GlycoMimetics Announces High Overall Response Rates, Low Induction Mortality, Promising Initial Survival Outcomes, and Supportive Biomarker Data from Phase 1/2 Trial of GMI-1271 in AML

On May 18, 2017 GlycoMimetics, Inc. (NASDAQ: GLYC) reported the release of abstracts containing new data from the ongoing Phase 2 clinical trial of its product candidate GMI-1271, an E-selectin antagonist, in patients with acute myeloid leukemia (AML) (Press release, GlycoMimetics, MAY 18, 2017, View Source [SID1234519253]). The data will be presented at the June annual meetings of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the European Hematology Association (EHA) (Free EHA Whitepaper). The data released by ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper), which reflect a late-January analysis, will be updated in posters presented at both meetings.

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In the ongoing Phase 2 trial, AML patients treated with GMI-1271, combined with chemotherapy, continue to experience higher-than-expected remission rates and lower-than-expected induction-related mortality rates in both arms of the trial. In addition, researchers have observed that baseline expression of the E-selectin ligand biomarker on leukemia cells was predictive of clinical response and tied to greater likelihood of achieving remission in the cohort of AML patients with relapsed/refractory disease, which supports the mechanism of action of GMI-1271. Treatment with GMI-1271 continues to be well tolerated, with no obvious incremental toxicity observed when GMI-1271 is added to chemotherapy.

According to Helen Thackray, MD, Chief Medical Officer, "The data has consistently shown good tolerability and high remission rates as well as lower than expected 30- and 60-day mortality rates in early evaluations of patients. We are increasingly confident that our investigational drug, GMI-1271, may play a role in addressing unmet needs in this cancer. It is particularly noteworthy to see in the relapsed/refractory cohort that patients who have higher levels of the E-selectin ligand biomarker on their leukemic blasts appear to be more likely to achieve remission of their disease. This observation builds directly on what we and others have reported in the preclinical and clinical settings about the key role E-selectin plays in many forms of cancer, including AML. Importantly, this provides what we believe is the first direct clinical evidence of the potential benefit of targeting of E-selectin in this difficult-to-treat population of AML patients."

Relapsed or Refractory Disease Arm: Abstract Data

Consistent with GlycoMimetics’ prior published research, the addition of GMI-1271 to mitoxantrone, etoposide and cytarabine (MEC) chemotherapy has been well-tolerated, with patients achieving a high overall response rate (ORR), low induction mortality, and promising initial survival outcomes. The data show that baseline expression of the E-selectin ligand biomarker was predictive of response. GlycoMimetics believes these results are better than what would be expected in this population, based on published historical controls in similar patients.

Highlights of the data reported in the published abstract include:

47 patients were enrolled.
30- and 60-day mortality were 0 and 7%, respectively.
ORR was 21/42 evaluable (50%).
Median Overall Survival in the Phase 1 portion was 7.6 months.
The median E-selectin ligand binding at baseline was 35% of blasts (range, 1-75%) and, importantly, was higher in those achieving remission.
The data from the ongoing Phase 2 trial were submitted to the U.S. Food and Drug Administration (FDA). As announced yesterday, GMI-1271 was granted Breakthrough Therapy designation from the FDA for the treatment of adult AML patients with relapsed/refractory disease. The FDA had previously granted Orphan Drug designation and Fast Track Status for GMI-1271 for the treatment of AML.

Newly Diagnosed, Treatment-Naïve, Elderly Arm: Abstract Data

In the published abstract, data reflects 17 of 24 enrolled and evaluable elderly patients. Highlights from the abstract include:

The remission rate (CR/CRi) was 12/17 (71%).
CR/CRi rate was 75% for patients with de novo disease and 67% for patients with secondary AML.
GlycoMimetics noted that the safety profile of the investigational drug, GMI-1271, in combination with chemotherapy is encouraging. Outcomes for elderly patients with AML remain poor, and tolerability of treatments is a key concern.

Deciphera Pharmaceuticals to Report Phase 1 Clinical Study Results with DCC-2618 at the 2017 American Society of Clinical Oncology Annual Meeting

On May 18, 2017 Deciphera Pharmaceuticals, a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported that a poster presentation that includes updated results from the ongoing Phase 1 clinical study of DCC-2618, the company’s pan-KIT and PDGFR inhibitor, will be presented at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place June 2 – 6, 2017, in Chicago, IL (Press release, Deciphera Pharmaceuticals, MAY 18, 2017, View Source [SID1234519252]). Details of the presentation are as follows:

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Poster Title: Pharmacokinetic-driven phase 1 study of DCC-2618 a pan-KIT and PDGFR inhibitor in patients with gastrointestinal stromal tumor (GIST) and other solid tumors.
Author: Filip Janku, M.D., Ph.D., The University of Texas MD Anderson Cancer Center
Session: Poster Discussion Session: Developmental Therapeutics – Clinical Pharmacology and Experimental Therapeutics
Abstract #: 2515
Date and Time: Monday, June 5, 2017, 8:00 AM – 11:30AM (CST)
Poster Discussion Session: Monday, June 5, 2017 11:30 AM-12:45 PM (CST)

"We are very pleased to present these important results and to provide an update on the clinical development of DCC-2618, our pan-KIT and PDGFR inhibitor in development for difficult to treat, KIT and/or PDGFRα-driven cancers with limited therapeutic options," said Michael D. Taylor, Ph.D., Deciphera’s President and Chief Executive Officer.

"These maturing data confirm earlier results presented at the 2016 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics," added Oliver Rosen, M.D., Deciphera’s Chief Medical Officer. "The encouraging clinical activity achieved in patients with a broad spectrum of mutations and prior therapies in our Phase 1 study supports the impressive translational data presented last month at the 2017 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting".

About DCC-2618
DCC-2618 is currently in a first-in-human Phase 1 clinical trial. DCC-2618 is a pan-KIT and PDGFRα kinase switch control inhibitor in clinical development for the treatment of KIT and/or PDGFRα-driven cancers, including gastrointestinal stromal tumors, glioblastoma multiforme and systemic mastocytosis.

SYROS TO PRESENT ON SY-1425 IN COMBINATION WITH STANDARD-OF-CARE AND TARGETED THERAPIES IN GENOMICALLY DEFINED SUBSETS OF AML AND MDS PATIENTS

On May 18, 2017 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of disease-driving genes, reported that the Company will present preclinical data on SY-1425, its oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, in combination with hypomethylating agents and anti-CD38 therapies in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) at the 22nd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) taking place June 22-25 in Madrid, Spain (Press release, Syros Pharmaceuticals, MAY 18, 2017, View Source;p=irol-newsArticle&ID=2273936 [SID1234519251]).

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Details on the presentation are as follows:

Date & Time: Friday, June 23, from 5:15 – 6:45 p.m. CEST
Presentation Title: Mechanistically Informed Combinations of SY-1425, a Potent and Selective RARα Agonist, with Hypomethylating or Anti-CD38 Targeted Agents in AML and MDS
Session Title: Acute myeloid leukemia – Biology 2
Presenter: Michael R. McKeown, Ph.D., Senior Scientist, Translational Medicine, Syros
Abstract Number: P188
Location: IFEMA – Feria de Madrid, Poster area (Hall 7)

SY-1425 is currently in a Phase 2 clinical trial exploring its safety and efficacy as both a monotherapy and in combination with azacitidine, a standard-of-care hypomethylating agent, in subsets of AML and MDS patients with high expression of RARA pathway-associated genes RARA and IRF8.

ERYTECH Provides Business Update and Financial Highlights For Q1 2017

On May 18, 2017 ERYTECH Pharma (Paris:ERYP) (ADR:EYRYY) (Euronext Paris: ERYP), the French biopharmaceutical company developing ‘tumor starvation’ treatments for acute leukemia and other oncology indications with unmet medical needs, reported provided a financial and business update for the first quarter of 2017 ended March 31, 2017 (Press release, ERYtech Pharma, MAY 18, 2017, View Source;p=irol-newsArticle&ID=2274066 [SID1234519250]).

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Business Highlights

ERYTECH reported positive Phase 2b data from the French multi-center study of Eryaspase for the treatment of metastatic pancreatic cancer. Results from the study showed significant improvement in both progression-free survival (PFS) and overall survival (OS). The study assessed eryaspase, L-asparaginase encapsulated in red blood cells, as a second-line treatment in combination with chemotherapy in 140-patients with metastatic pancreatic cancer. Eryaspase was added to the standard of care (gemcitabine or FOLFOX) and compared to the standard of care alone in a 2-to-1 randomization.
The company successfully raised €70.5 million in a private placement. ERYTECH issued 3,000,000 new ordinary shares. The company intends to utilize the proceeds from this capital raise to prepare the company for the further clinical development of its pipeline candidates, including the launch of a potential Phase 3 trial in pancreatic cancer and evaluation of clinical development opportunities for eryaspase (GRASPA) in other solid tumor indications. A portion of the capital will be used for general corporate purposes and working capital.
ERYTECH announced the launch of a single arm, multi-center, multi-national investigator-initiated Phase 2 study of eryaspase in approximately 30 acute lymphoblastic leukemia (ALL) patients at 23 sites across 7 Nordic and Baltic countries. The study aims to evaluate the biological activity, safety, and immunogenicity profile of eryaspase in combination with the NOPHO ALL 2008 multi-agent chemotherapy protocol as a second-line therapy in pediatric and adult ALL patients (1 to 45 years old) who experienced hypersensitivity reactions to PEG-asparaginase or silent inactivation.
ERYTECH presented promising preclinical findings on the anti-tumor activity of its new product candidate, erymethionase, at the 2017 ASCO (Free ASCO Whitepaper) GI Symposium and at AACR (Free AACR Whitepaper) 2017. Data obtained from the study demonstrated that erymethionase in combination with daily vitamin B6 supplementation can inhibit tumor growth in a murine model of human gastric adenocarcinoma.
The company entered into a collaboration with Fox Chase Cancer Center for the preclinical development of erymethionase in homocystinuria, a rare inherited disease caused by a deficiency in the enzyme cystathionine beta-synthase (CBS), which is critical for normal methionine metabolism. Under this collaboration, FCCC and ERYTECH will study the potential of erymethionase to lower homocysteine and methionine in the homocystinuria mouse model (CBS-deficient mice) developed at FCCC.
The company presented encouraging preclinical data supporting ERYMMUNE as a potentially strong immunotherapy at the World ADOPT Summit 2017 and 10th Symposium of Vaccinology. Study results showed that ERYMMUNE technology was responsible for a inducing an efficient and antigen-specific immune response for effective cancer immunotherapy.
Financial Highlights

ERYTECH’s key financial figures for the first quarter of 2017, compared with the same period of the previous year are summarized below:
Key figures (in thousands of euros):

Q1 (3 months)
2017 Q1 (3 months)
2016 Variation
Revenues 0 0 0
Other income 1,222 684 538
Total operating income 1,222 684 538
Operating expenses:
Research & development (5,847) (3,638) (2,209)
General & administrative (1,906) (1,473) (433)
Total operating expenses (7,753) (5,111) (2,642)
Operating loss (6,531) (4,427) (2,104)

Financial income 21 97 (77)
Income tax (13) 4 (17)
Net Loss (6,523) (4,325) (2,198)
Net loss for the first quarter of 2017 was €6.5 million, compared to net loss of €4.3 million for the same period of last year. The €2.2 million increase reflected the increased activity to advance the company’s preclinical and clinical development programs. The increase was driven by higher service and contracting fees, mostly related to the clinical and regulatory progress of product development projects, and higher personnel costs, following the staffing of key positions in the preclinical, clinical and pharmaceutical operations domains. Other income, which was mostly comprised of research and development tax credits and grants, increased in the same proportion.

As of March 31, 2017, ERYTECH had cash and cash equivalents totaling €30.5 million, compared with €40.6 million on March 31, 2016. Total net cash utilization was €7.1 million in the first quarter of 2017, which compares with net cash utilization of €5.1 million in the first quarter of 2016. The net loss of the period as well as the net cash utilization in the first quarter of 2017 reflected the increased activity in product development and supporting company’s operations.
A private placement to U.S. and European investors was completed in April 2017. The net proceeds of approximately €64.5 million will enable the company to fund the continued development of its product candidates, and mainly to finance the preparatory steps for the launch of contemplated Phase 3 studies in pancreatic cancer and adult ALL. The company is also sufficiently funded to assess the clinical development opportunities of eryaspase (GRASPA) for the treatment of other solid tumor indications, in addition to its ongoing preclinical and clinical programs.
Gil Beyen, ERYTECH’s Chief Executive Officer commented, "In the first quarter of 2017, we made tremendous progress in our clinical and preclinical R&D programs. We are very pleased with the highly positive results from our Phase 2b trial of eryaspase in metastatic pancreatic cancer patients. The findings from this trial further validate our eryaspase product candidate and our technology platform, and we are now determining next steps to advance the product candidate in pancreatic cancer and in other solid tumors. All of this without losing sight of our development in acute leukemia. The work for resubmission of our European Marketing Authorization Application in ALL is ongoing and the launch of an investigator initiated trial in collaboration with the Nordic Society of Pediatric Hematology and Oncology shows our commitment to bringing GRASPA to ALL patients in need. Next to eryaspase, we have made noteworthy advances in our preclinical programs, notably with erymethionase and our ERYMMUNE platform for which we presented promising preclinical results at different conferences. The very successful private placement in April confirms investors’ confidence in our programs and provides a strong basis to prepare the company for the implementation of its ambitious value-creation strategy. We look forward to accomplishing more milestones this year, including the resubmission of our European MAA for GRASPA in relapsed and refractory ALL, regulatory interactions regarding our further developments in ALL and pancreatic cancer both in Europe and the US, and the Phase 2 results of our AML study."

First Quarter 2017 Conference Call Details

Investors and analysts wishing to participate can access the call via the following teleconferencing numbers:

USA: +1 6467224907 United-Kingdom: +44 2030432440
Switzerland: +41 225809022 Germany: +49 69222229031
France: +33 172001510 Belgium: +32 24029640
Sweden: +46 850334664 Finland : +358 942599700
Netherlands: +31 107138194
Confirmation Code: 64683084#

The webcast can be followed live online via the link:
View Source;Name=&Conference=135308821&PIN=64683084

Following the live call, a replay will be available for 90 days. To listen to the replay, please dial:

USA: +1 877 64 230 18
United-Kingdom: +44(0) 2033679460
France: +33(0)1 72 00 15 00
Confirmation Code: 308821#

Additionally, an archive of the webcast will be available on the "Webcast" section of the Company’s investor relations site at www.erytech.com

Next financial updates:

Financial highlights for the 2nd quarter of 2017: September 11, 2017 (after market close), followed by a conference call and webcast on September 12, 2017 (3:00pm CET/9:00am ET)
Upcoming participations at investor conferences:

BioEquity Europe 2017, May 22-23, Paris
Biotech Agora Conference, May 23, Paris
Gilbert Dupont Annual Healthcare Conference, May 30, Paris
Jefferies 2017 Global Healthcare Conference, June 6-9, New-York
Journée Valeurs Moyennes (SFAF), June 13, Paris
European MidCap Spring Event, June 28, Paris

Bellicum Announces Clinical Presentations at the 22nd Congress of the European Hematology Association

On May 18, 2017 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported that three abstracts have been accepted for presentation at the 22nd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) being held in Madrid, Spain on June 22-25, 2017 (Press release, Bellicum Pharmaceuticals, MAY 18, 2017, View Source;p=RssLanding&cat=news&id=2273891 [SID1234519217]).

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Bellicum’s presentation of the overall cohort of children with malignant and non-malignant diseases treated with BPX-501 was selected as one of the Congress’ five best abstracts, and will be reviewed during the Presidential Symposium.

Additional oral and poster presentations selected include clinical data on BPX-501 for the treatment of pediatric leukemias, hemoglobinopathies and erythroid disorders. The abstracts are now available online at the EHA (Free EHA Whitepaper) conference website.

EHA Presentation Details

Oral Presentation – Presidential Symposium
Title: BPX-501 Donor T-Cell Infusion (with Inducible Caspase 9 Suicide Gene) Facilitates HLA Haploidentical Stem Cell Transplant in Children with Both Hematological Malignancies and Non-Malignant Conditions
Session Title: Presidential Symposium
Date: Friday, June 23
Time: 3:45 – 4:00 PM CEST
Location: Hall A
Abstract Code: S146

Oral Presentation
Title: Impact of Post-Transplant Infusion of Donor T-Cells Genetically Modified with Inducible Caspase 9 Suicide Gene (BPX-501 Cells) on Children with Leukemia Given Alpha-Beta T-Cell Depleted Haplo-HSCT
Session Title: Stem cell transplantation – Clinical 1
Date: Saturday, June 24
Time: 5:00 – 5:15 PM CEST
Location: Room N103
Abstract Code: S495

Poster Presentation
Title: The Use of BPX-501 Donor T-Cell Infusion (with Inducible Caspase 9 Suicide Gene) Together with HLA-Haploidentical Stem Cell Transplant to Treat Children with Hemoglobinopathies and Erythroid Disorders
Session Title: Stem cell transplantation – Clinical 1
Date: Friday, June 23
Time: 5:15 – 6:45 PM CEST
Location: Poster Area (Hall 7)
Abstract Code: P381