bluebird bio Update from ASH 2016

bluebird bio presented at ASH this weekend.

Some highlights.

Updated interim clinical data from the Northstar (HGB-204) study of LentiGlobin drug product in transfusion-dependent β-thalassemia (TDT) confirm patients with non-β0/β0 genotypes and ≥12 months of follow-up have stopped regular transfusions; patients with β0/β0 genotypes and ≥12 months of follow-up had a median reduction in transfusion volume of 63%.

“In addition, we are pleased by the robust vector copy number and high proportion of LVV+ CD34+ stem cells in the drug product manufactured using transduction enhancers for the first patient to be treated in the Phase 3 Northstar-2 study. If the clinical correlation between drug product VCN and hemoglobin production observed in the Northstar study continues with drug product manufactured utilizing process 2, we are hopeful that LentiGlobin drug product with higher VCNs will consistently yield clinically meaningful outcomes for patients with TDT across all genotypes.”

The announcement from bluebird.

bluebird bio is honored to have presented new data from multi-center studies of LentiGlobinTM drug product in patients with transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) at the American Society of Hematology (ASH) Annual Meeting this week.
To summarize, bluebird bio provided updates including:
- Updated interim clinical data from the Northstar (HGB-204) study of LentiGlobin drug product in TDT confirm patients with non-β0/β0 genotypes and ≥12 months of follow-up have stopped regular transfusions; patients with β0/β0 genotypes and ≥12 months of follow-up had a median reduction in transfusion volume of 63%
- Drug product vector copy numbers (DP VCNs) for the first HGB-207 and HGB-206 patients who will be treated using LentiGlobin drug product made using our improved manufacturing process (process 2) are 2.9 and 3.3, respectively
- Updated interim clinical data from seven subjects in the HGB-206 study of LentiGlobin drug product in SCD underscore the need for recently implemented protocol amendments seeking to improve HbAT87Q production in this population
A full recap of what was presented at ASH can be found in the following two press releases:
http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2227726
http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2227319
Nick really said it best - “Our focus is learning, adjusting and implementing to innovate on behalf of the patients we aim to serve. This year we have made tremendous progress against this objective. In 2016, we focused on further enhancing our LentiGlobin programs in TDT and SCD by implementing high potential manufacturing and protocol amendments while advancing our lead bb2121 oncology program and commercialization capabilities. We are encouraged by the data presented at ASH and how that has informed our plans for 2017.”

patients with β0/β0 genotypes and ≥12 months of follow-up had a median reduction in transfusion volume of 63%

This means frequency of transfusion decrease? is it going to complete stop?

I haven't seen the graph to see if there was any improvement in the  β0/β0 genotype patients, so I don't know if it has plateaued. It does mean transfusions are greatly reduced in these patients, but I don't know if they will ever be fully transfusion free from gene therapy, but with new drugs coming, they will probably become transfusion free once something like Luspatercept is available. bluebird does hope that once they begin trials on  β0/β0 patients with the new protocol that it will be enough to free them from transfusions.

Hi Andy,

Is bluebird planning a new protocol for b0/b0 patients? any idea when it will start? can you please point me to the reference I'd like to read more about it. thanks

CrazyPharm,

I don't know if bluebird has posted the information but both Andy and I have spoken with them and they are quite confident about their new protocol and its ability to free b0/b0 from transfusions.

I will post more once I get further information or once I have a link to this information.