Analyst Conference Summary

biotechnology

conference date: July 29, 2021, @ 5:00 AM Pacific Time
for quarter ending: June 30, 2021 (Q2, second quarter 2021)

Forward-looking statements

Overview: Completed FDA and EU submissions for mitapivat for pyruvate kinase deficiency.

Basic data (GAAP):

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

Net income was negative $86 million, down sequentially from $1.87 billion, and up from negative $90 million year-earlier.

EPS (diluted GAAP) was negative $1.41, down sequentially from $26.95, and down from negative $1.31 year-earlier.

Guidance:

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

Conference Highlights:

Jackie Fouse, CEO of Agios said "Our first quarter as a company solely focused on genetically defined diseases was marked by several significant milestones, most notably our U.S. and EU regulatory submissions for mitapivat for the treatment of adults with PK deficiency, bringing us one step closer to potentially delivering the first disease-modifying treatment for people with this serious and underserved condition." Q2 2021 was the first full quarter since the sale of the cancer division to Servier.

In Q2 for mitapivat in adults with PK deficiency, submitted an NDA to the FDA in the US and a marketing authorization in the EU. 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.

Completed the sale of the oncology portfolio to Servier in a transaction worth up to $2 billion plus royalties on March 31, 2021, and will repurchase $1.2 billion of stock by year-end 2021.

In Q1 2021 entered collaboration with 23andMe for PK deficiency carrier status report. Also launching a patient education program in Q2 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. 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.

Agios plans to initiate 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 $1.73 billion, down sequentially from $2.36 billion. No debt. In Q2 repurchased 10.5 million shares from Bristol-Myers at $50.41 per share.

Cost of Sales $0 million. GAAP operating expenses were $91 million, consisting of: $62 million for R&D and $29 million for G&A. Loss from operations was $91 million. Gain from oncology business sale $2 million. Interest expense was $0.1 million. Other income $6.5 million. Net loss from discontinued operations $3.4 million.

Q&A summary:

How frequently to PKD patients see their doctors? The issue is not getting patients to come back in, most are monitored, frequency depending on severity of disease. We are focussed on patient profiling, identification, and physician education.

Filings Hem or rate disease? FDA team is cardiology/renal. They have been working with us a long time and are well-acquainted with the disease. They guided on designing the trials.

Pricing? Premature. If the larger sickle cell market is approved, we will price based on the data available then.

Size of sales and marketing team? 20 sales reps, a few nurse-educators. We have experts working with us in the EU, but may partner there.

Patient id, numbers? We have identified about 1,000 patients so far in the US and EU. In a Spanish screening we saw about 20% of anemia patients diagnosed with PKD. We expect testing to accelerate now that we have our full team in the field. At first payers will require exceptions to make payments. It may take time for physicians to work through access challenges.

Ad Com and Priority Review? We will find out 60 days from when we filed, so mid-August. It is too early to tell if there will be an Ad Com meeting.

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