Latest news with #IND-enabling
Business Upturn
14-05-2025
- Business
- Business Upturn
Ernexa Therapeutics Establishes Texas Subsidiary to Support Continued Development of ERNA-101 and Future Clinical Operations
CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) — Ernexa Therapeutics (Nasdaq: ERNA), developing innovative cell therapies for the treatment of advanced cancer and autoimmune disease, today announced the formation of ErnexaTX2, a wholly owned subsidiary based in Texas. The new entity has been established to support ongoing preclinical development of the company's lead program, ERNA-101 for ovarian cancer, and to lay the operational groundwork for anticipated clinical activity in 2026. 'As we continue to progress ERNA-101 through key preclinical milestones, we're also setting the stage for a seamless transition into clinical work,' said Sanjeev Luther, President and CEO of Ernexa Therapeutics. 'The formation of ErnexaTX2 reflects both our long-term commitment to the Texas ecosystem and our strategic readiness to support clinical manufacturing, regulatory interactions, and site activation in the years ahead.' The subsidiary will support ongoing research being conducted under a sponsored research agreement with Michael Andreeff, M.D., Ph.D., from The University of Texas MD Anderson Cancer Center . Dr. Andreeff is an internationally recognized cell therapy expert and a longtime leader in targeting the tumor microenvironment in ovarian and other solid tumors. In parallel with scientific advancements, Ernexa continues to evolve its infrastructure in anticipation of future Investigational New Drug (IND)-enabling activities. With its new footprint in Texas, the company is positioned to accelerate clinical partnerships, navigate regulatory planning, and engage local biomanufacturing partners to support scale-up efforts. ERNA-101 remains on track for IND-enabling studies in 2025, with first-in-human studies targeted for 2026. About Ernexa Therapeutics Ernexa Therapeutics (NASDAQ: ERNA) is developing innovative stem cell therapies for the treatment of advanced cancer and autoimmune disease. Ernexa's core technology focuses on engineering induced pluripotent stem cells (iPSCs) and transforming them into induced mesenchymal stem cells (iMSCs). Ernexa's allogeneic synthetic iMSCs provide a scalable, off-the-shelf treatment, without needing patient-specific cell harvesting. ERNA-101 is the company's lead cell therapy product, designed to activate and regulate the immune system's response to recognize and attack cancer cells. ERNA-102 is a cell therapy product designed to target inflammation and treat autoimmune disease. The company's initial focus is to develop ERNA-101 for the treatment of ovarian cancer. For more information, visit . Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are intended to be covered by the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements, in some cases, can be identified by terms such as 'believe,' 'may,' 'will,' 'estimate,' 'continue,' 'anticipate,' 'design,' 'intend,' 'expect,' 'could,' 'plan,' 'potential,' 'predict,' 'seek,' 'should,' 'would,' 'contemplate,' 'project,' 'target,' 'objective,' or the negative version of these words and similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Ernexa's actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including, without limitation, risks and uncertainties related to: progress and possible outcomes of the Company's lead research project, ERNA-101, and future research projects. Forward-looking statements are based upon Ernexa's current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. For a detailed description of Ernexa's risks and uncertainties, you are encouraged to review its documents filed with the SEC including its recent filings on Form 8-K, Form 10-K and Form 10-Q. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Ernexa does not undertake any obligation to update the forward-looking statements contained herein to reflect events that occur or circumstances that exist after the date hereof, except as required by applicable law. Media & Investor Relations Contact [email protected] Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same.
Yahoo
13-05-2025
- Business
- Yahoo
Sharp Therapeutics Corp. Nominates Clinical Candidate for Gaucher's Disease
Pittsburgh, Pennsylvania and Toronto, Ontario--(Newsfile Corp. - May 13, 2025) - Sharp Therapeutics Corp. (TSXV: SHRX) ("Sharp" or the "Company"), a pre-clinical-stage biotechnology company developing small molecule therapies to treat genetic diseases, announces that it has nominated a small molecule compound from its GBA program for clinical development in Gaucher's disease. Sharp has launched its clinical development program for Gaucher's disease by nominating a compound from the SEL-148,721 series of GBA1-restoring small molecules to enter IND-enabling studies. The company plans to begin compound scale-up and formal safety studies during the second half of 2025. If successful, the Company expects to file an IND application with the Food and Drug Administration and enter Phase I clinical trials in 2026. About the SHRX Gaucher's Program. Sharp discovered the SEL-148,172 series by applying its Disco™ discovery platform to identify compounds that enhance mutant GBA functional activity. Gaucher's disease is caused by mutation(s) in the GBA enzyme that reduce enzymatic function leading to disease. The candidate compound restores enzymatic activity, which has been shown to be an effective means of treating Gaucher's. Current Gaucher's treatments include recombinant replacement enzyme therapies, which require regular infusions with some patients developing allergic resistance to therapy. Sharp's candidate compound is an orally-available small molecule making it much more convenient for patients, and much more efficient to produce and distribute reliably. "This is the first program from our platform to enter FDA-reportable studies and is a milestone for Sharp as it transitions to a clinical stage company," said Scott Sneddon, CEO/CSO of Sharp. "The compounds show robust activity in animal models, and more importantly, in cells taken from Gaucher's patients containing several of the most common GBA mutations," he added. "These compounds are also brain penetrant, leaving the prospect for treating CNS manifestations of Gaucher's, a market not effectively treated by existing therapies." The scientific data supporting the compounds will be presented at the GBA1 Conference in Montreal starting June 5, 2025 of which Sharp is also a meeting sponsor. About Sharp Therapeutics Corp. - First-Choice Therapies for Genetic Diseases Sharp Therapeutics is a pre-clinical stage company developing first-choice small-molecule therapeutics for hereditary disorders. The Company's discovery platform combines novel high throughput screening technologies, with compound libraries computational optimized based on the physics and biology of cellular trafficking defects and allosteric activation of proteins. The platform produces small molecule compounds that restore activity in mutated proteins giving the potential to treat genetic disorders with conventional pill-based medicines. For additional information on Sharp, please visit: Sharp Therapeutics Sneddon, PhD, JDCEO/CSOEmail: scott@ Caution Regarding Forward-Looking Information Certain statements contained in this press release constitute "forward-looking information" as such term is defined in applicable Canadian securities legislation. The words "may", "would", "could", "should", "potential", "will", "seek", "intend", "plan", "anticipate", "believe", "estimate", "expect" and similar expressions are intended to identify forward-looking information. All statements other than statements of historical fact may be forward-looking information. Such statements reflect Sharp's current views and intentions with respect to future events, and current information available to Sharp, and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements that may be expressed or implied by such forward-looking information to vary from those described herein should one or more of these risks or uncertainties materialize. Should any factor affect Sharp in an unexpected manner, or should assumptions underlying the forward-looking information prove incorrect, the actual results or events may differ materially from the results or events predicted. Any such forward-looking information is expressly qualified in its entirety by this cautionary statement. Moreover, Sharp does not assume responsibility for the accuracy or completeness of such forward-looking information. The forward-looking information included in this press release is made as of the date of this press release and Sharp undertakes no obligation to publicly update or revise any forward-looking information, other than as required by applicable law. Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release. To view the source version of this press release, please visit Sign in to access your portfolio
Yahoo
04-03-2025
- Business
- Yahoo
Laekna Announces IND Approval of LAE120 (a Novel USP1 Inhibitor) for Treatment of Advanced Solid Tumors by FDA
A novel, allosteric and highly potent USP1 inhibitor, LAE120 exhibits robust tumor inhibitory activity across various xenograft models Significantly accelerated the progress of drug discovery leveraging cutting-edge AI models Another potential anti-tumor drug candidate, LAE118 (a mutant-selective PI3Kα inhibitor) also in IND-enabling studies LOS ANGELES, March 04, 2025--(BUSINESS WIRE)--Laekna ( announced that the U.S. Food and Drug Administration (FDA) has approved the IND for LAE120, an internally discovered USP1 inhibitor, for the treatment of advanced solid tumors. LAE120 is a novel, allosteric and highly potent USP1 inhibitor, displaying monotherapy potency and combination activity with PARP inhibitor in HRD (homologous recombination deficiency) cancers. It has a unique chemical structure differentiated from all the other disclosed USP1 inhibitors and is expected to induce a different conformational change in USP1. LAE120 shows robust tumor inhibitory activity across various xenograft models such as MDA-MB-436 and K562 as a single agent and exhibits synergistic effect in combination with PARP inhibitors. It also demonstrates good therapeutic windows in GLP long-term toxicology study. Laekna is actively exploring partnerships to accelerate the clinical development of LAE120. "Leveraging our deep know-how and extensive expertise in drug discovery, Laekna has developed a distinctive portfolio of innovative drug candidates through the close collaboration of our Med Chem, Biology and AIDD (AI-driven Drug Discovery) teams, continuously advancing preclinical drug candidates into clinical stage. Laekna has also significantly accelerated the progress of drug discovery by utilizing cutting-edge artificial intelligence tools," said Dr. Justin Gu, Chief Scientific Officer of Laekna. "We look forward to bringing novel drugs to patients as swiftly as possible," he added. Advancing Diversified Pipelines Laekna is actively advancing preclinical drug candidates. In the fourth quarter of 2024, another internally discovered anti-tumor drug candidate, LAE118, a potentially best-in-class, mutant-selective PI3Kαinhibitor, has advanced to IND-enabling study. PI3Kα mutations are prevalent in patients with breast, colorectal, lung, endometrial, and numerous other cancers. However, the first-generation drugs targeting PI3Kα inhibit the wild-type and mutant PI3Kα with equal potency, which raises concerns of tolerability and therapeutic efficacy. As a novel allosteric inhibitor, LAE118 demonstrates excellent potency and selectivity towards various PI3Kα mutants. With superior anti-cancer efficacy and tolerability than other current PI3Kα inhibitors, LAE118 is potentially the best-in-class pan-mutant-selective PI3Kα inhibitor. Laekna has presented the preclinical characterization of LAE118 at the San Antonio Breast Cancer Symposium (SABCS) in December 2024. LAE118 is in IND-enabling studies and IND is expected to be filed in the fourth quarter of 2025. Strategic Partnerships to Accelerate Globalization "We will continue to advance and expand our product portfolio in the therapeutic areas where we have accumulated tremendous experience and extensive know-how," said Dr. Chris Lu, Chief Executive Officer of Laekna. "In November 2024, the Group has entered into a clinical collaboration agreement with Lilly (NYSE:LLY) to support and accelerate global clinical development of LAE102 for the treatment of obesity. We plan to pursue strategic partnerships with global leading pharmaceutical companies to accelerate clinical development and commercialization of our drug candidate assets. We keep advancing and expanding our pipeline and are committed to bringing life-changing medicines to more people around the world," he added. About Laekna Stock Code: Founded in 2016, Laekna is a science-driven, clinical-stage biotechnology company committed to bringing novel therapeutics to patients with cancer, metabolic diseases and liver fibrosis patients around the world. As of June 30, 2024, Laekna has initiated seven clinical trials for LAE102, LAE002(afuresertib), LAE001 and LAE005 to address unmet medical needs in obesity and cancers. LAE102 is our internally discovered antibody against ActRIIA. It has been shown in the pre-clinical studies to increase lean mass and decrease fat mass. We've obtained IND approvals from the FDA and the CDE for LAE102 in obesity indication and are advancing the Phase I clinical trial in China. Blocking Activin-ActRII pathway could promote muscle regeneration and decrease fat mass. Laekna team has accumulated tremendous experiences and deep know-how in this specific field and is developing more drug candidates (LAE103 and LAE123), in addition to LAE102, to maximize the value of targeting ActRII receptors. In the cancer area, Laekna has built a comprehensive portfolio of drug candidates, covering the treatment of breast cancer, prostate cancer, ovarian cancer and PD-1/ PD-L1 drug-resistant solid tumors. LAE002 (afuresertib) is a potent AKT inhibitor that inhibits all three AKT isoforms (AKT1, AKT2 and AKT3) as well as one of the only two AKT inhibitors in late-stage development for breast and prostate cancer globally. Laekna has commenced the Phase III clinical trial (AFFIRM-205) for LAE002 in patients with HR+/HER2- breast cancer. Laekna, Inc. ( was listed on the Main Board of The Stock Exchange of Hong Kong Limited (the "Hong Kong Stock Exchange") on June 29, 2023. For more information, please visit: or Forward-Looking Statements This press release may contain certain "forward-looking statements" which are not historical facts, but instead are predictions about future events based on Laekna's current beliefs, assumptions and expectations, commonly identified by words such as "would", "may", "expects", "believes", "plans", "intends", "projects" and other terms with similar meaning. Although we believe that our predictions are reasonable, future events are inherently uncertain and our actual future results or performance may be materially different from what we expect. Accordingly, you are strongly cautioned that reliance on any forward-looking statements is subject to significant known and unknown risks and uncertainties. All forward-looking statements contained herein are qualified by reference to the cautionary statements set forth in this section. All information provided in this press release is as of the date of this press release and are based on assumptions that we believe to be reasonable as of this date, and we do not undertake any obligation to update any forward-looking statement, except as required under applicable law. View source version on Contacts IR ir@ Media communication@ Corporate and Business Development BD@ Sign in to access your portfolio
