Analyst Conference Summary

biotechnology

conference date: May 5, 2022, @ 5:00 AM Pacific Time
for quarter ending: March 31, 2022 (Q1, first quarter 2021)

Forward-looking statements

Overview: First Pyrukynd revenue; not much traction yet.

Basic data (GAAP):

Revenue was $0.8 million, up sequentially from $0 million, and up from $0 million year-earlier.

Net income was negative $95 million, flat sequentially from negative $95 million, and down from $1.87 billion year-earlier, when a large one-time payment was received from sale of the oncology business.

EPS (diluted GAAP) was negative $1.74, flat sequentially from negative $1.74, and down from $26.96 year-earlier.

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 "the U.S. approval of Pyrukynd in adults with PK deficiency was Agios' first step toward changing treatment paradigms for people with genetically defined diseases, starting with developing the first disease-modifying therapy for a rare, debilitating, lifelong hemolytic anemia. We are now poised to expand our impact for many more patients, including adults with PK deficiency in the EU, pediatric PK deficiency patients and people living with thalassemia, sickle cell disease and low- to intermediate-risk myelodysplastic syndrome. I am tremendously proud of our team’s incredible work on the U.S. launch of Pyrukynd, ongoing regulatory interactions in the EU, five pivotal clinical trials planned or underway, the expansion of our PK activation portfolio with AG-946 and our innovative research engine. We continue to execute in each of these arenas, motivated and inspired by our connections to patients."

Mitapivat in adults with PK deficiency, for treatment of hemolytic anemia, 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; more than 3,500 kits ordered by end of Q1 2022. MyAgios program to educate patients online. PK patients have been tremendous self advocates. Will take some time to get reimbursements, so does not expect much income until at least Q3 2022. But believes there could be 4,000 on-label patients in the U.S., which could lead to annual revenue of $200 to $225 million.

In April 2022 results from the Activate Phase 3 clinical study evaluating Pyrukynd in adults with PK deficiency who do not receive regular transfusions were publishing in the New England Journal of Medicine.

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

Now working on extensive label expansion. 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 Q1 2022 Agios completed the single ascending dose and multiple ascending dose healthy volunteer cohorts of the Phase 1 study of novel PK activator AG-946 and identified doses for the Phase 1 sickle cell disease cohort and Phase 2a study in low- to intermediate risk MDS (myelodysplastic syndrome). Also initiated the sickle cell disease cohort of the Phase 1 study of AG-946. 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 $1.18 billion, down sequentially from $1.29 billion. No debt.

Cost of Sales $0.3 million. GAAP operating expenses were $102 million, consisting of: $70 million for R&D and $32 million for SG&A. Loss from operations was $95 million. Interest income was $0 million. Other income $0 million. Net income from discontinued operations $ million.

Q&A summary:

Reimbursement? Conversations with payers are positive. Currently using medical exceptions process. Should get updates on prior authorization in next couple of quarters.

Initial patient type? Seeing across the entire spectrum of PKD patients. No particular concentrations seen.

Stocking impact on revenue? Initial bolus? There was no bolus, it is happening as the patients show up, get on programs. Physicians are not actively calling their patients in. Revenue based on orders from distribution partners.

For AG-946, we have been able to identify a dose-limiting toxicity with thromobocytopenia. It is manageable, as everything returned to baseline when dosing was discontinued. We had already identified an effective, lower dose for sickle cell and MDS. So we are well on track.

Number of patient starts? Not giving specifics, it is still early in the launch of a rare condition. The medical exception process takes a while. This is how we expected things to go. We are already seeing script refills. We are only using one specialty distribution pharmacy.

Cost of sales as percent of revenue, going forward? Should trend down as volumes go up, we have some fixed costs. It is a small molecule, so gross margins should be healthy. We are being careful with SG&A spend, so that should be the run rate, and for op ex generally.

Difference between registry of 1200 patients and projection of 4000 patients? It is rare and poorly understood. We triangulated data from many different sources to get the higher estimate. We believe there is a like number of patients in Europe.

Of the 3,500 AnemiaID kits ordered, about one-quarter have been completed. Results about as expected, seeing strong interest in the program.

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