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Pretty quiet round here. This should be good for CYRX . . .
EMA panel recommends approval of Bluebird Bio's first gene therapy
Tamara Mathias MARCH 29, 2019
https://www.reuters.com/article/us-bluebird-bio-ema-zynteglo/ema-panel-recommends-approval-of-bluebird-bios-first-gene-therapy-idUSKCN1RA1AV
(Reuters) - A European Medicines Agency panel on Friday recommended a conditional marketing approval for a gene therapy from Bluebird Bio Inc as a genetic blood disorder treatment, setting the stage for the U.S. biotech to win its first regulatory nod. Final approval depends on the European Commission, which generally follows recommendations from the Committee on Human Medicinal Products (CHMP). The decision is expected in the second quarter, Bluebird said in a statement.
The drug Zynteglo, formerly known as LentiGlobin, was developed to treat a specific group of patients with transfusion-dependent beta thalassemia (TDT).
Zynteglo is intended for patients 12 years and older who need regular blood transfusions to manage their disease and have no matching donor for a stem cell transplant, CHMP said here Pharmaceutical companies have been investing heavily in potentially life-changing gene therapies over the past few years, but patients and insurers expect exorbitant price tags. Bluebird did not confirm Zynteglo’s price, but said it plans to implement a payment system that staggers costs in installments over a five-year period.
The company has previously said it believes the “intrinsic value” of the one-time infusion is about $2.1 million per patient.
Wedbush Securities analyst David Nierengarten has said in a note last month that he expected Bluebird to price much lower, at about $800,000 per patient in the European Union. Spark Therapeutics Inc in December 2017 won the first U.S. approval for its gene therapy to treat a rare form of blindness and priced it at $850,000 per patient in the United States.
TDT patients have a genetic mutation that hampers their ability to produce hemoglobin, the protein in red blood cells that carries oxygen through the body.
Patients are diagnosed at an early age and must receive lifelong blood transfusions every few weeks. “We basically make your bone marrow a manufacturing plant for producing hemoglobin,” Chief Executive Officer Nick Leschly told Reuters. “We actually take out your cells, we use a virus to fix the cells and then we put the modified cells back in the body. And they go to the bone marrow and start producing what we’ve hopefully instructed those cells to do.”
Bluebird plans to file for regulatory approval of Zynteglo in the United States before the end of this year and is also testing the gene therapy in patients with sickle cell disease. Bluebird hopes to have approvals for four new treatments, including one for multiple myeloma, by 2022, Leschly said.
"shame on us?" I think you mean shame on them!
Yep, right on time according to CB . . .
CYRXorbust Thursday, 03/14/19 12:06:02 PM
Re: hyperopia post# 11589
. . . I made it clear to him to NOT vote in our favorite board member who loves to unload his shares the second he gets his hands on them. should be right around the corner from that
And BTW - epcjmc, you could have purchased shares in the spring of 2017 cheaper than that, and then sold them in the summer of 2018 for an even larger profit than that with just a couple keystrokes.
The third Friday in March, June, September, and December are quadruple witching days - when stock options, stock futures, stock index options, and stock index futures all expire simultaneously and cause very high volumes, especially the last hour of trading.
It was $35M. And no, it hasn't expired. They still have $31.6M available. From yesterday's 10-K:
On August 24, 2018, we entered into a sales agreement (the “Sales Agreement”) with Jefferies LLC (“Jefferies”) under which we can sell up to an aggregate offering price of up to $35 million of the Company’s common stock (the “Shares”), from time to time through an “at the market” equity offering program (“ATM Prospectus”). . . . .
. . . During the year ended December 31, 2018, the Company received net proceeds of $3.4 million through the sale of 248,839 shares of its common stock, after deducting sales commissions and other offering expenses of $44,200, that were offset against the proceeds from this offering.
BTW - that would be an average selling price of about $13.66
Maybe for management’s 2019 Omnibus Equity Incentive Plan?
See? I knew this would happen when you guys authorized them to increase the share count.
All in all, it was a good quarter and a solid year. 40 net new trials were added this quarter, and 120 clinical trials were added for the year, for an impressive total of 357. Cryoport said they held nearly 40% of the clinical trial market in the Regenerative Medicine space, which is a good thing, because clinical trials looks to be where the bulk of their revenues will come from for the next year or two.
So, 4 MAA's were filed in Europe and 2 BLA's were filed in the United States during 2018, and we found out from some good questioning (from the B. Riley FBR analyst, Andrew D’Silva) that 5 are Cryoport clients. This is one of the few things that management offered guidance on last year and they were accurate on that. But so far, no news on when to expect approvals or when they will begin commercial sales. They now anticipate six additional BLA and MAA submissions from Cryoport’s clients in 2019. Novartis expects to increase Kymriah revenues fourfold, and Gilead is projecting sales of Yescarta to double this year, but it still looks to be a slow ramp for commercial revenues.
And contrary to a belief held by a certain poster here, (cough cough) the company said it is actively pursuing potential acquisitions. Shelton said “we'll look at software companies, we'll look at components of manufacturing that could provide strategic advantage. We'll also look at adjacencies and we talked about some of those in the past, like storage and like, again, software and other services that we think fit with our Company. So, we definitely are looking at them, but we have yet to pull the trigger.”
Don't worry CB, I think you will still have another chance to sell in the teens.
Although the company doesn’t provide revenue forecasts, I would like to point out that I did. And I was pretty damn close. . .
Cryoport reported quarterly revenue of 4.0M, 4.6M, 5.3M, 5.7M, and 19.6M for the year. And reported 2.1M in commercial revenue for the year.
Now here’s my post from a year ago:
hyperopia Wednesday, 03/14/18 10:13:28 AM
Re: Rev3lation post# 11146
Post # 11149 of 11580
. . . I look at the clinical trials, animal health and reproductive as the base-line revenue, which grew about 50% overall last year. . If this continues at this rate, that alone would be $18M for 2018. If it only grows at 40% then it would be $16.8M, and if it increases to 60% it would be $19.2M. To be conservative,I think it will be at the lower end of this range - say $17M. Then the question is: how much will commercial revenue add to this baseline? I’m being conservative and currently estimating only about $2M for the year, for total revenues in 2018 of $19M. I have lowered this estimate twice; from $23M to $21M after last quarter’s conference call and then from $21M to $19M after this last call. Right now my model shows these quarterly estimates for 2018: Q1 3.52 Q2 4.31 Q3 5.23 Q4 5.9 = 18.96. This represents my conservative estimate, and I have run a range of scenarios and hope to revise this estimate upward as we get further updates throughout the year
Ha! I would have taken the under, as my line would have been over or under one! Interesting new format and surprising that they seem to have lost a few analysts. Wonder if there is some blowback from going to Petrichor for funding?
Hi H2R. I actually think Antares’ management is being conservative with their 2019 guidance, as my model shows ~120M for 2019, and revenues begin rolling in the second half of the year and into 2020. I expect many more 52 week highs. Best of luck with your investments as well.
gi197845. It’s a little difficult to take you seriously, but I will answer this post just to counter the FUD.
LPCN’s TLANDO is not competition at this time because it is not approved.
Lipocine has received Complete Response Letters (CRL’s) from the FDA for TLANDO both times they applied for approval. The letter last year called for data from an ambulatory blood pressure monitoring study that would provide "definitive evidence" that Tlando, a testosterone replacement therapy, offers clinically meaningful increases in blood pressure for men who have deficiencies of the hormone. Regulators also want Lipocine to confirm the reliability of testosterone and maximum serum concentration (Cmax) data connected to Tlando, and to explain why the pre-specified Cmax secondary endpoints for the drug aren't applicable here. Additionally, Lipocine must come up with stopping criteria that adequately identify patients who should discontinue Tlando treatment.
Antares’ Xyosted is a relatively painless self-administered weekly auto-injection to the abdomen as shown here:
https://www.xyosted.com/application/files/8515/4335/5813/IFU-_FINAL.pdf
Antares: Sales Growth Guidance for 2019 is Extremely Bullish (ATRS, $3.60, Buy)
https://smithonstocks.com/antares-sales-growth-guidance-for-2019-is-extremely-bullish-atrs-3-60-buy/
Hi Doc logic
you wrote:
THANK YOU very much IkeEsq, barcode, notbrad, Poor Man, H2R, pairofoldsocks and any other ASM attendees who took the time and effort to share their notes, thoughts, and impressions from the meeting. It is very much appreciated.
CN, I agree that it was a decent deal, and it shows Cryoport is turning the corner. Less than 2 years ago they raised capital at a nearly 50% discount at $2! What I have a problem with, is Petrichor characterizing the deal as a “partnership” and an “investment” like they intended to be long-term shareholders, when in fact, that wasn’t their intention at all, and they actually appear to be getting ready (like CB) to sell into the very next pop. I mean they didn’t even bother to wait six months or a year to register them. And then for Shelton to further the mischaracterization, pretending that he thoughtfully structured the deal that way because he actually cared about diluting shareholders. As my father likes to say; “Don’t piss on my back and tell me it’s raining!”
Hi flip, my grandfather died of GBM in 2016 and his doctor recommended using the Optune device. He had to pay out of pocket nearly $20k per month since medicare didn’t cover this “treatment” and he passed less than a year after being diagnosed, nearly broke. At the time, I didn’t know much about GBM or Optune, and I now regret not becoming more informed, because I had my suspicions. While I haven’t read all your posts, your recent revelations about the Optune trial are like a gut punch, and make me both sick and infuriated. Thanks for that. :)
Well that sure didn’t take long . . .
https://www.sec.gov/Archives/edgar/data/1124524/000114420419003169/tv511690_s3.htm
So Tadd Wessel, Founder and Managing Partner of Petrichor, said, "We are excited to partner with Cryoport . . .” and Sir Bruce Keogh, a member of Petrichor's Advisory Board commented, "We are pleased to be investors in Cryoport.“
Jerrell Shelton, CEO of Cryoport, said, regarding the transaction, "Petrichor is a premier healthcare-dedicated investment firm who has worked with us to structure this transaction to minimize dilution to our shareholders."
Yeah some “partnership” . . . and “pleased investors?” Really? And how exactly is that minimizing dilution Jerell? What a bunch of #@%&!
What? You’re not buying the “pursuit of targeted, strategic acquisitions” line? Or the new addition to the Board of Directors for his experience in “seeking, defining, negotiating, and implementing strategic collaborations?” I guess I naively thought it’s possible or even probable the cash was at least in part for an acquisition. Not that it really matters anyway, a $10-20M acquisition is not going to move the needle either. I’m already on record as speculating on Be The Match as the target since they are already integrated, in the same way I think Cryoport will be absorbed by World Courier/AmerisourceBergen eventually.
As far as Dacos, I just translated their website from Danish to take a peak at their products, clients, and size (one director and three administrators), then yawned along with the market.
https://translate.google.com/translate?hl=en&sl=da&u=https://dacos.dk/&prev=search
mediocrity? You flatter them.
Uh CB, you didn't sell this summer when it was hovering near $17? What price are you waiting for?
Yeah, that and insurance . . . don't forget the insurance.
Well Dacos says “We receive requests for these solutions on a daily basis” so that implies there could be at least a couple a day. “Leading manufacturer” and “global client base” sound good, but Dacos appears to be a relatively small company, so I doubt this partnership will really move the needle. The market apparently gave the news a big yawn.
The Russell indexes are only reconstituted once a year in June and May is “ranking month.” The lowest market cap last year was around $150M, so while it’s certainly possible, it’s probably not likely.
Here's some chatter . . Zacks: “we have discontinued coverage of CYRX, our prior opinions, outlook, analysis, revenue and EPS estimates, financial model and price target should be considered no longer reliable”
It's looking like a pretty good call right about now . . down 15% from there. I'm surprised it held up as well as it did yesterday while the market was tumbling, but looks like it caught up. Today is trip witch.
Wow. So now Cryoport is buying Huawei? JK ;))
I know speculation is rampant over there but it looks like a real estate transaction to me.
Less - I was highlighting the investment thesis in the CEO's own words. It may have been 7 years ago, but the investment thesis hasn't changed. I will admit that I took some editorial liberties to make the point and save the readers time, but did post the entire interview.
Remember this interview with Linda F. Powers?
Highlights:
Q: What attracted you to Northwest Biotherapeutics?
Ms. Powers: What attracted me to Northwest Biotherapeutics was that they have a platform technology that should be applicable to pretty much all cancers, and which is offering really a major improvement in survival times for patients, with no toxicity . . . .
Ms. Powers: We are entering the home stretch now. . . It is the operational aspect of completing the clinical trial, which is a huge managerial task with a lot of moving parts. There are also a lot of issues in regard to the market strategy, the pricing strategy, the reimbursement strategy and the manufacturing.
Q: Why should potential investors pay attention to Northwest Biotherapeutics?
Ms. Powers: Biotech investments are high-risk/high-return investments. However, Northwest Biotherapeutics has survived, produced strong results and gotten to such a late stage in the process, where we have greatly reduced the risk: “de-risking” as the investor community likes to call it. In addition to the de-risking, investors can still have the upside as the market cap of Northwest is still very low.
Northwest has now reached the stage where it has consistent, strikingly positive results across multiple different cancers, and across many years, with a decade
worth of clinical trials. Yet, as just mentioned, Northwest’s market cap has not yet gone through its inflection, so the whole upside is still there. That is the kind of combination of circumstances that is well worth looking at for an investor.
Q: Final thoughts, what should people remember most about Northwest Biotherapeutics?
Ms. Powers: I would just refer your readers as a comparison to look at the inflection, the value run-up that Dendreon went through when it reached the end of its late-stage clinical trial and had a successful outcome of that trial. They went from a market cap of $300 million to a market cap of $5 billion and that is an amazing upside potential. . . . so stay tuned!
http://www.ceocfointerviews.com/interviews/NWBO-NorthwestBio11-CEOCFO-Article.pdf
Agree that this theory is unlikely. Cryoport has alluded to a strategic acquisition a couple of times now, including this financing news PR:
“This financing provides capital to further expand the breadth and depth of our solutions offerings in the rapidly developing cell therapy markets and life sciences industry at large, including the pursuit of targeted, strategic acquisitions.”
If Cryoport is making preparations to support Northwest Bio’s commercial launch, they would not make this information public until Northwest Bio makes it known first, but they would likely need to raise some capital.
And yes, I have read SOS, and think Michael Lewis could write an amazing book about the Northwest Bio story.
Just throwing it out there. It certainly could be separate, coincidental events. The timing is interesting and Petrichor seems an odd choice and not the typical investment bank that Cryoport has used in the past, so perhaps they were another party’s choice.
The sale of the property in the UK by Northwest Bio ensures they will have the necessary funds to finish the trial and complete the next steps to file BLA’s. I think Northwest Bio will finally release the long awaited topline data in the next six months (possibly in a few months), and they would like to make the big unveil on the grand stage at ASCO. I think they waited this long until the data matured to increase the likelihood of success. And yes, there are issues with the trial, but I think they will reveal unprecedented survival data in this landmark glioblastoma trial that will be irrefutable. It will take several months to analyze the data and prepare BLA’s, so it may be early 2020 for the regulatory authorities to approve it.
Cryoport has a logistics center near each commercial customer’s manufacturing facility, so I would think Northwest Bio would be the same. The UK may be the first filing. Since Cryoport was used in the clinical trial, and the cryologistics are a part of the CMC section of the BLA, Cryoport would have to ensure that they could handle commercial volumes by the BLA filing. It may take Cryoport at least six months to scale up, so they would have to make a move soon.
One simple way this could fit is if Petrichor (or another entity) bought the property from Northwest Bio, and then sold a portion to Cryoport and plans to further develop the rest of the property or sell it.
Cryoport may be behind the UK Property Transaction. Today they announced a significant financing from Petrichor Healthcare Capital Management.
https://www.prnewswire.com/news-releases/cryoport-announces-25-million-investment-from-petrichor-healthcare-capital-management-300767257.html
Cryoport provides cryogenic logistic services for Novartis and Gilead for their CAR-T therapies, and has logistics depots next to both of their manufacturing facilities. Cryoport provided logistics services for Northwest Biotherapeutics during their trial and would require a significant amount of warehouse space for commercial production, and would begin preparations well in advance. As I noted on a post on the CYRX board, “Sir Bruce Keogh, is a member of Petrichor's Advisory Board and recent appointee to the Board of Directors of UK-based non-profit Cell and Gene Therapy (CGT) Catapult.”
https://petrichorcap.com
What We Do
Petrichor makes structured equity and credit investments across the global healthcare sector, with a focus on Biopharmaceuticals, Medical Technology, and Diagnostics. We provide flexible and tailored investment solutions for late and commercial stage companies. We work with both public and private companies to support their long-term growth efforts and have experience underwriting product acquisitions, product development activities, and commercial launches or expansions, among other types of situations. Our investment structures are designed to provide capital solutions that enable leading healthcare innovators to reach the next stage of their evolution. Petrichor acts as a strategic advisor, partnering with management, Boards of Directors, and other investors to help ensure the success of our companies.
Very interesting . . .Sir Bruce Keogh, is another member of Petrichor's Advisory Board and recent appointee to the Board of Directors of UK-based non-profit Cell and Gene Therapy (CGT) Catapult. Anyone else think this financing may be related to the recent purchase of Northwest Bio’s UK Property Transaction?
Decent terms. Wonder who Cryoport is buying?
“This financing provides capital to further expand the breadth and depth of our solutions offerings in the rapidly developing cell therapy markets and life sciences industry at large, including the pursuit of targeted, strategic acquisitions.”
And small world . . . the new Board of Director for Antares Pharma, Peter Greenleaf, is on the Petrichor Advisory Board.
https://petrichorcap.com
I think this was an initial grant of options (20 thousand at Thursday’s closing price of $3.11) to the newly appointed Director, Peter Greenleaf, under management’s Equity Compensation Plan. They vest over 12 quarters so he can’t exercise them yet.
H2R, I think you misunderstood. I meant Teva/Antares has more competition. I posted those articles to give background about the epinephrine competition and to point out (for those that might not know) that Teva/Antares are producing the only AB rated generic (the only easily substituted EpiPen) which is not just another generic epinephrine product like Amneal’s Adrenaclick, Kale’s Auvi-Q and Adamis’ Symjepi syringe. In theory this rating should help Teva/Antares gain a greater market share from Mylan. I think ADMP’s Symjepi will take a very small market share.
Why, after years of waiting and a year of EpiPen shortages, (now that Teva/Antares finally got approved) suddenly there’s even more competition?
The competition in this space is heating up. More background here:
https://www.pharmacytimes.com/contributor/andrew-abe-pharmd/2018/10/path-to-approval-first-truly-generic-epipen
Path to Approval First Truly Generic EpiPen
OCTOBER 08, 2018
The very first EpiPen (epinephrine) generic was approved by the FDA in August, and will hopefully be available to pharmacies in the coming months. But what is the true impact of this approval? And wasn’t there already a generic EpiPen on the market? That is half correct.
Previously, EpiPen was rated in the orange book as BX, which meant there was insufficient data to determine therapeutic equivalence to another product. Yet, we saw a 'generic' EpiPen being advertised. What happened was that another company, which owned an epinephrine product (Adrenaclick), also made an authorized generic.
An authorized generic is different from a true generic in that it is esentially the same as the brand name product, just marketed as a generic product. This authorized generic was marketed as a 'generic' epinephrine pen with the same active ingredient as EpiPen. But because EpiPen and the authorized generic for Adrenaclick were not truly generic, these products were not interchangable, and pharmacies would have to call a provider’s office to switch to the authorized generic.
Highlights for pharmacists:
If you receive a prescription for EpiPen, you can switch to the AB rated generic epinephrine by Teva. You would not need to call provider for permission to make switch.
If you receive a prescription for epinephrine pen, you can fill for what you have in stock and what is covered by insurance.
If you receive a prescription for any other any other brand epinephrine, it would not be interchangable to any other brand epinephrine.
The approval of a drug and device combination is exceptionally tricky as patents protect both the drug and the device separately. The high cost of brand name drug device combinations has lead the FDA to announce their Drug Competition Action Plan in 2017 which seeks to improve the generic drug approval process.
While the generic EpiPen might be the first generic drug device combination approved, it will definitely not be the last. Hopefully, this approval will lead the way for more approvals of generic drug device combinations.
Also:
With EpiPen competitors bearing down, can Mylan maintain its lead?
https://www.fiercepharma.com/pharma/opportunity-at-cafeteria-door-emergency-epinephrine-solutions-challenge-market-leader-mylan
Antares: Multiple New Product Launches Make for Extreme Complexity in Earnings Forecasts for 2019 to 2021 (ATRS, Buy, $3.59)
https://smithonstocks.com/antares-multiple-new-product-launches-make-for-extreme-complexity-in-earnings-forecasts-for-2019-to-2021-atrs-buy-3-59/
The good news keeps flowing. No wonder it's down today!
Yes H2R, patience is necessary, but often rewarded. I think 2019 will be a very good year for us in more ways than one! Best wishes