After Years Of Setbacks, Marinus Closes In On Rare Epilepsy Opportunity

Ganaxolone Potential In Numerous Rare Conditions

After multiple failed attempts to treat rare conditions, Marinus is on the cusp of success with ganaxolone in genetic epilepsy, and has secured a new European partner in Orion.

Epilepsy brain
CDKL5 deficiency disorder (CDD) is a severe genetic form of epilepsy which can strike within the first three months of life. • Source: Alamy

After 18 years of setbacks and disappointments, Marinus Pharmaceuticals has submitted to the US Food and Drug Administration its candidate ganaxolone to treat a rare genetic form of epilepsy.

Established back in 2003, the company has just one drug candidate, GABA-A modulator ganaxolone, and over the years has seen it fail in trials against a string of conditions,

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Business

HAYA Raises $65m To Take First Long Non-Coding RNA Program Into The Clinic

 
• By 

HTX-001 targeting the long non-coding RNA known as Wisper is being developed for non-obstructive hypertrophic cardiomyopathy. HAYA partnered with Lilly last year in obesity.

Sanofi R&D Head’s Words Of Wisdom For Biotechs

 
• By 

Houman Ashrafian tells delegates at Swiss Biotech Day that ‘literally only two things matter in our industry – target credentialing and differentiated pharmacology. The rest is execution.’

Shionogi In $1bn Deal For Japan Tobacco’s Pharma Ops

 
• By 

Mid-sized Japanese pharma firm to acquire tobacco giant’s pharma operations and take private the listed Torii Pharmaceutical business, in bid to build sales reach and strengthen manufacturing.

Sarepta Slammed By Double Blow: Elevidys Sales Miss, Prasad’s CBER Appointment

 
• By 

Sarepta’s stock price was battered after Vinay Prasad was appointed to lead the US FDA’s CBER, then the company’s shares were bruised when Q1 Elevidys sales came in below consensus.

More from Scrip

Sarepta Slammed By Double Blow: Elevidys Sales Miss, Prasad’s CBER Appointment

 
• By 

Sarepta’s stock price was battered after Vinay Prasad was appointed to lead the US FDA’s CBER, then the company’s shares were bruised when Q1 Elevidys sales came in below consensus.

Marea Eyes Cardioprotective Benefit With ANGPTL4 Inhibition

 
• By 

Phase IIa data for ANGPTL4-targeted antibody shows reduced triglycerides and remnant cholesterol, which Marea asserts indicates potential to protect against cardiovascular outcomes.

BeiGene To Trade Beach For Chalet In Swiss Redomiciling As BeOne

 

The biotech saw a slight quarter-over-quarter decline in sales from the BTK inhibitor Brukinsa, which it attributed to seasonality, while revenues overall missed some analyst estimates.