Knopp Biosciences Enters Collaboration with Leading UK Investigators to Commence Phase 2 Clinical Trial of Dexpramipexole in Severe Eosinophilic Asthma

Knopp Biosciences LLC, a privately held drug discovery and development
company focused on delivering breakthrough treatments for inflammatory
and neurological diseases with high unmet need, today announced a
collaboration with a consortium of leading medical researchers in the
United Kingdom to evaluate the ability of Knopp’s lead drug candidate,
dexpramipexole, to reduce exacerbations in people with severe
eosinophilic asthma.

The project is chiefly funded by the National
Institute for Health Research (NIHR) and Medical
Research Council (MRC) of the UK. The Chief Investigator for the
Phase 2 multi-center, 52-week trial is Professor Salman Siddiqui,
Professor of Airway Diseases at the University of Leicester and
Consultant Respiratory Physician at Leicester’s Hospitals.

Dexpramipexole is an orally available small molecule shown to
selectively reduce eosinophil levels in multiple clinical trials,
including in a Phase
2 study in hypereosinophilic syndrome (HES) and a Phase
2 trial in chronic rhinosinusitis with nasal polyps. Knopp is
advancing dexpramipexole into Phase 2 development in severe asthma and
Phase 3 development in HES. Eosinophils are white-blood cells that play
a central role in several debilitating conditions, including asthma,
HES, and other inflammatory diseases.

In the Phase 2 UK trial, dexpramipexole 300 mg daily or placebo will be
administered to 100 asthmatic patients with elevated eosinophil counts.
The primary outcome measure is the annual rate of severe exacerbations.
Secondary outcomes include the CompEx endpoint capturing asthma clinical
deterioration, changes in lung function, and quality of life. The study
will be conducted at approximately 12 clinical sites in the United
Kingdom by a number of the world’s leading asthma investigators
associated with the BEAT Severe Asthma Consortium. ‘

“Dexpramipexole is an oral tablet that lowers the number of eosinophils
in the blood by stopping them from growing in the person’s bone
marrow—the part of the body that produces new blood cells,” Professor
Siddiqui noted. “We want to see if this treatment will be effective in
reducing asthma attacks in patients with the T2-high subtype of severe
asthma. This work has the potential to provide an alternative to oral
steroids or biologic injections for this patient group.”

“We welcome this partnership with the University of Leicester and the
BEAT Severe Asthma consortium to study the clinical utility of
dexpramipexole on reducing exacerbations in patients with severe asthma
and eosinophilia,” said Michael Bozik M.D., CEO of Knopp Biosciences.
“In parallel with Knopp-sponsored trials, the UK study will
significantly enrich our understanding of dexpramipexole’s potential
role as an alternative to biologics in severe asthma.”

ABOUT KNOPP BIOSCIENCES LLC

Knopp Biosciences, based in Pittsburgh, PA, USA, is a privately held
drug discovery and development company focused on delivering
breakthrough treatments for inflammatory and neurological diseases with
a high unmet need. Knopp’s clinical-stage small molecule,
dexpramipexole, is entering Phase 2 clinical trials in eosinophilic
asthma and Phase 3 development in hypereosinophilic syndrome. Knopp’s
preclinical Kv7 platform is directed to small molecule treatments for
neonatal epileptic encephalopathy, other rare epilepsies, tinnitus, and
neuropathic pain. Please visit www.knoppbio.com.

ABOUT THE UNIVERSITY OF LEICESTER

The University of Leicester is led by discovery and innovation – an
international centre for excellence renowned for research, teaching and
broadening access to higher education. It is among the top 25
universities in the Times Higher Education REF Research Power rankings
with 75% of research adjudged to be internationally excellent with
wide-ranging impacts on society, health, culture, and the environment.
The University is home to just over 20,000 students and approximately
3,000 staff.

Find out more: https://le.ac.uk/about

ABOUT THE NATIONAL INSTITUTE FOR HEALTH RESEARCH

The National Institute for Health Research (NIHR) is the UK’s largest
funder of health and care research. The NIHR:

• Funds, supports, and delivers high quality research that benefits the
  NHS, public health and social care
• Engages and involves patients, carers, and the public in order to
  improve the reach, quality and impact of research
• Attracts, trains and supports the best researchers to tackle the
  complex health and care challenges of the future
• Invests in world-class infrastructure and a skilled delivery workforce
  to translate discoveries into improved treatments and services
• Partners with other public funders, charities and industry to maximise
  the value of research to patients and the economy

The NIHR was established in 2006 to improve the health and wealth of the
nation through research, and is funded by the Department of Health and
Social Care. In addition to its national role, the NIHR commissions
applied health research to benefit the poorest people in low- and
middle-income countries, using Official Development Assistance funding.

This press release contains "forward-looking statements," including
statements relating to planned regulatory filings and clinical
development programs. All forward-looking statements are based on
management's current assumptions and expectations and involve risks,
uncertainties and other important factors, specifically including the
uncertainties inherent in clinical trials and product development
programs, the availability of funding to support continued research and
studies, the availability or potential availability of alternative
therapies or treatments, the availability of patent protection for the
discoveries and strategic alliances, as well as additional factors that
may cause Knopp's actual results to differ from our expectations. There
can be no assurance that any investigational drug product will be
successfully developed or manufactured or that final results of clinical
studies will be supportive of regulatory approvals required to market a
product. Knopp undertakes no obligation to update or revise any such
forward-looking statements, whether as a result of new information,
future events or otherwise.

Knopp's pipeline consists of investigational drug products that have
not been approved by the U.S. Food and Drug Administration. These
investigational drug products are still undergoing clinical study to
verify their safety and effectiveness.

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