SuperGen Inc. reported data from a combination trial that included its anticancer drug, Nipent, in allogeneic bone marrow transplant patients with a variety of leukemias and lymphomas at the 44th annual meeting of the American Society of Hematology in Philadelphia.
SuperGen, of Dublin, Calif., presented Phase II data on 90 patients who were given a regimen of photopheresis, radiation and Nipent two days before an allogeneic bone marrow transplant. Ninety-four percent of patients, or 85 out of 90, experienced full bone marrow engraftment within 60 days. Most transplant patients have a remission rate of 40 percent, but 77 percent of the patients in the Nipent trial achieved remission.
There was only a 10 percent incidence of graft-vs.-host disease in the trial, compared to a rate between 40 percent and 50 percent in current transplant regimens, SuperGen said. After one year, 87 percent of patients (n=78) were still alive.
Nipent is indicated as a single-agent treatment for both untreated and alpha-interferon-refractory hairy-cell leukemia patients with active disease as defined by clinically significant anemia, neutropenia, thrombocytopenia or disease-related symptoms. SuperGen's CEO and chairman said the company would "devote the resources necessary to further study this very promising development."
SuperGen's stock (NASDAQ:SUPG) rose 11 cents Monday to close at $4.29.
In other news from the meeting:
• Alexion Pharmaceuticals Inc., of Cheshire, Conn., presented preliminary results of an ongoing open-label Phase Ib trial examining eculizumab in 11 paroxysmal nocturnal hemoglobinuria patients. Eculizumab administration for three months was associated with a 68 percent reduction in the need for blood transfusions, and an 81 percent reduction in biochemical parameters of hemolysis. Clinical paroxysms were reduced from 2.1 days of paroxysms/patient/month to 0.2 days of paroxysms/patient/month, a reduction of 90 percent. Eculizumab also is being studied in a Phase IIb trial in rheumatoid arthritis and a Phase II trial in membranous nephritis.
• Avigen Inc., of Alameda, Calif., said its gene transfer product for the treatment of hemophilia B, Coagulin-B, achieved therapeutic levels of circulating Factor IX in an ongoing clinical trial that included six subjects. The interim data from the ongoing Phase I/II trial enrolled patients who are given Avigen's adeno-associated virus gene therapy vector containing the Factor IX gene, infused into the liver. The company said the trial is a "great stride forward in the development of a clinically practical gene therapy for hemophilia B." Avigen's stock (NASDAQ:AVGN) fell $1.65 Monday, or 17.6 percent, to close at $7.75.
• Celgene Corp., of Warren, N.J., said preliminary results were presented from a Phase II trial of Revimid in relapsed or refractory multiple myeloma. The data demonstrated that 39 of 46 evaluable patients (85 percent) with progressive disease experienced a reduction or stabilization in their paraprotein levels. Five of seven patients who experienced progressive disease on Revimid monotherapy subsequently experienced a reduction or stabilization of paraprotein levels after Revimid plus dexamethasone combination therapy. Forty-four of 46 evaluable patients (96 percent) experienced a reduction or stabilization of paraprotein levels after either Revimid monotherapy or Revimid plus dexamethasone combination therapy.
• Cell Therapeutics Inc., of Seattle, presented data on Trisenox, saying the drug alone or in combination with vitamin C or chemotherapy in patients with relapsed/refractory multiple myeloma is well tolerated with manageable side effects and it produces clinically relevant responses in the majority of patients. Also, disease control was observed in almost all evaluable patients in the multiple myeloma studies. It also presented a Phase II study that enrolled 24 patients who were treated with Trisenox five days a week, two weeks on and two weeks off in repeated four-week cycles until disease progression or response was achieved. Twenty relapsed and four refractory patients were treated, most having failed two or more prior chemotherapy regimens with seven failing stem cell transplants. Three patients were not evaluable for response. Ten of 21 patients (48 percent) had an objective response as measured by a greater than 25 percent decrease in serum myeloma protein and eight patients (38 percent) had stable disease, for an overall disease control rate of 86 percent.
• Genitope Corp., of Redwood City, Calif., reported results of a Phase II trial of an adjuvant that is administered with the company's personalized vaccine-like immunotherapy, GTOP-99, for the treatment of follicular non-Hodgkin's lymphoma (fNHL). The data demonstrate that an abbreviated course of the adjuvant GM-CSF, recombinant human granulocyte macrophage-colony stimulating factor, can induce an immune response in fNHL patients when administered with GTOP-99. GTOP-99 was developed by Genitope and a pivotal Phase III randomized study is ongoing at more than 25 sites in the U.S. and Canada in patients with fNHL. It is expected to enroll 360 evaluable patients.
• Genta Inc., of Berkeley Heights, N.J., in collaboration with Aventis SA, of Frankfurt, Germany, presented data on the first 23 patients in a Phase II trial of Genasense in patients with chronic lymphocytic leukemia (CLL). Data showed that two patients who had failed four or more prior treatment regimens achieved a partial response. Eleven patients achieved stabilization of their disease, five of whom had failed four or more treatments. Eight of 19 patients achieved greater than 50 percent decrease in the size of enlarged lymph nodes. Eight of 16 patients achieved greater than 50 percent decrease in the size of enlarged liver or spleen. Also, circulating CLL cells were reduced by more than 50 percent in nine patients. Genasense works by inhibiting the production of Bcl-2.
• Immunomedics Inc., of Morris Plains, N.J., presented Phase I/II data on epratuzumab in indolent and aggressive non-Hodgkin's lymphoma (NHL). Eighteen percent showed an objective response among all 51 patients (various histology types and doses) with indolent NHL, but at the optimal dose of 360 mg/m2 weekly for four weeks, 43 percent of the follicular patients showed an objective response, with one-third being complete responders. The best results in the aggressive NHL group were found in patients with diffuse large-cell lymphoma, showing an objective response of 34 percent at the 240 mg/m2 dose and 16 percent at the 360 mg/m2 dose, with half of these patients being complete responders. Also, the company presented a new humanized antibody against the CD20 marker of normal and malignant B-lymphocytes, called hA20. The antibody was reported as having similar constructs as the humanized CD22-binding antibody, epratuzumab.
• Inex Pharmaceuticals Corp., of Vancouver, British Columbia, presented interim results from two Phase II trials showing its lead anticancer product, Onco TCS, can work in combination with other cancer drugs to reduce the size of tumors in patients with aggressive non-Hodgkin's lymphoma (NHL). Inex released interim results from 69 patients in a Phase II trial in which Onco TCS was used in combination first-line therapy. All 69 patients responded to the therapy. Sixty-seven patients (97 percent) completely responded and had all tumors eliminated. Two patients partially responded and had tumor volume decreased by more than 50 percent, for an overall response rate of 100 percent. Inex also released interim results from a Phase II trial in which 9 patients were treated with a combination of Onco TCS and Rituxan (rituximab). A total of 6 patients responded to the therapy - three patients (33 percent) completely responded and had their tumors eliminated and three patients partially responded and had tumor volumes decreased by more than 50 percent, for an overall response rate of 67 percent. The company is planning a new drug application filing on Phase II/III data in aggressive non-Hodgkin's lymphoma. (See BioWorld Today, Dec. 4, 2002.)
• Ligand Pharmaceuticals Inc., of San Diego, said Ontak (denileukin diftitox) might benefit patients with chronic lymphocytic leukemia, B- and T-cell non-Hodgkin's lymphoma and graft-vs.-host disease after allogeneic hematopoietic stem cell transplantation, according to five abstracts from clinical trials presented or published at the meeting. In February 1999, the FDA granted Seragen Inc., a wholly owned subsidiary of Ligand, marketing approval for Ontak for the treatment of patients with persistent or recurrent CTCL whose malignant cells express the p55 (CD25) component of the interleukin-2 receptor.
• Medarex Inc., of Princeton, N.J., said treated with MDX-060, its fully human antibody, resulted in tumor regression in SCID mice xenografted with CD30-positive lymphomas. Two of five mice experienced a complete disappearance of established subcutaneous tumors. In a second study, three of five mice showed no signs of disease after treatment in a disseminated tumor animal model. MDX-060 is in a Phase I/II study evaluating the product in up to 40 patients with refractory or relapsed Hodgkin's lymphoma, anaplastic large-cell lymphoma and other CD30-positive lymphomas.