Category Archives: Stem Cell Therapy

This article was originally published here

Stem Cells Transl Med. 2022 May 28:szac032. doi: 10.1093/stcltm/szac032. Online ahead of print.

ABSTRACT

MSC (a.k.a. mesenchymal stem cell or medicinal signaling cell) cell therapies show promise in decreasing mortality in acute respiratory distress syndrome (ARDS) and suggest benefits in treatment of COVID-19-related ARDS. We performed a meta-analysis of published trials assessing the efficacy and adverse events (AE) rates of MSC cell therapy in individuals hospitalized for COVID-19. Systematic searches were performed in multiple databases through November 3, 2021. Reports in all languages, including randomized clinical trials (RCTs), non-randomized interventional trials, and uncontrolled trials, were included. Random effects model was used to pool outcomes from RCTs and non-randomized interventional trials. Outcome measures included all-cause mortality, serious adverse events (SAEs), AEs, pulmonary function, laboratory, and imaging findings. A total of 736 patients were identified from 34 studies, which included 5 RCTs (n = 235), 7 non-randomized interventional trials (n = 370), and 22 uncontrolled comparative trials (n = 131). Patients aged on average 59.4 years and 32.2% were women. When compared with the control group, MSC cell therapy was associated with a reduction in all-cause mortality (RR = 0.54, 95% CI: 0.35-0.85, I 2 = 0.0%), reduction in SAEs (IRR = 0.36, 95% CI: 0.14-0.90, I 2 = 0.0%) and no significant difference in AE rate. A sub-group with pulmonary function studies suggested improvement in patients receiving MSC. These findings support the potential for MSC cell therapy to decrease all-cause mortality, reduce SAEs, and improve pulmonary function compared with conventional care. Large-scale double-blinded, well-powered RCTs should be conducted to further explore these results.

PMID:35640138 | DOI:10.1093/stcltm/szac032

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Efficacy and Safety of MSC Cell Therapies for Hospitalized Patients with COVID-19: A Systematic Review and Meta-Analysis - DocWire News

Richard Stone, MD: How do we treat the patient like this?

Eunice Wang, MD: In this instance, Id look at the overall presentation of the patient. AML [acute myeloid leukemia] therapy in the current era requires that we individualize the treatment regimen for the patient and disease. This woman has undergone intensive or definitive therapy for her breast cancer. Shes still functionally active, has an excellent performance status, and doesnt have any evidence of organ dysfunction, eg, liver, kidney, or heart. Her activity level is normal. Shes continuing to work full-time, chasing high school students around.

Id think about offering her an intensive chemotherapy approach, potentially followed by allogeneic stem cell transplant for curative intent. Because we know that in most patients with a secondary therapyrelated AML, the best outcomes are achieved by the performance of an allogeneic stem cell transplant as postconsolidation therapy. What are your thoughts on this?

Richard Stone, MD: I agree with everything you said. When I think about intensive chemotherapy, I think about whether the patient can tolerate it and whether it will benefit the patient. Because there are certain patients who can tolerate intensive chemotherapy, but Im not sure [they] would benefit from it. Its still an open question. In other words, patients with a TP53 mutation and complex karyotype may not benefit from intensive chemotherapy. They may not [have] better [results] with azacitidine and venetoclax, but at least theyre not going to be as sick.

Without any proof, I have a predilection to treat the patients [with high-risk disease] with less intensive chemotherapy, because it isnt likely to be worse and will likely be better tolerated. Thats unproven at the moment. For this patient, Id use an intensive approach because of her age, favorable mutational status, and good performance status. I have a similar view that she should get an intensive regimen. What do you think are the choices for an intensive regimen?

Eunice Wang, MD: This patient is over the age of 60, which makes it more straightforward. But based on her age and the diagnosis of a secondary therapyrelated AML, my preference would be to treat her with CPX-351, or liposomal cytarabine-daunorubicin. That offers her the best opportunity to be free of disease in 3 to 5 years.

There were the results of a phase 3 randomized clinical trial where patients aged 60 to 75 [years] with secondary AML were [randomly assigned] to receive up-front induction of chemotherapy with either CPX-351that liposomal formulation is dosed on days 1, 3, and 5[or] the standard continuous infusion [of] cytarabine and daunorubicin, our 7+3 gold standard. In that trial, patients could get 2 cycles of the CPX-351 induction or 7+3. They could get consolidation with a CPX-351 vs a 5+2. Eligible patients could then go on to subsequent transplant.

The 5-year results were published in 2021 and substantiate the early findings that led to FDA approval, where about 18% in the CPX-351 group and 8% in the 7+3 group were alive and disease-free at 5 years. Of the patients who had gotten CPX-351 followed by transplant, most impressively, 53% were alive. The overall survival for those patients who had gone to subsequent transplant from CPX-351 hadnt been reached yet. I was impressed with that because in the past, patients who got therapy-related AML rarely went into remission or long-term response. Very few of themless than 10% with standard therapy[survived for] 3 to 5 years. Based on those data, Id be offering CPX-351 to this patient.

The only question would beand I throw this back to youwhat if she were 59 [years old]? Would you treat her similarly? Because the trial was for patients aged 60 to 75 years. If she were 55 or 45 [years old], would you do the same thing? Or would you restrict your interpretation of that trial to the [patients aged] 60 to 75?

Richard Stone, MD: Thats a great question. The FDA believes you could extend the [use] of CPX-351 to younger folks because they approved it in an age-agnostic fashion, which was surprising because, as you pointed out, the data were only [from results of] patients aged 60 to 75 [years]. Its possible that a 7+3 regimen would have been better than CPX-351 for younger patients. They did a trial in all ages, [but] it didnt show better results. They did a trial with older adults, and it was only the ones who had secondary AML who seemed to benefit from CPX-351 compared with 7+3. Its a little unclear, but I believe its because CPX-351 seems to release ARA-C [cytosine arabinoside] and daunorubicin, a so-called favorable molar ratio, to kill the cells. Thats more theoretical, but its true. It also [remains] in the bone marrow a little longer, which accounts for its mild suppression. Maybe thats another reason why its a little better.

The answer to your question is that Id use it in a younger patient who had secondary AML. Curiously, in another post hoc analysis done by R. Coleman Lindsley, [MD, PhD], of the CPX-351 vs 7+3 trial that you mentioned, [patients] with TP53 mutations didnt benefit from CPX-351 compared with 7+3. Thats another situation where you throw up your hands. [These] were [patients aged] 60 to 75, but Id use it in the right patient under 60 [years of age]. In fact, theres an ongoing trial in Europe for patients regardless of their history of MDS [myelodysplastic syndrome] or prior treatment that will compare 7+3 with CPX-351. Maybe it will turn out to be a better induction regimen than 7+3 alone. We need to wait and see.

We talked about CPX-351 compared with azacitidine-venetoclax in the retrospective studies that were published at the American Society of Hematology meeting. CPX-351 is similar to 7+3 chemotherapy, but different in that its given episodically on days 1, 3, and 5. As you mentioned, its possible to give this [treatment] outpatient if the patient isnt ill, and we do that too. It saves hospitalization. Its also financially toxic to the inpatient service to pay for CPX-351, which is very expensive compared with 7+3. But you may save money by not admitting the patient for as many days. We bring them in on day 10 at the start of their expected nadir, because almost all of them will [have] some fever and neutropenia, and its easier to have them in the hospital when that happens than outpatient [service].

We find CPX-351 to be well tolerated in general, with no hair loss, minimal GI [gastrointestinal] toxicity, and as you mentioned, prolonged myelosuppression. Those are the main considerations with CPX-351. Its cardiotoxic. Its hard to know how it compares with standard daunorubicin, or even doxorubicin, for those who have secondary AML. [For] your patient, its important to consider the prior doxorubicin if they were treated for breast cancer or another cancer. We get echocardiograms frequently for our CPX-351 patients. Any other thoughts about that, Eunice?

Eunice Wang, MD: As you mentioned, there are a lot of things to recommend in terms of the toxicity profile. Our patients are excited about the fact that they dont lose their hair and [wont] be inpatient for 40, 50, or 60 days. In general, there are still the complications from infection, but the ability to do part of the regimen outpatient and then do all the regimen outpatient in the consolidation setting while waiting for possible transplant improves quality of life. [It] has been demonstrated in other studies that, as compared with 7+3 and standard consolidation, patients have almost 50% improved quality-of-life scores on various questionnaires when asked about the comparison between the 2. Thats an important thing to keep in mind as were tailoring therapy for the patient and the disease.

Transcript edited for clarity.

Continued here:
CPX-351 in Treatment of AML and Clinical Considerations - OncLive

As more treatments continue to enter the market for multiple myeloma (MM), the cost of associated treatments is expected to continue climbing, with the researchers of the study emphasizing the need for more effective and options to help mitigate costs.

A new French study is highlighting the high cost burden associated with multiple myeloma (MM), as well as variability in treatments used in later lines of therapy. The findings were published in The European Journal of Health Economics.

As more treatments continue to enter the market for MM, the cost of associated treatments is expected to continue climbing, with the researchers of the study emphasizing the need for more effective and options to help mitigate costs.

Understanding patient [health care resource utilization] patterns, disease burden, and health-related expenditure is important when evaluating the potential value of new treatments and facilitates targeted improvements in MM management, explained the researchers. Analyses of health insurance databases can guide public health care decisions, monitor various types of medical expenditures, inform epidemiological studies, evaluate medical practices or health system experimentations, and can be used for international comparisons.

The researchers included real-world data from 6400 patients diagnosed with MM between 2013 and 2018, finding an average annual cost of 58,300 (approximately $62,500). Notably, costs were highest in the first year after diagnosis, with a mean total annual cost per patient in the first exceeding 72,400 (approximately $77,600) and the monthly exceeding 7100 (approximately $7600).

The cost of treatment and hospitalizations contributed the most to costs, with an overall rate of 6.3 hospitalizations per patient per year. Nearly all (96.6%) patients underwent some type of hospitalization for any cause, and had at least 1 overnight hospital stay (93.1%). Notably, hospitalization accounted for a greater proportion (48.6%) of total costs in the first year after diagnosis than the average of the rest of the years of follow-up (38.1%).

With each line of therapy (LOT), the monthly costs associated with treatment increased. Monthly costs increased from 2447 (approximately $2600) during the first LOT to 7026 (approximately $7500) during fifth LOT and subsequently.

Increase in hospitalisation costs associated with subsequent LOT was likely related to the increasing age and decreasing health of patients requiring ongoing treatment for MM, described the researchers. This is supported by a retrospective study of hospitalised French patients, which identified an association between age and duration of hospital stay. The cost of hospitalisations for an event of interest constituted a greater proportion of hospitalisation costs among patients at later LOT, indicating that declining patient condition conferred a greater burden to the health care system beyond the cost of drugs.

Health care resource utilization was lower for patients who received stem cell transplantation, although their hospital stays were associated with higher costs, largely due to the more expensive procedure.

Nearly all (97.1%) patients received an identifiable treatment throughout the study period, with the most frequent regimens being those that included bortezomib. Bortezomib-based regimens accounted for 62.2% of the 8865 regimens, and 96.7% of patients received the combination as their first LOT. During second and third LOT, lenalidomide-based regimens were most common. By the fourth line of therapy and later, the researchers identified 37 different regimens.

Reference

Bessou A, Colin X, Nascimento J, et al. Assessing the treatment pattern, health care resource utilisation, and economic burden of multiple myeloma in France usingthe Systme National des Donnes de Sant (SNDS) database:a retrospective cohort study. Eur J Health Econ. Published online May 25, 2022. doi:10.1007/s10198-022-01463-9

Excerpt from:
High Economic Burden of MM, Particularly Within 1 Year of Diagnosis, Says French Study - AJMC.com Managed Markets Network

You should read the following discussion and analysis of our financial conditionand results of operations together with the condensed consolidated financialstatements and related notes included in Part I, Item 1 of this Quarterly Reporton Form 10-Q (this "Quarterly Report") and with the audited financial statementsand the related notes included in our Annual Report on Form 10-K for the fiscalyear ended December 31, 2021 filed with the Securities and Exchange Commissionon March 18, 2022. Certain of the information contained in this discussion andanalysis or set forth elsewhere in this Quarterly Report, including informationwith respect to plans and strategy for our business, includesforward-looking statements that involve risks and uncertainties. As a result ofmany factors, including those factors set forth in the section entitled "RiskFactors", in Part II, Item 1A of this Quarterly Report, our actual results coulddiffer materially from the results described in or implied by theforward-looking statements contained in the following discussion and analysis.You should carefully read the section entitled "Risk Factors" to gain anunderstanding of the important factors that could cause actual results to differmaterially from our forward-looking statements. Please also see the section ofthis Quarterly Report entitled "Cautionary Note RegardingForward-Looking Statements." The events and circumstances reflected in ourforward-looking statements may not be achieved or may not occur, and actualresults could differ materially from those described in or implied by theforward-looking statements contained in the following discussion and analysis.As a result of these risks, you should not place undue reliance on theseforward-looking statements. We assume no obligation to revise or update anyforward-looking statements for any reason, except as required by law.OverviewWe are a clinical-stage biotechnology company dedicated to enabling curesthrough hematopoietic stem cell therapy. We are focused on the development andcommercialization of safer and more effective conditioning agents and mRNA-basedstem cell engineering to allow for expanded use of stem cell transplantation andex vivo gene therapy, a technique in which genetic manipulation of cells isperformed outside of the body prior to transplantation. We are also developingnovel therapeutics directed at diseased hematopoietic stem cells.Our drug development pipeline includes multiple product candidates designed toimprove hematopoietic stem cell therapy. Our lead product candidate, JSP191, isin clinical development as a novel conditioning antibody that clearshematopoietic stem cells from bone marrow in patients prior to undergoingallogeneic stem cell therapy or stem cell gene therapy. We plan to initiate aregistrational clinical study in acute myeloid leukemia ("AML") patientsundergoing stem cell transplantation by the end of the first quarter of 2023.Based on the single agent depletion observed in our Phase 1 study ofmyelodysplastic syndrome ("MDS") patients undergoing stem cell transplant, weare also initiating a pilot study of JSP191 as a therapeutic in lower-risk MDS,which we expect to commence in the second half of this year. Beyond JSP191, weare developing stem cell grafts transiently reprogrammed using mRNA that have acompetitive advantage over endogenous hematopoietic stem cells ("HSCs"),enabling higher levels of engraftment designed to remove the need for highlytoxic conditioning of the patient and lower the risk of other seriouscomplications that limit current stem cell transplants. We plan to continue toexpand our pipeline to include other novel stem cell therapies based on immunemodulation, graft engineering and cell or gene therapies. Our goal is to expandthe use of curative stem cell transplant and gene therapies for all patients,including children and the elderly.Stem cell transplantation is among the most widely practiced forms of cellulartherapy and has the potential to cure a wide variety of diseases, includingcancers, genetic disorders, and autoimmune diseases. Yet currently, patientsmust receive highly toxic and potentially life-threatening conditioning agentsto prepare their bone marrow for transplantation with either donor stem cells ortheir own gene-edited stem cells. Younger, fitter patients capable of survivingthese toxic side effects are typically given myeloablative, or high-intensity,conditioning whereas older or less fit patients are typically given reducedintensity, but still toxic, conditioning which leads to less effectivetransplants. These toxicities include a range of acute and chronic effects tothe gastrointestinal tract, kidneys, liver, lung, endocrine, and neurologictissues. Depending upon the conditioning regimen, fitness of the patient, andcompatibility between the donor and recipient, the risk of transplant-relatedmortality ranges from 10% to more than 50% in older patients. Less toxic ways tocondition patients have been developed to enable transplant for older patientsor those with major comorbidities, but these regimens risk less potent diseaseelimination and higher rates of disease relapse. Even though stem cell therapycan be one of the most powerful forms of disease cure, these limitations ofnon-targeted conditioning regimens have seen little innovation over the pastdecade. 20Our lead product candidate, JSP191, is a monoclonal antibody designed to blockthe specific signal on stem cells required for survival. It is currently indevelopment as a highly targeted conditioning agent prior to stem cell therapyas well as a therapeutics in lower-risk MDS patients, which we expect tocommence in the second half of 2022. We are also sponsoring two clinical studiesof JSP191 as a conditioning agent prior to stem cell transplant. The firstclinical study is an open label Phase 1/2 trial in two cohorts of severecombined immunodeficiency ("SCID") patients: patients with a history of a priorallogeneic transplant for SCID but with poor graft outcomes and newly diagnosedSCID patients. The primary endpoint in this study is to evaluate the safety andtolerability of JSP191. The secondary goal of this study is to evaluate theefficacy of JSP191 as a conditioning agent in conjunction with a stem celltransplant. Based on preliminary results from our ongoing Phase 1/2 clinicaltrial, we believe JSP191 has demonstrated the ability as a single agent toenable engraftment of donor HSCs as determined by donor chimerism, or thepercentage of bone marrow cells in the patient that are of donor origin aftertransplant. Engraftment was observed in seven out of ten T-B-NK+ SCID patientswith prior allogeneic transplant, as evidenced by CD15+ donor chimerism of morethan 5% averaged from 12-24 weeks post-transplant. Increased nave donor T cellproduction was observed in the majority of T-B-NK+ subjects, as well as clinicalimprovement. No JSP191 treatment-related serious adverse events ("SAEs") havebeen reported to date and pharmacokinetics have been consistent with earlierstudies in healthy volunteers. We expect to complete enrollment in this Phase1/2 clinical trial by mid-2023.

The FDA has granted rare pediatric disease designation to JSP191 as aconditioning treatment for patients with SCID. In addition, the FDA grantedorphan drug designation to JSP191 for conditioning treatment prior tohematopoietic stem cell transplantation.

We expect our expenses will increase substantially in connection with ourongoing and planned activities, as we:

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Business Impact of the COVID-19 Pandemic

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External research and development costs include:

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parties that conduct preclinical and clinical activities on our behalf and

manufacture our product candidates;

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that have no alternative future uses;

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Internal research and development costs include:

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stock-based compensation expense for our research and development personnel;

? other expenses and allocated overheads incurred in connection with our research

Our future research and development costs may vary significantly based onfactors, such as:

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preclinical development activities;

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Other Income (Expense), Net

Three Months Ended March 31, 2022 and 2021

The following table summarizes our results of operations for the three monthsended March 31, 2022 and 2021 (in thousands):

Research and Development Expenses

The following table summarizes our research and development expenses for thethree months ended March 31, 2022 and 2021 (in thousands):

Our external costs by program for the three months ended March 31, 2022 and 2021were as follows (in thousands):

General and Administrative Expenses

Liquidity and Capital Resources

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Contractual Obligations and Commitments

We have contractual obligations and commitments as described in Note 9,Commitments and Contingencies, within our condensed consolidated financialstatements included in Part I, Item 1 of this Quarterly Report.

Our future financing requirements will depend on many factors, including:

? the timing, scope, progress, results and costs of research and development,

preclinical and non-clinical studies and clinical trials for our current and

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approval of our current and future product candidates;

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support a marketing application;

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or other intellectual property rights;

? expenses incurred to attract, hire and retain skilled personnel;

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? the impact of the COVID-19 pandemic, which may exacerbate the magnitude of the

10,752

Cash Flows Used in Operating Activities

Net cash used in operating activities was $14.2 million and $6.2 million for thethree months ended March 2022 and 2021, respectively.

Cash Flows Used in Investing Activities

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JASPER THERAPEUTICS, INC. Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - Marketscreener.com

Albany NY, United States: According to the report, the global cell culture market was valued at US$ 10.5 Bn in 2020 and is projected to expand at a CAGR of 9% from 2021 to 2031. The global cell culture market is driven by development of new products, technological advancements, and increase in use of single-use bioprocessing systems during the forecast period. The cell culture market in Asia Pacific is anticipated to expand at the fastest CAGR during the forecast period due to high unmet clinical needs, improvements in the healthcare infrastructure, and increase in focus on research & development activities.

Rise in Demand and Approvals for Biosimilar Products & Other Biologic Therapeutics to Drive Market

Cell culture is one of the most important steps for the production of biosimilar antibodies, as cell culture helps increase efficiency & productivity and reduce the cost of manufacture. The increase in demand & approvals for biosimilars products and other biologic therapeutics for the treatment of chronic diseases in developed as well as developing countries has led to the demand for efficient and cost-effective products. This is expected to drive the global cell culture market during the forecast period.

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Moreover, rise in demand for cost-effective and highly efficient cell culture products such as bioreactors, media, reagents, and sera for the production of high-yield cell lines has led to a surge in the number of new product launches. This is likely to provide lucrative opportunities in the global cell culture market during the forecast period. Major manufactures strive to expand their product portfolio by launching new and advanced systems for large-scale production, which is cost-effective and has low risk of contamination.

For instance, in 2018, Merck launched capsule filters that are designed to decrease the risk of contamination in a bioreactor. These filters are used for the separation of mycoplasma and bacteria from cell culture media. However, ethical issues associated with the use of fetal bovine serum, stringent regulations, and high cost of infrastructure for cell culture are projected to hamper the growth of global cell culture market during the forecast period.

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Technological Advancements in Cell Culture Production Media & Instruments to Drive Demand for Protein-based Therapeutics

In terms of product, the global cell culture market has been classified into instruments, media, sera, and reagents. The instruments segment has been categorized into cell culture vessels (bioreactors), carbon dioxide incubators, biosafety cabinets, cryogenic tanks, and others. Technological advancements for improving the efficiency and reducing the risk of cross contamination are anticipated to propel the instruments segment during the forecast period.

The media segment has been split into chemically defined, classical media, protein free media, Lysogeny media, serum free media, and specialty media. The efficiency of different media used for cell culture production is expected to drive the media segment over the next few years.

The sera segment has been bifurcated into fetal bovine serum and others. Sera are used as cell culture supplements consisting of growth factors, nutrients, and other important trace elements. The reagents segment has been segregated into albumin, amino acid, attachment factors, growth factors & cytokines, protease inhibitor, thrombin, and others. Applications such as stem cell research have vast potential in future. Stem cell culture assists in standardization of drug production and enables production of a number of cell lines & related products.

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Traditional pharmaceutical therapeutics help in treating only disease symptoms, whereas stem cell therapies assist in treating the cause of the disease. Hence, further research in the field of stem cell culture for development of drugs presents significant opportunities in the market in the near future.

Increase in Usage of Cell Culture Products in Drug Development & Manufacturing to Propel Pharmaceutical Companies Segment

Based on end-user, the global cell culture market has been divided into biotechnology companies, pharmaceutical companies, academic institutes, and research institutes. The pharmaceutical companies segment dominated the global market in 2020, and the trend is anticipated to continue during the forecast period due to increase in usage of cell culture products in drug development and manufacturing. The biotechnology companies segment is expected to account for a significant market share by 2031. The growth of the segment can be attributed to increase in the number of biotechnology companies and rise in strategic collaborations among market players to expand global presence.

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Asia Pacific to Dominate Global Market

In terms of region, the global cell culture market has been segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is anticipated to account for a major share of the global market during the forecast period, owing to the presence of key players, increase in research & development activities, and new drug approvals.

Shift in trend toward continuous processing is expected to drive the cell culture market in North America. The cell culture market in Asia Pacific is at a pivotal point. Increase in focus of key players on expansion in the region, large untapped population, and rise in awareness about healthcare augment the cell culture market in Asia Pacific. For instance, Thermo Fisher expanded its Fisher BioServices and cryogenic service capabilities in Japan.

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Thermo Fisher Scientific and Merck KGaA to Lead Global Market

Key players covered in this report are Sartorius AG, Thermo Fisher Scientific, Inc., Eppendorf AG, GE Healthcare, Corning Incorporated, Becton, Dickinson and Company, Merck KGaA, Lonza, VWR International, LLC, and PromoCell GmbH. Companies operating in the global cell culture market focus on strategic collaborations for developing new products in the emerging markets such as Asia Pacific and Latin America.

For instance, in May 2017, Merck announced the launch of EX-CELL Advanced HD perfusion medium, which helps increase the production yield and streamline the regulatory compliances.

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CAR T-cell Therapy Market: The global CAR T-cell therapy market is driven by increase in incidence of cancer and strong product pipeline. North America dominated the global CAR T-cell therapy market in 2020 and the trend is anticipated to continue during the forecast period.

Psoriatic Arthritis Therapeutics Market: The global psoriatic arthritis therapeutics market is expanding at a rapid pace due to high prevalence and increase in incidence rate of psoriatic arthritis and new product development and commercialization across the globe.

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Cell Culture Market to Gain US$ 27.6 Bn by 2031, Burgeoning Demand for Biosimilars and Biologic Therapeutics Fueling Market Growth - BioSpace

Bijal Shah, MD, MS, discusses the results of a phase 2 pilot study investigating CPX-351 in adults with high-risk relapsed/refractory acute lymphoblastic leukemia, as well as next steps for research in this patient population.

CPX-351 (Vyxeos), a liposomal formulation of cytarabine and daunorubicin provided encouraging activity and overall response rates in adults with high-risk relapsed/refractory acute lymphoblastic leukemia (ALL) or mixed phenotype leukemia, according to data from a phase 2 trial (NCT03575325).1

The small open-label, single-arm pilot study featured 6 patients with B-cell ALL, including 1 with B-myeloid disease. Additionally, 5 patients had T-cell ALL, including 4 with an early T-cell phenotype. Moreover, a TP53 mutation was noted in 5 patients. Of the 10 evaluable patients, 3 had a complete response (CR) or CR with incomplete count recovery (CRi), and further research with this agent is warranted, according to Bijal Shah, MD, MS.

We were happy with how CPX-351 performed in this pilot cohort, Shah said. This sets up a benchmark for expectations with single-agent CPX-351. It also tells us about who were going to be enrolling into relapsed and refractory trials as we go forward with the approvals of these novel agents in B-cell ALL.

In an interview with OncLive, Shah, associate member in the Department of Malignant Hematology at Moffitt Cancer Center, discussed the results of the phase 2 pilot study, as well as next steps in the patient population.

Shah: When we talk about how T cells kill, theres the assumption that its largely a perforin/granzyme mediated cell-killing event. What was shared was that there may be other mechanisms involved here, including the induction of apoptosis in lymphoblasts, and what the investigators saw on a [CRISPR/Cas9] screen was that when you lose some of the proteins that help mediate apoptosis, you lose your CAR T-cell killing efficiency. There are 2 pieces to that.

One is that it seems like those cells can grow more rapidly. More proliferative leukemias are harder to kill. The other part of the equation is the T cells are less effective in eliminating those cells, despite perforin and granzyme production, and, classically, what we think of as T-cell mediated killing. Some of that may relate to the fact that the T cells are becoming exhausted as they proceed down this path and keep trying to kill this blast population that doesnt want to accept defeat.

When we do genetic testing of leukemia, were not often looking at mutations or other changes along the apoptosis pathway. But it does suggest that we need to think more carefully, particularly in the more proliferative leukemias, to see whether there is an enrichment for those mutations that come with that enhanced resistance to CAR [T-cell therapy]. This fits into a whole category that were seeing as they relate to intrinsic CAR T-cell resistance.

We have talked about CD19 antigen downregulation, as well as CD58 and how that may affect CAR T-cell therapy and cancer interactions. Were starting to now get a flavor for all these things, touching specifically on intrinsic features in lymphoma. I hope were also going to start to better understand tumor microenvironment features.

Dr Ruella talked about his studies looking at the microbiome and how that may affect T-cell biology. We know that antigen-presenting cells, or along that same spectrum, myeloid dendritic suppressor cell populations, can affect both the expansion and the contraction of T cells over time. There are a lot of moving parts here [in figuring out how we can] improve the activity of CAR T-cell therapies and the durability of cell killing.

[Regarding] toxicity, it seems like that may be more reaction to the T-cell growth rather than the T cells directly driving this phenotype. Targeting some of these pieces of the tumor microenvironment also improves toxicity, which, interestingly, may also improve T-cell activity and the durability of that activity.

Its a rapidly evolving field, and its great to see. Hopefully well get to a point where we can talk about CAR T-cell therapy as a replacement for chemotherapy. Thats the ultimate goal.

Although Im excited about [CAR T-cell therapy as a replacement for chemotherapy], it is true that there are other things that are happening, including a trial that we did examining [CPX-351].

This is the liposomal formulation of daunorubicin and cytarabine. We want to get rid of chemotherapy, but as it turns out, not everyone is an immunotherapy candidate. Weve seen this in clinical practice. There are [patients] who are still on immunosuppressive therapy following an allogeneic stem cell transplant, and there are [patients] who dont have B-cell ALL. T-cell ALL remains an area that were struggling to target from an immunotherapeutic standpoint. We wanted to try to understand what we could accomplish [with this agent].

This was a pilot study with 11 patients. The observations were interesting, and 1 of the key observations was who we enrolled. We have novel trials for ALL, including with CAR T-cell immunotherapy. Six [patients] who came onto [the study] had B-cell ALL, including 1 B-myeloid overlap.

Of the remaining 5 patients with T-cell ALL, 4 had an early T-cell phenotype. That tells us where some of our gaps are. When we look to the biology, 5 of the 10 evaluable patients had a TP53 mutation. [Additionally], 2 had [Philadelphia chromosomelike] changes, speaking to the high-risk nature of the patients who were enrolled to the study. They had a median of 3 prior lines of therapy, with a range of 1 to 7. Seven had primary refractory disease. When we talk about prior therapies for the B-cell ALL cohort, that included blinatumomab [Blincyto; n = 5], inotuzumab ozogamicin [Besponsa; n = 2], and CAR T-cell therapy [n = 1]. [Moreover], 3 patients had prior [allogeneic stem cell transplant]. This was a very heavily pretreated population, with [a] high [prevalence of] TP53 mutations and high rates of early T-cell precursor and B-myeloid phenotypes. [These were] the groups that you would expect since we dont have great therapies for this group of patients.

We did see pancytopenia during induction as you would expect with cytarabine and daunorubicin. We did see febrile neutropenia in 9 [patients]. In terms of severe infections, it wasnt what I would have predicted, particularly having been involved in the studies of CPX-351 in acute myeloid leukemia. We had 1 patient who [died] from pneumonia. This was a patient who had B-cell ALL, and she failed to collect for CAR T-cell therapy [since] she had low lymphocyte counts. We had hoped to intubate her and support her through the process, but she and her family decided to move to comfort measures in lieu of intubation.

A second patient developed grade 3 sepsis, which resolved without any significant issues. All other infections in the remaining patients were grade 1 to 2. The only atypical thing that we saw [in terms of] severe, unexpected adverse [effects] was 1 gastrointestinal bleed. This was in a patient who had prior allogeneic transplant, and there was a concern that this may have reflected some component of graft-versus-host disease, [GVHD], although we couldnt confirm that. We had 3 grade 1 spontaneous subdural bleeds. [Those patients] all had low platelets, but we do see low platelets commonly in ALL without bleeding. This was a novel observation.

We had 1 patient develop veno-occlusive disease, but this is a patient who had prior inotuzumab [ozogamicin] and allogeneic transplant, so it was difficult to connect that with the CPX-351 therapy.

The infections that we saw were largely mild, meaning grade 1 to 2. Although we did see some unexpected cases of bleeding, [they] were incidentally appreciated in most cases and resolved.

The safety of this therapy was impressive in our patients with advanced ALL. The median time to neutrophil recovery was around 33 days. There were 2 patients who had refractory disease who didnt recover their platelets, but among the remaining patients, [recovery] was around 30 days. We did allow consolidation, so just as in the ALL trial, patients received an induction dose and then subsequent consolidation doses where the CPX-351 was given at a lower dose.

We saw 1 episode of foot cellulitis. This was a patient who had recurrent foot cellulitis prior to enrolling on the study. [We also observed] 1 episode of myocarditis. That ultimately was not attributed to the study drug.

In terms of responses, 3 patients achieved a CR or CRi including 2 in the early T-cell precursor group and 1 who had B-cell ALL.

We did see responses in the TP53 cohort, so that was very encouraging. [Of those 4 patients], 1 had a CR/CRi, and 1 showed a reduction of blasts but not enough to meet the criteria for CR. One went on to receive consolidation, and one was bridged to a donor leukocyte infusion.

It seemed like we were succeeding in terms of supporting these patients. The median time to next therapy was about 2.5 months, so 76 days. The median overall survival for the patients was 218 days, so it seemed to come with some clinical benefit.

With regard to what happened when patients progressed, after CPX-351, we only saw responses to any subsequent therapy in 2 patients. The other 8 patients were refractory to every subsequent therapy that was administered, ranging from chemotherapy to targeted therapy. This speaks to the nature of the patients that were enrolled.

Next steps are to figure out what we can do with CPX-351. That may be combining it with novel agents like venetoclax [Venclexta] or even asparaginase [crisantaspase, erwinase]. It may also mean continuing to treat more intensively since we really didnt see any significant toxicity in the consolidation phases.

Once patients moved to the lower intensity CPX-351, they were more likely to progress, if they didnt go on to transplant. We would probably want to think about continuing the induction dosing for at least 1 additional cycle to help deepen responses for some of these patients.

Originally posted here:
Pilot Study Shows Promising Results With CPX-351 in Relapsed/Refractory ALL - OncLive

"Grand View Research, Inc. Market Research And Consulting."

According to a new report published by Grand View Research, the market growth can be primarily attributed to the increasing prevalence of chronic conditions, such as cancer, infections, autoimmune diseases, diabetes mellitus, cardiovascular diseases, and nephrological diseases. This has led to an increase in research, facilitating the high adoption of primary cell cultures.

Primary Cell Culture Industry Overview

The global primary cell culture market size was valued at USD 3.4 billion in 2020 and is expected to reach USD 8.0 billion by 2028, projecting to expand at a CAGR of 11.6% during forecast period.

The market growth can be primarily attributed to the increasing prevalence of chronic conditions, such as cancer, infections, autoimmune diseases, diabetes mellitus, cardiovascular diseases, and nephrological diseases. This has led to an increase in research, facilitating the high adoption of primary cell cultures.

Gather more insights about the market drivers, restrains and growth of the Global Primary Cell Culture Market

According to the American Cancer Society, there are an estimated 16.9 million cancer patients/survivors in 2020. According to the WHO (2020), 71.0 million people worldwide are suffering from chronic hepatitis C infection. Hence, the rising number of chronic disorders being diagnosed is driving the demand for research on advanced therapies, which is expected to boost the market growth in the coming years.

In addition, the rising usage of primary cell cultures for in-vitro testing and drug screening can be further attributed to the growth of the market for primary cell culture. These are derived from tissues, which facilitates researchers to study cellular structure in the in vivo state, showcasing their normal functioning. As a result, they are used as model systems to study cell biochemistry and physiology, aging processes, signaling studies, and metabolic processes, as well as the effect of toxic compounds and drugs.

COVID-19 pandemic is acting as a positive catalyst for the growth of the market for primary cell culture. Researchers are increasingly using primary cell culture for understanding the infection. Standardized and characterized epithelium cell culture models are facilitating the understanding of the physical barrier destroyed by the coronavirus. These models can help mimic the functions and properties of the respiratory tract, leading to a breakthrough for a research outcome turning into a medical application.

Moreover, the primary immune cell culture has been proven to be very beneficial in the study of immune repertoire to produce antibodies and antigens for the COVID-19 virus. As per the findings by the scientists from the German Primate Center in Gottingen, the COVID-19 virus could block the infection in lung cells with TMPRSS2, an inhibitor for serine protease. The results have been validated with the experiment using primary cell culture.

Primary Cell Culture Market Segmentation

Based On the Product Insights, the market is segmented into primary cells, reagents and supplements and media

Based On the Separation Methods Insights, the market is segmented into explant method, enzymatic degradation, mechanical separation and others

Based On the Cell Type Insights, the market is segmented into animal and human.

Based on the Application Insights, the market is segmented into tissue culture & tissue engineering, vaccine production, gene therapy and regenerative medicine, toxicity testing and drug screening, cancer research, model system, virology, prenatal diagnosis, stem cell therapy and others.

Based on the Regional Insights, the market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa

Browse through Grand View ResearchsBiotechnology IndustryResearch Reports.

Market Share Insights:

Key Companies Profile:

The companies are launching new and advanced products for primary cell culture. The new Gibco CTS OpTmizer Pro SFM helps in enhancing donor T (lymphocyte) cell proliferation. It is a new media solution for targeting the metabolism of healthy donors, for the efficient production of cost-effective cell therapies.

Some of the prominent players in the primary cell culture market include:

Order a free sample PDF of the Primary Cell Culture Market Intelligence Study, published by Grand View Research.

About Grand View Research

Grand View Research is a full-time market research and consulting company registered in San Francisco, California. The company fully offers market reports, both customized and syndicates, based on intense data analysis. It also offers consulting services to business communities and academic institutions and helps them understand the global and business scenario to a significant extent. The company operates across multitude of domains such as Chemicals, Materials, Food and Beverages, Consumer Goods, Healthcare, and Information Technology to offer consulting services.

Web: https://www.grandviewresearch.com

Media ContactCompany Name: Grand View Research, Inc.Contact Person: Sherry James, Corporate Sales Specialist U.S.A.Email: Send EmailPhone: 1888202951Address:Grand View Research, Inc. 201 Spear Street 1100 San Francisco, CA 94105, United StatesCity: San FranciscoState: CaliforniaCountry: United StatesWebsite: https://www.grandviewresearch.com/industry-analysis/primary-cell-culture-market

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To view the original version on ABNewswire visit: Primary Cell Culture Market Mechanical Separation Segment Is Expected To Witness A Lucrative CAGR Of 11.7% Till 2028, Owing To Advancements In Mechanical Separation Process | Grand View Research, Inc

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Primary Cell Culture Market Mechanical Separation Segment Is Expected To Witness A Lucrative CAGR Of 11.7 - Benzinga

A Vale man is in need of 50,000 for vital medical care in Mexico.

James Monks, 29, was diagnosed with multiple sclerosis following a wakeboarding accident last year.

In the months since he has been in steady decline, prompting his friends to launch a fundraiser to pay for stem cell therapy.

Alistair Taylor, a friend of James since their schooldays at Prince Henrys High School, has been delighted with the generosity shown thus far.

He said: Were practically joined by the hip so when he became unwell, I just had to do something.

We launched the Go Fund Me page at about 4.30pm on Wednesday and in less than three days weve raised more than 27,000.

Its been overwhelming, especially to see some of the names donating. There are people we went to school with and people we havent seen in years donating.

Theres been an awful lot of bad news recently so its so great to see people coming together and trying to help James.

James was diagnosed with MS following a wakeboarding accident last year. Before his diagnosis, friends described him as "the picture of health"

Alistair is hoping to raise 50,000 which would pay for James to go to Mexico to receive stem cell therapy.

The procedure would see James immune system wiped out by chemotherapy and rebuilt via stem cell transplant.

James is currently in discussions with medical consultants and is hoping to fly over in July.

Alistair added: Though there are no guarantees with MS, this therapy is James best chance at stopping the disease and giving James his life back.

MS is a time-sensitive disease and given his quick deterioration, the lottery of waiting to see how bad his condition gets leaves us no option but to seek this treatment abroad.

James Monks, with his partner Hazel Jenkins

Before diagnosis, James friends described him as the picture of health and he had been caring for his grandmother with dementia in Harvington.

He had also been helping his father, who has Parkinson's Disease, and his mother, who is battling breast cancer, all alongside his job.

Alistair believes this stress, coupled with a wakeboarding crash in August 2021, was the perfect storm to trigger the dormant MS in his system.

He added: James is a person who has always been generous with his time and always been the first to help others in need.

Now it is his time to be supported.

To donate, visit James Go Fund Me page here.

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Vale man with MS needs 50,000 for treatment in Mexico - Evesham Journal

-- Data Demonstrate Rapid and Complete Wound Healing, with Six of Eight (75%) Patients Achieving Complete Healing within Four Weeks; All Remained Healed Through End of Follow-Up ---- All Patients with Pain at Baseline Reported Zero Pain by Week 3 ---- KPI-012 was Well-Tolerated with No Significant Safety Issues Observed ---- On Track to Submit Investigational New Drug Application (IND) and Initiate Phase 2/3 Trial in 4Q 2022 --

ARLINGTON, Mass., May 01, 2022 (GLOBE NEWSWIRE) -- Kala Pharmaceuticals, Inc. (NASDAQ:KALA), a commercial-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies for diseases of the eye, today reported clinical data from a Phase 1b trial of KPI-012, its novel, cell-free secretome therapy for the treatment of severe ocular diseases driven by impaired healing. As previously disclosed, treatment with KPI-012 was well tolerated and resulted in significant improvements in patients with various persistent corneal epithelial defect (PCED) etiologies, with complete healing of the PCED in six of eight evaluable patients. The data will be presented on Tuesday, May 3 in a poster session at the 2022 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting.

The clinical activity observed to date with KPI-012 is encouraging, said Valeria Snchez-Huerta, M.D. FACS, Medical Director at Asociacin para Evitar la Ceguera en Mxico (Association to Prevent Blindness in Mexico) and an investigator in the Phase 1b trial. PCED is a disease of impaired corneal healing which, if left untreated, can lead to infection, corneal perforation and irreversible vision loss. Achieving rapid and complete wound healing, as well as a reduction in PCED-related pain, and an improvement in visual acuity and corneal opacity, in patients with a range of underlying etiologies is remarkable, particularly after such a short treatment duration. Based on these early data, I believe KPI-012 could become the first treatment to address PCED across all etiologies and I look forward to further evaluating its potential in later-stage studies.

PCED, which is defined as a persistent non-healing corneal defect or wound that is refractory to conventional treatments, is a rare disease with an estimated incidence in the United States of 100,000 cases per year. PCED can have various etiologies, including neurotrophic keratitis, surgical epithelial debridement, microbial/viral keratitis, corneal transplant, limbal stem cell deficiency and mechanical and chemical trauma and can lead to corneal ulceration, perforation, stromal scarring, secondary infections and significant vision loss.

Healing after corneal injury follows a highly coordinated process involving growth factors, cell signaling, proliferation, migration and extracellular matrix remodeling. In patients with PCED, there is an imbalance of key biomolecules, including growth factors and cytokines, which results in significant inflammation, impaired innervation and disruption of the protective corneal epithelial and stromal layers. KPI-012 was designed specifically to address this imbalance: it is a novel, human bone marrow-derived mesenchymal stem cell (MSC) secretome containing numerous human biomolecules, including protease inhibitors, matrix proteins, growth factors and neurotrophic factors, that provide a multifactorial mechanism of action to address impaired corneal healing across numerous etiologies.

We are pleased to present these exciting data from the first KPI-012 clinical trial, said Kim Brazzell, Ph.D., Head of Research and Development and Chief Medical Officer at Kala Pharmaceuticals. These data, which served as the foundation for our acquisition of Combangio last year, highlight the potential of KPI-012 to deliver a novel approach to treating PCED, as well as other rare front and back of the eye diseases. Our goal remains to advance KPI-012 into a Phase 2/3 trial for PCED later this year, as we aim to deliver new and better options to people living with severe ocular surface diseases.

The poster presentation is now available on the Kala Pharmaceuticals website at https://investors.kalarx.com/presentations.

Highlights from the ARVO Presentation

The single-arm, prospective, open-label Phase 1b clinical trial enrolled 12 patients, including three who were enrolled in a safety lead-in cohort and nine enrolled in an efficacy cohort. Within the efficacy cohort, patients presented with PCED of various etiologies and durations ranging from 15 to 871 days. Patients were treated with twice daily KPI-012 for up to four weeks, with follow-up occurring at two, four and 12 weeks after their last dose of therapy. The key efficacy endpoint was complete healing of the corneal defects evaluated by corneal staining. Other efficacy endpoints included reduction in defect size, visual acuity, and corneal opacity. Safety measures included tolerability/pain, intraocular pressure and adverse events.

Eight patients were evaluable for efficacy assessment; one participant was ineligible due to a non-treatment related adverse event. Improvement was seen in seven of the eight evaluable patients, with six of the eight achieving complete healing by the end of Week 4, including four patients who were healed by the end of Week 1 and one patient who was healed by the end of Week 2. All six healed patients remained healed through the end of the follow-up period. In addition, improvement in PCED lesion size was observed in both patients who did not experience full wound healing. Across all eight patients, the mean improvement in lesion size from baseline to end of treatment was -16.23 mm. KPI-012 was well-tolerated in the trial.

Clinical Development Plans

Kala plans to file an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) and, subject to regulatory clearance, initiate a Phase 2/3 clinical trial of KPI-012 in PCED patients in the fourth quarter of 2022. Kala believes this trial could serve as the first of two required pivotal trials. The FDA has granted KPI-012 Orphan Designation for the treatment of PCED and the Company believes it could also meet the criteria for fast-track and breakthrough designations.

In addition, Kala believes the multifactorial mechanism of action of KPI-012 also makes it a platform technology and is evaluating KPI-012 for potential expansion toindicationsfor rarefront of the eye diseases, such as limbal stem cell deficiency and Sjogrens Syndrome, as well as select rareback of the eye diseases, such as retinitis pigmentosa and optic neuritis.

About Kala Pharmaceuticals, Inc.

Kala is a commercial-stage biopharmaceutical company focused on the discovery, development, and commercialization of innovative therapies for diseases of the eye. Kala has applied its AMPPLIFYmucus-penetrating particle (MPP) Drug Delivery Technology to two ocular therapies, EYSUVIS(loteprednol etabonate ophthalmic suspension) 0.25% and INVELTYS(loteprednol etabonate ophthalmic suspension) 1%. The Company also has a pipeline of development programs including a clinical-stage secretome product candidate, KPI-012, initially targeting persistent corneal epithelial defects (PCED) and multiple proprietary new chemical entity (NCE) preclinical development programs targeted to address unmet medical needs, including both front and back of the eye diseases. For more information on Kala, please visitwww.kalarx.com.

Forward Looking Statements:

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. Any statements in this press release about Kalas future expectations, plans and prospects, including but not limited to statements about Kalas expectations with respect to KPI-012, the future development or commercialization of KPI-012, conduct and timelines of clinical trials, Kalas plans to progress its pipeline of preclinical development programs targeted to address front and back of the eye diseases, constitute forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including those discussed in the Risk Factors section of Kalas Annual Report on Form 10-K, most recently filed Quarterly Report on Form 10-Q and other filings Kala makes with the Securities and Exchange Commission. These forward-looking statements represent the Companys views as of the date of this release and should not be relied upon as representing the Kalas views as of any date subsequent to the date hereof. Kala does not assume any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Investor Contacts:

Jill Steierjill.steier@kalarx.com 781-996-5252

Hannah Deresiewiczhannah.deresiewicz@sternir.com 212-362-1200

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Kala Pharmaceuticals to Present Clinical Data for KPI-012, its Mesenchymal Stem Cell Secretome Product, for the Treatment of PCED at the 2022 ARVO...

Long-term clinical trial data continues to support the use of axi-cel (axicabtagene ciloleucel) in treating patients with relapsed/refractory large B-cell lymphoma (LBCL), according to data presented at the 2022 Tandem Meeting.

Researchers presented five-year data from the phase 2 ZUMA-1 clinical trial, including the one- and two-year event-free survival findings.

In this updated five-year analysis, axi-cel induced long-term (overall survival) ... among treated patients, according to Dr. Caron A. Jacobson, the medical director of the Immune Effector Cell Therapy Program and senior physician at Dana-Farber Cancer Institute and an assistant professor of medicine at Harvard Medical School. She continued, Between the four- and five-year analysis, the time to next therapy curve remains stable and 92% of patients remained alive without a need for subsequent therapy, which may be suggestive of a cure in these patients.

Investigators reported a five-year overall survival rate of 42.6% following treatment with axi-cel. In the population of patients who experienced a complete response, the five-year overall survival rate was 64.4% and the median overall survival was not reached. Additionally, 63% of complete responders were alive at the five-year data cut off. At the four-year data cutoff, one patient died at month 63 and one experienced progressive disease at month 54.

To be considered for treatment, patients were required to have LBCL, including diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma or transformed follicular lymphoma. Patients were also required to have not responded to their last chemotherapeutic treatment or have relapsed 12 months or less following autologous stem cell transplant, which is a stem cell transplant using the patients own healthy cells. Treatment with a previous anti-CD20 monoclonal antibody and anthracycline was also necessary.

Those who underwent treatment received a conditioning regimen of cyclophosphamide and fludarabine for three days to get them ready for the main line of treatment. This was followed by axi-cel.

The main goal of the study was overall response rate with first response assessment four weeks following infusion. Key secondary end points included overall survival, safety and translational evaluations.

A total of 111 patients were enrolled on the study, eight of whom did not undergo treatment for one of the follow reasons: side effects (four patients), no measurable disease (two patients), death due to disease progression (one patient), and manufacturing failure (one patient); this left 103 patients to undergo conditioning. Of these patients, two were not treated due to side effects and death, respectively.

The data cutoff was Aug. 11, 2021 and the median follow-up was 63.1 months.

Additional findings from the trial highlighted a median time to next anticancer therapy of 8.7 months following infusion. A total of 34% of patients were alive at cutoff with no subsequent therapy or retreatment with axi-cel. Two patients who had prior progression underwent new anticancer therapy.

The five-year overall survival rates among those who had or had not experienced an event-free survival at month 12 were 5.3% vs 90.9%, respectively.

Events were classified as instances when the cancer recurred or became worse.

The median overall survival was 8.3 months among those who experienced an event and was not reached in those who did not experience an event. Additionally, the five-year overall survival rates among those who did or did not have an event at month 24 were 11.3% and 92.3%, respectively. Moreover, the median overall survival in both respective groups was 9.2 months and not reached.

Investigators also determined that early CAR-T cell expansion was associated with ongoing response at 60 months. The median peak CAR T levels appeared to be numerically higher in those who had an ongoing response at month 60 and lower in those who relapsed or did not respond to treatment. Similarly, another trend was observed in those who experienced CAR-T cell expansion by area under the curve from day 0 to 28.

A total of 58% of patients had died by the cutoff date. No new safety signals had been observed as of the five-year data cutoff, including serious side effects or secondary malignancies related to treatment.

Patients most commonly died due to progressive disease (45 patients), side effects (four patients), secondary malignancies (one patient) or other reasons (nine patients).

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Long-Term Axi-Cel Data 'May Be Suggestive of a Cure' in Patients With Large B-Cell Lymphoma - Curetoday.com