On September 3, 2020 Phosplatin Therapeutics, a clinical stage pharmaceutical company focused on oncology therapeutics, reported that new clinical data from a dose escalation study (NCT 03409458) of lead candidate PT-112, an immunogenic cell death inducer, used in combination with PD-L1 checkpoint inhibitor avelumab, will be presented at the upcoming European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020 taking place September 19-21 (Press release, Phosplatin, SEP 3, 2020, View Source [SID1234564442]). Avelumab is co-developed and co-commercialized by Merck KGaA, Darmstadt, Germany and Pfizer Inc. This abstract is one of only seven abstracts that have been selected for the Mini Oral presentation format in the Investigational Immunotherapy category.

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Title: "Phase 1b dose escalation study of novel immunogenic cell death (ICD) inducer PT-112 plus PD-L1 inhibitor avelumab in solid tumors"

Presenter: Daniel D. Karp, MD, Professor, Division of Cancer Medicine, Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center

Session Date / Time: Released 9am CET September 18, 2020

Availability: On demand streaming for the duration of the conference

Abstract Number: 1026MO

Abstracts selected for the on-demand Mini Oral format will be made publicly available at 12:05am CET on Friday September 18, 2020. The Mini Oral presentation will be available on the ESMO (Free ESMO Whitepaper) website (registration required) from 9am CET on September 18, 2020.

"The data to be presented at the ESMO (Free ESMO Whitepaper) Virtual Congress 2020 further validate our development hypothesis with PT-112, and the demonstration of its feasibility in combination with immune checkpoint inhibition," said Robert Fallon, co-founder and chief executive officer, Phosplatin Therapeutics. "There is a large, unmet need for patients with advanced cancer not responding to immunotherapy, who essentially have no standard of care. The combination of PT-112 with avelumab has a strong underlying combination rationale based upon PT-112’s immunogenic cell death properties, and offers potential for advancing the treatment landscape."

The study was conducted as part of a collaboration agreement between Phosplatin Therapeutics, Pfizer, Inc. and Merck KGaA, Darmstadt, Germany (EMD Serono in the US and Canada). Under the terms of the collaboration, Phosplatin Therapeutics is the Sponsor of Phase 1b/2a clinical trials in several indications. Pfizer and Merck KGaA supply avelumab for the trials.

About PT-112

PT-112 is the first small molecule conjugate of pyrophosphate developed in oncology therapeutics. PT-112 promotes immunogenic cell death (ICD), or the release of damage associated molecular patterns (DAMPs), that lead to downstream immune effector cell recruitment in the tumor microenvironment. PT-112 represents a potential best-in-class small molecule inducer of this immunological form of cancer cell death and is under Phase II development. The first in-human study of PT-112 demonstrated an attractive safety profile and evidence of long-lasting responses among heavily pre-treated patients and won "Best Poster" at the ESMO (Free ESMO Whitepaper) 2018 Annual Congress within the Developmental Therapeutics category. The novelty of its pyrophosphate moiety also results in osteotropism, or the propensity of the drug to reach the mineralized bone. This property is of interest in cancer types that originate in bone or frequently lead to metastatic bone involvement, such as metastatic castrate-resistant prostate cancer (mCRPC). The first human clinical results in mCRPC were presented at the 2020 Genitourinary Cancers Symposium.

Avelumab Approved Indications

Avelumab (BAVENCIO) is indicated in the US for the maintenance treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) that has not progressed with first-line platinum-containing chemotherapy. BAVENCIO is also indicated for the treatment of patients with locally advanced or metastatic UC who have disease progression during or following platinum-containing chemotherapy, or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

Avelumab in combination with axitinib is approved in the US for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

In the US, the FDA granted accelerated approval for BAVENCIO for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.

Avelumab Important Safety Information from the US FDA-Approved Label

The warnings and precautions for avelumab (BAVENCIO) include immune-mediated adverse reactions (such as pneumonitis and hepatitis [including fatal cases], colitis, endocrinopathies, nephritis, and other immune-mediated adverse reactions as a single agent or in combination with axitinib [which can be severe and have included fatal cases]), infusion-related reactions, hepatotoxicity in combination with axitinib, major adverse cardiovascular events (MACE) in combination with axitinib [which can be severe and have included fatal cases], and embryo-fetal toxicity.

Common adverse reactions (reported in at least 20% of patients) in patients treated with BAVENCIO monotherapy include fatigue, musculoskeletal pain, diarrhea, nausea, infusion-related reaction peripheral edema, decreased appetite, urinary tract infection and rash. Common adverse reactions (reported in at least 20% of patients) in patients receiving BAVENCIO in combination with axitinib include diarrhea, fatigue, hypertension, musculoskeletal pain, nausea, mucositis, palmar-plantar erythrodysesthesia, dysphonia, decreased appetite, hypothyroidism, rash, hepatotoxicity, cough, dyspnea, abdominal pain and headache. Grade 3-4 hematology laboratory value abnormalities reported in at least 10% of patients with Merkel cell carcinoma treated with BAVENCIO monotherapy include lymphopenia; in patients receiving BAVENCIO in combination with axitinib, grade 3-4 clinical chemistry abnormalities include blood triglyceride increased and lipase increased.

On September 3, 2020 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the central nervous system, reported an update on progress for the U.S. manufacturing of Berubicin, the Company’s lead drug candidate, in preparation for upcoming clinical trials (Press release, CNS Pharmaceuticals, SEP 3, 2020, View Source [SID1234564441]).

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As previously announced, the Company implemented a dual-track drug product manufacturing strategy and engaged U.S.-based Pharmaceutics International, Inc. ("Pii") and Italy-based BSP Pharmaceuticals S.p.A. ("BSP") for the production of Berubicin. By engaging two separate manufacturers on two separate continents, CNS expects to mitigate COVID-19-related delay risks, diversify its supply chain and provide for localized availability of Berubicin.

Under this dual-track strategy, the Company has achieved several key milestones in its manufacturing efforts and is providing an update on the progress made with its U.S. manufacturer, Pii. First, CNS completed synthesis of Berubicin active pharmaceutical ingredient (API) and shipped API to both manufacturers to prepare an injectable form of Berubicin for clinical use. In preparation for production, CNS and Pii have now agreed on the manufacturing procedure and packaging components for Berubicin and selected a sterile filter manufacturer. The Company has also completed and reviewed a draft of the batch record. Importantly, the Company and Pii completed a successful laboratory simulation of the lyophilization cycle. The Company expects to begin manufacturing of Berubicin at Pii during the third quarter of this year.

"As we prepare to initiate our upcoming Berubicin clinical trials, our execution both on the clinical and manufacturing fronts remain paramount to our success," stated John Climaco, CEO of CNS Pharmaceuticals. "We continue to be encouraged as our partner Pii has now delivered upon many of the critical steps necessary to ensure the quality and availability of Berubicin. We look forward to keeping you updated on our progress as we continue our preparations to submit an IND for Berubicin during the fourth quarter of this year."

CNS’s preparations for filing an IND entail both extensive clinical and manufacturing initiatives. In addition to the progress the Company has made in its manufacturing efforts, it has recently announced critical achievements made on the clinical front. The Company engaged Worldwide Clinical Trials ("Worldwide") as the contract research organization (CRO) for its upcoming Berubicin clinical trials. Worldwide specializes in therapeutic areas with unmet medical needs, including CNS disorders and oncology. Worldwide will work closely with CNS to provide proactive insight and operational support for its upcoming trials. Additionally, the Company engaged Berry Consultants, a leading clinical statistical consulting group, to advise on its Phase 2 trial design for Berubicin. Berry Consultants uses Bayesian statistics to provide innovative clinical trial designs and analysis. The Company has also completed the Phase 1 Clinical Study Report, or CSR, which is now ready for publication.

CNS was recently granted Orphan Drug Designation (ODD) for its lead product, Berubicin, for the treatment of malignant gliomas. The designation provides Berubicin with a special status that can help accelerate its development to treat malignant gliomas by providing CNS with the potential for market exclusivity upon the drug’s approval. The Company plans to file an IND for Berubicin with the FDA during the fourth quarter of 2020.

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.