On September 8, 2020 Silence Therapeutics plc, AIM:SLN and NASDAQ: SLN ("Silence" or "the Company") a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, reported that provides a research and development update (Press release, Silence Therapeutics, SEP 8, 2020, View Source [SID1234568596]).

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Pipeline progress

SLN360

·Silence’s wholly owned lead product candidate SLN360 for the treatment of cardiovascular disease associated with high Lipoprotein(a), or Lp(a), levels, has received approval from the Food and Drug Administration (FDA) for an Investigational New Drug (IND) application to start dose escalation studies in healthy volunteers and secondary prevention patients with elevated Lp(a). SLN360 is designed to address increased cardiovascular risk associated with raised levels of Lp(a), which is considered to affect up to 10% of the world’s population. Whilst we are aiming to start dosing healthy volunteers in the Phase 1 trial by the end of the year, the Covid-19 pandemic may put this timing at risk.

SLN124

Silence has initiated dosing in its randomised, double-blind, placebo-controlled Phase 1 single-ascending dose study of SLN124, the Company’s wholly owned product candidate for beta-thalassaemia and myelodysplastic syndrome (MDS), in up to 24 healthy volunteers. This marks the first dose of a Silence siRNA therapeutic delivered to humans using the Company’s proprietary GalNAc-siRNA platform. GalNAc is a naturally occurring sugar that binds specifically to a receptor which is highly expressed on liver cells, or hepatocytes. Coupling GalNAc sugars to stabilized siRNA molecules allows them to specifically target liver cells and carry out their function.
The FDA recently granted SLN124 orphan drug designation (ODD) for the treatment of adult beta-thalassemia. The FDA previously granted SLN124 ODD for MDS and rare paediatric designation for beta-thalassaemia. SLN124 also has ODD for the treatment of beta-thalassaemia from the European Medical Agency.
Complement Collaboration

·Silence is pleased to announce work commenced on the second target being explored under its complement pathway RNAi collaboration with Mallinckrodt, which triggered a $2.0 million research milestone payment to the Company. Silence would be entitled to receive an additional $2.0 million research milestone payment if work commences on a third target in the collaboration.

Iain Ross, Executive Chairman of Silence Therapeutics, said: "In what has been a busy and successful year for Silence, we have continued to make significant progress with our R&D pipeline. We are delighted to announce today that we have received FDA approval of the IND application for our wholly owned lead product candidate, SLN360, which is designed to address a significant unmet medical need in cardiovascular disease for people born with high Lp(a) levels. We have also made strong progress with our wholly owned product candidate, SLN124, having now dosed the first healthy volunteer. This marks the first dose of a Silence GalNAc-siRNA given to humans and is the culmination of several years’ work in developing this platform. This is truly an exciting time for Silence and we look forward to providing further updates as we move ahead."

On September 8, 2020 Genenta Science, a clinical-stage biotechnology company pioneering the development of a hematopoietic stem cell gene therapy for cancer (Temferon), reported that they will present data from the TEM-GBM study at the Advanced Therapies Congress & Expo 2020 (Press release, Genenta Science, SEP 8, 2020, View Source [SID1234568502]).

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Dr. Carlo Russo, Chief Medical Officer and Head of Development at Genenta, will provide an overview of Temferon, the company’s pipeline and the latest preliminary clinical data in patients affected with glioblastoma multiforme, on September 9, at 6.50pm CET.

On September 8, 2020 Sysmex Inostics, Inc., a global leader and pioneer in blood-based circulating cell-free tumor DNA (ctDNA) analysis for oncology, reported the publication of a clinical study investigating the utility of cfDNA-based markers using the highly sensitive OncoBEAM RAS assay, an enhanced digital PCR test optimized for high sensitivity blood-based mutation detection, as a prognostic tool in patients with metastatic pancreatic ductal adenocarcinoma (PDAC) (Press release, Sysmex Inostics, SEP 8, 2020, View Source [SID1234568256]).

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Pancreatic cancer is the fourth leading cause of cancer related death worldwide. Moreover, PDAC accounts for 90% of all pancreatic cancers with an overall 5-year survival of less than 8%, the lowest survival rate of all cancers. Patients diagnosed with PDAC suffer from a poor prognosis which is attributed to diagnosis at an advanced stage, due to late onset of symptoms and lack of reliable biomarkers for early detection. Moreover, most patients with late stage disease exhibit resistance to available therapy modalities.

The primary initiating genetic event for PDAC is a KRAS mutation, occurring in 94% of pancreatic ductal tumors. KRAS mutational status is typically analyzed in tumor tissue but obtaining biopsy specimens from pancreatic lesions – especially during chemotherapy treatment – may be difficult and requires invasive procedures. Tumor tissue for biomarker testing is only available at diagnosis and is not feasible to access during treatment. CfDNA-based liquid biopsy analysis of KRAS mutations using OncoBEAM technology represents a minimally-invasive tool to measure levels of circulating KRAS mutations to assist in the prognosis determination and management of PDAC patients.

The study published by investigators at the University of Córdoba, Spain indicates that the dynamics of circulating RAS mutations may better correlate with patient outcomes and survival compared with CA19-9 protein blood-based marker testing. CA19-9 is usually found in higher concentrations in pancreatic cancer, however, it is not a tumor specific biomarker and can be elevated for reasons unrelated to cancer. In this comparative analysis, a significant correlation was found between the increase in RAS mutation levels detected in plasma by the OncoBEAM RAS test (r = 0.65, p = 0.02), but not in CA19-9 (r = 0.09, p = 0.78) and survival time. Overall, greater increases in levels circulating KRAS mutation during patient monitoring predicted shorter survival time, with researchers noting that KRAS mutant cfDNA shows great promise as real-time biomarker of tumor response.

Lead author Dr. Toledano-Fonseca of Maimónides Biomedical Research Institute of Córdoba (IMIBIC), said, "Remarkably, sensitive plasma KRAS mutation testing using OncoBEAM alongside of CA19-9 and fragmentation analysis greatly helped prognosis stratification of metastatic PDAC patients. Our results support KRAS MAF as a valuable complementary tool for monitoring the response to chemotherapy treatment in metastatic PDAC patients."

The publication, titled "Circulating Cell-Free DNA-Based Liquid Biopsy Markers for the Non-Invasive Prognosis and Monitoring of Metastatic Pancreatic Cancer" was published in Cancers, July 1, 2020, by Marta Toledano-Fonseca et al.: View Source

On September 8, 2020 Don Hayden, Chairman and CEO of WindMIL Therapeutics, reported that it will present at Citi’s 15th Annual BioPharma Virtual Conference on September 8, 2020 at 4:05 pm EDT (Press release, WindMIL Therapeutics, SEP 8, 2020, View Source [SID1234568096]).

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On September 8, 2020 Nanospectra Biosciences, Inc., a medical device company pioneering a novel use of nanomedicine for selective thermal ablation, reported that it has closed on an initial $3 million in a Series B-1 financing with total approved capital of up to $6 million in the raise (Press release, Nanospectra Biosciences, SEP 8, 2020, View Source [SID1234565071]). The equity round was led by Sirtex Medical as a strategic investor with additional participation from existing and new investors. Sirtex is a leading U.S. based provider of targeted liver cancer therapies via their proprietary SIR-Spheres Y-90 microspheres with global operations. Kevin P. Smith, Sirtex’s General Counsel and Executive Vice President, Business Development, will join the Nanospectra Board of Directors.

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AuroLase, Nanospectra’s lead product, is the first and only ultra-focal ablation therapy designed to maximize treatment efficacy while minimizing side effects typically associated with surgery, radiation and alternative focal therapies. Proceeds from the financing will be used to further develop AuroLase, including full completion of the ongoing pivotal study for prostate cancer tissue ablation, filing of a 510(k) with the U.S. Food and Drug Administration (FDA) for marketing clearance, and preparing for commercialization. In addition, Sirtex and Nanospectra have agreed to exclusively negotiate for a defined period of time for access to AuroLase in certain geographies outside of the U.S.

"We are thrilled to welcome Sirtex Medical as a strategic investor due to their shared commitment and understanding of the clinical and patient benefits of minimally-invasive oncology therapies," said David Jorden, CEO of Nanospectra. "With established global operational capabilities and distribution, they are well positioned to advise on our commercialization efforts in both the U.S. and other geographies. We look forward to Sirtex’s active engagement as we proceed with our clinical programs."

The AuroLase pivotal study for the focal ablation of prostate tissue via nanoparticle directed near infrared irradiation is approved by the FDA under the original Investigational Device Exemption (IDE) and follows the successful first-in-human pilot study that enrolled 46 subjects at three U.S. sites. The ongoing pivotal study will enroll up to 60 patients at up to eight clinical trial sites throughout the U.S. and the combined dataset from both studies will constitute the clinical package Nanospectra will submit to the FDA.

"Nanospectra’s AuroLase is a perfect complement to our technology approach and corporate strategic focus on minimally-invasive therapies," said Kevin R. Smith, CEO of Sirtex Medical. "We look forward to collaborating with Nanospectra to explore and develop innovative therapies to meet unmet medical needs in patients with various cancer conditions."