OMER in FierceBiotech re"TMA"s (ALXN's Solirus see below)
Omeros soars on fresh promise for its rare disease treatment:(ALEXION's Soliris) August 18, 2015 | By Damian Garde
Omeros ($OMER) is blueprinting a Phase III program for its lead drug after disclosing some positive data from an ongoing mid-stage trial, news that sent the biotech's share price soaring.
The antibody therapy, OMS721, is designed to clear blood clots from bodily organs, treating a group of very rare ailments called thrombotic microangiopathies, or TMAs. In new results from a small, three-pronged Phase II trial, patients getting the middle dosage of Omeros' injected treatment charted an increase in platelet counts, lower dangerous blood cell fragmentation and a boost in the blood protein haptoglobin, the company said.
Omeros is still enrolling the high-dose arm of the study, but the one patient who has completed four-week OMS721 therapy showed signs consistent with both the middle group and the low-dose cohort disclosed earlier this year, the company said.
On the safety said, Omeros said there have been no serious drug-related adverse events tied to the antibody. Two possible safety roadblocks identified in the first patient group amounted to nothing upon investigation, according to the company, and no further infection scares have since arisen.
Now Omeros believes it's on track to start mapping out a Phase III study for OMS721, expecting its 9-patient Phase II trial to spotlight an ideal dose for late-stage development. The new data sent Omeros' shares up about 65% on Tuesday.
OMS721 secured the FDA's fast-track designation last month, promising access to top agency officials and giving Omeros a shot at winning an accelerated approval for the treatment.
- read the statement
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Soliris and aHUS Soliris® (eculizumab) is the first and only therapy approved for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated microangiopathy.1 Soliris is approved for the treatment of patients with aHUS in nearly 40 countries, including the United States (U.S.), European Union (EU), and Japan.
About aHUS aHUS is a genetic, chronic, ultra-rare disease that can progressively damage vital organs, potentially leading to stroke, heart attack, kidney failure, and premature death.2,3 aHUS is caused by genetic abnormalities that result in chronic uncontrolled complement activation leading to complement-mediated thrombotic microangiopathy (TMA) — the formation of blood clots in small blood vessels throughout the body.2,4
aHUS affects both adults and children. Patients with aHUS face a lifelong risk of TMA, which may lead to sudden, catastrophic, and life-threatening damage to the kidney, brain, heart, and other vital organs, and premature death.2,3
Prior to the approval of Soliris for the treatment of aHUS, doctors relied on disease management strategies that did not specifically target uncontrolled complement activation, the underlying cause of TMA. Seventy-nine percent (79%) of all patients with aHUS died, required kidney dialysis, or had permanent kidney damage within three years after diagnosis despite plasma exchange or plasma infusion.5 Moreover, 33 to 40 percent of patients died or progressed to end-stage renal disease with the first clinical manifestation of aHUS despite supportive care.5,6
Treating Patients with aHUS with Soliris As a first-in-class treatment that specifically targets uncontrolled complement activation in patients with aHUS, Soliris represents a major step forward in the care of adults and children with this disease. In clinical studies, patients with aHUS experienced a reduction in terminal complement activity after the first dose, and this reduction was sustained with ongoing Soliris treatment.7,8,9
More Information Visit the Soliris website to learn more about aHUS and treatment with Soliris.
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