May 1, 2025 4:45pm

Positioned to navigate the future with cash and runways

Q1/25 earnings:

  • Blueprint Medicine (BPMC) Net income of $500 K or -$0.01 per share, $149.4 M in revenue, $899.8 M in cash with a 2027 runway; BPMC shares have climbed nearly 3% since the beginning of the year. The stock has decreased 2% in the last 12 months.
  • Alnylam Pharmaceuticals (ALNY) Net loss of -$57.5 M or -$0.44, revenue or $594.2 M, $2.63 B in cash with a 2028 runway. Year to date, shares of SAGE have rallied 40% against the industry’s decline of 4%. ALNY shares have risen 12% since the beginning of the year. The stock has increased 83% in the last 12 months.
  • Moderna (MRNA) Net loss of -$1 B or -$2.52, revenue of $108 M, $8.4 in cash with a 2028 runway.

Coming: BLFS and NTLA  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 portends new month of May… https://www.regmedinvestors.com/articles/13901

Wednesday night’s RegMed Investors (RMi) Closing Bell: the sector breathes as canary-in-the coal-mine flaps its wings … https://www.regmedinvestors.com/articles/13900

 

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

 

Thursday: The Dow closed UP +83.42 points or +0.21%, the S&P closed UP +35.05 points or +0.63% while the Nasdaq closed UP +264.40 points or +1.52%

  • Indexes closed positive as cell and gene therapy sector dives negative

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

  • The ISM manufacturing index, considered a key measure of broader economic health, edged lower to 48.7% on the month, down 0.3% point from March but better than the 47.8% consensus forecast. Any reading below 50% represents contraction for the sector, but historically a sustained level above 42.3% is considered consistent with economic expansion. The survey showed declining readings for production, new export orders, imports and inventories. Employment nudged up 1% to 46.5% while prices also rose, but just slightly to 69.8%.

Thursday’s advance/decline line opened with a negative 16 incliner, 17 decline and 2 flats ending with a negative close of 14 incliners, 19 decliners and 2 flats      

Metrics:  Thursday, the IBB was down -1.70%, the XBI was down -0.46% while the VIX was down -0.23 points or -0.93% at 24.47

 

Q2/25 – May – 1 negative close

  • 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

 

Thursday’s Closing UP (10 of 14)

  • Blueprint Medicine (BPMC +$9.75 after Wednesday’s +$1.02),
  • Ionis Pharmaceuticals (IONS +$1.00 after Wednesday’s +$0.90),
  • Mesoblast (MESO +$0.54 after Wednesday’s -$0.51 after Tuesday’s +$0.49 after Monday’s -$0.40),
  • BioLife Solutions (BLFS +$0.49 after Wednesday’s -$0.60 after Tuesday’s +$0.24 after Monday’s -$0.49),
  • Lenz Therapeutics (LENZ -$0.36 after Wednesday’s +$1.89 after Tuesday’s -$0.65 after Monday’s +$0.40
  • Solid Biosciences (SLDB +$0.34 after Wednesday’s +$0.26 after Tuesday’s -$0.18 after Monday’s -$0.07),
  • uniQure NV (QURE +$0.24 after Wednesday’s+$0.90 after Tuesday’s -$0.11),
  • Regenxbio (RGNX +$0.22),
  • AxoGen (AXGN +$0.21 after Wednesday’s -$0.24 after Tuesday’s +$0.33 after Monday’s +$0.22)
  • Sage Therapeutics (SAGE +$0.19 after Wednesday’s -$0.31 after Tuesday’s -$0.17),

Flat (2)

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

Thursday’s Closing DOWN (10 of 19): 

  • Alnylam Pharmaceuticals (ALNY -$8.11 after Wednesday’s +$6.05 after Tuesday’s +$2.34 after Monday’s +$3.29
  • Moderna (MRNA -$1.51 after Wednesday’s +$0.72 after Tuesday’s +$0.36 after Monday’s +$0.24),
  • CRISPR Therapeutics (CRSP -$0.94 after Wednesday’s +$1.27 after Tuesday’s -$0.48 after Monday’s -$0.39
  • MiMedx (MDXG -$0.56 after Wednesday’s -$0.04 after Tuesday’s +$0.09 after Monday’s -$0.08),
  • Intellia Therapeutics (NTLA -$0.23 after Wednesday’s +$0.67 after Tuesday’s -$0.28 after Monday’s +$0.17),
  • Verve Therapeutics (VERV -$0.17),
  • Brainstorm Cell Therapeutics (BCLI -$0.14),
  • Vericel (VCEL -$0.10 after Wednesday’s -$1.34 after Tuesday’s +$0.62 after Monday’s -$0.99
  • Beam Therapeutics (BEAM -$0.10),
  • Caribou Biosciences (CRBU -$0.0491 after Wednesday’s -$0.0014),

 

The BOTTOM LINE: The Nasdaq popped holding onto Thursday gains to close over 1.5% higher, while the Dow winning streak keeps on rolling.

  • The Russell 2000 also cracked 1% to the upside, not seeing much of a sell-off there.
  • 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,

 

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!

 

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>

 

May ‘25: understand the “flow” …

  • 5/1 - Thursday closed negative with 14 positive, 19 negative and 2 flats

 

What could 2025 have in store for the capital access space – DESPERATE   

  • "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:   

  • Thursday: Blueprint Medicine (BPMC), Ionis Pharmaceuticals (IONS) and Mesoblast (MESO)
  • 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)

The worst three (3) in the session: 

  • Thursday: Alnylam Pharmaceuticals (ALNY), Moderna (MRNA) and CRISPR Therapeutics (CRSP)
  • 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)

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.