Investor enthusiasm can wax and wane -- sometimes dramatically. Consider CRISPR Therapeutics (CRSP 0.34%), which is trading for around $61 a share, down from its high of $220.20 on Jan. 15, 2021.
But there is hope yet. The company is on the cusp of having its first therapy approved by the Food and Drug Administration (FDA). That therapy, exa-cel, was developed by CRISPR and Vertex Pharmaceuticals as a one-time curative treatment for transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD).
Ten years from now, if the company makes this leap from a clinical-stage biotech to a commercial one, it likely will be trading for as much as $300 a share. Here's why.
Exa-cel had strong performances in phase 2/3 trials, ending painful vaso-occlusive crises and the need for blood transfusions in all 31 SCD patients who were dosed with it. In another trial to treat TDT, 42 of the 44 people who took the therapy no longer needed transfusions, and the other two needed far fewer transfusions.
The therapy takes a patient's own hematopoietic stem cells, then edits them to make high levels of fetal hemoglobin red blood cells, essentially engineering the body to end a genetic defect. There are fewer than 200,000 people in the U.S. with SCD, and fewer than 5,000 with beta thalassemia, according to the National Center for Advancing Translational Sciences.
CRISPR and Vertex turned in their biologics license applications (BLAs) of exa-cel to treat SCD and TDT this spring and on June 8, the FDA said it had accepted the BLAs, granting priority review for exa-cel as an SCD therapy, with a Prescription Drug User Fee Act (PDUFA) date for Dec. 8. The FDA granted a standard review for the drug as a TDT therapy, setting a PDUFA date of March 30, 2024.
That means, as early as next year, CRISPR could be earning product revenue with the company receiving 40% of the cut and Vertex the rest. And that's just the start. The curative gene-editing therapies that CRISPR is developing are revolutionary compared to traditional palliative care. For now, it is looking at fixing maladies that involve a gene with a single mutation, but that's just the start. Even at a high cost, gene-editing therapies that, in essence, cure diseases could be more cost effective over a patient's lifetime, particularly for chronic conditions.
Exa-cel could be a blockbuster therapy for CRISPR, ending the lifelong need for blood transfusions for certain SCD and TDT patients. The Institute for Clinical and Economic Review (ICER) recently said the therapy could be worth $1.9 million per treatment. At that price, it wouldn't take long for CRISPR to turn a profit.
Looking down the line for more approvals
If exa-cel is approved, it unlocks the rest of the company's pipeline because it serves as a proof of concept for the company's gene-editing process. A decade from now, it's possible that the company could have several blockbuster therapies.
One of them could be CTX310, which is expected to enter into clinical trials this year. It is one of the company's few in-vivo therapies, which apply therapeutic genes, gene modulators, and gene-editing tools directly into a patient's cells. CTX310 targets the angiopoietin-related protein 3 (ANGPTL3) to reduce the risk of cardiovascular disease. The protein is often linked to higher rates of coronary artery disease.
Whereas SCD and TDT are relatively rare, coronary artery disease is the most common type of heart disease in the United States, killing 375,476 people in the U.S. in 2021, according to the Centers for Disease Control and Prevention.
Another therapy with strong potential is CTX110, which is being tested to treat B-cell cancers, including B-cell lymphomas, acute lymphoblastic leukemia (ALL), and chronic lymphocytic leukemia (CLL ). The drug did well in a phase 1 trial to treat large B-cell lymphoma, showing an objective response rate of 67% and a complete response rate of 41% in heavily pretreated patients.
CRISPR also has four chimeric T-cell (CAR-T) therapies -- CTX119, CTX130, CTX112, and CTX131 -- in the works as oncology therapies. They target specific proteins to inhibit tumors or prod an immune response.
Not your typical clinical-stage biotech
Because of collaboration revenue, CRISPR is in a much better financial position than most clinical-stage biotech companies that don't have a product yet. As of the first quarter, CRISPR had $1.89 billion in cash, enough to fund operations for at least three more years. If exa-cel is approved, the company will be able to put that cash to use toward more research and development.
Nobody knows exactly where CRISPR will be in 10 years, but compared to most clinical-stage biotechs, there's at least a road map of where the company could be. The company has already shown the wherewithal to advance its science. The key for CRISPR is how well it handles the jump to marketing and producing its products.
