February 8, 2019 7:46am

During quarterly results i.e. earnings “season” – value is skewed; the forward question is what is the cash position and “runway” status?

News: uniQure N.V. (QURE) updated clinical data in patients treated in its ongoing P2b study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant, for the treatment of patients with severe and moderately severe hemophilia B. Updated data presented at EAHAD show that all three (3) patients have demonstrated increasing and sustained FIX levels after the one-time administration of AMT-061. Mean FIX activity for the three patients at twelve weeks increased to 38% of normal, exceeding threshold FIX levels generally considered sufficient to eliminate or significantly reduce the risk of bleeding events.

Pre-open indications:  1 BUY, 4 SELLs and 3 TRADEs

 

You can lead a horse to water but you can't make him drink' is a proverb which means that you can give investors an opportunity but can’t force them to make it!

Once we get through quarterly LPS (loss-per-share) financial results <notice I didn’t say – earnings season, it will be easier for investors to possibly recognize a short or a near-term view; but my fear is that Q4 numbers aren't going to look much better than the Q3/18 numbers.

 

U.S. stock index futures are falling again further

Dow futures are DOWN -0.58% (-145 points) and NASDAQ futures are DOWN -0.84% (-58 points)

 

U.S. stock index futures were lower, with market participants increasingly worried about a broadening global economic slowdown;

European stocks hovered around the flat line;

Asia markets decline amid fresh concerns over US-China trade

 

Data docket: none

 

Friday’s indications in the pre-market:

The iShares Nasdaq Biotechnology (IBB) is NOT indicating

The SPDR S&P Biotech ETF (XBI) is indicating a NEGATIVE -0.5% DOWNSIDE

The Health Care Select Sector SPDR ETF (XLV) is NOT indicating

The iShares Russell 2000 (IWM) is indicating a NEGATIVE -0.5% DOWNSIDE

 

Henry’omics:

Thursday’s sector losses extended the sell-off into Friday, spurred by uncertainty surrounding the China-U.S. trade talks and “our” universes pessimistic sentiment over quarterly results.

TGIF, maybe Monday will be better for the oversold …

 

Out and about:

uniQure N.V. (QURE updated clinical data in patients treated in its ongoing P2b  study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant, for the treatment of patients with severe and moderately severe hemophilia B.

These data were presented in an oral session at the Annual Congress of the European Association for Hemophilia and Allied Disorders (EAHAD) and show that therapeutic levels of Factor IX (FIX) activity continue to be sustained in all three patients up to sixteen weeks after a single administration of AMT-061.* AMT-061 has been granted Breakthrough Therapy Designation by the US FDA and access to the Priority Medicine (PRIME) regulatory initiative by the European Medicines Agency.

The first patient achieved FIX activity of 48% of normal at sixteen weeks after administration. FIX activity in the second patient was 25% of normal at fourteen weeks after administration, and the third patient achieved FIX activity of 51% of normal at twelve weeks after administration. The second and third patients had previously screen-failed and were excluded from another gene therapy study due to pre-existing neutralizing antibodies to a different AAV vector. Reported FIX activity was measured using an activated partial thromboplastin time (aPTT) assay performed at a central laboratory.

 

Thursday’s evening post, “blood drips from the cracks in the sector. The sector was already on edge as quarterly LPS (loss-per-share) results i.e. earnings began today!”

Last night’s percentage (%) indicators: 

  • Ranged of the 7 upside was +0.29% (HSGX) to +1.10% (ONVO) while the 37 downside ranged from -0.03% (KOOL) to -67.82% (SLDB) with 1 flat close (BSTG).

Volume stat: 

  • 2 out of the 7 upside had higher (than the 3 month average) volume
  • 11 out of the 37 downside experienced greater volume (than the 3 month average)

 

Companies in my headlights – It’s your decision; I provide an idea and context:

uniQure NV (QURE) closed down -$1.10 to $33.18 and has a positive +$0.50 or +1.51% pre-market indication. MT-061 is to give all people living with hemophilia B access to a one-time treatment capable of normalizing FIX activity and eliminating the need for replacement therapy, without the risk of immune responses that require immunosuppression or may lead to a loss of efficacy,” – BUY;

Organovo Holdings (ONVO) closed up +$0.0108 to $0.99 and reported Q3 results with a quarterly loss of $0.06 per share versus the Zacks Consensus Estimate of a loss of $0.07. This compares to loss of $0.07 per share a year ago. These figures are adjusted for non-recurring items although revenue estimates were a miss. The aftermarket indication is a negative -$0.02 or -2.10% - SELL;

Solid Bioscience (SLDB) closed down again -$15.15 to $7.19 after Wednesday’s -$0.70 to $22.34 and has another negative aftermarket of -$0.19 or -2.64% to $9.00 after news of preliminary findings from IGNITE DMD, the P1/2 dose-ascending clinical trial evaluating the safety and efficacy of SGT-001 microdystrophin gene transfer for the treatment of Duchenne muscular dystrophy (DMD). Initial three-month biopsy data showed low levels of microdystrophin protein expression – Maintaining SELL;

Applied Genetic Technologies (AGTC) closed up +$0.01 to $3.14 after Wednesday’s -$0.02 to $3.13 with a quarterly net loss of $4.18 M or -$0.23 per share – Maintaining SELL;

Pluristem (PSTI) closed down again -$0.04 to $1.01 after Wednesday’s -$0.07 to $1.05 as news of a $50 M financing by Jeffries hits the stock – Maintaining SELL;

Where, when and what are the accelerants for these equities:

CRISPRS Therapeutics (CRSP) closed down -$2.85 to $29.78 after Wednesday’s +$0.19 to $32.63, Tuesday’s -$0.14 to $32.44, Monday’s -$0.51 to $31.92 and last Friday’s $31.92. There is a positive +$0.29 or +0.97% aftermarket indication – TRADE;

Editas Medicine (EDIT) closed down -$1.67 to $19.43 after Wednesday’s -$0.04 to $21.10, Tuesday’s-$0.77 to $21.14, Monday’s $21.91 and last Friday’s $21.61, There is a positive +$0.19 or +0.98% aftermarket indication– TRADE;

Intellia Therapeutics (NTLA) closed down -$1.07 to $12.79 after Wednesday’s +$0.07 to $13.86, Tuesday’s -$0.72 to $13.79, Monday’s $14.51 and last Friday’s $14.04. There is a positive +$0.12 or +0.94% aftermarket indication – TRADE;

 

Opinions expressed are those of the author and are subject to change, and not intended to be a forecast of future events, a guarantee of future results, nor investment advice.

Whether information or intelligence is good, bad or somewhere in between; I put into context what is relevant and useful for investors.  All investments are subject to risks. Investors should consider investment objectives.

Henry McCusker, the editor and publisher of RegMed Investors does not hold or have positions in securities referred to in this publication.