CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company developing transformative gene-based medicines for serious diseases, has released its financial results for Q3 ending September 30, 2024.
“We are making strong progress across our pipeline of in vivo and ex vivo CRISPR-based therapies,” stated Dr. Samarth Kulkarni, CEO and Chairman of CRISPR Therapeutics. “In addition to the ongoing success of CASGEVY’s launch, we are excited that CASGEVY has received regulatory approval in Switzerland and Canada for patients aged 12 and older with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). As we continue to advance our trials in oncology, autoimmune, diabetes, and cardiovascular fields, we anticipate key data milestones over the next 9-12 months.”
Recent Highlights and Outlook
Hemoglobinopathies and CASGEVY™ (Exa-cel)
CASGEVY has received approval for patients aged 12+ with SCD and TDT in Switzerland and Canada, adding to prior approvals in the U.S., EU, and other regions. CRISPR Therapeutics and Vertex Pharmaceuticals are collaborating on CASGEVY, with Vertex leading global development, manufacturing, and commercialization efforts. Forty-five Authorized Treatment Centers (ATCs) are now active worldwide, with approximately 40 patients having begun cell collection. Vertex has secured reimbursement agreements in the UK and early access approvals in Italy.
Two global Phase 3 studies of CASGEVY in children aged 5-11 with SCD or TDT have completed enrollment. CRISPR is also advancing next-generation approaches to expand treatment options, including an anti-CD117 ADC for targeted conditioning and in vivo hematopoietic stem cell editing.
Immuno-Oncology and Autoimmune Diseases
CRISPR’s next-generation CAR T cell therapies, CTX112™ and CTX131™, target CD19 and CD70 and have demonstrated enhanced cell expansion and cytotoxicity. Preliminary results from the Phase 1 trial of CTX112 in CD19-positive B-cell malignancies will be presented at the ASH 2024 Annual Meeting, showing a 67% overall response rate (ORR) and 44% complete response rate (CRR), with some responses lasting over six months. CTX112 is also in trials for systemic lupus erythematosus, with potential expansion to other autoimmune diseases. CTX131 continues trials in solid tumors and T cell lymphomas, with data expected in 2025.
In Vivo Programs
CRISPR’s lipid nanoparticle (LNP) platform supports in vivo CRISPR/Cas9 liver delivery, with CTX310 and CTX320 in Phase 1 trials targeting cardiovascular disease. CTX310 targets ANGPTL3 for lipid disorders, while CTX320 targets LPA for cardiovascular risk, with updates anticipated in 2025. Additional preclinical programs targeting refractory hypertension and acute hepatic porphyrias are also underway, with clinical trials expected in late 2025.
Regenerative Medicine
CTX211™, an allogeneic, gene-edited beta islet cell therapy for Type 1 Diabetes (T1D), is in Phase 1 trials. Vertex holds non-exclusive rights to CRISPR’s technology for T1D therapies, with CRISPR eligible for development milestones and royalties on future products.
Q3 2024 Financial Summary
- Cash Position: $1.94 billion as of September 30, 2024, an increase from $1.7 billion at the end of 2023, primarily from a $280 million stock offering and a $200 million milestone payment from Vertex.
- R&D Expenses: $82.2 million, down from $90.7 million in Q3 2023, due to reduced external research costs.
- G&A Expenses: $17.4 million, compared to $18.3 million in Q3 2023.
- Collaboration Expense: $11.2 million, down from $23.4 million in Q3 2023, due to CASGEVY program cost deferrals.
- Net Loss: $85.9 million, compared to $112.2 million in Q3 2023.
