On November 1, 2018 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported financial results and provided a business update for the three months ended September 30, 2018 (Press release, Lexicon Pharmaceuticals, NOV 1, 2018, View Source;2018.htm [SID1234530613]).

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"In the third quarter, we and our collaborator, Sanofi, have been working diligently with regulatory agencies to make sotagliflozin available for patients with type 1 diabetes as quickly as possible and we continue to make progress in growing XERMELO for its current indication," said Lonnel Coats, Lexicon’s president and chief executive officer. "Our collaborator, Ipsen, continues to gain approvals and market authorizations for XERMELO in Europe. We are making good progress on our pipeline. By end of year, we expect to start a clinical trial for telotristat ethyl, the investigational form of XERMELO, in biliary tract cancer as well as announce data for LX2761 in diabetes and LX9211, a neuropathic pain candidate, in healthy volunteers."

Third Quarter Product and Pipeline Highlights

XERMELO (telotristat ethyl) 250 mg

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XERMELO was approved in Australia in September for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by somatostatin analog (SSA) therapy.
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In the third quarter, Ipsen launched XERMELO in several European countries including Sweden and Switzerland.

Sotagliflozin

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In September, clinical data for sotagliflozin were presented at the European Association for the Study of Diabetes (EASD) 54th annual meeting. Data from patient exit interviews from a sotagliflozin Phase 3 study were also presented, reporting meaningful improvements in patient reported outcomes.

Third Quarter 2018 Financial Highlights

Revenues: Revenues for the three months ended September 30, 2018 decreased to $6.9 million from $26.9 million for the corresponding period in 2017, primarily due to lower revenues recognized from the collaboration and license agreement with Sanofi, partially offset by an increase in net product revenues. Net product revenues for the three months ended September 30, 2018 included $6.3 million from net sales of XERMELO in the U.S., up 19% from the prior year quarter and 5% from the second quarter of 2018.

Cost of Sales: Cost of sales related to sales of XERMELO for each of the three months ended September 30, 2018 and 2017 was $0.6 million.

Research and Development (R&D) Expenses: Research and development expenses for the three months ended September 30, 2018 decreased to $13.8 million from $39.1 million for the corresponding period in 2017, primarily due to lower external clinical development costs relating to sotagliflozin.

Selling, General and Administrative (SG&A) Expenses: Selling, general and administrative expenses for the three months ended September 30, 2018 decreased to $15.6 million from $16.7 million for the corresponding period in 2017, primarily due to decreased marketing costs.

Net Loss: Net loss for the three months ended September 30, 2018 was $27.5 million, or $0.26 per share, compared to a net loss of $30.7 million, or $0.29 per share, in the corresponding period in 2017. For the three months ended

September 30, 2018 and 2017, net loss included non-cash, stock-based compensation expense of $2.9 million and $2.6 million, respectively.

Cash and Investments: As of September 30, 2018, Lexicon had $187.3 million in cash and investments, as compared to $310.8 million as of December 31, 2017.

Anticipated Near-Term Milestones

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4Q 2018 – Phase 1b data for LX2761 in type 2 diabetes
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4Q 2018 – Phase 1a data for LX9211 (neuropathic pain candidate) in healthy volunteers
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4Q 2018 – Initiation of clinical development of telotristat ethyl in biliary tract cancer
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March 22, 2019 – PDUFA date for sotagliflozin in type 1 diabetes

Conference Call and Webcast Information

Lexicon management will hold a live conference call and webcast today at 8:00 am EDT / 7:00 am CDT to review its financial and operating results and to provide a general business update. The dial-in number for the conference call is 888-645-5785 (U.S./Canada) or 970-300-1531 (international). The conference ID for all callers is 6394419. The live webcast and replay may be accessed by visiting Lexicon’s website at www.lexpharma.com/investors. An archived version of the webcast will be available on the website for 14 days.

About XERMELO (telotristat ethyl)

Discovered using Lexicon’s unique approach to gene science, XERMELO (telotristat ethyl) is the first and only approved oral therapy for carcinoid syndrome diarrhea in combination with SSA therapy in adults inadequately controlled by SSAs. XERMELO targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within metastatic neuroendocrine tumor (mNET) cells. Lexicon has built the in-house capability and infrastructure to launch and market XERMELO in the U.S., where it retains all commercialization rights. Lexicon also retains rights to market XERMELO in Japan. Lexicon has established a license and collaboration agreement with Ipsen to commercialize XERMELO in Europe and other countries outside of U.S. and Japan.

XERMELO was approved by the U.S. Food and Drug Administration on February 28, 2017 and by the European Commission on September 19, 2017 for the treatment of carcinoid syndrome diarrhea in combination with SSA therapy in adults inadequately controlled by SSA therapy. Carcinoid syndrome is a rare condition that occurs in patients living with metastatic NETs (mNETs) and is characterized by frequent and debilitating diarrhea. XERMELO targets the overproduction of serotonin inside mNET cells, providing an additional treatment option for patients suffering from carcinoid syndrome diarrhea.

XERMELO (telotristat ethyl) Important Safety Information

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Warnings and Precautions: XERMELO may cause constipation, which can be serious. Monitor for signs and symptoms of constipation and/or severe, persistent, or worsening abdominal pain in patients taking XERMELO. Discontinue XERMELO if severe constipation or severe, persistent, or worsening abdominal pain develops.
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Adverse Reactions: The most common adverse reactions (≥5%) include nausea, headache, increased gamma-glutamyl-transferase, depression, flatulence, decreased appetite, peripheral edema, and pyrexia.
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Drug Interactions: If necessary, consider increasing the dose of concomitant CYP3A4 substrates, as XERMELO may decrease their systemic exposure. If combination treatment with XERMELO and short-acting octreotide is needed, administer short-acting octreotide at least 30 minutes after administering XERMELO.

For more information about XERMELO, see Full Prescribing Information at www.xermelo.com.

About Sotagliflozin

Discovered using Lexicon’s unique approach to gene science, sotagliflozin is an investigational oral dual inhibitor of two proteins responsible for glucose regulation known as sodium-glucose co-transporter types 1 and 2 (SGLT1 and SGLT2). SGLT1 is responsible for glucose absorption in the gastrointestinal tract, and SGLT2 is responsible for glucose reabsorption by the kidney.

Lexicon entered into a collaboration and license agreement with Sanofi in November 2015 under which Lexicon granted Sanofi an exclusive, worldwide (excluding Japan), royalty-bearing right and license to develop, manufacture and commercialize sotagliflozin. Lexicon is responsible for all clinical development activities relating to type 1 diabetes and has exercised an exclusive option to co-promote and have a significant role, in collaboration with Sanofi, in the commercialization of sotagliflozin for the treatment of type 1 diabetes in the U.S. Sanofi is responsible for all clinical development and commercialization of sotagliflozin for the treatment of type 2 diabetes worldwide (excluding Japan) and is solely responsible for the commercialization of sotagliflozin for the treatment of type 1 diabetes outside the U.S. (excluding Japan). A New Drug Application and a Marketing Authorization Application for sotagliflozin are currently under review at the U.S. Food and Drug Administration and the European Medicines Agency, respectively, and the product has not yet been approved for use in the U.S. or in Europe.

On November 1, 2018 argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported updated data from the ongoing Phase 1 dose-escalation part of its Phase 1/2 clinical trial of cusatuzumab (ARGX-110) in acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) (Press release, argenx, NOV 1, 2018, View Source [SID1234530646]). As of the data cut-off on July 16, 2018, a 92% (11/12 patients) overall response rate (ORR) was observed, with 42% of patients achieving minimal residual disease (MRD) negativity. The median response duration was 6.9 months as of the data cut-off date.

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Data from this ongoing AML trial are expected to be further updated, including ORR and duration of response, during a company workshop to be held around the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

"The clinical data published today are encouraging and underscore our decision to move forward into a Phase 2 clinical trial of cusatuzumab in AML, which we launched earlier this year. These data also support the deep understanding of the disease biology of CD70 as demonstrated by the preclinical work of our collaborators at the University of Bern, highlighting cusatuzumab’s potential to offer a novel mechanism of action in AML against leukemic blasts and leukemic stem cell compartments," commented Nicolas Leupin, MD, Chief Medical Officer of argenx. "We look forward to providing an update on key metrics from the Phase 1 dose-escalation of the Phase 1/2 trial, including response rate and duration of response, at our annual workshop around ASH (Free ASH Whitepaper)."

Phase 1/2 Results for Cusatuzumab in AML

In this trial, argenx evaluated the safety, tolerability and efficacy of cusatuzumab in combination with azacytidine (AZA) in an open-label, Phase 1/2 clinical trial in 12 newly diagnosed AML patients unfit for intensive chemotherapy. The data published today are from all 12 patients across four dose cohorts (1 mg/kg, 3 mg/kg, 10 mg/kg and 20 mg/kg) from the Phase 1/2 dose-escalation trial.

As of the data cut-off on July 16, 2018, the ORR across the 12 patients was 92% (11/12 patients), including 9 patients (82%) with a complete response with or without hematologic recovery (CR/CRi), 1 patient (9%) who reached morphologic leukemia-free status, and 1 (9%) partial response (PR). The median duration on study as of data cut-off was 6.9 months, ranging from 2 to 14.4 months, with 7 patients still on study. Five patients (42%) achieved MRD negativity as measured by flow cytometry. Translational data demonstrated that cusatuzumab monotherapy and in combination with AZA

significantly reduced leukemic stem cells in the bone marrow of AML patients. Cusatuzumab continued to be well-tolerated in AML patients across the different doses. More details can be found here.

Cusatuzumab in Cutaneous T-cell Lymphoma

argenx also announced today updated results from its non-randomized, open-label, multicenter Phase 1/2 trial of cusatuzumab in 27 patients with cutaneous T-cell lymphoma (CTCL). More details can be found here.

argenx announced in May 2018 that it is no longer devoting resources to the development of cusatuzumab in CTCL in order to focus on the drug candidate’s potential in AML.

About Cusatuzumab

Cusatuzumab (ARGX-110) is an investigational SIMPLE Antibody targeting CD70, an immune checkpoint target involved in hematological malignancies, several solid tumors and severe autoimmune diseases. Cusatuzumab is designed to: block CD70, kill cancer cells expressing CD70 through complement dependent cytotoxicity, enhanced antibody-dependent cell-mediated phagocytosis and enhanced antibody-dependent cell-mediated cytotoxicity, and restore immune surveillance against solid tumors (Silence K. et al. mAbs 2014; 6 (2):523-532). Cusatuzumab is currently being evaluated in patients with hematological malignancies, including a Phase 1/2 trial in combination with azacitidine in patients with newly diagnosed acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) and the Phase 2 part of a Phase 1/2 trial in patients with relapsed/refractory cutaneous T-cell lymphoma (CTCL). Preclinical work on cusatuzumab in AML was performed in collaboration with the Tumor Immunology Lab of Prof. A. F. Ochsenbein at the University of Bern, who won, together with Prof. Manz at the University Hospital of Zürich, the prestigious 2016 Otto Naegeli Prize for his breakthrough research on CD70/CD27 signaling with therapeutic potential for cancer patients.

On November 1, 2018 In conjunction with DURECT Corporation’s (Nasdaq: DRRX) reported third quarter 2018 financial results press release, you are invited to listen to a conference call that will be broadcast live over the internet on Wednesday, November 7, 2018 at 4:30 pm Eastern Time (1:30 pm Pacific Time) (Press release, DURECT, NOV 1, 2018, http://investors.durect.com/phoenix.zhtml?c=121590&p=irol-newsArticle&ID=2374981 [SID1234530663]).

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A live audio webcast of the presentation will be available by accessing DURECT’s homepage at www.durect.com and clicking "Investor Relations." If you are unable to participate during the live webcast, the call will be archived on DURECT’s website under Audio Archive in the "Investor Relations" section.

On November 1, 2018 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that updated data for umbralisib (TGR-1202), the Company’s once-daily PI3K delta inhibitor, and ublituximab (TG-1101), the Company’s novel glycoengineered anti-CD20 monoclonal antibody, have been selected for presentation at the upcoming 60thAmerican Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting and exposition, to be held December 1-4, 2018, at the San Diego Convention Center in California (Press release, TG Therapeutics, NOV 1, 2018, View Source [SID1234532243]). Abstracts are now available online and can be accessed on the ASH (Free ASH Whitepaper) meeting website at www.hematology.org. Abstract highlights and presentation details are outlined below.

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Abstract Highlights:

Umbralisib + Ublituximab + Pembrolizumab Triple Therapy Oral Presentation:
89% ORR (8 of 9) observed in relapsed or refractory Chronic Lymphocytic Leukemia (CLL) patients with 75% ORR (3 of 4) in BTK refractory CLL patients
Notably 2 of 4 BTK-refractory CLL patients achieved a response to umbralisib plus ublituximab ("U2") alone, prior to the addition of pembrolizumab
50% ORR (2 of 4) observed in patients with Richter’s Transformation (RT)
Both responders were ibrutinib refractory and achieved durable Complete Responses (CRs) (time on therapy 15+ months and 7+ months)

Umbralisib + Ublituximab + Bendamustine Triple Therapy Poster Presentation:
85% ORR (11 of 13) observed in relapsed or refractory Follicular Lymphoma (FL) patients, including 54% CRs
48% ORR (12 of 25) observed in relapsed or refractory Diffuse Large B-cell Lymphoma (DLBCL) patients, including 32% CRs
Oral Presentation Details:

Title: Phase I/II Study of Umbralisib (TGR-1202) in Combination with Ublituximab (TG-1101) and Pembrolizumab in Patients with Relapsed/Refractory CLL and Richter’s Transformation
Publication Number: 297
Oral Session: 642. CLL: Therapy, excluding Transplantation: Cellular Therapy and Immunomodulation in CLL
Session Date and Time: Sunday, December 2, 2018; 7:30 AM – 9:00 AM PT
Presentation Time: 8:00 AM PT
Location: Marriott Marquis San Diego Marina, Pacific Ballroom 20
Presenter: Anthony R. Mato, MD, Memorial Sloan-Kettering Cancer Center, New York, NY
Poster Presentation Details:

Title: Combination of Umbralisib, Ublituximab, and Bendamustine Is Safe and Highly Active in Patients with Advanced Diffuse Large B-Cell Lymphoma and Follicular Lymphoma
Abstract Number: 4197
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster III
Date and Time: Monday, December 3, 2018; 6:00 PM – 8:00 PM PT
Location: San Diego Convention Center, Hall GH
Presenter: Matthew A. Lunning, DO, University of Nebraska Medical Center, Omaha, NE
Following each presentation, the data presented will be available on the Publications page of the Company’s website at View Source

TG THERAPEUTICS INVESTOR & ANALYST EVENT

TG Therapeutics will also host a reception on Sunday, December 2, 2018 beginning at 7:30 PM PT with featured presentations beginning promptly at 8:00 PM PT. The event will take place at the Marriott Gaslamp in San Diego California. The event will be webcast live and will be available on the Events page, located within the Investors & Media section of the Company’s website at View Source, as well as archived for future review. This event will also be broadcast via conference call. To access the conference line, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), and reference Conference Title: TG TherapeuticsDecember 2018 Investor & Analyst Event.

On November 1, 2018 Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) reported that nine abstracts from its complement research and development program have been accepted for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, December 1 to 4, 2018 (Press release, Alexion, NOV 1, 2018, View Source [SID1234530490]). Key data will include both new analyses and previously announced results from the two Phase 3 studies of ALXN1210, the Company’s investigational long-acting C5 complement inhibitor, in patients with paroxysmal nocturnal hemoglobinuria (PNH), in addition to further data on Soliris (eculizumab) for the treatment of PNH and atypical hemolytic uremic syndrome (aHUS). Collectively, the breadth of the data to be presented at ASH (Free ASH Whitepaper) demonstrates continued progress extending the company’s leadership in understanding and treating rare complement-mediated diseases.

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The accepted abstracts are listed below and are now available on the ASH (Free ASH Whitepaper) website.

ALXN1210

A Phase 3 Study of Ravulizumab (ALXN1210) versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria Naive to Complement Inhibitors: Results of a Subgroup Analysis with Patients Stratified by Baseline Hemolysis Level, Transfusion History, and Demographics. Abstract ID#: 110623 – Oral Presentation, December 3, 2018, 11:00-11:15 a.m. PST, Grand Hall C.

Results from a Phase 3, Multicenter, Non-Inferiority Study of Ravulizumab (ALXN1210) Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with Eculizumab. Abstract ID#: 119147 – Oral Presentation, December 3, 10:30-10:45 a.m. PST, Grand Hall C.

Ravulizumab (ALXN1210) versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria: Pharmacokinetics and Pharmacodynamics Observed in Two Phase 3 Randomized, Multicenter Studies. Abstract ID#: 110858 – Oral Presentation, December 3, 10:45-11:00 a.m. PST, Grand Hall C.

A Prospective Analysis of Breakthrough Hemolysis in 2 Phase 3 Randomized Studies of Ravulizumab (ALXN1210) versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria. Abstract ID#: 110874 – Poster Presentation, December 2, 6:00-8:00 p.m. PST, Hall GH.

Soliris (eculizumab)

Efficacy of Eculizumab in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria in the International PNH Registry. Abstract ID#: 111306 – Poster Presentation, December 3, 6:00-8:00 p.m. PST, Hall GH.

Economic Benefit of Early In-hospital Diagnosis and Treatment Initiation of Eculizumab in aHUS. Abstract ID#: 112893 – Poster Presentation, December 2, 6:00-8:00 p.m. PST, Hall GH.

PNH

Prognostic Value of Clone Size in Paroxysmal Nocturnal Hemoglobinuria (PNH) for Thrombotic Events in Untreated Patients in the International PNH Registry. Abstract ID#: 111324 – Poster Presentation, December 1, 6:15-8:15 p.m. PST, Hall GH.

Baseline Characteristics of Patients with Paroxysmal Nocturnal Hemoglobinuria Identified in the Department of Defense Database. Abstract ID#: 113478 – online.

The Value of Population Based Data to Study Rare Diseases: An Example Using the Department of Defense Healthcare System. Abstract ID#: 113497 – online.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, debilitating, and potentially life-threatening ultra-rare blood disorder that can strike men and women of all races, backgrounds, and ages without warning, with an average age of onset in the early 30s.1,2,3 PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years.2 In patients with PNH, chronic, uncontrolled activation of the complement system, a component of the body’s immune system, results in hemolysis (the destruction of red blood cells)4, which in turn can result in progressive anemia, fatigue, dark urine, and shortness of breath.5,6,7 The most devastating consequence of chronic hemolysis is thrombosis (the formation of blood clots), which can damage vital organs and cause premature death.8 Historically, it had been estimated that one in three patients with PNH did not survive more than five years from the time of diagnosis.2 PNH is more common among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS).9,10,11 In certain patients with thrombosis of unknown origin, PNH may be an underlying cause.4

About ALXN1210

ALXN1210 is an innovative, investigational, long-acting C5 inhibitor discovered and developed by Alexion that works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system that, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG). In Phase 3 clinical studies in complement inhibitor-naïve patients with PNH, and patients with PNH who had been stable on Soliris, intravenous treatment with ALXN1210 every eight weeks demonstrated non-inferiority to intravenous treatment with Soliris every two weeks, with numeric results for all primary and key secondary endpoints favoring ALXN1210. ALXN1210 is also currently being evaluated in a Phase 3 clinical study in complement inhibitor-naïve patients with aHUS, administered intravenously every eight weeks. In addition, Alexion plans to initiate a Phase 3 clinical study of ALXN1210 delivered subcutaneously once per week as a potential treatment for patients with PNH and aHUS.

ALXN1210 has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S., EU, and Japan, and for the subcutaneous treatment of patients with aHUS in the U.S.

About Soliris (eculizumab)

Soliris is a first-in-class complement inhibitor that works by inhibiting the C5 protein in the terminal part of the complement cascade, a part of the immune system that, when activated in an uncontrolled manner, plays a role in severe rare and ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG). Soliris is approved in the U.S., EU, Japan, and other countries as the first and only treatment for patients with PNH and aHUS, in the EU as the first and only treatment of refractory generalized MG (gMG) in adults who are anti-AchR antibody-positive, in the U.S. for the treatment of adult patients with gMG who are anti-AchR antibody-positive, and in Japan for the treatment of patients with gMG who are AChR antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin (IVIG) therapy or plasmapheresis (PLEX). Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS).

Soliris has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S., EU, Japan, and many other countries, for the treatment of patients with aHUS in the U.S., EU, and many other countries, for the treatment of patients with MG in the U.S. and EU, for the treatment of patients with refractory gMG in Japan, and for the treatment of patients with neuromyelitis optica spectrum disorder (NMOSD) in the U.S., EU, and Japan. Alexion and Soliris have received some of the pharmaceutical industry’s highest honors for the medical innovation in complement inhibition: the Prix Galien USA (2008, Best Biotechnology Product) and France (2009, Rare Disease Treatment).

For more information on Soliris, please see full prescribing information for Soliris, including BOXED WARNING regarding risk of serious meningococcal infection, available at www.soliris.net.

Important Soliris Safety Information

The U.S. prescribing information for Soliris includes the following warnings and precautions: Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Comply with the most current Centers for Disease Control (CDC)’s Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients with complement deficiencies. Immunize patients with meningococcal vaccines at least two weeks prior to administering the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risk of developing a meningococcal infection. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected. Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the Soliris REMS, prescribers must enroll in the program. Enrollment in the Soliris REMS program and additional information are available by telephone: 1-888-SOLIRIS (1-888-765-4747) or at www.solirisrems.com.

Patients may have increased susceptibility to infections, especially with encapsulated bacteria. Aspergillus infections have occurred in immunocompromised and neutropenic patients. Children treated with Soliris may be at increased risk of developing serious infections due to Streptococcus pneumoniae and Haemophilus influenza type b (Hib). Soliris treatment of patients with PNH should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established. Administration of Soliris may result in infusion reactions, including anaphylaxis or other hypersensitivity reactions.

In patients with PNH, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, nasopharyngitis, back pain, and nausea. In patients with aHUS, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, diarrhea, hypertension, upper respiratory infection, abdominal pain, vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea, urinary tract infections, and pyrexia. In patients with gMG who are anti-AchR antibody-positive, the most frequently reported adverse reaction observed with Soliris treatment in the placebo-controlled clinical study (≥10%) was musculoskeletal pain.