On November 4, 2021 The Pancreatic Cancer Action Network (PanCAN) and CDISC reported the release of a new Therapeutic Area User Guide setting the first-ever global data standards specifically for pancreatic cancer (Press release, PanCAN, NOV 4, 2021, View Source [SID1234594631]). The project was funded through a two-year grant awarded to CDISC by PanCAN and is designed to lead to greater efficiencies and data sharing among the pancreatic cancer scientific community.

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The User Guide includes global, nonproprietary clinical metadata standards and core precision medicine-focused concepts designed for pancreatic cancer to enable global researchers to efficiently collect, organize and analyze data across studies, facilitating the development of treatments that make a difference in the lives of pancreatic cancer patients. It is intended to drive operational efficiencies, expedite the regulatory review process, enable data sharing and reduce the time it takes to bring safe and effective treatments to market.

"Through this partnership with CDISC to standardize data submissions to regulatory agencies, we are creating opportunities to streamline drug development in the pancreatic cancer space," said Sudheer Doss, PhD, PanCAN’s Chief Business Officer. "With a disease as difficult to treat as pancreatic cancer, patients can’t afford to wait. By accelerating clinical advancements through these efficiencies, we hope to improve patient outcomes and, ultimately, increase survival."

"We are grateful to PanCAN for partnering with CDISC to allow the development of these crucial standards," said Rhonda Facile, VP, Partnerships and Development, CDISC. "It is our hope that the research community will swiftly adopt this Therapeutic Area User Guide to conduct more powerful and meaningful research to enable the development of treatments and therapies to treat this devastating disease."

Two years in the making, the User Guide for pancreatic cancer is now freely available via the CDISC website. To date, CDISC has developed Therapeutic Area User Guides for over 40 disease areas.

Pancreatic cancer is currently the third leading cause of cancer-related death in the U.S., with an overall five-year survival rate of just 10 percent. In 2021 more than 60,000 Americans will be diagnosed with pancreatic cancer and approximately 48,000 will die from the disease, underscoring the scientific collaboration to develop new and better treatment options for patients.

To learn more about pancreatic cancer, please visit pancan.org.

Regeneron has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On November 4, 2021 Incyte (Nasdaq:INCY) reported that numerous abstracts highlighting data from its oncology portfolio will be presented at the upcoming 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 (Press release, Incyte, NOV 4, 2021, View Source [SID1234594359]).

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"We are excited for the opportunity to present, along with our partners, more than 35 abstracts and share progress from Incyte’s oncology portfolio at this year’s ASH (Free ASH Whitepaper) meeting," said Peter Langmuir, M.D., Group Vice President, Oncology Targeted Therapeutics, Incyte. "The oral presentations, including new data from the CITADEL program evaluating parsaclisib in three types of lymphoma as well as results from the RE-MIND2 study of tafasitamab plus lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma, highlight the strength of our robust oncology portfolio and underscore our commitment to addressing urgent medical needs for people with cancer."

Select key abstract presentations from Incyte-developed and partnered programs include:

Oral Presentations

Parsaclisib

Efficacy and Safety of Parsaclisib in Patients with Relapsed or Refractory Marginal Zone Lymphoma: Primary Analysis from a Phase 2 Study (CITADEL-204) (Abstract #44. Session: 623. Mantle Cell, Follicular, and Other B-Cell Lymphomas: Clinical and Epidemiological: Targeted Therapy in Low Grade Lymphoma. Saturday, December 11, 9:45 a.m. ET)

Efficacy and Safety of Parsaclisib in Patients with Relapsed or Refractory Mantle Cell Lymphoma Not Previously Treated With a BTK Inhibitor: Primary Analysis From a Phase 2 Study (CITADEL-205) (Abstract #382. Session: 623. Mantle Cell, Follicular, and Other B-Cell Lymphomas: Clinical and Epidemiological: Front-line Induction Therapy and Novel Agents After Relapse. Sunday, December 12, 10:15 a.m. ET)

Efficacy and Safety of Parsaclisib in Patients With Relapsed or Refractory Follicular Lymphoma: Primary Analysis From a Phase 2 Study (CITADEL-203) (Abstract #813. Session: 623. Mantle Cell, Follicular, and Other B-Cell Lymphomas: Clinical and Epidemiological: Front-line Induction Therapy and Novel Agents After Relapse. Monday, December 13, 5:00 p.m. ET)

Pemigatinib: Myeloproliferative Neoplasms (MPN)

A Phase 2 Study of Pemigatinib (FIGHT-203; INCB054828) in Patients with Myeloid/Lymphoid Neoplasms (MLNs) with Fibroblast Growth Factor Receptor 1 (FGFR1) Rearrangement (MLNFGFR1) (Abstract #385. Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Novel Therapies for MPNs and JAK Inhibitors for Myelofibrosis. Sunday, December 12, 9:30 a.m. ET)

Ponatinib

Post hoc Analysis of Responses to Ponatinib in Patients with Chronic-Phase Chronic Myeloid Leukemia (CP-CML) by Baseline BCR-ABL1 level and Baseline Mutation Status in the OPTIC Trial1 (Abstract #307. Session:#623. Chronic Myeloid Leukemia: Clinical and Epidemiological: Mechanisms of Resistance and Expanded Therapies. Saturday, December 11, 4:00 p.m. ET)

Ruxolitinib: Graft-Versus-Host Disease (GVHD)

Validation of Amphiregulin as a Monitoring Biomarker During Treatment of Life-Threatening Acute GVHD: A Secondary Analysis of 2 Prospective Clinical Trials (Abstract #259. Session: 722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Treatment of Acute and Chronic Graft vs. Host Disease. Saturday, December 11, 2:00 p.m. ET)

Ruxolitinib: Myeloproliferative Neoplasms (MPN)

The Interaction Between IPSS Score and JAK2 Mutation Identifies Patients at Different Vascular Risk in Primary Myelofibrosis2 (Abstract #236. Session: #634. Myeloproliferative Syndromes: Clinical and Epidemiological: Risk Stratification and Prognostication. Saturday, December 11, 2:15 p.m. ET)

A Real-World Evaluation of the Association Between Elevated Blood Counts and Thrombotic Events in Polycythemia Vera (Analysis of Data from the REVEAL Study)(Abstract #239. Session:634. Myeloproliferative Syndromes: Clinical and Epidemiological: Risk Stratification and Prognostication. Saturday, December 11, 3:00 p.m. ET)

Tafasitamab

Tafasitamab Plus Lenalidomide Versus pola‑BR, R2, and CAR-T: Comparison of Outcomes from RE-MIND2, an Observational Retrospective Cohort Study in Relapsed or Refractory Diffuse Large B-Cell Lymphoma3 (Abstract #183. Session:905. Outcomes Research-Lymphoid Malignancies: Lymphoma/CLL Real-World Data. Saturday, December 11, 12:30 p.m. ET)

Poster Presentations

All accepted posters in Poster I are available from 9:00 a.m. – 7:30 p.m. ET on Saturday, December 11. All accepted posters in Poster II sessions are available from 9:00 a.m. – 8:00 p.m. ET on Sunday, December 12. All accepted posters in Poster III sessions are available from 9:00 a.m. – 8:00 p.m. ET on Monday, December 13.

INCB000928: Myeloproliferative Neoplasms (MPN)

A Phase 1/2, Open-label, Multicenter Study of INCB000928 Monotherapy in Patients with Anemia due to Myelodysplastic Syndromes or Multiple Myeloma (Abstract #3707. Session:637. Myelodysplastic Syndromes — Clinical and Epidemiological: Poster III. Monday, December 13)

INCB057643: Myeloproliferative Neoplasms (MPN)

A Phase 1 Study of INCB057643 Monotherapy in Patients with Relapsed or Refractory Myelofibrosis (INCB 57643-103) (Abstract #2574. Session:634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II. Sunday, December 12)

Parsaclisib

A Phase 2, Multicenter, Single-arm, Open-Label study of Parsaclisib, a PI3Kδ inhibitor, in Relapsed or Refractory Follicular Lymphoma in China4 (Abstract #3536. Session: 623. Mantle Cell, Follicular, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster III. Monday, December 13)

Ponatinib

Dose Modification Dynamics of Ponatinib in Patients with Chronic-Phase Chronic Myeloid Leukemia (CP-CML) from the PACE and OPTIC Trials1(Abstract #2550. Session: 632. Chronic Myeloid Leukemia: Clinical and Epidemiological: Poster II. Sunday, December 12)

Multicenter, Prospective and Retrospective Observational Cohort Study of Ponatinib in Patients with CML in Italy: Primary Analysis of the OITI Trial (Abstract #3603. Session:632. Chronic Myeloid Leukemia: Clinical and Epidemiological: Poster III. Monday, December 13)

Ruxolitinib: GVHD

Disability Associated with Chronic Graft-Versus-Host Disease after Allogeneic Hematopoietic Cell Transplantation (Abstract #4060. Session:904. Outcomes Research—Non-Malignant Conditions: Poster III. Monday, December 13)

Update of Multicenter, Retrospective Evaluation of Overall Response and Failure Free Survival Following Ruxolitinib Therapy for Heavily Pre-Treated Chronic GVHD Patients with Steroid-Failure: A Proposal of Risk Score Model for Failure-Free Survival2(Abstract #3905. Session: #722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Poster III. Monday, December 13)

Patient-Reported Outcomes (PROs) Among Patients with Steroid-Refractory or Dependent Chronic Graft-vs-Host Disease (cGVHD) Randomized to Ruxolitinib (RUX) vs Best Available Therapy (BAT)2(Abstract #3909. Session:Session: #722. Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Poster III. Monday, December 13)

Ruxolitinib: Myeloproliferative Neoplasms (MPN)

Does Early Intervention in Myelofibrosis Impact Outcomes? A Pooled Analysis of the COMFORT I and II Studies (Abstract #1505. Session:634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster I. Saturday, December 11)

Community Versus Academic Practice in Essential Thrombocythemia and Myelofibrosis: Differences in Clinical Characteristics, Diagnosis, Treatment Patterns, and Symptom Burden (Analysis of Data from the MOST Study) (Abstract #1497. Session:634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster I. Saturday, December 11)

ADORE: A Randomized, Open-Label, Phase 1/2 Open-Platform Study Evaluating Safety and Efficacy of Novel Ruxolitinib Combinations in Patients with Myelofibrosis2(Abstract #1489. Session:#634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster I. Saturday, December 11)

Characteristics of High-Risk Polycythemia Vera Patients with Suboptimal Response to First-Line Therapy who Switched to Ruxolitinib vs. Those who did not Switch: Findings from PV-SWITCH, a Multinational, Retrospective Chart Review Study2(Abstract #3646. Session:#634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster III. Monday, December 13)

Labor Market Attachment in Patients with Myeloproliferative Neoplasms: A Nationwide Matched Cohort Study2(Abstract #3627. Session:#634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster III. Monday, December 13)

Ruxolitinib + Parsaclisib Combination Studies: Myeloproliferative Neoplasms (MPN)

A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of Add-On Parsaclisib in Patients with Myelofibrosis who have Suboptimal Response to Ruxolitinib (Abstract #1502. Session:634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster I. Saturday, December 11)

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Ruxolitinib plus Parsaclisib in Patients With JAK- and PI3K-Inhibitor Treatment–Naive Myelofibrosis (Abstract #2579. Session:634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II. Sunday, December 12)

Subgroup Analysis from a Phase 2 Study of the Efficacy and Safety of Parsaclisib, a Selective PI3Kδ Inhibitor, in Combination with Ruxolitinib in Patients with Myelofibrosis (MF) (Abstract #3647. Session:634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster III. Monday, December 13)

Tafasitamab

inMIND: A Phase 3 Study of Tafasitamab Plus Lenalidomide and Rituximab Versus Placebo plus Lenalidomide and Rituximab for Relapsed/Refractory Follicular or Marginal Zone Lymphoma (Abstract #2421. Session:623. Mantle Cell, Follicular, and Other Indolent B Cell Lymphomas: Clinical and Epidemiological: Poster II. Sunday, December 12)

Preferences and Perceptions Regarding Treatment Decision-Making for Relapsed or Refractory Diffuse Large B-Cell Lymphoma (R/R DLBCL)3 (Abstract #1928. Session:902. Health Services Research—Lymphoid Malignancies: Poster I. Saturday, December 11)

The Impact of Prior Treatment with a CD19 Targeting Monoclonal Antibody on Subsequent Treatment with CD19 Targeting CART Cell Therapy in Preclinical Models3(Abstract #2412. Session:622. Lymphomas: Translational—Non-Genetic: Poster II. Sunday, December 12)

The SUMOylation Inhibitor TAK-981 in Combination with the CD19-Targeting Antibody Tafasitamab Shows Enhanced Anti-Tumor Activity in Preclinical B-Cell Lymphoma Models3(Abstract #2268. Session:605. Molecular Pharmacology and Drug Resistance: Lymphoid Neoplasms: Poster II. Sunday, December 12)

First-MIND: A Phase Ib, Open-Label, Randomized Study to Assess Safety of Tafasitamab or Tafasitamab + Lenalidomide in Addition to R‑CHOP in Patients with Newly Diagnosed Diffuse Large B-cell Lymphoma3(Abstract #3556. Session:626. Aggressive Lymphomas: Prospective Therapeutic Trials: Poster III. Monday, December 13)

Full abstracts will be available on ASH (Free ASH Whitepaper)’s website and in a special online-only issue of Blood, ASH (Free ASH Whitepaper)’s official journal. More information regarding ASH (Free ASH Whitepaper) 2021 can be found on ASH (Free ASH Whitepaper)’s website: View Source

About Jakafi (ruxolitinib)

Jakafi (ruxolitinib) is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. FDA for treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea, in adults with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF, for treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older and for the treatment of chronic GVHD after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older.

Jakafi is marketed by Incyte in the U.S. and by Novartis as Jakavi (ruxolitinib) outside the U.S. Jakafi is a registered trademark of Incyte. Jakavi is a registered trademark of Novartis AG in countries outside the U.S.

About Monjuvi (tafasitamab-cxix)

Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP).

In the United States, Monjuvi (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In Europe, Minjuvi (tafasitamab) received conditional approval, in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT).

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in several ongoing combination trials.

Minjuvi and Monjuvi are registered trademarks of MorphoSys AG. Tafasitamab is co-marketed by Incyte and MorphoSys under the brand name Monjuvi in the U.S., and marketed by Incyte under the brand name Minjuvi in the EU.

XmAb is a registered trademark of Xencor, Inc.

About Ponatinib (Iclusig) Tablets

Ponatinib (Iclusig) targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

In the EU, Iclusig is approved for the treatment of adult patients with chronic phase, accelerated phase or blast phase chronic myeloid leukemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation, or the treatment of adult patients with Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

Click here to view the Iclusig EU Summary of Medicinal Product Characteristics.

Incyte has an exclusive license from Takeda Pharmaceuticals International AG to commercialize ponatinib in the European Union and 29 other countries, including Switzerland, UK, Norway, Turkey, Israel and Russia. Iclusig is marketed in the U.S. by Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited.

On November 4, 2021 Magenta Therapeutics, Inc. (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines designed to bring the curative power of stem cell transplants to more patients, reported financial results for the third quarter ended September 30, 2021, and recent program highlights (Press release, Magenta Therapeutics, NOV 4, 2021, View Source [SID1234594376]).

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"We are pleased with our recent execution across the portfolio as we look to continue to allocate our capital efficiently to our value-creating opportunities," said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta Therapeutics. "In mid-September, we cleared the IND process for the MGTA-117 targeted conditioning program and are now conducting the clinical trial start-up activities with the continued expectation of opening the trial this year. The MGTA-145 stem cell mobilization program also continues to advance with a fully enrolled investigator-initiated trial in multiple myeloma patients, a Phase 2 clinical trial in allogeneic transplant and the expected start of a Phase 2 clinical trial evaluating mobilization and collection of stem cells from patients with sickle cell disease."

Business Highlights:

In October 2021, Magenta welcomed Jeffrey Humphrey, M.D. to its Executive Team as Chief Medical Officer.

Program Highlights:

MGTA-145 Stem Cell Mobilization and Collection

Recent and Planned Activity:

Autologous Stem Cell Transplant: Multiple Myeloma

Investigator-Initiated Phase 2 Clinical Trial Design, Topline Data and Next Steps.
Trial Design: Surbhi Sidana, M.D., Assistant Professor of Medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy at Stanford University School of Medicine led this investigator-initiated, Phase 2 open-label clinical trial. The trial evaluated the ability of MGTA-145, in combination with plerixafor, to mobilize stem cells for autologous stem cell transplantation in patients with multiple myeloma.
Top-Line Clinical Data in Poster Presentation at 2021 ASH (Free ASH Whitepaper) Annual Meeting: Top line clinical data from the fully enrolled investigator-initiated clinical trial will be included in a poster presentation at the ASH (Free ASH Whitepaper) Annual Meeting, held December 11-14, 2021. As disclosed separately, the clinical data showed that eighty-eight percent (88%) of patients (22/25) treated with MGTA-145 plus plerixafor met the primary endpoint of sufficient stem cell mobilization and collection for transplant. Also, as of the time of the data submission for the ASH (Free ASH Whitepaper) meeting, all patients (18/18) transplanted with stem cells mobilized by MGTA-145 plus plerixafor successfully engrafted. MGTA-145 plus plerixafor was well tolerated.
Next Steps in Multiple Myeloma: The results from this investigator-initiated trial represent a positive step forward in the development of MGTA-145, in combination with plerixafor, as a potential first line stem cell mobilization regimen. Based on the encouraging collection and engraftment data, the company intends to explore further development of MGTA-145 in a Phase 2b clinical setting. This approach would enable a comprehensive evaluation of the multiple myeloma patient population and allow for adjustments of dosing and administration which the company, in both cases, has identified as opportunities for optimization as a result of this investigator-initiated study and the company’s other MGTA-145 development efforts.
Allogeneic Stem Cell Transplant: Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL) and Myelodysplastic Syndromes (MDS)
Phase 2 Clinical Trial and Next Steps.
Trial Design. This Phase 2 clinical trial is designed to evaluate MGTA-145 in combination with plerixafor, in the mobilization and collection of stem cells from allogeneic donors for transplant in patients with AML, ALL and MDS.
Next Steps. Based on what Magenta has learned to date from the totality of the MGTA-145 program-related clinical trial data and other relevant information, Magenta believes it has identified an opportunity to optimize certain elements of the dosing and administration of the MGTA-145 mobilization regimen. Accordingly, Magenta intends to amend the Phase 2 allogeneic clinical trial to include a higher dose of MGTA-145 that matches the dose level used in the Phase 2 multiple myeloma clinical trial.
Stem Cell Mobilization of Patients with Sickle Cell Disease in Collaboration with bluebird bio. Magenta expects to open the Phase 2 clinical trial in December 2021. The trial is designed to evaluate the utility of MGTA-145 in combination with plerixafor, for the mobilization and collection of stem cells in patients with sickle cell disease where mobilization and collection is difficult and there is a clear unmet medical need.
MGTA-117 Targeted Conditioning

Recent and Planned Activity:

Phase 1/2 Clinical Trial Start-Up Activities Ongoing. The IND for the company’s MGTA-117 antibody-drug conjugate (ADC) targeted conditioning program is active with the U.S. Food and Drug Administration (FDA). The company expects to open the multi-center Phase 1/2 clinical trial in December 2021. The Phase 1/2 trial is designed to utilize dose escalating cohorts to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of MGTA-117 as a single dose with possible anti-tumor therapeutic benefit in patients with relapsed/refractory AML and MDS. As previously disclosed, Magenta expects to work with the FDA on an ongoing basis to transition the clinical trial to the primary target population of hematopoietic stem cell (HSC) transplant-eligible patients with AML and MDS after adequate data related to the safety, pharmacokinetics and pharmacodynamics of MGTA-117 have been collected in this initial patient population. As the program progresses, Magenta also plans to explore MGTA-117 as a targeted conditioning agent prior to the delivery of gene-corrected cells associated with stem cell gene therapy.
Oral Presentation at the 2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. As disclosed by Magenta in a separate press release, preclinical data showing that a single dose of a tool CD117 antibody drug conjugate (CD117-ADC) supports efficient engraftment of gene-modified CD34+ stem cells in a rhesus gene therapy model. The CD117-ADC utilized in this study had minimal toxicities unlike busulfan conditioning. The data will be the subject of an oral presentation at the ASH (Free ASH Whitepaper) Annual Meeting on December 13, 2021.
Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2021, were $192.6 million, compared to $148.8 million as of December 31, 2020. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into the third quarter of 2023.

Research and Development Expenses: Research and development expenses were $10.8 million in the third quarter of 2021, compared to $11.8 million in the third quarter of 2020. The decrease was driven primarily by the completion of the GMP manufacturing activities to support the IND application for MGTA-117 and future clinical trials offset by an increase in personnel related costs.

General and Administrative Expenses: General and administrative expenses were $7.5 million for the third quarter of 2021, compared to $6.6 million for the third quarter of 2020. The increase was primarily due to an increase in personnel related costs.

Net Loss: Net loss was $17.4 million for the third quarter of 2021, compared to net loss of $17.7 million for the third quarter of 2020.

On November 4, 2021 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that an abstract detailing new data from a Phase 2 study evaluating selinexor, a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound, in patients with myelofibrosis (MF) previously treated with JAK inhibition has been selected for an oral presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) 2021 Annual Meeting and Exposition in Atlanta, GA on December 11-14, 2021 (Press release, Karyopharm, NOV 4, 2021, View Source [SID1234594404]).

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"JAK inhibition is the current standard of care for patients with myelofibrosis; however, patients whose disease fails treatment with ruxolitinib have a poor prognosis, with an expected survival of approximately 14 months, and there are no approved treatment options other than JAK inhibitors," said Srinivas Tantravahi, MBBS, MRCP, University of Utah Hospital and principal investigator of the Phase 2 study. "Interim results from this Phase 2 study demonstrated that once weekly single agent oral selinexor resulted in compelling spleen volume reduction rates in myelofibrosis patients who received at least 24 weeks of treatment, with 33% of those patients achieving a response, defined as ≥35% SVR. In addition to spleen responses, there was improvement in anemia status and symptom scores in these patients. The responses were durable with the first patient on treatment for more than two years. The sustained responses and well-tolerated safety profile highlight selinexor’s potential in patients with myelofibrosis who have either progressed following ruxolitinib or cannot tolerate JAK inhibition. We look forward to sharing these exciting results with the broader medical and scientific community at ASH (Free ASH Whitepaper) this year."

"Once weekly, low-dose selinexor is an oral agent with a unique mechanism of action that has demonstrated strong single-agent activity in myelofibrosis patients with disease refractory to ruxolitinib," said Jatin Shah, MD, Chief Medical Officer of Karyopharm. "Importantly, there are no other classes of drug approved other than JAK inhibitors and a new class of effective drugs is a critical need for patients. Based on these encouraging data, we look forward to dosing the first patient in a new, company-sponsored Phase 2 study evaluating single-agent selinexor versus physician’s choice in patients with previously treated myelofibrosis during the fourth quarter of 2021."

Results from the Phase 2 Study Evaluating Selinexor in Patients with MF Refractory or Intolerant to JAK Inhibitors

The results were based on the open label, prospective, investigator-initiated single center study in adult patients with primary or secondary MF with resistance or intolerance to JAK inhibitor therapy (NCT03627403). Selinexor was administered orally at a dose of 80mg or 60mg once weekly to 12 patients. The primary endpoint of the study is to assess the efficacy of selinexor on SVR. Median duration of prior JAK inhibitor therapy was 22 months and 11 out of 12 patients had MF refractory to ruxolitinib.

As of the data cutoff, the median duration of treatment was 36 weeks. In the nine patients who were on treatment for over 24 weeks, SVR of ≥25% and 35% occurred in four (44%) and three (33%) patients, respectively. The most common treatment related adverse event was weight loss (grade 2 in four patients and grade 3 in one patient). This was manageable with treatment interruption and dose reduction, except in one patient who discontinued treatment. Overall, selinexor demonstrated single-agent activity with sustained spleen responses in patients with JAK inhibitor refractory MF and long-term administration of selinexor was well tolerated. Updated data will be presented at the meeting.

Company to Host Investor Day Event

Karyopharm will host an Investor Day event on Wednesday, December 8, 2021 from 10:00 a.m. to 12:30 p.m. ET to outline its commercial and pipeline priorities and objectives. The event will feature presentations from Karyopharm management and recognized thought leaders in multiple myeloma, gynecological malignancies, and other core focus indications. The event will take place virtually and will be accessible via conference call and webcast. Full details will be made available closer to the Investor Day.

Details for the ASH (Free ASH Whitepaper) 2021 abstracts are as follows:

In total, 17 abstracts were selected for presentation at the meeting, including five oral presentations and 12 posters.

Oral Presentations

Title: A Phase 2 Study to Evaluate the Efficacy and Safety of Selinexor in Patients with Myelofibrosis Refractory or Intolerant to JAK Inhibitors
Presenter: Srinivas Tantravahi, University of Utah
Abstract #: 143
Session Type: Oral Presentation
Session: Myeloproliferative Syndromes: Clinical and Epidemiological: Non-JAK Inhibitor Therapies for Myelofibrosis
Date and Time: Saturday, December 11, 2021 at 1:00 p.m. ET

Title: Transcriptomic Correlates of Response to Selinexor in Multiple Myeloma Reveal a Predictive Signature
Presenter: Paula Restrepo, Icahn School of Medicine at Mount Sinai
Abstract #: 457
Session Type: Oral Presentation
Session: Multiple Myeloma and Plasma Cell Dyscrasias: Clinical and Epidemiological: Multiple Myeloma and Waldenstrom Macroglobulinemia: Exploring Biomarkers in the Era of Personalized Medicine
Date and Time: Sunday, December 12, 2021 at 12:00 p.m. ET

Title: Enhanced p53 Activation by Dual Inhibition of MDM2 and XPO1 Disrupts MYC Transcriptional Program and Restores Sensitivity to BCL-2 Inhibition in Ven/HMA Resistant AML
Presenter: Yuki Nishida, Saga University
Abstract #: 505
Session Type: Oral Presentation
Session: Molecular Pharmacology and Drug Resistance: Myeloid Neoplasms: Novel Strategies to Overcome Resistance to BCL-2 Inhibition
Date and Time: Sunday, December 12, 2021 at 4:30 p.m. ET

Title: Rationale for Selinexor Treatment in Daratumumab-Refractory MM Patients Identified by Paired Ex Vivo Drug Sensitivity and RNA-Seq
Presenter: Suresh Kumar Balasubramanian, Wayne State University
Abstract #: 683
Session Type: Oral Presentation
Session: Molecular Pharmacology and Drug Resistance: Lymphoid Neoplasms: Targeting
Mitochondrial Survival Pathways
Date and Time: Monday, December 13, 2021 at 3:45 p.m. ET

Title: Comparison of Salvage Autologous Hematopoietic Cell Transplantation with Outcomes Following Selinexor Combinations Among Double/Triple Refractory Myeloma Patients
Presenter: Praneeth Sudalagunta, H Lee Moffitt Cancer Ctr
Abstract #: 893
Session Type: Oral Presentation
Session: Multiple Myeloma and Plasma Cell Dyscrasias: Basic and Translational: Myeloma Pathogenesis and Novel Targets
Date and Time: Monday, December 13, 2021 at 7:15 p.m. ET

Poster Presentations

Title: Efficacy and Safety of Selinexor-Containing Regimens in Patients with Multiple Myeloma Previously Treated with Anti-CD38 Monoclonal Antibodies (αCD38 mAb)
Presenter: Suzanne Lentzsch, Columbia University Irving Medical Center
Abstract #: 1651
Session Type: Poster Presentation
Session: Myeloma and Plasma Cell Dyscrasias: Clinical-Prospective Therapeutic Trials: Poster I
Date and Time: Saturday, December 11, 2021 at 5:30 – 7:30 p.m. ET

Title: Effects of Cytogenetic Risk on Outcomes in Multiple Myeloma Treated with Selinexor, Bortezomib, and Dexamethasone (XVd)
Presenter: Nizar Bahlis, University of Calgary
Abstract #: 1634
Session Type: Poster Presentation
Session: Multiple Myeloma and Plasma Cell Dyscrasias: Clinical and Epidemiological: Poster I
Date and Time: Saturday, December 11, 2021 at 5:30 – 7:30 p.m. ET

Title: Selinexor in Combination with Daratumumab-Bortezomib and Dexamethasone for the Treatment of Relapse or Refractory Multiple Myeloma: Initial Results of the Phase 2, Open-label, Multicenter GEM-SELIBORDARA Study
Presenter: Paula Rodríguez- Otero, Clínica Universidad de Navarra
Abstract #: 1677
Session Type: Poster Presentation
Session: Myeloma and Plasma Cell Dyscrasias: Clinical-Prospective Therapeutic Trials: Poster I
Date and Time: Saturday, December 11, 2021 at 5:30 – 7:30 p.m. ET

Title: Once Weekly Oral Selinexor, Pomalidomide, and Dexamethasone in Relapsed Refractory Multiple Myeloma
Presenter: Darrell White, QEII Health Sciences Center, Dalhousie University
Abstract #: 2748
Session Type: Poster Presentation
Session: Myeloma and Plasma Cell Dyscrasias: Clinical-Prospective Therapeutic Trials: Poster II
Date and Time: Sunday, December 12, 2021 at 6:00 – 8:00 p.m. ET

Title: Selinexor-Based Regimens in Patients with Multiple Myeloma after Prior Anti-B-Cell Maturation Antigen Treatment
Presenter: Muhamed Baljevic, University of Nebraska Medical Center
Abstract #: 2751
Session Type: Poster Presentation
Session: Myeloma and Plasma Cell Dyscrasias: Clinical-Prospective Therapeutic Trials: Poster II
Date and Time: Sunday, December 12, 2021 at 6:00 – 8:00 p.m. ET

Title: Single Cell RNA Sequencing of a Selinexor Clinical Trial Reveals Overexpression of Alternative Nuclear Export Pathways Associated with Resistance to Selinexor in RRMM Patients
Presenter: Yael Cohen, Tel Aviv Sourasky Medical Center
Abstract #: 2725
Session Type: Poster Presentation
Session: Multiple Myeloma and Plasma Cell Dyscrasias: Clinical and Epidemiological: Poster I
Date and Time: Sunday, December 12, 2021 at 6:00 – 8:00 p.m. ET

Title: Selinexor Enhances NK Cell Activation Against Lymphoma Cells Via Downregulation of HLA
Presenter: Matthew Blunt, University of Southampton
Abstract #: 2411
Session Type: Poster Presentation
Session: Lymphomas: Translational—Non-Genetic: Poster II
Date and Time: Sunday, December 12, 2021 at 6:00 – 8:00 p.m. ET

Title: Molecular Response Patterns in Relapsed/Refractory AML Patients Treated with Selinexor and Chemotherapy
Presenter: Piroska Klement, Hannover Medical School
Abstract #: 2369
Session Type: Poster Presentation
Session: Acute Myeloid Leukemias: Biomarkers, Molecular Markers and Minimal Residual Disease in Diagnosis and Prognosis: Poster II
Date and Time: Sunday, December 12, 2021 at 6:00 – 8:00 p.m. ET

Title: Updated Efficacy of Eltanexor Monotherapy in Patients with Higher Risk Hypomethylating Myelodysplastic Syndrome Primary Refractory to Hypomethylating Agents
Presenter: Sangmin Lee, Weill Cornell Medical College
Abstract #: 3676
Session Type: Poster Presentation
Session: Myelodysplastic Syndromes — Clinical and Epidemiological: Poster III
Date and Time: Monday, December 13, 2021 at 6:00 – 8:00 p.m. ET

Title: Clinical Outcomes in Patients with Dose Reduction of Selinexor in Combination with Bortezomib, and Dexamethasone (XVd) in Previously Treated Multiple Myeloma from the BOSTON Study
Presenter: Sundar Jagannath, Mount Sinai School of Medicine
Abstract #: 3793
Session Type: Poster Presentation
Session: Multiple Myeloma and Plasma Cell Dyscrasias: Clinical and Epidemiological: Poster III
Date and Time: Monday, December 13, 2021 at 6:00 – 8:00 p.m. ET

Title: Selinexor in Combination with R-GDP for Patients with Relapsed/Refractory B-Cell Lymphoma: SELINDA Phase Ib LYSA Study
Presenter: Marie Maerevoet, Jules Bordet Institute
Abstract #: 1411
Session Type: Poster Presentation
Session: Aggressive Lymphomas: Prospective Therapeutic Trials: Poster I
Date and Time: Saturday, December 11, 2021 at 5:30 – 7:30 p.m. ET

Title: A Phase 2/3, Multicenter Randomized Study of Rituximab-Gemcitabine-Dexamethasone-Platinum (R-GDP) with or without Selinexor in Patients with Relapsed/Refractory Diffuse Large B-Cell Lymphoma (RR DLBCL)
Presenter: Seung Tae Lee, University of Maryland School of Medicine
Abstract #: 1420
Session Type: Poster Presentation
Session: 626. Aggressive Lymphomas: Prospective Therapeutic Trials: Poster I
Date and Time: Saturday, December 11, 2021, 5:30-7:30PM ET

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). XPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion. Karyopharm received accelerated U.S. Food and Drug Administration (FDA) approval of XPOVIO in July 2019 in combination with dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. NEXPOVIO (selinexor) has also been granted conditional marketing authorization for adult patients with heavily pretreated multiple myeloma by the European Commission. Karyopharm’s supplemental New Drug Application (sNDA) requesting an expansion of its indication to include the treatment for patients with multiple myeloma after at least one prior therapy was approved by the FDA on December 18, 2020. In June 2020, Karyopharm received accelerated FDA approval of XPOVIO for its second indication in adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple cancer indications, including as a potential backbone therapy in combination with approved myeloma therapies (STOMP) and in endometrial cancer (SIENDO), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with approved therapies in a variety of tumor types to further inform Karyopharm’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at:

Tel: +1 (888) 209-9326
Email: [email protected]

XPOVIO (selinexor) is a prescription medicine approved:

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).
In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti–CD38 monoclonal antibody (Xd).
For the treatment of adult patients with relapsed or refractory diffuse large B–cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.
Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony–stimulating factors.
Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.
Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.
Serious Infection: Monitor for infection and treat promptly.
Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.
Embryo–Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.
Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3–4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.
The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.
The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3–4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.
Use In Specific Populations
Lactation: Advise not to breastfeed.

For additional product information, including full prescribing information, please visit www.XPOVIO.com.