Sucampo Pharmaceuticals (SCMP) expects to be generating revenue from three continents -- North America, Europe and Asia -- by the end of 2013 from its two approved prostones products: Amitiza for gastrointestinal diseases and Rescula for ophthalmics. "This is in line with our goal to be a global company," CFO Cary Claiborne said in an interview with BioTuesdays.com. "We are the only company developing and commercializing prostone compounds globally."
Prostones are functional fatty acid metabolites that occur naturally in the body. Prostones work as selective ion channel activators, promoting fluid secretion, enhancing cell protection and acting locally to restore normal function in cells and tissues. Sucampo's two prostone-based compounds have an excellent safety profile, with Amitiza targeting the ClC-2-chloride channel and Rescula targeting the BK-potassium channel. Their therapeutic potential was first identified by Sucampo's founders, Drs. Ryuji Ueno and his wife, Sachiko Kuno. The company has over 580 issued patents on its proprietary prostone technology.
Sucampo's Prostones Are Highly Potent Ion-Channel Activators
Claiborne says that Amitiza has been used for more than seven years in the U.S., with more than seven million prescriptions written for patients suffering from chronic idiopathic constipation (CIC) and irritable bowel syndrome with constipation (IBS-C). "These are big markets with quality of life issues for patients," he said following a presentation at the BIO CEO conference in New York last month. CIC affects 14% to 16% of the adult population globally, he notes, and accounts for 92,000 hospitalizations annually in the U.S. IBS affects 15% of the adult population globally, of which one-third have IBS-C. Phase III studies of Amitiza in IBS-C demonstrated rapid and long-term relief, he adds.
Claiborne says the company is awaiting a decision from the FDA in late April for its supplemental new drug application for Amitiza to treat opioid-induced constipation (OIC), which results from chronic use of opioids for pain relief. Constipation is the most common reason for discontinuation of opioid therapy, affecting between 40% and 80% of patients who take opioids. There is currently no approved pharmaceutical oral product for the two million to 2.5 million moderate to severe OIC patients in the U.S.
In November, the FDA raised concerns that the chronic use of so-called mu-opioid receptor antagonist constipation medicines under development by several companies may cause cardiovascular safety issues. "While other prospective products for OIC are getting delayed by FDA, we are pleased that the FDA granted priority review of Amitiza for OIC," Claiborne suggests, noting that OIC represents the next big revenue driver for the company. Sucampo is in line for a $10-million milestone payment from its North American marketing partner, Takeda Pharmaceutical (OTCPK:TKPHF), on the first sale of Amitiza in the U.S. for the treatment of OIC. Sucampo receives tiered royalties of 18% to 26% on annual net sales of Amitiza by Takeda, allowing Sucampo to recoup some $106 million of its R&D expenses to date.
In Europe, Amitiza was approved for the treatment of CIC in the UK in 2012 and in Switzerland in 2009. Claiborne says the company plans to sell the drug itself in those markets. Last week, the company also announced that it had completed filings in both the UK and Switzerland for OIC. "We also plan to submit filings in other EU markets this year to expand the use of Amitiza in CIC patients," Claiborne adds. In Japan, Sucampo received a $15-million milestone from marketing partner, Abbott Japan, in the fourth quarter of 2012 when it launched Amitiza for the treatment of chronic constipation, targeting primary care and specialist physicians. "Growth of Amitiza sales in Japan is a priority this year," he contends.
Global AMITIZA Approvals and Regulatory Filings
Claiborne says Sucampo plans to take advantage of a U.S. new label for Amitiza, which removed a requirement that women had to obtain a negative pregnancy test prior to starting on the drug. The new label also clarified information about the use of Amitiza in pregnant and/or nursing women and expanded information about the drug's mechanism of action. The company also plans to develop and seek approval for Amitiza in pediatric constipation and develop a liquid formulation of the drug for pediatric and long-term care markets. There is currently no approved prescription medication for pediatric constipation. Sucampo also plans to evaluate the potential of Amitiza for other new indications, including mixed irritable bowel syndrome.
Last month, Sucampo, its partner company, R-Tech Ueno, and Takeda filed a patent infringement lawsuit in the U.S. District Court for the District of Delaware against Anchen Pharmaceuticals and Par Pharmaceuticals related to an abbreviated new drug application that Anchen filed with the FDA to market a generic version of Amitiza oral capsules, in both the 8 mcg and 24 mcg strengths. The lawsuit claims infringement of six patents that are listed in the FDA's Orange Book and scheduled to expire between 2020 and 2027. Last week, Sucampo reported that net income for 2012 jumped to $4.8 million, or 12 cents a share, from a loss of $17.3 million for 2011. Revenue rose 49% to $81.5 million from $54.8 million.
In addition to its 2013 plans for Amitiza and Rescula, the company also updated its prostone pipeline. The company said it expects to complete its oral mucositis Phase Ia trial for cobiprostone in the second quarter of 2013 and to initiate a Phase Ib/IIa trial in the fourth quarter of this year. In addition, it plans to complete a Phase IIa trial for spinal stenosis with its SPI-017 prostone candidate in the fourth quarter.
Sucampo's Clinical Pipeline
In February, Sucampo's development partner, R-Tech Ueno, signed an agreement with the Japan Science and Technology Agency for the development of unoprostone isopropyl for retinitis pigmentosa. The Japanese government will provide the majority of funding for Phase III clinical development costs of the program. Sucampo said it is co-developing unoprostone isopropyl with R-Tech Ueno and may file for FDA approval of the product for retinitis pigmentosa in the future, assuming the trials are successful.
Sucampo also has become an emerging player in ophthalmics, with the recent launch of Rescula in the U.S. for lowering intraocular pressure (IOP) in patients with open-angle glaucoma, which is the most common form of glaucoma, or ocular hypertension. "We have already received positive feedback from many in the ophthalmology and optometry communities regarding Rescula's unique mechanism of action as a BK channel activator," Claiborne says. Rescula is being launched with 40 sales reps, some of whom have moved over from selling Amitiza, he notes, adding that 40 reps can cover 52% of the top prescribing physicians in the glaucoma space.
Glaucoma is a group of ocular diseases with various causes that ultimately are associated with a progressive optic neuropathy leading to loss of vision. "Reduction in IOP is currently the only modifiable risk factor for patients with glaucoma and ocular hypertension," he says, adding that "Rescula is believed to reduce elevated IOP by increasing the outflow of aqueous humor through the trabecular meshwork in the eye." Claiborne explains that Rescula was originally approved in 2000 in the U.S. and was marketed by CIBA Vision. When Novartis acquired CIBA, the drug was returned by Novartis, and Sucampo acquired the rights in 2009. "The cornerstone of our strategy was to have the FDA update the label to reflect our better understanding of how the drug worked," he recalls. That was achieved in December, and Rescula can now be used as a first-line agent or with other topical ophthalmics to lower IOP.
RESCULA: Only Nonprostaglandin That Lowers IOP Throughout Day (12 h) With Excellent Systemic Safety Profile
Even though 67% of the glaucoma market is now generic, Claiborne points out that there is a subset of patients who can't tolerate the side effects of standard of care prostaglandin drugs for IOP, including severe red eye. "Because of that, those patients have to come off prostaglandins and find an alternative treatment," he adds. "That's the subset we're going after. We believe it represents 15% to 20% of patients being treated for glaucoma by prostaglandins. Overall, the glaucoma market is a $3 billion a year market. And we think we stack up well, because our new label reflects the differences in how Rescula works and how prostaglandins work."