PBT 0.00% 4.8¢ prana biotechnology limited

Orphan drug approval...

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    From kadaicher on ymb :
    • kadaicher1 • 1 hour 29 minutes ago
      Repost Orphan drug, Approval, Trial size, Cost, Market Cap and SP
      This time no links. It is obvious Prana may not need to do much more to NDA this, if they get good 2 yr safety data.
      Case Study1

      Approved: 2006
      Rare Disease:  paroxysmal nocturnal hemoglobinuria, a blood disease so rare that it affects only about 4,000 Americans
      Phase l Patients: 11 Cost: $0.02
      Phase II 11 0.04
      Phase III 184 8.7
      Totals 206 $9.3m
      Approved drugs on market 1. Price of treatment per year est $569,000 Most expensive in world.
      Total Revenue $555,146,000
      Profit/Loss $177,536,000

      Case Study2
      Company: NPS Pharmaceuticals NPSP Drug: Gattex Market Cap: $4.9B SP: $45.87
      Approved: Dc 2012
      Rare Disease:  adults with short bowel syndrome (SBS) 10,000 to 15000 in US
      Phase l Patients: 8 Cost est: $.2m
      Phase II 16 .6m
      Phase III 169 8.0m
      Totals 193 $ 8.8m
      Approved drugs on market 1 Pricing 295,000 per year.
      Total Revenue $155,592,000
      Profit/Loss (13,504,000)

      Case Study3
      Company: Seattle Genetics SGEN Drug: Adcetris, Market Cap: $4.03B SP: $32.55
      Approved: Aug 2011
      Rare Disease:Hodgkin lymphoma (HL) and a rare lymphoma known as systemic anaplastic large cell lymphoma (ALCL)
      Phase l Patients: 45 Cost: $1.0m
      Phase II 102 $3.7m
      Phase III 0 0
      Totals 147 $4.7m
      Approved drugs on market 1. Cost of drug $13,500-per-dose
      Total Revenue $269,264,000
      Profit/Loss (62,861,000) Less

      Sentiment: Strong Buy


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      • kadaicher1 • 1 hour 6 minutes ago
        Somehow had the Soliris company stats cut out of my post.
        Company: Alexion Pharmaceuticals Drug: Soliris, Market Cap: $34.89B SP: $175.63
        I got the price below wrong reading another board, but it is still pretty high. Something like .5m per yr.

        This is for the trolls
        "Earlier this month at BioEurope Spring in Turin, Jeremy Springhorn, Vice President Corporate Development at Alexion, talked in some detail about eculizumab’s clinical path. In the early days of development, Alexion explored potential therapeutic applications in large and lucrative indications such as psoriasis and rheumatoid arthritis. The antibody has been in clinical development since 1996 but it failed in 5 clinical trials before demonstrating the efficacy signal in paroxysmal nocturnal hemoglobinuria (PNH). Once the right indication was identified, the clinical development picked up pace and Soliris was approved in 2007 by the FDA. Second indication to gain approval was atypical hemolytic uremic syndrome (aHUS), an ultra-rare genetic disease that leads to kidney failure, damage to other vital organs and premature death."
        Prana is also exploring different applications and with over 1000 different analogs of the drug. Prana has the advantage of being to tweak MPACs in different directions, and the technology is already there. Less

        Sentiment: Strong Buy


      • kadaicher1 • 1 hour 19 minutes ago
        For those who do not know, Prana already have Orphan Drug designation for PBT2 in HD, which is the next trial.
        Frankly I think some of those orphan prices are a disgrace. There are valid arguments for pricing orphan drugs high, but Soliris may have overdone it. That would be something like $2M per patient for life.
        One thing is that looking at the financial statements, all revenue, then some, is being pumped back into further drug development.
        That would probably be the case with Prana also, but it doesn't seem to hurt the market Caps of those Orphan companies, which all only have one drug on the market. Less

        Sentiment: Strong Buy

        • 1 Reply to kadaicher1
        • kadaicher1 • 55 minutes ago
          From Forbes on the subject of pricing new Orphan drugs.
          NPS Pharmaceuticals announced that it was pricing Gattex, its drug for short bowel syndrome, at $295,000 per patient per year, about triple what analysts on Wall Street expected. It is the fourth drug approved in 2012 to be priced at more than $200,000 per patient per year. The others are: Kalydeco for cystic fibrosis (maker :Vertex Pharmaceuticals); Elelyso for Gaucher’s disease (Protalix and Pfizer); and Juxtapid for homozygous familial hypercholesterolemia (Aegerion Pharmaceuticals.) That represents 10% of the drugs approved by the Food and Drug Administration last year and a 44% increase in the number of such high-priced rare disease drugs on the market.

          Francois Nader, the chief executive of NPS Pharmaceuticals, spoke with me this morning about how he and his team priced Gattex. Here is a roadmap for how a medicine can be priced at an annual cost equivalent to a house. These bullet points reflect NPS’ reasons for pricing the medicine as it did. Less

          Sentiment: Strong Buy

          • 1 Reply to kadaicher1
          • kadaicher1 • 52 minutes ago
            Medical value: Gattex is the first non-surgical treatment for short bowel syndrome, disease which requires patients to get their nutrition parenterally, or through a vein. A sixth of the patients on the drug can be weaned from this parenteral nutrition. “this is a product that significantly improves the daily living of these patients,” says Nader. “They feel liberated and for them it is a life-changing event.”
            Direct costs: Parenteral nutrition can on its own cost $100,000 a year. But Nader says there are other costs that can be as high as $500,000 per patient, and that Gattex is expected to reduce those costs, which include hospitalization, as well, although that has not been shown in clinical studies. Short bowel syndrome also leads to increased mortality.
            Indirect costs: Gattex improves quality of life and allows some patients to return to work, so that they no longer receive disability payments and become productive members of society.
            The orphan drug premium: NPS found that there may be as few as 3,000 short bowel syndrome patients in the country, fewer than expected, after it polled companies that provide parenteral nutrition. Drugs for rare diseases cost more. “Until recently orphan products were called neglected diseases. I think that society and payers are willing to pay a premium for these diseases that were ignored.”
            Will insurers pay? NPS says it polled insurers, patients, and doctors about what they would be willing to pay, and Nader says that they would have been able to pay a price even higher than what it is charging. Nader says that if the existing short-bowel treatment, Zorbtive, were used on an annual basis, it would cost $290,000 per year t00.
            How, though, does anyone pay for such a medicine? The answer is that pricing of these rare disease drugs, known as ultra-orphan drugs in the biotech industry, are not paid for the way that most medicines are. Nobody buys them out of pocket, and the manufacturer refuses to grant discounts to insurers or to Less

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