While buysiders do not expect any sector-moving milestones in the wake of a catalyst-heavy third quarter, they will have at least 21 Phase III milestones and 23 PDUFA dates on their radar.
But the biggest milestones on their minds are commercial milestones: the launches of chimeric antigen receptor T cell (CAR T) therapies Kymriah tisagenlecleucel from Novartis AG and Gilead Sciences Inc.’s newly acquired axicabtagene ciloleucel.
FDA approved Kymriah, the first CAR T therapy, on Aug. 30 to treat patients up to 25 years old with B cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. Kymriah consists of autologous T cells modified to target CD19.
"A lot of people have said the data looks great, but it's such a difficult thing to commercialize," said Loncar Fund's Brad Loncar. "The most serious issue to watch is training new centers to handle toxicities like cytokine release syndrome."
"There is a learning curve that has to be navigated very carefully. It is important that companies roll these treatments out in a controlled manner so that each new center is educated properly and not rushed," he added.
According to its label, Kymriah is available only through a restricted program under a REMS. The REMS includes certification of hospitals and clinics that dispense Kymriah, and training personnel who prescribe or administer the treatment to manage cytokine release syndrome (CRS) and neurological toxicities.
Novartis will restrict the initial distribution of Kymriah to 30-35 centers.
Investors are confident that axicabtagene ciloleucel, which Gilead gained through its acquisition of Kite Pharma Inc., will be approved. It is under FDA Priority Review to treat refractory aggressive non-Hodgkin's lymphoma (NHL) with a Nov. 29 PDUFA date.
“It is important that companies roll these treatments out in a controlled manner.”
"There's no panel being called, so we believe this points towards a smooth approval process," HBM Partners' Ivo Staijen told BioCentury.