CD123

Funding to accelerate clinical validation of AvenCell's proprietary Universal Switchable CAR-T cell therapy platform, for the treatment of a wide range of hematologic malignancies and auto-immune diseases New investors F-Prime Capital, Eight Roads Ventures Japan, Piper Heartland Healthcare Capital and NYBC Ventures join Novo Holdings alongside founding investor Blackstone Life Sciences Michael Bauer of Novo Holdings and Nihal Sinha from F-Prime Capital to join AvenCell's Board of Directors WATERTOWN, Mass., Oct. 22, 2024 /PRNewswire/ -- AvenCell Therapeutics, Inc. ("AvenCell"), a leading clinical-stage cell therapy company focused on advancing both autologous and allogeneic switchable CAR-T cell therapies, today announced that it has raised $112 million in Series B financing. The financing was led by global life sciences investor Novo Holdings. New investors F-Prime Capital, Eight Roads Ventures Japan, Piper Heartland Healthcare Capital and NYBC Ventures also participated in the round alongside founding investor Blackstone Life Sciences. As part of this financing, Michael Bauer, Ph.D., Partner, Venture Investments, Novo Holdings, and Nihal Sinha, MB BChir, Partner, F-Prime Capital will join AvenCell's Board of Directors. This latest financing will support ongoing clinical validation of AvenCell's proprietary, switchable universal CAR-T cell therapy platform that generates CAR-T cells that can rapidly be turned "Off" and "On" even after they are administered to a patient. The universal platform was developed to more safely and effectively treat a wide range of hematologic malignancies compared to conventional cell therapies. Current clinical assets utilizing AvenCell's universal platform include AVC-101, a highly-differentiated autologous CAR-T cell candidate, and AVC-201, a CRISPR-engineered allogeneic CAR-T cell candidate. Both products target the antigen CD123 found on most Acute Myeloid Leukemia (AML) cells. The ongoing trials are investigating both products for the treatment of relapsed/refractory AML, which has a high unmet medical need, and very limited treatment options for patients. In addition, AvenCell has several pipeline candidates entering the clinic over the next two years. "We are excited by the progress of our pipeline and believe our next-generation immunotherapies have the power to address significant unmet patient needs," said Andrew Schiermeier, Ph.D., Chief Executive Officer, AvenCell Therapeutics. "AvenCell is working to transform the standard of care through switchable, adaptable and readily available cell therapy treatments that can better treat a wide range of difficult-to-treat cancer and autoimmune diseases. The support of Novo Holdings and this leading group of new investors will be integral to our ability to progress and bring these therapies to patients." "AvenCell's universal switchable technology and CRISPR-engineered allogeneic platforms are first-of-its-kind and represent a step change in the field of cell therapy. Both AVC-101 and AVC-201 have already yielded encouraging safety and efficacy results in early clinical trials in a very difficult to treat disease like AML. Our investment reflects our confidence in these assets and the future of cell therapy, as well as our long-held strategy to support companies transforming care solutions to enhance patient outcomes," said Michael Bauer, Partner, Venture Investments, Novo Holdings. "AvenCell's switchable CAR-T platform represents a paradigm shift in cell therapy, offering unprecedented control over treatment dynamics," said Nihal Sinha, MB BChir, Partner at F-Prime Capital. "The ability to modulate CAR-T cell activity post-infusion could address critical safety and efficacy challenges in current therapies. We look forward to supporting AvenCell's journey in advancing these promising cell therapies through clinical development, with the potential to address significant unmet needs in the treatment landscape." About AvenCell Therapeutics AvenCell derives its name from the French word "avenir" to reflect the aim to be the FUTURE of cell therapy. AvenCell is building a truly transformative cell therapy company that targets difficult-to-treat cancers, with its lead programs focusing on acute myeloid leukemia (AML) and additional programs targeting other hematological malignancies. AvenCell was formed with the goal to create truly allogeneic cells that persist as long or longer than autologous therapies and develop a universal and switchable construct that allows complete control and target redirection of T cells after they are infused into a patient. Integration of these two platforms allows for complete separation of the manufacturing of cells from ultimate patient and cancer target, thus providing significant scalability potential at orders of magnitude more efficient than current approaches. AvenCell Therapeutics, Inc. was launched in 2021 by Blackstone Life Sciences, Cellex Cell Professionals, and Intellia Therapeutics. AvenCell is headquartered in Watertown, Massachusetts with additional R&D and manufacturing operations in Dresden, Germany. For more information, visit . Follow AvenCell on social media: LinkedIn. SOURCE AvenCell Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

AvenCell Therapeutics on Tuesday said it collected $112 million to continue clinical studies of two experimental acute myeloid leukemia CAR-T cell therapies. The Series B raise comes despite cold investor sentiment toward cancer cell therapies as of late. “We see quite a lot of investors out there who have invested previously in cell therapy and haven’t been pleased with how that’s played out,” AvenCell CEO Andrew Schiermeier said. “But it comes back to the basics, which is if you have very strong science and very strong clinical data, the investors are certainly happy to listen,” Schiermeier added. The round was led by Novo Holdings, and included investors such as F-Prime Capital, Eight Roads Ventures Japan, Piper Heartland Healthcare Capital and NYBC Ventures, as well as AvenCell’s founding investor Blackstone Life Sciences. AvenCell was born out of gene editing technology from Intellia Therapeutics, money from Blackstone, and an acquisition of the German biotech GEMoaB. The company is building switchable CAR-T therapies that can be turned on and off using a protein. The idea is that doing so can reduce the serious toxicities associated with the therapies. AvenCell is testing its approach in acute myeloid leukemia, where no CAR-T therapies have been successfully developed yet. Part of that lack of success, Schiermeier said, is due to the toxicity associated with the target for AML called CD123. “The more aggressively you go after CD123, the more you wind up with unacceptable toxicity,” he said. “So it did serve as a perfect initial indication for us to go with what we call our switchable CAR concept.” Novo Holdings partner Michael Bauer said the bar for investing in an AML cell therapy is lower than one for other blood cancers such as multiple myeloma or B cell lymphomas, where CAR-T therapies have already been approved. “AML is the last frontier in heme,” said Bauer, who is joining AvenCell’s board. Bauer said that Novo Holdings had looked at GEMoaB, but hadn’t invested at the time because it also had a stake in Arcellx, which was developing a similar approach. Novo Holdings has since exited Arcellx (which is now a public company), AvenCell has produced clinical data, and “​​the valuation is as adjusted to what we believe is more reasonable,” Bauer said. Last year, AvenCell shared data showing that in its first 19 patients in an early-stage study, the overall response rate was 53%. Schiermeier said the company now has data with 26 patients in the dose escalation phase of its study and “the first handful” of patients in the dose expansion phase. He also said there are “a couple of patients who have quite long-term complete responses — beyond six months.” AvenCell is also developing a second AML therapy that uses gene editing technology. That cell therapy also targets CD123, but is allogeneic, meaning that it doesn’t require a patient’s own cells. The company announced in February that it dosed its first patient with the allogeneic CAR-T cell therapy. The biotech is also interested in the autoimmune space, where many cell therapy companies have now migrated. As for when it will start work in that space, Schiermeier said, “I don’t think it is too far away.”

VIP943 demonstrates promising safety and tolerability and achieves two complete responses to date in Phase 1 dose-escalation study, reinforcing the program’s potential and validating the VersAptx™ Platform technology Vincerx focusing resources on continued development of VIP943 Expected cash runway into early 2025 Management to host webcast and Q&A today at 5:00 PM EDT to review pipeline and corporate updates, followed by commentary from key opinion leader Dr. M. Yair Levy Oct. 07, 2024 -- Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, announced two complete responses in the ongoing first-in-human, Phase 1 dose-escalation study of VIP943, the Company’s next-generation antibody-drug conjugate (ADC) being evaluated in relapsed/refractory acute myeloid leukemia (AML), higher-risk myelodysplastic syndrome (HR-MDS), and B-cell acute lymphoblastic leukemia (B-ALL). The Company also provided pipeline and corporate updates. The ongoing Phase 1 dose-escalation study of VIP943 has enrolled 22 patients to date across several escalating dose cohorts (0.2 to 1.3 mg/kg once weekly). These 22 patients represent a ‘hard-to-treat’ salvage population, which rarely responds to monotherapy. Nine patients (six AML; three HR-MDS) have received at least three doses of an efficacious dose of VIP943 (i.e., ≥1.0 mg/kg). Of these nine patients, four (44%) remain on study. So far, one patient with relapsed AML has achieved complete remission with incomplete hematologic improvement (CRi) and one patient with HR-MDS has achieved complete remission with limited count recovery (CRL) based on international consensus response criteria. These response criteria are widely recognized as an approvable benchmark in AML and MDS studies, further underscoring the significance of these early results. “We are excited by the emerging data from our Phase 1 study of VIP943, showing clinical responses in difficult-to-treat patients," said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx. "We believe these promising clinical responses highlight the potential of VIP943 as a best-in-class therapy for CD123+ hematologic malignancies and validate our VersAptx platform’s ability to create safer, more effective bioconjugates by overcoming the challenges of historical ADCs.” As of August 2024, VIP943 has shown favorable safety and tolerability, with no dose-limiting toxicities reported in 22 patients. Serious adverse events (SAEs) have been consistent with expectations for this patient population. The most common SAEs included pneumonia (three patients, 14%), and cellulitis and febrile neutropenia (two patients each, 9%). Only one patient (5%) experienced a drug-related SAE (Grade 3 diarrhea). Target engagement (i.e., receptor occupancy) has been demonstrated by binding of VIP943 to CD123+ peripheral blood blasts from patients with AML from the Phase 1 study. Maximal receptor occupancy of 84% was achieved in the highest dose cohort (1.3 mg/kg). Across all the cohorts, receptor occupancy was retained for less than 96 hours. Concurrent decreases in CD123+ peripheral blood blasts were also observed after dosing. These pharmacodynamic (PD) markers show that VIP943 is binding to and eliminating CD123+ malignant cells. Preliminary pharmacokinetic (PK) data continues to show low release of payload (≤1% in plasma). The half-life of VIP943 is less than 96 hours, and no accumulation occurs with repeat dosing. These PK and PD results have prompted evaluation of twice weekly dosing of VIP943 as a potential “induction” regimen. Enrollment in the once weekly and twice weekly dosing cohorts is ongoing. Dr. Hamdy continued, “Our initial clinical results demonstrate that VersAptx is a next-generation platform that overcomes key challenges associated with traditional ADCs. The PK profile shows that our linker is stable, cleaving exclusively inside cells without extracellular degradation. Our PD results coupled with clinical responses confirm the payload effectively kills cancer cells in peripheral blood and bone marrow without harming nearby healthy tissue. This innovative design with proof-of-concept in Phase 1 reinforces our confidence in VersAptx as a transformative platform for ADC development.” The company anticipates providing another data update on the ongoing Phase 1 VIP943 study by the end of the year. Dr. M. Yair Levy, Director of Hematologic Malignancies Research at Texas Oncology added, "Although this is still early data, VIP943 is clearly differentiated from other ADCs, particularly with its favorable safety profile. We’re not seeing neutropenia as a dose-limiting toxicity, which is encouraging and may allow the drug to move into earlier lines of therapy in combination. I look forward to the continued development of VIP943 and its potential to improve treatment options for patients with CD123+ malignancies.” VIP236 is Vincerx’s first-in-class small molecule drug conjugate (SMDC) being evaluated in an ongoing first-in-human, Phase 1 dose-escalation study as a monotherapy in patients with advanced solid tumors. As of September 2024, 29 patients have been enrolled. Of these patients, 20 were evaluable per-protocol for response from the every 2- or 3-week schedule; nine of 20 patients had stable disease for a disease control rate of 45%. In addition, one of these subjects has been on treatment for over 300 days and four additional patients were on study for more than 120 days, demonstrating promising monotherapy duration of response in patients with advanced cancer. VIP236 continued to show a favorable safety and tolerability profile in these 29 patients, with no instances of the dose-limiting side effects commonly associated with camptothecins, such as life-threatening diarrhea, severe stomatitis/mucositis, or interstitial lung disease. These results support the potential role of VIP236 as a strong combination agent for the treatment of advanced cancers. Considering the promising VIP236 clinical data, the Company intends to pursue a strategic partner to champion its future development for the benefit of patients. “We've made significant progress in identifying an effective dose and schedule. We believe it is now crucial to study VIP236 in the right patient population, which could include triple-negative breast cancer and gastric cancer, where camptothecins are used, especially in combination with other anticancer agents,” added Dr. Hamdy. By transitioning VIP236 to a partnering asset, the Company plans to streamline its operations and focus its efforts on the continued development of its lead ADC, VIP943. Enitociclib, a highly selective CDK9 inhibitor, is currently being evaluated in a Phase 1 dose-escalation study in combination with venetoclax and prednisone for relapsed/refractory diffuse large B-cell lymphoma (DLBCL) and peripheral T-cell lymphoma (PTCL), in collaboration with the National Institutes of Health (NIH). As of September 2024, the study reported four partial responses (PRs) in seven patients (57% overall response rate), including one patient with double hit lymphoma (DH-DLBCL) and three patients with PTCL. All responses occurred in patients considered refractory by SCHOLAR-1 criteria and included one patient with prior CAR-T therapy. The study is currently enrolling in the third dose level (enitociclib 30 mg [efficacious dose] and venetoclax 600 mg) with two patients enrolled to date. Additionally, in a separate Phase 1 study of enitociclib as a monotherapy (30 mg), one patient with transformed follicular lymphoma achieved a metabolic PR. As of September 2024, this patient remains on enitociclib monotherapy after more than 26 months. Overall, these clinical results continue to show the promising safety, tolerability, and efficacy of enitociclib for the treatment of relapsed/refractory lymphoma. The Company is actively focused on finding a strategic partner to continue the development of this asset. VIP236, the first-in-class SMDC from the VersAptx Platform, consists of an αvβ3 integrin binder, a neutrophil elastase linker cleaved in the tumor microenvironment, and a camptothecin payload optimized for high permeability and low active efflux. VIP236 was designed to deliver its payload to advanced/metastatic solid tumors that express αvβ3. VIP236 is being evaluated in a first-in-human, Phase 1 dose escalation study in patients with advanced malignancies (NCT05712889). VIP943, the first ADC from the VersAptx platform, consists of an anti-CD123 antibody, a unique linker cleaved intracellularly by legumain, and a novel kinesin spindle protein inhibitor (KSPi) payload enhanced with Vincerx’s CellTrapper™ technology. Vincerx’s proprietary effector chemistry (linker + payload) was designed to reduce non-specific release of the payload and ensure payload accumulation in cancer cells versus healthy cells. The increased therapeutic index has the potential to address challenges associated with many ADCs by improving efficacy and reducing severe toxicities. VIP943 is being evaluated in a Phase 1 dose-escalation trial in patients with relapsed/refractory AML, HR-MDS, and B-ALL who have exhausted standard therapeutic options (NCT06034275). Enitociclib, a highly selective CDK9 inhibitor, is currently being evaluated in a Phase 1 dose-escalation study (NCT05371054) in combination with venetoclax and prednisone for DLBCL and PTCL, in collaboration with the NIH. Enitociclib has demonstrated favorable safety and PK, with significant clinical benefits across various indications, including durable complete metabolic remissions in patients with double-hit (DH)-DLBCL and stable disease in solid tumors, notably in ovarian cancer, suggesting promising potential for future combination studies. VersAptx is a versatile and adaptable next-generation bioconjugation platform. The modular nature of this innovative platform allows the combination of different targeting, linker, and payload technologies to develop bespoke bioconjugates that address different cancer biologies. With this platform, (i) antibodies and small molecules can be used to target different tumor antigens, (ii) linkers can be designed to reduce non-specific release of the payload, cleave intracellularly or extracellularly, and conjugate to single or multiple payloads, and (iii) payloads can be designed with reduced permeability using our CellTrapper technology to ensure accumulation in cancer cells or to be permeable for release in the tumor microenvironment. The VersAptx platform allows the development of bioconjugates designed to address the safety and efficacy challenges of historical ADCs. Vincerx Pharma, Inc. is a clinical-stage biopharmaceutical company committed to developing differentiated and novel therapies to address the unmet medical needs of patients with cancer. Vincerx has assembled a seasoned management team with a proven track record of successful oncology drug development, approvals, and value creation. Vincerx’s diverse pipeline consists of the next-generation antibody-drug conjugate, VIP943, in Phase 1; small molecule-drug conjugate, VIP236, in Phase 1; preclinical antibody-drug conjugate, VIP924; CDK9 inhibitor, enitociclib, in an NIH-sponsored Phase 1; and VersAptx, its versatile and adaptable, next-generation bioconjugation platform. The content above comes from the network. if any infringement, please contact us to modify.