Latest news with #Sobi
Yahoo
10-07-2025
- Business
- Yahoo
Apellis Sells Aspaveli Ex-US Royalties to Sobi for Up to $300M
Apellis Pharmaceuticals Inc. (NASDAQ:APLS) is one of the most promising stocks according to Wall Street analysts. On July 1, Apellis Pharmaceuticals announced a capped royalty purchase agreement with Sobi. Under this agreement, Apellis will receive up to $300 million in exchange for 90% of its future ex-US royalties for Aspaveli (systemic pegcetacoplan). This non-dilutive financing includes an upfront payment of $275 million in cash and up to $25 million in milestone payments contingent upon European Medicines Agency/EMA approval of Aspaveli for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Apellis will retain exclusive commercialization rights for systemic pegcetacoplan in the US, where it is marketed as EMPAVELI. A biomedical scientist in a lab coat conducting research on biopharmaceutical compounds. The transaction reinforces the shared confidence between Apellis and Sobi in the growth potential of Aspaveli/EMPAVELI in rare kidney diseases. Aspaveli/EMPAVELI is currently approved in the European Union, other global countries, and the US for the treatment of paroxysmal nocturnal hemoglobinuria/PNH, which is a rare blood disorder. Apellis Pharmaceuticals Inc. (NASDAQ:APLS) is a commercial-stage biopharmaceutical company that discovers, develops, and commercializes novel therapeutic compounds for diseases with unmet needs. While we acknowledge the potential of APLS as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey.
Yahoo
06-07-2025
- Business
- Yahoo
Morgan Stanley Raises PT on Apellis Pharmaceuticals (APLS) to $26, Keeps a Hold Rating
Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) is one of the 13 Best Pharma Stocks to Buy According to Wall Street Analysts. On July 1, analyst Judah Frommer from Morgan Stanley raised the firm's price target on Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) to $26.00 from $25.00 while maintaining a Hold rating on the stock. Frommer attributed the rating to the company's market position and recent financial strategies. A biomedical scientist in a lab coat conducting research on biopharmaceutical compounds. He cited the company's significant royalty purchase agreement with Sobi, securing up to $300 million and including a $275 million upfront payment, stating that it would slash Sobi's royalty obligations on Aspaveli sales by 90%. This would result in immediate financial benefits for Apellis and may even ease investor concerns regarding the company's profitability. Despite this positive development, Frommer maintained a cautious outlook for Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) due to its future prospects. He stated that while the anticipated regulatory decisions from the FDA and CHMP and the launches of Empaveli/Aspaveli in new indications are significant events, they bring in as much uncertainty as confidence in the company's market potential. Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) is a commercial-stage biopharmaceutical company that discovers, develops, and commercializes novel therapeutic compounds for treating diseases with unmet needs. Its product portfolio primarily includes EMPAVELI and SYFOVRE. SYFOVRE treats geographic atrophy secondary to age-related macular degeneration (GA), while EMPAVELI treats paroxysmal nocturnal hemoglobinuria (PNH). While we acknowledge the potential of APLS as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None.
Yahoo
01-07-2025
- Business
- Yahoo
Apellis to Receive up to $300 Million from Royalty Purchase Agreement with Sobi for Ex-U.S. Royalties of Aspaveli® (systemic pegcetacoplan)
Apellis to receive $275 million upfront and up to $25 million in milestone payments upon EMA approval in C3G and IC-MPGN Non-dilutive financing further strengthens Apellis' balance sheet Apellis retains full U.S. commercialization rights for systemic pegcetacoplan Transaction underscores shared confidence in the meaningful growth potential in rare kidney diseases WALTHAM, Mass., July 01, 2025 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced a capped royalty purchase agreement with Sobi® under which Apellis will receive up to $300 million in exchange for 90% of Apellis' future ex-U.S. royalties for Aspaveli® (systemic pegcetacoplan). Under the companies' 2020 collaboration agreement, Apellis is eligible for tiered royalties on ex-U.S. sales of Aspaveli ranging from high teens to high twenties. Apellis retains exclusive commercialization rights for systemic pegcetacoplan in the United States, where the product is marketed as EMPAVELI®. 'This transaction reflects our shared conviction in the potential of Aspaveli/EMPAVELI to transform the treatment landscape for patients with rare diseases, including C3G and IC-MPGN,' said Timothy Sullivan, chief financial officer, Apellis. 'Through our collaboration, Sobi has developed a deep understanding of Aspaveli/EMPAVELI's potential to significantly improve patient outcomes and deliver long-term value as a rare disease franchise. Importantly, the non-dilutive funding from this transaction further strengthens our balance sheet and provides significant operational flexibility as we approach sustainable profitability, expand the reach of our approved products, and advance our innovative pipeline.' Aspaveli/EMPAVELI is approved in the European Union, other countries globally, and the U.S. for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It is currently under review in the European Union and the U.S. for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare kidney diseases. An opinion by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) is expected before year-end. In the U.S., the Prescription Drug User Fee Act (PDUFA) action date is July 28, 2025. 'We are pleased to reaffirm our ongoing partnership with Apellis and share their strong belief in Aspaveli/EMPAVELI's potential to deliver significant long-term growth,' said Guido Oelkers, chief executive officer, Sobi. 'We are confident in our ability to reach patients with C3G and IC-MPGN globally following regulatory approvals, by leveraging our broad commercial footprint, deep rare disease expertise, and proven success in PNH.' Transaction details Under the terms of the agreement, Sobi will acquire 90% of Apellis' ex-U.S. royalties for Aspaveli for $275 million in cash. Apellis will also be eligible for up to $25 million in milestone payments upon EMA approval of Aspaveli for C3G and IC-MPGN. The agreement is subject to defined caps tied to Aspaveli's performance. Sobi retains 90% of ex-U.S. royalties until these caps are achieved, after which 100% of all ex-U.S. royalties revert to Apellis. About the Apellis and Sobi CollaborationApellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and its opt-in rights for future development programs are unchanged, exercisable at any time prior to commercialization. Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About C3 Glomerulopathy (C3G) and Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Approximately 50% of people living with C3G and primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or lifelong dialysis.1 Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence.2 The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.3 About ApellisApellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit or follow us on X and LinkedIn. Apellis Forward-Looking Statement Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute 'forward-looking statements' within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding regulatory approval for the treatment of patients with C3G and IC-MPGN in the United States and other jurisdictions and commercial growth or prospects for systemic pegcetacoplan for those indications in the United States and other jurisdictions. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether systemic pegcetacoplan will receive approval for those indications from the FDA or equivalent foreign regulatory agencies when expected or at all; whether the commercialization of systemic pegcetacoplan for C3G or IC-MPGN in the United States or other jurisdictions will provide significant additional revenues or allow the Company to achieve profitability; and any other factors discussed in the 'Risk Factors' section of Apellis' Annual Report on Form 10-K with the Securities and Exchange Commission on February 28, 2025 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Contacts: MediaTracy Vineismedia@ InvestorsNeil Carnahan, CFA References1. C3 glomerulopathy. National Institute of Health, Genetics Home Reference. Accessed November 21, 2019. 2. Tarragón, B, et al. C3 Glomerulopathy Recurs Early after Kidney Transplantation in Serial Biopsies Performed within the First 2 Years after Transplantation. Clinical Journal of the American Society of Nephrology. August 2024; 19(8)1005-1015. doi: 10.2215/CJN.0000000000000474.3. Data on file using literature in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Medscape
30-06-2025
- Health
- Medscape
FDA Approves First Treatment for MAS in Still Disease
The FDA has approved the interferon gamma-blocking antibody emapalumab-lzsg (Gamifant) for the treatment of macrophage activation syndrome (MAS) in known or suspected Still disease. This is the first FDA-approved treatment for MAS in the United States. The indication is specifically for adult and pediatric (newborn and older) patients with MAS in Still disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. Emapalumab-lzsg was first approved in 2018 for adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy. "With our expertise in primary hemophagocytic lymphohistiocytosis, we understand the urgency of managing MAS quickly to improve patient outcomes," said Guido Oelkers, PhD, the CEO of the drug's manufacturer, Sobi, in a press release. "Gamifant is already an established therapy making a meaningful difference for patients with primary HLH, and with this approval, we are excited about the opportunity to positively impact patients affected by MAS in Still's disease.' The approval for this indication was supported by pooled data from a phase 2 and a phase 3 clinical trial: More than half (54%) of participants had a complete response at week 8, and 82% achieved clinical MAS remission at week 8. "With Gamifant now as the first FDA-approved treatment for MAS, we have a new therapeutic option that helps control hyperinflammation and reduce our reliance on high-dose glucocorticoids,' added Alexei Grom, MD, professor of pediatrics and research director in the Division of Rheumatology at Cincinnati Children's Hospital Medical Center, Ohio, in a press statement. Emapalumab-lzsg is administered via intravenous infusion and is available in 5-mg/mL and 25-mg/mL solutions in a single-dose vial. For this new indication, the most common side effects are viral infections, such as cytomegalovirus infection or reactivation, or rash. Patients on emapalumab-lzsg should not receive live or live attenuated vaccines. Healthcare providers should consider prophylactic treatment for herpes zoster, Pneumocystis jirovecii, and fungal infections.
Yahoo
30-06-2025
- Health
- Yahoo
Sobi gains FDA approval for Gamifant to treat HLH/MAS
The US Food and Drug Administration (FDA) has approved Sobi's Gamifant (emapalumab) to treat adults and children with haemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) associated with Still's disease, including juvenile idiopathic arthritis (sJIA), where there is a reduced response or intolerance to glucocorticoids, or cases of recurrent MAS. Gamifant functions as an anti-interferon gamma (IFNγ) monoclonal antibody. It is offered in three strengths:10mg/2 ml, 50mg/10ml and 100mg/20ml vials, administered intravenously. Sobi CEO Guido Oelkers stated: 'With our expertise in primary hemophagocytic lymphohistiocytosis, we understand the urgency of managing MAS quickly to improve patient outcomes. 'Gamifant is already an established therapy making a meaningful difference for patients with primary HLH, and with this approval, we are excited about the opportunity to positively impact patients affected by MAS in Still's disease.' The FDA approval was based on pooled data from two pivotal studies: the Phase III EMERALD trial and NI-0501-06. A complete response (CR) at week eight was observed in 54% of patients, while 82% achieved clinical MAS remission at the same interval. Safety and tolerability profiles were consistent with those seen in earlier clinical trials. HLH/MAS can be a severe complication within rheumatic diseases such as systemic sJIA and adult-onset Still's disease. This rare disorder is characterised by hyperinflammation driven by IFNy, leading to symptoms like persistent high fever, elevated ferritin levels, coagulopathies, cytopenias and hepatosplenomegaly. In February 2025, the company obtained FDA approval for Gamifant's supplemental biologics licence application. Gamifant is also authorised in the US for patients with primary HLH with refractory, persistent or progressive disease or intolerant to standard therapy. "Sobi gains FDA approval for Gamifant to treat HLH/MAS" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
