Companies Archives - Page 38 of 60

Ultragenyx Announces the Completion of the Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease

NOVATO, Calif., March 8, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the Phase 1 study of UX001 for hereditary inclusion body myopathy (HIBM) has been completed. 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. UX001 is the first program from the company's pipeline to enter the clinic since its founding in 2010. The FDA Office of Orphan Products Development has granted orphan drug designation for UX001 for the treatment of HIBM.

"Ultragenyx continues to make excellent progress in advancing and expanding our clinical development pipeline with the completion of our first clinical study," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "We deeply appreciate the participation and enthusiastic support of the HIBM patient community for our Phase 1 study. We anticipate releasing the results from this study within the next couple of months, and will look forward to presenting the data at a scientific meeting in the fall. We plan to initiate a Phase 2 trial of SA-ER in patients with HIBM in the second quarter of this year."

The Phase 1 clinical study evaluated the pharmacokinetics (PK) and safety of UX001 in 28 HIBM patients. The study tested five different single-dose levels in each group of six subjects. Subjects then underwent repeat dosing at four dose levels over 7 days to establish the steady-state pharmacokinetics and safety of repeat doses of UX001. Ultragenyx anticipates data from the Phase 1 study in April 2012.

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.

Ultragenyx Pharmaceutical - 03/09/2012

Milestone Pharmaceuticals Appoints Paul Truex to the Board of Directors

MONTREAL, March 5, 2012 (GLOBE NEWSWIRE) Milestone Pharmaceuticals Inc., a cardiovascular drug development company, today announced the appointment of Paul F. Truex to its Board of Directors. Mr. Truex is Chief Executive Officer, Founder and a Director of Anthera Pharmaceuticals Inc. (Nasdaq:ANTH), a biopharmaceutical company focused on developing drugs to treat serious illnesses, including cardiovascular and autoimmune diseases.

Anthera Pharmaceuticals / Milestone Pharmaceuticals - 03/05/2012

Milestone Pharmaceuticals Appoints Paul Truex to the Board of Directors

MONTREAL, March 5, 2012 (GLOBE NEWSWIRE) Milestone Pharmaceuticals Inc., a cardiovascular drug development company, today announced the appointment of Paul F. Truex to its Board of Directors. Mr. Truex is Chief Executive Officer, Founder and a Director of Anthera Pharmaceuticals Inc. (Nasdaq:ANTH), a biopharmaceutical company focused on developing drugs to treat serious illnesses, including cardiovascular and autoimmune diseases.

"Mr. Truex brings relevant operating, deal-making and financing experience to Milestone's Board," commented Philippe Douville, Chief Executive Officer of Milestone. "He exhibits a high level of passion to the business of drug development, and we look forward to benefiting from his guidance as Milestone pursues its strategy in preparing for its first clinical studies."

Mr. Truex was responsible for the initial acquisition of Anthera's intellectual property from negotiations with pharmaceutical companies. Prior to founding Anthera Pharmaceuticals, Mr. Truex served as a Founder, Director, President and CEO of Peninsula Pharmaceuticals. During that time, Mr. Truex raised over $81 million of institutional investment and strategic partner capital. Peninsula was acquired by Johnson and Johnson in April of 2005 for $245 million in an all cash transaction. Over the span of his career, Mr. Truex has successfully raised over $150 million in venture investments and more than $200 million in various public equity and debt offerings, including Anthera's Initial Public Offering in 2010. Prior to Peninsula, he served as a Senior Executive of Versicor (acquired by Pfizer) and participated in the company's successful $55 million initial public offering in 2000. Before joining Versicor, Mr. Truex worked at Eli Lilly and Company where he served in various marketing and sales roles during the launch of three different products for the primary care physician market (Actos' Evista' and Humalog 75/25).

Mr. Truex obtained his Masters of Business Administration in Marketing and Finance from Indiana University and a Bachelor of Arts degree in Economics from the University of Waterloo. Mr. Truex is a member of the Board of Directors of Trius Therapeutics, Eiger Biopharmaceuticals and Waterloo Pharmaceuticals, Inc.

Mr. Truex will replace Mr. Cedric Bisson who has retired from Milestone's Board of Directors. The members of Milestone's Board are grateful to Mr. Bisson for his unique and outstanding contribution to the development of the company over the past six years and wish him much success in his future endeavors.

About Milestone Pharmaceuticals

Milestone is a drug development company developing novel small molecule therapeutics based on clinically validated mechanisms for cardiovascular diseases. Milestone's lead product is a novel and potent short-acting calcium channel antagonist for the systemic treatment of transient cardiovascular conditions such as atrial arrhythmias and angina. The Company has assembled a world-class scientific advisory board of key opinion leaders with significant cardiovascular expertise in cardiology, regulatory affairs, and drug development. Milestone was founded in 2005 and initially financed by iNovia Capital, Fonds BioInnovation and an angel investor. For more information, please visit www.milestonepharma.com

Anthera Pharmaceuticals / Milestone Pharmaceuticals - 03/05/2012

BrainCells Inc. Announces the Successful Completion of the Single Ascending Dose Study With BCI-838 and the Initiation of the Multiple Ascending Dose Study

SAN DIEGO, Feb. 29, 2012 /PRNewswire/ -- BrainCells Inc., a leading biotechnology company developing novel compounds for the treatment of central nervous system (CNS) diseases, announced today that the Phase 1 single ascending dose (SAD) study of BCI-838 has been successfully completed and that the company has initiated the Phase 1 multiple ascending dose (MAD) study. The SAD study evaluated BCI-838 for safety, tolerability, pharmacokinetics and food effect in healthy male subjects. Single oral doses of BCI-838 up to 900 mg were administered and the drug was well tolerated. No serious adverse events were reported and all adverse events were mild in intensity, transient, and resolved without sequelae.

BrainCells - 02/29/2012

BrainCells Inc. Announces the Successful Completion of the Single Ascending Dose Study With BCI-838 and the Initiation of the Multiple Ascending Dose Study

SAN DIEGO, Feb. 29, 2012 /PRNewswire/ -- BrainCells Inc., a leading biotechnology company developing novel compounds for the treatment of central nervous system (CNS) diseases, announced today that the Phase 1 single ascending dose (SAD) study of BCI-838 has been successfully completed and that the company has initiated the Phase 1 multiple ascending dose (MAD) study. The SAD study evaluated BCI-838 for safety, tolerability, pharmacokinetics and food effect in healthy male subjects. Single oral doses of BCI-838 up to 900 mg were administered and the drug was well tolerated. No serious adverse events were reported and all adverse events were mild in intensity, transient, and resolved without sequelae.

"BCI-838's biological activity mimics that of ketamine, an intravenous anesthetic, which has been shown to be efficacious in treating patients with TRD. Since ketamine is associated with a number of unwanted side effects such as short-term dissociation and psychosis, safer compounds that act in a similar fashion promise to revolutionize the way the disease is managed for these patients," said Robert Williamson, Chief Executive Officer.

BCI-632 increases synaptic glutamate by inhibiting the mGlu2/3 auto-receptor, which is located predominantly at the pre-synaptic site. As in the case of ketamine, the long-lasting efficacy of BCI-632 can be blocked by either inhibition of AMPA receptors, mTOR or the BDNF signaling pathway. In contrast to ketamine, BCI-632 does not cause psychosis or dissociative effects. In addition, BCI-632 also stimulates serotonin release and, after chronic dosing, hippocampal neurogenesis.

"BCI-838 is an oral prodrug for the active compound BCI-632 that is an mGluR2/3 antagonist," said John Hutchinson, Ph.D. Senior Vice President of Research. "BCI-632 has been shown to work acutely in a range of animal models of depression, anxiety, cognition and Alzheimer's disease."

BrainCells - 02/29/2012

Selventa Granted Patent for Discovery of Biomarker Profiles

CAMBRIDGE, Mass. – January 23, 2012 – Selventa™, a biomarker discovery company that enables personalized healthcare through stratification of patients based on disease-driving mechanisms, today announced that the U. S. Patent & Trademark Office has issued the Company U.S. Patent No 8,802,109, titled "Computer-aided Discovery of Biomarker Profiles in Complex Biological Systems." This patent, which was issued on December 20, 2011, relates to methods and techniques that facilitate discovery of biomarkers and thus aid in the development of predictive and prognostic diagnostic tests for therapeutics targeting complex multi-factorial diseases.

In particular, the patent describes a method of biomarker discovery using a model representative of one or more causative biophysical or biochemical relationships underlying a biological state in a biological system of interest. A candidate set of biological data is then compared against the model to discern a candidate biomarker for the biological state. A sample received from a patient can then be assayed for the presence of the candidate biomarkers to discern a biological state of the patient.

"Using the technique, biomarkers may be developed to predict efficacy or toxic effects of a drug. This may permit a researcher or physician to know, in advance, whether the presenting patient will benefit from or be harmed by administration of the drug," said David de Graaf, Ph.D., President and CEO of Selventa. "In addition, the patented technique facilitates the development of biomarkers as diagnostics for a disease, as a means of making treatment decisions or as a means of stratifying patients in a clinical trial. This will likely improve the success of clinical trials by screening for subjects that are more likely to benefit from the drug and avoiding those who are unlikely to respond."

About Selventa
Founded in 2002 and privately-held, Selventa collaborates with partners to match the optimal treatments for the right patients. The company analyzes molecular patient data to identify key disease-driving mechanisms and accelerates the development process to clarify therapeutic and diagnostic decisions through identification and development of biomarkers for patient stratification. Selventa engages in strategic relationships with leading pharmaceutical and life science companies including clinical research organizations, diagnostic companies and reference laboratories to enable personalized healthcare primarily in autoimmune diseases and oncology. For more information, visit www.selventa.com.

Selventa - 02/29/2012

FDA grants orphan drug designation to Ultragenyx UX003 for treatment of MPS 7

February 28, 2012

Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7. MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the degradation of the glycosaminoglycans dermatan sulfate (DS) and heparan sulfate (HS).

UX003 is a recombinant human Beta-glucuronidase intended as an enzyme replacement therapy for the treatment of MPS 7. MPS 7 was originally described in 1973 by William Sly, MD, St. Louis University School of Medicine, and is also known as Sly Syndrome. Ultragenyx in-licensed the MPS 7 program from St. Louis University.

Ultragenyx Pharmaceutical - 02/28/2012

FDA grants orphan drug designation to Ultragenyx UX003 for treatment of MPS 7

"Obtaining orphan drug designation for UX003 is a significant achievement for Ultragenyx that adds value to our development pipeline and attests to the importance of this product candidate in filling an unmet medical need," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "We look forward to continuing to collaborate with Dr. Sly and colleagues at St. Louis University to advance this urgently needed therapy into clinical testing."

Ultragenyx Pharmaceutical - 02/28/2012

Plexxikon receives European approval for Zelboraf

Plexxikon, a member of Daiichi Sankyo Group, has received European Commission approval for Zelboraf (vemurafenib) for the monotherapy treatment of adult patients with BRAF mutation-positive unresectable or metastatic melanoma.

Zelboraf is designed to selectively inhibit the BRAF mutation that occurs in about half of all cases of melanoma.

Zelboraf and its companion diagnostic, the cobas 4800 BRAF V600 mutation test, have been approved in the US, Switzerland, Israel, Brazil, New Zealand and Canada.

Plexxikon CEO Peter Hirth said the approval of Zelboraf by the European Commission marks a significant advancement for European patients with metastatic melanoma who historically have had very limited treatment options.

"We are very pleased that our strategy to co-develop Zelboraf along with a companion diagnostic helped accelerate the availability of this personalized medicine for these patients," Hirth added.

Plexxikon - 02/21/2012

Diabetes drug developer Lumena raises $2.5M; compound regulates blood sugar

February 21, 2012

Diabetes treatment developer Lumena Pharmaceuticals has secured the $2 million it needs to start clinical trials and a little bit extra for good measure.

The company’s first round of investment was oversubscribed, leading to a haul of $2.5 million, according to amended filings made with the Securities and Exchange Commission. Lumena’s investors are venture capital firms Pappas Ventures and RiverVest Venture Partners. The new capital means the company can proceed to human tests for its novel type 2 diabetes treatment, which aims to develop a pill to help patients regulate their blood sugar.

Lumena Pharmaceuticals - 02/21/2012