Resilience Hope and Being a Survivor – Curetoday.com

When I first heard the word "resilience" I did not really understand what it meant, I was listening to a presentation about a third world mission trip and about how people survive their hardships there. But now, I personally understand the meaning and am ready to share my journey.

When my husband was diagnosed with cancer, I understood what may be ahead, since I am a nurse with many years of experience. The first two years of his journey were stressful, but we had each other, enjoyed our life and worked through the decisions and experience. The turning point was when he his cancer returned, and he started radiation therapy. Then, we hit what felt like a big wall.

I was diagnosed with breast cancer.

I remember sharing this news with him and he stated, "I am stronger than you think" and he proved that to be so true over the next 3 years.

Our journey together is a story of resilience, sharing hope and caring for each other. His cancer involved traveling over 3 hours to a medical center for surgery, needing an artificial airway and nutrition via a feeding tube. We spent our time together going to the local cancer center on separate days for each of our own chemotherapy treatments. As a nurse, I cared for him even during my own radiation treatment period and still kept working at my job as much as possible.

During that time, I kept thinking, is this what it takes to have that resilience? Is resilience what it takes to keep caring, maintaining hope and surviving a day at a time. If you look up the word, " resilience", there are terms like hardiness, strength and toughness. However, I have learned that it means much more.

The experience of a cancer journey together is more than what those words convey. I would often say to myself, " I am stronger than I thought too" not realizing I was beginning to understand resilience. When looking inward, you begin to understand the human spirit and what we face in life cannot truly be explained to others, but only matures every day with every hurdle we tackle and overcome.

I am a widow now and a cancer survivor, who looks back on that time in our lives, with joy of being together, with pride at how we both endured and how much stronger we became because of it. Hope was always in front of us, leading us through each day.

You should never lose hope, because that optimism is what builds the courage that leads to learning what resilience really means.

Read more:

Resilience Hope and Being a Survivor - Curetoday.com

Read more
Pregnant and positive | Scientists look for COVID-19 immunity in the womb – WHAS11.com

PENNSYLVANIA, USA Brianna Boriosi, 30, and her husband Marc both are considered essential employees because they work in inpatient drug and alcohol treatment services. They are now self-quarantining at home because they both tested positive for COVID-19.

What makes their case more complicated and worrisome is that Brianna is 7-and-a-half months pregnant, due June 24. Of course she immediately called her doctors.

They told me I was their first positive pregnancy case with COVID-19 in their practice, Brianna said.

Almost immediately after she found out she was positive she was getting calls from the Pennsylvania Department of Health.They wanted to know what the circumstances were leading up to getting infected, but also they hoped Brianna would give consent for test samples.

Once the babys born, they talked about maybe taking some of the umbilical cord or the blood in there, Brianna said. As well as the placenta, and if Im willing, looking into possibly donating stem cells.

It didnt take long for her to say yes.

There was a possibility that the baby will either be immune or have some of these antibodies.

While doctors believe the virus will not get passed onto her unborn child, scientists hope what they find through tests can help them to get to a vaccine and the cure.

Anything I can do to help or donate or whatever, Im definitely all about it.

Her positive test could lead to keeping everyone negative for the virus in the future. Brianna wants that for their baby as well as their two-year-old son.

Because both Brianna and Marc are positive, doctors presume their son is as well. The couple is thankful that he hasnt shown any symptoms, and they hope and pray it stays that way.

RELATED: Feds to track, publicly share info on nursing home COVID-19 outbreaks

RELATED: Rise in accidental poisonings coincides with coronavirus lockdowns

RELATED: Reports suggest many may have had coronavirus with no symptoms

Contact reporter John Charlton atjcharlton@whas11.com. Follow him onTwitter (@JCharltonNews) andFacebook.

See the rest here:

Pregnant and positive | Scientists look for COVID-19 immunity in the womb - WHAS11.com

Read more
NovelStem Announces New Research Collaborations for Stem-Cell Technology Platform in Cancer Immunotherapy and COVID-19 Resistance; Reports Progress…

BOCA RATON, Fla. and JERUSALEM, Israel, April 21, 2020 (GLOBE NEWSWIRE) -- NovelStem International Corp. (OTC Pink: NSTM), a biotechnology company focused on its stem cell-based technology platform, developed by Israel-based affiliate, NewStem Ltd., announced two new collaborations to apply NewStems technology platform, one to research a potential cancer immunotherapy drug and another to research genes responsible for COVID-19. NovelStem also announced the identification and completion of analysis of resistance to a dozen standard-of-care anti-cancer treatments.

NovelStem CEO, Jan Loeb, added, We are very impressed with the pace and expanding scope of NewStems development work. They recently completed analysis for a dozen chemotherapy resistance diagnostics, while also expanding their work to include an externally funded drug development research project and a genetic research collaboration around COVID-19. This work underscores the substantial potential value of NewStems unique platform to accelerate genetic research, as compared to other approaches, and deliver improved patient care.

NewStem, a spinoff of Yissum, The Hebrew University of Jerusalems technology-transfer company, is 27.3% owned by NovelStem. NewStems diagnostic solutions are based on the research of specialized stem cells that carry just one set of chromosomes (haploid cells) by Professor Nissim Benvenisty, Director of the Azrieli Center for Stem Cells and Genetic Research at the Hebrew University. NewStem holds intellectual property rights related to stem cells, including genome-wide screening methodologies.

NewStem CEO, Ayelet Dilion-Mashiah, said, While NewStem remains focused principally on its genetic-driven personalized diagnostic and therapy for cancer patients, we are excited to expand the application of our novel technology platform to support other drug development programs and revenue streams, including for novel diseases such as COVID-19.

COVID-19 Research CollaborationSevere acute respiratory syndrome coronavirus 2 (SARS-CoV-2) is the virus strain that causes the pandemic coronavirus disease 2019 (COVID-19), a respiratory illness. Under a collaboration with the Azrieli Center for Stem Cells and Genetic Research at the Hebrew University, using NewStems haploid human embryonic stem cell platform technology and genome-wide screening methodologies, research was initiated for identifying genes that regulate the pathogenicity of the SARS-CoV-2 virus. This research is intended to help develop methods to generate resistance to SARS-CoV-2 and enable new ways to find therapies for the devastating disease.

Cancer Immunotherapy CollaborationA leading NASDAQ biopharma company has executed a collaboration agreement with NewStem to fund research utilizing NewStems technology platform. The research is to support the development of a potential new biopharmaceutical in the field of cancer immunotherapy as well as to pursue the potential discovery of new drug targets. The project is expected to take approximately 12 months. Should the research progress to support a successful immunotherapy, NewStem would be entitled to further milestone payments as well as royalties based on sales.

Cancer Treatment Resistance Diagnostics ProgramDrug resistance in tumors is a major cause of cancer treatment failure, yet in nearly 50% of cancer cases this resistance is recognized only after the completion of the first course of treatment. NewStems diagnostic technology can predict patients resistance to anti-cancer drugs prior to treatment, potentially allowing for better, targeted cancer treatments and related cost benefits.

NewStem is advancing its specialized human stem cell-based approach for predicting patients resistance to cancer therapy, allowing for better, targeted personal-oncology treatments with the potential to significantly reduce incidents of anti-cancer drug resistance. NewStem has now completed the screening of resistance diagnostics for a dozen standard-of-care cancer treatments, up from five treatments in mid-2019. The molecules screened represent a significant portion of treatment protocols for cancer cases.

NewStem is validating the performance of its screening results in conjunction with retrospective genetic and clinical data of patients previously treated with anti-cancer drugs. The testing for the chosen drug and indications is expected to be finalized within 6 months and then be followed by the regulatory clearance process.

Story continues

About NovelStem International Corp. http://www.novelstem.comNovelStem has a 27.3% stake in NewStem Ltd. which is advancing its novel stem-cell-based diagnostic technology for predicting patients resistance to cancer therapies, allowing for better, targeted cancer treatments with the potential to reduce incidents of drug resistance. The technology is also being used for genetic research related to other medical therapies. NovelStem will increase its ownership to 33% based upon completion of an additional $1 million investment. NovelStem also owns 50% in Netco Partners, which owns the Net Force publishing franchise.

About NewStem Ltd.NewStem Ltd. is advancing novel stem-cell-based technology utilized for the development of new diagnostics and therapeutics. The most advanced product of NewStem is a diagnostic predicting patients resistance to cancer therapy, allowing for better, targeted personal-oncology treatments with the potential to reduce incidents of anti-cancer drug resistance. NewStem is a spinoff of Yissum, The Hebrew University of Jerusalems technology-transfer company. NewStems diagnostic solutions are based on the research of human haploid pluripotent stem cells (hHPSCs) by Professor Nissim Benvenisty, Director of the Azrieli Center for Stem Cells and Genetic Research at the Hebrew University. NewStem holds the intellectual property, reagents and experience required for hHPSC isolation, differentiation, genetic manipulation, immunogenicity and tumorigenicity.

Forward-Looking Statements Statements in this press release and its hyperlinks may be forward-looking statements within the meaning of federal securities laws. The matters discussed herein that are forward-looking statements are based on current board and management expectations that involve risks and uncertainties that may result in such expectations not being realized. Actual outcomes and results may differ materially from what is expressed or forecasted in such forward-looking statements due to numerous potential risks and uncertainties, including, but not limited to, the success of NewStems research and future commercialization of its diagnostics utilizing human haploid pluripotent stem cells, competition in the area of genetic diagnostics, the ability to retain key personnel involved in research and development, the ability to secure appropriate regulatory approvals, and the ability to fund future investment in NewStem. Such forward-looking statements speak only as of the date on which they are made.

NovelStem Investor RelationsBill Jones or David Collins Catalyst IR(212) 924-9800NSTM@catalyst-ir.com

Link:

NovelStem Announces New Research Collaborations for Stem-Cell Technology Platform in Cancer Immunotherapy and COVID-19 Resistance; Reports Progress...

Read more
Planned Clinical Trial of Allogeneic Stem Cell Therapy Remestemcel-L in Patients with COVID-19 – Cancer Network

Mount Sinai Health System announced that they will be using remestemcel-L (Ryoncil), an innovative allogeneic stem cell therapy, in patients with coronavirus disease 2019 (COVID-19).

Additionally, Mount Sinai indicated that they will play a central role in a clinical trial for patients with severe acute respiratory distress syndrome, which affects individuals with severe cases of COVID-19.

Remestemcel-L has previously been tested in patients who have had a bone marrow transplant, who can experience an overactive immune response similar to that observed in severe cases of COVID-19.

Mount Sinai began administering remestemcel-L to patients in late March under the FDAs compassionate use program. The therapy was given to 10 patients with moderate to severe cases of COVID-19-related acute respiratory distress syndrome (ARDS), most of whom were on ventilators, and the doctors saw encouraging results.

We are encouraged by what we have seen so far and look forward to participating in the randomized controlled trial starting soon that would better indicate whether this is an effective therapy for patients in severe respiratory distress from COVID-19, Keren Osman, MD, medical director of the Cellular Therapy Service in the Bone Marrow and Stem Cell Transplantation Program at The Tisch Cancer Institute at Mount Sinai and associate professor of Hematology and Medical Oncology at the Icahn School of Medicine at Mount Sinai, said in a press release.

The randomized clinical trial evaluating the therapeutic benefit and safety of remestemcel-L will be conducted at Mount Sinai, which will serve as the clinical and data coordinating center. The stem cell therapy will be evaluated in 240 patients with COVID-19-related ARDS in the US and Canada. Moreover, the trial will be conducted as a public-private partnership between the Cardiothoracic Surgical Trials Network.

The coronavirus pandemic has caused exponential increases of people suffering with acute respiratory distress syndrome, requiring intubation and mechanical ventilation with many dying, Annetine Gelijns, PhD, the Edmond A. Guggenheim Professor of Health Policy at the Icahn School of Medicine at Mount Sinai, said in a press release. We have designed a clinical trial that will expeditiously determine whether the stem cell therapy will offer a life-saving therapy for a group of patients with a dismal prognosis.

Remestemcel-L consists of mesenchymal stem cells. The therapy was previously assessed in a phase III trial in children who had graft-versus-host disease (GVHD), which can occur after bone marrow transplants. Further, the inflammation that occurs in GVHD is the result of a cytokine storm. A similar cytokine storm has been found to take place in patients with COVID-19 who develop acute respiratory distress syndrome.

These stem cells have shown excellent response rates in severe graft-versus-host disease in children, John Levine, MD, professor of Hematology, Medical Oncology, and Pediatrics at the Icahn School of Medicine at Mount Sinai, who is also the co-director of the Mount Sinai Acute GVHD International Consortium (MAGIC), said in a press release. Mesenchymal stem cells have a natural property that dampens excessive immune responses.

Some institutions have also begun testing anti-IL-6 agents, such as tocilizumab (Actemra), for the treatment of cytokine release syndrome in patients with COVID-19 who develop acute respiratory distress syndrome.

Reference:

Mount Sinai Leading the Way in Innovative Stem Cell Therapy for COVID-19 Patients [news release]. New York, NY. Published April 9, 2020. newswise.com/coronavirus/mount-sinai-leading-the-way-in-innovative-stem-cell-therapy-for-covid-19-patients/?article_id=729684&sc=dwhr&xy=10019792. Accessed April 15, 2020.

Link:

Planned Clinical Trial of Allogeneic Stem Cell Therapy Remestemcel-L in Patients with COVID-19 - Cancer Network

Read more
‘Stem cell therapy more effective on Covid-19’ – Korea Biomedical Review – Korea Biomedical Review

I dont know why people pay attention only to vaccines and treatments against the new coronavirus. Stem cell therapies are more useful to treat Covid-19.

So claimed Lee Hee-young, president of the Korean Association of Stemcell Therapy, at a news conference in Seoul, Monday. He called for active use of stem cell therapies to treat Covid-19 patients.

Several studies have proved the effects of autologous stem cells in treating acute respiratory distress syndrome (ARDS), which is the leading cause of death in Covid-19 patients, Lee said. The concept of stem cell therapy is the same as that of blood transfusion or bone marrow transplantation. Decades of cell therapies have proved that stem cell therapy is safe.

While the development of a treatment or a vaccine against Covid-19 takes a long time and it may not be able to treat patients immediately because of virus mutation possibilities, stem cell therapies can restore damaged lungs directly, Lee claimed.

It is more important to restore damaged lungs than to fight the virus. Stem cell therapy restores the lungs, giving patients time to beat the virus, he went on to say. However, people are paying attention to vaccine or treatment candidates only. This is why I am holding a news conference.

Lee pointed out that the local environment makes it difficult to use stem cell therapies. Thus, the government should ease regulations on the management and use of cell culture facilities so that doctors can perform stem cell therapies with simple cell culture, he said.

As long as physicians have a positive pressure facility and a culture kit, they can separate and culture cells with simple training, he said. If the authorities allow doctors to perform stem cell therapies with a disposable mobile culture autonomously, the cost of stem cell therapies will go down significantly.

Lee added that he asked related officials to include such rules in the Act on Safety and Support for Advanced Regenerative Medicine and Advanced Biopharmaceuticals, which is to take effect in the second half of the year.

same@docdocdoc.co.kr

< Korea Biomedical Review, All rights reserved.>

Original post:

'Stem cell therapy more effective on Covid-19' - Korea Biomedical Review - Korea Biomedical Review

Read more
Mustang Bio Receives Advanced Therapy Medicinal Product Classification from European Medicines Agency for MB-107 Lentiviral Gene Therapy for X-Linked…

NEW YORK, April 20, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the European Medicines Agency (EMA) has granted Advanced Therapy Medicinal Product (ATMP) classification to MB-107, Mustangs lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. The U.S. Food and Drug Administration (FDA) previously granted Regenerative Medicine Advanced Therapy (RMAT) designation to MB-107 for the treatment of XSCID in August 2019.

EMA grants ATMP classifications to new therapeutics that are based on genes or cells and intended as long-term or permanent therapeutic solutions to acute or chronic human diseases at a genetic, cellular or tissue level. The ATMP program provides specific regulatory guidelines for preclinical development, manufacturing and product quality testing of ATMPs and offers incentives, including fee reductions for regulatory advice, recommendations and evaluation and certification of quality and non-clinical data.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are extremely encouraged that the EMA has granted MB-107 with ATMP classification, an important step in establishing our path to market approval and commercialization in Europe. This classification complements the RMAT designation we received last year from the FDA and brings us closer to realizing our goal of commercializing MB-107 for XSCID patients, as these patients are in desperate need of innovative and potentially curative treatment options.

MB-107 is currently being assessed in two Phase 1/2 clinical trials for XSCID: the first in newly diagnosed infants under the age of two at St. Jude Childrens Research Hospital (St. Jude), UCSF Benioff Childrens Hospital in San Francisco and Seattle Childrens Hospital and the second in patients over the age of two who have received prior hematopoietic stem cell transplantation at the National Institutes of Health. Under a licensing partnership with St. Jude, Mustang intends to develop the lentiviral gene therapy for commercial use as MB-107.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

Read the original:

Mustang Bio Receives Advanced Therapy Medicinal Product Classification from European Medicines Agency for MB-107 Lentiviral Gene Therapy for X-Linked...

Read more
Athersys: Abundance Of Opportunity – Seeking Alpha

Athersys is a late-stage biotechnology company developing their stem cell product MultiStem for multiple critical care indications. At present, they have two programs in late-stage development, ischemic stroke and acute respiratory distress syndrome (ARDS). Their partner in Japan, Healios, is also concurrently evaluating MultiStem for the same two indications. Additionally, they have multiple mid-stage and pre-clinical programs currently in development. Their product MultiStem, is an allogeneic, off-the-shelf cellular therapy derived from the bone marrow of healthy, consenting adults. MultiStem is highly scalable and can produce vast quantities of doses from a single donor bank. In this article, I will address the major potential upcoming catalysts and opportunities for Athersys and its partner Healios, as well as the value proposition for investors.

ARDS Program

The first program I will address is the ARDS program. In 2016, Athersys initiated a phase 1/2a exploratory study (MUST-ARDS) to assess its MultiStem product candidate in patients with moderate to severe ARDS for the possibility of clinical benefit. This study completed enrollment in 2018, and promising results were announced in January 2019 here. The safety-focused primary outcome measures, taken from clinicaltrials.gov were frequency of sustained hypoxemia or hypotension within 4 hours and suspected unexpected serious adverse reactions (SUSARs) within 24 hours.

This slide from Athersys' latest company presentation clearly addresses the primary endpoints:

Source: Athersys April 2020 corporate presentation here

Though this important study was not powered for efficacy outcomes, it revealed several key positive trends, including more ventilator-free days, more ICU-free days, and a lower mortality rate among those patients receiving MultiStem than those patients receiving placebo. Athersys' ARDS program subsequently received Fast Track designation from the FDA and was added as a late-breaking presentation at the American Thoracic Society International Conference in May 2019. It is important to note that Athersys ARDS program is the only current one with FDA Fast Track designation. A video clip of MUST-ARDS study primary investigator Dr. Geoff Bellingan can see seen here, as he discusses Multistem cell therapy for ARDS at the conference.

In 2018, Athersys also expanded an existing collaboration here with its Japan partner Healios, adding the ARDS indication to the original agreement here from 2016 that included ischemic stroke. The partnership gives commercialization rights to Healios in Japan for certain indications in exchange for potential aggregate development, regulatory filing, and commercialization milestone payments, as well as sales royalties to Athersys. I will expand on these payments as potential near to mid-term financial catalysts further in the article.

After the collaboration expansion, Healios subsequently initiated an open-label, phase 2 ARDS trial (ONE-BRIDGE) in Japan to evaluate MultiStem in patients who have developed pneumonia-induced ARDS, one of the most prevalent causes of ARDS. The primary endpoint of the trial is number of ventilator-free days out of the first 28 days after MultiStem administration. Additionally, Healios has obtained Orphan Regenerative Medicine Designation in Japan for this program here, which will enable them to receive a priority review upon submission of a marketing application. Post-hoc analysis from Athersys phase 1/2a exploratory trial showed promising results in this sub-population of ARDS patients, as depicted in this slide from Athersys April 2020 company presentation.

Source: Athersys April 2020 corporate presentation here

ARDS takes on a whole new level of importance right now as the world is struggling to control the coronavirus (COVID-19) pandemic. One of the leading causes of death from coronavirus is complications from pneumonia-induced ARDS. This puts Athersys and its partner Healios in a unique and favorable position to quickly address this area of unmet need. Athersys recently announced a large, multicenter phase 2/3 pivotal clinical trial to treat patients with moderate to severe COVID-19 induced ARDS here. This important study, known as the MACOVIA (MultiStem Administration for COVID-19 Induced ARDS) trial, will evaluate approximately 400 patients with moderate to severe ARDS induced by the novel coronavirus disease (COVID-19) and is expected to begin enrolling patients in Q2 2020. Athersys Chairman and CEO Gil Van Bokkelen speaks about their ARDS program in this interview with Fox News.

Per this Form 8-K filing with the SEC, Athersys is currently in discussions with the Biomedical Advanced Research and Development Authority (BARDA) to expedite the advancement of MultiStem to treat patients with ARDS resulting from the COVID-19 epidemic and other potential pandemic outbreaks. According to the company here, MultiStem therapy was designated by BARDA as highly relevant for COVID-19 and other pathogens. This is significant when taken in the context of the BARDA BAA Medical Countermeasures Acquisition Process here. BARDA evaluates proposals using the criteria (listed in order of importance) of program relevance, overall scientific and technical merits of the proposal, and offerors capabilities and related experience, including qualifications, capabilities, and experience of the proposed key personnel. Though BARDA does not publicly list its classifications of relevance, it stands to reason that they would have their own internal method for classifying potential therapies, given that relevance is the most important criteria in the evaluation process. If Athersys and BARDA complete contract negotiations, a contract could then be awarded. This could include funding, technical assistance and core services, ranging from a clinical research organization network to Centers for Innovation in Advanced Development and Manufacturing, and a fill-finish manufacturing network. A contract with BARDA would represent a significant near-term catalyst for Athersys.

Healios recently announced the inclusion of an additional cohort of COVID-19 induced ARDS patients to their existing ARDS trial here. Importantly, the results of the five newly added COVID-19 ARDS cases could enroll quickly given the seriousness of the current pandemic, and will be verified separately from the 30 originally planned ONE-BRIDGE patients. Per the company, the addition of this COVID-19 cohort will have no effect on the progress of the originally planned clinical trial. Though the ONE-BRIDGE trial has an estimated primary completion date of December 31, 2020 on clinicaltrials.gov, Healios CEO Hardy Kagimoto stated on their most recent company update on February 20, 2020 here (at the 11:25 mark of the video) that the trial may finish early. Also, Healios Director Greg Bonfiglio recently disclosed at the Phacilitate Leaders World & World Stem Cell Summit 2020 conference that the ARDS trial should finish in May (next month) if enrollment continues as expected. The video of the conference is here, and he makes remarks about Healios from the 9:45 mark to around 11:05. An announcement by Healios that they have completed enrollment of their ARDS trial early would provide another significant catalyst to Athersys. Remember that Healios has Orphan Designation for this program in Japan and would receive priority review for approval. If the ARDS indication is approved in Japan, Athersys is entitled to success-based filing and regulatory approval milestones for ARDS aggregating up to $50 million per this Form 8-K current report filing with the SEC, which would provide a cash infusion to Athersys balance sheet. Per the same 8-K, Athersys will also be entitled to receive tiered royalties on the aggregate product sales starting in the low double digits and increasing incrementally into the high teens depending upon net sales levels, as well as potential sales milestones of up to $175 million.

The second program I will address is the acute ischemic stroke program. Acute ischemic stroke represents another tremendous area of unmet need. Athersys and Healios are both currently conducting registrational trials for acute ischemic stroke, called MASTERS-2 and TREASURE, respectively. Athersys phase 3 program is being conducted under a special protocol assessment (SPA) and has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA. These important designations were awarded by the FDA after the results of Athersys phase 2 trial (MASTERS-1) were published. Healios ischemic stroke program in Japan has been awarded Sakigake designation in Japan, which qualifies the program for expedited review. This slide from the Ministry of Health, Labour and Welfare (MHLW) in Japan depicts the designation criteria and advantages for therapies utilizing Sakigake designation. Under designation criteria, #2 clearly states "prominent effectiveness can be expected". The MASTERS-1 phase 2 data set was used to make this determination. A key designation advantage is a prioritized review within 6 months of submission.

Source: MHLW summary of Sakigake strategy here

Though the primary endpoint for Athersys phase 2 study was not statistically significant at 90 days, detailed post hoc analysis found that patients who were administered MultiStem within 36 hours saw dramatic improvements over patients who received only placebo at both 90 and 365 days. Again, all the regulatory designations were awarded after the results were published. The following table from Athersys April 2020 company presentation depicts these results:

Source: Athersys April 2020 corporate presentation here

Healios phase 2/3 TREASURE trial for acute ischemic stroke was initiated in November 2017. The primary endpoint is proportion of subjects with an excellent outcome defined by the functional assessments where mRS score of 1 (scale, 0 to 6), NIHSS score of 1 (scale, 0 to 42), and BI score of 95 (scale 0-100) at day 90. The trial rationale here states by administering MultiStem within 36 hours onset, there was a significant improvement of an efficacy compared with placebo within 36 hours (Excellent outcome was 16.1% in MultiStem group and 0% in Placebo group, p=0.02). Additionally, if the group using both rt-PA and endovascular thrombectomy was excluded, the effectiveness of MultiStem compared with placebo was confirmed. The following table from Healios FY19 results presentation effectively depicts this:

Source: Healios FY2019 financial results presentation here

The TREASURE trial is due to complete enrollment sometime this summer, per recent disclosures. The most recent disclosure was from Healios Director Greg Bonfiglio at the Phacilitate Leaders World & World Stem Cell Summit 2020 conference, where he stated the TRESASURE trial should complete enrollment by September (see aforementioned video). This is significant for both Athersys and Healios as 90-day primary outcome measure data would be available by around the end of 2020. Healios annual target market in ischemic stroke is 62,000 patients. If approved in Japan, this represents a large market opportunity for Healios and a major catalyst for Athersys. Per this Form 8-K current report filed with the SEC, Athersys may be entitled to receive additional success-based development and regulatory approval milestone payments aggregating up to $30 million, as well as potential sales milestones of up to $185 million. Per the same 8-K, Athersys would also receive tiered royalties on product sales, starting in the low digits and increasing incrementally depending on net sales levels.

Athersys Phase 3 MASTERS-2 trial started in July 2018 and continues to see steady enrollment, as stated by the company. This will represent an even larger market opportunity than Japan and is tentatively projected to be completed with enrollment around the end of 2020. For a detailed listen into the science behind this important program, I recommend this podcast featuring Dr. Robert Willie Mays, the Co-founder and Vice President of Regenerative Medicine and Head of Neuroscience Programs at Athersys.

Athersys has other key MultiStem programs currently in development. Most recently, Athersys announced the FDA has authorized the initiation of a Phase 2 clinical trial evaluating MultiStem cell therapy in Trauma patients here. This trial is supported by the Medical Technology Enterprise Consortium (MTEC). MTEC is a 501c(3) biomedical technology consortium collaborating under an Other Transaction Agreement (OTA) with the U.S. Army Medical Research and Development Command (USAMRDC). Athersys also has Fast Track designation and a SPA in place for their Phase 3-ready acute graft versus host disease program, as well as Orphan Drug designation by the FDA and EMA. Another intriguing indication in which Athersys collaborator Emily R. Thompson recently completed a preclinical study is the novel delivery of cellular therapy to reduce ischemia reperfusion injury in kidney transplantation here. Ms. Thompson received the prestigious European Society for Organ Transplantation (ESOT) Leonardo da Vinci Transplant Award in 2019 for her work in this area using MultiStem cells. Athersys has also conducted extensive preclinical work in hemorrhagic stroke, traumatic brain injury here , spinal cord injury here , and many other indications, demonstrating the versatility of MultiStem cell therapy. Many of their preclinical programs have the ability to move straight to phase 2 development.

Preparing To Commercialize

Athersys and Healios are vigorously preparing for commercialization. Recently, Athersys made two important hires to their senior management team. The first was the hiring of new CFO Ivor Macleod, an experienced biotech insider that brings valuable experience from stints with prominent pharmaceutical companies such as Eisai Inc., Merck, and others. Equally as impressive is the hiring of Maia Hansen, the new Senior Vice President, Operations and Supply Chain. Ms. Hanson previously served as a Senior Partner at McKinsey & Company, a global management consulting firm, where she worked with clients around the world to develop and optimize end-to-end operations and global supply chains.

Healios recently established a marketing and sales team to rapidly develop sales activities after they apply for and receive approval for marketing, as stated in this press release. Manufacturing initiatives are also picking up. In 2017, Athersys entered into an agreement here with Nikon Cell Innovation, a subsidiary of Nikon Corporation, to manufacture MultiStem for Healios in Japan. Athersys also previously announced that it successfully transferred its intellectual property for MultiStem to Nikon Cell Innovation. On April 14, 2020, Nikon Cell Innovation disclosed in this press release that it has obtained a license for manufacturing regenerative medicine products. In my opinion, this clearly indicates that they will have a product to manufacture sooner than later.

As referenced by Athersys Chairman and CEO Gil Van Bokkelen in this earnings call from Q3 2019, Athersys has engaged in other key commercialization initiatives such as the hiring of a leading contract research organization (CRO) in 2019 to assist both Athersys and Healios in their preparations of information and materials needed to support rolling submissions and priority reviews for both ischemic stroke programs in Japan, the U.S., Europe and other relevant jurisdictions. Athersys is also developing a proprietary cryogenic storage system called SIFU that will provide many logistical advantages, as depicted in this slide from their April 2020 company presentation:

Source: Athersys April 2020 corporate presentation here

Per this new Form 8-K SEC filing , Athersys currently has approximately $37.5 million of cash and cash equivalents on the balance sheet as of April 9, 2020. This includes about $7 million of proceeds from Healios full exercise of 4 million shares from existing warrants, detailed in this recent press release. It's important to note that the large warrant exercise by partner Healios represents a major insider transaction. The Form 8-K also states that discussions between Athersys and BARDA are continuing regarding a potential collaboration, for which Athersys has submitted its formal proposal. A formal collaboration with BARDA, if achieved, could result in additional funding to Athersys. Lastly, at the time of the writing of this article, Athersys was conducting a public offering of common stock which will yield around $50 million. This offering is scheduled to close around April 20, 2020. It is my view that the recent public offering should not be seen as an indication that the BARDA deal is off the table, but instead as an effort to quickly ramp up the MACOVIA trial while the contract process finalizes.

I believe Athersys presents a favorable value proposition for potential investors. They currently have two late-stage programs in development, both with potential near and mid-term catalysts that could provide substantial value for shareholders. In my opinion, other positive indicators outlined in this article provides additional evidence that these programs are significantly de-risked. Dawson James Securities currently assigns a 1-year price target of $12, per their most recent company report dated April 15, 2020. As the time of the writing of this article, Athersys shares were trading around $2.10 per share. I believe these shares are extremely undervalued at these levels given the multiple market opportunities in critical areas of unmet need, and currently present a very compelling buying opportunity for potential investors.

As with any investment, there are certain risks to my thesis. For the ARDS clinical trials, enrollment could take longer than expected. This would move the timelines for the expected catalysts to a later date. However, I believe these trials will enroll in a timely manner given the huge need for therapeutics to treat COVID-19 induced ARDS patients. For the acute ischemic stroke clinical trials, there is the possibility that the COVID-19 pandemic could affect future enrollment rates. A recent disclosure by Athersys though indicated that the pandemic has not had a significant adverse effect on their business. Finally, BARDA could somehow choose not to initiate an alliance with Athersys. I believe this scenario is highly unlikely given the relevance of Athersys' ARDS program as it pertains to the COVID-19 pandemic.

Disclosure: I am/we are long ATHX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

See the rest here:

Athersys: Abundance Of Opportunity - Seeking Alpha

Read more
Northwestern Scientists Awarded Top Honors for Achievement in Clinical Research – Northwestern University NewsCenter

Northwestern University scientists received top honors from the Clinical Research Forum as part of its 2020 Top Ten Clinical Research Achievement Awards program, taking home the associations highest honor and capturing more finalist nominations than any other institution.

The remarkable success of these brilliant and dedicated investigators shows the strength and breadth of Northwesterns clinical research program and demonstrates our shared commitment as an institution to groundbreaking science that transforms human health, said Eric G. Neilson, MD, vice president for medical affairs and Lewis Landsberg Dean, Northwestern University Feinberg School of Medicine.

John Rogers, PhD, the Louis Simpson and Kimberly Querrey Professor of Materials Science and Engineering, Biomedical Engineering and Neurological Surgery, was awarded the prestigious Herbert Pardes Clinical Research Excellence Award for the research study that best shows a high degree of innovation and creativity, advances science and has an impact upon human disease.

Richard Burt, MD, chief of Immunotherapy and Autoimmune Diseases in the Department of Medicine, was chosen to receive the Distinguished Clinical Research Award. Norrina Allen, PhD, associate professor of Preventive Medicine in the Division of Epidemiology, and Daniela Matei, MD, the Diana, Princess of Wales Professor of Cancer Research and a professor of Medicine in the Division of Hematology and Oncology, were also named to the list of top 20 finalists.

Rogers and Burt are members of the Northwestern University Clinical and Translational Sciences (NUCATS) Institute. The Clinical Research Forum, an organization dedicated to supporting the clinical translational research enterprise and promoting understanding and support for clinical research and its impact on health and healthcare, celebrates outstanding research accomplishments that exemplify innovation and impact on human disease.

Northwestern studies honored by the Clinical Research Forum are:

Skin-like Devices for Wireless Monitoring of Vital Signs in Neonatal Intensive Care (John Rogers, PhD), published in Science. Reporting on the development and validation of a pair of soft, flexible wireless sensors that replace the tangle of wire-based sensors that currently monitor babies in hospitals neonatal intensive care units. The study concluded that that the wireless sensors provided data as precise and accurate as that from traditional monitoring systems, and were gentler on a newborns fragile skin and allow for more skin-to-skin contact with the parent, which has been shown to improve the health of infants and promote emotional bonding.

Hematopoietic Stem Cell Transplantation for Frequently Relapsing Multiple Sclerosis (Richard Burt, MD), published in JAMA. Reporting the success of a process called hematopoietic stem cell transplantation, which temporarily shuts down and reboots patients immune systems with the application of a patients own stem cells, this study demonstrated significant improvement over the current therapies. The study found benefits for patients which no drug had been able to accomplish before.

Associations of Dietary Cholesterol or Egg Consumption with Incident Cardiovascular Disease and Mortality (Norrina Allen, PhD), published in JAMA.

The results of this large study found that adults who ate more eggs and dietary cholesterol had a significantly higher risk of cardiovascular disease and death from any cause.

The study suggested that current U.S. dietary guideline recommendations for dietary cholesterol and eggs, one of the richest sources of dietary cholesterol among all commonly consumed foods, may need to be re-evaluated.

Adjuvant Chemotherapy plus Radiation for Locally Advanced Endometrial Cancer (Daniela Matei, MD), published in New England Journal of Medicine.

This study found that radiation combined with chemotherapy did not increase recurrence-free survival in women with stage III/IVA endometrial cancer, normally the standard of care in these cases.

Endometrial cancer, which begins in the uterus, is the most common gynecologic cancer with most cases occurring in women after age 55, and both occurrence of and mortality from the disease are rising.

Nominees and Top Ten Awardees were announced at the end of January, and the Herbert Pardes Clinical Research Excellence Award and the Distinguished Clinical Research Achievement Awards were announced virtually on April 15.

Learn more about Northwestern University Feinberg School of Medicine at https://www.feinberg.northwestern.edu/.

More:

Northwestern Scientists Awarded Top Honors for Achievement in Clinical Research - Northwestern University NewsCenter

Read more
Sangamo Therapeutics (SGMO), Mogrify announce collaboration and exclusive license agreement for Mogrify’s iPSC- and ESC-derived regulatory T cells -…

As you were browsing http://www.streetinsider.com something about your browser made us think you were a bot.

If you are interested in http://www.streetinsider.com content, APIs are available. Please contact us here http://www.streetinsider.com/signup_content.php

After completing the CAPTCHA below, you will immediately regain access to http://www.streetinsider.com.

Continue reading here:

Sangamo Therapeutics (SGMO), Mogrify announce collaboration and exclusive license agreement for Mogrify's iPSC- and ESC-derived regulatory T cells -...

Read more
War Paint to Bone Paint: MIT Spinout Aims to Transform Regenerative Meds – Xconomy

XconomyBoston

Driven by 20 years in the military, the CEO of Theradaptive hopes his firms regenerative paint technology platform can transform bone repair, spinal fusion, and sports medicine.

The Boston-based firm, spun out of MIT, has a computational platform it says turns recombinant proteins into material-binding variants that behave like a paint. This can then be used to coat implants, devices, and injectable carriers to enable long-term local delivery without off-target effects.

Our approach takes protein sequences that we discover using a lab-based test and optimizes them in several ways using our computational processes, CEO Luis Alvarez (pictured below) told this publication.

For example, we can take a set of sequences which exhibit tight binding to a given implant material and make those sequences easier to manufacture, bind tighter, and able to survive terminal sterilization.We can also tune the strength of binding so that you can predict the release rate of the protein. This has allowed us to make protein therapeutics that stick to implants like a paint and make them bioactive.

Alvarez himself served 20 years in the US Army, experience that drove him to develop the technology and target therapies for traumatic orthopedic injuries.

During my time in Iraq, I witnessed service members who suffered traumatic injuries undergo amputations weeks or months after the initial wound, because there was no reliable method for regenerating the bone, he said.

I spent two years as a graduate student at MIT after West Point, but afterreturning fromIraq I asked the Army to allow me to return to MIT to get my PhD in biological engineeringso that I could develop the technology to regenerate tissues.

As such, Alvarez believes that veterans can bring a unique perspective to problem solving and a warrior ethos that is essential in getting things done.I see tremendous opportunity for veterans to help push healthcare innovations into the hands of those who need it most, he said.

Alvarez developed the tech as a Hertz Foundation Fellow at MIT and spun the firm out in 2017. So far, the company has raised over $9 million in non-dilutive funding from the US Department of Defense and the Maryland Stem Cell Research Fund and has also taken investments from strategic partners.

Theradaptives tech is being implemented to establish a best-in-class product pipeline in bone repair, spinal fusion, and sports medicine, tapping into a potential market worth $80 billion, according to the firm.

We have applied our modification to over a dozen proteins with therapeutic applications in these areas and have proven our ability to coat various implant types, including meshes, stents and wafers, with these proteins, Alvarez said. For example, in the area of mesh repair we have two proteins that enhance tissue repair locally so that the mesh can be resorbed and eventually replaced by native tissue. In the area of local chemotherapy, we are able to safely bind very high doses of therapeutic proteins and release them locally at a known rate.

The platform nature of the technology allows Theradaptive to both license the technology out and to develop its own therapeutics.

We started by licensing our technology to firms with existing 501(k) implants that are compatible with our material-binding therapeutic technology, said Alvarez. Additional licensing opportunities are being sought to allow the firm to pursue in-house programs in soft tissue repair, and localized therapeutics. The firms lead candidate, AMP2 for bone repair and spinal fusion, is looking to enter the clinic in 2021 and as such the firm is considering investment from institutional investors.

We also have an early-stage program in cell therapy enhancement where we use our implants to potentiate cell therapies that other companies are developing.

Earlier this year, Theradaptive opened an R&D facility in Frederick, Maryland, expanding capabilities in therapeutic manufacturing, implant design, and additive manufacturing of medical implants. Now plans are underway to establish cGMP manufacturing capabilities to produce therapeutics that will be going into clinical trials in 2021.

When complete, the plant will have the capacity to produce multi-gram quantities of recombinant protein per campaign, using a 200 L stainless-steel bioreactor system and several fermenters along with large capacity chromatography columns, Alvarez said.

Main image: iStock/CIPhotos

Dan Stanton is Xconomy's managing editor and is based in France. You can reach him at dan.stanton@informa.com.

See more here:

War Paint to Bone Paint: MIT Spinout Aims to Transform Regenerative Meds - Xconomy

Read more