Lisa Dreyer - Page 29 - Pappas Capital

Lumena Pharmaceuticals Launches Global Clinical Program to Evaluate Safety and Efficacy of LUM001 in Children with Alagille Syndrome

First patient dosed in U.K. in Phase II IMAGO Study; Phase II ITCH Study to start in U.S.

Cooperative Research and Development Agreement established with the National Institutes of Health and the Childhood Liver Disease Research and Education Network (ChiLDREN)

SAN DIEGO – September 26, 2013 – Lumena Pharmaceuticals, a company developing oral therapeutics for rare liver diseases, today announced the initiation of a global clinical program to evaluate its drug candidate LUM001 in children with Alagille syndrome (ALGS). The first patient has been dosed in the IMAGO Phase II study being conducted in the U.K., and enrollment of pediatric patients is expected to begin later this year in the ITCH Phase II study in the U.S.

Lumena Pharmaceuticals - 09/26/2013

Ultragenyx selected as 2013 Fierce 15 Company

FierceBiotech selected Ultragenyx Pharmaceutical as one of the 2013 Fierce 15 Companies.

What makes Ultragenyx fierce: The most recent addition to the pipeline is KRN23, an antibody designed to treat rare cases of X-linked hypophosphatemia that is now wrapping a Phase I/II study at Kyowa Hakko Kirin. At the time KRN23 showed up on Ultragenyx's radar, it was the only rare-disease program at the Japanese pharma company, says CEO Emil Kakkis. When the deal was done just days ago, it was Ultragenyx's fourth midstage therapy.

Ultragenyx Pharmaceutical - 09/24/2013

Ultragenyx Announces Positive Data from Retrospective Study of UX007 Triheptanoin in Patients with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Treatment with triheptanoin appears to reduce the frequency and length of hospitalizations associated with LC-FAOD disease.

NOVATO, CA –September 16, 2013—Ultragenyx Pharmaceutical Inc., a biotechnology company, announced the release of positive data from a retrospective protocol-driven medical record review oftriheptanoin treatment of patients with long-chain fatty acid oxidation disorders(LC-FAOD). LC-FAODpatients have severe morbidity and mortality with frequent complications and hospitalizations. The study evaluated the impact of triheptanoin treatment on the rate and extent of hospitalizationsin 20 of 24 patients who have been treated with triheptanoin for up to 13 years as part of a compassionate use protocol and consented to be part of the retrospective study. The study compared the incident rate for the major medical events before and after triheptanoin treatment, including the total number of hospitalizations and hospital days per year due to all causes,muscle rupture (rhabdomyolysis), hypoglycemia, or cardiomyopathy.

Ultragenyx Pharmaceutical - 09/16/2013

Chimerix Presents Brincidofovir (CMX001) Adenovirus Phase 2 Results

Positive Phase 2 Study Results Presented as Late Breaker at ICAAC

DURHAM, NC, September 11, 2013 – Chimerix, Inc. (NASDAQ: CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced the results from its exploratory Phase 2 Study 202 evaluating brincidofovir (CMX001) in hematopoietic cell transplant (HCT) recipients with early adenovirus (AdV) infection. Study 202 was the first trial of an antiviral agent in AdV infection. Brincidofovir (CMX001) is an investigational oral nucleotide analog lipid-conjugate that has demonstrated activity against all pathogenic families of double-stranded DNA (dsDNA) viruses, including herpesviruses, adenoviruses, and polyomaviruses.

Chimerix - 09/11/2013

Chimerix Initiates Phase 3 SUPPRESS Trial of Brincidofovir (CMX001) for Prevention of CMV in HCT Recipients

DURHAM, N.C., Sept. 9, 2013 (GLOBE NEWSWIRE) -- Chimerix, Inc. (Nasdaq:CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced initiation of dosing in the Phase 3 SUPPRESS trial (ClinicalTrials.gov ID: NCT01769170). SUPPRESS is evaluating brincidofovir (CMX001) for the prevention of cytomegalovirus (CMV) infection, the most significant infectious disease in hematopoietic cell transplant (HCT) recipients. Brincidofovir is an investigational oral nucleotide analog lipid-conjugate that has demonstrated activity against all pathogenic double-stranded DNA (dsDNA) viruses, including herpesviruses, adenoviruses, and polyomaviruses.

Chimerix - 09/09/2013

LipoScience Announces New Data Supporting High-Density Lipoprotein Particle (HDL-P) Number As Marker Of Cardiovascular Risk

JUPITER study data published in Circulation, suggest HDL-P may be a better marker of residual risk than HDL cholesterol or apolipoprotein A-1 in patients on statin therapy

RALEIGH, N.C., Sept. 4, 2013 /PRNewswire/ -- LipoScience, Inc., (NASDAQ: LPDX) a diagnostic company pioneering a new field of personalized nuclear magnetic resonance (NMR) diagnostics to advance the quality of patient care in cardiovascular, metabolic and other diseases, today announced the publication of data supporting the measurement of high-density lipoprotein particle (HDL-P) number as a marker of cardiovascular risk in patients on cholesterol-lowering statin therapy. The JUPITER data analysis appeared September 3, 2013 in the online version of the journal Circulation in advance of the September 10, 2013 print issue.

LipoScience - 09/04/2013

Ultragenyx Announces Collaboration with Kyowa Hakko Kirin to Develop and Commercialize Phase 2-stage KRN23 for X-linked Hypophosphatemia

Exclusive partnership will advance global clinical development of
KRN23, an antibody for a rare bone disease

Novato, CA – September 3, 2013--Ultragenyx Pharmaceutical Inc.today announced it has entered into a collaboration and license agreement with Kyowa Hakko Kirin Co., Ltd. (KHK),to develop and commercialize KRN23. KRN23 is a recombinant fully human monoclonal IgG1 antibody intended to treat X-linked hypophosphatemia (XLH). KHK is currently completing a Phase 1/2 study in adults with XLH in the US and Canada. The two companies plan to initiate a pediatric XLH program in 2014.

Ultragenyx Pharmaceutical - 09/03/2013

Syndax Pharmaceuticals Secures $26.6 Million Series B Financing for Advancement of Epigenetic Therapies for Treatment-Resistant Cancers

Company Prepares for Pivotal Phase 3 Study of Entinostat, Most Advanced HDAC Inhibitor in Development for ER+ Metastatic Breast Cancer

WALTHAM, Mass., Aug. 27, 2013 — Syndax Pharmaceuticals Inc., which is developing epigenetic therapies for treatment-resistant cancers, today announced it has secured a $26.6 million Series B financing. Participating investors included Domain Associates, MPM Capital, Forward Ventures and RusnanoMedInvest (RMI).

Syndax - 08/27/2013

Chimerix Announces Top Line Data From CMX001 Phase 2 Adenovirus Study and Late-Breaker Presentation at ICAAC

-Encouraging results from the first proof-of-concept study for adenovirus infection support continued clinical development of CMX001 as a first-in-class broad-spectrum antiviral for prevention of double-stranded DNA viral infections

-Data accepted for oral late-breaker presentation at the ICAAC Annual Meeting on September 10th

DURHAM, N.C., Aug. 14, 2013 (GLOBE NEWSWIRE) -- Chimerix, Inc. (Nasdaq:CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced top line data from the exploratory Phase 2 study of CMX001 as preemptive therapy for adenovirus (AdV) infection, the first interventional trial in AdV infection. In this trial in allogeneic hematopoietic cell transplant (HCT) recipients, the 100 mg dose given twice weekly (BIW) demonstrated decreased levels of AdV viremia and showed a potential benefit in reducing both progression to AdV disease and all-cause mortality, compared to subjects who received placebo or CMX001 given once weekly (QW). Planned intent-to-treat analyses as well as exploratory analyses in specific patient groups were consistent in trends favoring the CMX001 BIW regimen over placebo, although statistical significance was not established in this exploratory study.

Chimerix - 08/14/2013

Ultragenyx Receives Approval of Clinical Trial Application (CTA) for a Phase 1/2 Trial TestingUX003 in Mucopolysaccharidosis Type 7 (MPS 7)

Ultragenyx Receives Approval of Clinical Trial Application (CTA)
for a Phase 1/2 Trial TestingUX003 in Mucopolysaccharidosis Type 7 (MPS 7)

NOVATO, CA – August 14, 2013 - Ultragenyx Pharmaceutical Inc., a biotechnology company, received Clinical Trial Application (CTA) approval from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. to conduct a Phase 1/2 clinical trial of UX003, recombinant human β-glucuronidase, in MPS 7, a rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the enzyme β-glucuronidase. MPS 7 is a severe multi-system disease resulting in cellular and organ dysfunction. There is no approved drug therapy.

Ultragenyx Pharmaceutical - 08/14/2013