Market Overview

Three Biotech Companies With Disruptive UPenn Technologies Nearing Major Catalyst


The University of Pennsylvania (UPenn) is one of America’s most premiere Ivy League research universities. Its place as a home of innovation can be traced back to its founder Benjamin Franklin who advocated an educational program that focused as much on practical education for commerce and public service as on the classics and theology. UPenn is also home to the first medical school and business school in North America. UPenn is among the top five research centers in the United States and in the world. Their academic research spending tops any Ivy League school in America with an $814 million dollar budget. In only the last ten years there have been nine members from UPenn that won a Nobel Prize. While UPenn’s history of innovation and development is impressive its latest biotech discoveries are revolutionary. What follows are three biotech companies with disruptive technologies nearing major catalysts which may offer investors above average returns.


Novartis: Programmed Killer T-Cells


CTL-019 was invented by UPenn’s famous researcher named Dr. Carl June and is now being developed by Novartis (NVS). Dr. June has done what no other researcher has done before – programed cancer killing T cells to wipe out the cancer and to then leave a population of memory cells that can be reactivated whenever cancer presents itself.  CTL019 uses CAR technology to reprogram a patient's own T cells to "hunt" cancer cells that express specific proteins, called CD19. After they have been reprogrammed, the T cells (now called CTL019) are re-introduced into the patient's blood; they proliferate and bind to the targeted CD19+ cancer cells and destroy them.


Keep an eye on this treatment as it has just won the FDA’s coveted breakthrough designation in June and has been showing success in clinical trials. In a Phase 2 clinical trial 7 adult patients with CLL (chronic lymphocytic leukemia) experienced complete remission of their disease and 19 pediatric patients with ALL (acute lymphoblastic leukemia) experienced complete remissions. Staying up on CTL019 is wise as results are expected on their Phase 2 clinical trial. 


Difensin Mimetics - A Powerful Multi Use Superbug Killer


Cellceutix Corporation (CTIX), a company that is now starting to gain the industry’s attention, licensed from UPenn a revolutionary new class of antibiotics called Defensin Mimetics, small-molecules that imitate natural human immunity.  In a way, a similar idea is to have the body “hunt” the bacteria, but doing it with synthetics. These new classes of antibiotics were developed by chemists at UPenn using sophisticated computers and complex 3-D software.  Defensins are very novel in that they electrostatically lock onto bacteria and other microorganisms and effectively perforate their outer membranes. Whereas conventional antibiotics must penetrate bacterial cell walls, defensins act directly on the surface membrane. To escape the defensins, bacteria would have to evolve entirely new outer membrane characteristics. Defensin mimetics have also been found to have anti-inflammatory and immune-modulatory properties as well. Therefore they have the potential to be the breakthrough treatment addressing huge unmet markets such as Oral Mucositis and Diabetic Foot Ulcers. 

Brilacidin, their leading defensing mimetic has just completed its Phase 2b trial for acute bacterial skin and skin structure infections (ABSSSI). What sets Cellceutix apart from other antibiotic companies is that in this Phase 2b trial, Cellceutix selected Cubist blockbuster daptomycin (Cubicin) for the comparator arm whereas the other antibiotic companies used vancomycin, a generic with growing resistance issues. Brilacidin is also being administered as a single dose regimen and a three day regimen versus standard seven day daptomycin therapy. If Brilacidin shows to be non-inferior to daptomycin in any dosing scheme and demonstrates to be safe and well tolerated, it will be a milestone moment in the world of antibiotics. Another gem that originated at UPenn.


The Father of DNA Vaccines


Inovio Pharmaceuticals, Inc. (INO) is a biopharmaceutical company that holds promising candidates in its pipeline for the treatment of cancer and infectious diseases. It is also carrying out research in proprietary technologies to develop new methods of vaccine delivery, design and formulation. 


A lot of the technology for Inovio came from the work of Dr. David Weiner a world class researcher at UPenn. Dr. Weiner, also known as the father of DNA vaccines, discovered how to genetically engineer plasmids to produce any protein that is normally produced by our DNA. These plasmids can be engineered to make antigens which alert the immune system to the presence of a disease and activate an army of specific T-cells to attack the disease.


Inovio's leading vaccine VGX-3100 is used to treat cervical intraepithelial neoplasia (CIN), a disease leading to the abnormal growth of squamous cells on the surface of the cervix. Phase 2 trial results which have just been released in late July showed that treatment with VGX-3100, Inovio's HPV16/18-specific immunotherapy, resulted in histopathological regression of CIN2/3 to CIN1 or no disease, meeting the study's primary endpoint.  In addition, the trial demonstrated clearance of HPV in conjunction with regression of cervical lesions. Robust T-cell activity was detected in subjects who received VGX-3100 compared to those who received the placebo. It is important to keep an eye on this company as the next catalyst may be positive communication with the FDA to advance VGX-3100 in clinical trials.

The preceding article is from one of our external contributors. It does not represent the opinion of Benzinga and has not been edited.


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