Plinabulin Combo May Be Superior Therapy for Prevention of Chemotherapy-Induced Neutropenia – Targeted Oncology

Treatment with plinabulin in combination with pegfilgrastim (Neulasta) achieved the primary end point and several key secondary end points in the phase 3 PROTECTIVE (Study 106) clinical trial, which evaluated the combination compared with pegfilgrastim alone for superiority in chemotherapy-induced neutropenia prevention, according to the interim results announced by BeyondSpring, developer of the drug.

These results demonstrate significant enhancement to granulocyte colony-stimulating factors (G-CSFs) with the combination compared with pegfilgrastim alone for the prevention of grade 4 or severe neutropenia, supporting the potential for superiority with the combination compared with pegfilgrastim alone.

These interim results from the PROTECTIVE-2 Phase 3 study, which compares the Plinabulin-Neulasta combination to Neulasta alone, have the potential to be clinically meaningful for cancer patients receiving chemotherapy, stated Douglas W. Blayney, MD, professor of Medicine at Stanford Medical School and global principal investigator of Plinabulins chemotherapy-induced neutropenia studies. Since most infections, hospitalizations, and other complications of chemotherapy-induced neutropenia occur in the first week after chemotherapy, it is particularly gratifying to see the combinations clinical benefit demonstrated.

The primary end point was the rare of severe neutropenia prevention in cycle 1 (P <.01). The key secondary end points included duration of severe neutropenia in cycle 1 (P <.05) and duration of severe neutropenia in the first 8 days of cycle 1 (P <.05). The achievement of these end points demonstrates the agents ability to provide early protection against severe neutropenia induced by chemotherapy.

The interim analysis of the double-blind, active-controlled, global PROTECTIVE-2 study was pre-specified for the first 120 patients accrued. The study also has procedures in place for the prevention of potential bias after the planned interim analysis, and BeyondSpring opted to be informed by independent statisticians on whether the pre-specified P values were met rather than the exact P values.

The study was designed to assess both the safety and efficacy of plinabulin in patients with breast cancer treated with docetaxel, doxorubicin, and cyclophosphamide (TAC) in a 21-day cycle. Plinabulin was administered in a 40 mg dose on day 1 with a 6 mg dose of pegfilgrastim on day 2, while pegfilgrastim was administered at the same 6 mg dose on day 2 in the control arm. TAC is considered a high-risk chemotherapy regimen associated with neutropenia.

PROTECTIVE-2 is a superiority study for chemotherapy-induced neutropenia efficacy compared with pegfilgrastim alone and is currently enrolling patients. Grade 4 neutropenia rate for TAC and pegfilgrastim is observed in 83% to 93% of patients, which represents a severe unmet medical need.

The study drug and G-CSF have complementary mechanisms of action for preventing chemotherapy-induced neutropenia. Plinabulin is a first-in-class differentiated immune and stem cell modulator, which is currently in late-stage clinical development as a potential treatment for increasing overall survival in patients with cancer in addition to its ability to alleviate chemotherapy-induced neutropenia.

Plinabulin is also under evaluation in a phase 3 study as a direct anticancer drug as treatment of patients with nonsmall cell lung cancer and 2 phase 3 studies for the prevention of chemotherapy-induced neutropenia. The data for this agent in preventing chemotherapy-induced neutropenia highlights its ability to boost the number of hematopoietic stem or progenitor cells (HSPcs), as well as lineage-/cKIT+/Sca1+ cells in mice. Plinabulins effects on HSPcs may explain its ability to treat chemotherapy-induced neutropenia as well as its ability to reduce chemotherapy-induced thrombocytopenia and increase circulating CD34-positive cells.

To be included in the study, patients should be candidates for TAC in either the adjuvant or neoadjuvant setting, have early-stage breast cancer, and have received no prior chemotherapy. Patients must also have an ECOG performance status of 0 or 1 and a life expectancy of at least 3 months. Patients cannot be included in the study if they have a history of myelogenous leukemia, myelodysplastic syndrome, or sickle cell disease; use of CYP3A4, CYP2D6 or P-glycoprotein inhibitors and inducers within 14 days prior to receiving the study drug; or have received an investigational agent or tumor vaccine within 2 weeks, any concurrent anticancer therapies, or a bone marrow or stem cell transplantation.

These results could help to confirm the patient benefit of Plinabulins different mechanism of action from the G-CSF-based agents, such as Neulasta, Blayney stated. Plinabulin appears to have chemotherapy-induced neutropenia protection in Week 1, and G-CSFs have protection in Week 2 of chemotherapy cycles. The combination should logically provide significantly better protection than Neulasta alone as shown in the interim readout. We are well on our way to confirming that the combination offers protection throughout the chemotherapy cycle, which is an unmet medical need.

Reference

BeyondSpring Announces Positive Topline Interim Results from PROTECTIVE-2 (Study 106) Phase 3 Trial Evaluating Superiority of Plinabulin in Combination with Neulasta for Chemotherapy-Induced Neutropenia Prevention. NewsRelease. BeyondSpring. June 15, 2020. Accessed June 15, 2020. https://bit.ly/2YDJbiQ

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Plinabulin Combo May Be Superior Therapy for Prevention of Chemotherapy-Induced Neutropenia - Targeted Oncology

Tech Start-up Launches Contest, Enabling Scientists to Promote Their Research After the Cancelation of Key Medical Research Meetings this Year -…

TORONTO, June 15, 2020 /PRNewswire/ --TodayBioRender, creator of the first web-basedgraphical tool for scientists to create publication and presentation-ready figures, announced its inauguralGraphical Abstract Contest, to give scientists a way to promote their research and win prize rewards. As key medical research meetings throughout the world have been canceled, postponed or moved to a virtual environment, BioRender is donating its $50K marketing budget to the science community in the form of cash prizes for a first-of-its-kind, worldwide, scientific art contest.

"This contest comes at a time when all conferences and meetings have been cancelled or postponed, so having the opportunity to be involved with these graphical abstracts has been a refreshing way to explore a wide variety of topics, including the pathology and immunology of SARS-CoV-2 infection," said Jarrod Dudakov, Assistant Professor of Immunology at Fred Hutch Cancer Center. "Graphical abstracts are an increasingly vital aspect of science communication and this competition, and BioRender more generally, contribute significantly toward raising the bar in the research community's progress in visual literacy."

At a time when scientists are racing for answers while also trying to help calm fears around the world, being able to network, share research, and present information in a unified, digestible manner is critical. Social distancing has made this a largely remote effort and presents a unique challenge for the science community to share research. To that end, BioRender created a virtual community to enable the continued sharing of research.

"At BioRender, we have always recognized the need for scientific knowledge to be universally understood and communicated through visuals that are clear, standardized, and accessible," said Shiz Aoki, co-founder and CEO of BioRender. "Our mission is to empower the world to communicate science faster through visuals. Graphical abstracts are quickly becoming the ideal way to lower the friction in communicating complex scientific concepts."

There are more than 5,000 entrants in the contest, which include original research from Stanford Medicine, John Hopkins Medicine, Harvard Medical School and Yale School of Medicine. Today BioRender selected People's Choice Award winners for each category of the contest:

Submitted research will be peer-reviewed and each submission will be eligible for cash prizes of up to $6,000. Submissions to theGraphical Abstract Contest contribute to a public gallery that the scientific community can virtually tour to learn about the latest in research while networking and voting for their favorites to win People's Choice Awards. Lead scientific researchers and renowned experts from around the world will come together to judge submissions within their specialization for visual appeal, clarity, accuracy, complexity, and ease of understanding with the best winning Expert's Choice Awards.

BioRender will announce the Expert's Choice Award finalists on June 17 and all winners will be announced on June 29.

About BioRender

At BioRender, we envision a world where science is universally communicated and understood through visuals. We believe science communication should always be fast, repeatable, and standardized. To help make that vision a reality, we are creating the standard visual language of biology and the suite of tools to communicate it.

Led by CEO and Co-founder Shiz Aoki, the Lead Medical Illustrator for National Geographic for a decade, our team is composed of environmentally and socially conscious engineers, science illustrators, designers, and entrepreneurs who are backed by an incredible group of investors, including Y Combinator.

Recently heralded in Nature as 'a staple of biomedical research drawings', our first product is a web-based graphical tool that enables scientists to create publication and presentation-ready figures up to 50 times faster than traditional tools. With a comprehensive library of 20,000+ icons and intuitive drag-and-drop functionality, this tool enables scientists across 30+ fields of life science to create professional illustrations that more effectively communicates their research.

Media Contact: Interdependence PR for BioRender Samantha Harber; [emailprotected] (406) 272-2627

SOURCE BioRender

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Children’s Hospital Los Angeles Ranked No. 1 Children’s Hospital in the Western U.S., No. 5 Nationally for Second Straight Year – Newswise

Newswise LOS ANGELES (June 16, 2020) Children's Hospital Los Angeles (CHLA) ranks again among the nations premier destinations for pediatric care, according to the U.S. News & World Report Best Children's Hospitals annual list released today.

CHLA not only retained its national No. 5 ranking in U.S. News Honor Roll of Best Childrens Hospitalswhich recognizes institutions with the most outstanding pediatric clinical careit continued its four-year streak of being the highest-scoring childrens hospital in the entire Western United States.

To make U.S. News & World Reports prestigious Best Childrens Hospitals Honor Roll, one must demonstrate the strongest achievements in clinical excellence, with a matchless team of expert, compassionate specialists committed to research and education as well as protocols that drive safety and quality and consistently lead to the best health outcomes for patients, says CHLA President and Chief Executive Officer Paul S. Viviano. "This honor affirms the work of every CHLA team member and our belief that when parents choose Childrens Hospital Los Angeles, they are choosing the best care for kids."

Every year, U.S. News scores nearly every major hospital and health system in the country and ranks them according to performance benchmarks, peer review, certifications, and other data provided by the hospital and third-party measurements of excellence. Children's hospitals are ranked separately from other facilities due to the specialized expertise, equipment and facilities required to care for infants, children and youth.

This year, U.S. News surveyed 118 pediatric medical centers, including hospitals that are freestanding or part of a larger institution. CHLA improved its ranking over last year in seven of the 10 pediatric specialty categories the survey considers, including a number two ranking for Neonatal Care. In all, the hospital earned top-10 recognition in seven of those categories:

CHLA has an organization-wide commitment to providing our patients the care they need no matter their circumstances, says CHLA Chief Medical Officer James Stein, M.D., MSc. "Our clinical teams often treat the most acute cases that are outside the scope or expertise of other childrens hospitals in California, and being named a Top-5 childrens hospital in the U.S. is a testament to the clinicians and staff who work every day to make sure each child receives the best care and experience possible.

Founded in 1901, Children's Hospital Los Angeles is a pediatric academic medical center built around its mission of creating hope and building healthier futures for children. Renowned for its world-class clinical care, leading-edge research and one of the largest and most successful pediatric training programs in the countryall while being the pediatric safety net hospital for the entire regionCHLA now sees more than 600,000 patient visits annually between its main hospital and five neighborhood care clinics.

CHLA physicians, nurses and clinical staff provide compassionate and lifesaving pediatric care for patients ranging from infants to young adults, hailing from all 50 states and more than 75 countries. Clinical care is led by physicians who are faculty members of the Keck School of Medicine of USC. Many of the hospital's achievements in care are made possible through a cohesive relationship between clinical experts at the bedside and the basic, translational, and clinical research conducted in The Saban Research Institute of CHLA.

In the past year, CHLA has had several notable achievements, including:

U.S. News and World Report works with research firm RTI International to develop its annual Best Children's Hospitals list, a collaboration between hospitals and the magazine to benchmark the performance of childrens hospitals for the benefit of parents and their children. The survey evaluates hundreds of data points, including patient survival and surgical complication rates; staffing, technology and special services; infection prevention and delivery of care; reputation among peer physicians nationwide (i.e. Where would the best pediatric specialists send their kids?); how involved parents are in their childrens care; and many other measurements of excellence.

U.S. News Media Group, the parent of U.S. News & World Report, announced the 2020-21 hospital rankings online at 12:01 a.m. EST on Tuesday, June 16. For additional information, please visit the Best Childrens Hospitals Honor Roll and specialty rankings page at usnews.com/childrenshospitals.

About Children's Hospital Los Angeles Founded in 1901,Children's Hospital Los Angelesis ranked the topchildrens hospital in California andfifth in the nation for clinical excellence with its selection to the prestigious U.S. News & World Report Honor Roll of childrens hospitals. Clinical care is led by physicians who are faculty members of the Keck School of Medicine of USC through an affiliation dating from 1932.The hospitalalso leads thelargest pediatric residency training program at a freestanding childrens hospital of its kind in the western United States.TheSaban Research Instituteof Childrens Hospital Los Angeles encompasses basic, translational and clinical research conducted at CHLA. The hospitals Global Health Program facilitates services for international patients from more than 75 countries. To learn more, follow us on Facebook, Instagram, LinkedIn and Twitter, and visit our blog for families (CHLA.org/blog) andour research blog (ResearCHLABlog.org).

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Children's Hospital Los Angeles Ranked No. 1 Children's Hospital in the Western U.S., No. 5 Nationally for Second Straight Year - Newswise

Targeted policy support for emerging biomedical innovations – Open Access Government

Like many states, the UK government has committed to supporting disruptive innovations.1 These are considered to hold greater potential for economic growth and development than incremental advances in established technologies. Within this broad strategy the bioeconomy, the area of industrial activity based on commercialising life sciences research is given a particular importance. The bioeconomy includes sectors like biofuels, agricultural biotechnology, and medical biotechnology.2 In the latter case, advances in medical biotechnologies hold promise for treating, and even curing, serious and chronic diseases as well as driving growth and prosperity. Regenerative medicine (RM), the biotechnology-based use of cells, tissues, and genes as medicinal products, is certainly disruptive in that they differ in important ways from traditional pharmaceuticals and medical devices.3

The UK has taken a number of policy measures to support the development of the RM industry. The Regenerative Medicine Platform funding schemes promote and co-ordinate academic translational research. The Catapult centres, including the Cell and Gene Therapy Catapult, the Medicines Discovery Catapult and the High Value Manufacturing Catapult, provide advice, facilities and infrastructure to support businesses, especially Small and Medium-sized Enterprises (SMEs); with potential to contribute to the RM value chain. The Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Office offers a RM advice service to help academic and commercial developers navigate the complex regulatory framework for biological therapies, while the recent Accelerated Access Review proposed a raft of measures to speed up the regulatory timeline for transformative new therapies more generally.4

However, it does not necessarily follow that all parts of the biomedical sector will be equally disrupted by any given RM technology, nor that all RM technologies will be disruptive in exactly the same way.5 The ESRC-funded Biomodifying Technologies project6 analysed three case studies of biotechnologies with disruptive potential: gene-editing which allows faster, more accurate genetic modification, induced pluripotent stem cell (iPSC) technology that allows an ordinary skin or blood cell to be turned into a stem cell capable of producing any tissue type in the human body, and 3D bioprinting which can produce three-dimensional structures made from living tissues.

Gene editing and iPSC are advances on earlier generations of genetic engineering and stem cell technologies. They align reasonably well with the existing skill sets, goals, equipment, and techniques of researchers working in both academic and commercial settings. They are not especially disruptive at the level of basic research. Bioprinting requires skills, tools and techniques from engineering, materials chemistry, computer-aided design, biology, and medicine. This has necessitated greater disruption in the form of organisational change, to create new research groups and foster collaborative learning across disciplines.

For all three technologies, there are also well-established pathways to extract near-term value from basic research: peer-reviewed publications, patent applications, and the market for reagents, tools, and equipment. Each case demonstrates clear growth in the number of papers, patents, and reagent/equipment sales, although the rate of acceleration is greatest for CRISPR-based gene editing and slowest for bioprinting.

The pathways to realise longer-term, clinical, and economic value are less well established for RM. The healthcare sector is seen as particularly resistant to disruptive innovations, due to the lengthy regulatory process and powerful incumbent firms, which have historically been wary of investing in RM.7 The process of scaling laboratory protocols for cell or gene-based therapies into industrial procedures, taking products through clinical trials to establish safety and efficacy, and securing reimbursement, is every bit as experimental and involves as much learning by trial and error as exploratory laboratory research, but with much higher financial stakes. Interest from incumbents appears to be growing, as recent years have seen an increase in the number of cell or gene-based therapies reaching the market. However, there is no off the shelf manufacturing solution, as different RM products have different attributes: in the industry there is a popular idiom the product is the process. This means that the acceleration seen at the basic R&D stage does not unproblematically translate into speedy translation further down the pathway.

Rather, initial clinical applications of gene editing, iPSC and bioprinting are targeted at a more limited range of niche applications. The niches for each technology are shaped by a number of critical factors. Smaller tissues, such as the eye require fewer replacement cells or lower titres of gene editing vector, which are more manageable with current manufacturing capacity. The challenges of manufacturing at scale, combined with high anticipated costs, combine to make narrowly defined subsets of disease categories, with high unmet need, a preferred route for commercial development, especially where there is potential for a disruptive new product to demonstrate significant Quality of Life gains over the current standard of care.

Indications that draw on procedures, standards and requirements established for previous therapies are seen as less risky and thus promising clinical targets. Gene editing to treat thalassemia and other blood disorders builds on decades of clinical expertise with the bodys haematopoietic (blood-forming) system, gained by treating leukaemia patients. Even treatments that were not ultimately successful such as foetal stem cell transplants for Parkinsons disease (PD) can provide expertise with clinical trials and regulation to support a next-generation iPSC-based cell therapy for PD.

While the government has rightly been wary of picking winners, as particular niches for early clinical adoption of biomodifying technologies become apparent they may require specific, targeted support, to complement the broader support for the field already provided by polices described above. Innovations in related fields such as biomaterials and automation, potentially supported by the High Value Manufacturing Catapult, are likely to improve manufacturing capacity and speed over time. These innovations may be relatively incremental in the manufacturing phase but could have disruptive effects further down the value chain at the clinical delivery phase, as greater supply makes biomodifying RM therapies accessible to less tightly defined patient cohorts. The next policy challenge will be to provide targeted support for clinical delivery whilst avoiding lock-in to infrastructure or procedures that would inhibit the evolution of the field over time.

The research underpinning this piece was supported by the Economic and Social Research Council grant number ES/P002943/1 and the Leverhulme Trust grant number RPG-2017-330

References

1 Department for Business, Industry and Industrial strategy (2017) Industrial Strategy: building a Britain fit for the future. https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/730048/industrial-strategy-white-paper-web-ready-a4-version.pdf

2 Department for Business, Industry and Industrial strategy (2018) Bioeconomy strategy: 2018 to 2030. https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/761856/181205_BEIS_Growing_the_Bioeconomy__Web_SP_.pdf

3 Open Access Government (2019) The promises and challenges of biomodifying technologies for the UK https://www.openaccessgovernment.org/biomodifying-technologies/68041/

4 Accelerated Access Review (AAR). (2016). Final Report: Review of Innovative Medicines and Medical Technologies. London: The Crown.

5 Joyce Tait & David Wield (2019) Policy support for disruptive innovation in the life sciences, Technology Analysis & Strategic Management, DOI: 10.1080/09537325.2019.1631449

6 Open Access Government (2019) The promises and challenges of biomodifying technologies for the UK https://www.openaccessgovernment.org/biomodifying-technologies/68041/

7 Joyce Tait & David Wield (2019) Policy support for disruptive innovation in the life sciences, Technology Analysis & Strategic Management, DOI: 10.1080/09537325.2019.1631449

Please note: This is a commercial profile

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Targeted policy support for emerging biomedical innovations - Open Access Government

Alzheimer’s: Could Fat Obtained From Liposuction Treat the Disease? – Gilmore Health News

The start-up Cell-Easy and the University Hospital of Toulouse have joined forces to carry out a trial to see if stem cells obtained from liposuction could help treat Alzheimers.

Liposuction Image Courtesy of Michael S. Schwartz, M.D

Alzheimers disease is the leading cause of dementia in the world. It accounts for 60 to 70% of cases. By 2015, 9.9 million people will have been diagnosed, a new case every three seconds according to the WHO. The disease most often manifests itself through memory disorders, and then other brain functions are affected. Gradually, daily tasks become more and more difficult and it becomes almost impossible for patients to adapt to new situations. The disease is currently incurable and, given the number of people affected, researchers are eagerly trying to at least slow down the progression of the disease. The treatment of stem cells to replace damaged cells with healthy ones is one of the therapeutic approaches that is increasingly being used. In France, the university hospital of Toulouse (CHU de Toulouse) declared on June 14 to 20 minutes a French journal that they will soon launch a study based on stem cells obtained by plastic surgeons during liposuction.

Read Also: Exifone Reverses DNA Damage in Mice Suffering From Dementia and Alzheimers in MIT Study

The start-up company Cell-Easy, based in Toulouse and dedicated to the large-scale production of stem cells, is the only structure of its kind in France. It recently obtained an authorization to carry out clinical trials with human stem cells obtained from fat tissue, which is available in almost unlimited quantities in the human body.

The first challenge for Cell-Easy was to reduce the cost of fat stem cell production by a factor of 10 in order to make this therapy available to as many people as possible. We have succeeded in this. Today we can open our pharmaceutical facility. Actually we already started equipping the laboratory. Production will start in about six months. The production capacity of the plant will reach 100,000 doses per year, explains Pierre Monsan, director of Cell-Easy and founder of the French biotechnology association in La Tribune de Toulouse.

We take this waste and turn it into medicine. Although stem cells often come from umbilical cords or painful lumbar punctures, our technology allows us to multiply them, he explained to 20 minutes a French journal.

Read Also: Alzheimers Breakthrough: An Antibody That Can Recognize the Disease Much Earlier Is Now Available

For this reason, the start-up has just signed a cooperation agreement with the University Hospital of Toulouse for a clinical study on Alzheimers disease, which is scheduled to start in early 2021. The researchers will accompany nine patients aged between 50 and 85 years who have just been diagnosed with the disease in the university hospitals of Toulouse and Montpellier.

Although the disease is characterized by chronic inflammation of the brain, which causes protein deposits, the scientists will test the anti-inflammatory effect of stem cells and their ability to slow down the progression of the disease, he continues. Therefore, the participants will be injected with stem cells via the bloodstream. Depending on the initial results, the cohort may then grow to about 50 people.

Read Also: Speaking Multiple Languages Can Delay the Onset of Alzheimers

Medical research into the treatment of Alzheimers disease is making great progress. Recently, scientists have discovered a promising clue to establish a biomarker for the disease in the retina. Indeed, the top layer of neurons in the retina of a mouse model of Alzheimers disease showed a change in its structural texture. Combined with data on changes in the thickness of this layer, the new measurement could be a more readily available biomarker for Alzheimers disease. Early detection of the disease could significantly improve the quality of life of millions of people worldwide.

https://www.20minutes.fr/sante/2799371-20200614-toulouse-quand-liposuccions-debouchent-espoir-traitement-maladie-alzheimer

https://toulouse.latribune.fr/innovation/2020-06-11/cell-easy-devient-le-premier-etablissement-a-produire-des-cellules-souches-en-france-850013.html

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Alzheimer's: Could Fat Obtained From Liposuction Treat the Disease? - Gilmore Health News

Advanced Cell Therapies Market 2020-2026: Analysis by Growth Factors, Key Trends and Competitive Strategies – Cole of Duty

The research study Advanced Cell Therapies market 2020 launched by ABRReports.com provides the detailed analysis of the current market status, investment plans, production and consumption, price trends, and analysis by the market player, by region, by type, by application and etc, and custom research can be added according to specific requirements

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The prime objective of this report is to help the user understand the market in terms of its definition, segmentation, market potential, influential trends, and the challenges that the market is facing with 10 major regions and 50 major countries. Deep researches and analysis were done during the preparation of the report. The readers will find this report very helpful in understanding the market in depth.

The key players covered in the Advanced Cell Therapies Market research report are:By Market Players:FRC Blood ServiceOkyanosAdvanced Cell Technology, IncFujifilmGamida CellVericelBioXcelleratorKolon TissueGeneLocate Bio LimitedRexgeneroTakuraAutolus

By TypeStem Cell TransplantsCAR T-cell Therapy

By ApplicationStem CellRegenerative Medicine

Click to access full report and Table of Content at https://www.abrreports.com/industry-insights/covid-19-impact-on-2020-2026-global-and-regional-advanced-cell-therapies-industry-production-sales-and-consumption-status-and-prospects-professional-market-research-report-standard-version

The data and the information regarding the market are taken from reliable sources such as websites, annual reports of the companies, journals, and others and were checked and validated by the industry experts. The facts and data are represented in the report using diagrams, graphs, pie charts, and other pictorial representations. This enhances the visual representation and also helps in understanding the facts much better.

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Chapter 1 Industry OverviewChapter 2 Global Advanced Cell Therapies Competition by Types, Applications, and Top Regions and CountriesChapter 3 Production Market AnalysisChapter 4 Global Advanced Cell Therapies Sales, Consumption, Export, Import by Regions (2015-2020)Chapter 5 North America Advanced Cell Therapies Market AnalysisChapter 6 East Asia Advanced Cell Therapies Market AnalysisChapter 7 Europe Advanced Cell Therapies Market AnalysisChapter 8 South Asia Advanced Cell Therapies Market AnalysisChapter 9 Southeast Asia Advanced Cell Therapies Market AnalysisChapter 10 Middle East Advanced Cell Therapies Market AnalysisChapter 11 Africa Advanced Cell Therapies Market AnalysisChapter 12 Oceania Advanced Cell Therapies Market AnalysisChapter 13 South America Advanced Cell Therapies Market AnalysisChapter 14 Company Profiles and Key Figures in Advanced Cell Therapies BusinessChapter 15 Global Advanced Cell Therapies Market Forecast (2021-2026)Chapter 16 Conclusions

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Advanced Cell Therapies Market 2020-2026: Analysis by Growth Factors, Key Trends and Competitive Strategies - Cole of Duty

Biotechs Working on COVID 19 – TheBull.com.au

14 June 2020 9min read

On 11 June Aussie investors got a wake-up call from the chairman of the US Federal Reserve, Jerome Powell, sending the ASX 200 plunging 3%, its worst close in six weeks. US markets followed suit, dropping 4.25% by midday on the 11th and closing down 6.9%, dropping 1,861 points. However, although the ASX closed the week lower down 1.89% the buyers returned to US markets on the last day of trading there, sending the Dow Jones Industrial Average up by 1.95%.

With countries all over the world getting back to business and concerns about the spread of COVID-19 fading from the public consciousness in general and investor consciousness in particular, global share markets remained confident in anticipation of a quick v-shaped recovery.

Powell threw water on that argument, in the official statement released by the Fed:

Skeptical investors may have noticed the tendency of market participants to accentuate whatever bits of positive news comes out, often ignoring negative undertows. Reaction to this announcement reversed the trend, grabbing on to the phrases in the statement ongoing public health crisis and medium term economic outlook despite statements the US economy would recover eventually.

The Chairman clearly linked the economic recovery to the coronavirus, citing issues like the length of time for treatments and a virus as well as rises in the number of infected as social distancing practices fall by the wayside.

The key point investors need to bring into focus is that despite declining cases in most countries and the accompanying willingness of the public to ignore warnings, COVID-19 has not gone away, even here in Australia.

While the numbers by state might be cause for optimism in most of the country, the push for a full reopening of Australian businesses and the easing of travel restrictions suggest at the very least the possibility of increasing cases here.

In the US 16 states are seeing increases in COVID-19 cases in the double digits, raising the alarm among some in that country that the dreaded second wave is soon at hand.

With COVID-19 still very a factor looming over economic recoveries everywhere, biotech stocks involved in any way in the search for a vaccine or treatments for the virus could prosper in the medium term if successful.

The ASX has one of its flagship stocks, CSL Limited (CSL) in a position to increase its already stellar reputation through its partnership role in the CoVIg-19 Plasma Alliance, comprised of leading experts from the industry. In addition, CSL is developing COVID-19 Immunoglobulin, a plasma product with the potential to treat people in the Australian market with respiratory complications due to COVID-19.

There are a number of small cap biotechs that merit attention, along with long time large cap heart breakers Mesoblast Limited (MSB). The following table lists them by market cap, along with price information.

Four of these stocks have turned in stellar year over year share price appreciation for their investors.

Mesoblast is involved in regenerative medicine with its three proprietary technologies Mesenchymal Precursor Cells (MPCs), culture-expanded Mesenchymal Stem Cells (MSCs), Dental Pulp Stem Cells (DPSCs) and expanded Hematopoietic Stem Cells (HSCs).

These technology platforms address complex medical issues involving inflammation and damaged tissue. The company has five treatments in various stages of clinical trials, with one Ryoncil approaching registration status with a launch in the US on the horizon. Ryoncil, or Remestemcel-L, treats inflammatory conditions of the kind associated with COVID-19 acute respiratory distress stress syndrome (ARDS). After a successful pilot trial the US FDA (Food and Drug Administration) cleared Remestemcel-L for a randomized controlled Phase 3 Trial involving 300 patients in 30 different sites across the US. Confident of a successful trial, Mesoblast has already concluded a capital raise totaling $138 million dollars to go towards the eventual manufacture of the treatment.

Atomo Diagnostics (AT1) is virtually a newborn on the ASX, listing on 30 April of 2020 with a first day closing price of $0.48, declining steadily to reach the current $0.30 per share. The company entered the ASX with both a professional use rapid diagnostic test (RDT) and a self-testing device for the HIV virus. Atomo, working with French in vitro diagnostic specialist NG Biotech SAS, has developed an RDT platform for professionals to test for the presence of antibodies in response to COVID-19. NG Biotech has granted exclusive distribution rights to Atomo for Australia, New Zealand, and other South East Asian countries, following NGs purchase of 1.5 million tests for use in France.

Dimerix Limited (DXB) got a huge boost on 4 June when the stock price jumped 54% with the announcement its lead candidate to treat kidney disease DMX-200 had been selected to join a global study (REMAP-CAP) on treatments for COVID-19 induced ARDS. The study is funded by both government and non-government organisations with an endorsement from the WHO (World Health Organisation).

The company listed on the ASX in 2015 following a reverse merger. Dimerix has another DMX-200 treatment for kidney scarring in clinical trials and another DMX-700 for COPD (chronic obstructive pulmonary disease) starting pre-clinical studies.

Cynata Therapeutics (CYP) is developing a platform using Cymerus technology for manufacturing mesenchymal stem cells (MSCs) for therapeutic use. The technology could be disruptive in that it could allow manufacture of stem cells for therapeutic use in commercial scale quantities from a single donation of a single cell.

The company has completed successful preclinical studies targeting different diseases, including asthma, heart attack, sepsis, and cytokine release syndrome (an inflammatory condition with infections.) FUJIFILM Corporation has licensed the technology from Cynata to treat graft-versus-host disease in people following a successful Phase 1 clinical trial.

On 11 March of 2020, the company announced it was in active discussions with international pharmaceutical companies to use the Cymerus technology platform to treat COVID-19 patients with severe symptoms.

On 8 May Cynata announced approval to begin a clinical trial in Australia to determine the efficacy of its stem cells from its Cymerus technology platform to treat ICU (intensive care unit) COVID-19 patients.

Impression Healthcare (IHL) is a custom fit dental products company that got involved in medical marijuana, with clinical trials for the treatment of gingivitis and gum disease. On 1 May the company announced a preclinical animal study to test its cannabinoid drug IHL-675A for treating a leading cause of deaths from COVIOD-19 sepsis-related ARDS. The trial began on 2 June. On 3 June Impression announced it was abandoning its oral device business to focus on medical marijuana in anticipation of significantly higher revenue for the June Quarter for its existing Icannex branded cannabinoids.

PharmAust Limited (PAA) has a business model aimed at a newer approach to drug development repurposing drugs approved for other treatment conditions. In the past, discoveries that a given drug to treat condition X could also treat condition Y were accidental. PharmAusts lead drug Monepantel (MPL) is a cancer treatment that has successfully completed Phase 1 clinical trials for use in both humans and dogs. The treatment for dogs is starting Phase 2 trials. The company also has a portfolio of cancer treatments in preclinical stages.

On 4 June, the company announced it had expanded its portfolio enhancement efforts to include a preliminary study on the effects of Monepantel on COVID-19 patients. Following positive results potential suppression of the virus by up to 95% PharmAust filed a patent application for the use of Monepantel in COVID-19 cases. The share price went up 40% on the news.

In yet another example of an ASX small cap biotech discovering treatments developed for other purposes could help in treating COVID-19, Noxopharm Limited (NOX) announced on 1 April it too was ready to get into the race to find effective treatments.

The companys lead candidate is a late stage cancer treatment primarily targeting prostate cancer idronoxil with the brand name Veyonda. Examining their preclinical work on the drug, Noxopharm found idronoxil suppressed the cytokine molecules responsible for triggering inflammation of the type affecting the respiratory systems of COVID-19 patients.

On 21 April, the company announced it was applying for US FDA (food and drug administration) approval to conduct a clinical study for Veyonda.

All of the companies listed here are developing treatments that may fade away at some time in the unknown future. Interested investors should also consider the potential of the companys other offerings as even with success in the treatments developed, their revenue generating potential could be short-lived.

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Biotechs Working on COVID 19 - TheBull.com.au

Global Regenerative Medicine Market By Type, Therapy, Application and Forecast Report by 2030 – Cole of Duty

The increasing incidence of genetic disorders and chronic diseases is one of the biggest factors responsible for the burgeoning sales of regenerative medicine throughout the globe. The rising adoption of sedentary lifestyles and unhealthy dietary habits of the people all around the world are the main reasons causing the high prevalence of chronic diseases across the world. According to the World Health Organization (WHO), almost 17.9 million deaths are recorded every year because of cardiovascular diseases. Moreover, the cardiovascular diseases account for nearly 31% of the total deaths occurring across the world every year.

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The other major factors propelling the demand for regenerative medicine are the soaring investments being made by the governments of many countries in research and development activities in the domain of regenerative medicine, surging number of regenerative medicine companies throughout the world, and rapid technological developments in tissue engineering areas and stem cell research. Due to these factors, the global regenerative medicine market is expected to exhibit huge expansion over the coming years.

Across the globe, the regenerative medicine market is predicted to record the fastest growth in the Asia-Pacific (APAC) region in the upcoming years. This is mainly credited to the improving healthcare facilities and infrastructure in the region and the subsequent rise in stem cell research in the developing nations of APAC. For instance, the Chinese government has recently approved R&D activities pertaining to the human embryonic stem cells, which has in turn, encouraged more research on the clinical potential of the stem cells in the country.

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Global Regenerative Medicine Market By Type, Therapy, Application and Forecast Report by 2030 - Cole of Duty

New CRISPR, gene therapy results strengthen potential for treatment of blood diseases – BioPharma Dive

Three people with the inherited blood diseases sickle cell and beta thalassemia remain free of burdensome blood transfusions and their worst symptoms, months after receiving an infusion of genetically modified stem cells.

One of the three, a young woman with a severe form of beta thalassemia, has now been followed for over a year since she was treated, while the second, a woman in her 30s with sickle cell disease, is more than nine months removed from her infusion. They are the first two patients in pioneering studies of a therapy, developed by CRISPR Therapeutics and Vertex, that's based on the gene editing technology known as CRISPR.

Both patients continue to respond to treatment, bolstering evidence of genetic medicine's potential to permanently alter the course of devastating hereditary conditions like sickle cell and transfusion-dependent beta thalassemia. A gene therapy developed by Bluebird Bio has shown similar potential.

First results from the two studies, disclosed last November, were "taking the promise of CRISPR and turning that into a reality," said Samarth Kulkarni, CRISPR Therapeutics' CEO, in an interview. The additional data and follow-up now available "show these effects can be long-lasting and durable."

And in beta thalassemia, the first patient's experience is now supported by results from another patient who was treated about five months ago. This individual has also been able to stop receiving blood transfusions.

Taken together, the two patients responses are "proof of concept," CRISPR Therapeutics and Vertex claim, that their approach to treating beta thalassemia has the potential to be curative.

In sickle cell, the companies are also hopeful. The one patient for whom they have data has not had a vaso-occlusive crisis, a painful episode caused by the disease's characteristic sickling of red blood cells, since her treatment.

"The clinical manifestation of the disease is different, but we see consistent outcomes across both diseases," said Bastiano Sanna, Vertex's head of cell and genetic therapies, in an interview.

Three other beta thalassemia patients and one other sickle cell disease patient have been treated in the two studies of CRISPR Therapeutics and Vertex's therapy, dubbed CTX001. If results continue to look positive, CTX001 could be another powerful way to help people for whom treatment options have long been limited.

CRISPR, an easy-to-use method of genetic surgery that's derived from a bacterial defense system, has become a mainstay in labs across the world for all types of experiments. Its potential use as a human therapeutic has drawn closer as companies harnessing the technology CRISPR Therapeutics, Editas Medicine and Intellia Therapeutics have advanced their research. CRISPR Therapeutics is the first of the three to deliver results from a clinical trial.

CRISPR and Vertex unveiled their updated results at the European Hematology Association's virtual meeting on Friday. Also being presented were the latest data from Bluebird's gene therapy, known as LentiGlobin.

Bluebird is much further along, having treated 60 patients with beta thalassemia and 37 with sickle cell disease across six different studies.

Updated results from three of those studies showed 23 of 27 evaluable patients with beta thalassemia were transfusion independent for at least a year following treatment. And in sickle cell, no serious vaso-occlusive crises were observed in the 18 patients who had at least six months of follow-up. An episode was previously reported in one patient several months after LentiGlobin treatment, but was judged to be non-serious.

One sickle cell patient died suddenly 20 months following infusion with LentiGlobin, Bluebird reported Friday. Both the treating physician and an independent study committee concluded the death, ruled to be cardiovascular in nature, was unlikely to be related to the gene therapy.

Both beta thalassemia and sickle cell are diseases caused by mutations in the beta globin gene, faulty DNA that results in either absent or warped hemoglobin. Without enough hemoglobin, patients' red blood cells can't carry needed oxygen throughout the body. And those with sickle cell have abnormal hemoglobin that makes red blood cells fragile and stiff, causing them to stick in blood vessels.

Both diseases require chronic blood transfusions, and can lead to organ damage and reduced lifespans. Treatment options are limited, although that's now changing. The Food and Drug Administration, over the past few years, has approved Reblozyl, for beta thalassemia, and Oxbryta and Adakveo, for sickle cell.

Adakveo reduces the frequency of vaso-occlusive crises, while Reblozyl and Oxbryta are chronic medicines meant to boost patients' hemoglobin levels.

CRISPR Therapeutics and Vertex, along with Bluebird, are trying to accomplish the same goal but in more dramatic fashion: raising hemoglobin levels high enough so patients can stop blood transfusions and, in sickle cell, avoid pain crises altogether.

CRISPR and Vertex use CRISPR/cas9 gene editing to modify the DNA of stem cells extracted from a patient's bone marrow. The cells are engineered to produce a type of hemoglobin that's present at birth but normally replaced soon after. Once returned to the body and engrafted in the bone marrow, these CRISPR'd cells substitute this so-called fetal hemoglobin for the missing adult hemoglobin.

In the three patients treated so far, that appears to be what's happened. Both beta thalassemia patients are producing hemoglobin at levels considered normal. The sickle cell patient now has enough fetal hemoglobin to dilute the effects of sickled hemoglobin, potentially helping to preserve red blood cells.

Crucially, CRISPR and Vertex shared data for the first time indicating a high percentage of edited cells are present in each patient's bone marrow, supporting their confidence that the effects of treatment might last.

Bluebird, by contrast, doesn't edit the DNA of extracted stem cells, but rather inserts a modified gene into those cells. Once infused and engrafted in a patient, the cells can produce gene therapy-derived hemoglobin.

In most beta thalassemia and sickle cell patients treated with Bluebird's LentiGlobin, hemoglobin levels rose to normal or near-normal levels.

LentiGlobin is already approved for certain beta thalassemia patients in Europe as Zynteglo. In the U.S., Bluebird has hit delays and pushed back when it expects to submit an application to the middle of next year. A filing for an accelerated approval in sickle cell would likely follow sometime in the second half of 2021.

CRISPR and Vertex, meanwhile, plan to enroll more patients into their two studies, which they hope could serve as sufficient for an approval application if positive, Kulkarni said.

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New CRISPR, gene therapy results strengthen potential for treatment of blood diseases - BioPharma Dive

CAG Regimen Shows Strong Results as Salvage Therapy in T-ALL – AJMC.com Managed Markets Network

Patients with relapsed/refractory T-cell lymphoblastic leukemia face poor outcomes, and are generally treated by salvage therapy followed by allogeneic hematopoietic stem cell transplant. A new study suggests an optimal option for salvage therapy.

Investigators in China say the cytarabine, aclarubicin, and G-SCF (CAG) regimen is highly effective and safe in patients with R/R-T-ALL, confirming the experience of a single cancer centers smaller study. The new research may help build consensus in a therapeutic area in which patients face stiff odds and no single salvage therapy has emerged as the standard of care.

Writing in the journal Cancer Medicine,1 a team of investigators including Hong-Hu Zhu, MD, PhD, of the First Affiliated Hospital of Zhejiang University, in China, notes that patients who are newly diagnosed with T-ALL have been treated more successfully in recent years, but survival among patients with relapsed or refractory T-ALL face long-term survival rates of less than 10%.

The only curable treatment is initiating a salvage regimen to achieve complete remission (CR) and then rapidly performing allogeneic hematopoietic stem cell transplantation (allo-HSCT), the authors write. Therefore, selecting an effective salvage regimen is vital for R/R-T-ALL.

Thats where the trouble comes in, though. Zhu and colleagues note that although a number of regimens are being used, there is no consensus on the optimal therapy. Back in 2008, a team of Chinese investigators published findings2 showing promising results with the CAG regimen, but the study size was small.

In an effort to better evaluate the regimen, Zhu and his team constructed a 6-center retrospective analysis of patients with R/R-T-ALL who were at least 16 years old and who underwent the CAG regimen as salvage therapy between 2012 and 2019. The team measured complete remission (CR), partial response (PR), overall survival (OS), and event-free survival (EFS).

Out of a total of 41 patients, 33 achieved CR after one cycle of the CAG regimen, and 2 more patients achieved PR. Six patients failed to respond. Early T-cell precursor status did not affect CR rates, which were around 80% for both ETP and non-ETP patients.

Of the 41 patients, 27 underwent successful allo-HSCT, the majority of whom (22) were in CR. After 2 years, OS was estimated at 68.8% and EFS was estimated at 56.5%.

Our multicenter results show that the CAG regimen is associated with a high CR rate of 80.5% and is well-tolerated, enabling most patients to bridge to allo-HSCT, Zhu and colleagues report. Thus, this regimen represents a novel option for adult R/R-T-ALL patients.

Zhu and colleagues said the most surprising finding was the very high overall response rate of 85.4%. Furthermore, the fact that 66% of patients were able to bridge to allo-HSCT was highly encouraging, they said. The investigators note that the regimen is most commonly used in China for the treatment of acute myeloid leukemia (AML).

The CAG regimen seems to have low rates of hematological and non-hematological adverse events, something found in the current study but also in use in AML.

The authors note that the cancer is so rare that it will be difficult to study in much larger trials, though they are currently planning a study that will include 18 cancer centers in China to validate the results. In the meantime, they write that the regimen represents a ray of hope in a difficult disease category.

The CAG regimen enabled most patients to bridge to allo-HSCT and achieve improved outcomes; thus, it represents a novel option for this population with poor prognosis, they conclude.

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CAG Regimen Shows Strong Results as Salvage Therapy in T-ALL - AJMC.com Managed Markets Network