Analyst Conference Summary

biotechnology

conference date: May 2, 2024, @ 5:00 AM Pacific Time
for quarter ending: March 31, 2024 (Q1, first quarter 2024)

Forward-looking statements

Overview: If data is good, will apply for Pyrukynd (mitapivat) for thalassemia to the FDA in Q4 2024. Still significant cash balances.

Basic data (GAAP):

Revenue was $8.1 million, up 14% sequentially from $7.1 million, and up 46% from $5.6 million year-earlier.

Net income was negative $92 million, up sequentially from negative $95.9 million, and down from negative $91 million year-earlier.

EPS (diluted GAAP) was negative $1.45, down sequentially from negative $na, and down from negative $1.47 year-earlier.

Guidance:

Conference Highlights:

Brian Goff, CEO of Agios said "We were delighted to report positive data from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia and look forward to announcing topline data from the Phase 3 ENERGIZE-T study in transfusion-dependent thalassemia in the second quarter of this year,” said Brian Goff, chief executive officer at Agios. “Mitapivat has the potential to become the first therapy approved for all thalassemia subtypes, and our commercial organization is actively preparing for a potential launch next year. Beyond thalassemia, we look forward to the RISE UP Phase 3 readout in sickle cell disease next year, with potential for approval in 2026, as we progress toward our vision of becoming a leading rare disease company with a potential multi-billion-dollar franchise in PK activation." Believes Pyrukynd launch will continue slow and steady while preparing for larger indications.

Preparing for Thalassemia launch, including market research and access. Plans to file NDA for mitapivat by end of 2024.

Pyrukynd revenue was $8.2 million, up 14% sequentially from $7.1 million, and up 46% from $5.6 million year-earlier. 120 patients on Pyrukynd and 188 have completed prescription enrollment forms. Discontinuations low so far, but label says check for efficacy at 6 months. Slow adoptation is because it is an ultra-rare disease with long times to confirm diagnosis.

In 2024 will get the transfusion dependent results in the two Phase 3 studies of Pyrukind in thalassemia, then apply to the FDA in Q4 2024, with a potential launch in 2025.In Q1 2021 Agios announced positive Phase 3 Mitapivat data in non-transfusion dependent alpha or beta-thalassemia. Should have Phase 3 transfusion-dependent by end of Q2 2024.

In sickle cell disease the Phase 2 data presented in Q4 2023 were positive. Enrolled the first patient in the Phase 3 study in Q4 2023, with enrollment completion by end of year 2024.

The Phase 3 trial for pediatric PK deficiency continues to enroll, with possible complete enrollment midyear and data readout 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].

Agios dosed the first AG-181 patient for phenylketonuria (PKU) agent, in Q1 2024.

An AG-946 Phase 2a study in anemia from low-risk MDS completed in Q2 2023; positive data reported in Q4 2023. 40% of patients achieved transfusion independence. Phase 2b will be double-blind, with multiple dosing levels. Looking at AG-946 in sickle cell as well.

The BCAT2 preclinical program targets acidemias.

Agios remains rights to a $200 million milestone payment and 15% royalties on US sales if Servier's vorasidenib is approved by the FDA for IDH mutant low-grade glioma. It met both its primary endpoint and key secondary endpoints in the Phase 3 trial. In Q1 2024 Servier announced FDA filing acceptance and priority review of an NDA for vorasidenib for the treatment of IDH-mutant diffuse glioma. PDUFA action date is August 20, 2024. As part of the divestiture of 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.

Cash (including equivalents & securities) ended at $714 million, down sequentially from $806 million. No debt.

GAAP operating expenses were $100 million, consisting of: Cost of goods $1 million; $69 million for R&D and $31 million for SG&A. Loss from operations was $82 million. Interest income was $9 million. Other income $1.6 million.

Q&A selective summary:

Factors to get data in Q2? This is a 48 week study. Timing depends on patients rolling over into ongoing study or not. We know their choices now, so could narrow timeframe. Filing will include all subtypes if second trial data is positive.

Novo Nordisk competition? They are behind us in sickle cell, we should launch first.

946 data release? We have not announced which conference. Focus is on launching Phase 2b.

We expect the second study to be positive, if it falls short we would look at the best path forward.

Will sales strategy differ between the two thalessemia types? We believe there is high unmet need in both populations. We plan to approach both populations in the same way. But with transfusion-dependent patients they visit doctors more regularly.

Cash runway? Runway is at least in 2026. Disciplined spend. Will look at royalty sales of vorasidenib. Runway does not include Ex-US partnering and any cash from that.

Placebo response data? 6% for the placebo is due to fluctuations in the transfusions, which can be symptom-dependent.

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