Download Now - The Burrill Report

PIPELINEThe Burrill Report❱❱ (continued)Clinical Trials for September 2012COMPANY TICKER DRUG INDICATION RESULT NOTESPeregrine PharmaceuticalsAlnylam PharmaceuticalsElevation PharmaceuticalsPHASE 1BioMarin PharmaceuticalsNasdaq:PPHMNasdaq:ALNYBavituximab Lung cancer Positive The interim data showed a statistically significant improvementin overall survival and a doubling of medianoverall survival in the bavituximab-containing arms comparedto the control arm.ALN-RSV01Respiratory SynyctialVirusPrivate EP-101 Chronic obstructivepulmonarydiseaseNasdaq:BMRNMerck NYSE: MRK lonafarnib Progeria (kid’saging)Antares PharmaNasdaq:ATRSFailed As reported previously, the study narrowly missed theprimary endpoint of reduced day 180 BOS in an “intentto-treat”analysis of confirmed RSV infected patients,but achieved statistically significant reductions inprospectively defined analyses of ITTc patients with their“last observation carried forward”, and of ITTc patientstreated “per protocol”.PositiveAs previously announced, EP-101 demonstrated a rapidonset, dose-related, statistically significant improvementin lung function compared to placebo.BMN-111 Achondroplasia Positive BMN-111 was generally well-tolerated. Mild, transient,self-limited hypotension was observed. The majority ofthese cases were asymptomatic, and only observed uponassumption of an upright posture following recumbence.No dose-limiting toxicities were identified outsideof these cardiovascular findings. Systemic exposure toBMN-111 was similar at these doses to what has beenobserved to cause growth in healthy and disease modelanimals. All adverse events were of mild severity.VIBEX MethotrexateRheumatoidarthritisVersartis Private VRS-317 Endocrine disordersCell TherapeuticsImmune TargetingSystemsNasdaq:CTICPositivePositivePositiveProgeria patients who used a farnesyl transferase inhibitorscalled lonafarnib were able to improve their cardiovascularhealth and increase their weight gain, problemsusually associated with the condition.The results of this study show that self-administration ofMTX using the VIBEX Medi-Jet is safe and well tolerated.Following standardized training by site personnel andreview of written instructions, all 101 patients performedthe self-administration successfully. In addition, theVIBEX Medi-Jet functioned correctly and as intended foreach and every administration thereby demonstratingreliability and robustness.The clinical data presented today demonstrate thatsingle doses of VRS-317 sustained IGF-1 responses in thenormal range in a dose dependent and tunable manner.Based on its potential to provide persistent biologicalresponses for up to one month, VRS-317 is currently theonly long acting rhGH compound with a future for oncemonthly dosing.Pacritinib Lymphoma Positive The phase 1 study data of the company’s highly selectiveoral JAK2 inhibitor pacritinib, or SB1518, showedencouraging anti-tumor activity and good tolerability in34 patients with relapsed/refractory lymphoma.Private Flunisyn Influenza A Positive The first phase I trial showed that Flunisyn generated statisticallysignificant cell-mediated immune responses tothe vaccine and was safe and well tolerated in vaccinatedgroups compared to placebo.(continued) ❱❱November 2012 42