Companies Archives - Page 35 of 59

MethylGene Announces Encouraging Data from Phase 1 Tumor Study

June 25, 2012

MethylGene Inc., a biopharmaceutical company, has announced the encouraging clinical data from the company's Phase I Met/VEGFR multi- kinase inhibitor MGCD265 study.

The MGCD265, a multitargeted oral tyrosine kinase receptor inhibitor of Met and VEGFR: Dose-escalation Phase I study provided an interim update on the monotherapy trial 265-101.

Trial 265-101 is an ongoing Phase I, multicenter, open-label trial. In this trial patients are treated with MGCD265 alone, dosed orally every day over a 21 day cycle. Data was presented on 57 patients with advanced metastatic or unresectable solid malignancies that were refractory to standard therapy and/or unlikely to derive clinical benefit from existing therapies.

In an ex vivo system designed to assess the biological activity of MGCD265 using plasma samples from study patients, increased plasma concentration of MGCD265 was associated with inhibition of Met phosphorylation in a dose-dependent manner, suggesting coverage of the biological target, Met, in the clinical setting.

Mirati Therapeutics - 06/25/2012

Liquidia Announces Product Development Collaboration with GlaxoSmithKline

RESEARCH TRIANGLE PARK, NC– June 20, 2012 -‐ Liquidia Technologies today announced the initiation of a broad, multi-‐year collaboration with GlaxoSmithKline (GSK), which has acquired exclusive rights to research and develop certain vaccine and inhaled product candidates using the company’s proprietary PRINT® (Particle Replication In Non-‐Wetting Templates) technology. Liquidia’s PRINT technology is a powerful and versatile nanoparticle technology product development and manufacturing platform that is changing the way companies engineer healthcare products.

Liquidia Technologies - 06/20/2012

Liquidia Announces Product Development Collaboration with GlaxoSmithKline

RESEARCH TRIANGLE PARK, NC– June 20, 2012 -‐ Liquidia Technologies today announced the initiation of a broad, multi-‐year collaboration with GlaxoSmithKline (GSK), which has acquired exclusive rights to research and develop certain vaccine and inhaled product candidates using the company’s proprietary PRINT® (Particle Replication In Non-‐Wetting Templates) technology. Liquidia’s PRINT technology is a powerful and versatile nanoparticle technology product development and manufacturing platform that is changing the way companies engineer healthcare products.

Full Release Attached

Liquidia Technologies - 06/20/2012

Liquidia Technologies signs lucrative deal with GSK

Liquidia Technologies, a small Research Triangle Park drug-development company, has signed a transformational licensing deal with pharmaceutical giant GlaxoSmithKline that could be worth up to several hundred million dollars.

Liquidia said Wednesday that GSK has licensed the rights to develop certain experimental vaccines and inhaled drugs using Liquidia’s proprietary nanotechnology, but the announcement didn’t include specifics. Liquidia said that a combination of payments from GSK -- including up-front money, research-and-development funding and milestone payments -- could amount to “up to several hundred million dollars” if all goes well.

Liquidia and GSK, which are collaborating on the drug-development efforts, are neighbors. London-based GSK has 3,800 workers at its U.S. headquarters in RTP and another 600 employees in Zebulon.

Liquidia was founded in 2004 based on the nanotechnology research of Joseph DeSimone, a chemist at N.C. State University and UNC-Chapel Hill. DeSimone founded the company with colleagues from UNC.

The privately held company has been nurtured by venture capital investments up to now, including funding provided by Triangle firms Pappas Ventures and the Wakefield Group.

“We are very pleased to have the opportunity to work with GSK, a company known for its commitment to scientific excellence, medicinal chemistry expertise and expansive knowledge of proprietary compounds that could potentially benefit from Liquidia’s...technology,” Liquidia CEO Neal Fowler said in a statement.

Liquidia has retained the rights to develop certain respiratory and vaccine medicines, as well as the right to use its technology to develop drugs in other therapeutic areas.

The company says that its PRINT technology -- Particle Replication In Non-Wetting Templates -- has the potential to develop next-generation drugs. For example, its technology is geared to deliver inhaled drugs to specific regions of the lung, which holds great promise for treating diseases such tuberculosis and cystic fibrosis.

Liquidia Technologies - 06/20/2012

Biotech All Stars Buy Castoff Experimental Merck Cancer Drug

Tesaro of Waltham, Mass., a tiny biotech now preparing for its initial public offering, just licensed a promising anti-cancer drug from Merck. The startup is attempting to repeat the success its top executives had turning Big Pharma’s castoffs into hits at MGI Pharma, which was bought by Eisai for $3.9 billion in 2008.

The drug, known as niraparib or MK-4827, is a medicine that targets a mutation that can cause certain hard-to-treat breast and ovarian cancers. AstraZeneca, Abbott Laboratories and Pfizer are also developing similar medicines. The deal was disclosed in a filing with the Securities and Exchange Commission.

At the same time that big drug companies are increasingly buying new medicines developed outside their own walls, they are also selling more of the medicines that they invent but don’t plan on developing to smaller biotechs. This has always happened – Cubist Pharmaceuticals’ antibiotic Cubicin was licensed from Eli Lilly more than a decade ago. But now it is becoming more commonplace, with Pfizer, for instance, talking about the option of selling experimental drugs to outside companies as a central part of its strategy.

Click here to read the full story.

TESARO - 06/06/2012

Liquidia Technologies Inc. Studies Show PRINT® Engineered Particles Hold Promise in the Delivery of Respiratory Therapeutics

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Liquidia Technologies today announced the results of two newly-released studies that illustrate the unique benefits of the company’s proprietary PRINT® (Particle Replication In Non-Wetting Templates) platform in the development of advanced respiratory therapeutics. Despite the high prevalence of lung disease, optimizing critical parameters of respiratory therapeutics to improve their pulmonary distribution and effectiveness has remained a challenge for the biopharmaceutical industry. These studies demonstrate how the PRINT platform can be used for respiratory drugs by precisely engineering particles with controlled shape, size, and chemistry, all characteristics that could lead to better lung delivery and therapeutic performance. Aspects of these findings were published in the Journal of Drug Delivery and presented at the Respiratory Drug Delivery Conference being held May 13-17, 2012 in Phoenix, Arizona.

Read the full article at BioSpace.

Liquidia Technologies - 05/15/2012

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