Agios Pharmaceuticals Reports Fourth Quarter and Full Year 2014 Financial Results
- AG-221 global registration program in hematologic malignancies expected to commence in second half 2015
- AG-120 Phase 1 expansion cohorts in hematologic malignancies expected to start in first half 2015, and first data from AG-120 Phase 1 solid tumor study expected to be presented in second half 2015
- AG-348 Phase 2 study in patients with pyruvate kinase deficiency expected to start in first half 2015
"Since our inception, we have remained steadfast in our commitment to patients, our vision and our culture in order to fundamentally change patients lives through our scientific leadership in dysregulated metabolism and development using precision medicine," said
Dr. Schenkein continued, "I am also thrilled to welcome to our leadership team
RECENT DEVELOPMENT PROGRAM AND BUSINESS HIGHLIGHTS
Cancer Metabolism: IDH Mutant Inhibitors in Collaboration with
AG-221: a first-in-class, oral, selective, potent inhibitor of the mutated IDH2 protein being evaluated in a broad range of blood and solid tumor cancers
- Agios continues to conduct the Phase 1 study in IDH2-mutant positive advanced hematologic malignancies, including acute myeloid leukemia (AML), with the primary goals of establishing the safety profile, determining the maximum tolerated dose (MTD) and assessing the clinical activity of AG-221 as a single agent. In parallel, the company continues to enroll patients in four expansion cohorts of 25 patients each as part of the Phase 1 study to assess the safety and tolerability of AG-221 once daily in patients with IDH2-mutant positive hematologic malignancies.
December 2014at the 56th Annual American Society of Hematology(ASH) Annual Meeting, new data in 73 patients with several types of advanced hematologic malignancies, including AML, were presented from the ongoing Phase 1 dose escalation study of AG-221. The data showed a favorable safety profile of AG-221 as a single agent as well as durable clinical activity for up to eight months and ongoing, with an overall response rate of 56 percent (25 of 45 evaluable) in these patients.
October 2014, Agios initiated a Phase 1/2 trial of AG-221 in patients with advanced solid tumors, including gliomas, as well as angioimmunoblastic T-cell lymphoma (AITL). This is designed to explore AG-221's potential beyond hematologic malignancies in a broad range of cancers that harbor an IDH2 mutation.
AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein being evaluated in a broad range of blood and solid tumor cancers
- Agios continues to advance two separate Phase 1 clinical trials evaluating AG-120 in patients with IDH1-mutant advanced hematologic malignancies and solid tumors, including glioma. The Phase 1 trials are multicenter, open-label, dose-escalation clinical studies designed to assess the safety and tolerability of AG-120 as a single agent in these cancers.
November 2014, the first data from the ongoing Phase 1 study of AG-120 in patients with IDH1-mutant advanced hematologic malignancies were presented at the 26th Annual EORTC-NCI-AACR Symposium. The data from 17 patients with relapsed and/or refractory AML showed a promising favorable safety profile, objective responses in seven out of 14 evaluable patients, including four complete remissions, early evidence of durability and a reduction of the 2HG biomarker. These results provided early validation of mutant IDH1 as a target in AML.
January 2015, Celgeneagreed that it would exercise its option to obtain an exclusive license outside the United Statesin accordance with the terms of collaboration agreement between the parties, subject to receipt of any required regulatory approvals, including any applicable clearance under the Hart-Scott-Rodino Act. Agios retains U.S. development and commercial rights to AG-120.
Rare Genetic Disorders of Metabolism: Wholly Owned PKR Activator
AG-348: a novel, first-in-class, oral activator of pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency
- Dosing has been completed in the Phase 1 multiple ascending dose (MAD) clinical trial of AG-348 in healthy volunteers.
December 2014at the 56th Annual ASH Annual Meeting, Agios researchers presented final clinical data from its Phase 1 single ascending dose (SAD) clinical trial and data from the first two cohorts of the Phase 1 MAD clinical trial of AG-348 in healthy volunteers. The data showed early proof-of-mechanism for AG-348 through substantial effects on two key biomarkers of pyruvate kinase activity and pathway activation.
- A natural history study of PK deficiency is also ongoing and has enrolled approximately 100 patients in more than 20 clinical sites globally. Natural history studies are important to confirm and further understand clinical characteristics, symptoms and disease complications and potentially support the design of future clinical trials.
Agios announced today that the company has expanded its leadership team. Agios appointed
Megan Paceto the newly created position of senior vice president of strategic operations and communications. Prior to joining Agios, Ms. Pace was senior vice president, corporate communications at Vertex Pharmaceuticals. Before that, she was senior director, public affairs and advocacy relations at Genentech, Inc.Agios also appointed Melissa McLaughlinto vice president of human resources (HR). Ms. McLaughlin was former vice president, HR for Hotels.com and the Expedia Affiliate Network. Before that, she held various HR leadership positions at Johnson & Johnson and The Gillette Company.
December 2014, Celgeneelected to extend the period of its exclusivity for an additional year to April 2016under its global strategic collaboration agreement with Agios. The extension marks the final year for the discovery phase of the collaboration and gives Celgenean exclusive option to certain drug candidates generated by Agios' cancer metabolism platform during that time. Agios will receive a $20 millionpayment as a result of the extension, which it expects to receive in the second quarter of 2015.
December 2014, Agios strengthened its board of directors. Its board elected Kaye Foster-Cheekas an independent director and a member of its compensation committee. Ms. Foster-Cheek is the former senior vice president, global human resources at Onyx Pharmaceuticals, Inc.an Amgensubsidiary.
Cancer Metabolism Program in Collaboration with
AG-221: Multiple studies of AG-221 planned for 2015 support speed and breadth in development for patients with cancers that carry an IDH2 mutation
- Phase 1 trial in advanced hematologic malignancies: Beginning in mid-2015, Agios expects to present new data from the ongoing Phase 1 trial at medical conferences in the year, including early data from the ongoing expansion cohorts, as well as molecular information from patient data.
- Global registration program: The company expects to initiate in the second half of 2015 a global registration program in hematologic malignancies.
- Frontline therapy trials: Also in the second half of 2015, Agios plans to initiate combination trials to evaluate AG-221 as a potential frontline treatment for patients with AML and a broad range of hematologic malignancies.
- Phase 1/2 solid tumor study: In 2015, Agios expects to continue dose escalation in the Phase 1/2 trial in patients with advanced solid tumors that carry an IDH2 mutation.
AG-120: Multiple studies planned for 2015/2016 support speed and breadth in development for patients with an IDH1 mutation
- Phase 1 trial in advanced hematologic malignancies: The company expects to provide new data from the ongoing Phase 1 study evaluating patients with IDH1 mutant positive advanced hematologic malignancies at a medical conference in mid-2015.
- Phase 1 expansion cohorts in hematologic malignancies: Agios plans to select a dose and schedule from the ongoing Phase 1 study and initiate expansion cohorts for AG-120 in hematologic malignancies as part of its ongoing Phase 1 study in the first half of 2015.
- Phase 1 solid tumor trial: The company expects to present the first data from the dose escalation portion of the Phase 1 advanced solid tumor trial at a medical conference in the second half of 2015.
- Frontline therapy trials: Agios plans to begin combination trials to evaluate AG-120 as a potential frontline treatment of AML and a broad range of hematologic malignancies in the second half of 2015.
- Global registration program: The company plans to initiate a global registration program in hematologic malignancies that harbor an IDH1 mutation by early 2016.
Rare Genetic Disorders of Metabolism
AG-348: First patients with pyruvate kinase (PK) deficiency to be treated in Phase 2 trial
- Phase 1 MAD clinical trial: Agios plans to present final data from the MAD clinical trial in healthy volunteers at a medical conference in mid-2015.
- Phase 2 clinical trial in patients: Agios expects to initiate a Phase 2 trial for AG-348 in the first half of 2015 in patients with PK deficiency, a rare hemolytic anemia.
Natural History study: Agios expects initial data from the study of the natural history of PK deficiency to be reported at a medical conference in mid-2015. This study is being conducted by
Children's Hospitalin Boston.
FULL YEAR 2014 FINANCIAL RESULTS
Cash, cash equivalents and marketable securities as of
Collaboration revenue was
Research and development (R&D) expenses were
General and administrative (G&A) expenses were
Net loss for the year ended
"We believe Agios entered 2015 in a strong financial position to support several significant upcoming milestones, including multiple early and late stage clinical trials for our three investigational medicines," said
FINANCIAL GUIDANCE FOR THE FULL YEAR 2015
Agios announced today that it expects to end 2015 with more than
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at
About Agios/Celgene Collaboration
AG-221 and AG-120 are part of Agios' global strategic collaboration with
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 Agios' expectations and beliefs about: the potential of IDH1/IDH2 and pyruvate kinase-R mutations as therapeutic targets; the potential benefits of Agios' drug candidates targeting IDH1/IDH2 or pyruvate kinase-R mutations, including
|AGIOS PHARMACEUTICALS, INC.|
|Consolidated Balance Sheet Data|
|Cash, cash equivalents and marketable securities||$ 467,447||$ 193,894|
|Collaboration receivable – related party||6,492||476|
|Deferred revenue – related party||38,411||57,639|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
For the Three Months Ended
For the Years
|Collaboration revenue – related party||$ 14,636||$ 6,744||$ 65,358||$ 25,548|
|Research and development||34,863||15,279||100,371||54,502|
|General and administrative||6,500||3,707||19,120||9,929|
|Total operating expenses||41,363||18,986||119,491||64,431|
|Loss from operations||(26,727)||(12,242)||(54,133)||(38,883)|
|Loss before provision for income taxes||(26,642)||(12,213)||(53,930)||(38,828)|
|Provision (benefit) for income taxes||22||169||(426)||579|
|Cumulative preferred stock dividends||—||—||—||(4,162)|
|Net loss applicable to common stockholders||$ (26,664)||$ (12,382)||$ (53,504)||$ (43,569)|
|Net loss per share applicable to common stockholders – basic and diluted||$ (0.76)||$ (0.40)||$ (1.59)||$ (2.83)|
|Weighted-average number of common shares used in net loss per share applicable to common stockholders – basic and diluted||35,121,705||31,153,340||33,667,024||15,415,373|
Agios Pharmaceuticals: Lora Pike617-649-8608 Senior Director, Investor Relations and Public Relations Lora.Pike@agios.com