Category Archives: Cell Therapy

REGULATED INFORMATION

No statistically significant difference in knee pain reduction between JTA-004, placebo and active comparator, 3months after treatment; favorable JTA-004 safety profile similar to placebo and comparator

Prime focus on the continued development and expansion of its mesenchymal stromal cell based allogeneic cell and gene therapy platform

Management to host conference call today at 4pm CEST / 10am EST - details provided below

Gosselies, Belgium, 30August 2021, 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, today announces that the Phase III knee osteoarthritis study with its enhanced viscosupplement JTA-004 did not meet the primary and consequently the key secondary endpoints.

The primary objective of the JTA-004 Phase III study was to demonstrate the efficacy of JTA-004 in reducing osteoarthritic knee pain compared to placebo as measured by the WOMAC pain subscale three months after treatment. A key secondary objective was the comparison between JTA-004 and comparator Hylan G-F 20 in knee pain relief at month 3. Despite JTA-004s favorable safety profile, the study did not achieve its main objectives as no statistically significant difference in pain reduction could be observed between any of the treatment, placebo and comparator groups, with all treatment arms showing similar efficacy.

A statistically significant difference in favor of JTA-004 and the active comparator versus placebo was seen in a post-hoc analysis in a subset of patients with higher pain scores at entry.

The Company, in collaboration with existing and potential partners, will consider the options for the future of JTA-004 development.

The execution of the study was flawless and a good safety profile was observed in line with previous results. These JTA-004 efficacy results are disappointing. Knee osteoarthritis studies are recognized across the industry to be challenging to evaluate. They are also frequently complicated by a high placebo effect. We will continue to analyze the data and will consider potential next steps, said Miguel Forte, Chief Executive Officer of Bone Therapeutics. We are now fully committed to the clinical development of our advanced MSC allogeneic cell and gene therapy platform. Bone Therapeutics is concentrating on the development of this platform for the large market of orthopedic indications, with ALLOB. The progress with this platform has enabled us to expand it to other indications, including immunomodulation.

Bone Therapeutics is focused on the development of its core assets, the allogeneic cell therapy platform, including ALLOB. ALLOB is currently being evaluated in a randomized, double-blind, placebo-controlled Phase IIb study in 178patients with fresh tibial fractures at risk of delayed or non-union. 5% to 10% of complicated long bone fractures evolve to delayed union and non-union. This study will assess the potential for a single percutaneous injection of ALLOB to accelerate fracture healing and prevent late-stage complications in these patients. Recruitment is expected to be completed in the first half of 2022 and topline results by the end of 2022. Should the pandemic continue, Bone Therapeutics may have to re-evaluate these timelines and, in that eventuality, will communicate again to the market.

Bone Therapeutics is intensifying its efforts to expand its preclinical and clinical pipeline with additional indications by enhancing and professionalizing the therapeutic capacity of its cell and gene therapy platform. This includes the development of a next generation of genetically engineered mesenchymal stromal cells (MSC) and the use of highly scalable and versatile cell sources such as induced pluripotent stem cells (iPSC).

Conference call

The management of Bone Therapeutics will host a conference call today at 4:00 pm CEST / 10:00 am EST. To participate in the conference call, please select your dial-in number from the list below quoting the conference ID 825 1002 3115#:

Belgium: +32 2 290 9360France: +33 1 7095 0103United Kingdom: +44 208 080 6592United States: +1 646 876 9923

About JTA-004 and Phase III knee osteoarthritis study

JTA-004 is Bone Therapeutics next generation of intra-articular injectable for the treatment of osteoarthritic pain in the knee. It consists of a unique mix of hyaluronic acid - a natural component of knee synovial fluid, plasma proteins, and a fast-acting analgesic. JTA-004 intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain.

The JTA-004 Phase III study is a controlled, randomized, double-blind trial. It evaluates the potential of a single, intra-articular injection of JTA-004 to reduce osteoarthritic pain in the knee, compared to placebo or Hylan G-F 20, the leading osteoarthritis treatment on the market. The study is being conducted in 22 centers across six European countries as well as Hong Kong. More than 700 patients with mild to moderate symptomatic knee osteoarthritis were treated in this study.

About Knee Osteoarthritis

Osteoarthritis (OA), also known as degenerative joint disease, is the most common chronic joint condition in which the protective cartilage in the joints progressively break down resulting in joint pain, swelling, stiffness and limited range of motion. The knee is one of the joints that are mostly affected by osteoarthritis, with an estimated 250 million cases worldwide.

The prevalence of knee osteoarthritis (KOA) is expected to increase in the coming years due to increasingly aging and obese population. Currently, there is no cure for KOA and treatments focus on relieving and controlling pain and symptoms, preventing disease progression, minimizing disability, and improving quality of life. Most drugs prescribed to KOA patients are topical or oral analgesics and anti-inflammatory drugs. Ultimately, severe KOA leads to highly invasive surgical interventions such as total knee replacement.

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a diversified portfolio of cell therapies at different stages ranging from pre-clinical programs in immunomodulation to mid stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell and gene therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company has initiated patient recruitment for the Phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available at http://www.bonetherapeutics.com.

For further information, please contact:

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0)71 12 10 00investorrelations@bonetherapeutics.com

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63 56catherine@bepublic.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: +33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

Read more from the original source:
Bone Therapeutics announces topline results from Phase III knee osteoarthritis study with its enhanced viscosupplement JTA-004 - GlobeNewswire

Each week onDocWire News, editors bring you the latest news and research in hematology and oncology.In case you missed it, here are this weeks top headlines.

Editors spoke with Spiro Rombotis, CEO of Cyclacel Pharmaceuticals, Inc., a company developing innovative therapies that target various phases of cell control for treating advanced cancers. Mr. Rombotis discussed the strides his company is making in developing and testing targeted therapies that may actually drive cancer cells to self-destruct.

Essentially, when a cancer cell has progressed to the point that if the generated its code contains mistakes that can be communicated to daughter cells, many of the own circulatory mechanisms of the body are dysfunctional or absent, he explained. By inhibiting both CDK2 and CDK9, we can reinstitute these mechanisms, or programed suicide, and engage those anti-cancer mechanisms you want to stop working and induce the cancer cell to commit suicide.

Watch the full interview to learn more.

Spiro Rombotis, CEO of Cyclacel Pharmaceuticals, on Innovative Targeted Therapies for Advanced Cancer

Primary care providers (PCP) play a critical role in cancer diagnosis, according to a new study from researchers at the University of Michigan Rogel Cancer Center. The findings showed that the older women who reported feeling included in healthcare decisions with their PCP were more likely to be up to date on both colorectal and cervical cancer screenings.

When the researchers compared women who completed both screenings compared to those who had only one screening, they found that communication about health decisions with a PCP was still associated with being up to date on both screenings compared to one.

This creates a hypothesis that comprehensive cancer screening may best be accomplished through a PCP-patient relationship and not through targeted single cancer site screenings, said coauthor Diane M. Harper, MD, MPH.

Primary Care Providers Play Important Role in Cancer Screening Uptake

Patients with schizophrenia can and should participate in cancer screening to the same extent as the general population, with assistance, according to a study published in Acta Psychiatrica Scandinavica. In this first-of-its-kind study, researchers conducted a randomized trial to assess the benefits of interventions for early cancer screening in patients with schizophrenia.

The dissemination of this intervention into routine clinical practice may help bridge the gap between early detection of cancer and mortality across people with schizophrenia and the general population, said the study authors.

Intervention is Needed to Increase Screening for Early Cancer Screening in Patients with Mental Illness

Finally, for men with low-risk to favorable intermediate-risk prostate cancer undergoing active surveillance, high-intensity interval training (HIIT) was found to be beneficial. A new study found that patients who underwent HIIT exercises three times per week experienced decreased PSA level and velocity and prostate cancer cell line LNCaP growth compared to standard care patients.

The HIIT group experienced decreased PSA level (1.1 g/L), PSA velocity (1.3 g/L/year), and prostate cancer cell line LNCaP growth (0.13 optical density unit) compared with the usual care group.

Demonstration that HIIT alone, without dietary changes, resulted in improved cardiorespiratory fitness and biochemical parameters in men with localized prostate cancer on active surveillance and growth inhibition at the cellular level is novel and noteworthy, write the authors of an accompanying editorial.

High-Intensity Interval Training Beneficial in Prostate Cancer

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Hem/Onc Roundup: Pharma CEO Talks Cellular Therapy, and More - DocWire News

CAMBRIDGE, Mass., Aug. 24, 2021 (GLOBE NEWSWIRE) -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Arbor Biotechnologies (Arbor) today announced a new collaboration to enhance efforts in developing ex vivo engineered cell therapies, using Arbors proprietary CRISPR gene-editing technology for select diseases.

The new agreement between Arbor and Vertex builds upon the companies first partnership established in 2018. Under this new partnership, Vertex will receive rights to use Arbors technology to research and develop ex vivo engineered cell therapies towards Vertexs goal of generating fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes, for next-generation approaches in sickle cell disease and beta thalassemia, and for the treatment of other diseases.

This new collaboration further expands our toolkit in cell and genetic therapies and, specifically, our work to discover and develop cell therapies for the treatment of multiple serious diseases, said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. We are excited to bring Arbors technology together with Vertexs ongoing programs and capabilities in diabetes, hemoglobinopathies and other diseases to create improved cell replacement therapies for broad populations of patients.

Arbor and Vertex share a common goal to improve the lives of people with life-threatening diseases through the discovery and development of innovative therapies, and Vertex has proven to be an ideal partner in that effort, said Devyn Smith, Ph.D., CEO of Arbor. This agreement demonstrates the versatility and strength of Arbors platform and reinforces our strategic vision of expanding our engineered cell therapy capabilities through partnerships with leading organizations in our industry, while we focus our internal portfolio efforts on genetic medicines.

About the CollaborationUnder the terms of the agreement, Arbor will receive an upfront cash payment and is eligible to receive up to $1.2 billion in potential payments based upon the successful achievement of specified research, development, regulatory and commercial milestones across up to seven potential programs. In addition,Vertexwill pay tiered royalties on future net sales on any products that may result from this collaboration.Vertex will also make an investment in Arbor in the form of a convertible note.

About VertexVertexis a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF,Vertexhas a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition,Vertexhas a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 inCambridge, Mass.,Vertex's global headquarters is now located inBoston'sInnovation Districtand its international headquarters is inLondon. Additionally, the company has research and development sites and commercial offices inNorth America,Europe,AustraliaandLatin America.Vertexis consistently recognized as one of the industry's top places to work, including 11 consecutive years onScience magazine'sTop Employers list and a best place to work for LGBTQ equality by theHuman Rights Campaign. For company updates and to learn more aboutVertex's history of innovation, visitwww.vrtx.comor follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

About ArborArbor Biotechnologies is an early-stage life sciences company discovering and developing the next generation of genetic medicines. Co-founded by Feng Zhang and David Walt, Arbor uses its proprietary discovery engine to discover unique and wholly owned CRISPR-based genetic modifiers with differentiated genetic editing capabilities. This portfolio of gene editors can be custom tailored to address the underlying pathology of genetic diseases. Arbors pipeline of genetic medicines is focused on bringing curative therapies to all patients with genetic disease. Following its strategic partnership with Vertex Pharmaceuticals to accelerate the path to the clinic for Arbors technologies, Arbor recently announced an agreement with Lonza. These partnerships further validate the breadth of applications of Arbors gene editing platform. For more information about Arbors technologies and innovations, visit arbor.bio.

Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation,statements by Bastiano Sanna, Ph.D., and Devyn Smith, Ph.D., in this press release, statements about the potential benefits and results that may be achieved through Vertexs collaboration with Arbor, statements regarding the future activities of the parties pursuant to the collaboration, statements regarding upfront and milestone payments, potential royalties on future sales and Vertexs investment in Arbor in the form of a convertible note. WhileVertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the companys beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the anticipated benefits and potential of Vertexs collaboration with Arbor may not be achieved, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy or other reasons, and other risks listed under the heading Risk Factors inVertex's annual report and subsequent quarterly reports filed with theSecurities and Exchange Commission(SEC) and available through Vertexs website atwww.vrtx.comand on the SECs website atwww.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Vertex Contact:

Investors:Michael Partridge, 617-341-6108orBrenda Eustace, 617-341-6187orManisha Pai, 617-429-6891

Media:mediainfo@vrtx.comorU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

Arbor Media Contact:Amy Bonanno, +1 914-450-0349abonanno@soleburytrout.comorpress@arbor.bio

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Vertex and Arbor Biotechnologies Establish New Partnership to Develop Novel ex vivo Engineered Cell Therapies - GlobeNewswire

Clinical Trials Arena speaks to Lineage Cell Therapeutics CEO Brian Culley about the firms lead candidate OpRegen for dry AMD and the unexpected cases of retinal restoration in an ongoing Phase I/IIa trial.

Age-related macular degeneration (AMD) is one of the worlds leading causes of blindness in people over the age of 50. There are two types of the disease wet AMD and dry AMD.

The less common wet AMD is well understood as being caused by leaky blood vessels and has numerous effective treatments available. Dry AMD causes layers of the macula (including the photoreceptors and the retinal pigment epithelium) to get progressively thinner and function less and less well. This is called atrophy.

Advanced cases of dry AMD are known as geographic atrophy (GA) because large sections of the retina stop functioning.

There are around two million people in the US suffering with this severe form of the condition, and there are currently no treatments available, representing a huge unmet need. Historically, dry AMD has been viewed as an inevitably progressive disease.

California-headquartered Lineage Cell Therapeutics is taking a very different approach to the disease compared to traditional pharmaceutical approaches, which often involve targeting inflammation with small molecules and antibodies and treating patients either orally or locally in the eye.

With its lead candidate OpRegen, which is in an ongoing Phase I/IIa open-label clinical study, Lineage is manufacturing brand new retina cells and taking a transplant approach to the condition.

Its been very exciting what we have seen in the clinic so far, the companys CEO Brian Culley tells Clinical Trials Arena.

The one-time therapy consists of allogeneic retinal pigment epithelium (RPE) cells and is administered subretinally in patients with dry AMD and GA. The patient is locally anaesthetised and the procedure only takes about 30 minutes.

The firm uses pluripotent stem cells as a starting material to manufacture the therapy. Just as flour can become bread or a cookie, [pluripotent] stem cells have within them the capability or the capacity to become any of the 200 cell types in your body, says Culley.

We instruct the cells to become a specific and exclusive type of cell and then we transplant those into the body. In the case of dry AMD we manufacture enormous numbers of retina cells and only retina cells, and then we transplant those cells to treat disease of the eye.

The manufacture of cells requires exquisite control over your process as you are manufacturing a dynamic living entity at scale, Culley says. If you cannot manufacture at large scale, you will never have an economically affordable solution.

The allogeneic rather than autologous nature of Lineages manufactured retinal cells provides advantages in scale, he argues.

There are approaches that people take even in dry AMD where they take cells from a persons eye, and they manipulate them and then they replace them, but here youre talking about personalised medicine, which sounds great until you consider the cost.

Weve invested significantly in our manufacturing skills and created a huge number of patents from our manufacturing techniques. We are already at the point where, in a three-litre bioreactor, we can manufacture the equivalent of 2500 clinical courses so many thousands of treatments can come from basically a milk jug and scaling up is straightforward because we grow the cells on little microcarriers. We grow the cells in three-dimensional space and that allows us to increase the volume because the cell doesnt really know the difference between growing in a tiny thimble or growing in a swimming pool.

Cell therapy brings with it the complexity of dealing with a whole cell rather than just a single molecule, but that complexity is also the key to this approach.

By the time retina cells are dying off, there are so many things going wrong in the eye that we dont think that a single molecule is going to have enough horsepower to be able to drive a clinical outcome, Culley says. So we think you have to replace the entire cell.

The ongoing Phase I/IIa clinical trial evaluating OpRegen enrolled 24 people. Twelve of the participants treated with the therapy had very advanced AMD and were legally blind.

Culley says this cohort was used to assess the treatments safety and not much was seen in terms of efficacy but some encouraging anatomical changes were observed.

In the eye, the vision cycles metabolic activity leads to a waste material called drusen, which is cleared by healthy retina cells. In some of these patients, a reduction in drusen was observed.

The next group of 12 patients disease was far less advanced and they had far better baseline vision than the first; some even still had their drivers licenses. At their earlier stage of disease, they had more retinal tissue with potential to be rescuable. When the trial moved into the group of patients with better starting vision, Culley and his team started to see very exciting effects in terms of visual acuity and anatomical changes.

We were really happy to see that in that group of individuals, weve had better results, Culley says. We have seen an increase in vision, so people are seeing more letters on an eye chart, compared to their untreated eye. The majority of untreated eyes have gotten worse, which is what you would expect over time. In the treated eye it is reversed; weve actually seen people gain vision.

No patients in the trial, which started treating patients with OpRegen five years ago, have rejected the cells, meaning all have stably integrated them. Most exciting of all are three cases of retinal restoration, which Culley says is really unprecedented stuff due to the progressive nature of the disease.

This is a new phenomenon, says Culley. Human beings cannot regenerate retinal tissue; when you lose retina cells, theyre gone forever. And thats what this disease is all about losing retina cells.

Over a year ago, while looking at images of a patients eyes after OpRegen treatment, doctors noticed retina cells growing back around the spot of GA.

We were absolutely blown away, Culley says. We actually sat on this initial finding for quite some time, because even though it was incredible, and exciting and maybe even predictable in some ways youre transplanting cells and if theyre taken up, survive and are functional why wouldnt this happen but nobody thought it was possible.

The question the firm focused on while analysing the data and sending it out to third-party independent retinal imaging experts was simple why did this patient experience retinal tissue regeneration while no one else did?

We think the answer was that patient got a very complete delivery of our cells all across that atrophic area, explains Culley.

Lineage repeated the procedure and got similarly extensive coverage across another two patients, and they also exhibited retinal restoration. So we now have three cases that have exhibited retinal restoration. Now its not a one-time thing we have some understanding, we could reproduce it, its quite clear.

One of the trial participants atrophy originally got smaller but now has grown back to the size it was three years ago meaning that essentially her AMD had been halted.

In the three years since I had the operation, the eye that was operated on has not deteriorated which I reckon is almost miraculous, said the patient, Sonia Cohen. Its really amazing. I cant imagine what my life would have been like if I had continued to deteriorate.

Culley says that with OpRegens clinical research thus far, his team has learned that being more aggressive with where they place the cells leads to better outcomes.

Now we know that so we can repeat that. I think over time, it will get better and better, and Id be really delighted if we get up to the point where it becomes like LASIK surgery, which is close to 100% success rate.

Later this year, Lineage is planning to discuss the design of its next trial with the US Food and Drug Administration. For now, the three cases of retinal restoration or reversal of AMD have provided a vital proof of concept.

We want to go as quickly as possible into the next study, and having the backing of this reversal is very exciting because, from a statistical perspective, we probably dont need 2,000 patients were talking about hundreds of patients.

On the future of cell therapy, Culley says it is a field that is quickly maturing. It used to be like the wild west but now companies like ours are doing these rigorous clinical trials to find out ways to control it and dry AMD is a really cool place to start, he says. But, there are 199 other cell types that we could investigate so there is a long-term medical story here that is really about ushering in a new branch of medicine manufacturing cells, transplanting them that Im really excited to be part of.

Dry AMD is not the firms only focus; it has also embarked on clinical trials for VAC2, an allogeneic dendritic cell therapy currently enrolling a Phase I trial for the treatment of non-small cell lung cancer and has treated 25 people with spinal cord injuries with its candidate OPC1 in a Phase I/II trial, which Culley says has been very emotional.

I know if I live long enough my retina cells are going to die off; if I smoke four packs of cigarettes [a day] I will get lung cancer, but nobody expects to get a spinal cord injury. All of these young people who fall off mountain bikes or get in car crashes and their lives are turned upside down. Their ability to just get some hand control to be able to move their wheelchair thats mobility and independence so if we can help regain mobility, thats freedom so I think the spinal cord program is really powerful.

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OpRegen: tackling the leading cause of blindness with cell replacement - Clinical Trials Arena

The CAR T-cell therapy (Chimeric Antigen Receptor) has been billed as a radical new way to cure cancer.

But since the US FDA approved the first cell-gene therapy to treat leukemia in 2018, the technology has not really caught on in the mainstream.

So is there a place for CAR-T therapy within the wider immuno-oncology market?

According to the latest data from Grandview Research, the market is about to explode from US$1 billion in 2020 to US$20.3 billion by 2028, with two million patients in 10 years.

Australia is also starting to adopt the technology. Earlier this month, the government approved the use of Yescarta, a type of CAR-T cell therapy thats used to treat patients with certain types of lymphoma a form of blood cancer.

Its expected up to 300 Australians per year will benefit from these therapies, which are available at selected public hospitals.

A handful of ASX-listed companies are also rapidly advancing their research into the T-cell therapy space.

Immutep (ASX:IMM) announced on Friday a patent grant in China for the use of its LAG-3 antibody molecules.

LAG-3 (or Lymphocyte Activation Gene-3) is a gene that codes a protein involved in the regulation of the immune system.

Immutep is currently studying how the LAG-3 immune control mechanism reacts with its lead drug,eftilagimod alpha.

Another ASX-listed company, AdAlta (ASX:1AD), has also just flagged its intention to enter the CAR-T space, signing a collaboration agreement with Carina Biotech last week.

AdAlta mainly focuses on its lead drug candidate, i-body AD-214, to treat fibrosis, and its entrance into CAR-T is a welcome boost for the sector.

Stockhead caught up with AdAlta CEO, Dr Tim Oldham, to ask him how CAR-T works and what the current challenges are.

CAR-T is all about reprogramming the bodys own immune system to fight cancer, Oldham explained to Stockhead.

T-cells themselves are a type of white blood cells found naturally, which patrol the body for signs of disease.

T-cells do this by using the receptors found on their surface to latch on to antigens of cancer cells, which are then killedby injecting them with toxins.

The trouble is, cancer cells are smart and can avoid detection.

The cancer cells are really good at hiding from the immune system by disguising themselves so the T-cells dont recognise them, Oldham said.

And so our immune system doesnt do a great job of tracking down cancer.

What the CAR-T cell therapy does is to reprogram the T-cells to break through the cancer cells defences.

The first step of the process is to collect white blood cells from the patients blood using a procedure called leukapheresis.

When the white blood cells are removed and T-cells separated, they are sent to the lab which then genetically modifies the patients T-cells receptors to recognise the specific cancer.

Its essentially like putting on a GPS on the T-cells back into your body, says Oldham.

According to Oldham, there are a few obstacles that need to be overcome before CAR-T therapies could really go mainstream.

Firstly, the therapy is currently only effective on blood cancers, and not on solid tumours.

Weve been really successful with blood cancers because its relatively easy to find tumour-specific antigens in blood cells, explained Oldham.

Solid tumours on the other hand, are much harder for three reasons.

Firstly, theres not as many tumour specific antigens known and the tumour cells dont necessarily express them all, so its much harder to zoom in and target these solid tumour cells.

The second problem is getting the T-cells into the solid tumour. With a blood cancer its easy because theyre all in the blood anyway.

The third challenge with targeting solid tumours, Oldham explained, is that a tumour is an environment thats designed to suppress the immune system to stop working. And this just makes it much harder for CAR-T cells to find and penetrate solid tumours.

Oldham says AdAltas collaboration with Carina will potentially address all three problems.

Our technology which we call i-bodies will enable us to really target the solid tumours, he said.

Oldham explained that AdAltas i-bodies are the next generation antibodies that are genetically modified and are approximately one-tenth the size of monoclonal antibodies.

The Carina technology meanwhile, will overcome the local penetration of the tumour in the immuno-suppression, and that combined with our i-bodies will give us a new way to hit CAR-T on solid tumours.

The other obstacle to mass adoption of CAR-T is the cost of these therapies.At the moment, prices are in the vicinity of US$400,000 for a single dose.

This is because every dose has to be made specific for each patient, Oldham said.

Another challenge is the safety, and there has been examples where patients have experienced really severe adverse events.

The safety issue is currently significant, but equally youre looking at a disease thats going to kill someone anyway.

Other ASX companies that focus on CAR-T therapies include Chimeric Therapeutics (ASX:CHM), Prescient Therapeutics (ASX:PTX), and Imugene (ASX:IMU).

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CAR-T therapy is potentially a $20bn market. Heres how it works and the ASX players in the space - Stockhead

NEW YORK, Aug. 30, 2021 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today reported operational highlights and financial results for the fourth quarter and full-year ended June 30, 2021 (FY2021).

During this calendar year we made significant progress in both regulatory and clinical outcomes for our lead product candidate, remestemcel-L, after experiencing a disappointing set-back last year said Silviu Itescu, Chief Executive of Mesoblast. We are pleased with recent recommendations by FDAs CBER to meet with the review team and address remaining CMC items for remestemcel-L in the treatment of steroid-refractory acute graft versus host disease in children. Additionally, our most recent meeting with the FDA has provided clarity on the pathway towards an emergency use authorization for remestemcel-L in the treatment of COVID ARDS.

Operational Highlights

Remestemcel-L Outcome of recent meeting with FDA on regulatory pathway for emergency use authorization in the treatment of COVID-19 ARDS:

Remestemcel-L in the treatment of steroid-refractory acute graft versus host disease (SR-aGVHD) in children:

Rexlemestrocel-L in the treatment of chronic heart failure and chronic low back pain:

Manufacturing

Financial Highlights

DETAILED CLINICAL ACTIVITIES FOR THE FISCAL YEAR FY2021

Remestemcel-L

Acute Respiratory Distress Syndrome due to COVID-19

Mesoblast recently presented results from the randomized controlled trial of remestemcel-L in 222 ventilator-dependent COVID-19 patients with moderate/severe acute respiratory distress syndrome (ARDS) at the biennial Stem Cells, Cell Therapies, and Bioengineering in Lung Biology and Diseases conference hosted by the University of Vermont, Burlington, VT, and at the International Society for Cell & Gene Therapy (ISCT) Scientific Signatures Series event on Cell and Gene-Based Therapies in Lung Diseases and Critical Illnesses.

The presented data included improved respiratory function in patients treated with remestemcel-L, as well as 90-day survival outcomes showing remestemcel-L significantly reduced mortality by 48% at 90 days compared to controls in a pre-specified exploratory analysis of 123 treated patients under 65 years old. The trial had been halted after the third interim analysis since the 30-day primary endpoint would not be attained.

Key presentation findings were:

Mesoblast plans to move forward with an additional Phase 3 trial in COVID-19 ARDS with the next step being to agree with the FDA the final protocol and potency assay.

Inflammatory Bowel Disease Crohns Disease and Ulcerative Colitis

A randomized, controlled study of remestemcel-L delivered by an endoscope directly to areas of inflammation and tissue injury in up to 48 patients with medically refractory Crohns disease and ulcerative colitis commenced at Cleveland Clinic in October 2020. The investigator-initiated study is the first in humans using local cell delivery in the gut and will enable Mesoblast to compare clinical outcomes using this delivery method with results from an ongoing randomized, placebo-controlled trial in patients with biologic-refractory Crohns disease where remestemcel-L was administered intravenously.

Rexlemestrocel-L

Chronic Heart Failure

The results from the landmark DREAM-HF randomized controlled trial in 537 treated patients with chronic heart failure with reduced left ventricular ejection fraction (HFrEF) who received rexlemestrocel-L (REVASCOR) or control sham, demonstrated that a single dose of rexlemestrocel-L resulted in substantial and durable reductions in heart attacks, strokes, and cardiac deaths. The trials primary endpoint of reduction in volume overload related hospitalizations was not achieved. The results of this trial identify New York Heart Association (NYHA) class II HFrEF patients as the optimal target population for greatest rexlemestrocel-L treatment effect, and therefore a focus for developing rexlemestrocel-L in the largest market in heart failure.

The incidence of heart attacks and strokes were reduced by 60% over a median follow-up period of 30 months following a single dose of rexlemestrocel-L in the entire population of 537 treated patients. The incidence of death from cardiovascular causes was reduced by 60% in the 206 patients with NYHA class II disease, a significant reduction which was evident in both ischemic and non-ischemic subgroups as well as diabetic and nondiabetic patients.

The results also show that the NYHA class II patients in the control group, following an initial period of approximately 20 months of disease stability, progressed to cardiac death rates in-line with NYHA class III patients. NYHA class II patients treated with a single dose of rexlemestrocel-L did not show such cardiac death progression.

The combination of the three pre-specified outcomes of cardiac death, heart attack or stroke into a single composite outcome - called the three-point major adverse cardiovascular events (MACE) is a well-established endpoint used by the FDA to determine cardiovascular risk. Rexlemestrocel-L reduced this three-point MACE by 30% compared to controls across the entire population of 537 treated patients. In the NYHA class II subgroup of 206 patients, rexlemestrocel-L reduced the three-point MACE by 55% compared to controls.

Mesoblast expects feedback from the FDA in the next quarter on the potential pathway to US regulatory approval for rexlemestrocel-L in patients with chronic heart failure.

Chronic Low Back Pain due to Degenerative Disc Disease

The results from the randomized controlled trial of its allogeneic mesenchymal precursor cell (MPC) therapy rexlemestrocel-L in 404 enrolled patients with chronic low back pain (CLBP) due to degenerative disc disease (DDD) refractory to conventional treatments indicate that a single injection of rexlemestrocel-L+hyaluronic acid (HA) carrier may provide a safe, durable, and effective opioid-sparing therapy for patients with chronic inflammatory back pain due to degenerative disc disease, and that greatest benefits are seen when administered earlier in the disease process before irreversible fibrosis of the intervertebral disc has occurred. The trial's composite outcomes of pain reduction together with functional responses to treatment were not met by either MPC group.

The rexlemestrocel-L+HA treatment group achieved substantial and durable reductions in CLBP compared to control through 24 months across the entire evaluable study population (n=391) compared with saline controls. Greatest pain reduction was observed in the pre-specified population with CLBP of shorter duration than the study median of 68 months (n=194) and subjects using opioids at baseline (n=168) with the rexlemestrocel-L+HA group having substantially greater reduction at all time points (1, 3, 6, 12, 18 and 24 months) compared with saline controls. There was no appreciable difference in the safety of MPC groups compared to saline control over the 24-month period of follow-up in the entire study population. In subjects using opioids at baseline, the MPC+HA demonstrated a reduction in the average opioid dose over 24 months, while saline control subjects had essentially no change.

There is a significant need for a safe, efficacious, and durable opioid-sparing treatment in patients with chronic low back pain due to severely inflamed degenerative disc disease. Mesoblast has filed a request and expects to receive feedback from the FDA on the pathway to US regulatory approval in patients with chronic low back pain due to degenerative disc disease.

Intellectual Property

Mesoblast has an extensive patent portfolio with over 1,000 patents and patent applications across 77 patent families, and patent terms extending through 2041. These patents cover composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells, and provide strong commercial protection for our products in all major markets, including the United States, Europe, Japan and China. During the fiscal year Mesoblast has significantly expanded its patent portfolio, focusing on areas of its strategic commercial interests.

Licensing agreements with JCR, Grnenthal, Tasly and Takeda highlight the strength of Mesoblast's extensive intellectual property portfolio covering mesenchymal lineage cells. Mesoblast will continue to use its patents to prosecute its commercial rights as they relate to its core strategic product portfolio. When consistent with the Companys strategic objectives, it may consider providing third parties with commercial access to its patent portfolio.

DETAILED FINANCIAL RESULTS

Financial Results for the Year Ended June 30, 2021 (FY2021)

In August we entered into a contractual amendment to extend the interest-only period of its current senior debt facility to at least January 2022 and as a result no loan repayments will be required prior to January 2022. Mesoblast is in active discussions to refinance the facility.

We expect to recognize the existing US$21.9 million of remestemcel-L pre-launch inventory on the balance sheet if we receive FDA approval.

As a result of the above and other remeasurements on revaluation of assets and liabilities, the loss after tax for FY2021 was US$98.8 million compared to US$77.9 million for FY2020. The net loss attributable to ordinary shareholders was 16.33 US cents per share for FY2021, compared with 14.74 US cents per share for FY2020.

Conference Call

There will be a webcast today, beginning at 7.00pm EDT (Monday, August 30, 2021); 9.00am AEST (Tuesday, August 31). It can be accessed via:https://webcast.boardroom.media/mesoblast-limited/20210826/NaN61036c41df5665001c97fc67

The archived webcast will be available on the Investor page of the Companys website: http://www.mesoblast.com

About Mesoblast

Mesoblast is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of late-stage product candidates which respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.

Mesoblast has a strong and extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets. The Companys proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has completed Phase 3 trials of rexlemestrocel-L for advanced chronic heart failure and chronic low back pain. Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid refractory acute graft versus host disease and moderate to severe acute respiratory distress syndrome. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

References / Footnotes

Forward-Looking Statements

This announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of Mesoblasts preclinical and clinical studies, and Mesoblasts research and development programs; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblasts ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblasts product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblasts product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblasts ability to enter into and maintain established strategic collaborations; Mesoblasts ability to establish and maintain intellectual property on its product candidates and Mesoblasts ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblasts expenses, future revenues, capital requirements and its needs for additional financing; Mesoblasts financial performance; developments relating to Mesoblasts competitors and industry; and the pricing and reimbursement of Mesoblasts product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

For more information, please contact:

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Operational Highlights and Financial Results for the Year Ended June 30, 2021 - GlobeNewswire

Cryo-Cell International, Inc. (Symbol CCEL), The Leader In Cord Blood And Cord Tissue Cryopreservation Services, Today Announced That The Companys Common Stock Has Been Approved For Listing On The Nasdaq Capital Market (Nasdaq).

Trading to Begin August 31, 2021 with Current Ticker CCEL

MIAMI, Aug. 30, 2021 (GLOBE NEWSWIRE) -- Cryo-Cell International has met Nasdaqs stringent financial, liquidity and corporate governance listing requirements. As a result, Cryo-Cell will be listed on Nasdaq as of Tuesday, August 31st 2021. This is expected to improve the liquidity of Cryo-Cell common stock, broaden its institutional shareholder base and ultimately enhance long-term shareholder value.

Cryo-Cells Chairman of the Board and Co-Chief Executive Officer, David Portnoy, stated: I am pleased to announce Cryo-Cells uplisting to Nasdaq, a major financial milestone that the Company had been considering, as previously stated several months ago. I believe that the significant gap between Cryo-Cells current market valuation and our intrinsic value should narrow as our visibility in the investment community grows, and this is a step in that direction. Cryo-Cell International has transformed in 2021 from being a profitable cord blood bank to a fully integrated, biopharmaceutical company that is expected to provide much needed access to experimental treatments for patients at the upcoming Cryo-Cell Institute for Cellular Therapies.

About Cryo-Cell International, Inc.

Founded in 1989, Cryo-Cell International, Inc. is the worlds first private cord blood bank. More than 500,000 parents from 87 countries have entrusted Cryo-Cell International with their babys cord blood and cord tissue stem cells. In addition to its private bank, Cryo-Cell International has a public banking program in partnership with Duke University. Cryo-Cells public bank has provided cord blood for more than 600 transplantations and operates cord blood donation sites across the U.S in prominent hospitals such as CedarsSinai Hospital in Los Angeles and Baptist Hospital in Miami. Cryo-Cells facility is FDA registered, cGMP-/cGTP-compliant and licensed in all states requiring licensure. Besides being AABB accredited as a cord blood facility, Cryo-Cell was also the first U.S. (for private use only) cord blood bank to receive FACT accreditation for adhering to the most stringent cord blood quality standards set by any internationally recognized, independent accrediting organization. Cryo-Cell owns the exclusive rights to PrepaCyte-CB, the industrys most advanced cord blood processing technology.

Cryo-Cells mission has been to provide clients with state-of-the-art cord blood and cord tissue cryopreservation services, raise awareness of the opportunity for expectant parents to bank or donate their babys cord blood and support the advancement of regenerative medicine. In February 2021, Cryo-Cell entered into a license agreement with Duke University that transformed Cryo-Cell into an autonomous, vertically integrated cellular therapy company that will be able to treat patients.

For more information, please visitIR.cryo-cell.com

Forward-Looking Statement

Statements herein the terms believes, intends, projects, anticipates, expects, and similar expressions as used are intended to reflect forward-looking statements of the Company. The information contained herein is subject to various risks, uncertainties and other factors that could cause actual results to differ materially from the results anticipated in such forward-looking statements or paragraphs, many of which are outside the control of the Company. These uncertainties and other factors include the impact of the COVID-19 pandemic on our sales, operations and supply chain, the success of the Companys global expansion initiatives and product diversification, the Companys actual future ownership stake in future therapies emerging from its collaborative research partnerships, the success related to its IP portfolio, the Companys future competitive position in stem cell innovation, future success of its core business and the competitive impact of public cord blood banking on the Companys business, the success of the Companys initiative to expand its core business units to include biopharmaceutical manufacturing and operating clinics, the uncertainty of profitability from its biopharmaceutical manufacturing and operating clinics, the Companys ability to minimize future costs to the Company related to R&D initiatives and collaborations and the success of such initiatives and collaborations, the success and enforceability of the Companys umbilical cord blood and cord tissue license agreements, together with the associated intellectual property and their ability to provide the Company with royalty fees, and those risks and uncertainties contained in risk factors described in documents the Company files from time to time with the Securities and Exchange Commission, including the most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and any Current Reports on Form 8-K filed by the Company. The Company disclaims any obligations to subsequently revise any forward-looking statements to reflect events or circumstances after the date of such statements.

For more information, please contact:Corporate Communications / Investor RelationsDiane Glanz, Rph, PharmD. Telephone: +1 (813) 749-2195Email: Investors@Cryo-Cell.com

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Cryo-Cell International Announces Uplisting to The Nasdaq Capital Market - GlobeNewswire

Company Developing FDA Cleared Phase III Cellular Therapy for COVID-19 Aims to Work with Regulators for Ultra-Rapid Clinical Translation

ELK CITY, Idaho, Aug. 30, 2021 /PRNewswire/ -- Therapeutic Solutions International, Inc., (OTC Markets: TSOI), announced today filing of an Investigational New Drug (IND) application with the Food and Drug Administration (FDA) to initiate a clinical trial of its StemVacs-V immunotherapy in patients with metastatic breast cancer.

On May 24th, 2021, the Company announced progress in its preclinical experiments supporting the use of the immunotherapy in human trials. Part of the experiments demonstrated superior activity of StemVacs-V to other immune system-based approaches that were cleared by the FDA to literally "choke" tumors to death by blocking their blood supply1.

"I am honored to be leading medical efforts in bringing this game-changing approach to patients with breast cancer," said Dr. James Veltmeyer, Chief Medical Officer of the Company. "The previous efforts to develop an immunotherapy to kill tumor blood vessels have yielded drugs such as Avastin, which despite limitations, possesses multi-billion dollars in annual sales. In my opinion the StemVacs-V approach is superior to others because it induces more potent and more diverse immune responses targeting cancer blood vessels."

"The filing of today's IND is a significant milestone demonstrating the ability of Therapeutic Solutions International to rapidly move a diverse immunotherapy product pipeline through the drug development cycle," said Famela Ramos, Vice President of Business Development. "We anticipate multiple synergies of our developmental efforts as our candidate's advance forward, with our lead candidate having been cleared by the FDA to initiate Phase III clinical trials.

"Through working with our advisors such as Dr. Francesco Marincola, Dr. Santosh Kesari, and Francisco Silva, who have all published on utilization of First-Generation tumor angiogenesis blocking immunotherapies2,3, we anticipate learning from previous experiences and to rapidly move forward in taking this approach to patients," said Timothy Dixon, President and CEO of the Company and co-inventor of the StemVacs platform. "We thank our collaborators who have worked tirelessly to make this IND filing a possibility in such a rapid manner."

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About Therapeutic Solutions International, Inc.Therapeutic Solutions International is focused on immune modulation for the treatment of several specific diseases. The Company's corporate website is http://www.therapeuticsolutionsint.com, and our public forum is https://board.therapeuticsolutionsint.com/

1 Therapeutic Solutions International Optimizes Targeting the "Achilles Heel" of Cancer Using Second Generation Tumor Endothelial Targeting Vaccine StemVacs-V iPSC (prnewswire.com)2 Safety of targeting tumor endothelial cell antigens | Journal of Translational Medicine | Full Text (biomedcentral.com)3 Cancer anti-angiogenesis vaccines: Is the tumor vasculature antigenically unique? | Journal of Translational Medicine | Full Text (biomedcentral.com)

ir@tsoimail.com

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Therapeutic Solutions International Files Investigational New Drug Application with FDA for Cancer Blood Vessel Targeting Immunotherapy StemVacs-V to...

Shoreline Launches into Era of Cell Therapy with $4 Billion in Partnerships

Shoreline Biosciences, an immunotherapy company formed in 2020 during the early days of the COVID-19 pandemic, was propelled into the proverbial catbirds seat this summer with two mega-deals, and now plans to double its employee base from 50 to 100 in the coming six to nine months.

Shoreline Biosciences is developing an off-the-shelf, targeted, allogeneic approach to natural killer (NK) and macrophage cell therapies, which Kleanthis G. Xanthopoulos, Ph.D., chairman and CEO, believes is one-of-a-kind.

This summers deals with Kite Pharmaceuticals, a Gilead Company, and BeiGene totaling $4 billion and boasting combined upfront payments of over $120 million to further develop its induced pluripotent stem cell (iPSC) programs are potent validation of Shorelines technology as well as its management. They go a long way toward ushering in what Xanthopoulos called the era of cell therapy.

Shorelines growing success is based on two pillars: solid science and experienced management.

You have to have the high science, but also enormous institutional know how, Xanthopoulos told BioSpace. These cells are finicky. There are significant challenges in manipulating them. Our founders have 20 years experience in this field.

BeiGene and Kite share our vision, and have extensive experience themselves BeiGene is incredible in protein engineering and Kite pioneered CAR T therapies, he said. So, when they chose to partner with Shoreline, they had performed competitive analysis and were saying, in essence, we believe in you and your capabilities.

These partnerships allow Shoreline to fast-forward its platforms.

For a company to bring so much capital and synergy to work is incredibly powerful, unique, and differentiated, Xanthopoulos said. It is difficult to find another early-stage preclinical company that has this kind of recognition and validation.

The new clinical data from Fate Therapeutics regarding its NK and T cell therapies help the field, too.

The science is very comprehensive, and we believe it will bring those programs forward, renewing interest in pluripotent stem cells and NK cells, Xanthopoulos surmised.

Shoreline Biosciences is developing off-the-shelf allogeneic therapies, bringing the benefit of stem cell therapies to many more patients than is possible with autologous transplants.

For autologous therapies, Xanthopoulos explains, It is costly to take patient cells, manipulate them in the lab, add guided chimeric antigen receptors, and return them to the patients There also are issues with rejection, as well as the more significant issues of cytokine release syndrome. Patients with aggressive tumors dont have the four to six weeks that method requires.

Shorelines allogeneic approach, therefore, creates targeted, off-the-shelf stem cell therapies.

The only effective way to do this is to start with pluripotent stem cells that can differentiate into more than 200 cell types, Xanthopoulos said. At Shoreline, we differentiate into hemopoietic cells. We are focused on NK cells and macrophages.

We have, basically, released the brakes for proliferating and activating the NK cells, he said, by editing out a negative regulator of activation and proliferation to create what essentially is a supercharged cell that is more metabolically fitted.

Preclinical studies in animals show the cells resist exhaustion, conferring a better pharmacokinetic profile and the need for fewer cytokines. Importantly, those efforts have been validated by independent researchers, in many published papers.

Results indicate the combination of pluripotent stem cells and NK cells results in a greater ability to kill various tumor cells using 5- to 10-fold less IL2 and IL15. Consequently, Xanthopoulos said, The overall therapy is less expensive.

Shoreline Biosciences plans to take this program into early human trials in the second half of 2022.

In 2023, we expect to file one or two Investigational New Drug (IND) applications, he said.

The benefit of an allogeneic iPSC approach to therapeutics development is clear.

Source: BioSpace

You can perform a lot of genomic edits at the pluripotent state without a great many technical problems, he noted.

For example, transducing macrophages with chimeric antigen receptors (CAR) requires specialized vectors and results in low yields. But, if you start with pluripotent stem cells, you can make the modifications, isolate a single clone with the characteristics you want, and generate trillions of cells. With one patient dose requiring approximately 100 million cells, this method can lower the approximate $400,000 cost of therapy substantially.

To do this for NK cells, Shoreline Biosciences created a unique, powerful engine and then determined how to decorate the resulting cells. After considering such issues as cell signaling, targeting, and toxicity, Xanthopoulos said, We engineered what you can think of as hooks where antibodies recognize and interact tightly, for higher killing activity.

As Xanthopoulos, a serial entrepreneur who has founded and operated four previous biotech companies, told BioSpace, Ive never been more excited. Stem cell therapies are proven. They are the next frontier of medicine.

They arent in the distant future, either. I think the era of cell therapies is here and now, and will dominate the landscape going forward. If we learn to make less immunogenic therapies, stem cell therapeutics will continue to increase momentum.

As Shoreline looks to the future, it is building an expansive team and is continuing to hire in all categories. We plan to double to about 100 employees in the next six to nine months, and have several outstanding investors onboard.

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Shoreline Launches with $4 Billion in Partnerships PharmaLive - PharmaLive

Christopher R. Flowers, MD, discusses the current therapies used for patients with relapsed/refractory diffuse large B-cell lymphoma and goes into specifics of the L-MIND trial after the long-term follow-up.

Christopher R. Flowers, MD,department chair of the Department of Lymphoma/Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, discusses the current therapies used for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) and goes into specifics of the L-MIND trial (NCT02399085) after the long-term follow-up.

There are multiple therapies and therapy combinations approved in this setting, including chimeric antigen receptor (CAR) T-cell therapy. The 2021 American Society of Clinical Oncology (ASCO) Annual Meeting provided some updated for trials in this space, including the multicenter, open-label, single-arm, phase 2 study L-MIND trial.

Transcription:

0:08 | What we have, however, seen is a dramatic change in the numbers of therapies that are available in the relapse setting. That's with the advent of polatuzumab [Polivy] combined with bendamustine and rituximab [Rituxan], tafasitamab [Monjuvi] combined with lenalidomide [Revlimid], and now loncastuximab tesirine [Zynlonta] approved in that third or later line space of therapy, and the advent of 3 different CAR T-cell therapies that are now approved in the relapse setting for DLBCL. Then selinexor [Xpovio] that's also approved in that space. So really a whole host of options. At this year's ASCO meeting, we saw an update on some of those trials, and particularly the one looking at the combination of tafasitamab and lenalidomide, or the L-MIND trial.

What were the design and efficacy of the L-MIND trial?

1:08 | When we look at the L-MIND trial, it's a trial that was published with updated results in Lancet Oncology in 2020 describing a patient population of about 81 patients. When you look at the characteristics of the population, the median age at the time of enrollment was about 72 years, which is relatively similar to what we would see for a general population of patients with DLBCL; 11% of those patients had a prior stem cell transplantation. This is generally an older patient population who did not have transplant as part of their course of care. The majority of patients had a median of 2 prior lines of therapy, so still relatively early in their treatment cycle. When you look at response rates, and particularly when you look at response rates by numbers or prior lines of therapy, what was presented in the updated results at ASCO showed for those patients who had 1 prior line of therapy, that the complete response rate was 48% versus those who had 2 or more prior lines of therapy, and the trial was divided about half and half between those 2 different groups. Those who had 2 or more prior lines of therapy had a complete response rate of 33%. The duration of response appeared to be quite durable for both groups being more than 44 months in both groups of responders.

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Reviewing Current Therapies in Relapsed DLBCL and the L-MIND Trial - Targeted Oncology