On December 9, 2019 CTI BioPharma Corp. (Nasdaq: CTIC) reported the presentation of data from the Company’s pacritinib development program, including results from the PAC203 Phase 2 clinical trial, at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 7-10 in Orlando, Florida (Press release, CTI BioPharma, DEC 9, 2019, View Source [SID1234552159]).

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"The data presented at ASH (Free ASH Whitepaper) underscore the clinical and scientific rationale for our ongoing PAC203 Phase 3 PACIFICA trial evaluating pacritinib at 200 mg BID in severely thrombocytopenic myelofibrosis patients," said Adam R. Craig, M.D., Ph.D. "Pacritinib has now been demonstrated to provide clinical benefit in treating severely thrombocytopenic myelofibrosis (platelet counts less than 50,000 per microliter) in three clinical trials, including two prior randomized Phase 3 studies. Further, the PAC203 Phase 2 results from the 200 mg BID cohort demonstrate a favorable risk-benefit profile for pacritinib when treating patients with advanced disease that have high mutational risk and long durations of prior ruxolitinib exposure. The totality of the data presented at ASH (Free ASH Whitepaper) reinforces our belief that pacritinib has the potential to be an important therapy for severely thrombocytopenic myelofibrosis patients, a population for whom available therapeutic options are limited and often ineffective."

"While currently available treatments for myelofibrosis convey clinical benefit to a majority of the population, patients who are severely thrombocytopenic, and are therefore considered high-risk, continue to lack safe and efficacious therapies," said Claire N. Harrison, M.D., Consultant Haematologist and Professor of Myeloproliferative Neoplasms in London, as well as Chair of the PACIFICA steering committee and Principal Investigator of the PAC203 Phase 2 trial. "Myelofibrosis patients with platelet counts of less than 50,000 per microliter have a median survival of approximately 8 months after discontinuation of first line ruxolitinib therapy. In the PAC203 Phase 2 trial, which represents one of the most advanced and heavily pre-treated myelofibrosis patient populations studied to date as demonstrated by a median prior exposure to ruxolitinib of 1.7 years and the high risk mutational profiles, pacritinib at 200 mg BID achieved an impressive response rate in the severely thrombocytopenic patient population of 17% with a promising safety profile, demonstrating its potential to change the treatment paradigm in this area of serious unmet medical need."

All posters and presentation materials will be available at ctibiopharma.com following the presentations.

Results of PAC203: A Randomized Phase 2 Dose-Finding Study and Determination of the Recommended Dose of Pacritinib

Results from the PAC203 Phase 2 trial are being presented today, Monday, December 9, at 10:30 AM ET, in an oral presentation session.

Abstract: No. 667

Summary: PAC203 enrolled patients with myelofibrosis who were intolerant of or who had not benefitted from prior treatment with ruxolitinib. Patients were randomized in equal measure across 3 dosing arms: 200mg twice-daily (BID), 100mg BID, and 100mg daily (QD), with randomization stratified by baseline platelet count. Patients were mostly thrombocytopenic and anemic at baseline, with a median platelet count was 55,000, and 44% of patients having baseline platelet counts of less than 50,000. Patients had been heavily pre-treated with ruxolitinib, with a median 1.7 years of prior exposure. The study endpoint was broadly defined as an analysis of safety and efficacy data across dosing arms based on data after all patients either reached week 24 or stopped study treatment.

Pacritinib was shown to be generally well tolerated across dosing cohorts, with the most common treatment-emergent non-hematologic adverse events (AEs) being gastrointestinal, including diarrhea (20.5%; Grade 3: 3.1%) and nausea (20%; Grade 3: 0.6%), distributed similarly across arms. The most common hematologic AEs were thrombocytopenia and anemia, both occurring at higher frequencies at the 200 mg dose BID (32% and 22% respectively); this did not, however, lead to higher rates of Grade 3/4 hemorrhage at higher doses (200 mg BID: 5.6%; 100 mg BID: 0%; 100 mg QD: 5.8%; all Grade 3). Similarly, the highest dose saw no excess in Grade 3/4 cardiac (200 mg BID: 3.7%; 100 mg BID: 7.3%; 100 mg QD: 3.7%; all Grade 3) or infectious (200 mg BID: 15%; 100 mg BID: 11%; 100 mg QD: 12%) AEs. In this cohort of advanced MF patients, there were 7 Grade 5 (fatal) AEs: 2 at 200 mg BID (sepsis, subdural hematoma), 3 at 100 mg BID (disease progression, subdural hematoma, heart failure), and 2 at 100 mg QD (sepsis, tuberculosis).

The 200 mg BID arm had the highest observed rates of SVR ≥35% (200 mg BID: 9.3%; 100 mg BID: 1.8%; 100 mg QD: 0.0%). Of the 5 patients with SVR ≥35% at the 200 mg BID dose, 4 had platelet counts <50,000/mL, representing a 17% (4/24) response rate among patients with severe thrombocytopenia. Though a dose response relationship was not observed in total symptom score (TSS) based on the threshold of 50% reduction in symptom score, as 4 patients achieved this endpoint on all arms, the median reduction in TSS was greatest for patients treated at 200 mg BID (200 mg BID: 27%%; 100 mg BID: 16%; 100 mg QD: 3%).

The data from PAC203 support the Phase 3 PACIFICA trial, currently underway to compare the safety and efficacy of 200 mg BID of pacritinib to Physician’s Choice in 180 adult myelofibrosis patients with severe thrombocytopenia (platelet counts of less than 50,000 per microliter).

The Oral JAK2/IRAK1 Inhibitor Pacritinib Demonstrates Spleen Volume Reduction in Myelofibrosis Patients Independent of JAK2V617F Allele Burden

Results from a retrospective analysis from two Phase 3 studies, PERSIST-1 and PERSIST-2, of pacritinib in myelofibrosis patients were presented in a poster session on Saturday, December 7.

Abstract: No. 1674

Summary: A retrospective analysis of PERSIST-1 and PERSIST-2 was performed in which outcomes were stratified by JAK2 V617F mutation status and allele burden. The efficacy endpoint for this study was the percentage of patients achieving ≥35% SVR at week 24 based on an intention-to-treat analysis. Analysis was based on pooled results across the two studies for patients treated with pacritinib and those treated with best available therapy (BAT), and assessed patients with JAK2 V617F for possible relationship between allelic burden and SVR at 24 weeks. The analysis showed pacritinib was associated with a significantly higher rate of SVR response than BAT among patients with low JAK2 allele burden (<50%), while no SVR response was observed for patients treated with BAT (including ruxolitinib) who had low JAK2 allele burden or JAK2 V617F-negative disease. This data suggests that pacritinib, a JAK2/IRAK1 inhibitor, may provide benefit over a wider range of patients with myelofibrosis compared to other JAK inhibitors, specifically patients with low JAK2 allele burden who were shown to have lower baseline platelet counts, more severe anemia and smaller spleen size.

Pacritinib Demonstrates Efficacy Versus Best Available Therapy in Myelofibrosis Patients with Severe Thrombocytopenia in Two Phase 3 Studies

Results from a retrospective analysis of PERSIST-1 and PERSIST-2 will be presented in a poster session today, Monday, December 9 at 6:00 PM ET.

Abstract: No. 4195

Summary: This analysis was performed in patients treated on PERSIST-1 and PERSIST-2 with a baseline platelet count <50,000/μL. At 24 weeks, significantly more patients achieved ≥35% SVR with pacritinib compared with BAT (P-values <0.01). Although not statistically significant, TSS reductions ≥50% nearly doubled for those receiving pacritinib versus BAT. The safety profile in patients with severe thrombocytopenia was generally manageable and consistent with the overall study populations from the two Phase 3 trials. This retrospective analysis represents the largest population of patients with MF and severe thrombocytopenia to be studied in clinical trials, a population with a serious unmet medical need, and the results illustrate the clinical activity of pacritinib in the treatment of these patients.

Molecular Analysis in the Pacritinib Dose-Finding PAC203 Study in Patients with Myelofibrosis Refractory or Intolerant to Ruxolitinib

Results from a baseline mutational analysis of patients enrolled in the Phase 2 PAC203 study of pacritinib in patients with myelofibrosis will be presented in a poster session today, December 9, 2019 at 6:00 PM ET.

Abstract: No. 4214

Summary: The baseline mutational analysis was performed on 105 (out of total 164 recruited, 161 treated) myelofibrosis patients in the PAC203 study. The PAC203 cohort, characterized by ruxolitinib failure and a high burden of anemia and thrombocytopenia, was shown to be a molecularly high-risk population, characterized by high incidence of HMR, TP53 and RAS mutations, high mutational burden, and low incidence of CALR mutations.

About Myelofibrosis and Severe Thrombocytopenia

Myelofibrosis is a type of bone marrow cancer that results in formation of fibrous scar tissue and can lead to severe anemia, weakness, fatigue and an enlarged spleen and liver. Patients with severe thrombocytopenia are estimated to make up more than one-third of patients treated for myelofibrosis, or approximately 18,000 people.1 Severe thrombocytopenia, defined as blood platelet counts of less than 50,000 per microliter, has been shown to result in overall survival rates of just 15 months.2 Thrombocytopenia in patients with myelofibrosis is associated with the underlying disease but has also been shown to correlate with treatment with ruxolitinib, which can lead to dose reductions, and as a result, may potentially reduce clinical benefit. Survival in patients who have discontinued ruxolitinib therapy is further compromised, with an average overall survival of seven to 14 months.3,4 There are currently no approved therapies available to treat myelofibrosis patients with severe thrombocytopenia, or patients who have failed ruxolitinib treatment, thereby making this a significant unmet medical need.

About Pacritinib

Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia and lymphoma. In addition to myelofibrosis, the kinase profile of pacritinib suggests its potential therapeutic utility in conditions such as acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and chronic lymphocytic leukemia (CLL), due to its inhibition of c-fms, IRAK1, JAK2 and FLT3.

On December 9, 2019 STORM Therapeutics, the biotechnology company focused on the discovery of small molecule therapies modulating RNA epigenetics reported, that its collaborator Dr. Konstantinos Tzelepis, Sir Henry Wellcome Fellow and visiting scientist at the Wellcome Sanger Institute, received the ASH (Free ASH Whitepaper)-BSH Abstract Achievement Award for his work on STORM Therapeutics’ lead programme, METTL3, at the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) held on 7th-10th December 2019 in Orlando, Florida (Press release, STORM Therapeutics, DEC 9, 2019, View Source;exposition-300971436.html [SID1234552158]).

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Dr. Tzelepis received the meritorious award for his abstract entitled ‘Pharmacological Inhibition of the RNA m6a Writer METTL3 As a Novel Therapeutics Strategy for Acute Myeloid Leukemia’ at the Abstract Achievement Award Ceremony which was held on Saturday, 7th December 2019 at 3:30pm (EST) at the Orange County Convention Center in Exhibit Hall B2-B4.

Dr. Tzelepis’s work, which was carried out in collaboration with the Wellcome Sanger Institute and the University of Cambridge, was presented in an oral presentation during the "802. Chemical Biology and Experimental Therapeutics: Novel Compounds and Mechanisms of Action" session on Sunday, 8th December 2019 at 9:30am-11:30am (EST) at the Orange County Convention Centre. The abstract and talk encapsulated the ground-breaking work made on targeting RNA modifying enzymes for cancer treatment and described the recent progress made with the METTL3 inhibitor.

STORM has identified small molecule inhibitors of METTL3 that are orally bioavailable and show pronounced anti-tumour efficacy in physiologically relevant, proof of concept animal models of Acute Myeloid Leukaemia (AML). The talk demonstrated that small molecule inhibition of METTL3 produces the same phenotype and effects previously described in one of STORM’s founder scientists’ publications using genetic models and validates METTL3 as a druggable target for cancer.

Keith Blundy, CEO of STORM Therapeutics, said: "STORM is progressing fast in its preclinical work with our multiple programmes to showcase the capabilities of our novel platform. STORM is a pioneer in RNA epigenetics and we are very pleased to hear that our collaborator is being recognized for the partnership research of METTL3. As the first company in the world to demonstrate in vivo activity of an RNA methyltransferase inhibitor, we are excited to be leading the field as we look to develop these highly innovative new treatment options for cancer patients."

On December 9, 2019 Menarini Silicon Biosystems, the pioneer of liquid biopsy and rare cell technologies, reported that new research utilizing the company’s CELLSEARCH Circulating Tumor Cell (CTC) test will be featured at the upcoming 2019 San Antonio Breast Cancer Symposium (SABCS) (Press release, Menarini Silicon Biosystems, DEC 9, 2019, View Source [SID1234552157]).

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Researchers from Northwestern University will present the results of three studies that used the FDA-cleared CELLSEARCH system to conduct CTC testing in a total of 463 breast cancer patients. The poster presentations provide preliminary insight into the use of liquid biopsy and CTC enumeration to help clinicians better understand the metastatic process and facilitate management of patients with metastatic breast cancer (MBC).

"Liquid biopsies enable us to monitor the disease over time and more fully understand the complexity, heterogeneity and evolution of the disease, which is critical to selecting better treatments," said Massimo Cristofanilli, M.D., F.A.C.P., Associate Director for Translational Research, Robert H. Lurie Comprehensive Cancer Center of Northwestern University. "While we need to expand these data to further prospective studies for validation, this new research marks an important step towards potentially expanding the role of liquid biopsy — particularly enumeration of CTCs and molecular analysis for accurate and real-time prediction and prognostic monitoring the metastatic process."

Dr. Cristofanilli co-authored all three CELLSEARCH studies being presented at SABCS**.

CELLSEARCH is the first and only clinically validated blood test cleared by the FDA for detecting and enumerating CTCs as an aid for physicians to manage patients with metastatic breast, prostate and colorectal cancers. The test is also approved by the Chinese National Medical Products Administration (NMPA) for use as an aid in monitoring metastatic breast cancer patients.

"There is a large body of research supporting the longstanding value of CTCs, which is one of the reasons many physicians are using CELLSEARCH in their clinics," said Fabio Piazzalunga, President and CEO of Menarini Silicon Biosystems, Inc. "Studies like these help to increase understanding of the value of liquid biopsy and CTC enumeration and play an important role in advancing precision medicine for metastatic breast cancer."

Poster Presentation Details:

Poster Session 3 – Thursday, December 12, 5:00 pm – 7:00 pm, Hall 1

Title:

Liquid biopsy methods and machine learning modeling to understand organ tropism and metastization behavior of metastatic breast cancer – P3-01-05

Authors:

Gerratana L et al.
Northwestern University, Chicago, IL; IRCCS CRO Aviano National Cancer Institute, Aviano, Italy.

Poster Session 4 – Friday, December 13, 7:00 am – 9:00 am, Hall 1

Title:

Clinical relevance of CK+/CD45+, dual-positive circulating cells (DPCells) in patients with metastatic breast cancer (MBC) – P4-01-07

Authors:

Reduzzi et al.
Comprehensive Cancer Center, Feinberg School of Medicine, Northwestern University, Chicago, IL.

Title:

Anatomical staging and value of CTCs in locally advanced breast cancer (LABC) and metastatic breast cancer (MBC) – P4-01-08

Authors:

Zhang Q et al.
Department of Medicine, University of Udine, Uniud, Italy; Department of Medicine, Northwestern University, Chicago, IL; Department of Pathology, Northwestern University, Chicago, IL.

The CELLSEARCH System is the most extensively studied CTC technology, with research published in more than 650 peer-reviewed publications. Providing valuable information to help physicians make patient-management decisions along with other clinical monitoring methods, the CELLSEARCH CTC test is performed at a reference laboratory using the CELLSEARCH System. The testing can be used throughout a given therapy for metastatic breast, colorectal, and prostate cancer to monitor a patient’s status by showing if their prognosis is favorable.

To learn more about the CELLSEARCH System, SABCS attendees can visit Menarini Silicon Biosystems at Booth #909.

SABCS will be held at the Henry B. Gonzalez Convention Center, San Antonio, Texas, Dec. 10-14, 2019.

On December 9, 2019 Jasper Therapeutics, Inc., a new biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies, reported that initial results from an ongoing Phase 1 dose-escalation study of its lead product candidate, JSP191 (formerly AMG191), will be presented today in an oral session at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (Press release, Jasper Therapeutics, DEC 9, 2019, View Source [SID1234552156]).

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JSP191, a humanized antibody targeting CD117, is designed to replace or reduce the toxicity of chemotherapy and radiation therapy as a conditioning regimen to prepare patients for hematopoietic cell transplantation. The Phase 1 clinical trial is evaluating JSP191 as a conditioning agent to enable stem cell transplantation in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that had poor outcomes.

"Life-threatening disorders such as SCID, and other conditions including autoimmune diseases and hematologic cancers, can be cured by hematopoietic cell transplantation, and those with certain genetic diseases can be cured with stem cell-directed gene therapies. However, the number of patients who can benefit from these approaches is limited because of the severe toxicity of the chemotherapy used for pre-transplant conditioning that is needed to allow room in the bone marrow for the stem cells to engraft," said Judith Shizuru, M.D., Ph.D., co-founder and member of the Board of Directors of Jasper Therapeutics. "We are encouraged by the initial Phase 1 study results of JSP191 in these fragile patients with SCID and plan to expand clinical development of this antibody beyond patients with SCID. We expect to initiate clinical trials of JSP191 in 2020 to evaluate it as a conditioning agent in patients undergoing hematopoietic cell therapy for acute myeloid leukemia, myelodysplastic syndrome and Fanconi anemia, and IND-enabling studies for sickle cell disease and autoimmune indications."

Details of the oral presentation follow:

Abstract Title: Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency (abstract #800)
Session Name: 721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Innovative Approaches in Allogeneic Transplantation for Pediatric or Nonmalignant Disorders
Presenter: Rajni Agarwal, M.D., Associate Professor of Pediatrics and Stem Cell Transplantation, the Stanford University School of Medicine
Time: 3:00 p.m. ET
Location: W311EFGH, Level 3, Orange County Convention Center

About Stem Cell Transplantation

Blood-forming, or hematopoietic, stem cells are cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Successful transplantation of hematopoietic stem cells is the only cure for most of these life-threatening conditions. Replacement of the defective or malignant hematopoietic stem cells in the patient’s bone marrow is currently achieved by subjecting patients to toxic treatment with radiation and/or chemotherapy that cause DNA damage and lead to short- and long-term toxicities, including immune suppression and prolonged hospitalization. As a result, many patients who could benefit from a stem cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191

JSP191 (formerly AMG191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1 dose-escalation trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant.

On December 9, 2019 Portola Pharmaceuticals, Inc. (Nasdaq: PTLA) reported new interim results from the Company’s ongoing Phase 2a study of cerdulatinib, an investigational, oral SYK/JAK inhibitor, in patients with relapsed/refractory follicular lymphoma (FL) receiving cerdulatinib alone or in combination with rituximab (Press release, Portola Pharmaceuticals, DEC 9, 2019, View Source [SID1234552155]). The data will be presented today during a poster session at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando (December 7-10).

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Data included safety and efficacy findings as of November 2019 for 42 patients who received single agent cerdulatinib at 30 mg twice daily (with the exception of two patients who initiated treatment at 35 mg) and 21 patients who received cerdulatinib at 30 mg twice daily in combination with a standard dosing regimen of rituximab. The number of prior treatment regimens including anti-CD20 antibody, bendamustine and other alkylating agents, and PI3K inhibitors ranged from one to 10, with a median of three.

Among the 42 patients in the cerdulatinib-only cohort, the overall response rate (ORR) was 48%; 7 patients (17%) achieved a complete response (CR), 13 patients (31%) achieved a partial response (PR) and 10 patients (24%) achieved stable disease (SD). To date, 16 of the 42 patients (38%) in the cerdulatinib-only cohort have been on study drug for at least 10 months.

Among the 21 patients evaluated for efficacy in the cerdulatinib and rituximab combination cohort, the ORR was 76%; 5 patients (24%) achieved a CR, 11 patients (52%) achieved a PR and 5 patients (24%) achieved SD. Of the 11 patients in this combination cohort who have been on one to three prior therapies, the ORR was 91% with a complete response rate of 36%.

Cerdulatinib was generally well-tolerated and the safety profile appeared similar in both the cerdulatinib-only and rituximab combination cohorts. The most common adverse events (AEs) occurring in ≥5% of all evaluable study patients were lipase increase (27%), neutropenia (18%), diarrhea (13%) and amylase increase (9%). The most common AEs in the combination cohort included lipase increase (32%), neutropenia (22%) and diarrhea (14%). The lipase and amylase changes were generally asymptomatic and not associated with pancreatitis. Additionally, there was no emergence of late-stage colitis, cardiac or liver abnormalities, or other evidence of cumulative toxicity.

"These interim results demonstrate that cerdulatinib provides sustained clinical activity and good tolerability in patients with relapsed/refractory follicular lymphoma, and that the tumor response to both monotherapy and combination therapy appears to deepen over time," said Paul Hamlin, M.D., medical director for the David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center. "It is encouraging that the combination of cerdulatinib and rituximab achieved an improved objective response rate (ORR) in heavily pre-treated patients compared to prior interim data, indicating it may have potential as a second-line therapy. We look forward to continuing the study and exploring an optimized dose of cerdulatinib and rituximab in this setting."

"Cerdulatinib is the most advanced SYK/JAK inhibitor of its kind in development for oncology, and has demonstrated its potential to inhibit two key survival pathways in Non-Hodgkin Lymphoma," said Jeff Myers, Portola’s interim chief medical officer. "The data presented at ASH (Free ASH Whitepaper) continues to demonstrate its safety and efficacy across a range of malignancies. Specific to follicular lymphoma, we are excited that the updated data showed an improved ORR with greater anti-tumor activity in combination with rituximab, and we look forward to exploring lower doses for potential use in the second-line setting."

ASH Poster Session Details – Monday, December 9, 2019, at 6:00 p.m. EST

Title:

Rapid and Durable Responses with the SYK/JAK Inhibitor Cerdulatinib in a Phase 2 Study in Relapsed/Refractory Follicular Lymphoma—Alone or in Combination with Rituximab

Session:

623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies: Poster III

Presenter:

Paul A. Hamlin, M.D., David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center

Location:

Hall B, Level 2 (Orange County Convention Center)

About the Phase 2a Study
The Phase 2a, open-label study was designed to assess the safety and efficacy of cerdulatinib in patients with relapsed/refractory FL (alone or in combination with rituximab), small lymphocytic lymphoma (SLL) and specific subtypes of T-cell Non-Hodgkin Lymphoma, including PTCL, AITL and CTCL.

Tumor response in the two cohorts evaluating patients with relapsed/refractory FL was assessed by Lugano classification, with treatment continued until disease progression or unacceptable toxicity. Tumor response assessments were performed at the end of cycle two and every three cycles thereafter.

About Cerdulatinib
Cerdulatinib is an investigational oral, dual spleen tyrosine kinase (SYK) and janus kinase (JAK) inhibitor that uniquely inhibits two key cell signaling pathways implicated in certain hematologic malignancies and autoimmune diseases. There is a strong rationale for inhibiting both SYK (B-cell receptor pathway) and JAK (cytokine receptors) in B-cell malignancies where both targets have been shown to promote cancer cell growth and survival.

The U.S. Food and Drug Administration granted cerdulatinib Orphan Drug Designation for the treatment of PTCL in September 2018.