Celgene Will Focus On Anti-BCMA Drug, As It Returns CAR-T Pack to bluebird bio

Published: Jun 05, 2015

Celgene (CELG) Will Focus On Anti-BCMA Drug, As It Returns CAR-T Pack to Bluebird
June 4, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor

Celgene Corporation is tightening the reins on an agreement with white-hot gene therapy bluebird bio , saying Thursday that it has chosen to narrow in on just one candidate, a myeloma drug, and return the rest.

“To be exclusive with one partner was not practical in the long term to remain flexible,” Rob Ross, Bluebird’s senior vice president of clinical development, said told analysts on a conference call.

A protein that is common in blood cancers, B-cell maturation antigens have attracted an enormous amount of interest because of their efficacy at treating diseases like multiple myeloma. The drug Celgene chose to keep, dubbed BB2121, has been just as effective, said Ross, who said it “dramatically shrank” multiple myeloma tumors and even treated patients whose cancer did not return.

Under the terms of the revised deal, Bluebird gets a $25 million payment to develop BB2121 into a Phase 1 clinical trial while developing next-generation anti-BCMA product candidates. Celgene has chosen to return its chimeric antigen receptor (CAR) T portfolio, which amps up the body’s own T-cells to kill cancer cells under a form of immunotherapy.

The initial deal, inked in 2013, gave Bluebird an undisclosed lump sum, with an additional $225 million per drug available via option rights and successful clinical and regulatory milestones. The company said that even if Celgene was taking a pass on most of its therapies, it was still pleased by the two-year partnership.

“We have successfully achieved the initial goal of our collaboration with Celgene —identifying a promising lead development candidate in the CAR T cell field — and we are excited to focus our Celgene collaboration on the development of anti-BCMA products,” said Nick Leschly, chief Bluebird, in a statement.

Celgene is a leader in developing and commercializing therapies for multiple myeloma, and we believe they are the best global partner for our first CAR T program. Together we look forward to entering the clinic early next year with BB2121 and continuing our collaboration around next-generation BCMA products.

Last week freshly IPO’d Bluebird said it will pay Five Prime Therapeutics, Inc. $130 million for a variety of CAR-T cell therapies, as the scrappy gene therapy firm attempts to muscle its way into the crowded, and lucrative, immuno-oncology space.

Under the terms of the deal, Bluebird will pay $1.5 million up front for the rights to a host of Five Prime antibodies that block an undisclosed, cancer-related target. Bluebird said in a statement that it hopes to use its own proprietary gene therapy technology to mold those into CAR-T treatments.

Five Prime is also eligible for up to $130 million if it hits specific development, regulatory and commercial milestones, as well as a slice of the royalties if Bluebird’s efforts ever become commercialized.

"CAR T cell therapies have emerged as a very promising approach for treating a number of cancers," said Lewis "Rusty" T. Williams, chief executive officer and president of Five Prime.

"Bluebird Bio brings a wealth of knowledge in the area of gene therapy, a key component of building CAR T therapeutics. We are also impressed by Bluebird Bio's development and manufacturing infrastructure,” he said.

“We feel that Bluebird Bio is well positioned to succeed with converting Five Prime's human antibodies to CAR T cell products that can benefit patients, and we are pleased that our proprietary platform continues to demonstrate its versatility in the field of immuno-oncology."

It’s been a busy spring for Bluebird. Two weeks ago its shares shot up more than 15 percent in morning trading, after the company released an abstract of data that shows its sickle cell anemia drug, LentiGlobin, has kept two patients transfusion free for 14 and 11 months, respectively.

Bluebird released the data from a February test as part of an abstract it will present in June at the European Hematology Association in Vienna, Austria.

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, initial data last fall 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.

Last week’s data had a similar effect on the company’s share price and had executives eager to trumpet the results and explain what they mean, which is that one of the patients treated has no had to seek hospitalization for his sickle cell and has even started producing anti-sickling properties.

“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 for Bluebird.

“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,” he said. “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.”

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