Biotech Values

[ghmm]

cUniQure (QURE):

I've been buying the stock of late. They have really been beaten down again since releasing Hemophilia B data in July, to the point that their EV is only ~40M.
1) The data to date in their Hemophilia B program has shown about 5% expression and some ALT elevations. Sparks has shown ~30% (21-42) and no ALT elevations which is perhaps why people are writing off QURE's Hemophilia B program. I find it interesting BMRN (which also uses AAV5) had more than a log increase in expression with a 3 fold increase in dose. Also BMRN's ALT elevations have been managed with prophylactic steroids (with apparently no loss of expression). The High dose in QURE's Hemophilia B program (4x the low dose) should have up to 6 months data by year end. The analysts notes I've seen have expectations in the 10-12% expression range. I would not be shocked for it to be substantially above that. One thing in QURE's favor is they in a sense have much longer data in Hemophilia B. The same Gene Cassette (though different vector) was used for the St. Jude trial (published in NEJM http://www.nejm.org/doi/full/10.1056/NEJMoa1407309?af=R&rss=currentIssue#t=article ), so they have now gone over 5 years if nothing else it demonstrates durability and safety.
2) The Sanfilippo B program may actually be their most advanced with encouraging one year data presented last September ( http://www.uniqure.com/news/267/182/Positive-Topline-Results-Announced-from-Phase-I-II-Trial-in-Sanfilippo-B-Syndrome-Patients-Using-uniQure-s-Novel-AAV5-Based-Gene-Therapy.html ) and 2.5 year data expected in Q1 '17. If the data continue to hold up they could start their pivotal program in '17.
3) The BMS partnership for up to 10 targets, primarily focused in cardio vascular area (S100A1 is the first target) is perhaps the most lucrative GT partnership to date with BioBucks up to 2.3B if all 10 targets are developed (140M to date). The animal data (pigs) had 1 year survival at 90% vs 10% in placebo. The company has said testing in man could start as early as 2017 or 2018. BMS has selected 4 targets. By target 6 they can up their stake to 14.9% and ultimately go to 19.9%
4) Other programs in/near human studies include parkinson's, Hemophilia A and Huntington's.
5) Though they use a much higher dose than Spark they do their own manufacturing with 2 facilities (insect viral line). They've said they have enough capacity to treat all the CV patients from one year of production (one reason sited for BMS choosing them).
6) They do in house research aside from trying to improve their vectors and payloads they've done work on retreatment which I found interesting.

- Biomarin also uses an insect viral line. The BMS partnership complicates things but I've wonder if at current market price QURE would be attractive to someone like them wanting to expand into GT and needing quite a lot of manufacturing capacity/expertise.

- An interesting comp is a transformed company Plasma Therapeutics who is now a Gene Therapy company with a new name of Abeona Therapeutics. There lead program in Sanfilippo Type A just announced dosing their third patient. No other GT program has dosed a patient. They do have some interesting areas they licensed programs (Sanfilippo B, Batten disease and a CRISPR program for Franconi Anemia). They appear to be a company which licenses programs and dose little (if anything) in house. Their EV is about 120M!