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Post by biposter on Nov 23, 2018 19:58:20 GMT
CPRX | Nasdaq | $2.94 | $302M m/c | $66M COH (9/28/18) | ~$8M/qtr. burn rate, but will increase
- Ultra-rare disease: LEMS (~3,000 cases in the U.S.) | Tx: Firdapse
- PDUFA date: 11/28/18
- Risk: FDA rejects (or postpones decision): $1.00-$1.50 pps tank
- Reward: FDA approves: $4 (if approval is already priced in); $5-$6+ (if not baked in)
- LEMS peak revenue: $300M-$900M
- If FDA approval, CEO: “commercial launch in early 2019”
- Two other indications in late Ph3 trials w/ TLD expected in 2H19
Here’s another imminent risky binary play: CPRX. The PDUFA date is next week on 11/28! Their focus is on ultra-rare diseases. Their orphan drug Firdapse has completed two Ph3 trials for the rare disease LEMS (autoimmune disorder affecting communication between nerves and muscles, resulting in severe muscle weakness). After their first Ph3 trial in 2015, which was positive, the FDA requested a second Ph3, which was completed in late 2017 (on their two primary endpts, the p-values were: 0.0004, 0.0003). CPRX submitted an NDA in May 2018 and got priority review from the FDA (PDUFA date: 11/28/18). If FDA approves next week, could see a nice pop (one risk, even on approval, is that some of that is already priced in). If FDA rejects (or delays its decision), then it’ll plummet. The data so far has been excellent, but the risk is the role of politics (drug pricing) on this decision because of Firdapse’s long history -- an equivalent drug has been given out for free but limited to only about ~20% the LEMS patient population (with 80% not having ready access to it), since it wasn’t FDA approved. Read AF’s old Street articles on CPRX to get the full backstory. But here’s an SA article out this week on CPRX that gives a good overview and seems to address and counter those objections. For more info. on this debate, click on the links in the SA article, and read the latest CPRX CC, where mgmt discusses their Expanded Access Program to make sure all LEMS patients have access to the drug): seekingalpha.com/article/4223988-catalyst-pharma-rare-orphan-drug-opportunity-low-risk. On their most recent CC (11/4/18), the CEO said that if upon approval, their plan is a “commercial launch in early 2019.” And if Firdapse is approved, there are three other rare diseases it can be used for, and two are in Ph3 trials with data expected in 2H19. If positive, they intend to go to FDA to expand the Firdapse label. In the SA article, it says CPRX used to give guidance of ~$300M-$900M peak revenue for LEMS (but probably hasn’t mentioned any figures publicly in recent years due to the controversy). So everything hangs on this FDA decision on Wed. If it’s a Go, they may very well be a revenue-generating co. in 2019, which could drastically affect the market’s evaluation of CPRX. Anyway, when reading ST posts on ONCS and SESN, you’ll find poster “bigBIOboom” twitting constantly. Though it’s tough to defend him against accusations that he’s a pumper with a screen name like “bigBIOboom” LOL, he seems to do thorough research and sticks to his top five biotech picks (instead of pumping & dumping an endless stream of picks). They’re all late-stage with a possible BLA or AA filing about a year or less away, with strong data in prior trials, and with a drug that has strong revenue potential. This, along w/ ONCS and SESN, was one of the five, so I tried to do some DD on it, and it seemed to be a very interesting pick.
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CPRX
Nov 29, 2018 7:37:48 GMT
Post by biposter on Nov 29, 2018 7:37:48 GMT
www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm627093.htm CPRX’s Firdapse got FDA approval for LEMS today. It did have BTD, ODD, and Priority Review from the FDA. Stock was halted AH, and they should PR the approval pre-market tomorrow morning. How the stock will respond is anyone’s guess. After tomorrow’s volatility, next step is for CPRX to give pricing info. for Firdapse. And as the CEO stated earlier this month about commercializing Firdapse in early 2019, the market’s valuation of this rare-disease company may then have to be radically adjusted. It was good to see this quote from the FDA in their announcement: “There has been a long-standing need for a treatment for this rare disorder,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “Patients with LEMS have significant weakness and fatigue that can often cause great difficulties with daily activites.” And there are two other Ph3 trials for Firdapse that were supposed to report data in 1H19 but has been pushed back to 2H19. So hopefully they report in mid-2019 and not late 2019, and if positive, Firdapse could then be approved for three rare diseases. After the co. mistakenly published a graphic yesterday indicating Firdapse approval, AF ominously tweeted that this did not bode well. Not sure what he meant, but expect a nice AF double-barrelled follow-up article tomorrow morning to add to the volatility LOL.
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CPRX
Nov 29, 2018 18:31:19 GMT
Post by nateb on Nov 29, 2018 18:31:19 GMT
Weird market, they get approval and the stock sells off.
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CPRX
Nov 29, 2018 19:37:18 GMT
nateb likes this
Post by biposter on Nov 29, 2018 19:37:18 GMT
ir.catalystpharma.com/news-releases/news-release-details/fda-approves-firdapser-amifampridine-treatment-lambert-eaton LOL Yeah, nateb, it even dropped as low as $2.32 today! Imagine if they’d gotten a CRL delay, this would probably be around $1.25. And if it was a flat-out FDA rejection, this would be $0.50-$0.75! Anyway, it seems to be recovering a bit. In the PR today, it said they’d hold a CC to discuss launch details on 12/13, instead of today, so I think that helped the “sell the news” momentum. It does say they will launch in early 1Q19, so once we get the pricing info., maybe the stock might start its move. We'll see.
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CPRX
Nov 30, 2018 18:24:13 GMT
Post by nateb on Nov 30, 2018 18:24:13 GMT
Yeah I don't get it. It is down again today. I have seen some speculation that they are about to do an offering, so maybe that's why? I am contemplating establishing a position, but I want to see the share price stabilize some. This is a troubling trend with bios lately: VSTM is another example of sell off post FDA approval. It used to be an FDA approval was a money making event. Maybe its just another sign of us being in the later stages of the bull market?
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Post by biposter on Nov 30, 2018 19:51:38 GMT
Yeah, not by AF, but a Stat article burying the lede with an inflammatory heading came out today. Start out with an individual’s story, throw out the name Martin Shkreli for good measure, include quotes calling the co. “exploitative” and “profiteers,” and you have a great article. And SA fanning the flames, sending out multiple alerts with this article’s heading, didn’t help. I mean, overall, it was a fair, balanced article, but the key point for the 80% of LEMS’ patients who do NOT have ready access to the drug, even through Jacobus’ program or from compounding pharmacies, is this:
- “[LEMS’ patient Leigh Shell] also tried to get the drug from Jacobus, but came up short. ‘Nobody responded to us, because we didn’t have a doctor that was connected with Jacobus to get it for free,’ Shell said. (Jacobus says it currently provides 3,4-DAP for about 350 LEMS patients.)”
- “Shell isn’t concerned about the cost: She called her insurer, Blue Cross Blue Shield, and they assured her they would pay for the drug. ‘Our insurance company said they view this as a specialty drug — saying that because so few people need it, they’ll cover it,’ Shell said. ‘They told us our out-of-pocket cost would only be $60 per month.’ Catalyst has said it plans to set up programs to help patients navigate insurance questions and apply for financial assistance once the drug is launched in the first quarter of 2019.”
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CPRX
Dec 13, 2018 21:35:48 GMT
Post by biposter on Dec 13, 2018 21:35:48 GMT
ir.catalystpharma.com/events-and-presentations Okay, $375,000 (before discounts & rebates) was higher than expected lol! I thought it would be *only* $200k-$300k lol! Stat had another article out today, but it’s behind a paywall. But I’m sure it’s a repeat of their other one a few weeks back, which never addressed the key question: What about the other 80% of LEMS patients who did not have ready access to Firdapse? Anyway, I thought they did a good job on the CC addressing all of these drug pricing concerns. So they said today that most diagnosed LEMS patients do have insurance. They now have a thorough commercialization plan already in effect to make sure most of these providers will insure Firdapse for LEMS. They confirmed that there are about 3,000 LEMS patients in the U.S., and they’ve definitively identified 1,500 patients through claims data from the past two years, and these 1,500 will be their “immediate addressable market.” The other 1,500 are misdiagnosed or undiagnosed (and they have robust plan underway to educate neurologists & neuromuscular specialists about LEMS). But their goal in 2019 is convert all 300 current patients on Firdapse (or its equivalent) from the Jacobus program or CPRX’s own expanded access program to their commercial product. What % of those 300 will continue with free product vs. commercial product was not clear. But it seems there will be a lag in build-up time transitioning these patients, getting insurers to formally cover Firdapse, and then the lag (60-90 days) in actual reimbursement time. Maybe that’s why the pps didn’t react much today. This is an orphan drug, so they have seven years’ exclusivity before generics. Compounding pharmacies cannot compound this drug, and it seems Jacobus can no give out its equivalent for free. So with the remaining 1,200 diagnosed LEMS patients, they said they will have commercial availability and launch for these patients starting on Feb 4th. Their royalty rate to BMRN (CPRX licensed Firdapse from Biomarin back in 2012? or so) is 14% of revenues up $100M in a given year (>$100M, 17% royalty rate). They also confirmed that their two other Ph3 trials will read out in mid-2019 (CMS: ~1,000-1,500 in the U.S.; MuSK-MG: ~3,000-4,800 in the U.S.). So maybe the pps may stagnate until mid-2019 until they book real revenues and the other two Ph3 trials release top-line data. They had $66M COH at the end of Q3 w/ an $8M/qtr. burn rate that months ago they said would increase. Might be an interesting play to build a full spec position over the next six months. But the CC is worth a listen.
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CPRX
Jan 4, 2019 20:51:55 GMT
JHam likes this
Post by biposter on Jan 4, 2019 20:51:55 GMT
ir.catalystpharma.com/news-releases/news-release-details/catalyst-pharmaceuticals-announces-publication-clinical-data PR out today -- they published data on their previously announced Ph2 MuSK trial, which was positive. The two primary endpts were statsig (p=0.0003; 0.0006) as well as all the secondary endpts. A more severe form, MuSK-MG, is now in a Ph3 trial with TLD expected in 2H19. This is a pivotal Ph3 trial with an SPA designation. Per their slide deck, there are ~3,000-4,800 MuSK-MG patients in the U.S., and there are no currently approved therapies. So Firdapse has been approved for LEMS (~3,000 in the U.S.) and is now also in two Ph3 trials: MuSK-MG (~3,000-4,800 U.S.) and CMS (~1,000-1,500 U.S.) with TLD here also expected in 2H19. CMS also currently has no approved therapies. So if the Firdapse MuSK-MG trial is also positive, then it looks like with the SPA, they should be filing their NDA later this year. So Firdapse is priced at $375,000, and their goal in 2019 is to get all 300 patients who have been taking the Firdapse equivalent onto the FDA-approved Tx. By the end of 2019, they say they expect the majority of insurance plans to cover Firdapse for LEMS. Btw, two BOD members bought a total of $250K worth of stock last month, and the CEO bought about $45K worth of stock in Dec. So the CEO now has 4.7m shares of CPRX stock. It’s interesting that just over a year ago (Nov 2017), the CEO and two other BOD members each bought 200,000sh of CPRX stock at $0.47.
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CPRX
Feb 4, 2019 22:49:53 GMT
Post by biposter on Feb 4, 2019 22:49:53 GMT
Uh-oh. I was hoping we’d escape notice from Elizabeth Warren and Trump, but it looks like we’re now in the crosshairs of Sen. Bernie Sanders.
“Senator Sanders to ask why drug, once free, now costs $375k”
- Because it was only free to ~10%-20% of LEMS patients. Eighty percent had no ready or any access at all! Yes, it was a good deal for those 20% who had connected doctors. But what about the other 80% left out in the cold?
- And even among those 20%, there were many hoops for the patients and their doctors to jump through to get the cumbersome filings/paperwork submitted on a regular basis to gain/maintain access. Yet, still, many patients had to drive hundreds of miles every few weeks to get their supply. Now, once insurance approves coverage, it will be mailed to their front doorstep.
- So before CPRX ran two very expensive full-on Ph3 trials, why didn’t Jacobus Pharmaceuticals or any other company out there do so over the past three decades? Because these Ph3 trials cost tens of millions of dollars to run! Who would’ve had the incentive to do so if they couldn’t price the drug accordingly post-approval (which was risky and not guaranteed, no matter how good the data results, in light of the drug-pricing political risks). Jacobus only started their trial after CPRX did so, and they too admit they were then going to commercialize Firdapse (though they never gave out any pricing info). Still, these trials were so expensive, Jacobus never did complete even a single trial, while CPRX completed two long Ph3 trials. Shouldn’t they be able to present a bill for such services? After all, we may be a socialist democrats, but, certainly, we are not communists.
- CPRX has set up a program where *every* LEMS patients will get Firdapse, even those who are unable to get insured.
- After their seven years of Orphan status & patent protection is over, generics will be able to step in. So they have a seven-year window to recoup their costs and make some profit. Of course, they’ll continue to try and patent new versions of Firdapse, but at least the key active ingredient will be available as a generic.
- And once insurance coverage is widely available, every single LEMS patient in U.S. will have ready access to Firdapse, not just the top 1%. I mean, top 20%...
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CPRX
Feb 6, 2019 19:37:55 GMT
JHam likes this
Post by biposter on Feb 6, 2019 19:37:55 GMT
tomwelch12345.wordpress.com/category/fda-violations/ Interesting. Jacobus Pharmaceuticals, the company providing the free 3,4 Dap (active ingredient of Firdapse) that all the CPRX critics point to, has their drug manufacturing facility inside an abandoned nuclear reactor test site! They’ve been repeatedly cited by the FDA over the past 16+ years for manufacturing and safety violations, and even issued an FDA “Warning Letter.” You can see some of these FDA letters above. At the bottom of the website, he includes H.C. Wainwright analyst Andrew Fein’s 2013 report on Jacobus Pharm. He gets down to some of the specific FDA violations and gives a little history of their facility (including a pic of the conical dome): “It housed a laboratory for low-level atomic research. The reactor became obsolete and was decontanimated, and ownership reverted back to to Walker-Gordon Laboratory Company. Today, it houses the offices of Walker-Gordon and the laboratories of Jacobus Pharmaceuticals. Though he applauds Jacobus for the service they provided LEMS patients, he concludes that when you bore down to the cost/benefit of therapy, quality control, and patient vigilance, the compounding landscape was “more like the Wild West.” I wonder why Adam Feuerstein and his Stat reporters never probe deeper on the Jacobus Pharm side of the debate. (Btw, I actually like AF and have a lot of respect for him. He’s a great reporter, calls it like he sees it, and seems to be a genuinely good guy. Except when he’s bashing my stock. Then he’s in cahoots with the hedgies, shorting my stock! lol) Why does AF want to stick with the status quo, excluding four out of five LEMS patients from access to the drug, and with pills possibly contaminated with radiation! (Okay, joking about the last part, but still… Would you want to take pills knowing it was made at that conehead dome?)
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CPRX
Feb 6, 2019 21:46:11 GMT
Post by JHam on Feb 6, 2019 21:46:11 GMT
tomwelch12345.wordpress.com/category/fda-violations/ Interesting. Jacobus Pharmaceuticals, the company providing the free 3,4 Dap (active ingredient of Firdapse) that all the CPRX critics point to, has their drug manufacturing facility inside an abandoned nuclear reactor test site! They’ve been repeatedly cited by the FDA over the past 16+ years for manufacturing and safety violations, and even issued an FDA “Warning Letter.” You can see some of these FDA letters above. At the bottom of the website, he includes H.C. Wainwright analyst Andrew Fein’s 2013 report on Jacobus Pharm. He gets down to some of the specific FDA violations and gives a little history of their facility (including a pic of the conical dome): “It housed a laboratory for low-level atomic research. The reactor became obsolete and was decontanimated, and ownership reverted back to to Walker-Gordon Laboratory Company. Today, it houses the offices of Walker-Gordon and the laboratories of Jacobus Pharmaceuticals. Though he applauds Jacobus for the service they provided LEMS patients, he concludes that when you bore down to the cost/benefit of therapy, quality control, and patient vigilance, the compounding landscape was “more like the Wild West.” I wonder why Adam Feuerstein and his Stat reporters never probe deeper on the Jacobus Pharm side of the debate. (Btw, I actually like AF and have a lot of respect for him. He’s a great reporter, calls it like he sees it, and seems to be a genuinely good guy. Except when he’s bashing my stock. Then he’s in cahoots with the hedgies, shorting my stock! lol) Why does AF want to stick with the status quo, excluding four out of five LEMS patients from access to the drug, and with pills possibly contaminated with radiation! (Okay, joking about the last part, but still… Would you want to take pills knowing it was made at that conehead dome?) Good lord.
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CPRX
Feb 7, 2019 17:36:54 GMT
nateb likes this
Post by biposter on Feb 7, 2019 17:36:54 GMT
t.co/twW85KH7IU Aye yai yai. Here’s Sen. Bernie Sander’s letter to HHS & MMS. He is breathing down heavy on us. I’m hoping CEO McEnany anticipated all this and had a good response/PR plan ready. This is a rare orphan drug treating not 30m for cholesterol or 3m for eczema or 300,000 for bowel disease or even 30,000 for some uncommon condition. It’s for only 3,000 people total in the U.S.! 3,000! And only for seven years before generics step in. So what are they supposed to charge after spending tens of millions of dollars getting it through the clinic? And who made them run a second expensive full-on Ph3 trial, even after the first Ph3 had p-values out to four decimal points! The data was excellent, but the FDA made them run another trial and spend boatloads more of cash. Maybe Sen. Sanders would prefer the old way of treating just one out ten LEMS patients with free nuclear pills.
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CPRX
Feb 7, 2019 19:57:18 GMT
Post by nateb on Feb 7, 2019 19:57:18 GMT
New drugs cost a lot of money because it costs a lot of money to develop and get them approved. Why is this so hard for people to understand? Investors demand a return on invested capital, so drug makers have to make money.
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CPRX
Feb 8, 2019 18:52:03 GMT
JHam likes this
Post by biposter on Feb 8, 2019 18:52:03 GMT
So it looks like cuz of Sen. Bernie Sanders’ letter, CPRX was singled out on nationally televised news last night. So, of course, NBC isolates the story of a single LEMS patient and gives no broader context. And after that bleeding-heart report, what does NBC conclude the patient in their story will now end up having to pay? $10 out of pocket as a co-pay. Now, how about ABC News run a report next week isolating a single LEMS patient telling his story about how the onerous FDA requirements for his doctor to file and submit all the paperwork necessary to secure a supply of this non-FDA-approved drug denied him access to this “life-changing treatment,” crippling his quality of life? Until now. www.nbcnews.com/health/health-news/family-outraged-over-life-changing-treatment-going-free-375-000-n968906 Family outraged over life-changing treatment going from free to $375,000 a yearWhen Will Schuller was an 18-year-old senior in high school in Overland Park, Kansas, something puzzling was happening. He was “big into running at the time” and seemingly in good health, but his mile time kept increasing. “I was getting much slower and slower times,” Will, who is a now a senior at the University of Tulsa studying mechanical engineering, told NBC News on Thursday. Eventually, he said, it got the point that within weeks he "couldn't run even half the distance." From there, it only got worse. Will struggled to walk from the school parking lot to his classes, and he couldn’t exercise. “Everyone knew I was this fit guy, and in just a matter of a month you see me struggling to walk from one side of the hallway to the other,” he said. “My sister would have to piggyback me up the stairs.” At that point, Will's parents had to pull him out of school. It took about six weeks for a neurologist to figure out what was going on. Right around Christmas 2014, Will was diagnosed with Lambert-Eaton Myasthenic Syndrome (LEMS), a rare neuromuscular disorder. LEMS is “a chronic autoimmune disorder that affects strength and muscle fatigue ability,” said Dr. Ted M. Burns, a professor of neurology at the University of Virginia. LEMS affects about 3,000 people in the U.S., Burns said, and it can drastically alter the quality of their life. There is a drug to treat LEMS that up until recently was free for patients through an FDA program called “compassionate use.” When Schuller got the drug, called 3,4-diaminopyridine, or 3,4-DAP, he instantly felt better. Ann Schuller, Will’s mother, said that about 40 minutes after her son took the pill for the first time, he was up and celebrating at the Mayo Clinic in Minnesota, where he was being treated. “He skips out to the lobby. And we skip on down to the next appointment. And I'm with the wheelchair trying to follow him back down,” she said. “I don't believe in miracles. But that was pretty darn close to one. ... It was a miracle drug, for sure.” Since 1992, 3,4-DAP, was made by Jacobus Pharmaceuticals, a small New Jersey company, until a different company, Catalyst, recently received the exclusive rights to the drug. Catalyst added a preservative, renamed it Firdapse, and is now charging north of $375,000 a year for the life-changing drug. “This is not a story about innovation. This is a story about exploitation,” Burns said. Will's parents said doctors at the Mayo Clinic warned them about an impending price increase, but they never imagined it would start costing hundreds of thousands of dollars. “We're paying something dramatically less than that,” said Bob Schuller, Will’s dad. “But everyone's premiums are going to go up as a result of this. So it's a cost to the entire system.” Sen. Bernie Sanders, I-Vt., sent a letter to Catalyst on Monday demanding an explanation for the new price of Firdapse, calling it "a blatant fleecing of American taxpayers." However, Catalyst says the cost increase is both legal and reasonable. "Our pricing strategy reflects our focus on ensuring broad and sustainable coverage from both private and public payers and assistance for patients in need," Catalyst CEO Patrick J. McEnany told investors in a conference call in December. Catalyst told NBC News that "most patients will pay approximately $10 out of pocket as a co-pay" for Firdapse each month and said it "will respond to Senator Sanders’ letter in a timely manner." Burns says that while it's true patients won't be bearing the brunt of the price increase, Catalyst bought the drug "knowing that they could charge whatever they wanted if they got FDA approval." “They could charge $10 million and I would have to find a way to pay for it,” Will said. “I don’t think I would be alive today without DAP. I would for sure be in a wheelchair." His mother has a question for Catalyst: "Why is my insurance company having to pay that much money for something that doesn't cost that much to make?" “Is that really the kind of system we want to have?” Ann Schuller asked.
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CPRX
Feb 10, 2019 8:27:29 GMT
Post by biposter on Feb 10, 2019 8:27:29 GMT
Okay, if that patient had a $3,800/mo. co-pay, then I’m fully onboard with all the critics, and CPRX deserves to be vilified. But even as princetonGB noted, CPRX purposely and deliberately set up Catalyst Pathways to deal with such matters. It’s unfortunate this patient wasn’t made aware, so CPRX still has a lot of work to do to inform all current 3,4-Dap patients. But Pathways should be able to address and resolve this exorbitant co-pay and reduce it down to about a $10/mo.
CPRX needs to get out ahead of all this, or they risk becoming the next Martin Shkreli. I'm sure they're monitoring $CPRX on Twitter, so hopefully they'll adequately address this public relations storm.
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CPRX
Feb 21, 2019 20:09:23 GMT
JHam likes this
Post by biposter on Feb 21, 2019 20:09:23 GMT
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CPRX
Feb 26, 2019 19:44:12 GMT
Post by biposter on Feb 26, 2019 19:44:12 GMT
ir.catalystpharma.com/news-releases/news-release-details/catalyst-pharmaceuticals-announces-publication-phase-3-clinical CPRX published its second Ph3 trial data in the Journal of Clinical Neuromuscular Disease.
- “The positive topline results from this trial were previously announced, but the full results of safety, efficacy, and other clinical data are now available online. The LMS-003 trial was a randomized, double-blind, placebo-controlled design. Both co-primary endpoints were met with statistical significance (QMG score: p=0.0004 and Subject Global Impression Score: p=0.0003). This was further supported by similar success with other endpoints (Triple Timed Up and Go walk test and the Clinical Global Impression of Improvement). The study provided statistically and clinically significant evidence that Firdapse was safe and effective in treating LEMS patients.”
www.reuters.com/article/us-usa-healthcare-catalyst/catalyst-pharmaceuticals-defends-375000-drug-price-after-bernie-sanders-rebuke-idUSKCN1QA1XT And, btw, this Reuters article from last week on CPRX’s response to Sen. Sanders is the kind we need out there: short, to-the-point, laying out the criticism, and concluding with the kicker: Catalyst Pharmaceuticals defends $375,000 drug price after Bernie Sanders rebuke
- “The company said, before its November approval, only about 200 of an estimated 3,000 LEMS patients in the US received some form of Firdapse, pointing to a large unmet medical need. ‘Now, for the first time, LEMS patients have confidence their therapy is FDA approved and is safe and effective,’ Catalyst's Chief Executive Officer Patrick McEnany said in a letter (bit.ly/2NkmIRU). The company said that Firdapse’s price was similar to other medicines that provide a significant clinical benefit in treating ultra-rare diseases, adding it believed the drug would be widely reimbursed by insurers for the small population it treats.”
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CPRX
Feb 28, 2019 20:36:00 GMT
Post by biposter on Feb 28, 2019 20:36:00 GMT
Not sure how that works. Can a sitting Senator or the FDA chief, with just the wave of the hand, authorize others to manufacture and distribute an FDA-approved drug? Per the commissioner’s tweet, doesn’t sound like they’ll approve those nuclear pills. Anyway, CPRX’s Pathways program still needs to get the kinks out, and then everyone will have affordable access to Firdapse. Those patients with crazy high co-pays should get some relief by contacting Pathways.
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CPRX
Mar 19, 2019 20:46:37 GMT
Post by biposter on Mar 19, 2019 20:46:37 GMT
ir.catalystpharma.com/events-and-presentations Good CC today.
- Firdapse was commercially launched on Jan 15th. (FDA approved Firdapse for LEMS [~3,000 cases in the U.S.] back in Nov. 2018)
- LEMS patients who were previously on free Jacobus Pharma drug or from compounding pharmacies have not missed a single dose of Firdapse.
- Almost all patients on Firdapse today are now paying an out-of-pocket expense of $10/mo. or less
- CEO: “So far, with few exceptions, every payer that has been presented with a claim, has indicated that they would cover Firdapse, including large, well-known payers such as Express Scripts, Cigna, Caremark, Aetna and Prime Therapeutics, which represents about 25 Blue Cross Blue Shield plans across the country.”
- CEO: “...two months into our launch. We have more than 300 LEMS patients that have been enrolled in Catalyst Pathways and more than 250 patients that are currently being treated with Firdapse. Additionally, 165 unique physicians have written at least one prescription for Firdapse through our Catalyst Pathways patient services hub. And what is especially gratifying is the fact that we've enrolled 39 patients in Catalyst Pathways, who have not previously had access to an amifampridine drug therapy.”
- CEO: “We anticipate that we’ll be in a position to provide revenue guidance for 2019 by the time that we announce first quarter results in early May. At that time, we should have enough data and experience to be able to project with confidence a range of revenues for the full year 2019.”
- Sales force is contacting 1,250 neurologists or neuromuscular specialists about LEMS/Firdapse.
- CCO: “I want to turn now to market access, where we also see positive developments. Market access uptake for Firdapse has progressed well since launch with high levels of formulary placement and reimbursement from the commercial, government and Medicare channels.”
- CCO: “our free bridge medication program via Catalyst Pathways has been working while ensuring that those patients do not experience a lapse in therapy. And importantly, for those patients who have received a positive coverage and reimbursement decision, we see that the average monthly co-pay out-of-pocket amount is only $7 and over 50% of patients are paying zero dollars per month. In fact, we believe that all patients who have reached out to utilize one of the financial assistance programs available to them are paying $10 per month or less for their Firdapse medication.”
- Analyst question re: politics/drug pricing issue. CEO: “as long as we feel that we are delivering value to our patients, which we are, and I think it's being recognized with all the various stakeholders, including physicians, patients, payers, that's what we're most concerned about. And we've not see or experienced necessarily any impact. But, again, it's early in the launch for us, but I'm not sure that that global issue is impacting us.” CCO: “And we don’t see that any of the proposed rule changes are currently causing a financial risk to us. And we're monitoring the progress of a lot of these different proposals. And of course, in the future, if we think it represents a risk, we’ll let you know.”
- Ph3 MuSK-MG top-line data in 2H19 (~3,000-4,800 cases in U.S.). Has SPA designation. Ph2 double-blind trial was positive (p=0.0003; 0.0006). If trial is positive, COO said they would file a supplemental NDA for MuSK-MG in 2020.
- Ph3 CMS top-line data in 2H19 (~1,000-1,500 cases in U.S.). Has Orphan Drug designation. If trial is positive, COO said they would file a supplemental NDA for CMS in 2020.
CEO/mgmt. seem to be doing a very good job on all fronts. No lapse in Firdapse treatment. $7/mo. co-pay, w/ half paying $0. Almost all payers (commercial, gov’t & Medicare) agreeing to cover Firdapse. By end of Q2, they will have reached their full 2019 target of treating all ~300 former 3,4-DAP patients, so they’re six-mo. ahead of schedule. In process of reaching the other 1,200 identified & 1,500 unidentified LEMS patients. Drug pricing/political risk is not impacting them. They’ve done a very good job addressing/answering all critics (critics who now have questions of their own that they’ve never addressed). Trial data results for two other key Ph3 trials in 2H19. Will provide revenue guidance for 2019 on their next ER/CC in May.
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CPRX
May 7, 2019 7:44:26 GMT
Post by biposter on May 7, 2019 7:44:26 GMT
Not sure how that works. Can a sitting Senator or the FDA chief, with just the wave of the hand, authorize others to manufacture and distribute an FDA-approved drug? Per the commissioner’s tweet, doesn’t sound like they’ll approve those nuclear pills. Anyway, CPRX’s Pathways program still needs to get the kinks out, and then everyone will have affordable access to Firdapse. Those patients with crazy high co-pays should get some relief by contacting Pathways.
Whoa, surprising news broke after-hours, crushing the stock. The FDA approved Jacobus’ Ruzurgi (Firdapse-equivalent) in pediatric use for LEMS. I guess Sen. Sanders’ appeal to the FDA was heard. Not sure exactly how this impacts CPRX. Their qtrly CC is on Monday, so we’ll see how they address this.
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