Novo medicamento em fase de pesquisa

Boa noite, boa tarde, bom dia! Anexo neste post uma mensagem em inglês para evitar problemas de tradução que recebi da Glaide Mara Martins Guilherme sobre a pesquisa de um novo medicamento para a ATTR. Obrigado Glaide por tudo. André. ------------------------------------------------------------------------------------------------------------------ Below please find the answers to your questions about our ALN-TTR program and therapeutic. Please know there are a few questions that we couldn’t answer fully due to the current development phase of the program but please let me know if you have any additional questions. In addition, you can find more in-depth information about our program including data, presentations, and podcasts at http://www.alnylam.com/capella/tag/ttr/ All my best, Maryellen Q&A 1 - What is the name of this new research (drug)? The the therapeutic that we are developing for Familial Amyloidosis Polyneuropathy is currently named ALN-TTR02. 2 - Tafamidis is intended to inhibit the production of amyloid and with that prevent disease from progressing. What is the action of this new drug? Our therapeutic approach or mechanism of action is RNAi therapeutics. RNAi is a natural mechanism for silencing specific genes. Genes provide cells with the instructions for making proteins, and proteins — or more specifically proteins made abnormally — are the cause of most human disease. When a gene is silenced, the cell stops making the protein specified by that gene, thereby reducing the occurrence of disease. Our RNAi therapeutic for TTR Amyloidosis has the potential to silence the TTR gene. Below is a link that explains RNAi http://www.pbs.org/wgbh/nova/body/rnai-explained.html Last November, we presented preliminary clinical data for ALN-TTR01. The ALN-TTR01 Phase I trial informed many things for our overall program, including safety and tolerability, proof of concept, extent of knock-down, pharmacodynamics, dosing, etc., and we are applying these learnings to our ALN-TTR02, our lead compound. Earlier this week, we initiated dosing in a Phase I clinical trial with ALN-TTR02. The trial is being conducted in the U.K. as a randomized, single-blind, single-ascending dose study, enrolling up to 32 healthy volunteer subjects. We expect to report data for this study in the 3rd quarter of 2012. We also expect to initiate a Phase II study with ALN-TTR02 in FAP patients in the 2nd half of this year with the start of pivotal trials with ALN-TTR02 in 2013. 3 - Since this new drug has a broader efficiency than Tafamidis, can it be employed simultaneously or the patient must use one or the other? This question is intended to clarify the use - because Tafamidis is already being consumed by some members of our Association, ABPAR (Brazilian Association of familial amyloidosis) - and, with this, to know if they could continue using Tafamidis and, at the same time, participate in this new research. We are excited by the news of approval of tafamidis in the EU, as it is great news for ATTR patients and also for our program, which we view as a complementary approach. 4 - When do you intend to insert do volunteers in the program. Is there a date already set? AND 5 - Pregnant or breastfeeding women will be allowed to participate in the trial? As stated above we expect to initiate a Phase II study with ALN-TTR02 in FAP patients in the 2nd half of this year. Details as to where the trials are being conducted as well as inclusion and exclusion criteria are still being developed. Maryellen Livingston Associate Director, Creative and Corporate Communications Alnylam Pharmaceuticals 300 Third Street - Cambridge, MA 02142