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10/07/2018

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

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

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

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

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

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

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

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

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

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

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

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

21/01/2004

Madison, N.J., and Munich, Germany, January 21, 2004 – Wyeth (NYSE: WYE) and Ingenium Pharmaceuticals AG announced today an agreement under which Ingenium shall provide Wyeth with novel genetic rat models with alterations in genes specified by Wyeth. The models will be developed using Ingenium’s INGENOtyping™ technology,which is the most rapid technology for generating genetic mouse models and which Ingenium has now established as the world’s first industrialized platform for developing genetic rat models. No financial terms of the agreement have been disclosed.As opposed to mouse “knockout” models, genetic rat models have not been broadly available, despite years of intense research worldwide, due to rat-specific limitations in the standard ES-cell based technology used for producing mouse models. Nevertheless, the rat remains the model of choice for many therapeutic areas, including cardiovascular, behavioural and neurological disease research.“Our INGENOtyping™ technology enables the development of rat genetic models across the genome, which is a significant advance for pharmaceutical development,” said Michael C. Nehls, M.D., Ph.D., CEO of Ingenium. “We are pleased to provide the advantages of this groundbreaking technology to Wyeth, a true leader in innovation.” Ingenium has created a “rolling-circle” production pipeline that represents a living, inbred, and gene-altered rat archive. On average, one year’s production represents an archive size sufficient for greater than two-fold mutational coverage on a gene-by-gene basis. This translates into an expected probability of over 90% to develop the desired gene-specific rat variant in less than 12 months. Ingenium’s INGENOtyping™ technology for genetic rat models is therefore comparable in time and efficiency to what current murine ES-cell-based technology provides for producing genetic mouse models. Wyeth is one of the world’s largest research-driven pharmaceutical and health care products companies. It is a leader in the discovery, development, manufacturing and marketing of pharmaceuticals, vaccines, biotechnology products and non-prescription medicines that improve the quality of life for people worldwide. The Company’s major divisions include Wyeth Pharmaceuticals, Wyeth Consumer Healthcare and Fort Dodge Animal Health. The basis of Ingenium’s business is its knowledge and expertise in generating the biological information critical to the discovery, validation and development of therapeutics. Ingenium’s drug discovery technologies combine functional genetics in the murine model system with proprietary, therapeutic goal-oriented biological screens and advanced pathway analysis. From the breadth of knowledge generated by the Wyeth-Ingenium Genetic Rat Model Agreement January 21, 2004 Page 2 of 2 company’s Deductive Genomics® technology, Ingenium is currently advancing a pipeline of novel models and biologically validated drug targets in the areas of inflammatory bowel disease, obesity/diabetes, and neurological disorders. The company’s INGENOtypingä platform offers the most rapid access to innovative rodent models for drug discovery and development and drug target validation. Ingenium has research partnership agreements with Elan Corporation, Bayer AG, F. Hoffmann-La Roche Ltd., Molecular Engines Laboratories, Sequenom Inc., and Oxagen Limited, in addition to numerous international academic collaborations. Ingenium and Deductive Genomics are registered U.S. trademarks.


For more information contact:
For Ingenium Gretchen L.P. Schweitzer Vice President Ingenium Pharmaceuticals AG mailto:gretchen.schweitzer@ingenium-ag.com


Visit website http://www.ingenium-ag.com

21/01/2004

Co-organisé par le Sénat et l’Essec, Tremplin Entreprises est le premier événement du capital investissement en France. Son objectif est de permettre à des entrepreneurs innovants, sélectionnés par un comité composé de professionnels du capital investissement et de la création d’entreprise, de rencontrer les investisseurs susceptibles de financer leur jeune entreprise ou leur projet de création.

Sofinnova Partners est un partenaire de premier rang, Jean-Bernard Schmidt est membre du Comité Executif, Alain Rodermann et Denis Lucquin sont membres des comités de sélection.

La sixième édition de Tremplin Entreprises distinguera les 6 et 7 juillet 2004, au Sénat, 30 lauréats, dans 3 catégories :
1) Technologies de l’information et de la communication,
2) Sciences de la Vie, et
3) Sciences de l’ingénieur (industrie, commerce, services).

Inscriptions (ouverture des candidatures le 2 février, clôture le 31 mars) et informations complémentaires sur http://www.tremplin-entreprises.senat.fr/


Visit website http://www.tremplin-entreprises.senat.fr

29/12/2003

 

19/12/2003

 

19/12/2003

 

12/12/2003

More than 50% of patients experience complete disappearance of tumors
Milan, Italy, 9 December 2003 – Novuspharma SpA (Nuovo Mercato: NOV.MI and NOV IM), a biopharmaceutical company focused on developing new cancer therapeutics, today announces results from a phase I study where Pixantrone was administered instead of doxorubicin as part of the CHOP regimen (the new CPOP combination), in patients with relapsed aggressive non-Hodgkin’s lymphoma (NHL). These initial results indicate that Pixantrone may be safely administered to relapsed patients who have previously received their maximum permitted dose of standard anthracyclines, while resulting in impressive response rates. Among the 22 patients evaluable for a response, 13 patients (59 percent) had a complete response (total disappearance of tumor), with 4 patients experiencing a 50 percent or greater shrinkage in their tumor. This corresponds to a major objective response rate of 77 percent. At the highest Pixantrone dose (150mg/m²), all 7 patients (100 percent) responded, with 6 patients (85 percent) experiencing a complete response. In the light of these highly promising results a phase II extension trial has been initiated, recruiting up to 25 patients in Europe. Results were presented on Sunday, December 7, at the 45th Meeting of the American Society of Hematology (ASH), in San Diego, California.
Updated results from a phase I/II trial of Pixantrone in the BSHAP regimen were also presented at ASH. In this trial Pixantrone was administered in combination with steroids, platinum and ARA-C, to 21 patients who had failed one or more prior multi-chemotherapeutic regimens for relapsed aggressive NHL. Sixty-Four percent of patients had disease refractory to prior chemotherapy. Of the 18 patients evaluable for response, 61 percent had a major tumor response, with 39 percent demonstrating a complete response and 22 percent experiencing a tumor shrinkage of at least 50 percent (PR). Furthermore, the updated results reveal that 6 patients (over half of those who responded) have proceeded to bone marrow transplant. Bone marrow transplant is currently the only procedure which can cure patients with relapsed aggressive NHL. No clinically significant cardiac events or decreases in cardiac function were reported.
CHOP and the new CHOP-variant regimen containing Pixantrone
The CHOP chemotherapy regimen (a combination of cyclophosphamide, vincristine, prednisone and doxorubicin) is the standard-of-care treatment for newly diagnosed (front line) aggressive NHL, which represents an estimated population of 14,500 patients. Response rates following CHOP in front line aggressive NHL can reach 70% and the regimen is potentially curative in up to 35 to 40% of patients. The prognosis is poor for patients who have a recurrence of the disease (relapsed patients). Despite its impressive anti-tumor activity, CHOP cannot be used to retreat the 60 to 65% of patients who will relapse following CHOP, due to the cumulative cardiotoxicity associated with one of its constituent agents, doxorubicin; a chemotherapy agent which belongs to the anthracycline family. The maximum life time recommended dose of doxorubicin ranges from 450mg/m² to 550mg/m² and this level is often reached during front line treatment with CHOP. There is estimated to be more than 100,000 patients with relapsed aggressive NHL in the US.
Pixantrone is an investigational drug that is being developed by Novuspharma, which was designed to potentially increase anti-tumor activity and decrease the potential for cardiac toxicity associated with the currently marketed anthracycline drugs. This trial examined the safety and potential efficacy of Pixantrone when substituted for doxorubicin in the CHOP regimen among patients who had failed prior doxorubicin containing CHOP therapy for aggressive NHL. Pixantrone’s efficacy in this context was predicted based on the synergy seen with cyclophosphamide and vincristine in preclinical studies.
Dose determination and safety results from the Pixantrone–CHOP variant trial


This phase I trial was an open-label, dose ranging study, with Pixantrone administered on day 1 of a 21 day cycle, in combination with standard fixed doses of cyclophosphamide, vincristine and prednisone. The trial’s primary objective was to identify the recommended dose of Pixantrone for subsequent studies, in addition to obtaining safety and preliminary efficacy data. The trial recruited 23 patients in total, across 4 dose levels: 80mg/m², 100mg/m², 120mg/m² and 150mg/m², the latter of which was identified as the maximum tolerated dose based on these study findings.


The majority of patients were elderly, with a median age of 67 (range 34-77) and had received a median of 3 prior regimens (range 1-9). Median prior anthracycline exposure was 535mg/m² doxorubicin, or equivalent (range 60 to 1002mg/m²). Toxicities were predominantly hematological (suppression of blood cell growth) and the dose limiting toxicity was neutropenia (low levels of white blood cells, which is commonly seen with chemotherapy treatment). No grade IV non-hematological toxicity was observed.


Cardiac side effects were infrequent. Two patients had an asymptomatic reduction in their cardiac ejection fraction (LVEF) to 20 percent below their baseline value. One patient with a history of coronary artery disease and diabetes developed Grade II angina pectoris. The low rate of cardiac events is very encouraging, considering the patients’ age and their level of pre-treatment with the traditional anthracyclines.


Efficacy results
The preliminary efficacy data indicate that Pixantrone is highly effective in the CHOP-variant regimen, with an overall response rate of 77% and disease control rate of 95%. Thirteen patients (59%) experienced a complete response (CR) or complete response unconfirmed (CRu), 4 (18%) experienced a partial response (PR) and 4 (18%) achieved stable disease (SD), out of 22 evaluable patients. Furthermore, patients responded at every Pixantrone dose level and 7 / 7 patients responded at the highest dose (150mg/m²). Patients continue to be followed for response duration.
First patient recruited in a phase II trial using the CHOP-variant regimen
Novuspharma also announces today that the first patient has been recruited in a phase II extension to this trial. This phase II is expected to recruit around 25 patients in Europe, who will receive CHOP-variant with Pixantrone at 150 mg/m². The trial’s primary objective is to obtain additional efficacy data in terms of objective response rate (CR + PR).
Professor Andreas Engert, Principal Investigator on the study commented: “The CHOP chemotherapy regimen has become standard-of-care in the treatment of front-line aggressive NHL, where it is associated with a high rate of complete remission and cure. The safety and preliminary efficacy data from this phase I/II trial suggest that substituting Pixantrone, in the place of doxorubicin in CHOP, produces a regimen that may be safely used in relapsed aggressive NHL patients. This could potentially allow relapsed patients to benefit from the high efficacy of CHOP a second time, providing them with a much needed treatment option. I look forwards to further studying Pixantrone in the CHOP-variant regimen.”  
Enquiries:
Novuspharma SpA  
Karl Hanks  Tel:     +39 335 7882247
Candice R. Douglass of CTI  Tel:     +1 206 850 6084
     
Financial Dynamics  
Sarah Macleod  Tel: +44 (0)20 7831 3113
Francetta Carr  

Notes to Editors
Novuspharma SpA and its merger agreement with Cell Therapeutics (CTI)
Novuspharma, based in Bresso, Milan, is an emerging biopharmaceutical company leveraging its expertise in the field of oncology to discover and develop innovative new treatments for cancer. It has three products in clinical development and a dynamic research program. Novuspharma was established in 1998 following the merger of Boehringer Mannheim and Hoffmann-La Roche, to exploit the R&D team’s proven track record in product development. On June 17th, 2003, Novuspharma announced it had signed a merger agreement with Cell Therapeutics (CTI) (NASDAQ CTIC) of Seattle. CTI is a public biopharmaceutical company, which markets TRISENOXâ in the US and Europe and is developing XYOTAX™ (CT-2103), which is in pivotal phase III trials for lung cancer.For further information, please visit the Company’s website at http://www.novuspharma.com/. For an explanation of technical terms please see www.novuspharma.com/nov/glossary/


Visit website http://www.novuspharma.com

10/12/2003

Geneva, Switzerland, 10 December 2003: Addex Pharmaceuticals SA, the drug development company focused on addiction and other central nervous system disorders, is pleased to announce the signing of a three year partnership with Fulcrum Pharma PLC (AIM: FUL), the drug development and strategic outsourcing services company.  The three year agreement is the first preferred supplier arrangement in which Fulcrum takes an equity stake in its partner and receives fees for service.


Under the terms of this agreement Fulcrum will be responsible for the provision of drug development resource and expertise to support Addex in the development of its product portfolio over the next 3 years and to assist Addex in building its own clinical capability.


As part of this agreement a letter of intent has been issued whereby Fulcrum will subscribe for shares in Addex’s next round of financing.


Jon Court, CEO of Fulcrum Pharma, commented:
“We are extremely impressed with Addex and their portfolio of compounds, and delighted to have been chosen as their development partner.  This agreement represents a significant step in the development of Fulcrum’s partnership strategy.”


Upon signing the agreement, Vincent Mutel, CEO of Addex Pharmaceuticals, remarked:
“This three year partnership is a great opportunity for us to solve our clinical resourcing needs and at the same time build knowledge of clinical development within Addex. In addition, we view Fulcrum’s commitment and investment as a strong validation of our portfolio.”


_____________________________________________
FOR FURTHER INFORMATION, PLEASE CONTACT :


Fulcrum Pharma PLC      
Jon Court, Chief Executive      +44 8707 107152
Geoffrey Smith, Finance Director    +44(0)8707104501 


Addex Pharmaceuticals      +41 22 737 0047
Karen Wilson
   
Buchanan Communications
Mary-Jane Johnson       +44 20 7466 5000


Seymour Pierce Limited      +44 20 7107 8000
Jonathan Wright
 
Notes to Editors:


About Fulcrum Pharma
Fulcrum Pharma plc is an independent, drug development company that is the first to offer global virtual drug development and strategic outsourcing services to the pharmaceutical industry. 


The Company has expertise in the design, execution and delivery of drug development programmes and relies on state of the art information technology and infrastructure in the supply of its services.


Through Fulcrum’s services to its clients, the Directors believe that there is the capacity to deliver products faster and more efficiently to the global pharmaceutical market. Fulcrum does this by using its skills in the design of drug programmes that deliver the necessary information for decision-making and product registration. In so doing, Fulcrum works closely with its clients to meet their strategic R&D goals.


Fulcrum Pharma is listed on the Alternative Investment Market of the London Stock Exchange having successfully floated in March 2000.



About Addex Pharmaceuticals SA
Addex Pharmaceuticals SA is a pharmaceutical company, based in Geneva, Switzerland, committed to the discovery, development and marketing of novel therapeutic compounds for the treatment of addiction and other central nervous system (CNS) disorders. The Addex team combines expertise in the field of addiction with broad experience in drug development.


The Company has specific know-how for the development of compounds which have modulatory activity on specific CNS targets.  Addex is applying this know-how to develop novel compounds that are expected to be useful for the treatment of a number of CNS disorders, including drug dependence.


Addex is backed by venture capitalists: Index Ventures, Sofinnova Partners and Techno Venture Management.


Visit website http://www.addexpharma.com

02/12/2003

The Biolipox’ Board of Directors has appointed Mr Håkan Åström as new Chairman of the Board. Biolipox is a pharmaceutical R&D company with a world-class research profile within the field of inflammatory disease, particularly asthma and allergy.
“The opportunity to get involved in the development of Biolipox is very inspiring. I chose Biolipox because the company is active in a field with extended possibilities”, says Håkan Åström. “Biolipox’ projects have great commercial potential through the collaboration with larger pharma companies”, Mr Åström continues.
Håkan Åström was previously Senior VP, Pharmacia Corporation and is presently member of the Board of both Biovitrum and Scandinavian Life Science AB.
“We are extremely pleased to see Mr Åström join the Board as Chairman. Håkan Åström has many years of experience of building financially solid companies. With a unique understanding of the pharmaceutical industry and an extensive network after a very successful career within Pharmacia Corporation, Mr Åström enters our company at a very important moment”, says Carl-Johan Dalsgaard, CEO of Biolipox. “
Biolipox was founded in 2000 by Professors Hans-Erik Claesson and Magnus Björkholm, both of the Karolinska Institute, after a seed financing by HealthCap. Based on a long tradition of world-leading research in the field of arachidonic acid, Biolipox’s current pipeline comprises drugs targeting respiratory inflammatory diseases, particularly allergic rhinitis, and asthma, and inflammatory pain.
The achievements which form the foundation for the understanding of the role of arachidonic acid in inflammatory processes were rewarded with the 1982 Nobel Prize. Professor Bengt Samuelsson, one of the Nobel Prize winners, is a member of the Board at Biolipox.
Biolipox business concept involves producing innovative pharmaceuticals which meet medical needs in inflammatory disease, primarily asthma and pain. This is achieved in collaboration with researchers all over the world. At the point where the efficaciousness of the substances is proven, Biolipox will also be entering into license agreements with the major pharmaceuticals companies on the further development, marketing and sales of the product.
For more information:
Carl-Johan Dalsgaard, CEO; mobile: +46 70 975 98 63
Håkan Åström; phone: +44 1 753 869 769, mobile: +46 703 74 72 13

Biolipox AB is a pharmaceutical R&D company, focused on creating novel, efficacious and cost effective therapeutic opportunities for respiratory conditions and other inflammatory disorders. Biolipox’ scientific platform is based on world-class arachidonic acid cascade research, performed at the Karolinska Institutet. The company has a broad portfolio of potential blockbusters in the respiratory therapy area and unique targets for novel anti-inflammatory and analgesic drugs.

Biolipox AB, Box 6280 , SE-102 34  Stockholm, Sweden.
Tel: +46-8-545 28 140, Fax: +46-8-545 28 141

Visit website http://www.orexo.com

25/11/2003

IDM (Immuno-Designed Molecules), société de biopharmacie spécialisée dans l’immunothérapie du cancer, a obtenu la délivrance d’un nouveau brevet au Japon qui protège ses produits MAK® et leur utilisation comme agent thérapeutique contre le cancer.


Les MAK constituent l’une des deux familles de produits d’immunothérapie appelés Cell Drugs développés par IDM contre le cancer. Les MAK sont composés de macrophages activés du patient, couplés dans certains cas avec des anticorps. Ils sont injectés au malade avec pour objectif d’éliminer les cellules tumorales.


Des brevets relatifs à cette approche thérapeutique ont déjà été accordés à IDM en Australie, au Canada et aux Etats-Unis.


IDM dispose depuis juin 2003 d’un premier brevet au Japon qui protège le procédé de préparation et d’utilisation de sa seconde famille de produits de thérapie cellulaire, les Dendritophages®.


Les Dendritophages sont des vaccins thérapeutiques expérimentaux contenant des cellules dendritiques du patient, chargées avec des antigènes tumoraux. Ils sont à l’étude pour évaluer leur capacité à prévenir les rechutes de certains cancers.


Le portefeuille de propriété industrielle d’IDM compte aujourd’hui 96  brevets délivrés (dont 19 aux Etats-Unis et deux au Japon) et 92 demandes de brevets en cours d’examen.


Visit website http://www.idm-biotech.com

19/11/2003

ExonHit Therapeutics identifies novel target
Paris, France… ExonHit Therapeutics announced that Allergan, Inc. (AGN), a global specialty pharmaceutical company, has initiated high throughput screening on a target generated pursuant to a 10 month old strategic alliance between the companies.  ExonHit is a privately held company that discovers neurodegenerative and cancer therapeutics and diagnostics based on alternative RNA splicing.


ExonHit has delivered to Allergan a small molecule target that shows efficacy in a previously unrecognized pathway identified utilizing DATASTM, ExonHit’s proprietary gene profiling technology.  This target will begin high throughput screening.


The strategic alliance was formed in December 2002 with the purpose of developing new drugs in the field of neurodegenerative diseases, pain and ophthalmology.  The dual goals of the agreement are to identify new molecular targets based on ExonHit Therapeutics’ gene profiling DATAS™ technology and to work collaboratively toward developing and commercializing unique compounds and commercial products based on these targets.     Les Kaplan Executive Vice President and President of R&D at Allergan stated, “I am pleased both with the productive working relationship and with the progress made to date”.


“Rapidly delivering a designated target and expecting to deliver one or two more within the next five months exemplifies the power of DATAS™ and of our ability to analyze the role of alternative RNA splicing events in disease,” commented Fabien Schweighoffer, Executive VP, Research, ExonHit Therapeutics, “I view the ExonHit approach as an engine for drug discovery and I’m pleased this approach is moving the alliance with Allergan in a promising direction and with impressive speed– it’s a process that has worked well for our diagnostic collaborations and internal therapeutic programs.”


ExonHit’s therapeutic pipeline includes a compound for Amyotrophic Lateral Sclerosis (commonly known as Charcot or Lou Gehrig’s disease) which is currently in a large Phase II clinical trial (400 patients) for evaluation of its safety and its efficacy on survival. This study is expected to be completed in the Fall 2004.  A Phase I trial is planned for a small molecule therapeutic with indications in several neurodegenerative diseases and retinitis pigmentosa.  Furthermore, two new chemical entities with anti-angiogenesis properties are in pre-clinical stages for the treatment of cancer and ocular revascularization disorders (the leading cause of blindness). 


In addition to its therapeutic programs, ExonHit has ongoing diagnostic collaborations with Roche Applied Sciences and bioMerieux.  Internal diagnostic projects include a prostate cancer diagnostic test, based on alternatively spliced isoforms of PSA and KLK2, and a blood-based diagnostic test for colon cancer.   ExonHit intends to out-license both tests in the near future.  



Forward-Looking Statements


This press release contains “forward-looking statements”, such as the statements of Dr. Kaplan, and Dr. Schweighoffer, and the parties’ goals of developing drug compounds and commercial products.  These statements are based on current expectations of future events.  If underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could vary materially from the parties’ expectations and projections. Risks and uncertainties include general industry and pharmaceutical market conditions; general domestic and international economic conditions; technological advances and patents attained by competitors; challenges inherent in new product marketing such as the unpredictability of market acceptance for a new pharmaceutical product or approved indication; potential difficulties in manufacturing a new product formulation; domestic and foreign health care reforms; and trends toward managed care and health care cost containment; and governmental laws and regulations affecting domestic and foreign operations.

About ExonHit Therapeutics

ExonHit Therapeutics is a privately-held company whose mission is to develop novel therapeutics and diagnostics for neurodegenerative diseases and cancer.  The company is a leader in alternative RNA splicing holding a premier scientific and intellectual property position in the field.  


Utilizing DATASTM, the company’s proprietary gene profiling technology, allows the detection of disease-relevant changes in mRNA populations resulting from alternative RNA splicing events, providing key insight into biological causes of pathology. DATASTM stands for: Differential Analysis of Transcripts with Alternative Splicing. 


DATASTM is central to internal drug discovery programs but the profiling technology also has broad applications.  ExonHit has several external therapeutic and diagnostic collaborators, and has used the technology to capitalize on the growing needs for pharmacogenomic tools, and toxicity prediction assays. 


ExonHit Therapeutics, founded in 1997, is headquartered in Paris, France and has a US facility in Gaithersburg, Maryland. The company has 70 employees worldwide and has raised a total of €47 million.



About Allergan, Inc.


Allergan, Inc., with headquarters in Irvine, California, is a technology-driven, global health care company providing eye ca


Visit website http://www.exonhit.com