OriCell Therapeutics Co.,Ltd.

SHANGHAI and ROSELAND, N.J., June 5, 2024 /PRNewswire/ -- Oricell Therapeutics, a pioneering clinical-stage biopharmaceutical company, has presented the two-year long-term follow-up results of OriCAR-017, an open-label Phase I study evaluating GPRC5D-targeted CAR-T therapy in patients with relapsed/refractory multiple myeloma (RRMM), in an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. Efficacy & Durability of OriCAR-017 in RRMM Patients The updated data demonstrates that OriCAR-017 elicits deep and durable responses in RRMM patients as well as a favorable safety profile, including those refractory to anti-CD38 therapies, PIs, IMIDs, and those who have failed BCMA targeting CAR-T treatment. The long-term efficacy and safety follow-up results support OriCAR-017 as a promising therapeutic approach for RRMM. Currently, Oricell is smoothly advancing the clinical development of OriCAR-017 in both China (NCT06182696) and the United States (NCT06271252). As of January 16, 2024, all ten patients responded well to OriCAR-017, with the last patient having completed a 24-month follow-up. The overall response rate (ORR) was 100.0%, with a stringent complete response rate (sCR) of 80.0% and a very good partial response rate (VGPR) of 20.0%. All patients achieved 100% minimal residual disease negativity (MRD) at day 28, which was further confirmed as 100% negative at the 3-months follow-up. The median duration of response (mDoR) was 10.43 months (95% CI, 5.00-17.00), the median progression-free survival (mPFS) was 11.37 months (95% CI, 5.93-18.00), and the median overall survival (mOS) had not yet been reached (7 patients were still under survival follow-up, 1 patient died due to disease progression, and 2 patients died from COVID-19). In the high-dose group, where 67% of patients were previously treated with BCMA CAR-T, the mDoR was 17.23 months (95% CI, 7.33-NR), and the mPFS was 19.10 months (95% CI, 8.30-NR). Currently, a BCMA CAR-T-treated patient is still in remission (>24 months). Safety Profile of OriCAR-017 in the Treatment of RRMM The therapy was well-tolerated, with 9 out of 10 patients (90%) experiencing Grade 1 cytokine release syndrome (CRS) and only 1 patient (10%) experiencing Grade 2 CRS. No Grade 3 or higher CRS was observed. The median time to CRS onset was 2 days (range 1-9 days), with a median duration of 6 days (range 3-9 days). No immune effector cell-associated neurotoxicity syndrome (ICANS) or dose-limiting toxicities (DLTs) were observed. There were no serious adverse events (SAEs) or treatment-related deaths, cerebellar disorders, or delayed infections reported. Pharmacokinetics (PK) of OriCAR-017 in RRMM Patients There were no pharmacokinetic differences between dose levels, with a Cmax of 7354.7 copies/μL and an AUC0-28 of 68587 copies•day/μg. At high doses, CAR-T cells were still detectable at 9 months, and after 21 months of follow-up, one patient had CAR-T cell expansion above the lower limit of quantitation (LLOQ). Patients with Tlast ≥ 9 months had a longer PFS compared to those with Tlast < 9 months. G5 Expression in Response to OriCAR-017 Treatment in RRMM No correlation was observed between antigen expression and therapeutic efficacy. Baseline data from all patients showed positive GPRC5D expression in bone marrow CD138+ plasma cells (MMPC), while 50% of relapsed patients showed downregulated expression detected by flow cytometry. OriCAR-017 Demonstrates Significant Potential in Severely Progressed RRMM Patients It's worth noting that the study included patients with complex and advanced disease characteristics. Among the ten R/R MM patients, 40% had extramedullary disease (EMD), 50% had undergone one or more BCMA-directed CAR-T treatments, 70% had high-risk cytogenetics, 70% were classified as ECOG 2 score status, and 80% were at International Staging System (ISS) stages II & III. OriCAR-017 demonstrates its potential as a safe and efficacious treatment option for RRMM patients, marking a substantial progress in the fight against multiple myeloma and offering hope to those facing demanding health conditions. Details for the presentation as below: Oral Presentation #7511 Title: OriCAR-017, a novel GPRC5D-targeting CAR-T, in patients with relapsed/refractory multiple myeloma: Long term follow-up results of phase 1 study (POLARIS). Session Type:Rapid Oral Abstract Session Session Title: Hematologic Malignancies—Plasma Cell Dyscrasia Link: About OriCAR-017 OriCAR-017, a chimeric antigen receptor (CAR) T cell therapy targeting GPRC5D, is a groundbreaking innovation in the treatment of relapsed/refractory multiple myeloma (RRMM). Leveraging Oricell Therapeutics' cutting-edge proprietary technology platforms, OriCAR-017 exhibits clearly differentiated binding avidity, persistence, anti-tumor efficacy and safety profile. OriCAR-017 received IND approval from FDA in Jan 2024 after its approval by NMPA in 2023. Impressive clinical results from the POLARIS study continue to be released. Currently, Oricell has assembled a skilled team to operate in both the U.S. and China, collaboratively accelerating pipeline development globally. SOURCE OriCell Therapeutics Co., Ltd.

SHANGHAI, Aug. 10, 2023 /PRNewswire/ -- August 10, 2023, in a groundbreaking development, Oricell Therapeutics Co., Ltd. ("Oricell") has received the green light for its Investigational New Drug (IND) application of OriCAR-017, a cutting-edge CAR-T cell therapy targeting GPRC5D to combat relapsed or refractory multiple myeloma (R/R MM). This remarkable milestone not only marks the world's first tier GPRC5D-targeting CAR-T therapy approved in China but also underlines Oricells prowess as a frontrunner in the biotech industry. About GPRC5D target GPRC5D, a G protein-coupled receptor that boasts high expression on multiple myeloma cells while maintaining low levels in normal tissues, has emerged as a promising therapeutic target for multiple myeloma. The revolutionary approach of independently targeting GPRC5D, regardless of BCMA expression levels, opens up new possibilities for patients who have exhausted conventional treatments or shown limited response to BCMA-targeting therapies. Earlier, data from the investigator initiated trial of OriCAR-017 (POLARIS) were presented at the 2022 ASCO (American Society of Clinical Oncology Annual Meeting) and the 2022 EHA (European Hematology Association Annual Meeting), attracting widespread attention due to its efficacy and safety advantages. The study enrolled 10 patients, included f ive patients who were previously treated with BCMA-targeted CAR T-cell therapy and four patients with extramedullary disease (EMD). According to the latest data, at a median follow-up time of 280 days (217 ~ 459 days), OriCAR-017 is turning heads with an impressive overall response rate (ORR) of 100% and a stringent complete response rate (sCR) of 80%. Additionally, compared to other therapies targeting the same antigen, OriCAR-017 demonstrated significant safety advantages, with only one case of grade 2 cytokine release syndrome (CRS) observed after treatment, while the rest were grade 1. Notably, no instances of immune effector cell-associated neurotoxicity syndrome (ICANS) and no grade 3 or higher skin toxicities or nail changes were reported, nor infection events were observed during long-term follow-up after treatment. Such reassuring safety data paves the way for reduced post-treatment clinical interventions, ultimately lightening the burden on healthcare providers and enhancing patient experiences. Oricell's Chairman and CEO, Helen Yang expressed, "OriCAR-017's approval as China's first and the world's leading GPRC5D-targeting CAR-T therapy is a pivotal moment for the biotech landscape. We are bridging the gap in late-line treatments for R/R MM, providing new hope to patients who have failed or responded poorly to different therapies targeting BCMA. The potential global impact of OriCAR-017 is undeniable, as we have been developing a global development plan for OriCAR-017 and are systematically advancing its registration for clinical trials in the United States. We have already obtained the 'Orphan Drug' designation from the Office of Orphan Products Development (OOPD) of the U.S. FDA." Helen is confident about the development prospects of this product and further stated, "with the IND clearance in China and seamless progress in overseas registrations and technology transfer, the stage is set for OriCAR-017 to become a game-changer in the fight against multiple myeloma. As Oricell continues to make strides in ground-breaking innovation, the future of cancer treatment looks more promising than ever before." SOURCE Oricell Therapeutics

Chroma Medicine, a biotech company developing therapies that turn genes on or off as a way of treating genetic disorders, has raised $135 million as it works to generate data that could show the advantages of its approach over other editing technologies. Genetic medicines in development employ a range of editing technologies that either cut or nick one or both strands of DNA. These edits can silence a problem-causing gene, but the nicks and cuts come with the risk of off-target edits. Silencing a gene in this manner isn’t a complete solution as the edited gene can still produce a gene product that causes problems, said Chroma CEO Catherine Stehman-Breen. Boston-based Chroma emerged from stealth in late 2021 with $125 million to support its research developing therapies that target the epigenome, the system that controls the expression of genes. Because this approach does not rely on cutting or nicking DNA, it doesn’t introduce the risks that come with those therapies, Stehman-Breen said. She added Chroma’s therapies could address multiple genes with a single therapy, a concept called multiplexing. While Chroma is keeping details about its programs under wraps for now, Stehman-Breen said these programs address areas of unmet medical need. She added that these programs have been selected “to demonstrate and validate the power of our technology, but also to demonstrate differentiation.” Chroma isn’t the only company aiming to leverage the epigenome. Omega Therapeutics is developing therapies called “epigenomic controllers” that dial gene expression up or down gene expression to treat disease. The Cambridge, Massachusetts-based biotech’s most advanced program targets the MYC gene and has reached Phase 1 testing in liver cancer and other solid tumors associated with this gene. The company expects preliminary data from this study later this year. Meanwhile, Tune Therapeutics is developing epigenomic therapies with multiplexing capabilities that tune multiple parts of the genome simultaneously. The Durham, North Carolina-based biotech is based on research from Duke University. Since its 2021 launch with $40 million in financing, the company has not disclosed its disease targets, other than to say its approach could address both rare and common diseases. So far, Chroma has mouse data and will be progressing to monkey studies quickly, Stehman-Breen said. Those results will enable the company to proceed to human testing, though she declined to specify timelines. But she said Chroma expects to present data from its preclinical research at an upcoming scientific meeting this year. GV led Chroma’s Series B financing, which added additional new investors Arch Venture Partners, DCVC Bio, and Mubadala Capital. Also participating in the round were earlier investors Alexandria Venture Investments, Atlas Venture, Casdin Capital, Cormorant Asset Management, Janus Henderson Investors, Newpath Partners, Omega Funds, Osage University Partners, Sofinnova Partners, T Rowe Price, and Wellington Management. Asked whether the new money is a crossover round, the type of financing that could support an IPO, Stehman-Brehn said Chroma is not specifying the nature of the new capital. But she added that Chroma will consider multiple different financing options and take advantage of the one that puts it in the best position to grow the company. Here’s a look back at some other recent life science financings: —Cancer can develop resistance to cell therapies. Cargo Therapeutics, which is developing CAR T-therapies that overcome this resistance, has raised $200 million for its pipeline, including a lead program that is on track to begin a pivotal test this summer. Third Rock Ventures, RTW Investments, and Perceptive Xontogeny Venture Fund co-led the Series A round of San Mateo, California-based Cargo. —Cell therapy developer Oricell Therapeutics closed $45 million in financing to support clinical trials in the U.S. The company’s most advanced program is a CAR T-therapy in Phase 1 testing. The new cash, which the Shanghai-based biotech describes as a Series B-1 financing, follows a $125 million Series B round that was announced last July. The additional financing was led by RTW investments. —TandemAI, a company whose technology is used in drug discovery, announced a $35 million Series A financing. The company says its tech platform integrates physics-based computational approaches with an in-house wet lab. The startup has operations in New York, San Diego, and China. Qiming Venture Partners led TandemAI’s financing. —Neuropsychiatric disease drug developer Transcend Therapeutics unveiled a $40 million Series A financing to support its clinical trial plans. The New York-based startup says it will use the new capital to launch multiple clinical trials with its “next-generation psychoactive compound.” Alpha Wave Global and Integrated Investment Partners led Transcend’s new financing. —Eluminex Biosciences raised $40 million in Series B financing to support development of its pipeline of retinal disease therapies. The Suzou, China-based company’s most advanced program is a corneal blindness therapy that has reached Phase 3 testing. Eluminex’s latest financing was led by Cenova Capital. —Abivax now has €130 million to apply toward a Phase 3 clinical trial in ulcerative colitis. Abivax’s drug, obefaximod, is a small molecule intended to reduce inflammation. Last fall, the Paris-based company began the pivotal study, which has a targeted enrollment of 1,200 ulcerative colitis patients across 36 countries. TCGX led what Abivax described as a crossover financing. But the biotech will still need more cash. The current financing is expected to last only until the end of 2024, when Abivax projects it will have results from the induction phase of the study—when patients get drug into their bodies to eliminate the symptoms. The next phase is maintenance therapy to keep the disease in remission, which will last until the end of 2025. Abivax estimates it will need to raise another €101 million to complete the clinical trial. —Avilar Therapeutics’ ambition to bring the drug modality of targeted protein degradation to disease-causing proteins on the outside of a cell has drawn interest from the venture arms of Sanofi and Astellas. They joined with Medical Excellence Capital to expand Avilar’s seed financing from $60 million in 2021 to $75 million now. Here’s more from a MedCity News profile story about Avilar, a startup formed by RA Capital Management. —Aera Therapeutics, the latest startup based on the research of genetic medicines pioneer Feng Zhang, emerged from stealth, revealing $193 million raised to date in combined Series A and B financings. Boston-based Aera aims to overcome limitations of current genetic medicines delivery approaches that use engineered viruses that carry their genetic payloads in capsids, or protein shells. These viruses can trigger an immune response and they don’t go to all tissues in the body. The technology of Aera forms capsid-like structures from human proteins. This platform includes an enzyme technology that uses programmable gene-editing enzymes. Aera says the compact size of these enzymes may help overcome the challenges of packaging and delivering genetic editors with currently available methods. —Hemab Therapeutics closed $135 million to support a pipeline of antibody drugs for rare bleeding disorders that have no approved treatments. The company’s lead program, in-licensed from Novo Nordisk, recently began a Phase 1/2 study in Glanzmann thrombasthenia. Access Biotechnology led the Series B financing for Hemab, which splits its operations between Copenhagen and Boston. —Hexagon Bio closed a $77.3 million Series B financing. The Menlo Park, California-based company discovers drugs by mining microbial genomes to find small molecules and their targets. The company says its computational and robotic capabilities have enabled it to build a proprietary database of microbial genomes to support its research. With the new cash, Hexagon plans to expand its headcount and its platform technology as it works toward nomination of drug candidates in oncology and infectious disease. Hexagon last raised cash in 2021, a $61 million Series A round. —With its Parkinson’s disease drug candidate on track to start Phase 2 testing and other neuroscience programs progress in the clinic, Cerevance raised $51 million. This expansion of its Series B financing boosted the round’s total to $116 million. Besides the plans for Parkinson’s drug CN424, Cerevance is also preparing to start a mid-stage test of a schizophrenia drug and a Phase 1 test of an amyotrophic lateral sclerosis therapy. Boston-based Cerevance discovers and develops drugs with a technology platform it calls NETseq. Apart from its internal programs, Cerevance has an Alzheimer’s disease drug discovery alliance with Merck. —Halda Therapeutics, founded by Yale University professor and targeted protein degradation pioneer Craig Crews, emerged from stealth to reveal a new drug modality backed by $76 million in financing to date. New Haven, Connecticut-based Halda develops small molecules that bring together two proteins, one of them cancer specific and the other with an essential function. This approach leads to the elimination of the essential function, which then causes the death of the cancer cell. Preclinical data for what Halda describes as a “hold and kill” mechanism were outlined in a poster presentation at the recent American Society of Clinical Oncology Genitourinary Cancers Symposium in San Francisco. Image: abluecup, Getty Images