Thursday, March 29, 2012

Eye on THR, raises EUR77.8 Million

Ophthalmic medicine firm ThromboGenics raises funds through a private placement. The funds will be used, in part, to execute US launch and commercialization of ocriplasmin, its pharmacological treatment for symptomaticVitreomacular Adhesion (VMA) including macular hole.
Full press release below.
Source: ThromboGenics NV
     
ThromboGenics Completes Private Placement Raising EUR77.8 Million
LEUVEN, Belgium, March 29, 2012/PRNewswire-FirstCall/ -- 
ThromboGenics NV (NYSE Euronext Brussels: THR), a biopharmaceutical company focused on developing innovative ophthalmic medicines, announces today that it has raised EUR77.8 million through a private placement via an accelerated bookbuilding procedure announced on Wednesday 28 March 2012 after the market closed. 
The private placement has allowed ThromboGenics to place 3,244,675 new shares with a range of domestic and international investors and qualified institutional buyers in the United States at a price of EUR24 per share, a 3.5% discount on the previous closing price. This represents 9.99% of the current number of outstanding shares and will bring the total number of shares after the issue to 35,691,432. The new shares will be admitted to listing on NYSE Euronext Brussels following their issuance, which is expected to take place on 3 April 2012. 
The funds will be used by ThromboGenics to: 
    
    - execute the U.S. launch and commercialization of ocriplasmin, potentially
      the first pharmacological treatment for symptomatic Vitreomacular Adhesion (VMA)
      including macular hole;
    - fund the clinical development of ocriplasmin for additional indications on a
      50:50 basis with Alcon, its commercialization partner for non U.S. markets;
    - strengthen its core ophthalmology franchise by in-licensing development-stage
      product candidates; and
    - for general corporate purposes.

KBC Securities acted as Global Coordinator. KBC Securities, BofA Merrill Lynch and Petercam acted as Joint Bookrunners for the placing. 
Dr Patrik De Haes, CEO of ThromboGenics said: "I would like to welcome new investors who participated in this over-subscribed placement, and thank existing shareholders for their continued support of the Company and our ophthalmic-focused strategy. The additional funds that we have raised will allow us to build a first-class U.S. commercial organization ahead of the anticipated launch of ocriplasmin. The potential of ocriplasmin to provide a new treatment paradigm for patients with symptomatic VMA including macular hole has been validated by our recent agreement with Alcon, giving it the commercial rights to this innovative product outside the U.S. The proceeds should also enable us to become a profitable, ophthalmology company that both creates value for our shareholders and brings drugs with significant benefits to patients." 
About ThromboGenics 
ThromboGenics is a biopharmaceutical company focused on developing and commercializing innovative ophthalmic medicines. The Company's lead product, ocriplasmin, has successfully completed two Phase III clinical trials for the pharmacological treatment of symptomatic Vitreomacular Adhesion (VMA). The MAA for ocriplasmin has been accepted for review in Europe and the BLA will be re-submitted in the U.S. by April 2012. Ocriplasmin is in Phase II clinical development for additional vitreoretinal conditions. 
In March 2012, ThromboGenics signed a strategic partnership with Alcon (Novartis) for the commercialization of ocriplasmin outside the United States. Under this agreement, ThromboGenics could receive up to a total of EUR375 million in up-front and milestone payments, plus an attractive level of royalties on Alcon's net sales of ocriplasmin. ThromboGenics and Alcon intend to share the costs equally of developing ocriplasmin for a number of new vireoretinal indications. 
ThromboGenics is also developing two novel antibody therapeutics in collaboration with BioInvent International. These are TB-402 (anti-Factor VIII), a long-acting anticoagulant in Phase II, and TB-403 (anti-PlGF), in Phase Ib/II for cancer which has been partnered with Roche. 
ThromboGenics is headquartered in Leuven, Belgium. The Company is listed on the NYSE Euronext Brussels exchange under the symbol THR. More information is available at http://www.thrombogenics.com. 
Important information about forward-looking statements 
This press release is for information purposes only and is not intended to constitute, and should not be construed as, an offer to sell or a solicitation of any offer to buy shares in the share capital of ThromboGenics NV (the "Company"). This announcement has been issued by and is the sole responsibility of the Company. 
This announcement is not for publication or distribution, directly or indirectly, in or into the United States, Australia, Canada, Japan or any other state or jurisdiction into which the same would be unlawful. The distribution of this announcement may be restricted by law in certain jurisdictions and persons into whose possession any document or other information referred to herein comes should inform themselves about and observe any such restriction. Any failure to comply with these restrictions may constitute a violation of the securities laws of any such jurisdictions. 
Certain statements in this press release may be considered "forward-looking". Such forward-looking statements are based on current expectations, and, accordingly, entail and are influenced by various risks and uncertainties. These forward-looking statements may be identified by references to strategy, plans, objectives, goals, future events or intentions. Forward-looking statements may and often do differ materially from actual results. The Company therefore cannot provide any assurance that such forward-looking statements will materialize. Additional information concerning risks and uncertainties affecting the business and other factors that could cause actual results to differ materially from any forward-looking statement is contained in the Company's Annual Report. Forward-looking statements speak only as of the date they are made. Each of the Company, KBC Securities NV, Merrill Lynch International and Petercam NV and their respective affiliates expressly disclaims any obligation or undertaking to update, review or revise any forward looking statement contained in this announcement whether as a result of new information, future developments or otherwise. 
Important notices regarding the placing 
This press release does not constitute an offer to sell or the solicitation of an offer to buy any securities of the Company, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale is not permitted or to any person or entity to whom it is unlawful to make such offer, solicitation or sale. This press release is not an offer of securities for sale into the United States, Canada, Australia or Japan or in any jurisdiction in which such offer or solicitation is unlawful. 
The new shares have not been and will not be registered under the United States Securities Act of 1933, as amended (the "US Securities Act") or with any securities regulatory authority of any state or other jurisdiction of the United States and may not be offered or sold in the United States except pursuant to registration or an exemption from the registration requirements of the Securities Act. There will be no public offering of securities in the United States. 
KBC Securities NV, Merrill Lynch International and Petercam NV, (the "Bookrunners") are acting exclusively for the Company and no-one else in connection with the placing. In addition certain of the Bookrunners or their affiliates may enter into financing arrangements and swaps with investors in connection with which such Bookrunners (or their affiliates) may from time to time acquire, hold or dispose of shares of the Company. They will not regard any other person as their respective clients in relation to the placing and will not be responsible to anyone other than the Company for providing the protections afforded to their respective clients, nor for providing advice in relation to the placing, the contents of this announcement or any transaction, arrangement or other matter referred to herein. 
None of KBC Securities NV, Merrill Lynch International and Petercam NV or any of their respective directors, officers, employees, advisers or agents accepts any responsibility or liability whatsoever for or makes any representation or warranty, express or implied, as to the truth, accuracy or completeness of the information in this announcement (or whether any information has been omitted from the announcement) or any other information relating to the Company, its subsidiaries or associated companies, whether written, oral or in a visual or electronic form, and howsoever transmitted or made available or for any loss howsoever arising from any use of this announcement or its contents or otherwise arising in connection therewith. 
This communication is being distributed only to, and is directed only at (a) persons outside the United Kingdom, (b) persons who have professional experience in matters relating to investments falling within Article 19(5) of the Financial Services and Markets Act 2000 (Financial Promotion) Order 2005, as amended (the "Order"), and (c) high net worth entities, and other persons to whom it may otherwise lawfully be communicated, falling within Article 49(2) of the Order (all such persons together being referred to as "relevant persons"). The securities referred to herein are only available to, and any invitation, offer or agreement to subscribe, purchase or otherwise acquire such securities will be engaged in only with relevant persons. Any person who is not a relevant person should not act or rely on this communication or any of its contents. 
In relation to each member state of the European Economic Area which has implemented the Prospectus Directive (as defined below) (each, a "Relevant Member State"), an offer to the public of any shares which are the subject of the placing may not be made in that Relevant Member State, except that an offer to the public in that Relevant Member State of any shares may be made at any time under the following exemptions under the Prospectus Directive, if they have been implemented in the Relevant Member State: (i) to qualified investors (as defined in the Prospectus Directive or implementing legislation in the Relevant Member State) ("Qualified Investors"); or (ii) to fewer than 100 natural or legal persons (other than Qualified Investors); or (iii) in any other circumstances which do not require the publication by the Company of a prospectus pursuant to Article 3 of the Prospectus Directive. For the purposes of this provision and when used elsewhere in this announcement, the expression an "offer of shares to the public", or any similar expression, in relation to any shares in any Relevant Member State means the communication in any form and by any means of sufficient information on the terms of the placing and the shares to be offered so as to enable an investor to decide to purchase or subscribe for the shares, as the same may be varied in that Member State by any measure implementing the Prospectus Directive in that Member State and the expression "Prospectus Directive" when used in this announcement means Directives 2003/71/EC and 2010/73/EC and includes any relevant implementing measure in each Relevant Member State. 
For further information please contact: 
    
    ThromboGenics NV
    Dr. Patrik De Haes, CEO
    Tel: +32-16-75-13-10
    patrik.dehaes@thrombogenics.com

    Chris Buyse, CFO
    Tel: +32-16-75-13-10
    chris.buyse@thrombogenics.com

    Citigate Dewe Rogerson
    David Dible/ Nina Enegren/ Sita Shah
    Tel: +44(0)207-638-9571
    nina.enegren@citigatedr.co.uk

Source: ThromboGenics NV




Tuesday, March 27, 2012

NBIX Phase III Trials on Track to Commence in 2Q2012

Neurocrine Biosciences (NASDAQ: NBIX), a development-stage biotech firm, develops treatments for neurological and endocrine hormone-related diseases, such as depression, insomnia, and menstrual pain. Its lead drug candidate, Elagolix, is designed to treat endometriosis. The firm's other therapeutic sectors including diabetes, movement disorders and anxiety. 
Full press release about pending Phase III Trials below.

SOURCE Neurocrine Biosciences, Inc.
Elagolix Endometriosis Phase III Trials on Track to Commence in Second Quarter 2012



SAN DIEGO, March 27, 2012 /PRNewswire/ -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) today announced that the United States Food and Drug Administration (FDA) has responded to the Special Protocol Assessment (SPA) filed with the FDA on the design of the Phase III program for elagolix in endometriosis. The FDA comments on the February 2012 SPA filing will be incorporated into the final pivotal trial designs. More importantly, the comments from the FDA are not expected to have an impact on the start of the Phase III clinical program of elagolix for endometriosis which is planned to begin during the second quarter of 2012.


"We are pleased with the FDA's response to the SPA, and are looking forward to the start of the Phase III program next quarter," said Kevin C. Gorman, President and Chief Executive Officer of Neurocrine Biosciences.



Endometriosis is associated with a multitude of symptoms, some of the most common of which include pain related both to menstruation (dysmenorrhea) as well as chronic pelvic pain throughout the menstrual cycle, and infertility. The World Endometriosis Research Foundation estimates that there are over 170 million women worldwide who suffer from endometriosis. 
About Neurocrine Biosciences
Neurocrine Biosciences, Inc. is a biopharmaceutical company focused on neurological and endocrine diseases and disorders. Our product candidates address some of the largest pharmaceutical markets in the world, including endometriosis, tardive dyskinesia, uterine fibroids, stress-related disorders, pain, diabetes, insomnia, and other neurological and endocrine-related diseases and disorders. Neurocrine Biosciences, Inc. news releases are available through the Company's website via the internet at http://www.neurocrine.com.
In addition to historical facts, this press release may contain forward-looking statements that involve a number of risks and uncertainties. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with Neurocrine's business and finances in general, as well as risks and uncertainties associated with the Company's GnRH program and Company overall. Specifically, the risks and uncertainties the Company faces with respect to the Company's GnRH program include, but are not limited to, risk that elagolix Phase III clinical trials will be delayed or not successfully initiated; risk that elagolix Phase III clinical trials will fail to demonstrate that elagolix is safe and effective for the treatment of endometriosis; risk associated with the Company's dependence on corporate collaborators for clinical development, commercial manufacturing and marketing and sales activities. With respect to its pipeline overall, the Company faces risk that it will be unable to raise additional funding required to complete development of all of its product candidates; risk relating to the Company's dependence on contract manufacturers for clinical drug supply; risks associated with the Company's dependence on corporate collaborators for commercial manufacturing and marketing and sales activities; uncertainties relating to patent protection and intellectual property rights of third parties; risks and uncertainties relating to competitive products and technological changes that may limit demand for the Company's products; and the other risks described in the Company's report on Form 10-K for the year ended December 31, 2011. Neurocrine undertakes no obligation to update the statements contained in this press release after the date hereof.
SOURCE Neurocrine Biosciences, Inc.


CONTACT: Investor Relations of Neurocrine Biosciences, +1-858-617-7600

Monday, March 26, 2012

VRX Agrees to Acquire Natur Produkt in Russia

Valeant Pharmaceuticals NYSE: VRX and TSX: VRX) develops and sells CNS (central nervous system) therapies, including Wellbutrin XL, an extended-release version of GlaxoSmithKline's antidepressant Wellbutrin; and Huntington's disease treatment Xenazine. The firm also makes Ultram ER, a long-acting version of J&J's chronic pain drug Ultram. Other products include acne treatment Acanya, anti-hypertensive Tiazac; diabetes drug Glumetza, and genital herpes treatment Zovirax. 
Full press release below about its agreement to acquire Natur Produkt in Russia is below.
Source:  http://www.valeant.com 

Valeant Pharmaceuticals Agrees to Acquire Natur Produkt in Russia

MISSISSAUGA, OntarioMarch 26, 2012 /PRNewswire/ -- Valeant Pharmaceuticals International, Inc. (NYSE: VRX and TSX: VRX) announced today that Valeant has signed an agreement to acquire Natur Produkt International, JSC, a specialty pharmaceutical company in Russia.  Natur Produkt has a significant presence in the over-the-counter (OTC) segment in several categories marketed under the umbrella brand, Natur Produkt.  Key brands include AntiGrippin, a leading cough and cold remedy, Anti Angin, Sage and Eucaplyptus MA brand names, leading sore throat remedies.
Valeant will acquire Natur Produkt for approximately $180 million, with an additional $5 million in potential future milestones.  Total revenue in 2011 was approximately $65 million and is expected to deliver double digit growth in 2012.  The transaction is expected to close by mid-year 2012, subject to certain closing adjustments and regulatory approvals, and is expected to be immediately accretive.
"We are pleased to add a leading consumer franchise to our growing operations in Russia," stated J. Michael Pearson, chairman and chief executive officer.  "Not only is the OTC market very attractive inRussia due to the self-pay element, but it is also one of the fastest growth components of the Russian healthcare system.  The addition of this high-growth operation, along with our previously announced acquisition of certain assets from Gerot Lannach, will bring our pro forma revenues in Russia to approximately $175 million by the end of 2012."
About Valeant Pharmaceuticals International, Inc.
Valeant Pharmaceuticals International, Inc. (NYSE/TSX: VRX) is a multinational specialty pharmaceutical company that develops, manufactures and markets a broad range of pharmaceutical products primarily in the areas of neurology, dermatology and branded generics.  More information about Valeant Pharmaceuticals International, Inc. can be found at www.valeant.com.
Caution Regarding Forward-Looking Information
To the extent any statements made in this document contain information that is not historical, these statements are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and may be forward-looking information as defined under applicable Canadian securities legislation (collectively, "forward-looking statements").
These forward-looking statements relate to, among other things, the closing of the acquisition of Natur Produkt by Valeant, the impact of Natur Produkt's business on Valeant's Russian portfolio, Valeant's growth initiatives and the expected timing of the acquisition to be accretive.  Forward-looking statements can generally be identified by the use of words such as "believe", "anticipate", "expect", "estimate", "intend", "continue", "plan", "project", "will", "may", "should", "could", "would", "target", "potential" and other similar expressions.  In addition, any statements that refer to expectations, projections or other characterizations of future events or circumstances are forward-looking statements.  Actual results may differ materially from those expressed or implied in such statements.  Important factors that could cause actual results to differ materially from these expectations include, among other things, the closing of the acquisition of Natur Produkt by Valeant, the impact of Natur Produkt's business on Valeant's Russian portfolio, Valeant's growth initiatives and the expected timing of the acquisition to be accretive, and the risk factors as detailed from time to time in Valeant's reports filed with the Securities and Exchange Commission ("SEC") and the Canadian Securities Administrators ("CSA"). 
Contact Information: Laurie W. Little
949-461-6002
laurie.little@valeant.com
SOURCE Valeant Pharmaceuticals International, Inc.

Electrical Pulse Tx Gives Pancreatic Cancer Patients New Hope

Electrical Pulse Treatment Gives Pancreatic Cancer Patients New Hope


Innovative Interventional Radiology Procedure Zaps Around Complicated Pancreatic Tumors, Improving Prognosis and Pancreatic Cancer Treatment Paradigm. Press release is below.
Source: www.SIRweb.org.
SAN FRANCISCOMarch 26, 2012 /PRNewswire-USNewswire/ -- Results of a study presented at the Society of Interventional Radiology's 37th Annual Scientific Meeting in San Francisco, Calif., signal a light at the end of the tunnel for individuals with inoperable locally advanced pancreatic cancer. A new procedure called irreversible electroporation or IRE uses microsecond electrical pulses to force open and destroy tumor cells around a vast and delicate network of blood vessels of the pancreas. The technique has been successful in treating primary and metastatic liver cancer, and IRE is now in the first stages of implementation as a treatment for pancreatic cancer.
"We think in another 15 to 24 months we will have a lot more evidence to support the use of irreversible electroporation for inoperable pancreatic cancer patients," said Govindarajan Narayanan, M.D., chief of vascular and interventional radiology, associate professor of clinical radiology and program director for the vascular interventional radiology fellowship at the University of Miami's Miller School of Medicine inMiami, Fla. "If we continue to get good results, this procedure could provide a huge benefit for people who honestly don't have a lot of choices. It could potentially change the rules of how these cases are managed," he added.
Pancreatic tumors are notoriously difficult to treat because any method that uses heat or cold to remove the cancer comes with too much risk of collateral damage to important blood vessels in and around the organ. IRE involves guiding electrode needles into the tumor, which damages cancer cells with a series of jolts of localized high-voltage electricity that break open the cell membranes—effectively killing the cancerous tissues around these blood vessels.
"People with locally advanced pancreatic cancer don't have a lot of treatment choices available. Irreversible electroporation gives these individuals a new treatment option and a potentially greater chance of survival," said Narayanan. "Without IRE, these individuals are essentially left with chemotherapy and radiation therapy. At this point, the prognosis for this group is pretty dismal and they have an estimated survival of less than one year. With this procedure, there is the potential to change the inoperable status in some of these patients and make them surgical candidates, he added.
An estimated 44,000 people will be diagnosed with pancreatic cancer and approximately 37,390 people will die of the disease this year, according to the American Cancer Society. About one in 71 people are expected to develop pancreatic cancer in their lifetime.
For this study, researchers gathered records for eight patients with locally advanced pancreatic cancer referred for percutaneous irreversible electroporation. All subjects had the procedure performed betweenDecember 2010 and September 2011. Pancreatic tumors were found to be between 2.5 and 6.8 centimeters in size, and each participant had formerly received a median of two other methods of treatment. A median span of time of about 9 months took place between diagnosis and treatment with IRE. Participants underwent post-procedure CT imaging to gauge the effectiveness of treatment. Out of the original eight inoperable individuals, two went on to have surgery. Both had successful resections and remain cancer-free after months following treatment.
IRE is performed with a technology dubbed NanoKnife, which has received clearance from the U.S. Food and Drug Administration for the surgical ablation of soft tissue.
"As we move forward there will be a lot of new technology and techniques like this that will help shape cancer care," said Narayanan.
More information about the Society of Interventional Radiology, interventional radiologists and minimally invasive treatments can be found online at www.SIRweb.org.
Abstract 8: "Downstaging Locally Advanced Pancreatic Adenocarcinoma (LAPC) With Vascular Encasement Using Percutaneous Irreversible Electroporation (IRE)," G. Narayanan, G. Arora , K.J. Barbery, T. Froud, J. Yrizarry, radiology, division of vascular/interventional radiology, University of Miami,Miami, Fla.; A. Livingstone, D. Franceschi, surgery, division of surgical oncology, University of Miami,Miami, Fla.; P. Hosein, C.M. Rocha Lima, medicine, division of hematology/oncology, University of MiamiMiami, Fla., SIR 37th Annual Scientific Meeting, March 24–29, 2012. This abstract can be found at www.JVIR.org.
About the Society of Interventional RadiologyInterventional radiologists are physicians who specialize in minimally invasive, targeted treatments. They offer the most in-depth knowledge of the least invasive treatments available coupled with diagnostic and clinical experience across all specialties. They use X-ray, MRI and other imaging to advance a catheter in the body, such as in an artery, to treat at the source of the disease internally. As the inventors of angioplasty and the catheter-delivered stent, which were first used in the legs to treat peripheral arterial disease, interventional radiologists pioneered minimally invasive modern medicine. Today, interventional oncology is a growing specialty area of interventional radiology. Interventional radiologists can deliver treatments for cancer directly to the tumor without significant side effects or damage to nearby normal tissue.
Many conditions that once required surgery can be treated less invasively by interventional radiologists. Interventional radiology treatments offer less risk, less pain and less recovery time compared to open surgery. Visit www.SIRweb.org.
The Society of Interventional Radiology is holding its 37th Annual Scientific Meeting March 24–29 at Moscone Center, San Francisco, Calif. The theme of the meeting is "IR Evidence," chosen to reflect interventional radiology's gathering, presenting and discussing results of care-changing investigations.




SOURCE Society of Interventional Radiology
 Local interviews and medical illustrations are available by contacting SIR's communications department staff: Ellen Acconcia, SIR communications manager/practice areas, eacconcia@SIRweb.org, (703) 460-5582, or Maryann Verrillo, SIR director of communications and public relations, mverrillo@SIRweb.org, (703) 460-5572.CONTACT: Ellen Acconcia, +1-703-460-5582, Maryann Verrillo, +1-703-460-5572, Gabrielle Lee (March 22-23), +1-212-453-2137, Gabrielle Lee (March 24), +1-225-266-0953, or the San Francisco Press Room (March 24-28), +1-415-978-3502, +1-415-978-3503, +1-415-978-3504, comm@SIRweb.org
Web Site: http://www.sirweb.org 

Wednesday, March 21, 2012

Healthcare IT News


U.S. Preventive Medicine aims to prevent huge health issues before they occur. Through its Prevention Plan, a cloud-based wellness platform, and its Macaw mobile app, U.S. Preventive Medicine enables individuals to determine their top health risks and receive a customized plan and coaching to lower those risks and become healthier. The company is accredited in wellness and health promotion by NCQA and disease management by URAC.  

Full press release about a new marketing agreement is below.

U.S. Preventive Medicine Announces Strategic Development and Marketing Agreement with Dossia
JACKSONVILLE, Fla. and DALLAS, March 21, 2012 /PRNewswire/ -- U.S. Preventive Medicine, Inc. today announced a strategic development and marketing agreement with Dossia Service Corporation, to offer The Prevention Plan™ - U.S. Preventive Medicine Inc.'s clinically-proven, cloud-based wellness platform – to employees of some of the world's largest and most respected companies through the Dossia Health Management System.
The Dossia Health Management System automatically aggregates users' information from a variety of sources including physicians, hospitals, health plans, labs, pharmacies, integrated home health devices and partner organizations, and securely stores the data in one location that is private, consumer controlled, portable, secure and lifelong. Dossia then integrates this aggregated personal health information with best in class health and healthcare tools, as well as multiple user engagement components to offer a rich and personalized experience. The goal is to enable a more engaged, informed, empowered - and ultimately healthier - consumer. 
With this agreement, employees of Dossia's member companies will be able to purchase an annual subscription to The Prevention Plan through the Dossia Marketplace with all results of the preventive medicine program being integrated into their Dossia Health Manager.
The Prevention Plan includes a comprehensive online Health Risk Appraisal, a basic lab panel including lipid profile and blood glucose, a web-enabled Prevention Plan, and a customized Report that highlights the consumer's top health risks and provides a personalized, step-by-step plan with activities and programs to reduce those risks. 
Dossia members will also receive unlimited access to the Prevention Plan's online tools and resources, discounts to Prevention Partner services, a dynamic Prevention Score(SM) that increases as the member take steps to improve his or her health, and access to Macaw™ The Prevention Plan-enabled smart phone app that syncs with the Prevention Plan as well as other mobile apps and wireless devices to serve as a complete personal health monitor.
"U.S. Preventive Medicine shares Dossia's founding principles of empowering individuals to make smarter, more proactive decisions about their health and we are honored to be part of Dossia's growing network of best-of-class health solutions. Together, we can leverage the power of prevention to help reduce the burden of risk and illness to society," said Dr. Ron Loeppke, M.D., Vice Chairman of U.S. Preventive Medicine. 
According to Mike Critelli, CEO of Dossia, "Having access to all of this personal health information in one place allows users to be more vigilant about the accuracy of their health records, more proactive about the daily lifestyle habits that impact their overall well-being, and better able to reduce health care costs from unnecessary usage and duplication of services. The Prevention Plan, with its clinically-proven preventive medicine platform will offer significant value to our Dossia Health Management Members."
About U.S. Preventive Medicine®  
U.S. Preventive Medicine is leading a global preventive health movement focused on saving lives and money by keeping people healthy and better managing chronic conditions before they progress through innovative products like The Prevention Plan and Macaw mobile app. The company is accredited in wellness and health promotion by NCQA and disease management by URAC.  
About Dossia 
Dossia is a non-profit organization consisting of several large U.S. employers who have united under a common vision: to empower their employees to make smarter more informed decisions about their healthcare. Through Dossia, they will leverage their combined influence to break down barriers to health information, which will help drive consumer-initiated change. The Dossia Founders group includes AT&T, Applied Materials, BP America, Cardinal Health, Intel, NantWorks, Pitney Bowes, sanofi-aventis, Vanguard Health Systems and Walmart. For more information, visit www.dossia.org.
SOURCE U.S. Preventive Medicine

Tuesday, March 20, 2012

TACT believes HT-100 has potential


HT-100 for Duchenne muscular dystrophy has been reviewed by the TREAT-NMD Advisory Committee on Therapeutics (TACT), who believes the compound has potential. The disease is rare, with only around 20,000 new cases a year, and its journey to get to this stage has been an innovative case study in the emerging phenomena of the new model of do-it-yourself drug development, i.e., the joint efforts by two families, guided by expert advice, to buy and develop a drug that holds promise in treating Duchenne muscular dystrophy. For the complete drug development story, see link
http://rareproject.org/2011/12/27/wall-street-journal-reports-do-it-yourself-drug-development-with-some-help/
Full press release is below.
Source: www.halotherapeutics.com
Halo Therapeutics Reports Favorable Independent Review of Lead Drug Candidate HT-100
International Advisory Committee of Neuromuscular Disease Experts Finds HT-100 "Ready for the Clinic" in Voluntary Review
NEWTON, Mass., March 20, 2012 /PRNewswire/ -- Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, announced today that the TREAT-NMD Advisory Committee on Therapeutics (TACT) has reviewed HT-100, Halo's drug candidate for Duchenne muscular dystrophy (DMD), and believes the compound has potential based on the proposed mechanism. TREAT-NMD is a global network dedicated to expediting the delivery of promising new therapies to patients with neuromuscular disease. TACT provides independent, objective guidance on the therapeutic potential of drug candidates (novel or repurposed), which are submitted for review on a voluntary basis. In its review of HT-100, TACT found the drug candidate to be "ready for the clinic."
"TACT offers the opportunity for objective review of potential new therapies to help industry, researchers and patient advocacy groups. The review provides multi-disciplinary advice on the development of the potential therapies in the context of a realistic development pathway," said Volker Straub of TREAT-NMD. "TACT evaluated the proposal from Halo Therapeutics and felt it was well prepared, and that the compound has potential based on the proposed mechanism. Overall the plan to progress to a clinical trial is realistic."
DMD is a progressive and fatal neuromuscular disorder that afflicts approximately 1 in 3,500 boys worldwide.  HT-100, Halo's proprietary formulation of halofuginone, is an orally available small molecule drug candidate intended to reduce fibrosis and promote healthy muscle fiber regeneration. HT-100 works by inhibiting the pathological fibrotic process in muscle and directly stimulating healthy muscle fiber regeneration. The U.S. Food and Drug Administration recently granted orphan drug designation to HT-100. The company will begin a phase 2 study in the second half of 2012.
"We submitted HT-100 for a TACT review knowing that informed, objective feedback on our pre-clinical data and development plans would be invaluable to our efforts," said Marc Blaustein, CEO of Halo Therapeutics. "We believe their recommendations for HT-100's advancement will hasten our progress toward delivering a safe and effective new therapy for DMD patients and their families."
A brief summary of TACT's review of HT-100 can be found at http://www.treat-nmd.eu/resources/tact/reviews/past/halofuginone-ht-100/.
About Duchenne Muscular Dystrophy    
Duchenne muscular dystrophy is caused by a mutation in the dystrophin gene, resulting in progressive muscle weakness. The disease manifests itself first in weakened skeletal muscles and eventually results in cardiac and pulmonary impairment. Corticosteroids are the current standard of care treatment for DMD.  While this treatment delays disease progression by several years, their prolonged use is typically associated with side effects and the treatment does not alter the ultimate outcome of the disease. Young men with Duchenne typically live into their twenties or early thirties.
About TREAT-NMD 
TREAT-NMD is a neuromuscular network that has developed the infrastructure to ensure that promising therapies reach patients as quickly as possible. TREAT-NMD was funded as a network of excellence by the EU with the FP6 programme (contract number: 036825) from January 2007 – December 2011.  Since its launch the network's focus has been on the development of tools that industry, clinicians and scientists need in order to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice clinical care for neuromuscular patients worldwide.
About Halo Therapeutics 
Based in Newton, Mass., Halo Therapeutics, LLC, is a clinical-stage biopharmaceutical firm focused on developing innovative therapies for rare fibrotic diseases like Duchenne muscular dystrophy. Founded through a collaboration between patient advocacy organizations and industry veterans, the company is partnering with the clinical community and patients to transform therapeutic options and, by extension, lives. For more information, please visit www.halotherapeutics.com.

Media Contacts

Name:
Marc B. Blaustein
Company:
Halo Therapeutics, LLC
Phone Number:
617.431.7250


Monday, March 19, 2012

RNAi therapeutics firm commencing Phase I



Alnylam’s RNAi drug delivery holds the promise of becoming the new face of disease control and treatment.  Alnylam, by making investments and fending off lawsuits and bad press, has positioned itself as a frontrunner in this category.  Alnylam's RNAi drug delivery works like this: it “runs interference” with RNA to prevent disease, by selectively shutting off harmful genes. Disease targets include genetic disorder ATTR (transthyretin-mediated amyloidosis) and RSV (respiratory syncytial virus), a pediatric viral infection. Its other R&D programs target oncology, cardiovascular and neurological disorders. Press release about Phase I clinical trial commencement with ALN-TTR02 is below.


Source: www.alnylam,com; date released: March 19, 2012
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). The study is aimed at evaluating the safety and tolerability of ALN-TTR02 in healthy volunteers. In addition, the study will evaluate the clinical activity of ALN-TTR02 based on measurements of serum levels of TTR, the disease-causing protein in patients with ATTR. ALN-TTR02 utilizes the company’s proprietary second-generation lipid nanoparticle (LNP) technology using the “MC3” lipid. Alnylam expects to present data from this study in the third quarter of 2012.
“ALN-TTR02 utilizes our proprietary second-generation LNP technology which has shown significant potency improvements in both pre-clinical and clinical studies. We very much look forward to our expected reporting of TTR knockdown data from the Phase I study in the third quarter of this year.”
“Advancement of ALN-TTR02 into the clinic is an important milestone in our ‘Alnylam 5x15’ product strategy. Indeed, ALN-TTR02 is our lead program in this effort and we expect to initiate a Phase II trial later this year, followed by a pivotal trial starting in 2013,” said Akshay K. Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. “ALN-TTR02 utilizes our proprietary second-generation LNP technology which has shown significant potency improvements in both pre-clinical and clinical studies. We very much look forward to our expected reporting of TTR knockdown data from the Phase I study in the third quarter of this year.”
“RNAi therapeutics represent a novel and exciting approach for the treatment of ATTR, as this new drug modality has the potential to make a meaningful impact in the treatment of this devastating disease. I am very encouraged by the recent results from Alnylam’s Phase I study with ALN-TTR01 which showed knockdown in serum levels of TTR, the pathogenic protein in this disease. These results are important because we believe TTR suppression has the potential of halting or even reversing disease pathology in ATTR patients,” said Teresa Coelho, M.D., Director, Unidade Clinica de Paramiloidose. “I support the continued advancement of this innovative medicine, and look forward to results from this new study, as well as additional clinical studies.”
The Phase I trial of ALN-TTR02 is being conducted in the U.K. as a randomized, single-blind, placebo-controlled, single-ascending dose study, enrolling approximately 32 healthy volunteer subjects. The primary objective of the study is to evaluate the safety and tolerability of a single dose of ALN-TTR02, with subjects being enrolled into five sequential cohorts of increasing doses ranging from 0.01 to 0.50 mg/kg. Secondary objectives include serial measurement of circulating TTR serum levels through at least day 56 following a single dose. Additional secondary objectives include plasma and urine pharmacokinetics of ALN-TTR02.
About Transthyretin-Mediated Amyloidosis
Transthyretin (TTR)-mediated amyloidosis (ATTR) is a hereditary, systemic disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier for thyroid hormones and retinol binding proteins. Mutations in TTR cause abnormal amyloid proteins to accumulate and damage body organs and tissue such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy. In its severest form, ATTR represents a major unmet medical need with significant morbidity and mortality as an orphan disease; FAP (familial amyloidotic polyneuropathy) affects approximately 10,000 people worldwide and FAC (familial amyloidotic cardiomyopathy) affects at least 40,000 people worldwide. ATTR patients with FAP have a mean life expectancy of five to 15 years from symptom onset and the only treatment option is liver transplantation; as a result there is a significant need for novel therapeutics to treat patients who have inherited mutations in the TTR gene.
About ALN-TTR Program
ALN-TTR is a systemically delivered RNAi therapeutic being developed for the treatment of ATTR. Pre-clinical studies have shown that treatment with Alnylam’s first-generation product candidate, ALN-TTR01, resulted in both prevention and regression of pathogenic TTR deposits in peripheral tissues including dorsal root ganglia, sciatic nerve, stomach, and intestines in animal models. In November 2011, Alnylam reported positive preliminary clinical results from its ALN-TTR01 Phase I, multinational clinical trial showing that ALN-TTR01 was generally safe and well tolerated and resulted in statistically significant lowering of TTR serum levels in ATTR patients. Alnylam has completed enrollment in the ALN-TTR01 Phase I trial and expects to present final data in the first half of 2012. In March 2012, Alnylam initiated a Phase I clinical trial with ALN-TTR02, its second-generation ATTR therapeutic candidate which utilizes an improved second-generation LNP formulation with the “MC3” lipid. In pre-clinical studies, this second-generation LNP technology demonstrated an over 10-fold improvement in potency. Data from the ALN-TTR02 Phase I trial are expected in the third quarter of 2012. In addition, Alnylam plans to start a Phase II multi-dose clinical study of ALN-TTR02 in ATTR patients in the second half of 2012 and, assuming positive results, expects to initiate a pivotal trial with ALN-TTR02 in 2013. Alnylam is also advancing ALN-TTRsc, which utilizes a GalNAc-conjugate delivery approach and subcutaneous dose administration. Alnylam plans to file an investigational new drug (IND) application or IND equivalent for ALN-TTRsc in the second half of 2012, with data expected in the first half of 2013. ALN-TTRsc has the potential to provide product differentiation and expansion in the ATTR indication.
About RNA Interference (RNAi)
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
About “Alnylam 5x15™”
The “Alnylam 5x15” strategy, launched in January 2011, establishes a path for development and commercialization of novel RNAi therapeutics to address genetically defined diseases with high unmet medical need. Products arising from this initiative share several key characteristics including: a genetically defined target and disease; the potential to have a major impact in a high unmet need population; the ability to leverage the existing Alnylam RNAi delivery platform; the opportunity to monitor an early biomarker in Phase I clinical trials for human proof of concept; and the existence of clinically relevant endpoints for the filing of a new drug application (NDA) with a focused patient database and possible accelerated paths for commercialization. By the end of 2015, the company expects to have five such RNAi therapeutic programs in clinical development, including programs in advanced stages, on its own or with a partner. The “Alnylam 5x15” programs include ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-APC for the treatment of hemophilia, ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-TMP for the treatment of hemoglobinopathies. Alnylam intends to focus on developing and commercializing certain programs from this product strategy itself in the United States and potentially certain other countries; the company will seek development and commercial alliances for other core programs both in the United States and in other global territories.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, ALN-APC for the treatment of hemophilia, and ALN-TMP for the treatment of hemoglobinopathies. As part of its “Alnylam 5x15TM” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in clinical development, including programs in advanced stages, on its own or with a partner by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for applications in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam’s VaxiRNA™ platform applies RNAi technology to improve the manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, statements regarding Alnylam's views with respect to the potential for RNAi therapeutics, including ALN-TTR02 and ALN-TTRsc, its expectations with respect to the timing and success of its clinical and pre-clinical trials, the expected timing of regulatory filings, including its plan to file an IND or IND equivalent application for ALN-TTRsc and to initiate clinical trials for ALN-TTR02 and ALN-TTRsc, its expectations regarding the reporting of data from its ALN-TTR01, ALN-TTR02 and ALN-TTRsc clinical trials, and Alnylam’s expectations regarding its “Alnylam 5x15” product strategy, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Alnylam’s ability to discover and develop novel drug candidates, successfully demonstrate the efficacy and safety of its drug candidates, including ALN-TTR02 and ALN-TTRsc, the pre-clinical and clinical results for its product candidates, which may not support further development of product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, obtaining regulatory approval for products, competition from others using technology similar to Alnylam’s and others developing products for similar uses, and Alnylam’s ability to establish and maintain strategic business alliances and new business initiatives, as well as those risks more fully discussed in the “Risk Factors” section of its most recent annual report on Form 10-K on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.