conference date: February 18, 2016 @ 5:30 AM Pacific Time
for quarter ending: December 31, 2015 (fourth quarter 2015)
Overview: Pipeline therapies continued to progress.
Basic data (GAAP):
Revenue was $6.2 million, down from $14.6 million year-earlier. All revenue is from collaborations.
Net loss was $40.6 million, worse than the $26.7 million loss year-earlier.
EPS negative $1.08, down from negative $0.76 year-earlier.
Agios expects to end 2016 with more than $180 million of cash, cash equivalents and marketable securities. The anticipated year-end 2016 cash position does not include any additional program-specific milestone payments. The company expects that its cash, cash equivalents and marketable securities would be sufficient to fund its operating expenses and capital expenditure requirements until late 2017.
In January 2016, Agios received a $25 million milestone payment from Celgene (CELG) for achievement of the first patient dosed in the Phase 3 IDHENTIFY study of AG-221 vs. standard of care chemotherapy in R/R AML that is IDH2 mutant-positive.
In Q4 new data from the dose-escalation phase and expansion cohorts from the ongoing Phase 1/2 study for AG-221 and the Phase 1 study of AG-120 were presented in December at the 2015 American Society of Hematology Annual Meeting (ASH).
In December, Agios initiated a Phase 1b, multicenter, international, open-label study of AG-221 or AG-120 in combination with induction and consolidation therapy in patients with newly diagnosed AML with an IDH mutation who are eligible for intensive chemotherapy.
Data from the ongoing Phase 1 dose-escalation trial of AG-10 in advanced IDH1-mutant positive solid tumors were presented in an oral presentation at the International Conference on Molecular Targets and Cancer Therapeutics in November.
Expected 2016 Milestones:
Celgene is collaborating on AG-221, AG-120, and AG-881.
Complete enrollment of both 125-patient expansion cohorts for the Phase 1/2 study of AG-221 and Phase 1 study of AG-120 in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) in the second half of 2016.
Initiate a global, registration-enabling Phase 3 study of AG-120 in frontline AML patients with an IDH1 mutation in the second half of 2016.
Initiate an expansion arm in high-risk myelodysplastic syndrome patients for AG-221 in 2016.
Initiate 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, in the first quarter of 2016.
Continue 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.
Present data from the expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive low grade glioma in the second half of 2016.
Initiate a randomized Phase 2 study of AG-120 in IDH1 mutant positive cholangiocarcinoma in the second half of 2016
Continue to enroll patients in the following ongoing clinical trials: Expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive solid tumors; and Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive solid tumors.
Present preclinical findings on a new research program focused on MTAP (methylthioadenosine phosphorylase) deleted cancers at the Keystone Symposia, February 21-25, 2016
Initiate preclinical development activities for the first molecule in the next wave of novel investigational cancer metabolism medicines.
Plans to submit the first data from DRIVE PK, a global Phase 2, open-label safety and efficacy trial of AG-348 in adult, transfusion-independent patients with pyruvate kinase (PK) deficiency, for presentation at the EHA in June 2016.
Plans to submit the first data from the Phase 1 study of AG-519 in healthy volunteers for presentation at EHA in June 2016. Preclinical findings about the molecule will also be submitted for presentation at EHA.
Agios will outline the clinical development plans for Agios PKR activators in beta-thalassemia in the second half of 2016.
Present the new findings from the Natural History Study of PK deficiency being conducted with Boston Children’s Hospital in the second half of 2016.
GAAP operating expenses were $47.1 million, consisting of: $36.9 million for R&D and $10.2 million for G&A. Interest income was $0.3 million.
Cash (including equivalents & securities) ended at $376 million. No debt.
All development costs for AG-221 are paid by Celgene, and 50% for AG-120 and AG-881.
Drive PK study early data? The study is open and continuing to enroll. The patients in the early data will have varying follow ups, and we will present hematological parameters.
AG-120 frontline trial decision? We considered a range of options for 120 and AG-221. 2nd and 3rd line patients have high unmet medical need. For AG-120 we believe first line patients are the way to go. We have not released details on the trial yet.
$50 million late-stage milestones for AG-120, pivotal trial milestone? $25 milestone for start of Phase 3. $25 million for last patient enrolled in expansion cohort. Both could come this year. Neither of these payments is included in the year-end cash guidance.
IDH inhibitors with cytotoxic agents in AML? Those studies started late in 2015, and the Vidaza study will start in Q1. We don't have a timeline for releasing data yet. We have no anticipated issues around combinations.
AG-221 or 881 milestones? Yes, for 221 there are up to $120 in milestones, starting with $25 million for Phase 3 initiation. $70 million in milestones are related to approvals and regulatory. The last $25 million is related to commercialization. For AG-881 there are $70 million in milestones, mainly for approval and regulatory. Altogether the three programs should provide a good source of capital over the next few years.
AG-519 plans? Studies are ongoing, but we hope to have data for EHA. We have not yet committed to moving forward, but we should have the data for the decision in 2016.
Differential activity across the many mutations involved in PKD? We want to have as much genotype data on the patients as we can. We want to see how outcomes correlate with mutations. In preclinical we concentrated on the most common types.
AG-120 low grade glioma study intent? We want to expand on some of the earlier findings like improvements in tumor volume. The expansion cohort eligibility criteria are to look deeper into the earlier findings.
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