|Celldex Reports Phase 1 Study Results of CDX-301; Amicus Advances Chaperone-ERT Combo Platform in Pompe Disease|
|By Staff and Wire Reports|
|Thursday, 14 February 2013 20:04|
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.
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.
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