Analyst Conference Summary

biotechnology

conference date: August 8, 2017 @ 5:00 AM Pacific Time
for quarter ending: June 30, 2017 (Q2, second quarter 2017)

Forward-looking statements

Overview: Agios is about to be a commercial stage company as IDHIFA, developed in partnership with Celgene, was granted approval by the FDA for certain patients suffering from Acute Myeloid Leukemia (AML), after the quarter ended.

Basic data (GAAP):

Revenue was $11.7 million, up sequentially from $10.5 million, and down from $7.0 million year-earlier. All revenue is from collaborations.

Net income was negative $83.1 million, down sequentially from negative $66.2 million, and down from negative $56.0 million year-earlier.

EPS (diluted GAAP) was negative $1.78, down sequentially from negative $1.56, and down from negative $1.47 year-earlier.

Guidance:

none. Will begin to record royalty revenue in Q3.

Conference Highlights:

David Schenkein, M.D., CEO of Agios said "Our second quarter progress was followed by a remarkable milestone for any biotechnology company, the full approval of our first product, IDHIFA, a treatment for patients with IDH2m R/R AML developed in partnership with Celgene. We are pursuing a similar regulatory strategy for ivosidenib, our wholly owned IDH1m inhibitor, and we remain on track to submit our NDA by year-end. With the pivotal program designed for AG-348 in PK deficiency, we are working to initiate two trials in the first half of 2018 for this rare anemia, which currently has no approved therapies.” Idhifa went from the lab to clinical approval in less than 8 years. Idhifa is now available to patients. "Idhifa will hopefully be the first of many precision medicines we will make available for patients."

Idhifa (AG-221, Enasidenib) for IDH2 positive relapsed/refractory AML (acute myeloid leukemia) was approved by the FDA on August 1, 2017. This is in partnership with Celgene and is based on Phase 1/2 data. The Phase 3 trial continues to confirm the data, despite the full approval. An MDS trial is also planned. Updated Phase 1/2 IDH2m R/R AML data was presented at ASCO. Idhifa is the only therapy approved in this specific indication. Celgene will market Idhifa and pay Agios tiered royalties, ranging from single digits to low double digits.

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 was initiated in the second quarter. An NDA could be submitted to the FDA by the end of 2017.

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 (ivosidenib) in IDH1 mutant positive cholangiocarcinoma in continued enrollment, which is expected to complete in 2019. Data showing durable stable disease from the Phase 1 expansion cohort was presented as ASCO.

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. Finalized the plan for the two pivotal trials to start in the first half of 2018, and described them in detail. Updated DRIVE PK study data will be presented at ASH.

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. 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. Agios paid a $3 million upfront payment towards this in the quarter.

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 $715.9 million, up sequentially from $503.2 million. No debt. In April raised $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 $95.9 million, consisting of: $79.8 million for R&D and $16.1 million for G&A. Loss from operations was $84.6 million. Interest income was $1.5 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:

AG-348 non-transfusion dependent trial plan? Increase in hemoglobin over multiple visits? NTD primary endpoint is an increase of hemoglobin (1.5) at six months. Then they are unblinded and placebo patients can cross over. There is no specific number of visits needed for over 1.5, it just is if a patient has a blip up, because we want to see a sustained response. PRO is a formal secondary endpoint.

ivosidenib expected to be eligible for full approval? Need to talk to the FDA first. The trials were designed in parallel, so we hope for the same treatment.

881? In clinic completed dose escalation for mutant-positive glioma. Working with Celgene on the future plan.

Idhifa six month label for patients without progression? The complete remissions generally occurred in a two to three month time frame. Median time to first response is 1.9 months. Best response CR/CRh is 3.7 months. The idea is to give Idhifa time for its best chance to work, in the absence of progression. We don't know how many patients will be on the drug for that long. We are so pleased with this language. It recognizes the unique mechanism of action.

Frontline AML, adult vs. adolescents? We are doing a Phase 1 trial with Celgene in which ivosidenib or Idhifa (with 7/3 chemo) is given based on the type of mutation. The design of the future trial is under discussion.

348 in pediatrics? Our commitment is to treat all patients, but we started with adults. We need to see its affects on hormones. We have backup molecules in case 348 is not appropriate to children.

348 natural history study enrollment? It is not a treatment study. We enrolled over 200 patients and it is now closed for enrollment. No one has tried to treat this disease before, so interest is high. In the pivotal program we will open a much larger number of sites around the globe.

We are seeking guidance in Europe to see whether we can seek approval with the current package or need to wait for the Phase 3 trial completion.

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