On October 28, 2020 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, 2020 (Press release, CRISPR Therapeutics, OCT 28, 2020, View Source [SID1234569213]).

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"The Nobel Prize in Chemistry provides a timely recognition of the groundbreaking potential of CRISPR/Cas9 as our team at CRISPR Therapeutics continues to make important progress in our immuno-oncology and hemoglobinopathies clinical trials. Earlier this month, we were pleased to report positive top-line data from our Phase 1 CARBON trial of CTX110 in patients with relapsed or refractory B-cell malignancies, which demonstrate the potential of CRISPR gene editing for the treatment of cancers. Enrollment in our CTX120 and CTX130 trials continue and we expect to report initial data for these programs next year," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "In parallel, our clinical trials in hemoglobinopathies continue to advance rapidly and we expect to report additional data from our CTX001 program later this year."

Recent Highlights and Outlook

Beta Thalassemia and Sickle Cell Disease

CRISPR Therapeutics and Vertex previously announced that, as of June 2020, seven patients had been dosed across its two Phase 1/2 studies of the investigational CRISPR/Cas9 gene-editing therapy CTX001 and presented data at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress from two TDT patients and one SCD patient. Additional patients have been enrolled and dosed in both TDT and SCD studies and the Company expects to report clinical data from more patients treated with CTX001 in addition to data from patients with longer follow-up in the fourth quarter.

The European Medicines Agency granted Priority Medicines (PRIME) designation to CTX001 for the treatment of severe SCD. CTX001 has also been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA and ODD from the European Commission for both TDT and SCD.

Immuno-Oncology

On October 21, 2020, CRISPR Therapeutics announced positive top-line results from its ongoing Phase 1 CARBON trial evaluating the safety and efficacy of CTX110, its wholly-owned allogeneic CAR-T investigative therapy targeting CD19+ B-cell malignancies. The data showed early evidence of a dose dependent response to CTX110 and, at DL3, demonstrated a 50% (2/4) complete response (CR) rate and an acceptable safety profile at DL3 or below. Both responders at DL3 remained in CR at the 3-month assessment. The Company expects to report additional data from this trial in 2021.

CRISPR Therapeutics received ODD from the FDA for CTX120, the Company’s wholly-owned allogeneic CAR-T investigative therapy targeting BCMA for the treatment of relapsed or refractory multiple myeloma. CRISPR Therapeutics continues to enroll and dose patients in a Phase 1 clinical trial assessing the safety and efficacy of CTX120. The Company expects to report top-line data from this trial in 2021.

CRISPR Therapeutics continues to enroll and dose patients in two independent Phase 1 clinical trials assessing the safety and efficacy of CTX130, the Company’s wholly-owned allogeneic CAR-T investigative therapy targeting CD70 for the treatment of both solid tumors and certain hematologic malignancies. The Company expects to report top-line data from this trial in 2021.

Regenerative Medicine

CRISPR Therapeutics, together with its partner ViaCyte, are planning to initiate a Phase 1/2 trial of its allogeneic stem cell-derived therapy for the treatment of Type 1 diabetes in 2021. CRISPR Therapeutics and ViaCyte entered into a strategic collaboration focused on the development and commercialization of novel regenerative medicines including gene-edited allogeneic stem cell-derived therapies for the treatment of diabetes in 2018. 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

In October, 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.
Third Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities of $1.4 billion as of September 30, 2020, compared to $945.1 million as of June 30, 2020, an increase of $420.1 million. The increase in cash was primarily driven by our July public offering, which resulted in net proceeds of approximately $484.8 million, offset by cash used for operations to support spending on the Company’s clinical and pre-clinical programs, as well as payroll and payroll-related expenses to support growth.

Revenue: Total collaboration revenue was $0.1 million for the third quarter of 2020 compared to $211.9 million for third quarter of 2019, which resulted from the collaboration agreements with Vertex effective in the third quarter of 2019.

R&D Expenses: R&D expenses were $71.0 million for the third quarter of 2020 compared to $57.2 million for the third quarter of 2019. The increase in expenses was driven by increased headcount and supporting facilities related expenses, as well as development activities supporting the advancement of the hemoglobinopathies program and wholly-owned immuno-oncology programs.

G&A Expenses: General and administrative expenses were $21.5 million for the third quarter of 2020 compared to $15.5 million for the third quarter of 2019. The increase in general and administrative expenses for the year was driven by headcount-related expense.

Net Loss: Net loss was $92.4 million for the third quarter of 2020 compared to net income of $138.4 million for the third quarter of 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 (RMAT), 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 (PRIME) designation from the European Medicines Agency (EMA) 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-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-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 investigative therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the 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 asset of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigative 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 targeting B-cell maturation antigen (BCMA), CTX120 has been granted Orphan Drug designation from the FDA.

About CTX130
CTX130, a wholly-owned asset of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigative 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.