Aegerion Reports On Their First Three Months in the Orphan Drug Market


Aegerion management team have had a  busy couple of days. Yesterday they provided their company’s first quarter earnings results and that was followed today with a presentation at the Needham Healthcare Conference. These two presentations were significant since they are the first reports of how the company is doing since launching its first drug in January 2013.

mar beer.jpgDuring a webcast accompanying the first quarter results, CEO Marc Beer highlighted the company’s success in launching their drug – Juxtapid (lomitapide) – for the treatment of homozygous familial hypercholesterolemia (HoFH).  

In the three months the drug has been available in United States, Mr. Beer said the company has worked aggressively with their sales team to ensure that the physicians understand HoFH and how to treat it with Juxtapid. This process involves FDA required REMs training that takes 20-40 minutes and the company is encouraged by the enthusiastic interest physicians have shown. Mr. Beers stated that these physician education programs are establishing Aegerion as a respected source of information in the cardiology community. Aegerion is also working with clinicians and payers to ensure that patients can get the drug prescribed and they are also providing patient education to make sure patients understand the importance of compliance.

Using this slow but meticulous approach, the company stated they now have 75 new patients currently prescribed Juxtapid since being launched in January 2013. Further, the re-orders for patients are on track and dropouts to date have been minimal. The company is working diligently and urgently to increase patient numbers.

The HoFH Market 

The company has estimated there are 3000+ HoFH patients in the U.S. and while the 75 patient population currently on Jutapid seems small, both Mr. Beers during the first quarter report webcast and Mr. Fitzpatrick, Chief Financial Officer during the Needham Healthcare Conference  webcast stated that the process of getting patients properly on Juxtapid takes 3-4 months. This is due to five hurdles the company had to overcome to get the patient on the therapy. They include: 1) enrolling physicians on REMS and receive; 2) engage and support patients after the prescription;  3) help confirm converge and reimbursement 4) initiate treatment; and 5) optimize dose and track patients.

At present, Aegerion has indentified 1800 U.S. clinicians who treat the majority of HoFH patients (70-80%) and are focusing their educational material on that group to allow the company to have the most efficient way to reach the majority of HoFH patients. Using this approach, the company expects to have 250-300 patients on therapy by the end of 2013 with net year end product sales of  $15 - $25 million. Furthermore, the company expects to gain approval in Europe later this year and they are also working at approvals elsewhere (i.e., Japan) as well as starting the process to expand Juxtapid’s indication (to include pediatric population).

The Finances

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While the company remains cautiously optimistic and realistic in their long term goals, they continue to function in the red. Net product sales for the first-quarter ended March 31, 2013 were $1.2 million while total expenses were $19 million. For the first-quarter ended March 31, 2013, net loss was $18.1 million, or $0.64 per share, compared with a net loss of $11.7 million, or $0.55 per share, for the same period in 2012.

During his presentation, Mr. Fitzpatrick stated that with their dedicated sales staff, expansion globally with increased indication, the company is hopeful that Juxtapid can eventually reach global sales of $500 million.

Safety and Efficacy of Juxtapid to Treat Patients with HoFH

HoFH is a rare genetic lipid disorder resulting in an accumulation of low-density lipoprotein (LDL-C) in the blood. Patients with untreated HoFH have extremely high LDL-C levels, typically between 400 mg/dL and 1,000 mg/dL. These patients are at severely high risk of premature cardiovascular events, such as heart attack or stroke. Mr. Fitzpatrick noted in the webcasts that most patients tend to die at an early age and that standard lipid lowering drugs tend to be ineffective. Further, many require expensive LDL apheresis (mechanical blood filtration) every 1-2 weeks to lower their LDL levels.  In contrast, the Juxtapid pill provides a safe and effective means to lower LDL levels in HoFH. As stated in a previous article and published in The Lancet, Cuchel et al conducted a single-arm, open-label, phase 3 study in 29 adults with HoFH. At the end of the 26 week efficacy phase, LDL cholesterol was reduced by 50% (baseline mean = 8.7 mmol/L vs week 26 mean =4.3 mmol/L; p<0·0001). Concentrations of LDL cholesterol remained reduced by 44% at week 56 (p<0·0001) and 38% at week 78 (p<0·0001). Gastrointestinal symptoms were the most common adverse event. Mr. Fitzpatrick stated that the company is working to address this adverse event to minimize patient discomfort.

It should be noted that in addition to Juxtapid, the FDA recently approved Isis/Genzyme’s  Kynamro (mipomersen) and patients with HoFH  now have two drugs to choose from. 

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