conference date: February 16, 2017 @ 5:00 AM Pacific Time
for quarter ending: December 31, 2016 (Q4, fourth quarter 2016)
Overview: Agios hopes to graduate from a clinical-stage to a commercial-stage company in 2017.
Basic data (GAAP):
Revenue was $22.6 million, up sequentially from $9.0 million, and up from $6.2 million year-earlier. All revenue is from collaborations.
Net loss was $56.5 million, sequentially from $62.8 million, and from a $40.9 million loss year-earlier.
EPS (diluted GAAP) was negative $1.34, up sequentially from negative $1.63, and down from negative $1.08 year-earlier.
Cash and expected milestone payments are sufficient to fund programs through at least the end of 2018.
David Schenkein, M.D., CEO at Agios said “In 2017, we are focused on making the transition to a commercial stage company by delivering our lead cancer programs to patients, bringing our first rare genetic disease program into pivotal development and advancing our next research program, focused on MTAP-deleted cancers, into the clinic.”
"2017 is set up to be an extraordinary year for Agios."
AG-221 (Enasidenib) NDA was submitted in December to the FDA in IDH2m positive relapsed/refractory AML (acute myeloid leukemia) . It could be approved for sale by the FDA before the end of 2017. This is in partnership with Celgene and is based on Phase 1/2 data. The Phase 3 trial continues. An MDS trial is also planned.
AG-120 (ivosidenib) 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 was presented at ASH showing impressive activity. The R/R AML expansion cohort should complete enrollment this year. A Phase 3 frontline AML study, in combination with Vidaza, for patients with IDH1 mutation ineligible for intensive chemotherapy is planned for first half of 2017.
Data was presented on November 10 at CTOS for AG-120 for IDH1 mutant chondrosarcoma. Expansion phase, positive data for low-grade IDH1 mutant glioma data was presented on November 18. Updated data will be presented in the second half of 2017.
Initiated a randomized Phase 3 study (ClarlDHy) of AG-120 in IDH1 mutant positive cholangiocarcinoma in December. Data from the Phase 1 expansion cohort to be presented in the first half of 2017. Fast track status was granted.
Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive hematologic malignancies completed the dose-escalation phase. AG-881 data was presented at ASH showing proof of mechanism. Another Phase 1 study for IDHm positive glioma should complete its dose escalation phase in the first half half of 2017.
Phase 1 study of AG-519 for PK deficiency in healthy volunteers enrollment continues. Preliminary data was presented at ASH.
Agios will outline the clinical development plans for Agios PKR activators in beta-thalassemia in 2016. Preclinical data was presented at ASH.
AG-348 data for Pyruvate Kinase Deficiency (PKD) hemoglobin levels was presented at ASH, showing compelling hemoglobin increases in about half of patients. A global pivotal trial is planned for the first half of 2018. Updated DRIVE PK study data is expected in first half of 2017.
Preclinical activities for MTAP (methylthioadenosine phosphorylase) deleted cancers will result in an IND submission to the FDA before the end of 2017. Updated data will be presented in March. 15% of all cancers have MTAP deletions.
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 $573.6 million, down sequentially from $622.6 million. No debt.
GAAP operating expenses were $80 million, consisting of: $65 million for R&D and $15 million for G&A. Loss from operations was $57 million. Interest income was $1 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.
AG-881 dose escalation, implied Phase 2 decision? We want to identify a recommended Phase 2 dose. AG-120 is ahead in glioma, but that should help us. We would have to work with Celgene on next steps. We will release data when we have a dose schedule.
AG-348 Drive PK optional third arm? We don't have plans to do a third arm. We feel the data we have is ample to do a forward plan.
AG-348 trial design requirements? We have a conceptual phase, then a presentation to regulators, which then may require some changes. We need to pull plans and protocols together, like patient population and sample sizes.
AG-221 filed for breakthrough designation? We don't share that level of detail of FDA discussions, but had announced orphan and fast track designations.
AG-221 data release? With Celgene the Enasidenib package was submitted in the fourth quarter. This quarter we should hear whether they accept the package, and a PDUFA date. We will update you when appropriate.
AG-881 as a backup molecule in hematological malignancies? 881 was developed as a backup for 120 with better brain penetration. It would only be tried in the heme space if 120 failed.
AG-120, what endpoint do you think the FDA will be focusing on? They will be interested in all of the endpoints mentioned and then some. Relapsed/Refractory AML patients with no treatment options, so response rates and durations, plus overall survival. Plus safety over time.
The lower does in 348 tend to be given because the hemoglogin response is so strong, patients have to be brought down to a normal range. For pediatrics, we need to complete the adult studies first, but we do plan to get to children.
We have a revenue recognition model for MTAP, so while there is a $8 million Celgene milestone if they pick up the molecule, you won't see a revenue jump.
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