Computer rendering of the CD55 protein, which protects blood cells from destruction. | Courtesy of Wikimedia Commons

Shares of Crestwood-based Apellis Pharmaceuticals spiked nearly 18 percent Monday after the company said new data from clinical studies showed that its APL-2 drug provided “meaningful improvement” in the treatment of two blood-related autoimmune diseases and a genetic blood disorder.

Apellis CEO Dr. Cedric Francois told Insider Monday that the data show not only promise for patients suffering from those diseases, but confirm the company’s expectation that APL-2 can function as a “platform opportunity,” or a base that can be used to target a host of related conditions.

The company said in news releases Sunday and Monday that new data from studies indicate that its APL-2 drug showed promising results for patients with two diseases in which the body’s immune system mistakenly attacks and destroys its own red blood cells — cold agglutinin disease (CAD) and warm autoimmune hemolytic anemia (wAIHA) — and for paroxysmal nocturnal hemoglobinuria, a disorder that leads to the premature death and impaired production of blood cells.

Both CAD and wAIHA lead to anemia and other associated signs and symptoms, according to the National Institutes of Health. In severe cases, wAIHA can lead to heart failure. PNH leaves patients susceptible to blocked veins and arteries, which can cause heart attacks or strokes; anemia and other bone marrow problems.

Apellis made the announcements at the 60th American Society of Hematology Annual Meeting and Exposition in San Diego.

Dr. Cedric Francois

Francois told Insider that the results leave company leaders hopeful that APL-2 “will allow us to give (doctors) new weapons” for various life-altering conditions for which patients have few or no treatment options.

Patients with CAD and wAIAH have no FDA-approved therapies, Francois said. But those treated with APL-2 experienced a “meaningful clinical benefit” in that they saw increased hemoglobin and reductions in lactate dehydrogenase, an enzyme that indicates tissue damage, the company said.

“The breadth of activity in these two distinct diseases is impressive,” Dr. Morie A. Gertz, chair emeritus of the Department of Medicine of the Department of Hematology at the Mayo Clinic, said in a news release from Monday. Gertz is the primary investigator on the CAD and wAIAH study.

Patients with PNH have one approved therapy, eculizumab, but Francois said in a news release from Sunday that a recent study showed that among patients who took the drug, more than 70 percent remained anemic, and 40 percent had at least one transfusion in the prior year. The drug also costs about $440,000 per year, according to ReutersThe PNH patients on APL-2, on the other hand, showed “highly meaningful” improvements in their amounts of hemoglobin and transfusion avoidance, Apellis said.

“The safety is also promising, and the subcutaneous route of administration is friendlier to patients,” said Dr. Raymond Wong, of the Department of Medicine and Therapeutics at Prince of Wales Hospital at the Chinese University of Hong Kong. Wong is a principal investigator for the PNH clinical trial.

Apellis also is testing APL-2 for geographic atrophy, an age-related form of macular degeneration, a blinding disease that makes it difficult for patients to perform routine tasks, such as recognizing faces or reading. Shares of Apellis fell 17 percent in mid-October after the company said that some patients taking APL-2 had suffered inflammation.

Francois on Monday reiterated that the company is sure the inflammation was a result of a manufacturing issue — not the drug itself — and that Apellis in two weeks would announce the results of nonhuman studies that aim to confirm that assessment.

On Monday shares of Apellis closed at $18.06, up $2.72, or 17.7 percent for the day. The Nasdaq rose about 1.5 percent.





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