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Apellis' (APLS) Blood Disorder Drug Empaveli Gets FDA Nod

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Apellis Pharmaceuticals, Inc. APLS announced that the FDA has granted approval to its new drug application (“NDA”) for its targeted C3 therapy, Empaveli (pegcetacoplan). The NDA was seeking approval for Empaveli as a monotherapy treatment for adult patients suffering from paroxysmal nocturnal hemoglobinuria (PNH). A rare blood disorder, PNH is associated with abnormally low hemoglobin levels as the disease destroys red blood cells.

The drug is approved for treatment-naïve patients as well as for patients switching from Alexion’s ALXN C5 inhibitor therapies for PNH — Soliris (eculizumab) and Ultomiris (ravulizumab).

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.

Shares of Apellis have declined 22.7% year to date compared with the industry’s 5.6% decrease.

The FDA approval was based on positive data from the head-to-head phase III study — PEGASUS — that compared Empaveli to Soliris for improvement in hemoglobin levels in PNH patients. Data from the study showed that treatment with Empaveli achieved an adjusted mean increase of 3.84 g/dL of hemoglobin compared to Soliris from baseline, over 16 weeks of treatment. Moreover, 85% of patients receiving Empaveli were transfusion free over 16 weeks of treatment compared to 15% for Soliris. Empaveli also demonstrated non-inferiority for transfusion avoidance compared to Soliris.

Moreover, treatment with Empaveli over 48 weeks resulted in a sustained improvement in hemoglobin with a mean increase from baseline of 2.7 g/dL per data from an open-label extension of PEGASUS study. This compared well with Soliris as treatment with the drug led to mean decline in hemoglobin by 1.5 g/dL at week 16. Although there is no direct comparison of study data for Empaveli and Ultomiris made by Apellis, it seems competitive and may be in favor of Empaveli.

The superiority of Empaveli compared to Soliris indicates potential for the drug as several patients on Soliris may switch to Empaveli. Alexion recorded sales of more than $1 billion from Soliris in the first quarter of 2021. Empaveli’s label also includes a box warning similar to Soliris and Ultomiris that it may increase the risk of meningococcal and other serious infections.

However, we note that the approval may not boost the prospects of Apellis any time soon as Alexion is a big competitor. Moreover, following completion of the acquisition of Alexion by AstraZeneca AZN, which is expected to close in the third quarter, the competitor will become even bigger. This may result in significant increase in operating expenses for Apellis going forward to support commercialization of Empaveli. Moreover, both Soliris and Ultomiris are already approved for treating atypical hemolytic uremic syndrome.

Meanwhile, Apellis is developing Empaveli as a potential treatment for adult patients with sporadic amyotrophic lateral sclerosis, a neurological disease, in a phase II study.

Apellis Pharmaceuticals, Inc. Price

Apellis Pharmaceuticals, Inc. Price
Apellis Pharmaceuticals, Inc. Price

Apellis Pharmaceuticals, Inc. price | Apellis Pharmaceuticals, Inc. Quote

Zacks Rank and Stock to Consider

Apellis currently carries a Zacks Rank #3 (Hold). BioNtech SE BNTX is a better-ranked biotech stock, sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

BioNTech’s earnings per share estimates have increased from $15.73 to $29.44 for 2021 and from $5.02 to $21.34 for 2022 over the past 30 days.

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