- On Sunday, at the American Society of Hematology meeting in Orlando, Novartis presented the latest results of a developmental CAR-T treatment for patients with refractory diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma. The treatment tested the CTL019 therapy.
- Another major presentation from Bluebird Bio, on the company's gene therapy LentiGlobin BB305, concerned investors and led to a more than 35% slump in Bluebird's stock in early Monday trading.
- Response rates for Novartis' treatment in refractory DLBCL and follicular lymphoma were 47% and 73%, respectively. These results will likely help position the treatment for a 2017 FDA submission
- CTL019 is also being tested in children with advanced leukemia. Results in this group have been promising.
- As for Bluebird, two patients with severe sickle cell disease (SCD) in a phase 1 trial who received LentiGlobin infusions had only about half of the amount of anti-sickling hemoglobin that would be required to make a major change in their disease three months after receiving the treatment.
In July 2014, the FDA granted Novartis' CLT019 a Breakthrough Therapy designation. Since then, the company and co-developer University of Pennsylvania Perelman School of Medicine have methodically tested CTL019 in diverse groups of patients with various types of blood cancers. The results have consistently been better than standard-of-care.
Each year, approximately 25,000 people are diagnosed with DLBCL, and another 10,000 people die. Ideally, patients treated with CTL019 would only need to receive one treatment. While CTL019 has plenty of upside, four patients developed an inflammatory, flu-like response, and two developed neurologic toxicity. The treatment was tested on a population of 26 adult patients, 15 with DLBCL and 11 with follicular lymphoma.
Still, demand will be high for CTL019 and other CAR therapies, although prices may reach as high as $450,000. The goal over time will be to make the therapies available to more patients at lower prices while addressing side effects quickly and effectively.
Bluebird shares have been in freefall on Monday morning thanks to the sobering data presented over the weekend. Gene therapy is a tricky business due to the highly personalized nature of the treatment (and, consequently, the often-divergent nature of the treatment's outcomes).
It wasn't all bad news for the biotech, as a pair of beta thalassemia patients treated with a single LentiGlobin infusion did not require blood transfusions nearly two years out, and five patients with a semi-working beta-globin gene didn't require transfusions for anywhere from 7.1 to 16.4 months.
For its part, the company says that it will begin a new clinical approach to the therapy, which has proved more complicated than it hoped, by further separating out the patient populations.