Analyst Conference Summary

biotechnology

conference date: July 30, 2020, @ 5:00 AM Pacific Time
for quarter ending: June 30, 2020 (Q2, second quarter 2020)

Forward-looking statements

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

Basic data (GAAP):

Revenue was $37.3 million, down 51% sequentially from $87.1 million, and up 42% from $26.2 million year-earlier.

Net income was negative $90.5 million, down sequentially from negative $40.3 million, and up from negative $109.9 million year-earlier.

EPS (diluted GAAP) was negative $1.31, down sequentially from negative $0.59, and up from negative $1.87 year-earlier.

Guidance:

Cash should be sufficient through the end of 2022. Expects full-year 2020 U.S. revenue for Tibsovo of $105-115 million.

Conference Highlights:

Jackie Fouse, CEO of Agios said "We made significant progress on our mitapivat clinical programs, including achieving proof-of-concept in sickle cell disease and planning for our pivotal development programs in both thalassemia and sickle cell disease. For the remainder of 2020, we are focused on the completion of our pivotal trials ACTIVATE and ACTIVATE-T for mitapivat in pyruvate kinase deficiency and securing regulatory feedback on the pivotal programs in both thalassemia and sickle cell disease to enable their initiation next year, the submission of a supplemental new drug application for TIBSOVO in cholangiocarcinoma in the first quarter of 2021, driving enrollment in our ongoing clinical trials and continued strong commercial execution."

National Comprehensive Cancer Network (NCCN) guidelines were updated to recommend treatment with Tibsovo (ivosidenib) for patients with advanced IDH1-mutant cholangiocarcinoma. Agios expects to receive EMA CHMP opinion for Tibsovo in relapsed or refractory acute myeloid leukemia (AML) with an IDH1 mutation by the end of 2020 and report mature overall survival data from ClarIDHy Phase 3 study in Q3 2020. Ff data are supportive, will file an sNDA for Tibsovo in previously treated IDH1-mutant cholangiocarcinoma in Q1 2021.

Some employees have returned to on-site work, planning to extend that despite the pandemic. In April 2020, Agios had anticipated some delays in clinical programs due to hospitals shifting resources to deal with the pandemic.

EU Tibosvo for AML application should get a CHMP opinion by year-end 2020. 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.

Tibsovo plus Vidaza is in a Phase 3 trial 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.

Tibsovo net sales were $27.6 million, up 21% sequentially from $22.7 million and up 101% from $13.7 million year-earlier. $3.3 million of revenue in the quarter was from royalties for Idhifa from Bristol, flat sequentially from $3.3 million and up from $2.7 million year-earlier. $6.4 million was from collaborations.

Agios plans to submit an sNDA to the FDA for Tibsovo for first line IDH1 mutant cholangiocarcinoma by year-end 2020.

AG-881 (vorasidenib) was chosen (in Q1 2019) as the molecule to continue in the IDH1-mutant glioma study. Initiated the registration-enabling Phase 3 INDIGO study of vorasidenib in Grade 2 non-enhancing glioma with an IDH mutation. 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.

AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, for methylthioadenosine phosphorylase (MTAP)-deleted tumors initiated expansion arms, including a single-agent arm in a variety of MTAP-deleted cancers and a combination arm in a solid tumor in Q3 of 2019. Phase 1 daa reported in October 2019 showed biomarker activity. In Q3 2019 Agios initiated two combination arms for the Phase 1 study of AG-270 in MTAP-deleted tumors, one evaluating AG-270 in combination with docetaxel in second-line non-small cell lung cancer and another in combination with nab-paclitaxel and gemcitabine in first or second-line pancreatic ductal adenocarcinoma. Bristol-Myers is collaborating on AG-270.

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 a- and ß-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 mid 2021.

Agios plans to initiate 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 $794 million, up sequentially from $613 million. No debt. In Q2 completed a $255 million purchase agreement with Royalty Pharma for Idhifa royalty rights and milestone payments.

Cost of Sales $0.7 million. GAAP operating expenses were $127.5 million, consisting of: $90.9 million for R&D and 36.0$ million for G&A. Loss from operations was $90.2 million. Interest net expense was $0.3 million.

Q&A:

Mitapivat timing? Hope to initiate a PK deficiency trial next year, it has to include centers in Europe. In Thalessemia and sickle cell trials are still being planned.

Mitapivat for sickle cell, differentiation from competition? It increases hemoglobin. Vs. other PKR activators, we have 4 years of experience and are getting a second molecule ready to enter the clinic. There could also be combination therapies. The current sickle cell drug Oxbryta only shows increased hemoglobin in about 50% of patients, and did not reduce BLCs.

Mitapivat sickle cell enrollment halt? We think it will reopen based on NIH communications. Data for ASH depends on patient enrollment by NIH. Treatment is for 8 weeks, then a taper. Do not know how many additional patients will make it into the ASH presentation data in December.

Tibsovo sales details? No obvious impact from Covid. Seeing new scripts and refills. Getting new subscribers, particularly in the community setting. But there could be a covid impact if patients don't show up for initial diagnosis. We are seeing increased Medicaid utilization. We have little insight into off-label use.

Any plans for sickle cell trials independent of NIH? There are no restrictions on us. We are performing a number of the assays for the trial. We tend to start our own trial in 2021.

Ideta molecule TRMC1 and TRMC5 combinations would be appropriate in the field.

Patient with VOC event in sickle cell trial, more info? The VOC occured during the taper. We saw no events during the actual treatment. VOC is a common event.

We have heard anecdotes of use of Tibsovo for cholangio.

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