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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.

HemoShear is a privately held biotechnology company that is changing the way drugs are discovered and developed, departing from traditional and often misleading scientific methods and animal studies in favor of translational tissue systems that accurately replicate human disease biology.  Its proprietary platform integrates best-in-class human disease systems, a comprehensive biorepository, interdisciplinary molecular and clinical disease expertise, and cutting-edge computational biology, together providing a unique and powerful lens to interpret biological mechanisms and human disease at a level not possible until now.  HemoShear’s drug discovery collaborations uncover new targets, elucidate previously unknown mechanisms, differentiate drug candidates, and predict efficacy and safety of drugs before entering the clinic.  HemoShear is collaborating to discover and develop new drugs with major pharmaceutical and biotechnology companies, five divisions of NIH and leading academic research institutions across several therapeutic areas, including oncology.

In November 2013, HemoShear announced a collaboration with NCI to initiate work to recreate the human tumor microenvironment in a physiologically relevant context that replicates human disease.  HemoShear has successfully completed the first stage of the NCI contract to create a tumor system that incorporates three essential cell-types, restores molecular signaling pathways and responds to three cancer treatments at human concentrations.  We know of no other systems that can assess drugs at human concentrations.   HemoShear is seeking $10 million from investors and/or pharmaceutical partners to engage in drug discovery collaborations and expand development of a wide range of tumor types and therapeutic approaches.

Company Background

DEKK-TEC, Inc. was founded in 1983 and specializes in the research and development of novel anticancer and hormonal technologies to improve the management of cancer and allied diseases. DEKK-TEC was presented the 2000 National Tibbett’s Award in recognition for its contributions to cancer research development in the SBIR program. The company is located in New Orleans, LA.

Technology Overview

4-Demethyl-4-cholesteryloxycarbonylpenclomedine (DM-CHOC-PEN) is a polychlorinated pyridine cholesteryloxycarbonate, which is being evaluated in Phase II clinical trials in patients with advanced lung, breast, melanoma and primary cancers involving the brain/CNS (IND 68,876).

DM-CHOC-PEN is an active and stable member of a larger series of carbonates and carbamates that has completed a Phase I clinical trial involving 26-patients with advanced cancer. Eleven of the latter patients had cancers involving the CNS, of which six (6) demonstrated objective responses/PFS – 1-breast, 2-melanoma, 1-sarcoma, 1-lung and 1-glioblastoma multiforme (GBM) cancers. Four of these patients had responses 1.3-3.5+ years in duration. These observations are supported by objective responses observed in pre-clinical studies with intracranial (IC) implanted human xenografts in mouse models – U251 and D54 GBM and MX-1 breast cancer [% long-term survival >52 days (%LTS) and complete response (%CR): +29/25 and +20/17, respectively, and in B-16 melanoma (%LTS/CR): 100/100%.

The drug is currently in a Phase II clinical trial – “Use of DM-CHOC-PEN as treatment for primary and metastatic cancers (lung, breast, melanoma) involving the CNS”. Objective responses are being verified in patients with lung cancer involving the brain. All trials support DEKK-TEC’s goal to include DM-CHOC-PEN in the treatment of CNS cancers. 

DM-CHOC-PEN’s MOA is via alkylation of DNA at N7- guanine and cellular senescence which means it could be added to O6 - guanine alkylators - BCNU, temozolamide (TMZ), etc. Thus, combination therapy is a possibility.

To date, the product has only demonstrated reversible hepatic toxicity in patients with prior liver disease/metastases. No hematologic, renal toxicities or neuro/psycho-performance abnormalities were noted in Phase I or in animal studies. Complete chemistry (incl. lipid profiles) and hematological lipid profiles are closely monitored. RECIST 1.1 was used to monitor responses.

Market Potential

Primary brain cancer (glioblastoma multiforme, GBM) is a dreaded cancer occurring in ~18,000 new patients annually in the US. In addition, approximately 20% of patients with all types of cancer will develop intracranial metastases. Approximately 150,000 patients will develop CNS metastastatic cancers from primary – lung, breast and melanoma in 2014. The latter are the most common primary cancers responsible for brain metastases and generally correlate with the distribution of the neoplasia in the population.

The survival for advanced GBM remains less than one year. For anaplastic astrocytoma and low-grade glioblastomas, it varies from 18 months to five years. Thus there are a sufficient number of patients available to treat with the drug. For metastatic CNS cancer, survival is 4-6 months. There are just under 200,000 patients annually in the US that are potential candidates for such a drug.

Competitive Advantage

In general, patients are living longer with cancer and have an increased incidence of developing CNS metastases - a 'safe haven' from systemic chemotherapy. Most drugs do not penetrate into the brain tissue. Thus a product to manage primary and metastatic CNS cancers has increasing demand. The prevalence of patients with these types of cancer presentations and the estimated markets make such a product worth developing.

DEKK-TEC’s product delivers across the BBB into CNS cancer sites, potentially reverses hepatic toxicity in patients with hepatic disease, and can be used with other drugs. 

Financial Overview

DEKK-TEC has raised almost $5 million in NIH grants, $2.2 million in licenses & milestone payments, & 1.8 million in Morgan personal funds, and half a million in LA State Tax Credits.

Intellectual Property

To date, DEKK-TEC’s commitment has been issued eight patents in the US; fifteen issued worldwide patents and four worldwide patents pending. The patents cover DEKK-TEC’s interest in penclomedine analogs, hormone delivery, phosphoramide mustards, phenylhydrazones and radiation devices.

Commercialization Strategy

In 2010, DEKK-TEC developed a c-GMP manufacturing facility (DEKK Pharmaceuticals, Inc.) to formulate and prepare unique and difficult delivery systems for Phase I drugs.

DEKK-TEC plans to raise $5 to $7 million to finish the Phase II trial and conduct the orphan drug trial. FDA funds are also a possible source of additional funding, though limited. The NCI Bridge Award is a possibility, but DEKK-TEC strives for a partner or financial associate.

Pipeline Products

DEKK-TEC is also developing 4-hydroperoxyifosfamide (HOOI) (pre-IND 381,783, US Pat 1,805,192) for use in the treatment of CNS cancers. The drug has a definite MOA (O6 –guanine alkylation); also active in pre-clinical studies against IC xenograft brain human tumors; Phase I trial studies are pending.

Management Team

• CEO and Medical Officer Lee Roy Morgan, MD, PhD is the founder who designed and synthesized DM-CHOC-PEN.
• Director of Research Andrew Rodgers, PhD developed the PK program.
• Director of Clinical Research Lisa Stokes, BSRN is a nurse oncologist and has worked with DEKK-TEC since 1986.
• Scientist Edmund Benes, BS works in the area of pharmaceutical and cell technology and has worked with DEKK-TEC since 1983.
• Clinical Pharmacologist Gerard Bastian, PhD collaborated with Dr. Rodgers to develop the PK program.
• Pharmacologist and biochemist David Adams, PhD is a consultant who has worked in collaboration with DEKK-TEC since 2006.

Galen Biotechnologies is an early-stage biotech company developing small molecule drugs to disrupt protein-protein interactions. Using genetically encoded-chemical fragment libraries we are rapidly advancing small molecules targeting Ras and Bcl-2 pathways in oncology. Our platform technology combines the strengths of encoded biologically relevant diversity with chemical evolution of pharmacophore structures to drive drug design with unparalleled efficiency against difficult protein-protein interaction targets.

Galen Biotechnologies was founded in 2013 and is located in the University of California, Santa Cruz QB3 incubator. The privately held company has raised $1M in nondilutive funding from research collaborations and SBIR grants and contracts (NIH-GMS, NCI, and DoD) in support of its platform. We anticipate a Series A fundraising in mid-2015 to accelerate our platform validation with the aim of progressing a lead molecule to full IND-enabling studies as well as exploring new targets in inflammation.