Our goal is to develop innovative therapies to help patients
suffering from rare genetic disorders
Pluvia is a spin-out from the University of Bergen (Norway) working to commercialize decade long research on protein structure and protein misfolding. In particular we are working to develop small molecule pharmacological chaperones with the potential to stabilize and correct the misfolding of proteins involved in selected loss-of-function diseases, thereby rescuing the enzymatic activity needed to modify the disease.
Our lead development program is focusing on phenylketonuria (PKU), a rare genetic disorder caused by mutations in the enzyme phenylalanine hydroxylase that result in defective degradation and accumulation of the amino acid phenylalanine, which becomes toxic to the brain.
We intend to leverage our unique drug discovery platform to target multiple rare disease caused by protein misfolding.
News
September 2024
Pluvia Biotech Presents Preclinical Data of potential PKU treatment PBAS499 at SSIEM Annual Symposium
This is the first time that PBAS499 data will be shared with the scientific community during the SSIEM conference, where rare genetic disease expert clinicians and scientists from all over the world will come together.
Ann Kari Grindheim, PhD, Director of Preclinical Development and presenter of the data, says “We are proud to share recent data that show the ability of our lead compound PBAS499 to stabilize a majority of the most frequent missense variants of the PAH enzyme. Furthermore, PBAS499 shows convincing efficacy in a PKU mouse model, as well as a clean safety profile, both in vitro and in vivo.”
May 2024
Pluvia Biotech Receives European Orphan Drug Designation
The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted Orphan Drug Designaton (ODD) for Pluvia`s lead compound PBAS499.
“The direct burden of the disease, as well as the burden of the diet, can have significant medical, psychological, and social impacts on these patients. Pluvia’s approach to treat the underlying cause of PKU by stabilizing the deficient PAH enzyme is a promising way to address this.” says Prof. Francjan van Spronsen, MD, PhD, Prof. of Pediatric Metabolic Disease, University Medical Center Groningen, The Netherlands.
April 2024
Pluvia Announces Research Collaboration with PTC Therapeutics.
A research collaboration has been established between Pluvia and PTC Therapeutics.
“We are very proud to be preparing global regulatory submissions for sepiapterin, our promising and differentiated therapy being developed for children and adults with PKU,” said Matthew B. Klein, M.D., PTC Chief Executive Officer. “A collaboration with the expert team at Pluvia allows us to continue to pursue our interest in building on our inborn errors of metabolism platform.”
Jan. 2024
Pluvia Receives Orphan Drug and Rare Pediatric Disease Designations from FDA.
The U.S. Food and Drug Administration has granted both the Orphan Drug Designation and the Rare Pediatric Disease Designation for Pluvia’s lead compound PBAS499 for the treatment of Phenylketonuria.
“We are excited about these valuable designations by the FDA to support Pluvia’s mission to develop a new oral therapy option for PKU patients. We look forward to continue our momentum to realize a Series A round early 2024 and to move PBAS499 to clinical Proof of Concept.” – Willem van Weperen, Pluvia’s CEO
Oct. 2023
We welcome our new CEO, Willem van Weperen, to the Pluvia team.
“We are delighted to welcome Willem as our new CEO. His relevant expertise and network align perfectly with Pluvia’s stage of development. Under his guidance, we believe Pluvia Biotech will make significant strides in developing a new therapeutic option for patients with PKU.” – Sveinung Hole, Chairman of the Board at Pluvia Biotech
OUR SCIENCE
Developing small molecule pharmacological chaperones
– a novel therapeutic approach to protein
misfolding diseases
Mutations in the DNA sequence of a gene may impact the structure and function of the encoded protein, resulting in genetic disorders. To date more than 6.000 genetic disorders have been identified, affecting an estimated 350 million individuals. Increasing knowledge on the pathogenic mechanisms has revealed that a large number of genetic disorders are associated with destabilization and misfolding of the coded proteins.
Pervasive unstable or misfolded proteins are associated with a variety of diseases. The harmful effect of the misfolded protein may be due to: (a) ‘loss-of-function’, due to effective degradation of the mutant proteins by the quality control system, as observed in many genetic disorders, such as PKU, porphyria, and cystic fibrosis, or (b) ‘gain-of-function’, as seen in many neurodegenerative diseases such as Alzheimer’s disease, Parkinson’s disease and Huntington’s disease, caused by formation of toxic amyloid aggregates.
Developing effective therapies for rare genetic- or degenerative diseases has proven to be challenging. One particularly promising strategy is the use of pharmacological chaperones that are able to prevent and/or correct misfolding by selectively binding to key sites of the proteins. Recently efforts to develop pharmacological chaperones have been successful in both cystic fibrosis, a life-threatening disease affecting lung function and in Fabry disease, a rare genetic lysosomal storage disorder.
The research group at the University of Bergen (UiB) was the first to envision and prove the potential of a pharmacological chaperone approach for the treatment of PKU. The research of this group on pharmacological chaperones, and on PKU in particular, has been highlighted in a number of top impact journals including Nature Reviews Drug Discovery (7-2008) and Science-Business eXchange (1 2008), among others.
The company intends to develop a proprietary technology platform
and leverage our unique drug discovery platform to target multiple
rare disease caused by protein misfolding.
OUR PROJECTS
Our lead project aims at giving PKU patients
a shot at a normal life
Pluvia is currently focusing on developing pharmacological chaperones as potential treatment of PKU, the most common inborn error of metabolism.
PKU is a rare inborn metabolic disorder affecting approximately 1:10,000 newborns. The disease is caused by mutations in the liver enzyme phenylalanine hydroxylase (PAH), resulting in a decreased capacity to convert the amino acid phenylalanine into tyrosine and the build-up of neurotoxic concentrations of phenylalanine. Testing for the disease is included in the newborn screening program throughout the Western world
If left untreated, classical PKU symptoms are severe mental, psychomotor and growth retardation, seizures, and psychiatric disorders. Within the first week of life patients need to follow restrictive low-protein diet for life. Even then, patients experience severe problems resulting in a low quality of life and large social burden. It has become increasingly clear that the outcome of the diet-treatment is suboptimal leading to neuropsychiatric and cognitive problems, growth retardation, nutritional deficiencies, and bone pathology, in addition to reduced quality of life. The only existing non-diet treatment for the condition, Kuvan®, allows for a slightly lighter diet regime for 20 – 30% of the PKU patients. This lack of optimal therapy means that very few of the patients can live a normal life today. There is clearly an unmet medical demand for this patient group.
Our vision is to develop a small-molecule chaperone to prevent the PAH mutation-associated misfolding and thereby restoring the native enzymatic activity of the patient’s own dysfunctional PAH.
OUR TEAM
A small, international team with a burning ambition
to make a difference
Willem van Weperen; CEO
Senior biotech leader with more than 25 years of experience in development and commercialization of therapies for patients with rare diseases in companies like Genzyme, Amicus, Myokardia and Intercept.
Torgeir Vaage; CFO
Extensive experience from the financial sector in Norway. For the last ten years been involved in a number of early stage biotechnology companies in the Nordic region in the capacity as CEO and CFO. He holds a MSc from Norges Handelshøyskole (Norway) and a PhD from UC Berkeley.
Prof Aurora Martinez; CTO
PhD in Biochemistry. Professor at Dept. of Biomedicine, University of Bergen. Expertise in biophysics, structural biology, drug design and cellular biology, investigating how structure determines molecular recognition, stability and function in selected biomolecular networks. Expertise in compound screening and early stage drug discovery, with special focus on PKU and development of novel therapies based on pharmacological chaperones.
Dr. Mikael Thomson; Drug Development Consultant
PhD in pharmacology and toxicology), Mikael Thomson has worked in the pharmaceutical area for close to 20 years, and comes with a broad background from drug development, extending from preclinical development through phase 2 clinical work, having worked with both large pharma and biotech start-ups in the Nordic region. He is a member of several pharmaceutical societies and has authored or co-authored more than 50 publications/abstracts related to early drug development as well co-author of several patents.
Dr. Ann Kari Grindheim; Director of non-clinical development
Ann Kari Grindheim is a pharmacist by training and has extensive experience from cell biology and biotechnology research and development from the University of Bergen. Ann Kari is in charge of all cell culture work in Pluvia as well as managing the preclinical development.
Dr. Karina Skjervheim Prestegård; Senior Scientist
Karina S. Prestegård has a Master of Science in Biology and PhD in Biomedicine from the University of Bergen. She has several years of experience from working with transgenic mouse models, and is in charge of planning and executing the in vivo experiments.
Altanchimeg Altankhuyag; Researcher
Altanchimeg Altankhuyag has a Master of Science in Molecular Biology (University of Bergen). She is doing the day-to-day maintenance of our mouse colonies, with genotyping as her main task. She is also involved in the planning and execution of the in vivo experiments.
Trond-André Kråkenes; Research technician
Trond-André Kråkenes has a Bachelors degree in Nanotechnology and is currently doing a Master in Nanoscience. Trond-André is in charge of performing enzyme activity assays and is also supporting other research activities in our project.
OUR INVESTORS
With us in our quest to help vulnerable patient populations
we have the support from a strong base
of Norwegian investors
We are pround to have attracted a strong base of Norwegian investors to support our ambition to develop ground-breaking new therapies for vulnerable patient populations with unmet medical needs:
Bergen Teknologioverføring
Bergen Teknologioverføring AS (BTO) is the technology transfer office in Bergen, owned by University of Bergen, Haukeland University Hospital, the Institute of Marine Research, SIVA, Western Norway University of Applied Sciences and NHH. BTO´s mission is to help its owners and partners to bring their research results out to the market, ensuring that knowledge and inventions benefit individuals, the industry and society as a whole.
Bergen Research Foundation
Bergen Research Foundation gives grants toward research and research supporting activities at the University of Bergen and Haukeland University Hospital, and other Norwegian research institutions if they cooperate with the institutions in Bergen. It also gives grants to support research at the University of Bergen and Haukeland University Hospital at the interface between basic research and clinical research. Since 2017 Bergen Research Foundation is also selectively investing in promising early-stage life science companies.
Sarsia Seed
Sarsia Seed AS is a Norwegian Seed Capital Fund which invests in Norwegian early phase technology companies within the energy/cleantech and biotechnology/life science sectors.
Investinor
Investinor is an evergreen investment company funded by the Norwegian government. Investinor invests in promising unlisted (private) companies aiming for international growth and expansion, and has a long-term perspective on its ownership in it´s portfolio companies.
