bluebird bio (BLUE) announced the U.S. Food and Drug Administration has approved Zynteglo, also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta-thalassemia in adult and pediatric patients who require regular red blood cell transfusions.
“Due to the complex nature of gene therapy, Zynteglo will be available exclusively at Qualified Treatment Centers, which are carefully selected based on their expertise in relevant areas such as stem cell transplantation, cell and gene therapy, and beta-thalassemia; and receive specialized training to administer Zynteglo.
Information on bluebird’s QTC network, as well as personalized support focused on the needs of each patient throughout their treatment journey and information on insurance coverage and access will be available through bluebird’s patient support program,” the company stated.
“The FDA approval of Zynteglo offers people with beta-thalassemia the possibility of freedom from burdensome regular red blood cell transfusions and iron chelation, and unlocks new possibilities in their daily lives.
Current thalassemia treatment
After more than a decade of research and clinical development, and through the perseverance of clinicians, patients, and their families, the approval of Zynteglo marks a watershed moment for the field of gene therapy.
As the first ex-vivo lentiviral vector gene therapy approved in the U.S. for the treatment of people with beta-thalassemia, we are ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care,” said Andrew Obenshain, CEO of bluebird bio.ย
Expensive Treatment
The lifetime cost of medical care for a patient with transfusion-dependent beta-thalassemia can reach up to $6.4M in the U.S. and the average total health care cost per patient per year is 23 times higher than the general population. bluebird estimates that there are approximately 1,300-1,500 individuals with transfusion-dependent beta-thalassemia in the U.S.
” bluebird has set the wholesale acquisition cost of Zynteglo in the U.S. at $2.8M, in “recognition of its robust and sustained clinical benefit demonstrated in clinical studies and its potential to alleviate a lifetime of health care costs associated with regular RBC transfusions and iron management.” Tom Klima, chief commercial and operating officer of bluebird bio, said.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Crispr, Vertex announce interim data from first two patients treated with CTX001
CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals Incorporated (VRTX) announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials.
Gene Editing shows promises in treating patients, Stockwinners
One patient with transfusion-dependent beta thalassemia received CTX001 in the first quarter of 2019 and data for this patient reflect nine months of safety and efficacy follow-up.
Vertex and CRISPR report positive data, Stockwinners
One patient with severe sickle cell disease received CTX001 in mid-2019 and data for this patient reflect four months of safety and efficacy follow-up.
These studies are ongoing and patients will be followed for approximately two years following infusion.
Several additional patients have been enrolled and have had drug product manufactured across the two studies.
The patient with TDT has the beta0/IVS-I-110 genotype and required 16.5 transfusions per year before enrolling in the clinical study. The patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion.
Stockwinners
Two serious adverse events occurred, neither of which the principal investigator considered related to CTX001: pneumonia in the presence of neutropenia and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved.
At nine months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells.
The patient with SCD experienced seven vaso-occlusive crises per year before enrolling in the clinical study. The patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion.
Three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis, and abdominal pain, all of which resolved.
At four months after CTX001 infusion, the patient was free of VOCs and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells.
William Blair
The firm upgrades Crispr Therapeutics to Outperform after ‘impressive’ data .
William Blair analyst Raju Prasad upgraded Crispr Therapeutics to Outperform from Market Perform after the company presented initial results from the ongoing Phase I/II trials of CTX001 for the treatment of beta thalassemia and sickle cell disease.
The analyst says today’s data cut was “impressive” as it exceeds the 30% fetal hemoglobin threshold that he viewed as critical.
To date, Crispr has shown initial proof-of-concept in beta thalassemia and sickle cell that exceeded expectations and de-risks its wholly owned immuno-oncology platform, Prasad tells investors in a research note. Further, given the “optionality” of the CRISPR-Cas9 platform and potential cost-effectiveness when compared with lentiviral-based therapies, Crispr could be a potential takeout candidate, adds the analyst.
Stockwinners
Goldman Sachs
Goldman Sachs analyst Salveen Richter raised his 12-month price target for Crispr Therapeutics (CRSP) to $75 from $52 while keeping a Neutral rating on the shares.
The first clinical results from the ongoing CTX001 trial impress, Richter tells investors in a research note. The analyst, who cautions the data represents small patient numbers, is “highly encouraged” with the profile of CTX001 and sees today’s positive data as initial validation of Crispr’s ex-vivo gene editing platform.
Roth Capital
Roth Capital analyst Tony Butler raised his price target for Crispr Therapeutics to $100 from $65 and maintained a Buy rating after the company and partner Vertex Pharmaceuticals (VRTX) announced the first CTX001 early clinical data in two patients.
In a research note to investors, Butler says that though only two patients have been treated with CTX001, these data provide a hint that CRISPR-Cas9 could be curative for hemoglobinopathies, adding that gene-editing is “clearly not a fiction.”
The results utilizing gene-editing with CRISPR-Cas9 to create allogenic CAR-T cells against various cancers may also increase its probability of success based on successful editing in the CLIMB trials, he said.
Cantor Fitzgerald
Cantor Fitzgerald analyst Alethia Young raised her price target for Vertex Pharmaceuticals (VRTX) to $229 from $217 after the company and partner Crispr Therapeutics (CRSP) announced the first CTX001 early clinical data in two patients.
The analyst finds the data “highly encouraging” and thinks this could be an “exciting pipeline program of focus for Vertex by this time next year.” She increased her probability of success for CTX001 to 20% from 10% and reiterates an Overweight rating on shares of Vertex.
Piper Jaffray
Piper Jaffray analyst Edward Tenthoff raised his price target for Crispr Therapeutics (CRSP) to $107 from $100 after the company and partner Vertex Pharmaceuticals (VRTX) reported first-ever CTX001 data on one beta thalassemia and one sickle cell disease patient. Tenthoff is “impressed by these early results showing potential curative effect and look for more patients and longer follow-up next year.” He reiterates an Overweight rating on Crispr Therapeutics.
CRSP is up $10 to $68.55. VRTX is up $5.07 to $215.07
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
