Bluebird bio completes split into two companies; BeiGene builds out Brukinsa data at ASH
Bluebird bio has been officially split in half.
Both sides of the once-singular cell and gene therapy company announced Thursday they have completed their split into two biotechs: A gene therapy-focused outfit that will retain the old avian name and a new oncology-focused firm that will go by 2seventy bio.
The move, executives say, will allow each company to focus on bringing therapies to market in fields that require very different types of expertise.
The split came after a series of delays and setbacks for the one of the most prominent biotechs of the last decade. Although bluebird saw Bristol Myers Squibb take the biotech’s CAR-T for multiple myeloma to approval this year, the company has faced repeated manufacturing delays for its gene therapies for sickle cell disease and beta-thalassemia, despite data suggesting the treatments could be near-curative.
The company has had greater success in Europe, where regulators approved the gene therapy for beta-thalassemia and cerebral adrenoleukodystrophy, an otherwise fatal brain disease. But the company decided this year that its therapies were not worth marketing at the rates European governments were willing to pay.
In Germany, for example, authorities offered to pay between $790,000 and $950,000 for treatment. Bluebird asked for $1.8 million, spread out over five years, with each installment given only if patients continue to see benefit.
Bluebird will now focus on getting its three gene therapies approved in the US. 2seventy will focus on another wave of CAR-Ts, including for solid tumors. — Jason Mast
BeiGene builds out Brukinsa data at ASH
Back in July, BeiGene reported results from a Phase III study looking at their Brukinsa drug in first-line chronic lymphocytic leukemia (CLL) or small lymphocytic leukemia (SLL) patients. On Thursday, the company revealed more data at ASH.
Though BeiGene only said the drug was “highly significant” at hitting the primary endpoint of progression-free survival, the p-values came in at a stellar p=<0.0001 and the hazard ratio at 0.42 for both an investigator-backed and independent assessment. The Brukinsa arms were compared to a chemotherapy combo control and the median follow-up was 26.2 months.
BeiGene CMO of Hematology Jane Huang told Endpoints News that while the company isn’t saying when it expects to file for regulatory approvals, it’s working to get Brukinsa to patients in “as many countries as possible.”
Among other figures, BeiGene revealed that Brukinsa had an estimated 24-month PFS of 85.5% compared to 69.5% for the control, though this wasn’t the primary endpoint. For overall response rate, Brukinsa saw a 94.5% rate while the control hit 85.3%.
But for complete response rate, Brukinsa actually fared worse off than the control. The drug saw only 6.6% of patients achieve complete response, compared to 15.1% on chemo.
As BeiGene tries to move Brukinsa into earlier lines of treatment, Huang said she expects a key differentiator will be patients’ ability to continue taking their other medications while getting Brukinsa. That could appeal particularly to the older patients who are usually those diagnosed with CLL and SLL, she said.
“The average person often takes these other drugs, the Prilosecs and the H2 blockers,” Huang said. “Those are things that affect your quality of life if you’re not able to be on them. If you need to take life-saving therapy, most people would take one over the other and take Tums all the time for their heartburn.” — Max Gelman
Aptose engages in $400M agreement to commercialize r/r AML therapy
California biotech Aptose Biosciences entered into an exclusive license agreement with South Korean pharma Hanmi to further develop and commercialize a myeloid kinome inhibitor.
According to a company announcement made this morning, the drug is known as HM43239, an oral clinical-stage myeloid kinome inhibitor designed to target distinct kinases operative in myeloid malignancies, including SYK and FLT3. And in an ongoing Phase I/II clinical trial, the candidate showed anti-leukemic activity and complete responses in patients with r/r AML.
While Hanmi grants Aptose exclusive worldwide rights to HM43239 for all indications, Hanmi will receive $12.5 million upfront — $5 million in cash and $7.5 million in Aptose shares. There’s also potential for Hanmi to receive up to $407.5 million in possible milestone payments.
“We believe that HM43239 has a clear development and commercial path, while being a natural fit with our strategic focus, technical expertise, and clinical experience,” said Aptose chairman, president and CEO William Rice in a statement. — Paul Schloesser