As good a theory as any. I'd be surprised if we didn't have results prior to May 1, and if it goes past May 1 it bodes ill (digging deep into secondary endpoints to find some positive signal).
Appears they missed the ASCO late breaking abstract deadline. Any other summer conferences they could present the data at? If so, they may try to release the top line results before the abstract deadline for that conference.
I was against the shelf when announced near 20, but have no complaints raising $60 million when diluting only 10% at 28.5. Great job by management. Net loss was only $15 m last year so this cash will go a long way. Odd that RBC dropped off as manager. Would like to see them use the $$$ to in-license another trigger technology in case UNA doesn't pan out.
It was odd that they elected to use only $60m of the $100m shelf; it's even odder that it's been 2 days yet the offering hasn't been priced. Either (1) the runup to $30 caught everyone off guard and they are pressuring folks who may have indicated interest at $25 or so to go to $30; or (2) the other $40m is going towards an equity stake for a new partner, and they are lining up the timing on the two components. Regardless, it's going to be an interesting couple of days....Also, we could see initiation of HCC PLK1 Phase II at any time. Would like to see it have more than 50 participants, making it much more robust than the GNET and ACC Phase II trial.
Not the end of the world if they already have institutions lined up to buy 5 million shares at $20. If this offering goes down like the ARWR offering then we'll fly to $25. If it goes down like the last one we'll regress to $12 or $15. The cash does give them flexibility. One thought - pay to convert the UNA license obtained from Marina (now Arturus's to license) to an exclusive license. Otherwise ARWR can swoop in and get a UNA license (and they already have a license so some of TKMR's delivery tech).
Take a look at the SMA natural history studies on pubmed and how the hammersmith results change over time. This shows the results related to the hammersmith score are good. The SMN protein level data is even more encouraging - the drug is working. And these measurements should be unassailable unlike SRPT's dystrophin measurements in muscle tissue. Isis would have never been able to obtain this protein level data without Biogen. Some think Isis gave up too much by partnering with Biogen, but this protein level data is worth it.
I know I'm going against the prevailing sentiment, but I'm against raising cash at this time. We don't need it - right now TKMR has enough cash to last it until sometime between late 2016 and mid-2017. That gets them past Ebola Phase I results and later animal testing, ALDH2 and HBV Phase I results, and the nomination of at least one if not two candidates. Let's let them fight over our 19 million shares at this point. If initiation of the HCC Phase II and mRNA data (or better yet, an mRNA deal) pushes us to $30 a share in the next couple of months, then ring the register for 2-3 million shares to get us another 2-3 years of cash. Otherwise, stay the course. I don't see any big RNAi failure (ALNY, ARWR, etc) in the next 3-4 months that would bring TKMR down with it. Another thought: will TKMR be able to get access to the chimp ARWR ran HBV tests on? If so, data from that testing could be yet another near term driver.
I've read where OGXI gets a 15-25% royalty on custirsen sales and also read where Isis gets a 5.5%-7% royalty on net sales which may drop to 4-4.5% in 2017. Here is my question: Is Teva paying OGXi 15-25% royalties AND paying Isis 4-7% royalties, or is Isis' royalty coming out of OGXI's royalty, reducing OGXi's royalty to somewhere around 10-20%?
The Muscular Dystrophy Association's annual Clinical Conference is March 16-19 in Chicago. Good chance Isis presents the topline data from the ISIS-SMN Phase I/II trials at this conference. MDA has provided significant funding both for the Isis compound itself and the development of the Hammersmith scoring system that will measure whether the Isis compound is successful.
The key to accelerating the share price will be (1) getting more institutional ownership; right now there is a wide chasm between ARWR's institutional ownership and TKMR's; and (2) partnering products; let's keep half the products wholly owned for home run potential, but partner the other half to de-risk and offset development costs so we don't have to do a bunch of secondaries. The HBV candidate would be a good one to partner. If Phase I data in chronic HBV patients is favorable could do ex-US partnership for large sum.
In its annual report ALNY reported $5.6 million in payments from MON last year. The annual payments MON makes to TKMR before it exercises its option are likely to be similar. TKMR's annual R&D expenses are around $24 million at this point. So, assuming a 75% margin on work it does under the MON contract, the MON payments will cover around 17% of TKMR's R&D costs. Great source of non-dilutive funding.
I have some $$$ in ARWR, but not nearly as much as in TKMR. I am pretty conservative, and am concerned that if there is a hiccup in ARWR's HepB program it could fall precipitously. Whereas with TKMR if any one of TTR02, Ebola, or PLK1 fail it has plenty to fall back on and won't drop too far. Also, note everyone thinks Isis is going after HepB based on recent patents and announcements related to their deal with GSK. On Nov. 26 Isis announced they were beginning clinical trials on an anti-viral designated GSK3 (presumably the HepB drug), and at the JPM conference a slide stated the GSK3 drug was in a Phase 1/2 trial that will readout in 2014. So it is possible Isis will announce multi-does data on their HepB drug in infected patients before ARWR even starts their multi-dose HepB trial.
No real news since the initiation of the GSK Phase I trial on Nov. 26. Should have data on SMN and EIF4E in the near future. They kept guiding the SMN data would come "late this year or early next year". I don't think "early next year" is March. Maybe they are quiet because they are working on a deal? Ex-US rights for APOCIII?
Dicerna guides that it will file an IND for a cancer drug this year and an orphan drug next year. Debuts with a $560m market cap. Tekmira may have 2 compounds approved by 2017 (TTR and Ebola), has a cancer drug in phase II that will readout by mid-year, guides it is filing two additional INDs this year and 1-2 more INDs in 2015, just signed a huge ag collaboration, and has a $240m market cap. I know where I'd rather be invested.
RNAi seems much safer than chemicals that have been used in the past. Once Monsanto completes long term testing fears should be allayed.
Ranked in order of likelihood event will occur:
- Decision on $5m payment related to ALNY-VSP
- Phase I/II data on TKM-PLK1
- Phase I data on TKM-Ebola
- ALNY Phase II data on TTR-SubQ (if inferior to patisiran could really move TKMR)
- Initiation of animal testing on TKM-Ebola
- File IND for TKM-ALDH
- File IND for TKM-HepB
- Initiation of Phase III for TKM-PLK1
- Partner LNP technology
- Expansion of biodefense project to include Marburg
- Additional Mariqbo data
- Ramp up of Mariqbo sales (unlikely)
- Disclosure of additional candidates INDs will be filed for in 2015
I wish there was some more detail on the deal with Monsanto. For instance, can TKMR get payments in the form of milestones and/or royalties after the option exercise, or is $86 m a cap on all compensation they will ever be paid by Monsanto? It's definitely a good deal, but if they can get decent milestones and royalties after the exercise then its a great one (and better than ALNY's).
Didn't get it done by January 1, but I'm thrilled with the ag deal. The up front payment itself should extend TKMR's cash runway to 2017. And the option payments and milestones should extend it out even more.