Venture Partner Mike Grey named EY Entrepreneur Of The Year San Diego
Venture Partner Mike Grey named EY Entrepreneur Of The Year San Diego
Ultragenyx Announces Results from Phase 1/2 Study of KRN23 in X-linked Hypophosphatemia in Adults
Treatment with KRN23 induces a sustained increase in serum phosphorus and increases in bone remodeling markers
Novato, CA— June 24, 2014 — 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 results from a multiple-dose study, conducted by Kyowa Hakko Kirin Pharma, Inc (KKP), of the investigational anti-FGF23 monoclonal antibody KRN23 (UX023) in adult patients with X-linked hypophosphatemia (XLH). XLH is an inherited metabolic bone disease characterized by short stature, skeletal deformities, bone pain, fractures, and muscle weakness. The data was presented at the 2014 ICE/ENDO joint meeting of The Endocrine Society and The International Congress of Endocrinology in Chicago.
Mirati Therapeutics Receives Orphan Drug Designation from U.S. Food & Drug Administration for Mocetinostat in Myelodysplastic Syndrome
SAN DIEGO, June 17, 2014 /PRNewswire/ -- Mirati Therapeutics, Inc. (NASDAQ: MRTX) announced that mocetinostat, the company's spectrum selective HDAC inhibitor, has been granted Orphan Drug Designation by the U.S. Food & Drug Administrationas a treatment for myelodysplastic syndrome (MDS). Mocetinostat is being developed in Phase 2 clinical studies in combination with Vidaza as a treatment for intermediate and high-risk MDS, as well as a single agent treatment in patients with diffuse large B-cell lymphoma (DLBCL) and bladder cancer targeting specific genetic mutations in histone acetylation that increase the likelihood of response in tumor cells.
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.
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.
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.
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.
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.
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.
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.
