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  • Cytokinetics Is Planning New Phase 3 Trial For Tirasemtiv In ALS Using New Endpoint [View article]
    Valid point.
    Oct 21 08:21 PM | Likes Like |Link to Comment
  • Cytokinetics Is Planning New Phase 3 Trial For Tirasemtiv In ALS Using New Endpoint [View article]
    Absolutely correct.
    Oct 21 02:43 PM | Likes Like |Link to Comment
  • Genervon ALS drug shows encouraging results [View news story]
    In the press release, it said that in the ALS trial that there were 8 patients randomized to drug and 4 to placebo. However, the press release stated that the drug treated patients were compared to historical controls rather than the four placebo patients randomized to the trial. This is very strange. Also, we have only aggregate data on the 8 patients and no baseline or endpoint data on the 8 patients in regard to ALSFRS-r or SVC. This makes it very difficult to interpret the data.
    Oct 21 10:15 AM | Likes Like |Link to Comment
  • Cytokinetics Is Planning New Phase 3 Trial For Tirasemtiv In ALS Using New Endpoint [View article]
    Obviously, tirasemtiv will not cure ALS which is a syndome, not a distinct disease, that may have hundreds of different causes. However, I think that a very significant percentage of drugs treat symptoms of disease. I would offer blood pressure lowering drugs, cholesterol lowering drugs and protein pump inhibitors for ulcers and GERD as interesting examples. The last I checked, these products seem to enjoy widespread usage.
    Oct 21 10:06 AM | Likes Like |Link to Comment
  • Cytokinetics Is Planning New Phase 3 Trial For Tirasemtiv In ALS Using New Endpoint [View article]
    I understand your cynicism. However, it is not a universal rule that once a drug has failed a phase 2 or 3 trial that that there is no hope. Consider Acadia's pimavanserin and Puma's neratinib.
    Oct 21 07:02 AM | 2 Likes Like |Link to Comment
  • Cytokinetics Is Planning New Phase 3 Trial For Tirasemtiv In ALS Using New Endpoint [View article]
    The mode of action of tirasmetiv is to increase muscle contracility. Its function in maintaining or improving respirations is to increase contractive force of muscles involved in respiration. It does not affect the steady deterioration of the motor neurons that are dying and that result in the decrease in muscle contractility. Given the same amount of neuronal stimulation, there is increased contractility. It is treating symptoms and not the cause of the disease.
    Oct 20 01:33 PM | 1 Like Like |Link to Comment
  • Discovery Laboratories: Collaboration With Battelle Is Validation Of Aerosurf's Potential By A Sophisticated Third Party [View article]
    You are correct that many phase 3 trials fail after apparently successful phase 2. However, in the case of Aerosurf we know that lucinactant is safe and effective because it is the active pharmaceutical in Surfaxin. The issue is whether the device can deliver sufficient amounts of lucinactant throughout the lung, As I mentioned earlier, this is more of an engineering than a drug development issue. Also, we know that the capillary aerosol generator drug device combination has been successful in lamb and pig models. All of this suggests to me that if it is established in phase 2b that adequate amounts of drug are delivered that the chances for a successful phase 3 following a successful phase 2b are much, much higher than for a new therapeutic entity. Obviously, nothing is for sure in biology.
    Oct 18 06:23 PM | 3 Likes Like |Link to Comment
  • Discovery Laboratories: Collaboration With Battelle Is Validation Of Aerosurf's Potential By A Sophisticated Third Party [View article]
    Phase 2b will show proof of concept and, if successful, believe me that will move the stock. Why do you believe otherwise?
    Oct 17 10:05 PM | 3 Likes Like |Link to Comment
  • Discovery Laboratories: Collaboration With Battelle Is Validation Of Aerosurf's Potential By A Sophisticated Third Party [View article]
    I can think of no greater insult than being likened to AF.
    Oct 17 04:58 PM | 3 Likes Like |Link to Comment
  • Discovery Laboratories: Collaboration With Battelle Is Validation Of Aerosurf's Potential By A Sophisticated Third Party [View article]
    Take a look at a previous comment in this article. Is starts with Aerosurf is a paradigm -----
    Oct 17 04:57 PM | Likes Like |Link to Comment
  • Discovery Laboratories: Collaboration With Battelle Is Validation Of Aerosurf's Potential By A Sophisticated Third Party [View article]
    Phillip Morris spent hundreds of millions of dollars on finding better ways to deliver nicotine and out of this came the capillary aerosol generator which is the core medical device technology of Aerosurf. Phillip Morris licensed this technology for medical use to DSCO. I think they are entitled to a small royalty. Ironic isn't it that Phillip Morris may contribute to one of the great advances in pulmonary medicine.
    Oct 17 04:56 PM | 2 Likes Like |Link to Comment
  • Discovery Laboratories: Collaboration With Battelle Is Validation Of Aerosurf's Potential By A Sophisticated Third Party [View article]
    Just met with Repligen yesterday. Fundamental story gets better and better. Report out next week,.
    Oct 17 04:52 PM | 1 Like Like |Link to Comment
  • Discovery Laboratories: Collaboration With Battelle Is Validation Of Aerosurf's Potential By A Sophisticated Third Party [View article]
    Aerosurf represents a paradigm shift in the practice of neonatology. The currently marketed animal surfactants and Surfaxin are given as liquid instillates that are administered by an endotracheal tube (intubation) directly into the lungs of a premature infant with respiratory distress syndrome or RDS. Through lessening surface tension in the lungs, surfactant makes it easier for a baby's lungs to expand and contract when used in conjunction with a mechanical ventilator. However, because of the considerable risks associated with intubation and mechanical ventilation, doctors will only give this treatment to babies with the most severe form of RDS.

    There are 360,000 preterm babies born each year and the youngest (23 to 26 weeks gestation) are almost always intubated, given a surfactant and put on a mechanical ventilator. This is about 45,000 babies. However, there are another 110,000 generally older babies (26 to 32 weeks gestational age) who are at risk of RDS that doctors are reluctant to treat in this way because of the risks associated with intubation and mechanical ventilation. They are usually given nasal continuous airway pressure (nCPAP). It essentially uses nose clips to administer air to the lungs and maintain positive pressure in the lungs to prevent them from collapsing during exhalation.

    While nCPAP is much safer, it cannot deliver surfactant to the babies’ lungs. This creates a dilemma for the neonatologist on whether to treat with intubation and mechanical ventilation or with nCPAP. This decision can affect life and death and long term morbidity for the baby. The “holy grail” for neonatologists would be to be able to give a surfactant along with nCPAP. This is the therapeutic target of Aerosurf

    Aerosurf has the potential to make DSCO an exceptional company. In valuing Aerosurf, I think that one should look at the experience of MedImmune and its first key drug Synagis. Aerosurf and Synagis have a great deal in common. Both address disease populations of about 100,000 US babies. Synagis was priced at about $7,500 similar to my projection for Aerosurf. Synagis went on to reach about $1 billion of sales and was the major reason that Astra Zeneca (AZN) paid $15 billion to acquire Medimmune. I am estimating sales of Aerosurf also could reach the $1 billion level in the 2022 or beyond period. This example drives home the ultimate potential for DSCO if Aerosurf is successfully developed.

    The development pathway for Aerosurf is long as for reasons explained in this report, I am estimating approval in late 2017 or 2018. However, I think that the promise of Aerosurf will add consistent value to the stock as the drug progresses through development to commercialization. Investors should keep in mind that the key active pharmaceutical ingredient in Aerosurf is the same as that in Surfaxin. Hence, we go into the phase II and III programs knowing that the drug contained in Aerosurf is safe and effective. The challenge is to show that it can be effectively and safely delivered in an aerosol form. It is more of an engineering challenge than a drug development challenge.
    Oct 17 10:41 AM | 3 Likes Like |Link to Comment
  • Discovery Laboratories: Collaboration With Battelle Is Validation Of Aerosurf's Potential By A Sophisticated Third Party [View article]
    CAG stands for capillary aerosol generator. I parenthesized this in my report (CAG) and SA picked this up as a ticker symbol.
    Oct 17 09:42 AM | Likes Like |Link to Comment
  • Discovery Laboratories: Collaboration With Battelle Is Validation Of Aerosurf's Potential By A Sophisticated Third Party [View article]
    CAG refers to capillary aerosol generator which is the medical device used in Aerosurf. i parenthesized CAG and SA picked this up as a ticker symbol.
    Oct 17 09:36 AM | 1 Like Like |Link to Comment
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