INOVIO Reports FDA Partial Clinical Hold for Planned Phase 2 / 3 Trial of COVID-19 Vaccine Candidate INO-4800

INOVIO | September 28, 2020

INOVIO (NASDAQ:INO), a biotechnology company focused on bringing to market precisely designed DNA medicines to treat and protect people from infectious diseases and cancer, announced that the U.S. Food and Drug Administration (FDA) has notified the company it has additional questions about the company's planned Phase 2/3 trial of its COVID-19 vaccine candidate INO-4800, including its CELLECTRA® 2000 delivery device to be used in the trial. Until the FDA's questions have been satisfactorily addressed, INOVIO's Investigational New Drug Application (IND) for the Phase 2/3 trial is on partial clinical hold. The company is actively working to address the FDA's questions and plans to respond in October, after which the FDA will have up to 30 days to notify INOVIO of its decision as to whether the trial may proceed. This partial clinical hold is not due to the occurrence of any adverse events related to INOVIO's ongoing expanded Phase 1 study of INO-4800, the conduct of which may continue and is not impacted by the FDA's notification. In addition, this partial clinical hold does not impact the advancement of INOVIO's other product candidates in development. INOVIO and its partners are continuing to prepare for a planned Phase 2/3 trial of INO-4800, following resolution of the FDA's partial clinical hold and subject to the receipt of external funding to conduct the trial.

Spotlight

In this paper, we sought to lay out the rationale for investing in small cap biotech. There are two key drivers of stock performance of small cap biotech: clinical data and acquisitions. As clinical data must be analyzed on a case-by-case basis, we have focused our efforts in this paper on factors that will drive M&A in this space. We have also examined the forces that worked to get us to the point we are today.

Spotlight

In this paper, we sought to lay out the rationale for investing in small cap biotech. There are two key drivers of stock performance of small cap biotech: clinical data and acquisitions. As clinical data must be analyzed on a case-by-case basis, we have focused our efforts in this paper on factors that will drive M&A in this space. We have also examined the forces that worked to get us to the point we are today.

Related News

INDUSTRIAL IMPACT

Adaptive Biotechnologies Announces New Data Demonstrating ImmunoSEQ® Technology Can Identify T-Cell Receptors Associated with Crohn’s Disease

Adaptive Biotechnologies | February 19, 2022

Adaptive Biotechnologies Corporation a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to diagnose and treat disease, presented data on T-cell receptor (TCR) sequences associated with Crohn’s disease (CD) during an oral presentation today at the 17th Congress of European Crohn’s and Colitis Organisation (ECCO) being held virtually February 16-19. The study identified and characterized TCR sequences associated with CD utilizing Adaptive’s immunoSEQ® technology, providing fundamental insights into the body’s response to CD at the cellular level. The immunoSEQ assay uses sequencing technology to decipher the complexity of the adaptive immune system. This multi-national study, which was also recently published in The Journal of Crohns and Colitis, utilized immunoSEQ technology to analyze TCRs from blood samples of 1,738 CD cases and 4,970 healthy donors. Intestinal tissue samples from a subset of 380 cases were also analyzed. Through this analysis, 1,121 CD-associated TCRs were identified in patients’ blood and verified in tissue. The identification of disease-specific TCRs is an important first step in identifying a signal and developing an algorithm to inform development of a diagnostic test for CD. “The study identified Crohn’s-specific TCRs in both the blood and tissue of patients with Crohn’s disease, including a large subset that are ’public,’ or shared among patients. These findings suggest that blood-based testing for this often-debilitating disease could be used to diagnose and more effectively manage the disease through the identification of these disease specific T-cell receptors.” Matthieu Allez, MD, PhD, Professor and Head, Department of Gastroenterology, Hospital Saint-Louis, Paris T cells can have a significant impact on inflammation in CD, but until now, disease-associated TCRs have been largely unknown and underleveraged in diagnostics for the disease.1 These findings show that immunoSEQ can successfully identify TCRs for CD in a blood sample, which is reflective of the TCRs in intestinal tissue. The average length of time from onset of symptoms to diagnosis for a CD patient can be 1-2 years in the US but may be much longer in other countries, so the potential to open a new path to identify the disease earlier is significant.2,3 Furthermore, the amount of Crohn’s-related TCRs can provide insights into disease characteristics such as the phenotype and location of the disease, with possible clinical implications. In addition to TCR findings, the analysis also studied the possible association between human leukocyte antigens (HLAs) alleles and CD-associated TCRs, which live on most cells in the body, by leveraging the novel immunoSEQ HLA Classifier. HLA alleles are genetic factors that have been found to contribute to a small portion of risk for CD. In this study, nearly 400 CD-associated TCRs were found to be associated with specific HLA alleles. These TCR associations highlight the importance of studying TCRs in the context of HLA type and potentially point to new risk factors and insights for CD such as the involvement of specific antigens that the immune system may be reacting to in people living with CD. “We’re excited to see these results and their potential to advance the scientific community’s understanding of the immune response to Crohn’s disease. The use of immunoSEQ and characterization of TCRs in the blood have the potential to uncover new knowledge on the development and progression of the disease, with the potential to eventually improve diagnostic options and disease management for people living with Crohn’s,” said Harlan Robins, PhD, Chief Scientific Officer and Co-Founder of Adaptive Biotechnologies. “We look forward to continuing our research and advancing the development of our T-Detect test to include an application in the diagnosis of Crohn’s disease.” CD is a subtype of inflammatory bowel disease, a group of diseases impacting about 6.8 million adults globally.4 Early treatment with effective medications can prevent disease progression towards complications, surgery and disability. However, CD is difficult to diagnose and treat, with more than half of patients initially misdiagnosed. No single blood test currently exists for diagnosis of CD. Instead, patients often undergo a series of tests – often invasive – in order to reach a conclusive diagnosis. Based on the results of this study, Adaptive is further investigating specific TCR signatures that are associated with CD related behavior and disease activity to further the development of T-Detect in this indication. Additional research will also focus on signal optimization and clinical validation to explore commercial utility. About the immunoSEQ® Assay Adaptive’s immunoSEQ Assay helps researchers make discoveries in areas such as oncology, autoimmune disorders, infectious diseases and basic immunology. The immunoSEQ Assay can identify millions of T- and/or B-cell receptors from a single sample in exquisite detail. The immunoSEQ Assay is used to ask and answer translational research questions and discover new prognostic and diagnostic signals in clinical trials. The immunoSEQ Assay provides quantitative, reproducible sequencing results along with access to powerful, easy-to-use analysis tools. The immunoSEQ Assay is for research use only and is not for use in diagnostic procedures. About T Detect™ T-Detect™ is a highly sensitive and specific diagnostic test under development for multiple diseases, translating the natural diagnostic capability of T cells into clinical practice. In 2018, Adaptive and Microsoft partnered to build a map of the immune system called the TCR-Antigen Map. This approach uses immunosequencing, proprietary computational modeling, and machine learning to map T-cell receptor sequences to disease-associated antigens for infectious diseases, autoimmune disorders and cancer. From a simple blood draw, T-Detect will leverage the map to provide an immunostatus for an individual, enabling early disease diagnosis, disease monitoring, and critical insights into immunity. T-Detect COVID is the first clinical test launched from this collaboration and the first commercially available T-cell test designed to detect recent or prior SARS-CoV-2 infections. T-Detect COVID is not FDA-cleared or approved, it has received an EUA from the FDA and is available for prescription use only. About Adaptive Biotechnologies Adaptive Biotechnologies is a commercial-stage biotechnology company focused on harnessing the inherent biology of the adaptive immune system to transform the diagnosis and treatment of disease. We believe the adaptive immune system is nature’s most finely tuned diagnostic and therapeutic for most diseases, but the inability to decode it has prevented the medical community from fully leveraging its capabilities. Our proprietary immune medicine platform reveals and translates the massive genetics of the adaptive immune system with scale, precision and speed to develop products in our Immune Medicine and Minimal Residual Disease (MRD) businesses. We have three commercial products and a robust clinical pipeline to diagnose, monitor and enable the treatment of diseases such as cancer, autoimmune conditions and infectious diseases.

Read More

AI

Iktos Partners with Kadmon to Use AI for New Drug Design

Iktos, Kadmon | May 19, 2021

Iktos, a company specializing in Artificial Intelligence for new drug design, announced today that it has signed a Research Collaboration Agreement with Kadmon, a clinical-stage biopharmaceutical company based in New York, USA, under which Iktos' generative modeling artificial intelligence (AI) technology will be used to allow the rapid and cost-effective design of novel drug candidates. Iktos will use its de novo structure-based generative modeling technologies to find novel compounds that meet a pre-defined target product profile as part of the deal, to speed up Kadmon's early-stage discovery efforts. Kadmon discovers, develops, and delivers small molecules and biologics for the treatment of human diseases. Intending to identify and develop new product candidates for significant unmet medical needs, Kadmon is expanding and incorporating novel drug discovery platforms. The AI technology developed by Iktos, which is focused on deep generative models, aids in the speed and efficiency of the drug discovery process. Iktos' technology creates virtual novel molecules that have all of the properties of a successful drug molecule automatically. This approach, which has been validated by Iktos' other collaborations, is an innovative approach to one of the most difficult problems in drug design: finding molecules that meet several important drug criteria at the same time, such as potency, selectivity, safety, and project-specific properties. Iktos' technology enables the creation of new hits with optimal protein-ligand interactions in early-stage discovery projects, as predicted by molecular modeling technology. This technique allows for a one-of-a-kind discovery of chemical space, as well as the development of innovative molecule designs with greater Freedom to Operate. Furthermore, allowing multi-parametric in silico optimization from the start of a project greatly reduces the hit finding and hit-to-lead optimization phases. About Iktos Iktos, a French start-up founded in October 2016, specializes in the development of artificial intelligence technologies for chemical research, especially medicinal chemistry, and new drug design. Iktos is working on a proprietary and innovative approach focused on deep learning generative models that allow users to build molecules in silico that follow all of the performance criteria of a small molecule discovery project using existing evidence. Iktos technology allows for significant efficiency gains in upstream pharmaceutical R&D. Iktos' software is utilized as both professional services and a SaaS software platform, Makya. Spaya, a synthesis planning software built on Iktos' proprietary AI technology for retrosynthesis, is also in the works.

Read More

CELL AND GENE THERAPY

IndyGeneUS AI Secures Pathogen Sequencing for COVID-19 Genomic Surveillance Initiatives

IndyGeneUS AI | May 20, 2021

Through collaborations, IndyGeneUS AI has acquired genomic analysis and testing capabilities. This pathogen sequencing capacity has emerged at a critical moment in efforts to improve genomic surveillance and monitor COVID-19 variant strains across the continent. IndyGeneUS founder and CEO Yusuf Henriques emphasized the importance of this work. "COVID-19 and its variants pose a danger to the entire African continent, so we must act quickly. Our partners understand the urgency of this mission and are united with their cooperation." Next-generation sequencing (NGS)-based genotyping, targeted sequencing, and comprehensive bioinformatics analysis are among the capabilities. Comparative genomic analysis, disease genetics, epigenomics, and genome mapping are some of the applications of our capabilities. Bradford Wilson, Ph.D., Chief Scientific Officer at IndyGeneUS AI, said, "These capabilities are pivotal for the Continent and the future of precision medicine." "We are currently in talks with several large pharmaceutical firms that see the value of what we are developing to combat infectious and non-communicable diseases alike," he said. In less than a year, IndyGeneUS AI has partnered with Kenyan organizations such as the KAVI Institute of Clinical Research at the University of Nairobi and Afya Rekod. We've also teamed up with Encrypgen, which has created a blockchain-encrypted marketplace for commercializing insights gleaned from our repository and compensating participants who contribute data. IndyGeneUS has certainly understood the increasing need for infrastructure to sustain genomic surveillance activities by developing sequencing capabilities. Following the introduction of COVID-19 variant strains, the World Health Organization (WHO) and the African Centres for Disease Control (Africa CDC) established a network of laboratories last year to improve the continent's capacity to identify emerging variants of concern. More funding and infrastructure are needed for these initiatives. IndyGeneUS intends to accelerate sequencing capabilities across Africa by using public and private partnerships. About IndyGeneUS AI IndyGeneUS AI is a genomics company based in Nairobi is also developing the world's largest block-chain encrypted repository of African clinical and multi-omics data for disease prevention and diagnosis, drug discovery and development, clinical disease management, and the promotion of precision health equity. Mr. Yusuf Henriques, COO Dr. Wilmot Allen, and CSO Dr. Bradford Wilson formed IndyGeneUS. The company, which is owned by African Americans and Africans, has offices in Washington, DC, and a presence in South Africa through the Founder Institute. The Founder Institute is the world's largest pre-seed startup accelerator.

Read More