December 1, 2021 5:16pm

Few happy endings or should I write outcomes today

News: Ultragenyx Pharmaceutical (RARE +$0.93) announced that the first patient has been dosed in its P1/2 study of UX053, an investigational messenger RNA (mRNA) therapy in development for the treatment of Glycogen Storage Disease Type III (GSDIII).

The Biostage Chronicles: the “pump” is back jump +$0.95 with 2,590 shares traded; one would think that the new “interim” CEO would put a hold on such activities. So, what is the value proposition and valuation criteria?

Pre-open indication results: 1 HIT, 7 MISS

Who else is tracking a broad representative of cell and gene therapy equities – with facts supported by real numbers?

If I have learned one thing as a former research analyst, venture and public markets investor to a journalist; it is that your life and your bank account are largely tied to your questioning of price targets.

The Dow closed DOWN -461.68 points (-1.34%); the S&P closed DOWN -53.96 points (-1.18%) while the Nasdaq closed DOWN -283.64 points (-1.83%)

 

Henry’omics:

Indexes closed lower on Wednesday after a morning rally faded as investors fretted about the latest coronavirus variant and the first evidence of its U.S. arrival while they also digested Fed Chair Powell's comments on surging inflation.

The Dow gave up a 500-point gain then returned to a positive +54.65 points, then again dropping -$179.04 points at 3:20 p.m. while the Nasdaq dropped $0.12 around 2:20 p.m. and turned further negative more -204.38 points and then closed even lower (see above) after the Centers for Disease Control and Prevention confirmed the first case of omicron in the U.S.- San Francisco, Ca.

The CBOE volatility index, the VIX fear gauge, was higher at 31.12 following Tuesday’s 27.19 after Monday’s 22.96.

 

Ultragenyx Pharmaceutical (RARE) News (continued): The two-part P1/2 clinical trial will evaluate the safety, tolerability and efficacy of UX053 in adults age 18 and older with GSDIII. Part 1 is open label and will enroll up to 10 patients who will receive a single ascending dose of UX053 administered via intravenous infusion. Part 2 is double-blind and will evaluate five repeat doses at escalating dose levels in up to 16 patients across four cohorts randomized 3:1 to UX053 or placebo.

  • The primary endpoints are treatment-emergent adverse events (TEAEs), serious TEAEs and related TEAEs in both parts of the study.
  • Secondary endpoints include pharmacokinetic parameters. Exploratory endpoints include clinician- and patient-reported outcomes, muscle strength, blood sugar, and biomarkers of liver, cardiac and muscle health.

 

Economic Data Docket: ADP’s private payroll data for November showed 534,000 jobs added that month, above expectations of 506,000. November’s IHS Markit manufacturing PMI came in at 58.3, lower than expected. October construction spending also rose slower than expected, but there was a positive historical revision to help offset the miss. The November manufacturing report from ISM, which matched expectations and showed a decline in prices paid.

 

RegMed/Stem/Cell and Gene therapy’s 35 covered equities’ Advance/Decline (A/D) lines: what’s happening …

  • Wednesday opened positive at 27 ups/6 downs, 1 flat and 1 acquired, flipped negative at the mid-day at 11/22, 2 flats and 1 acquired, ending with a negative close of 4 ups/29 downs, 1 flat and 1 acquired;
  • Tuesday opened positive at 18 ups/15 downs, 1 flat and 1 acquired, flipped negative at the mid-day at 12/22 and 1 acquired, ending with a positive close of 19 ups/15 downs and 1 acquired;
  • Monday opened negative at 12 ups/20 downs, 2 flat and 1 acquired, stayed negative at the mid-day at 6/27, 1 flat and 1 acquired, ending with a negative close of 4/31 and 1 acquired;

 

RegMed Investors’ (RMi) pre-open: “what went down, does come back up. Hopeful better outcomes and share pricing effects in December?” …  https://www.regmedinvestors.com/articles/12205

 

Pre-open indication results: 1 HIT: < Cellectis SA (CLLS +$0.18)> and 7 MISS < Biostage (BSTG +$0.95), Ionis Pharmaceuticals (IONS -$0.89), CRISPR Therapeutics (CRSP -$4.62), Global Blood Therapeutics (GB -$1.13), Editas Medicine (EDIT -$2.25), Sage Therapeutics (SAGE -$1.76), Sangamo Therapeutics (SGMO -$0.28)>

 

The Biostage (BSTG) Chronicles: Wednesday closed up +$0.95 with 2,590 shares traded after Tuesday closed down again -$0.02 with 2,143 shares traded after Monday closed down -$0.40 to $2.00 with 6,025 shares traded.

  • New CEO and chairman with a personal investment.
  • My view, “the grifters are still running the company”
  • NO clinical trial from a one (1) and 3/4-year-old IND and a negligence rejection of reimbursement in a terminal death suit.
  • What’s there to invest in, the “Sword of Damocles” terminal death suit insurance claim rejection hanging over the companies non-going operations?

 

Key Metric - volume

Sector volume LOW with 1 of the 4-upside having higher than the 3-month average volume with INCREASED volume of 13 of 29-downside having higher than the 3-month average volume;

 

The 52-week losers:

  • CRISPR Therapeutics (CRSP) reached a 52-week low of $75.88
  • Homology Medicine (FIXX) reached a 52-week low of $4.94
  • Ionis Pharmaceuticals (IONS) reached a 52-week low of $26.13

 

Jumping with share pricing momentum (4 of 4):

  • Biostage (BSTG +$0.95 - "pumped"),
  • Ultragenyx (RARE +$0.93 after Tuesday’s +$1.52 and Monday’s -$2.78),
  • Cellectis SA (CLLS +$0.18),
  • Adverum Biotechnologies (ADVM +$0.01 after Tuesday’s -$0.13),

Hammered in today’s market (10 of 29):

  • Intellia Therapeutics (NTLA -$8.09 after Tuesday’s +$3.28 and Monday’s -$1.40),
  • CRISPR Therapeutics (CRSP -$4.52 after Tuesday’s +$1.92)
  • Alnylam Pharmaceuticals (ALNY -$3.87 after Tuesday’s +$3.06 and Monday’s -$7.79),
  • Fate Therapeutics (FATE -$2.88 after Tuesday’s+$3.24),
  • Editas Medicine (EDIT -$2.25 after Tuesday’s +$1.40 and Monday’s -$1.11),
  • Vericel (VCEL -$2.01 after Tuesday’s -$0.10 and Monday’s -$1.00),
  • Sage Therapeutics (SAGE -$1.76 after Tuesday’s +$0.38 and Monday’s -$0.79)
  • Regenxbio (RGNX -$1.70 after Tuesday's +$1.81 and Monday’s -$0.77),
  • uniQure NV (QURE -$1.43 after Tuesday’s -$0.67),
  • Global blood Therapeutics (GBT -$1.13),

Closing Flat:

  • 1 – ReNeuron (RENE.L) and 1 - Stemline Therapeutics (STML) – acquired

 

Stats:

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

  • Wednesday, the IBB closed down -2.26% and XBI closed down -3.56%
  • Tuesday, the IBB closed down -1.18% and XBI closed up +1.06%
  • Monday, the IBB closed up +0.75% and XBI closed down -1.48%

The CBOE Volatility Index (VVIX: INDEX) tracked:

  • Wednesday was up +3.93 points or +14.45% at 33.12
  • Tuesday was up +4.23 points or +18.42% at 27.19
  • Monday was down -5.66 points or -19.78% at 22.96

 

December, the second month of Q4/21:

Wednesday (12/1) closed negative with 4 incliners, 29 decliners, 1 flat and 1 acquired

 

The BOTTOM LINE:  Another dive into the pool of sector negativity.

However, it could be a wee-bit of that we are about to enter into TAX SELLING for year’s end!

In any case, I wouldn’t ramp up your exposure at the current levels.

There's also nothing wrong with standing pat after reducing exposure earlier in the month.

Reiterating, “the stem, cell and gene therapy sector is still seen as vulnerable to extreme low volume moves in either direction, as share pricing risk stimulates their susceptibility!”

I’m STILL a “beware or the cautious man” whose focus has always been “warning analysis” … earnings’ season is NOT over … my advice, trim and skim any new highs if one can!”

WHY do I keep analyzing Biostage (BSTG): When one sees an inextricable wrong and morally repulsive scenario; it takes courage, resolve and patience to stay the course of asking the questions without response!

Who is defining the metrics for investors and keeping you notified of the sector and market fluctuations?  What I provide is a trusted source of share pricing intelligence – it’s more than opinion, I deal in the facts and numbers that back them up.

 

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.