On November 7, 2024 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported financial results for the third quarter ended September 30, 2024, and provided recent business highlights (Press release, Arcellx, NOV 7, 2024, View Source [SID1234647987]).

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"We believe the data from the recently published ASH (Free ASH Whitepaper) abstracts continues to differentiate anito-cel’s clinical profile as a potentially best-in-class treatment option for multiple myeloma patients," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "The 30.2-month median progression-free survival demonstrated in our Phase 1 study in a challenging patient cohort coupled with the promising results from our iMMagine-1 Phase 2 registrational study highlight the potential impact we could have for patients. That impact is further enhanced by the high tolerability demonstrated through both the Phase 1 and iMMagine-1 studies to date, where notably, no delayed or non-ICANS neurotoxicities were observed in the over 140 patients treated to date. Patients and clinicians evaluate cell therapies on their safety, efficacy, delivery reliability, service, and accessibility. We believe we’re well positioned to deliver on these important factors in a differentiated way that best serves the multiple myeloma community. Our partnership with Kite allows us to leverage their established global commercial capabilities, positive brand recognition with physicians, and industry-leading manufacturing reliability and turnaround times which we believe contributes to our competitive advantage. It’s an exciting time at Arcellx! We are preparing for the commercial launch of anito-cel as there remains an unmet need for a therapy that physicians can use across a broad patient population."

Recent Business Progress

Announced presentations at the 66th American Society for Hematology Annual Meeting and Exposition:

Phase 2 Registrational Study of Anitocabtagene Autoleucel for the Treatment of Patients With Relapsed and/or Refractory Multiple Myeloma: Preliminary Results From the iMMagine-1 Trial (abstract #1031)

As detailed in the abstract (#1031) as of June 1, 2024, 58 patients had received anito-cel infusion with ≥2 months of follow-up after infusion, with a median follow-up of 10.3 months (range, 2.0-17.8). The median age was 66 years (range, 38-77). Patients had received a median of four prior lines of treatment (range, 3-8) with 26 patients (45%) having received only three prior lines of treatment. Forty patients (69%) were triple-class refractory and 20 (34%) were penta-class refractory.

Investigator-assessed overall response rate (ORR) per International Myeloma Working Group (IMWG) criteria was 95% (55/58) with a complete response/stringent complete response (CR/sCR) rate of 62% (36/58). Of those evaluable for minimal residual disease (MRD) testing (n=39), 36 (92%) achieved MRD negativity at least to the level of 10-5. The Kaplan–Meier-estimated 6-month progression-free survival (PFS) and overall survival (OS) rates (95% CI) were 90% (77-96) and 95% (85-98), respectively. Median (mPFS) and median OS have not yet been reached.

No delayed neurotoxicities, including no parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome have been observed to date. Forty-six patients (79%) had either no cytokine release syndrome (CRS) (n=9, 16%) or Grade (Gr) 1 CRS (n=37, 64%). Thirty-one patients (53%) had no fever or CRS in the first four days of anito-cel. Any Grade CRS was observed in 49 patients (84%; Gr3/4 0%). Any Grade ICANS was observed in 5 patients (9%; Gr3 2%), with all cases resolved without sequelae. Three deaths occurred due to adverse events (AEs) (both related and unrelated; retroperitoneal hemorrhage, CRS, and fungal infection). No additional treatment or therapy-related deaths or Grade ≥3 CRS or ICANs events have occurred to date. Cytopenias were the most common Grade ≥3 treatment-emergent AEs; 36 patients (62%) had Grade ≥3 neutropenia, 15 (26%) had Grade ≥3 thrombocytopenia, and 15 (26%) had Grade ≥3 anemia.

Conclusions

Preliminary results from the first 58 patients in the Phase 2 iMMagine-1 study demonstrate deep and durable responses and manageable safety in a high-risk fourth line or higher (4L+) RRMM population including triple- and penta-class refractory disease. Notably, no delayed neurotoxicities, including no cranial nerve palsies, Guillain-Barré syndrome, or Parkinsonian-like symptoms have been observed with anito-cel to date. Updated Phase 2 data with a more recent data cut will be presented at the oral presentation during ASH (Free ASH Whitepaper).

Presentation details:

Speaker: Ciara Freeman, M.D., Ph.D., H. Lee Moffitt Cancer Center
Session Name: 655. Multiple Myeloma: Cellular Therapies: Unleashing Cell Therapies Against Myeloma
Session Date: Monday, December 9, 2024
Session Time: 4:30 p.m. – 6:00 p.m.
Presentation Time: 5:30 p.m.
Location: Marriott Marquis San Diego Marina, Pacific Ballroom Salons 24-26
Publication Number: 1031
Submission ID: 198499

Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Patients With Relapsed and/or Refractory Multiple Myeloma (RRMM) (abstract #4825)

In the Phase 1 study, 40 patients were enrolled and 38 patients received anito-cel. All 38 patients demonstrated investigator-assessed clinical response per 2016 IMWG criteria, (ORR, 100%) with 30 CR/sCR (≥CR rate, 79%), 5 very good partial response (≥VGPR rate, 92%), and 3 partial response (PR). Of those evaluable for MRD testing (n=28), 25 (89%) achieved MRD negativity at 10-5. With a median follow-up of 38.1 months, median OS was not reached and median PFS was 30.2 months. The safety profile was manageable with no delayed neurotoxicities observed to date, including no parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome. Further investigations of anito-cel are ongoing in 4L+ RRMM (iMMagine-1, NCT05396885) and in earlier lines (iMMagine-3, NCT06413498).

Presentation details:

Speaker: Michael R. Bishop, M.D., The University of Chicago
Session Name: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities
Session Date: Monday, December 9, 2024
Presentation Time: 6:00 p.m. – 8:00 p.m.
Location: San Diego Convention Center, Halls G-H
Publication Number: 4825
Submission ID: 201080

Health Related Quality of Life (HRQoL) in Relapsed/Refractory Multiple Myeloma (RRMM): A Systematic Literature Review (SLR) and Meta-Analysis (abstract #4721)

Quantifying pre-treatment HRQoL burden is important as a reference for contextualizing baseline patient burden as emerging therapies for RRMM continue to evolve. This SLR synthesized studies that reported data for key multiple myeloma HRQoL instruments. It found that patients with RRMM had clinically meaningful impairments from population norms in important domains, such as Global Health Status and cognitive, physical, and emotional functioning. The SLR also found that pre-treatment HRQoL worsened with increasing lines of therapy.

Presentation details:

Speaker: Rahul Banerjee, M.D., Fred Hutchinson Cancer Center
Session Name: 653. Multiple Myeloma: Clinical and Epidemiological: Poster III
Session Date: Monday, December 9, 2024
Presentation Time: 6:00 p.m. – 8:00 p.m.
Location: San Diego Convention Center, Halls G-H

Treatment Patterns and Outcomes in Triple-Class Exposed Patients with Relapsed and Refractory Multiple Myeloma: Findings from the Flatiron Database (abstract #6962)

In order to understand the contemporary unmet need in the rapidly evolving treatment landscape for patients with triple-class exposed RRMM – those exposed to immunomodulatory drugs, proteasome inhibitors, and anti-CD38 monoclonal antibodies – in the 4L+ setting, a retrospective cohort study using the Flatiron Health electronic health record (HER) was conducted (sample size=594). This study found no clear standard of care in the 4L+ setting, and suboptimal health outcomes under the current treatment landscape (ORR=34%, PFS=4.1 months, and OS=15.4 months), emphasizing an urgent need for more effective and durable therapies for patients in this setting. This abstract will be published in a supplemental issue of Blood in November 2024.

First patients dosed in iMMagine-3, a global randomized Phase 3 study, assessing anito-cel in patients previously treated with both an immunomodulatory (IMiD) drug and an anti-CD38 monoclonal antibody. Kite is manufacturing for this study.

Third Quarter 2024 Financial Highlights

Cash, cash equivalents, and marketable securities:

As of September 30, 2024, Arcellx had cash, cash equivalents, and marketable securities of $676.7 million. Arcellx anticipates that its cash, cash equivalents, and marketable securities will fund its operations into 2027.

Collaboration revenue:

Collaboration revenue were $26.0 million and $15.0 million for the quarters ended September 30, 2024 and 2023, respectively, an increase of $11.0 million. This increase was primarily driven by the December 2023 expansion to the license and collaboration agreement with Kite Pharma, Inc.

R&D expenses:

Research and development expenses were $39.2 million and $43.8 million for the quarters ended September 30, 2024 and 2023, respectively, a decrease of $4.6 million. This decrease was primarily driven by an expense in 2023 associated with our Lonza manufacturing services agreement. The decrease was partially offset by increased costs relating to other preclinical pipeline programs and increased personnel costs, which include non-cash stock-based compensation expense.

G&A expenses:

General and administrative expenses were $20.5 million and $16.0 million for the quarters ended September 30, 2024 and 2023, respectively, an increase of $4.5 million. This increase was primarily driven by increased personnel costs, which include non-cash stock-based compensation expense.

Net losses:

Net losses were $25.9 million and $39.3 million for the quarters ended September 30, 2024 and 2023, respectively.

Upcoming Webcast Event:

Arcellx will host a live webcast event with an expert panel of clinicians on Monday, December 9, 2024, at 8:30 p.m. PT to discuss clinical results from its Phase 1 and iMMagine-1 trials. The event will be accessible from Arcellx’s website at www.arcellx.com in the Investors section. A webcast replay will be archived and available for 30 days following the event.

On November 7, 2024 IASO Biotherapeutics ("IASO Bio"), a biopharmaceutical company dedicated to discovering, developing, manufacturing and commercializing innovative cell therapy and antibody products, reported that the results of the phase 1b/2 clinical study FUMANBA-1 of its fully human anti-BCMA CAR-T therapy, Equecabtagene Autoleucel (trade name: Fucaso), for the treatment of relapsed/refractory multiple myeloma (R/RMM), have been published in the leading medical journal JAMA Oncology (Press release, IASO Biotherapeutics, NOV 7, 2024, View Source [SID1234647986]). The study evaluated the efficacy and safety of Equecabtagene Autoleucel in patients with R/RMM who had previously received ≥3 lines of prior therapies. The results demonstrated that Equecabtagene Autoleucel achieved a high overall response rate and durable remission in patients, with a favorable safety profile.

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JAMA Oncology published clinical data on 103 patients who received infusion of Equecabtagene Autoleucel (Fucaso) as of September 9, 2022, with a median follow-up of 13.8 months (range: 0.4-27.2 months). In terms of efficacy, among the 101 evaluable patients, the overall response rate (ORR) was 96.0% (97/101), and the stringent complete response/complete response rate (sCR/CR) was 74.3% (75/101). Among the 89 patients without prior CAR-T therapy, the ORR was 98.9% (88/89), and the sCR/CR rate was 78.7% (70/89). In these 101 patients, the median time to response was 16 days (range: 11-179), while the median duration of response (DOR) and median progression-free survival (PFS) had not been reached yet. The 12-month PFS rate was 78.8% (95% CI: 68.6-86.0). Additionally, 95% (96/101) of the patients achieved minimal residual disease (MRD) negativity, with a median time to MRD negativity of 15 days (range: 14-186). All patients with sCR/CR achieved MRD negativity, and the median duration of MRD negativity had not been reached.

In terms of safety, 93.2% (96/103) of the patients experienced cytokine release syndrome (CRS), most of which were grade 1 or 2, with only one patient experiencing grade 3 or higher CRS. Only 1.9% (2/103) of the patients developed immune effector cell-associated neurotoxicity syndrome (ICANS), with one case each of grade 1 and grade 2.

Notably, updated data from this study, as of December 31, 2022, was presented during the 2023 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The median follow-up had increased to 18.07 months, with an ORR of 96.1% among the 103 evaluable patients. The sCR/CR rate was 77.7%, and all patients who achieved CR or better exhibited 100% MRD negativity. Among the 91 patients without prior CAR-T therapy, the ORR reached 98.9%, with an sCR/CR rate of 82.4% and an MRD negativity rate of 97.8%. Notably, 81.7% of these patients remained MRD-negative at 12 months post-infusion, with a 12-month PFS rate was 85.5%. In addition, Equecabtagene Autoleucel could showed prolonged persistence in the body, at 12 months post-infusion, 50% of patients had a vector copy number (VCN) above the limit of detection, and 40% still had detectable VCN persistence at 24 months.

Professor Lu-gui Qiu, from the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, stated: "The results of the FUMANBA-1 study demonstrate the encouraging efficacy and safety of Equecabtagene Autoleucel in treating patients with relapsed/refractory multiple myeloma. Its groundbreaking fully human scFv design overcomes the high immunogenicity issues commonly associated with animal-derived CAR-T cells while maintaining optimal affinity for BCMA-expressing tumor cells. Its excellent dissociation kinetics facilitates rapid expansion and long-term persistence of Equecabtagene Autoleucel in vivo."

Professor Huang He, from The First Affiliated Hospital, Zhejiang University School of Medicine, stated, "Equecabtagene Autoleucel (Fucaso) was approved in China in June 2023. Over the past year since its launch, it has brought significant survival benefits to patients with relapsed/refractory multiple myeloma in China and from overseas. Meanwhile, we have accumulated valuable real-world experience, and we expect this innovative therapy to benefit more patients."

Professor Chunrui Li, from Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology, stated: "Equecabtagene Autoleucel is the world’s first fully human CAR-T therapy to be approved. It’s binding to BCMA, utilizing both heavy and light chains with optimal affinity, facilitates dissociation from BCMA antigen after tumor cell killing, thereby reducing CAR-T cell self-exhaustion. Along with lower immunogenicity, this leads to rapid, deep, and sustained remission in patients with multiple myeloma."

Dr. Jie Chen, Chief Medical Officer of IASO Bio, stated: "We are delighted that the phase 1b/2 clinical study data of Equecabtagene Autoleucel for the treatment of R/RMM has been published in JAMA Oncology. The results of this study, which targeted patients with R/RMM who had previously received ≥3 lines of prior therapies are very inspiring. We thank the FUMANBA-1 research team for their rigorous scientific approach and high-standard execution. Currently, IASO Bio is actively conducting and advancing the phase III clinical study (FUMANBA-3) of Equecabtagene Autoleucel for the treatment of multiple myeloma patients who have received 1-2 lines of prior therapies. We anticipate this clinical study, based on clinical data from Chinese patients, will yield positive results soon, bringing new treatment option for more patients."

About FUMANBA-1 study

FUMANBA-1 is a single-arm, open-label phase 1b/2 registrational clinical study conducted in 14 Chinese centers to assess the efficacy and safety of the Equecabtagene Autoleucel in patients with R/RMM who have received ≥3 lines of prior therapies. The trial enrolled patients with RRMM who had previously received at least three lines of therapy, including proteasome inhibitors and immunomodulatory agents-based chemotherapy regimens, and had disease progression on their last line of therapy. Patients who had previously received BCMA CAR-T therapy were also eligible for enrollment.

About Equecabtagene Autoleucel (Fucaso)

Equecabtagene Autoleucel (Fucaso) is an innovative fully-human anti- BCMA CAR-T cell therapy which uses lentivirus as a gene vector to transfect autologous T cells. The CAR contains a fully-human scFv, CD8a hinge and transmembrane, and 4-1BB-mediated co-stimulation and CD3ζ activation domains. Based on rigorous molecular structure screening and comprehensive in vitro and in vivo functional evaluations, Fucaso demonstrates rapid and potent efficacy, accompanied by exceptional long-term persistence in vivo, enabling patients to achieve higher and deeper remissions, providing continuous protection and care for those suffering from multiple myeloma.

On November 7, 2024 Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported financial results for the third quarter ended September 30, 2024, and provided a corporate update (Press release, Ryvu Therapeutics, NOV 7, 2024, View Source [SID1234647985]).

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Pawel Przewiezlikowski, Chief Executive Officer of Ryvu Therapeutics, said:

– We have significantly accelerated enrollment in RVU120 Phase II studies since September thanks to the activation of numerous new sites in the RIVER-52 and RIVER-81 studies for patients with r/r AML and HR-MDS and the launch of the REMARK study for patients with LR-MDS. We are looking forward to launching the fourth RVU120 Phase II study – POTAMI-61 – shortly, which will focus on patients with myelofibrosis. By the end of the year, we plan to summarize the initial data from the most advanced RVU120 clinical development paths at an investor event scheduled for December 12. In parallel, we are preparing to launch JASPIS-01, a Phase II study of MEN1703 (SEL24) in patients with DLBCL, while also advancing our promising preclinical pipeline.

Q1-Q3 2024 SUMMARY AND RECENT CORPORATE EVENTS

Pipeline progress

In early 2024, Ryvu launched two Phase II studies with RVU120: (i) the RIVER-52 study investigating RVU120 as monotherapy in two genetically defined cohorts of patients with relapsed or refractory (r/r) AML and in a cohort of patients with HR-MDS, and (ii) the RIVER-81 study investigating RVU120 in combination with venetoclax in patients with r/r AML.
The RIVER-52 study was initially launched at clinical sites in Poland and Italy. Starting in September 2024, the study expanded to Spain, France and Canada. During the summer, the site activation process was slower than expected, primarily due to the limited availability of site staff. This staff shortage, combined with the presence of competing clinical studies at several sites impacted the actual enrollment rate vs. original estimates. Increased site activation efforts and additional measures to maximize enrollment resulted in a return to expected patient enrollment levels starting in September 2024. As of October 31, 2024, 33 sites had been activated for enrollment, more than doubling the number of active sites since the status reported on August 31, 2024, when 16 sites were active. Ryvu aims to activate a total of 46 sites by the end of Q4 2024.
The RIVER-81 study was initially launched at clinical sites in Poland and Italy, followed by the activation of additional sites in Spain and France. As of October 31, 2024, 33 sites had been activated out of a total of 34 sites that the Company plans to activate in the aforementioned four countries by the end of the year.
In September, Ryvu announced the dosing of the first patient in REMARK, a Phase II study evaluating the efficacy of RVU120 as a monotherapy for the treatment of patients with low-risk myelodysplastic syndromes (LR-MDS). The REMARK study has commenced enrollment of patients across five countries: Poland, Germany, France, Spain and Italy. Up to approximately 25 clinical sites are planned to be activated across these countries. REMARK is being conducted as an investigator-initiated study through the EMSCO network with Prof. Uwe Platzbecker, a globally renowned expert in the field of LR-MDS, as the Coordinating Principal Investigator.
POTAMI-61, a Phase II study investigating RVU120 both as a monotherapy and in combination therapy to treat patients with myelofibrosis (MF), was initially launched at clinical sites in Poland and Italy. As of October 31, 2024, five clinical sites have been activated for enrollment, with a total of 18 sites planned to be activated across these two countries by the end of 2024. Patient dosing is expected to commence shortly.
At the 2024 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) (ENA) Symposium (October 23-25, Barcelona, Spain), Ryvu presented clinical and preclinical data from RVU305 (MTA-cooperative PRMT5 inhibitor), the ONCO Prime discovery platform, RVU120 (CDK8/19 inhibitor), and the WRN inhibitor program. Key takeaways:
Ryvu has developed a potentially best-in-class MTA-cooperative PRMT5 inhibitor, RVU305, demonstrating favorable drug-like properties and effective PRMT5 inhibition dependent on MTA binding.
Ryvu has developed a proprietary platform, ONCO Prime, to discover novel synthetic lethal (SL) inhibitors targeting key oncogenic drivers such as KRAS and other mutations. ONCO Prime uses human intestinal stem cell (hISC)-derived cancer model cells, patient-derived xenografts (PDXs), and clinical samples to conduct genomic and functional analyses.
Ryvu is developing a series of potent and selective WRN helicase inhibitors that demonstrate pronounced efficacy in tumors with high microsatellite instability (MSI-H). The compounds exhibit favorable pharmacokinetics, achieving optimal exposure and target engagement, further enhancing their therapeutic potential in MSI-H cancers.
RVU120 is being tested in patients with solid tumors in an ongoing Phase I/II clinical trial, AMNYS-51. RVU120 has demonstrated a manageable safety profile across multiple dose levels and dosing schedules in patients with advanced or metastatic solid tumors with early signs of activity in patients with adenoid cystic carcinomas (ACC).
On September 9, 2024, the Management Board decided to advance Ryvu’s potentially best-in-class PRMT5 inhibitor RVU305 to further steps of preclinical development, including toxicology and API/IMP manufacturing, targeting IND/CTA filing in H2 2025.
Key business events

In October 2024, Ryvu Therapeutics and nCage Therapeutics announced a research collaboration to develop a next-generation antibody-drug conjugate (ADC) platform.
In October 2024, Ryvu announced the conclusion of an agreement for the operational execution of the MEN1703 (SEL24) Phase II clinical trial in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), known as the JASPIS-01 study. The agreement pertains to the operational execution of Part 1 of the JASPIS-01 study. The company’s partner, Menarini Group, will fully reimburse all costs associated with the agreement.
From March to September 2024, Ryvu fulfilled conditions for the disbursement of all three tranches of financing from the EIB. In total, Ryvu has now received EUR 22 million from the EIB.
In June 2024, Ryvu concluded a funding agreement with the Polish Agency for Enterprise Development ("PARP") and expects to receive approximately USD 6.6 million (PLN 26.3 million) in grant funding over five years to support its proprietary ONCO Prime discovery platform.
In May 2024, Ryvu obtained the status of Associate Partner within the IPCEI Med4Cure program with its PANACEA-NOVO project – a unique platform for the discovery of new therapeutic targets with potential in the treatment of rare cancers, combined with several early discovery campaigns for innovative drugs. Ryvu expects that potential future grant funding may cover 75-80% of Ryvu’s total project costs, which are estimated to be PLN 142.5 million.
In February 2024, Ryvu announced that it had achieved the second milestone under the license agreement with Exelixis and received a USD 2.0 million (PLN 7.9 million) payment.
UPCOMING INDUSTRY AND INVESTOR EVENTS

Central European Technology Forum – CETEF’24, (Krakow, Poland) November 18-19. Paweł Przewięźlikowski, CEO of Ryvu, will deliver a presentation entitled: "From discovery to a new hope for blood cancer patients".
Life Science Open Space Summit, (Krakow, Poland) November 28-29. Kamil Sitarz, COO of Ryvu, will deliver a presentation entitled: "Building project value through clinical development".
Investor Event: RVU120 Program Progress and Data Update – Thursday, December 12, 10:00 AM CET to discuss the ongoing RVU120 Phase II studies.
Q3 2024 FINANCIAL UPDATE

Cash Position – On September 30, 2024, Ryvu Therapeutics held USD 65.2 million in cash, cash equivalents, and bonds, compared to USD 63.7 million on December 31, 2023. On November 4, 2024, Ryvu Therapeutics held USD 58.1 million in cash, cash equivalents, and bonds.

Operating Revenues – In the first three quarters of 2024, Ryvu recognized total operating revenues (including grants) of USD 18.6 million, compared to USD 11.9 million in corresponding period of 2023.

Operating costs, related primarily to research and development expenditures, excluding the valuation of NodThera shares and non-cash cost of valuation of the Incentive Program for the first three quarters of 2024, amounted to USD 38.2 million, compared to USD 27.4 million for the same period last year.

Net Loss Attributable to Common Shareholders – In the first three quarters of 2024, the net loss attributable to common shareholders, excluding the non-cash cost of the Incentive Program, amounted to USD 18.5 million, compared to USD 13.5 million in the previous year.

On November 7, 2024 Cellenkos Inc., a clinical-stage biotechnology company focused on developing allogeneic, off-the-shelf, T regulatory (Treg) cell therapies for inflammatory disease areas of high unmet needs and autoimmune disorders, reported that its CK0804 Phase 1b study data in myelofibrosis has been selected for oral presentation at the 66th Annual Meeting & Exposition of the American Society of Hematology (ASH) (Free ASH Whitepaper), December 7-10, 2024, in San Diego, California (Press release, Cellenkos, NOV 7, 2024, View Source [SID1234647984]).

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The oral presentation will feature the safety and efficacy data from Cellenkos’ Phase 1b trial evaluating CK0804 in patients with myelofibrosis who have suboptimal responses to prior therapies, including ruxolitinib. CK0804 is an off-the-shelf, allogeneic, non-HLA matched, CXCR4-enriched Treg cell therapy and is administered in fixed dose of 100 million cells infused every 28 days for 6 doses. CK0804 has shown early promise in addressing this high unmet medical need.

Oral Presentation Details (View Source)

Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Advancing MPN Care: Innovative Therapies and Clinical Breakthroughs in Myelofibrosis
Date: Monday, December 9, 2024
Time: 5:00 PM (Presentation)
Location: Manchester Grand Hyatt San Diego, Harbor Ballroom DEFG
Publication Number: 999
Title: A Phase Ib, Open-Label Study of Add-on Therapy with CK0804 in Participants with Myelofibrosis and Suboptimal Response to Ruxolitinib
Submission ID: 211587
The study data to be presented demonstrates the safety and efficacy of CK0804 in patients with myelofibrosis who had previously failed standard treatments. Key findings from the trial include significant improvements in symptom burden, spleen volume reduction, blood transfusion dependence, and systemic inflammatory cytokine levels. Importantly, CK0804 was well-tolerated, with no significant drug-related adverse events observed, aside from one patient with a sulfa drug allergy who had an infusion reaction.

Study Highlights

Nine patients, with a median of two (range, 1-6) prior lines of treatment, were treated with CK0804.
Improvements in fatigue and overall symptom burden were reported by all patients, with four out of six evaluable patients showing spleen volume reduction (SVR).
Two patients who were transfusion-dependent showed a reduction in their monthly need for transfusions by the end of the treatment period.
Longitudinal data indicates sustained benefits, including improvements in hemoglobin levels and inflammatory markers.
Following the initial success of the Phase 1b trial, the Data Safety Monitoring Board has approved an expansion cohort to further explore the safety and efficacy of CK0804. This expansion includes an induction phase of four weekly doses, followed by five monthly doses, and active participant enrollment is ongoing (NCT05423691).

About Myelofibrosis
Myelofibrosis is a rare, chronic, and progressive blood cancer that causes scar tissue to form in the bone marrow, disrupting the production of normal blood cells. Patients with myelofibrosis often experience debilitating symptoms such as fatigue, spleen enlargement, and night sweats. Approximately 16,000 to 18,500 people in the U.S. are living with myelofibrosis, and those who fail to respond adequately to current treatments including ruxolitinib, face limited options and a poor prognosis. Inflammation is a key driver for disease pathogenesis and progression in myelofibrosis.

About CK0804
CK0804 is an investigational, allogeneic, off-the-shelf Treg cell therapy that leverages the CXCR4/CXCL12 axis to suppress inflammatory cytokines implicated in myelofibrosis pathogenesis. CXCR4 enriched Tregs home faster to bone marrow compared to unmanipulated Tregs. Derived from clinical-grade umbilical cord blood and manufactured using Cellenkos’ proprietary CRANE process, CK0804 does not require HLA matching, making it an ideal therapeutic option for patients in need of prompt intervention. The therapy is administered intravenously and can be infused in an outpatient setting.

On November 7, 2024 cTRL Therapeutics, a biotechnology company advancing next-generation cell therapies for solid tumors, reported that it will present new data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting 2024, taking place November 6-10 in Houston, TX (Press release, CTRL Therapeutics, NOV 7, 2024, View Source [SID1234647983]). The company will showcase two poster presentations that underscore the therapeutic potential of circulating tumor-reactive lymphocytes (cTRLs), enabled by the company’s proprietary IsoQore cell isolation platform.

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"We are excited to present data that demonstrate the robust tumor reactivity and therapeutic potential of cTRLs in patients with limited treatment options," said Ruben Rodriguez, PhD, Head of Research at cTRL Therapeutics. "These findings highlight the potential of our IsoQore platform to unlock the therapeutic use of cTRLs, previously thought to be impossible, offering a new way forward for patients with solid tumors."

Poster Presentations:

Abstract 401: Anti-tumor activity of circulating tumor-reactive lymphocytes (cTRLs) isolated from checkpoint-refractory melanoma patients that failed TIL manufacturing.
This poster will present data on cTRLs isolated from melanoma patients who had failed conventional TIL manufacturing. The results demonstrate strong tumor reactivity in cTRLs, with higher frequency of tumor-reactive TCR repertoire compared to TILs, providing a more potent anti-tumor response. The data underscore the potential of cTRLs as a simpler and more effective alternative to traditional cell therapy approaches like TIL therapy.
Poster Presentation: Friday, Nov. 8

Abstract 400: Circulating tumor-reactive lymphocytes (cTRLs) isolated from colorectal cancer (CRC) patients are reactive against autologous tumors and show less exhaustion than tumor-infiltrating lymphocytes (TILs).
This presentation highlights the potential of cTRLs isolated from CRC patients to outperform TILs. The data show that cTRLs exhibit lower levels of exhaustion markers (PD-1, CD39) and higher expression of memory/activation markers (CD27, CD28) and improved functionality and tumor reactivity compared to paired TILs. These findings position cTRLs as a promising new polyclonal T-cell therapy, eliminating the need for surgical tumor resection and expanding patient eligibility.
Poster Presentation: Saturday, Nov. 9
"These data highlight the strong potential of cTRLs as a next-generation approach in cell therapy," said Derrell Porter, M.D., CEO of CTRL Therapeutics. "With a non-invasive blood draw instead of surgical resection, and superior cell fitness compared to traditional cell therapies, cTRLs could offer patients a more accessible, scalable, and effective treatment option. We look forward to sharing these findings with the scientific community at SITC (Free SITC Whitepaper)."

cTRL Therapeutics continues to advance its proprietary IsoQore platform, which isolates and expands high-quality cTRLs from peripheral blood, paving the way for a new era of cell therapies that address key limitations of current modalities.