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drulletje drie 15 november 2019 16:25

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quote:
RW1963 schreef op 15 november 2019 15:53:
ai, ai, ai, dit doet even pijn aan de ogen :-(

Niet erg beetje omlaag na deze stijging! Wel zaak dat we volgende week weer beetje blijven liggen!

Tom3 15 november 2019 18:39

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quote:
RW1963 schreef op 15 november 2019 15:53:
ai, ai, ai, dit doet even pijn aan de ogen :-(

Gewoon een zonnebril opzetten.

Het lijkt wel of een CMO niet met pensioen mag gaan. Given gaat pas in het voorjaar van 2020 weg en heeft alle tijd de nieuwe mensen in te werken.

ir.arrowheadpharma.com/node/14961/pdf

Tom3 16 november 2019 22:41

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Voor de mensen die graag willen weten waarin ze investeren:
www.ncbi.nlm.nih.gov/pmc/articles/PMC...

Het beschrijft uitstekend de toekomst van RNAi tov de gevestigde antilichaam en small molecule collega's. Nu het probleem van de "delivery" in de cel is opgelost, wordt het vinden van de "targets" de grootste uitdaging. Arrowhead ligt hier kop. Echter een terugkeer van een Roche valt ook niet uit te sluiten nu ze recentelijk de Galnac "delivery" techniek hebben gekocht van Dicerna. Eerder hebben ze $ 2,4 miljard neergeteld voor een onderneming die zich heeft toegelegd op DNA-RNA analyse (Foundation Medicine Inc).

Tom3 18 november 2019 14:53

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Novo Nordisk sluit een ontwikkeldeal met Dicerna. De RNAi sector wordt steeds gevraagder. Ik heb ook de indruk dat de prijzen voor dergelijke deals omhoog vliegen.

drulletje drie 18 november 2019 15:37

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Arrowhead Presents New Clinical Data on Cardiometabolic Candidates ARO-APOC3 and ARO-ANG3 at AHA Scientific Sessions 2019

- Company Expects to Initiate Phase 3 Clinical Trials Next Year
PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals Inc. (ARWR) today presented updated Phase 1 clinical data on its two RNAi-based cardiometabolic candidates, ARO-APOC3 targeting apolipoprotein C-III (APOC3) being developed as a potential treatment for patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS), and ARO-ANG3 targeting angiopoietin like protein 3 (ANGPTL3) being developed for the treatment of dyslipidemias, such as homozygous familial hypercholesterolemia (HoFH), and metabolic diseases. The data were presented in two late-breaking oral presentations at the American Heart Association (AHA) Scientific Sessions 2019, in Philadelphia.
Bruce Given, M.D., chief operating officer and head of R&D at Arrowhead, said: “The data presented at AHA on cardiometabolic candidates ARO-APOC3 and ARO-ANG3 are further validation of the TRiM™ platform and Arrowhead’s ability to consistently develop RNAi therapeutics that achieve deep and durable levels of gene knockdown across a broad range of targets. In particular, the knockdown in APOC3 and ANGPTL3 proteins and the resulting reductions in triglycerides and various lipid parameters strongly support our plan to initiate Phase 3 studies in 2020.”
Presentation Details:
RNA Interference Targeting Apolipoprotein C-III Results in Deep and Prolonged Reductions in Plasma Triglycerides
Session: Late Breaking Science VI: New Frontiers in Lipid Therapy
Date and Time: November 18, 2019 from 9:32 AM EST
Authors: Dr. Christie Ballantyne, presenting on behalf of Dr. Christian Schwabe, et al.
Key points presented on the AROAPOC31001 Phase 1/2a clinical study included the following:
Safety and tolerability
40 subjects enrolled to receive a single dose (24 active, 16 placebo)
No serious or severe adverse events (AEs) reported
One AE of moderate transient ALT elevation (peak of 210 U/L on Day 22) in a subject receiving ARO-APOC3 who had elevated ALT at baseline (65 U/L), with return to baseline by Day 85 (61 U/L).
8 Local Injection Site Reactions (LISRs) – all rated mild, more common at higher doses
Activity
Dose dependent reductions in serum APOC3 were observed
Mean maximum reduction from baseline in serum APOC3 levels ranged from 72% [10 mg dose] to 94% [100 mg dose]
Reduction in serum APOC3 levels was maintained through the end of study with week 16 mean reductions of 70% [25 mg dose] to 91% [100 mg dose]
Reductions in triglycerides (TGs) and VLDL-C were observed
Mean maximum reduction from baseline in serum TGs ranged from 53% (77 mg/dL) [10 mg dose] to 64% (92 mg/dL) [100 mg dose]
Mean maximum reduction from baseline in serum VLDL-C ranged from 53% (16 mg/dL) [10 mg dose] to 68% (19 mg/dL) [50 mg dose]
Reduction in serum TG and VLDL-C was maintained through the end of study, with week 16 mean reductions of 41%-55% for TG and 42-53% for VLDL-C
Changes in LDL- and HDL- cholesterol were observed
Mean maximum reduction from baseline in serum LDL-C of 12% (19 mg/dL) [25 mg dose] to 25% (35 mg/dL) [10 mg dose]
Dose dependent increase in serum HDL-C with mean maximum increase from baseline in serum HDL-C from 30% (13 mg/dL) [10 mg dose] to 69% (32 mg/dL) [100 mg dose]
Serum HDL-C increases were maintained through the end of study, with week 16 mean increases of 28% (12 mg/dL) [10 mg dose] to 52% (22 mg/dL) [100 mg dose]
Multiple dose evaluations in patients with severe hypertriglyceridemia and/or familial chylomicronemia syndrome are underway
RNA Interference Targeting Hepatic Angiopoietin-Like Protein 3 Results in Prolonged Reductions in Plasma Triglycerides and LDL-C in Human Subjects
Session: Late Breaking Science VI: New Frontiers in Lipid Therapy
Date and Time: November 18, 2019 from 9:38 AM EST
Authors: Dr. Gerald Watts, et al.
Key points presented on the AROANG1001 Phase 1/2a clinical study included the following:
Safety and tolerability
40 subjects enrolled to receive a single dose (24 active, 16 placebo)
No drug related severe or serious AEs
Two AEs of mild transient elevations in ALT (one active, one placebo)
ALT elevation in one subject on ARO-ANG3 confounded by concomitant ingestion of herbal supplement with known liver toxic profile (Peak ALT 192 U/L Day 99, normal by Day 113)
1 mild drug related LISR
Activity
Dose dependent reductions in serum ANGPTL3 were observed
Mean maximum reduction from baseline in ANGPTL3 ranged from 55% (50 ng/mL) [35 mg] to 83% (63 ng/mL) [300 mg]
Reductions in ANGPTL3 were maintained through end of study, with week 16 mean reductions of 43% (42 ng/mL) [35 mg] to 75% (57 ng/mL) [300 mg]
Dose dependent reductions in TGs and VLDL-C were observed
Mean maximum TG reduction from baseline of 31% (38 mg/dL) [35 mg] to 66% (167 mg/dL) [200 mg]
Mean maximum VLDL-C reduction from baseline of 30% (8 mg/dL)[35 mg] to 65% (33 mg/dL) [200 mg]
Reduction in TG and VLDL-C maintained through end of study in 200 mg and 300 mg cohorts, with week 16 mean reductions of 47% to 53% for TG, and 49% to 51% for VLDL-C
Changes in LDL- and HDL- cholesterol were observed
Mean maximum HDL-C reduced by 8% (4 mg/dL) [35 mg] to 26% (12 mg/dL) [300 mg]
HDL-C mean reductions at week 16 of up to 16% (7 mg/dL) [200 mg]
Mean maximum LDL-C reduced by 9% (16 mg/dL) [200 mg] to 30% (48 mg/dL) [300 mg]
LDL-C mean reductions at week 16 of up to 28% (46 mg/dL) [100 mg] after single dose
Mean maximum reduction in LDL-C with 200 mg single dose was blunted by two subjects in this cohort with increasing LDL-C post-dose
Multiple dose healthy volunteer data at 200 mg dose demonstrates similar reductions to 100 mg and 300 mg doses of 33%-46% reduction in LDL-C from baseline two weeks after a second dose
Multiple dose evaluations in patients with non-alcoholic fatty liver disease (NAFLD) (cohort 5), hyperlipidemia while on statins (cohort 6), familial hypercholesterolemia (cohort 7), severe hypertriglyceridemia (cohort 8) are underway
Copies of the presentations can be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

seekingalpha.com/pr/17702466-arrowhea...

drulletje drie 18 november 2019 16:08

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seekingalpha.com/article/4307438-arro...

DeZwarteRidder 18 november 2019 16:11

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quote:
drulletje drie schreef op 18 november 2019 16:08:
seekingalpha.com/article/4307438-arro...

Conclusion: I'm waiting to see which way the share price moves ahead of next results

This is far from the best time to be buying Arrowhead. Investors who bought at the beginning of 2019 must be rubbing their hands in glee. But it could be that we have reached saturation point.

On the other hand, if RNAi drugs start to crush it in phase 3 clinical trials and are approved for commercialization, we could see Arrowhead stock disappear beyond the $100 mark.

Waiting until after the next set of results will mean losing out if they prove to be another catalyst for dramatic share price gain. Hence, I will be watching closely for signs of progress, and plan to invest, all being well, shortly before the next set of results, due on November 25th.

drulletje drie 18 november 2019 16:17

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quote:
DeZwarteRidder schreef op 18 november 2019 16:11:
[...]

Conclusion: I'm waiting to see which way the share price moves ahead of next results

This is far from the best time to be buying Arrowhead. Investors who bought at the beginning of 2019 must be rubbing their hands in glee. But it could be that we have reached saturation point.

On the other hand, if RNAi drugs start to crush it in phase 3 clinical trials and are approved for commercialization, we could see Arrowhead stock disappear beyond the $100 mark.

Waiting until after the next set of results will mean losing out if they prove to be another catalyst for dramatic share price gain. Hence, I will be watching closely for signs of progress, and plan to invest, all being well, shortly before the next set of results, due on November 25th.

Kan het wel volgen maar hij noemt niet dat er een heleboel shorts nog met hun handen in het haar zitten. Vraag is wat die gaan doen denk?

geluk_verder 18 november 2019 17:42

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Kan wel zijn dat we enkele weken vlak gaan liggen. Is niet erg me dunkt.

Hulskof 18 november 2019 18:08

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quote:
DeZwarteRidder schreef op 18 november 2019 16:11:
[...]

Conclusion: I'm waiting to see which way the share price moves ahead of next results

This is far from the best time to be buying Arrowhead. Investors who bought at the beginning of 2019 must be rubbing their hands in glee. But it could be that we have reached saturation point.

On the other hand, if RNAi drugs start to crush it in phase 3 clinical trials and are approved for commercialization, we could see Arrowhead stock disappear beyond the $100 mark.

Waiting until after the next set of results will mean losing out if they prove to be another catalyst for dramatic share price gain. Hence, I will be watching closely for signs of progress, and plan to invest, all being well, shortly before the next set of results, due on November 25th.

De man gelooft er dus in, maar wacht nog een week voor hij instapt, uit vrees voor een correctie.

[verwijderd] 18 november 2019 22:48

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quote:
DeZwarteRidder schreef op 14 november 2019 20:31:
Heeft unieke manier gevonden om Alzheimer te genezen:
Dus kleine kans op enorme winsten:
------------------------------------------------------------------
Cortexyme Inc Company Profile

Cortexyme, Inc. is a clinical-stage biopharmaceutical company. The Company is focused on providing disease-modifying therapeutics to treat Alzheimer’s and other degenerative diseases. The Company is focused on a infectious pathogen tied to neurodegeneration in humans and animal models. The Company’s lead drug candidate, COR388, is an orally-administered, brain-penetrating small molecule gingipain inhibitor designed for the treatment of Alzheimer’s disease. COR388 is designed to target an upstream driver of multiple Alzheimer’s pathological pathways, including amyloid beta production, inflammation and neurodegeneration, in contrast to mechanisms of action targeting downstream effects, such as amyloid plaques and tau tangles.

Zag CRTX vanavond van 25 naar begin 29$ klimmen maar kan nog geen reden vinden waarom.

nelis h 19 november 2019 16:11

1

weer een fijn dagje
2 a 3 jaartjes wachten op Market cap 20B en dan pas afbouwen overwegen :)

drulletje drie 19 november 2019 16:18

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quote:
nelis h schreef op 19 november 2019 16:11:
weer een fijn dagje
2 a 3 jaartjes wachten op Market cap 20B en dan pas afbouwen overwegen :)

Ik teken er echt voor maar ik vrees dat we binnen die termijn overgenomen zijn door J&J !

RW1963 20 november 2019 17:48

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ai, ai, we zakken weer
(ik zeg dit omdat we de vorige keer weer stegen toen ik dit zei :-) )

RW1963 20 november 2019 19:08

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Wat is er aan de hand? Al > 6% daling :-(

Hulskof 20 november 2019 22:55

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Gewoon een gezonde correctie.

[verwijderd] 20 november 2019 23:00

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Yep, just another day at the office.
Een beetje van Maggi en een beetje van Trump.
Mag nog iets zakken svp, wil wel weer een ritje maken maar niet al te duur.

nelis h 21 november 2019 10:43

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ARO-HIF gaat naar de kliniek, dit is een mijlpaal, de 1ste RNA "buiten de lever"

clinicaltrials.gov/ct2/show/NCT041697...

Tom3 24 november 2019 01:03

1

Novartus doet $ 10 miljard bod op een door Alnylam ontwikkelde cholesterol-verlager. Arrowhead heeft een soortgelijk product, maar dan beter volgens velen. Het worden mooie tijden voor de RNAi sector:

www.reuters.com/article/us-medicines-...

DeZwarteRidder 24 november 2019 09:51

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quote:
Tom3 schreef op 24 november 2019 01:03:
Novartus doet $ 10 miljard bod op een door Alnylam ontwikkelde cholesterol-verlager. Arrowhead heeft een soortgelijk product, maar dan beter volgens velen. Het worden mooie tijden voor de RNAi sector:

November 23, 2019 / 8:32 PM / Updated 13 hours ago
Novartis nears deal to buy U.S. biotech firm Medicines Co for about $7 billion: WSJ

2 Min Read
(Reuters) - Swiss drugmaker Novartis AG (NOVN.S) is nearing an agreement to acquire U.S. biotechnology firm The Medicines Co (MDCO.O) for about $7 billion, the Wall Street Journal reported on Saturday.

The deal, in which Novartis has agreed to pay $85 a share, could be announced this weekend, the Journal reported, citing people familiar with the matter.

Going by a fully diluted share count, the agreement is worth about $10 billion, according to the report.

Novartis declined to comment. The Medicines Co did not respond to a request for comment on Saturday.

The deal could broaden the Swiss drugmaker’s cabinet of heart medicines and shore up growth threatened by patent expirations.

Novartis has been hunting for a $5 billion acquisition in the United States, two banking sources told Reuters earlier this week without identifying a target.

New Jersey-based The Medicines Co’s top drug candidate is cholesterol-lowering drug inclisiran for heart patients. Novartis has historically had a strong cardiovascular drug franchise, but lost ground when Diovan, once a $6 billion-per-year seller, lost patent protection in 2012 and left the company without an immediate, innovative follow-up product.

Novartis has since been building up its portfolio, which now includes Entresto, a $1 billion seller for heart failure, as well as an experimental RNA-targeting molecule from Ionis Pharmaceuticals (IONS.O) that it licensed earlier this year for $150 million.

Reporting by Kanishka Singh in Bengaluru; editing by Jonathan Oatis and Chizu Nomiyama

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