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-Enrollment ongoing in Phase 1/2 clinical trials of CTX001™ for patients with severe hemoglobinopathies-
-IND and CTA approved for CTX110™, wholly-owned allogeneic CAR-T cell therapy targeting CD19+ malignancies-
-On track to initiate Phase 1/2 clinical trial for CTX110 in 1H 2019-
-$437.5 million in cash as of
“This past quarter, we began an important new period for
Recent Highlights and Outlook
- Beta thalassemia
April 2019, CRISPR Therapeuticsand its partner, Vertex, announced that the U.S. Food and Drug Administration( FDA) had granted Fast Track Designation for CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT). In February 2019, CRISPR and Vertex announced that the first patient had been treated with CTX001 in a Phase 1/2 clinical study of patients with TDT, marking the first company-sponsored use of a CRISPR/Cas9 therapy in a clinical trial. Enrollment in the Phase 1/2 study in patients with TDT is ongoing.
- Sickle Cell Disease
The companies are also evaluating CTX001 for the treatment of sickle cell disease (SCD) and received Fast Track Designation for CTX001 from the
FDAin January 2019for SCD. The companies announced in February 2019that the first patient had been enrolled in a Phase 1/2 clinical study of CTX001 in severe SCD in the U.S. and is expected to be infused with CTX001 in mid-2019. Enrollment in the Phase 1/2 study in patients with SCD is ongoing.
- Beta thalassemia
Earlier this year, the
FDAapproved CRISPR Therapeutics’ Investigational New Drug (IND) application for CTX110, its wholly-owned allogeneic CAR-T cell therapy targeting CD19+ malignancies. Additionally, the Company has obtained approval from Health Canadafor its Clinical Trial Application (CTA). CRISPR Therapeuticsremains on track to initiate a Phase 1/2 trial to assess the safety and efficacy of CTX110 in the first half of 2019 and continues to work closely with various clinical sites to begin the trial. The Company’s proprietary CRISPR-based allogeneic CAR-Ts have the potential to create the next-generation of cell therapies that may have a superior product profile compared to current autologous therapies and allow accessibility to broader patient populations. CRISPR Therapeuticscontinues to advance additional allogeneic CAR-T candidates including: CTX120™, targeting B-cell maturation antigen (BCMA) for the treatment of multiple myeloma; and CTX130™, targeting CD70 for the treatment of solid tumors and hematologic malignancies. Earlier this month, the Company presented preclinical data targeting multiple solid tumor types with anti-CD70 allogeneic CAR-T cells, further demonstrating consistent expression, durability, selectivity and potent cell killing. The Company also presented data targeting CD33 that showed potent preclinical activity against acute myeloid leukemia (AML) cells. These data were presented during poster sessions at the 2019 American Association for Cancer Research(AACR) Annual Meeting. CRISPR Therapeuticswill present additional data at the upcoming 2019 American Society of Gene and Cell Therapy(ASGCT) Annual Meeting demonstrating the potential of CRISPR/Cas9 allogeneic CAR-T cell candidates for multiple oncology targets. CRISPR/Cas9 allogeneic CAR-T cells show consistently high percent CAR expression, while maintaining durable potency, low exhaustion, and lack of alloreactivity (#841). The Company will also present data evaluating homology-independent CRISPR/Cas9 off-target assessments methods (#134).
- Corporate Development
- The U.S. Patent Office issued four patents to Dr.
Emmanuelle Charpentier, together with The Regents of the University of Californiaand University of Vienna(collectively, CVC). Additionally, CVC was recently granted its third patent in Europe. These patents expand CVC’s wide-ranging CRISPR-Cas9 gene-editing patent portfolio.
- Earlier this year,
CRISPR Therapeuticsannounced strategic collaborations with StrideBio, Inc.and ProBioGen. The Company continues to examine new technologies and capabilities in support of existing programs.
- In February,
CRISPR Therapeuticsproposed to elect John T. Greeneand Katherine A. High, M.D. to its Board of Directors at the Company’s upcoming annual general meeting to be held later this year. Together, they will bring significant strategic and operational experience to CRISPR Therapeutics.
- The U.S. Patent Office issued four patents to Dr.
First Quarter 2019 Financial Results
- Cash Position: Cash as of
March 31, 2019, was $437.5 million, compared to $456.6 millionas of December 31, 2018, a decrease of $19.1 million. Cash used in operations drove the decrease, offset by $25.7 millionin net cash provided by financing activities during the quarter.
- Revenues: Total collaboration revenues were
$0.3 millionfor the first quarter of 2019 compared to $1.4 millionfor first quarter of 2018. CRISPR’s collaboration revenue is attributable to its research partnerships with Casebia and Vertex. Cost sharing on the Vertex co-development and co-promotion agreement related to hemoglobinopathies is not included in revenue, but instead as an offset to expense in R&D.
- R&D Expenses: R&D expenses were
$33.8 millionfor the first quarter of 2019 compared to $19.5 millionfor the first quarter of 2018. The increase was driven by headcount and services expense supporting the advancement of the hemoglobinopathies program, the broadening of the wholly-owned immuno-oncology portfolio, as well as increased investment in the Company’s CRISPR/Cas9 platform research.
- G&A Expenses: General and administrative expenses were
$14.9 millionfor the first quarter of 2019 compared to $8.8 millionfor the first quarter of 2018. The increase was driven by headcount-related expense and external professional and consulting service expense.
- Net Loss: Net loss was
$48.4 millionfor the first quarter of 2019 compared to a loss of $28.3 millionfor the first quarter of 2018, driven predominantly by increased R&D expense in the quarter.
CRISPR Forward-Looking Statement
This press release may contain a number of “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics’ expectations about any or all of the following: (i) clinical trials (including, without limitation, the timing of filing of clinical trial applications and INDs, any approvals thereof and the timing of commencement of clinical trials), development timelines and discussions with regulatory authorities related to product candidates under development by
Condensed Consolidated Statements of Operations
(Unaudited, In thousands except share data and per share data)
|Three Months Ended March 31,|
|Research and development||33,822||19,519|
|General and administrative||14,929||8,836|
|Total operating expenses||48,751||28,355|
|Loss from operations||(48,423||)||(26,997||)|
|Total other (expense) income, net||100||(1,217||)|
|Net loss before income taxes||(48,323||)||(28,214||)|
|Provision for income taxes||(85||)||(86||)|
|Foreign currency translation adjustment||8||12|
|Reconciliation of net loss to net loss attributable to common shareholders:|
|Net loss per share attributable to common shareholders - basic and diluted||$||(0.93||)||$||(0.62||)|
|Weighted-average common shares outstanding used in calculating net loss per share attributable to common shareholders - basic and diluted||52,093,208||45,877,428|
Condensed Consolidated Balance Sheets Data
(Unaudited, in thousands)
|March 31, 2019||December 31, 2018|
|Total shareholders' equity||379,810||392,195|
WCG on behalf of CRISPR
Source: CRISPR Therapeutics AG