No Falls.
More Freedom.

Parkinson’s
Patients

Many Parkinson’s Patients live with the fear of falling during daily life activities, which can result in severe injuries. Falls are caused by problems in gait (movement) linked to disease symptoms like motor impairment, cognitive impairment, visual impairment, depression, anxiety and sleep deprivation.

FALL:
AN UNEXPECTED EVENT
IN WHICH THE PERSON
COMES TO REST ON
THE GROUND, FLOOR
OR LOWER LEVEL.

The fear of a Fall is a serious impediment to move freely and enjoy life. A treatment that reduces Falls would have a high impact on the quality of life of many Parkinson’s Patients and could strongly reduce healthcare costs.

of Parkinson’s Patients fall each year.

is the US annual cost for hospitalization, medical care and nursing care

Program

Golden Gait Therapeutics is developing the world’s first investigational drug for the reduction and prevention of Falls. In a clinical study (Phase 2), 45% of Parkinson’s Patients fell less due to taking the investigational drug.

This was shown with the support of Prof. Lieberman, medical doctor of Muhammad Ali.

Investigational Drug

Placebo

Besides reducing Falls, Golden Gait Therapeutics investigational drug has shown additional benefits related to improved gait (movement). This could point towards patients feeling safer on their feet.

Freezing Of Gait (FOG) is defined as a brief, episodic absence or marked reduction of forward progression of the feet despite the intention to walk. It is one of the most debilitating motor symptoms in patients with Parkinson’s Disease (PD) as it may lead to Falls and a loss of independence.

Postural Stability is the ability to control the body position in space for the purpose of movement and balance. It is necessary for maintaining a static position and for assisting body coordination in dynamic position changes.

Trend to improve LID was observed, supported by a significant improvement of LID on ambulation. LID is uncontrolled, involuntary movement associated with levodopa (L-DOPA), used to treat Parkinson’s Disease. It often involves hyperkinetic movements, including chorea, dystonia, and athetosis.

Next to a strong reduction in Falls,
the investigational drug
is safe & well-tolerated. 

01

90% of subjects tolerated escalation
to 6 mg four times a day (maximum dose).

02

No Serious Adverse Effects (SAEs) during NP002 dose escalation or at maintenance dose.

03

No nicotine withdrawal symptoms based on MNWS-R (Minnesota Withdrawal Scale-Revised) scale.

04

No impulsivity signal based on JayMIDI (Jay Modified Minnesota Impulsive Disorders Interview).

05

No worsening of Parkinson’s symptoms based on UPDRS (Unified Parkinson’s Disease Rating Scale).

06

NP002 has the same incidence of discontinuation as placebo (17%).

07

Few cases of nausea can be treated with standard drugs.

Development

Golden Gait Therapeutics is confirming the observations from its previous Phase 2 clinical study.

More Parkinson’s Patients will receive our investigational drug over a longer period of time, while experienced Parkinson’s Disease clinicians will monitor closely their Fall Frequency, Freezing Of Gait (FOG) and Postural Stability.

Science

The Pedunculopontine Nucleus (PPN) is the putative “Fall prevention center” of the brain.

It provides cholinergic inputs (blue arrows) to thalamus, frontal cortex and other parts of the brain affecting gait, balance and cognition.

As PPN degeneration affects the cholinergic system, Fall prevention has been related to the activation of acetylcholine receptors. Golden Gait Therapeutics investigational drug is a small molecule that activates such a receptor.

This receptor activation by our investigational drug is designed to overcome the cholinergic cell loss in the PPN observed in Parkinson’s Disease (PD) patients, probably the most important neurological PD defect/degeneration next to dopamine deficiencies.

Intellectual
Property

Golden Gait Therapeutics has a strong Intellectual Property (IP) portfolio of patents in multiple countries.

Composition and methods
for treatment related to
Falls and Fall frequency in
neurodegenerative diseases
(expiry: 2037)

Use methods and drug
product.

Marketing exclusivity for 3
years in US if Falls
Indication is first for a
nicotine product.

Marketing exclusivity for 7
years in US and 10 years in
Europe if orphan drug
designation is granted.

Team

Management

Friedhelm Blobel, PhD

CEO & Board Member

Board Member (ImaginAb, ENTvantage) Former Director (Sciclone, Hoechst, Roche, etc.)

Thomas Wessel, MD PhD

Chief Medical Officer

Former Director (Janssen, Sunovion, Acorda, Flex, Asceneuron, Relmada)

Catherine Brisson, PhD

Regulatory Executive

Former Director (Neurana, Stellar, MacuSight)

Kenneth Payie, PhD

Manufacturing & Formulation Executive

Director & Founder (Mapp Bio, KGP) Former Director (Lilly, Vanthys)

Jascha Blobel, PhD

Business Development Executive

Director & Founder (Molomics) Former Director (Intelligent Pharma)

Board of Directors

Edgar Engleman, Prof

Board Advisor

Prof. of Pathology & Medicine (Stanford University) Founder & Partner (Vivo Capital) Founder (Genelabs, Dendreon, Medeor, Bolt, Tranquis) Board Member (multiple healthcare companies)

Ari Azhir, PhD

Board Member & Founder

Director & Founder (Neuvivo)
Director (Aquyre Bioscience)

CP Liu, PhD

Board Member

Cofounder (Angarus, Bolt, Medeor, Tranquis)

Friedhelm Blobel, PhD

Board Member & CEO

Board Member (ImaginAb, ENTvantage) Former Director (Sciclone, Hoechst, Roche, etc.)

Media