Latest news with #CML
Time of India
3 days ago
- Health
- Time of India
Cancer fighter voted; why didn't you, Ludh West?
Ludhiana: A 68-year-old Veena Sood of Rishi Nagar, a former banker who is fighting chronic myeloid leukaemia (CML), a bone marrow and blood-related cancer, for the last thirty years, was among the first few voters at Polytechnic College polling booth in the company of her husband, Vinod Sood, who also retired from a bank. Veena Sood has been exercising her right to vote for 40-odd years now and both her husband and she are happy to vote. She claimed to have voted for a candidate she thinks will take care of development needs of the seat.
Yahoo
5 days ago
- Business
- Yahoo
Enliven Therapeutics, Inc. (ELVN) Backed by Goldman Sachs After Strong Trial Data
Enliven Therapeutics, Inc. (NASDAQ:ELVN) is among the best NASDAQ stocks under $50 to buy. On Monday, analysts at Goldman Sachs started coverage on Enliven Therapeutics, Inc. (NASDAQ:ELVN) with a Buy rating and a $37.00 price target, implying a rise of about 60% from the current levels. The firm's confidence in the stock stems from favorable Phase 1 data for the company's chronic myeloid leukemia (CML) solution. The investment bank noted that ELVN-001's sustained efficacy data, presented during the European Hematology Association meeting, highlighted a 47% overall cumulative major molecular response (MMR) rate by 24 weeks and a 41% response in tyrosine kinase inhibitor (TKI)-resistant patients. These results surpassed the fourth-generation Scemblix by Novartis (SIX:NOVN) figures, with a difference of 10% and 32%, respectively. A biologist in a laboratory examining a microscope for small molecule inhibitors. The company is all set to launch a Phase 3 trial in 2026, which in itself is a $3 billion opportunity. If we look at the three-year return, Enliven Therapeutics, Inc. (NASDAQ:ELVN) exhibited a return of a whopping 1,980.18% in contrast to the 64.53% return by the market. Calling this simply an 'amazing' performance would be an understatement. Enliven Therapeutics, Inc. (NASDAQ:ELVN), headquartered in Boulder, Colorado, is a clinical-stage biopharmaceutical company. The company focuses on the recognition and development of small-molecule inhibitors to assist people living with cancer, improving survival and overall well-being. While we acknowledge the potential of ELVN as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None.
Medscape
6 days ago
- Health
- Medscape
Asciminib: High Responses in Second-Line Chronic-Phase CML
Asciminib, a first-in-class BCR - ABL1 TKI, showed high molecular response rates and was well tolerated in the first prospective trial of its kind evaluating the drug as a dose-escalated second-line therapy for patients with chronic-phase chronic myeloid leukemia (CML) who had suboptimal responses to earlier therapies. METHODOLOGY: The interim analysis on the single-arm, open-label study, conducted at 85 trial sites in the US, included 101 patients with chronic-phase CML who discontinued a prior TKI due to intolerance or suboptimal responses. Patients were treated with at least one dose of asciminib 80 mg once daily. If BCR - ABL levels were above 1% at week 24, the dose was increased to 200 mg daily, and if BCR - ABL levels were above 0.1% at 48 weeks, the dose was increased from 80 to 200 mg daily, or from 200 mg daily to 200 mg twice daily, or patients could be taken off the study. - levels were above 1% at week 24, the dose was increased to 200 mg daily, and if - levels were above 0.1% at 48 weeks, the dose was increased from 80 to 200 mg daily, or from 200 mg daily to 200 mg twice daily, or patients could be taken off the study. Those with any grade 3 or 4 or persistent grade 2 toxicities that were refractory to optimal management were ineligible for dose escalations. Reasons for prior treatment discontinuation were due to a lack of efficacy in 56.4% and intolerance in 43.6% of patients. TAKEAWAY: At week 24, a major molecular response was achieved by 44.4% of patients, with 25.4% achieving a deep molecular response (MR4 or better). In all, 11.1% of patients were given dose escalations upon failing to achieve response milestones. Among 101 patients receiving at least one dose, asciminib was well tolerated by most patients. Asciminib's safety profile was that observed in previous studies, with no new or worsening safety findings observed. Grade 3 and higher adverse events (AEs) included hypertension (8.9 %), thrombocytopenia (6.9%), and neutropenia (5.9%). AEs overall led to dose adjustment or interruption in 26.7% of patients and discontinuation in four patients. IN PRACTICE: 'Asciminib, in contrast with other FDA-approved TKIs, binds to the ABL myristoyl pocket, which may reduce off-target effects compared to the other competitive TKI's,' said first author David Jacob Andorsky, MD, of the Rocky Mountain Cancer Centers, US Oncology Research, Boulder, Colorado. 'Results from ASC2ESCALATE, the first prospective trial of asciminib in second-line chronic-phase CML with dose escalation in patients not achieving response milestones, further support asciminib as a treatment option in second-line chronic-phase CML,' he said. 'The outcomes in patients with asciminib dose escalations continue to be explored, with analyses planned for future presentations,' Andorsky noted. SOURCE: The analysis was presented at the American Society of Clinical Oncology (ASCO) 2025 in Chicago. DISCLOSURES: This study was sponsored by Novartis Pharmaceuticals. Andorsky reported having relationships with AbbVie, AstraZeneca, Celgene, and Novartis.
The Hindu
15-06-2025
- Health
- The Hindu
Our body's crimson tide: the evolving treatment landscape in haematology
Historically, treatment for blood cancers, relied heavily on chemotherapy. However, this 'carpet bombing' approach, apart from destroying cancer cells also affects healthy, rapidly-dividing cells. This can lead to significant side effects like vomiting ,immunosuppression, hair loss and anaemia. While chemotherapy remains vital, the goal has always been more precise, less toxic options. A deeper understanding of cancer cell biology has paved the way for 'targeted therapy'—drugs designed as 'surgical strikes' against molecular targets predominantly found on or within cancer cells. This approach aims for greater efficacy with a more manageable side-effect profile, sparing normal cells to a larger extent. Imatinib - the dawn of targeted therapy in haematology A pivotal moment in targeted therapy arrived with Imatinib, approved in 2001 for Chronic Myeloid Leukaemia (CML). CML is driven by the Philadelphia chromosome, a genetic abnormality creating the BCR-ABL fusion gene that fuels uncontrolled white blood cell proliferation. Before Imatinib, CML was often fatal within years. Imatinib was designed to block the activity of this BCR-ABL protein. Its success was revolutionary, transforming CML from a deadly disease into a manageable chronic condition for most patients, who could now achieve long-term remission and a good quality of life with a daily pill. This validated the concept of molecularly targeted therapy and spurred massive research into similar approaches for other cancers, heralding a new frontier in cancer treatment . Harnessing the immune system --the rise of immunotherapy in haematology Our immune system, with its innate (first-line, non-specific) and adaptive (specialised, memory-forming) arms, is designed to eliminate threats, including cancerous cells. Immunotherapy aims to boost or re-engage the patient's immune system to fight cancer. Various types of antibody based therapies such as Monoclonal Antibodies (mAbs) antibodies target specific antigens on cancer cells. Bispecific Antibodies (e.g., BiTEs - Bispecific T-cell Engagers)have two binding sites-- one for a cancer cell antigen and another for a T-cell (an immune killer cell). Antibody-Drug Conjugates (ADCs) are 'armed antibodies' and combine an antibody's targeting precision with a potent cytotoxic drug . These immunotherapies offer highly effective options ,which earlier approved for resistant cancers ,are being increasingly used in frontline settings. For thousands battling life-threatening blood cancers and other devastating diseases, a Bone Marrow Transplant (BMT) offers a beacon of hope – a potential cure when other treatments have failed. This remarkable medical procedure involves replacing a patient's diseased or damaged bone marrow with healthy stem cells from the patient (autologous BMT) or a healthy donor (allogenic BMT) . Recent advances in BMT BMT is a critical treatment option for a range of blood conditions. These include benign conditions such as sickle cell anaemia, thalassemia, aplastic anaemia, inherited immune deficiency and metabolic disorders as well as malignant conditions like high risk acute leukemias, myelodysplastic syndrome, relapsed leukemias, myelomas and relapsed /refractory lymphomas. Earlier, allogenic bone marrow transplant were done only with HLA matched donors (related or unrelated) .The probability of finding a matched donor was only 30%. Now however, haploidentical (Haplo) BMT, a groundbreaking approach, allows for transplants using donors (typically family members like parents or children) who are only a half-match for the patient's HLA involves the use of PTCY - Post transplant cyclophosphamide, where chemotherapy is administered on day +3 and day +4 after infusion of the stem cells. This has dramatically increased donor availability, meaning most patients who need a transplant can now find a suitable donor. Traditionally, BMT involved high-dose chemotherapy and/or radiation (myeloablative conditioning) to wipe out the patient's marrow. Reduced Intensity Conditioning (RIC) Transplant regimens use lower, less toxic doses, making transplants an option for older patients or those with other health conditions who might not tolerate aggressive conditioning. This relies more on the graft versus leukemia effect, where the donor cells act against the cancer of the recipient . Improved Graft Manipulation: Scientists area also developing sophisticated ways to process donor stem cells before infusion such as TCR α/β depletion which removes specific T-cells (T-cell receptor alpha/beta cells) from the donor graft that are primarily responsible for causing graft versus host disease (GVHD). With ongoing research and innovation, the future promises even better outcomes, reduced side effects, and wider applicability, offering a renewed lease on life to countless individuals around the world. CAR T-cell therapy: living drugs to combat cancer Chimeric Antigen Receptor (CAR) T-cell therapy is a groundbreaking immunotherapy that engineers a patient's own T-cells into potent cancer-killing 'living drugs.' The process involves collecting a patient's T-cells, genetically modifying them in the lab to express a CAR that recognises a specific antigen on their cancer cells (e.g., CD19 on B-cell leukaemias/lymphomas), expanding these engineered cells, and then infusing them back into the patient. Once infused, CAR T-cells seek out and destroy cancer cells expressing the target antigen. This therapy has achieved unprecedented success in patients with relapsed or refractory B-cell acute lymphoblastic leukaemia and certain non-Hodgkin lymphomas, leading to high remission rates and several FDA-approved products. However, challenges include significant potential side effects like Cytokine Release Syndrome (CRS) and neurotoxicity (ICANS), along with high manufacturing complexity and cost. Gene therapy, AI and the importance of foundational clinical skills Gene therapy offers curative potential for inherited haematological disorders by modifying a patient's cells, often by introducing a functional gene or correcting a faulty one. Haematopoietic stem cells (HSCs) from bone marrow are prime targets, as modified HSCs can repopulate the marrow with corrected cells, offering a permanent solution. Significant progress has been made in diseases such as sickle cell disease, Beta-thalassemia and Hemophilia A in this regard. While transformative, challenges like long-term durability, cost, and access remain. Artificial Intelligence (AI) is emerging as a powerful tool in haematology, capable of analysing vast and complex datasets to enhance diagnosis, treatment, and research. Key applications include: AI-powered diagnostics: AI algorithms analyse blood smears and bone marrow biopsies to identify and classify cells with high accuracy, potentially assisting pathologists and improving diagnostic efficiency. AI also helps interpret complex genomic data from NGS. Accelerating drug discovery: AI can identify novel therapeutic targets, predict drug efficacy and toxicity, and repurpose existing drugs for haematological conditions, streamlining development. It can also aid in patient stratification for trials and accelerate data analysis, potentially speeding up drug approvals. AI can also integrate diverse patient data to predict individual responses to therapies, helping clinicians choose optimal treatment plans. It is poised to act as an 'AI physician assistant,' augmenting human expertise rather than replacing it, leading to more precise and personalised care. In this era of remarkable technological progress, from gene editing to AI, it's vital to remember that the foundations of good medical practice such as thorough history taking, comprehensive physical examinations and focussed investigations are paramount in arriving at the right diagnosis. The future of haematology lies in the synergistic integration of traditional medical wisdom and cutting-edge technology, ensuring holistic patient care and continued progress against blood disorders. This is the second story of the two-part series. You can read the first story here. (Dr. Steve Thomas, is a clinical haemato-oncologist and BMT physician at Sri Ramachandra Medical College, Porur, Chennai. stev07thomas@
Yahoo
13-06-2025
- Health
- Yahoo
Enliven Therapeutics Announces Updated Positive Data from Phase 1 Clinical Trial of ELVN-001 in CML at EHA 2025 Congress
Reported cumulative MMR rate of 47% (25 of 53) by 24 weeks with 32% (13 of 41) of patients achieving MMR by 24 weeks, which continues to compare favorably to precedent Phase 1 trials of approved BCR::ABL1 TKIs ELVN-001 continues to demonstrate a favorable safety and tolerability profile across all dose levels with 90 patients enrolled and a median treatment duration of ~29 weeks at cutoff Enliven will host a webcast and conference call today, June 13, at 1:30 p.m. ET BOULDER, Colo., June 13, 2025 /PRNewswire/ -- Enliven Therapeutics, Inc. (Enliven or the Company) (Nasdaq: ELVN), a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapeutics, today announced updated, positive data from the Phase 1 ENABLE clinical trial evaluating ELVN-001 in patients with chronic myeloid leukemia (CML) in an oral presentation at the European Hematology Association (EHA) 2025 Congress taking place June 12-15 in Milan, Italy, and virtually. "Thanks to the success of tyrosine kinase inhibitors (TKIs), patients with CML now have a near-normal life expectancy. As a result, treatment goals have evolved beyond response and survival to also prioritize quality of life and tolerability," said Andreas Hochhaus, Professor of Internal Medicine, Hematology and Oncology and Head of the Department of Hematology and Medical Oncology at the Jena University Hospital, Germany. "However, significant unmet needs remain, particularly related to treatment resistance and intolerance, across all lines of therapy. The data from ELVN-001 are encouraging, showing an efficacy, safety and tolerability profile that compare favorably to approved BCR::ABL1 inhibitors, despite being studied in a more heavily pretreated population. I look forward to future data, which could support ELVN-001 as a promising new option for patients who need better long-term disease management." "We are highly encouraged by the ELVN-001 data, specifically as it relates to the consistency of the cumulative and achieved MMR rates as the Phase 1 trial progresses, with more evaluable patients and longer duration of treatment," said Helen Collins, M.D., Chief Medical Officer of Enliven. "While MMR is the efficacy endpoint in CML, safety and tolerability are equally critical given the chronic nature of the disease. ELVN-001 was reported to be well tolerated across all evaluated doses and had low levels of dose reductions and discontinuations, which we believe is the key sign of a favorable safety and tolerability profile. We believe ELVN-001 has the potential to offer best-in-class efficacy and tolerability, which are key attributes for people living with CML. We look forward to sharing additional data in the future." ELVN-001 is a potent, highly selective, potentially best-in-class small molecule kinase inhibitor designed to specifically target the BCR::ABL gene fusion, the oncogenic driver for patients with CML. ELVN-001 Data Highlights Patient Demographics As of the cutoff date of April 28, 2025, 90 patients have been enrolled in the ongoing Phase 1 trial across dose levels ranging from 10 mg once a day (QD) to 80 mg twice a day (BID). The vast majority of patients (80%) remain on study with a median treatment duration of ~29 weeks. Patients enrolled were heavily pretreated: 67% of patients received three or more unique prior TKIs, including 58% of patients who received prior asciminib and 43% of patients who received prior ponatinib. 72% of patients had discontinued their prior TKI due to lack of efficacy. Encouraging ELVN-001 Efficacy Data by 24 Weeks Of the enrolled patients, 53 with typical BCR::ABL1 transcripts and without T315I mutations were evaluable for major molecular response (MMR) by 24 weeks. 25 of 53 (47%) evaluable patients were in MMR by 24 weeks, with 13 of 41 (32%) achieving and 12 of 12 (100%) maintaining MMR. Of those resistant to their last TKI, 14 of 34 (41%) were in MMR by 24 weeks. Of those previously treated with asciminib or ponatinib, 12 of 34 (35%) were in MMR by 24 weeks. All patients who achieved or maintained MMR were still in MMR at the time of data cutoff. These data continued to compare favorably to precedent Phase 1 MMRs for approved BCR::ABL1 TKIs, particularly given the more heavily pretreated patient population in the ELVN-001 clinical trial. Specifically, the achieved MMR rate by 24 weeks of 32% compares favorably with historical data from less heavily pretreated patients receiving asciminib, which showed achieved MMR rates of 24% in the Phase 1 trial and 25% in the ASCEMBL Phase 3 trial. ELVN-001's Safety Profile Consistent with High Selectivity for ABL1 ELVN-001 remains well-tolerated across all evaluated doses. Only 3.4% (3 of 87) of patients had dose reductions due to treatment-emergent adverse events (TEAEs) and 4.6% (4 of 87) of patients discontinued due to TEAEs. The majority of TEAEs were low frequency and low grade, and the hematologic TEAE profile was similar to or better than the approved TKIs. Only 2.3% (2 of 87) of patients experienced ≥ Grade 3 non-hematologic treatment-related AEs. No evidence to date of enhanced cardiovascular toxicity and no treatment-related arterial occlusive events (AOEs). The maximum tolerated dose was not reached, and no exposure-toxicity relationship was observed. ELVN-001 Pharmacokinetic (PK) Profile The PK profile supports once-daily dosing with flexible administration requirements, including the ability to take with or without food. There is low potential for drug-drug interactions, an important advantage given that the average CML patient takes approximately five concurrent medications. "We believe there remains significant opportunity to improve upon existing therapies," said Sam Kintz, Co-founder and Chief Executive Officer of Enliven. "Based on today's encouraging Phase 1 update, we believe ELVN-001 has the potential to compete across all lines of therapy. We believe that precedent registrational trials in CML provide a roadmap for the regulatory pathway for ELVN-001, and the use of biomarker-based endpoints, like MMR, enables smaller, faster studies. Importantly, historical Phase 1 data in late-line CML trials have predicted success in subsequent pivotal trials. Building off this exciting update, we expect to initiate our first head-to-head Phase 3 pivotal trial in 2026 and remain confident in ELVN-001 and its potential positioning in the future in the CML treatment paradigm." The oral presentation titled: "ENABLE: A Phase 1a/1b Study of ELVN-001, a selective active site inhibitor of BCR::ABL1, in patients with previously treated CML" will be presented by Andreas Hochhaus, Professor of Internal Medicine, Hematology and Oncology and Head of the Department of Hematology and Medical Oncology at the Jena University Hospital, Germany later today. A copy of the presentation will be available on the "Program Presentations & Publications" section of the Company's website at Webcast and Conference Call InformationEnliven will host a live webcast and conference call today at 1:30 p.m. ET / 7:30 p.m. CEST. To participate in the live event, please register using this link. Following registration, participants will have access to dial in numbers and a unique passcode should they prefer to participate by phone. The event and accompanying slides can also be accessed by visiting the investor relations section of the Company's website at An archived webcast will be available on the Company's website following the event. About the ENABLE TrialThe ENABLE study (NCT05304377) is a Phase 1 study of ELVN-001 in patients with previously treated CML. The trial is currently in Phase 1a/1b development and is a dose escalation and expansion trial designed to evaluate safety and tolerability and to determine the recommended dose for further clinical evaluation of ELVN-001 in patients with CML with and without T315I mutations that is relapsed, refractory or intolerant to TKIs. Secondary endpoints include pharmacokinetics, MMR by central quantitative reverse transcriptase polymerase chain reaction, duration of MMR, BCR::ABL1 transcript levels and complete hematologic response. Enliven is preparing for the potential start of a pivotal trial for ELVN-001 in 2026. About ELVN-001ELVN-001 is a potent, highly selective, potentially best-in-class small molecule kinase inhibitor designed to specifically target the BCR::ABL gene fusion, the oncogenic driver for patients with chronic myeloid leukemia. As a highly selective active site inhibitor, ELVN-001 has a mechanism of action that is complementary to allosteric BCR::ABL1 inhibitors, which may play an increasingly important role in the standard of care. ELVN-001 was also designed to have activity against the T315I mutation, the most common BCR::ABL1 mutation, which confers resistance to nearly all approved TKIs, as well as activity against mutations known to confer resistance to allosteric BCR::ABL1 inhibitors. About Enliven Therapeutics Enliven is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapeutics to help people not only live longer, but live better. Enliven aims to address existing and emerging unmet needs with a precision oncology approach that improves survival and enhances overall well-being. Enliven's discovery process combines deep insights in clinically validated biological targets and differentiated chemistry to design potentially first-in-class or best-in-class therapies. Enliven is based in Boulder, Colorado. Forward-Looking StatementsThis press release contains forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended) concerning Enliven and other matters that involve substantial risks and uncertainties. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations and financial condition, or otherwise, based on current beliefs of the management of Enliven, as well as assumptions made by, and information currently available to, management of Enliven. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements in this press release include, but are not limited to, statements regarding the potential of, and plans regarding, market opportunities, and expectations regarding Enliven's programs, including ELVN-001; expected milestones for ELVN-001, including the potential timing for a start of a pivotal trial for ELVN-001; and statements by Enliven's Chief Executive Officer, Chief Medical Officer and the Professor of Internal Medicine, Hematology and Oncology and Head of the Department of Hematology and Medical Oncology at the Jena University Hospital, Germany. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various risks and uncertainties, including, without limitation: the limited operating history of Enliven; the ability to advance product candidates through preclinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize or license, or identify and complete strategic alternatives for, product candidates; the outcome of preclinical testing and early clinical trials for product candidates and the potential that the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials, including extrapolations or predictions regarding the safety and efficacy of ELVN-001 based on comparisons to published results of trials of other products, which may be different when evaluated in head-to-head studies; Enliven's limited resources; the risk of failing to demonstrate safety and efficacy of product candidates; Enliven's limited experience as a company in designing and conducting clinical trials; the potential for interim, topline, and preliminary data from Enliven's preclinical studies and clinical trials to materially change from the final data; potential delays or difficulties in the enrollment or maintenance of patients in clinical trials; developments relating to Enliven's competitors and its industry, including competing product candidates and therapies; the potential market opportunity for any of Enliven's programs; the decision to develop or seek strategic collaborations to develop Enliven's current or future product candidates in combination with other therapies and the cost of combination therapies; the ability to attract, hire, and retain highly skilled executive officers and employees; the ability of Enliven to protect its intellectual property and proprietary technologies; the scope of any patent protection Enliven obtains or the loss of any of Enliven's patent protection; reliance on third parties, including medical institutions, contract manufacturing organizations, contract research organizations and strategic partners; geo-political developments, general market or macroeconomic conditions; Enliven's ability to obtain additional capital to fund Enliven's general corporate activities and to fund Enliven's research and development; and other risks and uncertainties, including those more fully described in Enliven's filings with the Securities and Exchange Commission (SEC), which may be found in the section titled "Risk Factors" in Enliven's Annual and Quarterly Reports on Form 10-K and 10-Q filed with the SEC and in Enliven's future reports to be filed with the SEC. Except as required by applicable law, Enliven undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. Head-to-Head Comparisons The Company has not performed any head-to-head trials for ELVN-001. As a result, the data referenced in this press release is derived from different clinical trials at different points in time, with differences in trial design and patient populations. As a result, conclusions from cross-trial comparisons cannot be made. View original content to download multimedia: SOURCE Enliven Therapeutics, Inc. Sign in to access your portfolio
