d gaser
01 Feb 2018
Marketing Authorization Application for Kymriah Received this info just a few minutes ago there's some Interesting info about time lines and manufacturing .BE HAPPYDAVE Novartis announces NEJM publication of updated analysis from ELIANA trial showing longer-term durable remissions with Kymriah(TM) in children, young adults with r/r ALL • Analysis of 75 patients with median follow-up of more than a year demonstrated an overall remission rate of 81% • Event-free survival and overall survival at six months were 73% and 90%, with median duration of remission not reached • Kymriah was detected in patients up to 20 months, demonstrating long-term persistence • Novartis is committed to bringing Kymriah to more patients with a regulatory application currently under review by the EMA for r/r ALL and r/r DLBCL based on Novartis global clinical trial program, including ELIANA The digital press release with multimedia content can be accessed here: Basel, January 31, 2018 - Novartis today announced updated results from the pivotal ELIANA clinical trial of KymriahTM (tisagenlecleucel), formerly CTL019, in relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL) have been published in The New England Journal of Medicine (NEJM). New data include longer-term follow-up and efficacy in 75 infused patients, analysis of expansion and persistence of Kymriah, and longer-term safety. Kymriah became the first chimeric antigen receptor T (CAR-T) cell therapy to receive regulatory approval in August 2017, when it was approved by the US Food and Drug Administration (FDA) for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse, based on previous results from the ELIANA study, which was conducted in collaboration with the University of Pennsylvania (Penn) and Children's Hospital of Philadelphia (CHOP). In the analysis of 75 infused patients with three or more months of follow-up, Kymriah demonstrated an overall remission rate of 81% (95% CI: 71% - 89%). Sixty percent of patients achieved complete remission (CR) and 21% of patients achieved CR with incomplete blood count recovery (CRi), with no minimal residual disease (MRD) detected among all responding patients (95% [58/61] by day 28). Median follow-up was 13.1 months. "Kymriah, the first FDA-approved CAR-T cell therapy, has shown the potential to be a definitive therapy, providing early, deep and durable remissions for children and young adults with relapsed or refractory ALL," said Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development. "These data are a testament to our commitment at Novartis for continued CAR-T cell therapy research to bring this therapy to as many patients as possible." Among patients who achieved CR/CRi, median duration of response was not reached. Remissions were durable with six-month relapse-free survival of 80%. Event-free survival was 73% at six months (95% CI: 60%-82%) and 50% at 12 months (95% CI: 35%-64%), with median event-free survival not reached. Overall survival in the 75 infused patients was 90% (95% CI: 81%-95%) at six months, and 76% (95% CI: 63%-86%) at 12 months. Kymriah was detected in patients up to 20 months. Median persistence of Kymriah was 168 days (range: 20-617; n=60 patients with CR/CRi) at data cutoff. All responding patients demonstrated B-cell aplasia (a low number of or absent B-cells), an on-target effect of treatment with Kymriah, and most received immunoglobulin replacement per local practice. Evaluable patients with a response at day 28 had a median time to maximum expansion of 10 days (5.7-28 days; n=60), whereas six patients with no response had a median time to maximum expansion of 20 days (13-63 days). Kymriah uses the 4-1BB costimulatory domain in its chimeric antigen receptor, which has shown to enhance early cellular expansion and long-term endurance of CAR-T cells. "We continue to be encouraged by th
d gaser
01 Feb 2018
Novartis release update regarding ELIANA trial Today Novartis have released their NEJM publication updating the analysis from the ELIANA trial it shows longer-term durable remissions with Kymriah(TM) in children, and young adults with r/r ALL .to read this document please go to www.novartis.com/news/media-releases/novartis-announces-nejm-publication-updated-analysis-from-eliana-trial-showing-longer-term-durable-remissions-kymriahtm-children-young-adults-rr-allBe happy DAVE
d gaser
24 Jan 2018
Novartis accounts show 25 Kymiral centres .REM certified Novartis have issued their accounts for the past 3 months and they say they have launched 33 treatment rooms • Kymriah launch in the US progressed well in the fourth quarter. 33 treatment centers are now REMs certified, 25 of those are fully operational and we are focused on ensuring access for patientsBE HAPPYDAVE Regulatory submissions and filings (in Q4)• Kymriah (tisagenlecleucel, formerly CTL019) filed with FDA for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) who are ineligible for or relapse after autologous stem cell transplant (ASCT), and with EMA for adult patients with r/r DLBCL who are ineligible for autologous stem cell transplant and children and young adult patients aged 3 to 25 years with relapsed or refractory B cell acute lymphoblastic leukemia. FDA granted Priority Review and EMA granted accelerated assessment of the submission. • ACZ885 (canakinumab) submitted supplemental Biologics License Application and Marketing Authorization Application for cardiovascular risk reduction to the FDA and EMA. • Sandoz proposed biosimilar pegfilgrastim (Amgen's Neulasta®
was accepted for regulatory review by EMA. • Sandoz proposed biosimilar adalimumab (AbbVie's Humira® was accepted for regulatory review by FDA in January.
d gaser
24 Jan 2018
OXB recives £3 million innovation grant . The government have awarded OXB with a £3m award, in order to support our viral vector manufacturing facility. BE HAPPYDave 01/23/2018 | 01
4pm GMTLondon, UK - 23 January 2018: Oxford BioMedica plc ('Oxford BioMedica' or 'the Group') (LSE:OXB), a leading gene and cell therapy group, has announced it that has been awarded a £3 million grant by the UK's innovation agency, Innovate UK, to support the UK's efforts to produce viral vectors and ensure adequate supply to meet future demand. Reference: corresponding Innovate UK announcement here: [link] The aims of this grant are closely aligned with the current government national priorities to make the UK a global hub for manufacturing advanced therapies, which will benefit economic growth and create and retain more highly skilled employment. The grant will be used to support investment in equipment for vector development, vector manufacture, storage and analytical equipment, as well as other items that are key for the operation of vector GMP facilities. In addition, a small part of the grant will be used to support the planning for the transition of GMP suites from the use of adherent to suspension cultures.
d gaser
17 Jan 2018
Novartis gets FDA Priority Review for Kymriah™ It appears Novartis have received US FDA Priority Review for Kymriah™ ( formerly CTLIO9) for adults with r/r DLBCL and EMA accelerated assessment for children, young adults with r/r B-cell ALL and adult patients with r/r DLBCLBe HappyDave Oxford, UK - 17 January 2018: Oxford BioMedica plc ("Oxford BioMedica" or "the Group" (LSE:OXB), a leading gene and cell therapy group, today notes an announcement by Novartis that the supplemental Biologics License Application (sBLA) for Kymriah™ (tisagenlecleucel, formerly CTL019) suspension for intravenous infusion for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who are ineligible for or relapse after autologous stem cell transplant (ASCT), has been accepted by the US Food and Drug Administration (FDA) for Priority Review. In addition, the European Medicines Agency (EMA) has granted accelerated assessment to the Marketing Authorisation Application (MAA) for Kymriah for the treatment of children and young adults with r/r B-cell acute lymphoblastic leukaemia (ALL) and for adult patients with r/r DLBCL who are ineligible for ASCT. CTL019 is a novel immunocellular therapy and a one-time treatment that uses a patient's own T cells to fight cancer. Priority Review and accelerated assessment are granted to therapies which may provide a significant improvement in the safety and effectiveness of the treatment of a serious disease, and the designations are intended to expedite the standard review time. If approved by the FDA and EMA, Kymriah would represent the first chimeric antigen receptor T cell (CAR-T) therapy available for two distinct indications in non-Hodgkin lymphoma and B-cell ALL. Oxford BioMedica is the sole manufacturer of the lentiviral vector that encodes CTL019. The Group signed an agreement with Novartis in July 2017 for the commercial and clinical supply of lentiviral vectors used to generate CTL019 and other undisclosed CAR-T products, for which Oxford BioMedica could potentially receive in excess of $100m from Novartis over the next three years. As announced in October 2014, Oxford BioMedica will also receive undisclosed royalties on potential future sales of Novartis CAR-T products..
warmfront
17 Jan 2018
More good news [link]
Trouble_Two
12 Jan 2018
Re: Hope this is the one! Or NOT!!!!!!! as usual.TT
Crisisurfer
09 Jan 2018
Re: Gene Therapy Hits a Peculiar Roadblo... Hi Marcher;You raise a valid point. However the cost of intervention has to be offset against the cost of existing treatment and care, which in the NHS is often very high!An expensive one shot treatment can therefore become economically viable.In this regard Prosavin holds particular promise if it becomes the delivery system and drug of choice, as the costs of Parkinson’s care and treatment mount significantly over the patients lifetime.I think this is s cute piece of marketing by JD, reminding the market that they’re not only one of the only companies with manufacturing capacity but also are aiming to have a production scalability that makes the Best place to come when a company is ready to deliver a treatment stream to the market, especially with price point in mind.Lastly Hans if you are around, as always your opinion would be most welcome as it would no doubt be more insightful than my own. I know we had a difference in the past, but that is indeed done and dusted. It would be good to have you back if you feel so inclined?
marcher
09 Jan 2018
Re: Gene Therapy Hits a Peculiar Roadblock: ... Extract....If a company wanted to deliver a gene therapy to the lung or liver, where the organ’s “surface area is huge,” the current price could be as much as $3 million per patient — “commercially unviable,” said Mr. Dawson of Oxford BioMedica.Oxford is improving its methods, he said, and should soon be able to cut that cost to approximately $300,000 per patient. Methods are improving, Mr. Dawson said, and his expectation is that it might cost a mere $30,000 for the viruses in the future.Unlikely to be adopted by NHS any time soon then!
Chicken Lips
08 Jan 2018
Hope this is the one! Once again OXB is set to deliver on its potential, how many times have I heard that down the years. This year does look different or am I just kidding myself. The Sun Times article certainly has helped...Having more than doubled this year to 8.85p on the back of test results that showed one of its treatments could help cure a form of childhood leukaemia, it is not necessarily an obvious choice for 2018. Nonetheless, Peel Hunt and Jefferies both have a price target of 13p per share, implying more than 40% upside.Formal approval of the aforementioned leukaemia treatment could lead to a reappraisal of what some analysts reckon could be a $1bn blockbuster. .....Lets hope so!!Tends to start the year well and then fade to grey. 2018 might juts be the year!
aegian
08 Jan 2018
Gene Therapy Hits a Peculiar Roadblock: A Virus Shortage On NOV. 27, 2017 the New York Times published this article on the difficulties of producing the virus vector in cell therapy:"Biotech companies have exciting plans to introduce treatments that may be transformative, sometimes curing genetic diseases with a single treatment. And the firms are itching to test their products.But they are struggling to obtain a critical component of the therapy: the disabled viruses used to slip good genes into cells that lack them.This delivery system lies at the heart of many forms of gene therapy; without the disabled viruses, there is no treatment. But manufacturing them is costly and onerous.The genes intended to fix a defect in the body are carried into each cell by a modified virus, usually a disabled version of an adenovirus or a lentivirus. These viruses must be custom-made in specialized facilities for each treatment.Continue reading the main storyRelated Coverage In a First, Gene Therapy Halts a Fatal Brain Disease OCT. 5, 2017 Companies Rush to Develop ‘Utterly Transformative’ Gene Therapies JULY 23, 2017 F.D.A. Approves Second Gene-Altering Treatment for Cancer OCT. 18, 2017 F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000 AUG. 30, 2017 F.D.A. Speeds Review of Gene Therapies, Vowing to Target Rogue Clinics NOV. 16, 2017Few gene-therapy companies have the factories or expertise to make the viruses for use in clinical trials, where standards are exacting and comprehensive. The firms that can do it are swamped with orders and requests. ...The result is a logjam. Firms exploring new gene therapies may wait for years in line for bespoke viruses, said Dr. Jim Wilson, director of the gene therapy program at the University of Pennsylvania’s Perelman School of Medicine.Delays arise at every step. The contract virus-maker has to translate the small-scale production used for research purposes into a recipe for commercial production, where standards are extensive and documentation exhaustive. And the maker has to negotiate a contract to do all this.Those two steps can easily take a year, said John Dawson, chief executive of Oxford BioMedica. When the contractor finally is ready to start making the viruses, it can be six months to a year before they are ready — assuming there are no glitches along the way. ...If a company wanted to deliver a gene therapy to the lung or liver, where the organ’s “surface area is huge,” the current price could be as much as $3 million per patient — “commercially unviable,” said Mr. Dawson of Oxford BioMedica.Oxford is improving its methods, he said, and should soon be able to cut that cost to approximately $300,000 per patient. Methods are improving, Mr. Dawson said, and his expectation is that it might cost a mere $30,000 for the viruses in the future.The costs of testing the drug and marketing it are, of course, out of his hands."[link]
techno foxx
05 Jan 2018
virus could treat brain tumours [link]
luguvalium
05 Jan 2018
New Year effect I am always struck by New Year effect that repeatedly pushes the OXB share price in an upward direction. I have observed it over many years now. It resembles the upward push demonstrated by the housing construction sector. In the latter case, this usually takes place over the first quarter by which time the New Year effect on OXB is long since over. Traded carefully though,there is money to be made and it would appear that for shares difficult to value like OXB, reality begins to kick in after the sense of the miraculous medical advances that may unfold over the new year have long since evaporated. However, I do feel that things could be different this time and it appears I am not alone. If the market is anything to go by the holding pattern exhibited since the good news was announced in the summer suggests that there is more belief than ever before in the OXB story. All that a holding pattern of this length requires is a catalyst and that came when the prestigious Sunday Times share tips were announced last week. Looking at the chart, and taking out the seasonality factor, my guess would have been that the catalyst would have come a little later in the year. For instance there could have been an FDA approval, a positive trading statement, a new contract, a farm out, a new clinical breakthrough, or dare I say it, takeover interest. All these things and more could still happen, but only now from a higher baseline, and one that others who stayed out of the share at the wrong time must surely be rueing and are are now chasing.As a seasoned observer, the fundamentals changed for me when the constant recourse to market, on the back of rights issues for more funding to stay alive came to an end, with the signing of a new long term loan and a new long term contract. Looking at OXB in 2018, what is there not to like? Yes, it is still a long way from fulfilling its true potential but the the seeds are there for a long, prosperous and health- generating future. Which is what I wish to all those who contribute constructively to this board.
d gaser
05 Jan 2018
Re: results from the JULIET clinical tri... Have you seen how many shares some of the big boys have ? ,and it doesn't affect their price .To be honest in my view it doesn't matter how many shares you have but more a matter of who is holding them , for example If 75% of your shares are held by institutions or directors ,then only 25% are free, but if you remove say another 15% which are held by institutions that don't have to declare their holding, as they hold less than 3% , then you can make a true market with the 10% left ,and as long as those numbers remain stable,then we have nothing to fear ,as it make's our share base workable ,and I would suggest that is bourn out by the way our shares have traded over the past few years .As long as a market can be made it's fine to hold onto billions of shares ,it only becomes a danger when some thing catastrophic happens, or the institutions sell them with out finding another institution to take ,that the share may be destabilised by the addition of the extra shares market , and that then devalues them on the open market, it's only then the traded OXB market becomes unworkable,.Many people say that consolidation is better, but is it ?, when you consolidate it's usually because the company have lost the market backing ,and can not raise further cash ,and in my experience of consolidation, it only keeps a bad company going for a few years longer , before it disappears completely ,. I'm thinking companie's like Honey ,CeNeS ,Angle ,Pro, wiggins ,tadpole , Henderson, and many others ,who all lost the markets backing ,and then went down that root, and then folded soon after, or their price fell back again .Luckily for OXB they no longer come into that category thanks to the Novartis contract ,plus OXB are backed by good money and are looking to be self sufficient with in a few years ,so no AR, I'm not worried by that figure, and never will be, and that's why I believe OXB can hold a market, even with those shares in circulation . Happy New Year every body BE HAPPYDave (The figures shown above are an example of how a company with a big share issue can still make a market in the same way that another company with less shares can )
green.as1953
04 Jan 2018
My GUESS is OXB will be taken over in 2018. GLAH
