On November 5, 2024 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported the acceptance of multiple oral and poster presentations, including the first clinical data from a Beam program, at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 7-10, 2024, in San Diego (Press release, Beam Therapeutics, NOV 5, 2024, View Source [SID1234647704]).

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"For people living with severe sickle cell disease, a serious medical condition with reduced life expectancy, stem cell transplant with genotoxic chemotherapy is the only currently available curative option. At Beam, we have a long-term commitment to delivering better treatments for these patients, first with BEAM-101 as a potentially superior autologous cell product, followed by ESCAPE, which seeks to eliminate chemotherapy from the transplant process altogether," said John Evans, chief executive officer of Beam. "Today represents an important milestone toward this vision as we unveil data with our base editing technology across both approaches to treating sickle cell disease. The initial results for BEAM-101 provide emerging clinical validation of base editing and of our preclinical hypothesis that more precise and efficient editing, without double-stranded DNA breaks, can lead to a differentiated product profile, with greater and more uniform induction of fetal hemoglobin, deeper reduction of sickle hemoglobin, and potentially faster engraftment."

"Our proof-of-concept data for ESCAPE in non-human primates demonstrate that base editing could enable antibody conditioning and engraftment for stem cell transplant without chemotherapy, a potential breakthrough in the field of hematology and for patients," said Giuseppe Ciaramella, Ph.D., president of Beam. "Along with the strong translation from preclinical to clinical of our BEAM-101 program, these data reflect the potential of base editing to enable new therapeutic possibilities for people suffering from serious diseases."

BEAM-101 Oral and Poster Presentations

Title: Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises

Abstract: 513

Oral Session: 801. Gene Therapies: Gene Editing and Replacement Therapies for Hemoglobinopathies: From Bench to Bedside

Presentation Time: Sunday, Dec. 8, 2024, at 10 a.m. PT

Location: San Diego Convention Center, Room 30

Presenter: Matthew M. Heeney, M.D., Dana-Farber/Boston Children’s Cancer and Blood Disorders Center

Key Highlights:

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Preliminary data as of a July 2, 2024, data cut from BEACON, a Phase 1/2 single-arm, open-label clinical trial evaluating the safety and efficacy of a single dose of BEAM-101 in patients with sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs).
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Initial safety profile was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation (HSCT).
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In all patients dosed (n=6), there were no ≥Grade 3 adverse events (AEs) or serious AEs related to treatment with BEAM-101.
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One patient died four months after BEAM-101 infusion due to respiratory failure that was determined by the investigator to be likely related to busulfan conditioning and deemed unrelated to BEAM-101. The case was reviewed by the Data Safety Monitoring Committee and the U.S. Food and Drug Administration. Pulmonary complications are a known cause of morbidity and, in rare cases, mortality in patients undergoing myeloablation with chemotherapy, such as busulfan, and stem cell transplantation.
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All patients dosed achieved their target cell dose with either 1 or 2 mobilization cycles (mean: 1.5).
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All 4 patients with ≥1 month of follow-up achieved neutrophil and platelet engraftment at a median of 17 (15–19) and 20 (11–34) days, respectively.
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All 4 patients experienced rapid and robust fetal hemoglobin (HbF) induction by Month 1 (>60%) and corresponding sickle hemoglobin (HbS) reduction (≤36%) in non-transfused blood, which was sustained over time.
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Markers of hemolysis normalized or improved for all 4 patients.
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No VOCs were reported by investigators post-treatment.
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These data support base editing of the HBG1/2 promoters as a therapeutic modality for the treatment of SCD and the ongoing development of BEAM-101.
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The presentation at ASH (Free ASH Whitepaper) will include additional data with more patients and longer follow-up.

Title: Impact of BEAM-101 Treatment on Red Blood Cell Hemoglobin Expression, Rheology and Sickling Properties: Initial data from the BEACON Phase 1/2 study of Autologous CD34+ Base Edited Hematopoietic Stem Cells in Sickle Cell Disease

Abstract: 4957
Poster Session: 801. Gene Therapies: Poster III

Session Time: Monday, Dec. 9, 2024, from 6-8 p.m. PT

Location: San Diego Convention Center, Halls G-H

Presenter: Priya S. Chockalingam, Ph.D., Beam Therapeutics

Key Highlights:

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Preliminary data as of a July 2, 2024, data cut includes exploratory biomarker assessments of red blood cell (RBC) hemoglobin expression, health and function in 3 patients for two or more of the following visits: screening, Month 1, 2, 3 and 6.
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Initial results demonstrated 98-99% of RBCs expressing HbF as early as Month 1, with near complete elimination of RBCs expressing solely HbS post-treatment with BEAM-101.
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The data showed reduction in RBC sickling, comparable to sickle cell trait, reduced cell adhesion and improved hemorheological properties of blood post-treatment with BEAM-101.
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The presentation at ASH (Free ASH Whitepaper) will include additional data with more patients and longer follow-up.

ESCAPE Oral Presentation:

Title: CD117 Antibody Conditioning and Multiplex Base Editing Enable Rapid and Robust Fetal Hemoglobin Reactivation in a Rhesus Autologous Transplantation Model

Abstract: 516

Oral Session: 801. Gene Therapies: Gene Editing and Replacement Therapies for Hemoglobinopathies: From Bench to Bedside

Presentation Time: Sunday, Dec. 8, 2024, at 10:45 a.m. PT

Location: San Diego Convention Center, Room 30

Presenter: Selami Demirci, Ph.D., National Institutes of Health

Key Highlights:

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Study investigated whether Beam’s Engineered Stem Cell Antibody Evasion (ESCAPE) approach, using CD117 monoclonal antibody (mAb) conditioning, could successfully achieve long-term engraftment and induce robust levels of HbF in an immunocompetent host. Researchers used a non-human primate (NHP) model where autologous CD34+ hematopoietic stem and progenitor cells were multiplex edited to target a single epitope on CD117 and the promoter regions of HBG1/2, allowing cells to avoid detection by the conditioning mAb while upregulating HbF.
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Two NHPs were conditioned with different doses (10 mg/kg and 25 mg/kg) of CD117 mAb, instead of busulfan, 7 days prior to transplantation. Post-transplant, further mAb treatments were administered to maintain competitive advantage for the edited cells.
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mAb-based conditioning was well tolerated, with no need for NHP supportive care.
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Both NHPs showed rapid and significant induction of HbF. The percentage of red blood cells expressing HbF (F-cells) reached 61% as early as 8 weeks post-transplant in one NHP and stabilized at ~85% by 35 weeks in both animals. Concurrently, levels of HbF increased rapidly, reaching ~55% at 35 weeks post-transplant in both animals.

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Data suggest that ESCAPE, combined with CD117 mAb conditioning and selection, can achieve long-term engraftment and induce high levels of HbF, offering a promising alternative to traditional genotoxic conditioning in autologous HSCT.
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The presentation at ASH (Free ASH Whitepaper) will include additional data.

BEAM-201 Poster Presentation:

Title: BEAM-201 for the Treatment of Relapsed and/or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia (T-ALL) or T-Cell Lymphoblastic Lymphoma (T-LL): Initial Data from the Phase (Ph) 1/2 Dose-Exploration, Dose-Expansion, Safety, and Efficacy Study of Multiplex Base-Edited Allogeneic Anti CD7 CAR-T-Cells

Abstract: 4838

Poster Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster III

Session Time: Monday, Dec. 9, 2024, from 6-8 p.m. PT

Location: San Diego Convention Center, Halls G-H

Presenter: Caroline Diorio, M.D., FRCPC, FAAP, Children’s Hospital of Philadelphia

Key Highlights:

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Initial data as of a June 11, 2024, data cut in 3 patients treated with BEAM-201 show a safety profile consistent with underlying disease, lymphodepletion and AEs associated with CAR-T therapy.
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A complete response (CRi/CR) was demonstrated in 2 of 3 patients at CAR-T cell doses <200 million. Both patients achieving a CRi/CR were deemed suitable for stem cell transplant following therapy.
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The presentation at ASH (Free ASH Whitepaper) will include additional data with more patients and longer follow-up.

ASH Investor Event Information

Beam will host a live and webcast investor event on Dec. 8, 2024, at 8:00 p.m. PT in San Diego to review the key presentations from this year’s ASH (Free ASH Whitepaper) meeting. The event will be webcast live and can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.beamtx.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On November 5, 2024 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported that Daniel Faga, president and chief executive officer of Anaptys, and/or other members of its senior management team, are scheduled to participate in multiple upcoming investor conferences (Press release, AnaptysBio, NOV 5, 2024, View Source [SID1234647703]):

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Guggenheim’s Inaugural Healthcare Innovation Conference, Boston, MA

Format – Fireside chat and one-on-one investor meetings
Date and Time – Wednesday, Nov. 13, 2024 at 1:30pm ET / 10:30am PT

Stifel 2024 Healthcare Conference, New York, NY

Format – Fireside chat and one-on-one investor meetings
Date and Time – Tuesday, Nov. 19, 2024 at 1:50pm ET / 10:50am PT

Piper Sandler 36th Annual Healthcare Conference, New York, NY

Format – Fireside chat and one-on-one investor meetings
Date and Time – Tuesday, Dec. 3, 2024 at 2:30pm ET / 11:30am PT

7th Annual Evercore HealthCONx Conference, Coral Gables, FL

Format – Fireside chat and one-on-one investor meetings
Date and Time – Wednesday, Dec. 4, 2024 at 1:45pm ET / 10:45am PT
Live webcasts of the fireside chats will be available on the investor section of the Anaptys website at View Source Replays of the webcasts will be available for at least 30 days following the events.

On November 5, 2024 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported financial results for the third quarter ended Sept. 30, 2024 and provided a business update (Press release, AnaptysBio, NOV 5, 2024, View Source [SID1234647702]).

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"We remain confident in the potential best-in-class profiles of our programs targeting BTLA and PD-1 co-inhibitory receptors to drive differentiated results as we approach multiple clinical catalysts and value drivers for Anaptys, including top-line Phase 2b data in AD for ANB032, our BTLA agonist, in December. We’ve also completed enrollment for the Phase 2b trial of rosnilimab, our PD-1 agonist, in RA and are narrowing our guidance for top-line data to February 2025," said Daniel Faga, president and chief executive officer of Anaptys. "Additionally, enrollment in healthy volunteers has commenced for the Phase 1 trial for ANB033, our anti-CD122 antagonist, and we look forward to disclosing the Phase 1b indication in 2025. Looking to the end of the year, we are on track to have four programs in clinical development."

Updates on Wholly Owned ICM Pipeline

ANB032 (BTLA agonist antibody)

Top-line Week 14 data for global Phase 2b trial in moderate-to-severe AD anticipated in December 2024
Enrolled approximately 200 patients in a placebo-controlled trial assessing three dose levels of subcutaneously administered ANB032 (randomized 1:1:1:1) for a 14-week treatment duration and then followed for a six-month off-drug follow-up period on well-established endpoints, including EASI-75 and IGA 0/1
Enrollment included approximately 15% of patients with Dupixent/anti-IL-13 treatment experience
Presented analyses that characterize a BTLA transcriptomic signature in AD at the European Academy of Dermatology and Venerology (EADV) Congress in September 2024
Poster presentation is available here
Rosnilimab (PD-1 agonist antibody)

Top-line Week 12 data for global Phase 2b trial in moderate-to-severe RA anticipated in February 2025
Completed enrollment of approximately 420 patients in a placebo-controlled trial assessing three dose levels of subcutaneously administered rosnilimab (randomized 1:1:1:1) for a 12-week treatment duration on well-established endpoints, including DAS28-CRP, CDAI and ACR20/50/70
At Week 14, rosnilimab-treated patients who achieve low disease activity, defined as CDAI<=10, are eligible to be dosed for an additional 16-week all-active treatment period and then followed for a three-month off-drug follow-up period
Enrollment ongoing for global Phase 2 trial in moderate-to-severe ulcerative colitis (UC)
132-patient placebo-controlled trial assessing two dose levels of subcutaneously administered rosnilimab (randomized 1:1:1) for a 12-week treatment duration on well-established endpoints, including clinical response on modified Mayo score (mMS), clinical remission on mMS and endoscopic remission
Rosnilimab and placebo-treated patients who achieved clinical response on mMS are eligible to continue on their assigned treatment for an additional 12 weeks, while patients on placebo who are non-responders will be crossed over to the high-dose rosnilimab treatment arm, in an all-active treatment period and then followed for a three-month off-drug follow-up period
In October 2024, an optional 26-week, blinded treatment extension period (TEP) was implemented for patients who remain in clinical response at Week 24 in the U.S.; EU implementation anticipated in early 2025
Top-line Week 12 data anticipated in Q1 2026
Presented data evaluating the PD-1 depletion and agonism mechanisms of rosnilimab in vitro with UC patient-derived PBMCs and a mouse model of colitis at the 2024 United European Gastroenterology Week (UEGW) in October 2024
Poster presentation is available here
ANB033 (anti-CD122 antagonist antibody)

Phase 1 trial initiated in healthy volunteers in October 2024
Phase 1b indication to be disclosed in 2025
ANB101 (BDCA2 modulator antibody)

Submitted investigational new drug (IND) application and plan to initiate enrollment for Phase 1 trial in healthy volunteers in Q1 2025
Legacy Clinical-Stage Cytokine Antagonist Programs Available for Out-Licensing

Presented full data from the Phase 3 GEMINI-1 and GEMINI-2 trials of imsidolimab (IL-36R) in generalized pustular psoriasis (GPP) at the EADV Congress in September 2024
Poster presentation is available here
Intend to out-license imsidolimab in 2024
GSK Immuno-Oncology Financial Collaboration

GSK anticipates top-line data in H1 2025 from COSTAR Lung Phase 3 trial comparing cobolimab, a TIM-3 antagonist, plus dostarlimab, a PD-1 antagonist, plus docetaxel to dostarlimab plus docetaxel to docetaxel alone in patients with advanced NSCLC who have progressed on prior anti-PD-(L)1 therapy and chemotherapy
GSK anticipates top-line data in Q4 2024 from the FIRST Phase 3 trial for platinum-based therapy with dostarlimab and niraparib versus platinum-based therapy as first-line treatment of Stage III or IV nonmucinous epithelial ovarian cancer
Cash Runway

Cash and investments of $458.0 million as of September 30, 2024 and reiterating cash runway through year-end 2026
Third Quarter Financial Results

Cash, cash equivalents and investments totaled $458.0 million as of September 30, 2024, compared to $417.9 million as of December 31, 2023, for an increase of $40.1 million due primarily to the $100.0 million underwritten registered direct offering completed in Q3 and $50.0 million received from the Sagard royalty monetization in Q2 offset by operating activities.
Collaboration revenue was $30.0 million and $48.2 million for the three and nine months ended September 30, 2024, compared to $3.3 million and $8.2 million for the three and nine months ended September 30, 2023. The increase in non-cash revenue is due to a $15.0 million commercial milestone earned for annual Jemperli sales exceeding $250.0 million and increased royalties recognized for sales of Jemperli.
Research and development expenses were $42.2 million and $121.3 million for the three and nine months ended September 30, 2024, compared to $30.9 million and $98.8 million for the three and nine months ended September 30, 2023. The increase was due primarily to development costs for rosnilimab, ANB032, ANB033 and ANB101 offset by a decrease in development costs for imsidolimab. The R&D non-cash, stock-based compensation expense was $4.0 million and $10.9 million for the three and nine months ended September 30, 2024 as compared to $2.2 million and $7.7 million in the same period in 2023.
General and administrative expenses were $10.6 million and $32.2 million for the three and nine months ended September 30, 2024, compared to $10.2 million and $31.7 million for the three and nine months ended September 30, 2023. The G&A non-cash, stock-based compensation expense was $4.2 million and $14.9 million for the three and nine months ended September 30, 2024 as compared to $5.6 million and $17.4 million in the same period in 2023.
Net loss was $32.9 million and $123.4 million for the three and nine months ended September 30, 2024, or a net loss per share of $1.14 and $4.46, compared to a net loss of $37.3 million and $121.4 million for the three and nine months ended September 30, 2023, or a net loss per share of $1.41 and $4.49.

On November 5, 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 that it will present clinical data in a poster presentation from its Phase 1 study (abstract #4825) of anitocabtagene autoleucel (anito-cel) in patients with relapsed or refractory multiple myeloma (RRMM); preliminary clinical data in an oral presentation from its iMMagine-1 study (abstract #1031) in patients with RRMM; and a health-related quality of life systematic literature review and meta-analysis (abstract #4721) in patients with RRMM in a poster presentation at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition taking place December 7-10, 2024, in San Diego, California (Press release, Arcellx, NOV 5, 2024, View Source [SID1234647695]). Additionally, an abstract (#6962) describing the treatment patterns and outcomes in triple-class exposed patients with RRMM will be published in a supplemental issue of Blood in November 2024. The company will also have a medical affairs booth (#1615) in Hall E of the San Diego Convention Center.

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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.

Webcast Event:

Arcellx will host a live webcast event with an expert panel of clinicians to discuss the clinical results on Monday, December 9, 2024 at 8:30 p.m. PT. 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.

About Multiple Myeloma

Multiple Myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient’s immune function. Multiple myeloma is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple co-morbidities and toxicities that can quickly escalate and become life-endangering.

About Anitocabtagene Autoleucel (anito-cel)

Anitocabtagene autoleucel (anito-cel, previously ddBCMA) is the first BCMA-directed CAR T-cell therapy to be investigated in multiple myeloma that utilizes Arcellx’s novel and compact binder known as the D-Domain. Anito-cel has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration.

About iMMagine-3, A Global Phase 3 Randomized Controlled Clinical Study

iMMagine-3 is a global Phase 3, randomized controlled study designed to compare the efficacy and safety of anitocabtagene autoleucel (anito-cel) with standard of care in patients with relapsed and/or refractory multiple myeloma (RRMM) who have received one to three prior lines of therapy, including an immunomodulatory drug (lMiD) and an anti-CD38 monoclonal antibody.

iMMagine-3 will enroll approximately 450 adult patients. Prior to randomization, investigator’s choice of SOC regimens include: pomalidomide, bortezomib, and dexamethasone (PVd); daratumumab, pomalidomide, and dexamethasone (DPd); carfilzomib, daratumumab, and dexamethasone (KDd); or carfilzomib and dexamethasone (Kd). Patients in the anito-cel arm will undergo leukapheresis and optional bridging therapy (with the SOC regimen selected by the investigator prior to randomization) followed by lymphodepleting chemotherapy (fludarabine 30 mg/m2/d and cyclophosphamide 300 mg/m2/d for 3 days) and one infusion of anito-cel (115×106 CAR+ T cells) on Day 1.

The primary endpoint is progression-free survival (PFS) per blinded independent review according to the 2016 IMWG uniform response criteria for MM with the hypothesis that anito-cel will prolong PFS compared to SOC. Key secondary endpoints include complete response rate (CR/sCR), minimal residual disease negativity, overall survival, and safety.

The iMMagine-3 study initiated in the second half of 2024 at approximately 130 study sites across North America, Europe, and the rest of the world.

On November 5, 2024 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer and autoimmune disease, reported that it will present new data from the ongoing Phase 1 TRAVERSE trial at the 2024 International Kidney Cancer Symposium (IKCS) and the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) annual meeting (Press release, Allogene, NOV 5, 2024, View Source [SID1234647694]). The trial evaluates ALLO-316, the Company’s first AlloCAR T product candidate for the potential treatment of solid tumors. These presentations will highlight data from 26 heavily pretreated patients with CD70 positive renal cell carcinoma (RCC) and will include details on the diagnostic and treatment algorithm used to mitigate treatment-associated hyperinflammatory response seen in some patients. The ongoing Phase 1 data supported the U.S. Food and Drug Administration’s (FDA) October 2024 decision to grant Regenerative Medicine Advanced Therapy (RMAT) designation for ALLO-316 based on clinical data from the TRAVERSE trial indicating its to address the unmet need for adult patients with advanced or metastatic CD70 positive RCC who have failed multiple standard RCC therapies, including an immune checkpoint inhibitor and a VEGF-targeting therapy.

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"We are looking forward to presenting the most recent Phase 1 data from our ongoing TRAVERSE trial at both SITC (Free SITC Whitepaper) and IKCS," said Zachary Roberts, M.D., Ph.D., EVP of Research & Development and Chief Medical Officer. "There is significant unmet need for these heavily pretreated patients with advanced RCC. In treating their disease, these patients rarely get any form of treatment break as their disease quickly progresses. This data highlights the potential of ALLO-316 to elicit a response from a single infusion, suggesting a breakthrough in allogeneic CAR T therapy for solid tumors. As the Phase 1 data matures, we plan to seek FDA’s input for the next stage of clinical development."

2024 International Kidney Cancer Symposium (IKCS)
Title: TRAVERSE: Updated safety and efficacy of ALLO-316 in advanced/metastatic clear cell renal cell carcinoma (ccRCC)
Presenter: Ritesh Kotecha, M.D., Memorial Sloan Kettering Cancer Center
Session Date and Time: Friday, November 8, 11:50 a.m. ET

The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) Annual Meeting
Title: ALLO-316 in patients with advanced or metastatic clear cell renal cell carcinoma (ccRCC): Updated safety and efficacy from the phase 1 TRAVERSE multicenter study
Presenter: Samer Srour, M.D., The University of Texas MD Anderson Cancer Center
Abstract Number: 322
Date and Time: Saturday, November 9, 12:15-1:45 p.m. CT, 7:00-8:30 p.m. CT

The ongoing Phase 1 TRAVERSE trial is designed to evaluate the safety, tolerability, and activity of ALLO-316 in patients with advanced or metastatic RCC. The development of ALLO-316 continues to advance the scientific understanding and applicability of the Dagger technology as the next-generation allogeneic platform with the ability to maximize the potential of a one-time infusion. In April 2024, the Company announced a $15 million award from the California Institute for Regenerative Medicine (CIRM) to support the ongoing TRAVERSE trial with ALLO-316 in RCC.

About ALLO-316 (TRAVERSE)
ALLO-316, an AlloCAR T investigational product, targets CD70 which is highly expressed in renal cell carcinoma (RCC). CD70 is also selectively expressed in several cancers, creating the potential for ALLO-316 to be developed across a variety of both hematologic malignancies and solid tumors. ALLO-316 utilizes the Dagger technology to optimize CAR T cell expansion and persistence to maximize the potential efficacy in solid tumors with a one-time infusion. The ongoing Phase 1 TRAVERSE trial is designed to evaluate the safety, tolerability, and activity of ALLO-316 in patients with advanced or metastatic clear cell RCC. In March 2022, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to ALLO-316, and in October 2024 the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to ALLO-316 based on its potential to address the unmet need for adult patients with CD70 positive advanced or metastatic RCC who have failed standard RCC therapies.