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CPRX
May 7, 2019 7:50:48 GMT
Post by biposter on May 7, 2019 7:50:48 GMT
Here’s a good, balanced article from STAT covering this new twist. I think there are three keys from this article: (1) the comment from Piper Jaffray about the legal questions about Ruzurgi infringing on CRPX’s orphan drug exclusivity; (2) will insurers even cover off-label uses when there’s already an approved LEMS drug on the market; (3) Jacobus gave out the drug free for 27 years, but in this article, they’re now saying this will put them $80M in the hole, so how exactly will they now price Ruzurgi?
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In a crafty move, FDA may have found a way to dampen controversy over a $375,000 rare-disease drugThe Food and Drug Administration just added an unexpected twist to a simmering controversy over a rare disease drug that earlier this year briefly became a poster child for high-priced medicines. In a surprise move, the agency approved a medicine from Jacobus Pharmaceuticals, a small, family-run company, for treating a neuromuscular disorder called Lambert-Eaton myasthenic syndrome, or LEMS, for children ages 6 to 17. However, the approval potentially adds unforeseen competition for Catalyst Pharmaceuticals (CPRX), which only last December won an FDA endorsement to market its own treatment for adults. In after-hours trading, Catalyst stock was down as much 44%. Here’s why: The FDA approval of the Catalyst drug, called Firdapse, set off a firestorm. Until then, a few hundred LEMS patients were able for years to obtain the Jacobus drug for free under a compassionate use program sanctioned by the FDA. But with its approval, Catalyst obtained seven years of market exclusivity, which meant Jacobus and compound pharmacies could no longer market their versions.
The move created an uproar because Catalyst decided to charge $375,000 list price, depending upon total patient dosing, for Firdapse. In February, U.S. Sen. Bernie Sanders (I-Vt.), who’s made high drug prices a raison d’etre, accused the drug maker of “corporate greed” and “immoral exploitation.” And he asked the FDA to permit Jacobus and compounders to resume suppliers of their treatments.The agency appears to have heeded the call. Jacobus has not yet disclosed a price for its drug, called Ruzurgi. But by approving it for children, the agency is making it possible for physicians to prescribe the Jacobus drug for any patient, regardless of age, because doctors are free to prescribe medicines for unapproved or so-called off-label uses. Interestingly, the FDA approval was predicated, in part, on data from studies involving adults. “If it’s on the market for children, it can be prescribed for adults,” said Dr. Donald Sanders, a Duke University researcher who worked with the Jacobus family when they first developed their LEMS drug and later helped design the clinical trial that was submitted to the FDA. “I don’t know of any drug that is approved for adults with LEMS other than Firdapse, but we already use many other medicines off label to treat LEMS. This sounds like a workaround.” An FDA spokeswoman sent us this: “The decision to treat a patient with a drug for an unapproved use is up to the treating health care professional and generally speaking, the practice of medicine is not regulated by the FDA. Good medical practice and the best interests of the patient require that physicians use legally available drugs, biologics, and devices according to their best knowledge and judgement.” Meanwhile, Piper Jaffray analyst Joseph Catanzaro wrote this: “While this is a different label indication than Firdapse’s adult LEMS label, it will no doubt raise questions around whether Ruzurgi will be used off-label in adult patients and whether Firdapse will be able to maintain the orphan drug price point it set at launch. However, we suspect that there will be legal questions around whether the approval of Ruzurgi infringes on Firdapse’s orphan drug exclusivity in LEMS.”
“We see this approval by the FDA simply as a way to combat the pricing rhetoric that has surrounded Firdapse since its approval late last year and bring a potential competitor to the market,” he continued, adding that there are “questions around whether the adult and pediatric populations represent two distinct orphan diseases.” The uncertainty helps explain investor reaction to the FDA approval, which was announced late Monday. A spokesman for Catalyst, which had indicated there are as many as 3,000 LEMS patients in the U.S., declined to comment. However, there are some extenuating factors. For one thing, a doctor may prescribe the Jacobus drug, but that doesn’t mean an insurance company will automatically provide coverage. Sometimes, insurers will not cover off-label use, although in this instance, the price of the Catalyst drug may provide some impetus — if the Jacobus drug costs significantly less.
As of Monday night, Laura Jacobus, who runs the privately held company, said a final decision hasn’t been made. “I haven’t given it much thought. This decision just came in, but obviously, it will be less than what they’re charging,” she told us. “But we’re carrying over the last 27 years the research, development, the compassionate use manufacturing. We’re probably $60 million in the hole. And the post-approval commitments are probably (going to cost) $10 million to $20 million.”However, she declined to discuss off-label usage. “We assume we can’t take care of adult patients, but we’d like to take care of pediatric patients,” she said. “That’s a role we’re pleased to provide.” One patient who had been taking the Jacobus drug under the compassionate use program but was forced to switch to Firdapse, told us she is excited by the approval. She is among several Firdapse patients who have complained the medicine is not as effective as the older Jacobus treatment. “Oh my gosh, this is very good news,” said Rebecca Hovde, who lives in Iowa. “I will definitely ask my doctor to switch to the Jacobus drug now.”
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CPRX
May 7, 2019 17:16:40 GMT
JHam likes this
Post by biposter on May 7, 2019 17:16:40 GMT
Here’s Oppenheimer’s report today on $CPRX (underlining/bolded mine). Looks like the key risk is how payers position Ruzurgi vs Firdapse. Surprise Jacobus Approval, But Unclear Impact On FirdapseThe approval of Jacobus' Ruzurgi (amifampridine free base) for the treatment of pediatric patients with LEMS comes as a surprise, as investors had all but written off this version of 3,4-DAP following Firdapse's approval (for adults, who comprise nearly all of the LEMS population) under the presumption that other forms would be blocked by orphan exclusivity. With CPRX shares -43% after Monday's close, the market appears to be factoring significant impact to Firdapse's commercial prospects from off-label use of a (presumably cheaper) agent. As we endeavor to better understand implications to CPRX, we maintain our Outperform rating as our initial impression is that of an over-reaction to the downside that may be driven more by fear than by fact. KEY POINTS ■ Questions remain, and with Firdapse's early launch indicating that access to paid drug has not been a meaningful issue for those either transitioning from EAP material or coming onto therapy anew, we are most focused on potential moves by payors that could position Ruzurgi ahead of Firdapse, i.e., as a formulary stepthrough, despite being an off-label use for most LEMS patients. ■ We may learn more about what may be a Firdapse/Ruzurgi dynamic on CPRX's 1Q results conference call, to be held Monday 5/13 at 8:30am ET shortly after the financials are disclosed at ~6am ET. ■ We expect to hear Firdapse's launch continuing on the strong trajectory described on the company's 4Q18 call held March 19, which will likely include sales as well as other metrics e.g. patient starts.
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CPRX
May 8, 2019 22:35:15 GMT
nateb likes this
Post by biposter on May 8, 2019 22:35:15 GMT
SA poster Joel2040 is saying that Jacobus’ Ruzurgi will be priced starting at ~$200k, maybe higher. I don’t know where he got the figure from (an analyst report not yet made public?):
- “it is not the same drug there are compound differences. One important factor is that Firdapse comes in a pill form that Jacobus does not have and the drug from Jacobus must be refrigerated when not in use Firdapse requires no refrigeration. The other differences noted in the Jacobus drug was that there were was an increase in seizures in patients taking their medicine vs Firdapse. Last but not least Jacobus drug will cost in the 100's of thousands of dollars too. Jacobus is throwing around a price starting at $200,000 per script but it will probably go higher based on the $375,000 being charged for Firdapse. So in the End all of Bernie's complaints and threats lead to what? 2 drugs that are high priced. At least people with LEMS disease have options available and with insurance coverage affordable to them.” seekingalpha.com/news/3459579-catalyst-pharma-minus-42-percent-fda-oks-firdapse-competitor?app=1&dr=1#email_link
So we’ll have to see what CPRX says on their CC this Mon., 5/13. From what I understand so far, reading what little info from analyst reports that have been posted publicly, I think these are the next steps we’ll have to watch CPRX navigate:
- Piper Jaffray analyst Joseph Catanzaro said: “However, we suspect that there will be legal questions around whether the approval of Ruzurgi infringes on Firdapse’s orphan drug exclusivity in LEMS.” So, first, we’ll have to see if CPRX mounts a legal challenge. If so, the downside is this could be a bad public relations move.
- Assuming no legal challenge, next we’ll have to see how Jacobus Pharma prices Ruzurgi. Laura Jacobus herself said in the STAT article that they’re ~$80M in the hole, so she seems to be guiding that it won’t be cheap. And if poster Joel2040 is correct, and pricing starts at ~$200k, then maybe the shine on Jacobus’ Robin Hood image starts to fade. Now we have two expensive LEMS drugs. Then would CPRX respond by dropping its list price to compete? If so, the fact that Firdapse is more stable and doesn’t have to be refrigerated is a big advantage. The LEMS patient isn’t tied to his home and can go about his life with Firdapse in pill form (eight tablets per 24-hr period). However, if Jacobus really undercuts CPRX and prices it, say, <$100k, then would CPRX be forced to cut its drug price by two-thirds/three-fourths, making a real impact on projected revenues?
- Then down the road, we’ll have to see how insurers respond to off-label prescriptions of Ruzurgi for adult LEMS patients. Unless there’s a huge price discrepancy, will payers cover a less-stable drug that needs refrigeration when a much more convenient version is available? But if there is big price difference, then as Oppenheimer said, would payers “position Ruzurgi ahead of Firdapse, i.e., as a formulary stepthrough, despite being an off-label use...”
- CPRX has two Ph3 trial readouts in the second half of this year. One for MuSK-MG (~3,000-4,800 cases in U.S.) and another for CMS (~1,000-1,500 cases in the U.S.). Both of these had Ph2 trial p-values at <0.0007. CMS has Orphan Drug designation, and MuSK-MG has an SPA designation. Both have no approved therapies, and CPRX has guided that if positive, they will go to the FDA to expand the Firdapse label for both indication in 2020. But then we face the same off-label issue with Jacobus’ Ruzurgi for these two other indications. So, again, it comes back to how Jacobus prices its drug.
Lots of new risks now to consider.
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CPRX
May 14, 2019 7:32:59 GMT
Post by biposter on May 14, 2019 7:32:59 GMT
ir.catalystpharma.com/news-releases/news-release-details/catalyst-pharmaceuticals-announces-first-quarter-2019-financial Q1 ER/CC today:
- $12.4M net revenues in Q1. Net loss: $645k.
- They “expect to substantially increase [Q2’s] revenue” over Q1’s.
- COH: $50.6M (as of 3/31/19)
- 380 LEMS patients now active on Firdapse.
- 306 out of the 380 are receiving insurance coverage
- 81 out of the 380 LEMS patients had never had access to amifampridine before and are now on Firdapse
- There are over 1,500 diagnosed LEMS patients. They’re now going after the remaining 1,200. “we've done a pretty exhaustive claims data analysis, and in the most recent two years of claims data from a very good source, we saw - approximately 1,500 patients that have been diagnosed.”
- Then there are another estimated 1,500 undiagnosed or misdiagnosed LEMS sufferers, who they’re also going after
- They’re contacting ~1,200 neurologists/neuromuscular specialists who have a high potential of having a LEMS diagnosed patient
- “we have seen continued and additional positive coverage by insurers such as Express Scripts, Cigna, Caremark, Aetna, United Optum and Prime Therapeutics, and now Kaiser as well as the VA and TRICARE.”
- “We care about the patient’s ability to pay for this medication, and if they don’t have insurance or are underinsured, they will be eligible for the very generous criteria into our patient assistance, or PAP program, or free medication.”
- “average monthly copay in Q1 was $5.66 a month”
- Top-line data for Ph3 MuSK-MG trial in 2H19. Also, top-line data for Ph3 CMS trial in 2H19. If positive data, will submit supplemental NDA to FDA to expand Firdapse label in 2020.
- Re: Jacobus’ Ruzurgi approval by the FDA for pediatric LEMS:
- They said they were “extremely surprised with the FDA’s decision to approve Jacobus Pharmaceuticals’ NDA for amifampridine in the treatment of LEMS in a pediatric population between the ages of 6 and less than 17. According to a Jacobus spokesperson there are less than 15 pediatric LEMS patients in the U.S. We are currently assessing our options and we expect to have more to say in that regard in the coming days."
- “We believe that this action by the FDA may affect whether pharmaceutical companies will invest in the development of other therapies to treat patients suffering from ultra-rare orphan designated diseases. Our management and legal teams along with our regulatory advisors are actively assessing the impact of the decision on our Company. The legality of the FDA’s actions and our options going forward. We will have more to say about this subject in the very near future.”
- In response to an analyst question: “I've never seen before a policy that where insurance company put something that's off label on to a formulary or policy, but we'll see how that plays out.”
- Piper Jaffray’s Joe Catanzaro: “But maybe just a question around your interactions with the FDA, I was just wondering, when you applied did the FDA or you guys ever bring up the potential for a pediatric label? And if I look at your FDA approval letter, it specifically states in there that because of your Orphan Drug Designation, you're exempt from having to test the efficacy, and safety and the claims indication in pediatric patients. Just wondering how you guys read that wording?” In reply, basically, a “no comment” from CPRX. They said they’ll say more in the coming days.
- Again, Piper Jaffray’s Joe Catanzaro: “Okay, fair enough. Maybe just one more, would you be able to point to anything similar where the FDA has approved the drug for pediatric indication the way they did for Jacobus or if there's any specific cases that have been litigated that you can point to?” CPRX: “we’ve not at this point.”
- In 10-Q, looks like CPRX is filing for Firdapse approval in Canada, as well.
So they did $12.4M in revenues in Q1. They said revenues will “substantially increase” in Q2. Firdapse costs ~$300k/yr (after discounts & rebates), which equals ~$25k/mo per patient. Firdapse officially launched on 1/15, but became widely available on 2/4. So for many/most patients, insurance covered Rx for maybe a month or two, probably. But CPRX should get a full quarter’s reimbursement for those 306 patients (and counting) for Q2’s E/R, which equals $22.95M. They were going to give guidance on projected full year’s revenue, but the Jacobus Ruzurgi FDA approval forced them to delay that. Now, unless different payers (say, commercial vs. gov’t) pay different amounts for the same drug, sticking with only those 306 at $300k/yr., that equals ~$90M in revenues for FY2019. But they’re now also going after the other 1,200 diagnosed LEMS patients. No guidance yet, but how many insured patients will be paying for the drug by the end of the year? Looking at Piper Jaffray’s Joe Catanzano’s great questions and CPRX’s guarded response, it seems to me that they’re really considering taking legal action. Not sure what kind of reputational hit they might take. And not sure how successful one can be taking on the FDA lol. But it looks like the FDA overstepped their bounds with this maneuver. And CPRX has said that they’ve never seen “an insurance co. put something that's off label on to a formulary…” So it seems like that risk is slight, but you never know. But the biggest risk right now is if Jacobus preempts CPRX’s (possible) legal maneuver and makes a sudden and surprising announcement, coming out with Ruzurgi pricing for real cheap at, say, $50k that really undercuts Firdapse. That would crush the stock, at least temporarily.
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CPRX
Jun 12, 2019 17:57:05 GMT
JHam likes this
Post by biposter on Jun 12, 2019 17:57:05 GMT
CPRX is suing the FDA lol. And Jacobus Pharm priced their drug Ruzurgi at ~$187,000/yr. From free to almost $200k. Will STAT now call out Jacobus as unethical, price-gouging opportunists? After all, they didn’t develop/invent amifampridine, as well. Well, not sure if you want to tick off the FDA lol. They have two pivotal Ph3 trial readouts at eoy, with excellent p-values in their Ph2 trials, and now let’s watch the FDA reject those two indications lol. But the FDA did overstep their bounds with that Jacobus Ruzurgi approval. It looks like the analysts didn’t think insurers would even have covered Ruzurgi off-label, so was this suit really necessary? Just hope this move doesn’t tick off Sen Sanders and put CRPX in the political crosshairs again.
- In a note published last month, SunTrust Robinson Humphrey’s Edward Nash suggested that despite suggestions to the contrary, “(W)e have not seen any precedent where payers cover an off-label drug for use in unapproved patient population”.
- Meanwhile, HC Wainwright’s Andrew Fein suggested that Jacobus, despite the reputation of a “modern-day Robin Hood” is not equipped with the infrastructure nor the experience to support a commercial push of Ruzurgi, even with the approval in hand.
- “Handicapped by legal reasons, we do not believe that Jacobus can openly promote off-label use in LEMS adults…It is unclear if and how Jacobus would be able to expand commercial penetration other than through promoting a proliferation of off-label use at academic centers that were enrolled in the compassionate program (which mounts to approximately 200 patients),” he wrote in a May note.
- A rival drug to Catalyst Pharmaceuticals' Firdapse has a low chance of being reimbursed by payers for its use in adults with Lambert-Eaton myasthenic syndrome (LEMS), SunTrust Robinson Humphrey analyst Edward Nash says CPRX won approval for its drug Firdapse in November to treat adults with LEMS, a rare autoimmune disorder
- "Physicians are unlikely to prescribe and payers are unlikely to reimburse Ruzurgi given Firdapse is the only approved product for adult LEMS patients," Nash says "We believe shares of CPRX are significantly undervalued at current levels," he says (TesttheWaters, ST, 5/31)
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CPRX
Aug 8, 2019 18:11:27 GMT
JHam likes this
Post by biposter on Aug 8, 2019 18:11:27 GMT
ir.catalystpharma.com/news-releases/news-release-details/catalyst-pharmaceuticals-announces-second-quarter-2019-financialGood qtr CC today:
- $28.8M revenue in Q2 ($12.4M rev in Q1)
- $11M net income in Q2 ($6M net loss in Q1)
- 409 LEMS patients now on Firdapse
- COH: $64.9M (as of 6/30/19); no debt
- Ph3 CMS data readout in 2H19 (but Ph3 MuSK-MG data results pushed back to 1H20, but full enrollment by YE19)
- “Global expansion of Firdapse for LEMS underway in Canada and Japan”
- Out of a total of 223 surveys from Firdapse patients, Catalyst Pathways received an avg score of 4.8 (on a 5 pt scale). 88% rated CP a “5.”
- Japan (after certain milestones are met, then expansion into most of Asia, South & Central America)
- New patients on a steady, more measured pace:
- No FY19 revenue guidance
- 223 unique prescribers have written at least one Firdapse script
- 138 3,4-DAP-naive patients are now on Firdapse (or just about to)
- CEO stressed they’re goal is that *all* adult LEMS patients have easy and affordable access to Firdapse. Not a single patient enrolled in CP has failed to receive Firdapse. Not one.
- Every LEMS patient on CP is assigned a personal CP liaison to help navigate prescription coverage, etc.
- 100 neurologists (who previously didn’t have ready access to 3,4-DAP) have now prescribed Firdapse
- 95% coverage approval. Those denied coverage given free Firdapse by Catalyst
- Avg patient co-pay: $5.60/mo.
- They expect to enroll about 15 patients per mo. Their goal is to have 500 patients on Firdapse by eoy.
- Now doing the hard work of bringing awareness to neurologists, patients about this rare, orphan disease
So good qtr all around. So ~15 patients/mo. w/ goal of 500 insured patients by eoy. So slower, steady revenue growth. They did have operating profits of $11M in Q2. Looks like locating the remaining 1,000 identified LEMS patients will take some time. Japan approval seems like it won’t happen until eoy. Ph3 CMS top-line data eoy is a catalyst. So maybe a continued slow, gradual pps climb. Was hoping it’d be $7-$8 by now. One downside risk is if they lose the lawsuit against the FDA, but I think they’ll win. We’ll see.
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CPRX
Oct 30, 2019 18:47:37 GMT
JHam likes this
Post by biposter on Oct 30, 2019 18:47:37 GMT
CPRX’s data on its Ph3 CMS trial failed. It’s such a rare disease, their trial size was only n=20 while taking four years. Their CSO said with so many CMS genetic sub-types, it was difficult to achieve statsig with n=20. Their Ph3 MuSK-MG trial data readout is still on schedule for 1H20 with enrollment completion by eoy. Unlike CMS, their MuSK-MG double-blind Ph2 trial had p-values of 0.0003; 0.0006. Plus, MuSK will have an n=60, so this one should be a much safer bet. And MuSK has ~3,000-4,500 cases in the U.S. CMS had ~1,000-1,500. Anyway, their Q2 ER should be next month. But their LEMS build-up is now only incremental, so I’m not expecting great numbers or anything. Their botched capital raise (supposedly for potential acquisitions, I believe) a month or so ago crushed the pps momentum, and then today’s failed results sank it even further. Still, they’re profitable, and hopefully we get good news of a Firdapse approval for LEMS in Japan and Canada in the coming months. Still have the FDA lawsuit risk. Catalyst Pharmaceuticals Announces Top-Line Results of CMS-001, a Phase 3 Trial of Firdapse® (Amifampridine Phosphate) in Patients with Congenital Myasthenic Syndromes (CMS)
October 30, 2019 at 6:03 AM EDT
- CMS-001 trial is the first and only double-blinded, placebo-controlled study ever conducted in genetically confirmed CMS patients
- Results in full population across all tested subtypes of CMS did not achieve statistical significance for the primary or secondary endpoints
- MuSK-Myasthenia Gravis Phase 3 trial and SMA Type-3 proof of concept study both remain on schedule
CORAL GABLES, Fla., Oct. 30, 2019 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (Nasdaq:CPRX), a commercial-stage biopharmaceutical company focused on developing innovative therapies for people with ultra-rare debilitating, chronic neuromuscular and neurological diseases, today announced top-line results from CMS-001, a Phase 3 study evaluating amifampridine phosphate for the symptomatic treatment of genetically confirmed Congenital Myasthenic Syndromes (CMS) in adults and children aged 2 years and above. The Company’s lead product, Firdapse® (amifampridine phosphate), is currently approved for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adults, and amifampridine phosphate is being investigated for the treatment of CMS and other neuromuscular and neurological disease to support applications to FDA for these indications. CMS-001 is the first ever double-blind, placebo-controlled, clinical trial conducted in genetically confirmed CMS patients. In the trial, 20 subjects were enrolled and 16 randomized, in a 2 period, 2 treatment crossover study designed to evaluate the efficacy and safety of amifampridine phosphate in patients (aged 2 years and above) diagnosed with certain genetic subtypes of CMS. While individual patient improvements were observed in some patient sub-groups, the trial did not meet its primary endpoint of subject global impression (SGI) or the secondary endpoint of muscle function measure (MFM) across all tested subtypes. Due to the rarity of CMS, this trial took almost 4 years to recruit. “While we are disappointed that this trial did not reach its primary or secondary endpoints in the evaluated CMS patient subtypes, we are pleased with the new valuable clinical information that these results will provide to the medical and scientific communities as we work to develop FDA-approved treatment options for patients with this disease” said Patrick J. McEnany, Chairman and Chief Executive Officer of Catalyst Pharmaceuticals. “We also remain committed to developing FDA-approved treatment options for other rare neuromuscular disorders.” Catalyst is scheduled to meet with the FDA before the end of the year to discuss the outcome of this clinical trial, and potential paths forward to seek approval of amifampridine phosphate for the symptomatic treatment of some subset of genetic subtypes of CMS. After receiving additional guidance, Catalyst will provide future updates on its plans for this potential indication. The Company also reports that it remains on track to complete enrollment in its clinical trial of amifampridine phosphate in patients with anti-MuSK antibody positive myasthenia gravis (MuSK-MG) before the end of this year, and to report top-line data from this trial in the first half of 2020. We also expect to report top-line results from our SMA Type-3 proof of concept study in the first half of 2020. Both of these diseases have a much more homogeneous patient population than CMS and should not present the same challenges. “We have made significant progress in genetically testing patients who were unable to previously get diagnosed,” added Steven Miller, Ph.D., Chief Operating Officer and Chief Scientific Officer of Catalyst Pharmaceuticals. “Due to the small patient prevalence, the low number of patients tested, and heterogeneity of the disease with a wide range of variation in clinical presentation across its more than 50 subtypes, it was challenging to demonstrate a statistically significant benefit across multiple subtypes.”
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