• Celgene (CELG) is due to release long-awaited midstage trial results on GED-0301, or mongersen, a drug for Crohn's disease that it licensed in 2014 for $700 million. The drug initially sparked some excitement on Wall Street because of its strong two-week remission rate, which beat even the leading injected anti-tumor necrosis factor drugs, such as AbbVie's (ABBV) Humira, Johnson & Johnson's (JNJ) Remicade and Amgen's (AMGN) Enbrel. But the next data set will cover a full year and evaluate patients by endoscopy rather than just symptoms.
Investors' confidence was somewhat shaken when Celgene refused to provide an interim analysis on the trial, which some interpreted as a lack of confidence in the numbers. Leerink analyst Geoffrey Porges wrote that a close inspection of earlier data also raises red flags.
"If the trial is consistent with the earlier phase I/II result (on which the original investment was made), the endoscopic remission rate would be in the 60-70% range, which is well above that seen with even the best biologicals or immunomodulators," Porges wrote in a July 25 research note. "In fact, our assessment of the earlier results, and the lack of CRP (C-reactive protein) response, which is a biomarker of Crohn's disease inflammatory activity, suggests to us that the result could be materially lower than the anti-TNFs, with remission rates more likely to be in the 20-30% range."
He noted that since GED-0301 is oral rather than injected, it could still find a market niche, though it would not be the mega-blockbuster initially hoped for.
• Kite Pharma (KITE) is expected to release headline data from Zuma-1, a phase-two trial of its CAR-T cell therapy KTE-019 in diffuse large B-cell lymphoma. CAR-T cells, or chimeric antigen receptor-T cells, are genetically engineered to attack cancerous cells. The trial results are crucial because Kite hopes to use them to file for FDA approval of the treatment, which could make it the first CAR-T therapy to hit the market next year.
RBC Capital Markets analyst Michael Yee estimated Friday that the study would yield a 60% to 70% overall response rate (percent of tumors that shrank) and perhaps a 40% complete response rate (tumor disappearance) based on earlier data. But in an initiation report Tuesday, BTIG analyst Dane Leone wrote that much depends on how the data stack up against results from competitors Juno Therapeutics (JUNO) and Novartis (NVS), which is as yet unknown.
"Novartis may present pivotal data sets at ASH 2016 along with the potential for first data from J&J's CD19 bi‐specific antibody," wrote Leone, referring to the American Society for Hematology conference in December. "Juno is also expected to present clinical updates from ongoing CD19 trials."
• Vertex Pharmaceuticals (VRTX) should provide a report on its "triplet" pills for cystic fibrosis, consisting of its already marketed drug, ivacaftor (Kalydeco), combined with experimental drug VX-661 and one of two additional drugs, VX-152 and VX-440.
Another trial of ivacaftor and VX-661 alone recently failed, but it did present a clean safety profile and analysts have been expecting a triplet to be the next-generation treatment in any case.
Also this month, the FDA is due to render a verdict on whether to expand the label on Vertex's already marketed CF drug Orkambi to children aged 6 to 11.
For the complete article please visit Investor's Business Daily
