June 12, 2020 8:03am
Rocks you to the ground and It’s going to hurt yet; one still has to get up
Pre-open indications: 7 BUYs
News: CRISPR Therapeutics (CRSP) and Vertex (VRTX) declared new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 P1/2 trials in transfusion-dependent beta thalassemia (TDT). Editas Medicine (EDIT) announced results from a pre-clinical, proof-of-concept study of EDIT-301. bluebird bio (BLUE) new data from ongoing P3 studies of betibeglogene autotemcel (beti-cel; formerly LentiGlobin™ for β-thalassemia gene therapy) show pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent β-thalassemia (TDT) achieve and maintain transfusion independence with hemoglobin (Hb) levels that are near-normal.
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Dow future are UP +1.89% (+474 points), S&P futures are UP +1.67% (+52 points) and NASDAQ futures are +1.54% (+148 points)
U.S. stock futures were higher Friday morning after growing worries of a resurgence in coronavirus cases sent equity prices plunging;
European markets had a choppy start as they looked to rebound from the previous session’s significant sell-off as the pan-European Stoxx 600 bounced either side of the flatline in early trade and was down -0.6% into the session;
Asia Pacific slipped with South Korea, Japan, Australia Hong Kong and the Mainland China dropping as the MSCI Asia ex-Japan index traded 1.51% lower.
Data docket: Consumer sentiment data along with the latest U.S. import and export numbers are scheduled for release
We’re back … not quite Á Dinosaur’s Story” but … mixed negative reviews, with some praise as it polarized short length running time and an interesting cast for characters (like some equities).
U.S. stocks on Thursday booked their worst daily plunge since fears about the economic impact of measures to curtail the spread of the COVID-19 pandemic took root in investors’ psyches back in March
Cue: say less, mean more!
News: CRISPR Therapeutics (CRSP) and Vertex (VRTX) follow-up
These data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress by Dr. Selim Corbacioglu, Professor of Pediatrics and the Chair of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Regensburg University Hospital, Regensburg, Germany.
CLIMB-111 Trial in Transfusion-Dependent Beta Thalassemia Updated Results
Data presented today at EHA demonstrate clinical proof-of-concept for CTX001 in TDT. Data include longer-duration follow-up data for the first patient with TDT treated with CTX001 and new data for the second TDT patient treated. CRSP and VRTX had announced initial data for the first TDT patient in November of 2019.
- Patient 1 with TDT has the β0/IVS-I-110 genotype, which is associated with a severe phenotype similar to β0/β0, and had a transfusion requirement of 34 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. After CTX001 infusion, two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia, and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. New data presented today show that at 15 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 14.2 g/dL, fetal hemoglobin of 13.5 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 100.0%. Bone marrow allelic editing was 78.1% at 6 months and 76.1% at one year.
- Patient 2 with TDT has the β0/IVS-II-745 genotype and had a transfusion requirement of 61 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. The patient achieved neutrophil engraftment 36 days after CTX001 infusion and platelet engraftment 34 days after infusion. After CTX001 infusion, two SAEs occurred, neither of which the PI considered related to CTX001: pneumonia and an upper respiratory tract infection; both subsequently resolved. At 5 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 12.5 g/dL, fetal hemoglobin of 12.2 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.4%.
News: Editas Medicine (EDIT)
- Announced results from a pre-clinical, proof-of-concept study of EDIT-301. EDIT-301 is being developed as a potentially best-in-class, durable medicine to treat sickle cell disease. EDIT-301 contains CD34+ hematopoietic stem cells from sickle patients that are edited at the HBG1/2 promoter in the beta-globin locus using Cas12a (also known as Cpf1) to induce fetal hemoglobin (HbF) where HbF-inducing mutations occur naturally.
- EDIT reported the data at the 25th Congress of the European Hematology Association being held virtually.
News: bluebird bio (BLUE)
- Announced that new data from ongoing P3 studies of betibeglogene autotemcel (beti-cel; formerly LentiGlobin™ for β-thalassemia gene therapy) show pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent β-thalassemia (TDT) achieve and maintain transfusion independence with hemoglobin (Hb) levels that are near-normal (≥10.5 g/dL).
- These data are being presented at the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.
Thursday post’s title: “wipe-out; share pricing was capsized by a wave of market data brought on by increasing COVID-19 cases and future economic uncertainties.’
- The NASDAQ closed DOWN -567.62 points (-5.27%);
- The IBB closed down -5.93% and XBI also closed down -5.53%
- Thursday opened negative and closed negative;
- Sector volume continued LOW with 1 out of the 2-upside having higher than the 3-month average volume and 10 out of the 31-downside having higher than the 3-month average volume;
- Thursday’s percentage (%) of the 2-upside were +1.42% (MDXG) to +1.90% (SGMO) while the 31-downside ranged from -1.43% (ATHX) to -15.75% (PGEN);
- June registered 3 positive and 6 negative closes
- May registered 9 negative, 11 positive closes and 1 holiday.
- April registered 10 negative, 11 positive closes and 1 holiday.
- March registered 11 negative, 10 positive closes and 1 neutral close.
- February registered 9 negative, 9 positive closes, 3 vacation days and 1 holiday.
- January registered 9 negative, 10 positive closes and 2 holidays.
Companies in my headlights – It’s your decision; I provide an idea and context
Adverum Biotechnologies (ADVM) closed down -$2.55 and has a positive +$0.68 or +3.19% aftermarket indication
Fate Therapeutics (FATE) closed down -$3.14 to $29.85 after Wednesday’s +$0.27, Tuesday’s +$4.41 to $32.72 and Monday’s +$0.10 to $28.31 and has a positive +$0.15 or +0.50% aftermarket indication. Post a closed offering with Johnson & Johnson Innovation-JJDC, Inc. participating in a private placement up to $50 million, which was priced at $28.31. A lot of new shares and many in safe hands.
CRISPR Therapeutics (CRSP) closed down -$3.33 after Wednesday’s -$0.23, Tuesday’s +$2.73 and has a +$1.64 or +3.325% aftermarket indication <review news flow above>;
Editas Medicine (EDIT) closed down -$2.48 and has a positive +$0.99 or +3.36% aftermarket indication <see news above>;
bluebird bio (BLUE) closed down -$5.20 after Wednesday’s +$1.55, Tuesday’s -$2.37 and Monday’s -$1.84) and has news that should/could move the share’s pricing (see above>
Sage Therapeutics (SAGE) closed down -$1.35 to after Wednesday' -$0.42 and has a positive +$0.15 or +0.50% aftermarket indication. SAGE cut operating costs, rejuggled its priorities, also trying to invigorate Sage-324 is about to enter P2 clinical trials for targeting essential tremor (a motor movement disorder), estimated to affect 6 million people in the U.S. In open-label (where patients' treatment bias is not eliminated) phase 3 studies, the drug demonstrated unverified promise of efficacy when given to patients, but has not yet had its efficacy and safety proven in placebo-controlled clinical trials. Another candidate, Sage-718, is also entering phase 2 clinical trials for targeting various cognitive disorders. The drug has demonstrated positive preliminary efficacy and safety profiles in P1 studies.
Pluristem Therapeutics (PSTI) closed DOWN -$0.41 after Wednesday’s +$0.49 to $7.54 and is OVERSOLD after news of activating clinical sites and initiation of enrollment in its P2 U.S. FDA study of PLX cells for the treatment of severe COVID-19 complicated by Acute Respiratory Distress Syndrome (ARDS). PSTI is focused on expanding to 25 clinical sites throughout the U.S.
The BOTTOM LINE: Short-term market contrarians, such as myself, were not particularly surprised by Thursday’s decline.
I keep saying streaking highs scare me!
A -5% tumble, fears of an emerging second wave of the COVID-19 virus and a sobering outlook from Fed set the consequence of/for a fall. It was the 28th time since 1952 that the S&P 500 has tumbled by at least 5% in a day and five (5) of the declines have been in the past three months alone.
The Fed cautioned of a -6.5% fall in U.S. GDP (gross domestic product) in 2020, denting hopes of an immediate “V-shaped” economic recovery in the aftermath of the coronavirus crisis, while several U.S. states reported a spike in coronavirus cases after reopening their economies. <CNBC>
And declines of this magnitude have historically been followed by sizable rebounds (in the days, weeks and months) – short steps or/and leap steps today!
This just could be an OPPURTUNITY to play “pick-up-some-stocks”!
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.