Fettleibigkeit ist in den USA nicht nur ein massives Gesundheitsthema, sondern auch gesellschaftspolitisch von großer Bedeutung. Nach zwei Todesfällen binnen 2 Monaten bei Teilnehmern einer laufenden Phase III Studie ist nun Panik in der Kiste. Ein zeitweiser Stopp der gesamten Studie durch die FDA vor wenigen Wochen dämpft die Hoffnungen auf positive Endergebnisse im ersten Quartal 2016. Alles aus für Zafgen? Totgesagte leben manchmal länger. Klar ist, dass die Patientengruppe in der Studie zu einer gesundheitlichen Hochrisikogruppe gehört. Entscheidend wird in den nächsten Tagen und Wochen der Befund, ob Beloranib in Zusammenhang mit den beiden Todesfällen stand, oder nicht.
Dear Mr. NN (name changed here for private policy)
Thank you for your email and questions. Providing the efficacy results from our trial prove to be very positive, we will be working to determine a path forward for the drug. We remain on track for data analysis and will communicate top-line results quickly once we have them. FDA has pledged to be responsive and to work with us constructively to understand the benefit/risk proposition for the drug.
With kind regards,
-------------------------------------------------- Thomas E. Hughes, Ph.D. Chief Executive Officer Zafgen, Inc. 175 Portland Street, 4th Floor Boston, MA 02114 Tel. +1 (617) 648-9813 email@example.com http://www.zafgen.com
From: GaborPis Sent: Friday, December 04, 2015 10:38 AM To: Tom Hughes Subject: polite request according to recent troubles Zafgen´s Belonarib study is facing
Dear CEO Dr. Hughes,
not only as a shareholder of Zafgen Inc. but much more than that - being aware of the immense potential of Beloranib for life improvement for thousands of people suffering from PWS and/or obesity, may I ask you: With the complete hold of the clinical study with Beloranib by FDA 48 hours ago, is there still hope that this hold can be lifted again and results form the Beloranib studies can be presented as scheduled by you in Q1-2016? And if so, is there any timeframe from your side defined with FDA when and how to look after and respond to their concerns and requests?
Thank you for paying attention to my polite request and all the best for your valuable efforts,
Positive phase 2 data for Zafgen's beloranib in obese type 2 diabetes patients
Following a 6-month treatment period, weight loss was 12.7% (p<0.0001) and 13.5 percent (p<0.0001) in the 1.8 mg and the 1.2 mg beloranib groups, respectively, and 3.1% in the placebo group. In addition, 2.0% decreases in HbA1c at 6 months in both beloranib groups vs 0.6% decrease in the placebo group were observed (Zafgen press release, 18 February 2016).
The one-year trial was terminated early because of an FDA clinical hold which was imposed following cases of pulmonary embolism among patients treated with beloranib in a phase 3 trial in Prader-Willi Syndrome patients. In this phase 2 trial there was one case pulmonary embolism in a patient treated with beloranib.
The strong efficacy data has the potential to make this drug best in class within obesity. However given the safety issues continued development in this population seems highly uncertain. Zafgen is reportedly in a dialogue with the FDA regarding the development of a risk mitigation strategy for beloranib that may allow for development in Prader-Willi Syndrome.