January 13, 2025 8:07am

After the December jobs report came in far above expectations sparking Fed rate hike fear

JP Morgan Healthcare Conference this week <a few presenters’ prelim Q4 and FY24 numbers in The Bottom Line>

Pre-open Indications: 2 Positive and 4 Negative Indications

Never leave an investor uninform

 

 

Remember that overnight and pre-open actions’ futures and markets doesn’t necessarily translate into actual trading in today’s market session.

My interpretation of the morning’s numbers is written to be informative; it’s built on what happened to make “it” happen today!

 

Friday’s night’s … RegMed Investors (RMi) Closing Bell: diminished expectations ignite weekly declines … https://www.regmedinvestors.com/articles/13762

Sage Therapeutics (SAGE) Confirms Receipt of Unsolicited Non-binding Acquisition Proposal from Biogen (BIIB) … https://www.regmedinvestors.com/articles/13763

 

Monday; the pre-open Dow futures are DOWN -0.13% or (-55 points), the S&P futures are DOWN -0.61% or (-35 points) and the Nasdaq futures are DOWN -0.88% or (-185 points)

  • Stock futures slipped Monday,
  • European markets were lower,
  • Asia-Pacific markets traded lower.

 

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

Friday: The Dow closed down -696.75 points or -1.63%, the S&P closed down -91.21 points or -1.54% while the Nasdaq closed DOWN -317.25 points or -1.63%

Friday’s advance/decline line mid-day ended with a negative close at the close of 29 incliner, 2 decliners and 4 flats

The Dow and S&P 500 both ended the week -1.9% lower, while the Nasdaq lost -2.3%, with all 3 indexes notching their 2nd consecutive weekly loss.

Economic Data Docket: Monthly U.S. federal budget

  • Data this week includes the December consumer price index on Wednesday morning. Before that, investors will parse wholesale inflation with December’s producer price index report on Tuesday. Wall Street also await commentary from Kansas City Fed President Jeffrey Schmid and New York Fed President John Williams on Tuesday.

 

Q1/25: January – 1 holiday, 1 market close, 2 negative and 4 positive closes

  • Q4/24: December 1 holiday, 15 negative and 5 positive closes
  • November 10 negative and 9 positive closes
  • October: 8 positive and 15 negative closes

 

Companies in my headlights – It’s your decision; I provide ideas and context

I post about “indication intelligence” looking toward investment conferences … to assist investors with insight into sector vulnerabilities and strengths. A dictionary definition of “indicate” refers to something less than a certainty; an indication could be a signal of being oversold or overbought, a recommendation, or grounds for inferring or a basis for believing.

Negative Indications:

Alnylam Therapeutics (ALNY) closed down -$2.57 after Wednesday’s +$4.60 with a negative -$0.88 or -0.37% pre-market

Ultragenyx Pharmaceuticals (RARE) closed down -$1.58 after Wednesday’s -$0.79, Tuesday’s +$1.78 and last Monday’s +$1.16 with a negative -$2.07 or -4.92% aftermarket

uniQure NV (QURE) closed down -$2.07 after Wednesday’s-$1.72 after Tuesday’s +$0.44 with a negative +$0.02 or -0.14% pre-market

Verve Therapeutics (VERV) closed down -$0.48 after Wednesday’s -$0.41 with a negative -$0.36 or -6.51% pre-market

 

Positive Indications:

Vericel (VCEL) closed down -$0.03 after Wednesday’s +$1.45 after Tuesday’s +$0.47 with a positive +$0.99 or +1.66% pre- market

Ionis Pharmaceuticals (IONS) closed down -$0.64 after Wednesday’s -$0.87 with a positive +$0.43 or +1.29% pre-market

 

The BOTTOM LINE: Watch-out below after an up/down week with a market close towards its end.

January ‘25 recap:

  • 1/10 - Friday’s advance/decline line mid-day ended with a negative close at the close of 29 incliner, 2 decliners and 4 flats
  • 1/9 - Thursday, markets were closed
  • 1/8 - Wednesday’s advance/decline line ended with a negative close at the close of 27 incliner, 27 decliners and 1 flat
  • 1/7 - Tuesday’s advance/decline line ended with a barely positive close at the close of 17 incliner, 16 decliners and 2 flats
  • 1/6 - Monday’s advance/decline line ended with a positive close at the close of 22 incliner, 12 decliners and 1 flat
  • 1/3 - Friday’s advance/decline line ended with a positive close at the close of 30 incliner, 8 decliners and 2 flats
  • 1 /2 - Thursday’s advance/decline line ended with a positive close at the close of 21 incliner, 12 decliners and 2 flats

 

Earnings are a-comin’ … from Q4/24 are due … as downdrafts follow as proven in past sessions!

  • Big Point to remember “Think about it; the biggest problem the cell and gene therapy sector has …  99% of them have no earnings! If rates are higher for longer, that means that sector companies will continue to struggle or be unable to raise capital and therefore be subject to debt servicing firms!” <Me>

 

Sector companies are issuing prelim revenue, guidance, pipeline for JP Morgan Healthcare conference:

Alnylam Pharmaceuticals (ALNY) … Full Year 2024 Preliminary Net Product Revenues of $1,646 M for ONPATTRO®, AMVUTTRA®, GIVLAARI®, and OXLUMO®, Representing 33% annual growth. 2025 Combined Net Product Revenue Guidance of $2,050 M to $2,250 M Positions Company to Achieve Alnylam P5x25 Goal of Non-GAAP Profitability. https://finance.yahoo.com/news/alnylam-announces-preliminary-fourth-quarter-210000604.html

AxoGen (AXGN) … Preliminary Q4/24 revenue is expected to be approximately $49.4 M, which represents a 15.1% increase over the Q4/23 driven by solid performance across the product portfolio. Full-year 2024 revenue is expected to be approximately $187.3 M, which represents a 17.8% increase over the full-year of 2023. Our positive performance reflects continued improvements in the execution of our commercial strategies, including a focus on high-potential accounts in Extremities and OMF-Head & Neck applications, adoption of Axogen’s complete peripheral nerve surgical algorithm across all procedures, and continued adoption of Resensation® as an expectation for post mastectomy breast reconstruction procedures. Expect full year gross margin to be above 75.5%. Total balance of cash, cash equivalents and investments on December 31, 2024, is expected to be approximately $39.5 M, representing an increase of approximately $2.5 M over that balance at the end of 2023.

Ultragenyx Pharmaceuticals (RARE) … Preliminary 2024 total revenue of $555 M to $560 M, exceeding top end of guidance, including Crysvita® revenue of $405 million to $410 M, and Dojolvi® revenue of $87 M to $89 M. 2025 expected total revenue guidance of $640 M to $670 M.

Blueprint Medicine (BPMC) … Updating peak systemic mastocytosis franchise revenue opportunity to $4 B, catalyzed by strong AYVAKIT® (avapritinib) launch and evolving SM prevalence estimates. Expect to achieve $2 B in AYVAKIT revenue by 2030.  BLU-808 demonstrates wide therapeutic window with rapid, robust and sustained tryptase reductions exceeding 80% in P1 healthy volunteer study.

Beam Therapeutics (BEAM) … More Than 40 Adult Sickle Cell Disease Patients Now Enrolled in BEACON Trial of BEAM-101; Beam Expects to Dose 30 Patients and Present Updated Data by Mid-2025. Initial Data from Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency Expected in First Half 2025. Dosing Anticipated to Commence in Phase 1/2 Trial of BEAM-301 in Glycogen Storage Disease Type 1a in Early 2025. IND-enabling Studies of ESCAPE Nongenotoxic Conditioning Approach Underway, with Healthy Volunteer Study of BEAM-103 Antibody Expected to Initiate by Year-end. Cash Runway Expected to Support Operating Plans into 2027, Now Inclusive of Commercial Readiness Activities for BEAM-101

Verve Therapeutics (VERV) … Initial data for the Heart-2 Phase 1b clinical trial of VERVE-102 targeting PCSK9 expected in second quarter of 2025. Dosing in the Heart-2 trial has moved to the 0.6 mg/kg cohort. VERVE-301 nominated as development candidate targeting the LPA gene; Verve to receive an associated milestone payment from Eli Lilly. Well-capitalized with cash runway extending into mid-2027

Moderna (MRNA) … Achieved 2024 product sales of $3.0 to 3.1 billion (unaudited) and an ending cash balance of approximately $9.5 billion (unaudited). Updates 2025 expected revenue range to $1.5 to 2.5 billion. Expects to reduce 2025 cash cost expenses by $1.0 billion with a plan for additional 2026 cost reductions of $0.5 billion. Updates 2025 expected ending cash balance to approximately $6.0 billion. Anticipates milestones across 10 prioritized programs, including up to three potential 2025 approvals and six registrational data readouts

Precigen (PGEN) … Completed BLA submission for PRGN-2012 for treatment of adults with RRP. Commercial readiness activities underway in anticipation of potential launch of PRGN-2012; Company started 2025 with approximately $100 million cash on-hand* with cash runway well into 2026, beyond the anticipated launch in the second half of 2025. According to recently updated internal analysis derived from review of claims data, the market opportunity for PRGN-2012 in RRP is estimated to be approximately 27,000 adult patients in the US. Immense market potential for AdenoVerse platform in other HPV6/11-driven indications, such as genital warts, which has significant unmet need with annual global incidence of more than 4 million and prevalence of more than 25 million, and HPV16/18-driven indications, such as cervical cancer and head and neck cancers –

CRISPR Therapeutics (CRSP) … launch of CASGEVY® continues to gain momentum. 2025 is poised to be a catalyst-rich year with key updates across several programs. Starting 2025 with a strong balance sheet with approximately $1.9 billion in cash, cash equivalents, and marketable securities.

 

What could 2025 have in store for the capital access space as Partners are the gift of life!

  • "You'll see a number of cell and gene therapy comandantes drive for secondaries – 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 the market… capital expansion is second.

I seem to be asking a question followed by a question …

  • Do we need to brace ourself for more economics and their down trending affect?
  • Is this market STILL “bubblicious” my concerns are macroeconomic and valuation

 

Welcome to my world of defining the “grey’ in our universe!

  • The sector is what it is, until it isn’t and even then, it doesn’t seem to be… as NOT much changes as the sector’s share pricing rides the waves of volatility, algorithms, electronic trading and short covering.
  • I am more frequently right than consequentially wrong; if I wanted to be liked, I wouldn’t have been an analyst/journalist.

 

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. All investments are subject to risks. Investors should consider investment objectives.

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.  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.

Henry McCusker, the editor and publisher of RegMed Investors could hold or have positions securities referred to in this publication and he will NOTIFY investors of holdings.