On December 6, 2017 Sirnaomics, Inc. (www.sirnaomics.com), a leading biopharmaceutical company in discovery and development of RNAi therapeutics, reported that the Office of Orphan Product Development division of the FDA has granted Orphan-drug designation to its leading therapeutic candidate, STP705, for the treatment of Cholangiocarcinoma (CCA) (Press release, Sirnaomics, DEC 6, 2017, View Source [SID1234523487]).
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Sirnaomics lead product candidate, STP705, is an siRNA (small interfering RNA) therapeutic which takes advantage of a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to directly knock down both TGF-β1 and COX-2 gene expression. The product has also received both US FDA and Chinese FDA IND approval for Hypertrophic Scar Reduction and it is expected to have efficacy in many diseases across multiple therapeutic areas. The Orphan Drug application was supported by recent results of in vitro and in vivo studies using a human cholangiocarcinoma cell line and its xenograft tumor in a mouse model.
"We are very pleased to reach this significant milestone and we are very excited to develop our lead product candidate for this oncology indication," stated Dr. Patrick Lu, founder and CEO. "This is the fourth major clinical milestone we have achieved in the past twelve months including our FDA and CFDA IND approval for STP705 for the treatment of Hypertrophic scar as well as our Orphan Drug Designation approval for Primary Sclerosing Cholangitis. The antifibrotic and antitumorigenic properties of STP705 may bring an alternative therapeutic approach for treatment of devastating cancers such as cholangiocarcinoma and others."
"The Orphan designation for CCA is a very important step for Sirnaomics and the positive data allows us to develop STP705 in a devastating oncology disease with no effective therapy," stated Dr. Michael Molyneaux, Sirnaomics’ CMO. "This Orphan designation aligns with our mission to alleviate human suffering and target diseases with high unmet clinical need and we anticipate filing an IND for CCA sometime in the first half of 2018."