Alexza Pharmaceuticals, Inc. (Nasdaq: ALXA) appeared before the Psychopharmacologic Drugs Advisory Committee (PDAC) today where the committee was divided on whether Adasuve should be approved as a single dose when used with FDA proposed REMS, with 9 votes FOR, 8 votes AGAINST and one abstention. It had earlier voted that Adasuve had been shown to be effective but voted that it would not be safe even with if used in conjunction with the REMS proposed by the FDA. It has a PDUFA date of February 4, 2012.
YM BioSciences Inc. (NYSE Amex: YMI), today reported at ASH updated results from the ongoing Phase 1/2 study of CYT387 for the treatment of myelofibrosis. The Phase 1/2 study has completed enrolment of 166 myelofibrosis patients across six study sites. 65% have Primary Myelofibrosis, 22% have Post-Polycythemia vera and 14% have Post-Essential thrombocythemia. 32 patients (19%) have discontinued the study, five for possibly or probably related adverse events.
Of the 68 patients who were transfusion dependent at baseline, to date 54% have become transfusion independent for a minimum of 12 weeks. Of the 26 patients who were dosed at 300mg QD and were transfusion dependent at baseline, to date 65% have become transfusion independent for a minimum of 12 weeks.
Of the 142 patients evaluable for spleen response, 31% achieved a response. To date, 49% of patients achieved more than a 50% maximal decrease in spleen size from baseline, with 87% achieving more than a 25% maximal decrease. Of the 51 patients who were dosed at 300mg QD and were evaluable for spleen response, 33% achieved a response. Eleven patients were evaluable for spleen response both by MRI and by palpation. The response rate was 64% by MRI (defined as a 35% decrease in spleen volume) and 45% by palpation (defined as a 50% decrease in spleen length).
YMI indicated that it hopes to initiate pivotal trials in mid-2012, likely with the 300mg dose.
CytRx Corporation (Nasdaq: CYTR) released results at ASH from the open-label, single-agent Phase 2 ENABLE trial of bafetinib in patients with relapsed or refractory B-cell chronic lymphocytic leukemia (B-CLL). Six of 12 (50%) of the evaluable patients achieved 30% or greater shrinkage of the lymph node and spleen, and four of 12 (33%) patients had stable disease. No serious adverse events (SAEs) were observed at the dose of 240 mg twice daily, which is the dose that would likely be used in any future clinical trials in chronic lymphocytic leukemia. Two patients remain in the trial. However, it noted that it will focus its efforts on its other two drug candidates, INNO-206 and tamibarotene and will need a partner to advance bafetinib. It faces either a reverse split to remain listed on NASDAQ or a delist to OTC, by early February 2012.
Geron Corporation (Nasdaq: GERN) announced the initiation of GRABM-B (GRN1005 Against BrainMetastases – Breast Cancer), a Phase 2 clinical trial to evaluate GRN1005 in patients with brain metastases arising from breast cancer.
Nektar Therapeutics (Nasdaq: NKTR) announced today that it has initiated its pivotal Phase 3 global clinical trial evaluating NKTR-102 as a single agent in women with metastatic breast cancer. The BEACON Study (BrEAst Cancer Outcomes withNKTR-102) is designed to include approximately 840 metastatic breast cancer patients who have had prior treatment with anthracycline, taxane and capecitabine in either the adjuvant or metastatic setting. The primary endpoint of the BEACON study will be overall survival.
Optimer Pharmaceuticals, Inc. (NASDAQ: OPTR) and Astellas Pharma Europe Ltd.,noted today that it received European Marketing Authorization (MA) for DIFICLIR (fidaxomicin) tablets for the treatment of adults with Clostridium difficile infections (CDI).
Pharmacyclics, Inc. (Nasdaq: PCYC), reported today interim results at ASH from its ongoing Phase 2 trial of PCI-32765 in MCL (Mantle Cell Lymphoma). This interim analysis included a total of 68 patients accrued to this Phase 2 trial. 51 patients (31 patients had bortezomib-naive disease, 20 patients had previously received bortezomib) had post-baseline tumor assessments and were thus evaluable for response. The ORR was 69% (35/51 patients). ORR was similar in bortezomib-naive and bortezomib-exposed patients (71% and 65%, respectively). At the time of this analysis 31 of 35 (89%) responding patients have ongoing responses with the median follow-up of 3.7 months. The most common adverse events reported in this trial were Grade 1 (mild) or 2 (moderate) fatigue, diarrhea and nausea. Three patients discontinued the study due to adverse events regardless of causality.
Immunomedics, Inc.(NASDAQ: IMMU) announced interim data from its Phase 1/2 trial of veltuzumab in patients with immune thrombocytopenia (ITP). Patients showed an overall objective response (OR) rate of 67%, including an 18% durable complete response (CR) rate, in 39 evaluable ). For the 9 patients with ITP for LESS than one year and treated with steroids and/or immunoglobulins, 7 (78%) achieved ORs, including 3 (33%) with CRs. For the rest of the patients who had ITP for more than 1 year, 19 of 30 patients (63%) achieved ORs, including CRs in 4 patients (13%) with ITP for 1.4, 2.0, 2.4 and 25 years.