신경보호효과 개선을 위한 약(Pridopidine)으로 현재 2임상시험 중에 있으며 헌팅턴병 증상을 늦추는 효과를 보인다고 합니다.

아직은 임상시험 단계인 약이며 추가 임상시험 단계등의 결과나오면 소식전해드리겠습니다.

Trial finds Teva drug could slow Huntington's disease

http://www.globes.co.il/en/article-teva-pridopidine-could-slow-huntingtons-d

isease-1001153229

Globes Correspondent 09/19/2016

Teva Pharmaceutical Industries Ltd. <http://www.tevapharm.com/>  today

announced what could be a major breakthrough in the treatment of

Huntington's Disease (HD) with positive results from its exploratory Phase

II PRIDE-HD study. The trial found a statistically significant impact on the

endpoint of the progression of Huntington Disease at 52 weeks following

treatment with Pridopidine at certain doses versus placebo. The effect of

Pridopidine was further evident in a sub-population of patients with early

stage HD, an effect first observed at 26 weeks.

"Slowing down the progression of this disease has proven to be impossible

until now. These findings give us a reason to believe we may be finally

making progress in slowing deterioration of disease," said Spyros

Papapetropoulos, Teva's VP Clinical Development, Neurodegenerative Diseases.

This was a 52-week, dose-ranging trial of Pridopidine twice daily versus

placebo, in the treatment of Huntington disease (HD). The study was directed

at measuring improvement in motor function and the effect on HD progression.

An unusually high placebo effect, extending beyond that expected from

previous studies, limited the ability to determine treatment effects on

assessments of HD motor scores. Evidence of symptomatic impact, however, was

seen in the early stage HD patient sub-population, with improvement in Total

Motor Score (TMS) and dystonia observed at 26 and 52 weeks in this patient

sub-set (stage 1 HD) at specific doses.

The discovery of Pridopidine's previously unknown mode of action as a potent

agonist of the Sigma 1 Receptor (S1R) resulted in a change in PRIDE-HD study

design, from a 26-week study focused on symptoms, to a 52-week study focused

on exploring pridopidine's potential impact on disease progression, as

measured by Total Functional Capacity (TFC).

"I am encouraged by these results, which provide us with clear insights into

the approach to be taken in Phase III development", said Michael Hayden,

President of Teva Global R&D and Chief Scientific Officer. "My obvious hope

is that this will provide the HD community with a medicine capable of

slowing down the progression of this devastating disease."

"These study results are very important for the HD community and for the

continued development of pridopidine. Firstly, pridopidine's safety profile

has been confirmed and extended. Secondly, we now have a clearer idea of the

dosages to study in Phase 3. Lastly, we have some of the most encouraging

evidence to date about an intervention which may slow the inexorable

functional decline of HD," said Karl Kieburtz, M.D., M.P.H., Director of the

Clinical & Translational Science Institute at the University of Rochester

Medical Center.

The results seen in this exploratory study will need to be confirmed in a

Phase III program that will be developed in collaboration with relevant

regulatory agencies.

Pridopidine is an investigational, oral small molecule being developed for

the treatment of HD that exerts its effect as an agonist of S1R. S1R plays a

key role in neuroprotection through increased production of brain-derived

neurotrophic factor (BDNF). Levels of BDNF are decreased in HD and other

neurodegenerative disorders including Parkinson's disease, Alzheimer's

disease and ALS.

================================

Huntington Study Group PRIDE HD Study Information:

http://huntingtonstudygroup.org/current-clinical-trials/pride-hd-study/

.       No information available yet on enrolling for the Phase 3 study.

.       No facilities in Florida participated in the Phase II study:

https://clinicaltrials.gov/ct2/show/study/NCT02006472?show_locs=Y#locn