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16/05/2019

Oversubscribed round to bring first products powered by enzymatic DNA synthesis technology to market

Paris, May 16, 2019. Sofinnova Partners, a leading European venture capital firm specialized in the life sciences, today announced that its portfolio company DNA Script, an industry leader in the manufacturing of synthetic nucleic acids using proprietary enzymatic technology, has raised $38.5 million in Series B financing. New shareholders LSP and BPIFrance joined the round, alongside existing shareholders Kurma Partners, Idinvest Partners, Illumina Ventures, and M Ventures (the corporate venture arm of Merck KGaA). Sofinnova Partners was the first institutional investor in DNA Script in 2016.

DNA Script is the world’s leading company in manufacturing synthetic nucleic acids using enzymatic technology. Founded in 2014 in Paris, the company aims to accelerate innovation in life sciences and technology delivering rapid, affordable, and high-quality DNA. Sixty years after the discovery of DNA, DNA Script’s revolutionary approach leverages billions of years of nature’s evolution in synthesizing DNA to enable genome scale synthesis.

The company offers a novel biochemical process for DNA and RNA synthesis, a fundamental tool used in biology research. At a recent academic conference, DNA Script presented its ability to synthesize 200nt of DNA with remarkable accuracy. This innovation may be used in numerous applications, including electronic data storage, by leveraging unprecedented capabilities of the molecule to store information. The fundraising allows DNA Script to further develop its unique enzymatic technology and nucleotide chemistry platform, and deliver the promise of same-day results.

Joško Bobanović, Partner at Sofinnova Partners, said: “We are excited that DNA Script, which we have backed from its first round of financing, was able to raise such a significant round. The company continues to deliver on its plan, and is now funded by a group of likeminded investors who support the team’s vision of creating a business that enables new applications for synthetic DNA and RNA in areas including drug discovery and development, agriculture, and industrial and food technologies.”

“Sofinnova Partners has been an excellent partner from a very early stage, just after the inception of our company,” said Thomas Ybert, CEO of DNA Script. “Since then, they have helped us on a daily basis to build DNA Script from the ground up. The team brings a strong expertise in the technology as well as one of the broadest global networks in the industry. Importantly, they also provide unconditional support and coaching to our entrepreneurs – whatever the challenge at hand,” he said.

This new funding reaffirms Sofinnova Partners’ investment strategy in the industrial biotech field, initiated in 2009. As a pioneer in this emerging and rapidly growing sector, Sofinnova Partners has a portfolio of 14 industrial biotech companies, backed through two dedicated funds: Sofinnova Green Seed Fund, which raised €22.5M in 2012, and Sofinnova IB I, which raised €125M in 2017.

About DNA Script
Founded in 2014 in Paris, DNA Script is the world’s leading company in manufacturing de novo synthetic nucleic acids using an enzymatic technology. The company aims to accelerate innovation in life sciences and technology through rapid, affordable and high-quality DNA synthesis. DNA Script’s approach leverages billions of years of natural evolution to enable genome-scale synthesis. The company’s technology has the potential to greatly accelerate the development of new therapeutics, enhanced diagnostics, sustainable chemical production, improved crops and DNA data storage.
www.dnascript.co

About Sofinnova Partners
Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Headquartered in Paris, France, the firm brings together a team of professionals from all over Europe, the U.S. and China. The firm focuses on paradigm shifting technologies alongside visionary entrepreneurs. Sofinnova Partners seeks to invest as a lead or cornerstone investor in seed, start-ups, corporate spin-offs and late stage companies. It has backed nearly 500 companies over more than 45 years, creating market leaders around the globe. Today, Sofinnova Partners has over €2 billion under management.
For more information: http://sofinnovapartners.com/.

Press contact for Sofinnova Partners

International
Kate Barrette
RooneyPartners LLC
kbarrette@rooneyco.com

France
Anne Rein
S&I
+ 33 6 03 35 92 05
anne.rein@strategiesimage.com

28/01/2019

Marcel Lubben appointed as Synvina’s Managing Director

AMSTERDAM, 27 January 2019 – Avantium has retaken full ownership of its YXY plants-to-plastics technology through the purchase of BASF’s shares in the Synvina joint venture. Avantium has appointed Marcel Lubben as Managing Director of Synvina to lead the commercialization of the YXY technology, effective 1 February 2019.

Avantium has regained the intellectual property, people and assets for YXY and has paid BASF €13.7 million for its equity stake in Synvina, as agreed in the Joint Venture Agreement. A net payment of €3.7 million was made for full ownership of the assets acquired by Synvina in the last two years. Avantium is redefining the path to commercialization for the YXY technology, which it developed to catalytically convert plant-based sugars into FDCA (furandicarboxylic acid) and materials such as the new plant-based packaging material PEF (polyethylenefuranoate). PEF can fulfil the demand of leading brands and consumers for next-generation materials that are environmentally friendly and recyclable, and has the potential to be a game changer in the circular economy. Avantium plans to make further announcements on its new strategy to commercialize YXY during the first half of 2019. Experienced chemicals and biotech executive Marcel Lubben is appointed Managing Director of Synvina and will join Victor Vreeken (Chief Op rations Officer) and Willem-Jan Meijer (Financial Director) in Synvina’s leadership team. Lubben worked for 25 years at DSM in various senior business roles, in licensing, corporate venture capital and technology. In his last role, he was president of Reverdia, a joint venture between DSM and Roquette for the production and sale of bio-based succinic acid.

Synvina has become a new business unit of Avantium, alongside the existing Catalysis and Renewable Chemistries business units. Avantium’s renewable chemistry programs include Dawn Technology™, which converts non-food feedstock to industrial sugars and lignin, and the Mekong technology, which transforms sugars into plant-based monoethylene glycol (MEG) used in the production of materials including renewable plastics and polyesters.

Tom van Aken, Chief Executive Officer of Avantium:
“Our belief in YXY and its market potential is rock-solid. Producing FDCA and PEF is a complex innovation process that requires a completely new value chain. I am convinced that the work done so far and the value chain we have built will spur the commercialization of PEF. We have the people, ambition and expertise to bring FDCA and PEF successfully to market, and are very pleased Marcel Lubben will strengthen the team to spearhead this.”

Marcel Lubben, new Managing Director of Synvina:
“Green, innovative materials are essential elements in the global transition to a circular economy. I have been following the progress on YXY for several years and am impressed by how it has been built out to a platform for unique materials with significant performance benefits. I am delighted to assume leadership of this Avantium business unit and bring this important technology to market.”

About Avantium
Avantium is a leading chemical technology development company and a forerunner in renewable chemistry. We develop technologies that enable production of sustainable products from plant-based raw materials. We work in partnership with like-minded companies around the globe to create revolutionary renewable chemistry solutions from invention to commercial scale. We also help clients in catalytic research by providing proprietary systems and services to improve their products and processes. Avantium’s shares are listed on Euronext Amsterdam and Euronext Brussels (symbol: AVTX). Our offices and headquarters are in Amsterdam, the Netherlands.

For more information:
Caroline van Reedt Dortland, Director Communications, Avantium
+31-20-586-0110 / +31-6-1340-0179
caroline.vanreedt-dortland@avantium.com

 

Summary Curriculum Vitae – Marcel Lubben

Previous positions
Jan 2014 to date: President at Reverdia (JV of DSM with Roquette)
Aug 2012 – Dec 2013: Vice President Bio-based Chemicals & Materials at DSM
Jan 2009 – Jul 2012: Vice President at DSM Innovation Center
May 2007 – Dec 2008: Vice President Marketing & Sales at DSM Biologics
April 2004 – April 2007: Head of BD Europe & Asia & Global Licensing Manager at DSM Biologics
Aug 2000 – March 2004: Business Development / Sales Manager at DSM Pharma Chemicals
Dec 1994 – July 2000: Project & Program Manager at DSM Research

Education
1990 – 1994 PhD Organic Chemistry at the University of Groningen, Netherlands
1985 – 1990 Major in Chemistry at the University of Groningen, Netherlands

28/01/2019

Marcel Lubben appointed as Synvina’s Managing Director

AMSTERDAM, 27 January 2019 – Avantium has retaken full ownership of its YXY plants-to-plastics technology through the purchase of BASF’s shares in the Synvina joint venture. Avantium has appointed Marcel Lubben as Managing Director of Synvina to lead the commercialization of the YXY technology, effective 1 February 2019.

Avantium has regained the intellectual property, people and assets for YXY and has paid BASF €13.7 million for its equity stake in Synvina, as agreed in the Joint Venture Agreement. A net payment of €3.7 million was made for full ownership of the assets acquired by Synvina in the last two years. Avantium is redefining the path to commercialization for the YXY technology, which it developed to catalytically convert plant-based sugars into FDCA (furandicarboxylic acid) and materials such as the new plant-based packaging material PEF (polyethylenefuranoate). PEF can fulfil the demand of leading brands and consumers for next-generation materials that are environmentally friendly and recyclable, and has the potential to be a game changer in the circular economy. Avantium plans to make further announcements on its new strategy to commercialize YXY during the first half of 2019. Experienced chemicals and biotech executive Marcel Lubben is appointed Managing Director of Synvina and will join Victor Vreeken (Chief Op rations Officer) and Willem-Jan Meijer (Financial Director) in Synvina’s leadership team. Lubben worked for 25 years at DSM in various senior business roles, in licensing, corporate venture capital and technology. In his last role, he was president of Reverdia, a joint venture between DSM and Roquette for the production and sale of bio-based succinic acid.

Synvina has become a new business unit of Avantium, alongside the existing Catalysis and Renewable Chemistries business units. Avantium’s renewable chemistry programs include Dawn Technology™, which converts non-food feedstock to industrial sugars and lignin, and the Mekong technology, which transforms sugars into plant-based monoethylene glycol (MEG) used in the production of materials including renewable plastics and polyesters.

Tom van Aken, Chief Executive Officer of Avantium:
“Our belief in YXY and its market potential is rock-solid. Producing FDCA and PEF is a complex innovation process that requires a completely new value chain. I am convinced that the work done so far and the value chain we have built will spur the commercialization of PEF. We have the people, ambition and expertise to bring FDCA and PEF successfully to market, and are very pleased Marcel Lubben will strengthen the team to spearhead this.”

Marcel Lubben, new Managing Director of Synvina:
“Green, innovative materials are essential elements in the global transition to a circular economy. I have been following the progress on YXY for several years and am impressed by how it has been built out to a platform for unique materials with significant performance benefits. I am delighted to assume leadership of this Avantium business unit and bring this important technology to market.”

About Avantium
Avantium is a leading chemical technology development company and a forerunner in renewable chemistry. We develop technologies that enable production of sustainable products from plant-based raw materials. We work in partnership with like-minded companies around the globe to create revolutionary renewable chemistry solutions from invention to commercial scale. We also help clients in catalytic research by providing proprietary systems and services to improve their products and processes. Avantium’s shares are listed on Euronext Amsterdam and Euronext Brussels (symbol: AVTX). Our offices and headquarters are in Amsterdam, the Netherlands.

For more information:
Caroline van Reedt Dortland, Director Communications, Avantium
+31-20-586-0110 / +31-6-1340-0179
caroline.vanreedt-dortland@avantium.com

 

Summary Curriculum Vitae – Marcel Lubben

Previous positions
Jan 2014 to date: President at Reverdia (JV of DSM with Roquette)
Aug 2012 – Dec 2013: Vice President Bio-based Chemicals & Materials at DSM
Jan 2009 – Jul 2012: Vice President at DSM Innovation Center
May 2007 – Dec 2008: Vice President Marketing & Sales at DSM Biologics
April 2004 – April 2007: Head of BD Europe & Asia & Global Licensing Manager at DSM Biologics
Aug 2000 – March 2004: Business Development / Sales Manager at DSM Pharma Chemicals
Dec 1994 – July 2000: Project & Program Manager at DSM Research

Education
1990 – 1994 PhD Organic Chemistry at the University of Groningen, Netherlands
1985 – 1990 Major in Chemistry at the University of Groningen, Netherlands

17/01/2019

PARIS, January 17, 2019 – HighLife SAS, a medtech company focused on the development of a unique transcatheter mitral valve replacement (« TMVR ») system to treat patients suffering from mitral regurgitation, announced today it had closed a € 32 million round of equity financing.

Proceeds from the financing will be used to complete the development of the company’s transcatheter transseptal mitral valve implantation program, which includes regulatory approval trials in Europe to obtain the CE mark and a first clinical trial in the U.S. via an Early Feasibility Study (EFS).

The Series B round was co-led by U.S. Venture Partners (« USVP ») and Andera Partners, with Sectoral Asset Management and Jose Calle Gordo, Chairman of the company, joining the round. Sofinnova Partners, which led the Series A round, continues to support the company and participated in this latest round of financing. Bryan Garnier & Co (Paris) acted as private placement agent for this financing.

« I am very pleased to announce the closing of the Series B financing round, which is essential to funding the development program of the company. The funds will support our mid-term strategy with a pivotal trial in Europe and a clinical trial in the U.S. » said Georg Börtlein, HighLife’s CEO and Founder. « We remain committed to bringing the best treatment option for the broadest patient population to market. Over the long term, we want to address any limitation that the field of TMVR faces and we will continue working towards that goal. »

Based in Paris, France, HighLife was founded in 2010 by Mr. Börtlein. Following Sofinnova Partners’ investment in HighLife in 2017, the venture capital firm’s Managing Partner Antoine Papiernik joined HighLife’s Board. In December 2017, Mr. Calle Gordo, most recently CEO of Biosensors and a longtime specialist in cardiovascular devices, was named chairman. In May 2018, Dr. Martin Rothman, interventional cardiologist and retired Chief Medical Officer of Medtronic, joined as CMO and member of the board. USVP will be represented on the board by Casey Tansey, while Andera Partners will be represented by Olivier Litzka.

« HighLife’s creative approach has already generated convincing data in this competitive field of mitral valve replacement. I am confident that the experienced management team will bring this technology to the level where it becomes an undisputed treatment option for mitral valve regurgitation patients, » said Mr. Tansey, General Partner at USVP.

« With our already solid experience in the structural heart field, we are very happy to contribute to and support HighLife. The exceptional level of collective experience brought together with the existing team and the new investors is a favorable omen for HighLife’s success » said Mr. Litzka, partner at Andera Partners.

« Sofinnova is thrilled to continue supporting HighLife through this new financing, which gives the company the means to demonstrate the clinical importance of its transseptal product, » said Mr. Papiernik.

« We are very proud that we could attract highly experienced and internationally recognized investors like USVP, Andera and Sectoral Asset Management. This speaks for the credibility of our development program, management team and the potential of our technology to serve a major yet underserved clinical need, » said Mr Calle Gordo.

HighLife’s technology targets optimal treatment outcomes as it can be delivered transseptally via the femoral vein in a reversible manner, and self-locates inside the native annulus. The transseptal route is the preferred route for both interventionists and patients, as it avoids any surgery.

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 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 or cornerstone investor in seed, start-ups, corporate spin-offs and late stage companies. It 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.
For more information: www.sofinnova.fr.

About USVP
U.S. Venture Partners (USVP) is a leading Silicon Valley venture capital firm, partnering with entrepreneurs to transform their ideas into world-changing companies. USVP has invested in over 400 companies spanning three decades, including: Box, Castlight Health, Check Point Software, Concur, GoPro, Guidewire Software, HotelTonight, Imperva, InsideSales.com, Inspire Medical, Intersect ENT, Omada Health, OncoMed, Prevoty, SanDisk, Sun Microsystems, Trunk Club, Trusteer, Yammer, and Zerto. USVP focuses on early-stage start-ups that transform cybersecurity, enterprise software, consumer mobile and e-commerce, and healthcare. The USVP team consists of former entrepreneurs, technologists, corporate executives, and financial professionals who assist with strategy, scaling, team building, product development, and business development. USVP is based in Menlo Park, California. More information can be found at www.usvp.com.

About Andera Partners
Andera Partners (previously Edmond de Rothschild Investment Partners) is a leading investor in minority investments into privately owned companies. The Management Company employs 56 people and has more than €2 billion under management. Its Life Sciences team of 12 professionals brings together deep experience both in the Life Science industry and in private equity and venture capital. The team has raised more than €800 million through its BioDiscovery franchise and is currently investing out of its €345 million BioDiscovery 5 fund, both in biotech and medtech, and all over Europe and the US. For more information, please visit www.anderapartners.com.

About Sectoral Asset Management
Sectoral Asset Management is an established global healthcare specialist. Sectoral leverages its expertise and capabilities to capture significant value creation across both public and private companies. More information about the firm and its track record is available at www.sectoral.com.

For more information, please contact:

HIGHLIFE Contact International Contact France
www.highlifemedical.com
Marion Janic Anne Rein
Tel : +33 (0)1 72 32 21 15 Tel: +1 (212) 223 4017 Tel: +33 6 03 35 92 05
info@highlifemed.com
mjanic@rooneyco.com
anne.rein@strategiesimage.com

17/01/2019

PARIS, January 17, 2019 – HighLife SAS, a medtech company focused on the development of a unique transcatheter mitral valve replacement (« TMVR ») system to treat patients suffering from mitral regurgitation, announced today it had closed a € 32 million round of equity financing.

Proceeds from the financing will be used to complete the development of the company’s transcatheter transseptal mitral valve implantation program, which includes regulatory approval trials in Europe to obtain the CE mark and a first clinical trial in the U.S. via an Early Feasibility Study (EFS).

The Series B round was co-led by U.S. Venture Partners (« USVP ») and Andera Partners, with Sectoral Asset Management and Jose Calle Gordo, Chairman of the company, joining the round. Sofinnova Partners, which led the Series A round, continues to support the company and participated in this latest round of financing. Bryan Garnier & Co (Paris) acted as private placement agent for this financing.

« I am very pleased to announce the closing of the Series B financing round, which is essential to funding the development program of the company. The funds will support our mid-term strategy with a pivotal trial in Europe and a clinical trial in the U.S. » said Georg Börtlein, HighLife’s CEO and Founder. « We remain committed to bringing the best treatment option for the broadest patient population to market. Over the long term, we want to address any limitation that the field of TMVR faces and we will continue working towards that goal. »

Based in Paris, France, HighLife was founded in 2010 by Mr. Börtlein. Following Sofinnova Partners’ investment in HighLife in 2017, the venture capital firm’s Managing Partner Antoine Papiernik joined HighLife’s Board. In December 2017, Mr. Calle Gordo, most recently CEO of Biosensors and a longtime specialist in cardiovascular devices, was named chairman. In May 2018, Dr. Martin Rothman, interventional cardiologist and retired Chief Medical Officer of Medtronic, joined as CMO and member of the board. USVP will be represented on the board by Casey Tansey, while Andera Partners will be represented by Olivier Litzka.

« HighLife’s creative approach has already generated convincing data in this competitive field of mitral valve replacement. I am confident that the experienced management team will bring this technology to the level where it becomes an undisputed treatment option for mitral valve regurgitation patients, » said Mr. Tansey, General Partner at USVP.

« With our already solid experience in the structural heart field, we are very happy to contribute to and support HighLife. The exceptional level of collective experience brought together with the existing team and the new investors is a favorable omen for HighLife’s success » said Mr. Litzka, partner at Andera Partners.

« Sofinnova is thrilled to continue supporting HighLife through this new financing, which gives the company the means to demonstrate the clinical importance of its transseptal product, » said Mr. Papiernik.

« We are very proud that we could attract highly experienced and internationally recognized investors like USVP, Andera and Sectoral Asset Management. This speaks for the credibility of our development program, management team and the potential of our technology to serve a major yet underserved clinical need, » said Mr Calle Gordo.

HighLife’s technology targets optimal treatment outcomes as it can be delivered transseptally via the femoral vein in a reversible manner, and self-locates inside the native annulus. The transseptal route is the preferred route for both interventionists and patients, as it avoids any surgery.

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 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 or cornerstone investor in seed, start-ups, corporate spin-offs and late stage companies. It 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.
For more information: www.sofinnova.fr.

About USVP
U.S. Venture Partners (USVP) is a leading Silicon Valley venture capital firm, partnering with entrepreneurs to transform their ideas into world-changing companies. USVP has invested in over 400 companies spanning three decades, including: Box, Castlight Health, Check Point Software, Concur, GoPro, Guidewire Software, HotelTonight, Imperva, InsideSales.com, Inspire Medical, Intersect ENT, Omada Health, OncoMed, Prevoty, SanDisk, Sun Microsystems, Trunk Club, Trusteer, Yammer, and Zerto. USVP focuses on early-stage start-ups that transform cybersecurity, enterprise software, consumer mobile and e-commerce, and healthcare. The USVP team consists of former entrepreneurs, technologists, corporate executives, and financial professionals who assist with strategy, scaling, team building, product development, and business development. USVP is based in Menlo Park, California. More information can be found at www.usvp.com.

About Andera Partners
Andera Partners (previously Edmond de Rothschild Investment Partners) is a leading investor in minority investments into privately owned companies. The Management Company employs 56 people and has more than €2 billion under management. Its Life Sciences team of 12 professionals brings together deep experience both in the Life Science industry and in private equity and venture capital. The team has raised more than €800 million through its BioDiscovery franchise and is currently investing out of its €345 million BioDiscovery 5 fund, both in biotech and medtech, and all over Europe and the US. For more information, please visit www.anderapartners.com.

About Sectoral Asset Management
Sectoral Asset Management is an established global healthcare specialist. Sectoral leverages its expertise and capabilities to capture significant value creation across both public and private companies. More information about the firm and its track record is available at www.sectoral.com.

For more information, please contact:

HIGHLIFE Contact International Contact France
www.highlifemedical.com
Marion Janic Anne Rein
Tel : +33 (0)1 72 32 21 15 Tel: +1 (212) 223 4017 Tel: +33 6 03 35 92 05
info@highlifemed.com
mjanic@rooneyco.com
anne.rein@strategiesimage.com

17/01/2019

Serial biotech entrepreneur joins leading small molecule tau modulation company

Lausanne, SWITZERLAND and Cambridge, MA, USA, January 17, 2019 – Asceneuron, an emerging leader in the development of orally bioavailable modulators of tau pathology for the treatment of neurodegenerative diseases, today announces the appointment of Peter Van Vlasselaer as Chair of the Board of Directors.

Peter Van Vlasselaer, PhD has over 20 years executive and entrepreneurial experience in the biotech industry. He was most recently the Founder, President and Chief Executive Officer of ARMO Biosciences, Inc. which shortly after its public offering (Nasdaq: ARMO) was acquired by Eli Lilly. Prior to this, he was President and Chief Executive Officer of iPierian (acquired by BMS), ARRESTO (acquired by Gilead) and AVIDIA (acquired by AMGEN). In addition to founding ARMO, Dr. Van Vlasselaer was the founder of ARRESTO, co-founder of TrueNorth (acquired by Bioverativ) and was a member of the start-up teams of InterMune (ITMN) and Dendreon (DNDN). He currently serves on the boards of BLADE Therapeutics, Comet Therapeutics and RGENIX. Dr. Van Vlasselaer has a degree in Zoology and a PhD in Immunology from the Catholic University of Leuven, Belgium. He was a Post-Doctoral Fellow in the Division of Immunology and Rheumatology at Stanford University Medical School and DNAX Research Institute. Dr. Van Vlasselaer has authored several peer reviewed scientific publications and book chapters and he is an inventor on multiple patents.

Dirk Beher, Chief Executive Officer and Founder of Asceneuron, commented:
“We are delighted to welcome Peter Van Vlasselaer to the Board. His extensive experience in all aspects of biotechnology, drug and corporate development will be invaluable as the Company progresses its orally-bioavailable tau modifiers through clinical development. With Peter’s addition to the board, our expanded US presence, and commitment to tau, Asceneuron is well positioned to revolutionize the treatment of neurodegenerative diseases.”

Peter Van Vlasselaer, new Chairman of Asceneuron, added:
“There is a strong industry and scientific interest in tau approaches to Alzheimer’s disease and orphan tauopathies such as progressive supranuclear palsy (PSP). I believe Asceneuron’s technology represents a significant opportunity in the next generation of drugs that could potentially transform the treatment of PSP and other tau-related neurodegenerative diseases. These disorders have devastating outcomes for patients and their families. I look forward to maximising the potential of these innovative new treatments and support the Company through this important phase of growth.”

Asceneuron’s lead program ASN120290 is a small molecule inhibitor of the enzyme O-GlcNAcase. Based on its unique mechanism of action, ASN120290 has the potential to become a first in class treatment for progressive supranuclear palsy (PSP) and other tau-related dementias.
The company recently announced the appointment of CNS specialist Dr Thomas C. Wessel as Chief Medical Officer. A clinical trial is ongoing with ASN120290 to quantify target engagement in the human brain using positron emission tomography (PET) the results of which will guide dose selection for an efficacy trial in PSP planned for later this year.

For further information, please contact:
Asceneuron
Dirk Beher, CEO
Email: asce-contact@asceneuron.com

Optimum Strategic Communications
Mary Clark, Supriya Mathur
Tel: +44 203 922 0891
Email: asceneuron@optimumcomms.com

About Asceneuron
Asceneuron is an emerging, clinical stage biotech company excelling in the development of orally bioavailable therapeutics for debilitating neurodegenerative disorders with high unmet medical need, such as orphan tauopathies, Alzheimer’s and Parkinson’s diseases. The lead program ASN120290, an O-GlcNAcase inhibitor, is being developed for the orphan tauopathy progressive supranuclear palsy (PSP). Asceneuron has completed a randomized, double-blind, placebo-controlled phase I study to assess the safety and tolerability of single and multiple doses of orally administered ASN120290. Asceneuron is a privately held company financed by a strong syndicate of investors consisting of Sofinnova Partners, M Ventures, SR One, Johnson & Johnson Innovation – JJDC, Inc. (JJDC) and Kurma Partners. For more information, please visit www.asceneuron.com.

About ASN120290
Asceneuron’s lead program ASN120290, an O-GlcNAcase inhibitor, is being developed for the orphan tauopathy progressive supranuclear palsy (PSP) and was recently granted Orphan Drug Designation by the US FDA for the treatment of PSP. ASN120290 has recently completed a randomized, double-blind, placebo-controlled phase I study to assess its safety and tolerability of single and multiple doses in healthy young and elderly volunteers. Data from that study were presented at the Alzheimer’s Association International Conference (AAIC) in Chicago July 22-26, 2018.

About Progressive Supranuclear Palsy (PSP)
PSP, also known as Steele-Richardson-Olszewski syndrome, is a rapidly progressing neurodegenerative disorder. PSP is often misdiagnosed because it is relatively rare and certain symptoms are similar to Parkinson’s disease. However, PSP is much more common than previously believed. Its prevalence is about three to six people per 100,000 individuals. Symptoms generally appear in the 60s-70s but can affect people from the age of 40 onwards. There are currently no treatments available to cure this disease.

17/01/2019

Serial biotech entrepreneur joins leading small molecule tau modulation company

Lausanne, SWITZERLAND and Cambridge, MA, USA, January 17, 2019 – Asceneuron, an emerging leader in the development of orally bioavailable modulators of tau pathology for the treatment of neurodegenerative diseases, today announces the appointment of Peter Van Vlasselaer as Chair of the Board of Directors.

Peter Van Vlasselaer, PhD has over 20 years executive and entrepreneurial experience in the biotech industry. He was most recently the Founder, President and Chief Executive Officer of ARMO Biosciences, Inc. which shortly after its public offering (Nasdaq: ARMO) was acquired by Eli Lilly. Prior to this, he was President and Chief Executive Officer of iPierian (acquired by BMS), ARRESTO (acquired by Gilead) and AVIDIA (acquired by AMGEN). In addition to founding ARMO, Dr. Van Vlasselaer was the founder of ARRESTO, co-founder of TrueNorth (acquired by Bioverativ) and was a member of the start-up teams of InterMune (ITMN) and Dendreon (DNDN). He currently serves on the boards of BLADE Therapeutics, Comet Therapeutics and RGENIX. Dr. Van Vlasselaer has a degree in Zoology and a PhD in Immunology from the Catholic University of Leuven, Belgium. He was a Post-Doctoral Fellow in the Division of Immunology and Rheumatology at Stanford University Medical School and DNAX Research Institute. Dr. Van Vlasselaer has authored several peer reviewed scientific publications and book chapters and he is an inventor on multiple patents.

Dirk Beher, Chief Executive Officer and Founder of Asceneuron, commented:
“We are delighted to welcome Peter Van Vlasselaer to the Board. His extensive experience in all aspects of biotechnology, drug and corporate development will be invaluable as the Company progresses its orally-bioavailable tau modifiers through clinical development. With Peter’s addition to the board, our expanded US presence, and commitment to tau, Asceneuron is well positioned to revolutionize the treatment of neurodegenerative diseases.”

Peter Van Vlasselaer, new Chairman of Asceneuron, added:
“There is a strong industry and scientific interest in tau approaches to Alzheimer’s disease and orphan tauopathies such as progressive supranuclear palsy (PSP). I believe Asceneuron’s technology represents a significant opportunity in the next generation of drugs that could potentially transform the treatment of PSP and other tau-related neurodegenerative diseases. These disorders have devastating outcomes for patients and their families. I look forward to maximising the potential of these innovative new treatments and support the Company through this important phase of growth.”

Asceneuron’s lead program ASN120290 is a small molecule inhibitor of the enzyme O-GlcNAcase. Based on its unique mechanism of action, ASN120290 has the potential to become a first in class treatment for progressive supranuclear palsy (PSP) and other tau-related dementias.
The company recently announced the appointment of CNS specialist Dr Thomas C. Wessel as Chief Medical Officer. A clinical trial is ongoing with ASN120290 to quantify target engagement in the human brain using positron emission tomography (PET) the results of which will guide dose selection for an efficacy trial in PSP planned for later this year.

For further information, please contact:
Asceneuron
Dirk Beher, CEO
Email: asce-contact@asceneuron.com

Optimum Strategic Communications
Mary Clark, Supriya Mathur
Tel: +44 203 922 0891
Email: asceneuron@optimumcomms.com

About Asceneuron
Asceneuron is an emerging, clinical stage biotech company excelling in the development of orally bioavailable therapeutics for debilitating neurodegenerative disorders with high unmet medical need, such as orphan tauopathies, Alzheimer’s and Parkinson’s diseases. The lead program ASN120290, an O-GlcNAcase inhibitor, is being developed for the orphan tauopathy progressive supranuclear palsy (PSP). Asceneuron has completed a randomized, double-blind, placebo-controlled phase I study to assess the safety and tolerability of single and multiple doses of orally administered ASN120290. Asceneuron is a privately held company financed by a strong syndicate of investors consisting of Sofinnova Partners, M Ventures, SR One, Johnson & Johnson Innovation – JJDC, Inc. (JJDC) and Kurma Partners. For more information, please visit www.asceneuron.com.

About ASN120290
Asceneuron’s lead program ASN120290, an O-GlcNAcase inhibitor, is being developed for the orphan tauopathy progressive supranuclear palsy (PSP) and was recently granted Orphan Drug Designation by the US FDA for the treatment of PSP. ASN120290 has recently completed a randomized, double-blind, placebo-controlled phase I study to assess its safety and tolerability of single and multiple doses in healthy young and elderly volunteers. Data from that study were presented at the Alzheimer’s Association International Conference (AAIC) in Chicago July 22-26, 2018.

About Progressive Supranuclear Palsy (PSP)
PSP, also known as Steele-Richardson-Olszewski syndrome, is a rapidly progressing neurodegenerative disorder. PSP is often misdiagnosed because it is relatively rare and certain symptoms are similar to Parkinson’s disease. However, PSP is much more common than previously believed. Its prevalence is about three to six people per 100,000 individuals. Symptoms generally appear in the 60s-70s but can affect people from the age of 40 onwards. There are currently no treatments available to cure this disease.

11/01/2019

• L’implant est bien toléré et préserve l’acuité visuelle périphérique résiduelle.
• Tous les patients perçoivent de la lumière au centre de leur champ visuel.
• La majorité des patients identifie des signaux complexes, des lettres ou des séquences de lettres.
• Ces résultats intérimaires positifs permettent de préparer l’étude pivot européenne multicentrique.

Paris, 8 janvier 2019 – 7h00 CET – Pixium Vision (FR0011950641 – PIX), société bioélectronique qui développe des systèmes de vision bionique innovants pour permettre aux patients ayant perdu la vue de vivre de façon plus autonome, annonce que son dispositif sous-rétinien PRIMA a atteint les objectifs de l’étude de faisabilité1 après 6 mois de suivi et de rééducation de patients atteints de la forme sèche de Dégénérescence Maculaire liée à l’Age (DMLA).
Khalid Ishaque, Directeur Général de Pixium Vision, commente : « Nous sommes particulièrement satisfaits du remarquable succès obtenu avec le dispositif PRIMA chez les patients atteints de la forme sèche de la DMLA. Ces résultats dépassent nos attentes. Ainsi, chez des patients ayant totalement perdu leur vision centrale, PRIMA permet à la majorité d’entre eux de commencer à identifier correctement des signaux et des lettres. Nous espérons que ces premiers résultats très positifs attirent les candidats éligibles pour l’étude de faisabilité PRIMA2 en cours de recrutement aux Etats-Unis. Après avoir franchi avec succès cette étape majeure, nous préparons dès maintenant l’étude clinique pivot européenne, un pas de plus vers le marquage CE de PRIMA. »
Les résultats cliniques intérimaires à 6 mois de PRIMA, micropuce photovoltaïque sans fil, chez les patients atteints de la forme sèche de DMLA, montrent :
• PRIMA peut être implanté de manière sûre sous la macula atrophique, et préserve l’acuité visuelle périphérique résiduelle, mesurée dans des conditions normalisées3.
• Restitution d’une perception lumineuse, chez tous les patients, dans la zone centrale de la rétine qui avait perdu toute activité visuelle, objectivée par des tests cliniques normalisés4.
1 Study of Compensation for Blindness with the PRIMA System in Patients with Dry Age-Related Macular Degeneration (PRIMA FS) https://www.clinicaltrials.gov/ct2/show/NCT03333954
2 Feasibility Study of Compensation for Blindness with the PRIMA System in Patients with Atrophic Dry Age Related Macular Degeneration (PRIMA-FS-US) https://clinicaltrials.gov/ct2/show/NCT03392324
3 Mesuré par le test ETDRS
4 Mesuré par les tests de micropérimétrie et de mesure du champ visuel Octopus

• Une bonne tolérance de l’implant sans effets indésirables sévères liés à l’implant. L’implant ne bouge pas après cicatrisation naturelle de la rétine, et reste stable chez tous les patients.
• L’identification de signaux, de chiffres et de lettres chez la majorité des patients. La rapidité et la justesse des identifications s’améliorent constamment au cours de la phase de rééducation.
• La mesure de l’acuité visuelle prosthétique centrale 5 jusqu’à 20/460 (LogMAR 1,37), chez des patients n’ayant plus aucune perception visuelle centrale naturelle. Les résultats d’acuité visuelle obtenus sont les meilleurs jamais atteints au regard des résultats publiés avec les technologies actuelles de vision prothétique.
Pixium Vision prépare dès à présent une importante étude pivot européenne multicentrique, phase nécessaire à l’obtention du marquage CE.
Prochain évènement : 8 février 2019 – Publication des résultats annuels 2018
5 Sur la base du test d’acuité visuelle LandoltC

Contacts
Pixium Vision
Didier Laurens, CFO
investors@pixium-vision.com
+33 1 76 21 47 68

Relations Presse
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

Relations Investisseurs USA
ICR
David Clair
david.clair@icrinc.com
+1 646 277 12 66

À PROPOS DE PRIMA
PRIMA est un implant miniature de nouvelle génération totalement sans fil ni connexion. Micro-puce photovoltaïque de 2 millimètres et 30 microns d’épaisseur, PRIMA est constitué de 378 électrodes. Implanté sous la rétine par chirurgie peu invasive, PRIMA convertit le signal infra-rouge, reçu d’un projecteur miniaturisé fixé sur une paire de lunette munie d’une mini-camera, en un signal électrique transmis au cerveau par l’intermédiaire du nerf optique. PRIMA est destiné au traitement des dystrophies rétiniennes. De par sa taille, conçue pour préserver la vision résiduelle des patients, PRIMA est particulièrement adapté à la prise en charge de la forme sèche de DMLA, la forme la plus fréquente de cette pathologie. PRIMA est également susceptible d’être développé dans la rétinite pigmentaire.

A PROPOS DE LA DEGENERESCENCE MACULAIRE LIEE A L’AGE (DMLA)
La dégénérescence maculaire liée à l’âge est la première cause de perte sévère de la vision et de cécité chez les personnes de plus de 65 ans en Europe et aux Etats-Unis. Selon les estimations1, la DMLA affecte environ 196 millions de personnes dans le monde, un chiffre en constante augmentation du fait du vieillissement de la population. Près de 1 000 nouveaux cas sont diagnostiqués quotidiennement en Europe et aux Etats-Unis. Il existe deux formes de DMLA : une forme humide pour laquelle les traitements de type anti-VEGF permettent de ralentir la progression de la maladie ; et une forme sèche, plus fréquente, pour laquelle il n’existe actuellement aucun traitement disponible. Plus de 5 millions de personnes souffrent d’une forme avancée de DMLA sèche, ou Atrophie Géographique (GA). Les personnes atteintes de cette pathologie rétinienne perdent graduellement leur vision centrale (responsable de la vision précise et détaillée comme la lecture et la reconnaissance des visages) du fait de la mort des photorécepteurs.

À PROPOS DE PIXIUM VISION
La mission de Pixium Vision est de créer un monde de vision bionique pour permettre à ceux qui ont perdu la vue de récupérer en partie leur perception visuelle et gagner en autonomie. Les systèmes de vision bionique de Pixium Vision sont associés à une intervention chirurgicale et à une période de rééducation.
Pixium Vision conduit des études cliniques de faisabilité avec PRIMA, son implant sous-rétinien miniaturisé et sans fil, chez des patients qui ont perdu la vue par dégénérescence rétinienne liée à la forme sèche de la Dégénérescence Maculaire Liée à l’Age (DMLA). Pixium Vision travaille en étroite collaboration avec des partenaires académiques de renommée mondiale tels que, l’Université Stanford en Californie, l’Institut de la Vision à Paris, le Moorfields Eye Hospital de Londres et l’Institute of Ocular Microsurgery (IMO) de Barcelone et l’UPMC de Pittsburgh (USA). La société est certifiée EN ISO 13485. Pixium Vision a reçu la qualification « Entreprise Innovante » par Bpifrance
1 Wong, W. L., Su, X., Li, X., Cheung, C. M. G., Klein, R., Cheng, C. Y., & Wong, T. Y. (2014). Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. The Lancet Global Health, 2(2), e106-e116 (https://www.thelancet.com/journals/langlo/article/PIIS2214-109X(13)70145-1/fulltext)

Pour plus d’informations : http://www.pixium-vision.com/fr
Suivez-nous sur @PixiumVision; www.facebook.com/pixiumvision
www.linkedin.com/company/pixium-vision

Avertissement :
Le présent communiqué contient de manière implicite ou expresse certaines déclarations prospectives relatives à Pixium Vision et à son activité. Ces déclarations dépendent de certains risques connus ou non, d’incertitudes, ainsi que d’autres facteurs, qui pourraient conduire à ce que les résultats réels, les conditions financières, les performances ou réalisations de Pixium Vision diffèrent significativement des résultats, conditions financières, performances ou réalisations exprimés ou sous-entendus dans ces déclarations prospectives. Pixium Vision émet ce communiqué à la présente date et ne s’engage pas à mettre à jour les déclarations prospectives qui y sont contenues, que ce soit par suite de nouvelles informations, événements futurs ou autres. Pour une description des risques et incertitudes de nature à entraîner une différence entre les résultats réels, les conditions financières, les performances ou les réalisations de Pixium Vision et ceux contenus dans les déclarations prospectives, veuillez-vous référer au chapitre 4 « Facteurs de risques » du document de référence de la Société enregistré auprès de l’Autorité des marchés financiers sous le numéro R.18-085 le 26 mars 2018, lequel peut être consulté sur les sites de l’Autorité des marchés – AMF (www.amf-france.org) et de Pixium Vision (www.pixium-vision.com).
IRIS® est une marque déposée de Pixium-Vision SA
Pixium Vision est coté sur Euronext (Compartiment C) à Paris
ISIN: FR0011950641 ; Mnemo: PIX
Pixium Vision est intégré à l’indice Euronext CAC All Shares
Les actions Pixium Vision sont éligibles PEA-PME et FCPI

11/01/2019

• Implant is well tolerated while preserving residual peripheral visual acuity
• All subjects report light perception in their central visual field
• Majority of patients identifying complex patterns, letters or letter sequences
• Interim positive data enables to prepare the European multi Centre pivotal study

Paris, France. January 8, 2019 – 7.00 AM CET – Pixium Vision (FR0011950641 – PIX), a bioelectronics company developing innovative bionic vision systems to enable patients who have lost their sight to lead more independent lives, announces its subretinal PRIMA system met the endpoints of the feasibility study1,at interim 6 months follow-up after implantation and rehabilitation for patients with advanced dry Age-related Macular Degeneration (AMD).
Khalid Ishaque, Chief Executive Officer Pixium Vision, stated: “We are very pleased with the remarkable results being achieved with PRIMA system with dry AMD patients. These results exceeded our initial expectations. For patients who had completely lost their central vision, PRIMA enabled majority of them to begin to correctly identify patterns and letters. We expect these remarkable first results to attract also eligible candidates for the PRIMA feasibility study2 recruiting in the USA. Following this successfully significant milestone, we look forward to the pivotal clinical phase in Europe and subsequent CE Mark for PRIMA.”

The interim clinical results at 6 months with PRIMA, a wireless sub-retinal photovoltaic microchip, in patients with advanced dry-AMD, show:
• PRIMA can be safely implanted under the atrophic macula while preserving the residual natural peripheral visual acuity, measured under standardized conditions3.
1 Study of Compensation for Blindness with the PRIMA System in Patients with Dry Age-Related Macular Degeneration (PRIMA FS) https://www.clinicaltrials.gov/ct2/show/NCT03333954
2 Feasibility Study of Compensation for Blindness with the PRIMA System in Patients with Atrophic Dry Age Related Macular Degeneration (PRIMA-FS-US) https://clinicaltrials.gov/ct2/show/NCT03392324
3 Standard vision tests used via ETDRS

• Successful elicitation of light perception in the central retinal area in all subjects who had no remaining central visual activity, validated by standardized clinical vision measures and tests4.
• The implant is well tolerated, with no device-related serious adverse events. Implant does not move after natural retina healing and remains stable in all patients.
• Identification of patterns, numbers, or letters, in a majority of the patients. The speed and accuracy of identifications improved continuously during the rehabilitation phase.
• Central prosthetic visual acuity5,measured up to 20/460 (LogMAR 1.37) within the former scotoma with no remaining natural central vision. The achieved prosthetic visual acuity to date is the best among those published from current visual prosthetic technologies.
Pixium Vision is preparing the larger European multicenter pivotal study required for the CE-mark.
Next event: 2018 Annual Results and Cash position – February 8th, 2019
4 Standard vision tests used via microperimetry and Octopus visual field test
5 Based on LandoltC visual acuity test

Contacts
Pixium Vision
Didier Laurens, CFO
investors@pixium-vision.com
+33 1 76 21 47 68

Media Relations
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

US Investor Relations
ICR
David Clair
david.clair@icrinc.com
+1 646 277 12 66

ABOUT PRIMA
PRIMA is a new generation miniaturized and totally wireless sub-retinal implant. The 2×2 millimeters wide and 30 microns thick photovoltaic chip contains 378 electrodes. Implanted under the retina via a minimally invasive surgical procedure, it acts like an array of tiny solar panel powered by pulsed near infrared light projected from a miniature projector integrated into augmented reality glasses, along with a mini-camera. PRIMA is designed to restore sight in patients blinded by retinal dystrophies – a very significant unmet medical need. The target population includes patients with atrophic dry Age-related Macular Degeneration (dry AMD), and also Retinitis Pigmentosa (RP). In addition to a clinical trial with five atrophic dry-AMD patients in France, PRIMA is approved for a similar five-patients study in USA.

ABOUT AGE-RELATED MACULAR DEGENERATION (AMD)
Age-related macular degeneration is the leading cause of severe vision loss and legal blindness in people over the age of 65 in North America and Europe. The global impact is significant with current projected estimates1 for people living with AMD of around 196 million people worldwide and expected rapid growth due to ageing population. Around 1000 new patients are diagnosed everyday just in Europe and USA. There are two forms of advanced AMD: the wet form, where treatment like anti-VEGF injections slows down the disease progression, and the dry form that is most frequent, where there is currently no curative treatment available. More than 5 million patients are afflicted with advanced dry AMD, also referred to as Geographic Atrophy. Patients suffering from this retinal dystrophy gradually lose their central vision (responsible for high visual acuity, e.g. for reading and face recognition) due to loss of photoreceptors.

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 and a rehabilitation period. Pixium Vision is in clinical stage with PRIMA, its sub-retinal miniature photovoltaic wireless implant system, designed for patients who have lost their sight due to outer retinal degeneration, initially for atrophic dry age-related macular degeneration (dry AMD). Pixium Vision collaborates closely with academic and research partners spanning across the prestigious Vision research institutions including Stanford University in California, Institut de la Vision in Paris, Moorfields Eye Hospital in London, Institute of Ocular Microsurgery (IMO) in Barcelona, and UPMC in Pittsburgh, PA. The company is EN ISO 13485 certified and qualifies as “Entreprise Innovante” by Bpifrance.
1 Wong, W. L., Su, X., Li, X., Cheung, C. M. G., Klein, R., Cheng, C. Y., & Wong, T. Y. (2014). Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. The Lancet Global Health, 2(2), e106-e116 (https://www.thelancet.com/journals/langlo/article/PIIS2214-109X(13)70145-1/fulltext)
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

02/01/2019

New York, US and Vienna, Austria, January 02, 2019 – HOOKIPA Pharma Inc. (“HOOKIPA”), a company developing a new class of immunotherapies targeting infectious diseases and cancers based on its proprietary arenavirus platform, today announced that it has achieved its first research milestone in its collaboration and license agreement with Gilead Sciences, Inc. (“Gilead”). The agreement, which was entered into in June 2018, grants Gilead exclusive rights to HOOKIPA’s TheraT® and VaxWave® investigational arenavirus-based immunization technologies for the development of immunotherapies against hepatitis B virus (HBV) and human immunodeficiency virus (HIV).

HOOKIPA has completed the first research milestone by designing and delivering 14 research-grade vectors to Gilead, along with the characterization of these vectors and delivery of a data package for the HIV program. Pursuant to the terms of the agreement, HOOKIPA is entitled to a sizeable milestone payment from Gilead.

Joern Aldag, HOOKIPA’s Chief Executive Officer said: “We are pleased to have achieved our first milestone within the collaborative HIV program with Gilead. This great achievement reflects our joint commitment to develop and deliver new treatment options for patients suffering from these infectious diseases. It also reflects HOOKIPA´s dedication to further validate our technology in infectious disease.”

Under the terms of the agreement, Gilead provided an upfront payment of $10 million and will fund all research and development activities. HOOKIPA will be eligible to receive milestone payments based upon the achievement of specified development, regulatory, and commercial milestones up to a total of approximately $400 million. HOOKIPA will also be eligible to receive tiered royalties on net sales.

About HOOKIPA
HOOKIPA Pharma Inc. is a clinical stage biopharmaceutical company developing a new class of immunotherapeutics, targeting infectious diseases and cancers based on its proprietary arenavirus platform that is designed to reprogram the body’s immune system.
HOOKIPA’s proprietary arenavirus-based technologies, VaxWave®*, a replication-deficient viral vector, and TheraT®*, a replication-attenuated viral vector, are designed to induce robust antigen specific CD8+ T cells and pathogen-neutralizing antibodies. Both, VaxWave® and TheraT®, are designed to allow for repeat administration while maintaining an immune response. TheraT® has the potential to induce CD8+ T cell response levels previously not achieved by other published immuno-therapy approaches. HOOKIPA’s “off-the-shelf” viral vectors target dendritic cells in vivo to activate the immune system.

HOOKIPA has successfully completed a Phase 1 trial of a VaxWave®-based prophylactic vaccine to protect against cytomegalovirus infection and has started dosing patients in a
Phase 2 trial in cytomegalovirus-negative patients awaiting kidney transplantation from cytomegalovirus-positive donors. To expand its infectious disease portfolio, HOOKIPA has entered into a collaboration and licensing agreement with Gilead Sciences, Inc. to jointly research and develop functional cures for HIV and Hepatitis B infections. HOOKIPA is building a proprietary immuno-oncology pipeline by targeting virally mediated cancer antigens, self-antigens and next-generation antigens.

TheraT® and VaxWave® are not approved anywhere globally and their safety and efficacy have not been established.

Find out more about HOOKIPA online at www.hookipapharma.com.

About Gilead Sciences, Inc.
Gilead Sciences, Inc. is a research-based biopharmaceutical company that discovers, develops and commercializes innovative medicines in areas of unmet medical need. The company strives to transform and simplify care for people with life-threatening illnesses around the world. Gilead has operations in more than 35 countries worldwide, with headquarters in Foster City, California.

*Registered in Europe; Pending in the US.

17/12/2018

LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec 17, 2018 – 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, today announced the publication of a peer-reviewed manuscript describing the previously announced interim results of a clinical trial of QR-110 for the treatment of Leber’s congenital amaurosis 10 (LCA10) in the journal Nature Medicine.
“It was enormously gratifying to see robust improvements in visual acuity and significant augmentations in the patient’s ability to detect lights, and impressive to observe these effects within the first three months following a single injection,” said Professor Artur V. Cideciyan, Ph.D., who was one of the co-investigators at the Scheie Eye Institute of the University of Pennsylvania. “LCA10 is a severe form of childhood blindness and this is a major step forward in the treatment of these previously incurable conditions,” said Professor Samuel G. Jacobson, M.D., Ph.D, who is the ophthalmologist caring for four of the patients enrolled in the study and is also a co-investigator at the Scheie Eye Institute. Published results detail a planned interim analysis of the ongoing PQ-110-001 first-in-human clinical study, evaluating QR-110 in patients with LCA10. The publication focused on a landmark analysis of eight subjects who reached three months of single dose treatment experience. Results demonstrate a substantive overall improvement in best corrected visual acuity (BCVA) with a mean improvement of -0.67 LogMAR (SEM 0.32) in the treated eye versus 0.02 LogMAR (SEM 0.05) in the contralateral (control) eye (p=0.011). The majority of patients showed an increase of at least -0.3 LogMAR in the treated eye, which is generally considered clinically meaningful, and equivalent to 15 letters, or three lines of improvement for individuals able to read a standard eye chart. Visual acuity improvements were also associated with concordant improvements in full-field stimulus thresholds to both blue and red light, improved mobility course navigation and ocular instability measurement. LCA10 is a severe inherited retinal dystrophy associated with mutations in the CEP290 gene. QR-110 is an RNA-based drug candidate that has the potential to restore sight or slow down the process of vision loss in patients with LCA10 by correcting the most common mutation causing LCA10, p.Cys998X. The ongoing Phase 1/2 PQ-110-001 study of QR-110 will be completed and in parallel a Phase 2/3 « ILLUMINATE » study is expected to be initiated in the first half of 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 LCA10 is the most frequent and one of the more severe forms. LCA10 is caused by mutations in the CEP290 gene, of which the p.Cys998X mutation is the most common. LCA10 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 LCA10 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 (also known as the c.2991+1655A>G 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. About the PQ-110-001 Phase 1/2 Trial
PQ-110-001 is a first-in-human open-label trial that enrolled 5 children (age 6 – 17 years) and 6 adults (≥ 18 years) who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. Patients are receiving four intravitreal injections of QR-110 into one eye; one injection every three months, with the other eye remaining untreated. Two dose levels are being tested, 80 μg (160 μg loading dose) and 160 μg (320 μg loading dose). The trial is being conducted at three specialized centers with significant expertise in genetic retinal disease: the University of Iowa, Iowa City, Iowa, U.S., the Scheie Eye Institute at the University of Pennsylvania, Philadelphia, U.S., and the Ghent University Hospital, Ghent, Belgium.
The primary objectives of the PQ-110-001 trial are safety and tolerability. Secondary objectives include pharmacokinetics, as well as restoration/improvement of visual function and retinal structure through ophthalmic endpoints, such as visual acuity (BCVA), mobility course, full field stimulus testing (FST), ocular instability (OCI), optical coherence tomography (OCT), and pupillary light reflex (PLR). Changes in quality of life in the trial subjects are also being evaluated.

About the Phase 2/3 pivotal “ILLUMINATE” Trial
The Company has agreed with the FDA to submit a protocol to start a Phase 2/3 trial that could serve as the sole registration trial, to be called « ILLUMINATE ». The preliminary design for “ILLUMINATE” is a double-masked, controlled, 12-month study. The trial is expected to initially enroll 30 patients with LCA10 due to one or two copies of the p.Cys998X mutation and could be adaptively repowered. The primary endpoints in this trial are expected to include visual acuity and the mobility course. The trial is expected to be conducted at centers in North America and select European countries. The trial is expected to start in the first half of 2019.
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 Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. 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.
ProQR Therapeutics N.V. | Zernikedreef 9, 2333 CK Leiden, The Netherlands | +31 88 166 7000 | info@proqr.com | www.proqr.com
These forward-looking statements include, but are not limited to, statements regarding QR-110 and its clinical development, including the completion of the ongoing Phase 1/2 clinical trial and commencement of the planned ILLUMINATE trial, trial design, regulatory pathway and therapeutic potential. 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, 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 Contact:
Lisa Hayes
Vice President of Investor Relations and Corporate Communications
T: +1 202 360 4855
ir@proqr.com

Media Contact:
Sara Zelkovic
LifeSci Public Relations
T: +1 646 876 4933
Sara@lifescipublicrelations.com