Analyst Conference Summary

biotechnology

conference date: February 25, 2021, @ 5:00 AM Pacific Time
for quarter ending: December 31, 2020 (Q4, fourth quarter 2020)

Forward-looking statements

Overview: Transitioning from an oncology focus to a genetic disease focus.

Basic data (GAAP):

Revenue was $44.0 million, up 27% sequentially from $34.7 million, and up 34% from $35.4 million year-earlier.

Net income was negative $97.7 million, up sequentially from negative $99 million, and up from negative $102.4 million year-earlier.

EPS (diluted GAAP) was negative $1.41, down sequentially from negative $1.43, and down from negative $1.60 year-earlier.

Guidance:

Cash should fund programs through 2022. After the Servier transaction should not need to raise more cash.

Conference Highlights:

Jackie Fouse, CEO of Agios said "This past year was a transformative one for Agios. The sale of our oncology business to Servier on attractive terms both allows our oncology portfolio to grow and flourish and facilitates our new laser focus on genetically defined diseases, where we anticipate a catalyst-rich year ahead for mitapivat across our three initial disease indications. In addition to our plans to file for approval for mitapivat in adults with PK deficiency in the U.S. and EU and launching our Phase 3 thalassemia trials later this year, we are pleased to share our pivotal strategy for mitapivat in sickle cell disease which we believe will enable us to pursue a broad label for patients who desperately need new treatment options."

Expects to complete the sale of the oncology portfolio to Servier in a transaction worth up to $2 billion plus royalties, following a shareholder vote on March 25, and execute a meaningful portion of the planned $1.2 billion capital return by year-end.

Tibsovo net sales were $39.1 million, up % sequentially from $31.7 million and up % from $19.6 million year-earlier. $2.9 million of revenue in the quarter was from royalties for Idhifa from Bristol, up sequentially from $0.7 million and down from $3.0 million year-earlier. $2.0 million was from collaborations.

For mitapivat, in Q4 2020 data was positive from two pivotal trials for PK (pyruvate kinase deficiency). Could file for PK in US and UK in 2021 with launches in 2022. Following talks with regulators, in February 2021 announced the Phase 2/3 trial design for sickle cell disease, with approval possible in 2026. Phase 1 sickle data had been presented at ASH in December 2020. In thalassemia plans to launch 2 Phase 3 trials by the end of 2021.

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

Cash (including equivalents & securities) ended at $670 million, down sequentially from $722 million. No debt.

Cost of Sales $1 million. GAAP operating expenses were $136 million, consisting of: $96 million for R&D and $40 million for G&A. Loss from operations was $92 million. Interest net expense was $1 million. Non-cash interest expense from sale of future revenue $6 million.

Q&A:

100 mg dosing data, timing of for sickle cell? Not guiding to Phase 2 yet, but will see more data from NIH trials in 2021.

Sickle cell primary endpoints, pain crisis? Competitor received accelerated approval on base of hemoglobin, no reduction of crisis. FDA sees VOC as a critical endpoint. If we hit on either primary endpoint the study is positive. We hope to have a compelling case, with reaching both endpoints as the best case scenario.

1,100 PK U.S. patients, update on count, identifying? About 1,000 patients, pandemic has slowed identification, but is growing over time. Our ID program should excede our testing goal, and we are seeing an ID rate higher than expected.

Powering on VOC endpoint? Not a co-primary endpoint. Two primary endpoints. Powering stats will be disclosed later. Current anti-VOC drug Adakveo has to be taken into account. EMA and FDA feedback is often inconsistent, our current plan balances factors giving us the best chance of a good outcome. Quantifying pain and improvements can be challenging.

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