Analyst Conference Summary

biotechnology

conference date: May 2, 2019, @ 5:00 AM Pacific Time
for quarter ending: March 31, 2019 (Q1, first quarter 2019)

Forward-looking statements

Overview: Revenue rose nicely y/y, but still has a long way to go to justify a $3 billion market capitalization.

Basic data (GAAP):

Revenue was $30.2 million, up less than 1% sequentially from $30.0 million, and up 243% from $8.8 million year-earlier.

Net income was negative $93.1 million, down sequentially from negative $91.8 million, and down from negative $90.8 million year-earlier.

EPS (diluted GAAP) was negative $1.59, flat sequentially from negative $1.58, and up from negative $1.63 year-earlier.

Guidance:

Has sufficient cash to last until the end of 2020.

Conference Highlights:

Jackie Fouse, CEO of Agios said "We continued to deliver significant progress across our oncology and rare genetic disease programs during the first quarter. Importantly, our U.S. commercial team is driving Tibsovo toward solid performance for its first full year on the market, and we are on track to achieve our internal forecast. We made progress on expansion opportunities for Tibsovo across the frontline AML setting. The FDA accepted our sNDA in newly diagnosed AML, the HOVON/AMLSG Phase 3 intensive chemotherapy combination study initiated, and we received Breakthrough Therapy Designation for the combination of Tibsovo and azacitadine. We also advanced our mitapivat program. Our two pivotal trials in pyruvate kinase deficiency remain on track to complete enrollment this year. We have dosed the first patient in our Phase 2 thalassemia study, and we now expect a NIH-sponsored study in sickle cell disease to initiate this year."

Believes Tibsovo revenue was lower than expected in Q1 because of usual Q1 deductable/donut hole issues resulting in giving more free doses, but sees stronger payments in Q2. Collaboration was up on a payment from Celgene.

Tibsovo (AG-120 or ivosidenib) in R/R AML patients with an IDH1 mutation received FDA approval in July 2018. Submitted a supplemental new drug application (sNDA) for single agent Tibsovo in newly diagnosed AML patients with an IDH1 mutation who are not eligible for standard treatment.

Tibsovo net sales were $9.1 million, down slightly sequentially from $9.4 million. $2.2 million of revenue in the quarter was from royalties for Idhifa from Celgene, flat sequentially from $2.2 million. $17.9 million was from collaborations.

The next steps for the expansion of Tibsovo revenue are EU approval (submitted end of 2018) and expansion to the front line setting for AML. Hopes to be able to treat all front-line AML patients with IDH1 mutation. Data so far are encouraging.

PDUFA date is June 21, 2019 for potential 2019 FDA approval of the supplemental new drug application (sNDA) for single agent Tibsovo for the treatment of patients with newly diagnosed AML with an IDH1 mutation who are not eligible for standard therapy. Updated data from Phase 1 studies of Tibsovo in newly diagnosed AML and First Data from Perioperative Study of Tibsovo and Vorasidenib were accepted for presentation at ASCO 2019.

Agios plans to submit an sNDA to the FDA for Tibsovo for second line or later IDH1 mutant cholangiocarcinoma by year-end 2019. The Phase 3 trial has completed enrollment.

In Q1 2019 Agios presented updated data from the ongoing Phase 1 combination trial of Tibsovo with azacitidine in patients with newly diagnosed AML with an IDH1 mutation at the 17th International Symposium on Acute Leukemias.

Plans to initiate a registration-enabling Phase 3 study of vorasidenib (AG-881) in low-grade glioma with an IDH1 mutation by year-end 2019. is currently working with regulators on the trial design. Phase 1 study for IDHm positive glioma should reported data at ASCO.

By end of Q3 2019 hopes to determine the recommended dose of AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in methylthioadenosine phosphorylase (MTAP)-deleted tumors; initiate expansion arms, including a single-agent arm in a variety of MTAP-deleted cancers and a combination arm in a solid tumor in the first half of 2019. Celgene is collaborating on AG-270.

In Q2 2019 initiated a Phase 1 dose-escalation trial of AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH), in lymphoma.

Will complete enrollment in two global pivotal trials for mitapivat (AG-348) in adults with pyruvate kinase (PK) deficiency by year-end 2019.

Agios dosed the first Phase 2 patient in Q1 and hopes to achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019. The NIH is sponsoring a sickle cell study to start in 2019.

AG-881 (vorasidenib) was chosen (in Q1 2019) as the molecule to continue in the IDH1-mutant glioma study. About 80% of patients are IDH1-mutant.

With Celgene, Agios plans to start a Phase 3 trial for frontline AML combining ivosidenib or enasidenib with 7+3 chemotherapy. Updated Phase 1/2 data from the trial combining Tibsovo or Idhifa with Vidaza was presented at ASCO.

Phase 3 AGILE trial expects full enrollment in 2020 now that event free survival is primary endpoint, in combination with azacitidine for newly diagnosed AML patients with IDH1 mutation ineligible for intensive chemotherapy.

A randomized Phase 3 study (ClarlDHy) of AG-120 (ivosidenib) in IDH1 mutant positive cholangiocarcinoma in continued enrollment, which is expected to complete in first half of 2019. If approved would be the first targeted therapy for this disease, which has about 3,000 IDH1+ patients annually.

Further combination trials with a variety of agents and target variants are planned for ivosidenib.

Cash (including equivalents & securities) ended at $708 million, down sequentially from $805 million. No debt.

Cost of Sales $0.3 million. GAAP operating expenses were $127 million, consisting of: $96 million for R&D and $32 million for G&A. Loss from operations was $97 million. Interest income was $4 million.

Q&A:

Quantify Q1 seasonality? Demand grew in Q1, but some patients who were paying in Q4 went back to free drug in Q1. We saw good volume growth in April. The underlying trends are quite good.

In Cholangiocarcinoma we are looking to double PFS, but positive would be compared to control arm. Tibsovo, taken orally once daily, any number of scenarios would be clinically beneficial, there are no other options.

Size of frontline AML population with expansion in June? Until we have the exact wording for the label, we can't be precise. From when we took our IDH1 inhibitors into the clinic until now, there are many new entrants in AML. We are talking about a number of combination trials, including three agent combinations, and various forms of sequencing.

AG-270 AACR data, picking cancer types? Study specified both daily and BID dosing. Daily dosing had shown good biomarker activity, BID revealed dose-limiting toxicity. Still studying dosing for going forward. AACR data showed inhibiting MAT affects DNA splicing and interacts with taxanes. That pushes us toward NSCLC and pancreatic cancer. So we selected them as our first two indication.

If patients have an IDH1 mutation, there is no reason patients should not be exposed to one of our agents over the course of their therapy.

The regulatory process continues to develop in the EU, and we are developing a commercial team there.

Elderly patients, new designation effects? Breakthrough designation gives an opportunity for Tibsovo plus azacitadine discussions with the FDA.

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