On April 28, 2020 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the first quarter ended March 31, 2020 (Press release, CRISPR Therapeutics, APR 28, 2020, View Source [SID1234556776]).

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"We made substantial progress in the last quarter despite the challenges posed by COVID-19. We are now progressing five cell therapy clinical trials in parallel targeting hemoglobinopathies and various cancers," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Our CTX001 and CTX110 programs continue to enroll patients, and we expect to report data for these programs this year. We are pleased to have begun treating patients in our CTX120 trial for the treatment of relapsed or refractory multiple myeloma and we expect to begin treating patients in our CTX130 trial in renal cell carcinoma and T-cell and B-cell hematologic malignancies in the second half of this year. Despite these unprecedented times, we continue to execute on our programs and we remain focused on our commitment to patients and their families."

Recent Highlights and Outlook

Beta Thalassemia and Sickle Cell Disease

CRISPR Therapeutics and its partner Vertex remain on track to provide additional data from the two ongoing Phase 1/2 studies of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with transfusion-dependent beta thalassemia and in patients with severe sickle cell disease in 2020. New data expected in 2020 include initial data from additional patients dosed in each of the Phase 1/2 studies and longer duration follow-up data for the first patients dosed in each study. Screening, enrollment and mobilization in these studies is ongoing, however no additional patients are scheduled to initiate conditioning or dosing at this time due to COVID-19.

Immuno-Oncology

Patient enrollment continues in a clinical trial to assess the safety and efficacy of CTX110, CRISPR Therapeutics’ wholly-owned allogeneic CAR-T cell therapy targeting refractory CD19+ B-cell malignancies. The multi-center, open label clinical trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110. If successful, CTX110 could enable off-the-shelf use of cell therapies and greatly expand their applicability and accessibility in treating patients with these hematologic malignancies. The Company expects to report top-line data for CTX110 at the end of 2020.

CRISPR Therapeutics has begun treating patients in a clinical trial to assess the safety and efficacy of CTX120, its wholly-owned allogeneic CAR-T cell therapy targeting BCMA for the treatment of relapsed or refractory multiple myeloma. The multi-center, open label trial is designed to enroll up to 80 patients and investigate several dose levels of CTX120.

Earlier this year, the U.S. Food and Drug Administration (FDA) accepted CRISPR Therapeutics’ Investigational New Drug (IND) application for CTX130, its wholly-owned allogeneic CAR-T cell therapy targeting CD70 for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. Additionally, CRISPR Therapeutics has obtained approval from Health Canada for its Clinical Trial Application (CTA). The Company expects to begin treating patients with CTX130 in the second half of this year.

Other Corporate Matters

Under the June 2019 collaboration agreement with Vertex to discover and develop gene editing therapies for the treatment of Duchenne Muscular Dystrophy (DMD) 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.

CRISPR Therapeutics is scheduled to present two posters during the virtual American Society of Gene & Cell Therapy 2020 Annual Meeting, to be held from May 12 to 15, 2020, via View Source, as follows:

Title: Dual Guide CRISPR/Cas9 Editing of the CCR5 Gene Provides Complete Protection Against HIV in Humanized Mouse Models (abstract #1046)
Session Title: Gene Targeting and Gene Correction
Date and Time: Thursday, May 14, 2020; 5:30 PM – 6:30 PM

Title: Multiplexing of Up to 10 Gene Edits Using CRISPR/Cas9 to Generate CAR-T Cells with Improved Function (abstract #1151)
Session Title: Cancer – Immunotherapy, Cancer Vaccines
Date and Time: Thursday, May 14, 2020; 5:30 PM – 6:30 PM

In February, CRISPR Therapeutics announced its proposal to elect Doug Treco, Ph.D. to its Board of Directors at the Company’s upcoming annual general meeting to be held later this year.
First Quarter 2020 Financial Results

Cash Position: Cash and cash equivalents as of March 31, 2020, were $889.7 million, compared to $943.8 million as of December 31, 2019, a decrease of $54.1 million. The decrease in cash was primarily driven by cash used in operating activities of $52.2 million to support spending on the Company’s clinical and pre-clinical programs, as well as payroll and payroll-related expenses to support growth. In April 2020, the Company received a milestone payment of $25 million from Vertex under the collaboration agreement for DMD and DM1, resulting in pro forma cash exceeding $900 million.

Revenue: Total collaboration revenue was $0.2 million for the first quarter of 2020 compared to $0.3 million for first quarter of 2019. Collaboration revenue primarily consisted of charges to partners for research activities.

R&D Expenses: R&D expenses were $54.2 million for the first quarter of 2020 compared to $33.8 million for the first quarter of 2019. The increase in expenses was driven by increased headcount and development activities supporting the advancement of the hemoglobinopathies program and wholly-owned immuno-oncology programs.

G&A Expenses: General and administrative expenses were $19.6 million for the first quarter of 2020 compared to $14.9 million for the first quarter of 2019. The increase in general and administrative expenses for the year was driven by headcount-related expense and higher facilities cost.

Net Loss: Net loss was $69.7 million for the first quarter of 2020 compared to net loss of $48.4 million for the first quarter of 2019.
About CTX001
CTX001 is an investigational ex vivo CRISPR 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 and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About CTX110
CTX110 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19, or CD19, for the treatment of CD19+ malignancies. A wholly-owned asset of CRISPR Therapeutics, CTX110 is being investigated in a clinical trial designed to assess the safety and efficacy of CTX110 for the treatment of relapsed or refractory B-cell malignancies. The multi-center, open-label clinical trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110.

About CTX120
CTX120 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting B-cell maturation antigen, or BCMA. A wholly-owned asset of CRISPR Therapeutics, CTX120 is being investigated in a clinical trial designed to assess the safety and efficacy of CTX120 for the treatment of relapsed or refractory multiple myeloma. The multi-center, open-label clinical trial is designed to enroll up to 80 patients and investigate several dose levels of CTX120.

About CTX130
CTX130 is a healthy donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. A wholly-owned asset of CRISPR Therapeutics, CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies.