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-Achieved target enrollment in CTX001 clinical trials for beta thalassemia (TDT) and sickle cell disease (SCD); regulatory submissions planned for late 2022-
-Reported positive results from the ongoing Phase 1 CARBON clinical trial evaluating the safety and efficacy of CTX110 for CD19+ B-cell malignancies; enrollment continues, with potential registrational trial incorporating consolidation dosing expected to initiate in Q1 2022-
-Implementing consolidation dosing protocols for CTX120™ and CTX130™ clinical trials; enrollment continues, with top-line data expected to report in 1H 2022-
-Regenerative medicine and in vivo programs continue to progress and remain on track-
“The third quarter marked significant progress across our portfolio,” said
Recent Highlights and Outlook
- Beta Thalassemia and Sickle Cell Disease
- Data presented to date for 22 patients with greater than 3 months of follow-up support the profile of CTX001 as a one-time functional cure for patients with TDT and severe SCD, showing consistent and durable benefit across all treated patients.
- Target enrollment has been achieved in the ongoing clinical trials for CTX001 in TDT and SCD, with planned regulatory submissions in late 2022.
October 12, 2021, CRISPR Therapeuticsannounced positive results from its ongoing Phase 1 CARBON trial evaluating the safety and efficacy of CTX110, its wholly-owned allogeneic chimeric antigen receptor T cell (CAR-T) investigational therapy targeting CD19+ B-cell malignancies. The data showed early evidence of a dose dependent response to CTX110, with overall response rates (ORR), complete response rates (CR) and durability similar to approved autologous CD19 CAR-T therapies on an intent-to-treat (ITT) basis. A single dose of CTX110 at DL2 and above resulted in a 58% ORR and 38% CR rate in large B-cell lymphoma (LBCL) patients on an ITT basis. The pharmacokinetic data provide a strong rationale that consolidation dosing can improve on an already competitive profile for CTX110. Based on the safety and efficacy profile, the Company plans to expand into a potential registrational trial that incorporates consolidation dosing in Q1 2022.
- In addition to CTX110,
CRISPR Therapeuticshas ongoing Phase 1 clinical trials assessing safety and efficacy of several dose levels for the following CAR-Ts: (i) CTX120, its wholly-owned allogeneic CAR-T investigational therapy targeting B-cell maturation antigen for the treatment of relapsed or refractory multiple myeloma; and (ii) CTX130, its wholly-owned allogeneic CAR-T investigational therapy targeting CD70 for the treatment of both solid tumors and certain hematologic malignancies. Based on the learnings from CTX110, the Company is implementing consolidation dosing protocols for its CTX120 and CTX130 clinical trials and expects to report top-line data in the first half of 2022.
- In October,
CRISPR Therapeuticsannounced two poster presentations at the Society for Immunotherapy of Cancer(SITC) 36th Annual Meeting, to be held both virtually and at the Walter E. Washington Convention Centerin Washington, D.C., from November 10 to 14, 2021. The Company also announced an oral presentation at the SITC 2021 Pre-Conference Program, The Evolution of Immunotherapy: An Exploration of Immunity Beyond T cells, CAR T in Solid Tumors and Novel Combinations, which will be held from 2:00 p.m.– 6:00 p.m. ETon November 10, 2021.
- Regenerative Medicine and In
CRISPR Therapeuticsand its partner ViaCyteremain on track to initiate a Phase 1/2 trial of their allogeneic stem cell-derived therapy for the treatment of Type 1 diabetes in 2021. 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 requiring immune suppression.
- The Company continues to make progress with its in vivo approaches for liver gene editing. The Company expects to move multiple programs utilizing in vivo approaches into the clinic in the next 18 to 24 months.
- Other Corporate Matters
- In October,
CRISPR Therapeuticsannounced the appointment of Brendan Smithas Chief Financial Officer. Mr. Smithbrings more than 20 years of financial, operational and strategic leadership experience, most recently as the Chief Financial Officer of Translate Bio.
- In October,
Third Quarter 2021 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were
$2,477.4 millionas of September 30, 2021, compared to $2,589.4 millionas of June 30, 2021. The decrease in cash of $112.0 millionwas primarily driven by cash used in operating activities to support ongoing research and development of the Company’s clinical and pre-clinical programs.
- Revenue: Total collaboration revenue was
$0.3 millionfor the third quarter of 2021, compared to $0.1 millionfor the third quarter of 2020. Collaboration revenue primarily consisted of revenue recognized in connection with our collaboration agreements with Vertex.
- R&D Expenses: R&D expenses were
$105.3 millionfor the third quarter of 2021, compared to $71.0 millionfor the third quarter of 2020. The increase in expense was driven by development activities supporting the advancement of the hemoglobinopathies program and wholly-owned immuno-oncology programs, as well as increased headcount and supporting facilities related expenses.
- G&A Expenses: General and administrative expenses were
$24.4 millionfor the third quarter of 2021, compared to $21.5 millionfor the third quarter of 2020. The increase in general and administrative expenses for the year was primarily driven by headcount-related expense.
- Net Loss: Net loss was
$127.2 millionfor the third quarter of 2021, compared to a net loss of $92.4 millionfor the third quarter of 2020.
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 edited 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 or eliminate transfusion requirements for patients with TDT and reduce or eliminate painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published as a Brief Report in
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
Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.
About the CRISPR-Vertex Collaboration
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.
This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.
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 program of
CTX130, a wholly-owned program of
CRISPR THERAPEUTICS® word mark and design logo, CTX001™, CTX110™, CTX120™, and CTX130™ are trademarks and registered trademarks of
CRISPR Therapeutics 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
Condensed Consolidated Statements of Operations
(Unaudited, In thousands except share data and per share data)
|Three Months Ended
||Nine Months Ended
|Research and development||105,321||71,008||304,163||184,581|
|General and administrative||24,352||21,539||78,675||62,442|
|Total operating expenses||129,673||92,547||382,838||247,023|
|(Loss) income from operations||(128,849||)||(92,399||)||519,226||(246,674||)|
|Total other income, net||1,101||160||3,806||5,804|
|Net (loss) income before income taxes||(127,748||)||(92,239||)||523,032||(240,870||)|
|Benefit (provision) for income taxes||595||(200||)||(4,123||)||(956||)|
|Net (loss) income||(127,153||)||(92,439||)||518,909||(241,826||)|
|Foreign currency translation adjustment||(24||)||31||(14||)||3|
|Unrealized loss on marketable securities||(117||)||(144||)||(673||)||(144||)|
|Comprehensive (loss) income||$||(127,294||)||$||(92,552||)||$||518,222||$||(241,967||)|
|Net (loss) income per common share — basic||$||(1.67||)||$||(1.32||)||$||6.85||$||(3.77||)|
|Basic weighted-average common shares outstanding||76,288,534||70,143,481||75,712,437||64,159,224|
|Net (loss) income per common share — diluted||$||(1.67||)||$||(1.32||)||$||6.44||$||(3.77||)|
|Diluted weighted-average common shares outstanding||76,288,534||70,143,481||80,554,682||64,159,224|
Condensed Consolidated Balance Sheets Data
(Unaudited, in thousands)
|Total shareholders' equity||2,512,923||1,664,234|
Source: CRISPR Therapeutics AG