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Orphan Drug Companies At The 15th Annual BIO CEO And Investor Conference: Celgene And Onyx

February 17, 2013

This is the first of a series of Orphan Druganaut Blog Posts that will recap the orphan drug companies at the 15th Annual BIO CEO & Investor Conference, held February 11 – 12, 2013, in New York City. This Blog Post reviews two companies that are competitors in the Multiple Myeloma (MM) market, Celgene and Onyx Pharmaceuticals.


On the first day of the meeting, Monday, February 11, 2013, Celgene Corporation’s Chairman & CEO, Robert Hugin, participates in a Fireside Chat (Webcast available) with Joel Sendek, Managing Director and Biotechnology Analyst at Stifel Nicolaus & Company. A Fireside Chat is a discussion between a company and Wall Street analysts, where recent company successes and the company’s future are reviewed.

This Fireside Chat came after several very important announcements in February by Celgene :

  1. FDA approval, ahead of the scheduled PDUFA date, of orphan drug Pomalyst (Pomalidomide) for the treatment of Relapsed and Refractory Multiple Myeloma (RRMM) on February 8, 2013
  2. Revlimid is granted regulatory approval in China on February 11, 2013, for the treatment of RRMM
  3. On February 11, 2013, an application for Revlimid, in patients with relapsed or refractory Mantle Cell Lymphoma (MCL – has FDA orphan drug designation) after prior therapy, is accepted by the FDA, and is assigned a Priority Review with a PDUFA date of June 5, 2013.

Mr. Hugin says that Pomalyst is very complementary to Celgene’s current MM program – Revlimid (Lenalidomide) and Thalomid (Thalidomide), which are 2 orphan drugs currently marketed as MM treatments in the US and internationally. Regulatory approval for Pomalyst is submitted in Europe, and Mr. Hugin is hopeful that an approval will occur in 2013.  He is hopeful that commercialization of Revlimid in China will happen in the middle of the 3rd Quarter 2013. In conclusion, it is an important strategy for Celgene’s future to expand indications and geographic roll-out for Revlimid.

Onyx Pharmaceuticals

On the second day of the meeting, Tuesday, February 12, 2013, Onyx Pharmaceuticals’ Chairman & CEO, N. Anthony (Tony) Coles M.D., participates in a Fireside Chat (Webcast available) with Steve Usdin, Senior Editor at BioCentury.

Onyx Pharmaceuticals’ orphan drug, Kyprolis (Carfilzomib) injection, receives FDA Accelerated Approval in July 2012 for MM treatment for patients who have received at least 2 prior therapies, and have demonstrated disease progression on or within 60 days of completion of the last therapy. Onyx Pharmaceuticals retains complete control in both the U.S. and Europe for Kyprolis. According to Dr. Coles, the global market for MM will grow to $8 – $10 billion by 2016 – 2017. There is currently no cure for MM and thus there is a large unmet need. 10,000 – 15,000 Americans per year, who have failed other treatments, will be eligible for Kyprolis. Kyprolis has more than $62 million in sales since the July 2012 launch. The plan is to expand the Kyprolis MM franchise for other indications: early-stage MM and for refractory patients. Kyprolis is not approved yet in the EU, where there are approximately 50,000 patients compared to the 40,000 patients in the US.  The plan is to have a local partner in the EU for Kyprolis.

Dr. Coles says that the majority of Onyx Pharmaceuticals’ success is from 2 things :

  1. Strong collaboration with patient advocacy groups, such as the multi-year collaboration with the Multiple Myeloma Research Foundation to accelerate personalized medicine approaches for MM
  2. To remain optimistic.

Dr. Coles also discusses orphan drug Nexavar (Sorafenib) which has approval for hepatocellular carcinoma or liver cancer. There is a large unmet need in Asia as shown by the following liver cancer demographics (all approximations) :

  1. US has 15,000 patients
  2. South Korea has 15,000 patients
  3. Japan has 40,000 patients
  4. China has 340,000; > ½ of the global incidences of liver cancer.

A high incidence of liver cancer in Asia is mostly due to the correlation of liver cancer with Hepatitis B and C. Onyx will partner only on a sales basis with Bayer. Nexavar has approval in South Korea, Taiwan, Japan, and China for liver cancer.

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