If certain milestones are met, the compounds will advance to clinical feasibility studies or proof-of-concept trials

Hyperion Therapeutics Inc., of South San Francisco, said population pharmacokinetic (PK) modeling and dosing simulations from Ravicti (glycerol phenylbutyrate) and Buphenyl (sodium phenylbutyrate) were reported in The Journal of Clinical Pharmacology based on data from four Phase II and III trials that enrolled patients with urea cycle disorders (UCDs

The combination has demonstrated early signals of activity with a disease control rate (complete response, partial response, minimal response or stable disease) of 82 percent and a clinical benefit rate (>/= minimal response) of 53 percent, including one complete response...In a single-agent Phase II ARRY-520 clinical study, the median overall survival was reported to be markedly longer in patients with low AAG as compared to patients with high AAG (20.2 vs. 4.5 months...Incyte Corp., of Wilmington

and Regado Biosciences Inc. – prepared to price IPOs this week, a fourth, Onconova Therapeutics Inc., filed a registration statement with the SEC, seeking to raise up to $75 million. (See BioWorld Today, May 1, 2013, May 17, 2013, and May 20, 2013.) Onconova filed its S-1 Friday evening after the market's close, hours after a similar filing by Conatus Pharmaceuticals Inc. (See BioWorld Today, June 17, 2013.) In its S-1, Onconova said it plans to use the proceeds to fund the clinical

Of 15 heavily treated intermediate or high-risk patients with myelodysplastic syndrome, six achieved a clinical response with a median duration of 92 days...The company said the updated results showed promise for clinical activity of the drugs in heavily pretreated MDS patients and confirmed demethylation activity of the medicine

Astex Pharmaceuticals Inc., of Dublin, Calif., presented updated clinical results of its hypomethylating agent, SGI-110, in a poster session at the 18th Congress of the European Hematology Association in Stockholm, Sweden...The update focused on details of the biological and clinical activity as well as safety in the group of intermediate or high-risk relapsed or refractory myelodysplastic syndromes patients treated in the dose-escalation, Phase I part of the SGI-110-01 study...Of the 15 patients, six

Meanwhile, Gary Burgess, senior vice president of clinical research and chief medical officer, joined Conatus in 2011 from Pfizer, where he oversaw the assimilation of Idun, including data integration, clinical oversight of ongoing Phase II studies, development of clinical plans and registration strategies for emricasan...In all, emricasan has completed nine clinical studies, including some at Pfizer, enrolling more than 500 participants, according to Conatus

Genmab A/S, of Copenhagen, Denmark, said teprotumumab (RG1507), an antibody developed by Genmab under a collaboration with Roche AG, of Basel, Switzerland, will restart clinical development in a Phase II study in patients with active thyroid eye disease...Clinical development will be conducted by River Vision Development Corp., which licensed teprotumumab from Roche...The company recently launched the Crohn's disease clinical trial and is actively recruiting participants with moderate to severe

The study demonstrated the superiority of ozenoxacin 1 percent cream, applied topically for five days, compared to placebo on both clinical and bacteriological endpoints by end-of-therapy visit (day six to seven

The new language makes it clear that "sponsors may have to demonstrate clinical superiority to obtain orphan-drug exclusivity even if they did not have to submit a plausible hypothesis of clinical superiority to obtain...Comments on the list and proposed rule are due by Aug. 12.

The compound is currently undergoing early stage clinical trials for the treatment of leukemia and solid tumor cancer, with rights to all oncology applications licensed to its partner, Eisai Co...And although full terms of the Series C funding round were not disclosed, they did include the hitting of milestones on the fibrosis clinical trial, according to PRISM Pharma CFO, Dai Takehara...The offering is expected to close on June 17.

Shares of Clovis (NASDAQ:CLVS), which jumped 100 percent earlier this month on data presented at the American Society of Clinical Oncology meeting in Chicago, closed Monday at $73.15, up $1.32. (See BioWorld Today, June 4, 2013

With data from the first clinical trial of anti-TNF polyclonal antibody AVX-470 in ulcerative colitis (UC) due toward the end of this year, Avaxia Biologics Inc...In May, it inked an extension to its deal with Galapagos NV to extend the clinical development of oral JAK1 inhibitor GLPG0543 into Crohn's disease...Exploratory endpoints in the study are looking for clinical efficacy and inflammation biomarkers

Vanda Pharmaceuticals Inc., of Washington, presented additional entrainment and patient-level clinical data at SLEEP 2013, the meeting of Associated Professional Sleep Societies in Baltimore, from its SET (Safety and Efficacy of Tasimelteon) and RESET (Randomized-withdrawal study of the Efficacy and Safety of Tasimelteon to treat non-24-hour disorder) Phase III studies of tasimelteon, a circadian regulator...In the SET study, tasimelteon achieved the primary endpoints of entrainment (synchronizing

It will use the proceeds to identify clinical-stage drugs that might have activity against LAM and to begin clinical trials

Funds will support the launch of bowel drug Linzess (linaclotide) in the U.S., and fund development to strengthen the clinical profile of Linzess and expand the product label...Proceeds will be used to fund clinical trials, R&D for additional alpha-1 antitrypsin (AAT) indications, expanding distribution capabilities for additional territories for marketing AAT product Glassia and for general corporate purposes

The National Institute of Allergy and Infectious Diseases awarded Duke University $2 million to initiate a clinical research network focused on antibacterial resistance...The group is expected to conduct early stage clinical evaluation of new antibacterial drugs and trials of approved antibacterials to reduce the risk of resistance

The financing is expected to fund the lead program to clinical proof of concept for at least one hearing disorder indication. (See BioWorld Today, Aug. 23, 2011

The Clinical Global Impression-Change (CGI-C) rating, a subjective physician-completed overall improvement scale, showed that at one and two weeks of treatment, 88 percent and 76 percent of subjects improved on N05 vs. 27 percent and 39 percent, respectively, on placebo (p < 0.001, p = 0.0016, respectively...No safety concerns relating to any of the aforementioned products have been identified in any preclinical and clinical data generated to date and there is no reason to believe that the