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Company Background

StemMed is a pre-clinical stage drug discovery, development, and testing company that is developing C188-9, a first-in-class, oral inhibitor of signal transducer and activator of transcription (Stat) 3, for treatment of ER-, PR-, HER2- (triple negative) breast cancer (TNBC). StemMed also provides state-of-the-art pre-clinical drug testing services using a panel of 44 breast cancer patient-derived xenograft (PDX) models developed from a diverse patient population, which includes 23 PDX models derived from patients with TNBC; 4 pancreatic cancer PDX models also are available.

Technology Overview 

Stat3 plays a central role in breast cancer stem cell development and has been validated as a drug target in these cells, as well as in the treatment of TNBC, for which no targeted therapy exists. StemMed used computer-based docking to screen 920,000 compounds and identified three small-molecule probes that targeted the phosphotyrosyl peptide-binding pocket within the Src homology 2 domain of Stat3. The most active probe, C188, reduced TNBC PDX tumor volumes and improved tumor-free survival of engrafted mice 4-fold when used in combination with standard chemotherapy. StemMed performed 2-D similarity screening, 3-D pharmacophore analysis, and 3-rounds of structure-activity relationship (SAR)-directed medicinal chemistry to identify C188-9, its lead, first-in-class drug for targeted treatment of TNBC.

Market Potential

The first patients that will be targeted to receive C188-9 will be those patients with locally invasive, metastatic, or treatment-refractory TNBC. An estimated 230,480 new cases of invasive breast cancer were diagnosed in the United States in 2011. Of these, ~40,000 will suffer breast cancer recurrence including 50% of those with TNBC. Unfortunately, standard therapies cannot eradicate the disease and these patients succumb to metastatic disease with a median survival of 2 years. The size of the U.S. market for C188-9 for metastatic/refractory TNBC on a yearly basis is ~20,000 cases for 7 cycles, or a total of 140,000 cycles. C188-9 will be administered orally once daily in the interval between cycles of first-line chemotherapy. Using a conservative figure of $2,000 per cycle, the income generated from 140,000 cycles of C188-9 would be $280 million.

Competitive Advantage

There are two orally bioavailable, small-molecule competitors of C188-9 in development, BP-1-102 and HJC0123. BP-1-102’s modest potency coupled with its low MTD will make it challenging to establish a safe and effective dose of BP-1-102 in humans. HJC0123’s mechanism of action has not been established and the mouse toxicity data presented in the original report was very limited. Other oral agents under development have mechanisms of action that are not understood, substantial toxicity, and did not demonstrate an efficacy signal in Phase I studies.

Financial Overview

StemMed’s drug testing revenue since 2010 totals $913,000. Revenue from STTR grant awards totals $417,000. StemMed also received $100,000 from PDX licensing and $213,000 from partner contributions.

StemMed needs $1 million in external funds for IND-enabling safety and PK studies and for IND filing.

Intellectual Property

StemMed has an exclusive license to ten patents issued to, or filed by, BCM and ownership of composition for C188-9. StemMed also has an exclusive license to use all 48 PDX models for drug testing services, as well as sublicensing to other companies for their in-house use.

Commercialization Strategy

StemMed’s objectives for the next two years are to continue developing its lead product candidate C188-9 by taking it through various studies. StemMed is also in discussions with Atara Biotherapeutics regarding licensing C188-9 for use in cachexia in chronic kidney disease.

Pipeline Products

StemMed has compelling pre-clinical results in cancer cell line xenograft models that also support the use of C188-9 either alone or with radiation therapy in patients with non-small cell lung cancer and head and neck squamous cell cancer. In addition, results from mouse pre-clinical models support its use in patients with cachexia secondary to chronic kidney disease or cancer, and in patients with idiopathic pulmonary fibrosis, scleroderma, inflammatory bowel disease, asthma, and immediate-type hypersensitivity reactions.

Management Team

• President, CEO, and director of drug discovery and development, David Tweardy, MD, discovered key molecular and cellular features of Stat3 during his 28-year research career supported by NIH and other extramural funding totaling over $16 million as PI.
• Vice president and director of drug testing, Michael Lewis, PhD, is an expert in breast cancer biology and developer of 48 breast and pancreatic cancer PDX models whose 15 year research career has been supported by NIH and other extramural funding totaling over $11 million as PI.
• Consultant and vice president of Texas BioAlliance, Jeffrey Larson, PhD, is a drug developer with extensive experience in pharmaceutical, biotechnology, and contract research industries, as well as a record of successful early- and late-stage regulatory meetings with the FDA.
• Consultant and Principal of Soller Regulatory & Research Services, R. William Soller, PhD, has extensive regulatory experience including serving as team leader and lead presenter for over 60 FDA or advisory committee meetings on drug development or postmarketing issues.