Biomarin Pharmaceutical Inc

On Friday last week, Sangamo Therapeutics (SGMO) and Pfizer (PFE) announced promising updated results from their Phase 1/2 ALTA trial.

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’ net revenues grew ~123% YoY to reach $216.6 million from $97.3 million.

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.

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

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

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

Today, Neurocrine Biosciences (NBIX) is trading at $67.91, a ~20.83% fall from its closing price of $85.76 on December 11.

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 total 21 analysts covering Tesaro (TSRO) stock, 13 have given it “buy” or higher ratings, and eight have given it “hold” ratings.

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.

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

Of the 23 analysts covering BioMarin Pharmaceutical (BMRN) in September, 19 of them have given the stock a “buy” or higher rating.

Stocks focused on gene therapy have garnered strong investor interest in 2018. In this series, we’ll analyze four of those companies.

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.

In the second quarter, Alkermes (ALKS) reported revenues of $304.6 million—compared to $218.8 million in the second quarter of 2017.

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

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.

Incyte (INCY) reported revenue of $521.5 million in the second quarter, a 59.7% rise YoY (year-over-year).

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.

Jazz Pharmaceuticals anticipates net revenues to be in the range of $1.88 million to $1.93 million in fiscal 2018.

Jazz Pharmaceuticals reported revenues of $500.5 million in Q2 2018 compared to $394.4 million in Q2 2017, a ~27% YoY increase and a ~13% sequential increase.

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.

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.

Vivus stock rose 60% between May 11 and May 18.

In December 2017, BioMarin Pharmaceuticals (BMRN) sold a rare pediatric disease priority review voucher to Novartis (NVS) for $125.0 million.

BioMarin Pharmaceuticals’ (BMRN) cash, cash equivalents, and investments increased from $1.3 billion in fiscal 2016 to $1.7 billion in fiscal 2017.

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

Of eight analysts tracking Tetraphase Pharmaceuticals, one analyst rated it as a “strong buy,” and six analysts gave it a “buy” rating.

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

On April 9, 2018, Novartis AG announced that it had entered into an agreement to acquire AveXis for ~$8.7 billion.

In March 2018, the EMA (European Medicines Agency) accepted BioMarin Pharmaceutical’s (BMRN) marketing authorization application for pegvaliase.

In 4Q17, BioMarin Pharmaceutical (BMRN) generated revenue of $358.3 million, compared with $300.1 million in 4Q16.

Wall Street analysts estimate that Ionis Pharmaceuticals (IONS) stock might increase 25.1% over the next 12 months.

Ionis Pharmaceuticals stock has fallen 6.9% over the last week. However, it rose 2.6% to close at $48.99 on March 26, 2018.

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

Ionis Pharmaceuticals (IONS) reported 7.5% growth in revenues to $172.3 million during 4Q17 as compared to revenues of $160.3 million during 4Q16.

Ionis (IONS) is a leading biopharmaceutical company focused on discovering, developing, and commercializing RNA-targeted therapies.

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’ Spinraza is a drug designed for the treatment of spinal muscular atrophy (or SMA), a type of severe motor-neuron disease.

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.

Gilead’s revenue Gilead Sciences (GILD) reported revenue of $6.5 billion in 3Q17, a 13% fall from the revenue of $7.5 billion seen in 3Q16. The 3Q17 figure includes product revenue of $6.4 billion, and royalty, contract, and other revenue of $110 million. Analysts expect revenue to fall ~21.9% to $5.7 billion in 4Q17. Geographical sales The above […]

Twenty-one analysts are tracking Alexion Pharmaceuticals in December 2017. Six of them are recommending a “strong buy,” while 12 are recommending a “buy.”

After 3Q17, Vertex Pharmaceuticals (VRTX) increased its guidance for cystic fibrosis product revenues, from $1.87 billion–$2.1 billion to $2.10 billion–$2.15 billion.

Recent developments In October 2017, Biogen (BIIB) and Eisai expanded their existing agreement on the joint development and commercialization of Alzheimer’s disease therapies under investigation. According to the terms of the agreement, Eisai has the option to co-develop and co-commercialize Biogen’s aducanumab for the treatment of Alzheimer’s disease patients. The present expansion of the agreement […]

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, BioMarin Pharmaceuticals’ (BMRN) Aldurazyme generated revenues of around $20 million, which was a ~6% increase on a year-over-year (or YoY) basis.

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.

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

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) Kuvan reported revenues of around $348 million, a ~45% year-over-year (or YoY) increase.

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

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 […]

Of the 21 analysts analyzing Alexion Pharmaceuticals, six analysts recommended a “strong buy” and 11 analysts recommended a “buy.”

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.

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

In 3Q16, Horizon Pharma’s (HZNP) net sales on a GAAP basis fell to $208.7 million. Non-GAAP net sales were $273.7 million.

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.

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

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.

Over the past year, VRTX stock has fallen 31.0%. But perhaps the entire biotechnology industry, not just Vertex, has suffered during the period.

BioMarin’s (BMRN) early-stage pipeline includes BMN-270, a gene therapy program for hemophilia A and vosoritide, which is indicated for achondroplasia.

BioMarin’s application for cerliponase alfa, an enzyme replacement therapy, is under review by the FDA for the treatment of CLN2 disease.

BioMarin Pharmaceutical (BMRN) expects its existing commercialized drugs to add $1.1 billion and $1.2 billion to its top line in 2016.

The Bloomberg consensus of 24 brokerage firms shows that 66.7% brokerage firms recommend Vertex as a “buy,” while 33.3% analyst issued a “hold” ratings.

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.

According to Horizon Pharma’s (HZNP) estimates, Procysbi holds a peak sales potential of more than $300 million.

The market was pleased with the merger deal between Horizon Pharma (HZNP) and Raptor Pharmaceuticals. HZNP shares rose to $18.89 on September 12.

Sanofi’s Specialty Care franchise consists of rare disease drugs, MS (multiple sclerosis) drugs, and oncology drugs and generated ~$3.3 billion in 1H16.

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

During its 2Q16 earnings call, BioMarin revised its 2016 revenue guidance. It now expects revenue to be $1.1 billion–$1.15 billion.

With increased M&A activity in the pharmaceuticals and biotechnology space, it wouldn’t be surprising if Roche Holding (RHHBY) acquired BioMarin (BMRN).

Shire anticipates receiving European Union approval for Natpara for the indication of hyperparathyroidism in 4Q16.

Vimizim generated $72.6 million during the first quarter of 2016. Strong growth has followed a continuously rising target population for the drug.

According to analyst estimates, BioMarin should earn $278.4 million and $273.5 million in 2Q16 and 3Q16, respectively. That’s a yearly sales growth of 11.1% and 30.9%, respectively.

BioMarin (BMRN) is expected to report 2Q16 earnings on August 4, 2016. “Buy” ratings have increased to 94.7% from 90% on June 29, 2016.