Orphazyme Raises Funds, Approaching Markets With Novel Niemann-Pick Therapy

Lead Compound A Heat Shock Protein Amplifier

Denmark’s Orphazyme has raised more funds to market its lead product, the heat shock protein amplifier, arimoclomol, in the US and Europe, and to evaluate the compound for other lysosomal storage and neurodegenerative disorders.  

Shield
Arimoclomol Increases Chaperone Proteins, Shielding Cells From Damage • Source: Shutterstock

Denmark’s biotech Orphazyme A/S is raising between $83m and $97m from an offering of American Depositary Shares (ADSs) on NASDAQ in the US and a private placement in Europe, as it nears the market with its lead candidate, arimoclomol, for the ultra-orphan disease, Niemann-Pick disease Type C. 

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 Rare Diseases

New Data Strengthen Case For RemeGen’s Telitacicept In gMG

 

Results from a China Phase III trial show the potential of RemeGen's fusion protein telitacicept in generalized myasthenia gravis. But study design differences make direct comparisons with argenx's FcRn inhibitor Vyvgart challenging.

Vocal For ‘Glocal’: How Menarini Is Adapting To Outperform In The Asia Pacific

 

Menarini's Asia-Pacific CEO talks to Scrip about how the Italian group is tailoring its portfolio and pricing approach to local markets making strong strides in primary care and consumer health, while also charting a distinct path in rare diseases and oncology. The China market is also a key focus including as a potential source of innovation as is partnering, where the group has a rich history.

Orphans Cling On To Growth Advantage As Market Share Heads Towards 20%

 

It might be the beginning of the end for the orphan drugs party but there is still sales growth enjoyment to be had for the sector, whose star performers are now looking increasingly like mainstream drugs.

Sanofi Set To Shake Up Crowded Hemophilia Space After Qfitlia Approval

 
• By 

The small interference RNA therapeutic can be used by patients regardless of inhibitor status.

More from Scrip

Camzyos nHCM Failure Could Open Door To Threat From Cytokinetics’ Aficamten

 

BMS’s Phase III ODYSSEY-HCM study did not show statistical significance for either of its two primary endpoints.

Taxes, Not Tariffs, Influence US Manufacturing, J&J CEO Says

 

The big pharma forecast a $400m expense this year related to tariffs, but those estimates only reflect medtech tariffs already announced, not potential pharma tariffs.

Disappointing Asthma Phase II Results Take Wind Out Of Sanofi’s Amlitelimab

 

Sanofi’s OX40L candidate missed a Phase II primary endpoint, but the company believes it can still work in a sub-group of ‘heterogeneous inflammatory asthma’ patients.