On February 16, 2021 Exact Sciences Corp. (Nasdaq: EXAS) reported that the company generated revenue of $466.3 million for the fourth quarter ended Dec. 31, 2020 and $1,491.4 million for the full year ended Dec. 31, 2020 (Press release, Exact Sciences, FEB 16, 2021, View Source [SID1234575120]).

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"Exact Sciences finished another transformative year by delivering strong fourth quarter results against a challenging backdrop because of the pandemic," said Kevin Conroy, chairman and CEO of Exact Sciences. "We are a leader in cancer diagnostics because of our people, scientific platform, and market-leading Cologuard and Oncotype tests. We aim to extend this leadership throughout the cancer continuum and bring additional tests to patients to help improve cancer outcomes."

Fourth Quarter 2020 Financial Results

For the three-month period ended December 31, 2020, as compared to the same period of 2019 (where applicable):

Total revenue was $466.3 million
Screening revenue was $249.7 million, an increase of 9 percent
Precision Oncology revenue was $117.6 million
COVID-19 testing revenue was $99.1 million
Gross margin including amortization of acquired intangible assets was 74 percent, and non-GAAP gross margin excluding amortization of acquired intangible assets was 79 percent
During the fourth quarter of 2020, the Company acquired Base Genomics, which was treated as an asset acquisition under U.S. GAAP and resulted in a $412.6 million charge to research and development expense
Net loss was $(436.8) million, or $(2.79) per share, compared to net income of $78.0 million, or $0.56 and $0.54 per basic and diluted share.
EBITDA was $(375.5) million and adjusted EBITDA was $87.9 million
Non-cash interest expense related to convertible debt was $21.3 million, compared to $11.5 million
Cash, cash equivalents and marketable securities were $1,840.0 million at the end of the quarter
Screening includes laboratory service revenue from Cologuard and revenue from Biomatrica products. Precision Oncology includes laboratory service revenue from global Oncotype products.

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."

Fourth Quarter Conference Call & Webcast
Company management will host a conference call and webcast on Tuesday, February 16, 2021, at 5 p.m. ET to discuss fourth quarter and full-year 2020 results. The webcast will be available at www.exactsciences.com. Domestic callers should dial 833-235-7650 and international callers should dial +1-647-689-4171. The access code for both domestic and international callers is 2892148.

An archive of the webcast will be available at www.exactsciences.com. A replay of the conference call will be available by calling 800-585-8367 domestically or +1-416-621-4642 internationally. The access code for the replay of the call is 2892148. The webcast, conference call and replay are open to all interested parties.

About Cologuard
Cologuard 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. Cologuard is included in the American Cancer Society’s (2018) colorectal cancer screening guidelines and the recommendations of the U.S. Preventive Services Task Force (2016) and National Comprehensive Cancer Network (2016). Cologuard 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 Cologuard if you have had precancer, have inflammatory bowel disease and certain hereditary syndromes, or have a personal or family history of colorectal cancer. Cologuard is not a replacement for colonoscopy in high risk patients. Cologuard performance in adults ages 45-49 is estimated based on a large clinical study of patients 50 and older. Cologuard 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 Cologuard. For more information about Cologuard, visit www.cologuardtest.com. Rx Only.

About Oncotype DX
The Oncotype DX portfolio of breast, colon and prostate cancer tests applies advanced genomic science to reveal the unique biology of a tumor in order to optimize cancer treatment decisions. In breast cancer, the Oncotype DX Breast Recurrence Score test is the only test that has been shown to predict the likelihood of chemotherapy benefit as well as recurrence in invasive breast cancer. Additionally, the Oncotype DX Breast DCIS Score test predicts the likelihood of recurrence in a pre-invasive form of breast cancer called DCIS. In prostate cancer, the Oncotype DX Genomic Prostate Score test predicts disease aggressiveness and further clarifies the current and future risk of the cancer prior to treatment intervention, and the Oncotype DX AR-V7 Nucleus Detect test helps determine which patients with metastatic castration-resistant prostate cancer (mCRPC) are resistant to androgen receptor (AR)-targeted therapies. The Oncotype DX AR-V7 Nucleus Detect test is performed by Epic Sciences at its centralized, CLIA-certified laboratory in San Diego and offered exclusively by Exact Sciences. With more than 1 million patients tested in more than 90 countries, the Oncotype DX tests have redefined personalized medicine by making genomics a critical part of cancer diagnosis and treatment. To learn more about Oncotype DX tests, visit www.OncotypeIQ.com, www.MyBreastCancerTreatment.org or www.MyProstateCancerTreatment.org.

On February 16, 2021 Exact Sciences Corp. (Nasdaq: EXAS) reported that it has entered into an agreement to acquire Ashion Analytics, LLC (Ashion) from The Translational Genomics Research Institute (TGen), an affiliate of City of Hope (Press release, Exact Sciences, FEB 16, 2021, View Source [SID1234575119]). Ashion is a CLIA-certified and CAP-accredited sequencing lab based in Phoenix, Arizona with the genomics testing capabilities necessary to address the increasingly complex needs of clinical, academic, and biopharma customers focused on precision cancer treatments. The team at Ashion will help accelerate the development of Exact Sciences’ precision oncology portfolio, including minimal residual disease (MRD) and other sequencing-based tests.

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This acquisition will build upon the recent license of the TARDIS technology from TGen and strengthen the relationship between the institutions through a planned 10-year research collaboration. TARDIS is a highly sensitive, patient-specific liquid biopsy-based test that can detect small amounts of tumor DNA in blood for use in MRD testing.

"Exact Sciences continues to strengthen the robust foundation established for Oncotype to lead precision oncology," said Kevin Conroy, chairman and CEO of Exact Sciences. "We’re excited to work with Ashion to bring the TARDIS technology to patients faster and continue to develop advancements in MRD testing. We’re thrilled to collaborate with TGen and City of Hope, complementing our relationships with other world-renowned cancer research leaders including Mayo Clinic and Johns Hopkins University."

Ashion leverages the genetic information from a patient’s tumor and normal genomes to provide industry-leading oncology solutions. Ashion developed GEM ExTra, one of the most comprehensive genomic cancer tests available, and provides access to whole exome, matched germline, and transcriptome sequencing capabilities. The team at Ashion will be instrumental in incorporating the TARDIS technology into Exact Sciences’ MRD test development.

Under the agreement, Exact Sciences would also enter a 10-year collaboration, bringing in the expertise of TGen and City of Hope, to develop differentiated MRD testing capabilities for patients and establish the clinical evidence necessary to drive adoption.

"This will be a groundbreaking research collaboration for TGen and City of Hope, as we continue to advance important scientific innovations and transformative treatment approaches for cancer care," said Robert Stone, president and CEO of City of Hope. "On the heels of our recent licensing agreement for our proprietary TARDIS technology, Exact Sciences is an ideal partner for this new collaboration, which aims to provide more information to physicians and their patients worldwide when diagnosing and treating cancer."

"TGen scientists have been at the forefront of the convergence of science and technology to accelerate cancer innovation, leading to the launch of Ashion and its flagship test GEM ExTra," said Jeffrey M. Trent, Ph.D., TGen president and research director. "The research collaboration with Exact Sciences allows us to work alongside an industry leader in cancer diagnostics to help ensure future breakthroughs and technology developments will bring greater benefit to more patients at a faster pace."

TGen became a part of City of Hope in 2016 to accelerate the speed at which scientists and medical staff convert research discoveries into cures for patients. City of Hope is a National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, making it a national leader in advancing research and treatment protocols.

Additional Information
The transaction is subject to customary closing conditions and regulatory approvals and is anticipated to close during the second quarter of 2021. XMS Capital is serving as financial advisor to Exact Sciences, and K&L Gates is serving as legal advisor. Citi is serving as financial advisor to TGen and City of Hope, and Jennings, Strouss & Salmon, P.L.C. is serving as legal advisor.

On February 16, 2021 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported preliminary and unaudited revenues for the fourth quarter and full-year 2020 and provided an update on key highlights for 2021 (Press release, CASI Pharmaceuticals, FEB 16, 2021, View Source [SID1234575118]).

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Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "We have taken a number of important strides as an organization in 2020, with four strategic pillars underpinning our overall corporate strategy: (1) Steady growth through ongoing business development, punctuated by the announcement of our licensing agreement with BioInvent in the fourth quarter, (2) Continued pre-commercialization preparations ahead of anticipated China NDA filing of CNCT19, CD19 CAR-T program, (3) Continued progress with respect to our current clinical pipeline, and (4) Strategic marketing execution and physician education with our commercial product, EVOMELA. Thanks to the tireless efforts of the CASI team, we have been able to successfully drive these initiatives forward and position ourselves for continued execution excellence in 2021."

Dr. He continued, "While the ongoing pandemic continues to present an array of headwinds from a macro perspective, our plans for 2021 are no less ambitious. We will continue to evaluate potential partners and/or assets that are tactically aligned with our own strategic growth objectives. We also expect to provide several key pipeline updates this year and remain on track to initiate our CID-103 study in multiple myeloma patients in the first quarter, while closely collaborating with our partners, Juventas and BioInvent, as they continue to execute internally. Our commercial team is making extensive preparations for the launch of CNCT19, for which Juventas is expecting to file a New Drug Application (NDA) with the National Medical Product Administration (NMPA) in 2021. And finally, we are pleased by the steady growth we have observed with EVOMELA, which has benefitted substantially from our continued marketing efforts and the manufacturing change we made in the second quarter; we look forward to reporting on our continued progress in the months to come."

Preliminary and Unaudited Fourth Quarter and Unaudited Full-Year 2020 Revenues, Cash Position, and 2021 EVOMELA (melphalan for injection) Revenue Guidance

The Company anticipates that it will report EVOMELA revenue of approximately $4.8 million for the fourth quarter ended December 31, 2020, and approximately $15 million for the full-year ended December 31, 2020, exceeding its projected $14 million guidance, and representing a 269% percent increase over 2019 revenue of $4.1 million.
The Company is targeting full-year 2021 revenue guidance of more than 50% growth over 2020 for EVOMELA.
CASI expects to report approximately $57.1 million of cash and cash equivalents as of December 31, 2020.
The Company’s fourth quarter and full-year 2020 revenues are preliminary and are subject to the completion of the Company’s 2020 audit. Complete fourth quarter and full-year 2020 financial results will be reported in March.
Key Highlights for 2021

CASI plans to continue to advance pipeline products through clinical trial stages in China and globally.

As previously announced, CNCT19 received Breakthrough Therapy Designation based on initial data from the ongoing single-arm, open-label, non-randomized, dose-escalation, Phase 1 study designed to determine the safety and efficacy of CNCT19 in B-ALL. The Phase 2 registration study in patients with B-NHL is currently enrolling, and we expect Juventas to initiate the Phase II registration study in B-ALL in Q1 2021. The commercial team is making preparations for the launch of CNCT19, for which Juventas is expecting to file an NDA with the NMPA in 2021.
Recently, the Company’s partner BioInvent presented early clinical data from their Phase 1/2a trial on BI-1206. Objective responses (2CRs, 4 PRs) were demonstrated in 6 out of 9 patients evaluated, providing exciting evidence that BI-1206 has the potential to restore the activity of rituximab in non-Hodgkin’s lymphoma patients who have relapsed after treatment with rituximab. CASI intends to file an IND for BI-1206 with the NMPA in 2021 to start the clinical trials in China.
Prior to EVOMELA’s entry into the Chinese market, an average of 800 stem cell transplants per year were conducted in the multiple myeloma (MM) treatment setting. Following EVOMELA’s launch in August of 2019, CASI worked closely with KOLs to drive market awareness and expedite adoption in the Chinese market. In 2020, more than 2,600 patients were treated with EVOMELA, representing a more than threefold increase over the previous year’s median usage. CASI continues to pursue a similar strategy with respect to marketing efforts and physician visits to further accelerate the adoption of stem cell transplantation as a standard of care in the MM treatment setting and will continue working to address the persistent high unmet need in this patient population.
Based on the current environment and timetable of our clinical sites, CASI is targeting the CID-103 Phase 1 study initiation in the first quarter of 2021.
Additionally, CASI plans to continue its commitment to actively engage in business development opportunities that will bring meaningful therapies to patients that fit within its product portfolio this year.

On February 16, 2021 Ziopharm Oncology, Inc. (the "Company") (Nasdaq: ZIOP), reported the Company will host a conference call and webcast to discuss financial results for the fourth quarter and full year 2020 on Thursday, February 25, 2021 at 4:30pm EDT (Press release, Ziopharm, FEB 16, 2021, View Source [SID1234575117]).

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The live call can be accessed by dialing 1-877-451-6152 (U.S. and Canada) or 1-201-389-0879 (International). The passcode for the conference call is 13715482. The live webcast may be accessed using the link here, or by visiting the "Investors" section of the Ziopharm website at www.ziopharm.com. The call will be recorded and available for replay on the Company’s website for approximately 90 days after the call.

Additionally, the Company announced today that management will host a virtual R&D Day on Thursday, March 11, 2021 at 11:00am EDT. Details for the R&D Day event, including the agenda and guest speakers, will be discussed during the fourth quarter conference call.

On February 16, 2021 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, CRISPR Therapeutics, FEB 16, 2021, View Source [SID1234575116]).

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"2020 was a pivotal year in the growth of CRISPR Therapeutics. Data presented at ASH (Free ASH Whitepaper) and published in The New England Journal of Medicine in December of last year provided important validation of our clinical program, CTX001, in TDT and SCD, while positive top-line results from our ongoing Phase 1 CARBON trial for CTX110 targeting CD19+ B-cell malignancies, reported in October 2020, demonstrated meaningful progress for our immuno-oncology program," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "We are entering 2021 with strong momentum and look forward to further advancing our programs as we enter a new phase of growth for the company."

Dr. Kulkarni added: "In 2021, we expect to complete enrollment in the CTX001 clinical trials and provide data updates on our three clinical allogeneic CAR-T programs. Additionally, we hope to make meaningful progress in bringing our large-scale manufacturing facility online and in building our commercial infrastructure."

Recent Highlights and Outlook

Beta Thalassemia and Sickle Cell Disease

In December 2020, CRISPR Therapeutics and its partner Vertex announced positive data on a total of 10 patients treated with the investigational CRISPR/Cas9-based gene-editing therapy, CTX001, in two ongoing Phase 1/2 clinical trials, CLIMB-Thal-111 and CLIMB-SCD-121 during the Scientific Plenary Session at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.
The companies also announced in December 2020 that The New England Journal of Medicine published an independently peer-reviewed article entitled "CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β Thalassemia." The article includes detailed information on the first patient with transfusion-dependent beta thalassemia (TDT) enrolled in CLIMB-Thal-111 and the first patient with severe sickle cell disease (SCD) enrolled in CLIMB-SCD-121, at 18 and 15 months of follow-up, respectively.

Enrollment and dosing are ongoing in the clinical trials for CTX001. More than 20 patients have been dosed with CTX001 across both trials to date. Completion of enrollment in both trials is expected in 2021. Additionally, the first patient with TDT treated in CLIMB-Thal-111 recently completed two years of follow-up and has enrolled in the long termlong-term follow-up trial, CTX001-131.

Immuno-Oncology

On October 21, 2020, CRISPR Therapeutics announced positive top-line results from its ongoing Phase 1 CARBON trial assessing the safety and efficacy of several dose levels of CTX110, its wholly-owned allogeneic CAR-T investigational therapy targeting CD19+, for the treatment of relapsed or refractory B-cell malignancies. The Company expects to report additional data from this trial in 2021.

CRISPR Therapeutics’ Phase 1 clinical trial assessing the safety and efficacy of several dose levels of CTX120, its wholly-owned allogeneic CAR-T investigational therapy targeting B-cell maturation antigen for the treatment of relapsed or refractory multiple myeloma, is ongoing. The Company expects to report top-line data from this trial in 2021.

CRISPR Therapeutics’ two independent Phase 1 clinical trials assessing the safety and efficacy of several dose levels of CTX130, its wholly-owned allogeneic CAR-T investigational therapy targeting CD70, for the treatment of both solid tumors and certain hematologic malignancies, are ongoing. The Company expects to report top-line data from these trials in 2021.

Regenerative Medicine

CRISPR Therapeutics and its partner ViaCyte plan to initiate a Phase 1/2 trial of their allogeneic stem cell-derived therapy for the treatment of Type 1 diabetes in 2021. The combination of ViaCyte’s stem cell capabilities and CRISPR’s gene editing capabilities has the potential to enable a beta-cell replacement product that may deliver durable benefit to patients without requiring immune suppression.

Other Corporate Matters

Earlier this month, CRISPR Therapeutics strengthened its senior management team with the appointment of Philippe Drouet as Chief Commercial Officer.

In December 2020, CRISPR Therapeutics announced the receipt of a grant from the Bill & Melinda Gates Foundation to research in vivo gene editing therapies for the treatment of HIV. The grant builds upon CRISPR Therapeutics’ proprietary CRISPR/Cas9 gene editing technology and expertise in editing hematopoietic stem cells and contributes to efforts to accelerate transformative medicines for global health.

In October 2020, Professor Emmanuelle Charpentier, CRISPR Therapeutics’ co-founder, was awarded the 2020 Nobel Prize in Chemistry for her groundbreaking work on the CRISPR/Cas9 system. She is Founding, Scientific and Managing Director of the Max Planck Unit for the Science of Pathogens and Honorary Professor at Humboldt University, Berlin, Germany.

In July 2020, CRISPR Therapeutics announced it entered into a lease agreement with Breakthrough Properties for a new location in Boston, Massachusetts. The new facility will consolidate CRISPR’s various office and laboratory locations in the greater Boston area into a single location and support the Company’s anticipated future growth for five to seven years from the date of occupancy, which is expected in 2022.

In June 2020, CRISPR Therapeutics announced that it is building a new cell therapy manufacturing facility in Framingham, Massachusetts, for clinical and commercial production of the Company’s investigational cell therapy product candidates. The facility is being designed to provide GMP manufacturing according to U.S. Food and Drug Administration and European Medicines Agency regulations and guidelines to support clinical supply and commercial product upon potential regulatory approval.

In April 2020, CRISPR Therapeutics announced that under its June 2019 collaboration agreement with Vertex to discover and develop gene editing therapies for the treatment of Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1 (DM1), CRISPR Therapeutics received a payment of $25 million from Vertex related to the achievement of a research milestone in the DM1 program. CRISPR Therapeutics is eligible to receive additional milestone payments from Vertex of up to $800 million for these two programs.
Fourth Quarter and Full Year 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $1,690.3 million as of December 31, 2020, compared to $943.8 million as of December 31, 2019. The increase in cash of $746.6 million was primarily driven by cash from financing activities of $1,016.1 million, which consists primarily of proceeds from the Company’s July public offering and funds received from its "at-the-market" offering during 2020.

Revenue: Total collaboration revenue was $0.2 million for the fourth quarter of 2020 compared to $77.0 million for fourth quarter of 2019, and $0.5 million for the year ended December 31, 2020, compared to $289.6 million for the year ended December 31, 2019. The decrease in collaboration revenue is primarily attributable to revenue recognized in connection with the sale of certain licenses under the Company’s collaboration with Vertex during the year ended December 31, 2019.

R&D Expenses: R&D expenses were $82.4 million for the fourth quarter of 2020 compared to $48.8 million for the fourth quarter of 2019, and $266.9 million for the year ended December 31, 2020, compared to $179.4 million for the year ended December 31, 2019. The increase in expense for the year was driven by development activities supporting the advancement of the hemoglobinopathies program and wholly-owned immuno-oncology programs, as well as increased headcount and supporting facilities related expenses.

G&A Expenses: General and administrative expenses were $25.8 million for the fourth quarter of 2020 compared to $17.3 million for the fourth quarter of 2019, and $88.2 million for the year ended December 31, 2020, compared to $63.5 million for the year ended December 31, 2019. The increase in general and administrative expenses for the year was driven by headcount-related expense.

Net Income/Loss: Net loss was $107.0 million for the fourth quarter of 2020 compared to income of $30.5 million for the fourth quarter of 2019, and net loss was $348.9 million for the year ended December 31, 2020, compared to income of $66.9 million for the year ended December 31, 2019.
About CTX001
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). CTX001 has also been granted Orphan Drug Designation from the European Commission, for both TDT and SCD, as well as Priority Medicines designation from the European Medicines Agency for SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About CLIMB-Thal-111
The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-SCD-121
The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CTX110
CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON trial.

About CARBON
The ongoing Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies.

About CTX120
CTX120, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen, or BCMA. CTX120 is being investigated in an ongoing Phase 1 single-arm, multi-center, open-label clinical trial designed to assess the safety and efficacy of several dose levels of CTX120 for the treatment of relapsed or refractory multiple myeloma.

Based on progress to date in this program, CTX120 has been granted Orphan Drug designation from the FDA.

About CTX130
CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively.