On Friday, small cap biotechArrowhead Pharmaceuticals (NASDAQ: ARWR) jumped 18.28% after hosting an investor & analyst R&D day to introduce its proprietary Targeted RNAi Molecule (TRiMTM) platform and to review its pipeline of RNAi therapeutic candidates. Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead Pharmaceuticals therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein (Arrowheads RNAi-based therapeutics leverage this natural pathway of gene silencing).
The press release stated:
The TRiMTM platform comprises the following components optimized, as needed, for each drug candidate: high affinity targeting ligands; various linker chemistries; structures that enhance pharmacokinetics; and highly potent RNAi triggers with sequence specific stabilization chemistries. The platform offers several competitive advantages, including:
Simplified manufacturing at reduced cost Multiple routes of administration (subcutaneous, intravenous, and inhaled) Faster time to clinical candidates Wide safety margins Promise of taking RNAi to tissues beyond the liver
The CEO was also quoted as saying:
“We are excited to introduce our new TRiMTM platform and the first development candidates to be advanced: ARO-HBV, targeting chronic hepatitis B infection; ARO-AAT, targeting alpha-1 antitrypsin deficiency liver disease; and, ARO-APOC3 and our newest candidate ARO-ANG3, targeting hypertriglyceridemia. TRiMTM is characterized by its ligand-mediated delivery strategy, structural simplicity, and trigger selection technology, which is an important part of TRiMTM that enables us to rapidly develop what we believe are optimal RNAi therapies. It also allows tissue-specific targeting and to that end we are announcing today that we have seen some very exciting results using TRiMTM technology to target genes and diseases in the lung. This opens up a host of new opportunities for disease targets previously not accessible to RNAi therapeutics and Arrowhead is thrilled to be leading the industry in these efforts.”
Traders were apparently optimistic about the new platform and shares have been rising over the past month:
Arrowhead Pharmaceuticals has also reported quarterly losses of $5.52M (3 months ending 2017-06-30), $6.04M (3 months ending 2017-03-31) and $12.09M (3 months ending 2016-12-31)and yearly losses of $81.72M (12 months ending 2016-09-30),$91.94M (12 months ending 2015-09-30),$58.63M (12 months ending 2014-09-30) and $31.14M (12 months ending 2013-09-30). As of the end of the previous quarter, Arrowhead Pharmaceuticals only had $75.14 million in Cash and Short Term Investments.