December 18, 2019 9:09am

Show me the share pricing reflects; validate the clinical path forward – no maybes

Pre-open indications: BUY – RENE.L, SLDB, GBT and RARE on news, caution is still an investment key word

News: Solid Biosciences (SLDB) gained 22% in pre-market trading after two patients who received doses of its experimental Duchenne muscular dystrophy (DMD) treatment showed that the therapy "has the potential" to benefit them. It also said that an adverse event reported by another patient in a clinical study has been resolved. Global Blood Therapeutics (GBT) has entered into a $150 million term loan agreement with Pharmakon Advisors LP, a leading global life sciences investment firm and manager of the BioPharma Credit funds. It’s still debt… a positive and a negative perception. Ultragenyx Pharmaceutical Inc. (RARE has sold to Royalty Pharma for $320 million a royalty right due to Ultragenyx from Kyowa Kirin Co., Ltd for the net sales of Crysvita® (burosumab) in the European Union (EU), the United Kingdom (UK), and Switzerland. Crysvita has received conditional marketing authorization in Europe for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency.  

Welcome to my world…


Does the accuracy of guidance, rumors and “runways” really set you off – selling frenzies are on the horizon?


Dow futures are UP +0.10% (+29 points), S&P futures are UP +0.09% (+3 points) and NASDAQ futures are UP +0.11% (+10 points)


U.S. stock index futures are staying with small percentage leanings on Wednesday;

European stocks were mixed following U.K. Prime Minister Boris Johnson’s vow to block an extension of EU trade talks beyond 2020, reviving fears of a “cliff-edge” Brexit;

Asian Pacific markets were also mixed, with Japanese shares leading losses after data revealed a sharp fall in exports.



Stability and sustainable are the key words for “our” universe of cell and gene therapy companies.

I “ain’t” feeling it!


At this point or day in time (December and end-of-year coming) – what is safe = NOTHING.


Tuesday night’s post’s title:  “sector breaks down as profiteering circles the upside. Strength was sold into as the overbought reversed to the oversold with moderate volume. It’s a question of the levels of share pricing risks that are acceptable.”

  • The NASDAQ closed UP +9.13 points (+0.10%);
  • The IBB closed down -0.62% and the XBI closed down -0.07%;
  • The range of the 16 upside was +0.03% (VCEL) to +15.28% (CLLS) while the 22 downside ranged from -0.14% (BMRN) to -10.91% (RENE.L); 
  • 7 out of the 16 upside had higher than the 3 month average volume;
  • 9 out of the 22 downside had higher than the 3 month average volume;

Review the content of Q4’s market:

  • December register 6 negative and 6 positive closes … so far
  • November registered 1 holiday, 12 positive and 8 negative close;
  • October registered 10 positive, 1 neutral and 14 negative closes;


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

Pre-open indications: SLDB, GBT and RARE - otherwise I am staying put


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.