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Apellis (APLS) Begins Dosing With Pegcetacoplan in ALS Study

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Apellis Pharmaceuticals, Inc. (APLS - Free Report) along with partner Swedish Orphan Biovitrum AB (Sobi) announced that they have dosed the first patient in the phase II MERIDIAN study, which is evaluating pegcetacoplan, a targeted C3 inhibitor for treating adult patients with sporadic amyotrophic lateral sclerosis (ALS), a neurological disease.

This registrational, double-blind, placebo-controlled study is evaluating the safety and efficacy of pegcetacoplan in around 200 adult patients with sporadic ALS. The primary endpoint of the study is to check the Combined Assessment of Function and Survival rank scores at week 52 of the treatment. The key secondary endpoints include measures of lung function, muscle strength and quality of life.

Per the company, the MERIDIAN study will illustrate whether pegcetacoplan has the potential to slow down disease progression in the given patient population. Currently, there are no approved therapies to slow the advance of ALS.

Last month, Apellis entered into a collaboration with Sobi for the global co-development and ex-U.S. commercialization of systemic pegcetacoplan. Per the deal, Sobi will pay up to $1.25 billion in payments plus tiered royalties ranging from high teens to high twenties to Apellis.

Shares of Apellis have rallied 35.5% so far this year against the industry’s decrease of 2.6%.


We note that earlier this week, the FDA accepted Apellis’ new drug application (NDA), which is seeking an approval for pegcetacoplan to treat paroxysmal nocturnal hemoglobinuria (PNH), a rare, chronic, life-threatening blood disorder. The candidate is currently under priority review in the United States with the decision from the regulatory body expected on May 14, 2021.

The NDA was based on data from the III PEGASUS study, which compared pegcetacoplan to Alexion Pharmaceuticals’ (ALXN - Free Report) Soliris in patients with PNH.

Meanwhile, Apellis submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for pegcetacoplan in September 2020 as a treatment of PNH.

Pegcetacoplan is also currently being investigated in two phase III studies (DERBY and OAKS) for addressing patients with geographic atrophy in age-related macular (GA). Data from these studies is expected to be released in the third quarter of 2021.

Additionally, pegcetacoplan is being studied for the treatment of cold agglutinin disease (CAD). The candidate is also being evaluated in phase II studies to treat four types of glomerular diseases, namely C3 glomerulopathy (C3G), IgA nephropathy, primary membranous nephropathy and lupus nephritis.

Zacks Rank & Stocks to Consider

Apellis currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biotech sector include Abeona Therapeutics Inc. (ABEO - Free Report) and Cara Therapeutics, Inc. (CARA - Free Report) , both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Abeona Therapeutics’ loss per share estimates have narrowed 8.3% for 2020 and 7.2% for 2021 over the past 60 days.

Cara Therapeutics’ loss per share estimates have narrowed 14.2% for 2020 and 22.5% for 2021 over the past 60 days.

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