Agios Pharmaceuticals Reports Third Quarter 2014 Financial Results
"2014 is an important year for the company, as we make progress on key clinical objectives for all three of our investigational medicines:
RECENT BUSINESS HIGHLIGHTS AND POTENTIAL MILESTONES
Cancer Metabolism: IDH Mutant Inhibitors in Collaboration with
AG-221: a first-in-class, oral, selective, potent inhibitor of the mutated IDH2 protein
An oral presentation of AG-221 in advanced hematologic malignancies will be presented at the
American Society of Hematology(ASH) Annual Meeting and Exhibition in San Francisco. The presentation, "AG-221, an Oral, Selective, First-in-Class, Potent Inhibitor of the IDH2 Mutant Metabolic Enzyme, Induces Durable Remissions in a Phase I Study in Patients with IDH2 Mutation Positive Advanced Hematologic Malignancies," will be presented by Eytan Stein, M.D., lead investigator and physician in the leukemia service at Memorial Sloan-Kettering Cancer Centeron Sunday, December 7, 2014at 4:30 p.m. PST( 7:30 p.m. EST). Also at ASH, two abstracts related to AG-221 were accepted for poster presentations. The accepted abstracts are available online on the ASH conference website: ash.confex.com/ash/2014/webprogram/start.html.
Agios plans to host an investor event in
San Franciscoon Monday, December 8, 2014beginning at 12:00 p.m. PST( 3:00 p.m. EST) to discuss AG-221 and the preclinical and clinical data presented at ASH. The event will be webcast live and can be accessed under "Events & Presentations" in the Investors & Media section of the company's website at www.agios.com.
- Agios continues to conduct the Phase 1 study in IDH2-mutant positive advanced hematologic malignancies with the primary goals of establishing the safety profile, determining the maximum tolerated dose (MTD), and assessing the clinical activity of AG-221. In October, the company initiated four expansion cohorts. The Phase 1 expansion cohorts will assess the safety and tolerability of AG-221 at 100 mg once daily in approximately 100 patients with IDH2-mutant hematologic malignancies, including acute myelogenous leukemia (AML).
In August, the
U.S. Food and Drug Administration( FDA) granted Agios Fast Track Designation for AG-221 for the treatment of patients with AML that harbor an IDH2 mutation. Fast Track is granted to facilitate frequent interactions with the FDAin an effort to expedite clinical development and submission of a new drug application for medicines with the potential to treat serious conditions.
- In October, Agios initiated a Phase 1/2 trial of AG-221 in patients with advanced solid tumors as the next step in evaluating AG-221's potential in a broad range of cancers that harbor the IDH2 mutation.
AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein
The first data from the ongoing Phase 1 trial of AG-120 in advanced IDH1-mutant positive hematologic malignancies will be presented in a late-breaking oral presentation at the 26th Symposium on Molecular Targets and Cancer Therapeutics hosted by the
European Organization for Research and Treatment of Cancer(EORTC), the National Cancer Instituteand the American Association for Cancer Research(EORTC-NCI-AACR), taking place in mid-November in Barcelona, Spain. The presentation will be given by Daniel Pollyea, M.D., lead investigator and physician of leukemia services at the University of Colorado School of Medicineon Wednesday, November 19, 2014, at 2:50 p.m. CET( 8:50 a.m. EST). In accordance with the symposium's embargo policy, these data remain under embargo until the day of the presentation on Wednesday, November 19at 12:01 a.m. CET( Tuesday, November 18at 6:01 p.m. EST).
Agios plans to host a conference call and webcast on
Wednesday, November 19at 4:00 p.m. CET( 10:00 a.m. EST) to review the Phase 1 clinical data for AG-120 being presented at the symposium. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 31391928. A replay of the call will be available approximately two hours after the conclusion of the call. The webcast can be accessed live or in archived form under "Events & Presentations" in the Investors & Media section of the company's website at www.agios.com.
- Agios continues to advance two separate Phase 1 clinical trials evaluating AG-120 in patients with IDH1-mutant advanced hematologic malignancies and IDH1-mutant advanced solid tumors. 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. The company expects to provide an update on the Phase 1 study evaluating patients with advanced solid tumors with an IDH1 mutation at a medical conference in 2015.
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
- Agios researchers plan to present the first clinical data from healthy volunteer studies with AG-348 in a poster presentation at the ASH Annual Meeting in December. Key results to be presented during the meeting include the first data on safety, pharmacokinetics and pharmacodynamics from the completed single ascending dose (SAD) and multiple ascending dose escalation (MAD) studies in healthy volunteers.
The company plans to review data presented for AG-348 at ASH in conjunction with the planned investor event on
December 8, 2014referenced above for AG-221.
- Agios recently completed enrollment in the ongoing Phase 1 MAD trial in healthy volunteers. Based on meeting the primary endpoints in the Phase 1 healthy volunteer studies, the company believes it will be in a position to initiate a Phase 2 clinical trial for AG-348 in early 2015 in patients with PK deficiency.
- A natural history study of PK deficiency is also ongoing and patient enrollment is on track. 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 expects to report initial data from its study of the natural history of PK deficiency at a medical conference in 2015.
THIRD QUARTER 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 income for the third quarter of 2014 was
"Agios continues to maintain a strong balance sheet ending the quarter with a cash position of
FINANCIAL GUIDANCE FOR THE FULL YEAR 2014
Agios is updating today its 2014 cash guidance, and expects to end the year with more than
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at
About AG-221 and IDH2
IDH1 and IDH2 are metabolic enzymes that are mutated in a wide range of hematologic and solid tumor malignancies, including AML. Normally, IDH enzymes help to break down nutrients and generate energy for cells. When mutated, IDH creates a molecule that alters the cells' genetic programming, and instead of maturing, the cells remain primitive and proliferate quickly. Agios believes that inhibition of these mutated proteins may lead to clinical benefit for the subset of cancer patients whose tumors carry them. AG-221 was developed by Agios as a selective, potent inhibitor of the mutated IDH2 protein. While most cancer treatments attempt to destroy the cancerous cells, AG-221 uniquely attacks its target – mutated IDH2 – and aids the maturation of the cells into functioning blood cells. AG-221 has received Orphan Drug Designation for AML and Fast Track Designation for patients with IDH2-mutant AML.
About Agios/Celgene Collaboration
AG-221 is a part of Agios' global strategic collaboration with
About AG-348 and Pyruvate Kinase (PK) Deficiency, a Rare, Inherited Hemolytic Anemia
Pyruvate kinase (PK) deficiency, a rare, inherited hemolytic anemia affecting children and adults, is caused by mutations that affect the activity of the metabolic enzyme pyruvate kinase-R (PKR), the form of pyruvate kinase that is present in red blood cells. The current standard of care for PK deficiency is supportive, including blood transfusions, splenectomy, chelation therapy to address iron overload and/or interventions for other treatment- and disease-related morbidities. Currently, there is no approved therapy to treat the underlying cause of PK deficiency. AG-348 is a first-in-class orally available, potent, selective small molecule activator of PKR, which, when mutated, leads to PK deficiency. AG-348 was discovered in the laboratory of Agios, and the company retains worldwide development and commercialization rights.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements, including 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|
|September 30,||December 31,|
|Cash, cash equivalents and marketable securities||$ 237,887||$ 193,894|
|Collaboration receivable – related party||18,759||476|
|Deferred revenue – related party||45,199||57,639|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Gross collaboration revenue – related party||$33,900||$6,268||$50,722||$18,804|
|Research and development||25,526||14,803||65,509||39,223|
|General and administrative||5,166||2,534||12,619||6,222|
|Total operating expenses||30,692||17,337||78,128||45,445|
|Income (loss) from operations||3,208||(11,069)||(27,406)||(26,641)|
|Income (loss) before provision for income taxes||3,256||(11,056)||(27,288)||(26,615)|
|(Benefit) provision for income taxes||(448)||121||(448)||410|
|Net income (loss)||3,704||(11,177)||(26,840)||(27,025)|
|Cumulative preferred stock dividends||—||(567)||—||(4,162)|
|Net income (loss) applicable to common stockholders||$3,704||$(11,744)||$(26,840)||$(31,187)|
|Net income (loss) per share applicable to common stockholders – basic||$0.11||$(0.52)||$(0.81)||$(3.08)|
|Net income (loss) per share applicable to common stockholders – diluted||$0.10||$(0.52)||$(0.81)||$(3.08)|
|Weighted-average number of common shares used in net income (loss) per share applicable to common stockholders – basic||34,495,076||22,744,486||33,176,801||10,111,735|
|Weighted-average number of common shares used in net income (loss) per share applicable to common stockholders – diluted||36,592,683||22,744,486||33,176,801||10,111,735|
Agios Pharmaceuticals: Lora Pike617-649-8608 Senior Director, Investor Relations and Public Relations Lora.Pike@agios.com