Alexion Pharmaceuticals announced positive topline results of ALXN1210

Alexion says Phase 3 ALXN1210 study shows patients can be switched from Solirs 

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Alexion Pharmaceuticals  announced “positive” topline results of a Phase 3 study of ALXN1210

Alexion Pharmaceuticals (ALXN)  announced “positive” topline results of a Phase 3 study of ALXN1210, the company’s investigational long-acting C5 complement inhibitor, which show that patients with paroxysmal nocturnal hemoglobinuria “can be effectively and safely switched from treatment with Soliris every two weeks to treatment with ALXN1210 every eight weeks.”

The study demonstrated non-inferiority of ALXN1210 to #Soliris in patients with PNH who had been stable on Soliris based on the primary endpoint of change in lactate dehydrogenase levels, a direct marker of complement-mediated hemolysis in PNH, the company said.

It adds that the study also demonstrated non-inferiority on all four key secondary endpoints: the proportion of patients with breakthrough hemolysis, the change from baseline in quality of life as assessed via the Functional Assessment of Chronic Illness Therapy-Fatigue Scale, the proportion of patients avoiding transfusion, and the proportion of patients with stabilized hemoglobin levels.

In addition, numeric results for all five endpoints favored ALXN1210. Notably, no patients treated with ALXN1210 experienced breakthrough hemolysis compared to five patients treated with Soliris, Alexion said.

It noted that ALXN1210 was generally well tolerated with a safety profile that is consistent with that seen for Soliris. “Once again ALXN1210 met the high bar set by Soliris in a second, large Phase 3 study.

Importantly, we now have robust data that patients with PNH can effectively and safely transition from Soliris to ALXN1210,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion.

“We are very pleased that the totality of the Phase 3 PNH data in more than 440 patients, which included patients who had never received a complement inhibitor and patients who were stable on Soliris and switched to ALXN1210, shows numeric results favoring ALXN1210 across all primary and key secondary endpoints, including breakthrough hemolysis.

We believe that the differentiated profile of ALXN1210 could be a meaningful improvement for patients and clinicians and look forward to moving rapidly to global regulatory filings in the U.S. and EU in mid-2018, followed by Japan later in the year.”


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Vertex initiates Phase 3 studies of its cystic fibrosis drugs

Vertex initiates Phase 3 studies of VX-445, Tezacaftor, and Ivacaftor

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Vertex initiates Phase 3 studies of VX-445, Tezacaftor, and Ivacaftor

Vertex Pharmaceuticals (VRTX) announced that it is initiating two Phase 3 studies of VX-445, tezacaftor and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis.

The first Phase 3 study will evaluate approximately 360 people with CF who have one copy of the F508del mutation and one minimal function mutation and is designed to support the submission of a New Drug Application in the U.S. using data from the study’s 4-week primary efficacy endpoint together with safety data through 12 weeks of treatment.

The second Phase 3 study will evaluate approximately 100 people with CF who have two copies of the F508del mutation, the most common genetic form of the disease, and is designed to support the submission of an application for approval in patients with two copies of the F508del mutation in the U.S. using data from the study’s 4-week primary efficacy endpoint together with 24-week safety data generated from the Phase 3 study in patients with one F508del mutation and one minimal function mutation.

The initiation of the study in people with two copies of the F508del mutation is supported by data announced today from a Phase 2 study that showed an incremental mean absolute improvement in percent predicted forced expiratory volume in one second of 11.0 percentage points from baseline through week four of treatment when VX-445 was added in people with CF who have two F508del mutations and were already receiving tezacaftor in combination with ivacaftor.

In the Phase 2 study, the VX-445 triple combination regimen was generally well tolerated, and the majority of adverse events were mild to moderate in severity.

VRTX closed at $158.81.


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