Aussage vom Chef: ?The range of conditions in the firing line of RNAi is impressive and diverse...We can go after diseases and undruggable targets that we could never go after before. There are going to be dozens of new drugs.?
Results: We demonstrate that to achieve deep HIF2? mRNA knockdown (KD), functionalizing HIF2 RNAi with PK enhancement and tumor targeting ligand (TTL) is required. Optimization of the HIF2 RNAi construct enabled a 10-fold improvement in potency. Evaluation of a loading dose regimen improved overall HIF2? mRNA KD compared to a single administration of equal total dosage. Utilizing this strategy, we demonstrated that silencing of HIF2? mRNA (85% KD) resulted in tumor growth inhibition in the A498 xenograft model. Significant improvement in overall survival was also seen in a patient derived xenograft model. Histology evaluation of tumor samples revealed extensive tumor destruction with clusters of apoptotic cells and necrosis. Follow-up studies suggest that loading doses can be administered four hours apart without loss in potency. This allows dosing to be completed in one day and may be more acceptable in clinical settings. The maximum HIF2? mRNA KD after a single dose of HIF2 RNAi was achieved about 7 days after dosing and sustained for about one week in the xenograft model. This suggests that dosing can likely be less frequent in clinical settings. An exploratory toxicity study in rats predicts a wide safety margin. Conclusions: We demonstrate that the TRiM? delivery platform can be utilized to deliver a RNAi therapeutic selectively targeting HIF2? for the treatment of ccRCC. This represents a novel therapeutic approach either as a monotherapy or in combination with other therapies in seeking better tolerated and/or more effective treatment for ccRCC. https://www.abstractsonline.com/pp8/#!/6812/presentation/1356
Arrowhead seeks an Alliance Manager to interface with current and future pharmaceutical partners. The Alliance Manager will be the primary day-to-day point of contact with existing and new strategic partners, as well as intra-company functions related to the relationships with partners. This will include timeline and technical progress communications as well as work progress in compliance with governing collaboration documents. The ideal candidate will be a highly motivated team player ready to contribute to a growing biotech organization.
The position reports to the Vice President, Program Management and will work closely with other senior managers at Arrowhead.
In January 2019, the Company entered into amendments to its existing lease for its Madison, Wisconsin research facility. The amendments add anadditional 13,000 square feet of office and laboratory space to the facility. The increased capacity of this new facility will accommodate increased researchand development personnel and manufacturing capabilities for the Company?s expanding pipeline of current and future drug candidates. See Note 11 foradditional discussion of these amendments
Sustained RNAi reduction of Z-AAT substantially reversed the AATD disease phenotype: Deeply reduced monomeric Z-AAT protein in the liver Reduced polymeric Z-AAT in the liver Prevented the dramatic increase in globule size seen in age-matched control PiZ mice Improved abnormal endoplasmic reticulum morphology Prevented inflammation Prevented/reduced expression of fibrosis, redox-regulation, stress, apoptosis and autophagosome- associated genes Resulted in abundance of healthy mitochondria
JNJ-3989 rapidly reduced hepatitis B surface antigen (HBsAg) in patients that had 24 weeks or more of HBsAg assay results (n=40) to thresholds possibly associated with improved chances of HBsAg seroclearance1 in many patients, after only 3 doses 100% of patients (40 of 40) achieved ?1.0 Log10 IU/mL HBsAg reduction 88% of patients (35 of 40) achieved HBsAg <100 IU/mL 43% of patients (17 of 40) achieved HBsAg <10 IU/mL 13% of patients (5 of 40) achieved HBsAg <1 IU/mL
PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has received clearance from the U.S. Food and Drug Administration to proceed with 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).
Arrowhead intends to initiate the adaptive design, Phase 2/3 study of ARO-AAT in patients with AATD associated liver disease at various sites in the U.S. in the second quarter of 2019, followed by various international sites in Europe, pending regulatory submission and review. The proposed primary objectives are to evaluate safety and pharmacodynamic dose response, and to evaluate efficacy, defined as an improvement in a histologic grading scale of AATD associated liver disease, and no worsening of liver fibrosis based on Ishak score on end of study biopsy. The company plans to provide additional study details following its initiation.
Anyone who is looking for a differentiator in the RNAi space can stop here. This is the first company in the history of the world to receive such an approval. Whether or not Mr. Market gets it is irrelevant (It doesn't). EVERY BP will now have to play ball with ARWR for two simple reasons. 1) Superior proven delivery capability, and 2) The first and only FDA approved commercialization path that allows drugs to move through the clinic without interim filings.
This latter point is completely lost on the investment community. Think about it. ARWR can cruise through the clinic without two previously required filings. That reduces time to market with drugs by many months if not years. Time is money. This reduces commercialization costs by tens of millions for every ARWR drug. Tha's tens of millions that immediately flow to the pre-tax line.
ARO-AAT will be commercialized in 2020. This approval increases ARWR's lead on ALNY's ALN-AAT02 drug by a MINIMUM of a year. The lead now stands at at least 2 years. And every ARWR drug will have a least a year or two advantage over the competition. And what's the competition? Broadly looking at the landscape of companies that MIGHT have competitive delivery instruments it's ALNY and DRNA. And that's a BIG MIGHT. On these down market days it is tough to think about why the market isn't taking this announcement more seriously. Everyone is playing defense. But the value-creation engine at ARWR continues and with balance sheet issues off the table this stock is a generational good bet. https://finance.yahoo.com/quote/ARWR/community?p=ARWR
On April 17, 2019, Arrowhead Pharmaceuticals, Inc. (?the Company?) entered into a new lease for its Pasadena, California corporate headquarters. The 91month office building lease between the Company and 177 Colorado Owner, LLC is for approximately 24,000 square feet of office space located at 177 E.Colorado Blvd, Pasadena, California, and this lease will replace the Company?s current corporate headquarters office lease. The increased capacity of thisnew office space compared to the Company?s current corporate headquarters will accommodate increased personnel as the Company?s pipeline of drugcandidates expands and moves closer to market. http://ir.arrowheadpharma.com/static-files/...-4eed-955d-aeadaf223334
Arrowhead Begins Triple Combination Cohort in Chronic HBV Patients and Earns $25 Million Milestone Payment from Janssen April 24, 2019 07:30 AM Eastern Daylight Time https://www.businesswire.com/news/home/20190424005179/en/ Christopher Anzalone, Ph.D., president and chief executive officer at Arrowhead, said: ?Both Arrowhead and Janssen share the aim to advance transformational medicines that achieve higher rates of functional cure with a finite treatment duration for patients with chronic hepatitis B viral infection. Beginning this new triple combination cohort in our ongoing AROHBV1001 study has the potential to generate valuable data rapidly.?
: webcast and conference call on May 8. 2019
PASADENA, Calif. --(BUSINESS WIRE)--Apr. 24, 2019-- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it will host a webcast and conference call on May 8, 2019 , at 4:30 p.m. EDT to discuss its financial results for the fiscal 2019 second quarter ended March 31, 2019 . http://ir.arrowheadpharma.com/news-releases/...al-2019-second-quarter
Bank of America Merrill Lynch 2019 Health Care Conference ? Las Vegas, May 14-16, 2019
May 15, 8:40 a.m. PDT ? Christopher Anzalone, Ph.D., Arrowhead?s president and chief executive officer, will deliver a corporate presentation
Vascular Discovery: From Genes to Medicine 2019 Scientific Sessions (ATVB|PVD) ? Boston, May 14-16, 2019
May 15, 11:00 a.m. EDT ? So Wong, Ph.D., Arrowhead?s director of biology, will deliver a poster presentation titled, ?Reduction in Angiopoietin-Like Protein 3 via RNA Interference Improves Dyslipidemias and Hepatic Steatosis?
2019 National Lipid Association Scientific Sessions ? Aventura, FL, May 16-19, 2019
May 18, 11:25 a.m. EDT ? So Wong, Ph.D., Arrowhead?s director of biology, will deliver a poster presentation titled, ?Personalized Medicine for Dyslipidemias by RNA Interference-Mediated Reductions in Apolipoprotein C3 or Angiopoietin-Like Protein 3?
20th Annual B. Riley FBR Institutional Investor Conference ? Beverly Hills, May 22-23, 2019
May 23, 8:30 a.m. PDT ? Christopher Anzalone, Ph.D., Arrowhead?s president and chief executive officer, will participate in a fireside chat presentation with Mayank Mamtani, B. Riley FBR research analyst
These are both attractive targets that could address a number of high-value, unmet medical needs, and we are the first to use RNAi against them in humans. They each provide a degree of optionality with respect to which patient populations and indications to study and pursue. For each target, there may be opportunities to treat well-defined orphan diseases such as familial chylomicronemia syndrome and homozygous familial hypercholesterolemia as well as higher prevalence diseases such as NASH or even primary and secondary prevention of cardiovascular disease. We will follow where the data lead us, and dependent on patient populations we choose to focus on, we could have a rapid path to pivotal studies. I expect that we will address clear orphan indications immediately, and I think it is even possible that we could be in pivotal studies for both ARO-APOC3 and ARO-ANG3 next year. We could then begin longer-term studies for larger indications concurrently given us a stage market approach where we move toward orphan markets quickly while we wait for studies of larger indications to mature and eventually go-to-market there as well. Our intention is to discuss our plans for the programs in more detail and describe the potential opportunities and development paths later this year. Importantly, we believe these targets are well suited for RNAi therapy, and we are the first company to use this modality against them in the clinic. We believe that we can build maximum value for our shareholders by focusing on being pioneers rather than followers, and ARO-APOC3 and ANG3 are good examples of this. More broadly, we intend to be first in our target markets where possible. If instances arise where there is an incumbent, we expect to enter a market only if we believe we have clear technological superiority. https://finance.yahoo.com/news/...-earnings-conference-075110997.html
: National Lipid Association Scientific Sessions
May 18, 2019 11:25 AM EDT
§ 2019 National Lipid Association Scientific Sessions
So Wong, Ph.D., Arrowhead?s director of biology, will deliver a poster presentation titled, ?Personalized Medicine for Dyslipidemias by RNA Interference-Mediated Reductions in Apolipoprotein C3 or Angiopoietin-Like Protein 3? http://ir.arrowheadpharma.com/events-and-presentations