Agios Pharmaceuticals Inc (NASDAQ:AGIO) announced its clinical development strategy for the company’s lead cancer metabolism and rare genetic metabolic disorders programs, along with insights into emerging research at its R&D Day today.

“With our lead IDH programs progressing through Phase 1 expansion cohorts and the initiation of the Phase 3 AG-221 study with our collaboration partner Celgene, we are moving closer to our goal of providing people with advanced AML with transformational new medicines as quickly as possible,” said David Schenkein, M.D., chief executive officer at Agios. “We will share our long-term vision for these programs at today’s R&D Day, as we hope to one day provide benefit to every patient diagnosed with an IDH mutant-positive cancer.”

“At our core, Agios is a research-driven organization, and I’m proud that our scientists have discovered AG-519, a novel PK activator, which represents our fifth new investigational medicine in just seven years. We continue to conduct research that we believe will enable us to make important advances for patients,” Dr. Schenkein continued.

IDH Program Updates

In clinical studies to date, AG-221 and AG-120, which target mutated IDH2 and IDH1, respectively, have demonstrated positive clinical single-agent activity with durable complete and partial responses and manageable safety profiles in patients with AML. Together with our collaboration partner Celgene, Agios remains committed to bringing AG-221 and AG-120 to patients as quickly and efficiently as possible by leveraging a clinical development strategy that maximizes both speed and breadth.

Agios today announced clinical development updates for AG-221 and AG-120 in AML, including:

  • Initiation of the AG-221 Phase 3 Study: The IDHENTIFY study of AG-221 is a Phase 3, international, multi-center, open-label, randomized clinical trial designed to compare the efficacy and safety of AG-221 versus conventional care regimens in patients 60 years or older with IDH2 mutant-positive AML that is refractory to or relapsed after second- or third-line therapy. Additional details can be found below and on This study is being conducted by Celgene.
  • Novel Design of the AG-221 and AG-120 Frontline Trials in AML:
    • For Newly Diagnosed AML Patients Eligible for Intensive Chemotherapy: A Phase 1b combination study of either AG-221 or AG-120 with standard induction (7+3, Ara-C and idarubicin/daunorubicin) and consolidation (Ara-C, or mitoxantrone with etoposide) chemotherapy is planned for initiation by the end of 2015.
    • For Newly Diagnosed AML Patients Not Eligible for Intensive Chemotherapy: A Phase 1/2 combination study of either AG-221 or AG-120 with VIDAZA® (azacitidine) is planned for initiation in the first quarter of 2016. This study has a Phase 1 component to determine the safety of the combinations, followed by a Phase 2 randomized component evaluating the safety and clinical activity of each investigational combination versus single-agent VIDAZA® using a primary endpoint of overall response rate.
  • ASH Data Presentations: New data from the ongoing Phase 1 dose-escalation and expansion studies of AG-221 and AG-120 in advanced hematologic malignancies have been accepted for presentation at the American Society of Hematology (ASH) Annual Meeting and Exhibition taking place December 5-8, 2015 in Orlando.
  • EORTC-NCI-AACR Data Presentation: The first data from the ongoing Phase 1 trial of AG-120 in advanced IDH1-mutant positive solid tumors have been accepted for oral presentation at EORTC-NCI-AACR International Conference on Molecular Targets and Cancer Therapeutics taking place November 5-9, 2015 in Boston.

Pyruvate Kinase (PK) Deficiency Program Updates

Agios is pioneering the development of its small molecule enzyme activators in PK deficiency, a rare genetic metabolic disorder with no disease-altering therapies.

  • AG-348: AG-348, a first-in-class orally available, potent, selective small molecule activator of pyruvate kinase-R (PKR), is on track and enrolling in DRIVE PK, a global Phase 2, open-label safety and efficacy trial in adult, transfusion-independent patients with PK deficiency.
  • AG-519: Agios announced today the development of its second PKR activator, AG-519. This program provides clinical development optionality for our PK activator portfolio and potentially opportunities in other hemolytic anemias where PK activation may be therapeutic. The development plan for AG-519 includes a placebo-controlled Phase 1 study in healthy volunteers, which is planned for the first quarter of 2016. This study will be an integrated single ascending dose (SAD) and multiple ascending dose (MAD) trial.

Research Program Updates

Agios has advanced and led the emerging field of cancer metabolism with its novel IDH1 and IDH2 programs, demonstrating significant potential benefit for AML patients whose cancers carry these mutations. Agios’ work in IDH exemplifies the company’s strategy of targeting metabolic vulnerabilities. The company continues to discover novel metabolic targets that meet a high bar for future development. Today at its R&D day, Agios will describe:

  • Its precision medicine strategy to discover novel cancer metabolism targets.
  • Novel research approach to rare genetic diseases using allosteric modulation to correct the underlying dysfunction in metabolic pathways.
  • The potential for PKR activators to provide clinical benefit in other indications, such as beta-thalassemia.(Original Source)

Shares of Agios are trading down 3.63% to $73.64 Friday. AGIO has a 1-year high of $138.85 and a 1-year low of $63.02. The stock’s 50-day moving average is $82.35 and its 200-day moving average is $100.24.

On the ratings front, Canaccord Genuity analyst John Newman reiterated a Hold rating on AGIO, with a price target of $93, in a report released yesterday. The current price target implies an upside of 21.7% from current levels. According to, Newman has a total average return of -5.4%, a 33.9% success rate, and is ranked #3617 out of 3779 analysts.

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.