Analyst Conference Summary

biotechnology

conference date: April 29, 2021, @ 5:00 AM Pacific Time
for quarter ending: March 31, 2021 (Q1, first quarter 2021)

Forward-looking statements

Overview: Transitioning from an oncology focus to a genetic disease focus. Completed sale of oncology business.

Basic data (GAAP):

Revenue was $0 million, down sequentially from $44.0 million, and down from $na million year-earlier.

Net income was $1.87 billion, up sequentially from negative $97.7 million, and up from $40 million year-earlier.

EPS (diluted GAAP) was $26.95, up sequentially from negative $1.41, and up from negative $0.59 year-earlier.

Guidance:

Cash should fund development without the need to raise additional equity.

Conference Highlights:

Jackie Fouse, CEO of Agios said "With the recent closing of the sale of our oncology business, we are excited to embrace a focused future in genetically defined diseases. In the weeks and months ahead, we look forward to a number of important catalysts, beginning with presentations at the EHA 2021 Virtual Congress, where we will share data from our ACTIVATE and ACTIVATE-T studies of mitapivat in adults with pyruvate kinase (PK) deficiency, as well as data from our Phase 2 study of mitapivat in thalassemia. The PK deficiency data will form the basis of our upcoming submissions for regulatory approval in the U.S. this quarter and in the EU mid-year. Additionally, we look forward to further exploring the impact of mitapivat in thalassemia and sickle cell disease as we launch two Phase 3 studies and a Phase 2/3 study, respectively, later this year. The courage and needs of the patients and families that we seek to serve continue to be our great motivation, and we are proud of our recent educational and patient-focused efforts that aim to make a meaningful impact on rare hemolytic anemia communities."

Completeed the sale of the oncology portfolio to Servier in a transaction worth up to $2 billion plus royalties March 31, 2021, and will repurchase $1.2 billion of stock by year-end 2021.

In Q1 2021 entered collaboration with 23andMe for PK deficiency carrier status report. Also launching a patient education program in Q2 2021.

For mitapivat, at EHA Virtual Congress, June 9-17, 2021 plans to present data from Phase 3 ACTIVATE study of mitapivat in adults with PK deficiency who do not receive regular transfusions; Phase 3 ACTIVATE-T study of mitapivat in adults with PK deficiency who receive regular transfusions; Phase 2 study of mitapivat in adults with alpha and beta thalassemia who do not receive regular transfusions. In thalassemia plans to launch 2 Phase 3 trials by the end of 2021. Also preparing for a sicle cell trial.

In Q3 2020 initiated first-in-human study in healthy volunteers for AG-946, a next-generation PKR activator. Should have data by end of 2021.

In Q1 2021 agreed to repurchase from Bristol-Myers Squib 7.1 million shares of Agios stock for $344.5 million or $48.38 per share.

Cash (including equivalents & securities) ended at $2.36 billion, up sequentially from $670 million. Gain was from sale of oncology business. No debt.

Cost of Sales $0 million. GAAP operating expenses were $91 million, consisting of: $58 million for R&D and $34 million for G&A. Loss from operations was $91 million. Interest net income was $3 million. Net income from discontinued operations $1.97 billion.

Q&A summary:

New indications for mitapivat and AG-946? MDS anemia could be addressed through wild-type PKR. PKM2 activation may open some doors, it is involved in a variety of diseases. It is expressed in the brain, retina, muscle and other tissues. We are still looking at non-clinical data.

Pricing thoughts? Ultra orphan indication, will price to reflect clinical value. But in sickle cell may have to adjust price due to the payer mix.

Our focus is on our internal development, but we do keep an eye open for outside, early interesting potential therapies.

Current repurchase plan is for up to $600 million, depends on stock price.

The 23 and Me chip looks at just the most common genetic variant. What we have seen thus far is very encouraging at identifying carriers.

We are about a year out from full approval for mitapivat, we are hiring sales team, contacting payers, etc.

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