Biomarin Pharmaceutical Inc

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

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.

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

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

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

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 Pharmaceuticals (ALXN) gained 2.5% on December 10, 2015. The stock went up after the FDA (Food and Drug Administration) approved its drug, Kanuma.

Horizon plans to drive Krystexxa through increased awareness and outreach, investing in its marketing and medical education as well as commercial infrastructure.

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

Baxalta’s (BXLT) hemophilia portfolio comprises of Advate, Adynovate, Rixubis, Recombinate, Hemofil M, Immunate, Immunine, and Prothromplex Total.

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

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.

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 (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.

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.

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

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

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

The Zulresso injection is Sage Therapeutics’ lead product candidate. The company has filed a new drug application.

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.

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 (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.

Analysts are estimating that Allergan’s (AGN) third-quarter revenues will decline 3.8% to $3.88 billion.

For Biogen, eight analysts recommended a “strong buy,” 12 recommended a “buy,” and nine recommended a “hold.”

Sage Therapeutics’ (SAGE) portfolio includes products under development for the treatment of life-threatening central nervous system (or CNS) disorders.

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.

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 (BLUE) released on September 5 its updated data from the Phase 2/3 Starbeam study evaluating investigational Lenti-D gene therapy.

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.

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

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.

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

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.

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 (AMGN) reported in Q2 2018 results yesterday, surpassing analysts’ EPS and revenue estimates.

BioMarin Pharmaceutical (BMRN) plans to release its Q2 2018 results on August 2. In Q1 2018, BMRN reported net revenues of $373.4 million.

On June 12, Sage Therapeutics announced its expedited development plan for SAGE-217.

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

Of the 20 analysts covering Spark Therapeutics in April 2018, four have given the stock “strong buy” recommendations.

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

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

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

Ionis Pharmaceuticals (IONS) reported a 9% rise in its revenue to $120.9 million in 3Q17 compared to $110.9 million in 3Q16.

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

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.

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

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.”

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.

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.”

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.

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

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

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

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

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

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 […]

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

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.

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.

With its continuous revenue fall, Gilead Sciences (GILD) could opt to restore its growth through acquisitions.

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.

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 (INCY) reported revenues of $246.3 million in 2Q16, an increase of 51% compared to $163 million in 2Q15.

Shire’s (SHPG) LSD (or lysosomal storage disorders) portfolio consists of Elaprase, Replagal, and Vpriv.

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.

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.

Following Brexit, global equity markets experienced a crash. Anticipations of weaker European and British economies are likely reasons for the fall.

Global big pharmaceutical companies that have major exposure to Europe will be impacted significantly by Brexit. The depreciating euro will impact sales.

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).

Vertex is trying to expand its product base beyond cystic fibrosis. It holds molecules in the oncology, respiratory, pain, spinal cord, and influenza spaces.

Gene therapy is the emerging platform for hemophilia care. If approved, it would be a paradigm.

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.

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

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.

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.

Incyte, a Delaware-based biopharmaceutical company, is focused on developing and commercializing oncology products to cater to unmet medical needs.

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.

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

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 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.

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.

Kuvan, with the active ingredient sapropterin dihydrochloride, is effective in reducing blood phenylalanine levels in PKU (or phenylketonuria) patients.

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

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.

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.

Let’s discuss BioMarin’s business model. It derives revenue from three sources, including product revenue. The latter accounts for ~98% of total revenue.

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%.

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.

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 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.

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.

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.