Apellis Pharmaceuticals Reports Second Quarter 2022 Financial Results
August 8, 2022
- Received U.S. FDA filing acceptance of NDA with Priority Review designation for intravitreal pegcetacoplan for treatment of geographic atrophy; PDUFA target action date of November 26, 2022
- Achieved $15.7 million in Q2 2022 EMPAVELI® (pegcetacoplan) U.S. net product revenues
- Dosed first patient in Phase 3 VALIANT study in IC-MPGN and C3G
- Cash and investments of $852.8 million as of June 30, 2022; expected cash runway into Q1 2024
- Conference call scheduled today at 4:30 p.m. ET
WALTHAM, Mass., Aug. 08, 2022 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in complement, today announced its second quarter 2022 financial results and business highlights.
“2022 is a transformational year for Apellis, and we have made great progress across the company in the first half. Starting with geographic atrophy, our NDA with intravitreal pegcetacoplan was recently accepted with priority review designation, putting us on track for an approval decision in November. This is our second NDA acceptance in less than two years, a monumental accomplishment for the team,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. “Geographic atrophy is a devastating disease that leads to irreversible blindness and, if approved, pegcetacoplan will be the first and only treatment available. We continue to work closely with the FDA to bring this treatment to patients as quickly as possible.”
Dr. Francois continued, “Additionally, we passed our first full year on the U.S. market with EMPAVELI for PNH and it is continuing to show impressive commercial momentum. Beyond PNH, we are advancing the broad platform potential of EMPAVELI with four late-stage clinical programs now underway, as well as our research programs with new molecules that we continue to advance towards IND submissions.”
Second Quarter 2022 Business Highlights and Upcoming Milestones:
Paroxysmal Nocturnal Hemoglobinuria (PNH) Commercial Progress
- Apellis recorded $15.7 million in EMPAVELI® (pegcetacoplan) U.S. net product revenues.
- Apellis presented new data at the European Hematology Association (EHA) Congress reinforcing the robust clinical, safety and health-related quality of life outcomes for PNH patients treated with EMPAVELI.
- Apellis was recognized as a 2022 Industry Innovation Award honoree for EMPAVELI in PNH by the National Organization for Rare Disorders (NORD) at the Rare Impact Award Ceremony
- Sobi submitted and received acceptance for a Japanese regulatory submission for a potential indication in PNH.
Rare Disease R&D Highlights
- Immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G): Apellis dosed the first patient in the Phase 3 VALIANT study of systemic pegcetacoplan for IC-MPGN and C3G in the second quarter of 2022.
- Apellis was granted Orphan Drug Designation (ODD) by the U.S. FDA for pegcetacoplan for the treatment for IC-MPGN. Apellis was previously granted ODD for pegcetacoplan for the treatment of C3G in 2018.
- Cold agglutinin disease (CAD): Sobi now expects to dose the first patient in the Phase 3 study of systemic pegcetacoplan for CAD in the second half of 2022.
- Amyotrophic lateral sclerosis (ALS): Apellis completed enrollment in its ongoing and potentially registrational Phase 2 MERIDIAN study with systemic pegcetacoplan for ALS in the first quarter of 2022. Top-line results are expected in mid-2023.
- Hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA): Sobi continues to enroll patients in its Phase 2 study evaluating the efficacy and safety of systemic pegcetacoplan in patients with HSCT-TMA.
- EMPAVELI + small interfering RNA (siRNA): Apellis is studying the combination of EMPAVELI and an siRNA, which may offer the potential to reduce the treatment frequency of EMPAVELI. Apellis expects to submit an Investigational New Drug (IND) application for its siRNA in the first half of 2023.
- Complement + gene therapy: Apellis is evaluating the role of C3 inhibition as an approach to enhance AAV-delivered gene therapies, with the potential to increase safety and tolerability, allow redosing, decrease the dose needed, and dose patients with pre-existing antibodies.
- In collaboration with Spark Therapeutics, Inc. and Apellis, in vitro data combining APL-9 with AAV administration was presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in May 2022.
Ophthalmology R&D Highlights
- Geographic atrophy (GA) secondary to age-related macular degeneration (AMD):
- In July, the FDA accepted and granted Priority Review designation for the intravitreal pegcetacoplan New Drug Application (NDA). The Prescription Drug User Fee Act (PDUFA) target action date is November 26, 2022. The FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application.
- Apellis expects to report top-line 24-month results from its Phase 3 DERBY & OAKS studies in September 2022.
- Apellis plans to submit a Marketing Authorization Application (MAA) to the European Medicines Agency by the end of 2022.
- Additional analyses from the 18-month DERBY and OAKS data were presented at multiple medical meetings, including the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting (May 2022), the Macula Society Annual Meeting (June 2022), and the American Society of Retina Specialists Annual Meeting (July 2022).
- APL-2006: Apellis expects to submit an IND for APL-2006, a bispecific C3 and VEGF inhibitor, and next generation wet AMD therapy, in the first half of 2023.
Neurology R&D Highlights
- APL-1030: Apellis continues to advance pre-clinical studies with APL-1030, a first-in-class, brain-active C3 inhibitor for neurodegenerative diseases.
Second Quarter 2022 Financial Results:
Cash. As of June 30, 2022, Apellis had $852.8 million in cash, cash equivalents, and short-term marketable securities, compared to $599.0 million in cash, cash equivalents, and short-term marketable securities as of June 30, 2021.
Total Revenue. Total revenue was $16.3 million for the second quarter of 2022, which consisted of $15.7 million in net product revenue from sales of EMPAVELI and $0.6 million in revenue associated with the Sobi collaboration.
Research and Development (R&D) Expenses.
- R&D expenses were $101.7 million for the second quarter of 2022, compared to $145.9 million for the same period in 2021.
- The decrease in R&D expenses for the second quarter ended June 30, 2022 was primarily attributable to the $50.0 million cost of research collaboration associated with the Beam arrangement recorded in the second quarter of 2021, combined with a decrease in contract manufacturing expenses, clinical trial costs, research and innovation costs and pre-clinical study expenses. These decreases were offset by an increase in personnel related costs due to having more employees in the three months ended June 30, 2022, decreases in contra research and development expense related to the Sobi transaction, and an increase in other research and development supporting activities primarily driven by regulatory, quality and medical affairs expenses.
General and Administrative (G&A) Expenses.
- G&A expenses were $63.2 million for the second quarter of 2022, compared to $49.0 million for the same period in 2021.
- The increase in G&A expenses for the second quarter ended June 30, 2022 was primarily attributable to increases in employee-related costs, professional and consulting fees and general commercial preparation activities.
Net Loss (Income). Apellis reported a net loss of $156.0 million in the second quarter 2022, compared to a net loss of $219.2 million for the same period in 2021.
Convertible Notes. As of June 30, 2022, the aggregate principal balance of the 3.500% Convertible Senior Notes (Notes) due 2026, net of unamortized debt issuance costs, was $189.3 million. On July 27, 2022, Apellis entered into an agreement with certain holders of its Notes to exchange approximately $75.6 million of the Notes for common stock. On July 28, 2022, Apellis entered into additional agreements to exchange approximately $22.5 million of the Notes for common stock. These exchange transactions closed on August 1 and August 5, 2022, respectively.
Conference Call and Webcast
Apellis will host a conference call and webcast to discuss its second quarter 2022 financial results and business highlights today, August 8, 2022, at 4:30 p.m. ET. To access the live call by phone, please pre-register for the call here. A live audio webcast of the event and accompanying slides may also be accessed through the “Events and Presentations” page of the “Investors and Media” section of the company’s website. A replay of the webcast will be available for 30 days following the event.
About EMPAVELI®/Aspaveli® (pegcetacoplan)
EMPAVELI®/Aspaveli® (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. EMPAVELI is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States, Australia, and Saudi Arabia, and Aspaveli, which is the European trade name for pegcetacoplan, is approved in the European Union and Great Britain. The therapy is also under investigation for several other rare diseases across hematology, nephrology, and neurology.
U.S. Important Safety Information for EMPAVELI
BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA
- Meningococcal infections may occur in patients treated with EMPAVELI and may become rapidly life-threatening or fatal if not recognized and treated early. Use of EMPAVELI may predispose individuals to serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B.
- Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria.
- Vaccinate patients at least 2 weeks prior to administering the first dose of EMPAVELI unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection.
- Vaccination reduces, but does not eliminate, the risk of serious infections. Monitor patients for early signs of serious infections and evaluate immediately if infection is suspected.
- EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the EMPAVELI REMS, prescribers must enroll in the program.
- Hypersensitivity to pegcetacoplan or to any of the excipients
- Not currently vaccinated against certain encapsulated bacteria, unless the risks of delaying EMPAVELI treatment outweigh the risks of developing a bacterial infection with an encapsulated organism
- Unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae
WARNINGS AND PRECAUTIONS
Serious Infections Caused by Encapsulated Bacteria
The use of EMPAVELI may predispose individuals to serious, life-threatening, or fatal infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B (Hib). To reduce the risk of infection, all patients must be vaccinated against these bacteria according to the most current ACIP recommendations for patients with altered immunocompetence associated with complement deficiencies. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI.
For patients without known history of vaccination, administer required vaccines at least 2 weeks prior to receiving the first dose of EMPAVELI. If immediate therapy with EMPAVELI is indicated, administer required vaccine as soon as possible and provide patients with 2 weeks of antibacterial drug prophylaxis.
Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider discontinuation of EMPAVELI in patients who are undergoing treatment for serious infections.
Because of the risk of serious infections, EMPAVELI is available only through a restricted program under a REMS. Under the EMPAVELI REMS, prescribers must enroll in the program and must counsel patients about the risk of serious infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated against encapsulated bacteria. Enrollment and additional information are available by telephone: 1-888-343-7073 or at www.empavelirems.com.
Systemic hypersensitivity reactions (e.g., facial swelling, rash, urticaria) have occurred in patients treated with EMPAVELI. One patient (less than 1% in clinical studies) experienced a serious allergic reaction which resolved after treatment with antihistamines. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved.
Monitoring PNH Manifestations after Discontinuation of EMPAVELI
After discontinuing treatment with EMPAVELI, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH levels along with sudden decrease in PNH clone size or hemoglobin, or reappearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues EMPAVELI for at least 8 weeks to detect hemolysis and other reactions. If hemolysis, including elevated LDH, occurs after discontinuation of EMPAVELI, consider restarting treatment with EMPAVELI.
Interference with Laboratory Tests
There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels.
The most common adverse reactions (incidence ≥10% of patients) with EMPAVELI vs. eculizumab were injection-site reactions (39% v. 5%), infections (29% v. 26%), diarrhea (22% v. 3%), abdominal pain (20% v. 10%), respiratory tract infection (15% v. 13%), viral infection (12% v. 8%), and fatigue (12% v. 23%).
USE IN SPECIFIC POPULATIONS
Females of Reproductive Potential
EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose.
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in complement, we ushered in the first new class of complement medicine in 15 years with the approval of the first and only targeted C3 therapy. We are advancing this science to continually develop transformative medicines for people living with rare, retinal, and neurological diseases. For more information, please visit http://apellis.com or follow us on Twitter and LinkedIn.
Apellis Forward-Looking Statement
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. 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 will be indicative of results that will be generated in future clinical trials; whether the results of the FILLY, DERBY, and OAKS trials are sufficient to support regulatory submissions; whether a submission for approval of intravitreal pegcetacoplan for GA on the basis of the FILLY, DERBY and OAKS trials will be accepted by the FDA or foreign regulatory agencies; whether intravitreal pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA when expected or at all; 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 pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company’s clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for CAD, C3G, IC-MPGN, HSCT-TMA, ALS or any other indication when expected or at all; 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 28, 2022 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.
[TABLES TO FOLLOW]
|APELLIS PHARMACEUTICALS, INC.|
|CONDENSED CONSOLIDATED BALANCE SHEETS|
|(Amounts in thousands, except per share amounts)|
|June 30||December 31,|
|Cash and cash equivalents||$||604,489||$||640,192|
|Other current assets||28,460||70,677|
|Total current assets||959,969||824,047|
|Property and equipment, net||5,892||6,177|
|Liabilities and Stockholders' Equity|
|Current portion of development liability||15,842||7,584|
|Current portion of right of use liabilities||5,312||4,115|
|Total current liabilities||126,793||131,847|
|Long-term development liability||333,729||345,151|
|Convertible senior notes||189,313||189,024|
|Commitments and contingencies (Note 14)||—||—|
|Preferred stock, $0.0001 par value; 10,000 shares authorized and zero shares |
issued and outstanding at March 31, 2022 and December 31, 2021
|Common stock, $0.0001 par value; 200,000 shares authorized|
|at March 31, 2022 and December 31, 2021; 106,440 shares |
issued and outstanding at March 31, 2022, and 97,524 shares
issued and outstanding at December 31, 2021
|Additional paid-in capital||2,289,201||1,857,430|
|Accumulated other comprehensive loss||(3,194||)||(2,090||)|
|Total stockholders' equity||334,418||198,662|
|Total liabilities and stockholders' equity||$||1,002,498||$||881,765|
|APELLIS PHARMACEUTICALS, INC.|
|CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS|
|(Amounts in thousands, except per share amounts)|
|For the three months ended June 30,||For the six months ended June 30,|
|Product revenue, net||$||15,654||$||623||$||27,763||$||623|
|Licensing and other revenue||668||—||2,940||—|
|Cost of sales||82||—||1,329||—|
|Research and development||101,661||95,943||192,606||179,955|
|Cost of research collaboration||—||50,000||—||50,000|
|General and administrative||63,203||48,967||114,390||89,546|
|Net operating income/(loss)||(148,624||)||(194,287||)||(277,622||)||(318,878||)|
|Loss on conversion of debt||—||—||—||(39,487||)|
|Loss from remeasurement of development derivative liability||—||(21,180||)||—||(38,264||)|
|Other (expense)/income, net||149||(61||)||(140||)||1,483|
|Net loss before taxes||(155,491||)||(219,191||)||(293,218||)||(402,850||)|
|Income tax expense||486||—||1,694||—|
|Other comprehensive (loss)/gain:|
|Unrealized (loss)/gain on marketable securities||(766||)||(35||)||(818||)||44|
|Foreign currency gain/(loss)||(369||)||(174||)||(286||)||(1,756||)|
|Total other comprehensive income/(loss)||(1,135||)||(209||)||(1,104||)||(1,712||)|
|Comprehensive loss, net of tax||$||(157,112||)||$||(219,400||)||$||(296,016||)||$||(404,562||)|
|Net loss per common share, basic and diluted||$||(1.46||)||$||(2.72||)||$||(2.88||)||$||(5.04||)|
|Weighted-average number of common shares used in net |
loss per common share, basic and diluted