Analyst Conference Summary

biotechnology

conference date: August 3, 2023, @ 5:00 AM Pacific Time
for quarter ending: June 30, 2023 (Q2, second quarter 2023)

Forward-looking statements

Overview: Revenue from Pyrukynd is still low, but ramping. Lots of cash. Bought a preclinical therapy from Alnylam.

Basic data (GAAP):

Revenue was $6.7 million, up 20% sequentially from $5.6 million, and up 50% from $5.6 million year-earlier.

Net income was negative $84 million, down sequentially from negative $81 million, and up from negative $92 million year-earlier.

EPS (diluted GAAP) was negative $1.51, down sequentially from negative $1.47, and up from negative $1.68 year-earlier.

Guidance:

cash is sufficient to operate through at least 2026.

Conference Highlights:

Brian Goff, CEO of Agios said "Since our last quarterly update, Agios has made tremendous progress executing across our industry-leading pipeline of PK activators, and today we are further expanding our portfolio beyond PK activation through focused business development. We announced positive data from the Phase 2 portion of the RISE UP study of mitapivat in sickle cell disease, completed enrollment in three clinical studies, licensed a compelling preclinical program from Alnylam, and continued to strengthen our commercial capabilities to support future anticipated launches. We look forward to the readout of the Phase 2a study of AG-946 in lower-risk MDS by the end of this year and the readouts of the Phase 3 studies of mitapivat in thalassemia next year." Believes Pyrukynd launch will be slow and steady, preparing for larger indications.

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.

Pyrukynd revenue was $6.7 million, up 20% sequentially from $5.6 million, and up 116% from $3.1 million year-earlier. Fifth full quarter since FDA approval. 147 patients on Pyrukynd and have completed prescription enrollment forms and 99 are on therapy, up 11% from Q1. Discontinuations low so far, but label says check for efficacy at 6 months. In Q4 2022 Pyrukynd received marketing authorization in the EU and Great Britain.

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. Hopes to enroll first patient in a Phase 3 study in Q4 2023. The Phase 3 trial for pediatric PK deficiency continues to enroll, with possible complete enrollment in 2024.

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 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 $947 million, down sequentially from $1.0 billion. No debt.

GAAP operating expenses were $100 million, consisting of: Cost of goods $1 million; $69 million for R&D and $30 million for SG&A. Loss from operations was $94 million. Interest income was $8 million. Other income $2 million.

R&D expense in Q2 2023 dropped about $5 million y/y due to force reductions made in 2022, but admin expenses increased by $2 million.

Q&A selective summary:

Licensing factors for PV treatment? PV is a large market that is ripe for disruption. The standard of care is not great. It fits well with our business development criteria and our capabilities. The therapy could be transformational for patients. There is likely a clear regulatory pathway. We have strong conviction in the probability of success. Too early to outline details of clinical program.

Preclinical data for PV asset is very good, safe with good knockdown in primates. Believes should compete well in the PV market.

Data for sickle cell trial may be presented at ASH. Will use 100 mg dose in Phase 3. Sample size will be much larger than in Phase 2.

MDS trial success criteria? We have a different mechanism of action. Enrollment in Phase 2a has been excellent. We are looking for safety and an efficacy signal. We believe this market is ripe for change. This is an oral therapy, which is particularly conventient for an older population.

We have employees who have already worked with RNAi compounds, which helps give us confidence in working on the Alnylam PV potential therapy.

MDS therapy vs. current therapies like Luspatercept (Reblozyl)? 75K or so patients in US and EU. Some similarities with thalassemia. Patients often have an acquired PK deficiency. We will publish more data at ASH. We have deep expertise in this field.

Pyrukynd launch trajectory? We are seeing positive provider feedback and payer support. It is rare, so ramp likely to continue slow and steady. Our sales work serves as a foundation for when we are able to expand to other indications. We see peak sales at $200 to $225 million in the U.S. just from PK deficiency.

For PV, RNAi vs. possible antibodies? The pathway is well-established. Plenty of room for convenience, safety and efficacy over competition. IND enabling studies this year.

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