So far, so good, in tests of treatments that could cure patients of two terrible inherited blood disorders.
At this week’s annual meeting of the American Society of Hematology, researchers reported on the rival approaches of
to fix the genetic causes of the painful capillary clogging of sickle cell disease, or the chronic transfusions needed in beta thalassemia. Bluebird’s clinical trials are further along, but both programs showed they could relieve patients’ symptoms.
While the prospect of rival cures is great for patients, investors might worry about the competition. Since Monday morning, shares of bluebird (ticker: BLUE) have slipped 7%, to $43.77 on Tuesday. Crispr stock (CRSP) has lost 8% in the two days, dropping to a Tuesday level of $149.88. Shares of Crispr’s partner on the blood treatments,
(VRTX), have stayed relatively flat, at $228.
Investors seemed more generous to contenders further back in the pack. After
(BEAM) showed ASH attendees that it could deftly repair these genetic disorders in preclinical settings, traders lifted Beam’s stock 25% from Friday’s close, to a Tuesday price of $75.
Bluebird has treated more than 100 patients with its gene therapies for thalassemia and sickle cell. In a Monday presentation, researchers discussed a trial that treated 32 sickle-cell patients with bluebird’s LentiGlobin therapy. It has been up to two years since some among the first 19 were treated and none have suffered the awful blood-vessel clogs they endured before the gene therapy.
On a conference call, bluebird clinical research executive Rich Colvin reminded listeners of the benefit that treatment brought to sickle-cell patients. Each severe sickling crisis, he said “represents a significant traumatic and painful experience, a hospitalization causing disruption in life, impeding the ability to go to school or to work and always with the possibility of any event becoming fatal.”
A Saturday presentation at ASH reviewed bluebird’s beti-cel thalassemia treatment. Sixty patients have been followed for as long as 6½ years, with the share remaining free of transfusions in bluebird’s Phase 3 study reaching 83% among adults and 87% among children.
The thalassemia gene therapy was approved in June by regulators in Europe, where it is being marketed as Zynteglo. But the first patients are only now being treated, because of the Covid-19 pandemic and lengthy negotiations with government health agencies over reimbursement for the one-time treatment.
In the U.S., bluebird’s thalassemia treatment has a breakthrough- therapy designation from the Food and Drug Administration. Bluebird plans to file its approval application in mid-2021. The FDA disappointed bluebird in November, however, by asking for longer patient follow-up before a sickle-cell application. Just in time for the ASH meeting, a report on the company’s sickle-cell study appeared in The New England Journal of Medicine.
ASH attendees also heard about the first 10 patients receiving the gene-editing therapy of Vertex and Crispr. All seven of the thalassemia patients have been transfusion-free, while all three of the treated sickle- cell patients have remained free of blood-clogging crises. A report on two of the Crispr cases also appeared in The New England Journal. On Wednesday at 8 a.m. Eastern time, the two companies will review their latest data in a webcast.
With each of the rival programs showing effectiveness, many analysts think bluebird will enjoy an advantage given its head start. At Mizuho Securities, analyst Difei Yang thinks bluebird is far undervalued. He rates the shares a Buy and says the stock can more than double, to $123.
RBC Capital Markets analyst Luca Issi sees pros and cons in the different techniques used by the rivals, but sees no evidence yet that those differences will matter in the clinic. But with Crispr up fourfold since March, to a $10 billion market capitalization, Issi sees the stock as priced for perfection. He rates it a Sector Performer.
Write to Bill Alpert at [email protected]