Category Archives: Stem Cell Therapy

The first patient with lymphoma received treatment with AB-205 in the phase 3 E-CELERATE trial assessing the novel cell therapy with high-dose chemotherapy and blood stem cell transplantation, according to a press release from Angiocrine Bioscience, the manufacturer of the therapy.

AB-205 is a genetically engineered cell therapy that is administered intravenously and consists of allogeneic engineered human endothelial cells. It is currently being assessed in the E-CELERATE trial, in which researchers will evaluate the efficacy and safety of this cell therapy for the treatment of damaged organ vascular stem cell niches from high-dose chemotherapy. Researchers will also see whether the cell therapy can prevent the progression of severe multiorgan complications, which can potentially prolong hospitalization and can be life threatening, according to the release.

According to the trials ClinicalTrials.gov listing, researchers estimate to enroll 148 patients aged 40 years and older with lymphoma. Patients who will be assigned treatment with AB-205 or placebo, and both groups will be treated with standard-of-care preventive and supportive therapies. All patients enrolled in the trial will be candidates for high-dose chemotherapy and autologous hematopoietic cell transplantation.

We are excited to initiate this pivotal phase 3 study, said Dr. Paul Finnegan, chief executive officer of Angiocrine Bioscience, in the release. We look forward to continuing to work with many of the leading cancer centers in the United States as we advance into the final clinical stages of this exciting program.

Patients with lymphoma are often treated with high-dose chemotherapy followed by blood stem cell transplantation with the intention to cure, according to the trials ClinicalTrials.gov listing. Despite this approach, high-dose chemotherapy may cause damage to health tissues resulting in complications related to the immune system, blood, gastrointestinal systems and other organs.

The estimated study completion date for the E-CELERATE trial is January 2023, according to ClinicalTrials.gov.

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First Patient With Lymphoma Receives Novel Cell Therapy in Large-Scale Trial - Curetoday.com

An application seeking the approval of a new indication for the combination of ibrutinib, bendamustine, and rituximab in adult patients with previously untreated mantle cell lymphoma has been submitted.

A Type II variation application seeking the approval of a new indication for the combination of ibrutinib (Imbruvica), bendamustine, and rituximab (Rituxan) in adult patients with previously untreated mantle cell lymphoma (MCL) who are not candidates for autologous stem cell transplant (ASCT) has been submitted to the European Medicines Agency (EMA).1

Findings from the phase 3 SHINE trial (NCT01776840), which examined the combination in patients with newly diagnosed MCL aged 65 years or older, is the basis of the application. Results showed that the combination demonstrated a significant improvement in progression-free survival (PFS) compared to rituximab alone.

MCL can be a difficult blood cancer to treat, and despite progress in this area of the last few years, an unmet need remains for new treatment approaches, Edmond Chan, MBChB, MD (Res), EMEA therapeutic area lead hematology, at Janssen-Cilag Limited, stated in a press release. This submission to the EMA is a testament to our commitment to deepening the impact ibrutinib can have for patients and represents an important step toward providing patients and health care professionals with the addition of targeted therapy to standard therapy.

The trial included 523 participants with MCL who had clinical stage II, III, or IV disease by Ann Arbor classification.2Eligibility for participation was open to patients with at least 1 measurable site of disease, and an ECOG performance status of 0 or 1. Additionally, patients could not have received prior therapies for their disease.

Patients were randomized 1:1 to receive either ibrutinib or placebo, both in combination with an open-label bendamustine plus rituximab background treatment for a maximum of 6 cycles. Of the participants who achieved a complete (CR) or partial response (PR), open-label background therapy with rituximab maintenance was continued every second cycle for up to 12 additional doses. Patients then received ibrutinib or placebo in addition to this background treatment.

In the experimental treatment arm, bendamustine was given intravenously (IV) at a dose of 90 mg/m2 on days 1 and 2 of cycles 1 through 6, and IV rituximab was delivered at a dose of 375 mg/m2on day 1 of cycles 1 through 6. If either CR or PR was achieved at this time, patients were administered rituximab at the same dose on day 1 of every second cycle for up to 12 additional doses. Additionally, ibrutinib was given orally, once a day at a dose of 560 mg, continually starting on day 1 of cycle 1. Either combination was given until progressive disease, intolerable toxicity, or study completion.

If stable disease following initial chemoimmunotherapy was achieved, patients continued to receive ibrutinib or placebo until progressive disease, intolerable disease, or study end. Patients who experienced disease progression needed to discontinue study treatment.

The primary end point of the trial was PFS, with secondary end points including overall survival, overall response rate, minimal residual disease negativity rate, duration of response, time to next treatment, number of participants affected by adverse events, toxicity, and other various pharmacokinetic measures.

The trial, which met its primary end point, will have its data shared at an upcoming medical conference, according to a press release issued by the Janssen Pharmaceutical Companies of Johnson & Johnson.

As the first approved BTK inhibitor, ibrutinib has now been used to treat more than 250,000 patients globally. It is also the first BTK inhibitor to be studied as a frontline treatment option for patients with MCL. We are committed to the continued development of ibrutinib in B-cell malignancies where unmet needs remain in our efforts to make meaningful differences and change outcomes for patients, Craig Tendler, MD, global head of late development, Diagnostic & Medical Affairs, Hematology & Oncology, at Janssen Research & Development, LLC, added in the press release.

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Approval Sought for Ibrutinib Combination in Patients With Mantle Cell Lymphoma - Targeted Oncology

Over the past seven years, Bayers investment arm has infused 50-plus companies with more than 1.3 billion. Leaps by Bayer is accelerating its dealmaking pace and Bayer is committing another 1.3 billion, which the multinational corporation estimates will fuel its investment vehicle for two more years.

Bayer announced the capital commitment on Friday during the companys Breakthrough Innovation Forum, an event that covered the corporations plans in healthcare and agriculture. Those two fields were the core focus areas when Bayer formed Leaps in 2015, aiming to invest in companies developing breakthrough solutions to big challenges facing humanity, challenges that the corporation termed leaps. At the start, Bayer identified 10 leaps. The healthcare leaps span genetic diseases; organ and tissue replacement; cancer; neurological disorders; autoimmune diseases and inflammation; and the application of data to health.

Leaps has not tipped its hand on its plans for future investments. But if Bayers dealmaking in recent years is any indication, cell and gene therapies are likely bets. In 2019, Bayer fully acquired BlueRock Therapeutics, a cell therapy developer it had formed three years earlier as a joint venture with venture capital firm Versant Ventures. A BlueRock cell therapy in development for Parkinsons disease began clinical testing last year. BlueRock develops off-the-shelf therapies from induced pluripotent stem cells. The experimental cell therapy DA01 is comprised of neurons that produce dopamine that Parkinsons patients lack. These cells are surgically transplanted in the brain, where its hoped they will produce dopamine and potentially offer a superior alternative to the older dopamine substitutes that are part of the current standard of care.

Bayers gene therapy investment includes the commitment of up to $4 billion to acquire AskBio, a gene therapy biotech whose pipeline most advanced programs are for Parkinsons and the neuromuscular disorder Pompe disease. And earlier this year, Bayer struck up a partnership that enables it to use Mammoth Biosciences CRISPR to develop new in vivo gene editing therapies. The initial focus of the Mammoth alliance is liver diseases. Bayer hasnt abandoned traditional small molecule drugs, but its toolbox for discovering them. Last August, Bayer paid $1.5 billion up front to acquire Vividion Therapeutics, a biotech whose technology finds binding pockets on proteins that had been considered undruggable.

In an interview during the J.P Morgan Health Care Conference in January, Christian Rommel, the head of research and development of Bayers pharmaceuticals division, told MedCity News that Bayers investments are part of a broader strategic transformation oriented around innovative new medicines, some of them in new modalities. Rommel said Bayer will look for additional acquisitions or partnerships that fit this strategy.

Its now in our DNA, he said. We will continue to look for things that enhance our capabilities and success of our pipeline.

Cell therapy investments by Leaps include Indapta, a biotech that is researching therapies employing natural killer immune cells. The most recent Leaps cell therapy investment is Affini-T Therapeutics. In late March, Leaps co-led a $175 million investment the startup, which is based on cell therapy research from the Fred Hutchinson Cancer Research Center.

Leaps has also deployed its cash toward artificial intelligence. It was among the investors in AI-based biotech company Recursion, which went public last year. Other companies whose AI drug discovery work has financial backing from Leaps include Dewpoint Therapeutics, which employs AI to develop biomolecular condensate drugs. Leaps has also shown interest in new approaches to developing protein drugs, co-leading the investment rounds of GRObio and Gandeeva Therapeutics.

The interest of Bayers investment arm in AI extends to healthcare software. Last summer, Leaps led a $90 million investment in Ada, a German startup that is developing AI-based software for checking symptoms. Leaps is also an investor in Transcarent, a healthcare navigation formed started by Livongo Health founder Glen Tullman.

Photo by Flicker user Bex Walton via a Creative Commons license

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Bayer reloads Leaps with 1.3 billion to step up investments in biotech innovation - MedCity News

While South Africans recently observed Human Rights Day, the notion of basic human rights continues to be a global topic of contention, particularly as the World Health Organisation (WHO) asserts that health is a fundamental human right.

The organisations director-general, Dr Tedros Adhanom Ghebreyesus, expands on this notion well, explaining that enjoying the highest attainable standard of health is a fundamental right of every person.

In SA, the publics right to health is protected by Section 27a of the constitution, which declares that everyone has the right to access to healthcare services. While this is embedded in our rights as citizens, it is often our misinterpretations and the failures of providers that lead to the collapse of such rights.

The right to adequate healthcare is particularly relevant when addressing dread diseases such as cancer, where specialised medical interventions can prolong or save a life. Most notable is the need for interventions to address more pressing cancers, such as hematologic malignancies, or blood cancers. These are orphan diseases and are not nearly as common as the more predominant types, such as breast and prostate cancer.

All cancers require immediate intervention, but once blood cancer is detected in a patient, the clock starts ticking to seek appropriate treatment. While conventional intensive treatments such as chemotherapy have proven useful, they can only drive the disease into remission and patients have a high likelihood of relapse.

Treatments such as blood stem cell therapy have a high probability of eradicating the disease. However, there have been challenges to the procedure in SA.

The department of health promulgated the Medical Schemes Act 131 in 1998, which featured an annexure that defines what stem cell transplantation is and how patients would qualify. It also noted that the act would be reviewed and updated every two years. However, this has not happened.

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Support for blood cancer patients is thin. This needs to change - TimesLIVE

AZALENA trial shows encouraging response rates, duration of response and survival including patients with myelodysplastic syndrome, chronic myelomonocytic leukemia, and acute myeloid leukemia with MDS-related changes.

Following allogenic stem cell transplant (allo-SCT), lenalidomide (Revlimid) at a dose of 5 mg a day can be added safely to the standard of care combination of azacitidine (Vidaza) and donor lymphocyte infusions (DLI) as salvage therapy for patients with relapse of myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML) with MDS-related changes, results from the phase 2 AZALENA study (NCT02472691) show.1

The open-label, single-arm, multicenter study investigated the safety and feasibility of lenalidomide in addition to the standard therapy of azacitidine and DLI as first salvage therapy for this patient population.

We have seen encouraging response rates, duration of response and survival including patients with early and/or hematologic relapse in AZALENA. Azacitidine, lenalidomide and DLI favorable modulated the balance between GvHD and [graft-versus-leukemia; GvL] and is an efficient treatment option for patients with myeloid neoplasms relapsing after allogeneic stem cell transplantation which should be further investigated in randomized trials, stated Thomas Schroeder, MD, assistant professor, section head, Department of Internal Medicine, Division of Hematology/Oncology, medical director of Radiation Oncology, University of New Mexico, during an presentation of the data at the 48th Annual EBMT Meeting.

AZALENA enrolled 50 participants aged 18-99 with first relapse of de novo or therapy-related MDS, CMML or AML in various participating centers across Germany.2 Other requirements for inclusion in the study included possibility of DLI, no previous therapy for relapse after allo-SCT, an ECOG status of 0-2, and no uncontrolled infection at the time of enrolment.

Of the patients enrolled in the trial, the median age was 63 years. Forty-six percent of the patients with AML (n = 23), 48% with MDS (n = 24) and 6% with CMML (n = 3). A majority of patients (72%) were not in remission at the time of transplantation but received standard-dose conditioning (68%).

Patients included in the study received a median of 275 treatment cycles and were given azacitidine as a standard of care at 75 mg for 7 days every 28 days for up to 8 cycles. DLIs were then administered after cycle 4, 6 and 8 at a dose of 5-10x105CD3+/kg (1st DLI), 1-5x106CD3/kg (2nd DLI) and 5-15x106CD3/kg (3rd DLI).

The investigational drug, lenalidomide, was also started on day 1 for 21 days every 28 days for 8 cycles at most. A starting dose of 2.5 mg of lenalidomide per day was given to the first 10 patients. If no dose limiting toxicity was observed at the time of a first interim analysis, the next 10 patients were to be treated with 5 mg per day. In case of no DLT after a second interim analysis, the remaining 30 patients were treated with 5 mg per day.

The primary end point of the study was to evaluate safety including the number, types, and severity of adverse events (AEs), graft versus host disease (GvHD) and hospitalization. The secondary end point of the study examined safety in regard to response, duration of response, and overall survival.

Findings revealed there to be an overall response rate of 56% (n = 28). The time to complete response was 113 days seen in the 50% of patients with a median of 4 cycles. A total of 20 patients with CR had received DLI. Of the patients included, 6% showed a partial response. The median OS for all patients was 21 months and the 1-year OS rate was 65%. Patients who achieved remission had a superior survival versus 9.7 months.

In regard to toxicity, almost a third of the patients had either grade 3 or 4 neutropenia (30%) or thrombopenia (38%) at the time of study entry. Of the 275 treatment cycles, 246 included 89% of patients receiving the combination therapy, and in 29 cycles, lenalidomide was omitted in 5 patients (11%).

Lenalidomide-related AEs which were grade 3 or higher were seen in some patients including 38% of patients (n = 19) having to be hospitalized at some point in the duration of the trial. Grade 3 AEs seen in patients included infections (20%), febrile neutropenia (10%), gastrointestinal disorders (6%), and nervous system disorders (6%). Grade 4 AEs consisted of infections (4%) and febrile neutropenia (2%).

Looking at GvHD, 30% of the patients suffered from acute GvHD and 38% chronic GvHD. Time to GvHD onset was approximately 112 days the majority of patients with GvHD (77%, n = 20) had received DLI.

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Addition of Lenalidomide to SOC Shows Feasibility, Safety in MDS, AML and CMML - Targeted Oncology

DetailsCategory: DNA RNA and CellsPublished on Wednesday, 23 March 2022 13:40Hits: 516

The BLA has been accepted for quality review and StemCyte anticipates licensure in 2023

BALDWIN PARK, CA, USA I March 22, 2022 I The BLA (Biologics License Application) for the "HPC-Cord Blood" product, was submitted to FDA on January 7th, 2022, and StemCyte was officially notified on March 8th, 2022, that the submission was acceptable to enter the biologics license quality review process.

The "HPC-Cord Blood" is an umbilical cord blood hematopoietic stem cell product intended for unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system. Since the first successful use of umbilical cord blood to treat Fanconi Anemia patients in 1988, there have been more than 40,000 successful umbilical cord blood transplants worldwide for the treatment of diseases of the hematopoietic and immune systems as well as inborn metabolic diseases.

In the past 20 years, StemCyte has provided more than 2,200 cord blood units for transplantation.

In the past 20 years, StemCyte has provided more than 2,200 cord blood units for transplantation to 1 in 20 patients worldwide who receive umbilical cord blood transplants. StemCyte's products consistently meet the quality standards of international accrediting bodies and are recognized and trusted as safe and effective by at least 350 transplant centers around the world including such well-known medical centers as: Duke University Hospital, UCLA Medical Center, Taiwan Chang Gung Memorial Hospital, National Taiwan University Hospital.

StemCyte is a regenerative cell therapy company that is developing cell therapy pipeline products as well as providing both public and private umbilical cord blood banking services. In addition to the US FDA approved Phase II multi-national and multi-center human clinical trial for its investigational spinal cord injury treatment, the cell therapy product lines also include several other human clinical trials in progress outside the US for treatment of acute ischemic stroke, chronic stroke, and cerebral palsy. Development of a cancer immunotherapy is planned to begin this year. StemCyte's cord blood bank is multi-ethnic with the matching rate for patients from different ethnic groups relatively high compared with other public cord blood banks. StemCyte considers umbilical cord blood banking its core competence, while actively pursuing new regenerative cell therapy indications. StemCyte's mission is to continue to develop its unique cord blood banking capabilities to achieve the goal of delivering lifesaving therapies to patients who suffered degenerative and other life-threatening diseases.

SOURCE: StemCyte

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StemCyte, a leading regenerative cell therapy company, has submitted its application for a biologics license (BLA) for its allogeneic umbilical cord...

Kenneth Research published a report onStem Cell Research in Indiawhich gives detailed analysis of the present market landscape. The report which is evaluated for the forecast period, i.e.,2022-2031, provides an in-depth analysis of the market, based on size, volume, latest trends, opportunities and challenges associated.

U.S. Market recovers fast; In a release on May 4th 2021, the U.S. Bureau and Economic Analysis and U.S. Census Bureau mentions the recovery in the U.S. International trade in March 2021. Exports in the country reached $200 billion, up by $12.4 billion in Feb 2021. Following the continuous incremental trend, imports tallied at $274.5 billion, picked up by $16.4 billion in Feb 2021. However, as COVID19 still haunts the economies across the globe, year-over-year (y-o-y) average exports in the U.S. declined by $7.0 billion from March 2020 till March 2021 whilst imports increased by $20.7 billion during the same time. This definitely shows how the market is trying to recover back and this will have a direct impact on the Healthcare/ICT/Chemical industries, creating a huge demand forStem Cell Research in IndiaProducts.

Get Sample Copy of This Report @https://www.kennethresearch.com/sample-request-10016694

According to the statistics by the World Bank, the current health expenditure (% of GDP) around the world increased from 9.08% in 2001 to 9.84% in 2018. Additionally, the current health expenditure per capita (current US$) increased from USD 492.99 in 2001 to USD 1110.84 in 2018. Moreover, growing concern for deaths caused due to various diseases and the need for treatment that can lower the crude death rate, which in the year 2019 recorded close to 7% (per 1000 people), are also anticipated to drive the market growth during the forecast period.

Final Report will add the analysis of the impact of COVID-19 on this industry.

The stem cell research in India is in its nascent stage and is gradually on a growth path of acceptance by people. The market is still in a phase of conducting research to establish itself as one of the best therapies for the widely prevalent incurable lifestyle diseases. Awareness campaigns and doctors are playing a key role educating people and especially would-be parents about the benefits associated with preserving stem cells.

The report begins with the introduction section which offers a brief insight of the concept of stem cell therapy and banking, the conventional sources of stem cells and the current and future therapeutic solutions for the most risky diseases. It then moves to the market overview section which provides an insight of the Indian stem cell therapy market, with highlight on the market size and growth. It also covers the market size and growth prospect of the stem cell banking market in India. In addition to these, the report gives a snapshot of the current and expected stem cell banking customers.

An analysis of the drivers explains the factors for growth of the industry that include favourable regulatory environment, high patient population, stem cell application in drug development, rising consumer awareness, a rise in medical tourism and an increase in research and development expenditure to ensure stem cell therapy as a viable treatment for the numerous diseases. The key challenges include high cost of therapy, capital intensive market and high development costs of stem cell resulting in slow progress of the market.

The next section of the report provides the progressive stages that the stem cell research is undergoing and the respective business prospect for each of the stages of business involved. The report then covers the key trends that are being observed in the stem cell research. Collaborations and partnerships are becoming a trend that is helping in healthy progress of the business, investments are pouring in from government, international organizations and pharmaceutical companies, serious efforts are being undertaken to raise awareness standards regarding stem cell therapy and the move is being popularized by stem cell banks by providing easy financing opportunities for banking stem cells.

Significant developments have emerged wherein new sources of stem cells have been identified dental stem cells and menstrual blood stem cells which have been discussed along with its benefits in comparison to cord blood stem cells.

The competition section gives overview of stem cell banks and stem cell research organizations in the country. The report highlights the features of the major players operating in the market in detail. It includes elaborate profile of the major players in the market along with their financial analysis. Porters Five Forces Analysis has been incorporated for a brief but effective understanding of the market scenario.

Thereafter, the report has a section on the recent developments that have taken place in the recent past in the stem cell research area followed by a section on strategic recommendations created after a thorough analysis of the industry. The strategic recommendations section focuses on some effective strategic decisions which can be taken up by companies to increase their market shares like public private partnerships, taking up measures to increase the number of cord blood samples and collaborations to a smooth progress of the stem cell research that will have a positive impact on the therapeutic segment.

Download Sample of This Strategic Report: https://www.kennethresearch.com/sample-request-10016694

The Market report answers the following questions:

What is the aim of the report?

What aspects regarding the regional analysis Market are included in this report?

Based on what factors are the key market players assessed in this report?

What is the key information extracted from the report?

For More Reports:X-Ray Equipment MarketCardiac Pacemaker MarketIn Vitro Diagnostics MarketBioinformatics Research MarketDental Implant Market

About Kenneth Research:

Kenneth Research provides scheduled syndicated reports that help industry professionals and organizations decipher market trends to take significant decisions and plan strategies. We cater to a wide range of industries including healthcare & pharmaceuticals, ICT & telecom, automotive & transportation, energy & power, chemicals, FMCG & food, aerospace & defense, among others. Our research team ensures to track and analyze the industry on a regular basis to offer strategic business consultancy services on a global level. We, at Kenneth Research are adept at capturing descriptive insights on crucial topics to help our clients make their informed decisions.

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Stem Cell Research in India Size and Outlook 2022, Manufacturing Demand, Industry Growth, Production Volume, Future Dynamics with Challenges and...

WATERTOWN, Mass.--(BUSINESS WIRE)--SQZ Biotechnologies (NYSE: SQZ), focused on unlocking the full potential of cell therapies for multiple therapeutic areas, today announced that it has been awarded a $2 million SBIR Phase II grant from the National Institute of General Medical Sciences, a division of the National Institutes of Health. Awarded through a competitive process, the two-year grant will support the development of cell engineering methods that are designed to reprogram a patients own immune cells directly into dopamine-producing neurons, a potential novel therapeutic approach for the treatment of Parkinsons disease.

Directly creating dopamine-producing neurons by reprogramming a patients own immune cells would be a major breakthrough and could support a new Parkinsons disease treatment paradigm, said Jonathan Gilbert, Ph.D., Vice President and Head of Exploratory Research at SQZ Biotechnologies. Unlike alternative allogeneic cell replacement approaches in development for Parkinsons disease, by using a patients own cells, treatment might not require chronic immunosuppression. Moreover, in altering cell fate with RNA-based cell engineering methods, no changes to the genome are likely to occur that could carry long-term risks.

Reprogramming a patients cells to replace lost or diseased cells has significant therapeutic potential. Beyond Parkinsons Disease, applications for cell replacement therapies include Multiple Sclerosis and Type 1 diabetes. However, traditional expensive, time-intensive, and inefficient cell reprogramming methods has hindered clinical progress and patient impact.

At the 2021 International Society for Stem Cell Research annual meeting, the company presented preclinical data showing that proprietary Cell Squeeze technology can be used to generate neurons from induced human pluripotent stem cells through the delivery of an mRNA encoding for a fate-specifying transcription factor.

With the support of the NIH grant, and building upon our experience in multiplex engineering of immune cells, SQZ researchers will attempt to generate dopaminergic neurons directly from somatic cells. The Cell Squeeze technology may allow for a unique complex combination of transcription factors, dosing, and timing.

About SQZ BiotechnologiesSQZ Biotechnologies Company is a clinical-stage biotechnology company focused on unlocking the full potential of cell therapies for patients around the world and has active programs in Oncology, Autoimmune and Infectious Diseases, as well as additional exploratory initiatives to support future pipeline growth. The companys proprietary Cell Squeeze technology offers the unique ability to deliver multiple biological materials into many cell types to engineer what we believe can be a broad range of potential therapeutics. With demonstrated production timelines under 24 hours and the opportunity to eliminate preconditioning and lengthy hospital stays, our approach could significantly broaden the therapeutic range and accessibility of cell therapies. The companys first therapeutic applications seek to generate target-specific immune responses, both in activation for the treatment of solid tumors and infectious diseases, and in immune tolerance for the treatment of autoimmune diseases. For more information, please visit http://www.sqzbiotech.com.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements relating to events our platform development, our product candidates, project funding, preclinical and clinical activities, progress and outcomes, development plans, manufacturing, clinical safety and efficacy results, therapeutic potential, market opportunities and disease prevalence. These forward-looking statements are based on management's current expectations. Actual results could differ from those projected in any forward-looking statements due to several risk factors. Such factors include, among others, risks and uncertainties related to our limited operating history; our significant losses incurred since inception and expectation to incur significant additional losses for the foreseeable future; the development of our initial product candidates, upon which our business is highly dependent; the impact of the COVID-19 pandemic on our operations and clinical activities; our need for additional funding and our cash runway; the lengthy, expensive, and uncertain process of clinical drug development, including uncertain outcomes of clinical trials and potential delays in regulatory approval; our ability to maintain our relationships with our third party vendors and strategic collaborators; and protection of our proprietary technology, intellectual property portfolio and the confidentiality of our trade secrets. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K, as updated by our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2021 and other filings with the U.S. Securities and Exchange Commission could cause actual results to differ materially from those indicated by the forward-looking statements. Any forward-looking statements represent management's estimates as of this date and we undertake no duty to update these forward-looking statements, whether as a result of new information, the occurrence of current events, or otherwise, unless required by law.

Certain information contained in this press release relates to or is based on studies, publications, surveys and other data obtained from third-party sources and our own internal estimates and research. While we believe these third-party sources to be reliable as of the date of this press release, we have not independently verified, and we make no representation as to the adequacy, fairness, accuracy or completeness of any information obtained from third-party sources.

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SQZ Biotechnologies Announces $2 Million Grant From the National Institutes of Health to Develop a Novel, Scalable Cell Replacement Therapy for...

SUNNYVALE, Calif., March 22, 2022 (GLOBE NEWSWIRE) -- BioCardia, Inc. [ BCDA], a developer of cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary disease, announces the designation of a new reimbursement code for the CardiAMP Cell Therapy procedure to transplant autologous bone marrow cells to treat heart failure from the U.S. Center for Medicare and Medicaid Services (CMS).

This new CMS code to be submitted by hospitals performing the CardiAMP cell therapy procedure is available April 1, 2022. The code provides clear reimbursement for the study procedure performed for both the treatment and control arms for the ongoing cell therapy pivotal trials in two cardiovascular indications: the CardiAMP Cell Therapy Heart Failure Trial (NCT02438306) and the CardiAMP Cell Therapy Chronic Myocardial Ischemia Trial (NCT03455725).

This most recent action by CMS further represents its commitment to improving the way ischemic heart failure is treated and is supportive of BioCardias therapeutic investigational product candidates furnished by a comprehensive approach to bone marrow cell harvest, processing, and delivery in a single procedure, commented Peter Altman, Ph.D., BioCardias CEO. Further, it provides additional clarity of CMS financial support for institutions conducting the CardiAMP Cell Therapy Heart Failure Trial and CardiAMP Chronic Myocardial Ischemia Trials. We are grateful for our ongoing collaboration with both CMS and the FDA as we continue to demonstrate the promise of our technology.

The new reimbursement code (designated C9782) is for a blinded procedure for New York Heart Association (NYHA) class ii or iii heart failure, or Canadian Cardiovascular Society (CCS) class iii or iv chronic refractory angina; transcatheter intramyocardial transplantation of autologous bone marrow cells or placebo control, autologous bone marrow harvesting and preparation for transplantation, left heart catheterization including ventriculography, all laboratory service and all imaging with or without guidance, performed in approved investigational device exemption (IDE) study.

Patients interested in learning about the CardiAMP Cell Therapy Trials can visit http://www.cardiamp.com or http://www.clinicaltrials.gov for more information.

About the CardiAMP Cell Therapy ProgramCardiAMP Cell Therapy uses a patients own (autologous) bone marrow cells delivered to the heart in a minimally invasive, catheter-based procedure to potentially stimulate the bodys natural healing response. The CardiAMP Cell Therapy Heart Failure Trial is the first multicenter clinical trial of an autologous cell therapy to prospectively screen for cell therapeutic potency in order to improve patient outcomes. CardiAMP Cell Therapy incorporates three proprietary elements not previously utilized in investigational cardiac cell therapy, which the company believes improves the probability of success of the treatment: a pre-procedural diagnostic for patient selection, a high target dosage of cells, and a proprietary delivery system that has been shown to be safer than other intramyocardial delivery systems and more successful for enhancing cell retention. CAUTION - Limited by United States law to investigational use.

About BioCardiaBioCardia, Inc., headquartered in Sunnyvale, California, is developing cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary disease. CardiAMP autologous and NK1R+ allogeneic cell therapies are the Companys biotherapeutic product candidates in clinical development. The Company's current products include the Helix transendocardial delivery system, the Morph steerable guide and sheath catheter portfolio and the AVANCE steerable introducer family. BioCardia also partners with other biotherapeutic companies to provide its Helix systems and development support to their programs studying therapies for the treatment of heart failure, chronic myocardial ischemia and acute myocardial infarction. The CardiAMP Cell Therapy Heart Failure Trial has been supported financially by the Maryland Stem Cell Research Fund and the Center for Medicare and Medicaid Services. For more information visit: http://www.BioCardia.com.

Forward Looking Statements:This press release contains forward-looking statements that are subject to many risks and uncertainties. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations. These forward-looking statements include, without limitation, statements relating to study enrollment expectations and the likelihood of safety and patient benefit, and ultimate success of our clinical cell therapy programs.

We may use terms such as believes, estimates, anticipates, expects, plans, intends, may, could, might, will, should, approximately or other words that convey the uncertainty of future events or outcomes to identify these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained herein, we caution you that forward-looking statements are not guarantees of future performance and that our actual results may differ materially from the forward-looking statements contained in this press release. As a result of these factors, we cannot assure you that the forward-looking statements in this press release will prove to be accurate. Additional factors that could materially affect actual results can be found in BioCardias Form 10-K filed with the Securities and Exchange Commission on March 30, 2021, under the caption titled Risk Factors. BioCardia expressly disclaims any intent or obligation to update these forward-looking statements, except as required by law.

INVESTOR CONTACT:David McClung, Chief Financial Officer[emailprotected](650) 226-0120

MEDIA CONTACT:Anne Laluc, MarketingEmail: [emailprotected] Phone: 650-226-0120

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The novel E-selectin antagonist uproleselan could augment the use of chemotherapy in patients with acute myeloid leukemia (AML) without increasing toxicity and potentially lessening some of the adverse effects associated with chemotherapy, according to David Sallman, MD.

Unlike some traditional combination therapies in oncology, where 2 or more agents target different aspects of a cancer, uproleselan can amplify the effects of chemotherapy by allowing the treatment to attack cancer cells that would normally go unaffected, Sallman added.

I would consider [uproleselan] more of an adjunct [treatment because] it is helping [the chemotherapy] target these cells that are not targetable, traditionally, by chemotherapy [alone] and allowing the chemotherapy to exert its normal function, Sallman said. Without uproleselan, this would likely not be possible.

In an interview with OncLive, Sallman, assistant member, Department of Malignant Hematology, Moffitt Cancer Center, provided perspective on the novel agent uproleselan, its efficacy in a phase 1/2 trial (NCT02306291), and its investigation in phase 3 trials in AML.

Sallman: To give a little bit of context, in general for AML, remissions can be achieved with both intensive and non-intensive therapies in sometimes up to 70% or greater of patients. However, relapse is a major issue, with a vast majority of patients relapsing unless they are bridged to allogeneic stem cell transplant. [There is always the concern] of having some leftover amount of disease after frontline therapy, which is associated with poor outcomes, even in those groups of patients that are ultimately bridged to transplant. Improving the depth of remission and targeting leukemic stem cells that may be left behind is of paramount importance to further improve treatment outcomes.

Uproleselan is a novel and specific E-selectin inhibitor. As a brief background, [E-selectin] is a vascular-adhesion molecule that is expressed on the vascular endothelium that helps leukocytes stick to the vessel wall. The thought is it helps keep these leukemic stem cells protected almost in a little house. A lot of times, these cells will not be dividing. Intensive chemotherapy does not target the cells that are not dividing, because it requires active cell cycle for that to occur.

There are other potential bone-marrow microenvironment markers that may be targeted, but this is the one that is furthest along. There is other important work that shows that E-selectin may help support and nurture these leukemic stem cells.

Ultimately, the goal is [determining whether] E-selectin [can] be used in conjunction with intensive chemotherapy to target these specific leukemic stem cells that are a major driver of relapse. One question with that is: Are the cells that are minimal residual disease [MRD] positive or left behind leukemic stem cells? Those comparisons and novel translational analyses, particularly on the single-cell level, [have yet to be done]. Essentially, the goal [is determining whether we can] target leukemic stem cells and help eradicate them in conjunction with more of our standard-of-care therapies.

The long-term goal is [to] improve the cure rates and overall survival [OS] for patients with AML. For a high percentage of this group, which has been predominantly targeting either relapsed/refractory leukemia or frontline patients that are over the age of 60 years, transplant is also part of that. [We are] also thinking of the long-term improvement of outcomes and prevention of relapse after transplant.

The goal is to improve the depth of remission and the duration of survival in patients that may not be able to go to transplant, but particularly in the setting of transplant, to improve the depth of remission to allow for the best long-term survival in patients that ultimately go to transplant.

[It is] a very specific inhibitor, so there should not be any increase in toxicity. It is specifically targeting E-selectin or CD62E and does not have any other significant off-target [effects]. It is very selective, and an interesting observation [from the phase 1/2 trial is that fewer instances of] mucositis, which is 1 of the most important toxicities of standard intensive chemotherapy, were seen [with the addition of uproleselan, and that may be a benefit. The hope is that we are not going to add any toxicity. If anything, we may have less toxicity [with the combination of uproleselan and chemotherapy].

By blocking E-selectin, uproleselan prevents this protective bone-marrow niche or microenvironment from protecting the cells from survival and eventually resistance to chemotherapy. By directly abrogating this E-selectin interaction of the bone-marrow microenvironment to the leukemic stem cell, it loses its protective house that should make it more susceptible to intensive chemotherapy.

I do not find it as much of a synergistic combination. It is not [like] a lot of clinical trials where when you use 2 [agents] together there is more, for example, apoptosis, cell-cycle arrest, [and] cytol effects against the leukemic cell.

The clinical trial of uproleselan first focused on patients with relapsed/refractory leukemia, and the chemotherapy backbone they used in this was MEC [mitoxantrone/etoposide/cytarabine]. Essentially, the investigators first did a phase 1 dose escalation, and the recommended phase 2 dose [RP2D] ultimately was found to be 10 mg/kg. They then did a phase 2 expansion of this combination with the RP2D of uproleselan in patients with relapsed/refractory AML. An important caveat is that most of these patients had failed just 1 line of therapy. Patients who fail more than 1 line of therapy are even less likely to have good outcomes and represent a distinct cohort when you are comparing them with other relapsed/refractory groups.

In a smaller cohort of 25 newly diagnosed patients over the age of 60 years, uproleselan [was combined] with standard 7+3 [chemotherapy] with idarubicin and cytarabine.

There are other salvage chemotherapy regimens such as FLAG [fludarabine/cytarabine/ granulocyte colony stimulating factor (G-CSF)], CLAG [cladribine/cytarabine/G-CSF], or CLAG-M [cladribine/cytarabine/G-CSF/mitoxantrone]. In retrospective studies, there has been overall no difference [between these regimens], but potentially, MEC has had slightly lower response rates. Additionally, in patients over the age of 60 years, cytarabine and daunorubicin [Vyxeos] is approved as frontline treatment and was superior to standard 7+3 intensive chemotherapy independent of age. Although the phase 1/2 trial focused on this similar group of patients, they were [required] to either have myelodysplastic-related changes, therapy-related history, or an antecedent myeloid neoplasm.

When we look at overall outcomes [with the addition of uproleselan to chemotherapy], the response rates are good. A composite complete remission [CR] was achieved in 41% of patients in the relapsed/refractory group, with a median OS of 8.8 months. In the frontline group, the composite CR rate was up to 72%, with a median OS of 12.6 months.

[It is worth noting the phase 1/2 trials were] single-arm studies. The response rates are good. The big challenge is figuring out whether [the response rates would be] any different [with] the absence of uproleselan. The problem in cross-trial comparisons is there are retrospective cohorts that have similar response rates and there are some that [have] worse [response rates]. The response rates appear to be at least as good and I would say on the favorable side of [treatment with chemotherapy alone.] Survival was not dramatically different in these cohorts of patients, particularly in the setting of additional salvage therapies that we have, such as targeted inhibitors and hypomethylating agent [HMA]/venetoclax [Venclexta]based combinations. The safety profile [of uproleselan] looked good. The critical next steps will be phase 3 clinical trials.

[Uproleselan] is a very selective inhibitor of E-selectin, and we do not expect a lot of increased toxicity [with the agent]. [It was] noteworthy that no dose-limiting toxicities were observed [in the phase 1 study]. Overall, likely from a combination of pharmacokinetic and pharmacodynamic data, the 10 mg/kg dose was utilized, [with] no significant increase, [and] if anything, a lower rate of mucositis, which can be a significant toxicity.

Clearly, [uproleselan] is safe. It does not add toxicity in combination with intensive chemotherapy. For example, [no] prolonged cytopenias were seen, which is often a major challenge when we are looking at novel combinations with intensive therapeutic options.

The good thing is uproleselan does not seem to add significant toxicities [to chemotherapy]. There can always be some infusional adverse [effects (AEs)], but for severe or life-threatening toxicities, which is really the focus when we are thinking about intensive chemotherapy, there is no increase [in toxicity with uproleselan]. Overall, the 60-day mortality rates were comparable. They are not dramatically lower, but are potentially on the low side in patients who are over the age of 60 years.

We have learned that non-randomized studies, particularly in this group of patients, are somewhat irrelevant in thinking [about] the long-term benefit of these medications, so conducting randomized trials is important. The data in Blood [on the phase 1/2 trial] clearly support the randomized phase 3 trials.

There are 2 main phase 3 trials ongoing right now: [NCT03701308 and NCT03616470]. Both are placebo-controlled studies, and they are mirrored after [the phase 1/2 study]. One is being done in the first-line setting in patients over the age of 60 [years and is evaluating] 7+3 chemotherapy with or without uproleselan and the other is being done in the salvage setting. In this case, the investigators are allowing 2 options: MEC or FLAG-IDA [fludarabine/cytarabine/idarubicin/G-CSF]. FLAG-IDA and CLAG-M are more commonly utilized regimens in the first-line salvage setting.

Those trials are both ongoing with a long-term primary end point of OS. Patients who are over the age of 60 years cannot have FLT3 mutations. They also cannot have an antecedent myeloid neoplasm or therapy-related disease. This is partially in the context that cytarabine and daunorubicin has an approval. There will be a little bit of overlap [between patient populations in the 2 trials]. For example, [treatment for] AML with myelodysplastic-related changes without those antecedent histories could be improved [with the addition of uproleselan]. Again, there are reasonable comparator arms and a clear primary end point to see whether the addition of uproleselan does improve outcomes either in the frontline setting or in the relapsed/refractory setting for first-line salvage therapy.

If these trials are positive, they would support [uproleselans] approval either in a frontline or relapsed/refractory space. What is nice about uproleselan is that its toxicity profile is good.

[It is important to note] that the landscape of frontline AML is changing quite quickly. For elderly [patients with AML who are] not fit for intensive chemotherapy, so classically over the age of 75 years or younger with specific comorbidities, HMAs in combination with venetoclax are a standard of care. Although, already at some academic centers, places are already favoring an HMA plus venetoclax over intensive chemotherapy in this group of patients who are 60 years of age or older. The question is: Are we going to move away from intensive chemotherapy? Does this [regimen] make [intensive chemotherapy] potentially less relevant? If the combination of an HMA and venetoclax becomes a standard for all patients over the age of 60 years, you could say response rates are comparable and MRD negativity rates are at least as good [compared with intensive chemotherapy]. We do not have the long-term data or data after allogeneic stem cell transplant [ASCT], which is a big [gap] in those datasets, but clearly some have already started to transition to [using an HMA plus venetoclax in that population].

There are prospective clinical trials comparing standard chemotherapy regimens in this age group with azacitidine and venetoclax. There is a clinical trial, not registrational, looking at uproleselan with an HMA plus venetoclax. We will eventually have some safety and outcome data there. [Would uproleselan have the same relevance] if we had an approved agent and the standard of care shifted? That is a critical question in the changing paradigm for [identifying] the best treatment.

Hopefully there will be some translational data. Are there subsets of patients that may have the greatest benefit with uproleselan? Could this therapy be used in conjunction with novel cellular therapy, which is a major focus, particularly in the relapsed/refractory space? There are 2 key trials with the goal of getting this agent approved. How it will eventually be utilized may change over time based on the results, but [we are] eagerly awaiting the data readout of these studies.

[It will be intriguing to see] additional translational data. Hopefully there will be robust single-cell analyses looking at leukemic stem cells. Can we really see, particularly in the placebo-controlled trial, the eradication of those? That will speak a lot to the mechanism of action of [uproleselan]. [Our improved technologies will help us understand] what happens preclinically vs in clinical trial patients. Therefore, I hope those key analyses will be done, and hopefully we will also [identify whether] there are subsets that may have the greatest benefit of therapy and may help us understand which patients to utilize that approach in, assuming the [trials] are positive.

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Adjunct Treatment With Uproleselan Could Enhance Effects of Chemotherapy in AML - OncLive