Analyst Conference Summary

biotechnology

conference date: February 13, 2025, @ 5:00 AM Pacific Time
for quarter ending: December 31, 2025 (Q4, fourth quarter 2025)

Forward-looking statements

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

Basic data (GAAP):

Revenue was $10.7 million, up 19% sequentially from $9.0 million, and up 51% from $7.1 million year-earlier.

Net income was negative $96.5 million, down sequentially from $948 million, and down from negative $95.9 million year-earlier.

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

Guidance:

none

Conference Highlights:

Brian Goff, CEO of Agios said "Agios had a transformative year in 2024, continuing to successfully deliver on all priorities. Our Pyrukynd franchise is poised for multi-billion-dollar potential, driven by the key milestones we achieved last year, including filing for regulatory approval in thalassemia across four markets and completing enrollment in our Phase 3 RISE UP study for sickle cell diseases. Backed by a strong balance sheet and a highly experienced team, Agios is focused on maximizing the potential Pyrukynd launches in thalassemia and sickle cell disease in 2025 and 2026, respectively, while advancing and diversifying our key pipeline programs and strategically deploying our capital to drive long-term growth. We are well positioned to bring significant value for shareholders, healthcare professionals and patients, as we build towards a breakout year in 2025." Believes Pyrukynd launch will continue slow and steady while preparing for larger indications. Plans on launch for Thalassemia in 2025. Believes total Pyrukynd opportunity is for multiple billions of dollars.

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 $10.7 million, up 19% sequentially from $9.0 million, and up 51% from $7.1 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. 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 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.

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 $1.5 billion, down sequentially from $1.66 million. No debt.

GAAP operating expenses were $na million, consisting of: Cost of goods $1.3 million; $82.8 million for R&D and $51.7 million for SG&A. Loss from operations was $na million. Interest income was $na million. Other income $na billion. Income tax $na million.

Q&A selective summary:

Plan for safety data post liver injury incident? We updated investors when we became aware of a new safety issue for thalassemia.

Mitapivat peak sales potential? In thalassemia 2 thirds of US patients have no approved therapy options. With sickle cell unmet need increased recently due to challenges with current options. A lot of conviction on potential.

Sickle cell trial protocol changes? We did review monitoring for liver enzymes. Now once a month for the first six months of exposure.

There is a need for more than one PK activation option in sickle cell, so we will be guided by the Tebapivat data on the potential path to approval.

Business development with cash? First priority is to maximize the launches. Then mid and early stage products. We have scaled up external search and evaluation, in rare diseases. Looking for significant value creation, so disciplined.

Mitapivat Thalessemia launch trajectory? Prepared for launch. Initial launch would not include a bolus. We would capture patients as they revisit their doctors. 65% of the population is out initial target, about 4000 patients.

Pediatric PK deficiency? Pediatric development is very hard. We now have experience with pediatric trials we can apply to future programs.

We are maximizing disease awareness now that we have a PDUFA date.

OpenIcon Analyst Conference Summaries Main Page

Search

More Analyst Conference Pages: