Analyst Conference Summary

biotechnology

conference date: May 4, 2023, @ 5:00 AM Pacific Time
for quarter ending: March 31, 2023 (Q1, first quarter 2023)

Forward-looking statements

Overview: Revenue from Pyrukynd is still insignificant, but ramping.

Basic data (GAAP):

Revenue was $5.6 million, up 30% sequentially from $4.3 million, and up 700% from $0.8 million year-earlier.

Net income was negative $81 million, down sequentially from $36.5 million, and up from negative $95 million year-earlier.

EPS (diluted GAAP) was negative $1.47, down sequentially from $na, and up from negative $1.74 year-earlier.

Guidance:

none

Conference Highlights:

Brian Goff, CEO of Agios said "Agios is the pioneering leader in PK activation . . . We closed screening of the Phase 3 studies of Pyrukynd in thalassemia with enrollment expected to be complete later this month, and look forward to the data readout of the Phase 2 portion of the Rise Up study of Pyrukynd in sickle cell disease in the middle of this year." Believes Pyrukynd launch will be slow and steady, preparing for larger indictions.

Pyrukynd revenue was $5.6 million. Fourth full quarter since FDA approval. 89 patients on Pyrukynd and 127 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. Screening for the trial has been closed, with enrollment completion expected in May. 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, with possible complete enrollment in 2024.

Working on taylored commercial launch strategies for the various upcoming PK treatable indications.

Believes there could be 4,000 on-label Pyrukynd patients in the U.S., which could lead to annual revenue of $200 to $225 million. Thalassemia and sickle cell potential patient numbers are much larger [18,000 and 120,000].

Servier's Phase 3 trial of vorasidenib in patients with residual or recurrent IDH mutant low-grade glioma met both its primary endpoint and key secondary endpoints. As part of the divestiture of the Agios oncology business to Servier, Agios retains rights to a potential $200 million milestone upon FDA approval of vorasidenib and 15% royalties on potential U.S. net sales.

Agios is working on a preclinical phenylketonuria (PKU) agent, with an IND filing expected by year-end 2023.

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.

Cash (including equivalents & securities) ended at $1.0 billion, down sequentially from $1.1 billion. No debt.

GAAP operating expenses were $96 million, consisting of: Cost of goods $0.6 million; $67 million for R&D and $28 million for SG&A. Loss from operations was $91 million. Interest income was $8 million. Other income $1.5 million.

Q&A selective summary:

Sickle cell scenarios? Hoping for success, should be seemless as is Phase 2/3. Would use the same clinical sites. Primary endpoint is hemoglobin improvement. Safety is key, especially to dose level. We want a hemoglobin plus outcomes dynamic. Being able to stay on the therapy chronically. We have some study flexibility for some of the scenarios.

AG-946 sickle cell cohort v. Pyrukynd? 946 timeline for SC data not available yet, and the safety profile is not as strong yet. Will get an MDS readout near year-end.

Vorasidenib? We do not have clarity on timing for milestone payment or FDA decision, but will update you when that is available.

PAH stabilizer vs. quban (sp?)? It is early. Our PAH program (for PKU)is designed to target unmet need. Hope is to provide differentiation from current products, with better safety or efficacy.

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