Statistically significant 68% (p<0.0001) proteinuria reduction compared to placebo, with reduction observed as early as Week 4 All secondary endpoints favored treatment with pegcetacoplan, including: Stabilized eGFR, a key measure of kidney function 71% of patients achieved zero C3c staining intensity, demonstrating complete clearance of C3c deposits Results consistent across subgroups including C3G and IC-MPGN, adolescent and adult patients, and native and post-transplant kidneysDemonstrated favorable safe ...

William Blair has initiated coverage on Apellis Pharmaceuticals, Inc. APLS and notes that Apellis was the first company to successfully target complement C3, which has led to the approval of two therapies: Empaveli (systemic pegcetacoplan) for paroxysmal nocturnal hemoglobinuria and Syfovre (intravitreal pegcetacoplan) for geographic atrophy secondary to age-related macular degeneration.William Blair writes that although Empaveli has established a solid position in the PNH market, short-term challenges are ...

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Thank you for reading my research on Seeking Alpha. If you want to learn even more about my method and how I discover these investment opportunities, please check out my subscription marketplace service, Compounding Healthcare , and sign up for a free trial.It has been almost a year since my previous Apellis Pharmaceuticals, Inc. (NASDAQ: APLS ) article , where I discussed the company's C3 inhibitor technology, as well as the prospects for their flagship treatment, pegcetacoplan. Despite my trepidation abou ...

Apellis Pharmaceuticals (APLS) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its June 2024 negative opinion on the regulatory filing for intravitreal pegcetacoplan to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Shares of the company lost 11.5% on Friday in response.Management expressed deep disappointment in the outcome, stating that millions of GA patients in the EU remain without treatmen ...

WALTHAM, Mass., Sept. 20, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its June 2024 negative opinion on the marketing authorization application of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration. “We are deeply disappointed by this outcome, which leaves millions of Europeans with GA ...

Core Insights - The Phase 3 VALIANT study demonstrated a statistically significant 68% reduction in proteinuria in patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) treated with pegcetacoplan compared to placebo [1][2] - Results were consistent across various subgroups, including different age groups and kidney transplant statuses, indicating broad applicability of the treatment [1][2] - The companies plan to submit data for regulatory approval in the US and EU, with applications expected in early 2025 [1][4] Study Details - The VALIANT study was a randomized, placebo-controlled, double-blinded trial involving 124 patients aged 12 and older, making it the largest trial for these rare kidney diseases [5] - The primary endpoint was the log-transformed ratio of urine protein-to-creatinine ratio (uPCR) at Week 26, with significant results achieved [5] - Key secondary endpoints also showed statistical significance, including composite renal endpoint and proteinuria reduction of at least 50% compared to baseline [2] Safety and Tolerability - Pegcetacoplan exhibited a favorable safety profile, with adverse event rates similar between treatment and placebo groups [3] - No cases of meningitis or serious infections attributed to encapsulated bacteria were reported [3] Market Potential - C3G and IC-MPGN are rare diseases affecting approximately 5,000 people in the US and up to 8,000 in Europe, with a significant portion of patients facing kidney failure within 5 to 10 years of diagnosis [6] - The treatment addresses an urgent need for therapies targeting the underlying causes of these diseases, which currently have no approved treatments [6] Company Collaboration - Sobi and Apellis have global co-development rights for systemic pegcetacoplan, with Sobi holding exclusive ex-U.S. commercialization rights [8]

Core Insights - The Phase 3 VALIANT study demonstrated a statistically significant 68% reduction in proteinuria in patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) treated with pegcetacoplan compared to placebo [1][2] - Results were consistent across various subgroups, including different age groups and kidney transplant statuses [1][2] - The companies plan to submit data for regulatory approval in the US and EU in early 2025 [1][4] Study Details - The VALIANT study was a randomized, placebo-controlled, double-blinded trial involving 124 patients aged 12 and older, making it the largest trial for these rare kidney diseases [5] - The primary endpoint was the log-transformed ratio of urine protein-to-creatinine ratio at Week 26, with additional key secondary endpoints showing significant results [2][5] - All patients who completed the study have enrolled in a long-term extension study [4] Safety and Efficacy - Pegcetacoplan showed a favorable safety profile, with adverse event rates similar between treatment and placebo groups [3][5] - No cases of meningitis or serious infections attributed to encapsulated bacteria were reported [3] Market Potential - C3G and IC-MPGN are rare diseases affecting approximately 5,000 people in the US and up to 8,000 in Europe, with a significant portion of patients facing kidney failure within 5 to 10 years of diagnosis [6] - Pegcetacoplan is already approved for treating paroxysmal nocturnal hemoglobinuria (PNH) and is being investigated for other rare diseases [7] Collaboration and Commercialization - Sobi and Apellis have global co-development rights for pegcetacoplan, with Sobi holding exclusive ex-U.S. commercialization rights [8] - Apellis plans to submit a supplemental new drug application to the FDA in early 2025 [4]

Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) Q2 2024 Results Conference Call August 1, 2024 8:30 AM ET Company Participants Meredith Kaya - SVP of IR & Strategic Finance Cedric Francois - Co-Founder & CEO Adam Townsend - COO Caroline Baumal - Chief Medical Officer Tim Sullivan - CFO Conference Call Participants Jonathan Miller - Evercore Anupam Rama - J.P. Morgan Yigal Nochomovitz - Citigroup Steven Seedhouse - Raymond James Colleen Kusy - Baird Phil Nadeau - TD Cowen Ivy Wang - Jefferies Eliana Merle - UBS ...

Apellis Pharmaceuticals, Inc. (APLS) incurred second-quarter 2024 loss of 28 cents per share (excluding loss on extinguishment of development liability), narrower than the Zacks Consensus Estimate of a loss of 33 cents. The company had incurred a loss of $1.02 per share in the year-ago quarter.Total revenues amounted to $199.7 million in the reported quarter, beating the Zacks Consensus Estimate of $189.5 million. In the year-ago quarter, the company had reported revenues of $95 million.The top line skyrock ...