April 30, 2025 4:43pm

From the impact of econs, earnings and tariffs as well as portfolio pain in pursuit of the extended-term goals being underestimated

Q1/25 earnings:

  • Ionis Pharmaceuticals (IONS) Net loss of $147 M or -$0.75 per share, $132 M in revenue, $2.1 B in cash with a 2027 runway; shares have lost about 14.7% since the beginning of the year versus the S&P 500's decline of -5.5%.
  • Sage Therapeutics (SAGE) Net income of -$62.2 M or -$1.01, collaboration revenue or $13.8 M, $424 M in cash with a 2027 runway. Year to date, shares of SAGE have rallied 40% against the industry’s decline of 4%.
  • MiMedx Group (MDXG) Net income of +$7 M or +$0.05, net sales of $88 M, $106 M in cash with a 2027 runway.

Coming: MRNA and ALNY 5/1, BLFS 5/8, RGNX 5/12

Never leave an investor uninformed!  

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

My mission is to provide clear, actionable guidance on a regular basis to help investors and traders navigate the uncertain and often irrational financial markets. I put hours of effort to create reporting, while leveraging my 40 years of operating and research experiences to analyze the key market-moving events and distill that into a cogent outlook.

 

RegMed Investors’ (RMi) pre-open: earnings and consequences … https://www.regmedinvestors.com/articles/13899   

Tuesday night’s RegMed Investors (RMi) Closing Bell: Fluctuation with causation … https://www.regmedinvestors.com/articles/13898

 

RegMed Investors (RMi) Research Note: Harvard Apparatus GT (OTCQB: HRGN) Chronicles: Yet another "screw job" for/on investors ... "wanting to effect a reverse split of its outstanding common stock" in its recent proxy statement filing!! … https://www.regmedinvestors.com/articles/13812

 

Wednesday: The Dow closed UP +141.74 points or +0.35%, the S&P closed UP +8.23 points or +0.15% while the Nasdaq closed DOWN -14.98 points or -0.09%

  • Indexes closed mixed Wednesday

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

  • Q1 gross domestic product (GDP)declined at a 0.3% rate <needs revision>
  • ADP private payroll growth slowing in April to just 62 K during the month
  • However, other data pointed to consumer spending growing up +0.7% in March, topping the 0.5% economists expected.

Wednesday’s advance/decline line opened with a negative 10 incliner, 23 decline and 2 flats ending with a positive close of 23 incliners, 10 decliners and 2 flats      

Metrics:  Wednesday, the IBB was up +1.83%, the XBI was up +1.07% while the VIX was up +0.50 points or +2.06% at 24.67

 

Q2/24 -April – 11 positive and 10 negative closes

  • Q1/25 – March, 10 positive and 12 negative closes
  • February – 12 holiday, 11 negative and 7 positive closes
  • January - 2 holidays, 1 market close, 10 negative and 10 positive closes

 

Wednesday’s Closing UP (10 of 23)

Alnylam Pharmaceuticals (ALNY +$6.05 after Tuesday’s +$2.34 after Monday’s +$3.29

  • Lenz Therapeutics (LENZ +$1.89 after Tuesday’s -$0.65 after Monday’s +$0.40
  • CRISPR Therapeutics (CRSP +$1.27 after Tuesday’s -$0.48 after Monday’s -$0.39
  • Blueprint Medicine (BPMC +$1.02),
  • Ultragenyx Pharmaceuticals (RARE +$0.91 after Tuesday’s +$0.09 after Monday’s -$0.11),
  • uniQure NV (QURE +$0.90 after Tuesday’s -$0.11),
  • Ionis Pharmaceuticals (IONS +$0.90),
  • Moderna (MRNA +$0.72 after Tuesday’s +$0.36 after Monday’s +$0.24),
  • Intellia Therapeutics (NTLA +$0.67 after Tuesday’s -$0.28 after Monday’s +$0.17),
  • Solid Biosciences (SLDB +$0.26 after Tuesday’s -$0.18 after Monday’s -$0.07),

Flat (2)

  • Harvard Apparatus RT (OTCQB: HRGN $0.00 after Tuesday’s -$0.11 after Monday’s $0.00)
  • Homology Medicine (FIXX)

Wednesday’s Closing DOWN (10 of 10): 

  • Vericel (VCEL -$1.34 after Tuesday’s +$0.62 after Monday’s -$0.99
  • BioLife Solutions (BLFS -$0.60 after Tuesday’s +$0.24 after Monday’s -$0.49),
  • Mesoblast (MESO -$0.51 after Tuesday’s +$0.49 after Monday’s -$0.40),
  • Sage Therapeutics (SAGE -$0.31 after Tuesday’s -$0.17),
  • AxoGen (AXGN -$0.24 after Tuesday’s +$0.33 after Monday’s +$0.22)
  • Agenus (AGEN -$0.16 after Tuesday’s +$0.33 after Monday’s +$0.22),
  • MiMedx (MDXG -$0.04 after Tuesday’s +$0.09 after Monday’s -$0.08),
  • bluebird bio (BLUE -$0.04),
  • Voyager Therapeutics (VYGR -$0.025),
  • Caribou Biosciences (CRBU -$0.0014),

 

The BOTTOM LINE:  Indexes and “our” universe recovered from steep early-session Wednesday as the “Street”capped a volatile month and investors digested a deluge of economic data,

  • Cell and gene therapy sector equities POPPED on the last session of April after turning lower AGAIN on Tuesday following Monday after Friday’s negative coming close to the end of month’s week and beginning of earnings reports and more macroeconomic data …

 

I was once told by a “very” prominent figure early in my career, “write it right, keep it tight without hype and never lose the stride to guide the reader – him” – they were pretty simple instructions to follow!

 

Reiterating … My message, “The Trump administration … realizes drugs need to be made in America, not in foreign companies to be imported.

  • Also, most drug research is American based and if Kennedy slows or impedes their development or more – he will be bounced faster than a bond sell-off!!
  • Mark my words …!!

My perspective is retail investors should NOT be dipping their toes back into the cell and gene therapy sector until after LPS (loss-per-share) earnings report.

  • NO all ins – just add those with solid partners, lengthened runways and solid cash positions leveraged by solid management teams – there I see losses going forward!

“I hate to be so negative or contrarian but, this is a NO spin zone and truth is its product; I can always be WRONG but, I am mostly EARLY!”

  • If I have learned one thing as a former research analyst in a venture and public fund, operating executive, x-FBI, x-military officer and an adjunct (MBA) business school professor now a journalist; my life and your portfolio are largely tied to knowledge of price movements and questioning pricing targets and more.

I don’t have much to write/say other than “electronic trading and uncle algo” slips in and out, back doors reaping sector disharmony and then pops right back for some profiteering.

  • What can I say, love ‘em, leave ‘em and welcome back to save the sector’s honor – “uncle algo and his electronic dwarfs.” <me>

 

April ‘25: understand the “flow” … it's over!

  • 4/30 - Wednesday closed positive with 23 positive, 10 negative and 2 flats
  • 4/29 – Tuesday closed negative with 14 positive, 20 negative and 1 flat
  • 4/28 - Monday closed negative with 15 positive, 19 negative and 2 flats
  • 4/25 – Friday closed negative with 12 positive, 20 negative and 3 flats
  • 4/24 – Thursday closed positive with 29 positive, 5 negative and 1 flat
  • 4/23 – Wednesday closed positive with 22 positive, 10 negative and 3 flats
  • 4/22 – Tuesday closed positive with 30 positive, 3 negative and 2 flats
  • 4/21 - Monday closed positive with 21 positive, 13 negative and 1 flat
  • 4/18 – Friday, markets closed for Good Friday
  • 4/17 – Thursday closed positive with 25 positive, 7 negative and 3 flats
  • 4/16 – Wednesday closed negative with 2 positive, 31 negative and 2 flats
  • 4/15 – Tuesday closed barely positive with 17 positive, 16 negative and 2 flats
  • 4/14 - Monday closed positive with 23 positive, 7 negative and 5 flats
  • 4/11 – Friday closed positive with 28 positive, 5 negative and 2 flats
  • 4/10 – Thursday closed negative with 4 positive, 30 negative and 1 flat
  • 4/9 – Wednesday closed positive with 33 positive, 1 negative and 1 flat
  • 4/8 - Tuesday closed negative with 4 positive, 30 negative and 1 flat
  • 4/7 - Monday closed negative with 13 positive, 20 negative and 2 flats
  • 4/4 – Friday closed negative with 3 positive, 30 negative and 2 flats
  • 4/3 – Thursday closed negative with 2 positive, 32 negative and 1 flat
  • 4/2 - Wednesday closed positive with 9 positive, 25 negative and 1 flat
  • 4/1 - Tuesday closed negative with 5 positive, 28 negative and 2 flats

 

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

  • "You'll see a number of cell and gene therapy companies DESPARITE drive for financings – 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:   

  • Wednesday: Alnylam Pharmaceuticals (ALNY), Lenz Therapeutics (LENZ) and CRISPR Therapeutics (CRSP)
  • Tuesday: Alnylam Pharmaceuticals (ALNY), Vericel (VCEL) and Mesoblast (MESO)
  • Monday: Alnylam Pharmaceuticals (ALNY), Blueprint Medicine (BPMC) and Agenus (AGEN)
  • Friday: Alnylam Pharmaceuticals (ALNY), Blueprint Medicine (BPMC) and Ultragenyx Pharmaceuticals (RARE)

The worst three (3) in the session: 

  • Wednesday: Vericel (VCEL), BioLife Solutions (BLFS) and Mesoblast (MESO)
  • Tuesday: Lenz Therapeutics (LENZ), CRISPR Therapeutics (CRSP) and Intellia Therapeutics (NTLA)
  • Monday: Vericel (VCEL), BioLife Solutions (BLFS) and Mesoblast (MESO)
  • Friday: Lenz Therapeutics (LENZ), Vericel (VCEL) and CRISPR Therapeutics (CRSP)

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.