Analyst Conference Summary

biotechnology

conference date: May 1, 2025, @ 5:00 AM Pacific Time
for quarter ending: March 31, 2025 (Q1, first quarter 2025)

Forward-looking statements

Overview: Slow product ramp, but PDUFA set for September 7, 2025 for much larger indication.

Basic data (GAAP):

Revenue was $8.7 million, down 19% sequentially from $10.7 million, and up 6% from $8.2 million year-earlier.

Net income was negative $89.3 million, up sequentially from negative $96.5 million, and down from negative $81.5 million year-earlier.

EPS (diluted GAAP) was negative $1.55, down sequentially from $na, and down from negative $1.45 year-earlier.

Guidance:

none

Conference Highlights:

Brian Goff, CEO of Agios said "We are pleased with our strong start to 2025, highlighted by the acceptance of our sNDA for thalassemia with a PDUFA goal date of September 7, 2025. Our engagement with the FDA is progressing as expected, and we are committed to bringing Pyrukynd to thalassemia patients, irrespective of genotype or transfusion needs. Looking ahead, our focus is also on delivering the topline results from the Phase 3 RISE UP study in sickle cell disease, which remains on track for year-end, and continuing to advance our early and mid-stage clinical programs. Supported by our strong financial position and highly experienced team, we are driving forward Pyrukynd's multi-billion-dollar potential while building a pipeline designed for lasting impact, with the goal of creating significant value for shareholders and delivering transformative therapies for patients." Has cash to add external assets to the pipeline.

Completed enrollment of Phase 3 sickle cell study in Q3 2024, with data expected late 2025, possible sickle cell launch in 2026. EU gave orphan status.

Pyrukynd (mitapivat) revenue was $8.7 million, down 19% sequentially from $10.7 million, and up 6% from $8.2 million year-earlier. 130 patients were on Pyrukynd. 223 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. The not regularly transfused study topline results were positive, in Q1 2025. Topline results for regularly transfused children did not reach pre-specified statistical significance, but were clinically meaningful.

Reported positive mitapivat full Phase 3 transfusion-dependent thalassemia results in December 2024. Applied to the FDA and EU in Q4 2024, with a PDUFA of September 7, 2025.

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) for low-risk MDD 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.

In mid-2025 Agios plans to file an NDA for AG-236 for polycemia vera. It is an siRNA targetting TMPRSS6.

Cash (including equivalents & securities) ended at $1.42 billion, down sequentially from $1.5 million. No debt.

GAAP operating expenses were $115 million, consisting of: Cost of goods $1 million; $73 million for R&D and $42 million for SG&A. Loss from operations was $107 million. Interest income was $16 million. Other income $1 billion. Income tax $0 million.

Q&A selective summary:

FDA next steps for thalassemia? No Ad Com. Pleased with progress.

Sickle cell market? Hope patients will have many options. No change in our program based on trial so far. Withdrawal of competitor increases the need for our therapy.

Ex-U.S. launches? The second most important geography for thal is the Saudi region. Will take some time for approval and getting on formularies. We have a strong partner in the gulf. For EU plan something similar, will have an update later.

FDA changes under new administration? No, communications have been normal.

Tebapivat color? Good Phase 1 data is sickle cell. Phase 2 starts midyear. Patient population is 100,000, with high unmet need. Patient responses to therapies varies greatly. We will decide how to move forward after we have more mitapivat data.

Thalassemia launch, transfusion depenence? Attractive rare disease market. Unmet need understood well on both categories. We are focussed on the non-transfusion dependent patients, but pyrukynd also will be of value for transfusion depenent patients.

Phase 2 of Tebapivat for sickle cell is mainly a dose finding study. No liver signal so far. Looking to match against the commercial market needs.

Pyrukynd pricing in future launch? Will talk after the PDUFA date. Thalassemia is a rare disease, we have positive feedback from payers on the product profile.

OpenIcon Analyst Conference Summaries Main Page

Search

More Analyst Conference Pages: