press release - Page 22 - Pappas Capital

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

Lumena Pharmaceuticals Raises $45 Million in Series B Financing

Funding to support clinical development of treatments for rare cholestatic liver diseases and nonalcoholic steatohepatitis

SAN DIEGO – March 11, 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 that it has secured $45 million in Series B financing. New Enterprise Associates (NEA) led the financing, with Adage Capital Management and RA Capital Management participating and joining existing investors Pappas Ventures, RiverVest Venture Partners and Alta Partners. Founded in 2011 by Pappas Ventures, Lumena will use the funding to advance the clinical development of LUM001, its lead product candidate, for the treatment of rare cholestatic liver disease in pediatric and adult patients, as well as LUM002 for the treatment of nonalcoholic steatohepatitis (NASH).

Lumena Pharmaceuticals - 03/11/2014

Ultragenyx Announces First Patient Enrolled in Phase 2 Study of Triheptanoin in Glucose Transporter Type-1 Deficiency Syndrome

Novato, CA, March 11, 2014 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the first patient enrolled in the Phase 2 study of triheptanoin (UX007) for the treatment of glucose transporter type-1 deficiency syndrome (Glut1 DS), at Columbia University. Glut1 DS, also known as De Vivo disease, is a rare, severely debilitating disease characterized by seizures, developmental delay, and movement disorder.

Ultragenyx Pharmaceutical - 03/11/2014

Study Finds the Corus® CAD Gene Expression Blood Test Influenced Primary Care Decision- Making in the Assessment of Patients with Suspected Obstructive Coronary Artery Disease

- IMPACT-PCP Study Results Reinforce the Clinical Utility of Corus CAD in the Primary Care Setting, Where Almost 8,000 Patients Present With Non-Acute Chest Pain and Related Symptoms Every Day -

- Clinicians Modified Their Diagnostic Strategy in 58% of Patients Following a Corus CAD Test Score -

PALO ALTO, Calif. – March 10, 2014 – CardioDx, Inc., a molecular diagnostics company specializing in cardiovascular genomics, today announced the publication of the IMPACT-PCP (Investigation of a Molecular Personalized Coronary Gene Expression Test on Primary Care Practice Pattern) trial reinforcing the value of Corus® CAD as an initial diagnostic test for the evaluation of patients presenting with non-acute symptoms suggestive of obstructive coronary artery disease (CAD) in the primary care setting. The study appears in the March 2014 issue of the Journal of the American Board of Family Medicine.

CardioDx - 03/10/2014

Marina Biotech Announces $6 MM Convertible Preferred Stock Financing and Conversion to Common Stock of the Company's Promissory Note

Company to Advance Its Preclinical and Clinical Rare Disease Programs

BOSTON, MA--(Marketwired - Feb 24, 2014) - Marina Biotech, Inc. (PINKSHEETS: MRNA), a leading oligonucleotide-based drug discovery and development company focused on rare diseases, announced today that it has entered into a binding term sheet with certain qualified investors, led by Steven T. Newby, a long-time biotechnology investor, for the issuance of convertible preferred stock at a conversion price equivalent to $0.75 per share of common stock resulting in gross proceeds of $6 million. In addition, the Company will issue to the investors warrants to purchase 6 million shares of common stock. The warrants will have an exercise price of $0.75 per share and are exercisable for a period of five years after the Company regains compliance with its reporting obligations under the Securities Exchange Act. The offering is expected to close on or about March 7, 2014, subject to the execution of a customary Securities Purchase Agreement regarding the transaction and the satisfaction of customary closing conditions. The Company also announced that the holders of the Company's Promissory Note have agreed to convert the remaining principal and interest on the Note to common stock at a conversion price of $0.75 and release their lien on the Company's intellectual property. Proceeds from the financing will be used to restart certain day-to-day operations, repay the Company's outstanding obligations, regain compliance with the Company's Exchange Act reporting obligations and advance the Company's preclinical and clinical rare disease programs.

Marina Biotech - 02/24/2014