On December 12, 2017 GlycoMimetics, Inc. (NASDAQ: GLYC) reported updated data from the Phase 1/2 trial evaluating the safety, tolerability and efficacy of GMI-1271 in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) and in older adults with newly diagnosed AML, including the following conclusions (Press release, GlycoMimetics, DEC 12, 2017, View Source [SID1234522581]):

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For patients with R/R AML treated at the Phase 2 dose (n = 54) and for whom median follow up was 6.6 months:

Clinical remission (CR+CRi) was 43%.
Median overall survival was 9.4 months (95% CI: 5.7 – 15.1 months; calculated by Kaplan Meier method). This compares favorably to a median overall survival of up to 5.4 months reported for historical, matched controls treated with mitoxantrone, etoposide and cytarabine (MEC) alone. 1,2
Median duration of remission was 11.1 months (95% CI: 5.8-NA; calculated by Kaplan Meier method).
For older patients with newly diagnosed disease (n=25) and for whom median follow up was 10.5 months:

Clinical remission rate was 68%.
Median overall survival was 15.8 months (95% CI: 10.3 – NA; calculated by Kaplan Meier method). This compares favorably to a historical median overall survival of approximately 12 months in matched controls treated with 7+3 chemotherapy alone. 3,4
Median duration of remission was 14.8 months (95% CI: 8.3 – NA; calculated by Kaplan Meier method).
Median event free survival was 11.3 months.
The data were presented yesterday during an oral scientific session at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Expo in Atlanta.

Across both populations, GMI-1271 was well tolerated with no obvious incremental toxicity observed and lower than expected rates of severe, debilitating, grade 3-4 mucositis reported (e.g., 3% incidence reported vs. historical 20-25% incidence with MEC alone).

"These new data from our Phase 1/2 clinical trial demonstrate that encouraging clinical outcomes are possible for both duration of remission and survival endpoints when GMI-1271 is added to chemotherapy in two distinct AML patient populations," noted Helen Thackray, M.D., FAAP, GlycoMimetics Senior Vice-President, Clinical Development and Chief Medical Officer. "Beyond the high response rates previously reported with GMI-1271, we can now point to additional long-term endpoints that further support our plan to move the drug candidate into a Phase 3 clinical trial scheduled to begin in mid-2018. Importantly, with respect to safety, the low mucositis rate in relapsed and refractory patients receiving MEC induction chemotherapy — where you would expect around 25% severe mucositis — is quite striking. This was predicted and explained by preclinical models in which GMI-1271 blocked inflammatory macrophages trafficking to the gut and thus prevented mucosal injury."

"These results continue to show that AML patients treated with GMI-1271 consistently perform better than expected," said Daniel J. DeAngelo, M.D., Ph.D., the trial’s lead investigator and Director of Clinical and Translational Research, Adult Leukemia Program, at the Dana-Farber Cancer Institute and Brigham and Women’s Hospital, who presented the data at the ASH (Free ASH Whitepaper) Annual Meeting. "Our Phase 2 population consists of very high-risk patients based on age, disease status, and cytogenetic risk factors. The updated data continue to support the concept that disrupting the relationship between leukemic cells and the protective bone marrow microenvironment, when combined with chemotherapy, could improve the outlook and prognosis for these patients."

The second oral presentation at the ASH (Free ASH Whitepaper) meeting highlighted a preclinical study in murine models of AML in which E-selectin was shown to be upregulated, and AML cells binding to E-selectin increased chemo-resistance by activating specific tumor cell survival signaling pathways. This effect within the bone marrow microenvironment is unique to E-selectin as compared to other vascular adhesion molecules and can be blocked by GMI-1271. This translational research provides important evidence that elucidates how treatment with GMI-1271 appears to be improving sensitivity to chemotherapy.

"Given response rates we’ve observed to date that suggest clinical benefit in combination with chemotherapy in two AML populations, this preclinical work provides important further support for the mechanism of action of GMI-1271," noted Dr. Thackray. "Together, the clinical and preclinical data we have shared at the ASH (Free ASH Whitepaper) Annual Meeting demonstrate that GMI-1271 could represent a novel and truly differentiated approach to treatment of AML," Dr. Thackray concluded.

Meeting abstracts are available on ASH (Free ASH Whitepaper)’s website.

GlycoMimetics to Hold Post-ASH Meeting Briefing in Boston on December 19

GlycoMimetics will hold a briefing for investors/analysts, which will also be available via webcast, to review the GMI-1271 program with a focus on the AML clinical data presented at the ASH (Free ASH Whitepaper) Annual Meeting, at the Langham Hotel in Boston, December 19, at 7:30 a.m. EST. Dr. DeAngelo will present the clinical data from the ASH (Free ASH Whitepaper) oral presentation and respond to questions from on-site participants.

On December 12, 2017 Geron Corporation (Nasdaq:GERN) reported four presentations related to imetelstat at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition held in Atlanta, Georgia from December 9-12, 2017 (Press release, Geron, DEC 12, 2017, View Source;p=RssLanding&cat=news&id=2322273 [SID1234522575]). Imetelstat is a telomerase inhibitor initially developed by Geron and exclusively licensed to Janssen Biotech, Inc. (Janssen) on a worldwide basis. The presentations are available on Geron’s website at www.geron.com/presentations.

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"We and our partner are pleased to have this first presentation of data from IMerge displaying an 8-week transfusion independence rate of 54% among the subset of patients who were naïve to lenalidomide and HMAs and who lacked del(5q), which suggests that imetelstat could offer lower risk MDS patients a much-needed treatment option," said John A. Scarlett, M.D., Geron’s President and Chief Executive Officer. "In addition, the non-clinical data presentations continue to support the potential use of imetelstat in multiple hematologic malignancies. We continue to be encouraged by the consistent interest in imetelstat by collaborators around the world."

Clinical Data Presentation

Title: Efficacy and Safety of Imetelstat in RBC Transfusion-Dependent (TD) IPSS Low/Int-1 MDS Relapsed/Refractory to Erythropoiesis-Stimulating Agents (ESA) (IMerge) (Abstract #4256)

This poster presentation described data from the first 32 patients enrolled in Part 1 of IMerge, the ongoing Phase 2/3 clinical trial of imetelstat in red blood cell (RBC) transfusion-dependent (TD) patients with lower risk MDS. TD is defined as an RBC transfusion requirement of ≥4 units over 8 weeks prior to entry into the trial. The primary efficacy endpoint is the rate of RBC transfusion-independence (TI) lasting at least 8 weeks, defined as the proportion of patients without any RBC transfusion during any consecutive 8 weeks since entry to the trial. Key secondary endpoints are the rate of 24-week TI and the rate of hematologic improvement-erythroid (HI-E), defined as a rise in hemoglobin of at least 1.5 g/dL above the pretreatment level for at least 8 weeks or a reduction of at least 4 units of RBC transfusions over 8 weeks compared with the prior RBC transfusion burden. IMerge is designed in two parts: Part 1 is a Phase 2, open-label, single-arm trial of imetelstat administered as a single agent by intravenous infusion, and Part 2 is designed to be a Phase 3, randomized, controlled trial.

Data as of October 2017 with a median follow-up of 66.1 weeks in Part 1 were presented. Part 2 has not yet begun.

Efficacy in the Overall Trial Population (n=32):

38% (12/32) of patients achieved ≥8-week RBC TI
Mean relative reduction in transfusion burden from baseline was 64%
16% (5/32) of patients achieved ≥24-week RBC TI, with the median TI duration exceeding one year in these patients
63% (20/32) of patients achieved an erythroid hematologic improvement (HI-E)
Efficacy in the Lenalidomide and HMA Naïve and Non-Del(5q) Subset (n=13):

54% (7/13) of patients achieved ≥8-week RBC TI
Mean relative reduction in transfusion burden from baseline was 71%
31% (4/13) of patients achieved ≥24-week RBC TI
69% (9/13) of patients achieved an HI-E
Safety Summary:

Cytopenias, particularly neutropenia and thrombocytopenia, were the most frequently reported adverse events which were predictable, manageable and reversible
Link to IMerge poster: Fenaux P, et al. ASH (Free ASH Whitepaper) 2017

Based on these data, Part 1 of IMerge is being expanded to enroll approximately 20 additional patients who are naïve to lenalidomide and HMA treatment and are non-del(5q) to increase the experience and confirm the benefit-risk profile of imetelstat in this refined target patient population. For more information about IMerge, please visit View Source

Non-Clinical Data Presentations

Data were presented from three non-clinical studies by academic scientists in collaboration with Janssen.

Title: Imetelstat, a Telomerase Inhibitor, Is Capable of Depleting Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells (Abstract #1654)

This poster presentation described the effects of imetelstat on normal and myelofibrosis (MF) stem and progenitor cells in non-clinical models. The data showed that imetelstat inhibited the proliferation and differentiation of MF progenitor and stem cells in hematopoietic colony assays and xenograft models, but had minimal effects on normal hematopoietic stem and progenitor cells in such experiments. Imetelstat treatment of MF stem and progenitor cells reduced telomerase activity and induced apoptosis. These data provide further support that imetelstat may selectively inhibit the proliferation of and promote apoptosis in MF progenitor and stem cells, suppressing the malignant clones that drive the disease in patients.

Title: Telomerase Inhibition Impairs Self-Renewal of b-Catenin Activated Myeloproliferative Neoplasm Progenitors (Abstract #2860)

This poster presentation described the potential impact of imetelstat on leukemia stem cells in non-clinical models of chronic myeloid leukemia (CML) in blast crisis. The data suggest that imetelstat plus dasatinib, a standard treatment for CML, may inhibit self-renewal of blast crisis cells in vitro compared with normal bone marrow progenitors. In mouse xenograft models of blast crisis CML, imetelstat treatment reduced the number of leukemia progenitor cells detected in bone marrow and spleen, decreased spleen size, inhibited the self-renewal capacity and prolonged survival compared to controls. These data suggest that imetelstat may inhibit proliferation of malignant progenitors in CML.

Title: Integrated Molecular Analysis Identifies Replicative Stress as Sensitizer to Imetelstat Therapy in AML (Abstract #798)

This oral presentation described imetelstat’s activity in human acute myeloid leukemia (AML) patient-derived xenograft models. The preclinical data demonstrated that imetelstat prolonged overall survival of AML xenografts derived from 17 out of 30 individual patient samples compared to saline-treated controls. Molecular analysis showed that sustained responders were associated with gene signatures of replicative stress at baseline. The data also suggest that inducing replicative stress with standard induction chemotherapy may sensitize poorly responding samples to imetelstat. These data build on previously published preclinical work and further support potential application of imetelstat in the treatment of AML.

Webcast Investor Event

Management will be hosting a live webcast of an analyst and investor meeting on December 19, 2017 at 8:30 a.m. ET to discuss the imetelstat clinical data in MDS presented at ASH (Free ASH Whitepaper). The audio and slide presentation webcast will be accessible at www.geron.com on the Investor Relations pages, under Events. Following the live presentation, the webcast will be archived and available for replay at the same address for a period of 30 days.

About Imetelstat

Imetelstat (GRN163L; JNJ-63935937) is a potent and specific inhibitor of telomerase that is administered by intravenous infusion. This first-in-class compound, discovered by Geron, is a specially designed and modified short oligonucleotide, which targets and binds directly with high affinity to the active site of telomerase. Preliminary clinical data suggest imetelstat might have disease-modifying activity by inhibiting the progenitor cells of the malignant clones associated with hematologic malignancies in a relatively select manner. Most commonly reported adverse events in imetelstat clinical studies include fatigue, gastrointestinal symptoms and cytopenias. Imetelstat has not been approved for marketing by any regulatory authority.

About the Collaboration with Janssen

On November 13, 2014, Geron entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc., to develop and commercialize imetelstat for oncology, including hematologic myeloid malignancies, and all other human therapeutics uses. Under the terms of the agreement, Geron received an upfront payment of $35 million and is eligible to receive additional payments up to a potential total of $900 million for the achievement of development, regulatory and commercial milestones, as well as royalties on worldwide net sales. All regulatory, development, manufacturing and promotional activities related to imetelstat are being managed through a joint governance structure, with Janssen responsible for these activities.

On December 12, 2017 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported the first results from the pivotal phase III MURANO study evaluating Venclexta/Venclyxto (venetoclax) plus MabThera/Rituxan (rituximab) compared to bendamustine plus MabThera/Rituxan (BR) for the treatment of people with relapsed or refractory chronic lymphocytic leukaemia (CLL) (Press release, Hoffmann-La Roche, DEC 12, 2017, View Source [SID1234522580]). The results showed that a fixed duration of treatment with Venclexta/Venclyxto plus MabThera/Rituxan significantly reduced the risk of disease progression or death (progression-free survival; PFS, as assessed by investigator) by 83% compared with BR (HR=0.17; 95% CI 0.11-0.25; p<0.0001). No new safety signals were observed. Venclexta/Venclyxto is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the United States and commercialised by AbbVie outside of the United States.

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"The MURANO study results indicate that Venclexta/Venclyxto plus MabThera/Rituxan has the potential to provide an important new chemotherapy-free option for people with previously treated chronic lymphocytic leukaemia," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "We are particularly encouraged by the magnitude of benefit observed across key efficacy measures compared to a current standard of care, and we look forward to discussing these results with health authorities."

Results from the study were featured in the official press programme of the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta on Monday, 11 December, and will be presented during the Late-Breaking Abstracts Session on Tuesday, 12 December, at 7.45am EST by John F. Seymour, MD, Peter MacCallum Cancer Centre and Royal Melbourne Hospital, Melbourne, Australia (Abstract #LBA-2).

Data from the MURANO study will be submitted to global health authorities, including the US Food and Drug Administration (FDA), which has granted Breakthrough Therapy Designation for Venclexta in combination with Rituxan for the treatment of relapsed or refractory CLL based on promising results from the phase Ib M13-365 study.

Venclexta was granted accelerated approval by the FDA in April 2016 for the treatment of people with CLL with 17p deletion, as detected by an FDA approved test, who have received at least one prior therapy. The MURANO study is part of the company’s commitment in the United States to convert the current accelerated approval of Venclexta to a full approval.

A robust clinical development programme for Venclexta/Venclyxto is ongoing, and additional results across multiple blood cancers including acute myeloid leukaemia (AML) and multiple myeloma (MM) were also presented at the ASH (Free ASH Whitepaper) Annual Meeting.

About the MURANO Study
MURANO (NCT02005471) is a phase III open-label, international, multicentre, randomised study evaluating the efficacy and safety of Venclexta/Venclyxto in combination with MabThera/Rituxan compared to bendamustine in combination with MabThera/Rituxan (BR). All treatments were of fixed duration. The study included 389 patients with relapsed or refractory chronic lymphocytic leukaemia (CLL) who had been previously treated with at least one, but not more than three, lines of therapy. Patients were randomly assigned in a 1:1 ratio to receive either Venclexta/Venclyxto plus MabThera/Rituxan (Arm A) or BR (Arm B). The primary endpoint of the study is investigator-assessed progression-free survival (PFS). Secondary endpoints include PFS assessed by independent review committee (IRC), overall response rate (ORR), complete response rate (with or without complete blood count recovery, CR/CRi), overall survival (OS), minimal residual disease (MRD) status, duration of response, event-free survival and time to next CLL treatment.

The results showed:

Patients in the study who received Venclexta/Venclyxto plus MabThera/Rituxan lived significantly longer without their disease worsening (PFS, as assessed by investigator) compared to those who received BR (HR=0.17; 95% CI, 0.11-0.25; p<0.0001; median PFS: not reached vs. 17.0 months, respectively).
At two years, 84.9% of patients in the Venclexta/Venclyxto plus MabThera/Rituxan arm had not experienced disease progression, compared to 36.3% of patients in the BR arm.
Consistent benefit was observed in all patient subgroups for Venclexta/Venclyxto plus MabThera/Rituxan compared to BR, including high-risk and low-risk groups.
IRC assessment was consistent; as assessed by IRC, Venclexta/Venclyxto plus MabThera/Rituxan reduced the risk of disease progression or death by 81% compared to BR (HR=0.19; 95% CI 0.13, 0.28; p<0.0001).
Clinical benefit observed for Venclexta/Venclyxto plus MabThera/Rituxan compared to BR was consistent across key secondary endpoints, including OS (HR=0.48, 95% CI 0.25-0.90; medians not yet reached), ORR as assessed by investigator (93.3% vs. 67.7%), CR/CRi as assessed by investigator (26.8% vs. 8.2%). These results were not statistically significant. In addition, higher rates of MRD-negativity at any time were observed with Venclexta/Venclyxto plus MabThera/Rituxan compared to BR (83.5% vs. 23.1%). MRD negativity was defined as less than one CLL cell in 10,000 leukocytes.
No new safety signals were observed with the treatment combination of Venclexta/Venclyxto plus MabThera/Rituxan. The most common Grade 3-4 adverse events with Venclexta/Venclyxto plus MabThera/Rituxan compared to BR, respectively, were low white blood cell count (57.7% vs. 38.8%), low red blood cell count (10.8% vs. 13.8%), low platelet count (5.7% vs. 10.1%), low white blood cell count with fever (3.6% vs. 9.6%), pneumonia (5.2% vs. 8.0%) and infusion reactions (1.5% vs. 5.3%).
About Venclexta/Venclyxto
Venclexta/Venclyxto is a small molecule designed to selectively bind and inhibit the BCL-2 protein, which plays an important role in a process called apoptosis (programmed cell death). Overexpression of the BCL-2 protein in CLL has been associated with resistance to certain therapies. It is believed that blocking BCL-2 may restore the signalling system that tells cells, including cancer cells, to self-destruct. Venclexta/Venclyxto is being developed by Roche and AbbVie. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the United States and commercialised by AbbVie outside of the United States.

Together, the companies are committed to further research with Venclexta/Venclyxto, which is currently being evaluated in phase III clinical trials for the treatment of CLL, along with studies in several other types of cancers. In the United States, Venclexta has been granted four breakthrough therapy designations by the FDA: in combination with Rituxan for people with relapsed or refractory CLL, as a monotherapy for people with relapsed or refractory CLL with 17p deletion; in combination with hypomethylating agents (azacitidine or decitabine) for people with untreated acute myeloid leukaemia (AML) ineligible for intensive chemotherapy, and in combination with low-dose cytarabine (LDAC) for people with untreated AML ineligible for intensive chemotherapy.

About Chronic Lymphocytic Leukaemia
Chronic lymphocytic leukaemia (CLL) is the most common type of leukaemia in the Western world.1 CLL mainly affects men and the median age at diagnosis is about 70 years.2 Worldwide, the incidence of all leukaemias is estimated to be over 350,0001 and CLL is estimated to affect around one-third of all people newly diagnosed with leukaemia.3

About Roche in haematology
For more than 20 years, Roche has been developing medicines that redefine treatment in haematology. Today, we are investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. In addition to approved medicines MabThera /Rituxan (rituximab), Gazyva/Gazyvaro (obinutuzumab), and Venclexta / Venclyxto (venetoclax) in collaboration with AbbVie, Roche’s pipeline of investigational haematology medicines includes Tecentriq (atezolizumab), an anti-CD79b antibody drug conjugate (polatuzumab vedotin/RG7596) and a small molecule antagonist of MDM2 (idasanutlin/RG7388). Roche’s dedication to developing novel molecules in haematology expands beyond malignancy, with the development of Hemlibra (emicizumab), a bispecific monoclonal antibody for the treatment of haemophilia A.

On December 12, 2017 Cyclacel Pharmaceuticals, Inc. (NASDAQ:CYCC) (NASDAQ:CYCCP) "Cyclacel" or the "Company"), a biopharmaceutical company developing oral therapies that target various phases of cell cycle control for the treatment of cancer and other serious disorders, reported results from the Company’s Phase 3 SEAMLESS study (Press release, Cyclacel, DEC 12, 2017, View Source [SID1234522578]). Cyclacel had previously announced top-line results from its Phase 3 SEAMLESS study in February 2017. The study enrolled elderly patients with newly diagnosed acute myeloid leukemia (AML) and compared alternating cycles of decitabine and sapacitabine versus decitabine. Data were reported at an oral presentation on Monday, December 11, at 6:45 PM EST at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta, Georgia.

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"Although the study did not reach its primary endpoint of superiority in survival, we are encouraged by the higher complete remission rate on the sapacitabine-decitabine arm, especially in the subgroup with low white blood cell count; additional analysis of the data should be pursued," said Hagop Kantarjian, M.D., Professor and Chair, Department of Leukemia, The University of Texas MD Anderson Cancer Center, and chair of the study.

"We are pleased to report detailed results of the SEAMLESS study, which as previously announced, did not reach its primary endpoint," said Spiro Rombotis, President and Chief Executive Officer of Cyclacel. "We believe that the subgroup results have defined a patient population for whom the decitabine-sapacitabine regimen may represent an improvement over low intensity treatment by decitabine alone. We plan to discuss the data, the statistical robustness of the subgroup results and the optimal baseline peripheral white blood cell (WBC) cutpoint with European and US regulatory authorities and will provide updates as appropriate. We are grateful to the patients, their families and the investigators for their contributions to this large study. In parallel, we are progressing our other clinical programs in transcriptional regulation with CYC065 and DNA damage response with sapacitabine-seliciclib in biomarker-selected patients with solid tumors, such as those with BRCA mutations or resistance to existing cancer therapies."

Study Design & Intent-to-Treat Results

The randomized, open label, Phase 3 SEAMLESS study enrolled 482 patients, aged 70 years or older, with newly diagnosed AML who were not candidates for or refused intensive therapy at 110 US and EU sites. Patients were stratified by WBC, antecedent hematologic disorder (AHD), and marrow blasts, and randomized 1:1 to receive either intravenous decitabine administered in alternating cycles with oral sapacitabine versus intravenous decitabine alone.

The trial did not meet its primary endpoint of demonstrating statistically significant improvement in overall survival. A higher complete remission (CR) rate, a secondary endpoint, was observed on the decitabine-sapacitabine arm (17% versus 11%). Other endpoints and safety were similar between the arms.

Prespecified Subgroup Analysis

Baseline WBC

In the less than 10,000 WBC subgroup (n=319) a trend towards improved overall survival (median 8.0 versus 5.8 months, HR=0.84 [0.66, 1.06], p=0.14) favoring decitabine-sapacitabine and a significantly higher CR rate (21.0% versus 8.6%, p=0.0017) was achieved on decitabine-sapacitabine.

In the 10,000 or more WBC subgroup (n=163) significantly better overall survival (median 3.8 versus 5.5 months, HR=1.57 [1.12, 2.19], p=0.007) was observed on decitabine. A trend in CR rate (8.3% versus 15.2%, p=0.18) favoring decitabine was observed but it did not reach statistical significance.

Prior AHD

In the subgroup with prior AHD (n=136) a significantly higher CR rate (16.7% versus 5.7%, p=0.0398) was achieved on decitabine-sapacitabine. There was a numerical difference in median survival (6.4 versus 5.0 months, HR=0.85 [0.59, 1.24], p=0.41) favoring decitabine-sapacitabine but overall survival did not reach statistical significance.

In the subgroup without prior AHD (n=346) there was a numerical difference in median survival (5.9 versus 6.7 months, HR=1.08 [0.86, 1.35], p=0.52) favoring decitabine and CR rate (16.6% versus 12.9%) favoring decitabine-sapacitabine but neither reached statistical significance.

Cytogenetics

In the subgroup with other than unfavorable cytogenetics (n=288) there was a numerical difference in median survival (8.2 versus 5.7 months, HR=0.89 [0.69, 1.15], p=0.38) and CR rate (19.9% versus 11.6%, p=0.16) favoring decitabine-sapacitabine but neither reached statistical significance.

In the subgroup with unfavorable cytogenetics (n=194) there was a numerical difference in median survival (3.8 months versus 5.7 months, HR=1.27 [0.94, 1.73], p=0.12) favoring decitabine but overall survival did not reach statistical significance. There was a numerical difference in CR rate favoring decitabine-sapacitabine (12.0% versus 9.6%) but it did not reach statistical significance.

In the subgroup of patients with below 50% and with 50% or higher bone marrow blasts there were no statistically significant differences in overall survival between the arms.

Presentation

The presentation (abstract 891), titled "Results of a Phase 3 Study of Elderly Patients with Newly Diagnosed AML Treated with Sapacitabine and Decitabine Administered in Alternating Cycles," is available on the Cyclacel website at www.cyclacel.com.

About Sapacitabine

Sapacitabine (CYC682), an orally-available nucleoside analogue, is currently being studied in an ongoing, extension of a Phase 1 study evaluating a combination regimen of sapacitabine and seliciclib, a first generation CDK inhibitor. Parts 1 and 2 of the study evaluated approximately 90 patients with advanced cancers. Part 3 is ongoing in patients with BRCA positive, breast, ovarian and pancreatic cancer. Over 1,000 patients with hematological malignancies and solid tumors have received sapacitabine.

About AML

AML is a rapidly progressing cancer of the blood characterized by the uncontrolled proliferation of immature blast cells in the bone marrow. The American Cancer Society estimates there will be approximately 21,380 new cases of AML and approximately 10,590 deaths from AML in the U.S. in 2017. AML is generally a disease of older adults and the median age is about 67 years. Newly diagnosed elderly patients with poor prognostic risk factors typically die within one year.

On December 12, 2017 Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, reported an update on its clinical development programs for CRS-207, a first-generation proprietary attenuated strain of Listeria that has been engineered to express the tumor-associated antigen mesothelin (Press release, Aduro Biotech, DEC 12, 2017, View Source;p=RssLanding&cat=news&id=2322291 [SID1234522577]). Based on preliminary results from its mesothelioma and ovarian studies, as well as a business and commercial assessment, the company has determined that it will not continue advancement of CRS-207 and will wind down each of its trials in mesothelioma, ovarian and gastric cancer. Aduro will be working closely with investigators to proceed in a manner that is aligned with the best interests of patients still being treated on these studies.

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"We would like to thank the patients, their families, our clinical investigators and staff for their time and commitment to these trials, which will contribute important data to the field of oncology. While we are disappointed with the results for CRS-207, our clinical development program was designed to quickly generate data that could inform timely decision-making and allow us to prioritize our portfolio accordingly," said Stephen Isaacs, chairman and chief executive officer of Aduro Biotech. "We will shift our focus and investment toward our STING agonist program, B-select antibodies and personalized neoantigen approach with pLADD. In our STING program in particular, there are several additional clinical trials under consideration to complement our ongoing Phase 1 dose escalation trial of ADU-S100 as well as our combination study with Novartis’ PD-1 checkpoint inhibitor, PDR-001. As a result of our portfolio decisions, we expect our current cash balance to be sufficient to fund planned activities for the next three years through 2020."

Conference Call with Management Today
Aduro’s management will host a conference call to review its programs and provide a general business update today at 8:30 am Eastern Time. To participate in the conference call, please dial (844) 309-0604 (domestic) or (574) 990-9932 (international) and refer to conference ID 2455008. Live audio of the conference call will be simultaneously webcast and available to members of the news media, investors and the general public under the investor section of the Aduro website at investors.aduro.com.

The webcast will be archived and available for replay for one month after the event.