Analyst Conference Summary

biotechnology

conference date: November 2, 2023, @ 5:00 AM Pacific Time
for quarter ending: September 30, 2023 (Q3, third quarter 2023)

Forward-looking statements

Overview: Revenue from Pyrukynd is still low. Waiting on data to expand label.

Basic data (GAAP):

Revenue was $7.4 million, up 10% sequentially from $6.7 million, and up 111% from $3.5 million year-earlier.

Net income was negative $91.3 million, down sequentially from negative $84 million, and down from negative $81.7 million year-earlier.

EPS (diluted GAAP) was negative $1.64, down sequentially from negative $1.51, and down from negative $1.49 year-earlier.

Guidance:

Cash at least into 2026.

Conference Highlights:

Brian Goff, CEO of Agios said "Agios is approaching a catalyst-rich period, with three mid-to-late-stage data readouts expected by the end of next year, and a total of six by the end of 2025. We are excited to report dosing of the first patient in the Phase 3 portion of the pivotal RISE UP study of our leading PK activator, mitapivat, in sickle cell disease and look forward to sharing more detailed data from the positive Phase 2 portion of RISE UP at an upcoming medical meeting. We look forward to future data readouts across our industry-leading pipeline of PK activators, including the Phase 2a study of AG-946 in lower-risk MDS by the end of this year and both Phase 3 studies of mitapivat in thalassemia next year." Believes Pyrukynd (mitapivat) launch will be slow and steady, preparing for larger indications.

Q3 revenue was lower than expected, just a fluctuation in patient add rate.

Pyrukynd revenue was $7.4 million, up 10% sequentially from $6.7 million, and up 111% from $3.5 million year-earlier. 160 patients on Pyrukynd and have completed prescription enrollment forms and 100 are on therapy. Discontinuations low so far, but label says check for efficacy at 6 months. Currently prescriptions from 142 physicians. Slow adoptation is because it is an ultra-rare disease.

In Q3 2023 Agios completed enrollment in the Phase 3 studies of Pyrukind in thalassemia, with readouts expected in 2024 and a potential launch in 2025. In sickle cell disease the Phase 2 data presented in Q2 2023 were positive. Enrolled the first patient in the Phase 3 study in Q4 2023. The Phase 3 trial for pediatric PK deficiency continues to enroll, with possible complete enrollment in 2024. A Phase 3 pediatric study of mitapivat in PK Deficiency also completed enrollment in Q3 2023.

Agios believes it is on track for data readouts for Phase 3 mitapivat for thalassemia in 2024. A launch for thalassemia is possible in 2025.

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 is working on a preclinical phenylketonuria (PKU) agent, with an IND filing expected by year-end 2023.

An AG-946 Phase 2a study in anemia from low-risk MDS completed in Q2 2023; open label, data expected before year end. Phase 2b will be double-blind.

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.

Cash (including equivalents & securities) ended at $872 million, down sequentially from $947 million. No debt.

GAAP operating expenses were $108 million, consisting of: Cost of goods $1 million; $82 million for R&D and $26 million for SG&A. Loss from operations was $101 million. Interest income was $8 million. Other income $1 million.

ASH is dedicating a session on PK activation for the first time.

Q&A selective summary:

MDS readout scope? Consistent with past topline data, full details will be held for a medical meeting. There is a bar for success. Goal is to set up 2b for maximum success. Enrolled faster than expected.

AG-946 opportunity? Good to have more than one PK activator. In addition to MDS will look at in in sickle cell disease. It gives us a lot of optionality.

In AG-946 2b we plan to enroll patients with a higher transfusion version than in 2a.

Pediatric PKD opportunity? About 3,000 to 8,000 potential patients total patients in U.S. About 20% are pediatric. It also gives a safety signal for the thalassemia and sickle cell populations.

Discontinuations? We expect variability q/q. So far discontinuation rates are low, but we are dealing with small numbers of patients so far.

Thrombocytosemia? Is more common in MDS patients, and some therapies have reported it as an adverse event. In our trials we have seen it at the highest dose, we are working to understand it. It is manageable and reversible.

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