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Alnylam Pharmaceuticals Inc.

Partner : Sanofi Genzyme

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Akshay Vaishnaw, M.D., Ph.D., SVP, Clinical Research, gave guidance for data from the ATLAS program. He stated, "ATLAS will enroll approximately 250 patients in three separate trials at over 100 clinical centers around the world and we expect to be fully enrolled in these studies over the next year, with top line results in mid to late 2019."
Source: Q2 2017 earnings conference call, 8/09/17.

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Alnylam and Sanofi Genzyme Initiate ATLAS Phase 3 Program with Investigational RNAi Therapeutic Fitusiran in Patients with Hemophilia A and B with or without Inhibitors

07.07.2017
- ATLAS to Evaluate Safety and Efficacy of Fitusiran Across Broad Spectrum of Patients Living with Hemophilia -

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today the initiation of the ATLAS Phase 3 clinical program for fitusiran. The global, multicenter program is designed to evaluate the safety and efficacy of fitusiran in three separate trials, including patients with hemophilia A and B with or without inhibitors and patients receiving prophylactic therapy. Fitusiran is an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of patients with hemophilia A and B, that is designed to lower levels of AT with the goal of promoting sufficient thrombin generation to restore hemostasis and prevent bleeding.

This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20170707005070/en/

"ATLAS will include patients with hemophilia A and B with or without inhibitors, and patients previously receiving on-demand or prophylactic therapy with replacement factors or bypassing agents," said Akin Akinc, Ph.D., Alnylam's Vice President and General Manager, Fitusiran. "We expect top-line data from the ATLAS trials in mid-to-late 2019."

"We are pleased to have initiated the ATLAS Phase 3 program with our colleagues at Alnylam," said Baisong Mei, M.D., Ph.D., Sanofi's Senior Global Project Head, Alnylam Portfolio. "The ATLAS studies, which are designed to evaluate the safety and efficacy of fitusiran across a spectrum of patients living with hemophilia, are expected to enroll approximately 250 patients across three separate trials conducted at over 100 clinical centers around the world."

The three separate trials are:

ATLAS-INH, a nine-month, open-label, randomized, active-controlled trial designed to enroll approximately 50 patients with hemophilia A or B with inhibitors receiving prior on-demand therapy. The study's primary endpoint is the annualized bleeding rate (ABR), and key secondary endpoints include the annualized spontaneous bleeding rate, annualized joint bleeding rate, and quality of life as measured by the Haem-A-QOL score.

ATLAS-A/B, a nine-month, open-label, randomized, active-controlled trial designed to enroll approximately 100 patients with hemophilia A or B without inhibitors receiving prior on-demand therapy. The study's primary endpoint is the ABR, and key secondary endpoints include the annualized spontaneous bleeding rate, annualized joint bleeding rate, and quality of life as measured by the Haem-A-QOL score.

ATLAS-PPX, an open-label, one-way crossover study designed to enroll approximately 100 patients with hemophilia A or B with or without inhibitors receiving prophylaxis therapy as prior standard of care. In this study, patients will receive standard of care prophylaxis for six months and then transition to fitusiran treatment for seven months. The ABR will be prospectively measured in both periods. The study's primary endpoint is the ABR in the fitusiran period and in the factor/bypassing agent prophylaxis period. Key secondary endpoints include the annualized spontaneous bleeding rate, annualized joint bleeding rate, and quality of life as measured by the Haem-A-QOL score.
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Source: press release, 7/07/17. http://investors.alnylam.com/releasedetail.cfm?ReleaseID=1032423

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Compound/DeviceSpecialtyIndicationCompound ClassTarget
Fitusiran (ALN-AT3sc)HematologyHemophiliaRNAi therapeuticAntithrombin (AT) gene

Mechanism of action: Fitusiran (ALN-AT3) is a small interfering RNA that targets the antithrombin (AT) gene for the treatment of hemophilia and rare bleeding disorders (RBD)

Phase of Development: III

Event Type: Data: Phase III trial results

Dates: 2019-07-01 - 2019-12-31

Results: Pending