Tumorend, LLC was founded in 2009 as a drug development, manufacturing and marketing company for a variety of products based upon the CM101 technology licensed from Vanderbilt University. The Company is located in the Louisiana Emerging Technology Center, on the LSU campus, in Baton Rouge, LA. The Company’s first product, having completed a successful Phase I clinical trial, is ready to enter Phase II clinical trials for the treatment of patients with solid organ cancer. The Company’s first veterinary product had a successful canine safety trial and is ready to enter field trials for dogs with solid organ cancer.
Target Markets/Business Opportunities
CM101, a tumor vasculature disruptor.
Tumorend’s initial product, CM101, targets patients with solid organ cancer, such as prostatic, liver, and refractory (multiple organs). We know that much progress has been made over the past 10 or so years in the diagnosis and treatment of solid organ cancer patients, but there remains an unmet medical need. This need includes improved efficacy and safety. Based on animal work and a published Phase I clinical trial (1) Tumorend believes that CM101 holds great promise in helping to meet that need in patients with solid organ cancer.
Market Size and Growth
In 2008, antineoplastic mAbs delivered sales of $13.6bn with a market share of 28.6% of total cancer pharmaceutical sales. The key antineoplastic mAbs which contributed to the market share of this class in 2008 were MabThera (11.0%), Avastin (10.3%), Herceptin (9.7%) and Erbitux (3.4%). Forecast size of the total antineoplastics market is estimated at $65.2bn in 2014. Based on the results from the Phase I study, Tumorend believes that CM101 will show dramatic improvement in efficacy and safety and will capture a significant share of the market.
During the investigation of newborn lung neovasculature inflammatory deaths from Group B streptococcus, it was discovered that the inflammation and coincident disruption of the neovasculature was a result of CM101, a complex carbohydrate with a 270k molecular weight. CM101 was tested in animals injected with various cancer cell lines and found to have tumor vascular disruption potential. An IND was approved and a Phase I study conducted (1). The mechanism of action has been shown to be the complement activating binding of CM101 to a specific neovasculature target lectin protein, HP59 (2). The CM101/HP59 complex on the inside of the tumor neovascular membrane recruits neutrophils to attack the tumor neovasculature and surrounding tumor resulting in an inflammatory reaction, which leads to tumor apoptosis (triggered cell death).
CM101 appears to have multiple advantages.
One advantage is its use of the body’s own defenses for initiation of a natural immunological response specifically targeting the tumor neovasculature and surrounding tumor area.
A second advantage is the low toxicity of the drug. Due to its mechanism of action CM101 should be safe in humans without cancer. As an anti-cancer drug, it is administered in dosages 600 times lower than the levels found in the blood of neonates who survive a GBS infection.
A third advantage is that CM101has the potential to reduce or eliminate the need for toxic chemotherapy in the treatment of solid organ cancer.
Clinical Results (Completed, Successful Phase I Safety Trial)
In a Phase I study cited below (1), 15 patients were treated with three intravenous infusions of CM101. Tumor responses in Phase I trials are rare (1-2%). In this trial 33% of the patients showed definite improvement including a classical Kaposi’s sarcoma with multiple lower extremity lesions, a metastatic adenocarcinoma of the small bowel and a hepatocellular carcinoma. All 15 of these solid organ cancer patients were refractory with very limited life expectancy. The results from this study demonstrated very positive effects in multiple tumor types.
Tumorend’s lead product is being advanced into formal clinical trials to treat patients with multiple solid organ tumors as well as refractory patients. The clinical trial plan is to conduct four Phase I/IIa studies, one each in refractory, hepatic, pancreatic and prostatic cancer patients. Dose levels and safety have already been established in a published Phase I Clinical safety trial (1).
1). DeVore RF, Hellerqvist CG, Wakefield GB, Wamil BD, Thurman GB, Minton PA, Sundell HW, Yan H-P, Carter CE, Wang Y-F, York GE, Zhang M-H, Johnson DH. ,1997, A phase I study of the antineovascularization drug CM101. J Clin Canc Res, 3, 365-372.
2). Fu C, Bardhan S, Cetateanu ND, Wamil BD, WangY, Yan H-P, Carter CE, Shi E, Venkov C, Yakes FM,
Page DL, Lloyd RS, Hellerqvist CG. Identification of a novel membrane protein HP59 with therapeutic potential
as a target of tumor angiogenesis. Clin. Cancer Research 7:4182-4194 (2001).
TumorEnd has conducted an USDA recommended canine safety trial on CM101 at the Louisiana State University College of Veterinary Medicine directed by Professor Rhett Stout, DVM, PhD. The safety trial showed no side effects in healthy dogs. This work opens the door to conducting field trials on tumor-bearing dogs for which we are working toward approval, and seeking funding.
TumorEnd is currently conducting research on the Structure-Activity-Relationship of CM101 supported by an NCI SBIR of $150,000 awarded August, 2012, along with $60,000 in state matching funds.
The patent portfolio consists of thirteen patents including patent 6,803,448, “GBS toxin receptor”, covering the HP59 receptor, a method of purifying the receptor, using the receptor to determine if metastasis are present, identifying other potential drug candidates, creating an inhibitor of neonatal onset disease by inhibiting the binding of GBS to the HP59 receptor, a vaccine for inhibiting mammalian angiogenesis and all other drugs that bind to HP59. This patent expires in 2024. The new health law protects biologicals for 12 years post-FDA approval.
Roger Laine,PhD, the president, has forty years of academic and business experience. Author of more than 150 publications and 25 patents, he is a world expert on carbohydrate chemistry and has direct experience with CM101, a large molecule carbohydrate. He was the third founding member of Glycomed, Inc., as Chief Scientist, he hired 25 PhD's, 10 Technical Scientists, in-licensed key technology and he helped take the company from founding through IPO and subsequent acquisition. Curriculum vitae: https://www.dropbox.com/s/18zr4ioqrc6971t/RAL%20CV%20June2012.pdf
Mr. Armand Alciatore, MBA, the CFO, has twenty years of experience in management consulting for the venture capital industry. He has founded or turned around 8 companies including medical devices, software, biotechnology, publishing, in the USA, Europe. Curriculum vitae: https://www.dropbox.com/s/tj737ew6pa7lak0/KMClinicalAlciatoreBiosketch.pdf
Dr. Khushi Matta, Chief Scientist, has 33 years of experience in chemistry of cancer-related carbohydrates at Roswell Park Memorial Cancer Center in Buffalo New York. Author of more than 250 publications and 8 patents, he is an internationally recognized expert in organic and chemo-enzymatic synthesis of oligosaccharide substrates, tumor markers and molecular decoys. Curriculum vitae: https://www.dropbox.com/s/vablbf6jeg6rgod/MattaCV2012.pdf
Dr. Betty Zhu, Chief Biochemist has 30 years experience in protein chemistry, carbohydrate chemistry and biochemistry, carbohydrate characterization and structure determination. She has published extensively in the areas of polylactosamine structure and function, glycosylation in Archae, and biochemical effectors of insects.
Dr. Carl Hellerqvist, Consultant, is the discoverer of the CM101 and HP 59 therapeutic concepts. He holds a permanent Docent Position at the Karolinska Institute of the University of Stockholm. He is Professor of Biochemistry Emeritus at Vanderbilt University in Nashville. He is a world recognized expert on the structure determination of carbohydrates, and on the vascular effects of Group B Streptococcal toxins. Curriculum vitae: https://www.dropbox.com/s/5ztytrjtbjk8ftx/biosketchHellerqvist12.pdf
Dr. John Sundsmo, consultant, regulatory and patent agent, was a complement system expert staff Scientist at Scripps, La Jolla, has 15 years of experience in startup biopharmaceuticals, and 15 years experience as a registered patent agent. He was recently VP Research for Stellar Biotechnologies, a KLH bioproduction company. Curriculum vitae: https://www.dropbox.com/s/g4thmrp5wgu7n8o/SundsmoCV_02_16_12.pdf
Dr. James Shook, consultant, Development and Regulatory Consultant with considerable experience in preclinical, manufacturing, preparation of INDs, clinical trials, NDAs and direct interaction with the FDA. He has over 35 years of experience in all aspects of the pharmaceutical industry and more than 25 NDA applications to the FDA.
Tumorend believes it is well positioned to compete in the human and canine oncology markets. The initial product targets a large market with unmet medical needs. Completion of the Phase I/IIa clinical trials with safety and some efficacy should prove attractive to potential pharmaceutical companies and venture companies for Series B financing at a significant step-up in valuation.
Funding and Exit Strategy
TumorEnd was awarded an NCI Phase I SBIR, August, 2012, $150,000 plus $60,000 State Matching Funds. Dr. Matta has a subcontract NIH research grant to TumorEnd, from the NCI through the University of Buffalo of $56500/year for 2 years.
The Company is seeking a stage one investment of 10M to fund the four Phase I/IIa clinical trials, the canine field trials and to bring the canine product to market. With a successful Phase I/II trial, the Company plans to file for an IPO or license to a pharmaceutical company to fund Phase III trials.
For more information please contact:
Armand Alciatore, CFO, (504) 390-1700.