On September 3, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs ("Regulus"), reported that Jay Hagan, President and Chief Executive Officer of Regulus, will present at the Wells Fargo Virtual Healthcare Conference on Wednesday, September 9, 2020 at 10:40 AM EDT (Press release, Regulus, SEP 3, 2020, View Source [SID1234564440]).

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A live webcast of the presentation will be available through the investor relations section of the Company’s website at www.regulusrx.com.

On September 3, 2020 Oramed Pharmaceuticals Inc. (Nasdaq: ORMP) (TASE: ORMP) (www.oramed.com), a clinical-stage pharmaceutical company focused on the development of oral drug delivery systems, reported that CEO Nadav Kidron will present a company overview at the H.C. Wainwright 22nd Annual Global Investment Conference, held virtually this year, September 14-16, 2020 (Press release, Oramed Pharmaceuticals, SEP 3, 2020, https://www.prnewswire.com/news-releases/oramed-to-present-at-hc-wainwright-global-investment-conference-301123771.html [SID1234564439]).

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Presentation Details:
Presenter: Nadav Kidron, CEO
Date: Tuesday, September 15, 2020
Time: 1:30 PM Eastern
Online

On September 3, 2020 The Leukemia & Lymphoma Society (LLS) and PRA Health Sciences (NASDAQ: PRAH) reported a partnership to launch a first-of-its-kind global master clinical trial to develop new treatments for children with relapsed acute leukemia (Press release, The Leukemia & Lymphoma Society, SEP 3, 2020, View Source;lymphoma-society-on-global-trial-for-children-with-acute-leukemia-301123949.html [SID1234564438]).

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The new LLS PedAL (Pediatric Acute Leukemia) master clinical trial will test, simultaneously, multiple targeted therapies for children who experience a relapse of their acute leukemia, (approximately 40% of children with acute myeloid leukemia (AML) and 20% of children with high-risk acute lymphoblastic leukemia (ALL) will, unfortunately, relapse). The study will take place at more than 200 sites worldwide that are part of the NCI-supported Children’s Oncology Group (COG) network of children’s hospitals, including those in the U.S., Australia, New Zealand, and Canada. The LLS PedAL team will also collaborate with other overseas partners in the UK and EU to implement the trial in those regions. Through these established clinical trial infrastructures, nearly every child in these regions who experiences a relapse of acute leukemia will have access to the trial.

The LLS PedAL trial will launch with several novel therapies to treat children with relapsed acute leukemia, with plans to add additional treatments as they become available. The study will ensure that every child with relapsed AML and many with ALL will be screened to identify their disease subtype and matched to the most appropriate targeted treatment. Some of these patients will participate in LLS PedAL substudies, while others will be referred to other treatments, including other open clinical trials, based on discussions with their physicians.

PRA’s Role

PRA will serve as the contract research organization for LLS PedAL, managing many aspects of the trial, from coordinating with all of the study sites, ensuring that the study protocols are executed properly, to monitoring the data quality and safety reporting. PRA will also provide guidance for submissions to the U.S. Food Drug Administration and international regulatory agencies. PRA will implement state of the art technology solutions that will allow the trial to be digitally-enabled to best fit the patients’ lifestyles, minimize the burden of participation, and keep the trial current throughout the lifecycle.

"PRA is proud to be the partner of LLS and its strong network of sites for the LLS PedAL trial," said Dr. Mark Sorrentino, Vice President, Center for Pediatric Clinical Development at PRA Health Sciences. "Patients and their parents are our focus, and they depend on clinical research as a care option, especially in the rarest of cases. We can leverage our strong pediatric and hematology/oncology expertise globally to help accelerate treatments to these kids and assist their families during a critical time."

Accelerating Drug Development for Children with Cancer

In 2017, the U.S. Congress passed The Research to Accelerate Cures and Equity (RACE) for Children Act into law, which went into effect on August 18, 2020.

The law requires pharmaceutical and biotechnology companies testing new molecularly targeted compounds for adult cancers to also evaluate them for children’s cancers. Put simply, the new law now will make it standard practice for drug companies to develop their targeted drugs for children with cancer.

"For too long, too many impediments have kept companies from efficiently testing drugs for children with cancer. Passing the RACE Act was a significant step toward opening the door to precision medicines that could make all the difference for kids," said Gwen Nichols, M.D., Chief Medical Officer of The Leukemia & Lymphoma Society. "The LLS PedAL trial now will bring the RACE Act into actual practice as the companies will be testing their novel agents in children with acute leukemia in the trial."

The PedAL trial, which is on target to open by summer 2021, is just one of the focus points in the LLS Children’s Initiative, a multi-year endeavor to help children with cancer through pediatric research grants, educational initiatives, patient and family support services, and advocacy.

With a rich history of breaking down siloes and bringing together leaders to fight blood cancers, LLS built LLS PedAL on the success of their Beat AML master trial for adult patients with AML. LLS and PRA have teamed up with the Children’s Oncology Group and the National Cancer Institute to bring the master trial and sub-trials to more than 200 sites across North America, with plans to expand the trial into Europe and Asia.

"LLS PedAL is an ambitious and complex initiative and we are pleased to have PRA Health Sciences as a partner to help bring this urgently needed master clinical trial for children to fruition," Nichols said.

On September 3, 2020 Natera, Inc. (NASDAQ: NTRA), a pioneer and global leader in cell-free DNA testing, reported that the CMS Molecular Diagnostics Program (MOLDX) has finalized a local coverage determination (LCD) to provide Medicare benefits for serial use of the Signatera molecular residual disease (MRD) test in patients with stage II or III colorectal cancer (CRC) (Press release, Natera, SEP 3, 2020, View Source [SID1234564437]). The final LCD is posted here and the billing and coding article here on the Centers for Medicare and Medicaid Services website.

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The final LCD is consistent with Medicare’s draft posted in August of 2019, covering two intended uses: (1) Patient risk stratification after surgical resection, to inform adjuvant treatment decisions, and (2) Recurrence monitoring with the same frequency as CEA, in patients with a previous cancer diagnosis but no ongoing evidence of disease. The LCD cites several published studies in its summary of evidence including one study showing Signatera’s ability to detect CRC recurrence up to 16.5 months earlier than imaging and CEA (average 8.7 months earlier).1

"Colorectal cancer remains one of the deadliest forms of cancer, but it’s also relatively unique as a disease where early detection of relapse is known to improve outcomes," said Scott Kopetz, MD, PhD, oncologist and professor, Department of Gastrointestinal (GI) Medical Oncology, The University of Texas MD Anderson Cancer Center, and Principal Investigator of Natera’s BESPOKE CRC study. "There is ample evidence suggesting that circulating tumor DNA will enable earlier relapse detection and improved decision-making in the adjuvant setting, and I look forward to seeing it used more in clinical practice."

Signatera has been validated across multiple cancer types to detect residual disease up to 2 years earlier than standard diagnostic tools, with virtually no false positives on a per sample level,1-4 and to help assess treatment response in conjunction with imaging.5 While a negative test result does not mean someone is definitely cancer-free, it does mean the risks of relapse or disease progression are significantly reduced.

"Medicare coverage marks a significant milestone in our mission to transform cancer care," said Solomon Moshkevich, General Manager of Natera’s oncology business. "This is the first covered indication for Signatera, with more in process as we continue to publish peer-reviewed studies supporting the validity of personalized ctDNA testing for treatment response monitoring and MRD assessment in multiple solid cancers."

On September 3, 2020 Alphamab Oncology (stock code: 9966 HK) reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to KN046, a recombinant humanized PD-L1/CTLA-4 bispecific antibody developed by Jiangsu Alphamab Biopharmaceuticals Co., Ltd. ("Jiangsu Alphamab"), a wholly-owned subsidiary of the company, for the treatment of thymic epithelial tumors (TET) (Press release, Alphamab, SEP 3, 2020, View Source [SID1234564436]). This is the second ODD awarded to Alphamab Oncology by FDA after the first ODD awarded to the subcutaneous PD-L1 antibody KN035 by FDA for the treatment of Biliary track cancer early this year.

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The Orphan Drug Designation from US FDA originates from the Orphan Drug Act which was enacted to encourage the development of innovative drugs to treat orphan diseases with target patient population less than 200,000 in the US. Drug candidates with Orphan Drug Designation qualify for seven-year market Orphan Drug Exclusivity (ODE). In addition, US FDA also rewards ODD drugs with comprehensive incentives including tax credit of 50% clinical trial cost, waiver of BLA user fee, subsidies for R&D costs, protocol assistance and expedited regulatory approval pathway. In 2019, 21 (44%) out of the 48 new drugs approved by US FDA are Orphan Drugs; and 8 out of the top 10 best-selling cancer drugs in US, have received Orphan Drug Designation for certain indications.

Thymic epithelial tumors (TET) is a group of thoracic tumors, predominantly thymomas and thymic carcinoma. The estimated number of patients with TET is about 7,000 to 10,000 cases in the United States in 2020. TET is inoperable and the metastatic TET has a very poor prognosis. There is currently no approved standard treatment for patients who have failed platinum-based chemotherapy; objective response rate of late-line chemotherapy or targeted therapy is less than 20%, and the median survival time is less than 12 months. There is an urgent need for effective drugs to improve the efficacy for patients. Research have demonstrated that thymus is an important organ for the development of T cells. Thymic epithelial tumor cells highly express PD-L1, so it is possible to benefit from immune checkpoint inhibitor therapy. The Australian Phase I clinical trial of KN046 has shown a high response rate, long-lasting response time and good safety in patients with thymic epithelial tumors. The phase II registration clinical trial to treat thymic carcinoma started recently . It is planned to be carried out in more than 10 research centers in China and the United States.

About KN046

KN046 is the world’s first recombinant humanized PD-L1/CTLA-4 bispecific invented by Alphamab. Its innovative designs include: a proprietary CTLA-4 domain antibody with a significantly improved safety profile; a bispecific antibody fused with PD-L1 antibody; engineered to target the tumor microenvironment with high PD-L1 expression, and Treg clearing function. The preclinical and clinical trial results of KN046 have shown promising efficacy and significantly reduced toxicity to human peripheral system, with the potential to become a breakthrough immuno-oncology therapy.

There are about 20 clinical trials of KN046 in different stages covering more than 10 types of tumors including NSCLC, TNBC, ESCC and pancreatic cancer in Australia and China. The results of these clinical trials have shown a preliminary profile of good safety and promising efficacy. US FDA has approved KN046 to enter phase II trial based on the clinical results in China and Australia. The phase III clinical trials to evaluate efficacy and safety of combination therapy of KN046 and platinum-based chemotherapy in patients with stage IV squamous non-small cell lung cancer have started in China.