Celldex Reports Phase 1 Study Results of CDX-301; Amicus Advances Chaperone-ERT Combo Platform in Pompe Disease Print E-mail
By Staff and Wire Reports   
Thursday, 14 February 2013 20:04
Below is a look at some of the headlines for companies that made news in the healthcare sector on February 14, 2013.

Celldex Therapeutics, Inc. (Nasdaq: CLDX)
presented final results from a Phase 1 study of CDX-301 (recombinant human Flt3L; rhuFlt3L) in healthy volunteers which strongly support the initiation of a pilot study in hematopoietic stem cell transplantation later this year. The data were presented by Michael Yellin, MD, Vice President of Clinical Science at Celldex, in an oral presentation at the American Society for Blood and Marrow Transplantation 2013 BMT Tandem Meetings. The study was conducted at Rockefeller University by Niroshana Anandasabapathy, MD, PhD, who worked as an instructor in clinical investigation in the laboratory of the late Nobel Laureate Ralph Steinman, MD.

"The final results from this study are consistent with previous clinical experience and demonstrate that CDX-301 is well-tolerated and can effectively mobilize hematopoietic stem cell populations as a single agent in healthy volunteers," said Dr. Yellin. "Based on the safety profile and the impressive increases observed for CD34+ stem cells and dendritic cells, we believe CDX-301 holds significant promise in both the allogeneic and autologous transplant setting, including the potential for combination with other molecules that could work in synergy with CDX-301 to further enhance the mobilization of hematopoietic stem cell populations. Published pre-clinical Flt3L studies also suggest that CDX-301 could potentially improve engraftment and reduce the risk of graph versus host disease. This may be especially important for older patients and patients who are in poor health."

Study results:  30 healthy volunteers were enrolled across seven cohorts. The first five cohorts assessed escalating doses of CDX-301 (from 1 mcg/kg to 75 mcg/kg) as a five-day regimen, while the final two cohorts assessed CDX-301 (at 25 mcg/kg) as seven- and 10-day regimens. All volunteers completed dosing and safety follow-up. Short-term dosing of five days resulted in a median of 10-fold increases in CD34+ cells—stem cells that have the ability to give rise to all cell types in the blood. Of note, on day 10 of dosing, a greater than 100-fold increase in dendritic cells—cells believed to play a critical role in the long-term improvement of transplant outcome—was observed in the peripheral blood. CDX-301 was generally well-tolerated with transient grade 1 lymphadenopathy observed in six volunteers (all treated at the highest dose levels of 25 mcg/kg and 75 mcg/kg). No anti-CDX-301 antibodies were detected in any volunteers through the end of study follow-up.

CDX-301 or rhuFlt3L is a potent cytokine that stimulates the expansion and differentiation of hematopoietic progenitor and stem cells. rhuFlt3L has demonstrated a unique capacity to increase the number of circulating dendritic cells in both laboratory and clinical studies. In addition, rhuFlt3L has shown impressive results in models of cancer, infectious diseases and inflammatory/autoimmune diseases. The clinical safety and activity of rhuFlt3L were originally demonstrated by Immunex (now Amgen) in clinical trials involving more than 150 healthy volunteers and 380 patients with cancer. Recognizing the promise of CDX-301, Celldex in-licensed the program from Amgen in 2009. Celldex's completion of this recent Phase 1 study confirms and builds upon the extensive prior clinical experience and reaffirms the significant potential for CDX-301.



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Amicus Therapeutics (Nasdaq: FOLD)
announced positive results from clinical and preclinical studies of the pharmacological chaperone AT2220 (duvoglustat HCl) in combination with ERT for Pompe disease at the Lysosomal Disease Network WORLD Symposium (LDN WORLD).

John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, "The advancement of our core platform technology in Pompe and other lysosomal storage disorders is a continued great step forward for Amicus. Across these serious genetic diseases, we are leveraging this chaperone-ERT combination platform to work towards improving currently marketed ERTs and to develop our own proprietary next-generation ERTs that incorporate our small molecule chaperones. These chaperone stabilizers have the potential to enhance ERT activity and tissue uptake while also significantly reducing the immunogenicity of the ERTs. Through these programs we hope to offer new benefits and treatment options for patients with lysosomal storage diseases."

Chaperone-ERT Combinations for Pompe Disease--AT2220 Co-Administered with Marketed ERTs--Positive results from a Phase 2 study (Study 010) established human proof-of-concept that co-administration of AT2220 just prior to infusing ERT (Myozyme/Lumizyme, or rhGAA enzymes) increases GAA enzyme activity in muscle tissue compared to ERT alone. These results appear in a poster 1 and will be featured in an oral platform presentation at LDN WORLD on Friday, February 15 at 9:15 am ET.

Based on these results, Amicus plans to initiate a repeat-dose clinical study in the third quarter of 2013 to evaluate a novel intravenous formulation of AT2220 (AT2220-IV) co-administered with Myozyme/Lumizyme. AT2220-IV when co-administered with ERT is designed to have an improved pharmacokinetic (PK) profile compared to oral AT2220 for all Pompe patients, many of whom are unable to swallow an oral small molecule. The upcoming clinical study will investigate multiple doses of AT2220-IV co-administered with Myozyme/Lumizyme every two weeks in Pompe patients. Objectives of the study are to characterize safety and PK for later evaluation of infants and special populations. Key parameters are expected to include GAA enzyme activity and AT2220 levels in plasma and muscle, as well as rhGAA antibody titers.

Next-Generation ERT (AT2220 Co-Formulated with a Proprietary Amicus ERT)--Preclinical studies of AT2220 co-formulated with rhGAA enzyme (Myozyme/Lumizyme) were presented for the first time in a poster 2 at LDN WORLD. This chaperone-ERT co-formulation resulted in up to 2.5-fold greater enzyme uptake in multiple disease-relevant tissues and led to greater glycogen reduction compared to rhGAA alone in GAA knock-out mice. Collectively these data suggest that AT2220 directly binds to and stabilizes rhGAA, potentially leading to a larger amount of properly folded, active enzyme available for uptake into tissue. AT2220 co-formulated with ERT may also mitigate Pompe ERT-related immunogenicity since properly-folded proteins are less prone to aggregation and less immunogenic.

Following the completion of these preclinical studies, Amicus entered into a contract with Laureate Pharmaceuticals for the manufacture of a proprietary rhGAA enzyme. Amicus is developing AT2220 co-formulated with this proprietary enzyme as a next-generation ERT for Pompe disease. Through this approach Amicus believes it has the potential to improve the properties of the rhGAA enzyme itself while incorporating AT2220 as a small molecule stabilizer to increase exposure and tissue uptake, and reduce immunogenicity relative to currently marketed ERTs. Successful development of a more stable ERT may also enable novel routes of delivery such as subcutaneous administration.



Also Thursday:



Anika Therapeutics, Inc. (Nasdaq: ANIK)
today announced that it plans to issue its fourth-quarter and year-end 2012 financial results after the close of the market on Wednesday, February 27, 2013.

William Dolphin, Ph.D., the CEO of regenerative medicine company Avita Medical Ltd. (ASX: AVH) (OTCQX: AVMXY), was interviewed by SNNLive last week.

Bioanalytical Systems, Inc. (NASDAQ:BASI)
today announced financial results for the first quarter ended December 31, 2012.

BIOLASE, Inc. (NASDAQ: BIOL)
, the world's leading dental laser manufacturer and distributor, announced today that Fred Furry, Chief Operating Officer and Chief Financial Officer, will participate in the Lazard Capital Markets 5th Annual Medical Technology Snowbird Conference.

Bristol-Myers Squibb Company (NYSE: BMY)
today announced that Ann Powell Judge will join the company as senior vice president, Human Resources, in March.

Cellular Biomedicine Group (OTCQB:EBIGD)
announced today that it has recently achieved both ISO 9001:2008 certification and ISO-14644 cleanroom certification.

Cepheid (Nasdaq: CPHD)
today announced that its Board of Directors has elected John Bishop as Chairman of the Board of Directors and named Cristina Kepner as Lead Independent Director, effective February 12, 2013.

Cerus Corporation (NASDAQ:CERS)
announced today that its fourth quarter and year-end 2012 financial results will be released on Thursday, February 28, 2013, after the close of the stock market.

CONMED Corporation (NASDAQ: CNMD)
today announced financial results for the fourth quarter and year ended December 31, 2012.

Corgenix Medical Corporation (OTC BB: CONX)
, a worldwide developer and marketer of diagnostic test kits, today filed its second quarter Form 10-Q and reported its financial results.

eHealth, Inc. (NASDAQ: EHTH)
, America's first and largest private health insurance exchange, announced today its financial results for the fourth quarter and fiscal year ended December 31, 2012.

FONAR Corporation (NASDAQ: FONR)
, The Inventor of MR Scanning™, reported today its second quarter financial results of fiscal 2013.

Guided Therapeutics, Inc. (OTCBB: GTHP) (OTCQB: GTHP)
today announced that it passed its annual independent quality audit and, with all external and mechanical testing completed, plans to apply the Edition 3 CE Mark to the LuViva® Advanced Cervical Scan for expanded commercial introduction of the product in select European countries.

IEC Electronics Corp. (NYSE MKT: IEC)
has announced that Jeff Schlarbaum, President, and the Company have mutually agreed that he will transition out of IEC Electronics.

IsoRay, Inc. (NYSE MKT: ISR)
, a medical technology company and innovator in seed brachytherapy and medical radioisotope applications for brain cancer, lung cancer, head and neck cancer, prostate cancer and gynecologic ("gyn") cancer announced its financial results for the quarter and six months ended December 31, 2012.

La Jolla Pharmaceutical Company (OTCQB: LJPC)
("La Jolla" and "Company"), a leader in the development of therapeutics targeting galectin proteins, announced today that the United States Patent and Trademark Office (USPTO) has issued an additional notice of allowance for claims covering its lead product candidate, GCS-100, from patent application number 13/588,877.

LifeApps Digital Media Inc. (OTCQB: LFAP)
today announced that they have executed a preliminary Letter of Intent relating to a proposed strategic alliance with MediSwipe Inc. (www.MediSwipe.com) (OTCQB: MWIP), a patient security solutions and financial products company for the medicinal marijuana and health care industry, and 800 Commerce Inc., a mobile marketing and payment solutions company, to produce mobile applications for Apple and Android platforms targeting the mobile healthcare sector.

Medivation, Inc. (NASDAQ: MDVN)
today announced that it will host a live teleconference with management to discuss fourth quarter and year-end 2012 financial results and provide a general business update on February 28, 2013 at 4:30 p.m. Eastern Time.

Oramed Pharmaceuticals Inc. (NASDAQ: ORMP)
(http://www.oramed.com), a developer of oral drug delivery systems, announced today that it has been in communication with the U.S. Food and Drug Administration (FDA) regarding its Investigational New Drug (IND) application for oral insulin.

Palatin Technologies, Inc. (NYSE MKT: PTN)
, a biopharmaceutical company developing targeted, receptor-specific peptide therapeutics for the treatment of diseases with significant unmet medical need and commercial potential, today announced results for its second quarter ended December 31, 2012.

Pharmacyclics, Inc. (Nasdaq: PCYC)
today reported financial results and recent developments for its six and three months ended December 31, 2012.

Optimer Pharmaceuticals, Inc. (Nasdaq: OPTR)
today announced that the February 2013Journal of the American Geriatrics Society published results from a multivariate analysis demonstrating that advancing age is a predictor of deterioration in treatment outcomes in patients with Clostridium difficile infection (CDI).

Teva Pharmaceutical Industries Ltd. (NYSE: TEVA)
announced today that the U.S. Food and Drug Administration (FDA) has approved its Abbreviated New Drug Application (ANDA) for the generic version of Shire’s Adderall XR® Capsules, 5mg, 10mg, 15mg, 20mg, 25mg and 30 mg capsules for the treatment of attention deficit hyperactivity disorder.

United Therapeutics Corporation (NASDAQ: UTHR)
announced today that the U.S. Food and Drug Administration (FDA) has acknowledged the resubmission of the new drug application (NDA) for treprostinil diolamine extended release tablets (oral treprostinil) for the treatment of pulmonary arterial hypertension.



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