Global Blood Therapeutics, Inc.

related costs and operational growth, partially offset by a decrease in non-cash stock compensation expense. Both R&D and SG&A expenses were slightly below our expectations in the first quarter, primarily due to the timing of certain expenses that we now expect to be recognized later in 2022. Net loss for the first quarter was $81 million compared to $75 million for the same period in 2021. Basic and diluted net loss per share for the first quarter was $1.26 per share compared with $1.21 per share for the same period in 2021. We ended the first quarter with cash, cash equivalents, and marketable securities of $662 million compared with $735 million at the end of 2021. Looking forward to the second quarter of 2022, we anticipate revenues in the range of $63 to $65 million. or sequential growth of 14% to 18%. We expect this will be driven by new prescription growth, including phasing in the first quarter, which was more back-end weighted in terms of growth of new prescriptions. And we expect this will be partially offset by an increase in growth to net and the timing of broad pediatric payer coverage. In summary, our first quarter results were in line with our expectations. and we expect that revenue will grow over the remainder of the year. In addition, we continue to be well-positioned with a strong balance sheet, allowing us to continue to make key investments for future growth. And with that, I will now turn the call over to DJ.

Thank you, Jeff, and good afternoon, everyone. As I've done in the past, I will provide an update on three key metrics that will give you further insight into our progress. These metrics are new prescriptions for Oxbrita, which informs underlying patient demand, the number of healthcare providers prescribing Oxbrita, which captures the progress we are making on adoption, and payer coverage, which speaks to the access environment for Oxbrita. First, new prescriptions. We delivered more than 1,200 new prescriptions during the quarter, our strongest demand quarter since Q1 of 2020. This was driven by the pediatric launch and incremental growth from the 12 and older group. Starting with the pediatric launch, we got off to a strong start in the quarter. We were able to leverage two years of Oxbrita education and awareness building from our original approval to quickly engage with healthcare providers on the pediatric label expansion and to train them on administering the new formulation in early January. Our team did an excellent job building excitement, particularly among pediatric hematologists and caregivers, and early feedback from this engagement has been overwhelmingly positive. This is reflected in the high number of new prescriptions in the quarter. Somewhat expected, the uptake in the pediatric population was strongest in the early part of the quarter when many physicians and patients had been eagerly awaiting the opportunity to start oxbritotherapy. As a result, many existing prescribers had some of their younger patients ready to initiate therapy shortly after approval. In terms of COVID-19, we did see some impact from Omicron in January, reflecting continued caution by SCD patients and caregivers. However, demand and field team access dynamics did improve in February as infections declined. Similarly, our market research shows that comfort levels for in-person care bounced back quickly in February, with most patients and caregivers preferring in-person visits for routine care. And in February, HCPs we surveyed indicated that the majority of their SED appointments were conducted in person. This is a good leading indicator that things are likely to start trending back to more normal healthcare engagements this year. Feedback from our field team supported what we heard from our market research. Anecdotally, our team saw patient volumes increase in February and March, with ongoing headwinds from COVID causing some adult patients to be cautious due to the increased risk to their health. On the provider side, we continue to see office turnover and staff shortages. And when we look at the claims data in Q1, SCD patient visits to HCP offices remain below pre-pandemic levels. We believe the net effect of all these trends contributed to the sequential growth in new prescriptions for the 12 and older age group, along with the momentum from our DTC and real-world evidence that was presented at ASH last year and recently published in expert review of hematology. We also believe the 12 and older segment benefited from HCP engagement around the pediatric launch, given that the majority of our targets treat a range of age groups. We also continue to see a broad range of patient characteristics for those prescribed Oxprida, such as baseline hemoglobin and VOC burden, suggesting that the prescribers are increasingly recognizing the importance of addressing polymerization and long-term health. The COVID environment has remained relatively stable from mid-Q1 through April, though we are closely watching the recent uptick in cases. Assuming COVID impact does not escalate, we anticipate that new prescriptions in the second quarter will be roughly flat compared to the first quarter. This reflects slightly lower pediatric new prescriptions following the strong start with patients that had been waiting for Uxbrita, offset by continued incremental improvement in the 12 and older group. Looking ahead to the second half of the year, if the environment continues to improve, we believe we have the potential to accelerate growth driven by our ongoing DTC campaign, new real-world data that we continue to publish, and the pediatric launch. The key leading indicators that we see as predictive of a return to sustained quarter-over-quarter growth are improvements in the industry-wide new-to-brand prescriptions and our patients' healthcare visits returning to pre-pandemic levels. While we are hopeful this will occur, we know SED patients remain cautious. For example, all the SED-focused community-based organization meetings planned for the second half of the year are taking place virtually. As we prepare for more SED patients to potentially return to face-to-face interactions with their healthcare providers, we are working to raise overall OxyBrita awareness through our ongoing DTC campaign. The metrics from the campaign, around targeted audience reach continue to exceed our original goals. In the first quarter, this was aided by several new tactics, including updating the campaign to reflect the pediatric approval and an improved ability to target SCD patients, which we believe contributed to a higher frequency of monthly engagement. In addition, we launched our branded and unbranded content on new channels, including YouTube, Pandora, and podcasts. visits to Oxbrita.com and SickleCellSpeaks.com reached an all-time high in the first quarter. We also are seeing encouraging growth in the number of SCD patients visiting their HCPs within one month of seeing our DTC advertisement. As we think about recent trends, the conversion rate for new prescriptions was consistent with prior quarters. Similarly, Oxbrita adherence, which includes compliance and persistence for patients in the first year of therapy, continued to be within the range of our prior quarters and analogs. As we gain more data on adherence for year two and beyond, we are seeing lower adherence compared to year one, as would be expected for any chronic medication. But we are encouraged that some patients in year one and beyond have restarted Oxprida, and that Oxprida adherence continues to trend better than SCD analogs. In addition, we continue to proactively roll out new tactics aimed at improving overall adherence, including long-term adherence. This includes additional services through our patient hub, GBT Source Solutions, which continues to be an important driver of patient engagement and adherence. For example, early feedback on the email newsletters and mobile messaging that we launched around the end of 2021 has been encouraging. These services are also available to patients via our specialty pharmacies, And the data shows that patients engaged with GBT Source or the similar services offered by these partners have better adherence rates than those that do not. So we plan to continue investing and driving utilization of these programs. Next, my second metric, healthcare provider penetration. During the quarter, total interactions with healthcare providers increased significantly compared to the fourth quarter, which was lower due to the holidays and lingering impact of the Delta variant. And despite the impact of the Omicron variant in January, in-person visits continued to improve as they have for several quarters, reaching around 50% of our Salesforce interactions with HCPs during the quarter. Against this backdrop, we added about 120 new prescribers in the quarter. This includes several prescribers from the 200 new targets we added for the pediatric launch. This group focuses almost entirely on patients that are 11 and younger. Encouragingly, With the momentum from the pediatric launch on new prescribers and re-engaging prior riders, March was our highest month for the number of active prescribers in the last 12 months. When we look at the breakdown of riders, we continue to see prescriptions being written by both specialists and non-specialists, which we believe is a positive trend for the long-term trajectory of the launch. Turning to payer coverage. We continue to have broad payer coverage for the 12 and older patient population with more than 90% of covered lives having access in the United States. And our focus is on making it easier for physicians to prescribe and patients to receive Oxbrita. In terms of coverage for the 4 to 11 age group, we made substantial progress and we are well on our way to achieving our goal of broad coverage by mid-2022, faster than we did with the adolescent and adult populations. Before turning the call over to Kim, I also want to provide a brief update on our commercial activities in Europe. We are gaining experience with more than 100 patients participating in our early access programs across Germany, France, and the UK. And following our European Commission approval in February, we are working to launch Oxbride in Germany in mid-May. We will have open pricing in Germany for the first year while we negotiate future reimbursement. There are around 3,000 sickle cell patients in Germany, and we anticipate adoption will be gradual, leading to minimal revenues for Europe in 2022. Separately, our team has also begun reimbursement negotiations in France and England. We have begun educating physicians in Europe on Oxtrida, including plans for a robust presence at the European Hematology Association, or EHA, meeting in June. At EHA, we plan to promote Oxtrida for the first time in Europe. engage with physicians, including our branded booth, and sponsor an educational program. We also recently held a successful internal launch meeting in Europe, where it was clear that our employees are excited about bringing Oxbride to the patients and to helping GBT achieve our mission around the world. And with that, Kim will now talk about the developments in our pipeline.

Thank you, DJ, and good afternoon, everyone. On today's call, I will provide an update on our efforts to further expand Oxpritis clinical evidence and geographic reach and our progress advancing our pipeline. Following on our regulatory approval in the European Union in February, we submitted for marketing authorization in Great Britain and believe we are on track for potential approval by mid-year. As a reminder, In the UK, Oxbrita was granted approval of Early Access Medicine Scheme, or EAMS, which provides two key advantages. First, patients that meet the eligibility criteria can gain early pre-licensed access to Oxbrita. We are pleased that UK patients have already started on Oxbrita through the EAMS designation. Second, medicines under EAMS that receive marketing authorization by the MHRA as well as a positive assessment by NICE, benefit from accelerated NHS England commissioning. In support of the pediatric launch, this week at the American Society of Pediatric Hematology Oncology, or AFSPO, conference, we will present results from the Sprite Expanded Access Program for Children with Sickle Cell Disease, age 4 to 11. The EAP data reinforces the efficacy and safety of treatment with Oxbrita in these patients as seen in the HOPE Kids 1 study. Those HOPE Kids 1 results were first presented at EHA last year and were published this April in Pediatric Blood and Cancer. Importantly, the majority of patients in the EAP had improved scores as measured by the patient and clinical global impressions of change scale. indicating that Oxprida treatment is positively impacting their lives. I also want to flag that a 77-patient, single-center, real-world experience study of Oxprida from Dr. Alan Anderson was published in late April in the European Journal of Hematology. Dr. Anderson presented this data, which showed a mean hemoglobin increase of 2 grams per deciliter and substantial improvements in patient quality of life at several meetings in 2021. At the annual EHA meeting in June, we plan to present new OXBRITO real-world data and other studies that reflect our efforts to generate new data. EHA abstracts will be announced later this month, and we are excited about our planned activities. Now let's turn to the pipeline. For Inclacimab, our P-selectin inhibitor, we are enrolling patients in our two Phase III studies collectively named Thrive. One is evaluating the reduction of VOCs over a 48-week treatment period based on Inclacimab's potential for quarterly dosing. We believe this would be a meaningful improvement for patients compared to monthly dosing and aligns well with the typical sickle cell disease practice schedule of quarterly office visits. The other phase three study is evaluating 90-day VOC readmission rates following an initial VOC hospitalization, which tragically occurs in around 50% of patients who experience an initial VOC. This study is enabled by Nklakamab's profile and aligned with its best-in-class potential. We are continuing to focus on enrolling Thrive as quickly as possible. Turning to GBT601, our next-generation hemoglobin polymerization inhibitor that we believe has potential to be a best-in-class therapy. At EHA, we aim to share more data on GBT601, including sickle cell disease patient EPO levels from the Phase I study. Separately, we aim to publish new preclinical data for GBT601 in a peer-reviewed journal later this year. Based on the impressive data we reported at ASH in December, we are working to aggressively advance GBT-601. Our plan is to initiate a Phase 2-3 clinical trial with the goal of submitting for a full regulatory approval with FDA. We expect the Phase 2-3 design will allow us to advance more quickly into the pivotal Phase 3 portion and are working towards our goal of initiating the Phase 2 portion by mid-2022. The purpose of the Phase II portion is to identify the optimal dose to advance into Phase III. As we explore higher doses, we believe they will lead to higher average occupancy and hemoglobin increases and, importantly, consistently improve the red blood cell health of patients to resemble that of a sickle cell trait individual. We are also exploring a 150 milligram dose of GBT-601 with patients from our phase one study. We are very excited that all six patients reached out to the clinical trial site wanting to restart therapy, providing us the opportunity to restart the study and explore a higher dose. In my experience, this is the first time I have seen the restart of a sickle cell study due to patient demand. Patients will receive a 150 milligram daily maintenance dose for six weeks. We anticipate data from this portion of the study will be available by the end of the year and include the same key and points as the data we reported at ASH in December. I am proud of the progress we are making with GBT-601 over a short time frame, while also continuing to drive enrollment in our enclaquimab studies and advancing our oxbrita studies. I firmly believe GVT has the leading clinical and pipeline programs in sickle cell disease. I'll now turn it back over to Ted.