Analyst Conference Summary

biotechnology

conference date: October 31, 2024, @ 5:00 AM Pacific Time
for quarter ending: September 30, 2024 (Q3, third quarter 2024)

Forward-looking statements

Overview: Received $1.1 billion for FDA approval of Vorasidenib

Basic data (GAAP):

Revenue was $9.0 million, up 5% sequentially from $8.6 million, and up 22% from $7.4 million year-earlier.

Net income was $948 million, up sequentially from negative $96 million, and up from negative $91 million year-earlier.

EPS (diluted GAAP) was $16.22, down sequentially from negative $1.69, and down from negative $1.64 year-earlier.

Guidance:

none

Conference Highlights:

Brian Goff, CEO of Agios said "We had a strong quarter, marked by several important advancements across our pipeline. We completed enrollment of our Phase 3 RISE UP study of mitapivat in sickle cell disease, on our way to sharing topline results in late 2025. Our Phase 2b study of tebapivat in lower-risk MDS was initiated, and we received orphan drug designation from the FDA to support the development of tebapivat in this indication. Our cash position was further strengthened by the receipt of $1.1 billion in payments. This will allow us to maintain this great momentum and fuel our next phase of growth, building towards a franchise with multi-billion-dollar potential. We remain focused on progressing our promising clinical programs to address the critical needs of rare disease patients and look forward to sharing our progress in the coming months." Believes Pyrukynd launch will continue slow and steady while preparing for larger indications. Plans on launch for Thalassemia in 2025.

The high EPS and net income v. the low revenue was due to an $889 million gain on sale of contingent payments and $200 million milestone from gain on sail of oncology business. In Q3 2024 the FDA approved vorasidenib, resulting in a $200 million milestone payment from Servier and a $905 million payment from Royalty Pharma.

Preparing for Thalassemia launch, including market research and access. Plans to file sNDA for mitapivat by end of 2024, with possible launch in 2025. Completed enrollment of Phase 3 sickle cell study in Q3 2024, with data expected late 2025, possible sickle cell launch in 2026. Will present additional data at ASH in December 2024.

Pyrukynd (mitapivat) revenue was $9.0 million, up 5% sequentially from $8.6 million, and up 22% from $7.4 million year-earlier. 127 patients were on Pyrukynd. 211 completed conscription enrollment forms. Slow adoptation is because it is an ultra-rare disease with long times to confirm diagnosis.

The Phase 3 trial for pediatric PK deficiency, regularly transfused, completed enrollment in Q2 2024. Topline results for regularly transfused children did not reach pre-specified statistical significance, but were clinically meaningful. The not regularly transfused study topline results are expected in 2025.

Reported positive Phase 3 transfusion-dependent thalassemia results in Q2 2024. Will apply to the FDA in Q4 2024, with a potential launch in 2025.In Q1 2021 Agios had announced positive Phase 3 Mitapivat data in non-transfusion dependent alpha or beta-thalassemia in January 2024.

Working on tailored commercial launch strategies for the various upcoming PK treatable indications. In Q2 2024 Agios entered into a distribution agreement with NewBridge Pharmaceuticals to advance commercialization of Pyrukynd for Thalassemia in the Gulf Cooperation Council region. NewBridge, a leading will commercialize Pyrukynd in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates.

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].

Tebapivat (AG-946) Phase 2b was initiated in Q3 2024, double-blind, with three dosing levels. Tebapivat granted Orphan Drug Designation in Q3 2024. Looking at tebapivat in sickle cell as well.

The BCAT2 preclinical program targets acidemias.

Vorasidenib (now Voranigo) was approved by the FDA in August 2024. In Q2 2024 Agios announced a $905 million agreement with Royalty Pharma, selling its vorasidenib royalty rights. $905 million was paid upon FDA approval of the drug. Royalty Pharma will receive the entirety of the 15% royalty on annual U.S. net sales of vorasidenib up to $1 billion, and a 12% royalty on annual U.S. net sales greater than $1 billion. Agios retains a 3% royalty on annual U.S. net sales greater than $1 billion.

Cash (including equivalents & securities) ended at $1.66 billion, up sequentially from $645 million. No debt.

GAAP operating expenses were $112 million, consisting of: Cost of goods $1 million; $72 million for R&D and $39 million for SG&A. Loss from operations was $103 million. Interest income was $13 million. Other income $1.1 billion. Income tax $53 million.

Q&A selective summary:

Capital allocation? Priority is potential launches. Then advance pipeline. Could expand pipeline internally or externally. Looking for a clear regulatory path. Agnostic re modality.

Sickle cell expectations, powering? RiseUp trial enrolled in about 12 months. One year durations. Both endpoints well powered. If we missed one of the primary endpoints we could try with a single primary endpoint plus a secondary one like fatigue. Up to regulators on what to accept.

Sickle cell community has been through a lot. We engage with that community. That is one reason recruitment went so well. Trial challenges come from co-morbidities and the disease itself. They are desperate for more working therapies. We have a very different mechanism of action from the competing trial of Oxbryta that was stopped.

MDS Tebapivat did meet its primary endpoint. We are testing higher doses in the 2b trial, hoping to get a stronger signal. MDS is a rapidly growing market with high unmet need. We have a differentiated mechansism v. competitors.

Payor view for thalassemia? Already engaging. Low awareness because it is a rare disease. Same team as had success with payors in PKD.

Africa site issues? Part of our trials are in Africa, where malaria is endemic. We have not seen the problems they saw with Oxbryta.

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