About TargaGenix

TargaGenix, Inc. (“the Company”) was founded in 2013 to develop novel compounds that are effective on both drug-resistant tumors and cancer stem cells.  In numerous preclinical models of cancer, including colon, pancreatic, prostate, breast and lung cancers the company’s lead product SBT-1214 has shown to completely eliminate the tumor.  Additional studies have shown that SBT-1214 is able to effectively down-regulate “stemness” gene expression and kill cancer stem cells.  Additional chemical modifications and a novel fatty-acid based nanoemulsion formulation result in a drug that has improved pharmacokinetics and pharmacodynamics properties as well as reduced toxicity.

 

The Company was founded by James E. Egan Ph.D MBA and the science is built on the pioneering discoveries of Dr. Iwao Ojima in chemistry and Dr. Mansoor Amiji in pharmacology.  TargaGenix’s senior management team and advisors have extensive experience in the clinical development of oncology drugs.  Dr. Ojima’s 40+ years of research laid the foundation for the TargaGenix technology which seeks to overcome both intrinsic and acquired mechanisms of drug-resistance in cancer as well as target cancer stem cells.  These cancer stem cells are highly drug resistant and are responsible for re-populating the tumor and metastasis.  It is only by targeting both the bulk tumor and the cancer stem cell population that a drug will be an effective long-term cancer therapy.

Near-term catalyst opportunity

 

The Company recently received $2.3 mm from the National Cancer Institute for the development of novel cancer stem cell therapeutics and the Company will use this money to fund IND-enabling toxicology studies. The Company seeks to raise $8 mm in two tranches to complete the IND filing and conduct a Phase 1/2 clinical study.  Significant value will be created with the successful completion of the Phase 1/2 study and the Company would seek to license the technology to a strategic partner or raise additional capital to fund a proof-of-concept Phase 2 study.  As evidenced by Celgene’s purchase of Abraxis for $2.9b there is significant industry interest in less toxic and more effective chemotherapeutic agents.  This agent also has the potential to be combined with immune-oncology agents as a tumor-debulking agent, reducing immune suppression and stimulation of natural killer cell, dendritic cell and cytotoxic T cell activation.

 

How TargaGenix’s Lead Compound Addresses the Current Compound Addresses the Current
Challenges in Cancer Treatment


Genomic Instability

  • Effective in tumors resistant to current treatments

  • Effective in tumors with multi-drug resistant (“MDR”) mechanisms such as efflux pumps

  • Effectively kills the cancer stem cell sub-population


Selectivity based on cell division or other cellular functions

  • Reduced toxicity due to tumor targeting


Response rate

  • High response rate

  • Mice cured in multiple MDR cell lines (pancreatic, prostate, colon, breast and lung cancer)

  • Can be combined with immuno-oncology for durable responses (de-bulk tumor, reduce immune suppression, drive T cells into tumor, generation of effector memory T cell population)


Formulation of cancer drugs

  • Novel nanoemulsion formulation improves pharmacology, increased bioavailability and retention time

  • Passive targeting to tumor

Technology

 

TargaGenix, Inc. (‘the Company’) is developing a novel nanoemulsion formulation of a novel chemotherapy for the treatment of cancer.  The product is a combination of a nanoparticle formulation encapsulating the novel 3rd generation taxane DHA-SBT-1214.  The discovery of DHA-SBT-1214 was the result of a 15+ year effort to discover new molecules that were effective against chemotherapy resistant tumors.  DHA-SBT-1214 has been shown in preclinical models that it can treat and cure animals with tumor types that are resistant to current chemotherapy treatments.  DHA-SBT-1214 has also been shown to effectively kill cancer stem cells and this property may represent the mechanism of the long-term efficacy seen with DHA-SBT-1214 treatment.  DHA-SBT-1214 acts as a pro-drug and is inactive in plasma and activated once it enters the tumor.   In multiple paclitaxel-resistant tumor models, DHA-SBT-1214 treatment resulted in sustained complete responses whereas paclitaxel and a fatty acid paclitaxel conjugate were ineffective. 

 

The second technology is the nanoparticle formulation of the DHA-SBT-1214 (“NE-DHA-SBT-1214”).  This formulation is designed to improve the delivery, reduce toxicity and potentially improve the efficacy of the drug. Nanoparticle-based delivery systems can take advantage of the enhanced permeability and retention (EPR) effect for passive tumor targeting. NE-DHA-SBT-1214 is positioned for development as a comprehensive targeted therapeutic modality to both de-bulk the tumor and eradicate CSC populations in multidrug resistant cancers.

 

Intellectual Property

 

The Company, has created a significant and valuable intellectual property estate.  The Company’s lead product DHA-SBT-1214 is covered by issued and pending United States and foreign patents and patent applications. In addition to patent protection DHA-SBT-1214 may be eligible for extended market exclusivity through statutory provisions that includes patent term restoration, data exclusivity and/or orphan drug exclusivity.  The product is to be developed in an orphan indication orphan drug designation, which would provide seven years exclusivity in addition to other benefits.

 

Conclusion

 

The three key factors to successful drug development are demonstrating a clinical benefit for a patented drug candidate, a lack of unacceptable drug related toxicity and having the necessary capital to fund the development program. The progress that TargaGenix has made to this point significantly increases the chances of success.  The drugs remarkable pre-clinical efficacy, the enhanced pharmacological profile and the management team’s collective experience significantly reduces the development risk.  As TargaGenix moves its lead product into the clinic, there will be a significant opportunity to increase the Company’s valuation and generate attractive returns for investors.

 

Financing Contact
James Egan, PhD MBA

President & CEO

25 Health Sciences Dr.

Suite 208

Tel: (516) 449-8148

jegan@targagenix.com

Current Challenges in Cancer Treatment

Genomic Instability

  • Rapid resistance to front-line treatment

  • Rapid repopulation of tumor and metastasis due to cancer stem cell sub-population


Selectivity based on cell division or other cellular functions

  • Toxicity


Response rate

  • Molecularly targeted – High response rate, transient responses

  • Immuno-oncology – Durable responses, low response rate (8-20%)


Formulation of cancer drugs

  • Poor pharmacology

  • Low retention times, insufficient quantities get to tumor