|Jazz Pharma and Concert Pharma Provide JZP-386 Update; ARIAD Reports Follow-up Data from Phase 1, PACE Trials of Ponatinib|
|By William Kent|
|Tuesday, 09 December 2014 20:13|
Jazz Pharmaceuticals plc (NASDAQ:JAZZ) and Concert Pharmaceuticals, Inc. (NASDAQ:CNCE) announced Phase 1 clinical data generated to date supports completing the Phase 1 evaluation of JZP-386 at the originally planned highest dose, which was not administered in the first Phase 1 trial due to a technical dosing issue. The existing Phase 1 clinical data was generated in a first-in-human trial evaluating the safety, pharmacokinetics, and pharmacodynamics of JZP-386; enrollment was completed in the third quarter. A second Phase 1 trial evaluating JZP-386 at the originally planned highest dose is expected to be initiated in the first quarter of 2015, with data expected in the second quarter of 2015.
JZP-386 is a deuterium-containing analog of sodium oxybate. The Phase 1 program is comparing JZP-386 to sodium oxybate versus placebo in healthy volunteers. The companies expect that the results from the upcoming Phase 1 trial will inform the next steps in the development program for JZP-386.
Sodium oxybate is the active ingredient in Xyrem®, a prescription medicine marketed in the United States by Jazz Pharmaceuticals to treat cataplexy and excessive daytime sleepiness in patients with narcolepsy, a serious neurological disorder that affects approximately 1 in 2000 people in the United States.
ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) announced safety and efficacy follow-up data on Iclusig® (ponatinib), its approved BCR-ABL inhibitor, in patients with a baseline T315I mutation from its Phase 1 and Phase 2 PACE trials in heavily pretreated patients with resistant or intolerant chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).
With a median follow-up of three years for CP-CML patients and nine months overall, Iclusig continues to exhibit responses in patients with the T315I mutation, for whom there is no other approved tyrosine kinase inhibitor (TKI) therapy. Among patients with the T315I mutation, major cytogenetic response (MCyR) was achieved by 92 percent (11/12) of chronic phase CML patients (CP-CML) in the Phase 1 trial and 72 percent (46/64) of CP-CML patients in the PACE trial; taken together, the combined MCyR rate for all T315I CP-CML patients was 75 percent. Long-term safety data show that careful benefit-risk evaluations should guide decisions to use and maintain ponatinib therapy, particularly in patients who may be at increased risk for arterial thrombotic events.
These data were featured on Monday December 8, at the 56th Annual Meeting of the American Society of Hematology (ASH) taking place in San Francisco.
“For CML patients with the T315I mutation, there were few treatment options available prior to ponatinib, and prognosis for these patients was poor. Now, with nearly six years of treatment for some patients in these trials, we have longer term data demonstrating that ponatinib continues to evoke responses in these CP-CML patients,” stated Michael J. Mauro, M.D., Leader, Myeloproliferative Neoplasms Programs, at the Memorial Sloan Kettering Cancer Center. “With these follow-up data, overall survival of 78 percent is now estimated at three years for chronic-phase patients with the T315I mutation in these trials.”
Phase 1 and PACE Data in Patients with T315I - The analysis describes the pooled efficacy and safety of ponatinib in patients with a T315I mutation detected at baseline from the ongoing Phase 1 dose-escalation study and Phase 2 PACE trials. The analysis includes 147 patients with a T315I mutation from the Phase 1 (n=19) and PACE (n=128) trials. Half of the 76 CP-CML patients with T315I remain on treatment and 44 (30%) total patients (all disease phases) with T315I remain on treatment in the respective trials. Data presented on these patients are as of September 26, 2014 for the Phase 1 trial and October 6, 2014 for the PACE trial.
“Ponatinib is the only TKI approved for patients with the T315I mutation of BCR-ABL, which is a common mutation that is associated with resistance in Philadelphia-positive leukemias,” said Frank G. Haluska, M.D., Ph.D., chief medical officer and senior vice president, clinical R&D at ARIAD Pharmaceuticals. “The combined data from the two ongoing trials of ponatinib show continued, clinically meaningful responses in this difficult-to-treat patient population.”
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