Kurs wird jedoch in NY gemacht. Firma ist im Gegenteil zu anderen Biotecs wirklich durchfinanziert und hat mit seinen Partner gutes Potential die Entwicklungen auch am Markt zu positionieren. Es ist ein Markt mit Mrd.Umsätzen. Der Pfeilkopf entwickelt sich dann zur CashCow. In einigen Jahren belâcheln wir dann die kurzfristige Kursrelevant.
Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1 Study of ARO-APOC3 for Treatment of Hypertriglyceridemia January 07, 2019 07:30 AM Eastern Standard Time https://www.businesswire.com/news/home/20190107005514/en/ Chris Anzalone, Ph.D., president and CEO of Arrowhead Pharmaceuticals, said: ?ARO-APOC3 will be our fifth product candidate leveraging the Targeted RNAi Molecule, or TRiMTM, platform to enter clinical studies. Addressing hypertriglyceridemia through reduction of liver-produced apoC-III offers multiple potential development opportunities, including both orphan indications, such as FCS, and large market indications. In AROAPOC31001, our first-in-human study, we intend to evaluate ARO-APOC3 in healthy volunteers and various patient populations with elevated triglycerides, which will help inform our strategy around the ideal development and regulatory paths to pursue.?
Bruce Given, M.D., chief operating officer and head of R&D at Arrowhead, said: ?We continue to advance our broad pipeline of RNAi therapeutics with the dosing of the first subjects in our Phase 1 study of ARO-ANG3. ANGPTL3 has emerged as a potentially important target to address dyslipidemias, such as elevated cholesterol and triglycerides that are not well-controlled with currently available medicines, and also metabolic diseases, such as NAFLD and NASH. The preclinical data for ARO-ANG3 have been promising, and our prior clinical results from the ARO-HBV and ARO-AAT programs, which also leverage the Targeted RNAi Molecule, or TRiMTM, platform, give us confidence in the potential for ARO-ANG3 to address significant unmet medical needs.?
Bruce Given, M.D., chief operating officer and head of R&D at Arrowhead, said: ?Patients with severe hypertriglyceridemia, and particularly patients with Familial Chylomicronemia Syndrome, or FCS, do not have adequate treatment options. Due to its activity as a triglyceride regulator, apoC-III has the potential to be an important therapeutic target for cardiovascular disease. The design of the ARO-APOC3 Phase 1 study is intended to provide a readout on safety and tolerability, as well as a robust view of the pharmacologic activity and duration of effect. ARO-APOC3 is Arrowhead?s second cardiometabolic candidate to enter clinical studies this year, following ARO-ANG3 for the treatment of dyslipidemias and metabolic diseases. It also represents the fifth clinical stage RNAi therapeutic derived from our proprietary Targeted RNAi Molecule, or TRiMTM, platform.?
PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for an adaptive Phase 2/3 trial with the potential to serve as a pivotal registrational study of ARO-AAT, the company?s second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).
Bruce Given, M.D., chief operating officer and head of R&D at Arrowhead, said: ?Our discussions with the FDA have been productive and helpful as we moved through the process of determining the Phase 2/3 adaptive trial design with appropriate surrogate endpoints. We look forward to initiating this trial and moving closer to potentially providing patients, physicians, and the alpha-1 community with a much-needed therapy for alpha-1 liver disease.
vorstellen, das sie dämnächst sogar schon ein Verkaufsteam für die USA aufbauen, und für den Rest der Welt einen fetten Deal machen? Bei der Geschwindigkeit die Arrowhead jetzt an den Tag legt, müsste Big Pharma schlange stehen? :-)
Arrowhead Pharmaceuticals, Inc. is committed to bringing new investigational medicines to patients with serious diseases as quickly and efficiently as possible. Arrowhead?s expanded access to investigational medicinesisa means by which investigational medicines are made available, under certain circumstances, to treat patients with serious diseases who are ineligible or unable to participate in an ongoingArrowhead clinical trial. https://arrowheadpharma.com/wp-content/uploads/...Expanded-Access.pdf
J, I wrote this on 3/13. Partially explains why SP is not higher.
"Danny, are you trying to tell us that markets are efficient? I have been an analyst/portfolio manager (now semi retired and doing other stuff) for 25+ years. I have made a living and then some off market inefficiency. I can tell you from the genius analyst's answers to recent and past questions to ARWR that they haven't a clue. And why should they? The JNJ deal took any possibility of a large underwritten deal off the table for the next five+ years. Do you think analysts get paid for being right? From picking stock winners and losers? No No. They get paid by bringing in banking deals. So why take the time to get really smart on a small development stage company when there is no banking business in it? No paycheck in it?
Getting back to market inefficiency...I wrote a bunch of put options on ARWR and took in a bunch of premium exactly because the market assigned an incorrect price on the underlying stock and a totally distorted price on the related options. Those puts expire Friday. I am going to do same on Friday. It's called adding Alpha. I can tell you that I cannot add one basis point of Alpha to the FANGS. Mr. market has appropriately sorted out their market caps/share prices. But ARWR is largely undiscovered and thus does not have an appropriate valuation assigned to it by Mr. Market. On a risk-adjusted basis this is the best opportunity I have seen in my career. The sell-side analysts don't give a hoot about it. It's too small for a large swath of the major mutual funds and pensions to care because owning 5% of a $2Bn market cap doesn't move the dial at a $30Bn fund. So while the "Market" trades ARWR shares at $19.50 or so, a HUGE part of the market is completely sidelined for structural reasons. Gotta go figure out what is the next put writing plan I need to execute."
J, In short, there is no one event that will all of a sudden drive the SP remarkably higher. I can say with certitude what SHOULD IMO drive the SP higher. FDA approval of what I have termed, "The Gottleib Construct. (GC)" GC is is the seamless transition from p2 to p3, in between which it is determined that both primary and secondary endpoints have been met. Normally when this happened a company must apply for early termination of a trial such that it can unblind all subjects and give drug to placebo patients. Then the company may file and IND for commercial compassionate use labeling in a p3 trial that is a called a post-marketing trial. In other words, this aggressive but possible process has included two filings. What I believe ARWR has filed to do is ask that the FDA allow ARWR to simply append their p2/3 filing with new data from the p2 part of the trial that proves out both primary and secondary endpoints have been achieved. Further, they have asked that the FDA grant commercial labeling without a p3 application and NDA. So rather than a shutdown filing and a p3 IND/NDA, ARWR is looking for what it has termed a seamless transition from p2 to pivotal p2 to p3 with no additional filing. Simply more robust data, all details of which would have been spelled out in the filing that ARWR announced on 3/18/19 after a huge interactive effort between the company and the FDA. I believe that is why BG and CA both emphasized that we will NOT likely see data from this study in 2019. Again BG emphasized that there would be a lot of subjects in the study bc for the FDA to grant the above they would have to have more than the standard data set they would accept for a normal orphan p2 study.
This would be a monumental grant if in fact the FDA allows this study to take place as I have suggested. It will affect not just ARO-AAT but also ANG3 APOC3 which appear to have been designed with a similar path in mind. I have further postulated that AMGN's recent changes to its AMG-890 LPa study was made to follow the ARO-AAT path - patients reduced for the p1 portion of the study. We'll see what the FDA grants but if they grant the above, this will shave years and many millions of development/commercialization dollars. The compressed time is most relevant and is 100% in line with Gottleib's vision for the FDA which is to get many more drugs on the market to create more competition to drive down drug prices. This is also Trump's vision and a big part of what I believe will be his proposed healthcare overhaul. I think we will know inside of 60 days. It is for this reason I believe the approval of this IND SHOULD power up the SP. First time in the history of the world and surely the rest of world will follow the FDA's lead.
Looking forward, we expect to accomplish the following: file 2-3 new CTAs every year; target a new cell type with the TRiM? platform every 18 months; and, have 10 TRiM? enabled candidates in clinical studies by the end of 2020.? ir.arrowheadpharma.com/node/13976/pdf Expanding extrahepatic capabilities to now include delivery to the lung, tumor, and muscle tissue