- Commenced Phase 3 Trial of APL-2 in Paroxysmal Nocturnal Hemoglobinuria (PNH); APL-2 in Geographic Atrophy (GA) Remains On Track to Advance into Phase 3 in 2H18 -

- Cash Position of $253.8 Million at Quarter-End -

CRESTWOOD, Ky. and CAMBRIDGE, Mass., July 31, 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 its second quarter 2018 financial results and business highlights.

“The second quarter of 2018 was marked by significant clinical progress of APL-2, particularly for the potential treatment of PNH,” said Cedric Francois, MD, PhD, founder and chief executive officer of Apellis. “At our R&D Day, we announced the earlier than anticipated enrollment of the first patient in the Phase 3 PEGASUS trial of APL-2 head-to-head with Soliris, and additional patient data from the ongoing PADDOCK and PHAROAH Phase 1b trials of APL-2 in patients with PNH. We also reported initial data from the Phase 2 proof-of-concept (POC) monotherapy trial of APL-2 in autoimmune hemolytic anemia (AIHA). In the second half of 2018, we plan to continue to push forward with the enrollment of our ongoing Phase 3 trial of APL-2 in PNH, to commence our Phase 3 program of APL-2 in GA, and to present additional data from the Phase 2 POC trial of APL-2 in AIHA and initial data from the Phase 2 POC trial in complement-dependent nephropathies.”

Business Highlights and Upcoming Milestones:

APL-2 in Geographic Atrophy

  • The Phase 3 program consisting of two trials of APL-2 in GA remains on track to begin in the second half of 2018.

APL-2 in PNH

  • Enrolled the first patient in the PEGASUS Phase 3 trial of APL-2 in PNH in June 2018.
  • Provided clinical update from the ongoing Phase 1b PHAROAH trial evaluating APL-2 in PNH in June 2018. The PHAROAH trial interim results showed that for the four patients remaining in the study as of the end of June, no transfusions were needed while receiving co-treatment with Soliris and APL-2, and patients maintained hematological benefits following Soliris dose reductions and weaning.
  • Provided clinical update from the ongoing Phase 1b PADDOCK trial evaluating APL-2 in PNH in June 2018 and April 2018. The PADDOCK trial interim results showed that the 13 patients on APL-2 that had not previously been treated with Soliris had improved hemoglobin, bilirubin, reticulocyte and LDH levels on average.

APL-2 In Other Indications

  • Reported initial data from the Phase 2 POC trial of APL-2 in AIHA in June 2018.
  • The data from the Phase 2 POC monotherapy trial of APL-2 in four types of complement-dependent nephropathies (IgA nephropathy, C3 glomerulonephropathy, primary membranous nephropathy and lupus nephritis) are expected in the second half of 2018.

Corporate Highlights

  • Completed a public offering of 5.5 million shares of common stock at $25.50 per share in April 2018, raising net proceeds of $131.3 million, after deducting underwriting discounts, commissions and offering expenses.
  • Strengthened the Company’s senior management team with the appointment of Lukas Scheibler, PhD, as Executive Vice President, Research and Translational Medicine in April 2018. Dr. Scheibler was most recently the head of R&D at Acucela and previously worked at Novartis for 13 years, focused on research & development, business development and clinical development.
  • Hosted the Company’s first R&D Day in June 2018, which included detailed presentations on APL-2 in GA, PNH, AIHA and CDN, as well as key opinion leader presentations and patient commentary on GA and PNH.

Second Quarter 2018 Financial Results:

As of June 30, 2018, Apellis had $253.8 million in cash and cash equivalents, compared to $152.9 million as of March 31, 2018. This includes $131.3 million in net proceeds raised in a public offering of 5.5 million shares of common stock at $25.50 per share in April 2018.

Apellis reported a net loss of $33.3 million for the second quarter of 2018, compared to a net loss of $12.1 million for the second quarter of 2017.

Research and development expenses were $27.5 million in the second quarter of 2018, compared to $10.4 million for the same period in 2017. The increase was primarily due to an increase of $12.1 million in clinical trial costs, an increase of $2.4 million in employee related costs primarily due to the hiring of additional personnel, an increase of $1.9 million in manufacturing expenses, an increase of $0.8 million related to research and development supporting activities and an increase of $0.2 million in pre-clinical study expenses, offset by a decrease of $0.3 million in device development expenses.

General and administrative expenses were $5.9 million in the second quarter of 2018, compared to $1.7 million in the second quarter of 2017. The increase was primarily due to a an increase in employee related costs of $1.6 million, an increase of $0.4 million in director stock option compensation, an increase in office, travel and related costs of $0.5 million, an increase in professional and consulting fees of $1.1 million, an increase in license agreement costs of $0.4 million, and an increase of $0.2 million in insurance costs.

About the PHAROAH Trial
PHAROAH is an ongoing open label Phase 1b safety and efficacy study of 270 mg of APL-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 PADDOCK Trial
PADDOCK is an ongoing open-label Phase 1b safety and efficacy study of 270 mg of APL-2 administered daily by subcutaneous injection to patients with PNH who have never received eculizumab. The PADDOCK study was initiated in December 2015 and is being conducted at multiple clinical sites outside of the United States.

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 http://www.apellis.com.

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 results obtained in preclinical studies and clinical trials, such as the results referenced in this release, will be indicative of results that will be generated in future clinical trials; whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; 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 submission; whether APL-2 will receive approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies; whether, if Apellis’ products receive approval, they will be successfully distributed and marketed; whether Apellis’ cash resources will be sufficient to fund the company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; 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, 2018, 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.  
CONSOLIDATED BALANCE SHEETS  
(Unaudited)  
   December 31, June 30,
    2017   2018 
Assets     
Current assets:     
Cash and cash equivalents  $  175,643,529  $  253,839,540 
Refundable research and development credit     1,297,361     1,996,996 
Prepaid assets     5,059,593     10,857,287 
Other current assets     14,823     7,695 
 Total current assets     182,015,306     266,701,518 
Other assets      116,150     116,150 
Total assets   $  182,131,456  $  266,817,668 
Liabilities and Stockholders' Equity     
Current liabilities:     
Accounts payable  $  3,663,253  $  5,133,017 
Accrued expenses     2,890,705     5,121,178 
 Total current liabilities     6,553,958     10,254,195 
Long-term liabilities:     
Term loan facility     19,806,944     20,143,217 
Promissory note - related party     6,583,402     6,618,150 
Common stock warrant liability     244,292     250,000 
Total liabilities     33,188,596     37,265,562 
Stockholders' equity:     
Preferred stock, $0.0001 par value; 10,000,000 shares authorized, and 
  zero shares issued and outstanding at December 31, 2017 and June 30,
  2018
     -     - 
Common stock, $0.0001 par value; 200,000,000 shares authorized
  at December 31, 2017 and June 30, 2018 and 50,334,152 shares
  issued and outstanding at December 31, 2017 and 56,154,438
  shares issued and outstanding at June 30, 2018
     5,033     5,615 
Additional paid in capital     298,201,480     433,880,750 
Accumulated deficit     (149,263,653)    (204,334,259)
 Total stockholders' equity     148,942,860     229,552,106 
Total liabilities and stockholders' equity   $  182,131,456  $  266,817,668 
      

 

APELLIS PHARMACEUTICALS, INC.   
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS   
(Unaudited)    
     
 Three Months Ended Six Months Ended
 June 30, June 30,
  2017   2018   2017   2018 
Operating expenses:       
Research and development$  10,422,059  $  27,537,619  $  17,653,143  $  44,940,509 
General and administrative   1,710,344     5,947,823     3,531,753     9,983,079 
Operating loss    (12,132,403)    (33,485,442)    (21,184,896)    (54,923,588)
Interest income (expense), net    5,854     184,009     15,966     (82,677)
Other income (expense), net   397     (32,867)    (7,995)    (64,341)
Net loss and comprehensive loss $  (12,126,152) $  (33,334,300) $  (21,176,925) $  (55,070,606)
Net loss per common share, basic and diluted $  (1.44) $  (0.61) $  (2.51) $  (1.05)
Weighted-average number of common shares used in net        
loss per common share, basic and diluted   8,428,366     54,691,833     8,428,366     52,534,806