Agios Pharmaceuticals Reports Third Quarter 2013 Financial Results
Successfully Completed Initial Public Offering and Advanced IDH2 Cancer Metabolism Program into Clinical Development
“Agios has made important progress this year toward realizing our
long-term vision of developing transformational medicines and building a
world-class biopharmaceutical company,” said
Recent Business Highlights
Completed initial public offering. Agios announced on
July 29, 2013that it had completed an initial public offering (IPO) of common stock, raising net proceeds of $111.0 million. In addition, Celgenepurchased $12.8 millionof Agios common stock in a separate private placement concurrent with the completion of the IPO.
- Initiated Phase 1 study of AG-221, an IDH2 mutant inhibitor for the treatment of cancer in patients with an IDH2 mutation. In September, Agios announced that the first patient was dosed in a Phase 1 study of AG-221 in advanced hematologic malignancies with an IDH2 mutation. This Phase 1, multi-center study is evaluating the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-221 in patients with advanced hematologic malignancies that harbor an IDH2 mutation. The first stage of the study is a dose-escalation phase in which cohorts of patients will receive ascending oral doses of AG-221 to determine the maximum tolerated dose and/or the recommended Phase 2 dose. At completion of the dose escalation phase, several expansion cohorts of patients will receive AG-221 to further evaluate the safety, tolerability and clinical activity of the maximum tolerated dose. Currently, four clinical trial sites are open and recruiting patients. AG-221 is an orally available, selective, potent inhibitor of the mutated IDH2 protein, making it a highly targeted therapeutic candidate for the treatment of patients with cancers with an IDH2 mutation.
Advanced AG-120, an IDH1 mutant inhibitor for the treatment of cancer in patients with an IDH1 mutation, toward Investigational New Drug (IND) filing. Agios has substantially completed IND-enabling studies of AG-120 and remains on track for initiating clinical trials in early 2014. AG-120 is an orally available, selective, potent inhibitor of the mutated IDH1 protein, and a highly targeted therapeutic candidate for the treatment of patients with cancers that harbor an IDH1 mutation. Phase 1 trials are being planned for patients with advanced solid and hematological malignancies that carry an IDH1 mutation.
Advanced AG-348, an activator of pyruvate kinase R (PKR) for the treatment of patients with pyruvate kinase deficiency (PK deficiency), into IND-enabling studies. Agios has initiated IND-enabling preclinical studies of AG-348, an orally available, potent small molecule activator of the PKR enzyme, which, when mutated, leads to PK deficiency, a form of hereditary hemolytic anemia. Pre-clinical in vitro data demonstrate that these activators can significantly enhance the activity of most of the common PKR mutations, making it a potential treatment for patients with PK deficiency.
Strengthened board of directors. In September, Agios announced
the addition of
Paul J. Clancy, executive vice president and chief financial officer at Biogen Idec, to its board of directors.
Agios expects to present preclinical data for each of its lead
programs at the annual
American Society for Hematology(ASH) meeting in December 2013.
- In early 2014, Agios anticipates submitting an IND and initiating Phase 1 clinical trials for AG-120 in patients with advanced solid and hematological malignancies that carry an IDH1 mutation.
- Agios anticipates submitting an IND and initiating Phase 1 clinical trials for AG-348 in 2014, including normal healthy volunteers and patients with PK deficiency.
Third Quarter 2013 Financial Results & Financial Guidance
Cash Position: Cash, cash equivalents and marketable securities
September 30, 2013were $208.4 million, compared to $128.0 millionas of December 31, 2012. The increase was primarily driven by net proceeds of $111.0 millionfrom Agios’ IPO and $12.8 millionfrom the concurrent private placement with Celgene, offset by cash used to fund operations.
Revenues: Collaboration revenue was
$6.3 millionin each of the third quarters of 2013 and 2012 and $18.8 millionin each of the nine months ended September 30, 2013and 2012. Collaboration revenue is primarily comprised of deferred revenue from payments received in previous periods from Agios’ collaboration agreement with Celgene.
R&D Expenses: Research and development expenses were
$14.8 millionin the third quarter of 2013 and $39.2 millionin the nine months ended September 30, 2013, compared to $9.8 millionand $29.8 millionin the comparable periods in 2012. The increase in R&D expense was largely due to increased spending on clinical activities as AG-221 entered Phase 1 development in September 2013, in addition to IND-enabling activities for the company’s AG-120 and AG-348 programs.
G&A Expenses: General and administrative expenses were
$2.5 millionin the third quarter of 2013 and $6.2 millionin the nine months ended September 30, 2013, compared to $1.6 millionand $5.5 millionin the comparable periods in 2012. The increase in G&A expenses was largely due to incremental expenses to support public company operations.
Net Loss: Net loss was
$11.2 millionfor the third quarter of 2013 and $27.0 millionfor the nine months ended September 30, 2013, compared to net loss of $4.6 millionand $14.8 millionfor the comparable periods in 2012.
- Financial Guidance: Agios expects that its cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses and capital expenditure requirements until at least the fourth quarter of 2016.
Conference Call Information
Agios will host a conference call and live audio webcast today at
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the
meaning of The Private Securities Litigation Reform Act of 1995. Such
forward-looking statements include those regarding Agios’ expectations
and beliefs about: the potential of IDH1/IDH2 and pyruvate kinase R
mutations as therapeutic targets; the potential benefits of Agios’
product candidates targeting IDH1/IDH2 or pyruvate kinase R mutations,
|AGIOS PHARMACEUTICALS, INC.|
|CONSOLIDATED BALANCE SHEET DATA (UNAUDITED)|
|(Amounts in thousands)|
|September 30,||December 31,|
|Cash, cash equivalents, and marketable securities||$||208,368||$||127,976|
|Stockholders' equity (deficit)||142,595||(72,024||)|
|AGIOS PHARMACEUTICALS, INC.|
|CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED)|
|(Amounts in thousands except per share data)|
Three Months Ended
Nine Months Ended
|Research and development||14,803||9,798||39,223||29,812|
|General and administrative||2,534||1,580||6,222||5,510|
|Total operating expenses||17,337||11,378||45,445||35,322|
|Loss from operations||(11,069||)||(5,110||)||(26,641||)||(16,498||)|
|Provision (benefit) for income taxes||121||(452||)||410||(1,648||)|
|Cumulative preferred stock dividends||567||1,798||4,162||5,393|
|Net loss applicable to common stockholders||($10,610||)||($2,847||)||($22,863||)||($9,396||)|
|Net loss per share applicable to common stockholders:|
|Basic and diluted||($0.47||)||($0.83||)||($2.26||)||($2.81||)|
|Weighted average shares outstanding:|
|Basic and diluted||22,744||3,439||10,112||3,348|