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FDA Grants SGX Orphan Drug Designation for Troxatyl(TM); SGX to Present Troxatyl(TM) Clinical Data at ASCO

SAN DIEGO, May 12 /PRNewswire/ -- Structural GenomiX, Inc. (SGX) announced today that it has been granted Orphan Drug Designation from the U.S. Food and Drug Administration for its lead product candidate, Troxatyl(TM) (troxacitabine), for the treatment of acute myelogenous leukemia (AML).

Troxatyl(TM) is a novel nucleoside analog that is currently being evaluated by the Company in a Phase 1/2 trial for the treatment of relapsed AML and in a Phase 1/2 trial for the treatment of various solid tumors. The Company plans to complete the Phase 1/2 AML trial and progress Troxatyl(TM) into a Phase 2 trial in relapsed and/or refractory adult AML patients in mid 2005.

Preliminary data from the Company's Phase 1/2 AML trial will be presented in a poster session at the American Society of Clinical Oncology (ASCO) 2005 Annual Meeting in Orlando, Florida.

Abstract Title: Troxacitabine administered by continuous infusion (CI) is
well tolerated and effective in adults with relapsed or refractory acute
myeloid leukemia

Poster Session: Leukemia, Lymphoma, Myeloma, and Transplantation (Adult)
Abstract ID: 3085
Date: Tuesday, May 17, 2005
Time: 8:30AM to 12:30PM

Orphan Drug Designation provides the sponsor of an orphan drug product with eligibility for: seven year marketing exclusivity following marketing approval; tax credits for clinical research; reduced NDA filing fees; grants for further clinical research and development; and assistance with the review of clinical trials protocols.

About Troxatyl(TM)

Troxatyl(TM) is an anti-cancer agent currently in Phase 1/2 acute myelogenous leukemia (AML) clinical trials. SGX plans to complete the ongoing Phase 1/2 studies and progress Troxatyl(TM) into Phase 2 in mid 2005. Over 600 patients have received Troxatyl(TM) in various Phase 1 and 2 studies where delivery of the drug was by bolus intravenous (IV) administration. Preclinical studies have shown that continuous IV infusion results in a significant increase in exposure of cancer cells to Troxatyl(TM). Evaluation of Troxatyl(TM) administered by continuous IV infusion in patients is currently ongoing in clinical studies.

About AML

AML is a hematopoietic stem cell disorder that is the most common form of leukemia, accounting for approximately 90 percent of all acute leukemias in adults. Although induction chemotherapy results in complete remission in 50-75 percent of patients, relapse is very common and long-term survival rates remain at less than 20 percent. Unfortunately, patients with relapsed AML at present have very limited treatment options pointing out the need for the development of new agents in this area.

About SGX

SGX is a biotechnology company focused on the discovery and development of innovative cancer therapeutics. SGX's lead product candidate is Troxatyl(TM), a Phase 1/2 novel cancer therapeutic that has been studied in Acute Myelogenous Leukemia as well as other malignancies including Myelodysplastic Syndrome and various solid tumors. SGX has developed a preclinical pipeline of novel oncology therapeutics using SGX FAST(TM) technology, a proprietary fragment-based approach to lead generation. The SGX pre-clinical oncology pipeline comprises novel inhibitors of the Gleevec® resistant BCR-ABL(T315I) mutant and dual specificity inhibitors of the MET-RON receptor tyrosine kinases. SGX is also pursuing a broad program of lead generation directed against a portfolio of validated oncology targets that include HSP-90 and the Aurora kinases. SGX has secured revenue generating drug discovery and development partnerships with leading pharmaceutical and biotechnology companies including Eli Lilly, Serono S.A. and Roche. For more information, please visit the company's website at www.stromix.com.

Forward-Looking Statements

This press release contains forward-looking statements, including those relating to SGX's plan to advance Troxatyl(TM) for the treatment of AML, and SGX's plans to complete the current Phase 1/2 trial and initiate Phase 2 trials in mid 2005. The clinical development of investigational pharmaceutical products is subject to risks and uncertainties. There can be no assurance that SGX's studies of any of its product candidates can be conducted within the time frame that the company expects, or that the studies will yield positive results. For further discussion of these and other risks and uncertainties, see the various disclosures made by SGX. SGX undertakes no duty to update forward-looking statements.

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Source: Structural GenomiX, Inc.

Revlimid, as oral treatment regimen in chronic lymphocytic leukemia

The clinical data from a Revlimid study in Chronic Lymphocytic Leukemia ( CLL ) were reported at an oral presentation during the 47th American Society of Hematology (ASH) Meeting.

The study reported that sixteen out of nineteen evaluable patients achieved stable disease or better after treatment with Revlimid, and experienced a median decrease of 61 percent in absolute lymphocyte count ( ALC ), a measure of tumor burden.
Five patients achieved complete response ( CR ), two of the five patients achieving molecular ( CR ). Ten patients achieved partial response ( PR ) and three patients achieved stable disease ( SD ).

Progressive disease ( PD ) was reported in three patients.

Chronic lymphocytic leukemia is a hematological cancer that affects approximately 75,000-100,000 people in the U.S.
About 10,000 new cases of chronic lymphocytic leukemia are diagnosed each year and an estimated 5,000 Americans are expected to die of CLL this year.

Thirty-two relapsed or refractory CLL patients, median age of 64 years ( age range: 47-75 ) were enrolled in the trial with all patients available for toxicity and nineteen patients available for response evaluation.

Revlimid was given at 25mg orally every day for 21 days followed by 7 days of rest on a 28-day cycle. Absolute lymphocyte count ( ALC ) at days 0, 7 and 30 were taken to determine direct anti-CLL effect of Revlimid.

Response was assessed at day 30, and then monthly using the National Cancer Institute-Working Group criteria.

CLL patients with stable disease or better response continued on therapy until complete response and those with progressive disease then received Rituximab at ( 375mg/m2 ) added to Revlimid.

Sixteen out of nineteen patients responded with a median decrease of 61% in absolute lymphocyte count ( ALC ), ( range: 55-70% ).

Three patients achieved complete response with 2 achieving a molecular complete response, ten patients achieved progressive disease and three patients achieved stable disease.
Four patients on treatment are too early for response assessment.

Two patients withdrew consent and five patients received less than two months of therapy due to toxicity.

Progressive disease has been observed in three patients, and therefore have received Rituximab. All three patients have responded to the combination therapy.

Toxicity profile was clinically manageable.

The most common side effect was a flare reaction ( tender swelling of lymph nodes and/or rash ) in almost all patients, and tumor lysis syndrome was noted in two patients.

Grade 3 / 4 toxicities included hematologic toxicities in seven patients and febrile neutropenia in three patients.

While the longest follow up is 12 months, further follow-up and analysis will ascertain the durability of these responses and establish the role of Revlimid as a potential treatment of patients with chronic lymphocytic leukemia.

Source: Celgene, 2005

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