Analyst Conference Summary

biotechnology

conference date: November 5, 2020, @ 5:00 AM Pacific Time
for quarter ending: September 30, 2020 (Q3, third quarter 2020)

Forward-looking statements

Overview: Revenue growing y/y, but earnings still deep in the red from spending for further pipeline development.

Basic data (GAAP):

Revenue was $34.7 million, down % sequentially from $37.3 million, and up 33% from $26.0 million year-earlier.

Net income was negative $99 million, down sequentially from negative $90.5 million, and up from negative $106 million year-earlier.

EPS (diluted GAAP) was negative $1.43, down sequentially from negative $1.31, and up from negative $1.81 year-earlier.

Guidance:

Has sufficient cash to fund operations through 2022. U.S. full 2020 revenue guidance of $113 to $115 million.

Conference Highlights:

Jackie Fouse, CEO of Agios said "In the third quarter, we made significant strides toward executing against our key 2020 priorities and getting our medicines to patients who need them. Most notably, we generated mature overall survival data from our Phase 3 ClarIDHy study of Tibsovo in previously treated cholangiocarcinoma, prepared for topline data readouts from our two global Phase 3 studies of mitapivat in pyruvate kinase deficiency and advanced our pivotal plans for mitapivat in thalassemia and sickle cell disease. We look forward to building on these achievements with a catalyst-rich fourth quarter and 2021."

EU Tibosvo for r/r AML application was withdrawn in Q3 2020. Reported mature overall survival data from ClarIDHy Phase 3 study in Q3 2020. Plans to file an sNDA for Tibsovo in previously treated IDH1-mutant cholangiocarcinoma in Q1 2021.

Growth of Tibsovo in the U.S. remains strong as prescriber base broadens and frontline approval ramps. There are about 10,000 newly diagosed IDH1/2 newly diagnosed AML patients in the US and EU combined, annually. Duration of use is increasing.

Tibsovo plus Vidaza is in a Phase 3 trial for frontline AML that should complete enrollment by year-end 2020. Investigator-sponsored studies are testing tibsovo with various other agents for AML. There is a Phase 1 expansion trial underway for R/R MDS. Plans submission for cholangiocarcinoma in Q1 2020.

Tibsovo net sales were $31.7 million, up 15% sequentially from $27.6 million and up % from $ million year-earlier. $0.7 million of revenue in the quarter was from royalties for Idhifa from Bristol, down sequentially from $3.3 million and down from $2.7 million year-earlier. Idhifa was impacted by a sales reserve change by BMS. $2.3 million was from collaborations.

In Q2 2020 presented updated data from the Phase 1 dose-escalation study of vorasidenib in IDH-mutant non-enhancing glioma at ASCO. Vorasidenib demonstrated prolonged disease control and encouraging preliminary activity, and a favorable safety profile.

For mitapivat, in Q2 2020 stablished clinical proof-of-concept in sickle cell disease based on a preliminary analysis of data on eight patients from the Phase 1 study being conducted in collaboration with NIH. Seven of eight (88%) evaluable patients experienced a hemoglobin increase, with five of eight patients (63%) achieving a hemoglobin increase of =1.0 g/dL from baseline. Also presented data on 13 patients from the Phase 2 study of mitapivat in non-transfusion-dependent alpha and beta-thalassemia at the EHA Annual Congress in June. Treatment with mitapivat induced a hemoglobin increase of =1.0 g/dL in 12 of 13 (92%) evaluable patients, including four of four (100%) a-thalassemia patients. Received Orphan Drug Designation from the FDA for mitapivat in thalassemia. Expects data from two pivotal trials for PK by end of 2020 and Q1 2021. Could file for PK in US and UK in 2021 with launches in 2022. Agios will host investor events on mitapivat on November 19 for PKR and December 8 for sickle cell disease.

In Q3 initiated first-in-human study in healthy volunteers for AG-946, a next-generation PKR activator, in Q3 2020.

With Bristol, Agios is enrolling a Phase 3 trial for frontline AML combining ivosidenib or enasidenib with 7+3 chemotherapy.

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

Cash (including equivalents & securities) ended at $722 million, down sequentially from $794 million. No debt.

Cost of Sales $0.6 million. GAAP operating expenses were $125 million, consisting of: $90 million for R&D and $35 million for G&A. Loss from operations was $90 million. Interest net expense was $1 million. Non-cash interest expense from sale of future revenue $10 million.

Q&A:

AG946 timeline? The study is ongoing, has dose cohorts in healthy volunteers, eventually a sickle cell cohort. Too early to give a timeline.

Phase 3 start of mitapivat for sickle cell should start next year.

ASH mitpivat sickle cell update, dosing difference? Important thing is values are change from baseline. Patients escalate dosing, whether they respond or not. The difference in 50 from 100 mg in the table are challenging. 50 mg dose is active, we expect 100 to be more, but will vary by patient. The question is patient benefit v. safety. Will have more detail in 1H 2021.

2025 $1 billion guidance, effect of pandemic, EU withdrawal? We set ambitious but achievable objectives. The $1 billion is mainly driven by Tibsovo for AML, based on label expansion. Included some mitapivat, but have other revenue that may come in later in the time period. So still $1 billion objective. Maybe a little better.

EU R/R AML Tibsovo. Main concern was the lack of a control arm. We had historical control data, but that was not enough for them. It was a modest revenue opportunity.

Yes, if the pandemic continues to have a resurgence, we could see a negative impact in Q4. We do benefit from Tibsovo being oral.

Discussed various indicators in sickle cell, including reducing VOC, hemoglobin. Hope is mitapivat addresses theres two main problems.

NIH study markers in ASH abstract for VOC reduction? We think it shows potential to reduce VOCs. The challenge is the lack of a direct correlation in magnitude. With caveats, overall we feel really good about what we are seeing.

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