PTC Therapeutics CFO: Rare Potential

1 00:00:06,310 --> 00:00:09,830 all week we've been highlighting some the hottest stocks out their new series 2 00:00:09,830 --> 00:00:10,570 biotech 3 00:00:10,570 --> 00:00:15,120 the next generation travel are taking a closer look at 12 10 4 00:00:15,120 --> 00:00:19,800 best-performing biotech names a 2014 school PTC therapeutics 5 00:00:19,800 --> 00:00:24,220 PT CT which rally to 15 percent leisure and I think there could be even more 6 00:00:24,220 --> 00:00:25,060 upside your 7 00:00:25,060 --> 00:00:29,080 whose PTC isn't orphan drug developer hit just like the company you know I 8 00:00:29,080 --> 00:00:29,750 told you 9 00:00:29,750 --> 00:00:34,010 of NPs former which we learned last week is being acquired by sharp for a monster 10 00:00:34,010 --> 00:00:34,530 premium 11 00:00:34,530 --> 00:00:38,690 PTC top small molecule therapies that modify 12 00:00:38,690 --> 00:00:42,559 are in a transcription and translation which swept fixing rare genetic 13 00:00:42,559 --> 00:00:43,329 disorders 14 00:00:43,329 --> 00:00:46,809 especially those that are caused by what are known as nonsense mutations 15 00:00:46,809 --> 00:00:51,209 in your DNA companies me drug trans lorna has been approved in me 16 00:00:51,210 --> 00:00:54,410 the EU produce a muscular dystrophy will call a DMD 17 00:00:54,410 --> 00:00:59,379 at only fatal working condition and the US launch cook on next year pending FDA 18 00:00:59,379 --> 00:01:00,019 approval 19 00:01:00,019 --> 00:01:03,339 the other by titration develop troopers for this disease including by Marie much 20 00:01:03,340 --> 00:01:04,239 spoke to yesterday 21 00:01:04,239 --> 00:01:08,470 but PTC is alone in targeting a specific subgroup D&D 22 00:01:08,470 --> 00:01:11,670 patients roughly 15 to 20 percent the overall population 23 00:01:11,670 --> 00:01:15,400 who particular genetic mutation that's why the DVD indication alone could 24 00:01:15,400 --> 00:01:18,190 potentially be worth a billion dollars apiece sales for PTC 25 00:01:18,190 --> 00:01:21,820 plus this drug is also being studies treatment for cystic by pro 26 00:01:21,820 --> 00:01:25,380 fibrosis wheres interface three in a group with inherited metabolic disorders 27 00:01:25,380 --> 00:01:27,119 known as MPs which base to 28 00:01:27,120 --> 00:01:31,730 tablet PC is a solid pipeline clearly a spinal muscular atrophy treatment 29 00:01:31,730 --> 00:01:33,740 they're developing novel with Roach 30 00:01:33,740 --> 00:01:37,350 entry on call the platform it targets the stem cells a progressive 31 00:01:37,350 --> 00:01:40,440 drug resistant tumors that's moving into Phase one you know earlier is that 32 00:01:40,440 --> 00:01:41,820 something about Mike's okay 33 00:01:41,820 --> 00:01:45,530 now PTC may have dropped dramatically your but it's also in the sweet spot 34 00:01:45,530 --> 00:01:49,790 what's being work been working biotech so take a close look machine kovacs 35 00:01:49,790 --> 00:01:51,880 chief financial officer PTC therapeutics 36 00:01:51,880 --> 00:01:55,679 find out more about this company and its prospects mister cohen welcome to make 37 00:01:55,680 --> 00:01:56,070 money 38 00:01:56,070 --> 00:01:57,450 this user 39 00:01:57,450 --> 00:02:01,170 nice to meet you watching I've gotta tell you when I was going through %uh 40 00:02:01,170 --> 00:02:05,460 my first thing I read this was like wait a second Rd MD we are no indications 41 00:02:05,460 --> 00:02:07,270 just my personal reno's working that 42 00:02:07,270 --> 00:02:10,440 you what he wants upsets apps upsets 43 00:02:10,440 --> 00:02:14,060 where no one else seems to be change conditions 44 00:02:14,060 --> 00:02:18,710 tell us about the D&D in window of what's so important they can get your 45 00:02:18,710 --> 00:02:23,000 that's right i mean so for all these rare diseases were talking about 46 00:02:23,000 --> 00:02:27,020 different mutations that ultimately lead to the loss in the protein addition 47 00:02:27,020 --> 00:02:30,270 muscular dystrophy that's these nonsense mutations that we're focused on 48 00:02:30,270 --> 00:02:33,300 its also truancy at it's also true in MPS 49 00:02:33,300 --> 00:02:36,730 so it was a ten-percent other people love it 5 percent of the people that 50 00:02:36,730 --> 00:02:37,350 disease 51 00:02:37,350 --> 00:02:41,739 yeah in to shed its between 10 and 15 percent we estimate around 13 percent 52 00:02:41,740 --> 00:02:46,440 in CF it's around 10 percent in NPS is actually but 62 eighty percent of 53 00:02:46,440 --> 00:02:47,370 patients okay 54 00:02:47,370 --> 00:02:52,010 how people know if they have that mutation they have to go and get 55 00:02:52,010 --> 00:02:56,010 Gina typically identified to have to sequence the DNA to confirm that it 56 00:02:56,010 --> 00:02:59,810 is a nonsense mutation as opposed to one any other type and deletion we do five 57 00:02:59,810 --> 00:03:00,300 years ago 58 00:03:00,300 --> 00:03:04,750 a it's getting faster and is getting cheaper to do it for the patients 59 00:03:04,750 --> 00:03:08,019 okay when the things that I notice was missus sad stuff for you 60 00:03:08,019 --> 00:03:11,090 actually everything you treat ourselves is very sad because 61 00:03:11,090 --> 00:03:14,370 is one where it looks like that if you get to people who were between five and 62 00:03:14,370 --> 00:03:15,220 six years old 63 00:03:15,220 --> 00:03:18,510 you might be able to say their lives looks like later on you can't 64 00:03:18,510 --> 00:03:22,149 your intuition you wanna treat these boys 65 00:03:22,150 --> 00:03:25,680 as the younger cool with that right it's boys that year 66 00:03:25,680 --> 00:03:29,730 its predominately boys because it's XLink genetic disorders what lies on the 67 00:03:29,730 --> 00:03:31,049 extra chromosome 68 00:03:31,049 --> 00:03:34,150 now if they don't have abstract Erum the 69 00:03:34,150 --> 00:03:37,209 all the research is that could be worth a billion dollars it seems like that if 70 00:03:37,209 --> 00:03:37,560 the 71 00:03:37,560 --> 00:03:41,110 if that they have to be treated for many years in an all-male they do died but 72 00:03:41,110 --> 00:03:41,380 that 73 00:03:41,380 --> 00:03:45,420 I healthcare me a health maintenance organization would have to pay millions 74 00:03:45,420 --> 00:03:46,400 over time 75 00:03:46,400 --> 00:03:49,959 to take care these people unless these people were identified and they take 76 00:03:49,959 --> 00:03:50,530 your drop 77 00:03:50,530 --> 00:03:55,280 into shenice some muscle wasting to sort out what we're trying to do is stabilize 78 00:03:55,280 --> 00:03:57,950 the boys to keep their healthy muscle healthy 79 00:03:57,950 --> 00:04:01,630 hot so that it doesn't isn't nope so easily damage 80 00:04:01,630 --> 00:04:05,630 in so you wanna T his body as possible this will be a chronic their people take 81 00:04:05,630 --> 00:04:06,790 it for the rest of their lives 82 00:04:06,790 --> 00:04:10,310 about cystic fibrosis again you're not trying to do what vertex is doing you 83 00:04:10,310 --> 00:04:11,750 got a specific subsets 84 00:04:11,750 --> 00:04:15,820 cyst yet this is a mutation that this different and distinct from the 85 00:04:15,820 --> 00:04:18,920 from the mutations that vertex is working on this is the nonsense 86 00:04:18,920 --> 00:04:20,330 mutations the class 1 87 00:04:20,329 --> 00:04:23,750 is actually the most severe form a cystic fibrosis 88 00:04:23,750 --> 00:04:27,670 hurt you are stumble on this whole are in a technology 89 00:04:27,670 --> 00:04:31,240 it's very different from companies that use a we're going to go cure cystic 90 00:04:31,240 --> 00:04:32,190 fibrosis 91 00:04:32,190 --> 00:04:36,310 you know we've got a different and unique approach to are in a biology into 92 00:04:36,310 --> 00:04:39,100 rare diseases unlike some of the enzyme replacement their eyes 93 00:04:39,100 --> 00:04:43,330 where you making the protein in a lab and then if using it we 94 00:04:43,330 --> 00:04:47,289 our approach is to enable the patience to produce their own protein 95 00:04:47,290 --> 00:04:51,660 using a small molecule approach now there's you're not proved to me this is 96 00:04:51,660 --> 00:04:53,910 not something as pie in the sky is happening right now 97 00:04:53,910 --> 00:04:57,200 yet last year was a monumental year for us because 98 00:04:57,200 --> 00:05:00,680 the European authorities approved Arjun this the first approval 99 00:05:00,680 --> 00:05:04,980 ever in this disease for the underlying cause and given that that means that 100 00:05:04,980 --> 00:05:08,480 you're going to have read the news and it would seem that the EU likes it what 101 00:05:08,480 --> 00:05:12,820 we've seen is it's very rare that do you like something that the FDA does it 102 00:05:12,820 --> 00:05:16,250 right now we we've already begun shipping to patients in Europe and 103 00:05:16,250 --> 00:05:17,620 actually in a lot of countries 104 00:05:17,620 --> 00:05:22,260 outside a europe to recognise the European approval in the US we've got 105 00:05:22,260 --> 00:05:26,780 the largest study ever to be done in DMD it's well under way i'll read out at the 106 00:05:26,780 --> 00:05:27,490 end of this year 107 00:05:27,490 --> 00:05:30,820 but it knows people look into it we'll see that it's really we still talking to 108 00:05:30,820 --> 00:05:32,250 him for people in Europe right 109 00:05:32,250 --> 00:05:35,720 means the numbers are not big yeah it's its it is a unique 110 00:05:35,720 --> 00:05:39,760 it's a 2500 potential patients in but I mean right now your role in just a 111 00:05:39,760 --> 00:05:41,210 handful people painful 112 00:05:41,210 --> 00:05:45,090 have found it so far right yeah we've got will work early in the launch right 113 00:05:45,090 --> 00:05:46,470 so how many right now 114 00:05:46,470 --> 00:05:50,220 with a look at we've just launched in Germany last month 115 00:05:50,220 --> 00:05:53,570 we started to shipping into France and to Spain 116 00:05:53,570 --> 00:05:57,480 earlier towards the end of last year okay because I want people to know that 117 00:05:57,480 --> 00:05:59,660 this is early but you do what you are gonna 118 00:05:59,660 --> 00:06:03,100 revenues from the spotlight and there's a bright future for yet no 119 00:06:03,100 --> 00:06:06,380 European launches are slower than the US right but they do 120 00:06:06,380 --> 00:06:10,120 gain momentum okay well now people know why it's up so much is rare 121 00:06:10,120 --> 00:06:14,410 and it's the only thing for these people at Shinko that's the Seattle 122 00:06:14,410 --> 00:06:18,230 PTC therapeutics nice very thorough presentation from this one 123 00:06:18,230 --> 00:06:21,510 if you wanna know more about it don't buy the stock and to you bread 124 00:06:21,510 --> 00:06:23,190 the presentation him ice pack

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