Pipeline

 

 

Niemann Pick C
 
CycloLab is developing a new chemical entity that is a promising API candidate for the treatment of the well-known lysosomal storage disease, Niemann Pick C. The standpoint of CycloLab in this debated field is that the best treatment can only be achieved if the structure-activity relationships are known, thus we tested dozens of cyclodextrin derivatives from our library that contains hundreds of CDs in various simple in vitro and in vivo animal models to shortlist candidates. This series of experiment lead to our lead compound which is orders of magnitudes more effective compared to HPBCD and which we can already manufacture under cGMP. Now CycloLab is looking for partners to finish the preclinical development and start clinical trials. Non-confidential information can be found here.

Antiviral agents
 
The potential of developing a cyclodextrin based antiviral has been widely published, their efficacy is proven against HIV, influenza, Zika virus, RSV and so on. Our standpoint is similar as in the NPC program, that understanding structure-activity relationships are the key to create an effective treatment. Now we are screening our complete CD library (over 100 cyclodextrins) against several viruses to better understand the mechanism of action and discover the best-working cyclodextrin. Information about the details of this program are subject to CDA between the parties. Non-confidential information can be found here.

SBECD-enabled Lonafarnib
 
One of the main pipeline activities of CycloLab is reformulating known APIs to improve their application profile. Under the scope of this call, we prepared and patented a clear solution of lonafarnib, an API used for the treatment of the rare genetic disease Hutchinson-Gilford progeria. Currently, lonafarnib is administered as a capsule or liquid suspension with a very limited bioavailability due to its extremely low solubility. Since our formulation is a clear liquid, its potential application is an oral solution with improved bioavailability or as an injection. Non-confidential information can be found here.

SBECD-enabled Panobinostat
 
Another asset from the reformulation pipeline of CycloLab is panobinostat solution, prepared with a proprietary technology. Panobinostat is approved as an oral capsule with limited bioavailability that can be substantially improved using the cyclodextrin-based clear solution. The API has a wide range of applications such as the treatment of Multiple myeloma, Diffuse Intrinsic Pontine Glioma or Lysosomal storage diseases. Non-confidential information can be found here.

Cyclodextrins as selective drug delivery systems
 
CycloLab is capable of attaching targeting groups to cyclodextrins opening unique possibilities for developing drug delivery systems. After selecting the suitable, untagged cyclodextrin for a target API in a screening process, a targeting group is chemically attached to the cyclodextrin. This delivery system is still capable to encapsulate the target compound and deliver it to the target cells lead by the targeting group. The API is then released at the place of action, acting selectively on the target cells. Targeting moieties and cyclodextrin cores of a great variety can be used in this platform to develop unique drug delivery systems. Targeting cancer cells and the blood brain barrier are on primary focus now. Information about the details of this program are subject to CDA between the parties. Non-confidential information can be found here.

Cyclodextrins in anti-COVID protective gear
 
The interaction of cyclodextrins with viral particles cannot be solely exploited to develop a potential NCE API, but also as a simpler solution to boost the protection efficacy of masks and other protective wearables. CycloLab won a grant in 2020 for the development of this technology and is now evaluating several compositions and application strategies. Information about the details of this program are subject to CDA between the parties.