On October 11, 2022 PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, reported expanded interim data in the Phase 2 clinical trial investigating the PDS0101-based triple combination therapy in advanced human papillomavirus (HPV)-positive cancers (Press release, PDS Biotechnology, OCT 11, 2022, View Source [SID1234621894]). This Phase 2 study is being conducted at the Center for Cancer Research (CCR) at the National Cancer Institute (NCI), one of the Institutes of the National Institutes of Health. The interim efficacy data from 37 HPV16-positive evaluable patients, including 29 patients in the checkpoint inhibitor (CPI) refractory arm, are consistent with the results presented at ASCO (Free ASCO Whitepaper) 2022 and affirm the selection of CPI refractory patients as the initial patient population for ongoing clinical development of the triple combination.

The NCI-led Phase 2 clinical trial (NCT04287868) is investigating PDS0101 in combination with two investigational immune-modulating agents – M9241, a tumor-targeting IL-12 (immunocytokine), and bintrafusp alfa, a bifunctional checkpoint inhibitor (PD-L1/ TGF-β) – in recurrent or metastatic HPV-positive cancers in patients who have failed prior therapy. The triple combination is being studied in CPI-naïve and -refractory patients with advanced HPV-positive anal, cervical, head and neck, vaginal, and vulvar cancers. Both M9241 and bintrafusp alfa are owned by Merck KGaA, Darmstadt, Germany.

Highlights of the expanded interim data are as follows:

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•Survival data: 66% (19/29) of HPV 16-positive CPI refractory patients in the cohort were alive at a median follow up of 16 months. Historically, this group has a median overall survival of only 3-4 months. 1

•Safety profile: 48% (24/50) of patients experienced Grade 3 treatment-related adverse events (AEs), and 4% (2/50) patients experienced Grade 4 AEs. There were no grade 5 treatment-related AEs

Results for HPV 16-positive checkpoint inhibitor naïve patients also continue to appear to be encouraging: 75% (6/8) of CPI naïve patients were alive at a median of 25 months of follow up. 38% (3/8) of responders had a complete response.

"The expanded interim data investigating the PDS0101-based triple combination therapy in advanced HPV-positive cancers continue to appear to show clinical signs of efficacy, durability and safety in an extremely challenging patient population with very few available treatment options," stated Dr. Frank Bedu-Addo, President and Chief Executive Officer of PDS Biotech. "Importantly, these results affirm the decision to explore this novel combination for the treatment of CPI refractory patients, who have no approved standard of care, and support development of a combination therapy to address the significant unmet need."

On October 11, 2022 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it has scheduled its third quarter 2022 financial results conference call and webcast for 5:00 a.m. Pacific Time (8:00 a.m. Eastern Time) on November 1, 2022 (Press release, Neurocrine Biosciences, OCT 11, 2022, View Source [SID1234621893]).

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The schedule for the press release and conference call / webcast is as follows:

NBIX

The webcast can also be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

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.