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Apellis (APLS) Files NDA for Pegcetacoplan in Geographic Atrophy

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Apellis Pharmaceuticals, Inc. (APLS - Free Report) announced that it has submitted a new drug application (“NDA”) to the FDA seeking approval for its targeted C3 therapy, pegcetacoplan, for the treatment of geographic atrophy (“GA”) secondary to age-related macular degeneration.

The regulatory body’s decision on the NDA filing acceptance is expected in August 2022.

The latest NDA was based on data from two phase III studies — DERBY and OAKS — at 12 and 18 months, as well as data from the phase II FILLY study at 12 months. Data from these studies showed that treatment with both monthly and every-other-month pegcetacoplan led to a clinically meaningful reduction of GA lesion growth across a broad heterogenous patient population.

Also, pegcetacoplan demonstrated a favorable safety profile in all three studies.

A marketing authorization application for pegcetacoplan to treat GA is expected to be filed in the second half of 2022 to the European Medicines Agency.

Shares of Apellis have declined 12.5% year to date compared with the industry’s decrease of 23.6%.

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We remind investors that in May 2021, the FDA approved pegcetacoplan (marketed as 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.

Empaveli is approved for treatment-naïve patients and for those switching from Alexion’s [now part of AstraZeneca (AZN - Free Report) ] C5 inhibitor therapies for PNH, namely Soliris and Ultomiris.

AZN closed the acquisition of rare-disease drugmaker, Alexion, for $39 billion last July.

The Alexion buyout strengthened AstraZeneca’s immunology franchise.

Last December, the European Commission approved Aspaveli to treat adult patients with PNH who are anemic after treatment with a C5 inhibitor for at least three months. The drug is marketed under the trade name Empaveli in the United States.

In the first quarter of 2022, Empaveli generated sales worth $12.1 million, up 31.5% sequentially.

The phase II MERIDIAN study is evaluating systemic pegcetacoplan for treating amyotrophic lateral sclerosis or ALS. Apellis completed enrollment in the study in the first quarter of 2022. Top-line data from the same is expected in mid-2023.

A potential label expansion of pegcetacoplan for GA and additional indications will boost sales and drive growth for the company.

Zacks Rank & Stocks to Consider

Apellis currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the biotech sector are Leap Therapeutics, Inc. (LPTX - Free Report) and AegleaBio Therapeutics, Inc. , both carrying Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

The Zacks Consensus Estimate for Leap Therapeutics’ loss per share has narrowed 11.1% for 2022 and 5.9% for 2023 in the past 60 days.

Earnings of Leap Therapeutics have surpassed estimates in three of the trailing four quarters and missed the same on the other occasion. LPTX delivered an earnings surprise of 1.92%, on average.

AegleaBio Therapeutics’ loss per share estimates narrowed 19.4% for 2022 and 30.2% for 2023 in the past 60 days.

Earnings of AegleaBio Therapeutics have surpassed estimates in two of the trailing four quarters and missed the same on the other two occasions. AGLE delivered an earnings surprise of 9.47%, on average.


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