Experimental genetic treatments maintained their promise of curing two ancient inherited blood disorders, in the latest data presented at a European medical conference.
The small numbers of patients treated with these gene-altering treatments continue to enjoy relief from the symptoms of sickle-cell disease and beta-thalassemia, according to presentations from researchers testing rival treatments from
(ticker: BLUE) and the team of
In trading on Monday, Crispr stock jumped 4%, to close at $62.45. Vertex eased back 0.3%, to $267.77, while bluebird stock slipped 0.6%, to $63.54.
Sickle-cell is a painful and often fatal genetic disorder in which misshapen red blood cells clog a person’s blood vessels. It occurs more frequently among those of sub-Saharan African origin, including 1 of every 365 black Americans. Beta-thalassemia leaves those who inherit it with anemia and a lifelong need for transfusions. It more often afflicts those whose genes trace to the Mediterranean, Middle East, and Asia.
Medicines have eased the symptoms of both disorders in recent years, but the therapies being developed by bluebird and Crispr aim to fix the problem once and for all, by introducing new genetic instructions into patients’ cells.
So far so good.
The new data are appearing at a virtual version of the European Hematology Association Congress meeting that occurs every spring, about six months after the annual meeting of its counterpart American Society of Hematology.
The two meetings offer semiannual updates on research programs like bluebird’s gene-therapy Lentiglobin—which extracts a patient’s stem cells, genetically alters them with new instructions, then infuses the stem cells back into the patient where they can grow into healthy blood cells.
The 22 beta-thalassemia patients in the bluebird Northstar-2 trial used to average more than a dozen transfusions a year. Since receiving the stem-cell treatment, about 90% have needed no transfusion. In a second study called Northstar-3, the patients had previously needed between 11 and 39 transfusions a year. After treatment, 11 of 13 patients have been off transfusion for more than six months.
Bluebird is calling its beta-thalassemia treatment zynteglo. The European Union approved it and in a conference call last Friday, bluebird said that it was preparing to treat patients in Germany. In mid-2021, it plans to apply for approval by the U.S. Food and Drug Administration.
The company has a stem cell program to treat sickle cell. At the December 2019 meeting of the American Society of Hematology, 17 patients had been treated. They averaged a 99% reduction in symptoms.
In the latest European update, the number of treated patients had risen to 25, with the same level of benefit. On its Friday call, bluebird said it hopes to apply in the second half of 2021 for FDA approval.
Crispr Therapeutics is testing a different kind of cure for both of the inherited blood disorders. It uses the new technology known as Crispr to edit the faulty genetic instructions in a patients’ cells. At the European conference on Friday, the company and its partner Vertex announced that they had successfully engrafted new genetic instructions in five beta-thalassemia patients and two sickle cell patients.
A hematology professor at Regensburg University Hospital, Selim Corbacioglu, told participants at the European conference that the first patient who got Crispr’s beta-thalassemia treatment had previously needed 34 transfusions a year. Now 15 months after the Crispr treatment, the patient is transfusion independent.
The first patient to get Crispr’s sickle cell treatment had previously suffered seven blood vessel blockages a year. Nine months after treatment, that patient has stayed free of such crises.
After a pause in recruitment under the Covid-19 lockdowns, Crispr and Vertex are now going to treat new patients in the two blood disorder studies. Patients will be followed for two years after their initial infusions.
The new data on Crispr’s products inspired Canaccord Genuity analyst Arlinda Lee to raise her price target on the Buy-rated stock, to $84 from $80, in a Sunday note.
While Crispr has many projects in its pipeline, Lee thinks that the Crispr/Vertex sickle-cell treatment is the most valuable. If successful, she thinks it could achieve annual sales of $1.8 billion—with Crispr’s half-share of the expected profit stream worth $25 a share today. The beta-thalassemia treatment could reach $670 million in annual sales, she estimates, for a discounted value of $12 per Crispr share.
Crispr’s technology has successfully transitioned from the lab to the clinic, the Canaccord analyst said.
Write to Bill Alpert at [email protected]