Analyst Conference Summary

biotechnology

conference date: February 15, 2024, @ 5:00 AM Pacific Time
for quarter ending: December 31, 2023 (Q4, fourth quarter 2023)

Forward-looking statements

Overview: If data is good, will apply for Pyrukynd for thalassemia to the FDA in Q4 2024.

Basic data (GAAP):

Revenue was $7.1 million, down 4% sequentially from $7.4 million, and up 65% from $4.3 million year-earlier.

Net income was negative $95.9 million, down sequentially from negative $91.3 million, and down from negative $36.5 million year-earlier.

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

Guidance:

Cash at least into 2026. That does not include the potential $200 million Servier milestone.

Conference Highlights:

Brian Goff, CEO of Agios said "The past 12 months have been remarkable for Agios. We reported positive data across our industry-leading pipeline of PK activators, including Phase 3 data in non-transfusion-dependent thalassemia, Phase 2 data in sickle cell disease and clinical proof-of-concept in lower-risk MDS, and expanded our preclinical pipeline by in-licensing a novel siRNA program from Alnylam. In 2024, we expect two additional Phase 3 readouts, including the Phase 3 study of mitapivat in transfusion-dependent thalassemia, and are actively preparing for a potential U.S. launch in thalassemia in 2025. Together with our strong cash position, Agios is poised for significant near- and long-term growth as we progress toward of our vision of becoming a leading rare disease company." Believes Pyrukynd (mitapivat) launch will be slow and steady, preparing for larger indications.

Preparing for Thalassemia launch, including market research and access.

Pyrukynd revenue was $7.1 million, down 4% sequentially from $7.4 million, and up 65% from $4.3 million year-earlier. 178 patients on Pyrukynd and have completed prescription enrollment forms and 109 are on therapy. 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 Q3 2023 Agios had completed enrollment in the two Phase 3 studies of Pyrukind in thalassemia. In Q4 2023 announced positive data in non-transfusion dependent. In 2024 will get the transfusion dependent results, apply to the FDA in Q4 2024, with a potential launch in 2025. 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 and data readout in 2024.

On August 3, 2023 announced a license agreement with Alnylam for an RNAi potential therapy, in preclinical stage, for polycythemia vera. Upfront payment is $17.5 million for exclusive global rights. Potential milestones total $130 million, and if commercialized will owe tiered royalties. As of November 2023 Agios is working on forwarding the program.

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. Still looking at trying this 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 varasidenib 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. Servier is expected to file for approval soon.

Cash (including equivalents & securities) ended at $806 million, down sequentially from $872 million. No debt.

GAAP operating expenses were $111 million, consisting of: Cost of goods $0.6 million; $77.5 million for R&D and $35.3 million for SG&A. Loss from operations was $95.9 million. Interest income was $na million. Other income $na million.

Full year 2023 revenue was $26.8 million. Net income negative $352 million. GAAP EPS negative $6.33.

Q&A selective summary:

Transfusion dependent study goals? We have a different primary endpoint than used for Reblozyl. We are using a 50% reduction in any 12 week rolling period. Better reflects the real world. Common secondary endpoints. The bar of 50% is higher than 33% they used. [WPM: Reblozyl sales $320 million in Q4 2023]

Non-transfusion dependent data, Phase 2 v. Phase 3? Did use different primary endpoint, in 2 used one time point, in 3 used rolling average of measurements. Did not see waning hemoglobin response. Always some fluctutation over time. Very confidend in the results, saw improved outcomes, not just hemoglobin.

Reblozyl (luspatercept) is not labelled for non-transfusion dependent thalassemia. There would be competition in transfusion dependent disease. The mechanism of action is very different. Ours produces better red blood cell health, while Reblozyl increased blood production. Our benefits go beyond improved hemoglobin production.

Powering assumptions in transfusion dependent? We do not think you need to increase total hemoglobin, their hemoglobin goes down between transfusions, we keep the blood cells healthier to make transfusions less necessary.

Reasoning behind multi-billion dollar prediction? From rapid progress in pipeline from ultra-rare to more prevelent diseases. Sickle cell population size is well characterized. Over 100,000 sickle cell patients in US alone. Thatlassemia is a mid-range opportunity, next year, then sickle cell in 2026.

We are heavilly focussed on our Phase 3 sickle cell enrollment right now.

PKD opportunity, really? Still expect $200 to $225 million per year in the U.S., eventually.

OpenIcon Analyst Conference Summaries Main Page

Search

More Analyst Conference Pages: