Orchard Therapeutics (NASDAQ:ORTX) Q1 2019 Earnings Conference Call Transcript

May 28, 2019 • 08:00 am ET


Orchard Therapeutics (NASDAQ:ORTX) Q1 2019 Earnings Conference Call Transcript


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Good day, ladies and gentlemen, and welcome to the Orchard Therapeutics First Quarter 2019 Earnings Call. At this time all participants are in a listen-only mode. Later we will conduct a Question-and-Answer session and instructions will be give at that time. (Operator Instructions). And as a reminder, today's conference call is being recorded.I'd now like to turn the conference over to Renee Leck, Director of Investor Relations. Please go ahead.

Renee Leck

Thanks, operator. Good morning, everyone, and welcome to Orchard's first quarter 2019 investor update. You can access slides for today's call by going to the investors section of our website, orchardtx.com.

Before we get started, I'd like to remind everyone that statements we make on this call will include forward-looking statements. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risk factors and uncertainties, including those set forth in the most recent Form 20-F filed with the SEC, and any other filings that we may make.

In addition, any forward-looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements.

With that, I'll turn the call over to President and CEO, Mark Rothera.

Mark Rothera

Thanks, Renee. Welcome, everyone, and I hope many of you enjoyed a long holiday weekend. On today's call, we plan to update you on the progress we have made over the last quarter and discuss this morning's exciting announcements related to the expansion of our neurometabolic disease portfolio with a new clinical-stage program.

Before we dive into the recent news, I'll spend just a few minutes reminding you about Orchard's vision and our scientific platform. We are a pure-play gene therapy company with a focused approach and singular vision, transforming the lives of patients with life-threatening diseases around the world. Our expertise lies in developing ex vivo autologous hematopoietic stem cell, or HSC, gene therapies with a focus today on rare genetic diseases. a platform technology that has been clinically validated. Recall that we now have clinical proof of concept in five programs in five different disease areas across three different physiological systems.

The majority of our remarks today will focus on our neurometabolic franchise because of this morning's news, but we also have programs in primary immune deficiencies and hemoglobinopathies.

In the primary immune deficiencies area, we are preparing regulatory filings in two diseases, ADA-SCID with an FDA filing anticipated in 2020 and Wiskott-Aldrich syndrome, or WAS, with FDA and EMA filings anticipated in 2021. In addition, we declared clinical proof of concept in X-CGD at ASH in December last year and are making good progress in designing the registration trial and end points that we would like to discuss with regulators in the coming quarters.

Our program in transfusion-dependent, beta-thalassemia also recently achieved clinical proof of concept, and as next steps we intend to design