What's new?
04/04/2018

• Sofinnova Crossover Fund 1 is the largest healthcare crossover fund focused on Europe.
• With this new fund, Sofinnova Partners actively pursues the strategy to broaden its Life Sciences platform to invest across the value chain, from seed stage to late-stage companies.
• Bpifrance* and CNP Assurances acted as sponsors for the fund.

Paris, France – April 4th, 2018 — Sofinnova Partners, a leading European venture capital firm specialized in Life Sciences, announced today the launch of Sofinnova Crossover I with €275 million ($340 million), above the original first close target of €250 million. With this new fund, Sofinnova Partners goes one step further in the execution of its growth plan, aimed at expanding its coverage across the Life Sciences space with dedicated sector teams.

Pursuing a strategy Sofinnova Partners has successfully applied for decades with its early-stage focused Capital Funds, Sofinnova Crossover I will invest in the biopharmaceutical and medical device sectors. The fund will focus primarily on therapeutic and game-changing companies driven by experienced management. As a lead or cornerstone investor, the fund will seek to invest in about 15 late stage private and public companies. About 80% of the investments will be made in European companies, with the remaining 20% outside of Europe primarily in North America. A dedicated, highly experienced team of four partners will invest Sofinnova Crossover I, leveraging on Sofinnova Partners’ wider experience, track record and organizational support. This new fund attracted premier international investors, predominantly sovereign funds, insurance companies, corporations and family offices. Commitments came from Europe, including France, Italy, Denmark, Ireland, and Switzerland but also from Asian investors in China and Singapore. In addition to Bpifrance* and CNP Assurances, investors include a major Chinese biopharmaceutical company, the Danish State Investment fund, and family offices like Fidim or KCK representing leading industrial families in Europe and Asia.

Antoine Papiernik, Chairman of Sofinnova Partners, said, “With the launch of this new crossover activity, Sofinnova builds upon its unique early stage track record. Many of the companies we initially funded have become over the years large, billion-euro companies, and we have gained invaluable experience in helping them to the next level. This fund will complete our investment platform across the life-sciences value-chain, allowing us to fund companies from the seed stage to the late-stage.”

Jacques Theurillat, Partner in the crossover team at Sofinnova Partners, added: “The European healthcare market has matured with hundreds of late stage private and public companies looking for growth capital, and Sofinnova Partners, with its name, track record and experience, is particularly well positioned to identify the best European deals and transform them into global leaders.”

Triago acted as placement agent and Clifford Chance Europe LLP acted as legal counsel on Sofinnova Crossover I.

* Bpifrance directly and with the “Investment for the Future” Program, managed by the SGPI and operated by Bpifrance

About Sofinnova Partners
Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, the firm brings together a team of professionals from all over Europe, the US and China. The firm focuses on paradigm shifting technologies alongside visionary entrepreneurs. Sofinnova Partners seeks to invest as a lead investor in start-ups and corporate spin-offs and has backed nearly 500 companies over more than 45 years, creating market leaders around the globe. Today, Sofinnova Partners has over €1.9 billion under management.

11/12/2017

Proceeds will be used to progress lead development programs through Proof-of-Concept clinical trials

Vienna, Austria, 11 December 2017 – Hookipa Biotech AG (“Hookipa”), a clinical stage biotech company pioneering an innovative class of immune activation therapies for oncology and infectious diseases, today announced that it has raised $59.6 million (€50.0 million) in an oversubscribed Series C financing.

The round was led by an undisclosed blue chip U.S. public investment fund specializing in life sciences, alongside other new investors HBM Partners, Hillhouse Capital, Sirona Capital and strategic investor Gilead. All current Hookipa investors, Sofinnova Partners, Forbion Capital Partners, Boehringer Ingelheim Venture Fund, Takeda Ventures and BioMedPartners participated in the round.

The proceeds of the fundraising will be primarily used to progress two proof-of concept clinical trials of Hookipa’s lead development programs, a phase 2 study of the Company’s prophylactic Cytomegalovirus (“CMV”) vaccine in solid organ transplant patients, and a phase 1 study of its TheraT® based active immunization therapy in patients with head & neck squamous cell carcinoma. In addition, Hookipa plans to expand its technology platform into other disease areas, such as prostate cancer.

Hookipa’s arenavirus-based vector technologies TheraT® and Vaxwave® have been designed to infect target cells and stimulate potent and long-lasting immune responses. HB-101, the Company’s Vaxwave®-based CMV vaccine successfully completed a phase 1 trial earlier this year, demonstrating safety and immunogenicity. TheraT® has been shown to elicit uniquely potent antigen-specific CD8+ cytotoxic T cell responses and strong tumor control in mice. TheraT® works by delivering tumor-associated antigen-specific immunization alongside the release of the alarmin interleukin-33 (IL-33), which is key in stimulating potent and protective CD8+ cytotoxic effector T lymphocytes.

Joern Aldag, Chief Executive Officer of Hookipa said: “Our vision is of a world in which the immune system actively controls infectious diseases and cancer, using monotherapy or combinations of medications. We welcome the funding and support from this group of leading current and new investors, who have recognized the potential, versatility, and uniqueness of our novel viral vector platform in this context. This financing allows us to progress two lead development programs through the major inflection points of clinical proof of concept, and to expand our clinical work to additional virology and oncology indications.”
About Hookipa Biotech
Hookipa Biotech is a clinical stage company developing next-generation immunotherapies for infectious diseases and cancer using novel proprietary arenavirus vector platforms.

Hookipa´s Vaxwave® technology presents a completely new replication-defective viral vector platform designed to overcome the limitations of current technologies. Vaxwave® is based on lymphocytic choriomeningitis virus (LCMV). In this vector the gene encoding the LCMV envelope protein, normally responsible for virus entry into target cells, has been deleted and replaced with a target gene of interest. The resulting vectors infect target cells and stimulate very potent and long-lasting immune response, however they can no longer replicate and are therefore non-pathogenic and inherently safe.

Hookipa’s TheraT® platform is based on an attenuated replicating virus and is capable of eliciting the most potent T cell responses – a crucial step in treating patients with aggressive cancers. Significant pre-clinical data demonstrates that TheraT® is a powerful modality capable of turning “cold tumors hot” which should result in an additional layer of efficacy in the fight against solid tumors. Specifically, TheraT® has proven to be safe in animals as well as capable of eliciting uniquely potent antigen-specific CD8+ cytotoxic T cell responses and strong tumor control in mice. The first clinical trial with HB-201 targeting human papilloma virus-induced head and neck cancer is currently being prepared. This immuno-oncology technology is further being leveraged to target tumor self-antigens or shared neoantigens.

Issued for and on behalf of Hookipa Biotech AG by Instinctif Partners. For further information please contact:

At the Company
Marine Popoff Communications Analyst Hookipa Biotech AG
Mpopoff@Hookipabiotech.com

Media enquiries
Sue Charles/ Ashley Tapp
Instinctif Partners
hookipa@instinctif.com
+44 (0)20 7866 7923

08/12/2017

Paris, December 8, 2017 – HighLife SAS, a medtech company focused on the development of a unique trans-catheter mitral valve replacement (TMVR)system to treat patients suffering from mitral regurgitation, announced today the appointment of Mr. Jose (“Pepe”) Calle Gordo as Chairman of its Board of Directors.

Pepe brings 30 years of broad business and management experience in the medical device industry, having served in various executive and managerial roles at Biosensors, Abbott, Guidant and Eli Lilly.

Up to recently, he was Vice Chairman of the Board of Directors of Biosensors, where he last served as Executive Director and CEO. During his tenure, based in Singapore, he turned around the company’s operating performance, forged strategic alliances and worked with the largest shareholder to successfully privatize and delist the company in order to create a 1$Bn value global player in interventional cardiology.

He previously led the global team that developed Xience™, a family of drug eluting stents and market leader for the treatment of coronary artery disease while serving as Vice President and General Manager of Drug Eluting Stents, Vascular Intervention at Guidant based in California.

As Vice President of Abbott Vascular based in Brussels, he later managed the international operations of Abbott Vascular outside of the US and led globally the commercial introduction of MitraClip™, a percutaneous treatment for mitral valve repair.

Mr. Calle Gordo graduated from Universidad Politecnica de Madrid, Spain in Biomedical and Electrical Engineering.

“I am delighted to join the HighLife team, I look forward to supporting the company and the board to make the HighLife mitral valve a reality for patients who can benefit from TMVR.” said Pepe.

Based in Paris (France), HighLife was started in 2010 by Georg Börtlein. Following the recent investment in HighLife from Sofinnova Partners, Antoine Papiernik joined the company’s board.

“We are excited to have Pepe Calle Gordo join the company as its Chairman. His long experience and track record in the interventional cardiology field is second to none.” said Antoine Papiernik, Managing Partner of Sofinnova Partners.

“I am honored and thrilled we could attract a chairman with the talent and experience of Pepe. His perfect and global understanding of structural heart and his ability in building operations come at an ideal moment in our company’s development stage.” said Georg Börtlein.

The HighLife technology relies on the initial placement of a ring component around the native mitral leaflets in a reversible manner. Once this first component’s position is confirmed, the bioprosthesis is delivered within minutes through the ring and finds its natural position inside the native annulus regardless of the access site. This approach allows for trans-septal access and delivery of the bioprosthesis after navigating up the femoral vein and reaching the native mitral valve via a puncture through the inter-atrial septum. Such route alleviates the need for a trans-apical puncture in the weakened heart muscle and is favored by physicians because further trauma to the patients is avoided.

About HighLife
HighLife SAS was established in 2010 and is headquartered in Paris, France, with offices in Irvine (California). It is focused on the development of a novel transcatheter replacement system for treating mitral regurgitation. The technology aims at a beating heart procedure reducing trauma to the patients.
Caution: The HighLife Transcatheter Mitral Valve is an investigational device and not available for sale.

About Sofinnova Partners
Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, the firm focuses on paradigm shifting technologies alongside visionary entrepreneurs. Sofinnova Partners seeks to invest as a founding and lead investor in start-ups and corporate spin-offs, and has backed nearly 500 companies over more than 40 years, creating market leaders around the globe. Today, Sofinnova Partners has over €1.6 billion under management. For more information, please visit: www.sofinnova.fr

29/11/2017

Key Updates
• ProQR’s drug candidate, QR-313 for dystrophic epidermolysis bullosa (DEB) receives orphan drug designation in the EU from the EMA.
• QR-313 also received U.S. ODD from the FDA in September 2017.
• QR-313 represents the fifth candidate in the company’s pipeline to receive ODD in the U.S. and EU.
• DEB is a severe genetic skin disease with no disease modifying treatments currently available.
• QR-313 targets the most common mutations within DEB, which are mutations in exon 73 of the COL7A1 gene and is designed for topical administration.
• QR-313 is expected to enter the clinic in 2018, with interim data also expected in 2018.

LEIDEN, the Netherlands, November 29, 2017 — ProQR Therapeutics N.V.(Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases including cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced that investigational drug QR-313 for dystrophic epidermolysis bullosa (DEB) has received orphan drug designation (ODD) from the European Medicines Agency (EMA). QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause in dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. DEB is a rare genetic disease that can lead to severe blistering of the skin resulting in high treatment burden and poor quality of life for patients.

In September 2017, QR-313 also received ODD from the FDA. This marks the fifth drug candidate in the company’s pipeline to receive ODD from the FDA and EMA. A first-in-human clinical trial of QR-313 is expected to be initiated in 2018, with interim data readout also expected in 2018.

“We are pleased to have orphan drug designation for our QR-313 program targeting dystrophic epidermolysis bullosa in both the U.S. and Europe,” said David M. Rodman, MD, Chief Development Strategy Officer of ProQR. “This represents another milestone for our company and highlights the unmet need for patients with this devastating disease. Our goal is to develop and actively advance a pipeline of programs that can treat DEB mutations in a targeted manner.”

About EMA Orphan Drug Designation (ODD)
In Europe, to qualify for orphan drug designation, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating, when the prevalence in the EU is not more than 5 in 10,000 (or it is unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development), and when no satisfactory method of diagnosis, prevention or treatment of the condition exist, or, if such method exists, the medicine will be of significant benefit to those affected by the condition. As incentives to encourage the development of orphan medicines, the EU offers protocol assistance specific for designated orphan medicines, 10 years of market exclusivity once the medicine is on the market, and fee reductions.

About QR-313
QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce a functional C7 protein, thereby restoring functionality of the anchoring fibrils.

About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*

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 “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. 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. These forward-looking statements include, but are not limited to, statements regarding QR-313 and the clinical development and the therapeutic potential thereof, statements regarding ODD and the potential benefits thereof, and statements regarding our pipeline of programs targeting DEB. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that we may not realize the intended benefits afforded by orphan drug designation for our QR-313 program targeting DEB, positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and 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. 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 and Media Contact:
Bonnie Ortega
T: +1 858 245 3983
ir@proqr.com

29/11/2017

Paris, France – November 29th. 2017. Sofinnova Partners, a leading Life Sciences venture capital firm focused on Europe, has appointed Maina Bhaman as Partner.

Maina Bhaman comes to Sofinnova Partners with a long track record as a successful healthcare investor. Prior to joining, she was Director of Healthcare Investment at Touchstone Innovations (formerly Imperial Innovations) in London (UK) since 2006. She has led or co-led numerous investments and sat on the Board of a number of UK biotech companies, such as Autifony, Cellmedica, Psioxus Therapeutics, Pulmocide, Topivert Pharma, Puridify (sold to GE), Thiakis (sold to Wyeth) and Respivert (sold to J&J). Previously, she worked in the R&D teams of several UK and US biotech companies, including Celltech, Oxford Glyco Sciences, Chimeric Therapies and GeneMedicine. Maina has a BSc from the University of Texas at Austin (United States) and an MBA from the Imperial Business School in London (United Kingdom).

As a long standing member of the biotech ecosystem, Maina has received strong professional and institutional recognition; and was named one of UK’s Top women in Biopharma several years in a row. With Maina Bhaman’s arrival, Sofinnova Partners continues to build a truly international team with a unique diversity.

Maina Bhaman says: “I am thrilled to join such an experienced team as Sofinnova Partners. We have worked together on a number of co-investments, and clearly share a common approach and vision. I am looking forward to continue to pursue my passion of backing entrepreneurs developing paradigm-shifting technologies that may significantly improve patients’ life”.

Antoine Papiernik, Managing Partner and Chairman of Sofinnova Partners, adds: “We are extremely happy to welcome Maina as a Partner in our team. Her expertise and track record are a perfect match with our team and strategy. This recruitment also furthers our international reach and will positively contribute to Sofinnova Partners’ growth”.

About Sofinnova Partners
Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, the firm focuses on paradigm shifting technologies alongside visionary entrepreneurs. Sofinnova Partners seeks to invest as a founding and lead investor in start-ups and corporate spin-offs, and has backed nearly 500 companies over more than 40 years, creating market leaders around the globe. Today, Sofinnova Partners has over €1.6 billion under management. For more information, please visit: www.sofinnova.fr

Media contact for SOFINNOVA PARTNERS
Anne REIN
Tel: +33 6 03 35 92 05
@: anne.rein@strategiesimage.com

15/11/2017

• European Medicine Agency’s PRIME status is granted to promising medicines that may offer a major therapeutic advantage over existing treatments or benefit patients with no treatment options.
• Accessing PRIME will support the optimization of Motrem’s development program.

Paris, November 15, 2017. Inotrem S.A., a biotechnology company specialized in the control of acute inflammatory syndromes, today announced that the European Medicines Agency (EMA) has granted access to its PRIority MEdicines (PRIME) scheme for its lead product MOTREMTM in the field of septic shock.

The purpose of PRIME created in 2016 by the EMA is to bring treatments to patients faster by providing early and enhanced support to medicines that have the potential to address patients’ unmet needs. Through the PRIME scheme, Inotrem will be able to optimize the development of its lead compound and accelerate EMA’s regulatory assessment. The inclusion of MOTREMTM in the PRIME program was supported by the following criteria: (i) there is an important unmet medical need for the treatment of septic shock, (ii) the efficacy of MOTREMTM could be proven in relevant preclinical models in vivo and (iii) data from a Phase 1 clinical trial showed tolerance in human subjects. Inotrem launched this year a Phase 2 multicenter clinical trial with patients suffering from septic shock in four European countries.

Septic shock is a serious and very debilitating acute condition with high mortality and associated long-term physical, psychological, and cognitive disabilities in survivors. Sepsis, which is characterized by an intense and excessive systemic inflammatory reaction in response to a serious infection, affects worldwide up to 1% of the population annually with a mortality rate of 25 to 40% placing it as the 10th leading cause of death in developed countries and the 1st cause of death in intensive care units. MOTREMTM is the formulation of the active ingredient LR12, a synthetic peptide capable of controlling the amplification loop of the inflammatory response by inhibiting the TREM-1 receptor, and as such brings the potential of improving hemodynamic parameters and survival rates of septic shock patients. There are currently no specific therapies approved for this indication, and Inotrem’s MOTREMTM aims at becoming the first mechanism-based personalized medicine for septic shock.

“EMA’s decision to grant our product the PRIME status is an important recognition of both Inotrem’s innovative therapeutic approach in the management of acute inflammation and the critical need for causal therapies in a severe condition such as septic shock. This is also the first time a product being developed in the critical care setting is receiving the PRIME status”, said Jean-Jacques Garaud, M.D., CEO and co-founder of Inotrem. “We are very pleased to be part of this program and look forward to working together with the EMA’s Committee for Medicinal Products for Human Use with their regulatory support to pursue our clinical development plan moving toward the MotremTM marketing authorization process”, added Margarita Salcedo Magguilli, CDO of Inotrem.

About Inotrem
Inotrem is a biotechnology company specialized in the control of excessive immune response that occurs in acute inflammatory syndromes in the critical care setting. The Company has developed a new concept of immunomodulation to control abnormal immune response to tissue injuries. The Company has been founded in 2013 in Nancy by Dr. Jean-Jacques Garaud, a former head of research and early development at the Roche Group, Prof. Sébastien Gibot and Dr. Marc Derive. The lead product of Inotrem (LR12) paves the way to new targeted treatments in septic shock and myocardial infarction. Inotrem is supported by leading European specialist investors — Sofinnova Partners, Edmond de Rothschild Investment Partners, Biomed Invest and Inserm Transfert Initiative.
www.inotrem.com

About TREM-1 and MOTREMTM (LR12)
Inotrem focuses on targeted immunotherapy for acute inflammatory syndromes in the critical care setting and has a significant expertise in the biology of the TREM-1 receptor. TREM-1 is an immune receptor expressed by cells mediating innate immunity as well as endothelial cells in tissue stress situations. The role of TREM-1 is an amplifier of the inflammatory response which has been characterized initially in septic shock, ischemia reperfusion injury, myocardial infarction, hemorrhagic shock, pancreatitis and renal failure. The TREM-1 pathway is one of the most overexpressed pathways during the “genomic storm” reported in patients with septic shock. The Activation of the TREM-1 pathway leads to an excessive inflammatory response involved in the transition from sepsis to septic shock.
LR12 is a synthetic peptide capable of controlling the amplification loop of the inflammatory response by inhibiting the TREM-1 receptor. Several preclinical septic shock models demonstrate therapeutic benefits of LR12 on several animal species, with a balanced inflammatory response, improved hemodynamic parameters and survival rates.
Inotrem collaborates with Roche Diagnostics on a personalized medicine strategy to develop a companion diagnostic test to allow a stratification of sepsis patients and identify those who are more likely to respond to Inotrem’s treatment. There is currently no specific therapies for this indication, and past attempts to develop dedicated treatments have failed.

13/11/2017

Key Updates
• The first patient has received the first dose of QR-110 in the Phase 1/2 safety & efficacy clinical trial (PQ-110-001: NCT03140969) in children and adults with Leber’s congenital amaurosis 10 (LCA 10).
• LCA 10 is one of the most prevalent forms of gene-related blindness in children worldwide and currently there are no therapies commercially available or in clinical development for this disease.
• QR-110 has received fast track designation by the U.S. Food and Drug Administration (FDA) and has been granted orphan drug designation in the United States and European Union.
• Interim safety and efficacy trial results from the majority of patients after 6 months of treatment are expected in 2018, full 12 month treatment data from all patients are expected in 2019.
LEIDEN, the Netherlands, November 13, 2017 — ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that the first patient has been dosed in the Phase 1/2 open-label trial assessing the safety, tolerability, pharmacokinetics and efficacy of QR-110. The trial will enroll approximately six adults and six children who have Leber’s congenital amaurosis 10 (LCA 10) due to the p.Cys998X mutation in the CEP290 gene. Subjects will receive a dose of QR-110 every three months for a total of four doses in one eye. The trial is planned to be conducted at three specialized centers: the University of Iowa, Iowa City, IA, US, the Scheie Eye Institute at the University of Pennsylvania, Philadelphia, PA, US and the Ghent University Hospital, Ghent, Belgium.
Benjamin Yerxa, PhD., Chief Executive Officer at Foundation Fighting Blindness, stated, “We are delighted with the launch of ProQR’s clinical trial for its treatment for people with devastating vision loss caused by the p.Cys998X mutation in CEP290. There are no other options for these patients, and furthermore, we believe most emerging gene replacement technologies do not have the capacity to deliver the large CEP290 gene to the retina.”
QR-110 is ProQR’s lead program in the ophthalmology pipeline that also includes two programs for Usher syndrome, a program for Fuchs endothelial corneal dystrophy and a program for Stargardt’s disease. QR-110 is ProQR’s second program to enter clinical development, following QR-010, which is being developed for the most common mutation causing cystic fibrosis.
“This announcement recognizes an important next step towards achieving our goal of developing precision medicines based on molecular diagnostics and our RNA therapeutic platform,” said David M. Rodman, MD, Chief Development Strategy Officer of ProQR. “The QR-110 program is the first in a planned series of ophthalmology trials utilizing our RNA therapy platform to target the underlying cause of blindness in patients with inherited forms of retinal dystrophy. We expect this trial will give us fundamental information regarding the safety, efficacy and developability of QR-110 in adults and children with LCA 10.”
Key facts on QR-110
• QR-110 aims to delay the progression of the disease or restore vision in people with LCA 10 due to the p.Cys998X mutation in the CEP290 gene.
• QR-110 is a single stranded RNA oligonucleotide designed to restore wild-type or normal CEP290 mRNA.
• In pre-clinical studies of QR-110, it was shown to convert close to 100% of the mutant mRNA to wild-type in a homozygous optic cup organoid model.
• A long half-life in the eye allowing for infrequent dosing.
• Administered through intravitreal administration, which is considered a routine procedure.
• A ProQR sponsored pre-evaluation/retrospective natural history study collecting data of 22 LCA 10 patients over a period of 16 years was completed in 2017 (Samuel G. Jacobson et al; Outcome Measures for Clinical Trials of Leber Congenital Amaurosis Caused by the Intronic Mutation in the CEP290 Gene. Invest. Ophthalmol. Vis. Sci. 2017;58(5):2609-2622.)
About the PQ-110-001 trial
PQ-110-001 is an open-label trial that will include approximately six children (age 6 – 17 years) and six adults (≥ 18 years) who have LCA 10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. During the trial, subjects will receive four intravitreal injections of QR-110 into one eye; one every three months. The QR-110 trial is expected to be conducted in three centers with significant expertise in genetic retinal disease in the US and Europe.
The objectives of the trial will include safety, tolerability, pharmacokinetics and efficacy as measured by restoration or improvement of visual function and retinal structure through ophthalmic endpoints such as visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR), mobility course and fixation stability. Changes in quality of life in the trial subjects will also be evaluated. Interim safety and efficacy trial results from the majority of patients after 6 months of treatment are expected in 2018, full 12 month treatment data from all patients in the trial are expected in 2019.

About Leber’s Congenital Amaurosis 10
Leber’s congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of which LCA 10 is the more severe forms. LCA 10 is caused by mutations in the CEP290 gene of which the p.Cys998X mutation is the most common. LCA 10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved or other products in clinical development that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA 10 because of this mutation.
About QR-110
QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore normal (wild-type) CEP290 mRNA leading to the production of normal CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation by the FDA.
The company presented promising pre-clinical data that supports the clinical development of QR-110 at several conferences including the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in 2016 and 2017. QR-110 has demonstrated the capacity to rescue a normal mRNA and protein profile in LCA 10 patient cells carrying one or two copies of the p.Cys998X mutation. In a LCA 10 patient-derived optic cup organoid model QR-110 rescued the mRNA profile which lead to a functional CEP290 protein. It was shown that QR-110 reaches the outer nuclear layer in vivo, the target site for therapeutic action in the eye, following a single intravitreal injection.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*

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 “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. 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. These forward-looking statements include, but are not limited to, statements regarding QR-110 and the clinical development and the therapeutic potential thereof, statements regarding PQ-110-001, including trial design and expected timing of results, statements regarding orphan drug designation and Fast Track designation, and statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in our innovation pipeline. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, that a Fast Track designation by the FDA may not actually lead to a faster development, regulatory review or approval process, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and 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. 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 and Media Contact:
Bonnie Ortega
T: +1 858 245 3983
ir@proqr.com

10/11/2017

Paris, November 10, 2017. Inotrem S.A., a biotechnology company specialized in the control of acute inflammatory syndromes, such as septic shock, today announced a R&D collaboration agreement with Roche Diagnostics to develop a companion diagnostic test using a soluble plasma circulating protein (sTREM-1) developed by Inotrem and the Roche proprietary Elecsys® platform. It is Roche Diagnostics’ first collaboration agreement with a start up biotech company.
Under the terms of the agreement, Roche and Intorem will work together to develop an in vitro robust prototype assay for quantitative measurement of soluble TREM-1 (sTREM-1) in plasma samples of septic shock patients. sTREM-1 is a marker of the activation of the TREM-1 immune amplification pathway and high sTREM-1 plasma concentrations have been shown to be associated to a negative outcome in septic shock patients. Measurement of sTREM-1 in blood could provide a valuable indicator for the diagnosis and outcome prediction of septic shock patients1,2,3.
Inotrem is currently conducting a clinical Phase 2 trial in patients with septic shock to demonstrate the benefit of its lead compound, Motrem™ (LR12) in the treatment of septic shock4. The collaboration with Roche Diagnostics may lead to a companion diagnostic supporting the development of Motrem™.
One of the main issues with septic shock is the heterogeneity of this patient population. This collaboration proposes to develop a test to allow a certain stratification of sepsis patients to ideally identify patients who are more likely to respond to Motrem treatment,” said Jean-Jacques Garaud, CEO and co-founder of Inotrem. “It shows our shared willingness to accelerate the development of targeted therapeutic solutions for these patients.”
The physiopathology of septic shock is characterized by an intense and excessive systemic inflammatory reaction in response to a serious infection5. Its consequences include the dysfunction of vital organs and major hemodynamic disorders that may prove fatal for patients. Activation of the TREM-1 pathway is recognized as a key factor contributing to septic shock6.
“As a leader in in vitro diagnostics solutions7, it is a great opportunity to participate to the opening of a new front in critical care medicine, particularly in an area known for its difficult and highly heterogeneous population in septic shock”, shared Jean-Claude Gottraux, Head of Roche Diagnostics, Centralized and Point of Care Solutions. “We are particularly pleased as this is the first time Roche Diagnostics is collaborating with a start-up biotech company.”

Media contact for Inotrem
Anne REIN | S&I | anne.rein@strategiesimage.com | +33 6 03 35 92 05

About Inotrem
Inotrem S.A., is a biotechnology company specialized in the control of acute inflammatory syndromes, such as septic
shock. The company has developed a new concept of immunomodulation to control unbalanced inflammatory
responses. Focusing on targeted immunotherapy for acute inflammatory syndromes in the critical care setting, the
company has been founded in 2013 by Dr. Jean-Jacques Garaud, a former head of research and early development
at the Roche Group, Prof. Sébastien Gibot and Dr. Marc Derive. Inotrem’s lead product candidate (LR12) opens new
personalized treatment options in a number of therapeutic indications such as septic shock or myocardial infarction.
Inotrem is supported by leading European investors — Sofinnova Partners, Edmond de Rothschild Investment
Partners, Biomed Invest and Inserm Transfert Initiative.
www.inotrem.com
@Inotrem_biotech

About TREM-1 and LR12
Inotrem focuses on targeted immunotherapy for acute inflammatory syndromes in the critical care setting and has a
significant expertise in the biology of TREM-1 receptor.
TREM-1 is an immunoreceptor expressed by innate immune cells. Upon activation, TREM-1 can directly amplify an
inflammatory response. TREM-1 was initially characterized for its pathophysiological role during septic shock, and
since, in other acute diseases such as ischaemia/reperfusion injury after myocardial infarction, haemorrhagic shock,
ischaemia-reperfusion, pancreatitis and acute kidney injury. TREM-1 is one of the most upregulated pathways during
the genomic storm observed in septic shock patients. Engagement of TREM-1 leads to a hyperactivated and exuberant
inflammatory response which is responsible for the onset and progression from sepsis to septic shock. Currently, there
is no specific causal treatment for septic shock, and previous attempts to develop treatments have failed.
LR12 is a synthetic peptide aiming at controlling the amplification loop of the inflammatory response by inhibiting the
TREM-1 receptor. The therapeutic efficacy of LR12 is documented in several preclinical septic shock models in different
species which have shown an appropriate inflammatory response, an improvement in hemodynamic parameters and
survival rates.

References
1 Gibot S, et al: Combination biomarkers to diagnose sepsis in the critically ill patient. Am J Respir Crit
Care Med. 2012 Jul 1;186(1):65-71.
2 Charles PE, et al: Significance of soluble triggering receptor expressed on myeloid cells-1 elevation in
patients admitted to the intensive care unit with sepsis. BMC Infect Dis. 2016 Oct 12;16(1):559.
3 Su L, et al: Role of sTREM-1 in predicting mortality of infection: a systematic review and metaanalysis.
BMJ Open. 2016 May 13;6(5):e010314.
4 Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of 3 Doses of MOTREM in Patients
With Septic Shock. A Randomised, Double-blind, Two-Stage, Placebo Controlled Study. (2017).
(registered at www.clinicaltrials.gov NCT03158948 ; Drug Product name: MOTREM, EudraCT
Number: 2016-005032-14).
5 Nduka OO, Parrillo JE. The pathophysiology of septic shock. Crit Care Clin. 2009 Oct;25(4):677-702
6 Bouchon A, Facchetti F, Weigand MA, Colonna M. TREM-1 amplifies inflammation and is a crucial
mediator of septic shock. Nature. 2001 Apr 26;410(6832):1103-7.
7 Source: Roche Annual Report 2014. January 2015. * Status 2016.
8 Derive M, et al: Soluble TREM-like transcript-1 regulates leukocyte activation and controls microbial
sepsis. J Immunol. 2012 Jun 1;188(11):5585-92.
9 Derive M, et al: Effects of a TREM-like transcript 1-derived peptide during hypodynamic septic shock
in pigs. Shock. 2013 Feb;39(2):176-82.

31/10/2017

This financing allows for the acceleration of the company’s clinical and R&D program

PARIS, October 30. 2017 – HighLife SAS, an early-stage medtech company focused on the development of a unique trans-catheter mitral valve replacement (TMVR) system to treat patients suffering from mitral regurgitation, announced today the closing of a € 12.3 million investment round led by Sofinnova Partners which becomes the main investor in the company. HighLife had previously secured financing from a corporate player, LivaNova PLC, which also participated in this financing round along with Georg Börtlein, the CEO and founder of the HighLife.

Based in Paris (France), HighLife was started in 2010 by Georg Börtlein, who was previously a co-founder with Professor Jacques Séguin and Chief Operating Officer at CoreValve Inc., a pioneer in the development of trans-catheter aortic valve implantation supported early on by Sofinnova Partners until the sale to Medtronic in 2009 for more than $700 million.

“We are excited to invest in HighLife and believe that their unique trans-septal approach is the next innovative disruption in trans-catheter valve technologies,” said Antoine Papiernik, Managing Partner of Sofinnova Partners, “We are also happy to partner with Georg Börtlein for the second time and confident that HighLife’s technology represents the next generation TMVR product needed to reach meaningful clinical adoption.”

“This investment enables us to implement our aggressive development plan and move forward with both our ongoing clinical programs and our R&D pipeline. We are currently recruiting patients in several European countries including France and Germany and are now planning to accelerate the enrollment of this initial clinical phase.” said Georg Börtlein.

The HighLife technology relies on the initial placement of a ring component around the native mitral leaflets in a reversible manner. Once this first component’s position is confirmed, the bioprosthesis is delivered within minutes through the ring and finds its natural position inside the native annulus regardless of the access site. This approach allows for trans-septal access and delivery of the bioprosthesis after navigating up the femoral vein and reaching the native mitral valve via a puncture through the inter-atrial septum. Such route alleviates the need for a trans-apical puncture in the weakened heart muscle and is favored by physicians because further trauma to the patients is avoided.
About HighLife
HighLife SAS, headquartered in Paris, France, with offices in Irvine (California), is an early-stage company established in 2010. It is focused on the development of a novel transcatheter replacement system for treating mitral regurgitation. The technology aims at a beating heart procedure reducing trauma to the patients.
Caution: The HighLife Transcatheter Mitral Valve is an investigational device and not available for sale.

About Sofinnova Partners
Sofinnova Partners is an independent venture capital firm based in Paris, France. For more than 40 years, the firm has backed nearly 500 companies at different stages of their development – pure creations, spin-offs, as well as turnaround situations – and worked alongside key entrepreneurs in the Life Sciences industry around the globe. With over €1.6 billion of funds under management, Sofinnova Partners has created market leaders with its experienced team and hands-on approach in building portfolio companies through to exit. For more information, please visit: www.sofinnova.fr

20/10/2017

Avantium N.V., (Euronext Amsterdam and Brussels: AVTX) (“Avantium” or “the Company”) a leading chemical technology company and forerunner in renewable chemistry, announces that today it has published its convocation of the Extraordinary General Meeting of shareholders (EGM) to be held at Avantium’s headquarters, Zekeringstraat 29, Amsterdam, The Netherlands on 30 November 2017 at 10.00 am CET. On the agenda is the appointment of Mr. Kees Verhaar as a new member to Avantium’s Supervisory Board.

Avantium’s Supervisory Board has nominated Kees Verhaar, who will join the Supervisory Board for a term of four years, subject to approval by shareholders at the EGM. He will succeed Mr. Jan van der Eijk, who had temporarily resumed his role as Chairman of the Supervisory Board of Avantium. Mr. Jan van der Eijk will resign with effect as of immediately after the EGM of 30 November 2017. He will continue in his role as Chairman of the Supervisory Board of Synvina, the Joint Venture of Avantium and BASF.

Once approved by the shareholders as a Supervisory Board member, Kees Verhaar will be appointed as Chairman of the Supervisory Board. His appointment will be effective immediately after the EGM of 30 November 2017.

Kees Verhaar’s experience as CEO of Arizona Chemicals, the world leading wood-based chemicals company and his 30-year track record in the chemical industry, which includes extensive collaboration with sophisticated financial investors are very valuable to support Avantium in building a winning renewable chemistry company. Kees Verhaar is currently chairman of the advisory board of three business units of Ten Cate, an international advanced materials company.

The convocation, agenda and explanatory notes for the EGM together with the resume of Mr. Kees Verhaar are published on the Company’s corporate website.
This is a public announcement by Avantium N.V. pursuant to section 17 paragraph 1 of the European Market Abuse Regulation (596/2014).
About Avantium
Avantium is a leading chemical technology company and a forerunner in renewable chemistry. Together with its partners around the world, Avantium develops efficient processes and sustainable products made from biobased materials. Avantium offers a breeding ground for revolutionary renewable chemistry solutions from invention to commercially viable production processes.
One of Avantium’s success stories is YXY technology, with which it created PEF: a completely new, high-quality plastic made from plant-based industrial sugars. Since October 2016 all YXY activities have been transferred to Synvina, the joint venture of Avantium and BASF.

Avantium is also working on a host of other groundbreaking projects such as the Zambezi process; a biorefinery technology to produce sugars for the production of chemicals and fuels from non-food materials. The proprietary process is highly feedstock-flexible allowing use of forestry residues (e.g. woodchips), corn stover, bagasse and produces a high purity 2G glucose product and lignin for energy and other applications. Avantium also provides advanced catalysis research services and systems to the leading chemical and petrochemical companies.

Avantium shares are listed on Euronext Amsterdam and Euronext Brussels (symbol: AVTX), its offices and headquarters are based in Amsterdam, the Netherlands. Over 120 highly skilled colleagues representing 20 nationalities, Avantium fosters a dynamic and enthusiastic workplace that is constantly seeking new ways to improve and expand the impact of advanced catalytic research & technology.
MEDIA CONTACT
For more information:
Dominique Levant, Marketing & Communications Officer
T: +31 20 586 01 32 – E: dominique.levant@avantium.com
or visit our website www.avantium.com.

19/10/2017

French regulatory Authority, ANSM, approves PRIMA’s feasibility study for advanced dry-AMD
Paris, France. October 19, 2017 – 7.00 AM CET – Pixium Vision (FR0011950641 – PIX), a company developing innovative bionic vision systems to enable patients who have lost their sight to lead more independent lives, today announces that its next-generation miniaturized wireless sub-retinal implant, PRIMA, to restore vision in patients affected by retinal dystrophies, received authorization from the French regulatory agency, Agence Nationale de Sécurité du Médicament et des Produits de santé (ANSM), to start a feasibility clinical study in patients with advanced dry age-related macular degeneration (dry-AMD).
Khalid Ishaque, Chief Executive Officer of Pixium Vision, commented: “The approval of the clinical study is a significant advance for the PRIMA system, our next generation wireless sub-retinal implant system, as well as for Pixium Vision. Conceived initially by the researchers at Stanford University, and successfully developed through to clinical stage by our team at Pixium Vision in close collaboration with numerous physicians and scientists, PRIMA enters an exciting phase of its development, with a first patient expected to be implanted before year end. With ageing population dynamics, advanced dry-AMD is a leading cause of irreversible vision loss1 with currently estimated over 4 million people without approved treatment option making it a significant unmet medical need.”
The clinical study entitled “Feasibility Study of Compensation for blindness with the PRIMA system in patients with dry age related macular degeneration”, is designed to evaluate the tolerance of PRIMA and to demonstrate the evoked central visual perception among patients who have lost their sight due to atrophic advanced dry-AMD. The study is planned to recruit 5 patients with interim evaluation at 6-month follow-up and longer term follow-up to 36 months. The study will be conducted at Fondation Ophtalmologique Rothschild and Hôpital des Quinze-Vingt in Paris with Dr. Yannick Le Mer, vitreoretinal surgeon and ophthalmologist, as principal investigator.
In parallel, Pixium Vision actively pursues its constructive discussions with the US Food and Drug Administration (FDA), in order to also prepare the feasibility study with PRIMA in the US.
Contacts
Pixium Vision
Didier Laurens, CFO
investors@pixium-vision.com
+33 1 76 21 47 68
@PixiumVision

Media Relations
France: Newcap Media
Annie-Florence Loyer – afloyer@newcap.fr
+33 1 44 71 00 12 / +33 6 88 20 35 59
Léa Jacquin – ljacquin@newcap.fr
+33 1 44 71 94 94
Media Relations International: Image Box PR
Neil Hunter
neil@imageboxpr.co.uk
Tel +44 (0)20 8943 4685

ABOUT PRIMA
PRIMA is a miniaturized new generation implant totally wireless. The PRIMA implant is a micro photovoltaic chip of 2 millimeters and 30 microns thick, PRIMA is equipped with 378 electrodes. Implanted under the retina via a less invasive surgical procedure, implant converts pulsed near infra-red invisible light signal received from the external glasses with an integrated mini-camera into electrical signals transmitted to the brain via the optic nerve. PRIMA is designed to treat retinal dystrophies, particularly aiming to treat advanced atrophic dry-AMD, the most prevalent form of Age-related Macular Degeneration, thanks to miniaturization and aimed to preserve patient’s residual peripheral vision. Prima is also intended to be evaluated at a later stage for treatment of vision loss from Retinitis Pigmentosa.

ABOUT PIXIUM VISION
Pixium Vision’s mission is to create a world of bionic vision for those who have lost their sight, enabling them to regain partial visual perception and greater autonomy. Pixium Vision’s bionic vision systems are associated with a surgical intervention as well as a rehabilitation period. Following the CE mark for its first bionic retinal implant systems, IRIS®II, Pixium Vision has been authorized to start clinical study in Human for PRIMA, a sub-retinal miniaturized wireless implant system. Pixium Vision collaborates closely with academic and research partners spanning across the prestigious Vision research institutions including the Institut de la Vision in Paris, the Hansen Experimental Physics Laboratory at Stanford University, and Moorfields Eye Hospital in London. The company is EN ISO 13485 certified. Pixium Vision is qualified “Entreprise Innovante” par Bpifrance
For more information, please visit: www.pixium-vision.com;
And follow us on: @PixiumVision; www.facebook.com/pixiumvision
www.linkedin.com/company/pixium-vision

Disclaimer:
This press release may expressly or implicitly contain forward-looking statements relating to Pixium Vision and its activity. Such statements are related to known or unknown risks, uncertainties and other factors that could lead actual results, financial conditions, performance or achievements to differ materially from Vision Pixium results, financial conditions, performance or achievements expressed or implied by such forward looking statements.
Pixium Vision provides this press release as of the aforementioned date and does not commit to update forward looking statements contained herein, whether as a result of new information, future events or otherwise.
For a description of risks and uncertainties which could lead to discrepancies between actual results, financial condition, performance or achievements and those contained in the forward-looking statements, please refer to Chapter 4 “Risk Factors” of the company’s Registration Document filed with the AMF under number R16-033 on April 28, 2016 which can be found on the websites of the AMF – AMF (www.amf-france.org) and of Pixium Vision (www.pixium-vision.com).
IRIS® is a trademark of Pixium-Vision SA
Pixium Vision is listed on Euronext Paris (Compartment C). Pixium Vision shares are eligible for the French tax incentivized PEA-PME and FCPI investment vehicles.
Pixium Vision is included in the Euronext CAC All Shares index
Euronext ticker: PIX – ISIN: FR0011950641 – Reuters: PIX.PA – Bloomberg: PIX:FP