Analyst Conference Summary

biotechnology

conference date: August 4, 2016 @ 5:30 AM Pacific Time
for quarter ending: June 30, 2016 (Q2, second quarter 2016)

Forward-looking statements

Overview: Pipeline therapies continued to progress. Although GAAP revenue was minimal, received $200 million upfront collaboration payment from Celgene.

Basic data (GAAP):

Revenue was $7.0 million, down sequentially from $31.3 million, and down from $13.2 million year-earlier. All revenue is from collaborations.

Net loss was $56.0 million, sequentially from $23.2 million, and down from a $32.0 million loss year-earlier.

EPS (diluted GAAP) was negative $1.47, up sequentially from negative $0.61, and down from negative $0.85 year-earlier.

Guidance:

none

Conference Highlights:

David Schenkein, M.D., CEO at Agios said "At EHA, we established clear proof-of-concept for AG-348 in pyruvate kinase deficiency, validating our novel approach to treat rare genetic metabolic disorders by correcting the underlying enzymatic defect and potentially establishing the first treatment for patients with this serious disease. This milestone sets the stage for pivotal development of our PKR activator program. In addition, our new strategic collaboration with Celgene enables Agios to expand into the emerging field of metabolic immuno-oncology, an important new field of cancer research."

The new strategic collaboration with Celgene announced in May for immuno-oncology resulted in a $200 million upfront payment. Ex-U.S. rights of AG-120 were transferred to Agios.

Will initiate a global, registration-enabling Phase 3 study of AG-120 in frontline AML patients with an IDH1 mutation in the second half of 2016. In the Phase 1 expansion study for relapsed/refractory AML,

Will initiate an expansion arm in high-risk myelodysplastic syndrome patients for AG-221 in 2016.

A Phase 1/2 frontline combination study of AG-221 or AG-120 with Vidaza (azacitidine) in newly diagnosed AML patients not eligible for intensive chemotherapy continued.

Continued to enroll patients in the following ongoing clinical trials: Phase 3 IDHENTIFY study of AG-221 vs. standard of care chemotherapy in R/R AML; Phase 1b frontline combination study of AG-221 or AG-120 with standard-of-care intensive chemotherapy in AML; Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive hematologic malignancies. In the quarter received orphan drug designation in Europe for AML. "Single agent activity for our investigational medicine is compelling."

Presenting data from the expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive low grade glioma in the second half of 2016.

Initiating a randomized Phase 2 study of AG-120 in IDH1 mutant positive cholangiocarcinoma in the second half of 2016

Presented data from DRIVE PK, a global Phase 2, open-label safety and efficacy trial of AG-348 in adult, transfusion-independent patients with pyruvate kinase (PK) deficiency, at the EHA in June. AG-348 achieved proof-of-concept with rapid and sustained hemoglobin increases. Also showed a good safety profile. Enrollment continues. Will provide a regulatory strategy by the end of the year.

Presented the first data from the Phase 1 study of AG-519 for PK deficiency in healthy volunteers for presentation at EHA in 2016. One of 54 patients developed grade 2 thrombocytopenia. Demonstrated proof of mechanism. Enrollment continues.

Agios will outline the clinical development plans for Agios PKR activators in beta-thalassemia in the second half of 2016.

Preclinical activities for MTAP (methylthioadenosine phosphorylase) deleted cancers will begin in 2016.

Will present the new findings from the Natural History Study of PK deficiency being conducted with Boston Children’s Hospital in the second half of 2016.

Will initiate preclinical development activities for the first molecule in the next wave of novel investigational cancer metabolism medicines.

GAAP operating expenses were $63.4 million, consisting of: $50.8 million for R&D and $12.6 million for G&A. Interest income was $0.5 million.

Cash (including equivalents & securities) ended at $512.3 million, up sequentially from $356 million largely due to a $200 million payment from Celgene, less operating expenses. No debt.

All development costs for AG-221 are paid by Celgene, and 50% for AG-881.

R&D expense was $50.8 million. G&A expense $12.6 million. Loss from operations $56.5 million. Interest income $0.5 million.

Q&A:

AG-519 number of patients treated? Total is 54. 30 in multiple ascending dose component. The trial is ongoing to understand PK and PD relationships to doses.

AG-519 thrombocytopenia? The more patients we dose without another incident, the more we can narrow the bracket for the incidence rate.

AG-221 cohort expansion study? This is a very important data set. We have to wait for it to mature before presenting at a meeting. We are pleased with the data and enrollment to date.

Non-missense mutations? That is about 20% of the population. There are also combined missense and non-missense patients. We are not limiting patient enrollment by genotype. We want to learn what we can at this point. There are already 200 mutations that have been characterized.

It is highly likely that we will pick just one molecule for PK deficiency diseases.

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