Sana Biotechnology Presentations at 2021 ASH Annual Meeting Highlight Progress with Platforms and CAR T Cell Programs

On December 12, 2021 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported that data at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place from Saturday, December 11 to Tuesday, December 14, 2021, which highlighted further progress with key technologies supporting Sana’s in vivo and ex vivo CAR T cell programs (Press release, Sana Biotechnology, DEC 12, 2021, View Source [SID1234596821]).

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"The data presented at ASH (Free ASH Whitepaper) showcase the progress we are making with Sana’s CAR T cell programs," said Terry Fry, M.D., Sana’s Head of T Cell Therapeutics. "The hypoimmune and fusogen technologies are designed to address significant challenges that lead to sub-optimal patient outcomes and prevent widespread utilization of cell and gene therapies, including cell persistence and cell-specific delivery. We continue to move these potential therapies toward clinical trials in patients, with a goal of filing two INDs as early as next year."

On Saturday, December 11, Sonja Schrepfer, M.D., Ph.D., Sana’s Head of Hypoimmune Platform, presented a poster (Abstract 1690) titled "Engineered hypoimmune allogeneic CAR T cells exhibit innate and adaptive immune evasion even after sensitization in humanized mice and retain potent anti-tumor activity." Data demonstrated continued progress with Sana’s hypoimmune allogeneic CAR T cell platform, showing in murine models that these gene-modified CAR T cells targeting CD19 can evade both the innate and adaptive immune systems without any evidence of a change in their ability to eliminate leukemia. This immune evasion was present in naïve subjects as well as in sensitized subjects that had previously rejected non-hypoimmune CAR T cells. In the study, the hypoimmune allogeneic CD19 CAR T cells did not induce activation of the adaptive immune system, T cells or B cells, in the treated subjects (p<0.0001 when compared to non-modified CD19 CAR T cells), and also evaded the subjects’ innate immune responses. These findings are an important step toward the possibility of "off-the-shelf" allogeneic CD19 CAR T cells that persist without immunosuppression, including in patients that have previously been treated with a CAR T therapy.

On Sunday, December 12, Terry Fry, M.D., presented a poster (Abstract 2769) titled "In vivo delivery of a CD20 CAR using a CD8-targeted fusosome in Southern pig-tail macaques (M. nemestrina) results in B cell depletion." The presentation outlined the potential to deliver a CAR gene to make CAR T cells in vivo. B cell depletion in these healthy non-human primates is used as a surrogate marker for an anti-tumor effect against B cell malignancies such as leukemia and lymphoma. Following the infusion of the CD8a-targeted fusosome carrying the gene for an anti-CD20 CAR into macaques, B cells were meaningfully reduced in 4 of 6 animals after 7 to 10 days. Scientists found the anti-CD20 CAR transcripts via measurements of mRNA expression in spleen cells isolated from treated animals; conversely, no expression was detected in tissues from control animals. Subjects in this study received no lymphodepleting chemotherapy. Additionally, the fusosome treatment was well-tolerated in all animals with no evidence of adverse effects. These findings suggest that the fusosome technology represents a novel therapeutic opportunity to treat patients with B cell malignancies, with the potential for in vivo delivery of the CAR gene to CD8 T cells.

On Sunday, December 12, Sana Scientist Christie Ciarlo, Ph.D., presented a poster (Abstract 2942) titled "CD4-targeted fusosomes are capable of transducing resting T helper cells to generate highly potent CAR T cells." The presentation highlighted the ability of select fusosomes to effectively target the correct cells and to deliver an integrating CAR payload that can develop CAR T cells in vivo. CD4-targeted CD19 CAR fusosomes efficiently transduced activated T cells (34% ± 1.5% CD4+CAR+; 0.54 ± 0.18 c/dg) and resting T cells (20% ± 0.5% CD4+CAR+; 0.28 ± 0.14 c/dg). The data showed that these fusosomes were specific to certain T cells based on their functionality and also that they could deliver their payloads to helper T cells without activation, opening up new potential pathways for in vivo cell therapies. Investigators concluded that targeting the CD4 co-receptor through in vivo delivery of a genetic payload can produce potent and functional CAR T cells, with the potential to target certain cancers.

MorphoSys presents latest data from the Phase 2 MANIFEST Study evaluating the potential of pelabresib in the treatment of myelofibrosis

On December 12, 2021 MorphoSys AG (FSE: MOR; NASDAQ: MOR) reported the latest data from the ongoing MANIFEST study, an open-label, Phase 2 clinical trial of pelabresib, an investigational BET inhibitor, in patients with myelofibrosis, a rare bone marrow cancer for which only limited treatment options are available (Press release, MorphoSys, DEC 12, 2021, View Source [SID1234596820]). These latest results, which included more patients and longer-term follow-up than previously reported data, suggest the potential of pelabresib in the treatment of myelofibrosis. These findings were presented during poster and oral sessions at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2021), held December 11 – 14, 2021 in Atlanta, Georgia and virtually.

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"These data reconfirm previously published results and reinforce the role pelabresib may play, if approved, in overcoming some of the challenges we face in treating myelofibrosis," said Malte Peters, M.D., MorphoSys Chief Research and Development Officer. "We are further exploring the effectiveness and safety of pelabresib as a first-line treatment for myelofibrosis in MANIFEST-2, an ongoing Phase 3 study. The latest results reaffirm our confidence in the MANIFEST-2 study, and we look forward to sharing findings from this trial once they become available."

At ASH (Free ASH Whitepaper) 2021, the latest data evaluating pelabresib as a first-line combination with ruxolitinib – the current standard of care – for patients with myelofibrosis who had not previously been treated with a JAK inhibitor (JAK inhibitor-naïve) were presented. As of September 10, 2021, the data cut-off, a total of 84 JAK inhibitor-naïve patients have been enrolled and received the combination. The data showed 68 percent (n=57) of patients treated with the combination achieved a >=35 percent reduction in spleen volume (SVR35) from baseline at week 24 and 60 percent (n=47) maintained SVR35 at week 48. Most patients also saw their symptoms reduced, with 56 percent (n=46) achieving >=50 percent reduction in total symptom score (TSS50) from baseline at week 24. At the time of the data cut-off, 53 patients (63 percent of the 84 patients) were still on treatment. No new safety signals were identified in the study. The most common hematologic adverse events were thrombocytopenia (12 percent, grade 3/4) and anemia (34 percent, grade 3/4). Non-hematological events included dyspnea (5 percent, grade 3) and respiratory tract infections (8 percent, grade 3/4).

Additionally, analyses from an exploratory endpoint presented at ASH (Free ASH Whitepaper) 2021 showed a reduction of megakaryocyte clustering in bone marrow and correlation with spleen volume reduction. Megakaryocytes are the cells in the bone marrow responsible for making platelets, and the clustering of these cells are one of the signs of myelofibrosis. The exploratory data, which require further evaluation, suggest the potential pelabresib may have in changing the course of myelofibrosis treatment, if approved.

"In my opinion, a challenge in treating myelofibrosis is knowing that despite available treatment options the disease will ultimately progress in the majority of patients diagnosed," said Srdan Verstovsek, M.D., Ph.D., professor of medicine and hematologist-oncologist at the MD Anderson Cancer Center and a MANIFEST investigator. "Identifying new, first-line treatment options will improve physicians’ ability to better manage the disease from the time of diagnosis. These latest data, although early in the investigational process, suggest that by combining pelabresib and ruxolitinib, we may have the potential to enhance the current standard of care in the first-line treatment of myelofibrosis."

Additional data from Arm 1 of the MANIFEST study were also presented in an oral presentation at ASH (Free ASH Whitepaper) 2021. In Arm 1, pelabresib is being evaluated as a monotherapy in patients with advanced myelofibrosis who are ineligible to receive, intolerant of, or refractory to JAK inhibitors, a population with very limited therapeutic options. Patients were divided into two cohorts, transfusion-dependent (TD) and non-transfusion-dependent (non-TD). For the TD cohort, the primary endpoint was conversion to transfusion independence (TI) for 12 consecutive weeks. In the non-TD cohort, the primary endpoint was SVR35 at week 24. At week 24, 11 percent (n=7) of patients reached SVR35. In addition, we observed 31 percent of patients had a spleen volume reduction of 25 percent or more (n=20) at week 24. Across all cohorts, 28 percent (n=18) of patients achieved TSS50. No new safety signals were identified in the study. The most common hematologic adverse events were thrombocytopenia (23 percent, grade 3/4) and anemia (15 percent, grade 3). Non-hematological events included diarrhea (6 percent, grade 3) and respiratory tract infections (5 percent, grade 3).

About Pelabresib
Pelabresib (CPI-0610) is an investigational selective small-molecule designed to promote anti-tumor activity by inhibiting the function of bromodomain and extra-terminal domain (BET) proteins to decrease the expression of abnormally expressed genes in cancer. Pelabresib is currently being investigated as a treatment for myelofibrosis and has not yet been evaluated or approved by any regulatory authorities.

About MANIFEST
MANIFEST is an open-label, Phase 2 clinical trial of pelabresib (CPI-0610) in patients with myelofibrosis (MF), a rare cancer of the bone marrow that disrupts the body’s normal production of blood cells.

Constellation Pharmaceuticals, an affiliate of MorphoSys, is evaluating pelabresib in combination with ruxolitinib in JAK-inhibitor-naïve MF patients (Arm 3), with a primary endpoint of the proportion of patients with a >=35% spleen volume reduction from baseline (SVR35) after 24 weeks of treatment. Constellation Pharmaceuticals is also evaluating pelabresib either as a monotherapy in patients who are resistant to, intolerant of, or ineligible for ruxolitinib and no longer on the drug (Arm 1) or as add-on therapy in combination with ruxolitinib in patients with a suboptimal response to ruxolitinib or MF progression (Arm 2). Patients in Arms 1 and 2 are being stratified based on transfusion-dependent (TD) status. The primary endpoint for the patients in cohorts 1A and 2A, who were TD at baseline, is conversion to transfusion independence for 12 consecutive weeks. The primary endpoint for patients in cohorts 1B and 2B, who were not TD at baseline, is the proportion of patients with a >=35% spleen volume reduction from baseline after 24 weeks of treatment.

ALX Oncology Announces Initial Data from ASPEN-02, the Ongoing Phase 1 / 2 Study of Evorpacept in Combination with Azacitidine, Demonstrating Safety and Preliminary Activity in Patients with Myelodysplastic Syndrome

On December 12, 2021 ALX Oncology Holdings Inc., ("ALX Oncology") (Nasdaq: ALXO), a clinical-stage immuno-oncology company developing therapies to block the CD47 checkpoint pathway, reported the presentation of initial clinical data from its ongoing trial evaluating evorpacept in combination with azacitidine for the treatment of patients with previously untreated higher-risk ("HR") or relapsed or refractory ("r/r") myelodysplastic syndrome ("MDS"). The new results, shared in a poster at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting [Abstract #2601], show that the combination of evorpacept and azacitidine is active and well tolerated (Press release, ALX Oncology, DEC 12, 2021, View Source [SID1234596819]). As of October 25, 2021, 22 patients with either previously untreated HR or r/r MDS have been treated with evorpacept in the Phase 1 dose escalation part of the study, administered at 20 mg/kg or 30 mg/kg once every 2 weeks ("Q2W") or 60 mg/kg once every 4 weeks ("Q4W") together with standard dosing of azacitidine. Median follow-up is 3.4 months, and accrual is ongoing.

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Evorpacept in combination with azacitidine was well tolerated (N=22) with no dose limiting toxicities, no observed treatment related serious adverse events, and a maximum administered dose of 60 mg/kg Q4W.
In 6 previously untreated HR MDS response-evaluable patients, 3 patients achieved an objective response ("OR") (2 complete response ("CR"), 1 marrow CR), and 2 patients achieved stable disease ("SD"). Two out of 4 transfusion dependent patients achieved transfusion independence on study.
Among 5 previously untreated HR MDS patients with TP53 mutation and complex cytogenetic abnormalities, 3 achieved an OR (2 CR and 1 marrow CR).
Five of 9 patients with response-evaluable relapsed or refractory MDS that had progressed upon prior hypomethylating agents achieved an OR (5 marrow CRs). In addition, 2 patients achieved SD.
"Evorpacept’s preliminary clinical activity seen in patients with a difficult to treat subset of MDS including disease with TP53 mutation, poor risk cytogenetics, and progression on prior hypomethylating agent regimens, is encouraging," said Guillermo Garcia-Manero M.D., Professor, Department of Leukemia, at MD Anderson Cancer Center, Houston, TX. "Additionally, evorpacept’s favorable initial tolerability profile in combination with azacitidine suggests it may be safely added without worsening cytopenias, which is particularly notable for this patient population."

"The initial tolerability and activity of evorpacept seen in ASPEN-02 further support CD47 as a relevant therapeutic target in patients with MDS," said Sophia Randolph M.D., Ph.D., Chief Medical Officer, ALX Oncology. "Evaluation of evorpacept in our myeloid malignancy program including studies in both MDS and acute myeloid leukemia is built on a strong scientific rationale and we are pleased to now also see initial clinical data supporting its role in enhancing the innate immune anti-cancer response."

Conference Call on December 13th at 8:00 a.m. EST

ALX Oncology will host a conference call on Monday, December 13, 2021 at 8:00 a.m. EST to further discuss the initial MDS data from ASPEN-02. In addition to ALX Oncology’s executive management team, Dr. Guillermo Garcia-Manero, Professor, Department of Leukemia, at MD Anderson Cancer Center, Houston, TX will be featured on the call to discuss the emerging clinical data in MDS patients.

To access the conference call, please dial (844) 467-7655 (U.S./Canada) or (409) 983-9840 (international) at least 10 minutes prior to the start time and refer to conference ID 7598031. Presentation slides will be available to download under "News & Events" (see "Events") in the Investors section of the ALX Oncology website at www.alxoncology.com.

Phase 3 Data Demonstrate TIBSOVO® (ivosidenib tablets) in Combination with Azacitidine Significantly Improves Event-Free Survival and Overall Survival in Patients with Previously Untreated IDH1-mutated Acute Myeloid Leukemia

On December 11, 2021 Servier, a growing leader in oncology committed to bringing the promise of tomorrow to the patients we serve, reported Phase 3 data demonstrating that TIBSOVO (ivosidenib tablets) in combination with the chemotherapy azacitidine significantly improved event-free survival (EFS) and overall survival (OS) compared to azacitidine plus placebo in adults with previously untreated IDH1-mutated acute myeloid leukemia (AML) who are not candidates for intensive chemotherapy (Press release, Servier, DEC 11, 2021, View Source [SID1234596887]). These data from the global AGILE study will be presented in an oral session on Monday, December 13, 2021 from 2:45 – 4:15 PM ET, Abstract #697 and featured in the official press program during the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

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Treatment with TIBSOVO in combination with azacitidine demonstrated a statistically significant improvement in EFS (hazard ratio [HR] = 0.33, 95% CI 0.16, 0.69, 1-sided P = 0.0011 1,2). In addition, the combination of TIBSOVO with azacitidine showed a statistically significant improvement in OS (HR = 0.44 [95% CI 0.27, 0.73]; 1-sided P = 0.0005), with a median OS of 24.0 months in the ivosidenib + azacitidine arm vs 7.9 months in the placebo + azacitidine arm.

"These significant findings from the AGILE Phase 3 study for TIBSOVO bolster our growing body of evidence supporting the rationale to target IDH1 mutations early in blood cancers like acute myeloid leukemia," said Susan Pandya, M.D., Vice President Clinical Development & Head of Cancer Metabolism Global Development Oncology & Immuno-Oncology, Servier Pharmaceuticals. "Up to 10 percent of patients with AML have mutations in the IDH1 enzyme, and current treatment options are limited, especially for those who are newly diagnosed and are not eligible for intensive chemotherapy."

Additional Study Results
Investigators reported on results of key secondary endpoints of the AGILE trial including:

Complete remission (CR) rate was 47.2% (n=34/72) for TIBSOVO in combination with azacitidine vs. 14.9% (n=11/74) for placebo plus azacitidine (p < 0.0001).
CR + complete remission with partial hematologic recovery rate (CR + CRh rate) was 52.8% (n=38/72) for TIBSOVO in combination with azacitidine vs. 17.6% (n=13/74) for placebo plus azacitidine (p < 0.0001).
Objective response rate (ORR) was 62.5% (n=45/72) for TIBSOVO in combination with azacitidine vs. 18.9% (n=14/74) for placebo plus azacitidine (p < 0.0001).
"We are excited about the potential to bring a new treatment option to patients with previously untreated IDH1-mutated AML. This further extends the significant clinical benefit for patients with acute myeloid leukemia and IDH1 mutations," said Patrick Therasse, M.D., Ph.D., Vice President, Head of Late Stage and Life Cycle Management in Oncology and Immuno-Oncology Therapeutic Area, Servier Group.

Acute myeloid leukemia is a rapidly progressing type of cancer, and the prognosis is often poor," said Stephane De Botton, M.D. Ph.D., Principle Investigator and Head of Multidisciplinary Hematology Committee at the Institut Gustave Roussy, Villejuif, France. "Our goal with treatment is to prolong overall survival, and the impressive clinical benefit following treatment with TIBSOVO in combination with azacitidine is incredibly promising for these patients with previously untreated IDH1-mutated acute myeloid leukemia."

Common all-grade adverse events (AEs) occurring in more than 20 percent of patients receiving TIBSOVO in combination with azacitidine vs. placebo plus azacitidine were nausea (42.3% vs. 38.4%), vomiting (40.8% vs 26.0%), diarrhea (35.2% vs 35.6%), pyrexia (33.8% vs 39.7%), anemia (31.0% vs 28.8%), febrile neutropenia (28.2% vs 34.2%), thrombocytopenia (28.2% vs 20.5%), neutropenia (28.2% vs 16.4%), constipation (26.8% vs 52.1%) and pneumonia (23.9% vs 31.5%).

The AGILE study has halted further enrollment due to compelling efficacy data for TIBSOVO.

Servier is in discussions with regulatory health authorities regarding submissions to expand the currently approved indications for TIBSOVO.

TIBSOVO[*] is currently approved in the U.S. as monotherapy for the treatment of adults with IDH1-mutant relapsed or refractory acute myeloid leukemia (AML), and for adults with newly diagnosed IDH1-mutant AML who are ≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy. Recently, TIBSOVO was approved as a first and only targeted therapy for patients with previously treated IDH1-mutated cholangiocarcinoma.

About NCT03173248 AGILE Phase 3 AML Trial
The AGILE trial is a global, Phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial designed to evaluate the efficacy and safety of TIBSOVO in combination with azacitidine compared with placebo in combination with azacitidine, in adults with previously untreated IDH1-mutated acute myeloid leukemia (AML) who are not candidates for intensive chemotherapy (≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy). The study’s primary endpoint is EFS, defined as the time from randomization until treatment failure, relapse from remission, or death from any cause, whichever occurs first. Treatment failure is defined as failure to achieve complete remission (CR) by Week 24.

Other key secondary endpoints included complete remission rate (CR rate), defined as the proportion of participants who achieve a CR; overall survival (OS), defined as the time from date of randomization to the date of death due to any cause; CR and complete remission with partial hematologic recovery (CRh) rate, defined as the proportion of participants who achieve a CR or CRh; and objective response rate (ORR), defined as the rate of CR, CR with incomplete hematologic recovery (CRi) (including CR with incomplete platelet recovery [CRp]), partial remission (PR), and morphologic leukemia-free state (MLFS).

About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow marked by rapid disease progression and is the most common acute leukemia affecting adults with approximately 20,000 new cases in the U.S., and 43,000 cases in Europe each year3,4. The majority of patients with AML eventually relapse. Relapsed or refractory AML has a poor prognosis5. The five-year survival rate is approximately 27%3. For 6 to 10 percent of AML patients, the mutated IDH1 enzyme blocks normal blood stem cell differentiation, contributing to the genesis of acute leukemia6.

New Data from Phase 3 GLOW Study Show Fixed-Duration Treatment with IMBRUVICA® (ibrutinib) Plus Venetoclax Demonstrated Deeper and Sustained Undetectable Minimal Residual Disease Outcomes in First-Line Chronic Lymphocytic Leukemia

On December 11, 2021 The Janssen Pharmaceutical Companies of Johnson & Johnson reported new data from two studies evaluating the efficacy and safety of IMBRUVICA (ibrutinib) plus venetoclax (I+V) as a potential fixed-duration treatment in adult patients with previously untreated chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) (Press release, Johnson & Johnson, DEC 11, 2021, View Source [SID1234596886]). These data were both featured today during the American Society of Hematology (ASH) (Free ASH Whitepaper) 2021 Annual Meeting. New secondary endpoint data from the Phase 3 GLOW study (NCT03462719) showed that fixed-duration treatment with I+V resulted in undetectable minimal residual disease (uMRD) responses that were deeper compared to patients treated with chlorambucil plus obinutuzumab (Clb+O), and an additional analysis showed that uMRD responses were better sustained during the first year post-treatment.1

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Updated results from the Phase 2 CAPTIVATE study (NCT02910583) of the same investigational regimen, now with a median 38 months of follow-up, further demonstrated sustained uMRD and disease-free survival (DFS). There were no new MRD relapses, clinical progressions or deaths with an additional year of study follow-up in patients with confirmed uMRD following 12 cycles of combined I+V who were randomized to placebo or continued IMBRUVICA.2

"GLOW and CAPTIVATE are part of a comprehensive development program continuing to evaluate the potential of IMBRUVICA-based therapy in patients with previously untreated CLL with various needs and risk factors, including those with high-risk disease," said Craig Tendler, M.D., Global Head of Late Development, Diagnostics and Medical Affairs, Hematology & Oncology, Janssen Research & Development, LLC. "With data from these two studies showing patients can achieve deep responses with this novel IMBRUVICA plus venetoclax combination, we believe this all-oral, once-daily, fixed-duration regimen offers patients the potential for treatment-free remissions and physicians the flexibility to use IMBRUVICA alone or as a combination therapy to meet the different goals and needs of patients."

Data on MRD Outcomes After Fixed-Duration IMBRUVICA Plus Venetoclax from the GLOW Study (Abstract #70)

The Phase 3 GLOW study is a randomized, open-label trial which evaluated the efficacy and safety of first-line, fixed-duration I+V vs. Clb+O in elderly patients (≥65 years of age) with CLL/SLL, or patients ages 18-64 with a cumulative illness rating scale (CIRS) score of greater than six or creatinine clearance less than 70 mL/min, without del(17p) or known TP53 mutations.1 Patients in the study were randomized to receive either I+V (n= 106) or Clb+O (n=105).1 Previously reported data were presented at the 2021 European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress and showed that the study met its primary endpoint of progression-free survival (PFS) as measured by an independent review committee (IRC).3

The prespecified secondary endpoint was rate of uMRD (uMRD < 10-4). MRD was evaluated via next-generation sequencing (NGS) and reported with cutoffs of < 10-4 and < 10-5.1 Rate of uMRD was reported at three and 12 months after end of treatment in both study arms.1

The data presented at ASH (Free ASH Whitepaper) demonstrated deeper responses at end of treatment and better sustained uMRD responses during the first year post-treatment with all-oral, once-daily fixed-duration I+V vs. Clb+O.1 Further, responses were proportionally deeper at the level of < 10-5 in the I+V arm vs. Clb+O arm in both peripheral blood (PB) and bone marrow (BM).1

"The GLOW study combines two highly active blood cancer treatments that act in a synergistic fashion by complementary mechanisms to deliver superior progression-free survival in the first-line treatment of CLL," said Arnon Kater†, M.D., Ph.D., Deputy Head of Hematology, Amsterdam University Medical Centers, University of Amsterdam and Chairman of the HOVON CLL Working Group, the Netherlands and principal study investigator. "These latest results show the potential to provide treatment-free remissions for patients through robust disease clearance in lymphoid tissue, blood and bone marrow, and early sustainability of those responses after stopping treatment."

GLOW Results:

With updated median follow-up of 34.1 months, the 30-month PFS was 80.5 percent with I+V vs. 35.8 percent for Clb+O.1
Rates of uMRD < 10-5 were higher with I+V vs. Clb+O in BM (40.6 percent vs. 7.6 percent) and in PB (43.4 percent vs. 18.1 percent).1
With I+V, deep responses < 10-5 were seen in patients with unmutated IGHV CLL, and depth of response was mirrored in PB (49.1 percent) and BM (45.5 percent).1
An additional analysis evaluated sustainability of uMRD response between three and 12 months following end of treatment; 80.4 percent of patients with I+V had sustained uMRD < 10-5 vs. 26.3 percent with Clb+O.1
PFS rate during the first-year post-treatment was sustained >90 percent with I+V, independent of BM or PB MRD status three months after end of treatment.1
Additional follow-up is warranted to confirm the long-term impact of MRD status on PFS.1
Data from the MRD Cohort of the Phase 2 CAPTIVATE (PCYC-1142) Study (Abstract #68)

The Phase 2 CAPTIVATE trial evaluated adult patients younger than 70 years, including patients with high-risk disease, in two cohorts: an MRD-guided cohort where treatment duration is guided by the patient’s MRD status after 12 cycles of combination I+V therapy; and a fixed-duration cohort where all patients stop therapy after 12 cycles of the combination, regardless of MRD status.2 The primary endpoints of the study included MRD negative response rate, DFS, and complete response rate. Data from the primary analysis from both the fixed-duration and MRD-guided cohorts were previously reported.4,5 Patients with high-risk disease included unmutated IGHV (60 percent of patients), del(17p)/TP53 mutation (20 percent), complex karyotype (19 percent), and del(11q) without del(17p) (17 percent). Patients in the MRD-guided cohort (n=164; median age, 58 years) who achieved uMRD [defined as having uMRD (<10–4 by 8-color flow cytometry) serially over at least three cycles and uMRD in both PB and BM with combination therapy], were randomized in a double-blinded fashion to continue treatment with IMBRUVICA monotherapy or placebo until disease progression.2 Patients in the MRD-guided cohort who did not achieve uMRD following 12 cycles of combination I+V therapy were randomized to continue IMBRUVICA monotherapy or the combination.2

DFS was defined as freedom from MRD relapse (≥10–2 confirmed on two separate occasions) and without progressive disease or death starting from randomization after 15 cycles of treatment. The two-year DFS rates post-randomization with time-limited treatment (randomized to placebo) was maintained at 95 percent with an additional year of study follow-up.2 There were no new MRD relapses, disease progressions, or deaths in patients with confirmed uMRD treated with placebo or IMBRUVICA.2 Early data suggest that patients who progress after time-limited treatment with I+V have the potential to be successfully retreated with single-agent IMBRUVICA.2

Additionally, the estimated 36-month PFS rates were 95.3 percent with placebo and 100 percent with IMBRUVICA (95 percent Confidence Interval [CI], 4.7 percent difference, -1.6–10.9, overall log-rank P=0.1573); placebo 82.7–98.8, IMBRUVICA 100–100).2 Ultimately, these results in patients randomized to placebo following an initial 12 cycles of the I+V combination support the potential for treatment-free remission with first-line, fixed-duration I+V, an all-oral, once-daily regimen. Among 12 patients who progressed after fixed-duration treatment, nine patients with available responses all had a partial response to single-agent IMBRUVICA with limited follow-up; three have pending responses.2

With a median study follow-up of 38 months, the safety profile of the I+V regimen in CAPTIVATE was consistent with known safety profiles of IMBRUVICA and venetoclax.2 The most common AEs of any Grade 13-24 months post-randomization were arthralgia (29 percent I+V; 22 percent IMBRUVICA monotherapy) and upper respiratory tract infection (20 percent I+V; 15 percent IMBRUVICA monotherapy).2 Grade ≥3 adverse events (AEs) were infrequent across randomized arms with the exception of neutropenia.2

About IMBRUVICA
IMBRUVICA (ibrutinib) is a once-daily oral medication that is jointly developed and commercialized by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company. IMBRUVICA blocks the Bruton’s tyrosine kinase (BTK) protein, which is needed by normal and abnormal B cells, including specific cancer cells, to multiply and spread. By blocking BTK, IMBRUVICA may help move abnormal B cells out of their nourishing environments and inhibits their proliferation.6,7,8

IMBRUVICA is approved in more than 100 countries and has been used to treat more than 250,000 patients worldwide. There are more than 50 company-sponsored clinical trials, including 18 Phase 3 studies, over 11 years evaluating the efficacy and safety of IMBRUVICA.

IMBRUVICA was first approved by the U.S. Food and Drug Administration (FDA) in November 2013, and today is indicated for adult patients in six disease areas, including five hematologic cancers. These include indications to treat adults with CLL/SLL with or without 17p deletion (del17p), and adults with Waldenström’s macroglobulinemia (WM), and adult patients with previously treated mantle cell lymphoma (MCL)*, as well as to treat adult patients with previously treated marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy*, and adult patients with previously treated chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy.9

*Accelerated approval was granted for MCL and MZL based on overall response rate. Continued approval for MCL and MZL may be contingent upon verification and description of clinical benefit in confirmatory trials.

Since 2019, the National Comprehensive Cancer Network (NCCN), recommends ibrutinib (IMBRUVICA) as a preferred regimen for the initial treatment of CLL/SLL and has Category 1 treatment status for treatment-naïve patients without deletion 17p/TP53 mutation and as a preferred treatment for treatment-naïve patients with deletion 17p/TP53 mutation. The NCCN Guidelines also recommend IMBRUVICA, with or without rituximab, as a preferred regimen for the treatment of relapsed/refractory MCL, as a Category 1 preferred regimen for both untreated and previously treated WM patients, and as a preferred regimen for relapsed/refractory MZL.10

For more information, visit www.IMBRUVICA.com.

IMBRUVICA IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hemorrhage: Fatal bleeding events have occurred in patients who received IMBRUVICA. Major hemorrhage (≥ Grade 3, serious, or any central nervous system events; e.g., intracranial hemorrhage [including subdural hematoma], gastrointestinal bleeding, hematuria, and postprocedural hemorrhage) occurred in 4% of patients, with fatalities occurring in 0.4% of 2,838 patients who received IMBRUVICA in 27 clinical trials. Bleeding events of any grade including bruising and petechiae occurred in 39%, and excluding bruising and petechiae occurred in 23% of patients who received IMBRUVICA, respectively.

The mechanism for the bleeding events is not well understood.

Use of either anticoagulant or antiplatelet agents concomitantly with IMBRUVICA increases the risk of major hemorrhage. Across clinical trials, 3.1% of 2,838 patients who received IMBRUVICA without antiplatelet or anticoagulant therapy experienced major hemorrhage. The addition of antiplatelet therapy with or without anticoagulant therapy increased this percentage to 4.4%, and the addition of anticoagulant therapy with or without antiplatelet therapy increased this percentage to 6.1%. Consider the risks and benefits of anticoagulant or antiplatelet therapy when co-administered with IMBRUVICA. Monitor for signs and symptoms of bleeding.

Consider the benefit-risk of withholding IMBRUVICA for at least 3 to 7 days pre- and post-surgery depending upon the type of surgery and the risk of bleeding.

Infections: Fatal and non-fatal infections (including bacterial, viral, or fungal) have occurred with IMBRUVICA therapy. Grade 3 or greater infections occurred in 21% of 1,476 patients who received IMBRUVICA in clinical trials. Cases of progressive multifocal leukoencephalopathy (PML) and Pneumocystis jirovecii pneumonia (PJP) have occurred in patients treated with IMBRUVICA. Consider prophylaxis according to standard of care in patients who are at increased risk for opportunistic infections.

Monitor and evaluate patients for fever and infections and treat appropriately.

Cytopenias: In 645 patients with B-cell malignancies who received IMBRUVICA as a single agent, grade 3 or 4 neutropenia occurred in 23% of patients, grade 3 or 4 thrombocytopenia in 8% and grade 3 or 4 anemia in 3%, based on laboratory measurements.

Monitor complete blood counts monthly.

Cardiac Arrhythmias and Cardiac Failure: Fatal and serious cardiac arrhythmias and cardiac failure have occurred with IMBRUVICA. Grade 3 or greater ventricular tachyarrhythmias occurred in 0.2% of patients, Grade 3 or greater atrial fibrillation and atrial flutter occurred in 4%, and Grade 3 or greater cardiac failure occurred in 1% of 1,476 patients who received IMBRUVICA in clinical trials. These events have occurred particularly in patients with cardiac risk factors, hypertension, acute infections, and a previous history of cardiac arrhythmias.

At baseline and then periodically, monitor patients clinically for cardiac arrhythmias and cardiac failure. Obtain an ECG for patients who develop arrhythmic symptoms (e.g., palpitations, lightheadedness, syncope, chest pain) or new onset dyspnea. Manage cardiac arrhythmias and cardiac failure appropriately, and if it persists, consider the risks and benefits of IMBRUVICA treatment and follow dose modification guidelines.

Hypertension: Hypertension occurred in 19% of 1,476 patients who received IMBRUVICA in clinical trials. Grade 3 or greater hypertension occurred in 8% of patients. Based on data from 1,124 of these patients, the median time to onset was 5.9 months (range, 0.03 to 24 months).

Monitor blood pressure in patients treated with IMBRUVICA and initiate or adjust anti-hypertensive medication throughout treatment with IMBRUVICA as appropriate.

Second Primary Malignancies: Other malignancies (10%), including non-skin carcinomas (4%), occurred among the 1,476 patients who received IMBRUVICA in clinical trials. The most frequent second primary malignancy was non-melanoma skin cancer (6%).

Tumor Lysis Syndrome: Tumor lysis syndrome has been infrequently reported with IMBRUVICA. Assess the baseline risk (e.g., high tumor burden) and take appropriate precautions. Monitor patients closely and treat as appropriate.

Embryo-Fetal Toxicity: Based on findings in animals, IMBRUVICA can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with IMBRUVICA and for 1 month after the last dose. Advise males with female partners of reproductive potential to use effective contraception during the same time period.

ADVERSE REACTIONS

B-cell malignancies: The most common adverse reactions (≥30%) in patients with B-cell malignancies (MCL, CLL/SLL, WM and MZL) were thrombocytopenia (54.5%)*, diarrhea (43.8%), fatigue (39.1%), musculoskeletal pain (38.8%), neutropenia (38.6%)*, rash (35.8%), anemia (35.0%)*, and bruising (32.0%).

The most common Grade ≥ 3 adverse reactions (≥5%) in patients with B-cell malignancies (MCL, CLL/SLL, WM and MZL) were neutropenia (20.7%)*, thrombocytopenia (13.6%)*, pneumonia (8.2%), and hypertension (8.0%).

Approximately 9% (CLL/SLL), 14% (MCL), 14% (WM) and 10% (MZL) of patients had a dose reduction due to adverse reactions. Approximately 4-10% (CLL/SLL), 9% (MCL), and 7% (WM [5%] and MZL [13%]) of patients discontinued due to adverse reactions.

cGVHD: The most common adverse reactions (≥20%) in patients with cGVHD were fatigue (57%), bruising (40%), diarrhea (36%), thrombocytopenia (33%)*, muscle spasms (29%), stomatitis (29%), nausea (26%), hemorrhage (26%), anemia (24%)*, and pneumonia (21%).

The most common Grade 3 or higher adverse reactions (≥5%) reported in patients with cGVHD were pneumonia (14%), fatigue (12%), diarrhea (10%), neutropenia (10%)*, sepsis (10%), hypokalemia (7%), headache (5%), musculoskeletal pain (5%), and pyrexia (5%).

Twenty-four percent of patients receiving IMBRUVICA in the cGVHD trial discontinued treatment due to adverse reactions. Adverse reactions leading to dose reduction occurred in 26% of patients.

*Treatment-emergent decreases (all grades) were based on laboratory measurements.

DRUG INTERACTIONS

CYP3A Inhibitors: Co-administration of IMBRUVICA with strong or moderate CYP3A inhibitors may increase ibrutinib plasma concentrations. Dose modifications of IMBRUVICA may be recommended when used concomitantly with posaconazole, voriconazole, and moderate CYP3A inhibitors. Avoid concomitant use of other strong CYP3A inhibitors. Interrupt IMBRUVICA if strong inhibitors are used short-term (e.g., for ≤ 7 days). See dose modification guidelines in USPI sections 2.3 and 7.1.

CYP3A Inducers: Avoid coadministration with strong CYP3A inducers.

SPECIFIC POPULATIONS

Hepatic Impairment (based on Child-Pugh criteria): Avoid use of IMBRUVICA in patients with severe hepatic impairment. In patients with mild or moderate impairment, reduce recommended IMBRUVICA dose and monitor more frequently for adverse reactions of IMBRUVICA.

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