CAMBRIDGE, Mass.—Biopharmaceutical company Aegerion Pharmaceuticals Inc. has entered into a definitive agreement with AstraZeneca to acquire Myalept (metreleptin for injection), an orphan product indicated to treat complications of leptin deficiency in patients with generalized lipodystrophy (GL).
Under the terms of the deal, Aegerion will render an upfront payment to AstraZeneca of $325 million to acquire the global rights to develop, manufacture and commercialize Myalept, subject to an existing distributor license with Shionogi that covers Japan, South Korea and Taiwan. The transaction does not include the transfer of any AstraZeneca employees or facilities.
Myalept is a recombinant analogue of human leptin. In conjunction with diet, it is currently indicated as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
Approved by the U.S. Food and Drug Administration in February 2014, Myalept is the only product approved in the United States for the treatment of GL. The product has orphan drug designation in the United States, European Union and Japan. Aegerion plans to evaluate its options to expand Myalept’s commercialization beyond the United States in the near term, planning to seek European Medicines Agency approval in GL with a potential Marketing Authorization Application filing. It is also evaluating the development and regulatory pathway for potential expansion into severe partial lipodystrophy indications.
Myalept is currently available to GL patients in the United States through AstraZeneca’s initial launch efforts. Aegerion expects a seamless transition of the product from company to company, as well as for patients currently on therapy. Aegerion anticipates that its current infrastructure and expertise in the commercialization of rare disease products will allow it to complete the launch in January 2015, assuming the closing occurs in early January as planned.
“Myalept is highly synergistic with [Aegerion’s] current operations across the entire organization, including sales force call points, patient support and global regulatory activity,” says Amanda Murphy, manager of investor relations and public relations at Aegerion. “It was a rare opportunity to acquire an FDA-approved asset with potential for near-term revenue contribution and positive operating income to support growth.”
Aegerion anticipates the launch of Myalept in the United States to require minimal additional resources, thanks to the exceptional level of synergy with the company’s current operations and many of the sales force targets surrounding its lead product Juxtapid.
“The primary call point for Myalept is approximately 3,200 adult endocrinologists treating generalized lipodystrophy patients, a subset of which are also treating homozygous familial hypercholesterolemia patients, and, in some cases, prescribing Juxtapid,” says Murphy.
In addition, the company expects there to be GL patients being treated within other specialty practices, including cardiology. Because Myalept is approved for pediatric GL patients, it also may be marketable to approximately 800 pediatric endocrinologists as well.
In an investor conference call on Nov. 6, Craig Fraser, president of U.S. and International and Global Supply for Aegerion, said the company has calculated a price for Myalept at approximately $325,000 per patient per year, assuming 100-percent compliance with therapy and an estimated average utilization of doses. Upon launch, the company anticipates a one-time price increase that more closely aligns the pricing with the severity of the disease and the extremely limited patient population. Aegerion has in place a comprehensive patient services program that provides support, including assistance with the reimbursement process for patients on therapy.
Aegerion estimates Myalept may garner a peak of $200 million to $250 million annually in net product sales, based on several key market assumptions, including successful U.S. commercialization, European approval of the product in GL and accurate prevalence estimates.
“As is the case in many rare diseases, often the greatest challenge is in understanding the dynamics of a new market, including the number of patients and their treatment journey,” Murphy tells DDNews. “Aegerion has completed a significant amount of diligence in this regard; however, there are significant learnings that come with first-hand experience in the field interacting with patients and their physicians.”
GL is an ultra-rare, progressive disease that Aegerion estimates affects roughly one in one million people. The disease results in abnormal or degenerative conditions in the body’s adipose tissue, including loss of subcutaneous fat, ectopic lipid deposition in various organs (including the liver and muscle) and hyperphagia and significant fatigue caused by leptin deficiency. The disease can also cause metabolic anomalies—such as severe insulin resistance, hypertriglyceridemia, diabetes mellitus and hepatic steatosis—that may resist conventional therapies. Uncontrolled, the disease can lead to severe organ damage and reduced life expectancy.
Myalept offers an alternative to the current standard of care for GL patients that involves treating the hyperglycemia and hypertriglyceridemia associated with the disease. These patients’ hyperglycemia is typically treated with insulin, often in high doses, and usually with limited effectiveness because of a high degree of insulin resistance. The patients’ hypertriglyceridemia is typically treated with triglyceride-lowering agents with modest effectiveness, but is often inadequate to eliminate symptoms.
“Generalized lipodystrophy is a rare condition with significant unmet medical need that can impact every aspect of a patient’s health,” Luke Miels, executive vice president of global product and portfolio strategy for AstraZeneca, said in a news release announcing the deal. “Myalept is the first therapy to provide a real option for treating complications of this disease, and we are pleased that patients will benefit from its progress under Aegerion as a company with expertise in rare diseases.”
“In the area of orphan drugs, patients often benefit from a company with a track record and singular focus in rare diseases,” Murphy says. “Aegerion has in place a comprehensive patient services program which provides support, including assistance with the reimbursement process for patients on therapy.”
