APLS Apellis Pharmaceuticals Inc

- Commenced Phase 3 Program of APL-2 in Geographic Atrophy (GA) -

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 third quarter 2018 financial results and business highlights.

“The third quarter of 2018 was very important for Apellis, as we initiated our Phase 3 program in GA and now have two clinical programs in Phase 3 following the initiation of the PEGASUS trial of APL-2 in paroxysmal nocturnal hemoglobinuria (PNH) patients in June,” said Cedric Francois, MD, PhD, founder and chief executive officer of Apellis. “This is a considerable accomplishment as we work towards the goal of treating patients with complement mediated diseases with APL-2 as an approved drug. We remain steadfast in our commitment to patients and voluntarily implemented a temporary pause in dosing in both our Phase 3 GA program and in our Phase 1b/2 Wet AMD trial last month following observations of non-infectious inflammation in patients that we believe resulted from a single manufacturing lot of APL-2. We do not believe the pause will delay the projected enrollment timeline of 18 months in the Phase 3 GA program and in the first half of December we plan to provide an update following confirmatory studies and review by the data safety monitoring board. We also look forward to sharing updates on our hematology programs later this quarter at the 2018 American Society of Hematology (ASH) conference.”

Business Highlights and Upcoming Milestones:

APL-2 in GA

• Initiated patient dosing in the Phase 3 GA program, consisting of two Phase 3 trials (DERBY & OAKS), in September 2018.
• Received Fast Track designation for the treatment of patients with GA from the U.S. Food & Drug Administration (FDA) in July 2018.
• Plan to provide an update on the Phase 3 GA program in the first half of December 2018.

APL-2 in PNH

• Provided interim results from the Phase 1b PHAROAH trial evaluating APL-2 in PNH patients on treatment with eculizumab (Soliris™) in September 2018. All four patients, who were severely anemic and transfusion dependent while on eculizumab, were transitioned to APL-2 monotherapy.
• Plan to provide an update on the ongoing Phase 1b PNH trials at the 2018 ASH conference in December.

APL-2 In Other Indications

• Plan to provide an update on the ongoing Phase 2 monotherapy trial of APL-2 in both warm autoimmune hemolytic anemia (wAIHA) and cold agglutinin disease (CAD) at the 2018 ASH conference in December.
• Expect 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) in 2019. The trial timeline has been updated from a four month data readout to a one year data readout.

Third Quarter 2018 Financial Results:

As of September 30, 2018, Apellis had $220.6 million in cash and cash equivalents, compared to $253.8 million as of June 30, 2018.

Apellis reported a net loss of $36.0 million for the third quarter of 2018, compared to a net loss of $11.6 million for the third quarter of 2017.

Research and development expenses were $29.5 million in the third quarter of 2018, compared to $9.5 million in the third quarter of 2017. The increase in research and development expenses was primarily attributable to an increase of $10.2 million in clinical trial costs associated with the preparation for and commencement of our Phase 3 clinical trials, an increase of $6.8 million in manufacturing expenses in connection with the supply for our Phase 3 clinical trials of APL-2, an increase of $2.5 million in employee related costs primarily due to the hiring of additional personnel, an increase of $0.3 million related to research and development supporting activities and an increase of $0.2 million in preclinical study expenses.

General and administrative expenses were $6.3 million in the third quarter of 2018, compared to $2.1 million in the third quarter of 2017. The increase in general and administrative expenses was primarily attributable to an increase in employee related costs of $2.0 million due to the hiring of additional personnel, an increase in professional and consulting fees of $0.9 million, an increase in license agreement costs of $0.7 million attributable to our achievement of milestones, an increase of $0.4 million in director stock option compensation and an increase of $0.2 million in insurance costs.

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).

About ApellisApellis 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. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.

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 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 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 November 13, 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.
CONDENSED CONSOLIDATED BALANCE SHEETS
		December 31,				September 30,
			2017				2018
Assets						(Unaudited)
Current assets:
Cash and cash equivalents		$	175,643,529			$	220,640,818
Refundable research and development credit			1,297,361				1,364,259
Prepaid assets			5,059,593				16,846,183
Other current assets			14,823				81,532
Total current assets			182,015,306				238,932,792
Other assets			116,150				212,979
Total assets		$	182,131,456			$	239,145,771
Liabilities and Stockholders' Equity
Current liabilities:
Accounts payable		$	3,663,253			$	5,468,800
Accrued expenses			2,890,705				10,565,640
Total current liabilities			6,553,958				16,034,440
Long-term liabilities:
Term loan facility			19,806,944				20,270,032
Promissory note - related party			6,583,402				6,636,449
Common stock warrant liability			244,292				250,000
Total liabilities			33,188,596				43,190,921
Stockholders' equity:
Preferred stock, $0.0001 par value; 10,000,000 shares authorized, and zero   shares issued and outstanding at December 31, 2017 and September 30, 2018			-				-
Common stock, $0.0001 par value; 200,000,000 shares authorized   at December 31, 2017 and September 30, 2018 and 50,334,152 shares   issued and outstanding at December 31, 2017 and 56,242,571   shares issued and outstanding at September 30, 2018			5,033				5,624
Additional paid in capital			298,201,480				435,915,657
Accumulated other comprehensive income			-				339,942
Accumulated deficit			(149,263,653	)			(240,306,373	)
Total stockholders' equity			148,942,860				195,954,850
Total liabilities and stockholders' equity		$	182,131,456			$	239,145,771

 

APELLIS PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(Unaudited)
	Three Months Ended								Nine Months Ended
	September 30,								September 30,
		2017				2018				2017				2018
Operating expenses:
Research and development	$	9,517,882			$	29,539,456			$	27,171,025			$	74,479,965
General and administrative		2,071,437				6,265,125				5,603,190				16,248,203
Operating loss		(11,589,319	)			(35,804,581	)			(32,774,215	)			(90,728,168	)
Unrealized foreign currency loss		—				(426,191	)			—				(426,191	)
Interest income (expense), net		16,847				280,891				32,813				198,214
Other income (expense), net		(801	)			(22,234	)			(8,796	)			(86,575	)
Net loss		(11,573,273	)			(35,972,115	)			(32,750,198	)			(91,042,720	)
Other comprehensive income:
Foreign currency translation		—				339,942				—				339,942
Comprehensive loss, net of tax	$	(11,573,273	)		$	(35,632,173	)		$	(32,750,198	)		$	(90,702,778	)
Net loss per common share, basic and diluted	$	(1.37	)		$	(0.64	)		$	(3.88	)		$	(1.69	)
Weighted-average number of common shares used in net   loss per common share, basic and diluted		8,442,072				56,201,299				8,432,983				53,770,400

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

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