Analyst Conference Summary

biotechnology

conference date: May 4, 2017 @ 5:00 AM Pacific Time
for quarter ending: March 31, 2017 (Q1, first quarter 2017)

Forward-looking statements

Overview: Agios hopes to graduate from a clinical-stage to a commercial-stage company in 2017, with the FDA PDUFA date set at August 30, 2017.

Basic data (GAAP):

Revenue was $10.5 million, down sequentially from $22.6 million, and down from $31.3 million year-earlier. All revenue is from collaborations.

Net loss was $66.2 million, down sequentially from $56.5 million, and down from a $23.6 million loss year-earlier.

EPS (diluted GAAP) was negative $1.56, down sequentially from negative $1.34, and down from negative $0.61 year-earlier.

In addition the share count ended at 42.3 million, up from 37.9 million year-earlier.

Guidance:

Cash is sufficient to last until the end of 2019.

Conference Highlights:

David Schenkein, M.D., CEO at Agios said “We are on track to execute against our remaining key 2017 goals, including finalizing the design of our global pivotal program for AG-348 in PK deficiency in the third quarter and submitting both an NDA for ivosidenib in R/R AML and our sixth IND, for AG-270, a development candidate focused on MTAP-deleted tumors, by the end of the year.” The sales force is being assembled.

Idhifa (AG-221, Enasidenib) NDA was submitted in December to the FDA in IDH2 positive relapsed/refractory AML (acute myeloid leukemia) . FDA PDUFA date is August 30, 2017. This is in partnership with Celgene and is based on Phase 1/2 data. The Phase 3 trial continues. An MDS trial is also planned. Update Phase 1/2 IDH2m R/R AML data will be presented at ASCO.

AG-120 (ivosidenib) in frontline AML patients with an IDH1 mutation could be submitted to the FDA for approval by the end of 2017 based on Phase 1/2 data. Data was presented at ASH showing impressive activity. The R/R AML expansion cohort completed enrollment. A Phase 3 frontline AML study, AGILE, in combination with Vidaza, for patients with IDH1 mutation ineligible for intensive chemotherapy should start this quarter.

AG-120 for IDH1 mutant chondrosarcoma expansion phase, positive data for low-grade IDH1 mutant glioma data was presented on November 18. Updated data will be presented in the second half of 2017.

A randomized Phase 3 study (ClarlDHy) of AG-120 in IDH1 mutant positive cholangiocarcinoma in continued enrollment. Data from the Phase 1 expansion cohort to be presented in the first half of 2017. Fast track status was granted last week. This is a high-unmet-need disease.

Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive hematologic malignancies completed the dose-escalation phase. Another Phase 1 study for IDHm positive glioma should complete its dose escalation phase in the first half half of 2017. Also looking at ivosidenib as a possible treatment for this disease.

Phase 1 study of AG-519 for PK deficiency in healthy volunteers enrollment continues.

AG-348 data for Pyruvate Kinase Deficiency (PKD) hemoglobin levels was presented at ASH, showing compelling hemoglobin increases in about half of patients.The FDA granted Fast Track designation last week. A global pivotal trial is planned for the first half of 2018. Updated DRIVE PK study data is expected in first half of 2017 at EHA. Also the Boston Children's Hospital Natural History Study completed enrollment.

Preclinical activities for MTAP (methylthioadenosine phosphorylase) deleted cancers will result in an IND submission for AG-270 to the FDA before the end of 2017. Updated data will be presented in March. 15% of all cancers have MTAP deletions. Celgene is collaborating on AG-270.

Agios entered into a global license agreement in April with Aurigene Discovery Technologies for undisclosed small molecule cancer targets.

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

Cash (including equivalents & securities) ended at $503.2 million, down sequentially from $573.6 million. No debt. In April raised another $270 million through a public stock offering. Believes now has cash to operate through the end of 2019, including investment in infrastructure and more trials and preclinical discovery.

GAAP operating expenses were $776 million, consisting of: $62.7 million for R&D and $14.8 million for G&A. Loss from operations was $67.0 million. Interest income was $0.9 million.

All development costs for AG-221 are paid by Celgene, and 50% for AG-881. But Agios is responsible for 100% of development expenses for AG-120.

Q&A:

Update glioma data, what will you be looking for? Low grade glioma has few therapies. We want further safety and efficacy data. Given natural history, treatment could go a long time, so tolerability is important. 881 is still in dose escalation.

Cholangiocarcinoma data at ASCO? In 2015 we had data on 25 patients. Now we have 75 patients with safety and efficacy data. Also looking for mechanism of action relation to outcome.

348, is breakthrough designation still possible? Fast Track status is independent of possible breakthrough designation.

Enasidenib data at ASCO, will we see all the expansion cohorts? Title of presentation indicates will see both dose expansion and expansion data.

For Idhifa we pay about one-third of commercialization costs, with Celgen paying the rest.

What is currently used to treat cholangiocarcinoma, what patients are in your trial? Reponse rates for platinum-based frontline therapy are about 20%, and most patients are dead within a year. After frontline there is no standard of therapy. Situation is grim. Our patients had prior chemotherapy or treatment. Radiation therapy is not used often in this disease.

AML frontline goals? Strategy is speed and breadth for any patient with an IDH mutation. Phase 3 decision was based on safety and efficacy of single agents. 7+3 therapy does have a high rate of complete remission, but a curative potential in only a small number. Our goal would be improved long-term survival and cure rate. For Vidaza as a single agent you expect about a 20% complete remission rate, then overall survival of about 10 months, with little curative potential.

20% to 25% of cholangiocarcinoma patients have an IDH mutation. It must be documented to be in our studies.

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