Companies Archives - Page 38 of 59

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

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

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

Research Triangle Park, North Carolina-based Lumena was founded last year. The company is developing a new diabetes treatment based on the role bile acids play as signaling agents in the gastrointestinal tract. When Lumena launched its fundraising efforts last year, CEO Michael Grey explained that most gastric bypass surgery patients are type 2 diabetics. In 80 percent of those patients, blood glucose levels normalized within days — a change that’s not attributed to weight loss. What happened was that bile was diverted.

In normal gastrointestinal function, bile is recirculated. But gastric bypass surgery also bypasses that mechanism. The presence of bile in the GI tract triggers receptors that release Glucagon-like peptide 1, or GLP-1, a hormone that helps regulate blood sugar. Lumena’s treatment would work within the GI tract to stimulate the body’s production of GLP-1. Unlike other drugs that circulate through the bloodstream, the targeted action could offer fewer side and effects compared to other diabetes treatments.

Lumena’s technology comes from company co-founder Slava Gedulin, a former scientist at Amylin Pharmaceuticals (NASDAQ: AMLN), a San Diego, California biotechnology company that develops diabetes and obesity treatments. Gedulin’s discovery came after he left Amylin and the company has no claims on Lumena’s technology.

Lumena Pharmaceuticals - 02/21/2012

First and Only Personalized Treatment for Deadliest Form of Skin Cancer Approved by Health Canada

~ Health Canada approves Zelboraf, a targeted medicine designed to inhibit cancer growth in patients known to have a common type of metastatic melanoma ~

MISSISSAUGA, ON, Feb. 16, 2012 /CNW/ - Roche announced today that Zelboraf (vemurafenib) was approved in Canada as monotherapy for the treatment of BRAF V600 mutation-positive unresectable or metastatic melanoma. A validated test is required to identify BRAF V600 mutation status.1

Melanoma is the deadliest and most aggressive form of skin cancer, killing 80 to 85 per cent of stage IV patients within five years.2 Approximately half of all patients with melanoma carry a genetic mutation in the BRAF gene.3 Zelboraf is the first and only medicine shown to improve survival in people with BRAF V600 mutation-positive metastatic melanoma. It works by targeting and inhibiting the mutated BRAF protein found in about half of all cases of melanoma.3

"The medical community has been waiting for a treatment that will extend patients' lives while vastly improving quality of life," says Dr. Joel Claveau , Dermatologist, Dermatologist and Pigmented Lesions Clinic, Hospital Hotel-Dieu de Quebec . "Zelboraf is part of an innovative concept called personalized medicine. With a quick genetic test, I can now have the confidence that I'm prescribing the right treatment to patients who will benefit from it."

In the pivotal BRIM3 study in treatment-naïve patients, Zelboraf was shown to reduce the risk of death by 56 per cent for people who received Zelboraf compared to those who received dacarbazine.3 The study also found that participants on Zelboraf had a 74 per cent reduced risk of dying or having their disease progress compared to those who received dacarbazine.3 In another study in patients who failed at least one prior systemic therapy (BRIM2), Zelboraf shrank tumours in 52 per cent of trial participants.3

Mark Wallis , a 49-year-old father of three, is one of the 50 per cent of melanoma patients who has the BRAF mutation. Wallis received Zelboraf as part of the BRIM3 trial.

"The cancer had spread to my liver, my bones, both lungs and numerous lymph nodes so I was in an incredible amount of pain. "I was increasingly worried about my life and how it was going to affect my family," says Wallis, a commercial airline pilot from Milton, Ontario. "Within days of taking Zelboraf, the extreme pain I experienced literally vanished. Within weeks, my tumours had shrunk in size. I felt like I had been given my life back."

Annette Cyr , chair of the Melanoma Network of Canada , lives with melanoma and is all too familiar with the unique challenges patients have faced until now.

"For Canadians living with metastatic melanoma, the approval of Zelboraf brings new hope," says Cyr. "Zelboraf offers BRAF-positive patients a chance to live; to raise their children, to spend time with loved ones and to continue to contribute to society."

Health Canada also recently approved the cobas® 4800 BRAF V600 Mutation Test,4 a diagnostic test developed by Roche to identify patients eligible for treatment with Zelboraf, enabling oncologists to prospectively identify patients who may benefit from the treatment.

"As someone who has been tested positive for the BRAF mutation, I understand the valuable role that Zelboraf can play in the treatment of metastatic melanoma," says Kathy Barnard , Founder, Save Your Skin Foundation. "Patients with melanoma simply don't have the luxury of time and there is tremendous value in knowing right away whether or not a treatment will be effective. Personalized medicines like Zelboraf ensure that patients can receive the best possible care for their type of melanoma."

Personalized medicine is an innovative approach to healthcare that identifies the specific characteristics of a patient's illness, enabling physicians to implement the most effective and efficient treatment plan for each individual patient.5

"We believe that investment in personalized medicines is critical for driving optimal patient care while maximizing the use of limited health care resources," says Ronnie Miller , President and CEO, Roche Canada . "Zelboraf is an innovative, targeted therapy that will deliver effective care and improve outcomes for patients who have been without adequate treatment options for many years."

About BRIM3 and BRIM2

BRIM3 is a global, randomized, open-label, controlled, multicentre, Phase III study that compared Zelboraf to dacarbazine chemotherapy, a standard of care, in 675 patients with previously untreated BRAF V600E mutation-positive, unresectable (inoperable) or metastatic melanoma. The endpoints of BRIM3 were overall survival (OS) and investigator-assessed progression-free survival (PFS). Other endpoints included confirmed investigator-assessed best overall response rate. BRIM2 is a global, single-arm, multicentre, open-label Phase II study that enrolled 132 patients with previously treated BRAF V600E mutation-positive, unresectable or metastatic melanoma. The primary endpoint of BRIM2 was confirmed best overall response rate as assessed by independent review. In BRIM3, a higher percentage of patients treated with Zelboraf (42%) than dacarbazine (18%) experienced serious adverse events (SAEs) and the most common treatment-related SAEs in the patients treated with Zelboraf were cutaneous squamous cell carcinoma (cuSCC).

About BRAF V600 Mutation Testing

The cobas® 4800 BRAF V600 Mutation Test is a real-time polymerase chain reaction assay diagnostic test developed by Roche enabling physicians to identify whether a person with metastatic melanoma is eligible for treatment with Zelboraf. This Health Canada-approved test was clinically validated in the BRIM2 and BRIM3 studies to identify tumors that carry the BRAF V600E mutation. The test is robust, rapid and accurate providing a higher sensitivity in detecting the V600E mutation than Sanger sequencing.9

About Zelboraf

Zelboraf is an oral, small molecule, kinase inhibitor indicated as a monotherapy for the treatment of BRAF V600 mutation-positive unresectable or metastatic melanoma. Zelboraf should not be used in patients with wild-type BRAF melanoma or in patients where the BRAF mutational status is not known.

Zelboraf is being co-developed under a 2006 license and collaboration agreement between Roche and Plexxikon, a member of the Daiichi Sankyo Group.

Plexxikon - 02/16/2012

Transplant antiviral strong in phase 2; Chimerix plans phase 3 start this year

February 6, 2012

North Carolina biotechnology company Chimerix reported positive phase 2 results for a broad spectrum antiviral agent expected to have applications fighting infections faced by bone marrow transplant patients.

Chimerix now plans to start phase 3 clinical trials on CMX001 later this year. The company is studying the compound for the prevention of cytomegalovirus diseases, or CMV, in patients who have had hematopoietic stem cell transplants. Durham, North Carolina-based Chimerix said that compared to a placebo, CMX001 had a statistically significant benefit in preventing CMV from entering the bloodstream or from causing CMV disease 13 weeks after a transplant. The data were presented at a blood and marrow transplant conference in San Diego last Friday.

CMV is a member of the herpesvirus group of viruses; like other herpesviruses CMV has the ability to remain dormant in the body for long periods of time. The virus can lead to serious disease and even death in transplant recipients, cancer patients and others who have compromised immune systems.

Chimerix - 02/06/2012

Transplant antiviral strong in phase 2; Chimerix plans phase 3 start this year

Chimerix says that at least 65 percent of transplant recipients faced moderate to high risk of CMV from reactivated virus from donor or recipient tissue. The disease can lead to potentially life threatening conditions such as nephritis, pneumonitis or hepatitis. Complications are also possible leading to rejection of a transplanted organ. Currently available compounds to fight CMV carry significant toxicities.

Venture capital-backed Chimerix last year raised $45 million in a series F round of financing led by New Leaf Venture Partners. Other investors include Pappas Ventures, Morningside Group Canaan Partners, Sanderling Ventures, Alta Partners, Asset Management Company and Frazier Healthcare Ventures.

CMX001 is Chimerix’s lead drug candidate. The compound has also drawn interest from the federal government. Last year, the Biomedical Advanced Research and Development Authority, or BARDA, awarded Chimerix a contract valued at up to $81 million if the company develops CMX001 into an antiviral agent for small pox.

Chimerix - 02/06/2012