Early week; early trials

A round-up of a few early trial efforts announced Monday, Sept. 23

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With the beginning of a new week, let's do around-up of a few early trial efforts announced Monday, Sept. 23:
Alnylam Pharmaceuticals Inc.
CAMBRIDGE, Mass.—RNAi therapeutics company Alnylamannounced positive interim results from its Phase I clinical trial ofALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting thetransthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR).
Results show that ALN-TTRsc administration led to "robust,consistent, and statistically significant knockdown of serum TTR protein levelsof up to 94 percent." In addition, knockdown of TTR, the disease causingprotein in ATTR, was reportedly found to be rapid, dose dependent, and durable.To date, ALN-TTRsc has been found to be generally safe and well tolerated inthis study.
"These new ALN-TTRsc results are a major milestonein our ATTR program, as well as our entire pipeline of RNAi therapeutics … Webelieve this level of consistent TTR knockdown is exceptional and unmatched,and we now aim to advance ALN-TTRsc in future clinical studies with the goal ofachieving approximately 90 percent TTR knockdown to maximize clinicalefficacy," said Dr. Akshay Vaishnaw, executive vice president and chief medicalofficer of Alnylam. "These new data support our belief that ALN-TTRsc has thepotential to be an important therapeutic for the treatment of familial amyloidoticcardiomyopathy (FAC), a disease for which there are no approved therapies. Withthese results in hand, we are well positioned for continued execution on thisprogram, which includes the initiation of a pilot Phase II study in FACpatients by the end of this year, and—assuming positive results—start of apivotal Phase III trial with ALN-TTRsc by the end of 2014."
BioMarin Pharmaceutical Inc. 
SAN RAFAEL, Calif.—BioMarin announced the dosingof its first patient in the Phase I/II trial for BMN 190, a recombinant human tripeptidylpeptidase 1 (rhTPP1) for the treatment of patients with neuronal ceroidlipofuscinosis type 2 (NCL-2), a form of Batten disease. This is the reportedlyfirst time that a patient with Batten Disease has been treated with an enzymereplacement therapy in a clinical trial setting.
"This program is representative of thecompany's core competency of developing life-altering enzyme replacementtherapies for serious unmet medical needs," said Dr. Hank Fuchs, executivevice president and chief medical officer of BioMarin. "We are inspired andmotivated by the patient and physician community and encouraged by the pharmacologicalactivity demonstrated in preclinical models."
The trial is an open-label, dose-escalation studyin patients with NCL-2. The primary objectives are to evaluate the safety and tolerabilityof BMN 190 and to evaluate effectiveness using an NCL-2-specific rating scalescore in comparison with natural history data after 48 weeks of treatment.Secondary objectives are to evaluate the impact of treatment on brain atrophyin comparison with NCL-2 natural history after 48 weeks of treatment and tocharacterize pharmacokinetics and immunogenicity. The study will enroll approximately22 subjects at up to 10 clinical sites for a treatment duration of 48 weeks.
TRACON Pharmaceuticals 
SAN DIEGO—TRACON, a biopharmaceutical company thatdevelops targeted therapies for oncology and ophthalmology, announced theinitiation of dosing in a clinical trial evaluating the combination of TRC105and axitinib, a vascular endothelial growth factor (VEGF) receptor tyrosinekinase inhibitor (TKI), to treat patients with metastatic renal cell carcinoma.TRC105, a novel monoclonal antibody to endoglin (CD105), is being studied inmultiple clinical trials in combination with agents that target the VEGFpathway. 
The clinical trial is a multicenter, open-label,nonrandomized, Phase Ib, dose-finding study of TRC105 in combination withstandard dose axitinib in patients with advanced renal cell carcinoma.
"Renal cell carcinoma is an angiogenesis-driventumor and despite multiple products approved to treat the disease, resistanceto treatment remains a serious challenge," said Dr. Charles Theuer, presidentand CEO of TRACON. "Combining TRC105 with axitinib in this study buildsupon our clinical experience in combining TRC105 with bevacizumab, presented atASCO in June, where data was presented indicating that the combination ofTRC105 and anti-VEGF treatment was well-tolerated and shrank tumors in patientswho failed prior treatment with VEGF inhibitors. This study is part of a broaddevelopment program for TRC105 that includes two ongoing randomized Phase IIbstudies with Avastin and a trial in combination with VEGF TKI treatment insarcoma."

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