Apellis Pharmaceuticals Reports Fourth Quarter and Full Year 2018 Financial Results
February 26, 2019
Phase 3 Geographic Atrophy Program Expected to Restart By or In Q2 2019
Phase 3 Trial of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria Expected to be Fully Enrolled by End of Q2 2019
Cash Position of
“Following our first clinical challenge as a public company, the response and execution from the Apellis team has been extraordinary. We expect to restart the Phase 3 Geographic Atrophy (GA) program during the next four months and continue to expect that we will be fully enrolled in both trials in the first quarter of 2020,” said
Business Highlights and Upcoming Milestones:
APL-2 in GA
December 2018, Apellis provided an update on the status of its Phase 3 program for APL-2 in patients with GA. Apellis expects enrollment of its two Phase 3 GA trials (DERBY & OAKS) will restart during the next four months and continues to expect that both trials will be fully enrolled by the end of Q1 2020. Apellis will provide an update on the status of its Phase 3 program for APL-2 in patients with GA during the next month.
October 2018, Apellis disclosed that the Company voluntarily implemented a pause in dosing in the DERBY and OAKS Phase 3 trials due to observed cases of non-infectious inflammation in patients treated from a single manufacturing lot of APL-2 intravitreal drug product.
APL-2 in Hematologic Diseases
- Apellis continues to expect that the Phase 3 PEGASUS trial assessing the safety and efficacy of APL-2 in patients with PNH compared to eculizumab (Soliris™) will be fully enrolled by the end of Q2 2019. Top-line data from the PEGASUS trial is expected in Q4 2019.
February 2019, Apellis announced that the U.S. Food & Drug Administration( FDA) granted fast-track designation to APL-2 for the treatment of patients with PNH.
February 2019, Apellis announced that the FDAgranted orphan drug designation to APL-2 for the treatment of patients with autoimmune hemolytic anemia (AIHA), which refers to both cold agglutinin disease (CAD) and warm antibody autoimmune anemia (wAIHA).
December 2018, Apellis presented interim data from its Phase 1b PADDOCK study of APL-2 in treatment-naïve patients with PNH at the 60th Annual Meeting of the American Society of Hematology.
December 2018, Apellis presented interim data from its Phase 2 PLAUDIT study of APL-2 in patients with AIHA, including CAD and wAIHA, at the 60th Annual Meeting of the American Society of Hematology.
- Apellis recently announced that it has expanded its management team to support the buildout of its commercial infrastructure. In
November 2018, Apellis announced the appointment of Adam Townsendas Chief Commercial Officer. Adam previously worked at Biogen with progressive roles in commercial and corporate development and prior to Biogen, held numerous leadership positions in Europeat EUSA Pharma as General Manager of the UK, Irelandand Nordic Region and as the Head of Commercial. In January 2019, Apellis announced the appointment of Thomas Lackneras Senior Vice President, Head of Europe. Prior to Apellis, Thomas developed the global commercial strategy and launch organization for Prothena Biosciences and previously was Biogen’s Vice President and Managing Director of Germany, Austriaand Switzerland.
December 2018, Apellis announced that the FDAgranted orphan drug designation to APL-2 for the treatment of C3 glomerulopathy (C3G).
Fourth Quarter and Full Year 2018 Financial Results:
Apellis reported a net loss of
Research and development expenses were
General and administrative expenses were
APL-2 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). To date, APL-2 has generally been well-tolerated. No significant infections have been observed in trials involving the systemic administration of APL-2, including the trials in PNH, AIHA or other trials.
About the DERBY and OAKS Trials
The DERBY and OAKS trials are 600-patient prospective, international, multicenter, randomized, double-masked, sham-injection controlled Phase 3 studies assessing the efficacy and safety of multiple intravitreal (IVT) injections of APL-2 in patients with Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD). For more information, please visit https://gastudy.com/.
About APL-2 in Hematologic Diseases
Apellis is currently evaluating APL-2 in PEGASUS, a Phase 3 trial to evaluate the efficacy and safety of APL-2 in patients with PNH as well as in two Phase 1b trials (PHAROAH and PADDOCK) for systemic administration. Previously reported interim data from these 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 warm autoimmune hemolytic anemia (wAIHA) or cold agglutinin disease (CAD). In this trial to date, APL-2 has demonstrated improvements in hemoglobin, reticulocytes, bilirubin and lactate dehydrogenase. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.
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 dosing in the Phase 3 GA program will resume when anticipated; 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 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
|APELLIS PHARMACEUTICALS, INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS|
|Three Months Ended December 31,||Year Ended December 31,|
|Research and development||$||13,132,853||$||30,805,611||$||40,303,878||$||105,285,576|
|General and administrative||4,859,961||6,390,981||10,463,151||22,639,184|
|Gain (loss) from remeasurement of fair value of warrants||(153,692||)||91,217||(153,692||)||85,509|
|Other income (expense), net||20,338||396,910||11,542||(110,758||)|
|Other comprehensive income (loss):|
|Foreign currency loss||—||(462,748||)||—||(122,807||)|
|Total other comprehensive loss||—||(462,748||)||—||(122,807||)|
|Comprehensive loss, net of tax||$||(18,255,896||)||$||(36,922,223||)||$||(51,006,094||)||$||(127,625,001||)|
|Net loss per common share, basic and diluted||$||(0.61||)||$||(0.65||)||$||(3.68||)||$||(2.34||)|
|Weighted-average number of common shares used in net|
|loss per common share, basic and diluted||30,007,513||56,254,314||13,870,949||54,396,483|
|APELLIS PHARMACEUTICALS, INC.|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|Cash and cash equivalents||$||175,643,529||$||176,267,666|
|Refundable research and development credit||1,297,361||1,473,591|
|Other current assets||14,823||364,113|
|Total current assets||182,015,306||202,439,221|
|Liabilities and Stockholders' Equity|
|Current portion of long-term debt||-||1,666,667|
|Total current liabilities||6,553,958||17,024,607|
|Term loan facility||19,806,944||18,722,321|
|Promissory note - related party||6,583,402||6,655,193|
|Common stock warrant liability||244,292||158,783|
|Preferred stock, $0.0001 par value; 10,000,000 shares authorized, and zero|
|shares issued and outstanding at December 31, 2017 and 2018||-||-|
|Common stock, $0.0001 par value; 200,000,000 shares authorized and|
|50,334,152 shares issued and outstanding at December 31, 2017 and|
|200,000,000 shares authorized and 56,279,307 shares issued and|
|outstanding at December 31, 2018||5,033||5,628|
|Additional paid in capital||298,201,480||437,855,681|
|Accumulated other comprehensive income||-||(122,807||)|
|Total stockholders' equity||148,942,860||160,972,655|
|Total liabilities and stockholders' equity||$||182,131,456||$||203,533,559|
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Source: Apellis Pharmaceuticals, Inc.