Analyst Conference Summary

biotechnology

conference date: August 1, 2024, @ 5:00 AM Pacific Time
for quarter ending: June 30, 2024 (Q2, second quarter 2024)

Forward-looking statements

Overview: Positive Phase 3 mitapivat data plus likely $1.1 billion vorasidenib cash infusion soonish.

Basic data (GAAP):

Revenue was $8.6 million, up 6% sequentially from $8.1 million, and up 28% from $6.7 million year-earlier.

Net income was negative $96 million, dowm sequentially from negative $92 million, and down from negative $84 million year-earlier.

EPS (diluted GAAP) was negative $1.69, down sequentially from negative $1.45, and down from negative $1.51 year-earlier.

Guidance:

none

Conference Highlights:

Brian Goff, CEO of Agios said "Based on the positive data generated in the Phase 3 ENERGIZE and ENERGIZE-T studies, mitapivat is the first therapy to demonstrate efficacy in all subtypes of thalassemia, and we look forward to filing for FDA review by the end of the year. we were pleased to bolster our cash position through a purchase agreement with Royalty Pharma for our vorasidenib royalty, with Agios now positioned to receive a total of $1.1 billion in payments upon FDA approval of vorasidenib." Believes Pyrukynd launch will continue slow and steady while preparing for larger indications.

Preparing for Thalassemia launch, including market research and access. Plans to file NDA for mitapivat by end of 2024, with possible launch in 2025. Then possible sickle cell launch in 2026.

Pyrukynd (mitapivat) revenue was $8.6 million, up 6% sequentially from $8.2 million, and up 28% from $6.7 million year-earlier. 128 patients were on Pyrukynd and 201 had completed prescription renrollment 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 Phase 3 transfusion-dependent thalassemia results in Q2 2024. Will apply to the FDA in Q4 2024, with a potential launch in 2025.In Q1 2021 Agios had announced positive Phase 3 Mitapivat data in non-transfusion dependent alpha or beta-thalassemia in January 2024.

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.

Working on taylored 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 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].

Agios dosed the first AG-181 patient for phenylketonuria (PKU) agent, in Q1 2024.

An AG-946 (now tebapivat) Phase 2a study in anemia from low-risk MDS completed in Q2 2023; positive topline data reported in Q4 2023. 40% of patients achieved transfusion independence. Phase 2b will be double-blind, with multiple dosing levels, should begin Q3 2024. Looking at AG-946 in sickle cell as well.

The BCAT2 preclinical program targets acidemias.

In Q2 2024 announced a $905 million agreement with Royalty Pharma, selling its vorasidenib royalty rights. $905 million will be paid upon FDA approval of the drug. PDUFA action date is August 20, 2024. 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. Agios will retains the $200 million milestone payment from Servier upon FDA approval. In Q1 2024 Servier announced FDA filing acceptance and priority review of an NDA for vorasidenib for the treatment of IDH-mutant diffuse glioma.

Cash (including equivalents & securities) ended at $645 million, down sequentially from $714 million. No debt.

GAAP operating expenses were $114 million, consisting of: Cost of goods $1.5 million; $77 million for R&D and $36 million for SG&A. Loss from operations was $106 million. Interest income was $8 million. Other income $2 million.

Q&A selective summary:

Newbridge transaction color? Second most important Thalassemia market. Believes best partner in region. Hope to get Saudi approval ASAP. Revenue split deal, no upfront payment. We have done clinical studies in the region. Breakthrough designation in Saudi is pretty unusual.

Vorasidenib cash use, M&A? Focus is efficient use of capital, priority is near-term launches. Internal pipeline development with AG-946. Acquiring a therapy would be very disciplined.

Sickle cell VOCs as an endpoint? Not all patients experience VOCs. Our drug can work for hemolytic anemia at various levels. We believe it can work for no VOC patients, but we could not enroll them in the trial because there would be no working measurement. Fatigue is another secondary endpoint that is important to patients.

Kids trial, was it underpowered, etc.? Ultra rare disease population. Thought sample size was adequate. Depends on data borrowed from adult study. 6 patients becoming transfusion free is a clear demonstration of an effect. So we are very excited about the results, including safety. We are looking at next steps for development.

946 (tebapivat) timeline? Phase 2b is the focus, Phase 2a data will be at an appropriate conference. It is a very important market for us.

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