Paul Rhodes, Ph.D.
Company Background
Metabolomx is commercializing a non-invasive, rapid, and inexpensive breath test for cancer with the potential to revolutionize cancer diagnosis.
Technology Overview
Metabolomx has developed technology enabling the identification of lung cancer from its metabolomic fingerprint in exhaled breath, currently in a second round of efficacy trials at Cleveland Clinic and other distinguished clinical centers. At the heart of the system is a high dimensional array of diversely reactive chemical indicators that change color upon interaction with volatile species or mixtures.
Using the first generation of the Metabolomx sensor a recent Cleveland Clinic study (Mazzone et al., 2012, Journal of Thoracic Oncology) reported 85 percent specificity and sensitivity for lung cancer detection, comparable to a CT scan, the present gold standard. In the first quarter of 2012, the Cleveland Clinic and National Jewish Health Center in Denver (led by Dr. Jim Jett, Editor-in-Chief of JTO) began testing a Metabolomx sensor over 100 times more sensitive than the version used in the Journal study. The Mayo Clinic, led by Dr. David Midthun, is scheduled to begin testing in the second quarter of this year. Metabolomx’s technology will be in the clinic at the country’s three top-ranked lung care centers (U.S. News and World Report), reflecting the promise of this new paradigm for lung cancer assessment.
Market Potential
First indication: A companion diagnostic to CT scan
Metabolomx’s first FDA indication will be as a diagnostic adjunct to an inconclusive CT scan. Results from the 53,000 patient National Lung Screening Trial (NSLT) indicate that a CT scan screening of high-risk patients extends life expectancy over 20 percent (NEJM, June 2011). The study has already prompted a powerful shift toward widespread use of CT, with Wellpoint recently announcing it will cover CT screening of the enormous at-risk population (more than 55 years of age, more than 30 pack years) identified in the study. However, CT generates a large number of false positives (the NLST found 27% of the high-risk group had a positive CT). Millions of people who have a positive CT are faced with the difficult decision of whether to monitor with follow-up CT or submit to an invasive and expensive biopsy, magnifying the need for a diagnostic adjunct to inconclusive CT. The estimated market size of this first indication is 10 million units per year in the U.S., with revenue of $1 billion.
Pre-screen of high-risk population to triage who should receive CT
The financial costs of CT, risks involved with radiation exposure, and the enormous size of the at-risk population defined in the NLST calls for a non-invasive, inexpensive initial test to better triage who should be screened by CT. Metabolomx expects to gather data on more than 1,000 patients to confirm that the breath test is a candidate pre-CT screen of the high-risk population. The estimated market size to pre-screen the high-risk population is 25 million units per year in U.S., with revenue of $2 billion.
Monitor efficacy of chemotherapy
Metabolomx is gathering data to confirm that the breath signature declines when chemotherapy is effective in curtailing tumor growth. The estimated potential market to monitor treatment is 15 million units per year in the U.S., with revenue of $1.5 billion.
Correlation between metabolomic breath fingerprint and effective treatment
Metabolomx is compiling an unmatched database allowing the post-hoc assessment of whether there is a predictive correlation between the metabolomic breath fingerprint and the efficacy of a particular treatment. With each treatment often tailored to intervene in a particular metabolic pathway, Metabolomx predicts that the metabolomic fingerprint in exhaled breath will allow individualized selection of treatment.
Competitive Advantage
The technology is a five minute, inexpensive (less than $100), non-invasive test for lung cancer and other cancers, based on the VOC profile present in the bloodstream and picked up in exhaled breath. Each cancer has its own “smell,” and canines have been documented more than a 95 percent accurate in detection across stages, signaling the extraordinary promise of this new paradigm.
Financial Overview
Metabolomx has received several government development contracts, including a NCI Phase I/Phase II Fastrack award of $1.135 million. The company seeks a $5 million Series A round to reach clinical quantification of the level of accuracy of the second generation lung cancer detection system (building on the 85 percent accuracy of the first generation system) and submission to the FDA. Financial information on Metabolomx is available to interested parties under NDA.
Intellectual Property
The company holds exclusive, worldwide rights to an estate of fundamental enabling technology, including both the sensor itself and application IP. Additional private information is available to interested parties under NDA.
Commercialization Strategy
The Metabolomx breath analysis instrument will be sold or leased to the same hospitals and testing centers that use CT. The bulk of the company revenue is from the disposable sensor arrays and the balance from equipment and support. The company’s first FDA indication will be as a diagnostic adjunct to indeterminate CT scan.
Pipeline Products
The first product is a breath test for lung cancer, but tests for other cancers will follow.
Management Team
Paul Rhodes, Ph.D., CEO leads a diverse group of technology companies, which have received $13 million in DARPA contracts to develop next-generation sensory systems.
Ray Martino, COO, who, during a 20-year career at Symbol Technologies was General Manager of its mobile business ($500 million in division sales) and then CTO of Symbol prior to its acquisition by Motorola for $3.9 billion in 2007.
Sung Lim, Ph.D., Chief Scientist, is a co-inventor of the company’s proprietary nanoporous pigment array optical sensing technology, along with Metabolomx’ co-founder, University of Illinois Professor Ken Suslick.
A Clinical Advisory Board has been formed, and includes deep practical expertise in FDA approval processes.
Metabolomx
CEO
Gary Robinson
Company Background
Celek Pharmaceuticals is addressing the need for new medicines to help patients suffering from cancers that are poorly served by current therapies. The company’s strategy is to enhance the value of in-licensed drug candidates by advancing them through proof-of-concept clinical trials. Formed as a Delaware LLC in 2009, Celek’s two founders, Graham Allaway, Ph.D., and Gary Robinson, Ph.D., are currently the sole employees.
Technology Overview
Celek’s lead product, CEL-031, is a clinical-stage targeted anticancer drug that selectively induces apoptosis in tumor cells by inhibiting cyclic GMP phosphodiesterases, which are overexpressed in human tumors. Currently in preclinical development for non-muscle invasive bladder cancer (NMIBC), CEL-031’s mechanism of action involves the degradation of β-catenin, a cell signaling protein that plays a key role in bladder cancer tumorigenesis. In clinical studies against advanced cancers, orally-administered CEL-031 showed evidence of efficacy and a good safety profile. CEL-031 should have greater clinical efficacy against NMIBC, where it will be administered intravesically (i.e., instilled transurethrally), the standard drug delivery route for this indication.
Market Potential
Bladder cancer is the fifth most common cancer in the U.S., with 70,000 new cases annually and 600,000 individuals living with the disease. Worldwide, there are approximately 400,000 new cases annually and the incidence is rising.
About 70 percent of new bladder cancer diagnoses are made at the non-muscle invasive stage. Current NMIBC treatments involve transurethral resection (TUR), often followed by intravesical chemotherapy using non-specific cytotoxic drugs such as mitomycin C, or immunotherapy with Bacillus Calmette Guerin (BCG). These treatments often fail, with five-year recurrence and progression rates of 50-70 percent and 20-30 percent, respectively. Current drugs also cause adverse side-effects and are hazardous to health care workers.
Since NMIBC is a chronic disease requiring lifelong monitoring and treatment, the lifetime cost per patient of treating bladder cancer is the highest of all cancers.
Despite the pressing need, few new drugs are in development for NMIBC. Celek is developing CEL-031 for two NMIBC indications: (i) perioperative administration following TUR, and (ii) BCG-refractory NMIBC. CEL-031’s estimated peak annual sales in these indications range from $510 million to $660 million.
Competitive Advantage
As the first targeted drug for NMIBC, CEL-031 represents a potential breakthrough in the treatment of patients with this disease. It should be possible to deliver CEL-031 safely at higher, more effective doses than current cytotoxic chemotherapies, resulting in dramatic reductions in rates of recurrence and progression. CEL-031’s favorable safety profile should also result in a substantial increase in the number of patients treated with CEL-031 compared to current drugs.
Financial Overview
Celek has raised more than $700,000 in funding, including investments by the principals and federal and state grants. The NCI awarded Celek a $176,000 Phase I SBIR contract supporting preclinical studies on CEL-031 for NMIBC. Celek is currently seeking to raise $3 million to support preclinical studies of CEL-031 in bladder cancer and acute myeloid leukemia (AML), and the initiation of a Phase I/II clinical trial in non-muscle invasive bladder cancer.
Intellectual Property
Celek obtained exclusive rights to CEL-031 from OSI Pharmaceuticals. CEL-031 as a composition of matter and methods of treating cancer with CEL-031 are covered by four issued U.S. patents (plus foreign equivalents). Additional patents cover analogs, methods of identifying anticancer compounds and combination therapies.
Commercialization Strategy
Celek plans to complete a proof-of-concept clinical trial of CEL-031 in NMIBC patients, then partner for later stage development/commercialization. Recent partnering deals in this therapeutic area have had attractive financial terms. Celek has already met with potential partners who indicated interest in the product.
Pipeline Products
Celek is also developing CEL-031 to treat advanced cancers using novel formulation and delivery technologies to increase concentrations of the drug in the body, thereby maximizing efficacy. The company is focusing on: (i) advanced bladder cancer, and (ii) acute myeloid leukemia (AML). A recently published independent study reported that CEL-031 has potent activity against tumor cells from AML patients, including those resistant to current drugs, and recommended clinical testing of CEL-031 against AML. CEL-031 would be eligible for Orphan Drug status in this indication.
Management Team
Graham Allaway, Ph.D., President and C.E.O, has spent 22 years in the biotechnology industry. As founding CEO of Panacos Pharmaceuticals, he played a key role in building that company from a private venture-backed start-up to a public company, while raising more than $125 million in private and public equity financing. Dr. Allaway also led Panacos’ drug discovery and development programs. Prior to Panacos, Dr. Allaway was CEO of Manchester Biotech and he previously led therapeutic R&D at Progenics Pharmaceuticals.
Gary Robinson, Ph.D., Chief Business Officer, has 20 years of experience in research, development, and commercialization of technologies and products in the physical and life sciences. Most recently, he was Senior Director of Business Development at Panacos Pharmaceuticals, where he led partnering, contracting, intellectual property and pre-launch marketing activities. Prior to Panacos, Dr. Robinson held business and corporate development positions at IGEN.
