More Good News From Bluebird Bio

Just released.
More good news from Bluebird. There is a very good chance that the process will be accelerated.
http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2049713

bluebird bio Announces Global Regulatory Strategy for LentiGlobin BB305 in Beta-Thalassemia Major
Company Plans to Pursue Conditional and Accelerated Registration Strategies in E.U. and U.S., Respectively
Plans to Pursue Conditional Approval in E.U. Based on Data from Ongoing Northstar and HGB-205 Studies through Adaptive Pathways Pilot Program
NIH RAC Will Review HGB-208 Pediatric Study Protocol on June 9, 2015
Conference Call and Webcast Today at 8:00AM ET
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 19, 2015-- bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, today announced that it has met with regulatory authorities in Europe and the United States to discuss potential approval pathways for its LentiGlobin BB305 product candidate for the treatment of beta-thalassemia major. These discussions have resulted in general agreement from both agencies regarding bluebird bio’s development plans, which could potentially result in accelerated approvals.
“We are very pleased with the outcome of these recent regulatory interactions,” commented David Davidson, M.D., chief medical officer. “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. The EMA Adaptive Pathways pilot program will allow us to pursue conditional approval for the treatment of beta-thalassemia major on the basis of clinical data from our ongoing HGB-204 and HGB-205 studies. This feedback brings us closer to achieving our vision of delivering one-time, potentially transformative gene therapy to patients.”
EMA Registration Strategy: Participate in Adaptive Pathways Pilot Program
bluebird bio is one of the first companies to participate in the European Medicines Agency’s (EMA) Adaptive Pathways (formerly referred to as Adaptive Licensing) pilot program, which is part of the EMA’s efforts to improve timely access for patients to new medicines. Based on several discussions involving the EMA, European Health Technology Assessment (HTA) agencies and patient advocacy organizations as part of this program, bluebird bio believes it is possible to seek conditional approval for the treatment of adults and adolescents with beta-thalassemia major on the basis of the totality of clinical data, in particular reduction in transfusion need, from the ongoing Northstar Study and supportive HGB-205 study. Conversion to full approval will be subject to the successful completion of the HGB-207 and HGB-208 clinical trials discussed below, supportive long-term follow-up data and “real-life” post-approval monitoring data.
FDA Registration Strategy: Pursue Accelerated Approval Based on HGB-207 (n=15) and HGB-208 (n=15)
In addition, bluebird bio has reached general agreement with the U.S. Food and Drug Administration (FDA) on the design of its planned clinical trials HGB-207 and HGB-208. Based on its discussions with the FDA, bluebird bio believes that data from these trials, together with data from the ongoing beta-thalassemia major clinical studies (Northstar and HGB-205), could form the basis for a Biologics License Application (BLA) submission for LentiGlobin BB305. HGB-207 and HGB-208 share similar trial designs and are differentiated primarily by patient age. HGB-207 will enroll adult and adolescent patients; HGB-208 will enroll pediatric patients.
bluebird bio has also reached general agreement with the FDA on:
Sample size: 15 patients per trial
Duration: 24 months of follow-up per patient
Primary endpoint: 12 months of transfusion independence
In the United States, if the LentiGlobin BB305 product candidate demonstrates acceptable efficacy and safety in these patient populations, these planned clinical trials could support an accelerated approval, with post-approval confirmatory evidence to be provided with longer-term follow-up of these trials. As a result of this regulatory feedback and as required of all gene therapy clinical trials, bluebird bio has filed both clinical study protocols with the National Institutes of Health (NIH) Recombinant DNA Advisory Committee (RAC). The RAC has notified bluebird bio that HGB-207 does not require an in-depth review or public RAC discussion. The RAC has also notified bluebird bio that the HGB-208 study protocol is scheduled for public review on June 9, 2015.
“We are grateful for the collaborative regulatory feedback from the FDA and EMA on the design of our pivotal studies, as well as feedback from the European HTA agencies and patient advocacy organizations that are participating in our Adaptive Pathways pilot project,” stated Anne-Virginie Eggimann, vice president of regulatory science. “We are looking forward to continuing our engagement with all of these stakeholders in the coming months to support the potential acceleration of the LentiGlobin BB305 program.”
Background on the EMA’s Adaptive Pathways Program
In establishing the Adaptive Pathways program, the EMA stated the following:
“The concept of Adaptive Pathways foresees either an initial approval in a well-defined patient subgroup with a high medical need and subsequent widening of the indication to a larger patient population, or an early regulatory approval (e.g. conditional approval), which is prospectively planned, and where uncertainty is reduced through the collection of post-approval data on the medicine's use in patients. This approach is particularly relevant for medicines with the potential to treat serious conditions with an unmet medical need and may reduce the time to a medicine's approval or to its reimbursement for targeted patient groups. It involves balancing the importance of timely patient access with the need for adequate, evolving information on a medicine's benefits and risks. The Adaptive Pathways approach builds on regulatory processes already in place within the existing European Union legal framework.”
The pilot was initiated in March 2014 and was called “Adaptive Licensing” at the time. EMA changed the name to Adaptive Pathways “to better reflect the idea of a life-span approach to bring new medicines to patients with clinical drug development, licensing, reimbursement, and utilization in clinical practice, and monitoring viewed as a continuum.”
Background on the FDA Process and NIH’s RAC
FDA approval must be obtained before clinical testing of biological products. Each clinical study protocol for a gene therapy product is reviewed by the FDA and the NIH, through its Recombinant DNA Advisory Committee (RAC). Within the FDA, the Center for Biologics Evaluation and Research (CBER) regulates gene therapy products. CBER works with the NIH and its RAC, which makes recommendations to the NIH on gene therapy issues and engages in a public discussion of scientific, safety, ethical and societal issues related to proposed and ongoing gene therapy protocols.
The NIH is responsible for convening the RAC to discuss protocols that raise novel or particularly important scientific, safety or ethical considerations at one of its quarterly public meetings. The Office of Biotechnology Activities (OBA) notifies the FDA of the RAC’s decision regarding the necessity for full public review of a gene therapy protocol. RAC proceedings and reports are posted to the OBA web site and may be accessed by the public.
Investor Conference Call and Webcast Information
bluebird bio will host a conference call and webcast on May 19, 2015 at 8:00 AM ET to review its LentiGlobin regulatory strategy. The event will be webcast live and can be accessed under "Calendar of Events" in the Investors and Media section of the company's website at www.bluebirdbio.com. Alternatively, investors may listen to the call by dialing (844) 825-4408 from locations in the United States and (315) 625-3227 from outside the United States.
About bluebird bio, Inc.
With its lentiviral-based gene therapy and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and T cell-based immunotherapy. bluebird bio’s clinical programs include Lenti-D™, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of childhood cerebral adrenoleukodystrophy, and LentiGlobin®, currently in three clinical studies: a global Phase 1/2 study, called the Northstar Study, for the treatment of beta-thalassemia major; a single-center Phase 1/2 study in France (HGB-205) for the treatment of beta-thalassemia major or severe sickle cell disease; and a separate U.S. Phase 1 study for the treatment of sickle cell disease (HGB-206). bluebird bio also has a preclinical CAR T immuno-oncology program in collaboration with Celgene Corporation, as well as discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies.
bluebird bio has operations in Cambridge, Massachusetts, Seattle, Washington, and Paris, France. For more information, please visit www.bluebirdbio.com.
Forward-Looking Statements
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s global regulatory strategy for LentiGlobin BB305, including the expected protocols for planned clinical trials and the timing of these clinical trials, whether these planned clinical trials will be sufficient to support regulatory submissions for marketing approval and the expected timing of any such submissions and decisions. In particular, it should be noted that the FDA normally requires two pivotal clinical studies to approve a drug or biologic product. Whether the planned HGB-207 and HGB-208 trials will be sufficient to support submission of a BLA for LentiGlobin BB305 will likely be a review issue to be discussed with FDA following completion of the trials. In addition, it should be noted that the EMA Adaptive Pathways program is a pilot program, and as such there is limited information and precedent regarding the potential outcomes for sponsors that participate in this program. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, the risk of a delay in the enrollment of patients in the Company’s clinical studies, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and regulatory submissions, the risk that the results of previously conducted studies involving similar product candidates will not be repeated or observed in ongoing or future studies involving current product candidates, the risk that our collaboration with Celgene will not continue or will not be successful, and the risk that any one or more of our product candidates will not be successfully developed and commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent annual report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.
Availability of other information about bluebird bio
Investors and others should note that we communicate with our investors and the public using our company website (www.bluebirdbio.com), our investor relations website (http://www.bluebirdbio.com/investor-splash.html), including but not limited to investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference calls and webcasts. You can also connect with us on Twitter @bluebirdbio, LinkedIn or our YouTube channel. The information that we post on these channels and websites could be deemed to be material information. As a result, we encourage investors, the media, and others interested in bluebird bio to review the information that we post on these channels, including our investor relations website, on a regular basis. This list of channels may be updated from time to time on our investor relations website and may include other social media channels than the ones described above. The contents of our website or these channels, or any other website that may be accessed from our website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

View source version on businesswire.com: http://www.businesswire.com/news/home/20150519005556/en/
Source: bluebird bio, Inc.
Investor Relations:
bluebird bio, Inc.
Jim DeTore, 339-499-9355
Chief Financial Officer
or
Media:
Pure Communications, Inc.
Dan Budwick, 973-271-6085

This is incredible,

The fact that they plan to start peads tells how how much confidence there is in the safety and effectiveness of this treatment!

Thank you for sharing Andy!


Sharmin

Please update my information
I know that this bluebird company is continuing  the French research that was done on a HB E patient and I remember that Andy had concerns over their work and believed that Dr.  Sadelain vector is much better and  has avoided the drawbacks of the French research.

Has this changed? What about Dr. Sadelain trails?

Manal,

Bluebird greatly advanced their previous method and they are having fantastic results. Meanwhile, Sloan Kettering has slowed down and only recently started a fourth patient. and Dr Sadelain has started a new company involved in cancer research. Bluebird's stock has gone from about $20/share to over $160/share and rising, so you can see, there is much confidence in their process.

Hope we hear that there is a definite cure soon .

So far the results have been incredible, some patients have become transfusion free within 12 days after receiving the infusion of corrected cells. 

Hopefully this treatment continues to be safe and becomes widely available for all. 

I am glad to hear that SK has started a 4th patient - I do like the idea of reduced intensity conditioning...

This is great news for all the thals and especially that Bluebird intend to start with peadiatric patients.
 

Andy,
does BB intend to try out reduced intensity chemo for its trails anytime soon?

Bluebird has no plans to try reduced intensity chemo.

It seems things are going so well with myeloablation - and there have been no serious side effects so far.  I am guessing as things stand, reducing the chemo will likely reduce the effectiveness of the treatment.   SK has been using reduced intensity chemo, but it seems they have not had the overwhelming success that bluebird is having.  I think the SK patients have reduced need for transfusions - but bluebird patients have actually been cured of the underlying disease.   The success is overwhelming and wonderful. 

Thank you so much for the update ...let's all pray for the best
Manal

The success is overwhelming and wonderful.

I agree, but can we really say that

bluebird patients have actually been cured of the underlying disease.

?

If their Hemoglobin levels don't rise to normal levels, does that not mean that the patients will be transfusion-independent, but their symptoms will persist in a mild(er) form? What is the final goal of bluebird bio? Transfusion independence or patients who are completely free of symptoms?

The HGB-205 study seems to have been going on for longer and the results look promising, though. The two beta majors seem to have a lot of HbA^T87Q activity and high Hb levels. Hopefully the patients in the Northstar study will also be able to get there. I mean, patient 1102's Hb is at 8.6 g/dL and the data tells us that she has already been in the follow-up phase for 6 months.

Does anyone know if there is any more data to be found than in here: http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=1995913? What levels did the patients start with? How did they change over time? (not just the snapshot from December 2014)

Sofear,

I think that bluebird bio's final goal is the cure patients and the achieve the highest hemoglobin levels possible and the best quality of life possible.  I believe their goal is to achieve hemoglobin levels near normal if possible.  

From my understanding, both of the beta zero thalassemia patients had achieved hemoglobin levels of 9.6 at very early stages of their treatment.  The Pakistani patient did not achieve engraftment until 29 days - so from the time of engraftment this level is very good.  Perhaps with more experience and further tweaking it may be possible to achieve even better results.  

For a beta zero patient - having no ability to produce adult hemoglobin at all - the only way to find out what the hemoglobin was before treatment would be to stop transfusing them - my son at the age of 3 months had a hemoglobin of 4.4 - had he not been tranfused he would have fallen even lower.  To allow someone to dip that low would be unethical and dangerous.  Personally, I think that for patients who have no ability to produce hemoglobin at all - being able to achieve a hemoglobin level of 9.6 is success.  I would be very happy if my son would be able to maintain hemoglobin close to 1.0 on his own.  Of course I would be even happier if his hemoglobin were 14.0  but I think this treatment has come as closer to a cure with little to no complications than any other procedure.  

I think that eliminating the need for constant fluctuating hemoglobin levels, transfusions, iron overload and chelation - in itself will greatly improve the lives of thals.  I am a thal minor and my hemoglobin at times is near 1.0 but I don't transfuse and my body has adapted to this level quite well.  

Transplants have great chances of graft failure, rejection, gvhd and death.  I think that this procedure offers nearly all of the benefits of a successful transplant, without the risks.  

The patient that achieved a hemoglobin level of 8.6 had beta E thalassemia.  Let us see what the results are when bluebird reveals their findings on June 13th in Europe and answers questions on June 15th. 

I am sure that this procedure will continue to evolve and become better and better - but we have a cure.  I hope the success continues and that it is available for everyone soon.  It is so great to see that the process has been fast tracked.  

The next full report from Bluebird will come in June. This preliminary report was released today. The news about the sickle cell patient created a stir. Bluebird's stock is now >$170/share. Bluebird hopes for a normal Hb in patients, but they will not speculate and won't comment on what is not yet supported by data. The next report will show us if patients continued to improve. My guess is yes. Keep in mind that trials are run to answer the questions and until they have data from many patients, with numerous thal mutations, and tried various dosing regimens, these questions have no solid answers. It is very interesting that the report on the sickle patient is so glowing. Dosing is a major issue to tackle with sickle cell and I believe what they have learned from the early thal patients has reaped benefits with the sickle patient. Of course, this means they are gaining a better understanding of what adequate dosing is for different types of patients.
http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2052256

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bluebird bio

News Release
bluebird bio to Present LentiGlobin Clinical Data at 20th Congress of European Hematology Association
Presenting new and updated data from HGB-205 study in beta-thalassemia major and severe sickle cell disease, including first patient with sickle cell disease ever treated with gene therapy

Company to host investor call on Monday, June 15, 2015 at 8:00 a.m. ET

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 21, 2015-- bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, today announced that data from the ongoing Phase 1/2 HGB-205 study of LentiGlobin BB305 Drug Product will be presented in an oral presentation on June 13, 2015 at the 20th Congress of the European Hematology Association (EHA) in Vienna, Austria.

“The early data included in our abstract provide further validation for our approach and important insights into the safety and mechanism of action of LentiGlobin in both beta-thalassemia and sickle cell disease,” said David Davidson, chief medical officer, bluebird bio. “As noted in the abstract, we are pleased to report that the two patients with beta-thalassemia major, on whom we first reported last year at EHA, remained transfusion independent at 14 and 11 months post-transplant. In addition, it is very encouraging that the patient with sickle cell disease is increasing production of HbAT87Q, which has anti-sickling properties, and has not had a post-treatment hospitalization for a sickle cell disease-related event. At EHA we will present further follow up data on all three subjects.”

Abstract Highlights (Data as of February 2015):

Beta-thalassemia: Beta-thalassemia major subjects (1201 and 1202) remained transfusion independent at 14 months and 11 months, respectively
Sickle Cell Disease: This subject (1204) entered the trial receiving chronic transfusions and began the process of being weaned from transfusions after day 37, receiving the last transfusion on day 88
Increasing production of HbAT87Q; the first-ever SCD patient treated with gene therapy (subject 1204) had a HbAT87Q level of 24% at 4.5 months follow up, compared to an HbAT87Q level of 9.6% at three months post-transplant
Note that this subject did not engraft until after month one, so their level of HbAT87Q production at months three and 4.5 are actually months two and 3.5, after engraftment
At 4.5 months follow up, total anti-sickling hemoglobin (HbAT87Q + HbF) was 31.6%
Subject 1204 has not had any hospitalizations for SCD-related complications post-transplant
Safety: No subject has experienced a drug product-related adverse event, and integration site analyses demonstrate highly polyclonal reconstitution without clonal dominance
Based on historical clinical observations in patients with SCD, bluebird bio believes that individuals who achieve ≥ 30 percent of anti-sickling hemoglobin (HbAT87Q + HbF) have the potential to reduce or eliminate the serious and life-threatening events associated with SCD.

The abstract is now available online on the EHA conference website. Information contained in the abstract reflects data available as of February 2015. Details of bluebird bio’s presentation are as follows:

Title: Outcomes of Gene Therapy for B-Thalassemia Major and Severe Sickle Cell Disease via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta Globin Vector
Abstract Code: S466
Session Name: Gene therapy, cellular immunotherapy and vaccination
Date: Saturday, June 13, 2015
Oral Presentation Time: 11:30 - 11:45 a.m. CET
Location: Reed Messe Vienna, Room Stolz 2

Investor Conference Call and Webcast Information
bluebird bio will host a conference call and webcast on June 15, 2015 at 8:00 a.m. ET to discuss the full data presented at EHA. The event will be webcast live and can be accessed under "Calendar of Events" in the Investors and Media section of the company's website at www.bluebirdbio.com. Alternatively, investors may listen to the call by dialing (844) 825-4408 from locations in the United States and (315) 625-3227 from outside the United States.

About bluebird bio, Inc.
With its lentiviral-based gene therapy and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and T cell-based immunotherapy. bluebird bio’s clinical programs include Lenti-D™, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of childhood cerebral adrenoleukodystrophy, and LentiGlobin®, currently in three clinical studies: a global Phase 1/2 study, called the Northstar Study, for the treatment of beta-thalassemia major; a single-center Phase 1/2 study in France (HGB-205) for the treatment of beta-thalassemia major or severe sickle cell disease; and a separate U.S. Phase 1 study for the treatment of sickle cell disease (HGB-206). bluebird bio also has a preclinical CAR T immuno-oncology program in collaboration with Celgene Corporation, as well as discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies.

bluebird bio has operations in Cambridge, Massachusetts, Seattle, Washington, and Paris, France. For more information, please visit www.bluebirdbio.com.

Forward-Looking Statements
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the advancement of, and anticipated milestones related to the Company’s product candidates, including LentiGlobin. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, the risk of a delay in the enrollment of patients in the Company’s clinical studies, the risk that the results of previously conducted studies involving similar product candidates will not be repeated or observed in ongoing or future studies involving current product candidates, the risk that our collaboration with Celgene will not continue or will not be successful, and the risk that any one or more of our product candidates will not be successfully developed and commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.



View source version on businesswire.com: http://www.businesswire.com/news/home/20150521005591/en/

Source: bluebird bio, Inc.

Investor Relations:
bluebird bio, Inc.
Jim DeTore, 339-499-9355
Chief Financial Officer
or
Media:
Pure Communications, Inc.
Dan Budwick, 973-271-6085

Its great. Thank Andy for sharing with us.

I hope the success continues and available for everyone soon.