On January 15, 2020 Bloom Science, a biopharmaceutical company focused on the discovery and development of precision living medicines for currently untreatable neurological diseases, reported signing of an agreement with Duke University, granting the Company an option to an exclusive license to intellectual property and technology related to unique strain isolates and libraries of genetic variants of Akkermansia genus bacteria developed in the laboratory of Raphael H. Valdivia, Ph.D (Press release, Bloom Science, JAN 15, 2020, View Source [SID1234556211]). The underlying principle of Dr. Valdivia’s approach is to isolate, characterize and genetically alter Akkermansia to understand how this beneficial microbe’s genes contribute to promoting immunological health. Bloom will leverage the technology to develop heirloom strain Akkermansia therapeutics, genetically optimized strains and discover novel chemical composition of the Akkermansia metabolome.

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"Multiple reported animal studies have demonstrated the high therapeutic potential of Akkermansia muciniphila in amyotrophic lateral sclerosis, epilepsy, multiple sclerosis, immune oncology, progeroid syndrome and metabolic diseases," said Christopher Reyes, PhD, CEO of Bloom Science. "Dr. Valdivia and his team’s extensive research will be foundational to our therapeutic development platform and furthers our strategy of investing in and expanding our multi-faceted approach to leveraging the gut-brain axis to discover and develop breakthrough therapies for the largest number of patients."

"We are very excited for the opportunity to generate Akkermansia strains with the most beneficial activities and accelerate their application as potential therapeutics, " said Dr. Valdivia, who will be joining Bloom as a scientific founder.

The financial terms of the agreement have not been disclosed.

On January 15, 2020 OncoTartis, Inc. and Children’s Cancer Institute jointly reported the publication of two research manuscripts in a leading onco-hematological journal Leukemia, both devoted to the OncoTartis’ clinical drug candidate OT-82 (Press release, OncoTartis, JAN 15, 2020, View Source [SID1234553281]).

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A significant proportion of blood cancers in both adults and children remain resistant to current treatments and are often fatal. The results of studies conducted collaboratively by teams in Buffalo and Sydney open a new treatment opportunity for patients with currently incurable blood cancers by introducing a new drug candidate shown to be highly effective in preclinical models.

The first paper describes the discovery, mechanism of action, toxicological profile and preclinical efficacy of OT-82 that was developed by OncoTartis for treatment of refractory leukemias and lymphomas. It takes advantage of the discovery of an extremely high dependence of malignancies originating from the hematopoietic system on elevated levels of nicotinamide dinucleotide (NAD), an essential component of multiple metabolic and stress response mechanisms of the cell. OT-82 inhibits one of the major enzymes, nicotinamide phosphoribosyl transferase (NAMPT) responsible for NAD production. Its further clinical development is specifically focused on blood-derived cancers as the main disease target. OT-82 is currently undergoing a multicenter Phase I trial in the US.

The second paper on OT-82 describes a study conducted at Children’s Cancer Institute (Sydney). The Institute has been a world leader in establishing a large collection of refractory children’s leukemias grown in laboratory mice for new drug testing. These are widely accepted to be the most clinically-relevant models of childhood leukemia anywhere in the world.

This collection was utilized to compare the efficacy of OT-82 with current treatments for childhood leukemia. OT82 demonstrated remarkable efficacy when used alone and was even more effective when used in combination with conventional treatments, thereby indicating promise for children with high risk blood cancers.

Currently, OT-82 is being tested in a clinical trial of adults with relapsed or refractory lymphoma. Following the successful completion of this trial, it is anticipated that the drug will proceed to clinical trial in children with high-risk acute lymphoblastic leukemia.

Professor Andrei Gudkov from Roswell Park Comprehensive Cancer Center in Buffalo NY, a Chief Scientific Officer of OncoTartis, said: "The main principle of the research strategy that led us to OT-82 was identification of a pharmacological agent specifically toxic for malignant cells of blood origin. We were surprised when the compound that came out of an unbiased search appeared to be a NAMPT inhibitor, an enzyme that has been considered as a target for cancer treatment but whose association with malignancies of blood origin was unknown. We are especially excited about the potential use of OT-82 for treatment of refractory childhood malignancies, an opportunity revealed in the joint studies with our Australian partners."

Professor Michelle Haber AM, Executive Director of Children’s Cancer Institute in Sydney, indicated this was a promising development for children with high-risk leukemia.

"For children with particularly aggressive leukemia, the options for effective yet safe treatments are extremely limited and therefore novel therapeutic options are urgently needed. If OT-82 is found to be safe and effective in adults, we are very hopeful that it may provide an exciting new treatment approach for the worst cases of paediatric leukemia, with the added potential benefit of allowing reductions in doses of chemotherapy and hence diminished adverse treatment side-effects for these children."

Dr Michelle Henderson, Senior Scientist, who together with Dr Klaartje Somers led the research at Children’s Cancer Institute, added: "In our hands, OT-82 has proven to be one of the more broadly active compounds tested so far in this panel of high-risk pediatric acute lymphoblastic leukemia." Dr Somers concluded: "OT-82 thus appears to be a promising anti-cancer drug for the treatment of a broad range of high-risk and aggressive pediatric acute lymphoblastic leukemia subtypes for which novel therapeutic options are urgently needed."

The research at Children’s Cancer Institute was possible thanks to the support of the National Cancer Institute (part of the US National Institutes of Health), The National Health and Medical Research Council of Australia, Cancer Australia, The Kids’ Cancer Project, The Leukaemia Foundation, Anthony Rothe Memorial Trust, Cancer Council NSW, Tenix Foundation, ISG Foundation, the Children’s Leukemia & Cancer Research Foundation (Perth) and the Australian Government Department of Education and Training.

On January 10, 2020 Concerto HealthAI reported an expansion of their existing Precision Oncology collaboration with Pfizer Oncology to advance Concerto HealthAI’s use-case engineered Real-World Data (RWD) for an array of clinical development initiatives across breast, lung, prostate and renal cell carcinoma cancers that will drive innovative new study designs and non-interventional studies (Press release, Concerto HealthAI, JAN 15, 2020, View Source [SID1234553254]).

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The collaboration, first announced in early 2019, builds on the success of the current utilization of Concerto HealthAI’s RWD to now also include claims data linked to clinical EMR data and linked next-generation sequence panel data for both research and potential regulatory purposes. Pfizer will also be one of the earliest adopters of Concerto HealthAI’s RWE enterprise solution, eurekaHealth 3.0, that provides the industry’s first integration of RWD and AI-enabled tools and models within an analytic cloud environment.

"We are looking forward to expanding our collaboration with Concerto HealthAI and exploring the application of analytics to RWD for generating RWE in genitourinary cancers, a key area of our Oncology strategy," said Chris Boshoff, MD, PhD, Chief Development Officer, Oncology, Pfizer Global Product Development.

"This expanded collaboration aims to find and help patients with some of the most devastating cancers," said Jeff Elton, PhD, CEO of Concerto HealthAI. "We believe that use of our high-precision, research-grade RWD, AI solutions, and scientific expertise can advance innovative study designs, like External Control Arms, providing confidence in the interpretation of study results and accelerated execution."

The collaboration uses RWD licensed from the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s (ASCO) (Free ASCO Whitepaper) CancerLinQ program and other partnered data sources. Concerto HealthAI’s RWD analyses have been jointly evolved with ASCO (Free ASCO Whitepaper) and the U.S. Food and Drug Administration, and others, and presented at ISPOR, ASCO (Free ASCO Whitepaper), and related forums and publications. A Concerto HealthAI-Pfizer joint steering committee will oversee the collaboration. The collaboration’s first outcomes research publications are expected in mid-2020.

On January 15, 2020 Artisan Bio, Inc., a stealth cell therapy engineering company, reported it has entered into a global research and collaboration agreement with Takeda Pharmaceutical Company Limited ("Takeda") for the discovery, development, and commercialization of novel cell therapy products (Press release, Artisan Pharma, JAN 15, 2020, View Source [SID1234553249]).

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Under the terms of the agreement, Artisan Bio will deploy its STAR platform and synthetic biology expertise to construct customized and precisely engineered cell therapies. Artisan will lead discovery efforts, including gene editing, and Takeda will be responsible for the development, manufacturing, and commercialization of the resulting cell therapy products.

"We are thrilled to collaborate with Takeda and combine our advanced cell engineering capabilities with Takeda’s visionary cellular therapy programs," said Ryan T. Gill, CEO of Artisan Bio. "Many limitations that exist in current generation cell therapies can be addressed through the precision engineering of cells for enhanced performance. This collaboration will accelerate the availability of more effective next-generation cellular therapies for patients with high unmet needs."

On January 15, 2020 GenScript, a world leading biotechnology company, reported its support of Avidea Technologies’ efforts to develop a groundbreaking peptide-based personalized cancer vaccine (Press release, GenScript, JAN 15, 2020, View Source [SID1234553248]).

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Research published in Nature Biotechnology on January 13, 2020 describes the systematic development of Avidea’s personalized cancer vaccine (SNP-7/8a) based on a self-assembling nanoparticle technology (referred to as "SNAP"), which was shown to more efficiently induce T-cell responses against tumor neoantigens compared to traditional vaccines. Developing and validating SNP-7/8a as a personalized cancer vaccine required synthesis of hundreds of unique neoantigen peptides, which were successfully synthesized by Genscript in support of this work.

"Screening hundreds of neoantigens in mice to validate the generalizability of SNP-7/8a was a tremendous undertaking that could not have been possible without the diligent efforts of GenScript’s peptide chemists who were able to rapidly synthesize even the most challenging sequences at the high purity required," said Avidea Technologies’ CEO Geoffrey Lynn, PhD, a primary author of the study.

The publication is particularly timely as personalized cancer vaccines are garnering increasing attention among the scientific and medical community as important components to enabling precision immuno-oncology. While traditional cancer treatment is largely based on "one size fits all" approaches such as chemotherapy and radiation, which can be limited by off-target effects and low efficacy for treating certain advanced cancers, precision immuno-oncology promises to reduce off-target toxicity and improve efficacy by training a patient’s own immune response, particularly T cells, to attack cancerous cells.

Key to enabling precision immuno-oncology is focusing the immune response against tumor-specific peptide antigens, particularly neoantigens, which are mutant peptides that are unique to cancerous tissue and are not found in healthy tissue. Current vaccines using neoantigen peptides have shown promise in initiating neoantigen-specific T-cell responses. However, the response itself is often not as robust as may be required for tumor regression, which may be in part due to the inability of conventional vaccine platforms to account for the broad variability of neoantigen peptide properties.

In order to address challenges associated with neoantigen peptide variability, Avidea Technologies, working in close collaboration with the Vaccine Research Center at the National Institutes of Health (NIH), developed a personalized cancer vaccine platform (SNP-7/8a) that ensures any neoantigen peptide, irrespective of the underlying amino acid sequence, can be self-assembled into uniform nanoparticles of an optimal size and composition for inducing T-cell responses.

To support this work, GenScript synthesized hundreds of challenging neoantigen peptides that were selected to have a wide range of properties, including neoantigen peptides with extremes of net charge and hydrophobicity, which was instrumental to enabling Avidea and NIH scientists to validate the generalizability of the SNP-7/8a platform as a personalized cancer vaccine. In order to accomplish these challenging syntheses, the GenScript team utilized their 15 years of experience in complex and hydrophobic peptide synthesis, high throughput LPS/SPS/microwave synthesis platforms, and patented quality control program.

"We are honored that Avidea chose to partner with GenScript to synthesize the hundreds of difficult peptides required for this study. As neoantigens can have quite variable properties, working on this project has strengthened our ability to manufacture large quantities of these difficult peptides," said Xin Zhang, associate director of GenScript’s peptide services. "We look forward to our continued collaboration with Avidea on future breakthrough neoantigen peptide vaccines to advance the precision immuno-oncology field."

Leveraging GenScript’s core strength in difficult peptide synthesis, Avidea and the NIH were able to demonstrate the utility of SNP-7/8a for improving formulation consistency and increasing the magnitude and breadth of neoantigen-specific T-cell responses in mice as compared with two gold-standard cancer vaccine technologies. Additionally, the SNP-7/8a platform was also shown to induce CD8 T-cell responses in non-human primates, which is predictive of responses in humans. Based on these encouraging data, Avidea is planning to advance SNP-7/8a to clinic in 2020.

GenScript was pleased to support these studies and hopes that such efforts and continued cooperation with Avidea and others in this space will lead to more efficacious precision immuno-oncology treatments and ultimately improved outcomes for patients.