Assembling my biofolio...
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ACRS -
Well to alleviate some of your worries they already reported results.
yes I've heard other JAK's for Alopecia but I don't know where any of the programs stand. It seems they are working on both oral and Topical formulations which I find interesting. I own CLVLY which is developing a drug for amongst other things Vitiligo and have come across some findings that JAK's have potential here. Its a disease that really could use better options (a lot of unapproved things, makeup and light therapy seem to be what is used no approved drug).
ACRS -
Their lead program is Seborrheic Keratosis (where most of the value is too) but the assets that caught my eye are actually their JAK programs for Alopecia and Vitiligo. Anyone here have an opinion on the company?
I've noticed that this is your first question about many drug companies mentioned on this board—e.g. #msg-127822905 is another instance. Not saying this is a bad thing!
RARX -
…We are developing our lead product candidate, RA101495,…a subcutaneous…injection,…for the treatment of paroxysmal nocturnal hemoglobinuria, or PNH. PNH is a rare, chronic, life-threatening, blood disorder where red blood cells are mistakenly attacked and destroyed by the complement system. We expect to initiate our Phase 2 clinical program for RA101495 in PNH patients in the first quarter of 2017 and release data in the second half of 2017.
PTGX -
PTGS (Protagonist Therapeutics) IPOs 8.63M* shares @$12.00
This is still in early clinical trials but results look promising. Has implications on HIF companies (FGEN AKBA etc), if this ever gets approved.
Interesting that somehow this is not showing up in Aevi's pipeline (former Medgenics - MDGN).
GNRO - P2B MS update
GeNeuro Provides Update on GNbAC1 Multiple Sclerosis Clinical Program
? Enrollment completed for CHANGE-MS study ahead of schedule; 6-month top-line results now expected in early 4Q2017
? ANGEL-MS study launched, dosing of first patient expected in April 2017 ? U.S. study in secondary progressive MS patients planned for 2H 2017
Geneva, Switzerland, 3 January 2017 – GeNeuro (Euronext Paris: CH0308403085 – GNRO), a biopharmaceutical company developing new treatments for autoimmune diseases, including multiple sclerosis (MS), today provided an update on GNbAC1, its multiple sclerosis drug candidate. GNbAC1, the first drug candidate directly targeting a potential cause of MS, is a monoclonal antibody designed to neutralise MSRV- Env, a pathogenic protein which has been associated with both the inflammatory and neurodegenerative components of the disease.
“Completing enrollment in CHANGE-MS several months sooner than previously anticipated is a significant achievement for our GNbAC1 development program and, most importantly, accelerates the timeline to potentially provide MS patients with a new and effective therapeutic option,” said Jesús Martin-Garcia, Chief Executive Officer at GeNeuro.
Full enrollment of the CHANGE-MS Phase 2b study of GNbAC1 has been completed, several months ahead of schedule, and is indicative of strong interest from physicians in this pioneering new treatment for MS. Based on this accelerated timeline, GeNeuro now expects the availability of top-line results from this study early in the fourth quarter of 2017, versus the previous estimate of by year-end 2017. The double-blind, placebo-controlled study, CHANGE-MS (Clinical trial assessing the HERV-W Env Antagonist GNbAC1 for Efficacy in Multiple Sclerosis), is evaluating the efficacy of GNbAC1 in reducing the number of new inflammatory lesions as well as measures of neurodegeneration on brain MRI in patients with remitting relapsing multiple sclerosis (RRMS). The CHANGE-MS Phase 2b study is fully funded through GeNeuro’s €362.5 million1 partnership with Servier, which was signed in 2014.
The recently announced ANGEL-MS (Assessing the HERV-W Env ANtagonist GNbAC1 for Evaluation in an open label Long-term Safety Study in patients with Multiple Sclerosis) study has now been launched, offering all patients having completed the Phase IIb CHANGE-MS study the opportunity to continue their treatment for an additional two years, providing additional efficacy and tolerance data. The first patient completing 12 months in CHANGE-MS, eligible to continuing treatment through ANGEL-MS, is expected to enrol in April 2017. Like CHANGE-MS, the ANGEL-MS study will be fully funded by Servier.
GeNeuro continues to work on its submission package in preparation for clinical trials in the U.S. A Phase 2 study in secondary progressive MS patients, a patient population distinct from RRMS patients, is now anticipated to begin during the second half of 2017. This study will evaluate the effect of repeated doses of GNbAC1 on safety and biomarkers of microglial activation, remyelination and neuroprotection.
“2016 has been a transformative year for GeNeuro, with the success of our IPO, the full recruitment of CHANGE- MS and the further strengthening of our team in order to execute additional clinical trials in 2017. We look forward to initiating these new studies and to the availability of the interim 6-month results from CHANGE-MS at the beginning of the fourth quarter of 2017,” Mr. Martin-Garcia added.
KALV -
KalVista has developed a proprietary portfolio of small molecule plasma kallikrein inhibitors targeting hereditary angioedema (HAE) and diabetic macular edema (DME). The Company’s portfolio of orally delivered plasma kallikrein inhibitors for HAE is expected to commence Phase 1 clinical trials in the third quarter of 2016. KalVista’s most advanced program, an intravitreally administered plasma kallikrein inhibitor for DME, has successfully completed Phase 1 clinical trials in DME patients…
CLLS—NYT feature on Calyxt:
www.nytimes.com/2017/01/09/science/genetically-edited-foods-crispr.html
Quote:
Dr. Mulligan of Harvard said he was not sure that people would see much difference between gene-edited and genetically modified. “The objection that people have is a more visceral and vague objection to messing with DNA,” he said. “It’s hard to see that the public would see the difference.”
As previously posted, I agree with the above, and I think CLLS is hoodwinking investors into assuming that gene-edited crops will be viewed by the general public as fundamentally different from GMOs.
SMMT +20% on no news I’m aware of. SRPT coattails?
SELB has generated no discussion whatsoever on this board since its IPO. I haven't even noticed a press release, although there must have been some.
WVE IPOs 7.33M* shares @$16.00:
finance.yahoo.com/news/wave-life-sciences-announces-pricing-113000429.html
Looks like the underwriters held off on the start of trading due to the bearish biotech market today.
See #msg-118310217 and #msg-118331163 (S-1 excerpt) for background.
*Assuming exercise of underwriter’s option.
IPF (FGEN/GBT) -
re 3019
The pancreatic data can go either way of course, but that is a near term event and they have been increasing the number of centers recruiting FWIW. I still view it as a long shot but it could have legs
re IPF substudy i presume you mean the data w SOC. As long as there is safety there i wouldn't worry about numbers and duration of exposure wrt efficacy bc if there is efficacy as monotherapy vs placebo, based on a pretty unique MOA there would be very good reason to think combo would work out so i think positive efficacy as monotherapy vs placebo would be a strongly positive event for FGEN
CLLS -
I am a little worried that I sound like a broken record, but I would suggest that, broadly, the risk to CLLS strategy is that their response rate and/or mortality rate will get substantively worse when they roll it out more broadly (i.e. to more trial centers, and even more patients within the same centers - so less time/care with each patient). I don't track CLLS routinely, but for example @BiotechBbqBeats notes after the first 2 pts, they have only 2 of 5 patients getting to MRD negative. And another way to think about this is to compare these early results to the first data from auto CAR-T.
More narrowly, again as I've noted before, the problem that CLLS hates to acknowledge is that they need to lympho-deplete MUCH more heavily than auto CAR-T. That means much more expense and likely higher mortality in order to get the same response. But I wouldn't expect this to really show up until they start rolling out to a lot of trial centers.
Finally, FWIW, I think they are really more of a competitor to BLCM than JUNO/KITE because, like BLCM, they require the lympho-depletion.
CCXI -
Not recently - I am more than 18 months stale. That said, in a *quick* look through:
a) The results of 168 in ANCA continue to look very good - e.g. beat SOC of steroids. It is reasonable that 168 now appears to be their primary focus going forward (i.e. expansion into other indications like aHUS).
b) I have no direct opinion on the chances of Ph2 success in aHUS, but will comment that many of the inflammatory diseases (like ANCA and aHUS) do seem to have a lot in common.
c) Bizarrely they don't seem to kill old, failed, drugs - e.g. their website still lists Vercirnon in Crohns and 140 in Diabetic Nephropathy.
d) The press release on their pancreatic cancer results don't look particularly encouraging FWIW. 30% ORR when paired with an entire regimen of chemo... .
If I get time I'd research the competition (i.e. other C5 inhibitors) and the market sizes.
(FGEN)
>>The retinal stuff may possibly be a Japanese regulatory thing.
I can confirm my supposition was correct. According to the company, this was the same request made to GSK and viewed as simply routine.
QURE -
Nothing too surprising given the evolving GT space. I actually think the market may be too harsh today especially since Hem B program has a good chance to be first to market.
ENTA -
The study also included those who were not cured with previous treatment with sofosbuvir (SOF) plus ribavirin (RBV) or with interferon (IFN) plus RBV, with or without SOF (44 patients, 42 percent).
Agree that there would likely be a short term boost - because anyone and everyone will get on the market and be able to charge through the roof (e.g. SRPT). But that brings medical cost controls all the sooner - and to all players. I.e. the boost will be temporary, then we hit massive implosion as the country realizes we're paying a fortune and 70% of the new drugs are actually worse than useless. (that said, as I noted earlier, it might be best overall if we went back to 50's standards in order to reach sanity again the quickest. But from about year 3 or 4 I'd expect the market to be largely unplayable - and with a looming disaster)
Note: I believe the difference in perspectives here is that your investment style is to try to anticipate the buzz. I have no inherent objection to this, but think it is ultimately very bad for the market and very bad for the country to suppose this ends well if the focus moves from science to buzz (i.e. marketing and politics). (Note that I have been surprised to watch multiple biotech investors morph from a science focus to a buzz focus during the big boom.)
Are you ready for ARQL's Tiva Ph3 NCC results in the next 2-3 months?
Suggest you aren't understanding. Without rigorous data it is, literally, impossible to sort it out. As a way to think about this, you yourself do not want to hold companies through their phase 3 binaries because 90% of phase 2s are completely inadequate to determine whether the drug is clinically useful. And the 'reformers' essentially want to move to something much much closer to phase 2 'proof' as adequate for approval.
(Examples of such proof are: single arm comparison to historicals (do you consider such phase 2s adequately dispositive to hold through a phase 3?), biomarkers (do you consider such ph 2s adequate to hold through a clinical endpoint ph3?)). And you undoubtedly are better at sussing out real phase 2 efficacy than 90% of MDs.
PS Sorry for being a little pointed, but really don't think people are thinking this through. Over the Xmas break I'll screed on some related topics because there are some things we could probably do to improve the process. But only around the edges. Essentially we are already fairly close to the Shannon's Limit of biomedical research (Shannon's Limit is a about how much information you can get through a system).
FWIW I couldn't agree more. I tweeted as much several weeks ago to @VinayPrasad82 @matthewherper think u'll find high %age long term inv mostly agree w ur positions. Science is an edge. Chaos is bad.
Further thought is that, again as I tweeted (to @matthewherper in this case), I hope if we are going to swing to a too permissive FDA, that we go whole hog back to the 1950s. I think that likely has the least long term damage because it will crash and burn quickly (quickly is all relative - perhaps 10 years before it becomes apparent to the public that something is very very wrong)
Neither had I; however, CGEN must be onto something big because the company is a constituent of $LCINDX :- )
CGEN is now an I-O company with a checkpoint inhibitor called COM701, slated to start phase-1 in 2017
ACHN -
To date in the MAD study, ACH-4471 has been generally well tolerated across three dose cohorts (200 mg, 500 mg or 800 mg given every 12 hours) with no treatment-related SAEs reported. Two cases of self-limited, ALT elevations (Grade 3 and 4) were observed post-treatment in the mid- and high dose groups, respectively, with neither subject exhibiting signs or symptoms of hepatic decompensation. Both subjects’ ALT levels normalized without intervention during follow up.
FGEN -
The retinal stuff may possibly be a Japanese regulatory thing. There are some Japanese studies of EPO and retinal issues
TRIL -
re dosing - they obviously have a dose that is active. it would be nice to know if serum concentrations are in a range where they expect tumor reductions based on preclinical but it's hard to translate from mouse anyway
the PFS in the 2 patients is nice but not POC. I suppose the long period in the DLBCL pt, which is an aggressive form of lymphoma that is normally fast growing, is something. FL is indolent so frankly totally meaningless to have no progression
I do think this is an overreaction. What would you think if forty seven inc in phase 1 didn't have anemia at all? It frankly wouldn't be a good sign bc then you question the drug's activity altogether. The fact platelets are also sensitive to Cd47 inhibition and they didn't se any makes me think TRIL's drug is more active. So if I were to bet on either of these horses it would be TRIL
FGEN -
the company can unblind the non dialysis study in 8 weeks so the limiting factor for reporting is the 26 week dialysis study since they have guided they want to report the data together.
Reasons to believe that uniQure is groomed to be acquired.
1) company is going through strategic review process
2) there are no plans to hire permanent CEO
3) new chairman of BOD is from Forbion (largest stake holder)
4) Forbion is known for M&A exists (Dezima & Akarna latest examples)
CCXI's +6% move today—not bad for mouse data.
CCXI - CCX872/NASH
http://ir.chemocentryx.com/releasedetail.cfm?ReleaseID=994124
ChemoCentryx's CCR2 Inhibitor CCX872 Shown to Reduce Liver Fibrosis in NASH Models
-- CCX872 reduced hepatic inflammation, steatosis, and scarring in models of non-alcoholic steatohepatitis (NASH) --
-- Findings reported at the American College of Gastroenterology (ACG) 2016 Annual Meeting --
MOUNTAIN VIEW, Calif., Oct. 18, 2016 (GLOBE NEWSWIRE) -- ChemoCentryx, Inc., (Nasdaq:CCXI) today announced the presentation of data from in vivo models of non-alcoholic steatohepatitis (NASH) with CCX872, a selective orally administered inhibitor of the chemokine receptor known as CCR2, at the American College of Gastroenterology (ACG) 2016 Annual Meeting, being held October 14-19, 2016.
The Company's CCR2 inhibitor CCX872 demonstrated significant reductions in liver fibrosis when compared to either placebo or a separate compound which is a dual inhibitor of the chemokine receptors CCR2 and CCR5 currently in clinical development by another party. The data suggest a potential application of CCX872 for the treatment of patients with NASH, a severe type of non-alcoholic fatty liver disease caused by chronic inflammation that can lead to fibrosis (scarring) of the liver. NASH affects three to five percent of the U.S. population.
"Liver fibrosis is a critical and particularly difficult to treat feature of NASH, with considerable impact on long-term mortality and liver transplantation. We are therefore very pleased to see the therapeutic effect of our CCR2 inhibitor CCX872 improving liver fibrosis in NASH disease models," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "NASH is the fastest growing cause of liver cancer and liver transplant in the U.S. and other parts of the world; one for which there are no currently approved treatments. We look forward to sharing additional data from our efforts in this disease."
Researchers believe that inhibiting CCR2 has several therapeutic benefits, including decreasing inflammatory macrophage infiltration into the liver, thus reducing hepatic inflammation and fibrosis. The data are being presented today in a poster titled "Reduction of Liver Fibrosis by CCR2 Antagonist CCX872 in Murine Models of NASH" (Abstract P2017, October 18, 10:30 a.m. to 4:00 p.m. PT) and are summarized as follows:
CCX872 is a potent and highly selective inhibitor of CCR2, with a marked capacity to reduce the migration of the cells responsible for liver inflammation central to fibrosis in NASH.
Two murine models of NASH were used to determine the efficacy of CCX872 in reducing fibrosis. One model used a high-fat diet (HFD) and the other model used a methionine-choline deficient diet (MCD), both of which are known to induce NASH.
In both models, treatment with CCX872 achieved a statistically significant reduction in liver fibrosis as compared to placebo control.
In the HFD model, there were statistically significant reductions in serum levels of alanine transaminase (ALT) and aspartate transaminase (AST), two key measures of liver injury, as well as reductions in the accumulation of inflammatory macrophages (which are derived from CCR2 precursor cells), and triglyceride and collagen content in the liver. Insulin sensitivity and glucose tolerance were also improved in the CCX872 treated group.
In the MCD model, CCX872 achieved statistically significant reductions in serum ALT concentrations and liver and collagen content as compared to placebo control.
In a side-by-side comparison in the murine MCD model, CCX872 was more efficacious than the CCR2/CCR5 dual inhibitor, which is elsewhere in clinical development, in reducing liver fibrosis.
Overall treatment with selective CCR2 inhibitor CCX872 improved liver fibrosis in NASH pre-clinical models.
(ADXS)—Current issue of Nature has feature article on cancer vaccines (behind paywall):
SMMT - WMS PR
http://hsprod.investis.com/servlet/HsPublic?context=ir.access&ir_option=RNS_NEWS&ir_client_id=4747&item=2655236829216768
SUMMIT THERAPEUTICS PRESENTS PRECLINICAL DMD DATA AT THE 21ST INTERNATIONAL CONGRESS OF THE WORLD MUSCLE SOCIETY
Summit reports on development of new, automated biomarker tools to evaluate disease status of muscle biopsies
Oxford, UK, 6 October 2016 - Summit Therapeutics plc (AIM: SUMM, NASDAQ: SMMT), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, announces the presentation of preclinical data at the 21st International Congress of the World Muscle Society, which is taking place in Granada, Spain from 4-8 October 2016. The data include further findings from Summit's programme to develop tools to measure biomarkers of muscle health for use in patient clinical trials evaluating utrophin modulator therapies.
"Our approach to DMD aims to maintain production of utrophin, a protein typically found in young and repairing muscle fibres, so that it can replace the missing dystrophin protein in mature muscle fibres," commented Dr Ralf Rosskamp, Chief Medical Officer of Summit. "The development of new, fully automated biomarker tools capable of evaluating the disease status of muscle biopsies will play an important role in clinical trials, including our ongoing PhaseOut DMD trial. In this trial, we seek to establish the potential of utrophin modulation, including our most advanced candidate ezutromid, as an effective treatment for all patients with this disease."
The findings reported at WMS highlight progress in the development of fully automated quantification techniques for muscle biopsies capable of measuring structural proteins such as utrophin and biomarkers of muscle regeneration. The techniques described are immunohistochemical based assays and digital tissue image analysis tools that can robustly measure utrophin, beta-dystroglycan (a member of the dystrophin protein complex) and developmental myosin (a biomarker of muscle fibre maturity) in individual muscle fibres across a whole biopsy section.
Patients with DMD have higher levels of immature, regenerating muscle fibres because the absence of dystrophin leads to a continual cycle of muscle fibre damage and repair. By seeking to replace missing dystrophin with utrophin, utrophin modulators such as ezutromid aim to maintain the integrity of muscle fibres and allow them to mature. The automated approaches described at WMS make it feasible to look at associations between fibre maturity and integrity and utrophin expression in individual fibres. This provides a basis to distinguish new and beneficial expression of utrophin caused by a utrophin modulating drug, from the expression of utrophin seen generally during fibre regeneration. This is an important step in understanding and characterising the activity of these drugs.
This biomarker research is being conducted in partnership with Flagship Biosciences using their computational Tissue Analysis (cTA(TM)) approach to accelerate precise muscle fibre biomarker quantification. This work builds on a recently published manual quantification approach that was developed by Summit and researchers at the Institute of Child Health, London with financial support from the charity Joining Jack. Further validation work to optimise the Flagship Biosciences' cTA(TM) approach is ongoing; it is expected the tools will be used in Summit's Phase 2 proof of concept trial of ezutromid, PhaseOut DMD.
Additional presentations at WMS 2016 reported preclinical data highlighting the potential of utrophin modulation as a disease-modifying treatment for all patients with DMD, regardless of the underlying fault in the dystrophin gene. This research was conducted at the University of Oxford under the UtroDMD alliance.
Copies of the presentations given at WMS 2016 are available from the 'Programmes' section of Summit's website, www.summitplc.com.
About PhaseOut DMD
PhaseOut DMD aims to provide proof of concept for ezutromid and utrophin modulation by measuring muscle fat infiltration, as well as by measuring utrophin protein and muscle fibre regeneration in muscle biopsies. The primary endpoint of the open-label trial is the change from baseline in magnetic resonance imaging parameters related to fat infiltration and inflammation of the leg muscles. Exploratory endpoints include the six-minute walk distance, the North Star Ambulatory Assessment and patient reported outcomes. PhaseOut DMD is a 48-week open-label trial expected to enrol up to 40 boys ranging in age from their fifth to their tenth birthdays at sites in the UK and the US.
Further information is available at: www.utrophintrials.com and https://clinicaltrials.gov/ct2/show/NCT02858362.
About Utrophin Modulation in DMD
DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties. Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin has a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company's lead utrophin modulator, ezutromid, is an orally administered, small molecule. DMD is an orphan disease, and the US Food and Drug Administration ('FDA') and the European Medicines Agency have granted orphan drug status to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the FDA.
About Summit Therapeutics
Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programs focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc).
SMMT -
I don't follow them that closely so I could be wrong. I had the impression the most likely event was a capital raise, followed by partnering their antibiotic with this not even being on the horizon.
As far as telegraphing deals I know you owned a company (I briefly had a small position) where the CEO was very specific about doing a deal and then meeting that timeline! In fact I think he did that on a couple of occasions....ARRY sound familiar?
Anyway congratulations on SMMT! Guess its still a high reward, high risk play.
Do you remember Pharmos (PARS)? See #msg-4911762 and #msg-17988168.
SMMT -
OK you were right but the upfront still puts it in the size that I wouldn't consider it a significant deal
OPHT > Is the negativity justified in light of the fact that REGN2176-3 and Fovista are chemically very different...
REGN's drug is an antibody..Fovista is an aptamer...
Aptamers vs antibodies...
(OPHT)—REGN’s anti-PDGFR drug—>no benefit as addend to Eylea in phase-2 AMD trial
SMMT:
Just to add to my skepticism on SMMT. It should be noted they are now on (at least) their third formulation of their Utrophin modulator. I think with their current market cap the street is sorta saying show me you have it figured out. I know that is one of the main things that keeps me from even a small position.
SRPT/DMD deals -
Perhaps you may be right about possibly a Summit deal sooner rather than later (if data good) but I am not convinced and still sticking to my thesis (CATB easily fits) smile. If it turns out their next generation stuff doesn't do much better (i.e. they don't try to for Exon 51, 45, 53) at getting to muscle/skeletal cells (cardiac pretty important in DMD) or If they do another big equity offer (in addition to selling PRV) in the next year or so I'd change my thinking.