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27/09/2017

Paris, France – September 27th, 2017 — Sofinnova Partners, a leading European venture capital firm specialized in Life Sciences, has appointed Henrijette Richter as Managing Partner. She joins Antoine Papiernik, Denis Lucquin, Graziano Seghezzi and Monique Saulnier in the company’s Managing Partnership. With a 14 years’ experience in venture capital, Henrijette brings her extensive industry experience and strong international network to Sofinnova Partners’ leadership.

Henrijette joined Sofinnova Partners in October 2014, after seven years at Novo Holdings A/S (the holding company in the Novo Group) where she co-founded Novo Seeds. Prior to that, she worked at Sunstone Capital and was part of the founding team when the fund spun out of The Danish Growth Foundation. Henrijette is a scientist by training, she holds a combined PhD and Industrial Scientist degree in Molecular Biology from the University of Copenhagen, and did her postdoctoral fellowship at MIT Center for Cancer Research, Cambridge, MA.

Since 2014, Henrijette has lead key investments such as in Asceneuron, a biotech company specialized in neurodegenerative diseases headquartered in Switzerland where she also serves on the Board of Directors. Henrijette also seeded and invested in Delinia, a US-based company specialized in the treatment of auto-immune disorders that was sold to Celgene Corporation in January 2017 for a total value of up to $775 M.

Henrijette Richter says: « I am honored by this new role and excited to take a more active role in Sofinnova Partners’ growth strategy to reinforce its global leadership in Life Sciences. I have the foremost respect for the team, its values, the culture of diversity, and the entrepreneur-oriented spirit it has been fostering worldwide for more than forty years ».

Antoine Papiernik, Chairman of Sofinnova Partners, adds: « Henrijette stands out for her investment track record and has demonstrated impressive team leading skills. She is a great addition to our team, and we are extremely pleased to welcome her to the Managing Partnership as Sofinnova Partners is entering a new phase of its development ».
Press contact for SOFINNOVA PARTNERS
Anne REIN
Tel: +33 6 03 35 92 05
@: anne.rein@strategiesimage.com

13/02/2018
• Foundation Fighting Blindness and ProQR enter into a partnership to develop QR-421a for Usher syndrome type 2A, targeting mutations in exon 13 of the causative USH2A gene.
• Foundation Fighting Blindness will provide milestone-based co-funding of up to $7.5 million to ProQR to advance the program into the clinic.
• QR-421a received orphan drug designation from the FDA.
• There are currently no therapies commercially available or in clinical development for the vision loss associated with Usher syndrome type 2A.
• QR-421a is part of ProQR’s growing ophthalmology pipeline which also includes lead candidate, QR-110 for Leber’s congenital amaurosis 10 currently in clinical trials, and three additional pipeline programs, QR-411 that addresses another genetic mutation resulting in Usher syndrome type 2A, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.
• QR-421a is expected to advance towards the clinic in 2018, with clinical data expected in 2019.
COLUMBIA, Md. and LEIDEN, the Netherlands, Feb. 12, 2018 — Foundation Fighting Blindness and ProQR Therapeutics N.V. (NASDAQ:PRQR), today announced that they have entered into a partnership to develop QR-421a for Usher syndrome 2A caused by an exon 13 mutation of the causative USH2A gene.  Under the agreement, Foundation Fighting Blindness will provide up to $7.5 million in funding to ProQR for the preclinical and clinical development of QR-421a, which is expected to advance towards the clinic in 2018, and safety and efficacy results from the Phase 1/2 trial in Usher syndrome patients are expected in 2019.
Usher syndrome is a devastating genetic disease in which patients first develop hearing loss and then progressive vision loss, thereby threatening their independence and quality of life. Currently there is no treatment for the ophthalmic manifestation of Usher syndrome type 2A.  QR-421a is a first-in-class RNA oligonucleotide that is being developed for the treatment of vision loss associated with the disease. QR-421a is designed to modify the RNA such that functional usherin protein is produced in the retina with the goal of stopping the progression of the disease and potentially gaining peripheral vision. ProQR in-licensed the technology underlying QR-421a from Radboud University Medical Center in the Netherlands, where it was invented by lead investigator Dr. Erwin van Wyck.
Foundation Fighting Blindness’ Clinical Research Institute (FFB-CRI) has also launched a natural history study in 120 people with USH2A mutations. The study — known as RUSH2A (“R” stands for “rate of progression”) — was launched in 2017 and is being conducted at about 20 clinical sites around the world. RUSH2A investigators will use a variety of technologies to monitor changes in vision and retinal structure to document and analyze disease progression. Knowledge and data obtained from this trial are intended to provide a better understanding of how USH2A mutations affect the severity and progression of vision loss and help to inform the development of QR-421a.
“Teaming with corporate partners to help promising therapies move through preclinical and clinical development is central to FFB’s strategy so we are very pleased to enter into this partnership with ProQR,” said Benjamin R. Yerxa, PhD, CEO at Foundation Fighting Blindness.  “The fact that there are currently no available treatments for Usher syndrome type 2A makes this work that much more exciting and critical.”
QR-421a for Usher syndrome is the second program in ProQR’s growing ophthalmology pipeline scheduled to enter clinical trials.  The lead program in the ophthalmology pipeline, QR-110, is currently in a Phase 1/2 safety and efficacy trial in adult and pediatric patients with Leber’s congenital amaurosis 10, due to the p.Cys998X mutation in the CEP290 gene.  This pipeline also contains several other molecules for genetic eye diseases, including QR-411 for Usher syndrome type 2A due to the PE-40 mutation, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.
“We are excited to team up with the Foundation Fighting Blindness to develop QR-421a for patients that suffer from Usher syndrome due to exon 13 mutations,” said Daniel A. de Boer, CEO of ProQR. “They are the leading private funder of retinal disease research with a very patient centric approach which is a core pillar of our strategy. Through this partnership with the Foundation we plan to gain access to important know-how to develop programs in retinal diseases. We expect that the additional funding will allow us to rapidly advance this novel therapy for this orphan disease with a severe unmet need.”
About Foundation Fighting Blindness
The Foundation Fighting Blindness was established in 1971 and has raised more than $725 million for research on preventing, treating and curing blindness caused by inherited retinal diseases. In excess of 10 million Americans, and millions more worldwide, experience or are at risk for vision loss due to retinal degenerations. Through its support of focused and innovative science, and by teaming with industry, the Foundation drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*
About QR-421a
QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome 2A due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional usherin protein that causes the disease. QR-421a is designed to repair the genetic defect in the RNA in the eye, such that it leads to the expression of a shortened but functional protein, thereby modifying the underlying disease. QR-421a has received orphan drug designation from the U.S. Food and Drug Administration.
About Usher Syndrome Type 2A
Usher syndrome is the leading cause of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. Usher syndrome type 2A is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene. To date, there are no treatments approved or products in clinical development that treat the vision loss associated with Usher syndrome type 2A.
PROQR FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding our collaboration with Foundation Fighting Blindness, including statements regarding the expected funding and benefits of such collaboration, our product candidates QR-110, QRX-1011, QRX-504 and QR-421a, including their clinical development and therapeutic potential, including future development plans.  Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, that a Fast Track designation by the FDA may not actually lead to a faster development, regulatory review or approval process, and that we may not be able to realize the potential benefits of orphan drug exclusivity, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.
ProQR Therapeutics N.V.:
Investor and Media Contact:
Bonnie Ortega
T: +1 858 245 3983
ir@proqr.com
Foundation Fighting Blindness:
Media Contact:
Rhea K. Farberman
T: +1 410 423-0635
RFarberman@FightBlindness.org
Sources: ProQR Therapeutics and Foundation Fighting Blindness
25/01/2018
Paris, 24 January 2018 – Gecko Biomedical (“Gecko”) has been granted a total of €6 million in funding under the Investments for the Future program (PIA) for its project proposal entitled “PIAVE” [Projets Industriels d’Avenir (Promising Industrial Projects)], which is piloted by the General Secretariate for Investment (SGPI) and operated by Bpifrance. This funding is a contribution towards the industrialization and development of innovative solutions in the area of tissue reconstruction, and the peripheral nerve in particular.
Gecko is a medical device company developing an exclusive platform of polymers allowing for tissue reconstruction. These synthetic polymers, which are biocompatible, bioresorbable, and activated by a specific type of light, can be applied onto internal tissues during surgical procedures (acting like an adhesive, a barrier or a filler product, depending on the type of surgery). They are also 3D-printable, allowing for the production of flexible, bioresorbable and very high-resolution implants.
This funding will provide support for the industrialization of Gecko’s polymers platform, as well as for the development of a new, innovative program that will enable the reconstruction of peripheral nerves, without sutures, using 3D-printed micro-conducts secured by one of the company’s exclusive adhesives.
A 1,150 m2 manufacturing site, powered by 100% green electricity, is currently under construction in Roncq (in the Hauts-de-France Region) and will feature four clean rooms (totalling 300 m2 ) and an analytical laboratory extending over 140 m2.
Christophe Bancel, CEO of Gecko Biomedical, said: “This funding will allow us to speed up our industrialization process and equip ourselves with a globally unique manufacturing site. Cutting-edge processes will be performed on this site to produce all our liquid polymers in individual, sterile conditions, ready for use by surgeons. We also intend to speed up a second phase in our strategy through the industrial and clinical development of our 3D printing platform for flexible, bioresorbable and high-resolution implants, by leveraging our polymers as a biomedical resin, as well as by optimising state-of-the-art 3D printing techniques”.
Marie Zwarg, the Area Leader for Healthcare within the management team for Bpifrance’s Industrial Sectors, added that: “Bpifrance has supported Gecko Biomedical since its creation. We are delighted to contribute to furthering the development of its top-level technological platform with multiple applications, while also contributing to its industrialization in France”.
About Gecko Biomedical
Gecko Biomedical is a privately owned medical device company based in Paris, France that is dedicated to the rapid development and commercialization of a unique biopolymer platform to address various unmet clinical needs.
The company’s platform is based on a proprietary polymer family with unique properties including superior biocompatibility, tunable bioresorbability, and adjustable tissue adherence. Furthermore, the polymer hydrophobicity, high viscosity and controlled “on demand” curing enables a unique and controlled delivery to targeted tissues or the creation of scaffolds.
Gecko Biomedical’s first product, SETALUMTM Sealant, is an innovative polymer dedicated for tissue reconstruction. It is targeted to vascular reconstruction as an initial indication. Its structure is tunable, allowing customization for various applications and tissue types. The polymer is part of a biopolymer platform family that is fully industrialized and highly versatile, with potential novel applications in other fields of tissue reconstruction such as guided tissue repair, and the field of localized drug delivery.
The Company’s technology is based on world-class research and intellectual property from the laboratories of Professor Robert Langer (MIT) and Professor Jeffrey M. Karp (Brigham and Women’s Hospital), who co-founded the company in 2013, alongside Christophe Bancel and Bernard Gilly from the iBionext Network. For more information, please visit: www.geckobiomedical.com. Twitter: @geckobiomedical
About the Investments for the Future program
The Investments for the Future program (PIA) benefits from 57 million euros and is piloted by the General Secretariate for Investment. It was established by the government to fund innovative and promising investments in France. Six national priorities have thus been identified to allow France to increase its potential for growth and employment:
• higher education, research and training,
• research development and transfer to the economic sphere,
• sustainable development,
• industry and SMEs,
• the digital economy,
• health and biotechnology.
Find out more on investments for the future: http://www.gouvernement.fr/secretariat-general-pour-linvestissement-sgpi
Twitter: @SGPI_avenir
Bpifrance | PRESS RELEASE | 24 JANUARY 2018 | 3
About PIAVE
PIAVE’s call for projects is directed at projects that fit within the new 9-solution approach of the New Face of Industry in France (Nouvelle France industrielle).
It covers two types of projects:
• R&D and industrialization work,
• work aimed at reinforcing competitiveness within strategic French sectors.
These projects must benefit several small and medium enterprises (SMEs), or independent mid-cap companies (MCCs), and must show a concrete and significant benefit to the sector and its structuring. They must also entail industrial activity, and must prove, on fruition, to be autonomous with regard to public support.
In order to be eligible, these projects must involve a program entailing a minimum spend of 3 million euros, as well as a remit entailing prospects for business and for employment:
• fulfilling one or more solutions for the New Face of Industry in France, particularly with regard to the implementation of a road map approved by the 9 solutions piloting committee. In this event, the project must be handled by a company, irrespective of its size. This company may involve other companies, laboratories or research establishments within a consortium;
• fulfilling the objectives of one of the strategic sector committees; the project may alternatively be supported by a structure that brings together a number of companies, or by an entity which represents companies in the sector (such as a professional federation, a special interest group or an association…).
A budget of 305 million euros will be allocated to the “Promising Industrial Projects” (“PIAVE”) initiative, by way of state assistance (involving subsidies and refundable advance payments).
About Bpifrance
Bpifrance funds companies, at every stage of their growth, through loans, guarantees and equity. Bpifrance supports them in their innovation projects, as well as supporting them internationally. Bpifrance also now ensures their export activity through a broad range of products. Also on offer to entrepreneurs are advisory services, universities, networking and acceleration programs designed for start-ups, SMEs and mid-cap companies.
Thanks to Bpifrance and its 48 regional sites, entrepreneurs benefit from a close point of contact, which is dedicated to them and effectively supports them in facing their challenges.
More information here: www.Bpifrance.fr – http://investissementsdavenir.bpifrance.fr/ – Follow us on Twitter:
@Bpifrance – @BpifrancePresse
Press Office contacts:
Gecko
Europe
Brice Epry
Telephone: +33 1 76 21 72 28
info@geckobiomedical.com
United States
Marion Janic
Telephone: +1-212-223-4017
mjanic@rooneyco.com
Bpifrance
Nicolas Jehly
Telephone: +33 1 41 79 95 12
nicolas.jehly@bpifrance.fr
General Secretariate for Investment
Vincent Deshayes
Telephone: +33 1 42 75 64 29
vincent.deshayes@pm.gouv.fr
05/01/2018
The feasibility clinical study is designed to evaluate PRIMA sub-retinal implant in patients with Atrophic Dry Age-related Macular Degeneration
Paris, France. January 4, 2018 – 7.00 AM CET – Pixium Vision (FR0011950641 – PIX), a company developing innovative bionic vision systems to enable patients who have lost their sight to lead more independent lives, announced today that it has received the approval from the US Food and Drug Administration (FDA) to begin the clinical feasibility study for PRIMA, Pixium Vision’s new-generation miniaturized wireless photovoltaic sub-retinal implant, in patients with Atrophic Dry Age-related Macular Degeneration (AMD).
“This first approval in the US will allow Pixium Vision to commence a feasibility study of the PRIMA device and follows a thorough review by the FDA. It also highlights the FDA’s recognition of PRIMA’s innovative potential to address the significant unmet need to treat Atrophic Dry-AMD,” said Khalid Ishaque, Chief Executive Officer of Pixium Vision “Atrophic Dry-AMD is a major cause of irreversible loss1 of the vision which affects approximately 4 million people and for whom there is currently no proven therapeutic solution. Alongside the ongoing clinical trial in France, the feasibility study in the United States marks a significant milestone for Pixium Vision with the mission to create a world of bionic vision for those who have lost their sight.”
The clinical feasibility study of PRIMA implant, to be conducted at the University of Pittsburgh Medical Center, will recruit up to five patients with vision loss that results from Atrophic Dry Age-related Macular Degeneration. The primary endpoint is restoration of visual perception as well as safety at a 12-month follow-up with a longer-term follow-up duration of 36 months. Pixium Vision expects to start the US study in the first half of 2018.
Contacts
Pixium Vision
Didier Laurens, CFO
investors@pixium-vision.com
+33 1 76 21 47 68
@PixiumVision
Media Relations
France: Newcap Media
Annie-Florence Loyer – afloyer@newcap.fr
+33 1 44 71 00 12 / +33 6 88 20 35 59
Léa Jacquin – ljacquin@newcap.fr
+33 1 44 71 94 94
Media Relations International: Image Box PR
Neil Hunter
neil@imageboxpr.co.uk
Tel +44 (0)20 8943 4685
ABOUT PRIMA
PRIMA is a miniaturized new generation implant totally wireless. The PRIMA implant is a micro photovoltaic chip of 2×2 millimeters and 30 microns thick, and is equipped with 378 electrodes. Implanted under the retina via a less invasive surgical procedure, the implant converts pulsed near infra-red light signal received from the external glasses with an integrated mini-camera into electrical signals transmitted to the brain via the optic nerve. PRIMA is designed to treat retinal dystrophies, particularly aiming to treat initially advanced atrophic dry-AMD, the most prevalent form of Age-related Macular Degeneration, thanks to miniaturization and aimed to preserve patient’s residual peripheral vision. Prima is also intended to be evaluated at a later stage for treatment of vision loss from Retinitis Pigmentosa.
ABOUT PIXIUM VISION
Pixium Vision’s mission is to create a world of bionic vision for those who have lost their sight, enabling them to regain partial visual perception and greater autonomy. Pixium Vision’s bionic vision systems are associated with a surgical intervention as well as a rehabilitation period. Following the CE mark for its first bionic retinal implant systems, IRIS®II, Pixium Vision is now conducting a clinical study in Human with PRIMA, its next generation sub-retinal miniaturized photovoltaic wireless implant system, 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 the Institut de la Vision in Paris, the Hansen Experimental Physics Laboratory at Stanford University, Moorfields Eye Hospital in London, and Institute of Ocular Microsurgery (IMO) in Barcelona. Pixium Vision is EN ISO 13485 certified and labeled “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
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
19/12/2017
Ecolab Inc., the global leader in water, hygiene and energy technologies and services has acquired a minority stake in Metgen OY. As part of the acquisition, Ecolab will be granted exclusive distribution rights globally for Metgen OY’s pulp and paper portfolio, as well as its wastewater enzyme portfolio. This business supplies differentiated custom blended enzymatic solutions to maximize biomass performance for various industries.
MetGen, headquartered in Kaarina, Finland, designs novel enzymatic solutions to improve energy efficiency and speed of processes in industries such as Pulp and Paper, Biofuels and Biochemicals.
“We are glad to welcome Ecolab as an investor in MetGen. Our business goals are fully aligned with Ecolab’s objective to support customers to achieve the best solutions in water technologies for the pulp and paper sector. MetGen is excited to work together with the Ecolab team to accelerate market growth through the global introduction of innovative, sustainable enzymatic solutions in various business segments,” said Alex Michine, CEO of MetGen.
The investment provides Ecolab with access to innovative custom enzymes to help producers improve machine efficiency, water and energy savings, product quality and profitability. The terms of the transaction were not disclosed.
“We are excited to work with MetGen and implement their innovative products into our offerings to enhance Ecolab’s ability to provide value-added solutions for our customers in the Pulp and Paper Industry,” said Jerome Charton, senior vice president and general manager, Nalco Water global paper, Ecolab’s water management business.
For more information, please contact:
Alex Michine, CEO, MetGen Oy, +358 40 543 3740
skype: alexmichine
www.metgen.com
MetGen Rakentajantie 26, 20780, Kaarina
WWW.METGEN.COM Phone: 02 237 7077
info@metgen.com
MetGen Oy
Rakentajantie 26,
20780 Kaarina
info@metgen.com
About MetGen Oy
MetGen designs and markets novel enzymatic solutions for the most challenging of industrial conditions to address our customer’s specific challenges. MetGen’s enzymes – MetZyme® – are industrial, highly-active, natural catalysts that accelerate chemical reactions and company uses advances in genetic engineering and microbiology to adapt enzymes to harsh industrial conditions and to handle a variety of lignocellulosic substrates. MetGen aims to be a widely recognized supplier of industrial enzymes, significantly contributing to the economics and sustainability of process industries such as pulp & paper, biofuels and biochemicals. MetGen’s competitive advantage is in tailoring or adapting enzymes to meet customer’s specific needs. www.metgen.com
About Ecolab
A trusted partner at more than one million customer locations, Ecolab (ECL) is the global leader in water, hygiene and energy technologies and services that protect people and vital resources. With 2016 sales of $13 billion and 48,000 associates, Ecolab delivers comprehensive solutions and on-site service to promote safe food, maintain clean environments, optimize water and energy use, and improve operational efficiencies for customers in the food, healthcare, energy, hospitality and industrial markets in more than 170 countries around the world. Follow us on Twitter @ecolab, Facebook at facebook.com/ecolab, LinkedIn at Ecolab or Instagram at Ecolab Inc.
11/12/2017

Jason Hannon, CEO, to host business update call

Dublin – Ireland, 11 December 2017 – Mainstay Medical International plc (“Mainstay” or the “Company”, Euronext Paris: MSTY.PA and ESM of the Irish Stock Exchange: MSTY.IE), a medical device company focused on bringing to market ReActiv8®, an implantable restorative neurostimulation system to treat disabling Chronic Low Back Pain, announces a positive outcome of the Interim Analysis in its U.S. Pivotal ReActiv8-B Study (the “Study”), comprising a definitive size and an estimated completion date.
Key updates relative to the Study (and its design) are as follows:
• The Study utilizes an adaptive trial design, inclusive of an Interim Analysis, to determine the definitive size of the Study of up to 232 patients in the pivotal cohort. With this adaptive design, Mainstay commenced the Study with a sample size of 128 patients pending the Interim Analysis;
• The independent Data Monitoring Committee (“DMC”) has completed the Interim Analysis, which is based on data from the first 58 patients in the pivotal cohort to complete the primary endpoint. The DMC has recommended continuation of the Study with a definitive size of 168 evaluable patients. The ultimate number of patients in the Study will be slightly higher than 168 due to the nature of the enrollment process;
• The DMC also reported that they have observed no safety concerns in the Study; and
• The Study is expected to be fully enrolled by the end of the second quarter of 2018, and the Company expects to announce full data readout towards the end of 2018.
133 patients have been implanted in the pivotal cohort in the Study to date and clinical study sites have continued to implant patients pending the outcome of the Interim Analysis.
The Study is intended to gather data in support of an application for pre-market approval (PMA) from the U.S. Food and Drug Administration (FDA), a key step towards commercialization of ReActiv8 in the U.S..
Richard Rauck MD, President and Founder, Carolinas Pain Institute, Medical Director for The Center for Clinical Research, Pain Fellowship Director at Wake Forest University School of Medicine and Chairman of the DMC said: “The outcome of the Interim Analysis has validated the adaptive Study design agreed with the FDA, which provided for a sample size of up to 232 patients in the pivotal cohort. By following this course, we have been able to determine an appropriate sample size for the Study. We also reviewed the safety data and I am pleased to report there are no safety concerns in the Study thus far.”
Jason Hannon, CEO, said: “We are pleased to now have a definitive enrollment goal for the Study, which compares favorably in total size to other neuromodulation studies. We will now push forward to the data collection point in a highly efficient manner, and I am confident we will complete the Study in the coming months and announce the full data readout towards the end of 2018.”

Press Release
Chris Gilligan MD, Chief, Division of Pain Medicine, Department of Anesthesiology, Perioperative and Pain Medicine, Brigham & Women’s Hospital, Assistant Professor of Anaesthesia, Harvard Medical School, and Principal Investigator for the ReActiv8-B Study said: “The treatment of Chronic Low Back Pain is a significant challenge around the world with many patients experiencing disabling effects for large portions of their lives. These patients can be left with few treatment options. I’m proud to be part of a clinical study that is analyzing whether we can use ReActiv8 to help patients truly restore their muscle function and thereby reduce the effects of chronic pain, rather than rely on short term analgesics such as opioids. I look forward to the compilation of the data.”
Investor Conference Call and Business Update
Jason Hannon will host an investor conference call to discuss this positive news and will also provide a broader business update at 16:00 GMT (11:00 EST) on 11 December 2017 in English.
Dial-ins for the call are outlined below:
Europe: +44 203 139 4830
Ireland: +353 1 696 8154
USA: +1 718 873 9077
Participant PIN: 65178331#

In addition to the update on the Interim Analysis, the business update will include the following developments:
• The Company continues to advance the initial commercialization of ReActiv8 in Europe. Our European commercial activities are initially focused on Germany, where we are working to drive adoption in a select number of high volume spine care centers to develop reference sites. Four centers in Germany and Ireland have implanted patients with ReActiv8 to date, and several additional sites have been trained. We have begun recruiting an experienced sales team of direct Mainstay employees. The team currently consists of eight people, located in key regions in Germany and one person in Ireland.
• Mainstay is planning to add to its investment in commercial infrastructure to expand commercialization in Europe and in preparation for commercialization in other markets including Australia and the U.S. We will be building a market development team of clinical experts to drive market penetration, identify the right physician partners, help educate the market about Reactiv8, and support implants.
• We will also increase our investment in the training of physicians; the education of referring physicians regarding the potential of ReActiv8; and in the collection and dissemination of clinical data regarding the expanding use of ReActiv8.
• Cash on hand at 30 November 2017 was $12.7 million. The Company on an ongoing basis explores potential financing opportunities to fund the business.

Press Release
This announcement contains inside information within the meaning of the EU Market Abuse Regulation 596/2014

About Mainstay
Mainstay is a medical device company focused on bringing to market an innovative implantable restorative neurostimulation system, ReActiv8®, for people with disabling Chronic Low Back Pain (CLBP). The Company is headquartered in Dublin, Ireland. It has subsidiaries operating in Ireland, the United States, Australia, Germany and the Netherlands, and its ordinary shares are admitted to trading on Euronext Paris (MSTY.PA) and the ESM of the Irish Stock Exchange (MSTY.IE).

About the ReActiv8-B Study
The ReActiv8-B Study is an international, multi-center, prospective randomized sham controlled blinded trial with one-way crossover conducted under an Investigational Device Exemption (IDE). In summary, this means that eligible subjects will have baseline data collected and then following verification that the enrollment criteria are met, ReActiv8 will be implanted. At the 14-day post implant follow up visit, half the patients will be randomized to receive appropriately programmed stimulation (the treatment arm), and half will be randomized to receive sham stimulation/minimal stimulation (the control arm) .The ReActiv8-B Study is designed to generate data to form part of the Pre-Market Approval Application (PMAA) of ReActiv8 to the FDA.

About Chronic Low Back Pain
One of the recognized root causes of CLBP is impaired control by the nervous system of the muscles that dynamically stabilize the spine in the low back, and an unstable spine can lead to back pain. ReActiv8 is designed to electrically stimulate the nerves responsible for contracting these muscles and thereby help to restore muscle control and improve dynamic spine stability, allowing the body to recover from CLBP.
People with CLBP usually have a greatly reduced quality of life and score significantly higher on scales for pain, disability, depression, anxiety and sleep disorders. Their pain and disability can persist despite the best available medical treatments, and only a small percentage of cases result from an identified pathological condition or anatomical defect that may be correctable with spine surgery. Their ability to work or be productive is seriously affected by the condition and the resulting days lost from work, disability benefits and health resource utilization put a significant burden on individuals, families, communities, industry and governments.
Further information can be found at www.mainstay-medical.com
CAUTION – in the United States, ReActiv8 is limited by federal law to investigational use only.

PR and IR Enquiries:
Consilium Strategic Communications (international strategic communications – business and trade media)
Chris Gardner, Jessica Hodgson, Hendrik Thys
Tel: +44 203 709 5700 / +44 7921 697 654
Email: mainstaymedical@consilium-comms.com

FTI Consulting (for Ireland)
Jonathan Neilan Tel: +353 1 765 0886
Email: jonathan.neilan@fticonsulting.com

NewCap (for France
Julie Coulot
Tél. : +33 1 44 71 20 40
Email: jcoulot@newcap.fr
AndreasBohne.Com/Kötting Consulting (for Germany)
Andreas Bohne
Tel: +49 2102 1485368
Email: abo@andreasbohne.com

Press Release
Investor Relations:
LifeSci Advisors, LLC
Brian Ritchie
Tel: +1 (212) 915-2578
Email: britchie@lifesciadvisors.com

ESM Advisers:
Davy
Fergal Meegan or Barry Murphy
Tel: +353 1 679 6363
Email: fergal.meegan@davy.ie or barry.murphy2@davy.ie

Forward looking statements
This announcement includes statements that are, or may be deemed to be, forward looking statements. These forward looking statements can be identified by the use of forward looking terminology, including the terms “anticipates”, “believes”, “estimates”, “expects”, “intends”, “may”, “plans”, “projects”, “should”, “will”, or “explore” or, in each case, their negative or other variations or comparable terminology, or by discussions of strategy, plans, objectives, goals, future events or intentions. These forward looking statements include all matters that are not historical facts. They appear throughout this announcement and include, but are not limited to, statements regarding the Company’s intentions, beliefs or current expectations concerning, among other things, the Company’s results of operations, financial position, prospects, financing strategies, expectations for product design and development, regulatory applications and approvals, reimbursement arrangements, costs of sales and market penetration.
By their nature, forward looking statements involve risk and uncertainty because they relate to future events and circumstances. Forward looking statements are not guarantees of future performance and the actual results of the Company’s operations, and the development of its main product, the markets and the industry in which the Company operates, may differ materially from those described in, or suggested by, the forward looking statements contained in this announcement. In addition, even if the Company’s results of operations, financial position and growth, and the development of its main product and the markets and the industry in which the Company operates, are consistent with the forward looking statements contained in this announcement, those results or developments may not be indicative of results or developments in subsequent periods. A number of factors could cause results and developments of the Company to differ materially from those expressed or implied by the forward looking statements including, without limitation, the successful launch and commercialization of ReActiv8®, the progress and success of the ReActiv8-B Clinical Study, the ability to raise additional capital to fund the Company’s business and the cost of such capital, general economic and business conditions, the global medical device market conditions, industry trends, competition, changes in law or regulation, changes in taxation regimes, the time required to commence and complete clinical trials, the time and process required to obtain regulatory approvals, currency fluctuations, changes in its business strategy, political and economic uncertainty. The forward-looking statements herein speak only at the date of this announcement.

11/12/2017

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

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

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

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

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

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

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

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

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

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

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

08/12/2017

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

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

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

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

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

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

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

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

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

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

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

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

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

29/11/2017

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

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

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

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

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

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

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

FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding QR-313 and the clinical development and the therapeutic potential thereof, statements regarding ODD and the potential benefits thereof, and statements regarding our pipeline of programs targeting DEB. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that we may not realize the intended benefits afforded by orphan drug designation for our QR-313 program targeting DEB, positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.:
Investor and Media Contact:
Bonnie Ortega
T: +1 858 245 3983
ir@proqr.com

29/11/2017

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

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

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

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

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

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

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

15/11/2017

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

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

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

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

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

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

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