- Sobi obtains global co-development and exclusive ex-
U.S. commercialization rights for systemic pegcetacoplan, a targeted C3 therapy - Apellis retains
U.S. commercialization rights for systemic pegcetacoplan and worldwide commercialization rights for ophthalmological pegcetacoplan (geographic atrophy program in Phase 3) - The companies will jointly advance systemic pegcetacoplan in five parallel registrational programs including two new hematological studies planned to start in 2021 (CAD and HSCT-TMA). These join ongoing registrational programs in hematology (PNH), nephrology (IC-MPGN/C3G) and neurology (ALS)
- Sobi will make an upfront payment of
$250 million to Apellis and$80 million in committed development reimbursements over four years and up to$915 million in regulatory and commercial milestones plus tiered double-digit royalties - Apellis to host conference call today at
8:30 a.m. ET
Sobi will receive global co-development and exclusive ex-US commercialization rights for systemic pegcetacoplan. Apellis retains
Apellis and Sobi plan to jointly advance the clinical development of systemic pegcetacoplan in five parallel registrational programs across hematology, nephrology, and neurology. These include new registrational programs in cold agglutinin disease (CAD) and hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA), both of which are expected to start in 2021. By controlling complement activation centrally, pegcetacoplan offers the potential to become a transformative new therapy in several rare diseases where patients have few or no treatment options today.
“This collaboration enables us to further expand on the broad platform potential of targeting C3 for serious rare diseases that impact hundreds of thousands of patients around the world,” said
“We are excited to collaborate with Apellis, a leader in targeted C3 therapies. The collaboration will significantly strengthen and broaden our late-stage R&D portfolio and be a catalyst for further internationalization. The products have an excellent fit with our strategic focus on hematology and immunology,” said
As part of the collaboration, Apellis and Sobi will co-develop systemic pegcetacoplan in the following rare diseases:
Hematology – Paroxysmal nocturnal hemoglobinuria (PNH), CAD, and HSCT-TMA
PNH represents the first potential indication to market for systemic pegcetacoplan. Marketing applications for pegcetacoplan for the treatment of PNH were submitted to the
Sobi will lead development activities for the Phase 3 study in CAD and a potentially registrational Phase 2 study in HSCT-TMA, both planned to start in 2021.
Nephrology – Immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G)
Apellis has initiated and will continue to lead a registrational program in IC-MPGN and C3G, which includes Phase 2 and Phase 3 studies.
Neurology – Amyotrophic lateral sclerosis (ALS)
Apellis has initiated and will continue to lead a potentially registrational Phase 2 study in ALS. Multiple other neurological conditions are under consideration for future clinical development.
About the Transaction
Sobi will make an upfront payment of
Per the terms of the agreement, Apellis will be responsible for all regulatory and commercial activities in
Conference Call and Webcast
Apellis will host a conference call and webcast to discuss its collaboration with Sobi today,
About Pegcetacoplan (APL-2)
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the
About Pegcetacoplan for Paroxysmal Nocturnal Hemoglobinuria (PNH)
In October, the
The NDA and MAA submissions for pegcetacoplan for the treatment of PNH are based on positive results from the Phase 3 PEGASUS study (APL2-302; NCT03500549), a multi-center, randomized, active-comparator controlled Phase 3 study in 80 adults with PNH. The primary objective of PEGASUS was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Pegcetacoplan is also being evaluated in the Phase 3
About PNH
PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria, and difficulty breathing (dyspnea). A retrospective analysis shows that, even on eculizumab, approximately 72% of people with PNH have anemia, a key indicator of ongoing hemolysis.1 The analysis also finds that 36% of patients require one or more transfusions a year and 16% require three or more.1
About Cold Agglutinin Disease (CAD)
CAD is a severe, chronic, rare blood disorder2 that currently has no approved therapies and impacts ~10,500 people across
About Hematopoietic Stem Cell Transplantation Thrombotic Microangiopathy (HSCT-TMA)
HSCT-TMA is rare blood disease that can be a fatal complication of a bone marrow transplant or HSCT.8 In HSCT-TMA, microscopic blood clots form in small blood vessels, leading to organ damage. The kidneys are commonly affected, although any organ may be involved.8 HSCT-TMA occurs in up to 40% of HSCT recipients;9 every year, there are ~9,000 allogeneic transplants in
About Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) and C3 Glomerulopathy (C3G)
IC-MPGN and C3G are rare, debilitating kidney diseases that affect ~18,000 people in
About Amyotrophic Lateral Sclerosis (ALS)
ALS is a devastating neurodegenerative disease that results in progressive muscle weakness and paralysis due to the death of nerve cells, called motor neurons, in the brain and spinal cord.19, 20 The death of motor neurons leads to the progressive loss of voluntary muscle movement required for speaking, walking, swallowing and breathing.19,20 In individuals with ALS, high levels of C3 are present at the neuromuscular junction21 where motor neurons communicate directly to muscle cells. Numerous studies suggest that elevated levels of C3 present throughout the motor system of ALS patients are likely to contribute to chronic neuroinflammation and the death of motor neurons.21,22,23 There are no treatments that stop or reverse the progression of ALS, which impacts ~225,000 patients worldwide.24
About Apellis
About Sobi
Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of hematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across Europe, North America, the Middle East, Russia and North Africa. In 2019, Sobi's revenue amounted to SEK 14.2 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at www.sobi.com.
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 relating to the implications of preliminary clinical data. 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 the company’s clinical trials will be fully enrolled and completed when anticipated; whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; whether results obtained in preclinical studies and clinical trials will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company’s clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G, IC-MPGN, ALS or any other indication when expected or at all; whether, if Apellis’ products receive approval, they will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on
Contacts:
Apellis
Media
media@apellis.com
+1 617 420 4839
Investors
sam@argotpartners.com / maghan@argotpartners.com
+1 212 600 1902
Sobi
Paula Treutiger, Head of Communication & Investor Relations
+ 46 733 666 599
paula.treutiger@sobi.com
+ 46 708 734 095
linda.holmstrom@sobi.com Apellis
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2 Sokol RJ, Hewitt S, Stamps BK. Autoimmune haemolysis: an 18-year study of 865 cases referred to a regional transfusion centre. Br Med J (
3 Catenion using physician and literature consensus
4 Website https://rarediseases.info.nih.gov/diseases/6130/cold-agglutinin-disease. Accessed
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6 Cold agglutinin disease. Genetic and
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10 Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides
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13 Noris M, et al. STEC-HUS, atypical HUS and TTP are all diseases of complement activation. Nature Reviews Nephrology. 8, 622–633 (2012)
14 ClearView Analysis using physician and literature consensus.
15 Complement 3 Glomerulopathy (C3G). National Kidney Foundation Website. https://www.kidney.org/atoz/content/complement-3-glomerulopathy-c3g. Accessed
16 C3 glomerulopathy.
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18 Cook HT. Evolving complexity of complement-related diseases: C3 glomerulopathy and atypical haemolytic uremic syndrome. Curr Opin Nephrol Hypertens. 2018 May;27(3):165-170.
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22 Woodruff, et al., PNAS January 7, 2014 111 (1) E3-E4
23 Lee, et al
24 Arthur K et al.
Source:
2020 GlobeNewswire, Inc., source