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-Initiated Phase 1/2 Clinical Trial of CTX001 in β-thalassemia-
-Targeting Initiation of Clinical Trial for CTX110, Targeting CD19+ Malignancies, in 1H 2019-
“As we enter the fourth quarter, we are actively moving our programs forward and expanding our clinical development activities in sickle cell disease, β-thalassemia and immuno-oncology,” said
Recent Highlights and Outlook
- Continued clinical development of CTX001 for the treatment of β-thalassemia and sickle cell disease (SCD).
º β-thalassemia: Enrollment for a Phase 1/2 trial in people with β-thalassemia is currently open at multiple clinical trial sites in
Europe, and the first patient has now enrolled in this trial. The Phase 1/2 trial is designed to assess safety and efficacy in adult transfusion-dependent non-beta zero/beta zero β-thalassemia patients. The first two patients in the trial will be dosed sequentially and, pending data from these initial two patients, the trial will be open for broader concurrent enrollment. This trial is designed to enroll up to 45 patients.
º Sickle Cell Disease: In the U.S.,
CRISPR Therapeutics, together with its partner, Vertex, recently announced that the FDAlifted the clinical hold on the CTX001 Investigational New Drug application (IND) for the treatment of SCD that was submitted earlier this year. The companies previously received regulatory approval to conduct a Phase 1/2 SCD in multiple countries in Europeand Canada. Similar to the trial in β-thalassemia, the first two patients in the trial will be dosed sequentially and, pending data from these initial two patients, the trial will be open for broader concurrent enrollment. This trial is designed to enroll up to 45 patients.
- Advancement of wholly-owned allogeneic CRISPR-based CAR-T cell therapies.
CRISPR Therapeuticsbelieves CRISPR/Cas9 has the potential to create the next-generation of CAR-T cell therapies that may have a superior product profile compared to current autologous therapies and allow broader accessibility for patient populations. The Company is targeting the initiation of a clinical trial for CTX110, its lead allogeneic CAR-T cell therapy targeted toward CD19+ malignancies, in the first half of 2019. CRISPR Therapeuticsis also advancing two additional allogeneic CAR-T candidates, targeting BCMA for multiple myeloma and CD70 for both hematologic malignancies and solid tumors. CRISPR Therapeuticswill present a poster at the Society for Immunotherapy of Cancer (SITC) Conference, November 7– 11, related to multiplex editing and production of allogeneic CAR-T therapies (#P279). Additionally, the Company will present a poster at the upcoming American Society of Hematology(ASH) 2018 Annual Meeting on December 1, highlighting further development and preclinical data for CTX120, an allogeneic “off the shelf” CAR-T cell, targeting BCMA in patients with multiple myeloma. The study showed consistent and high percent CAR expression while retaining cytotoxic capacity over multiple in vitro re-challenges, demonstrating durable potency and lack of exhaustion (#1921).
- Entered strategic collaboration with ViaCyte.
CRISPR Therapeuticsand ViaCyte, Inc.entered into a collaboration focused on the discovery, development, and commercialization of gene-edited allogeneic stem cell derived islet cell progenitors which may offer curative benefit to patients with insulin-requiring diabetes. CRISPR gene editing offers the potential to protect the transplanted cells from the patient’s immune system by ex vivo editing immune-modulatory genes within the stem cell line used to produce the pancreatic-lineage cells. 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 the need for immune suppression.
- Completed successful public offering. In September,
CRISPR Therapeuticsannounced the completion of a public offering of 4,210,526 common shares at a public offering price of $47.50per share, resulting in net proceeds of approximately $185.7 millionafter deducting underwriting discounts and commissions, original issuance taxes and offering expenses.
- Strengthened global patent portfolio with the issuance of a second patent, U.S. Patent No. 10,113,167 (“the ‘167 patent") by the U.S. Patent and Trademark Office (USPTO). The Regents of the
University of California, the University of Viennaand Emmanuelle Charpentier, Ph.D. (collectively, "UC") were granted the ‘167 patent in October 2018. This patent, together with U.S. Patent Number 10,000,772 (“the ‘772 patent”) granted to UC in June 2018, cover optimized dual- and single-guide RNA compositions of CRISPR/Cas9 and uses thereof in various environments, including eukaryotic cells and human therapeutics compositions. Both the ‘167 and the ’772 patents claim priority to a U.S. provisional application, filed by UC on May 25, 2012.
Third Quarter 2018 Financial Results
- Cash Position: Cash as of
September 30, 2018was $487.3 million, compared to $319.7 millionas of June 30, 2018, an increase of $167.6 million. The increase in cash was primarily driven by the September follow-on offering.
- Revenues: Total collaboration revenues were
$0.6 millionfor the third quarter of 2018 compared to $2.4 millionfor the third quarter of 2017. CRISPR's collaboration revenue is primarily attributable to revenue recognized for work outside the hemoglobinopathies programs. 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
$39.8 millionfor the third quarter of 2018 compared to $17.8 millionfor the third quarter of 2017. The increase of $22.0 millionin expense was primarily due to CRISPR Therapeutics’ $15 millioninvestment in the ViaCyte collaboration as well as increasing expenditures on hemoglobinopathy and immuno-oncology programs.
- G&A Expenses: General and administrative expenses were
$10.2 millionfor the third quarter of 2018 compared to $8.1 millionfor the third quarter of 2017. The increase of $2.1 millionin expense was primarily driven by legal services expense and headcount related expense.
- Net Loss: Net loss was
$50.7 millionfor the third quarter of 2018 compared to a loss of $24.7 millionfor the third quarter of 2017, driven by the increase in R&D and G&A expense described above.
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
|Nine Months Ended
|Research and development||39,820||17,845||84,972||49,770|
|General and administrative||10,175||8,112||31,752||24,522|
|Total operating expenses||49,995||25,957||116,724||74,292|
|Loss from operations||(49,432||)||(23,570||)||(113,715||)||(65,620||)|
|Total other (expense) income, net||(1,142||)||(430||)||(3,357||)||(1,548||)|
|Net loss before income taxes||(50,574||)||(24,000||)||(117,072||)||(67,168||)|
|Provision for income taxes||(137||)||(707||)||(319||)||(1,330||)|
|Foreign currency translation adjustment||(6||)||8||(15||)||38|
|Reconciliation of net loss to net loss attributable to common shareholders:|
|Net loss per share attributable to common
shareholders - basic and diluted
|Weighted-average common shares outstanding used in calculating net loss per share attributable to common shareholders - basic and diluted||47,391,988||40,088,718||46,709,388||39,904,863|
Condensed Consolidated Balance Sheets Data
(Unaudited, in thousands)
|September 30, 2018||December 31, 2017|
|Total shareholders' equity||417,314||187,832|
WCG on behalf of CRISPR
Source: CRISPR Therapeutics AG