Agios Pharmaceuticals Inc (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic disorders of metabolism, reported financial results and business highlights for the first quarter ended March 31, 2015 and announced late stage clinical development plans for AG-221 and AG-120.

“Leveraging the foundation we built in 2014 and our unique scientific approach, the expansion of the AG-221 and AG-120 trials in hematologic malignancies positions Agios as a late-stage clinical development company,” said David Schenkein, M.D., chief executive officer of Agios. “We are on track to achieve our remaining milestones for 2015, which include several important initiations: a global Phase 3 study for AG-221 in previously treated AML patients with an IDH2 mutation, clinical development for our third IDH mutant inhibitor, AG-881, and the first study evaluating AG-348 in patients with PK deficiency. We also plan to share the first data from AG-120 in solid tumor patients with an IDH1 mutation in the second half of the year. Our financial position remains strong to support all of these efforts.”

AG-221 AND AG-120 CLINICAL DEVELOPMENT UPDATES

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

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

Added Additional Cohort to AG-221’s Ongoing Phase 1 Expansion Study in Hematologic Malignancies

  • AG-221 is currently being evaluated in an ongoing Phase 1 trial that includes a dose escalation phase and four expansion cohorts of 25 patients each evaluating patients with relapsed or refractory AML who are 60 years of age and older and transplant ineligible, relapsed or refractory AML patients under age 60, untreated AML patients who decline standard of care chemotherapy, and patients with other IDH2-mutant positive hematologic malignancies. Based on encouraging data from the dose escalation phase, Celgene and Agios have expanded the Phase 1 trial to add an additional more homogenous cohort of 125 patients with IDH2 mutant-positive AML who are in second or later relapse, refractory to second-line induction or reinduction treatment, or have relapsed after allogeneic transplantation. Consistent with the ongoing expansion cohorts, AG-221 will be administered at a dose of 100 mg once daily. This multicenter, single-arm, open-label, expanded clinical trial will be conducted at the current treatment centers in the U.S. and France. The primary objectives of the study are to confirm the safety and clinical activity of AG-221 in a select, highly resistant AML population.
  • Agios also continues to conduct a Phase 1/2 clinical trial evaluating AG-221 in IDH2-mutant positive advanced solid tumors, including gliomas, as well as angioimmunoblastic T-cell lymphoma (AITL).

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

Initiated Three Expansion Cohorts in Ongoing Phase 1 Study in Hematologic Malignancies

  • AG-120 is currently being evaluated in an ongoing Phase 1 trial in patients with advanced hematologic malignancies. Three expansion cohorts have been added to this study and will evaluate AG-120 in 175 patients with IDH1 mutated advanced hematologic malignancies at approximately 15 clinical trial sites in the U.S. and France. The first cohort will evaluate a more homogenous population of 125 AML patients who relapsed after bone marrow transplantation, are in second or later relapse, refractory to second line induction or reinduction treatment. The second cohort will evaluate 25 untreated AML patients, and the third cohort will evaluate 25 patients with IDH1 mutated advanced hematologic malignancies not eligible for cohorts one or two. AG-120 will be administered at a 500 mg once daily oral dose, in 28-day cycles. The study’s primary objectives are to confirm the safety and clinical activity of AG-120.
  • Agios also continues to advance a Phase 1 clinical trial evaluating AG-120 in patients with IDH1-mutant positive advanced solid tumors, including glioma.

“Our novel IDH inhibitors are advancing at an important time for cancer patients, as many do not respond to current standard of care chemotherapy,” said Chris Bowden, M.D., chief medical officer of Agios. “By demonstrating the safety and efficacy of AG-120 and AG-221 in later-stage studies in more highly defined patient populations, we hope to change the treatment paradigm and establish the utility of these IDH inhibitors as potential important new therapies for a significant portion of people diagnosed with AML worldwide.”

ADDITIONAL CLINICAL DEVELOPMENT AND BUSINESS HIGHLIGHTS

Cancer Metabolism: IDH Mutant Inhibitors in Collaboration with Celgene

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

  • Celgene exercised its option to obtain an exclusive license outside the United States for AG-120 in the first quarter.

AG-881: a brain-penetrant, pan-IDH mutant inhibitor

  • In April, Agios announced that it selected its third IDH mutant inhibitor, AG-881, for clinic development and entered into a new joint worldwide development and profit share collaboration with Celgene for this investigational medicine.

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

  • In March, AG-348 was granted orphan drug designation for the treatment of PK deficiency by the U.S. Food and Drug Administration (FDA).
  • A natural history study of PK deficiency is also ongoing. 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.

UPCOMING MILESTONES

AG-221 Clinical development milestones in collaboration with Celgene

  • Present first data from Phase 1 hematological malignancy expansion cohorts at EHA in Vienna, June 11-14, 2015.
  • Initiate combination trials to evaluate AG-221 as a potential frontline treatment for patients with AML and a broad range of hematologic malignancies in the second half of 2015.
  • Initiate a global Phase 3 registration-enabling study in relapsed/refractory AML patients that harbor an IDH2 mutation in the second half of 2015.
  • Continue dose escalation in the Phase 1/2 trial in patients with advanced solid tumors or AITL that carry an IDH2 mutation in 2015.

AG-120 Clinical development milestones in collaboration with Celgene

  • Present new data from the ongoing Phase 1 study evaluating patients with IDH1 mutant positive advanced hematologic malignancies at EHA.
  • Present first data from the Phase 1 trial in advanced solid tumors at a medical conference in the second half of 2015.
  • 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.
  • Intend to initiate a global registration-enabling Phase 3 study in AML patients that harbor an IDH1 mutation in the first half of 2016.

AG-881: Clinical development milestones in collaboration with Celgene

  • Initiate Phase 1 clinical development of AG-881 in the second quarter of 2015.

AG-348: Clinical development milestones for wholly owned PKR activator

  • Present final data for the Phase 1 multiple ascending dose (MAD) clinical trial of AG-348 in healthy volunteers and the first data from a natural history study of PK deficiency, a rare hemolytic anemia at EHA.
  • Two abstracts submitted by Boston Children’s Hospital on the natural history study have also been accepted for presentation at EHA.
  • Expect to initiate a Phase 2 trial for AG-348 in the first half of 2015 in patients with PK deficiency.

FIRST QUARTER 2015 FINANCIAL RESULTS

Cash, cash equivalents and marketable securities as of March 31, 2015 were $440.0 million, compared to $467.4 million as of December 31, 2014. The decrease was driven by cash used to fund operating activities of approximately$32.2 million, which was offset by cost reimbursements of approximately $6.6 million made by Celgene during the first quarter of 2015 from the AG-221 program.

Total revenue was $34.2 million for the first quarter of 2015, compared to $8.4 million for the comparable period in 2014. The increase reflects revenues recognized under the company’s collaboration agreement with Celgene.

Research and development (R&D) expense was $32.4 million, including $2.6 million of stock- based compensation expense in the first quarter of 2015, compared to $17.4 million, including $1.1 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 $7.0 million, including $2.4 million of stock-based compensation expense, in the first quarter of 2015, compared to $3.3 million, including $0.4 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 first quarter of 2015 was $5.0 million, compared to net loss of $12.2 million for the comparable period in 2014.

FINANCIAL GUIDANCE FOR THE FULL YEAR 2015

Agios is reiterating that it expects to end 2015 with more than $320.0 million of cash, cash equivalents and marketable securities. The anticipated year end 2015 cash position includes two one-time payments from Celgene: a $20.0 millionresearch extension fee, received in May 2015, and a $10.0 million initial payment for the AG-881 collaboration, expected to be received in the second quarter of 2015, and does not include any additional program-specific milestone payments. The company expects that its cash, cash equivalents and marketable securities would be sufficient to fund its operating expenses and capital expenditure requirements until late 2017. (Original Source)

Shares of Agios closed yesterday at $96.14 . AGIO has a 1-year high of $138.85 and a 1-year low of $31.51. The stock’s 50-day moving average is $99.76 and its 200-day moving average is $103.79.

On the ratings front, Agios has been the subject of a number of recent research reports. In a report issued on April 30, Canaccord Genuity analyst John Newman downgraded AGIO to Hold, with a price target of $103, which implies an upside of 7.1% from current levels. Separately, on April 20, Oppenheimer’s Ling Wang initiated coverage with a Hold rating on the stock .

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, John Newman and Ling Wang have a total average return of 6.7% and -6.6% respectively. Newman has a success rate of 46.7% and is ranked #845 out of 3594 analysts, while Wang has a success rate of 26.5% and is ranked #3365.

In total, 4 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 $96.14 which is 24.6% 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.