What's new?

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

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

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

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

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

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

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

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


Paris, France – May 22, 2017 — Sofinnova Partners, a leading European venture capital firm specialized in Life Sciences, has appointed Graziano Seghezzi as Managing Partner. He joins Antoine Papiernik, Denis Lucquin, and Monique Saulnier in the company’s Managing Partnership. Graziano’s appointment boosts Sofinnova Partners’ international leadership.

Graziano began his career in venture capital in 2001 at Sofinnova Partners where he was in charge of identifying and assessing investment opportunities. Throughout his career, he has focused on company creation through the establishment of start ups, spin-offs, and accelerators. He was the seed investor of Omthera Pharmaceuticals, listed on Nasdaq and then sold to Astra Zeneca, of Glycovaxyn sold to GSK and of Creabilis sold to Sienna Biopharmaceuticals. He also invested and is on the Board of Breath Therapeutics, Corvidia Therapeutics, Crescendo Biologics, Hookipa Biotech, Inotrem and Mission Therapeutics. Graziano also co-founded BiovelocITA, Italy’s first biotech accelerator. Between 2003 and 2006, he worked at Index Ventures in Geneva.

A scientist by training, Graziano spent five years working in academic research at NYU School of Medicine (USA), studying oncology and cardiovascular diseases. He holds a degree in genetics and microbiology from the University of Pavia (Italy) and an MBA from RSM-Erasmus University (Netherlands).

Graziano Seghezzi says: « I have tremendous respect for the Sofinnova team both on a professional and on a personal level; I am therefore particularly happy to be promoted Managing Partner. This new role comes at a pivotal moment for the partnership which has devised an ambitious growth strategy. With my extensive international expertise, I am looking forward to actively contribute to further develop Sofinnova Partners’ global leadership in Life Sciences ».


Antoine Papiernik, Chairman of Sofinnova Partners adds: « Graziano came to Sofinnova Partners fifteen years ago, as he was changing careers from academic researcher to biotech investor. Today, he has built a true investment track record both in Europe and in the United States; we are very pleased to welcome him to the Managing Partnership ».


Key updates
• QR-010 Phase 1b clinical trial on track to present top-line data in cystic fibrosis (CF) patients in mid-2017.
• Investigational new drug (IND) application for QR-110, ProQR’s lead program in ophthalmology, was cleared by the FDA to start a clinical trial in both adult and pediatric LCA 10 patients.
• David M. Rodman, MD was appointed as Chief Development Strategy Officer of ProQR, and will lead the translational development effort to rapidly advance the pipeline programs into the clinic.
• ProQR was granted two key patents, protecting QR-010 for cystic fibrosis in the US and EU.
• Pre-clinical data was presented for three programs in the ophthalmology pipeline targeting LCA 10 and Usher syndrome at the ARVO annual meeting in May 2017

LEIDEN, the Netherlands, May 17, 2017 – ProQR Therapeutics N.V. (Nasdaq:PRQR), today announced results for the first quarter of 2017.
“As we announced earlier this week, we are all devastated by the unexpected passing of our co-founder and vice-chairman of the supervisory board, Henri A. Termeer,” said Daniel de Boer, Chief Executive Officer of ProQR. “Henri was a great mentor, a passionate patient advocate and a key factor in establishing ProQR. His passion to do the right thing in the interest of patients is unparalleled. We are honored to have had the opportunity to work with him so closely and learn from his wealth of experience. He was a true inspiration for all of us, and we will continue to build on the path he helped us to set out. Although he will be deeply missed, our supervisory board, with co-founder and chairman Dinko Valerio, James Shannon, Alison Lawton and Antoine Papiernik, continues to be very strong with broad experience in all aspects of running a biotechnology business and we are extremely motivated to continue to build on the path he helped us set.”
“With that in mind, this is an important phase for the Company in our goal to translate our rich pipeline into a diversified portfolio of development programs. We are very pleased with the clearance of the IND for QR-110, enabling us to now advance our second molecule into clinical development aiming to make a meaningful difference for LCA 10 patients. We are also excited to add Dave Rodman to our team who will further strengthen our development team, with the goal of efficiently and rapidly driving our programs through to patients. Looking forward to the upcoming summer, we are very excited for the expected completion of the Phase 1b trial in our lead program QR-010. We have been treating >64 CF patients with our lead compound QR-010 and are excited to get to the data and progress the compound into next trials.”

Corporate Highlights
• In March, the Company announced that it appointed David M. Rodman, MD as Chief Development Strategy Officer. Dr. Rodman has had a long career in drug development including leadership roles in translational medicine, rare disease drug development, and RNA therapeutics. Dr. Rodman’s experience includes a leadership role in developing two approved medicines for CF at Vertex Pharmaceuticals, as Vice President and head of respiratory drug development. He was also the head of translational medicine at Novartis Institute for Biomedical Research. More recently, he was the Chief Medical Officer at MiRagen and Nivalis. Expansion of the ProQR management team will allow the Company to further realize the potential of RNA therapeutics as well as expand business capabilities needed to advance the development of our product candidates.
Subsequent events
• In April, the Company announced that with the clearance of the investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA), ProQR can now start clinical development of QR-110 in Leber’s congenital amaurosis Type 10 (LCA 10) patients. The trial, named 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 and the second eye will serve as a control. 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 objective will be to assess safety and tolerability of QR-110. Secondary objectives are to evaluate pharmacokinetics and efficacy, which is measured by specialized ophthalmic tests including visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR) and a mobility course. Fixation stability and 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.
• In April, the Company announced the grant of two key patents protecting QR-010 in the US and EU. These patents provide the Company exclusive rights for QR-010 for the treatment of CF until at least July 2033. US patent no. 9,605,255 is directed to methods of targeting RNA for the most common mutation in CF, called F508del, using oligonucleotides to restore the function of the CFTR protein. Last year, ProQR also received the grant of the equivalent European patent (EP 2 852 668 B1). Apart from these ProQR owned patents, ProQR has an exclusive license to US patent no. 9,617,535 from Massachusetts General Hospital covering QR-010.
• During the 2017 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) held on May 7 – 11, 2017 in Baltimore, MD, USA, the Company presented three abstracts, including additional positive pre-clinical proof-of-concept data for QR-110 in LCA 10 and pre-clinical data for two programs, QRX-411 and QRX-421, each targeting specific mutations that result in Usher syndrome.
• The Company announced that it will host an R&D Day on June 15th in New York where Company executives and external experts will present ProQR’s pipeline of development and early stage programs in detail.

Financial Highlights
At March 31, 2017, ProQR held cash and cash equivalents of €52.1 million, compared to €59.2 million at December 31, 2016. Net cash used in operating activities during the three month period ended March 31, 2017 was €8.8 million, compared to €7.8 million for the same period last year.
Research and development costs increased to €8.0 million for the quarter ended March 31, 2017 from €6.9 million for the same period last year and comprised of allocated employee costs including share-based payments, the costs of materials and laboratory consumables, outsourced activities, license and intellectual property costs and other allocated costs. The increase in expenses was primarily due to the advancement of our pipeline, which included clinical development of QR-010 and QR-110, pre-clinical development of QR-313. The remainder represents increased investments in our other pipeline programs.
General and administrative costs decreased to €2.3 million for the quarter ended March 31, 2017 from €2.6 million for the same period last year.
Net result for the three month period ended March 31, 2017 was a €10.5 million loss or €0.45 per share, compared to a €10.2 million loss or €0.44 per share for the same period last year. For further financial information for the period ending March 31, 2017, please refer to the financial statements appearing at the end of this release.

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-010
QR-010 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the F508del mutation. The F508del mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is designed to bind to the defective CFTR mRNA and to restore CFTR function. QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a small, handheld aerosol delivery device which nebulizes QR-010 into a mist inhaled directly into the lungs. QR-010 has been granted orphan drug designation in the United States and the European Union and fast-track status by the FDA. The QR-010 project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.

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 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 truncated but functional C7 protein and thereby restores functionality of the anchoring fibrils.

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-010, QR-110 and QR-313, and the clinical development and the therapeutic potential thereof, statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in our innovation pipeline, statements regarding release of clinical data, and statements regarding our patent estate. 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, 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.



Berlin, Germany, May 16, 2017 – NOXXON Pharma N.V. (Alternext Paris: ALNOX), a biotechnology company whose core focus is on improving cancer treatment by targeting the tumor microenvironment, today announced the signing of an agreement with the National Center for Tumor Diseases (NCT) in Heidelberg under which the NCT will conduct a trial evaluating NOXXON’s lead product candidate NOX-A12 in combination with Keytruda® (pembrolizumab) in metastatic pancreatic and colorectal cancer. In some preclinical studies, NOX-A12 has shown an ability to make the immediate area surrounding a model tumor, the socalled tumor microenvironment, more accessible to the immune system. The ability of many tumors to use the tumor microenvironment to hide from the immune system is believed to contribute to the insensitivity of some tumors towards checkpoint inhibitors, such as Keytruda®.
The NCT is a leading center for cancer research and treatment, located in Heidelberg, Germany. It was jointly founded by the German Cancer Research Center (DKFZ), Heidelberg University Hospital, Medical Faculty Heidelberg and German Cancer Aid (Deutsche Krebshilfe) in 2004 to conduct interdisciplinary research for preventing and treating cancer to ultimately benefit patients.

The NCT investigators leading the clinical trial include Prof. Dr. Dirk Jäger, Managing Director, head of the clinical and tumor immunology research groups, and Dr. Niels Halama, Group Leader, both recognized leaders in clinical cancer research with significant experience in studying the tumor microenvironment in a clinical setting. Throughout his career, Prof. Dr. Jäger has focused on tumor and immunology as well as the interdisciplinary connections between both fields, both scientifically and clinically.

NOXXON’s Chief Medical Officer, Dr. Jarl Ulf Jungnelius, commented: “Dr. Jäger and Dr. Halama are experts in the treatment of metastatic cancer patients as well as the tumor microenvironment. We are extremely pleased that they will be collaborating with NOXXON on this groundbreaking study.”

Prof. Dr. Jäger, Managing Director of the NCT Heidelberg, commented: “This trial will enable us to explore the potential of NOX-A12 in combination with Keytruda® to benefit patients with few viable treatment options. Importantly, the trial will help us to better understand the ability of NOX-A12 to modify the tumor microenvironment and make it more accessible to the immune system to facilitate tumor destruction.”
For more information, please contact:
NOXXON Pharma N.V.
Aram Mangasarian, Ph.D., Chief Executive Officer
Tel. +49 (0) 30 726 2470
Florent Alba
Tel. +33 (0) 1 44 71 98 55

NOXXON Pharma N.V. is a clinical-stage biopharmaceutical company focused on cancer treatment. NOXXON’s goal is to significantly enhance the effectiveness of cancer treatments including immuno-oncology approaches (such as immune checkpoint inhibitors) and current standards of care (such as chemotherapy and radiotherapy). NOXXON’s Spiegelmer® platform has generated a proprietary pipeline of clinical-stage product candidates including its lead cancer drug candidate NOX-A12, which is the subject of a clinical immuno-oncology collaboration agreement with Merck & Co. / MSD (NYSE: MRK) to study NOX-A12 combined with Keytruda® (pembrolizumab) in pancreatic and colorectal cancer. NOXXON is supported by a strong group of leading international investors, including TVM Capital, Sofinnova Partners, Edmond de Rothschild Investment Partners, DEWB, NGN and Seventure. NOXXON has its statutory seat in Amsterdam, The Netherlands and its office in Berlin, Germany. Further information can be found at: www.noxxon.com

About the National Center for Tumor Diseases (NCT) Heidelberg
The NCT Heidelberg is a joint institution of the German Cancer Research Center, Heidelberg University Hospital and German Cancer Aid. The NCT’s goal is to link promising approaches from cancer research with patient care from diagnosis to treatment, aftercare and prevention.
The interdisciplinary tumor outpatient clinic is the central element of the NCT. Here the patients benefit from an individual treatment plan prepared in a timely manner in interdisciplinary expert rounds, the so-called tumor boards. Participation in clinical studies provides access to innovative therapies. The NCT thereby acts as a pioneering platform that translates novel research results from the laboratory into clinical practice. The NCT cooperates with self-help groups and supports them in their work. Since 2015, a second site for the NCT beside Heidelberg has been under development in Dresden.

Certain statements in this communication contain formulations or terms referring to the future or future developments, as well as negations of such formulations or terms, or similar terminology. These are described as forward-looking statements. In addition, all information in this communication regarding planned or future results of business segments, financial indicators, developments of the financial situation or other financial or statistical data contains such forward-looking statements. The company cautions prospective investors not to rely on such forward-looking statements as certain prognoses of actual future events and developments. The company is neither responsible nor liable for updating such information, which only represents the state of affairs on the day of publication.


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.


Dublin – Ireland, 9 May 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 (“CLBP”), announces the first sale and implant of ReActiv8 in Ireland.
The ReActiv8 implant was performed at St. Joseph’s Hospital, part of the Beaumont Hospital Group, in Dublin, by Dr. Josh Keaveny and Dr. Alexander Moudrakovski, Consultants in Anaesthesia and Pain Medicine.
Dr. Keaveny commented: “ReActiv8 represents a novel approach to Chronic Low Back Pain which addresses the underlying cause of the condition. We now have a new option for treating patients who have suffered from debilitating back pain for years who are not candidates for spine surgery and have attempted many other conventional therapies without adequate relief.”
ReActiv8 works by electrically stimulating the nerves responsible for contracting the key stabilizing muscles of the lumbar spine. Activation of these muscles to restore functional spine stability has been shown to facilitate recovery from Chronic Low Back Pain.
Peter Crosby, CEO of Mainstay, commented: “As part of our early commercialization strategy, we are building a network of reference sites in Europe, who can champion ReActiv8 and help expand the market. As an Irish company, Ireland is our home market, and we are pleased to partner with Dr. Keaveny and Dr. Moudrakovski to establish a foothold in our second European market following the start of commercialization in Germany.”
Mainstay received CE Marking for ReActiv8 supported by positive results from the ReActiv8-A Clinical Trial that demonstrated a clinically important, statistically significant and lasting improvement in pain, disability and quality of life in people with disabling CLBP and few other treatment options.


• 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,
Auris Medical AG


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.


• Vaccine was safe and elicited a potent immune response
• Cytomegalovirus (CMV) infection is the leading cause of birth defects in the developed world, occurring in 1 – 2.5 % of all newborns and conferring risk of deafness and impaired intellectual development
Vienna, Austria, 4 May 2017 – Hookipa Biotech AG, a company pioneering a new class of immunotherapies for oncology and infectious diseases, today presented the un-blinded safety and immunogenicity data through month four from the Company’s phase 1 first-in-human trial of HB-101, a vaccine against human cytomegalovirus (CMV), based on Hookipa’s proprietary Vaxwave® platform. The data was presented at the CMV 2017 Conference (www.cmv2017.nl/home) in Leeuwenhorst, The Netherlands. Further follow-up safety and immunogenicity results through month 12 of the study are expected in November 2017.

Joern Aldag, Hookipa’s CEO, said; “These clinical data show that Hookipa’s Vaxwave® technology and, specifically, the bivalent CMV vaccine candidate HB-101 are safe and immunogenic. We are pleased to note that the vaccine elicited potently CMV-neutralizing antibodies and high frequencies of CMV-specific CD8+ T cells with as few as two doses of the vaccine. These results provide confidence that Hookipa can establish HB-101 as a best-in class CMV development program. Our team around Dr. Anders Lilja did an amazing job running this program and we are actively gearing up for Phase 2 efforts.”

HB-101 is a bivalent CMV vaccine candidate based on Vaxwave® vectors expressing two human CMV antigens, the tegument protein pp65 and a truncated isoform of the fusion protein gB. The safety and immunogenicity of HB-101 were evaluated in a randomized, placebo-controlled, double-blind phase I dose-escalating trial (NCT02798692). Three cohorts of 18 subjects per cohort were enrolled. In each cohort, 14 subjects received a high, medium or low dose of the vaccine, respectively, and four received placebo. Vaccine and placebo were administered on day zero, and one and three months after first injection. Safety and reactogenicity data were collected and reviewed by an independent data and safety monitoring board. Immunogenicity readouts included humoral and cellular responses against gB, cellular responses against pp65, as well as humoral and cellular responses against the vector.

Over all three dose groups, 98% of the subjects who received vaccine, and zero percent of the subjects who received placebo, showed detectable CMV-neutralizing antibody titers after three doses (93% of the subjects in the lowest dose group and 100% of the subjects in the medium and high dose groups). In the highest dose group, 100% seroconversion was already observed after the second vaccination and 79% of the subjects who received vaccine in this group, showed titers within the range of seropositive controls. Similarly, all three dose groups of the vaccine induced robust and statistically significant cellular immune responses when compared to placebo. When looking specifically at pp65-specific CD8+ T cells, a key biomarker for cellular immunity against cytomegalovirus, the medium and high dose groups induced clinically and statistically significant responses when compared to placebo.

About Hookipa Biotech
Hookipa Biotech is developing next-generation immunotherapies for infectious diseases and cancer using novel proprietary arenavirus vector platforms. By end April 2017, Hookipa has raised EUR 15 million in non-dilutive funds and EUR 37 million equity investment from internationally renowned venture capital investors including Sofinnova Partners, Forbion Capital Partners, Boehringer Ingelheim Venture Fund, Takeda Ventures and BioMedPartners. Additional information on Hookipa is available at www.hookipabiotech.com.

About Vaxwave®
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) and 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 responses, however they can no longer replicate and are therefore non-pathogenic and inherently safe.
About Cytomegalovirus
Cytomegalovirus (CMV) is a ubiquitous human pathogen that is the leading cause of congenital infection worldwide, occurring in 1 – 2.5 % of all newborns in the developed world. Newborns infected with CMV are at risk of deafness and impaired intellectual development. Cytomegalovirus is also a severe pathogen for transplant recipients causing end organ diseases. The development of CMV vaccines is a widely acknowledged public health priority.

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

At the Company
Marine Popoff
Communications Analyst
Hookipa Biotech AG

Media enquiries
Sue Charles/ Daniel Gooch/ Alex Bannister
Instinctif Partners
+44 (0)20 7866 7905


Toulouse, FRANCE, Ann Arbor, UNITED-STATES, April 18, 2017, 8.00 AM – Cerenis Therapeutics (FR0012616852 – CEREN – PEA PME eligible), an international biopharmaceutical company dedicated to the discovery and development of innovative lipid metabolism therapies (“good cholesterol”) for treating cardiovascular and metabolic diseases, today announces that de Phase 1 clinical trial with CER-209, a P2Y13 receptor agonist, has been initiated.
Dr. Jean-Louis Dasseux, founder and CEO of Cerenis, declares: « According to the American Liver Foundation, NASH is one of the leading causes of cirrhosis in adults in the United-States. 25% of adults having NASH will develop a cirrhosis, and there currently are no approved therapies for these diseases. In addition, epidemiological studies demonstrate that the cardiovascular risk is increased in patients with hepatic steatosis and that the cardiovascular diseases associated are the leading causes of death in patients with liver steatosis1,2.
We believe that CER-209, new patented molecule coming from our research, has the potential to play an innovative part by both addressing hepatic steatosis and atherosclerosis. CER-209’s major asset in NASH and NAFLD treatment lies in its ability to promote HDL recognition and lipid elimination by the liver, through the activation of natural metabolic pathways mediated by the P2Y13 receptor.
The entry of CER-209 into clinical development makes part of Cerenis’ strategy which is based on a rich portfolio of several products at different development stages and operating through diversified mechanism of action, which constitutes as many growth drivers for the company”.
Incidence of NAFLD and NASH is increasing, now becoming common diseases of the liver in part related to the rise in obesity and diabetes rates. NAFLD, a precursor of NASH, is a universal disorder that is now considered as the most common liver disease in the western world, impacting 30% of the world’s population, according to a publication in the World Journal of Hepatology.
Launch of the Phase 1 clinical study with CER-209, within the framework of an IND
The launch follows the FDA IND (Investigational New Drug application) approval, granted in December 2016, to initiate the clinical development of CER-209 in healthy volunteers for the clinical investigation of Non-Alcoholic Fatty Liver Disease (NAFLD) and Non-Alcoholic Steato-Hepatitis (NASH).
1 Franque S. M. et al. Journal of Hepatology, 2016, vol. 65, 425-443
2 World J Gastroenterol 2015 June 14; 21(22): 6820-6834

The objective of this randomized, double blind and placebo controlled trial, is to evaluate efficacy and tolerance and also the pharmacokinetic/pharmacodynamics following the administration of CER-209’ increasing doses in healthy volunteers.
CER-209, an agonist of the P2Y13 receptor, is well suited to the treatment of NAFLD and NASH
CER-209, a selective novel agonist of the P2Y13 receptor decreased both atherosclerosis and liver steatosis in preclinical models. CER-209 caused an increased uptake of high-density lipoprotein-cholesterol (HDL-c) in the liver that is associated with stimulation of bile acid secretion. Repeated dose administration stimulated the apoA-I synthesis and formation of small HDL particles, known to be atheroprotective. CER-209-treated plasma samples had high cholesterol efflux capacity for the mobilization of cellular cholesterol in vitro compared with the placebo group. CER-209 induced a decrease in atherosclerotic plaques in aorta and carotids as well as a remarkable decrease in steatosis in validated preclinical models.
In preclinical models, CER-209 resulted in a marked reduction in overall steatohepatitis as determined by reductions in cholesterol, triglycerides and fatty acids in the liver compared with placebo. Furthermore, CER-209 produced considerable decreases in liver enzymes (ALT and AST) in the plasma. These effects suggest the restoration of liver integrity and indicate a strong potential for CER-209 to treat liver disease such as Non-Alcoholic Steato-Hepatitis (NASH) and Non-Alcoholic Fatty Liver Disease (NAFLD) while reducing the risks associated with cardiovascular disease.

About CER-209
CER-209 is the first drug candidate in the category of oral P2Y13 receptor agonists. The P2Y13 receptor is a member of the P2Y receptor family, a well-known receptor family including the P2Y12 receptor that is the target of successful drugs such as the anti-thrombotic agent Clopidogrel (Plavix®). CER-209 is a specific agonist of the P2Y13 receptor and does not interact with the P2Y12 receptor. In preclinical studies CER-209 promotes HDL recognition by the liver and increases Reverse Lipid Transport (RLT), thereby impacting atherosclerosis regression. Because of the favorable metabolic effects observed in the liver, CER 209 may also offer a new mechanism for the treatment of Non-Alcoholic Fatty Liver Disease (NAFLD) and Non-Alcoholic Steato-Hepatitis (NASH).

About Cerenis: www.cerenis.com
Cerenis Therapeutics is an international biopharmaceutical company dedicated to the discovery and development of innovative HDL and other therapies for the treatment of cardiovascular and metabolic diseases. HDL is the primary mediator of the reverse lipid transport, or RLT, the only natural pathway by which excess cholesterol is removed from arteries and is transported to the liver for elimination from the body.
Cerenis is developing a portfolio of HDL therapies, including HDL mimetics for patients with genetic HDL deficiency, as well as drugs which increase HDL for patients with a low number of HDL particles to treat atherosclerosis and associated metabolic diseases including Non-Alcoholic Fatty Liver Disease (NAFLD) and Non-Alcoholic Steato-Hepatitis (NASH).
Cerenis is well positioned to become one of the leaders in this innovative lipid metabolism therapeutic market, with a broad portfolio of programs in development.

Jean-Louis Dasseux
+33 (0)5 62 24 09 49

Investors relations
Emmanuel Huynh / Louis-Victor Delouvrier
+33 (0)1 44 71 98 53

Media relations
Nicolas Merigeau
+33 (0)1 44 71 94 98


Collaborative team to progress novel USP30 inhibitors to develop potential therapies for Parkinson’s disease
CAMBRIDGE, UK – 11 April 2017 – On World Parkinson’s day, Mission Therapeutics, a drug discovery and development company focused on selectively targeting deubiquitylating enzymes (DUBs) to treat neurodegenerative diseases, cancer and other diseases with high unmet medical need, announced that it has been awarded a grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF).
This research grant will support the testing of Mission Therapeutics’ potent and selective USP30-targeted inhibitors in translationally relevant stem cell-derived Parkinson’s disease models developed by Professor Richard Wade-Martins and his research group at the University of Oxford.
USP30, a mitochondrial associated DUB, has been implicated in the control of mitophagy – a process where dysfunctional mitochondria are selectively cleared from the cell. Failure of mitochondrial quality control may lead to degeneration of the highly active substantia nigra neurons in the brain, a pathological mechanism which results in Parkinson’s disease.
The inhibition of USP30 is being studied by Mission Therapeutics to see if this promotes mitophagy and thus improves cellular resilience in this and other neurodegenerative diseases. The objective of the research collaboration with Professor Richard Wade-Martins is to test Mission’s potent and selective USP30 inhibitors in a range of disease models – induced Pluripotent Stem Cells (iPSC)-derived from patients with sporadic and familial Parkinson’s disease.
Shalini Padmanabhan, PhD, Associate Director of Research Programs at MJFF:
“USP30 is one of the more promising DUBs associated with mitophagy, in terms of published data and feasibility of compound development. We hope that this collaboration between Mission Therapeutics and Oxford Parkinson’s Disease Centre will promote our understanding of the mechanisms and consequences of USP30 inhibition in Parkinson’s disease.”
Dr Michael Koslowski, Executive Vice President, Research and Development and Chief Medical Officer of Mission Therapeutics, added:
“Receiving funding from the Michael J. Fox Foundation is a great accolade, recognizing the quality of the research being done by Mission Therapeutics and Prof. Wade-Martins and his group. The collaborative study will provide key data that will guide the clinical development strategy of our USP30 inhibitor programme. We are working hard to find new ways in which to tackle this difficult disease, which is especially highlighted during this World Parkinson Awareness week, for patients and their families.”

Mission Therapeutics Ltd
Anker Lundemose MD PhD
Chief Executive Officer
Tel: +44 (0) 1223 497199
Instinctif Partners
Melanie Toyne-Sewell / Eileen Paul / Priya Kalia
Tel: +44 (0) 20 7457 2020
Westwicke Partners (U.S.)
Chris Brinzey
Tel: +1 339-970-2843
About Mission Therapeutics
Mission Therapeutics, an early-stage drug development company targeting the ubiquitin pathway for the treatment of cancer, neurodegenerative, and other diseases of unmet need. The Company has built a leading platform for the discovery and development of first-in-class, small-molecule drugs that selectively target deubiquitylating enzymes (DUBs) – an emerging drug class that is attracting significant commercial interest in the area of protein homeostasis.
Mission has strong links with key academic and research centers, including Cancer Research UK Laboratories and the University of Cambridge’s Jackson Laboratories at the Gurdon Institute, and a leadership team that has broad international, commercial and scientific experience.
In February 2016, the Company completed an $86m financing that was led by Imperial Innovations and Woodford Patient Capital Trust and included participation from existing investors Sofinnova Partners, Roche Venture Fund, Pfizer Venture Investments and SR One. Mission Therapeutics was founded in 2011 and is based at the Babraham Research Campus, Cambridge, UK.

About the Michael J. Fox Foundation for Parkinson’s Research
As the world’s largest nonprofit funder of Parkinson’s research, the Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson’s patients, business leaders, clinical trial participants, donors and volunteers.
In addition to funding more than $700 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson’s research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson’s disease clinical trials.