Further to this discussion, for the first time since that 2/29/16 PR, the NSS may be considered to have attained the minimum requirements for an HUD filing - a necessary prerequisite to an HD exemption. While in itself no guarantee of commercial prospect, a well-deserved HDE will definitively boost future enrollment and pay the rent. Another conversion or two in the coming months could easily "ice" any question of probable benefit, and also holds the promise to pick up the tab for ongoing research. The Feds might even see the promise in stem cell integration - from there, it becomes anyone's guess. This one's up in the air, and I intend to play it as a winner...
The following excerpt is from the 2/29 Invivo PR updating the INSPIRE study protocol:
"The OPC for The INSPIRE Study is defined as 25% or more of the patients in the study demonstrating an improvement of at least one ASIA Impairment Scale (AIS) grade by six months post-implantation. Since The INSPIRE Study is designed to enroll 20 patients with complete (AIS A) spinal cord injuries (inclusive of the 5 patients enrolled in the company’s pilot trial) the OPC equates to having five patients convert to any other AIS grade by six months post-implantation. Natural history databases indicate that between 12-16% of patients with complete (AIS A) thoracic injury will spontaneously convert without treatment six months after injury."
Though I am optimistic with results to date, I see no easy path to early approval. Final assessments will be made based upon all the endpoint criteria, and the PR goes on to outline that final HDE consideration-making will not be based solely on AIS conversion data...but also to include data beyond baseline OPC. There's still a long path forward, and this past week's news - though very positive - is far from end of story. The coming year should be loaded with great trading catalysts. I am building a core position, trading as opportunity presents...
"What, in your opinion, is the size of the market for these indications...?"
IMO, very small...if looked at individually. In the aggregate, the tax incentives derived from developing Cabo as an orphan drug candidate could provide substantial savings to overall development costs. Most of the diseases addressed in this NCI-sponsored trial are orphan indications, and Cabo has already seen some limited off label prescription in several of those listed.
Another shotgun signaling approach
I'm around...just in from a long, long road trip....watchin'...
A badly injured mare has taken all of our time of late. She seems to be on the mend...tho still pretty bad.
I'll post as I can... GLTA
The CSD position was posted 7/7, and appears unfilled with last nights updated addition of two finance strategy positions - also of interest. This coming week could prove interesting, as a turning point in finances appears inevitable. Interesting that the short position remains so robust with so many growing avenues to de-risk the long thesis.
This excerpt is from the Sept 2013 EXEL PR introducing Celestial:
"Based on available clinical trial data, the primary endpoint assumes a median OS of 8.2 months for the placebo arm. A total of 621 events provide the study with 90% power to detect a 32% increase in OS (HR = 0.76). Interim analyses are planned once 50% and 75% of events have been observed, respectively."
"What is the anticipated timing on Celestial?"
Despite perceptions of slow recruitment, the primary completion date remains unchanged at 10/16. Company guidance suggests top line results in 2017. Placebo challenge and PD-1 buzz may have slowed initial enrollment, but METEOR success may have produced an uptick in sign-ups due to increased confidence. I begin to suspect an interim reporting is at hand, and it shouldn't come as any surprise after 3 years - should it?
Latest EXEL job postings include shopping for a CSD proficient in interim analysis and data cleansing (excerpted below) To my knowledge, the only pending interim of any consequence is CELESTIAL. Stay ready.
Perform activities related to interim analysis and data base lock e.g. data cleaning clinical data review. Analyze and interpret study data and support preparation of reports for the FDA. Proactively provide feedback on emerging clinical and competitive trends."
"Cabozantinib in the treatment of advanced renal
cell carcinoma: design, development, and potential place in the therapy"
Drug Design, Development, & Therapy July 2016 Grassi et al...
Open access @ DovePress...
"...I see it was noted that one of the analysts following the stock has info on the scripts written thus far for Cabo..."
That'd be Michael Schmidt of Leerink, and he cited IMS Health as his source. Google them or check out a Wikipedia review. They are held in high esteem by the Street, and widely held to be a premier source of market intelligence and prescription data. JMO, but I consider that the TRx numbers might be spot on. See Capt's earlier post...
1.) The MAA transfer of cabozantinib has taken place. In the summary of opinion, the applicant is listed as Ipsen, though - at the time application was actually filed - the applicant was Exelixis. This was anticipated, and that expectation has been met without celebration. An unacknowledged job well done...
2.) Also based upon the succinct wording of the SoO, we can infer that a revised application was made to reflect a stat-sig survival benefit. This is good news, as it indicates that any EC decision for approval in RCC will include an OS benefit on the label. No holes in this story... Despite the seemingly long wait, all our ducks seem to be in a row here. On another note, I just did a quick preview of the IV board: there's a high probability I'll be posting over that way in the near future...once I'm comfortable shifting formats. It appears that Yahoo has lost motivation in addressing the best interests of its clientele, and the posters to this MB are amongst the best with whom I have ever shared thoughts. Some folks never do miss the sweetness of the water till the well runs dry. Adios, muchachos...