MDS is a bone marrow failure disorder
Recent News

Onconova is evaluating oral rigosertib in combination with azacitidine in a Phase 2 trial for patients with 1st-line MDS/AML.

Phase 2 09-08 Trial (NCT01926587)

A Phase 1/2 study of the combined administration of oral rigosertib with azacitidine in patients with myelodysplastic syndrome or acute myeloid leukemia.

Status: Enrolling


The Pivotal MDS Trial INSPIRE is Now Recruiting Patients

INternational Study of Phase III Intravenous RigosErtib

STUDY DESCRIPTION
A Phase III, international, randomized, controlled study of Rigosertib + best supportive care versus physician’s choice of treatment + best supportive care in patients with myelodysplastic syndrome (MDS) after failure of a hypomethylating agent (HMA).

PRIMARY ENDPOINTS
Overall survival in the intention-to-treat population and in patients with very high risk per the Revised
International Prognostic Scoring System (Greenberg et al, Blood 2012).

INTERNATIONAL TRIAL
More than 150 trial sites.

For additional information on this study, please call the INSPIRE help line at 1-267-759-3676 or visit www.clinicaltrials.gov, identifier: NCT02562443.

Rigosertib is an investigational agent and is not approved by the FDA or other regulatory agencies worldwide as a treatment for any indication.


Sage Bionetworks and Celgene Corporation announce the launch of Journey PRO, a mobile health research study to improve the lives of Chronic Anemia Patients

For Release:
Monday, August 14, 2017

Sage Bionetworks and Celgene Corporation announce the launch of Journey PRO, a mobile health research study to improve the lives of Chronic Anemia Patients

 

Smartphone-based app as a tool to collect and analyze patient-reported data leading to improved therapeutic developments and to provide study information back to the patient participants to use in managing their health.

Seattle, WA – Sage Bionetworks, a non-profit biomedical research accelerator, together with Celgene Corporation (NASDAQ: CELG) today announced the launch of Journey PRO, a mobile health research study designed to improve the understanding of disease burden on people living with chronic anemia due to myelodysplastic syndromes (MDS), myelofibrosis, and beta-thalassemia.

This study uses mobile and wearable technologies to quantify the daily burden of chronic anemia on patients living with these diseases. The study utilizes the Apple ResearchKit framework to collect data from participants using surveys, neurological self-assessments using the BrainBaseline cognitive testing software from Digital Artifacts, health data collected from the iPhone HealthKit, and fitness tracking wearables, among others. By following participants using these quantitative assessments, we aim to develop a tool to evaluate the efficacy of new treatments for reducing the impact of these diseases on patients.

“Understanding the impact of disease on daily living is important to patients as well as researchers,” said Lara Mangravite, President of Sage Bionetworks and PI of the study. “For this reason, Journey PRO will provide direct and immediate information back to research participants to help them manage their health.” The Journey PRO app was designed with input from members of the chronic anemia community to support patients in the management of their health. Participants may use the app to track key health data (e.g., transfusions, lab values) and the scheduling of appointment dates. Participants will also be able to visualize their study data and may choose to download the data and share it with their healthcare team. This design was developed in consultation with patient representatives recruited through the MDS Foundation (www.mds-foundation.org), Cooley’s Anemia Foundation (www.thalassemia.org), and the Leukemia and Lymphoma Society (www.lls.org).

Journey PRO is open to participants over the age of 18 living in the United States with an iPhone model 5 or newer with iOS 8 or later. The research term encourages patients with a diagnosis of myelodysplastic syndromes (MDS), myelofibrosis, and beta-thalassemia to participate in this study. Individuals without a diagnosis of chronic anemia can also participate by providing valuable comparison data to further illustrate the difference in quality of life measures between the chronic anemia population and the general population. The Journey PRO app is available immediately for download from the App Store.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit www.celgene.com. Follow Celgene on Social Media: @CelgenePinterestLinkedInFacebook and YouTube.

About Sage Bionetworks (www.sagebase.org)

Sage Bionetworks is a nonprofit biomedical research organization, founded in 2009, with a vision to promote innovations in personalized medicine by enabling a community-based approach to scientific inquiries and discoveries. In pursuit of this Mission, Sage Bionetworks is working with others to assemble an information Commons for biomedicine that (1) is supported by an open compute space (Synapse: www.synapse.org), (2) supports open research collaborations and innovative DREAM Challenges, and (3) empowers citizens and patients with the tools to partner with researchers and share their data through Sage’s BRIDGE platform (http://sagebase.org/bridge/) in order to drive the research studies that matter most to them.

Contact: Lara Mangravite
lara.mangravite@sagebase.org
206 667-6044

Diane Gary
Diane.gary@sagebase.org
206-667-2102


PEVONEDISTAT-2001

headerimage

 

PEVONEDISTAT-2001

Takeda Pharmaceuticals International Co. is currently enrolling patients for a Phase 2 clinical trial of the study drug pevonedistat. The purpose of this study is to evaluate the efficacy and safety of pevonedistat plus azacitidine versus single-agent azacitidine in participants with higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia and low-blast acute myelogeneous leukemia.

This study will look at the overall survival, event-free survival and response to treatment in people who take pevonedistat and azacitidine compared to people who take single-agent azacitidine.

This multi-center trial will be conducted worldwide and enroll approximately 117 participants.

Patients may qualify for this study if:

  • 18 years of age or older
  • Patients have intermediate, high, or very high-risk MDS based on the Revised International Prognostic Scoring System (IPSS-R), a standard prognostic tool, or have CMML
  • Patients have low-blast AML defined as 20% to 30% myeloblasts in the bone marrow (low-blast AML), and ≤30% myeloblasts in the peripheral blood and considered appropriate for azacitidine based therapy

In order to refer a patient with MDS, CMML, or low-blast AML for enrollment to this study and review eligibility criteria, physicians/health care providers should visit www.clinicaltrials.gov(NCT02610777)

Contact: 1-877-674-3784; globaloncologymedinfo@takeda.com

Pevonedistat is an investigational agent and is not approved by the FDA
or other regulatory agencies worldwide as a treatment for any indication.

Takeda Oncology Logo


Updated Data from Phase 1 Trial of Oral IDHIFA® (enasidenib) Demonstrate Complete Responses and Duration of Response in Patients with Relapsed or Refractory AML and an IDH2 Mutation

celgene

40.3% Overall Response Rate (ORR) with Median Duration of Response of 5.8 Months and 19.3% Complete Response (CR) Rate with Median Duration of Response of 8.8 Months in Patients With a CR

Overall Safety Profile was Consistent with Previously Reported Data

Simultaneous Online Publications of Clinical and Translational Data Presented in Journal Blood

CHICAGO – – (BUSINESS WIRE) – – Celgene Corporation (NASDAQ:CELG) and Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) today announced new efficacy and safety data from the ongoing Phase 1 dose-escalation and expansion study evaluating investigational oral IDHIFA® (enasidenib) in patients with relapsed or refractory acute myeloid leukemia (R/R AML) and an isocitrate dehydrogenase-2 (IDH2) mutation. IDHIFA is an investigational first-in-class, oral, targeted inhibitor of the mutant IDH2 enzyme, which demonstrated an overall response rate of 40.3 percent, including a complete response rate of 19.3 percent in the study. The data were presented in an oral session at the American Society of Clinical Oncology (ASCO) Annual Meeting and simultaneously published online in the journa Blood.*

“The updated results, including duration of response, from the Phase 1 study reinforce the potential for enasidenib as a first-in-class therapy for patients with relapsed or refractory AML and an IDH2 mutation,” said Michael Pehl, President, Hematology/Oncology at Celgene. “Patients have very few treatment options for relapsed or refractory AML, so we are eager to advance this potential targeted therapy as quickly as possible.”

As of April 15, 2016, a total of 239 patients with advanced hematologic malignances and an IDH2 mutation were enrolled into the Phase 1 study, of which 176 patients had R/R AML. Data reported include patients receiving enasidenib at total daily doses ranging from 50 mg to 650 mg in the dose-escalation arm and 100 mg once daily in the Phase 1 expansion arms. A maximum tolerated dose was not reached. The median age of the patients enrolled in the study is 70 (ranging from 19-100). Patients with R/R AML received a median of two prior lines of therapy (ranging from one to 14).

The overall safety profile observed for enasidenib was consistent with previously reported data. Twenty-four percent of patients had treatment-related serious adverse events (SAEs), notably IDH differentiation syndrome (8%), leukocytosis (4%), tumor lysis syndrome (3%) and hyperbilirubinemia (2%). The most common treatment-emergent AEs were nausea (46%) hyperbilirubinemia (45%), diarrhea (40%) and fatigue (40%).

Data from 176 R/R AML patients with an IDH2 mutation demonstrated a 40.3 percent (71 of 176 patients) overall response rate, which was the primary endpoint of the study. Further, the complete response rate was 19.3 percent (34 of 176 patients). Median duration of response was 5.8 months [95% CI 3.9, 7.4] for all patients who responded and 8.8 months [95% CI 6.4, NR] for patients who achieved a CR. Median time to first response was 1.9 months (0.5-9.4) and median time to CR was 3.8 months (0.5-11.2). Median overall survival (OS) for R/R AML patients as observed in the study was 9.3 months [95% CI 8.2, 10.9]. Additional results including qualitative improvement in response over time, improvement in hematological parameters over time, OS for patients achieving a CR and transfusion independence were also reported.

“In addition to the complete response in this study, we also observed changes in responses and hematologic parameters over time,” said Eytan Stein, M.D., lead investigator and attending physician in the leukemia service at Memorial Sloan Kettering Cancer Center. “This suggests that differentiation of myeloblasts – made possible by inhibition of mutated IDH2 – may drive the clinical efficacy of enasidenib.”

“Targeting IDH mutations is thought to allow for the differentiation of malignant cells and introduces a new paradigm in the treatment of AML,” said Chris Bowden, M.D., chief medical officer of Agios. “These data show that IDH inhibition plays an important role in segments of AML and will continue to inform our research into this novel class of potential therapies.”

Additional Data Available – IDH Differentiation Syndrome & Translational Analyses
A separate analysis of IDH-inhibitor-associated differentiation syndrome (IDH-DS) associated with enasidenib was also presented as a poster discussion during the ASCO meeting and detailed the findings of an independent Differentiation Syndrome Review Committee (DSRC). The committee reviewed investigator reported IDH-DS cases and determined that 13 of the 27 potential cases were consistent with IDH-DS (11.9% of 109 patients). These data demonstrate that the signs and symptoms of IDH-DS are recognizable. IDH-DS represents a novel clinical finding in patients with mutated IDH2 AML treated with enasidenib, and is likely due to its purported mechanism of action, differentiation of leukemic cells.

In addition to the clinical data publication, additional analyses describing the mechanism of action of enasidenib were also published online in Blood. An analysis of patient samples confirmed that the preclinical efficacy and mechanism of action of mutated IDH2 inhibition by enasidenib is through differentiation of AML cells. The authors conclude that the data provide insights into enasidenib resistance to inform future mechanism-based combination treatment studies.

Clinical Development
Enasidenib continues to be studied in the following ongoing clinical trials:

  • Phase III IDHENTIFY study evaluating the efficacy and safety of enasidenib versus conventional care regimens in older patients with R/R AML with an IDH2 mutation (NCT02577406)
  • Phase 1b study of either enasidenib or ivosidenib in combination with standard induction and consolidation chemotherapy in newly diagnosed AML (NCT02632708)
  • Phase 1/2 study of either enasidenib or ivosidenib in combination with azacitidine in newly diagnosed AML (NCT02677922)

The New Drug Application (NDA) for IDHIFA is currently under Priority Review with the U.S. Food and Drug Administration for the treatment of patients with relapsed or refractory AML with an IDH2 mutation. The NDA has been given a Prescription Drug User Fee Act (PDUFA) action date of Aug. 30, 2017.

Ivosidenib (AG-120, wholly owned by Agios) is an investigational, oral, targeted inhibitor of the mutant IDH1 enzyme.

About AG221-C-001

Study AG221-C-001 includes three parts: a Phase 1 dose escalation, a part 1 (Phase 1) expansion and a Phase 2 expansion.

The Phase 1 dose escalation study was designed to determine the maximum tolerated dose and recommended Phase 2 dose, and to evaluate efficacy and safety of enasidenib (AG-221/CC-90007) in subjects with advanced hematologic malignancies with an IDH2 mutation. The Part 1 expansion further evaluated the safety, tolerability, and efficacy of enasidenib in subjects with R/R AML, untreated AML, myelodysplastic syndrome or other advanced hematologic malignancies with an IDH2 mutation. Based on the clinical activity observed in R/R AML subjects, the Phase 2 expansion was designed to assess efficacy of enasidenib at recommended 100 mg daily dose and to further evaluate safety in subjects with R/R AML and with IDH2 mutation. The study was not designed or statistically powered to reach a conclusion on OS. A phase 3 randomized controlled trial with OS as a primary endpoint has been initiated.

About Acute Myelogenous Leukemia (AML)

AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia affecting adults. Undifferentiated blast cells proliferate in the bone marrow rather than mature into normal blood cells. AML incidence significantly increases with age, and according to the American Cancer Society, the median age of onset is 66. The vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML. The five-year survival rate for AML is approximately 20 to 25 percent. IDH2 mutations are present in about 8 to 19 percent of AML cases.

About Agios

Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has multiple first-in-class investigational medicines in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit the company’s website at www.agios.com.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit www.celgene.com. Follow Celgene on Social Media: @CelgenePinterestLinkedInFacebook and YouTube.

About Agios/Celgene Collaboration

IDHIFA® (enasidenib) and AG-881 are part of Agios’ global strategic collaboration with Celgene Corporation focused on cancer metabolism. Under the terms of the 2010 collaboration agreement, Celgene has worldwide development and commercialization rights for IDHIFA. Agios continues to conduct clinical development activities within the IDHIFA development program and is eligible to receive reimbursement for those development activities and up to $95 million in remaining payments assuming achievement of certain milestones and royalties on net sales. Celgene and Agios intend to co-commercialize IDHIFA in the U.S. Celgene will reimburse Agios for costs incurred for its co-commercialization efforts.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similar expressions. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections, and speak only as of the date they are made. Neither Celgene nor Agios undertake any obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond each company’s control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in the Annual Report on Form 10-K and other reports of each company filed with the Securities and Exchange Commission.

Hyperlinks are provided as a convenience and for informational purposes only. Neither Celgene nor Agios bears any responsibility for the security or content of external websites.

Celgene
Investors:
+1-908-673-9628
ir@celgene.com
or
Media:
+1-908-673-2275
media@celgene.com
or
Agios
Investors:

Kendra Adams, 617-844-6407
Senior Director, Investor & Public Relations
Kendra.Adams@agios.com
or
Renee Leck, 617-649-8299
Senior Manager, Investor & Public Relations
Renee.Leck@agios.com
or
Media:
Holly Manning, 617-844-6630
Associate Director, Corporate Communications
Holly.Manning@agios.com

Source: Celgene Corporation

News Provided by Acquire Media


Celgene Press Release

celgeneagios

CELGENE AND AGIOS ANNOUNCE COLLABORATIONS WITH ABBOTT FOR
DIAGNOSTIC IDENTIFICATION OF IDH MUTATIONS IN AML

Companion diagnostic technology to be utilized with enasidenib (AG-221/CC-90007) and AG-120 development programs for relapsed/refractory acute myeloid leukemia (AML)

Approximately 20% of AML patients have an IDH mutation

SUMMIT, NJ and Cambridge, Mass. – (Oct. 12, 2016) – Celgene Corporation (NASDAQ: CELG) and Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) today announced each company has entered into collaboration agreements with Abbott (NYSE: ABT), a leader in diagnostic technologies, to develop and commercialize companion diagnostic tests on Abbott’s m2000 RealTime System to identify isocitrate dehydrogenase (IDH) mutations in acute myeloid leukemia (AML) patients. Celgene is currently developing enasidenib (AG-221/CC-90007), an IDH2 mutant inhibitor, for the treatment of patients with relapsed or refractory AML who have an IDH2 mutation. Agios is developing AG-120, an IDH1 mutant inhibitor, for the treatment of patients with relapsed or refractory AML who have an IDH1 mutation.

IDH1 and IDH2 mutations occur in approximately 20% of AML patients. An article published online this week in the journal Leukemia (Medeiros, Leukemia 2016) concluded that advances in the understanding of the genetics underlying myeloid malignancies are driving an era of development for targeted treatments such as IDH mutant inhibitors. The authors recommend that IDH mutational analysis should become part of the routine AML diagnostic workup and repeated at relapse to identify patients who may be eligible for targeted investigational treatments currently under clinical study.

“AML is a complex and heterogeneous disease, making it difficult to treat,” said Han Myint, M.D., Vice President, Global Medical Affairs, Myeloid for Celgene. “IDH mutations lead to aberrant DNA methylation, causing a block in myeloid differentiation that leads to disease progression. Molecular profiling is important to identify genomic mutations which may have prognostic and potential treatment implications for patients with AML.”

Abbott’s m2000rt RealTime System, is a polymerase chain reaction (PCR) instrument designed to enable clinical laboratories to automate PCR and results analysis, simplifying the complex and manual steps often associated with molecular diagnostics. Both Celgene and Agios have incorporated this screening into clinical trial designs, including the recently initiated Phase 3 IDHENTIFY trial comparing enasidenib with conventional therapy in older patients with an IDH2 mutation and relapsed or refractory AML (NCT02577406).

“The field of personalized medicine is advancing at a rapid pace for a broad range of medical conditions, especially within hematology-oncology,” said Chris Bowden, M.D., chief medical officer at Agios. “Our collaboration with Abbott will provide a test to help identify AML patients with IDH mutations who are in need of treatment options.”

The m2000 system has not been FDA cleared or approved for use with enasidenib or AG-120.
Enasidenib and AG-120 have not been approved for any use in any country.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit www.celgene.com. Follow Celgene on Social Media: @Celgene, Pinterest, LinkedIn, FaceBook and YouTube.

About Agios
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic metabolic disorders through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has multiple first-in-class investigational medicines in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit the company’s website at www.agios.com.

Forward-Looking Statements

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similar expressions. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections, and speak only as of the date they are made. Neither Celgene nor Agios undertake any obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond each company’s control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in the Annual Report on Form 10-K and other reports of each company filed with the Securities and Exchange Commission.

# # #

Contacts:

For Celgene:
Investors:
(908) 673-9628 investors@celgene.com

Media:
(908) 673-2275 media@celgene.com

For Agios:
Kendra Adams, 617-844-6407
Senior Director, Investor & Public Relations
Kendra.Adams@agios.com

Renee Leck, 617-649-8299
Senior Manager, Investor & Public Relations
Renee.Leck@agios.com


CELGENE CORPORATION AND SAGE BIONETWORKS ANNOUNCE TECHNOLOGY COLLABORATION – Press Release

celgene

Contacts:

For Celgene:

Investors:
(908) 673-9628
investors@celgene.com

Media:
(908) 673-2275
media@celgene.com

For Sage:
Media: (206) 667-3192

CELGENE CORPORATION AND SAGE BIONETWORKS ANNOUNCE TECHNOLOGY COLLABORATION TO DEVELOP OBSERVATIONAL STUDY USING THE APPLE RESEARCHKIT FRAMEWORK

Observational study to examine the burden of chronic anemia in myelodysplastic syndromes and beta-thalassemia

SUMMIT, NJ and SEATTLE, Wash.– (Oct. 18, 2016) – Celgene Corporation (NASDAQ: CELG) and Sage Bionetworks today announced a collaboration to develop an iPhone application utilizing the Apple ResearchKit framework to improve the understanding of the burden of disease for patients living with chronic anemia due to myelodysplastic syndromes (MDS) or beta-thalassemia.

Smartphone-based apps like Sage Bionetworks’ mPower for Parkinson’s disease are increasingly being utilized as a tool by clinical study researchers to collect and analyze increasing volumes of patient-reported data in order to better capture and understand disease burden better and to improve therapeutic developments. Smartphone clinical study apps also enable the return communication of important study information to the patient participants.

“We stand at a point where technology is unlocking the ability to capture patient reported outcomes,” said Michael Pehl, President, Hematology & Oncology for Celgene. “Through our collaboration with Sage Bionetworks and the evolving capability of smartphones and wearables as robust data collection devices, we believe we will be able to provide important new insights for patients with MDS and beta-thalassemia. We are pleased to be working alongside Sage Bionetworks and the patient community on this important project.”

Celgene and Sage Bionetworks have chosen to address chronic anemia caused by myelodysplastic syndromes and beta-thalassemia. These diseases impose a great burden on affected individuals that is difficult to understand and quantify, and typically have clinical endpoints outside traditional measures. The collaboration seeks to move to a paradigm where this and other information like physiological testing are collected on a multi-dimensional and regular basis.

Already a leader in MDS, Celgene is currently developing three assets in the clinic across myelodysplastic syndromes and beta-thalassemia (CC-486, luspatercept, and enasidenib (AG-221/CC-90007)).

In addition to helping collect difficult to quantify data, the new mobile study will collect neurological assessments of patients using cognitive testing software from BrainBaseline, a leading technology for the self-assessment of cognitive performance.  The app will also be an important channel for two-way communication and support for patients living with their disease – allowing them to understand their physical functioning and other symptoms of anemia.

Dr. Lara Mangravite, President of Sage Bionetworks stated, “We are thrilled to partner with Celgene to explore the use of sensor-based technologies to quantify the daily burden of disease in patients with chronic anemia.  This is a first of its kind exploration from which we hope to gain insights that can be used to understand the impact of chronic anemia.”

Celgene and Sage Bionetworks are working closely with the MDS Foundation (www.mds-foundation.org) and Cooleys Anemia Foundation (www.thalassemai.org) in defining the right elements for capture in the application to ensure patient relevance and applicability.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit www.celgene.com. Follow Celgene on Social Media: @CelgenePinterestLinkedInFaceBook and YouTube.

About Sage Bionetworks (www.sagebase.org) 

Sage Bionetworks is a nonprofit biomedical research organization, founded in 2009, with a vision to promote innovations in personalized medicine by enabling a community-based approach to scientific inquiries and discoveries. In pursuit of this Mission, Sage Bionetworks is working with others to assemble an information Commons for biomedicine that (1) is supported by an open compute space (Synapse: www.synapse.org), (2) supports open research collaborations and innovative DREAM Challenges, and (3) empowers citizens and patients with the tools to partner with researchers and share their data through Sage’s BRIDGE platform (http://sagebase.org/bridge/) in order to drive the research studies that matter most to them.

Forward-Looking Statements

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similar expressions. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections, and speak only as of the date they are made. Celgene undertakes no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond Celgene’s control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in Celgene’s Annual Report on Form 10-K and other reports filed with the Securities and Exchange Commission.


Onconova Enrolls First Patient in Europe for Phase 3 INSPIRE Trial of Rigosertib in Higher-Risk Myelodysplastic Syndromes

NEWTOWN, Pa., March 21, 2016 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ:ONTX), a clinical- stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, today announced the enrollment of the first European patient in Salzburg, Austria for the Phase 3 INSPIRE trial for IV rigosertib as a treatment for higher-risk myelodysplastic syndromes (HR-MDS) following failure of hypomethylating agent (HMA) therapy. The first patient in this global trial was enrolled at the MD Anderson Cancer Center in December 2015.

Read Press Release Here


Syros Press Release

CAMBRIDGE, Mass., September 22, 2016 – Syros Pharmaceuticals (NASDAQ: SYRS) announced today that the first patient has been dosed in the Phase 2 clinical trial of its lead drug candidate, SY-­1425, a first-­in-­class selective retinoic acid receptor alpha (RARα) agonist, in genomically defined subsets of patients with relapsed or refractory acute myeloid leukemia (AML) or high-­risk myelodysplastic syndrome (MDS) identified using a novel biomarker discovered by its gene control platform.

Read Press Release Here


Clinical Study – IMerge Lower-Risk MDS

IMERGE

Current Status: Part 1 recruitment complete; Part 2 is not yet open for recruitment. For additional details, refer to the Geron press release (12Sep2016) at http://ir.geron.com/phoenix.zhtml?c=67323&p=irol-newsArticle&ID=2201055.

Janssen Research & Development, LLC is conducting a Phase 2/3 clinical study referred to as “IMerge”, with the study drug Imetelstat, which is a first-in-class telomerase inhibitor. With its novel mechanism of action, Imetelstat may provide clinical benefit to MDS patients. In this study, Imetelstat is administered as a 2-hour intravenous infusion every 28 days.

IMerge is a study for people with MDS who need blood transfusions due to anemia (low red blood cell counts). People with low or intermediate-1 risk MDS that has relapsed or is refractory to Erythropoiesis-Stimulating Agents (ESAs) treatment are enrolled in the study. This study is being conducted at multiple hospitals and institutions around the world, in approximately 80 sites globally.

For more information about this clinical study, please visit www.clinicaltrials.gov (NCT02598661).


Lenalidomide Improves Quality of Life in Non-Del5q MDS

Treatment with lenalidomide (Revlimid) improved health-related quality of life (HRQoL) compared with placebo after 24 weeks for low to intermediate risk patients with non-deletion 5q myelodysplastic syndromes (MDS), according to a secondary endpoint analysis of the MDS-005 trial presented at the 2015 International MDS Symposium.

Results from MDS-005, originally presented at the 2014 ASH Annual Meeting, demonstrated that significantly more patients treated with lenalidomide achieved red blood cell transfusion independence (RBC-TI) of at least 56 days compared with placebo (26.9% [43/160 patients] vs 2.5% [2/79 patients]; P <.001), the primary endpoint of the multicenter randomized placebo-controlled study.

Based on the results of MDS-005, Celgene plans to submit a regulatory filing with the FDA in the second half of 2015.

In the current analysis, presented by Valeria Santini, MD, HRQoL was assessed as a secondary endpoint using the European Organization for Research and Treatment of Cancer QLQ-C30 questionnaire at baseline, every 12 weeks thereafter, and at discontinuation.

The questionnaire gathered data on fatigue, dyspnea, physical functioning, emotional functioning and global quality of life from 122 patients who received lenalidomide and 56 who received placebo. All patients included in the study had low or intermediate risk, transfusion dependent MDS that was non-del-5q, and were unresponsive or refractory to erythropoiesis-stimulating agents.

Patients underwent a baseline HRQoL assessment. At week 12, mean changes in HRQoL scores from baseline were not significantly different between treatment arms for the preselected domains. However, by week 24, HRQoL score changes favored lenalidomide versus placebo for all preselected domains. After adjusting for baseline scores, improvement was statistically significant for emotional functioning (P = .047) but not other domains.

“This analysis provides new insights into the clinical results of lenalidomide in non-del-5q patients,” said Santini. “We now have a better understanding of how achievement of transfusion independence impacts quality of life measures.”

RBC-TI of at least 8 weeks was associated with significant improvement (P <.01) across all preselected domains, with improvements also exceeding the prespecified threshold for clinically meaningful change.

In the study, the most common adverse events associated with lenalidomide were related to myelosuppression, including neutropenia (64.4% vs 11.4%) and thrombocytopenia (39.4% vs 7.6%). Grade 3/4 neutropenia occurred in 61.9% and 12.7% in the lenalidomide and placebo groups, respectively, and grade 3/4 thrombocytopenia occurred in 35.6% versus 3.8%.

The FDA granted lenalidomide Subpart H approval in 2005 for patients with transfusion-dependent anemia due to low or intermediate-1 risk MDS associated with a deletion 5q cytogenetic abnormality. The drug is currently only available under a special restricted distribution program. The Risk Evaluation and Mitigation Strategy (REMS) program is designed to avoid embryo-fetal exposure to lenalidomide, as the drug is linked to birth defects or death of unborn babies. Risk impacts both women and men, as lenalidomide can pass into human semen.

In 2006 lenalidomide was approved for use in combination with dexamethasone in patients with multiple myeloma who have received one prior therapy. In February 2015, the FDA expanded this multiple myeloma indication to include use in combination with dexamethasone in newly diagnosed patients. The drug also received approval for the treatment of mantle cell lymphoma in 2013 for use in patients whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib (Velcade).

Santini V, Almeida A, Giagounidis A, et al. The effect of lenalidomide on health-related quality of life (HRQoL) in patients with MDS: results from the MDS-005 trial. Leukemia Research. 2015;39:1s (suppl; abstr 116).

View original publication of article


Celgene Corporation Enters into Strategic Immuno-Oncology Collaboration with AstraZeneca to Develop PD-L1 Inhibitor Program for Patients with Serious Blood Cancers

Program to Focus Initially on Non-Hodgkin’s Lymphoma, Myelodysplastic Syndromes and Multiple Myeloma

Studies Planned to Start in Second Half 2015

BOUDRY, Switzerland–(BUSINESS WIRE)– Celgene International II Sàrl, a wholly owned subsidiary of Celgene Corporation (NASDAQ:CELG) today announced that it has entered into a strategic collaboration with MedImmune Limited, a wholly owned subsidiary of AstraZeneca PLC, to develop and commercialize an anti-PD-L1 inhibitor, MEDI4736, for hematologic malignancies. Approximately 1.75 million patients globally suffer from blood cancer and many are in need of new treatment options.

MEDI4736 is a human monoclonal antibody directed against programmed cell death ligand 1 (PD-L1), which helps tumors avoid detection by the immune system. Tumor cells use PD-L1 to turn off the immune system just as it begins to mount a response against them. MEDI4736 helps turn the immune system back on, allowing it to continue its attack on cancer.

“The potential of rationally combining immunotherapies such as MEDI4736 with existing and novel hematology compounds creates new opportunities for patients with blood cancers to live longer, better lives,” said Jacqualyn A. Fouse, Ph.D., President, Global Hematology and Oncology for Celgene. “This strategic collaboration leverages the deep expertise of AstraZeneca/MedImmune in immuno-oncology along with the experience of Celgene in the study and treatment of blood cancers. This collaboration advances Celgene’s already deep, diverse scientific platform to include checkpoint inhibitors, an area of significant promise in hematology.”

Dr. Bahija Jallal, Executive Vice President at MedImmune, said: “We are excited about our strategic collaboration with Celgene, a globally recognized leader in treatments for hematological cancers. This agreement is a great example of how we are accelerating the development of medical innovation in our portfolio in collaboration with other experts, in order to bring life-enhancing new medicines to patients faster. Together with Celgene, we are designing a programme for our anti-PD-L1 that will explore its full clinical potential as a game-changing treatment that could activate the patients’ immune system to fight and change the course of blood cancers in this area of high unmet need.”

Under the terms of the agreement, Celgene will collaborate with AstraZeneca to develop the anti-PD-L1 antibody MEDI4736 in hematology and make an upfront payment of $450 million. Celgene will lead clinical development across all new clinical trials within the collaboration and be responsible for all costs associated with these trials until December 31, 2016, after which it is responsible for 75% of these costs. Celgene will also be responsible for the global commercialization of approved MEDI4736 indications in hematology, and will receive royalty rates starting at 70 percent of worldwide sales from all uses in hematology. Royalty rates will decrease gradually to 50 percent over a period of four years after the first date of commercial sales. This collaboration agreement will become effective upon the expiration or termination of the applicable waiting periods under all applicable antitrust laws.

This strategic collaboration will initially focus on the development of MEDI4736 as combination therapy with Celgene’s pipeline of products and other novel agents for hematologic disorders. Over time, the collaboration could expand to include other assets.

MEDI4736 is not approved in any country for any indication.

About MEDI4736

MEDI4736 is a human monoclonal antibody directed against programmed cell death ligand 1 (PD-L1), which helps tumors avoid detection by the immune system. MEDI4736 is currently being evaluated in several disease states, including lung, melanoma and head and neck cancer.

About Hematologic Malignancies

The World Health Organization estimates that 1.75 million patients had a hematologic malignancy in 2012. These diseases include lymphoma, leukemia, multiple myeloma and myelodysplastic syndromes. The global incidence of blood cancers continues to rise. By 2030, the incidence of blood cancer overall is predicted to rise by 46 percent, and that of non-Hodgkin’s lymphoma and multiple myeloma by 50 and 60 percent, respectively.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit www.celgene.com. Follow us on Twitter @Celgene, and on Pinterest and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similar expressions. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in Celgene Corporation’s Annual Report on Form 10-K and other reports filed with the Securities and Exchange Commission.

All registered trademarks are owned by Celgene Corporation.


VI. International Eurasian Congress of Hematology

VI. International Eurasian Congress of HematologyCONGRESSINVITATION
from the Hematology Specialists Association, Turkey:

 

Dear Colleagues,

We are pleased to announce that VI. International Eurasian Congress of Hematology will be held in the Mardan Palace Hotel in Antalya between 14-18 October 2015.

In 2014, our congress has made progress on becoming one of the world’s leading international conventions with the participation of our colleagues from 34 different countries.

We believe deep in our hearts that with its special concept, the Eurasian Congress of Hematology will fill in a significant gap in our region and serve not only scientifically but also culturally.

Looking forward to meeting with your respected selves in the Mardan Palace Hotel in Antalya between 14-18 October, we would like to extend our kindest regards.

Prof. Dr. Süleyman Dinçer, President of Hematology Specialists Association, Turkey
Prof. Dr. Giuseppe Saglio, Congress President


ASTX727 Clinical Trial for Low to High-Risk MDS/CMML Patients

Astex Pharmaceuticals is conducting a Phase 1-2 clinical trial to determine if ASTX727 is effective for the treatment of patients with IPSS low, intermediate -1, intermediate -2, or high risk MDS (including CMML).

Read more

ASTX727 Study Fast Facts
Additional ASTX727 Study Information


Announcing a Clinical Study for Patients with Myelodysplastic Syndromes (MDS)

NOW ENROLLING: NCT01566695

QUAZAR Lower-Risk MDS is a study for people with MDS who need blood transfusions due to low red blood cell counts (called anemia) and low platelet counts (called thrombocytopenia).

You may qualify for this study if you*

  • Are age 18 years or older
  • Have been diagnosed with MDS
  • Have low red blood cell counts and are dependent on blood transfusions
  • Have low blood platelet counts

You may not be eligible for this study if you*

  • Have had previous stem cell transplants
  • Have been treated with VIDAZA® (azacitidine for injection) or DACOGEN® (decitabine for injection)

*Additional criteria apply.

For more information about this study, call 646-307-8079 or toll-free at 866-743-9791, e-mail QUAZARMDSstudy@emergingmed.com or visit
https://www.celgeneclinicaltrials.com/quazar-mds
https://www.celgeneclinicaltrials.com/quazar-aml


NICE Proposes to Recommend Lenalidomide for MDS

NICE has issued final draft guidance recommending lenalidomide (also known as Revlimid and marketed by Celgene) as an option, for treating myelodysplastic syndromes.

Myelodysplastic syndromes (MDS), which are diagnosed in around 2000 people each year in England, are a group of bone marrow disorders characterised by the underproduction of one or more types of blood cells due to dysfunction of the marrow. MDS can lead to life threatening disease including acute myeloid leukaemia (AML), as well as anaemia and increased risk of bleeding and infections.

This appraisal focused on the use of lenalidomide for treating people with a specific type of MDS that is characterised by a chromosomal abnormality called an isolated deletion 5q cytogenetic abnormality. At the moment the main treatment option for people with the particular kind of MDS considered in this appraisal is best supportive care including regular blood transfusions.

Commenting on the final draft guidance, Sir Andrew Dillon, NICE chief executive, said: “The committee heard from clinical experts that lenalidomide is an effective therapy. Celgene – who market lenalidomide – worked with us to provide enough evidence to make it possible for us to recommend it for this group of people.

“Celgene provided a revised analyses and further information on their proposal for a reduction in the cost of the drug to the NHS (patient access scheme).”

Celgene’s patient access scheme involves the NHS paying for lenalidomide treatment for up to 26 monthly cycles. The company will provide the drug free of charge for those people who receive more than 26 monthly cycles.

The draft guidance is now with consultees, who have the opportunity to appeal against it. Until NICE issues final guidance, NHS bodies should make decisions locally on the funding of specific treatments.

Important new survey started by EHA – the European Haematology Association. EHA is interested in finding out if certain medicines are not available in some parts of the country – or in some parts of Europe. Patients, Caregivers, and Healthcare professionals are encouraged to complete this survey. The information from this survey will educate European policy makers to take measures that will help provide effective medicines that are not affordable or are not marketed in specific countries.


New Ways to Manage MDS: A New Resource for Patients and Loved Ones

Having trouble explaining Myelodysplastic Syndromes in terms your loved ones can understand? This new episode of the health and wellness program HEALTHY BODY, HEALTHY MIND can help explain it for you. If you would like to receive your FREE DVD, please call 609-298-1035 or 800-637-0839 or email dmurray@devmdsfound.org

Learn more


GREAT NEWS: Italian Study Supports Use of Revised International Prognostic Scoring!

Under the aegis of the MDS Foundation, the International Working Group for Prognosis in MDS generated an improved method analyzing MDS patient prognosis more precisely than the initial IPSS.

Read more


Revlimid® Approved in Europe for Use in MDS

The European Medicines Agency (EMA) has approved Revlimid® for the treatment of MDS patients with transfusion-dependent anemia associated with the del(5q) chromosomal abnormality.

Read more


Revised International Prognostic Scoring System for MDS

IWG-PM Logo Under the aegis of the MDS Foundation, the International Working Group for Prognosis in MDS (IWG-PM) analyzed clinical features and outcome data from over 7000 patients and generated a method analyzing MDS patient prognosis more precisely than the initial IPSS. Novel components of this prognostic system include: five rather than three cytogenetic prognostic subgroups with specific and new classifications of a number of less common cytogenetic subsets, splitting the low marrow blast percentage value, and depth of cytopenias. In addition to the major prognostic variables of marrow blasts, cytogenetics and peripheral cytopenias, additive features for survival include patient age, performance status, serum ferritin and LDH. The IPSS-R calculator tool is accessible through the following URLs : https://www.mds-foundation.org/ipss-r-calculator/index.php or http://www.ipss-r.com. An iPhone App for the IPSS-R calculator tool is also accessible through the Apple Store (enter MDS IPSS-R). This IPSS-R should prove beneficial for predicting the clinical outcomes of untreated MDS patients and aiding design and analysis of clinical trials in this disease. Data for both calculator tools are derived from the article: Greenberg P, Tuechler H, Schanz J, et al, Revised International Prognostic Scoring System (IPSS-R) for Myelodysplastic Syndromes. Blood 120: 2454-2465, 2012.


MDS Foundation a Valued Partner of GuideStar


We are proud to be a valued partner of GuideStar, America’s first information service publicizing information about nonprofit organizations. Access the GuideStar link for additional financial information or to learn more about ways to give an annual or planned gift to fund MDS programs and services.

http://www.guidestar.org/organizations/22-3283911/myelodysplastic-syndromes-foundation.aspx


Launch of Fast Track for First Prescription™ Service through Celgene Patient Support® Fast Track for First Prescription Program™

Fast Track for First PrescriptionTM is an optional Celgene Patient Support® service which helps patients on an oral Celgene product receive their first prescription faster.

Additional details about eligibility and enrollment are addressed below.

For more information, or for any questions, call Celgene Patient Support® at 1-800-931-8691.

Which patients are eligible for this program?
  • Patients on an oral Celgene product receiving their first prescription
  • Patients with documented proof of insurance
  • Patients who are registered in a Celgene risk management (REMS) program and have a valid authorization number
How are eligible patients enrolled in this program?
  • Prescriber must fax the Fast Track Cover Sheet and the Patient Prescription Form with the authorization number and the patient risk category (i.e., adult female of childbearing potential, adult male, etc) to 1-800-822-2496
  • Patient’s contact information and insurance information should be included
  • Prescriber must ensure patient has completed their Celgene risk management (REMS) survey
What can a patient expect after being enrolled in this program?

The specialty pharmacy will contact the patient to:

  • Review their insurance benefits for the prescribed product (i.e., co-pay responsibilities and potential financial co-pay assistance options)
  • Provide appropriate counseling (if applicable)
  • Set up a delivery date for shipment of the medication

Am J Med: MDS Supplement

View the MDS Supplement in the American Journal of Medicine. This supplement is supported by Celgene Corporation. These articles are available on the internet through open access, allowing interested parties to download the entire supplement for free.


Review answers to commonly asked questions or get answers to your questions from an MDS expert