Analyst Conference Summary

biotechnology

conference date: February 23, 2023, @ 5:00 AM Pacific Time
for quarter ending: December 31, 2022 (Q4, fourth quarter 2022)

Forward-looking statements

Overview: Revenue from Pyrukynd is still insignificant.

Basic data (GAAP):

Q4 revenue was $4.3 million, up 23% sequentially from $3.5 million, and up 65% from $2.6 million year-earlier. Non-operating revenue was $128 million from Tibsovo royalty rights.

Net income was $36.5 million, up sequentially from negative $82 million, and up from negative $98.6 million year-earlier.

EPS (diluted GAAP) was negative $na, up sequentially from negative $1.49, and up from negative $na year-earlier.

Full 2022 results:

Revenue was $14.23 million, up from $0 million in 2021.

Net income was negative $232 million, down from $1.60 billion in 2021.

EPS (diluted GAAP) was negative $4.23, down from $26.55 in 2021.

Guidance:

Believes has cash to reach cash-flow positive. No specific revenue guidance for 2023 yet.

Conference Highlights:

Brian Goff, CEO of Agios said "We are poised for significant near- and long-term growth and look forward to a productive 2023, anticipating the readout of our Phase 2 sickle cell disease study and the completion of enrollment in our Phase 3 thalassemia studies, driving toward two additional Pyrukynd indications by 2026." Believes Pyrukynd launch will be slow and steady, preparing for larger indictions.

On October 27, 2022, Agios announced that Sagard Healthcare bought Agios's rights to 5% royalties on U.S. sales of Tibsovo for a one-time payment of $131.8 million.

Pyrukynd revenue was $4.3 million. Third full quarter since FDA approval. 78 patients on Pyrukynd and 105 have completed prescription enrollment forms. Discontinuations low so far, but label says check for efficacy at 6 months. In Q4 2022 Pyrukynd received marketing authorization in the EU and Great Britain.

In 2023 Agios expects to complete enrollment in the Phase 3 studies of Pyrukind in thalassemia. In sickle cell disease the Phase 2 data should be presented mid-year, followed by a decision on whether to conduct a Phase 3 trial. The Phase 3 trial for pediatric PK deficiency continues to enroll.

22 Agios abstracts were presented at ASH 2022.

Continuing to work on payer reimbursement. MyAgios program educates patients online. Will take some time to get reimbursements, perhaps a full year. Believes there could be 4,000 on-label patients in the U.S., which could lead to annual revenue of $200 to $225 million.

Now working on extensive label expansion. In Q1 2022 the first patients were dosed in all three pivotal Phase 3 trials for not regularly transfused and regularly transfused adults with thalassemia, and for sickle cell disease.

An AG-946 Phase 2a study in MDS is expected to complete by year-end 2023; this will be open label. Phase 2b will be double-blind.

The BCAT2 preclinical program targets acidemias. Also working on a preclinical phenylketonuria (PKU) agent with an IND filing expected by year-end 2023.

Cash (including equivalents & securities) ended at $1.1 billion, up sequentially from $1.03 billion. No debt.

GAAP operating expenses were $103 million, consisting of: Cost of goods $0.4 million; $70 million for R&D and $33 million for SG&A. Loss from operations was $37 million. Interest income was $ million. Other income $ million.

Q&A selective summary:

Phase 2 sickle cell readout? Primary endpoint is hemoglobin response, secondary is rate of COC, but that data will be limited by the time on drug. We also have investigator sponsored trials where patients have been exposed longer, so we will look at the totality of the data.

EU sale of Pyrukynd? Looking for EU partners, looking for a partner just for ultra-rare PK deficiency does not make sense. So looking for partners who would work for an expanded label.

Reauthorization so far? Has had some patients not reauthorize, but rate is low overall. Getting a positive reception from patients, physicians, and payers.

Expanding the breadth of prescribing is our core focus, but it takes time, given the ultra-rare nature of the disease. Very few doctors have more than 1 or 2 PKD patients.

Longer term use of Pyrukynd is showing the data gets stronger over time.

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