Full Press Release Details
Agios Announces Phase 1 Data from Dose Expansion Cohorts of AG-120 in Patients with
IDH1 Mutant Positive Glioma and Chondrosarcoma
- Durable Stable Disease Observed for Patients with Non-enhancing Low Grade Glioma and
Chondrosarcoma Patients; 42% of Glioma Patients Still on Treatment
- AG-120 Well-tolerated in Pre-Treated Populations -
- Potential for Centralized Volumetric Assessments to Shape Future Development in Glioma -
CAMBRIDGE, Mass., November 18, 2016 Agios Pharmaceuticals, Inc. (Nasdaq:AGIO), a leader in the fields of cancer metabolism and rare genetic
metabolic disorders, today announced the first data from the dose expansion cohorts of the Phase 1 study evaluating single agent AG-120 in isocitrate dehydrogenase-1 (IDH1) mutant positive glioma and chondrosarcoma. The glioma data were presented
today at the Society for Neuro-Oncology (SNO) Annual Meeting in Scottsdale, AZ and the chondrosarcoma data were presented last week at the annual meeting of the Connective Tissue Oncology Society (CTOS) in Lisbon, Portugal.
Glioma and chondrosarcomas are extremely difficult-to-treat diseases where patients are in need of new therapies, said Chris Bowden, M.D., chief
medical officer at Agios. These Phase 1 dose expansion data are encouraging, as they continue to demonstrate a well-tolerated safety profile for AG-120 at a fixed daily dose of 500 mg. The prolonged stable disease in both patient populations
is encouraging in light of AG-120 s unique differentiation mechanism of action. In addition, our initial experience utilizing centralized volumetric assessments in patients with glioma has been informative, and along with our ongoing AG-881
Phase 1 trial, will help determine the next steps in clinical development.
In glioma, AG-120 has the potential to help a large number of
patients with IDH1 mutations, said Ingo Mellinghoff, M.D., Memorial Sloan Kettering Cancer Center, an investigator for the study. The SNO presentation is the first look at data for AG-120 in a defined cohort of glioma patients where we
evaluated the potential for volumetric analyses to improve our understanding of the response patterns beyond the conventional bi-dimensional methods. This methodology could be instrumental in developing more effective, targeted therapies for
patients with this disease.
The Phase 1 trial is assessing the safety and tolerability of AG-120 in advanced solid tumors, including glioma,
intrahepatic cholangiocarcinoma (IHCC) and chondrosarcomas that harbor an IDH1 mutation. The dose-escalation phase was followed by four expansion cohorts in the following patient groups.
Enrollment is now complete for the dose escalation and expansion
Glioma Expansion Data Presented at SNO Annual Meeting
As of the August 1, 2016 data cut off, 66 patients have been treated with single agent AG-120, and 28 patients (42%) remain on treatment.
A safety analysis conducted for all 66 treated patients as of the data cut-off demonstrated that AG-120 was well-tolerated with a favorable safety profile in
from 65 response-evaluable patients as of the data cut-off showed:
Chondrosarcoma Expansion Data Presented at CTOS Annual Meeting
Agios also analyzed data from 21 chondrosarcoma patients as of September 23, 2016 in the dose escalation (n=12) and expansion cohorts (n=9) and 7 remain
Glioma presents in varying degrees of tumor
aggressiveness, ranging from slower growing (low grade glioma) to rapidly progressing (high grade glioma-Glioblastoma Multiforme). Common symptoms include seizures, memory disturbance, sensory impairment neurologic deficits and seizures. The
long-term prognosis is poor with a five-year survival rate of 33 percent. Median survival is 12-15 months for glioblastoma and 2-5 years for anaplastic glioma. IDH1 mutations are highly prevalent, accounting for approximately 68-74 percent of low
grade glioma and secondary glioblastoma.
About Chondrosarcoma
Chondrosarcoma is a heterogeneous group of cancers that arise from cartilage in the bone and joint. It is the most common type of bone cancer with 700-1,000
people diagnosed per year in the U.S. IDH1/2 mutations occur in approximately 46-63 percent of central chondrosarcomas. The prognosis is based on disease burden for localized disease, there is curative potential with surgery, but metastatic
disease has a low five-year survival rate. Radiation is not effective, and chemotherapy is of limited benefit. Treatment for metastatic disease is mainly palliative.
Agios Pharmaceuticals is focused on
discovering and developing novel drugs to treat cancer and rare genetic disorders of metabolism 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 cancer metabolism and rare genetic disorders of metabolism in clinical and/or preclinical development. All Agios programs
focus on genetically identified patient populations, leveraging the company s knowledge of metabolism, biology and genomics. For more information, please visit Agios website at
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking
statements include those regarding the potential of IDH1/IDH2 as therapeutic targets; the potential benefits of Agios product candidates targeting IDH1/IDH2, including AG-120 and AG-881; its plans regarding future data presentations; and the
potential benefit of its strategic plans and focus. The words anticipate, believe, estimate, expect, intend, may, plan, predict, project,
would, could, potential, possible, hope and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying
words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios current expectations and beliefs. For example, there can be no guarantee that any
product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios product candidates will successfully continue. There can be no guarantee
that any positive developments in Agios business will result in stock price appreciation. Management s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties
relating to a number of other important factors, including: Agios results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing
of decisions made by the U.S. FDA and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios ability to obtain and maintain requisite regulatory approvals and to enroll patients
in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing;
Agios ability to maintain key collaborations, such as its agreements with Celgene; and general economic and market conditions. These and other risks are described in greater detail under the caption Risk Factors included in
Agios Quarterly Report on Form 10-Q for the quarter ended September 30, 2016, and other filings that Agios may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release
speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Agios Pharmaceuticals:
Kendra Adams, 617-844-6407
Senior Director, Investor &
Renee Leck, 617-649-8299
Senior Manager, Investor & Public Relations