September 2, 2025 4:56pm

As I had written, “September has been the weakest month of the year for stock performance”.  Although collab deals articulate coming upsides for sector developmental treatments!

News: Arrowhead Pharmaceuticals (ARWR +$3.65 to $25.68) and Novartis (NVS +$0.09 or -0.52% to $126.64) inked a licensing deal for an experimental Parkinson's drug, sending the share price surging; NVS agreed to pay ARWR $200 M up front and up to $2 B in additional milestones, plus royalties on sales to license its pre-clinical drug, ARO-SNCA. Vertex Pharmaceuticals (VRTX +$9.98 to $01.00) initiated a multi-target drug discovery collaboration with Enlaza Therapeutics’ (private) proprietary War-Lock™ small format drug conjugates and T-cell engagers for certain autoimmune diseases and improved conditioning in sickle cell disease and beta thalassemia. Enlaza will receive $45 M inclusive of an upfront payment, an equity investment and eligibility to receive more.

Remember, “The sector is what it is, until it isn’t, even if it doesn’t seem to be, it yet will be.”  Never leave an investor uninformed! 

 

I answer or qualify the sector’s equity’s movement; with an emphasis on which company is gaining or losing stride in your portfolio for the short-term

 

Monday’s RegMed Investors’ (RMi) pre-open: What and which news is good news? … https://www.regmedinvestors.com/articles/14087

RegMed Investors (RMi) Research Note: Harvard Apparatus GT (OTCQB): Just another wrinkle – Q2/25’s 10-Q – liabilities exceed cash position … https://www.regmedinvestors.com/articles/13812

RegMed Investors (RMi): New website coming soon! Same insights. Fresh design … https://www.regmedinvestors.com/articles/14083

 

Tuesday: The Dow closed DOWN -249.07 points or -0.55%, the S&P closed DOWN -44.72 points or -0.69% while the Nasdaq closed DOWN – 175.922points or -0.82%

  • Theme of the session, investors weighed the latest on tariffs while kicking-off a seasonally poor month for equities; rising yields also worry investors

Henry’omics: We need to more than consider the economic environment to comprehend the micro re “our” universe of cell and gene therapy

  • The Markit Purchasing Managers' Manufacturing Index, or PMI, rose to 53 in August but missed the expected reading of 53.3. Still, the result was higher than the 49.8 reading in July. The Institute for Supply Management Manufacturing Index met expectations at 48.7, exceeding July's 48 reading.

Tuesday’s my covered sector’s advance/decline line opened positive with 32 incliners, 7 decliners and 1 flat ending with a positive close of 22 incliners, 18 decliners and 0 flat

Metrics:  Tuesday, the IBB was up +2.19%, the XBI was up +3.03% while the VIX was up +1.05 points or +6.51% at 17.17

 

As of 9/2/25 – I added to my 35 cell and gene therapy coverage (now 40) to strength my comparable universe:

  • Capricor Therapeutics (CAPR)
  • Vertex (VRTX)
  • Rocket Therapeutics (RCKT)
  • Arrow Pharmaceuticals (AWWR)
  • Entrada Therapeutics (TRDA)

 

Q3 – 1 holiday and 1 positive closes

  • August – 11 negative and 9 positive closes
  • July – 1 market holiday, 13 positive and 9 negative closes

Q2/25: June – 1 market holiday, 1 neutral, 9 negative and 11 positive closes, May – 1 market holiday, 10 negative and 11 positive closes and April – 11 positive and 10 negative closes

 

Tuesday Closing UP (10 of 22) after Monday’s holiday

  • Ionis Pharmaceuticals (IONS +$14.86 after Friday’s +$0.01),
  • Vertex Pharmaceuticals (VRTX +$9.98),
  • Arrowhead Pharmaceuticals (ARWR +$3.65),
  • Ultragenyx Pharmaceuticals (RARE +$2.07 after Friday’s -$0.34),
  • CRISPR Therapeutics (CRSP +$0.83 after Friday’s -$1.22),
  • uniQure NV (QURE +$0.68 after Friday’s +$0.07),
  • BioNTech SE (BNTX +$0.62 after Friday’s -$1.93),
  • Lenz Therapeutics (LENZ +$0.62 after Friday’s +$0.43),
  • Alnylam Pharmaceuticals (ALNY +$0.49 after Friday’s -$5.82),
  • BioLife Solutions (BLFS +$0.29 after Friday’s +$0.13),

Flat (0)

Tuesday’s Closing DOWN (10 of 18): Monday was a holiday

  • IQV Holdings (IQV - $3.07 after Friday’s +$2.03),
  • Vericel (VCEL -$1.76 after Friday’s +$0.47),
  • Mesoblast (MESO -$0.71),
  • Supernus Therapeutics (SUPN -$0.58 after Friday’s -$0.25)
  • AxoGen (AXGN -$0.43),
  • Prime Medicine (PRME -$0.12),
  • Editas Medicine (EDIT -$0.10),
  • Entrada Therapeutics (TRDA -$0.09),
  • Harvard Apparatus RT (OTCQB: HRGN -$0.07 after Friday’s -$0.34)
  • Compass Therapeutics (CMPX -$0.06),
  • Beam Therapeutics (BEAM -$0.05 after Friday’s -$0.49),

 

The BOTTOM LINE:  Cell and gene therapy sector equities opened Tuesday to the upside and quickly traded lower in the late morning, to close in the upside starting September off … whew, lucky and luckier!

  • Coming-off a strong August while waiting for is the release of August’s jobs report on Friday and how it will influence the Fed’s interest rate decision coming mid-month
  • The Nasdaq 100 fell below a key short-term momentum indicator today, implying the index is in a downtrend. The benchmark is trading below its 50-day moving average level of 23,109.73 for the first time since April 30, weighed down by a slide in some tech names.
  • A 30-year Treasury of 5% is an also headwind
  • Volatility spiked on Tuesday, with a raft of macroeconomic concerns, including President Trump’s tariffs, weighing on investors/adding to uncertainty.

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!”
  • Yet, I was again on target … surtprising at today's 5 a.m.

Earnings: As earnings slowly come to the last few releases, collaborator revenues and runways has instilled share pricing with forward stability

  • Q2 results so far:  4 net incomes and 25 net losses of 29 releases to date

 

September: understand the “flow” …

  • 9/2 -Tuesday closed positive with 22 positive, 18 negative and 0 flat of 40 covered
  • 9/1 -Monday was a holiday

August’s final week:

  • 8/29 – Friday closed negative with 7 positive, 27 negative and 1 flat of 35 covered
  • 8/28 – Thursday closed negative with 15 positive, 17 negative and 3 flats
  • 8/27 – Wednesday closed negative with 16 positive, 17 negative and 2 flats
  • 8/26 – Tuesday closed positive with 25 positive, 9 negative and 1 flat
  • 8/25 – Monday closed negative with 4 positive, 29 negative and 2 flats

 

“I hate to be percieved as negative or contrarian but, this is a NO spin zone and facts/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 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” – they were pretty simple instructions to follow!

 

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

The top three (3) performing in the session:   

  • Tuesday: Ionis Pharmaceuticals IONS), Vertex Pharmaceuticals (VRTX) and Arrow Pharmaceuticals (ARWR)
  • Monday was a holiday
  • Friday: IQVIA Holdings (IQV), Lenz Therapeutics (LENZ) and Vericel (VCEL)

The worst three (3) in the session: 

  • Tuesday: IQVIA Holdings (IQV), Vericel (VCEL) and Mesoblast (MESO)
  • Monday was a holiday
  • Friday: Alnylam Pharmaceuticals (ALNY), Mesoblast (MESO) and BioNTech (BNTX)

 

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.