Investors Rejoice as bluebird bio Plots Road to First-Ever U.S. Gene Therapy Approval

Published: May 20, 2015

Investors Rejoice as Bluebird Plots Road to First-Ever U.S. Gene Therapy Approval
May 19, 2015
By Mark Terry and Riley McDermid, Breaking News Staff

Cambridge, Mass.-based bluebird bio announced today that it had recent discussions with regulators in Europe and the U.S. regarding accelerated approval for its LentiGlobin BB305 therapeutic for the treatment of beta-thalassemia major.

In Europe, the company will be participating in the European Medicines Agency (EMA) Adaptive Pathways pilot program. This is an attempt on the part of the EMA to improve access to new medicines for patients. The company indicates that in discussions with the EMA’s European Health Technology Assessment (HTA) agencies and patient advocate organizations, it may be able to get conditional approval for the use of the drug on the basis of total clinical data from its ongoing study. Full approval would then depend on the successful completion of the clinical trials for HGB-207 and HGB-208, long-term follow-up and what they call “real-life” post-approval monitoring data.

In the U.S., the company has come to a general agreement with the U.S. Food and Drug Administration (FDA) on the design of planned clinical trials for the two compounds. The company expects the data could be the foundation for a Biologics License Application (BLA) submission for LentiGLobin BB305, which shares similar trial design as HGB-207 and HGB-208.

“We are very pleased with the outcome of these recent regulatory interactions,” said David Davidson, Bluebird’s chief medical officer in a statement. “We look forward to advancing our beta-thalassemia major program based on data both from our ongoing studies as well as two planned open-label studies with a sample size of 15 patients each.”

Bluebird Bio’s focus is on gene therapy as a one-time treatment option for patients with genetic and rare diseases. To date no gene therapy procedure has been approved in the U.S. In Europe, only one gene therapy has been approved, uniQure ’s Glybera. Glybera hasn’t hit the market yet.

Bluebird’s approach is to insert a functional human beta-globin gene into the patient’s hematopoietic stem cells outside the body, then returning the engineered cells to the patient via an autologous stem cell transplant.

On Feb. 2, 2015, Bluebird announced that the FDA had granted LentiGlobin BB305 Breakthrough Therapy designation, which is used to expedite the development and review of a potential drug candidate that is expected to be used to treat a serious or life-threatening diseases.

In the case of LentiGlobin BB305, data from an ongoing Phase I/II Northstar (HGB-204) and HGB-205 studies looked at eight patients with beta-thalassemia that were treated with LentiGlobin. In the first four patients, treatment resulted in sufficient hemoglobin production to decrease the need for transfusion support among the patients. That news sent shares of the company up 70 percent the day it was announced, as the market looked eagerly for signs that Bluebird’s LentiGlobin BB305 could be a panacea for blood diseases.

Bluebird’s LentiGlobin BB305 extracts blood stem cells and then infuses them with a working version of the malfunctioning gene that had caused the disease. People with the disease must undergo monthly blood transfusions in order to survive—but if Bluebird’s therapy continues to be successful, they may now be freed from that burden. The number of people affected is not huge, but is certainly significant: Around 40,000 babies world-wide and between 1,000 and 3,000 in the U.S. are born with the condition each year.

The new therapy is so effective, Wall Street is champing at the bit to see it be rushed into later-stage trials to bring it to market more quickly. Some analysts have long said Bluebird has a “robust” proof of concept for the therapy so far, said Joshua Schimmer, a biotech analyst with Piper Jaffray.

“We hosted a call with a hemoglobinopathy specialist. Based on this and our assessment of the data reported for LentiGlobin in beta-thal, we conclude the product has achieved robust proof of concept which is also likely to translate into success in sickle cell anemia,” wrote Schimmer in a note to investors.

The results of this data, though hardly extensive, are apparently promising enough for the FDA and the EMA to consider conditional approval for use of the drug.

Still, although researchers are optimistic about the effectiveness of gene therapy, to date it has been a difficult technique to make work. On April 26, 2015, Celladon Corporation announced that its Phase IIb CUPID2 trial, a cardiovascular gene therapy trial, failed to meet primary or secondary endpoints.

However, San Francisco-based Audentes Therapeutics is also working in the space and recently presented new data at the American Society of Gene and Cell Therapy regarding its gene therapy treatment for X-linked Myotubular Myopathy, AT001, and for Pompe Diseases (AT002). These were proof-of-concept trials that the company hope will lead into advancement into clinical trials.

Bluebird generated a lot of buzz earlier this year when its chief executive told an audience at a closely watched industry event held by J.P. Morgan that the company expects data on its potentially-disease ending sickle cell treatment by the end of 2015.

Nick Leschly, CEO of Bluebird, also said that LentiGlobin BB305 keeps track of production a key marker in keeping down the cost of clinical trials and measuring data.

Leschly made the comments at the J.P. Morgan Healthcare Conference in January in San Francisco and is the oldest and largest conference of its type. It includes 300 of the largest biotech, healthcare and biopharma companies presenting their top-line data and estimates to 4,000 eager bankers, analysts, institutional investors, hedge funds and journalists. He presented the following milestones for Bluebird in the next two years:

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