Foundation Medicine Launches FoundationOne®Tracker ctDNA Monitoring Assay for Research Use in Partnership with Natera

On June 8, 2021 Foundation Medicine, Inc. and Natera, Inc. (NASDAQ: NTRA), reported the launch of the research use version of FoundationOneTracker, Foundation Medicine’s personalized circulating tumor DNA (ctDNA) monitoring assay (Press release, Foundation Medicine, JUN 8, 2021, View Source [SID1234583719]). FoundationOne Tracker uniquely combines Foundation Medicine’s tissue-based comprehensive genomic profiling (CGP) platform with Natera’s expertise in ctDNA monitoring.

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FoundationOne Tracker uses optimized algorithms for identifying tumor-specific variants and a personalized assay design that allows for the detection of ctDNA in plasma for use in both advanced- and early-stage research applications. Additionally, the creation of a robust, fully integrated workflow between Foundation Medicine and Natera is expected to provide rapid inclusion of ctDNA monitoring results into retrospective research studies.

"The addition of FoundationOne Tracker for research use provides our biopharma and academic partners access to Foundation Medicine’s broad, advanced-stage CGP footprint," said Foundation Medicine’s Chief Scientific Officer Priti Hegde. "In partnering with Natera, we have been able to leverage the best of both of our technologies to provide a cost-effective and efficient path to bring more personalized monitoring tools to our partners, and down the line, to physicians and patients, to help inform their treatment strategy."

"This partnership will help accelerate personalized ctDNA monitoring as the new standard of care in oncology," said Solomon Moshkevich, Natera’s general manager of oncology. "With Foundation Medicine’s strong track record of scientific leadership and its broad footprint within biopharma, we expect FoundationOne Tracker to become an important new tool for accelerating and improving drug development in oncology."

Indo-Irish Biotech startup CyGenica secures funding from SOSV to accelerate cancer and rare genetic disease therapy

On June 8, 2021 CyGenica has reported that it has raised USD $1.4 million in a seed fund investment round, led by global venture capital investor SOSV (Press release, CyGenica, JUN 8, 2021, View Source [SID1234633831]).

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CyGenica’s disruptive technology addresses the problem of delivering large-molecule therapeutics into living cells without damaging them or triggering an adverse immune response.

The current investment seed round of USD 1.4 million was led by venture capital investor SOSV. Other investors participating in the round included the VOYAGER Health-Tech fund, David Rowan, founder of Voyagers.io, and angel investors Sharaf Yamand Sami Mikati.

CyGenica intends to utilize the investment to accelerate the development of its disruptive drug delivery technology for genome editing, seeking to be a key partner of biopharmaceutical companies in the advancement of cutting-edge therapeutics for cancer and rare diseases to improve patient’s health and quality of life.

"The challenge of delivering drugs for cancer and genetic therapies, be it genes/RNAs/CRISPRs across cell membranes without damaging the cells and triggering an adverse immune response remains a complex hurdle in the pharmaceutical industry. Our groundbreaking technology functions like a universal USB drive. It acts as a nanomachine which can deliver multitudes of cargoes carrying molecular information such as drugs and genetic therapeutics in an efficient, targeted manner without any toxicity and minimum immunogenicity. This will revolutionize drug delivery and lead to better patient outcomes," said Dr Nusrat Sanghamitra, Co-founder and CEO of CyGenica, talking about the latest development.

"This current financing round has brought a diversified international network on board. This will help de-risk our technology, expand our leadership team and take us significantly closer to our goal of enabling safe and targeted intracellular delivery of genetic therapies for cancer and rare diseases," Nusrat further said.

"I am delighted to be leading this investment because CyGenica has solved one of the most pressing problems in biotech: delivery. We are incredibly excited to be part of this revolution," said Bill Liao, Partner SOSV.

"Life-changing healthcare innovation isn’t only coming out of the established life-science hubs. The VOYAGERS Health-Tech Fund is particularly excited to support CyGenica, a remarkable company born in Odisha, India, with the potential to transform targeted drug delivery without the normal side effects that cancer and other patients have had to bear. The VOYAGERS community will do all we can to support Dr Nusrat Sanghamitra and her team in their important mission," said David Rowan, Founder of Voyagers.io.

Clarity expands clinical sites for SARTATE™ neuroblastoma trial

On June 8, 2021 Clarity Pharmaceuticals, a clinical stage radiopharmaceutical company focused on the treatment of serious disease, reported that its clinical trial of 64/67Cu SARTATE for paediatric patients with neuroblastoma has been expanded to include five sites in the U.S (Press release, Clarity Pharmaceuticals, JUN 8, 2021, View Source [SID1234583704]).

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"We are very excited to expand into additional clinical sites which are now open for recruitment in the trial of Clarity’s lead product in neuroblastoma," commented Dr Alan Taylor, Clarity’s Executive Chairman. "Some of the initial data we have received to date from our first clinical site has been shared at one of the industry’s leading conferences, the Society of Nuclear Medicine and Molecular Imaging (SNMMI) Mid-Winter Meeting 2021 and was very well received. We look forward to continuing the 64/67Cu SARTATE clinical trial in this important patient population in some of the leading cancer centres in the U.S."

The 67Cu SARTATE trial is a Peptide Receptor Radionuclide Therapy administered to paediatric patients with high-risk neuroblastoma. It is a multi-centre, dose-escalation, open label, non-randomised, Phase 1/2a theranostic clinical trial with the following confirmed clinical sites:1

Memorial Sloan Kettering Cancer Centre (MSK);
Cincinnati Children’s Hospital Medical Centre;
Medical University of South Carolina;
University of Texas Southwestern Medical Centre; and
University of Wisconsin.
Neuroblastoma most often occurs in children younger than 5 years of age and presents when the tumour grows and causes symptoms. It is the most common type of cancer to be diagnosed in the first year of life and accounts for around 15% of paediatric cancer mortality.2 High-risk neuroblastoma accounts for approximately 45% of all neuroblastoma cases. Patients with high-risk neuroblastoma have the lowest 5-year survival rates at 40%-50%.3

Dr Taylor said: "We are very pleased to have received strong support on the development of 64/67Cu SARTATE for neuroblastoma to date from numerous supporters, such as the five clinical sites, Clarity’s team, our collaborators, and the U.S. Food and Drug Administration in granting both diagnostic 64Cu SARTATE and therapeutic 67Cu SARTATE products Orphan Drug Designations and Rare Paediatric Disease Designations. We believe this highlights the importance of SARTATE development in this important patient population to improve the prognosis of children with high-risk neuroblastoma, where current treatment strategies are limited. We are looking forward to further progressing this trial at five clinical sites and getting closer to our ultimate goal of better treating children and adults with cancer."

References
ClinicalTrials.gov Identifier: NCT04023331
Nadja C. Colon and Dai H. Chung 2011, "Neuroblastoma", Advances in Pediatrics, <View Source>
Valeria Smith and Jennifer Foster 2018, "High Risk Neuroblastoma Treatment Review", Children, <View Source>

VBI Vaccines Granted FDA Fast Track Designation for VBI-1901 for the Treatment of Recurrent GBM

On June 8, 2021 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for VBI-1901, VBI’s cancer vaccine immunotherapeutic candidate for the treatment of recurrent glioblastoma (GBM) patients with first tumor recurrence (Press release, VBI Vaccines, JUN 8, 2021, View Source [SID1234583720]). Fast Track Designation facilitates the development and expedites the review of new therapies to treat serious conditions and fill an unmet medical need.

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"This Fast Track Designation provides additional medical validation and is a meaningful milestone for our development of VBI-1901 as we work to provide clinical benefit for patients who have few treatment options available today," said Francisco Diaz-Mitoma, M.D., Ph.D., VBI’s Chief Medical Officer. "Building on the encouraging data seen to-date – including updated tumor responses and improvement in overall survival compared to historical controls as presented at ASCO (Free ASCO Whitepaper) – we look forward to working closely with the FDA as we progress this cancer vaccine immunotherapeutic candidate with the hope of improving outcomes for adults with recurrent GBM."

About Fast Track Designation

The Fast Track program facilitates the expedited development and review of new drugs or biologics that are intended to: 1) treat serious or life-threatening conditions, and 2) demonstrate the potential to address unmet medical needs. A therapeutic that receives Fast Track Designation is eligible for some or all of the following: 1) more frequent meetings with FDA to discuss the development plan and data needed to support approval, 2) more frequent written communication from FDA relating to the design of the proposed clinical trials and use of biomarkers, 3) Accelerated Approval and Priority Review, if relevant criteria are met, and 4) Rolling Review, which means the company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, instead of waiting until all sections of the application are completed.

Fast Track Designation was granted to VBI-1901, adjuvanted with granulocyte macrophage colony-stimulating factor (GM-CSF), for the treatment of first-recurrent GBM.

ASCO 2021 Poster – Phase 2a Tumor Response Data and Overall Survival (OS) Data Update

The e-poster presentation highlighted patient-specific tumor response data and improvement in overall survival (OS) compared to historical controls across both arms:

Study arm 1 : VBI-1901 + granulocyte-macrophage colony-stimulating factor (GM-CSF)
6-month and 12-month OS : 80% (n=8/10) and 60% (n=6/10), respectively
2 partial responses (≥ 50% tumor reduction) and 2 stable disease observations – 40% disease control rate
Study arm 2 : VBI-1901 + GSK’s AS01B adjuvant system1
6-month OS : 89% (n=8/9) – 12-month OS not yet reached
5 stable disease observations – 50% disease control rate
With few options for recurrent GBM patients, historical control data have demonstrated overall survival to be ~60% at 6-months and ~30% at 12-months after treatment with a monotherapy.2

The e-poster can be found on the "Posters" page of the "News & Resources" section of VBI’s website.

To learn more about the ongoing Phase 1/2a study, visit clinicaltrials.gov (Identifier: NCT03382977).

About VBI-1901 and GBM

VBI-1901 is a novel cancer vaccine immunotherapeutic candidate developed using VBI’s enveloped virus-like particle (eVLP) technology to target two highly immunogenic cytomegalovirus (CMV) antigens, gB and pp65. Scientific literature suggests CMV infection is prevalent in multiple solid tumors, including glioblastoma (GBM). GBM is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, 12,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and has a high mortality.

3Brain AG CorePlate™ technology combines microchips and AI in a revolutionary new approach to drug discovery

On June 8, 2021 3Brain AG has reported a new technology based on a unique combination of microchips and AI that promises to change the way drug candidates are selected at the preclinical level to enter into clinical trials – and to maximize their chances of success (Press release, Lifescience Newswire, JUN 8, 2021, View Source [SID1234583739]).

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Using CorePlate technology, preclinical cell assays are performed in multiwell plates that integrate a microchip in each well to collect rich information from cells, pre-process cell data and streamline it to the paired software. As a result, much more and better data can be extracted, which can be referred to as cell big data. This is fed to an AI with the aim of simplifying interpretation and enabling researchers to discover more information about drugs being tested.

Preclinical studies are vital for early identification of any safety and efficacy issues, and for increasing the chance of success of those drugs entering clinical trials. However, common estimates show that there is still a lot to improve in this area as 90% of the drugs that enter into human trials fail and never reach the market. Among the factors responsible for this high failure rate are the limitations of conducting preclinical tests on animal models. The vast majority of drugs that pass tests on animals do not later work on humans because disease mechanisms vary significantly between species.

New biotechnologies like human stem cells offer more successful alternatives to animal models. These may be in the form of cell culture assays or more sophisticated spheroid and organoid assays that can be used as testbeds for drugs. Human cell-based assays hold the promise to revolutionize drug development, improving the success rate and pushing animal tests towards extinction.

Whatever cell-based assay one wants to investigate, traditional approaches extract cell information via optical systems or a few sensors embedded in plastic substrates. Typically, these substrates are multiwell plates with 24, 96 or even more wells, where each well houses the cell samples to be measured. From these wells, the acquired information travels on limited bandwidth across long distances (compared to the cell size) before reaching a CPU where it gets processed. Long distances and bandwidth issues affect the quality and quantity of information and, consequently, prevent an accurate inspection of cell networks. The CorePlate technology devised by 3Brain disrupts this paradigm by bringing the processing unit in contact with the cells.

20210604 CorePlate press release_other imgs_1"We want to radically change the way preclinical drug screening is performed, offering pharmaceutical companies the opportunity to manage their pipelines more efficiently," says Mauro Gandolfo, CEO and co-founder of 3Brain AG. "Basically, we take traditional plastic multiwell plates and we turn them into intelligent devices with multi-core processing power, which can be programmed according to research needs. Integrated intelligence is a remarkable distinction from other instruments in the preclinical space. Importantly, despite the large amounts of data CorePlate devices can process, the user can still easily generate results thanks to the AI-driven interpretation of data. With the CorePlate technology, pharmaceutical companies can save time and money by better filtering and prioritizing drug candidates. Our ultimate goal is to provide our users with tools that help them more readily identify new cures for diseases."

20210604 CorePlate press release_other imgs_23Brain AG has been working on microchip technology applied to life science for more than 15 years. Alessandro Maccione, CSO and co-founder of 3Brain AG, says, "We already have results showing that our technology is superior in identifying the effects of compounds on several disease models. We started focusing on brain diseases, such as Alzheimer’s and epilepsy, given the high burden that they represent for our society. In addition, we are expanding to other sectors such as cardiac safety, which is one of the main reasons drugs fail in clinical trials. At the same time, driven by our appetite for innovation, we are very attentive to the opportunities offered by advanced cell models such as spheroids and organoids. That’s why we plan to have our CorePlate devices shipped in two "flavors", a planar one that can work with more traditional cell assays and a three-dimensional one where the sensing layer of the microchip is on microneedles that can collect cell information from the inside of tissues and organoids."

Kilian Imfeld, CTO and co-founder of 3Brain AG, adds, "The processing power we have integrated into a CorePlate device is really striking. You can think of it as a modern multi-core CPU, but where the microchip is specifically designed for biological signals. Consequently, we call it BioSPU, BioSignal Processing Unit. Such power allows us to simultaneously process real-time information coming from hundreds of thousands of cells. This continuous data streaming allows for the observation of even subtle changes induced in the cells during the measurement session, such as those caused by the compound under investigation. At the moment we have developed a 6-well CorePlate and we are working on a 24-well and a 96-well format to be released over the coming years. Along the way, our microchips will continue to integrate more functionalities to align with the increased complexity while also maintaining an easy and efficient user experience."