Bluebird Bio Inc (BLUE)

[Moose412]

This is the best I can find:

"The FDA issued a rare disease priory review voucher (PRV) accompanying the approval for Casgevy but not for Lyfgenia. Companies can use these vouchers to expedite FDA’s review of other drugs.

Analysts at Cantor suspected that because bluebird already got a PRV on Zynteglo, which is a twin therapy to Lyfgenia, the FDA may have viewed the request for a new PRV as double-dipping."

https://www.fiercepharma.com/pharma/fda-approves-bluebird-sickle-cell-disease-gene-therapy-can-lyfgenia-overcome-crisprs-halo

[novicetrader]

Do we know if the PRV been actually declined for BLUE? Or is it simply being assumed that it's not happening since there's been no confirmation on it?

[Moose412]

I would be thrilled w a $10/sh buyout. I believe BLUE is worth far more, but nonetheless.

[stocksrising]

There will likely be several Class Action ambulance chasers, but Blue has made it clear on/in several PR’s that it’s NOT 💯 until FDA says so!


“Closing of the transaction remains subject to the approval of lovo-cel and receipt of a PRV from the FDA, as well as customary closing conditions”.

Sucks for US because of near term dilution about to hit!!

Wouldn’t be surprised if major Pharma Co. Buys them out ??? $10ish X’s 110mm shrs out = $1b+ EV

3 approved drugs generating $250-500mm in revs over next couple years discounted back plus other potential indications with their IP technology??
Total guess, but nothing surprises anymore! :)

[Moose412]

I expect to see another class-action investigation by Pomerantz, but it won't go anywhere. It's not deserved.

But given the profile of this race, it's absurd that the FDA would do this.

No reason trading shouldn't be at $5, with all three drugs. The lack of PRV is mind-boggling, given the indication of 12 yrs+. Monday is going to be interesting.

[stocksrising]

I’m getting so many Alerts concerning tomorrow’s Oral presentation of Updated Long Term Data(ASH Conference ) that could be band aid for now!
ZYNTEGLO….

Also, you prob already saw BLUE’s Safe Harbor lingo ‘Expected, on or Before’ and MS saying somewhat similar:

Bluebird Bio, Inc.

BLUE
-39.5%
+ Free Alerts
shares are trading higher Friday after Morgan Stanley upgraded Bluebird Bio from Underweight to Equal-Weight and raised the price target to $7.

The Details:

Morgan Stanley analyst Jeffrey Hung upgraded Bluebird Bio from Underweight to Equal-Weight and raised the price target from $3 to $7 ahead of an expected FDA decision.

Hung said that Morgan Stanley believes the company's drug lovo-cel is likely to be approved for sickle cell disease
“by the December 20 PDUFA date”
and that the stock will continue to move up heading into the FDA decision.

Hung also said that they expect investor focus to return to the company's gradual launches for Skysona, Zynteglo, and lovo-cel in the longer term.

Bluebird Bio shares have grabbed the interest of short-sellers with 23.16% of available BLUE shares being sold short, according to the latest data from Benzinga Pro.

More importantly, the disapproval of PRV is mind boggling and perhaps more damaging:


The Company’s BLA for lovo-cel was previously accepted for priority review by the FDA for patients with sickle cell disease ages 12 and older who have a history of vaso-occlusive events (VOEs) and has a “Prescription Drug User Fee Act (PDUFA) GOAL date of December 20, 2023”. bluebird “may be eligible” for a PRV should lovo-cel be approved for patients under the age of 18”

[Moose412]

Thanks, thought I was going crazy, missing something I should've seen, when it fact it was never said. It SEEMS like the FDA is out to get BLUE.

FDA tracker still has it on their calendar for the 20th. https://www.fdatracker.com/fda-calendar/

I'm curious if the cost of the crispr/vertex includes the required month long hospital stay. I bet it doesn't.

[stocksrising]

Long-term Follow-up Data From bluebird’s Gene Therapy Program in Sickle Cell Disease Support Durable, Potentially Curative Benefits Through Stable Production of Anti-Sickling Adult Hemoglobin and Resolution of Vaso-Occlusive Events
Oral presentation to include data on 47 patients through five years of follow-up (median 35.5 months, range 0.3-61 months)

Endpoints of sVOE-CR and VOE-CR achieved in 94% (32/34) and 88% (30/34) of evaluable patients respectively

100% of adolescents (10/10) experienced complete resolution of VOEs and sVOEs, providing evidence of potential benefit for this population

Impact on hemolysis markers and health-related quality of life measures further support potential therapeutic benefits

SOMERVILLE, Mass.--(BUSINESS WIRE)--Dec. 9, 2023-- bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or “bluebird”) today announced new and updated efficacy, safety and health-related quality of life (HRQoL) data from the Phase 1/2 HGB-206 Group C and Phase 3 HGB-210 studies of lovotibeglogene autotemcel (lovo-cel) gene therapy for sickle cell disease through five years of follow-up (median 35.5 months, range 0.3-61 months). Data were highlighted in a press briefing at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition and will be presented in an oral presentation on Monday, December 11, 2023 at 4:30 p.m. Pacific Time.

“Years of long-term follow-up continue to suggest that lovo-cel has the potential to address the underlying cause of sickle cell disease and provide robust clinical benefits for patients,” said Richard Colvin, M.D., Ph.D., chief medical officer, bluebird bio. “The data presented at ASH give us confidence that these results can be sustained over time and may translate to meaningful and lasting impact on quality of life for people living with sickle cell disease who need and deserve new treatment options.”

The analysis presented at ASH focused on 47 people living with sickle cell disease who received lovo-cel in the HGB-206 Group C and HGB-210 studies following enhancements to the treatment process and manufacturing protocols. Over 85% of patients required ≤2 mobilization cycles prior to infusion. As of the February 13, 2023 cutoff date, all patients had stable production of anti-sickling adult hemoglobin after infusion through last follow-up (median >40% HbAT87Q), and vaso-occlusive events (VOEs) and severe vaso-occlusive events (sVOEs) were eliminated or significantly reduced in all patients, further suggesting that lovo-cel has shown a durable impact on the underlying cause of sickle cell disease. The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.

Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease, with the most patients treated and longest follow-up in the field. As of February 13, 2023, 59 patients were treated across the entire clinical development program with follow-up beyond 8 years in the earliest treated patients.

The therapy was approved on December 8, 2023 by the U.S. Food and Drug Administration and is currently marketed as LYFGENIA in the U.S.

Updated efficacy data continue to support transformational impact on VOE burden

In the studies, VOEs are defined as episodes of acute pain with no medically determined cause other than a vaso-occlusion, lasting more than two hours and severe enough to require care at a medical facility. This includes acute chest syndrome requiring oxygen treatment and/or blood transfusion, acute hepatic sequestration, acute priapism lasting 2 hours and requiring care at a medical facility and acute splenic sequestration. sVOEs require a 24-hour hospital stay or emergency room visit, or at least two visits to a hospital or emergency room over a 72-hour period, with both visits requiring intravenous treatment; all VOEs of priapism are also considered sVOEs.

As of February 13, 2023, 34 of the 47 patients treated in HGB-206 Group C and HGB-210 were evaluable for the primary and secondary endpoints of complete resolution of VOEs and sVOEs with a median follow-up 36.3 months (12.1, 61).

32/34 patients (94%) experienced complete resolution of sVOEs, maintained for a median (min, max) of 35.8 months (20.2, 61).
30/34 patients (88.2%) experienced complete resolution of all VOEs, maintained for a median (min, max) of 35.8 months (20.2, 61).
Patients who experienced VOEs at any time post-treatment through long-term follow-up (n=8) experienced significant reduction in VOE frequency and severity compared to before treatment.
All 8 patients experienced a reduction in VOEs of at least 50%.
Hospital days and admissions were reduced by as much as 100% (annualized median hospital days reduced from 15.75 (3.5, 136) pre-treatment to 2.20 (0.0, 25.4); (annualized median reduction in hospital days was 85.5% (31.7%, 100%).
Results of a sub-analysis of data from adolescent patients, presented for the first time, showed complete resolution of VOEs and sVOEs in 10/10 (100%) of patients during the 6-18 month enrollment period.
“These new data provide further evidence that lovo-cel can provide significant improvements in quality of life for people living with sickle cell disease and that the effects are durable for at least five years,” said Julie Kanter, M.D., a lovo-cel investigator and director of the University of Alabama Birmingham Adult Sickle Cell Clinic and associate professor in the Division of Hematology and Oncology. “We see meaningful changes in hemoglobin, excellent production of anti-sickling hemoglobin, and improvement in all markers of hemolysis which further reinforces the clinical effects of lovo-cel. This one-time, transformative therapy has the potential to target the underlying cause of the disease and reduce both pain and fatigue—outcomes that matter to people living with sickle cell disease and their families. These findings support the positive impact of lovo-cel on the biology of sickle cell disease and on patients’ clinical outcomes and quality of life.”

Clinical outcomes were further supported by improvements in total hemoglobin and markers of hemolysis

Red blood cells normally break down in the body through a naturally occurring process called hemolysis. In sickle cell disease, hemolysis happens too quickly due to the fragility of sickled red blood cells, resulting in hemolytic anemia. With the exception of patients with alpha-thalassemia trait, all patients had improvement in total hemoglobin and markers of hemolysis; improvements were sustained through last follow-up. As of the February 13, 2023 cut-off date:

Following engraftment, non-transfused total Hb and %Hb fractions stabilized by approximately 6 months after lovo-cel infusion.
Median percent of gene-therapy derived anti-sickling adult hemoglobin (HbAT87Q) was maintained generally at >40% of non-transfused total Hb throughout follow-up.
From six months post-infusion through the last visit, several indicators of the health of red blood cells suggest that treatment with lovo-cel improved biological markers for sickle cell disease to normal or near-normal levels. Lactate dehydrogenase and indirect bilirubin levels normalized and reticulocyte counts approached normal levels.
The majority of patients experienced sustained improvements in key domains of health-related quality of life measures

The burden of sickle cell disease impacts every aspect of patients' lives. Health-related quality of life (HRQoL) findings in patients treated in HGB-206 Group C were generated using the Patient Reported Outcomes Measurement Information System 57 (PROMIS-57), a validated instrument in sickle cell disease.

Improvements in key domains including pain interference, pain intensity, and fatigue were demonstrated, showing statistically significant improvements as early as month six which were sustained up to 36 months for pain intensity and 48 months for pain interference and fatigue. These findings provide a broader understanding of the potential impact to daily life over time following treatment with lovo-cel.

[stocksrising]

Not sure of the Answers (questions asked), However it is un-typical of FDA to make a decision prior to published PDUFA date and this was surprise to even Morgan Stanley( increased price target- Upgrades Bluebird Bio to Equalweight From Underweight, Boosts Price Target to $7 From $3) on same day as approval…looks like today BLUE Pr’d updated Safety and stressed that the deaths/ side affects were from earlier versions:

Safety results summary

The majority of adverse events in patients treated in HGB-206 Group C and HGB-210 were attributed to underlying sickle cell disease or conditioning with busulfan. Nonserious adverse events related to lovo-cel included infusion reactions (abdominal discomfort, decreased diastolic blood pressure, and nasal congestion) each in one patient (2.1% each). Serious adverse events related to lovo-cel were reported in two patients with comorbid alpha-thalassemia trait and they included two SAEs each of Anemia (4.3%) and 1 SAE of Myelodysplastic syndrome (2.1%), the diagnosis of which remains under evaluation. One patient died due to sudden cardiac death which was deemed unrelated to lovo-cel.

As previously reported, cases of acute myeloid leukemia were observed in two patients from the HGB-206 Group A cohort who were treated with an EARLIER VERSION of the therapy prior to enhancements to the treatment and manufacturing processes. Both patients died due to aforementioned leukemia.

No graft failure, replication-competent lentivirus or vector-mediated insertional oncogenesis was observed across the entire clinical development program.

[Golden Cross]

That sucks

[Moose412]

After the unsubstantiated claim by the FDA of blood cancer that caused the reorg of BLUE to BLUE and 2Seventy and the resolution of that, this is absolutely obscene to include the black box warning. An absolutely obscene course of action.

I have a question - did I miss the FDA announcing somewhere that they would be making this decision on BLUE before the 12/20 date previously published?

I do have a second question - as recently as June of 2023, BLUE's website listed "multiple undisclosed" in its pipeline. What happened to those?

[novicetrader]

BLUE/VRTX/CRSP- All 3 Crisper tickers down on the 1st time approval news. I suppose Adam F is also a big factor in the downdraft, death knell for companies whenever he gets negative on them.

[TheFinalCD]

LOL THIS IS INTERESTING

pic.twitter.com/T9nOyS8yrm

— Goose Data (wa/wa) (@GooseData) December 8, 2023

[TheFinalCD]

THANKS FOR THE INFO

$VRTX $CRSP Casgevy $2.2 million, no black box.$BLUE Lyfengia $3.1 million, black box.

Bluebird will have to lower its price, right? How can it not?

Also, Bluebird confirmed to us that it DID NOT get a pediatric rare disease voucher for this approval.

Recall, the company…

— Adam Feuerstein ✡️ (@adamfeuerstein) December 8, 2023

[TheFinalCD]

Casgevy, developed by partners Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP), and bluebird bio's (BLUE.) Lyfgenia were approved for people aged 12 years and older.
https://www.reuters.com/business/healthcare-pharmaceuticals/us-approves-two-gene-therapies-sickle-cell-disease-2023-12-08/

HERES WHY https://investorshub.advfn.com/boards/read_msg.aspx?message_id=173389940

[stocksrising]

Adam F confirmed what/why sell off


$VRTX $CRSP Casgevy $2.2 million, no black box.

$BLUE Lyfengia $3.1 million, black box.

Bluebird will have to lower its price, right? How can it not?

Also, Bluebird confirmed to us that it DID NOT get a pediatric rare disease voucher for this approval.

Recall, the company had presold the voucher for $100M, assuming it would get one.

[stocksrising]

Yes!!!! GLTA

Bluebird bio has won U.S. Food and Drug Administration approval of its Lyfgenia gene therapy for sickle-cell disease, making it one the first two cell-based gene therapies to win a U.S. green light for the inherited blood disorder.

The FDA on Friday said the approval covers Lyfgenia for the treatment of patients 12 years of age and older with sickle-cell disease and a history of vaso-occlusive events.

The FDA nod, nearly two weeks ahead of the target action date of Dec. 20, comes on the same day the agency cleared the Casgevy gene-therapy from CRISPR Therapeutics and Vertex Pharmaceuticals, marking the first U.S. approval for a drug using the gene-editing technology known as Crispr.

[murocman]

Approved!

[Monksdream]

Early Barchart 100
https://www.barchart.com/stocks/performance/percent-change/advances?timeFrame=today&viewName=main&screener=overall&orderBy=percentChange&orderDir=desc

[Golden Cross]

Think it will get that $7 target very soon

[stocksrising]

Morgan Stanley Upgrades
Bluebird Bio to Equalweight From Underweight, Boosts Price Target to $7 From $3

[Golden Cross]

Nice..

[stocksrising]

As end of Q3 —-
Ended quarter with $227M in cash, cash equivalents, marketable securities and restricted cash –But they do burn, so for sure Cap raise in Q1, 2024 ( mgmt said the same)

[Golden Cross]

Really liking $BLUE here... Believe they have $171.0M. cash...

[stocksrising]

From your lips to market Gods ears 🙏
In excess of 100k shares in various accts!! $1mm value by e.o.m sets up a very Merry Happy New Year!! :) GLTA

[Moose412]

Approval will be had, making the run 3/3. The big breaker will be pricing reveal and contrast against whatever CRSPR/Vertex do. I'm anticipating $10 by end of month/year. My conviction is even more so given that practically nobody is talking about Blue. Absolutely my biggest position ever, value wise. IMO, Blue will never see $3.50 again, let lone the 2s!

[stocksrising]

Yes, either double digit run with Approval or back into 2-3’s or less!! Fingers crossed, 2nd biggest position after RVNC & TARA ;)

[Golden Cross]

Dec 20th Big date here $BLUE

[stocksrising]

bluebird bio to Present New and Updated Data from Gene Therapy Programs in Sickle Cell Disease and Beta-Thalassemia at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition
Mentioned: BLUE
--Updated long-term efficacy, safety, quality of life data and iron management outcomes from adult and pediatric patients with transfusion-dependent beta-thalassemia (n=63) treated with beti-cel through 9 years of follow-up (n=1) will be delivered in two poster presentations

bluebird bio, Inc. (Nasdaq: BLUE) today announced that new long-term efficacy, safety and health-related quality of life (HRQoL) follow-up data from its lentiviral vector (LVV) gene therapy programs in patients with sickle cell disease who have a history of vaso-occlusive events and patients with beta-thalassemia who require regular red blood cell transfusions will be presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.

[stocksrising]

Good guess , but history shows this company burns cash, and ~$103mm could help if approved…they will still come to market for raise, but hopefully at higher levels, especially if Sickle Cell approved..

FYI:
FDA panel greenlights approval of first human drug using CRISPR

Food and Drug Administration (FDA) advisers cleared the way for the first FDA approval of a human drug using CRISPR technology.

The news: FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee said exa-cel, the sickle cell gene therapy developed by BIO members Vertex Pharmaceuticals and CRISPR Therapeutics, is safe for clinical use.

[Moose412]

Been quite the sell-off, as you predicted. But with yesterday's 8-K w the pre-sale of the PRV, this is looking really nice.

[Monksdream]

[Monksdream]

BLUE trying to break the $4 resistance

[Moose412]

And IT is in! Edit: Going back to review, other notices like this I received in April 4th for two other tickers, and noticed there was the same notice from April 4 23 for BLUE. Still bullish AF!

[The maintenance requirement for your holding below has changed. This may increase or decrease your available purchasing power.

Account: XXXX-ZZZZ
Holding Ticker Symbol: BLUE
Holding Description: BLUEBIRD BIO INC COM
New requirement: 100%
Old requirement: 25%]

[Moose412]

Bear raid over soon, me thinks.

[stocksrising]

2 approved Drugs, Priority review and Pdufa date in Q4 on Sickle Cell, and this pig can’t move out of the sloppp!! Wtf

Prob sold off bcuz Auth. shares doubling and near term Capital raise again!! Dilution should be in its corporate name!! Certainly in its DNA

[Moose412]

Crazy action today. Waiting patiently for a brokerage notice about margin requirements for BLUE.

[Moose412]

Continuously.

[stocksrising]

Bear Raid!

[stocksrising]

If we can get this PR , should move high single digits.. then sell off on Capital raise, then double digits!! Otherwise, down she goes to $2-3ish!


• The Company anticipates BLA acceptance of lovo-cel for SCD in Q2 2023.

[Moose412]

I expect *new* news soon.

[stocksrising]

8K filing couple days ago showing $350mm shelf dinging ticker

[Moose412]

That's a pretty nice 10Q and a terribly sweet 8k today.

https://app.quotemedia.com/data/downloadFiling?webmasterId=90423&ref=317472177&type=HTML&symbol=BLUE&cdn=49eb2e78599a3f8993e1dbd77103d6c4&companyName=bluebird+bio+Inc.&formType=10-Q&formDescription=General+form+for+quarterly+reports+under+Section+13+or+15%28d%29&dateFiled=2023-05-09


https://app.quotemedia.com/data/downloadFiling?webmasterId=90423&ref=317472175&type=PDF&symbol=BLUE&cdn=a27bb9bb8a132409a5d38d92346a9d24&companyName=bluebird+bio+Inc.&formType=8-K&formDescription=Current+report+pursuant+to+Section+13+or+15%28d%29&dateFiled=2023-05-09

["The first quarter of 2023 saw us continue to establish and scale the commercial model for ex-vivo gene therapy in the US through the
launches of ZYNTEGLO and SKYSONA and lay the foundation for revenue generation for years to come for bluebird," said Andrew
Obenshain, chief executive officer, bluebird bio. "Additionally, with the submission of the lovo-cel BLA for sickle cell disease in April,
bluebird has taken a pivotal step towards realizing its most significant opportunity yet -- bringing a transformative gene therapy to
individuals living with sickle cell disease."]

[Moose412]

I have a similar position - 98,050 in the high $4 avg., from $3 -$7.6x.

BLUE will be 3/3. Patience.

[stocksrising]

FYI

bluebird bio Submits Biologics License Application (BLA) to FDA for lovotibeglogene autotemcel (lovo-cel) for Patients with Sickle Cell Disease (SCD) 12 years and Older with a History of Vaso-Occlusive Events.

The severity of sickle cell disease, and its impact on patients and caregivers, has been underappreciated and overlooked for far too long. Transformative therapies for this community are long overdue," said Andrew Obenshain, chief executive officer, bluebird bio. "We are pleased to have satisfied the Agency's questions about comparability to enable our BLA submission, and to take this important step toward making lovo-cel available for individuals living with SCD."

Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease. The BLA submission is based on efficacy results from 36 patients in the HGB-206 Group C cohort with a median 32 months of follow-up and two patients in the HGB-210 study with 18 months of follow-up each. The BLA submission also includes safety data from 50 patients treated across the entire lovo-cel program, including six patients with six or more years of follow-up.

The FDA previously granted lovo-cel orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation for the treatment of SCD.
Plus a new 13g filing last week :)

[stocksrising]

Here is an example from a few accounts that hold Blue( cant show all, don’t need certain peeps to see, lol but it adds up to ~100k :)

Total value
Shares owned
Average cost
Today's gain/loss
$154,800.00
45,000.000
$5.59
+$3,600.00 (+2.38%)


Shares owned
Average cost
$17,200.00
5,000.000
$4.50

[Moose412]

You picked up 90k shares since September?

[stocksrising]

I have ~100,000 reasons why I’m hoping
So! Lol :)

[Moose412]

I fully believe it will be 3 for 3.

[stocksrising]

Either kevin Tang buying more,
Or these tards are close to Application approval…we shall see soon