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In The Spotlight: Ra Pharmaceuticals

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Shares of Ra Pharmaceuticals Inc. (RARX) are up nearly 22% so far this month compared to the 8.5% gain of the iShares Nasdaq Biotechnology ETF (IBB) index during the same period.

Ra Pharma is a clinical-stage biopharmaceutical company focused on developing drugs for the treatment of serious diseases that are caused by excessive or uncontrolled activation of the complement system, a critical component of the immune system.

The Company's lead drug candidate is RA101495 that is being developed for convenient self-administered subcutaneous injection for the treatment of paroxysmal nocturnal hemoglobinuria.

Paroxysmal nocturnal hemoglobinuria, or PNH, is a rare, chronic, life-threatening, blood disorder where red blood cells are mistakenly attacked and destroyed by the complement system. The only approved drug for the treatment of PNH is Alexion Pharmaceuticals Inc.'s (ALXN) Soliris.

A phase II clinical program for RA101495 SC in PNH patients is underway. Initial data from this program was reported in June 2017, additional interim results in December 2017, and top line results were reported in February of this year.

Based on the top line results of the phase II program, the Company is planning to conduct a phase III program of RA101495 SC in PNH in the second half of 2018.

The treatment potential of RA101495 is also being explored in generalized Myasthenia Gravis (gMG), and a phase II trial in this indication is underway.

Myasthenia gravis, or MG, is a rare, complement-mediated, autoimmune disease that causes weakness in the skeletal muscles. It can occur at any age but most commonly begins for women before the age of 40 and men after the age of 60. Last October, Alexion's Soliris was greenlighted by the U.S. regulatory agency, becoming the first new FDA-approved treatment for patients with gMG in more than 60 years.

Ra Pharma's phase II trial of RA101495 in generalized Myasthenia Gravis (gMG) is of 12-week duration, and is designed to enroll approximately 36 patients. As of June 5, 2018, 19 patients were dosed in the trial.

Top-line data from the phase II generalized Myasthenia Gravis trial is expected in the first half of 2019.

In a bid to evaluate RA101495 in multiple complement-mediated renal indications, such as atypical hemolytic uremic disorder (aHUS) and lupus nephritis, the Company initiated a phase 1b clinical trial of RA101495 SC in patients with renal impairment in January of this year.

Enrollment in the phase 1b clinical trial of RA101495 SC in aHUS was completed recently, and data due mid-2018 is awaited.

Atypical hemolytic uremic disorder is a chronic, ultra-rare, and life-threatening disease which leads to sudden, catastrophic, and life-threatening damage to the kidney, brain, heart, and other vital organs, and premature death. For this life-threatening disease too, Soliris is the only FDA-approved drug.

In addition to the clinical programs of RA101495, Ra Pharma is pursuing discovery and preclinical programs to treat a variety of ocular, renal and inflammatory diseases. The Company, in collaboration with Merck & Co. Inc., is also developing an oral peptide for a non-complement cardiovascular target.

Alexion's Soliris, approved for the treatment of three diseases, for which Ra Pharma is also developing a drug, is the world's most expensive drug. According to pharmacy benefits firm Prime Therapeutics, the wholesale acquisition cost of Soliris is more than $700,000 for the first year of therapy. Soliris raked in net product sales of $3.14 billion in 2017 compared to $2.84 billion in 2016.

If all goes well as planned, Alexion will be making a new addition in its PNH drug arsenal early next year as the Company has submitted a Biologics License Application to the FDA for approval of ALXN1210, an investigational long-acting C5 complement inhibitor, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).

Will RA101495, if approved after clearing the clinical trial and regulatory hurdles, prove to be on par with Soliris? Data to be presented in the coming months and years will give an idea about how Ra Pharma's RA101495 will stack up against Soliris.

Key Numbers...

Accumulated deficit: $139.7 million
Cash on hand: $109 million
Shares Outstanding: 32.29 million
3 month average volume: 213.65K

(Data sourced from Yahoo finance)

Shares of Ra Pharma have traded in a range of $4.78 to $17.90 over the last 1 year. The stock closed Thursday's trading at $12.09, up 9.31%. ALXN closed the day's trading at $134.29, up 2.42%.

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