Apellis Pharmaceuticals Reports First Quarter 2019 Business Update and Financial Results
May 8, 2019
Resumed Dosing in Phase 3 Geographic Atrophy Program
Announced Innovative Collaboration & Financing with
Cash Position of
“Apellis made considerable clinical and corporate progress during the first quarter. We resumed dosing in our Geographic Atrophy (GA) program while maintaining our original enrollment timeline guidance, strengthened our capital position and continued enrollment in the Phase 3 PEGASUS trial in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), which we expect to fully enroll by the end of the second quarter of 2019,” said
Business Highlights and Upcoming Milestones:
APL-2 in GA
March 2019, Apellis announced that, with the agreement of the independent safety monitoring committee, it resumed dosing in its two Phase 3 trials for APL-2 in patients with GA (DERBY & OAKS). This followed an in-depth investigation into the cause of non-infectious inflammation in patients treated from a single manufacturing lot of APL-2 intravitreal investigational material, as well as modifications to the manufacturing process in order to eliminate an impurity in the active pharmaceutical ingredient (API) suspected of being the source of the non-infectious inflammation. Following the modifications, no inflammation was observed in ten low-vision patients dosed in a Phase 1b trial with new intravitreal APL-2 drug product. Apellis believes that it has sufficient supply of APL-2 manufactured using the modified process to conduct the entire Phase 3 GA program. The Company continues to expect to have both trials fully enrolled by the end of the first quarter of 2020.
APL-2 in Hematologic Diseases
February 2019, Apellis announced a novel, risk-sharing collaboration to support the development of APL-2 in hematologic indications with SFJ Pharmaceuticals, a global drug development company backed by Blackstone Life Sciences and Abingworth. Under the terms of the agreement, Apellis received a $60 millionupfront payment and up to an additional $60 millionpayable based on Apellis meeting specific, pre-defined clinical milestones that are expected in 2019 and associated with the PNH development program, and subject to Apellis meeting certain capital requirements, with the potential for additional payments subject to mutual agreement, while retaining exclusive worldwide rights to APL-2 in all indications.
February 2019, Apellis announced that the U.S. Food and Drug Administration( FDA) granted Fast Track designation to APL-2, for the treatment of all patients with PNH, superseding the prior Fast Track designation granted in December, 2016 for APL-2 for the subset of PNH patients who continue to experience hemolysis requiring blood transfusions despite receiving therapy with eculizumab.
February 2019, Apellis announced that the FDAgranted orphan drug designation to APL-2, for the treatment of autoimmune hemolytic anemia (AIHA), which includes both cold agglutinin disease (CAD) and warm autoimmune hemolytic anemia (wAIHA).
- Initial clinical testing indicates that the pharmacokinetic (PK) profile of APL-2 administered subcutaneously, 2x weekly at a dose of 1,080 mg, both with the 510k approved pump used in the PEGASUS Phase 3 trial and with the Enable enFuse™ device, is comparable to that of APL-2 administered 1x daily at a dose between 270 mg and 360 mg. Based on the data generated to date, the Company believes that the tolerability profiles are comparable.
- 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 will be fully enrolled by the end of the second quarter of 2019. Top-line data from the PEGASUS trial is expected in the fourth quarter of 2019.
March 2019, Apellis completed a public offering of 6.9 million shares of common stock, which included 900,000 shares issued upon the exercise in full by the underwriters of their option to purchase additional shares at the public offering price, at a public offering price of $17.00per share, raising net proceeds of $109.6 million, after deducting underwriting discounts, commissions and offering expenses.
- Expanded the leadership team and continued to build the commercial team. During the quarter,
Federico Grossiwas appointed to the position of Chief Medical Officer, Lukas Scheiblerwas appointed to the position of Chief Innovation Officer and Ahmad Sadr was appointed to the position of SVP of Technical Operations.
First Quarter 2019 Financial Results:
Apellis reported a net loss of
Research and development expenses were
General and administrative expenses were
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, in patients with PNH who are being treated with eculizumab or who are naïve to complement inhibitor treatment, in patients with autoimmune hemolytic anemia and in patients with C3G and other glomerular diseases.
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 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 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 warm autoimmune hemolytic anemia (wAIHA) or cold agglutinin disease (CAD). In this trial to date, patients treated with APL-2 have experienced 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 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 March 31,|
|Research and development||$||17,402,890||$||40,479,899|
|General and administrative||4,035,255||8,170,671|
|Loss on extinguishment of debt||—||(1,208,132||)|
|Loss on remeasurement of development derivative liability||—||(736,000||)|
|Other income (expense), net||(31,473||)||(253,177||)|
|Other comprehensive income (loss):|
|Foreign currency gain||—||2,162|
|Total other comprehensive loss||—||2,162|
|Comprehensive loss, net of tax||$||(21,736,304||)||$||(50,572,205||)|
|Net loss per common share, basic and diluted||$||(0.43||)||$||(0.87||)|
|Weighted-average number of common shares used in net
loss per common share, basic and diluted
|APELLIS PHARMACEUTICALS, INC.|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|December 31,||March 31,|
|Cash and cash equivalents||$||176,267,666||$||288,246,706|
|Refundable research and development credit||1,473,591||1,755,639|
|Other current assets||364,113||15,570|
|Total current assets||202,439,221||310,527,863|
|Property and equipment, net||977,918||1,041,063|
|Liabilities and Stockholders' Equity|
|Current portion of long-term debt||1,666,667||-|
|Current portion of right of use liabilities||—||1,127,720|
|Total current liabilities||17,024,607||20,386,473|
|Development derivative liability||—||60,736,000|
|Term loan facility||18,722,321||—|
|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, $0.0001 par value; 200,000,000 shares authorized
at December 31, 2018 and March 31, 2019 and 56,279,307 shares
issued and outstanding at December 31, 2018 and 63,218,476 shares
issued and outstanding at March 31, 2019
|Additional paid in capital||437,855,681||552,209,429|
|Accumulated other comprehensive loss||(122,807||)||(120,645||)|
|Total stockholders' equity||160,972,655||224,754,892|
|Total liabilities and stockholders' equity||$||203,533,559||$||318,353,078|
Liza Sullivanlsullivan@denterlein.com 617.482.0042 (office) 917.981.7494 (mobile) Investor Contact: Alex Kaneakane@w2ogroup.com 212.301.7218 (office) 929.400.2691 (mobile)
Source: Apellis Pharmaceuticals, Inc.