The key point from FDA is that CVOT w\ill NOT be required for approval! Docs are highly likely to use this ORALl drug, whicht will have a much lower price then Praulent or Repatha off label for the larger market of primary prevention.
One of the greatest challenges in treating patients with AML is the very low white count which is almost always present; the low white count markedly increases the risk of sepsis. It is certainly no surprise that a further reduction in white blood count APPARENTLY caused by selinexor would lead to an increase in the incidence of sepsis. The numbers involved are very, and this may turn out to be a statistical fluke. In any case, it was very appropriate for KPTI tto reduce the dose, Keep in mind that the outlook for patients with AML, particularly, ederly patients, is very poor. There has been no significant improvement in the treatment for AML in over 30 years and there is clearly a very large unmet need. I don't believe that the reduction in seliexor dosage is a major problem in the development of the drug. The huge drop in the price of the stock appears to me to make it a compelling buy, which I have done. It is not uncommon for adjustment of dosage in phase 1/2 studies and this is the exact purpose for early phase trials. The huge unmet need for a new drug for AML is likely to make the FDA more willing to accept a drug with even small improvement of the outcome for patients with AML
It is a shame that the FDA decided to make a "case" out of Valor. The entire study of all age groups just barely missed statical significance, and the benefit in pre-defined sub-groups patients 60 and patients with resistant disease or early relapse is quite clear. Personally, I see nothing wrong with pre-defined sub-group analysis. In fact, Novartis got approval for paninobast for multiple myeloma despite a negative review by the physician review panel. Nonetheless. what is done is done. However,I think it is highly likely that vosaroxin will be approved in the EU and Japan where this more consideration of the totality of resuts. EU and Japan are not an insignificant market. Vosaroxin is also being studied in the US by very prominent investigator initiated trials for front line therapy of AML and myelodysplastic syndrome. So there is still hope for a US approva in other indications then relpsed AML l. In addition, SNSS has a drug which looks pretty good as a pan Raf inhibitor for solid malignancies. Now at a market cap of $75 mil, I thin SNSS represents a good speculation.