Companies Archives - Page 36 of 60

Achillion gets FDA incentives for hepatitis C drug

NEW HAVEN, Conn. Achillion Pharmaceuticals Inc. said Tuesday it will receive incentives from regulators as it studies a new experimental treatment for the hepatitis C virus.

The Food and Drug Administration awarded fast track status to Achillion's drug candidate ACH-3102. The agency's decision will allow Achillion to file its application for marketing approval piece by piece instead of waiting to collect all the clinical trial data for the drug, and Achillion will be able to consult with the FDA more often as it develops the drug. The FDA may also choose to review the full application in six months instead of the usual 10 months.

Achillion said it started clinical trials of ACH-3102 on Wednesday. Those trials are intended to test the drug's safety and its side effects rather than its effectiveness. The company plans to study a combination of ACH-3102 with its most advanced drug candidate, ACH-1625. Achillion hopes that combined, the drugs could be part of a hepatitis C a regimen that could treat the disease without interferon. Interferon is a common part of hepatitis C treatment but can cause serious side effects, including flu-like symptoms that can last for months.

Shares of Achillion gained 1 cent to $6.90 in midday trading. Earlier the stock rose as much as 7.4 percent.

Achillion Pharmaceuticals - 05/15/2012

Liquidia Founder Dr. Joseph DeSimone Elected into National Academy of Sciences

Dr. Joseph DeSimone Joins Elite List of Scientific Pioneers

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Liquidia Technologies today announced its founder, Dr. Joseph DeSimone, was elected a member of the National Academy of Sciences (NAS) for his excellence in original scientific research in the field of chemical engineering. Membership in the NAS is one of the highest honors given to a scientist or engineer in the United States. Among the renowned NAS members are Albert Einstein, Thomas Edison, Orville Wright, and Alexander Graham Bell. Nearly 200 living Academy members have won Nobel Prizes. Dr. DeSimone will be inducted into the Academy next April during its 150th annual meeting in Washington, D.C.

"Joe's scientific contributions across several diverse industries is nothing short of impressive and his election into the National Academy of Sciences is rare and well deserved honor," said Neal Fowler, CEO of Liquidia Technologies, "Joe's innovation and vision lie at the foundation of what we do at Liquidia and are the impetus behind a technology that is revolutionizing the development of healthcare products for a global population."

Dr. DeSimone is Chancellor's Eminent Professor of Chemistry at UNC-Chapel Hill and William R. Kenan Jr. Distinguished Professor of Chemical Engineering at North Carolina State University. He is also an adjunct member at Memorial Sloan-Kettering Cancer Center in New York. Dr. DeSimone has received over 40 major awards and recognitions including the 2010 AAAS Mentor Award, the 2009 NIH Director's Pioneer Award, and the 2008 Lemelson-MIT Prize. In 2005 he was elected to the National Academy of Engineering and the American Academy of Arts and Sciences. Dr. DeSimone has published more than 280 scientific articles and has 130 patents to his name with over 120 patents pending. He received his B.S. in chemistry in 1986 from Ursinus College in Collegeville, PA, and his Ph.D. in chemistry in 1990 from Virginia Tech.

The National Academy of Sciences is a private, nonprofit honorific society of distinguished scholars engaged in scientific and engineering research, dedicated to the furthering science and technology and to their use for the general welfare. Established in 1863, the National Academy of Sciences has served to "investigate, examine, experiment, and report upon any subject of science or art" whenever called upon to do so by any department of the government.

ABOUT LIQUIDIA

Liquidia Technologies is developing highly precise particle-based vaccines and therapeutics for the prevention and treatment of human disease. Combining a deep understanding of particle-based drug development with breakthrough small molecule and biological therapeutics, Liquidia is engineering vaccines and therapeutics that have the potential to dramatically improve the quality of human life. In addition to its own products, Liquidia licenses its PRINT® particle technology and its GMP manufacturing capabilities to support proprietary programs advanced by collaborators. The company was founded in 2004 and is located in Research Triangle Park, North Carolina. www.liquidia.com.

Liquidia Technologies - 05/03/2012

Ultragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease

NOVATO, Calif., May 1, 2012 /PRNewswire/ Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced results from a first-in-human, multi-center, Phase 1 study of UX001 in patients with hereditary inclusion body myopathy (HIBM) showing that UX001 was well-tolerated with an expected extended release profile on absorption after oral administration. UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency. Based on the Phase 1 results, Ultragenyx plans to initiate an international, multi-center, randomized, double-blind, placebo-controlled, parallel group Phase 2 study of UX001 in HIBM patients in the second quarter of this year.

Ultragenyx Pharmaceutical - 05/01/2012

Ultragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease

NOVATO, Calif., May 1, 2012 /PRNewswire/ Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced results from a first-in-human, multi-center, Phase 1 study of UX001 in patients with hereditary inclusion body myopathy (HIBM) showing that UX001 was well-tolerated with an expected extended release profile on absorption after oral administration. UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency. Based on the Phase 1 results, Ultragenyx plans to initiate an international, multi-center, randomized, double-blind, placebo-controlled, parallel group Phase 2 study of UX001 in HIBM patients in the second quarter of this year.

The Phase 1 clinical trial was a multi-center, sequential dose-escalation study designed to characterize the safety, tolerability and pharmacokinetics of UX001 Sialic Acid-Extended Release (SA-ER) tablets in patients with HIBM disease. Twenty-six subjects received SA-ER tablets orally at one of five (5) dose levels in the single-dose phase and one of four (4) dose levels in the repeat-dose phase. Preliminary data showed that SA-ER was well-tolerated at the doses evaluated, with no serious adverse events reported. The adverse event profile was unremarkable and showed no pattern or dose dependent relationship. Pharmacokinetic analysis from the study showed that single doses of SA-ER are absorbed and provide significant drug levels over a 12-16 hour period. On repeated 3 times per day dosing, serum free sialic acid concentrations reached relatively steady levels over a 24 hour cycle.

"We are encouraged by the results of this early stage clinical trial which suggest SA-ER in single and repeated oral dosing is well-tolerated and has the potential to be a treatment for patients suffering from HIBM," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "Based on these favorable Phase 1 results, we look forward to moving quickly to initiate a Phase 2 study of UX001."

Ultragenyx plans to present the Phase 1 data at the 17th Annual International World Muscle Society Congress in Perth, Australia later this year.

About HIBM

HIBM is also known as GNE myopathy, distal myopathy with rimmed vacuoles (DMRV) and Nonaka disease. HIBM is a severe, adult-onset, progressive, genetic neuromuscular disease caused by a deficiency of an enzyme in the first step of sialic acid biosynthesis needed for the modification of proteins and fats. Patients with HIBM typically begin to have weakness and abnormal walking at 18 to 30 years of age. Over the ensuing 10 to 20 years, many patients progressively lose significant functional ability and become wheelchair-bound. There are no current treatments for this disease.

About Ultragenyx

Ultragenyx is a privately held, developmental stage biotechnology company committed to bringing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases, also known as orphan and ultra-orphan diseases, to market. The company focuses on rare metabolic diseases that affect small numbers of patients, but for which the unmet medical need is high and there are no effective treatments. Ultragenyx intends to build a sustainable pipeline of safe and effective therapies to address these underserved diseases. Ultragenyx' lead program, UX001, is being evaluated as a potential treatment for hereditary inclusion body myopathy (HIBM), also known as GNE myopathy.

The company is led by an experienced management team in rare disease therapeutics. Ultragenyx is striving toward an improved model for successful rare disease drug development which has the potential to increase efficiency while maintaining appropriate safety and efficacy standards. The company believes that it can deliver significant value to patients by building a high quality pipeline of rare disease therapeutics and efficiently transforming good science into great medicine.

For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

Ultragenyx Pharmaceutical - 05/01/2012

CardioDx Honored as 2012 Gold Edison Award Winner

Corus CAD Wins at the Edison Awards Gala; Awards Celebrate 25 Years of Honoring Innovators and Innovation

PALO ALTO, Calif. – April 30, 2012 – CardioDx, Inc., a pioneer in the field of cardiovascular genomic diagnostics, today announced that Corus® CAD has been honored as a gold winner in the Science & Medical category of the internationally-renowned 2012 Edison Awards. The awards, which were presented last week at a gala in New York, symbolize the innovation personified by Thomas Alva Edison, inspiring America’s drive to remain at the forefront of creativity and ingenuity in the global economy.

Click here to read the full press release.

CardioDx - 04/30/2012

LipoScience Receives 2012 Diagnostic Marketing Association Creative Communications First Place Award

NMR LipoProfile® test campaign communicates importance of LDL-particles in cardiovascular disease risk and management

RALEIGH, N.C.- April 26, 2012 (BUSINESS WIRE) -- LipoScience, Inc., an in vitro diagnostic company advancing patient care by developing high value proprietary clinical diagnostic tests using nuclear magnetic resonance (NMR) technology, today announced that it has received first place recognition at the 2012 Diagnostic Marketing Association (DxMA) Creative Communications Awards for the integrated print and online category for LipoScience’s NMR LipoProfile® test campaign.

The award was presented at the 2012 DxMA awards reception on April 25 in Chicago. The DxMA recognizes excellence in diagnostics marketing, advertising and promotional programming. LipoScience partnered with Motivation Mechanics, a Philadelphia-based marketing research and communications strategy firm, on the campaign.

LipoScience - 04/26/2012

LipoScience Receives 2012 Diagnostic Marketing Association Creative Communications First Place Award

NMR LipoProfile® test campaign communicates importance of LDL-particles in cardiovascular disease risk and management

RALEIGH, N.C.--(BUSINESS WIRE)--LipoScience, Inc., an in vitro diagnostic company advancing patient care by developing high value proprietary clinical diagnostic tests using nuclear magnetic resonance (NMR) technology, today announced that it has received first place recognition at the 2012 Diagnostic Marketing Association (DxMA) Creative Communications Awards for the integrated print and online category for LipoScience’s NMR LipoProfile® test campaign.

“It is an honor to be recognized by the DxMA for our clinical communication initiatives,” said Mark Kirtland, Vice President, Marketing and Business Development at LipoScience. “The goal of our educational campaign continues to focus on increasing awareness of the NMR LipoProfile test and communicating the clinical value it can provide physicians and their patients in managing cardiovascular disease risk.”

“We are extremely proud of this industry award, which acknowledges the efforts of our marketing team to educate the broader physician community,” said Richard O. Brajer, President and Chief Executive Officer of LipoScience.

LipoScience - 04/26/2012

Ultragenyx Relocates Company Headquarters to Support Operational Expansion

NOVATO, Calif., April 23, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that it has moved its corporate headquarters to a larger facility in the Bel Marin Keys area of Novato, CA. The relocation will accommodate current and future growth of Ultragenyx' staff and expand operations in the areas of clinical development, regulatory affairs, technical operations, and administration. The company will continue to lease laboratory facilities at the Buck Institute in Novato which currently houses the company's research staff.

"Our move to our new corporate headquarters will enable us to support the advancement of our pipeline of treatments for rare and ultra-rare diseases and grow our team as needed," said Emil Kakkis, MD, PhD, Chief Executive Officer, who founded Ultragenyx in 2010. "We want to express our appreciation to the elected officials and city of Novato for their support of Ultragenyx, and we look forward to continuing to be an integral part of the Novato community."

Ultragenyx will celebrate the relocation of its corporate headquarters by holding a ribbon-cutting ceremony on Friday, April 27, 2012, at 3:30pm at its new location.

The address of the new corporate headquarters is 60 Leveroni Court, Novato, CA 94949, and the new corporate phone number is 415-483-8800.

Ultragenyx Pharmaceutical - 04/23/2012

Liquidia figuring out how to ‘unleash’ technology

DURHAM – Durham-based Liquidia Technologies’ is working on developing vaccines using tiny particles manufactured as carriers of substances meant to trigger or boost the body’s immune response. The goal is to “finely tune” that response, and increase vaccine efficiency and safety, the company’s chief executive said.

But the start-up company could also branch into other applications with its technology, Liquidia’s CEO Neal Fowler said Wednesday. Speaking as part of a panel at the Nanotech Commercialization Conference in Durham, Fowler said the company has established a deal with Procter & Gamble for the technology’s use in consumer products, and could look into other areas outside of life science as well.

The two-day conference, hosted by the NanoBusiness Commercialization Association and other groups, was focused on businesses in that use nanotechnology, which is technology conducted at a very small scale – the nano scale.

Charles Hamner, chair of The Hamner Institutes for Health Sciences, a nonprofit research organization that’s based in the Research Triangle Park, said the business development using nanotechnology is still in the innovation stage of development, but he believes it’s about to enter the growth stage, which is the stage before maturity.

Hamner, who was also on the panel alongside Fowler, said he believes resources need to be allocated to help grow nanotechnology’s use. John Hardin, executive director of the N.C. Department of Commerce’s Office of Science & Technology, advocated for the government’s role in allocating public resources to help stimulate early stage business sectors like business using nanotechnology.

Hardin said has office has been pushing nanotechnology, but added that his office also been selected for review by state legislators that he believes will lead to reduced funding.

“(We’re) now at a prime time to support nanotechnology and its growth,” Hamner said.

As for Liquidia’s development, Fowler said the company is now focused on working on its “second generation” vaccine candidates – vaccines for malaria and pneumonia.

The first generation involved work on an influenza vaccine candidate. The company announced the launch of a Phase 1 clinical trial for a seasonal influenza vaccine in 2010, which was a trial that helped the company show the safety of its technology, Fowler said.

In February of last year, the company announced a collaboration with the international nonprofit PATH Malaria Vaccine Initiative to explore using the company’s technology in the development of “next generation” malaria vaccines.

In March, Liquidia announced a $10 million equity investment from the Bill & Melinda Gates Foundation for development and commercialization of vaccines and therapeutics with a focus on malaria and flu vaccines.

Then in December, Liquidia announced a collaborative agreement with PATH to do preclinical proof-of-concept studies for a next-generation pneumonia vaccine.

“We’re in preclinical animal models now, we hope to get to human in the not-too-distant future,” Fowler said.

The company’s board has also had discussions about other applications for the company’s technology, Fowler said, including spinning out one application, or licensing it for another, as a way to generate revenue.

“We’ve had a lot of discussions about the right time to do that,” he said. “A lot of other applications outside vaccines are at a very early stage,” he added.

The company now has a partnership “looking at a couple consumer products,” with Procter & Gamble, a company that manufactures personal care, cleaning, laundry detergents, prescription drugs and other products.

“Our challenge going forward is, our opportunity will be, how to pace those events to get the technology (going) faster and faster,” Fowler said. “(We) had to go through three or four years of understanding what we have on our hands, (we’re) now figuring out how to unleash it.”

The company could use its particle technology across an array of products, he said, speaking generally.

“The world is full of particles, (we could) produce things for diagnostics, consumer products, solar panels to green building materials,” he said. “We can use our technology for any of that.”

Liquidia Technologies - 04/06/2012

Orphan Diseases and the Future of BioPharma, the Focus of the 2012 Orphan Disease Forum

WASHINGTON(BUSINESS WIRE) Thought leaders from dozens of biotech and pharmaceutical corporations from across the globe will gather June 19 20 to discuss innovations and regulations pertaining to Orphan Diseases, at the 2012 Orphan Disease Forum, as part of the 2012 BIO International Convention in Boston, MA.

The forum is co-organized by Centric Health Resources and the National Organization for Rare Disorders (NORD), and is hosted by the Biotechnology Industry Organization.

We are pleased to partner with NORD and Centric on this two-day forum to raise awareness for rare diseases and to improve the health of patients with debilitating and life-threatening diseases,? said Jim Greenwood, President and CEO of BIO. Thought leaders at the forefront of the international drug marketplace use this forum to advance therapies and cures for impacted patients.?

Industry consultants, biotech, pharmaceutical and venture capital companies and health benefits managers attend this two-day forum, as part of the BIO International Convention, the largest global gathering for the biotechnology industry. The forum and the Convention offer networking and partnering opportunities with policymakers, scientists, CEOs and newsmakers, and hundreds of sessions covering biotech trends, policy issues and technological innovations.

Forum agenda highlights include:

Are We Heading into a Pharmaceutical and Biotechnology Market of Orphan and Narrow Indications?
Industry experts will discuss the changing orphan drug environment. Perhaps the conditions are right for a new strategy: getting early market FDA approval with efficient initial funding, then later broadening the indications.
Tuesday, June 19, 8:30 a.m. 9:45 a.m.
Speakers: Emil D. Kakkis, PhD, President and Chief Executive Officer, Ultragenyx, Inc., Edward Mathers, Partner, New Enterprise Associates, Inc., David Savello, PhD, Senior Vice President, Development Operations, XenoPort, Inc. and Ron Smith, Vice President, Strategic Development, Blue Cross Blue Shield of North Carolina.

The Convention also features the BIO Business Forum, a unique platform for biotechnology and pharmaceutical companies, academic research institutions, and investors from around the world to gather and discuss strategic opportunities. For registration, conference agenda and exhibitor information, visit 2012 BIO International Convention.

For more information: Visit http://www.bio.org

BIO is pleased to recognize the leadership provided by the 2012 Orphan Disease Forum sponsors. The sponsors include: Alnylam Pharmaceuticals, Genzyme, a Sanofi Company and Millennium: The Takeda Oncology Company.

Ultragenyx Pharmaceutical - 04/06/2012