Apellis Pharmaceuticals Reports Second Quarter 2019 Business Update and Financial Results
Completed Enrollment in Phase 3 Paroxysmal Nocturnal Hemoglobinuria (PNH) Head-to-Head Study in Patients Treated with Eculizumab; Top-line Data Anticipated in December 2019
Expects to Initiate Phase 3 Cold Agglutinin Disease (CAD) Trial in Early 2020
Cash Position of $289.1 Million at Quarter-End
CRESTWOOD, Ky. and WALTHAM, Mass., July 31, 2019 (GLOBE NEWSWIRE) -- 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 its second quarter 2019 financial results and business highlights.
“As we continue to evolve towards becoming a commercial stage company with a deep pipeline, the second quarter of 2019 encompassed several significant advancements for Apellis,” said Cedric Francois, CEO and co-founder of Apellis. “First, we completed enrollment in the Phase 3 PEGASUS trial for APL-2 in patients with PNH and will release top-line data in December 2019. Second, we now expect to commence a Phase 3 trial for APL-2 in patients with CAD in early 2020, which would represent the third indication for APL-2 in pivotal trials. Finally, our pipeline continues to progress, with Phase 2 complement-dependent nephropathy data anticipated in November 2019 at the American Society of Nephrology (ASN) Kidney Week and with the development of APL-9 for the prevention of complement immune system activation coincident with adeno-associated virus (AAV) vector administration for gene therapies. These advances highlight Apellis’ relentless commitment to delivering innovative therapies that target diseases through the modulation of the complement system.”
Business Highlights and Upcoming Milestones:
APL-2 in Systemic Indications
- In June 2019, Apellis the completion of enrollment of 80 patients for its Phase 3 trial (APL2-302) assessing the safety and efficacy of APL-2 in patients with paroxysmal nocturnal hemoglobinuria (PNH) compared to eculizumab. Apellis expects to release top-line data in December 2019.
- In June 2019, Apellis updated data from its Phase 2 trial (APL2-CP-AIHA-208) of APL-2 in patients with autoimmune hemolytic anemia (AIHA), including cold agglutinin disease (CAD) and warm antibody autoimmune hemolytic anemia (wAIHA). Data from the PLAUDIT study were at the 24th Annual Congress of the European Hematology Association (EHA). Based on the data, Apellis expects to move forward with a Phase 3 trial of APL-2 in patients with CAD in early 2020. Apellis will continue to evaluate the potential of APL-2 for patients with C3+ wAIHA.
- Apellis expects that data from the ongoing Phase 2 monotherapy trial of APL-2 in four types of complement-dependent nephropathies (IgA nephropathy, C3 glomerulopathy, primary membranous nephropathy and lupus nephritis) will be presented at the American Society of Nephrology (ASN) Kidney Week in November 2019.
APL-2 in Geographic Atrophy (GA)
- Apellis expects that the two Phase 3 trials for APL-2 in patients with GA will be fully enrolled by the end of the first quarter of 2020.
APL-9
- In July 2019, Apellis its plans to develop APL-9 for the prevention of complement immune system activation coincident with AAV vector administration for gene therapies. In Phase 1 testing in healthy volunteers, APL-9 demonstrated control of complement through modulation of C3 within 1 hour of administration, that lasted up to 12 hours after the end of the intravenous infusion.
Corporate & Other Highlights
- In July 2019, Apellis the expansion of its leadership team with the appointment of Lucia Celona as Chief People Officer. Celona will be responsible for overseeing the strategy, development, and execution of all aspects of the Company’s Human Resources function. Prior to joining Apellis, Celona was the Chief HR and Communications Officer at Bioverativ.
- In June 2019, Apellis that it entered into an amendment to the development funding with SFJ Pharmaceuticals®. Under the amendment, SFJ agreed to pay the Company an additional $20 million to support the development of APL-2 for the treatment of patients with PNH. In connection with this additional payment, the Company agreed to increase the amount it will pay to SFJ upon regulatory approval for APL-2 in patients with PNH by the FDA or the EMA by $35 million (or $70 million if regulatory approval is granted by the FDA and the EMA).
Second Quarter 2019 Financial Results:
As of June 30, 2019, Apellis had $289.1 million in cash and cash equivalents, compared to $176.3 million as of December 31, 2018. This includes $109.6 million in net proceeds raised in the public offering of common stock in March 2019 and $100.0 million in payments received through the collaboration with SFJ Pharmaceuticals.
Apellis reported a net loss of $71.1 million for the second quarter of 2019, compared to a net loss of $33.3 million for the second quarter of 2018.
Research and development expenses were $50.7 million in the second quarter of 2019, compared to $27.5 million for the same period in 2018. The increase was primarily attributable to an increase of $6.3 million in clinical trial costs, an increase of $12.5 million in manufacturing expenses, an increase of $3.8 million in employee related costs primarily due to the hiring of additional personnel, an increase of $0.4 million related to research and development supporting activities, and an increase of $0.6 million in pre-clinical study expenses, offset by a decrease of $0.4 million in device development expenses.
General and administrative expenses were $12.8 million in the second quarter of 2019, compared to $5.9 million for the same period in 2018. The increase was primarily attributable to an increase in employee related costs of $3.0 million due to the hiring of additional personnel, an increase in professional and consulting fees of $3.6 million, an increase in general office costs of $0.7 million and an increase of $0.1 million in insurance costs offset by a decrease of $0.4 million in license agreement costs and $0.2 million in director stock compensation expense.
About APL-2 (pegcetacoplan)
APL-2, an investigational drug, is designed to inhibit the complement cascade centrally at C3 and may have the potential to treat a wide range of complement-mediated diseases more effectively than is possible with partial inhibitors of complement. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation (classical, lectin, and alternative). Apellis is currently evaluating APL-2 in clinical studies in patients with geographic atrophy (GA), in patients with paroxysmal nocturnal hemoglobinuria (PNH) who are being treated with eculizumab or who are naïve to complement inhibitor treatment, in patients with warm autoimmune hemolytic anemia (wAIHA) or cold agglutinin disease (CAD) and in patients with complement-dependent nephropathies. For additional information regarding our clinical trials, visit .
About APL-2 in Hematologic Diseases
Apellis is currently evaluating APL-2 in PEGASUS, a Phase 3 trial for patients with PNH currently on treatment with eculizumab, as well as in two Phase 1b trials (PHAROAH and PADDOCK) for systemic administration to patients with PNH. Previously reported interim data from these Phase 1b trials showed improvements in lactate dehydrogenase and hemoglobin levels in patients who are suboptimal responders to eculizumab and untreated patients, respectively. Apellis is also testing APL-2 in a Phase 2 open-label trial assessing the safety, tolerability, efficacy, and PK of multiple subcutaneous (SC) doses of APL-2 administered daily in patients with wAIHA or CAD. In this trial to date, APL-2 has shown the potential to improve hemoglobin, reticulocytes, bilirubin and lactate dehydrogenase levels.
About APL-9
APL-9, an investigational drug, is designed to modulate the complement cascade centrally at C3 and may have the potential to treat a range of complement-mediated conditions more effectively than is possible with partial inhibitors of complement. APL-9, is a second-generation C3 modulator that leverages the same mechanism of action as Apellis’ lead compound, APL-2 (pegcetacoplan), but has a lower molecular weight and shorter half-life. APL-9 is designed to be intravenously administered for acute use whereas APL-2 is designed for chronic subcutaneous or intravitreal administration.
About Apellis
Apellis Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds for the treatment of a broad range of life-threatening or debilitating autoimmune diseases based upon complement immunotherapy through the inhibition of the complement system at the level of C3. Apellis is the first company to advance chronic therapy with a C3 inhibitor into clinical trials. For additional information about Apellis and APL-2, please visit .
Forward-Looking Statements
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 APL-2 or APL-9 will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of such clinical trials will warrant regulatory submissions and whether APL-2 will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, wAIHA or any other indication; 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 July 31, 2019 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.
| APELLIS PHARMACEUTICALS, INC. | ||||||||
| CONDENSED CONSOLIDATED BALANCE SHEETS | ||||||||
| December 31, | June 30, | |||||||
| 2018 | 2019 | |||||||
| Assets | (Unaudited) | |||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 176,267,666 | $ | 289,128,569 | ||||
| Refundable research and development credit | 1,473,591 | 1,950,938 | ||||||
| Prepaid assets | 24,333,851 | 13,977,215 | ||||||
| Other current assets | 364,113 | 3,355,995 | ||||||
| Total current assets | 202,439,221 | 308,412,717 | ||||||
| Non-current Assets: | ||||||||
| Right-of-use Assets | — | 6,644,312 | ||||||
| Property and equipment, net | 977,918 | 1,439,108 | ||||||
| Other assets | 116,421 | 207,873 | ||||||
| Total assets | $ | 203,533,559 | $ | 316,704,010 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 10,254,938 | $ | 13,811,730 | ||||
| Accrued expenses | 5,103,002 | 20,229,694 | ||||||
| Current portion of long-term debt | 1,666,667 | - | ||||||
| Current portion of right of use liabilities | — | 1,549,649 | ||||||
| Total current liabilities | 17,024,607 | 35,591,073 | ||||||
| Long-term liabilities: | ||||||||
| Development derivative liability | — | 109,840,000 | ||||||
| Term loan facility | 18,722,321 | - | ||||||
| Promissory note | 6,655,193 | 6,693,408 | ||||||
| Right-of-use liabilities | — | 5,243,491 | ||||||
| Other liabilities | 158,783 | 250,000 | ||||||
| Total liabilities | 42,560,904 | 157,617,972 | ||||||
| Stockholders' equity: | ||||||||
| Preferred stock, $0.0001 par value; 10,000,000 shares authorized, and zero shares issued and outstanding at December 31, 2018 and March 31, 2019 | — | — | ||||||
| Common stock, $0.0001 par value; 200,000,000 shares authorized at December 31, 2018 and March 31, 2019 and 56,279,307 shares issued and outstanding at December 31, 2018 and 63,218,476 shares issued and outstanding at March 31, 2019 | 5,628 | 6,367 | ||||||
| Additional paid in capital | 437,855,681 | 557,632,026 | ||||||
| Accumulated other comprehensive loss | (122,807 | ) | (121,977 | ) | ||||
| Accumulated deficit | (276,765,847 | ) | (398,430,378 | ) | ||||
| Total stockholders' equity | 160,972,655 | 159,086,038 | ||||||
| Total liabilities and stockholders' equity | $ | 203,533,559 | $ | 316,704,010 | ||||
| APELLIS PHARMACEUTICALS, INC. | ||||||||||||||||
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | ||||||||||||||||
| (Unaudited) | ||||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2018 | 2019 | 2018 | 2019 | |||||||||||||
| (Unaudited) | ||||||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 27,537,619 | $ | 50,698,427 | $ | 44,940,509 | $ | 91,178,326 | ||||||||
| General and administrative | 5,947,823 | 12,778,011 | 9,983,079 | 20,948,682 | ||||||||||||
| Operating loss | (33,485,442 | ) | (63,476,438 | ) | (54,923,588 | ) | (112,127,008 | ) | ||||||||
| Loss on extinguishment of debt | — | — | — | (1,208,132 | ) | |||||||||||
| Loss from remeasurement of development derivative liability | — | (9,104,000 | ) | — | (9,840,000 | ) | ||||||||||
| Interest expense | (603,656 | ) | (159,497 | ) | (1,270,743 | ) | (753,002 | ) | ||||||||
| Interest income | 787,665 | 1,421,485 | 1,188,066 | 2,288,503 | ||||||||||||
| Other (expense)/income, net | (32,867 | ) | 228,286 | (64,341 | ) | (24,892 | ) | |||||||||
| Net loss | $ | (33,334,300 | ) | $ | (71,090,164 | ) | (55,070,606 | ) | (121,664,531 | ) | ||||||
| Other comprehensive gain: | ||||||||||||||||
| Foreign currency gain | — | (1,332 | ) | — | 830 | |||||||||||
| Total other comprehensive gain | — | (1,332 | ) | — | 830 | |||||||||||
| Comprehensive loss, net of tax | $ | (33,334,300 | ) | $ | (71,091,496 | ) | $ | (55,070,606 | ) | $ | (121,663,701 | ) | ||||
| Net loss per common share, basic and diluted | $ | (0.61 | ) | $ | (1.12 | ) | $ | (1.05 | ) | $ | (2.01 | ) | ||||
| Weighted-average number of common shares used in net loss per common share, basic and diluted | 54,691,833 | 63,263,901 | 52,534,806 | 60,580,646 | ||||||||||||
Media Contact: Ryan Wade 781.209.6460 x8148 (office) 781.801.5206 (mobile) Investor Contact: Alex Kane 212.301.7218 (office) 929.400.2691 (mobile)
