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Open enrollment in the Phase II study began in November 2012, following successful completion of an earlier Phase I safety lead-in study. An independent Safety Monitoring Committee determined that no dose-limiting toxicities had occurred in Phase I, that TSC had been well tolerated in all patients, and that open enrollment in Phase II should be authorized.

“The speed of accrual for our GBM trial has been remarkable,” said David R. Jones, MD, Chief Medical Officer of Diffusion Pharmaceuticals. “This speaks to the huge unmet medical need in GBM and the desire of clinicians to identify much-needed new therapies and move them forward as rapidly as possible. We are also quite pleased at the safety profile of TSC and believe this played a significant role in the rapid rate of patient enrollment.”

Dr. James Larner and Dr. Jason Sheehan of the University of Virginia Medical Center are Study Co-Chairs for this trial.  A summary of the study is available online at http://clinicaltrials.gov/ct2/show/NCT01465347?term=tsc&rank=5.

Separately, the Company announced that it is initiating a new financing round to fund further clinical development.

“About $6 million dollars of new convertible debt will be issued to accredited investors,” said Diffusion CEO David G. Kalergis. “We expect most of it to be placed with our existing shareholders, although we may add several additional accredited investors from our network.”

About TSC

Trans sodium crocetinate (TSC) is a proprietary, first-in-class oxygen diffusion enhancing compound currently formulated for intravenous administration.  Based on a novel mechanism of action, TSC safely re-oxygenates hypoxic tissue without causing hyper-oxygenation of normal tissue. Published studies demonstrate a tripling of survival and superior tumor control when TSC is combined with radiation and chemotherapy in animal models of primary brain cancer.  The US Food and Drug Administration has granted Orphan Designation for TSC in the treatment of both GBM and brain metastases. The potential patient population for TSC in these two brain cancers is about 183,000 patients annually.

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“This Orphan Drug designation for TSC is an important step for Diffusion Pharmaceuticals,” said David Kalergis, the company’s Chief Executive Officer. “There is currently no approved drug for the treatment of this condition, and TSC, if successful, would provide a much-needed treatment option. When combined with TSC’s progress in the on-going Phase II trial in GBM patients, we are hopeful that this new Orphan Drug designation will help us to initiate a trial of TSC in brain metastases as soon as possible.”

Brain metastases, or cancer cells which have migrated to the brain from other locations in the body, affect about 170,000 patients in the US every year, and better treatment is considered a pressing unmet medical need.  Currently, treatment may include surgical removal of the tumor bulk and limited radiation therapy. There are no drugs approved by the FDA for the treatment of brain metastases, although doctors may sometimes prescribe certain chemotherapy drugs “off label.” Factors within the micro-environment of metastatic tumor tissue, especially oxygen deprivation (hypoxia), are thought to play a role in treatment resistance and tumor recurrence. TSC is believed to enhance the diffusion of oxygen into this hypoxic micro-environment, making treatment-resistant tumor cells up to three times more susceptible to standard radiation treatment.

About Orphan Drug Designation

The United States Orphan Drug Act of 1983 was created to promote and support the development of new drug therapies for diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation provides a sponsor seven years of market exclusivity for the designated therapeutic indication in the United States, from the point at which the therapy is granted marketing approval thereby offering competitive protection. Orphan Drug Designation also provides access to regulatory support from the FDA, potential FDA fee reductions and tax credits related to development expenses.

About TSC

Trans sodium crocetinate (TSC) is a proprietary, first-in-class small molecule belonging to the novel “oxygen diffusion-enhancing compound” family of drugs. Its mechanism of action is unlike that of any previous drug, combining potent efficacy-enhancing properties with an excellent human safety profile. Many cancers, including GBM and brain metastases, contain cells that are resistant to treatment because of diminished oxygen levels (hypoxia) within the tumor. TSC causes more oxygen to diffuse into hypoxic tumor tissue without affecting normal tissue, thereby enhancing the tumor killing power of conventional treatments. Published animal studies in GBM show that TSC controls tumor growth and triples survival without observed side-effects when used in conjunction with standard-of-care radiation.

About Diffusion Pharmaceuticals

Diffusion Pharmaceuticals is a clinical-stage company developing first-in-class drugs which target the numerous unmet medical needs characterized by hypoxia at the cellular level. These proprietary small molecules work by a novel mechanism of action that enhances the diffusion of oxygen selectively to hypoxic tissue. Potential breakthrough clinical applications include oncology, cardiovascular diseases, stroke and respiratory disorders. Diffusion’s lead program is the use of TSC in the treatment of solid cancerous tumors. A Phase II clinical study of TSC dosed concurrently with radiation in newly diagnosed GBM is currently enrolling patients at twenty cancer centers throughout the US.  A Phase II clinical trial of TSC in the treatment of brain metastases is being planned. Diffusion Pharmaceuticals, which is privately held, is located in Charlottesville, Virginia.

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The Phase II trial will explore TSC’s effects on tumor regrowth, patient quality-of-life and overall survival in newly diagnosed GBM patients when dosed in conjunction with conventional radiation therapy. The study will enroll a total of fifty patients at twenty cancer institutions throughout the U.S., with a target date of complete enrollment by early 2013. Patients in the Phase I brain cancer study were treated at six cancer institutions around the country. Fourteen additional centers will be open for the Phase II study, which has already begun enrolling patients. Further information about the study may be viewed online at Glioblastoma Clinical Trial Phase II.

“This confirmation of TSC’s safety and dosing regimen have prompted the immediate expansion of treatment to a larger number of newly diagnosed GBM patients,” said David G. Kalergis, MBA/JD, the CEO of Diffusion Pharmaceuticals. “The timely opening of Phase II means that statistically-powered efficacy data should be available on schedule later in 2013.”

The Phase II study builds on several previous Phase I/II studies establishing TSC’s human safety profile, pharmacokinetics and dosing regimen in eighty human subjects, including normal healthy volunteers, peripheral artery disease, and GBM patients. The current study will focus on the efficacy and safety of TSC as an addition to the standard-of-care in newly diagnosed GBM patients. Study endpoints include TSC’s effects on tumor regrowth as determined by MRI, the patient’s quality-of-life during and after their treatment, and their overall survival at one and two years after treatment.

TSC was granted an FDA Orphan Drug designation for its use in GBM in 2011, and an Orphan Drug designation for its use in brain metastases is pending. Orphan Drug designation confers substantial benefits, including enhanced patent protection and marketing rights, waiver of certain FDA filing fees, and favorable tax treatment.

Dr. James Larner and Dr. Jason Sheehan of the University of Virginia Health Sciences Center are Study Co-Chairs for this multi-institutional trial. Other cancer institutions participating in the trial may be found at Glioblastoma Trial Locations for Phase II.

About TSC

Trans sodium crocetinate (TSC) is a proprietary, first-in-class, small molecule belonging to the novel “oxygen diffusion enhancing compound” family of drugs. Its mechanism of action is unlike that of any previous drug, combining potent efficacy-enhancing properties with a very well tolerated human safety profile. Many cancers, including GBM and brain metastases, contain cells that are resistant to treatment because of diminished oxygen levels (hypoxia) within the tumor. TSC causes more oxygen to diffuse into hypoxic tumor tissue without affecting normal tissue, thereby enhancing the tumor-killing power of conventional treatments. Published animal studies in GBM show that TSC controls tumor growth and triples survival without observed side effects, when used in conjunction with standard-of-care radiation and approved chemotherapy.

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“Enrollment of patients in our clinical trial of TSC is actually ahead of schedule,” said David G. Kalergis, Chief Executive Officer.  “This financing will help ensure the trial’s continued timely execution, with full enrollment expected in the first quarter of 2013.”

Dr. James Larner and Dr. Jason Sheehan of the University of Virginia Medical Center are Study Co-Chairs for this trial. A listing of all trial sites, including contact information, may be viewed online at http://clinicaltrials.gov/ct2/show/NCT01465347?term=tsc&rank=4

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“These new patents are important additions to our intellectual property portfolio, providing strong protection through at least 2027,” said David G. Kalergis, Diffusion’s CEO.

Currently the Company has 7 issued U.S. patents, 10 issued foreign patents and more than 70 pending patent applications filed in all major international markets.

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Patients enrolled in the first cohort were treated at a dose level of 0.25 mg/kg of TSC with three doses a week for three weeks.  TSC was dosed by iv injection in conjunction with the existing standard of care (radiation and chemotherapy) for newly diagnosed GBM.  An independent Safety Monitoring Committee met on July 17, 2012, and authorized enrollment in Cohort 2 after determining that no dose-limiting toxicities had occurred and that TSC was well tolerated in these patients.

“The finding by the independent Safety Monitoring Committee confirms the excellent clinical safety profile seen previously in TSC,” said David G. Kalergis, MBA/JD, the CEO of Diffusion Pharmaceuticals. “Enrollment in the second cohort is already underway, and we are on schedule to meet our goal of full study enrollment and efficacy evaluations over the next year.”

This Phase I/II TSC multi-center study builds on previous Phase I/II single and multi-center studies establishing TSC’s human safety profile, pharmacokinetics and dosing regimen in 71 human subjects. The goal of the current study is to establish efficacy and safety of TSC as an addition to the current standard of care in newly diagnosed GBM patients.  Clinical endpoints include progression free survival at 6 months (PFS) and overall survival (OS) at 1 year and 2 years.  The first such endpoint will be reached in the third quarter of 2013, at which time the Company will be communicating with the U.S. Food and Drug Administration (FDA) regarding the prospect of Accelerated Approval of TSC.  TSC has previously been granted an FDA “Orphan Designation” for this use.

Dr. James Larner and Dr. Jason Sheehan of the University of Virginia Medical Center are Study Co-Chairs for this trial.  A listing and contact information for all the trial sites may be viewed online at Clinicaltrials.gov/TSC_Study.

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David Kalergis, CEO, accepted the award for the company before hundreds gathered in Richmond for the 2012 Annual Virginia Healthcare Innovators conference.  Kalergis credited the foundational discoveries of the mechanism of action by Professor Emeritus John Gainer at UVa, and the Charlottesville and Virginia scientific and business community for supporting and enabling the company’s development of this important novel treatment.

Diffusion Pharmaceuticals, LLC of Charlottesville, received the award for the development of a new drug trans sodium crocetinate (TSC) for treatment of solid tumors, including brain tumors, by a novel mechanism of action, altering the diffusion of oxygen in hypotoxic tumor tissue without adversely affecting normal tissue.

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“This is an important development for Diffusion and TSC’s clinical program, and potentially for patients suffering from GBM,” said David Kalergis, the company’s Chief Executive Officer.  “We are currently initiating a phase I/II clinical trial of TSC to test the drug’s safety and effectiveness in patients newly diagnosed with GBM.”

The standard treatment for GBM includes surgical removal of the tumor bulk, followed by radiation and chemotherapy.  Factors within the tumor micro-environment of GBM tissue are thought to account for tumor recurrence, often within months.  TSC is believed to enhance oxygen diffusion into this micro-environment, making treatment-resistant tumor cells up to three times more susceptible to standard radiation and chemotherapy treatments.

About Orphan Drug Designation

The United States Orphan Drug Act of 1983 was created to promote and support the development of new drug therapies for diseases that affect fewer than 200,000 people in the United States.  Orphan Drug Designation provides a sponsor seven years of market exclusivity for the designated therapeutic indication in the United States, from the point at which the therapy is granted marketing approval thereby offering competitive protection.  Orphan Drug Designation also provides access to regulatory support from the FDA, potential FDA fee reductions and tax credits related to development expenses.

About TSC

TSC is a novel small molecule that treats oxygen deficiency (hypoxia) at the cellular level by enhancing the diffusion of oxygen selectively to oxygen-deprived tissues.  TSC creates more hydrogen bonds among the water molecules which make up the bulk of blood plasma, reducing resistance and enabling a faster rate of oxygen diffusion through the plasma.  TSC’s ability to safely promote the movement of oxygen to hypoxic tissue has been consistently demonstrated in animal models, including cancer, stroke and heart attack.  In addition, TSC has been tested in safety and dose-ranging clinical trials in 70 human subjects.  These studies, which showed that TSC was well-tolerated with minimal side effects, also provided valuable pharmacokinetic and dosing information for the next phase of clinical development.

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“This financing is another positive step as we prepare to open our clinical trial of TSC in newly diagnosed brain cancer patients,” said David G. Kalergis, Chief Executive Officer.

Since its founding, Diffusion Pharmaceuticals has raised a total of $23.9 million to date including $21.3 million in private funds, and $2.6 million in Department of Defense research and development contracts.

About TSC

TSC is a novel small molecule that treats oxygen deficiency (hypoxia) at the cellular level by enhancing the diffusion of oxygen selectively to oxygen-deprived tissues.  TSC creates more hydrogen bonds among the water molecules which make up the bulk of blood plasma, reducing resistance and enabling a faster rate of oxygen diffusion through the plasma.  TSC’s ability to safely promote the movement of oxygen to hypoxic tissue has been consistently demonstrated in animal models, including cancer, stroke and heart attack.  In addition, TSC has been tested in safety and dose-ranging clinical trials in 70 human subjects.  These studies, which showed that TSC was well-tolerated with minimal side effects, also provided valuable pharmacokinetic and dosing information for the next phase of clinical development.

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