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.
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.
Sage Therapeutics’ (SAGE) portfolio includes products under development for the treatment of life-threatening central nervous system (or CNS) disorders.
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.
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.
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.
Ionis Pharmaceuticals’ (IONS) Volanesorsen is a drug for the treatment of familial chylomicronemia syndrome (or FCS) and familial partial lipodystrophy (or FPL).
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.
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.
A total of 20 analysts were analyzing BioMarin Pharmaceuticals in July 2017. Six analysts recommended a “strong buy,” while nine analysts suggested a “buy.”
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 […]
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.
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.
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.
BioMarin’s (BMRN) early-stage pipeline includes BMN-270, a gene therapy program for hemophilia A and vosoritide, which is indicated for achondroplasia.
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.
Sanofi’s Specialty Care franchise consists of rare disease drugs, MS (multiple sclerosis) drugs, and oncology drugs and generated ~$3.3 billion in 1H16.
With increased M&A activity in the pharmaceuticals and biotechnology space, it wouldn’t be surprising if Roche Holding (RHHBY) acquired BioMarin (BMRN).
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.
With the continued uptake of Orkambi along with the label expansion efforts, Vertex Pharmaceuticals’s (VRTX) top line in fiscal 2016 is expected to reach $1.7 billion–$1.8 billion.
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.
BioMarin plans to file a Biologics License Application with the FDA for Pegvaliase, which is indicated to treat phenylketonuria, by the end of fiscal 2016.
