InteRNA Technologies B.V.
Breakthrough for treatment of cancer, and other diseases
InteRNA Technologies BV is a Utrecht based biotech company that is developing new therapeutics for cancer and other diseases. The approach is based on science developed at the Hubrecht Institute in the early 2000s. The company is a pioneer in the discovery and development of a new class of drugs, called microRNAs. MicroRNAs have the potential to achieve biological and therapeutic outcomes that no other drug platform can and as such may provide a breakthrough in drug effectiveness.
InteRNA: an attractive investment case
The existence of microRNAs in the human body was first discovered in 2000; their role in cancer was first implicated only in 2002.
MicroRNAs are small RNA molecules that regulate the translation of your DNA code into all protein functions in a cell, and play an important role in cell growth and proliferation.
• To date, an extensive family of microRNA compounds has been discovered that have potential as pharmaceuticals for diverse disease indications, including cancer and orphan diseases.
• InteRNA’s founders discovered 25% of all human microRNAs currently known worldwide. The company is one of the true pioneers in the field.
• InteRNA has developed a proprietary drug discovery platform based on a library of 1100 microRNA drug candidates. For the last 4 years InteRNA systematically screened and validated these drug candidates for therapeutic potential.
• InteRNA now has multiple drug candidates for cancer in its development pipeline. In addition, the compounds may also be valuable for several other indications, including eye and neurodegenerative diseases.
• The main focus is currently on developing a targeted therapy for melanoma, the most deadly form of skin cancer. Current melanoma therapies only target one oncogenic pathway so that patients quickly develop resistance. As such, there is currently no long lasting effective therapy.
• A microRNA addresses multiple disease pathways at the same time, achieving biological and therapeutic outcomes that no other drug class can. This provides a potential breakthrough in drug effectiveness.
• Patent claims on the lead compound in the melanoma program have already been granted in the US and Australia and are pending in Europe and other countries.
• Treatment for melanoma continues to be a high unmet medical need due to the unsatisfactory efficacy and significant toxicities of the currently available drugs. It is one of the few types of cancer of which the frequency and death rates are still increasing .
•The market for melanoma therapeutics is estimated to grow with a 16.6% growth rate to US$2.6 billion in 2019 .
 US National Cancer Institute Bulletin, 2013;  Global Data, 2012
InteRNA has a unique product engine
InteRNA’s miRNA target identification and validation platform has delivered multiple novel drug candidates. With the in vivo Proof of Concept (PoC) of an intravenously administered formulated, chemically modified anti-angiogenic miRNA mimic in a mouse glioblastoma tumor model, the validated platform has shown to select bona fide drug candidates from the functional screens.
Preclinical product pipeline; Lead program on melanoma
Based on further validation in additional assays and mouse cancer model studies, the Company selected 3 programs (B-Raf pathway in melanoma, anti-angiogenesis, specific tumor cell death induction in head&neck cancer) for further preclinical and clinical development, of which targeting the validated oncogenic B-Raf pathway in melanoma is the lead program. InteRNA also established in vivo efficacy data in mouse xenograft models representing B-Raf mutant melanoma for its lead product candidate, miR-3157. As miR-3157 directly regulates the expression of proteins (e.g. Aurora B kinase) that play a role in the resistance to the B-Raf kinase inhibitor Zelboraf® (currently the first-line medical treatment option for oncogenic B-Raf mutant melanoma patients), this miRNA-based compound shows promise to qualify for treatment of melanoma patients that are intrinsic resistant to or became resistant upon treatment with Zelboraf. These patients have no treatment options left.
InteRNA has a worldwide exclusive license and full commercial rights to patent applications
covering a large collection of unique miRNA sequences. These cover different classes of claims ranging from product claims to method claims. Additional patent applications were filed in 2010-2013. These applications cover both method claims and use claims, protecting specific miRNA compounds for therapeutic use. Feedback from the US and EU patent offices indicate these claims are held novel, inventive and susceptible of industrial application.