January 8, 2019 1:13pm

The sector “feels the profiting” after one piece of news from Novartis’ (NVS) of its sickle cell disease (SCD) drug secured the FDA’s breakthrough therapy designation for its ability to prevent debilitating painful vaso-occlusive crisis for patients affected by the group of inherited red blood cell disorders that typically afflict those of African ancestry. Global Blood Therapeutics (GBT) dives -$1.60 or -3.67% to $41.97.

Trading and low volume runs their course as the NASDAQ dips negatively and flips back

… With an overbought “situation” and profit-taking swing


As I had stated last evening, “the sector is on fire but, will volatility induce a price to pay? Confidence perceived is not always value achieved!”

The 6 W’s: Who, what, where, when, why and what of it …


WHY the decline --- investors are left vulnerable by the lack of headlines from the JPMorgan healthcare conference!

Pre-open post, “time to take some or a little profit off the table. Whenever I see my +15% threshold met and yesterday’s double digit gains as many companies trot out their priorities for 2019 but, can they be met (?) – Execution has two connotations! Start building some “dry powder” for post Q4 LPS (loss-per-share) earnings season.”

The iShares Nasdaq Biotechnology (IBB) indicated a POSITIVE +0.48% UPSIDE which had dipped -0.58% and is trading +0.36% at the mid-day.


When the Advance/Decline Line (A/DL) of 45 covered companies:   

  • The open was positive with the A/DL to 33/9 and 3 flat;
  • The first hour dived negative with an A/DL of 18/25 and 2 flats;
  • The second hour continued to fall with an A/DL of 18/24 and 2 flats;
  • The mid-day stayed negative with an A/DL of 18/24 and 3 flat;

Some CRISPERs got “hit” after an upward flight: CRSP -$0.40 or -1.18% to $33.82, EDIT -$0.10 or -0.40% to $24.83 and NTLA -$0.43 or -2.89% to $14.27 – still trading equities!


bluebird Bio (BLUE -$2.27 or -2% to $111.37) took a hit after announcing that it is developing a plan to sell gene-replacement therapy with annual payments contingent on continued effectiveness finding a way to handle the expected seven-figure cost of its experimental gene therapy: paying on installment.

Alnylam Pharmaceuticals (ALNY +$2.77 or +3.39%) rose after announcing its preliminary Q4/18 global net product revenues for its lead drug, Onpattro, and provided additional updates on the product’s commercial launch. However, shares of ALNY fell 36.7% in the past year compared with the industry’s decline of 18.8%.

  • The unaudited Q4/18 global net product revenues of Onpattro were $11-$12 million, with more than 200 patients receiving treatment with commercial Onpattro in the US and EU, since launch. This reflects strong patient and physician demand, and very good commercial execution by ALNY’s U.S. and EU teams. About 550 patients worldwide, including patients on commercial drug, patients in clinical studies and those in the company’s global Expanded Access Program (EAP), were being treated with Onpattro. In 2019, the company expects to continue its global launch of Onpattro and potentially expand the label for the drug to include ATTR amyloidosis patients with cardiomyopathy.


Leading the downside: SAGE (-$6.95 or -5%), BLUE (-$1.84 or -1.62%), RGNX (-$1.29 or -2.75%), GBT (-$1.90 or -4.37%) and BLFS (-$0.96 or -7.31%) while the “upsiders”: ALNY (+$2.86 or +3.5%), BMRN (+$1.66 or +1.84%) and RARE (+$0.54 or +1.21%) while RENE.L (+$1.50 or +$3.06%) rebounded

 While AST, VYGR and BSTG remain flat enjoying being “splat” versus a hammering.

I keep talking about uncertainty as many fear the trading of the recent highs.


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.