Biomarin Pharmaceutical Inc

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

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.

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.

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

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.

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

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

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

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.

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.

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.

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.

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.

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.

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

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.

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

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%.