May 17, 2023 6:40pm

However, “What goes up with ceremony, goes down with disdain followed by the opposite effect”

News: Ultragenyx (RARE +$2.46) the FDA has agreed to a protocol amendment to the P1/2 study of GTX-102 in pediatric patients with Angelman syndrome that enables the company to harmonize dose ranges in the U.S. with those being used in ex-U.S. cohorts of the study. Outside of the U.S., the P1/2 study has been actively enrolling and dosing patients in the expansion cohorts to verify the GTX-102 dose and treatment regimen that will be used in the Phase 3 program.

Pre-Open Indications: 2 Hits and 1 Miss

I write this blog/newsletter to inform investors with facts and supporting numbers focused on what they need to hear that others will not say or write about - truth!  

Subscription is coming, it is not conscription but, an offer to join our collective of like-minded investors!  Join me … in the NO spin zone.

If I have learned one thing as a former research analyst in a venture and public fund, operating executive, adjunct (MBA) business school professor now a journalist; it is that your life and your portfolio are largely tied to your knowledge of price movements and questioning pricing targets.

 

The Dow closed UP +408.63 points (+1.24%), the S&P closed UP +48.87 points (+1.19%) while the Nasdaq closed UP +157.51 points (+1.28%)

 

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 gained during Wednesday's session following Tuesday’s selloff as investors remain hopeful that the debt-ceiling talks will produce a breakthrough.

“Equities may trade in a tight range until an outcome is observed with the biggest downside risk coming if we enter Memorial Day weekend without a solution, given the early June X-date.”

A solid quote, Sam Stovall, chief investment strategist at CFRA Research, said he expects that markets are stuck in neutral, even as the news becomes “more and more optimistic” on the debt ceiling. He cited recent trends in the 10-year Treasury yield, as well as strength indicators in the S&P 500.

  • “They’re not really giving any kind of a clue as to which way they’re likely to break, meaning favorably or negatively. We’re still in an up channel for the S&P 500. But I think we are in search of a catalyst to either break out or break down,” Stovall said.

Economic Data Docket: April housing starts came in line with expectations, at 1.4 million. That’s in line with the 1.4 million expected.

  • Building permits issued fell 1.5% to an annualized 1.41 million units in April and registered a 21.1% drop year-over-year as multi-family permits issued tanked 9.7% month-over-month.

 

Wednesday (5/17) … RegMed Investors’ (RMi) pre-open: “When you’re on a roller coaster, the only thing you can be sure of is you’ll end up back where you started. Welcome to my world of defining the “grey’ in our universe!” … https://www.regmedinvestors.com/articles/12963

 

Pre-Open Indications: 2 Hits < Beam Therapeutics (BEAM +$1.10), Sage Therapeutics (SAGE +$0.86)> and 1 Miss < Blueprint Medicine (BPMC +$0.62)>  

 

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

  • Wednesday’s advance/decline line opened negative at 5 up/ 26 down and 4 flats, stayed negative with 15 up/ 18 down and 2 flats at the mid-day, ending with a positive close of 9/24 and 2 flats

 

Key Metrics:

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

  • Wednesday, the IBB was up +0.22% and the XBI was up +0.83%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Wednesday was down -1.12 points or -6.23% at 16.87

 

Closing Down (9 of 9):

  • Chinook Therapeutics (KDNY -$0.84 after Tuesday’s -$1.05),
  • Regenxbio (RGNX -$0.36 after Tuesday’s -$1.99 and Monday’s +$2.38),
  • Generation Bio (GBIO -$0.35),
  • Biostage (OTCQB: BSTG -$0.30 after Tuesday’s $0.00 and Monday’s +$0.32)
  • MiMedx (MDXG -$0.31),
  • Vericel (VCEL -$0.20),
  • Solid Biosciences (SLDB -$0.11 after Tuesday’s +$0.01),
  • Compass Therapeutics (CMPX -$0.03),
  • Adverum Biotechnologies (ADVM -$0.0095)

Flat (2);

  • Mesoblast (MESO)
  • Homology Medicine (FIXX)

Closing Up (10 of 24):

  • CRISPR Therapeutics (CRSP +$2.71 after Tuesday’s -$4.25 and Monday’s +$2.05),
  • Ultragenyx (RARE +$2.46 after Tuesday’s -$2.33 and Monday’s +$1.92),
  • Alnylam Pharmaceuticals (ALNY +$2.26 after Tuesday’s -$15.23 and Monday’s +$0.84),
  • BioLife Solutions (BLFS +$1.68 after Tuesday’s +$0.78),
  • Beam Therapeutics (BEAM +$1.10 after Tuesday’s -$2.13 and Monday’s +$2.66)
  • Sage Therapeutics (SAGE +$0.86 after Tuesday’s -$1.82 and Monday’s +$1.62),
  • Voyager Therapeutics (VYGR +$0.66 after Tuesday’s +$0.97 and Monday’s -$0.04),
  • Blueprint Medicine (BPMC +$0.62 after Tuesday’s -$1.30),
  • uniQure NV (QURE +$0.56 after Tuesday’s -$1.66 and Monday’s -$0.22),
  • Verve Therapeutics (VERV +$0.52),

 

Q2/23 – May

  • Wednesday closed positive with 24 incliners, 9 decliners and 2 flats

 

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

I follow the dictum, quoting Churchill that “short words are best, and the old words when short are best of all.”

“The BIGGEST risk to the economy – the clock is ticking for a divided U.S. Congress to raise the federal government's debt ceiling, which currently stands at over $31 trillion but risks running out of cash as soon as June 1.

From the title, hope and optimism are NOT technical indicators.

The FTC's lawsuit, filed yesterday, to block Amgen's (AMGN) $28 billion acquisition of Horizon Therapeutics continued to weigh on healthcare – biotech, cell and gene therapy equities, which was one of the day's worst-performing sectors. Amgen was down by -0.32%, while Merck & Co. (MRK) lost -1.14%. Johnson & Johnson (JNJ) was also down 0.22%, its 11th drop in 12 days.

As I have stated, “The cell and gene therapy sector could experience yet another the “flight of Icarus” …  since “our” universe have oscillated between gains and losses since the first of 2023!”

There is VALUE out there however, it’s about playing or betting the timing of the sector. Even as I had stated, “I wouldn’t be buying into this rally … I would be selling into this upside!”

A very appropriate quote, “You don't do well by trying to be right; it is impossible for humans. You do well by figuring out when you're wrong faster than others do.” <Nassim Nicholas Taleb>

 

There are clear winners — and losers — at the start of May.

The top three (3) performing in the session:

·         Wednesday: CRISPR Therapeutics (CRSP), Ultragenyx (RARE) and Alnylam Pharmaceuticals (ALNY)

·         Tuesday: Voyager Therapeutics (VYGR), BioLife Solutions (BLFS) and Mesoblast (MESO)

·         Monday: Beam Therapeutics (BEAM), Intellia Therapeutics (NTLA) and Regenxbio (RGNX)

While The worst three (3) in the session:

·         Wednesday: Chinook Therapeutics (KDNY), Regenxbio (RGNX) and Generation Bio (GBIO)

·         Tuesday: Alnylam Pharmaceuticals (ALNY), CRISPR Therapeutics (CRSP) and Ultragenyx (RARE)

·         Monday: bluebird bio (BLUE), uniQure NV (QURE) and MiMedx (MDXG)

 

Earnings:

RegMed Investors (RMi) Research Note: Q1/23 earnings reporting dates, net losses, cash positions and runway outcomes … https://www.regmedinvestors.com/articles/12923  -

Sector earnings season has drawn to a close …

·         One (1) net income from 34 reported to date:

 

WHY (?) do I keep posting, so investors can get a daily update of the sector's share pricing and some individual companies TRUTHS while electronic trading chews on our portfolio edges.

I am definitely considered a contrarian depending on the session – one has to be a watcher of the ups and downs as well as measurer of sentiment. I am also a NO trust investor but, a validate and verify journalist/analyst.

 

The NO spin zone! U.S. public shareholders deserve accountability ...

My question that resounds, that should be asked is how many companies are at the end of sentiments …  leash?

 

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.