On February 25, 2019 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, 2018 (Press release, CRISPR Therapeutics, FEB 25, 2019, View Source [SID1234533633]).
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"This past year was truly transformational for CRISPR Therapeutics as we achieved milestones across our key programs in β-thalassemia, sickle cell disease and immuno-oncology. We’re pleased with the progress we’ve made in 2018, especially in clinical execution and the expansion of our development pipeline. We also strengthened our team with key new hires, positioning us well as we advance to the next stage of development. This progress brings us closer to realizing our mission of bringing transformative therapies to patients with serious diseases," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics.
Dr. Kulkarni added: "Over the next two years, we expect to generate data from clinical trials across multiple indications as we bring CRISPR technology to patients. In addition, we are making deliberate steps to scale the Company as we advance programs across a number of therapeutic areas while continuing to bolster our proprietary CRISPR platform."
Recent Highlights and Outlook
Hemoglobinopathies
° β-thalassemia
CRISPR Therapeutics, together with its partner, Vertex, announced that the first patient has been treated with CTX001 in a Phase 1/2 clinical study of patients with transfusion-dependent beta thalassemia (TDT), marking the first company-sponsored use of a CRISPR/Cas9 therapy in a clinical trial. The Phase 1/2 open-label trial is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT, non-beta zero/beta zero subtypes. The first two patients in the trial will be treated sequentially and, pending data from these initial two patients, the trial will open for broader concurrent enrollment. The companies plan to target presentations of data at scientific conferences once there is sufficient data on multiple patients.
° Sickle Cell Disease
CRISPR Therapeutics, together with its partner, Vertex, are also investigating CTX001 for the treatment of severe sickle cell disease (SCD) and announced that the first patient has been enrolled in a Phase 1/2 clinical study of CTX001 in severe SCD in the U.S. and is expected to be infused with CTX001 in mid-2019. The Phase 1/2 open-label trial is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. Similar to the trial in beta thalassemia, the first two patients in the trial will be treated sequentially prior to broader concurrent enrollment. The companies plan to target presentations of data at scientific conferences once there is sufficient data on multiple patients. CTX001 was recently granted Fast Track Designation by the U.S. Food and Drug Administration for the treatment of SCD.
Immuno-Oncology
° CRISPR Therapeutics is on track to initiate a clinical trial for CTX110, its wholly-owned allogeneic CAR-T cell therapy targeting CD19+ malignancies, in the first half of 2019. CRISPR/Cas9 has the potential to create the next-generation of CAR-T cell therapies that may have a superior product profile compared to current autologous therapies and allow accessibility to broader patient populations. The Company continues to advance two additional allogeneic CAR-T candidates; CTX120, targeting B-cell maturation antigen (BCMA) for the treatment of multiple myeloma; and CTX130, targeted towards CD70 for the treatment of both solid tumors and hematologic malignancies. In November, the Company presented a poster at the Society for Immunotherapy in Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting related to multiplex editing and production of allogeneic CAR-T therapies. Additionally, the Company presented a poster at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2018 Annual Meeting in December, highlighting further development and preclinical data for CTX120. The study showed maintained cytotoxic capacity over multiple in vitro re-challenges, demonstrating durable potency and reduced susceptibility to exhaustion.
Other Programs
° In September, CRISPR Therapeutics and ViaCyte, Inc. announced a collaboration focused on the discovery, development, and commercialization of gene-edited allogeneic stem cell derived islet cell progenitors which may offer curative benefit to patients with insulin-requiring diabetes. The combination of ViaCyte’s stem cell capabilities and CRISPR Therapeutics’ gene editing capabilities has the potential to enable a beta-cell replacement product that may deliver durable benefit to patients without the need for immune suppression.
° Earlier this year, CRISPR Therapeutics announced strategic collaborations with StrideBio, Inc. and ProBioGen. The Company’s collaboration with StrideBio expands upon an existing agreement to generate engineered AAV capsids with improved properties for in vivo gene editing programs and now includes additional undisclosed applications. CRISPR Therapeutics and ProBioGen announced a collaboration focused on the development of novel in vivo delivery modalities for CRISPR/Cas9 leveraging ProBioGen’s existing technology and expertise.
° In November, CRISPR Therapeutics and MaxCyte announced the expansion of an existing collaboration by entering into a non-exclusive commercial license agreement allowing CRISPR Therapeutics to deploy MaxCyte’s Flow Electroporation Technology to develop CRISPR/Cas9-based therapies in immuno-oncology. The collaboration builds on an existing agreement which allowed for the development of commercial therapeutics for hemoglobin-related diseases.
Company Building
° CRISPR Therapeutics continued to expand core capabilities in critical areas with the addition of key new talent across several functions.
° In February, CRISPR Therapeutics proposed to elect John T. Greene and Katherine A. High, M.D. to its Board of Directors at the Company’s upcoming annual general meeting to be held later this year. Together, they will bring significant strategic and operational experience to CRISPR Therapeutics.
Fourth Quarter and Full Year 2018 Financial Results
Cash Position: Cash and cash equivalents as of December 31, 2018 were $456.6 million, compared to $239.8 million as of December 31, 2017, an increase of $216.8 million, which was primarily driven by the net proceeds of $307.1 million from the sale of shares in follow-on financing rounds executed in January and September of 2018, offset by the Company’s use of $96.2 million for operating activities.
Revenue: Total collaboration revenue was $0.1 million for the fourth quarter of 2018 compared to $32.3 million for fourth quarter of 2017, and $3.1 million for the year ended December 31, 2018, compared to $41.0 million for the year ended December 31, 2017. The decrease in annual revenue is primarily attributable to deferred revenue recognized in 2017 in conjunction with the execution of the Company’s collaboration agreement with Vertex. During 2018 and going forward, Vertex funding of hemoglobinopathies programs are categorized as a contra-expense as opposed to revenue.
R&D Expenses: R&D expenses were $28.8 million for the fourth quarter of 2018 compared to $20.0 million for the fourth quarter of 2017, and $113.8 million for the year ended December 31, 2018 compared to $69.8 million for the year ended December 31, 2017. The increase in expense for the year was driven by greater investment in CRISPR’s lead hemoglobinopathies program partnered with Vertex, one-time expense associated with beginning our ViaCyte collaboration and expenses from the Company’s wholly owned immuno-oncology and in vivo programs.
G&A Expenses: General and administrative expenses were $16.5 million for the fourth quarter of 2018 compared to $11.3 million for the fourth quarter of 2017, and $48.3 million for the year ended December 31, 2018 compared to $35.8 million for the year ended December 31, 2017. The increase in general and administrative expenses for the year was driven by increased professional services and employee-related costs associated with our growing organization.
Net Income/Loss: Net loss was $47.6 million for the fourth quarter of 2018 compared to income of $0.1 million for the fourth quarter of 2017, and net loss was $165.0 million for the year ended December 31, 2018 compared to a loss of $68.4 million for the year ended December 31, 2017.