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-Reported positive top-line results from the Company’s ongoing Phase 1 CARBON trial evaluating the safety and efficacy of CTX110™, targeting CD19+ B-cell malignancies-
- Received Rare Pediatric Disease designation from the
-Received Orphan Drug Designation (ODD) for Phase 1 clinical trial of CTX120™, targeting B-cell maturation antigen (BCMA)-
-Began treating patients in two Phase 1 clinical trials of CTX130™, targeting CD70 for the treatment of both solid tumors and certain hematologic malignancies-
“The Nobel Prize in Chemistry provides a timely recognition of the groundbreaking potential of CRISPR/Cas9 as our team at
Recent Highlights and Outlook
- Beta Thalassemia and Sickle Cell Disease
CRISPR Therapeuticsand 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 Congressfrom 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.
European Medicines Agencygranted 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 Commissionfor both TDT and SCD.
October 21, 2020, CRISPR Therapeuticsannounced 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 Therapeuticsreceived 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 Therapeuticscontinues 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 Therapeuticscontinues 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 Therapeuticsand ViaCyteentered 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.
- In October, Professor
Third Quarter 2020 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities of
$1.4 billionas 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 millionfor 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.
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
CTX001 is being developed under a co-development and co-commercialization agreement between
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.
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.
CTX110, a wholly owned program of
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.
CTX120, a wholly-owned asset of
Based on progress to date in this program targeting B-cell maturation antigen (BCMA), CTX120 has been granted Orphan Drug designation from the FDA.
CTX130, a wholly-owned asset of
CRISPR Forward-Looking Statement
This press release may contain a number of “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by
CRISPR THERAPEUTICS® word mark and design logo, CTX001™, CTX110™, CTX120™, and CTX130™ are trademarks and registered trademarks of
Condensed Consolidated Statements of Operations
(Unaudited, In thousands except share data and per share data)
|Three Months Ended
||Nine Months Ended
|Research and development||71,008||57,246||184,581||130,601|
|General and administrative||21,539||15,519||62,442||46,216|
|Total operating expenses||92,547||72,765||247,023||176,817|
|Income (Loss) from operations||(92,399||)||139,163||(246,674||)||35,757|
|Total other income (expense), net||160||(466||)||5,804||1,003|
|Net income (loss) before income taxes||(92,239||)||138,697||(240,870||)||36,760|
|Provision for income taxes||(200||)||(274||)||(956||)||(444||)|
|Net income (loss)||(92,439||)||138,423||(241,826||)||36,316|
|Foreign currency translation adjustment||31||(12||)||3||(14||)|
|Unrealized loss on marketable securities||(144||)||—||(144||)||—|
|Comprehensive income (loss)||$||(92,552||)||$||138,411||$||(241,967||)||$||36,302|
|Net income (loss) per common share - basic||$||(1.32||)||$||2.52||$||(3.77||)||$||0.68|
|Basic weighted-average common shares
|Net income (loss) per common share - diluted||$||(1.32||)||$||2.40||$||(3.77||)||$||0.65|
|Diluted weighted-average common shares
Condensed Consolidated Balance Sheets Data
(Unaudited, in thousands)
|Total shareholders' equity||1,343,301||939,425|
WCG on behalf of CRISPR
Source: CRISPR Therapeutics AG