January 9, 2024 4:51pm

News from JPM Healthcare 2024 conference equities weaker locking-in downside

Pre-open Indications: 7 Hits and 2 Miss

If you’re looking for sector intel, ideas and facts or Insight in a volatile climate, RMi provides the extenuating factors of share pricing.

Never leave an investor uninformed! A trusted source of factual reporting!

With no sigh of relief today, I answer or qualify equity’s thrown dice, in which company should investors put or keep in their portfolio for the near and long term

Have you heard of Albert Einstein’s definition of insanity? The one where he says it’s “doing the same thing over and over again and expecting different results?”


Tuesday: The Dow closed DOWN -157.85 points or -0.42%, the S&P closed DOWN -0.15 points or -7.04% while the Nasdaq closed UP +13.94 points or +0.09%



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 S&P 500 and Dow slipped on Tuesday, pressured by a modest rise in Treasury yields as investors question the timing and size of the Fed's interest rate cuts in 2024 ahead of inflation data this week (Thursday and Friday). While the Nasdaq struggled yet, closed positive.

Economic Data Docket: The NFIB Small Business Optimism Index increased to 91.9, up 1.3 points and better than the Dow Jones estimate for 90.8. While the index showed improvement, this marked the 24th month in a row below the long-term average of 98.

  • While still on the pessimistic side, the share of small business owners expecting better business conditions over the next six months improved to a net negative 36%, up 6 points on a monthly basis and 25 percentage points better than a year ago.


Pre-Open: RegMed Investors’ (RMi) pre-open: “JPM Healthcare conference, the name of the game this week, dominating institution’s attention. Reiterating, “Who will have a seat when the presentations begin, which will benefit from the investment gamut after when the music stops or slows? The curse of a dramatic upside… as uncle algo and his electronic trading dwarf’s circle.”  https://www.regmedinvestors.com/articles/13278


Pre-Open Indications: 7 Hits < AxoGen (AXGN -$0.12), Beam Therapeutics (BEAM +$0.10), Caribou Bioscience (CRBU +$0.00), CRISPR Therapeutics (CRSP -$0.06), Ultragenyx Pharmaceuticals (RARE -$1.43), Alnylam Pharmaceuticals (ALNY +$1.37), Verve Therapeutics (VERV +$0.01)> 2 Miss < Sage Therapeutics (SAGE +$1.27), BioLife Solutions (BLFS -$0.01)>


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

  • Tuesday’s advance/decline line was negative at the open with 10 incliners, 23 decliners and 2 flats, ending with a negative close of 14 incliners, 18 decliners and 3 flats
  • Monday’s advance/decline line was negative at the open with 14 incliners, 20 decliners and 1 flat, ending with a positive close of 28 incliners, 6 decliners and 1 flat


Ebb and flow of MY covered sector cell and gene therapy session daily “endings”:

Q1/24: 1 holiday, 3 negative and 3 positive closes


  • 1 holiday, 10 negative and 10 positive closes
  • November – 11 positives, 1 holiday and 10 negative closes
  • October – 12 negative and 10 positive closes


Key Metrics:

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

  • Tuesday, the IBB was down -0.32% and the XBI was up +0.19%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Tuesday was down -0.32 point or -2.45% at 12.76


Tuesday’s Closing Down (10 of 18):

  • Blueprint Medicine (BPMC -$2.33 after Monday’s +$2.34
  • Ultragenyx Pharmaceuticals (RARE -$1.43 after Monday’s +$2.18
  • Intellia Therapeutics (NTLA -$0.78 after Monday’s +$1.44),
  • Harvard Apparatus RN (HRGN -$0.27 after Monday’s $0.00)
  • MiMedx (MDXG -$0.19),
  • Voyager Therapeutics (VYGR -$0.19),
  • Editas Medicine (EDIT -$0.19),
  • uniQure NV (QURE -$0.18),
  • AxoGen (AXGN -$0.12),
  • bluebird bio (BLUE -$0.10),

Flat (3):

  • Adverum Biotechnologies (ADVM)
  • Precigen (PGEN)
  • Caribou Biosciences (CRBU)

Tuesday’s Closing Up (10 of 14):

  • Vericel (VCEL +$2.52 after Monday’s +$2.27)
  • Solid Biosciences (SLDB +$2.10),
  • Alnylam Pharmaceuticals (ALNY +$1.37 after Monday’s +$7.45
  • Sage Therapeutics (SAGE +$1.27 after Monday’s +$3.63
  • Ionis Pharmaceuticals (IONS +$0.56 after Monday’s +$1.62)
  • Fate Therapeutics (FATE +$0.26),
  • Regenxbio (RGNX +$0.23 after Monday’s -$2.01),
  • Compass Therapeutics (CMPX +$0.12),
  • Beam Therapeutics (BEAM +$0.10 after Monday’s +$1.79),


Q1/24 – January

  • Tuesday closed negative with 14 incliners, 18 decliners and 3 flats
  • (1/8) Monday closed positive with 28 incliners, 6 decliners and 1 flat


The BOTTOM LINE: the cell and gene therapy sector showed a touch of weakness on Tuesday.

  • The Russell 2000 backtracked 0.9% with an hour left in the session. Meanwhile, the iShares Russell 1000 Value (IWD) ETF gave back almost all of Monday's gains, losing nearly 0.7%. But iShares Russell 1000 Growth (IWF) edged a bit higher.
  • Back from the good old days of JPM Healthcare conference boosting appeal to cell and gene therapy companies … which came with Tuesday’s volatility and more than a few of sector downfalls!
  • As the Nasdaq gained +0.09% after Monday’s 2%.

Some News from the JPM Healthcare:

  • J&J is acquiring Ambrx for $2 billion. Merck is acquiring Harpoon Therapeutics for $680 million. Interestingly, both are oncology assets, with Ambrx focused on the red-hot ADC space

    AI: Nvidia made a splash to a standing room only crowd announcing it is partnering with Amgen to build Generative AI models to advance data analysis and drug discovery.Cytokinetics (CYTK). The latter soared 12% on Monday on reports that Novartis (NVS) is in talks to buy the developer of small molecule drugs that modulate muscle function in patients with certain conditions.

  • Biogen CEO Christopher Viehbacher said on Monday that getting insurers to pay for Leqembi, the newly approved Alzheimer’s treatment developed in partnership with Eisai, “has not been an issue,”
  • Ultragenyx Pharmaceuticals (RARE) will soon secure $50 million in Series A funding for Amlogenyx, its Alzheimer’s gene therapy spinout, CEO Emil Kakkis told Endpoints News at JPM on Monday. “We have a lead investor signed on, and we have several investors who are on board. . . . We’re trying to get the last two, three people to finish it.”
  • Vertex Pharmaceuticals’ subsidiary ViaCyte Inc. is not pushing through with its diabetes deal with CRISPR Therapeutics, the Swiss biotech announced Monday. The partners—which had recently won the FDA’s approval for Casgevy (exagamglogene autotemcel), the first CRISPR-based gene therapy for sickle cell disease—were supposed to advance a gene-edited stem cell therapy for diabetes. Vertex’s opt-out of the deal will take effect in early February 2024.
  • Athersys filed for Chapter 11 on January 5, according to an SEC filing.  All assets of the regenerative medicine and cell therapy company are being divested to its research partner, Healio, to the tune of $2 million in the form of a credit bid.   The bankruptcy filing was not a surprise. After reporting disappointing results from its MultiStem pivotal trial in October 2023, the company said it was exploring options but, if unable to obtain adequate financing, would have to file for protection under bankruptcy laws to “conduct an orderly wind down of operations.” Athersys ended the third quarter of 2023 with only $1 million in cash, despite cost reduction efforts which included layoffs earlier in the year. Even a $10.4 million raise from investors and licensing partners in November was not enough to stave off Chapter 11.  Healios will now take the reins on Athersys’ MultiStem program, which has been in development since 1994. The off-the-shelf therapy developed from adult stem
  • The Alliance for Regenerative Medicine predicts up to 17 cell and gene therapy approvals this year and defends high prices, while FDA’s Peter Marks expresses concern about manufacturing costs.


Remember, “News, financings, Q4 earnings LPS (loss-per-share) and “runways” will be the under-current or headwind for January through March 2024. Pricing for gene therapies will continue to dominate share ascensions and downfalls!” <me>


Tuesday’s backdrop: released highlights of pipeline updates as JPM Healthcare conference evolves …

Vericel (VCEL): presents Wednesday at 10:30 a.m.


  • Vericel Corporation (VCEL) shares rallied 7% in the last trading session to close at $34.79. This move can be attributable to notable volume with a higher number of shares being traded than in a typical session. This compares to the stock's 4% loss over the past four weeks.
  • VCEL is expected to post quarterly earnings of $0.14 per share in its upcoming report, which represents a year-over-year change of +16.7%. Revenues are expected to be $63.24 million, up 20% from the year-ago quarter. <ZACKS>


The top three (3) performing in the session:

  • Tuesday: Vericel (VCEL), Solid Biosciences (SLDB) and Alnylam Pharmaceuticals (ALNY),
  • Monday: Alnylam Pharmaceuticals (ALNY), Sage Therapeutics (SAGE) and Blueprint Medicine (BPMC)

The worst three (3) in the session:

  • Tuesday: Blueprint Medicine (BPMC), Ultragenyx Pharmaceuticals (RARE) and Intellia Therapeutics (NTLA)
  • Monday: Regenxbio (RGNX), Homology Medicine (FIXX) and Agenus (AGEN)


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 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.