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Juxtapid And Kynamro: Race Of Two Orphan Drugs To Market

March 6, 2013

Aegerion Pharmaceuticals’ orphan drug Juxtapid (Lomitapide), an oral once-a-day treatment for Homozygous Familial Hypercholesterolemia (HoFH), beat competitor Genzyme and Isis Pharmaceutials’ orphan drug Kynamro (Mipomersen) to market. Juxtapid’s price of $235,000-$295,000/year is much higher than Kynamro’s $176,000/year. It will be interesting to observe over time, whether Aegerion Pharmaceuticals’ pricing and launch strategy changes due to the competitor’s much lower price. Both companies this week give presentations :

  1. Aegerion Pharmaceuticals’ March 6, 2013 webcast with company updates and 2012 financial results
  2. Isis Pharmaceuticals’ March 6, 2013 webcast at the Cowen & Company 33rd Annual Healthcare Conference.

On March 6, 2013, at 8:30 AM ET, Aegerion Pharmaceuticals hosts a conference call and live audio webcast, presenting a business update of the company and a discussion on the fourth quarter and year-end 2012 financial results.

Here are a few highlights from Aegerion Pharmaceuticals’ presentation :

–  Review of the FDA December 2012 approval of once-a-day capsule, Juxtapid for HoFH :

  1. Boxed warning of potential for liver toxicity
  2. Restricted distribution through Risk Evaluation & Mitigation Strategies (REMS) requiring the patient to have lab or clinical diagnosis consistent with HoFH.

– Review of Juxtapid Launch :

  1. Launches in January 2013 in US
  2. Estimated US # of patients is 3,000
  3. 400+ REMS trained physicians
  4. 85 prescriptions written to date globally
  5. Currently 25 sales representatives with an average 16 years of experience with orphan disease backgrounds
  6. Estimation of 100 academic lipid specialists, 400 speciality lipidologists in community-based setting, & 1,300 cardiologists
  7. Lipid Specialty Managers (LSMs) focus on targeting about 1,800 physicians
  8. Expects to have 250 – 300 patients on Juxtapid globally by the end of 2013
  9. Pricing at $295,000/patient/year.

– Review of patent protection and potential exclusivity extensions for Juxtapid in the US & EU

– EU decision on Juxtapid approval expected Mid-2013 & to launch in the late 3rd Quarter 2013

– Clinical update on Juxtapid for Japanese & pediatric patients

– Expects $15 – $25 million global net revenues for FY 2013.

According to a Boston Business Journal article, Aegerion Pharmaceutials’ stock as of 2:00 PM, March 6, 2013, rose “ …. 90% since the Oct. 18, 2012 news of the FDA advisory board vote, and 20% in just the last 5 days, thanks in part to favorable ratings from analysts …..”. Marc Beer, CEO and Director of Aegerion Pharmaceuticals, says in a statement, “….The initial ramp of our commercial launch is on track to achieve our previously stated guidance of 250 – 300 patients globally on therapy by the end of 2013.”

On the same day Aegerion Pharmaceuticals is giving their financial presentation, their competitor in the HoFH space, Isis Pharmaceuticals is having a webcast at the Cowen & Company 33rd Annual Healthcare Conference. Lynne Parshall, COO at Isis Pharmaceuticals, gives a presentation on the company :

– Kynamro injection receives FDA approval in January 2013 & $25 million milestone payment from Genzyme partner

– Genzyme asks for a re-examination of EMA’s CHMP December 2012 negative opinion for Kynamro & anticipates a new opinion in the 2nd Quarter 2013

– Commercial activities for Kynamro’s launch is underway :

  1. Physicians qualified under REMS
  2. Prescriptions written.

– Reviews extensive Pipeline with 28 drugs.

References

SeekingAlpha.com March 6, 2013 “Aegerion Pharmaceuticals Management Discusses Q4 2012 Results – Earnings Call Transcript”.

Please Note: “View from starting block of a competition swimming pool” by Rufino Uribe [CC-BY-SA-2.0] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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