September 5, 2023 4:53pm

Coming off a mild winning week (3 of 5 sessions), investors have to contend with September’s seasonal effects, which historically marks the weakest month for equities

Pre-open Indications: 4 Hits and 0 Miss

News: uniQure N.V. (QURE -$0.86) the FDA) has cleared the IND application for AMT-260, the gene therapy candidate for refractory mesial temporal lobe epilepsy (MTLE). AxoGen (AXGN -$0.15) launched Axoguard HA+ Nerve Protector. Axoguard HA+ has the potential to greatly impact patients who suffer from compression nerve injuries, particularly where the injury is in proximity to high mobility joints.”

Subscription is coming, it is not conscription but, an offer to join our collective of like-minded investors!  Join me … in the NO spin zone.

Never leave an investor uninformed! 

If I have learned one thing as a former research analyst in a closed end - venture and public fund, multiple operating roles, x-military officer and x-FBI, an adjunct (MBA) business school professor now a journalist; my life and your portfolio are largely tied to our knowledge of the biotech universe’s patterns, price movements and questioning pricing targets.


The Dow closed DOWN -195.74 or -0.56%, the S&P closed DOWN – 18.94 or -0.42% while the Nasdaq closed DOWN -10.86 points or -0.08%



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 Dow, S&P and Nasdaq kicked-off the first trading session of a holiday shortened week in a dive into the abyss weighed down by a jump in crude oil prices after Saudi Arabia extended its 1-million-barrels per day voluntary oil production cuts.

Also, treasury yields also popped, straining risk assets. The yield on the 10-year Treasury surged more than 8 basis points to 4.258%.

From the title, AMT-260 comprises an AAV9 vector that locally delivers two engineered miRNAs designed to degrade the GRIK2 gene and suppress the aberrant expression of glutamate receptor subtype GLUK2 that is believed to trigger seizures in patients with refractory MTLE.

Economic Data Docket: Factory orders decreased in July, though not by as much as expected, the Commerce Department reported. New orders for manufactured goods declined 2.1% on the month, which was better than the Dow Jones estimate for a drop of 2.3%. A 5.2% slide in durable goods demand was the big driver behind the decrease as orders for transportation equipment tumbled 14.3%. Orders rose 2.3% in June and had been up for four straight months.

  • Oil prices rose after Saudi Arabia extended its 1-million-barrels per day voluntary oil production cuts. West Texas Intermediate futures popped 2% to trade above $87 per barrel, reaching their highest levels since November.


Friday (9/5) … RegMed Investors’ (RMi) Opening Bell: “a challenging September ahead? Historically the weakest month for equities, and investors will be sifting through economic reports including fresh inflation data ahead of the Fed’s September policy meeting.” …


Pre-Open indications: 4 Hits < Alnylam Pharmaceuticals (ALNY -$0.92), Vericel (VCEL -$1.06), BioLife Solutions (BLFS +$0.12), Regenxbio (RGNX -$0.66)> and 0 Miss


52-week low:

uniQure NV (QURE) -$0.86 at $7.86 -- were down more than 11% as of 1:06 p.m. on Tuesday after announcement that the FDA had cleared its IND application for AMT-260, a treatment for refractory mesial temporal lobe epilepsy (MTLE). The disease is a chronic neurologic disorder, considered the most common form of focal epilepsy (which affects one side of the brain). At one point on Tuesday morning, the stock fell to a 52-week low of $7.40.

·         1st, investors who were waiting for an opportunity to sell a stock that was down more than 65% this year sold on the news. A P1/2 clinical trial for AMT-260 is expected to begin in the fourth quarter.

·         2nd, QURE will be delisted from the S&P 600 index as of Sept. 18, following an announcement by the index on Sept. 1. The S&P 600 is used by 18 different exchange-traded funds (ETFs) in the United States. uniQure initially was added to the S&P 600 list in 2021, and falling off the list means the stock is less likely to be traded, weakening its position.

·         Although, QURE had $628.6 million in cash; reporting Q2 revenue of $2.4 million, up 380% year over year, and a Q2 loss of $46 million, compared to a loss of $46.2 million in Q2/22. <me and Motley Fool>


RegMed/Stem/Cell and Gene Therapy’s 35 covered equities’ Advance/Decline (A/D) lines: what happened – differences …

  • Tuesday’s advance/decline line opened negative at 15 up/ 17 down and 3 flats, stayed negative with 6 up/ 28 down and 1 flat at the mid-day, ending with a negative close of 8 incliners, 25 decliners and 2 flats
  • Monday was a holiday
  • Friday’s advance/decline line opened positive at 28 up/ 2 down and 5 flats, stayed positive with 27 up/ 8 down and 0 flat at the mid-day, ending with a positive close of 28 incliners, 6 decliners and 1 flat


Ebb and flow:

Q3: September – 1 positive and 1 negative closes

·         August – 9 positive and 14 negative closes


Key Metrics:

The iShares NASDAQ Biotechnology (IBB) and the SPDR S&P ETF (XBI) indicators:

  • Tuesday, the IBB was down -1.44% and the XBI was down -1.82%
  • Monday was holiday
  • Friday, the IBB was up +1.01% and the XBI was up +2.42%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Tuesday was up +0.19. point or +1.37% at 14.01
  • Monday was holiday
  • Friday was down -0.50 point or -3.68% at 13.07


Closing Down (10 of 25):

  • Vericel (VCEL -$1.32 after Friday’s +$1.05),
  • Ionis Pharmaceuticals (IONS -$1.16),
  • Blueprint Medicine (BPMC -$0.95 after Friday’s +$0.78),
  • uniQure NV (QURE -$0.86),
  • Voyager Therapeutics (VYGR -$0.84),
  • Alnylam Pharmaceuticals (ALNY -$0.83 after Friday’s +$3.35),
  • Prime Medicine (PRMC -$0.78 after Friday’s -$0.46),
  • Regenxbio (RGNX -$0.69 after Friday’s +$0.43),
  • Verve Therapeutics (VERV -$0.61 after Friday’s +$0.58),
  • CRISPR Therapeutics (CRSP -$0.27 after Friday’s +$0.52),

Flat (2);

  • Bellicum Pharmaceuticals (BLCM)
  • Homology Medicine (FIXX)

Closing Up (8 of 8):

  • Caribou Biosciences (CRBU +$0.34 after Friday’s -$0.01),
  • Intellia Therapeutics (NTLA +$0.28),
  • Sage Therapeutics (SAGE +$0.28 after Friday’s +$0.29),
  • Ultragenyx Pharmaceuticals (RARE +$0.25 after Friday’s +$0.99),
  • Beam Therapeutics (BEAM +$0.22 after Friday’s +$0.28),
  • BioLife Solutions (BLFS +$0.15 after Friday’s +$0.19),
  • Harvard Apparatus RN (+$0.01),
  • Mesoblast MESO +$0.01 after Friday’s -$0.12),


Q3/23 – September

  • (9/5) Tuesday closed negative with 8 incliners, 25 decliners and 2 flats
  • Monday was holiday
  • (9/1) Friday closed positive with 28 incliners, 6 decliners and 1 flat


The BOTTOM LINE: I try to keep it simple … and short!

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

Tuesday, investors returned from a long weekend grappled with oil prices' highest levels in over a year and fresh data showing China's economy is still struggling to recover.


The cell and gene therapy sector equity closed negative on Tuesday post Friday’s positive close; the previous Monday was a holiday with the come-back a glaring downslide - leading with light trading volumes.


Fed Governor Christopher Waller said on Tuesday the latest round of economic data was giving the U.S. central bank space to see if it needs to raise interest rates again, while noting that he currently sees nothing that would force a move toward boosting the cost of short-term borrowing again. <Reuters>

The remainder of the week looks slow on the economic data front, and investors will continue to digest U.S. jobs data from Friday which gave signs to suggest the labor market may be cooling. That’s what the Federal Reserve wants to see as it works to battle historically high inflation through interest-rate hikes. <Barrons>

“With a relatively light economic calendar this week, traders will be watching to see how the market behaves in early September—which has been, historically, the weakest month for the stock market,” Chris Larkin, managing director of trading and investing at E-Trade from Morgan Stanley, wrote Tuesday. “Since 1957, September has been a down month for the S&P 37 times and an up month only 29 times.”

WHY these quotes, it’s about retail investors and … financing the sector – it ain’t good, actually downright bad!


Right up front, “I also 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!”


Another company bites the dust with more bad news, Infinity Pharmaceuticals (INFI -$0.0104 to $0.097) cleared-out its executive suite in the wake of a failed merger agreement that had already cost 21 jobs.

·         Previously (7/25/23) INFI announced a series of actions designed to reduce its burn rate and enhance our ability to maximize value of eganelisib, following the termination of its previously announced merger agreement with MEI Pharma, Inc.


Being “greedy when others are fearful” is easier said than done

When a cell and gene therapy sector equity sells-off to or below a buy point, investors face a tough decision: hold tight, exit or trim the position. There's not necessarily a "right" answer. Sometimes the stock will bounce right back, others will keep falling — perhaps after briefly bouncing. A more-cautious approach may make more sense in the current volatile market.


The top three (3) performing in the session:

Tuesday: Intellia Therapeutics (NTLA), Caribou Biosciences (CRBU) and Sage Therapeutics (SAGE)

Monday was a holiday

Friday: Alnylam Pharmaceuticals (ALNY) – 6x, Vericel (VCEL) and Ultragenyx Pharmaceuticals (RARE)


While The worst three (3) in the session:

Tuesday: Ionis Pharmaceuticals (IONS), Blueprint Medicine (BPMC) and Vericel (VCEL)

Monday was a holiday

Friday: Prime Medicine (PRMC), Mesoblast (MESO) and Brainstorm Cell Therapeutics (BCLI)


I am definitely considered a contrarian depending on the session – one has to be a watcher of the ups and downs as well as measurer of sentiment. I am also a NO trust investor but, a validate and verify journalist/analyst.

The NO spin zone! U.S. public shareholders deserve accountability …


Harvard Apparatus Regenerative Technology (HRGN) formerly Biostage (OTCQB: BSTG) … as of 7/20/23 -- so many issues according to the 8-K and 10-K!

•              Accumulated deficit:  $88.5 M (newest 10-K)

Going Concern

·         HRGN has incurred substantial operating losses since its inception, and as of June 30, 2023 had an accumulated deficit of approximately $88.5 million and will require additional financing to fund future operations.

·         HRGN will NEED to raise additional funds to fund its operations. WHO and WHICH U.S. investors would INVEST in a BASICALLY Chinese company with their history??

·         Are they back pumping the price +$0.01 and volume – as 5,757 shares traded – reinforcing MY view as a Ponzi scheme!! <9/5/23>


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 its 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.