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

February 4, 2013

UPDATED 02/11/2013List of Presenting Companies & Webcast Links

The BIO CEO & Investor Conference takes place in New York City, February 11 – 12, 2013. The event is the “largest investor conference focused on established and emerging publicly traded biotech companies.” The event features presentations by pharmaceutical companies and biotechs. CEOs from several companies will be in the spotlight during public interviews with industry analysts, “Fireside Chats”. The following are some of the orphan drug companies with orphan drug catalysts that one should look for in the presentations (all times are ET (New York)) :

FEBRUARY 11, 2013 (Monday)

* 1)  Celgene Corporation’s Chairman & CEO, Robert Hugin, 8:00 AM :

–  Recent Abraxane Phase III results for pancreatic cancer

–  Apremilast for Behcet’s Disease receives January 2013 FDA Orphan Drug Designation (ODD)

–  FDA PDUFA date 02/10/13 for Pomalyst (Pomalidomide) for Relapsed and Refractory Multiple Myeloma (RRMM).

2)    NPS Parmaceuticals (10:30 AM ET)Dr. Francois Nader, President & CEO :

– Gattex launch and pricing for Short Bowel Syndrome (SBS)

– Natpara status for Hypoparathyroidism.

FEBRUARY 12, 2013 (Tuesday)

* 1)  Onyx Pharmaceutical’s Chairman & CEO, N. Anthony Coles, MD, 7:45 AM) :

–  Marketed results of Kyprolis for Multiple Myeloma (MM)

–  Nexavar study results for thyroid cancer.

2) Exelixis (8:30 AM) :

     – Commercial launch of newly approved Cometriq for Medullary Thyroid Cancer (MTC)

– Additional indications for building the Cometriq franchise. 

** 3)   Orphan Drugs: Making Rare Diseases Rarer, 9:00 AM – 9:55 AM :


  1. Marc Beer, CEO, Aegerion Pharmaceuticals
  2. John F. Crowley, Chairman & CEO, Amicus Therapeutics)
  3. Pamela Gavin, Senior VP, National Organization for Rare Disorders (NORD)
  4. Hans GCP Schikan, PharmD, CEO, Prosensa
  5. Alvin V. Shih, MD, COO, Pfizer Rare Disease Research Unit.

4)  ZIOPHARM Oncology (10:00 AM) :

– Palifosfamide Phase III updates for Soft Tissue Sarcoma (STS) & Small Cell Lung Cancer (SCLC).

5)  Aegerion Pharmaceuticals (10:30 AM) :

– Juxtapid launch for Homozygous Familial Hypercholesterolemia (HoFH).

6)  Raptor Pharmaceutical (11:00 AM) :

–  Update on Procysbi’s FDA PDUFA date which is extended by 3 months from January 30, 2013 to April 30, 2013.

7)  Prosensa Therapeutics (11:30 AM) :

–  Recent January 2013 FDA ODD for exon-skipping portfolio for Duchenne Muscular Dystrophy (DMD)

– Drisapersen Phase III for DMD.

8)  Protalix BioTherapeutics (11:30 AM) :

–  Update status on Elelyso for Gaucher Disease.

9)  Soligenix (3:00 PM) :

–  Update on SGX203’s FDA “Fast Track” designation for mild-to-moderate pediatric Crohn’s Disease (CD)

–  Update on 4 orphan drugs in Pipeline.

* 10)  BioMarin Pharmaceuticals’ CEO, Jean-Jacques Bienaime, 3:30 PM :

– GALNS (BMN-110) Phase III for Mucopolysaccharidosis (MPS) IVA (Marquio A Syndrome)

– PEG-PAL Phase II for Phenylketonuria (PKU).

*  “Fireside Chats” which “feature candid discussions between Biopharma CEOs and Wall Street analysts. Hear about recent company successes, what keeps the C-suite up at night, and where the industry’s leading companies are headed in 2013”

** Therapeutic Workshop

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

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