conference date: May 5, 2016 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2016 (Q1, first quarter 2016)
Overview: Pipeline therapies continued to progress.
Basic data (GAAP):
Revenue was $31.3 million, sequentially from $6.2 million, and down from $34.2 million year-earlier. All revenue is from collaborations.
Net loss was $23.2 million, up sequentially from $40.6 million, and down from a $5.0 million loss year-earlier.
EPS (diluted GAAP) was negative $0.61, up sequentially from negative $1.08, and down from negative $0.13 year-earlier.
David Schenkein, M.D., CEO at Agios said "Enrollment is complete ahead of schedule for the Phase 2 expansion cohort for the Phase 1/2 study of AG-221 in advanced AML. This achievement is crucial to getting this potential new therapy to patients as quickly as possible. Additionally, we are pleased that the first data from our Phase 2 DRIVE PK study of AG-348 will be presented at EHA this June, along with the first data from the Phase 1 study of AG-519 in healthy volunteers."
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.
Celgene is collaborating on AG-221, AG-120, and AG-881.
Completed enrollment of both 125-patient expansion cohorts for the Phase 1/2 study of AG-221. Enrollment for Phase 1 study of AG-120 in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) should complete in the second half of 2016.
Will 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.
Will initiate an expansion arm in high-risk myelodysplastic syndrome patients for AG-221 in 2016.
Initiated 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 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. In the quarter received orphan drug designation in Europe for AML.
Presenting 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.
Initiating a randomized Phase 2 study of AG-120 in IDH1 mutant positive cholangiocarcinoma in the second half of 2016
Continued 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.
Presented preclinical findings on a new research program focused on MTAP (methylthioadenosine phosphorylase) deleted cancers at the Keystone Symposia, February 21-25, 2016.
Will 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 for PK deficiency 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.
Will 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 $54.9 million, consisting of: $44.0 million for R&D and $10.8 million for G&A. Interest income was $0.4 million.
Cash (including equivalents & securities) ended at $356 million, down $20 million sequentially from $376 million. No debt.
All development costs for AG-221 are paid by Celgene, and 50% for AG-120 and AG-881.
So more PK patient data at EHA than previously thought, and duration? Enrollment for Drive PK trial is open. Patients are at various stages, including an extension phase. So the data will cover various durations.
Options with Celgene, could we see more equity investment? This extension is wrapping up the 2010 research agreement. We will update you as soon as the decision is available.
AG221 AML cohort expansion, does "crucial" mean necessary and sufficient? Our goal is to do all the trials we need to quickly bring these molecules to market. All the data is important, including expansion cohorts.
We will do a press release on where there AHA abstracts will be present. What is in the abstract will be updated at the actual meeting.
We think that increased hemoglobin is one of the measures that are important in the DRIVE PK study. Patients feel better when their hemoglobin goes up.
We have not guided towards accelerated approval of AML. We have seen the data we have seen to date is compelling. Only one or two new drugs have been approved for AML in the past 20 or 30 years.
AG-221 AML faster than expected enrollment, does that indicate their are more IDH1 patients than previously thought? We don't have updated guidance on the prevalence of the mutation. But AG-120 is on track for the rest of the year.
We still have not made a commitment to start a beta-thalassemia trial, but will let you know before the end of the year.
Will you have mature OS data in AG-221 before talking to regulators? It takes some time to follow up to get overall survival.
We are not ready to state what our combination development strategy would be for AG-221, but there are some interesting molecules out there.
AG-120 frontline Phase 3, will patients be ineligible for induction? They can go on to transplant, but historically it is pretty rare. We have not announced a trial design yet.
Progression despite IDH1 level suppression? We did release data that 2HD was still suppressed despite progression. That shows the drug still found its target, rather than being evaded by the cancer. It opens the possibility of treating in multiple lines because you always want to suppress that clone. But we would need to prove that with clinical trials.
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