February 15, 2024 4:45pm

News: Intellia Therapeutics (NTLA +$0.19) and ReCode Therapeutics (private) a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF). CF is a genetic disease caused by mutations in the CFTR gene, leading to the accumulation of thick mucus in the lungs, digestive systems and other organs. CF can result in life-threatening infections, respiratory failure and other serious complications.

Pre-open Indications: 4 Hits and 2 Miss

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A 360-degree review of the who, why, when and what happened in the sector as the market and economics surround your portfolio defense. The Bottom Line includes earnings reviews and coming “whispers”

Never leave an investor uninformed! A trusted source of factual reporting!

Today is the perfect example of the sector being what it is, until it isn’t and even then, it doesn’t seem to be … that is after taking a deep breath and holding it

Earnings, net losses, and per-share losses, runways and guidance have begun Q4 and FY23 which WILL be a crucial  force in the sector as history has taught us i.e., investors.

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! 

 

Thursday: The Dow closed UP +349.12 points or +0.91%, the S&P closed UP +29.13 points +0.58% while the Nasdaq closed UP +47.03 points or +0.30%

 

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 climbed for a second day Thursday, helped by lower Treasury yields, as Wall Street clawed back the steep losses suffered earlier in the week.

Treasury yields fell, with the yield on the benchmark 10-year Treasury slipping 3 basis points to 4.24%. The 2-year Treasury yield fell 2 basis points to 4.56%.

Economic Data Docket: retail sales dropped 0.8% in January. That’s much more than the 0.3% decline expected by economists, according to Dow Jones. This raised some concern about the strength of the U.S. consumer under the weight of sticky inflation and high interest rates, and sent Treasury yields down.

 

Thursday’s RegMed Investors’ (RMi) pre-open: “got a broom? A sweep of the portfolio downside risks pre-earnings usually needed; with another round of economic data.” …  https://www.regmedinvestors.com/articles/13332

 

Pre-open Indications: 4 Hits: < Ultragenyx Pharmaceuticals (RARE +$0.60), Sage Therapeutics (SAGE -$0.34), Mesoblast (MESO +$0.115), Agenus (AGEN -$0.275)> 2 Miss < Prime Medicine (PRME +$0.94), Vericel (VCEL +$1.81)>

 

Advance/Decline (A/D) Line:

  • Thursday’s advance/decline line at the open was positive with 27 incliner, 6 decliners and 2 flats; ending with a positive close of 28 incliners, 7 decliners and 0 flat

 

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

Q1/24: February, 4 negative and 7 positive closes

  • January: 2 holiday, 12 negative and 8 positive closes

 

Key Metrics:

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

  • Thursday, the IBB was up +1.28% and the XBI was up +2.30%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Thursday was down -0.20 point or -1.39% at 14.18

 

Thursday’s Closing Down (7 of 7):

  • Alnylam Pharmaceuticals (ALNY -$16.72 after Wednesday’s +$2.15 after Tuesday’s -$7.34 and Monday’s -$0.69),
  • Ionis Pharmaceuticals (IONS -$3.79),
  • Sage Therapeutics (SAGE -$0.34 after Wednesday’s +$2.73),
  • Agenus (AGEN -$0.27),
  • Adverum Biotechnologies (ADVM -$0.10 after Wednesday’s -$0.19 after Tuesday’s +$0.30),
  • Harvard Apparatus GN (HRGN -$0.10 after Wednesday’s $0.00),
  • Bellicum Pharmaceuticals (BLCM -$0.017),

Thursday’s Closing Up (10 of 28):

  • Blueprint Medicine (BPMC +$10.77 after Wednesday’s +$3.63 after Tuesday’s -$4.37 and Monday’s +$1.00
  • CRISPR Therapeutics (CRSP +$5.44 after Wednesday’s +$6.26 after Tuesday’s -$3.66 and Monday’s +$6.43),
  • Vericel (VCEL +$1.81 after Wednesday’s +$1.61 after Tuesday’s -$1.47 and Monday’s +$0.64),
  • Regenxbio (RGNX +$1.66 after Wednesday’s +$0.75 after Tuesday’s -$1.32 and Monday’s +$1.15
  • Prime Medicine (PRME +$0.94),
  • Solid Biosciences (SLDB +$0.68 after Wednesday’s +$1.30),
  • Ultragenyx Pharmaceuticals (RARE +$0.60 after Wednesday’s +$1.68 and Tuesday’s -$2.76),
  • Fate therapeutics (FATE +$0.59),
  • AxoGen (AXGN +$0.59),
  • Verve Therapeutics (VERV +$0.53),

 

Q1/24 – February, this week

  • Thursday closed positive with 28 incliners, 7 decliners and 0 flat
  • Wednesday closed positive with 31 incliners, 3 decliners and 1 flat
  • Tuesday closed negative with 2 incliners, 33 decliners and 0 flat
  • (2/12) Monday closed positive with 28 incliners, 7 decliners and 0 flats

 

The BOTTOM LINE: The cell and gene therapy sector continued to maintain its elevated position higher in semi-volatile trading on Thursday after Wednesday's pop following Tuesday’s selloff session and a “hot” inflation report.

Earnings:

Ultragenyx Pharmaceuticals (RARE +$0.60)

Q4 net loss of -$123.19 M or -$1.52 per share, FY23 net loss of -$606,639 M or -$8.25 per share; a cash position of $777 M

  • 2023 Total Revenue of $434 million, Crysvita® revenue of $328 million and Dojolvi® revenue of $71 million,
  • 2024 Financial Guidance: Total Revenue between $500 million and $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million,
  • 2024 guidance for Net Cash Used in Operations expected to be less than $400 million
  • Runway Q3/2026; I say … needs an offering!

 

Blueprint Medicines' (BPMC +$10.77)

Net loss was -$110.9 M or -1.82 pre share and -$570 M or -$8.37 per share for FY23 while cash position was $767.2 M and a 2026 runway.

  • Revenue from rare-disease drug, Ayvakit, topped Q4/23 expectations, and BPMC stock surged.
  • For 2024, sales are expected to be a lower $360 million to $390 million.
  • Ayvakit treats several forms of cancer, advanced and idiopathic systemic mastocytosis. Patients with these diseases experience a damaging buildup of mast cells on their organs.
  • During Q4, Ayvakit generated $71 million in sales, growing 31% sequentially.
  • That beat expectations for $67.1 million <according to FactSet>.
  • BPMC stock has a strong Relative Strength Rating of 92 out of a best-possible 99. The RS Rating means BPMC stock ranks in the top 8% of all stocks in terms of 12-month performance, according to IBD Digital.
  • Shares are below their 50-day line, but remain above their 200-day moving average, <IBD>

Alnylam Pharmaceuticals (ALNY -$16.72)

Net loss from Q4 = $96.6 M or -$1.10 per share and FY23 -$202.618 M or -$3.52 per share;   a cash position of $2.44 B due to the receipts of a $310 million upfront payment from Roche in connection with partnership to co-develop and co-commercialize zilebesiran, a $100 million payment from Regeneron in connection with the achievement of certain criteria during early clinical development for its CNS program, ALN-APP, and nearly $150 million from employee option award exercises,.

  • For Q4/23, reported revenue of $439.72 million, up 31.2% over the same period last year. EPS came in at -$1.10, compared to -$1.68 in the year-ago quarter.
  • The reported revenue represents a surprise of -1.31% over the Zacks Consensus Estimate of $445.57 million. With the consensus EPS estimate being -$1.20, the EPS surprise was +8.33%.
  • Product revenues, net: $346.29 million versus $339 million estimated by 10 analysts on average. Compared to the year-ago quarter, this number represents a +32.3% change.
  • Net revenues from research collaborators: $76.41 million versus the 10-analyst average estimate of $98.87 million. The reported number represents a year-over-year change of +8.2%. Royalty revenue: $17.02 million compared to the $11.68 million average estimate based on 10 analysts. The reported number represents a change of +527% year over year.
  • Net Product Revenues- Givlaari: $59.30 million versus $59.26 million estimated by eight analysts on average. Compared to the year-ago quarter, this number represents a +26% change. Net Product Revenue- Oxlumo: $32.73 million versus the eight-analyst average estimate of $32.98 million. The reported number represents a year-over-year change of +37.4%.
  • Net Product Revenues- Onpattro: $79.01 million versus $75.19 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a -35.4% change. Net Product Revenue- Amvuttra: $175.25 million versus the seven-analyst average estimate of $173.63 million. <ZACKS>

 

Vericel (VCEL) … earnings whisper

  • Vericel Corporation (VCEL) is expected to deliver a year-over-year increase in earnings on higher revenues when it reports results for the quarter ended December 2023.
  • Expected to post quarterly earnings of $0.18 per share in its upcoming report, which represents a year-over-year change of +50%.
  • Revenues are expected to be $65 million, up 23.4% from the year-ago quarter. <ZACKS>

CRISPR Therapeutics AG (CRSP) … earnings’ whispers

  • is expected to deliver a year-over-year increase ….
  • In earnings on higher revenues when it reports results for the quarter ended December 2023.
  • Revenues are expected to be $138.09 million, up 1380800% from the year-ago quarter.
  • For the last reported quarter, it was expected that CRISPR Therapeutics AG would post a loss of $1.98 per share when it actually produced a loss of $1.41, delivering a surprise of +28.79%. <ZACKS>

Ionis Pharmaceuticals (IONS) … earnings whispers

  • Expected to post quarterly loss of $0.78 per share in its upcoming report, which represents a year-over-year change of -110.8%.
  • Revenues are expected to be $183.6 million, up 20.8% from the year-ago quarter.
  • The consensus EPS estimate for the quarter has been revised 12.59% lower over the last 30 days to the current level.
  • For the last reported quarter, it was expected that Ionis Pharmaceuticals would post a loss of $1.01 per share when it actually produced a loss of $1.03, delivering a surprise of -1.98%.
  • Over the last four quarters, the company has beaten consensus EPS estimates three times. <ZACKS>

Intellia Therapeutics (NTLA) … earnings whispers

  • Expects a year-over-year decline in earnings on higher revenues when Intellia Therapeutics, Inc. (NTLA) reports results for the quarter ended December 2023.
  • NTLA is expected to post quarterly loss of $1.47 per share in its upcoming report, which represents a year-over-year change of -5%.
  • Revenues are expected to be $15.1 million, up 11.3% from the year-ago quarter. <ZACKS>

 

The Harvard Apparatus RN (HRGN) formerly the Biostage (BSTG) Chronicles: shows how the “pimp/pump and promote” works …

HRGN closed down -$0.10 with 1,090 shares traded on Thursday with 590 shares traded after Wednesday’s +$0.00 with 1,182 shares traded, Tuesday’s (down) -$0.43 with 1.961 shares traded following Monday’s -$0.02 with 5,850 shares traded <3-month average = 1,390 shares)

  • Did you notice how it barely trades unless volume is “pumped” – is it by DST Capital, run by the president of HRGN’s Hong Yu and neighbor, Mrs. Mrs. bin Zhao of Weston, Mass – funded by Lui Dong Hai, chairman of Dixintong Technology Group (D. Phone, the largest smartphone retailer in China) the husband of Mrs. bin Zhao??

This question needs to be answered as the NEED to desperately finance by private placement – past Chinese investors must be feeling … WHAT?

  • Making promises to Chinese investors, management can't keep isn't a real strategy, especially when you're asking/raising money to continue operations as HRGN was Out-of-Cash 1/2/24!

 

The top three (3) performing in the session:

  • Thursday: Blueprint Medicine (BLMC), CRISPR Therapeutics (CRSP) and Vericel (VCEL)
  • Wednesday: CRISPR Therapeutics (CRSP), Blueprint Medicine (BLMC) and Sage Therapeutics (SAGE)
  • Tuesday: Adverum Biotechnologies (ADVM) and Homology Medicine (FIXX)
  • Monday: CRISPR Therapeutics (CRSP), Beam Therapeutics (BEAM) and Intellia Therapeutics (NTLA)

The worst three (3) in the session:  

  • Thursday: Alnylam Pharmaceuticals (ALNY), Ionis Pharmaceuticals (IONS) and Sage Therapeutics (SAGE)
  • Wednesday: Adverum Biotechnologies (ADVM), uniQure NV (QURE) and Homology Medicine (FIXX)
  • Tuesday: Alnylam Pharmaceuticals (ALNY), Blueprint Medicine (BLMC) and CRISPR Therapeutics (CRSP)
  • Monday: Alnylam Pharmaceuticals (ALNY), Ionis Pharmaceuticals (IONS) and Graphite Bio (GRPH)

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.