Noah Stansfield

Celsion’s GEN-1, an IL-12 DNA plasmid vector, was determined to have a satisfactory safety profile and acceptable risk/benefit by an independent Data Safety Monitoring Board (DSMB) that has recommended dosing to continue.

Celsion, a clinical-stage company specializing in DNA-based immunotherapy and next-generation vaccines, made the announcement following a pre-planned interim safety review of patients treated in the multicenter phase 1/2 OVATION 2 study (NCT03393884). Based on the DSMB’s recommendation, the OVATION 2 study will continue to dose patients with 100 mg/m

The DSMB analyzed safety data and concluded that weekly doses of GEN-1 in combination with standard neoadjuvant chemotherapy (NACT) were well-tolerated over a 6-month treatment course. There were no dose-limiting toxicities reported.

Interim efficacy data from the clinical trial has shown that the patients treated with GEN-1 in addition to NACT had improved rates of complete tumor resection compared with the patients in the control arm who only received treatment with NACT. The study primarily aims to achieve a 33% improvement in 

 in the GEN-1 treatment arm. Tumor regression is being categorized via Chemotherapy Response Score (CRS).

"Findings from our OVATION 2 study show a consistent favorable trend in both surgical outcome and tumor response, which is further supported by translational data of the tumor microenvironment," Nicholas Borys, MD, Celsion's executive vice president and chief medical officer, said in a statement.

All participants will undergo a NACT regimen consisting of paclitaxel 175 mg/m

 IV administered over 3 hours followed by carboplatin AUC 6 IV over 1 hour repeated every 3 weeks for 6 cycles. The patients in the experimental arm will additionally receive the 100 mg/m

 dose of GEN-1 on Days 8 and 15 of the first NACT cycle and then on Days 1, 8, and 15 of the subsequent 21-day NACT cycles for a total of 17 treatments.

The trial is open to female patients over the age of 18 who have a suspected histologic diagnosis of epithelial ovarian, fallopian tube, or primary peritoneal carcinoma. Patients who have received prior treatment with GEN-1 or have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to GEN-1 will be excluded from the study. Analysis for PFS will be conducted after all patients have been followed for at least 16 months or a minimum of 80 events have been observed.

GEN-1 is developed with Celsion’s proprietary TheraPlas platform and is encased in a nanoparticle delivery system, which addresses toxicity issues typical of IL-12 administration. The therapy enables IL-12 delivery via cell transfection and local secretion at therapeutic levels.

 Toxicity is further reduced by targeted administration to the tumor location. In addition to 

, studies have indicated potential for its use in treating colorectal and brain cancers.

"Preclinical and clinical data gives us every reason to believe in GEN-1's promise for ovarian cancer patients along with the support from leading medical researchers of the Gynecological Oncology Group (GOG),” Michael H. Tardugno, Celsion's chairman, president, and chief executive officer, added to the statement.

 “FDA Fast Track Designation for GEN-1 in advanced ovarian cancer coupled with the GOG's interest in forging a partnership to develop GEN-1 in ovarian cancer will assist Celsion in its plans for an accelerated registrational program."

Enrollment for the OVATION 2 study is expected to be completed by the third quarter of 2022, and PFS data are expected 12 months after patient enrollment is completed.

1. Celsion reports Data Safety Monitoring Board unanimous recommendation to continue dosing patients in the phase II portion of the OVATION 2 study with GEN-1 in advanced ovarian cancer. News release. Celsion Corporation. June 21, 2022. 

https://firstwordpharma.com/story/5599045

2. GEN-1: Harnessing the potential of IL-12 for targeted immunotherapy. Website. Celsion Corporation. Accessed June 22, 2022.

Articles

The phase 1/2 OVATION 2 study is expected to complete enrollment by the third quarter of this year. 

Gene-Mediated Immunotherapy for Ovarian Cancer Clears Safety Hurdle, Will Continue Dosing  

, Novartis) was shown to be both effective and well-tolerated for children expected to develop spinal muscular atrophy (SMA) type 1 or type 2, according to findings from SPR1NT (NCT03505099), a phase 3 multicenter, single-arm trial that were reported in 2 separate papers published in 

 The data were previously presented at the European Academy for Neurology Virtual Congress in 2021 and the 2022 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference.

All children in both the type 1 (2 copies of the 

) cohorts achieved the primary endpoint of independent sitting

 and nearly all children in both cohorts achieved other age-appropriate milestones including standing and walking.

 None of the children in the study required respiratory support nor nutritional support, and there were no serious treatment-related adverse events observed.

"The robust data from both the 2- and 3-copy SPR1NT cohorts are being published together for the first time, further supporting the significant and clinically meaningful benefit of Zolgensma in 

," said Shephard Mpofu, MD, SVP, chief medical officer, Novartis Gene Therapies, in a statement.

 "When treated with Zolgensma prior to the onset of symptoms, not only did all 29 patients enrolled in SPR1NT survive, but were thriving—breathing and eating on their own, with most even sitting, standing, and walking without assistance."

Of the 44 newborns screened for the SPR1NT study, 14 were excluded for reasons including clinical signs of SMA at screening or immediately before dosing (n=4), peroneal nerve to tibialis anterior CMAP <2 mV (n=4), and elevated anti-AAV9 titers (n=2).

 The type 1 cohort ultimately enrolled 14 presymptomatic infants with genetically confirmed SMA and 2 

 copies (71% female), while the type 2 cohort enrolled 15 infants (60% female) with 3 

 copies. All infants were treated with a single infusion of onasemnogene abeparvovec (median age for type 1 cohort, 21 days; median age for type 2 cohort, 32 days). Results were compared to a matched Pediatric Neuromuscular Clinical Research natural-history cohort (n = 23).

All children in both cohorts began oral prednisolone therapy 1 day prior to onasemnogene abeparvovec infusion (completing a median of 60 days for the type 1 cohort; median 63 days for the type 2 cohort).

 In all, 159 treatment-emergent adverse events (TEAEs) were observed in the type 1 cohort. However, of the 71% in this cohort who experienced a TEAE deemed related to the treatment, none were considered to be serious.

 In the type 2 cohort, 166 TEAEs were reported, with 53% of children in this cohort experiencing a TEAE deemed related to treatment, none of which were considered serious.

 A single Grade 3 hepatotoxicity event occurred in the type 2 cohort that was deemed related to treatment, but resolved with augmented prednisolone.

 Overall, the data related to adverse events from both cohorts were in line with previous data and no new safety signals were identified.

“Limitations of SPR1NT include the relatively small number of participants, the use of the PNCR external comparator group, and the exclusion of children with baseline CMAP <2 mV,” first author Kevin A. Strauss, MD, medical director, Clinic for Special Children, and colleagues noted.

Strauss and colleagues concluded that the results of this study highlight the urgency for universal newborn screening for SMA in order to allow for early identification and intervention.

“[T]reating individuals who demonstrate no symptoms of disease remains controversial,” they wrote in the paper reporting on the type 2 cohort, who typically would show symptoms by the age of 18 months. “SMA offers an example of what can be achieved when newborn screening identifies at-risk infants who can potentially be spared the consequences of severe, debilitating weakness. Children with 3 copies of 

 have a greater likelihood of developing SMA type 2 or type 3, but SPR1NT demonstrates that treating 3-copy children before the appearance of SMA symptoms essentially allows them to grow and develop as normal children. This represents a remarkable evolution in the standard of care for SMA: from a reactive to a proactive paradigm, from a focus on patients who survive to children who thrive.”

The patients enrolled in SPR1NT have since been invited to participate in a long-term follow-up study to continue to collect safety and efficacy data. Updates evaluating motor function gains are planned for publication later this year.

1. Strauss KA, Farrar MA, Muntoni F, et al. Onasemnogeneabeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Nature Medicine. Published online June 17, 2022. doi10.1038/s41591-022-01866-4

2. Strauss KA, Farrar MA, Muntoni F, et al. Onasemnogeneabeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial. Nature Medicine. Published online June 17, 2022.doi: 10.1038/s41591-022-01867-3

3. Novartis announces Nature Medicine publication of Zolgensma data demonstrating age-appropriate milestones when treating children with SMA presymptomatically. News release. Novartis. June 17, 2022. 

https://firstwordpharma.com/story/5597735

All children in the phase 3 SPR1NT study achieved the primary end point of independent sitting. 

Children Treated With Zolgensma for Presymptomatic SMA Achieve Age-Appropriate Motor Milestones

Adoptive cellular therapy targeting the KRAS G12D hot-spot mutation with cells engineered to express a 

 gene resulted in tumor regression in a 71-year-old patient with metastatic 

, according to findings from a case study published in 

An overall objective partial response of 62% was achieved in the regression of metastatic lung lesions at 1-month follow-up, and an overall partial response of 72% in tumor regression was achieved by the 6-month mark. Additionally, the engineered cells made up 2.4% of all circulating T cells at 6-months post-treatment.

“Although the durability of the clinical response in our patient remains to be determined, this case report shows that TCR gene therapy targeting the KRAS G12D hot-spot mutation was able to mediate the regression of metastatic pancreatic cancer” first author Rom Leidner, MD, medical director, Head & Neck Cancer Program, Providence Cancer Center and Earle A Chiles Research Institute, and colleagues wrote in a brief report.

While TCR gene therapy can target intracellular tumor-specific antigens, the investigators noted that it is dependent on the specific HLA genotype of the patient.

 Therefore, the TCR therapy used in this study, which is restricted by HLA-C*08:02, is only applicable for a low percentage of potential patients. HLA-C*08:02 is only expressed in about 11% of black persons and 8% of white persons in the United States and is expressed in even lower percentages in most other racial and ethnic groups.

“However, additional TCRs targeting KRAS G12D and other hot-spot KRAS mutants restricted by different HLA molecules that have been identified could extend TCR gene therapy against mutant KRAS to a larger number of patients,” Leidner and colleagues noted.

 “The immunologic targeting of mutant KRAS with vaccines in patients with pancreatic cancer has also been attempted, but the clinical usefulness of this approach remains unclear.”

A post-treatment biopsy to assess the tumor infiltration by the transduced T cells was not possible due to the rapidness of the tumor regression, also limiting the study.

Prior to infusion, the patient received 600 mg intravenous tocilizumab to prevent cytokine release syndrome followed by intravenous cyclophosphamide. The patient then received a single infusion of 16.2×10

 autologous T cells that had been genetically engineered to express 2 allogeneic HLA-C*08:02–restricted TCRs targeting mutant KRAS G12D. Post-infusion, the patient received intravenous high-dose interleukin-2 (600,000 IU per kilogram; one dose every 8 hours, 5 doses total) to support cell expansion.

After treatment, the patient experienced nausea and myelosuppression, which the investigators noted have been associated with cyclophosphamide preconditioning therapy. The patient also experienced hypotension during the post-treatment course of filgrastim administration. However, the investigators noted that “no toxic effects of the engineered T-cell therapy were observed.”

The investigators also mentioned a similar recent case study with less conclusive results, in which a patient with pancreatic cancer was treated under a separate single-patient investigational new drug application, with autologous T cells engineered to express the 2 HLA-C*08:02–restricted KRAS G12D–reactive TCRs. The cells were manufactured under different in vitro growth conditions without additional immune modulation with tocilizumab.

“Radiographic imaging 1 month after the infusion showed modest regression of the patient’s lung metastases and stable liver lesions. However, despite high levels of T-cell persistence in the blood, the patient had progressive disease and died 6 months after receipt of the therapy,” Leidner and colleagues noted.

The investigators concluded that prospective clinical trials are warranted to determine the therapeutic potential of this therapy in pancreatic cancer and other cancers that express KRAS G12D.

1. Leidner R, Silva NS, Huang H, et al. Neoantigen T-cell receptor gene therapy in pancreatic cancer (Research Summary). N Engl J Med.Published online June 9, 2022. doi:10.1056/NEJMoa2119662.

2. Leidner R, Silva NS, Huang H, et al. Neoantigen T-cell receptor gene therapy in pancreatic cancer. N Engl J Med. 2022;386:2112-9. doi:10.1056/NEJMoa2119662.

The treated patient achieved an overall partial response of 72% in tumor regression.

Case Study: Neoantigen TCR Gene Therapy Yields Metastatic Pancreatic Cancer Regression  

A long-acting human IL-7, NT-I7, fused with hybrid Fc (rhIL-7-hyFc) was shown to enhance the proliferation, persistence, and cytotoxicity of human CAR T cells in xenogeneic mouse models and murine CAR T cells in syngeneic mouse models, leading to long-term tumor-free survival, according to results from a preclinical study recently published in 

 These results indicate that NT-17, which is being developed by NeoImmuneTech, could prove useful in improving suboptimal CAR T-cell activity.

In murine models of both acute myeloid leukemia and B-cell lymphoma, NT-17 significantly prolonged survival by enhancing the efficacy of CAR-T therapy. Additionally, NT-17 allowed for a 5-fold reduction in the number of CAR T-cells needed at the time of infusion to prolong survival.

“Enhancement of CAR T cell activity has the potential to improve patient outcomes by reducing target positive relapse in patients with CD19+ B cell malignancies,” the investigators wrote.

The study utilized sophisticated mouse models of B-cell lymphoma and acute myeloid leukemia and an immune competent syngeneic mouse model of acute promyelocytic leukemia.

 NT-17 was shown to protect CD19-targeting CAR –T-cells from cell death, which promoted their proliferation in the presence of CD19+ tumor cells. While CAR T-cells that expanded in the presence of NT-I7 were less differentiated, it was noted that they possessed equivalent effector cytokine secreting abilities.

“Importantly, we did not see any evidence of toxicity with the combination of mCART19 and rhIL-7-hyFc in the immunocompetent mice,” the investigators noted.

 “Additionally, murine CAR T-cell numbers in immunocompetent mice did not reach the astronomical levels seen with human CAR T-cells in NSG mice after rhIL-7-hyFc. The NSG model provides proof of principle that this reagent is effective on human CAR T-cells in vivo, but the lack of endogenous lymphocytes in these mice is likely responsible for the massive expansion of CAR T-cells, and in human subjects, the kinetics of CAR T-cell expansion is expected to be more akin what was seen in the immunocompetent mice.”

Se Hwan Yang, PhD, president and chief executive officer of NeoImmuneTech, Inc., said in a statement, “Those results demonstrate the broad applicability of NT-I7 for cellular therapy, in addition to its well-documented benefits as a long-acting human IL-7 that has the potential to amplify T cells across the subsets, boost the immune system, and enhance the anti-tumor response in people with hematologic malignancies and solid tumors.”

A multisite phase 1b clinical trial (NCT05075603) is currently underway to pursue the results of this study further, and will investigate NT-I7 with CD19-targeting CAR T-cells for the treatment of relapsed/refractory large B-cell lymphomas. NT-I7 will be administered via intramuscular injection following tisagenlecleucel (tisa-cel; Kymriah, Novartis) infusion. Primary outcome measures include safety and tolerability with end points such as incidence, nature, and severity of adverse events and the maximum tolerated dose. Secondary outcome measures include preliminary anti-tumor activity of NT-I7 with end points such as overall response rate, duration of response, progression-free survival, and overall survival.

1. Kim MY, Jayasinghe R, Devenport JM, et al. A long-acting interleukin-7, rhIL-7-hyFc, enhances CAR T cell expansion, persistence, and anti-tumor activity. Nature Communications. 2022; 13(3296). doi:10.1038/s41467-022-30860-0

2. A study in Nature Communications reports that NeoImmuneTech's NT-I7 enhances CAR-T cell expansion, persistence and anti-tumor activity. News release. NeoImmuneTech, Inc. June 14, 2022. 

https://www.prnewswire.com/news-releases/a-study-in-nature-communications-reports-that-neoimmunetechs-nt-i7-enhances-car-t-cell-expansion-persistence-and-anti-tumor-activity-301567985.html

A multisite phase 1 clinical trial for NT-I7 is underway in patients with relapsed/refractory large B-cell lymphoma. 

NT-I7 Boosts CAR T-Cell Expansion, Persistence, and Anti-Tumor Activity in Preclinical Study

CTX130, a first-in-class CD70-targeting allogeneic CAR-T therapy, was shown to have an acceptable safety profile and to produce clinically meaningful responses in patients with heavily pretreated relapsed/refractory (r/r) T-cell lymphoma (TCL), according to findings from COBALT-LYM (NCT04502446), an ongoing global, multicenter, phase 1 clinical trial.

2022 European Hematology Association Annual Meeting

, June 9-17, 2022, in Vienna, Austria and virtual.

Among the 18 patients who had received 1 of 4 dose levels (DL) of CTX130 (DL1, 3x10

) with at least 28 days of follow-up, there were no cases of graft versus host disease nor any instances of tumor lysis syndrome, and no dose-limiting toxicities.

In terms of clinical responses, a higher percentage of patients responded at higher dose levels, with the overall response rate in DL3 and higher (n=10) being 70%, with 30% of these patients achieving complete remission and 40% achieving a partial response. Furthermore, 90% of patients who received DL3 or higher experienced some clinical benefit, which the investigators defined as stable disease or better response.

Based on these results, the investigators concluded that CTX130 has an acceptable safety profile in patients with heavily pretreated r/r TCL and has the potential to provide clinically meaningful benefit.

"We are very pleased with the preliminary results from our COBALT-LYM trial, which showed efficacy and safety that suggest that CTX130, the first allogeneic CAR-T directed against the novel target CD70, can produce deep responses in patients with relapsed or refractory T cell lymphomas," said Samarth Kulkarni, PhD, chief executive officer, 

 "Additionally, we may be able to further optimize the profile by continuing our consolidation dosing strategy. These data reinforce our belief that engineered cell therapies are the future in our fight against cancer and we are well-positioned to be leaders in this field."

As of December 2021, the ages of the patients enrolled in the study ranged from 39 to 78 (median, 67). All patients had been heavily pretreated with systematic therapies (median, 4) and all patients were refractory to their most recent line of therapy. The study included 8 patients with peripheral TCL and 10 patients with cutaneous TCL. Patients who had previously been treated with any CD70-targeting agents were excluded from participation in the study.

Adverse events included infections, cytokine release syndrome (CRS), and immune effector cell-associated neurotoxicity syndrome (ICANS). However, all cases of CRS and ICANS were grades 1 or 2 and either required no specific intervention or resolved following standard CRS management, and neither the frequency nor severity of CRS increased in patients after being redosed with CTX130. Of note, 1 death occurred but was deemed unrelated to the study drug, and there were no treatment-related deaths during the trial.

"While overall survival in a subset of patients with T cell lymphoma has improved with front-line combination chemotherapy, relapsed or refractory patients continue to have very limited treatment options," said Swaminathan P. Iyer, MD, professor, lead of the T Cell Lymphoma Program, Department of Lymphoma/Myeloma, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, in a statement.

 "The data from the CTX130 trial demonstrate the potential of cell therapies as a new treatment modality for these patients. I am particularly encouraged by the response rates and safety data, which suggest that treatment with CTX130 could elicit clinically meaningful responses, including complete responses, in patients with difficult-to-treat T cell lymphomas."

In addition to the COBALT-LYM study, CTX130 is also being investigated for the treatment of r/r renal cell carcinoma in a phase1 clinical trial called COBALT-RCC (NCT04438083).

1. Iyer SP, Sica RA, Ho PJ, et al. The COBALT-LYM study of CTX130: a phase 1 dose escalation study of CD70-targeted allogeneic CRISPR-Cas9–engineered CAR T cells in patients with relapsed/refractory (r/r) T-cell malignancies. Presented at: 2022 Hybrid Congress of the European Hematology Association (EHA). June 9-12, 2022; Vienna, Austria. Abstract #S262

2. CRISPR Therapeutics presents positiveresults from its phase 1 COBALT-LYM trial of CTX130 in relapsed or refractory T cell malignancies at the 2022 European Hematology Association (EHA) Congress. News release. CRISPR Therapeutics. June 12, 2022. https://firstwordpharma.com/story/5593297

CTX130 is also being investigated for the treatment of r/r renal cell carcinoma in a phase 1 clinical trial called COBALT-RCC (NCT04438083).

CTX130 Shows Acceptable Safety Profile, Clinically Meaningful Responses in R/R T-Cell Lymphoma 

A 5-plus year follow-up study has demonstrated durable efficacy of tisagenlecleucel (tisa-cel; Kymriah, Novartis) in pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL), with 55% of patients still alive after more than 5 years post-treatment and 42% achieving 5-year event-free survival (95%CI, 29-54), according to findings from ELIANA, a phase 2 global multicenter study (NCT02435849) presented at the 

Of the 79 patients who received tisa-cel, 82% achieved complete remission or complete remission with incomplete hematologic recovery within 3 months post-infusion (95% CI, 72-90). The 5-year relapse-free survival rate for these patients was 44% (95% CI, 31-56) and median relapse-free survival was 43 months. Based on these results, the researchers concluded that tisa-cel could be a potentially curative treatment option for patients with r/r B-ALL.

“These data mark a moment of profound hope for children, young adults and their families with relapsed or refractory B-cell ALL, as relapse after five years is rare,” according to Stephan Grupp, MD, PhD, section chief of the Cellular Therapy and Transplant Section, and inaugural director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy at Children's Hospital of Philadelphia (CHOP), said in a statement.

 “Since the approval of Kymriah nearly five years ago, we have been able to offer a truly game-changing option to patients who previously faced a five-year survival rate of less than 10 percent.”

The ages of patients involved in the study ranged from 3 to 24 years (median, 11). All patients had been pretreated with 1 to 8 prior lines of therapy (median, 3), and 61% of the patients had a history of prior stem cell transplant. The 5-year overall survival rate among all study participants was 55% (95% CI, 43-66). Notably, there was no significant difference observed in any efficacy end point between participants age 18 or older who received tisa-cel versus those younger than 18. The researchers noted that 82% of the patients received IVIG at some point post-infusion.

Commonly reported adverse events occurring more than 1 year post-infusion included infection (20%) and cytopenias (6%). Investigators noted that 14% of patients experienced cytopenias lasting more than 1 year but not more than 5. 

Additionally, the investigators noted that no new or unexpected adverse events were reported during long term follow-up.

“These results strengthen our confidence in CAR-T cell therapies as a truly transformative and paradigm-shifting advance in cancer care, as well as our commitment to continue developing this technology with next-generation platforms,” stated Jeff Legos, executive vice president, global head of oncology & hematology development, at Novartis.

Additional studies from Novartis’ CAR-T program presented at the 2022 EHA Congress include updates from the first-in-human dose escalation trials with PHE885 in adults with r/r multiple myeloma

 and with YTB323 in adults with r/r diffuse large B-cell lymphoma.

1. Rives S, Maude SL, Hiramatsu H, et al. Tisagenlecleucelin pediatric and young adult patients (Pts) with relapsed/refractory (R/R) B-Cell acute lymphoblastic leukemia (B-ALL): Final analyses from the ELIANA study. Presented at: 2022 Hybrid Congress of the European Hematology Association (EHA). June 9-12, 2022; Vienna, Austria. Abstract #S112

2. Novartis five-year Kymriah data show durable remission and long-term survival maintained in children and young adults with advanced B-cell ALL. News release. Novartis. June 12, 2022.https://www.novartis.com/news/media-releases/novartis-five-year-kymriah-data-show-durable-remission-and-long-term-survival-maintained-children-and-young-adults-advanced-b-cell-all

3. Sperling AS, Nikiforow S, Derman B, et. al. Phase I study data update of PHE885, a fully human BCMA-directed CAR-T cell therapy manufactured using the T-Charge™ platform for patients with relapsed/refractory (r/r) multiple myeloma (MM). Presented at: 2022 Hybrid Congress of the European Hematology Association (EHA). June 9-12, 2022; Vienna, Austria. Abstract #P1446

4. Munshi, N. et. al. Phase II study of PHE885, a B-cell maturation antigen-directed chimeric antigen receptor t-cell therapy, in adults with relapsed/refractory (r/r) multiple myeloma (MM). Presented at: 2022 Hybrid Congress of the European Hematology Association (EHA). June 9-12, 2022; Vienna, Austria. Abstract #PB1983

5. Dickinson, M. et. al. Phase I study of YTB323, a chimeric antigen receptor (CAR)-T cell therapy manufactured using T-Charge™, in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL). Presented at: 2022 Hybrid Congress of the European Hematology Association (EHA). June 9-12, 2022; Vienna, Austria. Abstract #S212

Among treated patients, 42% achieved 5-year event free survival.  