What's new?
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/

17/12/2014

BURLINGTON, Mass., Dec. 12, 2014 – Flexion Therapeutics, Inc. (Nasdaq:FLXN) today announced the pricing of its underwritten public offering of 5,040,000 shares of common stock at a price to the public of $17.00 per share. Flexion has also granted the underwriters a 30-day option to purchase up to an additional 756,000 shares of common stock. The offering is expected to close on December 17, 2014, subject to customary closing conditions.

BMO Capital Markets Corp. and RBC Capital Markets, LLC are acting as joint book-running managers. Needham & Company, LLC is acting as lead manager and Janney Montgomery Scott and Summer Street Research Partners are acting as co-managers.

Registration statements relating to the shares to be sold in the offering have become effective. The offering is being made only by means of a prospectus. A copy of the prospectus relating to the offering may be obtained from BMO Capital Markets Corp., 3 Times Square, 27th Floor, New York, NY 10036, Attention: Equity Syndicate Department, Telephone: (800) 414-3627, Email: bmoprospectus@bmo.com; or from RBC Capital Markets, LLC, 200 Vesey Street, 8th Floor, New York, NY 10281; Attention: Equity Syndicate Department, Telephone: (877) 822-4089, Fax: (212) 428-6260.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of Flexion’s securities. No offer, solicitation or sale will be made in any jurisdiction in which such offer, solicitation or sale is unlawful.

About Flexion Therapeutics
Flexion is a clinical-stage specialty pharmaceutical company focused on the development and commercialization of novel pain therapies. The company is currently advancing a portfolio of injectable drug candidates that have the potential to provide better and more persistent analgesia compared with existing therapy. The company’s lead program, FX006, is an intra-articular sustained release steroid in development for patients with moderate to severe OA pain. The company also has two additional product candidates, FX007, a locally administered TrkA receptor antagonist for post-operative pain, and FX005, an intra-articular, sustained-release p38 MAP kinase inhibitor for end-stage OA patients.

Forward-Looking Statements
Statements in this press release regarding matters that are not historical facts, including expectations regarding the completion and timing of Flexion’s proposed public offering, are forward-looking statements. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, risks and uncertainties associated with market conditions and the satisfaction of customary closing conditions related to the proposed offering, as well as other risks and uncertainties described in Flexion’s filings with the Securities and Exchange Commission (SEC), including under the heading « Risk Factors » in Flexion’s Annual Report on Form 10-K for the year ended December 31, 2013 and subsequent filings with the SEC. You are encouraged to read Flexion’s filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and Flexion undertakes no obligation to update or revise any of the statements.

 

CONTACT: Media Contact

Jamie Lacey-Moreira
PressComm PR, LLC
T: 410-299-3310
jamielacey@presscommpr.com

 

Corporate Contact
Lisa Davidson, MBA
Vice President, Finance and Administration

16/12/2014

Interim analysis indicates annualized six-month height velocity is comparable to the active comparator, Genotropin®, given at an equivalent cumulative weekly dose

Copenhagen, Denmark, December 15, 2014 – Ascendis Pharma A/S, a biotechnology company that applies its innovative TransCon technology to address significant unmet medical needs, today announced positive six-month interim results from its ongoing Phase 2 pediatric study to evaluate once-weekly TransCon Growth Hormone in children with growth hormone deficiency, or GHD. This interim analysis consists of 25 patients, representing approximately 50% of the anticipated total enrollment in the study, completing all six months of treatment.

“It is encouraging to see the significant mean annualized height velocity achieved by TransCon Growth Hormone at the six-month time point,” stated Dr. Ron Rosenfeld, member of the Ascendis Growth Hormone Advisory Board and Professor of Pediatrics at Stanford University (emeritus) and Professor of Pediatrics at Oregon Health and Science University (emeritus). “Based on this interim analysis, once-weekly TransCon Growth Hormone appears comparable to gold-standard daily growth hormone therapies in terms of efficacy, safety and tolerability, with a more convenient dosing regimen that may allow for improved patient compliance and treatment outcomes.”
“We are pleased with the progress we are making with the development of once-weekly TransCon Growth Hormone,” stated Jan Mikkelsen, President and Chief Executive Officer. “We look forward to completing our Phase 2 pediatric study and reporting top-line data in mid-2015. Based on the results observed thus far, we believe that TransCon Growth Hormone has the potential to be a best-in-class therapy for growth hormone deficient patients.”

Highlights from the interim analysis are:
 mean annualized height velocities among the three dosing levels administered weekly ranged from 11.9 cm for the 0.14 mg/kg/week dose to 14.5 cm for the 0.30 mg/kg/week dose, which were comparable to 11.5 cm for the active comparator, daily injections of

Genotropin® at a 0.21 mg/kg/week dose;
 there have been no reports of serious or unexpected adverse events;
 injection site reactions were generally mild and transient and occurred in only a few patients and there have been no observations of injection site nodule formation or lipoatrophy;
 maximum hGH blood concentration is comparable between equivalent weekly doses of TransCon Growth Hormone and daily hGH; and
 a dose-proportional increase in IGF-I levels was observed following dosing of the three TransCon Growth Hormone dose levels. Transient point values of IGF‐I SDS > +2 have been observed in a small number of patients and only in the high-dose treatment arm. The ongoing Phase 2 pediatric study is being conducted to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of TransCon Growth Hormone in treatmentnaïve pre-pubertal patients with GHD, who meet internationally recognized criteria for GHD. The study is a 6-month multi-center, randomized study comparing three dose levels of TransCon Growth Hormone (0.14; 0.21; and 0.30 mg hGH/kg/week), administered once per week, to the active control Genotropin (0.21 mg hGH/kg/week), administered as a daily injection.

About Growth Hormone Deficiency
Growth hormone deficiency, or GHD, is a serious orphan disease affecting both children and adults. In children, GHD manifests with short stature, metabolic abnormalities, cognitive deficiencies and poor quality of life. Adult GHD is associated with premature mortality and neuropsychiatriccognitive, cardiovascular, neuromuscular, metabolic and skeletal abnormalities. The global market for daily injections of human growth hormone was approximately $3 billion in 2013. There are currently no long-acting growth hormone treatment options available in the United States or Europe. The current standard of care for the treatment of GHD requires patients to receive daily injections of hGH over many years. The administrative burden of daily injections often results in poor patient compliance and can lead to suboptimal treatment outcomes.

About TransCon Growth Hormone
Ascendis Pharma is developing once-weekly TransCon Growth Hormone, an investigational new drug, to address the burden of daily injections and suboptimal treatment outcomes that can result from poor patient compliance. TransCon Growth Hormone is a prodrug that releases unmodified growth hormone, thus maintaining the same mode of action as currently prescribed daily growth hormone therapies. Clinical studies of TransCon Growth Hormone have demonstrated a comparable efficacy, safety, tolerability and immunogenic profile to that of daily growth hormone. If approved, TransCon Growth Hormone may reduce the burden of daily treatment by requiring significantly fewer injections, which may improve patient compliance and treatment outcomes. Ascendis Pharma has successfully completed a Phase 2 study of TransCon Growth Hormone in adults with GHD and is currently conducting a Phase 2 study in children with GHD. Ascendis Pharma expects to report top‐line data from the Phase 2 pediatric study in mid-2015.

About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology, which combines the benefits of prodrug and sustained release technologies, to develop a pipeline of best‐in‐class therapeutics that address significant unmet medical needs. The TransCon technology can be applied to new and existing drug therapies, including proteins, peptides and small molecules, to create prodrugs that provide for the predictable and sustained release of an unmodified parent drug. The Ascendis Pharma pipeline includes TransCon Growth Hormone, a proprietary program that has completed a Phase 2 study in adults with growth hormone deficiency, or GHD. Ascendis Pharma is currently conducting a Phase 2 study of TransCon Growth Hormone in children with GHD.
Ascendis Pharma is also developing TransCon Treprostinil for the treatment of pulmonary arterial hypertension. In addition to its proprietary programs, Ascendis Pharma has formed collaborations focused on market-leading products in large markets that are of strategic importance to its collaboration partners. These collaborations are with Sanofi in diabetes and an undisclosed market leader in the field of ophthalmology.

Contact:
Martin Auster, M.D. Thomas P. Soloway
Chief Business Officer Chief Financial Officer
(650) 617-3403 (650) 617-3409
MA@AscendisPharma.com TS@AscendisPharma.com

10/12/2014

Don deBethizy Joins NOXXON Supervisory Board

Berlin, Germany and Boston, USA – 10 December 2014 – NOXXON Pharma has appointed Don deBethizy to the Company’s Supervisory Board. In his last position as President and Chief Executive Officer of Santaris Pharma A/S, deBethizy initiated and managed the successful acquisition of the company by Roche. Previously, he was Executive Chairman of Danish bio-venture firm, Contera Pharma and oversaw its sale to Korean Bukwang Pharmaceutical. From 1997 to 2012, Dr. deBethizy was co-founder and CEO of Targacept, Inc. (NASDAQ:TRGT). He is currently on the Board of Directors of Newron Pharmaceuticals (SIX: NWRN). Don deBethizy holds a BS degree in Biology from the University of Maryland and received a Ph.D. in Toxicology from Utah State University. He will join the current Supervisory Board of NOXXON which consists of seven industry and investor representatives. “It is my pleasure to welcome Don to the team, as he is a highly qualified, experienced pharmaceutical company executive with significant experience of drug and technology development,” said Iain Buchanan, CEO of NOXXON Pharma. “His past experience with Santaris’ locked nucleic acid (LNA) platform and Targacept’s NNR Therapeutics are particularly relevant, and will be increasingly useful to NOXXON as we will continue to further progress and develop our pipeline.” “NOXXON has very promising compounds in development based on its unique Spielgelmer® platform, and the company is at an exciting inflection point,” said Dr. deBethizy. “I look forward to contributing to Board discussions and helping the management with company strategy and product development.”

 

About NOXXON Pharma

NOXXON Pharma is a biopharmaceutical company pioneering the development of a new class of proprietary therapeutics called Spiegelmers. Spiegelmers are chemically synthesized L-stereoisomer oligonucleotide aptamers, a non-immunogenic alternative to antibodies. NOXXON has a diversified portfolio of clinical-stage Spiegelmer® therapeutics: • Lexaptepid pegol (NOX-H94), an anti-hepcidin Spiegelmer®, has completed a Phase IIa pilot study in cancer patients with anemia and is now being studied in EPO-hyporesponsive dialysis patients. Hepcidin is the key regulator of iron metabolism and responsible for the iron restriction leading to anemia of chronic disease. • Olaptesed pegol (NOX-A12), an anti-CXCL12/SDF-1 (CXC chemokine ligand 12 / Stromal Cell-Derived Factor-1) Spiegelmer®, has completed Phase IIa studies in two hematological cancers, multiple myeloma (MM) and chronic lymphocytic leukemia (CLL). Studies for treatment of glioblastoma have also been designed and olaptesed pegol has received Orphan Drug Designation from the FDA and EMA for this indication. CXCL12 is a chemokine known to be involved in tumor invasion, metastasis and resistance to therapy. • Emapticap pegol (NOX-E36), an anti-CCL2/MCP-1 (C-C chemokine ligand 2 / Monocyte Chemoattractant Protein-1) Spiegelmer®, has completed a Phase IIa proof-of-concept study in patients with type 2 diabetes with albuminuria and a Phase IIb study is in the planning stages. CCL2 is a pro-inflammatory chemokine involved in the recruitment of immune cells to inflamed tissues. The Spiegelmer® platform provides the company with powerful and unique discovery capabilities, which have generated a number of additional leads under preclinical investigation. Located in Berlin, Germany, NOXXON is a well-financed mature biotech company with a strong syndicate of international investors, and approximately 55 employees. For more information, please visit: www.noxxon.com Contact: NOXXON Pharma AG Instinctif Partners Emmanuelle Delabre T: +49-30-726247-0 edelabre@noxxon.com Robert Mayer / Andreas Zunhammer T: +49-89-30905189-13 / -11 noxxon@instinctif.com

10/12/2014

Berlin, Germany and Boston, USA, Germany – 05 December 2014 – NOXXON Pharma will disclose promising data from two independent clinical Phase IIa studies of the anti-CXCL12/SDF-1 Spiegelmer® olaptesed pegol (NOX-A12) and show preclinical data supporting broad use of olaptesed pegol in combination with anti-cancer therapeutic antibodies at the 56th annual meeting of the American Society of Hematology (ASH) in San Francisco, CA from 06-09 December 2014.
In the first study, olaptesed pegol was administered with bortezomib and dexamethasone (VD) in patients with relapsed multiple myeloma (MM). In the second study, relapsed chronic lymphocytic leukemia (CLL) patients were treated with olaptesed pegol combined with bendamustine and rituximab (BR). Data from all 28 patients in each study will be presented.

In both studies, anti-CXCL12/SDF-1 Spiegelmer® olaptesed was able to mobilize cancer cells from protective niches in the body. This mobilization reflects olaptesed pegol’s ability to block tumor-microenvironment interactions and to modify the bone marrow environment to make it less receptive for malignant cells.

In patients with relapsed MM, an overall response rate (ORR) of 68% including 18% very good partial responses (vgPR) and 7% complete responses (CR) was achieved in the 28 patients. Importantly, treatment with olaptesed pegol was not associated with additional toxicity on top of VD. Notably, the response rate was essentially the same in patients with high-risk cytogenetics and patients not bearing the high-risk mutations/translocations.

In relapsed CLL patients, 82% overall response rate (ORR) and 14% complete response (CR) rate was observed which compares very favorably with historical controls receiving the underlying BR therapy. Further, all high-risk patients1 responded with a partial response (PR) or complete response (CR).
Preclinical work showed that in contrast to tested inhibitors of BTK (ibrutinib) and PI3Kδ (idelalisib), olaptesed does not inhibit antibody mediated cellular cytotoxicity or phagocytosis of rituximab. Furthermore, it not only mobilizes malignant cells, but also increases the number of circulating immune effector cells.2 Olaptesed pegol could thus be a partner of choice for anti-cancer monoclonal antibodies.
1 As defined by Stilgenbauer and Zenz (2010)
2 Vater A. et al. (2013), Clinical Pharmacology & Therapeutics

The titles and contributors for the three above mentioned poster presentations at ASH are as follows:
 Saturday, December 06, 2014, 5:30 PM-7:30 PM, West Building, Level 1 (Moscone Center), Session 653, Publication number: 2111
Final Results from the Phase IIa Study of the Anti-CXCL12 Spiegelmer® Olaptesed Pegol (NOX-A12) in Combination with Bortezomib and Dexamethasone in Patients with Multiple Myeloma
Heinz Ludwig, Katja Weisel, Maria Teresa Petrucci, Xavier Leleu, Anna Maria Cafro, Laurent Garderet, Niklas Zojer, Robin Foa, Richard Greil, Ibrahim Yakoub-Agha, Anna Kruschinski, Thomas Dümmler, Kai Riecke, and Monika Engelhardt
 Saturday, December 06, 2014, 5:30 PM-7:30 PM, West Building, Level 1 (Moscone Center); Session 642, Publication number: 1996
Results from a Phase IIa Study of the Anti-CXCL12 Spiegelmer® Olaptesed Pegol (NOX-A12) in Combination with Bendamustine/Rituximab in Patients with Chronic Lymphocytic Leukemia
Michael Steurer, Marco Montillo, Lydia Scarfò, Francesca Romana Mauro, Johannes Andel, Sophie Wildner, Livio Trentin, Ann Janssens, Sonja Burgstaller, Anna Kruschinski, Thomas Dümmler, Kai Riecke, Paolo Ghia, Federico Caligaris-Cappio and Marco Gobbi
 Monday, December 08, 2014, 6:00 PM-8:00 PM, West Building, Level 1 (Moscone Center), Session 625, Publication number: 4499
Comparison of Ibrutinib, Idelalisib and Olaptesed Pegol on the Immune Effector Function Mediated by Rituximab
Dirk Zboralski, Axel Vater and Anna Kruschinski
Members of NOXXON’s drug development team and clinical investigators will be at the ASH conference to explain the mode of action and clinical potential of this innovative drug candidate.
About NOXXON Pharma
NOXXON Pharma is a biopharmaceutical company pioneering the development of a new class of proprietary therapeutics called Spiegelmers. Spiegelmers are chemically synthesized L-stereoisomer oligonucleotide aptamers, a non-immunogenic alternative to antibodies. NOXXON has a diversified portfolio of clinical-stage Spiegelmer® therapeutics:
 Lexaptepid pegol (NOX-H94), an anti-hepcidin Spiegelmer®, has completed a Phase IIa pilot study in cancer patients with anemia and is now being studied in EPO-hyporesponsive dialysis patients. Hepcidin is the key regulator of iron metabolism and responsible for the iron restriction leading to anemia of chronic disease.
 Olaptesed pegol (NOX-A12), an anti-CXCL12/SDF-1 (CXC chemokine ligand 12 / Stromal Cell-Derived Factor-1) Spiegelmer®, has completed Phase IIa studies in two hematological cancers, multiple myeloma (MM) and chronic lymphocytic leukemia (CLL). Studies for treatment of glioblastoma have also been designed and olaptesed pegol has received Orphan Drug Designation from the FDA and
EMA for this indication. CXCL12 is a chemokine known to be involved in tumor invasion, metastasis and resistance to therapy.
 Emapticap pegol (NOX-E36), an anti-CCL2/MCP-1 (C-C chemokine ligand 2 / Monocyte Chemoattractant Protein-1) Spiegelmer®, has completed a Phase IIa proof-of-concept study in patients with type 2 diabetes with albuminuria and a Phase IIb study is in the planning stages. CCL2 is a pro-inflammatory chemokine involved in the recruitment of immune cells to inflamed tissues.
The Spiegelmer® platform provides the company with powerful and unique discovery capabilities, which have generated a number of additional leads under preclinical investigation. Located in Berlin, Germany, NOXXON is a well-financed mature biotech company with a strong syndicate of international investors, and approximately 55 employees.

For more information, please visit: www.noxxon.com
Contact:
NOXXON Pharma AG
Instinctif Partners
Emmanuelle Delabre
T: +49-30-726247-0
edelabre@noxxon.com
Robert Mayer / Andreas Zunhammer
T: +49-89-30905189-13 / -11
noxxon@instinctif.com

04/12/2014

Proceeds Will Be Used to Advance Clinical Programs in Growth Hormone Deficiency and Pulmonary Arterial Hypertension Copenhagen, Denmark, December 1, 2014 / — Ascendis Pharma A/S, a biotechnology company that applies its innovative TransCon technology to address significant unmet medical needs, today announced the successful completion of a $60 million Series D financing. The financing was co-led by U.S.-based Sofinnova Ventures, OrbiMed and Vivo Capital, and included Janus Capital Management LLC, Venrock, RA Capital Management, Rock Springs Capital and Sectoral Asset Management. The company’s largest existing shareholder, Paris-based Sofinnova Partners, also participated in the financing. In conjunction with the financing, James Healy, M.D., Ph.D., of Sofinnova Ventures, Jonathan Silverstein, J.D., of OrbiMed, and Albert Cha, M.D., Ph.D., of Vivo Capital have joined the Ascendis Pharma board of directors.

The proceeds from the Series D financing are expected to be used to fund late-stage clinical trials of TransCon Growth Hormone for the treatment of growth hormone deficiency, or GHD, and in 2015, to initiate a Phase 1 proof-of-concept program of TransCon Treprostinil to treat pulmonary arterial hypertension. Proceeds are also expected to be used to support drug manufacturing, advance the development of preclinical drug candidates, and to enhance the Ascendis Pharma clinical development and research organizations.

“We are pleased to have some of the world’s leading healthcare investors participate in our Series D financing, join our board of directors and demonstrate their support for our technology platform and clinical development programs,” stated Jan Mikkelsen, President and Chief Executive Officer of Ascendis Pharma. “This Series D financing will fund important advances in our product development pipeline and improvements in our technology platforms, all with the goal of accelerating our ability to bring innovative new therapies to market.”

Jonathan Silverstein, J.D., Partner and Co-Head of Global Private Equity at OrbiMed, stated, “We believe that the proprietary approach employed by Ascendis Pharma to create long-acting prodrugs holds the promise to meet unmet medical needs in a broad array of therapeutic areas. We are particularly encouraged by the clinical results from the recent interim analysis of the company’s ongoing Phase 2 pediatric study of TransCon Growth Hormone in the field of GHD.”

Rafaèle Tordjman, M.D., Ph.D., Managing Partner at Sofinnova Partners and a member of the Ascendis Pharma board of directors, commented, “We are pleased to participate in this investment alongside a group of experienced biotechnology investors. The closing of this financing and addition of our new board members heralds a new stage for Ascendis Pharma, a company we have backed since 2007.”

About Growth Hormone Deficiency
Growth hormone deficiency, or GHD, is a serious orphan disease affecting both children and adults. In children, GHD manifests with short stature, metabolic abnormalities, cognitive deficiencies and poor quality of life. Adult GHD is associated with premature mortality and neuropsychiatric-cognitive, cardiovascular, neuromuscular, metabolic and skeletal abnormalities. The global market for daily injections of human growth hormone was approximately $3 billion in 2013. There are currently no long-acting growth hormone treatment options available in the United States or Europe. The current standard of care for the treatment of GHD requires patients to receive daily injections over many years. The administrative burden of daily injections often results in poor patient compliance and can lead to suboptimal treatment outcomes.

About TransCon Growth Hormone
Ascendis Pharma is developing once-weekly TransCon Growth Hormone, an investigational new drug, to address the burden of daily injections and suboptimal treatment outcomes that can result from poor patient compliance. TransCon Growth Hormone is a prodrug that releases unmodified growth hormone, thus maintaining the same mode of action as currently prescribed daily growth hormone therapies. Clinical studies of TransCon Growth Hormone have demonstrated a comparable efficacy, safety, tolerability and immunogenic profile to that of daily growth hormone. If approved, TransCon Growth Hormone may reduce the burden of daily treatment by requiring significantly fewer injections, which may improve patient compliance and treatment outcomes. Ascendis Pharma has successfully completed a Phase 2 study of TransCon Growth Hormone in adults with GHD and is currently enrolling children with GHD in a Phase 2 pediatric study. Ascendis Pharma expects to report interim data from its Phase 2 pediatric study in December of 2014, and top‐line data from the entire study in mid-2015.

About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology, which combines the benefits of prodrug and sustained release technologies, to develop a pipeline of best‐in‐class therapeutics that address significant unmet medical needs. The TransCon technology can be applied to existing drug therapies, including proteins, peptides and small molecules, to create prodrugs that provide for the predictable and sustained release of an unmodified parent drug. Ascendis Pharma has a diversified and balanced pipeline. TransCon Growth Hormone is a proprietary program that has completed a Phase 2 study in adults with growth hormone deficiency, or GHD, and is currently enrolling children with GHD in a Phase 2 study. Ascendis Pharma is also developing TransCon Treprostinil for the treatment of pulmonary arterial hypertension. In addition to its proprietary programs, Ascendis Pharma has formed collaborations focused on leading products in large markets that are of strategic importance to its collaboration partners. These collaborations are with Sanofi in diabetes and an undisclosed market leader in the field of ophthalmology.

Contact:
Thomas P. Soloway Martin Auster, M.D. Chief Financial Officer Chief Business Officer (650) 617-3409 (650) 617-3403 TS@AscendisPharma.com MA@AscendisPharma.com

04/12/2014

– Company Resumes Recruitment in Pivotal Phase 2b Trial; Initiation of Phase 3 Clinical Trial Planned for Early 2015
– Conference Call Scheduled for Tomorrow, December 2, 2014 at 9:00 a.m. ET

BURLINGTON, Mass., Dec. 1, 2014  — Flexion Therapeutics, Inc. (Nasdaq:FLXN) today announced that the U.S. Food and Drug Administration (FDA) has notified the company that it has lifted the clinical hold on Flexion’s lead drug candidate FX006, based upon the company successfully completing the FDA’s requested testing and investigation related to what was originally reported as a single case of septic arthritis of the knee. As a result, Flexion intends to immediately resume recruitment and dosing in its pivotal Phase 2b trial of FX006, and to initiate a planned Phase 3 trial in early 2015. FX006 is a first-in-class injectable, sustained-release, intra-articular steroid treatment in development for patients with moderate to severe osteoarthritis (OA) pain.
Michael Clayman, M.D., Flexion Therapeutics President and CEO, said, « We are pleased to have been able to rapidly provide data to the FDA that allowed the agency to remove the clinical hold. Now we can continue to advance FX006, which has the potential to make a real difference for the many individuals who suffer from OA pain. »
On September 16, 2014, the FDA placed a clinical hold on FX006 due to a single occurrence of what was then described as an infection in the injected knee joint of a patient in the pivotal Phase 2b trial. The subsequent clinical hold letter from the FDA requested that the company:
• Determine whether the study drug was the source of infection by recovering both the specific study drug vials used in the treatment of the patient who experienced the infection, as well as unused study drug vials from the clinical site where the patient was injected and test them for contamination, and
• Explore other potential causes for infection, including a compromise of sterile procedures during injection.
In accordance with these FDA requests, the company performed industry standard contamination tests through a certified third-party sterility-testing firm, which demonstrated that no microbial growth could be detected in any of the used or unused vials. As a result, the company concluded that study drug was not contaminated. The company also explored other potential causes of possible infection including contamination during the preparation or administration of FX006. Following consultations with the principal investigator, the study coordinator that prepared the study drug, and the healthcare professional that administered the injection at the clinical site, the company could not find any indication that sterile procedures were compromised during the injection. There have been no other infections noted in the approximately 100 other patients dosed with FX006 in this trial.
On October 28, 2014, the company received notification that based on the highly atypical nature of this patient’s presentation as it relates to septic arthritis and the subsequent clinical course, which was most consistent with rheumatoid arthritis, the principal investigator has changed the initial serious adverse event diagnosis from septic arthritis, possibly related to study drug treatment, to inflammatory arthritis, unrelated to study drug treatment. The original, and only, positive synovial fluid culture obtained from this patient is presumed to be a false positive, which occurs in about five percent of such cases. Thus there have been no confirmed diagnoses of septic arthritis among the more than 300 patients treated with FX006 in all clinical trials to date and no serious adverse events that have been attributed to study drug.

About Flexion Therapeutics
Flexion is a clinical-stage specialty pharmaceutical company focused on the development and commercialization of novel pain therapies. The company is currently advancing a portfolio of injectable drug candidates that have the potential to provide better and more persistent analgesia compared with existing therapy. The company’s lead program, FX006, is an intra-articular sustained release steroid in development for patients with moderate to severe OA pain. The company also has two additional product candidates, FX007, a locally administered TrkA receptor antagonist for post-operative pain, and FX005, an intra-articular, sustained-release p38 MAP kinase inhibitor for end-stage OA patients.
Conference Call
Flexion’s management team will host a conference call and webcast at 9:00 a.m. EST tomorrow, December 2, 2014, to discuss the removal of the clinical hold and to provide a clinical timing update. The dial-in number for the conference call is toll-free (855) 770-0022 for domestic participants and (908) 982-4677 for international participants. A live webcast of the conference call can also be accessed through the « Investors » tab on the Flexion Therapeutics website at www.flexiontherapeutics.com. A webcast replay will be available online after the call.

Forward-Looking Statements
Statements in this press release regarding matters that are not historical facts, including statements relating to the future of Flexion, its ongoing development of its product candidates, expectations of resuming the FX006 Phase 2b clinical trial, plans and timing for initiating a Phase 3 trial, anticipated clinical and other milestones (including the timing of such milestones) and potential benefits of FX006 are forward-looking statements. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, risks associated with the process of discovering, developing and obtaining regulatory approval for drugs that are safe and effective for use as human therapeutics, the fact that Flexion relies on third parties to manufacture and conduct the clinical trials of its product candidates, which could delay or limit their future development or regulatory approval, the fact that Flexion will require additional capital, including prior to completing Phase 3 development of, filing for regulatory approval for, or commercializing, FX006 or any of its other product candidates and may be unable to obtain such additional capital in sufficient amounts or on terms acceptable to it, and other risks and uncertainties described in Flexion’s filings with the Securities and Exchange Commission (SEC), including under the heading « Risk Factors » in Flexion’s Annual Report on Form 10-K for the year ended December 31, 2013 and subsequent filings with the SEC. You are encouraged to read Flexion’s filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this press release, and Flexion undertakes no obligation to update or revise any of the statements.

CONTACT: Media Contact
Jamie Lacey-Moreira
PressComm PR, LLC
T: 410-299-3310
jamielacey@presscommpr.com

Corporate Contact
Lisa Davidson, MBA
Vice President, Finance and Administration
Flexion Therapeutics, Inc.
T: 781-305-7765
ldavidson@flexiontherapeutics.com

Source: Flexion Therapeutics, Inc.

24/11/2014

LEIDEN, the Netherlands, Nov. 24, 2014 — ProQR Therapeutics N.V. (Nasdaq:PRQR), an innovative biopharmaceutical company engaged in the discovery and development of RNA-based therapeutics for the treatment of severe genetic disorders, today announced results for the third quarter of 2014.
« ProQR has made significant accomplishments, both financially and operationally, in 2014, » said Daniel de Boer, Chief Executive Officer of ProQR. « With the successful completion of our recent initial public offering, we have strengthened our balance sheet, which will allow us to advance the clinical development of QR-010 for the treatment of patients with cystic fibrosis and continue to build our platform and pipeline of proprietary programs. »

Financial Highlights
Net cash generated by financing activities during the three and nine months ended September 30, 2014 was €80.2 million and €119.7 million, respectively. This includes the net proceeds from our IPO in the third quarter of 2014 and the net proceeds from the private placement in the second quarter, as well as proceeds from borrowings, representing receipts from our Innovation credit grant. As of September 30, 2014, we held cash and cash equivalents of €115.3 million. Net cash used in operating activities during the three and nine months ended September 30, 2014 was €4.3 million and €9.5 million, respectively.
Research and development costs were €2.5 million for the three months ended September 30, 2014, compared to €0.7 million for the same period in 2013. Research and development costs for the nine months ended September 30, 2014 were €7.0 million compared to €1.3 million for the same period in 2013. The increase reflects the expansion of research and development activities to support the further development of our lead product candidate, QR-010, for the treatment of cystic fibrosis, as well as the development of our other pipeline product candidates, including QR-110 for the treatment of Leber’s Congenital Amaurosis, the leading genetic cause of childhood blindness.
General and administrative costs were €2.4 million for the three months ended September 30, 2014, compared to €0.2 million for the same period in 2013. General and administrative costs for the nine months ended September 30, 2014 were €4.5 million compared to €0.4 million for the same period in 2013. The increase reflects the preparatory costs associated with the Company’s IPO on the Nasdaq Global market, a grant to ProQR’s protection foundation, and increased costs reflecting the Company’s growth.
Net loss for the three months ended September 30, 2014 was €3.4 million or €0.43 per share, compared to €0.9 million or €0.14 per share for the same period in 2013. Net loss for the nine months ended September 30, 2014 was €10.1 million or €1.45 per share, compared to €1.6 million or €0.30 per share for the same period in 2013. For further financial information for the period ending September 30, 2014, please refer to the financial statements appearing at the end of this release.

Corporate Highlights
• Entered into an agreement with Cystic Fibrosis Foundation Therapeutics, Inc. (« CFFT »), a subsidiary of the Cystic Fibrosis Foundation, in August 2014, pursuant to which CFFT agreed to provide us with up to $3 million to support the clinical development of QR-010. The Cystic Fibrosis Foundation is a professional patient advocacy organization that supports select CF drug development programs by providing financial support and access to its Therapeutic Development Network, a network of CF key opinion leaders, as well as to an extensive clinical trial network infrastructure.

• Successfully completed our initial public offering on NASDAQ in September 2014, placing 8,625,000 ordinary shares at a public offering price of $13.00 per share, which includes the exercise in full by the underwriters of their option to purchase 1,125,000 additional ordinary shares, resulting in aggregate net proceeds, after underwriting discounts and commissions and other estimated offering expenses, of approximately $102 million (€79.3 million).

• Appointed Alison Lawton as a member of our Supervisory Board of Directors in September 2014. Ms. Lawton brings significant operational, international, regulatory and senior management experience within the pharmaceutical and biotechnology industries. She currently serves as a board member of Cubist Pharmaceuticals, Inc. (Nasdaq:CBST) and Verastem, Inc. (Nasdaq:VSTM) and previously served as Chief Operating Officer of OvaScience, Inc. (Nasdaq:OVAS).
Subsequent Events
• Appointed Smital Shah as our Chief Financial Officer in October 2014. Ms. Shah has a track record of management and leadership experience in the biopharmaceutical industry as well as in investment banking, with a particular focus on financial strategy and capital markets.

• Entered into an agreement with PARI Pharma GmbH in October 2014, pursuant to which the Company is granted exclusive access to the use of the PARI e-flow nebulizer for the administration of nucleotide-based drugs in ∆F508 mutation in cystic fibrosis, with the exclusive option to expand this exclusivity to the use in other CF mutations. Pursuant to the terms of the agreement, we have made an upfront payment, hourly fees for development work and are obligated to make sales-based royalty payments after market authorization.

About ProQR
ProQR is an innovative biopharmaceutical company engaged in the discovery and development of RNA-based therapeutics for the treatment of severe genetic disorders, with an initial focus on Cystic Fibrosis. Utilizing our unique, proprietary RNA repair technologies, we believe we will be able to treat genetic disorders in which a single protein is defective due to certain types of genetic mutation. We believe that this is a unique approach that offers advantages compared with small molecule, gene therapy and other therapeutic strategies.

 

view the entire press release

 

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 initiation of clinical trials of QR-010 and statements regarding our ongoing and planned discovery and development of product candidates. 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 described under the heading « Risk Factors » in ProQR’s prospectus filed with the Securities and Exchange Commission dated September 19, 2014. 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.

ProQR:
Smital Shah
Chief Financial Officer
T: +1 650 464 9879
ir@proqr.com
Media inquiries:
Gretchen Schweitzer
MacDougall Biomedical Communications
Direct: +49 172 861 8540
Main: +49 89 2424 3494
gschweitzer@macbiocom.com
Source: ProQR Therapeutics N.V.

19/11/2014

Geneva, Switzerland, 18 November 2014 – ObsEva, a Swiss biopharmaceutical company dedicated to the development and commercialization of innovative drugs for women’s reproductive medicine, announced today the initiation of a Phase 3-enabling, Phase 2 study of its lead compound, OBE001, a novel orally active oxytocin receptor antagonist. The IMPLANT study is designed to assess the safety and efficacy of a range of doses of OBE001 compared to placebo in women undergoing embryo transfer after IVF/ICSI. The primary objective of this study is to assess the increase in clinical pregnancy rate. “Initiation of this study is an important milestone for our young company and demonstrates ObsEva’s expertise and operational capabilities as we continue to execute our product development plan” stated Ernest Loumaye, CEO and Co-Founder of ObsEva. The study is a prospective, dose-finding randomised, parallel group, double-blind, placebo-controlled study. It is planned to recruit 240 patients at 30 specialist IVF centres across 6 European countries including Belgium, Czech Republic, Denmark, Poland, Spain and UK. “The study will recruit women who are at risk of embryo implantation failure due to the presence of uterine contractions at the time of embryo transfer” added Andrew Humberstone, Head of Clinical Operations of ObsEva. “They will be administered a single, oral dose of OBE001 or placebo before embryo transfer”. The specialist IVF centre at Brussels Hospital University (UZB) in Belgium has been the first site to be initiated on November 17, 2014. Professor Herman Tournaye is the principal investigator for the IMPLANT study at that site. “Implantation is a key to success in ART, however, it largely remains a black box. Given the fact that uterine contractions may have a negative impact on this process, as a clinician, I am glad to have the opportunity to participate to the IMPLANT study which assesses the potential of this new compound to inhibit uterine contractions and improve success rates in ART” said Pr. Herman Tournaye, Head of the Centre for Reproductive Medicine, Brussels Hospital University (UZB), Belgium. About Assisted Reproductive Technology (ART) and About OBE001 Infertility affects about 10% of reproductive-aged couples. Every year, worldwide, about 1.6 million of ART/IVF treatments are performed of which 176’000 are in the USA and 588’000 in the EU. The success rate of such treatment is highly dependent of the age of the woman and is on average 20-25% Take Home Baby rate per treatment cycle. The cost of one ART/IVF cycle varies between USD 8’000-15’000 in the USA, EUR 2’000-10’000 in the EU and USD 2’000-6’000 in Japan. There is good evidence that uterine contractile activity at the time of embryo transfer could expel embryos from the uterus. It is estimated that about 30% of patients undergoing embryo transfer have pronounced uterine contractions. OBE001 is a new generation of oxytocin antagonist with the potential to inhibit uterine contractions at the time of embryo transfer, thereby enhancing embryo implantation during ART and increase ART Take Home Baby rates. About ObsEva ObsEva SA is a clinical stage, Swiss biopharmaceutical company dedicated to the development and commercialization of innovative drugs for women’s reproductive medicine. ObsEva was founded in November 2012, by Ernest Loumaye MD, PhD and André Chollet PhD. Ernest Loumaye is a specialist in female reproductive medicine with 20 years of experience in the biopharmaceutical industry. Ernest Loumaye was previously Co-Founder and CEO of PregLem SA, a successful biopharmaceutical company which was acquired by Gedeon Richter in 2010. André Chollet is specialist in medicinal and pharmaceutical chemistry with more than 30 years of experience in various positions in the biopharmaceutical industry. André Chollet was responsible for the preterm labor program at Serono before the acquisition of the company by Merck KGaA. ObsEva’s founding assets are innovative products at early stages of clinical development addressing preterm labor and infertility treatment as well as additional indications in reproductive medicine. For more information, please visit www.obseva.com. For further information, please contact ObsEva CEO Office: Delphine Renaud Tel: +41 22 552 1550 Email: delphine.renaud@obseva.ch

07/11/2014

Amsterdam 7 November, 2014. MyTomorrows, a pioneering healthcare platform that facilitates patient access to experimental treatments, has raised €4.5m in its first institutional financing. The round was led by Balderton Capital, the leading European tech investor, and Sofinnova Partners, a Paris-based venture capital fund with a 40 year legacy in life sciences investments. MyTomorrows was founded by seasoned physicians and biotech entrepreneurs. Co-founders include Dr. Ronald Brus, former CEO of Crucell, a vaccine company acquired by Johnson & Johnson for $2.4bn in 2011. In Europe, each year around 1 million patients with debilitating diseases face their diagnosis with little or no therapeutic options for their condition. In addition, about 95% of these patients do not meet the eligibility criteria to enter a clinical trial of an investigational drug. In order to address this medical urgency, regulators often allow patients to access experimental drugs through so-called compassionate use programmes. While potentially life-saving, these are highly complex and bureaucratic solutions for patients, physicians and drug providers. MyTomorrows is an innovative online platform that uses existing legal frameworks to facilitate patient/physician access to a number of promising experimental treatments. Although still in development, these medicines have shown initial signs of therapeutic benefit, and importantly, they have an acceptable safety profile. The platform currently offers drugs in oncology, CNS and orphan diseases, conditions regarded as high unmet medical needs. “The team at MyTomorrows have each experienced the frustrations of the current system. Rather than accept it, we are working hard to improve it for all, for good. I am delighted to welcome Balderton and Sofinnova Partners, the two leading VC investors in technology and life sciences, to the myTomorrows team. More than just capital, they bring vast global experience and network as we help drug developers reach doctors and patients facing unmet medical needs,” commented CEO/Founder Ronald Brus, MD. This financing round will enable expansion into new disease areas and into additional European territories. The proceeds will also develop the platform with decision-making tools that identify those patients who will most benefit from the therapies. Finally, the financing will help establish business operations in the US. “Governments recognise that there is a real need for access to early stage drugs and have devised compassionate laws to let this happen. MyTomorrows’ platform unlocks the intention of the existing legislation, allowing doctors and patients to get treatment quickly, cost effectively and with due diligence. This is an application of technology which can make a massive and meaningful difference to people’s lives,” added Mark Evans, General Partner Balderton Capital. “MyTomorrows brings innovative investigational treatments to patients with very severe pathologies who have exhausted all other therapeutic options. If you are one of these patients, getting access to new drugs is incredibly difficult. MyTomorrows’ platform creates a controlled environment in which a product under development can be efficiently provided to those in need,” commented Antoine Papiernik, Managing Partner, Sofinnova Partners. myTomorrows, Access for Good™

07/11/2014

Data Presented at VIVA and TCT 2014 Demonstrate Safety and Effectiveness

FREMONT, Calif. & LAS VEGAS – Nov 5, 2014-Shockwave Medical, a pioneer in the treatment of peripheral and coronary vascular disease, today announced positive clinical results from DISRUPT PAD, a single-arm multicenter study evaluating the safety and utility of Lithoplasty™ balloon catheters for the treatment of peripheral artery disease, at the Vascular Interventional Advances (VIVA) Annual Conference in Las Vegas, Nev.
“Calcified vascular lesions remain a major impediment to modern percutaneous therapy for vascular disease”
In advanced vascular disease, atherosclerosis becomes calcified deep inside the vessel walls, limiting blood flow. These deposits are difficult to treat because they limit the effectiveness of current endovascular devices, making today’s interventions challenging and prone to both procedural and long-term failure. Lithoplasty is a novel balloon-based technology that utilizes integrated lithotripsy, a pulsatile mechanical energy commonly used to break up kidney stones, to disrupt both superficial and deep calcium and normalize vessel wall compliance prior to low-pressure balloon dilatation. Lithoplasty is designed to be naturally gentle to soft tissue (non-diseased portions of the vessel) while remaining hard on calcium, the tissue that limits vessel expansion and the effectiveness of current technologies.
Early clinical evaluation from 35 patients with calcified vascular stenosis of the superficial femoral artery (SFA) and popliteal artery demonstrated safe and effective dilatation of calcified stenosis with no acute failures, favorable residual stenosis, and no re-intervention out to 30 days, with no major adverse events. Primary efficacy results demonstrated 100% success, defined as ability to achieve less than 50% residual stenosis using Lithoplasty with or without adjunctive angioplasty. Device success was 87%, defined as ability to achieve less than 50% residual stenosis using Lithoplasty alone. Importantly, an average residual stenosis of 23% (initial 76%), with no difference in the ability to dilate lesions between moderate (36%) and severely (64%) calcified lesions, was noted. Thirty-day patency assessed by duplex ultrasound was 100%.
“Calcified vascular lesions remain a major impediment to modern percutaneous therapy for vascular disease,” said Marianne Brodmann, M.D., of the Medical University of Graz, Austria, principal investigator of the study, who presented the results at the conference. “The results of this trial demonstrate that, unlike current devices that treat only superficial calcium, Shockwave’s Lithoplasty system promises to be effective on all types of calcium, including deep calcium – the type known to limit vessel expansion.”
“We are excited about these excellent results, which suggest that Lithoplasty technology can address a substantial unmet clinical need in a large subset of patients with peripheral artery disease,” said Shockwave Medical CEO and co-founder Daniel Hawkins. “These results build upon the findings from our coronary First-In-Man study presented earlier this fall at the Transcatheter Cardiovascular Therapeutics (TCT) conference, which demonstrated the promise of Lithoplasty for the treatment of calcified coronary lesions. We look forward to confirming these promising findings in additional future studies, including the European multi-center DISRUPT CAD study planned for next year.”

First-In-Man Results Presented at TCT 2014
Results from the First-In-Man study of five patients with moderate to severe coronary calcification presented in September at the annual TCT conference in Washington, D.C. demonstrated safety, tolerability, deliverability, and effectiveness for Lithoplasty as a pre-treatment of calcified coronary lesions prior to stenting.
“Disruption of calcium within the vessel wall with Lithoplasty enables the use of low balloon pressures to dilate lesions and improve blood flow. This has the potential to minimize barotrauma and soft tissue vascular injury common to traditional balloons and existing endovascular technologies – an exciting breakthrough for patients with difficult-to-treat coronary calcifications, which continues to be a major problem in cardiology today,” said Todd Brinton, M.D., co-founder of Shockwave Medical and Clinical Associate Professor of Interventional Cardiology, Stanford University.
About Shockwave’s Lithoplasty™ System
Unlike current devices that treat only superficial calcium, Shockwave’s Lithoplasty system is designed to be effective on all types of calcium, including deep – the type known to limit vessel expansion. Delivered on a standard balloon catheter platform, Lithoplasty combines the calcium disrupting power of lithotripsy with the familiarity and simplicity of a balloon in a single enabling device. Lithoplasty applies a brief series of powerful mechanical pulses designed to safely travel through soft tissue, disrupt and pre-treat calcium. The integrated balloon is then dilated at low pressures to expand the lesion evenly, potentially minimizing acute soft tissue injury that could lead to the need for additional interventional treatments, or long-term restenosis (re-blockage).
Early clinical results demonstrate safety, tolerability, deliverability, and effectiveness for Lithoplasty as a treatment for patients with peripheral artery disease and as a pre-treatment of calcified coronary lesions prior to stenting. Shockwave has completed patient enrollment for a CE study of the technology in the peripheral vasculature. Clinical work has also been conducted in coronary vessels, and will begin in aortic valves next year.
To view an animation of Lithoplasty visit http://shockwavemedical.com/technology.

About Shockwave Medical
Shockwave Medical, based in Fremont, Calif., is pioneering Lithoplasty™ technology, a promising breakthrough for the treatment of peripheral and coronary vascular disease and aortic stenosis. Delivered on a standard balloon catheter platform, Lithoplasty combines the calcium disrupting power of lithotripsy with the familiarity and simplicity of a balloon in a single enabling device. For more information visit www.shockwavemedical.com.

Contacts
Shockwave Medical
Nicole Osmer, 650-454-0504
nicole@nicoleosmer.com