Crispr Therapeutics expects to make history in 2023 with the first-ever request for approval of a gene-editing drug. The bid could stoke long beleaguered CRSP stock.
The company shares a name with CRISPR gene-editing technology. CRISPR, the technology, is a method of permanently altering the genes at the center of inherited diseases. So far, nothing has come close to crossing the finish line for regulatory approval.
But Crispr, the company, and partner Vertex Pharmaceuticals hope to change that with what they believe to be a cure for two blood diseases.
Samarth Kulkarni, Crispr's chief executive, says the requests for approval in the U.S. and Europe will be a milestone for the entire genetic medicines space. He recalled the approval of the first recombinant protein almost 40 years ago from Roche's Genentech.
"It had all the same dynamics as CRISPR because people weren't sure that proteins (could be) a therapeutic modality," he told Investor's Business Daily. "Today, proteins and antibodies form 50% of the entire pharma market. I believe that gene editing and CRISPR will be a mainstay in medicine and wouldn't be surprised if, in the next 10 years, it accounts for a third of the full pharma market."
CRSP Stock Ramps After Presentation
Crispr and Vertex have tested their gene-editing approach in patients with sickle cell disease and beta thalassemia, a pair of devastating blood diseases. In sickle cell, patients experience episodes of pain called vaso-occlusive crises. Beta thalassemia patients often depend on regular blood transfusions.
The treatment involves editing a patient's own stem cells to produce high levels of fetal hemoglobin, a protein that carries oxygen within red blood cells. The companies hope doing so will correct abnormal or low levels of adult hemoglobin in patients with these diseases.
In a recent presentation, Crispr and Vertex noted that 42 out of 44 beta thalassemia patients who received their gene-editing drug no longer needed transfusions. One had been transfusion-free for more than three years. The two patients who still needed transfusions had a 75%-89% drop in transfusion volume.
None of the 31 sickle cell patients experienced vaso-occlusive crises following treatment. At the time, one patient was nearly three years out from treatment. Since the data cutoff in February, that patient has exceeded the three-year mark.
Notably, both groups of patients showed high and sustained levels of fetal hemoglobin. While two patients with beta thalassemia experienced serious side effects related to treatment, both patients recovered.
After the presentation at the American Society of Hematology, CRSP stock popped north of 4%.
FDA Submission Soon To Wrap
The companies are actively submitting information to the Food and Drug Administration in the hopes of gaining approval. That process is expected to wrap in the first quarter. Further, a European submission is expected to finish within the next few days.
From there, the entire biotech segment will be watching how the FDA approaches the review process. The possibility of an expedited review period exists, but the FDA may ask a panel of independent experts to weigh in on the drug. This process has implications for the entire industry, Crispr CEO Kulkarni says.
"If we get to the point where we have the first CRISPR therapy approved, it opens up the prospect to many more companies and many more diseases," he said. "It's going to be a clearing event for the entire industry."
Those promising dynamics aside, CRSP stock has struggled as of late. Crispr stock is well known as one of Cathie Wood's ARK Invest holdings.
Shares began an astronomical run in late 2018. That ended with a peak in January 2021. Since then, CRSP stock has tumbled. It has a low Relative Strength Rating of 14 out of a best-possible 99. In terms of 12-month performance, CRSP stock ranks toward the bottom one-fifth, according to IBD Digital.
Verve Hold Hangs On CRSP Stock
The FDA is actively evaluating an onslaught of new applications to begin testing CRISPR in people.
The agency recently put a study from Verve Therapeutics on hold. Verve wants to use CRISPR to permanently lower high cholesterol. Regulators asked for additional preclinical safety data and new safety measures in the study.
The news had a broad impact on gene-editing stocks including CRSP stock, which fell almost 3%.
But analysts aren't by and large concerned.
William Blair analysts rate CRSP stock with an outperform rating. There is already a handful of gene therapies on the market in the U.S. Gene therapies insert a healthy version of a mutated gene to correct diseases. They also expect six other gene therapies to join the crew in 2023.
"The FDA feedback received by Verve seems to read as a cautious stance from the FDA on the gene-editing nature of the product including extra precaution with slow dose escalation, while taking all necessary steps to safeguard potential off-target or (inheritable) editing side effects," the analysts said in a note to clients.
But Crispr and Vertex could be the first to win approval for a gene-editing drug. These drugs actively change mutated genes.
One-And-Done Approach To Medicine
Crispr's Kulkarni says the sickle cell and beta thalassemia treatment is just the first. Crispr also is working on cancer treatments called CAR-T. Traditional CAR-T involves extracting a patient's own immune cells and editing them to find cancer cells.
Crispr — and companies like Allogene, Cellectis, among others — want to sidestep part of that process. They plan to use healthy donor cells to create off-the-shelf CAR-T treatments. CRSP stock analysts hope the so-called allogeneic process will offer a less expensive and faster route than autologous CAR-T, which uses a sick patient's own cells.
"These stories are remarkable," Kulkarni says of patients who've received Crispr's allogeneic CAR-T.
"There are patients who were heavily pretreated," he said. "They came in after three, four, five different rounds of chemo and other medicines. Nothing worked. And they got allogeneic CAR-Ts and are now, in one case, past two years cancer-free."
Further, Crispr also is developing procedures in regenerative medicine. In this space, it partners with ViaCyte — soon to be acquired by Vertex — to develop an artificial pancreas. This involves editing pancreatic cells to prevent the body from rejecting them. The companies put those edited cells inside an implantable device. That's in earlier-stage testing, though, and has little impact on CRSP stock today.
In Vivo Editing Is Next
Another Crispr project is in vivo editing. Its other approaches require ex vivo editing, a process involving the editing of cells outside the body before infusion. To edit in vivo means changing cells or genes inside the body. There, Crispr hopes to edit genes responsible for high triglycerides and cholesterol.
There are already less permanent and already well-established medicines to treat some of these diseases. The first treatment for high cholesterol is a daily pill. But Kulkarni notes compliance is often low. Also, it leaves people with a remaining risk of cardiovascular problems like heart attack and stroke.
Kulkarni expects an evolution in how people view medicine to reach the CRISPR space. That could also have an impact on CRSP stock.
"It's not surprising that people are saying, 'Why would I gene edit if I have a temporary solution?' " he said. "But the world is changing very quickly and what you're going to see, I think, is people are going to say, 'Well, I'm just going to do a one-and-done. Why would I take an injection every so often? Why do I need to take these pills everyday?' You'll see a dramatic change in how people perceive these."
CRSP Stock: A Clearing Event?
The broad biotech stock market has trended sideways for several months.
But shares of companies working in CRISPR have struggled more recently. CRSP stock is now trading below its key moving averages at a seven-month low, according to MarketSmith.com.
The CRISPR world has a history of major delays, Kulkarni says.
"If we can prevent those delays and get approval in a relatively expedited fashion and bring a landmark medicine to patient suffering from sickle cell disease and beta thalassemia, it will be a clearing event for the sector," he said.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
