Uproleselan

Checkpoint inhibitors transformed cancer treatment, bringing to patients immunotherapies now widely used to treat a range of tumors. But there are different types of checkpoints that interact with the immune system, and the scope of this drug research is expanding. Valora Therapeutics focuses on sugar molecules found all over cells and a particular mechanism that regulates them. The preclinical startup emerged from stealth on Wednesday with $30 million to advance its research. One way that cells communicate with the immune system is through glycans, sugar molecules found on the surfaces of all cells. The presence of these molecules are a key way that the body distinguishes foreign cells from cells that belong to the body. Communication happens as glycans displayed on one cell are recognized by lectins, proteins on other cells that bind to glycans. But in some diseases, this communication is dysregulated. In cancer, that can mean tumors send signals that suppress the immune system. In autoimmune disease, the signaling can lead to excessive immune cell activity. San Diego-based Valora targets the glyco-immune checkpoint, a regulatory pathway in which glycans interact with lectins, said Miguel Garcia-Guzman, the startup’s co-founder and CEO. Valora’s drugs are engineered antibodies. The antibody component provides the targeting ability for the therapy, just like currently available therapeutic antibodies. But Valora brings additional functionality with a domain that directly binds to glycans, blocking their interaction with lectins. Valora calls these drugs antibody-lectin chimeras, or AbLecs. Consumer / Employer Health Executives on Digital Transformation in Healthcare Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare. By MedCity News “In the case of cancer, we try to reduce the glyco-immune checkpoint so the immune system becomes active again,” Garcia-Guzman said. “In the case of autoimmune disease, we do the contrary.” Valora’s science is based on the research of Carolyn Bertozzi, the Stanford University professor credited with inventing bioorthogonal chemistry, chemical reactions that happen in living systems without interfering with native biochemical processes. Applications of this chemistry include drug targeting, and Bertozzi’s work in this field was recognized with the 2022 Nobel Prize in Chemistry. Garcia-Guzman’s experience includes posts at Vertex Pharmaceuticals and Rakuten Medical, a cancer drug developer that he founded. He learned about AbLecs from a pre-print that was published in 2022, and he reached out to Bertozzi to discuss forming a company around the science. Valora was founded in 2023 with a license to Bertozzi’s Stanford research. Bertozzi is a scientific co-founder of the startup. The other scientific co-founder is Jessica Stark, an MIT professor of biological engineering and chemical engineering whose postdoctoral research in Bertozzi’s lab focused on identifying and targeting glycans that act as immune checkpoints for cancer. Garcia-Guzman said the importance of the glyco-immune checkpoint in cancer and autoimmune disease has gained recognition in the last decade. But there are many glycans, and it’s difficult to modulate the ones that are unknown. He added that he was drawn to the AbLec technology because this approach is both simple and powerful in the way that it addresses the pathological signaling that you want to modulate. Health IT Reducing Clinical and Staff Burnout with AI Automation As technology advances, AI-powered tools will increasingly reduce the administrative burdens on healthcare providers. By Dr. Michael Blackman, Chief Medical Officer at Greenway Health There are other companies pursuing glycobiology drug research. GlycoMimetics was developing small molecule drugs that block certain lectins. Lead program uproleselan, an experimental treatment for acute myeloid leukemia, hit a setback over the summer when the FDA said it needed another study. That program’s future is now uncertain as GlycoMimetics is serving as a vehicle for Crescent Biopharma to go public via a reverse merger. Meanwhile, Palleon Pharmaceuticals is another glycobiology company based on Bertozzi’s research. The Waltham, Massachusetts-based biotech is developing drugs that edit glycans as a way to treat cancers and autoimmune disease. Palleon recently reported Phase 1/2 data demonstrating proof of mechanism in cancer. Garcia-Guzman said Valora’s new funding will enable the startup to develop in phases. The first phase is platform exploration and optimization with a broad range of AbLec molecules that use different antibodies and lectins. This work will be applied to both cancer and autoimmune disease. The next phase will be prioritizing the mechanisms most promising for therapeutic development. After that, the company will work to toward the preclinical research that could support an investigational new drug application. Garcia-Guzman said Valora is not currently looking for partnerships with large pharmaceutical companies, but the broad potential applications of the company’s technology may lead to such alliances in the future. Valora’s $30 million seed round was co-led by Avalon BioVentures, Bregua Corporation, and TigerGene. Alexandria Ventures and Correlation Ventures also participated. The startup is based at Avalon’s accelerator in San Diego.

ROCKVILLE, Md.--( BUSINESS WIRE )--GlycoMimetics, Inc. (Nasdaq: GLYC) today announced that data from multiple clinical trials studying uproleselan in patients with acute myeloid leukemia (AML) have been accepted for presentation at the 66 th American Society of Hematology (ASH) Annual Meeting and Exposition taking place in San Diego, California, on December 7-10, 2024. ASH Annual Meeting abstracts may be accessed online at www.hematology.org . Details of GlycoMimetics presentations are as follows (all times PT): Publication Number: 733 Type: Oral Title: Efficacy and Safety of Uproleselan Combined with Chemotherapy Vs. Chemotherapy Alone in Relapsed/Refractory Acute Myeloid Leukemia: Findings from an International Phase 3 Trial Presenter: Daniel DeAngelo, M.D., Ph.D. Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: New Treatment Approaches for AML Session Date/ Presentation Time: Monday, December 9, 2024, 10:30 AM – 12:00 PM Publication Number: 1503 Type: Poster Title: Updated Results of a Phase I Study of Uproleselan Combined with Azacitidine and Venetoclax for the Treatment of Older or Unfit Patients with Treatment Naive Acute Myeloid Leukemia Presenter: Brian Jonas, M.D., Ph.D. Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster I Session Date/ Presentation Time: Saturday, December 7, 2024, 5:30 PM - 7:30 PM Publication Number: 4262 Type: Poster Title: Final Results of a Phase II Study of Uproleselan Combined with Cladribine and Low-Dose Cytarabine for Patients with Treated Secondary Acute Myeloid Leukemia (ts-AML) Presenter: Caitlin Rausch, PharmD. Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster III Session Date/Presentation Time: Monday, December 9, 2024, 6:00 – 8:00 PM About GlycoMimetics, Inc. GlycoMimetics is a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers, including AML, and for inflammatory diseases. The company’s scientific approach is based on an understanding of the role that carbohydrates play in cell recognition. Its specialized chemistry platform can be used to discover small molecule drugs, known as glycomimetics, that alter carbohydrate-mediated recognition in diverse disease states, including cancers and inflammation. The company’s goal is to develop transformative therapies for diseases with high unmet medical need. GlycoMimetics is headquartered in Rockville, MD in the BioHealth Capital Region. Learn more at www.glycomimetics.com . Forward-Looking Statements To the extent that statements contained herein are not descriptions of historical facts, they are forward-looking statements reflecting the current beliefs and expectations of the management of GlycoMimetics, Inc. (“GlycoMimetics,” “we,” “us,” or “our”). Forward-looking statements include, but are not limited to, the expected presentation of clinical data for uproleselan, and any other statement containing terminology such as “may,” “will,” “should,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “intends,” or “continue,” or the negative of these terms or other comparable terminology. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels of activity, performance, or achievements to be materially different from those discussed, implied or otherwise anticipated by such statements. You are cautioned not to place undue reliance on such forward-looking statements, which are current only as of the date of this presentation. For a further description of the risks associated with forward-looking statements, as well as other risks facing GlycoMimetics, please see the risk factors described in the Company’s Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission on March 27, 2024 and the Company’s Quarterly Reports on Form 10-Q filed with the SEC on May 9, 2024 and August 8, 2024, as well as other reports we file with the U.S. Securities and Exchange Commission from time to time, including those factors discussed under the caption “Risk Factors” in such filings. Forward-looking statements speak only as of the date of this presentation, and GlycoMimetics undertakes no obligation to update or revise these statements, except as may be required by law.