Drug Detail

Information about Retaspimycin hydrochloride

Generic Name
Retaspimycin hydrochloride
Brand Name (US)
Drug Type
Tyrosine Kinase Inhibitor
Approval Status
Phase 3
Overall Strategy
KIT Protein Based
Destroy KIT
Drug Category
HSP90 inhibitor

CAMBRIDGE, Mass., April 15, 2009 (GLOBE NEWSWIRE) -- Infinity Pharmaceuticals, Inc. today announced that, based on the recommendation of its independent data monitoring committee (IDMC), Infinity has elected to terminate the RING trial, a double-blind, placebo-controlled international Phase 3 registration trial of IPI-504 (retaspimycin hydrochloride) in patients with refractory gastrointestinal stromal tumors (GIST). See the links under Links and Trial Results.

The IDMC's recommendation yesterday evening to halt the trial was based on an early review of safety data from the first 46 patients enrolled in the study, which showed a higher than anticipated mortality rate among patients enrolled in the treatment arm.
The heat shock chaperone protein (HSP-90) has emerged as a promising target for cancer therapy. Infinity Pharmaceuticals’ IPI-504 is one of many experimental heat shock protein inhibitors vying for a spot in the cancer market. Data from the recently completed Phase I trial of this drug was presented at the 2008 American Society of Clinical Oncology conference (ASCO) in May.
In preclinical work performed with Dr. Jonathan Fletcher’s lab at Brigham and Women’s Hospital in Boston, Infinity Pharmaceuticals has demonstrated that IPI-504 kills GIST cancer cells as effectively as Gleevec in vitro (in the test tube). Moreover, when cancer cells have mutations that make them resistant to Gleevec, IPI-504 kills these cells with even greater effectiveness. The more mutated the GIST cells become, the more sensitive they are to IPI-504.
IPI-504 is Infinity’s novel anti-cancer agent that potently and selectively inhibits Hsp90. IPI-504 has broad anti-tumor activity in animal models as a single agent as well as in combination with existing anti-cancer therapeutics.

Research shows that inhibition of Hsp90 forces cancer cells to “commit suicide” through a process of programmed cell death or apoptosis



Infinity halts the phase III RING trial in GIST


LRG story - IPI-504 completes phase I


LRG story - Infinity launches trial of IPI-504 for GIST


LRG story - Resistant GIST may fall to new inhibitor


LRG story - IPI-504 enters phase III


Infinity website - pipeline, IPI-504

Trials of this drug


Safety Study of IPI-504 in Patients With Gastrointestinal Stromal Tumors (GIST) or Soft Tissue Sarcomas (STS)


Study of IPI-504 in GIST patients following failure of at least imatinib and sunitinib

Trial results

  Infinity Announces Clinical Data Confirming Activity and Safety of IPI-504, a Novel Hsp90 Inhibitor, in Patients With Refractory GIST
  Infinity Halts RING Trial in Advanced Gastrointestinal Stromal Tumors
  ASCO 2008 - Inhibition of heat shock protein 90 (Hsp90) with the novel agent IPI-504 in metastatic GIST following failure of tyrosine kinase inhibitors (TKIs) or other sarcomas: Clinical results from phase I trial.
  PDF presentation - Results from the Phase I Trial of IPI-504 in TKI resistant GIST and other sarcomas
  Infinity halts phase III RING trial in GIST due to higher than anticipated mortality rate among patients in the treatment arm
  2010 GI ASCO: Final results from a phase III study of IPI-504 vs placebo in GIST patients following failure of kinase inhibitor therapies
  Poster: Final results from a phase III study of IPI-504 vs placebo in patients with GIST following failure of TKI therapy

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