conference date: November 3, 2016 @ 5:00 AM Pacific Time
for quarter ending: September 30, 2016 (Q3, third quarter 2016)
Overview: Seven abstracts accepted for ASH, pipeline looking good.
Basic data (GAAP):
Revenue was $9.0 million, up sequentially from $7.0 million, and up from $5.5 million year-earlier. All revenue is from collaborations.
Net loss was $62.8 million, down sequentially from $56.0 million, and down from a $40.3 million loss year-earlier.
EPS (diluted GAAP) was negative $1.63, up sequentially from negative $1.47, and down from negative $1.07 year-earlier.
Expects to end the year with over $550 million in cash. That should last through 2018.
David Schenkein, M.D., CEO at Agios said “As we head into year end, we are focused on supporting the enasidenib NDA submission with our partner Celgene and planning for our next steps in clinical development for our PKR and IDH portfolios based on data we will present at a number of upcoming medical meetings this quarter.”
Raised $173 million selling common stock in September.
Seven abstracts accepted for ASH (American Society of Hematology) meeting in December. Three are oral presentations.
AG-221 (Enasidenib) is on track for an NDA to the FDA in IDH2m positive relapsed/refractory AML (acute myeloid leukemia) by year end. This is in partnership with Celgene and is based on Phase 1/2 data. The Phase 3 trial continues. [WPM: that is a significant acceleration of the timeline for 221]. An MDS trial is also planned.
AG-120 in frontline AML patients with an IDH1 mutation could be submitted to the FDA for approval in 2017 based on Phase 1/2 data. Data will be presented at ASH showing impressive activity. The R/R AML expansion cohort should complete enrollment this year. A Phase 3 frontline AML study is planned for 2017.
Data will be presented on November 10 at CTOS for AG-120 for IDH1 mutant chondrosarcoma. Expansion phase data for low-grade IDH1 mutant glioma data will be presented on November 18.
Initiated a randomized Phase 2 study of AG-120 in IDH1 mutant positive cholangiocarcinoma.
Will initiate an expansion arm in high-risk myelodysplastic syndrome patients for AG-221 in 2016.
A Phase 1/2 frontline combination study of AG-221 or AG-120 with Vidaza (azacitidine) in newly diagnosed AML patients not eligible for intensive chemotherapy continued.
Continued to enroll patients in the following ongoing clinical trials: Phase 3 IDHENTIFY study of AG-221 vs. standard of care chemotherapy in R/R AML; Phase 1b frontline combination study of AG-221 or AG-120 with standard-of-care intensive chemotherapy in AML.
Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive hematologic malignancies continued enrollment. AG-881 data will be presented at ASH.
Phase 1 study of AG-519 for PK deficiency in healthy volunteers enrollment continues. Preliminary data to be presented at ASH.
Agios will outline the clinical development plans for Agios PKR activators in beta-thalassemia in 2016. Preclinical data will be presented at ASH.
AG-348 data for Pyruvate Kinase Deficiency (PKD) hemoglobin levels will be presented at ASH.
Preclinical activities for MTAP (methylthioadenosine phosphorylase) deleted cancers will begin in 2016.
Will initiate preclinical development activities for the first molecule in the next wave of novel investigational cancer metabolism medicines.
Cash (including equivalents & securities) ended at $622.6 million, up sequentially from $512.3 million. Raised $173 million ($162 million net) selling common stock. No debt.
GAAP operating expenses were $72.5 million, consisting of: $60.6 million for R&D and $11.9 million for G&A. Interest income was $0.7 million.
All development costs for AG-221 are paid by Celgene, and 50% for AG-881. But Agios is responsible for 100% of development expenses for AG-120.
PKR activator program selection? We are on track to get AG-120 and PKR molecule selection and pivotal trial design, but the regulatory meetings could be late in December, and we would communicate the decisions early in 2017.
Milestone payment with Celgene NDA on 221? No, the milestones are based on ex-U.S. activities.
AG-120 chondrosarcoma data to be presented? Safety and efficacy in the cohorts for dose escalation and expansion. We are discussing the next steps internally and with investigators.
Decision not to present updated 221 data before the filing? Celgene and Agios are aligned in not wanting to put data in the public domain before the regulators have a chance to vet it.
Combination trial timelines? The trials are ongoing, when we have an adequate data set we will publish the data. We can't give timeline guidance yet, but the trials are accruing well.
Can one determine PKD disease severity on the basis of genotype? We see people with identical genotypes having different disease severity, but we will learn more as data becomes available. Our goal is to treat all patients.
MTAP opportunity, targets? It is an exciting program. This deletion is present in about 15% of all human tumors, varying by tumor type. We are focussed on getting a molecule into preclinical development. There are multiple possible targets.
519 thrombocytopenia? Literature indicates there are multiple mechanisms causing this. We expect the effect to be rare. PKD patients have blood monitored regularly, so it could be monitored. With other drugs it is rare and when you stop dosing it goes away.
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