On October 28, 2019 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the third quarter ended September 30, 2019 (Press release, CRISPR Therapeutics, OCT 28, 2019, View Source [SID1234549924]).
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"In 2019, we’ve made significant progress across several development programs, including ongoing enrollment of our CTX001 trials in beta thalassemia and severe sickle cell disease, with preliminary data expected from these programs later this year. We also began treating patients in our clinical trial for CTX110, our allogeneic CAR-T therapy, and are advancing additional CAR-T candidates toward clinical development," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Looking forward, we expect a robust 2020, with continued focus on execution as we anticipate conducting five clinical trials in parallel."
Recent Highlights and Outlook
Beta Thalassemia and Sickle Cell Disease
Enrollment is ongoing at six clinical trial sites in the U.S., Canada and Europe for the Phase 1/2 study of CTX001 in patients with transfusion-dependent beta thalassemia (TDT) and at ten clinical trial sites in the U.S., Canada and Europe for the study in patients with severe sickle cell disease (SCD).
The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion for orphan drug designation (ODD) of CTX001 for the treatment of TDT. In addition, we are expanding the TDT patient population for CTX001 to include beta zero/beta zero subtypes.
The Company expects to release preliminary safety and efficacy data from the ongoing Phase 1/2 clinical trials in late 2019.
Immuno-Oncology
The Company has begun treating patients in a Phase 1/2 trial to assess the safety and efficacy of CTX110, its wholly-owned allogeneic CAR-T cell therapy targeting CD19+ malignancies. The multi-center, open label trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110. The study is currently enrolling at five clinical trial sites in the U.S. and Australia. In addition, the Company obtained approval from Health Canada for its Clinical Trial Application (CTA). The Company believes its CRISPR-based allogeneic CAR-Ts may have a superior product profile compared to current autologous therapies and allow accessibility to broader patient populations.
The Company expects to initiate a Phase 1/2 clinical trial of CTX120, targeting B-cell maturation antigen (BCMA) for the treatment of multiple myeloma, in the first half of 2020. CRISPR Therapeutics continues to advance additional allogeneic CAR-T candidates toward clinical development including CTX130, targeting CD70 for the treatment of solid tumors and hematologic malignancies. The Company continues to scale its capabilities to enable rapid advancement of these programs into and through the clinic.
The Company recently announced it entered into a license agreement with KSQ Therapeutics whereby CRISPR Therapeutics gained access to KSQ intellectual property (IP) for editing certain novel gene targets in its allogeneic oncology cell therapy programs, and KSQ gained access to CRISPR Therapeutics’ IP for editing novel gene targets identified by KSQ as part of its current and future eTILTM (engineered tumor infiltrating lymphocyte) cell programs. The agreement further strengthens the Company’s proprietary allogeneic CAR-T platform.
The Company will present a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference on November 9, 2019 related to single-cell RNA sequencing and functional assessment of healthy donor and cancer patient-derived T and CAR-T cells (#P187).
Regenerative Medicine
On September 17, 2019, CRISPR Therapeutics, in collaboration with ViaCyte, presented positive in vitro data for a potential immune-evasive cell replacement therapy for diabetes at the 55th Annual Meeting of the European Association for the Study of Diabetes (EASD) in Barcelona, Spain. The oral presentation included new data that demonstrate the successful differentiation of CRISPR-edited human pluripotent stem cells to pancreatic precursor cells.
Other Corporate Matters
Vertex has exercised the options granted under the collaboration it established with CRISPR Therapeutics in 2015 to in-license three additional targets for the development of treatments using CRISPR-based gene editing. The targets include the cystic fibrosis transmembrane conductance regulator (CFTR) gene and two undisclosed targets. Under the terms of the agreement, CRISPR Therapeutics will receive an upfront payment of $30 million in connection with the option exercise and has the potential to receive up to $410 million in development, regulatory and commercial milestones and royalties on net product sales for each of the three targets, and Vertex will receive exclusive rights to develop and commercialize products related to these targets globally. The research term of the Company’s 2015 collaboration with Vertex has now expired, and Vertex no longer holds rights to in-license additional targets under that agreement.
The Company recently announced proposed plans that Casebia Therapeutics, previously a joint venture between CRISPR Therapeutics and Bayer, would operate under the direct management of CRISPR Therapeutics. Upon closing of the transaction, Casebia Therapeutics would focus on the development of its lead programs in hemophilia, ophthalmology and autoimmune diseases, with Bayer having opt-in rights for two products at IND submission. The transaction is subject to negotiation and execution of definitive agreements as well as certain customary conditions. The Company and Bayer are negotiating the definitive agreements and, subject to the finalization of the definitive agreements and satisfaction of closing conditions, anticipate to close the transaction in the fourth quarter of 2019.
Third Quarter 2019 Financial Results
Cash Position: Cash as of September 30, 2019, was $629.7 million, compared to $427.9 million as of June 30, 2019, an increase of $201.8 million as increased cash operating expenses were offset by $68.6 million net proceeds from financing activities and $175 million upfront payments received from Vertex related to the 2019 collaboration agreement announced in June.
Revenues: Total collaboration revenues were $211.9 million for the third quarter of 2019 compared to $0.6 million for third quarter of 2018 with the increase primarily driven by the collaboration agreement with Vertex.
R&D Expenses: R&D expenses were $57.2 million for the third quarter of 2019 compared to $39.8 million for the third quarter of 2018. The increase was driven by increased headcount and services expense supporting the advancement of the hemoglobinopathies program, the broadening of the Company’s wholly-owned immuno-oncology portfolio, as well as increased investment in the Company’s CRISPR/Cas9 platform research and some non-cash expense related to the Company’s collaboration with Vertex.
G&A Expenses: General and administrative expenses were $15.5 million for the third quarter of 2019 compared to $10.2 million for the third quarter of 2018. The increase was driven by increased headcount-related expense and external professional and consulting service expense.
Net Income (Loss): Net Income was $138.4 million for the third quarter of 2019 compared to a loss of $50.7 million for the third quarter of 2018, driven predominantly by increased revenue recognized in connection with the Company’s collaboration with Vertex.