Analyst Conference Summary

biotechnology

conference date: August 4, 2022, @ 5:00 AM Pacific Time
for quarter ending: June 30, 2022 (Q2, second quarter 2022)

Forward-looking statements

Overview: First full quarter of Pyrukynd revenue.

Basic data (GAAP):

Revenue was $5.6 million, up sequentially from $0.8 million, and up from $0 million year-earlier.

Net income was negative $91.8 million, up sequentially from negative $95 million, and down from negative $86.2 million year-earlier.

EPS (diluted GAAP) was negative $1.68, up sequentially from negative $1.74, and down from negative $1.41 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 "As we look ahead to the next chapter for the company, Agios is operating from a position of strength. We are executing our commercial launch of Pyrukynd, the first therapy for a rare, debilitating, lifelong blood disorder; we also have five pivotal trials underway, multiple early-stage studies planned or ongoing, a promising preclinical pipeline and a strong balance sheet providing optionality for the future growth of the business. As I reflect on my legacy as CEO of Agios, I am proud of the bold and strategic decisions we have made to maximize and accelerate our impact for people with genetically defined diseases." Focus is on moving most promising programs forward while in-licensing potential therapies.

Pyrukynd revenue was $3.1 million. Milestone revenue (from former oncology business) was $2.5 million.

Brian Goff will become CEO on August 8, 2022. Jackie Fouse will become Chair of the Board.

Pyrukynd (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.

Commercial team is now engaging with doctors about PK deficiency. AnemiaID program set up in 2021; more than 4.200 kits ordered by end of Q2 2022, with about 25% completed. In Q2 52 unique patients completed prescription enrollment forms and there were 37 patients on drug. Continuing to work on payer reimbursement. MyAgios program educates patients online. Will take some time to get reimbursements, perhaps a full year, 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. In Q2 2022 Agios initiated Phase 3 studies of Pyrukynd in pediatric patients with PK deficiency who are not regularly transfused and who are regularly transfused, respectively. In Q2 2022 clinical and translational data were presented at EHA, including new data supporting the potential benefits of Pyrukynd treatment in adults with PK deficiency.

An EMA Pyrukynd decision for PK definiency is expected before the end of 2022.

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.

In Q2 an AG-946 sickle cell cohort was initiated. 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. A Phase 2a study in MDS is expected to start by year-end 2022.

The BCAT2 preclinical program targets acidemias. Also working on a preclinical phenylketonuria (PKU) agent.

Cash (including equivalents & securities) ended at $1.10 billion, down sequentially from $1.18 billion. No debt.

GAAP operating expenses were $103 million, consisting of: Cost of Sales $0.4 million; $74.5 million for R&D and $28.3 million for SG&A. Loss from operations was $97.6 million. Interest income was $1.8 million. Other income $4.0 million.

Q&A summary:

Start forms v. on drug patients? Encouraged by trend. Takes 4 to 8 weeks for patients to get on therapy. We see this launch as slow and steady. Also, patients are titrating, are checked at 6 months, some will drop off at that point.

Response criteria by payers? Too early to see any trend, will depend in part on physician assessments.

Test (AnemiaID) is for hemolytic deficiency, it is not specific to PK deficiency. The disease is geographically disperse and patients are not concentrated in any center of excellence.

Thalessemia in non-transfusion dependent cohort opportunity? Same physicians for thalessemia as PKD. Thalessemia patients need a convenient oral therapy. Mechanisms of actions, v. Reblozyl, are very different.

Much repetition.

Sequential revenue growth? Of the 37 on therapy, some have refilled. Too early to quantify future growth, but there was no bolus of patients in Q1 or Q2.

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