November 29, 2023 5:33pm

Some of yesterday’s ups were stripped, and yet the cell and gene therapy sector closed up

News: uniQure N.V. (QURE -$0.18) the FDA has cleared the Investigational New Drug (IND) application for AMT-191, a gene therapy candidate for Fabry disease. REGENXBIO (RGNX -$0.61) the first patient received RGX-202 at dose level 2 in the P1/2 AFFINITY DUCHENNE® trial.

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After careful examination of the sector and breathe trends, I STILL come away more cautious. Having lived through the rise and multiple declines of “our” universe’s stock price.

We're in a period of sector rotation, and people are trying to figure out what cell and gene therapy sector equities at are going to continue to do well and what are the stocks that are going to suffer.

Investors must be better informed on what fluctuating economics and markets can do to an equity, or what impact indications or expectations can have.

 

The Dow closed UP +13.57 points or +0.04%, the S&P closed DOWN -4.31 points or -0.09% while the Nasdaq closed DOWN -23.27 points or -0.16%

 

Henry’omics:

We need to more than consider the economic environment - rising rates and inflation to comprehend the micro re “our” universe of cell and gene therapy companies …

Indexes were mixed on Wednesday … as a robust upward GDP revision eased recession fears, while Federal Reserve officials' remarks raised questions about the duration of the central bank's restrictive policy ahead of inflation data due early Thursday.

Economic Data Docket: gross domestic product in the third quarter grew at a stronger-than-forecast annual rate of 5.2%. The boost came primarily from revisions to government spending and investments in nonresidential structures.

 

News (continued): REGENXBIO (RGNX) RGX-202 is an investigational one-time AAV Therapeutic for Duchenne muscular dystrophy (Duchenne), using the NAV® AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain as well as a muscle-specific promoter to support a targeted therapy for improved resistance to muscle damage associated with Duchenne. "Progressing to dose level 2 is an important milestone in our updated strategic plans and for accelerating the development of RGX-202," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "There is a large unmet need for new therapies for boys with Duchenne, and the market is capable of supporting multiple gene therapies. We believe RGX-202 has unique, differentiating features that support its potential to be a best-in-class product."

 

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

  • Wednesday’s advance/decline line was positive with 28 incliners, 6 decliners and 1 flat at the open, the mid-day continued positive with 22 inclined, 11 decliners and 2 flats, ending with a positive close of 19 incliners, 13 decliners and 3 flats

 

Ebb and flow of MY covered sector cell and gene therapy session daily “endings”:

Q4:  November – 11 positive, 1 neutral and 9 negative closes

·         October – 12 negative and 10 positive closes

 

Key Metrics:

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

  • Wednesday, the IBB was up +0.96% and the XBI was up +1.19%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Wednesday was up +0.28 point or +2.21% at 12.97

 

Wednesday’s Closing Down (10 of 13):

  • CRISPR Therapeutics (CRSP -$1.57 after Tuesday’s -$0.95 and Monday’s +$1.64),
  • Beam Therapeutics (BEAM -$1.18 after Tuesday’s +$0.61 and Monday’s -$0.51),
  • Verve therapeutics (VERV -$0.73 after Tuesday’s +$0.37 and Monday’s -$0.56),
  • Regenxbio (RGNX -$0.61),
  • BioLife Solutions (BLFS -$0.24 after Tuesday’s -$0.69 and Monday’s -$0.64),
  • uniQure NV (QURE -$0.18 after Tuesday’s -$0.04 and Monday’s +$0.14),
  • Cellectis SA (CLLS -$0.14 after Tuesday’s +$0.33),
  • Edita Medicine (EDIT -$0.13)
  • Solid Biosciences (SLDB -$0.11 after Tuesday’s -$0.26 and Monday’s +$0.30),
  • MiMedx (MDXG -$0.11),

Flat (3):

  • Harvard Apparatus GN (HRGN)
  • Graphite Bio (GRPH)
  • Brainstorm Cell Therapeutics (BCLI)

Wednesday’s Closing Up (10 of 19):

  • Alnylam Pharmaceuticals (ALNY +$2.65 after Tuesday’s +$2.17 and Monday’s -$3.72),
  • Ionis Pharmaceuticals (IONS +$1.07 after Tuesday’s -$1.36),
  • Ultragenyx Pharmaceuticals (RARE +$0.65 after Tuesday’s -$0.15 and Monday’s -$0.53),
  • Vericel (VCEL +$0.31 after Tuesday’s -$1.04 and Monday’s +$0.34),
  • AxoGen (AXGN +$0.26 after Tuesday’s +$0.24 and Monday’s +$0.03),
  • Sage Therapeutics (SAG E+$0.21 after Tuesday’s +$0.16 and Monday’s -$0.39),
  • Intellia Therapeutics (NTLA +$0.18 after Tuesday’s +$0.10 and Monday’s -$0.38),
  • Voyager Therapeutics (VYGR +$0.13),
  • Caribou BioSciences (CRBU +0.13 after Tuesday’s +$0.08 and Monday’s +$0.09)
  • Blueprint Medicine (BPMC +$0.11 after Tuesday’s -$0.75 and Monday’s +$0.67),

 

Q4/23 – November

  • Wednesday closed positive with 19 incliners, 13 decliners and 3 flats

 

The BOTTOM LINE: Everybody needs a day off for doctor’s (yearly physical) appointments followed by tests in a.m. Thursday to validate good health’s continuation.

  • With the end of November in sight, investors are weighing whether to punch out some sector equities or buy up some bottom liners.
  • Reiterating, “Investors NEED to start formulating their portfolio outlook into next year.”

 

The session started off with solid gains from the big three indexes as bond yields retreated again, though stocks retreated a bit after Richmond Federal Reserve President Thomas Barkin sounded more hawkish tones than Fed Gov. Christopher Waller did the day prior.

  • Federal Open Market Committee members will pay close attention to Thursday’s core personal-consumption expenditures price index.

 

Markets are looking for further signs that inflation is cooling. Prior to the market’s open, the Bureau of Economic Analysis said the U.S. economy grew at a 5.2% annual rate in the third quarter, up from a previous figure of 4.9%.

  • Chris Larkin, managing director, trading and investing at E*TRADE from Morgan Stanley told Barron’s that although the figure was revised up, consumer spending was revised down from the previous numbers. Such "bad news" is good news for those betting the Federal Reserve may turn to rate cuts next year. <Barron’s>

 

On the mark quote: The market has had huge returns, so there's certainly profit taking and repositioning; there's some consolidation going on here." <Tim Ghriskey, senior portfolio strategist Ingalls & Snyder in New York>

 

What’s behind the numbers:

  • Benchmark 10-year notes were down 5.8 basis points to 4.278%, from 4.336% late on Tuesday. The 30-year bond was last down 6.2 basis points to yield 4.4619%, from 4.524%. The 2-year note was last was down 8.9 basis points to yield 4.6475%, from 4.736%.

 

It's time to think ahead to December. It's usually an important month for the Nasdaq.

  • Don’t FORGET portfolio tax selling and a bit of rebalancing
  • And then comes January and with worries that the U.S. economy will be slowing down, the potential for continued Middle East violence and, of course, the real prospect of bitterly contested elections across all of the United States. <The Street>

 

Sign of the times, some bad news, or is it?

Generation Bio Co. (GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, announced a strategic reorganization to prioritize investment in the development of its cell-targeted lipid nanoparticle (ctLNP) delivery system for wholly-owned programs in extrahepatic cell types.

Generation Bio intends to continue to develop ctLNP for immune cells as part of its collaboration with Moderna and to develop its immune-quiet DNA (iqDNA) for its lead hemophilia A and other programs.

The reorganization actions and financial impacts are as follows:

  • Streamlining R&D to develop wholly-owned ctLNP programs for extrahepatic cell types
  • Continuing to develop immune cell programs with Moderna
  • Continuing to develop the iqDNA platform for its lead hemophilia A and other programs
  • Extending cash runway into the second half of 2027
  • The company will reduce its total workforce by 40%, while preserving core R&D capacity. Several of Generation Bio’s leadership team members will also depart as part of the reorganization.

 

The top three (3) performing in the session:

  • Wednesday: Alnylam Pharmaceuticals (ALNY) – 2x, Ionis Pharmaceuticals (IONS) and Ultragenyx Pharmaceuticals (RARE)
  • Tuesday: Alnylam Pharmaceuticals (ALNY), Beam Therapeutics (BEAM) and Verve Therapeutics (VERV)
  • Monday: CRISPR Therapeutics (CRSP), Blueprint Medicine (BPMC) and Vericel (VCEL)

The worst three (3) in the session:

  • Wednesday: CRISPR Therapeutics (CRSP, Beam Therapeutics (BEAM) and Verve Therapeutics (VERV)
  • Tuesday: Ionis Pharmaceuticals (IONS), Vericel (VCEL) and CRISPR Therapeutics (CRSP),
  • Monday: Alnylam Pharmaceuticals (ALNY), BioLife Solutions (BLFS) and Verve Therapeutics (VERV) 

 

Welcome to my world of defining the “grey’ in our universe!

  • Right up front, “I also hate to be so negative or contrarian but, this is a NO spin zone and facts are its product; I can always be WRONG but … I am mostly EARLY!”

 

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.