Biomarin has presented the Second Quarter 2013 Financial Results Conference Call today. These conferences are important because allow investors and stakeholders to have updated information about the company and use to be a requirement by authorities for those companies with shares in the stock market. The presenters gave some information about the development of BMN-111.
As the blog visitors may already know, the phase 2 study was announced to be starting during this quarter after positive results Biomarin obtained in the phase 1 study.
Today, however, during the conference call, Biomarin said that the phase 2 study was put on partial clinical hold by the Food and Drug Administration (FDA). The presenters informed that the agency, although having approved the progress of the development of BMN-111 after the phase 1 study, is looking for more data related to pharmacokinetics (study of what the drug does to the body) and the adequate dose for children with achondroplasia. Biomarin also informed that there will be a new meeting with FDA to understand the needs and that the company remains confident in initiating the phase 2 study during the last quarter of 2013 or the first quarter of 2014.
FDA and other major Regulatory Agencies have been increasing requirements for studies involving children. For instance, in the past, pediatric doses for a new drug approved for adults were just extrapolated from those of the adults. Now, if an experimental drug could be used in children, tests would must be performed first in a pediatric population before it could be approved for them.
Regarding BMN-111, this would be only a personal guess about FDA reasons to put it on partial clinical hold since I don't have any extra information besides those given today during the conference call. There is the possibility that FDA could be requesting that a first group of children entering the phase 2 should have additional tests included in the protocol design to better define the pharmacokinetics in this population, presuming that those tests were not already included there. We will have to wait and see.
If you are interested in listening to the BMN-111 relevant parts of the presentation, just click on the link provided above and listen to the following segments beginning at: 19:30; 22:30; 25:20; 30:00; 37:35 and 54:55.
You can also go to the press release published by Biomarin, where it describes the current status of BMN-111 clinical development.
To read more about CNP and BMN-111, you can go to these articles published in the blog:
- CNP, the first potential pharmacological therapy for achondroplasia
- Biomarin announces phase I clinical trial of BMN-111
- Updated information about Biomarin's BMN-111 clinical development
- C-type natriuretic peptide (CNP) is important for midface growth
- Treating achondroplasia: the effects of BMN-111 in mice
- Treating achondroplasia: concepts and misconcepts about the potential therapies in development
- The mechanism of action of CNP in the FGFR3 pathway showed by a new study