the first year of a pancreatic cancer diagnosis, underscoring the urgent need for new and more effective treatment options.

"Pancreatic cancer patients don’t have time to wait for new treatments to be approved through the standard clinical trial model," said Julie Fleshman, JD, MBA, PanCAN’s president and CEO. "As the first pancreatic cancer nonprofit organization to develop, sponsor, and lead an adaptive clinical trial platform, we are disrupting the current clinical trial system in order to accelerate progress for patients fighting this disease."

Eligible pancreatic cancer patients will be able to enroll in PanCAN’s Precision Promise at one of 15 Clinical Trial Consortium sites nationwide. Sites were selected through a competitive, peer-review process and include:

Cedars-Sinai Medical Center (Los Angeles, Calif.)
Dana-Farber/Harvard Cancer Center (Boston, Mass.)
Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance/University of Washington (Seattle, Wash.)
Johns Hopkins Medicine (Baltimore, Md.)
Memorial Sloan Kettering Cancer Center (New York City, N.Y.)
Moores Cancer Center at UC San Diego Health (San Diego, Calif.)
Perelman School of Medicine University of Pennsylvania (Philadelphia, Pa.)
Perlmutter Cancer Center/NYU Langone Health (New York City, N.Y.)
The University of Chicago (Chicago, Ill.)
The University of Texas MD Anderson Cancer Center (Houston, Texas)
UC San Francisco Helen Diller Family Comprehensive Cancer Center (San Francisco, Calif.)
University of Florida Health – Cancer Center (Gainesville, Fla.)
Virginia Mason Medical Center (Seattle, Wash.)
Washington University School of Medicine (St. Louis, Mo.)
Weill Cornell Medicine (New York City, N.Y.)
A full list of institutions that are open and actively enrolling patients can be found at pancan.org/precisionpromise/locations.

In partnership with Tempus, a leader in artificial intelligence and precision medicine, every patient enrolled in PanCAN’s Precision Promise trial will undergo broad-panel genomic testing. Precision promise will also support follow-up biopsies to learn how their tumor is responding to treatment. Through the adaptive nature of PanCAN’s Precision Promise, data will be constantly monitored, and treatment arms can be discontinued if results do not look promising.

"By designing a more patient-centric trial platform, we are able to test promising new therapies more quickly and learn from fewer patients if a treatment is working," said Diane Simeone, MD, chair of the PanCAN Precision Promise Steering Committee and Precision Promise Principal Investigator at Perlmutter Cancer Center at NYU Langone Health. "The patients that enroll will also receive best-in-class supportive care alongside treatment with biomarker testing to better understand why treatments work in some patients but not in others. This approach should serve as a model for the next generation of trials – not only for pancreatic cancer, but for all diseases."

PanCAN is the trusted and unbiased leader that is bringing together key stakeholders – investigators, clinicians, industry, pancreatic cancer thought-leaders and regulatory authorities – to make Precision Promise possible.

"Through groundbreaking initiatives like Precision Promise, PanCAN is committing to taking bold action in the fight against pancreatic cancer," said Hilarie Koplow-McAdams, Venture Partner at New Enterprise Associates and chair of PanCAN’s Board of Directors, who lost her father to pancreatic cancer in 2012. "Anyone who has been personally touched by this disease understands the urgency needed to develop new treatment options. With PanCAN taking the initiative to drive innovation in this space, we can make meaningful progress."

PanCAN is grateful to its generous donors as well as its Scientific & Medical Affairs Industry Members who help make research initiatives like Precision Promise possible. Industry members include: AbbVie, AstraZeneca, IPSEN, Pfizer, Rafael Pharmaceuticals, Tempus, TriSalus Life Sciences, and Tyme Technologies, Inc.

To learn more about Precision Promise and PanCAN’s commitment to research, visit pancan.org. Pancreatic cancer patients and caregivers can receive personalized support and resources, and more information about Precision Promise, through PanCAN’s Patient Services or by calling 877-2-PANCAN.

For the latest organization updates, follow the PanCAN on Facebook, Twitter, and Instagram.

On October 13, 2020 Linnaeus Therapeutics, Inc. (Linnaeus), a privately held clinical-stage biopharmaceutical company focused on the development and commercialization of novel small molecule oncology therapeutics, reported that it has dosed the first patient in its phase 1/2 adaptive-design clinical trial of its lead-product-candidate, LNS8801, in combination with KEYTRUDA (pembrolizumab) in patients who had previous clinical benefit from immune checkpoint inhibitors and then subsequently progressed (Press release, Linnaeus Therapeutics, OCT 13, 2020, View Source [SID1234568436]). This marks the first time any company has dosed a patient in a clinical trial specifically targeting the G protein-coupled estrogen receptor (GPER) in combination with pembrolizumab. The initiation of the study follows the successful identification of the recommended phase 2 dose (RP2D) in the Company’s ongoing study of LNS8801.

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LNS8801 is an orally bioavailable small molecule that is a highly specific and potent agonist of the GPER. LNS8801 is currently finishing the dose escalation portion of its phase 1/2 study assessing the safety, tolerability, pharmacokinetics and preliminary efficacy in patients with advanced cancer.

"We are extremely pleased to begin testing LNS8801 in combination with pembrolizumab in this patient population. In our phase 1 trial of LNS8801 we have seen biological and clinical signals that suggest LNS8801 in combination with pembrolizumab may have activity based on the preclinical rationale and reverse resistance to immune checkpoint inhibitors. We currently have six outstanding academic comprehensive cancer centers all enthusiastically recruiting patients for this promising study," commented Patrick Mooney, MD, chief executive officer of Linnaeus. "We believe that LNS8801 with pembrolizumab has potential to provide meaningful clinical benefit for patients with cancer, and we look forward to providing updates over the course of the study."

The study entitled, "A Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, and Antitumor Activity of LNS8801 in Patients with Advanced Cancer Including Immunotherapy Refractory Expansion Cohorts with and without Pembrolizumab," was designed in two parts. The phase 1 dose-escalation portion of the trial assessed the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of LNS8801. Data from the study has allowed Linnaeus to determine the RP2D and safely open the phase 2 dose-expansion cohorts. Linnaeus anticipates opening several more phase 2 dose-expansion cohorts exploring LNS8801 activity as a monotherapy and in combination with other compounds in defined patient populations in the coming year.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

About LNS8801
LNS8801 is an orally bioavailable and highly specific agonist of GPER whose activity is dependent on the expression of GPER. GPER activation suppresses well-known tumor-associated genes, such as c-Myc and PD-L1. In preclinical cancer models, LNS8801 displays potent antitumor activities across a wide range of tumor types, rapidly shrinking tumors and inducing immune memory.

In the ongoing phase 1/2 study in humans, LNS8801 monotherapy has been safe and well-tolerated. Additionally, LNS8801 has demonstrated target engagement and clinical benefit in advanced cancer patients. Data from the phase 1/2 study is anticipated to be presented in a peer-reviewed setting in 2021.

On October 13, 2020 miR Scientific, LLC, a healthcare company whose purpose is to transform global cancer management by providing early and highly accurate detection, characterization and monitoring of disease, reported that it has received Breakthrough Device Designation by the U.S Food & Drug Administration for its miR Sentinel PCC4 Assay (miR Sentinel Prostate Test) (Press release, miR Scientific, OCT 13, 2020, View Source [SID1234568435]). FDA Breakthrough Device designation is granted to novel medical devices that have the potential to provide more effective treatment or diagnosis of life-threatening or irreversibly debilitating diseases or conditions.

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The Company’s miR Sentinel Prostate Test is a new method to analyze small non-coding RNAs (sncRNA) derived from a simple, non-invasive urine specimen from age-eligible men. Using only the expression level of these sncRNAs, a proprietary Statistical Classification Algorithm derives scores that classify patients according to the likelihood of being in the following four possible groups: no molecular evidence of prostate cancer; low-risk; intermediate-risk; or high-risk prostate cancer. Initial validation data for the miR Sentinel Prostate Test was published in the September 2020 issue of The Journal of Urology. The Company will initially commercialize its test as a Laboratory Developed Test in the US. Through the Breakthrough Device Program, miR Scientific will work more closely and frequently with the FDA to expedite its review of the miR Sentinel Prostate Test.

In comparison to currently available technologies alone, the specificity, sensitivity and empirical NPV and PPV of the miR Sentinel Prostate Test may significantly reduce mortality and decrease the number of hospitalizations, physician visits and reduce recovery time. The Company designed the miR Sentinel Prostate Test to revolutionize a patient’s disease journey when used by a provider in conjunction with other clinical findings and/or laboratory tests.

"We are highly appreciative that the FDA’s breakthrough designation process provided miR Scientific with invaluable feedback, which we have incorporated into our current clinical prospective studies to support our full marketing authorization plan" said Sam Salman, Chairman and CEO of miR Scientific. "Receiving this designation achieves another important milestone in the development of our novel and ground-breaking technology. We believe that the accuracy and non-invasive feature of our award-winning technology will impact the lives of millions of men and forever positively change the standard of care for urological cancers".