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 | | Exhibit 99.1 |
Apellis Pharmaceuticals Announces that All Four Severely Anemic Soliris™ - Treated Patients with Paroxysmal Nocturnal Hemoglobinuria in Ongoing Phase 1b Trial Have Been Transitioned toAPL-2 Monotherapy
PHAROAH Trial Resultsto-Date ShowAPL-2 Monotherapy Eliminated Transfusion Dependency and Improved Markers of Anemia in PNH Patients on Soliris™
CRESTWOOD, Ky. and WALTHAM, Mass., Sept. 04, 2018 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals, Inc. (Nasdaq:APLS), a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, today announced an update on itsUS-based Phase 1b PHAROAH trial for patients with paroxysmal nocturnal hemoglobinuria (PNH). The ongoing PHAROAH trial is evaluating treatment withAPL-f2, a novel inhibitor of complement factor C3, in patients on treatment with eculizumab (Soliris™) who are severely anemic and transfusion-dependent.
Patients enrolled in the PHAROAH trial were initiallyco-treated withAPL-2 and eculizumab, with the potential for discontinuation of eculizumab at the discretion of the treating physician. Two of the six patients were removed from treatment withAPL-2 due to pregnancy andBMI-associated comorbidities. Four of the six patients continued in the study for more than 32 weeks. In these four patients,co- treatment withAPL-2 resulted in an improvement of hemoglobin levels and other markers for anemia and none of the four patients required a transfusion during theco-treatment period with eculizumab, which ranged from 17 to 20 months. Earlier this year Apellis announced that three of the four patients discontinued treatment with eculizumab and were continuing to receiveAPL-2. The fourth patient has now discontinued treatment with eculizumab and is continuing to receiveAPL-2. All four patients have improved hemoglobin and reticulocytes as compared to the baseline established with eculizumab monotherapy and have achieved lactate dehydrogenase (LDH) levels below the upper limit of normal (ULN).
| | | | | | |
| | | Eculizumab Monotherapyi | | APL-2 + Eculizumabii | | APL-2 Monotherapyiii |
Hemoglobin (g/dL)* | | 8.9 | | 11.9 | | 11.4 |
Annual Transfusions (avg.) | | 6.0 | | 0 | | 0 |
LDH (ULN)* | | 1.0x | | 0.8x | | 0.9x |
Reticulocytes (ULN)* | | 2.7x | | 1.2x | | 0.8x |
Patient Years (Total) | | NA | | 5.9 Years | | 1.9 Years |
Multiple of Eculizumab Label Dose (900mgx2wk.) | | 1.6x | | 1.0x | | — |
| * | Average last available reading for all four patients on each dosing regimen |
| (i) | last reading during eculizumab monotherapy prior toco-treatment withAPL-2 |
| (ii) | last reading duringco-treatment and prior toAPL-2 monotherapy |
| (iii) | last reading while onAPL-2 monotherapy |
“By eliminating transfusion dependency and improving markers of anemia in patients in the study,APL-2 has given these patients a meaningful clinical benefit over eculizumab monotherapy,” said Dr. Cedric Francois, M.D., Ph.D.,co-founder and CEO of Apellis. “This is precisely the benefit that we are aiming to confirm in our ongoing PEGASUS Phase 3head-to-head trial against eculizumab.”
In addition to the PHAROAH trial and the ongoing Phase 3head-to-head trial against eculizumab,APL-2 is also being evaluated in the PADDOCK Phase 1b trial in treatment naïve patients. Apellis previously reported that the patients in the PADDOCK trial experienced a mean 3.5 g/dL improvement in hemoglobin and an average reduction in LDH to within the normal range after daily subcutaneous administration ofAPL-2 for at least 28 days.
To date, subcutaneousAPL-2 has been well-tolerated with cumulative systemic exposure of over 18 patient years of treatment onAPL-2. No significant infections or thromboembolic events have been observed.
About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes and/or some degree of bone marrow dysfunction. A significant subset of patients treated with the current standard of care still suffer from debilitating anemia and transfusion dependence.
About the PHAROAH Trial
PHAROAH is an ongoing open label safety and efficacy study of 270 mg ofAPL-2 administered daily by subcutaneous injection as a complementary therapy to patients with PNH who continue to be anemic (Hb <10 g/dL at screening or have a history of at least one transfusion in the previous year) despite treatment with eculizumab. The PHAROAH study was initiated in November 2014 and is being conducted at multiple clinical sites in the United States.
About the PEGASUS Phase 3 Trial
PEGASUS is a70-patient, randomized,head-to-head study comparingAPL-2 monotherapy to eculizumab monotherapy in patients currently on treatment with eculizumab who have a hemoglobin level <10.5 g/dL, regardless of eculizumab dose or transfusion history. Patients will beco-treated for 4 weeks with twice weekly subcutaneous infusions of 1080 mg ofAPL-2 alongside their existing eculizumab regimen and then randomized 1:1 to eitherAPL-2 monotherapy or eculizumab monotherapy and monitored for 56 weeks. The primary endpoint will be change in hemoglobin from baseline to Week 16. Secondary endpoints include change from baseline to Week 16 in absolute reticulocyte count, LDH levels and FACIT-fatigue score. The number of packed red blood cell units transfused from Week 4 to 16 will also be compared.
