Bluebird bio and Celgene: Encouraging Clinical Trial Results for Last Resort Multiple Myeloma Treatment

A Great Day for Bluebird bio and Celgene

Read the Clinical Trial Results for Myeloma

What a Great Day!

Jobs reaching the moon. Inflation not yet showing any concerning signs. A fiercely rebounding stock market. Those who are still listening to pessimistic opinions are missing the fact that technologies and biotechnology are standing at the starting line. They are not at the finish line. For biotech firms that are ready, willing and able the sky is the limit. Tech firms have finally discovered the ways, the leads and assembled the leaders.

They are experimenting in the right territories and improving on the sophisticated, yet, easy to use tools which are expected to perform unprecedented miracles.

There is plenty of more good and encouraging news in addition to the economy. We chose to only  post one piece of news today. We will tackle many of the rest in the Prohost Letter Issue #431.   

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Bluebird Bio and Celgene

Bluebird bio is a gene therapy, gene editing and immune-oncology firm; as observed per the nature of the firm’s research and development as well as its pipeline products’ actions.

On May 1, 2019 bluebird bio (BLUE) and Celgene Corporation (CELG) announced that the New England Journal of Medicine (NEJM) published interim results from CRB-401 phase 1 study of bb2121. The companies’ lead investigational BCMA-targeted chimeric antigen receptor (CAR) T-cell therapy for patients with relapsed and refractory multiple myeloma (MM).

The NEJM article reported manageable safety and deep and durable responses in the first 33 patients infused with bb2121 BCMA-targeted CAR T-cells. The recruited patients were heavily pre-treated with a median of seven prior multiple myeloma treatment regimens. The prior treatments included: immunomodulatory drugs, proteasome inhibitors and daratumumab in the majority of patients and all but one had previously received an autologous stem cell transplant.

Clinical Results for bb2121

Treatments with bb2121 resulted in an 85% objective response rate (ORR) with 45% of patients achieving a complete response (CR) and an additional 27% of patients achieving a very good partial response (VGPR); to yield a ≥ VGPR rate of 73%. Sixteen responding patients were evaluable for assessment of minimal residual disease (MRD); all tested MRD negative at one or more time points. 

Responses to bb2121 CAR T-cell infusion occurred early. Responses were durable, with a median duration of response of 10.9 months. Researchers observed that greater CAR T-cell expansion occurred in responding patients. Observed responses were independent of tumor or serum BCMA levels. Median progression-free survival among all 33 patients was 11.8 months.

Safety results for bb2121

The most common grade ≥3 events in the first 33 patients were hematologic toxicities. These include: neutropenia (85%), leukopenia (58%), anemia (45%) and thrombocytopenia (45%). Neurotoxicity all-grades occurred in 14 (42%) patients; 13 (39%) were grade ≤2 and one patient (3%) had grade 4 neurotoxicity which resolved within one month. Twenty-five (76%) patients experienced cytokine release syndrome; 23 (70%) were grade ≤2 events and two (6%) were grade 3 events. It is good to learn that all events were reversible.

Infection occurred in 14 (42%) patients; two were grade 3 (6%) and there was no grade 4 events. However, peak CAR T cell expansion was higher in patients with cytokine release syndrome and CAR T-cells remained detectable in the blood in 57% of patients at six months following infusion.

Celgene’s Comments

Alise Reicin, M.D., President, Global Clinical Development for Celgene observed that the results from CRB-401 demonstrate that BCMA is a promising target in the treatment of patients with multiple myeloma. She said that the developing firms continue to be encouraged by the potential of bb2121 as a first-in-class BCMA-targeted CAR T-cell therapy, calling the data compelling considering the fact that the relapsed/refractory patients have been treated with almost all MM- existing products.

Bluebird bio and Celgene continue the follow up of patients in their recently fully enrolled pivotal KarMMa trial. Furthermore, they are evaluating the potential for bb2121 in earlier lines of MM treatment in other KarMMa trials.

Prohost Observations

In immune-oncology, bluebird bio comprises bb2121 and bb21217; CAR T products both for multiple myeloma (MM). The  bb2121 is in four clinical trials for: first, second, third and fourth line therapy for MM. The current data came from a clinical trial for fourth line MM treatment. In other words, when all other treatments have failed.

In addition, the second CAR T called bb21217 is being developed with Celgene as another fourth line immune-oncology treatment for MM.

The CAR T-cells were produced from each patient’s own blood cells which were modified using a proprietary lentiviral vector encoding the anti-BCMA CAR.

In November 2017 the FDA granted bb2121 Breakthrough Therapy Designation (BTD). In Europe the European Medicines Agency granted bb2121 PRIority Medicines (PRIME) eligibility.    

The rest of the bluebird bio pipeline comprises products for severe genetic diseases; including: cerebral adrenoleukodystrophy, 2 Transfusion-dependent Beta-Thalassemias and 2 products for sickle cell disease.

Certainly, the recent data from the CRB-401 trial are, indeed, compelling; knowing, in fact, that the recruited patients have already exhausted almost all existing chemical and biological targeted therapies.

Celgene and Bluebird bio anticipate the potential approval of bb2121 in the U.S. to be in the second half of 2020.  

Prohost appreciates bluebird bio. We believe it will not take long for this firm to become a Top-Tier biotech firm sitting in the Prohost Portfolio Table #1; among the largest and most accomplishing biotech and biopharmaceutical firms.

In conclusion, we feel that Celgene has recently become a greater asset for Bristol-Myers Squibb which is in contrast to the pressure exerted on BMY by traders and general investors. That is to say,we believe BMY will soon fiercely rebound.     

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