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Apellis' (APLS) Empaveli Succeeds in Treatment-Naive PNH Study

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Apellis Pharmaceuticals, Inc. (APLS - Free Report) and its partner Swedish Orphan Biovitrum (Sobi) announced that the phase III study — PRINCE — evaluating their targeted C3 therapy, Empaveli (pegcetacoplan) in treatment-naïve paroxysmal nocturnal hemoglobinuria (PNH) patients met its co-primary endpoints.

Data from the study showed that treatment with Empaveli achieved statistically superiority in hemoglobin stabilization and reduction in lactate dehydrogenase (LDH) compared to standard of care (SoC; excludes complement inhibitors) after 26 weeks of treatment. Please note that high levels of LDH indicate some form of tissue damage, including red blood cells.

Data from the PRINCE study showed that 86% of patients treated with Empaveli achieved hemoglobin stabilization, defined as less than 1g/dL decrease in hemoglobin levels in the absence of blood transfusions, compared to 0% for SoC. Moreover, mean LDH levels decreased 90% from baseline versus 14% for SoC.

Meanwhile, Empavel also met several secondary endpoints of the PRINCE study, including improvements in hemoglobin levels and transfusion avoidance, with statistical superiority.

A rare blood disorder, PNH is associated with abnormally low hemoglobin levels as the disease destroys red blood cells.

Shares of Apellis have plunged 15.6% so far this year compared with the industry’s decrease of 4.9%.

Please note that the PRINCE study evaluated twice-daily 1080mg dose of Empaveli compared to SoC in treatment-naïve patients, defined as patients who have not received treatment with any complement inhibitor within three months prior to screening.

We remind investors that the FDA approved Empaveli as a monotherapy treatment for adult patients suffering from PNH for treatment-naïve patients as well as for patients switching from Alexion’s C5 inhibitor therapies for PNH — Soliris (eculizumab) and Ultomiris (ravulizumab).

The FDA approval was based on positive data from the head-to-head phase III study — PEGASUS — that demonstrated improvement in hemoglobin levels in PNH patients following treatment with Empaveli compared to Soliris.

Notably, a marketing authorization application seeking approval for Empaveli as treatment for PNH patients is under review in Europe. A potential approval is anticipated in the second half of 2021.

Apart from PNH, Apellis is developing Empaveli for systemic administration in several indications. Empaveli is being evaluated as a monotherapy in two phase III studies, namely, DERBY and OAKS, for treating patients with geographic atrophy. Top-line data from the same is expected in the third quarter of 2021.

We note that Empaveli has just been approved by the FDA and is yet to generate incremental revenues for the company. The drug will likely face stiff competition from Soliris and Ultomiris, which are also approved for treating PNH. Also, Alnylam Pharmaceuticals (ALNY - Free Report) is conducting early clinical studies to develop cemdisiran, an RNAi therapeutic targeting C5. Nomacopan, a small protein C5 complement inhibitor, is being developed by Akari Therapeutics (AKTX - Free Report) in late-stage studies. Stiff competition in the target market will be a challenge for Apellis.

Zacks Rank

Apellis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.

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