Analyst Conference Summary

biotechnology

conference date: February 14, 2019, @ 5:00 AM Pacific Time
for quarter ending: December 30, 2018 (Q4, fourth quarter 2018)

Forward-looking statements

Overview: Significant Tibsovo revenue, but not clear if it will be a blockbuster. New CEO. Plenty of cash.

Basic data (GAAP):

Revenue was $30.0 million, up 97% sequentially from $15.2 million, and up 206% from $9.8 million year-earlier.

Net income was negative $91.8 million, up sequentially from negative $94.7 million, and down from negative $88.3 million year-earlier.

EPS (diluted GAAP) was negative $1.58, up sequentially from negative $1.63, and down from negative $1.81 year-earlier.

Guidance:

Cash should last until at least the end of 2020.

Conference Highlights:

Jackie Fouse, CEO of Agios said "I’m excited to join the Agios team on the heels of a transformational year for the company. During 2018, we launched our first wholly owned oncology medicine, expanded our clinical programs across both oncology and rare genetic diseases, and continued to advance our robust research pipeline. We start 2019 with a strong foundation on which to build and with the opportunity to make a meaningful impact on patients’ lives and our business. Our objectives for this year focus on broadening the potential of our IDH inhibitors in AML and solid tumors, advancing mitapivat and AG-270 through clinical development, and remaining steadfast in our pursuit of great science." New positive data was presented at ASH.

Effective February 1, 2019, CEO David Schenkein, M.D., transitioned to the role of executive chairman of the board of directors and Jacqualyn Fouse, Ph.D., will succeed Dr. Schenkein as CEO.

Tibsovo (AG-120 or ivosidenib) in R/R AML patients with an IDH1 mutation received FDA approval in July 2018. An EMA submission should be made before the end of 2018. Executing on launch plan. 60% of payments were from Medicare. Submitted a supplemental new drug application (sNDA) for single agent Tibsovo in newly diagnosed AML patients with an IDH1 mutation who are not eligible for standard treatment in

Tibsovo net sales were $9.4 million, up sequentially from $4.5 million. $2.2 million of revenue in the quarter was from royalties for Idhifa from Celgene, up sequentially from $2.0 million. $18.4 million was from collaborations.

The next steps for the expansion of Tibovo revenue are EU approval (submitted end of 2018) and expansion to the front line setting for AML. Hopes to be able to treat all front-line AML patients with IDH1 mutation. Data so far are encouraging.

There are a large number of key pipeline milestones that can be achieved in 2019.

Potential 2019 FDA approval of 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 and subsequent launch in this indication in the U.S.

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 has completed enrollment.

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.

By end of 2019 hopes to determine the recommended dose of AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in methylthioadenosine phosphorylase (MTAP)-deleted tumors; initiate expansion arms, including a single-agent arm in a variety of MTAP-deleted cancers and a combination arm in a solid tumor in the first half of 2019. Celgene is collaborating on AG-270.

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

Will complete enrollment in two global pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency by year-end 2019.

Achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019.

With Celgene, Agios plans to start a Phase 3 trial for frontline AML combining ivosidenib or enasidenib with 7+3 chemotherapy. Updated Phase 1/2 data from the trial combining Tibsovo or Idhifa with Vidaza was presented at ASCO.

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.

A randomized Phase 3 study (ClarlDHy) of AG-120 (ivosidenib) in IDH1 mutant positive cholangiocarcinoma in continued enrollment, which is expected to complete in first half of 2019. If approved would be the first targeted therapy for this disease, which has about 3,000 IDH1+ patients annually.

Updated MDS data for single agent ivodienib was presented in a poster at ASH. Further combination trials with a variety of agents and target variants are planned for ivosidenib.

Cash (including equivalents & securities) ended at $805 million, down sequentially from $878 million. No debt.

Cost of Sales $0.7 million. GAAP operating expenses were $126 million, consisting of: $94 million for R&D and $32 million for G&A. Loss from operations was $96 million. Interest income was $5 million.

Q&A:

FDA warnings on differention syndromes? The data match our Tibsovo label, so no impact. It is an important adverse event associated withe Tibsovo, so we are

Cholangia Tibsovo PFS design? 96% power to detect a hazard ratio of 0.5. Our control arm PFS assumption is 2 to 3 months.

Salesforce in EU? In position of strength. We think with the growing portfolio the best route is to have our own salesforce in some EU nations.

Idhifa duration? Just over 4 months. Too early to say for Tibsovo, but should be similar to Idhiba.

Does cash provide for commercialization in Europe? We are carefully gating the plans in Europe. The guidance to 2020 for cash includes the EU build.

Munich data this month? Tibsovo plus a azacitidine Phase 1/2 will have a longer follow up, so mainly duration.

There are over 300 mutations in PK, we are looking at broader categories for mitapivat. 80% have at least one mis-sense mutation, so they would be the target population.

OpenIcon Analyst Conference Summaries Main Page

Search

More Analyst Conference Pages: