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Bayer-successful-start-to-2021

On May 12, 2021 The Bayer Group reported that had a successful start to 2021 (Press release, Bayer, MAY 12, 2021, View Source [SID1234579768]). "We’re seeing a good operational performance overall, but we were impacted by negative currency effects as expected," said CEO Werner Baumann when the quarterly statement for the first quarter of 2021 was published on Wednesday . "Our Crop Science Division achieved encouraging sales growth in an improved market environment. Sales at Pharmaceuticals were steady, and in the Consumer Health Division we performed better than the competition." The company confirmed the full-year outlook it issued in February 2021.

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Group sales in the first quarter rose by 2.8 percent on a currency- and portfolio-adjusted basis (Fx & portfolio adj.) to 12.328 billion euros. EBITDA before special items fell by 6.2 percent to 4.118 billion euros. Negative currency effects impacted sales by 938 million euros and EBITDA before special items by 337 million euros. EBIT increased by 23.4 percent to 3.083 billion euros. This included net special gains of 15 million euros (Q1 2020: net special charges of 639 million euros), with income from a patent dispute related to the blood-clotting medicine Jivi more than offsetting the expenses for the ongoing restructuring program. Net income rose by 40.3 percent to 2.089 billion euros. Core earnings per share from continuing operations fell by 3.0 percent to 2.59 euros.

Free cash flow came in at minus 3.226 billion euros (Q1 2020: minus 793 million euros), the decline being mainly due to the settlement payments for litigations in the United States. This was also partly the reason for the 13.0 percent increase in net financial debt against the end of 2020, to 33.933 billion euros as of March 31, 2021.

Growth at Crop Science, especially in Latin America and Asia/Pacific

Bayer lifted sales in its agricultural business (Crop Science) by 6.4 percent (Fx & portfolio adj.) to 6.646 billion euros, with particularly strong growth in the Latin America and Asia/Pacific regions. Sales at Herbicides advanced by a significant 13.3 percent – mainly as a result of volume gains in all regions and higher prices, especially for Roundup. Business also improved substantially at Fungicides (plus 22.0 percent) and Vegetable Seeds (plus 13.9 percent). The Fungicides business benefited from volume and price increases in the Latin America region, particularly for the product Fox Xpro. This business also expanded in Asia/Pacific as the market situation normalized following the previous year’s restrictions related to the COVID-19 pandemic. Sales at Vegetable Seeds rose in all regions. The 3.4 percent (Fx & portfolio adj.) increase in sales at Soybean Seed & Traits was driven by volume gains in North America. Sales at Corn Seed & Traits remained at the level of the prior-year quarter (Fx & portfolio adj. minus 0.4 percent). This business developed positively in the Europe/Middle East/Africa region and in Latin America, partly as a result of higher prices, whereas it shrank in North America, in part due to a license expiration.

EBITDA before special items at Crop Science decreased by 6.2 percent to 2.448 billion euros, giving a margin of 36.8 percent. Positive effects from price and volume increases and the contributions from the ongoing efficiency programs did not fully offset the negative currency effects of 252 million euros.

As regards the litigation involving glyphosate-based Roundup products in the United States, approximately 96,000 current claims overall are covered by settlement agreements, or did not meet the settlement program eligibility criteria. The company continues to negotiate with plaintiffs’ counsel to reach agreements in the remainder of current cases. A new proposal for the management of future cases agreed upon with and supported by plaintiffs’ class counsel has been submitted to the responsible judge in California for preliminary approval. For the revised proposal, both parties worked diligently to address the points mentioned by the court.

Pharmaceuticals: significant advances for Xarelto and Eylea

Sales of prescription-only medicines (Pharmaceuticals) came in at 4.365 billion euros and were thus level with the prior-year period on a currency- and portfolio-adjusted basis (Fx & portfolio adj. minus 0.4 percent). In China, sales declines for Glucobay and Avelox as a result of the tender procedures implemented in 2020 were offset by growth in other products, especially the oral anticoagulant Xarelto. In the United States, the division benefited particularly from the launch of the cancer drug Nubeqa.

Bayer raised sales of Xarelto by 6.5 percent (Fx & portfolio adj.). This was mainly due to expanded volumes in China and Russia, while business in Germany declined substantially. A considerable increase of 15.8 percent (Fx & portfolio adj.) in sales of the ophthalmology drug Eylea was driven by positive development in Japan and markedly higher volumes in Europe, China and Canada. The business with the oral contraceptives YAZ/Yasmin/Yasminelle grew by 12.3 percent (Fx & portfolio adj.), with increases especially in China, Japan and the Middle East. However, there was a distinct drop in sales of the Kogenate/Kovaltry/Jivi blood-clotting medicines (Fx & portfolio adj. minus 17.9 percent) and the cancer drug Nexavar (Fx & portfolio adj. minus 21.7 percent) due to competition.

EBITDA before special items at Pharmaceuticals declined by 6.0 percent to 1.498 billion euros, yielding a margin of 34.3 percent. Earnings were diminished by price declines in sales along with negative currency effects of 57 million euros, which were offset to some extent by volume gains and cost reductions. There was also a positive effect on research and development expenses that came partly from the proportionate recognition of proceeds from the sale of a priority review voucher in the United States.

Consumer Health: performance ahead of competition cycling over exceptionally strong prior-year quarter

Following an exceptionally strong prior-year quarter with double-digit percentage growth, sales of self-care products (Consumer Health) registered a 4.4 percent decrease (Fx & portfolio adj.) to 1.252 billion euros in a declining market. The performance remained ahead of competition with growth momentum in the Dermatology and Nutritionals categories, with business up by 6.6 percent and 4.7 percent, respectively (Fx & portfolio adj.). However, the continuing high level of protective and hygiene measures and the ongoing lockdowns had a negative impact on sales, particularly on cough and cold products. This led to a decline of 30.1 percent (Fx & portfolio adj.) in the Allergy & Cold category. While sales in Asia/Pacific and Latin America showed continued positive growth, sales in North America and Europe/Middle East/Africa were markedly affected by the extraordinarily mild cough and cold season.

EBITDA before special items at Consumer Health moved back 3.0 percent to 292 million euros. Earnings were diminished by negative currency effects of 26 million euros. At the same time, the EBITDA margin before special items improved by 1.8 percentage points to 23.3 percent, largely on account of lower marketing expenses in the currently volatile market environment as well as one-time gains from the divestment of three non-core brands.

Sustainability efforts further enhanced

Bayer also made good progress in the area of sustainability in the first quarter. As already announced in February, the company is stepping up its efforts to achieve an even more inclusive and diverse workforce and corporate culture, targeting gender parity on all management levels for the year 2030. Also in February, the Consumer Health Division, for example, launched its Nutrient Gap Initiative to help close the gap in the supply of vitamins and minerals to people in underserved regions. The program aims to reach 50 million people annually in these regions by 2030 through direct action and in partnership with major NGOs. The Nutrient Gap Initiative will contribute to the attainment of the Consumer Health Division’s sustainability goal of providing 100 million people in underserved communities with access to everyday health care by 2030.

Avidity Biosciences Reports First Quarter 2021 Financial Results and Recent Highlights

On May 12, 2021 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the first quarter ended March 31, 2021 and highlighted recent corporate progress (Press release, Avidity Biosciences, MAY 12, 2021, View Source [SID1234579767]).

"AOC 1001, the first program from our AOC platform, is advancing toward the clinic and is on track to begin a Phase 1/2 study in adults with DM1 in the second half of this year. Our skeletal muscle pipeline is also progressing as planned while we continue to expand our discovery efforts to other indications," said Sarah Boyce, president and chief executive officer. "We are proud to be at the forefront of RNA innovation by delivering on the promise of our AOC platform and pipeline."

Mike MacLean, chief financial officer, added, "We are off to a great start this year with a strong financial position of over $300 million at the end of the first quarter. This allows us to progress AOC 1001 while continuing to invest in our other pipeline programs and our AOC platform, which we believe is a sustainable engine with the potential to have a broad impact across both rare and other serious diseases."

AOC Platform and Pipeline Highlights

Advanced Lead Program, AOC 1001 for DM1, and Broad Pipeline for Untreated Rare Muscle Diseases.

In April 2021, Avidity presented preclinical data for AOC 1001 for myotonic dystrophy type 1 (DM1) in an oral presentation as part of the Emerging Science Session at the American Academy of Neurology (AAN) 2021 Virtual Annual Meeting. Avidity scientists have demonstrated activity and potency of siRNAs against the dystrophy protein kinase (DMPK) gene in muscle cells derived from patients with DM1. These data demonstrated activity in the nucleus and cytoplasm. The presentation also showed that single doses of AOC 1001 produced 75% reductions in DMPK mRNA expression in preclinical studies that were maintained for months post-dosing. Following regulatory clearance, Avidity remains on track with plans to begin a Phase 1/2 clinical study of AOC 1001 in adults with DM1 in the second half of 2021.

Avidity also advanced additional programs in its muscle franchise including a program for facioscapulohumeral muscular dystrophy (FSHD) and three programs for Duchenne muscular dystrophy (DMD). The AOC FSHD program and the lead AOC DMD program targeting Exon 44 are the most advanced. Avidity remains on track with plans to commence clinical trials for both programs in 2022, following additional preparatory preclinical studies and regulatory clearance.
Upcoming Events

Avidity’s Virtual Investor & Analyst Event Series – Volume 1. Recently, Avidity launched its Virtual Investor and Analyst Event Series. The first virtual event entitled "Engineering Antibody Oligonucleotide Conjugates" will be held on Wednesday, May 19th, 2021 at 8am PT/11am ET.

The virtual event will feature a panel discussion with leading RNA therapeutics key opinion leaders Phillip D. Zamore, Ph.D., Chair, RNA Therapeutics Institute at UMASS and Steven F. Dowdy, Ph.D., Professor of Cellular and Molecular Medicine at UCSD. To register for the event or for more information, please email [email protected].
First Quarter 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $307.9 million as of March 31, 2021, compared to $328.1 million as of December 31, 2020.
Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $2.7 million for the first quarter of 2021 compared with $1.4 million for the first quarter of 2020.
Research and Development (R&D) Expenses: R&D expenses, including external and internal costs associated with research activities, primarily relate to the progression of the company’s research on AOC 1001 and other muscle programs. These expenses were $20.7 million for the first quarter of 2021 compared with $5.5 million for the first quarter of 2020. The increase was primarily driven by the progression of AOC 1001 toward the clinic, as well as research for other programs.
General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs, and patent filing and maintenance fees. These expenses were $5.9 million for the first quarter of 2021 compared with $2.0 million for the first quarter of 2020. The increase was primarily due to higher personnel costs (including noncash stock-based compensation), professional fees and insurance costs related to being a public company.

Aptose to Present at Upcoming Investor Conferences

On May 12, 2021 Aptose Biosciences Inc. (Nasdaq: APTO; TSX: APS), a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, reported that the Aptose management team will participate in upcoming investor conferences (Press release, Aptose Biosciences, MAY 12, 2021, View Source [SID1234579766]):

2021 RBC Capital Markets Global Healthcare Virtual Conference

Date: Wednesday, May 19, 2021
Time: 11:30 AM – 11:55 AM EDT
Format: Fireside Chat moderated by Gregory J. Renza, M.D., Equity Research – Biotechnology

Conference Information: Link
Jefferies Virtual Healthcare Conference

Date: Wednesday, June 2, 2021
Time: 2:00 PM – 2:25 PM EDT
Format: Aptose Slide Presentation and Webcast

Conference Information: Link
The Aptose management team also will be hosting 1×1 meetings during the events.

Caelum and Alexion Announce Upcoming CAEL-101 Data Presentations at the European Hematology Association Congress 2021

On May 12, 2021 Caelum Biosciences and Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) reported that two abstracts on CAEL-101, a potentially first-in-class amyloid fibril targeted therapy, have been accepted for e-poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress 2021, taking place virtually from June 9 to 17, 2021 (Press release, Alexion, MAY 12, 2021, View Source [SID1234579765]). Both presentations will include new data from the Phase 2 open-label dose escalation study evaluating the safety and tolerability of CAEL-101 in combination with standard-of-care (SoC) therapy in AL amyloidosis. One will feature data studying safety and tolerability in AL amyloidosis patients treated with 1000 mg/m2 CAEL-101 in combination with cyclophosphamide-bortezomib-dexamethasone plus daratumumab. The second will include new data further supporting the selection of the 1000 mg/m2 dose for the ongoing Phase 3 study and demonstrating the possible stabilization of cardiac disease as assessed by Mayo staging.

The abstracts accepted for e-poster presentations are listed below and are now available on the EHA (Free EHA Whitepaper) website. All e-poster presentations will be made available on the virtual congress platform on June 11, 2021 at 09:00 a.m. CEST (3:00 a.m. EDT) and will be available throughout the duration of the Congress.

Safety and Tolerability of CAEL-101 in Combination with Cyclophosphamide-Bortezomib-Dexamethasone and Daratumumab in Patients with AL Amyloidosis. Abstract #EP1017– e-poster presentation; session title: Myeloma and other monoclonal gammopathies – Clinical.

Safety and Tolerability of CAEL-101 in Patients with AL Amyloidosis in a Phase 2 Study for a Median of 38 Weeks. Abstract #EP1018 – e-poster presentation; session title: Myeloma and other monoclonal gammopathies – Clinical.

As was previously announced, the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101 in combination with SoC therapy in AL amyloidosis has begun. Enrollment is underway in two parallel Phase 3 studies – one in patients with Mayo stage IIIa disease (ClinicalTrials.gov Identifier: NCT04512235) and one in patients with Mayo stage IIIb disease (ClinicalTrials.gov Identifier: NCT04504825) – and will collectively enroll approximately 370 patients globally. The Phase 2 program continues with the ongoing evaluation of CAEL-101 in combination with SoC therapy plus daratumumab.

About CAEL-101

CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicine Agency as a potential therapy for patients with AL amyloidosis.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs. This deposition can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant.

AL amyloidosis is a rare disease but is the most common form of systemic amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

Alexion Announces Upcoming Data Presentations at the 26th European Hematology Association Virtual Congress

On May 12, 2021 Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) reported that five abstracts have been accepted for presentation at the 26th annual European Hematology Association (EHA) (Free EHA Whitepaper) 2021 virtual Congress, taking place from June 9 through June 17, 2021 (Press release, Alexion, MAY 12, 2021, View Source [SID1234579764]). During the meeting, data will be presented further supporting the long-term safety of ULTOMIRIS (ravulizumab-cwvz) over two years, including reduction of thrombotic (blood clot) risk, in complement inhibitor-naïve and complement inhibitor-experienced patients with PNH, and reaffirming the critical role of complete C5 complement inhibition in treating this rare disease.

Also being presented are new Phase 3 interim analysis data that evaluate the efficacy and safety of ULTOMIRIS in pediatric patients as well as the previously announced topline Phase 3 results for the weekly subcutaneous formulation of ULTOMIRIS in adults with PNH.

"Our data presentations at this year’s EHA (Free EHA Whitepaper) Congress underscore the clinical value of ULTOMIRIS – the standard of care for adults living with PNH – and continue to build on its well-established safety and efficacy profile," said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. "These data also demonstrate further progress in our efforts to continue innovating for patients – supporting the use of ULTOMIRIS in children and adolescents and via a convenient, self-administered subcutaneous formulation."

The U.S. Food and Drug Administration (FDA) granted priority review for ULTOMIRIS in children and adolescents with PNH and has set a Prescription Drug User Fee Act (PDUFA) target action date of June 7, 2021. The interim analysis that will be presented at EHA (Free EHA Whitepaper) is from the largest study of pediatric PNH patients to date.

The accepted abstracts are listed below and are now available on the EHA (Free EHA Whitepaper) website. All e-poster presentations will be made available on the virtual congress platform on June 11, 2021 at 09:00 CEST (3:00 a.m. EDT) and will be available throughout the duration of the Congress.

Oral Presentation

Ravulizumab Reduces the Risk of Thrombosis in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria and High Disease Activity: 2-Year Data from A Phase 3, Open-Label Study. Oral presentation ID #S301. The pre-recorded oral presentation will be published on the virtual Congress platform on June 11, 2021 at 09:00 CEST (3:00 a.m. EDT), with a live Q&A session on June 14, 2021. The session begins at 11:00 CEST (5:00 a.m. EDT).

ePoster Presentations

Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of Ravulizumab In Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria: Interim Analysis of A Phase 3, Open-Label Study. ePoster presentation, abstract ID #EP590.

Safety of Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria: Study 301/302 2-Year Results. ePoster presentation, abstract ID #EP594.

Ravulizumab and Eculizumab Reduce Transfusions in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria: Evidence from Three Real-World Databases: TriNetX US EMR, TriNetX US Claims and KOMODO Health. ePoster presentation, abstract ID #EP1337.

Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients with PNH Previously Treated with Eculizumab: Results from A Phase 3 Randomized, Open-Label Study. ePoster presentation, abstract ID #EP586.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

PNH is a serious ultra-rare blood disorder with devastating consequences. It is characterized by the destruction of red blood cells, which is also referred to as hemolysis. PNH occurs when the complement system—a part of the body’s immune system—over-responds, leading the body to attack its own red blood cells. PNH often goes unrecognized, with delays in diagnosis from one to more than five years. Patients with PNH may experience a range of symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-colored urine and anemia. The most devastating consequence of chronic hemolysis is the formation of blood clots, which can occur in blood vessels throughout the body, damage vital organs, and potentially lead to premature death. The prognosis of PNH can be poor in many cases, so a timely and accurate diagnosis—in addition to appropriate treatment—is critical to improving patient outcomes.

About ULTOMIRIS

ULTOMIRIS (ravulizumab-cwvz) is the first and only long-acting C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. ULTOMIRIS is administered intravenously every eight weeks or, for pediatric patients less than 20 kg, every four weeks, following a loading dose. ULTOMIRIS is approved in the United States (U.S.), European Union (EU) and Japan as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH). It is also approved in the U.S. and Japan for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients, as well as in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS. To learn more about the regulatory status of ULTOMIRIS in the countries that we serve, please visit www.alexion.com.

About SOLIRIS

SOLIRIS (eculizumab) is a first-in-class C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the terminal complement cascade over-responds, leading the body to attack its own healthy cells. SOLIRIS is administered intravenously every two weeks, following an introductory dosing period. In many countries around the world, SOLIRIS is approved to treat paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), adults with generalized myasthenia gravis (gMG) who are acetylcholine receptor (AchR) antibody positive and/or adults with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive. SOLIRIS is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). To learn more about the regulatory status of SOLIRIS in the countries that we serve, please visit www.alexion.com.

INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS (ravulizumab-cwvz)

INDICATIONS

What is ULTOMIRIS?

ULTOMIRIS is a prescription medicine used to treat:

adults with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).
adults and children 1 month of age and older with a disease called atypical Hemolytic Uremic Syndrome (aHUS). ULTOMIRIS is not used in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
It is not known if ULTOMIRIS is safe and effective in children with PNH.

It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about ULTOMIRIS?

ULTOMIRIS is a medicine that affects your immune system and can lower the ability of your immune system to fight infections.

ULTOMIRIS increases your chance of getting serious and life-threatening meningococcal infections that may quickly become life-threatening and cause death if not recognized and treated early.
You must receive meningococcal vaccines at least 2 weeks before your first dose of ULTOMIRIS if you are not vaccinated.
If your doctor decided that urgent treatment with ULTOMIRIS is needed, you should receive meningococcal vaccination as soon as possible.
If you have not been vaccinated and ULTOMIRIS therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.
If you had a meningococcal vaccine in the past, you might need additional vaccination. Your doctor will decide if you need additional vaccination.
Meningococcal vaccines reduce but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: headache with nausea or vomiting, headache and fever, headache with a stiff neck or stiff back, fever, fever and a rash, confusion, muscle aches with flu-like symptoms and eyes sensitive to light.
Your doctor will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last ULTOMIRIS dose. It is important to show this card to any doctor or nurse to help them diagnose and treat you quickly.

ULTOMIRIS is only available through a program called the ULTOMIRIS REMS. Before you can receive ULTOMIRIS, your doctor must: enroll in the ULTOMIRIS REMS program; counsel you about the risk of meningococcal infection; give you information and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with a meningococcal vaccine, and if needed, get revaccinated with the meningococcal vaccine. Ask your doctor if you are not sure if you need to be revaccinated.

ULTOMIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilis influenzae type b (Hib) if treated with ULTOMIRIS. Call your doctor right away if you have any new signs or symptoms of infection.

Who should not receive ULTOMIRIS?

Do not receive ULTOMIRIS if you have a meningococcal infection or have not been vaccinated against meningococcal infection unless your doctor decides that urgent treatment with ULTOMIRIS is needed.

Before you receive ULTOMIRIS, tell your doctor about all of your medical conditions, including if you: have an infection or fever, are pregnant or plan to become pregnant, and are breastfeeding or plan to breastfeed. It is not known if ULTOMIRIS will harm your unborn baby or if it passes into your breast milk. You should not breastfeed during treatment and for 8 months after your final dose of ULTOMIRIS.

Tell your doctor about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment.

If you have PNH and you stop receiving ULTOMIRIS, your doctor will need to monitor you closely for at least 16 weeks after you stop ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in your red blood cell count, tiredness, blood in your urine, stomach-area (abdomen) pain, shortness of breath, blood clots, trouble swallowing, and erectile dysfunction (ED) in males.

If you have aHUS, your doctor will need to monitor you closely for at least 12 months after stopping treatment for signs of worsening aHUS or problems related to a type of abnormal clotting and breakdown of your red blood cells called thrombotic microangiopathy (TMA). Symptoms or problems that can happen with TMA may include: confusion or loss of consciousness, seizures, chest pain (angina), difficulty breathing and blood clots or stroke.

What are the possible side effects of ULTOMIRIS?

ULTOMIRIS can cause serious side effects including infusion-related reactions. Symptoms of an infusion-related reaction with ULTOMIRIS may include lower back pain, pain with the infusion, feeling faint or discomfort in your arms or legs. Tell your doctor or nurse right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion reaction, including: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, and feel faint or pass out.

The most common side effects of ULTOMIRIS in people treated for PNH are upper respiratory infection and headache.

The most common side effects of ULTOMIRIS in people with aHUS are upper respiratory infection, diarrhea, nausea, vomiting, headache, high blood pressure and fever.

Tell your doctor about any side effect that bothers you or that does not go away. These are not all the possible side effects of ULTOMIRIS. For more information, ask your doctor or pharmacist. Call your doctor right away if you miss an ULTOMIRIS infusion or for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see the accompanying full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections/sepsis.

INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS (eculizumab)

INDICATIONS

What is SOLIRIS?

SOLIRIS is a prescription medicine used to treat:

patients with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH) .
adults and children with a disease called atypical Hemolytic Uremic Syndrome (aHUS). SOLIRIS is not for use in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
adults with a disease called generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.
adults with a disease called neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.
It is not known if SOLIRIS is safe and effective in children with PNH, gMG, or NMOSD.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about SOLIRIS?

SOLIRIS is a medicine that affects your immune system and can lower the ability of your immune system to fight infections.

SOLIRIS increases your chance of getting serious and life-threatening meningococcal infections that may quickly become life-threatening and cause death if not recognized and treated early.
You must receive meningococcal vaccines at least 2 weeks before your first dose of SOLIRIS if you are not vaccinated.
If your doctor decided that urgent treatment with SOLIRIS is needed, you should receive meningococcal vaccination as soon as possible.
If you have not been vaccinated and SOLIRIS therapy must be initiated immediately, you should also receive two weeks of antibiotics with your vaccinations.
If you had a meningococcal vaccine in the past, you might need additional vaccination. Your doctor will decide if you need additional vaccination.
Meningococcal vaccines reduce but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: headache with nausea or vomiting, headache and fever, headache with a stiff neck or stiff back, fever, fever and a rash, confusion, muscle aches with flu-like symptoms, and eyes sensitive to light.
Your doctor will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 3 months after your last SOLIRIS dose. It is important to show this card to any doctor or nurse to help them diagnose and treat you quickly.

SOLIRIS is only available through a program called the SOLIRIS REMS. Before you can receive SOLIRIS, your doctor must enroll in the SOLIRIS REMS program; counsel you about the risk of meningococcal infection; give you information and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with the meningococcal vaccine and, if needed, get revaccinated with the meningococcal vaccine. Ask your doctor if you are not sure if you need to be revaccinated.

SOLIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) if treated with SOLIRIS. Certain people may be at risk of serious infections with gonorrhea. Certain fungal infections (Aspergillus) may occur if you take SOLIRIS and have a weak immune system or a low white blood cell count.

Who should not receive SOLIRIS?

Do not receive SOLIRIS if you have a meningococcal infection or have not been vaccinated against meningitis infection unless your doctor decides that urgent treatment with SOLIRIS is needed.

Before you receive SOLIRIS, tell your doctor about all of your medical conditions, including if you: have an infection or fever, are pregnant or plan to become pregnant, and are breastfeeding or plan to breastfeed. It is not known if SOLIRIS will harm your unborn baby or if it passes into your breast milk.

Tell your doctor about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment. It is important that you have all recommended vaccinations before you start SOLIRIS, receive 2 weeks of antibiotics if you immediately start SOLIRIS, and stay up-to-date with all recommended vaccinations during treatment with SOLIRIS.

If you have PNH, your doctor will need to monitor you closely for at least 8 weeks after stopping SOLIRIS. Stopping treatment with SOLIRIS may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in the number of your red blood cell count, drop in your platelet count, confusion, kidney problems, blood clots, difficulty breathing, and chest pain.

If you have aHUS, your doctor will need to monitor you closely during and for at least 12 weeks after stopping treatment for signs of worsening aHUS symptoms or problems related to abnormal clotting (thrombotic microangiopathy). Symptoms or problems that can happen with abnormal clotting may include: stroke, confusion, seizure, chest pain (angina), difficulty breathing, kidney problems, swelling in arms or legs, and a drop in your platelet count.

What are the possible side effects of SOLIRIS?

SOLIRIS can cause serious side effects including serious infusion-related reactions. Tell your doctor or nurse right away if you get any of these symptoms during your SOLIRIS infusion: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, and feel faint or pass out. If you have an infusion-related reaction to SOLIRIS, your doctor may need to infuse SOLIRIS more slowly, or stop SOLIRIS.

The most common side effects in people with PNH treated with SOLIRIS include: headache, pain or swelling of your nose or throat (nasopharyngitis), back pain, and nausea.

The most common side effects in people with aHUS treated with SOLIRIS include: headache, diarrhea, high blood pressure (hypertension), common cold (upper respiratory infection), stomach-area (abdominal) pain, vomiting, pain or swelling of your nose or throat (nasopharyngitis), low red blood cell count (anemia), cough, swelling of legs or feet (peripheral edema), nausea, urinary tract infections, and fever.

The most common side effects in people with gMG treated with SOLIRIS include: muscle and joint (musculoskeletal) pain.

The most common side effects in people with NMOSD treated with SOLIRIS include: common cold (upper respiratory infection), pain or swelling of your nose or throat (nasopharyngitis), diarrhea, back pain, dizziness, flu like symptoms (influenza) including fever, headache, tiredness, cough, sore throat, and body aches, joint pain (arthralgia), throat irritation (pharyngitis), and bruising (contusion).

Tell your doctor about any side effect that bothers you or that does not go away. These are not all the possible side effects of SOLIRIS. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch, or call 1-800-FDA-1088.

Please see the accompanying full Prescribing Information and Medication Guide for SOLIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections.