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Analyzing CRYO-CELL International (OTCMKTS:CCEL) and Harsco (OTCMKTS:HSC) – Redmond Register

CRYO-CELL International (OTCMKTS:CCEL) and Harsco (NYSE:HSC) are both small-cap medical companies, but which is the better business? We will compare the two companies based on the strength of their valuation, risk, analyst recommendations, profitability, institutional ownership, dividends and earnings.

Valuation and Earnings

This table compares CRYO-CELL International and Harscos revenue, earnings per share (EPS) and valuation.

Analyst Ratings

This is a summary of current ratings and recommmendations for CRYO-CELL International and Harsco, as provided by MarketBeat.com.

Harsco has a consensus price target of $26.67, suggesting a potential upside of 325.99%. Given Harscos higher probable upside, analysts clearly believe Harsco is more favorable than CRYO-CELL International.

Volatility & Risk

CRYO-CELL International has a beta of -0.25, suggesting that its stock price is 125% less volatile than the S&P 500. Comparatively, Harsco has a beta of 2.27, suggesting that its stock price is 127% more volatile than the S&P 500.

Profitability

This table compares CRYO-CELL International and Harscos net margins, return on equity and return on assets.

Institutional & Insider Ownership

0.3% of CRYO-CELL International shares are owned by institutional investors. Comparatively, 92.2% of Harsco shares are owned by institutional investors. 54.0% of CRYO-CELL International shares are owned by company insiders. Comparatively, 1.8% of Harsco shares are owned by company insiders. Strong institutional ownership is an indication that hedge funds, endowments and large money managers believe a company is poised for long-term growth.

Summary

Harsco beats CRYO-CELL International on 8 of the 12 factors compared between the two stocks.

CRYO-CELL International Company Profile

Cryo-Cell International, Inc. engages in the cellular processing and cryogenic cellular storage with a focus on the collection and preservation of umbilical cord blood stem cells for family use. It provides cord tissue service that stores a section of the umbilical cord tissue, a source of mesenchymal stem cells that are used in regenerative medicine to treat a range of conditions, including heart, kidney, ALS, wound healing, and auto-immune diseases. The company also manufactures and sells PrepaCyte CB processing system, a technology used to process umbilical cord blood stem cells. It stores approximately 500,000 cord blood and cord tissue specimens worldwide. The company markets its cord blood stem cell preservation services directly to expectant parents, as well as by distributing information through obstetricians, pediatricians, childbirth educators, certified nurse-midwives, and other related healthcare professionals. Cryo-Cell International, Inc. was founded in 1989 and is headquartered in Oldsmar, Florida.

Harsco Company Profile

Harsco Corporation provides industrial services and engineered products worldwide. The company operates in three segments: Harsco Metals & Minerals, Harsco Industrial, and Harsco Rail. The Harsco Metals & Minerals segment provides on-site services of material logistics, product quality improvement, and resource recovery for iron, steel, and metals manufacturing; and value added environmental solutions for industrial co-products, as well as produces industrial abrasives and roofing granules. The Harsco Industrial segment manufactures and supplies custom-engineered and manufactured air-cooled heat exchangers for the natural gas, natural gas processing, and petrochemical industries; industrial grating products, such as metal bar grating configurations for industrial flooring, and safety and security applications in the energy, paper, chemical, refining, and processing industries. It also offers heat transfer products, such as boilers and water heaters for commercial and institutional applications; and high-security fencing products. The Harsco Rail segment designs and manufactures safety systems for transportation and industrial applications; and equipment, after-market parts, and services for the maintenance, repair, and construction of railway track. It serves private and government-owned railroads, and urban mass transit systems. Harsco Corporation was founded in 1853 and is headquartered in Camp Hill, Pennsylvania.

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Analyzing CRYO-CELL International (OTCMKTS:CCEL) and Harsco (OTCMKTS:HSC) - Redmond Register

15 Good News Stories To Tackle The COVID-19 Sadness – IFLScience

For Earth, bleak times lay ahead. TheCOVID-19 diseaseis known to cause respiratory illness and fever, but some extra symptoms sweeping across the globe right now seem to be stress, fear, and anxiety. To provide some light relief in these dark times, weve collated 15 of our favorite good news stories to remind you that not everything is awful. Hold tight everybody, 2021 will come eventually.

The Super Pink Moon is comingYou might be stuck at home as part of your self-isolation, but luckily the night sky is about to put on quite a show as April sees the return of the Super Pink Moon. Full moons happen every month and were given different names by the Native Americans to map out the year based on significant events that ran in tandem with the occurrence of a full Moon. Aprils is known as the pink moon because it appeared at the same time as pink spring flowers. This Aprils will be a Super Pink Moon as it is the second supermoon of the year, a term used to describe the slightly enlarged appearance of the Moon as its fully illuminated by the Sun due to Earths position between the two. Quarantine or no, if you've got access to a window you should be able to catch sight of this beauty on April 7 and when you do, think of all the other people looking up at the same moon. Self isolation doesn't mean you're alone.

Mice have been cured of diabetesAn astonishing discovery at the Washington University School of Medicine in St. Louis has revealed that human stem cells could be successfully engineered to cure diabetes in mice, offering an avenue of hope for the treatment of this debilitating disease. They used human pluripotent stem cells, cells that have the capacity to become any cell in the body, to create insulin-producing pancreatic beta cells. The engineered stem cells supplemented the diabetic mices inability to produce insulin, curing them of the disease for 9 months to a year before relapse occurred.

Theres a new green fuel in townHydrogen fuel was fast shaping up to be a hopeful route for a zero-emissions means of running things, but its costly production in terms of energy was affecting hopes for it being a sustainable resource. A team in Tokyo has now managed to refine the process to yield 25 times more hydrogen than previous methods all while using thrifty ingredients including light and a specific kind of rust. Combined with all the solar power breakthroughs currently occurring, green energy is on the up.

A crash course in what not to do, according to one Stanford University psychologist.

Babies love baby talkEven if it makes your skin crawl to hear adults cooing over little uns, it turns out babies across the globe are universally partial to baby talk. The news comes fromStanford psychologist Michael Frank who led the largeststudyto date looking at how the different ways adults speak is received by babies across the world. While all babies were fans, older babies liked it best and even showed a preference for baby talk in their native language as they likely recognized it most even if they couldnt speak it yet. The overall winner was oohs and coos, so think twice before scorning your new-parent friends for embarrassing you in public the babies have spoken.

Important change in the winds for HIV treatmentShortly after a UK man became the second person cured of HIV a fantastic breakthrough in the treatment of this once devastating disease theres more good news in the UK as PrEP, a preventative drug that prevents HIV infection, will finally be available nationwide on the NHS having already been made available in Scotland. After a 3-year study involving 20,000 participants, the drug will be made available to those at higher risk of exposure from April. PrEP is already available in the US and you can find PrEP providers near you here.

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Plasters finally take a step towards racial inclusivityMajor UK superstore Tesco has taken the long-awaited step to introduce skin tone diversity into their range of bandaids. Previously, widely available bandaids, or plasters in the UK, have mainly catered to Caucasian individuals and the racial oversight was brought to light by a moving Tweet from Domonique Apollon in April 2019 after he wore a bandaid suitable for his skin tone for the first time. Longtime readers of Malorie Blackman's literary series Noughts and Crosseswill appreciate this poignant detail becoming a reality, as will those watching the current BBC dramatization available to watch via iPlayer in the US (excellent for those self-isolating).

Universal flu vaccine passes integral stageWatchers of the Pandemic documentary on Netflix (we wouldnt recommend catching up now if you missed it) may remember the plight of flu-fighting epidemiologists as the constantly shape-shifting nature of influenza meant strains were annually moving beyond existing vaccinations. Now, a universal vaccine is becoming a reality as for the first time a vaccine, called FLU-v, has been developed that can induce immune responses that last at least six months. Phase I and II of the clinical trial have been approved meaning its safety for use in human subjects and we hotly await what comes next for the groundbreaking vaccine.

Top marks for lights out in dark sky nationSometimes a bit of darkness can be a good thing, and when it comes to nighttime, the tiny South Pacific island of Niue tops the charts. The International Dark-Sky Association (IDA) is a non-profit working to protect our most precious natural spaces from light pollution, and this year chose Niue as the first entire country ever to be accredited as a Dark Sky Place. This classification recognizes responsible lighting policies that preserve the natural darkness of nighttime carrying with it endless benefits for the biological cycles of animals, plants and humans.

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People hating on National Parks created beautiful artIn a glimmering example of you cant please everybody, artist Amber Share decided to take some of the best worst reviews of National Parks in America and turn them intotourism posters, showing that we can still make something funny in the face of people's negativity. You can see the whole collection on her Instagram account @subparparks, but a personal favorite has to be the above magnificent minimization of Yellowstone.

CRISPR may hold the key for curing genetic blindnessSurgeons at Oregon Health & Science Institute have attempted to use gene hacking to cure Leber congenital amaurosis, a genetic condition that leads to the onset of blindness in early childhood. By directly gene editing within the patients eye, researchers hope to ...take people who are essentially blind and make them see," according to researchers.

The Arctic seed vault in Svalbard is thrivingLast month saw an enormous glut of 60,000 seed samples added to the ever-growing collecting in the Svalbard Global Seed Vault. Tucked beneath a mountain in Norway's Svalbard archipelago, the initiative began with hopes to create a Noahs ark for plant diversity to protect our green spaces should a global catastrophe occur up top. The collection now includes 1.05 million seed varieties including the first-ever donation from an indigenous US tribe. Nicknamed the "Doomsday vault", we may need it sooner than thought.

Sea sponges can sneeze, and the footage is amazingThe aah and choo of asneezing sea spongehas been caught on camera for the first time and the recording is hilarious. Stumbled upon almost by accident, the discovery came about while researchers were observing sea cucumbers and sea urchins sniffing the sea floor. The video shows the two-part sneeze of a tulip-shaped sponge as it expands before contracting, expelling particles as it goes. Researchers arent yet sure what the sneezes are in response to. Lets hope its not a case ofthe suds.

Vernal equinox brings early springThe times might be dark but for the Northern hemisphere, the days wont be, as spring arrives on March 19, the earliest date in 124 years. The variation in the date is the result of leap years and daylight savings time. It should be noted this is the astronomical definition of spring, which refers specifically to the position of Earth's orbit in relation to the Sun, so perhaps dont expect to hear a gay little spring song in your garden just yet.

Its possible some dinosaurs could GLOW IN THE DARKA titillating discovery published in the journal Historical Biology recently revealed that some dinosaurs may have glowed in the dark thanks to ultraviolet fluorescing feathers and horns. Many extant bird species are tetrachromats, defined by a fourth cone in their retina that means they can see the UV spectrum. Co-author Jamie Dunning's work on the photoluminescence of puffin beaks under UV light inspired the questions, could dinosaurs have this too? We'd like the answer to be yes, please. The only thing cooler than dinosaurs is glow-in-the-dark dinosaurs.

If you need more positivity in your life right now, take a look at these ingenious social distancing moments from around the world that will restore your faith in humanity.

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15 Good News Stories To Tackle The COVID-19 Sadness - IFLScience

Immatics and Molmed become the subjects of very different deals – Vantage

How do companies seal a deal when executives cannot shake hands? Maybe the management teams of Arya Sciences and Immatics bumped elbows when the latter was bought by the blank cheque company on Tuesday in a deal worth $252m. As for the Japanese group AGCs 240m ($267m) offer for Italys Molmed, presumably execs could not meet in person at all given that Italy is still locked down.

Be that as it may an acquisition and a bid have been arranged somehow, proving that though the Covid-19 pandemic has had a deadening effect on deal-making it has not annihilated it altogether. The interesting aspect is how Immatics and Molmed both cell therapy players, albeit of different kinds will develop from here.

A stark mission

Arya Sciences Acquisition Corp was set up to do what its name suggests: its sole purpose is to effect a merger or similar combination with one or more businesses. It is controlled by the hedge fund Perceptive Advisors, and floated on Nasdaq in October 2018, raising $144m.

Immatics will receive $148m of cash from Aryas trust account, which holds the IPO proceeds plus interest, when the deal closes in the second quarter. Perceptive and other institutional investors, including Redmile and Wellington Partners, have committed a further $104m more in pipe funding.

Arya saysImmatics T-cell receptor-based candidates for solid tumours hold the kind of disruptive potential the investment vehicle was looking for. Immatics has two main product classes, adoptive cell therapies, which use natural or engineered T cells against cancer, and T-cell receptor bispecifics, which bind to tumour-specific peptides and to immunomodulating T-cell surface proteins.

Gaining access to the US capital markets will allow Immatics to advance its projects through the clinic. The group expects topline phase I/II data from three TCR projects and one bispecific by the end of this year.

Going public represents a long-held goal for Immatics. The company was already eyeing the US exchanges two years ago (Why Immatics could soon become the next listed cell therapy player, July 16, 2018).

Long term

Molmed could take a very different route if AGCs bid for it is accepted, becoming a part of a much larger whole. Japans AGC is the largest glass company in the world, but is also active in the fields of ceramics, electronics and chemicals.

AGC considers its life sciences business a strategic priority, and aims to get the units sales above 100bn by 2025. Buying Molmed it is offering 0.518 per Molmed share, at a premium of 110% will allow ACG to move into gene and cell therapies.

It will not get there fast. Molmed withdrew Zalmoxis, designed to reduce the risk or rejection of an imperfectly matched stem cell transplant, from sale in Europe after a confirmatory phase III trial failed last summer. Its next most advanced product is its Car-T project CAR44v6, in a phase I/II trial for acute myeloid leukaemia and multiple myeloma. This looks unlikely to yield data before 2023.

Molmed does have another string to its bow that could appeal to AGC: it is the first company in Europe to have obtained GMP manufacturing authorisation for cell and gene therapies ex vivo, and offers this to other groups. For example, it produces Strimvelis, Orchard Therapeutics gene therapy for the immunodeficiency disorder ADA-SCID.

Closure is odds-on, since Molmeds largest shareholder, Fininvest the holding company of the family of the former Italian prime minister Silvio Berlusconi has agreed to tender its 23% stake. Hopefully, in buying this very specialised company, AGC knows what it is doing.

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Immatics and Molmed become the subjects of very different deals - Vantage

Claudia Rodrigues is removed from the clinic where she was hospitalized because of the coronavirus – Crypto Dictation

The new coronavirus is spreading all over the world and, after arriving in Brazil, has changed the lives of most Brazilians. The government's recommendation is that people stay at home and take all recommended hygiene care, thus preventing the virus from spreading in the country.

The greatest concern of the government is with the people who are part of the group considered at risk, which are the elderly and people who already have some type of chronic disease. These people are the ones most likely to have complications if they become infected with the virus.

Actress Claudia Rodrigues is one of those people who are part of the risk group and who must have redoubled health care so that it does not become contaminated with covid-19. The actress has been battling multiple sclerosis for 20 years, which is an autoimmune disease that affects the central nervous system.

The actress has already been hospitalized several times in a serious condition in times of crisis of multiple sclerosis and at the moment was hospitalized in a clinic in the state of So Paulo, following the autoimmune treatment.

Due to government recommendations and fearing contamination by covid-19, the actress was taken home.

The treatment that actress Claudia Rodrigues performed at the clinic in So Paulo is very delicate, due to her health condition in relation to multiple sclerosis, and for that reason, extra care is needed with the artist, as she already has low immunity due to disease.

Due to the situation faced by the actress, her doctors and family members chose to redouble the artist's health care and for this very reason, they decided that it was better that she be taken home and that she should remain in isolation at her residence.

Adriane Bonato, who is Claudia Rodrigues' businesswoman, revealed that the actress is at her home and that she is performing exercises on the spot. The businesswoman reaffirmed that the artist has low immunity due to having undergone stem cell transplantation and that, for this very reason, she runs a very high risk in relation to the new virus.

According to Adriane, Claudia Rodrigues will remain at home for the next 30 days, preventing herself from being infected by the new coronavirus.

Like most Brazilians, many Celebrity are at home complying with government resolutions on social isolation to prevent the mass spread of the new coronavirus. Many artists are taking advantage of the large number of followers in their profiles to try to make the population aware of the importance of following the recommendations of the Ministry of Health and especially the recommendation to be quarantined at home.

Big names like Luciano Huck, Ana Maria Braga and many others have posted videos asking Brazilians for empathy and to remain in social isolation. The artists Preta Gil and Fernanda Paes Leme, who were diagnosed with the disease, are sharing their experiences with the new coronavirus. They are keeping followers informed about how the isolation is going and the symptoms they are feeling about the coronavirus.

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Claudia Rodrigues is removed from the clinic where she was hospitalized because of the coronavirus - Crypto Dictation

Stem Cell Therapy Market 2020 Demand with Global Forecast by Top Leading Players: Osiris Therapeutics, Medipost Co., Anterogen Co., Pharmicell Co.,…

New Jersey, United States: A qualitative research study accomplished by Verified Market Research titled 2020-2026 Global and Regional Stem Cell Therapy Market: Industry Production, Sales and Consumption Status and Prospects Professional Market Research Report is the most up to date report which comprises the latest trends that influence the market competition in the forecast period from 2020 to 2026. The report presents different market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. Primarily, the report introduces market demands and the present position of the Stem Cell Therapy market.The report completes the value chain and downstream and upstream essentials.

Global Stem Cell TherapyMarketwas valued at USD 86.62 million in 2016 and is projected to reach USD 221.03million by 2025, growing at a CAGR of 10.97% from 2017 to 2025.

Our expert analyst has categorized the market into product type, application/end-user, and geography. All the segments are analyzed based on their market share, growth rate, and growth potential. The growth potential, market share, size, and prospects of each segment and sub-segment are portrayed in the report. This thorough evaluation of the segments would help the players to focus on revenue-generating areas of the Stem Cell Therapy Market.

A number of leading manufacturers mention in the Stem Cell Therapy Market research report are focusing on expanding operations in regions, as they exhibit potential business opportunities. The Stem Cell Therapy Market report classifies the market dynamics and trends in the global and regional market considering several aspects including technology, supplies, capacity, production, profit, and price.

Stem Cell Therapy Market: Research Methodology

1. Primary Research:

2. Secondary Research:

During our Secondary research, we collect information from different sources such as databases, regulatory bodies, gold and silver-standard websites, articles by recognized authors, certified publications, white papers, investor presentations and press releases of companies, and annual reports.

Data collection module is used for data collection and analysis of the base year. The market data is analyzed and estimated using statistical models and systematic market. The main research methodology used for the preparation of reports, including data mining, primary (industry experts) validation and top-down analysis, market overview and guidance, the company market share analysis, measurement standards, and analysis of the stock sellers.

Vendor Competitive Analysis:

The report focuses on the strategies considered by the market participants to gain a major share in the Stem Cell Therapy market. Through this, the competitors will get an overview of the competitive landscape so they can make business decisions. Leading players working in the global market are analyzed with their company information, product profile, product specification, picture, capacity, production, price, cost, global investment plans, and supply-demand scenarios.

Stem Cell Therapy Market Regional Coverage

The Middle East and Africa (GCC Countries and Egypt)North America (the United States, Mexico, and Canada)South America (Brazil etc.)Europe (Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

Ask For Discount @ https://www.verifiedmarketresearch.com/ask-for-discount/?rid=24113&utm_source=PN24&utm_medium=002

Table of Content

1 Introduction of Stem Cell Therapy Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Stem Cell Therapy Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Stem Cell Therapy Market, By Deployment Model

5.1 Overview

6 Stem Cell Therapy Market, By Solution6.1 Overview

7 Stem Cell Therapy Market, By Vertical

7.1 Overview

8 Stem Cell Therapy Market, By Geography8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Stem Cell Therapy Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

Complete Report is Available @ https://www.verifiedmarketresearch.com/product/Stem-Cell-Therapy-Market/?utm_source=PN24&utm_medium=002

We also offer customization on reports based on specific client requirement:

1- Free country level analysis for any 5 countries of your choice.

2- Free Competitive analysis of any market players.

3- Free 40 analyst hours to cover any other data points

About us:

Verified market research partners with the customer and offer an insight into strategic and growth analyzes; Data necessary to achieve corporate goals and objectives. Our core values are trust, integrity and authenticity for our customers.

Analysts with a high level of expertise in data collection and governance use industrial techniques to collect and analyze data in all phases. Our analysts are trained to combine modern data collection techniques, superior research methodology, expertise and years of collective experience to produce informative and accurate research reports.

Contact us:

Mr. Edwyne FernandesCall: +1 (650) 781 4080Email: [emailprotected]

Tags: Stem Cell Therapy Market Size, Stem Cell Therapy Market Trends, Stem Cell Therapy Market Forecast, Stem Cell Therapy Market Growth, Stem Cell Therapy Market Analysis, Stem Cell Therapy Market Business Opportunities and Stem Cell Therapy Market Outlook

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Stem Cell Therapy Market 2020 Demand with Global Forecast by Top Leading Players: Osiris Therapeutics, Medipost Co., Anterogen Co., Pharmicell Co.,...

Forty Seven, Inc. Reports Fourth Quarter and Full Year 2019 Financial Results and Recent Business Highlights – BioSpace

MENLO PARK, Calif., March 20, 2020 (GLOBE NEWSWIRE) -- Forty Seven Inc., (Nasdaq:FTSV), a clinical-stage, immuno-oncology company focused on developing therapies to activate macrophages in the fight against cancer, today reported financial results for the fourth quarter and full year ended December 31, 2019 and provided a business update.

In 2019, Forty Seven transformed into a multi-asset, late-stage development company with clear paths to registration in two distinct, underserved patient populations. In parallel, we entered into several new partnerships designed to accelerate the development of magrolimab and FSI-174, and allow us to evaluate both compounds more rapidly across a range of indications and combination paradigms, said Mark McCamish, M.D., Ph.D., President and Chief Executive Officer of Forty Seven. Following the recently announced acquisition by Gilead, and with the benefit of their resources and capabilities, we are even better positioned to build on this momentum and deliver on our foundational vision of developing novel immunotherapies that help patients defeat their cancers.

Dr. McCamish continued, Like so many others, we are closely monitoring COVID-19, and have recently instituted a number of proactive measures to mitigate the spread of the virus and protect the safety, health and well-being of the patients, families and healthcare professionals involved in our clinical development programs, as well as our employees. While we are working diligently to limit the impact of COVID-19 on our ongoing clinical trials, we, together with our contract research organization, decided to delay the initiation of our Phase 1 trial of FSI-174 in healthy volunteers in order to support physicians and hospitals in devoting their resources to treating COVID-19 patients, and avoid exposing healthy volunteers to unnecessary risk. We will continue to evaluate the pandemic and expect to re-visit the timing of potential trial initiation in the second quarter.

Fourth Quarter and Recent Business Highlights:

Magrolimab Clinical Programs:Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)

Diffuse Large B Cell Lymphoma (DLBCL)

Solid Tumors

FSI-174:

FSI-189:

Corporate:

Fourth Quarter and Full Year 2019 Financial Results:

About Forty Seven, Inc.Forty Seven, Inc.is a clinical-stage immuno-oncology company that is developing therapies targeting cancer immune evasion pathways and specific cell targeting approaches based on technology licensed fromStanford University. Forty Sevens lead program, magrolimab, is a monoclonal antibody against the CD47 receptor, a dont eat me signal that cancer cells commandeer to avoid being ingested by macrophages. This antibody is currently being evaluated in multiple clinical studies in patients with myelodysplastic syndrome, acute myeloid leukemia and non-Hodgkins lymphoma. In March 2020, Forty Seven entered into a definitive agreement to be acquired by Gilead Sciences, Inc., which is expected to close during the second quarter of 2020.

Additional Information and Where to Find It

This communication is for informational purposes only and is neither an offer to purchase nor a solicitation of an offer to sell shares of Forty Seven, nor is it a substitute for any tender offer materials that Gilead, its acquisition company or Forty Seven has or will file with the SEC. A solicitation and an offer to buy shares of Forty Seven will be made only pursuant to an offer to purchase and related materials that Gilead has filed with the SEC. At the time the tender offer was commenced, Gilead filed a Tender Offer Statement on Schedule TO with the SEC, and Forty Seven filed a Solicitation/Recommendation Statement on Schedule 14D-9 with the SEC with respect to the tender offer. FORTY SEVENS STOCKHOLDERS AND OTHER INVESTORS ARE URGED TO READ THE TENDER OFFER MATERIALS (INCLUDING AN OFFER TO PURCHASE, A RELATED LETTER OF TRANSMITTAL AND CERTAIN OTHER TENDER OFFER DOCUMENTS) AND THE SOLICITATION/RECOMMENDATION STATEMENT BECAUSE THEY CONTAIN IMPORTANT INFORMATION WHICH SHOULD BE READ CAREFULLY BEFORE ANY DECISION IS MADE WITH RESPECT TO THE TENDER OFFER. The Offer to Purchase, the related Letter of Transmittal and certain other tender offer documents, as well as the Solicitation/Recommendation Statement, has been sent to all stockholders of Forty Seven at no expense to them. The Tender Offer Statement and the Solicitation/Recommendation Statement are available for free at the SEC's web site at http://www.sec.gov. Additional copies may be obtained for free by contacting Gilead or Forty Seven. Free copies of these materials and certain other offering documents will be made available by Gilead by mail to Gilead Sciences, Inc., 333 Lakeside Drive, Foster City, CA 94404, attention: Investor Relations, by phone at 1-800-GILEAD-5 or 1-650-574-3000, or by directing requests for such materials to the information agent for the offer, which will be named in the Tender Offer Statement. Copies of the documents filed with the SEC by Forty Seven will be available free of charge under the Investors section of Forty Sevens internet website at ir.fortyseveninc.com.

Forward-Looking Statements:

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as expect, potential, plans, will, believe, and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. These statements include those related to the timing and outcome of results from the Phase 1b trial evaluating magrolimab in combination with azacitidine for the treatment of MDS and AML, the potentially-registration enabling clinical development program for magrolimab in higher-risk MDS, the single-arm, registration enabling trial evaluating the combination of magrolimab and rituximab in heavily pre-treated relapsed or refractory DLBCL patients, and other ongoing trials of 5F9 for the treatment of ovarian and colorectal cancer; the timing of and quality of results from investigational new drug-application enabling studies for FSI-189 and FSI-174 and their respective potential for approval by the FDA; the timing and success of research and development plans for Rockets and Forty Sevens respective platforms, product candidates and collaboration; the timing and success of research and development plans for bluebirds and Forty Sevens respective platforms, product candidates and collaboration; the business combination with Gilead and related matter; post-closing operations and the outlook for the companies respective businesses, including, without limitation, the ability of Gilead to advance Forty Sevens product pipeline, including magrolimab, FSI-174 and FSI-189; filings and approvals relating to the transaction; the expected timing of the completion of the transaction; the ability to complete the transaction considering the various closing conditions; difficulties or unanticipated expenses in connection with integrating the companies; Forty Sevens ability to fund its clinical programs and the sufficiency of its cash and short-term investments, and Forty Sevens financial outlook; and any assumptions underlying any of the foregoing.

Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Risks and uncertainties that could cause the actual results to differ from expectations contemplated by such forward-looking statements include: the potential product candidates that Forty Seven develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; such product candidates may not be beneficial to patients or successfully commercialized; uncertainties as to the timing of the business combination with Gilead; the possibility that various closing conditions for the business combination may not be satisfied or waived, including that a governmental entity may prohibit, delay or refuse to grant approval for the consummation of the transaction; the effects of the business combination on relationships with employees, other business partners or governmental entities; the difficulty of predicting the timing or outcome of FDA approvals or actions, if any; the impact of competitive products and pricing; other business effects, including the effects of industry, economic or political conditions outside of the companies control; transaction costs; actual or contingent liabilities; and other risks and uncertainties detailed from time to time in the companies periodic reports filed with the U.S. Securities and Exchange Commission (the SEC), including current reports on Form 8-K, quarterly reports on Form 10-Q and annual reports on Form 10-K, as well as the Schedule 14D-9 filed by Forty Seven and the Schedule TO and related tender offer documents filed by Gilead and Toro Merger Sub, Inc., a wholly owned subsidiary of Gilead. All forward-looking statements are based on information currently available to Gilead and Forty Seven, and Gilead and Forty Seven assume no obligation and disclaim any intent to update any such forward-looking statements.

For more information please visit http://www.fortyseveninc.com or contactinfo@fortyseveninc.com.

For journalist enquiries please contact Sarah Plumridge atfortyseven@hdmz.comor phone (312) 506-5218.

For investor enquiries please contact Hannah Deresiewicz at Stern Investor Relations Inc. athannah.deresiewicz@sternir.comor phone (212) 362-1200.

Forty Seven Inc.Statements of Operations and Comprehensive Loss Data(In thousands, except share and per share data)

Forty Seven Inc.Selected Balance Sheet Data(in thousands)

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Forty Seven, Inc. Reports Fourth Quarter and Full Year 2019 Financial Results and Recent Business Highlights - BioSpace

Platelet Rich Plasma and Stem Cell Alopecia Treatment Market : Drivers, Restraints, Opportunities, and Threats (2019-2025) – Packaging News 24

Global Platelet Rich Plasma and Stem Cell Alopecia Treatment Market Report 2019 Market Size, Share, Price, Trend and Forecast is a professional and in-depth study on the current state of the global Platelet Rich Plasma and Stem Cell Alopecia Treatment industry.

The report also covers segment data, including: type segment, industry segment, channel segment etc. cover different segment market size, both volume and value. Also cover different industries clients information, which is very important for the manufacturers.

There are 4 key segments covered in this report: competitor segment, product type segment, end use/application segment and geography segment.

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For competitor segment, the report includes global key players of Platelet Rich Plasma and Stem Cell Alopecia Treatment as well as some small players.

Companies Mentioned in the Report

The report also profiles major players operating in the global platelet rich plasma & stem cell alopecia treatment market based on various attributes, such as company overview, financial overview, pipeline portfolio, product portfolio, business strategies, and recent developments. The players covered in the report include Kerastem, Eclipse, Regen Lab SA, Stemcell Technologies, Inc., RepliCel Life Sciences, Histogen, Inc., and Glofinn Oy.

The global platelet rich plasma & stem cell alopecia treatment market has been segmented as below:

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Important Key questions answered in Platelet Rich Plasma and Stem Cell Alopecia Treatment market report:

What will the market growth rate, Overview, and Analysis by Type of Platelet Rich Plasma and Stem Cell Alopecia Treatment in 2024?

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What is Dynamics, This Overview Includes Analysis of Scope and price analysis of top Manufacturers Profiles?

Who Are Opportunities, Risk and Driving Force of Platelet Rich Plasma and Stem Cell Alopecia Treatment market? Knows Upstream Raw Materials Sourcing and Downstream Buyers.

Who are the key manufacturers in space? Business Overview by Type, Applications, Gross Margin, and Market Share

What are the opportunities and threats faced by manufacturers in the global market?

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The content of the study subjects, includes a total of 15 chapters:

Chapter 1, to describe Platelet Rich Plasma and Stem Cell Alopecia Treatment product scope, market overview, market opportunities, market driving force and market risks.

Chapter 2, to profile the top manufacturers of Platelet Rich Plasma and Stem Cell Alopecia Treatment , with price, sales, revenue and global market share of Platelet Rich Plasma and Stem Cell Alopecia Treatment in 2019 and 2015.

Chapter 3, the Platelet Rich Plasma and Stem Cell Alopecia Treatment competitive situation, sales, revenue and global market share of top manufacturers are analyzed emphatically by landscape contrast.

Chapter 4, the Platelet Rich Plasma and Stem Cell Alopecia Treatment breakdown data are shown at the regional level, to show the sales, revenue and growth by regions, from 2019 to 2025.

Chapter 5, 6, 7, 8 and 9, to break the sales data at the country level, with sales, revenue and market share for key countries in the world, from 2019 to 2025.

Chapter 10 and 11, to segment the sales by type and application, with sales market share and growth rate by type, application, from 2019 to 2025.

Chapter 12, Platelet Rich Plasma and Stem Cell Alopecia Treatment market forecast, by regions, type and application, with sales and revenue, from 2019 to 2025.

Chapter 13, 14 and 15, to describe Platelet Rich Plasma and Stem Cell Alopecia Treatment sales channel, distributors, customers, research findings and conclusion, appendix and data source.

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Platelet Rich Plasma and Stem Cell Alopecia Treatment Market : Drivers, Restraints, Opportunities, and Threats (2019-2025) - Packaging News 24

Disruptions in Cancer Care in the Era of COVID-19 – Medscape

Editor's note: Find the latest COVID-19 news and guidance in Medscape's Coronavirus Resource Center.

Even in the midst of the COVID-19 pandemic, cancer care must go on, but changes may need to be made in the way some care is delivered.

"We're headed for a time when there will be significant disruptions in the care of patients with cancer," said Len Lichtenfeld, MD, deputy chief medical officer of the American Cancer Society (ACS), in a statement. "For some it may be as straightforward as a delay in having elective surgery. For others it may be delaying preventive care or adjuvant chemotherapy that's meant to keep cancer from returning or rescheduling appointments."

Lichtenfeld emphasized that cancer care teams are going to do the best they can to deliver care to those most in need. However, even in those circumstances, it won't be life as usual. "It will require patience on everyone's part as we go through this pandemic," he said.

"The way we treat cancer over the next few months will change enormously," writes a British oncologist in an article published in the Guardian.

"As oncologists, we will have to find a tenuous balance between undertreating people with cancer, resulting in more deaths from the disease in the medium to long term, and increasing deaths from COVID-19 in a vulnerable patient population. Alongside our patients we will have to make difficult decisions regarding treatments, with only low-quality evidence to guide us," writes Lucy Gossage, MD, consultant oncologist at Nottingham University Hospital, UK.

The evidence to date (from reports from China in Lancet Oncology) suggests that people with cancer have a significantly higher risk of severe illness resulting in intensive care admissions or death when infected with COVID-19, particularly if they recently had chemotherapy or surgery.

"Many of the oncology treatments we currently use, especially those given after surgery to reduce risk of cancer recurrence, have relatively small benefits," she writes.

"In the current climate, the balance of offering these treatments may shift; a small reduction in risk of cancer recurrence over the next 5 years may be outweighed by the potential for a short-term increase in risk of death from COVID-19. In the long term, more people's cancer will return if we aren't able to offer these treatments," she adds.

One thing that can go on the back burner for now is routine cancer screening, whichcan bepostponed for now in order to conserve health system resources and reduce contact with healthcare facilities, says the ACS.

"Patients seeking routine cancer screenings should delay those until further notice," said Lichtenfeld. "While timely screening is important, the need to prevent the spread of coronavirus and to reduce the strain on the medical system is more important right now."

But as soon as restrictions to slow the spread of COVID-19 are lifted and routine visits to health facilities are safe, regular screening tests should be rescheduled.

The American Society of Clinical Oncology (ASCO) has issued new guidance on caring for patients with cancer during the COVID-19 outbreak.

First and foremost, ASCO encourages providers, facilities, and anyone caring for patients with cancer to follow the existing guidelines from the Center for Disease Control and Prevention (CDC) when possible.

ASCO highlights the CDC's general recommendation for healthcare facilities that suggests "elective surgeries" at inpatient facilities be rescheduled if possible, which has also been recommended by the American College of Surgeons.

However, in many cases, cancer surgery is not elective but essential, it points out. So this is largely an individual determination that clinicians and patients will need to make, taking into account the potential harms of delaying needed cancer-related surgery.

Systemic treatments, including chemotherapy and immunotherapy, leave cancer patients vulnerable to infection, but ASCO says there is no direct evidence to support changes in regimens during the pandemic. Therefore, routinely stopping anticancer or immunosuppressive therapy is not recommended, as the balance of potential harms that may result from delaying or interrupting treatment versus the potential benefits of possibly preventing or delaying COVID-19 infection remains very unclear.

Clinical decisions must be individualized, ASCO emphasized, and suggestedthe following practice points be considered:

For patients already in deep remission who are receiving maintenance therapy, stopping treatment may be an option.

Some patients may be able to switch from IV to oral therapies, which would decrease the frequency of clinic visits.

Decisions on modifying or withholding chemotherapy need to consider both the indication and goals of care, as well as where the patient is in the treatment regimen and tolerance to the therapy. As anexample, the riskbenefit assessment for proceeding with chemotherapy in patients with untreated extensive small-cell lung cancer is quite different than proceeding with maintenance pemetrexed for metastatic nonsmall cell lung cancer.

If local coronavirus transmission is an issue at a particular cancer center, reasonable options may include taking a 2-week treatment break or arranging treatment at a different facility.

Evaluate if home infusion is medically and logistically feasible.

In some settings, delaying or modifying adjuvant treatment presents a higher risk of compromised disease control and long-term survival than in others, but in cases where the absolute benefit of adjuvant chemotherapy may be quite small and other options are available, the risk of COVID-19 may be considered an additional factor when evaluating care.

For patients who are candidates for allogeneic stem cell transplantation, a delay may be reasonable if the patient is currently well controlled with conventional treatment, ASCO comments. It also directs clinicians to follow the recommendations provided by the American Society of Transplantation and Cellular Therapy and from the European Society for Blood and Marrow Transplantation regarding this issue.

Finally, there is also the question of prophylactic antiviral therapy: Should it be considered for cancer patients undergoing active therapy?

The answer to that question is currently unknown, says ASCO, but "this is an active area of research and evidence may be available at any time."

For more from Medscape Oncology, join us on Twitter and Facebook.

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Disruptions in Cancer Care in the Era of COVID-19 - Medscape

In vivo Comparison of the Biodistribution and Toxicity of InP/ZnS Quan | IJN – Dove Medical Press

Li Li,1,2 Yajing Chen,1 Gaixia Xu,2,3 Dongmeng Liu,1 Zhiwen Yang,1 Tingting Chen,1 Xiaomei Wang,1 Wenxiao Jiang,1 Dahui Xue,1 Guimiao Lin1

1Base for International Science and Technology Cooperation: Carson Cancer Stem Cell Vaccines R&D Center, Shenzhen Key Laboratory of Synthetic Biology, Department of Physiology, School of Basic Medical Sciences, Shenzhen University, Shenzhen 518055, Peoples Republic of China; 2Key Laboratory of Optoelectronics Devices and Systems of Ministry of Education/Guangdong Province, College of Optoelectronic Engineering, Shenzhen University, Shenzhen 518060, Peoples Republic of China; 3Guangdong Key Laboratory for Biomedical Measurements and Ultrasound Imaging, School of Biomedical Engineering, Health Science Center, Shenzhen University, Shenzhen 518055, Peoples Republic of China

Correspondence: Guimiao LinSchool of Basic Medical Sciences, Shenzhen University Health Sciences Center, Shenzhen 518060, Peoples Republic of ChinaTel/ Fax +86-755-86671903Email gmlin@szu.edu.cn

Introduction: Indium phosphide (InP) quantum dots (QDs) have shown a broad application prospect in the fields of biophotonics and nanomedicine. However, the potential toxicity of InP QDs has not been systematically evaluated. In particular, the effects of different surface modifications on the biodistribution and toxicity of InP QDs are still unknown, which hinders their further developments. The present study aims to investigate the biodistribution and in vivo toxicity of InP/ZnS QDs.Methods: Three kinds of InP/ZnS QDs with different surface modifications, hQDs (QDs-OH), aQDs (QDs-NH2), and cQDs (QDs-COOH) were intravenously injected into BALB/c mice at the dosage of 2.5 mg/kg BW or 25 mg/kg BW, respectively. Biodistribution of three QDs was determined through cryosection fluorescence microscopy and ICP-MS analysis. The subsequent effects of InP/ZnS QDs on histopathology, hematology and blood biochemistry were evaluated at 1, 3, 7, 14 and 28 days post-injection.Results: These types of InP/ZnS QDs were rapidly distributed in the major organs of mice, mainly in the liver and spleen, and lasted for 28 days. No abnormal behavior, weight change or organ index were observed during the whole observation period, except that 2 mice died on Day 1 after 25 mg/kg BW hQDs treatment. The results of H&E staining showed that no obvious histopathological abnormalities were observed in the main organs (including heart, liver, spleen, lung, kidney, and brain) of all mice injected with different surface-functionalized QDs. Low concentration exposure of three QDs hardly caused obvious toxicity, while high concentration exposure of the three QDs could cause some changes in hematological parameters or biochemical parameters related to liver function or cardiac function. More attention needs to be paid on cQDs as high-dose exposure of cQDs induced death, acute inflammatory reaction and slight changes in liver function in mice.Conclusion: The surface modification and exposure dose can influence the biological behavior and in vivo toxicity of QDs. The surface chemistry should be fully considered in the design of InP-based QDs for their biomedical applications.

Keywords: InP/ZnS quantum dots, surface chemistry, in vivo, biodistribution, nanotoxicology

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Edited Transcript of SRPT earnings conference call or presentation 26-Feb-20 9:30pm GMT – Yahoo Finance

BOTHELL Mar 21, 2020 (Thomson StreetEvents) -- Edited Transcript of Sarepta Therapeutics Inc earnings conference call or presentation Wednesday, February 26, 2020 at 9:30:00pm GMT

* Alexander G. Cumbo

Sarepta Therapeutics, Inc. - Executive VP & Chief Commercial Officer

* Douglas S. Ingram

Sarepta Therapeutics, Inc. - President, CEO & Director

Sarepta Therapeutics, Inc. - Executive VP of R&D and Chief Medical Officer

* Ian M. Estepan

Sarepta Therapeutics, Inc. - Senior VP of Corporate Affairs & Chief of Staff

Sarepta Therapeutics, Inc. - SVP of Gene Therapy

Sarepta Therapeutics, Inc. - Executive VP, CFO & Chief Business Officer

* Christopher N. Marai

Nomura Securities Co. Ltd., Research Division - MD & Senior Analyst of Biotechnology

* Debjit D. Chattopadhyay

H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst

* Peter B. Kim

Sanford C. Bernstein & Co., LLC., Research Division - VP

Ladies and gentlemen, thank you for standing by, and welcome to the Sarepta Therapeutics Fourth Quarter 2019 Earnings Call. (Operator Instructions) As a reminder, today's program may be recorded.

I would now like to introduce your host for today's program, Ian Estepan, Senior Vice President, Chief of Staff and Corporate Affairs. Please go ahead, sir.

Ian M. Estepan, Sarepta Therapeutics, Inc. - Senior VP of Corporate Affairs & Chief of Staff [2]

Thank you so much, John, and thank you all for joining today's call. Earlier today, we released our financial results for the fourth quarter and full year 2019. The press release is available on our website at http://www.sarepta.com, and our 10-K was filed with the SEC earlier this afternoon. Joining us on the call today are Doug Ingram, Sandy Mahatme, Bo Cumbo, Dr. Gilmore O'Neill and Dr. Louise Rodino-Klapac. After our formal remarks, we'll open up the call for Q&A.

I'd like to note that during this call, we will be making a number of forward-looking statements. Please take a moment to review our slide on the webcast, which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, any of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements, and any and such risks can materially and adversely affect the business, the results of operations and trading prices for Sarepta's common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual report on Form 10-K filed with the Securities and Exchange Commission as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today based on subsequent events or circumstances.

And with that, I'd like to turn the call over to Doug Ingram for our corporate update.

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Douglas S. Ingram, Sarepta Therapeutics, Inc. - President, CEO & Director [3]

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Thank you, Ian. Good afternoon, and thank you all for joining Sarepta Therapeutics Fourth Quarter 2019 Conference Call.

In 2018, we defined our vision to become one of the world's leaders in precision genetic medicine to treat rare disease, founded both on our precise and efficient RNA platform and on the build of a gene therapy engine capable of rapidly advancing multiple constructs through development into the patient community. In 2019, we executed, further matured and brought that vision into greater focus. And in 2020 -- through 2020 we will, if successful, realize much of that vision.

We have an enormous number of milestones in 2020. But before we discuss them, let us review the progress that we have made in 2019. I will begin with our RNA platform.

As we announced at the JPMorgan conference in January, our fourth quarter 2019 revenue stands at $100 million. In our third full year since launch, our 2019 revenue was $381 million, a 26% increase over prior year. I will remind you that we have never taken a price increase since launch, so our growth comes from continuing to serve the Duchenne community.

Our 2020 guidance for EXONDYS is $420 million to $430 million. As we are just launching VYONDYS, we will wait until later this year before providing revenue guidance, but you can expect the launch curve similar to that of EXONDYS.

In the fourth quarter, we obtained FDA approval for our second RNA therapy, VYONDYS 53. The approval of VYONDYS was a win for objective evidence-based decision-making. It was a win for hard-working professionals at the FDA neurology division that was responsible for this review, and most importantly, it was a win for exon 53 amenable patients.

With regulatory pathway reconfirmed, we submitted our rolling NDA for casimersen, having announced positive results earlier in 2019. Assuming casimersen is approved, we will have 3 therapies capable of treating approximately 30% of the Duchenne community in the United States. We will have doubled the number of patients who may benefit from our PMO technology versus EXONDYS alone, and we will be among the exceedingly small number of biotechnology companies who have internally discovered, developed and brought to the patient community 3 or more medicines.

In 2019, we commenced our multi-ascending dose study for our next-generation PMO technology, the peptide-conjugated PMO or PPMO for short.

Now let's move on to our gene therapy engine. There, we've made great progress in 2019 as well. Starting with SRP-9001, our gene therapy for the treatment of Duchenne muscular dystrophy using our microdystrophin construct. We have completed all dosing in what became a 41-patient, placebo-controlled trial, Study 102. Patients are now crossing over at the end of their 48-week period. By now, between our first proof-of-concept study, our main study for 102 and our crossover, we have dosed more than 30 Duchenne boys with active gene therapy. The study continues uninterrupted, and the last patient last visit should occur in December of this year.

We have designed our next placebo-controlled trial using our commercial process material, and we've taken initial feedback from the agency. This trial, which we call Study 301 is designed as a global placebo-controlled, multi-center trial. We have made significant progress on manufacturing. With our partners Thermo Fisher and Catalent, we have built significant capacity with a dedicated facility completed in Lexington, Massachusetts and even greater capacity than that built at Catalent. Our hybrid manufacturing approach is taking shape with ADPD expertise at our Columbus site and a dedicated ADPD site in Burlington, Massachusetts. This intellectual hub has been responsible for some of our most meaningful advances in 2019.

Consider that we have now achieved at scale, commercially viable yields for SRP-9001, we announced at JPMorgan that we had commenced engineering runs. By now, I can tell you that we have commenced our GMP runs for SRP-9001, and we're making great progress on assay development as well.

We've made great progress on our limb-girdle pipeline in 2019. To remind you, LGMD, or limb-girdle muscular dystrophy, is an umbrella name for a collection of serious, often fatal neuromuscular diseases. None of these diseases have available therapies, so the opportunity to bring a better life for these patients is compelling. In the first quarter of 2019, we exercised our option and acquired Myonexus, gaining access to its 5 LGMD programs. And then we later entered into a license option with NCH to gain access to Dr. Zarife Sahenk's LGMD candidate for LGMD2A. These 6 programs together have the potential of providing treatments for over 70% of patients with LGMD.

In the first quarter of 2019, we presented expression and safety data from our first 3 patient proof-of-concept cohort for LGMD2E, and it was impressive. Expression was 50% on IHC and 30% -- 37% abnormal on Western blot. We came back in the fourth quarter, and we updated with 9-month functional data, indicating that every child was improving on every functional end point.

We commenced 1 additional higher-dose, 3-patient cohort in 2019 at a 4x higher dose with the goal of making a dose selection in 2020 this year.

Moving on to the rest of our gene therapy engine. 2019 was equally consequential. With our partner, Lysogene, we commenced a gene therapy trial for MPS IIIA or Sanfilippo Syndrome Type A devastating neurological lysosomal storage disease. We built out our gene therapy center of excellence in Columbus, Ohio. Our center of excellence is already building new constructs and advancing the science of gene therapy.

We entered into 14 transactions in 2019, and we in-licensed or purchased 18 new constructs, bringing the total number of research and development programs to 42 across our 2 platforms. And we have employed a clever incubation strategy that allows us to build an enormously large pipeline while still permitting us to remain laser-focused on our near-term objectives and milestones.

And of course, we entered into a transformational alliance with Roche in the fourth quarter of 2019 where Roche will take SRP-9001 to patients outside the United States. This alliance, by far, the largest ex-U. S. single candidate, license and biopharmaceutical history, validates our approach, our progress and the value of our program, but it also serves our mission. If SRP-9001 proves successful, Roche, with its very impressive ex-U. S. resources and international expertise, will bring our therapy to far more patients far faster than we could have ever done on our own. And it places us in an enviable position with the resources to drive our vision and to execute our plans. With the close of our alliance this quarter, we have well over $2 billion of cash on our balance sheet today; add to that the fact that we have just entered into an agreement to sell our VYONDYS priority review voucher for $111 million; add again to that our revenue this year for EXONDYS and VYONDYS, and it should become clear that we are well positioned with the resources, the assets and the talent to drive our ambitious strategy to fruition.

Looking forward, you will see that 2020 is dense with milestones. So starting with our gene therapy portfolio for 2020, with respect to SRP-9001, we will continue to execute Study 102 with our 48-week last patient last visit in December of this year. We will unblind, evaluate and release those results, which should occur in the first quarter of 2021.

We are preparing to commence our commercial supply trial, Study 301. Broadly, we have 3 work streams for Study 301. We must complete site initiation and training. We must complete our assay work, our engineering work and our GMP runs. And if all goes well, we should have GMP material released this July. We need to work with the division to obtain their concurrence on the commencement of Study 301. So of course, there's a lot to do here, but the team is making exceptional progress to date.

With respect to our LGMD pipeline, we have dosed all 3 patients now in our high-dose cohort for LGMD2E. We will have expression and safety results available in the second quarter, and we anticipate announcing that data at an appropriate medical meeting in the second quarter. We will make a formal dose selection decision in the third quarter. We will complete the assay and process development work for LGMD2E with the goal of having GMP material available in time to commence a trial in early 2021. We will also begin the ADPD work for other of our LGMD constructs as well.

We will continue our dialogue with the FDA and come to a view on the development and regulatory pathway for LGMD2E and then the remainder of the LGMD pipeline. Our goal is to have all of that completed by year-end, so we could commence a trial with commercial process material early next year.

We've also dosed 17 patients on our MPS IIIA gene therapy program and intend to complete all the dosing by the middle of the year. Our collaborator on CMT, otherwise known as Charcot-Marie-Tooth, Dr. Zarife Sahenk at Nationwide Children's Hospital, had intended to commence our proof-of-concept study for CMT last year but did not have NCH released material, enabling her to do that. That material should be available this year, and Dr. Sahenk intends to commence that study in 2020.

In addition to our gene therapy center of excellence in Columbus, Ohio, we are also building a separate gene-editing innovation center under the guidance of Dr. Charlie Gersbach of Duke University in Durham, North Carolina and should have that largely complete this year.

We have also significant milestones for our RNA platform this year. We should complete our rolling submission for casimersen in the second quarter of 2020. We plan to result -- to release the results from our PROMOVI study at the MDA Scientific Conference in March. These results from patients that met the enrollment criteria for 201/202, that's the study, which formed the basis for the eteplirsen approval, are consistent with the 201/202 data set. And we will have dosing and safety insight on our next-generation RNA platform, the PPMO, this year as well. If the PPMO is successful, it could be a significant advancement in our RNA technology and platform.

In summary, we have an enormous amount of work to do this year. But that work will be profoundly consequential for Sarepta and, of course, more importantly, for the patients that we serve. To those who may say our plans are ambitious, I would agree. But they are not driven by hubris. They are formed instead by the binding conviction founded on objective evidence that the science of genetic medicine has come of age, that a revolution in health care is upon us now, and that Sarepta is playing a leading role in translating that science to practical therapies that improve countless lives otherwise stolen by serious, rare genetic diseases. And it is in that spirit that I would invite you to join Sarepta and rare disease patients in the U.S. and around the world in recognizing Rare Disease Day this Saturday, February 29, as we continue to bring awareness about rare diseases and the work that remains to bring therapies to patients fighting those diseases every day.

And with that, I will turn the call over to Sandy to provide an update on the financials. Sandy?

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Sandesh Mahatme, Sarepta Therapeutics, Inc. - Executive VP, CFO & Chief Business Officer [4]

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Thanks, Doug. Good afternoon, everyone. Over the course of 2019, we advanced the business in several significant ways: we beat revenue guidance for EXONDYS 51; launched another of our RNA medicines, VYONDYS 53; significantly bolstered our financial position; and struck several new licensing deals, bringing our total number of development programs up to 42. We also started a partnership with Roche that closed earlier this month and that brought in $1.15 billion into the company. This collaboration brings significant capital to fully fund our pipeline, including our sharing payments, and it provides us access to Roche's significant expertise and greatly expand the global opportunity for our lead gene therapy program, SRP-9001.

Now moving to the financials. This afternoon's press release provided details for the fourth quarter of 2019 on a non-GAAP basis as well as a GAAP basis. The press release is available on Sarepta's website. Please refer to it for full reconciliation of GAAP to non-GAAP.

Net product revenue for the fourth quarter of 2019 was $100.1 million compared to $84.4 million for the same period of 2018. The increase primarily reflects higher demand for EXONDYS 51. On a GAAP basis, the company reported a net loss of $235.7 million and $140.9 million or $3.16 and $2.05 for basic and diluted shares for the fourth quarter of 2019 and '18, respectively.

We reported a non-GAAP net loss of $116.9 million or $1.57 per basic and diluted share in the fourth quarter of 2019 compared to a non-GAAP net loss of $58.7 million or $0.85 per basic and diluted shares in the fourth quarter of 2018.

In the last quarter of 2019, we recorded approximately $15.6 million in cost of sales compared to $13.1 million in the same period of last year. The increase was driven by royalties due to BioMarin Pharmaceuticals and University of Western Australia as well as higher production costs as a result of increasing demand for EXONDYS 51.

On a GAAP basis, we recorded $223.1 million and $146.2 million in R&D expenses for the fourth quarters of 2019 and 2018, respectively, which is a year-over-year increase of $76.9 million. This increase is primarily related to $40 million of increasing expenses in clinical and manufacturing, a $10.8 million increase in compensation and other personnel expenses as well as a $10.4 million increase in milestone cadence.

On a non-GAAP basis, R&D expenses were $135.4 million for the fourth quarter of 2019 compared to $77 million for the same period in 2018, an increase of $58.4 million. The year-over-year growth in non-GAAP R&D expenses was driven primarily due to a continuing ramp-up of our micro-dystrophin distribution program, our ESSENCE program and initiation of certain post-market studies for EXONDYS 51.

Turning to SG&A. On a GAAP basis, we recorded $81.4 million and $64.2 million of expenses in the fourth quarters of 2019 and 2018, respectively, a year-over-year increase of $17.2 million. On a non-GAAP basis, the SG&A expenses were $65.8 million for the fourth quarter of last year compared to $52.9 million for the same period of 2018, an increase of $12.9 million. The year-over-year increase was driven by significant organizational growth and expansion, supporting our commercial launch as well as 40 therapies in various stages of development across several therapeutic modalities.

On a GAAP basis, we recorded $4.8 million in other expenses for the fourth quarter of 2019 compared to $2.3 million of expenses for the same period of 2018. The unfavorable change is primarily driven by increase in interest expense, which is recognized for our new term loans that was received by the company in December of 2019.

We had approximately $1.1 billion in cash, cash equivalents and investments as of the end of last year. In addition to the closing of our alliance with Roche this quarter, we have well over $2 billion in cash on our balance sheet today.

With that, I would like to turn the call over to Bo for a commercial update. Bo?

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Alexander G. Cumbo, Sarepta Therapeutics, Inc. - Executive VP & Chief Commercial Officer [5]

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Thank you, Sandy. Good afternoon, everyone. Toward our 2019 objectives around execution and our commitment to deliver on our stated goals, I am pleased to report the following on behalf of the organization. We exceeded revenue consensus expectations for both the fourth quarter and the full year of 2019, totaling $100.1 million and $380.8 million, respectively. As Doug mentioned, our 2020 guidance for EXONDYS 51 is $420 million to $430 million. In terms of continuing to serve the community, we know that there are additional patients who may benefit from EXONDYS 51, and we will continue to overcome access and reimbursement challenges to get patients on therapy.

golodirsen, or VYONDYS 53, received accelerated approval by the FDA on December 12, 2019. VYONDYS 53 treats Duchenne muscular dystrophy patients who are amenable to skipping exon 53. Acting with urgency and the knowledge that patients were waiting, we launched VYONDYS 53 within 24 hours of FDA approval, just as we did with EXONDYS 51. We submitted all of our compendia, contracting and reporting requirements, and vyondys53.com, a critically important resource for families, went live. While we are leveraging our deep knowledge and expertise for the EXONDYS 51 launch, it is important to understand that there will be standard procedures and required reimbursement policies associated with launching a new drug with a unique NDC or National Drug Code. Our team is prepared to work through these requirements as we have in the past, and we anticipate a measured and steady launch trajectory for VYONDYS 53, resembling the launch curve for EXONDYS 51. The only difference is that we are preparing and planning for the amenable exon 53 space to be competitive.

In support of our goal to increase access for VYONDYS 53, we are pursuing a multi-pronged strategy. Although commercial and state Medicaid plans now have a much better understanding of Duchenne, we are continuing to educate about disease progression and the benefits of treatment. Our goal is to work towards coverage to be all-inclusive regardless of ambulation status, age or gender. We do expect commercial payers to have medical policies in place faster than Medicaid. We also understand that from our previous launch that the mix of commercial to Medicaid patients will adjust over time, and we believe it will eventually move towards a 50-50 mix or higher for Medicaid.

Further, we are continuing to engage with state Medicaid plans regarding the CMS guidance letter on the obligation of state Medicaid to make accelerated approval treatments available to patients. As you know, this is critically important for Duchenne based on the percent of patients covered under Medicaid plans.

While the launch over Christmas holiday did delay some physician and patients seeking treatment during that period of time, we have been receiving START Forms from top-tier centers across the U.S. and are working with health care providers to ensure they are educated around amendability for skipping exon 53, as this population is different from exon 51 with some exceptions. Epidemiology suggests that VYONDYS 53 can serve approximately 8% of the Duchenne community. But we will have to take into consideration that there are a number of patients already enrolled in or being recruited for clinical trials, or have a deletion that would be amenable to exon 51 and, therefore, could already be on EXONDYS 51.

With that said, we continue to have conversations with health care providers about the number of patients within their clinics and with payers about the number of patients eligible for treatment under their plan. We are working with both health care providers and payers to get all amenable patients on VYONDYS 53 as soon as possible. Our mission to be the global leader in precision-genetic medicine started with EXONDYS 51 and have continued with the approval and launch of VYONDYS 53. We are now preparing for the potential launch of casimersen for patients amenable to skipping exon 45. Behind these important medicines is an industry-leading pipeline of programs, 42 in all, driven by new modalities designed to treat complex rare diseases, including MPS IIIA and neuromuscular dystrophy. Sarepta is working with urgency and is focused on understanding the epidemiology and global prevalence of these diseases.

We are continuing to refine our analysis and uncover additional insights while collaborating with top neuromuscular specialists. Each day, we are learning more about these diseases. And with each piece of evidence that we gather, we are able to apply these insights to our disease awareness and patient identification efforts that are already underway.

2019 was a great -- was a year of great accomplishments, not only for the commercial organization but the company overall. Looking to the future, patient care will continue to be our driving force as we translate scientific innovations into medicines designed to improve the lives of patients around the world.

And with that, I'll turn the call back over to Doug.

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Douglas S. Ingram, Sarepta Therapeutics, Inc. - President, CEO & Director [6]

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Thank you, Bo, and thank you, Bo and Sandy. With that, let's open the call for questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from the line of Ritu Baral from Cowen.

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Ritu Subhalaksmi Baral, Cowen and Company, LLC, Research Division - MD & Senior Biotechnology Analyst [2]

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Doug, can you let us know when the last patients for gene therapy was dosed? Basically, what is the shortest follow-up period, both for microdystrophin as well as limb-girdle? And can you talk about the safety profile, especially liver, especially platelets, that you've seen in that time period for both programs?

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Douglas S. Ingram, Sarepta Therapeutics, Inc. - President, CEO & Director [3]

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Well, I can just tell you very broadly that as I sort of backward engineer, we're going to have the -- for the 41-patient study, 102, the last patient last visit will be in December. So if you work backwards, you'll see that the last patient was right at -- I think it actually might have been the very first weekend in 2020. So that was the first 41 patients dosed. We're continuing on an ongoing basis to those patients on crossover as well. There's a significant number, as I've mentioned to you now. But in the Study 102, between the proof-of-concept 101, between the main 41-patient study and between the crossovers, we've dosed over 30 patients with active therapy. We have now dosed 6 patients with limb-girdle, both the previous dose and now the higher dose in limb-girdle. And of course, a lot of that's blinded, so that we'll all see together both the safety and -- the full safety and efficacy. But broadly speaking, I will say, again, consistent with our preclinical models, we have never seen anything that looks like a complement or reductions in platelet counts below the normal level. So things continue as they were. Study 102 continues, completely uninterrupted, making great progress there. The exciting thing about 102 is that we'll have last patient in December, and we'll have a readout in the first quarter of 2021. And I will remind you that is a readout, not merely on expression and on safety but also on function using NSAA. Thank you for that question.

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Operator [4]

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Our next question comes from the line of Tazeen Ahmad from Bank of America.

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Edited Transcript of SRPT earnings conference call or presentation 26-Feb-20 9:30pm GMT - Yahoo Finance