On October 11, 2022 Elevar Therapeutics, Inc., a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, reported a positive pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) for its investigational drug rivoceranib in combination with camrelizumab as a treatment option for hepatocellular carcinoma (HCC), the most common type of liver cancer and cause of more than 830,000 annual deaths worldwide (Press release, Elevar Therapeutics, OCT 11, 2022, View Source [SID1234621892]). The meeting reaffirmed Elevar’s plans to file an NDA for the combination of rivoceranib plus camrelizumab as early as is feasible in 2023.

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Also, Elevar remains committed to filing an NDA for rivoceranib as a monotherapy treatment option for adenoid cystic carcinoma (ACC) by the end of 2022.

"Our team at Elevar is very pleased with the results of our pre-NDA meeting with respect to rivoceranib plus camrelizumab as a treatment option for HCC, continuing a collaborative process with the FDA that allows us to maintain our timeline for the upcoming rivoceranib FDA filings in two indications with clearly demonstrated unmet medical need," said Saeho Chong, chief executive officer of Elevar. "As evidenced by data shared this year, rivoceranib is proving to be efficacious in both monotherapy, for ACC, and in combination with an anti-PD1 inhibitor, for HCC, and we are excited to continue its regulatory development in the months ahead."

Elevar in September announced during the annual Congress of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) that in a Phase 3 study, camrelizumab plus rivoceranib significantly prolonged overall survival and progression-free survival, and improved overall response rate versus sorafenib, a standard first-line treatment for unresectable HCC.

With efficacy results generally consistent across all subgroups, the data suggested the combination confers a benefit in a global unresectable HCC population. Also, it demonstrated efficacy among those with hepatitis C virus-based etiology, which comprises the majority of U.S. HCC cases1.

In June, Elevar announced at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting that in its Phase 2 clinical trial (Study RM-202) of rivoceranib monotherapy in patients with progressive recurrent or metastatic ACC, rivoceranib demonstrated clinical effectiveness, as indicated by substantially reduced tumor progression during the six months after rivoceranib treatment compared to the tumor progression during the six months prior to rivoceranib treatment.

1. Yoshizawa H. Hepatocellular carcinoma associated with hepatitis C virus infection in Japan: Projection to other countries in the foreseeable future. Oncology. 2002;62(Suppl 1):8–17. [PubMed: 11868791]; El-Serag HB, Rudolph KL. Hepatocellular carcinoma: Epidemiology and molecular carcinogenesis. Gastroenterology. 2007;132:2557–76. [PubMed: 17570226]

About Hepatocellular Carcinoma (HCC)

HCC is the most common type of primary liver cancer. It most frequently occurs in people with chronic liver diseases, such as cirrhosis caused by hepatitis B or hepatitis C infection. HCC typically has a poor prognosis and a lack of treatment options and is therefore a condition with an urgent medical need.

About Adenoid Cystic Carcinoma (ACC)

Adenoid Cystic Carcinoma (ACC) is a rare malignancy that occurs within the secretory glands, most commonly in the major and minor salivary glands of the head and neck, but also found in the breast, skin and elsewhere. It is diagnosed in about 4 of every 1 million people each year – representing a combined 3,100 annual cases in the U.S., EU and Japan – and it afflicts more than 200,000 patients throughout the world, accounting for 5-7% of all head and neck malignancies, according to the Adenoid Cystic Carcinoma Research Foundation. There is no approved standard of care for R/M ACC patients. A previous study showed a baseline progression-free survival of 2.8 months for ACC (Kang EJ, et al. Clin Cancer Res. 2021;27:5272-5279).

About Camrelizumab

Camrelizumab (SHR-1210) is a humanized monoclonal antibody targeting the programmed death-1 (PD-1) receptor. Blockade of the PD-1/PD-L1 signaling pathway is a therapeutic strategy showing success in a wide variety of solid and hematological cancers. Camrelizumab is developed by Hengrui Pharma and has been studied in more than 5,000 patients. Currently, 50 clinical trials are underway in a broad range of tumors (including liver cancer, lung cancer, gastric cancer and breast cancer et al.) and treatment settings.

Camrelizumab, under the brand name AiRuiKa, is currently approved for eight indications in China, including monotherapy for the treatment of HCC (second-line), relapsed/refractory classic Hodgkin’s lymphoma (third-line), esophageal squamous cell carcinoma (second-line) and nasopharyngeal carcinoma (third-line or further), and in combination with chemotherapy for the treatment of non-small cell lung cancer (non-squamous and squamous), esophageal squamous cell carcinoma and nasopharyngeal carcinoma in the first-line setting. The U.S. Food and Drug Administration granted Orphan Drug Designation to camrelizumab for advanced HCC in April 2021.

About Rivoceranib

Rivoceranib is the first small-molecule tyrosine kinase inhibitor (TKI) approved in gastric cancer in China (November 2014). Rivoceranib is a highly potent inhibitor of vascular endothelial growth factor receptor 2 (VEGFR-2), a primary pathway for tumor angiogenesis. VEGFR-2 inhibition is a clinically validated approach to limit tumor growth and disease progression. Rivoceranib is co-developed by Hengrui Pharma in China and by Elevar Therapeutics, Inc. globally (excluding China). It has been studied in more than 6,000 patients worldwide and was well tolerated in clinical trials with a comparable safety profile to other TKIs and VEGF inhibitors. Rivoceranib is currently being studied as a monotherapy and in combination with chemotherapy and immunotherapy in various solid tumor indications. Clinical studies are ongoing in multiple solid tumor types including gastric cancer (as a monotherapy and in combination with paclitaxel), hepatocellular carcinoma (HCC) (in combination with camrelizumab), adenoid cystic carcinoma (as monotherapy) and colorectal cancer (in combination with Lonsurf). Orphan Drug Designations have been granted in gastric cancer (U.S., EU and South Korea), in adenoid cystic carcinoma (U.S.) and in HCC (U.S.). Elevar holds the global rights (excluding China) and has partnered for the development and marketing of rivoceranib with HLB-LS in South Korea. Rivoceranib, under the name apatinib, is currently approved in China for advanced gastric cancer and in second-line advanced HCC by the Chinese-territory license-holder, Hengrui Pharma, under the brand name Aitan.

On October 11, 2022 Viewpoint Molecular Targeting, Inc. ("Viewpoint" or the "Company"), a radiopharmaceutical company developing precision lead-212-based α-particle oncology therapeutics and complementary diagnostic imaging agents, reported the award of two additional Small Business Innovation Research (SBIR) phase II grants totaling $4 million (Press release, Viewpoint Molecular Targeting, OCT 11, 2022, https://viewpointmt.com/viewpoint-molecular-targeting-announces-two-new-grant-awards-of-2-million-each/ [SID1234621891]). The first award of $2 million is intended to assess image-guided dosimetry-based alpha particle therapy for neuroblastoma. The second award of $2 million was awarded for the purpose of combining receptor-targeted alpha particle therapy and immunotherapy to achieve complete responses in metastatic melanoma. These recently awarded grants bring the total amount of SBIR awards of approximately $17 million through October 2022 from the National Institutes of Health (NIH) and National Cancer Institute (NCI) to our principal collaborators at the University of Iowa, including Michael K. Schultz, PhD, our co-founder and Chief Scientific Officer, and Frances L. Johnson, MD, our co-founder and Chief Operating Officer.

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"We are grateful that our science has been recognized since the Company’s inception with such significant support from the NIH and NCI to support our targeted alpha therapy development activities," said Thijs Spoor, CEO of Viewpoint Molecular Targeting.

Dr. Schultz commented, "as our researchers continue to expand the potential applications of targeted alpha therapies generally, and the compelling science behind Pb-212 specifically, we continue to be excited by the transformational nature we think our emerging discoveries will have on the lives of cancer patients."

On October 11, 2022 Illumina Inc. (NASDAQ: ILMN), a global leader in DNA sequencing and array-based technologies, reported a strategic research collaboration with AstraZeneca, a global, science-led biopharmaceutical company, to accelerate drug target discovery by combining their strengths in artificial intelligence (AI) based genome interpretation and genomic analysis techniques along with industry expertise (Press release, Illumina, OCT 11, 2022, View Source [SID1234621890]). The collaboration will evaluate whether a combined framework of these technologies can increase the yield and confidence of target discovery to find promising drugs based on human omics insights.

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Illumina in a strategic research collaboration with AstraZeneca, will combine their strengths in artificial intelligence (AI) based genome interpretation and genomic analysis techniques to accelerate drug target discovery.
Illumina in a strategic research collaboration with AstraZeneca, will combine their strengths in artificial intelligence (AI) based genome interpretation and genomic analysis techniques to accelerate drug target discovery.
"Illumina and AstraZeneca are uniquely positioned to improve the efficiency of pharma pipelines by leveraging industry-leading abilities to identify genetic variants that contribute to human disease," said Joydeep Goswami, chief strategy and corporate development officer, interim chief financial officer at Illumina. "By identifying genes that show evidence of human disease causality, the combined framework has the potential to prioritize drug candidates with increased likelihood of approval."

The collaboration leverages Illumina’s next generation of AI-based interpretation tools, PrimateAI and SpliceAI, in combination with AstraZeneca’s analysis framework for rare variant genomic discoveries—alongside the latter’s own AI tools, including JARVIS and in silico predictors like missense tolerance ratio. As part of the research collaboration a framework combining the two companies’ AI-based tools will be adopted by AstraZeneca’s Centre for Genomics Research to analyze large-scale multi-omics data sets in its digital biobank. The complementary AI tools work to more confidently pinpoint genetic variants that contribute to human diseases, a critical step in the process of developing effective and safe therapies.

Slavé Petrovski, head of AstraZeneca’s Centre for Genomics Research, Discovery Sciences, R&D, said: "Continuous innovation in the AI tools and frameworks that are applied to the growing human genomics, transcriptomics, and proteomics medical research resources will enable us to answer some of the toughest questions and contribute to our aims of uncovering novel drug targets with a higher probability of success while also characterizing patient subgroups that are most likely to benefit from the treatments we discover."

"The next generation of drug discovery lies at the intersection of human genetics and AI, making this a potentially pivotal research collaboration that combines Illumina’s industry-leading ability to interpret genomes at scale with AstraZeneca’s extensive capabilities in large-scale human genetics research," said Alex Aravanis, chief technology officer of Illumina.

The research collaboration focuses on the ability of a combined framework to deliver differential performance across a broad range of human diseases. Upon its successful outcome, the two companies will assess opportunities for a long-term partnership.

On October 11, 2022 Nimbus Therapeutics, a clinical-stage company that is working to design and develop breakthrough medicines through its powerful computational drug discovery engine, reported it has entered into a research collaboration and exclusive, worldwide license agreement with Eli Lilly and Company (Lilly) for the development and commercialization of novel targeted therapies that activate a specific isoform of AMPK for the treatment of metabolic diseases (Press release, Nimbus Therapeutics, OCT 11, 2022, View Source [SID1234621889]).

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Leveraging Nimbus’s computational drug discovery engine and expertise in structure-based drug design, Nimbus is uniquely positioned to develop isoform-selective small molecule activators of AMPK with potential therapeutic application to a broad range of metabolic disorders.

"AMPK is a high-value target for the treatment of metabolic diseases, and drug developers have faced challenges for many years in identifying isoform-selective AMPK activators for tissue-specific therapeutic interventions. Nimbus has established a successful track record in developing and progressing highly-selective small molecules to the clinic against hard-to-interrogate targets, which is a demonstration of the power of our computational and structural approach to drug discovery," said Peter Tummino, Ph.D., Chief Scientific Officer at Nimbus. "We are excited to partner with Lilly and benefit from their deep expertise in metabolic diseases, including diabetes, obesity and related disorders."

"We look forward to collaborating with this innovative team at Nimbus, which has an established record of developing highly-specific small molecule modulators against important therapeutic targets," said Ruth Gimeno, Ph.D., Lilly’s Senior Vice President, Diabetes Research and Clinical Investigation. "Developing therapies together that target AMPK helps address a critical need and is part of Lilly’s ongoing efforts to expand treatment options available to patients with metabolic disorders."

Under the collaboration, Nimbus will be responsible for research activities, and Lilly will be responsible for development and commercialization activities worldwide. Financial consideration for Nimbus includes a series of payments, funding and milestones spread through research, development and commercialization, potentially reaching up to $496 million in total. Nimbus is eligible to receive tiered royalties on global net sales ranging from mid single- to low double-digits.

On October 11, 2022 GT Medical Technologies, Inc. reported the enrollment of the first patient into the GESTALT (GammaTile Enhanced Stupp ALTernative Trial) of GammaTile Surgically Targeted Radiation Therapy (STaRT) for patients with newly diagnosed glioblastomas (Press release, GT Medical Technologies, OCT 11, 2022, View Source [SID1234621888]).

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The GESTALT Clinical Trial will include immediate radiation with GammaTile Therapy followed by the Stupp protocol.

Glioblastoma (GBM) is the most common primary malignant brain tumor in adults, yet effective treatment remains a challenge. In 2005, the Stupp protocol became the standard of care—tumor resection, followed by adjuvant temozolomide (TMZ) and external beam radiation therapy (EBRT) several weeks post-surgery. Unfortunately, patient outcomes remain poor, and there have been few advancements over the last decades.

GammaTile Therapy is the only GBM treatment to be FDA cleared in the past decade and one of only 7 FDA-cleared or -approved GBM treatments in the past 50 years. In clinical trials and patient case studies, GammaTile Therapy provides immediate radiation therapy at the time of surgical resection giving patients an effective treatment alternative for newly diagnosed malignant and recurrent brain tumors of all types.

GammaTiles are radiation implants specifically designed for use in the brain. Each resorbable collagen GammaTile is embedded with Cs-131 radiation sources. The neurosurgeon implants GammaTiles in the cavity created by tumor removal. They begin working immediately to combat residual tumor cells.

The GESTALT Clinical Trial will include immediate radiation with GammaTile Therapy followed by the Stupp protocol. The total radiation administered in this clinical trial with GammaTile Therapy and EBRT will be within standard radiation treatment levels.

"Our goal is to demonstrate how starting radiation immediately at the completion of resection and combining it with the Stupp protocol can improve outcomes," said Matt Likens, president and CEO of GT Medical Technologies. "Since GBM is highly proliferative, approximately 50% of patients experience rapid early progression before they can start the Stupp protocol treatment."

"This is an important study built on sound clinical data with the potential to reshape how we approach glioblastoma treatments in the future. We are pleased to offer this study to our patients at M Health Fairview," commented Dr. Clark Chen, the Lyle French Chair and Head of the Department of Neurosurgery, University of Minnesota Medical School. Dr. Chen played an instrumental role in designing the GESTALT Clinical Trial and leads the GESTALT Clinical Team at this site.