The data monitoring committee recently conducted an interim analysis (blinded to the company) of this ongoing ALNY (Notes Page) trial. They recommended stopping enrollment at the current 90 patients (the trial was originally planned for 76, then amended to adaptively enroll up to 120). This trial is meant to confirm results from a small phase 2a study that indicated that the RNAi therapeutic ALN-RSV01 lowered the risk of bronchiolitis obliterans syndrome (BOS) in respiratory syncytial virus (RSV)-infected lung transplant patients (50% incidence placebo vs 7% on drug). Note that this drug is administered via nebulizer (not delivered via lipid nanoparticle technology like other ALNY pipeline candidates). The company has previously stated that there is a small chance this trial could form the basis for registration for this indication. Cubist $CBST has the right to opt-in to the program for North American rights following final results from this phase 2b trial – expected mid-2012.
Antares Pharma Announces Issuance of New U.S. Patent Covering LibiGel® (Link)
BioSante Pharmaceuticals Announces Issuance of New LibiGel(R) Method of Use Patent (Link)
These PRs announced the issuance to AIS (Notes Page) of a new Method of Use patent covering the testosterone gel product LibIGel. The US patent (#8,067,399) entitled “Method and apparatus for transdermal or transmucosal application of testosterone“expires December 29, 2028. BPAX (Notes Page) stated that “The patent protects the use of LibiGel in treating FSD and HSDD, and the bottle we used in our clinical work, as well as in the intended commercial product.” I need to do more research to investigate how useful this new IP will really be for LibiGel. As a reminder, BPAX has licensed LibiGel rights from AIS for the US and certain other nations and phase 3 efficacy data is expected in December 2011.
Onyx Pharmaceuticals Announces FDA Acceptance of New Drug Application for Carfilzomib for the Treatment of Relapsed and Refractory Multiple Myeloma (Link)
This is an important event for Ligand $LGND – which acquired a license agreement between Cydex (since bought by LGND) and Proteolix (since bought by Onyx). Carfilzomib utilizes Captisol formulation technology, and as such LGND is owed payments for Captisol material, milestones, and royalties on sales. You can read more details on the LGND stock notes page, but ONXX will pay an undisclosed milestone payment to Ligand on the acceptance of the NDA (a larger milestone would be due on FDA approval). $2m of this milestone payment will be paid to Cydex shareholders via a CVR from the buyout. Based on precedent of other LGND deals, I would estimate that the $2m represents 50-75% of the total milestone. There is no word yet as to the assigned PDUFA date (and therefore whether ONXX was granted priority vs standard review).
Infinity and Mundipharma Extend Global Strategic Alliance (Link)
INFI (Notes Page) is a biotech with an atypical partnership/funding model. They have licensed rights to all of their programs (except HSP90 inhibitor IPI-504) to MundiPharma and Purdue. They receive annual R&D expense payments that fund a somewhat outrageously high cash burn rate. In this regard, investors in INFI don’t have to worry about any near-term fundraising risk, but there is also little in the way of potential partnership catalysts (and we are a ways away from any significant clinical trial data). Todays PR contains two pieces of information. First, INFI announced they had drawn a $50m line of credit from MundiPharma (this had been previously forecast to happen before year-end 2011) – this cash basically will help fund G&A and the HSP90 program into 2014, since R&D costs for the other programs are reimbursed. Second, Mundipharma agreed to provide $50m in funding for the PI3K program for 2013. This is on top of a total of $110m for 2012 (covering PI3K and IPI-926 hedgehog inhibitor programs). The additional 2013 funding for the IPI-926 program will not be determined until after the phase 2 pancreatic cancer trail results and an end-of-phase 2 meeting the with FDA takes place.