Biomarin Pharmaceutical Inc
Latest Biomarin Pharmaceutical Inc News and Updates

BioMarin Has Suffered from Kyndrisa’s Rejection
The recent fall in BioMarin’s stock price is due to the rejection of its drug Kyndrisa, indicated for Duchenne muscular dystrophy.

Gauging Analysts’ Views on Tesaro Stock
In November, of the total 21 analysts covering Tesaro (TSRO) stock, 13 have given it “buy” or higher ratings, and eight have given it “hold” ratings.

What to Expect from BioMarin Pharmaceuticals in 2017
In 2016, BioMarin Pharmaceuticals (BMRN) reported revenues of around $1.1 billion, a ~25% increase year-over-year (or YoY).

What Spark Therapeutics’ Valuation Trend Indicates
Spark Therapeutics’ (ONCE) operating expenses decreased from $78.48 million in Q2 2017 to $59.78 million in Q2 2018.

How BioMarin Pharmaceutical Is Positioned Financially in August
In the second quarter, BioMarin Pharmaceutical (BMRN) reported revenues of $372.8 million compared to $317.4 million in Q2 2017.

Amgen Rises by 2% as XBI’s Large-Cap Stocks Rally
Amgen closed at $157.60 and was trading above its 50- and 100-day moving averages. The stock also witnessed a rise in trading volumes.

Alexion Continues to Rise on FDA’s Approval of Kanuma
Alexion Pharmaceuticals (ALXN) gained 2.5% on December 10, 2015. The stock went up after the FDA (Food and Drug Administration) approved its drug, Kanuma.

Revenue Drivers for Krystexxa, Horizon’s Orphan Biologic Drug
Horizon plans to drive Krystexxa through increased awareness and outreach, investing in its marketing and medical education as well as commercial infrastructure.

Overview of BioMarin: History and Product Portfolio
Here we present an overview of BioMarin. It’s based in California and was founded in 1997. It focuses on therapies for life-threatening rare genetic diseases.

BioMarin’s Disappointing 3Q16 Earnings
BioMarin Pharmaceutical (BMRN) reported its 3Q16 earnings on October 27, 2016. Revenues were $279.9 million, which was lower than Wall Street analysts’ projections.

Could Baxalta Maintain Its Leadership in the Hemophilia Space?
Baxalta’s (BXLT) hemophilia portfolio comprises of Advate, Adynovate, Rixubis, Recombinate, Hemofil M, Immunate, Immunine, and Prothromplex Total.

BioMarin Leads in the Market for Mucopolysaccharidoses Disorders
BioMarin holds three drugs in its portfolio indicated for different types of mucopolysaccharidoses disorders. They are Vimizim, Naglazyme, and Aldurazyme.

BMN 270: A Big Valuation Catalyst for BioMarin
On March 1, 2016, BioMarin received orphan drug designation for BMN 270 from the FDA. BioMarin’s stock jumped by about 6.96% the same day.

Why Sesen Bio Stock Jumped Over 37% this Week
Sesen Bio (SESN), formerly known as Eleven Biotherapeutics, is developing next-generation antibody-drug conjugate therapies. The stock jumped ~26.1% to $2.27 yesterday.

Horizon Therapeutics or BioMarin: Which Is a Better Bet in July?
Horizon Therapeutics (HZNP) is up 23.13%, and BioMarin (BMRN) is down 6.62% year-to-date. Let’s study the drivers and risks for both these rare disease players in greater detail.

A Look at Sarepta Therapeutics’ Recent Developments
In the first nine months of 2018, Sarepta Therapeutics’ (SRPT) net income and EPS amounted to -$221.0 million and -$3.38, respectively, compared to -$26.7 million and -$0.47 in the same period the prior year.

How Is Sarepta Therapeutics Positioned in January?
In the first nine months of 2018, Sarepta Therapeutics’ net revenues grew ~123% YoY to reach $216.6 million from $97.3 million.

Sarepta Therapeutics Stock Rose 96% in 2018
On January 4, Sarepta Therapeutics (SRPT) stock closed at $115.43, which represents ~8.21% growth from its prior close.

Sage Therapeutics’ Operational Performance
In fiscal 2018 and fiscal 2019, Sage Therapeutics (SAGE) is expected to generate revenues of $79.41 million and $36.63 million, respectively.

Sage Therapeutics: Analyzing New Product Candidates
The Zulresso injection is Sage Therapeutics’ lead product candidate. The company has filed a new drug application.

How BioMarin Pharmaceutical’s Financials Look in November
BioMarin Pharmaceutical’s (BMRN) net revenues over the first nine months of this year amounted to $1.1 billion compared to $955.3 million in the same period the prior year.

Gauging Analysts’ Views on Clovis Stock
In November, of the 12 analysts covering Clovis Oncology (CLVS) stock, seven have given it “buy” or higher ratings, and five have given it “hold” ratings.

Allergan Beats EPS and Revenue Estimates: Q3 2018 Earnings
Allergan (AGN) released its Q3 2018 earnings today. The company beat Wall Street analysts’ estimates for EPS and revenues, and it reported non-GAAP EPS of $4.25 on revenues of $3.91 billion.

Allergan’s Q3 2018 Estimates: Revenue Decline Expected
Analysts are estimating that Allergan’s (AGN) third-quarter revenues will decline 3.8% to $3.88 billion.

Biogen: Analysts’ Recommendations on October 19
For Biogen, eight analysts recommended a “strong buy,” 12 recommended a “buy,” and nine recommended a “hold.”

Here Are the Key Products in Sage’s GABA Receptor Portfolio
Sage Therapeutics’ (SAGE) portfolio includes products under development for the treatment of life-threatening central nervous system (or CNS) disorders.

Estimates and Recommendations for Shire on September 19
Wall Street analysts expect Shire (SHPG) to report a 2.7% rise in revenue to ~$15.56 billion in 2018 compared to ~$15.16 billion in 2017.

Estimates and Recommendations for Bluebird Bio on September 6
Wall Street analysts estimate that Bluebird Bio (BLUE) will report a 14.6% growth in revenues to ~$40.6 million in 2018 compared to $35.4 million in 2017.

Bluebird Bio’s Data for Lenti-D Investigational Gene Therapy
Bluebird Bio (BLUE) released on September 5 its updated data from the Phase 2/3 Starbeam study evaluating investigational Lenti-D gene therapy.

How Analysts View BioMarin Pharmaceutical in August
On August 30, 2018, BioMarin Pharmaceutical (BMRN) stock closed at $99.26 which is 7% below its 52-week high of $106.20 on August 2.

Here’s What Tesaro’s Valuation Trend Indicates
Tesaro stock has been on a downward trajectory for the past 52 weeks. From a high of $135 on September 5, 2017, it has corrected to $27 in August.

Sarepta Therapeutics’ Q2 2018 Earnings and Revenue Growth
Sarepta Therapeutics (SRPT) released its Q2 2018 earnings on August 8, surpassing analysts’ estimates for EPS and revenues.

How Ionis’s Q2 2018 Earnings Trended
Ionis reported 5% YoY growth in revenues to $117.7 million during Q2 2018 as compared to $112.3 million during the second quarter of 2017.

Bluebird Bio Misses Analysts’ Estimates in Q2 2018
Clinical-stage biotechnology company Bluebird Bio (BLUE) released its Q2 2018 results yesterday, missing analysts’ revenue and EPS estimates.

What Analysts Recommend for Bluebird Bio Stock
As previously discussed, analysts expect Bluebird Bio (BLUE) to post revenue of $9.6 million, a net adjusted loss of ~$123.5 million, and EPS of -$2.35 in the second quarter.

What to Expect from Shire’s Q2 2018 Earnings on July 31
Shire (SHPG) is set to release its Q2 2018 earnings on July 31. Wall Street analysts expect EPS of $3.67 on revenues of $3.8 billion.

Amgen Beats Estimates, Reports Growth in Q2 2018
Amgen (AMGN) reported in Q2 2018 results yesterday, surpassing analysts’ EPS and revenue estimates.

How Does BioMarin Pharmaceutical Look ahead of Its Q2 Earnings?
BioMarin Pharmaceutical (BMRN) plans to release its Q2 2018 results on August 2. In Q1 2018, BMRN reported net revenues of $373.4 million.

Sage Therapeutics’ GABA Receptor–Based Products
On June 12, Sage Therapeutics announced its expedited development plan for SAGE-217.

How Analysts View BioMarin Pharmaceuticals and Peers in April
Of the 23 analysts covering BioMarin Pharmaceuticals in April 2018, eight analysts gave the stock a “strong buy” rating.

Analysts’ Ratings for Spark Therapeutics and Its Peers in April 2018
Of the 20 analysts covering Spark Therapeutics in April 2018, four have given the stock “strong buy” recommendations.

Key Updates on Akcea Therapeutics’ Volanesorsen
Ionis Pharmaceuticals’ (IONS) Volanesorsen is a drug for the treatment of familial chylomicronemia syndrome (or FCS) and familial partial lipodystrophy (or FPL).

Alexion Pharmaceuticals in 4Q17 and Fiscal 2017
In 4Q17, Alexion Pharmaceuticals (ALXN) generated revenues of $910 million, which reflected ~10% growth on a YoY (year-over-year) basis.

How Biogen’s Spinraza Is Positioned for 2018
In 4Q17, Biogen’s (BIIB) Spinraza generated revenues of $363 million, which reflected 34% quarter-over-quarter growth.

A Look at Ionis’s 3Q17 Revenue Stream
Ionis Pharmaceuticals (IONS) reported a 9% rise in its revenue to $120.9 million in 3Q17 compared to $110.9 million in 3Q16.

How Was Incyte’s Revenue Stream in 3Q17?
Incyte’s total product royalty revenue was $44.5 million in 3Q17, a rise compared to $29.6 million in 3Q16.

BioMarin’s Strong Pipeline Could Be a Long-Term Growth Driver
After success in the company’s phase 1/2 trial with BMN 270, an investigational gene therapy for hemophilia A, BioMarin Pharmaceuticals (BMRN) is expected to start phase 3 trials.

How Is Jazz’s Defitelio Positioned after 2Q17?
In 2Q17, Jazz Pharmaceuticals’ (JAZZ) Defitelio generated revenues of ~$30 million, which represents a ~10% fall YoY and a ~17% fall QoQ.

What Analysts Recommend for Acadia Pharmaceuticals in September
Of the nine analysts covering Acadia Pharmaceuticals in September 2017, two rated the stock a “strong buy,” six rated it a “buy,” and one rated it a “hold.”

This Part of Bioverativ’s Pipeline Could Be a Major Long-Term Growth Driver
In June 2017, the FDA accepted Bioverativ’s investigative new drug application for BIVV001, a drug designed to treat prophylaxis from bleeding associated with hemophilia A.

Behind Bioverativ’s Analyst Recommendations This September
Of the 12 analysts tracking Bioverativ (BIVV), three recommend a “strong buy” for the stock, while one recommends a “buy,” and seven recommend a “hold.”

How Are Orkambi and Kalydeco Positioned after 2Q17?
In 2Q17, Vertex Pharmaceuticals’ (VRTX) Orkambi generated revenues of around $324 million, which reflected a whopping ~32% growth on a year-over-year (or YoY) basis and ~10% growth on a quarter-over-quarter basis.

Understanding Incyte’s 2Q17 Revenue Stream
Incyte’s 2Q17 revenue growth was driven by the increased demand for Jakafi.

What Drove Biogen Stock in 2Q17?
Biogen (BIIB) stock has risen ~0.40% in 2Q17 and ~4.1% year-to-date as of July 7, 2017.

The Rise of Amgen Stock in 2Q17
Amgen (AMGN) stock has risen ~5.8% in 2Q17, while it has risen ~17.4% year-to-date as of July 7, 2017.

What Analysts Recommend for BioMarin Pharmaceuticals
A total of 20 analysts were analyzing BioMarin Pharmaceuticals in July 2017. Six analysts recommended a “strong buy,” while nine analysts suggested a “buy.”

BioMarin Expects These Drugs to Generate Steady Revenues
In 2016, BioMarin Pharmaceuticals’ (BMRN) Naglazyme generated revenues of around $297 million, a ~2% year-over-year (or YoY) decline.

Developments for Ionis Pharmaceuticals after 1Q17
Ionis’s recent developments As discussed earlier, Ionis Pharmaceuticals (IONS) is focused on the development of drugs for the treatment of diseases with limited or no therapeutic options. Corporate developments In April 2017, Ionis entered into a collaborative agreement with Suzhou Ribo Life Science for the development and commercialization of RNA[1.ribonucleic acid]-targeted therapeutics in China. In […]

Strensiq: Key Growth Driver for Alexion Pharmaceuticals in 2017
In 1Q17, Alexion Pharmaceuticals’ (ALXN) Strensiq generated revenue of ~$74 million, which reflects ~4% quarter-over-quarter and ~124% YoY growth.

Vertex Has Adopted Multiple Approaches to Treat Cystic Fibrosis
Vertex Pharmaceuticals (VRTX) is aiming to increase the number of patients eligible to be treated with its drugs to include the entire CF patient population.

Horizon Pharma’s Steep Fall in December: What Happened?
On December 8, 2016, Horizon Pharma (HZNP) announced that its Phase 3 STEADFAST trial for Actimmune didn’t meet its primary endpoint. Horizon stock fell 22.5% that day.

How Could Gilead Sciences Restore Its Growth?
With its continuous revenue fall, Gilead Sciences (GILD) could opt to restore its growth through acquisitions.

Why Europe Is an Important Market for Vertex’s Orkambi
Although there are 12,000 eligible patients in Europe who are 12 years and older, Orkambi hasn’t yet received reimbursement approvals for these patients.

How Gilead Dominates the HCV Space
In January 2012, Gilead Sciences (GILD) acquired Pharmasset and got access to sofosbuvir. In December 2013, the FDA approved sofosbuvir under the brand name Sovaldi.

Incyte’s Profitability Improves, Allowing for Revised Guidance
Incyte (INCY) reported revenues of $246.3 million in 2Q16, an increase of 51% compared to $163 million in 2Q15.

How Will Shire’s LSD Portfolio Perform in 2016?
Shire’s (SHPG) LSD (or lysosomal storage disorders) portfolio consists of Elaprase, Replagal, and Vpriv.

The Word on the Street: How Analysts See BioMarin Now
According to Bloomberg, among 20 analysts, 90% recommend BioMarin as a “buy,” and the remaining 10% of analysts have issued a “hold” rating for the company.

The Evolution of Hemophilia Treatment—And What It Means for BioMarin
BioMarin’s BMN 270 is a gene therapy being investigated for hemophilia A. On April 20, it announced early data of the phase-1 and phase-2 study of BMN 270.

Why Did Alexion Fall after Brexit?
Following Brexit, global equity markets experienced a crash. Anticipations of weaker European and British economies are likely reasons for the fall.

Effects of Brexit on Pharmaceuticals and the Biotechnology Industry
Global big pharmaceutical companies that have major exposure to Europe will be impacted significantly by Brexit. The depreciating euro will impact sales.

BioMarin Withdraws Application for Kyndrisa: Stock Falls 2.5%
As of June 1, 2016, BioMarin Pharmaceutical (BMRN) was the most underperforming stock among the large-cap stocks of the SPDR S&P Biotech ETF (XBI).

How Does Vertex Plan to Expand Beyond Cystic Fibrosis?
Vertex is trying to expand its product base beyond cystic fibrosis. It holds molecules in the oncology, respiratory, pain, spinal cord, and influenza spaces.

Could Gene Therapy Be a Next-Generation Treatment for Hemophilia?
Gene therapy is the emerging platform for hemophilia care. If approved, it would be a paradigm.

How Does Shire Lead the Angioedema Space?
Cinryze and Firazyr are Shire’s (SHPG) two drugs in its hereditary angioedema portfolio. Together, these two drugs crossed $1 billion in sales in fiscal 2015.

Defitelio: Volume and Pricing Challenges
Jazz Pharmaceuticals’ Defitelio is the first and only approved treatment that increases survival in VOD patients with multi-organ dysfunction (or MOD).

Analysts Are Optimistic About Shire
According to a Bloomberg consensus of 15 brokerage firms, 86.7% of analysts rated Shire a “buy,” and 13.3% rated it a “hold.” None issued a “sell” rating for the stock.

Why Did Shire Fall after Shire-Baxalta Acquisition Announcement?
On January 11, 2016, Shire (SHPG) announced a merger agreement with Baxalta for a whopping $32 billion. The Shire-Baxalta deal is still in progress and should close by mid-2016.

How Does Incyte’s Valuation Compare to Peers?
Incyte, a Delaware-based biopharmaceutical company, is focused on developing and commercializing oncology products to cater to unmet medical needs.

Shire’s Acquisition Gives It Natpara and Gattex
Gattex is the first and only analog of glucagon-like peptide-2 (or GLP-2) indicated for short bowel syndrome. The drug is known as Gattex in the United States and Revestive in Europe.

Which Large-Cap Biotech Stock Fell the Most Last Week?
Large-cap biotech stocks struggled during the week ending March 18, 2016. The large-cap stocks within the iShares Nasdaq Biotechnology ETF (IBB) suffered the most, falling 3.9% during the week.

Alexion Pushes Down IBB’s Performance
Alexion Pharmaceuticals (ALXN) was the biggest loser among IBB’s large-cap stocks.

How Have IBB’s Large-Cap Stocks Performed?
IBB has approximately 14 large-capitalization stocks in its portfolio with a market capitalization of more than $10 billion as of February 19, 2016.

Vertex’s Cystic Fibrosis Drugs and Their Controversial Prices
Vertex Pharmaceuticals priced Kalydeco at $294,000 per year and recently increased the price to $311,000. Vertex is not the only company pricing its drugs at the higher end.

Orkambi: Recent Addition to the Vertex Portfolio
In July 2015, Vertex Pharmaceuticals’ (VRTX) Orkambi received FDA approval for treating cystic fibrosis in the United States for patients 12 years and older with two copies of the F508del mutation.

What Are BioMarin’s Products for Treating Phenylketonuria?
Kuvan, with the active ingredient sapropterin dihydrochloride, is effective in reducing blood phenylalanine levels in PKU (or phenylketonuria) patients.

BioMarin’s Firdapse for Lambert-Eaton Myasthenic Syndrome
Firdapse is indicated for Lambert-Eaton Myasthenic syndrome. The chemical name of Firdapse is amifampridine phosphate and its active ingredient is 3,4-DAP.

Vimizim: A Recent Addition to BioMarin’s Portfolio
In 2014, Vimizim (elosulfase alfa) received marketing approval by the FDA, followed by the approval of the European Medicines Agency for treating MPS IV A.

Naglazyme: One of the Costliest Drugs in the United States
The wholesale cost per patient for Naglazyme is around $485,747 per year. The drug has been effective in improving walking and stair-climbing capacity.

BioMarin’s Business Model and Its 3 Sources of Revenue
Let’s discuss BioMarin’s business model. It derives revenue from three sources, including product revenue. The latter accounts for ~98% of total revenue.

How Would Kyndrisa Treat Duchenne Muscular Dystrophy?
The FDA advisory committee has given an unfavorable opinion to Kyndrisa. But most analysts estimate the probability of FDA approval for the drug to be about 50%.

FDA Committee Raises Concerns about Kyndrisa’s Efficacy, Safety
On November 24, 2015, the Peripheral and Central Nervous System Drugs Advisory Committee of the FDA discussed clinical data submitted by BioMarin to support Kyndrisa’s (drisapersen) NDA.

FDA Sets December 27, 2015, as Kyndrisa’s PDUFA Date
The FDA has set December 27, 2015, as the PDUFA date for BioMarin Pharmaceutical’s (BMRN) Kyndrisa (drisapersen) for Duchenne Muscular Dystrophy (or DMD) Amenable to Exon 51 Skipping.

BioMarin Falls 16.81% in Week Ended September 25
BioMarin Pharmaceutical (BMRN) fell 16.81% and closed at $108.69 last week. That’s below the 20-day, 50-day, and 100-day moving averages. The 14-day RSI was 28, or oversold territory.

Top Ten of XBI Outperform by Market Cap
The average return of the top ten IBB stocks was -4.73%, while the average return of top ten XBI stocks was -3.89% for the week ended September 4, 2015.

Top Ten XBI Stocks by Market Cap Outperform August 25–31
The top ten stocks of the SPDR S&P Biotech ETF (XBI) outperformed the top ten stocks of the iShares Nasdaq Biotechnology ETF (IBB) and the Health Care Select Sector SPDR ETF (XLV) for August 25–31.