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WSCS 2015: Clinical and Business Overview - Exciting Clinical Readouts for 2016

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WSCS 2015: Clinical and Business Overview - Exciting Clinical Readouts for 2016

2015 Clinical and Business Overview - Exciting Clinical Readouts for 2016.

Video Transcript


Robert Margolin, MBS, Cognate Bioservices, Inc

Hello's I believe I am the only panelist for this reason to I had another person that was Donna system that wasn't able to make it to see you this afternoon so this presentation usually takes me about 15 or 20 minutes I'm a stretch it out to 25 minutes but there's really two main objectives of the presentation number 12 percent.

Everyone with more of a data overview of the cell and gene therapy industry number of companies clinical trials disease indications things of that nature and then once that's finished I'll take you through each month of this past year to show you some major clinical and financial milestones that we as an industry to start off my name's Rob are going I am the vice president of corporate development for cognate bio services cognate is a cell therapy focused contract manufacturing organization but over the years I have maintained a database it was used to be used for the Alliance for regenerative medicine we continue to work on it over a cognate.

I would say it's one of the more complete and accurate data bases in the industry so to start off if you look at the industry at large in this is against cell and gene therapy we have a total of about five hundred and eighty companies that make up this industry this could be a little larger or smaller depending on who you talk to you about in our database we have about five hundred and eighty companies three hundred and two of those are in North America a hundred and sixty-six in Europe 92 in Asia and we have a few other scattered in South America, Australia, New Zealand of these five hundred and eighty companies those included cell therapy companies as well as tool and service companies.

We look at just a therapeutic company are looking at about a hundred and thirty four major companies on the cell therapy side and a prodigally a hundred and eleven on the gene therapy side of those on a cell therapy side about seventy percent of public 30% private and gene therapy it's the exact reciprocal so of those hundred and thirty-four products and the cell therapy space we are tracking two hundred and eighty-nine clinical trials are a lot of times.

You'll see people present that there is maybe thirty thousand cell therapy clinical trials worldwide these are industry- sponsored trials and then on the gene therapy front hundred eleven products that are in right now a hundred and fifty-four trials of those in the cell therapy a hundred and seventy-three or preclinical 257 or actually clinics and a hundred and seven preclinical and ninety-two and is one through three trials when you take a look at the products on the market we have 34 cells therapies products that are marketed today most of those being for musculoskeletal dermatology oncology and inflammation / immunology and then on the gene therapy front.

We have unit cure's globe era when you start looking at these technologies in what are the major indications they're targeting of course we know that there is a plethora of diseases and indications that these are being developed for but the phase 3 the majority of the phase 3 technologies are being used in oncology cardiovascular dermatology in musculoskeletal on the cell therapy front and cardiovascular oncology and dermatology for the gene therapies phase two fairly similar trends but we start seeing CNS come into play for the cell therapy use and quite a bit more activity in the hematology space and the cardiovascular space on the gene therapy front and you know as you go into the phase once again CNS starts developing further and you can just see that the trends of some of the more first or second generation technologies to the more modernones and then when you go into the preclinical.

You can see how at this point CNS actually becomes the most dominant of the therapeutic categories so that gives you a bit of a flavor of what's in the pipeline and now moving over to the actual events of the year this is for September in September we saw two data announcements we had to do a bio tech released phase 2 results for pancreatic cancer brainstorm announces phase 2 analysis for a less we had two new trial start one Capricorn another foresight story we had various cell completed its enrollment for the ischemic dilated cardiomyopathy trial and then we had to special designations in January.

One was for cut farm after its tough use large B cell lymphoma C arty technology and give me to sell received FDA and EMA orphan drug designation in January on the business front adder al raised fifty-one point four million in Series D we saw a few partnerships we hadn't Elian of artists around the crisper technology another crisper collaboration between Astra Zeneca and a few other academic centers and Jon and Kate announced their partnership and then we had care of three acquire or on site for tell me ten million upfront and fifty million in milestones and in lorry on also acquired ZIOPHARM the major news for January was the engine deal fifty million up front 252 and milestone payments so moving on 2 February.

In this is going to be somewhat of a granular presentation so if no one wants to listen to all these data announcements I will not be offended if you like him have a good so far for February we had announced more announces phase one to trial we had a few new trial start we had lion bio in the immuno therapy space and two additional trials by Cytori and again an FDA breakthrough designation to Bluebird bio for Pawlenty globe in beta-thalassemia technology on the business from February we had an IPO by bone therapeutics another IPO by spark 480 5.2.

We saw further collaboration between Caton NCI and Voyager and Genzymehad a collaboration for CNS disorders a hundred million upfront and 745 million in milestones which was the big data announcement for February moving on to March we had clinical data announcements by strata tack for severe burns we saw the phase 2 trial top-line data from neural stem we had a these 12 trial initiate with Darius and that is for the spinal cord injury and Mary Jean U Phase 1 24 there'd NDC vaccine for a male and again to special designations would start becoming a theme for 2015 we had a torah with FDA breakthrough and comedic with GMA orphan drug both for Epstein Barr virus specific immuno therapy type of products on the business front seem a therapeutics had a 44 million Series A round collects had its very successful 228 million IPO Durham the collaboration with Novartis for 200 million upfront Fuji film acquired cellular dynamics.

The Madison Wisconsin based drug discovery company and Kate acquired TESOL factor EBV the news of the month was the adorn and Novartis deal moving on 2 April we had the other sis fees to mufti stem stroke data which was unfortunately not as positive as some of us were hoping for we had data from Juno Okada to trial started by rebels sell for the chronic Achilles tendon Osos we also a trial start by Cardin 3 which is at this point in time now selling ad and we had enrolled in completions by Capricorn and stem cells into special designations one biceps story and the second one for Caprica or in California on the business front.

We had a series of finances this month's and bio had a 67 million dollar IPO Voyager 860 million in Series B Johnson 256 million Series B dimension 65 Series B or threats and west by our is forty million on a follow-on and Unicare had an 88. 5 million follow-on these elastic send extended its collaboration with Celgene which again happened about a month ago for the second time MedImmune had the partnership with Juno more mad entered the party filled by exercising its options with San Raphael and bristol-myers Squibb and Eunacure had to cut collaboration for cardiovascular disease with fifty million dollars up front and four hundred and seventy-one million dollars in milestones moving on to may.

We had clinical data announcements by Juventuswas successful in their ischemic heart failure trial stem cells Inc published phase 12 top-line data for their spinal cord injury trial and then we got a plus revenue come out from the ASCO event for adaptive you in Juneau Novartis and several other companies in the immuno therapy space burner on started phase 1 trial so those are our friends over in the UK and runner on also received FDA fast track for its rhinitis pimentos trial and fibrous also received a rare pediatric disease decade designation on the business front we had a thought the adaptive immune IPO 491 million for Gen X bio reads 70.5 in a series D and Juventus had a 13.5 million dollar financing the on the business front for deals and collaborations we saw Bluebird enter a licensing agreement with five prime our company cognate acquired Akron bio technologies organ did a partnership with L'Oreal for development of 3d bio printed skin and then we also had the Juno feet collaboration and Juno and Adidas medicine.

I mean this was really a big month for Juno here as they also acquired stage Cell Therapeutics and then lastly we had formula pharmaceuticals acquired an allergen Adam party platform and the major news story for this for the month of May was a When tech and this was thirty million signing fee and up to three hundred million dollars in milestones so June the major news continuing on with the Juno news was in June Celgene committed a hundred and fifty million upfront 850 million and common stock probably making it the first billion dollar deal in the cell therapy space.

We had a bluebird announced clinical data for its beta tha lassemia sickle cell anemia trials music blasts published fees to diet data for diabetic nephropathy trial and avalanche had some mixed data for its wet age-related macular degeneration we had new trials initiated by Q therapeutics and Utah Capricorn kicked off its nap 100 to its allogeneic study TXL extended its base to you to be study site or II completed enrollment in its osteoarthritis.

trial very cell in June met its it stated that its be LA for the MAAC I would be the end of 2015 for knee cartilage defects and then lion Juventus and I believe it's pronounced AV six also receive special designations on the business front we also had a IPO by selling had a65 million Series B by noon on which is the newer immunotherapy company the Boston area and Santana X-rays seventy million in a Series day Blue birds regained its rights to its Carty programs outside of BCM a collaboration with Celgene same month Bluebird establish collaboration with tight we saw Seattle Genetics in Newman partner up obviously.

The cell gene june audeal that we mentioned earlier and a few other acquisitions probably the biggest one being a.m. and Ag farm acquiring cord blood registry for seven hundred million dollars in July we had to adapt immune data come out for the multiple heloma trial we also saw depth immune kickoff at trial for non-small lung cell cancer five or so and interxion initiated a phase one to trial for dermal least I never know how to say that one but will skip and enrollment completion zara has completed its enrollment for its phase 3trial for a GSO 2003 and selling had completed its final patient infusion for its part 1 phase 3 trial that's the cardiovascular trial which hopefully will be seeing some data come out within the next within the next quarter to on the business front.

We had an IPO by created us Gen sites the gene therapy company raised 36 million in a Series be called Genesis at ten million in SeriesB and then we saw the Binges Ideas in the GTC collaboration to develop therapies in ophthalmology this is a hundred and twenty-four million upfront and up

We also saw a few more special designations 14 days with GMA orphan drug designation in a dentist getting both USA and EMA orphan drug for x-linked my tubular sympathy and then on the business front we had sent again receiving a Series A round Adidas medicine had a hundred and twenty million in a Series Band then attack had 14 million IPO and then lastly read the Novo collaboration which had 27.5 up front and seven hundred million dollars and milestones for September and we're getting there we have a few more months to get through September.

We had the clinical data announcements from Munich year some really great data coming out of you pan for the Novartis CTL 019 patients were they saw significant ninety-plus percent remission in that trial Novo had its fees to be trial data come out we add a new dimension and safe Tory initiate trials adorocompleted its Roman and its metastatic pancreatic cancer trial and music blasts with its partner JCR Pharma had their product approved in Japan for special designations we sought to mention TXL Amaranths all receive either fast-track orphan drug or orphan drug designations and on the business front we had their Genex bio.

I feel 459 million as well as a few other follow-on since series an and your dentist acquired carnage in sciences as well and September moving on 2 October we had clinical data announcements by spark Music blast site Tory a plethora of my undies and trial initiations and again a few more special designations Byron GEnx cell cure neuro sciences and AGT see a dentist therapeutics 365 million in Series We had the dimension therapeutics IPA orIPO and the major deal with the vertex and crisper therapeutics collaboration on the cast 19 editing technology and that was what a place for the news of the Month in Octobers for November which is the last month in a cover for this presentation we had to clinical data announcement from organized Genesis that Apple graft received marketing approval and Switzerland we had a few new trials kick off one by a farmer or refractory mantle cell lymphoma a GTC had another trial that they kicked off and another one for Kate on the special designations.

We had another EMA and FDA orphan drug by bone therapeutics and on the business side we had 35 million raised from night star X and a 42.5 million Series B four solid et and then lastly we saw Astellas acquired Okada therapeutics which was originally the advanced cell technologies and the news of the month they selected for November was the Apple graft receiving the approval in Switzerland so now with that being the overview and we start looking into the trends for this past year I think one major trend that we saw there's been a real acceleration in the number of special designations especially for some of these technologies in the cancer and immuno therapy space these companies are literally flying through the clinical pipeline which is really exciting and hopefully we'll see some of these products even get to market in 2016 and 2017 so 2014 I think was really the kickoff of the tautologous gene modified T cell therapies.

I did we started seeing this year is not only do we have tautologous immuno therapy products but we're on our seem at all against an allogeneic modified T cell therapies and also other immune cells such as modified natural killer cells and dendrite cells etc eteraso we also saw a lot of companies in 2015 us' companies initiate trials in the EU and also a lot of the EU companies starting to initiate trials in the USA and basically multinational trials from some news of the news pieces of the month one of the major transition.

I would say 2015 is the most interest that we've seen thus far in cell and gene therapy from Big Pharma and the big biotech companies you know we saw amgen and quiet Genzyme Voyager Novartis adorobristol-myers Squibb and Uni-care lily and bio and tech Celgene Juneau etcetera these deals right here alone totaled up to one point five billion dollars with 5.

3 billion in potential milestone so I think this is an all-time high for our industry and really showing that this is an industry that's maturing and it has technologies that are getting closer to the market this I actually took from the Alliance for regenerative medicines Q-two quarterly data rapport.

What you can see when you look at the total financing for the first half of 2014 and then you look at it on top for 2015you can see that our industry has done incredibly well in 2015 and I think we're going to see similar trends for 2016 and without that concludes my overview for 2015unfortunately since I don't have my partner here for the 2016 readouts I think we'll have to hold out one but I think you can see from this presentation you can start to expect get a good understanding of what kind of data announcements will be coming out for the next few quarters so what that like to say thank you and if anyone has any questions to answer them.

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