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The Companies That Led 2017’s Biotech Rally
https://benzinga.com/z/10927494
by market cap categories
https://seekingalpha.com/news/3319875-top-2017-ytd-performers-healthcare
My two favorites on the list! Hoping 2018 is a repeat.
Happy Holidays folks!
Top 2017 YTD performers—healthcare
https://seekingalpha.com/news/3319875-top-2017-ytd-performers-healthcare
Leading 2017 gainers in healthcare are:
Marinus Pharmaceuticals MRNS: +808%
PolarityTE COOL: 714%
XOMA XOMA: 507%
Madrigal Pharmaceuticals MDGL: 479%
AVEO Pharmaceuticals AVEO: 413%
Sangamo Therapeutics SGMO: 405%
CymaBay Therapeutics CBAY: 403%
Nektar Therapeutics NKTR: 365%
Esperion Therapeutics ESPR: 354%
Pieris Pharmaceuticals PIRS: 344%
Dynavax Technologies DVAX: 336%
ImmunoGen IMGN: 324%
Bellerophon Therapeutics BLPH: 316%
Spectrum Pharmaceuticals SPPI: 303%
Exact Sciences EXAS: 293%
BioLife Solutions BLFS: 287%
Pulse Biosciences PLSE: 285%
Immunomedics IMMU: 281%
Viking Therapeutics VKTX: 281%
Looks like GS jinxed it LOL. Two other factors that might have contributed to the overall crypto market crash, both pertaining to Bitcoin Cash.
1) CNBC collusion
http://bitcoinist.com/cnbc-collusion-roger-ver-bcash/
2) Insider trading allegations at Coinbase
https://www.theverge.com/platform/amp/2017/12/20/16800940/coinbase-bitcoin-cash-fork-insider-trading-probe
The market seems to be recovering though.
RXDX fun fact—It was born out of a reverse merger into an oil and gas company!
NTRK has been a successful TKR to pursue. Both $LOXO and $RXDX incorporated in 2013 and billion dollar co's 4 years later. RXDX born out of a reverse merge into an oil and gas company!
— M (@bio_clouseau) December 22, 2017
ARRY (+8%) Contributes Rights And Assets Related To Arry-797 Drug Program To Yarra Therapeutics
https://www.reuters.com/article/brief-array-biopharma-contributes-rights/brief-array-biopharma-contributes-rights-and-assets-related-to-arry-797-drug-program-to-yarra-therapeutics-idUSFWN1OM0DW
Diabetes Is Officially the Messiest Drug Market
https://www.bloomberg.com/news/articles/2017-12-21/steglujan-fda-approval-messy-diabetes-market-gets-messier
-A crowded space gets more so as the FDA approves Pfizer/Merck's Steglujan.
Goldman Is Setting Up a Cryptocurrency Trading Desk
https://www.bloomberg.com/news/articles/2017-12-21/goldman-is-said-to-be-building-a-cryptocurrency-trading-desk
Heard about this. Sounds fishy and I would avoid.
Bain Makes Bank in Two Rebounding Biotechs (MRNS, DRNA)
http://www.barrons.com/articles/bain-makes-bank-in-two-rebounding-biotechs-1513861107
JMP potential take-out targets: MDGL, HRTX, ADMS, MRNS
JMP Securities' Potential Takeout Targets: $MRNS $MDGL $ADMS $HRTX pic.twitter.com/FsPwidBemG
— Titan V (@titan_v16) December 21, 2017
FDA Closes Loophole, Enforces Pediatric Studies for Drug Developers
http://www.raredr.com/news/fda-closes-loophole-enforces-pediatric-studies-for-drug-developers
Today, the U.S. Food and Drug Administration (FDA) took steps toward preventing drug developers from sidestepping pediatric studies.
A draft guidance was issued to sponsors of drugs and biological products that submit orphan drug designation requests. It provides clarity on requirements for orphan drug designation, noting that the designation will not be granted unless the pediatric subpopulation meets 2 criteria.
The Clarification of Orphan Drug Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases states that if orphan drug designation for a pediatric subpopulation of a common disease is requested by a sponsor, it will not be granted if the prevalence of the disease is greater than 200,000, even if the pediatric subpopulation prevalence is below 200,000, unless:
— The disease in the pediatric population constitutes a valid orphan subset, and the drug meets all other criteria for orphan designation; or
— The sponsor can adequately demonstrate that the disease in the pediatric subpopulation is a different disease from the disease in the adult population, and the drug meets all other criteria for orphan designation. For example, if, as a scientific matter, efficacy from clinical studies in the adult population could not be extrapolated to the pediatric subpopulation, such information may help demonstrate that the disease in the pediatric and adult populations may be considered different diseases.
"The scientific and medical communities recognize that treating children safely and effectively requires data from pediatric studies. Children are different from adults in the way they may metabolize a given drug and in the side effects they may experience. Addressing the inadequate testing of drugs in pediatric populations has been a priority for the FDA, the medical community and Congress, and has led to important laws to ensure this important, vulnerable population is not overlooked,” said FDA Commissioner Scott Gottlieb, M.D. in the official FDA press release.
An example given in the draft guidance suggests that, previously, if the FDA grants pediatric-subpopulation designation for a sponsor’s drug for pediatric ulcerative colitis (UC) and the sponsor submits a New Drug Application (NDA) and Biologics Drug Application (BLA) for its drug to treat UC in adults, the sponsor would be exempt from having to conduct pediatric studies by having the pediatric-subpopulation designation for pediatric UC. Obtaining pediatric-subpopulation designation provides orphan incentives to study the drug in the pediatric population with the disease but does not mandate the sponsor to conduct those studies.
The FDA intends to discontinue the granting of pediatric-subpopulation designation to close this loophole.
“Unfortunately, some of the efforts intended to encourage pediatric drug development have been used by some drug developers to avoid their obligations to study drugs in pediatric populations,” said Gottlieb. “One such area is the longstanding practice of designating pediatric subpopulations of common diseases as orphan conditions. By making this designation, drug developers took advantage of an unintended loophole in a law that was, to the contrary, specifically designed to ensure that drugs are studied for pediatric indications. Today, we’re closing that loophole and announcing that we no longer intend to grant pediatric-subpopulation designations through the orphan drug program."
The agency is encouraging public comment on the draft guidance.
Array BioPharma Announces Strategic Collaboration with Pfizer
https://www.prnewswire.com/news-releases/array-biopharma-announces-strategic-collaboration-with-pfizer-300572969.html
—Novel combinations of binimetinib (MEK), avelumab (PD-L1), and talazoparib (PARP inhibitor) to be studied across tumor types
BOULDER, Colo., Dec. 19, 2017 /PRNewswire/ -- Array BioPharma (Nasdaq: ARRY) announced today that it has entered into a clinical trial collaboration agreement with Pfizer to investigate the safety and efficacy of several novel anti-cancer combinations, including Array's MEK inhibitor, binimetinib, with Pfizer's investigational PARP inhibitor talazoparib, and avelumab*, a human anti-PD-L1 IgG1 monoclonal antibody.
The companies are entering into this collaboration to explore the potential benefits of combining molecularly targeted therapeutics with the body's innate cancer-fighting abilities using immunotherapy.
"Array is excited to announce this partnership with Pfizer, an established global leader in Oncology therapeutics," said Ron Squarer, Chief Executive Officer, Array BioPharma. "These novel approaches combining targeted therapy and immunotherapy hold great potential to help patients fighting cancer in different indications, with an initial main focus on lung and pancreatic cancer."
"Preclinical data indicate that combining binimetinib with an immune checkpoint inhibitor and talazoparib could be a rational combination to test in the clinic," said Chris Boshoff, M.D., Ph.D., Senior Vice President and Head of Immuno-Oncology, Early Development and Translational Oncology, Pfizer Global Product Development. "We are looking forward to initiating the clinical studies with Array BioPharma to explore anti-tumor activity across various novel combination strategies, including both doublet and triplet approaches."
Under the terms of the agreement, Array and Pfizer will collaborate on a Phase 1b clinical trial to explore a series of novel combinations, investigating the safety and efficacy of the combination of binimetinib, avelumab and talazoparib across various tumor types. A multi-arm clinical trial is expected to establish recommended doses of different regimens combining the drugs. Initially the focus will be in non-small cell lung cancer (NSCLC) and pancreatic cancer, and additional indications will be explored at a later stage. The study is expected to begin by the third quarter of 2018, and results will be used to determine optimal approaches to further clinical development of these combinations.
Under the collaboration agreement, the trial will be sponsored and funded by Pfizer, with Array providing binimetinib supply.
*Avelumab is jointly developed by Merck KGaA, Darmstadt, Germany and Pfizer. Avelumab is under clinical investigation for treatment of NSCLC and pancreatic cancer and has not been demonstrated to be safe and effective for these indications. There is no guarantee that avelumab will be approved for these indications by any health authority worldwide.
About Binimetinib
MEK is a key protein kinase in the MAPK signaling pathway (RAS-RAF-MEK-ERK). Research has shown this pathway regulates several key cellular activities including proliferation, differentiation, survival and angiogenesis. Inappropriate activation of proteins in this pathway has been shown to occur in many cancers, such as melanoma, colorectal and thyroid cancers. Binimetinib is a late-stage, small molecule MEK inhibitor which targets key enzymes in this pathway. Binimetinib is an investigational medicine and is not currently approved in any country.
Binimetinib is being studied in clinical trials in advanced cancer patients, including the Phase 3 COLUMBUS trial in patients with BRAF-mutant melanoma and the Phase 3 BEACON CRC trial in patients with BRAF V600E-mutant colorectal cancer.
About Avelumab
Avelumab is a human anti-programmed death ligand-1 (PD-L1) antibody. Avelumab has been shown in preclinical models to engage both the adaptive and innate immune functions. By blocking the interaction of PD-L1 with PD-1 receptors, avelumab has been shown to release the suppression of the T cell-mediated antitumor immune response in preclinical models. Avelumab has also been shown to induce NK cell-mediated direct tumor cell lysis via antibody-dependent cell-mediated cytotoxicity (ADCC) in vitro. In November 2014, Merck KGaA, Darmstadt, Germany, and Pfizer announced a strategic alliance to co-develop and co-commercialize avelumab.
Avelumab is currently being evaluated in the JAVELIN clinical development program, which involves at least 30 clinical programs, including nine Phase III trials, and more than 7,000 patients across more than 15 different tumor types, including gastric/gastroesophageal junction, non-small cell lung cancer, renal cell carcinoma and ovarian cancer. For a comprehensive list of all avelumab trials, please visit clinicaltrials.gov.
Indications in the US
The FDA granted accelerated approval for avelumab (BAVENCIO®) for the treatment of (i) adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC) and (ii) patients with locally advanced or metastatic urothelial carcinoma (UC) who have disease progression during or following platinum-containing chemotherapy, or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. These indications are approved under accelerated approval based on tumor response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
Would new combos be PIIM-based or EP+Tavo like PISCES is what I often wonder. If latter, they can start from a p2 study but if former, from p1. However PIIM-based trials are likely to be more potent.
Ideally they should start multiple p1 PIIM-based trials but that would only be possible with financial backing from MRK or someone else in the form of an upfront payment. I find it unlikely that they will have to wait until conclusion of PISCES to see some of these other trials started. We’ll see.
8 Takeout targets to watch in 2018
https://www.biopharmadive.com/news/8-takeout-targets-to-watch-in-2018/513000/
The 3 Best Biotech Stocks of 2017
https://finance.yahoo.com/news/3-best-biotech-stocks-2017-140900573.html
Biotech stocks rocked in 2017. At least a lot of them did. The SPDR S&P Biotech ETF has chalked up a gain of 40% with only a couple of weeks left in the year -- twice the return of the S&P 500 index.
But which biotech stocks performed best of all? Excluding biotechs with tiny market caps below $200 million, three stocks take the top honors for the year: Sangamo Therapeutics (NASDAQ: SGMO), XOMA Corporation (NASDAQ: XOMA), and Marinus Pharmaceuticals (NASDAQ: MRNS). Here's what made 2017 such a fantastic year for these biotech stocks.
Sangamo Therapeutics
Sangamo Therapeutics ranks as the third best biotech stock of 2017, with a sizzling gain of more than 440%. The company began the year with a market cap of a little over $200 million and is on track to finish with a valuation topping $1.4 billion.
While Sangamo made solid progress from the outset of the year, an update in May lit a fire beneath the stock. When the biotech provided its first-quarter results on May 10, it also announced a partnership with Pfizer in developing gene therapy programs for hemophilia A. This endorsement by one of the world's largest drugmakers provided a big boost for Sangamo, whose gene therapy program uses zinc finger nuclease genome-editing technology, an approach that has been eclipsed over the past few years by newer technologies such as CRISPR.
There was plenty of other good news for Sangamo this year as well. The biotech received orphan-drug and fast-track designations for several of its pipeline candidates. The first patients received treatment in a couple of phase 1/2 clinical studies evaluating gene therapies SB225 and SB913. Probably the only factor holding Sangamo stock back somewhat was the company's public stock offering, which generated gross proceeds of $83.4 million.
XOMA Corporation
XOMA Corporation claimed the second-highest performance in 2017 among biotech stocks, with its share price soaring close to 700%. Earlier this year, XOMA was a nano-cap stock valued around $25 million. It will probably end 2017 with a market cap of $275 million or more.
Nearly all of XOMA's gains have come since late August. The company announced on Aug. 25 that it was licensing anti-IL-1 beta antibody gevokizumab to Novartis. Under the terms of this deal, XOMA received $31 million in upfront payments (including a $5 million equity investment) plus potential milestone payments and tiered royalties on future sales of gevokizumab.
That proved to be just the beginning of good news for XOMA related to product licensing deals. A few weeks after the Novartis announcement, XOMA earned a $3 million milestone payment from a 2015 licensing arrangement with Nanotherapeutics for an experimental anti-botulism drug. In October, XOMA announced three additional licensing deals for its phage display libraries for antibody discovery. And on Dec. 7, the biotech reported that it had licensed XOMA 358, an experimental drug targeting treatment of hypoglycemia, to small drugmaker Rezolute.
Marinus Pharmaceuticals
The award for best-performing biotech stock of all in 2017 goes to Marinus Pharmaceuticals. Marinus' stock skyrocketed over 700%, edging out XOMA. Marinus' market cap was below $20 million at the beginning of the year. The biotech's valuation now is close to $330 million.
It's been an eventful year for Marinus. In June, the FDA granted orphan-drug designation to ganaxolone as a treatment for CDKL5 Disorder, a rare genetic type of epilepsy. Marinus announced top-line data from its phase 2 study of the drug in September. Those results were overwhelmingly positive. On Dec. 13, Marinus was added to the Nasdaq Biotechnology Index, which meant that several exchange-traded funds were buying the company's shares.
2018 should be another huge year for Marinus. The biotech expects to begin a pivotal clinical study of ganaxolone in treating CDKL5 Disorder next year. Marinus should also report results from a couple of phase 2 studies of the drug in 2018, one in treating postpartum depression and the other in treating refractory status epilepticus.
Honorable mentions
While Marinus, XOMA, and Sangamo were the best-performing biotech stocks this year, a couple of other biotechs deserve honorable mentions. Alnylam Pharmaceuticals (NASDAQ: ALNY) stock more than tripled in 2017, while Nektar Therapeutics (NASDAQ: NKTR) stock soared more than 350%. In a sense, Alnylam's and Nektar's gains are even more impressive than the top three biotech stocks, since the two companies started out the year with market caps of $1.8 billion and $3.2 billion, respectively.
Alnylam's share price had more than doubled by August, but the really big news for the biotech came in September. On Sept. 20, the company reported positive results from a late-stage study of RNAi drug patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy. Alnylam is now pursuing U.S. and regulatory approval for the drug.
Nektar's story is similar. The stock posted solid gains throughout most of 2017, but really took off in November. Nektar announced positive clinical results in November for two pipeline candidates -- mu-opioid receptor agonist NKTR-181 and cancer drug NKTR-214.
With their relatively large market caps, it will probably be more difficult for Alnylam and Nektar to achieve similar gains in 2018. However, don't be surprised if all of these biotech stocks -- the three winners and the two honorable mentions -- perform well next year.
“A pig farmer who was treated with IL-12/Keytruda after his melanoma lesions had already failed to respond to Keytruda, Opdivo, & Yervoy. 3 mths after the combo treatment, the patient’s tumors stabilized, then started to shrink..followed for 2 yrs, still in PR.”
Found the screenshot. iHub doesn't allow ST links so you'll have to replace the 'g' with an 'h' below after copying it into your browser.
gttps://stocktwits.com/Vburke1999/message/96811028
Just want to make it clear that I do not think of this anecdotal post as a strong indicator of the upcoming data.
I wasn’t aware of IV being tested for the first time in PPD. Thanks for sharing.
BTW just came across the below link. Looks good. Hoping data is the same.
MAGNOLIA Postpartum Depression Study
https://magnoliastudy.com/
Also for what it’s worth, this Facebook post was circulating on StockTwits few months ago. I can’t find the original post (with FB screenshot) but had the text forwarded by someone.
Tiffany Seaman
September 14 at 11:00pm
I had an appointment this afternoon. While in the waiting room, a young woman was having moments of crying as she filled out paperwork. I couldn't help but feel that she was in a place that I have been before. My heart hurt for her. Many people asked if she needed any help, but she just responded that she was okay. We just never know what people are going through.
I know I have posted about this before, but my doctor shared with me that their office is one of 10 sites nationally to be offering the drug trial for a new drug that is specifically designed for treating postpartum depression (and is supposed to have immediate effects), and that it is having amazing results (Ganaxolone). Please share if you know of anyone needing help!
Jnana launches with $50M to spotlight an unexplored protein territory
https://endpts.com/jnana-launches-with-50m-to-spotlight-an-unexplored-protein-territory/
Boston startup Jnana Therapeutics is launching with a $50 million Series A round to develop a drug discovery platform it hopes will push the envelope of protein science.
The company is looking to discover oncology targets (among other disease areas) by investigating how proteins known as SLC transporters function across cells and in tissues.
“These proteins are amongst the oldest in biology, and afford avenues to address virtually all major diseases,” said the company’s CEO Amir Nashat in a statement. “After 50 years of drug development, only a handful of SLCs have been harnessed to treat disease, leaving over 400 members unstudied.”
In the past, these proteins have been studied one by one. Jnana’s edge is that its tech can sift through the whole body of SLC transporters and develop small-molecule drugs against them.
“Our proprietary chemistry and biology platform allows us to systematically advance medicines based on the biology of these ancient information gathering systems,” said Nashat, who is also managing partner at Polaris Partners, one of Jnana’s backers.
Seed funders Polaris Partners and Avalon Ventures were joined in the Series A syndicate by Versant Ventures, AbbVie Ventures, and Pfizer R&D Innovate.
5 Biotech Buyout Candidates for 2018
https://www.fool.com/investing/2017/12/13/3-biotech-buyout-candidates-for-2018.aspx
-The Republican-inspired U.S. tax reform could spur an acquisition bonanza in biotech next year. Here's a look at five companies that might be prime targets.
With the Republican tax plan steaming through both houses of Congress, 2018 is shaping up to be a banner year in terms of consolidation for the biotechnology industry. Biopharmaceutical heavyweights Amgen (NASDAQ:AMGN), Gilead Sciences (NASDAQ:GILD), Johnson & Johnson (NYSE:JNJ), and Pfizer (NYSE:PFE), after all, each have stately sums of cash overseas that could become available if this proposed tax reform becomes reality.
Each of these cash-rich pharma giants also has a glaring weakness in their underlying product portfolio that could be shored up by additional M&A activity. Amgen is dealing with the loss of exclusivity for its blood-conditioning medicines Neupogen and Epogen, Gilead Sciences is experiencing a rapid decline in its hepatitis C franchise and facing new threats to its dominance in HIV, J&J's blockbuster arthritis drug Remicade is battling generic competition now, and Pfizer's top-selling pain medication, Lyrica, is near the end of its patent life as well.
With the Republican tax bill and the strong demand for new growth products in mind, I think Biogen (NASDAQ:BIIB), BioMarin (NASDAQ:BMRN), bluebird bio (NASDAQ:BLUE), Geron Corp. (NASDAQ:GERN), and Sage Therapeutics (NASDAQ:SAGE) are all likely to get taken out in 2018. Here's why.
Biogen's a quick fix for an ailing top-line
While Biogen doesn't have the type of robust clinical pipeline that offers deep value to potential suitors, the biotech's market-leading multiple sclerosis franchise, and its new spinal muscular atrophy drug, Spinraza, are two pretty good reasons to put it on a list of potential buyout candidates.
Taken together, these two growth drivers would make Biogen an immediately accretive acquisition in most buyout scenarios, after all. Biogen's market cap of $69 billion is also arguably the perfect size for, say, a Pfizer that needs to bulk up first before eventually splitting into two separate companies -- a legacy products business, and an innovative healthcare company. Pfizer also doesn't necessarily need an influx of clinical candidates to make a deal worthwhile, making Biogen an ideal candidate.
Will 2018 be BioMarin's last year?
BioMarin is the perpetual buyout candidate -- only to never get bought out. The underlying reason is that this rare-drug specialist, that now has six products on the market, sports a rather weighty valuation, to put it mildly. With a price-to-sales ratio of 12.33, BioMarin is, in fact, one of the most expensive biotech stocks in the world today.
However, BioMarin's top-notch clinical pipeline that's advancing high-value candidates for indications such as dwarfism and hemophilia A could tip the balance in favor of a buyout in a cash-rich environment. Biopharma giants like J&J, after all, have shown a willingness to pay top dollar for promising acquisitions when they come with longer-term growth prospects. And BioMarin's present market cap of less than $16 billion wouldn't be much of a barrier for any of the aforementioned companies -- that is, if they wanted a top-tier orphan drug product portfolio and clinical pipeline.
Bluebird is a top buyout candidate in 2018
Of these five buyout candidates, I think Bluebird is the probably the strongest lock to get taken out in 2018. The company's newly unveiled CAR-T data in multiple myeloma, after all, is simply too compelling to ignore for companies like J&J, or Bluebird partner Celgene Corporation. Long story short, bluebird's CAR-T candidate, bb2121, produced response levels in advanced multiple myeloma patients that could catapult this therapy to megablockbuster status in breakneck fashion.
Adding fuel to the fire, bluebird also has two other high-value, late-stage clinical candidates in the works for the treatment of transfusion-dependent beta-thalassemia and cerebral adrenoleukodystrophy, respectively. So with a price tag of only perhaps $15 billion to $18 billion in a buyout scenario, Bluebird comes across as a near surefire buyout candidate next year.
I think Celgene will be the company that brings home this top prize in biotech. With all that's at stake in multiple myeloma, after all, Celgene simply can't afford to let Bluebird get away. That being said, I wouldn't be surprised if Amgen, J&J, or Gilead also made a play for bluebird in 2018.
Geron will either sink or swim in 2018
Geron, a small-cap oncology company, is heading into 2018 with its very existence at stake. Bottom line: We should know whether the biotech's telomerase inhibitor, imetelstat, is a viable blood cancer drug by the end of next year. If not, there's no realistic path for Geron to continue as a going concern.
The bright side is that Geron, and its development partner J&J, recently released some rather intriguing early-stage data for imetelstat in myelodysplastic syndromes (MDS) that support their thesis in this rare bone marrow disorder, as well as some other non-clinical data suggesting a broader utility in blood cancers in general.
The short version of the story is that imetelstat may turn out be an important new drug for patients with a specific form of MDS, a treatment of last resort for patients with advanced myelofibrosis (MF), and a backbone therapy for other blood-based cancers. J&J, by virtue of its insider status, is the obvious potential suitor for Geron.
What's the likelihood of a buyout? Provided imetelstat's overall efficacy profile in advanced MF are compelling enough to warrant further development, I'd imagine that J&J would begin negotiations, and that Geron would listen intently.
Geron CEO John Scarlett, after all, has a history of selling promising experimental-stage biotech companies well before their lead candidates begin to realize their commercial potential. And at 66 years of age, he probably isn't planning on seeing imetelstat through another five to perhaps 10 years of clinical development. So my view is that Geron is either going bankrupt or getting bought out in 2018.
Sage is building an extremely attractive depression franchise
After hitting on both a late-stage trial for postpartum depression with its lead clinical candidate brexanolone, and a mid-stage trial for major depressive disorder with SAGE-217 this year, Sage Therapeutics is almost certainly on the radar of most Big Pharmas at this point. The anti-depression drug market, after all, is worth tens of billions in annual sales.
The big ticket item is that SAGE-217 appears more than capable of producing approvable results in a forthcoming late-stage trial, based on its outstanding mid-stage results, where the drug proved to be both highly efficacious and well tolerated. In short, SAGE-217 may have what it takes to be a franchise-level anti-depression drug, which is a highly sought-after commodity in the pharmaceutical industry.
Sage's $6.7 billion market cap is also no impediment whatsoever for top dogs like Pfizer that already have a global footprint in the psych medication space. The only question, therefore, is whether a suitor wants to take the risk to wait for SAGE-217's pivotal stage data. Another company, after all, could scoop it up in the interim.
Are any of these stocks worth buying right now?
As buyout scenarios often fail to materialize, you should arguably never buy a stock simply for its potential as an acquisition target. That said, I'd be surprised if Bluebird or Sage made it all the way through 2018 without at least one offer. Both of these companies are developing products that could turn out to be disruptive forces in their respective indications, after all.
Biogen and BioMarin, on the other hand, are probably only strong acquisition targets in the event that the Republican tax bill results in a sizable cash influx for the top players in the field. And Geron will have to prove itself with additional imetelstat data to become a bona fide takeover candidate.
So while I do own a small position in Geron, I think Bluebird and Sage are the two most compelling buys of the bunch. These two emerging drugmakers should produce strong returns on capital regardless of their fate as buyout candidates, making them suitable growth vehicles for most types of investors.
I agree re: the chart! On the block trade however, it appears it could have been due to rebalancing of the Nasdaq Biotech Index. Few others that were added to the NBI had similar block trades apparently.
https://twitter.com/Slambucket/status/942188039014461440
It's a risky bet for sure and as a result I have a small position only. I'm only placing a small bet with the big guys. I am assuming (and hoping) Bain isn't advertising Marinus on their website for a small and quick flip only. Just trying some luck here.
https://www.baincapital.com/businesses/scaling-innovation-life-sciences#portfolio
BTW re: the block purchase, it might actually be due to rebalancing of the Nasdaq Biotech Index. Few other stocks that were added to the NBI had similar block trades apparently.
https://twitter.com/Slambucket/status/942188039014461440
Regards
MRNS—A $6.5M (827K shares) block trade at close yesterday
Could have been an institution from the 10M share @ $3.75 raise on Sep 14 taking advantage of the 3 month lockup expiring on Dec 14. On the flip side, someone likes the prospects of the upcoming PPD readout, IMO.
Makes sense and I agree. If I had a $3.75 entry and the stock was trading at $9.87 I would take some profits as well. Just curious as to why someone would sell at LOD at close and not in smaller chunks throughout the day at a higher average. Anyway, this 877k lot was only a small fraction of the $3.75 offering. Looking forward to January.
You didn’t explicitly say that, but you said people are better off giving their money to foundations (than investing in ONCS) who give that money to clinicians who don’t have vested interests. Weren’t you implying ONCS clinicians have vested interest and hence the data can’t be trusted? If not, what were you implying with that post? Thanks.
I was responding to your conflict of interest and legitimacy concerns (in this message chain). Do you think ONCS UCSF data may be illegitimate?
All scientific papers/publications have stock ownership disclosure in case you didn’t know. Adil Daud for instance discloses ONCS stock ownership in all papers/presentations. Algazi, the UCSF (ONCS) trial lead, does not disclose any ownership. Do you have any proof that he owns stock in ONCS?
Are you also suggesting the data ONCS has produced (from the UCSF trial) is illegitimate?
Why do you believe clinicians at ONCS don’t know what they are doing?
Also Algazi from UCSF does not have stock ownership in ONCS.
How exactly do foundations cure diseases? Just curious.
Why so negative doc?
827,699 share lot bought at $7.89 at close. Orchestrated drop or just someone seizing the opportunity to load up cheap?
SAGE/MRNS—Rogerm, I partially agree with your post. SAGE will without a doubt be the first to market compared to MRNS and has the advantage of (higher) oral bioavailability. While MRNS’ current Magnolia trial is IV-based, oral activity has already been established in the CDKL5 study and MRNS will also be launching the Amaryllis study that uses oral Ganaxolone in PPD (if I were them I would restructure this into an MDD study). Now it will still be a larger pill/volume than SAGE’s due to the lower oral bioavailability, but will that matter to patients if MRNS competes with SAGE on pricing or marketing? Also Ganax’s half life is longer than Sage’s so theoretically at least, Ganax. should work as a once a day pill. Without a doubt, MRNS will need more time to run the trials and prove all this but with the right financial support or under the right ownership, things can speed up very fast. By the way, regarding pill size and frequency, Gabapentin (neurontin) for example doses 600mg 3 times a day and at its peak sales were $1B a year. I think the $10B depression market has room for more than one player.
For MRNS we can view PPD as just a means to get a readout at this point. We now know that for this class of drugs positive PPD predicts MDD thanks to SAGE. MDD is now the main target for both companies due to massive market opportunity. CCD-1042 (Ganaxolone) is already being tested in MDD by Mass General. With recent SAGE developments and a positive PPD for MRNS I would expect an MRNS-sponsored MDD trial immediately, perhaps with big pharma involvement.
https://clinicaltrials.gov/ct2/show/NCT02900092?term=Ganaxolone&draw=2&rank=14
Lastly, oral Ganax has produced great results in CDKL5 compared to what is currently available. I wouldn’t say MRNS has nothing to show. I know you meant in PPD. Anyway, if this is the market perception, I would expect to see a big pop with the stock if the p2 data expected next quarter are positive. Regards.
I haven’t had a chance to look into the filings for the warrants situation. Anyway I think they will be (or have already been) exercised.
SAGE has shown that success in PPD is translatable in MDD. If MRNS provides positive PPD, I think they are going to get acquired to speed up MDD trial. They also presented positive p2 data in CDKL5 (an Orphan indication) in September. Lastly, for what it’s worth, Bain Capital Life Sciences led MRNS’ last offering at $3.75. Transactions are in Bain’s blood. If PPD is positive I think they will push the board to sell the company (15 employees only).
MRNS—Laidlaw initiates coverage with an $18 PT
https://twitter.com/ratingsnetwork/status/941284446342348800
MRNS is up more than 700% this year but still < $350M MC (SAGE at $7B). MRNS is reporting p2 PPD data in early 2018 (expecting January). Some room for market cap appreciation if the data is positive, IMO.
MRNS initiated as a Buy by Laidlaw with an $18 PT.
LAIDLAW Marinus Pharmaceuticals ( $MRNS - $8.08)
— BIOTECH (@_B_I_O_T_E_C_H_) December 14, 2017
Initiation of Coverage – Ganaxolone Quietly Gaining Steam in Significant Unmet Medical Needs PT $18.00
I see VKTX repeating MRNS story. They did an offering at $3.75 and started trading above $4 the following day, slowly rising to where it is now before data next month. Their offering was led by Bain Capital. Viking seems to have attracted that institutional interest after MDGL data. I think the next few months will see some steady rise.
— — —
PS. I know this is frowned upon but I see MRNS seeing a big upwards correction on ppd p2 data in Jan. Their benchmark is SAGE (similar drugs, same indication) so may be worth looking into. GL all. 2018 is going to be a good year for us.
Did SAGE Therapeutics Just Break the CNS Curse?
https://www.biospace.com/article/opinion-did-sage-therapeutics-just-break-the-cns-curse-/
Here's hoping for a neuroscience renaissance
Drug development is tough at the best of times, but creating new medicines for central nervous system (CNS) disorders is particularly brutal. Just look at the track record of Alzheimer's drugs, or the many failed trials in diseases ranging from Parkinson's to Lou Gehrig's disease.
In fact, one of the last truly groundbreaking innovations in neuropharmacology--the development of Prozac, the first of the SSRIs--happened 30 years ago. A lot has happened since, of course, from the development of many other SSRIs to the related SNRIs to innovations in schizophrenia and elsewhere. But CNS drugs have a dismal rate of success and, not coincidentally, the field has seen a slowdown in new research. Many major drug companies, including AstraZeneca, GlaxoSmithKline, Merck, Novartis, Pfizer and Sanofi, have winnowed or entirely shuttered their neuroscience programs.
Around half of patients with depression or anxiety aren't adequately addressed by existing drugs. This is just one of the reason why the World Health Organization has labeled lack of neuroscience innovation as a "crisis."
All of that is a backdrop to recent news from SAGE Therapeutics. The company has had quite a year, reporting in November that its drug brexanolone was effective in rapidly relieving the symptoms of post-partum depression in the majority of new mothers who were treated. And just this month, there was potentially more important news: SAGE-217 quickly put 64% of patients who took it into remission from major depression disorder. And both drugs work with an entirely different mechanisms than existing antidepressants.
This is obviously great news for SAGE, its investors, and patients who may ultimately be helped by these drugs. But it could also open up a new area of research and, with luck, bring about a small renaissance in neuroscience.
When No One Else Was Looking…
In some respects, SAGE is a beneficiary if the industry's growing disenchantment with CNS research. The hints that both brexanolone and SAGE-217 had the potential to address depression have been around for years, but few companies were interested in pursuing that path.
That's because both of these drugs work by modulating GABA and the GABA-glutamate balance in the brain. They aren't unique in this regard; the benzodiazapines (Xanax, etc.) also work through a GABAergic mechanism. But it is exactly because benzos don't work in depression that many researchers have dropped this line of inquiry.
A notable exception is Bernhard Luscher from Pennsylvania State University, who got some attention last year for a study showing that increasing GABA production appeared to improve symptoms of depression in mice. His work follows on older research (including his own) indicating that other GABAergic or glutamate-blocking drugs may have antidepressant effects. That includes Lamictal (lamotrigine), which was originally approved as an anticonvulsant in 1994 but got a label for bipolar disorder in 2003 and has since become a first-line treatment. It also, intriguingly, includes Rilutek (riluzole). This glutamate blocker is only approved for amyotrophic lateral sclerosis, but has been studied in depression with some promising results. (You may notice the common link of these drugs working in both convulsions and mood disorders--which is part of the reason SAGE is also pursuing its drugs in epilepsy, Parkinson's, and tremor). But at least one theory is that SSRIs work by indirectly affecting the GABA-glutamate balance, explaining why it takes so long for them to work. SAGE-217, in contrast, took effect very quickly.
Certainly SAGE's drug isn't perfect--its effects appeared to lessen over time, and many patients reported some of the sleepiness associated with benzodiapines. There's little information on whether it may also have some of the habit-forming effects associated with those drugs. These offer areas for potential improvement that may now attract a good deal more research. The bottom line is that SAGE may have a long headstart in a promising area because everyone else was looking the other way. You can bet that more companies will start to pay attention to the GABA literature now.
2017 has been about bright new things in bioscience--gene therapy, CRISPR, RNAi, CAR-T, and more. But one of the most exciting new frontiers in CNS research over the coming years may turn out to be something old.