Analyst Conference Summary

biotechnology

conference date: February 13, 2020, @ 5:00 AM Pacific Time
for quarter ending: December 30, 2019 (Q4, fourth quarter 2019)

Forward-looking statements

Overview: Revenue ramping, but on a small base, so a long way from break even.

Basic data (GAAP):

Revenue was $35.4 million, up 36% sequentially from $26.0 million, and up 18% from $30.0 million year-earlier.

Net income was negative $102 million, up sequentially from negative $106 million, and down from negative $92 million year-earlier.

EPS (diluted GAAP) was negative $1.60, up sequentially from negative $1.81, and down from negative $1.58 year-earlier.

Guidance:

Full year 2020 Tibsovo revenue expected between $105 and $115 million.

Conference Highlights:

Jackie Fouse, CEO of Agios said "I'm more confident than ever in the strength of our team and our ability to make a meaningful impact on the lives of patients through great science, a deep pipeline and differentiated therapies. In 2020, our clinical development team is focused on advancing our Phase 3 PK deficiency studies in order to submit a new drug application in 2021, finalizing our pivotal development plan for the PK activation program in thalassemia and establishing proof-of-concept in sickle cell disease. In addition, we are driving enrollment in several Phase 3 studies for our IDH inhibitors in both malignant hematology and solid tumors. Our commercial team is focused on achieving an ambitious revenue target for Tibsovo and increasing market development activities in preparation for a potential launch in PK deficiency."

2025 Vision is for 4 commercial medicines with at least 8 indication. Hopes for 6 or more molecules in the clinic, and positive cash flow.

Increasing treatment duration with Tibsovo is the main driver of revenue growth at present.

EU Tibosvo for AML application should get a CHMP opinion by year-end 2020. Growth of Tibsovo in the U.S. remains strong as prescriber base broadens and frontline approval ramps. There are about 10,000 newly diagosed IDH1/2 newly diagnosed AML patients in the US and EU combined, annually.

Tibsovo plus Vidaza is in a Phase 3 trial that should complete enrollment by year-end 2020. Investigator-sponsored studies are testing tibsovo with various other agents for AML.

Tibsovo net sales were $19.6 million, up 13% sequentially from $17.4 million. $3.0 million of revenue in the quarter was from royalties for Idhifa from Bristol, up sequentially from $2.7 million. $12.9 million was from collaborations.

Agios plans to submit an sNDA to the FDA for Tibsovo for first line IDH1 mutant cholangiocarcinoma by year-end 2020.

In Q3 2019 results from the Phase 3 ClarIDHy study of Tibsovo in previously treated IDH1 mutant cholangiocarcinoma reported at the European Society for Medical Oncology Congress demonstrating significant improvement in progression free survival compared to placebo.

Initiated a registration-enabling Phase 3 study of vorasidenib (AG-881) in low-grade glioma with an IDH1 mutation at year-end 2019.

AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, for methylthioadenosine phosphorylase (MTAP)-deleted tumors initiated expansion arms, including a single-agent arm in a variety of MTAP-deleted cancers and a combination arm in a solid tumor in Q3 of 2019. Phase 1 daa reported in October 2019 showed biomarker activity. In Q3 2019 Agios initiated two combination arms for the Phase 1 study of AG-270 in MTAP-deleted tumors, one evaluating AG-270 in combination with docetaxel in second-line non-small cell lung cancer and another in combination with nab-paclitaxel and gemcitabine in first or second-line pancreatic ductal adenocarcinoma. Bristol-Myers is collaborating on AG-270.

Completed enrollment in two global pivotal Phase 3 trials for mitapivat (AG-348) in adults with pyruvate kinase (PK) deficiency. Phase 2 positive data was published in Q3 2019 in the New England Journal of Medicine. Phase 3 topline data expected by year-end 2020. There are no currently approved therapies for this indication.

Agios dosed the first Phase 2 patient in Q1 and hopes to achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019. The NIH is sponsoring a sickle cell study to start in 2019.

AG-881 (vorasidenib) was chosen (in Q1 2019) as the molecule to continue in the IDH1-mutant glioma study. By end of 2019 will initiate the registration-enabling Phase 3 INDIGO study of vorasidenib in Grade 2 non-enhancing glioma with an IDH mutation.

With Bristol, Agios is enrolling a Phase 3 trial for frontline AML combining ivosidenib or enasidenib with 7+3 chemotherapy.

Agios expects to receive IND clearance for AG-946, a next generation PKR activator (post mitipicat), and initiate a first-in-human study in healthy volunteers in the first half of 2020.

Further combination trials with a variety of agents and target variants are planned for ivosidenib.

In Q2 2019 initiated a Phase 1 dose-escalation trial of AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH), in lymphoma.

Cash (including equivalents & securities) ended at $718 million, up sequentially from $541 million. $277 million was raised in the quarter by a follow-on stock offering. No debt.

Cost of Sales $0.3 million. GAAP operating expenses were $141 million, consisting of: $106 million for R&D and $35 million for G&A. Loss from operations was $106 million. Interest income was $4 million.

Q&A:

Tibsovo EU filing? BMS decided to withdraw application, wait for Phase 3 data. We are arguing that the patient population being rare, this is the best data set we are going to get, and there is unmet medical need. We also have some EU real world data that should illuminate risk/benefit for IDH1 mutations. We are not denying that submitting a non-randomized dataset to be a challenge.

PKD 1000 identified patients? About half in US, half in EU. We are increasing our field team to indentify more potential patients before potential approval at the end of 2021.

The cash flow break-even goal for 2025 does not include revenue for drugs that may be just coming to market in that time frame.

AG-946 plan? Sickle cell data and thalasemia will be presented at EHA. If 946 passes muster in healthy volunteers, we will look at all three diseases. In PKD it has potential for a broader spectrum of activity, against more mutations. Could be more effective with wild-type. But we can't make decisions until we see data. There are 300 mutations that have been described, so there are unknowns.

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