Analyst Conference Summary

biotechnology

conference date: November 1, 2017 @ 5:00 AM Pacific Time
for quarter ending: September 30, 2017 (Q3, third 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), during the quarter.

Basic data (GAAP):

Revenue was $11.4 million, down sequentially from $11.7 million, and up from $9.0 million year-earlier. All revenue is from collaborations.

Net income was negative $77.1 million, up sequentially from negative $83.1 million, and down from negative $62.8 million year-earlier.

EPS (diluted GAAP) was negative $1.59, up sequentially from negative $1.78, and up from negative $1.63 year-earlier.

Guidance:

none

Conference Highlights:

David Schenkein, M.D., CEO of Agios said "We achieved two key 2017 goals in the third quarter with the approval and launch of Idhifa with our partner Celgene for patients with IDH2m R/R AML and the design completion of the AG-348 pivotal program in PK deficiency. We are now focused on completing the NDA for our first wholly owned product, ivosidenib for IDH1m R/R AML and will present the core data from the submission next month at ASH. In addition, the year-end submission of our IND for AG-270 targeting MTAP-deleted tumors remains on track, highlighting our commitment to remain a research-focused organization pursuing novel science with the potential to change patients’ lives.”  

$0.7 million of revenue in the quarter was from royalties for Idhifa.

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 should complete enrollment in 2021. Phase 1 expansion phase data for AML will be presented at ASH. An NDA for R/R AML could be submitted to the FDA by the end of 2017.

AG-120 (ivosidenib) NDA for IDH1m positive relapsing/remitting AML is planned for submission by the end of 2017. Agios fully owns AG-120.

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. T

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 for Pyruvate Kinase Deficiency (PKD) hemoglobin levels new data to be presented at ASH. Has Fast Track designation. Finalized the plan for the two pivotal trials to start in the first half of 2018, and described them in detail. Agios fully owns 348.

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.

AG-881 preclinical data for IDHm solid and hematological malignancies was presented an international conference, showing efficacy at suppressing the target molecule.

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 $641.7 million, up sequentially from $715.9 million. No debt.

GAAP operating expenses were $90.4 million, consisting of: $72.9 million for R&D and $17.5 million for G&A. Loss from operations was $79.0 million. Interest income was $1.9 million.

Q&A:

Frontline AML at ASH, what is the standard of care response rate? For 7 + 3 chemo, there are younger patients with CR at 60% to 70%. With Vidaza CR more like 20%. We added 120 to each, with the goal of understanding feasibility.

Idhifa sales details? Too early, and Celgene will be in charge of communications.

Gating factors for initiating the PKD (348) trial? We are really pleased with regulator input from the FDA and EU. We are vetting sites. Both studies will be global.

Safety is clearly the key gating factor when combining with 7 + 3. We showed ivosidenib can be safely added. We are optimistic about the study.

120 v. 881, why not against placebo? We can't measure 2hg in the plasma of patients with glioma. We want to bring the best drug forward. 881 has been optimized for brain penetration. We want to understand what the impacts are, and how 120 and 881 differentiate in practice. Some patients will get neither drug. The vast majority of patients who have glioma are IDH1 positive.

Talked about the variety of cancers with MTAP deletion, will concentrate on patients with the deletions in both alleles.

When we prepare to launch ivosidenib we will need to increase our SG&A spend.

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