DARA's KRN5500 Receives Orphan Drug Designation From FDA; InterMune Reports Phase 3 ASCEND Trial Results of Pirfenidone Print E-mail
By Staff and Wire Reports   
Tuesday, 25 February 2014 20:01
Below is a look at some of the headlines for companies that made news in the healthcare sector on February 25, 2014.

DARA BioSciences, Inc. (NASDAQ: DARA)
, an oncology supportive care specialty pharmaceutical company dedicated to providing healthcare professionals a synergistic portfolio of medicines to help cancer patients adhere to their therapy and manage side effects arising from their cancer treatments, announced the U.S. FDA  has granted Orphan Drug Designation to KRN5500 for the parenteral treatment of painful, chronic, chemotherapy-induced peripheral neuropathy that is refractory to conventional analgesics. KRN5500 is a novel, non-opioid, non-narcotic intravenous product currently in Phase 2 clinical development. "We are absolutely thrilled to receive Orphan Drug Designation, and appreciate the hard work of the FDA's Office of Orphan Product Development over the many months in reviewing and ultimately approving the KRN5500 application for orphan designation.

We believe this Orphan Drug Designation will expedite the development of KRN5500 for patients with cancer who suffer from chronic neuropathic pain brought on by potent chemotherapeutic agents," said David J. Drutz, M.D., Chief Executive Officer and Chief Medical Officer of DARA BioSciences. "KRN5500, is a candidate to treat chronic neuropathic pain induced by chemotherapy, and fits perfectly into our corporate strategy of providing clinicians and patients with access to a synergistic portfolio of oncology supportive care products."

With the orphan drug designation, DARA is committed to evaluating various funding sources for the clinical advancement of KRN5500.


InterMune (NASDAQ: ITMN)
announced top-line data from ASCEND, a Phase 3 trial evaluating pirfenidone in patients with idiopathic pulmonary fibrosis (IPF), demonstrated that pirfenidone significantly reduced IPF disease progression as measured by change in percent predicted forced vital capacity (FVC) from Baseline to Week 52 (rank ANCOVA p<0.000001). Additionally, significant treatment effects were demonstrated on both of the key secondary endpoints of six-minute walk test distance (6MWD) change (p=0.0360) and progression-free survival (PFS) (p=0.0001).

"We are pleased to report these top-line ASCEND Phase 3 results," said Dan Welch, Chairman, Chief Executive Officer and President of InterMune. "Based on the strength of the ASCEND results, InterMune is preparing a resubmission of our New Drug Application for pirfenidone to the U.S. Food and Drug Administration (FDA), which we expect to submit by early third quarter of this year. We would like to thank our collaborators, patients and their families for their participation in ASCEND and their contributions to IPF research."

Primary Endpoint -- The magnitude of the treatment effect of pirfenidone was measured by comparing the proportion of patients in the pirfenidone and placebo groups experiencing either a clinically meaningful change in FVC, or death. A 10% decline in FVC in an individual IPF patient is considered clinically meaningful and strongly predicts mortality. At Week 52, 16.5% of patients in the pirfenidone group experienced an FVC decline of 10% or more or death, compared with 31.8% in the placebo group, representing a 47.9% reduction in the proportion of patients who experienced a meaningful change in FVC or death. Additionally, at Week 52 the data demonstrated that 22.7% of patients in the pirfenidone group experienced no decline in FVC, compared with 9.7% in the placebo group, representing a 132.5% increase in the proportion of patients whose FVC did not decrease between Baseline and Week 52.

Dr. Talmadge King, Chair, Department of Medicine, University of California San Francisco and Co-chair of the ASCEND protocol steering committee, said, "IPF is an unpredictable, debilitating and ultimately fatal disease, and safe and effective treatments are desperately needed to alter the course of this challenging and complex condition. The ASCEND data demonstrated that pirfenidone significantly reduced decline in lung function and significantly increased the proportion of patients who had no decline, which is an important advance in the field. The results for 6MWT distance, PFS and mortality provide important supportive evidence of pirfenidone's efficacy."

Key Secondary Endpoints -- The ASCEND protocol pre-specified 6MWD and PFS as the two key secondary endpoints. Change from Baseline to Week 52 in 6MWD is a measure of exercise tolerance. A 50-meter decrement in walk distance is considered an independent predictor of mortality in an individual patient with IPF. In ASCEND, pirfenidone reduced by 27.5% the proportion of patients who experienced a decline in 6MWD of 50 meters or greater (p=0.0360).

PFS is a measure of time before death or a disease-progression event. A PFS event was defined in the protocol as any of the following: death, percent predicted FVC decrement of 10% or greater or 6MWD decrement of 50 meters or greater. In ASCEND, pirfenidone reduced the risk of death or disease progression by 43% compared to placebo (Hazard Ratio [HR]=0.57; 95% confidence interval, 0.43-0.77; p=0.0001).

Additional Secondary Endpoints -- Three additional secondary endpoints were pre-specified in the ASCEND protocol: all-cause mortality, treatment-emergent IPF-related mortality and change from Baseline to Week 52 in dyspnea (shortness of breath). The two mortality analyses were pre-specified for both the ASCEND study and the pooled population of the ASCEND study and the previous Phase 3 CAPACITY studies through 52 weeks. Due to the relatively low overall mortality rate in patient populations in the time frames studied in a single IPF study such as ASCEND, pooled analyses of ASCEND and CAPACITY data provide more statistical power and a more precise estimate of the treatment effect of pirfenidone on mortality.

In the pre-specified mortality analysis of the ASCEND study alone, there were fewer events of all-cause mortality (HR=0.55, log rank p=0.1045) and of treatment-emergent IPF-related mortality (HR=0.44, log rank p=0.2258) in the pirfenidone group compared to the placebo group. ASCEND was not powered to show a difference on these endpoints. The relationship of death to IPF was determined in ASCEND by a blinded adjudication committee.

The pre-specified analyses of the pooled population (N=1,247) across ASCEND and the two Phase 3 CAPACITY studies (taking CAPACITY mortality data through Week 52) showed that the risk of all-cause mortality was reduced by 48% in the pirfenidone group compared to the placebo group (HR=0.52, log rank p=0.0107). Additionally, in the pooled population the risk of treatment-emergent IPF-related death in the pirfenidone group compared to placebo was reduced by 68% (HR=0.32, log rank p=0.0061).

The secondary endpoint of dyspnea, measured by the UCSD SOBQ questionnaire, was not achieved (p=0.1577).

Safety and Tolerability -- In ASCEND, pirfenidone showed a favorable safety profile and was generally well tolerated.

A total of 93.5% and 94.6% of patients completed the study, died or had a lung transplant by study day 365 in the pirfenidone and placebo groups, respectively. The percentage of patients discontinuing treatment due to an adverse event was 14.4% in the pirfenidone group and 10.8% in the placebo group. Serious adverse events (SAEs) were reported in 19.8% of patients in the pirfenidone group and 24.9% in the placebo group. Hospitalizations due to respiratory, thoracic and mediastinal SAEs were reported in 3.6% of patients in the pirfenidone group and 11.2% in the placebo group.

The most common AEs with higher incidence in the pirfenidone group were primarily gastrointestinal (e.g., nausea and dyspepsia) and skin-related (e.g., rash). The GI and rash AEs were generally mild to moderate in severity, manageable, reversible and only infrequently led to treatment discontinuations.

Elevations of aminotransferase levels at least 3 times the upper limit of normal occurred in 2.9% of pirfenidone patients (including one case associated with a bilirubin increase) vs. 0.7% of placebo patients. In general, these elevations occurred early, were manageable and reversible, and were similar to those observed in previous pirfenidone studies.

The safety and tolerability profile of pirfenidone was generally consistent with observations from the previous Phase 3 CAPACITY studies, open-label extension studies and post-marketing experience.

"These results from the ASCEND trial provide compelling evidence of a clinically meaningful treatment effect of pirfenidone with generally favorable safety and tolerability findings, which is very encouraging for patients suffering from this fatal and relentless disease," said Paul W. Noble, Chair, Department of Medicine, Cedars-Sinai Medical Center, Los Angeles, Calif. and Co-chair of the ASCEND protocol steering committee. "Importantly, the overall safety observations from ASCEND complement and corroborate the robust safety database that already exists from the InterMune-sponsored clinical studies of pirfenidone and extensive post-marketing experience outside the United States."

InterMune intends to present additional data from the ASCEND study at the 2014 American Thoracic Society International Conference in May.

Also Tuesday:

Athersys, Inc. (Nasdaq:ATHX)
will release its 2013 fourth quarter and annual financial results at approximately 4:00 PM EST on Thursday, March 13, 2014, and host a conference call shortly thereafter at 4:30 PM EST to review the results.

BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX)
, a pharmaceutical company focused on the development and commercialization of treatments for rare and infectious diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for intravenous (i.v.) peramivir that was submitted to the FDA in December 2013.  

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN)
, a global leader in the development and commercialization of therapies for rare genetic diseases, today announced that Dan Maher, Senior Vice President, Product Development will present at the Cowen and Company 34th Annual Healthcare Conference on March 4th at 11:20am ET.

BioReference Laboratories, Inc. (Nasdaq:BRLI)
announced today it plans to issue earnings results for the first quarter 2014 before the open of market trading on Tuesday, March 4, 2014.

CAS Medical Systems, Inc. (Nasdaq:CASM)
, a leader in medical devices for non-invasive patient monitoring, announced today that it will report financial results for the fourth quarter and full-year 2013 on Thursday, March 6, 2014, prior to the market's opening.

Celldex Therapeutics, Inc. (Nasdaq:CLDX)
will release fourth quarter and year end 2013 financial results on Monday, March 3, 2014 before the U.S. financial markets open.

ChemoCentryx, Inc. (Nasdaq:CCXI)
, today announced that Thomas J. Schall, Ph.D., President and Chief Executive Officer, will present at the Cowen and Company 34th Annual Health Care Conference on Tuesday, March 4, 2014, at 10:40 a.m. Eastern Time at The Boston Marriott Copley Place hotel in Boston, MA.

Chimerix, Inc. (Nasdaq:CMRX),
a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced that Kenneth I. Moch, President and CEO of Chimerix, will present at the Cowen 34th Annual Health Care Conference on Tuesday, March 4, 2014 at 10:40am EST at the Boston Marriott Copley Place in Boston.

Curis, Inc. (Nasdaq:CRIS)
, an oncology-focused company developing novel, targeted drug candidates for the treatment of human cancers, today announced that the Company will present at the Cowen and Company 34th Annual Health Care Conference at 4:50 p.m. ET on March 3, 2014, in Boston.

Harris & Harris Group, Inc. (Nasdaq:TINY)
, an investor in transformative companies enabled by disruptive science, notes the announcement by its portfolio company, Metabolon, Inc., that it has signed several exclusive multi-year, royalty-based research collaboration and licensing agreements with Patia Biopharma, a Mexican biotech company working to make personalized preventive medicine a reality in Mexico and Latin America.

Hyperion Therapeutics, Inc. (Nasdaq:HPTX)
today announced that results of the Phase 2 trial of glycerol phenylbutyrate (GPB) for the treatment of hepatic encephalopathy (HE) were published in the March 2014 issue of Hepatology.

Integra LifeSciences Holdings Corporation (Nasdaq:IART)
today reported its financial results for the fourth quarter ending December 31, 2013. Total revenues for the fourth quarter were $220.8 million, reflecting an increase of $6.3 million, or 3.0%, over the fourth quarter of 2012.

Integra LifeSciences Holdings Corporation (Nasdaq:IART)
today announced that Kenneth Burhop has joined Integra as Corporate Vice President, Chief Scientific Officer. Dr. Burhop will be responsible for setting Integra's strategic scientific vision and roadmap, and will lead the Company's portfolio prioritization and management, and the scientific evaluation of corporate development and new product opportunities.

Landec Corporation (Nasdaq:LNDC)
, a materials science company that develops and markets innovative and patented products for healthy living applications in food and biomedical markets, is expanding commercial applications for its patented BreatheWay® technology packaging solutions.

LeMaitre Vascular, Inc. (Nasdaq:LMAT)
, a provider of peripheral vascular devices and implants, today reported Q4 2013 and full-year 2013 financial results.

Marrone Bio Innovations, Inc. (Nasdaq:MBII)
, a leading global provider of bio-based pest management and plant health products, announced today it has hired James B. Boyd as Vice President and Chief Financial Officer (CFO).

M Line Holdings, Inc. (OTCQB:MLHC)
, a leading provider of products, assemblies and services to the precision high tech segment of both the aerospace and medical industries as well as a seller of high end pre-owned Japanese Computer Numerically Controlled ("CNC") Equipment, today announced that the Company completed the construction of a new addition to its Elite Machine Tool division's current space needed to meet its increased demand.

Medical Marijuana Inc. (OTC Pink:MJNA)
is pleased to inform shareholders and the general public that HempMedsPX™—a corporate portfolio company of Medical Marijuana Inc. and the exclusive master distributor and contracted marketing company for CannaVest Corp. and Medical Marijuana Inc.—is the exclusive award sponsor of the San Diego Regional Chamber of Commerce's 143rd Anniversary celebration on Tuesday, February 25th at the Hilton Bayfront Hotel in San Diego, California.

Merit Medical Systems, Inc. (Nasdaq:MMSI)
, a leading manufacturer and marketer of proprietary disposable devices used primarily in cardiology, radiology and endoscopy, today announced that it will participate at Raymond James 35th Annual Institutional Investors Conference being held March 2-5, 2014 at the JW Marriott Grande Lakes in Orlando.

Pain Therapeutics, Inc. (Nasdaq:PTIE)
today announced that Remi Barbier, Chairman, President & CEO, will present at Cowen and Company's 34th Annual Health Care Conference on Monday, March 3 at 3:30 p.m. Eastern Time at the Copley Place Hotel.

Premier, Inc. (Nasdaq:PINC)
has been named a 2014 IBM Beacon Award winner for information management innovation using big data and analytics.

Repligen Corporation (Nasdaq:RGEN)
today announced that it will report fourth quarter and fiscal year 2013 financial results on Thursday, March 6, 2014.

Stereotaxis, Inc. (Nasdaq:STXS)
today reported financial results for the fourth quarter and full year ended December 31, 2013.

Stereotaxis, Inc. (Nasdaq:STXS)
announced today that it has entered into a definitive agreement with Medix Japan, Inc. and Hokushin Medical Co. Ltd. to distribute its Niobe® Magnetic Navigation System for cardiac ablations in Japan.

Swisher Hygiene Inc. (Nasdaq:SWSH) (TSX:SWI)
, a leading provider of essential hygiene and sanitizing solutions, announced today that it expects to timely file its 2013 Form 10-K and release fourth quarter and full year 2013 results by March 17, 2014.

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