Release Details

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 $176.3 Million at Year-End

WALTHAM Mass. and CRESTWOOD, Ky., Feb. 26, 2019 (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 fourth quarter and full year 2018 financial results and business highlights.

“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 Cedric Francois, CEO and co-founder of Apellis. “We anticipate full enrollment in the Phase 3 PEGASUS Paroxysmal Nocturnal Hemoglobinuria (PNH) trial by the end of the second quarter and continue to build out our commercial capabilities prior to reporting top-line data in the PEGASUS trial in the fourth quarter of this year. In the second quarter of 2019, we look forward to sharing additional data from our clinical trials of APL-2 in patients with cold agglutinin disease (CAD) and warm antibody autoimmune anemia (wAIHA), and in the second half of this year we will present updates on the exploratory treatment of APL-2 in glomerular diseases with complement involvement.”

Business Highlights and Upcoming Milestones:

APL-2 in GA

• In 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.
• In 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.
• In 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.
• In February 2019, Apellis announced that the FDA granted 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).
• In 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.
• In 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.

Other

• 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 Townsend as 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 Europe at EUSA Pharma as General Manager of the UK, Ireland and Nordic Region and as the Head of Commercial. In January 2019, Apellis announced the appointment of Thomas Lackner as 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, Austria and Switzerland.
• In December 2018, Apellis announced that the FDA granted orphan drug designation to APL-2 for the treatment of C3 glomerulopathy (C3G).

            
Fourth Quarter and Full Year 2018 Financial Results:

As of December 31, 2018, Apellis had $176.3 million in cash and cash equivalents, compared to $175.6 million as of December 31, 2017.

Apellis reported a net loss of $36.5 million for the fourth quarter of 2018, compared to a net loss of $18.3 million for the fourth quarter of 2017. For the full year of 2018, Apellis reported a net loss of $127.5 million, compared to a net loss of $51.0 million for the full year of 2017.

Research and development expenses were $30.8 million in the fourth quarter of 2018, compared to $13.1 million for the same period in 2017. For the full year of 2018, research and development expenses were $105.3 million, compared to $40.3 million for the full year of 2017. The increase was primarily due to an increase of $39.7 million in clinical trial costs associated with the preparation for and commencement of our Phase 3 clinical trials, an increase of $14.6 million in manufacturing expenses in connection with the supply for our Phase 3 clinical trials of APL-2, an increase of $9.3 million in compensation and related personnel costs primarily due to increased headcount in 2018, an increase of $1.1 million related to preclinical study expenses, and an increase of $0.3 million in research and development supporting activities, offset by a slight decrease of $0.1 million in device development expenses.

General and administrative expenses were $6.4 million in the fourth quarter of 2018, compared to $4.9 million for the same period in 2017. For the full year of 2018, general and administrative expenses were $22.6 million, compared to $10.5 million for the full year of 2017. The increase was primarily due to an increase in employee-related costs of $5.9 million due to the hiring of additional personnel, an increase in professional and consulting fees of $3.2 million, an increase in license agreement costs of $1.0 million, an increase in insurance costs of $0.8 million, an increase of $0.6 million in information technology expenses, and an increase in office, travel and related costs of $0.7 million. The increased employee related costs of $5.9 million consisted of $3.6 million related to an increase in salaries and benefits primarily due to the hiring of additional members of our management team, $1.4 million related to stock option expense associated with the grants of stock options to employees and $0.9 million in recruitment expense. The increased professional and consulting fees of $3.2 million primarily consisted of an increase of $1.0 million in legal fees, an increase of $0.9 million in accounting fees, an increase of $0.9 million in public company costs and an increase in consulting fees of $0.4 million.

About APL-2
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.

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 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 United States Food and Drug Administration 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’ Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 26, 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 STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
	Three Months Ended December 31,								Year Ended December 31,
	2017				2018				2017				2018
Operating expenses:
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
Operating loss		(17,992,814	)			(37,196,592	)			(50,767,029	)			(127,924,760	)
Gain (loss) from remeasurement of fair value of warrants		(153,692	)			91,217				(153,692	)			85,509
Interest expense		(374,749	)			(624,365	)			(374,749	)			(2,512,956	)
Interest income		245,021				873,355				277,834				2,960,771
Other income (expense), net		20,338				396,910				11,542				(110,758	)
Net loss		(18,255,896	)			(36,459,475	)			(51,006,094	)			(127,502,194	)
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
		December 31,
		2017				2018
Assets
Current assets:
Cash and cash equivalents		$	175,643,529			$	176,267,666
Refundable research and development credit			1,297,361				1,473,591
Prepaid assets			5,059,593				24,333,851
Other current assets			14,823				364,113
Total current assets			182,015,306				202,439,221
Other assets			116,150				1,094,338
Total assets		$	182,131,456			$	203,533,559
Liabilities and Stockholders' Equity
Current liabilities:
Accounts payable		$	3,663,253			$	10,254,938
Accrued expenses			2,890,705				5,103,002
Current portion of long-term debt			-				1,666,667
Total current liabilities			6,553,958				17,024,607
Long-term liabilities:
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
Total liabilities			33,188,596				42,560,904
Stockholders' equity:
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	)
Accumulated deficit			(149,263,653	)			(276,765,847	)
Total stockholders' equity			148,942,860				160,972,655
Total liabilities and stockholders' equity		$	182,131,456			$	203,533,559

Media Contact:
Tully Nicholas
tnicholas@denterlein.com
617.482.0042  (office)
860.490.0218  (mobile)

Investor Contact:
Alex Kane
akane@w2ogroup.com
212.301.7218  (office)
929.400.2691  (mobile)

 

Source: Apellis Pharmaceuticals, Inc.