Press Release

FDA Approves Start of Innovative On-Ambulance Phase 2 Clinical Trial for Treatment of Acute Stroke by Diffusion Pharmaceuticals

Trial to be Led by Experts from University of California Los Angeles (UCLA) and University of Virginia (UVA) To Expand Treatment Window, Improve Patient Outcomes for Stroke

Company Release - 9/12/2018 9:00 AM ET

CHARLOTTESVILLE, Va., Sept. 12, 2018 (GLOBE NEWSWIRE) -- Diffusion Pharmaceuticals Inc. (Nasdaq: DFFN), a cutting-edge biotechnology company developing new treatments for life-threatening medical conditions by improving the body’s ability to bring oxygen to the areas where it’s needed most, announced today that it has received FDA approval to enroll patients in an ambulance-based Phase 2 clinical trial testing its lead drug, trans sodium crocetinate (TSC), for the treatment of acute stroke.

The trial, named PHAST-TSC (Pre-Hospital Ambulance Stroke Trial-TSC), will involve 23 hospitals across urban, suburban, and rural areas in Los Angeles and Central Virginia, working closely with approximately 150 emergency medical transport groups. Results for the PHAST-TSC trial could be available in just under two years – a critically fast turnaround for this potential improvement in the front-line medical treatment of stroke, which afflicts nearly 800,000 Americans each year at an estimated cost of over $34 billion to the U.S. economy.

Nearly two million brain cells die each minute during a stroke. As a result, medical providers seek to provide treatment for patients as quickly as possible during the hours immediately after stroke onset. TSC, which will be administered by paramedics while the stroke victim is still in the ambulance, may offer new hope for these patients by increasing the amount of oxygen directed to affected tissue, potentially reducing cell death and improving patient outcomes. In addition to stroke, TSC has shown safety and efficacy to date in oncology, with a Phase 3 trial in glioblastoma (GBM) brain cancer currently enrolling.  

The Study Chair for the PHAST-TSC trial is Jeffrey Saver, M.D., professor of clinical neurology and director of the stroke unit at the David Geffen School of Medicine at UCLA. Co-Principal Investigators for the study are Andrew Southerland, M.D. (UVA) and Nerses Sanossian, M.D. (University of Southern California).

“Dr. Saver and his team at UCLA have been pioneers in on-ambulance therapy, and, along with Dr. Johnston and her team at the University of Virginia, we have the expertise necessary to test TSC’s effectiveness in patients suffering from acute stroke,” said John L. Gainer, PhD, Diffusion’s Chief Science Officer and inventor of the TSC molecule. “While combining the talents of a pharmaceutical company with those of two of our country’s leading academic medical centers is, perhaps, a paradigm shift for creating new clinical trials, it is one which will hopefully produce substantial benefits for stroke victims and their families.”

“TSC was originally designed for the US military to help treat life-threatening injuries on the battlefield – a place where every minute can be the difference between life and death.  This FDA approval to begin patient enrollment in PHAST-TSC gives us the opportunity to show that treatment with TSC could make an impact on a different battlefield, one where stroke patients are also in a race against the clock,” said David Kalergis, Chairman and CEO of Diffusion. “As we return to our company’s roots in emergency medicine and work to identify possible strategic partnerships, we will also continue applying TSC to other areas of medicine, including GBM brain cancer in our ongoing Phase 3 trial.”

“In addition to testing TSC in acute stroke, the novelty of this study is that patients will receive treatment in the ambulance prior to hospital arrival,” said Karen C. Johnston, MD, MSc, Harrison Distinguished Professor of Neurology and Associate Vice President for Clinical & Translational Research at UVA.  “Dr. Saver and Dr. Southerland are both leaders in the development of innovative approaches to treating acute stroke patients in the ambulance.”

Historically, advancements in safe and effective pre-hospital treatment for patients immediately following a stroke have eluded researchers. One reason is that ischemic stroke (resulting from a clot in the brain) and hemorrhagic stroke (caused by a burst blood vessel in the brain) produce almost identical symptoms. Treatment is complicated by the fact that differentiating between these two stroke types requires special imaging equipment usually found only in-hospital.

There are no existing ischemic stroke therapeutics which can be administered without first diagnosing the type of stroke.  For hemorrhagic stroke there are currently no approved therapeutics of any kind, either on-ambulance or in-hospital.

Despite numerous stroke clinical trials to date involving “neuroprotectants” – drugs intended to minimize the death of brain cells – none have shown sufficient efficacy to be approved. TSC is also a neuroprotectant, but unlike many of the other drugs which have been studied previously for stroke, TSC is designed to stabilize brain oxygen levels, starting from the time the patient first reaches the ambulance.

The one currently FDA-approved stroke drug, Genentech’s Tissue Plasminogen Activator (tPA) dissolves blood clots, but can only be used in ischemic stroke and may prove harmful if given to hemorrhagic stroke patients. Preclinical studies suggest that patient outcomes may improve synergistically from the use of TSC and tPA, potentially without increasing risk to patients. Since TSC can be administered upon ambulance arrival for any acute stroke, whether ischemic or hemorrhagic, it could become the immediate first treatment for any acute stroke, with hospital-administered treatments to follow.

Additional information about the PHAST-TSC trial

The Phase 2, randomized, double-blind, placebo controlled PHAST-TSC trial will enroll 160 patients, with 128 coming from up to 20 hospitals in the greater Los Angeles area and 32 patients from 3 central Virginia hospitals.  Half the patients will be randomized to receive TSC and half to placebo.  The primary end point will be mortality or patient neurological disability as measured by the modified Rankin scale administered 90 days after stroke occurrence. Because the follow-up period in the trial is only 90 days post-treatment, complete data collection is expected within 21 months of first patient enrollment, offering a timely and cost-effective assessment of a potential breakthrough therapeutic in one of healthcare’s most significant unmet medical needs. 

The PHAST-TSC Diffusion-sponsored clinical trial is a cooperative effort by Diffusion’s Chief Science Officer and inventor of the TSC molecule, Dr. John L. Gainer, PhD and Diffusion’s Scientific Advisory Board Chair, Dr. Guy Chisolm, PhD, in close cooperation with the Director of UCLA’s Stroke Center, Jeffrey Saver, M.D and the Chair of UVA’s Department of Neurology Karen Johnston, M.D. Co-Principal Investigators for the study are Andrew Southerland, M.D. (UVA) and Nerses Sanossian, M.D. (University of Southern California). Other important participants include the approximately 150 emergency medical transport groups based in Los Angeles and Central Virginia. Diffusion will be seeking additional financing or a partnering arrangement to help fund the trial.

More information about Diffusion’s clinical programs, including both its INTACT Phase 3 trial in inoperable GBM brain cancer and its PHAST-TSC acute stroke program, are available on Diffusion’s Website at www.diffusionpharma.com/clinicalprograms.

About Diffusion Pharmaceuticals Inc.

Diffusion Pharmaceuticals Inc. is an innovative biotechnology company developing new treatments that improve the body’s ability to bring oxygen to the areas where it’s needed most —offering new hope for the treatment of life-threatening medical conditions.

Diffusion’s lead drug, Trans Sodium Crocinate (TSC) was originally developed in conjunction with the Office of Naval Research, which was seeking a way to treat hemorrhagic shock caused by massive blood loss on the battlefield. 

Evolutions in research have led to Diffusion’s focus today: Fueling Life by taking on some of medicine’s most intractable and difficult-to-treat diseases, including stroke, GBM brain cancer, pancreatic cancer, and brain metastases. In each of these diseases, hypoxia – when essential tissue in your body is deprived of oxygen – has proved to be a significant obstacle for medical providers and the target for TSC’s novel mechanism.

In 2018, the Company began enrolling patients in its Phase 3 INTACT program, using TSC to target inoperable GBM brain cancer. It’s in-ambulance Phase 2 acute stroe protocol was granted FDA clearance to proceed in September, 2018.  Additional pre-clinical data supports the potential use of TSC as a treatment for other conditions where hypoxia plays a major role, such as myocardial infarction, respiratory diseases such as COPD, peripheral artery disease, and neurodegenerative conditions such as Alzheimer’s and Parkinson’s.

In addition, RES-529, the Company’s PI3K/AKT/mTOR pathway inhibitor that dissociates the mTORC1 and mTORC2 complexes, is in the preclinical testing phase for GBM.

Diffusion is headquartered in Charlottesville, Virginia—a hub of advancement in the life science and biopharmaceutical industries and is led by CEO David Kalergis, a 30-year industry veteran and company co-founder. 

Forward-Looking Statements

To the extent any statements made in this news release deal with information that is not historical, these are forward-looking statements under the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements about the company's plans, objectives, expectations  and intentions with respect to future operations and products, the potential of the company's technology and product candidates, the anticipated timing of future clinical trials, and other statements that are not historical in nature, particularly those that utilize terminology such as "would," "will," "plans," "possibility," "potential," "future," "expects," "anticipates," "believes," "intends," "continue," "expects," other words of similar meaning, derivations of such words and the use of future dates. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Uncertainties and risks may cause the company's actual results to be materially different than those expressed in or implied by such forward-looking statements. Particular uncertainties and risks include: general business and economic conditions; the company's need for and ability to obtain additional financing or partnering arrangement; and the difficulty of developing pharmaceutical products, obtaining regulatory and other approvals and achieving market acceptance, and the various risk factors (many of which are beyond Diffusion’s control) as described under the heading “Risk Factors” in Diffusion’s filings with the United States Securities and Exchange Commission. All forward-looking statements in this news release speak only as of the date of this news release and are based on management's current beliefs and expectations. Diffusion undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Contact:
Rob Corradi
rob@penpublicaffairs.com
410-212-5483

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Source: Diffusion Pharmaceuticals Inc.