Genentech has revised its timeline ...

2008-05-16T08:05:58-04:00

Genentech has revised its timeline forecast for availability of final efficacy and safety results for the C-08 study from 2010 to 2009. The study, involving more than 2,700 patients, is being conducted by the National Surgical Adjuvant Breast and Bowel Project (NSABP) and is sponsored by the National Cancer Institute (NCI). The new timeline forecast is based primarily on the rate of NSABP's data collection, rapid patient enrollment, and a higher than planned number of Stage III patients. These updates do not change Genentech�s assessment of the study's probability of success. An independent data monitoring committee (DMC) for the study assesses safety and efficacy every six months and recommends whether adequate information is available to stop or continue the trial. Genentech has been informed that the last interim assessment took place in Q2 2008 and the DMC recommended that the trial continue as planned. An early look at safety results from the C-08 study will be among the more than 185 scientific abstracts regarding Avastin that will be published or presented at the ASCO meeting. Avastin studies will be featured in 11 oral presentations, including a late-breaker presentation of AVADO, a Roche-sponsored Phase III study of Avastin plus chemotherapy as a first-line treatment for metastatic breast cancer. Analyses of data from the AVADO study will be available on May 31, 2008. Encouraging results will also be presented from a Phase II study of Avastin in glioblastoma multiforme (GBM), an aggressive type of brain cancer. In addition, several studies will be presented that provide further support of Avastin's safety as a first-line treatment for patients with advanced, non-squamous, non-small cell lung cancer (NSCLC) and metastatic colorectal cancer

An abstract submitted to the Annual...

2008-05-16T07:46:26-04:00

An abstract submitted to the Annual Meeting of the American Society of Clinical Oncology (ASCO), entitled "Phase Ia Safety and Pharmacokinetic Study of Oral NV-196 in Patients with Solid Tumours" is now available. The abstract reports on a human clinical study of oral triphendiol which demonstrated a good safety profile and successful pharmacokinetics

Array BioPharma and AstraZeneca ann...

2008-05-16T07:35:23-04:00

Array BioPharma and AstraZeneca announced plans for two additional randomized Phase 2 trials for AZD6244 (ARRY-886). The trials, which will begin during the second half of 2008, will test AZD6244 in combination with a cytotoxic chemotherapeutic agent; one trial will be in melanoma patients and the other in non small cell lung cancer patients

Omrix Biopharmaceuticals announced ...

2008-05-16T07:30:50-04:00

Omrix Biopharmaceuticals announced that Larry Ellberger was named Chairman of the Board of Directors and Pamela McNamara was elected a Director at the Company's 2008 Annual Meeting of Stockholders held in New York City. Mr. Ellberger succeeds Fredric Price who served as Omrix' Chairman from January 2005. Mr. Ellberger has been a member of the Company�s Board since August 2006 and was previously Chairman of the Compensation Committee. He will continue to be a member of the Audit Committee and will Chair the Governance and Nominating Committee. Mr. Ellberger is also a member of the Board of AVANT Immunotherapeutics, Inc. and TransPharma Medical Ltd., and is a Founding Partner of consulting firm HVA, Inc. Ms. McNamara will Chair the Compensation Committee and be a member of the Audit Committee. Ms. McNamara has been Chief Executive Officer of CRF, Inc., a clinical trial data management and mobile technology company since October 2003

Valeant Pharmaceuticals announced t...

2008-05-15T08:10:16-04:00

Valeant Pharmaceuticals announced that the Food and Drug Administration (the FDA) has entered an administrative order in the public interest to stay the approval of generic fluorouracil cream 5% until May 30, 2008 because there are outstanding questions regarding this approval that the Agency must consider. The FDA hopes to conclude its review by May 30, 2008 and Spear has agreed to an administrative stay of its ANDA approval until May 30, 2008. According to the administrative order by the FDA, signed by Associate Commissioner for Policy and Planning, Jeffrey Shuren, marketing, sales and shipment under ANDA 77-524 are prohibited during the pendency of this administrative stay

Sepracor announced that its Board o...

2008-05-15T08:00:52-04:00

Sepracor announced that its Board of Directors has approved the appointment of Robert F. Scumaci as Executive Vice President and Chief Financial Officer. Mr. Scumaci most recently served as Executive Vice President, Corporate Finance, Administration and Technical Operations at Sepracor. Mr. Scumaci will assume his new role on May 20, 2008, replacing David P. Southwell, who is resigning to pursue other interests but will remain a consultant to the company through the end of the year

Human Genome Sciences announced tha...

2008-05-15T07:57:48-04:00

Human Genome Sciences announced that Kevin P. McRaith has joined the Company as Vice President, Hepatology Franchise. Mr. McRaith, formerly Vice President, Sales & Marketing- Hematology, Genentech Corporation, will report to Barry A. Labinger, Executive Vice President and Chief Commercial Officer

Theratechnologies announced a strat...

2008-05-15T07:55:51-04:00

Theratechnologies announced a strategic agreement with both the Massachusetts General Hospital ("MGH") and Dr. Steven Grinspoon to explore the use of tesamorelin in relative growth hormone deficient abdominally obese (GHDAO) subjects. MGH, under the direction of Dr. Grinspoon, will sponsor and conduct a clinical trial with tesamorelin in subjects that have excess visceral adipose tissue (VAT) with a moderate growth hormone deficiency and who are abdominally obese. The combination of these factors is now being recognized as an important contributor to increased risk of cardiovascular disease. Therefore, one of the reasonable approaches to lower the cardiovascular risk in these subjects is to decrease VAT levels with tesamorelin to replenish growth hormone secretion, and to measure certain key markers of cardiovascular risk. Theratechnologies has demonstrated in various Phase 2 and 3 clinical trials that tesamorelin significantly and specifically reduces VAT in certain patient populations

Allos Therapeutics reported interim...

2008-05-15T07:49:36-04:00

Allos Therapeutics reported interim response and safety data from the Company's pivotal Phase 2 PROPEL trial of PDX (pralatrexate) in patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). Twenty-nine percent (n=19) of the first 65 evaluable patients enrolled in the trial experienced either a complete or partial response, as assessed by central independent oncology review. Forty-five percent (n=29) of the first 65 evaluable patients experienced either a complete or partial response, as assessed by the PROPEL investigators. Patients are considered evaluable if they received at least one dose of PDX and their diagnosis of PTCL has been confirmed by independent review. The median duration of response for these patients cannot be estimated at this time due to the current length of follow up. The most common drug related grade 3/4 adverse events were mucositis and thrombocytopenia, which were observed in 14% and 23% of patients, respectively. Patients received a median of three prior treatment regimens

MorphoSys AG announced that at the ...

2008-05-15T07:44:35-04:00

MorphoSys AG announced that at the Company's annual shareholder meeting on Wednesday, May 14, 2008, its shareholders confirmed the reappointment of Dr. Gerald Moller, Dr. Daniel Camus, Dr. Metin Colpan and Dr. Geoffrey N. Vernon to the Supervisory Board. Dr. Moller was reappointed as Chairman of the Supervisory Board. In addition to the confirmation of the appointments to the Supervisory Board, all other management proposals put to vote were passed at the meeting, which took place in Munich. As part of the shareholder assembly agenda, the shareholders approved a three-for-one split of the Company's common stock

NexMed entered into a binding commi...

2008-05-14T11:39:36-04:00

NexMed entered into a binding commitment with one of its largest shareholders for a $3 million line of credit. Additional information is available in NexMed's 8-K filing for this transaction

Treat symptoms of idiopathic Parkin...

2007-11-30T11:32:12-04:00

Teva Pharmaceutical Industries announced that the U.S. Food and Drug Administration (FDA) has granted tentative approval for the Company's Abbreviated New Drug Application (ANDA) to market its generic version of GlaxoSmithKline's Requip (Ropinirole HCl) Tablets, Eq. 0.25 mg base, 0.5 mg base, 1 mg base, 2 mg base, 3 mg base, 4 mg base and 5 mg base. Final approval of Teva's Ropinirole HCl Tablets is expected upon expiry of patent protection for the brand product on May 19, 2008. Upon final approval, Teva's product will be the AB-rated generic equivalent of Requip Tablets, and will be indicated for the treatment of the signs and symptoms of idiopathic Parkinson's disease as well as treatment of moderate to severe primary restless leg syndrome, 11/302/2007.

Hepatitis C virus (HCV)

2008-02-19T09:01:54-04:00

Amarillo Biosciences announced that the U.S. Food and Drug Administration has allowed the Investigational New Drug (IND) application submitted by the Company to test its low dose oral interferon in a Phase 2 hepatitis C clinical trial to go into effect. Accordingly, CytoPharm Inc., the Company's partner in Taiwan, will fund and conduct a clinical trial of 144 chronic hepatitis C patients in Taiwan. The patients will receive one of two different dosages of oral human interferon alpha or placebo. The aim of the trial is to reduce relapse rate for those patients who have completed the standard combination therapy, consisting of high dose injectable interferon alpha and Ribavirin given orally. Although most patients respond to the standard therapy, up to 40% of those with certain viral genotypes relapse after treatment. The trial is expected to start in the 2nd quarter of 2008, 2/19/2008.

Alzheimer's disease

2008-01-28T11:56:52-04:00

Medivation announced that, based on its end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), the Company plans to begin a pivotal confirmatory Phase 3 trial of Dimebon for mild-to-moderate Alzheimer's Disease in the second quarter of 2008. The FDA informed Medivation that the company's previously completed trial conducted in Russia can be used as one of the two pivotal studies required to support the approval of Dimebon to treat mild-to-moderate Alzheimer's disease, as long as a significant proportion of the sites in the confirmatory Phase 3 trial are located in the United States. The Phase 3 clinical trial will enroll approximately 525 patients with mild-to-moderate Alzheimer's disease at sites in the United States, Europe and South America. Patients will be randomized to one of three treatment groups: Dimebon 20 mg three times per day (TID); Dimebon 5 mg TID; and placebo. Patients will be treated for six months and may not be taking any other Alzheimer's disease drugs. The primary endpoints are the Alzheimer's Disease Assessment Scale - cognitive subscale (ADAS-cog) and the Clinician's Interview-Based Impression of Change plus caregiver interview (CIBIC-plus), 1/28/2008. Medivation ammouced that the benefits of Dimebon over placebo in its double-blind, placebo-controlled Phase II study in mild-to-medium Alzheimer's disease were statistically significant on all five study endpoints at 12 months. On every endpoint studied, the benefits of Dimebon over placebo at one year were stable or greater when compared to benefits at 6 months. The endpoints included all of the most frequently studied aspects of Alzheimer's disease: cognition, overall clinical function, activities of daily living, and behavioral problems, 6/11/2007. Medivation announced that six-month results from its randomized, double-blinded, placebo-controlled Phase 2 efficacy trial of Dimebon(TM) in patients with mild-to-moderate Alzheimer's disease demonstrated that patients treated with Dimebon were significantly improved compared to patients taking placebo on all five efficacy endpoints studied, which assessed cognitive function, memory, ability to perform tasks of daily living, global function and behavior. New data presented in Salzburg included the magnitude and levels of statistical significance by which the Dimebon-treated patients outperformed the placebo-treated patients on the Mini Mental State Exam (MMSE: p<0.0001), the Alzheimer's Disease Cooperative Study Group-Activities of Daily Living (ADCS-ADL: p=0.002), and the Neuropsychiatric Inventory (NPI: p=0.006). As previously reported, Dimebon-treated patients also demonstrated significant improvement versus placebo on both the primary and key secondary endpoints in this study -- the Alzheimer's Disease Assessment Scale-cognitive subscale (ADAS-cog: p<0.0001) and the Clinician's Interview-Based Impression of Change with a caregiver interview (CIBIC-plus: p<0.0001). Dimebon was well tolerated in this study, 3/19/2007. Phase 2 complete and the Company plans to enter Phase 3, 11/15/2006.

Post-Herpetic Neuralgia

2007-12-18T13:53:47-04:00

DURECT announced positive results from a Phase IIa clinical trial for ELADUR, DURECT's proprietary investigational transdermal pain patch. In this study of patients suffering from post-herpetic neuralgia, ELADUR showed improved pain control versus placebo during the 3 day continuous treatment period. In addition, ELADUR appeared well tolerated overall, and patients treated with ELADUR and placebo exhibited similar safety profiles. This Phase IIa study was a randomized, multi center, double blind, placebo controlled, two-way cross over study of 60 patients suffering from post herpetic neuralgia (post shingles pain or PHN). The objectives of the study were to assess the safety as well as the magnitude, duration and characteristics of analgesic activity of ELADUR. DURECT anticipates that detailed results will be submitted for presentation at the American Pain Society Annual Meeting in May 2008, 12/18/2007. DURECT has started Phase II dosing in the U.S. under an FDA-accepted Investigational New Drug (IND) application for TRANSDUR-Bupivacaine (DUR-843), a transdermal pain patch for patients suffering from Post-Herpetic Neuralgia (post-shingles pain or PHN). DURECT's Phase I trial for TRANSDUR-Bupivacaine, initially reported on December 11, 2006, demonstrated good safety, tolerability and drug release for up to 3 days. TRANSDUR-Bupivacaine is intended to provide up to 3 days of pain relief for patients suffering from PHN, as compared to a wearing time limited to 12 hours with currently available patches, 1/3/2007. DURECT announced that it has successfully completed Phase I clinical trials with a new product, DUR-843, which is intended to treat a persistent pain condition. We believe that the persistent pain market remains underserved and that DUR-843 has the potential to provide several advantages over existing pain medications, 12/11/2006.

Dupuytren's disease

2007-12-17T10:23:57-04:00

Auxilium Pharmaceuticals completed patient enrollment in the Company's second U.S. phase III pivotal trial (CORD I) and its Australian phase III study (CORD II) of XIAFLEX (clostridial collagenase for injection) for the treatment of Dupuytren's contracture. In accordance with the study design, all enrolled patients have received their first injection of either XIAFLEX or placebo. Due to the high level of interest from patients and physicians, the Company was able to exceed its enrollment targets in both studies, with greater than 300 patients enrolled in the CORD I and CORD II studies combined. The Company had targeted enrolling 216 patients in CORD I and 60 patients in CORD II. Will report top line efficacy results from these two trials in the second quarter of 2008 and that we are on track to file a Biologics License Application with the Food and Drug Administration in early 2009, 12/17/2007. Auxilium Pharmaceuticals announced that the first patients have been dosed in the Company's second U.S. phase III pivotal trial and the Australian phase III study for XIAFLEX for the treatment of Dupuytren's contracture, a disabling and recurring condition in which the joints in the hand contract, impairing patients' ability to straighten and move their fingers, 9/10/2007. Auxilium Pharmaceuticals received clearance to resume Phase III clinical trials for XIAFLEX for the treatment of Dupuytren's contracture, 8/16/2007. AA4500 was effective against Dupuytren's contracture in an early Phase III trial, 2/20/2007. AA4500 met the primary endpoint of therapeutic success rate in a pivotal Phase 2/3 trial to treat Dupuytren's disease. In the double-blind trial in 35 patients, preliminary data showed that the injectable form of collagenase ABC enzyme had a therapeutic success rate of 91% vs. 0% for placebo (p<0.001). Therapeutic success was defined as reduction of the contracture of the affected joint to less than 5 degrees, allowing the hand to be flat when placed on a table, 6/21/2006.

Moderate/severe chronic pain; deter...

2007-12-06T09:07:26-04:00

Pain Therapeutics and King Pharmaceuticals announced positive results of a Phase III study of Remoxy(tm) in patients with chronic pain. Over 400 patients with osteoarthritis participated in this pivotal study. The study met the primary endpoint (p is less than 0.01) that was prospectively defined by the U.S. Food and Drug Administration (FDA) during the Special Protocol Assessment process. Remoxy, an investigational drug, is an abuse deterrent version of long acting oxycodone, a powerful painkiller available only by prescription. Remoxy is intended to meet the needs of physicians or pharmacists who appropriately prescribe or dispense long acting oxycodone and who seek to minimize the risks of abuse, misuse or diversion. The FDA has agreed in writing that a single Phase III pivotal study is needed to support the regulatory approval of Remoxy. As a result, Pain Therapeutics expects to file a New Drug Application for Remoxy in Q2 2008, 12/6/2007. Pain Therapeutics and King Pharmaceuticals reached the enrollment target of 400 patients for a Phase III study with Remoxy(tm). Remoxy, an investigational drug, is a proprietary abuse-deterrent version of long-acting oxycodone, a strong opioid painkiller. This randomized, double-blinded, placebo-controlled study enrolled patients in the U.S. with moderate-to-severe osteoarthritic pain. Following a titration period, patients were randomized to either twice-daily Remoxy (10-80 mg daily) or placebo for 12 weeks. The primary endpoint is change in pain scores during the treatment period. Top line results of this study are expected in Q4 2007, after the last patient completes the three-month treatment period. This Remoxy study is being conducted under the auspices of a Special Protocol Assessment (SPA) from the U.S. Food and Drug Administration (FDA). The SPA specifies the Phase III trial objective, design, clinical endpoints and statistical analyses needed to support FDA approval. These features are considered binding, i.e., the FDA will not later alter its perspective unless public health concerns unrecognized at the time of protocol assessment are evident. Under the SPA, one successful Phase III study is required to file a New Drug Application for Remoxy, 7/11/2007. Successfully completed a Special Protocol Assessment with the U.S. Food and Drug Administration (FDA). As a result, the companies are commencing a pivotal Phase 3 trial with Remoxy in 400 patients with severe chronic pain, 2/16/2006. The primary objective of the Phase 3 study was to evaluate pain relief. Results demonstrated a statistically significant difference (p<0.05) between Remoxy and placebo in the study's primary endpoint: percent decrease in pain scores from baseline to final study visit, measured with a standard Likert Pain Scale. The secondary objective of this study was to evaluate patients' quality of life. Results demonstrated statistically significant differences (p<0.05) between Remoxy and placebo in each component of the WOMACTM Osteoarthritis Index, in physical function measured by a standard SF-12 Health Survey and in patients' self-reported Quality of Analgesia, 9/9/2005. Phase 3 enrollment complete; results reported in the third quarter of 2005, 6/8/2005. Pain Therapeutics conducted a head-to-head comparison of the abusability of Remoxy versus Oxycontin. In this clinical study, 10 human volunteers consumed a crushed dose of Remoxy or Oxycontin with a glass of water. The study was repeated in the same subjects two days later, only this time they consumed a crushed dose of Remoxy or Oxycontin with high-proof alcohol. During each study, levels of oxycodone (the active ingredient in both drugs) were measured from the volunteers' blood samples. In both cases, oxycodone levels were significantly lower in the Remoxy group than in the Oxycontin group within 120 minutes of ingestion (p<0.05), when drug abusers presumably expect to get high. The data from this study provides fresh evidence that Remoxy has the potential to deter oxycodone abuse since it is absorbed much more slowly than Oxycontin when crushed and consumed. Pain Therapeutics plans to initiate a Phase 3 study with Remoxy later this month, 12/6/2004. Has received regulatory clearance to initiate clinical studies with Remoxy in the United States. The Company had been conducting clinical studies with Remoxy in England and pre-clinical studies in the U.S., 9/2/2004. New study results in humans demonstrates that Remoxy is significantly less abusable than Oxycontin. In a head-to-head clinical comparison of the two drugs, Oxycontin released over 200% more drug than Remoxy (p=0.03) in an abuse study in high-proof alcohol and over 170% more drug in a chewing study (p=0.02) in the first hour of the studies (when abusers presumably expect to get high). The Company also announced Remoxy is bioequivalent to Oxycontin, meaning the two drugs provide similar levels of oxycodone in people when used as intended, 6/29/2004. Phase 1 initiated, 1/15/2004. IND filed with the FDA, 11/5/2003.

Chronic pain

2007-12-06T09:33:26-04:00

DURECT announced that the pivotal Phase III trial for Remoxy had successfully met its primary endpoint (p<0.01) that was prospectively defined by the U.S. Food and Drug Administration (FDA) during the Special Protocol Assessment process. Remoxy, an investigational drug based on DURECT's patented ORADUR technology, is an abuse deterrent version of long acting oxycodone, a powerful painkiller available only by prescription. Remoxy is intended to meet the needs of physicians or pharmacists who appropriately prescribe or dispense long acting oxycodone and who seek to minimize the risks of abuse, misuse or diversion. These successful results were reported by Pain Therapeutics and King Pharmaceuticals; Pain Therapeutics is DURECT's licensee of the rights to this investigational drug, and they in turn have sublicensed the commercialization rights to King Pharmaceuticals. According to Pain Therapeutics and King Pharmaceuticals, the FDA has agreed in writing that a single Phase III pivotal study is needed to support the regulatory approval of Remoxy. As a result, Pain Therapeutics has stated that they expect to file a New Drug Application for Remoxy in Q2 2008, 12/6/2007. DURECT reported that patient recruitment has been completed in the pivotal Phase III study with Remoxy, an abuse resistant pain medicine under development based on DURECT's patented ORADUR technology incorporating the opioid oxycodone. This event was announced today by Pain Therapeutics and King Pharmaceuticals; Pain Therapeutics is DURECT's licensee of the rights to this drug candidate and they in turn have sublicensed the commercialization rights to King Pharmaceuticals, 7/11/2007.

Breast cancer

2007-12-05T10:42:33-04:00

Phase 2 data expected in 2Q08. ADVENTRX Pharmaceuticals has completed patient enrollment in its Phase 2 clinical trial of ANX-510, or CoFactor, for the treatment of advanced breast cancer. The Phase 2 clinical trial is a single arm, multicenter study to evaluate the safety and efficacy of treatment with CoFactor plus 5-fluorouracil (5-FU) in advanced breast cancer patients who have failed anthracycline and taxane chemotherapies. Patients are treated with CoFactor followed by 5-FU administered by IV bolus weekly for 6 weeks, with tumor and safety assessments every 8 weeks. The primary endpoint for the study is objective response rate as defined by Response Evaluation Criteria in Solid Tumors (RECIST) criteria, and secondary endpoints are duration of response, progression free survival, overall survival and incidence and severity of adverse events, as defined by the National Cancer Institute (NCI) Common Terminology Criteria. A total of 32 patients were enrolled in this study, 12/5/2007. ADVENTRX Pharmaceuticals announced the initiation of a Phase II clinical trial using CoFactor in the treatment of refractory metastatic breast cancer. CoFactor (ANX-510) is a folate-based biomodulator drug designed to enhance the activity and reduce associated toxicity of the widely used cancer chemotherapeutic agent 5-fluorouracil, 12/27/2006.

Multiple sclerosis

2008-01-28T09:48:53-04:00

Acorda Therapeutics announced results from a thorough QT study of Fampridine-SR. This study evaluated the potential to cause an increase in the electrocardiographic QT interval. Fampridine-SR, at both therapeutic and supratherapeutic doses, was found to be no different than placebo. The U.S. Food and Drug Administration requires thorough QT studies for all new drugs seeking regulatory approval, as increases in the QT interval (corrected for changes in heart rate, or QTc) may signify an increased risk of developing malignant cardiac arrhythmias. This double-blind trial compared the electrocardiographic effects of Fampridine-SR, given at a therapeutic (10 mg twice daily) and supratherapeutic dose (30 mg twice daily), to placebo and moxifloxacin in 208 healthy subjects. Moxifloxacin is a positive control known to increase the QT interval. The placebo-corrected QTc mean change from baseline (using the individual correction method for heart rate, or QTci) for the therapeutic and supratherapeutic doses of Fampridine-SR were 0 and 1 milliseconds, respectively. Moxifloxacin demonstrated QT prolongation consistent with previous clinical experience. In addition to no changes in the mean QTci interval, none of the subjects in the Fampridine-SR cohort showed increases in the QTci of greater than 30 milliseconds, nor did any of the Fampridine-SR subjects display a QTci interval that exceeded 480 milliseconds at any time, 1/28/2008. Acorda Therapeutics completed enrollment for its Phase 3 clinical trial of Fampridine-SR in multiple sclerosis (MS). The trial is designed to evaluate the safety and efficacy of Fampridine-SR in improving walking ability in people with MS. 240 patients were enrolled at 39 clinical trial sites in the United States and Canada. The Company expects data from the trial in the second quarter of 2008, 11/30/2007. Acorda Therapeutics has begun a second Phase 3 clinical study of Fampridine-SR in multiple sclerosis (MS), with the randomization of its first patient into the treatment phase of the study. The study is expected to enroll approximately 200 patients at 35 leading MS clinical centers in the United States and Canada. Fifteen centers have been initiated and are in the process of screening subjects for the trial. The MS-F204 study, which is conducted under a Special Protocol Assessment (SPA) issued by the Food and Drug Administration (FDA), will evaluate the safety and efficacy of Fampridine-SR in improving walking ability in people with MS. An SPA is a process in which the FDA provides guidance on a Phase 3 clinical trial whose data will form the primary basis for an efficacy claim. Pending clinical results from MS-F204, FDA has agreed that this study together with the previous Phase 3 study would be adequate to support a New Drug Application (NDA) for Fampridine-SR. The primary outcome measure for the study will be a walking response criterion, defined as a consistent improvement in walking speed as measured by the Timed 25-Foot Walk. The secondary outcome measure for this study is the Lower Extremity Manual Muscle Test, 6/6/2007. Acorda Therapeutics announced positive results from its Phase 3 clinical trial of Fampridine-SR on walking in people with multiple sclerosis (MS). Statistical significance was achieved on all three efficacy criteria defined in the Special Protocol Assessment (SPA) by the Food and Drug Administration (FDA). A significantly greater proportion of people taking Fampridine-SR had a consistent improvement in walking speed, the study's primary outcome, compared to people taking placebo (34.8 percent vs. 8.3 percent) as measured by the Timed 25-Foot Walk (p less than 0.001). In addition, the effect was maintained in this study throughout the 14-week treatment period (p less than 0.001) and there was a statistically significant improvement in the 12-Item MS Walking Scale (MSWS-12) for walking responders vs. non-responders (p less than 0.001). The average increase in walking speed over the treatment period compared to baseline was 25.2 percent for the drug group vs. 4.7 percent for the placebo group. Increased response rate on the Timed 25-Foot Walk was seen across all four major types of MS. In addition, statistically significant increases in leg strength were seen in both the Fampridine-SR Timed Walk responders (p less than 0.001) and the Fampridine-SR Timed Walk non-responders (p=0.046) compared to placebo, 9/25/2006. Completed enrollment of its Phase 3 clinical trial of Fampridine-SR in multiple sclerosis. The study, which is based on a Special Protocol Assessment (SPA) issued by the Food and Drug Administration (FDA), is evaluating the safety and efficacy of Fampridine-SR in improving walking ability in people with MS, 3/3/2006. Phase 3 data expected in third quarter of 2006.

Immediate treatment of patients wit...

2007-10-07T15:17:36-04:00

The Medicines Company announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's supplemental new drug application (sNDA) for a modified dosing regimen of Angiomax (bivalirudin) for the treatment of acute coronary syndromes (ACS), specifically in patients with unstable angina or non-segment elevation myocardial infarction (NSTEMI). The Company expects the FDA to act on this filing in the second quarter of 2008. Angiomax is currently approved for patients undergoing percutaneous coronary intervention (PCI), commonly referred to as angioplasty, 9/24/2007. The Medicines Company announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's supplemental new drug application (sNDA) for a modified dosing regimen of Angiomax (bivalirudin) for the treatment of acute coronary syndromes (ACS), specifically in patients with unstable angina or non-segment elevation myocardial infarction (NSTEMI). The Company expects the FDA to act on this filing in the second quarter of 2008. Angiomax is currently approved for patients undergoing percutaneous coronary intervention (PCI), commonly referred to as angioplasty, 9/24/2007. The Medicines Company announced it submitted a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) for Angiomax (bivalirudin) in support of a proposed new dosage for immediate treatment of patients with acute coronary syndromes (ACS). Angiomax is currently approved for patients undergoing percutaneous coronary intervention (PCI), commonly referred to as angioplasty, 8/7/2007. One-year findings from the landmark ACUITY trial show that acute coronary syndrome (ACS) patients in the Angiomax (bivalirudin) alone treatment group had similar rates of ischemic clinical outcomes compared with more complicated standard therapy, confirming previous findings, which showed similar ischemia at 30 days, and nearly 50% fewer episodes of major bleeding. At one year, the mortality rate of patients treated in the Angiomax alone treatment group was 3.8%, compared to 4.4% in the control treatment group. A separate analysis found that, in patients with ACS, having a major bleeding episode within 30 days following treatment nearly triples the risk of death up to one year later, making major bleeding a more powerful predictor of mortality than even a heart attack, 3/26/2007.

Depression and/or anxiety

2007-10-30T08:02:32-04:00

Phase 1. Phase 2 is expected to begin in 2Q08.

Prevention of acute graft-versus-ho...

2007-08-19T20:00:49-04:00

DOR BioPharma announced that patient enrollment is commencing in a randomized, double blinded, placebo controlled Phase 2 clinical trial of orBec for the prevention of acute graft-versus-host disease (GVHD) after allogeneic hematopoietic cell transplantation (HCT) with myeloablative conditioning regimens. The trial is being conducted by Paul Martin, MD at the Fred Hutchinson Cancer Research Center in Seattle, WA and is being supported, in large part, by a National Institute of Health (NIH) grant. The Phase 2 trial will seek to enroll up to 138 (92 orBec and 46 placebo) patients. The primary endpoint of the trial is the proportion of subjects who develop acute GVHD with severity sufficient to require systemic immunosuppressive treatment on or before day 90 after transplantation. Patients in the orBec group will begin study drug at the start of the conditioning regimen and continue through day 75 following HCT. Trial enrollment is expected to complete in the second quarter of 2008, 7/12/2007.

HIV-associated lipodystrophy

2008-04-14T10:59:45-04:00

Theratechnologies announced that the last patient has completed 26 weeks of treatment in the confirmatory Phase 3 trial testing tesamorelin in HIV-associated lipodystrophy. The Company now expects to be in a position to present top-line, 26-week results of this confirmatory Phase 3 trial by the end of the first half of 2008, 4/14/2008. Theratechnologies announced that new data from its first 52-week Phase 3 study testing tesamorelin in HIV-associated lipodystrophy were presented as a poster (Poster Number 943) on February 4, 2008 at the 15th Conference on Retroviruses and Opportunistic Infections (CROI) in Boston, Massachusetts. The purpose of the extension phase of the study was to evaluate the safety profile at 52 weeks, which demonstrated that tesamorelin was overall well tolerated. In general, at 52 weeks, tesamorelin showed a clinically significant decrease in triglyceride levels with maintenance of cholesterol levels within normal range. Furthermore, tesamorelin, at 52 weeks, had no clinical effect on glucose parameters and resulted in improved body composition including the selective maintenance of VAT loss without affecting subcutaneous adipose tissue and limb fat. As a final note, the reduction of visceral adipose tissue was significant in both males and females at 52 weeks compared to baseline levels, 2/6/2008, Theratechnologies announced positive 52-week results of its Phase 3 clinical trial, evaluating the long-term safety profile of the Company's lead compound, tesamorelin (TH9507), in patients with HIV-associated lipodystrophy. The 52-week results are consistent with the safety profile observed in the first 26 weeks of treatment and show that tesamorelin is well tolerated. In addition, tesamorelin's efficacy is confirmed as patients on treatment for 52 weeks lost 18% of their visceral adipose tissue (VAT) compared to baseline, 10/1/2007. Theratechnologies has learned from the US Food and Drug Administration (FDA) that, based on the information known today, the tesamorelin (TH9507) clinical development program is moving in the right direction. The FDA Division of Metabolic and Endocrine Drug Products had recently requested an update on the status of Theratechnologies' clinical trial program and preliminary results for tesamorelin. Upon review of the documentation, the FDA indicated that they were reassured especially given that thus far there are no signals of glucose intolerance or other safety concerns. With the information known today, the FDA indicated that it does not anticipate that additional clinical outcome trials would be required, 9/11/2007. Theratechnologies has notified its principal investigators to end recruitment by September 11, 2007 for its confirmatory Phase 3 clinical trial using tesamorelin (TH9507) for the treatment of HIV-associated lipodystrophy. Patients recruited on or before this date will be screened and those eligible will be officially enrolled in the study within the next few weeks, 9/4/2007. Theratechnologies enrolled the first patient in its second Phase 3 clinical trial testing TH9507 in HIV-associated lipodystrophy. The objective of the new study is to confirm, for regulatory purposes, the beneficial effects seen in the Company's first Phase 3 study, the top-line results of which were made public in December 2006, 1/31/2007. Theratechnologies announced that its ThGRF (TH9507) met the primary endpoint of a significant reduction in visceral adipose tissue versus placebo in a Phase III trial in 412 patients with HIV-associated lipodystrophy. In the trial, the analog of growth hormone releasing factor showed a 20% reduction in VAT at 26 weeks versus placebo (p<0.001). Theratechnologies expects to start a confirmatory Phase III trial in North America and Europe 1Q'07, 12/19/2006. Theratechnologies completed the efficacy phase (26 weeks of treatment) of its first Phase 3 clinical trial testing TH9507 in HIV-associated lipodystrophy. The Company is now proceeding to assemble and refine the data with a view to locking the database in December. Once these steps have been completed, the results will be analyzed and made public. This Phase 3 trial is a multi-center, double-blind, randomized, placebo-controlled study conducted in 41 centers in the United States and Canada. The study is examining the safety and efficacy of a daily administration of 2 milligrams of TH9507 for a period of 26 weeks. The primary endpoint is a reduction of visceral adipose tissue, or VAT, which is a risk factor for cardiovascular disease and type 2 diabetes, 10/25/2006. Theratechnologies announced a Special Protocol Assessment (SPA) from the U. S. Food and Drug Administration for the design of its second pivotal Phase 3 trial evaluating TH9507 for the treatment of HIV-associated lipodystrophy. Company plans call for the new Phase 3 trial to start in the first quarter of 2007. Theratechnologies launched its TH9507 pivotal Phase 3 program in March 2005. The first 412-patient clinical trial began in June 2005 and patient enrolment at 43 clinical sites across North America was completed on schedule in March 2006 with results expected around the end of this year. The second trial will test TH9507 in approximately 400 additional patients in North America and Europe. It is patterned after the first trial and is intended to confirm its results. The FDA has now formally evaluated the protocol for this second trial through the SPA process. The SPA provides the Company with binding written agreement that the proposed design and analysis of the study are adequate to support a marketing application provided the study is performed in accordance with the SPA and the results are successful, 8/23/2006. Theratechnologies announced that an independent Data and Safety Monitoring Board (DSMB) has recommended that its Phase 3 clinical study on TH9507 for the treatment of HIV associated lipodystrophy be continued as currently being conducted. The DSMB is comprised of independent medical experts and is responsible for monitoring the safety aspects of the Phase 3 clinical study, 7/6/2006. Theratechnologies completed patient enrollment for its first Phase 3 clinical trial using TH9507 for the treatment of HIV-associated lipodystrophy, 3/23/2006.

Prevent respiratory depression caus...

2008-03-31T11:59:42-04:00

Cortex Pharmaceuticals was notified by the German regulatory agency which approves the use of narcotic drugs that it can proceed with the trial which will use alfentanil, an opiate analgesic. With this approval the enrollment of subjects into the dose response study evaluating the ability of CX717 to prevent respiratory depression caused by opiate analgesics has begun. The study is a placebo controlled, double blinded, cross-over design with three doses of CX717compared to placebo. Depending on the pace of subjects being enrolled, Cortex at this point believes that top-line data will be available from this study by the end of June, 2008, 3/31/2008

Post-operative ileus (POI)

2008-03-12T08:13:10-04:00

Wyeth Pharmaceuticals and Progenics Pharmaceuticals announced preliminary findings from the first of two phase 3 clinical trials of intravenous methylnaltrexone being evaluated for the management of postoperative ileus (POI) in patients recovering from segmental colectomy surgical procedures. Preliminary results from the phase 3 clinical trial conducted by Wyeth showed that treatment did not achieve the primary end point of the study: a reduction in time to recovery of gastrointestinal function (i.e., time to first bowel movement) as compared to placebo. The study also did not show that secondary measures of surgical recovery, including time to discharge eligibility, were superior to placebo. In this clinical study, methylnaltrexone was administered intravenously in doses of 12 or 24 mg every six hours and was generally well tolerated. The second phase 3 trial of intravenous methylnaltrexone for management of POI is being led by Progenics and is similar in design to the Wyeth study reported today. Progenics announced on January 8, 2008 that it had completed enrollment in this trial, with results expected to be reported by midyear, 3/12/2008. Wyeth Pharmaceuticals and Progenics Pharmaceuticals announced the initiation of the second of two global, pivotal phase 3 clinical trials to evaluate the safety and efficacy of intravenous methylnaltrexone for the treatment of post-operative ileus (POI). The first phase 3 trial was initiated in September 2006, 12/20/2006. Wyeth Pharmaceuticals and Progenics started a double-blind, placebo-controlled international Phase III trial of IV methylnaltrexone to treat post-operative ileus (POI) in about 500 patients. The primary endpoint is duration of POI as measured by time to first bowel movement. Secondary endpoints include safety and other measures of gastrointestinal recovery, such as time to discharge eligibility. The trial has an SPA from FDA. The partners expect to begin a second Phase III trial of IV methylnaltrexone this year and hope to submit an NDA in late 2007 or early 2008, 9/20/2006. In Phase 2, patients who received MNTX following major abdominal surgery exhibited an acceleration of gastrointestinal recovery by at least one day on average compared to placebo. Significant improvements were seen in clinically important measures of gastrointestinal recovery: time to first bowel movement and discharge eligibility from the hospital. MNTX was generally well tolerated in this study, with no reports of serious adverse events related to the drug. There is currently no approved therapy for post-operative bowel dysfunction, a debilitating gastrointestinal impairment following abdominal or other major surgeries and a leading cause of prolonged patient hospital stays and increased healthcare costs, 1/20/2005. Target enrollment has been reached in the phase 2 study of its investigational drug, methylnaltrexone (MNTX) for the treatment of post-operative ileus, 10/7/2004. Phase 2 initiated, 5/13/2003.

Pancreatic insufficiency

2008-02-22T13:02:22-04:00

Eurand announced that the New Drug Application (NDA) for Zentase (EUR-1008) was accepted and has been granted priority review by the United States Food and Drug Administration, 2/22/2008. Eurand has completed the filing of its New Drug Application (NDA) with the Food and Drug Administration (FDA) for Zentase (EUR-1008), the Company's lead product candidate for the treatment of exocrine pancreatic insufficiency, 12/20/2007. Eurand expects to complete its gastrointestinal (GI) bioavailability study of Zentase before the end of November 2007. The Company expects to finalize its NDA submission before the end of the year, at which time it will request priority review from the Food and Drug Administration (FDA). Eurand initiated its rolling NDA submission in June 2007 and was granted fast track designation by the FDA. Eurand announced results that were presented at the recent European Cystic Fibrosis Society meeting in Antalya, Turkey, held June 13-16. Results from two phase III studies of Zentase (EUR-1008) show a statistically and clinically significant improvement in the absorption of fat, protein and nutrients in patients suffering from Exocrine Pancreatic Insufficiency (EPI). Absorption levels were measured by coefficient of fat (CFA) and coefficient of nitrogen (CNA) absorption, common measurements for malabsorption in EPI patients. In the pivotal study, the mean CFA after Zentase was 88.3 percent and the mean CNA after Zentase was 87.2 percent. Levels under placebo were 62.8 and 65.7 percent respectively (p=0.001). Scientific evidence suggests that to normalize EPI, treatment needs to raise CFA levels to at least 85 percent. These data were the basis for the recent rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Zentase as a treatment for EPI. EPI is a potentially lethal disease marked by the deficiency of digestive enzymes normally produced by the pancreas. This deficiency results in poor digestion and reduced absorption of nutrients which, if left untreated, can lead to malnutrition, impaired growth, weakened immune response, and shortened life expectancy. Treatment with pancreatic enzyme products (PEPs) replaces enzymes lost through the disease. Currently, there are no known alternative therapies to PEPs for EPI, 6/19/2007. Eurand Pharmaceuticals initiated the rolling submission of its new drug application (NDA) with the U.S. Food and Drug Administration (FDA) for Zentase for the treatment of Exocrine Pancreatic Insufficiency (EPI). Zentase (formerly EUR-1008), the company's lead product candidate, has been granted fast track designation by the FDA. Zentase is a zero-overfill, highly stable, porcine-derived, pancreatic enzyme product (PEP) designed to meet FDA guidelines for PEPs. In April, 2004, the FDA mandated that all manufacturers of EPI drug products file a NDA and receive approval for their products by April 2008 or be subject to regulatory action, 6/12/2007.

Depression

2008-02-08T10:34:41-04:00

Labopharm completed North American Phase III clinical trial for its once-daily formulation of the antidepressant trazodone (study 04ACL3-001) achieved statistical significance for the primary efficacy endpoint (p value of 0.0183). The study also demonstrated significantly improved patient sleep patterns in favour of trazodone. Labopharm plans to file a New Drug Application (NDA) for its once-daily formulation of trazodone with the U.S. Food and Drug Administration later this year. Study 04ACL3-001 is a randomized, double-blind, two-arm, multi-centre study comparing the efficacy and safety of Labopharm's once-daily trazodone formulation to placebo, in patients with major unipolar depressive disorder, over an eight-week period. In the study, 412 patients were randomized to treatment with Labopharm's once-daily trazodone, or placebo. Within the initial two-week titration period, patients were titrated every three to four days to an optimal dose (maximum dose of 375 mg/day). Patients were then maintained at the optimal dose for the remainder of the study. The study was conducted at 40 centers across the U.S. and Canada. The primary efficacy endpoint of the study was to compare the change in the Hamilton Rating Scale for Depression (HAMD-17) total score from baseline to the end of the study in the once-daily trazodone group versus the placebo group. Statistical significance was achieved for the primary endpoint (p value of 0.0183) and under additional methods of analysis as specified in the Statistical Analysis Plan (SAP). The overall drop out rate in the study was 25.5%. The drop out rate was 21% in the placebo group and 30.2% in the once-daily trazodone group. In the once-daily trazodone group, 4% of patients discontinued treatment due to somnolence or sedation. The drop out rate observed in this study is comparable to drop out rates in typical depression studies, 2/8/2008. Labopharm expects to have results for the Phase 3 trial of its once-daily antidepressant drug trazodone by April 2008, after completing enrollment of the key study ahead of schedule. More than 400 patients have been enrolled in the study, with the final patient scheduled for randomization by the end of the week. Results from the study are expected at the beginning of the second quarter of 2008, James Howard-Tripp, president and chief executive, said in a statement. The study, which is randomized and double-blind, compares the efficacy and safety of Labopharm's once-daily formulation with a placebo in patients with major unipolar depressive disorder, 9/25/2007.

Arterial thrombo-embolic diseases

2008-01-10T13:27:09-04:00

Arena Pharmaceuticals announced positive results from a single dose Phase 1a clinical trial of APD791 and the initiation of a multiple dose Phase 1b clinical trial to further evaluate the compound's safety, pharmacokinetics and pharmacodynamics. APD791 is Arena's internally discovered oral drug candidate intended for the treatment of arterial thrombosis. The Phase 1a trial was a randomized, placebo-controlled, double-blind, single-ascending dose trial in 90 healthy male and female volunteers. Doses originally intended for study ranged from 1 mg to 160 mg, but due to excellent tolerability the maximum dose was increased to 320 mg. Doses were well tolerated, without any dose related adverse events, such that a maximum tolerated dose could not be defined despite achieving high concentrations in blood. APD791 was rapidly absorbed and exposures were generally related to dose. Terminal half-life (t1/2) of the parent plus active metabolites was also related to dose, and was approximately 11 hours at the higher doses. Dose dependent inhibition of serotonin-mediated amplification of platelet aggregation was demonstrated starting at the 1 mg dose, supporting APD791 preclinical data and establishing initial clinical validation of APD791's novel mechanism of action. Based on the positive Phase 1a results, a Phase 1b trial was initiated. The Phase 1b trial is a randomized, placebo-controlled, double-blind, multiple-ascending dose trial in up to 50 healthy male and female volunteers between the ages of 19 and 45 years old. In addition to evaluating APD791's safety and tolerability profile, the trial will also evaluate the pharmacokinetics and pharmacodynamics of multiple oral doses of APD791 over a period of one week. Results from the Phase 1b trial are anticipated in mid 2008, 1/9/2008. Arena Pharmaceuticals initiated dosing in a Phase 1 clinical trial evaluating APD791, Arena's orally administered, internally discovered drug candidate intended for the treatment of arterial thrombo-embolic diseases. This Phase 1 trial is planned to enroll up to 72 healthy adult volunteers and is primarily intended to evaluate the safety and tolerability of single ascending doses of APD791. In addition, the trial will also evaluate the pharmacokinetics and pharmacodynamics of APD791, 7/18/2007.

Hereditary deficiency (HD) of antit...

2008-02-25T09:44:58-04:00

GTC Biotherapeutics announced that ATryn has completed enrollment in the pivotal study for the treatment of patients with hereditary antithrombin deficiency, or HD, undergoing high-risk surgical or childbirth procedures, 2/25/2008. GTC Biotherapeutics announced that ATryn has met the statistical requirements for the primary endpoint in the pivotal study for the treatment of patients with hereditary antithrombin deficiency, or HD, undergoing high-risk surgical or childbirth procedures. The primary endpoint was demonstration of non-inferiority to plasma-derived antithrombin in preventing clinically relevant deep vein thromboses, or DVTs, or other thromboembolisms. GTC also announced that it has initiated the filing of the associated Biologics License Application, or BLA, by submitting the preclinical and manufacturing sections. The last section to be filed is expected to be the full clinical study results around mid-year, once all reviews of the data have been completed. GTC is seeking priority review in conjunction with the BLA submission, and on that basis anticipates approval of the BLA approximately 6 months after submission of the last section. GTC previously received approval to commence the BLA filing on a rolling basis when ATryn was granted fast track status by the Food and Drug Administration, or FDA. Separately, ATryn has also been designated an orphan drug for the HD indication. The completed comparator arm of the study is based on historical data gathered under a prospective clinical protocol from patients who had been previously treated with plasma-derived antithrombin products while undergoing similar high-risk procedures to those performed in the active arm. There were no clinically relevant DVTs or other thromboembolisms in these patients. A minimum of 31 evaluable HD patients are required for the ATryn treatment trial. Results for 14 of these patients were already obtained from the previous study that supported ATryn's approval in the European Union for HD patients undergoing surgical procedures. Seventeen additional patients have been treated, of which 16 are considered evaluable. One patient is considered unevaluable due to the course of treatment deviating from the clinical protocol. None of the additional patients had clinically relevant DVTs or other thromboembolisms during the evaluation period. On this basis, the study has already met the statistical requirements for non-inferiority. Although the primary endpoint has already been met, enrollment is being extended through February to include an additional patient to ensure that the minimum number of evaluable patient procedures agreed upon with the FDA is satisfied, 2/4/2008. GTC Biotherapeutics completed recruitment into the historical arm of the comparative clinical study of ATryn in the treatment of patients with hereditary antithrombin deficiency, or HD, undergoing high-risk surgical or childbirth procedures. This study is expected to support a Biologics License Application, or BLA, submission with the United States Food and Drug Administration, or FDA. Evaluable records for 37 patients were obtained, meeting the required minimum of 35 HD patients. The data from the historical records were gathered under a prospective clinical protocol observing patients who had been treated with plasma-derived antithrombin products. A minimum of 31 evaluable HD patients are required for the ATryn treatment trial. Results for 14 of these patients were already obtained from the study that supported ATryn's approval in the European Union for HD patients undergoing surgical procedures. Due to patient scheduling decisions, top line results of the clinical study are now expected to be available around the end of January. The ongoing study is designed to determine the non-inferiority of ATryn to plasma-derived antithrombin products in reducing the incidence of deep vein thrombosis or other thromboembolisms in this challenging patient population, 1/7/2008. GTC Biotherapeutics announced that the US Food and Drug Administration, or FDA, has designated ATryn a "fast track product" entitled to accelerated FDA review for the hereditary antithrombin deficiency indication. The FDA has also granted GTC permission to submit the associated Biologics License Application, or BLA, for ATrynÃÂ® on a rolling basis. Fast track designation is provided to those products that are intended to treat serious or potentially life threatening conditions for which there is an unmet medical need. The BLA requesting marketing approval for ATryn will be submitted as sections are completed rather than waiting for all sections to be submitted together, enabling FDA review to begin sooner. GTC anticipates filing the initial sections with the FDA in the fourth quarter and completing the rolling submission after all clinical data is gathered, analyzed, and available for the BLA, which is planned to be by the end of the first quarter of 2008, 9/4/2007. GTC Biotherapeutics reported that the European Commission has granted market authorization to ATryn, GTC's recombinant form of human antithrombin, for the prophylaxis of venous thromboembolism in surgery of patients with congenital antithrombin deficiency. Antithrombin is a naturally occurring plasma protein that has both anticoagulant and anti-inflammatory properties. GTC produces ATryn in the milk of goats that have a transgene for human antithrombin. ATryn is the first transgenically produced protein to be approved for human therapeutic use anywhere in the world. ATryn is also the first recombinant antithrombin product approved anywhere in the world and the first antithrombin product, whether recombinant or derived from the human blood supply, that has been approved through the centralized EMEA procedure for use in all 25 countries of the European Union, 8/2/2006. Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) has adopted a positive opinion on the market authorization application (MAA) for ATryn, GTC's recombinant form of human antithrombin. The CHMP has recommended that ATryn be granted market authorization for the prophylaxis of venous thromboembolism in surgery of patients with congenital antithrombin deficiency. ATryn may be given in association with heparin or low molecular weight heparin in these situations. The CHMP opinion recommends granting market authorization under the EMEA's procedures for exceptional circumstances. Final market authorization by the European Commission is expected in about three months. The positive CHMP opinion followed a defined process this week that included a review of GTC's submission of the grounds supporting re-examination of the previous opinion as well as a review of responses to specific questions posed by the CHMP to an independent expert panel composed of internationally recognized experts in the fields of hematology and hemostasis. These review activities are an integral part of the regulatory process for re-examination of a prior opinion, 6/2/2006. GTC Biotherapeutics has been told that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) intends to issue a negative opinion on the market authorization application (MAA) for ATryn, GTC's recombinant form of human antithrombin. On the basis of recent conversations, GTC understands that the CHMP, after excluding data from pregnant patients, determined that an insufficient number of surgical patients were enrolled to support approval. In addition, GTC understands that the CHMP has concerns about sufficient immunologic data and the lack of clinical data from ATryn produced with an additional filtration step. GTC intends to take advantage of the appeal process to request a CHMP re-examination of GTC's submission, 2/23/2006. GTC Biotherapeutics has filed its response to the European Medicines Agency's (EMEA) List of Outstanding Issues generated during review of GTC's Market Authorization Application (MAA) for ATryn , its recombinant form of human antithrombin. GTC estimates that the EMEA will make a determination on the MAA before the end of October, 2005. GTC also recently began the recruitment process for a pivotal clinical study of ATryn for the hereditary antithrombin deficiency indication as allowed by the United States Food and Drug Administration, 7/6/2005. GTC Biotherapeutics plans to respond by July 8, 2005 to the European Medicines Agency's (EMEA) List of Outstanding Issues generated during review of GTC's Market Authorization Application (MAA) for ATryn , its recombinant form of human antithrombin. This response date follows from meetings between GTC and the EMEA. The EMEA has accepted the response date as a formal extension of the MAA review schedule. Assuming timely filing of the response, GTC estimates that the EMEA could make a determination on the MAA before the end of October, 2005, 4/25/2005. Submitted its responses to the Consolidated List of Questions generated by the European Medicines Agency (EMEA) as part of the review of a Marketing Authorization Application (MAA) for ATryn, GTC's recombinant form of human antithrombin. Expect response by April of 2005, 12/20/2004. The trial showed that treatment with ATryn was found to be efficacious in preventing the development of symptomatic deep vein thromboses (DVT) and other thromboembolic complications which may occur in patients with confirmed hereditary antithrombin deficiency who are undergoing high risk surgical procedures or labor and delivery, 2/27/2004. European Medicines Evaluation Agency (EMEA) has accepted for review GTC's Market Authorization Application (MAA) submission of ATryn, 2/27/2004. Announced the submission of a Market Authorization Application (MAA) to the European Medicines Evaluation Agency (EMEA) for ATryn, its recombinant human antithrombin product, 1/26/2004. Enrollment into the efficacy study for recombinant human antithrombin III (rhATIII) has been completed. The results of the efficacy study will be included in a filing requesting marketing authorization in Europe. A total of 14 patients have been enrolled and treated in this study. The efficacy study was designed to assess the ability of rhATIII to prevent blood clots in patients that have a hereditary antithrombin deficiency and are giving birth or undergoing a major surgical procedure. GTC expects to file for marketing authorization in Europe by February 2004, 12/17/2003. Initiation of a safety and efficacy study of its recombinant human antithrombin III (rhATIII) for the treatment of patients with a hereditary deficiency (HD) of the antithrombin blood protein. This study is expected to be the final clinical trial before submission for European registration, 12/6/2002. Pharmacokinetic (PK) study, with each patient monitored over a six-day period. Infusion of rhATIII was well tolerated and no serious adverse drug reactions were reported. The study showed that administration of rhATIII produced an increase in plasma antithrombin activity similar to that observed when plasma-derived antithrombin products are used in routine clinical practice. A dosing regimen based on this data has now been developed for an efficacy trial, which is expected to begin before the end of 2002, 9/6/2002.

Follicular B cell non-Hodgkin's lym...

2008-04-09T08:41:54-04:00

Favrille announced that it has reached the data cutoff date for the Company's Phase 3 registration trial of Specifid (mitumprotimut-T, formerly FavId) following Rituxan in patients with follicular B-cell non-Hodgkin's lymphoma (NHL). As of the data cutoff date, 205 of the 349 patients randomized have experienced disease progression (relapsed) according to investigator determination. Final analysis will be based on a central radiology assessment of the patients' CT scans. Unblinding of the data is expected to occur in June 2008, 4/9/2008. Favrille announced that an independent Data Monitoring Board (DMB) was recently convened to assess time to tumor progression (TTP) for patients in the control group of the Company's pivotal Phase 3 clinical trial of FavId. The DMB assessment, which was conducted in accordance with the clinical protocol under Favrille's Special Protocol Assessment (SPA) with the Food & Drug Administration (FDA), indicated that the control group behavior is consistent with data reported in the published literature. Based in part on the guidance provided by the DMB, Favrille has determined April 2008 to be the appropriate time for data cutoff of the Phase 3 trial. The Company anticipates that the analysis of the data will be completed and made available no later than July 2008, 10/25/2007. Phase 3 enrollment complete. Analysis of the primary endpoint of the trial, time to disease progression, is expected during the second half of 2007. In addition, Favrille recently announced that it has been granted Fast Track designation from the FDA for FavId. A Fast Track designation indicates that the FDA will expedite the review of a new drug that is intended to treat a serious or life-threatening condition and that demonstrates the potential to address an unmet medical need, 1/30/2006. Favrille announced long-term follow-up data from a physician-sponsored Phase 2 clinical trial of FavId following high dose therapy and autologous stem cell transplantation in patients with non-Hodgkin's lymphoma (NHL). A total of 15 patients were treated in the trial, of which nine remain in complete remission up to 61 months post-transplant. The median number of prior regimens for all patients was three, ranging from one to 10. The data showed that the majority of patients in the trial developed a rapid and tumor-specific immune response, often measured following a single dose of FavId, 2/12/2007. Favrille announced that the Data Monitoring Committee (DMC) completed its prospectively planned interim analysis of a secondary endpoint in the first 233 patients enrolled in the Company's ongoing pivotal, placebo-controlled Phase 3 clinical trial of FavId following Rituxan induction therapy in patients with follicular B- cell non-Hodgkin's lymphoma (NHL). The interim analysis showed a high percentage of patients converted to complete remission over time. The objective response rate (ORR) was first assessed eight weeks following Rituxan induction treatment, at which time it was 64%, with 18% of patients in complete remission. The best response rate was assessed at three-month intervals and ORR was shown to increase to 70%, with 46% of patients in complete remission. Treatment with FavId or placebo was initiated after the first response assessment. The blinded data showed that 41% of patients who were assessed as stable disease or partial remission at the end of Rituxan induction treatment had a response improvement, and 80% of those patients converted from partial to complete remission. The DMC indicated that this prospectively planned interim analysis did not demonstrate a statistically significant difference between treatment and control groups in the secondary endpoint of response improvement. The primary endpoint in the trial is time to disease progression (TTP). The interim analysis was conducted on 233 randomized patients who had been followed for 12 months or more. The trial has randomized a total of 349 patients, approximately 200 of whom have not progressed and remain on study, 11/13/2006. Completed enrollment in the Company's pivotal Phase 3 clinical trial of FavId, 1/30/2006. FDA has granted Fast Track designation for FavId, 1/12/2006. In Phase 2, A total of 103 patients were enrolled in the multi-center, open-label Phase 2 trial, of which 89 had stable disease or a better response to Rituxan and received FavId, including 55 who were relapsed from or refractory to prior treatments and 34 who were treatment-naive. The Phase 2 trial was closed to enrollment in December 2003. In particular, treatment-naive patients who responded to an initial course of Rituxan have demonstrated the longest TTP, with only 17 percent of the patients in this subpopulation (4 of 23) having progressed to date. At a median observation period of approximately 22 months, only 19 percent of the total treatment-naive population (10 of 35) and 13 percent of the Rituxan-responder population (10 of 44) have progressed. The median TTP of the 23 patients in the chemotherapy relapse population is projected at 24.2 months, compared to published historical data of 10.2 months in a similar population. The overall clinical response rate in the Phase 2 trial increased from 49 percent at month 3 following Rituxan alone to 65 percent following the initiation of FavId. In addition, 29 percent of patients improved their condition from stable disease to partial response after the initiation of FavId (12 of 42), 5 percent improved from stable disease to complete response (2 of 42) and 21 percent improved from partial response to complete response (9 of 43) for an overall Response Improvement of 27 percent (23 of 85) in the trial, 12/13/2005. A total of 103 patients were enrolled in this Phase 2 trial, of which 89 had a stable disease or better response to Rituxan and received FavId, including 55 who were relapsed from or refractory to prior treatments and 34 who were treatment-naive. The overall clinical response rate measured in the trial increased from 49 percent following Rituxan alone to 64 percent with the addition of FavId. In addition, the progression-free survival rate in the treatment-naive and chemotherapy relapse populations remained at approximately 70 percent at 18 months from trial enrollment. Notably, at the time of the abstract's submission in August 2005, the 71 patients in this Phase 2 trial who would be eligible for the Company's ongoing Phase 3 trial had not reached median time to disease progression (TTP). The Phase 2 trial was closed to enrollment in December 2003, 11/16/2005. First patient has been enrolled in a physician-sponsored Phase 2 clinical trial evaluating the Company's lead product candidate, FavId in Europe. Both treatment-naive patients and patients who are relapsed or refractory to prior treatments for their disease are eligible for the trial, 6/6/2005. Favrille announced that it has enrolled 220 eligible patients into its Phase 3 registration clinical trial testing its lead product candidate, FavId, following Rituxan in patients with follicular non-Hodgkin's lymphoma (NHL). This represents approximately 65% of the 342 randomized, evaluable patients required to be enrolled in the trial. As originally projected, the Company expects to complete enrollment on schedule by year end, 5/31/2005.

Lung cancer

2007-10-24T08:12:11-04:00

Active Biotech AB presented the final results of the previously reported clinical Phase I study of the cancer project ANYARA in combination with Taxotere in patients with non-small cell lung cancer. The results show that ANYARA can be given safely in combination with Taxotere (Sanofi-Aventis), an established drug for the treatment of non-small cell lung cancer. Thirteen patients with advanced non-small cell lung cancer were included in the study. Observed side effects during ANYARA treatment were expected and included transient fever, hypotension and nausea. Other toxicities recorded in the study were typical of Taxotere. Out of ten evaluable patients, two received a partial response (a tumor reduction of at least 30 percent), including one patient that had previously failed with Taxotere treatment and 5 patients had stable disease. ANYARA is presently developed primarily for the treatment of Renal Cancer and a pivotal phase II/III is ongoing with an interim analysis planned for mid 2008, 10/24/2007. Active Biotech has commenced enrollment for a clinical Phase I combination study of its candidate drug, ANYARA, with the cancer (chemotherapy) drug, Taxotere. The study is designed to evaluate the two drugs in combination as a treatment for non-small cell lung cancer. This dose-escalation study, expected to include between 20 and 30 patients, will examine the response to increasing doses of ANYARA in combination with a fixed dose of Taxotere. Taxotere will be administered according to the established dosing schedule; ANYARA will be dosed at levels expected to have an anti-tumor effect, 11/8/2005.

Diabetes and obesity

2008-01-23T23:30:43-04:00

Genaera announced that the investigational new drug (IND) application submitted to the US Food and Drug Administration (FDA) for trodusquemine (MSI-1436) in type 2 diabetes is now in effect. This application represents the second IND filed by the Company in 2007 for MSI- 1436. A previous IND for the treatment of obesity, under which Genaera is currently conducting phase 1 clinical trials, has been in effect since April 2007. MSI-1436 is a novel therapeutic for the treatment of type 2 diabetes and obesity which acts by regulating insulin and leptin receptor signaling through inhibition of the tyrosine phosphatase, PTP-1B. In October 2007, Genaera completed the first phase 1 study of MSI-1436 in healthy obese and overweight subjects. MSI-1436 is currently being evaluated in a second phase 1 ascending single dose study in overweight and obese type 2 diabetics (study MSI-1436C-103) under Genaera's obesity IND. Additional phase 1 studies will explore the potential of MSI-1436 in type 2 diabetics and the Company plans to pursue type 2 diabetes as its lead indication in phase 2, 1/23/2008. Genaera announced that dosing of subjects has begun in study MSI-1436C-103, the ascending single dose Phase 1 study of trodusquemine (MSI-1436), in overweight and obese type 2 diabetics. MSI-1436 is a novel therapeutic for the treatment of type 2 diabetes and obesity which works centrally and peripherally to regulate insulin and leptin receptor signaling through inhibition of its novel target enzyme PTP-1B. Study 103 is similar in design to the previously completed Study 101 which was conducted in healthy obese and overweight volunteers. The current study will continue to expand the safety database for MSI-1436 and establish the pharmacokinetics of the drug in a population of type 2 diabetics who are poorly controlled on metformin. The study will also evaluate a range of secondary endpoints including short-term insulin/glucose control indices, glucose tolerance and evaluation of insulin sensitivity, 1/17/2008. Genaera presented data from its dose-escalating Phase 1 clinical trial of trodusquemine (Study MSI-1436C-101), the Company's product candidate for the treatment of obesity. In the study, trodusquemine was found to be well-tolerated at likely clinical doses, and no serious adverse events were seen in healthy overweight and obese adults. Genaera also announced that the Company has designated 40 mg/m2 of trodusquemine as the maximum tolerated single dose for subjects in Study 101, as a result of nausea. The Company plans to move forward with evaluations of single doses of trodusquemine within the likely clinically effective range for the drug in type 2 diabetics then proceed to study the safety and pharmacokinetics of the drug in several multiple dose scenarios. Study MSI-1436C-101, the first trodusquemine Phase 1 trial and the subject of today's poster session, is a double-blind, randomized, placebo-controlled, single-group assignment safety study with seven subjects per group enrolled in sequential dose groups of healthy overweight or obese adult male and female volunteers who have a body mass index (BMI) of 27-40. Five subjects in each dosing group received trodusquemine while the other two subjects in the dosing group received placebo. Participants were studied at a single center, to evaluate the safety, tolerability and pharmacokinetics (PK) of a single intravenous dose of trodusquemine. The study began with a four day, in-house study period. Post dosing evaluations were conducted for 72 hours in-house, and follow-up visits were scheduled on days seven, 14 and 21. In this trial, trodusquemine was well tolerated, and there were no serious adverse events reported. In addition to the favorable safety profile of this study, the pharmacokinetic profile of trodusquemine showed a consistent pattern with minimal subject-to-subject variability and linearity across the range of doses studied. The Company expects to conduct its second ascending single dose Phase 1 clinical trial (Study MSI-1436C-103) of trodusquemine in obese type 2 diabetics. The trial will evaluate the PK and safety of trodusquemine in this population as well as provide the first opportunity to study short-term glucose control indices, glucose tolerance and insulin sensitivity, 10/23/2007. Genaera announced interim data from the first Phase 1 clinical study of trodusquemine (MSI-1436) for the treatment of obesity. Anthony DelConte, M.D., Vice President, Clinical Research and Development, presented "Trodusquemine: A Novel Aminosterol for the Treatment of Obesity and Related Metabolic Disorders," during the IBC 12th Annual World Congress on Drug Discovery & Development of Innovative Therapeutics, August 6-8 in Boston. The presentation summarized the clinical trial design of the single ascending dose Phase 1 study of MSI-1436 to evaluate safety and pharmacokinetics of the drug in healthy obese volunteers. Dr. DelConte provided interim data from the initial cohorts in the double-blind, randomized, placebo-controlled trial which included safety and pharmacokinetic (PK) data from 20 treated subjects and eight vehicle controls in four sequential dose groups. According to Dr. DelConte, PK profiles from the study thus far show a predictable pattern with minimal subject-to-subject variability and linearity across the range of doses studied. To date, no serious adverse events have been reported. It was emphasized, 8/8/2007. Genaera announced that dosing of subjects has begun in study MSI-1436C-101, the Phase 1, first-in-man study of trodusquemine (MSI- 1436), its novel anti-obesity compound. MSI-1436 works centrally and peripherally to regulate insulin and leptin receptor signaling through inhibition of its novel target enzyme PTP-1B. This randomized, vehicle-controlled study, being conducted at a leading obesity clinical research center in Kansas City, is enrolling healthy overweight and obese volunteers to evaluate the safety, tolerability, and pharmacokinetics of ascending single doses of intravenously administered MSI- 1436. This ascending single-dose protocol will initially enroll approximately 35 subjects and is expected to be completed in the second half of 2007, 5/17/2007. Genaera has begun enrolling subjects in the first human clinical study of trodusquemine (MSI-1436) under the Investigational New Drug (IND) application for the obesity compound submitted to the U.S. Food and Drug Administration (FDA) in March 2007. The phase 1 study will enroll healthy overweight and obese volunteers to assess the safety and pharmacokinetics of ascending single doses of trodusquemine. The Company expects to dose the first subjects by the end of May 2007. Trodusquemine is a centrally and peripherally-acting appetite suppressant and the first highly selective inhibitor of protein tyrosine phosphatase 1B (PTP1B), an enzyme target for the treatment of diabetes and obesity. Trodusquemine has produced consistent, sustainable weight loss in a variety of animal models and appears to overcome metabolic readjustment, which often limits sustained weight loss during caloric restriction. In addition, trodusquemine has shown the ability to reverse co-morbidities associated with obesity such as abnormal glucose metabolism and cholesterol elevation, 5/7/2007.