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10/07/2018

Paris, France, July 10th. 2018. Sofinnova Partners, a leading venture capital firm specialized in Life Sciences, today announced that Otsuka Holdings is acquiring its portfolio company ReCor Medical, a medical device company specialized in the treatment of hypertension. The terms of the acquisition are being withheld due to non-disclosure obligations.

ReCor Medical was created in 2009 by Sofinnova Partners, Mano Iyer – who was then entrepreneur-in-residence at Sofinnova Partners and now Chief Operating Officer of ReCor – and Professor Jacques Seguin, MD, who became a large private investor in ReCor. Prof. Seguin was previously founder and CEO of CoreValve, a past Sofinnova portfolio company and a leader in the transcatheter valve replacement space, which was sold to Medtronic. Sofinnova Partners was the sole venture capital investor in ReCor Medical and remained its largest shareholder until the sale to Otsuka.

ReCor Medical is an innovative medical device company that developed the Paradise System, a proprietary ultrasound ablation system for renal denervation (RDN). RDN is a new potential therapeutic approach for the treatment of hypertension, one of the most prevalent medical conditions. ReCor recently announced positive results of its landmark RADIANCE-HTN SOLO hypertension study at EuroPCR 2018.

Antoine Papiernik, Managing Partner at Sofinnova Partners and ReCor Board Member, said: “ReCor perfectly illustrates our investment strategy: we worked hand-in-hand with Mano Iyer to create the business vision and plan for ReCor. We then founded and funded the company, and opened our network of experts, key opinion leaders and board members to help grow it. We brought trusted entrepreneurs Jay Watkins as Chairman and Andy Weiss as CEO to help guide and operate the company through to a corporate transaction to our partner Otsuka.”

Jay Watkins, Chairman of ReCor Medical said: “Sofinnova Partners remains one of few VCs willing to fund early-stage med-tech ventures targeting large and important new markets. The firm played a critical role throughout ReCor’s life, and has proven to be a reliable, value-added partner for the company. The field of renal denervation has been a complex one over the last few years with periods of euphoria and periods of doubt. Sofinnova Partners’ support remained constant throughout, helping to build a strong partnership with Otsuka and then navigate through the challenges to a very successful trade sale.”

Mano Iyer, Founder and COO of ReCor Medical added: “ReCor is a success story because Sofinnova Partners, consistent with its philosophy, saw the value of an opportunity which did not yet exist. It had the vision to create and fund the company, not only in the very beginning, but also during the critical early years. Despite the dramatic swings in the field, Sofinnova Partners’ confidence in me and in the management team was essential to keep us motivated when others lost hope. This great exit is therefore particularly sweet.”

Andrew M. Weiss, CEO of ReCor Medical adds: “I came to ReCor thanks to Antoine Papiernik’s introduction to the company. With his help, our team developed the partnership with Otsuka and was able to remain focused on value creation. The recent announcement of our positive RADIANCE-HTN SOLO study results and now the merger with Otsuka demonstrate that our teamwork with Sofinnova Partners was successful. We now have an opportunity to transform the treatment of hypertension and benefit millions of potential patients while providing a solid return for our investors. I look forward to continuing to work to make this technology a possible standard of care in hypertension treatment”.

For more information, please contact:
SOFINNOVA PARTNERS
International: Anne Rein
Tel: +33 6 03 35 92 05
e-mail: anne.rein@strategiesimage.com
United States: Kate Barrette
Tel: +1 212 223 0561
e-mail: kbarrette@rooneyco.com

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

About ReCor Medical, Inc.
ReCor Medical is a medical device company that designs and manufactures the Paradise System, a proprietary ultrasound ablation system for renal denervation (RDN). RDN is a new potential therapeutic approach for the treatment of hypertension, one of the most prevalent medical conditions. The Paradise System is approved for sale in the EU and bears a CE mark, but is not approved for sale in the United States. The System’s intravascular catheters denervate renal nerves by combining the protection of water-based cooling of the renal artery with high intensity ultrasound energy for circumferential renal nerve ablation. The Paradise System has been studied in clinical trials of approximately 300 patients to date. Following the positive outcomes of the RADIANCE-HTN SOLO trial, ReCor will continue its evaluations of Paradise in RADIANCE-HTN TRIO (a feasibility study of patients with resistant hypertension) and REQUIRE (a pivotal study of patients with resistant hypertension in Japan and Korea), and launch the RADIANCE II pivotal study (a study of patients with moderate hypertension) in the United States and Europe.
http://www.recormedical.com/

About Otsuka Holdings Co., Ltd. and Otsuka Medical Devices Co., Ltd.
Otsuka Holdings Co., Ltd. is the holding company of the Otsuka group, a global healthcare group headquartered in Tokyo, Japan. With operations in pharmaceuticals, nutraceuticals, medical devices and other health-related businesses, the group generated worldwide sales of JPY1,240 billion in the fiscal year ended December 2017.
http://www.otsuka.com/en/

Established in 2011, Otsuka Medical Devices Co., Ltd. is a fully-owned subsidiary of Otsuka Holdings and one of its core operating subsidiaries. Otsuka Medical Devices focuses on the development and commercialization of endovascular devices that provide new therapeutic options in areas where patient needs cannot be met through pharmaceutical or other conventional treatment.
Otsuka Medical Devices conducts the REQUIRE trial for renal denervation in hypertensive patients (n=140), who are uncontrolled on 3 or more medications including a diuretic, in Japan and Korea through its subsidiary JIMRO Co., Ltd.
http://www.omd.otsuka.com/en/

15/06/2017

Montreal, Canada, June 15th, 2017. BioAmber Inc. (NYSE: BIOA, TSX: BIOA) is pleased to announce the launch of BIO-SA™ pharmaceutical grade. This new grade of material provides a USP/NF and FCC Grade of bio-succinic acid manufactured under the United States Food and Drug Administration’s (FDA) good manufacturing practices (GMP) for food and Excipients.  The United States Pharmacopeia (USP), the National Formulary (NF), and the Food Chemicals Codex (FCC) are the public pharmacopeia standards for medicines, food ingredients, dietary supplement products, and ingredients.

These standards are used by regulatory agencies and manufacturers to ensure products are of the appropriate identity, strength, quality, purity and consistency.

“Having met the stringent requirements contained in the NF and FCC reflects BioAmber’s continued dedication to quality through our best-in-class production and purification processes. By achieving this new benchmark, our global customers in the food, pharmaceutical and dietary supplement industries can be assured BioAmber’s facility adheres to the rigorous quality control standards set by the United States Food and Drug Administration (FDA).” said Fabrice Orecchioni, BioAmber’s President & COO. “This grade will allow BioAmber to supply these high-value industries with commercial volumes of an FDA regulated bio-succinic acid, a grade previously unavailable to these markets before today”, he added.

About BioAmber
BioAmber (NYSE: BIOA, TSX: BIOA) is a renewable materials company. Its innovative technology platform combines biotechnology and catalysis to convert renewable feedstock into building block materials that are used in a wide variety of everyday products including plastics, paints, textiles, food additives and personal care products.  For more information visit www.bio-amber.com

Forward-Looking Statements
This press release contains forward-looking statements, which are subject to substantial risks, uncertainties and assumptions. These statements often include words such as “believe,” “expect,” “anticipate,” “intend,” “plan,” “estimate,” “seek,” “will,” “may” or similar expressions. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee that the events and circumstances reflected in the forward-looking statements will be achieved or occur and the timing of events and circumstances and actual results could differ materially from those projected in the forward- looking statements. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.   For additional disclosure regarding these and other risks faced by BioAmber, see disclosures contained in BioAmber’s public filings with the SEC including, the « Risk Factors » section of BioAmber’s most recent Annual Report on Form 10-K and the recent quarterly report on Form 10-Q.

BioAmber Investor Contact
Roy McDowall
Sr. VP Business Development
Tel (514) 844 8000 ext 260
roy.mcdowall@bio-amber.com

15/06/2017

Key program features and updates:

  • ProQR to host an R&D day in New York today, June 15, from 8:00am to 1:00pm Eastern Standard Time. The live webcast can be accessed at www.proqr.com/rd-day.
  • Cystic Fibrosis (CF): Full data from the nasal potential difference (NPD) study for QR-010, ProQR’s lead molecule for CF that is studied in two clinical trials in patients, will be presented, along with an update on the ongoing Phase 1b study, for which topline data are expected in September 2017. Steven M. Rowe, M.D., MSPH, a renowned expert in cystic fibrosis research, will discuss the importance of NPD as a biomarker and the unmet needs remaining in the CF treatment landscape.
  • Leber’s Congenital Amaurosis (LCA): Details of the clinical trial for QR-110, ProQR’s lead molecule in the ophthalmology pipeline. Additional preclinical data will be presented.
  • Ophthalmology pipeline: An update and data for the next four programs in the ophthalmology pipeline will be provided, including: QRX-411 and QRX-421 for Ushers syndrome, QRX-504 for Fuchs Endothelial Corneal Dystrophy (FECD) and QRX-1011 for Stargardt’s Disease. Stephen M. Rose, Ph.D., Chief Research Officer at Foundation Fighting Blindness will provide a background on inherited retinal diseases.
  • Dystrophic epidermolysis bullosa (DEB): an update on key pre-clinical functional and delivery data, and an update on preparation for clinical development to be provided for QR-313. M. Peter Marinkovich, M.D., a dermatologist and Director of the Stanford EB disease clinic, will discuss the unmet need in DEB and the current treatment landscape.
  • Axiomer®: ProQR will introduce its novel, proprietary RNA editing platform technology.  Art Levin, Ph.D., an internationally recognized expert on the development of oligonucleotide-based therapeutics, will discuss the evolution of RNA therapeutics.
  • As of March 31, 2017, the Company’s current cash of €52.1 million provides a runway into Q3 2018.

LEIDEN, the Netherlands, June 15, 2017 – ProQR Therapeutics N.V. (Nasdaq:PRQR), a development stage RNA therapeutics company will provide an update on its product candidates today at an investor event, and introduce Axiomer®, a novel RNA platform technology it pioneered. The R&D Day is hosted by the Company’s management team and will include perspectives from several key opinion leaders. ProQR’s pipeline now includes two clinical programs, one preclinical program and two programs ready to enter development.

“Since last year we have made good progress on executing on our strategy to develop life-changing therapies for patients in need, through a diversified pipeline with a balanced risk profile,” said Daniel A. de Boer, CEO of ProQR. “We are pleased to show the progress we have made in our RNA therapeutics pipeline at our second R&D day.  Following QR-010 for CF, QR-110 is now in clinical trials for LCA 10, and our third molecule, QR-313 for DEB will move to clinical trials early next year.  We are focusing on three important genetic diseases, all with high unmet needs, and all which we believe could greatly benefit from our unique RNA oligonucleotide approach. Within the next 18 months we will have generated clinical data in patients in all these programs.”

Axiomer®editing the RNA
ProQR is pioneering a next-generation RNA technology called Axiomer®, which we believe has the potential to yield a new class of medicines for genetic diseases. Axiomer® can make single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells. The Axiomer® “Editing Oligo Nucleotides”, or EONs, recruit an endogenously expressed RNA editing system called ADAR, which it can direct to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G). A member of the Scientific Advisory Board, Dr. Levin will present the landscape and evolution of RNA therapeutics and provide his perspective on this unique and proprietary platform technology.

“While our focus and priority is on clinical development of our most advanced RNA-based therapeutics to help patients with CF, LCA 10 and DEB, we continue to innovate in RNA science. This innovation effort has led to the discovery of a novel RNA editing technology that we believe can address the underlying cause of a broad range of genetic defects at the RNA level,” said Daniel de Boer.  “The invention and patenting of Axiomer® can drive drug discovery and development of a new class of therapeutics, independently and through partnerships.”

The R&D Day will feature presentations by ProQR senior management including Daniel de Boer (Chief Executive Officer), Noreen Henig (Chief Medical Officer), Peter Adamson (Head of Ophthalmology), Gerard Platenburg (Chief Innovation Officer) and David Rodman (Chief Development Strategy Officer).  Discussions will focus on a review and introduction of several near- and medium term value drivers and progress on the Company’s pipeline. In addition, several leading medical researchers will discuss the state of the art in research and development in relation to the company’s pipeline:

  • Steven M. Rowe, M.D., MSPH, Professor, Department of Medicine, Pediatrics, and Cell Developmental & Integrative Biology, and Director Gregory Fleming James Cystic Fibrosis Research Center University of Alabama.

Dr. Rowe is a respected academic physician scientist and a pioneer in the field of personalized therapeutics for cystic fibrosis, cutting-edge discovery in airway disease biology, and translational research in COPD.  He is an international authority in the design and conduct of clinical trials targeting the basic CF defect, and has made key advances in the measurement and interpretation of CFTR function. He directs the Cystic Fibrosis Research Center at UAB, which involves over 100 faculty members and has been continuously funded for over 25 years.  A board-certified physician, Dr. Rowe serves as a Special Consultant for Translational Science for the Cystic Fibrosis Foundation.  He presently has a laboratory of over 25 individuals, embracing lung research from basic discovery, to translational science, to clinical application.

  • Stephen M. Rose, Ph.D., Chief Research Officer at Foundation Fighting Blindness.

Dr. Rose oversees the day-to-day operations of the Foundation Fighting Blindness’ Science Department. He also works closely with the clinical arm of the Foundation to establish a seamless pipeline of studies to move preventions and treatments into clinical trials, partnering with biotech and pharma to maximize potential commercialization.  Prior to joining the Foundation in 2004, Dr. Rose was a Director in the NIH Office of Science Policy, where he provided oversight on issues regarding recombinant DNA, including human gene transfer clinical protocols. Dr. Rose currently sits on the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee and is a Health Research Alliance Board member.

  • M. Peter Marinkovich, M.D., Associate Professor, Blistering Disease Clinic Department of Dermatology, Stanford University School of Medicine.

Dr. Marinkovich is an Associate Professor of Dermatology, a faculty member of the Program in Epithelial Biology and the Stanford Cancer Biology Program. He has an interest in inflammatory skin disease and is Director of the Stanford Epidermolysis Bullous Disease and Psoriasis Clinics. He is also an attending dermatologist at the VA Palo Alto Medical Center. Dr. Marinkovich’s research focuses on pathogenesis and therapy of epidermolysis bullosa, psoriasis, hair disorders and skin cancers.

  • Art Levin, Ph.D., international RNA expert and member of the ProQR Scientific Advisory Board.

Dr. Levin has three decades of experience in RNA drug development from discovery through drug registration, both in large pharma and biotech companies. He has been key to the development of numerous of oligonucleotides, including the first approved antisense medicines, and the first microRNA-targeted therapeutic in clinical trials. Dr. Levin has published over 60 scientific articles and served as a director of the Oligonucleotide Therapeutics Society.  He has served on ProQR’s Scientific Advisory Board since the company’s inception.

R&D Day Event details

Today, ProQR will host an R&D day in New York, NY from 8:00am to 1:00pm ET.  Please email Ronen Abergel, rabergel@troutgroup.com to receive more information and to reserve a seat.

Webcast
The live webcast can be accessed at www.proqr.com/rd-day. The archived webcast of the presentation will be accessible from the ‘Investor Relations’ section of ProQR’s website (www.proqr.com) under ‘Events and Presentations’. The archived webcast will be available for 90 days following the presentation date.

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 the therapeutic potential of our RNA technology, our innovation programs, the timing of our clinical programs and availability of data and our R&D day. 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, 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.

Contact:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com

08/06/2017

 Achèvement de l’étude de tolérance en doses uniques du CER-209
 Aucun problème de sécurité et de tolérance lié au candidat-médicament
 La pharmacocinétique observée soutient une prise unique quotidienne de CER-209
L’étude de tolérance des doses multiples est désormais prête à être lancée

Toulouse, FRANCE, Ann Arbor, ETATS-UNIS, 7 juin 2017, 19h00 – Cerenis Therapeutics (FR0012616852 – CEREN – Éligible PEA PME), société biopharmaceutique internationale dédiée à la découverte et au développement de thérapies innovantes basées sur le métabolisme des lipides pour le traitement des maladies cardiovasculaires et métaboliques, annonce aujourd’hui des résultats positifs de la Phase I de CER-209 en dose unique dans le traitement des Hépatites Graisseuses Non Alcooliques (NAFLD) et de la Stéato-Hépatite Non Alcoolique (NASH).
L’objectif de l’étude de tolérance en doses uniques menée aux Etats-Unis était d’évaluer la sécurité, la tolérance ainsi que le profil pharmacocinétique de CER-209 lors de la prise par voie orale d’une dose unique. Des doses croissantes de 1, 3 ,10 et 30 mg ont été testées sur 24 sujets, traités en 4 cohortes de 6 patients. Dans chaque cohorte, 4 sujets ont été traités avec le candidat-médicament de l’étude tandis que les 2 autres ont reçu le placebo.
« Les résultats positifs de l’étude de tolérance après la prise de doses uniques nous permettent de procéder à la prochaine étape du développement clinique de CER-209, à savoir l’étude de sécurité et de tolérance après la prise de doses multiples. En l’absence de solutions de traitement des NAFLD et de la NASH, CER-209 pourrait jouer un rôle majeur dans le traitement de la stéatose hépatique et de l’athérosclérose. L’avantage majeur de CER-209 dans le traitement des NAFLD et de la NASH réside dans sa capacité à favoriser la reconnaissance des HDL et l’élimination des lipides par le foie, via l’activation des voies naturelles métaboliques par l’intermédiaire du récepteur P2Y13. De plus, la confirmation du profil pharmacocinétique de CER-209 qui autorise une prise unique quotidienne orale, constitue une très bonne nouvelle pour l’adoption du traitement par les patients », déclare le Dr Jean-Louis Dasseux, fondateur et Directeur Général de Cerenis.
CER-209, un agoniste du récepteur P2Y13, est bien adapté au traitement de la NAFLD et de la NASH.
La NAFLD, précurseur de la NASH est un problème de santé majeur, cette indication étant aujourd’hui considérée comme la maladie du foie la plus fréquente dans le monde occidental, impactant au niveau mondial près de 30% de la population1. Les études épidémiologiques démontrent que le risque cardiovasculaire augmente chez les patients atteints de stéato-hépatite et que les maladies cardiovasculaires associées représentent les principales causes de décès chez les patients souffrant d’une stéatose du foie1,2.
Dans ces modèles précliniques, CER-209 entraîne une réduction marquée de la stéato-hépatite telle que déterminée par une réduction des taux de cholestérol, de triglycérides et d’acides gras dans le foie par rapport au placebo. En outre, CER-209 induit des diminutions importantes des enzymes hépatiques (ALT et AST) dans le plasma. Ces effets suggèrent le rétablissement de l’intégrité du foie et montrent le fort potentiel de CER-209 pour le traitement des maladies du foie telles que les NAFLD et la NASH tout en diminuant le risque des maladies cardiovasculaires associées. CER-209, qui exerce son effet bénéfique sur la stéatose du foie via une action spécifique sur la voie d’élimination du cholestérol, dispose d’un solide potentiel pour le traitement des NAFLD et de la NASH.

A propos du récepteur P2Y13
Le récepteur P2Y13 est un membre de la famille bien connue des récepteurs P2Y, qui comprend notamment le récepteur P2Y12, cible de médicaments à succès tels que l’agent anti-thrombotique Clopidogrel (Plavix®). La déficience du récepteur P2Y13 dans les modèles précliniques se traduit par une réduction des sécrétions de lipides biliaires, de leur élimination et de celle des acides biliaires dans les selles.
La déficience conduit à un dysfonctionnement du transport retour du cholestérol (ou RLT) depuis les macrophages jusqu’aux selles. L’activation du récepteur P2Y13 par de petites molécules, agissant comme ligands, stimule l’élimination des HDL du plasma sanguin ainsi que la reconnaissance des HDL par le foie, augmentant par conséquent la sécrétion des lipides biliaires et stimulant globalement le RLT3.

A propos du CER-209
CER-209 est le premier candidat-médicament dans sa catégorie, celle des agonistes du récepteur P2Y13. CER-209 est un agoniste spécifique du récepteur P2Y13 et il n’interagit pas avec le récepteur P2Y12. Les études précliniques ont montré que le CER-209 augmente la reconnaissance des HDL par le foie et améliore le transport retour du cholestérol (RLT), conduisant finalement à la régression de la plaque d’athérome. En raison des effets métaboliques favorables observés sur le foie au cours des expériences précliniques, le CER-209 pourrait offrir un mécanisme nouveau pour le traitement des Hépatites Graisseuses Non Alcooliques (NAFLD) et de la Stéato-Hépatite Non Alcoolique (NASH).

A propos de Cerenis : www.cerenis.com
Cerenis Therapeutics Holding est une société biopharmaceutique internationale dédiée à la découverte et au développement de thérapies innovantes basées sur le métabolisme de lipides pour le traitement des maladies cardiovasculaires et métaboliques. Le HDL est le médiateur primaire du transport retour du cholestérol (ou RLT), la seule voie métabolique par laquelle le cholestérol en excès est retiré des artères et transporté vers le foie pour élimination du corps.
Cerenis développe un portefeuille de thérapies HDL, dont des mimétiques de particules HDL pour traiter la déficience génétique en HDL, ainsi que des molécules qui augmentent le nombre de particules HDL chez les patients qui en ont peu pour le traitement de l’athérosclérose et des maladies métaboliques associées telles que les Hépatites Graisseuses Non Alcooliques (NAFLD) et la Stéato-Hépatite Non Alcoolique (NASH).
Cerenis est bien positionné pour devenir un des leaders du marché des thérapies basées sur le métabolisme des lipides avec un riche portefeuille de programmes en développement.

Contacts :
Cerenis
Jean-Louis Dasseux
CEO
info@cerenis.com
+33 (0)5 62 24 09 49

NewCap
Relations Investisseurs
Emmanuel Huynh / Louis-Victor Delouvrier
cerenis@newcap.eu
+33 (0)1 44 71 98 53

NewCap
Relations Médias
Nicolas Merigeau
cerenis@newcap.eu
+33 (0)1 44 71 94 98

31/05/2017

Key Updates
• ProQR receives Fast Track designation by the U.S. Food and Drug Administration (FDA). Closer interaction with FDA could potentially accelerate the development of QR-110 in patients with Leber’s Congenital Amaurosis Type 10 (LCA 10).
• LCA 10 is one of the most prevalent forms of gene-related blindness in children worldwide and currently there are no therapies commercially available or in clinical development.
• QR-110 is currently in clinical development with the planned Phase 1/2 open-label trial (PQ-110-001) that will assess the safety, tolerability, pharmacokinetics and efficacy of multiple administrations of QR-110 in one eye of each patient and will include approximately 6 adults and 6 children with LCA 10.
• Top-line trial results are expected in 2018.

LEIDEN, the Netherlands, May 31, 2017 – ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for QR-110, the lead molecule in its ophthalmology pipeline. QR-110 is being developed for the treatment of patients with Leber’s Congenital Amaurosis Type 10 (LCA 10), a rare genetic disease that causes individuals to lose sight, often in the first years of life. QR-110 is a novel investigational RNA oligonucleotide targeting LCA 10 due to the p.Cys998X mutation, which is one of the most prevalent forms of gene-related blindness in children and currently there are no disease modifying therapies commercially available or in clinical development.
Fast Track designation is granted by the FDA to drugs that are under development for serious conditions and have the potential to fulfill an unmet medical need. It was established with the intention to bring promising drugs to patients sooner by facilitating the development with more frequent FDA interactions and expediting the review process.
“QR-110 is a unique and elegant approach to addressing the underlying genetic defect that leads to blindness in individuals with LCA 10 due to the p.Cys998X mutation. We are very pleased with granting of the Fast Track designation by the FDA for this program as it highlights the need for innovative and efficacious medicines for this devastating disease for which there is currently nothing available,” said Noreen R. Henig, Chief Medical Officer of ProQR. “We are also excited to be able to initiate our first trial for QR-110 as a multidose study and for that we will benefit from the Fast Track Designation. We believe development of QR-110 also opens the possibilities for RNA approaches to target other causes of genetic blindness. We are building our pipeline in ophthalmology and will use our rapid development approach to QR-110 as a model for how to bring RNA therapeutics to patients in need.”

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 Type 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-110
QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis Type 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore wild-type CEP290 mRNA leading to the production of wild-type 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.

About Leber’s Congenital Amaurosis Type 10
Leber’s congenital amaurosis is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of which LCA Type 10 (LCA 10) is one of the more severe forms. LCA 10 is caused by mutations in the CEP290 gene of which the p.Cys998X mutation is most common. LCA 10 leads to progressive loss of vision causing most patients to lose their sight in the first few years of life. To date, there are no treatments approved or products in clinical development that treat the underlying cause of the disease. Although prevalence rates vary, we believe approximately 2,000 people in the Western world have LCA 10 because of this mutation.

About the PQ-110-001 Study
PQ-110-001 is an open-label trial that will include approximately 6 children (age 6- 17 years) and 6 adults (≥ 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients will receive four intravitreal injections of QR-110 into one eye; one every three months for one year. The QR-110 trial is expected to be conducted in three centers with significant expertise in genetic retinal disease in the US and Europe.
The primary endpoints will be safety and tolerability. Secondary efficacy endpoints will assess the pharmacokinetics and restoration/improvement of visual function and retinal structure through ophthalmic tests such as visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR), mobility course and fixation stability. Changes in quality of life in LCA subjects will also be evaluated. Top-line results from the trial are expected to be available in 2018.

FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding QR-110 and the clinical development and the therapeutic potential thereof, statements regarding PQ-110-001, including trial design and expected timing of results, statements regarding Fast Track designation, and statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in our innovation pipeline. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, that a Fast Track designation by the FDA may not actually lead to a faster development, regulatory review or approval process, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com

22/05/2017

Paris, France – 22 mai 2017 — Sofinnova Partners, un leader du capital-risque en Europe spécialisé dans les sciences de la vie, annonce la promotion de Graziano Seghezzi en tant que Managing Partners. Il rejoint le Managing Partnership composé de Antoine Papiernik, Denis Lucquin, et Monique Saulnier. Cette nomination donne un coup d’accélérateur à la dimension internationale de Sofinnova Partners.

C’est en 2001 que Graziano a démarré sa carrière dans le capital-risque chez Sofinnova Partners où il est chargé de trouver et analyser les nouvelles opportunités d’investissement. Tout au long de son parcours, il s’est engagé pour promouvoir la création d’entreprises, à la fois par des créations de start up, mais aussi par la réalisation de spin-offs et la mise en place d’accélérateurs. Il a été l’investisseur de démarrage d’Omthera Pharmaceuticals, initialement cotée sur le Nasdaq puis vendu à Astra Zeneca, de Glycovaxyn, vendu à GSK, et de Creabilis vendu à Sienna Biopharmaceuticals. Il a également investi et siège au conseil d’administration de Breath Therapeutics, Corvidia Therapeutics, Crescendo Biologics, Hookipa Biotech, Inotrem et Mission Therapeutics. Il a co-fondé BioVelocita, le premier accélérateur italien. Entre 2003 et 2006, Graziano travaille chez Index Ventures à Genève.

Scientifique de formation, il a passé cinq ans comme chercheur médical, spécialisé dans l’oncologie et les maladies cardiovasculaires, à l’Ecole de Médecine de NYU (Etats Unis). Graziano est diplômé de l’Université de Pavia (Italie) en génétique et microbiologie et détient un MBA de RSM Erasmus University.

Graziano Seghezzi déclare : « J’ai un respect immense pour l’équipe de Sofinnova, tant sur le plan professionnel que personnel ; et suis particulièrement fier d’avoir été promu Managing Partner. Ce nouveau rôle intervient à un moment charnière pour le Managing Partnership qui vient de définir un ambitieux projet de croissance. Avec mon expertise internationale, je suis heureux de participer à la consolidation du leadership global de Sofinnova Partners dans les sciences de la vies ».

Antoine Papiernik, Président de Sofinnova Partners, poursuit : « Graziano a démarré chez Sofinnova Partners il y a quinze ans alors qu’il entamait sa reconversion professionnelle de chercheur à investisseur dans les biotech. Aujourd’hui, Graziano a bâti un track record exceptionnel à la fois en Europe et aux Etats Unis, et nous sommes ravis qu’il rejoigne ainsi le Managing Partnership ».

 

Contact presse pour SOFINNOVA PARTNERS

Anne REIN

Tel:     +33 6 03 35 92 05

@:      anne.rein@strategiesimage.com

 

17/05/2017

L’ETUDE DE NOXXON DANS LE CANCER DU PANCREAS METASTATIQUE ET LE CANCER COLORECTAL METASTATIQUE, MENEE DANS LE CADRE D’UN ACCORD DE COLLABORATION AVEC MERCK & CO/MSD, SE DÉROULERA DANS UN CENTRE DE CANCÉROLOGIE DE RENOMMÉE MONDIALE

Berlin, Allemagne, le 16 mai 2017 – NOXXON Pharma N.V. (Alternext Paris : ALNOX), une société biopharmaceutique développant principalement des traitements contre le cancer ciblant le microenvironnement tumoral, annonce aujourd’hui la signature d’un accord avec le Centre National des Maladies Tumorales (NCT) de Heidelberg, selon lequel ce dernier mènera une étude évaluant son produit candidat phare NOX-A12 associé au Keytruda® (pembrolizumab) dans le cancer du pancréas métastatique et le cancer colorectal métastatique. Certaines études précliniques ont montré que NOX-A12 était capable de rendre la zone entourant la tumeur dans un modèle tumoral, appelée microenvironnement tumoral, plus accessible au système immunitaire. Les chercheurs pensent que la capacité de nombreuses tumeurs à utiliser le microenvironnement tumoral pour se cacher du système immunitaire contribue à l’insensibilité de certaines tumeurs aux inhibiteurs de point de contrôle, tels que le Keytruda®.

Le Centre NCT est un centre leader dans la recherche et le traitement des cancers, situé à Heidelberg en Allemagne. Il a été fondé en 2004 conjointement par le Centre de Recherche sur le Cancer Allemand (DKFZ), l’Hôpital Universitaire de Heidelberg, la Faculté de Médecine de Heidelberg et la Ligue Allemande contre le Cancer (Deutsche Krebshilfe) pour mener des travaux de recherche interdisciplinaire sur la prévention et le traitement du cancer pour le bénéfice des patients.

Les investigateurs principaux de l’étude clinique menée dans le Centre NCT sont le Prof. Dirk Jäger, Directeur du Centre NCT et Chef des groupes de recherche clinique et sur l’immunologie des tumeurs, ainsi que le Dr. Niels Halama, Responsable du Groupe. Tous deux sont des leaders reconnus dans la recherche clinique sur le cancer, possédant en outre une expérience significative dans l’étude du microenvironnement tumoral dans un contexte clinique. Au cours de sa carrière, le Prof. Jäger s’est spécialisé en immunologie et sur l’étude des tumeurs, ainsi que sur les connexions interdisciplinaires entre ces deux domaines, du point de vue scientifique et clinique.

Le Dr. Jarl Ulf Jungnelius, Directeur médical chez NOXXON, déclare : « Le Dr. Jäger et le Dr. Halama sont des spécialistes du traitement des cancers métastatiques, ainsi que du microenvironnement tumoral. Nous sommes très heureux de pouvoir collaborer avec eux sur cette étude sans précédent. »

Le Prof. Jäger, Directeur du Centre NCT de Heidelberg, commente : « Cette étude nous permettra d’explorer les bénéfices potentiels de NOX-A12 associé au Keytruda® chez des patients pour lesquels il existe peu d’options thérapeutiques viables. L’étude nous aidera aussi à mieux comprendre la capacité de NOX-A12 à modifier le microenvironnement tumoral et à le rendre plus accessible au système immunitaire afin de faciliter la destruction de la tumeur. »
Pour plus d’informations, merci de contacter :

NOXXON Pharma N.V.
Aram Mangasarian, Ph.D., Président Directeur Général
Tél. +49 (0) 30 726 2470
amangasarian@noxxon.com

NewCap
Florent Alba
Tél. +33 (0) 14 471 98 55
falba@newcap.fr
À propos de NOXXON
NOXXON Pharma N.V. est une société biopharmaceutique développant principalement des traitements contre le cancer. L’objectif de NOXXON est d’améliorer significativement l’efficacité des traitements anticancéreux, notamment les approches immuno-oncologiques (inhibiteurs de point de contrôle immunitaire) et les traitements actuels plus courants (chimiothérapie et radiothérapie). La plateforme de Spiegelmers de NOXXON a permis le développement d’un portefeuille exclusif de produits candidats au stade clinique, dont son candidat médicament anticancéreux phare, NOX-A12 qui est le sujet d’un collaboration en immuno-oncologie avec Merck & Co. Inc / MSD (NYSE:MRK) pour réaliser une étude clinique sur NOX-A12 associé au Keytruda® (pembrolizumab) dans le cancer du pancréas et le cancer colorectal. NOXXON est soutenu par des investisseurs internationaux de renom, dont TVM Capital, Sofinnova Partners, Edmond de Rothschild Investment Partners, DEWB, NGN et Seventure. Son siège social se situe à Amsterdam, aux Pays-Bas et ses bureaux à Berlin, en Allemagne. De plus amples informations peuvent être consultées sur www.noxxon.com
À propos du Centre National des Pathologies Tumorales (NCT) de Heidelberg
Le Centre National des Pathologies Tumorales (NCT) de Heidelberg est une institution mixte associant le Centre de Recherche sur le Cancer allemand (DKFZ), l’Hôpital Universitaire de Heidelberg et la Ligue allemande contre le Cancer (Deutsche Krebshilfe). L’objectif du NCT est d’intégrer des approches prometteuses issues de la recherche sur le cancer à tous les stades de la prise en charge des patients (diagnostic, traitement, suivi et prévention). La clinique pluridisciplinaire d’oncologie ambulatoire constitue l’élément central du NCT. Ici, les patients bénéficient d’un plan de traitement personnalisé, élaboré selon un calendrier optimisé dans le cadre de réunions de concertation pluridisciplinaires (RCP). La participation à des études cliniques permet d’accéder à des thérapies innovantes. Le NCT est une plateforme pionnière qui traduit dans la pratique clinique les résultats de la recherche effectuée en laboratoire. Le NCT coopère avec des groupes d’entraide, et les assiste dans leur travail. Le NCT travaille, depuis 2015, à la mise en place d’un deuxième site à Dresde outre celui de Heidelberg.
Déclarations prospectives
Ce communiqué contient des déclarations prospectives ou des termes se rapportant aux développements futurs ou futurs, ainsi que les négations de telles formulations ou termes, ou une terminologie similaire. Ces déclarations ne constituent pas des faits historiques. Ces déclarations comprennent des projections et des estimations ainsi que les hypothèses sur lesquelles celles-ci reposent, des déclarations portant sur des projets, des objectifs, des intentions et des attentes concernant des résultats financiers, des événements, des opérations, des services futurs, le développement de produits et leur potentiel ou les performances futures. La société ne prend aucun engagement de mettre à jour les informations et déclarations prospectives, qui ne représente que l’état des choses le jour de la publication.

12/05/2017

Industry Veteran Joins Company to Accelerate Commercialization and Growth
FREMONT, Calif. –May 10, 2017–Shockwave Medical, a pioneer in the treatment of calcified cardiovascular disease, today announced the appointment of Doug Godshall as president and CEO.
Godshall was most recently the CEO and a director of HeartWare International, Inc. (NASDAQ:HTWR), a leader in the left ventricular assist device (LVAD) market where he served for 10 years until the company was acquired by Medtronic in August of 2016 for $1.1 billion. Prior to HeartWare, Godshall held various executive and leadership positions at Boston Scientific including president of the Vascular Surgery Division and a member of Boston Scientific’s Operating Committee. He also served as vice president, Business Development, where he was responsible for more than 70 transactions, including many in the arenas of coronary and peripheral interventions.
“Shockwave represents a rather remarkable combination: the company’s Lithoplasty system is addressing significant unmet clinical needs using a truly novel approach to the problem; there is a near term, substantial commercial opportunity; and the company has a product pipeline that can carry it for years into the future,” said Mr. Godshall. “From the time I first learned of Shockwave, I have heard remarkably consistent feedback about the profound difference this system makes on some of the most challenging interventional procedures. The team has done a superb job bringing the technology from concept through clinical validation, and I am honored to be given this opportunity to take the reins from one of the company’s founders, Daniel Hawkins, who has worked tirelessly to make this vision a reality.”
“Doug brings a proven track record and a wealth of directly relevant experience to the company,” said Daniel Hawkins, co-founder of Shockwave Medical. “He shares our passion for Lithoplasty technology and its potential role in the treatment of patients across a wide range of advanced cardiovascular disease. We are thrilled to have him leading the company through the next phases of growth and expansion.”
“We are excited to welcome an executive of Doug’s caliber and experience to the Shockwave team at this important time in the company’s development,” added Jay Watkins, the company’s chairman of the Board. “His unique combination of skills gives us a rare opportunity to accelerate commercialization simultaneously in both coronary and peripheral applications, while continuing to advance our valve program. All of us in the organization are extremely grateful to Daniel Hawkins for showing the vision and leadership needed to move this technology successfully from the bench to the patient over these past seven years.”
About Shockwave Medical’s Lithoplasty® System The Shockwave Medical Lithoplasty System integrates angioplasty balloon catheter devices with the calcium-disrupting power of sonic pressure waves, known as lithotripsy. Each Lithoplasty catheter incorporates multiple lithotripsy emitters activated with the touch of a button after the integrated balloon is inflated. Once activated, these emitters produce therapeutic sonic pressure waves that are inherently tissue-selective, passing through the balloon and soft vascular tissue, preferentially disrupting the calcified plaque inside the vessel wall by creating a series of micro-fractures. When the calcium has been modified, the vessel can be dilated using low pressures, thereby enabling even historically challenging patients to be treated effectively with minimal injury to the vessel.
To view an animation of the Lithoplasty System visit http://shockwavemedical.com.
The Shockwave Medical Peripheral Lithoplasty System is commercially available in Europe and the United States and is intended for lithotripsy-enhanced balloon dilatation of lesions, including calcified lesions, in the peripheral vasculature, including the iliac, femoral, ilio-femoral, popliteal, infra-popliteal, and renal arteries. Not for use in the coronary or cerebral vasculature.

About Shockwave Medical
Shockwave Medical, based in Fremont, Calif., is working to reshape interventional therapy with Lithoplasty® Technology for the treatment of calcified peripheral vascular, coronary vascular and heart valve disease. For more information, visit www.shockwavemedical.com.

09/05/2017

Dublin – Irlande, le 9 mai 2017 – Mainstay Medical International plc (« Mainstay » ou la « Société » ; Euronext Paris : MSTY.PA et l’ESM de la Bourse irlandaise : MSTY.IE), une société de dispositifs médicaux dédiée à la commercialisation de ReActiv8®, un dispositif de neurostimulation implantable destiné à traiter la lombalgie chronique invalidante, annonce la première vente ainsi que la première pose de ReActiv8® en Irlande.
La pose a été réalisée par les docteurs Josh Keaveny et Alexander Moudrakovski, médecins anesthésistes spécialistes de la prise en charge de la douleur, à l’hôpital St Joseph, établissement faisant partie du Beaumont Hospital Group, à Dublin.
Dr. Keaveny déclare : « ReActiv8® représente une nouvelle approche dans la prise en charge de la lombalgie chronique invalidante en ciblant la cause sous-jacente de cette pathologie. Nous disposons désormais d’une nouvelle option thérapeutique pour traiter les patients qui souffrent de douleurs dorsales invalidantes depuis des années, qui ne sont pas candidats à une chirurgie de la colonne vertébrale et qui ont fait l’expérience de nombreuses autres thérapies conventionnelles, sans aboutir à un soulagement suffisant ».
ReActiv8® agit par stimulation électrique des nerfs responsables de la contraction des muscles stabilisateurs de la colonne vertébrale. Il a été démontré que l’activation de ces muscles pour restaurer la stabilité fonctionnelle du rachis facilite la guérison des patients souffrant de lombalgie chronique invalidante.
Peter Crosby, CEO de Mainstay, commente : « Nous construisons un réseau de sites de référence en Europe, l’une des premières étapes de notre stratégie de commercialisation, afin de promouvoir ReActiv8® et soutenir l’élargissement du marché. Naturellement, l’Irlande étant notre marché domestique, nous nous réjouissons du partenariat noué avec les docteurs Josh Keaveny et Alexander Moudrakovski visant à établir l’ancrage de Mainstay sur le deuxième marché européen de la Société, après le début de la commercialisation en Allemagne ».
Mainstay a obtenu le marquage CE pour ReActiv8® sur la base des résultats positifs de l’essai clinique ReActiv8-A, qui a mis en évidence des améliorations cliniquement importantes, statistiquement significatives et durables de la douleur, de la capacité fonctionnelle et de la qualité de vie des personnes atteintes de lombalgie chronique invalidante, et disposant d’options thérapeutiques limitées.

09/05/2017

• Trial outcomes provide further evidence for safety of Keyzilen® in chronic intermittent use
• Exploratory efficacy results suggest potential benefits of repeating treatment cycles
Zug, Switzerland, May 9, 2017 – Auris Medical Holding AG (NASDAQ: EARS), a clinical-stage company dedicated to developing therapeutics that address important unmet medical needs in otolaryngology, today announced key results from AMPACT1 (AM-101 in the Post-Acute Treatment of Peripheral Tinnitus 1), the open-label extension study of the Phase 3 TACTT2 clinical trial. The study provides additional confirmation on the long-term safety of Keyzilen® and suggests the potential for further therapeutic benefits from repeated treatment.
« We are very pleased that the AMPACT1 trial showed good tolerance and safety for Keyzilen® with chronic intermittent use and thus confirms the positive results from the AMPACT2 trial, » commented Thomas Meyer, Auris Medical’s founder, Chairman and Chief Executive Officer. « In addition, we are glad to see from exploratory efficacy data that the TACTT2 patients who continued into AMPACT1 on average were able to reduce the burden and severity of their tinnitus with repeated treatment. »
AMPACT1 is the second of two open-label extension studies with Keyzilen® that were conducted in response to a request from the US Food and Drug Administration (FDA) for safety data from chronic intermittent use of Keyzilen®for up to 12 months. Participants who completed the TACTT2 trial were offered the option to roll over into AMPACT1, while still blinded to the treatment allocation. Patients were given the choice to receive up to three treatment cycles with each cycle comprising three intratympanic administrations of Keyzilen®, followed by a treatment-free observation period of 12 weeks.
TACTT2 and AMPACT1 were primarily conducted in North America. Patients enrolled in TACTT2 within three months from tinnitus onset, i.e. during the acute stage. Of the 316 patients who completed the TACTT2 trial, 257 patients rolled over into AMPACT1 and provided safety data; 228 of these patients provided exploratory efficacy data. At the time of enrollment into AMPACT1, all patients were in the post-acute stage.
The primary safety endpoint of AMPACT1 was the incidence of clinically relevant hearing deterioration five weeks after the start of a treatment cycle. In line with the results from previous trials with Keyzilen®, such incidence was low (6%), with no signs of treatment-related effects. Over the course of AMPACT1, the hearing threshold at the average of 4, 6 and 8 kHz showed only little change. The vast majority of adverse events that were considered related to the study drug or treatment procedure were rated as either mild or moderate in intensity. Three patients experienced a total of four non-fatal, serious adverse events, none of which was considered related to the study drug. Confirming previous data, about 93% of tympanic membranes were already closed at the time of the first follow-up visit.
Exploratory efficacy analyses of AMPACT1 show improvements in the Tinnitus Functional Index (TFI) as well as other tinnitus metrics. The TFI decreased on average by 8.2 points (95% confidence interval 6.2 to 10.1; baseline of 42.7 points) to the last follow-up visit. The more treatment cycles the patients received, the larger the reduction in the TFI was; the difference between three cycles and one cycle reached statistical significance. Similar results were achieved on subjective tinnitus loudness and tinnitus annoyance. In addition, 41% of AMPACT1 participants achieved a reduction in their tinnitus severity (extreme-severe-moderate-mild-none) by at least one grade and 28% reported that their tinnitus severity had improved « much » or « very much » compared to baseline.
Auris Medical expects to announce results from TACTT3, the European placebo-controlled sister trial to TACTT2, in early 2018. The trial has been extended to recruit an additional 60 patients in each of the acute and post-acute strata (i.e. up to three months and between three and six months from onset), and enrollment is ongoing.

About Acute Inner Ear Tinnitus
Tinnitus is the perception of sound without external acoustic stimulation. Tinnitus of the inner ear may be caused by various injuries to the cochlea, the organ of hearing, such as overexposure to noise. Tinnitus that has been present for less than three months is considered acute, while tinnitus that has been present for over three months is considered chronic. Tinnitus of the inner ear often has a serious impact on ability to sleep, relax or concentrate, which may lead to tiredness, irritation, anxiety or depression. There is no universal standard of care for tinnitus of the inner ear and efficacy of a pharmacological treatment for tinnitus of the inner ear has not yet been conclusively demonstrated.

About Keyzilen® (AM-101)
Keyzilen® is a small molecule N-methyl-D-aspartate (NMDA) receptor antagonist formulated in a biocompatible gel and delivered by intratympanic injection. Keyzilen® is in development for treatment of acute tinnitus of the inner ear. Emerging evidence suggests that NMDA receptors in the cochlea play a major role in the occurrence of tinnitus following acute injury to the inner ear; e.g. from exposure to excessive noise, infections, disturbances in inner ear blood supply, or the administration of certain ototoxic drugs. Persistent overexpression of NMDA receptors may lead to pathologic excitation of auditory nerve fibers, which in the brain is perceived as tinnitus. The development of Keyzilen® is based on research conducted at the INSERM Institute for Neurosciences, and patents have been granted in more than 40 countries worldwide so far.

About Auris Medical
Auris Medical is a Swiss biopharmaceutical company dedicated to developing therapeutics that address important unmet medical needs in otolaryngology. The Company is focused on the Phase 3 development of treatments for acute inner ear hearing loss (AM-111) and for acute inner ear tinnitus (Keyzilen®; AM-101) by way of intratympanic administration with biocompatible gel formulations. In addition, Auris Medical is pursuing intranasal betahistine for Meniere’s disease and vestibular vertigo (AM-125) as well as early-stage research and development projects. The Company was founded in 2003 and is headquartered in Zug, Switzerland. The shares of Auris Medical Holding AGtrade on the NASDAQ Global Market under the symbol « EARS. »

Forward-looking Statements
This press release may contain statements that constitute « forward-looking statements » within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements are statements other than historical fact and may include statements that address future operating, financial or business performance or Auris Medical’s strategies or expectations. In some cases, you can identify these statements by forward-looking words such as « may, » « might, » « will, » « should, » « expects, » « plans, » « anticipates, » « believes, » « estimates, » « predicts, » « projects, » « potential, » « outlook » or « continue, » and other comparable terminology. Forward-looking statements are based on management’s current expectations and beliefs and involve significant risks and uncertainties that could cause actual results, developments and business decisions to differ materially from those contemplated by these statements. These risks and uncertainties include, but are not limited to, the timing and conduct of clinical trials of Auris Medical’s product candidates, including the likelihood that the TACTT3 clinical trial with Keyzilen® will not meet its endpoints, the acceptability of the data from AMPACT1 and AMPACT2 in support of a potential new drug application to the FDA and other regulators, the clinical utility of Auris Medical’s product candidates, the timing or likelihood of regulatory filings and approvals, Auris Medical’sintellectual property position and Auris Medical’s financial position, including the impact of any future acquisitions, dispositions, partnerships, license transactions or changes to Auris Medical’s capital structure, including future securities offerings; the use of proceeds from Auris Medical’s recent equity offering and the ability of Auris Medical to finance its operations in the future. These risks and uncertainties also include, but are not limited to, those described under the caption « Risk Factors » in Auris Medical’s Annual Report on Form 20-F and future filings with the Securities and Exchange Commission. Forward-looking statements speak only as of the date they are made, and Auris Medical does not undertake any obligation to update them in light of new information, future developments or otherwise, except as may be required under applicable law. All forward-looking statements are qualified in their entirety by this cautionary statement.

Company contact: Cindy McGee, Head of Investor Relations and Corporate Communications, +41 61 201 1350, investors@aurismedical.com
Media contact: David Schull, Russo Partners, 1-858-717-2310,
david.schull@russopartnersllc.com
Auris Medical AG

09/05/2017

HAYWARD, CA (May 08, 2017) – RefleXion Medical, a medical equipment company developing the first biology-guided radiotherapy (BgRT) system for targeted, personalized radiotherapy, announced today that industry veteran Todd Powell has been appointed President, CEO and a Board of Directors member. Powell spent the past 29 years developing innovative products while leading global organizations in cancer care. During a distinguished 11 year career at Elekta AB, a world leader in radiation oncology solutions, Powell held senior executive roles of increasing responsibility. Most recently, he served as Executive Vice President of Comprehensive Oncology Solutions where he led a business unit with more than $1B in revenue and 1,200 employees across North America, Europe and Asia.
From 1992 through 2005, Powell worked at IMPAC Medical Systems, a leader in information systems for radiation and medical oncology. In 1997 he became VP of Product Engineering & Development and was involved with the company’s successful public offering and subsequent acquisition by Elekta in 2005. Powell began his career as an Oncology Marketing and Business Specialist at Varian Medical Systems.
He completed Executive Innovation and Leadership Programs at Stanford Graduate School of Business in 2010, and earned a Bachelor of Physics and Mathematics from California State University-Chico in 1990.

« I’m excited to join the tremendous innovators at RefleXion Medical, » said Powell. « Radiation therapy is a cost-effective pillar of cancer treatment, and RefleXion is developing the most profound advance in radiotherapy I’ve seen in decades. By leveraging biological signals from tumors during treatment, this company has the potential to significantly improve cancer care for millions of patients across the world. »
« Todd has a sterling record of achievement in our industry, » said Samuel Mazin, Ph.D., Founder and Chief Technology Officer of RefleXion. « He shares our passion for dramatically transforming and improving how cancer patients are treated with radiotherapy. He also knows how to grow a successful global brand and business. We feel fortunate to have him heading our fast-growing team. »

About RefleXion Medical
RefleXion Medical is a privately held medical device company developing the first biology-guided radiotherapy (BgRT) system for cancer treatment. By leveraging Positron Emission Tomography (PET) in a novel way, RefleXion’s patented technology causes tumors to continuously signal their location during treatment, potentially revolutionizing treatment of metastatic disease. RefleXion is backed by premier investment firms Sofinnova Partners, KCK Group, Pfizer Venture Investments, Venrock and Johnson & Johnson Innovation – JJDC, Inc. The company has also received grant funding from the National Cancer Institute (NCI) Small Business Innovation Research (SBIR) Program. For more information, visit www.reflexionmedical.com and follow @reflexionmed on Twitter.