Hemoglobinuria

Generated $199.7 million in 2Q 2024 revenues, including $179.1 million in U.S. net product sales $154.6 million for SYFOVRE® (pegcetacoplan injection) $24.5 million for EMPAVELI® (pegcetacoplan) Initiated a re-examination of the marketing authorization application of SYFOVRE with the European Medicines Agency (EMA); expect a final decision in 4Q 2024 Plan to report topline Phase 3 data with systemic pegcetacoplan in C3G / IC-MPGN in August 2024Cash and cash equivalents of $360.1 million as of June 30, 2024; projected revenues and cash expected to be sufficient to fund operations to positive cash flow Management to host conference call today at 8:30 a.m. ET WALTHAM, Mass., Aug. 01, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), today announced its second quarter 2024 financial results and business highlights. “We have continued to execute well and progress our programs over the past six months. SYFOVRE delivered strong growth and is reaching more people living with geographic atrophy, EMPAVELI continues to elevate the standard of care in PNH, and we are now very well positioned financially to support the business for the long term,” said Cedric Francois, M.D., Ph.D., chief executive officer at Apellis. “We are looking forward to a catalyst-driven second half of this year, as we continue to drive strong commercial execution in the U.S, prepare for the topline readout from our Phase 3 VALIANT trial with systemic pegcetacoplan for C3G and IC-MPGN later this month, and seek a final decision from the European regulatory authorities for SYFOVRE in GA in the fourth quarter.” Second Quarter 2024 Business Highlights and Upcoming Milestones Ophthalmology Highlights SYFOVRE for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD): Generated $154.6 million in SYFOVRE U.S. net product revenue in the second quarter 2024.SYFOVRE remains the market leader in GA, delivering more than 84,000 SYFOVRE doses to physician practices in the second quarter (over 79,000 commercial vials and approximately 5,000 samples). More than 330,000 SYFOVRE injections are estimated to have been administered through June 2024, including clinical trials.Effective July 1, 2024, two large national pharmacy benefit managers have made SYFOVRE the preferred product on their commercial formularies. Initiated a re-examination of the marketing authorization application of SYFOVRE with the EMA following a negative opinion from the Committee for Medicinal Products for Human Use (CHMP) received in June 2024. There were multiple dissenting votes to the negative opinion by CHMP members. Additionally, members of the Ad Hoc Expert Group meeting agreed that size of GA lesion is an acceptable primary outcome measure for a trial in GA and that microperimetry is the best available functional measure in GA.Expect a final opinion to be issued by the CHMP in the fourth quarter of 2024. Paroxysmal Nocturnal Hemoglobinuria (PNH) Highlights EMPAVELI for the treatment of PNH: Generated $24.5 million in EMPAVELI U.S. net product revenue in the second quarter of 2024.Continued high patient compliance rates of 97%. R&D Highlights C3 glomerulopathy (C3G) and primary immune complex glomerulonephritis (IC-MPGN): Topline data from the Phase 3 VALIANT study of systemic pegcetacoplan is expected in August 2024. VALIANT is a randomized, placebo-controlled, double-blinded, multi-center study designed to evaluate the safety and efficacy of systemic pegcetacoplan in 124 patients aged 12 and up with either C3G or IC-MPGN, and either pre- or post-kidney transplant.The primary endpoint is the log transformed ratio of urine protein-to-creatinine ratio (uPCR) at week 26 compared to baseline. 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.APL-3007 (small interfering RNA silencing C3): Continue to expect to report topline data from the Phase 1 dose escalation study in 2024. Second Quarter 2024 Financial Results Total Revenue. Total revenue was $199.7 million for the second quarter of 2024, which consisted of $154.6 million of SYFOVRE U.S. net product revenue, $24.5 million of EMPAVELI U.S. net product revenue, and $20.5 million in licensing and other revenue associated with the Sobi collaboration. Total revenue was $95.0 million for the second quarter of 2023, which consisted of $67.3 million of SYFOVRE U.S. net product revenue, $22.3 million in EMPAVELI U.S. net product revenue and $5.3 million in revenue associated with the Sobi collaboration. Cost of Sales. Cost of sales were $23.1 million for the second quarter 2024, compared to $8.4 million for same period in 2023. The increase in cost of sales was primarily driven by higher volume from commercial sales and product provided under our patient assistance programs, an increase in revenue associated with the Sobi collaboration, an increase in royalty expense, and an increase in expenses incurred related to excess or obsolete inventory. R&D Expenses. R&D expenses were $78.0 million for the second quarter of 2024, compared to $95.7 million for the same period in 2023. The decrease in R&D expenses was primarily attributable to a decrease in program specific external costs, non-program specific external costs, and compensation and related personnel costs. Selling, General and Administrative (SG&A) Expenses. SG&A expenses were $128.1 million for the second quarter of 2024, compared to $111.4 million for the same period in 2023. The increase was primarily attributable to increases in personnel related costs, commercial and marketing activities and office costs, which were partially offset by a decrease in professional and consulting fees Net Loss. Apellis reported a net loss of $37.7 million for the second quarter 2024, compared to a net loss of $122.0 million for the same period in 2023. Cash. As of June 30, 2024, Apellis had $360.1 million in cash and cash equivalents, compared to $351.2 million in cash and cash equivalents as of December 31, 2023. In May 2024, Apellis refinanced existing debt with up to $475 million non-dilutive credit facility from Sixth Street.Apellis anticipates its cash, combined with expected product revenues, will be sufficient to fund its projected operating expenses and capital expenditures to positive cash flow. Conference Call and WebcastApellis will host a conference call and webcast to discuss its second quarter 2024 financial results and business highlights today, August 1, 2024, at 8:30 a.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 SYFOVRE® (pegcetacoplan injection)SYFOVRE® (pegcetacoplan injection) is the first-ever approved therapy for geographic atrophy (GA). By targeting C3, SYFOVRE is designed to provide comprehensive control of the complement cascade, part of the body’s immune system. SYFOVRE is approved in the United States for the treatment of GA secondary to age-related macular degeneration. 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. It is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally. The therapy is also under investigation for several other rare diseases across hematology and nephrology. U.S. Important Safety Information for SYFOVRE® (pegcetacoplan injection) CONTRAINDICATIONS SYFOVRE is contraindicated in patients with ocular or periocular infections, and in patients with active intraocular inflammation WARNINGS AND PRECAUTIONS Endophthalmitis and Retinal Detachments Intravitreal injections, including those with SYFOVRE, may be associated with endophthalmitis and retinal detachments. Proper aseptic injection technique must always be used when administering SYFOVRE to minimize the risk of endophthalmitis. Patients should be instructed to report any symptoms suggestive of endophthalmitis or retinal detachment without delay and should be managed appropriately. Retinal Vasculitis and/or Retinal Vascular Occlusion Retinal vasculitis and/or retinal vascular occlusion, typically in the presence of intraocular inflammation, have been reported with the use of SYFOVRE. Cases may occur with the first dose of SYFOVRE and may result in severe vision loss. Discontinue treatment with SYFOVRE in patients who develop these events. Patients should be instructed to report any change in vision without delay. Neovascular AMD In clinical trials, use of SYFOVRE was associated with increased rates of neovascular (wet) AMD or choroidal neovascularization (12% when administered monthly, 7% when administered every other month and 3% in the control group) by Month 24. Patients receiving SYFOVRE should be monitored for signs of neovascular AMD. In case anti-Vascular Endothelial Growth Factor (anti-VEGF) is required, it should be given separately from SYFOVRE administration. Intraocular Inflammation In clinical trials, use of SYFOVRE was associated with episodes of intraocular inflammation including: vitritis, vitreal cells, iridocyclitis, uveitis, anterior chamber cells, iritis, and anterior chamber flare. After inflammation resolves, patients may resume treatment with SYFOVRE. Increased Intraocular Pressure Acute increase in IOP may occur within minutes of any intravitreal injection, including with SYFOVRE. Perfusion of the optic nerve head should be monitored following the injection and managed as needed. ADVERSE REACTIONS Most common adverse reactions (incidence ≥5%) are ocular discomfort, neovascular age-related macular degeneration, vitreous floaters, conjunctival hemorrhage. Please see accompanying full Prescribing Information for more information. U.S. Important Safety Information for EMPAVELI® (pegcetacoplan) BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risks of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor.Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS. CONTRAINDICATIONS Hypersensitivity to pegcetacoplan or to any of the excipientsFor initiation in patients with unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B WARNINGS AND PRECAUTIONS Serious Infections Caused by Encapsulated BacteriaEMPAVELI, a complement inhibitor, increases a patient’s susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis (caused by any serogroup, including non-groupable strains), and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of EMPAVELI treatment is contraindicated in patients with unresolved serious infection caused by encapsulated bacteria. Complete or update vaccination against encapsulated bacteria at least 2 weeks prior to administration of the first dose of EMPAVELI, according to the most current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI. Note that, ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent EMPAVELI therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible. The benefits and risks of treatment with EMPAVELI, as well as the benefits and risks of antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by encapsulated bacteria. Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of EMPAVELI in patients who are undergoing treatment for serious infections. EMPAVELI is available only through a restricted program under a REMS. EMPAVELI REMSEMPAVELI is available only through a restricted program under a REMS called EMPAVELI REMS, because of the risk of serious infections caused by encapsulated bacteria. Notable requirements of the EMPAVELI REMS include the following: Under the EMPAVELI REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risks, signs, and symptoms of serious infections caused by encapsulated bacteria, provide patients with the REMS educational materials, ensure patients are vaccinated against encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, prescribe antibacterial drug prophylaxis if patients’ vaccine status is not up to date and treatment must be started urgently, and provide instructions to always carry the Patient Safety Card both during treatment, as well as for 2 months following last dose of EMPAVELI. Pharmacies that dispense EMPAVELI must be certified in the EMPAVELI REMS and must verify prescribers are certified. Further information is available at www.empavelirems.com or 1-888-343-7073. Infusion-Related ReactionsSystemic 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 EMPAVELIAfter 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 TestsThere 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. ADVERSE REACTIONSMost common adverse reactions in patients with PNH (incidence ≥10%) were injection site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, pain in extremity, hypokalemia, fatigue, viral infection, cough, arthralgia, dizziness, headache, and rash. USE IN SPECIFIC POPULATIONS Females of Reproductive PotentialEMPAVELI 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. Please see full Prescribing Information, including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and Medication Guide. About ApellisApellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across serious retinal, rare, and neurological diseases. For more information, please visit http://apellis.com or follow us on Twitter and LinkedIn. Apellis Forward-Looking StatementStatements 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 regarding the expected timing of clinical data, and the review of the marketing authorization application of SYFOVRE by the EMA. 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 benefit/risk profile of SYFOVRE following the events of retinal vasculitis will impact the Company’s commercialization efforts; whether SYFOVRE will receive approval from foreign regulatory agencies for GA when expected or at all, including the impact of the reported events of retinal vasculitis on the likelihood and timing of such approvals; whether the Company’s clinical trials will be completed when anticipated; whether results obtained in 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 systemic pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for C3G and IC-MPGN or any other indication when expected or at all; the period for which the Company believes that its cash resources will be sufficient to fund its operations; and other factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 27, 2024 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. Media Contact:Tracy Vineismedia@apellis.com617.420.4839 Investor Contact:Meredith Kayameredith.kaya@apellis.com617.599.8178 APELLIS PHARMACEUTICALS, INC.CONDENSED CONSOLIDATED BALANCE SHEETS(Amounts in thousands, except per share amounts) June 30, December 31, 2024 2023 Assets (Unaudited) Current assets: Cash and cash equivalents $360,087 $351,185 Accounts receivable 304,433 206,442 Inventory 152,772 146,362 Prepaid assets 28,971 38,820 Restricted cash 1,339 1,114 Other current assets 12,372 22,408 Total current assets 859,974 766,331 Non-current assets: Right-of-use assets 16,321 16,745 Property and equipment, net 3,836 4,345 Long-term inventory 23,021 — Other assets 1,330 1,309 Total assets $904,482 $788,730 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $38,270 37,516 Accrued expenses 122,781 127,806 Current portion of development liability — 75,830 Current portion of lease liabilities 6,214 6,441 Deferred revenue 1,903 — Total current liabilities 169,168 247,593 Long-term liabilities: Long-term development liability — 239,817 Long-term credit facility 364,025 — Convertible senior notes 93,186 93,033 Lease liabilities 11,085 11,454 Other liabilities 2,691 2,312 Total liabilities 640,155 594,209 Commitments and contingencies (Note 13) Stockholders' equity: Preferred stock, $0.0001 par value; 10,000 shares authorized and zero shares issued and outstanding at June 30, 2024 and December 31, 2023 — — Common stock, $0.0001 par value; 200,000 shares authorized at June 30, 2024 and December 31, 2023; 121,687 shares issued and outstanding at June 30, 2024, and 119,556 shares issued and outstanding at December 31, 2023 12 12 Additional paid-in capital 3,209,245 3,035,539 Accumulated other comprehensive loss (3,362) (3,542)Accumulated deficit (2,941,568) (2,837,488)Total stockholders' equity 264,327 194,521 Total liabilities and stockholders' equity $904,482 $788,730 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, 2024 2023 2024 2023 (Unaudited) (Unaudited)Revenue: Product revenue, net $179,136 $89,645 $342,211 $128,444 Licensing and other revenue 20,549 5,324 29,798 11,370 Total revenue: 199,685 94,969 372,009 139,814 Operating expenses: Cost of sales 23,100 8,379 43,309 16,188 Research and development 77,947 95,658 162,647 205,684 Selling, general and administrative 128,081 111,373 257,587 213,466 Operating expenses: 229,128 215,410 463,543 435,338 Net operating loss (29,443) (120,441) (91,534) (295,524)Loss on extinguishment of development liability (1,949) — (1,949) — Interest income 3,184 6,002 6,488 11,395 Interest expense (9,359) (7,341) (16,326) (14,869)Other income / (expense), net 24 (63) (475) (341)Net loss before taxes (37,543) (121,843) (103,796) (299,339)Income tax expense 114 194 284 476 Net loss $(37,657) $(122,037) $(104,080) $(299,815)Other comprehensive gain/(loss): Foreign currency translation 163 (21) 180 79 Total other comprehensive income 163 (21) 180 79 Comprehensive loss, net of tax $(37,494) $(122,058) $(103,900) $(299,736)Net loss per common share, basic and diluted $(0.30) $(1.02) $(0.84) $(2.57)Weighted-average number of common shares used in net loss per common share, basic and diluted 123,904 119,316 123,430 116,594

Generated $172.3 million in 1Q 2024 revenues, including $163.1 million in U.S. net product sales $137.5 million for SYFOVRE® (pegcetacoplan injection) $25.6 million for EMPAVELI® (pegcetacoplan) Anticipates CHMP opinion for pegcetacoplan in GA no later than July 2024On track to report topline Phase 3 data with systemic pegcetacoplan in C3G / IC-MPGN in mid-2024Cash and cash equivalents of $325.9 million as of March 31, 2024; projected revenues and cash expected to be sufficient to fund operations for foreseeable futureManagement to host conference call today at 8:30 a.m. ET WALTHAM, Mass., May 07, 2024 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), today announced its first quarter 2024 financial results and business highlights. “We made excellent progress against our strategic priorities during the quarter, including bringing SYFOVRE to more patients in the U.S., advancing EMPAVELI within multiple high unmet need areas, and progressing our earlier-stage pipeline,” said Cedric Francois, M.D., Ph.D., co-founder and chief executive officer of Apellis. Dr. Francois continued, “SYFOVRE performance continues to exceed expectations in the U.S., with 20% revenue growth in the first quarter, and we are working closely with the EU regulators towards our goal of bringing pegcetacoplan to the GA community in Europe. EMPAVELI continues to elevate the standard of care in PNH, and we are excited about the upcoming topline readout from our Phase 3 VALIANT trial with EMPAVELI for C3G and IC-MPGN. With two commercial products and an emerging pipeline, we are making tremendous progress on our vision to develop life-changing medicines for people living with some of the most challenging diseases.” First Quarter 2024 Business Highlights and Upcoming Milestones Ophthalmology Highlights SYFOVRE for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD): Generated $137.5 million in SYFOVRE U.S. net product revenue in the first quarter of 2024.SYFOVRE is the #1 chosen treatment for GA, delivering approximately 77,000 SYFOVRE doses to physician practices in the first quarter of 2024 (72,000 commercial doses and 5,000 samples). Approximately 250,000 SYFOVRE injections are estimated to have been administered through March 2024, including clinical trials. Launched a branded direct-to-consumer (DTC) awareness campaign in April 2024. The branded campaign builds on the previous disease state awareness campaigns and is aimed at educating GA patients about SYFOVRE.Announced that the European Medicines Agency (EMA) has reset the pegcetacoplan marketing authorization application (MAA) review to day 180, the last phase of the initial assessment. The update follows the judgment by the Court of Justice of the European Union (CJEU) on March 14, 2024, which ruled on the organization of EMA’s expert groups. This decision by EMA is strictly procedural in response to the CJEU judgment and is not related to the pegcetacoplan data package.Apellis now anticipates a CHMP opinion no later than July 2024. Leading retina specialist Dr. Philip Ferrone joined Apellis in March 2024 as chief medical retina advisor. Paroxysmal Nocturnal Hemoglobinuria (PNH) Highlights EMPAVELI for the treatment of PNH: Generated $25.6 million in EMPAVELI U.S. net product revenue in the first quarter of 2024.Continued high patient compliance rates of 97%. R&D Highlights C3 glomerulopathy (C3G) and immune complex glomerulonephritis (IC-MPGN): Topline data from the Phase 3 VALIANT study of systemic pegcetacoplan is expected in mid-2024. VALIANT is a randomized, placebo-controlled, double-blinded, multi-center study designed to evaluate the safety and efficacy of systemic pegcetacoplan in 124 patients aged 12 and up with either C3G or IC-MPGN, and either pre- or post-kidney transplant.The primary endpoint is the log transformed ratio of urine protein-to-creatinine ratio (uPCR) at week 26 compared to baseline. 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.APL-3007 (small interfering RNA silencing C3): Continue to expect to report topline data from the Phase 1 dose escalation study in 2024. First Quarter 2024 Financial Results Total Revenue. Total revenue was $172.3 million for the first quarter of 2024, which consisted of $137.5 million of U.S. net product revenue of SYFOVRE, $25.6 million of U.S. net product revenue of EMPAVELI, and $9.3 million in licensing and other revenue associated with the Sobi collaboration. Total revenue was $44.8 million for the first quarter of 2023, which consisted of, $18.4 million of U.S. net product revenue of SYFOVRE, $20.4 million in net product revenue of EMPAVELI and $6.0 million in revenue associated with the Sobi collaboration. Cost of Sales. Cost of sales was $20.2 million for the first quarter of 2024, compared to $7.8 million for same period in 2023. The increase in cost of sales was primarily driven by an increase due to higher volume from commercial sales and product provided under our patient assistance programs, an increase in royalty expense, and an increase in expenses incurred related to excess or obsolete inventory. R&D Expenses. R&D expenses were $84.7 million for the first quarter of 2024, compared to $110.0 million for the same period in 2023. The decrease in R&D expenses for the first quarter of 2024 was primarily attributable to a decrease in program specific external costs, a decrease in compensation and related personnel costs, and a decrease in non-program specific external costs, which were partially offset by a $15.0 million one-time expense in the first quarter related to the discontinuation of systemic pegcetacoplan for cold agglutinin disease (CAD). Selling, General and Administrative (SG&A) Expenses. SG&A expenses were $129.5 million for the first quarter of 2024, compared to $102.1 million for the same period in 2023. The increase in SG&A expenses for the first quarter of 2024 was primarily attributable to an increase in personnel related costs, an increase in professional and consulting fees and general commercial preparation activities, and higher office costs, which were partially offset by a decrease in travel expenses and a decrease in insurance expenses. Net Loss. Apellis reported a net loss of $66.4 million for the first quarter 2024, compared to a net loss of $177.8 million for the same period in 2023. Cash. As of March 31, 2024, Apellis had $325.9 million in cash and cash equivalents, compared to $351.2 million in cash and cash equivalents as of December 31, 2023. In February 2024, Apellis received $98.8 million in non-dilutive, cash proceeds related to the unwind of its capped call transactions in connection with the issuance of the Company’s 3.500% Senior Convertible Notes due 2026.Apellis anticipates its cash balance, combined with projected sales of EMPAVELI and SYFOVRE, will be sufficient to fund operations for the foreseeable future. Conference Call and WebcastApellis will host a conference call and webcast to discuss its first quarter 2024 financial results and business highlights today, May 7, 2024, at 8:30 a.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 SYFOVRE® (pegcetacoplan injection)SYFOVRE® (pegcetacoplan injection) is the first-ever approved therapy for geographic atrophy (GA). By targeting C3, SYFOVRE is designed to provide comprehensive control of the complement cascade, part of the body’s immune system. SYFOVRE is approved in the United States for the treatment of GA secondary to age-related macular degeneration. 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. It is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally. The therapy is also under investigation for several other rare diseases across hematology and nephrology. U.S. Important Safety Information for SYFOVRE®(pegcetacoplan injection) CONTRAINDICATIONS SYFOVRE is contraindicated in patients with ocular or periocular infections, and in patients with active intraocular inflammation WARNINGS AND PRECAUTIONS Endophthalmitis and Retinal Detachments Intravitreal injections, including those with SYFOVRE, may be associated with endophthalmitis and retinal detachments. Proper aseptic injection technique must always be used when administering SYFOVRE to minimize the risk of endophthalmitis. Patients should be instructed to report any symptoms suggestive of endophthalmitis or retinal detachment without delay and should be managed appropriately. Retinal Vasculitis and/or Retinal Vascular Occlusion Retinal vasculitis and/or retinal vascular occlusion, typically in the presence of intraocular inflammation, have been reported with the use of SYFOVRE. Cases may occur with the first dose of SYFOVRE and may result in severe vision loss. Discontinue treatment with SYFOVRE in patients who develop these events. Patients should be instructed to report any change in vision without delay. Neovascular AMD In clinical trials, use of SYFOVRE was associated with increased rates of neovascular (wet) AMD or choroidal neovascularization (12% when administered monthly, 7% when administered every other month and 3% in the control group) by Month 24. Patients receiving SYFOVRE should be monitored for signs of neovascular AMD. In case anti-Vascular Endothelial Growth Factor (anti-VEGF) is required, it should be given separately from SYFOVRE administration. Intraocular Inflammation In clinical trials, use of SYFOVRE was associated with episodes of intraocular inflammation including: vitritis, vitreal cells, iridocyclitis, uveitis, anterior chamber cells, iritis, and anterior chamber flare. After inflammation resolves, patients may resume treatment with SYFOVRE. Increased Intraocular Pressure Acute increase in IOP may occur within minutes of any intravitreal injection, including with SYFOVRE. Perfusion of the optic nerve head should be monitored following the injection and managed as needed. ADVERSE REACTIONS Most common adverse reactions (incidence ≥5%) are ocular discomfort, neovascular age-related macular degeneration, vitreous floaters, conjunctival hemorrhage. Please see accompanying full Prescribing Information for more information. U.S. Important Safety Information for EMPAVELI® (pegcetacoplan) BOXED WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risks of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor.Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS. CONTRAINDICATIONS Hypersensitivity to pegcetacoplan or to any of the excipientsFor initiation in patients with unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B WARNINGS AND PRECAUTIONS Serious Infections Caused by Encapsulated Bacteria EMPAVELI, a complement inhibitor, increases a patient’s susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis (caused by any serogroup, including non-groupable strains), and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of EMPAVELI treatment is contraindicated in patients with unresolved serious infection caused by encapsulated bacteria. Complete or update vaccination against encapsulated bacteria at least 2 weeks prior to administration of the first dose of EMPAVELI, according to the most current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI. Note that, ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information. If urgent EMPAVELI therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible. The benefits and risks of treatment with EMPAVELI, as well as the benefits and risks of antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by encapsulated bacteria. Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of EMPAVELI in patients who are undergoing treatment for serious infections. EMPAVELI is available only through a restricted program under a REMS. EMPAVELI REMS EMPAVELI is available only through a restricted program under a REMS called EMPAVELI REMS, because of the risk of serious infections caused by encapsulated bacteria. Notable requirements of the EMPAVELI REMS include the following: Under the EMPAVELI REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risks, signs, and symptoms of serious infections caused by encapsulated bacteria, provide patients with the REMS educational materials, ensure patients are vaccinated against encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, prescribe antibacterial drug prophylaxis if patients’ vaccine status is not up to date and treatment must be started urgently, and provide instructions to always carry the Patient Safety Card both during treatment, as well as for 2 months following last dose of EMPAVELI. Pharmacies that dispense EMPAVELI must be certified in the EMPAVELI REMS and must verify prescribers are certified. Further information is available at www.empavelirems.com or 1-888-343-7073. Infusion-Related Reactions 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. ADVERSE REACTIONS Most common adverse reactions in patients with PNH (incidence ≥10%) were injection site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, pain in extremity, hypokalemia, fatigue, viral infection, cough, arthralgia, dizziness, headache, and rash. 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. Please see full Prescribing Information, including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and Medication Guide. About Apellis Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first-ever therapy for geographic atrophy, a leading cause of blindness around the world. We believe we have only begun to unlock the potential of targeting C3 across serious retinal, rare, and neurological diseases. For more information, please visit http://apellis.com or follow us on Twitter and LinkedIn. Apellis Forward-Looking StatementStatements 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 regarding the expected timing of clinical data, the review of the marketing authorization application of SYFOVRE by the EMA. 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 benefit/risk profile of SYFOVRE following the events of retinal vasculitis will impact the Company’s commercialization efforts; whether SYFOVRE will receive approval from foreign regulatory agencies for GA when expected or at all, including the impact of the reported events of retinal vasculitis on the likelihood and timing of such approvals; whether the Company’s clinical trials will be completed when anticipated; whether results obtained in 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 systemic pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for C3G and IC-MPGN or any other indication when expected or at all; the period for which the Company believes that its cash resources will be sufficient to fund its operations; and other factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 27, 2024 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. Media Contact:Lissa Pavlukmedia@apellis.com617.977.6764 Investor Contact:Meredith Kayameredith.kaya@apellis.com617.599.8178 APELLIS PHARMACEUTICALS, INC.CONDENSED CONSOLIDATED BALANCE SHEETS(Amounts in thousands, except per share amounts) March 31, December 31, 2024 2023 Assets(Unaudited) Current assets: Cash and cash equivalents$325,923 $351,185 Accounts receivable 267,837 206,442 Inventory 161,283 146,362 Prepaid assets 43,163 38,820 Restricted cash 1,103 1,114 Other current assets 12,119 22,408 Total current assets 811,428 766,331 Non-current assets: Right-of-use assets 14,994 16,745 Property and equipment, net 4,195 4,345 Other assets 1,313 1,309 Total assets$831,930 $788,730 Liabilities and Stockholders' Equity Current liabilities: Accounts payable$26,788 37,516 Accrued expenses 101,399 127,806 Current portion of development liability 77,287 75,830 Current portion of lease liabilities 6,257 6,441 Deferred Revenue 3,560 — Total current liabilities 215,291 247,593 Long-term liabilities: Long-term development liability 244,426 239,817 Convertible senior notes 93,109 93,033 Lease liabilities 9,770 11,454 Other liabilities 2,658 2,312 Total liabilities 565,254 594,209 Stockholders' equity: Preferred stock, $0.0001 par value; 10,000 shares authorized and zero shares issued and outstanding at March 31, 2024 and December 31, 2023 — — Common stock, $0.0001 par value; 200,000 shares authorized at March 31, 2024 and December 31, 2023; 121,267 shares issued and outstanding at March 31, 2024, and 119,556 shares issued and outstanding at December 31, 2023 12 12 Additional paid-in capital 3,174,100 3,035,539 Accumulated other comprehensive loss (3,525) (3,542)Accumulated deficit (2,903,911) (2,837,488)Total stockholders' equity 266,676 194,521 Total liabilities and stockholders' equity$831,930 $788,730 APELLIS PHARMACEUTICALS, INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(Amounts in thousands, except per share amounts) For the Three Months Ended March 31, 2024 2023 (Unaudited)Revenue: Product revenue, net$163,075 $38,800 Licensing and other revenue 9,250 6,046 Total revenue: 172,325 44,846 Operating expenses: Cost of sales 20,209 7,809 Research and development 84,701 110,027 Selling, general and administrative 129,505 102,093 Operating expenses: 234,415 219,929 Net operating loss (62,090) (175,083)Interest income 3,303 5,393 Interest expense (6,967) (7,529)Other (expense) /income, net (499) (277)Net loss before taxes (66,253) (177,496)Income tax expense 170 282 Net loss$(66,423) $(177,778)Other comprehensive gain/(loss): Foreign currency translation 17 100 Total other comprehensive income 17 100 Comprehensive loss, net of tax$(66,406) $(177,678)Net loss per common share, basic and diluted$(0.54) $(1.56)Weighted-average number of common shares used in net loss per common share, basic and diluted 122,957 113,872

The United States Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NM5072 for the treatment of anemia in PNH patients. The ODD provides NM5072 with a range of benefits, including financial incentives, regulatory support, extended market exclusivity, and extended patent life, ensuring that this groundbreaking treatment can reach those who need it. NM5072 is an anti-Properdin antibody that selectively blocks the alternative pathway (AP) while maintaining the integrity of the classical pathway required for fending off infections. The drug has completed phase I trial in 48 healthy volunteers with a clean safety pro was well-tolerated across all doses tested up to 20 mg/kg. Total AP inhibition was achieved, with its duration exhibiting a dose-dependent response. NM5072 is being positioned to enter Phase II trial for the treatment of naïve PNH patients as the first clinical indication. The drug is expected to be useful in other complement-mediated and complement-associated disorders, further expanding the patient population for multiple clinical trials. CLEVELAND, April 15, 2024 (GLOBE NEWSWIRE) -- NovelMed today announced that the Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to NM5072, an Alternative Pathway (AP) blocker anti-Properdin antibody, for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). NM5072 is being developed for PNH patients in the United States and globally. The FDA grants ODD status to new medication intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. PNH is a rare, chronic, inflammatory, and hemolytic disease that occurs when PNH Red Blood Cells (RBCs) breakdown within and outside the blood vessel, causing anemia, which can be fatal. In addition to PNH RBCs, other cells, including Neutrophils, Monocytes, and Platelets, also drive cellular destruction and inflammation, leading to long-term effects on the patient’s health. If the disease remains untreated or partially treated, patients will continue to have anemia and inflammation, potentially leading to chronic pain, fatigue, and other common symptoms of PNH. Dr. Rekha Bansal, Chief Executive Officer said, “PNH is a rare disease which involves a range of blood cells that contribute to debilitating symptoms for patients, including anemia, fatigue, and severe pain with shorter life span if remains untreated. The current standard of care requires further improvements. We are hopeful that NM5072, with its unique mechanism of action that targets the top of the complement cascade, could become a promising treatment to improve outcomes in these patients.” NM5072 is under regulatory review for multiple indications in the USA and globally. “We are thrilled by the FDA’s decision, which highlights the demand for groundbreaking therapies for individuals grappling with PNH,” comments Robert Bard, VP Regulatory Affairs. This Orphan Drug Designation approval represents a remarkable stride in therapeutic innovation, showcasing NM5072’s distinct capability to selectively inhibit the alternative pathway (AP) while safeguarding the classical pathway essential for combating infections in PNH patients. NM5072 is another drug in our pipeline that has received Orphan Drug Designation in the United States. Orphan Drug Designation (ODD) The ODD from the FDA is a special designation granted to facilitate the development and assessment of new medicines. The FDA’s Office of Orphan Products Development has granted ODD for NM5072 which is in development for the treatment and prevention of rare diseases or conditions affecting fewer than 200,000 people in the US. This designation offers various advantages which include the potential for market exclusivity lasting seven years upon FDA approval, eligibility for tax credits related to qualified clinical trials, waiver of application fees, reduced annual product fees, clinical protocol assistance, and the potential qualification for expedited development programs. NM5072 (Anti-Properdin Monoclonal Antibody) NM5072 is being developed by NovelMed as a potential first-in-class monoclonal antibody that selectively blocks parts of the immune system responsible for the disease, thus inhibiting alternative pathway cascades. Blocking Properdin function is expected to block the lysis of PNH RBCs, which are implicated in the pathophysiology of anemia in PNH. NM5072 is currently an investigational drug that selectively binds to Properdin, a protein of the AP. This monoclonal antibody serves as an efficacious blocker of the AP without affecting the part of the immune system required for fighting infections. NM5072 has successfully completed a phase I trial in healthy volunteers with an expected safety profile. This potent drug is in various regulatory phases of development. With its much longer half-life, the Anti-Properdin molecule is a pioneering biologic. “While the drug is currently in the form of an IV formulation, subcutaneous formulations are being planned for future trials,” said Dr. Bansal. Regulatory preparations and review of Phase II trials for multiple indications, including PNH, C3 Glomerulopathy (C3G) and Atypical Hemolytic Uremic Syndrome (aHUS) are underway. Complement-Mediated Disorders The dysregulation of the Alternative Pathway (AP) is a pivotal factor influencing the trajectory of numerous acute and chronic rare diseases, spanning disciplines such as hematology, ophthalmology, nephrology, dermatology, and neurology. Among these, Paroxysmal Nocturnal Hemoglobinuria (PNH) emerges as a prominent indication for complement blockers heading towards FDA approval. In the context of PNH, the current armamentarium of treatment options remains limited and imperfect, often compromising pathogenic clearance or inducing hyperlipidemia. PNH manifests with a spectrum of clinical features, including low PNH-RBC clone size, hemolysis, hemoglobinuria, reduced hemoglobin levels, elevated lactate dehydrogenase (LDH), and a plethora of other symptoms that can precipitate chronic complications, potentially leading to premature mortality if left untreated. Chronic symptoms and multisystem organ damage are recurrent in PNH patients. While FDA-approved anti-C3, C5, and Factor B drugs provide potential for symptom management in PNH treatment, they should be approached with caution due to their significant side effects and broader limitations on labels. The imperative to develop additional treatment modalities for PNH, characterized by both efficacy and safety, remains unequivocally recognized, holding the promise of enhancing disease outcomes and improving the quality of life for affected individuals. About NovelMed Dedicated to pioneering innovative biologics for a range of complement-mediated diseases, NovelMed stands at the forefront of conceptualizing and validating an anti-Properdin antibody for chronic complement-mediated and complement-associated disorders. Progressing its leading product candidate through clinical trials, the company primarily focuses on addressing hemolytic and renal disorders initially. “Driven by a commitment to ingenuity and compassion, NovelMed is committed to delivering groundbreaking diagnostics and therapies that redefine the treatment landscape for patients with rare diseases,” said Dr. Rekha Bansal, Chief Executive Officer. Positioned as a frontrunner in the development of therapies targeting the Alternative Pathway (AP), NovelMed is devoted to addressing debilitating diseases across hematology, ophthalmology, Nephrology, dermatology, and neurology. Our R&D team is currently engaged in the development of a new diagnostic marker aimed at identifying complement-mediated diseases. This synergistic strategy is geared towards broadening the spectrum of diseases that can be effectively and confidently treated using NM5072. Seeking Partnership to Advance the Development of Drug candidates for Rare Diseases - NovelMed is actively seeking licensing, investment, partnership, and acquisition opportunities to propel NM5072 through further development and approval across multiple rare disease indications in the US and globally. For more information about NM5072, please visit . Contact: Ya Gao BD@novelmed.com (216) 440-2696 The expected efficacy of the NM5072 treatment A photo accompanying this announcement is available at NM5072, anti-Properdin antibody, for treating PNH and other rare diseases By blocking Properdin function, NM5072 is expected to block the PNH RBC lysis, which is implicated in the pathophysiology of anemia in PNH.