PropThink: RPTP To Rise On Proven Strategy & Upcoming FDA Decision
(Thomson Reuters ONE) -
By Ivan Deryugin
Raptor Pharmaceutical (NASDAQ:RPTP) is developing treatments for rare diseases
and has a compelling pipeline positioned to take advantage of unmet medical
needs. The company has a strong chance to secure approval for its lead product
candidate Procysbi (previously known as RP103) in several months for the
treatment of nephropathic cystinosis, and clinical data is solid, offering
patients a more convenient and safer choice relative to existing treatments.
Raptor is working on several new indications for Procysbi, and if the treatment
is approved on its PDUFA date of April 30th, the company is likely to quickly
gain a leadership position in the nephropathic cystinosis treatment market;
analysts see annualized sales of up to $153 million (Raptor's current market
capitalization is around $271 million). Investors who add to or initiate
positions in the stock should see profits leading up to approval as the average
price target for Wall St. analysts is $9.00 a share, indicating up to a doubling
of the stock on positive news and PR103's commercial potential.
Big advantages and pay offs for treatments of ultra-rare disorders. Developing
drugs for ultra-rare diseases continues to offer significant opportunities for
niche biotech companies and their shareholders. A focused strategy aimed at
filling an unmet need in a very small patient population provides key advantages
such as patient and physician advocacy, faster and more efficient regulatory
review, government funding and special market exclusivities, and nearly
guaranteed health insurance coverage. Companies like Alexion Pharmaceuticals
(NASDAQ:ALXN), which sells Soliris for the treatment of PNH and aHUS, have
proven that the business strategy can generate substantial returns. Sarepta
Therapeutics (NASDAQ:SRPT), for instance, with its focus on treating Duchenne
Muscular Dystrophy, outperformed the broader markets YTD by roughly 400% despite
that its candidates are still in development. Raptor Pharmaceutical, with a
development program geared towards ultra-rare disorders and an upcoming FDA
decision on its lead candidate, is a prime example of a company positioned to
benefit from a substantially underserved market.
Procysbi: A Promising Treatment with an Upcoming PDUFA Date
Raptor is a development-stage biotechnology company with several assets in its
pipeline, the most notable of which is Procysbi. The drug is a treatment for
nephropathic cystinosis, an extraordinarily rare disease with only 2,000 cases
worldwide and just 500 in the United States. The disease, which causes the amino
acid cysteine to accumulate in the body's cells, affects 1 in 100,000-200,000
newborns each year and leads to poor growth, kidney failure, brain damage, and
often-premature death. Nephropathic cystinosis is currently treated primarily by
cysteamine therapy, marketed as Cystagon by Mylan (NYSE:MYL). Procysbi, a
delayed release version of standard cysteamine therapy, aims to make treating
this disease far easier with half the dosing frequency that its predecessor
requires and fewer side effects. Procysbi is designed to be delivered only twice
a day, versus the four daily doses required for Cystagon, including one in the
middle of the night. It's no wonder then that providers report high instances of
patient noncompliance when using Cystagon, particularly as this population is
largely children and adolescents. Continue reading.
See "RPTP To Rise On Proven Strategy & Upcoming FDA Decision" in full at
PropThink.com.
About PropThink
PropThink is an intelligence service that delivers long and short trading ideas
to investors in the healthcare and life sciences sectors. Our focus is on
identifying and analyzing technically-complicated companies and equities that
are grossly over or under-valued. We offer daily market coverage, weekly feature
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Source: PropThink via Thomson Reuters ONE
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Datum: 28.12.2012 - 16:58 Uhr
Sprache: Deutsch
News-ID 215866
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