Assistant Managing Editor Proving that big pharma firms aren't the only ones interested in the orphan disease space, venture investors are getting behind 2010 start-up Ultragenyx Pharmaceutical Inc...Funds will be used to prepare and submit a new drug application for lead product lomitapide in homozygous familial hypercholesterolemia (HoFh), complete clinical and nonclinical studies in HoFH, advance development of the drug in pediatric and adolescent patients and for general corporate purposes

Ambit Biosciences Corp., of San Diego, and partner Astellas Pharma Inc., of Tokyo, reported data from an interim analysis of a Phase II study of AC220, a selective FLT3 inhibitor, as a once-daily monotherapy in acute myeloid leukemia patients with FLT3-ITD activating mutations who have relapsed or are refractory to other treatments...Clinical response data was reported for 53 patients and showed a composite complete remission rate (CRc) of 45 percent, with the majority of CRc cases represented by

Although Oliver Fetzer, president and CEO of Cerulean Pharma Inc., said he thinks the existing regulatory framework is robust enough to evaluate the safety and efficacy of nanopharmaceuticals, he is encouraged by the thoughtful approach the FDA is taking in providing clarity for this innovative technology...A biotech on the leading edge of nanotechnology, Cerulean, of Cambridge, Mass., has been interacting with the FDA in bringing its nanopharmaceuticals into clinical development...The revision

The drug was previously included as part of a six-year option agreement with GlaxoSmithKline plc, but the big pharma passed on the compound in 2008...The South San Francisco-based firm raised $165 million a few months later to push cabozantinib through clinical trials. (See BioWorld Today, Dec. 6, 2010, and March 11, 2011.) The results of initial trials seemed to garner mixed feedback at ASCO...Astellas Pharma Global Development Inc., of Deerfield, Ill., said AGS-1C4D4 reduced tumor formation and

It was a really big discovery and generated a lot of interest in big pharma," he added...Proceeds will allow the firm to advance product candidates in its portfolio toward clinical development...Proceeds are expected to help complete the ongoing 240-patient trial of DCVax-L for the treatment of glioblastoma multiforme and advance other programs in the pipeline.

Adeona Pharmaceuticals Inc., of Ann Arbor, Mich., announced that analyses of a clinical study evaluating reaZin, a gastroretentive, sustained-release, once-daily oral tablet formulated from zinc (150 mg) and amino acid cysteine (100 mg) for treatment of Alzheimer's disease, indicated that the older the reaZin treatment patient, the greater the amount of cognitive benefit compared to the placebo patients of the same age...Celgene Corp., of Summit, N.J., and Acceleron Pharma Inc., of Cambridge, Mass

We have a network of pharma experts who all told us, if it was their compound it would be given the green light to go into clinical development...We'll get clinical proof of concept and then think about it...The company said it will use the funds to complete Phase IIa studies with lead Smac mimetic drug candidate, TL32711, in solid tumors and to initiate clinical studies in hematological cancers.

That drug also is part of the large AstraZeneca collaboration, and the big pharma firm is positioning TC-5214 as an add-on therapy in MDD, which would pit it against Abilify (aripiprazole, Bristol-Myers Squibb Co...Proceeds will be used to fund clinical trials, working capital and other general corporate purposes. (See BioWorld Today, May 19, 2011.)

Based on those difficulties, in addition to the need to sharpen the toxicology profile and a potential requirement for an altered dosage, the companies opted to terminate clinical development of EVT 101...Pharma Two B, of Rehovot, Israel, said it is planning a Phase II study of combination therapy P2B001, in development to treat the early stages of Parkinson's disease

The New York-based big pharma, which paid $50 million in cash and equity up front and would have owed up to $390 million more in milestones, cited reprioritization as its reason to return rights last fall, despite the drug's impressive Phase II data in GBM. (See BioWorld Today, Sept. 7, 2010.) Analysts have estimated that the Phase III rindopepimut program could run Celldex about $50 million...Antares Pharma Inc., of Ewing, N.J., closed an underwritten public offering of 12.5 million shares of

VistaGen's platform, known as Human Clinical Trials in a Test Tube, actually combines several stem cell technologies, including those developed by Gordon Keller, who heads up VistaGen's scientific advisory board and works out of a lab at the University Health Network's McEwen Centre for Regenerative Medicine in Toronto...It also has a clinical-stage candidate, AV-101, an oral prodrug designed to convert in the brain into active metabolite 7-chlorokynurenic acid, an agonist of N-methyl-D-aspartate

Chimerix Inc., of Durham, N.C., presented clinical data at the International Conference on Antiviral Research in Sofia, Bulgaria, showing that the majority of immunocompromised patients with severe adenovirus infections who were treated with Chimerix's lead antiviral compound, CMX001, had a greater than 99 percent decrease in viral load compared to baseline after two weeks of therapy...The trials will enroll a total of 1,700 subjects at 72 clinical sites, and are expected to conclude in 2012

A.P. Pharma Inc., of Redwood City, Calif., will raise $4.5 million in convertible notes in a private placement deal with certain investors, including Tang Capital Partners...In other news, A.P. Pharma said it met with the FDA March 31 to discuss a complete response letter regarding its new drug application for APF530 for chemotherapy-induced nausea and vomiting...The discussion topics included dosing system, clarification of assays for manufacture and quality control, and a QT/metabolism clinical

raised $77 million in a Series G to finance its full house of five clinical-stage cancer candidates, plus a trio of preclinical candidates and multiple research-stage programs...Merrimack's first major pharma deal, in 2009, focused on MM-121...Net proceeds are expected to be $10.9 million.

Ascletis anticipates advancing those programs through clinical proof-of-concept and then finding a big pharma partner to handle late-stage work and worldwide commercialization...The specialty pharma firm anticipates selling 5 million shares priced between $14 and $16 per share

Acacia Pharma, of Cambridge, UK, closed a $10 million financing round, with new investor Lundbeckfond Ventures joining Gilde Healthcare in the Series A...Proceeds will fund the company's acquisition of intellectual property from Inovio Pharmaceuticals Inc., of Blue Bell, Pa., and to advance its clinical development pipeline

The two trials were initiated in France in October 2010 and conducted by Forenap Pharma, part of the Forenap Groupe, of Rouffach, France...Medivation Inc., of South San Francisco, and Tokyo-based Astellas Pharma Inc...Among PPI-461 recipients there have been only transient clinical adverse events with no dose-related or treatment-related patterns of specific adverse events or laboratory abnormalities

And it suffered some tough breaks in 2008, including a temporary hold on then-lead clinical program mocetinostat (MGCD0103) and the termination of its partnership with Celgene Corp...The Met target in particular has been linked to tumor cell survival, and MethylGene has benefited from promising data from competitor Exelixis Inc., which wowed investors last year with its Met inhibitor XL184 in prostate cancer. (See BioWorld Today, Nov. 19, 2010.) MethylGene plans initially to target non-small-cell

FibroGen Inc., of San Francisco, and partner Astellas Pharma Inc., of Tokyo, moved FG-4592, a hypoxia-inducible factor prolyl hydroxylase inhibitor, into Phase IIb testing in anemia in chronic kidney disease (CKD) patients both on dialysis and not on dialysis...clinical benefit as assessed by three measures: a validated clinician scale, patient-reported outcome and magnetic resonance imaging measuring SMF volume