On April 30, 2020 Perrigo Company plc (NYSE; TASE: PRGO) reported financial results for the first quarter ended March 28, 2020 (Press release, Perrigo Company, APR 30, 2020, View Source [SID1234556848]).

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President and CEO Murray S. Kessler commented, "During these unprecedented times, I am honored to be surrounded by our more than 11,000 dedicated employees who are making tremendous sacrifices to help ensure that our essential products remain available to consumers and patients who need them. Our top priorities remain our employees’ well-being and business continuity, while at the same time supporting the communities where we live and work."

Kessler continued, "Thanks to these courageous employees, we were able to report another quarter of strong growth across all business segments with robust sales and profitability above expectations. While we started the year off with similarly strong trends that we experienced in the fourth quarter of 2019, our business surged significantly in March in response to the global demand created by the COVID-19 pandemic. Worth noting, not only did our team keep the business running, they also made significant further progress on our Consumer Self-Care transformation. Perrigo is clearly well-positioned for a ‘New-Normal’ future, which will need Quality, Affordable Self-Care Products more than ever before."

First Quarter Financial Highlights

Consolidated first quarter net sales were $1.3 billion, an increase of 14.2% compared to the prior year quarter. Excluding exited businesses(1) and the impact of currency, net sales increased 17.6%.
Worldwide Consumer first quarter net sales increased 16.2% compared to the prior year quarter. Excluding exited businesses and the impact of currency, Worldwide Consumer net sales were 20.7% higher year-over-year.
Consumer Self-Care Americas ("CSCA") achieved record first quarter net sales of $701 million, or growth of 20.4% versus the prior year quarter highlighted by 15.0% organic(2) growth; Consumer Self-Care International ("CSCI") first quarter net sales grew 9.1% versus the prior year quarter highlighted by 8.1% organic growth.
Reported diluted EPS for the first quarter of 2020 was $0.77 per diluted share as compared to EPS of $0.47 in the prior year quarter.
Adjusted diluted EPS for the first quarter of 2020 increased 6.5% to $1.14 as compared to $1.07 per diluted share in the prior year quarter.
See attached Appendix for reconciliation of adjusted (non-GAAP) to reported (GAAP) financial measures.

(1) Exited businesses excludes $20 million from the divested animal health business in the prior year period, which was previously included in the Consumer Self-Care Americas segment, and $4 million from the divested Canoderm prescription product in the prior year period, which was previously included in the Consumer Self-Care International segment. Full year 2019 net sales of Canoderm were $13 million and adjusted operating income was $8 million.

(2) Organic net sales growth excludes the 2019 acquisition of Ranir, exited businesses and the impact of currency.

Refer to Tables I – IV at the end of this press release for a reconciliation of non-GAAP adjustments to the current year and prior year periods and additional non-GAAP information. The Company’s reported results are included in the attached Condensed Consolidated Statements of Operations, Balance Sheets and Statements of Cash Flows.

First Quarter 2020 Consolidated Results Versus First Quarter 2019

Consolidated net sales for the first quarter of calendar year 2020 increased 14.2% to $1.3 billion. Net sales increased 17.6% compared to the first quarter 2019, excluding exited businesses of $23 million and unfavorable currency movements of $13 million. This was driven by strong consumer demand for Worldwide Consumer products due to higher illnesses and higher allergens, new products including the launch of generic albuterol sulfate, and the addition of Ranir. These drivers were positively impacted by a surge in demand for certain products due to consumer and customer behavior surrounding the COVID-19 pandemic, which added an estimated $90 million to $110 million in net sales to Perrigo in the quarter. These gains were partially offset by pricing pressure, specifically in the Prescription Pharmaceuticals ("RX") segment, and $11 million in discontinued products. Organic net sales were up 11.0%.

Reported net income was $106 million, or $0.77 per diluted share, versus net income of $64 million, or $0.47 per diluted share in the prior year period. Excluding certain charges as outlined in Table I, first quarter 2020 adjusted net income was $157 million, or $1.14 per diluted share, versus $146 million, or $1.07 per diluted share, for the same period last year. The adjusted diluted EPS increase was due primarily to strong growth across the Worldwide Consumer businesses and the addition of Ranir. These were partially offset by RX business price erosion on testosterone gel 1.62%, which launched in the prior year with 180-day market exclusivity, exited businesses and a 200 basis points increase in the adjusted effective tax rate equating to $0.03 per share.

Worldwide Consumer Self-Care First Quarter 2020 Results Versus First Quarter 2019

Worldwide Consumer is comprised of the CSCA segment, CSCI segment and Corporate.

Worldwide Consumer Self-Care delivered record first quarter net sales of $1.1 billion, an increase of 16.2%. Net sales excluding $23 million in exited businesses and $14 million from the impact of currency increased 20.7%. Organic net sales were up 12.3%.

First quarter reported gross profit margin was 36.5%. Adjusted gross profit margin of 38.4%, was 220 basis points lower year-over-year due to 1) higher growth of lower margin store brand products, partially driven by the COVID-19 pandemic, 2) lower operational efficiencies due primarily to a carryover impact from 2019 and Company prioritization of products most needed by society during the COVID-19 pandemic, 3) the addition of Ranir oral self-care products, which have a lower gross margin profile than the existing portfolio, and 4) the impact from exited businesses. These more than offset the margin contribution of new products and positive pricing.

Reported operating margin was 8.7%. Adjusted operating margin was 14.0%, or 100 basis points higher year-over-year due primarily to operating leverage on gross margin flow-through, savings from Project Momentum and the addition of Ranir, which had a relatively higher operating margin profile than the existing portfolio in the quarter. These were partially offset by higher employee compensation costs, including a $4 million special bonus to front-line employees.

CSCA First Quarter 2020 Results Versus First Quarter 2019

Consumer Self-Care Americas achieved record first quarter net sales of $701 million, an increase of 20.4%, and included $55 million in net sales attributable to Ranir. Excluding the exited animal health business, CSCA net sales increased 24.8%. Organic net sales were up 15.0%.

The OTC business delivered solid net sales growth driven by 1) overall OTC category growth and increased demand related to COVID-19, 2) continued robust growth in e-commerce, 3) Perrigo market share gains from store brand competitors due to greater consumer purchases of existing and new products, and 4) increased store brand penetration market-wide versus national brands of 60 basis points according to IRI MULO data, for the 13-weeks ending March 22, 2020.

Growth in the nutrition business was driven by 1) infant formula category growth and increased demand related to COVID-19, 2) the December 2019 store brand infant formula launch at a major retailer, and 3) growth in customer e-commerce activities. These drivers were partially offset by lower contract pack sales, which were due to planned contract production and sales that took place in the fourth quarter 2019.

First quarter reported gross margin was 30.8%. Adjusted gross margin of 31.4% was 110 basis points lower than the prior year due primarily to 1) lower operational efficiencies related to a carryover impact from 2019 and Company prioritization of products most needed by society during the COVID-19 pandemic, and 2) the exited animal health business, which had a relatively higher gross margin. These were partially offset by favorable product mix and the addition of Ranir.

Reported operating margin was 17.8%. Adjusted operating margin increased 130 basis points to 19.6%, driven by operating leverage on gross margin flow-through and lower operating expenses resulting from Project Momentum savings.

CSCI First Quarter 2020 Results Versus First Quarter 2019

Consumer Self-Care International net sales increased 9.1% to $383 million. Excluding unfavorable currency movements of $13 million and exited businesses, net sales were higher by 14.1%. Organic net sales grew 8.1%.

Net sales growth was due primarily to 1) brand OTC sales attributed to COVID-19 in the upper respiratory and vitamins, minerals and supplements (VMS) categories, and growth in the U.K. store brand business, 2) strong new product sales of $30 million driven by new launches including XLS-Medical Forte 5 and in the ACO skincare line, and 3) $21 million in net sales from Ranir. These drivers were partially offset by lower net sales of existing products in weight management, which is within the healthy lifestyle category, and lower net sales in France.

Reported gross margin was 47.0%. Adjusted gross margin of 51.4% declined 250 basis points due primarily to the addition of Ranir oral self-care products, which have a relatively lower gross margin than the overall CSCI portfolio. In addition, lower operational efficiencies were partially offset by favorable brand product mix.

Reported operating margin was 6.5%. Adjusted operating margin of 16.7% improved 130 basis points due primarily to operating leverage on gross margin flow-through and lower advertising and promotion expense.

RX First Quarter 2020 Results Versus First Quarter 2019

RX net sales increased 6.5% to $258 million due primarily to new product sales of $58 million led by the launch of generic albuterol sulfate inhalation aerosol. This was partially offset by expected pricing pressure associated with testosterone gel 1.62%, which had 180-day market exclusivity in the first quarter of 2019. Discontinued products, consisting of lower margin distribution products, were $5 million.

Reported gross margin was 34.1% and adjusted gross margin was 42.3%. The 630 basis point decline in adjusted gross margin was due primarily to competitive price pressure and lower operational efficiencies compared to last year. These were partially offset by higher gross profit from the incremental sales of generic albuterol sulfate inhalation aerosol.

Reported operating margin was 20.0%. Adjusted operating margin was 28.7%, or 520 basis points lower due primarily to gross margin flow-through, which was partially offset by lower administrative expenses.

Fiscal 2020 Outlook

Due to the volatility and uncertainty associated with the COVID-19 pandemic and its potential impact on demand, the Company’s ability to manufacture and supply product and overall fluidity of the current environment, amongst other factors, the Company is not providing updated fiscal 2020 guidance at this time.

On April 30, 2020 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported that Janssen-Cilag International NV (Janssen) received a Committee for Medicinal Products for Human Use (CHMP) Positive Opinion from the European Medicines Agency (EMA) recommending approval of a DARZALEX (daratumumab) subcutaneous (SC) formulation for the treatment of adult patients with multiple myeloma in frontline and relapsed/refractory settings (Press release, Halozyme, APR 30, 2020, View Source [SID1234556847]). The CHMP’s Positive Opinion for daratumumab SC formulation applies to all current daratumumab indications including newly diagnosed and transplant-ineligible patients, as well as relapsed or refractory patients.

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The DARZALEX SC formulation utilizes Halozyme’s ENHANZE drug delivery technology and reduces the treatment time from several hours to approximately five minutes with comparable efficacy to intravenous DARZALEX and lower rates of infusion-related reactions.

"We are excited that a subcutaneous formulation of DARZALEX is one step closer to transforming the treatment experience for multiple myeloma patients and physicians in the European Union," said Dr. Helen Torley, president and chief executive officer. "Once available, multiple myeloma patients may benefit from a shorter infusion time when compared with a multi-hour intravenous infusion."

The CHMP opinion is supported by data from Janssen’s Phase 3 COLUMBA study, which investigated subcutaneously administered DARZALEX in comparison to intravenous DARZALEX in patients with relapsed and refractory multiple myeloma, and the Phase 2 PLEIADES study. In the COLUMBA study, subcutaneous DARZALEX, using ENHANZE drug delivery technology, was found to be non-inferior to intravenous DARZALEX with regard to the co-primary endpoints of overall response rate and maximum Ctrough concentration on day 1 of the third treatment cycle prior to dose.

About ENHANZE Technology
Halozyme’s proprietary ENHANZE drug delivery technology is based on its patented recombinant human hyaluronidase enzyme (rHuPH20). rHuPH20 has been shown to remove traditional limitations on the volume of biologics that can be delivered subcutaneously (just under the skin). By using rHuPH20, some biologics and compounds that are administered intravenously may instead be delivered subcutaneously. ENHANZE may also benefit subcutaneous biologics by reducing the need for multiple injections. This delivery has been shown in studies to reduce health care practitioner time required for administration and shorten time for drug administration.

On April 30, 2020 Achieve Life Sciences, Inc. (Nasdaq: ACHV), a clinical-stage pharmaceutical company committed to the global development and commercialization of cytisinicline for smoking cessation and nicotine addiction, reported that it has entered into definitive agreements for a private placement of its securities for gross proceeds of approximately $1.9 million, prior to deducting placement agent commissions and estimated offering expenses (Press release, OncoGenex Pharmaceuticals, APR 30, 2020, View Source [SID1234556846]).

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The private placement will be for up to 5,615,653 units at a price of $0.33 per unit, with each unit consisting of one share of common stock and a warrant to purchase 0.75 shares of common stock. The private placement will close in two tranches based on the execution of definitive documents on April 27, 2020 and April 28, 2020. The warrants for the first closing have an initial per share exercise price of $0.362, and the warrants for the second closing have an initial per share exercise price of $0.366. All warrants will be exercisable beginning on the six-month anniversary of the initial closing of the offering and will have a five-year term. The Company intends to use the proceeds from the private placement to fund clinical research and development, and for general working capital.

Paulson Investment Company, LLC, acted as the exclusive placement agent in connection with this offering.

The Company has additionally agreed to file a registration statement to register the resale of the shares of common stock included in the units and underlying the investor and placement agent warrants within 60 days of the closing of the offering.

The Company offered and sold the securities described above in reliance on Section 4(a)(2) of the Securities Act of 1933, as amended, and Regulation D promulgated thereunder.

About Achieve & Cytisinicline
Tobacco use is currently the leading cause of preventable death and is responsible for more than eight million deaths annually worldwide1. It is estimated that 28.7% of cancer deaths in the U.S. are attributable to cigarette smoking2. Achieve’s focus is to address the global smoking health epidemic through the development and commercialization of cytisinicline.

Cytisinicline is a plant-based alkaloid with a high binding affinity to the nicotinic acetylcholine receptor. It is believed to aid in smoking cessation by interacting with nicotine receptors in the brain by reducing the severity of nicotine withdrawal symptoms and by reducing the reward and satisfaction associated with smoking.

As an approved, branded product in Central and Eastern Europe for more than two decades, it is estimated that over 20 million people have used cytisinicline to help combat nicotine addiction.

On April 30, 2020 Emergent BioSolutions Inc. (NYSE: EBS) reported financial results for the three months ended March 31, 2020 (Press release, Emergent BioSolutions, APR 30, 2020, View Source [SID1234556845]).

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"Emergent is uniquely positioned to respond to the unprecedented challenges of the COVID-19 pandemic," said Robert G. Kramer Sr., president and chief executive officer of Emergent BioSolutions. "We are deploying our decades of experience in vaccines and therapeutics development and manufacturing, our well-established platform technologies, and our significant development and manufacturing capabilities. Our goal is to create multiple innovative solutions to deliver on our commitments to our customers and patients, while continuing to safeguard our employees."

Signed a contract development and manufacturing (CDMO) agreement, valued at $135 million, to be U.S. manufacturing partner of Johnson & Johnson’s lead vaccine candidate for COVID-19. Negotiations continue on a long-term commercial supply agreement for large-scale drug substance manufacturing, anticipated to begin in 2021.

Initiated development of two investigational plasma-derived therapies. COVID-Human Immune Globulin (COVID-HIG), a human plasma-derived product candidate, for which the Company subsequently received $14.5 million in Health and Human Services (HHS) funding, is being developed as a potential treatment for COVID-19 in severe hospitalized and high-risk patients and will be included in at least one of the studies of the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, evaluating potential treatments for COVID-19. COVID-Equine Immune Globulin (COVID-EIG) is also being developed as an equine plasma-derived therapy candidate for potential treatment of severe disease in humans.

Signed a CDMO agreement with Novavax, Inc. to provide development services, drug substance and drug product manufacturing for its experimental vaccine candidate for COVID-19, NVX-CoV2373.

Signed a CDMO agreement with Vaxart, Inc. to provide development services and drug substance manufacturing to produce its experimental oral vaccine candidate for COVID-19.

Q1 2020 AND OTHER RECENT BUSINESS ACCOMPLISHMENTS

Received agreement from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) on the company’s proposed development plan to use Serum Neutralizing Antibodies (SNA) as surrogate endpoint to predict likely clinical benefit of CHIKV VLP, the company’s chikungunya virus virus-like particle (VLP) vaccine candidate, in a Phase 3 safety and immunogenicity study anticipated in late 2020.

Received positive opinion and subsequent approval from EMA of Vaxchora Cholera Vaccine (recombinant, Live, Oral), making it the only single-dose oral cholera vaccine approved across all European Union member states, the UK, and the European Economic Area countries, indicated for active immunization against disease caused by Vibrio cholerae serogroup 01 in adults and children from 6 years of age. Commercial launch is being planned for late 2020.

Signed a CDMO agreement with Novavax, Inc. to provide drug substance manufacturing of NanoFluTM, its seasonal influenza vaccine candidate.

Announced the appointment of Dr. Karen Smith as chief medical officer with responsibility for leading and further establishing Emergent’s global integrated capability in clinical development, medical affairs, and regulatory affairs.

Submitted a data package to the FDA in support of extending the shelf life of NARCAN (naloxone HCl) Nasal Spray from 24 to 36 months, with an expected review by FDA to take approximately six months.

2020 FINANCIAL PERFORMANCE (unaudited)

(I) Quarter Ended March 31, 2020 (Q1)

Revenues

Total Revenues
For Q1 2020, total revenues were $192.5 million, a slight increase over 2019. Total revenues reflect a decline in product sales revenues partially offset by an increase in CDMO and contracts and grants revenues.
Product Sales
For Q1 2020, product sales were $148.2 million, a decrease of $4.8 million or 3% as compared to 2019. The change primarily reflects increases in sales of Anthrax Vaccines and NARCAN Nasal Spray offset by a decrease in ACAM2000, as previously anticipated.

Contract Development and Manufacturing
For Q1 2020, revenue from the Company’s contract development and manufacturing operations was $21.7 million, an increase of $5.8 million or 36% as compared to 2019. The increase primarily reflects increased demand across development services, drug substance and drug product offerings.

Contracts and Grants
For Q1 2020, revenue from the Company’s development-based contracts and grants was $22.6 million, an increase of $0.9 million as compared to 2019. The increase primarily reflects revenues associated with a grant received related to our PC2A (diazepam) auto-injector drug-device product candidate.
Operating Expenses

Cost of Product Sales and Contract Development and Manufacturing
For Q1 2020, cost of product sales and contract manufacturing was $76.9 million, a decrease of $14.9 million or 16% as compared to 2019. The decrease primarily reflects a decrease in sales of ACAM2000 and raxibacumab.
Research and Development (Gross and Net)

For Q1 2020, gross R&D expenses were $42.7 million, a decrease of $3.4 million or 7% as compared to 2019. The decrease primarily reflects a decline of costs associated with the Company’s FLU-IGIV product candidate. During 2019, the Company was incurring costs associated with phase 2 clinical trials for FLU-IGIV.
For Q1 2020, net R&D expense, which reflects investments made in development programs that are not currently funded in whole or in part by third-party partners and is calculated as gross research and development expenses minus contracts and grants revenue, was 20.1 million, a decrease of $4.3 million or 18% as compared to 2019. The decrease is also attributable to a decline of costs associated with the Company’s FLU-IGIV product candidate. The Q1
2020 and Q1 2019 net R&D expense was 12% of adjusted revenue (total revenue less contracts & grants).

Selling, General and Administrative

For Q1 2020, selling, general and administrative expenses were $69.7 million, an increase of $4.3 million or 7% as compared to 2019. The increase primarily reflects additional expenses related to staffing to support the Company’s growth.

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Amortization of Intangible Assets

For Q1 2020, amortization of intangible assets was $14.8 million was consistent with amortization of intangible assets of $14.5 million in Q1 2019.
Income Taxes

For Q1 2020, the income tax benefit in the amount of $8.8 million was consistent with the benefit during the Q1 2019 as a percentage of net loss during the periods.

Net Loss & Adjusted Net Income (Loss)

For Q1 2020, the Company recorded net loss of $12.5 million, or $0.24 per diluted share, versus net loss of $26.0 million, or $0.51 per diluted share, in 2019.

For Q1 2020, the Company recorded adjusted net income of $0.3 million, or $.01 per diluted share, versus adjusted net loss of $5.2 million, or $.10 per diluted share, in 2019. (1)

Adjusted EBITDA

For Q1 2020, the Company recorded adjusted EBITDA of $15.3 million versus $8.4 million in 2019. (1)

2020 FINANCIAL FORECAST

The Company’s financial forecast for 2020 includes the anticipated impact of the following items:

A full year of product sales, including the following ranges for key components of the product portfolio:

NARCAN Nasal Spray: $285 million – $315 million;

Anthrax Vaccines: $270 million – $300 million;

ACAM2000: $180 million – $200 million;

Contract development and manufacturing revenue of $125 million – $145 million;

Deliveries of raxibacumab to the Strategic National Stockpile (SNS) under the anticipated follow-on procurement contract with HHS;

Domestic and international sales of the other medical countermeasures that comprise Other Product sales;

Continued improvement of gross margin (a combination of product sales and CDMO services) in a range of 200 – 400 basis points annually, driven by improved product mix;

Continued investment in internally funded development projects most notably the anticipated Phase 3 studies for the CHIKV VLP and FLU-IGIV product candidates as well as the Phase 1/2 study for COVID-EIG, among other R&D projects.

Emergent has assessed the risks to its business associated with the COVID-19 pandemic and has adopted measures to mitigate those risks as they are understood today, and accordingly is providing this outlook for 2020. Despite the lack of expected material disruption to the company’s business, the management team continues to assess the business and operational implications associated with the pandemic and market conditions on its employees, patients and customers.

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The outlook for 2020 does not include estimates for potential new corporate development or other M&A transactions.

Q2 2020 REVENUE FORECAST

For Q2 2020, the Company forecast for total revenues is $270 million – $300 million.

FOOTNOTES

(1) See "Reconciliation of Net Loss to Adjusted Net Loss and Adjusted EBITDA" for a definition of terms and the reconciliation tables.

CONFERENCE CALL, PRESENTATION SUPPLEMENT, AND WEBCAST INFORMATION
Company management will host a conference call at 5:00 pm (Eastern Time) today, April 30, 2020, to discuss these financial results. The conference call and presentation supplement can be accessed from the Company’s website or through the following:

Live Teleconference Information:
Dial in: [US] (855) 766-6521; [International] (262) 912-6157
Conference ID: 3784302

Live Webcast Information:
Visit View Source for the live webcast feed.

A replay of the call can be accessed at www.emergentbiosolutions.com under "Investors."

On April 30, 2020 Bristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion, recommending the approval of Reblozyl (luspatercept) for the treatment of Adult patients with transfusion-dependent anemia due to very low-, low- and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response or are ineligible for erythropoietin-based therapy (Press release, Bristol-Myers Squibb, APR 30, 2020, View Source [SID1234556844]).

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Adult patients with transfusion-dependent anemia associated with beta thalassemia.
This CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, Reblozyl would be the first erythroid maturation agent approved in the EU, representing a new class of therapy for eligible patients. The safety and efficacy results provided in the application are from the pivotal Phase 3 MEDALIST and BELIEVE studies, evaluating the ability of Reblozyl to effectively address anemia associated with MDS and beta thalassemia, respectively.

"Patients with myelodysplastic syndromes who experience anemia have limited treatment options, and some have been shown to not respond to available erythropoietin-based therapies," said Uwe Platzbecker, M.D., Head of Clinic and Policlinic for Hematology and Cell Therapy, Leipzig University Hospital and lead investigator of the MEDALIST study. "If approved, the introduction of a new class of therapy in Reblozyl could provide a promising option to help relieve patients from the burden of regular transfusions to manage their disease."

"Today’s positive CHMP opinion of Reblozyl is an important milestone for adult beta thalassemia patients in the EU who have limited treatment options to address anemia, a serious consequence of the disease," said Maria Domenica Cappellini, M.D., Professor of Medicine, University of Milan, Fondazione IRCCS Ca Granda and lead investigator of the BELIEVE study. "Reblozyl has the potential to significantly decrease the number of red blood cell transfusions patients need."

"This decision by the CHMP is an important step towards making this first-in-class therapy an option for eligible patients with anemia due to beta thalassemia or myelodysplastic syndromes," said Diane McDowell, M.D., vice president, Hematology Global Medical Affairs, Bristol Myers Squibb. "We, and our partners at Acceleron, look forward to the opportunity to make this treatment option available in the EU and are extremely appreciative of the patients, families and individuals who continue to help us progress important research in a range of serious diseases."

About MEDALIST

MEDALIST is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of luspatercept plus best supportive care (BSC) versus placebo plus BSC in adults with IPSS-R-defined very low-, low- or intermediate-risk non-del(5q) myelodysplastic syndromes (MDS). All patients were red blood cell (RBC) transfusion-dependent and were either refractory or intolerant to prior erythropoiesis stimulating agent (ESA) therapy, or were ESA naïve and unlikely to respond due to endogenous serum erythropoietin levels of ≥ 200 U/L, and had no prior treatment with disease modifying agents. Results of the MEDALIST trial were first presented during the Plenary Session of the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and were selected for the Best of ASH (Free ASH Whitepaper). The New England Journal of Medicine published the MEDALIST trial results in January 2020.

About MDS

MDS are a group of closely related blood cancers characterized by ineffective production of healthy red blood cells, white blood cells and platelets, which can lead to anemia and frequent or severe infections. People with MDS who develop anemia often require regular blood transfusions to increase the number of healthy red blood cells in circulation. Frequent transfusions are associated with an increased risk of iron overload, transfusion reactions and infections. There are approximately 50,000 patients with MDS in the EU5 countries.

About BELIEVE

BELIEVE is a Phase 3, randomized, double-blind, placebo-controlled multi-center study comparing luspatercept plus BSC versus placebo plus BSC in adults who require regular RBC transfusions (6-20 RBC units per 24 weeks with no transfusion-free period greater than 35 days during that period) due to beta thalassemia. Results of the BELIEVE trial were first presented at the 2018 ASH (Free ASH Whitepaper) Annual Meeting and selected for the Best of ASH (Free ASH Whitepaper). The New England Journal of Medicine published the BELIEVE trial results in March 2020.

About Beta Thalassemia

Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy RBCs, often leading to severe anemia – a condition that can be debilitating and can lead to more severe complications for patients – as well as other serious health issues. Treatment options for anemia associated with beta thalassemia are limited, consisting mainly of frequent RBC transfusions that have the potential to contribute to iron overload, which can cause serious complications such as organ damage. Across the United States, Germany, France, Italy, Spain and the United Kingdom, there are approximately 17,000 patients with beta thalassemia.

About Reblozyl

Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:

anemia in adult patients with beta thalassemia who require regular red blood cell transfusions, and
anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.

Important Safety Information

WARNINGS AND PRECAUTIONS

Thrombosis/Thromboembolism

In adult patients with beta thalassemia, thromboembolic events (TEE) were reported in 8/223 (3.6%) REBLOZYL-treated patients. TEEs included deep vein thrombosis, pulmonary embolus, portal vein thrombosis, and ischemic stroke. Patients with known risk factors for thromboembolism (splenectomy or concomitant use of hormone replacement therapy) may be at further increased risk of thromboembolic conditions. Consider thromboprophylaxis in patients at increased risk of TEE. Monitor patients for signs and symptoms of thromboembolic events and institute treatment promptly.

Hypertension

Hypertension was reported in 10.7% (61/571) of REBLOZYL-treated patients. Across clinical studies, the incidence of Grade 3 to 4 hypertension ranged from 1.8% to 8.6%. In patients with beta thalassemia with normal baseline blood pressure, 13 (6.2%) patients developed systolic blood pressure (SBP) ≥130 mm Hg and 33 (16.6%) patients developed diastolic blood pressure (DBP) ≥80 mm Hg. In adult patients with MDS with normal baseline blood pressure, 26 (29.9%) patients developed SBP ≥130 mm Hg and 23 (16.4%) patients developed DBP ≥80 mm Hg. Monitor blood pressure prior to each administration. Manage new or exacerbations of preexisting hypertension using anti-hypertensive agents.

Embryo-Fetal Toxicity

REBLOZYL may cause fetal harm when administered to a pregnant woman. REBLOZYL caused increased post-implantation loss, decreased litter size, and an increased incidence of skeletal variations in pregnant rat and rabbit studies. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for at least 3 months after the final dose.

ADVERSE REACTIONS

Beta Thalassemia

Serious adverse reactions occurred in 3.6% of patients on REBLOZYL. Serious adverse reactions occurring in 1% of patients included cerebrovascular accident and deep vein thrombosis. A fatal adverse reaction occurred in 1 patient treated with REBLOZYL who died due to an unconfirmed case of acute myeloid leukemia (AML)
Most common adverse reactions (at least 10% for REBLOZYL and 1% more than placebo) were headache (26% vs 24%), bone pain (20% vs 8%), arthralgia (19% vs 12%), fatigue (14% vs 13%), cough (14% vs 11%), abdominal pain (14% vs 12%), diarrhea (12% vs 10%) and dizziness (11% vs 5%)
Myelodysplastic Syndromes

Grade >3 (≥2%) adverse reactions included fatigue, hypertension, syncope and musculoskeletal pain. A fatal adverse reaction occurred in 5 (2.1%) patients
The most common (≥10%) adverse reactions included fatigue, musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity reactions, hypertension, headache, upper respiratory tract infection, bronchitis, and urinary tract infection
LACTATION

It is not known whether REBLOZYL is excreted into human milk or absorbed systemically after ingestion by a nursing infant. REBLOZYL was detected in milk of lactating rats. When a drug is present in animal milk, it is likely that the drug will be present in human milk. Because many drugs are excreted in human milk, and because of the unknown effects of REBLOZYL in infants, a decision should be made whether to discontinue nursing or to discontinue treatment. Because of the potential for serious adverse reactions in the breastfed child, breastfeeding is not recommended during treatment and for 3 months after the last dose.