Analyst Conference Summary

biotechnology

conference date: October 31, 2019, @ 5:00 AM Pacific Time
for quarter ending: September 30, 2019 (Q3, third quarter 2019)

Forward-looking statements

Overview: Tibsovo revenue up 27% q/q to $17.4 million. But R&D spend continues to overwhelm the bottom line.

Basic data (GAAP):

Revenue was $26.0 million, down 1% sequentially from $26.2 million, and up 71% from $15.2 million year-earlier.

Net income was negative $106.2 million, up sequentially from negative $109.9 million, and down from negative $94.7 million year-earlier.

EPS (diluted GAAP) was negative $1.81, up sequentially from negative $1.87, and down from negative $1.63 year-earlier.

Guidance:

Believes has cash to continue through 2020; longer if milestone payments are received.

Conference Highlights:

Jackie Fouse, CEO of Agios said "As we look ahead to the remainder of 2019, we are focused on achieving our remaining key milestones, including completing enrollment in our mitapivat PK deficiency pivotal program, establishing proof of concept for mitapivat in thalassemia, initiating our pivotal trial of vorasidenib in low-grade glioma and submission of the Tibsovo supplemental new drug application for IDH1 mutant cholangiocarcinoma. These milestones are critical steps toward realizing the value-creation potential for both our oncology and rare genetic disease portfolios in 2020 and beyond." Will share more data at ASH in December. 90% of American AML physicians are now testing for IDH1 mutations.

EU application should get a CHMP opinion on Tibsovo in the second half of 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. Investigator-sponsored studies are testing tibsovo with various other agents for AML.

Tibsovo net sales were $17.4 million, up 27% sequentially from $13.7 million. $2.7 million of revenue in the quarter was from royalties for Idhifa from Celgene, flat sequentially from $2.7 million. $5.5 million was from collaborations.

Hopes to be able to treat all front-line AML patients with IDH1 mutation. Data so far are encouraging. Also resumed study of Myelodysplastic Syndromes.

Agios received FDA approval on May 2, 2019 for the supplemental new drug application (sNDA) for single agent Tibsovo for the treatment of patients with newly diagnosed AML with an IDH1 mutation who are not eligible for standard therapy. Updated data from Phase 1 studies of Tibsovo in newly diagnosed AML and First Data from Perioperative Study of Tibsovo and Vorasidenib were accepted for presentation at ASCO 2019.

Agios plans to submit an sNDA to the FDA for Tibsovo for second line or later IDH1 mutant cholangiocarcinoma by year-end 2019. The Phase 3 trial had positive results.

In Q3 2019 esults 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.

Plans to initiate a registration-enabling Phase 3 study of vorasidenib (AG-881) in low-grade glioma with an IDH1 mutation by year-end 2019. is currently working with regulators on the trial design. Phase 1 study for IDHm positive glioma should reported data at ASCO.

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. Celgene is collaborating on AG-270.

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

Will complete enrollment in two global pivotal Phase 3 trials for mitapivat (AG-348) in adults with pyruvate kinase (PK) deficiency by year-end 2019. Phase 2 positive data was published in Q3 2019 in the New England Journal of Medicine.

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

Phase 3 AGILE trial expects full enrollment in 2020 now that event free survival is primary endpoint, in combination with azacitidine for newly diagnosed AML patients with IDH1 mutation ineligible for intensive chemotherapy.

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

Cash (including equivalents & securities) ended at $541 million, down sequentially from $624 million. No debt.

Cost of Sales $0.3 million. GAAP operating expenses were $135 million, consisting of: $102 million for R&D and $33 million for G&A. Loss from operations was $109 million. Interest income was $3 million.

Q&A:

ASH presentations? Drive PK abstract gives sense of safety and durability of responses. Long term safety is really important for this disease. Activate trial would continue to validate that.

Thalessemia? A small Phase 2, open label trial, looking for a 1 gm. or higher increase in hemoglobin. We are testing to see if we can activate wild type PKR.

Idhifa, Bristol commitment to Celgene programs? We can't predict, we have a good partnership with Celgene. Idhifa combination trials are ongoing and could contribute to label expansion. We will have to wait to see what happens with Bristol.

Mitapivat patient types? We have been identifying patients for years now. It is a rare disease with no existing therapies, so id was low in the past. All patients with a diagnosis and anemia could potentially be treated. In our trials we picked patients with genotype profiles and a maximum low hemoglobin level. The picture is likely to evolve over time.

Tisovo trends in frontline AML, perhaps with HMAs? We have seen growth in both frontline and relapsed/ refractory, but we have a limited view of the type ratio. Roughly half of front-line use is with HMAs, though we only promote the monotherapy, per the label.

Low grade glioma patients tend to be younger, 40 to 50 years of age. Survival is typically 10 years or more. Following surgery there is gradual progression of disease despite radiation and chemotherapy. Other choice is watch and wait. A population could take a drug once a day, well-tolerated drug to stave off radiation and chemo. The intent is to delay progression. We will report control arm expectations at a later time.

AG-270 preclinical to highly pretreated patients? Really difficult. Intention is to get into earlier lines of patients, and we are sticking with that strategy.

Hovon study interim readout in 2020? None for 2020, conducted by international groups, working on getting sites up and run.

AG-270 combination dose? Both chemo agents we are trying have their own toxicity levels. We are starting with 100 mg daily. That dose reaches the SAM reduction target. But we could go up to 200 mg. We don't thing we need to do a lot of dose exploration.

GBM has a high unmet medical need, but we don't think that is where our drug is likely to work well.

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