press release - Page 23 - Pappas Capital

Thrasos Presents Preclinical Data Demonstrating THR-184 Prevents Loss of Kidney Function Following Acute Ischemic Injury

Results presented at the ERA-EDTA congress led to initiation of ongoing Phase 2 clinical study in patients undergoing cardiac surgery

MONTREAL, June 3, 2014 – Thrasos Therapeutics, a biotherapeutics company focused on delivering new solutions for kidney disease, today presented preclinical results showing that its lead development compound THR-184 can effectively protect against loss of kidney function following acute ischemic injury in rat. Results of the studies were presented at the 51st European Renal Association - European Dialysis and Transplant Association (ERA-EDTA) Congress taking place in Amsterdam, The Netherlands.

Thrasos Therapeutics - 06/03/2014

Plexxikon Announces Promising Preliminary PLX3397 Phase 1 Extension Data in Patients with Pigmented Villonodular Synovitis (PVNS)

79 Percent of Patients Achieved Partial Response; 21 Percent Had Stable Disease with Targeted Therapy PLX3397

BERKELEY, Calif., May 14, 2014 (BUSINESS WIRE) -- Plexxikon, a member of the Daiichi Sankyo Group, announced today promising, proof-of-concept Phase 1 extension clinical data with PLX3397 in pigmented villonodular synovitis (PVNS), a type of rare, often locally aggressive, musculoskeletal neoplasm that arises from the soft tissues of joints and tendons. Interim data from this ongoing trial show that all evaluable patients treated with PLX3397 achieved either partial responses or stable disease. PLX3397 is a novel, oral small molecule that potently and selectively inhibits CSF1R, KIT and oncogenic FLT3 kinases, which play important roles in cancer. CSF1R, in particular, has been shown to be a primary driver in PVNS. These data are being released today as part of the American Society of Clinical Oncology (ASCO) 50th Annual Meeting Press Program. More detailed data will be presented at the ASCO 50th Annual Meeting, being held May 31-June 3 in Chicago.

Plexxikon - 05/14/2014

Shire adds to rare disease portfolio with acquisition of Lumena Pharmaceuticals, bringing late stage compounds for rare GI/hepatic conditions

Acquisition of Lumena Pharmaceuticals, a biopharmaceutical company with late stage rare disease pipeline assets
Adds to Shire's rare diseases portfolio and leverages this expertise, and is a perfect combination with Shire's already strong Gastrointestinal (GI) presence
Adds LUM001 in Phase 2 development for four rare and devastating hepatic diseases, two pediatric and two adult with a potential 2016 approval and LUM002 a Phase 2-ready candidate for the treatment of non-alcoholic steatohepatitis (NASH)
Very attractive opportunity to develop treatments for significant unmet need in rare cholestatic liver diseases as well as a treatment for non-alcoholic steatohepatitis (NASH)
Shire will acquire Lumena Pharmaceuticals for an upfront payment of $260 million in cash, plus a payment for net cash at closing, and near-term contingent milestone payments related to ongoing clinical trials
These two compounds, and Shire's full portfolio, will be discussed in more detail at an Investor Day later in 2014.

Dublin, Ireland and San Diego, U.S. – May 12th, 2014 – Shire plc (LSE: SHP, NASDAQ: SHPG) and Lumena Pharmaceuticals, Inc., a biopharmaceutical company with rare disease pipeline assets, announce the acquisition of Lumena Pharmaceuticals by Shire.

Lumena Pharmaceuticals - 05/12/2014

TESARO Announces Successful Achievement of Primary and All Secondary Endpoints in Third and Final Phase 3 Trial of Rolapitant

Achieved Primary Endpoint of Complete Response (CR) in the Delayed Period (24 to 120 Hours) Following Initiation of Chemotherapy

Achieved Key Secondary Endpoints of CR in the Acute and Overall Periods

Achieved All Secondary Endpoints, Including No Significant Nausea

Adverse Event Profile Consistent with Earlier Clinical Trials

New Drug Application (NDA) Submission to U.S. FDA On Track for Mid-2014

WALTHAM, Mass., May 12, 2014 (GLOBE NEWSWIRE) -- TESARO, Inc. (Nasdaq:TSRO), an oncology-focused biopharmaceutical company, today announced positive top-line results from the third and final Phase 3 trial of rolapitant, an investigational neurokinin-1 (NK-1) receptor antagonist in development for the prevention of chemotherapy-induced nausea and vomiting (CINV). The rolapitant arm in this trial, which enrolled patients receiving cisplatin-based, highly emetogenic chemotherapy (HEC), successfully achieved statistical significance over the standard therapy arm for the primary and all secondary endpoints. The adverse event profile for rolapitant remains consistent with that seen in previous clinical studies.

TESARO - 05/12/2014

Lumena Pharmaceuticals Now Dosing Patients in the INDIGO Phase 2 Clinical Trial of LUM001 in Pediatric Patients with Progressive Familial Intrahepatic Cholestasis

CAMEO Phase 2 Clinical Trial of LUM001 Now Enrolling Adults with Primary Sclerosing Cholangitis

SAN DIEGO – May 9, 2014 – Lumena Pharmaceuticals (Lumena), a biopharmaceutical company focused on the development and commercialization of novel products for rare cholestatic liver diseases and serious metabolic disorders, today announced the dosing of the first patient in the INDIGO Phase 2 clinical trial of its lead drug candidate, LUM001, in children with progressive familial intrahepatic cholestasis (PFIC). Additionally, the CAMEO Phase 2 clinical trial of LUM001 in adults with primary sclerosing cholangitis (PSC) is open for enrollment.

Lumena Pharmaceuticals - 05/09/2014

CoLucid Pharmaceuticals, Inc., Announces Agreement from the FDA on a Special Protocol Assessment (SPA) for the Phase 3 Trial of Lasmiditan in Migraine Including Patients with Risk Factors for Cardiovascular Disease

Durham, NC, MAY 6, 2014/PRNewswire/ -- CoLucid Pharmaceuticals, Inc., a privately held biopharmaceutical company, has reached agreement with the U.S. Food and Drug Administration (FDA) for the planned SAMURAI study. The objective of this trial is to evaluate the safety and efficacy of two doses of Lasmiditan in comparison to placebo for the treatment of acute migraine. Patients with risk factors for cardiovascular disease will be included in the study.

The SPA agreement includes two novel endpoints for the approval of acute migraine therapies—a primary endpoint of the proportion of patients who are free of headache pain at 2 hours and a key secondary endpoint of the proportion of patients who no longer suffer from their most bothersome associated symptom of migraine (nausea, photophobia, phonophobia) at 2 hours.

CoLucid Pharmaceuticals - 05/06/2014

New Study Results Further Reinforce that the Corus® CAD Test Helps Primary Care Clinicians Make Effective Referral Decisions for Patients with Suspected Obstructive Coronary Artery Disease

- Patients with a Low Corus CAD Score had 94% Reduced Odds of Referral to a Cardiologist -

- REGISTRY I Study Results Add to the Established Evidence Base Supporting the Clinical Utility of the Corus CAD Gene Expression Test in Real-World Clinical Practice -

PALO ALTO, Calif. – [May 05, 2014] - CardioDx, Inc., a molecular diagnostics company specializing in cardiovascular genomics, today announced the publication of the REGISTRY I study, which examined the assessment of non-acute chest pain and related symptoms that may be suggestive of obstructive coronary artery disease (CAD) in the primary care setting with the Corus® CAD blood-based gene expression test. The study, published online in the American Journal of Medical Quality in May 2014, found a strong association between cardiac referral rates by low (≤15) and elevated (>15) Corus CAD score groups, with only 6% (10/167) of the low Corus CAD score patients versus 70% (122/175) of the elevated Corus CAD score patients being referred for further cardiac evaluation (P < .0001). Corus CAD is the first and only commercially available blood-based gene expression test that provides a current-state assessment of obstructive CAD in non-diabetic patients presenting with typical or atypical symptoms. With a 96% negative predictive value and 89% sensitivity, Corus CAD can help clinicians accurately rule out obstructive CAD as the source of their patients’ symptoms, so that they may investigate other non-cardiac causes.

CardioDx - 05/05/2014

Ultragenyx Announces Positive Data From Phase 2 Study of Sialic Acid Extended-Release at Emerging Sciences Session of American Academy of Neurology Annual Meeting

Upper Extremity Muscle Strength Preserved Over 48 Weeks in HIBM Patients on 6 Grams
NOVATO, Calif., April 30, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of detailed results from a 48-week Phase 2 clinical study of sialic acid extended-release (SA-ER, UX001) tablets in 47 patients with hereditary inclusion body myopathy (HIBM; also known by its new name as GNE myopathy), a rare, progressive muscle-wasting disease. SA-ER is designed to replace the deficient sialic acid substrate in patients with HIBM.

Ultragenyx Pharmaceutical - 04/30/2014

MUSC Foundation for Research Development welcomes Art Pappas to board

Pappas to share venture capital expertise in pharmaceutical and life sciences industries

CHARLESTON, SC - April 23, 2014 - The Medical University of South Carolina Foundation for Research Development (FRD) has announced that venture capitalist Art Pappas has joined the organization’s Board of Directors, effective immediately.

Uncategorized - 04/23/2014

Ultragenyx Announces Preliminary Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7

Data Presented at American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting

NOVATO, Calif., March 27, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of preliminary data from the Phase 1/2 study of recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of mucopolysaccharidosis 7 (MPS 7, Sly syndrome).

Ultragenyx Pharmaceutical - 03/27/2014