February 6, 2025 4:44pm

The break is possibly the most important shot of the game; determining the layout of share pricing, futures, econs and news breaking seemingly control which gets the upside shots of the sector, and sets the tone for who is in the hole or the lead

News: Ultragenyx Pharmaceutical (RARE +$0.37) reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA). RARE stated that treatment with UX111 led to a rapid decline in CSF-HS levels within the 1st month, regardless of patient age or disease stage. As of 8/24, the median CSF-HS reduction was 65% across all treated patients and 66% in the modified intention-to-treat (mITT) group.

Earnings: MDXG, BPMC, IONS, SAGE, MRNA and ALNY – so far <see The Bottom Line>

I say today what others won't, so you can do what others can't!

Never leave an investor uninformed!  

VIP: I will be partially unavailable in the week 2/3 – 2/7/25 due my “better half or the babe” underwent successful surgery yet will continue to be hospitalized – say a prayer please!

 

On point, short on words, long on facts and being judicious!

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

 

Thursday: The Dow closed DOWN -125.65 points or -0.28%, the S&P closed UP +22.09 points or +0.36% while the Nasdaq closed UP +99.66 points or +0.51%

  • Indexes closed mixed jobs report and earnings coming

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

  • Labor costs, productivity come in below expectations. Unit labor costs, which measure compensation minus productivity, rose at a seasonally adjusted annualized rate of 3% for the October-through-December period in 2024. That was smaller than the 3.3% Dow Jones estimate and brought the cost increase over the four-quarter period to 2.7%. Labor productivity rose 1.2% for the period, below the 1.4% estimate, and brought the annual rate down to 2.3%.

Thursday’s advance/decline line opened with a negative close at the close of 13 incliner, 18 decliners and 4 flats ending with a negative close of 8 incliners, 24 decliners and 2 flats

Metrics:  Thursday, the IBB was down -1.77%, the XBI was down -1.34% while the VIX was down -0.33 points or -2.10% at 15.44

 

Q1/25 – February – 2 negative and 2 positive closes

  • January - 2 holidays, 1 market close, 10 negative and 10 positive closes

Q4/24 –

  • December - 1 holiday, 6 positive and 15 negative closes
  • November – 1 holiday, 10 negative and 10 positive closes              
  • October: 8 positive and 15 negative sessions

 

Thursday’s Closing UP (8 of 8)

  • Alnylam Pharmaceuticals (ALNY +$1.94 after Wednesday’s +$5.31 after Tuesday’s -$3.55 and Monday’s +$3.24)
  • Ultragenyx Pharmaceuticals (RARE +$0.41 after Wednesday’s +$1.19 after Tuesday’s -$0.76 and Monday’s +$0.39),
  • Compass therapeutics (CMPX +$0.12),
  • Precigen (PGEN +$0.11),
  • Solid Biosciences (SLDB +$0.09),
  • uniQure NV (QURE +$0.08),
  • Sage Therapeutics (SAGE +$0.02),
  • Sangamo Therapeutics (SGMO +$0.01),

Flat (2)

  • Harvard Apparatus RT (OTCQB: HRGN),
  • Homology Medicine (FIXX)

Thursday’s Closing DOWN (10 of 24): 

  • Vericel (VCEL -$2.10 after Wednesday’s +$2.37 after Tuesday’s -$0.37 and Monday’s +$0.52),
  • CRISPR Therapeutics (CRSP -$1.61 after Wednesday’s +$1.73 after Tuesday’s -$0.49 and Monday’s -$1.59),
  • Moderna (MRNA -$1.37 after Wednesday’s +$0.81 after Tuesday’s -$2.38 and Monday’s -$2.87),
  • Blueprint Medicine (BPMC -$0.75 after Wednesday’s -$3.03 after Tuesday’s -$1.90 after Monday’s -$1.03),
  • AxoGen (AXGN -$0.55 after Wednesday’s +$0.80),
  • Ionis Pharmaceuticals (IONS -$0.54 after Wednesday’s +$1.53 after Tuesday’s -$0.49 and Monday’s +$0.23),
  • BioLife Solutions (BLFS -$0.53),
  • Intellia Therapeutics (NTLA -$0.38 after Wednesday’s +$0.89),
  • Verve Therapeutics (VERV -$0.27),
  • bluebird bio (BLUE -$0.27),

 

The BOTTOM LINE:  Thursday “flipped the bird” negative after Wednesday and Tuesday’s session closed positive upending Monday's negative close.

  • I don’t have much to write/say other than “electronic trading and uncle algo” slipped out the back door after its previous visit.

Quotes to be considered …

  • “Today’s price action definitely has felt idiosyncratic, and that’s a lot of times what we see during earnings season where investors focus on individual company fundamentals.”
  • Meanwhile, investors seem to have shaken off worries around tariffs, which began on Monday after President Donald Trump announced a 10% levy on Chinese imports over the weekend. Sentiment improved after the president paused duties on Mexican and Canadian goods. < Zachary Hill, head of portfolio management at Horizon Investments>

 

Macro news ... Eli Lilly (LLY) shares are up about 3% after posting Q4/24.

  • CEO Dave Ricks reiterated that Eli Lilly continues to believe the market for its Zepbound obesity treatment is “hundreds of millions of people globally.”
  • As for the forecast, JPMorgan said the fiscal 2025 outlook for $58 billion to $61 billion in revenue is “highly achievable.” Bank of America analyst Tim Anderson agreed and noted it doesn’t assume a ramp-up in the rate of new Mounjaro and Zepbound prescriptions, which “some may find reassuring.”
  • One of the next big developments will be the release of clinical trial data for obesity pill orforglipron.

 

Earnings are a-comin’ … from Q4/24 and FY24 are due …

  • Sage Therapeutics (SAGE) - Tuesday, 2/11
  • Blueprint Medicine (BPMC) – Thursday, 2/13
  • Alnylam Pharmaceuticals (ALNY), Thursday, 2/13
  • Moderna (MRNA) - Friday, 2/14
  • Ionis pharmaceuticals IONS) – Wednesday, 2/19
  • MiMedx (MDXG) – Wednesday, 2/26

 

February ‘25: understand the “flow” …

  • 2/6 – Thursday ended with a negative close at the close of 8 incliner, 24 decliners and 3 flats
  • 2/5 - Wednesday ended with a positive close at the close of 26 incliner, 4 decliners and 5 flats
  • 2/4 - Tuesday ended with a positive close at the close of 24 incliner, 9 decliners and 2 flats
  • 2/3 – Monday - ended with a negative close at the close of 8 incliner, 25 decliners and 2 flats

 

As I have written “Many sector companies are engaged in cost realignment programs, which won’t be realized consequences over the next few Qs where the operating expense levels; spending on SG&A (selling, informational, and administrative) and R&D (research and development) facilitate the need to extend runways and enable expectation of clinical innovations.”

  • "I think the market's going to come back to earnings, the first couple of months of 2025, and 1st H/25 will be a shifting cell and gene therapy sector due to earnings releases... I still believe stocks will swing upward pre and them more post releases

Big Point to remember “Think about it; the biggest problem the cell and gene therapy sector it’s all to the future …  99% of them have no earnings!

  • If rates are higher for longer, that means that sector companies will continue to struggle or be unable to raise capital and therefore be subject to debt servicing firms!”
  • 2024 saw very little life IPOs (initial public offering) market and secondaries – I have seen private investments, mortgages, sell-offs, as companies deferred and struggle to gain further market access i.e., public. <Me>

What could 2025 have in store for the capital access space?

  • "You'll see a number of cell and gene therapy comandantes drive for secondaries – they’re desperate for cash, but I do think a lot of cell and gene therapy sector companies NEED to be focused on what is driving their own growth —they're focused on a lot of change BUT … that's happening in pipelines … capital expansion is second.

 

Why do I keep repeating, so investors can make the connection …

The top three (3) performing in the session:   

  • Thursday: Alnylam Pharmaceuticals (ALNY), Ultragenyx Pharmaceuticals (RARE) and Compass Therapeutics (CMPX)
  • Wednesday: not listed
  • Tuesday: Blueprint Medicine (BPMC), Ultragenyx Pharmaceuticals (RARE) and Regenxbio (RGNX)
  • Monday: Alnylam Pharmaceuticals (ALNY), Vericel (VCEL) and Ultragenyx Pharmaceuticals (RARE

The worst three (3) in the session: 

  • Thursday: Vericel (VCEL), CRISPR Therapeutics (CRSP) and Moderna (MRNA)
  • Wednesday: Not listed
  • Tuesday: Alnylam Pharmaceuticals (ALNY), Moderna (MRNA) and Ionis Pharmaceuticals (IONS)
  • Monday: Moderna (MRNA), CRISPR Therapeutics (CRSP) 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 it’s 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.