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Agios Announces Data from Ongoing Phase 1 Trial of AG-120 Showing Durable Responses in Patients with Advanced Hematologic Malignancies

On December 5, 2015 Agios Pharmaceuticals, Inc. (Nasdaq:AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, reported new data from the ongoing Phase 1 study evaluating single agent AG-120, a first-in-class, oral, selective, potent inhibitor of mutant isocitrate dehydrogenase-1 (IDH1), in advanced hematologic malignancies (Press release, Agios Pharmaceuticals, DEC 5, 2015, View Source [SID:1234508406]).

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The data are being presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH) (Free ASH Whitepaper) taking place December 5-8, 2015 in Orlando. AG-120 is being developed in collaboration with Celgene.

Data as of October 1, 2015 from 87 patients with advanced IDH1 mutant positive hematologic malignancies confirmed a favorable safety profile consistent with previously reported data and showed durable clinical activity in 78 dose-escalation patients. Twenty-nine patients remain on study as of the analysis. Efficacy data is provided from the dose-escalation phase of the study only, where an overall response rate of 35 percent (27 of 78 response-evaluable patients) and a complete remission rate of 15 percent (12 of 78 response-evaluable patients) were observed. Patients were on study treatment for up to 14.1 months with a median duration of treatment of 2.9 months (ranging from 0.1 to 14.1 months). Data continue to show durable clinical activity for AG-120, with responses maintained for up to 12.5 months and a median duration of response of 5.6 months (previously unreported).

"With the addition of 30 new patients to the study, AG-120 has maintained durable responses and continues to demonstrate an impressive single-agent overall response rate of 35 percent," said Courtney DiNardo, M.D., lead investigator and assistant professor, department of leukemia at the University of Texas MD Anderson Cancer Center. "These data further validate AG-120’s potential to provide clinical benefit for IDH1 mutant relapsed/refractory AML patients with few, if any effective therapeutic options."

"These data increase our confidence in the favorable safety and efficacy profile of AG-120," said Chris Bowden, M.D., chief medical officer of Agios. "Our focus remains on bringing AG-120 to patients with IDH1 mutant cancers as quickly as possible, and we look forward to continuing to enroll our 125-patient expansion arm in relapsed/refractory AML and initiating two frontline studies to reach additional IDH1 mutant AML patients in need of better options."

About the Ongoing Phase 1 Trial for AG-120 in Advanced Hematologic Malignancies

AG-120 is being evaluated in an ongoing Phase 1 trial that includes a dose escalation phase and four expansion cohorts, including:

Arm 1: 125 IDH1 mutant positive AML patients who relapsed after bone marrow transplantation, are in second or later relapse, refractory to second line induction or reinduction treatment

Arm 2: 25 untreated IDH1 mutant positive AML patients who are not candidates for standard-of-care chemotherapy

Arm 3: 25 patients with other non-AML IDH1 mutant, relapsed or refractory advanced hematologic malignancies

Arm 4: 25 patients with relapsed IDH1 mutant positive AML not eligible for arm 1 or standard of care

Data reported are from patients treated with AG-120 administered from 200 mg to 1,200 mg total daily doses as of October 1, 2015. The median age of these patients is 68 (ranging from 36-89). Patients received a median of two prior lines of therapy (ranging from zero to five). A safety analysis was conducted for all 87 treated patients and an efficacy analysis was conducted in the evaluable population of 78 dose-escalation patients, which includes all patients with a pre-AG-120 screening assessment and day 28 or later response assessment or an earlier discontinuation for any reason.

Safety Data

Of the 87 treated patients, 78 were from the dose-escalation phase and nine from the expansion.

The majority of adverse events reported by investigators were mild to moderate, with the most common being fatigue, diarrhea, pyrexia and nausea.
51 patients experienced at least one serious adverse event (SAE), the majority being disease related.
A maximum tolerated dose (MTD) has not been reached.
19 patients discontinued from the study due to death, and all were considered unrelated to AG-120.
All cause mortality at 30 and 60 days was 10.3 percent and 18.4 percent, respectively.

Efficacy Data

Twenty-seven out of 78 response-evaluable patients from the dose-escalation achieved investigator-assessed objective responses for an overall response rate of 35 percent.

Of the 27 patients who achieved an objective response, there were 12 complete remissions (CR), seven CRs with incomplete platelet recovery (CRp), six marrow CRs (mCR), one CR with incomplete hematologic recovery (CRi) and one partial remission (PR).
Patients were on study treatment for up to 14.1 months with a median duration of treatment of 2.9 months (ranging from 0.1 to 14.1 months).

Data continue to show durable clinical activity for AG-120, with responses maintained for up to 12.5 months and a median duration of response of 5.6 months.

2015 Milestones for AG-120 in Hematologic Malignancies

Remaining milestones for AG-120 in 2015 include:

Continue to enroll patients in the expansion cohort of 125 patients with IDH1 mutant positive AML who relapsed after bone marrow transplantation, are in second or later relapse, refractory to second line induction or reinduction treatment.
Initiate a Phase 1b combination study of either AG-221 or AG-120 with standard induction (7+3, Ara-C and idarubicin/daunorubicin) and consolidation (Ara-C, or mitoxantrone with etoposide) chemotherapy in newly diagnosed AML patients eligible for intensive chemotherapy by the end of 2015.

Investor Event and Webcast Information

Agios will host an investor event on Monday, December 7, 2015 beginning at 12:00 p.m. ET in Orlando to review data presented at ASH (Free ASH Whitepaper), including new data from the ongoing studies of AG-221 and AG-120. The event will be webcast live and can be accessed under "Events & Presentations" in the Investors and Media section of the company’s website at www.agios.com.

About IDH Mutations and Cancer

IDH1 and IDH2 are two metabolic enzymes that are mutated in a wide range of hematologic and solid tumor malignancies. Normally, IDH enzymes help to break down nutrients and generate energy for cells. When mutated, IDH increases production of an oncometabolite 2-hydroxyglutarate (2HG) that alters the cells’ epigenetic programming, thereby promoting cancer. 2HG has been found to be elevated in several tumor types. Agios believes that inhibition of the mutated IDH proteins may lead to clinical benefit for the subset of cancer patients whose tumors carry them.

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. Less than 10 percent of U.S. AML patients are eligible for bone marrow transplant and 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. IDH1 and IDH2 mutations are present in about 15 to 23 percent of AML cases.

TG Therapeutics, Inc. Recaps Schedule of Data Presentations at the 57th American Society of Hematology Annual Meeting

On December 04, 2015 TG Therapeutics, Inc. (Nasdaq:TGTX) reported the schedule of data presentations for their lead compounds at the upcoming 57th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH) (Free ASH Whitepaper), to be held December 5-8, 2015, at the Orange County Convention Center in Orlando, Florida (Press release, TG Therapeutics, DEC 4, 2015, View Source [SID:1234508403]).

Presentations on TG-1101 and TGR-1202 at the ASH (Free ASH Whitepaper) meeting include the following:

Clinical Posters:

Title: Ublituximab + TGR-1202 Demonstrates Activity and Favorable Safety Profile in Relapsed/Refractory B-Cell NHL and High-Risk CLL: Phase I Results
Abstract Number: 1538
Session: 624. Lymphoma: Therapy with Biologic Agents, excluding Pre-Clinical Models: Poster I
Date and Time: Saturday, December 5, 2015; 5:30 PM- 7:30 PM ET
Location: Orange County Convention Center, Hall A
Presenter: Matthew Lunning, DO

Title: A Phase I Trial of TGR-1202, a Next Generation Once Daily PI3K-Delta Inhibitor in Combination with Obinutuzumab Plus Chlorambucil, in Patients with Chronic Lymphocytic Leukemia
Abstract Number: 2942
Session: 642. CLL: Therapy, excluding Transplantation: Poster II
Date and Time: Sunday, December 6, 2015; 6:00 PM-8:00 PM ET
Location: Orange County Convention Center, Hall A
Presenter: Daruka Mahadevan, MD, PhD

Title: Ublituximab (TG-1101), A Novel Glycoengineered Anti-CD20 Monoclonal Antibody, in Combination With Ibrutinib is Highly Active in Patients With Relapsed And/Or Refractory Mantle Cell Lymphoma; Results of a Phase II Trial
Abstract Number: 3980
Session: 624. Lymphoma: Therapy with Biologic Agents, excluding Pre-Clinical Models: Poster III
Date and Time: Monday, December 7, 2015; 6:00 PM- 8:00 PM ET
Location: Orange County Convention Center, Hall A
Presenter: Kathryn Kolibaba, MD

Title: TGR-1202, a Novel Once Daily PI3K-Delta Inhibitor, Demonstrates Clinical Activity with a Favorable Safety Profile in Patients with CLL and B-Cell Lymphoma
Abstract Number: 4154
Session: 642. CLL: Therapy, excluding Transplantation: Poster III
Date and Time: Monday, December 7, 2015; 6:00 PM- 8:00 PM ET
Location: Orange County Convention Center, Hall A
Presenter: Owen O’Connor, MD, PhD

Non-Clinical Oral Presentation:

Title: Disruption of the mTOR-eIF4F Axis By Selectively Targeting PI3Kdelta and Proteasome Potently Inhibits Cap Dependent Translation of c-Myc in Aggressive Lymphomas
Abstract Number: 593
Oral Session: 625. Lymphoma: Pre-Clinical – Chemotherapy and Biologic Agents: Novel Therapies and Targets in Lymphoma
Date and Time: Monday, December 7, 2015; 10:30 AM – 12:00 PM ET
Presentation Time: 11:30 AM ET
Location: Orange County Convention Center, Tangerine 1 (WF1)
Presenter: Changchun Deng, MD, PhD

A copy of the above referenced abstracts can be viewed online through the ASH (Free ASH Whitepaper) meeting website at www.hematology.org.

TG Therapeutics will also host a reception on Monday, December 7th, 2015 beginning at 7:45pm ET, with featured presentations beginning promptly at 8:00pm ET. The event will take place at the Hyatt Regency Orlando in the Bayhill 17/18 Room. This event will be webcast live and will be available on the Events page, located within the Investors & Media section of the Company’s website at www.tgtherapeutics.com, as well as archived for future review. This event will also be broadcast via conference call. In order to access the conference line, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), and reference Conference Title: TG Therapeutics 2015 Investor & Analyst Event.

Stemline Therapeutics Announces Five Presentations, Including SL-401 Clinical Update From Ongoing Pivotal BPDCN Trial, at the Upcoming American Society of Hematology (ASH) Annual Meeting

On December 4, 2015 Stemline Therapeutics, Inc. (Nasdaq:STML) reported that SL-401 and SL-801 will be the subject of five poster presentations at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, being held December 5-8, 2015 at the Orange County Convention Center in Orlando, FL (Press release, Stemline Therapeutics, DEC 4, 2015, View Source [SID:1234508402]).

Investigators will present updated clinical data from the lead-in and ongoing expansion stages of the SL-401 pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN), as well as three additional presentations highlighting preclinical data supporting SL-401’s clinical development in mastocytosis as a single agent and in multiple myeloma in combination with approved agents. SL-801 will be the subject of a presentation detailing its broad preclinical anti-cancer activity in both solid and hematologic cancers in anticipation of the start of clinical trials.

Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, commented, "On Monday evening, investigators will present initial efficacy and safety data from the lead-in and ongoing expansion stages of the SL-401 pivotal trial in BPDCN. We are very pleased with the initial outcomes of the ongoing study. Our experience from the lead-in stage enabled us to develop a regimen with specific dosing parameters that, since implementation, has generated a therapeutic window with manageable safety and high levels of clinical activity."

Details on the presentations are listed below and abstracts are available on the ASH (Free ASH Whitepaper) conference website. Additionally, all abstracts and posters will be available on the Stemline website soon after the presentations.

SL-401 Presentations

Lead-in Stage Results of a Pivotal Trial of SL-401, an Interleukin-3 Receptor (IL-3R) Targeting Biologic, in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) or Acute Myeloid Leukemia (AML)
Lead Author: Marina Konopleva, MD, PhD
Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX
Date: Monday, December 7, 2015
Presentation Time: 6:00 PM – 8:00 PM
Location: Orange County Convention Center, Hall A

A Novel Agent SL-401 Triggers Anti-Myeloma Activity By Targeting Plasmacytoid Dendritic Cells: Implications for a Novel Immune-Associated Mechanism
Lead Author: Arghya Ray, Ph.D.
The LeBow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center, Department of Medical Oncology, Dana Farber Cancer Institute, Harvard Medical School, Boston, MA
Date: Sunday, December 6, 2015
Presentation Time: 6:00 PM – 8:00 PM
Location: Orange County Convention Center, Hall A

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Patient-Derived Xenografts Are Faithful Genomic and Phenotypic Models of Primary Leukemia and Respond to the IL3 Receptor Targeting Agent SL-401 In Vivo
Lead Author: Amanda Christie, B.A.
Department of Hematologic Oncology, Dana Farber Cancer Institute, Harvard Medical School, Boston, MA
Date: Monday, December 7, 2015
Presentation Time: 6:00 PM – 8:00 PM
Location: Orange County Convention Center, Hall A

CD123 Immunostaining in Systemic Mastocytosis: Differential Expression in Disease Subgroups and Potential Prognostic Value
Lead Author: Animesh Pardanani, MBBS, Ph.D.
Department of Hematology, Mayo Clinic College of Medicine, Rochester, MN
Date: Sunday, December 6, 2015
Presentation Time: 6:00 PM – 8:00 PM
Location: Orange County Convention Center, Hall A

SL-801 Presentations

SL-801, a Novel, Reversible Inhibitor of Exportin-1 (XPO1) / Chromosome Region Maintenance-1 (CRM1) with Broad and Potent Anti-Cancer Activity
Lead Author: Janice Chen, Ph.D.
Stemline Therapeutics, Inc., New York, NY
Date: Monday, December 7, 2015 ?
Presentation Time: 6:00 PM – 8:00 PM ?
Location: Orange County Convention Center, Hall A

Spectrum Pharmaceuticals Highlights 18 Abstracts at the 57th Annual Meeting of the American Society of Hematology (ASH) in Orlando, Florida, December 5-8, 2015

On December 4, 2015 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in Hematology and Oncology, reported key presentations of clinical and scientific data related to its products at the 57th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), being held in Orlando, Florida, from December 5-8, 2015 (Press release, Spectrum Pharmaceuticals, DEC 4, 2015, View Source [SID:1234508399]).

For more information about the ASH (Free ASH Whitepaper) annual meeting and for a complete list of abstracts, please refer to the conference website at View Source

Merrimack Announces Presentations at the 2015 San Antonio Breast Cancer Symposium

On December 4, 2015 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) reported that it will present on its innovative nanoliposomal platform for patients with metastatic breast cancer at the 2015 San Antonio Breast Cancer Symposium, December 8 – 12, 2015 at the Henry B. Gonzalez Convention Center in San Antonio, Texas (Press release, Merrimack, DEC 4, 2015, View Source [SID:1234508397]).

The presentations include a trials-in-progress abstract for the MM-302 HERMIONE Phase 2 clinical trial and a trials-in-progress abstract for the Phase 1 clinical trial investigating potential predictive response markers for ONIVYDE (irinotecan liposome injection), also known as MM-398 or "nal-IRI." MM-302 is a HER2 targeted liposomal encapsulation of doxorubicin.

Poster Sessions:

HERMIONE: A phase 2, randomized, open label trial comparing MM-302 plus trastuzumab with chemotherapy of physician’s choice plus trastuzumab, in anthracycline naive HER2-positive, locally advanced/metastatic breast cancer patients previously treated with pertuzumab and ado-trastuzumab emtansine (T-DM1) (Abstract OT3-01-01)
Ongoing Trials Poster Session 3, Ongoing Trials – HER2
Presentation: Friday, December 11, 2015 (5:00 PM CT)
Exhibit Hall A-B

A phase 1 study in patients with metastatic breast cancer to evaluate the feasibility of magnetic resonance imaging with ferrumoxytol as a potential biomarker for response to treatment with nanoliposomal irinotecan (nal-IRI, MM-398) (Abstract OT3-02-14)
Ongoing Trials Poster Session 3, Ongoing Trials – Chemotherapy
Presentation: Friday, December 11, 2015 (5:00 PM CT)
Exhibit Hall A-B