Shire Developing Oral Chelator
March 28, 2013 – Shire, a specialty biopharmaceutical company, is currently in clinical trials with a new iron chelator. Below, Shire responds to questions from CAF concerning the status of the drug, currently known as SPD602.
How did Shire become interested in SPD602?
In April 2012, Shire acquired FerroKin BioSiences, Inc. and the iron chelator treatment SPD602 (formerly FBS0701) aimed to treat chronic iron overload following numerous blood transfusions in both children and adults with hereditary and acquired anemias.
Shire’s interest in SPD602 was considered a strategic step in developing Shire’s hematology business, and adds a differentiated product in development with global rights in a global market. There remains a significant unmet need for a once-a-day, oral iron chelator in a convenient dosage form for the treatment of transfusional iron overload with an improved safety profile than currently available treatments. We believe SPD602 has the potential to meet that need. We hope to use our expertise in hematology coupled with our proven ability to progress products through the development pipeline to bring SPD602 to patients. This acquisition marks an important step for Shire in building a business that serves the growing needs of patients with hematological disorders. In addition, SPD602 fits into Shire’s portfolio of development and commercialization of differentiated specialist products prescribed by specialist physicians (i.e.; hematologists, hematologist-oncologists).
What stage is the drug currently in? Do you have a rough timeline for the drug’s development (assuming best case scenario)?
SPD602 is currently under phase 2 clinical development.
- One phase 2 study has completed
- There are two additional phase 2 studies currently recruiting globally to assess safety, efficacy & tolerability of SPD602 in the following patient populations:
- SPD602-202 is a clinical trial in pediatric and adolescent patients with transfusional iron overload
- SPD602-203 is a clinical trial in adult patients with transfusional iron overload
- Subjects from both of the above studies may also be eligible to participate in a long-term safety extension study (SPD602-301).
- Please refer to http://www.clinicaltrials.gov/ for further information regarding the above-referenced trials.
Shire anticipates initiating phase 3 trials globally in 2014.
What findings have come from the studies thus far?
In study, SPD602-201/CTP-04, SPD602 was well tolerated at both doses of 16mg/kg/d and 32, mg/kg/d salt (14.5 and 29 mg/kg/d, respectively) when initially examined after 24 weeks of treatment. This response was seen in patients with common co-morbidities such as hepatitis and renal impairment. The overall frequency of AEs related to drug was low (<5%), with a short duration and generally mild nature. Data from this phase 2 study reflected that SPD602 was effective at reducing hepatic iron concentration in a majority of patients receiving 32 mg/kg/d salt (29 mg/kg/d).
At 48 weeks of treatment, SPD602 was well tolerated up to 36 mg/kg/d salt (40mg/kg/d); a maximum tolerated dose was not identified. No safety signals were noted at the higher doses. Treatment-related GI AEs were infrequent and Creatinine was stable. SPD602 was effective at reducing hepatic iron concentration in a majority (68%) of patients dosed at 36 mg/kg/d salt (40mg/kg/day).
Based on the results of studies so far, how would it be administered and how often?
Dosing will be determined following results of phase 2/3 studies. Currently Shire is exploring once and twice daily oral dosing regimens from 16-75mg/kg/d.
Has Shire had any previous involvement with treatments for thalassemia?
This is Shire’s first endeavor with a treatment for thalassemia, although Shire has vast experience in specialty drug development, including another hematology product, Xagrid for essential thrombocythemia.
What else do you think is important for the thalassemia community to know about it?
Shire aspires to be as brave as the people we help. We strive to identify new and holistic ideas and solutions that deliver a demonstrated value for society. We challenge ourselves to keep improving while putting patients at the center of everything we do. Shire’s “Brave” culture distinguishes us from others, and keeps us focused on our purpose: to enable people with life-altering conditions to lead better lives.
From CAF: Alexis Thompson, MD, MPH, a member of the CAF Medical Advisory Board and Director of Hematology Services at Lurie Children’s Hospital of Chicago, states that “Many patients with thalassemia have benefitted from advances in both non-invasive methods for iron monitoring as well as availability of newer oral iron chelating agents; however, side effects are unacceptable to some patients, and others seem to have inadequate responses even though they are taking their current chelation medications as prescribed. The early studies performed with SPD602 in patients with thalassemia have been very encouraging in terms of demonstrating efficacy of a new drug for iron control with side effects that are generally tolerable. Ongoing research to provide additional choices for effective iron chelation could expand the number of patients with good iron control and could potentially extend lives.”