CCR2

The FDA is yet to approve a therapy specifically for FSGS. \n Orphan drug specialist Amicus Therapeutics is paying Dimerix $30 million cash for U.S. rights to the phase 3 prospect DMX-200, establishing it as a challenger to Travere Therapeutics in a rare kidney disease. New Jersey-based Amicus’ hunt for de-risked assets that could address a significant unmet medical need led it to Australia. Melbourne-based Dimerix began a phase 3 study of the CCR2 inhibitor DMX-200 in focal segmental glomerulosclerosis (FSGS) kidney disease in 2022. The Australian biotech recently aligned with the FDA on the use of proteinuria as an appropriate primary endpoint for traditional approval. Dimerix reported interim results on the effect of DMX-200 on proteinuria last year. With enrollment well underway and another interim analysis coming up, data that could support filings for FDA approval is in sight.As the program advances, Amicus could pay Dimerix up to $75 million in development and regulatory milestones. A further $35 million is tied to the first sale of the drug. Beyond that, Amicus has committed up to $410 million in sales milestones and $40 million tied to potential future indications.   The FDA is yet to approve a therapy specifically for FSGS. That could change soon. Travere filed for FDA approval of Filspari in the indication this March. Amicus’ willingness to bet on DMX-200 despite the potential of a rival that is near approval reflects a belief that there is room for multiple approaches in FSGS.Jeffrey Castelli, chief development officer at Amicus, said on an earnings call Thursday that DMX-200 “is specifically targeting the inflammatory signaling in the damaged kidney.” Other molecules, in contrast, are focused on hemodynamics. Some patients will respond better to one drug than the other, Castelli said, and physicians will try to figure out which is the right approach for each individual.  “With DMX-200, there are ways you can look at how much the inflammatory component is driving things. Those are going to be the patients we\'re going to see respond best in the trials, and those are going to be the patients that I think doctors will want to focus on,” Castelli said. ChemoCentryx, now part of Amgen, was developing a CCR2 inhibitor with Vifor Fresenius Medical Care Renal Pharma until a phase 2 flop scuttled the FSGS program in 2020. Castelli discussed the differences between DMX-200 and earlier candidates, contrasting attempts to directly inhibit MCP-1 binding to the downstream approach of Dimerix’s molecule. “What happened with traditional binding inhibitors is MCP-1 couldn\'t dock. It actually led to a rebound effect where MCP-1 levels dramatically increase, which then would compete with the inhibitor and kind of lead to a muting effect,” Castelli said. “The beauty of the downstream blocking is that MCP-1 can still dock. That leads to MCP-1 degradation.”Amicus believes the mechanism of DMX-200 will “really maximize the ability to shut down the monocyte inflammation,” Castelli said. The downstream effects will also allow “monocytes to continue their job elsewhere ... so they can still fight infections as they should outside of the kidney.”

Funding to support clinical evaluation of STX-0712, drive additional pipeline programs, and target new applications of company’s proprietary CyTAC™ and TicTAC™ platforms. Phase 1 trial of STX-0712 to assess safety and preliminary antitumor activity of novel CCR2-CyTAC therapy in patients with CMML. BOSTON, MA, USA I April 9, 2025 I Solu Therapeutics, a biotechnology company pioneering therapies to eliminate disease-driving cells in cancer, immunology, and other therapeutic areas, today announced the successful completion of a $41 million Series A financing that included participation from five new investors – Eli Lilly and Company, Biovision Ventures, Pappas Capital, Hengdian Group Capital (HgC), and The Leukemia & Lymphoma Society Therapy Acceleration Program® – as well as continued support from existing Solu investors Longwood Fund, DCVC Bio, Santé Ventures, Astellas Venture Management, and Alexandria Venture Investments. The company also announced initiation of treatment of the first patient in its first-in-human Phase 1 clinical trial evaluating STX-0712 in patients with resistant/refractory chronic myelomonocytic leukemia (CMML) and other hematologic malignancies. “In the short period since our initial seed funding, Solu Therapeutics has rapidly advanced to a clinical-stage company targeting areas of high unmet medical need for patients,” said Philip J. Vickers, President and CEO at Solu Therapeutics. “With this Series A round we are grateful for the continued support from our existing investors and are excited to welcome several new investors who recognize the potential of our novel CyTAC (Cytotoxicity Targeting Chimera) and TicTAC (Therapeutic Index Control Targeting Chimera) platforms. These technologies have demonstrated an unprecedented ability to unlock high-value cell surface targets that are beyond the reach of traditional antibodies, making it possible to eliminate disease-driving cells with greater precision and efficacy.” Proceeds from the Series A financing will be used to complete dose escalation and expansion of the company’s lead CCR2-CyTAC program, STX-0712, for the treatment of CMML. Additionally, the funding will support the generation of new development candidates, including a novel, first-in-class mast cell depletor for immunological diseases, initiation of new discovery programs targeting pathogenic cells, further pipeline expansion, and exploration of new applications for the CyTAC and TicTAC platforms. STX-0712 is designed to selectively eliminate CCR2-positive malignant monocytes in patients with advanced hematologic malignancies, with an initial focus on CMML. The Phase 1 trial of STX-0712 is an open-label, multicenter study designed in two parts. Part A will focus on dose escalation to determine the maximum tolerated dose and/or minimum effective dose, enrolling participants with resistant/refractory CMML. Part B will further evaluate safety, tolerability, recommended Phase 2 dose and preliminary antitumor activity of STX-0712. “Our team is thrilled to initiate this first-in-human clinical trial of STX-0712, marking a significant step forward in our mission to develop innovative therapies for patients with high unmet medical needs,” said Sergio Santillana MD, Chief Medical Officer. “By directly depleting the CCR2-positive malignant monocytes driving CMML, STX-0712 has the potential to offer a highly specific and targeted therapy for patients who currently have limited treatment options available. This trial represents an important milestone for Solu Therapeutics as we work to bring novel and potentially more effective targeted therapies to patients with CMML and other hematologic malignancies.” In December 2024, Solu Therapeutics presented preclinical data at the 2024 American Society of Hematology (ASH) Annual Meeting showing robust ex-vivo activity of STX-0712 against CCR2-positive monocytes in CMML patient samples. CMML is characterized by elevated monocyte counts and dysplastic bone marrow features with limited treatment options. About STX-0712 STX-0712 is a CyTAC designed to target the G-Protein Coupled Receptor CCR2, a selective marker expressed at high levels on malignant monocytes that are key drivers of disease in CMML and other hematologic malignancies. By eliminating CCR2-positive cells, STX-0712 has the potential to offer a more targeted and effective treatment option with minimal effects on non-malignant cells. About Solu Therapeutics Solu Therapeutics is a biotechnology company cofounded by Longwood Fund and committed to developing an innovative class of therapeutic agents that uniquely pair small molecules with monoclonal antibodies to eliminate disease-driving cells in cancer, immunology, and other therapeutic areas. By leveraging its proprietary CyTAC (Cytotoxicity Targeting Chimera) and TicTAC (Therapeutic Index Control Targeting Chimera) platforms, Solu is advancing therapies that combine the target-binding capability of small molecules with the therapeutic power of biologics. Our lead product candidate, STX-0712, is currently in Phase 1 clinical development for the treatment of advanced hematologic malignancies including CMML. For more information, visit www.solutherapeutics.com . SOURCE: Solu Therapeutics

STX-0712 showed robust activity against CCR2-positive monocytes in CMML and AML patient samples BOSTON, mA, USA I December 9, 2024 I Solu Therapeutics, a biotechnology company pioneering therapies to eliminate disease-driving cells in cancer, immunology, and other therapeutic areas, today announced positive preclinical data on STX-0712, its novel CCR2-CyTAC ™ (Cytotoxicity Targeting Chimera), for the treatment of chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML). Results were presented over the weekend in two poster sessions at the American Society of Hematology (ASH) Annual Meeting, held December 7-10, 2024, in San Diego, California. “The encouraging results from our preclinical studies in CMML and AML highlight the unique power of our CyTAC platform to precisely target and eliminate disease-driving cells,” said Sergio Santillana, Chief Medical Officer of Solu. “STX-0712’s ability to selectively target CCR2-positive malignant monocytes in these two challenging blood cancers showcases the platform’s potential to support the development of transformative treatments for different types of cancer and other diseases. We look forward to advancing STX-0712 into clinical trials and continuing to leverage our platform for other high-need diseases.” Preclinical Results for STX-0712 in CMML Key Findings : To access the full abstract, click here . Preclinical Results for STX-0712 in AML Key Findings : To access the full abstract, click here . “The results from these studies represent a significant milestone for Solu, as they reinforce the potential of STX-0712 in the treatment CMML and AML, two diseases with limited targeted therapies available,” said Brandon Turunen, Co-founder, Chief Technology Officer, and Head of Drug Discovery at Solu. “By targeting the underlying drivers of these diseases, STX-0712 could offer a highly targeted approach to treatment while maintaining a strong safety profile. We look forward to advancing this promising program to clinical-stage research in 2025.” About STX-0712 STX-0712 is a CyTAC targeting the G-Protein Coupled Receptor (GPCR) CCR2, a selective marker expressed at high levels on malignant monocytes that are key drivers in certain hematologic cancers. By targeting CCR2, STX-0712 is designed to selectively eliminate these malignant cells. About Solu Therapeutics Solu Therapeutics is a biotechnology company dedicated to developing next-generation therapeutics to eliminate disease-driving cells in cancer, immunology and other therapeutic areas. The company’s proprietary CyTAC (Cytotoxicity Targeting Chimera) and TicTAC™ (Therapeutic Index Control Targeting Chimera) platforms enable the development of innovative medicines that combine the target-binding capability of small molecules with the therapeutic power of biologics. Solu Therapeutics is committed to advancing the field of oncology, immunology and other therapeutic areas by bringing transformative therapies to patients in need. SOURCE: Solu Therapeutics