GM6

Published

dm+d

Unassigned

New Medicines

Amyotrophic lateral sclerosis (motor neurone disease)

Information

New molecular entity
Not Known
Genervon

Development and Regulatory status

None
None
Phase II Clinical Trials
Yes

May 17: Genervon is planning a PIII trial in 2017 in the US [4].


Apr 15. Designated orphan drug and fast-track status in US for treatment of ALS. [1]

Category

First-in-class analogue of embryonic motoneuronotrophic factor that act as a neurogenesis modulator and apoptosis inhibitor
ALS is the most common form of motor neurone disease; annual incidence of about 2 cases per 100,000 population and prevalence is about 5-7 per 100,000. [3]
Amyotrophic lateral sclerosis (motor neurone disease)
Intravenous

Trial or other data

Genervon has completed a phase 2 RCT in ALS (NCTNCT01854294) with 12 participants: this showed clinically and statistically significant benefits over 12 weeks. Further trials are planned. [1, 2] The company allocates a different synonym to the drug depending on indication - GM604 relates to use in ALS. [1]