Caribou Biosciences Jumps on Off-the-Shelf Cancer Therapy Data
Phase 1 results for its lead therapy show efficacy and durability in lymphoma patients comparable to existing personalized treatments, boosting its allogeneic platform.
Shares of Caribou Biosciences (NASDAQ: CRBU) surged Monday after the biotechnology company announced positive clinical data for its lead cancer treatment, positioning it as a potentially viable “off-the-shelf” alternative to complex, personalized cell therapies.
In morning trading, Caribou’s stock climbed approximately 4.5% to $2.53. The positive momentum follows the release of data from its ANTLER Phase 1 trial for vispa-cel (CB-010), an allogeneic CAR-T cell therapy designed to treat relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL).
The results provide significant clinical validation for Caribou's approach, which uses healthy donor cells to create a standardized, readily available treatment. This model aims to overcome the significant logistical and manufacturing hurdles of current autologous CAR-T therapies, which are engineered from a patient's own cells in a costly and time-consuming process.
According to the company's press release, vispa-cel demonstrated impressive efficacy and durability. In a cohort of 35 patients who received the therapy with an optimized profile, the treatment achieved an 86% overall response rate (ORR) and a 63% complete response (CR) rate. Perhaps more significantly, the 12-month progression-free survival rate was 53%, indicating a durable effect.
The therapy was also well-tolerated. Critically for an allogeneic treatment, there were no reported cases of graft-versus-host disease (GvHD), a potentially life-threatening complication where donor cells attack the patient's body.
"We believe these results demonstrate that an allogeneic CAR-T cell therapy can achieve the efficacy and durability of autologous treatments," said Rachel Haurwitz, PhD, Caribou's president and chief executive officer. She highlighted that vispa-cel's safety profile could make it suitable for outpatient administration, further broadening its potential patient access compared to the lengthy hospital stays required for many existing treatments.
This data is crucial for Caribou as it seeks to compete in a market dominated by autologous therapies from major pharmaceutical players. Autologous treatments, while effective, can take weeks to manufacture, a delay that critically ill patients may not be able to afford. An effective, pre-manufactured allogeneic therapy could represent a paradigm shift in the cell therapy landscape.
Mehdi Hamadani, MD, an investigator on the ANTLER trial, noted the significance of the findings, stating that vispa-cel's "efficacy and durability are comparable to autologous CAR-T therapies." He added that its off-the-shelf availability and tolerability profile "could expand access to CAR-T cell therapy for patients in the second-line setting and beyond."
Investors are watching these developments closely. Prior to the announcement, Wall Street analysts held a strong buy consensus on CRBU, with an average price target of $8.86, suggesting significant potential upside from its current valuation. The company's market capitalization stood at approximately $225 million following Monday's stock price increase.
Looking ahead, Caribou plans to engage with the U.S. Food and Drug Administration (FDA) to refine the design of a pivotal Phase 3 trial, the final step before seeking potential marketing approval. The successful ANTLER data provides a strong foundation for these discussions as the company advances what it hopes will be a best-in-class, off-the-shelf cancer therapy.