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.
The company released the secondary endpoint ON SCHEDULE- I don't know if they were trying to pump up share price.
Yes, I can and will recommend this stock, which is priced for failure on primary end point when in fact the chances of success are quite high. Keep in mind that the company picked the group with the best response, secondary AML, in two ph 2 studies. Furthermore, as indicated in the press release, remission rate is highly correlated with PFS and OS data.
Despite great advances in several hematologic malignancies such as CML, CLL, multiple myeloma and lymphomas, there has been no significant improvement in AML therapy in over 30 years. CPX-351 holds
orphan drug status and has fast track designation from the FDA, and the study has been supported by the Leukemia and Lymphoma Society. Even if the OS data in early 2016 fails to be +, I think the drug still stands a good chance of gaining approval based on the improved rate of CR. so far demonstrated. CR is needed to perform bone marrow transplant, which if successful, offers the best chance for a durable remission in AML. The drug could certainly garner a few hundred mil in revenue per year, which would be huge for this company with such a tiny market cap..
Also keep in mind that Heron has a second CINV drug, HTX-019, in clinical development. The company says HTX-019 could be submitted for approval in the second half of 2016. Heron is also working on a post-surgical pain drug, HTX-011, which is likened to Pacira Pharmaceuticals' (PCRX) commercially successful Exparel. I spoke with one significant investor in Heron who believes HTX-011 is the company's "sleeper hit" pipeline product.
Significantly improved response rate and overall survival in 60 with refractory disease or early relapse seems assured based on data from EHA, and the drug almost certainly will be used in off label cases as well.