bluebird bio, Inc.

Good day and thank you for standing by. Welcome to the Bluebird Bio second quarter results and commercial launch progress call. At this time, all participants are in listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 11 on your telephone. You will then hear an automated message advising that your hand is raised. Please be advised that today's conference is being recorded. I would now like to hand the conference over to Ms. Courtney O'Leary, Investor Relations. Please go ahead.

Good morning, everyone, and thank you for joining today's call. I am Courtney O'Leary, Investor Relations at Bluebird Bio. Before I begin, let me review our Safe Harbor Statement. Today's discussion contains statements that are forward-looking under the Private Securities Litigation Reform Act of 1995 and including expectations regarding our future financial results and financial position, in addition to statements about the company's plans, expectations, or intentions regarding our business, regulatory progress, and commercialization plans. Such statements are based on current expectations and assumptions that are subject to risks and uncertainties and involve a number of risk factors that could cause actual results to differ materially from projected results. A description of these risks is contained in our most recent form 10-Q and our other filings with the SEC, which are available on the investor relations section of our website, www.bluebirdbio.com. With me on the call is Andrew Obenshame, our CEO, who is going to provide some opening remarks on Bluebird's strategic position in vision, and then review our Q2 results. Then Tom Klima, Chief Commercial and Operating Officer, will dive deeper into the positive momentum in our commercial launches and highlight the Lobosol market opportunity ahead of us. They will then be joined by Chris Krawchuk, Chief Financial Officer, and Rich Colvin, Chief Medical Officer, for Q&A. With that, I will turn the call over to Andrew.

Thanks, Courtney, and good morning, everyone. I want to ground our call this morning on why we're here. A few weeks ago, we heard from a young adult with sickle cell disease who was treated in our HPV 206 study. This was a real privilege. Reflecting on life since gene therapy, she shared, it's amazing. My mind is open to the world of possibilities. So much is in front of me that I want to accomplish. There's so much more for me than what my previous medical teams had envisioned for me themselves. I'm excited about it. And this is why we're here and what we mean when we talk about bringing patients and families more Bluebird days. Over the past decade, Bluebird has established ourselves as a gene therapy leader. proving our clinical, regulatory, and commercial capabilities. Clinically, we have the longest and most robust gene therapy program in the field, with over 180 patients treated across eight clinical trials with up to nine years of follow-up and over a decade of gene therapy research. We have an established regulatory track record with two FDA gene therapies on the market and a third BLA currently under priority review. And now we are making strides commercially. actively launching, achieving reimbursement, and treating patients with two therapies today. We occupy a unique strategic position in the gene and cell therapy industry. There are only a small handful of companies, all large cap, that currently have the commercial and cell gene capabilities that we do. On the opposite side of the spectrum, there are more than 250 companies studying gene therapies preclinically or clinically who lack a gene therapy infrastructure. with new companies being formed every month. We believe that with our unique capabilities, Bluebird is in an attractive strategic position as one of the only standalone commercial gene and cell therapy companies. Our extensive platform of gene therapy expertise includes manufacturing experience, particularly with viral production and cell processing, more than a decade of R&D experience, and an established commercial infrastructure. Our strategic vision for the future is clear, to evolve into an industry-leading gene therapy company with opportunity for expansion and growth for years to come. We have put in place the pieces that we need to be a successful biotechnology company. We are on a path to profitability in the near term and anticipate additional growth and scale within the next five years. We conservatively estimate a combined multi-billion dollar revenue opportunity in the U.S. for Zynteglo and LovaCell, if approved. We have a focused commercial footprint with the ability to cover U.S. transplant centers with a relatively modest but effective field presence. At scale, we estimate a gross margin of at least 70% as we invest in manufacturing quality capacity. And my comments pertain only to the U.S. There's also significant upside potential with Holio and Global Rights for all three therapies, particularly for Zanteglo and Logosol. I will move now to how our business is performing today and the updates from the second quarter. I will summarize the highlights and additional details can be found in our Q2 release this morning and in our 10Q. First, it's incredibly exciting to be here, almost a year after the FDA approvals of Zanteglo and SkySona. The initial traction we are seeing in these launches is a testament to the robust commercial viability of our platform. For Zanteglo and SkySona, we have 16 patient starts to date. Assuming all of these collections result in infusions, this translates to a potential $45 million in gross revenue over time. We see that continue to accelerate quarter over quarter. Our therapies are being covered by insurance, and there have been no ultimate denials from commercial or government payers for either therapy. And we've activated 15 QTCs, Qualified Treatment Centers, which is sufficient for the SkySona and Zantegla launches, But we anticipate an aggressive ramp up to 40 to 50 QTCs by year end to support the potential launch of LovaCell next year. Looking ahead to LovaCell for sickle cell disease, we have the opportunity to make a tremendous difference to change the lives of the more than 20,000 individuals living with severe sickle cell disease in the U.S. that may be appropriate for gene therapy. In June, our VLA was accepted and granted priority review by the FDA for the treatments of patients 12 and over with a history of vaso-occlusive events, or VOEs. At this time, the FDA has not requested an advisory committee meeting. Keep in mind, this is the third LVV gene therapy they've reviewed from Bluebird, which gives us great confidence. And we look forward to a decision from the FDA by the end of this year for the Purdue for date of December 20th, and anticipated commercial launch in early 2024. Additionally, the value of our therapy and need for rapid, equitable access for patients is already being recognized. Just a few weeks ago, ICER reported that LogoCell is cost-effective up to a price of $2.26 million. In fact, ICER rated LogoCell's clinical effectiveness more favorably than direct competitors when compared to current standard of care. And financially, we are confident in our lean and efficient business model. We are focused on one mission and have the expertise and agility to deliver for patients and our business. For the quarter, we reported $6.8 million combined product revenue for Centeglo and SkySona. As of June 30th, we had $291 million in cash, cash equivalents, marketable securities, and restricted cash. We remain on track with our full year 2023 cash burn guidance in the range of $270 to $300 million and continue to prudently deploy capital and make investments to maximize stakeholder value as we launch into Teglo and SkySona and prepare for the launch of LogoSell. We continue to estimate that we have a cash runway into the fourth quarter of 2024, including the restricted cash. As previously disclosed, this estimate of cash runway includes approximately $45 million of restricted cash, which is currently unavailable for use. Without the release of our restricted cash, our runway is in the second quarter of 2024. Now, I would like to turn the call over to Tom to dive deeper into our commercial launch progress and momentum in greater detail.