Analyst Conference Summary

biotechnology

conference date: February 24, 2022, @ 5:00 AM Pacific Time
for quarter ending: March 31, 2021 (Q4, fourth quarter 2021)

Forward-looking statements

Overview: On February 17, 2022 received FDA approval of Pyrukynd (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 $95 million, down sequentially from negative $89 million, and up from negative $98 million year-earlier.

EPS (diluted GAAP) was negative $1.74, down sequentially from negative $1.48, and up from negative $1.41 year-earlier. Share count decreased from about 69 million last year to about 54 million.

Guidance:

Cash expected sufficient to "execute its operating plan through major catalysts and to cash-flow positivity."

Conference Highlights:

Jackie Fouse, CEO of Agios said "With last week's FDA approval of Pyrukynd for the treatment of hemolytic anemia in adults with PK deficiency, we have delivered the first therapy for a rare, debilitating, lifelong disease – and we have set the stage for Agios' next chapter as a leader in the genetically defined disease space. We have made incredible progress over the past year, following our transformational decision to divest our oncology business and accelerate and expand our genetically defined disease portfolio. We are poised to expand our impact to thalassemia, sickle cell disease, myelodysplastic syndrome and beyond, and I am optimistic about the future we are building together with patient communities, our clinical and research collaborators and our dedicated Agios team."

Mitapivat in adults with PK deficiency, received FDA marketing authorization on February 17, 2022. Had previously 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 in Q4.

Commercial team is now engaging with doctors about PK deficiency. AnemiaID program set up in 2021. Will take some time to get reimbursements, so does not expect much income until at least Q3.

Pausing share repurchases to use capital for development.

An EMA Pyrukynd decision for PK definiency is expected by year-end 2022.

In late 2021 or 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. At ASH in Q4 2021 more data from prior trials was presented. Pediatric patient PK deficiency Phase 3 studies will begin around the middle of 2022.

In Q4 2021 Phase 1 AG-946 data was presented at ASH. In Q3 2020 initiated first-in-human study in healthy volunteers for AG-946, a next-generation PKR activator. A Phase 2a study in MDS is expected to start by year-end 2022.

Cash (including equivalents & securities) ended at $1.29 billion, down sequentially from $1.40 billion. No debt.

Cost of Sales $0 million. GAAP operating expenses were $105 million, consisting of: $73 million for R&D and $32 million for G&A. Loss from operations was $105 million. Gain from oncology business sale $3 million. Interest income was $0.3 million. Other income $3 million. Net income from discontinued operations $4 million.

Q&A summary:

Launch metrics? Diagnosis, getting prescriptions, assuring access. It is an underdiagnosed disease. Formulary listings.

Previously diagnosed PKD patient visits? In past year has been once a year or more. With an approved therapy we hope our awareness campaign will get patients to visit their doctors.

Sickle cell portion of study? Looking for any signal, including hemolytic anemia in general. The program gives us the opportunity to move on to MDS. Riseup Sickle study has two endpoints, VOC and anemia. Phase 2 results allow us to pick a dose for the Phase 3 trial.

Business development strategy? Given our internal progress, we are seeing more complementary mechanisms outside. Prefer preclinical or early stage assets. Inborn areas of metabolism, particularly blood diseases, would make sense. Could also be a research partnership.

Feedback from payers on pricing? We talked with them before pricing, feedback is positive. Based on value and unmet disease. [$334,880 annually, before discounts] Physicians have been positive, this is the first and only approved treatment.

EU plan? If approved, it will take 12 to 18 months to get reimbursement approval. We are looking for a partner or partners.

Sickle cell pricing strategy? If we hit both primary endpoints it will be be superior to the agents on the market today, which only address one. Pricing depends on the actual data outcomes and feedback from the broader community. We have a lot of flexibility over time as the label expands.

Any prescriptions yet? We have been having interactions, we do not expect presciptions these first two weeks.

We have not seen any concerns about dose titration.

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