Vertex Pharmaceuticals Inc

Vertex Pharmaceuticals (VRTX) reported its 2Q16 earnings on July 27, 2016. VRTX surpassed Wall Street analysts’ projections and reported revenue of $431.6 million.

Alnylam Pharmaceuticals (ALNY) is currently developing three investigational therapies—Patisiran, Revusiran, and ALN-TTRsc02—for the treatment of patients with hereditary ATTR amyloidosis.

Through the acquisition of Raptor Pharmaceuticals, Horizon Pharma (HZNP) will gain access to Procysbi and Quinsair and expand in the orphan drug space.

Biogen’s (BIIB) R&D expenses for 4Q15 were $542 million, or 19% of its total revenue, including a $60 million payment to Mitsubishi Tanabe.

In 4Q17, Pfizer’s BeneFix reported revenues of $150 million, which represents a ~11% decline YoY and a ~1% decline quarter-over-quarter.

On January 10, 2018, Vertex Pharmaceuticals announced that Orkambi has secured regulatory approval from the European Commission to treat CF patients ages six to 11 years.

Analysts expect Incyte Corporation’s revenue to rise ~26.7% to $413.8 million in 4Q17 compared to $326.5 million in 4Q16.

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

Since demand from the target population for prophylaxis is rising rapidly, Bioverativ’s Alprolix and Eloctate are expected to see solid demand trends in 2017.

Bioverativ (BIVV) expects its 2017 GAAP and non-GAAP operating margins to fall 38.0%–42.0% and 43.0%–47.0%, respectively.

In 1Q17, Bioverativ reported revenues close to $259.0 million, driven by its focus on a commercial strategy for its hemophilia products and optimal cost management.

The acquisition of True North Therapeutics has paved the way for Bioverativ’s (BIVV) entry into cold agglutinin disease (or CAD).

Vertex Pharmaceuticals’ (VRTX) drugs, Orkambi (lumacaftor/ivacaftor) and Kalydeco (ivacaftor), are capable of treating around 30,000 cystic fibrosis (or CF) patients.

In March 2017, Vertex Pharmaceuticals (VRTX) announced promising results from two phase-3 clinical trials, Evolve and Expand.

In 2016, Vertex Pharmaceuticals (VRTX) reported revenues of around ~$1.7 billion, which reflected a whopping 65% YoY (year-over-year) growth.

Alexion Pharmaceuticals has planned five clinical trials in 2017 to test the potential of investigational next-generation C5 antibody, ALXN1210, as a treatment option for complement-mediated diseases.

To promote the use of Spinraza for SMA, Biogen (BIIB) has been actively educating and creating awareness for the drug among physician and patient communities.

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.

Alexion (ALXN) has been serving the atypical hemolytic uremic syndrome, or aHUS, market for the past five years.

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

Vyvanse is Shire’s leading ADHD drug, constituting 80% of the company’s ADHD portfolio. In fiscal 2015, Vyvanse earned the company $1.7 billion.

While mature biotechnology companies with drugs in multiple disease segments earn an average of 30%–40% EBITDA, margins of companies targeting only rare diseases can vary due to unique business models.

As part of its research and development, Vertex Pharmaceuticals (VRTX) has been actively exploring new cystic fibrosis (or CF) drugs as well as other indications for its existing drug Kalydeco.

On July 2, 2015, the FDA (U.S. Food and Drug Administration) approved Orkambi, a combination drug of lumacaftor and ivacaftor, for treating cystic fibrosis (or CF) patients.

Vertex Pharmaceuticals’ (VRTX) business model includes revenues in three areas: products, royalties, and collaboration. There’s significant variability in royalty and collaboration revenues.