Coronado Biosciences receives recommendation to continue Falk phase 2 trial of TSO in Crohn's disease

Coronado Biosciences, Inc. (Nasdaq: CNDO), a biopharmaceutical company focused on the development of novel immunotherapy agents for the treatment of autoimmune diseases and cancer, today announced that it received from Dr. Falk Pharma GmbH (Falk), its development partner, a recommendation from the independent data monitoring committee that conducted an interim analysis (blinded to Falk) of clinical data from the initial 120 patients to continue Falk's Phase 2 clinical trial in Europe evaluating Trichuris suisova (TSO) in Crohn's disease. The committee noted no safety concerns and a positive efficacy trend in its recommendation that the study continue. See the full release @

http://www.proactivenewsroom.com/Blog/bid/85980/Coronado-Biosciences-Announces-Independent-Data-Monitoring-Committee-Recommendation-to-Continue-Falk-Phase-2-Trial-of-TSO-in-Crohn-s-Disease

Daily Dose Updates:

• CNDO received from Dr. Falk Pharma GmbH (Falk), its development partner, a recommendation from the independent data monitoring committee that conducted an interim analysis (blinded to Falk) of clinical data from the initial 120 patients to continue Falk's Phase 2 clinical trial in Europe evaluating Trichuris suis ova (TSO) in Crohn's disease.
• The committee noted no safety concerns and a positive efficacy trend in its recommendation that the study continue.
• Falk has advised they are adopting the committee's recommendations to increase the sample size and to conduct a subsequent interim analysis at the time the trial reaches approximately 250 patients.
• The Falk trial, entitled Double-blind, randomised, placebo-controlled, multi-centre phase II study to evaluate the efficacy and safety of three different dosages of oral Trichuris suis ova (TSO) suspension in active Crohn's disease, is being conducted in Europe and was initially expected to enroll approximately 212 patients and to evaluate three different dosages of TSO versus placebo.

Northwest Biotherapeutics ($NWBO) completes significant milestones in DCVax-L program for brain cancer in Germany

Northwest Bioterapeutics (OTCBB: NWBO) joined the Fraunhofer IZI Institute in announcing that, working closely together over the course of more than 13 months, the parties have completed significant milestones in NW Bio's DCVax-L program for brain cancer in Germany.

• As a result, the company is poised to move forward in the near term in Germany with both its clinical trial program and Hospital Exemption cases under Section 4B of the German Drug Act
• A four-month long technology transfer of the manufacturing process for DCVax-L was carried out by NW Bio, its contract manufacturer, Cognate BioServices and Fraunhofer IZI. In addition, a multi-month process was completed to create the extensive documentation required to apply for manufacturing authorization in accordance with Section 13 of the German Drug Act (AMG), the application was submitted to the responsible pharmaceutical supervisory authority, and the 9-month long regulatory process has been completed up to the point of the official acceptance inspection.
• The parties are now awaiting that final inspection, which is the last step required before the manufacturing authorization can be granted and manufacturing can proceed for clinical trials and Hospital Exemption cases.
• The inspection is scheduled for June 2012.

Biotime ($BTX) demonstrates method for the manufacture of cartilage producing cells from human embryonic stem cells

BioTime (NYSE: BTX) and its wholly owned subsidiary OrthoCyte Corporation reported today a means of manufacturing cartilage from human embryonic stem cells that is suited for industrial scale-up of a product for the treatment of osteoarthritis.

• The paper, published online in the peer-reviewed journal Regenerative Medicine, characterizes a progenitor cell line produced from human embryonic stem cells using proprietary ACTCellerate technology.
• The study reports that the cells are capable of regenerating cartilage with long sought-after identification markers.
• The study also shows that the cells can be directly expanded on a scale needed for industrial manufacture, which will be necessary in order to make transplantable cells available in commercial quantities.

Achillion ($ACHN): Mixed Reaction from Analysts, UBS Downgrades

UBS comments on the full data from the phase 2 trial of ACH-1625 and sees the data as suggestive of a safety signal with LFT events that appear dose dependent.

• UBS notes a potential potency issue as well
• Target remains $7
• Analyst is Matthew Harrison

Achillion Pharmaceuticals (NASDAQ: ACHN) today announed announced at the Annual Meeting of the European Association for the Study of the Liver (EASL) International Liver Congress that in the second segment of its Phase 2a trial of ACH-1625, 94 to 100% of patients with treatment naïve genotype 1 chronic hepatitis C virus (HCV) achieved a complete early virologic response (cEVR) after 12 weeks of treatment with ACH-1625 in combination with pegylated interferon alfa-2a and ribavirin (P/R).

• ACH-1625 in combination with P/R for up to 12 weeks was safe and well tolerated and produced high viral response rates regardless of dose level or IL28B genotype status.
• Michael Kishbauch, President and CEO of Achillion, said, "As we finalize this Phase 2 trial and report on SVR later this year, we are now focused on initiating our all-oral program for the treatment of HCV. With the recent submission of an IND for ACH-3102, our second-generation NS5A inhibitor, we look forward to initiating its Phase 1 program this quarter and rapidly advancing toward a therapeutic, interferon-free combination trial evaluating ACH-1625 plus ACH-3102 during Q4 of this year."
• In Segment 2 of this Phase 2a trial, a total of 58 subjects with HCV were enrolled, randomized and stratified by IL28B genotype, including CT and TT, which is a marker of a patient's responsiveness to interferon, to receive one of three doses of once-daily ACH-1625 (200 mg, 400 mg or 800 mg) in combination with P/R for 12 weeks of therapy.
  • Of the patients enrolled, the majority had HCV genotype 1a (n=35 (60%)), with remaining patients having HCV genotype 1B (n=20) or genotype 1 (n=3). Approximately 71% of the patients were IL28B genotype CT/TT, the more difficult to treat mutation, 64% were male and 17% were African American. No viral breakthroughs were observed during treatment. Results demonstrated rapid virological response (RVR) at week 4, cEVR and end of treatment (EOT) responses for patients returning for EOT visit to date can be viewed in a table here.
  • Safety results from both segments of the trial were similar to those observed and previously reported during clinical trials of ACH-1625.
• In a separate poster presentation, the clinical virology of NS3 variants from patients enrolled in Segment 1 of the Phase 2a clinical trial evaluating multiple ascending doses of ACH-1625 in combination with P/R was presented.
  • Sequencing of baseline to post-treatment samples revealed that there were no mutations at loci 155, 156, or 168 of NS3 protease, which represent common mutations that may confer resistance to protease inhibitors.

BioTime ($BTX) : R&D Day in NYC today

BioTime Investor Day

BioTime, Inc. is pleased to invite you to attend a unique forum featuring in depth presentations from the Company’s key collaborators and subsidiaries. A keynote address will be presented by BioTime and OncoCyte Board member, Andrew von Eschenbach, M.D., former FDA Commissioner and Director of the National Cancer Institute.  

Monday, April 23, 2012
2:00PM – 6:15PM with cocktail reception to follow

Harvard Club of New York City, Biddle Room
27 W.44th Street, New York, NY (between 5th & 6th Avenues)

PRESENTING SUBSIDIARIES:

• Cell Cure NeuroSciences, Ltd. - Leading supplier of therapeutic cells for the treatment of retinal and neural degenerative disease. OpRegen™ cell therapy platform utilizes proprietary technology that drives differentiation of human embryonic stem cells into retinal pigment epithelial (RPE) cells.
• OncoCyte Corporation – Oncology-focused diagnostics company leveraging embryonic stem cell-derived technology in order to provide earlier detection and improve patients’ quality and length of life by specifically removing malignant tumors without affecting nearby normal body tissue.
• OrthoCyte Corporation – Developer of cellular therapeutics for orthopedic disorders. The division’s lead project is the development of human embryonic progenitor (hEP) cell lines for cartilage repair, including osteoarthritis. 
• ReCyte Therapeutics, Inc. – Developer of therapeutics for cardiovascular and blood diseases based on its ReCyte™ iPS technology. This platform reverses the developmental aging of human cells and generates embryonic vascular and blood progenitors from the ReCyte cell lines for therapeutic use in age-related vascular and blood disorders, such as coronary disease and heart failure. 
• LifeMap Sciences, Inc. – Developer of a discovery platform, including a web-based database, to aid R&D efforts in the stem cells field, using embryonic stem cells, progenitor cells, induced pluripotent stem cells and other relevant cell types.
• BioTime Asia, Ltd. – Developer and marketer of BioTime stem cell technology within Asian geographies, with a particular focus within China. The Asian region offers significant developmental advantages, such as dense patient populations, flexible regulation and public acceptance, and abbreviated clinical application timelines.

Reserverlogix (RVX.CN) : Announces Mechanism of Action for RVX-208

• RVX.CN announced that RVX-208 increases apolipoprotein A-I (ApoA-I) production and that its data shows RVX-208 to be an inhibitor of the Bromodomain and Extraterminal Domain (BET) proteins.
• RVX-208 acts on BET proteins, including BRD4, a member of the BET-protein family, leading to increased transcription of the ApoA-I gene followed by production of more ApoA-I protein.
• Clinical benefits of RVX-208 are currently being explored in two concurrent Phase 2B clinical trials (SUSTAIN and ASSURE), led by Cleveland Clinic.
• As previously announced, Conference Call and Webcast today at 11ET: 800.319.4610 or 604.638.5340

Keryx ($KERX) Biopharmaceuticals announces positive top-line results from phase 3 study of ferric citrate (Zerenex) in Japan

Keryx Biopharmaceuticals (NASDAQ: KERX) announced today that its Japanese partners, Japan Tobacco (2914.JP) and Torii Pharmaceutical (4551.JP) have announced positive top-line results from a Phase 3 study of ferric citrate (Zerenex) in Japan for the treatment of hyperphosphatemia in end-stage renal disease patients on hemodialysis. This study is part of an ongoing Phase 3 program for ferric citrate in Japan for the treatment of hyperphosphatemia.

• The Phase 3 study, conducted in Japan, evaluated the efficacy and safety of ferric citrate against an active control, sevelamer hydrochloride, over 12 weeks in hemodialysis patients with hyperphosphatemia.
• In the top-line results, which evaluated the change of serum phosphorus from baseline, the primary endpoint of efficacy met non-inferiority to sevelamer hydrochloride. Furthermore, there were no clinically significant findings on safety and tolerability of ferric citrate within the treatment period.
• JT/Torii stated that it is aiming to submit the marketing application for ferric citrate in Japan in the fiscal year ending March 31, 2013.
• Zerenex is also in a Phase 3 clinical program in the United States for the treatment of hyperphosphatemia (elevated phosphate levels) in patients with end-stage renal disease on dialysis, which is being conducted pursuant to a Special Protocol Assessment agreement with the FDA.

PolyMedix, Inc ($PYMX) announces topline results from phase 2 study evaluating PMX-30063

PolyMedix (OTCBB: PYMX) announced topline results from a Phase 2 proof-of-concept study evaluating PMX-30063, a first-in-class investigational antibiotic, for the treatment of Acute Bacterial Skin and Skin Structure Infections (ABSSSI) caused by Staph aureus.

• The study objectives were met, demonstrating clinical efficacy and safety in all evaluated doses of PMX-30063.
• In the study, patients were first evaluated at day 3 for clinical response using FDA's most recent ABSSSI Guidance. Patients receiving low, medium, or high doses of PMX-30063 experienced high clinical response rates at Day 3.
• Clinical Response at Day 3
  • Per Protocol: Low Dose 85.0%; Medium Dose 71.4%; High Dose 89.7%; Daptomycin 74.5%
  • mITT: Low Dose 81.4%; Medium Dose 67.6%; High Dose 77.8%; Daptomycin 74.5%
  • ITT: Low Dose 79.6% Medium Dose 68.5%; High Dose 75.9%; Daptomycin 75.5%
• All regimens of PMX-30063 for all patient populations and time points showed early, high and sustained clinical responses. The 95% confidence intervals for the day 7, 10 and 28 assessments illustrate the consistency of clinical responses for all dosing arms of PMX-30063 compared to active control.
• In the study, PMX-30063 appeared to be safe and was generally well-tolerated. As expected and consistent with previous clinical studies, patients receiving PMX-30063 commonly reported sensations of numbness and tingling that were generally characterized as mild and resolved following treatment. No patient stopped treatment as a result of these sensations. Other treatment-related adverse event rates were similar across all treatment arms.
• There was one treatment-related serious adverse event that was at least possibly drug-related reported in each PMX-30063 study arm. Treatment-related serious adverse events included an instance of hypertension in the medium and high dose regimens, which discontinued therapy, and an instance of increased platelets in the low dose regimen.
• As previously announced, PolyMedix plans to discuss the results of this PMX-30063 Phase 2 clinical trial on its conference call to be held today at 9:00 am Eastern Time and to present study details, including microbiologic data, at a future medical meeting(see linked comment).

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