Latest news with #GvHD
Yahoo
5 hours ago
- Business
- Yahoo
4 Emerging Graft versus Host Disease Therapies That Could Change the Treatment Landscape
Key companies, such as CSL Behring, Equillium, Biocon, and medac, are advancing their assets through late-stage graft versus host disease clinical trials, driving innovation in the graft versus host disease market and creating significant growth opportunities. LAS VEGAS, June 12, 2025 /PRNewswire/ -- Graft versus host disease (GvHD) is an immune-driven disorder caused by a complex interplay between the donor's and recipient's adaptive immune systems. It typically manifests in two main forms: acute (aGvHD) and chronic (cGvHD). As per DelveInsight's analysis, approximately 60,000 allogeneic transplants and around 55,000 GvHD cases occurred in the 7MM in 2024. These numbers are projected to grow at a notable CAGR over the forecast period from 2025 to 2034. Corticosteroids like prednisone and methylprednisolone are the primary first-line treatments, often combined with other immunosuppressants. Mild GcHD is managed with topical steroids, while systemic cases require stronger immunosuppressive therapy. Several treatments for GvHD have received FDA approval in the US, including ORENCIA (abatacept), JAKAFI/JAKAVI (ruxolitinib), IMBRUVICA (ibrutinib), and REZUROCK (belumosudil), among others. In the upcoming GvHD treatment market landscape, there are a plethora of companies investigating agents in various stages of development. To know more about the graft versus host disease clinical trials market, visit @ Graft versus Host Disease Market DelveInsight estimates that the market size for GvHD is expected to grow from USD 1.4 billion in 2024 with a significant CAGR of 8.2% by 2034. This anticipated growth is driven by advancements in treatment options, greater healthcare access, and a rising prevalence of the condition, which together foster higher demand for innovative and effective therapies. The current pipeline for graft versus host disease includes a range of drugs with diverse mechanisms of action (MoA). CSL964 (Alpha 1 Antitrypsin), EQ001 (Itolizumab; Bmab600), MaaT013, and MC0518 are the most promising drugs that are in the late-stage of development for GvHD treatment. Ongoing research and current trials have the potential to change the GvHD market. Keen to know how the GvHD market will evolve by 2034? Find out @ Graft versus Host Disease (GvHD) Market Forecast Apart from this, several GvHD drugs currently in the early stages of development include RLS-0071 by ReAlta Life Sciences, Vimseltinib by Deciphera Pharmaceuticals, ASC-930 by ASC Therapeutics, RGI-2001 by REGiMMUNE, CYP-001 by Cynata Therapeutics, arsenic trioxide (As2O3) by BioSenic (Medsenic), TRX103 (Tregs) by Tr1X, TCD601 (Siplizumab) by ITB-MED, F-652 by Evive Biotech, RHPRG4 by Lubris BioPharma, XBI302 by Xbiome, RG6287 by Genentech, ALTB-168 by AltruBio, and SER-155 by Seres Therapeutics. Now, let's examine the late-stage pipeline therapies under investigation for GvHD treatment CSL Behring's ZEMAIRA CSL964 Alpha-1 Antitrypsin, an Alpha1-Proteinase Inhibitor (A1-PI) developed by CSL Behring, is being studied for the treatment of steroid-refractory acute graft-versus-host disease (aGvHD) and for the prevention of aGvHD in high-risk patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). It is currently in Phase III clinical trials for the treatment of steroid-refractory aGvHD and is also being evaluated in Phase II/III trials for its potential in preventing aGvHD. Equillium/Biocon's EQ001 EQ001 (Itolizumab; Bmab600) is a first-in-class immune-modulating antibody that targets CD6 to suppress the activation and migration of harmful T cells responsible for releasing pro-inflammatory cytokines in autoimmune and inflammatory conditions such as GvHD, moderate-to-severe uncontrolled asthma, and lupus nephritis. By acting on the CD6-ALCAM signaling pathway, Itolizumab selectively inhibits pathogenic effector T cells (Teffs) while preserving regulatory T cells (Tregs), which are essential for immune system balance. Currently, EQ001 is undergoing a Phase III clinical trial in combination with corticosteroids as a first-line therapy for March 2025, Equillium reported topline Phase III EQUATOR trial results for itolizumab in first-line aGVHD. While the study did not show a meaningful difference in CR or ORR at Day 29 versus placebo, itolizumab demonstrated statistically significant and clinically meaningful improvements in longer-term outcomes, including CR at Day 99, duration of response, and failure-free survival. In April 2025, the FDA declined to grant Breakthrough Therapy designation or support an Accelerated Approval pathway for itolizumab, citing limitations in the EQUATOR study data. Discover which therapies are expected to grab major GvHD market share @ Graft versus Host Disease Treatment Market MaaT Pharma's MaaT013 MaaT013 is a standardized, donor-derived microbiome ecosystem therapy characterized by high richness and diversity. It includes BUTYCORE, a consortium of bacterial species known for producing anti-inflammatory short-chain fatty acids. The therapy is designed to reestablish the balance between the patient's gut microbiome and immune system, aiming to enhance immune tolerance and responsiveness, thereby addressing steroid-resistant, gastrointestinal-dominant aGvHD. MaaT013 has been granted Orphan Drug Designation (ODD) by both the US FDA and the EMA. In December 2024, MaaT Pharma shared encouraging updated results from its Early Access Program at the ASH 2024 Annual Meeting. Subsequently, in January 2025, the company announced promising topline findings from the Phase III ARES trial, where MaaT013 achieved a 62% overall gastrointestinal response rate by Day 28, marking a significant advancement as a third-line treatment for GI-aGvHD. medac's MC0518 MC0518 is an investigational mesenchymal stromal cell (MSC) therapy developed by Medac GmbH, currently undergoing clinical trials for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGvHD) following allogeneic hematopoietic stem cell transplantation. This therapy leverages the immunomodulatory properties of MSCs to mitigate the severe inflammatory responses characteristic of SR-aGvHD, a condition where the donor's immune cells attack the recipient's tissues despite steroid treatment. The IDUNN trial, a pivotal Phase III study, is evaluating the efficacy and safety of MC0518 compared to the best available therapy (BAT) in pediatric and adolescent patients. The primary endpoint is the overall response rate (ORR) at Day 28, with secondary objectives including overall survival (OS) up to 24 months and freedom from treatment failure (FFTF) within six months. Preclinical assessments have demonstrated that MC0518 is well-tolerated, with no evidence of tumorigenicity or significant adverse effects in animal models. Discover more about drugs for GvHD in development @ Graft versus Host Disease Clinical Trials The anticipated launch of these emerging therapies for GvHD are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the GvHD market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight's latest published market report, titled as Graft versus Host Disease Market Insight, Epidemiology, and Market Forecast – 2034, will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the GvHD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The GvHD market report offers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total allogenic transplant cases Total Graft Versus Host Disease Cases Type-specific Cases of Graft Versus Host Disease Acute Graft Versus Host Disease Cases by Grading Acute Graft Versus Host Disease Cases by Organ Involvement Chronic Graft Versus Host Disease Cases by Grading Chronic Graft Versus Host Disease Cases by Organ Involvement Total Treated Cases of Graft Versus Host Disease Mortality Adjusted Treated Cases of Graft Versus Host Disease The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM GvHD market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this GvHD market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the GvHD market. Also, stay abreast of the mitigating factors to improve your market position in the GvHD therapeutic space. Related Reports Graft versus Host Disease Epidemiology Forecast Graft versus Host Disease Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted GvHD epidemiology in the 7MM, i.e., the United States, EU4 (Germany, Spain, Italy, France) and the United Kingdom, and Japan. Graft versus Host Disease Pipeline Graft versus Host Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key GvHD companies, including Abbisko Therapeutics, Equillium, Theriva Biologics, Seres Therapeutics, CytoMed Therapeutics, Beijing Tide Pharmaceutical, CTI BioPharma, ViGenCell, Lipella Pharmaceuticals, Cellestia Biotech, Seres Therapeutics, Jiangsu HengRui Medicine Therapeutics, Genentech, AltruBio, Orca Bio, GSK, Amgen, among others. Acute Graft versus Host Disease Pipeline Acute Graft versus Host Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acute GvHD companies, including MaaT Pharma, Medac, CSL Behring, Humanigen, Ironwood Pharmaceuticals, ReAlta Life Sciences, Roche, Incyte Corporation, among others. Ocular Graft versus Host Disease Pipeline Ocular Graft versus Host Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ocular GvHD companies, including Cambium Medical Technologies, Glia LLC., Ocular Discovery, Selagine, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact UsShruti Thakur info@ +14699457679 Logo: View original content: SOURCE DelveInsight Business Research, LLP
Associated Press
03-06-2025
- Business
- Associated Press
Priothera Strengthens Board with Appointment of Industry Veteran, Dr. Hans Menssen
Highly experienced clinical development executive joins Priothera Board as mocravimod progresses through global Phase 3 trial as adjunctive and maintenance treatment in AML patients undergoing allo-HCT Saint-Louis, France and Dublin, Ireland – 3rdJune 2025– Priothera Ltd., a late-stage biopharma company pioneering the development of mocravimod, a novel oral sphingosine 1 phosphate (S1P) receptor modulator, to treat hematologic malignancies, today announced the appointment of Dr. Hans Menssen, MD, PhD, BBA, to its Board of Directors. Dr. Menssen most recently served as Senior Global Program Clinical Head at Novartis Oncology, where he led clinical programs across acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), multiple myeloma, chronic myeloid leukemia (CML), myelofibrosis, acute and chronic GvHD, and B-cell malignancies, with a focus on registration-stage development. Over his career, in addition to Novartis, he has held senior roles at Bayer Schering Pharma and Philogen SpA, with a track record of supporting oncology innovation across modalities including small molecules, antibodies, and cell therapies. ' Hans brings deep strategic insight in hematology, clinical development, and regulatory science,' said Florent Gros, Co-Founder and CEO of Priothera. ' His experience across global drug development programs and his understanding of what it takes to bring innovative therapies to patients will make him a valuable voice on our Board as we progress the global Phase 3 MO-TRANS study of mocravimod in AML patients undergoing allo-HCT. We are very pleased to welcome him to the Board.' A board-certified hematologist and oncologist, Dr. Menssen also holds academic appointments at Charité – University Medicine Berlin in his capacity as assistant professor (Priv-Doz. Dr. med), where he continues to teach and supervise medical research. He has contributed to numerous clinical and translational research efforts throughout his career. He earned his medical degree from the University of Cologne, completed postdoctoral research at the Wistar Institute at the University of Pennsylvania, and holds a Bachelor of Business Administration in health economics from GSBA Zurich in partnership with SUNY (State University of New York). ' I am very pleased to join Priothera's Board at such a key point in its evolution,' said Dr. Hans Menssen. ' Mocravimod represents a promising and differentiated approach to improving outcomesin AML patients undergoing allo-HCT, and I look forward to supporting the team in advancing this important program.' Priothera's Board of Directors is composed of: *** About mocravimod Mocravimod (KRP203) is a synthetic S1P receptor modulator being developed for the adjunctive treatment of AML to enhance the curative potential of allo-HCT. Mocravimod's dual mechanism of action preserves the graft-versus-leukemia (GvL) effect, critical for eliminating cancer cells while reducing the risk of graft-versus-host disease (GvHD), a major complication following allo-HCT. This novel treatment approach – mocravimod being the only S1P receptor modulator in development to treat blood cancers – tackles a high unmet medical need and aims to improve patients' quality of life. About Priothera Priothera is a late-stage biopharma company pioneering the development of mocravimod, a potential new standard of care in hematologic cancers, in combination with cellular therapies such as hematopoietic cell transplantation and CAR-T cell therapies. Mocravimod is being developed as an adjunctive and maintenance therapy for hematological malignancies, focusing initially on acute myeloid leukemia (AML), in combination with allogeneic hematopoietic cell transplant (allo-HCT). Mocravimod is currently the only treatment with the potential to reduce transplant side effects of graft-versus-host disease (GvHD) without compromising the graft's anticancer effect against leukemia (Graft-versus-Leukemia, or GvL), thereby enhancing the curative potential of allo-HCT. Founded in 2020, Priothera operates in France, with headquarters in Dublin. The company is led by a highly experienced management team with deep expertise in hematology, oncology, immunology and cell-based therapies. Priothera is backed by leading international life sciences investors, including Fountain Healthcare Partners, abrdn, EarlyBird Venture Capital, BEI and Bpifrance Grand Est. For more information please visit or follow Priothera on LinkedIn Contacts
Yahoo
02-06-2025
- Business
- Yahoo
MiNK Therapeutics Awarded Prestigious NIAID Grant to Advance Allo-iNKT Cell Therapy for Prevention of GvHD in Stem Cell Transplant Patients
Non-dilutive NIH funding supports development of MiNK's allogeneic iNKT platform for immune regulation in high-risk HSCT settings NEW YORK, June 02, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering allogeneic, off-the-shelf invariant natural killer T (iNKT) cell therapies, today announced it has been awarded a grant from the National Institute of Allergy and Infectious Diseases (NIAID), a part of the National Institutes of Health (NIH). The grant will support development of MiNK's allo-iNKT cell therapy platform for the prevention and treatment of graft-versus-host disease (GvHD) following hematopoietic stem cell transplantation (HSCT), in collaboration with the University of Wisconsin. 'This non-dilutive funding from NIAID underscores the growing recognition of iNKT cells as a unique and powerful modality in immune regulation,' said Jennifer Buell, PhD, President and Chief Executive Officer of MiNK Therapeutics. 'The work led by Dr. Gumperz and her team at the University of Wisconsin has provided important mechanistic insights into how allo-iNKT cells may not only prevent graft-versus-host disease (GvHD) but also improve the success of engraftment. Through our preclinical and clinical collaboration, we aim to address the needs of the nearly 50% of patients undergoing allogeneic stem cell transplants who are at risk for this serious and potentially life-threatening complication. This award both validates the promise of our iNKT platform and accelerates its development in a high-priority area of unmet medical need.' GvHD is a severe immune complication that can occur after allogeneic HSCT, often leading to multi-organ damage and high mortality. iNKT cells are uniquely suited for this setting due to their natural ability to regulate immune responses, promote tissue repair, and suppress inflammatory pathways. 'Our partnership with MiNK unites their cutting-edge iNKT manufacturing with our deep expertise in transplant immunology at the University of Wisconsin-Madison,' said Jenny E. Gumperz, PhD, Professor of Medical Microbiology & Immunology, University of Wisconsin School of Medicine and Public Health. 'iNKT cells can calm the destructive allo-immune response that drives GvHD, while preserving the patient's ability to fight infection—a balance current therapies struggle to achieve. NIAID's support allows us to speed this science toward the clinic and, ultimately, give transplant patients a safer path to long-term survival.' About MiNK Therapeutics MiNK Therapeutics is a clinical-stage biopharmaceutical company pioneering the development of allogeneic invariant natural killer T (iNKT) cell therapies and precision-targeted immune technologies. MiNK's proprietary platform is designed to restore immune balance and drive cytotoxic immune responses across cancer, immune-mediated diseases, and pulmonary immune failure. MiNK's lead asset, AGENT-797, is an off-the-shelf, allogeneic iNKT cell therapy currently in clinical development for the treatment of graft-versus-host disease (GvHD), solid tumors, and critical pulmonary immune collapse. MiNK is also advancing a pipeline of T cell receptor (TCR)-based therapies and neoantigen discovery tools that enable tumor- and tissue-specific immune activation with broad potential application. With a scalable, cryopreserved manufacturing process and a differentiated mechanism that bridges innate and adaptive immunity, MiNK is committed to developing next-generation immune reconstitution therapies that are accessible, durable, and applicable across a wide range of indications. For more information, visit or @MiNK_iNKT. Information that may be important to investors will be routinely posted on our website and social media channels. Forward Looking Statements This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements regarding the therapeutic potential, anticipated benefit, plans and timelines of iNKT cells, as well as the collaboration between MiNK and Agenus. These forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially. These forward-looking statements are subject to risks and uncertainties, including the factors described under the Risk Factors section of the most recent Form 10-K, Form 10-Q and the S-1 Registration Statement filed with the SEC. MiNK cautions investors not to place considerable reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this press release, and MiNK and Agenus with no obligation to update or revise the statements, other than to the extent required by law. All forward-looking statements are expressly qualified in their entirety by this cautionary statement. Investor Contact917-362-1370 investor@ Media Contact781-674-4428communications@ Gumpertz et al., Harnessing invariant natural killer T cells to control pathological inflammation. Frontiers. 2022. Gumpertz et al., iNKT cells coordinate immune pathways to enable engraftment in nonconditioned hosts. Life Sciences Alliance. 2021.
Yahoo
16-05-2025
- Business
- Yahoo
Q1 2025 Mink Therapeutics Inc Earnings Call
Jennifer Buell; Independent Director; Mink Therapeutics Inc Christine Klaskin; Independent Director; Mink Therapeutics Inc Operator Thank you for standing by. My name is Rosell, and I will be your conference operator today. At this time, I would like to welcome everyone to the MiNK Therapeutics first quarter 2025 financial results. After the speaker remarks, there will be a question-and-answer session. If you would like to ask a question during this time, simply press start, followed by the number one on your telephone keypad. If you would like to withdraw your question, press start one again. I will now turn the conference call to Jack Jennifer Ball, head of investor relations, please go ahead. Thank you, operator, and thank you all for joining us today. Today's call is being webcast and will be available on our website for replay. I'd like to remind you that this call will include forward-looking statements, including those related to our clinical development, regulatory and commercial plans, timelines for data release and partnership opportunities. These statements are subject to risks and uncertainties. Please refer to our SEC filings available on our website for a detailed description of these risks. Joining me today are Dr. Jennifer Buell, President and Chief Executive Officer, and Christine Klaskin, principal Financial and Accounting Officer. Now, I'd like to turn the call over to Doctor Bell to highlight our progress from this quarter. Jennifer Buell Thanks very much, Jack. I appreciate it and thank you all for joining us today. This quarter we've made meaningful progress towards our mission, and that's delivering scalable, durable, off the shelf iNKT cell therapies to patients with solid tumors and other immune-related diseases. In the Q1 of 2025 we executed across three critical areas, and those include clinical progress. We presented new data in solid tumors, specifically in second line gastric cancer, at the inaugural AACR IO conference, and we showed immune activation and very early clinical activity and responses in patients who were otherwise refractory to checkpoint modulating the capital side, we continue to reduce our operating cash burn and operate extremely efficiently with a further reduction of about 47% year on year, preserving our ability to invest in our core programs. We've been able to continue to advance our clinical trials through external financing, and those include the advancement of our second line gastric cancer and also the development of two programs, one in ARDS and the other in GvHD, which I'll talk about in just a moment. We are advancing confidential discussions for proposals, each of which could extend our runway and accelerate our impact, and I'm going to go into those in some detail, just a as is core to our strategy, and it has been. It's essential to unlocking the full potential of our technology, our iNKT platform in oncology, in immunology, inflammatory diseases, and of course our next generation engineered cell therapy. Our platform is really broad and deep. It allows us to take full advantage of what these cells can do, and we remain at the forefront of advancing iNKT cell biology off the shelf in patients with immune-related today I'm pleased to share that we have 3 distinct proposals, each aligned with one of our key therapeutic areas in oncology and cancer. We're focusing on advancing 797 and solid tumor cancers, building on the momentum from our gastric and testicular cancer programs. I'll highlight a little bit more about some upcoming data in testicular cancer, but in the meantime, we did just recently present data at ACR that I'll share with you in a few on immunology and inflammatory conditions. This supports our development of iNKT cells in acute inflammation such as respiratory distress, as well as inflammatory conditions such as graft versus host disease, an area of great interest to our team. And a proposal on our next generation pipeline. This encompasses our car iNKT therapy, our TCR iNKT therapy, and our proprietary neo antigen discovery platforms with the aim of creating highly targeted off the shelf immune therapies. These transactions and proposals are not exclusive, in fact, given their distinct focus areas and complementary capabilities of these proposed partners. These proposals may be mutually reinforced reinforcing, each bringing differentiated capital, infrastructure, and scientific expertise to accelerate progress within their respective together, these proposals reflect strong external conviction in the value of our iNKT platform and represent a rare opportunity to diversify capital, reduce pollution, and accelerate development in multiple high impact areas for MiNK. We're engaging with focus and urgency and expect to advance one or more of these in the very near term. We'll continue to keep you abreast, and we plan to host a more formal presentation regrouping with our key stakeholders to be able to announce these in due I'm going to turn and highlight a couple of key elements of our programs and our progress to date. In solid tumors, we're particularly encouraged by the continued momentum in our solid tumor program, and as I mentioned at the ASCO GI and AACR IO inaugural meetings, we presented new data from our phase 2 investigator sponsored trial that's being housed at Memorial Sloan Kettering under the leadership of Dr. Yelena Janjigian, the chief of gastrointestinal study is evaluating HLA NKTs or agenT-797, in combination with two differentiated checkpoint modulating antibodies, botensilimab and balstilimab. On top of standard of care chemotherapy in patients with second line advanced gastric cancer. This is a population with no effective therapies in the second line setting. The data demonstrate that iNKT cells when delivered systemically, they rapidly traffic to the tumor microenvironment where they engage both innate and adaptive immune pathways. This is different than what you see with conventional T cells and NK cell activity, what we've observed is that we were looking at tumors that effectively were in immune desert. No CD8 T cells, therefore no ability to immunologically manage the cancer. And what we observed is upon systemic infusion of 797, we can transform a cold tumor into an immunologically active or hot tumor, promoting these very important CD8 T cells infiltration, activating dendritic cells and reversing immune exhaustion, and these are in cancers that are resistant to PD1 blockade. These findings support our core thesis. iNKT cells act as immunologic first responders, initiating multi-layered anti-tumor responses through direct tumor killing or cytotoxicity and immune orchestration. We anticipate sharing additional updated clinical updates later this year in the beginning of next parallel, we expect a peer reviewed publication describing a complete response in a patient with metastatic testicular cancer. This patient was treated on our phase 1 trial with 797, and they were treated with 797, or alloy iNKTs alone in this setting. The patient had progressed through platinum-based chemotherapy, autologous stem cell transplantation, radiation, and checkpoint and idget-based regimens prior to enrolling in the trial. Following a single infusion of agenT-797, the patient achieved a durable, complete clinical, radiological and biochemical remission. Treatment was delivered without lymph depletion or HLA matching and showed no evidence of cytokine relief syndrome or post treatment analysis reveals elevated interferon gamma. We're observing some robust tumor activity by immune effector cells, and we're also observing peripheral persistence. Our cells still continue to persist beyond 6 months, which give us a large therapeutic window to continue to dose these patients. This case exemplifies the unique biology of iNKT cells, their ability to rapidly ho to tumors, dismantle immunosuppressive barriers, and activate both NK and CD8 T cells. Even in tumors previously unresponsive to immune our gastric cancer findings, this finding reinforces iNKT cells as a novel, off the shelf immune therapy platform with the potential to deliver durable benefit and hard to treat solid tumors. Beyond oncology, we're continuing to advance 797 in immune-related diseases such as respiratory distress, acute respiratory distress syndrome, and graft versus host disease. Our INKP platform showed early on and continues to show compelling promise in immune-mediated diseases where inflammation, immune dysregulation, and poor treatment options converge to create really devastating clinical realities for ARDS, a life-threatening condition with no FDA approved therapies, AgenT-797 is shown the potential to change the treatment paradigm. As we published in Nature Communications and presented at the American Thoracic Society, our data demonstrated improved survival and meaningful inflammatory control and critically ill ventilated patients, many of whom would otherwise face mortality rates exceeding 70%. We, on the other hand, observed survival rates exceeding 70%, truly signals observed in a high-risk ICU population is a powerful indication of iNKT's steroid resistant anti-inflammatory activity and their ability to reduce secondary infections and their impact on pulmonary function and immune tonology. Consistent with the new leadership and priorities at the FDA, we are working urgently to make our therapies accessible through well-designed clinical trials, compassionate use programs, and accelerated development pathways that reflect the seriousness and unmet nature of these conditions. The agency's increased receptivity to novel immune-based approaches, especially in indications like ARDS, give us further confidence in our regulatory path graft versus host disease, we're prepared to initiate a phase 1 trial, a 797 of patients undergoing allergenic bone marrow transplant. We've spoken to you about this before, and as advancing this program has been in part contingent upon securing financing to be able to advance this really responsibly and efficiently. TBHC remains one of the most severe and unpredictable complications of transplants. Often leading to often leading to multi-organ damage, prolonged hospitalization, poor quality of life, and disease progression. Our iNKT approach, which requires no lymph depletion, no genetic matching, and poses minimal to no risk of GvHD itself, is uniquely suited for this setting. The trial will be supported primarily through external partnerships, allowing us to advance this high impact program with minimal capital reinforcing the momentum, we were recently selected for probable funding by the National Institute of Allergy and Infectious Diseases. We expect the formal award. We were recently notified just a couple of weeks ago that we expect the formal award by June. This would provide critical, non-dilutive funding and a strong endorsement from one of the world's most respected federal research agencies and with this award, MIC will launch a collaboration of pre-clinical and clinical research with our colleagues and scientific advisors at the University of ARDS and TVVHC represent a large underserved market where MP platform can deliver outsized impact. We remain committed to advancing these programs rapidly, guided by scientific conviction and a growing mandate to bring transformative immune-based therapies to patients in need. And on the operational efficiency side, we have been continuing to expand our work in the field by reducing and reducing operating burn. We have continued to retain our top scientific leaders. We continue to internalize operational execution of our programs, including data management and clinical research activities which has allowed us to operate far more efficiency in a far less capital-intensive actions further reflect our commitment to financial discipline and operational focus. With that, I'll turn the call over to Christine for a review of the financials. Christine Klaskin Thank you, Jen. We ended the quarter with a cash balance of USD3.2 million cash used in operations for the three months ended March 31, 2025, was USD1.3 million. This is reduced from USD2.5 million for the same period in 2024. Our net loss for the first quarter of 2025 was USD2.8 million or USD0.70 per share. This compares to USD3.8 million or USD1.10 per share for the first quarter of 2024. Thank you. And operator, we are now ready to take questions. Operator At this time, I would like to remind everyone in order to ask a question, press star, then the number one in your telephone keypad. We will pause for just a moment to compile the roster. Your first question comes from the line of Emily Bodnar with HC Wainwright. Please go ahead. Hi, good morning. Thanks for taking my questions. This first one on the testicular patient. Congrats on the CR there. Are you able to say how long after treatment was initiated that the CR was observed and if you can comment on, I guess your overall plan in testicular cancer going forward and if there's any other indication that you're still looking at. Jennifer Buell Emily, thanks for the question, and this is this publication is expecting out somewhat imminently, and that information will be in the publication, but I can share with you this is a unique case and it exemplifies the value of immune therapy. It's not surprising that in the 12 month follow up period, the patient actually had disease stabilization, and we were monitoring the patient and not less than a year after that, so 24 months, the patients came back in to see the PI of the study with a complete remission and no other treatment. So this patient had been treated by the investigator, continued his clinical treatment with the investigator clinical observations, with no additional treatment put into the patient after the single infusion. And the complete response was formally designated at month 24 after the initial treatment of in addition, the patient had multiple lesions. The disease was really quite widespread, and you'll see this outlined in the paper and what was really quite intriguing was disease reduction really in all of the lesions, including the liver, and that's a very important biomarker for us. We are seeing activity by NKTs in active disease in the liver. We've observed this in our phase one study, we've also observed it in our gastric cancer trial, and now we've observed it with this testicular cancer patient has a lung mat that appears to be indolent at this point, that he does not want to undergo a biopsy, but the disease appears to the nodule appears to have nothing but dead tissue. Based on all of the scanning that has been completed. So we're really quite enthusiastic about this, and it has encouraged us to continue to do another survival sweep and clinical interrogation of other patients on the trial. What we found to be most intriguing when we presented the data, we presented essentially with a median of 12 months of follow up and we had some responses in the trial, but predominantly we saw long-term disease stabilization and this includes in patients with pancreatic cancer, non-small cell lung cancer appendiceal and gastric. But in those observations when we stopped the, we concluded the follow up period of the trial, we may be underestimating the ultimate clinical benefit of iNKT cells. So, we'll be getting a further clinical sweep of these patients and updating the field on the findings. Okay, great. And on the phase two gastric trial, are you still kind of on track for initial efficacy data in the second half of this year? Jennifer Buell That's what we're on target to do. They're continuing to enroll, and we'll be in touch with Dr. Janjigian about the soonest presentation. So we are, we have been looking at some GI specific conferences as well as some of the major oncology conferences for an update and a clinical presentation. It's ultimately within her discretion, so it will be no later than early next year. That would be the latest, but we're still on track. We're still targeting to get something out by the end of this year. Okay, great. Lastly, I'm just curious in terms of the funding that you mentioned from the NAYA if you've kind of heard of any changes or delays in government funding just with all this new news lately. Jennifer Buell Well, I'm with you. We, so we had heard of a delay. We expected this at the beginning of the year. So, the 6-month delay is, the delays that we have seen globally have impacted us. However, we're, we were reassured to get a formal notification from NAYA that we can expect to hear that we had probable funding and can expect to hear conclusively in June. NAYA has not been as heavily impacted as some of the other agencies, and so this for us is a high priority for the government and for the agency graft versus host disease, and our technology presents a really novel way of addressing this problem with engraftment success and reduction in GvHD and better clinical outcomes. So, we're optimistic and the most recent correspondence from the government continues to boost our optimism. Okay great. Thanks for taking the questions. Thank you. Operator Again, if you would like to ask a question, press star one in your telephone cable. Your next question comes from the line of Max with William Blair. Please go ahead. And, one, thanks for the update, wondering if, maybe just go over some of the details of the GVAC trial. Are you still planning on booking acute patients and maybe any thoughts on kind of prior treatments or what type of patients you'll be looking to enroll. Jennifer Buell Yeah. Thanks so much, Matt. So, there are two places where we will ultimately be setting into GvHD, and the first with this financing support and with the priority at University of Wisconsin to bring this forward, and this is under the leadership of Jenny Gumpers, who's a scientific advisor and wrote the seminal paper on the mechanism of iNKTs and GvHD. The opportunity for us in steroid refractory acute GvHD represents a very fast path forward. That's what we have identified and developed a phase one program for that. We have also developed a phase one program for prophylaxis, and that's engraftment success and a reduction in GvHD. And in that disease setting, we have a pathway that may be even faster. Both of these will be going to the regulators for a discussion with them imminently. And then we will choose the priority program to advance, but both opportunities for us, I'm going to have Tiago Favano, who's been working with the investigators in the clinical development of this speak just a little bit more to the enrichment that we're planning at this time. Hi, thanks for your question. So, for the phase one, we're going to explore not only the GvHD but also a few other complications of transplants that still represent an unmet need even though we do have available treatment. And drugs for prevention, but the other effects, they still represent unmet needs. So based on prior robot literature and some of our own studies, we expect the Ng not only to prevent or combat GvHD, but also to prevent infections, contribute to a better engraftment, faster and better engraftment. And and also prevent maintaining leukemia effects to prevent disease relapse. So we all know that on the treatment of GvHD patients get immunosuppressed and that makes it easier for them to have relapse or infections, which is a major we in this phase one, we are going to observe all these other effects on top of preventing the GvHD. Which paved the way for phase 2 that Jen explained, we will explore in treatment of steroid refractor GvHD and then another opportunity in prevention which represents an even faster way for approval. Jennifer Buell Thanks. And then, I'm going to add something to this. The, there are two things happening in parallel. One is the funding opportunity and if the award is as we anticipate it will be, which is the full committed funding, then we will have an opportunity to in our own hands, interrogate both prophylaxis, as well as mitigation in steroid refractory patients. And so that's why we've developed two programs to be able to do that. In the case that we can fund independently with the grant funding one program, there's a strategic collaborator who's at the table right now and has shared a proposal with us to advance the other, which is the prophylactic study. Okay, thanks for your please, Jen. Jennifer Buell Thank you, Matt. Operator That ends the session. I will now turn the call back over to Jennifer Buell for closing remark. Please go ahead. Jennifer Buell Thank you, operator. Thank you all for joining us today. We look forward to interacting with you in the upcoming days. Operator Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect. 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Yahoo
13-05-2025
- Business
- Yahoo
Graft Versus Host Disease (GvHD) Treatment Markets and Companies: Global Strategic Business Report 2025-2030
Pharmaceutical Investments in Novel Immunosuppressive Drugs and Biologics Have Expanded Treatment Choices Dublin, May 13, 2025 (GLOBE NEWSWIRE) -- The "Graft Versus Host Disease (GvHD) Treatment - Global Strategic Business Report" has been added to global market for Graft Versus Host Disease (GvHD) Treatment was estimated at US$2.9 Billion in 2024 and is projected to reach US$4.5 Billion by 2030, growing at a CAGR of 7.3% from 2024 to 2030. This comprehensive report provides an in-depth analysis of market trends, drivers, and forecasts, helping you make informed business decisions. The report includes the most recent global tariff developments and how they impact the Graft Versus Host Disease (GvHD) Treatment growth in the GvHD treatment market is driven by increasing stem cell transplant procedures, rising incidence of steroid-refractory GvHD cases, and advancements in targeted immunotherapy. As more patients undergo hematopoietic stem cell transplants for cancer and autoimmune diseases, the demand for effective GvHD prevention and treatment options continues to pharmaceutical investments in novel immunosuppressive drugs and biologics have expanded treatment choices, offering better long-term management of GvHD. The increasing role of biomarker-driven precision medicine is also shaping the market, enabling more personalized and effective treatment regimens. Furthermore, government funding and clinical research initiatives are accelerating the approval and commercialization of new GvHD therapies, ensuring sustained market ScopeThe report analyzes the Graft Versus Host Disease (GvHD) Treatment market, presented in terms of market value (US$ Thousand). The analysis covers the key segments and geographic regions outlined Organ Transplant Immunosuppressant Drug Class (Calcineurin Inhibitors, Antiproliferative Agents, mTOR Inhibitor, Steroids, Other Drug Classes); Organ Transplant Immunosuppressant Drugs Transplant (Kidney, Liver, Heart,Lung, Pancreas, Other Transplant Types).Geographic Regions/Countries: World; United States; Canada; Japan; China; Europe (France; Germany; Italy; United Kingdom; Spain; Russia; and Rest of Europe); Asia-Pacific (Australia; India; South Korea; and Rest of Asia-Pacific); Latin America (Argentina; Brazil; Mexico; and Rest of Latin America); Middle East (Iran; Israel; Saudi Arabia; United Arab Emirates; and Rest of Middle East); and Insights: Market Growth: Understand the significant growth trajectory of the Calcineurin Inhibitors segment, which is expected to reach US$1.9 Billion by 2030 with a CAGR of a 9.0%. The Antiproliferative Agents segment is also set to grow at 5.0% CAGR over the analysis period. Regional Analysis: Gain insights into the U.S. market, estimated at $800.8 Million in 2024, and China, forecasted to grow at an impressive 11.7% CAGR to reach $960.5 Million by 2030. Discover growth trends in other key regions, including Japan, Canada, Germany, and the Asia-Pacific. Key Questions Answered: How is the Global Graft Versus Host Disease (GvHD) Treatment Market expected to evolve by 2030? What are the main drivers and restraints affecting the market? Which market segments will grow the most over the forecast period? How will market shares for different regions and segments change by 2030? Who are the leading players in the market, and what are their prospects? Report Features: Comprehensive Market Data: Independent analysis of annual sales and market forecasts in US$ Million from 2024 to 2030. In-Depth Regional Analysis: Detailed insights into key markets, including the U.S., China, Japan, Canada, Europe, Asia-Pacific, Latin America, Middle East, and Africa. Company Profiles: Coverage of players such as AbbVie Inc., Astellas Pharma Inc., AstraZeneca plc, Bristol Myers Squibb, Eli Lilly and Company and more. Complimentary Updates: Receive free report updates for one year to keep you informed of the latest market developments. Select Competitors (Total 44 Featured): AbbVie Inc. Astellas Pharma Inc. AstraZeneca plc Bristol Myers Squibb Eli Lilly and Company F. Hoffmann-La Roche Ltd. Gilead Sciences, Inc. GlaxoSmithKline plc Incyte Corporation Jazz Pharmaceuticals plc Johnson & Johnson Services Inc. Merck & Co., Inc. Mesoblast Limited Novartis AG Osiris Therapeutics, Inc. Pfizer Inc. Sanofi SA Syndax Pharmaceuticals Takeda Pharmaceutical Company Ltd. Tonix Pharmaceuticals Tariff Impact Analysis: Key Insights for 2025Global tariff negotiations across 180+ countries are reshaping supply chains, costs, and competitiveness. This report reflects the latest developments as of April 2025 and incorporates forward-looking insights into the market analysts continuously track trade developments worldwide, drawing insights from leading global economists and over 200 industry and policy institutions, including think tanks, trade organizations, and national economic advisory bodies. This intelligence is integrated into forecasting models to provide timely, data-driven analysis of emerging risks and Included in This Edition: Tariff-adjusted market forecasts by region and segment Analysis of cost and supply chain implications by sourcing and trade exposure Strategic insights into geographic shifts Buyers receive a free July 2025 update with: Finalized tariff impacts and new trade agreement effects Updated projections reflecting global sourcing and cost shifts Expanded country-specific coverage across the industry Key Attributes Report Attribute Details No. of Pages 296 Forecast Period 2024-2030 Estimated Market Value (USD) in 2024 $2.9 Billion Forecasted Market Value (USD) by 2030 $4.5 Billion Compound Annual Growth Rate 7.3% Regions Covered Global MARKET OVERVIEW Influencer Market Insights Tariff Impact on Global Supply Chain Patterns Graft Versus Host Disease (GvHD) Treatment - Global Key Competitors Percentage Market Share in 2024 Competitive Market Presence - Strong/Active/Niche/Trivial for Players Worldwide in 2024 MARKET TRENDS & DRIVERS Growing Incidence of Hematopoietic Stem Cell Transplants Drives Demand for GvHD Treatments Biologics and Targeted Therapies Strengthen the Business Case for Advanced GvHD Treatment Options Rising Research Funding and Clinical Trials Activity Expand Pipeline and Market Opportunities Increased Use of Prophylactic Therapies Throws the Spotlight on Preventive GvHD Strategies CAR-T and Cell Therapy Advancements Generate New Demand for GvHD Management Solutions Personalized Medicine Trends Accelerate Adoption of Biomarker-Based GvHD Diagnostics High Mortality and Morbidity Rates Associated with Chronic GvHD Sustain Clinical Focus Immunosuppressive Drug Advancements Drive Shift from Broad to Selective GvHD Treatments Rising Adoption of Real-World Evidence in Clinical Evaluation Supports Market Validation Health Insurance Coverage Expansion for Cell Therapy Drives Access to GvHD Treatment Regulatory Harmonization Across Regions Simplifies Global Product Launches Biotech M&A Activity Throws the Spotlight on GvHD Pipeline Consolidation Telemedicine Integration in Post-Transplant Care Expands Patient Access to GvHD Monitoring For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
