ZUG, Switzerland and BOSTON - CRISPR Therapeutics (NASDAQ:CRSP), a leader in gene-based medicine with a market capitalization of $4.3 billion, reported significant advancements in their ongoing Phase 1/2 clinical trial of CTX112™ for CD19-positive B-cell malignancies. According to InvestingPro data, the company maintains a strong financial position with more cash than debt on its balance sheet. The company announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX112 for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma.
The trial data, presented at the 2024 American Society of Hematology Annual Meeting, showed that CTX112, an allogeneic CAR T cell therapy, was well-tolerated and effective in producing complete remissions in patients who have undergone multiple prior treatments. The study included 12 subjects, with doses ranging from 30 x 106 to 600 x 106 CAR+ T cells. The results indicated a 67% objective response rate and a 50% complete response rate across all dose levels.
Safety profiles were promising with no reported cases of Grade ≥3 infections or Graft versus Host Disease (GvHD). All observed cases of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were manageable and of low grade. InvestingPro analysis shows that 12 analysts have recently revised their earnings expectations upward, suggesting growing confidence in the company's clinical programs. Discover more insights with InvestingPro's comprehensive research report, available for over 1,400 US stocks.
The RMAT designation by the FDA is part of a program to expedite the development and review of regenerative medicine therapies that show potential to address unmet medical needs for serious conditions. This status allows for more frequent interactions with the FDA and potential for accelerated approval.
CTX112 is also being evaluated in a Phase 1 clinical trial for systemic lupus erythematosus (SLE), with the possibility of expanding into additional autoimmune indications in the future. The company expects to provide a comprehensive update on its oncology and autoimmune programs in mid-2025.
CRISPR Therapeutics has previously achieved a milestone with the approval of the first CRISPR-based therapy in 2023 and continues to explore gene editing as a means to develop treatments for a variety of diseases. The company reported revenue of $202.8 million in the last twelve months, with a notably strong liquidity position reflected in a current ratio of 21.64. Given the stock's beta of 1.63, investors should note its higher volatility compared to the broader market. Based on InvestingPro Fair Value analysis, the stock is currently fairly valued. The latest findings support the potential of CTX112 as an off-the-shelf CAR T therapy option and highlight the company's commitment to innovative treatments for patients with significant medical needs.
The information reported is based on a press release statement from CRISPR Therapeutics.
In other recent news, CRISPR Therapeutics has experienced several important developments. TD Cowen has maintained its Sell rating on the company, based on recent performance and clinical data, while Oppenheimer and Leerink Partners have reiterated their Outperform ratings, with price targets of $95.00 and $67.00 respectively. CRISPR Therapeutics' third-quarter financial results disclosed operating expenses of $110.1 million and a cash position of $1.94 billion.
The company, in collaboration with Vertex Pharmaceuticals (NASDAQ:VRTX), continues to progress with the launch of the gene-editing therapy Casgevy, which has seen an increase in patient engagement. Despite this, no revenue was generated from the infusion of Casgevy in one patient during the third quarter. However, Needham revised its sales projections for Casgevy, forecasting $17 million for the fiscal year 2024, a decrease from the initially expected $43 million.
CRISPR Therapeutics is also making strides with its in vivo, immuno-oncology, and autoimmune pipelines, with CTX131 and CTX112 entering Phase I studies. The company's ASH abstract for CTX112 reported a 67% overall response rate and a 44% complete response rate among nine patients. The recent developments indicate a focus on increasing patient numbers and further differentiating its clinical data in the competitive field of gene editing therapies.
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