August 18, 2022 4:52pm

The "Slipping and Sliding" song seems to be referring to messing up (slipping) and messing up' on purpose (sliding) – a response to inflation and possibly the costs of developing therapies and their pricing after an approval

News: bluebird bio (BLUE -$0.97) dives after its first approval from the U.S. FDA following its business split late last year. BLUE’s therapy Zynteglo, or the treatment of beta-thalassemia, which causes an oxygen shortage in the body and often leads to liver and heart issues is expected to face some resistance from insurers due to its steep price ($2.8 M).

Pre-open indications: 7 HIT and 2 MISS

WHY and HOW is this market affecting the cell and gene therapy sector … “The Bottom Line” underlines the framework of share pricing actions.  Who else is tracking a broad grouping of cell and gene therapy equities – can you afford to not read facts supported by real numbers?

No tunnel vision here, I get into the weeds of daily share pricing and causes 

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 +18.72 points (+0.06%); the S&P closed UP +9.70 points (+0.23%) while the Nasdaq closed UP +27.22 points (+0.21%)



The Dow, S&P and Nasdaq was up slightly …

Traders and strategists saw Wednesday's minutes from the Fed's July meeting as suggesting policymakers may be less aggressive than previously thought when they raise interest rates in September.

Economic Data Docket: Initial jobless data fell to 250,000 for the week ended Aug. 13, while existing home sales dropped nearly 6% in July.


RegMed Investor’s (RMi) Pre-Open: “a mixed trend with a bit of a bounce-back. Adding to some diminished positions could expose limited uptrends while cutting losses of others or moving to cash” …


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

  • Thursday’s advance/decline line opened negative at 6 up/ 28 down and 1 flat, stayed negative with 5 up/28 and 2 flats at the mid-day, ending with a negative close of 14/20 and 1 flat.


Pre-open Indications: 0 HITs, 7 MISS < Biostage (BSTG +$0.15 with 790 shares traded), Chinook Therapeutics (KDNY +$0.14), Ionis Pharmaceuticals (IONS +$0.16), Ultragenyx (RARE +$1.42), uniQure NV (QURE +$0.53), Homology Medicine (FIXX -$0.10), Regenxbio (RGNX +$0.43)> 2 MISS < Beam Therapeutics (BEAM -$1.98), bluebird bio (BLUE-$0.97),


Key Metrics: the why's ...

  • Thursday - Sector volume was LOW with 2 of the 14-upside having higher than the 3-month average volume with LOW volume of 5 of 20-downside having higher than the 3-month average volume;

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

  • Thursday, the IBB was down -1.13% and the XBI was down -0.78%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Thursday was down -0.38 points or -1.91% at 19.52


Jumping with share pricing momentum (10 of 14):

  • Ultragenyx (RARE +$1.42 after Wednesday’s -$2.06, Tuesday’s -$1.89 and Monday’s -$0.37),
  • Vericel (VCEL +$1.08),
  • BioLife Solutions (BLFS +$1.02 after Wednesday’s -$2.62, Tuesday’s -$0.66 and Monday’s +$1.42),
  • uniQure NV (QURE +$0.53),
  • Regenxbio (RGNX +$0.43),
  • CRISPR Therapeutics (CRSP +$0.41 after Wednesday’s -$4.11, Tuesday’s -$4.29 and Monday’s +$1.48),
  • Alnylam Pharmaceuticals (ALNY +$0.32 after Wednesday’s -$5.02, Tuesday’s -$2.86 and Monday’s -$2.84),
  • Voyager Therapeutics (VYGR +$0.28),
  • Ionis Pharmaceuticals (IONS +$0.16),
  • Chinook Therapeutics (KDNY +$0.14 after Wednesday’s -$0.83 and Tuesday’s +$1.49),


  • Adverum Biotechnologies (ADVM)

Closing down (10 of 20):

  • Beam Therapeutics (BEAM -$1.98 after Wednesday’s -$4.00),
  • bluebird bio (BLUE -$0.97 after Wednesday’s +$0.20),
  • Verve Therapeutics (VERV -$0.57 after Wednesday’s -$1.05, Tuesday’s -$0.69 and Monday’s +$2.27),
  • Editas Medicine (EDIT -$0.38 after Wednesday’s -$1.02, Tuesday’s -$1.12 and Monday’s -$0.31),
  • Intellia Therapeutics (NTLA -$ after Wednesday’s -$4.61, Tuesday’s -$4.25 and Monday’s +$0.68),
  • AxoGen (AXGN -$0.28),
  • Biostage (BSTG -$0.15 after Wednesday’s +$0.15, Tuesday and Monday’s $0.00),
  • Sage Therapeutics (SAGE -$0.15 after Wednesday’s +$0.25),
  • MiMedx (MDXG -$0.12),
  • Cellectis SA (CLLS -$0.11),


August, Q3/2022:

  • Thursday closed negative with 14 incliner, 20 decliners and 1 flat
  • Wednesday closed negative with 6 incliner, 28 decliners and 1 flat
  • Tuesday closed negative with 7 incliner, 25 decliners and 3 flats
  • Monday closed positive with 26 incliner, 8 decliners and 1 flat


The BOTTOM LINE: I try to keep it simple … and short!

bluebird bio’s (BLUE) approval of Zynteglo is reaping negative results re its pricing which COULD be an ISSUE for other and coming gene therapy therapies.

Re BLUE’s therapy Zynteglo and questions of costs. Some history re pricing: Novartis in 2019 was forced to offer discounts and work out "outcome-based" installments payments for its $2.1 million therapy Zolgensma after insurers balked at the drug's price. BLUE expects to start the treatment process for patients in the fourth quarter. No revenue is, however, expected from the therapy in 2022 as the treatment cycle would take an average of 70 to 90 days from initial cell collection to final transfusion. <Reuters>

There are really three camps, electronic algorithms, traders versus investors who STILL don’t comprehend if the worst is behind us and those “entities” that continue to buy into selloffs.

It was expected to see the magnitude of the lack of EPS strength in Q2 and after the prior quarter (Q1) in the face of the difficulty that investors are facing in the inflation continuum of soon to be experiencing rising rate environment which is about to get worse!

I am still hobbling after a back injury (never have I had an issue) with walking stick for use and have trouble sitting on a chair for a period of time (with a pillow on hard chair versus my usual business chair).

The cell and gene therapy sector is STILL experiencing the “flight of Icarus” …  since “our” universe have oscillated between gains and losses since the first of the year!

There are ONLY net and per share losses in Q2. SG&A’s are, I believe “OUT-OF-CONTROL”, CEOs are skimming shareholders with HIGH salaries and perks as share pricing gets hammered on alternate sessions.

The real question is how recently have CEOs bought-into their depreciated share pricing such as two: AVRO and AGTC.

As I have “noticed” yet again from to date reporting, “Most reporting sector companies … to date have reported net losses, lagging revenue estimates … although AXGN, FATE, RGNX, FIXX and NTLA beat on revenues generating forward motion while ATM use is up.”

 I am also NOT convinced that the lows are in the cell and gene therapy sector are behind us.”

The coming weeks of August will reveal more about the cell and gene therapy sector's state of health.

I find it very interesting to evaluate share price over earnings and a shortage of revenue reporting as a proxy for share pricing performance.

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


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.