GEMoaB Announces Data Presentations Supporting Key Features of Its UniCAR Platform At the Upcoming American Association for Cancer Research (AACR)…

DRESDEN, Germany, May 15, 2020 /PRNewswire/ -- GEMoaB, a biopharmaceutical company focused on the development of next-generation immunotherapies for hard-to-treat cancers, today announced acceptance of three presentations on pre-clinical data for its proprietary universal CAR-T platform (UniCAR) targeting acute leukemia and solid tumors at the 2020 Virtual Annual Meeting of the American Association for Cancer Research (AACR II) being held from June 22-24.

CAR-T cell therapy holds great promise for treating a wide range of malignancies. Nevertheless, the CAR-T approach faces multiple challenges, including the risk of acute and long-term toxicities, a current lack of suitable targets, insufficient engraftment and persistence and a microenvironment hostile to CAR-T cells especially in solid tumors.

The AACR poster presentations highlight GEMoaB's rapidly switchable universal CAR-T platform, UniCAR. The UniCAR platform promises an improved therapeutic window and increased efficacy and safety over conventional CAR-T therapies in hematological malignancies and solid tumors.

"At this year's AACR meeting II, we are pleased to present important pre-clinical data from our rapidly switchable UniCAR platform," said Armin Ehninger, Ph.D., Chief Scientific Officer of GEMoaB. "Our data suggest the opportunity to actively target CD123 in acute leukemias as well as PSMA and PD-L1 in solid tumors due to UniCAR's rapid switch on/off capability. In solid tumor models, they also show potentially superior tumor penetration, expansion and persistence capabilities as well as a reduced risk of immunosuppression by the tumor microenvironment."

The data further support the ongoing clinical development of UniCAR in hematological malignancies and solid tumors. A Phase IA dose-finding study of the first UniCAR asset, UniCAR-T-CD123, for the treatment of relapsed/refractory AML and ALL is ongoing. A Phase IA study with UniCAR-T-PSMA directed against CRPC and other PSMA-expressing late-stage solid tumors will be initiated by H2 2020.

GEMoaB's poster presentations at AACR II:

AboutGEMoaB

GEMoaB is a privately-owned, clinical-stage biopharmaceutical company that isaiming to become a globally leading biopharmaceutical company. By advancing its proprietary UniCAR, RevCAR and ATAC platforms, the company will discover, develop, manufacture and commercialize next-generation immunotherapies for the treatment of cancer patients with a high unmet medical need.

GEMoaB has a broad pipeline of product candidates in pre-clinical and clinical development for the treatment of hematological malignancies as well as solid tumors. Its clinical stage assets GEM333, an Affinity-Tailored Adaptor for T-Cells (ATAC) with binding specificity to CD33 in relapsed/refractory AML, and GEM3PSCA, an ATAC with binding specificity to PSCA for the treatment of castrate-resistant metastatic prostate cancer and other PSCA expressing late stage solid tumors, are currently investigated in Phase I studies and globally partnered with Bristol-Myers Squibb. A Phase IA dose-finding study of the first UniCAR asset, UniCAR-T-CD123 for treatment of relapsed/refractory AML and ALL is ongoing, UniCAR-T-PSMA against CRPC and other PSMA-expressing late-stage solid tumors, is planned to be tested in a Phase IA study initiated by H2 2020.

Manufacturing expertise, capability and capacity are key for developing cellular immunotherapies for cancer patients. GEMoaB has established a preferred partnership with its sister company Cellex, a world leader in manufacturing hematopoietic blood stem cell products and a leading European CMO for CAR-T cells, co-operating in that area with several large biotech companies.

About UniCAR

GEMoaB is developing a rapidly switchable universal CAR-T platform, UniCAR, to improve the therapeutic window and increase efficacy and safety of CAR-T cell therapies in more challenging cancers, including solid tumors. Standard CAR-T cells depend on the presence and direct binding of cancer antigens for activation and proliferation. An inherent key feature of the UniCAR platform is a rapidly switchable on/off mechanism (less than 4 hours after interruption of TM supply) enabled by the short pharmacokinetic half-life and fast internalization of soluble adaptors termed targeting modules (TMs). These TMs provide the antigen-specificity to activate UniCAR gene-modified T-cells (UniCAR-T) and consist of a highly flexible antigen-binding moiety, linked to a small peptide motif recognized by UniCAR-T.

About ATAC

GEMoaB's platform of Affinity-Tailored Adaptors for T-Cells (ATAC) is characterized by high binding affinity to tumor antigens and lower affinity to the CD3 antigen on effector T-cells, preventing T-cell auto-activation in pre-clinical models. Safety and tolerability of the treatment are also increased by the relatively short serum half-life (60 min). The use of fully humanized antibodies reduces the risk of immunogenicity even in case of chronic dosing. Half-life extended ATACs are in pre-clinical development.

More information can be found at http://www.gemoab.com.

Forward-looking Statements

This announcement includes forward-looking statements that involve risks, uncertainties and other factors, many of which are outside of our control, that could cause actual results to differ materially from the results and matters discussed in the forward looking statements. Forward looking statements include statements concerning our plans, goals, future events and or other information that is not historical information.

The Company does not assume any liability whatsoever for forward-looking statements. The Company assumes that potential partners will perform and rely on their own independent analyses as the case may be. The Company will be under no obligation to update the Information.

Logo - https://mma.prnewswire.com/media/947610/GEMoaB_Logo.jpg

For further information please contact:Constanze Medack c.medack@gemoab.com; Tel.: +49 351-4466-45027

Investor Contact:Michael Pehl m.pehl@gemoab.com Tel.: +49 351-4466-45030

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GEMoaB Announces Data Presentations Supporting Key Features of Its UniCAR Platform At the Upcoming American Association for Cancer Research (AACR)...

Michael Hiltzik: Don’t be taken in by stem cell firms offering unsubstantiated therapies for COVID-19 – Rome News-Tribune

If you think this can help you, Austin Wolff said earnestly into the camera, its worth a shot .It can only help.

Wolff was speaking on a YouTube video produced for the Novus Center, a Studio City business run by his mother, Stephanie, selling stem cell-related products said to treat chronic pain, sexual performance issues and the effects of aging.

In recent weeks, Novus has begun directing its pitch at potential customers fearful about the effects of the novel coronavirus, implying that its stem cell exosome vapor the supplies for which can be shipped overnight to customers homes can improve lung strength, the immune system and ward off viruses and disease. (Exosomes are a form of cellular secretion.)

These are opportunistic businesses, and COVID-19 for them is an opportunity.

Novus videos bristle with formal disclaimers. Its not going to cure anything, Austin Wolff says on one video. You should only do this if you want to try it.

But the videos seem aimed at viewers desperate for any possible defense against a pandemic whose implacable spread seem to grow more frightening with every passing day.

Novus charges $10,000 for the shipment of vials containing the exosomes and nebulizing equipment. Stephanie Wolff says the business, which has been open for four years, has served about a dozen customers worried about COVID-19 in the last month or two.

Promoters of untested and unlicensed stem cell treatments have jumped into the coronavirus market with both feet, says Leigh Turner, a bioethicist at the University of Minnesota who has been tracking the spread of clinics pitching these treatments to consumers for years.

The direct-to-consumer clinics have pivoted their marketing message to treating or preventing COVID-19, Turner told me. Thats not really shocking, in a way; these are opportunistic businesses, and COVID-19 for them is an opportunity.

In a paper scheduled to be published shortly in the prestigious journal Cell Stem Cell, Turner examines how these businesses are preying on public fears and anxieties about the pandemic.

Typically, their claims fall short of actually promising cures or even specific treatments; that holds at bay the Food and Drug Administration, which has sought to shut down clinics offering unproven therapies for conditions such as Alzheimers, diabetes, multiple sclerosis and erectile dysfunction.

Some use the language of immune booster or preventive intervention, Turner says. Theyre not trying to treat somebody whos in an ICU bed. Its more the worried well theyre going after people who are anxious, fearful of the pandemic, and susceptible to claims that a stem cell procedure will reduce their chance of becoming infected.

These treatments can come with a healthy price tag, ranging hundreds to thousands of dollars. But they run up against one indisputable fact: There are no approved stem cell treatments for COVID-19.

Those are the words of Martin F. Pera, a leading stem cell researcher who is editor-in-chief of Stem Cell Reports, the open-access journal of the International Society for Stem Cell Research. The society issued a stern warning March 6 against claims that stem cells can be used to treat people infected with COVID-19.

As for the products sold by Novus, the FDA warned consumers in December that there are currently no FDA-approved exosome products. The agency stated that certain clinics across the country offering such products to patients deceive patients with unsubstantiated claims about the potential for these products to prevent, treat or cure various diseases or conditions.

Weve reported for years on the proliferation of clinics selling purported therapies based on stem cell injections costing as much as $15,000 each.

These treatments arent supported by scientific research, typically arent covered by insurance, and have been targets of an FDA crackdown. (Turner did groundbreaking work with UC Davis biologist Paul Knoepfler in 2016, sounding the alarm about the spread of these clinics.)

In early April, the FDA sent letters to two stem cell firms, Dynamic Stem Cell Therapy of Henderson, Nev., and Kimera Labs of Miramar, Fla., that it said had been marketing their products for the treatment or prevention of COVID-19 and warning them that any such products would have to meet regulatory standards for drugs. But the agency didnt explicitly threaten them with legal consequences. Kimera is the supplier of exosomes to Novus.

Frightened laypersons arent the only targets of claims for cellular treatments for COVID-19. So are decision-makers and government regulators.

The FDA came under fire in March when it issued an emergency use authorization to allow the prescribing of two antimalarial drugs, chloroquine and hydroxychloroquine, for COVID-19 patients. The action came after President Trump had been relentlessly promoting the drugs as potential game changers in the battle against COVID-19.

Less than a month later, the FDA issued a warning against using the drugs against COVID-19 because of reports of serious heart problems in COVID-19 patients who had taken them, as well as the absence of evidence that they were safe and effective for treating the disease.

It wouldnt be surprising to see more companies and clinics showing up in the media and on cable television hawking unsubstantiated stem cell treatments for COVID-19. On May 4, the San Diego stem cell firm Giostar issued a news release asserting that it had received approval for a COVID-19 clinical trial using stem cells to treat COVID-19 patients, under the approval of the United States Food and Drug Administration (FDA) expanded access for compassionate use program.

Is this plausible? Weve reported before that Giostar had made untrue claims about its scientific connections: Several legitimate stem cell scientists the firm listed as members of its scientific advisory board said they had no connection with Giostar and had repeatedly asked that their names be removed from its website. The company has also acknowledged that it had exaggerated the professional credentials of its co-founder and chairman, Anand Srivastava.

Giostars claim in its news release that it would conduct the clinical trial under the FDAs expanded access for compassionate use program is curious. That program, which allows doctors to prescribe unapproved drugs as a last resort for people suffering from life-threatening diseases with no established cure, covers patients for whom enrollment in a clinical trial is not possible.

In other words, there doesnt seem to be such a thing as a clinical trial conducted subject to the expanded access program.

Giostar didnt respond to our request for comment. The FDA would say only that it generally cannot disclose information about an unapproved application, which certainly suggests that Giostar hasnt won the approval it claims.

In the frenzied search for COVID-19 treatments, it may be difficult to distinguish promising efforts from those just grasping at the main chance.

What we have right now is a COVID-19 gold rush, Turner says. Businesses are seeing this as a terrific opportunity to get their applications for investigative new drug trials approved by the FDA a process that can take years and generally requires the submission of extensive evidence from lab and animal studies.

The direct-to-consumer pitches by clinics reviewed in Turners paper typically fuse pseudoscience, which is what theyre offering, with more credible forms of science. He found numerous references in these pitches to research from China, often of doubtful scientific significance.

A Pennsylvania clinic offering stem cell treatment to support lung health during COVID-19, for example, cited a report from a Beijing hospital where seven patients were injected with stem cells all saw significant improvement in COVID-19 related pneumonia, according to the clinics press news . It quoted its CEO stating, This goes to support the wide range of healing and restoration that can be provided by (stem cell) therapy.

However, as Turner observes, the report didnt specify the severity of the subjects pneumonia, the source of the stem cells, or results from a control group. At best you can say that no one seemed to be harmed, but its hard to draw any firm conclusions about efficacy.

The Novus Center hangs its pitch on what Stephanie Wolff describes as a study thats ongoing in China right now using exosomes to help with viral load, to help with inflammation of the lung, to help with pneumonia, to help with infection.

The reference, however, is to a clinical trial in Wuhan that had not even begun to recruit test subjects at the time of its latest public report, which is dated Feb. 25. The researchers didnt expect their trial to be completed until July 31.

As weve written before, the proliferation of stem cell clinics selling untested and unlicensed therapies has been a public health crisis for years. The COVID-19 pandemic will only deepen the crisis as clinics add the coronavirus to their menu of treatment claims.

Despite its crackdown campaign, the FDA has never taken strong enough action against this corner of the healthcare industry. It should act without delay to shut down opportunistic initiatives, or more innocent Americans will find their health, and their pocketbooks, at ever greater risk.

2020 Los Angeles Times

Visit the Los Angeles Times at http://www.latimes.com

Distributed by Tribune Content Agency, LLC.

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Michael Hiltzik: Don't be taken in by stem cell firms offering unsubstantiated therapies for COVID-19 - Rome News-Tribune

Mike Tyson lifts lid on training regime and stem-cell therapy ahead of comeback – Metro.co.uk

Iron Mike will break his soul to get back in peak physical condition (Pictures: Instagram / @MikeTyson)

Heavyweight boxing legend Mike Tyson has explained his gruelling training regime as he bids to return to the ring and says he is still feeling weird after using stem-cell therapy.

The 53-year-old showed off his incredible, age-defying speed and power in training footage he uploaded to Instagram last week, declaring he was back and ready to take part in exhibition fights for charity.

Iron Mike has already been inundated with potential challengers to fight, with old heavyweight rival Evander Holyfield posting his own video in response to Tyson and opening the door to a potential third fight between the pair.

Joe Rogan recently suggested that Tysons incredible speed and power was not down to natural hard work alone, and now the veteran boxer has revealed the extreme lengths he is going to in order to get back in shape.

Speaking on the Rock the Bells Radio show on SiriusXM, Tyson was asked by rapper LL Cool J how he would get in peak shape in just six to eight weeks and replied: Really I would just change my diet and just do cardio work. Cardio has to start, you have to have your endurance to go and do the process of training.

So something to do is get in cardio, I would try and get two hours of cardio a day, make sure you get that stuff in. Youre gonna make sure youre eating the right food.

For me its almost like slave food. Doing what you hate to do but doing it like its nothing. Getting up when you dont want to get up. Thats what it is. Its becoming a slave to life.

People think a slave to life is just enjoying drugs and living your life. Being a slave to life means being the best person you can be, being the best you can possibly be, and when you are at the best you can possibly be is when you no longer exist and nobody talks about you. Thats when youre at your best.

Probed further on the mental aspect of preparing for a fight, Tyson continued: My mind wouldnt belong to me. My mind would belong to somebody that disliked me enough to break my soul, and I would give them my mind for that period of time.

Six weeks of this and Id be in the best shape Ive ever dreamed of being in. As a matter of fact, Im going through that process right now. And you know what else I did, I did stem-cell research.

After LL Cool J asked if that meant Tysons white blood cells had been spun and then put back in, Tyson continued: Yes. As they took the blood it was red and when it came back it was almost transfluid [sic], I could almost see through the blood, and then they injected it in me. And Ive been weird ever since, Ive got to get balanced now.

Tyson first revealed that he had undergone stem cell treatment which is usually used to treat or prevent a disease or condition in an Instagram live chat with Shaquille ONeal earlier this month.

You know what I had done? I had stem cell therapy, said Iron Mike. I feel like a different person but I cant comprehend why I feel this way. Its really wild what scientists can do.

MORE: Evander Holyfield demands bizarre no knockouts rule for potential Mike Tyson comeback fight

MORE: Joe Rogan shocked by fu*king sensational training videos of Mike Tyson

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Mike Tyson lifts lid on training regime and stem-cell therapy ahead of comeback - Metro.co.uk

Mum’s brutal stem cell treatment has ‘all been worth it’ as she enjoys time with family – Grimsby Live

A Scunthorpe mum who underwent 'brutal' stem cell treatment says the hardest part was not being able to see her daughters.

Joanne While has recently passed the six month anniversary of the treatment to wipe out and then regrow her immune system.

The mum-of-three was diagnosed with Multiple Sclerosis (MS) at the age of 31, and wasn't eligible for trial treatments in the UK.

The HSCTtreatment in Mexico saw her undergo chemotherapy and then have stem cells transplanted in the hopes of stopping the damage that the MS was causing. Some MS patients have also seen their symptoms be reversed from this.

"It was a very harsh, brutal treatment. I had to be kept in a special apartment where I protected from all germs. There was a lot of sickness and just getting out of bed some days was difficult," Joanne said.

"The hardest part was being away from my family. My ex-partner was very kind in taking a months unpaid leave to come to Mexico and help me through the treatment.

"At the end of the day, all of the treatment has been worth it.

"I had a brain MRI scan two months ago which showed that the MS lesions hadnt grown since last time. My body is still recovering, so time will tell exactly how good the news is."

The HSCT (haematopoietic stem cell transplantation) treatment cost the family 45,000 including flights and visas. They launched an online fundraising page last year to help cover the costs.

With her weak immune system still being rebuilt, Joanne has been shielding since before the coronavirus outbreak began.

Her family have adopted extremely strict hygiene measures at the Scunthorpe home to keep her healthy during this critical time.

HSCT is a chemotherapy-based medical procedure that wipes out the immune system and reboots it using a patient's own stem cells, which are harvested from their blood or bone marrow,

The aim is to reset the immune system to stop it attacking the rest of the body, therefore halting the progression of the MS.

It is the only medical procedure currently available that has halted the progression of the majority of patients undertaking it.

HSCT is currently available only on a trial basis in the UK, and only for individuals who have been unsuccessful with the range ofdisease modifying therapies. Each time it fails, irreparable damage is being done and the disease continues to progress.

"Before the outbreak, it had just gotten to the point where I was able to venture out for a coffee, but of course all of that has stopped now," Joanne said.

"I had to pull my daughters out of school early to minimise the risk of them bringing the virus home. Now we regularly sanitise the house and change clothes whenever we have to enter or leave in order to keep it as clean as possible.

"My eldest daughter, who is 24, has been wonderful as my carer. She has stopped work to prevent her from catching any infections.

"Im often tired and need a three hour nap in the afternoon, which can be difficult with a five-year-old in the house. Its been a balancing act, but Im so grateful to everyone who helped me during or since the treatment.

"Shielding can be frustrating, but its all about your mindset when you look at it. Its not that I cant go outside I get to be at home in my favourite place with my daughters."

Due to her compromised immune system, Joanne has had to start her vaccinations again and has just been given those that are usually given to babies.

Tests have also shown that her white blood count has recently decreased to the point it was in Mexico, although Joanne has hopeful it will recover.

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HSCT can initially cause mobility issues and stiffness in muscles, which Joanne is having physio to manage.

She is documenting her recovery on her Facebook page 'Jo's HSCT Journey'.

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Mum's brutal stem cell treatment has 'all been worth it' as she enjoys time with family - Grimsby Live

Global Stem Cell Partnering Terms and Agreements 2010-2020 – ResearchAndMarkets.com – Business Wire

DUBLIN--(BUSINESS WIRE)--The "Global Stem Cell Partnering Terms and Agreements 2010-2020" report has been added to ResearchAndMarkets.com's offering.

This report provides comprehensive understanding and unprecedented access to the stem cell partnering deals and agreements entered into by the worlds leading healthcare companies.

The report provides a detailed understanding and analysis of how and why companies enter Stem Cell partnering deals. These deals tend to be multicomponent, starting with collaborative R&D, and proceed to commercialization of outcomes.

This report provides details of the latest Stem Cell agreements announced in the life sciences since 2010.

The report takes the reader through a comprehensive review Stem Cell deal trends, key players, top deal values, as well as deal financials, allowing the understanding of how, why and under what terms, companies are entering Stem Cell partnering deals.

The report presents financial deal term values for Stem Cell deals, listing by headline value, upfront payments, milestone payments and royalties, enabling readers to analyse and benchmark the financial value of deals.

The middle section of the report explores the leading dealmakers in the Stem Cell partnering field; both the leading deal values and most active Stem Cell dealmaker companies are reported allowing the reader to see who is succeeding in this dynamic dealmaking market.

One of the key highlights of the report is that over 600 online deal records of actual Stem Cell deals, as disclosed by the deal parties, are included towards the end of the report in a directory format - by company A-Z, stage of development, deal type, therapy focus, and technology type - that is easy to reference. Each deal record in the report links via Weblink to an online version of the deal.

In addition, where available, records include contract documents as submitted to the Securities Exchange Commission by companies and their partners. Whilst many companies will be seeking details of the payment clauses, the devil is in the detail in terms of how payments are triggered - contract documents provide this insight where press releases and databases do not.

The initial chapters of this report provide an orientation of Stem Cell dealmaking.

A comprehensive series of appendices is provided organized by Stem Cell partnering company A-Z, stage of development, deal type, and therapy focus. Each deal title links via Weblink to an online version of the deal record and where available, the contract document, providing easy access to each deal on demand.

The report also includes numerous tables and figures that illustrate the trends and activities in Stem Cell partnering and dealmaking since 2010.

In conclusion, this report provides everything a prospective dealmaker needs to know about partnering in the research, development and commercialization of Stem Cell technologies and products.

Analyzing actual contract agreements allows assessment of the following:

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/84edx3

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Global Stem Cell Partnering Terms and Agreements 2010-2020 - ResearchAndMarkets.com - Business Wire

Kleo Pharmaceuticals to Present Preclinical Data Highlighting the Synergistic Potential of Kleo Asset KP1237 and Autologous NK Cells in the Treatment…

NEW HAVEN, Conn., May 15, 2020 (GLOBE NEWSWIRE) -- Kleo Pharmaceuticals, Inc., a leading company in the field of developing next-generation, fully synthetic bispecific compounds designed to emulate or enhance the activity of biologics, today announced preclinical data for the companys lead program, KP1237 in combination with autologous, cytokine-induced, memory-like (CIML) natural killer (NK) cells with low dose IL-2 in multiple myeloma (MM). KP1237 is a CD38-targeting antibody recruiting molecule (ARMTM). These data, to be presented as a poster at the 2020 American Society of Clinical Oncology (ASCO) Annual Meeting being held May 2931, will support the initiation of an upcoming Phase 1/2 clinical trial for MM patients receiving an autologous stem cell transplant (ASCT), who remain minimum residual disease positive (MRD+)

This research, in collaboration with Dr. Rizwan Romee, Director of the Haploidentical Donor Transplantation Program at the Dana Farber Cancer Institute, will be presented as a poster and highlight the activity of the combination of KP1237 with CIML NK cells against CD38-expressing MM target cells. Several in vitro and ex vivo experiments show how KP1237 targets the NK cell therapy to the tumor cells and increases their cytotoxicity. These data led to the clinical exploration of this combination product in the high unmet medical need population of multiple myeloma patients who are MRD+ pre-ASCT.

These data mark an important milestone for the ARM platform as we advance towards the first clinical trials for our growing company, said Doug Manion, Kleos Chief Executive Officer and Chairman of the Board. Initiation of these trials will allow us to demonstrate clinical proof of concept and, ultimately, facilitate the expansion of our technology platforms across indications. Additionally, this milestone moves us closer to our primary goal of having a meaningful impact on patient survival and quality of life.

ARMs are unique, bispecific molecules composed of two active ends connected by a linker. One of the ends binds to a target molecule on a cancer cell, while the other end can bind to and thus recruit all endogenous IgG antibodies circulating in the body, which then bind to and activate NK cells. Therefore, ARMTM molecules behave similarly to chimeric antigen receptors to target immune cells to tumors, though their synthetic nature eliminates the need for genetic engineering of the cells. KP1237, by targeting CD38 expressed on the surface of multiple myeloma cells, facilitates NK-cell mediated killing of these tumor cells. The modular design enable ARMTM molecules to be broadly applicable as targeting tolls for all types of NK cell products across a range of tumor types.

Details of the poster presentation are as follows:

Title: A first-in-class ex vivo combination between cytokine-induced memory like (CIML) NK cells and a CD38 targeting antibody recruiting molecule (ARM) as a novel approach to target NK cells without cellular engineering for the treatment of multiple myeloma.Session: Hematologic MalignanciesPlasma Cell DyscrasiaAbstract: #8523

This ASCO abstract is now available at https://meetinglibrary.asco.org/record/187657/abstract. The poster presentation will include additional data not available in the abstract.

About Kleo Pharmaceuticals, Inc.

Kleo Pharmaceuticals is a unique biotechnology company developing next-generation, bispecific compounds designed to emulate or enhance the activity of biologics based on the groundbreaking research of its scientific founder Dr. David Spiegel at Yale University. Kleos compounds are designed to direct the immune system to destroy cancerous or virally infected cells and are currently in development for the treatment of various diseases, including multiple myeloma and COVID-19. Compared to biologics, Kleos compounds are smaller and more versatile, leading to potentially improved safety and efficacy. They are also much faster and more efficient to design and produce, particularly against novel targets. Kleo develops drug candidates based on its proprietary technology platforms, all of which are modular in design and enable rapid generation of novel immunotherapies that can be optimized against specified biological targets and combined with existing cell- or antibody-based therapies. These include Antibody Recruiting Molecules (ARMs) and Monoclonal Antibody Therapy Enhancers (MATEs). Biohaven Pharmaceutical Holding Company (NYSE:BHVN) and PeptiDream Inc. (Nikkei:PPTDF) are investors in Kleo Pharmaceuticals. For more information visit http://kleopharmaceuticals.com.

Forward-Looking StatementsThis news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of the Company's management. All statements, other than statements of historical facts, included in this press release regarding the Company's plans and objectives, expectations and assumptions of management are forward-looking statements. The use of certain words, including the words "estimate," "project," "intend," "expect," "believe," "anticipate," "will, "plan," "could," "may" and similar expressions are intended to identify forward-looking statements. The forward-looking statements are made as of this date and the Company does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.

CONTACT INFORMATION

LifeSci Advisors (Investors)

Irina Koffler

646-970-4681

ikoffler@lifesciadvisors.com

Kleo Pharmaceuticals (Press)

Brian Dowd, PhD

203-390-9375

bdowd@kleopharmaceuticals.com

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Kleo Pharmaceuticals to Present Preclinical Data Highlighting the Synergistic Potential of Kleo Asset KP1237 and Autologous NK Cells in the Treatment...

Cost-Effectiveness Analysis of Tisagenlecleucel in the Treatment of Re | CEOR – Dove Medical Press

Josep Maria Ribera Santasusana,1 Alejandra de Andrs Saldaa,2 Nuria Garca-Muoz,3 Joana Gostkorzewicz,2 Diana Martnez Llins,3 Cristina Daz de Heredia4

1Clinical Hematology Department, Catalan Institute of Oncology - Hospital Germans Trias i Pujol, Barcelona, Spain; 2Health Economics and Outcomes Research, Novartis Farmacutica S.A., Madrid, Spain; 3Oblikue Consulting, S.L., Barcelona, Spain; 4Paediatric Oncology and Hematology Department - Hematopoietic Stem Cell Transplantation, Hospital Universitari Vall dHebron, Barcelona, Spain

Correspondence: Diana Martnez LlinsOblikue Consulting, S.L., C/Comte dUrgell, 240, 2-D, Barcelona 08036, SpainTel +34 93 252 1377Fax +34 93 737 9984Email diana.martinez@oblikue.com

Purpose: Tisagenlecleucel, a chimeric antigen receptor T-cell (CAR-T) therapy, is a promising alternative for the management of children and young adults with relapsed and refractory B-cell acute lymphoblastic leukemia (r/r ALL). The aim of this study was to determine whether treatment with tisagenlecleucel is a cost-effective intervention compared with salvage chemotherapy in paediatric and young adult patients with r/r ALL in Spain.Materials and Methods: A partitioned survival model of monthly cycles with three health states was used (event-free survival, progressive/relapsed disease and death). A lifetime time horizon and the Spanish National Health System perspective were adopted. During the first 5 years, permanence in the different health states was determined according to the results in the clinical studies. In successive years, mortality tables of the Spanish general population adjusted by standardized mortality rate for survivors of childhood cancer were used. Clinical, economic, and quality of life parameters were drawn from clinical trials and the literature. Only direct health costs (pharmacological costs and the costs derived from health resource use) were included. The robustness of the results was evaluated in a sensitivity analysis.Results: This cost-effectiveness analysis showed a greater benefit (10.10 and 8.97 life-years gained [LYGs] and quality-adjusted life-years [QALYs] gained, respectively) and a higher cost ( 258,378.40) for tisagenlecleucel compared to salvage chemotherapy. The resulting incremental cost-effectiveness and cost-utility ratios were 25,576.80 per LYG and 28,818.52 per QALY gained, respectively. In the sensitivity analysis, all the results were below 50,000/QALY.Conclusion: Tisagenlecleucel would represent a cost-effective intervention for the treatment of children and young adults with r/r ALL in Spain.

Keywords: ALL, cost-effectiveness, tisagenlecleucel, acute lymphoblastic leukaemia, Spain, CAR-T

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Cost-Effectiveness Analysis of Tisagenlecleucel in the Treatment of Re | CEOR - Dove Medical Press

Here’s Why Big Buyers Are Excited About This Cancer Immunotherapy Stock – Motley Fool

Unlike many other mid-cap biotech companies, shares of Fate Therapeutics (NASDAQ:FATE) are almost entirely owned by institutional investors. Just what exactly does the company have in its pipeline that could entice so much interest from smart money?

As of now, over 90% of the company's 78 million shares outstanding are owned by institutional investors, representing a more than $2 billion bet that the company's pipeline candidates will be successful.As many biotech investors know, mid-cap biotech companies have significant risks associated with their clinical programs, especially when their leading candidates are only in phase 1, as the case with Fate Therapeutics.

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The company has garnered so much interest because it's at the forefront of developing the next generation of cellular immunotherapies. The method being investigated by biotechs and researchers around the world is chimeric antigen receptor T-cells (CAR T-cell) immunotherapy, which is rather complicated and inefficient. First, blood plasma from the body of a patient or a suitable donor is withdrawn. Then, it is separated into plasma and T-cells. Next, millions of T-cells need to be genetically engineered to target specific receptors within cancer cells. Finally, those activated T-cells are infused into the patient's body, where they (hopefully) will produce an immune response that attacks the cancer.

The whole process can cost up to $425,000 per patient and takes between two to three weeks. And that's only the overhead costs: After mark-ups and profit margins are factored in, one course of CAR T-cell treatment can cost up to $1.5 million. Hence, there exists a dire need for cancer immunotherapy options that are both effective and affordable -- and Fate Therapeutics intends to fill that void.

The company claims it has developed a new generation of cellular immunotherapies based on induced pluripotent stem cell (iPSC) technology. Using this method, a single stem cell clone can morph into more than 200 different types of cells via genetic engineering, which can then be mass-produced and stored. When cancer patients need a specific type of antibody in their system, they would be able to request the corresponding iPSC on demand from a cell bank.

This was truly a remarkable innovation, and the company wasted no time in defending its intellectual property. Fate Therapeutics currently possesses more than 250 patents for its iPSC technology and has six experimental therapies in clinical trials. One of those is FT500, which is in phase 1 clinical trials.

FT500 is a biologic composed of natural killer cells, which provide the first line of defense against tumors and other pathogens, derived from a clonal master iPSC. In preclinical studies, the biologic showed strong expressions in activating receptors in the body's immune cells against cancer cells.

An interim data release in December revealed that FT500 had been well-tolerated by 12 patients, with no safety or serious autoimmune issues. In addition, six out of 11 patients in both monotherapy and combination arms witnessed stable disease progression, mean that their tumors neither grew by more than 20% in size nor shrank by more than 30%. It is worth noting patients were only eligible for the trial if they had advanced-stage cancers that had failed to respond to standard immune checkpoint inhibitors such as Opdivo and Keytruda. The study is expected to conclude in 2022.

In my opinion, Fate Therapeutics offers investors more than enough reasons to buy. First, it has hundreds of patents covering a new, potentially revolutionary, immunotherapy technology. Second, it has the backing of institutional investors that together own the large majority of the company. Third, the company's financials are sound: it has more than $300 million in cash and equivalents on its balance sheet, no debt, and operating expenses of $100 million annually. That gives it roughly three years to produce some success with its clinical programs.

Finally, the company possesses a fair shot of achieving phase advancement for its pipeline, and it was recently further de-risked when it inked a multibillion-dollar collaboration agreement with Johnson & Johnson (NYSE:JNJ). In all, this is a great stock that biotech investors who have a tolerance for risk will not want to miss.

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Here's Why Big Buyers Are Excited About This Cancer Immunotherapy Stock - Motley Fool

Group of Genes Have Altered Expression in Autism – Technology Networks

Autism has long been associated only with behavioral and environmental factors, but the role of genetics in its development is now increasingly evident. Some 100 genes have been found to play a role in autism spectrum disorder, and another thousand are being studied to the same end.The diagnosis and treatment of the disorder on a genomic basis are hindered by this variability. However, a new study conducted at the University of So Paulo's Institute of Biosciences (IB-USP) in Brazil points to a common gene expression profile regardless of the DNA mutations in any autistic individual.

"We found a group of genes that's dysregulated in neural progenitor cells, which give rise to neurons, and in neurons themselves," said Maria Rita dos Santos e Passos-Bueno, a professor at IB-USP. In other words, while the DNA of different individuals with autism displays different alterations, the behavior of these genes is similar in all such people and differ from that observed in the brains of people without the disorder.

Passos-Bueno is affiliated with the Human Genome and Stem Cell Research Center (HUG-CELL), a Research, Innovation and Dissemination Center (RIDC) supported by So Paulo Research Foundation - FAPESP and hosted by the University of So Paulo (USP).

The study was supported by FAPESP via two research grants awarded under the programs So Paulo Researchers in International Collaboration (SPRINT) and Multiuser Equipment (EMU). The results are reported in the journal Molecular Psychiatry, a Nature publication.ExperimentsSamples of brain tissue cannot be taken from living people, so the researchers conducted in vitro experiments using a technique called cell reprogramming.

"We took dental pulp cells from people with and without autism, and from these, we created pluripotent stem cells, which can be transformed into any type of cell. In this way, we were able to create in the laboratory neural cells with the same genomes as those of the patients," said Karina Griesi Oliveira, the first author of the article. Oliveira has a PhD in genetics from IB-USP and is a researcher in the Albert Einstein Israeli Education and Research Institute (IIEP).

Five individuals with high-functioning autism and one with low-functioning autism were selected for the study; all six had heterogeneous genetic profiles. A control group comprised six healthy subjects.

"The study bore out the hypothesis that, while the origin of autism is multifactorial and different in each person, these different alterations can lead to the same problems in the functioning of their neurons," Oliveira said.

The induced pluripotent stem cells (iPSCs) were reprogrammed to simulate two stages in the development of the human brain: neural progenitor cells, which give rise to neurons, and neurons at a stage equivalent to those of a fetus between the 16th and 20th weeks of gestation.

The researchers then analyzed these cell transcriptomes, consisting of all their RNA molecules. RNA acts as an intermediary that converts the information in a gene into proteins, which in turn govern cell behavior.

"By counting the RNA molecules, we were able to determine gene expression with a considerable degree of precision," Oliveira said.

The researchers next used mathematical models to determine which genes were differentially expressed in both groups (with and without autism), arriving at those responsible for synapses and neurotransmitter release, i.e., genes that modulate communication among neurons. This process influences the functioning of the entire organism, but above all, the brain.

This set of genes, some of which have been associated with autism in previous research, displayed increased activity in neurons. "Some of them were dysregulated in iPSC-derived neural cells from autists studied in other research, and in neurons from postmortem brain tissue belonging to individuals with autism, validating the method," Passos-Bueno said.

On the other hand, this second analysis using postmortem tissue data showed decreased gene expression at the time of death. "We don't know the reason for the difference, but it's consistent evidence that expression of this group of genes is involved in autism spectrum disorder," Oliveira said.Clinical relevanceThe study also provides more evidence that autism begins to develop during gestation. "The study points to a disturbance in fetal neurodevelopment that alters neuronal functioning, so that the child is born with altered gene expression," Passos-Bueno said.

This knowledge may contribute to the diagnosis of autism, currently based on the clinical analysis of symptoms.

Imaging, blood tests and genetic sequencing cannot help diagnose the disorder in the vast majority of suspected cases. "A major genetic error causes autism in some 30% of patients, but the origin of the disorder is multifactorial in 70%, with several alterations to DNA causing clinical symptoms, so that interpretation of the genetic data is still complex," Passos-Bueno explained.

The research line may also favor the development of more effective treatment strategies. "To treat a genetic disease, you have to understand what the genes are doing wrong. The alterations to neurotransmitter control have never been demonstrated so clearly," said Mayana Zatz, a professor at IB-USP and HUG-CELL's principal investigator.ReferenceGriesi-Oliveira et al. (2020). Transcriptome of iPSC-derived neuronal cells reveals a module of co-expressed genes consistently associated with autism spectrum disorder. Molecular Psychiatry. DOI: https://doi.org/10.1038/s41380-020-0669-9

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Group of Genes Have Altered Expression in Autism - Technology Networks

Mesoblast raises $138m from investors on hopes for COVID-19 treatment – Sydney Morning Herald

The key objective of the fundraising is to have sufficient capital to scale up the manufacturing of Remestemcel-L, the biotech's anti-inflammatory treatment used to treat graft-vs-host disease in children and is now trialling for acute respiratory distress caused by the coronavirus.

An early study of a dozen COVID-19 patients paved the way for broader clinical trials of the product, which are now occurring throughout the United States. It is hoped the treatment can help reduce mortality rates for patients who become seriously ill with COVID-19 by providing some relief to acute respiratory distress syndrome, a response where lung tissue gets destroyed in the fight against the virus.

While the results of the trials and the required US Food and Drug Administration approval are still some way off, Dr Itescu said the company had tapped investors to ensure it could capitalise on any good news. "This is how you build a company - you have to [focus] on development and commercialisation and manufacturing. You have to put the building blocks in place," he said.

Billionaire investor Alex Waislitz and chairman of Thorney Opportunities Fund, which is Mesoblast's third-largest shareholder with 6 per cent, said: "I think Meso is now finally well on the way to becoming a major Australian success story and Im hopeful the Australian government will recognise Mesos potential and encourage them to develop their stem cell manufacturing capabilities in Australia rather than lose them to another country such as the United States or elsewhere."

The $1.8 billion ASX-listed company's share price more than doubled between the end of March to the start of May on news of the coronavirus trials. Shares were down by 2 per cent to $3.36 in late trade on Wednesday.

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The company could have a treatment available next year if all pieces fall into place, though Dr Itescu said a successful vaccine was critical. "We hope there will be a vaccine available in the next 12 months. Without a vaccine, the numbers [of COVID-19 patients] will overwhelm health systems," he said.

He predicted Australia had one of the best chances of any developed nations of stopping a second wave of the virus, but things looked less promising overseas. "Social distancing needs to be maintained and opening up needs to be very judicious. I think given how well it has been controlled, I think the opening up has a really good prospect of preventing it [second waves]."

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Mesoblast raises $138m from investors on hopes for COVID-19 treatment - Sydney Morning Herald