Agios Pharmaceuticals Inc (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, today reported business highlights and financial results for the second quarter ended June 30, 2015.

“In the first half of 2015, we continued to drive rapid and meaningful progress across our development programs, as well as selecting our fourth molecule for development, AG-881, and advancing it into two Phase 1 studies,” said David Schenkein, M.D., chief executive officer of Agios. “Additionally, we recently initiated DRIVE PK, a Phase 2 study of AG-348 in PK deficiency patients, and are on track to initiate two global, registration-enabling Phase 3 studies of AG-221 and AG-120 in patients with acute myeloid leukemia in the next six to 12 months. These efforts bring us closer to our goal of being a late-stage biopharmaceutical company capable of changing the treatment paradigm for people with cancer and rare genetic disorders.”

KEY UPCOMING MILESTONES

Agios anticipates the following milestones from its IDH clinical development programs in collaboration with Celgene:

AG-221: a first-in-class, oral, selective, potent inhibitor of the mutated IDH2 protein

  • Initiate a global Phase 3 registration-enabling study in relapsed/refractory acute myeloid leukemia (AML) patients who harbor an IDH2 mutation by the end of 2015.
  • Initiate combination trials to evaluate AG-221 as a potential frontline treatment for patients with AML who harbor an IDH2 mutation by the end of 2015.

AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein

  • Begin combination trials to evaluate AG-120 as a potential frontline treatment for AML patients who harbor an IDH1 mutation by the end of 2015.
  • Initiate a global registration-enabling Phase 3 study in AML patients who harbor an IDH1 mutation in the first half of 2016.
  • Present data on the ongoing Phase 1 study of AG-120 in IDH1 mutant-positive advanced solid tumors at a medical meeting in the fourth quarter of 2015.

RECENT DEVELOPMENT UPDATES IN CANCER METABOLISM

Agios has provided the following updates on its clinical development programs in collaboration with Celgene:

AG-221

  • New data from the dose-escalation phase and expansion cohorts from the ongoing Phase 1 study evaluating single agent AG-221 were presented in June at the 20th Congress of the European Hematology Association (EHA). Read the full AG-221 data here.
  • As of July, Agios completed the dose-escalation portion of the Phase 1 study of AG-221 in IDH2 mutant-positive hematologic malignances. The expansion cohorts are on track, including the fifth expansion cohort of 125 patients with IDH2 mutant-positive AML who are in second or later relapse, refractory to second-line induction or re-induction treatment, or have relapsed after allogeneic transplantation.
  • Dose escalation continues in the Phase 1/2 trial in patients with advanced solid tumors and angioimmunoblastic T-cell lymphoma (AITL) who carry an IDH2 mutation.

AG-120

  • New data from the dose-escalation phase of the ongoing Phase 1 study evaluating single agent AG-120 in advanced hematologic malignancies were presented in June at EHA. Read the full AG-120 data here.
  • Also announced in June, the U.S. Food and Drug Administration (FDA) granted Agios orphan drug designation for AG-120 for the treatment of patients with IDH1 mutant-positive AML.
  • In May, Agios announced that the FDA granted Fast Track designation to AG-120 for the treatment of patients with IDH1 mutant-positive AML.
  • As of July, Agios completed the dose-escalation portion of the Phase 1 study of AG-120 in IDH1 mutant-positive hematologic malignances. The three expansion cohorts to evaluate AG-120 in 175 patients with IDH1-mutated advanced hematologic malignancies, including one cohort with 125 patients with relapsed and/or refractory AML, are on track.

AG-881: a brain-penetrant, first-in-class, oral, potent pan-inhibitor of the mutated IDH1 and IDH2 proteins

  • In June, Agios announced that the first patient was dosed in a Phase 1, open-label, dose-escalation and expansion study of single agent AG-881 in patients with IDH mutant-positive advanced solid tumors, including gliomas.
  • In August, the first patient was dosed in a second dose-escalating and expansion trial for patients with IDH mutant-positive advanced hematologic malignancies, whose cancer has progressed on a prior IDH inhibitor therapy.

RECENT DEVELOPMENT UPDATES IN RARE GENETIC METABOLIC DISORDERS

AG-348: a novel, first-in-class, oral activator of pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency

  • In June, final data from the Phase 1 multiple-ascending dose (MAD) study in healthy volunteers were presented at EHA, establishing clear proof-of-mechanism for AG-348. Read the full AG-348 data, as well as data from Boston Children’s natural history study, here.
  • Also in June, Agios initiated DRIVE PK, a global Phase 2, open-label safety and efficacy trial in adult, transfusion-independent patients with PK deficiency. The first patient was dosed in July.

CORPORATE UPDATE

Agios plans to host and webcast an investor event in October 2015 in an effort to provide background and education on the novel programs it is targeting and the diseases the company aims to treat. Additional details will be provided in the coming weeks.

SECOND QUARTER 2015 FINANCIAL RESULTS

Cash, cash equivalents and marketable securities as of June 30, 2015 were $434.0 million, compared to $467.4 millionas of December 31, 2014. The decrease was driven by cash expenditures for operating activities of approximately $64.9 million, which was offset by cash received from Celgene of approximately $43.3 million during the six months endedJune 30, 2015 related to our collaboration agreements.

Collaboration revenue was $13.2 million for the second quarter of 2015, compared to $8.4 million for the comparable period in 2014. The increase reflects revenues recognized under the company’s collaboration agreements withCelgene, including $8.8 million related to the delivery of the U.S. and ex-U.S. licenses for AG-881.

Research and development (R&D) expense was $36.4 million, including $4.6 million of stock- based compensation expense in the second quarter of 2015, compared to $22.6 million, including $1.4 million in stock-based compensation expense for the comparable period in 2014. The increase in R&D expense was primarily due to increased costs to support advancement of the company’s lead investigational medicines toward later-stage development.

General and administrative (G&A) expense was $8.9 million, including $3.6 million of stock-based compensation expense, in the second quarter of 2015, compared to $4.2 million, including $1.0 million of stock-based compensation expense, for the comparable period in 2014. The increase in G&A expense was largely due to increased headcount and other professional expenses to support growing operations.

Net loss for the second quarter of 2015 was $31.9 million, compared to net loss of $18.3 million for the comparable period in 2014. (Original Source)

Shares of Agios Pharmaceuticals closed yesterday at $111.85. AGIO has a 1-year high of $138.85 and a 1-year low of $36.44. The stock’s 50-day moving average is $109.70 and its 200-day moving average is $109.15.

On the ratings front, Agios Pharmaceuticals has been the subject of a number of recent research reports. In a report issued on June 16, MLV & Co. analyst Arlinda Lee reiterated a Buy rating on AGIO, with a price target of $130, which represents a potential upside of 16.2% from where the stock is currently trading. Separately, on June 15, Canaccord Genuity’s John Newman reiterated a Hold rating on the stock and has a price target of $93.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Arlinda Lee and John Newman have a total average return of 6.5% and 16.8% respectively. Lee has a success rate of 52.3% and is ranked #991 out of 3727 analysts, while Newman has a success rate of 60.3% and is ranked #131.

Overall, 3 research analysts have assigned a Hold rating and one research analyst has given a a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $115.67 which is 3.4% above where the stock closed yesterday.

Agios Pharmaceuticals Inc is engaged in the development of medicines to treat cancer metabolism and inborn errors of metabolism, which are a subset of orphan genetic metabolic diseases.