• Amicus Therapeutics, of Cranbury, N.J., announced positive results from two recently completed Phase I clinical studies of AT2220 (1-deoxynojirimycin HCl) for Pompe disease. The results show that AT2220 was well-tolerated in healthy volunteers with good oral bioavailability and pharmacokinetic parameters. AT2220 is designed to selectively bind to and stabilize acid a-glucosidase (GAA), the enzyme deficient in Pompe disease. These results will be presented at the American Society of Human Genetics Annual Meeting in San Diego.

• Can-Fite BioPharma Ltd., of Petach Tikva, Israel, will conduct a confirmatory Phase IIb trial as part of the ongoing development of CF101 for the treatment of rheumatoid arthritis. In July, Can-Fite published the results of a Phase IIb study with CF101 in combination with Methotrexate indicating that the ACR20 response, which was the primary efficacy end point of the study, showed no difference between the CF101-treated and placebo groups. However, a substantial difference in favor of CF101 was seen in the ACR50, the ACR70 and the EULAR. Can-Fite estimates that the trial will be initiated in the first quarter of 2008 and may take about one year to complete, including data analysis.

• Dendreon Corp., of Seattle, has completed enrollment of over 500 patients in the Phase III IMPACT (IMmunotherapy for Prostate AdenoCarcinoma Treatment, also known as D9902B) clinical trial of Provenge (sipuleucel-T), its investigational active cellular immunotherapy for advanced prostate cancer. The study is a double-blind, randomized, placebo-controlled trial to measure overall survival in men with metastatic hormone-refractory prostate cancer receiving Provenge versus placebo. Earlier this year, following a positive recommendation from an outside panel of experts, Dendreon received a complete response letter from the FDA that asked for additional evidence supporting the efficacy of Provenge. Subsequently, the FDA said it will accept either a positive interim or positive final analysis of overall survival from the IMPACT study to amend the biologics license application and support the efficacy claim for Provenge. An interim analysis for overall survival is expected to be performed in the second half of 2008.

• Dynavax Technologies Corp. has begun dosing of Tolamba , its novel ragweed allergy therapy, to subjects as part of an environmental exposure chamber study. A total of 300 subjects are expected to participate. Subjects are being screened based on a history of ragweed allergy and a positive skin test. Subjects will be exposed to ragweed allergen in the chamber to confirm their ragweed allergic disease and establish their baseline level of symptoms. They will be re-exposed in the chamber to determine the effect of the six-week, six-injection Tolamba regimen as compared to placebo. Data from this study are expected in the first half of 2008 and, if positive, will be followed by a pivotal field study in the 2009 ragweed season, with a potential BLA submission planned for 2010.

• Hyperion Therapeutics Inc., of South San Francisco, has enrolled the first patient in a Phase I/II trial of Glyceryl Tri (4-Phenylbutyrate) (GT4P) in patients with urea cycle disorders (UCDs). The study will evaluate the safety, tolerability and ammonia scavenging effects of GT4P compared to Buphenyl (sodium phenylbutyrate) in patients with UCDs. GT4P is a prodrug of Buphenyl. Up to 10 adults with stable UCD will be included in the trial, which is being conducted at U.S.-based clinical sites. Treatment is expected to last three to four weeks.

• MGI Pharma Inc., of Minneapolis, said results of a randomized, double-blind, multi-center, pivotal Phase III trial of Aquavan (fospropofol disodium) Injection in patients undergoing flexible bronchoscopy showed it met its primary and secondary endpoints. A total of 252 patients were randomized and received either a 6.5 mg/kg dose of Aquavan or a control dose of 2.0 mg/kg. Among patients treated with an initial bolus dose of 6.5 mg/kg (n=150) of Aquavan, the sedation success rate was 88.7% compared to 27.5% of patients in the control arm (n=102) (p<0.001). The treatment success rate among patients treated with the initial bolus dose of 6.5 mg/kg Aquavan was 91.3% compared to 41.2% for control (p<0.001). Of those patients in the 6.5 mg/kg Aquavan arm, 94.6% indicated that they would be willing to be treated again with the same sedative dose, compared to 78.2% of those in the control arm (p<0.001). Additionally, 83.3% of patients in the 6.5 mg/kg Aquavan arm reported that they did not recall being awake during the procedure, compared to 55.4% for control (p<0.001). The data were included in a new drug application submitted to the FDA on Sept. 27.

• Morria Biopharmaceuticals plc, of London, has initiated a Phase I tolerance study of MRX-4 in 16 patients suffering from allergic rhinitis. The randomized, placebo-controlled, double blind clinical trial is the first of two modules focusing on safety for Morria's first human study in AR. MRX-4 is nasally administered as a single dose either with or without an allergen challenge to determine tolerance. Results are expected by the end of the fourth quarter of 2007.

• Speedel Holding Ltd, of Basel, Switzerland, will resume development of SPP301 (avosentan) as a potential breakthrough therapy for diabetic kidney disease. A pivotal Phase III clinical ASCEND trial was stopped in December 2006 from concerns about patient safety based on a recommendation from the Data Safety Monitoring Board, following a significant imbalance in fluid retention in patients among the study arms (placebo, 25mg per day, 50mg per day of SPP301 on top of standard therapy). SPP301 is an endothelin A receptor antagonist, a class of drug known to have an effect on fluid retention. But after analyzing data from the halted study, plus data from a new study of how SPP301 affects fluid retention in human volunteers and ongoing preclinical and technical assessments, Speedel plans to start a new Phase IIb dose finding study with SPP301 in 2008. The company also has decided not to continue development on its own of SPP200 (pegmusirudin) for patients undergoing haemodialysis, and is now in active discussions to partner the asset.

• Summit Corp. plc, of Oxford, UK, has begun a Phase I clinical trial of SMT D002 for the treatment of seborrhoea (excessive sebum production), a symptom of Parkinson's disease and the primary cause of acne. The recruitment of 18 healthy volunteers for the trial is complete and dosing is underway. Treatment is expected to last for five weeks. The study will examine the effects of repeat oral doses of SMT D002 on sebum excretion rates. An earlier double-blind Phase I study in healthy volunteers using a single oral dose showed a 70 percent reduction in sebum excretion. Summit expects to report the results from this new study during first quarter of 2008.

• Yale Cancer Center, of New Haven, Conn., said researchers have begun recruiting 60 men for a clinical trial investigating an experimental new drug, oral phenoxodiol, as a potential first line therapy for prostate cancer. All patients will receive 400 mg of oral phenoxodiol every eight hours daily for 28 consecutive days (1 cycle). Treatment outcome will be evaluated after three cycles (12 weeks). Patients with disease progression will be taken off the study. Responding and stable disease patients will remain on study until disease progression or for a maximum of 12 cycles, approximately 12 months. The primary endpoint is to determine the proportion of patients given phenoxodiol that have a 50 percent post-therapy prostate specific antigen (PSA) decline at 12 weeks in patients with androgen independent disease who are chemotherapy naïve and rising PSA after radical prostatectomy or radiotherapy that are androgen dependent.