July 28, 2022 4:42pm

How long to suffer as some gene and cell therapy discovery “engines” aren’t returning quarterly or even yearly ROI while GDP data report confirms “Technical Recession”

Pre-open indications: 3 HITs, 2 MISS and 1 Pump/Promote

News: Ionis Pharmaceuticals (IONS +$0.35) announced positive topline results of the P2b RE-THINC ESRD study of fesomersen, formerly IONIS-FXI-LRx, in patients with end-stage renal disease (ESRD) on hemodialysis.

Earnings: ALNY (+$1.55) review; <see Henry’omics>

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 +332.04 points (+1.03%); the S&P closed UP +48.82 points (+1.21%) while the Nasdaq closed UP +130.17 points (+1.08%)



Indexes rose on Thursday after initially dipping after the GDP report but rebounded as investors still questioned for the likelihood of a recession – which we are already in waiting for further rate hikes.

Ionis Pharmaceuticals (IONS) News (continued from title):  In the study, fesomersen achieved its primary outcome measure of no increase in incidence of major bleeding and clinically relevant non-major bleeding as compared to placebo. Data from the study show that fesomersen, administered monthly at 40 mg, 80 mg and 120 mg for up to 48 weeks, was safe and well-tolerated. Fesomersen also demonstrated substantial and statistically significant reductions in Factor XI activity levels.

Economic Data Docket: Q2 GDP slipped 0.9%. This marked the 2nd straight negative Q for GDP, a metric which has historically often coincided with economic downturns. Also, initial jobless claims showed a slight moderation in first-time filings for unemployment insurance, totaling 256,000 last week after 261,000 filings the prior week.

Sector Earnings: Alnylam Pharmaceuticals (ALNY) reported a Q2 Loss, missing revenue estimates. a Q2 loss of $2.29 per share versus the Zacks Consensus Estimate of a loss of $1.62. This compares to loss of $1.61 per share a year ago. These figures are adjusted for non-recurring items.

·         Q2 report represents an earnings surprise of -41.36%. A quarter ago, it was expected that this RNA interference drug developer would post a loss of $1.93 per share when it actually produced a loss of $2, delivering a surprise of -3.63%.

·         ALNY posted revenues of $224.82 million for Q2, missing the Zacks Consensus Estimate by 13.10%. This compares to year-ago revenues of $220.55 million.

·         ALNY shares have lost about 16.3% since the beginning of the year versus the S&P 500's decline of -15.6%.


RegMed Investor’s (RMi) Pre-Open: “Q2 GDP and sector earnings initiation. Determine the session.” …  https://www.regmedinvestors.com/articles/12538


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 3 up/ 31 down and 1 flat, stayed negative with 12 up/ 22 and 1 flat at the mid-day, ending with a negative close of 13/22 and 0 flat.


Pre-open Indications: 3 HITs < CRISPR Therapeutics (CRSP -$3.03), Verve Therapeutics (VERV -$2.00), Editas Medicine (EDIT -$0.44), 3 MISS < Sage Therapeutics (SAGE +$0.14). Alnylam Pharmaceuticals (ALNY +$1.55) following earnings, Biostage (BSTG +$0.50 with 600 shares traded).


Key Metrics:

  • Thursday - Sector volume was LOW with 1 of the 13-upside having higher than the 3-month average volume with LOW volume of 0 of 22-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 up +0.10% and the XBI was down -1.12%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Thursday was down -0.87 points or -3.74% at 22.37


Jumping with share pricing momentum (13 of 13):

  • Alnylam Pharmaceuticals (ALNY +$1.15 after Wednesday’s +$1.97, Tuesday’s -$1.20 and Monday’s -$2.01),
  • Vericel (VCEL +$1.24 after Wednesday’s +$1.81, Tuesday’s +$0.15 and Monday’s +$0.18),
  • BioLife Solutions (BLFS +$1.10 after Wednesday’s +$0.89, Tuesday’s -$0.12 and Monday’s -$0.94),
  • Ultragenyx (RGNX +$1.07),
  • Biostage (BSTG +$0.50 after Wednesday’s -$0.05 after Tuesday’s $0.00 and Monday’s -$0.45),
  • Ionis Pharmaceuticals (IONS +$0.35),
  • Mesoblast (MESO +$0.26),
  • uniQure NV (QURE +$0.22 after Wednesday’s +$0.98),
  • Sage Therapeutics (SAGE +$0.14),
  • Caribou Therapeutics (CRBU +$0.11),
  • Voyager Therapeutics (VYGR +$0.10),
  • Solid Biosciences (SLDB +$0.045),
  • Bellicum Pharmaceuticals (BLCM +$0.01),

Closing down (10 of 22):

  • CRISPR Therapeutics (CRSP -$3.03 after Wednesday’s +$1.81, Tuesday’s +$0.62 and Monday’s +$0.68),
  • Beam Therapeutics (BEAM -$2.13 after Wednesday’s +$3.67, Tuesday’s +$0.17 and Monday’s +$1.09),
  • Verve Therapeutics (VERV -$2.00 after Wednesday’s +$1.04, Tuesday’s -$1.27 and Monday’s -$0.83),
  • Intellia Therapeutics (NTLA -$1.87 after Wednesday’s +$2.18, Tuesday’s +$0.12 and Monday’s +$0.32),
  • Fate Therapeutics (FATE -$1.02 after Wednesday’s +$1.79, Tuesday’s +$0.64 and Monday’s -$0.30),
  • Chinook Therapeutics (KDNY -$0.60),
  • Regenxbio (RGNX -$0.49 after Wednesday’s +$1.05, Tuesday’s +$0.57 and Monday’s +$0.03),
  • Editas Medicine (EDIT -$0.44),
  • bluebird bio (BLUE -$0.33),
  • Sangamo Therapeutics (SGMO -$0.12),


July, Q3/2022:

  • Thursday closed negative with 13 incliner and 22 decliners
  • Wednesday closed positive with 31 incliner and 4 decliners
  • Tuesday closed negative with 13 incliner, 19 decliners and 3 flats
  • Monday closed negative with 20 incliner, 14 decliners and 1 flat


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

I am NOT convinced the lows are in the cell and gene therapy sector.

Sector expectations are really low as earnings season begins next week; remember, a little bit of good news can go a long way when you have low expectations.

Volume is still low …

The coming next two (2) weeks will reveal more about the cell and gene therapy sector's state of health.

Next week … the cell and gene therapy sector earnings continue full-force – Q2 earnings: Sage Therapeutics (SAGE) on 8/2; and AxoGen (AXGN), Editas Medicine (EDIT), Fate Therapeutics (FATE), MiMedx (MDXG) and Vericel (VCEL) on Wednesday, 8/3, Agenus (AGEN) and Intellia therapeutics (NTLA) and Sangamo Therapeutics (SGMO) on 8/4 followed by Ionis Pharmaceuticals (IONS) on 8/9.

investors should be bracing for a milder-than-usual earnings season and downwardly LPS (loss-per-share) predictions,

I find it very interesting to evaluate share price over earnings as a proxy for performance.

We always need a villain and they are multiplying – “Earnings’ season is coming, investors NEED to focus on LPS (loss-per-share), collaboration revenues and cash positions i.e., runways”.

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.