July 31, 2023 4:51pm

News: Ultragenyx Pharmaceutical (RARE +$0.01) has begun dosing the second dose-escalation cohort in its pivotal Phase 1/2/3 Cyprus2+ study following completion of dosing and safety review in the first cohort. RARE's investigational AAV9 gene therapy is designed to deliver stable expression of the ATP7B copper transporter following a single intravenous infusion, with the goal of normalizing copper metabolism in patients with Wilson disease.

Ionis Pharmaceuticals (IONS +$1.64) completed enrollment in the P3 CARDIO-TTRansform cardiovascular outcomes study of eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a large and growing patient population at risk of life-threatening cardiovascular (CV) events. The study enrolled more than 1,400 patients.

Pre-Open Indications: 2 hits and 1 Miss

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If I have learned one thing as a former research analyst in a venture and public fund, operating executive, 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 price movements and questioning pricing targets.

 

The Dow closed UP +100.90 points (+0.28%), the S&P closed UP +6.88 points (+0.15%) while the Nasdaq closed UP +29.37 points (+0.21%)

 

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 …

Indexes closed up Monday as markets braced for a busy earnings week and looked to finish up July on a positive note.

In the month of July, The Dow has jumped 3.2%, with the S&P 500 up 2.9% and the Nasdaq has gained 3.8%.

Economic Data Docket: jobs report. Economists expect the U.S. economy to have added 200,000 jobs in July. Nonfarm payrolls increased 209,000 in June.

 

Monday (7/31) … RegMed Investors’ (RMi) pre-open: “Cash out July in its last session of month. And ring in earnings’ releases LPS (loss-per-share) expectation and consensus.” … https://www.regmedinvestors.com/articles/13055

 

Pre-Open Indications: 2 Hits < Blueprint Medicine (BPMC +$2.88), Alnylam Pharmaceuticals (ALNY -$3.89)> and 1 Miss < Sage Therapeutics (SAGE -$0.28)>

 

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

  • Monday’s advance/decline line opened positive at 28 up/ 6 down and 1 flat, stayed positive with 19 up/ 14 down and 2 flats at the mid-day, ending with a positive close of 20 incliners, 15 decliners and 2 flats
  • Friday’s advance/decline line opened positive at 30 up/ 3 down and 2 flats, stayed positive with 31 up/ 4 down and 0 flat at the mid-day, ending with a positive close of 31 incliners, 4 decliners and 0 flat

 

Key Metrics:

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

  • Monday, the IBB was down -0.55% and the XBI was up +0.62%
  • Friday, the IBB was up +1.52% and the XBI was up +2.45%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Monday was up +0.31 point or +2.33% at 13.64
  • Friday was down -1.08 point or -7.49% at 13.33

 

Closing Down (13 of 13):

  • Vericel (VCEL -$1.86),
  • Alnylam Pharmaceuticals (ALNY -$3.89 after Friday’s +$4.42)
  • Beam Therapeutics (BEAM -$0.51 after Friday’s +$1.57
  • Sage Therapeutics (SAGE -$0.28 after Friday’s -$0.84),
  • Cellectis SA (CLLS -$0.08 after Friday’s -$0.05),
  • Fate Therapeutics (FATE -$0.08),
  • Generation Bio (GBIO -$0.07),
  • Solid Biosciences (SLDB -$0.07),
  • Bellicum Pharmaceuticals (BLCM -$0.03),
  • Intellia Therapeutics (NTLA -$0.03 after Friday’s +$1.39
  • bluebird bio (BLUE -$0.02),
  • Agenus (AGEN -$0.01),
  • Sangamo Therapeutics (SGMO -$0.005)

Flat (2):

  • Harvard Apparatus RM (HNGN) after Friday’s -$0.09
  • Mesoblast (MESO)

Closing Up (10 of 20):

  • Blueprint Medicine (BPMC +$2.88 after Friday’s +$3.38),
  • Ionis Pharmaceuticals (IONS +$1.64 after Friday’s +$1.29
  • Verve Therapeutics (VERV +$0.59),
  • uniQure NV (QURE +$0.51),
  • CRISPR Therapeutics (CRSP +$0.44 after Friday’s +$1.45),
  • Voyager Therapeutics (VYGR +$0.24),
  • Prime Medicine (PRME +$0.23 after Friday’s +$0.55
  • Compass Therapeutics (CMPX +$0.20),
  • Regenxbio (RGNX +$0.19 after Friday’s +$0.80),
  • BioLife Solutions (BLFS +$0.14),

 

Q3/23 – July

Monday (7/31) closed positive with 20 incliners, 13 decliners and 2 flats

Friday closed positive with 31 incliners, 4 decliners and 0 flat

Thursday closed negative with 4 incliners, 30 decliners and 1 flat

Wednesday closed positive with 20 incliners, 14 decliners and 1 flat

Tuesday closed negative with 11 incliners, 23 decliners and 1 flat

Monday (7/24) closed negative with 4 incliners, 31 decliners and 0 flat

Friday closed positive with 19 incliners, 14 decliners and 2 flats

Thursday closed negative with 9 incliners, 26 decliners and 0 flat

Wednesday closed positive with 19 incliners, 15 decliners and 1 flat

Tuesday closed positive with 19 incliners, 16 decliners and 0 flats

Monday (7/17) closed positive with 18 incliners, 17 decliners and 0 flats

Friday closed negative with 8 incliners, 24 decliners and 3 flats

Thursday closed negative 16 incliners, 17 decliners and 2 flats

Wednesday closed positive with 22 incliners, 10 decliners and 3 flats

Tuesday closed positive with 18 incliners, 14 decliners and 3 flats

Monday (7/10) closed positive with 25 incliners, 7 decliners and 3 flats

Friday closed positive with 24 incliners, 11 decliners and 0 flat

Thursday closed negative with 11 incliners, 24 decliners and 0 flat

Wednesday closed negative with 13 incliners, 22 decliners and 0 flat

Tuesday (7/4) was a holiday

Monday (7/3) closed positive with 20 incliners, 13 decliners and 2 flats

 

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.”

 

RegMed Investors (RMi) Research Note: Q2/2023 earnings reporting date announcements … https://www.regmedinvestors.com/articles/13044

 

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!”

 

The top three (3) performing in the session:

Monday: Blueprint Medicine (BPMC), Ionis Pharmaceuticals (IONS) and Verve Therapeutics (VERV)

Friday: Alnylam Pharmaceuticals (ALNY), Blueprint Medicine (BPMC) and Ultragenyx (RARE)

 

While The worst three (3) in the session:

Monday: Alnylam Pharmaceuticals (ALNY), Vericel (VCEL) and Beam Therapeutics (BEAM)

Friday: Sage Therapeutics (SAGE), Harvard Apparatus (HRGN - formerly Biostage (BSTG) and Cellectis SA (CLLS)

 

Interesting Stats:

Q2 '23 M&As announced: 17

Q2 '23 M&As completed: 7

Q3 '23 M&As announced: 3 <so far>

Recently: Korro Bio (private) and Frequency Therapeutics (FREQ) announce merger; combined genetic medicine company will have $170 M in cash runway to last until 2026

 

Good news:

Reata Pharmaceuticals (RETA) acquired by Biogen (BIIB) in $7 billion deal. The maker of an approved therapy for a rare neurological condition, in a deal worth $7.3 billion. The transaction price of $172.50 per share is a nearly 60% premium from Reata’s share price at Thursday’s close. The deal is expected to close by the end of the year.

 

Some BAD news:

Biogen (BIIB) to cut 1,000 jobs as its business is in transition as roll-out of new Alzheimer drug, it developed and prepares for the possible approval of a treatment for depression next week.

Passage Bio (PASG) executes job cuts of 26% at Philadelphia gene therapy developer include the CFO and chief technical officer.

Humanigen (HGEN) is considering bankruptcy or other insolvency proceedings.

 

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!

·         Question: is HRGN just another Ponzi scheme (with their pump/promote buy and sell agenda) as shares are bought to replace those sold?

·         Accumulated deficit:  $85,901 M

·         Will HRGN ESCAPE the scandals when its “former” corporate name was used by a former chairman and CEO Green initiating scandal plagued utilization of an Italian surgeon and the $5 M it took to settle out the executive responsibility as it was SHIFTED to the company shareholders and Harvard Bio (HBIO)???

 

My question that resounds, that should be asked is how many companies are at the end of sentiments …  leash?

 

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.