May 14, 2020 8:01am

The challenge is to consider what can be confirmed, indicated or reversed to tip the sector’s share pricing “direction”

Pre-open indications:  7 BUYs and 3 SELLs

Quarterly results: BioLife Solutions (BLFS)

News: Ultragenyx Pharmaceutical (RARE) announced positive longer-term safety and efficacy data from the first three cohorts of the ongoing P1/2 study of DTX301. Solid Biosciences Inc. (SLDB) presented clinical biomarker data from its SGT-001 micro-dystrophin gene therapy clinical trial for the treatment of Duchenne muscular dystrophy (Duchenne). uniQure N.V. (QURE) presented new preclinical data on its gene therapy candidates AMT-150 for Spinocerebellar Ataxia type 3, AMT-190 for Fabry disease, and AMT-180 for the treatment of Hemophilia A.

What I provide is an “intelligence daily” to inform the RegMed/cell and gene therapy sector and its investors utilizing indication and warning analysis. Check the BOTTOM LINE <read more>

Dow future are DOWN -0.61% (-142 points), S&P futures are DOWN -0.55% (-16 points) and NASDAQ futures are DOWN -0.60% (-55 points)

 

U.S. stock futures are falling over concerns of reawakening the U.S. economy after a sell-off as pessimism sets back-in;

European stocks retreated after an economic warning from the U.S. Fed’s Chairman Powell as the pan-European Stoxx 600 dropped -0.8%;

Asia Pacific stocks fell as a data release showed employment in Australia falling in April. Japan fell, South Korea’s Kospi also declined along with Mainland Chinese and Hong Kong’s while Australia closed lower.

 

Daily sector news:

  • Ultragenyx Pharmaceutical (RARE) announced positive longer-term safety and efficacy data from the first three cohorts of the ongoing P1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. Six of nine patients in the study have responded to the gene therapy (three females, three male), including all three patients in Cohort 3 who are now confirmed responders. The three previously disclosed complete responders, who have discontinued all ammonia scavengers and liberalized their diet, remain clinically and metabolically stable after longer-term follow-up.
  • uniQure N.V. (QURE)  presented new preclinical data on its gene therapy candidates AMT-150 for Spinocerebellar Ataxia type 3, AMT-190 for Fabry disease, and AMT-180 for the treatment of Hemophilia A. SCA3, also known as Machado-Joseph disease, is caused by a CAG-repeat expansion in the ATXN3 gene that results in an abnormal form of the protein ataxin-3. People with SCA3 experience brain degeneration that results in movement disorders, rigidity, muscular atrophy and paralysis. There is currently no treatment available that slows the progressive course of this lethal disease. AMT-150 is a one-time, intracisternally-administered, AAV gene therapy incorporating the proprietary miQURE™ silencing technology that is designed to halt ataxia in early manifest SCA3 patients.
  • Solid Biosciences Inc. (SLDB) presented clinical biomarker data from its SGT-001 micro dystrophin gene therapy clinical trial for the treatment of Duchenne muscular dystrophy (Duchenne) on May 13 at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting, which is being held virtually. In addition, data from studies characterizing novel adeno-associated virus (AAV) vectors engineered for muscle gene delivery were presented in a poster session.

 

Daily Docket: seasonally adjusted employment in Australia fell by 594,300 people in April as compared to March, according to data released by the country’s Bureau of Statistics.

  • The total 2.981 million new claims for unemployment insurance brought the coronavirus crisis total to nearly 36.5 million, by far the biggest loss in U.S. history. Last week’s count was revised up by 7,000 to 3.176 million.

  • Economist expect a total of 2.7 million of Americans to have filed unemployment insurance during the week ending May 9;
  • A record of 20.5 million jobs were lost in April alone as the coronavirus-induced economic shutdown tore through the economy, sending the unemployment rate skyrocketing to 14.7%.

 

Henry’omics:

Investors got a dose of reality, yesterday … it comes down to support – that was lacking and resistance – that refused to prevent the decline for it had no tools to fortify its walls.

Remember, the movement of the roller-coaster, one always and up where they started;  May’s ticket got punched with  (2) declining sessions followed two (2) advancing sessions after a negative session and three (3) positive sessions started by May beginning with a negative closing session – that’s 4 negative and 5 positive closes.

Despite the sharp drop on Wednesday, the S&P 500 remains more than 28% above its March 23 low. The Dow has also rallied more than 27% since then as shares of major tech companies surged.

 

The issue of the market’s push-pull continues to be expectation of the risk-reward ratio for equities as slippage enters the danger zone and the economic response of re-opening businesses!

 

Wednesday night’s title: “winners and losers depend on timing of entry and escape as the Fed’s chair Powell warns of “downside risks”, which I have been touting for weeks; which also opens an opportunity to BUY as one moves past the slippage … s l o w l y. Think of the “limbo” dance, how low will they go before a re-entry.”

  • The NASDAQ closed DOWN -139.38 points (-1.55%);
  • the IBB closed down -1.11% and XBI also closed down -1.99%;
  • Wednesday closed negative at 3/31 and 1 flat;
  • Volume INCREASED with 2 out of the 3-upside having higher than the 3-month average volume and remained LOW with 11 out of the 31-downside having higher than the 3-month average volume. The percentage (%) of the 3-upside were +0.70% (MDXG) to +6.67% (MESO) while the 31-downside ranged from -0.37% (STML) to -8.61% (CLLS).    

Q2:

  • May registered 4 negative, 5 positive closes
  • April registered 10 negative, 11 positive closes and 1 holiday.

Q1:

  • March registered 11 negative, 10 positive closes and 1 neutral close.
  • February registered 9 negative, 9 positive closes, 3 vacation days and 1 holiday.
  • January registered 9 negative, 10 positive closes and 2 holidays.

 

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

Sell:

  • bluebird bio (BLUE) closed down again -$1.04 after Tuesday’s -$3.37 and has a negative -$0.73 or -1.31% aftermarket indication with the FDA initially declining to move forward on the application for their experimental multiple myeloma treatment so, they’re re-submitting data. https://finance.yahoo.com/news/bristol-myers-squibb-bluebird-bio-210000241.html ;
  • Fate Therapeutics (FATE) closed down -$1.31 after Tuesday’s +$1.38 and has a negative -$0.44 or -1.60% aftermarket indication;

BUY:

  • BioLife Solutions (BLFS) closed down -0.33 after Tuesday’s +$0.34 and has a positive +$0.10 or +0.67% aftermarket indication reporting earnings with revenue declared in a release of May 14’s Q1;
  • Cellectis SA (CLLS) closed down -$1.29 and has a positive +$2.40 or +17.52% aftermarket indication, too many rumors to substantiate why but, numbers and percentage speak;
  • Editas Medicine (EDIT) closed down -$0.97 after Tuesday’s -$1.95 and has a positive $0.49 or +2% aftermarket indication;
  • Ultragenyx Pharmaceutical (RARE) closed down -$2.21 after Tuesday’s -$1.07 and Monday’s +$3.52 and announced (Wednesday after close) positive longer-term safety and efficacy data which initiated a positive +$0.67 or +1.05% aftermarket indication;
  • Adverum Biotechnologies (ADVM) closed down -$1.41 and has a positive +$0.35 or +2.01% aftermarket indication;
  • Pluristem (PSTI) closed down -$0.57 and has a positive +$0.09 or +0.98% aftermarket indication; traders are always stripping upside value yet, PSTI rides a COVID-19 treatment “wave”.
  • ReNeuron (RENE.L) closed down again -$13.50 after Tuesday’s -$7.00 with a positive +$8.50 US pre-indication;
  •  

Maintaining Sell into Strength:

  • Athersys (ATHX) closed up +$0.045 after Tuesday’s down -$0.21,Monday’s +$0.43 and Friday’s +$0.21 with a lot of volume to sell into as trials will take time plus a negative as far as taking PPP funds from government will simultaneously raising $57.6 M. ATHX has a positive +$0.02 or +0.65% pre-market indication;

 

The BOTTOM LINE: Sentiment has been scratched and injured … too may times to count on both hands in these last few months.

I keep talking about indications that appear and disappear while numerous signals are sorted through.

Reducing uncertainty can take many forms and involve many types of analysis, but I always try to enhance understanding of what is known, what is happening, where share pricing … might drive value enhancement or declines considering the trajectory of indication “signals”.

I also try to balance the analysis of multiple assessments to challenge, inform, influence and determine potential of outcomes ...known, unknown and those unappreciated.

MY goal is to make investors more knowledgeable about the possible trajectory of share pricing; take it or ignore it – I’m right a lot more than I’m wrong!

 

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.