On August 2, 2022 Exact Sciences Corp. (Nasdaq: EXAS), a leading provider of cancer screening and diagnostic tests, reported that the company generated revenue of $521.6 million for the second quarter ended June 30, 2022, compared to $434.8 million for the same period of 2021 (Press release, Exact Sciences, AUG 2, 2022, View Source [SID1234617263]).

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"Exact Sciences’ strong second quarter results reflect meaningful progress toward our vision to help eradicate cancer by providing patients better information before diagnosis and throughout their treatment," said Kevin Conroy, chairman and CEO. "We’re focusing on getting more people tested with Cologuard and Oncotype, prioritizing our highest impact projects to reach profitability, and generating high-quality evidence for our pipeline of cancer diagnostic tests."

Second quarter 2022 financial results

For the three-month period ended June 30, 2022, as compared to the same period of 2021 (where applicable):

Total revenue was $521.6 million, an increase of 20 percent
Total revenue, excluding COVID-19 testing, increased 26 percent
Screening revenue was $353.9 million, an increase of 34 percent
Excluding PreventionGenetics acquisition, Screening revenue was $343.7 million, an increase of 30 percent
Precision Oncology revenue was $154.0 million, an increase of 12 percent
COVID-19 testing revenue was $13.8 million, a decrease of 58 percent
Gross margin including amortization of acquired intangible assets was 68 percent, and non-GAAP gross margin excluding amortization of acquired intangible assets was 72 percent
Net loss was $166.1 million, or $0.94 per share, compared to a net loss of $176.9 million, or $1.03 per share
EBITDA was $(111.4) million and adjusted EBITDA was $(45.8) million
Cash, cash equivalents, and marketable securities were $728.0 million at the end of the quarter
Screening includes laboratory service revenue from Cologuard tests, PreventionGenetics, and immaterial revenue from Biomatrica and Oncoguard Liver products. Precision Oncology includes laboratory service revenue from global Oncotype products and therapy selection products, including oncomap and oncomap ExTra, formerly known as Oncotype Map and GEM ExTra, respectively.

2022 outlook

The company anticipates revenue of $1,980-$2,022 million during 2022, assuming:

Screening revenue of $1,350-$1,372 million, including $40-$42 million from PreventionGenetics,
Precision Oncology revenue of $580-$590 million, including the divestiture of the Oncotype DX Genomic Prostate Score test, and
COVID-19 testing revenue of $50-$60 million.
Revenue guidance has been updated from the previously expected range of $1,985-$2,032 million, which assumed:

Screening revenue of $1,350-$1,372 million, including $40-$42 million from PreventionGenetics,
Precision Oncology revenue of $595-$610 million, and
COVID-19 testing revenue of $40-$50 million.
Non-GAAP disclosure

In addition to the company’s financial results determined in accordance with U.S. GAAP, the company provides non-GAAP measures that it determines to be useful in evaluating its operating performance. The company presents EBITDA, adjusted EBITDA, as well as non-GAAP gross margin and non-GAAP gross profit. EBITDA and adjusted EBITDA consist of net loss after adjustment for those items shown in the table below. The company defines non-GAAP gross profit and non-GAAP gross margin as GAAP gross profit and GAAP gross margin, respectively, excluding amortization of acquired intangible assets. The amortization of acquisition-related intangible assets used in the calculation of non-GAAP gross profit and non-GAAP gross margin pertain only to the amortization associated with developed technology acquired and recorded through purchase accounting transactions. The amortization of these intangible assets will recur in future periods until such intangible assets have been fully amortized. The company believes that these non-GAAP measures are useful in evaluating the company’s operating performance. The company uses this non-GAAP financial information to evaluate ongoing operations and for internal planning and forecasting purposes. Non-GAAP financial information, when taken collectively, may be helpful to investors because it provides consistency and comparability with past financial performance. However, non-GAAP financial information is presented for supplemental information purposes only, has limitations as an analytical tool and should not be considered in isolation or as a substitute for financial information presented in accordance with U.S. GAAP. For example, non-GAAP gross margin and non-GAAP gross profit exclude the amortization of acquired intangible assets although such measures include the revenue associated with the acquisitions. For a reconciliation of these non-GAAP measures to GAAP, see below "EBITDA and Adjusted EBITDA Reconciliations" and "Non-GAAP Gross Profit and Non-GAAP Gross Margin Reconciliations."

Second quarter conference call & webcast

Company management will host a conference call and webcast on Tuesday, August 2, 2022, at 5 p.m. ET to discuss second quarter 2022 results. The webcast will be available at exactsciences.com. Domestic callers should dial 888-330-2384 and international callers should dial +1-240-789-2701. The access code for both domestic and international callers is 4437608.

An archive of the webcast will be available at exactsciences.com. A replay of the conference call will be available by calling 800-770-2030 domestically or +1-647-362-9199 internationally. The access code for the replay of the call is 4437608. The webcast, conference call, and replay are open to all interested parties.

About Cologuard

The Cologuard test was approved by the FDA in August 2014, and results from Exact Sciences’ prospective 90-site, point-in-time, 10,000-patient pivotal trial were published in the New England Journal of Medicine in March 2014. The Cologuard test is included in the American Cancer Society’s (2018) colorectal cancer screening guidelines and the recommendations of the U.S. Preventive Services Task Force (2021) and National Comprehensive Cancer Network (2016). The Cologuard test is indicated to screen adults 45 years of age and older who are at average risk for colorectal cancer by detecting certain DNA markers and blood in the stool. Do not use the Cologuard test if you have had precancer, have inflammatory bowel disease and certain hereditary syndromes, or have a personal or family history of colorectal cancer. The Cologuard test is not a replacement for colonoscopy in high risk patients. The Cologuard test performance in adults ages 45-49 is estimated based on a large clinical study of patients 50 and older. The Cologuard test performance in repeat testing has not been evaluated.

The Cologuard test result should be interpreted with caution. A positive test result does not confirm the presence of cancer. Patients with a positive test result should be referred for diagnostic colonoscopy. A negative test result does not confirm the absence of cancer. Patients with a negative test result should discuss with their doctor when they need to be tested again. Medicare and most major insurers cover the Cologuard test. For more information about the Cologuard test, visit cologuard.com. Rx only.

About Exact Sciences’ Precision Oncology portfolio

Exact Sciences’ Precision Oncology portfolio delivers actionable genomic insights to inform prognosis and cancer treatment after a diagnosis. In breast cancer, the Oncotype DX Breast Recurrence Score is the only test shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer. The Oncotype DX test is recognized as the standard of care and is included in all major breast cancer treatment guidelines. The Oncomap ExTra test applies comprehensive tumor profiling, utilizing whole exome and whole transcriptome sequencing, to aid in therapy selection for patients with advanced, metastatic, refractory, relapsed, or recurrent cancer. With an extensive panel of approximately 20,000 genes and 169 introns, the Oncomap ExTra test is one of the most comprehensive genomic (DNA) and transcriptomic (RNA) panels available today. Exact Sciences enables patients to take a more active role in their cancer care and makes it easy for providers to order tests, interpret results, and personalize medicine by applying real-world evidence and guideline recommendations. To learn more, precisiononcology.exactsciences.com.

About PreventionGenetics

Founded in 2004 and located in Marshfield, Wisconsin, PreventionGenetics is a CLIA and ISO 15189:2012 accredited laboratory. PreventionGenetics delivers clinical genetic testing of the highest quality at fair prices with exemplary service to people around the world. PreventionGenetics has 25 PhD geneticists on staff and provides tests for nearly all clinically relevant genes including the powerful and comprehensive germline whole genome sequencing test, PGnome and whole exome sequencing test, PGxome. PreventionGenetics was acquired by Exact Sciences in December 2021.

On August 2, 2022 Exact Sciences Corp. (NASDAQ: EXAS), a global leader in cancer diagnostics, reported that it has entered a collaboration agreement with the West German Study Group (WSG), an international research institution that focuses on practice-changing clinical studies in breast cancer (Press release, Exact Sciences, AUG 2, 2022, View Source [SID1234617262]). Together, Exact Sciences and WSG plan to conduct a prospective, multicenter validation study in hormone receptor (HR)-positive, HER2-negative early breast cancer patients. The study is intended to demonstrate the ability of Exact Sciences’ tumor-informed ctDNA liquid biopsy test to detect minimal residual disease (MRD) and to collect important long-term follow-up and outcome data.

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The combined analysis, called ADAPT Triad, is expected to include data from approximately 3,000 German patients enrolled in two ongoing WSG ADAPT trials and one WSG registry study. All patients included in the analysis are stratified using the Oncotype DX Breast Recurrence Score test. The study is part of Exact Sciences’ global initiative to generate clinical validation data for its tumor-informed ctDNA MRD liquid biopsy test, currently in development.

"This ADAPT Triad project with WSG offers a tremendous opportunity to help inform treatment decisions and recurrence monitoring, and ultimately improve future outcomes for breast cancer patients," said Rick Baehner, MD, chief medical officer of Precision Oncology for Exact Sciences. "In addition to helping validate our MRD assay, we’re also eager to better understand the potential synergies involving the predictive and prognostic information provided by our Oncotype DX Breast Recurrence Score test. WSG’s expertise in breast cancer research makes them ideal collaborators to conduct these important MRD studies in HR positive, HER2-negative early breast cancer."

The ADAPT Triad MRD analysis study in breast cancer builds on Exact Sciences and the National Surgical Adjuvant Breast and Bowel Project’s (NSABP) CORRECT-MRD II clinical validation study to detect MRD in colorectal cancer patients. These studies will help create a solid evidence foundation for Exact Sciences’ MRD program in two core cancer types where the company has established leadership.

"The WSG’s ADAPT study program examines ways to achieve patient-specific decision-making for the treatment of early breast cancer based on biological markers," said Professor Nadia Harbeck, Scientific Director of the WSG and Head of the Breast Centre at LMU Klinikum Munich, Germany. "Partnering with our long-time collaborators at Exact Sciences to evaluate the association of post-initial therapy, pre-recurrence ctDNA with distant recurrence-free interval is a worthy addition to our program and will help change the treatment and monitoring program for solid tumors."

About Minimal Residual Disease
Minimal residual disease (MRD) refers to the presence of tumor-specific DNA in the body after cancer treatment. These fragments of genetic information, known as circulating tumor DNA (ctDNA), are shed into the blood by solid tumors as part of the tumor growth cycle. Their presence may indicate that a tumor is likely to return. Solid tumors are typically undetected until they are advanced enough to be picked up by a radiologic image or physician examination. The detection of ctDNA at extremely low levels has the potential to provide crucial insights that may help discover cancer recurrence earlier and inform treatment decisions.

About Exact Sciences’ MRD Program
The Exact Sciences’ MRD program is powered by our in-house capabilities to advance a tumor-informed or a tumor-naïve solution, with an initial focus on tumor-informed (bespoke) with whole exome or whole genome sequencing. This approach identifies somatic genomic alterations in DNA extracted from the patient’s tumor tissue and detects a subset of these mutations in ctDNA present in the patient’s blood. The MRD test that Exact Sciences is developing is intended for patients diagnosed with solid tumor malignancies to detect ctDNA before, during, and after cancer treatment. Such information may be used for guidance of adjuvant therapy decisions and/or for monitoring of cancer recurrence, in conjunction with other clinicopathological findings, providing more than 12 million testing opportunities in the United States alone.1

On August 2, 2022 Exact Sciences Corp. (Nasdaq: EXAS), a leading provider of cancer screening and diagnostic tests, reported that the company generated revenue of $521.6 million for the second quarter ended June 30, 2022, compared to $434.8 million for the same period of 2021 (Press release, Exact Sciences, AUG 2, 2022, View Source [SID1234617261]).

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Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Exact Sciences’ strong second quarter results reflect meaningful progress toward our vision to help eradicate cancer by providing patients better information before diagnosis and throughout their treatment," said Kevin Conroy, chairman and CEO. "We’re focusing on getting more people tested with Cologuard and Oncotype, prioritizing our highest impact projects to reach profitability, and generating high-quality evidence for our pipeline of cancer diagnostic tests."

Second quarter 2022 financial results

For the three-month period ended June 30, 2022, as compared to the same period of 2021 (where applicable):

Total revenue was $521.6 million, an increase of 20 percent
Total revenue, excluding COVID-19 testing, increased 26 percent
Screening revenue was $353.9 million, an increase of 34 percent
Excluding PreventionGenetics acquisition, Screening revenue was $343.7 million, an increase of 30 percent
Precision Oncology revenue was $154.0 million, an increase of 12 percent
COVID-19 testing revenue was $13.8 million, a decrease of 58 percent
Gross margin including amortization of acquired intangible assets was 68 percent, and non-GAAP gross margin excluding amortization of acquired intangible assets was 72 percent
Net loss was $166.1 million, or $0.94 per share, compared to a net loss of $176.9 million, or $1.03 per share
EBITDA was $(111.4) million and adjusted EBITDA was $(45.8) million
Cash, cash equivalents, and marketable securities were $728.0 million at the end of the quarter
Screening includes laboratory service revenue from Cologuard tests, PreventionGenetics, and immaterial revenue from Biomatrica and Oncoguard Liver products. Precision Oncology includes laboratory service revenue from global Oncotype products and therapy selection products, including oncomap and oncomap ExTra, formerly known as Oncotype Map and GEM ExTra, respectively.

2022 outlook

The company anticipates revenue of $1,980-$2,022 million during 2022, assuming:

Screening revenue of $1,350-$1,372 million, including $40-$42 million from PreventionGenetics,
Precision Oncology revenue of $580-$590 million, including the divestiture of the Oncotype DX Genomic Prostate Score test, and
COVID-19 testing revenue of $50-$60 million.
Revenue guidance has been updated from the previously expected range of $1,985-$2,032 million, which assumed:

Screening revenue of $1,350-$1,372 million, including $40-$42 million from PreventionGenetics,
Precision Oncology revenue of $595-$610 million, and
COVID-19 testing revenue of $40-$50 million.
Non-GAAP disclosure

In addition to the company’s financial results determined in accordance with U.S. GAAP, the company provides non-GAAP measures that it determines to be useful in evaluating its operating performance. The company presents EBITDA, adjusted EBITDA, as well as non-GAAP gross margin and non-GAAP gross profit. EBITDA and adjusted EBITDA consist of net loss after adjustment for those items shown in the table below. The company defines non-GAAP gross profit and non-GAAP gross margin as GAAP gross profit and GAAP gross margin, respectively, excluding amortization of acquired intangible assets. The amortization of acquisition-related intangible assets used in the calculation of non-GAAP gross profit and non-GAAP gross margin pertain only to the amortization associated with developed technology acquired and recorded through purchase accounting transactions. The amortization of these intangible assets will recur in future periods until such intangible assets have been fully amortized. The company believes that these non-GAAP measures are useful in evaluating the company’s operating performance. The company uses this non-GAAP financial information to evaluate ongoing operations and for internal planning and forecasting purposes. Non-GAAP financial information, when taken collectively, may be helpful to investors because it provides consistency and comparability with past financial performance. However, non-GAAP financial information is presented for supplemental information purposes only, has limitations as an analytical tool and should not be considered in isolation or as a substitute for financial information presented in accordance with U.S. GAAP. For example, non-GAAP gross margin and non-GAAP gross profit exclude the amortization of acquired intangible assets although such measures include the revenue associated with the acquisitions. For a reconciliation of these non-GAAP measures to GAAP, see below "EBITDA and Adjusted EBITDA Reconciliations" and "Non-GAAP Gross Profit and Non-GAAP Gross Margin Reconciliations."

Second quarter conference call & webcast

Company management will host a conference call and webcast on Tuesday, August 2, 2022, at 5 p.m. ET to discuss second quarter 2022 results. The webcast will be available at exactsciences.com. Domestic callers should dial 888-330-2384 and international callers should dial +1-240-789-2701. The access code for both domestic and international callers is 4437608.

An archive of the webcast will be available at exactsciences.com. A replay of the conference call will be available by calling 800-770-2030 domestically or +1-647-362-9199 internationally. The access code for the replay of the call is 4437608. The webcast, conference call, and replay are open to all interested parties.

About Cologuard

The Cologuard test was approved by the FDA in August 2014, and results from Exact Sciences’ prospective 90-site, point-in-time, 10,000-patient pivotal trial were published in the New England Journal of Medicine in March 2014. The Cologuard test is included in the American Cancer Society’s (2018) colorectal cancer screening guidelines and the recommendations of the U.S. Preventive Services Task Force (2021) and National Comprehensive Cancer Network (2016). The Cologuard test is indicated to screen adults 45 years of age and older who are at average risk for colorectal cancer by detecting certain DNA markers and blood in the stool. Do not use the Cologuard test if you have had precancer, have inflammatory bowel disease and certain hereditary syndromes, or have a personal or family history of colorectal cancer. The Cologuard test is not a replacement for colonoscopy in high risk patients. The Cologuard test performance in adults ages 45-49 is estimated based on a large clinical study of patients 50 and older. The Cologuard test performance in repeat testing has not been evaluated.

The Cologuard test result should be interpreted with caution. A positive test result does not confirm the presence of cancer. Patients with a positive test result should be referred for diagnostic colonoscopy. A negative test result does not confirm the absence of cancer. Patients with a negative test result should discuss with their doctor when they need to be tested again. Medicare and most major insurers cover the Cologuard test. For more information about the Cologuard test, visit cologuard.com. Rx only.

About Exact Sciences’ Precision Oncology portfolio

Exact Sciences’ Precision Oncology portfolio delivers actionable genomic insights to inform prognosis and cancer treatment after a diagnosis. In breast cancer, the Oncotype DX Breast Recurrence Score is the only test shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer. The Oncotype DX test is recognized as the standard of care and is included in all major breast cancer treatment guidelines. The Oncomap ExTra test applies comprehensive tumor profiling, utilizing whole exome and whole transcriptome sequencing, to aid in therapy selection for patients with advanced, metastatic, refractory, relapsed, or recurrent cancer. With an extensive panel of approximately 20,000 genes and 169 introns, the Oncomap ExTra test is one of the most comprehensive genomic (DNA) and transcriptomic (RNA) panels available today. Exact Sciences enables patients to take a more active role in their cancer care and makes it easy for providers to order tests, interpret results, and personalize medicine by applying real-world evidence and guideline recommendations. To learn more, precisiononcology.exactsciences.com.

About PreventionGenetics

Founded in 2004 and located in Marshfield, Wisconsin, PreventionGenetics is a CLIA and ISO 15189:2012 accredited laboratory. PreventionGenetics delivers clinical genetic testing of the highest quality at fair prices with exemplary service to people around the world. PreventionGenetics has 25 PhD geneticists on staff and provides tests for nearly all clinically relevant genes including the powerful and comprehensive germline whole genome sequencing test, PGnome and whole exome sequencing test, PGxome. PreventionGenetics was acquired by Exact Sciences in December 2021.

On August 2, 2022 Calidi Biotherapeutics, Inc. (Calidi), a clinical-stage biotechnology company that is pioneering allogeneic cell-based platforms to revolutionize oncolytic virus therapies, reported that City of Hope has received U.S. Food and Drug Administration (FDA) authorization to proceed with a phase 1 physician-sponsored clinical trial that will use Calidi’s licensed oncolytic virotherapy platform, NSC-CRAd-S-pk7 (NeuroNova), a cutting-edge therapeutic candidate comprising tumor-tropic neural stem cells delivering an oncolytic adenovirus selectively to tumor sites in patients with recurrent high-grade glioma (Calidi’s NNV-2 program) (Press release, Calidi Biotherapeutics, AUG 2, 2022, View Source [SID1234617260]).

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The phase 1 physician-sponsored clinical trial will be an open-label, non-randomized, multicenter study. Once the phase 1 trial is funded, it will address the safety and tolerability of administering serial doses of NeuroNova in adult patients with recurrent histologically confirmed high-grade gliomas (WHO grade III or IV). Secondary endpoints will evaluate treatment efficacy, including progression-free and overall survival as well as any immune response to NeuroNova. This physician-sponsored study will be led by principal investigator Jana Portnow, M.D., Professor in City of Hope’s Department of Medical Oncology & Therapeutics Research and Co-Director of the Brain Tumor Program at City of Hope, a National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network. City of Hope is one of the largest cancer research and treatment organizations in the United States.

A previously completed phase 1 dose escalation clinical trial, in newly diagnosed glioma patients, of a single dose of NSC-CRAd-S-pk7 (NeuroNova) given as an adjunct to radiation and temozolomide (Calidi’s NNV-1 program) demonstrated that NeuroNova was well tolerated in the patient population and showed promising preliminary results of efficacy [J Fares et al, Lancet Oncology, 2021, 22(8):1103-1114].

"Worldwide, an estimated 270,000 people were diagnosed with a primary brain tumor in 2020, with a five-year survival rate of only 5%. Recurrent glioma patients have poor prognosis with a median survival of less than one year. Developing our drug candidate for this indication represents a significant milestone that will empower us to help more individuals survive this devastating disease," said Calidi Biotherapeutics CEO and Chairman of the Board Allan J. Camaisa. "As Calidi undertakes this endeavor, we are pleased to have exclusive commercialization rights to this technology and thrilled to collaborate with two scientific trailblazers who have worked together on this project for over a decade: Dr. Karen Aboody, Professor in the Department of Stem Cell Biology and Regenerative Medicine, Scientific Leader of the Neuro-oncology Disease Team at City of Hope, and Dr. Maciej (Matt) Lesniak, Chair of the Department of Neurological Surgery, at Northwestern University Feinberg School of Medicine."

"Our team at City of Hope is excited to be a part of the development of NeuroNova. Despite their potential, the first-generation oncolytic virus therapies given as free virus were not very effective — most likely due to rapid inactivation by the patient’s immune system. Our new platform uses stem cells like a ‘Trojan horse’ to shield the oncolytic viral particles from immune inactivation and deliver them to the tumor sites. This results in significantly more virus distributed at the tumor sites, inducing a greater self-amplifying anti-tumor response. This may also result in a secondary anti-tumor immune response," said City of Hope’s Dr. Karen Aboody.
"In addition to significant experience with this technology, City of Hope has a proven track record in GMP manufacturing of cell and gene therapy agents."

About NeuroNova
The NeuroNova platform, NSC-CRAd-S-pk7, is an allogeneic, "off-the-shelf" therapy comprised of an immortalized Neural Stem Cell (NSC) line loaded with an engineered oncolytic adenovirus. Upon surgical resection of a tumor, NSC-CRAd-S-pk7 is injected into the walls of the resection cavity, resulting in viral infection and destruction of any remaining tumor cells. Calidi holds an exclusive worldwide licensing agreement for patents covering the NSC-CRAd-S-pk7 technology.

On August 2, 2022 Blueprint Medicines Corporation (NASDAQ: BPMC) reported that financial results and provided a business update for the second quarter ended June 30, 2022 (Press release, Blueprint Medicines, AUG 2, 2022, View Source [SID1234617259]).

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"We are now one year into the AYVAKIT launch in advanced systemic mastocytosis (SM) and continue to see a significant expansion of our prescriber base as well as strong patient demand, reinforcing our conviction in the growth opportunity in SM," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "We are on track to announce topline data from our registration-enabling PIONEER trial in August, which will catalyze our ability to bring AYVAKIT’s transformative potential to patients living with non-advanced SM. In addition, we continue to make significant progress across our pipeline of innovative investigational medicines in EGFR-mutant and CDK2-vulnerable cancers. We look forward to sharing more about our strategic vision, including the opportunities we see in SM, EGFR-mutant lung cancer, and CDK2-vulnerable cancers, and how our expanding research platform will continue to drive innovation at an Investor Day on November 1, 2022 in New York, NY. With our strong revenue performance, a clinical-stage pipeline of five significant assets, and well over $1 billion in cash on our balance sheet as of today, we are uniquely positioned to drive near- and long-term value for all of our stakeholders by delivering transformative precision medicines to patients around the world."

Second Quarter 2022 Highlights and Recent Progress

AYVAKIT/AYVAKYT (avapritinib): systemic mastocytosis (SM) and PDGFRA gastrointestinal stromal tumor (GIST)

Reported global net product revenues of $28.5 million for the second quarter of 2022.
Presented new retrospective analyses at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Congress, showing that AYVAKIT significantly improved overall survival in patients with advanced SM when compared to real-world data for prior best available therapies, including midostaurin and cladribine. Read the press release here.
Upon request by the FDA, changed the primary endpoint for the registrational PIONEER Part 2 trial of AYVAKIT in patients with non-advanced SM to the mean change in total symptom score (TSS).
GAVRETO (pralsetinib): RET-altered cancers

As previously recorded and reported by Roche, GAVRETO global product sales were 7 million CHF, which excludes sales in the Greater China territory driven by CStone Pharmaceuticals.
Received approval in Hong Kong, China, via the collaboration with CStone Pharmaceuticals, for the treatment of RET fusion-positive non-small cell lung cancer (NSCLC).
Corporate

Announced strategic financing collaborations with Sixth Street Partners (Sixth Street) and Royalty Pharma plc (Royalty Pharma) (NASDAQ: RPRX) for up to $1.25 billion, of which $175 million was funded as of June 30, 2022, and an additional $400 million funded in July 2022. Read the press release here.
Licensed a development candidate-stage KIT exon 13 inhibitor, and licensed the compound to IDRx, Inc., a recently launched clinical-stage biopharmaceutical company, in exchange for a 15 percent Series A preferred equity investment and up to $217.5 million in future milestone payments and tiered percentage royalty payments.
Published an inaugural Corporate Responsibility Report, highlighting the company’s long-standing commitment to delivering sustainable value to patients with cancer and blood disorders, as well as the communities in which Blueprint Medicines operates. Read the press release here and access the report here.
Announced the appointment of Habib Dable, former Chief Executive Officer of Acceleron Pharma, Inc., to the company’s Board of Directors. Read the press release here.
Key Upcoming Milestones

The company plans to achieve the following near-term milestones:

Present topline data from the registrational PIONEER Part 2 trial for avapritinib in non-advanced SM in August 2022 and submit a supplemental new drug application to the FDA for AYVAKIT in non-advanced SM in the second half of 2022.
Share the strategic vision for Blueprint, including opportunities in SM, EGFR-mutant lung cancer, and CDK2-vulnerable cancers, and how our expanding research platform has the potential to deliver the promise of precision medicine to more patients at an Investor Day on November 1, 2022 in New York, NY.
Present updated BLU-945 monotherapy data and initial dose escalation data for BLU-945 in combination with osimertinib from the Phase 1/2 SYMPHONY trial in EGFR-mutant NSCLC in the second half of 2022.
Present initial clinical data from the Phase 1/2 HARMONY trial of BLU-701 in EGFR-mutant NSCLC in the second half of 2022.
Present initial data from the HARBOR trial of BLU-263 in non-advanced SM in the second half of 2022.
Present initial clinical data from the Phase 1/2 CONCERTO trial of BLU-451 in EGFR-mutant NSCLC in the first half of 2023.
Present initial clinical data from the Phase 1/2 VELA trial of BLU-222 in CDK2-vulnerable cancers in the first half of 2023.
Second Quarter 2022 Results

Revenues: Revenues were $36.5 million for the second quarter of 2022, including $28.5 million of net product revenues from sales of AYVAKIT/AYVAKYT and $8.0 million in collaboration revenues. Blueprint Medicines recorded revenues of $27.3 million in the second quarter of 2021, including $8.5 million of net product revenues from sales of AYVAKIT/AYVAKIT, $2.9 million of net product revenues from sales of GAVRETO and $15.9 million in collaboration revenues.
Cost of Sales: Cost of sales was $4.9 million for the second quarter of 2022, as compared to $6.5 million for the second quarter of 2021.
R&D Expenses: Research and development expenses were $128.5 million for the second quarter of 2022, as compared to $80.0 million for the second quarter of 2021. This increase was primarily due to increased costs associated with the progression of our clinical trials and increased costs related to early discovery effort. Research and development expenses included $10.5 million in stock-based compensation expenses for the second quarter of 2022.
SG&A Expenses: Selling, general and administrative expenses were $58.7 million for the second quarter of 2022, as compared to $49.3 million for the second quarter of 2021. This increase was primarily due to increased costs associated with expanding our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT. General and administrative expenses included $14.9 million in stock-based compensation expenses for the second quarter of 2022.
Net Loss: Net loss was $159.7 million for the second quarter of 2022, or a net loss per share of $2.68, as compared to a net loss of $108.4 million for the second quarter of 2021, or a net loss per share of $1.86.
Cash Position: As of June 30, 2022, cash, cash equivalents and investments were $947.2 million, as compared to $1,034.6 million as of December 31, 2021. Cash as of June 30, 2022, does not include $400 million gross proceeds received from our strategic non-dilutive financing agreements with Sixth Street, which closed and were funded in July 2022.
Financial Guidance

Blueprint Medicines anticipates approximately $180M to $200M in total revenues in 2022, including approximately $115M to $130M in AYVAKIT net product revenues. The company continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss second quarter 2022 financial results and recent business activities. The conference call may be accessed by dialing 844-200-6205 (domestic) or 929-526-1599 (international), and referring to conference ID 694684. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.