August 4, 2022 7:31pm

It’s coming

Pre-open indications: 7 HITs and 6 MISS

News: Global Blood Therapeutics Inc. (GBT) were up +$14.06 or +41.44% in premarket trading after Bloomberg reported that the company is "attracting takeover interest," citing people familiar with the matter.

Earnings’ Update: below

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 DOWN -85.68 points (-0.26%); the S&P closed DOWN -3.23 points (-0.08%) while the Nasdaq closed UP +52.42 points (+0.41%)



Stock market trades mixed as investors are torn by rising jobless claims and falling oil prices.

Indexes wavered Thursday as traders awaited Friday’s July jobs report, which will give the latest snapshot on the labor market and the health of the economy.

The Biostage (BSTG) Chronicles: How will stock reverse affect earnings?  BOD approved on 7/28 amending and restating their Certificate of Incorporation to affect a reverse split of the issued and outstanding common stock at a ratio of not less than 1-for-1.25 and not greater than 1-for-5. What about all those PPM (private placements) as an offering follows?

Barrons’ questions re gene editing safety: A gene-editing treatment from Intellia Therapeutics (NTLA) led to elevated liver enzymes in one patient with polyneuropathy, prodding NTLA stock to topple Thursday.

M&A is it a trend to begin? Amgen scooped-up commercial biotech ChemoCentryx $CCXI and its recently approved rare disease drug for $3.7 billion out of the cash stockpile. The deal comes in at $52 a share — a hefty increase over the $24.11 close. The drug is called Tavneos (avacopan), a complement factor C5a inhibitor OK’d to treat anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis, an autoimmune disease which can be lethal.

·         For Amgen, it’s a chance to add a drug aimed at small vessel inflammation to a portfolio of drugs that includes a knockoff of Rituxan, which had been the only drug approved for ANCA-associated vasculitis before Tavneos arrived. And there are three early clinical stage drugs — chemoattractant receptors for other inflammatory conditions — in the ChemoCentryx pipeline as well as an oral checkpoint inhibitor. <EndPoints>

Economic Data Docket: Initial jobless claims rose to 260,000, up 6,000 from last week. The increase was in line with analysts' expectations.

·         A slight uptick in weekly jobless claims, was reported weighing on investors watching for signs that labor market strength is dwindling. The July jobs report, scheduled to be released Friday, will show how employers hired last month. In addition, investors will get an updated look at inflation data from the July consumer price index report next week.


Earnings’ Update:

Intellia Therapeutics (NTLA) reported a net loss of -$100.7 M or -$1.33 per share and a cash position of $906.9 M (an ATM of -$38.9 M and a draw of -$14.3 M from employee stock plans) with a runway until 2025.

Sangamo Therapeutics (SGMO) reported a net loss of -$43.2 m or -$0.29 and a cash position of $363.7 M with a runway until 2025.

Voyager Therapeutics (VYGR) reported a new loss of -$19.1 M or -$0.50 per share and a cash position of 148.1 M with a runway until 2024.

Biostage (BSTG) reported a net loss of -$1.3 M or -0.12 per share and a cash position of $4.6M with a runway until Q2/2023 and preparing a stock reverse to execute a financing by non-U.S. Chinese investors.

bluebird bio (BLUE) reported a net loss of -$100.1 M or -$1.36 per share and a cash position of $218 M (used $8 M from a $24.7M ATM) with a runway until 2024 but, exploring alternative financing


RegMed Investor’s (RMi) Pre-Open: “post earnings, remember Isaac Newton’s apple, gravity caused a bonk on the head. Substitute algorithms for the apple, another warning” …


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

  • Thursday’s advance/decline line opened positive at 19 up/ 14 down and 2 flats, stayed positive with 23 up/11 down and 1 flat at the mid-day, ending with a positive close of 23/9 and 2 flats.


Pre-open Indications: 7 HITs <Biostage (OTCQB: BSTG -$0.40 with 208 shares traded); CRISPR Therapeutics (CRSP +$0.33), Agenus (AGEN -$0.05), Intellia Therapeutics (NTLA -$6.27), Sangamo Therapeutics (SGMO-$0.03); Sell into Strength:  Verve Therapeutics (VERV -$0.37); Global Blood Therapeutics (GBT +$14.06), > 6 MISS < Editas Medicine (EDIT +$0.46), Caribou Biosciences (CRBU +$0.00),  Alnylam Pharmaceuticals (ALNY +$12.04), Ions Pharmaceuticals (IONS +$2.27),  Ultragenyx (RARE +$2.17), AxoGen AXGN +$0.03)>


Key Metrics:

  • Thursday - Sector volume was LOW with 8 of the 23-upside having higher than the 3-month average volume with LOW volume of 5 of 9-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 +2.07% and the XBI was up +5.11%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Thursday was down -0.38 points or -1.73% at 21.57


Jumping with share pricing momentum (10 of 23):

  • Global Blood Therapeutics (GBT +$14.06),
  • Alnylam Pharmaceuticals (ALNY +$12.04 after Wednesday’s +$70.04, Tuesday’s +$2.02 and Monday’s -$2.09),
  • Ionis Pharmaceuticals (IONS +$2.45 after Wednesday’s +$3.84 and Tuesday’s -$0.31),
  • Sage Therapeutics (SAGE +$2.32),
  • Ultragenyx (RARE +$2.17 after Wednesday’s +$2.32, Tuesday’s +$0.65 and Monday’s -$2.65),
  • Chinook Therapeutics (KDNY +$1.11),
  • BioLife Solutions (BLFS +$0.90),
  • Regenxbio (RGNX +$0.75 after Wednesday’s +$2.18, Tuesday’s +$0.77 and Monday’s -$0.33),
  • Editas Medicine (EDIT +$0.46 after Wednesday’s +$2.21 after Tuesday’s +$0.64),
  • uniQure NV (QURE +$0.41),


  • Mesoblast (MESO) and Caribou Biosciences (CRBU $0.00)

Closing down (9 of 9):

  • Intellia Therapeutics (NTLA -$6.27 after Wednesday’s +$7.64, Tuesday’s +$1.32 and Monday’s -$2.02),
  • Vericel (VCEL -$1.30 after Wednesday’s -$0.08),
  • Biostage (BSTG -$0.40 after Wednesday’s $0.00, Tuesday’s $0.00 and Monday’s $0.00)
  • Verve Therapeutics (VERV -$0.37 after Wednesday’s +$2.83, Tuesday’s +$1.04 and Monday’s -$2.02),
  • Fate Therapeutics (FATE -$0.26 after Wednesday’s +$2.18, Tuesday’s +$0.59 and Monday’s -$0.49),
  • Cellectis SA (CLLS -$0.20),
  • Beam therapeutics (BEAM -$0.13 after Wednesday’s +$3.87),
  • Agenus (AGEN -$0.05),
  • Sangamo Therapeutics (SGMO -$0.03),


August, Q3/2022:

  • Thursday closed positive with 23 incliner, 9 decliners and 2 flats


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

As I have been writing … “I am STILL … NOT convinced the lows are in the cell and gene therapy sector.”

Volume is still low …

Although, I’m STILL a firm believer in the flight of Icarus …  since the cell and gene therapy equities have oscillated between gains and losses since the first of the year... a bigger “slip” is on the way – maybe NOT today but, it’s coming!

Hold-on (to the railing) as our portfolios weather these volatile sessions as an ordinary practice.

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

This week, the cell and gene therapy sector Q2 earnings continue full-force: Precigen (PGEN) and Global Blood Therapeutics (GBT) on 8/8; Agenus (AGEN), BioLife Solutions (BLFS) and Ionis Pharmaceuticals (IONS) on 8/9 and Brainstorm cell Therapeutics (BCLI) on 8/15.

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.