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17/10/2019

The firm manages €2B across its life sciences platform, €1B of which has been raised in the last 4 years

Paris, France – October 17th, 2019 — Sofinnova Partners, a leading European venture capital firm based in Paris, London and Milan and specialized in Life Sciences, announced today the close of its latest early-stage healthcare venture capital fund Sofinnova Capital IX, oversubscribed at €333 million. The firm now has more than €2B under management with more than €1B raised in the last four years across its platform of life sciences funds.

Pursuing the strategy it has consistently applied over the years for its flagship early-stage Capital funds, Sofinnova Capital IX will invest in the healthcare industry and more specifically in the biopharmaceutical and medical device sectors. Sofinnova Partners will seek to invest as a founding and lead investor in start-ups and corporate spin-offs, and focus on therapeutic, paradigm-shifting technologies and products alongside visionary entrepreneurs. Sofinnova Capital IX will invest about two thirds of its funds in European companies, and one third outside of Europe, primarily in North America.

The new fund has commitments from leading institutional investors, predominantly endowment funds, insurance companies, pension funds, sovereign funds, corporates and family offices, many of whom are continuing a long and successful relationship with the Sofinnova family of Funds. The majority of commitments came in from Europe, including France, Italy, Ireland, Denmark, Germany, Switzerland, the United Kingdom and Luxembourg, but also from leading North American investors in the U.S. and Canada, as well as major investors from Asia.
Antoine Papiernik, Managing Partner and Chairman of Sofinnova Partners, said, “Our experienced team, stable strategy and exit track record resonated well with investors, hence the success of our fundraising for this latest Capital fund. Over the last three years, we have completed nine remarkable exits in the portfolio for a total enterprise value of almost 4 billion euros.”

The launch of Sofinnova Capital IX follows the formation, in just the last three years, of Sofinnova Crossover I, a fund investing in pre- and post-IPO companies; Sofinnova MD Start III, a medical device acceleration fund; Sofinnova Industrial Biotech I, a fund dedicated to industrial biotech; and Sofinnova Telethon Fund I, a fund dedicated to seed investments in gene and cell therapies based out of Milan, Italy. With these focused franchises managed by dedicated specialist teams, Sofinnova Partners has established a unique and comprehensive platform of investment vehicles across the life sciences investment value chain.

Triago acted as placement agent and Clifford Chance acted as legal counsel on Sofinnova Capital IX.

About Sofinnova Partners
Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Headquartered in Paris, France, with offices in London and Milan, the firm brings together a team of 40 professionals from all over Europe, the U.S. and Asia. The firm focuses on paradigm-shifting technologies alongside visionary entrepreneurs. Sofinnova Partners invests across the Life Sciences value chain as a lead or cornerstone investor, from very early-stage opportunities to late-stage/public 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, please visit: www.sofinnovapartners.com

Media Contacts:
Kate Barrette
RooneyPartners LLC
+1.212.223.0561
kbarrette@rooneyco.com

France
Anne Rein
S&I
+33 6 03 35 92 05
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.

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

13/12/2018

Abiomed Invests $15 Million in Shockwave

Santa Clara, Calif. – December 11, 2018 – Shockwave Medical, a pioneer in the development and commercialization of intravascular lithotripsy to treat complex calcified cardiovascular disease, today announced an investment and collaboration agreement with Abiomed, Inc. As outlined by the agreement, Abiomed will invest $15 million in Shockwave and the two companies will collaborate on a training and education program in the United States and Germany focused on the benefits of complementary use of their respective technologies.
Shockwave’s intravascular lithotripsy (IVL) technology employs sonic pressure waves to safely crack vascular calcium within the vessel wall, which enables arteries to expand under low pressure and become more compliant. Shockwave markets its Shockwave M5 Peripheral Intravascular Lithotripsy Catheter in the United States and Europe. With increasing frequency, the Shockwave M5 catheter is being used in patients with heavily calcified Iliac arteries in order to facilitate the transfemoral delivery of sophisticated devices with catheters, including transcatheter heart valves (TAVR) and Abiomed’s Impella®. IVL enables this patient group to benefit from these life-saving therapies when they would otherwise be ineligible for the procedure or would be at increased risk for procedural complications. In Europe, Shockwave also markets its coronary catheter – Shockwave C2 – which is used to treat severely calcified de novo coronary artery disease.
“While we are still early in our commercial scaling both in the US and Europe, I am pleased with how positively our Shockwave technology has been received and how many different types of patients and vessels our customers are able to safely treat with our IVL system,” said Doug Godshall, President and CEO of Shockwave Medical. “We are delighted to be able offer patients our solution in combination with Abiomed’s Impella technology using a minimally invasive approach, which should meaningfully improve outcomes. With Abiomed’s best-in-class approach to training and education, Shockwave will be able to more efficiently increase awareness and introduce IVL to customers, which we believe will help them better treat their most challenging patients. We are encouraged to see the positive clinical response we have witnessed to date.”

About Shockwave Medical’s Intravascular Lithotripsy System
Shockwave Medical’s IVL System leverages similar principles to urologic lithotripsy, which has been used as a safe and effective treatment to break up kidney stones for several decades.

The generator produces energy that travels through the connector cable and catheter to an array of miniaturized lithotripsy emitters located near the calcified lesion. With the integrated balloon expanded to ultra-low pressure, a small electrical discharge at the emitters vaporizes the fluid within the balloon, creating a rapidly expanding bubble that collapses within microseconds. The bubble’s expansion and collapse generates a series of sonic pressure waves that travel through the fluid-filled balloon and pass through soft vascular tissue, selectively cracking any hardened calcified plaque inside the vessel wall. After the calcium has been fractured, the integrated balloon can be expanded, performing angioplasty safely at low pressures. To view an animation of the Intravascular Lithotripsy procedure visit
http://shockwavemedical.com.

About Shockwave Medical
Shockwave Medical, based in Santa Clara, Calif., is developing and commercializing innovative intravascular lithotripsy technology for the treatment of calcified peripheral vascular, coronary vascular and heart valve disease. For more information, visit www.shockwavemedical.com.

13/12/2018

Paris, France – December 13, 2018  – 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 today that it has been awarded the Prix Galien 2018 in the “Research Project” category for its PRIMA device for the treatment of dry Age-related Macular Degeneration (AMD).
The Galien Foundation attributes this award to Pixium Vision in recognition of the innovative technology of its PRIMA wireless sub-retinal implant, a “brain-machine” interface applied to the eye to create bionic vision in the treatment of persons with dry AMD. The PRIMA device was chosen from a selection of premier projects supported by prestigious academic institutions.
Khalid Ishaque, CEO of Pixium Vision, comments: “It is with pleasure that our entire team of scientific, engineering and medical researchers receives this prestigious award. The Galien Award attests to the very innovative nature of our PRIMA technology owing to elicit visual perception for patients with dry AMD. We are extremely honoured that the Galien Foundation recognizes PRIMA’s major technological and medical advances, which offer new possibilities in the field of bionic vision for the treatment of retinal diseases. We would like to thank the first patients in our clinical trial for their contribution in this breakthrough human endeavour, which will enable us to continue pursuit of our mission.”
“We are proud to accompany Pixium Vision in developing its breakthrough technology, today awarded the Galien Prize. We congratulate the whole team for this highly valuable scientific recognition.” stated Chahra Louafi et Maïlys Ferrère, Investment Directors at Bpifrance.
Created as a “pharmaceutical research award” in France in 1970, the prestigious Prix Galien is recognised by the French authorities for its public interest and has an international reputation. Each year, the Prix Galien recognises the best healthcare innovations in five categories: pharmaceutical products, medical systems, e-health, patient care and research. The Prix Galien is awarded by a jury comprised of 50 international experts in healthcare. Their independence, authority and reputation confirm the excellence and credibility of the Galien Prize.

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.
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

13/12/2018

• Funding will be used to further expand product capabilities and fuel company growth
• Industry-leader is bringing software abstraction and automation to biological R&D and manufacturing

12 December – LONDON – Synthace Ltd., the company behind the leading cloud software platform for automating and improving the success rate of biological research and development, today announced the closing of a $25.6m Series B financing round led by Horizons Ventures with additional investment from Luminous Ventures, SOSV and select other individual and large family office investors. Synthace will use the new funds to drive continued product development and build upon its cell and gene therapy customer base to accelerate global awareness of its solutions.
Synthace is a leader in Computer Aided Biology, a new paradigm that comprises two domains: the Digital and the Physical. The Digital, powered by artificial intelligence, includes software for designing and simulating biological systems, as well as methods of collating, structuring and analysing experimental data. The Physical, enabled by automation, includes systems that allow for the seamless transfer of biological designs into real ‘wet lab’ experiments via logistics simulation and execution.
In October 2018, Synthace launched a white paper, Computer Aided Biology: Delivering Biotechnology in the 21st Century which provides the most complete industry vision to date for how biological research will be transformed by an emerging ecosystem of cloud connected and digitally empowered research tools that augment human capabilities, accelerating the transition of biology towards becoming an engineering discipline.
Synthace CEO Tim Fell said: “Digital-to-physical workflows have transformed the semiconductor, aerospace, automobile and many other industries. Now it is the turn of the biotechnology industry, and we are grateful for the support of Horizons Ventures and our other new investment partners who share Synthace’s vision of how to facilitate that change.”
Patrick Zhang, of Horizons Ventures, commented: “We are at a pivotal point in the development and use of biotechnology. We believe that Synthace will lead the industry’s transition to Computer Aided Biology, owing to its truly disruptive and cutting-edge AI driven experiment design capability and experiment execution technology. We look forward to working with the team on this next exciting stage of Synthace’s development.”
Bob Wiederhold, Synthace Chairman, concluded: “The realization is setting in across industry and academia that the complexity of biology can only be properly addressed with advanced software and automation. This investment in Synthace is a significant step in turning that recognition into reality.”
This announcement follows previous distinctions from Gartner and the World Economic Forum, where the impact of the Synthace platform in enabling the Lab of the Future and Industry 4.0 was recognised.

About Synthace
Based in London, Synthace is developing Antha, a language and software platform specifically for biology that lets researchers aim higher and achieve better results, faster. Antha is designed to make reproducible and scalable workflows that can be readily edited and shared, and easily automated on labs’ existing equipment. With customers across pharma, agritech and industrial biotechnology Synthace has been recognised by the World Economic Forum as a Technology Pioneer that is helping shape the Fourth Industrial Revolution – a technological revolution that will fundamentally alter the way we live, work and relate to one another. For more information, visit: www.synthace.com.

21/11/2018

New York, US and Vienna, Austria, November 14, 2018 – HOOKIPA Pharma Inc. (“HOOKIPA”), a company developing an innovative class of immunotherapies for oncology and infectious diseases using its proprietary arenavirus technologies, today announced the appointment of Julie O’Neill to its Board of Directors as a Non-Executive Director.

A business professional with more than two decades of executive experience in senior leadership roles, Ms. O’Neill was formerly Executive Vice President, Global Operations at Alexion Pharmaceuticals, Inc. where, for five years, she led the Global Operations business including product development, manufacturing, quality, supply chain and global real estate functions. Prior to joining Alexion Pharmaceuticals, Inc., she was Vice President of Operations at Gilead Sciences where she helped establish the Company’s Irish subsidiary and manufacturing operations and held a variety of other cross-functional global leadership roles.

Throughout her career in pharmaceutical operations, Ms. O’Neill has held a number of senior roles at the Board and Committee level. She is currently an Independent Director for DBV Technologies SA, and is both a Board member and Audit & Risk Committee Chair for the National Institute for Bioprocessing Research & Training (NIBRT). She also serves as the Chairperson for the Strategic Advisory Board at the School of Pharmacy, Trinity College Dublin.

Ms. O’Neill is a Chartered Director and holds a BSc in Pharmacy from Trinity College Dublin and an MBA from University College Dublin.

Commenting on her appointment, Julie O’Neill said: “Immuno-oncology is very much at the forefront of modern medicine and HOOKIPA’s Vaxwave® and TheraT® platforms have the potential to target a broad range of infectious diseases and tumors. At this point in its development HOOKIPA is shifting its emphasis from research to clinical development and therefore operations, manufacturing, and quality control are growing in importance. I am looking forward to joining the HOOKIPA team and supporting them in their ambition to take increasing control of the manufacturing process. I am excited at the chance to be able to contribute to the Company’s growth and operations at such an important stage in its development.”

Dr. Jan van de Winkel, Chairman of the Board of Directors at HOOKIPA, added: “In the next several years HOOKIPA plans to build its own GMP manufacturing capabilities, one of the most critical functions for viral vector medicine research and development. I am delighted to welcome Julie to HOOKIPA and believe that her operations and manufacturing management expertise complement nicely the capabilities of our current Board. My colleagues and I look forward to expanding the Board and we will enjoy working with Julie.”

 

About HOOKIPA
HOOKIPA Pharma Inc. is a clinical stage company developing medicines designed to prevent and potentially cure infectious diseases and cancer. With this goal in mind, we created proprietary technologies to reprogram and stimulate the immune system.

Our proprietary Arenavirus technologies, Vaxwave®, a replication-deficient viral vector, and TheraT®, a replication-attenuated vector, are designed to induce potent antigen specific CD8+ T cells and pathogen-neutralizing antibodies. Both, Vaxwave® and TheraT®, are designed to be administered repeatedly while maintaining an immune response. TheraT® has the potential to induce CD8+ T cell responses to tumor antigens and reach frequencies and potencies matching or exceeding those observed after adoptive T cell therapy. Our “off-the-shelf” viral vectors target dendritic cells in vivo to activate the immune system. In immuno-oncology, this mechanism enables the immune system to fight solid tumors systemically.

We have successfully completed a Phase 1 trial of a Vaxwave®-based prophylactic vaccine to protect against CMV infections. A Phase II trial in solid organ transplant patients is about to start. Alongside, we have forged a partnership with Gilead Sciences Inc. to jointly research and develop functional cures for HIV and Hepatitis B infections. We are building a proprietary immuno-oncology pipeline by targeting TheraT® towards HPV+ head and neck squamous cell carcinoma and prostate cancer.

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

Issued for and on behalf of HOOKIPA Pharma Inc. by Instinctif Partners. For further information please contact:

HOOKIPA
Marine Popoff
Communications & Investor Relations Manager
Marine.Popoff@HookipaPharma.com

Media enquiries
Sue Charles/ Ashley Tapp
Instinctif Partners
Hookipa@Instinctif.com
+44 (0)20 7457 2020

13/11/2018

Bresso (MI), Italy – November 12, 2018 – EryDel SpA (www.erydel.com), a biotech company specializing in the development and commercialization of drugs and diagnostics delivered through autologous red blood cells, today announced the appointment of Stanley J. Musial as Chief Financial Officer, effective immediately.
“The addition of Stan to the management team comes at a critical time as EryDel further strengthens its management team,” remarked Dr. Luca Benatti, Chief Executive Officer of EryDel. “Stan’s financial leadership and industry expertise, as well as his significant fund-raising and transactional experience, are invaluable assets, as EryDel moves its pipeline through development and ultimately to the commercial market.”

Stan has held senior financial management positions for both publicly-held and privately-held companies, becoming experienced in executing growth strategies, raising capital, and mergers and acquisitions. Stan most recently spent nearly six years as Executive Vice President and Chief Financial Officer of Egalet Corporation, a fully integrated specialty pharmaceutical company focused on innovative treatments for pain, which Stan took public in 2014, and was part of a team that transformed the company from development stage to revenue generation. Prior to that,
Stan held the position of Chief Financial Officer at Prism Pharmaceuticals, Inc., Strategic Diagnostics, Inc., and similar positions with other biotechnology, medical device and healthcare services companies. Stan began his career with KPMG LLP, earned an MBA in finance from Temple University and a BS degree in accounting from the Pennsylvania State University, and is a Certified Public Accountant.

Mr. Musial commented, “EryDel has a robust platform and a diverse pipeline that should lead to multiple opportunities to address important unmet medical needs. I am excited to join the team and contribute to advancing these product candidates by pursuing financial and operational strategies that enable the company to navigate challenges successfully and achieve meaningful clinical, regulatory, and commercial milestones.”

About Ataxia Telangiectasia
Ataxia Telangiectasia (AT) is a rare genetic disease caused by biallelic mutations in the ataxia telangiectasia mutated (ATM) gene, for which no established therapy is currently available. ATM encodes a PI3Kinase protein shown to play a pivotal role in response to DNA damage and cell cycle control. Homozygosity for ATM mutations result in a multisystemic disorder, involving mainly the nervous and immune systems. The major clinical feature of AT is severe progressive neurodegeneration from early infancy. Specific features include progressive ataxia of the trunk and limbs, involuntary movements, oculomotor apraxia, difficulties with speech and swallowing, and delayed peripheral neuropathy. Other clinical features of patients with the classical phenotype include oculocutaneous telangiectasia, immunodeficiency with recurrent respiratory tract infections, radiosensitivity and an increased incidence of cancer.

About EryDel
EryDel SpA is a biotechnology company specialized in the development of drugs delivered through red blood cells (RBCs) by using a proprietary medical device technology. Its most advanced product, EryDex System (EDS) is under late stage development for the treatment of Ataxia Telangiectasia, a rare autosomal recessive disorder for which no established therapy is currently available. EryDex has received Orphan Drug designation for the treatment of AT both from the FDA and the EMA. A completed pilot Phase II trial in AT patients demonstrated statistically significant efficacy of EDS on both the primary and secondary efficacy measures. An international multi-center, Phase III pivotal study, ATTeST, is being conducted. EryDel has a pipeline of preclinical programs that use its proprietary RBC’s delivery technology for the treatment of other rare diseases.
The ATTeST project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 667946”.
Media contact: Emanuela Germi at +39 02 36504470 or emanuela.germi@erydel.com

16/10/2018

Bresso (MI), Italy – October 15, 2018 – EryDel SpA (www.erydel.com), a biotech company specializing in the development
and commercialization of drugs and diagnostics delivered through autologous red blood cells, today announced the
appointment of Ronan W. Gannon as Chief Commercial Officer, effective immediately.
“I am pleased to welcome Ronan to EryDel,” said Dr. Luca Benatti, Chief Executive Officer of EryDel. Ronan’s commercial
expertise in rare diseases and blood products will be critical as we advance our comprehensive development program
for EryDex, including our pivotal Phase 3 clinical study (ATTeST) in Ataxia Telangiectasia and seek to transition EryDel
from a late-stage research and development company to a commercial-stage organization. We look forward to his
contribution as a member of our leadership team.”
Ronan brings more than 25 years of industry experience across large, midsize and startup life science companies. Before
joining EryDel, Ronan led global commercial operations at Radius Health, where he helped transform the company from
a late-stage development startup into a fully integrated biopharmaceutical company. Prior to that, Ronan spent ten
years at CSL Behring where he held several leadership roles including US Vice President Marketing and North America
general management roles in the US and Canada. During his tenure at CSL Behring he launched six orphan drugs. Prior
to CSL, Ronan led sales and marketing teams at Zeneca Pharmaceuticals, and GlaxoSmithKline across several therapeutic
areas including biologicals, oncology, and asthma. He received an MBA from Northeastern University, a BA from the
University of Richmond and holds alumni status from the Harvard Business School.
“I am delighted to join EryDel as the company begins planning for the commercialization of EryDex,“ said Mr. Gannon.
“EryDel has played a significant role in developing an innovative treatment for Ataxia Telangiectasia, a life-threatening
disease. I look forward to working with the team to continue ongoing community and market development efforts, and
to advance a robust commercialization strategy for the launch of EryDex.”

About Ataxia Telangiectasia
Ataxia Telangiectasia (AT) is a rare genetic disease caused by biallelic mutations in the ataxia telangiectasia mutated
(ATM) gene, for which no established therapy is currently available. ATM encodes a PI3Kinase protein shown to play a
pivotal role in response to DNA damage and cell cycle control. Homozygosity for ATM mutations result in a multi-systemic
disorder, involving mainly the nervous and immune systems. The major clinical feature of AT is severe progressive
neurodegeneration from early infancy. Specific features include progressive ataxia of the trunk and limbs, involuntary
movements, oculomotor apraxia, difficulties with speech and swallowing, and delayed peripheral neuropathy. Other
clinical features of patients with the classical phenotype include oculocutaneous telangiectasia, immunodeficiency with
recurrent respiratory tract infections, radiosensitivity and an increased incidence of cancer.

About EryDel
EryDel SpA is a biotechnology company specialized in the development of drugs delivered through red blood cells (RBCs)
by using a proprietary medical device technology. Its most advanced product, EryDex System (EDS) is under late stage
development for the treatment of Ataxia Telangiectasia, a rare autosomal recessive disorder for which no established
therapy is currently available. EryDex has received Orphan Drug designation for the treatment of AT both from the FDA
and the EMA. A completed pilot Phase II trial in AT patients demonstrated statistically significant efficacy of EDS on both
the primary and secondary efficacy measures. An international multi-center, Phase III pivotal study, ATTeST, is being
conducted. EryDel has a pipeline of preclinical programs that use its proprietary RBC’s delivery technology for the
treatment of other rare diseases.
The ATTeST project has received funding from the European Union’s Horizon 2020 research and innovation programme
under grant agreement No 667946”.
Media contact: Emanuela Germi at +39 02 36504470 or emanuela.germi@erydel.com