December 29, 2025 4:39pm

Taking profits and as RARE’s P3 did NOT achieve statistical significance against the primary endpoints <read below>

Last week of December, Q4 and FY25

Pre-open Signal Results: 3 Hits and 1 Miss

RMi collects, curates, interprets and disseminates cell and gene therapy sector (C&GT) fact-based news, trusted and verified intel and share pricing data to bridge the gap to an investment decision

Never leave a retail investor uninformed!  It’s not always time to buy or sell; but it is time to KNOW and why! I chose to speak-up when many analysts have shut-up!

 

Looking forward to 2026, investors are likely to focus on whether these high-performing biotech stocks can sustain their momentum through clinical execution, regulatory milestones, commercialization progress, and potential strategic transactions.

 

News: Ultragenyx Pharmaceutical (RARE -$14.47 or -42.32% to $19.72) stock plunged in huge volume and trading; and was temporarily halted Monday morning. Results from P3 studies on its monoclonal antibody named setrusumab did NOT achieve statistical significance against the primary endpoints for reduction in fracture rate. Setrusumab is being tested as a treatment for osteogenesis imperfecta, a disease-causing brittle bone.

 

Today’s session falls within the Santa Claus Rally, a historically strong 7-day stretch (12/24/25 – 1/5/26) for stocks that spans the final days of December and early January with thin trading volumes

Monday: The Dow closed DOWN -249.04 points or -0.51%, the S&P closed DOWN -24.20 points or -0.35% while the Nasdaq closed DOWN -118.748 points or -0.50%

  • Theme of the session: the throes of the Santa Claus rally period

Henry’omics: We need to more than consider the macro-economic environment to comprehend the micro re “our” universe of cell and gene therapy (C&GT) sector

  • Key releases are the minutes from the Fed's latest policy meeting, due out tomorrow, and the Labor Department's weekly jobless claims, expected Wednesday.

Monday’s (my) 40-company covered sector’s advance/decline line opened negative with 7 incliners, 3 decliners and 2 flats ending with a negative close of 12 incliners, 24 decliners and 4 flats

The CBOE Fear (VIX) index, Monday closed at 14.15 after Friday closed at 13.60, Thursday was Christmas, Wednesday closed at 13.80, Tuesday’s 13.95 following the previous Monday’s 14.11

Metrics: Monday …

  • The RUT was down -14.55 points or -0.57%,
  • The XLV was down -0.24 points or -0.15%,
  • The NBI was down -42.83 points or -0.74%;
  • The XBI was down -1.54 points or -1.23%
  • The IWM was down -1.54 or -0.61%;
  • The IBB was down -1.27 points or -0.74%,
  • The VIX was up +0.55 points or +4.04% at to 14.15

 

Q4/25 – December – 1 holiday, 8 positive and 12 negative closes

  • November – 1 holiday, 8 positive and 11 negative closes
  • October -1 neutral, 11 positive and 12 negative closes

 

Monday Closing UP (14 of 14) 

  • BioNTech (BNTX +$0.68 after Friday’s -$0.51),
  • AxoGen (AXGN +$0.65 after Friday’ s +$0.16),
  • Capricor Therapeutics (CAPR +$0.35 after Friday’s +$0.01).
  • Precigen (PGEN +$0.29),
  • IQVIA Holdings (IQV +$0.22 after Friday’s +$0.80),
  • BioLife Solutions (BLFS +$0.22 after Friday’s -$0.51),
  • Cellectis SA (CLLS +$0.21),
  • Regenxbio (RGNX +$0.14 after Friday’s -$0.30),
  • Wave Life Sciences (WVE +$0.09 after Friday’s -$0.48),
  • Ionis Pharmaceuticals (IONS +$0.05 after Friday’s -$0.72),
  • Generation Bio (GBIO +$0.04),
  • MiMedx (MDXG +0.01),

Flat (4)

  • Adverum Biotechnologies ADVM) – acquired
  • Brainstorm Cell Therapeutics (BCLI $0.00 after Friday’s +$0.08)
  • Harvard Apparatus RT (OTCQB: HRGN $0.00 after Friday’s +$0.04 with 50 shares traded),
  • Rocket Therapeutics (RCKT)

Monday’s Closing DOWN (10 of 24): 

  • Ultragenyx Pharmaceuticals (RARE -$14.47 after Friday’s -$0.39),
  • Vertex (VRTX -$3.12),
  • Alnylam Pharmaceuticals (ALNY -$1.77),
  • CRISPR Therapeutics (CRSP -$1.11 after Friday’s -$1.63),
  • Mesoblast (MESO -$0.54 after Friday’s +$0.10),
  • Arrowhead Pharmaceuticals (ARWR -$0.54),
  • Lenz Therapeutics (LENZ -$0.35 after Friday’s -$0.27)
  • Supernus Therapeutics (SUPN -$0.32),
  • Sarepta Therapeutics (SRPT -$0.30),
  • Intellia therapeutics (NTLA -$0.30)

 

The Bottom Line: More of the … WHYs

  • Stuck in the red zone while trimming share pricings
  • Volume was higher on the Nasdaq and considerably higher on the NYSE compared with Friday. On the Nasdaq, falling stocks topped rising issues 5-to-2. On the NYSE, losers edged out winners by nearly 2-to-1.
  • Followed by the 10-year Treasury yield shedding 2 basis points to 4.11%

As I had also written, cash is king for investors who want to keep some liquidity and avoid having to sell in a down market,

  • I would be following share pricing’s ascension on current biotech, cell and gene therapy sector upsides; so, some profiteering can be “situated” while maintaining a position, less elevated!

Most C&GT sector equities dramatically dived on Monday ... 

 

Re Capricor Therapeutics (CAPR +$0.07 after Friday’s -$0.27 after Wednesday’s -$0.57) …  I am STICKING to my CONVICTION that CAPR is overbought and whose valuation is disconnected from fundamentals.

  • Doubts still abound re: regulatory fragility and marginal HOPE-3 results and valuation concerns remain a key hang-up!
  • CAPR remains a structurally challenged, chronically unprofitable company whose valuation is disconnected from fundamentals.
  • It has begun; timing will be everything!
  • The higher CAPR rises, the steeper the fall!
  • Is the RISK woth it ...??

Take it a session at a time … the REAL question is … how long will this sentiment and conviction “run” last in the “clean-out-the-exposure” December?

 

5th and last week of December, Q4 and FY25

  • Monday closed negative with 12 positive, 24 negative and 4 flats

4th week of December:

  • Friday closed negative with 11 positive, 28 negative and 1 flat
  • Thursday was a holiday
  • Wednesday closed positive with 31 positive, 5 negative and 4 flats - not published
  • Tuesday closed negative with 14 positive, 24 negative and 2 flats
  • Monday closed positive with 30 positive, 8 negative and 2 flats

 

As the leading voice of cell and gene therapy investors; I am NOT always a doom and gloom analyst/journalist but a man who has been “there” (BUY, SELL, VC and multiple operating roles) with the gray hair and a readership following with real numbers to prove it!

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

  • I hate to be so negative or contrarian but, this is a NO spin zone and facts and truth are its products; I can always be WRONG but, I am mostly EARLY!
  • if I wanted to be liked, I wouldn’t have been an analyst/journalist.

 

Why do I keep repeating, framed in a different para, so investors can make the connection

The top three (3) performing in the session:   

  • Monday: BioNTech (BNTX), AxoGen (AXGN) and Capricor Therapeutics (CAPR)
  • Friday: IQVIA Holdings (IQV), AxoGen (AXGN) and Mesoblast (MESO),
  • Thursday: was a holiday

The worst three (3) in the session:

  • Monday: Ultragenyx Pharmaceuticals (RARE), Vertex (VRTX) and Alnylam Pharmaceuticals (ALNY)
  • Friday: CRISPR Therapeutics (CRSP) and Moderna (MRNA) and Ionis Pharmaceuticals (IONS)
  • Thursday: was a holiday

 

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.