Last update 02 May 2024
ProQR Therapeutics NV
Last update 02 May 2024
Overview
Tags
Congenital Disorders
Eye Diseases
Endocrinology and Metabolic Disease
Oligonucleotide
Antisense oligonucleotides
Disease domain score
A glimpse into the focused therapeutic areas
No Data
Technology Platform
Most used technologies in drug development
No Data
Targets
Most frequently developed targets
No Data
| Disease Domain | Count |
|---|---|
| Endocrinology and Metabolic Disease | 3 |
| Nervous System Diseases | 3 |
| Top 5 Drug Type | Count |
|---|---|
| Oligonucleotide | 7 |
| Antisense oligonucleotides | 4 |
Related
23
Drugs associated with ProQR Therapeutics NVTarget |
Mechanism usherin modulators |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhasePhase 2/3 |
First Approval Ctry. / Loc.- |
First Approval Date- |
Target |
Mechanism Cep290 modulators [+1] |
Active Org. |
Originator Org. |
Active Indication |
Drug Highest PhasePhase 2/3 |
First Approval Ctry. / Loc.- |
First Approval Date- |
Target |
Mechanism RHO gene transference |
Active Org. |
Originator Org. |
Active Indication |
Drug Highest PhasePhase 2 |
First Approval Ctry. / Loc.- |
First Approval Date- |
22
Clinical Trials associated with ProQR Therapeutics NVA Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with early to moderate vision loss.
Start Date15 Dec 2021 |
Sponsor / Collaborator |
A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Sirius)
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Start Date08 Dec 2021 |
Sponsor / Collaborator |
An Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Helia)
PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with RP due to mutations in exon 13 of the USH2A gene, for an anticipated period of 24 months, or until provision of continued treatment by other means is available, provided the subject's benefit-risk determination remains positive.
Start Date16 Sep 2021 |
Sponsor / Collaborator |
100 Clinical Results associated with ProQR Therapeutics NV
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0 Patents (Medical) associated with ProQR Therapeutics NV
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3
Literatures (Medical) associated with ProQR Therapeutics NV14 Mar 2023·Molecular therapy. Nucleic acids
Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C
Article
Author: Kaltak, Melita ; de Bruijn, Petra ; Piccolo, Davide ; Lee, Sang-Eun ; Dulla, Kalyan ; Hoogenboezem, Thomas ; Beumer, Wouter ; Webster, Andrew R. ; Collin, Rob W. J. ; Cheetham, Michael E. ; Platenburg, Gerard ; Swildens, Jim
Stargardt disease type 1 (STGD1) is the most common hereditary form of maculopathy and remains untreatable. STGD1 is caused by biallelic variants in the ABCA4 gene, which encodes the ATP-binding cassette (type 4) protein (ABCA4) that clears toxic byproducts of the visual cycle. The c.5461-10T>C p.[Thr1821Aspfs∗6,Thr1821Valfs∗13] variant is the most common severe disease-associated variant, and leads to exon skipping and out-of-frame ABCA4 transcripts that prevent translation of functional ABCA4 protein. Homozygous individuals typically display early onset STGD1 and are legally blind by early adulthood. Here, we applied antisense oligonucleotides (AONs) to promote exon inclusion and restore wild-type RNA splicing of ABCA4 c.5461-10T>C. The effect of AONs was first investigated in vitro using an ABCA4 midigene model. Subsequently, the best performing AONs were administered to homozygous c.5461-10T>C 3D human retinal organoids. Isoform-specific digital polymerase chain reaction revealed a significant increase in correctly spliced transcripts after treatment with the lead AON, QR-1011, up to 53% correct transcripts at a 3 μM dose. Furthermore, western blot and immunohistochemistry analyses identified restoration of ABCA4 protein after treatment. Collectively, we identified QR-1011 as a potent splice-correcting AON and a possible therapeutic intervention for patients harboring the severe ABCA4 c.5461-10T>C variant.
01 Oct 2019·Journal of Aerosol Medicine and Pulmonary Drug DeliveryQ4 · MEDICINE
The Cystic Fibrosis-Like Airway Surface Layer Is not a Significant Barrier for Delivery of Eluforsen to Airway Epithelial Cells
Q4 · MEDICINE
Article
Author: Brinks, Vera ; Lipinska, Katarzyna ; de Jager, Miranda ; Beumer, Wouter ; Button, Brian ; Livraghi-Butrico, Alessandra ; Henig, Noreen ; Matthee, Bianca
Background: Eluforsen (previously known as QR-010) is a 33-mer antisense oligonucleotide under development for oral inhalation in cystic fibrosis (CF) patients with the delta F508 mutation. Previous work has shown that eluforsen restores CF transmembrane conductance regulator (CFTR) function in vitro and in vivo. To be effective, eluforsen has first to reach its primary target, the lung epithelial cells. Therefore, it has to diffuse through the CF airway surface layer (ASL), which in CF is characterized by the presence of thick and viscous mucus, impaired mucociliary clearance, and persistent infections. The goal of this study was to assess delivery of eluforsen through CF-like ASL. Methods and Results: First, air-liquid interface studies with cultured primary airway epithelial cells revealed that eluforsen rapidly diffuses through CF-like mucus at clinically relevant doses when nebulized once or repeatedly, over a range of testing doses. Furthermore, eluforsen concentrations remained stable in CF patient sputum for at least 48 hours, and eluforsen remained intact in the presence of various inhaled CF medications for at least 24 hours. When testing biodistribution of eluforsen after orotracheal administration in vivo, no differences in lung, liver, trachea, and kidney eluforsen concentration were observed between mice with a CF-like lung phenotype (ENaC-overexpressing mice) and control wild-type (WT) littermates. Also, eluforsen was visualized in the airway epithelial cell layer of CF-like muco-obstructed mice and WT littermates. Finally, studies of eluforsen uptake and binding to bacteria prevalent in CF lungs, and diffusion through bacterial biofilms showed that eluforsen was stable and not absorbed by, or bound to bacteria. In addition, eluforsen was found to be able to penetrate Pseudomonas aeruginosa biofilms. Conclusions: The thickened and concentrated CF ASL does not constitute a significant barrier for delivery of eluforsen, and feasibility of oral inhalation of eluforsen is supported by these data.
01 Jan 2019·PLoS OneQ3 · CROSS-FIELD
Evaluation of eluforsen, a novel RNA oligonucleotide for restoration of CFTR function in in vitro and murine models of p.Phe508del cystic fibrosis
Q3 · CROSS-FIELD
ArticleOA
Author: Beumer, Wouter ; Swildens, Jim ; Leal, Teresinha ; Noel, Sabrina ; Anthonijsz, Herma ; van der Horst, Geert ; Kuiperij-Boersma, Hester ; Potman, Marko ; van Putten, Charlotte ; Biasutto, Patricia ; Platenburg, Gerard ; de Jonge, Hugo ; Henig, Noreen ; Ritsema, Tita
Cystic fibrosis (CF) is caused by mutations in the gene encoding the epithelial chloride channel CF transmembrane conductance regulator (CFTR) protein. The most common mutation is a deletion of three nucleotides leading to the loss of phenylalanine at position 508 (p.Phe508del) in the protein. This study evaluates eluforsen, a novel, single-stranded, 33-nucleotide antisense oligonucleotide designed to restore CFTR function, in in vitro and in vivo models of p.Phe508del CF. The aims of the study were to demonstrate cellular uptake of eluforsen, and its efficacy in functional restoration of p.Phe508del-CFTR both in vitro and in vivo. In vitro, the effect of eluforsen was investigated in human CF pancreatic adenocarcinoma cells and human bronchial epithelial cells. Two mouse models were used to evaluate eluforsen in vivo. In vitro, eluforsen improved chloride efflux in CF pancreatic adenocarcinoma cell cultures and increased short-circuit current in primary human bronchial epithelial cells, both indicating restoration of CFTR function. In vivo, eluforsen was taken up by airway epithelium following oro-tracheal administration in mice, resulting in systemic exposure of eluforsen. In female F508del-CFTR mice, eluforsen significantly increased CFTR-mediated saliva secretion (used as a measure of CFTR function, equivalent to the sweat test in humans). Similarly, intranasal administration of eluforsen significantly improved nasal potential difference (NPD), and therefore CFTR conductance, in two CF mouse models. These findings indicate that eluforsen improved CFTR function in cell and animal models of p.Phe508del-CFTR-mediated CF and supported further development of eluforsen in human clinical trials, where eluforsen has also been shown to improve CFTR activity as measured by NPD.
76
News (Medical) associated with ProQR Therapeutics NV14 Mar 2024
DUBLIN, March 13, 2024 /PRNewswire/ -- The "Royalty Rates in Pharmaceutical and Biotechnology Deals" report has been added to
ResearchAndMarkets.com's offering.
Royalty Rates in Pharmaceutical and Biotechnology Deals is your one-stop source for providing real-deal information on hundreds of transactions, including the technology licensed, royalty rates, license fees, upfront and milestone payments.
This report provides details of the latest partnering deals which disclose a royalty rate, announced in the pharmaceutical, biotechnology and diagnostic sectors. The report provides details of partnering deals disclosing royalty rates from 2010 to 2024.
The report provides an overview of how and why companies enter partnering deals where a royalty is payable upon commercialization of the compound or technology.
Understanding the flexibility of a prospective partner's negotiated deals terms provides critical insight into the negotiation process in terms of what you can expect to achieve during the negotiation of terms. Whilst many smaller companies will be seeking details of the payments clauses, the devil is in the detail in terms of how payments are triggered - contract documents provide this insight where press releases and databases do not.
This report contains a comprehensive listing of all partnering deals announced since 2010 where a quantitative royalty rate has been disclosed, as recorded in the Current Agreements deals and alliances database. Each deal record and royalty disclosure is available in further detail via a link to online copy of the deal including actual contract document, where available, as submitted to the Securities Exchange Commission by companies and their partners.
The initial chapters of this report provide an orientation of royalty rate trends in pharma and biotech since 2010. Chapter 1 provides an introduction to the report, whilst chapter 2 provides an overview of the trends in royalty rates in the pharma and biotech sectors since 2010.
Chapter 3 provides a more detailed insight into the structure of a royalty rate clause and how it fits with the other financial terms of the partnering deal.
Chapter 4 provides an insight into companies active in disclosing royalty rates as well as those deals attracting the highest royalty rates.
Chapter 5 provides comprehensive and detailed access to deals which disclose a royalty rate since 2010 where a contract document is available. Contract documents provide an in-depth insight into the actual deal terms agreed between the parties with respect to the royalty rate.
In addition the report includes a comprehensive deal directory listing of all deals where a royalty rate has been disclosed announced since 2010. Each listing is organized as a directory by company A-Z, stage of development at signing, therapeutic area, and technology type. Each deal title links via hyperlink to an online version of the deal record including, where available, the actual contract document.
The report also includes numerous table and figures that illustrate the trends in royalty rates in pharma and biotech deal making since 2010. In conclusion, this report provides everything a prospective dealmaker needs to know about royalty rates in the pharma and biotech sector.
Royalty Rates in Pharmaceutical and Biotechnology Deals provides the following benefits:
Understand royalty rate trends since 2010
Analysis of the structure of royalty clauses with real life case studies
Browse collaboration and licensing deals which reveal a royalty rate
Benchmark analysis - identify market value of transactions
Financials terms - upfront, milestone, royalties
Directory of deals by company A-Z, therapy focus and technology type
Access contract documents - insights into deal structures
Due diligence - assess suitability of your proposed deal terms for partner companies
Save hundreds of hours of research time
Analyzing actual contract agreements allows assessment of the following:
What are the precise royalty rates granted?
What is actually granted by the agreement to the partner company?
What exclusivity is granted?
What is the payment structure for the deal?
How are sales and payments audited?
What is the deal term?
How are the key terms of the agreement defined?
How are IPRs handled and owned?
Who is responsible for commercialization?
Who is responsible for development, supply, and manufacture?
How is confidentiality and publication managed?
How are disputes to be resolved?
Under what conditions can the deal be terminated?
What happens when there is a change of ownership?
What sublicensing and subcontracting provisions have been agreed?
Which boilerplate clauses does the company insist upon?
Which boilerplate clauses appear to differ from partner to partner or deal type to deal type?
Which jurisdiction does the company insist upon for agreement law?
Key Topics Covered:
Executive Summary
Chapter 1 - Introduction
1.1. What are royalties?
1.2. History of royalty rates
1.3. Royalties in pharmaceuticals and biotechnology
1.4. Royalties versus revenue share
1.5. Overview of the report
Chapter 2 - An overview of pharmaceutical and biotechnology royalty rates
2.1. Trends in royalty rates 2010 - 2024
2.2. How do revenue shares figure?
2.3. A review of recent literature
2.4. Royalty rates in the future
Chapter 3 - The royalty clause in pharmaceutical and biotechnology deals
3.1. Partnering agreement structure
3.2. Structure of a typical royalty clause
3.3. Example royalty clauses
3.3.1. Case study 1
3.3.2. Case study 2
3.3.3. Case study 3
3.3.4. Case study 4
Chapter 4 - Companies actively disclosing royalty rates
4.1. Most active companies in disclosing royalty rates
Chapter 5 - Royalty rate contract directory
Explore royalty rates within the deal contract document to gain greater insight
Deal directory
Deal Directory - Royalty rates in deals - by company A-Z
Deal Directory - Royalty rates in deals - by therapy area
Deal Directory - Royalty rates in deals - by stage of development at signing
Deal Directory - Royalty rates in deals - by technology type
Royalty rate references
Example royalty rate deal contract document
List of Figures
Figure 1: Trends in pharma and biotech royalty rates, 2010 - 2024
Figure 2: Components of the partnering deal structure
Figure 3: Components of the partnering royalty clause
Figure 4: Most active companies disclosing royalty rate 2010 - 2024
Figure 5: Licensing agreement for rhAAT - rEVO Biologics, LFB Biotechnologies
Companies Mentioned
2seventy bio
3a-diagnostics
3D Medicines
4SC
23andMe
Abbott Laboratories
Abbvie
Abeona Therapeutics
Ablynx
Abraxis BioScience
ABVC BioPharma
ACADIA Pharmaceuticals
Acceleron Pharma
Acelrx Pharmaceuticals
Acer Therapeutics
Acerus
Acrotech Biopharma
Actavis (acquired by Watson)
Active Biomaterials
Acura Pharmaceuticals
Adamis Pharmaceuticals
Adaptimmune
ADC Therapeutics
Adeona Pharmaceuticals
Adial Pharmaceuticals
Aditx Therapeutics
Adlai Nortye
Aduro BioTech
Advanced Dosage Forms
Advanz Pharma
Advaxis
Advirna
Aegerion Pharmaceutical
Aeglea BioTherapeutics
Aerie Pharmaceuticals
AEterna Zentaris
Aexon Labs
Affimed Therapeutics
Afritex Medical Products
AFT Pharmaceuticals
Agenus Bio
Agios Pharmaceuticals
Aguettant
AiBtl BioPharma
AIT Therapeutics
Akcea Therapeutics
Akebia Therapeutics
Albert Einstein College of Medicine
Alchemia
Alder Biopharmaceuticals
Alector
Alexion Pharmaceuticals
Alexza Pharmaceuticals
Alfasigma
Alfa Wassermann
Allergan
Allergan (name changed from Actavis)
Almirall
Alnylam Pharmaceuticals
Alora Pharmaceuticals
Alpha Biopharma
Alternavida
Altman Enterprises
AltrazealTrading
Altum Pharmaceuticals
Alvogen
Amarantus BioSciences
American Type Culture Collection (ATCC)
Amgen
Amherst Pharmaceuticals
Amorfix Life Sciences
Ampio Pharmaceuticals
Amryt Pharma
Amylin Pharmaceuticals
Amyris
AnaBios
AnaptysBio
AnGes MG
Angion Biomedica
AnnaMed
Antares Pharma
Antibe Therapeutics
Antigen Express
Applied Therapeutics
Apricus Biosciences
Aquestive Therapeutics
Aradigm
Aratana Therapeutics
Arcellx
Arch Pharmalabs
Arctic Vision
Arcturus Therapeutics
Arcus Biosciences
Ardelyx
Arena Pharmaceuticals
Arrowhead Pharmaceuticals
Artiva Biotherapeutics
Arvinas
Ascendis Pharma
Ascentage Pharma
Ashvattha Therapeutics
Assembly Biosciences
Astellas Pharma
Astellas Pharma Europe
Asterias Biotherapeutics
AstraZeneca
Athenex
Atossa Therapeutics
Atreca
Audeo Oncology
Augmenta
Aurigene Discovery Technologies
Aurinia Pharmaceuticals
Authentic Brands Group
AVEO Oncology
Avita Therapeutics
Avivagen
Axovant Gene Therapies
Axsome Therapeutics
Aytu BioPharma
Azaya Therapeutics
Azure Biotech
Baker Brothers Investments
BAKX Therapeutics
Baliopharm
BASF
Baxter International
Bayer Healthcare
Bayer Innovation
Baylor Research Institute
Beam Therapeutics
BeiGene
Beijing Defengrei Biotechnology
Belgian Volition
Bellerophon Therapeutics
Benitec Biopharma
Besins Healthcare
Beth Israel Deaconess Medical Center
BetterLife Pharma
Bharat Biotech
BIND Biosciences
BioCorRx
BioCryst Pharmaceuticals
Bioeq IP
Biogen
BioGene Technology
Biohaven Pharmaceuticals
BioInvent
BiolineRX
BioMarin Pharmaceutical
BioNovion
BioNTech
BioNxt Solutions
Biopharmex
BiopharmX
BioPorto
Bioprocess Capital Ventures
BioProspect
Biostem US
Biotech Investment
Biotechnology Value Fund
BioTime Acquisition
Bioton
Biovail
BioZone Laboratories
BioZone Pharmaceuticals
Blackstone
Bluebird Bio
Blueprint Medicines
BMV Medica
Boehringer Ingelheim
Bone Therapeutics
Braeburn Pharmaceuticals
BrainStorm Cell Therapeutics
Breathtec BioMedical
Brickell Biotech
BridgeBio Pharma
Bridge Biotherapeutics
Bristol-Myers Squibb
Caleco Pharma
Calico
Can-Fite BioPharma
Cancer Prevention Pharmaceuticals
Cannassure Therapeutics
CanniMed Therapeutics
Cann Pharmaceutical Australia
Canntab Therapeutics
Cannus Partners
Cantex Pharmaceuticals
Cara Therapeutics
Cardigant Medical
Cardium Therapeutics
CareFusion
Carna BioSciences
Cary Pharmaceuticals
CASI Pharmaceuticals
Castle Creek Biosciences
Catalent
CB2 Therapeutics
Celdara Medical
Celgene
Cell Cure Neurosciences
Cellvera
Cel Sci
Celyad
Cereal Ingredients
Cerebain Biotech
Cerus
CGA 369
cGreen
Chi-Med
Chiesi Farmaceutici
Chimerix
Chong Kun Dang
Chongqing Jingdong Pharmaceutical
Cimtech
Cipher Pharmaceuticals
Cipla
Circassia
Clal Biotech
Clalit Health Services
Clearmind Medicine
Clearside Biomedical
Clemson University
Cleveland BioLabs
Clinigen
CMIC
CNS Pharmaceuticals
Cocrystal Pharma
Coeptis Pharmaceuticals
CohBar
Cohen-McNiece Foundation
Coherus Biosciences
Colby Pharmaceuticals
Collegium Pharmaceuticals
Columbia Laboratories
Columbia University
Common Sense
Conatus Pharmaceuticals
Concile
Consilient Health
ContraFect
Coronado Biosciences
Cosmofix
Cosmo Pharmaceuticals
Coya Therapeutics
CRISPR Therapeutics
Crossject
Crucell
CSL
CTI BioPharma
CTT Pharmaceutical
Cullinan Oncology
Cumberland Pharmaceuticals
Cures Within Reach
CureTech
Curis
Currax Pharmaceuticals
CURx Pharmaceuticals
Cutanea Life Sciences
Cybin
CymaBay Therapeutics
Cypress Bioscience
Cytodyn
Cytokinetics
Cytori Therapeutics
Daewoong Pharmaceutical
Daiichi Sankyo
Dana-Farber Cancer Institute
Dance Biopharm
DAS Therapeutics
Defiante Farmaceutica
Denali Therapeutics
DepoMed
Derma Sciences
Dermavant Sciences
Dermin
Dianomi Therapeutics
Dicerna Pharmaceuticals
DNA Biomedical Solutions
Dr. Falk Pharma
Dr. Reddy's Laboratories
Dragonfly Therapeutics
DRI Capital
Drugs for Neglected Diseases Initiative
DS Healthcare
Duke University
Dyax
Eagle Pharmaceuticals
Eagle Pharmaceuticals (US)
Easton Pharmaceuticals
Echo Therapeutics
Ecology Coatings
Eczacibasi Pharmaceuticals
Eddingpharm
Egalet
Eisai
Elencee
Eli Lilly
Elite Pharmaceuticals
Emergent BioSolutions
Emory University
Enalare Therapeutics
Endocyte
Endo Pharmaceuticals
EnGeneIC
Enochian Biosciences
Entera Bio
Entest BioMedical
EnWave
Epizyme
Epocal
Esperion Therapeutics
Eternity Healthcare
Eton Pharmaceuticals
Eureka Genomics
EUSA Pharma
Evolva
Ewopharma
Exact Sciences
Exelixis
ExScientia
Eyevance
Fairview Health System
FasTrack Pharmaceuticals
Ferozsons Laboratories
Ferring Pharmaceuticals
Fibrocell Science
Foghorn Therapeutics
Forest Laboratories
Fosun Pharmaceutical
Fox Chase Chemical Diversity Center
Fresenius Kabi Pharmaceuticals
FSD Pharma
Fusion Pharmaceuticals
FusionVax
Galapagos
Galmed Pharmaceuticals
GE Healthcare
Genentech
Generex Biotechnology
Genevant Sciences
Genexine
Genfit
Genmab
Gentium
Genzyme
Geron
Gilead Sciences
Glaukos
Glenmark Generics Inc USA
Glenmark Pharmaceuticals
Goldfinch Bio
Government of Israel
GPCR Therapeutics
Gravocore
Grifols
GSK
GT Biopharma
Hai Kang Life
Hail First Pharma
Hainan Sihuan Pharmaceutical
Halo Labs
Halozyme Therapeutics
Hansen Medical
Health Canada
Healthcare Royalty Partners
Health Discovery
Heartseed
Heat Biologics
Hefei Life Science
Hefei Tianhui Incubation of Technologies
HekaBio
Helsinn Therapeutics
HemoCleanse
Hemogenyx
HemPup
Henlius Biotech
Hetero Labs
HIBM Research Group
Hikma Pharmaceuticals
Hoffmann La Roche
Hollister Biosciences
Hookipa Pharma
Horizon Pharma Inc
Huadong Medicine
HyperGenomics
I-mab
Icahn School of Medicine at Mount Sinai
Ice + Jam
Iconovo
IDC-GP Pharm
IDEAYA Biosciences
Idera Pharmaceuticals
Idorsia
Idun Pharmaceuticals
IGM Biosciences
iMetabolic Biopharma
Immugenyx
Immunic
ImmunityBio
Immunomedics
Immunomic Therapeutics
Immunovative Therapies
ImmVira
Impax Laboratories
Imprimis Pharmaceuticals
In4Derm
iNAP
Incannex Healthcare
Incuron
Incyte
Indiana University
Infinity Pharmaceuticals
InflaRx
Inhibitor Therapeutics
Innate Pharma
Innocoll
Innovate Biopharmaceuticals
Innovation Pharmaceuticals
Innovent Biologics
Innovet
Innovus Pharmaceuticals
INO Therapeutics
Inspiration Biopharmaceuticals
Instrumentation Laboratory
IntelGenx
Intellia Therapeutics
IntelliCell BioSciences
Intellikine
Internet Marketing Consortium
Intrexon
Intuitive Surgical
Invekra
Inverness Medical Innovations
Ionis Pharmaceuticals
Ipsen
Iroko Pharmaceuticals
Ironwood Pharmaceuticals
Israeli National Authority for Technological Innovation
Itrom Pharmaceutical Group
Ivantis
Jaguar Health
Janssen Biotech
Janssen Pharmaceuticals
Jazz Pharmaceuticals
JFC Technologies
Jiangsu Alphamab
Jiangsu Lianhuan Pharmaceuticals
Jiangsu NHWA Pharmaceutical
Ji Xing Pharmaceuticals
John Morley Foods
Joincare Pharmaceutical
Journey Medical
Jubilant Life Sciences
Jumpcan Pharmaceutical
Juniper Pharmaceuticals
Junshi Biosciences
Jupiter Wellness
Juventas Therapeutics
K2M
Kadmon Pharmaceuticals
Kaken Pharmaceutical
Kalbe Genexine Biologics
KaloBios Pharmaceuticals
Karveer Meditech
KemPharm
Kensey Nash
Kieran Murphy
Kinetic Concepts
Kiniksa Pharmaceuticals
Kintor
Kiromic Biopharma
Kite Pharma
Kitov Pharma
Klaria
Klinge Biopharma
Knighton Foods
Knight Therapeutics
Kodiak Sciences
Kowa
Kraig Biocraft Laboratories
KVK-Tech
Kwang Dong Pharmaceutical
Kymera Therapeutics
Kyowa Hakko Kirin
Kyverna Therapeutics
L1 Systems
Laboratories Acbel
Lantheus Holding
Launch Therapeutics
Lee Moffitt Cancer Center
Legend Biotech
LegoChem Biosciences
Level Brands
LFB Biotechnologies
Lifestyle Delivery Systems
Ligand Pharmaceuticals
Lineage Cell Therapeutics
Link Health
Lipidor
Lipocine
Lixte Biotechnology
Lorna Linda University
Lotus Pharmaceuticals
Loxo Oncology
Lubrizol
Lumenis
Lundbeck
Lung Therapeutics
MabCure
Macrogenics
Magenta Therapeutics
MainPointe
Mallinckrodt Pharmaceuticals
Marvanal
Marv Enterprises
Massachusetts General Hospital
Matrix Biomed
Mayne Pharma
McComb Foundation
McEwen Centre for Regenerative Medicine
Meda
Medica
Medica Avante
Medical Technologies Innovation Asia
Medice Arzneimittel Putter
Medicines Patent Pool Foundation
Medicox
MediGene
Medigus
MedImmune
Medison Pharma
Medivation
Medivir
Medovex
MEI Pharma
Melmed
Memory Dx
Mercer University
Merck and Co
Merck KGaA
Merck Serono
Merus
Methodist Hospital Houston
Micromet
Mikah Pharma
Millennium
Mimi's Rock
Mindset Pharma
Ministry of Health
Labor and Welfare
MiNK Therapeutics
Miraculins
Miralogx
Mira Pharmaceuticals
Miromatrix Medical
Mitsubishi Tanabe Pharma
Moderna
Modis Therapeutics
Moleculin Biotech
Mologen
Momenta Pharmaceuticals
Monash University
Monopar Therapeutics
Morgan Stanley
MorphoSys
Mount Sinai Health System
MultiCell Immunotherapeutics
Munich Leukemia Laboratory
Mylan Laboratories
Mylan Pharmaceuticals
Myovant Sciences
NanoVibronix
NanoViricides
NantCell
NantKwest
Nantong Jinghua Pharmaceutical
NantWorks
Napo Pharmaceuticals
Natac Biotech
Natco Pharmaceuticals
National Green Biomed
Nektar Therapeutics
Neogenomics
Neotope
Nephrogenex
Nephros
Nestle Health Science
Neuraxpharm
Neurocrine Biosciences
NeuroRx
NeuroSigma
NeuroVive Pharmaceutical
NeuroVive Pharmaceutical Asia
NewLeaf Brands
Newsoara Biopharma
New York University
NexMed
NextCure
Nextleaf Solutions
Nexvet
NGM Biopharmaceuticals
Nicox
Nkarta Therapeutics
NLS Pharmaceutics
Nordic Bioscience
Norgine
NorthStar Medical Radioisotopes
Notable Labs
Novadaq Technologies
NovaDel Pharma
Novan
Novan Therapeutics
Novartis
Novartis Vaccines and Diagnostics
Novocure
Novo Nordisk
Novus Biologicals
NRx Pharmaceuticals
Nucorion
Numab
Nunzi Pharmaceutical
Nurix Therapeutics
Nutech Medical
NutraOrigin
NXTGEN
Nymox Pharmaceutical
Oberland Capital
Ocugen
Oculus Innovative Sciences
Ocumension Therapeutics
Omeros
Oncologie
Oncolytics Biotech
OncoMed Pharmaceuticals
Onconova Therapeutics
Onegevity
One Way Liver Genomics (OWL)
ONK Therapeutics
Ono Pharmaceutical
Onxeo
Onyx Pharmaceuticals
Opexa Therapeutics
Opthea
OptiBiotix
OptiNose
Oragenics
Oramed Pharmaceuticals
Orexigen Therapeutics
Organovo
Orgenesis
Orion
Orox Pharmaceuticals
OrthoCyte
Otsuka
OvaMed
Ovid Therapeutics
Oxford BioMedica
Oxis Biotech
Oxygen Biotherapeutics
OxySure Systems
Oyster Point Pharma
Pan-Provincial Vaccine Enterprise
Parnell Pharmaceuticals
Par Pharmaceutical
PDL BioPharma
PDS Biotechnology
Penwest Pharmaceuticals
Pernix Therapeutics
Perugia University
Pfenex
Pfizer
Pharmacyclics
PharmaCyte Biotech
PharmaMar
PharmaNova
Pharmastrip
Philogen
Phosphagenics
Photocure ASA
Phusion Laboratoies
Phyto Nutricare
Pipex Pharmaceuticals
Plandai Biotechnology
Pluristem Therapeutics
POINT Biopharma
Portola Pharmaceuticals
Poseida Therapeutics
Pozen
PreMD
Premier Biomedical
Professional Compounding Centers of America
Progenics Pharmaceuticals
Prometic Life Sciences
ProQR Therapeutics
Prosidion
Protagonist Therapeutics
Protalix BioTherapeutics
Proteonomix
Prothena
ProtoKinetix
Pulmatrix
Puma Biotechnology
QED Therapeutics
QRxPharma
Quick Med Technologies
Quigley Corporation
Quoin Pharmaceuticals
Rayner Surgical
Reata Pharmaceuticals
Recordati
Recro Pharma
RedHill Biopharma
Reedy Creek Investments
RegenaStem
Regeneron Pharmaceuticals
Regenxbio
Regnum
Relay Therapeutics
Relief Therapeutics
Relmada Therapeutics
RenalytixAI
ReNeuron
Repare Therapeutics
Replicel Life Sciences
Repligen
Reprise Biomedical
Resarci Therapeutics
Retrophin
rEVO Biologics
Revolution Medicines
Rigel Pharmaceuticals
RNL Bio
Roche
Rockefeller University
Roivant Sciences
Royalty Pharma
RST Biomedikal Sanayi
Ruthigen
RXi Pharmaceuticals
S2 Therapeutics
SACCO
Sagard Healthcare Royalty Partners
Sage Therapeutics
Salix Pharmaceuticals
Sam Amer
Sanara MedTech
Sandoz
Sanofi
Sanofi-Aventis
Sanofi-Pasteur
Sanpellegrino Cosmetics
Santarus
Santen Pharmaceutical
Sanuwave Health
SA Pathology
Sapientia Pharmaceuticals
Sarepta Therapeutics
Schrodinger
Scopus BioPharma
Seagen
Seattle Genetics
Seelos Therapeutics
Sekris Biomedical
Senetek
Senior Paradise
Sensory Cloud
Seqirus
Seres Therapeutics
Sermonix Pharmaceutical
Sesen Bio
SevenScore Pharmaceuticals
Shanghai Pharmaceuticals Holdings
Sheba Medical Center
Shenzhen BioScien Pharmaceuticals
Shield Therapeutics
Shionogi
Shire Pharmaceuticals
Shore Therapeutics
Siemens Healthcare Diagnostics
Sigma-Tau
Sinclair Pharmaceuticals
Sinobiopharma
Sinopharm
Sio Gene Therapies
Skinvera
Skyline Diagnostics
Solarvest BioEnergy
Soliance
Soligenix
Sonoma BioTherapeutics
Sorrento Therapeutics
Spectranetics
Spencer Pharmaceutical
Spinal USA
St. Jude Children's Research Hospital
STADA Arzneimittel
Staidson (Beijing) BioPharmaceuticals
Stanford University
Stealth BioTherapeutics
Stellar Biotechnologies
St Jude Medical
Stoke Therapeutics
Strongbridge Biopharma
Sucampo AG
SUDA Pharmaceuticals
Sumitomo Dainippon Pharma
Sunesis Pharmaceuticals
Sunovion Pharmaceuticals
Sun Pharmaceutical
SuperNova Diagnostics
Sutro Biopharma
Swedish Orphan Biovitrum
SymBio Pharmaceuticals
Symbollon
Syndax Pharmaceuticals
Synergy Strips
Syntopix
Tactical Relief
Taiba
TaiGen Biotechnology
Taiho
Takeda Pharmaceutical
Talaris Therapeutics
Tango Therapeutics
Target Group
Tauriga Sciences
TB Alliance
TearLab
Technion Research and Development Foundation
Teijin
Telomir Pharmaceuticals
Tesaro
Tetra Bio-Pharma
TetraLogic Pharmaceuticals
Teva Pharmaceutical Industries
Texas A&M University
Texas Heart Institute
TFF Pharmaceuticals
TG Therapeutics
Therakine
TherapeuticsMD
Theravance
Theravance Biopharma
The Regents of the University of California
Thorne Research
Threshold Pharmaceuticals
TiGenix
Tilray
Titterington Veterinary Services
Tiziana Life Sciences
Tocagen
TONIX Pharmaceuticals
Torii Pharmaceutical
TRACON Pharmaceuticals
Tramorgan
Transgene
Travere Therapeutics
Trellis Bioscience
Trevena
Trimedyne
Trimeris
TrovaGene
TTFactor
Tufts University
Turnstone Biologics
Tyme Technologies
Ultragenyx Pharmaceuticals
Uluru
Unigene Laboratories
UniQure
Universal Cells
University College Dublin
University Health Network
University of Alberta
University of California
University of Central Florida Research Foundation
University of Colorado
University of Florida Research Foundation
University of Geneva
University of Louisville
University of Missouri
University of Notre of Dame
University of Pennsylvania
University of Texas
University of Virginia
University of Western Sydney
University of Zurich
UOP
Uppsalagruppen Medical
Upstream Peripheral Technologies
UroGen Pharma
Valeant Pharmaceuticals
ValiPharma
Valneva
Valor Biotherapeutics
Vanderbilt University
Vasomune Therapeutics
Vaxent
VBL Therapeutics
Veloxis Pharmaceuticals
Veracyte
Verenium
Verity Pharmaceuticals
VeroScience
Vertex Pharmaceuticals
Vida Concepts
Vifor Pharma
ViiV Healthcare
Virbac
Viropharma
VistaGen Therapeutics
Vividion Therapeutics
Vivus
VolitionRX
Voronoi
Voyager Therapeutics
Vyera Pharmaceuticals
VYNE Therapeutics
Vyrix Pharmaceuticals
Watson Pharmaceuticals
WAVE Life Sciences
Wigen Biomedicine
Wisconsin Alumni Research Foundation
World Class Extractions
WPD Pharmaceuticals
WUGEN
Xbrane Bioscience
Xencor
Xlife Sciences
XPhyto Therapeutics
XTL Biopharmaceuticals
Xynomic Pharmaceuticals
Yale University
Yeda Research and Development Company
Yeugnam University
Yissum Research Development
ZAI Laboratory
Zalicus
Zenitech
Zenith Epigenetics
Ziopharm Oncology
Zogenix
ZS Pharma
Zymeworks
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SOURCE Research and Markets
VaccineAcquisition
13 Mar 2024
DUBLIN--(
BUSINESS WIRE
)--The
"Royalty Rates in Pharmaceutical and Biotechnology Deals"
report has been added to
ResearchAndMarkets.com's
offering.
Royalty Rates in Pharmaceutical and Biotechnology Deals
is your one-stop source for providing real-deal information on hundreds of transactions, including the technology licensed, royalty rates, license fees, upfront and milestone payments.
This report provides details of the latest partnering deals which disclose a royalty rate, announced in the pharmaceutical, biotechnology and diagnostic sectors. The report provides details of partnering deals disclosing royalty rates from 2010 to 2024.
The report provides an overview of how and why companies enter partnering deals where a royalty is payable upon commercialization of the compound or technology.
Understanding the flexibility of a prospective partner's negotiated deals terms provides critical insight into the negotiation process in terms of what you can expect to achieve during the negotiation of terms. Whilst many smaller companies will be seeking details of the payments clauses, the devil is in the detail in terms of how payments are triggered - contract documents provide this insight where press releases and databases do not.
This report contains a comprehensive listing of all partnering deals announced since 2010 where a quantitative royalty rate has been disclosed, as recorded in the Current Agreements deals and alliances database. Each deal record and royalty disclosure is available in further detail via a link to online copy of the deal including actual contract document, where available, as submitted to the Securities Exchange Commission by companies and their partners.
The initial chapters of this report provide an orientation of royalty rate trends in pharma and biotech since 2010. Chapter 1 provides an introduction to the report, whilst chapter 2 provides an overview of the trends in royalty rates in the pharma and biotech sectors since 2010.
Chapter 3 provides a more detailed insight into the structure of a royalty rate clause and how it fits with the other financial terms of the partnering deal.
Chapter 4 provides an insight into companies active in disclosing royalty rates as well as those deals attracting the highest royalty rates.
Chapter 5 provides comprehensive and detailed access to deals which disclose a royalty rate since 2010 where a contract document is available. Contract documents provide an in-depth insight into the actual deal terms agreed between the parties with respect to the royalty rate.
In addition the report includes a comprehensive deal directory listing of all deals where a royalty rate has been disclosed announced since 2010. Each listing is organized as a directory by company A-Z, stage of development at signing, therapeutic area, and technology type. Each deal title links via hyperlink to an online version of the deal record including, where available, the actual contract document.
The report also includes numerous table and figures that illustrate the trends in royalty rates in pharma and biotech deal making since 2010. In conclusion, this report provides everything a prospective dealmaker needs to know about royalty rates in the pharma and biotech sector.
Royalty Rates in Pharmaceutical and Biotechnology Deals provides the following benefits:
Understand royalty rate trends since 2010
Analysis of the structure of royalty clauses with real life case studies
Browse collaboration and licensing deals which reveal a royalty rate
Benchmark analysis - identify market value of transactions
Financials terms - upfront, milestone, royalties
Directory of deals by company A-Z, therapy focus and technology type
Access contract documents - insights into deal structures
Due diligence - assess suitability of your proposed deal terms for partner companies
Save hundreds of hours of research time
Analyzing actual contract agreements allows assessment of the following:
What are the precise royalty rates granted?
What is actually granted by the agreement to the partner company?
What exclusivity is granted?
What is the payment structure for the deal?
How are sales and payments audited?
What is the deal term?
How are the key terms of the agreement defined?
How are IPRs handled and owned?
Who is responsible for commercialization?
Who is responsible for development, supply, and manufacture?
How is confidentiality and publication managed?
How are disputes to be resolved?
Under what conditions can the deal be terminated?
What happens when there is a change of ownership?
What sublicensing and subcontracting provisions have been agreed?
Which boilerplate clauses does the company insist upon?
Which boilerplate clauses appear to differ from partner to partner or deal type to deal type?
Which jurisdiction does the company insist upon for agreement law?
Key Topics Covered:
Executive Summary
Chapter 1 - Introduction
1.1. What are royalties?
1.2. History of royalty rates
1.3. Royalties in pharmaceuticals and biotechnology
1.4. Royalties versus revenue share
1.5. Overview of the report
Chapter 2 - An overview of pharmaceutical and biotechnology royalty rates
2.1. Trends in royalty rates 2010 - 2024
2.2. How do revenue shares figure?
2.3. A review of recent literature
2.4. Royalty rates in the future
Chapter 3 - The royalty clause in pharmaceutical and biotechnology deals
3.1. Partnering agreement structure
3.2. Structure of a typical royalty clause
3.3. Example royalty clauses
3.3.1. Case study 1
3.3.2. Case study 2
3.3.3. Case study 3
3.3.4. Case study 4
Chapter 4 - Companies actively disclosing royalty rates
4.1. Most active companies in disclosing royalty rates
Chapter 5 - Royalty rate contract directory
Explore royalty rates within the deal contract document to gain greater insight
Deal directory
Deal Directory - Royalty rates in deals - by company A-Z
Deal Directory - Royalty rates in deals - by therapy area
Deal Directory - Royalty rates in deals - by stage of development at signing
Deal Directory - Royalty rates in deals - by technology type
Royalty rate references
Example royalty rate deal contract document
List of Figures
Figure 1: Trends in pharma and biotech royalty rates, 2010 - 2024
Figure 2: Components of the partnering deal structure
Figure 3: Components of the partnering royalty clause
Figure 4: Most active companies disclosing royalty rate 2010 - 2024
Figure 5: Licensing agreement for rhAAT - rEVO Biologics, LFB Biotechnologies
Companies Mentioned
2seventy bio
3a-diagnostics
3D Medicines
4SC
23andMe
Abbott Laboratories
Abbvie
Abeona Therapeutics
Ablynx
Abraxis BioScience
ABVC BioPharma
ACADIA Pharmaceuticals
Acceleron Pharma
Acelrx Pharmaceuticals
Acer Therapeutics
Acerus
Acrotech Biopharma
Actavis (acquired by Watson)
Active Biomaterials
Acura Pharmaceuticals
Adamis Pharmaceuticals
Adaptimmune
ADC Therapeutics
Adeona Pharmaceuticals
Adial Pharmaceuticals
Aditx Therapeutics
Adlai Nortye
Aduro BioTech
Advanced Dosage Forms
Advanz Pharma
Advaxis
Advirna
Aegerion Pharmaceutical
Aeglea BioTherapeutics
Aerie Pharmaceuticals
AEterna Zentaris
Aexon Labs
Affimed Therapeutics
Afritex Medical Products
AFT Pharmaceuticals
Agenus Bio
Agios Pharmaceuticals
Aguettant
AiBtl BioPharma
AIT Therapeutics
Akcea Therapeutics
Akebia Therapeutics
Albert Einstein College of Medicine
Alchemia
Alder Biopharmaceuticals
Alector
Alexion Pharmaceuticals
Alexza Pharmaceuticals
Alfasigma
Alfa Wassermann
Allergan
Allergan (name changed from Actavis)
Almirall
Alnylam Pharmaceuticals
Alora Pharmaceuticals
Alpha Biopharma
Alternavida
Altman Enterprises
AltrazealTrading
Altum Pharmaceuticals
Alvogen
Amarantus BioSciences
American Type Culture Collection (ATCC)
Amgen
Amherst Pharmaceuticals
Amorfix Life Sciences
Ampio Pharmaceuticals
Amryt Pharma
Amylin Pharmaceuticals
Amyris
AnaBios
AnaptysBio
AnGes MG
Angion Biomedica
AnnaMed
Antares Pharma
Antibe Therapeutics
Antigen Express
Applied Therapeutics
Apricus Biosciences
Aquestive Therapeutics
Aradigm
Aratana Therapeutics
Arcellx
Arch Pharmalabs
Arctic Vision
Arcturus Therapeutics
Arcus Biosciences
Ardelyx
Arena Pharmaceuticals
Arrowhead Pharmaceuticals
Artiva Biotherapeutics
Arvinas
Ascendis Pharma
Ascentage Pharma
Ashvattha Therapeutics
Assembly Biosciences
Astellas Pharma
Astellas Pharma Europe
Asterias Biotherapeutics
AstraZeneca
Athenex
Atossa Therapeutics
Atreca
Audeo Oncology
Augmenta
Aurigene Discovery Technologies
Aurinia Pharmaceuticals
Authentic Brands Group
AVEO Oncology
Avita Therapeutics
Avivagen
Axovant Gene Therapies
Axsome Therapeutics
Aytu BioPharma
Azaya Therapeutics
Azure Biotech
Baker Brothers Investments
BAKX Therapeutics
Baliopharm
BASF
Baxter International
Bayer Healthcare
Bayer Innovation
Baylor Research Institute
Beam Therapeutics
BeiGene
Beijing Defengrei Biotechnology
Belgian Volition
Bellerophon Therapeutics
Benitec Biopharma
Besins Healthcare
Beth Israel Deaconess Medical Center
BetterLife Pharma
Bharat Biotech
BIND Biosciences
BioCorRx
BioCryst Pharmaceuticals
Bioeq IP
Biogen
BioGene Technology
Biohaven Pharmaceuticals
BioInvent
BiolineRX
BioMarin Pharmaceutical
BioNovion
BioNTech
BioNxt Solutions
Biopharmex
BiopharmX
BioPorto
Bioprocess Capital Ventures
BioProspect
Biostem US
Biotech Investment
Biotechnology Value Fund
BioTime Acquisition
Bioton
Biovail
BioZone Laboratories
BioZone Pharmaceuticals
Blackstone
Bluebird Bio
Blueprint Medicines
BMV Medica
Boehringer Ingelheim
Bone Therapeutics
Braeburn Pharmaceuticals
BrainStorm Cell Therapeutics
Breathtec BioMedical
Brickell Biotech
BridgeBio Pharma
Bridge Biotherapeutics
Bristol-Myers Squibb
Caleco Pharma
Calico
Can-Fite BioPharma
Cancer Prevention Pharmaceuticals
Cannassure Therapeutics
CanniMed Therapeutics
Cann Pharmaceutical Australia
Canntab Therapeutics
Cannus Partners
Cantex Pharmaceuticals
Cara Therapeutics
Cardigant Medical
Cardium Therapeutics
CareFusion
Carna BioSciences
Cary Pharmaceuticals
CASI Pharmaceuticals
Castle Creek Biosciences
Catalent
CB2 Therapeutics
Celdara Medical
Celgene
Cell Cure Neurosciences
Cellvera
Cel Sci
Celyad
Cereal Ingredients
Cerebain Biotech
Cerus
CGA 369
cGreen
Chi-Med
Chiesi Farmaceutici
Chimerix
Chong Kun Dang
Chongqing Jingdong Pharmaceutical
Cimtech
Cipher Pharmaceuticals
Cipla
Circassia
Clal Biotech
Clalit Health Services
Clearmind Medicine
Clearside Biomedical
Clemson University
Cleveland BioLabs
Clinigen
CMIC
CNS Pharmaceuticals
Cocrystal Pharma
Coeptis Pharmaceuticals
CohBar
Cohen-McNiece Foundation
Coherus Biosciences
Colby Pharmaceuticals
Collegium Pharmaceuticals
Columbia Laboratories
Columbia University
Common Sense
Conatus Pharmaceuticals
Concile
Consilient Health
ContraFect
Coronado Biosciences
Cosmofix
Cosmo Pharmaceuticals
Coya Therapeutics
CRISPR Therapeutics
Crossject
Crucell
CSL
CTI BioPharma
CTT Pharmaceutical
Cullinan Oncology
Cumberland Pharmaceuticals
Cures Within Reach
CureTech
Curis
Currax Pharmaceuticals
CURx Pharmaceuticals
Cutanea Life Sciences
Cybin
CymaBay Therapeutics
Cypress Bioscience
Cytodyn
Cytokinetics
Cytori Therapeutics
Daewoong Pharmaceutical
Daiichi Sankyo
Dana-Farber Cancer Institute
Dance Biopharm
DAS Therapeutics
Defiante Farmaceutica
Denali Therapeutics
DepoMed
Derma Sciences
Dermavant Sciences
Dermin
Dianomi Therapeutics
Dicerna Pharmaceuticals
DNA Biomedical Solutions
Dr. Falk Pharma
Dr. Reddy's Laboratories
Dragonfly Therapeutics
DRI Capital
Drugs for Neglected Diseases Initiative
DS Healthcare
Duke University
Dyax
Eagle Pharmaceuticals
Eagle Pharmaceuticals (US)
Easton Pharmaceuticals
Echo Therapeutics
Ecology Coatings
Eczacibasi Pharmaceuticals
Eddingpharm
Egalet
Eisai
Elencee
Eli Lilly
Elite Pharmaceuticals
Emergent BioSolutions
Emory University
Enalare Therapeutics
Endocyte
Endo Pharmaceuticals
EnGeneIC
Enochian Biosciences
Entera Bio
Entest BioMedical
EnWave
Epizyme
Epocal
Esperion Therapeutics
Eternity Healthcare
Eton Pharmaceuticals
Eureka Genomics
EUSA Pharma
Evolva
Ewopharma
Exact Sciences
Exelixis
ExScientia
Eyevance
Fairview Health System
FasTrack Pharmaceuticals
Ferozsons Laboratories
Ferring Pharmaceuticals
Fibrocell Science
Foghorn Therapeutics
Forest Laboratories
Fosun Pharmaceutical
Fox Chase Chemical Diversity Center
Fresenius Kabi Pharmaceuticals
FSD Pharma
Fusion Pharmaceuticals
FusionVax
Galapagos
Galmed Pharmaceuticals
GE Healthcare
Genentech
Generex Biotechnology
Genevant Sciences
Genexine
Genfit
Genmab
Gentium
Genzyme
Geron
Gilead Sciences
Glaukos
Glenmark Generics Inc USA
Glenmark Pharmaceuticals
Goldfinch Bio
Government of Israel
GPCR Therapeutics
Gravocore
Grifols
GSK
GT Biopharma
Hai Kang Life
Hail First Pharma
Hainan Sihuan Pharmaceutical
Halo Labs
Halozyme Therapeutics
Hansen Medical
Health Canada
Healthcare Royalty Partners
Health Discovery
Heartseed
Heat Biologics
Hefei Life Science
Hefei Tianhui Incubation of Technologies
HekaBio
Helsinn Therapeutics
HemoCleanse
Hemogenyx
HemPup
Henlius Biotech
Hetero Labs
HIBM Research Group
Hikma Pharmaceuticals
Hoffmann La Roche
Hollister Biosciences
Hookipa Pharma
Horizon Pharma Inc
Huadong Medicine
HyperGenomics
I-mab
Icahn School of Medicine at Mount Sinai
Ice + Jam
Iconovo
IDC-GP Pharm
IDEAYA Biosciences
Idera Pharmaceuticals
Idorsia
Idun Pharmaceuticals
IGM Biosciences
iMetabolic Biopharma
Immugenyx
Immunic
ImmunityBio
Immunomedics
Immunomic Therapeutics
Immunovative Therapies
ImmVira
Impax Laboratories
Imprimis Pharmaceuticals
In4Derm
iNAP
Incannex Healthcare
Incuron
Incyte
Indiana University
Infinity Pharmaceuticals
InflaRx
Inhibitor Therapeutics
Innate Pharma
Innocoll
Innovate Biopharmaceuticals
Innovation Pharmaceuticals
Innovent Biologics
Innovet
Innovus Pharmaceuticals
INO Therapeutics
Inspiration Biopharmaceuticals
Instrumentation Laboratory
IntelGenx
Intellia Therapeutics
IntelliCell BioSciences
Intellikine
Internet Marketing Consortium
Intrexon
Intuitive Surgical
Invekra
Inverness Medical Innovations
Ionis Pharmaceuticals
Ipsen
Iroko Pharmaceuticals
Ironwood Pharmaceuticals
Israeli National Authority for Technological Innovation
Itrom Pharmaceutical Group
Ivantis
Jaguar Health
Janssen Biotech
Janssen Pharmaceuticals
Jazz Pharmaceuticals
JFC Technologies
Jiangsu Alphamab
Jiangsu Lianhuan Pharmaceuticals
Jiangsu NHWA Pharmaceutical
Ji Xing Pharmaceuticals
John Morley Foods
Joincare Pharmaceutical
Journey Medical
Jubilant Life Sciences
Jumpcan Pharmaceutical
Juniper Pharmaceuticals
Junshi Biosciences
Jupiter Wellness
Juventas Therapeutics
K2M
Kadmon Pharmaceuticals
Kaken Pharmaceutical
Kalbe Genexine Biologics
KaloBios Pharmaceuticals
Karveer Meditech
KemPharm
Kensey Nash
Kieran Murphy
Kinetic Concepts
Kiniksa Pharmaceuticals
Kintor
Kiromic Biopharma
Kite Pharma
Kitov Pharma
Klaria
Klinge Biopharma
Knighton Foods
Knight Therapeutics
Kodiak Sciences
Kowa
Kraig Biocraft Laboratories
KVK-Tech
Kwang Dong Pharmaceutical
Kymera Therapeutics
Kyowa Hakko Kirin
Kyverna Therapeutics
L1 Systems
Laboratories Acbel
Lantheus Holding
Launch Therapeutics
Lee Moffitt Cancer Center
Legend Biotech
LegoChem Biosciences
Level Brands
LFB Biotechnologies
Lifestyle Delivery Systems
Ligand Pharmaceuticals
Lineage Cell Therapeutics
Link Health
Lipidor
Lipocine
Lixte Biotechnology
Lorna Linda University
Lotus Pharmaceuticals
Loxo Oncology
Lubrizol
Lumenis
Lundbeck
Lung Therapeutics
MabCure
Macrogenics
Magenta Therapeutics
MainPointe
Mallinckrodt Pharmaceuticals
Marvanal
Marv Enterprises
Massachusetts General Hospital
Matrix Biomed
Mayne Pharma
McComb Foundation
McEwen Centre for Regenerative Medicine
Meda
Medica
Medica Avante
Medical Technologies Innovation Asia
Medice Arzneimittel Putter
Medicines Patent Pool Foundation
Medicox
MediGene
Medigus
MedImmune
Medison Pharma
Medivation
Medivir
Medovex
MEI Pharma
Melmed
Memory Dx
Mercer University
Merck and Co
Merck KGaA
Merck Serono
Merus
Methodist Hospital Houston
Micromet
Mikah Pharma
Millennium
Mimi's Rock
Mindset Pharma
Ministry of Health
Labor and Welfare
MiNK Therapeutics
Miraculins
Miralogx
Mira Pharmaceuticals
Miromatrix Medical
Mitsubishi Tanabe Pharma
Moderna
Modis Therapeutics
Moleculin Biotech
Mologen
Momenta Pharmaceuticals
Monash University
Monopar Therapeutics
Morgan Stanley
MorphoSys
Mount Sinai Health System
MultiCell Immunotherapeutics
Munich Leukemia Laboratory
Mylan Laboratories
Mylan Pharmaceuticals
Myovant Sciences
NanoVibronix
NanoViricides
NantCell
NantKwest
Nantong Jinghua Pharmaceutical
NantWorks
Napo Pharmaceuticals
Natac Biotech
Natco Pharmaceuticals
National Green Biomed
Nektar Therapeutics
Neogenomics
Neotope
Nephrogenex
Nephros
Nestle Health Science
Neuraxpharm
Neurocrine Biosciences
NeuroRx
NeuroSigma
NeuroVive Pharmaceutical
NeuroVive Pharmaceutical Asia
NewLeaf Brands
Newsoara Biopharma
New York University
NexMed
NextCure
Nextleaf Solutions
Nexvet
NGM Biopharmaceuticals
Nicox
Nkarta Therapeutics
NLS Pharmaceutics
Nordic Bioscience
Norgine
NorthStar Medical Radioisotopes
Notable Labs
Novadaq Technologies
NovaDel Pharma
Novan
Novan Therapeutics
Novartis
Novartis Vaccines and Diagnostics
Novocure
Novo Nordisk
Novus Biologicals
NRx Pharmaceuticals
Nucorion
Numab
Nunzi Pharmaceutical
Nurix Therapeutics
Nutech Medical
NutraOrigin
NXTGEN
Nymox Pharmaceutical
Oberland Capital
Ocugen
Oculus Innovative Sciences
Ocumension Therapeutics
Omeros
Oncologie
Oncolytics Biotech
OncoMed Pharmaceuticals
Onconova Therapeutics
Onegevity
One Way Liver Genomics (OWL)
ONK Therapeutics
Ono Pharmaceutical
Onxeo
Onyx Pharmaceuticals
Opexa Therapeutics
Opthea
OptiBiotix
OptiNose
Oragenics
Oramed Pharmaceuticals
Orexigen Therapeutics
Organovo
Orgenesis
Orion
Orox Pharmaceuticals
OrthoCyte
Otsuka
OvaMed
Ovid Therapeutics
Oxford BioMedica
Oxis Biotech
Oxygen Biotherapeutics
OxySure Systems
Oyster Point Pharma
Pan-Provincial Vaccine Enterprise
Parnell Pharmaceuticals
Par Pharmaceutical
PDL BioPharma
PDS Biotechnology
Penwest Pharmaceuticals
Pernix Therapeutics
Perugia University
Pfenex
Pfizer
Pharmacyclics
PharmaCyte Biotech
PharmaMar
PharmaNova
Pharmastrip
Philogen
Phosphagenics
Photocure ASA
Phusion Laboratoies
Phyto Nutricare
Pipex Pharmaceuticals
Plandai Biotechnology
Pluristem Therapeutics
POINT Biopharma
Portola Pharmaceuticals
Poseida Therapeutics
Pozen
PreMD
Premier Biomedical
Professional Compounding Centers of America
Progenics Pharmaceuticals
Prometic Life Sciences
ProQR Therapeutics
Prosidion
Protagonist Therapeutics
Protalix BioTherapeutics
Proteonomix
Prothena
ProtoKinetix
Pulmatrix
Puma Biotechnology
QED Therapeutics
QRxPharma
Quick Med Technologies
Quigley Corporation
Quoin Pharmaceuticals
Rayner Surgical
Reata Pharmaceuticals
Recordati
Recro Pharma
RedHill Biopharma
Reedy Creek Investments
RegenaStem
Regeneron Pharmaceuticals
Regenxbio
Regnum
Relay Therapeutics
Relief Therapeutics
Relmada Therapeutics
RenalytixAI
ReNeuron
Repare Therapeutics
Replicel Life Sciences
Repligen
Reprise Biomedical
Resarci Therapeutics
Retrophin
rEVO Biologics
Revolution Medicines
Rigel Pharmaceuticals
RNL Bio
Roche
Rockefeller University
Roivant Sciences
Royalty Pharma
RST Biomedikal Sanayi
Ruthigen
RXi Pharmaceuticals
S2 Therapeutics
SACCO
Sagard Healthcare Royalty Partners
Sage Therapeutics
Salix Pharmaceuticals
Sam Amer
Sanara MedTech
Sandoz
Sanofi
Sanofi-Aventis
Sanofi-Pasteur
Sanpellegrino Cosmetics
Santarus
Santen Pharmaceutical
Sanuwave Health
SA Pathology
Sapientia Pharmaceuticals
Sarepta Therapeutics
Schrodinger
Scopus BioPharma
Seagen
Seattle Genetics
Seelos Therapeutics
Sekris Biomedical
Senetek
Senior Paradise
Sensory Cloud
Seqirus
Seres Therapeutics
Sermonix Pharmaceutical
Sesen Bio
SevenScore Pharmaceuticals
Shanghai Pharmaceuticals Holdings
Sheba Medical Center
Shenzhen BioScien Pharmaceuticals
Shield Therapeutics
Shionogi
Shire Pharmaceuticals
Shore Therapeutics
Siemens Healthcare Diagnostics
Sigma-Tau
Sinclair Pharmaceuticals
Sinobiopharma
Sinopharm
Sio Gene Therapies
Skinvera
Skyline Diagnostics
Solarvest BioEnergy
Soliance
Soligenix
Sonoma BioTherapeutics
Sorrento Therapeutics
Spectranetics
Spencer Pharmaceutical
Spinal USA
St. Jude Children's Research Hospital
STADA Arzneimittel
Staidson (Beijing) BioPharmaceuticals
Stanford University
Stealth BioTherapeutics
Stellar Biotechnologies
St Jude Medical
Stoke Therapeutics
Strongbridge Biopharma
Sucampo AG
SUDA Pharmaceuticals
Sumitomo Dainippon Pharma
Sunesis Pharmaceuticals
Sunovion Pharmaceuticals
Sun Pharmaceutical
SuperNova Diagnostics
Sutro Biopharma
Swedish Orphan Biovitrum
SymBio Pharmaceuticals
Symbollon
Syndax Pharmaceuticals
Synergy Strips
Syntopix
Tactical Relief
Taiba
TaiGen Biotechnology
Taiho
Takeda Pharmaceutical
Talaris Therapeutics
Tango Therapeutics
Target Group
Tauriga Sciences
TB Alliance
TearLab
Technion Research and Development Foundation
Teijin
Telomir Pharmaceuticals
Tesaro
Tetra Bio-Pharma
TetraLogic Pharmaceuticals
Teva Pharmaceutical Industries
Texas A&M University
Texas Heart Institute
TFF Pharmaceuticals
TG Therapeutics
Therakine
TherapeuticsMD
Theravance
Theravance Biopharma
The Regents of the University of California
Thorne Research
Threshold Pharmaceuticals
TiGenix
Tilray
Titterington Veterinary Services
Tiziana Life Sciences
Tocagen
TONIX Pharmaceuticals
Torii Pharmaceutical
TRACON Pharmaceuticals
Tramorgan
Transgene
Travere Therapeutics
Trellis Bioscience
Trevena
Trimedyne
Trimeris
TrovaGene
TTFactor
Tufts University
Turnstone Biologics
Tyme Technologies
Ultragenyx Pharmaceuticals
Uluru
Unigene Laboratories
UniQure
Universal Cells
University College Dublin
University Health Network
University of Alberta
University of California
University of Central Florida Research Foundation
University of Colorado
University of Florida Research Foundation
University of Geneva
University of Louisville
University of Missouri
University of Notre of Dame
University of Pennsylvania
University of Texas
University of Virginia
University of Western Sydney
University of Zurich
UOP
Uppsalagruppen Medical
Upstream Peripheral Technologies
UroGen Pharma
Valeant Pharmaceuticals
ValiPharma
Valneva
Valor Biotherapeutics
Vanderbilt University
Vasomune Therapeutics
Vaxent
VBL Therapeutics
Veloxis Pharmaceuticals
Veracyte
Verenium
Verity Pharmaceuticals
VeroScience
Vertex Pharmaceuticals
Vida Concepts
Vifor Pharma
ViiV Healthcare
Virbac
Viropharma
VistaGen Therapeutics
Vividion Therapeutics
Vivus
VolitionRX
Voronoi
Voyager Therapeutics
Vyera Pharmaceuticals
VYNE Therapeutics
Vyrix Pharmaceuticals
Watson Pharmaceuticals
WAVE Life Sciences
Wigen Biomedicine
Wisconsin Alumni Research Foundation
World Class Extractions
WPD Pharmaceuticals
WUGEN
Xbrane Bioscience
Xencor
Xlife Sciences
XPhyto Therapeutics
XTL Biopharmaceuticals
Xynomic Pharmaceuticals
Yale University
Yeda Research and Development Company
Yeugnam University
Yissum Research Development
ZAI Laboratory
Zalicus
Zenitech
Zenith Epigenetics
Ziopharm Oncology
Zogenix
ZS Pharma
Zymeworks
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Vaccine
13 Mar 2024
Preclinical and translational pipeline program data for AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 anticipated in 2024, building further momentum toward the clinicAccelerated development of Axiomer™ for CNS applications via new research partnership with Rett Syndrome Research TrustCompleted divestment of sepofarsen and ultevursen ophthalmic programs to Laboratoires Théa€118.9 million cash and cash equivalents as of December 31, 2023 providing runway into mid-2026 LEIDEN, Netherlands & CAMBRIDGE, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2023, and provided a business update.
“Over the past year as we exclusively focused our strategy on RNA editing, ProQR made important progress with Axiomer, our ADAR-mediated RNA editing technology platform,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “We announced our initial pipeline programs, AX-0810 and AX-1412, targeting NTCP and B4GALT1, respectively, and presented preclinical platform data across a variety of targets. Our platform has demonstrated robust editing, including reporting up to 70% editing of ACTB in the liver of non-human primates, as well as functional protein data with the liver target ANGPTL3 in mice. Additionally, we further strengthened our leading global IP estate for ADAR-mediated RNA editing, which was upheld when challenged by multiple parties in various jurisdictions. We continued to execute and build on the successes achieved during the first two years of our collaboration with Eli Lilly, and formed a new partnership with the Rett Syndrome Research Trust. We also completed the divestment of late-stage ophthalmic assets, sepofarsen and ultevursen, to Laboratoires Théa who will continue the development of these therapies for patients, as ProQR continues to focus exclusively on advancing our RNA editing platform and pipeline.” De Boer continued, “In 2024, we anticipate sharing in vitro, in vivo, and translational data for our initial pipeline programs using Axiomer, and remain on track to enter the clinic in late 2024/early 2025. Along with our extensive body of preclinical proof of concept data for the platform, partnership with Eli Lilly, leading IP position, and strong cash position providing runway into mid-2026, we believe that ProQR is well positioned to continue to execute on our strategy and advance our mission of changing lives through transformative RNA therapies.” Recent Progress In February, ProQR announced that it further strengthened its leading intellectual property estate for ADAR-mediated RNA editing by successfully defending against an opposition filed in Japan by a strawman against ProQR’s granted patent JP 7244922, which is related to the broad concept of using chemically modified oligonucleotides to target specific adenosines within target RNA using endogenous enzymes. The Japanese Patent Office rejected the strawman’s opposition and indicated that all claims were to be maintained as granted to ProQR.In January, ProQR highlighted its continued progress in advancing its Axiomer™ RNA editing technology platform, with new data presented at the 5th International Conference on Base Editing, Prime Editing & Related Enzymes (Deaminet 2024), including: Platform demonstrates robust in vivo editing capabilities reporting up to 70% editing of ACTB in the liver of non-human primates (NHPs) and mice; andFunctional effect demonstrated in mice in vivo via modulation of ANGPLT3 protein properties leading to favorable increase in LPL enzymatic activity and meaningful impact of biomarkers. In January, ProQR and the Rett Syndrome Research Trust (RSRT) formed a partnership focused on utilizing ProQR’s Axiomer™ RNA editing technology platform to design and develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndrome. RSRT awarded ProQR approximately $1 million as a research grant for the initial phase of the project, which encompasses editing oligonucleotide design and optimization, including evaluation in in vivo models for editing efficiency and MECP2 protein recovery. It is the intent of the partnership to be continued by an expanded co-funding arrangement following the initial discovery work.In December, ProQR completed the divestment of sepofarsen and ultevursen to Laboratoires Théa, who will continue the development of these programs for patients with LCA10 and Usher syndrome. The transaction agreement provided ProQR with an initial payment of €8 million and the Company may be eligible for up to €165 million in further development, regulatory, and commercial earn-out payments upon related achieved milestones, as well as double-digit royalties based on commercial sales in the US and EU. Anticipated Upcoming Events Additional Axiomer™ platform data updates throughout 2024.Preclinical and translational pipeline program data for AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1: In vitro and in vivo data for AX-0810 for Cholestatic Diseases targeting NTCP in mid 2024, and In vitro and in vivo data for AX-1412 for Cardiovascular Diseases targeting B4GALT1 in the second half of 2024. Continue to progress on key steps required to advance AX-0810 and AX-1412 to clinical trials with translational data updates ahead of entry to clinic, which is on track for late 2024/early 2025.Potential initial Trident preclinical data in late 2024 – Trident is ProQR’s early stage RNA editing pseudouridylation platform designed to enable the suppression of nonsense mutations and premature stop codons, which can edit a uridine (U) into a pseudouridine.Potential additional new pipeline target announcements in 2024.Continue to execute on partnership with Eli Lilly and Company (Lilly), with potential data updates to come in 2024 along with potential milestone income from existing partnership, and potential option to exercise for expansion of deal to 15 targets, which would result in a $50 million opt-in payment to ProQR.ProQR may selectively form new partnerships, which could include multi-target discovery alliances, or product alliances on specific programs.
Year End 2023 Financial Highlights At December 31, 2023, ProQR held cash and cash equivalents of €118.9 million, compared to €94.8 million at December 31, 2022. Net cash generated by operating activities during the full year ended December 31, 2023 was €21.5 million, compared to €68.5 million net cash used in operating activities for the same period in 2022. The Company experienced a net positive cash flow from operating activities in 2023 primarily due to the receipt of the Lilly up-front payment of $60 million in February 2023. Research and development costs for the year ended December 31, 2023 were €25.1 million, compared to €50.9 million for the same period in 2022. Research and development costs for the year end December 31, 2022 included costs related to the winding down of ophthalmology programs, including the clinical trials. General and administrative costs for the year ended December 31, 2023 were €16.2 million, compared to €18.7 million for the same period in 2022.
Net loss for the year ended December 31, 2023 was €27.7 million or €0.35 per diluted share, compared to €64.2 million, or €0.90 per diluted share for the same period ended December 31, 2022. For further financial information for the period ended December 31, 2023, please refer to our 2023 Annual Report on Form 20-F and our Statutory Annual Report which will be available on our website, www. proqr.com under Financials and Filings. About Axiomer™ ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.
About Biliary Atresia (BA) and Primary Sclerosing Cholangitis (PSC)
Cholestatic disorders refer to a group of diseases presenting excessive and toxic buildup of bile acids in the liver due to bile ducts dysfunction. This leads to liver damage and a range of debilitating symptoms. Without treatment, liver damage can progress through various stages, ultimately leading to liver failure and elevated risk of liver malignancy, affecting life expectancy. Cholestatic diseases remain leading causes of liver transplantation. There are no approved therapies for primary sclerosing cholangitis (PSC) for adults and biliary atresia (BA) for pediatrics It is estimated that 80,000 and 20,000 individuals have PSC and BA, respectively, in North America and in Europe.
About AX-0810 targeting NTCP The majority of the bile acids present in the liver cells originate from the enterohepatic reuptake cycle. The key transporter responsible for hepatic uptake of bile acids from portal circulation is the sodium (Na+)-taurocholate cotransporting polypeptide (NTCP, SLC10A1 gene) expressed in the liver. AX-0810 is designed to introduce a loss of function variant in SLC10A1 RNA that has been found in human genetics to prevent re-uptake of bile acids in liver via NTCP. Based on its mechanism of action, AX-0810 has the potential to become a disease modifying treatment for PSC and BA primarily among other cholestatic diseases. About Cardiovascular Diseases Cardiovascular diseases (CVDs) are a group of health conditions that affect the heart and blood vessels, such as atherosclerosis which can lead to severe problems like heart attacks, heart failure, and stroke. CVDs represent the leading cause of disability and death in the world. Approximately 18 million people die every year from CVDs representing one third of all the global deaths. Despite available lipid lowering therapies and hypertension medications, the risk of CVDs is still projected to increase rapidly over the coming years. About AX-1412 targeting B4GALT1 Gene–based analysis of rare beta-1,4-galactosyltransferase 1 (B4GALT1) missense variant (p.Asn352Ser) is known to lead to B4GALT1 protein loss of function and showed an association with decreased coronary artery disease. These beneficial effects are mediated by hypo-galactosylation of the apolipoprotein B100 and fibrinogen, known – independent – drivers of increased risk of CVDs. AX-1412 introduces a protective variant into B4GALT1 RNA to address the remaining residual risk of developing cardiovascular diseases. ProQR intends to advance AX-1412 targeting B4GALT1 to early clinical proof of concept stage, then would seek to partner this program. About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at www.proqr.com. Forward Looking Statements
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, preclinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, our Axiomer™ platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and our ability to expand preclinical in vivo and in vitro data, the timing, progress and results of our preclinical studies and other development activities, including the release of data related thereto, our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development, the potential of our technologies and product candidates, the collaboration with Lilly and the intended benefits thereof, and our financial position and cash-runway. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability and conflicts. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. ProQR Therapeutics N.V. Investor contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorPeter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@lifesciadvisors.com Media contact:Robert StanislaroFTI ConsultingT: +1 212 850 5657robert.stanislaro@fticonsulting.com
Financial StatementOligonucleotideLicense out/in
100 Deals associated with ProQR Therapeutics NV
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100 Translational Medicine associated with ProQR Therapeutics NV
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Pipeline
Pipeline Snapshot as of 03 Jul 2024
The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3
Discovery
4
4
Preclinical
Phase 2 Clinical
1
2
Phase 3 Clinical
Other
12
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Current Projects
| Drug(Targets) | Indications | Global Highest Phase |
|---|---|---|
Ultevursen ( usherin ) | Usher Syndrome type 2 More | Phase 3 Clinical |
Sepofarsen ( Cep290 ) | Retinal Dystrophies More | Phase 3 |
QR-1123 ( RHO ) | Retinitis More | Phase 2 |
AX-0810 ( NTCP ) | Liver Diseases More | Preclinical |
QR-1011 ( ABCA4 ) | Stargardt Disease More | Preclinical |
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