April 20, 2022 5:05pm

By means of yesterday’s short-lived high and today’s low volume melted showed the Icarus metaphor of danger

News: Ultragenyx Pharmaceutical (RARE +$0.62) the first patient has been dosed in a clinical study for the treatment of Osteogenesis Imperfecta (OI) in patients ages 5 to <26 years of age. The pivotal Orbit study is a P2/3 study of setrusumab (UX143), an investigational anti-sclerostin fully human monoclonal antibody in development for the treatment of OI in children and adults with OI sub-types I, III and IV.

Pre-open indications: 8 HITs and 4 MISS

What I provide is an intelligence daily. RMi outlines a daily bell weather of “indications” about what you need to know; it’s what could happen or materialize after the opening and what might happen during the session.

Who else is tracking a broad grouping of cell and gene therapy equities – can you afford to not read facts supported by real numbers?

If I have learned one thing as a former research analyst, venture and public fund investor now a journalist; it is that your life and your bank account are largely tied to your knowledge of price movements and questioning pricing targets.


The Dow closed UP +249.79 points (+0.72%); the S&P closed DOWN -2.75 points (-0.06%) while the Nasdaq closed DOWN -166.59 points (-1.22%)



Indexes were mixed on Wednesday as traders recognized some failures of Q1 earnings results scaring investors away from buying ahead of earnings’ reporting.

As treasury bond yields turned positive for the first time in two years, investors are now re-assessing their appetite for stocks amid a mixed first quarter earnings season. <TheStreet>

Earnings seem to be the only catalysts, as the cell and gene therapy sector is preparing their reporting; so far, dates are listed in the Bottom Line at end of this post.

News (continued): The Orbit study builds on the findings of the 12-month P2b ASTEROID study, which demonstrated that treatment with setrusumab resulted in a clear, dose-dependent and statistically significant effect on bone formation and bone density at multiple anatomical sites among adult participants with OI,” said Camille Bedrosian, M.D., chief medical officer at RARE. “The Orbit Phase 2/3 study is designed to study fracture risk reduction in a younger population, where the risk of fracture is higher and where we can potentially have the greatest impact on their future health.”


52-week low:

  • Beam Therapeutics (BEAM) at $42.64,
  • Homology Medicine (FIXX) at $1.91,
  • Intellia Therapeutics (NTLA) at $54.91
  • Precigen (PGEN) at $1.60

Unusual volume spike:

  • Verastem Oncology (VSTM) +$0.03 with 1.755 M shares traded, after Tuesday’s-$0.17 with 2.66 M shares traded after Monday’s +$0.06 with 5.505 M shares after Thursday’s +$0.24 to $1.77 with 30 M shares traded


RegMed Investors’ (RMi) pre-open: “I am still a believer in the Icarus myth and the danger of dramatic upside flight on low volume wings. Don't get too excited by one good day” … https://www.regmedinvestors.com/articles/12396


RegMed/Stem/Cell and Gene Therapy’s 35 covered equities’ Advance/Decline (A/D) lines: what happened …

  • Wednesday opened negative at 4 up/30 downs and 1 flat, flipped positive at the mid-day at 18/14 and 3 flats, ending with a negative close of 15/20,


Pre-open Indications: 8 HITs: <Biostage (BSTG -$0.48 with 1,131 shares traded; Pluristem (PSTI +$0.02), uniQure NV (QURE -$0.08), SELL into Strength: Vericel (VCEL +$1.61), Verastem (VSTM +$0.03); Downside indication: Beam Therapeutics (BEAM -$0.93), Editas Medicine (EDIT -$0.41), Intellia Therapeutics (NTLA -$2.20)> 4 MISS < CRISPR Therapeutics (CRSP -$1.38), Ionis Pharmaceuticals (IONS +$2.04), Ultragenyx (RARE +$0.62), Voyager Therapeutics (VYGR +$0.55)>


Key Metrics:

  • Wednesday - Sector volume was LOW with 2 of the 15-upside having higher than the 3-month average volume with LOW volume of 1 of 20-downside having higher than the 3-month average volume;

The iShares NASDAQ Biotechnology (IBB) and the SPDR S&P ETF (XBI) indicators:

  • Wednesday, the IBB was up +0.53% and the XBI was up +0.22%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Wednesday was down -1.05 points or -4.91% at 20.32


Jumping with share pricing momentum (10 of 15):

  • Alnylam Pharmaceuticals (ALNY +$6.75 after Tuesday’s +$4.52 and Monday’s -$10.66),
  • Ionis Pharmaceuticals (IONS +$2.04 after Tuesday’s-$0.05 and Monday’s +$0.04),
  • Vericel (VCEL +$1.61 after Tuesday’s +$2.19),
  • Global Blood Therapeutics (GBT +$0.97 after Tuesday’s +$0.67 and Monday’s -$1.42),
  • Regenxbio (RGNX +$0.72 after Tuesday’s +$0.78),
  • Ultragenyx (RARE +$0.62 after Tuesday’s -$0.34 and Monday’s -$9.96),
  • Voyager Therapeutics (VYGR +$0.55 after Tuesday’s +$0.53 and Monday’s +$0.09),
  • Sage Therapeutics (SAGE +$0.39 after Tuesday’s +$0.63 and Monday’s -$1.43),
  • AxoGen (AXGN +$0.28),
  • Chinook Therapeutics (KDNY +$0.27 after Tuesday’s +$0.63),

Hammered in today’s market (10 of 20):

  • Intellia Therapeutics (NTLA -$2.20),
  • BioLife Solutions (BLFS -$1.42),
  • CRISPR Therapeutics (CRSP -$2.38),
  • Beam Therapeutics (BEAM -$0.93 after Tuesday’s +$0.72 and Monday’s -$2.42),
  • Biostage (BSTG -$0.48 after Tuesday’s +$0.48 - promoted),
  • Editas Medicine (EDIT -$0.41),
  • Fate Therapeutics (FATE -$0.17 after Tuesday’s +$1.29 and Monday’s -$2.29),
  • Brainstorm Cell Therapeutics (BCLI -$0.12),
  • uniQure NV (QURE -$0.08),
  • Mesoblast (MESO -$0.05 after Tuesday’s -$0.08),


April, Q2/2022:

  • Wednesday closed negative with 15 incliners and 20 decliners


The BOTTOM LINE:  I always expect the expected as I wrote this a.m., “I am still a believer in the Icarus myth and the danger of dramatic upside flight on low volume wings.”

Also reiterating, “Investors are losing confidence and it shows up in the ever rotating roller-coaster of share pricing.”

State the obvious, investors are NOT in control of daily market driven sector appreciation or downward dives.

The number one takeaway for investors should be to watch how your stock reacts daily more than before and after news and earnings specifically.

Cell and gene therapy equities DON’T always rally on good news but, seriously dive on bad news.

I try to keep it simple …

We are getting close to Q2 reporting season … yet, I can’t write away my misgivings about the daily sessions. 

Earnings’ season is upon us as Alnylam Pharmaceuticals (ALNY) reports Thursday,4/28 followed by Sage Therapeutics (SAGE) on 5/3 and AxoGen (AXGN), Ionis Pharmaceuticals (IONS), MiMedx (MDXG) and Vericel (VCEL) on 5/4 so far.

Volume again was also barely there … as earnings might get even more ugly.

I am still more than worried about investment bank pricey analyst valuations that (in my mind’s eye) have NO basis.

I stick to my view of anticipating the risks – as my careers focus has been “warning analysis”.


Who is defining the metrics for investors and keeping you notified of the sector and market fluctuations?  What I provide is a trusted source of share pricing intelligence – it’s more than opinion, I deal in the facts and numbers that back them up.


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.

Regulation Analyst Certification (Reg AC): The research analyst primarily responsible for the content of this report certifies the following under Reg AC: I hereby certify that all views expressed in this report accurately reflect my personal views about the subject company or companies and its or their securities. I also certify that no part of my compensation was, is or will be, directly or indirectly, related to the specific recommendations or views expressed in this report.