Analyst Conference Summary

biotechnology

conference date: May 8, 2017 press release; May 18 conference at 5:30 A.M. Pacific time
for quarter ending: March 31, 2017 (first quarter 2017, Q1)

Forward-looking statements

Overview: The conference date is different from the press release of Q1 results in order to present drug data.

Basic data (GAAP):

Revenue was $0.0 million, down sequentially from $0.5 million, and down from $0.5 million in the year-earlier quarter.

Net loss was $32.5 million, up sequentially from a loss of $35.9 million, and down from a $23.1 million loss year-earlier.

EPS was negative $0.56, up sequentially from negative $0.60, and down from negative $0.41 year-earlier.

Guidance:

Conference Highlights:

Robert Bazemore, president and chief executive officer. "We have continued to advance tazemetostat in multiple clinical trials in a range of solid tumors and hematological malignancies, and as both a monotherapy and in combination with other anti-cancer agents. We look forward to reporting interim data from our Phase 2 study in molecularly defined solid tumors in our conference call next week and from our Phase 2 study in relapsed or refractory FL and DLBCL in June."

On May 4 Epizyme announced it had earned a $10 million milestone payment from GlaxoSmithKline for a novel methyltransferase inhibitor initiation of toxicology studies.

Epizyme has a global development plan for tazemetostat (an EZH2 inhibitor), which includes a phase 2 study in patients with NHL which has completed enrolling three of five cohorts. Interim data from all 5 cohorts will be presented at ICML on June 14. Tazemetostat in combination with prednisolone in relapsed/refractory DLBCL, is being added as the sixth cohort of the Phase 2 NHL study. Also added a follicular lymphoma cohort in January.

Tazemetostat is also in a three-arm phase 2 study in adult patients with certain genetically defined solid tumors which initiated dosing in the quarter, and a dose-escalation and dose-expansion phase 1 study in pediatric patients with certain genetically defined solid tumors.

Data was presented at ASCO. Tazemetostat is a first-in-class orally administered EZH2 inhibitor. The interim data is from the Phase 2 trial for INI1-negative epithelioid sarcoma. The regulatory meeting for this indication was held and a registration path for accelerated approval was determined. Three other arms of the trial, for different solid cancers.

For epithelioid sarcoma, which is rare and aggressive and characterized by the loss of INI1 protein, the results were positive. 31 patients were in the study group, ranging in stage of the disease and in number of lines of prior therapy. Current treatment has limited benefit. 13% of patients had objective responses (2 first line, 2 5th line) and 19% had stable disease, but there were no complete responses. So 32% had disease control, the primary endpoint of the study. Median progression free survival has reached 5.7 months.

Two patients from the Phase 1 trial have gone without disease progression for over two years.

Safety in the trial was about the usual: lots of low-grade adverse events, only a few grade three or greater, and those appeared to be related to disease progression.

Epizyme plans to meet with regulatory authorities, beginning with the FDA in mid-2017, to review data to be presented at a meeting in June and to discuss registration strategies based on the NHL and solid tumor studies. Received Fast Track designation for DLBCL with EZH2 activating mutations.

Tazemetostat enrollment completed in the Company's Phase 2 clinical study in adult patients and with INI-1 negative synovial sarcoma. Will not be continuing with synovial sarcoma due to poor results, at least as a monotherapy, though it did pass futility and had 30% of patients with stable disease.

Phase 1/2 study in pediatric patients with solid tumors is expected to reach recommended dosing in 2017. Objective responses have been observed for patients with INI1-negative tumors.

A Phase 2 trial for tazemetostat for mesothelioma characterized by BAP1 loss-of-function started in August and is expected to complete enrollment in 2017, with preliminary data in 2018.

A Phase 1b study of tazemetostat with Tecentriq for patients with with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) was continuing by Genentech/Roche.

Epizyme continued clinical evaluations of tazemetostat as a combination therapy, including a phase 1/2 study with R-CHOP in front-line high-risk patients with diffuse large B-cell lymphoma in collaboration with the Lymphoma Study Association in France.

Two new combination studies with tazemetostat will be initiated in 2017.

A dose-escalation study of pinometostat in pediatric patients with MLL-r acute leukemia is continuing enrollment. Epizyme is partnering with Celgene for potential clinical development of pinometostat in combination with other agents.

Epizyme has preclinical work underway with five new targets that are planned for introduction into the clinic by 2020.

Three programs are partnered with GSK and three with Celgene.

Cash and equivalents ended at $211 million, down sequentially from $242 million. Epizyme believes it has sufficient funding though at least the third quarter of 2018. Longer if milestone payments are received or new partnership revenue comes in.

Operating expenses of $33.0 million consisted of $24.7 million for R&D and $8.3 million for general and administrative. Loss from operations was $33.0 million. Other income was $0.4 million.

Q&A:

NDA submission in 2018? Acceptance of the NDA is up to the FDA. Based on the pace in the study, we believe we can submit in 2018. We are already seeing responses in pediatric and mesothelioma; we would like to see the broadest initial label possible.

Response rates influenced by number of prior therapies? For ES there is not a lot of prior data. So far, the number of prior therapies does not seem to matter. Epithelioid sarcoma does not respond well to existing therapies. That might not be the case for other types of cancer we test for further down the line.

The two patients with long-term survival had had disease progression despite other therapies prior to coming on trial.

Bar for approval? There are not any other therapies approved specifically for epithelioid sarcoma. The FDA wants to see a clinically-meaningful benefit, and durability, and totality of evidence. We believe we will be able to show an impressive totality of evidence. If we get accelerated approval we would need a confirmatory trial and are working towards the protocol.

Natural history, were all patients progressing rapidly on study entry? The vast majority had progression before coming on therapy. Unfortunately there is not a lot of natural history data. Local control is usually surgical. Some patients in our study presented with metastatic disease, which is far more aggressive. Seems to be 10 to 12 months to death for those patients.

Incidence and prevalence? 100 new cases per year? Our belief is that it is under-diagnosed. Molecular characterization by INI1 loss is recent. May be similar to "gist" sarcoma. So likely higher than 1% of 12,000.

Proxies for this type of data getting FDA approval? No high response rates for this disease, cited one drug for approval with a 4% response rate. In other words, varies by the disease.

30% disease control rate hurdle for other diseases? It does not translate to NHL, which has different expectation. Epithelioid vs. synovial, we had much more literature for synovial. It was for 12 weeks for stable disease. For epithelioid we used an 8-month hurdle and many patients remained controlled beyond that.

It is too early to estimate a launch date based on accelerated approval, but NDA should be submitted in 2018.

Do you think this will be a frontline therapy? Some of the patients in the trial were frontline. It will be up to the FDA. It would be our intent to get the frontline label.

EU? We have patients from the EU. We will be looking towards Europe.

OpenIcon Analyst Conference Summaries Main Page

Search

More Analyst Conference Pages: