CAR-T Was Created to Stay
BlueBird Bio CAR-T Results
Bluebird Bio (BLUE) presentation was well received at ASCO after the firm stated results demonstrating that all the 15 evaluable patients in the clinical trial of its chimeric antigen receptor T cell (CAR-T) drug “bb2121 responded to treatment. Data were convincing demonstrating 89% of the patients observed their tumors shrink with 27% seeing their cancers vanish. The fact that no progression of cancer occurred is also encouraging. So far they the developing firms did not see dose-limiting toxicities for the drug,
The positive results came from an early-stage clinical trial of Bluebird’s/Celgene drug bb2121 in patients with relapsed/refractory multiple myeloma. Bb2121 had a 100% overall response rate in three of four groups of dosed patients, with the other group having an overall response rate of 33%. The patients treated with bb2121 had received various treatments prior to bb2121.
Michael Pehl, Celgene’s hematology and oncology chief said that none of the patients in the active dose groups saw the cancer progress. “The consistency, depth and durability of these patients’ responses coupled with a manageable safety profile is very exciting, and we believe will provide hope for patients in this setting.”
The results are preliminary, but they are convincing enough for analysts and oncology specialists to see Bluebird’s solid scientific fundamentals and its promising pipeline of CAR-T immunotherapy products. The stock is trading around $103.28 Up around $5.35 in today’s trading.
We believe Bluebird is proving itself. The market cap, however, which is around $4.56 billion, and the fact that the firm’s product bb2121 is in early phase trial make us believe that BLUE is in the overbought territory.
We would prefer to wait for a while before buying into the stock, as we believe we can get it at a 10%, or even more discount.
A CAR-T PDUFA DATE
TISAGENLECLEUCEL-T (CTL 109)
According to a note posted by the FDA, independent committee of experts from its Oncologic Drugs Advisory Committee (ODAC) will meet on July 12, 2017 to discuss data from CAR-T Product tisagenlecleucel-T (CTL 109)
Novartis product will be the first CAR-T candidate to be evaluated for approval by a government agency and the outcome might also put the first CAR-T product on the U.S. market to be followed by approvals in EU and around the world. The outcome of the meeting could lead to the approval of the first CAR T immunotherapy product.
Expected to have a PDUFA date this year too is Kite Pharma (KITE), which has already completed its rolling submission for its rival candidate, KTE-C19 a couple of months ago.
If approved, Novartis’ drug CTL 109 could become a blockbuster generating billions of dollars a year and so will be the CAR-T product KTE-C19 CAR-T of Kite. As we said in a previous article, the products, although similar in structure, they aim at treating different cancers. In case of FDA approvals Novartis drug would be approved for relapsed/refractory B-cell acute lymphoblastic leukaemia (ALL) in children and young adults between three and 25 years of age, while Kite’s drug would be approved for non-Hodgkin lymphoma (NHL)
So, there will be no competition between the two drugs.
Ahead of the meeting FDA staff will prepare a document outlining key information and the regulator’s thoughts on the therapy.
The rewards are great, saving condemned lives. Yet, the adverse effects are still huge and life-threatening, which could spoil the parties for many patients. As we keep describing it, the cytokine release syndrome (CRS) can be fatal in some cases. The good news is physicians are becoming capable of preventing it or managing it when it happens. Novartis’s drug CTL109 hasn’t had and deaths in its trials, and the other firms, including Kite (KITE), Bluebird (BLUE), Juno (JUNO), Cellectis (CLLS), Celyad (CYAD), Bellicum (BLCM), Adaptimmune (ADAP) and others are doing a much better job regarding safety.
An approval, or approvals of CAR-T immunotherapy this year if materialize would affect the whole group of CAR-T developers. The efforts spent on perfecting this approach will never stop until they manage to prevent the occurrence of the cytokine release syndrome and to make CAR-T effective in treating solid cancers. Both wishes will materialize, as the efforts are coming from many sources, including academic institutions, research institutions and government agencies.
Reaching its optimal safety and efficacy, the CAR-T revenues will be colossal. All we can say is good luck
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