Analyst Conference Summary

biotechnology

conference date: November 3, 2021, @ 5:00 AM Pacific Time
for quarter ending: September 30, 2021 (Q3, third quarter 2021)

Forward-looking statements

Overview: Filed for FDA and EU approval of mitapivat for PK deficiency.

Basic data (GAAP):

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

Net income was negative $89 million, down sequentially from negative $86 million, and up from negative $99 million year-earlier.

EPS (diluted GAAP) was negative $1.48, down sequentially from negative $1.41, and down from negative $1.15 year-earlier.

Guidance:

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

Conference Highlights:

Jackie Fouse, CEO of Agios said "As we look ahead to the end of the year and to 2022, Agios is extremely well-positioned to enter our next phase of growth, with our first genetically defined disease commercial launch on the horizon, the expected initiation of three pivotal adult trials and two pediatric PK deficiency trials and a robust pipeline filled with optionality and possibility. We look forward to sharing more insights on our clinical and preclinical pipeline and our commercial launch efforts at our investor day in November." Patient support infrastructure and sales team are in place.

In Q3 for mitapivat in adults with PK deficiency, submitted an NDA to the FDA in the US and a marketing authorization in the EU. PDUFA date is February 17, 2022. Launched myAgios patient support services for people living with PK deficiency and their caregivers, providing tailored support, educational resources and opportunities to connect with other patients and caregivers in the community. Also completed hiring of sales and support team for the launch. Over 1,000 orders for AnemiaID have been received as of end of July 2021.

Pausing share repurchases to use capital for development.

There will be an Investor Day on November 17, 2021.

For mitapivat, at EHA Virtual Congress, June 9-17, 2021 presented full postive data from Phase 3 ACTIVATE study of mitapivat in adults with PK deficiency who do not receive regular transfusions and Phase 3 ACTIVATE-T study of mitapivat in adults with PK deficiency who receive regular transfusions. More data will be presented at ASH, with abstracts online on Nov. 4. The Phase 2 study of mitapivat in adults with alpha and beta thalassemia who do not receive regular transfusions met its primary endpoint. In thalassemia plans to launch 2 Phase 3 trials by the end of 2021. Also preparing for a sickle cell trial to launch by year end 2021.

In Q3 2021 initiated two more Phase 3 studies of mitapivat in not regularly transfused and regularly transfused adults with thalassemia by year-end 2021. Will start a Phase 2/3 study in sickle cell disease by year-end. More data will be sumitted at ASH in December.

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

Cash (including equivalents & securities) ended at $1.40 billion, down sequentially from $1.73 billion. No debt. In Q3 used $254 million for repurchasing shares.

Cost of Sales $0 million. GAAP operating expenses were $91 million, consisting of: $64 million for R&D and $27 million for G&A. Loss from operations was $91 million. Gain from oncology business sale $2 million. Interest income was $0.3 million. Other income $5 million. Net loss from discontinued operations $5 million.

Q&A summary:

Sickle cell study? Phase 3 primary endpoints are hemoglobain response and analysed rate of occlusion crises. We only need to hit one to succeed.

Hydroxyurea in sickle cell trials? We do accept patients with that, but stratify. It is too early to tell if it matters, we would do a subgroup analysis at the end of Phase 3.

PK prevalence, identification of patients? 3,000 to 8,000 in U.S. plus EU. Believes the number will continue to grow. Our planning assumes 3,000.

AG-946 data, plan? We will give that at Investor Day.

Increasing volumes for PK tests? Saw an uptick in requests in Q3, probably due to first full quarter with a team in the field. We are near 2,000 tests. Expects results to fluctuate. Spanish study was controlled, now we are in all-comers, so expect a difference. In Spain we saw 20%, that is still the upper end of the range. Not ready to report current results yet.

We expect commercial payers to sign on for reimbursement first, Medicaid and Medicare are expected to take longer.

In addition to the genetic test there is already an enzyme test already in use.

Share repurchases, other use of cash? We repurchased 23% of shares already. We can accelerate our pipeline. External BD valuations have been coming down. So we might fing a BD deal. We retain the option of using the cash for repurchases. When we received the funds from Servier we always planned to be flexible. We will talk more about incremental investments in our pipeline on Investor Day.

We are not planning to pivot to 946 in sickle cell. We are moving as fast as we can on mitapivat.

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