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Orphan Drug Companies At The J.P. Morgan Healthcare Conference

January 7, 2013

       Several orphan drug companies will be presenting to investors this week, January 7 – 10, 2013, at the 31st Annual J.P. Morgan Healthcare Conference in San Francisco. The Conference Schedule and Webcasts are available here by registering. The following are some of the orphan drug companies with orphan drug catalysts that one should look for in the presentations :

JANUARY 7, 2013

1) Celgene orphan drug catalysts (Webcast at 11:00 a.m. ET) :

– Abraxane Phase III results for pancreatic cancer

– FDA PDUFA date for Pomalidomide for relapsed/refractory multiple myeloma on or about 02/10/13.

2) Onyx Pharmaceuticals orphan drug catalysts (Webcast at 9:30am PT (12:30pm ET) :

– Marketed results of Kyprolis for Multiple Myeloma (MM)

– Nexavar study results for thyroid cancer.

3) Vertex Pharmaceuticals orphan drug catalyst (Webcast at 10:00 AM Pacific Standard Time) :

– Kalydeco’s long-term plan for their Cystic Fibrosis (CF) franchise.

4) BioMarin Pharmaceuticals orphan drug catalysts at (Webcast at 10:30 a.m. PT) :

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

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

5) Biogen Idec orphan drug catalyst at (Webcast at 5:30 p.m. ET)  :

– Recent announcement of failure of Dexpramipexole for ALS.

6) Ariad Pharmaceuticals orphan drug catalyst (Webcast at 4:30 p.m. PT) :

– Iclusig launch.

7) Prosensa orphan drug catalyst (Presentation at  4.30pm PST) :

– Drisapersen Phase III for Duchenne Muscular Dystrophy (DMD).

JANUARY 8, 2013)

1) Aegerion Pharmaceuticals orphan drug catalyst (Webcast at 5:00 pm ET) :

– Juxtapid launch for Homozygous Familial Hypercholesterolemia (HoFH).

JANUARY 9, 2013

1) Spectrum Pharmaceuticals orphan drug catalyst (Webcast at 7:30 AM PT) :

– Belinostat Phase III results for Peripheral T-Cell Lymphoma.

2) NPS Pharmaceuticals orphan drug catalysts (Webcast at 09:00 PT) :

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

– Natpara status for Hypoparathyroidism.

3) Isis Pharmaceuticals orphan drug catalyst (Live Audiocast at 1:30 p.m. PT) :

– Kynamro update for Homozygous Familial Hypercholesterolemia (HoFH).

4) Ziopharm Oncology orphan drug catalyst (Webcast at 2:30 p.m. PT) :

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

5) Sarepta Therapeutics orphan drug catalyst (Webcast at 4:30pm Pacific) :

– Eteplirsen catalysts for 2013 for Duchenne Muscular Dystrophy (DMD).

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