The genetic medicine pioneer
said Monday morning that U.S. regulators will let it resume clinical trials of its gene therapies for sickle cell disease and another severe blood cell disorder.
Bluebird (ticker: BLUE) is the second company recently cleared of concerns that its gene therapy had caused cancers in clinical trial participants. In April,
(QURE) was allowed to resume a study of its hemophilia gene therapy. The news could help investors regain confidence in bluebird and other gene therapy stocks.
Bluebird’s news seemed to find a cautiously good reception among traders. The stock was up 2.5% by midday Monday, at $31.70, against a 0.1% rise in the Nasdaq Composite Index. Back in February, when the U.S. Food and Drug Administration imposed a clinical hold on bluebird’s studies, the stock plunged to $26 from $46.
“The company is working closely with study investigators and clinical trial sites to resume all study activities as soon as possible,” said bluebird in its announcement.
Like other early genetic medicine developers, bluebird is using a gene-augmentation approach that uses hollowed-out viruses to transfer functional versions of a gene into the cells of patients suffering from a disease caused by their inheriting a nonfunctional gene. Decades ago, primitive gene therapies triggered cancers in study recipients, so gene-transfer therapy companies and the FDA have been vigilant in testing newer approaches. “Patient safety continues to be our utmost priority,” said bluebird’s president of severe genetic diseases,
in Monday’s announcement.
Before the February pause, bluebird was conducting Phase 2 and 3 trials of gene therapies for sickle cell — a crippling red blood cell disorder, most common among people of African descent — and Phase 3 trials of therapies for beta-thalassemia — another inherited red cell disorder that causes bleeding.
Bluebird also paused its marketing of the thalassemia treatment in Europe, where it has been approved. The clinical holds followed a leukemia diagnosis in one sickle-cell patient and a suspected case of precancerous myelodysplastic syndrome in another. The second proved a false cancer alarm- — upon investigation by clinical trial doctors, the company and the FDA — while the leukemia case proved unrelated to that patient’s gene therapy.
A crowd of genetic medicine companies are racing to develop therapies for sickle cell, including companies employing newer gene-editing techniques, such as
Besides knocking bluebird’s stock, the FDA’s February action clobbered the stock of
(AVRO), which, like bluebird, uses hollowed-out lentiviruses to deliver gene therapy. The February pause of bluebird studies caused Avrobio stock to drop to $11.50 from around $17 in a day. At mid-Monday, Avrobio stock was up 7%,, to $9.35.
The slow progress of gene therapy trials has tried the patience of investors in stocks like
(SRPT) and uniQure. A liver cancer diagnosis in one of uniQure’s hemophilia patients halted its studies in December and pulled its stock down to $36 from around $45 . Since the FDA lifted the hold on uniQure’s program in April, after researchers concluded that the cancer wasn’t caused by gene therapy, uniQure stock has risen to $37 on Monday from $32.
There are other puzzles for bluebird investors to solve. The company is in the process of splitting itself into two pieces. One piece will continue developing treatments for rare diseases like sickle cell, while the other will market cell-therapies for cancer, like the promising Abecma treatment for multiple myeloma that bluebird is commercializing with
Bristol Myers Squibb
Bluebird’s good news Monday from the FDA should be positive for the entire gene therapy space, said Mizuho Securities analyst Difei Yang, in a note. The sector’s stocks have been out of favor, said Yang, on concerns over safety, manufacturing and the high price tags of the first commercialized gene therapies. She thinks the treatments from the like of bluebird, uniQure, and Avrobio will transform clinical outcomes for patients, and rates them as Buys. Bluebird could more than double, to $70, says Yang, while uniQure could rise almost 50%, to $52.
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