4:10 PM
Oct 06, 2017
 |  BioCentury  |  Finance

Coasting on catalysts

Investors are watching CAR T, ASH, ophthalmology and Orphan therapies in 4Q17

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.”

Brad Loncar, Loncar Fund

"There's no panel being called, so we believe this points towards a smooth approval process," HBM Partners' Ivo Staijen told BioCentury.

But the buysiders wonder whether Gilead’s price and pricing model will be similar to Novartis’. The pharma launched Kymriah with a $475,000 price tag and an outcomes-based arrangement with CMS. CMS will not pay for the treatment for patients who do not respond within one month.

"Will they strike a similar deal to Novartis? I am really curious about the price and the way it will be commercialized," said LSP's Joep Muijrers.

Axicabtagene, which also comprises autologous T cells genetically modified to target CD19, is also under review in Europe to treat relapsed or refractory diffuse large B cell lymphoma (DLBCL), transformed follicular lymphoma and primary mediastinal B cell lymphoma (PMBCL) in patients ineligible for autologous stem cell transplantation (ASCT). A decision is expected in 2018.

Out of ASH

Some buysiders will be watching for incremental cancer immunotherapy and hemophilia updates out of the American Society of Hematology (ASH) meeting Dec. 9-12 in Atlanta.

Muijrers hopes to see data that "gives us more conviction as to what future combo events will look like.

While he did not name specific combinations, he said his portfolio includes Aduro Biotech Inc. and CytomX Therapeutics Inc., which both have agents that could expand the range of indications that benefit from checkpoint inhibitors.

CytomX is using its Probody platform to improve the safety of cancer immunotherapies including checkpoint inhibitors, antibody-drug conjugates (ADCs) and T cell-engaging bispecific antibodies by restricting their activity to tumors.

Lead candidate CX-072 is a Probody targeting PD-L1 that in Phase I/II testing to treat lymphoma or solid tumors. Data are expected in 2018.

Aduro is developing cyclic dinucleotide-based STING agonists for cancer. Lead program ADU-S100 is in a Phase Ib trial in combination with Novartis' PD-1 antibody PDR001 to treat advanced solid tumors or lymphomas....

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