Apellis Pharmaceuticals
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- -
- Employees
- 706
- Market Cap
- -
- Introduction
Apellis Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company, which engages in the discovery, development, and commercialization of novel therapeutic compounds to treat diseases with high unmet needs. Its products include EMPAVELI and SYFOVRE. The company was founded by Candace Rose Depp, Pascal Deschatelets, Cedric Francois, and Alec Machiels on September 25, 2009 and is headquartered in Waltham, MA.
Pharma Leadership Shuffle: Key Executive Moves in Early 2025 Reshape Industry Landscape
• The pharmaceutical industry has experienced significant leadership changes in early 2025, with major transitions at companies including Pfizer, Boehringer Ingelheim, and Takeda.
• Former FDA CDER Director Patricia Cavazzoni's move to Pfizer as Chief Medical Officer has sparked "revolving door" criticisms, highlighting ongoing concerns about regulatory-industry transitions.
• Several biotechnology firms have strengthened their executive teams with specialized expertise in clinical development, particularly in areas like stroke treatment, antibody-drug conjugates, and neuropsychiatric therapies.
Boehringer Ingelheim Launches Phase II Trial of First-in-Class Oral Treatment for Geographic Atrophy
• Boehringer Ingelheim has initiated the JADE Phase II clinical study to evaluate BI 1584862, a potential first-in-class oral treatment for geographic atrophy, a severe form of age-related macular degeneration.
• Geographic atrophy affects over 5 million people worldwide with more than 40% considered legally blind, creating a significant unmet need for treatments that can slow disease progression and preserve vision.
• The company is simultaneously advancing a second compound, BI 771716, an antibody fragment designed to penetrate retinal layers, demonstrating Boehringer's dual-pathway approach to addressing this debilitating eye condition.
Novartis' FABHALTA Poised to Transform Treatment Landscape for Multiple Complement-Mediated Diseases
• FABHALTA (iptacopan), Novartis' first-in-class oral factor B inhibitor, has received FDA approvals for paroxysmal nocturnal hemoglobinuria (PNH), IgA nephropathy (IgAN), and C3 glomerulopathy (C3G), marking a significant shift from traditional intravenous therapies.
• The drug's oral administration route and ability to control both intravascular and extravascular hemolysis addresses key limitations of current therapies, potentially expanding the market for complement-targeted treatments estimated at over $1.2 billion in 2024.
• With patent protection extending to 2041 and ongoing investigations for additional indications including myasthenia gravis and atypical hemolytic uremic syndrome, FABHALTA is expected to significantly reshape the complement inhibitor market landscape.
FDA to Phase Out Animal Testing Requirement for Monoclonal Antibodies and Drugs
• The FDA announced it will phase out requirements for animal testing of monoclonal antibodies and other drugs, citing the availability of "more effective, human-relevant methods" for safety evaluation.
• The initiative aims to improve drug safety, accelerate evaluation processes, reduce R&D costs, and ultimately lower drug prices by implementing AI-based computational models, humanoid models, and real-world human data.
• This regulatory shift, enabled by the bipartisan FDA Modernization Act 2.0 of 2022, represents what FDA Commissioner Marty Makary calls "a paradigm shift in drug evaluation" that could expedite development of new treatments.
Merida Biosciences Launches with $121M to Target Disease-Causing Antibodies in Autoimmune Conditions
• Merida Biosciences has secured $121 million in Series A financing co-led by Bain Capital Life Sciences, BVF Partners, and Third Rock Ventures to develop therapeutics targeting pathogenic antibodies in autoimmune diseases.
• The company's proprietary platform aims to develop Fc-based biologics that selectively eliminate harmful antibodies while sparing healthy immune components, with lead programs focusing on Graves' disease, food allergies, and primary membranous nephropathy.
• Former Apellis Pharmaceuticals COO Adam Townsend will lead Merida as CEO, positioning the company's approach as potentially offering more targeted alternatives to current treatments without broad immunosuppression.
Biotech Leadership Shuffle Continues as Industry Navigates Transformation in Q2 2025
• Novo Nordisk undergoes significant executive restructuring following the departure of EVP Camilla Sylvest, with rare disease leader Ludovic Helfgott taking over expanded commercial strategy responsibilities.
• Galapagos faces major leadership transition as CEO Paul Stoffels announces retirement plans while CFO Thad Huston departs, amid the company's ongoing transformation into a cell therapy-focused biotech.
• Multiple biotechs including Hansa Biopharma, Creyon Bio, and Sutro Biopharma appoint new CEOs as the industry continues strategic realignment efforts.
• Emerging companies across oncology, rare diseases, and AI-driven drug development strengthen their leadership teams with experienced executives from major pharmaceutical companies.
Character Biosciences Secures $93M to Advance Precision Therapies for Age-Related Macular Degeneration
• Character Biosciences has raised $93 million in Series B funding to advance two drug candidates for age-related macular degeneration (AMD) into clinical trials this year.
• The company's lead candidates include CTX114, targeting geographic atrophy, and CTX203, designed to prevent progression in intermediate AMD patients by regulating lipid transport.
• Character's precision medicine approach involves analyzing data from over 6,500 AMD patients to identify genetic subtypes, potentially improving upon current treatments like Syfovre and Izervay.
Zervimesine Shows Promise in Geographic Atrophy Trial, Building on Success in Multiple CNS Disorders
• Cognition Therapeutics' Phase 2 MAGNIFY trial of zervimesine demonstrates slower lesion growth in geographic atrophy patients compared to placebo after 6 months of treatment.
• The drug previously showed significant benefits in dementia with Lewy bodies trials, with patients showing 86% improvement in neuropsychiatric symptoms and 62% better motor function versus placebo.
• Zervimesine also demonstrated strong results in Alzheimer's disease trials, achieving a 95% reduction in cognitive decline in a biomarker-defined subgroup compared to placebo.
EMA Validates Aspaveli® Extension Application for Rare Kidney Diseases C3G and IC-MPGN
• The European Medicines Agency has validated the application to extend Aspaveli® (pegcetacoplan) use for treating C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis, two rare kidney diseases currently lacking approved treatments.
• Phase 3 VALIANT study demonstrated significant 68% reduction in proteinuria with pegcetacoplan compared to placebo (p<0.0001), along with stabilization of kidney function and reduced C3c staining intensity.
• The potential approval could provide the first targeted treatment for approximately 8,000 European patients affected by these conditions, with possible US launch in second half of 2025.
Astellas Seeks First Japanese Approval for Geographic Atrophy Treatment with Avacincaptad Pegol
• Astellas Pharma has submitted a New Drug Application to Japan's MHLW for avacincaptad pegol, potentially becoming the first approved treatment for geographic atrophy in Japan.
• The submission is supported by GATHER1 and GATHER2 clinical trials, which demonstrated the drug's ability to slow GA lesion growth with a favorable safety profile over two years.
• Geographic atrophy affects over 5 million people globally, with 66% at risk of becoming legally blind without treatment, highlighting the significant unmet medical need.
Apellis' Syfovre Approved in Australia for Geographic Atrophy
• Apellis Pharmaceuticals' Syfovre (pegcetacoplan) has been approved in Australia for treating geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
• Syfovre is the first and only approved treatment for GA in Australia, offering a new option to slow the progression of this irreversible vision loss.
• The approval is based on Phase 3 OAKS and DERBY studies, which demonstrated that Syfovre slowed GA progression with a generally well-tolerated safety profile.
• Affecting over 75,000 Australians, GA leads to progressive vision loss, impacting independence and quality of life, making this approval a significant advancement.
FDA Accepts Aldeyra's Reproxalap NDA for Dry Eye Disease with AbbVie Collaboration
• The FDA has accepted Aldeyra Therapeutics' New Drug Application (NDA) for reproxalap, a topical ocular therapy for dry eye disease, with a PDUFA date of April 2, 2025.
• Reproxalap, a first-in-class RASP modulator, has demonstrated significant clinical efficacy and safety in reducing the signs and symptoms of dry eye disease in multiple late-stage trials.
• Aldeyra has expanded its collaboration with AbbVie to accelerate pre-commercial activities, with AbbVie covering 60% of the costs and a potential $100 million upfront payment upon exercising its option.
• The partnership includes up to $300 million in milestone payments and a profit-sharing model for U.S. commercialization, positioning reproxalap as a potential game-changer in dry eye disease treatment.
UNITY Biotechnology Appoints Federico Grossi as Chief Medical Officer
• UNITY Biotechnology appointed Federico Grossi as Chief Medical Officer, enhancing its leadership with his extensive experience in ophthalmology and drug development.
• Dr. Grossi previously led the development of SYFOVRE for geographic atrophy at Apellis Pharmaceuticals, bringing a proven track record to UNITY.
• His appointment is timely as UNITY anticipates Phase 2b ASPIRE trial results for UBX1325 in diabetic macular edema (DME).
• UNITY aims to leverage Dr. Grossi's expertise to advance its pipeline of senolytic treatments for age-related retinal diseases.
Emerging Therapies Show Promise for Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
• IC-MPGN affects approximately 7,100 individuals across the 7MM, with the US accounting for 55% of cases, highlighting a significant unmet need for targeted treatments.
• The IC-MPGN market in the US, valued at approximately $12 million in 2023, is projected to grow by 2034 due to increased diagnosis and the introduction of premium-priced emerging therapies.
• Late-stage drugs like Iptacopan (Novartis) and Pegcetacoplan (Apellis) are under development, offering potential as targeted complement inhibitors for this rare kidney disease.
• Current IC-MPGN treatment relies on off-label drugs such as immunosuppressants and steroids, underscoring the need for approved therapies addressing the root cause of the disease.
FDA Rejects Astellas' Izervay Update; Cytokinetics and Bayer Announce Aficamten Licensing Deal
• The FDA rejected Astellas' request to update Izervay's prescribing information for bi-monthly injections due to a statistical matter, maintaining Apellis' dosing advantage.
• Cytokinetics granted Bayer exclusive rights to develop and commercialize aficamten in Japan, with potential milestone payments for Cytokinetics.
• Jupiter Bioventures, founded by Norman Sharpless and Nathaniel David, launched with $70 million to create startups focused on cancer and other diseases.
Novartis and Apellis Advance C3G Therapies Towards FDA Submission
• Novartis' Fabhalta (iptacopan) demonstrated significant proteinuria reduction in the Phase III APPEAR-C3G trial, meeting its primary endpoint and supporting regulatory submissions.
• Apellis' Empaveli (pegcetacoplan) showed a substantial reduction in proteinuria and C3c deposit clearance in the Phase III VALIANT trial, indicating early efficacy in C3G patients.
• Nephrologists express a strong interest in new C3G therapies that can slow eGFR decline and reduce proteinuria, addressing the urgent need for innovative treatments.
• Spherix data suggests treatment preferences may be influenced by administration route, with Fabhalta's oral administration potentially offering an advantage.
Emerging Therapies Offer Hope for Geographic Atrophy Treatment
• Several therapies are under evaluation in clinical trials for geographic atrophy (GA) secondary to age-related macular degeneration (AMD), addressing the unmet need for effective treatments.
• Complement pathway inhibitors like ANX007, danicopan, and AVD-104 are being investigated to modulate the inflammatory response and cell lysis associated with GA progression.
• Gene therapies such as JNJ-1887 and cell-based therapies like ASP7317 and RG6501 are showing promise in protecting retinal cells and improving visual function in GA patients.
• Antioxidants like elamipretide, glideuretinal, and AREDS2 supplements are also being explored for their potential to reduce GA lesion progression and improve visual outcomes.
Handok's Empaveli (pegcetacoplan) Receives Korean Health Insurance Coverage for PNH Treatment
• Handok's Empaveli (pegcetacoplan), a C3-targeted therapy, has secured health insurance coverage in Korea for paroxysmal nocturnal hemoglobinuria (PNH) treatment.
• Empaveli addresses both intravascular and extravascular hemolysis, offering an alternative for PNH patients who cannot use or have not responded well to C5 inhibitors.
• Clinical trials, including PEGASUS and PRINCE, demonstrated Empaveli's superiority over eculizumab in improving hemoglobin levels and reducing the need for transfusions.
• Reimbursement criteria prioritize use for patients with contraindications to C5 inhibitors or those with inadequate response or side effects from C5 inhibitor treatments.
Advances in Multiple Myeloma Treatment Highlighted at IMS 2024
• Updated results from the TRIMM-2 study showed that talquetamab, daratumumab, and pomalidomide achieved an 82% overall response rate in relapsed/refractory multiple myeloma.
• The CEPHEUS trial demonstrated that adding subcutaneous daratumumab to VRd improved minimal residual disease negativity in newly diagnosed, transplant-ineligible multiple myeloma patients.
• CARTITUDE-4 trial updates revealed ciltacabtagene autoleucel reduced the risk of death by 45% compared to standard care in lenalidomide-refractory multiple myeloma.
• The RedirecTT-1 study indicated that talquetamab plus teclistamab delivered high rates of durable responses in triple-class exposed relapsed/refractory multiple myeloma.
AI-Powered OCT Analysis Enhances Understanding of Geographic Atrophy Progression and Treatment Response
• AI-driven analysis of OCT images offers a more precise and automated method for assessing geographic atrophy (GA) progression compared to traditional methods.
• Post-hoc analysis of the FILLY trial demonstrates AI's superior accuracy in predicting GA growth rates using OCT data compared to expert ophthalmologists.
• Studies of the OAKS and DERBY trials reveal that AI-quantified changes in retinal pigment epithelium (RPE) and ellipsoid zone (EZ) loss correlate with the therapeutic effects of pegcetacoplan.
• AI-based tools, accessible via cloud technology, hold promise for improving clinical endpoints and benefiting healthcare providers and patients in managing GA.
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